TRIM72

BEIJING, China I March 24, 2025 I SineuGene Therapeutics Co., Ltd. (“SineuGene”), a clinical-stage biotech company pioneering innovative therapies for neurological disorders, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for SNUG01 — a first-in-class TRIM72 (Tripartite Motif Protein 72)-targeted gene therapy candidate for amyotrophic lateral sclerosis (ALS). The clearance authorizes a global Phase I/IIa clinical trial designed to evaluate SNUG01’s safety, tolerability, and preliminary efficacy in adults with ALS through a dose-escalation and expansion study. 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 post-diagnosis 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. SNUG01 leverages groundbreaking research carried out by Dr. Yichang Jia’s laboratory at Tsinghua University, whose team identified TRIM72 as a multifunctional neuroprotectant. The therapy utilizes a recombinant adeno-associated virus serotype 9 (rAAV9) capsid to deliver the human TRIM72 gene via intrathecal administration. Preclinical studies demonstrate TRIM72’s ability to counteract ALS pathogenesis through several synergistic mechanisms such as 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 In an investigator-initiated trial (IIT) at Peking University Third Hospital, SNUG01 demonstrated a favorable safety and tolerability profile, along with early efficacy signals in both functional clinical assessments and key biomarkers of neurodegeneration. These results enhance the translational validation of TRIM72, bridging target discovery, preclinical models, and human proof-of-concept. Unlike mutation-targeting gene therapies, SNUG01 leverages multiple neuroprotective mechanisms, offering a critical advantage for over 90% of ALS patients with sporadic (non-familial) disease. This unique multi-target approach positions SNUG01 as a first-in-class candidate for the majority of ALS cases, addressing an urgent unmet need in this underserved population. SineuGene will partner with a leading global consortium of academic and clinical institutions to conduct a multi-regional clinical trial, expediting validation of SNUG01’s safety and therapeutic efficacy profile across a diverse ALS patient populations. About SineuGene SineuGene Therapeutics Co., Ltd., often referred to as “SineuGene,” is a biotechnology company focused on developing innovative therapies for neurological diseases. Established in late 2021, the company builds on over a decade of scientific research conducted by Dr. Yichang Jia’s team from School of Medicine, Tsinghua University. SineuGene uses advanced technologies such as adeno-associated virus (AAV) vector-mediated gene delivery and editing and antisense oligonucleotide (ASO)- based gene expression regulation. The company has created a diverse pipeline that targets major neurological diseases, including Amyotrophic Lateral Sclerosis (ALS), stroke, Parkinson’s disease, Alzheimer’s disease, Spinocerebellar Ataxia type 3, and Huntington’s disease. SineuGene is dedicated to transforming groundbreaking research into effective treatments, aiming to meet the unmet medical needs in the field of neurology. Website: www.sineugene.com SOURCE: SineuGene Therapeutics