Biotech
5 Early-Stage RNA Therapeutics Startups Developing New Medicines
DealBio tracks early-stage RNA therapeutics startups across Europe and North America. These 5 are developing new medicines by replacing, silencing, reading or reprogramming RNA-linked biology.
- RNA Therapeutics
- Drug Delivery
- Oncology
- Autoimmune Disease
- Rare Disease
RNA therapeutics no longer describe a single drug format. Some programmes replace or silence RNA directly. Others use RNA sequences as molecular triggers, encode instructions in mRNA or target the enzymes that control how RNA behaves.
Across this group, the common investment question is not simply whether RNA is involved. It is whether each startup can connect a specific RNA mechanism to delivery, target selectivity and a development path that produces a medicine with a clear advantage over existing treatment.
At a Glance
- NorthMiRsCanada2022MicroRNA replacement for sepsis-induced organ injury
- CALDRESwitzerland2025GalNAc-siRNA for cholestatic liver disease
- Umlaut.bioGermany2024Small molecules targeting tRNA modification
- Akribion TherapeuticsGermany2024RNA-guided cell depletion in cancer
- Flip TherapeuticsUnited Kingdom2024mRNA reprogramming of disease-driving T cells
Developing microRNA replacement therapy for organ failure caused by sepsis.
- Country
Canada- Founded
- 2022
- Sector
- Biotech
- Funding stage
- Seed or earlier
Why NorthMiRs stands out
NorthMiRs is translating a microRNA signal identified in sepsis biology into a defined synthetic drug candidate. The startup has secured $1.2 million in non-equity and competition funding, has licensed rights to the patent application behind NM-001 and emerged from University of Toronto research led by co-founders Claudia dos Santos and Gilbert Walker, both Tier 1 Canada Research Chairs.
Early translational support also includes a preprint with preclinical lipid-nanoparticle delivery data and selection for the MaRS–adMare Therapeutics Accelerator. CEO Samantha McWhirter adds formulation expertise from a University of Toronto PhD focused on lipid nanoparticle drug-delivery systems.
What NorthMiRs is building
NM-001 is a synthetic replacement for miR-187-3p, a microRNA, a small RNA molecule that regulates gene activity by controlling how messenger RNA is used or degraded, depleted in the hearts of septic patients. NorthMiRs packages the replacement in a lipid nanoparticle, a small lipid-based particle used to carry RNA or other molecules into cells, with the aim of restoring the regulatory signal and reducing sepsis-related organ injury.
The programme is preclinical, the stage before human trials when a treatment is studied in laboratories and often in animal models, and is designed as a single intravenous dose within six hours of suspected sepsis. Rather than using variable cell therapy, the startup has rebuilt an effect identified through long-running stem-cell research as a defined synthetic RNA medicine.
02
CALDRE
Developing liver-directed RNA medicines for cholestatic and fibrotic disease.
- Country
Switzerland- Founded
- 2025
- Sector
- Biotech
- Funding stage
- Seed or earlier
Why CALDRE stands out
CALDRE is advancing a liver-directed siRNA programme with disease-model evidence already behind the lead candidate. ClagalsiR has demonstrated claudin-3 target engagement and therapeutic activity in an established cholestasis model, giving the startup a concrete preclinical readout rather than only a target hypothesis.
The startup has secured CHF 150,000 from Gebert Rüf Stiftung through InnoBooster and CHF 150,000 through Venture Kick. Co-founder Felix Baier completed a University of Bern PhD on liver tight-junction regulation, while Ulysses von Salis brings major life-science transaction experience.
What CALDRE is building
ClagalsiR, CALDRE’s preclinical claudin-3-targeting GalNAc-siRNA candidate, is designed to reduce claudin-3 in hepatocytes. GalNAc, a sugar-based targeting group used to direct certain medicines to liver cells, carries the siRNA toward hepatocytes, the main functional cells of the liver. siRNA, small interfering RNA, reduces production of a target protein by degrading its messenger RNA.
Claudin-3, a protein involved in tight junctions that regulate the barrier between adjacent cells, is being modulated to increase fluid movement into bile. In cholestasis, reduced or blocked bile flow from the liver, CALDRE aims to reduce the concentration of damaging bile components and protect liver tissue. The startup is advancing the preclinical candidate toward studies needed to support an investigational clinical application.
Developing drugs that block disease-linked chemical changes to RNA.
- Country
Germany- Founded
- 2024
- Sector
- Biotech
- Funding stage
- Seed or earlier
- Founders
Why Umlaut.bio stands out
Umlaut.bio approaches RNA therapeutics from the target side rather than by delivering an RNA payload. Its founding biology is supported by a 2025 Cell paper led by co-founder Bastian Linder, showing how a tRNA modification can stabilize cancer-related mRNA and sustain disease-linked signalling.
