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

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via 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.

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