SineuGene’s SNUG01 AAV Gene Therapy Granted FDA Orphan Drug Designation for ALS

SineuGene Therapeutics, a clinical-stage biotechnology company focused on gene therapies for neurological diseases, today 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: A First-in-Class AAV Gene Therapy for ALS

SNUG01 is a first-in-class gene therapy targeting TRIM72 (Tripartite Motif Containing 72), designed to provide broad neuroprotection in ALS, a rare and fatal neurodegenerative disorder affecting approximately 30,000 individuals in the United States. This AAV gene therapy, developed via SineuGene’s proprietary AAV technology platform, utilizes a rAAV9 vector to deliver the human TRIM72 gene to neurons via intrathecal administration.

Preclinical studies indicate that TRIM72 expression may counteract ALS pathogenesis through multiple mechanisms, including reducing oxidative stress, restoring mitochondrial homeostasis, and inhibiting neuroinflammatory cascades. An investigator-initiated trial (IIT) in China recently demonstrated SNUG01’s favorable safety and tolerability, along with early signs of clinical efficacy and biomarker improvement. Unlike ALS therapies targeting specific genetic mutations, SNUG01’s broad neuroprotective mechanism holds the potential to benefit over 90% of ALS patients with sporadic disease, a population currently lacking effective treatment options.

FDA Designation Paves Way for Clinical Development

The FDA’s Orphan Drug Designation provides SineuGene with significant incentives, including tax credits for clinical trials, exemption from BLA user fees, and potential eligibility for seven years of marketing exclusivity upon approval. This designation follows the FDA’s clearance of SNUG01’s Investigational New Drug (IND) application 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 in both the U.S. (including Massachusetts General Hospital) and China. PackGene is a proud partner in the development of SNUG01, providing comprehensive AAV vector services. These services encompass plasmid and AAV process development and optimization, GMP production, analytical method development and validation, quality research, product release testing, stability studies, and complete CMC (Chemistry, Manufacturing, and Controls) data compilation, all aimed at accelerating the market launch of SineuGene’s SNUG01 gene therapy product.

About ALS

ALS is a rapidly progressive neurodegenerative disorder characterized by motor neuron degeneration, leading to muscle weakness, atrophy, and eventually respiratory failure. Median survival is 3–5 years. Existing therapies offer limited benefits, slowing progression only marginally, with no curative options available.