Five-year-old girl that was part of Lysogene’s gene therapy clinical trial dies

pharmafile | October 16, 2020 | News story | Medical Communications  

A five-year-old girl had died while taking part in Lysogene’s clinical trial investigating its gene therapy.

The child was suffering from Sanfilippo syndrome, which is a rare genetic disease that alters brain development and leads to premature death. Lysogene’s gene therapy is a treatment for central nervous system diseases in children.

The company released a statement saying that the immediate cause of death was unknown and there was no evidence that it was linked to the girl being administered the therapy. It added that the company was “profoundly sad” at the child’s passing and were collecting information surrounding the conditions of her death. The company is monitoring the other 19 participants in the trial closely. 

This follows the FDA previously placing the trials on hold due to finding “localised findings on MRI images at the intracerebral injection sites” which suggested “a potential connection to delivery.”

The company has signed a deal with Sarepta Therapeutics to give it exclusive commercial rights to the treatment in the US and outside of Europe. 

Conor Kavanagh

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