Philadelphia, October 7, 2025 — GEMMABio, a leading genetic medicines company founded by gene therapy pioneer Dr. James M. Wilson, has launched Rare Therapeutics, Inc. (RareTx) — a clinical-stage affiliate dedicated to developing and delivering gene therapies for ultra-orphan diseases.
RareTx opens its doors with a portfolio targeting lysosomal storage diseases that affect the central nervous system (CNS), including GM1 gangliosidosis, Krabbe disease, and metachromatic leukodystrophy (MLD) — devastating pediatric disorders with limited or no existing 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. Wilson, GEMMABio’s President, CEO and Founder.
A Model Built for the Rarest of the Rare
Ultra-orphan diseases — those affecting just a handful of patients worldwide — often fall outside the scope of traditional biopharma pipelines. The economics simply don’t work: one-time gene therapies are expensive to develop and hard to justify under conventional reimbursement models.
“Charging high prices for these one-and-done treatments is not a global solution,” the company stated, noting that the result is a widening gap between what science can achieve and what current systems will support.
RareTx aims to bridge that gap through what Dr. Wilson calls “a unique business based on novel financing, development and commercial strategies.” It’s a model that emphasizes scalability, access, and global reach over traditional market size — effectively rewriting the rules for how ultra-rare diseases can be addressed.
Strategic Design of RareTx — What’s New, What’s Inherited
RareTx represents both continuity and innovation. It builds upon the scientific and operational foundation that GEMMABio established since its formation in 2024, when Wilson transitioned from academia at the University of Pennsylvania’s Gene Therapy Program to launch GEMMABio as an independent platform for translational genetic medicine.
In its first year, GEMMABio acquired three clinical-stage programs from Passage Bio — the same GM1, Krabbe, and MLD candidates now forming RareTx’s lead portfolio. Those assets gave the company an immediate foothold in high-need pediatric indications and an established preclinical and regulatory foundation.
What’s new is how RareTx intends to execute. Rather than approaching each disease as a standalone project, the company is organized around two therapeutic platforms — one focused on monogenic diseases of the CNS, and the other on heart and skeletal muscle. This approach allows for shared technology, manufacturing, and regulatory pathways that can accelerate development and reduce cost.
“RareTx’s strategy to develop products for disabling or lethal ultra-orphan diseases under therapeutic platforms will increase probabilities of success, accelerate timelines and reduce capital needs,” said Dr. Annalisa Jenkins, Chair of GEMMABio’s Board.
RareTx will also rely on a global network of public-private partnerships and regional centers of excellence to expand patient access. Leveraging GEMMABio’s existing collaborations, Brazil and Abu Dhabi will serve as regional hubs for Latin America and the Middle East — regions often left out of the advanced therapeutics ecosystem.
A Moral Imperative
For patient advocates, the formation of RareTx represents more than a strategic step — it’s a moral one.
“Time is against us, and it is unthinkable to turn back now, just as transformative therapies are finally ready for the clinic,” said Rojan Vakili, a former Board Member for the Cure GM1 Foundation whose young son lives with GM1 gangliosidosis. “There is a moral imperative for all stakeholders to work together and design bold new approaches befitting this unprecedented era in medicine.”
By embedding that patient perspective at the core of its model, RareTx seeks to redefine what “access” means in the age of genetic medicine — ensuring that innovation reaches those who need it most, not just those who can afford it.
Global Access, Local Impact
GEMMABio’s broader mission has always centered on democratizing access to genetic therapies. Recent partnerships with Brazil’s Fiocruz Institute and the Abu Dhabi Department of Health have laid the groundwork for this vision, providing local infrastructure for clinical development and manufacturing. RareTx will now operationalize that global vision with a focus on the rarest conditions, testing whether a sustainable and equitable model for gene therapy delivery is finally within reach.
“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 Jenkins.
As RareTx begins operations, the company faces both scientific and systemic challenges — from regulatory complexity to long-term economic sustainability. Yet for Wilson and his team, the equation remains simple: the science is ready, and the need is urgent.
If RareTx can prove that ultra-rare gene therapy development is not only possible but replicable, it could redefine how the world delivers innovation to its most overlooked patients.
Full press release: https://www.prnewswire.com/news-releases/gemmabio-launches-rare-therapeutics-inc-to-develop-and-deliver-gene-therapies-for-ultraorphan-diseases-302576274.html
