Actio Biosciences
Actio Biosciences is a clinical-stage biotechnology company using a proprietary genetics platform to develop oral small molecule precision medicines for rare Mendelian diseases, with two clinical-stage programs targeting KCNT1-related epilepsy and Charcot-Marie-Tooth disease type 2C.
- Company typePrivate
- Founded2021
- HeadquartersSan Diego, United States
- Headcount11–50
- GTM typeB2B
- OfferingHardware or Manufacturing
What Actio Biosciences does
Actio Biosciences is a clinical-stage biotechnology company founded in October 2021 in San Diego, California, developing precision small molecule therapeutics for rare and genetically defined diseases. The company is built around its proprietary Rare Disease Target Atlas, a human genetics platform that combines Mendelian genetics, bioinformatics, and medicinal chemistry to identify and validate drug targets derived from rare-disease biology, then expand those insights into more prevalent indications under what Actio calls its "One to Many" strategy. Its pipeline comprises three programs: ABS-1230, an oral KCNT1 inhibitor currently in the KYRON Phase 1b/2 trial for KCNT1-related epilepsy; ABS-0871, an oral TRPV4 inhibitor that completed Phase 1a in healthy volunteers for Charcot-Marie-Tooth disease type 2C (CMT2C); and a third undisclosed-target discovery program in genetic epilepsy with planned CNS expansion.
The company's discovery engine integrates target identification from rare Mendelian genes, mutation-agnostic small molecule design, transgenic-mouse model characterization under a partnership with The Jackson Laboratory Rare Disease Translational Center, and population-genetics/ML-driven indication expansion. Both lead programs have received FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations, with ABS-1230 also accepted into the FDA's Rare Disease Evidence Principles (RDEP) process. The initial target populations are small—an estimated ~2,500 U.S. patients each for KCNT1-related epilepsy and TRPV4-positive CMT2C—and there are currently no approved disease-modifying therapies for either indication.
Actio is pre-revenue, funded by $121 million in cumulative equity financing: a $55 million Series A in September 2023 led by Canaan and DROIA Ventures, and a $66 million Series B in June 2025 co-led by Regeneron Ventures and Deerfield Management. Future revenue is expected to derive from direct commercialization of pipeline assets in the United States (and via the Australia-based Phase 1a), out-licensing, or partnership transactions with larger pharmaceutical companies; the company currently has no commercial products or pricing model.
Actio Biosciences firmographics
Firmographics- Name
- Actio Biosciences
- Legal name
- Actio Biosciences, Inc.
- Website
- https://www.actiobiosciences.com
- Company type
- Private
- Founded year
- 2021
- Operating status
- Operating
- Headcount range
- 11–50 employees
- Short description
- Actio Biosciences is a clinical-stage biotechnology company using a proprietary genetics platform to develop oral small molecule precision medicines for rare Mendelian diseases, with two clinical-stage programs targeting KCNT1-related epilepsy and Charcot-Marie-Tooth disease type 2C.
- Ownership category
- akta.pro rank
Actio Biosciences industry classification
Industry- Product category
- Rare Disease Therapeutics
- NAICS
- Research and Development in Biotechnology (except Nanobiotechnology) (541714)
- SIC
- Pharmaceutical Preparations (2834)
- akta.pro primary industry
- Rare Neurology & Neurodegenerative Disorder Therapies (HLAIAIAD)
Keywords
Where Actio Biosciences is headquartered
LocationHeadquarters
- HQ city
- San Diego
- HQ country
- United States
- HQ region
- North America
Offices1 record
Markets served
Actio Biosciences business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Operations, Marketing or Sales
Revenue model
- Pre-revenue clinical-stage pipeline: Actio Biosciences is a clinical-stage biotechnology company with no approved or commercialized products. Current revenue model is not yet established; future revenue is expected to derive from commercialization of pipeline assets (ABS-1230, ABS-0871, third program) through direct sales, licensing, or partnerships with larger pharmaceutical companies.
- Capital funding (Series A and B): Operations are funded through equity financing rounds — $55M Series A (Sept 2023) led by Canaan and DROIA Ventures, and $66M Series B (June 2025) co-led by Regeneron Ventures and Deerfield Management, with no current product-based revenue.
Go-to-market motion1 record
Distribution channels2 records
Marketing channels7 records
Actio Biosciences product offering
Product offeringCore offering
Actio Biosciences is a clinical-stage biotechnology company that uses a proprietary human genetics platform called the Rare Disease Target Atlas—combining Mendelian genetics, bioinformatics, and medicinal chemistry—to discover and develop precision small molecule drugs. Its pipeline includes ABS-1230 (a KCNT1 inhibitor for KCNT1-related epilepsy), ABS-0871 (a TRPV4 inhibitor for Charcot-Marie-Tooth disease type 2C), and an undisclosed third genetic-epilepsy program, with plans to expand each rare-disease program into more prevalent indications sharing the same underlying biology.
