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Lysogene Confirms Child’s Death in Phase II/III Gene Therapy Trial

A five-year-old girl with Mucopolysaccharidosis Type IIIA (MPS IIIA) has died in the Phase II/III AAVance clinical trial (NCT03612869), designed to evaluate the efficacy of Lysogene’s gene therapy candidate LYS-SAF302 in improving or stabilizing the neurodevelopmental state of MPS IIIA patients, the company has acknowledged.

In a statement, Lysogene said the immediate cause of death was unknown, that additional information was being collected, and that no evidence had surfaced linking the death to the administration of LYS-SAF302.

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