Man first in line for personalized CRISPR treatment dies

Few details are available about Horgan’s death. It is unclear when or even if he received the experimental treatment. A spokesperson for the UMass Chan School of Medicine deferred all questions to cure a rare disease. A spokesman for the organization says that no further information is being provided at this time. Richard Horgan did not respond to requests for comment.

The Associated Press first reported the death on Friday. Cure Rare Disease posted a statement on its website earlier this week confirming the death. The organization said “multiple teams across the country” are working to understand what happened, but warned it “could take up to four months” to find answers.

Richard Horgan founded the nonprofit in 2017 when he was a student at Harvard Business School. His goal was to find a cure for his brother’s form of Duchenne muscular dystrophy, which is caused by a mutation that blocked his ability to make an important muscle protein called dystrophin.

Terry was already too old to participate in clinical trials for experimental treatments for the disease, which often target young boys.

By 2019, his brother had assembled a team of researchers at Charles River Laboratories, UMass Chan Medical School, Yale University and other institutions to help design a customized treatment for Terry. The treatment used a new version of CRISPR to turn on a backup copy of the dystrophin gene that mysteriously lies dormant in our muscles.

After more than three years of creating the treatment, testing it in the lab and manufacturing it, the U.S. Food and Drug Administration gave the group permission to administer the treatment to Terry this summer. Soon after Cure Rare Disease announced the positive news in August, Richard Horgan told the Globe that his brother was “soon” to receive the treatment.

Although several companies are developing treatments based on CRISPR gene editing technology, and early data suggest they may be safe and effective, there are no approved drugs based on the approach.

The clinical trial was supposed to be the first time anyone received gene-editing therapy for muscular dystrophy. This was the first time that gene editing therapy was tailored to address a single individual’s unique genetic mutation. And it was the first time a new version of CRISPR technology, commonly known as epigenome editing, was tested on humans.

Epigenome editing aims to tune gene expression up or down. Many researchers hope that the method will prove to be a safer alternative to gene editing treatments that make permanent changes to the DNA itself. It is unclear if this new technology played a role in Terry’s death.

One aspect of the treatment that wasn’t entirely new was the use of engineered viruses to deliver the instructions for the gene-editing therapy into Terry’s cells. Like many gene therapy companies, Cure Rare Disease packaged the treatment into adeno-associated viruses. High doses of gene therapies based on these viruses have been linked to deaths in other experimental gene therapies.

Experts not involved in the study have also cautioned that Terry was much older than most participants in MD drug trials.

Monkel Lek, a scientist at the Yale School of Medicine who designed the treatment, and Dr. Brenda Wong, a physician specializing in muscular dystrophy at UMass Chan Medical School who oversaw the clinical trial, did not respond to requests for comment.

Cure Rare Disease said in a statement that it was committed to sharing its findings with other researchers and that it would continue to work on 18 more experimental treatments in the pipeline.

Ryan Cross can be reached at [email protected]. Follow him on Twitter @RLCscienceboss.

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