Drug intelligence / Profile preview

jwk002

Development stage: Phase 1
Lead developer: Genevector
Modality: Viral Vectors → Gene Addition/Replacement → Gene Therapies
Administration: Subretinal

Overview

JWK002 is a gene therapy drug for X-linked juvenile retinoschisis (XLRS) that uses AAV8 to deliver the Retinoschisin (RS1) gene into retinal cells. It works by specifically expressing the RS1 gene in photoreceptor cells to restore retinal structure and function in patients with XLRS disease. The therapy is designed to achieve long-term expression following a single subretinal injection, potentially providing an effective treatment option for patients with this rare genetic eye condition.

Other names: Recombinant adeno-associated virus 8 (AAV8) expressing human retinoschisin (RS1) gene

Targets

RS1 (Retinoschisin)ATP1A (Sodium/potassium-transporting ATPase)

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