Lysogene’s Drug Candidate Receives Pediatric Disease Designation

The FDA granted rare pediatric disease designation to Lysogene’s drug candidate, LYS-GM101, for treatment of patients with GM1 gangliosidosis, a severe neurodegenerative disease.

LYS-GM101 has the potential to replace the defective gene in the cells of GM1 patients, which will allow for the production of the functional enzyme and prevent the progressive nature of the neurological damage caused by GM1.

If approved, Lysogene would be eligible to receive a transferable priority review voucher.