Abeona Therapeutics (ABEO) 10-K 16 Mar 20 2019 FY Annual report Financial data

Document and Entity Information

Document and Entity Information - USD ($)	12 Months Ended
	Dec. 31, 2019	Mar. 09, 2020	Jun. 30, 2019
Document and Entity Information
Entity Registrant Name	ABEONA THERAPEUTICS INC.
Entity Central Index Key	0000318306
Document Type	10-K
Document Period End Date	Dec. 31, 2019
Amendment Flag	false
Current Fiscal Year End Date	--12-31
Entity a Well-known Seasoned Issuer	No
Entity a Voluntary Filer	No
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Accelerated Filer
Entity Small Business Flag	true
Entity Emerging Growth Company	false
Entity Shell Company	false
Entity Public Float			$ 131,277,000
Entity Common Stock, Shares Outstanding		83,622,135
Document Fiscal Period Focus	FY
Document Fiscal Year Focus	2019

Consolidated Balance Sheets

Consolidated Balance Sheets - USD ($)	Dec. 31, 2019	Dec. 31, 2018
Current assets:
Cash and cash equivalents	$ 129,258,000	$ 18,750,000
Short-term investments		66,218,000
Receivables		81,000
Prepaid expenses and other current assets	3,132,000	3,802,000
Total current assets	132,390,000	88,851,000
Property and equipment, net	13,157,000	9,443,000
Right-of-use lease assets	8,047,000
Licensed technology, net	36,178,000	43,042,000
Goodwill	32,466,000	32,466,000
Other assets and restricted cash	1,144,000	597,000
Total assets	223,382,000	174,399,000
Current liabilities:
Accounts payable	3,763,000	6,122,000
Accrued expenses	5,543,000	3,936,000
Current portion of lease liability	1,699,000
Current portion of payable to licensor	27,400,000	10,000,000
Deferred revenue	296,000	296,000
Total current liabilities	38,701,000	20,354,000
Long-term lease liabilities	6,251,000
Payable to licensor, net of current portion		20,000,000
Total liabilities	44,952,000	40,354,000
Commitments and contingencies
Stockholders' equity:
Common stock - $0.01 par value; authorized 200,000,000 shares; issued and outstanding 83,622,135 at December 31, 2019; issued and outstanding 47,944,486 at December 31, 2018	836,000	479,000
Additional paid-in capital	664,064,000	543,754,000
Accumulated deficit	(486,470,000)	(410,188,000)
Total stockholders' equity	178,430,000	134,045,000
Total liabilities and stockholders' equity	$ 223,382,000	$ 174,399,000

Consolidated Balance Sheets (Pa

Consolidated Balance Sheets (Parenthetical) - $ / shares	Dec. 31, 2019	Dec. 31, 2018
Statement of Financial Position [Abstract]
Common stock, par value	$ 0.01	$ 0.01
Common stock, shares authorized	200,000,000	200,000,000
Common stock, shares issued	83,622,135	47,944,486
Common stock, shares outstanding	83,622,135	47,944,486

Consolidated Statements of Oper

Consolidated Statements of Operations - USD ($)	12 Months Ended
	Dec. 31, 2019	Dec. 31, 2018	Dec. 31, 2017
Revenues:
Total revenues		$ 2,998,000	$ 837,000
Expenses:
Research and development	48,566,000	38,698,000	16,989,000
General and administrative	20,705,000	20,106,000	10,943,000
Depreciation and amortization	7,819,000	2,360,000	741,000
Total expenses	77,090,000	61,164,000	28,673,000
Loss from operations	(77,090,000)	(58,166,000)	(27,836,000)
Interest and miscellaneous income	1,208,000	1,506,000	525,000
Interest and other expense	(400,000)	(11,000)	(8,000)
Net loss	$ (76,282,000)	$ (56,671,000)	$ (27,319,000)
Basic and diluted loss per common share	$ (1.51)	$ (1.19)	$ (0.66)
Weighted average number of common shares outstanding - basic and diluted	50,354,596	47,528,248	41,636,752
Foundation Revenues [Member]
Revenues:
Total revenues		$ 2,796,000
License Revenues[Member]
Revenues:
Total revenues			602,000
Royalties [Member]
Revenues:
Total revenues		$ 202,000	$ 235,000

Consolidated Statements of Stoc

Consolidated Statements of Stockholders' Equity - USD ($)	Common Stock [Member]	Additional Paid-in Capital [Member]	Accumulated Deficit [Member]	Total
Beginning balance at Dec. 31, 2016	$ 403,000	$ 431,168,000	$ (332,473,000)	$ 99,098,000
Beginning balance, shares at Dec. 31, 2016	40,254,457
Stock option-based compensation expense		4,644,000		4,644,000
Restricted stock-based compensation expense		1,272,000		1,272,000
Issuance of common stock in connection with public offering, net of offering costs	$ 58,000	86,116,000		86,174,000
Issuance of common stock in connection with public offering, net of offering costs, shares	5,750,000
Issuance of common stock in connection with the exercise of stock options		344,000		344,000
Issuance of common stock in connection with the exercise of stock options, shares	81,719
Issuance of common stock in connection with the exercise of $8.00 warrants	$ 6,000	4,994,000		5,000,000
Issuance of common stock in connection with the exercise of $8.00 warrants, shares	625,000
Issuance of common stock in connection with the exercise of $5.00 warrants	$ 2,000	883,000		885,000
Issuance of common stock in connection with the exercise of $5.00 warrants, shares	176,932
Net loss			(27,319,000)	(27,319,000)
Ending balance at Dec. 31, 2017	$ 469,000	529,421,000	(359,792,000)	170,098,000
Ending balance, shares at Dec. 31, 2017	46,888,108
Cumulative effect adjustment of ASC 606 on January 1, 2018			6,275,000	6,275,000
Stock option-based compensation expense		8,178,000		8,178,000
Restricted stock-based compensation expense		688,000		688,000
Issuance of common stock in connection with the exercise of stock options	$ 4,000	2,240,000		2,244,000
Issuance of common stock in connection with the exercise of stock options, shares	360,853
Issuance of common stock in connection with the exercise of $5.00 warrants	$ 6,000	3,227,000		3,233,000
Issuance of common stock in connection with the exercise of $5.00 warrants, shares	646,763
Issuance of common stock in connection with cashless warrant exercises
Issuance of common stock in connection with cashless warrant exercises, shares	48,762
Net loss			(56,671,000)	(56,671,000)
Ending balance at Dec. 31, 2018	$ 479,000	543,754,000	(410,188,000)	134,045,000
Ending balance, shares at Dec. 31, 2018	47,944,486
Stock option-based compensation expense		7,338,000		7,338,000
Restricted stock-based compensation expense		899,000		899,000
Issuance of common stock and pre-funded warrants in connection with public offering, net of offering costs	$ 324,000	95,648,000		95,972,000
Issuance of common stock and pre-funded warrants in connection with public offering, net of offering costs, shares	32,382,945
Issuance of common stock under open market sale agreement	$ 31,000	16,930,000		16,961,000
Issuance of common stock under open market sale agreement, shares	3,086,950
Issuance of common stock in connection with the exercise of stock options	$ 3,000	966,000		969,000
Issuance of common stock in connection with the exercise of stock options, shares	303,129
Issuance of common stock in connection with restricted share awards	$ 4,000	(4,000)
Issuance of common stock in connection with restricted share awards, shares	354,625
Shares returned in connection with arbitration ruling on licensing agreement	$ (5,000)	(1,467,000)		(1,472,000)
Shares returned in connection with arbitration ruling on licensing agreement, shares	(450,000)
Net loss			(76,282,000)	(76,282,000)
Ending balance at Dec. 31, 2019	$ 836,000	$ 664,064,000	$ (486,470,000)	$ 178,430,000
Ending balance, shares at Dec. 31, 2019	83,622,135

Consolidated Statements of St_2

Consolidated Statements of Stockholders' Equity (Parenthetical) - $ / shares	Dec. 31, 2018	Dec. 31, 2017
Class of warrant or right, exercise price of warrants or rights	$ 5	$ 5
Warrants One [Member]
Class of warrant or right, exercise price of warrants or rights	$ 5	8
Warrants Two [Member]
Class of warrant or right, exercise price of warrants or rights		$ 5

Consolidated Statements of Cash

Consolidated Statements of Cash Flows - USD ($)	12 Months Ended
	Dec. 31, 2019	Dec. 31, 2018	Dec. 31, 2017
Cash flows from operating activities:
Net loss	$ (76,282,000)	$ (56,671,000)	$ (27,319,000)
Adjustments to reconcile net loss to cash used in operating activities:
Depreciation and amortization	7,819,000	2,360,000	741,000
Stock option-based compensation expense	7,338,000	8,178,000	4,644,000
Restricted stock-based compensation expense	899,000	688,000	1,272,000
Accretion and interest on short-term investments	(266,000)	(626,000)
Accretion of right-of-use lease assets	858,000
Net gain on write-off of licensed technology			(127,000)
Non-cash loss on arbitration ruling on licensing agreement	367,000
Change in operating assets and liabilities:
Receivables	81,000	26,000	17,000
Prepaid expenses and other current assets	670,000	(1,067,000)	(2,580,000)
Other assets	3,000	40,000	(11,000)
Accounts payable, accrued expenses and lease liabilities	(1,707,000)	7,665,000	(1,301,000)
Payable to licensor	(2,600,000)
Deferred revenue		296,000	2,009,000
Net cash used in operating activities	(62,820,000)	(39,111,000)	(22,655,000)
Cash flows from investing activities:
Capital expenditures	(6,309,000)	(9,243,000)	(860,000)
Acquisition of licensed technology	(199,000)	(10,251,000)
Purchases of short-term investments		(136,092,000)
Proceeds from maturities of short-term investments	66,484,000	70,500,000
Net cash provided by (used in) investing activities	59,976,000	(85,086,000)	(860,000)
Cash flows from financing activities:
Proceeds from issuance of common stock and pre-funded warrants in public offering, net of offering costs	95,972,000
Proceeds from issuance of common stock in public offering, net of offering costs			86,174,000
Proceeds from open market sales of common stock	16,961,000
Proceeds from exercise of $5.00 warrants		3,233,000	885,000
Proceeds from exercise of stock options	969,000	2,244,000	344,000
Proceeds from exercise of $8.00 warrants			5,000,000
Net cash provided by financing activities	113,902,000	5,477,000	92,403,000
Net increase/(decrease) in cash, cash equivalents and restricted cash	111,058,000	(118,720,000)	68,888,000
Cash, cash equivalents and restricted cash at beginning of year	19,310,000	138,030,000	69,142,000
Cash, cash equivalents and restricted cash at end of year	130,368,000	19,310,000	138,030,000
Supplemental cash flow information:
Cash and cash equivalents	129,258,000	18,750,000	137,750,000
Restricted cash	1,110,000	560,000	280,000
Total cash, cash equivalents and restricted cash	130,368,000	19,310,000	138,030,000
Payable for acquisition of licensed technology		30,000,000
Write off of licensed asset and corresponding liability			4,000,000
Shares returned in connection with arbitration ruling on licensing agreement	1,472,000
Cash paid for interest		$ 11,000	$ 8,000

Consolidated Statements of Ca_2

Consolidated Statements of Cash Flows (Parenthetical) - $ / shares	Dec. 31, 2019	Dec. 31, 2018	Dec. 31, 2017
Statement of Cash Flows [Abstract]
Class of warrant or right, exercise price of warrants or rights	$ 5	$ 5	$ 5
Class of warrant or right, exercise price of warrants or rights	$ 8	$ 8	$ 8

