Intellia Therapeutics (NTLA) 10-Q 3 Jun 16 2016 Q1 Quarterly report Financial data

Document and Entity Information

Document and Entity Information - shares	3 Months Ended
	Mar. 31, 2016	Jun. 02, 2016
Document And Entity Information [Abstract]
Document Type	10-Q
Amendment Flag	false
Document Period End Date	Mar. 31, 2016
Document Fiscal Year Focus	2,016
Document Fiscal Period Focus	Q1
Trading Symbol	NTLA
Entity Registrant Name	INTELLIA THERAPEUTICS, INC.
Entity Central Index Key	1,652,130
Current Fiscal Year End Date	--12-31
Entity Filer Category	Non-accelerated Filer
Entity Common Stock, Shares Outstanding		35,996,265

Consolidated Balance Sheets

Consolidated Balance Sheets - USD ($) $ in Thousands	Mar. 31, 2016	Dec. 31, 2015
Current Assets:
Cash and cash equivalents	$ 64,192	$ 75,816
Accounts receivable	1,000	1,000
Prepaid expenses and other current assets	745	810
Total current assets	65,937	77,626
Property and equipment, net	4,228	2,708
Other assets	5,375	1,805
Total Assets	75,540	82,139
Current Liabilities:
Accounts payable	1,206	1,360
Accrued expenses	2,973	2,788
Current portion of deferred revenue	6,781	6,547
Total current liabilities	10,960	10,695
Deferred revenue, net of current portion	2,753	3,765
Other long-term liabilities	$ 349	$ 323
Commitments and contingencies (Note 6)
Convertible preferred stock (Series B, Series A-2, Series A-1, Junior and Founder), $0.0001 par value; 36,500,000 shares authorized and 36,316,628 shares issued and outstanding; aggregate liquidation preference of $95,946	$ 88,620	$ 88,557
Stockholders' Deficit:
Common stock, $0.0001 par value; 50,000,000 shares authorized; 2,558,755 shares issued and outstanding	0	0
Additional paid-in capital	1,483	735
Accumulated deficit	(28,625)	(21,936)
Total stockholders' deficit	(27,142)	(21,201)
Total Liabilities and Stockholders' Deficit	$ 75,540	$ 82,139

Consolidated Balance Sheets (Pa

Consolidated Balance Sheets (Parenthetical) - USD ($) $ in Thousands	Mar. 31, 2016	Dec. 31, 2015
Common stock, par value	$ 0.0001	$ 0.0001
Common stock, shares authorized	50,000,000	50,000,000
Common stock, shares issued	2,558,755	2,558,755
Common stock, shares outstanding	2,558,755	2,558,755
Convertible Preferred Stock (Series B, Series A-2, Series A-1, Junior and Founder) [Member]
Convertible preferred stock, par value	$ 0.0001	$ 0.0001
Convertible preferred stock, shares authorized	36,500,000	36,500,000
Convertible preferred stock, shares issued	36,316,628	36,316,628
Convertible preferred stock, shares outstanding	36,316,628	36,316,628
Convertible preferred stock, aggregate liquidation preference	$ 95,946	$ 95,946

Consolidated Statements of Oper

Consolidated Statements of Operations - USD ($) shares in Thousands, $ in Thousands	3 Months Ended
	Mar. 31, 2016	Mar. 31, 2015
Income Statement [Abstract]
Collaboration revenue	$ 1,777	$ 1,286
Operating expenses:
Research and development	5,225	1,371
General and administrative	3,246	1,110
Total operating expenses	8,471	2,481
Operating loss	(6,694)	(1,195)
Interest income	5
Loss before income taxes	(6,689)	(1,195)
Benefit from income taxes	0	102
Net loss	$ (6,689)	$ (1,093)
Net loss per common unit, basic and diluted		$ (0.85)
Net loss per share attributable to common stockholders, basic and diluted	$ (9.89)
Weighted average common units outstanding, basic and diluted		1,284
Weighted average shares outstanding, basic and diluted	676

Consolidated Statements of Cash

Consolidated Statements of Cash Flows - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2016	Mar. 31, 2015
CASH FLOWS FROM OPERATING ACTIVITIES:
Net loss	$ (6,689)	$ (1,093)
Adjustments to reconcile net loss to net cash (used in) provided by operating activities:
Depreciation and amortization	187	33
Equity-based compensation	812	115
Benefit from intraperiod tax allocation		(102)
Changes in operating assets and liabilities:
Prepaid expenses and other current assets	65	(68)
Accounts payable	(87)	360
Accrued expenses	(134)	(561)
Deferred revenue	(778)	6,070
Other assets	(2,823)
Other long-term liabilities	26	68
Net cash (used in) provided by operating activities	(9,421)	4,822
CASH FLOWS FROM INVESTING ACTIVITIES:
Purchases of property and equipment	(1,008)	(516)
Net cash used in investing activities	(1,008)	(516)
CASH FLOWS FROM FINANCING ACTIVITIES:
Proceeds from sale of Class A-2 preferred units		4,644
Payments to acquire in-process research and development	(200)	(300)
Payment of preferred unit and preferred stock issuance costs	(100)	(16)
Payment of proposed public offering costs	(895)
Net cash (used in) provided by financing activities	(1,195)	4,328
Net (decrease) increase in cash and cash equivalents	(11,624)	8,634
Cash and cash equivalents, beginning of period	75,816	9,845
Cash and cash equivalents, end of period	64,192	18,479
SUPPLEMENTAL DISCLOSURES OF CASH FLOW INFORMATION:
Purchases of property and equipment unpaid at period end	918	307
Acquisition of in-process research and development unpaid at period end	400	$ 1,400
Financing costs incurred but unpaid at period end	$ 454

