Only patient in N-of-1 CRISPR trial for Duchenne muscular dystrophy dies

Only patient in N-of-1 CRISPR trial for Duchenne muscular dystrophy dies

Published: Nov 07, 2022

By Alex Keown

BioSpace

A patient taking part in an FDA-sanctioned clinical trial assessing a CRISPR-based therapy for a rare form of Duchenne muscular dystrophy (DMD) has died.

Terry Horgan, the only patient in the CRD-TMH-001 trial of a novel CRISPR therapeutic, died last week. Horgan is the brother of the founder of Cure Rare Disease (CRD), a non-profit biotech that was spearheading the trial.

Few details related to the circumstances of Horgan’s death are known at this time, including whether or not he had been dosed with the one-time gene therapy.

In a brief statement issued last week, Cure Rare Disease announced the death of Horgan and said the details are being studied by multiple teams across the country. A review of the data could take up to four months before a cause of death is known, according to the nonprofit.

The trial was greenlit by the FDA in August. Horgan was slated to receive the CRISPR-based therapy, CRD-TMH-001, which is designed to treat muscle promoter and exon 1 mutations on the dystrophin gene. The goal of the therapy was to stabilize or potentially reverse the progression of symptoms associated with DMD. 

“The comprehensive work these teams are doing is critical to gaining a clear understanding of the outcome of the CRD-TMH-001 trial and to shedding additional light on the challenges of gene therapy broadly,” CRD stated in its announcement.

Horgan was expected to be dosed at UMass Chan Medical School. Trial protocols were to have him followed for up to 15 years to determine the length of efficacy. Following the administration of CRD-TMH-001, Horgan was also expected to remain at the hospital for monitoring in order to ensure there were no adverse reactions to the therapy.

When data is available, CRD noted it will share the findings from the CRD-TMH-001 trial with the scientific community. 

Sara Wiley, a communications manager with UMass Chan Medical School, told BioSpace the school will defer to CRD on information related to Horgan.

“UMass Chan respects the family’s wishes and Cure Rare Disease will be the only source of information for answering questions about this matter,” she said.

CRD was founded by Rich Horgan in order to find a treatment that would help his brother, who was first diagnosed with DMD in 1999. Duchenne is an X-linked degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. The disease affects one in 3,500 male births. It is rare in females.

DMD is associated with specific errors in the gene that codes for dystrophin, a protein that plays a key structural role in muscle fiber function. Muscles lacking dystrophin are more susceptible to damage. The condition is universally fatal, and death usually occurs before the age of 30.

Beyond DMD, CRD is working to advance 18 different therapeutics in its pipeline. The organization aims to develop therapies for rare genetic diseases, including different types of limb-girdle muscular dystrophy, ASSSL1 gene-related myopathy and spinocerebellar ataxia type 3. The only asset in CRD’s pipeline to reach the clinic is CRD-TMH-001. 

Source: BioSpace