What Neumirna Therapeutics does

Neumirna Therapeutics ApS is a Copenhagen-based, clinical-stage biotechnology company developing miRNA-targeting antisense oligonucleotide (ASO-LNA) therapies for neurological diseases. The company's proprietary platform uses chemically modified ASOs with locked nucleic acid (LNA) chemistry to inhibit overactive microRNAs, aiming to rebalance disease-relevant brain networks rather than merely suppress symptoms. Its lead candidate NMT.001 targets miR-134 for drug-resistant epilepsy and is positioned for Phase 1/2a clinical trials (MiRNova study) starting in early 2027. The pipeline includes NMT.002 (miR-27b targeting ASO for Parkinson's disease, preclinical), plus two undisclosed candidates (NMT.003 and NMT.004) in screening, supported by a 24-36 month target-identification-to-clinic development cadence.

The company was founded in 2020 by Dr. Henrik Klitgaard (discoverer of the SV2A mechanism underlying UCB's Keppra and Brivaracetam), Prof. Sakari Kauppinen (discoverer of Miravirsen, the world's first miRNA-targeted drug at Santaris Pharma), and serial biotech entrepreneur Lars Hellerung Christiansen. Neumirna operates from Copenhagen, Basel, and Boston with 1-10 employees, and is led by CEO Ellen K. Donnelly (ex-Abliva AB, Pfizer neuroscience), CDO Ina Brünig-Traebert (ex-Roche RNA therapy program for Angelman Syndrome), and CMO Dr. Ferenc Tracik (ex-NeuroSense, Biogen, Novartis). The Scientific Advisory Board includes Prof. Jacqueline French (NYU Langone) and Prof. Karl Kieburtz (University of Rochester).

Neumirna is pre-revenue and monetizes through future product commercialization or licensing rather than current sales. Capital is sourced from a €20M Series A (January 2025, co-led by Angelini Ventures and Invivo Partners) plus non-dilutive grants from The Michael J. Fox Foundation ($650K), CURE Epilepsy ($250K), the Epilepsy Foundation ($200K), and Innovation Fund Denmark (5M DKK Innobooster). Customers are future-facing: target patient populations include the ~50 million people worldwide with epilepsy, of whom approximately one-third are treatment-resistant, as well as Parkinson's disease patients. Go-to-market relies on scientific conferences (AES, AD/PD), investor/partnering events (BIO Europe, BioEquity, Jefferies, NLS Days), patient advocacy engagement, and academic collaborations with institutions including Aalborg University, RCSI, DTU, University of Copenhagen, and Vall d'Hebron University Hospital.

Name

Neumirna Therapeutics

Legal name

Neumirna Therapeutics ApS

Website

https://neumirna.com

Company type

Private

Founded year

2020

Operating status

Operating

Headcount range

1–10 employees

Short description

Neumirna Therapeutics is a Copenhagen-based clinical-stage biotechnology company developing miRNA-targeting antisense oligonucleotide (ASO-LNA) therapies for neurological diseases, with lead candidate NMT.001 advancing toward Phase 1/2a trials in drug-resistant epilepsy in 2027.

Ownership category

akta.pro rank

Product category

Neurology RNA Therapeutics

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Pharmaceutical Preparations (2834), Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

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

akta.pro secondary industries

RNA Therapeutics Clinical Development (trial design, endpoints, patient stratification) (HLAAADAH), RNA Therapeutics Platforms & Design (sequence design, target ID/validation, AI/bioinformatics) (HLAAADAG), RNA Chemistry & Oligonucleotide Engineering (backbones, modifications, stereochemistry) (HLAAADAE)

Keywords

• RNA therapeutics
• Antisense oligonucleotides
• Neurological disease treatments
• MicroRNA targeting
• Epilepsy drug development

Headquarters

HQ city

Copenhagen

HQ country

Denmark

HQ region

Europe

Offices3 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. RNA Therapeutics Development: Pre-revenue biotech company developing RNA-based therapeutics for neurological diseases. Revenue generation will come from future product sales following regulatory approval.

Go-to-market motion2 records

Distribution channels1 record

Marketing channels6 records

Core offering

Neumirna Therapeutics develops miRNA-targeting antisense oligonucleotide (ASO-LNA) therapies for neurological diseases, with a lead candidate NMT.001 in clinical development for drug-resistant epilepsy. The company operates a proprietary drug discovery platform spanning target identification, ASO design, in vitro screening, lead optimization, and IND-enabling studies, designed to advance candidates from idea to clinic within 24-36 months. Its pipeline includes NMT.001 (miR-134, epilepsy, Phase 1/2a), NMT.002 (miR-27b, Parkinson's, preclinical), and two undisclosed preclinical programs.

Product overview

Neumirna Therapeutics is a biotech company developing a portfolio of miRNA-targeting antisense oligonucleotide (ASO-LNA) therapies for neurological diseases. The company's core offering consists of a proprietary ASO-LNA platform and multiple pipeline candidates: NMT.001 (lead candidate targeting miR-134 for drug-resistant epilepsy, in Phase 1/2a), NMT.002 (targeting miR-27b for Parkinson's disease, preclinical), and two undisclosed programs (NMT.003 and NMT.004). The platform enables targeted intrathecal delivery of RNA therapeutics designed to slow or reverse disease progression by modulating microRNAs—master regulators of gene expression in neurological disorders.

