Cure Rare Disease Receives FDA Approval to Administer First-in-Human CRISPR Therapeutic

The first-of-its-kind, CRISPR-based therapeutic aims to stop the progression of Duchenne muscular dystrophy in a single-patient dosing

Excerpt from the Press Release:

BOSTON–(BUSINESS WIRE)–Cure Rare Disease (CRD) – a Boston-based 501c3 nonprofit biotech – announces the approval from the U.S. Food and Drug Administration (FDA) to administer its very first therapeutic. The drug, named CRD-TMH-001, treats muscle promoter and exon 1 mutations on the dystrophin gene. With the IND approval process complete, the FDA has given the go-ahead, and dosing of the drug will occur imminently at UMass Chan Medical School.

The approval marks the culmination of a collaborative partnership, spanning a three-year period, with leading researchers, scientists and clinicians focused on developing break-through therapies for rare neuromuscular diseases, including the Lek Lab at Yale University, Charles River Laboratories and many other collaborators. The therapeutic will upregulate an alternate form (isoform) of the dystrophin protein using CRISPR technology with the goal of stabilizing, or potentially reversing, symptom progression of Duchenne muscular dystrophy

“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,” explains Richard Horgan, founder and CEO of Cure Rare Disease. “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.”

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