At Sarepta, we are committed to exploring every modality of precision genetic medicine that may help advance our mission to rescue lives otherwise impacted or stolen by rare disease. We believe gene editing, although early, has the potential to revolutionize the treatment of disease caused by genetic mutations.

 

gene editing

Gene Editing Technology Applied to Rare Diseases

Sarepta announced plans to build a Gene Editing Innovation Center (GEIC) in Durham, North Carolina. The GEIC will be led by a world-class scientific team made up of Louise Rodino-Klapac, PhD, Sarepta’s Senior Vice President of Gene Therapy and Charlie Gersbach, PhD, Director, Center for Advanced Genomic Technologies, Rooney Family Associate Professor of Biomedical Engineering, Director, Center for Biomolecular and Tissue Engineering, Duke University.

The GEIC will focus on developing gene editing therapies that aim to treat the majority of patients with Duchenne muscular dystrophy. Drs. Rodino-Klapac and Gersbach and their team will pursue a differentiated scientific approach that utilizes a proprietary dual cut strategy for predictable and accurate editing.

While collaborating with Sarepta, Dr. Gersbach will retain his professorship at Duke University. Sarepta has a separate collaboration underway with Duke University for gene editing and CRISPR-Cas9.