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PUMCH-E101
- Development stage
- Unknown
- Lead developer
- Peking Union Medical College Hospital
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Gene Silencing → Gene Therapies, Gene Editing → Gene Therapies
- Administration
- Intravitreal
01
Overview
PUMCH-E101 is an investigational gene therapy based on adeno-associated virus (AAV) technology, designed for the treatment of inherited retinal diseases caused by mutations in the RDH12 gene. The therapy involves a single intravitreal injection intended to deliver a functional copy of the RDH12 gene to retinal cells, aiming to restore or preserve vision in affected individuals. The drug is being developed and trialed by Peking Union Medical College Hospital, with clinical studies currently focused on patients with RDH12 retinopathy—a form of inherited retinal degeneration that leads to progressive vision loss. Early-phase clinical trials are ongoing to evaluate its safety and preliminary efficacy[6][8][10].
02
Targets
RDH12 (Retinol dehydrogenase 12)
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