SineuGene Announces FDA IND Clearance for SNUG01, the First-in-Class TRIM72-Targeted Gene Therapy for ALS
On March 24, 2025, SINEUGENE (Beijing) Biotechnology Co., Ltd. (“SINEUGENE”) announced that SNUG01, its self-developed global first-in-class gene therapy targeting TRIM72, has officially received Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA) for the treatment of amyotrophic lateral sclerosis (ALS, commonly known as “Lou Gehrig’s disease”). This approval marks the entry of the drug into Phase I/IIa international multicenter registrational clinical trials, systematically evaluating its safety, tolerability, and preliminary efficacy in adult patients with ALS, thereby achieving a critical transition from basic research to clinical application.
Amyotrophic lateral sclerosis (ALS) is a progressive, fatal neurodegenerative disease that affects both upper and lower motor neurons. Patients gradually lose motor, swallowing, and respiratory functions, with a median survival of only 3–5 years. As one of the most common motor neuron diseases in adults, ALS currently has no cure worldwide, and existing therapies can only modestly slow disease progression. Leveraging the original target discoveries from Professor Ji Yichang’s laboratory at Tsinghua University, SINEUGENE has focused on researching the pathogenic mechanisms of ALS and successfully developed SNUG01, a gene therapy drug with global intellectual property rights, offering new hope for treatment to patients.
SNUG01 utilizes recombinant adeno-associated virus serotype 9 (rAAV9) as a vector to deliver the human TRIM72 gene specifically to neurons via intrathecal (IT) injection. TRIM72 may mitigate neuronal damage in patients with amyotrophic lateral sclerosis (ALS) through multiple mechanisms, including membrane repair, restoration of antioxidant and mitochondrial functions, and reduction of stress granule formation.
In preclinical animal studies, SNUG01 demonstrated significant neuroprotective effects. In a prior investigator-initiated clinical study led by Peking University Third Hospital, SNUG01 exhibited favorable safety and tolerability, with encouraging positive signals observed in efficacy endpoints and biomarkers, thereby completing the validation chain from target discovery through preclinical development to human data. Compared with gene therapies targeting ALS caused by specific genetic mutations, SNUG01’s multi-faceted neuroprotective mechanisms enable it to address a broader range of ALS subtypes, particularly offering a potential therapeutic option for the 90% of patients with sporadic ALS who currently have no effective treatment.
SINEUGENE will join hands with global research and medical partners to continuously advance international multicenter clinical trials, accelerate the validation of the clinical value of this breakthrough therapy, and embark on a new journey to reshape the treatment landscape for amyotrophic lateral sclerosis (ALS).
SINEUGENE (Beijing) Biotechnology Co., Ltd. (hereinafter referred to as “SineuGene”) was established in late 2021. Headquartered in Changping District, Beijing, it is a biopharmaceutical company dedicated to the research and development of innovative therapies for neurological disorders. Leveraging over a decade of scientific expertise from Professor Ji Yichang’s team at the School of Medicine, Tsinghua University, SineuGene employs technologies such as adeno-associated virus (AAV) vector-mediated gene expression and editing, as well as antisense oligonucleotide (ASO)-mediated regulation of gene expression. The company has established multiple product pipelines aimed at addressing neurological conditions including amyotrophic lateral sclerosis (ALS), stroke, Parkinson’s disease, Alzheimer’s disease, spinocerebellar ataxia type 3 (SCA3), and Huntington’s disease.