Overview and Basis of Presentat

Overview and Basis of Presentation	3 Months Ended
	Mar. 31, 2016
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Overview and Basis of Presentation	1. Overview and Basis of Presentation Intellia Therapeutics, Inc. (collectively referred to with its wholly-owned, controlled subsidiary, Intellia Securities Corp., as “Intellia” or the “Company”) is a gene editing company focused on developing potentially curative therapeutics utilizing a recently developed biological tool known as CRISPR/Cas9. The consolidated financial statements of the Company included herein have been prepared, without audit, pursuant to the rules and regulations of the Securities and Exchange Commission (“SEC”). Certain information and footnote disclosures normally included in financial statements prepared in accordance with accounting principles generally accepted in the United States of America have been condensed or omitted from this report, as is permitted by such rules and regulations. Accordingly, these consolidated financial statements should be read in conjunction with the financial statements and notes thereto included in the Company’s Prospectus that forms a part of the Company’s Registration Statement on Form S-1 (File No. 333-210689), which was filed with the SEC pursuant to Rule 424(b)(4) on May 5, 2016 (the “Prospectus”). The unaudited consolidated financial statements include the accounts of Intellia Therapeutics, Inc. and its subsidiary. All intercompany transactions and balances of the subsidiary have been eliminated in consolidation. In the opinion of management, the information furnished reflects all adjustments, all of which are of a normal and recurring nature, necessary for a fair presentation of the results for the reported interim periods. The Company considers events or transactions that occur after the balance sheet date but before the financial statements are issued to provide additional evidence relative to certain estimates or to identify matters that require additional disclosure. The three months ended March 31, 2016 and 2015 are referred to as the first quarter of 2016 and 2015, respectively. The results of operations for interim periods are not necessarily indicative of results to be expected for the full year or any other interim period. On May 11, 2016, the Company completed an initial public offering (“IPO”) of its common stock, which resulted in the sale of 6,900,000 shares, including all additional shares available to cover over-allotments, at a price of $18.00 per share. The Company received net proceeds before expenses from the IPO of $115.5 million after deducting underwriting discounts and commissions paid by the Company. In preparation for the IPO, the Company’s Board of Directors and stockholders approved a one-for-1.7 reverse stock split of the Company’s common stock effective April 25, 2016. All share and per share amounts in the consolidated financial statements and notes thereto have been retroactively adjusted, where necessary, to give effect to this reverse stock split. In connection with the closing of the IPO, all of the Company’s outstanding convertible preferred stock automatically converted to common stock at a one-for-0.6465903 ratio as of May 11, 2016, resulting in an additional 23,481,956 shares of common stock of the Company becoming outstanding. In addition, the Company issued a total of 3,055,554 shares of common stock for $55.0 million in two separate, concurrent private placements upon the closing of the IPO. The significant increase in shares outstanding in May 2016 is expected to impact the year-over-year comparability of the Company’s (loss) earnings per share calculations for the next twelve months.

Summary of Significant Accounti

Summary of Significant Accounting Policies	3 Months Ended
	Mar. 31, 2016
Accounting Policies [Abstract]
Summary of Significant Accounting Policies	2. Summary of Significant Accounting Policies Recent Accounting Pronouncements In May 2014, the Financial Accounting Standards Board (“FASB”) issued Accounting Standards Update (“ASU”) No. 2014-09, Revenue from Contracts with Customers (Topic 606), Revenue From Contracts With Customers: Identifying Performance Obligations and Licensing In August 2014, the FASB issued ASU No. 2014-15, Disclosure of Uncertainties About an Entity’s Ability to Continue as a Going Concern Presentation of Financial Statements—Going Concern In April 2015, the FASB issued ASU No. 2015-05, Customer’s Accounting for Fees Paid in a Cloud Computing Arrangement Internal-Use Software In February 2016, the FASB issued ASU 2016-02, Leases Leases In March 2016, the FASB issued ASU, 2016-09, Improvements to Employee Share-Based Payment Accounting Compensation – Stock Compensation

Fair Value Measurements

Fair Value Measurements	3 Months Ended
	Mar. 31, 2016
Fair Value Disclosures [Abstract]
Fair Value Measurements	3. Fair Value Measurements The Company classifies fair value based measurements using a three-level hierarchy that prioritizes the inputs used to measure fair value. This 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 market prices in active markets for identical assets or liabilities; Level 2, observable inputs other than quoted market prices included in Level 1 such as quoted market prices for markets that are not active or other inputs that are observable or can be corroborated by observable market data; and Level 3, unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities, including certain pricing models, discounted cash flow methodologies and similar techniques that use significant unobservable inputs. The Company’s financial instruments as of March 31, 2016 and December 31, 2015 consisted primarily of cash and cash equivalents, accounts receivable and accounts payable. As of March 31, 2016 and December 31, 2015, the Company’s financial assets recognized at fair value consisted of the following: Fair Value as of March 31, 2016 Total Level 1 Level 2 Level 3 (In thousands) Cash equivalents $ 30,005 $ 30,005 $ — $ — Total $ 30,005 $ 30,005 $ — $ — Fair Value as of December 31, 2015 Total Level 1 Level 2 Level 3 (In thousands) Cash equivalents $ 30,000 $ 30,000 $ — $ — Total $ 30,000 $ 30,000 $ — $ —

Accrued Expenses

Accrued Expenses	3 Months Ended
	Mar. 31, 2016
Payables and Accruals [Abstract]
Accrued Expenses	4. Accrued Expenses Accrued expenses consisted of the following: March 31, 2016 December 31, (In thousands) Employee compensation and benefits $ 625 $ 1,281 In-process research and development obligation 400 600 Research and development and professional expenses 1,948 907 Accrued expenses $ 2,973 $ 2,788 In July 2014, the Company entered into agreements with Caribou Biosciences, Inc. (“Caribou”), under which the Company received a license for certain patents and limited research and development services from Caribou. The in-process research and development obligation represents the portion of the Company’s obligation under these agreements that is attributable to the license.

