Hemgenix (etranacogene dezaparvovec-drlb) - CSL Behring
Beqvez (fidanacogene elaparvovec-dzkt) - Pfizer
Zolgensma (onasemnogene abeparvovec-xioi) - Novartis
Roctavian (valoctocogene roxaparvovec-rvox) - BioMarin
Elevidys (delandistrogene moxeparvovec-rokl) - Sarepta Therapeutics, Roche
Casgevy (exagamglogene autotemcel) - Vertex, CRISPR Therap
https://www.aasld.org/tlm-26/mte-24-gene-therapies-and-hepatotoxicity-ticketed
 
Tamir Diamond; Rajender Reddy
 
Aug 4, 2026
 
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