List of RNA Therapies Companies in United Kingdom - 10

Cambridge Nucleomics is a biotechnology company that specializes in fast and easy RNA measurement at scale with single-molecule accuracy.

Maintaining musculoskeletal health is the keystone of overall health as we get older. As we age our physical condition gradually deteriorates, leading to loss of strength, fitness and mobility. In most cases, this isn’t due to a simple decision to stop exercising. It is due to the breakdown of musculoskeletal function caused by disease. Musculoskeletal diseases such as osteoarthritis, tendinopathy and lower back pain are the primary causes of disability and early retirement in people over the age of 65. Currently, there are no approved disease-modifying drugs for these diseases, meaning that physical decline in old age is inevitable. At Causeway, we believe that physical decline in old age is not inevitable and in indeed may be reversible. To address this, we are developing a range of therapies that treat common MSK diseases that will allow us to remain fit and active into our nineties and beyond. Staying active as we get older also reduces the risk of developing osteoporosis, dementia, diabetes, obesity, cancer and cardiovascular disease.

Eleven Therapeutics is a biotechnology company leading the AI revolution in nucleic acid therapeutics. They harness the power of combinatorial chemistry and AI to transform RNAi drug development into a programmable process.

Designer Sponge RNAs for Cellular Health Restoration

At Harness Therapeutics we have one mission – physiological upregulation of targets that have the promise to provide disease-modifying therapeutics for neurodegenerative diseases. We use a range of mRNA-targeted oligonucleotide-based methodologies to alter the post-transcriptional regulation of protein synthesis and so increase the target protein in a controlled manner. Our focus on neurodegenerative disease is driven by our passion to develop truly disease-modifying therapies for diseases that affect so many people, yet for which there are few if any therapies that can slow or halt the progression of the disease. The ability to effect controlled increases of protein levels is essential in being able to safely drug targets with narrow therapeutic windows, something that existing platforms cannot achieve. This is critical for neurodegenerative disease as there are now a number of targets that have strong genetic validation linked to disease progression but require controlled increases rather than knock-down or over-expression. Our goal is to open up the target space for neurodegenerative disease and be able to advance effective treatment solutions for the tens of thousands of patients and their families who are facing the certainty of irreversible disease progression, loss of quality of life, and ultimately death. Our name reflects our approach, built and refined since our formation in 2020; harnessing the body’s endogenous cellular processes controlling protein synthesis to achieve controlled increases in protein levels in the safest possible manner. We use our deep expertise in RNA biology/bioinformatics and neuro cell biology to probe the intricate regulation of each target and identify multiple upregulation opportunities. Our multi-year experience focussed on physiological upregulation has allowed us to develop bespoke assays and analytical techniques to screen for constructs that drive small increases in protein, which is a key challenge in this space. Our sole focus on neurodegeneration has allowed us to build deep expertise in the biology of neurons and our targets, and the best possible models for disease – critical in being able to de-risk and translate candidates to the clinic. Neurodegenerative disorders are chronic conditions that destroy parts of the nervous system over time, especially the brain. They result in progressive loss of cognitive and motor function and eventually, death. The most common neurodegenerative diseases include Alzheimer’s disease, Parkinson’s disease, Amyotrophic lateral sclerosis (ALS), Motor neuron disease (MND), Huntington’s disease (HD), Spinal muscular atrophy (SMA), and Spinocerebellar ataxia (SCA). It is estimated that more than 50 million people worldwide are affected by neurodegenerative diseases and this number is increasing as overall life expectancy increases. Most of these conditions are much more likely to happen in people over 65, but some conditions like Huntington’s disease and ALS often appear much earlier.

Ochre Bio is a biotechnology company developing RNA therapies for chronic liver diseases. Using a combination of genomic deep phenotyping, precision RNA medicine, and testing in live human donor livers, Ochre is developing therapies for important liver health challenges, from increasing donor liver supply to reducing cirrhosis complications. To learn more, please visit www.ochre-bio.com.

Poolbeg Pharma plc is committed to the development and commercialisation of innovative medicines targeting diseases with a high unmet medical need, with a growing emphasis on rare and orphan diseases. Its model focusses upon developing its exciting clinical assets and commercialising approved and marketed drugs to fund the development of its robust pipeline of innovative products, thereby driving significant value creation. Poolbeg is led by an experienced leadership team with a history of delivering significant shareholder value. The team has been strengthened by the appointment of three former members of the Amryt Pharma plc leadership team, with the intention of repeating Amryt's success and generating near term revenues. Poolbeg's clinical programmes target large addressable markets including cancer immunotherapy-induced CRS, infectious disease, and metabolic conditions such as obesity with the development of an oral GLP-1R agonist. It uses a cost-effective development philosophy to generate high quality human data to support partnering and further development. Its AI-led infectious disease programmes analyse unique data from human challenge trials to identify clinically relevant drug targets and treatments, leading to faster development and greater commercial appeal.

Sireau Labs is a new biotech based in Cambridge, UK, that is using mRNA technology to develop ground-breaking treatments for rare genetic diseases. Sireau Labs is engineering solutions for rare diseases with science that is at the forefront of precision medicine. We are in a daily race to save lives stolen or impacted by rare disease.

We are an RNA delivery and therapeutics company. Our proprietary Bio-Courier® technology addresses the limitations of other RNA delivery technologies through the hybridization of organic materials with inorganic bioabsorbable silicon. Our Bio-Courier portfolio includes silicon stabilized hybrid lipid nanoparticles (sshLNP) that improve the stability, safety, and transfection efficiency of RNA. We use sshLNP to develop a pipeline of RNA therapeutics for rare genetic disorders and are maximizing the potential of our technology through research partnerships. Led by founder and leading biomaterials specialist Dr Suzanne Saffie-Siebert, SiSaf is a venture capital backed private company. Headquartered in Guildford, UK, we have fully integrated research labs and bio-analytical facilities, a scalable technology to fast-track development, and a growing patent estate with extensive freedom to operate.

VaxEquity is a biotechnology company amplifying the impact of RNA vaccines and therapeutics, through using its next generation self-amplifying RNA (saRNA) platform. The Company uses saRNA to provide greatly enhanced protein expression, combining better tolerability with slower release and thermostability. VaxEquity is pioneering a next generation approach by modifying its saRNA to modulate the immune response and thereby overcoming current limitations. The flexibility of the Company’s platform ensures that its broad pipeline of assets can be designed, developed, and manufactured rapidly and at scale. VaxEquity was founded in 2020 by Professor Robin Shattock, the Head of Immunology of Infection at Imperial College London and is led by a world class team who are pioneers in the field of RNA.