Income Taxes

Income Taxes	3 Months Ended
	Mar. 31, 2016
Income Tax Disclosure [Abstract]
Income Taxes	5. Income Taxes The Company did not record a federal or state income tax provision or benefit for the three months ended March 31, 2016 due to an expected loss before income taxes to be incurred for the year ended December 31, 2016, as well as the Company’s continued maintenance of a full valuation allowance against its net deferred tax assets. During the three months ended March 31, 2015, the Company allocated $2.6 million from the total fixed amount of consideration under a collaboration agreement with Novartis Institutes for BioMedical Research, Inc. (“Novartis”) to the carrying value of Class A-1 Collaboration

Commitments and Contingencies

Commitments and Contingencies	3 Months Ended
	Mar. 31, 2016
Commitments and Contingencies Disclosure [Abstract]
Commitments and Contingencies	6. Commitments and Contingencies Commitments In the first quarter of 2016, the Company entered into a ten-year agreement to lease office and laboratory space in Cambridge, Massachusetts under an operating lease agreement, with an option to terminate the lease at the end of the sixth year and an option to extend the term of the lease for an additional three years. Upon the execution of this lease, the Company provided a $2.2 million security deposit, which has been recorded in other assets on the consolidated balance sheet. Future minimum lease payments under this lease by year as of March 31, 2016 are as follows: (In thousands) Future minimum lease payments in year ending December 31: 2016 $ 370 2017 4,464 2018 4,598 2019 4,736 2020 4,878 Thereafter 9,786 Total future minimum lease payments $ 28,832

Collaboration

Collaboration	3 Months Ended
	Mar. 31, 2016
Organization, Consolidation and Presentation of Financial Statements [Abstract]
Collaboration	7. Collaboration In December 2014, the Company entered into a strategic collaboration agreement with Novartis focused on the ex vivo Agreement Structure Under the terms of the collaboration, the Company and Novartis may research potential therapeutic, prophylactic and palliative applications of the CRISPR/Cas9 platform in HSCs and CAR T cells. Within the HSC therapeutic space, Novartis may obtain exclusive rights to a limited number of HSC targets, to be selected by Novartis in a series of selection windows, the last of which closes 90 days before the fifth anniversary of the effective date of the collaboration agreement. If Novartis does not exercise its selection rights within each selection window, any such rights will be deemed forfeited by Novartis. Novartis is required to use commercially reasonable efforts to research, develop or commercialize at least one HSC product directed to at least one of their selected HSC targets. The Company also agreed to collaborate with Novartis on research activities for CAR T cell targets under a research plan agreed upon by both parties. After completion of the research and development activities contemplated by the CAR T cell program research plan, Novartis will assume sole responsibility for developing any products arising from that research plan and will be responsible for additional costs and expenses of developing, manufacturing and commercializing selected research targets. Novartis is required to use commercially reasonable efforts to research, develop or commercialize at least one CAR T cell product directed to at least one of their selected CAR T cell targets. In the last two years of the collaboration term, Novartis will have the option to select a limited number of targets for research, development and commercialization of in vivo in vivo in vivo The Company received an upfront technology access payment from Novartis of $10.0 million in January 2015 and is entitled to additional technology access fees of $20.0 million and quarterly research payments of $1.0 million, or up to $20.0 million in the aggregate, during the five-year research term. For each product under the collaboration, the Company may be eligible to receive (i) up to $30.3 million in development milestones, including for the filing of an investigational new drug application and for the dosing of the first patient in each of Phase IIa, Phase IIb and Phase III clinical trials, (ii) up to $50.0 million in regulatory milestones for the product’s first indication, including regulatory approvals in the United States (“U.S.”) and European Union (“EU”), (iii) up to $50.0 million in regulatory milestones for the product’s second indication, if any, including U.S. and EU regulatory approvals, (iv) royalties on net sales in the mid-single digits, and (v) net sales milestone payments of up to $100.0 million. The Company may also be eligible to receive payments for: (i) each additional HSC target selected by Novartis beyond its initial defined allocation, (ii) each in vivo Collaboration Revenue Through March 31, 2016, excluding amounts allocated to Novartis’ purchase of the Company’s Class A-1 and Class A-2 Preferred Units, the Company had recorded a total of $17.4 million in cash and accounts receivable under the Novartis agreement. Through March 31, 2016, the Company has recognized $7.9 million of collaboration revenue, including $1.8 million and $1.3 million in the three months ended March 31, 2016 and 2015, respectively, in the consolidated statements of operations related to this agreement. As of March 31, 2016 and December 31, 2015, the Company had deferred revenue of $9.5 million and $10.3 million, respectively, related to this agreement.

