RARE Daily

Cure Rare Disease Looks for Answers and Will Share Findings after Death in N-of-1 CRISPR Trial

November 7, 2022

Marie Daghlian

Terry Horgan, brother of Cure Rare Disease Founder and CEO, Rich Horgan, recently passed away while participating in the N-of-1 clinical trial of a first-in-human CRISPR therapeutic to treat Duchenne muscular dystrophy, the nonprofit reported.

Duchenne muscular dystrophy is a rare, genetic muscle-wasting disease caused by a mutation in the gene that codes for production of the protein dystrophin. It impacts all muscle types, leading to loss of mobility and eventual respiratory and cardiac failure. The disease affects 1 in 3,500 male births and is extremely rare in females.

The U.S. Food and Drug Administration had given the go-ahead to administer the drug, named CRD-TMH-001, in August. CRD-TMH-001, treats muscle promoter and exon 1 mutations on the dystrophin gene. It was developed through a collaborative project under Cure Rare Disease’s commitment to advance therapeutics for rare disease patients who had been previously told that there was no hope for them.

The therapeutic was developed for one-time administration with the patient followed for a period of 15 years to track progress. In order to protect patient confidentiality, CRD said it would be unable to comment on the trial once it begins.

Terry Horgan died in mid-October, two months after FDA approved the trial, which was seen as a significant accomplishment that could benefit other rare disease patients.

 In a statement to the CRD community, the nonprofit said details of the trial are currently being studied by multiple teams and it could take up to four months to gain a clear understanding of the outcome of the CRD-TMH-001 trial and to shedding additional light on the challenges of gene therapy broadly.

“Cure Rare Disease was founded with a commitment to transparency and collaboration as we believe both are critical to moving science forward for rare disease patients. To that end, we intend to share the findings from the CRD-TMH-001 trial with the scientific community, not only to support the continued advancement of the additional 18 therapeutics in the CRD pipeline, but also to further the understanding of AAV-based gene therapies and applications among the larger communities for the treatment of other rare diseases,” wrote CRD. 

Photo: Terry and Rich Horgan, founder and CEO of Cure Rare Disease

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