Girl Died in Clinical Gene Therapy Study Dies in the USA – 10/15/2020


The French biotechnology company Lysogene announced, on Thursday (15), the death of one of its patients – a five-year-old girl treated in the United States as part of a clinical study that evaluates a genetic treatment for a rare pediatric disease and incurable.

In a statement, the company stated that “the immediate cause of death is currently unknown” and that, “at this stage, there is no evidence that this event is related to the administration of the product” used in this international clinical study.

The patient is a 5-year-old girl who was not hospitalized at the time of her death. She had previously received treatment at one of Lysogene’s four clinical testing centers in the United States and returned home, the company told AFP.

The objective of the LYS-SAF302 clinical trial is to develop gene therapy for mucopolysaccharidosis type IIIA (MPS IIIA), a serious pediatric neurodegenerative disease of genetic origin. Still without a cure, she also known as Sanfilippo Syndrome type A.

To date, 18 patients have been treated in this clinical study, in Europe and the United States. The company said it was “deeply saddened by the death of this child” and remains “committed to the LYS-SAF302 program and the Sanfilippo patient community”.

The company added that it is gathering “additional information” and will conduct an investigation to determine the cause of death.


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