Equity-Based Compensation

Equity-Based Compensation	3 Months Ended
	Mar. 31, 2016
Disclosure of Compensation Related Costs, Share-based Payments [Abstract]
Equity-Based Compensation	8. Equity-Based Compensation Equity-based compensation expense is classified in the consolidated statements of operations as follows: Three Months Ended March 31, 2016 2015 (In thousands) Research and development $ 454 $ 56 General and administrative 358 59 Total $ 812 $ 115 Founder Stock and Restricted Stock Compensatory Founder Stock and restricted stock are valued at the fair value of the underlying security. The Company valued these awards by taking into consideration its most recently available valuation performed by management and the board of directors, considering the most recently available third-party valuations of the Company’s securities as well as additional qualitative factors. The following table summarizes the Company’s compensatory Founder Stock activity for the three months ended March 31, 2016: Number of Weighted Unvested Founder Stock as of January 1, 2016 436,509 $ 0.37 Vested (42,244 ) 0.37 Unvested Founder Stock as of March 31, 2016 394,265 $ 0.37 The following table summarizes the Company’s restricted stock activity for the three months ended March 31, 2016: Number of Weighted Unvested restricted stock as of January 1, 2016 1,945,036 $ 0.78 Vested (170,807 ) 0.73 Unvested restricted stock as of March 31, 2016 1,774,229 $ 0.81 As of March 31, 2016, there was $3.3 million of unrecognized equity-based compensation expense related to Founder Stock and restricted stock that is expected to vest. These costs are expected to be recognized over a weighted average remaining vesting period of 2.7 years. Stock Options The weighted-average fair value of options, estimated as of the grant date using the Black-Scholes option pricing model, was $4.98 per option for those options granted during the three months ended March 31, 2016. There were no stock option awards granted in the three months ended March 31, 2015. Key assumptions used to apply this pricing model were as follows: Three Months Ended Risk-free interest rate 1.3 % Expected life of options 6.0 years Expected volatility of underlying stock 87.7 % Expected dividend yield 0.0 % The following is a summary of stock option activity for the three months ended March 31, 2016: Number of Weighted Average Exercise Price per Weighted Average Remaining Contractual Term Aggregate Intrinsic Value (In years) (In thousands) Outstanding at January 1, 2016 456,374 $ 6.04 Granted 2,161,558 6.83 Outstanding at March 31, 2016 2,617,932 $ 6.69 9.9 $ 212 Exercisable at March 31, 2016 — $ — — $ — As of March 31, 2016, there was $11.8 million of unrecognized compensation cost related to stock options that are expected to vest. These costs are expected to be recognized over a weighted average remaining vesting period of 3.8 years.

Loss Per Share

Loss Per Share	3 Months Ended
	Mar. 31, 2016
Earnings Per Share [Abstract]
Loss Per Share	9. Loss Per Share The Company calculates basic earnings (loss) per common unit by dividing income (loss) allocable to common unitholders by the weighted average number of common units outstanding, calculates basic earnings (loss) per incentive unit by dividing income (loss) allocable to incentive unitholders by the weighted average number of incentive units outstanding and calculates basic earnings (loss) per share by dividing income (loss) allocable to common stockholders by the weighted average number of common shares outstanding. During periods of income, the Company allocates to participating securities a proportional share of income determined by dividing total weighted average participating securities by the sum of the total weighted average common shares and participating securities (the “two-class method”). The Company’s preferred units, preferred stock, common units, common stock, incentive units and restricted common stock have rights to earnings and to participate in distributions of the Company and are therefore considered to be participating securities. Participating securities have the effect of diluting both basic and diluted earnings per share during periods of income. During periods of loss, the Company allocates no loss to preferred units or preferred stock because they have no contractual obligation to share in the losses of the Company. The Company computes diluted earnings (loss) per share after giving consideration to the dilutive effect of preferred units, preferred stock, common units, common stock, incentive units and restricted common stock that are outstanding during the period, except where such units would be anti-dilutive. Basic and diluted loss per common unit was calculated as follows: Three Months Ended (In thousands except Net loss $ (1,093 ) Weighted average common units outstanding, basic and diluted 1,284 Net loss per common unit, basic and diluted $ (0.85 ) Following the Reorganization, the Company calculates loss per share attributable to common stockholders based on its outstanding common stock. Basic and diluted loss per share attributable to common stockholders was calculated as follows: Three Months Ended (In thousands except Net loss $ (6,689 ) Weighted average shares outstanding, basic and diluted 676 Net loss per share attributable to common stockholders, basic and diluted $ (9.89 ) In May 2016, the Company issued an additional 6,900,000 shares of common stock in connection with its IPO and 23,481,956 shares of common stock in connection with the automatic conversion of its convertible preferred stock upon the closing of the IPO. In addition, the Company issued a total of 3,055,554 shares of common stock in two separate, concurrent private placements upon the closing of the IPO. The issuance of these shares will result in a significant increase in the Company’s weighted average shares outstanding and impact the year-over-year comparability of the Company’s (loss) earnings per share calculations for the next twelve months. The following common stock equivalents were excluded from the calculation of diluted loss per unit or share because their inclusion would have been anti-dilutive: Three Months Ended March 31, 2016 2015 (In thousands) Preferred units — 20,682 Unvested common units — 1,014 Unvested incentive units — 1,479 Unvested restricted stock 1,774 — Stock options 2,618 — 4,392 23,175

Related Party Transactions

Related Party Transactions	3 Months Ended
	Mar. 31, 2016
Related Party Transactions [Abstract]
Related Party Transactions	10. Related Party Transactions In July 2014, the Company issued Caribou Therapeutics Holdco, LLC, a wholly-owned subsidiary of Caribou Biosciences, Inc., or Caribou, 8,110,599 Junior Preferred Units. As a result of this and related transactions, Caribou owned 19.2% of the Company’s fully diluted equity as of March 31, 2016. During each of the three months ended March 31, 2016 and 2015, the Company recognized $0.4 million in research and development expense and, as of March 31, 2016 and December 31, 2015, had current obligations of $0.4 million and $0.6 million, respectively, related to license and service agreements entered into with Caribou. In addition, the Company recognized $0.2 million in general and administrative expense during each of the three months ended March 31, 2016 and 2015, related to the Company’s obligation to pay 30.0% of Caribou’s patent prosecution, filing and maintenance costs under its intellectual property license agreement with Caribou. In connection with its entry into the collaboration and license agreement and related equity transactions with Novartis, the Company issued Novartis 4,761,905 Class A-1 Preferred Units and 2,666,666 Class A-2 Preferred Units. In August 2015, Novartis acquired 761,905 shares of the Company’s Series B Preferred Stock. As a result of these transaction, Novartis collectively owned 18.2% of the Company’s fully diluted equity as of March 31, 2016. Refer to Note 7, Collaboration During the three months ended March 31, 2016 and 2015, the Company recognized $1.8 million and $1.3 million, respectively, in collaboration revenue related to this collaboration. As of March 31, 2016 and December 31, 2015, the Company had recorded accounts receivable of $1.0 million and deferred revenue of $9.5 million and $10.3 million, respectively, related to this collaboration.

