AcuraStem’s Associate Vice President, Discovery and Early Development, Dr. Wen-Hsuan (Wen) Chang, will present at the 5th International Oligonucleotides and Peptides Conference (IOPC) on June 26, 2024, in Milan. Her presentation, titled "Accelerating ASO Optimization through Humanized Preclinical Models on a Patient-based Platform," highlights AcuraStem's commitment to advancing neurodegenerative disease research through its proprietary iNeuroRx® technology platform.
Dr. Chang will discuss the progress of AcuraStem’s UNC13A program, emphasizing the potential of antisense oligonucleotides (ASOs) targeting the UNC13A gene. An overwhelming majority of ALS cases involve TDP-43 pathology, where the RNA-binding protein, typically found in the nucleus, is mislocalized to the cytoplasm in affected neurons. This mislocalization leads to RNA processing errors, incorporating a cryptic exon into the UNC13A messenger RNA, and causing a loss of UNC13A protein. This loss is 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 that 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.
The 5th IOPC, a premier event for innovators in the field of oligonucleotides and peptides, offers insights into discovery, delivery, and formulation alongside manufacturing processes.
AcuraStem is dedicated to advancing ALS research through its iNeuroRx® platform, which enables the discovery of innovative, effective, and broadly acting treatments. The company’s participation in this important industry event provides a valuable opportunity to showcase its progress in developing groundbreaking therapies for neurodegenerative diseases and transitioning these innovations from the laboratory to the clinic.
For more information about AcuraStem’s pioneering work, please visit acurastem.com.
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, 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