Significant Contracts - Additional Information (Detail) | Dec. 13, 2019USD ($)Product | Oct. 26, 2015USD ($) | Oct. 31, 2019License | Sep. 30, 2020USD ($) | Jun. 30, 2020USD ($) | Mar. 31, 2020USD ($) | Dec. 31, 2019USD ($) | Sep. 30, 2019USD ($) | Jun. 30, 2019USD ($) | Mar. 31, 2019USD ($) | Sep. 30, 2020USD ($)Option | Sep. 30, 2019USD ($) | Dec. 31, 2016USD ($) | Apr. 30, 2020USD ($) | Dec. 12, 2019 |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Non-current deferred revenue | | | | | $ 11,776,000 | | | $ 11,776,000 | | | | $ 11,776,000 | | | | |
Collaboration revenue | [1] | | | | 148,000 | | | | $ 211,928,000 | | | 349,000 | $ 212,574,000 | | | |
Current deferred revenue | | | | | 610,000 | | | 960,000 | | | | 610,000 | | | | |
Research and development expense | [2] | | | | 71,008,000 | | | | 57,246,000 | | | $ 184,581,000 | 130,601,000 | | | |
Date of joint venture agreement | | | | | | | | | | | | Dec. 19, 2015 | | | | |
Operating expenses | | | | | 92,547,000 | | | | 72,765,000 | | | $ 247,023,000 | 176,817,000 | | | |
Equity method investment | | | | | | | | | | | | | | $ 36,500,000 | | |
Unrealized equity method losses | | | | | | | | | 3,430,000 | | | | 5,467,000 | $ 36,500,000 | | |
Net loss of joint venture | | | | | (92,439,000) | $ (79,656,000) | $ (69,731,000) | | 138,423,000 | $ (53,699,000) | $ (48,408,000) | (241,826,000) | 36,316,000 | | | |
Casebia Therapeutics Limited Liability Partnership [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Research and development expense | | | | | | | | | 100,000 | | | | 700,000 | | | |
Equity method investment, ownership percentage | | | | | | | | | | | | | | | | 50.00% |
Unrealized equity method losses | | | | | | | | | 3,300,000 | | | | 5,500,000 | | | |
Net loss of joint venture | | | | | | | | | 22,600,000 | | | | 54,900,000 | | | |
Unrecognized equity method losses in excess of Company's interest | | | | | | | | | | | | | 70,100,000 | | | |
Business combination, consideration transferred | | | | | | | | | | | | 41,000,000 | | | | |
Net assets acquired | | | | | $ 16,000,000 | | | | | | | 16,000,000 | | | | |
Fair value of controlling interest obtained | | | | | | | | | | | | $ 32,000,000 | | | | |
Casebia Therapeutics Limited Liability Partnership [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Date of formation of joint venture entity | | | | | | | | | | | | Feb. 12, 2016 | | | | |
Equity method investment, ownership percentage | | | | | 50.00% | | | | | | | 50.00% | | | | |
License and Service [Member] | Casebia Therapeutics Limited Liability Partnership [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Collaboration revenue | | | | | | | | | 100,000 | | | | 500,000 | | | |
Non-Exclusive Research License [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Transaction price allocated to remaining performance obligations | | | $ 500,000 | | | | | | | | | | | | | |
Non-Exclusive Research License [Member] | Valuation, Market Approach | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Estimated standalone selling price for collaboration agreement | | | 1,000,000 | | | | | | | | | | | | | |
Retirement Agreement [Member] | Casebia Therapeutics Limited Liability Partnership [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Percentage of partnership interests | | 100.00% | | | | | | | | | | | | | | |
Partnership outstanding exchange amount | | $ 22,000,000 | | | | | | | | | | | | | | |
Operating expenses | | $ 6,000,000 | | | | | | | | | | | | | | |
2019 Option Agreement [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Research and development expense | | | | | $ 4,400,000 | | | | | | | $ 8,600,000 | | | | |
2019 Option Agreement [Member] | Casebia Therapeutics Limited Liability Partnership [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Number of options exercised under agreement | Option | | | | | | | | | | | | 2 | | | | |
Equity method investment, ownership percentage | | | | | 50.00% | | | | | | | 50.00% | | | | |
Business combination, consideration transferred | | | | | | | | | | | | $ 25,000,000 | | | | |
Payment to acquire business | | | | | | | | | | | | 16,000,000 | | | | |
2019 Option Agreement [Member] | Casebia Therapeutics Limited Liability Partnership [Member] | Research And Development Services | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Fair value of obligations | | | | | $ 20,200,000 | | | | | | | 20,200,000 | | | | |
2019 Option Agreement [Member] | Casebia Therapeutics Limited Liability Partnership [Member] | Future Delivery of up to Two Options [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Fair value of obligations | | | | | 4,800,000 | | | | | | | 4,800,000 | | | | |
2019 Option Agreement [Member] | Casebia Therapeutics Limited Liability Partnership [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Number of products | Product | | 2 | | | | | | | | | | | | | | |
Non-refundable one-time option payment | | $ 20,000,000 | | | | | | | | | | | | | | |
2019 Option Agreement [Member] | Research and Development [Member] | Casebia Therapeutics Limited Liability Partnership [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Business combination, consideration transferred | | | | | | | | | | | | 20,200,000 | | | | |
