15 October 2020 - - A patient has passed away in the AAVance clinical trial (NCT03612869), a global Phase 2/3 gene therapy trial for the treatment of Mucopolysaccharidosis Type IIIA (MPS IIIA, also known as Sanfilippo syndrome type A), French gene therapy platform company Lysogene (PAR: LYS) (FR0013233475 LYS) said.

MPS IIIA is a serious, life-threatening, inherited neurodegenerative lysosomal storage disorder characterized by intractable behavioral problems and developmental regression resulting in early death.

The immediate cause of death is currently unknown and additional information is being collected.

At this time, there is no evidence that the event is linked to the study drug administration.
Lysogene is diligently following per study protocol the 18 patients who have been treated in the clinical trial.

Lysogene is profoundly saddened by the passing of this child and extends its deepest sympathies to the family.

The company remains committed to the LYS-SAF302 development program and the Sanfilippo patient community.

The company plans to provide further information on the LYS-SAF302 program based on both ongoing data collection and future regulatory status updates.

Lysogene is a gene therapy company focused on the treatment of orphan diseases of the central nervous system.

The company has built a unique capability to enable a safe and effective delivery of gene therapies to the CNS to treat lysosomal diseases and other genetic disorders of the CNS.

A phase 2/3 clinical trial in MPS IIIA in partnership with Sarepta Therapeutics, Inc. is ongoing and a phase 1/3 clinical trial in GM1 gangliosidosis is in preparation.

In accordance with the agreements signed between Lysogene and Sarepta Therapeutics, Inc., Sarepta Therapeutics, Inc. will hold exclusive commercial rights to LYS-SAF302 in the United States and markets outside Europe; and Lysogene will maintain commercial exclusivity of LYS-SAF302 in Europe.

Lysogene is also collaborating with an academic partner on a gene therapy approach for the treatment of Fragile X syndrome, a genetic disease related to autism.