Rutgers University signed two agreements with Massachusetts-based Santhera Pharmaceuticals to advance pioneering research for the treatment of congenital muscular dystrophy.
A licensing agreement and a research collaboration complement the ongoing work of Peter Yurchenco, professor of the Department of Pathology and Laboratory Medicine at Rutgers Robert Wood Johnson Medical School. Yurchenco is a pioneer in a novel gene therapy approach for the treatment of LAMA2-deficient congenital muscular dystrophy.
LAMA2-related muscular dystrophy causes weakness and atrophy of the skeletal muscles, which are used for movement. The condition varies in severity from a severe, early-onset type to a milder, late-onset form.
“Gene replacement is a promising therapeutic option for the treatment of LAMA2 MD. We have been working on continuously optimizing linker proteins engineered from extracellular matrix proteins which will aid in advancing such gene therapy approach towards clinical use,” said Yurchenco in a statement.
Yurchenco’s strategy uses two linker proteins that are composed of domains derived from extracellular matrix proteins agrin, laminin and nidogen. According to an announcement from Rutgers, the approach has led to the restoration of muscle fiber basement membranes in animal models and to the recovery of muscle force and size, increased body weight and markedly prolonged survival. The research demonstrates strong evidence for disease-modifying potential.
“Santhera is excited to extend its collaborative network for this therapeutic approach, now including experts from Rutgers University,” added Santhera’s Chief Medical Officer and Head of Development Dr. Kristina Sjöblom Nygren in a statement. “This will add value to our gene therapy program for LAMA2 MD and complements the work already underway with the Biozentrum at the University of Basel, which was awarded a grant by Innosuisse in 2019. Both of our collaboration partners have pioneered this field and will work closely with Santhera, clinical experts and the patient community to establish the best way to bring this approach to clinical use.”
Congenital muscular dystrophies are inherited neuromuscular diseases characterized by early-onset weakness and poor muscle tone, often alongside progressive muscle weakness, joint contractures and respiratory issues. Laminins are proteins that help maintain muscle fiber stability by anchoring to muscle receptors. LAMA2-related muscular dystrophy is one of the most common forms of congenital muscular dystrophy, caused by mutations in the LAMA2 gene encoding the alpha2 subunit of laminin-211.
The National Organization of Rare Disorders reports that approximately 250,000 people in the United States are affected by different kinds of muscular dystrophies.