CRISPR Therapeutic Ready to Dose in Duchenne Muscular Dystrophy


A single patient will be dosed with CRD-THM-001 in an upcoming trial.

The FDA has given investigational new drug application (IND) clearance to Cure Rare Disease’s CRISPR therapeutic, CRD-TMH-001, for treating Duchenne muscular dystrophy (DMD).

Following the approval, a single-patient trial will begin dosing at UMass Chan Medical School. Chan, in collaboration with the Lek Lab at Yale University, Charles River Laboratories, and other researchers, scientists, and clinicians, developed the therapy as part of a 3-year partnership.

“I’m pleased to share that the US FDA has approved our IND application to treat our first patient Terry. We received word just recently and will move into clinical trials in a couple of days, first with immune suppression and finally with administering of this first in human CRISPR therapeutic,” Richard Horgan, founder and chief executive officer, Cure Rare Disease, said in a video. “We’ve done more than develop a drug, we've developed a new framework by which we can treat and develop drugs for other rare and ultra-rare diseases that have no treatment.”

WATCH NOW: Craig M. McDonald, MD, on Safety and Efficacy of DMD Cell Therapy

CRD-TMH-001 is designed to upregulate an alternate isoform of the dystrophin protein with CRISPR technology, addressing muscle promoter and exon 1 mutations in the dystrophin gene. Cure Rare Disease believes the therapy has the potential to stabilize or even reverse DMD progression.

“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,” Horgan added to the statement. “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.”

In the trial, the participants will receive a single dose of CRD-THM-001 intravenously and will be monitored in the hospital for several days for serious adverse events (AEs). The patient will then be followed up for 15 years for safety and efficacy.

"It is inspiring to see Cure Rare Disease achieve FDA approval for the first-in-human IND using CRISPR-transactivator technology to treat a rare mutation causing Duchenne muscular dystrophy. Learnings from this significant accomplishment will undoubtedly benefit other rare disease patients and we look forward to continuing to collaborate with CRD in the future to help patients. We wish both the patient and family the best for the upcoming clinical trial, " Terence Flotte, MD, Dean, Provost & Executive Deputy Chancellor, UMass Chan, added to the statement.

Cure Rare Disease Receives FDA Approval to Administer First-in-Human CRISPR Therapeutic. News release. Cure Rare Disease. August 10, 2022.
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