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Crucible Therapeutics
Yorkshire
Founded 2023
siRNA gene therapies targeting root causes of ALS and frontotemporal dementia
Crucible Therapeutics Limited is a preclinical-stage biotechnology company developing siRNA-based gene therapies for Amyotrophic Lateral Sclerosis and Frontotemporal Dementia caused by the C9orf72 gene mutation.
Its therapeutic approach depletes the SRSF1 protein to suppress production of toxic dipeptide repeat proteins in motor neurons, delivered intrathecally to target the central nervous system.
The therapy acts on both sense and antisense repeat RNA simultaneously, distinguishing it from existing approaches.