Glafabra License Exclusive Rights to Issued Patent
Glafabra Therapeutics and the Medical College of Wisconsin Sign Exclusive License to the Lentiviral Vector Technology Behind the Live-cel Gene Therapy Platform
The agreement grants Glafabra exclusive rights, in the field of Fabry disease and Gaucher disease, to MCW’s issued and pending patents covering an improved lentiviral vector transfer plasmid, securing the vector technology used to manufacture Glafabra’s lead program, GT-GLA-S03, ahead of a planned 2027 IND filing.
PARK CITY, Utah, and MILWAUKEE, Wisconsin, September 11, 2026. Glafabra Therapeutics, a preclinical, pre-IND gene therapy company developing Live-cel, a lentivirus-mediated autologous stem-cell gene therapy platform for lysosomal storage disorders, and the Medical College of Wisconsin (MCW) today announced they have entered into an exclusive patent license agreement covering MCW’s improved lentiviral vector transfer plasmid technology. The agreement took effect on August 18, 2026.
The licensed patent estate, MCW Case C2030, is directed to an improved lentiviral vector transfer plasmid and methods of its use. It includes U.S. Patent 12,540,336, issued February 3, 2026, together with a pending U.S. continuation application and pending applications in Canada and Europe.
The vector transfer plasmid is a core component of the manufacturing process used to produce Glafabra’s gene-modified autologous cell products, and the license places that component under exclusive control for the licensed indications.
Terms of the agreement
Under the agreement:
Exclusivity and field. MCW granted Glafabra an exclusive, royalty-bearing license to make, have made, use, offer for sale, sell, and import products covered by the licensed patents for the treatment of Fabry disease and Gaucher disease, with the right to grant sublicenses.
Patent estate. The license covers U.S. Patent 12,540,336 and the pending applications in the same family, including a U.S. continuation application directed to indication-specific claims, together with their divisionals, continuations, reissues, and foreign counterparts.
Territory. The United States, together with each country in which a licensed patent or application is pending, and a defined mechanism for Glafabra to request additional countries.
Consideration. Glafabra will pay an upfront license issue fee, development and commercial milestone payments, a low single-digit running royalty on net sales of licensed products, a share of non-royalty sublicensing consideration, and a one-time payment upon a change of control. Specific financial terms were not disclosed.
Diligence. Glafabra has committed to defined development milestones, including submission of an Investigational New Drug application for a Fabry product, first patient enrolled, and subsequent clinical, regulatory, and commercial milestones, with a parallel schedule for a Gaucher product.
Reserved rights. MCW retains customary rights to publish and to use the licensed technology for internal research and educational purposes, and the license is subject to the rights of the U.S. government under 35 U.S.C. 200-212.
Why the license matters
For a cell-based gene therapy, the transfer plasmid is not a peripheral reagent. It is the element that determines how the therapeutic gene is packaged and delivered into a patient’s own hematopoietic stem cells, and it is embedded in the chemistry, manufacturing, and controls package that FDA will review. Bringing that component under an exclusive license, rather than relying on a research-use arrangement, gives Glafabra a defined and defensible position on the vector technology as it prepares its IND and engages potential partners.
The agreement also aligns the two indications in which the modality now carries durable human data. Glafabra’s Fabry program follows the five-year FACTS proof-of-concept experience generated by the company’s scientific co-founders in Canada, and an independent peer-reviewed first-in-human report has described five-year normalization of glucocerebrosidase activity in Gaucher disease following a single autologous lentiviral CD34-positive cell infusion. Licensing Fabry and Gaucher together under one estate matches the company’s development sequence.
Management commentary
“The vector is where a cell therapy program lives or dies, and this agreement puts the vector technology at the center of Live-cel on a clear, exclusive footing for the two indications we are taking forward,” said Dr. Chris Hopkins, Chief Executive Officer of Glafabra Therapeutics. “We completed our FDA INTERACT meeting in July with a defined path to an IND. Closing this license removes an intellectual property question from that path and lets us put our attention where it belongs, on manufacturing readiness and getting a first patient dosed.”
