5-year-old girl treated in French gene therapy trial dies in the United States


The girl died at home several months after receiving the therapy (representation)

Paris, France:

A five-year-old girl with a rare neurodegenerative disease has died in the United States while participating in a gene therapy trial led by French biotechnology company Lysogene, the company said Thursday.

The little girl suffered from Sanfilippo syndrome or type III mucopolysaccharidosis, a rare genetic disorder that impairs brain development after birth and leads to premature death.

In a statement, Lysogene, a company developing gene therapy for diseases of the central nervous system in children, said that “the immediate cause of death is currently unknown” and that there was so far “no evidence that the event is related to the administration of the study drug “.

He said he was “deeply saddened by the death of this child” and was gathering “additional information” on the circumstances.

The company’s share price fell 19% to 2.05 euros in the morning session in Paris.

The girl was one of 19 people treated in the trial at eight hospitals in Europe and the United States.

She died at home several months after receiving the therapy, which consisted of a single injection, at one of four treatment sites in the United States, Lysogene told AFP.

In its statement, the company said it is closely monitoring the remaining 18 patients and remains “committed to the LYS-SAF302 development program.”

On June 5, the United States Food and Drug Administration (FDA) ordered a clinical suspension of the trial after observing “localized results on MRI images at intracerebral injection sites” suggesting “a potential link with the ‘delivery”.

In a statement at the time, the company said “no clinical symptoms have been observed that could be directly attributed to the MRI results observed.”

(Except for the title, this story was not edited by GalacticGaming staff and is posted from a syndicated feed.)


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