A significant step toward ensuring global access to advanced therapies for rare diseases was taken with the announcement of Rare Therapeutics, Inc. (RareTx), a new clinical-stage affiliate launched by GEMMABio, a global genetic medicines company. This new entity is dedicated exclusively to advancing accessible and sustainable gene therapies for ultra-orphan diseases, beginning its work with treatments for lysosomal storage disorders.

This initiative was created to address the critical failure of traditional biopharmaceutical companies to develop treatments for these conditions due to the limited markets and significant reimbursement challenges associated with one-time, high-priced treatments, which severely restricts global access and leaves the majority of rare disease communities without hope. Dr. James Wilson, President, CEO, and Founder of GEMMABio, affirmed that the goal of RareTx is to ensure all patients realize the promise of genetic medicines, independent of how rare their disease is or their country's ability to pay, through the use of best-in-class technologies in the context of a unique business model based on novel financing, development, and commercial strategies.

RareTx's portfolio centers on therapeutic platforms for monogenic diseases affecting the central nervous system (CNS) or heart/skeletal muscle, with lead clinical-stage CNS programs targeting the devastating ultra-orphan conditions of GM1 gangliosidosis, Krabbe disease, and metachromatic leukodystrophy (MLD). To accelerate timelines and reduce capital needs, the company's strategy involves developing products under these therapeutic platforms, and critically, delivering them through an international coalition of public-private partnerships, regional centers of excellence, and established hubs in Brazil and Abu Dhabi, thereby creating a commercial path to fulfill GEMMABio's mission to serve these long-underserved, ultra-rare disease communities.