BALTIMORE, Sept. 19, 2025 /PRNewswire/ — The 150th American Neurological Association Annual Meeting (ANA 2025) was successfully held in Baltimore, USA, from September 13 to 16, 2025. The conference brought together leading global experts, scholars, and industry leaders in the field of neuroscience to discuss the latest research advances and therapeutic strategies for neurological diseases. DarwinCell delivered an oral presentation in the “Neuromuscular Disease SIG” session and presented a poster showcasing the latest research data on its novel MSC-based drug ALT001 for the treatment of amyotrophic lateral sclerosis (ALS). The company’s poster, recognized for its innovation and scientific value, received the “Abstract of Distinction” at this year’s Annual Meeting and attracted wide attention on site.
Clinically, ALS is characterized by progressively developing skeletal muscle weakness, atrophy, fasciculations, bulbar palsy, and pyramidal tract signs, ultimately leading to death due to respiratory and circulatory system failure. The age of onset is trending younger, with a minority of patients developing symptoms as early as around 20 years old [1]. The incidence of ALS in China is approximately 0.6 per 100,000 people, with a prevalence of about 3.1 per 100,000 and a typical survival period of 3-5 years [2-5]. Currently, the primary treatments available in China are still Riluzole and Edaravone [4], highlighting an urgent need for new therapeutic approaches.
ALT001 demonstrates promising clinical benefits. Both in vitro studies and IIT studies on more than 40 ALS patients are conducted. In vitro experiments showed that cells treated with ALT001 restored cell viability after oxidative damage. Progressive loss of anterior horn motoneurons and muscle atrophy due to the lack of neuronal innervation are hallmarks of ALS. We found that ALT001 protected motoneurons against neurotoxicity, and ameliorated muscle atrophy in SOD1G93A mice. In agreement with these results, the mutant mice treated with ALT001 exhibited improved motor functions and extended lifespan in comparison to mice treated with vehicle or Riluzole. We then carried out multiple IITs including a double-blinded study on the safety and the efficacy of ALT001 on ALS patients. After 2-weeks of administration of ALT001, most patients reported positive outcomes, as demonstrated by elevated performance, ALSFRS-R and modified Norris scores.
“We are greatly honored to share the latest data on ALT001 at this global academic forefront conference,” said Ms. Wang Yu, Founder and CEO of DarwinCell. “These results reinforce our confidence in further developing ALT001 for ALS and other major neurological diseases. ALT001 is designed to combine the functional diversity of cell therapies with the practicality of traditional biologics, aiming to provide patients with more effective, safer, and accessible treatment options. The company remains committed to being clinically-oriented and driven by scientific innovation, continuously deepening our presence in neuroscience and regenerative medicine. Darwin will fully advance the IND application processes for ALT001 in both China and the U.S., hoping to bring new hope to patients worldwide as soon as possible.”
About DarwinCell
DarwinCell founded in 2016, is a national high-tech enterprise focused on innovative treatments for neurological diseases. Leveraging its proprietary protein polymer extraction technology (ECIWEP), the company has built a comprehensive technology platform from drug discovery to clinical translation. Through deep collaborations with numerous leading medical and research institutions, DualityBio is dedicated to becoming an internationally leading innovator in neural repair technology. For more information, please visit https://darwincell.net/en.
Reference
[1]Chinese Society of Neurology, Amyotrophic Lateral Sclerosis Collaboration Group. (2022). Chinese Expert Consensus on the Diagnosis and Treatment of Amyotrophic Lateral Sclerosis (2022). Chinese Journal of Neurology, 55(6), 8.
[2]Feldman EL, Goutman SA, Petri S, et al. Amyotrophic lateral sclerosis. *Lancet*. 2022;400(10360): 1363-1380.
[3]van Es MA,Hardiman O,Chio A,et al. Amyotrophic lateral sclerosis[J].Lancet,2017,390 (10107):2084-2098.
[4]NORRIS S P, LIKANJE M N, ANDREWS J A. Amyotrophic lateral sclerosis: update onclinicalmanageme nt[J]. Curr Opin Neurol. 2020; 33(5):641- 648.
[5]Talbott EO, Malek AM, Lacomis D. The epidemiology of amyotrophic lateral sclerosis. *Handb Clin Neurol*. 2016;138: 225-238.
