C3 is Driving Progress Towards a Cutting-Edge Cure

Coalition to Cure Calpain 3 (C3) is bringing cutting-edge science to drive progress toward a cure for limb-girdle muscular dystrophy type 2A (LGMD2A/R1). Some of our proudest work yet has been to help support and harness incredible new scientific understanding in genomics to bring an end to this devastating disease. In celebration of ten years of progress in our mission to fund research for a cure and raise global awareness, this week brings number nine in our top ten highlights: C3 is driving progress towards a gene therapy for LGMD2A/R1.

Gene therapy is at the vanguard of pharmaceutical science. It has shown tremendous promise as a potential curative treatment in numerous diseases caused by a single malfunctioning gene, including common forms of muscular dystrophy and other LGMD subtypes. We believe that gene therapy holds great promise to treat LGMD2A/R1, caused by a malfunctioning calpain 3 gene. In 2017, we launched the C3 Gene Therapy Initiative to bring this powerful new science to bear on LGMD2A/R1 and jumpstart clinical studies to advance potential therapies.

To-date, C3 has funded six gene therapy projects, committing over $700,000 towards the development of a gene therapy for LGMD2A/R1. These include some of the latest scientific approaches:

• AAV-mediated gene replacement therapy
• CRISPR-mediated gene editing
• Non-viral gene therapy

The C3 Gene Therapy Initiative has generated exciting results. Dr. Zarife Sahenk of Nationwide Children’s Hospital has led a groundbreaking LGMD2A/R1 AAV-mediated gene therapy program. Thanks in part to a C3 research grant in 2018, Dr. Sahenk’s work has begun testing AAV-mediated gene therapy approaches in an animal model.

In May 2019, Sarepta Therapeutics, Inc., a leader in muscular dystrophy and gene therapy research, announced that it signed an agreement with Nationwide Children’s Hospital giving Sarepta the exclusive option to Nationwide’s gene therapy candidate to treat LGMD2A/R1.

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C3 has also helped to build enthusiasm and knowledge in the space. In spring 2019, we convened the LGMD2A/R1 Gene Therapy Workshop, bringing together world-class researchers, clinicians, and regulators. Workshop findings are guiding grant reviews and associated investments in development up through an Investigational New Drug (IND) filing and are also informing opportunities to attract vital industry and venture capital.

And C3 serves as a scientific advocate engaging companies interested in developing gene therapies for LGMD2A/R1. We scan the horizon for more promising work in this field, hopeful that it has life-changing potential for people living with this disease.

To continue this important work, we need your help.C3 has a pinpoint focus: to drive research for an LGMD2A/R1 cure. Every dollar counts to help grow support and research for this overlooked subtype of muscular dystrophy.

Please donate now so that we can continue our strong momentum. Through your generous financial support we can do even more in the next 10 years to change the lives of patients in need.

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