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.
RNAi brain shuttle platform for neurodegenerative disease drug delivery
Aerska is a biotechnology company developing RNA interference medicines to treat, delay and prevent diseases of the brain. The company's antibody oligonucleotide conjugate platform acts as a brain shuttle, enabling RNAi therapeutics to cross the blood brain barrier and reach the central nervous system through systemic…
Clinical-stage precision oncology developing first-in-class TACC3 inhibitor therapeutics
Coiled Therapeutics is a UK-listed, clinical-stage oncology company developing precision cancer therapeutics against structurally complex, previously undruggable targets. Its lead candidate, AO-252, is a first-in-class, orally administered small molecule inhibitor of TACC3 currently in Phase I trials for advanced…
AI-powered RNAi drug discovery company developing hepatic medicines
Tangram Therapeutics is a biotechnology company combining artificial intelligence and RNA interference to discover and develop novel medicines. Its proprietary chemistry platform silences disease-driving genes in hepatocytes, and its AI platform drives target identification and siRNA design. The pipeline includes…
Oncolytic virus immunotherapies engineered to destroy solid tumours
Bioarchitech is a preclinical-stage biotechnology company developing oncolytic virus-based immunotherapies for the treatment of common solid tumours. The company engineers vaccinia virus to encode antibody-like immunotherapy molecules within the viral genome, enabling localised delivery of immune-activating payloads…
Liver-directed RNA medicines to restore function in chronic liver disease
Apterna is a preclinical biotechnology company developing RNA-based medicines to restore function in damaged livers. Its lead programme is a liver-directed therapy, given by injection under the skin every few months, that silences a single gene which limits the liver's ability to recover from injury. Animal studies…
siRNA gene therapies targeting root causes of ALS and frontotemporal dementia
Crucible Therapeutics Limited is a preclinical-stage biotechnology company developing siRNA-based gene therapies for Amyotrophic Lateral Sclerosis and Frontotemporal Dementia caused by the C9orf72 gene mutation. Its therapeutic approach depletes the SRSF1 protein to suppress production of toxic dipeptide repeat…
Engineered iPSC-derived exosomes for tissue-targeted drug delivery
Excellio Labs is a biotech developing engineered exosome platforms for targeted drug delivery. The technology exploits the inherent tissue-specificity of iPSC-derived extracellular vesicles, which are further surface-modified to create precision drug carriers capable of delivering nucleic acids, proteins, and small…
AI-driven delivery platform for siRNA therapies beyond the liver
Undecimal Bio is a biotechnology company developing targeted delivery technologies for small interfering RNA (siRNA) therapeutics beyond the liver. Its DELiveri platform combines large-scale combinatorial chemistry, functional cell-based screening and AI to identify synthetic conjugates that transport siRNA into…
Antisense oligonucleotide therapies for TUBB4A-associated leukodystrophies
SynaptixBio is a biotechnology company developing antisense oligonucleotide therapies for severe inherited leukodystrophies. Its lead programme targets TUBB4A-associated hypomyelinating disorders, including hypomyelination with atrophy of the basal ganglia and cerebellum (H-ABC). The approach uses synthetic…
In vivo immune cell reprogramming for autoimmune and inflammatory disease
Flip Therapeutics is a biotechnology company developing in vivo immune cell engineering therapies for chronic autoimmune and inflammatory diseases, including multiple sclerosis, type 1 diabetes and inflammatory bowel disease. The company reprograms pathogenic T cells into homeostatic, anti-inflammatory cells directly…
DNA nanorobot platform for nucleic acid detection and bioanalysis
Nanovery is a biotechnology company developing a patented Nucleic Acid Nanorobotics (NANs) platform for enzyme-free detection and quantification of nucleic acid targets. Synthetic DNA nanorobots generate fluorescent signals in response to specific sequences, enabling direct quantification of antisense…
Needle-free solid dose vaccine formulation and delivery platform
aVaxMed Limited is a UK biotech developing the aVaxziPen platform, a proprietary needle-free, thermally stable, solid dose vaccine delivery system. The technology encapsulates vaccine antigens into unit solid doses administered via a pen applicator, removing the need for needles and reducing cold chain requirements.…
siRNA therapeutics targeting liver-expressed disease genes
Silence Therapeutics is a clinical-stage biotechnology company developing siRNA therapeutics using its proprietary platform. The platform enables precise silencing of disease-associated genes expressed in the liver, without permanently altering DNA. Its pipeline addresses cardiovascular disease, haematological…
Circular RNA therapeutics and scalable RNA production
RNAvate is a Cambridge biotechnology spinout developing circular RNA (circRNA) technologies for RNA medicines, founded from research at the MRC Laboratory of Molecular Biology. Its platform focuses on scalable circRNA synthesis and purification to support durable therapeutic protein expression. Published laboratory…
RBC derived nanoparticles for RNA delivery to brain and immune cells
Crane Biosciences is a biotechnology company developing a non-immunogenic RNA delivery platform based on nanoparticles derived from red blood cell membranes. By using the body's own cellular architecture as a delivery vehicle, the platform achieves substantially higher in vivo delivery efficiency to target tissues…
Stem cell-derived exosome platform for targeted drug delivery
ReNeuron is a biotechnology company developing a stem cell-derived exosome platform for targeted therapeutic delivery. Founded on over two decades of stem cell science, including the world's first clinical trial of conditionally immortalised neural stem cell therapy for stroke disability, the company has pivoted to…
mRNA vaccine manufacturer and R&D partner
Moderna is a company developing and manufacturing mRNA-based medicines and vaccines across infectious disease, oncology and rare disease. Its UK Innovation and Technology Centre, opened at Harwell in September 2025, is the first facility in the UK capable of onshore mRNA vaccine manufacturing, with capacity for up to…
Programmable RNA for scalable cell engineering and biomanufacturing
Plurify is a biotechnology company developing PluRNA, a programmable RNA platform for sustained protein expression without genome integration. Its technology is designed to engineer cells through controllable RNA-based programmes, with applications in stem-cell therapy manufacturing, cell purification, biologics…
LNP-mRNA growth factor therapy to restore hearing loss
CamGene Therapeutics is a preclinical biotechnology company developing lipid nanoparticle-delivered mRNA medicines to treat hearing loss at its underlying cellular cause. Its lead approach uses an LNP-mRNA growth factor therapeutic designed to regenerate cochlear nerve cells and reduce inflammation, aiming to restore…
European hub for RNAi therapeutics developer
Alnylam UK is the European operational headquarters of Alnylam Pharmaceuticals, the pioneer of RNA interference therapeutics. Based in Maidenhead, the UK office serves as Alnylam's primary base for European commercial operations, medical affairs, and regulatory activities. Alnylam has six approved RNAi medicines on…