2019 Option Agreement [Member] | Certain Options [Member] | Casebia Therapeutics Limited Liability Partnership [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Business combination, consideration transferred | | | | | | | | | | | | 4,800,000 | | | | |
Co-commercialization Agreement [Member] | Casebia Therapeutics Limited Liability Partnership [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Percentage of research and development costs | | 50.00% | | | | | | | | | | | | | | |
Percentage sharing of profit (loss) from sale of product | | 50.00% | | | | | | | | | | | | | | |
2019 Casebia Agreement [Member] | Other Current Liabilities [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Research obligation | | | | | 11,600,000 | | | | | | | 11,600,000 | | | | |
2019 Casebia Agreement [Member] | Other Long-term Liabilities [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Research obligation | | | | | 4,800,000 | | | | | | | 4,800,000 | | | | |
2019 Casebia Agreement [Member] | Casebia Therapeutics Limited Liability Partnership [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Equity method investment | | | | | 0 | | | | | | | 0 | | | | |
Fair value of assets and liabilities | | | | | 16,000,000 | | | | | | | 16,000,000 | | | | |
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Nonrefundable upfront payment received | | | 75,000,000 | | | | | | | | | 175,000,000 | | | | |
Milestone payment receivable | | | | | 410,000,000 | | | | | | | 410,000,000 | | | | |
Transaction price | | | | | | | | | | | | 268,600,000 | | | | |
Non-current deferred revenue | | | | | 11,800,000 | | | 11,800,000 | | | | 11,800,000 | | | | |
Non-cash consideration received | | | | | | | | | | | | 10,000,000 | | | | |
Transaction price allocated to remaining performance obligations | | | | | 12,000,000 | | | | | | | 12,000,000 | | | | |
Collaboration revenue | | | | | 0 | | | | 211,900,000 | | | 0 | 212,100,000 | | | |
Current deferred revenue | | | | | 600,000 | | | 900,000 | | | | 600,000 | | | | |
Maximum potential future payments | | | | | | | | | | | | 800,000,000 | | | | |
Contract asset | | | | | 25,000,000 | | | | | | | 25,000,000 | | | | |
Research and development expense | | | | | 12,500,000 | | | | 7,500,000 | | | 31,500,000 | 21,300,000 | | | |
Reimbursements from research and license agreements | | | | | 6,800,000 | | | | $ 3,800,000 | | | 17,800,000 | $ 11,800,000 | | | |
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | Research and Development [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Variable consideration received | | | 19,300,000 | | | | | | | | | 25,000,000 | | | | |
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | Collaboration Target Options [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Milestone payment receivable | | | | | 420,000,000 | | | | | | | 420,000,000 | | | | |
Transaction price allocated to remaining performance obligations | | | | | 46,700,000 | | | | | | | 46,700,000 | | | | |
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | DM1 R&D Services [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Estimated standalone selling price for single collaboration | | | | | | | | | | | | 1,700,000 | | | | |
Transaction price allocated to remaining performance obligations | | | | | 1,100,000 | | | | | | | 1,100,000 | | | | |
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | DM1 R&D Services [Member] | Research and Development [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Variable consideration received | | | | | | | | | | | | 800,000 | | | | |
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | DMD License [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Estimated standalone selling price for single collaboration | | | | | | | | | | | | 224,600,000 | | | | |
Transaction price allocated to remaining performance obligations | | | | | 151,100,000 | | | | | | | 151,100,000 | | | | |
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | DM1 License [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Estimated standalone selling price for single collaboration | | | | | | | | | | | | 76,200,000 | | | | |
Transaction price allocated to remaining performance obligations | | | | | 51,300,000 | | | | | | | 51,300,000 | | | | |
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | Specified Target Option Material Right | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Estimated standalone selling price for single collaboration | | | | | | | | | | | | 17,500,000 | | | | |
Transaction price allocated to remaining performance obligations | | | | | 11,800,000 | | | | | | | 11,800,000 | | | | |
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | Collaboration Target Options Material Rights One [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Estimated standalone selling price for single collaboration | | | | | | | | | | | | 25,000,000 | | | | |
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | Collaboration Target Options Material Rights Two [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Estimated standalone selling price for single collaboration | | | | | | | | | | | | 22,200,000 | | | | |
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | Collaboration Target Options Material Rights Three [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Estimated standalone selling price for single collaboration | | | | | | | | | | | | 22,200,000 | | | | |
