Patient-Based Therapeutics

Many scientists believed a single, effective treatment for all patients wouldn't be possible, instead focusing on genetic forms of disease, but AcuraStem is showing how it can be done.

News & Events

December 20, 2024

At the 35th International Symposium on ALS/MND, held from December 6-8, 2024, at the Montreal Convention Center, AcuraStem Chief Scientific Officer Marcel van der Brug delivered a key presentation titled 'Development of an UNC13A Cryptic Exon Skipping Antisense Oligonucleotide as a Treatment for ALS.

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December 4, 2024

AcuraStem announced the appointment of Marcel van der Brug, Ph.D., as Chief Scientific Officer. Dr. van der Brug is an accomplished neuroscientist with extensive expertise in neurodegenerative diseases and therapeutic development.

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September 9, 2024

At the RNA Leaders USA Congress, held September 4-5, 2024, at the San Diego Convention Center, AcuraStem CEO Sam Alworth delivered a key presentation titled 'Accelerating the Development of More Effective Treatments for ALS & FTD Using the iNeuroRx® Technology Platform.'

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September 3, 2024

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) as part of CIRM's Therapeutic Translational Research Projects program, which accelerates the preclinical development of innovative therapies.

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August 26, 2024

AcuraStem, a patient-based biotechnology company pioneering the development of treatments for neurodegenerative diseases, today announced the appointment of Dr. Rafael Renteria as Principal Scientist, hiPSC Platform.

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August 12, 2024

AcuraStem, a patient-based biotechnology company advancing treatments for amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other neurodegenerative diseases, announced today that CEO Sam Alworth has been invited to speak at the RNA Leaders USA Congress, scheduled for September 4-5, 2024, at the San Diego Convention Center.

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June 24, 2024

AcuraStem, a patient-based biotechnology company pioneering the development of treatments for neurodegenerative diseases, today announced the appointment of Dr. Ananya Sengupta as Associate Director of Program Management

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June 21, 2024

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

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June 18, 2024

AcuraStem, a patient-based biotechnology company pioneering the development of treatments for neurodegenerative diseases, today announced it has named Dr. Wen-Hsuan Chang as Associate Vice President of Discovery and Early Development

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June 18, 2024

AcuraStem proudly sponsored the recent IgNight ALS event in Washington, D.C., a poignant evening dedicated to celebrating Steve Gleason and his profound contributions to the ALS community

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June 3, 2024

AcuraStem, a patient-based biotechnology company pioneering how treatments are developed for neurodegenerative diseases, announced the expansion of its team with three new team members, bringing a fresh influx of expertise to its pioneering platform.

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May 17, 2024

AcuraStem’s Associate Vice President, Dr. Wen-Hsuan (Wen) Chang, to Present at 3rd Annual ALS Drug Development Summit and Participate in Panel and Roundtable Discussions

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February 27, 2024

AcuraStem CEO, Sam Alworth, will present on the company's trailblazing approach to amyotrophic lateral sclerosis (ALS) therapies at the MDA Clinical & Scientific Conference on March 4th.

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February 27, 2024

AcuraStem’s Head of Target Validation, Dr. Wen-Hsuan Chang, presented an update on the company's innovative UNC13A program at the 14th Annual California ALS Research Summit on January 19, 2024

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January 30, 2024

AcuraStem Leverages iNeuroRx® Platform to Drive Innovative SYF2 and UNC13A Treatments Forward Toward Clinical Trial

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September 25, 2023

Agreement enables the development of therapeutics, including AcuraStem’s AS-202 Candidate, an antisense oligonucleotide that suppresses levels of the PIKFYVE kinase

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June 15, 2023

AcuraStem, a patient-based biotechnology company pioneering how treatments are developed for neurodegenerative diseases, is pleased to announce its sponsorship of the Gordon Research Seminar and Conference on "Amyotrophic Lateral Sclerosis (ALS) and Related Motor Neuron Diseases Mechanisms, Modeling and Translational Therapeutics."

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May 29, 2023

Join AcuraStem's CEO, Sam Alworth, at the forthcoming 2023 BIO International Convention in Boston from June 5-8, 2023. AcuraStem is at the cutting edge of developing treatments for neurodegenerative diseases, including sporadic Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD).

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April 10, 2023

AcuraStem's CEO, Sam Alworth, is set to present on the company’s latest advances in the development of a PIKFYVE antisense oligonucleotide (ASO) therapeutic for diverse forms of FTD (FTD) at the Alzheimer's Drug Discovery Foundation's (ADDF) Defeating Dementia Symposium on April 13.

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March 20, 2023

AcuraStem’s progress in advancing an antisense oligonucleotide (ASO) targeting PIKFYVE and development of therapeutic candidates targeting SYF2 in preclinical studies has been featured in a newly posted BioSpace article.

