Drug intelligence / Profile preview

AAV214

Development stage
Unknown
Lead developer
Abeona Therapeutics
Modality
Gene Therapies
Administration
Intravenous
01

Overview

**AAV214** is an investigational recombinant adeno-associated virus capsid developed by Abeona Therapeutics for use as a **gene therapy delivery vector**. Available public information indicates that AAV214 is part of Abeona's next-generation AAV capsid platform and has been studied preclinically as a vector with enhanced tropism and transduction relative to AAV9 in central nervous system and muscle-focused applications. In published and patent-disclosed preclinical work, AAV214 has been used to deliver transgenes such as **CLN3** and reporter genes, supporting potential application in inherited neuromuscular and neurologic disorders, including muscular diseases and CLN3 Batten disease. The specific therapeutic mechanism depends on the encoded transgene cargo, while the vector modality itself functions by mediating in vivo gene transfer after systemic administration.

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