DMD drug developers stake out territory in rare disease market
This article was originally published in Scrip
Parents of boys with Duchenne muscular dystrophy (DMD) have had little good news during the past few months due to setbacks for Prosensa and Sarepta Therapeutics, but three different biotechnology company presentations at the 32nd Annual JP Morgan Healthcare Conference offered signs of hope for therapies that may keep their sons from losing the ability to walk, eat and breathe on their own.
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Several companies have DMD drug candidates waiting in the wings to follow Sarepta’s Exondys 51 into the commercial market after FDA approval of the exon-skipping therapy, which could be the backbone for future combination treatment regimens.
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The start-up is built around four business units – technology, manufacturing, R&D and therapeutics – based on internal and external expertise. Kriya will take its first gene therapies into the clinic in 2023.