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Cure Rare Disease gets FDA approval to administer first-in-human CRISPR therapeutic

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The U.S. Food and Drug Administration has granted approval for Cure Rare Disease to administer its first-in-human CRISPR therapeutic, according to a press release.

The therapeutic, CRD-TMH-001, aims to stop the progression of Duchenne muscular dystrophy in single-patient dosing. The first patient is expected to be dosed at UMass Chan Medical School

“The success of this collaborative project and the development framework that CRD has built demonstrates there is a more efficient way to develop therapeutics for rare disease patients who were previously told that there was no hope for them,” said Richard Horgan, founder and CEO of Cure Rare Disease, in a press release. “This is just the beginning of CRD’s efforts to develop more therapeutics to treat rare and ultra-rare diseases, and we look forward to leveraging this approach to continue breaking down barriers in the drug development process for patients who need effective treatments now. As we celebrate this accomplishment, it is imperative that the US Centers for Medicare & Medicaid Services understand the financial hurdles that the rare disease community must overcome and consider the implementation of a reimbursement model to make this growing pathway accessible to more patients.”

Read the full press release here.

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