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OPGx-CNGB1
- Development stage
- Unknown
- Lead developer
- Opus Genetics
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies
- Administration
- Ophthalmic
01
Overview
OPGx-CNGB1 is an adeno-associated virus (AAV) gene therapy developed by Opus Genetics for the treatment of retinitis pigmentosa caused by mutations in the CNGB1 gene. This late-onset form of retinitis pigmentosa specifically affects rod photoreceptors. The therapy utilizes AAV-mediated gene replacement to deliver a functional copy of the CNGB1 gene, which encodes a subunit of the cyclic nucleotide-gated channel essential for rod cell function and survival. By restoring the production of functional CNGB1 protein, the therapy aims to preserve and potentially improve vision in patients with this specific genetic mutation. OPGx-CNGB1 is currently in Phase 1 clinical development, supported by a consortium including the Foundation for the NIH's Bespoke Gene Therapy Consortium.
- Other names
- CNGB1-AAV gene therapyCNGB-1-AAV gene therapyCNGB 1-AAV gene therapy
02
Targets
CNGB1 (Cyclic nucleotide-gated channel beta-1)
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