Rare Therapeutics

Rare Therapeutics is a private clinical-stage biotechnology company focused on developing gene therapies for ultra-orphan diseases. Launched on October 7, 2025, the company operates as an affiliate of GEMMABio and aims to improve global access and affordability for genetic medicines in very small patient populations. Its initial portfolio centers on lysosomal storage disorders including GM1 gangliosidosis, Krabbe disease, and metachromatic leukodystrophy. The company emphasizes platform-based development, next-generation gene delivery and manufacturing technologies, and public-private partnership models to support worldwide distribution.