The startup has raised approximately €3 million from biotech investors including BaseLaunch, High-Tech Gründerfonds, LBBW Venture Capital and InnoGrowth BW. CEO Karsten Fischer adds senior biotech operating and financing experience, while the founding research was conducted in Lars Steinmetz’s group at the European Molecular Biology Laboratory.
What Umlaut.bio is building
Umlaut.bio is developing small-molecule inhibitors of enzymes that add disease-associated chemical marks to transfer RNA. Transfer RNA, or tRNA, is a small RNA molecule that helps translate messenger RNA into protein by carrying amino acids to the protein-building machinery. Blocking those enzymes is intended to change RNA regulation upstream of protein production, creating a drug strategy based on RNA biology without delivering RNA itself.
The discovery platform combines high-throughput compound screening with mass spectrometry, a laboratory method used to identify and measure molecules based on their mass and electrical charge, to find active compounds and confirm that the intended RNA modification has been reduced. The startup is developing programmes across cancer and inflammatory disease.
Developing RNA-guided therapies to selectively destroy cancer cells.
- Country
- Germany
- Founded
- 2024
- Sector
- Biotech
- Funding stage
- Seed or earlier
Why Akribion Therapeutics stands out
Akribion Therapeutics pairs a programmable intracellular RNA trigger with a destructive nuclease. A May 2026 study showed reduced HPV-positive head and neck tumor growth in a patient-derived mouse model after local nanoparticle delivery, providing preclinical evidence for the core cell-depletion concept.
The startup raised an €8 million seed round co-led by CARMA FUND and RV Invest and holds an exclusive pharmaceutical licence from BRAIN Biotech for G-dase E. Founder Paul Scholz is the principal inventor of the platform, while Michael Krohn and Lukas Linnig bring R&D and biotech operating experience.
What Akribion Therapeutics is building
G-dase E, Akribion’s RNA-guided nuclease platform designed to destroy selected cells, uses a guide RNA, an RNA molecule that directs a nuclease to a matching genetic sequence, to recognize a selected RNA sequence inside a cell. A nuclease is an enzyme that cuts DNA or RNA. When the matching sequence is present, the nuclease activates and degrades DNA and RNA inside that cell, triggering cell destruction rather than making a precise genetic edit.
The initial programme targets HPV, a family of viruses whose high-risk types can cause cervical, throat and other cancers, in throat cancer. Viral RNA provides a molecular signature that can distinguish targeted cells. In the mouse work described in the live DealBio Brief, lipid nanoparticles carried nuclease mRNA, the molecule that carries instructions from DNA to the machinery that builds proteins, and an HPV16-targeting guide for local delivery.
Developing RNA medicines to reprogram immune cells for autoimmune disease.
- Country
United Kingdom- Founded
- 2024
- Sector
- Biotech
- Funding stage
- Seed or earlier
Why Flip Therapeutics stands out
Flip Therapeutics is building an RNA-delivery approach around immune-cell reprogramming rather than immune suppression alone. Medicines Discovery Catapult committed £500,000 plus scientific support for payload validation and nanoparticle delivery, while the startup emerged from a Deep Science Ventures–AbbVie initiative.
CEO David Pajerowski led Phase I and Phase II technical development of Moderna’s personalised cancer-vaccine programme. Co-founder Mathias Pawlak brings immune-cell biology and biotech R&D experience, and Anis Gammage has contributed to multiple biotech spinouts at Deep Science Ventures.
What Flip Therapeutics is building
Flip combines proprietary mRNA payloads with lipid nanoparticle delivery to reprogram T cells directly inside the body. T cells are immune cells that help coordinate immune responses and can recognize and destroy infected or abnormal cells. The payloads are intended to change disease-driving T cells into anti-inflammatory cells within affected tissue.
The platform is being developed for chronic autoimmune and inflammatory disease, with payload validation and delivery integration as current priorities. Its therapeutic thesis depends on using RNA instructions to shift local immune behaviour rather than repeatedly suppressing inflammation system-wide.
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Investment perspective
RNA therapeutics are widening from a modality category into a set of different control strategies. In this group, RNA can be the replacement molecule, the silencing mechanism, the trigger that activates a nuclease, the instruction carried by an mRNA payload or the biological substrate targeted by a small molecule.
For investors, that makes delivery and mechanism fit especially important. The strongest programmes are not simply RNA-based; they connect a specific RNA role to a tractable tissue, a measurable biological effect and a development path that can generate progressively stronger evidence. Liver targeting, local tumor delivery and lipid nanoparticles already provide concrete routes for several of these startups, while others are testing whether new layers of RNA regulation can become druggable.
Methodology
DealBio selected these five early-stage startups from its coverage of health and longevity startups, focusing on therapeutics in which RNA is central to the medicine’s payload, targeting logic, gene-silencing mechanism or biological target. The selection spans microRNA replacement, liver-directed siRNA, small-molecule control of RNA modification, RNA-guided cell depletion and mRNA-based immune-cell reprogramming.
DealBio builds its Startup Briefs using publicly available startup materials, ecosystem sources, market references and professional profiles.