Sarepta to tap into CRISPR treatment potential

Biopharmaceutical company Sarepta Therapeutics is exploring the potential of CRISPR/Cas9 gene editing technology to treat Duchenne muscular dystrophy (DMD).

The US firm, which specialises in developing cures for DMD, has agreed to license the IP rights covering DMD treatment through CRISPR/Cas9 technology from Duke University for an undisclosed sum.

Both parties declined to disclose the terms of the transaction, but Sarepta said in a statement that the licence has been issued on an exclusive basis.

A genetic condition, DMD is the result of an absence of the dystrophin protein which leads to deterioration of muscle strength.

According to the UK’s National Health Service it is more common in young males and people with the condition do not live beyond their thirties.

Dr Charles Gersbach, an associate professor in Duke University’s biomedical engineering department, has been conducting research on correcting mutations in the dystrophin gene that is the source of DMD mutation.

“The underlying premise of Gersbach’s approach is to restore dystrophin expression by removing or ‘excising’ exons from the dystrophin gene,” said Sarepta.

Douglas Ingram, chief executive of Sarepta, added:“Gene editing has the potential to revolutionise the treatment of diseases with genetic mutations.”

In July, the Broad Institute of MIT and Harvard announced it had entered talks to create a worldwide licensing pool for CRISPR/Cas9 patents.

The Broad said it hoped a patent pool would create a “one-stop shop” for commercial users to license CRISPR patents without needing to navigate a “complex patent and licensing landscape”.

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