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Sarepta bags drug to fully unlock DMD gene therapy opportunity

July 2, 2020

Sarepta Therapeutics has secured a license to a drug that could enable more patients to receive its gene therapies. The deal grants Sarepta the exclusive right to use Hansa Biopharma’s imlifidase to eliminate neutralizing antibodies in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophy (LGMD) patients.

Many gene therapies, including those in development in DMD and LGMD at Sarepta, use a type of adeno-associated virus (AAV) to get the genetic material into cells. Some patients have preexisting antibodies against AAV, rendering them ineligible for treatment with gene therapies that rely on the vector. One study found 17% of patients have antibodies against Sarepta’s AAVrh74 vector.

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