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EMD-202
- Development stage
- Preclinical
- Lead developer
- EmendoBio
- Modality
- Peripheral Blood HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies, Gene Therapies, Bone Marrow HSCs → Hematopoietic Stem Cells → Adult Stem Cells → Stem Cell Therapies → Cell Therapies
- Administration
- Ophthalmic, Intravenous
01
Overview
EMD-202 is a preclinical gene editing therapy developed by Emendo Biotherapeutics for the treatment of cone-rod dystrophies, a group of inherited retinal disorders. The drug utilizes gene modulation techniques and is classified as a hematopoietic stem cell therapy. EMD-202 is part of Emendo's ophthalmic program and leverages their OMNI technology platform to target genetic mutations associated with cone-rod dystrophy. The primary mechanism involves gene modulation, though specific molecular targets are not detailed in public sources[1][3][4][8].
02
Targets
RPE65 (Retinal pigment epithelium-specific 65 kDa protein)
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