Nature of Operations and Summar

Nature of Operations and Summary of Significant Accounting Policies	12 Months Ended
	Dec. 31, 2019
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Nature of Operations and Summary of Significant Accounting Policies	NOTE 1 - NATURE OF OPERATIONS AND SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES Nature of Operations Abeona Therapeutics Inc. (together with our subsidiaries, “we,” “our,” “Abeona” or the “Company”), a Delaware corporation, is a clinical-stage biopharmaceutical company developing gene and cell therapies for life-threatening rare genetic diseases. Our lead clinical programs consist of: (i) EB-101, an autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa (“RDEB”), (ii) ABO-102, an adeno-associated virus (“AAV”)-based gene therapy for Sanfilippo syndrome type A (“MPS IIIA”) and (iii) ABO-101, an AAV-based gene therapy for Sanfilippo syndrome type B (“MPS IIIB”). We have additional AAV-based gene therapies in various developmental stages designed to treat the CLN1 and CLN3 forms of Batten Disease, cystic fibrosis and retinal diseases. In addition, we are developing next-generation AAV-based gene therapies through our novel AIM™ capsid platform and internal AAV vector research programs. A summary of the significant accounting policies applied in the preparation of the accompanying consolidated financial statements follows: Principles of Consolidation The consolidated financial statements include the financial statements of Abeona Therapeutics Inc. and our wholly-owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. Uses and Sources of Liquidity The financial statements have been prepared on the going concern basis, which assumes the Company will have sufficient cash to pay its operating expenses, as and when they become payable, for a period of at least 12 months from the date the financial report was issued. As of December 31, 2019, we had cash and cash equivalents of $129.3 million and net assets of $178.4 million. For the year ended December 31, 2019, we had cash outflows from operations of $62.8 million. We have not generated any significant product revenues and have not achieved profitable operations. There is no assurance that profitable operations will ever be achieved, and, if achieved, could be sustained on a continuing basis. In addition, development activities, clinical and nonclinical testing, and commercialization of our products will require significant additional financing. We are subject to a number of risks similar to other life science companies, including, but not limited to, risks related to the successful discovery and development of product candidates, obtaining the necessary regulatory approval to market our product candidates, raising additional capital, development of competing drugs and therapies, protection of proprietary technology and market acceptance of our products. As a result of these and other risks and the related uncertainties, there can be no assurance of our future success. Based upon our current operating plans, we believe that we have sufficient resources to fund operations through the second quarter of 2021 with our existing cash and cash equivalents. We will need to secure additional funding in the future, to carry out all of our planned research and development activities. If we are unable to obtain additional financing or generate license or product revenue, the lack of liquidity and sufficient capital resources could have a material adverse effect on our future prospects. Use of Estimates The preparation of consolidated financial statements in conformity with accounting principles generally accepted in the United States of America (“U.S. GAAP”) requires management to make estimates and assumptions that affect the reported amount of assets and disclosure of contingent assets and liabilities at the date of the consolidated financial statements and the reported amounts of revenue and expenses during the reported period. Actual results could differ from these estimates and assumptions. Cash and Cash Equivalents We consider all highly liquid investments with a maturity of three months or less when purchased to be cash equivalents. We maintain deposits primarily in financial institutions, which may at times exceed amounts covered by insurance provided by the U.S. Federal Deposit Insurance Corporation (“FDIC”). We have not experienced any losses related to amounts in excess of FDIC limits. Short-term Investments Short-term investments consist of investments in U.S. government, U.S. agency and U.S. treasury securities. We determine the appropriate classification of the securities at the time they are acquired and evaluate the appropriateness of such classifications at each balance sheet date. We classify our short-term investments as available-for-sale pursuant to Accounting Standards Codification (“ASC”) 320, Investments – Debt and Equity Securities We review our short-term investments for other-than-temporary impairment whenever the fair value of a marketable security is less than the amortized cost and evidence indicates that a short-term investment’s carrying amount is not recoverable within a reasonable period of time. Receivables Receivables are reported in the consolidated balance sheets at net realizable value. We continually evaluate the creditworthiness of our customers and their financial condition and generally do not require collateral. The allowance for doubtful accounts is based upon reviews of specific customer balances, historic losses, and general economic conditions. As of December 31, 2019 and 2018, no allowance was recorded as all accounts are considered collectible. There were no receivables outstanding as of December 31, 2019. Property and Equipment Property and equipment are recorded at cost. Depreciation is provided using the straight-line method over estimated useful lives ranging from three to seven years for equipment and five to ten years for leasehold improvements. Expenditures for major renewals and betterments that extend the useful lives are capitalized. Expenditures for normal maintenance and repairs are expensed as incurred. The cost of assets sold or abandoned, and the related accumulated depreciation are eliminated from the accounts and any gains or losses are recognized in the accompanying consolidated statements of operations of the respective period. Leases In February 2016, the Financial Accounting Standards Board issued Accounting Standards Update (“ASU”) 2016-02, Leases Leases Right-of-use lease assets represent our right to use an underlying asset for the lease term and lease liabilities represent our obligation to make lease payments arising from the lease. The measurement of lease liabilities is based on the present value of future lease payments over the lease term. As our leases do not provide an implicit rate, we use our incremental borrowing rate based on the information available at the lease commencement date in determining the present value of future lease payments. The right-of-use asset is based on the measurement of the lease liability and includes any lease payments made prior to or on lease commencement and excludes lease incentives and initial direct costs incurred, as applicable. Rent expense for our operating leases is recognized on a straight-line basis over the lease term. We do not have any leases classified as finance leases. Our leases do not have significant rent escalation, holidays, concessions, material residual value guarantees, material restrictive covenants or contingent rent provisions. Our leases include both lease (e.g., fixed payments including rent, taxes, and insurance costs) and non-lease components (e.g., common-area or other maintenance costs), which are accounted for as a single lease component as we have elected the practical expedient to group lease and non-lease components for all leases. Most leases include one or more options to renew. The exercise of lease renewal options is typically at our sole discretion; therefore, the majority of renewals to extend the lease terms are not included in our right-of-use assets and lease liabilities as they are not reasonably certain of exercise. We regularly evaluate the renewal options and when they are reasonably certain of exercise, we include the renewal period in our lease term. Additional information and disclosures required under ASC 842 are included in Note 6. Licensed Technology We have entered into agreements to license the rights to certain technologies. We recorded the purchase price paid for the license, which represents fair value, on our consolidated balance sheet. We maintain licensed technology on our consolidated balance sheet until either the licensed technology agreement underlying it is completed or the asset becomes impaired. When we determine that an asset has become impaired or we abandon a project, we write down the carrying value of the related intangible asset to its fair value and take an impairment charge in the period in which the impairment occurs. Licensed technology is amortized over the life of the patent or the agreement and periodically reviewed for impairment. We test our intangible assets for impairment on an annual basis, or more frequently if indicators are present or changes in circumstance suggest that impairment may exist. Events that could result in an impairment, or trigger an interim impairment assessment, include the receipt of additional clinical or nonclinical data regarding our drug candidate or a potentially competitive drug candidate, changes in the clinical development program for a drug candidate or new information regarding potential sales for the drug. In connection with each annual impairment assessment and any interim impairment assessment, we compare the fair value of the asset as of the date of the assessment with the carrying value of the asset on our consolidated balance sheet. In 2019, 2018 and 2017, we did not impair any licensed technology. Goodwill As of December 31, 2019 and 2018, goodwill of $32.5 million was recorded on the Company’s consolidated balance sheet. In accordance with ASC 350, Intangibles — Goodwill and Other, Restricted Cash In November 2016, the Financial Accounting Standards Board issued ASU 2016-18, Statement of Cash Flows (Topic 230): Restricted Cash Segments The Company operates in a single segment. The Company’s chief operating decision maker, its Executive Chairman, manages the Company’s operations on a consolidated basis for the purpose of allocating resources. Revenue Recognition Effective January 1, 2018, we adopted Accounting Standards Update (“ASU”) 2014-09, Revenue from Contracts with Customers Revenue Recognition The table below presents the impact of the adoption of ASC 606 on our statement of operations for the year ended December 31, 2018. For the year ended December 31, 2018 Under Effect of As Reported ASC 605 ASC 606 Under ASC 606 Revenues Foundation revenues $ - $ 2,796,000 $ 2,796,000 License revenues 602,000 (602,000 ) - Total revenues 804,000 2,194,000 2,998,000 Loss from operations (60,360,000 ) 2,194,000 (58,166,000 ) Net loss (58,865,000 ) 2,194,000 (56,671,000 ) Basic and diluted loss per common share (1.24 ) 0.05 (1.19 ) Foundation revenues: As a result of the adoption of ASC 606, we decreased deferred revenue and the accumulated deficit by $2.6 million as of January 1, 2018. As of both December 31, 2018 and 2019, we received $5.7 million of the grants. In accordance with ASC 606, we record revenue to match expenses for the advancement of the Company’s clinical stage gene therapies for MPS IIIA and MPS IIIB, the production of which is the primary performance obligation. We recorded foundation revenues of $0 in 2019 and $2.8 million in 2018. We allocated the transaction price based on the expected costs plus a margin approach. The aggregate amount of the transaction price allocated to the performance obligations that are unsatisfied as of December 31, 2019 and 2018 was $0.3 million. We expect the unsatisfied performance obligations to be recognized in the next 12 months. License revenues: Revenue from Contracts with Customers (Topic 606): Identifying Performance Obligations and Licensing Royalty revenues: . Research and Development Expenses Research and development costs are expensed as incurred. Research and development expenses include, but are not limited to, payroll and personnel expense, lab supplies, preclinical, development cost, clinical trial expense, outside manufacturing and consulting. The cost of materials and equipment or facilities that are acquired for research and development activities and that have alternative future uses are capitalized when acquired. General and Administrative Expenses General and administrative expenses primarily consist of personnel, contract personnel, personnel-related expenses to support our administrative and operating activities, facility costs and professional expenses (i.e., legal expenses) and investor relations fees. Income Taxes Income taxes are accounted for under the asset and liability method. Deferred tax assets and liabilities are recognized for the future tax consequences attributable to differences between the consolidated financial statement carrying amounts of existing assets and liabilities and their respective tax bases and operating loss and tax credit carryforwards. Deferred tax assets and liabilities are measured using enacted tax rates expected to apply to taxable income in the years in which those temporary differences are expected to be recovered or settled. The effect on deferred tax assets and liabilities of a change in tax rates is recognized in income in the period that includes the enactment date. A valuation allowance is provided for deferred tax assets to the extent their realization is in doubt. We account for uncertain income tax positions in accordance with ASC 740, Income Taxes Loss Per Common Share We have presented basic and diluted loss per common share on the statement of operations. Basic and diluted net loss per share is computed by dividing net loss by the weighted-average number of shares of common stock and shares underlying “pre-funded” warrants outstanding during the period. The “pre-funded” warrants are included in the computation of basic net loss per share as the exercise price is negligible and they are fully vested and exercisable. We do not include the potential impact of dilutive securities in diluted net loss per share, as the impact of these items is anti-dilutive. Potential dilutive securities result from outstanding stock options and “non-pre-funded” warrants. We did not include the following potentially dilutive securities in the computation of diluted net loss per common share during the periods presented: For the years ended December 31, 2019 2018 2017 Warrants 70,000 1,820,686 2,934,685 Stock options 6,055,395 5,841,805 5,429,727 Total 6,125,395 7,662,491 8,364,412 Stock-Based Compensation We account for stock-based compensation expense in accordance with ASC 718, Stock Based Compensation The following table summarizes stock option-based option compensation for 2019, 2018 and 2017, which was allocated as follows: For the years ended December 31, 2019 2018 2017 Research and development $ 3,932,000 $ 3,913,000 $ 1,668,000 General and administrative 3,406,000 4,265,000 2,976,000 Stock option-based compensation expense included in operating expense 7,338,000 8,178,000 4,644,000 Total stock option-based compensation expense 7,338,000 8,178,000 4,644,000 Tax benefit - - - Stock option-based compensation expense, net of tax $ 7,338,000 $ 8,178,000 $ 4,644,000 The following table summarizes restricted stock-based compensation for 2019, 2018 and 2017, which was allocated as follows: For the years ended December 31, 2019 2018 2017 Research and development $ 454,000 $ - $ - General and administrative 445,000 688,000 1,272,000 Restricted stock-based compensation expense included in operating expense 899,000 688,000 1,272,000 Total restricted stock-based compensation expense 899,000 688,000 1,272,000 Tax benefit - - - Restricted stock-based compensation expense, net of tax $ 899,000 $ 688,000 $ 1,272,000

Short-Term Investments

Short-Term Investments	12 Months Ended
	Dec. 31, 2019
Investments, Debt and Equity Securities [Abstract]
Short-Term Investments	NOTE 2 – SHORT-TERM INVESTMENTS The following table summarizes the available-for-sale investments held as of December 31, 2018. There were no available-for-sale investments as of December 31, 2019. Description Fair value U.S. government and agency securities and treasuries $ 66,218,000 The amortized cost of the available-for-sale investments is adjusted for amortization of premiums and accretion of discounts to maturity. There were no material realized gains or losses recognized on the sale or maturity of available-for-sale investments during the years ended December 31, 2019 or 2018.

Property and Equipment

Property and Equipment	12 Months Ended
	Dec. 31, 2019
Property, Plant and Equipment [Abstract]
Property and Equipment	NOTE 3 - PROPERTY AND EQUIPMENT Property and equipment consist of the following: December 31, 2019 December 31, 2018 Laboratory equipment $ 7,031,000 $ 4,366,000 Furniture and office equipment 1,710,000 1,454,000 Leasehold improvements 8,573,000 4,774,000 Construction work-in-progress - 409,000 17,314,000 11,003,000 Less: accumulated depreciation and amortization 4,157,000 1,560,000 Property and equipment, net $ 13,157,000 $ 9,443,000 Depreciation and amortization on property and equipment was $2.6 million, $1.2 million and $0.2 million for 2019, 2018 and 2017, respectively.

