Santhera Signs Agreements in Gene Therapy Research for Congenital Muscular Dystrophy with Rutgers University

Pratteln, Switzerland, May 06, 2020 – Santhera Pharmaceuticals (SIX: SANN) announces the signing of two agreements with Rutgers, The State University of New Jersey as part of its program to advance gene therapy research for the treatment of LAMA2-deficient congenital muscular dystrophy (LAMA2 MD or MDC1A). Under the agreements, Santhera gains rights to intellectual property developed at Rutgers on certain gene constructs that will be further studied under a collaboration agreement.

Santhera has entered into a license agreement with Rutgers, The State University of New Jersey and a collaboration with Prof. Peter Yurchenco, a pioneer in a novel gene therapy approach for the treatment of LAMA2 MD. These agreements complement the ongoing collaboration of Santhera with Prof. Markus Rüegg from the Biozentrum of the University of Basel [1]. Previous collaborative work by Prof. Rüegg and Prof. Yurchenco has established the potential of this approach in animal models.

The novel gene therapy strategy developed by these leading experts uses two linker proteins that are composed of domains derived from extracellular matrix proteins agrin, laminin and nidogen [2-5]. In animal models for LAMA2 MD, this approach has led to restoration of muscle fiber basement membranes, recovery of muscle force and size, increased overall body weight and markedly prolonged survival thus demonstrating strong evidence for disease modifying potential [2].

The coordinated work of both collaborations will further advance Santhera´s effort to bring this innovative gene therapy approach to patients with LAMA2 MD.

“Gene replacement is a promising therapeutic option for the treatment of LAMA2 MD,” said Peter D. Yurchenco, MD, PhD, Professor at Rutgers Robert Wood Johnson Medical School, USA. “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.”

“Santhera is excited to extend its collaborative network for this therapeutic approach, now including experts from Rutgers University,” added Kristina Sjöblom Nygren, MD, Chief Medical Officer and Head of Development of Santhera. “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, 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.”