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BALTIMORE, Sept. 19, 2025 ~ The 150th American Neurological Association Annual Meeting (ANA 2025) was a resounding success as it brought together some of the most prominent experts, scholars, and industry leaders in the field of neuroscience. Held in Baltimore, USA from September 13 to 16, 2025, the conference served as a platform for discussions on the latest research advancements and therapeutic strategies for neurological diseases.
One of the highlights of the conference was an oral presentation by DarwinCell in the "Neuromuscular Disease SIG" session. The company also presented a poster showcasing their latest research data on ALT001, a novel MSC-based drug for the treatment of amyotrophic lateral sclerosis (ALS). This poster was recognized for its innovation and scientific value and received the prestigious "Abstract of Distinction" award at this year's Annual Meeting. It garnered significant attention from attendees at the conference.
ALS is a debilitating disease characterized by progressive skeletal muscle weakness, atrophy, fasciculations, bulbar palsy, and pyramidal tract signs. It ultimately leads to death due to respiratory and circulatory system failure. The age of onset for ALS is getting younger, with some patients experiencing symptoms as early as 20 years old [1]. In China, the incidence of ALS is approximately 0.6 per 100,000 people with a prevalence rate of about 3.1 per 100,000 [2-5]. The typical survival period for ALS patients is only 3-5 years [2-5]. Currently, Riluzole and Edaravone are the primary treatments available in China [4], highlighting an urgent need for new therapeutic approaches.
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DarwinCell's ALT001 has shown promising clinical benefits in both in vitro studies and Investigator Initiated Trials (IITs) involving more than 40 ALS patients. In vitro experiments have demonstrated that cells treated with ALT001 were able to restore cell viability after oxidative damage. Additionally, ALT001 has been found to protect motoneurons against neurotoxicity and ameliorate muscle atrophy in SOD1G93A mice, a common model for ALS. These results were further supported by improved motor functions and extended lifespan in mutant mice treated with ALT001 compared to those treated with a vehicle or Riluzole.
Multiple IITs, including a double-blinded study, have also been conducted to evaluate the safety and efficacy of ALT001 on ALS patients. After just two weeks of administration, most patients reported positive outcomes, as evidenced by elevated performance on the ALS Functional Rating Scale-Revised (ALSFRS-R) and modified Norris scores.
"We are honored to have the opportunity to share our latest data on ALT001 at this prestigious global conference," said Ms. Wang Yu, Founder and CEO of DarwinCell. "These results further strengthen our confidence in developing ALT001 for ALS and other major neurological diseases. Our goal is to combine the functional diversity of cell therapies with the practicality of traditional biologics to provide patients with more effective, safer, and accessible treatment options. We remain committed to being clinically-oriented and driven by scientific innovation as we continue to deepen our presence in neuroscience and regenerative medicine. We are actively working towards advancing the IND application processes for ALT001 in both China and the U.S., with the hope of bringing new hope to patients worldwide as soon as possible."
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The success of DarwinCell's presentation at ANA 2025 has sparked excitement among attendees and renewed hope for potential breakthroughs in treating ALS. With their dedication to scientific innovation and commitment to improving patient outcomes, DarwinCell is poised to make significant contributions in the field of neuroscience and regenerative medicine.
One of the highlights of the conference was an oral presentation by DarwinCell in the "Neuromuscular Disease SIG" session. The company also presented a poster showcasing their latest research data on ALT001, a novel MSC-based drug for the treatment of amyotrophic lateral sclerosis (ALS). This poster was recognized for its innovation and scientific value and received the prestigious "Abstract of Distinction" award at this year's Annual Meeting. It garnered significant attention from attendees at the conference.
ALS is a debilitating disease characterized by progressive skeletal muscle weakness, atrophy, fasciculations, bulbar palsy, and pyramidal tract signs. It ultimately leads to death due to respiratory and circulatory system failure. The age of onset for ALS is getting younger, with some patients experiencing symptoms as early as 20 years old [1]. In China, the incidence of ALS is approximately 0.6 per 100,000 people with a prevalence rate of about 3.1 per 100,000 [2-5]. The typical survival period for ALS patients is only 3-5 years [2-5]. Currently, Riluzole and Edaravone are the primary treatments available in China [4], highlighting an urgent need for new therapeutic approaches.
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DarwinCell's ALT001 has shown promising clinical benefits in both in vitro studies and Investigator Initiated Trials (IITs) involving more than 40 ALS patients. In vitro experiments have demonstrated that cells treated with ALT001 were able to restore cell viability after oxidative damage. Additionally, ALT001 has been found to protect motoneurons against neurotoxicity and ameliorate muscle atrophy in SOD1G93A mice, a common model for ALS. These results were further supported by improved motor functions and extended lifespan in mutant mice treated with ALT001 compared to those treated with a vehicle or Riluzole.
Multiple IITs, including a double-blinded study, have also been conducted to evaluate the safety and efficacy of ALT001 on ALS patients. After just two weeks of administration, most patients reported positive outcomes, as evidenced by elevated performance on the ALS Functional Rating Scale-Revised (ALSFRS-R) and modified Norris scores.
"We are honored to have the opportunity to share our latest data on ALT001 at this prestigious global conference," said Ms. Wang Yu, Founder and CEO of DarwinCell. "These results further strengthen our confidence in developing ALT001 for ALS and other major neurological diseases. Our goal is to combine the functional diversity of cell therapies with the practicality of traditional biologics to provide patients with more effective, safer, and accessible treatment options. We remain committed to being clinically-oriented and driven by scientific innovation as we continue to deepen our presence in neuroscience and regenerative medicine. We are actively working towards advancing the IND application processes for ALT001 in both China and the U.S., with the hope of bringing new hope to patients worldwide as soon as possible."
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The success of DarwinCell's presentation at ANA 2025 has sparked excitement among attendees and renewed hope for potential breakthroughs in treating ALS. With their dedication to scientific innovation and commitment to improving patient outcomes, DarwinCell is poised to make significant contributions in the field of neuroscience and regenerative medicine.
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