Licensed Technology

Licensed Technology	12 Months Ended
	Dec. 31, 2019
Goodwill and Intangible Assets Disclosure [Abstract]
Licensed Technology	NOTE 4 – LICENSED TECHNOLOGY On November 4, 2018, we entered into a license agreement with REGENXBIO to obtain rights to an exclusive worldwide license (subject to certain non-exclusive rights previously granted for MPS IIIA), with rights to sublicense, to REGENXBIO’s NAV AAV9 vector for the development and commercialization of gene therapies for the treatment of MPS IIIA, MPS IIIB, CLN1 Disease and CLN3 Disease. In return for these rights, REGENXBIO received a guaranteed $20 million upfront payment, $10 million of which was paid on signing of the agreement on November 4, 2018 and $10 million of which was originally required under the agreement to be paid by November 4, 2019. In addition, REGENXBIO will receive a total of $100 million in annual fees, payable upon the second through sixth anniversaries of the agreement, $20 million of which is guaranteed and payable on November 4, 2020. REGENXBIO is also eligible to receive potential commercial milestone payments of up to $60 million as well as royalties payable in the low double digits to low teens on net sales of products incorporating the licensed intellectual property. The license is amortized over the life of the patent of eight years. On November 1, 2019, we entered into an amendment of the original license agreement. The amended agreement replaced the $10 million payment due on November 4, 2019 with a $3 million payment due on November 4, 2019 and an additional $8 million payment (which includes $1 million of interest) due on April 1, 2020. The payment due on April 1, 2020 and the guaranteed amount of $20 million due on November 4, 2020 are recorded as payable to licensor on the consolidated balance sheet. On August 3, 2016, we announced that we entered into an agreement (the “EB Agreement”) with EB Research Partnership (“EBRP”) and Epidermolysis Bullosa Medical Research Foundation (“EBMRF”) to collaborate on gene therapy treatments for EB. The EB Agreement became effective August 3, 2016 on the execution of two licensing agreements with The Board of Trustees of Leland Stanford Junior University (“Stanford”). On August 3, 2016, we recorded the issuance of 375,000 of our common shares to each of EBRP and EBMRF and recorded licensed technology of $2.45 million, which was being amortized over 20 years. In connection with an arbitration proceeding relating to the EB Agreement, on May 15, 2019, the arbitrator issued a decision in favor of the Company requiring the Company to cancel any and all shares of its common stock issued to EBRP and EBMRF that were still in their possession. As a result, we recorded the return of 450,000 shares of our common stock and the reversal of the licensed technology from our financial statements. The net of these transactions resulted in a non-cash charge to expense of $0.3 million recorded during the year ended December 31, 2019. On May 15, 2015, we acquired Abeona Therapeutics LLC, which had an exclusive license through Nationwide Children’s Hospital to the AB-101 and AB-102 patent portfolios for developing treatments for patients with Sanfilippo Syndrome Type A and Type B. The license is amortized over the life of the license of 20 years. Licensed technology consists of the following: December 31, 2019 December 31, 2018 Licensed technology $ 42,606,000 $ 44,859,000 Less accumulated amortization 6,428,000 1,817,000 Licensed technology, net $ 36,178,000 $ 43,042,000 The aggregate estimated amortization expense for intangible assets remaining as of December 31, 2019 is as follows: 2020 $ 5,174,000 2021 5,174,000 2022 5,174,000 2023 5,174,000 2024 5,174,000 Thereafter 10,308,000 Total $ 36,178,000 Amortization on licensed technology was $5.2 million, $1.2 million and $0.5 million for the years ended December 31, 2019, 2018 and 2017, respectively.

401(k) Plan

401(k) Plan	12 Months Ended
	Dec. 31, 2019
Retirement Benefits [Abstract]
401(k) Plan	NOTE 5 – 401(k) PLAN We have a tax-qualified employee savings and retirement plan (the “401(k) Plan”) covering all our employees. Pursuant to the 401(k) Plan, employees may elect to reduce their current compensation by up to the statutorily prescribed annual limit ($19,000 in 2019, $18,500 in 2018 and $18,000 in 2017) and to have the amount of such reduction contributed to the 401(k) Plan. The 401(k) Plan is intended to qualify under Section 401 of the Internal Revenue Code so that contributions by employees or by us to the 401(k) Plan, and income earned on 401(k) Plan contributions, are not taxable to employees until withdrawn from the 401(k) Plan, and so that contributions by us, if any, will be deductible by us when made. At the direction of each participant, we invest the assets of the 401(k) Plan in any of over 50 investment options. Company contributions under the 401(k) Plan were $0.4 million, $0.3 million and $0 in 2019, 2018 and 2017, respectively.

Commitments and Contingencies

Commitments and Contingencies	12 Months Ended
	Dec. 31, 2019
Commitments and Contingencies Disclosure [Abstract]
Commitments and Contingencies	NOTE 6 – COMMITMENTS AND CONTINGENCIES Operating Leases We lease space under operating leases for manufacturing and laboratory facilities and administrative offices in Cleveland, as well as administrative offices in New York. We also lease office space in Madrid, Spain as well as certain office equipment under operating leases, which have a non-cancelable lease term of less than one year and therefore, we have elected the practical expedient to exclude these short-term leases from our right-of-use assets and lease liabilities. We can terminate the operating leases for our manufacturing and laboratory facilities and administrative offices in Cleveland early at December 31, 2020 and pay for unamortized tenant improvements. Our total lease costs and unamortized tenant improvements would total $1.7 million by exercising the early termination provision. Components of lease cost under ASC 842 are as follows for the year ended December 31, 2019: Year ended December 31, 2019 Operating lease cost $ 1,591,000 Variable lease cost $ 322,000 Short-term lease cost $ 133,000 The following table presents information about the amount and timing of cash flows arising from operating leases under ASC 842 as of December 31, 2019: Maturity of lease liabilities: 2020 $ 1,699,000 2021 1,713,000 2022 1,727,000 2023 1,741,000 2024 1,781,000 Thereafter 1,885,000 Total undiscounted operating lease payments 10,546,000 Less: imputed interest 2,596,000 Present value of operating lease liabilities $ 7,950,000 Balance sheet classification: Current portion of lease liability $ 1,699,000 Long-term lease liability 6,251,000 Total operating lease liabilities $ 7,950,000 Other information: Weighted-average remaining lease term for operating leases 73 months Weighted-average discount rate for operating leases 9.6 % Rent expense for the years ended December 31, 2018 and 2017 under ASC 840 was $1.3 million and $0.7 million, respectively. Manufacturing We have engaged a contract manufacturer to assist us with developing and defining the processes necessary to manufacture our RDEB product candidate. We had a remaining commitment of $6.4 million at December 31, 2019. The amount is payable based on the completion of specific activities outlined in the contracted project plan; we expect to spend the entire $6.4 million in 2020. Legal We are not currently subject to any material pending legal proceedings as of December 31, 2019.

Fair Value Measurements

Fair Value Measurements	12 Months Ended
	Dec. 31, 2019
Fair Value Disclosures [Abstract]
Fair Value Measurements	NOTE 7 - FAIR VALUE MEASUREMENTS We calculate the fair value of our assets and liabilities that qualify as financial instruments and include additional information in the notes to the consolidated financial statements when the fair value is different than the carrying value of these financial instruments. The estimated fair value of receivables, prepaid expenses, other assets, accounts payable, accrued expenses, payable to licensor and deferred revenue approximate their carrying amounts due to the relatively short maturity of these instruments. U.S. GAAP defines fair value as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants at the measurement date. This guidance establishes a three-level fair value hierarchy that prioritizes the inputs used to measure fair value. The hierarchy requires entities to maximize the use of observable inputs and minimize the use of unobservable inputs. The three levels of inputs used to measure fair value are as follows: ● Level 1 – Quoted prices in active markets for identical assets or liabilities. ● Level 2 – Observable inputs other than quoted prices included in Level 1, such as quoted prices for similar assets and liabilities in active markets; quoted prices for identical or similar assets and liabilities in markets that are not active; or other inputs that are observable or can be corroborated by observable market data. ● Level 3 – Unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets and liabilities. This includes certain pricing models, discounted cash flow methodologies and similar valuation techniques that use significant unobservable inputs. The guidance requires an entity to maximize the use of observable inputs and minimize the use of unobservable inputs when measuring fair value. We have segregated all financial assets and liabilities that are measured at fair value on a recurring basis (at least annually) into the most appropriate level within the fair value hierarchy based on the inputs used to determine the fair value at the measurement date in the table below. Financial assets and liabilities measured at fair value on a recurring and non-recurring basis as of December 31, 2019 and 2018 are summarized below: Description December 31, 2019 Level 1 Level 2 Level 3 Total Gains/(Losses) Non-recurring Assets: Licensed technology, net $ 36,178,000 $ - $ - $ 36,178,000 $ (367,000 ) Goodwill 32,466,000 - - 32,466,000 - Description December 31, 2018 Level 1 Level 2 Level 3 Total Gains/(Losses) Recurring Assets: Short-term investments $ 66,218,000 $ - $ 66,218,000 $ - $ - Non-recurring Assets: Licensed technology, net $ 43,042,000 $ - $ - $ 43,042,000 $ - Goodwill 32,466,000 - - 32,466,000 -

Stockholders' Equity

Stockholders' Equity	12 Months Ended
	Dec. 31, 2019
Equity [Abstract]
Stockholders' Equity	NOTE 8 – STOCKHOLDERS’ EQUITY 2019 Public Offering of Common Stock and “Pre-Funded” Warrants On December 24, 2019, we closed an underwritten public offering of 32,382,945 shares of common stock at a public offering price of $2.50 per share. In addition, as part of the offering, we sold to an existing investor “pre-funded” warrants to purchase up to an aggregate of 9,017,055 shares of common stock at a purchase price of $2.4999 per pre-funded warrant, which equals the public offering price per share of the common stock less the $0.0001 per share exercise price of each pre-funded warrant. The gross proceeds to the Company were approximately $103.5 million, before deducting the underwriting discounts and commissions and estimated offering expenses payable by the Company. There were 9,017,055 “pre-funded” warrants to purchase a total of 9,017,055 shares of common stock outstanding at December 31, 2019. The “pre-funded” warrants are currently exercisable and have no expiration date. 2017 Public Offering of Common Stock On October 19, 2017, we closed an underwritten public offering of 5,750,000 shares of common stock, at a public offering price of $16.00 per share. The gross proceeds to the Company were approximately $92.0 million before deducting the underwriting discounts and commissions and estimated offering expenses payable by the Company. “Non-Pre-Funded” Warrants “Non-pre-funded” warrants to purchase a total of 70,000 and 1,820,686 shares of common stock were outstanding at December 31, 2019 and 2018, respectively. “Non-pre-funded” warrants to purchase 1,750,686 shares of common stock expired during 2019. We received cash from the exercise of “non-pre-funded” warrants of $0, $3.2 million and $5.9 million during 2019, 2018 and 2017, respectively. The warrants had various exercise prices and terms as follows as of December 31, 2019: Summary of Warrants Warrants Exercise Expiration 2015 Financing (a) 20,000 $ 6.05 July 31, 2020 2015 Financing (b) 50,000 $ 11.00 May 11, 2020 Total 70,000 a) In connection with the offering on July 31, 2015, the placement agent received warrants to purchase 20,000 shares of common stock at $6.05 per share. The warrants are currently exercisable and expire on July 31, 2020. b) In connection with the offering on May 11, 2015, the placement agent received warrants to purchase 50,000 shares of common stock at $11.00 per share. The warrants are currently exercisable and expire on May 11, 2020.

