AcuraStem

Name

AcuraStem

Legal name

AcuraStem Incorporated

Website

https://acurastem.com

Company type

Private

Founded year

2016

Operating status

Operating

Headcount range

11–50 employees

Ownership category

akta.pro rank

Product category

Neurological Therapeutics / Neurodegenerative Drug Discovery

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Antisense Oligonucleotides (ASO) Therapeutics (splice modulation/knockdown) (HLAAADAC)

akta.pro secondary industries

Neurology & CNS Specialty Pharmaceuticals (HLAIACAC), Neurology & Psychiatry (CNS) Pharmaceuticals (HLAIAAAF)

Keywords

• Antisense oligonucleotide therapeutics
• Neurodegenerative disease drug discovery
• Patient-derived disease models
• ALS FTD therapeutics
• Stem cell drug discovery

Headquarters

HQ city

Pasadena

HQ country

United States

HQ region

North America

Offices2 records

Markets served

GTM type

B2B

Offering type

Services

Cost components

Personnel, Technology or R&D, Operations, Marketing or Sales, Infrastructure

Revenue model

1. Licensing and Milestone Payments: AcuraStem generates revenue through licensing agreements with pharmaceutical partners. The company received milestone payments totaling up to $580 million from Takeda for PIKFYVE therapeutics including AS-202, plus tiered royalties on potential net sales of commercial products.
2. Grant Funding: Pre-commercial biotech company funded primarily through research grants from government agencies and foundations including NIH, DOD, CIRM, Target ALS, and others. Multiple grants supporting ALS and FTD research programs.

Go-to-market motion1 record

Distribution channels2 records

Marketing channels5 records

Core offering

AcuraStem is a patient-based biotechnology company developing antisense oligonucleotide (ASO) therapeutics for neurodegenerative diseases, primarily amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Its proprietary iNeuroRx® platform generates patient-derived neurons from blood or skin biopsies to recapitulate human disease processes and discover broadly-acting treatments targeting TDP-43 pathology.

Product overview

AcuraStem is a patient-based biotechnology company developing a portfolio of antisense oligonucleotide (ASO) therapeutics for neurodegenerative diseases. The company's core offering centers on the iNeuroRx® disease-modeling technology platform, which enables discovery and development of treatments using patient-derived neurons. Key therapeutic programs include: AS-241 (lead UNC13A-targeted clinical candidate for ALS/FTD), AS-202 (PIKFYVE-targeted ASO licensed to Takeda), and SYF2-targeted therapeutics. The platform supports both discovery and optimization of ASO candidates across multiple therapeutic targets.

Differentiator

Problem solved

Functional benefit

Brands

• iNeuroRx: Proprietary best-in-class disease-modeling technology platform for discovering novel treatments for neurodegenerative diseases using patient-derived neurons

Products and services

• AS-241 Lead clinical candidate antisense oligonucleotide (ASO) targeting UNC13A for the treatment of ALS and FTD. Designed to address TDP-43 pathology by suppressing cryptic exon inclusion and restoring normal UNC13A expression and synaptic function in ALS patient-derived neurons.
• AS-202 (PIKFYVE program) Antisense oligonucleotide drug candidate targeting PIKFYVE kinase for ALS treatment. Addresses neurodegeneration by expelling toxic protein aggregates and restoring healthy neuronal function via multivesicular body exocytosis. Licensed to Takeda for clinical development, regulatory affairs, and global commercialization.
• SYF2-Targeted Therapeutics Program Preclinical antisense oligonucleotide program targeting SYF2, a pre-mRNA splicing factor that addresses both gain and loss of TDP-43 pathology in ALS. SYF2 suppression reverses TDP-43 pathology and improves TDP-43 function in patient-derived neuron models.

Quantifiable outcome

• TDP-43 pathology present in approximately 97% of ALS patients addressed by UNC13A-targeting AS-241 vs only 2% addressed by Qalsody
• +3 more outcomes

Segments3 records

Ideal customer profiles3 records

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

AI capability2 records

Feature5 records

Partnerships

Nine partnerships are on record, tiered core, major and minor.

