AcuraStem Sponsors ALS Network's Champions for Cures and Care Gala
AcuraStem’s Head of Target Validation, Dr. Wen-Hsuan Chang, presented an update on the company's innovative UNC13A program at the 14th Annual California ALS Research Summit on January 19, 2024. AcuraStem’s UNC13A program focuses on the development of antisense oligonucleotides (ASOs) targeting the UNC13A gene, which is a key risk factor in amyotrophic lateral sclerosis (ALS) and Frontotemporal Dementia (FTD). Dr. Chang's presentation emphasized the potential of this approach to alter the course of these devastating diseases.
Dr. Chang detailed the science behind AcuraStem's UNC13A program, highlighting its significant advancement in understanding ALS's pathophysiology, with a focus on the crucial role of UNC13A. An overwhelming majority of ALS cases, and roughly half of FTD cases, are characterized by TDP-43 pathology. TDP-43, a RNA-binding protein typically found in the nucleus, is mislocalized to the cytoplasm in affected neurons, leading to RNA processing errors. These errors result in the incorporation of a cryptic exon into the UNC13A messenger RNA, causing a loss of UNC13A protein. This loss is further exacerbated by a risk variant in the UNC13A gene, closely linked to the TDP-43 binding site and associated with reduced survival in ALS and FTD patients. AcuraStem's iNeuroRx® platform has successfully replicated UNC13A pathology in patient neurons, enabling the rapid development and testing of ASOs. These ASOs effectively suppress the cryptic exon and restore normal UNC13A function, offering a promising therapeutic approach for TDP-43 proteinopathies and a deeper understanding of UNC13A's role in disease progression.
Dr. Chang’s presentation also covered the broader implications of the UNC13A research, including how it fits into the landscape of ALS and neurodegenerative disease research. With TDP-43 proteinopathies being a common feature in a significant portion of ALS, FTD, and even Alzheimer’s cases, targeting the UNC13A gene represents a novel and promising approach.
Furthermore, Dr. Chang shared insights into the rigorous testing process of these ASOs and the complexities and potential pathways for transitioning research from laboratory settings to clinical application—highlighting how AcuraStem’s proprietary scientific methodologies uniquely equip them to pioneer advancements in this domain. The development of ASOs as a therapeutic option for ALS and FTD is part of a broader shift towards precision medicine in neurology, which aims to tailor treatments to individual genetic profiles and disease mechanisms. This approach could not only improve outcomes for patients with ALS and FTD but also serve as a model for tackling other neurodegenerative disorders.
Additionally, for the second year in a row, AcuraStem was a proud sponsor of the ALS Network's Champions for Cures and Care Gala, an annual event that coincides with the research summit, dedicated to recognizing and celebrating significant contributions towards combating ALS.
The gala, a cornerstone event for the ALS community, aims to bring together researchers, patients, and advocates to honor significant achievements and encourage ongoing support for ALS research and patient care. It serves as a reminder of the critical need for funding and the collective effort required to drive advancements in treatment and care for those affected by ALS.
The sponsorship of the ALS Network's Champions For Cures and Care gala and Dr. Chang's presentation are reflective of AcuraStem's dedication to the ALS community and its mission to bring groundbreaking therapies from the lab to the patients who need them most. Through its research and community engagement, AcuraStem continues to play a pivotal role in the global effort to understand and combat neurodegenerative diseases.
AcuraStem invites the broader community to continue supporting ALS research and advocacy efforts. Contributions to the ALS Network can make a meaningful difference in the lives of those affected by ALS and their families.
To find out more about supporting the ALS Network, please visit: alsnetwork.org.
About AcuraStem
AcuraStem is a patient-based biotechnology company pioneering treatments for neurodegenerative diseases including sporadic ALS and FTD. AcuraStem’s best-in-class disease modeling platform, iNeuroRx®, enables the discovery of innovative, effective, and broadly acting treatments. The team’s strong expertise in ASO technology provides for the rapid advancement of treatments to the clinic. AcuraStem’s research is funded in part by support from the Alzheimer’s Drug Discovery Foundation, Harrington Discovery Institute, Alzheimer’s Association, Rainwater Charitable Foundation, Muscular Dystrophy Association, Department of Defense and the National Institute of Neurological Disorders and Stroke.
Contact:
Kissy Black
Director of Communications, AcuraStem
kblack@acurastem.com
615.310.1894