Drug intelligence / Profile preview

JWK010

Development stage
Unknown
Lead developer
Chengdu Genevector Biotechnology
Modality
Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration
Ophthalmic
01

Overview

JWK010 is an investigational gene therapy being developed by West China Hospital of Sichuan University for the treatment of oculocutaneous albinism type 1 (OCA1). OCA1 is a genetic disorder caused by mutations in the *TYR* gene, which encodes the enzyme tyrosinase, essential for the production of melanin pigment. JWK010 is designed to deliver a functional copy of the *TYR* gene to ocular tissues via suprachoroidal injection. This delivery method is intended to target the retinal pigment epithelium and other posterior segment structures to restore tyrosinase activity and melanin production, potentially improving visual function and retinal development in affected individuals. The therapy is currently being evaluated in a single-arm interventional study involving pediatric subjects to assess its safety, tolerability, and effects on visual function and retinal structure.

02

Targets

TYR (Tyrosinase)

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