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RT101

Development stage: Preclinical
Lead developer: Regel Therapeutics
Modality: CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
Administration: Oral

Overview

RT101 is a preclinical gene therapy developed by Regel Therapeutics. It utilizes the company's proprietary T3 platform, also known as Targeted EpiEditing, which employs a non-editing, deactivated CRISPR system to restore normal gene expression. This technology is designed for treating genetic disorders. RT101 is the lead preclinical program targeting Dravet Syndrome, a severe form of epilepsy. The development of RT101 is supported by non-dilutive funding from the HS Chau Foundation, administered through the Women in Enterprising Science (WIES) Program at the Innovative Genomics Institute (IGI).

Targets

SCN1A (Voltage-gated sodium channel protein type 1 subunit alpha)

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