What Lario Therapeutics does

Lario Therapeutics is a privately held, preclinical biopharmaceutical company founded in 2021 as a spin-out from Epidarex Exeed, the therapeutic discovery engine of Epidarex Capital. The company is headquartered in Edinburgh, UK (Company Number SC716204) with a team of approximately 8 employees and operates as an independent private limited company with seed investment from Epidarex Capital and Golgi Neurosciences (the latter following a transfer from Axxam).

Lario develops first-in-class precision medicines in the form of selective small-molecule inhibitors of voltage-gated neuronal calcium channels—specifically CaV1.3, CaV2.3, and CaV2.1—targeting severe neurological disorders including developmental and epileptic encephalopathies (DEE69, CDKL5-deficiency disorder), drug-refractory epilepsies, Parkinson's disease, PTSD, genetic hemiplegic migraine, and autism-related syndromes. Its pipeline rests on genetically validated targets with disease-modifying intent rather than symptomatic mechanisms, and the lead CaV2.3 programme is positioned to enter IND-enabling studies in 2026. The company collaborates with the Oxford Parkinson's Disease Centre on Parkinson's biology and engages with patient-advocacy organisations such as CACNA1E International and the Loulou Foundation's CDKL5 Forum.

The business is purely R&D-stage and pre-commercial; there are no approved products, no pricing model, and no commercial customers. Lario is funded entirely through non-dilutive grants rather than equity or product revenue—chiefly a $6 million Michael J. Fox Foundation award in 2024 and a combined $2.4 million ($1.5M MJFF + $0.9M/£700K Wellcome) award in February 2026—and plans to monetise its pipeline post-IND through pharmaceutical licensing or co-development agreements with larger commercial partners.

Name

Lario Therapeutics

Legal name

Lario Therapeutics Limited

Website

https://lariotx.com

Company type

Private

Founded year

2021

Operating status

Operating

Headcount range

1–10 employees

Short description

Lario Therapeutics is a preclinical Edinburgh-based biopharmaceutical company developing first-in-class small-molecule inhibitors of voltage-gated neuronal calcium channels (CaV1.3, CaV2.3, CaV2.1) as disease-modifying precision medicines for severe neurological disorders including epilepsy, DEE69, CDKL5-deficiency, Parkinson's disease, and PTSD.

Ownership category

akta.pro rank

Product category

Neurology Pharmaceuticals

NAICS

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

SIC

Pharmaceutical Preparations (2834), Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Non-prescription / OTC Digital Therapeutics (HLACANAC)

akta.pro secondary industries

Rare Neurology & Neurodegenerative Disorder Therapies (HLAIAIAD), Neurology & Psychiatry (CNS) Pharmaceuticals (HLAIAAAF)

Keywords

• Precision neuroscience therapeutics
• Calcium channel modulators
• Epilepsy drug development
• Parkinson's disease therapeutics
• Neurological disorder treatments

Headquarters

HQ city

Edinburgh

HQ country

United Kingdom

HQ region

Europe

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Non-dilutive Grant Funding: Company is in preclinical development stage with no commercial products or revenue. Funded entirely through grant funding from The Michael J. Fox Foundation ($6M in 2024, $1.5M in 2026) and Wellcome Trust (£700,000/$900K in 2026) for research and development activities.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels3 records

Core offering

Lario Therapeutics is a preclinical biopharmaceutical company developing first-in-class, selective small-molecule inhibitors of voltage-gated neuronal calcium channels (CaV2.3, CaV1.3, and CaV2.1) as disease-modifying precision medicines for severe neurological disorders. Its pipeline targets developmental and epileptic encephalopathies (DEE69, CDKL5-deficiency disorder), drug-refractory epilepsies, Parkinson's disease, genetic hemiplegic migraine, neurodevelopmental disorders, and PTSD. The company has not yet commercialized any product and is funded through research grants and seed investment.

Product overview

Lario Therapeutics is a biopharmaceutical company developing first-in-class precision medicines as disease-modifying treatments for severe neurological disorders. The company's pipeline consists of three distinct calcium channel modulator programs: CaV2.3 Modulators (in clinical development for DEE69, CDKL5-deficiency disorder, drug refractory epilepsies, and Parkinson's disease), CaV2.1 Modulators (for genetic hemiplegic migraine and neurodevelopmental disorders), and CaV1.3 Modulators (for Parkinson's disease, treatment resistant hypertension, and autism-related syndromes). The company was founded in 2021 as a spin-out from Epidarex Exeed and is based in Edinburgh, UK.

