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RP20-RLBP1
- Development stage
- Unknown
- Lead developer
- InnoVec Pharmaceutical Technology
- Modality
- Gene Therapies
- Administration
- Intravitreal
01
Overview
RP20-RLBP1 (also known as PUMCH-E111) is an adeno-associated virus (AAV) gene therapy candidate being developed by InnoVec Pharmaceutical Technology in collaboration with Peking Union Medical College Hospital (PUMCH). It is designed to treat Retinitis Pigmentosa type 20 (RP20), a rare form of hereditary retinal dystrophy caused by mutations in the RLBP1 gene. The RLBP1 gene encodes the cellular retinaldehyde-binding protein (CRALBP), which is essential for the visual cycle in the retinal pigment epithelium and Müller cells. By delivering a functional copy of the RLBP1 gene, the therapy aims to restore CRALBP activity, thereby maintaining the visual cycle and preventing further vision loss. RP20-RLBP1 is currently in Phase 1 clinical development, with the first patient dosed in 2026.
- Other names
- RP20-RLBP1RP-20-RLBP1RP 20-RLBP1PUMCH-E111PUMCH-E-111PUMCH-E 111
02
Targets
RLBP1 (Retinaldehyde Binding Protein 1)
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