The girl who was 5 years old, died in the US during the French Gene Therapy Trial. The little girl had been suffering from Sanfilippo syndrome or mucopolysaccharidosis type III that is a rare genetic disease that changes brain development after birth and causes premature death. On Thursday A five-year-old girl with a rare neurodegenerative disease has died in the US while taking part in a gene therapy trial which is run by French biotechnology company Lysogene. Lysogene is a company that develops gene therapy for central nervous system diseases in children.
The firm Lysogene said in a statement that “the immediate cause of death is still unknown” and that there was “no evidence that the event is linked to the study drug administration”. The firm further said it was ” saddened by the passing of this child” and busy in “collecting additional studies” about the circumstances. The death of this child has affected the share price of the company. The share price of the company in Paris has dropped 19 per cent to 2.05 euros in morning trading.
Reason Behind The Little Girl Death
The reason for her immediate death is still unknown to the company. The girl was one of 19 people who was being treated in the trial conducted at eight hospitals in Europe and the United States. Lysogene told that she had died at home several months after completing the therapy that is consisting of a single injection, at one of four treatment sites in the US,
The company said in its statements that it was looking the remaining 18 patients closely and remained “committed to the LYS-SAF302 development program”. On June 5, the US Food and Drug Administration (FDA) requested a clinical hang on the preliminary after watching “limited discoveries on MRI pictures at the intracerebral injection destinations” recommending “a possible association with conveyance”