Gene Therapy for Sanfilippo Syndrome: Latest Updates


Ralph Laufer, PhD, chief scientific officer, Lysogene, discussed LYS-SAF302, its mechanism of action, trial data, and further research.

“Preliminary biomarker findings indicate that LYS-SAF302 is biologically active in this patient population, and has the potential to provide a therapeutic benefit... The [AAVance] trial will complete in mid-2022 when all patients in the main cohort have reached 2 years of treatment. In parallel with the trial, we are also carrying out a video study of the patients that captures real-life behaviors in their home environment.”

Developing effective gene therapies for neurodegenerative lysosomal storage disorders is personal for Lysogene 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). The trial has enrolled 20 participants with MPS type 3A and previously reported reductions in cerebrospinal fluid sulfate heparin levels as well as GM2 and GM3 ganglioside compared with baseline. The company is also developing 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.

GeneTherapyLive spoke with Aiach and Ralph Laufer, PhD, chief scientific officer, Lysogene, to learn more about LYS-SAF302, its mechanism of action, data from previous trials assessing the gene therapy, and further research being conducted with the therapy including a real-world experience video study.

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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