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Santhera Starts Collaboration in Gene Therapy Research for Congenital Muscular Dystrophy with the Biozentrum, University of Basel, Co-Financed by Innosuisse

Pratteln, Switzerland, May 21, 2019 – Santhera Pharmaceuticals (SIX: SANN) announces its collaboration with the Biozentrum of the University of Basel to advance gene therapy research for the treatment of LAMA2-deficient congenital muscular dystrophy (LAMA2 MD or MDC1A). The program is supported by public funding for innovation in Switzerland through a grant from Innosuisse – the Suisse Innovation Agency. Innosuisse and Santhera will jointly invest CHF 1.2 million into this preclinical research collaboration.

Santhera has entered in a collaboration with Prof. Markus Rüegg from the Biozentrum, University of Basel, who pioneered a novel gene therapy approach for the treatment of LAMA2 MD. The simultaneous transgenic expression of specifically designed small protein domains, so-called linker proteins, helps to overcome structural and functional loss of muscle fibers in a mouse model for LAMA2 MD, demonstrating strong preclinical evidence for disease modifying potential. This novel gene therapy strategy developed by Prof. Rüegg and coworkers will use two linker proteins that are composed of domains derived from extracellular matrix proteins agrin, laminin and nidogen [1-4]. 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 [1]. The preclinical research collaboration between Santhera and the University of Basel will explore the feasibility of gene delivery by standard viral vectors as a basis for subseq…

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