N-of-1 CRISPR trial ends with a death as nonprofit digs into what happened

04 Nov 2022
endpts.com
CollaborateGene TherapyAccelerated Approval
Almost two months after the nonprofit Cure Rare Disease received FDA clearance to administer its first-in-human CRISPR therapeutic for Duchenne muscular dystrophy, the nonprofit announced that the one patient in the trial, Terry Horgan, the brother of the nonprofit’s founder and CEO, Rich Horgan, passed away.
Researchers are still trying to figure out what exactly happened with the single shot CRISPR therapy, the nonprofit said in a statement, and that process may take up to four months.
The therapy, known as CRD-TMH-001, treats certain mutations on the dystrophin gene, with the goal of stabilizing or potentially reversing the progression of debilitating DMD symptoms. Dosing of the drug took place at the University of Massachusetts Medical School.
“The loss of any patient in any trial is immense loss for the family and for the caregiving team,” UMass Chan Medical School Dean Terence Flotte told Endpoints News via email. “This patient was very courageous, and we are all saddened by the outcome. CRD is working with a multidisciplinary, multi-institutional group of scientists to learn the most that can possibly be learned from this case, so as to inform the safe conduct of future gene therapies, whether they be for DMD or for other genetic diseases.”
Cure Rare Disease said in a statement that it will share the findings from the trial with the scientific community. The trial was launched after three years of development with partners including the Lek Lab at Yale University and contract research firm Charles River Laboratories.
A spokesperson with Cure Rare Disease also told the Associated Press that the nonprofit filed a report on the death with the FDA as required.
But the nonprofit isn’t the only one developing a gene therapy in DMD, and isn’t the only one that has run into issues.
Pfizer’s Phase Ib trial for its gene therapy for DMD was halted in 2021 after a young man in it died.
One of Pfizer’s Duchenne gene therapy trials put on hold in wake of patient death as high-dose AAV concerns still cloud field
In April, however, the trial hold was lifted and Pfizer opened its first US sites for a global Phase III trial assessing its gene therapy for DMD, known as fordadistrogene movaparvovec.
Sarepta and Roche are also working on their own gene therapy for DMD, and have applied with the FDA for an accelerated approval. The therapy, SRP-9001, initially failed a portion of a Phase II study in early 2021, but a second part showed patients had statistically significant improvements in motor abilities and later showed that motor function improved after one year.
Aiming for fourth nod, Sarepta files another DMD gene therapy to FDA; Axsome headed toward migraine resubmission
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