Drug Profile

RGT 0474060

Alternative Names: RGT-0474060

Latest Information Update: 27 Mar 2026

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At a glance

Originator Rgenta Therapeutics
Class Small molecules
Mechanism of Action PMS1 protein inhibitors

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity Yes

Highest Development Phases

Preclinical Huntington's disease

Most Recent Events

25 Feb 2026 Preclinical trials in Huntington's disease in United Kingdom (PO)
25 Feb 2026 Pharmacodynamics, pharmacokinetics and adverse event data from preclinical trial in Huntington’s disease released by Rgenta Therapeutics
25 Feb 2026 Rgenta Therapeutics plans to enter clinical trials for Huntington’s Disease in 2027 (PO)

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