Product overview
Actio Biosciences is a clinical-stage biotechnology company built around a single genetics-driven precision medicine platform — the proprietary Rare Disease Target Atlas — which combines a vast rare-disease target database with advanced bioinformatics, Mendelian genetics, biology, and medicinal chemistry. On top of this shared platform sit three pipeline modules: ABS-1230 (a KCNT1 inhibitor currently in the KYRON Phase 1b/2 trial for KCNT1-related epilepsy), ABS-0871 (a TRPV4 inhibitor that completed Phase 1a for Charcot-Marie-Tooth disease type 2C), and a third undisclosed-target program in Discovery for genetic epilepsy with CNS expansion. Each clinical-stage module is designed to be developed first in a rare Mendelian indication and then expanded into more prevalent diseases sharing the same underlying biology, an approach Actio calls "from one to many."
Differentiator
Problem solved
Functional benefit
Brands
- KYRON: Name of Actio's Phase 1b/2 clinical trial evaluating ABS-1230 in children and young adults aged one month through 21 years with KCNT1-related epilepsy; also represented by the Kyron trial logo at ActioTrials.com.
Products and services
- ABS-1230 Potent and selective orally available small molecule inhibitor of the KCNT1 potassium ion channel, designed to treat KCNT1-related epilepsy (a rare pediatric developmental epileptic encephalopathy) and being explored for additional genetic epilepsies. Holds FDA Fast Track, Rare Pediatric Disease, and Orphan Drug designations, and was accepted into the FDA Rare Disease Evidence Principles process.
- ABS-0871 First-in-class orally available small molecule inhibitor of the TRPV4 calcium-permeable ion channel being developed initially for Charcot-Marie-Tooth disease type 2C (CMT2C) and TRPV4-related neuromuscular and bone disorders, with potential expansion into overactive bladder. Holds FDA Rare Pediatric Disease, Fast Track, and Orphan Drug designations for CMT2C.
- Program 3 (Undisclosed Target) Third pipeline program at the end of the Discovery phase targeting an undisclosed gene for a rare genetic epilepsy, with planned expansion into other central nervous system (CNS) disorders. Actio owns full rights to the program.
Companies that use Actio Biosciences
Customer profileSegments3 records
Ideal customer profiles2 records
Actio Biosciences technology and API
TechnologyTechnology focussed Yes
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
AI capability4 records
Feature5 records
Actio Biosciences partnerships and signals
Strategic signalPartnerships
Five partnerships are on record, tiered flagship and core.
- The Jackson Laboratory (JAX)flagshipActio established a partnership with the Rare Disease Translational Center at The Jackson Laboratory to develop and implement new transgenic mouse models of rare disease pathways. The partnership enables evaluation of targets and phenotypes of interest in high-quality genetically engineered mouse models at scale, supporting Actio's lead TRPV4 program and broader pipeline.
- Charcot-Marie-Tooth AssociationcoreActio is a STAR Alliance Partner of the Charcot-Marie-Tooth Association. The collaboration supported ABS-0871's receipt of FDA Orphan Drug and Rare Pediatric Disease Designations in August 2024 via the CMT Association's Patients as Partners platform, and continues to support patient outreach for ABS-0871's clinical development in CMT2C.
- KCNT1 Epilepsy FoundationcoreAdvocacy partnership with the KCNT1 Epilepsy Foundation to support patient education, family resources and recruitment for ABS-1230 clinical trials. The Foundation's co-founder Justin West, M.D., has publicly endorsed Actio's KYRON Phase 1b/2 trial.
- Ben Cravatt, Ph.D. (Scripps Research Institute)coreProfessor and Norton B. Gilula Chair of Chemical Biology at The Scripps Research Institute; joined Actio's Board of Directors in May 2024. Brings chemical proteomics expertise aligned with Actio's biology-chemistry-genetics integration.
- Emil Kakkis, M.D., Ph.D. (Ultragenyx)coreCEO, president and director of Ultragenyx; joined Actio's Board of Directors in July 2024. Brings rare disease drug development expertise and longstanding advocacy through the EveryLife Foundation for Rare Diseases.
Scale indicators9 records
Recent moves6 records
Expansion highlights6 records
Actio Biosciences competitors and assessment
Company assessmentDirect peers
- Maze Therapeutics: Clinical-stage genetics-driven biotech that translates insights from rare genetic variants into small molecule therapies for both rare and common diseases - directly comparable to Actio's 'One to Many' Mendelian-genetics platform approach.
