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rAAV2-VMD2-hMERTK
- Development stage
- Phase 1
- Lead developer
- King Khaled Eye Specialist Hospital
- Modality
- Gene Therapies
- Administration
- Subretinal
01
Overview
rAAV2-VMD2-hMERTK is a gene therapy vector designed to treat retinitis pigmentosa caused by mutations in the MERTK gene. It consists of a recombinant adeno-associated virus serotype 2 (rAAV2) carrying the human MERTK gene under the control of the VMD2 promoter, which specifically targets expression to retinal pigment epithelium (RPE) cells. The therapy aims to restore MERTK function in RPE cells to prevent accumulation of photoreceptor outer segment debris in the subretinal space, which leads to photoreceptor degeneration.
02
Targets
MERTK (MER proto-oncogene, tyrosine kinase)
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