MONROVIA, Calif., December 6, 2021 — AcuraStem (www.acurastem.com), a patient-based drug discovery platform company developing novel therapeutics for amyotrophic lateral sclerosis (ALS) and neurodegenerative diseases, announced today that the company will present posters featuring two of the company’s ALS drug development programs at the Motor Neuron Disease Association (MNDA) 32nd International Symposium on ALS/MND, being held virtually on December 7-10, 2021.
Scientific cofounder, Dr. Justin K. Ichida, PhD, and Head of Research, Dr. Wen-Hsuan Chang, PhD, will detail — for the first time publicly — the preclinical results of AcuraStem’s program investigating PIKFYVE suppression as a treatment for sporadic ALS, and also disclose for the first time the target of its 2nd program, a spliceosome-associated factor that can enhance normal TDP-43 function and reduce neurotoxic TDP-43 aggregates.
While a number of companies are pursuing approaches that target the known causative mutations in ALS, there is a dearth of disease modifying treatments in the pipeline for sporadic ALS,” said Dr. Ichida. “The preclinical data we will be presenting shows that PIKFYVE suppression engages a novel therapeutic mechanism that exploits an unexplored mode of protein homeostasis. The approach is highly effective in diverse models of ALS in vitro and in vivo, and is very promising for the sporadic ALS patient population.”
The disease-modifying potential of PIKFYVE for ALS was discovered by Dr. Ichida, in a landmark study appearing in Nature Medicine in 2018 (Shi Y et al Nature Med 2018). Using in vitro disease models derived from ALS patients, Dr. Ichida and colleagues discovered that reducing PIKFYVE levels was the most effective way to reduce motor neuron degeneration in these models.
Details of the presentations are as follows:
Title: Suppression of RNA splicing factors as a therapeutic strategy to address familial and sporadic ALS
Authors: J. Ichida, G. Linares, Y. Li, M. Santana, S. Hong, J. Rubin-SIgler, E. Lee and W. Chang
Presenting Author: Dr. Justin Ichida
Date/Time: December 7, 2021, 8:50am—10:20pm PST
Poster/Abstract Number: TST-05
Link to Abstract
Title: AS-202, a potent and safe PIKFYVE suppressing antisense oligonucleotide therapy for familial and sporadic ALS
Authors: W. Chang, S. Hung, G. Ichida, Y. Eoh, S. Hong, M. Santana, S. Mendonca, E. Lee and J. Ichida
Presenting Author: Dr. Wen-Hsuan Chang
Date/Time: December 9, 2021, 8:50am—10:20am PST
Poster/Abstract Number: TST-17
Link to Abstract
AcuraStem is a patient-based drug discovery platform company developing novel drug programs for neurodegenerative diseases. 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