SineuGene Therapeutics Co., Ltd. has received U.S. FDA clearance for its Investigational New Drug (IND) application for SNUG01, a first-in-class TRIM72-targeted gene therapy for amyotrophic lateral sclerosis (ALS). This clearance allows for a global Phase I/IIa clinical trial to evaluate SNUG01’s safety, tolerability, and preliminary efficacy in adult ALS patients through dose escalation and expansion.
ALS, a progressive neurodegenerative disease with a median survival of 3-5 years post-diagnosis, currently lacks effective treatments. SNUG01, developed from research at Tsinghua University, utilizes an rAAV9 capsid to deliver the human TRIM72 gene intrathecally. Preclinical studies suggest TRIM72’s potential to counteract ALS through multiple mechanisms, including reducing oxidative stress, restoring mitochondrial homeostasis, and inhibiting neuroinflammation.
An investigator-initiated trial at Peking University Third Hospital demonstrated SNUG01’s safety and early efficacy signals in clinical assessments and neurodegeneration biomarkers. These results validate TRIM72’s therapeutic potential.
SNUG01’s multi-target approach, unlike mutation-specific therapies, offers a significant advantage for the majority of ALS patients with sporadic disease. SineuGene will partner with global institutions to conduct a multi-regional clinical trial, expediting SNUG01’s evaluation.
About SineuGene:
SineuGene Therapeutics, established in 2021, develops innovative therapies for neurological diseases. Leveraging AAV-mediated gene delivery and ASO-based gene regulation, SineuGene’s pipeline targets ALS, stroke, Parkinson’s, Alzheimer’s, Spinocerebellar Ataxia type 3, and Huntington’s disease. The company aims to translate research into effective treatments for neurological disorders.
About PackGene
PackGene Biotech is a world-leading CRO and CDMO, excelling in AAV vectors, mRNA, plasmid DNA, and lentiviral vector solutions. Our comprehensive offerings span from vector design and construction to AAV, lentivirus, and mRNA services. With a sharp focus on early-stage drug discovery, preclinical development, and cell and gene therapy trials, we deliver cost-effective, dependable, and scalable production solutions. Leveraging our groundbreaking π-alpha 293 AAV high-yield platform, we amplify AAV production by up to 10-fold, yielding up to 1e+17vg per batch to meet diverse commercial and clinical project needs. Moreover, our tailored mRNA and LNP products and services cater to every stage of drug and vaccine development, from research to GMP production, providing a seamless, end-to-end solution.
