1. Abeona Therapeutics Inc.
2. Alexion Pharmaceuticals Inc.
3. Amicus Therapeutics
4. ArmaGen Inc.
5. BioMarin
6. CHIESI USA Inc.
7. Denali Therapeutics Inc.
8. Freeline Therapeutics
9. Homology Medicines Inc.
10. Horizon Pharma PLC
11. Idorsia Pharmaceuticals Ltd.
12. JCR Pharmaceuticals Co. Ltd.
13. Lysogene
14. Neurogene Inc.
15. Novartis AG
16. Orchard Therapeutics Limited
17. Pfizer Inc.
18. Protalix
19. Sangamo Therapeutics Inc.
20. Sanofi S.A.
21. Spark Therapeutics Inc.
22. Swedish Orphan Biovitrum AB
23. Takeda Pharmaceutical Company Limited
24. Ultragenyx Pharmaceutical Inc.