Differentiator

Problem solved

Functional benefit

Quantifiable outcome

• IND-enabling studies planned for 2026 for leading CaV2.3 programme in developmental and epileptic encephalopathies
• +3 more outcomes

Segments2 records

Ideal customer profiles2 records

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Four partnerships are on record, tiered core and minor.

• Oxford Parkinson’s Disease CentrecoreStrategic or Co-development Partner · 22 July 2024Collaboration with Professor Richard Wade-Martins and the Oxford Parkinson’s Disease Centre (OPDC) for preclinical research on CaV2.3 calcium channel inhibition as a disease-modifying approach for Parkinson’s disease. The OPDC will contribute key scientific insights and evaluate Lario compounds in state-of-the-art patient-derived stem cell models of Parkinson’s disease.
• CACNA1E InternationalminorStrategic or Co-development Partner · 21 May 2024Parent-led international organization founded six months prior to May 2024, raising awareness about CACNA1E-related Developmental Epileptic Encephalopathy Type 69 (DEE69) and encouraging development of targeted treatments. Lario CEO and CSO presented at inaugural family conference.
• Loulou FoundationcoreStrategic or Co-development Partner · 7 November 2023Established by the Loulou Foundation, the CDKL5 Forum is the largest annual conference on CDKL5 Deficiency Disorder. The foundation presented the 'Company Making a Difference Award' to Lario for its precision medicine approach in genetic epilepsies, recognising the CDKL5-CaV2.3 link as a key discovery.
• Epidarex ExeedcoreStrategic or Co-development Partner · 1 January 2021Lario Therapeutics was founded in 2021 as a spin-out from Epidarex Exeed, the therapeutic discovery engine of Epidarex Capital. Epidarex Exeed provided the foundational technology and scientific expertise for the calcium channel drug discovery platform.

Scale indicators3 records

Recent moves7 records

Expansion highlights5 records

Direct peers

• Xenon Pharmaceuticals: Clinical-stage CNS biotech developing ion channel modulators for epilepsy and other neurological disorders. Most directly comparable peer given shared focus on voltage-gated ion channel pharmacology for CNS indications.
• Praxis Precision Medicines: Clinical-stage company developing precision medicines for CNS disorders including rare epilepsies (DEEs). Overlaps with Lario's CaV2.3 program targeting DEE69 and CDKL5-deficiency disorder.
• Longboard Pharmaceuticals: CNS-focused clinical-stage biotech developing therapeutics for rare epilepsies and neurodevelopmental disorders (acquired by Lundbeck in 2024). Directly comparable in indication focus.
• Marinus Pharmaceuticals: Specialty pharma commercialising ganaxolone for CDKL5-deficiency disorder and refractory epilepsies. Highly relevant peer as Lario's CaV2.3 program also targets CDKL5-deficiency disorder.
• Biohaven Pharmaceuticals: Clinical-stage neuroscience biotech developing therapeutics across migraine, epilepsy, and rare CNS disorders. Comparable as a CNS specialty pharma pipeline company.
• Neumora Therapeutics: Well-funded clinical-stage neuroscience biotech developing precision medicines for brain diseases. Comparable as a recent platform-style CNS precision medicine company backed by venture capital.

Emerging players

• Encoded Therapeutics: Clinical-stage gene therapy company targeting CNS genetic epilepsies and neurodevelopmental disorders. Comparable as a precision-medicine platform for rare neurological diseases, though using a different modality.
• Stoke Therapeutics: Clinical-stage biotech developing antisense oligonucleotides for rare genetic neurodevelopmental disorders including Dravet and ADPKD. Comparable in precision-medicine approach to genetically defined CNS disorders.

Broad incumbents

• Sage Therapeutics: Established CNS biopharma (Zurzuvae/zuranolone) with broader portfolio across neurology and psychiatry. Relevant as a CNS-focused specialty pharma comparator.
• UCB: Global pharma with major epilepsy franchise (Briviact, Vimpat, Keppra) and active investment in rare epilepsy pipelines. Logical long-term acquirer/partner for Lario's CaV2.3 program.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• LinkedInlinkedin.com/company/lario-therapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles8 records

Funding overview

Funding rounds2 records

Investors2 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 Lario Therapeutics