GEMMABio Launches RareTx to Pioneer Global Access to Gene Therapies for Ultra-Rare Diseases

HILADELPHIA, PA – Oct 07 , 2025 – Gemma Biotherapeutics (“GEMMABio”), a leading genetic medicines company, today announced the formation of Rare Therapeutics, Inc. (“RareTx”), a new clinical-stage affiliate dedicated exclusively to advancing gene therapies for ultra-orphan diseases.

RareTx is launching with an initial focus on several promising treatments for lysosomal storage diseases that affect the central nervous system (CNS) or heart/skeletal muscle. The lead clinical-stage programs target GM1 gangliosidosis (GM1), Krabbe disease, and metachromatic leukodystrophy (MLD), all ultra-orphan diseases with limited treatment options and devastating clinical outcomes.

Addressing the Access Gap for Ultra-Orphan Diseases

The launch of RareTx is a direct response to the market failures that leave most rare disease patients without access to potential treatments. Traditional biopharmaceutical companies often avoid developing therapies for ultra-orphan diseases due to limited markets and significant global reimbursement challenges associated with high-cost, one-time treatments.

“Our goal with RareTx is to assure 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,” said Dr. James Wilson, President, CEO and Founder of GEMMABio.

RareTx’s strategy centers on a unique business model utilizing novel financing, development, and commercial strategies to make gene therapies accessible globally.

Global Coalition and Platform Approach

RareTx plans to deliver treatments through an international coalition of public-private partnerships and regional centers of excellence. As an affiliate of GEMMABio, RareTx has established key relationships, with Brazil and Abu Dhabi set to serve as regional hubs for gene therapy in Latin America and the Middle East, respectively.

Dr. Annalisa Jenkins, Chair of the Board for GEMMABio, stated that RareTx will fulfill GEMMABio’s mission by creating a “commercial path for patients with ultra-rare diseases who have long been underserved.”

By focusing on therapeutic platforms directed at disabling or lethal monogenic diseases, RareTx aims to increase the probability of success, accelerate timelines, and reduce capital needs. The company’s initial focus on CNS diseases positions it to substantially expand its portfolio within its specialized therapeutic platforms.

Rojan Vakili, a former Board Member for the Cure GM1 Foundation, highlighted the ethical imperative driving the need for this model: “There is a moral imperative for all stakeholders to work together and design bold new approaches befitting this unprecedented era in medicine.”