Stock Option Plans

Stock Option Plans	12 Months Ended
	Dec. 31, 2019
Share-based Payment Arrangement [Abstract]
Stock Option Plans	NOTE 9 - STOCK OPTION PLANS Our stock-based employee compensation plans are described below: 2015 Equity Incentive Plan Under our 2015 Equity Incentive Plan, as amended, up to 10,000,000 shares of our authorized but unissued common stock are reserved for issuance to employees, consultants, or to non-employee members of the Board or to any member of the board of directors (or similar governing authority) of any affiliate of the Company. The 2015 Equity Incentive Plan, approved by our stockholders on May 7, 2015, replaced the previously approved stock option plan (the “2005 Equity Incentive Plan”). The maximum contractual term of awards is generally 10 years. Stock Options: ● Expected volatility – we estimate the volatility of our share price at the date of grant using a “look-back” period which coincides with the expected term, defined below. We believe using a “look-back” period which coincides with the expected term is the most appropriate measure for determining expected volatility. ● Expected term – we estimate the expected term using the “simplified” method, as outlined in Staff Accounting Bulletin No. 107, “Share-Based Payment.” ● Risk-free interest rate – we estimate the risk-free interest rate using the U.S. Treasury yield curve for periods equal to the expected term of the options in effect at the time of grant. ● Dividends – we use an expected dividend yield of zero because we have not declared or paid a cash dividend, nor do we have any plans to declare a dividend. We used the following weighted-average assumptions to estimate the fair value of the options granted for the years indicated: For the years ended December 31, 2019 2018 2017 Expected volatility 108 % 109 % 111 % Expected term 5.1 years 5 years 5 years Risk-free interest rate 2.21 % 2.62 % 1.81 % Expected dividend yield 0.00 % 0.00 % 0.00 % Summarized information for the 2015 Equity Incentive Plan is as follows: Options Weighted- Outstanding options at January 1, 2017 4,443,000 $ 4.94 Granted, fair value of $11.35 per share 1,077,000 14.31 Exercised (81,719 ) 4.22 Expired/forfeited (325,314 ) 5.52 Outstanding options at December 31, 2017 5,112,967 $ 9.07 Granted, fair value of $10.23 per share 1,497,300 $ 12.95 Exercised (360,853 ) 6.22 Expired/forfeited (724,009 ) 10.65 Outstanding options at December 31, 2018 5,525,405 $ 8.08 Granted, fair value of $5.14 per share 1,490,490 $ 6.53 Exercised (303,129 ) 3.20 Expired/forfeited (917,371 ) 7.95 Outstanding options at December 31, 2019 5,795,395 $ 7.96 Non-vested options at December 31, 2017 2,641,832 $ 8.21 Non-vested options at December 31, 2018 2,355,719 $ 10.41 Non-vested options at December 31, 2019 2,342,706 $ 8.78 The intrinsic value related to the outstanding options under this plan was $0.7 million, $8.4 million and $46.0 million at December 31, 2019, 2018 and 2017, respectively. The intrinsic value related to the exercisable options under this plan was $0.6 million, $6.0 million and $25.6 million at December 31, 2019, 2018 and 2017, respectively. The total intrinsic value of the options exercised was $0.4 million, $3.2 million and $0.6 million during 2019, 2018 and 2017, respectively. Further information regarding options outstanding under the 2015 Equity Incentive Plan at December 31, 2019 is summarized below: Weighted-average Weighted-average Range of exercise prices Number of options outstanding Remaining life in years Exercise price Number of options exercisable Remaining life in years Exercise price $ 2.26 $ 5.44 1,989,150 7.0 $ 3.66 1,462,346 6.5 $ 3.46 5.90 9.23 2,425,240 7.6 7.35 1,253,189 6.2 7.32 11.43 14.45 456,619 8.5 13.49 204,025 8.4 13.56 15.25 17.30 924,386 8.0 16.09 533,129 8.0 16.07 5,795,395 3,452,689 At December 31, 2019, the total compensation cost related to non-vested options not recognized is $14.4 million. The expected weighted average period over which the total compensation costs related to non-vested options is 2.7 years. Restricted Common Stock During the year ended December 31, 2019, 22,000 shares of restricted common stock were forfeited, and no shares became vested. At December 31, 2019, the total compensation cost related to restricted common stock not recognized is $0.5 million. The expected weighted average period over which the total compensation costs related to restricted common stock is 0.2 years. 2005 Equity Incentive Plan Under the 2005 Equity Incentive Plan, as amended, shares of our authorized but unissued common stock were reserved for issuance to employees, consultants, or to non-employee members of the Board or to any member of the board of directors (or similar governing authority) of any affiliate of the Company. As of January 20, 2015, no additional shares were available for grant under the 2005 Equity Incentive Plan. A total of 260,000 options were outstanding and exercisable under this plan at December 31, 2019. Summarized information for the 2005 Equity Incentive Plan is as follows: Options Weighted- Outstanding options at January 1, 2017 328,560 $ 14.57 Expired/forfeited (11,800 ) 21.53 Outstanding options at December 31, 2017 316,760 $ 14.31 Expired/forfeited (360 ) 150.00 Outstanding options at December 31, 2018 316,400 $ 14.15 Expired/forfeited (56,400 ) 19.75 Outstanding options at December 31, 2019 260,000 $ 12.94 The intrinsic value related to the outstanding or exercisable options under this plan was $0, $0.5 million and $1.5 million at December 31, 2019, 2018 and 2017, respectively. Further information regarding options outstanding under the 2005 Equity Incentive Plan at December 31, 2019 is summarized below: Weighted-average Weighted-average Range of exercise prices Number of options outstanding Remaining life in years Exercise price Number of options exercisable Remaining life in years Exercise price $ 3.25 $ 3.25 120,000 0.1 $ 3.25 120,000 0.1 $ 3.25 11.50 30.50 136,100 4.1 18.48 136,100 4.1 18.48 113.50 122.50 3,900 0.9 117.65 3,900 0.9 117.65 260,000 260,000

Income Taxes

Income Taxes	12 Months Ended
	Dec. 31, 2019
Income Tax Disclosure [Abstract]
Income Taxes	NOTE 10 - INCOME TAXES Income tax expense differs from the statutory amounts for each of the following years: 2019 2018 2017 Income taxes at U.S. statutory rate $ (16,020,000 ) $ (11,901,000 ) $ (9,289,000 ) Current year reserve 15,976,000 11,877,000 (25,175,000 ) Expenses not deductible 44,000 24,000 46,000 Rate change - - 34,418,000 Total tax expense $ - $ - $ - Deferred taxes are provided for the temporary differences between the financial reporting bases and the tax bases of our assets and liabilities. The temporary differences that give rise to deferred tax assets and liabilities were as follows: December 31, 2019 December 31, 2018 Deferred tax assets (liabilities): Net operating loss carryforwards $ 50,802,000 $ 38,761,000 General business credit carryforwards 3,939,000 3,214,000 State credits 2,934,000 3,012,000 Property, equipment and goodwill (28,000 ) (72,000 ) Stock options 7,960,000 6,316,000 Derivatives - (57,000 ) Deferred revenue 62,000 62,000 Intangible assets 1,625,000 399,000 Other 28,000 80,000 Gross deferred tax assets 67,322,000 51,715,000 Valuation allowance (67,322,000 ) (51,715,000 ) Net deferred taxes $ - $ - At December 31, 2019, we had approximately $241.9 million of net operating loss carryforwards and approximately $3.9 million of general business credit carryforwards. These carryforwards expire as follows: Net operating General business credit 2020 $ 5,125,000 $ 226,000 2021 5,378,000 56,000 2022 8,230,000 431,000 2023 5,434,000 362,000 2024 8,711,000 287,000 Thereafter 99,946,000 2,536,000 $ 132,824,000 $ 3,898,000 At December 31, 2019, we had approximately $109.1 million of net operating loss carryforwards that do not expire and can be carried forward indefinitely. We acquired MacroChem Corporation on February 25, 2009 and Somanta Pharmaceuticals, Inc. on January 4, 2008. Both of these corporations were loss-making entities at the time of acquisition. As a result, the net operating losses related to those acquisitions may be subject to annual limitations as well. On December 22, 2017, President Donald Trump signed the Tax Cuts and Jobs Act (the “TCJA”). The TCJA reduces the U.S. federal corporate tax rate from 34% to 21%. At December 31, 2017, we recalculated our deferred tax assets and liabilities based on the rates at which they are expected to reverse in the future, which is generally 21%, and recorded a provisional adjustment of $34.4 million. In 2018, the accounting for the income tax enactment-date effects of the TCJA were completed and there was no adjustment to the provisional amount recorded in 2017.

Quarterly Financial Data (Unaud

Quarterly Financial Data (Unaudited)	12 Months Ended
	Dec. 31, 2019
Quarterly Financial Information Disclosure [Abstract]
Quarterly Financial Data (Unaudited)	NOTE 11 – QUARTERLY FINANCIAL DATA (UNAUDITED) The following table contains quarterly financial information for 2019 and 2018. We believe that the following information reflects all normal recurring adjustments necessary for a fair statement of the information for the periods presented. The operating results for any quarter are not necessarily indicative of results for any future period. Quarters Ended December 31, September 30, June 30, March 31, Foundation revenues $ - $ - $ - $ - Royalties - - - - Research and development 9,605,000 10,917,000 16,307,000 11,737,000 General and administrative 4,734,000 4,700,000 5,612,000 5,659,000 Loss from operations (16,411,000 ) (17,649,000 ) (23,976,000 ) (19,054,000 ) Net loss (16,431,000 ) (17,372,000 ) (23,924,000 ) (18,555,000 ) Basic and diluted loss per common share (0.30 ) (0.35 ) (0.49 ) (0.39 ) Weighted average number of common shares outstanding – basic and diluted 54,718,776 49,721,753 48,961,988 47,948,421 Quarters Ended December 31, September 30, June 30, March 31, Foundation revenues $ 369,000 $ 1,687,000 $ 259,000 $ 481,000 Royalties 113,000 22,000 17,000 50,000 Research and development 9,470,000 13,150,000 7,916,000 8,162,000 General and administrative 7,631,000 4,970,000 4,627,000 2,878,000 Loss from operations (18,010,000 ) (16,916,000 ) (12,557,000 ) (10,683,000 ) Net loss (17,479,000 ) (16,419,000 ) (12,243,000 ) (10,530,000 ) Basic and diluted loss per common share (0.36 ) (0.34 ) (0.26 ) (0.22 ) Weighted average number of common shares outstanding – basic and diluted 47,944,486 47,794,394 47,303,518 47,060,523

