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Agreements Related to Intellectual Property - Additional Information (Details)
€ in Thousands, $ in Thousands
12 Months Ended
Nov. 30, 2017
EUR (€)
Nov. 30, 2017
USD ($)
Jul. 31, 2016
EUR (€)
Jul. 31, 2016
USD ($)
Mar. 31, 2016
EUR (€)
Mar. 31, 2016
USD ($)
Dec. 31, 2024
USD ($)
Patent
Milestone
Dec. 31, 2023
Milestone
Dec. 31, 2022
Milestone
Dec. 31, 2020
USD ($)
Dec. 31, 2019
USD ($)
Dec. 01, 2022
USD ($)
Nov. 30, 2017
USD ($)
Feb. 28, 2017
USD ($)
Jul. 31, 2016
USD ($)
Mar. 31, 2016
USD ($)
License Agreement for Danon Disease                                
License Agreement [Abstract]                                
Research and development costs                   $ 50            
REGENXBIO, Inc. License                                
License Agreement [Abstract]                                
Options right expiry term               4 years                
License Agreement for DD with RGNX                                
License Agreement [Abstract]                                
Research and development costs             $ 7,000                  
Potential future obligation             $ 13,000                  
Term of agreement             10 years                  
Termination notice period             6 months                  
Number of patents | Patent             2                  
Royalties payable on fees received             20.00%                  
Payment and expenses for license fee             $ 2,000       $ 1,000          
Number of additional milestones achieved | Milestone             0 0 0              
License Agreement for PKD with CIEMAT Group                                
License Agreement [Abstract]                                
Research and development costs         € 30 $ 30                    
Potential future obligation         € 1,400                     $ 1,500
Termination notice period             90 days                  
Number of least uninterrupted years             2 years                  
License Agreement for FA with CIEMAT Group and FIBHNJS                                
License Agreement [Abstract]                                
Research and development costs     € 100 $ 100                        
Potential future obligation     € 5,000                       $ 5,500  
Termination notice period             90 days                  
Number of least uninterrupted years             2 years                  
License Agreement for LAD-I with CIEMAT and UCLB | Maximum                                
License Agreement [Abstract]                                
Potential future obligation                           $ 1,500    
License Agreement for LAD-I with CIEMAT Group and UCLB                                
License Agreement [Abstract]                                
Research and development costs € 30 $ 40                            
Potential future obligation € 1,400                       $ 1,500      
Termination notice period             90 days                  
Number of least uninterrupted years             2 years                  
License Agreement for BAG3 ith Temple University | Maximum                                
License Agreement [Abstract]                                
Potential future obligation                       $ 19,250