Gene Therapy for GM1 Gangliosidosis: Latest Updates


Ralph Laufer, PhD, chief scientific officer, Lysogene, discussed gene therapy programs in GM1 gangliosidosis and Fragile X syndrome.

"In early 2021 we obtained approvals from the MHRA in the United Kingdom, the FDA, and the ANSM in France to start the clinical trial with LYS-GM101 in patients which GM1 gangliosidosis. The trial is an open-label, stage adaptive clinical trial with natural history data and external control, which we are conducting at 4 clinical sites: 2 in the US, 1 in France and 1 in the UK. We are conducting, in parallel, a video outcome and parent interview study whose data will be complementary to the clinical endpoints."

Lysogene’s mission of developing effective gene therapies for neurodegenerative lysosomal storage disorders is personal for founder Karen Pignet-Aiach, whose own child was born with mucopolysaccharidosis type 3 (MPS type 3), also known as Sanfilippo syndrome.

Lysogene’s lead program for Sanfilippo syndrome, dubbed LYS-SAF302, is being evaluated in the phase 2/3 AAVance trial (NCT03612869), which has reported positive preliminary data in 20 participants. Another candidate in development is LYS-GM101 for the potential treatment of GM1 gangliosidosis, which is being evaluated in a phase 1/2 clinical trial (NCT04273269). The trial dosed its first participant in August 2021 out of an expected 16.

GeneTherapyLive spoke with Ralph Laufer, PhD, chief scientific officer, Lysogene, to learn more about LYS-GM101, its mechanism of action, and positive preclinical data. Laufer also discussed a new program in Fragile X syndrome in which Lysogene is partnering with SATT Conectus and the Institute of Genetics and Molecular and Cellular Biology.

Lysogene announces first patient in the United States dosed with LYS-GM101 investigational gene therapy for the treatment of GM1 gangliosidosis. News release. Lysogene. August 30, 2021.
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