Santhera and Rutgers Partner on Gene Therapy Work for LAMA2 MD

Santhera Pharmaceuticals announced the signing of two agreements with Rutgers University related to the development of potential gene therapies for LAMA2-deficient congenital muscular dystrophy (LAMA2 MD).

Included are a license agreement as well as a collaboration with Peter Yurchenco, MD, PhD, a professor at Rutgers’ Robert Wood Johnson Medical School with gene therapy expertise. Under the terms of the agreements, Santhera gains intellectual property rights to certain gene therapy constructs developed at the university.

LAMA2 MD — also called congenital MD type 1A — is caused by mutations in the gene LAMA2, which codes for the laminin-alpha 2 protein required to build muscle fibers. As a result, very early in life LAMA2 MD patients experience problems in the extracellular matrix (ECM) — the protein network that provides structural and biochemical support to cells and tissues.

Yurchenco’s research has focused on the use of two linker proteins to make a gene therapy more effective. Linker proteins are short chains of amino acids (the building blocks of proteins) that help to space out larger protein domains to prevent unwanted interactions. Specifically, the team’s linker proteins contain portions derived from ECM proteins known as agrin, laminin, and nidogen.

The partnership complements an ongoing collaboration between Santhera and Markus Rüegg, PhD, a professor at the Biozentrum of the University of Basel, in Switzerland.

In mouse models of LAMA2 MD, prior work by Yurchenco, Rüegg and their colleagues showed that this approach corrected ECM deficits, increased muscle force and size, increased body weight, and extended lifespan by more than five times compared to untreated mice.

“Gene replacement is a promising therapeutic option for the treatment of LAMA2 MD,” Yurchenco said in a press release. “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.”

Added Kristina Sjöblom Nygren, MD, Santhera’s chief medical officer and head of development: “Santhera is excited to extend its collaborative network for this therapeutic approach, now including experts from Rutgers University.”

“This will add value to our gene therapy program for LAMA2 MD and complements the work already under way with the Biozentrum at the University of Basel,” she added.