Adynxx
Agilis Biotherapeutics
Amicus Therapeutics
Antisense Therapeutics
Asklepios BioPharmaceutical
AveXis
Axovant Gene Therapies
Bamboo Therapeutics
Biogen
Ionis Pharmaceuticals
Jazz Pharmaceuticals
MeiraGTx Holdings
Novartis
Orchard Therapeutics
Pfizer
PTC Therapeutics
RegenxBio
Roche
SanBio
Sylentis
Takara Bio
UniQure NV
ViroMed
Voyager Therapeutics
WAVE Life Sciences
Xalud Therapeutics