Subsequent Events

Subsequent Events	3 Months Ended
	Mar. 31, 2016
Subsequent Events [Abstract]
Subsequent Events	11. Subsequent Events Collaboration with Regeneron Pharmaceuticals, Inc. In April 2016, the Company entered into a license and collaboration agreement with Regeneron Pharmaceuticals, Inc. (“Regeneron”). The agreement includes a product component to research, develop and commercialize CRISPR/Cas-based therapeutic products primarily focused on gene editing in the liver as well as a technology collaboration component, pursuant to which the Company and Regeneron will engage in research and development activities aimed at discovering and developing novel technologies and improvements to CRISPR/Cas technology to enhance the Company’s gene editing platform. Under this agreement, the Company also has the ability to access the Regeneron Genetics Center and proprietary mouse models to be provided by Regeneron for a limited number of the Company’s liver programs. Under the terms of the collaboration, the Company and Regeneron have agreed to a target selection process, whereby Regeneron may obtain exclusive rights for up to 10 targets to be chosen by Regeneron during the collaboration term, subject to various adjustments and limitations set forth in the agreement. Of these 10 total targets, Regeneron may select up to five non-liver targets, while the remaining targets will be focused in the liver. At the inception of the agreement, Regeneron selected the first of its ten targets, which is subject to a co-development and co-commercialization arrangement between the Company and Regeneron. The Company retains the exclusive right to solely develop products for certain indications. During the target selection process, the Company has the right to choose additional liver targets for its own development using commercially reasonable efforts. Certain targets that either the Company or Regeneron select may be subject to further co-development and co-commercialization arrangements at the Company or Regeneron’s option, as applicable. In addition, subject to certain restrictions, Regeneron will be able to replace a limited number of targets with substitute targets upon the payment of a specified replacement fee, in which case exclusive rights to the replaced target revert to the Company. Regeneron’s target selections are subject to certain additional restrictions, including that non-liver targets are not the subject of ongoing or planned research and development by the Company or are not the subject of a collaboration or pending collaboration with a third party. A joint steering committee consisting of an equal number of representatives from the Company and Regeneron will oversee the general strategies and activities undertaken by the parties under the collaboration. Research activities under the collaboration will be governed by evaluation and research and development plans that will outline the parties’ responsibilities under, anticipated timelines of and budgets for, the various programs. The Company will assist Regeneron with the preliminary evaluation of liver targets, and Regeneron will be responsible for preclinical research and the conduct of clinical development, manufacturing and commercialization of products directed to each of its exclusive targets under the oversight of the joint steering committee. The Company may assist, as requested by Regeneron, with the later discovery and research of product candidates directed to any target. For each selected target, Regeneron is required to use commercially reasonable efforts to submit regulatory filings necessary to achieve initial IND acceptance for at least one product directed to each applicable target, and following IND acceptance for at least one product, to develop and commercialize such product. In connection with this collaboration, Regeneron agreed to purchase $50.0 million of the Company’s common stock in a private placement concurrent with the Company’s IPO, and the Company received a nonrefundable upfront payment of $75.0 million. In addition, the Company is eligible to earn, on a per-licensed target basis, (i) up to $25.0 million in development milestones, including for the dosing of the first patient in each of Phase I, Phase II and Phase III clinical trials, (ii) up to $110.0 million in regulatory milestones, including for the acceptance of a regulatory filing in the U.S. and U.S. and ex-U.S. regulatory approvals, and (iii) up to $185.0 million in sales-based milestone payments. The Company is also eligible to earn royalties ranging from the high single digits to low teens, in each case, on a per-product basis, which royalties are potentially subject to various reductions and offsets and are further subject to the Company’s existing low single-digit royalty obligations under the Caribou license agreement. The collaboration term ends in April 2022, provided that Regeneron may make a one-time payment of $25.0 million to extend the term for an additional two-year period. The agreement will continue until the date when no royalty or other payment obligations are due, unless earlier terminated in accordance with the terms of the agreement. Regeneron’s royalty payment obligations expire on a country-by-country and product-by-product basis upon the later of (i) the expiration of the last valid claim of the royalty-bearing patents covering such product in such country, (ii) 12 years from the first commercial sale of such product in such country, or (iii) the expiration of regulatory exclusivity for such product. The Company may terminate the agreement on a target-by-target basis if Regeneron or any of its affiliates institutes a patent challenge against our CRISPR/Cas or certain other background patent rights. The Company may also terminate the agreement on a target-by-target basis if Regeneron does not proceed with the development of a product directed to a selected target within specified periods of time. Regeneron may terminate the agreement, without cause, upon 180 days written notice to us, either in its entirety or on a target-by-target basis, in which event, certain rights in the terminated targets and associated intellectual property revert to us, as described in the agreement. Following such termination, the Company may owe Regeneron royalties, in certain circumstances, up to mid-single digits on any terminated targets that we subsequently commercialize on a product-by-product basis for a period of 12 years after the first commercial sale of any such products. Either party may terminate the agreement either in its entirety or with respect to the technology collaboration or one or more of the targets selected by Regeneron, in the event of the other party’s uncured material breach.

