Congratulations to GenSci Changhua on receiving implied clinical trial approval from the National Medical Products Administration for SNUG01, the world’s first gene therapy drug for ALS

SineuGene (Beijing) Biotechnology Co., Ltd. (“SineuGene”) announced that SNUG01, its self-developed, first-in-class gene therapy drug targeting TRIM72, officially received implied clinical trial approval from the National Medical Products Administration (NMPA) on August 14, 2025. The proposed indication is amyotrophic lateral sclerosis (ALS, commonly known as “Lou Gehrig’s disease”). As a strategic partner of SineuGene, PackGene extends its sincere congratulations on this significant progress.

This IND approval marks another important milestone for SNUG01 following its clinical trial clearance from the U.S. Food and Drug Administration (FDA) in March 2025 and its Orphan Drug Designation granted by the FDA in June 2025. It signifies that SNUG01 has entered the development stage of a China-U.S. international multicenter clinical trial (MRCT).

SNUG01 is a first-in-class gene therapy product developed by SineuGene based on its AAV technology platform. It is the world’s first gene therapy drug using TRIM72 as the GOI (Gene of Interest). This target was discovered in an ALS model constructed using next-generation targeted gene knock-in technology by the laboratory of Professor Yichang Jia at the School of Basic Medicine, Tsinghua University. SNUG01 uses recombinant adeno-associated virus serotype 9 (rAAV9) as the vector and precisely delivers the human TRIM72 gene to neurons via intrathecal (IT) injection. Preclinical studies have shown that TRIM72 may protect neurons and delay motor neuron degeneration in ALS patients through multiple mechanisms, including membrane repair, antioxidant and mitochondrial function restoration, and reduction of stress granule formation. The completed investigator-initiated trial (IIT) of SNUG01 has preliminarily demonstrated good safety and tolerability, while also showing positive signals in efficacy endpoints and biomarker improvement.

The SNUG01 Phase I/IIa international multicenter clinical trial will be led by Professor Merit Cudkowicz, Director of the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital in the United States. The China study will be led by Professor Dongsheng Fan’s team at Peking University Third Hospital, in collaboration with Professor Zhiying Wu’s team at the Second Affiliated Hospital of Zhejiang University School of Medicine and Professor Zhangyu Zou’s team at Fujian Medical University Union Hospital. These research centers are all authoritative institutions in the field of clinical research on neurodegenerative diseases in China and abroad, with extensive experience in ALS clinical trials. The clinical trial will adopt unified international standards and assessment systems to systematically evaluate the safety, tolerability, and preliminary efficacy of SNUG01 in Chinese and U.S. populations.

PackGene is honored to provide AAV vector services for this project, covering plasmid and AAV process development and optimization, GMP manufacturing, analytical method development and validation, quality studies, product release testing, stability studies, and the preparation of complete CMC documentation, helping accelerate the commercialization process of SineuGene’s SNUG01 gene therapy product. Looking ahead, PackGene will continue to deepen its expertise in the field of gene therapy, provide one-stop CMC solutions for drug innovation, and support the advancement of more high-quality projects.

Expert Remarks: Advancing the International Clinical Translation of Original Innovative Drugs

Professor Merit Cudkowicz, Director of the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, said: “The dual IND approvals in China and the United States accelerate and validate the strategy of developing potentially transformative therapies for ALS patients worldwide. We are pleased to collaborate on this study and look forward to evaluating the potential impact of SNUG01 on ALS patients.”

Professor Dongsheng Fan of Peking University Third Hospital, the principal investigator in China, said: “Based on the positive signals observed in the previous IIT study, we are confident in the clinical potential of SNUG01. Going forward, through multicenter collaboration between China and the United States, we will systematically evaluate the safety and efficacy of SNUG01 in adult ALS patients, and we look forward to validating the clinical trial value of SNUG01 as soon as possible, providing better treatment options for ALS patients worldwide.”

Management Remarks: Accelerating the Global Registration Clinical Trial Process of SNUG01 to Benefit Patients Worldwide

Professor Yichang Jia, founder of SineuGene and Vice Dean of the School of Basic Medicine at Tsinghua University, said: “The discovery of the TRIM72 target is the result of more than a decade of fundamental research by the Tsinghua research team. The smooth advancement of its clinical translation demonstrates the potential and responsibility of original Chinese scientific research in addressing major disease challenges. We look forward to SNUG01 changing the current global treatment landscape for ALS.”

