What Vico Therapeutics does

Vico Therapeutics B.V. is a clinical-stage genetic medicines company founded in 2019 and headquartered in Leiden, the Netherlands. It develops antisense oligonucleotide (ASO) therapies that target the CAG trinucleotide repeat expansion mutation underlying polyglutamine (polyQ) diseases — a group of nine rare neurodegenerative disorders including Huntington's disease (HD) and the spinocerebellar ataxias SCA1 and SCA3. The company is currently pre-revenue.

The company's core platform, VICOMER, is an oligonucleotide-based RNA modulation engine that designs ASOs capable of inhibiting translation, modulating splicing, editing RNA, degrading transcripts, or activating expression depending on the genetic defect targeted. Its lead clinical candidate, VO659, is an allele-preferential ASO designed to reduce mutant huntingtin protein while preserving wild-type protein function, and is positioned as the only clinical-stage candidate directly targeting the pathogenic CAG repeat expansion shared across all nine polyQ diseases. VO659 has demonstrated a 38% reduction in cerebrospinal fluid mutant huntingtin (mHTT) and a 2.5% reduction in neurofilament light chain (Nf-L) at four months, with a long half-life supporting potential twice-yearly intrathecal dosing. Beyond VO659, the pipeline includes preclinical/early programs for Rett syndrome (MECP2), familial Alzheimer's disease, and three undisclosed CNS programs.

Vico's business model is pre-commercial R&D funded by venture capital. To date it has raised approximately €65.8 million (~$84 million) across Series A (~$31M, July 2020) and Series B (€54M in January 2024 plus an €11.5M second closing in June 2024). The lead asset is being advanced in a multi-country European Phase 1/2a basket trial in HD, SCA1, and SCA3 (target enrollment ~71 participants, NCT05822908), with US expansion enabled by FDA IND clearance in February 2026. The company holds both FDA and EMA Orphan Drug Designations for VO659 in Huntington's disease. Customers at the development stage are clinical trial investigators and specialized neurology centers, with the end market being patients with rare neurodegenerative disorders — a population with no approved disease-modifying therapies.

Name

Vico Therapeutics

Legal name

Vico Therapeutics B.V.

Website

https://vicotx.com

Company type

Private

Founded year

2019

Operating status

Operating

Headcount range

11–50 employees

Short description

Vico Therapeutics is a clinical-stage Dutch biotech developing antisense oligonucleotide (ASO) therapies that target the CAG repeat expansion mutation causing polyglutamine diseases, with lead candidate VO659 in Phase 1/2a trials for Huntington's disease and spinocerebellar ataxias.

Ownership category

akta.pro rank

Product category

Rare Neurological Genetic Medicines

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Scientific Research and Development Services (5417)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

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

akta.pro secondary industries

Rare Neurology & Neurodegenerative Disorder Therapies (HLAIAIAD), RNA Therapeutics Clinical Development (trial design, endpoints, patient stratification) (HLAAADAH), RNA Therapeutics Regulatory, Quality & Pharmacovigilance (GxP, comparability, post-market) (HLAAADAJ)

Keywords

• Antisense oligonucleotide therapies
• RNA modulating therapeutics
• Polyglutamine disease treatment
• Rare neurological drug development
• Genetic medicine platform

Headquarters

HQ city

Leiden

HQ country

Netherlands

HQ region

Europe

Offices1 record

Markets served

GTM type

B2C

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Pre-revenue / Pipeline Development Stage: Vico Therapeutics is a clinical-stage genetic medicines company that is pre-revenue. The company is funded through venture capital financing rounds (Series B) to advance its pipeline of ASO therapies. No commercialized products or revenue-generating activities are described.

Go-to-market motion1 record

Distribution channels2 records

Marketing channels4 records

Core offering

Vico Therapeutics is a clinical-stage genetic medicines company developing antisense oligonucleotide (ASO) RNA-modulating therapies for rare genetic neurological diseases. Its lead investigational therapy, VO659, is an allele-preferential ASO designed to directly target the CAG repeat expansion mutation that causes all nine known polyglutamine diseases, including Huntington's disease, spinocerebellar ataxia type 1 (SCA1), and spinocerebellar ataxia type 3 (SCA3). The company also operates the VICOMER discovery platform and additional pipeline programs in Rett syndrome, familial Alzheimer's disease, and undisclosed CNS indications.

Product overview

Vico Therapeutics is a clinical-stage genetic medicines company with a unified therapeutic platform focused on RNA-modulating antisense oligonucleotide (ASO) therapies for rare genetic neurological diseases. The company's lead product is VO659, currently in Phase 1/2a clinical development for Huntington's disease, SCA1, and SCA3. The VICOMER platform serves as the foundational technology enabling the design of fit-for-purpose ASOs for various genetic defects. The pipeline includes programs for Rett Syndrome and Familial Alzheimer's Disease, plus three undisclosed CNS programs.