Summary of Significant Accoun17

Summary of Significant Accounting Policies (Policies)	3 Months Ended
	Mar. 31, 2016
Accounting Policies [Abstract]
Recent Accounting Pronouncements	Recent Accounting Pronouncements In May 2014, the Financial Accounting Standards Board (“FASB”) issued Accounting Standards Update (“ASU”) No. 2014-09, Revenue from Contracts with Customers (Topic 606), Revenue From Contracts With Customers: Identifying Performance Obligations and Licensing In August 2014, the FASB issued ASU No. 2014-15, Disclosure of Uncertainties About an Entity’s Ability to Continue as a Going Concern Presentation of Financial Statements—Going Concern In April 2015, the FASB issued ASU No. 2015-05, Customer’s Accounting for Fees Paid in a Cloud Computing Arrangement Internal-Use Software In February 2016, the FASB issued ASU 2016-02, Leases Leases In March 2016, the FASB issued ASU, 2016-09, Improvements to Employee Share-Based Payment Accounting Compensation – Stock Compensation

Fair Value Measurements (Tables

Fair Value Measurements (Tables)	3 Months Ended
	Mar. 31, 2016
Fair Value Disclosures [Abstract]
Summary of Financial Assets Recognized at Fair Value	As of March 31, 2016 and December 31, 2015, the Company’s financial assets recognized at fair value consisted of the following: Fair Value as of March 31, 2016 Total Level 1 Level 2 Level 3 (In thousands) Cash equivalents $ 30,005 $ 30,005 $ — $ — Total $ 30,005 $ 30,005 $ — $ — Fair Value as of December 31, 2015 Total Level 1 Level 2 Level 3 (In thousands) Cash equivalents $ 30,000 $ 30,000 $ — $ — Total $ 30,000 $ 30,000 $ — $ —

Accrued Expenses (Tables)

Accrued Expenses (Tables)	3 Months Ended
	Mar. 31, 2016
Payables and Accruals [Abstract]
Schedule of Accrued Expenses	Accrued expenses consisted of the following: March 31, 2016 December 31, (In thousands) Employee compensation and benefits $ 625 $ 1,281 In-process research and development obligation 400 600 Research and development and professional expenses 1,948 907 Accrued expenses $ 2,973 $ 2,788

Commitments and Contingencies (

Commitments and Contingencies (Tables)	3 Months Ended
	Mar. 31, 2016
Commitments and Contingencies Disclosure [Abstract]
Schedule of Future Minimum Lease Payments	Future minimum lease payments under this lease by year as of March 31, 2016 are as follows: (In thousands) Future minimum lease payments in year ending December 31: 2016 $ 370 2017 4,464 2018 4,598 2019 4,736 2020 4,878 Thereafter 9,786 Total future minimum lease payments $ 28,832

Equity-Based Compensation (Tabl

Equity-Based Compensation (Tables)	3 Months Ended
	Mar. 31, 2016
Schedule of Equity-Based Compensation Expense	Equity-based compensation expense is classified in the consolidated statements of operations as follows: Three Months Ended March 31, 2016 2015 (In thousands) Research and development $ 454 $ 56 General and administrative 358 59 Total $ 812 $ 115
Summary of Weighted Average Assumptions Used to Compute Fair Value of Option Granted	Key assumptions used to apply this pricing model were as follows: Three Months Ended Risk-free interest rate 1.3 % Expected life of options 6.0 years Expected volatility of underlying stock 87.7 % Expected dividend yield 0.0 %
Summary of Stock Option Activity	The following is a summary of stock option activity for the three months ended March 31, 2016: Number of Weighted Average Exercise Price per Weighted Average Remaining Contractual Term Aggregate Intrinsic Value (In years) (In thousands) Outstanding at January 1, 2016 456,374 $ 6.04 Granted 2,161,558 6.83 Outstanding at March 31, 2016 2,617,932 $ 6.69 9.9 $ 212 Exercisable at March 31, 2016 — $ — — $ —
Founder Stock [Member]
Summary of Stock Activity	The following table summarizes the Company’s compensatory Founder Stock activity for the three months ended March 31, 2016: Number of Weighted Unvested Founder Stock as of January 1, 2016 436,509 $ 0.37 Vested (42,244 ) 0.37 Unvested Founder Stock as of March 31, 2016 394,265 $ 0.37
Restricted Stock [Member]
Summary of Stock Activity	The following table summarizes the Company’s restricted stock activity for the three months ended March 31, 2016: Number of Weighted Unvested restricted stock as of January 1, 2016 1,945,036 $ 0.78 Vested (170,807 ) 0.73 Unvested restricted stock as of March 31, 2016 1,774,229 $ 0.81

Loss Per Share (Tables)

Loss Per Share (Tables)	3 Months Ended
	Mar. 31, 2016
Earnings Per Share [Abstract]
Schedule of Basic and Diluted Net Loss Per Share	Basic and diluted loss per common unit was calculated as follows: Three Months Ended (In thousands except Net loss $ (1,093 ) Weighted average common units outstanding, basic and diluted 1,284 Net loss per common unit, basic and diluted $ (0.85 ) Following the Reorganization, the Company calculates loss per share attributable to common stockholders based on its outstanding common stock. Basic and diluted loss per share attributable to common stockholders was calculated as follows: Three Months Ended (In thousands except Net loss $ (6,689 ) Weighted average shares outstanding, basic and diluted 676 Net loss per share attributable to common stockholders, basic and diluted $ (9.89 )
Potential Dilutive Securities Excluded from Computation of Diluted Net Loss Per Common Share	The following common stock equivalents were excluded from the calculation of diluted loss per unit or share because their inclusion would have been anti-dilutive: Three Months Ended March 31, 2016 2015 (In thousands) Preferred units — 20,682 Unvested common units — 1,014 Unvested incentive units — 1,479 Unvested restricted stock 1,774 — Stock options 2,618 — 4,392 23,175

Overview and Basis of Present23

Overview and Basis of Presentation - Additional Information (Detail) - Subsequent Event [Member] $ / shares in Units, $ in Millions	May. 11, 2016USD ($)$ / sharesshares	Apr. 25, 2016	May. 31, 2016shares
IPO [Member]
Organization Consolidation And Presentation Of Financial Statements Disclosure [Line Items]
Number of common stock issued upon conversion of shares	6,900,000		6,900,000
Common stock price per share | $ / shares	$ 18
Proceeds from issuance initial public offering | $	$ 115.5
Reverse stock split ratio of common stock		0.588
Reverse stock split description		One-for-1.7 reverse stock split
Effective date of reverse stock split		Apr. 25, 2016
Convertible preferred stock conversion ratio	One-for-0.6465903
Conversion of convertible preferred stock into common stock	23,481,956		23,481,956
Private Placement [Member]
Organization Consolidation And Presentation Of Financial Statements Disclosure [Line Items]
Number of common stock issued upon conversion of shares	3,055,554		3,055,554
Number of common stock issued upon conversion of value | $	$ 55

