AcuraStem CSO Marcel van der Brug Presents at the 35th International Symposium on ALS/MND

MONTREAL, December 20, 2024 – At the 35th International Symposium on ALS/MND, held from December 6-8, 2024, at the Montreal Convention Center, AcuraStem Chief Scientific Officer Marcel van der Brug delivered a key presentation titled 'Development of an UNC13A Cryptic Exon Skipping Antisense Oligonucleotide as a Treatment for ALS.' AcuraStem CEO Sam Alworth and Dr. Wen-Hsuan Chang, Associate Vice President, Discovery and Early Development, were also in attendance, emphasizing the company’s dedication to leadership in this critical research area.

AcuraStem was a proud sponsor of the symposium, which is recognized as the foremost gathering for exchanging insights and the latest research findings in the fight against Amyotrophic Lateral Sclerosis (ALS) and Motor Neuron Diseases (MND). This event annually attracts over 1,000 global delegates, including leading figures from biotechnology, pharmaceuticals, and academic research, fostering critical discussions on innovative therapies.

During his talk, Dr. van der Brug outlined the promising advances made by AcuraStem in the development of antisense oligonucleotides (ASOs) targeting the UNC13A gene, a genetic risk factor for ALS and Frontotemporal Dementia (FTD). He detailed the mechanism by which the UNC13A risk variant contributes to disease pathology—by exacerbating an RNA processing error in neurons that results in the inclusion of intronic sequence  as a cryptic exon in UNC13A RNA transcript. This misprocessing leads to a loss of UNC13A RNA transcripts and subsequently protein, critically impacting neuron function.

AcuraStem’s novel ASOs are designed to prevent this cryptic exon inclusion, thereby restoring normal UNC13A protein function. The patient-derived disease models, developed through the iNeuroRx® technology platform, are instrumental in rapidly translating these laboratory discoveries into potential clinical treatments. Dr. van der Brug emphasized the significant therapeutic potential of the UNC13A ASOs in treating and potentially slowing the progression of neurodegenerative diseases like ALS and FTD.

AcuraStem remains committed to pushing the boundaries of medical science to discover effective treatments for ALS and FTD. Through its innovative approach and rigorous scientific research, the company is determined to transition these novel therapeutics from the laboratory to the clinic, offering hope to patients and families affected by these devastating diseases.

For more information about AcuraStem’s efforts in neurodegenerative disease research, please visit acurastem.com.