• Target ALScoreStrategic or Co-development Partner · 10 March 2026Two-year research grant to advance SYF2-targeted therapeutics for ALS. Collaboration with academic investigators including Philip C. Wong (Johns Hopkins University) and Wilfried Rossoll (Mayo Clinic Jacksonville) to understand SYF2 biology and advance medicines toward clinic.
• Takeda PharmaceuticalscoreOEM/ Whitelabel/ Licensing Partner · 25 September 2023License agreement for PIKFYVE-targeted therapeutics including AS-202. Takeda receives exclusive worldwide license; AcuraStem eligible for upfront and milestone payments up to $580 million plus tiered royalties. Takeda responsible for clinical development, regulatory affairs, and global commercialization.
• University of Southern California (USC)coreStrategic or Co-development PartnerCo-founders Justin Ichida and others from USC. PIKFYVE target exclusively licensed from USC Stevens Center for Innovation. Ongoing collaboration with Dr. Ichida's lab on patient-based disease modeling and target discovery.
• Johns Hopkins UniversitymajorStrategic or Co-development PartnerCollaboration with Philip C. Wong lab on TDP-43-dependent RNA splicing research and fluid biomarker development for SYF2-targeted therapeutics.
• Mayo Clinic JacksonvillemajorStrategic or Co-development PartnerCollaboration with Wilfried Rossoll lab on cutting-edge proteomics and neuroproteostasis expertise focused on TDP-43 proteinopathy for SYF2 program.
• Barrow Neurological InstitutemajorStrategic or Co-development PartnerRobert Bowser serves on Scientific Advisory Board; co-director of national ALS biorepository and post-mortem tissue bank supporting research efforts.
• CMT Research FoundationminorStrategic or Co-development PartnerPartnership to test new drugs for Charcot-Marie-Tooth disease (CMT4B1) with Dr. Alessandra Bolino at Ospedale San Raffaele, Milan. Successfully identified preclinical drug candidate for CMT2A.
• Destum PartnersminorOthersTransaction advisor for Takeda licensing agreement.
• Foley HoagminorOthersLegal counsel to AcuraStem for Takeda licensing agreement.

Scale indicators4 records

Recent moves7 records

Expansion highlights6 records

Direct peers

• Ionis Pharmaceuticals: Ionis is the leading antisense oligonucleotide (ASO) platform company and developed Spinraza and, with Biogen, Qalsody for ALS. AcuraStem is developing CNS ASOs using the same modality and targeting the same patient populations, making Ionis the closest direct comparable.
• Wave Life Sciences: Wave is a clinical-stage ASO company developing stereopure oligonucleotides for neurological and rare diseases, including ALS programs. Its PRISM platform competes head-to-head with iNeuroRx-derived ASOs in CNS indications.

Broad incumbents

• Biogen: Biogen co-developed and commercializes Qalsody (tofersen) for SOD1 ALS with Ionis. As an established CNS-focused pharma with the only approved ALS ASO on market, Biogen is the broad incumbent against which AcuraStem's AS-241 will compete for TDP-43-related ALS market share.
• Denali Therapeutics: Denali is a larger, well-capitalized neurodegeneration biotech with multiple CNS programs in clinical development. It is a broader incumbent that targets overlapping neurodegenerative indications, though its modality mix (small molecules and biologics with BBB-delivery technology) differs from AcuraStem's pure ASO focus.
• Alector: Alector is a clinical-stage neuroscience company developing immuno-neurology therapeutics for ALS, FTD, and Alzheimer's. Although its modality (antibodies) differs, it competes for the same neurodegenerative patient populations and partnership interest.

Emerging players

• QurAlis: QurAlis is a clinical-stage biotech focused specifically on ALS and FTD, developing ASO and small-molecule therapeutics that target TDP-43 and other ALS mechanisms. Like AcuraStem, it pursues broad-population ALS therapies rather than single-gene approaches.
• ProMIS Neurosciences: ProMIS develops antibody and ASO therapeutics for ALS, FTD, and Alzheimer's centered on TDP-43 and other misfolded proteins. It is an emerging player with a directly overlapping TDP-43 thesis and similar pipeline stage.
• Passage Bio: Passage Bio is a clinical-stage genetic medicines company developing AAV-delivered therapies for CNS disorders including FTD/ALS-spectrum indications. It is an emerging player with overlapping neurodegenerative disease focus but using gene therapy rather than ASOs.
• Entrada Therapeutics: Entrada develops intracellular delivery-enabled therapeutics (oligonucleotides and proteins) for neuromuscular diseases including Duchenne and potentially ALS/FTD. Its EEV platform is complementary to ASO delivery and a partial competitor for the same patient base.

Regional players

• Sage Therapeutics: Sage is a CNS-focused biotech that has developed and commercialized CNS therapies (Zurzuvae). While focused on depression/epilepsy rather than ALS/FTD, it is a relevant public-market comparable for CNS biotech valuation and partnership dynamics.

Market position

Strengths4 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• Xx.com/acurastem
• Facebookfacebook.com/AcuraStem
• LinkedInlinkedin.com/company/12903559

Revenue estimate

Valuation estimate

Number of profiles

Profiles13 records

Subsidiaries1 record

Funding overview

Funding rounds10 records

Investors8 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about AcuraStem