Death of a 5-year-old girl treated with gene therapy

A girl treated with gene therapy in the United States dies.

(Illustration) -

A child treated in the United States as part of a clinical study evaluating gene therapy treatment has died, French biotech Lysogene announced Thursday.

The five-year-old girl suffered from a rare and incurable pediatric disease.

The company specifies in a press release that "the immediate cause of death is currently unknown" and that "at this stage, there is no evidence that this event is linked to the administration of the product" used in this international clinical study in phase 2/3 (on a three-phase testing procedure).

The little patient who was not hospitalized at the time of her death.

She had already received the treatment several months ago at one of Lysogene's four US clinical trial centers and had returned to her home, the company said.

The clinical suspension of the study ordered last June

The LYS-SAF302 clinical trial aims to develop a gene therapy for mucopolysaccharidosis type IIIA (MPS IIIA), which is a serious neurodegenerative pediatric disease, of genetic origin, currently incurable and also known as Sanfilippo's disease. type A.

On June 5, the American Medicines Agency (FDA) ordered the clinical suspension of the study after the observation on MRI images of "signals localized at intracerebral injection sites", suggesting a "potential link with 'administration of the product,' according to a company statement, which specified that 'no clinical symptoms can be directly attributed to these MRI observations'.

18 patients still followed

As of that date, 19 of the 20 patients in the study had received an injection of the product at one of the eight hospital sites where the company is conducting its trials in the United States and Europe.

No new treatment has been carried out since.

Lysogene said it is "deeply saddened by the death of this child" and maintains "its commitment to the LYS-SAF302 program and the Sanfilippo patient community."

The company continues to monitor the remaining 18 patients, which are "enough" to continue the study and develop a drug, according to the company, since the treatment consists of a single injection.

The company claims to be compiling "additional information" and will conduct an investigation to define the causes of death.

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• Child

• Genetic

• Disease

• Health

• United States