- BridgeBio Pharma: Genetic-disease focused biotech building multiple precision medicine programs from genetically validated targets, including in rare epilepsies and rare neuromuscular disease - directly comparable to Actio's genetically validated small molecule pipeline.
- Praxis Precision Medicines: Clinical-stage company developing therapies for genetic epilepsies and other CNS disorders driven by defined genetic mutations - directly comparable to Actio's KCNT1 program and broader genetic epilepsy pipeline.
- Xenon Pharmaceuticals: Clinical-stage company developing treatments for epilepsy and other neurological disorders, including genetically defined epilepsies - directly comparable to Actio's KCNT1-related epilepsy program and ABS-1230.
Broad incumbents
- Ultragenyx Pharmaceutical: Established commercial-stage rare disease company led by Emil Kakkis (on Actio's board) with multiple approved and clinical-stage therapies for rare genetic diseases - provides a mature comparable for Actio's rare disease development model and commercial pathway.
- BioMarin Pharmaceutical: Large commercial rare-disease biotech with multiple approved therapies for ultra-rare genetic conditions, including rare neuromuscular disorders - directly comparable to Actio's CMT2C (TRPV4) program in target patient profile and regulatory approach.
Emerging players
- CAMP4 Therapeutics: Emerging clinical-stage company using regulatory genomics to develop RNA-based therapeutics for rare genetic diseases - comparable to Actio in platform-driven rare disease strategy and stage.
- Stoke Therapeutics: Clinical-stage company developing antisense oligonucleotide medicines for genetic epilepsies and other rare genetic diseases - closely comparable in patient population and disease area to Actio's genetic epilepsy pipeline.
- Ionis Pharmaceuticals: Established antisense RNA therapeutics company with multiple rare disease programs including in rare neuromuscular disorders and genetic neurological diseases - adjacent comparable in modality and indication overlap with Actio's TRPV4 and KCNT1 programs.
Others
- Regeneron Pharmaceuticals: Large biopharmaceutical company whose Regeneron Ventures arm co-led Actio's Series B; Regeneron is a leading developer of genetics-based therapeutics and a natural strategic acquirer or partner for Actio's pipeline.
Market position
Strengths5 records
Weaknesses5 records
Competitive moat5 records
Key risks5 records
Key highlights7 records
Customer concentration
Actio Biosciences social profiles
Digital presenceActio Biosciences compliance and trust
Trust signalCompliance8 records
Actio Biosciences financial estimates
Financial estimateRevenue estimate
Valuation estimate
Actio Biosciences leadership team
Management profileNumber of profiles
Profiles10 records
Actio Biosciences funding detail
Funding detailFunding overview
Funding rounds4 records
Investors7 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Actio Biosciences 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 Actio Biosciences
What does Actio Biosciences do?
Actio Biosciences is a clinical-stage biotechnology company that uses a proprietary human genetics platform called the Rare Disease Target Atlas—combining Mendelian genetics, bioinformatics, and medicinal chemistry—to discover and develop precision small molecule drugs. Its pipeline includes ABS-1230 (a KCNT1 inhibitor for KCNT1-related epilepsy), ABS-0871 (a TRPV4 inhibitor for Charcot-Marie-Tooth disease type 2C), and an undisclosed third genetic-epilepsy program, with plans to expand each rare-disease program into more prevalent indications sharing the same underlying biology.
Is Actio Biosciences a public or private company?
Actio Biosciences is a private company. It is classified as venture growth investor backed and is currently operating.
When was Actio Biosciences founded?
Actio Biosciences was founded in 2021. It employs 11 to 50 people.
Where is Actio Biosciences based?
Actio Biosciences is headquartered in San Diego, United States, in the North America region.
How does Actio Biosciences make money?
Two revenue lines are on record. Pre-revenue clinical-stage pipeline is the primary driver. The others are capital funding (Series A and B).
Who are Actio Biosciences's main competitors?
Direct peers on record are Maze Therapeutics, BridgeBio Pharma, Praxis Precision Medicines and Xenon Pharmaceuticals. Broad incumbents are Ultragenyx Pharmaceutical and BioMarin Pharmaceutical. Emerging players are CAMP4 Therapeutics, Stoke Therapeutics and Ionis Pharmaceuticals. Regeneron Pharmaceuticals is listed as an others.
Does Actio Biosciences have an API?
No public API is recorded for Actio Biosciences.
What industry is Actio Biosciences in?
Actio Biosciences's product category is Rare Disease Therapeutics. Its primary akta.pro industry code is HLAIAIAD, Rare Neurology & Neurodegenerative Disorder Therapies. Its NAICS code is 541714 and its SIC code is 2834.