Sarepta Therapeutics Signs Exclusive Global Collaboration

Sarepta Therapeutics, Inc. recently announced it has signed a research collaboration agreement with Duke University, granting the company an option to an exclusive license to intellectual property and technology related to CRISPR/Cas9 technology developed in the laboratory of Charles A. Gersbach, PhD. The underlying premise of Dr. Gersbach’s approach is to restore dystrophin expression by removing or “excising” exons from the dystrophin gene. This includes a strategy to excise exons potentially enabling treatment for a majority of the DMD patient population. Sarepta will collaborate with Dr. Gersbach’s lab to advance the CRISPR platform and take the lead on clinical development.

“Gene editing has the potential to revolutionize the treatment of diseases with genetic mutations. We are particularly excited about the potential it holds for DMD patients,” said Douglas Ingram, Sarepta’s President and Chief Executive Officer. “We will work closely with Dr. Gersbach, a pioneer in applying the CRISPR technology to treat Duchenne, to advance a program that builds upon the established body of research by Dr. Gersbach and his team. Today’s agreement exemplifies our strategy of investing in and advancing a multi-faceted array of potential therapies for the largest number of individuals with DMD by leveraging our own research and development efforts, as well as forging external partnerships with the field’s best and brightest minds.”

“Although early, CRISPR technology represents hope for a large percentage of individuals with DMD. Excising certain exons has the potential to correct a majority of DMD mutations. Toward that goal, we’ve shown in mouse models that we can excise exons from the dystrophin gene, leading to restoration of a functional dystrophin protein and improvements in muscle strength,” said Dr. Gersbach, an associate professor in Duke University’s Department of Biomedical Engineering. “We are pleased to be partnering with Sarepta, a leader in the development of DMD therapies. The Company’s dedication to the patient community, and their goal to pursue a variety of scientific approaches, makes them an ideal partner for Duke and our team of researchers in pursuing our goal of translating the science into a treatment.”

The Gersbach Lab is dedicated to applying innovative methods in molecular and genetic engineering to advancing regenerative medicine, treating genetic disease, and enhancing our understanding of fundamental biological processes. In particular, the Gersbach Laboratory aims to develop new technologies to modify genome sequences, epigenomic regulation, and cellular gene networks in a precise and targeted manner. Examples of technologies used in Dr. Gersbach’s research include genome and epigenome editing with CRISPR/Cas9 and other DNA-targeting systems, protein engineering, directed evolution, genetic reprogramming, and optogenetics.

The work of Dr. Gersbach and his team falls within the larger field of genome editing for cell and gene therapy that can be applied to diverse diseases and disorders (Nelson, Robinson-Hamm, and Gersbach, Nature Reviews Neurology 2017). A primary example of Dr. Gersbach’s work in this area is developing genome editing methods such as CRISPR/Cas9 to correct mutations to the dystrophin gene that cause DMD. Dr. Gersbach and his team have used genome editing to correct mutations in patient cells and demonstrated restored dystrophin expression in patient cells in culture and after transplantation into skeletal muscle in mouse models (Ousterout et al., Molecular Therapy 2013; Molecular Therapy 2015; Nature Communications 2015). More recently, they have extended this work to correcting dystrophin mutations in vivo in mouse models of DMD (Nelson et al., Science 2016).

Duke University’s Pratt School of Engineering is a vibrant teaching and research institution dedicated to training the next generation of leaders and exploring the frontiers of engineering to develop solutions to societal challenges. Ranked among the top 20 engineering schools in the nation by U.S. News & World Report, the school includes highly interdisciplinary academic programs in biomedical engineering, civil and environmental engineering, electrical and computer engineering and mechanical engineering and materials science. With more than $70 million annually in research expenditures, Duke Engineering is home to major research centers focused on areas including biomolecular and tissue engineering, metamaterials, photonics, environmental implications of nanotechnology, materials science, materials genomics and quantum computing, among others.
Sarepta Therapeutics is a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying Duchenne muscular dystrophy (DMD) drug candidates. For more information, visit www.sarepta.com.