Differentiator

Problem solved

Functional benefit

Products and services

• VO659 VO659 is Vico's lead allele-preferential antisense oligonucleotide (ASO) investigational therapy designed to directly target the CAG repeat expansion mutation that causes all nine known polyglutamine diseases. It selectively reduces disease-causing mutant huntingtin (HTT), ATXN1, and ATXN3 proteins while preserving wild-type protein function, and is currently in Phase 1/2a clinical development for Huntington's disease, SCA1, and SCA3. The therapy is administered intrathecally and is being explored at a long-acting twice-annual dosing regimen. VO659 has received both FDA and EMA Orphan Drug Designation for Huntington's disease.

Quantifiable outcome

• 38% reduction in cerebrospinal fluid mutant huntingtin protein (CSF mHTT) and 2.5% reduction in CSF Nf-L in HD patients dosed with VO659 at four months
• +3 more outcomes

Segments3 records

Ideal customer profiles1 record

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Two partnerships are on record, tiered core.

• CHDI FoundationcoreStrategic or Co-development PartnerCHDI Foundation is a nonprofit biotech that collaborates with Vico on Huntington's disease research. Vico's CSO and CMO presented VO659 data at CHDI's 21st Annual HD Therapeutics Conference. CHDI supports HD therapeutic development through scientific collaboration.
• European Huntington's Disease Network (EHDN)coreStrategic or Co-development PartnerVico presented positive interim Phase 1/2a clinical data at the EHDN & Enroll-HD 2024 meeting, highlighting first clinical data for VO659 in Huntington's disease treatment.

Scale indicators5 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Wave Life Sciences: Clinical-stage ASO developer with multiple allele-selective Huntington's disease programs (WVE-003 targeting mHTT, WVE-004 for ALS). Directly comparable to Vico as both pursue allele-preferential ASO approaches in CNS, competing for similar patient populations, KOL relationships, and partnership economics.
• uniQure: Developing AMT-130, an AAV gene therapy for Huntington's disease currently in clinical trials. Competes with Vico in the same disease space with a different modality (gene therapy vs. ASO), targeting the same HTT-lowering mechanism and similar clinical investigator networks.
• Triplet Therapeutics: Was developing ASO therapies targeting the CAG repeat expansion in polyQ diseases, directly overlapping Vico's lead mechanism. Although Triplet shifted strategic direction, it represents the closest historical peer in the specific polyQ repeat-targeting approach.
• Stoke Therapeutics: Clinical-stage company developing antisense oligonucleotides (TANGO platform) for CNS genetic diseases including Dravet syndrome and ADOA. Comparable ASO modality, CNS focus, rare disease positioning, and similar clinical-stage development trajectory.
• ProQR Therapeutics: Clinical-stage Dutch biotech developing Axiomer RNA editing oligonucleotide platform for genetic diseases. Same Leiden/Netherlands origin, comparable oligonucleotide modality, and similar rare disease CNS focus.

Broad incumbents

• Roche (Genentech): Developed tominersen, the most advanced huntingtin-lowering ASO program that halted in Phase 3. As a broad incumbent in CNS and rare disease with substantial resources, Roche represents both the benchmark and the cautionary tale for VO659, with potential interest in acquiring or partnering with Vico given the mechanism's allele-preferential differentiation.
• Ionis Pharmaceuticals: The leading antisense oligonucleotide platform company with multiple approved and clinical-stage ASO drugs (Spinraza, Tegsedi, Waylivra). Represents the gold standard ASO platform with extensive CMC, regulatory, and clinical infrastructure relevant to Vico's ASO development.
• Sage Therapeutics: CNS-focused biotech with Huntington's disease programs in development alongside its approved Zurzuvae. Comparable in CNS rare disease focus and neurology KOL relationships, though Sage pursues small molecules rather than ASOs.
• BioMarin Pharmaceutical: Rare disease biotech where Vico's CSO Nicole Datson previously led drug discovery. BioMarin has CNS rare disease programs and substantial ASO/oligonucleotide experience through partnerships, providing an established incumbent comparable to Vico's specialty rare neurology focus.

Emerging players

• Locanabio: RNA-targeted gene therapy company where Vico's CEO Micah Mackison previously served as CBO. Operates in the same RNA-modulation and genetic neurological disease space, providing a comparable development-stage peer and a connection point in management.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/vico-therapeutics
• Xx.com/VicoThera
• Facebookfacebook.com/Vico-Therapeutics-BV-116309576584596

Compliance2 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles4 records

Funding overview

Funding rounds3 records

Investors11 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 Vico Therapeutics