XML 91 R57.htm IDEA: XBRL DOCUMENT v3.20.4
Significant Contracts - Additional Information (Detail)
1 Months Ended 2 Months Ended 3 Months Ended 12 Months Ended
Dec. 13, 2019
USD ($)
Product
Dec. 18, 2018
USD ($)
Nov. 15, 2018
USD ($)
shares
Sep. 24, 2018
USD ($)
shares
Sep. 17, 2018
USD ($)
Installment
Oct. 26, 2015
USD ($)
Dec. 31, 2019
USD ($)
Oct. 31, 2019
License
Dec. 18, 2018
USD ($)
Nov. 14, 2018
USD ($)
Dec. 31, 2020
USD ($)
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 ($)
Dec. 31, 2020
USD ($)
Option
Dec. 31, 2019
USD ($)
Dec. 31, 2018
USD ($)
Apr. 30, 2020
USD ($)
Dec. 12, 2019
Mar. 31, 2016
Feb. 12, 2016
Research and Development Arrangement, Contract to Perform for Others [Line Items]                                                  
Non-current deferred revenue             $ 11,776,000       $ 11,776,000       $ 11,776,000       $ 11,776,000 $ 11,776,000          
Current deferred revenue             960,000       2,341,000       960,000       2,341,000 960,000          
Research and development expense [1]                                     $ 266,946,000 179,362,000 $ 113,773,000        
Date of joint venture agreement                                     Dec. 19, 2015            
Operating expenses                     108,131,000 $ 92,547,000 $ 80,733,000 $ 73,743,000 66,033,000 $ 72,765,000 $ 55,301,000 $ 48,751,000 $ 355,154,000 242,850,000 162,067,000        
Stock-based compensation expense                                     66,018,000 49,524,000 39,260,000        
Net loss of joint venture                                     (348,865,000) 66,858,000 (164,981,000)        
Issuance of common shares, value                                     973,381,000 414,789,000 307,053,000        
Net proceeds from issuance of common shares                                     982,289,000 415,019,000 307,053,000        
Other (expense) income                                     $ 6,379,000 26,033,000 (1,210,000)        
Casebia Therapeutics Limited Liability Partnership [Member]                                                  
Research and Development Arrangement, Contract to Perform for Others [Line Items]                                                  
Research and development expense                                       700,000 3,800,000        
Reimbursements from research and license agreements                                       200,000 900,000        
Date of formation of joint venture entity                                     Feb. 12, 2016            
Equity method investment, ownership percentage                                             50.00% 50.00% 50.00%
Stock-based compensation expense                                       5,500,000 4,300,000        
Unrecognized equity method losses in excess of Company's interest                                       72,000,000.0          
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] | Equity Method Investment, Nonconsolidated Investee or Group of Investees [Member]                                                  
Research and Development Arrangement, Contract to Perform for Others [Line Items]                                                  
Net loss of joint venture $ 58,800,000                                       52,500,000        
Research and Development [Member]                                                  
Research and Development Arrangement, Contract to Perform for Others [Line Items]                                                  
Stock-based compensation expense                                     $ 35,120,000 23,273,000 17,557,000        
License and Service [Member] | Casebia Therapeutics Limited Liability Partnership [Member]                                                  
Research and Development Arrangement, Contract to Perform for Others [Line Items]                                                  
Collaboration revenue                                       500,000 2,500,000        
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 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                                       13,200,000          
2019 Option Agreement [Member] | Casebia Therapeutics Limited Liability Partnership [Member]                                                  
Research and Development Arrangement, Contract to Perform for Others [Line Items]                                                  
Equity method investment, ownership percentage                     50.00%               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            
Number of options exercised under agreement | Option                                     2            
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 [Member] | Casebia Therapeutics Limited Liability Partnership [Member]                                                  
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] | 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               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%                                                
2015 Casebia Agreement [Member]                                                  
Research and Development Arrangement, Contract to Perform for Others [Line Items]                                                  
Equity method investment                     0               0            
2019 Casebia Agreement [Member] | Other Current Liabilities [Member]                                                  
Research and Development Arrangement, Contract to Perform for Others [Line Items]                                                  
Research obligation             7,000,000.0               7,000,000.0         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               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            
Research Collaboration Agreement [Member]                                                  
Research and Development Arrangement, Contract to Perform for Others [Line Items]                                                  
Cost sharing percentage on specified costs             60.00%                                    
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         11,800,000          
Non-cash consideration received                                     10,000,000.0            
Transaction price allocated to remaining performance obligations             11,900,000               11,900,000         11,900,000          
Revenue                                     500,000 289,100,000          
Current deferred revenue             $ 900,000       400,000       900,000       400,000 900,000          
Collaboration revenue                                       30,000,000.0          
Maximum potential future payments                                     800,000,000            
Research and development expense                                     48,600,000 29,200,000 20,200,000        
Reimbursements from research and license agreements                                     28,200,000 15,900,000 13,800,000        
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            
Milestone payment receivable                     410,000,000.0               410,000,000.0            
Transaction price allocated to remaining performance obligations                     46,700,000               46,700,000            
Revenue                                       76,700,000          
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            
Revenue                                       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            
Revenue                                       289,100,000          
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            
Revenue                                       202,400,000          
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               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] | Waiving of Vertex?s Material Right [Member]                                                  
Research and Development Arrangement, Contract to Perform for Others [Line Items]                                                  
Revenue                                       6,700,000          
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               $ 825,000,000            
Percentage of exchange payment of research and development costs                                     50.00%            
Collaborative arrangement, license rights reacquired by waiving payment owed                                     $ 10,000,000.0            
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            
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               $ 6,700,000            
Vertex Pharmaceuticals Incorporated and Certain of its Subsidiaries [Member] | Non-Exclusive Research License [Member] | Research and Development Services [Member]                                                  
Research and Development Arrangement, Contract to Perform for Others [Line Items]                                                  
Revenue                                       $ 100,000 600,000        
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                                      
ViaCyte [Member] | Research Collaboration Agreement [Member]                                                  
Research and Development Arrangement, Contract to Perform for Others [Line Items]                                                  
Research and development expense                                         15,000,000.0        
Amount payable under collaboration agreement         $ 15,000,000.0                                        
Number of installments for amount payable | Installment         2                                        
Issuance of common shares | shares     214,512 165,636                                          
Issuance of common shares, value     $ 8,100,000 $ 7,500,000                                          
Key provisions of collaborative agreement description                                     The agreement includes certain provisions such that in the event ViaCyte sold shares received from the Company for less than $15.0 million in combined net proceeds, the Company would owe ViaCyte the deficient amount. In the event ViaCyte sold shares received from the Company for greater than $15.0 million in combined net proceeds, ViaCyte would owe the Company the surplus amount.            
Net proceeds from issuance of common shares                 $ 7,500,000 $ 6,900,000                              
Deficient amount payable                   $ 600,000                              
Deficient amount paid in cash   $ 600,000                                              
Total consideration paid                                         16,200,000        
Other (expense) income                                         $ (1,200,000)        
Collaboration agreement period         6 years                                        
Cost sharing percentage on specified costs             40.00%                                    
ViaCyte [Member] | Research Collaboration Agreement [Member] | Convertible Promissory Note [Member]                                                  
Research and Development Arrangement, Contract to Perform for Others [Line Items]                                                  
Additional amount payable under certain circumstances         $ 10,000,000.0                                        
[1] Including the following amounts of research and development from a related party, see Note 9