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | Joint Development Agreement [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Up-front payment received | | | 7,000,000 | | | | | | | | | $ 7,000,000 | | | | |
Agreement description | | | | | | | | | | | | In connection with the JDA, the Company received a $7.0 million up-front payment from Vertex and subsequently received a one-time low seven-digit milestone payment upon the dosing of the second patient in a clinical trial with the initial product candidate. | | | | |
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | Exercise of Exclusive Option [Member] | Collaboration Target Options [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Milestone payment receivable | | | | | 10,000,000 | | | | | | | $ 10,000,000 | | | | |
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | 2019 Collaboration Agreement [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Nonrefundable upfront payment received | | | | | | | | | | | | 175,000,000 | | | | |
Maximum potential payments | | | | | 825,000,000 | | | | | | | $ 825,000,000 | | | | |
Percentage of exchange payment of research and development costs | | | | | | | | | | | | 50.00% | | | | |
Collaborative arrangement, license rights reacquired by waiving payment owed | | | | | | | | | | | | $ 10,000,000 | | | | |
Non-cash consideration expensed upon execution of collaborative agreement | | | | | | | | | | | | 10,000,000 | | | | |
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | 2015 Collaboration Agreement [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Milestone payment receivable | | | | | 395,000,000 | | | | | | | $ 395,000,000 | | | | |
Percentage of exchange payment of research and development costs | | | | | | | | | | | | 50.00% | | | | |
Number of options would not be exercised under agreement | Option | | | | | | | | | | | | 1 | | | | |
Number of remaining options under agreement | Option | | | | | | | | | | | | 4 | | | | |
Non-current deferred revenue | | | | | 57,800,000 | | | | | | | $ 57,800,000 | | | | |
Number of options exercised under agreement | Option | | | | | | | | | | | | 4 | | | | |
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | 2015 Collaboration Agreement [Member] | Collaborative Arrangement Material Rights Fourth Exclusive License | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Estimated standalone selling price for single collaboration | | | | | | | | | | | | $ 10,000,000 | | | | |
Transaction price allocated to remaining performance obligations | | | | | $ 6,700,000 | | | | | | | $ 6,700,000 | | | | |
Vertex Pharmaceuticals Inc [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Estimated standalone selling price for single collaboration | | | 19,300,000 | | | | | | | | | | | | | |
Transaction price allocated to remaining performance obligations | | | 19,300,000 | | | | | | | | | | | | | |
Estimated standalone selling price for collaboration agreement | | | 118,600,000 | | | | | | | | | | | | | |
Vertex Pharmaceuticals Inc [Member] | Collaboration Target Options [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Transaction price allocated to remaining performance obligations | | | 57,700,000 | | | | | | | | | | | | | |
Vertex Pharmaceuticals Inc [Member] | Collaboration Target Options Material Rights One [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Estimated standalone selling price for single collaboration | | | 45,600,000 | | | | | | | | | | | | | |
Vertex Pharmaceuticals Inc [Member] | Collaboration Target Options Material Rights Two [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Estimated standalone selling price for single collaboration | | | 38,400,000 | | | | | | | | | | | | | |
Vertex Pharmaceuticals Inc [Member] | Collaboration Target Options Material Rights Three [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Estimated standalone selling price for single collaboration | | | 17,300,000 | | | | | | | | | | | | | |
Vertex Pharmaceuticals Inc [Member] | Rights Four [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Estimated standalone selling price for single collaboration | | | 17,300,000 | | | | | | | | | | | | | |
Vertex Pharmaceuticals Inc [Member] | 2019 Collaboration Agreement [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Milestone payment receivable | | | | | | | | | | | | | | | $ 25,000,000 | |
Vertex Pharmaceuticals Inc [Member] | 2015 Collaboration Agreement [Member] | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Aggregate amount in option exercise payments received | | | | | | | | $ 30,000,000 | | | | | | | | |
Number of exclusive license targets | License | | | | 3 | | | | | | | | | | | | |
Vertex Pharmaceuticals Inc [Member] | Beta Globin | | | | | | | | | | | | | | | | |
Research and Development Arrangement, Contract to Perform for Others [Line Items] | | | | | | | | | | | | | | | | |
Estimated standalone selling price for single collaboration | | | 48,900,000 | | | | | | | | | | | | | |
Transaction price allocated to remaining performance obligations | | | $ 23,800,000 | | | | | | | | | | | | | |
| |
[1] | Including the following revenue from a related party, see Notes 7 & 12 | |
[2] | Including the following research and development expense with a related party, see Notes 7 & 12 | |