GM1 Gangliosidosis (GM1) Gene Therapy

About Rare Therapeutics

RareTx is a private, clinical-stage company tackling the significant unmet need in ultra-rare genetic diseases through advanced gene therapies. The company differentiates itself via a platform-based development approach and an innovative commercial strategy centered on an international coalition of partnerships, aiming to ensure sustainable and affordable access. Founded in 2018 and based in Cambridge, MA, it is building on the pioneering gene therapy work of Dr. Jim Wilson to advance treatments for pediatric-onset disorders with devastating prognoses.

Therapeutic Areas