Differentiator

Problem solved

Functional benefit

Products and services

• NMT.001 First-in-class antisense oligonucleotide (ASO-LNA) therapy targeting microRNA-134 (miR-134) for the treatment of drug-resistant epilepsy. Designed for intrathecal delivery to the central nervous system with potential for twice-yearly administration and disease-modifying effects, intended for patients whose seizures are not controlled by existing anti-seizure medications.
• NMT.002 ASO-LNA compound targeting microRNA-27b (miR-27b) for the treatment of Parkinson's disease. Currently in preclinical development, with the program aiming to deliver a disease-modifying RNA therapy for progressive movement disorders in collaboration with Motac Neuroscience and the Center for RNA Medicine at Aalborg University.
• NMT.003 ASO-LNA compound with undisclosed target for an undisclosed neurological indication. Currently in the screening stage of development as part of Neumirna's multi-asset pipeline.
• NMT.004 ASO-LNA compound with undisclosed target for an undisclosed neurological indication. Currently in the screening stage of development as part of Neumirna's multi-asset pipeline.
• ASO-LNA Development Platform Proprietary drug discovery and development platform that combines antisense oligonucleotide (ASO) chemistry with locked nucleic acid (LNA) modifications for targeted intrathecal delivery of RNA-based therapeutics. Spans target identification, ASO design, in vitro screening, lead optimization, in vivo proof of concept, and CMC/toxicology, with the goal of moving from idea to clinical testing within 24-36 months.

Quantifiable outcome

• NMT.001 demonstrated adequate safety margins across relevant preclinical studies, supporting continued progress toward the clinic
• +2 more outcomes

Segments2 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Eight partnerships are on record, tiered core and supporting.

• Motac NeurosciencecoreStrategic or Co-development Partner · 1 August 2023Collaborating on MJFF-funded project for disease-modifying RNA therapies for Parkinson's disease, bringing expertise in preclinical models for neurodegenerative diseases.
• Center for RNA Medicine at Aalborg UniversitycoreStrategic or Co-development Partner · 1 August 2023Academic partner in Parkinson's disease research collaboration, contributing research excellence in RNA medicine.
• Aalborg UniversitycoreStrategic or Co-development PartnerAcademic collaborator on drug discovery pipeline for Parkinson's disease RNA therapies. Home of Center for RNA Medicine.
• Royal College of Surgeons Ireland (RCSI)supportingStrategic or Co-development PartnerAcademic collaborator listed on company website.
• DTU (Technical University of Denmark)supportingStrategic or Co-development PartnerAcademic collaborator listed on company website.
• University of CopenhagensupportingStrategic or Co-development PartnerAcademic collaborator listed on company website.
• Vall d'Hebron University HospitalsupportingStrategic or Co-development PartnerClinical collaborator with Dr. Manuel Toledo from Scientific Advisory Board.
• Epilepsy Study ConsortiumsupportingStrategic or Co-development PartnerCollaborator founded by Scientific Advisory Board member Dr. Jacqueline French, helps define how early-stage epilepsy trials are conducted.

Scale indicators7 records

Recent moves7 records

Expansion highlights7 records

Broad incumbents

• Biogen: Large biotech with deep CNS franchise, including Spinraza (ASO for SMA). Comparable in CNS therapeutic focus and serves as a likely M&A or commercialization partner for late-stage CNS ASOs.
• Alnylam Pharmaceuticals: Commercial-stage leader in RNA interference therapeutics (siRNA), including Onpattro and Givlaari. Adjacent RNA-based therapeutic modality targeting CNS/neurological indications.
• Roche / Genentech: Pharma incumbent with significant ASO/CNS pipeline including tofersen (with Ionis) for SOD1 ALS and former development of Rugonersen (now Roche-led). Key competitor and potential partner for Neumirna's ASO-LNA programs.
• Ionis Pharmaceuticals: Established leader in antisense oligonucleotide drug development with a broad CNS pipeline including Spinraza (marketed) and tofersen. Sets the competitive baseline for ASO technology, manufacturing, and CNS clinical development.

Direct peers

• Wave Life Sciences: Clinical-stage company developing stereopure antisense oligonucleotides for neurological diseases (e.g., Huntington's, ALS). Directly comparable platform technology and CNS disease focus.
• Stoke Therapeutics: Clinical-stage biotech developing antisense oligonucleotide therapies for neurological diseases (e.g., STK-001 for Dravet syndrome). Closest direct peer to Neumirna, also pursuing disease-modifying CNS ASOs with intrathecal delivery.

Emerging players

• Skyhawk Therapeutics: Emerging player developing small-molecule RNA splicing modulators for neurological diseases. Comparable in CNS therapeutic focus and RNA-targeting approach but uses different chemistry.
• Atalanta Therapeutics: Emerging biotech developing RNAi therapeutics for neurological diseases including Huntington's. Comparable in CNS RNA-targeted approach and disease-modifying positioning.
• Denali Therapeutics: Clinical-stage biotech focused on neurodegeneration with multiple modalities. Comparable in CNS therapeutic area focus and disease-modifying positioning, though broader modality mix.
• ProQR Therapeutics: Clinical-stage RNA editing / oligonucleotide company with CNS programs. Comparable in modality (RNA-based) and therapeutic area (neurological/genetic diseases).

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/neumirna-therapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles17 records

Funding overview

Funding rounds1 record

Investors3 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 Neumirna Therapeutics