Selonterra C9orf72 modulator

Selonterra's C9orf72 modulator is an investigational small molecule currently in the lead optimization stage for the treatment of Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD). The drug is designed to target the C9orf72 gene product, which is the most common genetic cause of both ALS and FTD, typically due to a hexanucleotide repeat expansion. By modulating C9orf72, the therapy aims to address the underlying disease pathology, potentially reducing the toxic effects associated with the mutation or restoring normal cellular function. This program represents a targeted approach to treating neurodegenerative diseases with a known genetic driver.

Other names

C9orf72 modulatorC-9orf72 modulatorC 9orf72 modulator