GEMMABio Launches Rare Therapeutics to Develop Gene Therapies for Ultra‑Orphan Diseases

Rare Daily Staff

Gemma Biotherapeutics, the genetic medicines company founded by gene therapy pioneer Jim Wilson, said it has spun out Rare Therapeutics, a clinical-stage company focused on advancing gene therapies for ultra-orphan diseases.

The company said traditional biopharmaceutical companies have not developed gene therapies for ultra-orphan diseases because of limited markets and reimbursement challenges associated with one-time treatments for those conditions. Charging high prices for these one-and-done treatments is not a global solution, it added, as this restricts access to a limited number of high-income individuals and countries. The approach has widened the gap between what is scientifically possible and what the current system supports, leaving most rare disease communities without access to potential treatments.

Rare Therapeutics’ strategy for developing products to treat disabling and lethal ultra-orphan diseases under therapeutic platforms aims to increase the probability of success, accelerate timelines, and reduce capital needs.

“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,” said Annalisa Jenkins, chair of the board for GemmaBio.

Treatments will be delivered through an international coalition of public-private partnerships and regional centers of excellence. As an affiliate of GemmaBio, RareTx has access to relationships in Brazil and Abu Dhabi, which will serve as regional hubs for gene therapy in Latin America and the Middle East.

The company’s initial candidates focus on treatments for lysosomal storage diseases. Lead clinical-stage central nervous system programs include GM1 gangliosidosis, Krabbe disease, and metachromatic leukodystrophy, ultra-orphan conditions with devastating clinical outcomes and limited treatment options. The company said it has the expertise and technology to substantially expand its portfolio within its two therapeutic platforms.

“Our goal with RareTx is to assure that all patients living with a rare disease realize the promise of genetic medicines, independent of how rare their disease is or the ability of their country to pay for it,” said Wilson, president and CEO of GemmaBio. “To achieve this, we will use best-in-class technologies in the context of a unique business based on novel financing, development, and commercial strategies.”