Clinical setbacks and controversial pricing may have put a dampener on the gene therapy sector, but one of the industry’s pioneers is looking for a way forward.
GEMMA Biotherapeutics, the biotech founded by gene therapy trailblazer James Wilson M.D., Ph.D., has launched a clinical-stage spinout focused specifically on ultra-orphan diseases.
The new company, dubbed Rare Therapeutics, has been tasked with ensuring that “all patients living with a rare disease will realize the promise of genetic medicines, independent of how rare their disease is or the ability of their country to pay for it,” Wilson said in an Oct. 7 release.
RareTx is launching with a clinical-stage pipeline that includes central-nervous-system-focused programs aimed at lysosomal storage disorders called GM1 gangliosidosis, Krabbe disease and metachromatic leukodystrophy. These ultra-orphan diseases have “devastating clinical outcomes and limited treatment options,” GEMMA explained in the release.
“To achieve this, we will use best-in-class technologies in the context of a unique business that is based on novel financing, development and commercial strategies,” according to Wilson, who set up GEMMA in 2024 after over 30 years of pioneering gene therapy work at the University of Pennsylvania.
The plan with RareTx is to deliver treatments through an “international coalition of public-private partnerships and regional centers of excellence.” The new biotech’s links with GEMMA means it can draw on the parent company’s existing relationships in Brazil and Abu Dhabi, which will “serve as regional hubs for gene therapy in Latin America and the Middle East, respectively," according to the release.
“GEMMABio's mission is to make gene therapies accessible globally, and RareTx will fulfill this mission by creating a commercial path for patients with ultra-rare diseases who have long been underserved,” Annalisa Jenkins, chair of GEMMA’s board, added in the release.