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Significant Contracts - Additional Information (Detail)
 1 Months Ended 3 Months Ended 9 Months Ended 12 Months Ended
Dec. 13, 2019
USD ($)
Product
Oct. 26, 2015
USD ($)
Oct. 31, 2019
License
Sep. 30, 2020
USD ($)
Jun. 30, 2020
USD ($)
Mar. 31, 2020
USD ($)
Dec. 31, 2019
USD ($)
Sep. 30, 2019
USD ($)
Jun. 30, 2019
USD ($)
Mar. 31, 2019
USD ($)
Sep. 30, 2020
USD ($)
Option
Sep. 30, 2019
USD ($)
Dec. 31, 2016
USD ($)
Apr. 30, 2020
USD ($)
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                     41,000,000.0        
Net assets acquired       $ 16,000,000.0             16,000,000.0        
Fair value of controlling interest obtained                     $ 32,000,000.0        
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   1,000,000.0                          
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 $ 22,000,000.0                            
Operating expenses $ 6,000,000.0                            
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                     $ 25,000,000.0        
Payment to acquire business                     16,000,000.0        
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 $ 20,000,000.0                            
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       16,000,000.0             16,000,000.0        
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member]                              
Research and Development Arrangement, Contract to Perform for Others [Line Items]                              
Nonrefundable upfront payment received   75,000,000.0                 175,000,000.0        
Milestone payment receivable       410,000,000.0             410,000,000.0        
Transaction price                     268,600,000        
Non-current deferred revenue       11,800,000     11,800,000       11,800,000        
Non-cash consideration received                     10,000,000.0        
Transaction price allocated to remaining performance obligations       12,000,000.0             12,000,000.0        
Collaboration revenue       0       211,900,000     0 212,100,000      
Current deferred revenue       600,000     900,000       600,000        
Maximum potential future payments                     800,000,000.0        
Contract asset       25,000,000.0             25,000,000.0        
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                 25,000,000.0        
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       420,000,000.0             420,000,000.0        
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                     25,000,000.0        
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   7,000,000.0                 $ 7,000,000.0        
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                     175,000,000.0        
Maximum potential payments       825,000,000.0             $ 825,000,000.0        
Percentage of exchange payment of research and development costs                     50.00%        
Collaborative arrangement, license rights reacquired by waiving payment owed                     $ 10,000,000.0        
Non-cash consideration expensed upon execution of collaborative agreement                     10,000,000.0        
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       395,000,000.0             $ 395,000,000.0        
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                     $ 10,000,000.0        
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                           $ 25,000,000.0  
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             $ 30,000,000.0                
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