The US Food and Drug Administration (FDA) approved the administration of a first-in-human clustered regularly interspaced short palindromic repeats (CRISPR) therapeutic to treat Duchenne muscular dystrophy (DMD). 

The therapeutic, called CRD-TMH-001, is developed by Cure Rare Disease (CRD). It targets mutations in the promoter region and exon 1 of the DMD gene. The aim is to stabilize or reverse the progression of the disease by upregulating a dystrophin protein isoform.

“This is just the beginning of CRD’s efforts to develop more therapeutics to treat rare and ultrarare 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,” Richard Horgan, founder and chief executive officer of CRD, said in a press release.

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One patient will be given the therapeutic as an intravenous injection at the University of Massachusetts Chan (UMass Chan) Medical School in Worcester and will be monitored in the hospital for several days for any uncontrolled adverse events. The patient will then be followed for 15 years.

Read more about the etiology of DMD

“It is inspiring to see Cure Rare Disease achieve FDA approval for the first-in-human [investigational new drug] using CRISPR-transactivator technology to treat a rare mutation causing Duchenne muscular dystrophy,” Terence Flotte, MD, dean, provost, and executive deputy chancellor at UMass Chan, said.

According to the press release, the company will not be able to comment on the trial once it begins, to protect patient confidentiality.

DMD is a rare disease caused by mutations in the gene encoding the dystrophin protein. Dystrophin is essential for the health of muscle tissues, and mutations causing no functional dystrophin protein to be produced by the body lead to progressive muscle weakness and wasting. Patients eventually lose the ability to walk and need a ventilator to breathe in the later stages of the disease.

Reference

Cure Rare Disease receives FDA approval to administer first-in-human CRISPR therapeutic. News release. Cure Rare Disease; August 10, 2022.

CRD-TMH-001 Cure Rare Disease Duchenne Muscular Dystrophy Researchers