SineuGene Therapeutics, a clinical-stage biotechnology company focused on gene therapies for neurological diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its gene therapy, SNUG01, for the treatment of amyotrophic lateral sclerosis (ALS).

SNUG01 is a first-in-class gene therapy targeting TRIM72 (Tripartite Motif Containing 72), designed to provide broad neuroprotection in ALS, a rare, fatal neurodegenerative disorder affecting approximately 30,000 individuals in the United States.

FDA Orphan Drug Designation status is granted for treatments of rare diseases affecting fewer than 200,000 people in the U.S. This designation provides SineuGene with a range of development and commercial incentives, including tax credits for qualified clinical trials, exemption from BLA user fees, and eligibility for seven years of marketing exclusivity ("orphan exclusivity") upon approval.

The designation follows the FDA's clearance of the Investigational New Drug (IND) application for SNUG01 in March 2025. SineuGene plans to initiate a Phase I/IIa international, multicenter trial to evaluate the therapy's safety, tolerability, and preliminary efficacy in adults with ALS. The study will enroll participants at clinical sites across both countries, including Massachusetts General Hospital in the U.S., which will join other sites in China.