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Significant Contracts - Additional Information (Detail)
1 Months Ended 3 Months Ended
Dec. 13, 2019
USD ($)
Product
Oct. 26, 2015
USD ($)
Oct. 31, 2019
License
Mar. 31, 2021
USD ($)
Option
Mar. 31, 2020
USD ($)
Dec. 31, 2019
USD ($)
Dec. 31, 2020
USD ($)
Apr. 30, 2020
USD ($)
Research and Development Arrangement, Contract to Perform for Others [Line Items]                
Non-current deferred revenue       $ 11,776,000     $ 11,776,000  
Collaboration revenue       539,000 $ 157,000      
Current deferred revenue       1,802,000     2,341,000  
Research and development expense       $ 90,565,000 54,193,000      
Date of joint venture agreement       Dec. 19, 2015        
Operating expenses       $ 115,082,000 73,743,000      
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%        
Business combination, consideration transferred       $ 41,000,000.0        
Net assets acquired       16,000,000.0        
Fair value of controlling interest obtained       32,000,000.0        
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 maximum 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       $ 4,300,000 1,900,000      
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%        
Number of products | Product 2              
Non-refundable one-time option payment $ 20,000,000.0              
Business combination, consideration transferred       $ 25,000,000.0        
Payment to acquire business       16,000,000.0        
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        
2019 Option Agreement [Member] | Research And Development Services | Casebia Therapeutics Limited Liability Partnership [Member]                
Research and Development Arrangement, Contract to Perform for Others [Line Items]                
Fair value of obligations       20,200,000        
2019 Option Agreement [Member] | Future Delivery of up to Two Options [Member] | Casebia Therapeutics Limited Liability Partnership [Member]                
Research and Development Arrangement, Contract to Perform for Others [Line Items]                
Fair value of obligations       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       2,700,000     7,000,000.0  
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        
2019 Casebia Agreement [Member] | Casebia Therapeutics Limited Liability Partnership [Member]                
Research and Development Arrangement, Contract to Perform for Others [Line Items]                
Fair value of assets and liabilities       16,000,000.0        
Equity method investment       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        
Transaction price       268,600,000        
Non-current deferred revenue       11,800,000     11,800,000  
Non-cash consideration received       10,000,000.0        
Transaction price allocated to remaining performance obligations       11,800,000        
Collaboration revenue       0 0      
Current deferred revenue       200,000     $ 400,000  
Maximum potential future payments       800,000,000.0        
Research and development expense       19,900,000 9,100,000      
Reimbursements from research and license agreements       10,600,000 $ 5,500,000      
Collaborative arrangement, license rights reacquired by waiving payment owed       10,000,000.0        
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        
Milestone payment receivable       410,000,000.0        
Transaction price allocated to remaining performance obligations       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        
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        
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        
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | Specified Target Option Material Right [Member]                
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        
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        
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        
Percentage of exchange payment of research and development costs       50.00%        
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        
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        
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 [Member]                
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        
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