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Duchenne gene therapy trial halted after serious reaction

18 November 2019
Appeared in BioNews 1024

A gene therapy trial for Duchenne muscular dystrophy (DMD) has been halted after a patient experienced serious side effects.

The clinical trial, run by the US life science company, Solid Biosciences, was put on hold by the FDA (Food and Drug Administration) after the patient had an adverse reaction in response to the experimental gene therapy, called SGT-001. The symptoms included a decrease in red blood cell count, acute kidney injury, over-activation of the immune system and reduced heart and lung function.

'We are encouraged that this patient is recovering,' said Ilan Ganot, chief executive officer and co-founder of Solid Biosciences in Cambridge, Massachusetts. 'In the coming weeks, we anticipate that we will have a better understanding of the biological activity and potential benefit of SGT-001.'

DMD is a severe muscle-wasting disease that affects around one in every 3500 newborn boys. The disease is caused by a mutation in the gene responsible for making an essential muscle protein, called dystrophin. The resulting deficiency or absence of dystrophin leads to a progressive decline in muscle strength. There is currently no cure for the disease. 

To date, six patients in the IGNITE DMD clinical trial, taking place at the University of Florida, have received a one-off intravenous infusion of SGT-001. The treatment is an adeno-associated virus (AAV)-based gene therapy, in which an inactivated virus is designed to deliver a synthetic, functional form of dystrophin, called microdystrophin, to muscles.

The first three patients, aged between four and 17 years, were given the lowest dose outlined in the study protocol. A second cohort of three patients subsequently received a higher dose, which was believed to have led to the adverse reaction reported to the FDA.

The trial was previously halted in March 2018, after a patient receiving the low dose of the therapy experienced a similar reaction, from which he later recovered. The trial was resumed in June of the same year after the study design was amended. 

Solid Biosciences have reported that all five other patients dosed in the trial are doing well and continue to be examined. They added that they will work with the FDA to determine the next steps for the trial, including how best to manage administration of the therapy.

'We remain committed to bringing meaningful new therapies to the Duchenne community and continue to believe in the differentiated construct of SGT-001 and the potential benefits it may offer to patients,' Ganot said.

Patient's 'serious' kidney injury halts gene therapy trial for Duchenne muscular dystrophy
Genetic Literacy Project |  15 November 2019
Solid Bio falls as FDA halts DMD trial over safety concerns
PMLiVE |  13 November 2019
Solid Biosciences’ Duchenne gene therapy trial halted after patient suffers serious toxicity
STAT |  12 November 2019
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