RNA therapeutics companies develop medicines that use RNA molecules to alter protein production, silence disease-associated genes or deliver therapeutic instructions to cells. These approaches support the treatment of genetic disorders, cancer, infectious disease and other conditions.
This category includes companies working on:
Browse 62 UK RNA therapeutics companies below.
RNA immunotherapies targeting novel tumour-specific antigens
Epitopea is a biopharma company developing off-the-shelf RNA-based immunotherapies for hard-to-treat cancers. Using spatial immunopeptidomics, genomics, and systems bioinformatics, the company identifies aberrantly-expressed tumour-specific antigens that are broadly shared across patients with the same tumour type.…
Non-viral intracellular drug delivery for solid tumours
Vitarka Therapeutics is a preclinical biotech company developing a non-viral drug delivery platform for targeted intracellular delivery of therapeutics to solid tumours. The technology uses pore-forming proteins to transport RNAi therapies and other payloads directly into cancer cells while limiting off-target…
AI drug discovery platform developing novel biologics for cancer and AMR
AMPLY Discovery is a biotechnology company developing an AI-driven drug discovery platform that combines machine learning, next-generation sequencing, and synthetic biology to identify novel biologic drug candidates from natural biological diversity. The platform digitises biological molecules, identifies treatment…
Small activating RNA therapeutics for gene upregulation and disease
MiNA Therapeutics is a clinical-stage biotechnology company pioneering small activating RNA medicines, a class of oligonucleotide therapeutics that restore or boost expression of underactive genes by activating transcription rather than silencing it. The approach targets diseases driven by loss of normal gene…
RNA-targeted drug discovery platform using machine learning
RevoNA Bio is a biotechnology company developing a high-throughput RNA biological platform for RNA-targeted drug discovery. The platform generates comprehensive RNA structural and functional datasets that feed a machine learning predictive engine, supporting hit identification, structure-activity relationship…
AI-designed extended-expression RNA therapeutics for cardio-metabolic disease
ScionxBio, formerly Eleven Therapeutics, is a biotechnology company developing xRNA, a chemically expanded RNA modality engineered to extend protein expression from days to months. Combinatorial chemistry and deep learning are combined to design molecules with enhanced stability and reduced immune sensing, enabling…
RNA-based therapeutics targeting neurodegenerative disease mechanisms
Harness Therapeutics is a Cambridge-based biotech developing oligonucleotide therapeutics that exploit post-transcriptional regulation to achieve controlled upregulation of disease-relevant proteins in the CNS. Its lead programme targets FAN1 nuclease in Huntington's disease to limit somatic DNA repeat expansion, with…
Global pharmaceutical company developing medicines across major disease areas
AstraZeneca is a multinational biopharmaceutical company engaged in the research, development, manufacture, and commercialisation of prescription medicines. Its pipeline and product portfolio span oncology, cardiovascular and metabolic disease, respiratory and immunology, rare diseases, and vaccines. The company…
RNA trans-splicing gene therapies for aggressive cancers
Spliceor is a University of Cambridge spinout developing RNA trans-splicing gene therapies for aggressive cancers. Its platform is designed to produce therapeutic messenger RNA selectively in targeted cells by joining segments from different RNA molecules. Machine learning helps refine the constructs and optimise…
RNA-based gene control systems for safer cell and gene therapies
Concinnity Genetics is a biotechnology company developing RNA-based control systems that enable precise regulation of gene therapies after administration. Its technology allows therapeutic gene expression to be modulated or reduced in response to side effects or clinical need, addressing a fundamental safety…
Cell-based platform for next-generation mRNA medicines
Sensible Biotechnologies is a biotechnology company developing a cell-based platform for the design, optimisation, and manufacturing of high-quality mRNA. Using engineered living cells and precision fermentation, the platform produces low-immunogenicity mRNA at scale, addressing fundamental limitations of conventional…
Longer-lasting RNA medicines and immune-stealthed CAR-T research
Cambridge RNA Technologies develops experimental RNA platforms intended to extend the duration of therapeutic gene expression. Its research includes technologies for longer-lasting RNA medicines. In collaboration with Anglia Ruskin University, the company has investigated immune-stealth approaches for donor-derived…
AI-guided RNA therapies for chronic metabolic diseases
Elarna Bio is a biotechnology company developing long-acting RNA therapeutics for chronic metabolic diseases. It combines AI-driven discovery with RNA engineering to improve the duration of therapeutic activity. The platform focuses on challenges associated with liver-targeted RNA treatments and is intended for…
RNA therapeutics platform targeting multiple disease pathways
SymphoRNA is a company developing RNA medicines designed to address complex diseases by targeting multiple biological pathways simultaneously. Its platform uses a polysaccharide-based delivery system to enable combination RNA therapies, with the aim of achieving outcomes that single-target approaches cannot. Spun out…
Programmable RNA delivery platform targeting non-hepatic tissues
Sixfold Bioscience is a biotechnology company developing a programmable RNA-based delivery platform that exploits the body's natural extracellular RNA transport pathways to direct therapeutic RNA molecules to specific non-hepatic tissues and cell types. The platform uses computationally designed chemical modifications…
Novel mRNA technology platform for safer, faster vaccines
CamMed Therapeutics (Cambridge Medixine Ltd) is a preclinical biotechnology company developing a novel mRNA technology platform for vaccines and therapeutics. Inspired by natural biological phenomena, its engineered RNA molecules are designed to deliver safer, easier and faster medicines to the clinic while reducing…
Supramolecular encapsulation chemistry for controlled active delivery
Aqdot is a chemical technology company developing supramolecular systems for capturing, protecting and releasing active ingredients. Its encapsulation platform is commercially applied to odour removal and industrial formulations, while its research extends to RNA delivery systems for vaccines and cancer therapies. The…
Silicon-based RNA delivery platform for rare disease therapeutics
SiSaf is a biopharmaceutical company developing RNA therapeutics using its proprietary platform, which combines bioabsorbable silicon with organic lipid materials to improve the stability, safety, and transfection efficiency of nucleic acid payloads. The platform addresses known limitations of conventional lipid…
Targeted nanoparticle formulations for nucleic acid delivery
XapHe is a biotechnology company developing peptide-based nanoparticles for therapeutic nucleic acid delivery. Its HAWC platform packages RNA and DNA payloads into formulations designed for cell entry and tissue targeting. The company supplies transfection kits and offers custom nanoparticle formulation services for…
Stabilised siRNA therapeutics platform targeting cardiovascular gene silencing
Argonaute RNA is a biopharmaceutical company developing small interfering RNA therapeutics for cardiovascular disease. Its siRNA platform uses proprietary chemical stabilisation to enable safe, targeted and temporary gene silencing in specific tissue cells, overcoming delivery and safety barriers that have constrained…