Lysogene has just announced that the FDA has approved their Investigational New Drug Application (IND) for a therapy for GM1 gangliosidosis, a rare pediatric condition. The investigational treatment is a gene therapy called LYS-GM101. It is the company’s second ever central nervous system gene therapy to begin a global clinical trial. This therapy is an adeno-associated viral vector.
This therapy has already received Orphan Drug Designation from the EU and Rare Pediatric Disease Designation from the FDA.
Additionally, Lysogene has already received clinical trial authorization by MHRA in the UK. The company plans to begin its global, multi-center, two-stage, single-arm trial in the first half of this year. This trial will include 16 patients who have early or late infantile GM1 gangliosidosis. This investigation will have both a safety phase and an efficacy phase. The trial number is NCT04273269.
The company is also sponsoring a natural history study for the disease run by Casimir Trials. This study will collect videos of patients living with the disease in order to assess how children function with the disease. The trial number for this investigation is NCT04310163 if you would like to learn more.
Other Things in the Works
Lysogene is currently working on therapies for a few other rare diseases. For instance, a Phase 2/3 trial for MPS IIIA is ongoing. Additionally, they are collaborating in the development of a therapy for Fragile X syndrome.
The company is also preparing to initiate an adaptive trial for GM1 gangliosidosis.
You can read more about this investigative therapy here.