Nature of Operations and Summ_2

Nature of Operations and Summary of Significant Accounting Policies (Policies)	12 Months Ended
	Dec. 31, 2019
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Nature of Operations	Nature of Operations Abeona Therapeutics Inc. (together with our subsidiaries, “we,” “our,” “Abeona” or the “Company”), a Delaware corporation, is a clinical-stage biopharmaceutical company developing gene and cell therapies for life-threatening rare genetic diseases. Our lead clinical programs consist of: (i) EB-101, an autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa (“RDEB”), (ii) ABO-102, an adeno-associated virus (“AAV”)-based gene therapy for Sanfilippo syndrome type A (“MPS IIIA”) and (iii) ABO-101, an AAV-based gene therapy for Sanfilippo syndrome type B (“MPS IIIB”). We have additional AAV-based gene therapies in various developmental stages designed to treat the CLN1 and CLN3 forms of Batten Disease, cystic fibrosis and retinal diseases. In addition, we are developing next-generation AAV-based gene therapies through our novel AIM™ capsid platform and internal AAV vector research programs.
Principles of Consolidation	Principles of Consolidation The consolidated financial statements include the financial statements of Abeona Therapeutics Inc. and our wholly-owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation.
Uses and Sources of Liquidity	Uses and Sources of Liquidity The financial statements have been prepared on the going concern basis, which assumes the Company will have sufficient cash to pay its operating expenses, as and when they become payable, for a period of at least 12 months from the date the financial report was issued. As of December 31, 2019, we had cash and cash equivalents of $129.3 million and net assets of $178.4 million. For the year ended December 31, 2019, we had cash outflows from operations of $62.8 million. We have not generated any significant product revenues and have not achieved profitable operations. There is no assurance that profitable operations will ever be achieved, and, if achieved, could be sustained on a continuing basis. In addition, development activities, clinical and nonclinical testing, and commercialization of our products will require significant additional financing. We are subject to a number of risks similar to other life science companies, including, but not limited to, risks related to the successful discovery and development of product candidates, obtaining the necessary regulatory approval to market our product candidates, raising additional capital, development of competing drugs and therapies, protection of proprietary technology and market acceptance of our products. As a result of these and other risks and the related uncertainties, there can be no assurance of our future success. Based upon our current operating plans, we believe that we have sufficient resources to fund operations through the second quarter of 2021 with our existing cash and cash equivalents. We will need to secure additional funding in the future, to carry out all of our planned research and development activities. If we are unable to obtain additional financing or generate license or product revenue, the lack of liquidity and sufficient capital resources could have a material adverse effect on our future prospects.
Use of Estimates	Use of Estimates The preparation of consolidated financial statements in conformity with accounting principles generally accepted in the United States of America (“U.S. GAAP”) requires management to make estimates and assumptions that affect the reported amount of assets and disclosure of contingent assets and liabilities at the date of the consolidated financial statements and the reported amounts of revenue and expenses during the reported period. Actual results could differ from these estimates and assumptions.
Cash and Cash Equivalents	Cash and Cash Equivalents We consider all highly liquid investments with a maturity of three months or less when purchased to be cash equivalents. We maintain deposits primarily in financial institutions, which may at times exceed amounts covered by insurance provided by the U.S. Federal Deposit Insurance Corporation (“FDIC”). We have not experienced any losses related to amounts in excess of FDIC limits.
Short-term Investments	Short-term Investments Short-term investments consist of investments in U.S. government, U.S. agency and U.S. treasury securities. We determine the appropriate classification of the securities at the time they are acquired and evaluate the appropriateness of such classifications at each balance sheet date. We classify our short-term investments as available-for-sale pursuant to Accounting Standards Codification (“ASC”) 320, Investments – Debt and Equity Securities We review our short-term investments for other-than-temporary impairment whenever the fair value of a marketable security is less than the amortized cost and evidence indicates that a short-term investment’s carrying amount is not recoverable within a reasonable period of time.
Receivables	Receivables Receivables are reported in the consolidated balance sheets at net realizable value. We continually evaluate the creditworthiness of our customers and their financial condition and generally do not require collateral. The allowance for doubtful accounts is based upon reviews of specific customer balances, historic losses, and general economic conditions. As of December 31, 2019 and 2018, no allowance was recorded as all accounts are considered collectible. There were no receivables outstanding as of December 31, 2019.
Property and Equipment	Property and Equipment Property and equipment are recorded at cost. Depreciation is provided using the straight-line method over estimated useful lives ranging from three to seven years for equipment and five to ten years for leasehold improvements. Expenditures for major renewals and betterments that extend the useful lives are capitalized. Expenditures for normal maintenance and repairs are expensed as incurred. The cost of assets sold or abandoned, and the related accumulated depreciation are eliminated from the accounts and any gains or losses are recognized in the accompanying consolidated statements of operations of the respective period.
Leases	Leases In February 2016, the Financial Accounting Standards Board issued Accounting Standards Update (“ASU”) 2016-02, Leases Leases Right-of-use lease assets represent our right to use an underlying asset for the lease term and lease liabilities represent our obligation to make lease payments arising from the lease. The measurement of lease liabilities is based on the present value of future lease payments over the lease term. As our leases do not provide an implicit rate, we use our incremental borrowing rate based on the information available at the lease commencement date in determining the present value of future lease payments. The right-of-use asset is based on the measurement of the lease liability and includes any lease payments made prior to or on lease commencement and excludes lease incentives and initial direct costs incurred, as applicable. Rent expense for our operating leases is recognized on a straight-line basis over the lease term. We do not have any leases classified as finance leases. Our leases do not have significant rent escalation, holidays, concessions, material residual value guarantees, material restrictive covenants or contingent rent provisions. Our leases include both lease (e.g., fixed payments including rent, taxes, and insurance costs) and non-lease components (e.g., common-area or other maintenance costs), which are accounted for as a single lease component as we have elected the practical expedient to group lease and non-lease components for all leases. Most leases include one or more options to renew. The exercise of lease renewal options is typically at our sole discretion; therefore, the majority of renewals to extend the lease terms are not included in our right-of-use assets and lease liabilities as they are not reasonably certain of exercise. We regularly evaluate the renewal options and when they are reasonably certain of exercise, we include the renewal period in our lease term. Additional information and disclosures required under ASC 842 are included in Note 6.
Licensed Technology	Licensed Technology We have entered into agreements to license the rights to certain technologies. We recorded the purchase price paid for the license, which represents fair value, on our consolidated balance sheet. We maintain licensed technology on our consolidated balance sheet until either the licensed technology agreement underlying it is completed or the asset becomes impaired. When we determine that an asset has become impaired or we abandon a project, we write down the carrying value of the related intangible asset to its fair value and take an impairment charge in the period in which the impairment occurs. Licensed technology is amortized over the life of the patent or the agreement and periodically reviewed for impairment. We test our intangible assets for impairment on an annual basis, or more frequently if indicators are present or changes in circumstance suggest that impairment may exist. Events that could result in an impairment, or trigger an interim impairment assessment, include the receipt of additional clinical or nonclinical data regarding our drug candidate or a potentially competitive drug candidate, changes in the clinical development program for a drug candidate or new information regarding potential sales for the drug. In connection with each annual impairment assessment and any interim impairment assessment, we compare the fair value of the asset as of the date of the assessment with the carrying value of the asset on our consolidated balance sheet. In 2019, 2018 and 2017, we did not impair any licensed technology.
Goodwill	Goodwill As of December 31, 2019 and 2018, goodwill of $32.5 million was recorded on the Company’s consolidated balance sheet. In accordance with ASC 350, Intangibles — Goodwill and Other,
Restricted Cash	Restricted Cash In November 2016, the Financial Accounting Standards Board issued ASU 2016-18, Statement of Cash Flows (Topic 230): Restricted Cash
Segments	Segments The Company operates in a single segment. The Company’s chief operating decision maker, its Executive Chairman, manages the Company’s operations on a consolidated basis for the purpose of allocating resources.
Revenue Recognition	Revenue Recognition Effective January 1, 2018, we adopted Accounting Standards Update (“ASU”) 2014-09, Revenue from Contracts with Customers Revenue Recognition The table below presents the impact of the adoption of ASC 606 on our statement of operations for the year ended December 31, 2018. For the year ended December 31, 2018 Under Effect of As Reported ASC 605 ASC 606 Under ASC 606 Revenues Foundation revenues $ - $ 2,796,000 $ 2,796,000 License revenues 602,000 (602,000 ) - Total revenues 804,000 2,194,000 2,998,000 Loss from operations (60,360,000 ) 2,194,000 (58,166,000 ) Net loss (58,865,000 ) 2,194,000 (56,671,000 ) Basic and diluted loss per common share (1.24 ) 0.05 (1.19 ) Foundation revenues: As a result of the adoption of ASC 606, we decreased deferred revenue and the accumulated deficit by $2.6 million as of January 1, 2018. As of both December 31, 2018 and 2019, we received $5.7 million of the grants. In accordance with ASC 606, we record revenue to match expenses for the advancement of the Company’s clinical stage gene therapies for MPS IIIA and MPS IIIB, the production of which is the primary performance obligation. We recorded foundation revenues of $0 in 2019 and $2.8 million in 2018. We allocated the transaction price based on the expected costs plus a margin approach. The aggregate amount of the transaction price allocated to the performance obligations that are unsatisfied as of December 31, 2019 and 2018 was $0.3 million. We expect the unsatisfied performance obligations to be recognized in the next 12 months. License revenues: Revenue from Contracts with Customers (Topic 606): Identifying Performance Obligations and Licensing Royalty revenues: .
Research and Development Expenses	Research and Development Expenses Research and development costs are expensed as incurred. Research and development expenses include, but are not limited to, payroll and personnel expense, lab supplies, preclinical, development cost, clinical trial expense, outside manufacturing and consulting. The cost of materials and equipment or facilities that are acquired for research and development activities and that have alternative future uses are capitalized when acquired.
General and Administrative Expenses	General and Administrative Expenses General and administrative expenses primarily consist of personnel, contract personnel, personnel-related expenses to support our administrative and operating activities, facility costs and professional expenses (i.e., legal expenses) and investor relations fees.
Income Taxes	Income Taxes Income taxes are accounted for under the asset and liability method. Deferred tax assets and liabilities are recognized for the future tax consequences attributable to differences between the consolidated financial statement carrying amounts of existing assets and liabilities and their respective tax bases and operating loss and tax credit carryforwards. Deferred tax assets and liabilities are measured using enacted tax rates expected to apply to taxable income in the years in which those temporary differences are expected to be recovered or settled. The effect on deferred tax assets and liabilities of a change in tax rates is recognized in income in the period that includes the enactment date. A valuation allowance is provided for deferred tax assets to the extent their realization is in doubt. We account for uncertain income tax positions in accordance with ASC 740, Income Taxes
Loss Per Common Share	Loss Per Common Share We have presented basic and diluted loss per common share on the statement of operations. Basic and diluted net loss per share is computed by dividing net loss by the weighted-average number of shares of common stock and shares underlying “pre-funded” warrants outstanding during the period. The “pre-funded” warrants are included in the computation of basic net loss per share as the exercise price is negligible and they are fully vested and exercisable. We do not include the potential impact of dilutive securities in diluted net loss per share, as the impact of these items is anti-dilutive. Potential dilutive securities result from outstanding stock options and “non-pre-funded” warrants. We did not include the following potentially dilutive securities in the computation of diluted net loss per common share during the periods presented: For the years ended December 31, 2019 2018 2017 Warrants 70,000 1,820,686 2,934,685 Stock options 6,055,395 5,841,805 5,429,727 Total 6,125,395 7,662,491 8,364,412
Stock-Based Compensation	Stock-Based Compensation We account for stock-based compensation expense in accordance with ASC 718, Stock Based Compensation The following table summarizes stock option-based option compensation for 2019, 2018 and 2017, which was allocated as follows: For the years ended December 31, 2019 2018 2017 Research and development $ 3,932,000 $ 3,913,000 $ 1,668,000 General and administrative 3,406,000 4,265,000 2,976,000 Stock option-based compensation expense included in operating expense 7,338,000 8,178,000 4,644,000 Total stock option-based compensation expense 7,338,000 8,178,000 4,644,000 Tax benefit - - - Stock option-based compensation expense, net of tax $ 7,338,000 $ 8,178,000 $ 4,644,000 The following table summarizes restricted stock-based compensation for 2019, 2018 and 2017, which was allocated as follows: For the years ended December 31, 2019 2018 2017 Research and development $ 454,000 $ - $ - General and administrative 445,000 688,000 1,272,000 Restricted stock-based compensation expense included in operating expense 899,000 688,000 1,272,000 Total restricted stock-based compensation expense 899,000 688,000 1,272,000 Tax benefit - - - Restricted stock-based compensation expense, net of tax $ 899,000 $ 688,000 $ 1,272,000

Nature of Operations and Summ_3

Nature of Operations and Summary of Significant Accounting Policies (Tables)	12 Months Ended
	Dec. 31, 2019
Schedule of Statement of Operations	The table below presents the impact of the adoption of ASC 606 on our statement of operations for the year ended December 31, 2018. For the year ended December 31, 2018 Under Effect of As Reported ASC 605 ASC 606 Under ASC 606 Revenues Foundation revenues $ - $ 2,796,000 $ 2,796,000 License revenues 602,000 (602,000 ) - Total revenues 804,000 2,194,000 2,998,000 Loss from operations (60,360,000 ) 2,194,000 (58,166,000 ) Net loss (58,865,000 ) 2,194,000 (56,671,000 ) Basic and diluted loss per common share (1.24 ) 0.05 (1.19 )
Schedule of Antidilutive Securities Excluded from Computation of Earnings Per Share	We did not include the following potentially dilutive securities in the computation of diluted net loss per common share during the periods presented: For the years ended December 31, 2019 2018 2017 Warrants 70,000 1,820,686 2,934,685 Stock options 6,055,395 5,841,805 5,429,727 Total 6,125,395 7,662,491 8,364,412
Schedule of Stock Based Compensation	The following table summarizes stock option-based option compensation for 2019, 2018 and 2017, which was allocated as follows: For the years ended December 31, 2019 2018 2017 Research and development $ 3,932,000 $ 3,913,000 $ 1,668,000 General and administrative 3,406,000 4,265,000 2,976,000 Stock option-based compensation expense included in operating expense 7,338,000 8,178,000 4,644,000 Total stock option-based compensation expense 7,338,000 8,178,000 4,644,000 Tax benefit - - - Stock option-based compensation expense, net of tax $ 7,338,000 $ 8,178,000 $ 4,644,000
Restricted Stock [Member]
Schedule of Stock Based Compensation	The following table summarizes restricted stock-based compensation for 2019, 2018 and 2017, which was allocated as follows: For the years ended December 31, 2019 2018 2017 Research and development $ 454,000 $ - $ - General and administrative 445,000 688,000 1,272,000 Restricted stock-based compensation expense included in operating expense 899,000 688,000 1,272,000 Total restricted stock-based compensation expense 899,000 688,000 1,272,000 Tax benefit - - - Restricted stock-based compensation expense, net of tax $ 899,000 $ 688,000 $ 1,272,000

Short-Term Investments (Tables)

Short-Term Investments (Tables)	12 Months Ended
	Dec. 31, 2019
Investments, Debt and Equity Securities [Abstract]
Schedule of Available-for-Sale Investments Held	The following table summarizes the available-for-sale investments held as of December 31, 2018. There were no available-for-sale investments as of December 31, 2019. Description Fair value U.S. government and agency securities and treasuries $ 66,218,000

Property and Equipment (Tables)

Property and Equipment (Tables)	12 Months Ended
	Dec. 31, 2019
Property, Plant and Equipment [Abstract]
Schedule of Property and Equipment	Property and equipment consist of the following: December 31, 2019 December 31, 2018 Laboratory equipment $ 7,031,000 $ 4,366,000 Furniture and office equipment 1,710,000 1,454,000 Leasehold improvements 8,573,000 4,774,000 Construction work-in-progress - 409,000 17,314,000 11,003,000 Less: accumulated depreciation and amortization 4,157,000 1,560,000 Property and equipment, net $ 13,157,000 $ 9,443,000

Licensed Technology (Tables)

Licensed Technology (Tables)	12 Months Ended
	Dec. 31, 2019
Goodwill and Intangible Assets Disclosure [Abstract]
Schedule of Licensed Technology	Licensed technology consists of the following: December 31, 2019 December 31, 2018 Licensed technology $ 42,606,000 $ 44,859,000 Less accumulated amortization 6,428,000 1,817,000 Licensed technology, net $ 36,178,000 $ 43,042,000
Schedule of Amortization Expense for Intangible Assets	The aggregate estimated amortization expense for intangible assets remaining as of December 31, 2019 is as follows: 2020 $ 5,174,000 2021 5,174,000 2022 5,174,000 2023 5,174,000 2024 5,174,000 Thereafter 10,308,000 Total $ 36,178,000

Commitments and Contingencies (

Commitments and Contingencies (Tables)	12 Months Ended
	Dec. 31, 2019
Commitments and Contingencies Disclosure [Abstract]
Schedule of Components of Lease Cost	Components of lease cost under ASC 842 are as follows for the year ended December 31, 2019: Year ended December 31, 2019 Operating lease cost $ 1,591,000 Variable lease cost $ 322,000 Short-term lease cost $ 133,000
Schedule of Supplemental Cash Flow Information Related to Leases	The following table presents information about the amount and timing of cash flows arising from operating leases under ASC 842 as of December 31, 2019: Maturity of lease liabilities: 2020 $ 1,699,000 2021 1,713,000 2022 1,727,000 2023 1,741,000 2024 1,781,000 Thereafter 1,885,000 Total undiscounted operating lease payments 10,546,000 Less: imputed interest 2,596,000 Present value of operating lease liabilities $ 7,950,000 Balance sheet classification: Current portion of lease liability $ 1,699,000 Long-term lease liability 6,251,000 Total operating lease liabilities $ 7,950,000 Other information: Weighted-average remaining lease term for operating leases 73 months Weighted-average discount rate for operating leases 9.6 %

Fair Value Measurements (Tables

Fair Value Measurements (Tables)	12 Months Ended
	Dec. 31, 2019
Fair Value Disclosures [Abstract]
Schedule of Fair Value, Assets and Liabilities Measured on Recurring and Non-Recurring Basis	Financial assets and liabilities measured at fair value on a recurring and non-recurring basis as of December 31, 2019 and 2018 are summarized below: Description December 31, 2019 Level 1 Level 2 Level 3 Total Gains/(Losses) Non-recurring Assets: Licensed technology, net $ 36,178,000 $ - $ - $ 36,178,000 $ (367,000 ) Goodwill 32,466,000 - - 32,466,000 - Description December 31, 2018 Level 1 Level 2 Level 3 Total Gains/(Losses) Recurring Assets: Short-term investments $ 66,218,000 $ - $ 66,218,000 $ - $ - Non-recurring Assets: Licensed technology, net $ 43,042,000 $ - $ - $ 43,042,000 $ - Goodwill 32,466,000 - - 32,466,000 -

Stockholders' Equity (Tables)

Stockholders' Equity (Tables)	12 Months Ended
	Dec. 31, 2019
Equity [Abstract]
Schedule of Warrants Outstanding at Various Exercise Prices	The warrants had various exercise prices and terms as follows as of December 31, 2019: Summary of Warrants Warrants Exercise Expiration 2015 Financing (a) 20,000 $ 6.05 July 31, 2020 2015 Financing (b) 50,000 $ 11.00 May 11, 2020 Total 70,000 a) In connection with the offering on July 31, 2015, the placement agent received warrants to purchase 20,000 shares of common stock at $6.05 per share. The warrants are currently exercisable and expire on July 31, 2020. b) In connection with the offering on May 11, 2015, the placement agent received warrants to purchase 50,000 shares of common stock at $11.00 per share. The warrants are currently exercisable and expire on May 11, 2020.

