New clinical-stage company will accelerate treatments for patients with ultra-rare diseases worldwide
PHILADELPHIA, Oct. 7, 2025 /PRNewswire/ -- Gemma Biotherapeutics ("GEMMABio") – a leading global genetic medicines company today announced the launch of Rare Therapeutics, Inc. ("RareTx"), a clinical-stage company focused exclusively on advancing gene therapies for ultra-orphan diseases. RareTx commences operations today with a focus on several promising treatments for lysosomal storage diseases.
Traditional biopharmaceutical companies are not developing gene therapies for ultra-orphan diseases due to the 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, as it restricts access to a limited number of high-income individuals and countries. This widens a growing gap between what can be done and what the current system will support, leaving most rare disease communities without access to potential 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. "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."
RareTx's portfolio is based on therapeutic platforms directed to monogenic disease that affect the central nervous system (CNS) or heart/skeletal muscle. The lead clinical-stage CNS programs include GM1 gangliosidosis ("GM1"), Krabbe disease ("Krabbe") and metachromatic leukodystrophy ("MLD"), ultra-orphan diseases which have devastating clinical outcomes and limited treatment options. RareTx has the expertise and technology to enable a substantial expansion of its portfolio within its two therapeutic platforms.
"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 Dr. Annalisa Jenkins, Chair of the Board for GEMMABio and a renowned biopharma thought leader with extensive industry experience.
RareTx's strategy to develop products for disabling/lethal ultra-orphan diseases under therapeutic platforms will increase probabilities of success, accelerate timelines and reduce capital needs. 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 key relationships in Brazil and Abu Dhabi, which will serve as regional hubs for gene therapy in Latin America and the Middle East, respectively.
"Rare disease families share personal stories of immense love and joy alongside profound grief as our loved ones experience progressive and irreversible decline. We work tirelessly to support the search for effective treatments and instill hope," said Rojan Vakili, MBA, a former Board Member for the Cure GM1 Foundation whose young son lives with GM1 gangliosidosis. "Time is against us, and it is unthinkable to turn back now, just as transformative therapies are finally ready for the clinic. There is a moral imperative for all stakeholders to work together and design bold new approaches befitting this unprecedented era in medicine."
For more information, please visit raretx.us.
About GEMMABio
GEMMABio is a therapeutics company focused on speeding the research of and global access to life-changing advanced therapies for those living with rare diseases. The company conducts research and product development functions to bring gene therapy discoveries from the bench to the bedside faster and affordably. GEMMABio is led by gene therapy industry pioneer Jim Wilson and his team of experts, who previously conducted their work in academia. Learn more about GEMMABio at gemmabiotx.com.
About Rare Therapeutics
Rare Therapeutics is a clinical-stage company and affiliate of GEMMABio. It is focused on developing and delivering accessible and sustainable gene therapies for ultra-orphan diseases. Through cutting-edge science and global partnerships, RareTx is committed to developing transformative therapies and making those therapies available to the people who need them most. Learn more about RareTx at raretx.us.
Contact: Scott Hoeflich
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SOURCE GEMMA Biotherapeutics
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