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RGT-0474060

Development stage: Preclinical
Lead developer: Rgenta Therapeutics
Modality: Small Molecules
Administration: Oral

Overview

RGT-0474060 is an orally bioavailable, brain-penetrant small molecule drug candidate developed by Rgenta Therapeutics for the treatment of Huntington's disease. It acts by inhibiting the expression of PMS1 (Postmeiotic Segregation Increased 1) at both the RNA and protein levels. By targeting PMS1, the drug modulates the splicing of CAG (cytosine-adenine-guanine) repeat expansions in the HTT gene, thereby halting somatic CAG repeat expansion, which is the primary driver of Huntington's disease progression. As of early 2026, the candidate is in IND-enabling studies with plans to initiate clinical trials in 2027.

Targets

PMS1 (PMS1 homolog 1)

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