AcuraStem, a pioneering patient-based biotechnology company specializing in neurodegenerative disease treatments, has announced a groundbreaking partnership with Takeda to develop and commercialize targeted therapeutics for Amyotrophic Lateral Sclerosis (ALS) and related conditions. The centerpiece of this collaboration is AS-202, an innovative antisense oligonucleotide (ASO) designed to combat ALS by targeting the PIKFYVE kinase.


A Novel Approach to Treating ALS

PIKFYVE represents a novel therapeutic target for ALS, and potentially Frontotemporal Dementia (FTD) as well. AcuraStem’s unique therapeutic strategy focuses on combating neurodegeneration by eliminating toxic protein aggregates and safeguarding healthy neuronal function. This innovative approach originated from research conducted by AcuraStem’s co-founder, Justin Ichida, PhD, who utilized patient-derived disease models. The technology, exclusively licensed to AcuraStem by the USC Stevens Center for Innovation, was further developed using AcuraStem’s iNeuroRx® disease modeling platform.

The research showed that ASO-mediated suppression of PIKFYVE could restore motor function, reduce neurodegeneration, and enhance survival in multiple in vivo models of both ALS and FTD.


A Transformative Partnership

Under the terms of the agreement, Takeda gains an exclusive worldwide license to AcuraStem’s PIKFYVE program. In return, AcuraStem will receive upfront and milestone payments potentially amounting to approximately $580 million if all future clinical, regulatory, and commercial milestones are met during the agreement’s term. Additionally, AcuraStem will be entitled to tiered royalties on potential net sales of any commercial products arising from this license.

Sam Alworth, CEO of AcuraStem, expressed enthusiasm about the partnership, stating, “Our mission at AcuraStem is to get our promising therapeutics to the patients who need them as quickly as possible. We are delighted to have secured a partner in Takeda who has world-class clinical development and commercial capabilities to deliver on our mission.”


Advancing ALS Treatment Together

AcuraStem will assume responsibility for specific activities related to advancing AS-202 IND enabling studies and characterizing potential backup ASOs. Meanwhile, Takeda will take charge of all other development activities, including clinical development, regulatory affairs, and global commercialization.

Sarah Sheikh, Head of the Neuroscience Therapeutic Area Unit at Takeda, expressed the company’s commitment to advancing treatment for neurological diseases like ALS. Sheikh highlighted AS-202’s potential to address the unmet needs of patients by targeting the pathological hallmark of ALS and other related conditions. The treatment’s unique dual mechanism of action addresses TDP-43 aggregation and improves TDP-43 function, making it a promising candidate for ALS therapy.


AcuraStem’s Dedication to Neurodegenerative Disease Research

Founded in 2016, AcuraStem remains dedicated to enhancing its patient-based iNeuroRx® technology platform. The company focuses on discovering more effective therapeutic approaches for neurodegenerative indications while swiftly advancing therapeutic programs, including existing programs like SYF2 and UNC13A for ALS and FTD.

Justin Ichida, PhD, co-founder of AcuraStem, commented on the partnership, stating, “AcuraStem has done an outstanding job of generating best-in-class molecules to target PIKFYVE in humans, and I am excited to see this move forward.” The collaboration between AcuraStem and Takeda promises to bring much-needed hope to patients suffering from ALS and related conditions, offering a potential breakthrough in treatment.

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PackGene is a CRO & CDMO technology company that specializes in packaging recombinant adeno-associated virus (rAAV) vectors. Since its establishment in 2014, PackGene has been a leader in the AAV vector CRO service field, providing tens of thousands of custom batches of AAV samples to customers in over 20 countries. PackGene offers a one-stop CMC solution for the early development, pre-clinical development, clinical trials, and drug approval of rAAV vector drugs for cell and gene therapy (CGT) companies that is fast, cost-effective, high-quality, and scalable. Additionally, the company provides compliant services for the GMP-scale production of AAVs and plasmids for pharmaceutical companies, utilizing five technology platforms, including the π-Alpha™ 293 cell AAV high-yield platform and the π-Omega™ plasmid high-yield platform. PackGene’s mission is to make gene therapy affordable and accelerate the launch of innovative gene drugs. The company aims to simplify the challenging aspects of gene therapy development and industrialization processes and provide stable, efficient, and economical rAAV Fast Services to accelerate gene and cell therapy development efforts from discovery phase to commercialization.

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