AcuraStem CEO, Sam Alworth, will present on the company's trailblazing approach to  amyotrophic lateral sclerosis (ALS) therapies at the MDA Clinical & Scientific Conference on March 4th. The presentation will center on AcuraStem's development of a UNC13A Cryptic Exon Skipping Antisense Oligonucleotide (ASO), showcasing the company's commitment to combating ALS through its groundbreaking iNeuroRx® platform.

The MDA Clinical & Scientific Conference, set to take place from March 3-6 at the Hilton Orlando in Florida, gathers together a prominent assembly of leading researchers, clinicians, and industry professionals. This year's conference, emphasizing the "Era of Treatment" for neuromuscular diseases, will provide an unparalleled opportunity for attendees to explore the latest in pre-clinical, translational, and clinical research.

Alworth's presentation will showcase the science behind AcuraStem’s UNC13A program, underscoring the potential of ASOs targeting the UNC13A gene. An overwhelming majority of ALS 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 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.

In addition to Alworth's oral presentation, he will also present Poster M224, scheduled for Monday, the 4th, between 6 and 8 PM. This poster will highlight AcuraStem's rigorous testing process, the challenges and potential pathways from laboratory research to clinical application, and the broader implications of targeting UNC13A in neurodegenerative disease research.

AcuraStem's presence at the MDA Clinical & Scientific Conference highlights the company's role as a frontrunner in neurology, showcasing its commitment to advancing precision medicine strategies that aim to enhance treatment results for individuals with ALS and related neurodegenerative conditions.

In September 2023, AcuraSteam announced it had entered into a license agreement with Takeda to develop and commercialize AcuraStem’s PIKFYVE targeted therapeutics including AS-202, an innovative ASO for the treatment of ALS.

To learn more about AcuraStem's transformative research, please visit




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 FoundationMuscular Dystrophy AssociationDepartment of Defense and the National Institute of Neurological Disorders and Stroke.


Kissy Black
Director of Communications, AcuraStem