National MPS Society Announces $4 Million Investment in Groundbreaking MPS IIIB Gene Therapy Program

Innovative Euro–U.S. collaboration aims to rapidly advance treatment for children with Sanfilippo syndrome type B.

DURHAM, NC, UNITED STATES, April 27, 2026 /EINPresswire.com/ -- The National MPS Society announced a landmark $4 million donor-directed grant to support a pioneering gene therapy program for MPS IIIB (Sanfilippo syndrome type B), marking one of the most significant research funding commitments in the organization’s history.

The initiative, titled the MPS IIIB Gene Therapy Euro–U.S. Research and Clinical Studies Program, led by Dr. Françoise Piguet at the Institut du Cerveau (Paris Brain Institute) represents a powerful international collaboration aimed at bringing a much-needed treatment to children living with this rare and devastating disease.

MPS IIIB is caused by a deficiency of the NAGLU enzyme, which is necessary to break down heparan sulfate in the body. The investigational therapy uses a modified adeno-associated virus (AAV) vector to deliver a functional copy of the NAGLU gene, targeting the underlying cause of the disease. The key characteristic of this program is the use of a novel AAV capsid that has been modified to very efficiently cross the blood-brain barrier after a simple intravenous injection and deliver a majority of the dose directly into the brain and surrounding cells.

This combination will significantly reduce the amount of vector needed to provide therapeutic benefit, which reduces manufacturing costs and allows more patients to be treated. The other benefit of this novel capsid is that the reduced dose needed for efficacy means it is much safer to deliver treatments to kids that already have systemic issues from their disease.

“This program reflects the urgency and determination of our community to accelerate meaningful treatments,” said Terri Klein, CEO of the National MPS Society. “Through the extraordinary generosity of a dedicated donor and the collaboration of global scientific leaders, we are taking a critical step toward changing the future for children with MPS IIIB.”

The program is designed to move rapidly into clinical application, with plans to initiate trials in both the United States and France within the next 10–14 months. Initial steps include expanded access treatment for the first U.S. patients, followed by a Phase I/II clinical trial enrolling children across both countries. In total, the program could treat approximately 15–17 children, with the potential to expand further based on manufacturing capacity.

The National MPS Society’s $4 million investment—fully funded through donor-directed contributions will be deployed in milestone-based tranches to advance the program from pre-clinical validation, GMP manufacturing, through regulatory preparation and clinical trial advancement.
This effort is further strengthened by more than $2 million in funding from French government sources and additional philanthropic contributions, underscoring the global commitment to advancing therapies for MPS IIIB.

The program brings together a world-class team of experts in gene therapy, clinical research, and regulatory science. Manufacturing will be conducted by U.S.-based Genezen, while regulatory oversight will be supported by Vanguard Clinical Associates—organizations with extensive experience in advancing innovative therapies through accelerated pathways.

Importantly, the program will leverage the U.S. expanded access (“n=1”) pathway to treat initial patients, enabling early safety and efficacy insights while formal clinical trials are underway.

“I believe that the Paris Gene Therapy Program and CapX capsid has the greatest potential benefit for these patients in my whole 30-year career in gene therapy,” said Dr. Scott Loiler, Chief Scientific Officer at the National MPS Society. “Families have driven this effort forward, and their commitment is helping to bring hope closer to reality.”

The National MPS Society remains committed to funding high-impact research, accelerating therapy development, and ensuring that families affected by MPS and ML have access to promising treatments as quickly as possible. With 30 years of gene therapy development experience, the CSO of the society will help guide and advise the program to completion.

About the National MPS Society
The National MPS Society is a nonprofit organization dedicated to curing, supporting, and advocating for individuals and families affected by mucopolysaccharidoses (MPS) and mucolipidoses (ML). Through research funding, education, and community support, the Society works to create a brighter future for those living with MPS and ML.

Media Contact:
Scott Loiler, Ph.D.
Chief Scientific Officer
National MPS Society
scott@mpssociety.org

Scott Loiler
National MPS Society
scott@mpssociety.org

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