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Revenue Arrangements - Narrative (Details)
 3 Months Ended
Jul. 28, 2020
USD ($)
Jun. 13, 2020
USD ($)
Jun. 11, 2020
USD ($)
obligation
Dec. 17, 2019
USD ($)
Mar. 31, 2021
USD ($)
Mar. 31, 2020
USD ($)
Dec. 31, 2020
USD ($)
Disaggregation of Revenue [Line Items]              
Short-term contract liability         $ 75,764,000   $ 72,050,000
Long-term contract liability         32,301,000   38,746,000
Contract assets amortization (less than)         100,000    
Accounts receivable         8,342,000   7,224,000
Revenue recognized         2,730,000    
Remaining performance obligation         108,100,000    
SOBI Purchase Agreement              
Disaggregation of Revenue [Line Items]              
Sale common stock     $ 25,000,000.0        
Redemption premium $ 14,500,000            
Short-term contract liability         72,000,000.0   68,300,000
Long-term contract liability         17,800,000   24,200,000
Contract assets         1,300,000    
Accounts receivable         8,300,000   6,900,000
Revenue recognized         11,100,000    
SOBI Purchase Agreement | Prepaid Expenses and Other Current Assets              
Disaggregation of Revenue [Line Items]              
Contract assets         1,000,000.0    
SOBI Purchase Agreement | Other Current Assets              
Disaggregation of Revenue [Line Items]              
Contract assets         300,000    
Collaborative Arrangement              
Disaggregation of Revenue [Line Items]              
Upfront cash payment     75,000,000.0        
Sales milestone payments     $ 630,000,000.0        
License and option agreement, written day notice period before cancellation     180 days        
Period after first commercial sale when the Company is eligible to receive royalties     10 years        
Number of obligations | obligation     3        
Development milestone     $ 5,000,000.0        
Sarepta Therapeutics, Inc.              
Disaggregation of Revenue [Line Items]              
Upfront cash payment   $ 2,000,000.0          
License and option agreement, written day notice period before cancellation   30 days          
Short-term contract liability         2,000,000.0   2,000,000.0
Revenue recognized         0 $ 0  
Option term   24 months          
License Agreement For Pompe Disease              
Disaggregation of Revenue [Line Items]              
Upfront cash payment       $ 7,000,000.0      
Sales milestone payments       $ 237,000,000.0      
Period after first commercial sale when the Company is eligible to receive royalties       10 years      
Short-term contract liability         1,700,000   1,700,000
Long-term contract liability         5,300,000   $ 5,300,000
Revenue recognized         $ 0 $ 0  
Initial up-front cash payment       $ 2,000,000.0      
Initial upfront execution fee payment       $ 5,000,000.0