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RGT-HD
- Development stage
- Preclinical
- Lead developer
- Rgenta Therapeutics
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
RGT-HD is a small molecule therapeutic candidate being developed by Rgenta Therapeutics for the treatment of Huntington's Disease. It is designed to target PMS1, a protein involved in the DNA mismatch repair (MMR) pathway. In Huntington's Disease, the progressive expansion of CAG repeats in the huntingtin (HTT) gene is a primary driver of neurodegeneration; PMS1 has been identified as a key genetic modifier that promotes this somatic expansion. By inhibiting PMS1, RGT-HD aims to stabilize or prevent the further expansion of these repeats, potentially slowing the progression of the disease. The program is currently in the IND-enabling stage of development.
02
Targets
PMS1 (PMS1 homolog 1)
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