C3 Awards Gene Therapy Initiative Grant to Dr. Zarife Sahenk

FOURTH GENE THERAPY INITIATIVE GRANT SUPPORTS PROMISING RESEARCH AT NATIONWIDE CHILDREN’S HOSPITAL

Coalition to Cure Calpain 3 (C3) is excited to announce that a new research grant has been awarded to Dr. Zarife Sahenk, Professor of Pediatrics and Neurology at Nationwide Children’s Hospital, to assess the safety and efficacy of adeno-associated viral (AAV)-mediated gene therapy in a mouse model of calpainopathy (also called limb girdle muscular dystrophy type 2A, or LGMD2A). This is the fourth project selected for funding as a part of C3’s Gene Therapy Initiative, which seeks to accelerate the understanding of gene therapy approaches for calpainopathy/LGMD2A.

SAHENK TO FOCUS ON “PRE-CLINICAL STUDIES FOR GENE THERAPY IN CALPAINOPATHY”

Dr. Sahenk, who is the Director of the Clinical and Experimental Neuromuscular Labs and a principal investigator, developed a gene therapy program for Charcot-Marie-Tooth disease at Research Institute, Center for Gene Therapy, Nationwide Children’s Hospital, where she also contributed to animal studies and gene therapy trials for Duchenne Muscular Dystrophy and other subtypes of LGMD. She recently demonstrated that LGMD2A is associated with impaired muscle regeneration after injury, and that this defect can be corrected by injection of the muscle with AAV attached to the calpain 3 gene. In the current project, “Pre-clinical studies for gene therapy in LGMD2A,” Dr. Sahenk will deliver the calpain 3 gene throughout the body of a mouse model for LGMD2A to test the safety and efficacy of this method, and to provide proof of principal data for a potential calpain 3 gene therapy for LGMD2A patients.

C3 Scientific Director Dr. Jennifer Levy explains, “There is extraordinary potential in gene therapy approaches for treating LGMD2A. C3 is supporting Dr. Sahenk’s project to test the effectiveness and safety of AAV-mediated delivery of the calpain 3 gene in a mouse model of LGMD2A. Results of this study, if positive, will provide proof-of-principle data for a calpain 3 gene therapy approach in LGMD2A patients.”

Dr. Sahenk notes, “We are very excited to start on this project with the support of C3. Our goal is to develop a strategy to offer gene therapy safely and effectively to patients with LGMD2A. We hope that these studies will pave the road to successful gene therapy clinical trials for this form of muscular dystrophy.”

LEARN MORE ABOUT THE C3 GENE THERAPY INITIATIVE

The C3 Gene Therapy Initiative seeks to accelerate the understanding of gene therapy as a treatment for LGMD2A by funding the research of multiple gene therapy approaches. If results from any current or future Gene Therapy Initiative research projects are positive, they can be progressed towards the initiation of clinical trials for LGMD2A patients. Bringing a therapy from the research lab to a clinical trial is a long and expensive process. C3 is currently funding early stage projects to answer critical questions about effectiveness and safety, which must be established before gene therapy can be tested in patients. In addition to gene therapy, C3 will continue to explore and fund other approaches that could lead to therapies that can slow the progression of the disease or cure LGMD2A.

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THANKS TO GENEROUS SUPPORTERS LIKE YOU!

C3 Awards Gene Therapy Initiative Grant to Dr. Zarife Sahenk

Coalition to Cure Calpain 3

C3 Awards Gene Therapy Initiative Grant to Dr. Zarife Sahenk

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