AcuraStem Associate Vice President, Discovery and Early Development, Dr. Wen-Hsuan Chang, Presents at the 15th Annual ALS Network Research Summit

SAN FRANCISCO, February 7, 2025 – At the 15th annual ALS Network Research Summit, held on January 16, AcuraStem's Associate Vice President, Discovery and Early Development, Dr. Wen-Hsuan Chang delivered a key presentation titled 'Development of an UNC13A ASO for ALS.'  

AcuraStem proudly sponsored the summit, which serves as a premier gathering for the latest research findings in the battle against Amyotrophic Lateral Sclerosis (ALS). This annual event attracts leaders from biotechnology, pharmaceuticals, and academic research, encouraging vital discussions on new therapies. 

During her presentation, Dr. Chang detailed the advances AcuraStem has driven in the development of antisense oligonucleotides (ASOs) that target the UNC13A gene—a known genetic risk factor for ALS. She outlined the mechanism by which the UNC13A risk variant exacerbates an RNA processing error in neurons, leading to the inclusion of a cryptic exon in UNC13A RNA transcripts. This aberrant processing results in a loss of UNC13A RNA transcripts and subsequently protein, critically impairing 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 AcuraStem’s iNeuroRx® technology platform, are instrumental in rapidly translating these laboratory discoveries into potential clinical treatments. Dr. Chang emphasized the significant therapeutic potential of the UNC13A ASOs in treating and potentially slowing the progression of neurodegenerative diseases like ALS and FTD. She also discussed how the iNeuroRx platform is optimally configured to identify the most impactful new targets for ALS, which can be rapidly pursued with antisense oligonucleotide therapeutics. 

AcuraStem continues to lead the way in developing effective treatments for ALS and FTD, driven by rigorous scientific research and a commitment to innovative approaches. Over the last several years the company has validated assays that comprehensively recapitulate ALS disease processes in a large number of ALS patient lines reflecting the diverse heterogeneity of the disease.  The company’s iNeuroRX® platform represents a new frontier in ALS drug discovery, integrating robust genetic insights, high-throughput screening, and disease-relevant phenotyping to deliver therapies with the highest likelihood of success.  

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