RT102

About Regel Therapeutics

Regel Therapeutics, founded in 2021 and based in Cambridge, USA, is developing epigenetic therapies for severe genetic diseases. Its core technology, the Targeted EpiEditing platform, combines a deactivated CRISPR-Cas9 system with epigenetic modulators and cell-type-specific AAV delivery vectors to correct gene expression exclusively in affected cells. The company is led by an experienced team, including CEO Stephen Farr, who previously led Zogenix to acquisition, and is advancing two lead preclinical programs for Dravet Syndrome and SCN2A Haploinsufficiency.

Therapeutic Areas