Stock Option Plans (Tables)

Stock Option Plans (Tables)	12 Months Ended
	Dec. 31, 2019
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Schedule of Weighted-average Assumptions to Estimate the Fair Value of the Options Granted	We used the following weighted-average assumptions to estimate the fair value of the options granted for the years indicated: For the years ended December 31, 2019 2018 2017 Expected volatility 108 % 109 % 111 % Expected term 5.1 years 5 years 5 years Risk-free interest rate 2.21 % 2.62 % 1.81 % Expected dividend yield 0.00 % 0.00 % 0.00 %
2015 Equity Incentive Plan [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Schedule of Options Activity	Summarized information for the 2015 Equity Incentive Plan is as follows: Options Weighted- Outstanding options at January 1, 2017 4,443,000 $ 4.94 Granted, fair value of $11.35 per share 1,077,000 14.31 Exercised (81,719 ) 4.22 Expired/forfeited (325,314 ) 5.52 Outstanding options at December 31, 2017 5,112,967 $ 9.07 Granted, fair value of $10.23 per share 1,497,300 $ 12.95 Exercised (360,853 ) 6.22 Expired/forfeited (724,009 ) 10.65 Outstanding options at December 31, 2018 5,525,405 $ 8.08 Granted, fair value of $5.14 per share 1,490,490 $ 6.53 Exercised (303,129 ) 3.20 Expired/forfeited (917,371 ) 7.95 Outstanding options at December 31, 2019 5,795,395 $ 7.96 Non-vested options at December 31, 2017 2,641,832 $ 8.21 Non-vested options at December 31, 2018 2,355,719 $ 10.41 Non-vested options at December 31, 2019 2,342,706 $ 8.78
Schedule of Options Outstanding and Exercisable	Further information regarding options outstanding under the 2015 Equity Incentive Plan at December 31, 2019 is summarized below: Weighted-average Weighted-average Range of exercise prices Number of options outstanding Remaining life in years Exercise price Number of options exercisable Remaining life in years Exercise price $ 2.26 $ 5.44 1,989,150 7.0 $ 3.66 1,462,346 6.5 $ 3.46 5.90 9.23 2,425,240 7.6 7.35 1,253,189 6.2 7.32 11.43 14.45 456,619 8.5 13.49 204,025 8.4 13.56 15.25 17.30 924,386 8.0 16.09 533,129 8.0 16.07 5,795,395 3,452,689
2005 Equity Incentive Plan [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Schedule of Options Activity	Summarized information for the 2005 Equity Incentive Plan is as follows: Options Weighted- Outstanding options at January 1, 2017 328,560 $ 14.57 Expired/forfeited (11,800 ) 21.53 Outstanding options at December 31, 2017 316,760 $ 14.31 Expired/forfeited (360 ) 150.00 Outstanding options at December 31, 2018 316,400 $ 14.15 Expired/forfeited (56,400 ) 19.75 Outstanding options at December 31, 2019 260,000 $ 12.94
Schedule of Options Outstanding and Exercisable	Further information regarding options outstanding under the 2005 Equity Incentive Plan at December 31, 2019 is summarized below: Weighted-average Weighted-average Range of exercise prices Number of options outstanding Remaining life in years Exercise price Number of options exercisable Remaining life in years Exercise price $ 3.25 $ 3.25 120,000 0.1 $ 3.25 120,000 0.1 $ 3.25 11.50 30.50 136,100 4.1 18.48 136,100 4.1 18.48 113.50 122.50 3,900 0.9 117.65 3,900 0.9 117.65 260,000 260,000

Income Taxes (Tables)

Income Taxes (Tables)	12 Months Ended
	Dec. 31, 2019
Income Tax Disclosure [Abstract]
Schedule of Income Tax Expense	Income tax expense differs from the statutory amounts for each of the following years: 2019 2018 2017 Income taxes at U.S. statutory rate $ (16,020,000 ) $ (11,901,000 ) $ (9,289,000 ) Current year reserve 15,976,000 11,877,000 (25,175,000 ) Expenses not deductible 44,000 24,000 46,000 Rate change - - 34,418,000 Total tax expense $ - $ - $ -
Schedule of Deferred Tax Assets and Liabilities	The temporary differences that give rise to deferred tax assets and liabilities were as follows: December 31, 2019 December 31, 2018 Deferred tax assets (liabilities): Net operating loss carryforwards $ 50,802,000 $ 38,761,000 General business credit carryforwards 3,939,000 3,214,000 State credits 2,934,000 3,012,000 Property, equipment and goodwill (28,000 ) (72,000 ) Stock options 7,960,000 6,316,000 Derivatives - (57,000 ) Deferred revenue 62,000 62,000 Intangible assets 1,625,000 399,000 Other 28,000 80,000 Gross deferred tax assets 67,322,000 51,715,000 Valuation allowance (67,322,000 ) (51,715,000 ) Net deferred taxes $ - $ -
Summary of Net Operating Loss and General Business Credit Carryforwards	These carryforwards expire as follows: Net operating General business credit 2020 $ 5,125,000 $ 226,000 2021 5,378,000 56,000 2022 8,230,000 431,000 2023 5,434,000 362,000 2024 8,711,000 287,000 Thereafter 99,946,000 2,536,000 $ 132,824,000 $ 3,898,000

Quarterly Financial Data (Una_2

Quarterly Financial Data (Unaudited) (Tables)	12 Months Ended
	Dec. 31, 2019
Quarterly Financial Information Disclosure [Abstract]
Schedule of Quarterly Financial Information	The operating results for any quarter are not necessarily indicative of results for any future period. Quarters Ended December 31, September 30, June 30, March 31, Foundation revenues $ - $ - $ - $ - Royalties - - - - Research and development 9,605,000 10,917,000 16,307,000 11,737,000 General and administrative 4,734,000 4,700,000 5,612,000 5,659,000 Loss from operations (16,411,000 ) (17,649,000 ) (23,976,000 ) (19,054,000 ) Net loss (16,431,000 ) (17,372,000 ) (23,924,000 ) (18,555,000 ) Basic and diluted loss per common share (0.30 ) (0.35 ) (0.49 ) (0.39 ) Weighted average number of common shares outstanding – basic and diluted 54,718,776 49,721,753 48,961,988 47,948,421 Quarters Ended December 31, September 30, June 30, March 31, Foundation revenues $ 369,000 $ 1,687,000 $ 259,000 $ 481,000 Royalties 113,000 22,000 17,000 50,000 Research and development 9,470,000 13,150,000 7,916,000 8,162,000 General and administrative 7,631,000 4,970,000 4,627,000 2,878,000 Loss from operations (18,010,000 ) (16,916,000 ) (12,557,000 ) (10,683,000 ) Net loss (17,479,000 ) (16,419,000 ) (12,243,000 ) (10,530,000 ) Basic and diluted loss per common share (0.36 ) (0.34 ) (0.26 ) (0.22 ) Weighted average number of common shares outstanding – basic and diluted 47,944,486 47,794,394 47,303,518 47,060,523

Nature of Operations and Summ_4

Nature of Operations and Summary of Significant Accounting Policies (Details Narrative) - USD ($)	12 Months Ended
	Dec. 31, 2019	Dec. 31, 2018	Dec. 31, 2017	Jan. 02, 2019	Jan. 02, 2018	Oct. 16, 2017	Dec. 31, 2016
Cash, cash equivalents and short-term investments	$ 129,258,000
Net assets	178,430,000
Net cash used in operating activities	(62,820,000)	$ (39,111,000)	$ (22,655,000)
Allowance for doubtful accounts receivable
Receivables		81,000
Right-of-use lease assets	8,047,000
Operating lease liability	7,950,000
Goodwill	32,466,000	32,466,000
Goodwill impairment
Stockholders' equity	178,430,000	134,045,000	170,098,000		$ 6,300,000		$ 99,098,000
Deferred revenue	296,000	296,000	2,600,000		2,600,000
Accumulated deficit	(486,470,000)	(410,188,000)			2,600,000
Grants Receivable	5,700,000	5,700,000
Aggregate amount to the performance obligations	300,000	300,000
Accounting Standards Update 2016-02 [Member]
Right-of-use lease assets				$ 8,900,000
Operating lease liability				$ 8,900,000
MPS IIIA [Member]
Value in installments for the advancement						$ 13,900,000
MPS IIIB [Member]
Value in installments for the advancement						$ 13,900,000
Foundation Revenues [Member]
Deferred revenue	$ 0	$ 2,800,000
Licenses [Member]
Deferred revenue			$ 3,700,000		3,700,000
Accumulated deficit					$ 3,700,000

Nature of Operations and Summ_5

Nature of Operations and Summary of Significant Accounting Policies - Schedule of Statement of Operations (Details) - USD ($)	3 Months Ended								12 Months Ended
	Dec. 31, 2019	Sep. 30, 2019	Jun. 30, 2019	Mar. 31, 2019	Dec. 31, 2018	Sep. 30, 2018	Jun. 30, 2018	Mar. 31, 2018	Dec. 31, 2019	Dec. 31, 2018	Dec. 31, 2017
Total revenues										$ 2,998,000	$ 837,000
Loss from operations	$ (16,411,000)	$ (17,649,000)	$ (23,976,000)	$ (19,054,000)	$ (18,010,000)	$ (16,916,000)	$ (12,557,000)	$ (10,683,000)	(77,090,000)	(58,166,000)	(27,836,000)
Net loss	$ (16,431,000)	$ (17,372,000)	$ (23,924,000)	$ (18,555,000)	$ (17,479,000)	$ (16,419,000)	$ (12,243,000)	$ (10,530,000)	$ (76,282,000)	$ (56,671,000)	$ (27,319,000)
Basic and diluted loss per common share	$ (0.30)	$ (0.35)	$ (0.49)	$ (0.39)	$ (0.36)	$ (0.34)	$ (0.26)	$ (0.22)	$ (1.51)	$ (1.19)	$ (0.66)
Foundation Revenues [Member]
Total revenues					$ 369,000	$ 1,687,000	$ 259,000	$ 481,000		$ 2,796,000
License Revenues[Member]
Total revenues											$ 602,000
Under ASC 605 [Member]
Total revenues										804,000
Loss from operations										(60,360,000)
Net loss										$ (58,865,000)
Basic and diluted loss per common share										$ (1.24)
Under ASC 605 [Member] | Foundation Revenues [Member]
Total revenues
Under ASC 605 [Member] | License Revenues[Member]
Total revenues										602,000
Effect of ASC 606 [Member]
Total revenues										2,194,000
Loss from operations										2,194,000
Net loss										$ 2,194,000
Basic and diluted loss per common share										$ 0.05
Effect of ASC 606 [Member] | Foundation Revenues [Member]
Total revenues										$ 2,796,000
Effect of ASC 606 [Member] | License Revenues[Member]
Total revenues										$ (602,000)

Nature of Operations and Summ_6

Nature of Operations and Summary of Significant Accounting Policies - Schedule of Antidilutive Securities Excluded from Computation of Earnings Per Share (Details) - shares	12 Months Ended
	Dec. 31, 2019	Dec. 31, 2018	Dec. 31, 2017
Total	6,125,395	7,662,491	8,364,412
Warrants [Member]
Total	70,000	1,820,686	2,934,685
Stock Options [Member]
Total	6,055,395	5,841,805	5,429,727