“Translating discoveries made at the Medical College of Wisconsin into therapies that reach patients is central to our mission,” says Kevin Boggs, Director, Office of Technology Development, Medical College of Wisconsin. “Glafabra has assembled the regulatory groundwork and clinical experience to advance this technology in lysosomal storage disorders, and we are pleased to see it move toward the clinic.”
About Fabry disease and GT-GLA-S03
Fabry disease is a rare, X-linked lysosomal storage disorder caused by deficient activity of the enzyme alpha-galactosidase A, leading to progressive accumulation of the substrate Gb3 and its derivative lyso-Gb3 and to multi-system organ damage. The current standard of care, enzyme replacement therapy, requires lifelong intravenous infusions every two weeks, produces a peak-and-trough enzyme cycle, and does not halt long-term organ damage. GT-GLA-S03 is designed to replace that infusion burden with a single outpatient procedure that delivers durable, steady-state enzyme expression from the patient’s own gene-modified cells, using a reduced-intensity, non-myeloablative conditioning regimen. Because the approach is autologous and uses no viral capsid, it is designed to be re-administrable and is not subject to the anti-capsid antibody exclusions that render a substantial fraction of adults ineligible for other gene therapy approaches. FDA granted Orphan Drug Designation to GT-GLA-S03 in March 2026, and Glafabra completed an INTERACT meeting with FDA on July 16, 2026.
Human proof of concept
The Live-cel platform is supported by five-year human proof-of-concept data from an investigator-initiated clinical trial (the FACTS trial, NCT02800070) conducted by Glafabra’s scientific co-founders, Dr. Jeffrey Medin and Dr. Ronan Foley, in Canada under Canadian regulatory oversight. Across five patients followed for five years, the trial showed a 48 percent reduction in plasma lyso-Gb3 from a no-enzyme-replacement-therapy baseline (p less than 0.0001), with no product-attributable serious adverse events. This work is proof of concept for the platform and is distinct from any Glafabra-sponsored IND program. Glafabra is pre-IND from FDA’s perspective.
About Glafabra Therapeutics
Glafabra Therapeutics is a preclinical, pre-IND gene therapy company developing Live-cel, a lentivirus-mediated autologous stem-cell gene therapy platform for lysosomal storage disorders. A single manufacturing process is applied across programs, with the lead program, GT-GLA-S03, in development for classic Fabry disease and a follow-on program, GT-GBA1-S05, in development for Gaucher disease. The platform is supported by five-year human proof-of-concept data from an investigator-initiated trial conducted by the company’s scientific co-founders in Canada. Glafabra is targeting an IND filing in 2027. Learn more at glafabra.com.
About the Medical College of Wisconsin
With a history dating back to 1893, the Medical College of Wisconsin is dedicated to leadership and excellence in education, patient care, research, and community engagement. More than 1,700 students are enrolled in MCW’s medical, graduate and pharmacy schools at campuses in Milwaukee, Green Bay, and Central Wisconsin. MCW’s School of Pharmacy opened in 2017. A major national research center, MCW ranks in the top 4% of U.S. research institutions (InCites Essential Science Indicators Dataset), is the largest research institution in the Milwaukee metro area and is the largest private research institution in Wisconsin. Annually, our faculty direct or collaborate on more than 5,100 research studies, including clinical trials. In the last 10 years, MCW faculty have received nearly $2 billion in external support for research, teaching, training, and related purposes. Additionally, our more than 1,900 physicians provide care in virtually every specialty of medicine, annually fulfilling more than 5.7 million patient visits.
Media and Company Contact
Dr. Chris Hopkins
Chief Executive Officer, Glafabra Therapeutics
chris@glafabra.com | +1 801 631 9114 | glafabra.com
Colleen McDonaldOffice of Communications, Medical College of Wisconsin
media@mcw.edu | 414.955. 8764 | mcw.edu
Forward-Looking Statements
This press release contains forward-looking statements, including statements regarding Glafabra’s development plans, the anticipated timing of a future IND filing, the scope, prosecution, and enforceability of the licensed patents, and the potential of the Live-cel platform. Forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties that could cause actual results to differ materially, including risks related to preclinical and clinical development, patent prosecution and validity, regulatory review and timing, manufacturing, and financing. Pending patent applications may not issue, and issued claims may be narrowed, challenged, or invalidated. Glafabra undertakes no obligation to update any forward-looking statement except as required by law.
