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NGRF-GMAC
- Development stage
- Preclinical
- Lead developer
- CellGenTech
- Modality
- Cell Therapies, Gene Therapies
- Administration
- Subcutaneous
01
Overview
NGRF-GMAC is an experimental autologous *ex vivo* gene therapy being developed by CellGenTech for the treatment of retinitis pigmentosa. The therapy utilizes the proprietary Genetically Modified Adipocyte (GMAC) platform, which involves harvesting a patient's adipose tissue via a minimally invasive lipectomy. These adipocytes are then genetically modified *ex vivo* to express or modulate the Nerve Growth Factor Receptor (NGFR) and subsequently reintroduced into the patient. This approach aims to provide neuroprotective effects to the retina, potentially slowing the progression of retinal degeneration and vision loss associated with retinitis pigmentosa. The program is currently in the preclinical stage and has received support from the Japan Agency for Medical Research and Development (AMED).
- Other names
- Nerve Growth Factor Receptor-GMACCellGenTech NGRF-GMAC
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