As gene therapies advance toward indications with larger patient populations, adeno-associated virus (AAV) manufacturers are under growing pressure to deliver high-quality vectors at substantially ...
AB-1002 is an investigational one-time gene therapy administered directly to the heart to promote production of a modified version (I-1c) of the naturally occurring protein inhibitor-1, designed to ...
Announced in June, positive topline results from registrational STAAR study in Fabry disease, including positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52-weeks across ...
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