Dr. Wen-Hsuan Chang, AcuraStem’s Head of Target Validation
to present at the 33rd International Symposium on ALS/MND
being held virtually on December 6-9, 2022
MONROVIA, Calif., December 6, 2022 — AcuraStem (acurastem.com), a near-to-clinic patient-based therapeutics company advancing treatments for a range of neurodegenerative disorders including Amyotrophic Lateral Sclerosis (ALS), Frontotemporal Dementia (FTD) and Charcot-Marie-Tooth Disorder (CMT), announced today that the Head of Target Validation, Dr. Wen-Hsuan Chang, Ph.D., will present a poster on the company’s newest therapeutic program, an UNC13A cryptic exon skipping antisense oligonucleotide as a treatment for ALS and FTD at the Motor Neuron Disease Association (MNDA) 33rd International Symposium on ALS/MND, being held virtually on December 6-9, 2022.
Dr. Chang will detail AcuraStem’s development program leveraging its best-in-class iNeuroRx® platform, composed of patient-derived disease models that accurately recapitulate UNC13A pathology, to develop novel and potent UNC13A antisense oligonucleotide (ASO) drug candidates. UNC13A is one of the most significant genetic risk factors for ALS and FTD. It was recently discovered that the risk variant exacerbates an RNA processing error in neurons in patients with TDP-43 pathology (97% of ALS patients and 50% of FTD patients). The error incorrectly includes intronic UNC13A DNA as an exon (a cryptic exon) in UNC13A mRNA leading to a loss of UNC13A protein function. Patients with two copies of the risk allele survive less than patients with one allele, who survive less than patients with normal DNA. Patients with two risk alleles have more cryptic exon than patients with one allele, who have more than patients with normal DNA. This additive risk strongly suggests that UNC13A dysfunction is central to TDP 43 pathology. AcuraStem’s UNC13A ASOs prevent the cryptic exon inclusion and restore normal UNC13A protein function.
Leaders in ASO design and development and ALS/FTD disease modeling, AcuraStem’s team is generating optimally effective UNC13A ASOs that prevent CE inclusion and restore normal UNC13A function. Details of the presentation are as follows:
Title: Development of an UNC13A cryptic exon skipping antisense oligonucleotide as a treatment for ALS and FTD
Presenting Author: Dr. Wen Chang
Date/Time: Dec 6, 2022 17.05 – 18.35 GMT
Poster/Abstract Number: TST-04
AcuraStem is a a near-to-clinic patient-based therapeutics company advancing treatments for a range of neurodegenerative disorders including Amyotrophic Lateral Sclerosis (ALS), Frontotemporal Dementia (FTD) and Charcot-Marie-Tooth Disorder (CMT). AcuraStem’s iNeuroRx® technology platform combines patient-derived disease models with human genetic data. The company is led by experts in disease modeling, machine learning and drug development.
Director of Communications, AcuraStem