Nature of Operations and Summ_7

Nature of Operations and Summary of Significant Accounting Policies - Schedule of Stock Based Compensation (Details) - USD ($)	12 Months Ended
	Dec. 31, 2019	Dec. 31, 2018	Dec. 31, 2017
Total stock based compensation expense	$ 7,338,000	$ 8,178,000	$ 4,644,000
Stock Option [Member]
Total stock based compensation expense	7,338,000	8,178,000	4,644,000
Tax benefit
Stock based compensation expense, net of tax	7,338,000	8,178,000	4,644,000
Stock Option [Member] | Research and Development Expense [Member]
Total stock based compensation expense	3,932,000	3,913,000	1,668,000
Stock Option [Member] | Selling General and Administrative Expenses [Member]
Total stock based compensation expense	3,406,000	4,265,000	2,976,000
Restricted Stock [Member]
Total stock based compensation expense	899,000	688,000	1,272,000
Tax benefit
Stock based compensation expense, net of tax	899,000	688,000	1,272,000
Restricted Stock [Member] | Research and Development Expense [Member]
Total stock based compensation expense	454,000
Restricted Stock [Member] | Selling General and Administrative Expenses [Member]
Total stock based compensation expense	$ 445,000	$ 688,000	$ 1,272,000

Short-Term Investments - Schedu

Short-Term Investments - Schedule of Available-for-sale Investments Held (Details) - USD ($)	Dec. 31, 2019	Dec. 31, 2018
Investments, Debt and Equity Securities [Abstract]
U.S. government and agency securities and treasuries		$ 66,218,000

Property and Equipment (Details

Property and Equipment (Details Narrative) - USD ($)	12 Months Ended
	Dec. 31, 2019	Dec. 31, 2018	Dec. 31, 2017
Property, Plant and Equipment [Abstract]
Depreciation and amortization	$ 2,600,000	$ 1,200,000	$ 200,000

Property and Equipment - Schedu

Property and Equipment - Schedule of Property and Equipment (Details) - USD ($)	Dec. 31, 2019	Dec. 31, 2018
Property and equipment, gross	$ 17,314,000	$ 11,003,000
Less: accumulated depreciation and amortization	4,157,000	1,560,000
Property and equipment, net	13,157,000	9,443,000
Laboratory Equipment [Member]
Property and equipment, gross	7,031,000	4,366,000
Furniture and Office Equipment [Member]
Property and equipment, gross	1,710,000	1,454,000
Leasehold Improvements [Member]
Property and equipment, gross	8,573,000	4,774,000
Construction Work-in-Progress [Member]
Property and equipment, gross		$ 409,000

Licensed Technology (Details Na

Licensed Technology (Details Narrative)	Apr. 01, 2020USD ($)	May 15, 2019shares	Nov. 04, 2018USD ($)	Aug. 03, 2016USD ($)Integer	Aug. 03, 2016USD ($)shares	May 15, 2015	Dec. 31, 2019USD ($)	Dec. 31, 2018USD ($)	Dec. 31, 2017USD ($)	Nov. 04, 2019USD ($)
Due to licensor current							$ 27,400,000	$ 10,000,000
Interest payable										$ 100,000
Value of licensed technology							36,178,000	43,042,000
Shares returned in connection with arbitration ruling on licensing agreement (in shares) | shares		450,000
Non-cash loss on arbitration ruling on licensing agreement							367,000
Amortization of intangible assets							$ 5,200,000	$ 1,200,000	$ 500,000
Abeona Therapeutics Llc [Member]
Finite-lived intangible asset, useful life						20 years
Eb Agreement [Member]
Finite-lived intangible asset, useful life				20 years
Number of license agreements research and development arrangements | Integer				2
Subsequent Event [Member]
Guaranteed and payable	$ 20,000,000
Maturity date	Nov. 4, 2020
Original License Agreements [Member]
License to be paid										10,000,000
Amended Agreements [Member]
License to be paid										3,000,000
Amended Agreements [Member] | April 1 2020 [Member]
License to be paid										$ 8,000,000
REGENXBIO [Member] | Licensing Agreements [Member]
Guaranteed upfront payment			$ 20,000,000
Payment on Execution of Contracts			10,000,000
Due to licensor current			10,000,000
Annual fees			100,000,000
Guaranteed and payable			$ 20,000,000
Maturity date			Nov. 4, 2020
Royalties payments			$ 60,000,000
Finite-lived intangible asset, useful life			8 years
EBRP [Member] | Eb Agreement [Member]
Issuance of common shares | shares					375,000
Value of licensed technology				$ 2,450,000	$ 2,450,000
EBMRF [Member] | Eb Agreement [Member]
Issuance of common shares | shares					375,000
Value of licensed technology				$ 2,450,000	$ 2,450,000

Licensed Technology - Schedule

Licensed Technology - Schedule of Licensed Technology (Details) - USD ($)	Dec. 31, 2019	Dec. 31, 2018
Goodwill and Intangible Assets Disclosure [Abstract]
Licensed technology	$ 42,606,000	$ 44,859,000
Less accumulated amortization	6,428,000	1,817,000
Licensed technology, net	$ 36,178,000	$ 43,042,000

Licensed Technology - Schedul_2

Licensed Technology - Schedule of Amortization Expense for Intangible Assets (Details) - USD ($)	Dec. 31, 2019	Dec. 31, 2018
Goodwill and Intangible Assets Disclosure [Abstract]
2020	$ 5,174,000
2021	5,174,000
2022	5,174,000
2023	5,174,000
2024	5,174,000
Thereafter	10,308,000
Total	$ 36,178,000	$ 43,042,000

401(k) Plan (Details Narrative)

401(k) Plan (Details Narrative)	12 Months Ended
	Dec. 31, 2019USD ($)Integer	Dec. 31, 2018USD ($)	Dec. 31, 2017USD ($)
Retirement Benefits [Abstract]
Maximum annual contribution per employee under 401(k) plan	$ 19,000	$ 18,500	$ 18,000
Number of investment options | Integer	50
Company contributions under 401 (k) Plan	$ 400,000	$ 300,000	$ 0

Commitments and Contingencies_2

Commitments and Contingencies (Details Narrative) - USD ($)	12 Months Ended
	Dec. 31, 2019	Dec. 31, 2018	Dec. 31, 2017
Operating lease, cost	$ 1,591,000
Rent expenses		$ 1,300,000	$ 700,000
RDEB Product [Member]
Contract manufacturer commitment	6,400,000
Contract manufacturer commitment, due in next twelve months	6,400,000
Leasehold Improvements [Member]
Operating lease, cost	$ 1,700,000
Maximum [Member]
Operating lease, term of contract	1 year

Commitments and Contingencies -

Commitments and Contingencies - Schedule of Components of Lease Cost (Details)	12 Months Ended
	Dec. 31, 2019USD ($)
Commitments and Contingencies Disclosure [Abstract]
Operating lease cost	$ 1,591,000
Variable lease cost	322,000
Short-term lease cost	$ 133,000

Commitments and Contingencies_3

Commitments and Contingencies - Schedule of Supplemental Cash Flow Information Related to Leases (Details) - USD ($)	Dec. 31, 2019	Dec. 31, 2018
Commitments and Contingencies Disclosure [Abstract]
2020	$ 1,699,000
2021	1,713,000
2022	1,727,000
2023	1,741,000
2024	1,781,000
Thereafter	1,885,000
Total undiscounted operating lease payments	10,546,000
Less: Imputed interest	2,596,000
Present value of operating lease liabilities	7,950,000
Current portion of lease liability	1,699,000
Long-term lease liability	6,251,000
Total operating lease liabilities	$ 7,950,000
Weighted-average remaining lease term for operating leases	73 months
Weighted-average discount rate for operating leases	9.60%

Fair Value Measurements - Sched

Fair Value Measurements - Schedule of Fair Value, Assets and Liabilities Measured on Recurring and Non-Recurring Basis (Details) - USD ($)	Dec. 31, 2019	Dec. 31, 2018
Licensed Technology Net [Member] | Fair Value Measurements Nonrecurring [Member]
Fair value disclosure of assets	$ 36,178,000	$ 43,042,000
Fair value disclosure of gain loss	(367,000)
Licensed Technology Net [Member] | Fair Value Measurements Recurring [Member] | Fair Value Inputs Level1 [Member]
Fair value disclosure of assets
Licensed Technology Net [Member] | Fair Value Measurements Recurring [Member] | Fair Value Inputs Level2 [Member]
Fair value disclosure of assets
Licensed Technology Net [Member] | Fair Value Measurements Recurring [Member] | Fair Value Inputs Level3 [Member]
Fair value disclosure of assets	36,178,000	43,042,000
Goodwill [Member] | Fair Value Measurements Nonrecurring [Member]
Fair value disclosure of assets	32,466,000	32,466,000
Fair value disclosure of gain loss
Goodwill [Member] | Fair Value Measurements Nonrecurring [Member] | Fair Value Inputs Level1 [Member]
Fair value disclosure of assets
Goodwill [Member] | Fair Value Measurements Nonrecurring [Member] | Fair Value Inputs Level2 [Member]
Fair value disclosure of assets
Goodwill [Member] | Fair Value Measurements Nonrecurring [Member] | Fair Value Inputs Level3 [Member]
Fair value disclosure of assets	$ 32,466,000	32,466,000
Short Term Investment [Member] | Fair Value Measurements Recurring [Member]
Fair value disclosure of assets		66,218,000
Fair value disclosure of gain loss
Short Term Investment [Member] | Fair Value Measurements Recurring [Member] | Fair Value Inputs Level1 [Member]
Fair value disclosure of assets
Short Term Investment [Member] | Fair Value Measurements Recurring [Member] | Fair Value Inputs Level2 [Member]
Fair value disclosure of assets		66,218,000
Short Term Investment [Member] | Fair Value Measurements Recurring [Member] | Fair Value Inputs Level3 [Member]
Fair value disclosure of assets

Stockholders' Equity (Details N

Stockholders' Equity (Details Narrative) - USD ($)	Dec. 24, 2019	Oct. 19, 2017	Dec. 31, 2019	Dec. 31, 2018	Dec. 31, 2017
Warrants to purchase			70,000
Warrants exercise price			$ 5	$ 5	$ 5
Issuance of common shares, value					$ 86,174,000
Proceeds from Warrant Exercises				$ 3,233,000	$ 885,000
Common Stock [Member]
Issuance of common shares					5,750,000
Issuance of common shares, value					$ 58,000
Non - Pre - Funded Warrants [Member]
Warrants to purchase			70,000	1,820,686
Proceeds from Warrant Exercises			$ 0	$ 3,200,000	$ 5,900,000
Non - Pre - Funded Warrants [Member] | Common Stock [Member]
Warrants to purchase			1,750,686
Warrant expiration, description			expired during 2019.
2019 Public Offering [Member]
Issuance of common shares	32,382,945
Shares issued, price per share	$ 2.50
Issuance of common shares, value	$ 103,500,000
2019 Public Offering [Member] | Pre - Funded Warrants [Member]
Warrants to purchase			9,017,055
Warrants exercise price	$ 0.0001
2019 Public Offering [Member] | Maximum [Member]
Warrants to purchase	9,017,055
Warrants exercise price	$ 2.4999
2017 Public Offering [Member]
Issuance of common shares		5,750,000
Shares issued, price per share		$ 16
Issuance of common shares, value		$ 92,000,000

Stockholders' Equity - Schedule

Stockholders' Equity - Schedule of Warrants Outstanding at Various Exercise Prices (Details) - $ / shares		12 Months Ended
		Dec. 31, 2019	Dec. 31, 2018	Dec. 31, 2017
Warrant outstanding		70,000
Warrant exercise price		$ 5	$ 5	$ 5
2015 Financing [Member]
Warrant outstanding	[1]	20,000
Warrant exercise price	[1]	$ 6.05
Warrants expiration date	[1]	Jul. 31, 2020
2015 Financing One[Member]
Warrant outstanding	[2]	50,000
Warrant exercise price	[2]	$ 11
Warrants expiration date	[2]	May 11, 2020
[1]	In connection with the offering on July 31, 2015, the placement agent received warrants to purchase 20,000 shares of common stock at $6.05 per share. The warrants are currently exercisable and expire on July 31, 2020.
[2]	In connection with the offering on May 11, 2015, the placement agent received warrants to purchase 50,000 shares of common stock at $11.00 per share. The warrants are currently exercisable and expire on May 11, 2020.

