November 1, 2017


Sarepta jumps into the CRISPR fray with Duke gene-editing pact

[This story was originally posted on the Boston Business Journal

By   –  Biotech Reporter, Boston Business Journal

Cambridge-based Sarepta Therapeutics said Tuesday that it will seek to develop drugs for Duchenne muscular dystrophy using CRISPR/Cas9 gene-editing technology, jumping into an increasingly competitive but still uncertain field.

Sarepta (Nasdaq: SRPT) said it had reached an agreement to exclusively license technology related to CRISPR/Cas9 from Duke University. Researchers there, led by Dr. Charles Gersbach, have shown that the technology can be used in mice to remove mutated parts of genes, or exons, and thereby restore an important protein called dystrophin that is needed for muscle maintenance.

In September 2016, Sarepta became the first company to win FDA approval for a Duchenne treatment. That drug, called Exondys 51, works by skipping over a specific gene mutation that prevents the creation of dystrophin in about 13 percent of patients with the disorder.

The Cambridge company, like others in the Duchenne field, is now seeking to develop a drug that would help all patients regardless of their mutation. To that end, Sarepta announced in January that it would partner with Ohio-based Nationwide Children’s Hospital to pursue gene therapy treatments, which work by replacing a faulty gene with a corrected one. Gene editing, a promising but still largely unproven technique, offers another potential avenue.

Sarepta did not disclose financial terms of the agreement with Duke on Tuesday. Shares of the company were largely unchanged when markets opened.

In a statement, Sarepta CEO Douglas Ingram said that gene editing “has the potential to revolutionize the treatment of diseases with genetic mutations.”

“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,” Ingram said.

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