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RPGR gene therapy (BST BIO)
- Development stage
- Preclinical
- Lead developer
- Janssen Research & Development
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Ophthalmic
01
Overview
BST BIO (Bavarian Systems and Technologies GmbH) is developing an adeno-associated virus (AAV)-based gene replacement therapy for the treatment of X-linked retinitis pigmentosa (XLRP). XLRP is a severe form of inherited retinal degeneration primarily caused by mutations in the *RPGR* (Retinitis Pigmentosa GTPase Regulator) gene, which is essential for the maintenance of photoreceptor cilia. The therapy utilizes an AAV vector to deliver a functional copy of the *RPGR* gene to the retina, aiming to restore protein function, preserve photoreceptor structure, and prevent further vision loss. The program is currently in the preclinical stage of development.
- Other names
- RPGR-AAV gene therapyBST BIO RPGR gene therapy
02
Targets
RPGR (Retinitis pigmentosa GTPase regulator)
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