AcuraStem
- Company typePrivate
- Founded2016
- HeadquartersPasadena, United States
- Headcount11–50
- GTM typeB2B
- OfferingServices
AcuraStem firmographics
Firmographics- 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
AcuraStem industry classification
Industry- 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
Where AcuraStem is headquartered
LocationHeadquarters
- HQ city
- Pasadena
- HQ country
- United States
- HQ region
- North America
Offices2 records
Markets served
AcuraStem business model
Business model- GTM type
- B2B
- Offering type
- Services
- Cost components
- Personnel, Technology or R&D, Operations, Marketing or Sales, Infrastructure
Revenue model
- 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.
- 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
AcuraStem product offering
Product offeringCore 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
Companies that use AcuraStem
Customer profileSegments3 records
Ideal customer profiles3 records
AcuraStem technology and API
TechnologyAPI detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
AI capability2 records
Feature5 records
AcuraStem partnerships and signals
Strategic signalPartnerships
Nine partnerships are on record, tiered core, major and minor.
- Target ALScoreTwo-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 PharmaceuticalscoreLicense 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)coreCo-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 UniversitymajorCollaboration with Philip C. Wong lab on TDP-43-dependent RNA splicing research and fluid biomarker development for SYF2-targeted therapeutics.
- Mayo Clinic JacksonvillemajorCollaboration with Wilfried Rossoll lab on cutting-edge proteomics and neuroproteostasis expertise focused on TDP-43 proteinopathy for SYF2 program.
- Barrow Neurological InstitutemajorRobert Bowser serves on Scientific Advisory Board; co-director of national ALS biorepository and post-mortem tissue bank supporting research efforts.
- CMT Research FoundationminorPartnership 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 PartnersminorTransaction advisor for Takeda licensing agreement.
- Foley HoagminorLegal counsel to AcuraStem for Takeda licensing agreement.
Scale indicators4 records
Recent moves7 records
Expansion highlights6 records
AcuraStem competitors and assessment
Company assessmentDirect 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
AcuraStem social profiles
Digital presenceAcuraStem financial estimates
Financial estimateRevenue estimate
Valuation estimate
AcuraStem leadership team
Management profileNumber of profiles
Profiles13 records
AcuraStem subsidiaries and ownership
Company hierarchySubsidiaries1 record
AcuraStem funding detail
Funding detailFunding overview
Funding rounds10 records
Investors8 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
AcuraStem M&A and investment
M&A and investmentM&A
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about AcuraStem
What does AcuraStem do?
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.
Is AcuraStem a public or private company?
AcuraStem is a private company. It is classified as founder individual operated bootstrapped and is currently operating.
When was AcuraStem founded?
AcuraStem was founded in 2016. It employs 11 to 50 people.
Where is AcuraStem based?
AcuraStem is headquartered in Pasadena, United States, in the North America region.
How does AcuraStem make money?
Two revenue lines are on record. Licensing and Milestone Payments are the primary driver. The others are grant Funding.
Who are AcuraStem's main competitors?
Direct peers on record are Ionis Pharmaceuticals and Wave Life Sciences. Broad incumbents are Biogen, Denali Therapeutics and Alector. Emerging players are QurAlis, ProMIS Neurosciences, Passage Bio and Entrada Therapeutics. Sage Therapeutics is listed as a regional player.
Does AcuraStem have an API?
No public API is recorded for AcuraStem.
What industry is AcuraStem in?
AcuraStem's product category is Neurological Therapeutics / Neurodegenerative Drug Discovery. Its primary akta.pro industry code is HLAAADAC, Antisense Oligonucleotides (ASO) Therapeutics (splice modulation/knockdown), with a secondary code of HLAIACAC, Neurology & CNS Specialty Pharmaceuticals. Its NAICS code is 541714 and its SIC code is 8731.