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AVST-101

Development stage: Preclinical
Lead developer: Avista Therapeutics
Modality: Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration: Intravitreal

Overview

AVST-101 is an AAV gene therapy candidate developed by Avista Therapeutics. It is being advanced for the treatment of X-linked retinoschisis (XLRS), a rare inherited retinal disease. The therapy aims to deliver genetic material to address the underlying cause of the disease. Forge Biologics is collaborating on its development and cGMP manufacturing, providing process development, toxicology, and analytical services.

Targets

RS1 (Retinoschisin)

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