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March 20, 2023

Fierce Biotech featured AcuraStem's pioneering work with PIKFYVE inhibitors in a recent article highlighting promising developments in treating both genetic and sporadic forms of ALS. The article discusses two recent studies led by Dr. Justin Ichida's lab at USC and the AcuraStem team that have identified drug candidates with potential to transform the disease course for a larger patient population beyond those with known genetic causes of ALS.

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March 20, 2023

A recent study published in the journal Cell Stem Cell and co-authored by AcuraStem CEO Sam Alworth, M.S., MBA, and AcuraStem co-founder Justin Ichida, Ph.D., et al., shows that suppressing the gene encoding spliceosome-associated factor SYF2 may alleviate symptoms of Amyotrophic Lateral Sclerosis (ALS) and improve neuron survival in both sporadic and genetic forms of the disease.

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January 20, 2023

AcuraStem announced today that CEO Sam Alworth, will present on the company’s latest advances in the development of a PIKFYVE antisense oligonucleotide (ASO) therapeutic for diverse forms of ALS and FTD at the 13th annual California ALS Research Summit

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December 5, 2022

Dr. Wen-Hsuan Chang, AcuraStem’s Head of Target Validation to present at the 33nd 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.

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November 4, 2022

Team AcuraStem is honored to continue its partnership with Team Nanci and the ALS Association Golden West Chapter in the fight against ALS as a sponsor of the Greater Los Angeles Walk to Defeat ALS® on Sunday, November 6, at Exposition Park in Los Angeles.

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December 15, 2021

AcuraStem (acurastem.com), a patient-based drug discovery platform company developing novel therapeutics for amyotrophic lateral sclerosis (ALS) and neurodegenerative diseases, announced the results of extensive preclinical studies on its lead program, AS-202, for ALS patients. The data were presented at the Motor Neuron Disease Association 32nd International Symposium on ALS/MND on December 9, by AcuraStem’s Head of Research, Wen-Hsuan Chang, Ph.D.

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December 6, 2021

AcuraStem will present posters at the 32nd International Symposium on ALS/MND.

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June 28, 2021

The CMT Research Foundation today announced it has partnered with AcuraStem, a patient-based drug discovery platform company, and Dr. Alessandra Bolino, to test a new drug to fight  CMT4B1. Dr. Alessandra Bolino, who will lead the research and testing, is a renowned expert in CMT and head of the Human Inherited Neuropathies Unit at the prestigious research hospital Ospedale San Raffaele in Milan. 

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May 25, 2021

AcuraStem has received an Amyotrophic Lateral Sclerosis Research Pro-gram (ALSRP) Therapeutic Development Award from the Department of Defense, Congressionally Directed Medical Research Programs (CDMRP) totaling $1 million.

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September 21, 2020

“We are excited to add our collaborator Dr. Robert Bowser to our advisory board. Bob is a worldwide leader in ALS biomarker development. His expertise will be critical as we look to validate key, CNS-specific, mechanistic biomarkers for our PIKFYVE programs,” said Sam Alworth, co-founder and chief executive officer of AcuraStem.

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June 22, 2020

Sam Alworth, has been invited to present at Translational Solutions’ Virtual Investor...

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February 4, 2020

The California ALS Research Summit held on Jan 24-25, 2020 was the tenth annual gathering of researchers, investigators, clinicians, biotech companies, government representatives, partner organizations, and advocates in ALS and related fields in the State of California. AcuraStem was proud to be on hand as mentor sponsors.

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November 8, 2019

Nov 8-10, 2019, Shanghai, China: Co-Founders Sam Alworth and Dr. Qing Liu will be speaking on November 9th, 2019 at this prestigious summit for innovators on the "Future of Drug Discovery for CNS Indications."

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November 7, 2019

AcuraStem was honored to once again partner with Team Nanci and the ALS Association Golden West Chapter in the fight against ALS as a sponsor of the 17th Annual Los Angeles County Walk to Defeat ALS® on Sunday, November 3, at Exposition Park in Los Angeles.

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November 3, 2019

Nov 3rd, 2019, Walk to Defeat ALS, Golden West Chapter: Team AcuraStem is fundraising for Team Nanci and the whole team will walk in LA on November 3rd. Come by our booth!

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October 26, 2019

October 26th, 2019, UCLA California NanoSystems Institute: CEO, Co-Founder Sam Alworth Presenting at the 5th Annual BCLA Biotech Summit "Mapping out LA’s Biotech Landscape"

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October 22, 2019

AcuraStem announced today that the company has triggered Phase II funding worth $3 million from its Fast Track Small Business Innovation Research (SBIR) grant awarded by the National Institute of Neurological Disorders and Stroke (NINDS), part of the National Institutes of Health (NIH).