Fair Value Measurements - Summa

Fair Value Measurements - Summary of Financial Assets Recognized at Fair Value (Detail) - USD ($) $ in Thousands	Mar. 31, 2016	Dec. 31, 2015
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash equivalents	$ 30,005	$ 30,000
Total	30,005	30,000
Level 1 [Member]
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items]
Cash equivalents	30,005	30,000
Total	$ 30,005	$ 30,000

Accrued Expenses - Schedule of

Accrued Expenses - Schedule of Accrued Expenses (Detail) - USD ($) $ in Thousands	Mar. 31, 2016	Dec. 31, 2015
Payables and Accruals [Abstract]
Employee compensation and benefits	$ 625	$ 1,281
In-process research and development obligation	400	600
Research and development and professional expenses	1,948	907
Accrued expenses	$ 2,973	$ 2,788

Income Taxes - Additional Infor

Income Taxes - Additional Information (Detail) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2016	Mar. 31, 2015
Income Tax Disclosure [Abstract]
Income tax provision	$ 0	$ (102)
Consideration received under collaboration agreement		2,600
Income tax provision within equity		1,000
Accrual for intraperiod tax allocation		$ 900

Commitments and Contingencies -

Commitments and Contingencies - Additional Information (Detail) - Cambridge, Massachusetts [Member] $ in Millions	3 Months Ended
	Mar. 31, 2016USD ($)
Operating Leased Assets [Line Items]
Operating lease arrangement term	10 years
Number of lease years before optional extension and early termination can be exercised	6 years
Capital lease obligation additional period of term of leases in years	3 years
Operating lease security deposit	$ 2.2

Commitments and Contingencies28

Commitments and Contingencies - Schedule of Future Minimum Lease Payments (Detail) $ in Thousands	Mar. 31, 2016USD ($)
Operating Leases, Future Minimum Payments Due, Fiscal Year Maturity [Abstract]
2,016	$ 370
2,017	4,464
2,018	4,598
2,019	4,736
2,020	4,878
Thereafter	9,786
Total future minimum lease payments	$ 28,832

Collaboration - Additional Info

Collaboration - Additional Information (Detail) - USD ($)	1 Months Ended	3 Months Ended		16 Months Ended
	Jan. 31, 2015	Mar. 31, 2016	Mar. 31, 2015	Mar. 31, 2016	Dec. 31, 2015
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items]
Difference between cash proceeds received and estimated fair value of preferred units			$ 2,600,000
Collaboration revenue		$ 1,777,000	1,286,000
Novartis [Member] | Class A Preferred Units [Member]
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items]
Proceeds from issuance of preferred stock	$ 9,000,000
Estimated fair value of units	11,600,000
Difference between cash proceeds received and estimated fair value of preferred units	2,600,000
Novartis [Member] | Maximum [Member]
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items]
Development based milestone payments under agreement	30,300,000
Regulatory based milestone payments for first indication	50,000,000
Regulatory based milestone payments for second indication	50,000,000
Sales based milestone payments under agreement	100,000,000
Novartis [Member] | Collaborative Arrangement [Member]
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items]
Collaboration revenue				$ 17,400,000
Technology access fees	20,000,000
Quarterly research payments	$ 1,000,000
Research term	5 years
Collaboration revenue		1,800,000	$ 1,300,000	7,900,000
Deferred revenue		$ 9,500,000		$ 9,500,000	$ 10,300,000
Novartis [Member] | Collaborative Arrangement [Member] | Upfront Technology Access Payment [Member]
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items]
Collaboration revenue	$ 10,000,000
Novartis [Member] | Collaborative Arrangement [Member] | Maximum [Member]
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items]
Total research funding	$ 20,000,000

Equity-Based Compensation - Sch

Equity-Based Compensation - Schedule of Equity-Based Compensation Expense (Detail) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2016	Mar. 31, 2015
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Equity-based compensation expense	$ 812	$ 115
Research and Development [Member]
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Equity-based compensation expense	454	56
General and Administrative [Member]
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Equity-based compensation expense	$ 358	$ 59

Equity-Based Compensation - Sum

Equity-Based Compensation - Summary of Stock Activity (Detail)	3 Months Ended
	Mar. 31, 2016$ / sharesshares
Founder Stock [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Number of Shares, Unvested, Beginning balance | shares	436,509
Number of Shares, Vested | shares	(42,244)
Number of Shares, Unvested, Ending balance | shares	394,265
Weighted Average Grant Date Fair Value per Share, Unvested, Beginning balance | $ / shares	$ 0.37
Weighted Average Grant Date Fair Value per Share, Vested | $ / shares	0.37
Weighted Average Grant Date Fair Value per Share, Unvested, Ending balance | $ / shares	$ 0.37
Restricted Stock [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Number of Shares, Unvested, Beginning balance | shares	1,945,036
Number of Shares, Vested | shares	(170,807)
Number of Shares, Unvested, Ending balance | shares	1,774,229
Weighted Average Grant Date Fair Value per Share, Unvested, Beginning balance | $ / shares	$ 0.78
Weighted Average Grant Date Fair Value per Share, Vested | $ / shares	0.73
Weighted Average Grant Date Fair Value per Share, Unvested, Ending balance | $ / shares	$ 0.81

