CIRM Supports AcuraStem with $7.5 Million Grant for ALS and FTD Research

CIRM Supports AcuraStem with $7.5 Million Grant to Advance AS-241

AcuraStem, a pioneering biotechnology firm focused on developing treatments for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), has recently been awarded a substantial grant of $7.5 million from the California Institute for Regenerative Medicine (CIRM). This funding aims to propel their lead clinical candidate, AS-241, towards first-in-human trials.

AS-241 is an innovative antisense oligonucleotide (ASO) targeting a fundamental molecular issue associated with ALS and FTD: the dysfunction of the TDP-43 protein. This pathological loss of nuclear TDP-43 contributes to the cryptic splicing of the UNC13A gene, which ultimately disrupts synaptic communication due to the loss of essential proteins. Astonishingly, TDP-43 pathology is evident in approximately 97% of ALS patients, irrespective of their genetic backgrounds, and it is also prevalent in many individuals with FTD and other neurodegenerative disorders.

What sets AS-241 apart is its potential for wide-ranging efficacy. Unlike the recently approved drug Qalsody®, which only targets the 2% of ALS patients with the SOD-1 mutation, AS-241 aims to address a crucial consequence of TDP-43 pathology affecting the majority of ALS patients.

In preclinical studies using AcuraStem's advanced iNeuroRx® platform, AS-241 has shown promise by restoring normal UNC13A expression and enhancing synaptic function in neurons derived from ALS patients. Supporting safety and satisfactory central nervous system exposure have also been demonstrated in vivo, providing a firm basis for CIRM's decision to fund AcuraStem’s innovative program.

Sam Alworth, co-founder and CEO of AcuraStem, expressed his enthusiasm regarding the grant: "CIRM funding undergoes one of the most rigorous scientific and translational review processes in the field. Their support reflects strong confidence in the quality of our science and the therapeutic potential of AS-241. This award is crucial for rapidly moving AS-241 towards a Phase 1 clinical trial, and we are honored by CIRM's endorsement of this program."

AcuraStem is dedicated to developing treatments for neurodegenerative diseases based on patient biology. The company’s iNeuroRx® platform allows them to create disease-modifying therapies that target the core issues of these devastating conditions. Supporting collaborations with prominent public and private organizations enable AcuraStem to accelerate the development of transformative therapies for patients, reflecting their commitment to addressing unmet medical needs.

CIRM, established by California voters, has played a pivotal role in advancing stem cell and gene therapies, with a noteworthy funding portfolio that exceeds $8.5 billion since its inception in 2004. The institute focuses on enabling promising discoveries through clinical trials while bolstering the state’s biotechnology industry.

This grant underscores a critical step in the landscape of ALS and FTD research, illuminating a path forward for AcuraStem to potentially change the lives of those affected by these challenging conditions. For ongoing developments, visit AcuraStem's official website and stay connected through their social media platforms.

For more information on CIRM and its groundbreaking initiatives in regenerative medicine, visit CIRM's website.