BEIJING, June 26, 2025 /PRNewswire/ -- 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.

About SNUG01

SNUG01, developed by SineuGene via its proprietary AAV technology platform, is a first-in-class gene therapy product using TRIM72 as the gene of interest (GOI). The TRIM72 was identified as a potential therapeutic gene based on foundational research from Dr. Yichang Jia's lab at Tsinghua University. SNUG01 utilizes a rAAV9 vector to deliver the human TRIM72 gene to neurons via intrathecal administration. Preclinical studies have shown that TRIM72 expression may counteract ALS pathogenesis through multiple mechanisms, including reducing oxidative stress by scavenging reactive oxygen species (ROS), restoring mitochondrial homeostasis, suppressing stress granule dysregulation, inhibiting neuroinflammatory cascades, and enhancing neuronal membrane repair capacity. This multi-dimensional action delays motor neuron degeneration in ALS.

A recently completed investigator-initiated trial (IIT) in China demonstrated SNUG01's favorable safety and tolerability profile, alongside early signs of clinical efficacy and biomarker improvement. Crucially, 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. These promising results support its continued global development.

About ALS

ALS is a rapidly progressive neurodegenerative disorder marked by motor neuron degeneration in the brain and spinal cord, leading to muscle weakness, atrophy, and eventually respiratory failure. Median survival time is 3–5 years. Despite being the most prevalent adult-onset motor neuron disease, existing therapies offer limited clinical benefits, slowing progression only marginally and leaving no curative options for patients.

About SineuGene

SineuGene Therapeutics Co., Ltd. ("SineuGene") is a clinical-stage biotech company pioneering innovative therapies, including AAV-based and nucleic acid-based treatments, for neurological disorders. Founded in late 2021, the company's initial pipeline was built upon foundational research led by Dr. Yichang Jia at Tsinghua University's School of Medicine. SineuGene leverages cutting-edge technologies, such as adeno-associated virus (AAV)-mediated gene delivery and antisense oligonucleotide (ASO)-based gene regulation, to develop a robust pipeline targeting central nervous system disorders including ALS, stroke, Parkinson's disease, Alzheimer's disease, Multiple System Atrophy, Spinocerebellar Ataxia type 3, and Huntington's disease. SineuGene is committed to translating scientific discoveries into transformative therapies for patients with serious unmet medical needs.

Website: www.sineugene.com  
Contact: Ms YAN Siying, yansiying@sineugene.com