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Linking up against LAMA2 MD
July/August 2020
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PRATTELN, Switzerland—Santhera Pharmaceuticals announced two agreements with Rutgers, The State University of New Jersey earlier in the second quarter focused on accelerating gene therapy research to treat LAMA2-deficient congenital muscular dystrophy (LAMA2 MD or MDC1A). Per the agreements, Santhera gained rights to IP developed on Rutgers for gene constructs that will undergo further investigation in a collaboration agreement. In animal models, the gene therapy approach in question restored muscle fiber basement membranes, led to recovery of muscle force and size, increased overall body weight, and extended survival times.
 
“Gene replacement is a promising therapeutic option for the treatment of LAMA2 MD,” said Dr. Peter D. Yurchenco, professor at Rutgers Robert Wood Johnson Medical School. “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.”

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