AcuraStem Secures $4 Million in Grant Funding from CIRM to Advance UNC13A Program for ALS and FTD

PASADENA, Calif., Sept. 3, 2024 /PRNewswire/ -- AcuraStem, a patient-based biotechnology company advancing treatments for amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative diseases, announced today that it has successfully secured $4 million in grant funding from the California Institute for Regenerative Medicine (CIRM).

This latest funding will specifically support AcuraStem's continued efforts to advance its targeted therapeutic program for the UNC13A gene, using its iNeuroRx^® technology platform. iNeuroRx^® has been instrumental in the company's development of targeted therapeutics focused on the UNC13A gene, which plays a crucial role in the pathophysiology of ALS and FTD.

An overwhelming majority of ALS cases, and roughly half of FTD cases, are characterized by TDP-43 pathology. TDP-43, an 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 and FTD patients. AcuraStem's iNeuroRx^® platform has successfully replicated UNC13A pathology in patient neurons, enabling the rapid development and testing of antisense oligonucleotides (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.

"The support from CIRM is a testament to the potential of our research and its capacity to address critical challenges in neurodegenerative disease treatment," said Sam Alworth, MS, MBA, co-founder and CEO of AcuraStem. "This funding will facilitate the development of our UNC13A program toward clinical trials, enabling us to leverage our advances to potentially revolutionize the treatment landscape for ALS and FTD."

Funding from CIRM was awarded following a highly competitive and rigorous peer-review process, reflecting the strength of AcuraStem's research and potential impact on the treatment of ALS and FTD.

For additional information, please visit acurastem.com and follow on LinkedIn, Facebook, and X.

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.

About the California Institute for Regenerative Medicine (CIRM)

At CIRM, we never forget that we were created by the people of California to accelerate stem cell treatments to patients with unmet medical needs, and act with a sense of urgency to succeed in that mission. To meet this challenge, our team of highly trained and experienced professionals actively partners with both academia and industry in a hands-on, entrepreneurial environment to fast track the development of today's most promising stem cell technologies.

With $5.5 billion in funding and more than 150 active stem cell programs in our portfolio, CIRM is one of the world's largest institutions dedicated to helping people by bringing the future of cellular medicine closer to reality.

Contacts:

Kissy Black
Director of Communications, AcuraStem
[email protected]
615.310.1894

Roxan Olivas
Lotos Nile
[email protected]
520-954-1634

SOURCE AcuraStem - Patient-Based Therapeutics