Dr. Lin Peng, co-founder and CEO of SineuGene, stated: “This NMPA implied clinical trial approval is an important milestone in the company’s development and in the clinical development of SNUG01. We sincerely thank our new and existing investors, investigators, and partners for their continued support. We will integrate high-quality global medical resources, accelerate the advancement of international multicenter clinical trials, efficiently validate the safety and clinical value of this first-in-class innovative drug, and promote Chinese innovation to benefit patients around the world as soon as possible.”

SineuGene will continue to advance the global clinical development of SNUG01 and accelerate its clinical development and registration worldwide, with a commitment to providing new treatment options for patients with ALS and other neurodegenerative diseases.

About Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis (ALS) is a progressive and fatal neurodegenerative disease involving both upper and lower motor neurons. Patients experience progressively worsening muscle weakness and atrophy, eventually affecting swallowing and respiratory functions. As one of the most common motor neuron diseases in adults, ALS has a median survival period of only 3–5 years, and existing therapies worldwide can only modestly delay disease progression. Unlike therapies that target only specific gene mutation-associated forms of ALS, SNUG01, with its multidimensional neuroprotective mechanisms, can delay motor neuron degeneration in ALS patients and is expected to cover a broader ALS patient population. In particular, it may provide a potential solution for patients with sporadic ALS (sALS), who account for 90% of all ALS cases and currently lack effective treatment options.

About SineuGene

Founded at the end of 2021, SineuGene is a clinical-stage biopharmaceutical company focused on gene therapy for neurological diseases. The company’s first pipeline originated from more than a decade of fundamental neuroscience research conducted by Professor Yichang Jia’s laboratory at Tsinghua University School of Medicine. SineuGene has established novel gene knock-in animal disease model platforms, including Drosophila, mouse, and Bama miniature pig models, to maximally recapitulate disease characteristics and identify more reliable innovative drug targets. The company has also established a central nervous system AAV screening platform and, combined with AAV receptor protein structure prediction, is developing novel AAV serotypes with strong targeting ability, high central nervous system delivery efficiency, and low immunogenicity. In addition, SineuGene has built a central nervous system small nucleic acid drug R&D platform covering AI-enabled small nucleic acid target screening, sequence design optimization, nucleic acid synthesis, chemical modification, and delivery technology development.

Based on AAV-mediated gene expression and small nucleic acid-mediated gene expression regulation technologies, SineuGene has established multiple product pipelines dedicated to tackling neurological diseases such as amyotrophic lateral sclerosis (ALS), multiple system atrophy (MSA), Parkinson’s disease (PD), stroke, Alzheimer’s disease (AD), and spinocerebellar ataxia type 3 (SCA3). In 2025, SNUG01, the company’s first pipeline for ALS, successively received implied clinical trial approvals from the FDA and NMPA, and the company is actively advancing a China-U.S. international multicenter clinical trial. Prior to this, SNUG01 had already been undergoing multicenter investigator-initiated clinical studies (IITs) simultaneously at Peking University Third Hospital, the Second Affiliated Hospital of Zhejiang University School of Medicine, and Fujian Medical University Union Hospital.

Since its establishment at the end of 2021, SineuGene has continued to gain recognition from the capital market for its original technology platform and R&D capabilities, with its financing progress closely aligned with its R&D milestones. The company has completed a seed round exclusively invested by the Beijing Life Science Park Innovation Investment Fund; a seed+ round followed by Shanghai Fengcang Innovation Fund; and an angel round co-led by Qiming Venture Partners and Shanghai Fengcang Innovation Fund, with participation from Hangzhou Zeming and the Shuimu Tsinghua Alumni Seed Fund. In 2024, SineuGene successfully completed a nearly RMB 100 million Pre-A financing round led by Zhongguancun Capital, with participation from Zhongbo Juli, Transfar Capital, and Sanmei Investment.

The funds will continue to be used for the construction of technology platforms, preclinical research on relevant pipelines, and the advancement of SNUG01 global clinical trials. The company sincerely thanks all investment institutions for their trust and support, and will continue to leverage capital resources to accelerate the global translation of innovative therapies.