Developing Gene Therapies for Indications With Unmet Needs


The chief executive and scientific officers of Lysogene discussed Lysogene’s patient-focused therapy development.

“We started our pipeline with a Sanfilippo-focused program, because I had a child affected with this syndrome. Back then, there were no programs around for this disease. That was the genesis of Lysogene. Later on, based on what we had learned on the Sanfilippo or MPS type 3 program, we decided to apply our knowledge and expertise to other indications.”

Lysogene is focused on developing gene therapies for neurodegenerative lysosomal storage disorders. This mission 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, LYS-SAF302, is being evaluated in the phase 2/3 AAVance trial (NCT03612869), which has enrolled 20 participants with MPS type 3A. The trial has previously reported reductions in cerebrospinal fluid sulfate heparin levels as well as GM2 and GM3 ganglioside compared to baseline. Another program, LYS-GM101, is being evaluated in a phase 1/2 clinical trial (NCT04273269) that dosed its first participant with GM1 gangliosidosis in August 2021.

GeneTherapyLive spoke with Aiach, who also serves as chair and chief executive officer, to learn more about Lysogene and its patient-focused therapy development. Ralph Laufer, PhD, chief scientific officer, also discussed the advantages of Lysogene’s AAV delivery methods.

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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