Hemgenix (etranacogene dezaparvovec-drlb) - CSL Behring
Adstiladrin (nadofaragene firadenovec-vncg) - Ferring
Zolgensma (onasemnogene abeparvovec-xioi) - Novartis
Kebilidi (eladocagene exuparvovec-tneq) - PTC Therapeutics
Roctavian (valoctocogene roxaparvovec-rvox) - BioMarin
Elevidys (delandistrogene moxeparvovec-rokl) - Sarepta Therapeutics, Roche
Glybera (alipogene tiparvovec) - uniQure, Chiesi, Xenon
Luxturna (voretigene neparvovec-rzyl) - Novartis, Roche
Review of Viral Shedding Profiles in Approved AAV-Based Gene Therapy Products: Implications for Safety and Environmental Impact (ASGCT 2026) - Mar 22, 2026 - Abstract #431; 522; Pres time: May 15, 2026; 08:00 AM - 09:45 AM; Location: MCEC Room 162AB (Level 1); "The GTPs were categorized based on their route of administration: locally administered (Luxturna, Upstaza, Glybera, Adstiladrin) and systemically administered (Beqvez, Roctavian, Hemgenix, Elevidys, Zolgensma)...Given that AAV is non-pathogenic to humans, it is feasible to standardize and simplify viral shedding monitoring in clinical studies. The standardization could streamline sampling schedules, reducing the burden on patients while still generating sufficient data to address potential transmission and environmental risks in compliance with regulatory requirements." 
Clinical • Gene therapy • Review • Duchenne Muscular Dystrophy • Gene Therapies • Hematological Disorders • Hemophilia • Hemophilia A • Movement Disorders • Muscular Atrophy • Muscular Dystrophy • Rare Diseases
https://pag.virtual-meeting.org/US/asgct2026/en-US/pag/presentation/684882
 
Ching-Ha Lai1,2
 
Mar 22, 2026
 
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