Stockholders' Equity - Schedu_2

Stockholders' Equity - Schedule of Warrants Outstanding at Various Exercise Prices (Details) (Parenthetical) - $ / shares	Jul. 31, 2015	May 11, 2015	Dec. 31, 2019	Dec. 31, 2018	Dec. 31, 2017
Warrant outstanding			70,000
Warrants exercise price			$ 5	$ 5	$ 5
Placement Agent [Member]
Warrant outstanding	20,000	50,000
Warrants exercise price	$ 6.05	$ 11
Warrants expiration date	Jul. 31, 2020	May 11, 2020

Stock Option Plans (Details Nar

Stock Option Plans (Details Narrative) - USD ($)	12 Months Ended
	Dec. 31, 2019	Dec. 31, 2018	Dec. 31, 2017	Dec. 31, 2016
Intrinsic value outstanding options	$ 400,000	$ 3,200,000	$ 600,000
2015 Equity Incentive Plan [Member]
Common stock authorized but unissued	10,000,000
Maximum contractual term	10 years
Intrinsic value outstanding options	$ 700,000	8,400,000	46,000,000
Intrinsic value exercisable options	600,000	$ 6,000,000	$ 25,600,000
Compensation cost related to non-vested options not recognized	$ 14,400,000
Weighted average period total compensation costs related to non-vested options	2 years 8 months 12 days
Shares of restricted common stock forfeited	22,000
Shares of restricted common stock vested
Compensation cost related to restricted common stock not recognized	$ 500,000
Weighted average compensation costs related to restricted common stock	2 months 12 days
Options outstanding and exercisable	5,795,395	5,525,405	5,112,967	4,443,000
2015 Equity Incentive Plan [Member] | Employees [Member]
Shares of restricted common stock granted	376,625
2005 Equity Incentive Plan [Member]
Intrinsic value outstanding options	$ 0	$ 500,000	$ 1,500,000
Options outstanding and exercisable	260,000	316,400	316,760	328,560

Stock Option Plans - Schedule o

Stock Option Plans - Schedule of Weighted-average Assumptions to Estimate the Fair Value of the Options Granted (Details)	12 Months Ended
	Dec. 31, 2019	Dec. 31, 2018	Dec. 31, 2017
Share-based Payment Arrangement [Abstract]
Expected volatility	108.00%	109.00%	111.00%
Expected term	5 years 1 month 6 days	5 years	5 years
Risk-free interest rate	2.21%	2.62%	1.81%
Expected dividend yield	0.00%	0.00%	0.00%

Stock Option Plans - Schedule_2

Stock Option Plans - Schedule of Options Activity (Details) - $ / shares	12 Months Ended
	Dec. 31, 2019	Dec. 31, 2018	Dec. 31, 2017
2015 Equity Incentive Plan [Member]
Beginning balance, Options, Outstanding options	5,525,405	5,112,967	4,443,000
Options, Granted, fair value	1,490,490	1,497,300	1,077,000
Options, Exercised	(303,129)	(360,853)	(81,719)
Options, Expired/forfeited	(917,371)	(724,009)	(325,314)
Ending balance, Options, Outstanding options	5,795,395	5,525,405	5,112,967
Options, Non-vested options	2,342,706	2,355,719	2,641,832
Beginning balance, Weighted- average exercise price, Outstanding options	$ 8.08	$ 9.07	$ 4.94
Weighted- average exercise price, Granted, fair value	6.53	12.95	14.31
Weighted- average exercise price, Exercised	3.20	6.22	4.22
Weighted- average exercise price, Expired/forfeited	7.95	10.65	5.52
Ending balance, Options, Outstanding options	7.96	8.08	9.07
Weighted- average exercise price, Non-vested options	$ 8.78	$ 10.41	$ 8.21
2005 Equity Incentive Plan [Member]
Beginning balance, Options, Outstanding options	316,400	316,760	328,560
Options, Expired/forfeited	(56,400)	(360)	(11,800)
Ending balance, Options, Outstanding options	260,000	316,400	316,760
Beginning balance, Weighted- average exercise price, Outstanding options	$ 14.15	$ 14.31	$ 14.57
Weighted- average exercise price, Expired/forfeited	19.75	150	21.53
Ending balance, Options, Outstanding options	$ 12.94	$ 14.15	$ 14.31

Stock Option Plans - Schedule_3

Stock Option Plans - Schedule of Options Activity (Details) (Parenthetical) - $ / shares	12 Months Ended
	Dec. 31, 2019	Dec. 31, 2018	Dec. 31, 2017
2015 Equity Incentive Plan [Member]
Fair value of granted	$ 5.14	$ 10.23	$ 11.35

Stock Option Plans - Schedule_4

Stock Option Plans - Schedule of Options Outstanding and Exercisable (Details) - $ / shares	12 Months Ended
	Dec. 31, 2019	Dec. 31, 2018	Dec. 31, 2017	Dec. 31, 2016
2015 Equity Incentive Plan [Member]
Number of options outstanding	5,795,395	5,525,405	5,112,967	4,443,000
Weighted-average Remaining life in years	10 years
Weighted-average Exercise price	$ 7.96	$ 8.08	$ 9.07	$ 4.94
Number of options exercisable	3,452,689
2015 Equity Incentive Plan [Member] | Range One [Member]
Range of exercise price, lower limit	$ 2.26
Range of exercise price, upper limit	$ 5.44
Number of options outstanding	1,989,150
Weighted-average Remaining life in years	7 years
Weighted-average Exercise price	$ 3.66
Number of options exercisable	1,462,346
Weighted-average Remaining life in years, exercisable	6 years 6 months
Weighted-average Exercise price, exercisable	$ 3.46
2015 Equity Incentive Plan [Member] | Range Two [Member]
Range of exercise price, lower limit	5.90
Range of exercise price, upper limit	$ 9.23
Number of options outstanding	2,425,240
Weighted-average Remaining life in years	7 years 7 months 6 days
Weighted-average Exercise price	$ 7.35
Number of options exercisable	1,253,189
Weighted-average Remaining life in years, exercisable	6 years 2 months 12 days
Weighted-average Exercise price, exercisable	$ 7.32
2015 Equity Incentive Plan [Member] | Range Three [Member]
Range of exercise price, lower limit	11.43
Range of exercise price, upper limit	$ 14.45
Number of options outstanding	456,619
Weighted-average Remaining life in years	8 years 6 months
Weighted-average Exercise price	$ 13.49
Number of options exercisable	204,025
Weighted-average Remaining life in years, exercisable	8 years 4 months 24 days
Weighted-average Exercise price, exercisable	$ 13.56
2015 Equity Incentive Plan [Member] | Range Four [Member]
Range of exercise price, lower limit	15.25
Range of exercise price, upper limit	$ 17.30
Number of options outstanding	924,386
Weighted-average Remaining life in years	8 years
Weighted-average Exercise price	$ 16.09
Number of options exercisable	533,129
Weighted-average Remaining life in years, exercisable	8 years
Weighted-average Exercise price, exercisable	$ 16.07
2005 Equity Incentive Plan [Member]
Number of options outstanding	260,000	316,400	316,760	328,560
Weighted-average Exercise price	$ 12.94	$ 14.15	$ 14.31	$ 14.57
Number of options exercisable	260,000
2005 Equity Incentive Plan [Member] | Range One [Member]
Range of exercise price, lower limit	$ 3.25
Range of exercise price, upper limit	$ 3.25
Number of options outstanding	120,000
Weighted-average Remaining life in years	1 month 6 days
Weighted-average Exercise price	$ 3.25
Number of options exercisable	120,000
Weighted-average Remaining life in years, exercisable	1 month 6 days
Weighted-average Exercise price, exercisable	$ 3.25
2005 Equity Incentive Plan [Member] | Range Two [Member]
Range of exercise price, lower limit	11.50
Range of exercise price, upper limit	$ 30.50
Number of options outstanding	136,100
Weighted-average Remaining life in years	4 years 1 month 6 days
Weighted-average Exercise price	$ 18.48
Number of options exercisable	136,100
Weighted-average Remaining life in years, exercisable	4 years 1 month 6 days
Weighted-average Exercise price, exercisable	$ 18.48
2005 Equity Incentive Plan [Member] | Range Three [Member]
Range of exercise price, lower limit	113.50
Range of exercise price, upper limit	$ 122.50
Number of options outstanding	3,900
Weighted-average Remaining life in years	10 months 25 days
Weighted-average Exercise price	$ 117.65
Number of options exercisable	3,900
Weighted-average Remaining life in years, exercisable	10 months 25 days
Weighted-average Exercise price, exercisable	$ 117.65

Income Taxes (Details Narrative

Income Taxes (Details Narrative) - USD ($)	12 Months Ended
	Dec. 31, 2019	Dec. 31, 2018	Dec. 31, 2017
Income Tax Disclosure [Abstract]
Net operating loss carryforwards	$ 132,824,000
General business credit carryforwards	3,898,000
Net operating loss carryforwards not expired and carried forward indefinitely, amount	$ 109,100,000
Income tax, description	On December 22, 2017, President Donald Trump signed the Tax Cuts and Jobs Act (the "TCJA"). The TCJA reduces the U.S. federal corporate tax rate from 34% to 21%.
Effective Income Tax Rate Reconciliation, at Federal Statutory Income Tax Rate, Percent	21.00%
Deferred tax asset and liabilities			$ 34,400,000

Income Taxes - Schedule of Inco

Income Taxes - Schedule of Income Tax Expense (Details) - USD ($)	12 Months Ended
	Dec. 31, 2019	Dec. 31, 2018	Dec. 31, 2017
Income Tax Disclosure [Abstract]
Income taxes at U.S. statutory rate	$ (16,020,000)	$ (11,901,000)	$ (9,289,000)
Current year reserve	15,976,000	11,877,000	(25,175,000)
Expenses not deductible	44,000	24,000	46,000
Rate change			34,418,000
Total tax expense

Income Taxes - Schedule of Defe

Income Taxes - Schedule of Deferred Tax Assets and Liabilities (Details) - USD ($)	Dec. 31, 2019	Dec. 31, 2018	Dec. 31, 2017
Income Tax Disclosure [Abstract]
Net operating loss carryforwards	$ 50,802,000	$ 38,761,000
General business credit carryforwards	3,939,000	3,214,000
State credits	2,934,000	3,012,000
Property, equipment and goodwill	(28,000)	(72,000)
Stock options	7,960,000	6,316,000
Derivatives		(57,000)
Deferred revenue	62,000	62,000
Intangible assets	1,625,000	399,000
Other	28,000	80,000
Gross deferred tax assets	67,322,000	51,715,000
Valuation allowance	(67,322,000)	(51,715,000)
Net deferred taxes			$ 34,400,000

Income Taxes - Summary of Net O

Income Taxes - Summary of Net Operating Loss and General Business Credit Carryforwards (Details)	Dec. 31, 2019USD ($)
Net operating loss carryforwards	$ 132,824,000
General business credit carryforwards	3,898,000
Tax Year 2020 [Member]
Net operating loss carryforwards	5,125,000
General business credit carryforwards	226,000
Tax Year 2021 [Member]
Net operating loss carryforwards	5,378,000
General business credit carryforwards	56,000
Tax Year 2022 [Member]
Net operating loss carryforwards	8,230,000
General business credit carryforwards	431,000
Tax Year 2023 [Member]
Net operating loss carryforwards	5,434,000
General business credit carryforwards	362,000
Tax Year 2024 [Member]
Net operating loss carryforwards	8,711,000
General business credit carryforwards	287,000
Thereafter [Member]
Net operating loss carryforwards	99,946,000
General business credit carryforwards	$ 2,536,000

Quarterly Financial Data (Una_3

Quarterly Financial Data (Unaudited) - Schedule of Quarterly Financial Information (Details) - USD ($)	3 Months Ended								12 Months Ended
	Dec. 31, 2019	Sep. 30, 2019	Jun. 30, 2019	Mar. 31, 2019	Dec. 31, 2018	Sep. 30, 2018	Jun. 30, 2018	Mar. 31, 2018	Dec. 31, 2019	Dec. 31, 2018	Dec. 31, 2017
Revenues										$ 2,998,000	$ 837,000
Research and development	$ 9,605,000	$ 10,917,000	$ 16,307,000	$ 11,737,000	$ 9,470,000	$ 13,150,000	$ 7,916,000	$ 8,162,000	48,566,000	38,698,000	16,989,000
General and administrative	4,734,000	4,700,000	5,612,000	5,659,000	7,631,000	4,970,000	4,627,000	2,878,000	20,705,000	20,106,000	10,943,000
Loss from operations	(16,411,000)	(17,649,000)	(23,976,000)	(19,054,000)	(18,010,000)	(16,916,000)	(12,557,000)	(10,683,000)	(77,090,000)	(58,166,000)	(27,836,000)
Net loss	$ (16,431,000)	$ (17,372,000)	$ (23,924,000)	$ (18,555,000)	$ (17,479,000)	$ (16,419,000)	$ (12,243,000)	$ (10,530,000)	$ (76,282,000)	$ (56,671,000)	$ (27,319,000)
Basic and diluted loss per common share	$ (0.30)	$ (0.35)	$ (0.49)	$ (0.39)	$ (0.36)	$ (0.34)	$ (0.26)	$ (0.22)	$ (1.51)	$ (1.19)	$ (0.66)
Weighted average number of common shares outstanding - basic and diluted	54,718,776	49,721,753	48,961,988	47,948,421	47,944,486	47,794,394	47,303,518	47,060,523	50,354,596	47,528,248	41,636,752
Foundation Revenues [Member]
Revenues					$ 369,000	$ 1,687,000	$ 259,000	$ 481,000		$ 2,796,000
Royalties [Member]
Revenues					$ 113,000	$ 22,000	$ 17,000	$ 50,000		$ 202,000	$ 235,000