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September 18, 2019

One of the compounds in a series under development by AcuraStem for ALS, was administered to a neuronal cell line derived from a patient with CMT2A, and showed improved cell survival resulting in the identification as a promising new, preclinical drug candidate for CMT2A

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January 15, 2019

MDA has awarded an MVP grant totaling $300,000 over two years to AcuraStem to support preclinical development of a novel small molecule therapeutic for ALS.

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December 20, 2018

CMT Research Foundation announced today it has entered into a collaborative research partnership with AcuraStem to test thousands of compounds aimed at producing effective treatments for Charcot-Marie-Tooth disease.

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November 8, 2018

Academy Award-winning actress Renee Zellweger encourages the audience to learn more about AcuraStem’s research work in amyotrophic lateral sclerosis (ALS) at the 16th Annual ALS Association Walk to Defeat ALS at Exposition Park in Los Angeles

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November 2, 2018

AcuraStem will join in the fight against ALS as a sponsor of the 16th Annual Los Angeles County Walk to Defeat ALS® on Sunday, November 4, at Exposition Park in Los Angeles. The team will walk with “Team Nanci” and be on hand at the AcuraStem booth to talk to patients and families affected by Amyotrophic Lateral Sclerosis (ALS) about its mission and research underway.

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August 30, 2018

AcuraStem has announced the members of its Scientific Board of Advisors comprised of PhDs, professors, tech entrepreneurs, and successful drug company veterans.

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July 27, 2018

AcuraStem announces the addition of Monica Zhou, Ph.D. to their team as Principal Scientist. Zhou is in charge of industrializing the expansion of AcuraStem’s groundbreaking iNeuroRx platform and its multiple applications, ensuring their accuracy and efficiency.

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June 18, 2018

AcuraStem a fast-growing and innovative biotech company located in Monrovia, California, has been awarded a $3.7 million Small Business Innovation Research (SBIR) Fast-Track grant (#R44NS105156) by the National Institute of Neurological Disorders and Stroke (NINDS) to continue research for the development of a small molecule therapeutic, “AS2015”, focused on treating patients with the genetic form of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) caused by expansion repeats in the gene C9ORF72.

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May 22, 2018

AcuraStem is pleased to announce that it has been selected as a finalist in the 2018 Start-Up Stadium event at the BIO International Convention, June 4-7 in Boston.

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April 20, 2018

Dr. Justin K. Ichida was recently profiled by Seema Sharma with Mendeley as a part of their series on stem cells and regenerative medicine. In the interview Dr. Ichida discusses his research, career path, and shares advice for those seeking a transition from postdoctoral to tenure-track.

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February 22, 2018

In a recently published article for The Hollywood Reporter, Acurastem co-founder Dr. Justin Ichida shares a personal account of how famed publicist and ALS patient Nanci Ryder, and her friends in the fight, have helped to accelerate groundbreaking in vitro research being done at his USC-based laboratory (results of which were published in a recent Nature Medicine article).

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February 13, 2018

AcuraStem co-founder Justin Ichida co-authored the groundbreaking research paper "Haploinsufficiency leads to neurodegeneration in C9ORF72 ALS/FTD human induced motor neurons," published in Nature Medicine February 5.

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December 5, 2017

This two-day event, which immediately precedes Rare Disease Day on February 28, 2018, will be held at the Icagen Oro Valley location (North of Tucson). The Symposium will feature presentations from a mix of academic and industry speakers from the pharmaceutical and biotechnology communities as well as key opinion leaders from foundations committed to the identification, treatment, and cure of rare diseases.

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December 4, 2017

The award recognizes Dr. Ichida's "pioneering work to convert patients’ skin cells into disease-affected neural cells, enabling them to screen thousands of drug-like compounds in search of potential therapeutics that could slow or stop ALS."

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December 1, 2017

AcuraStem has been awarded a $225 thousand Phase I SBIR grant by the National Institute on Aging (NIA), part of the National Institutes of Health. The grant will fund continued research of AcuraStem’s laboratory models of age-associated ALS, supporting the evaluation of different molecular methods to create motor neurons in the laboratory that more accurately reproduce the ALS disease state in aged patients.

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November 16, 2017

The Governor’s Office of Business and Economic Development (GO-Biz) announced that the California Competes Tax Credit (CCTC) committee approved $69 million in tax credits for 90 companies projected to create 6,236 jobs and make $1.2 billion worth of new capital equipment investments across California.

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October 16, 2017

At the 15th Annual LA County Walk to Defeat ALS on October 15, 2017, Justin walked as a member of Team Nanci, alongside teammates Renee Zellweger, Courtney Cox, Matt Bomer, and others.

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