Equity-Based Compensation - Add

Equity-Based Compensation - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Millions	3 Months Ended
	Mar. 31, 2016	Mar. 31, 2015
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Total unrecognized stock-based compensation expense	$ 3.3
Weighted average grant date fair value per share	$ 4.98
Stock option awards granted	2,161,558	0
Unrecognized compensation cost related to stock options	$ 11.8
Founder Stock and Restricted Stock [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Weighted average period of unrecognized compensation costs	2 years 8 months 12 days
Stock Options [Member]
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Weighted average period of unrecognized compensation costs	3 years 9 months 18 days

Equity-Based Compensation - S33

Equity-Based Compensation - Summary of Weighted Average Assumptions Used to Compute Fair Value of Option Granted (Detail)	3 Months Ended
	Mar. 31, 2016
Disclosure of Compensation Related Costs, Share-based Payments [Abstract]
Risk-free interest rate	1.30%
Expected life of options	6 years
Expected volatility of underlying stock	87.70%
Expected dividend yield	0.00%

Equity-Based Compensation - S34

Equity-Based Compensation - Summary of Stock Option Activity (Detail) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended
	Mar. 31, 2016	Mar. 31, 2015
Disclosure of Compensation Related Costs, Share-based Payments [Abstract]
Number of Options, Outstanding, Beginning Balance	456,374
Number of options, Granted	2,161,558	0
Number of Options, Outstanding, Ending Balance	2,617,932
Number of Options, Exercisable	0
Weighted Average Exercise Price per Share, Outstanding, Beginning Balance	$ 6.04
Weighted Average Exercise Price per Share, Granted	6.83
Weighted Average Exercise Price per Share, Outstanding, Ending Balance	6.69
Weighted Average Exercise Price per Share, Exercisable	$ 0
Weighted Average Remaining Contractual Term, Outstanding	9 years 10 months 24 days
Aggregate Intrinsic Value, Outstanding	$ 212

Loss Per Share - Schedule of Ba

Loss Per Share - Schedule of Basic and Diluted Net Loss Per Share (Detail) - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands	3 Months Ended
	Mar. 31, 2016	Mar. 31, 2015
Earnings Per Share [Abstract]
Net loss	$ (6,689)	$ (1,093)
Weighted average common units outstanding, basic and diluted		1,284
Weighted average shares outstanding, basic and diluted	676
Net loss per common unit, basic and diluted		$ (0.85)
Net loss per share attributable to common stockholders, basic and diluted	$ (9.89)

Loss Per Share - Additional Inf

Loss Per Share - Additional Information (Detail) - Subsequent Event [Member] - shares	May. 11, 2016	May. 31, 2016
IPO [Member]
Earnings Loss Per Share [Line Items]
Number of common stock issued upon conversion of shares	6,900,000	6,900,000
Conversion of convertible preferred stock into common stock	23,481,956	23,481,956
Private Placement [Member]
Earnings Loss Per Share [Line Items]
Number of common stock issued upon conversion of shares	3,055,554	3,055,554

Loss Per Share - Potential Dilu

Loss Per Share - Potential Dilutive Securities Excluded from Computation of Diluted Net Loss Per Common Share (Detail) - shares shares in Thousands	3 Months Ended
	Mar. 31, 2016	Mar. 31, 2015
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Potential dilutive securities excluded from computation of diluted net loss per common share	4,392	23,175
Preferred Units [Member]
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Potential dilutive securities excluded from computation of diluted net loss per common share		20,682
Unvested Common Units [Member]
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Potential dilutive securities excluded from computation of diluted net loss per common share		1,014
Unvested Incentive Units [Member]
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Potential dilutive securities excluded from computation of diluted net loss per common share		1,479
Unvested Restricted Stock [Member]
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Potential dilutive securities excluded from computation of diluted net loss per common share	1,774
Stock Options [Member]
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Potential dilutive securities excluded from computation of diluted net loss per common share	2,618

Related Party Transactions - Ad

Related Party Transactions - Additional Information (Detail) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2016	Mar. 31, 2015	Dec. 31, 2015	Aug. 31, 2015	Jul. 31, 2014
Related Party Transaction [Line Items]
Research and development expense	$ 5,225	$ 1,371
General and administrative expenses	$ 3,246	1,110
Caribou [Member]
Related Party Transaction [Line Items]
Junior preferred units issued					8,110,599
Percentage of shares owned	19.20%
Research and development expense	$ 400	400
Current obligations related to license and service agreements with related party	400		$ 600
General and administrative expenses	$ 200	$ 200
Percentage of related party obligation	30.00%	30.00%
Novartis [Member] | Collaborative Arrangement [Member]
Related Party Transaction [Line Items]
Percentage of shares owned	18.20%
Collaboration revenue	$ 1,800	$ 1,300
Accounts receivable	1,000		1,000
Deferred revenue	$ 9,500		$ 10,300
Novartis [Member] | Class A-1 Preferred Units [Member] | Collaborative Arrangement [Member]
Related Party Transaction [Line Items]
Junior preferred units issued	4,761,905
Novartis [Member] | Class A-2 Preferred Units [Member] | Collaborative Arrangement [Member]
Related Party Transaction [Line Items]
Junior preferred units issued	2,666,666
Novartis [Member] | Series B Preferred Stock [Member] | Collaborative Arrangement [Member]
Related Party Transaction [Line Items]
Preferred stock, shares acquired				761,905

Subsequent Events - Additional

Subsequent Events - Additional Information (Detail) - Collaborative Arrangement [Member] - Regeneron Pharmaceuticals Inc. [Member] - Subsequent Event [Member] $ in Millions	1 Months Ended
	Apr. 30, 2016USD ($)
Subsequent Event [Line Items]
Purchase of common stock through private placement	$ 50
Upfront payment received	75
One time collaboration payment	$ 25
Collaboration term extension period	2 years
Royalty payment obligation expiration period	12 years
Termination period of agreement	180 days
Maximum [Member]
Subsequent Event [Line Items]
Development based milestone payments under agreement	$ 25
Regulatory based milestone payments under agreement	110
Sales based milestone payments under agreement	$ 185