OTARMENI (lunsotogene parvec-cwha)
Modality: AAV Gene Therapy
Approved Products
CMC product library
CMC-focused profiles of approved gene therapy, cell therapy, biologic, and advanced modality products, organized around product design, route, dose expression, quality attributes, analytical controls, and public regulatory source documents.
Current focus: FDA-approved AAV gene therapies
Expanding to biologics, cell therapies, and other advanced modalities
Product Profiles
ITVISMA (onasemnogene abeparvovec-brve)
Modality: AAV Gene Therapy
ZOLGENSMA (onasemnogene abeparvovec-xioi)
Modality: AAV Gene Therapy
ROCTAVIAN (valoctocogene roxaparvovec-rvox)
Modality: AAV Gene Therapy
LUXTURNA (voretigene neparvovec-rzyl)
Modality: AAV Gene Therapy
KEBILIDI (eladocagene exuparvovec-tneq)
Modality: AAV Gene Therapy
HEMGENIX (etranacogene dezaparvovec-drlb)
Modality: AAV Gene Therapy
ELEVIDYS (delandistrogene moxeparvovec-rokl)
Modality: AAV Gene Therapy
BEQVEZ (Fidanacogene elaparvovec-dzkt)
Modality: AAV Gene Therapy