List of Adeno-associated viral vector (AAV) Companies in United Kingdom - 26

4basebio is enabling next generation cell and gene therapies and vaccines with its technologies and solutions. We are able to design, manufacture and supply application-specific synthetic DNA or mRNA as well as targeted non-viral vectors for the delivery of nucleic acid payloads. Our novel synthetic DNA technology offers unique customisation potential, rapid turn-around times, and improved safety profiles, addressing the current limitations of plasmids and other DNA technologies. We currently offer four DNA construct types, each ideally suited for use in viral and non-viral vector applications, genome editing, vaccines and therapeutics, and DNA vaccine applications, respectively. Our proprietary non-viral delivery system, Hermes™, is a nanoparticle vector that can be customised to target cells or tissues of interest for a range of applications. This combines the safety and efficiency of lipid-based nanoparticles (LNPs) with the specificity of a targeting system, addressing the shortcoming associated with both viral vectors and non-targeted LNPs.

Akamis is a clinical-stage oncology company whose mission is to leverage its groundbreaking T-SIGn® platform to positively impact the lives of people living with cancer. We are developing a portfolio of solid tumor-targeted T-SIGn® therapeutics which aim to enable a patient’s own immune system to recognize, attack, and clear their cancer. Akamis’ growing pipeline of T-SIGn® therapeutics is anchored by its lead clinical-stage program, NG-350A, which is being investigated in an ongoing Phase 1 clinical study in patients with metastatic or advanced epithelial tumors. Akamis has a number of T-SIGn® platform-focused collaborations with leaders in the immuno-oncology field including BMS, Merck, and the Parker Institute for Cancer Immunotherapy (PICI)

Ascend is a development partner that supports life-science innovators in bringing gene therapy products to patients. Our team has deep expertise in gene therapy development and commercialization to offer unparalleled collaboration while working to develop your products from the bench through the clinic and beyond. Scientific innovation driven by quality and responsibility are at the core of our business. To set a new standard in the industry, we have invested in a world-class analytical toolkit within our flexible and scalable AAV platform of next-gen technologies, all built to find the perfect balance of yield and quality for each process. Let’s aim higher and develop the next wave of accessible life-saving therapies together!

Driven by innovative science and our entrepreneurial culture, we are focused on the delivery of life-changing medicines that are fuelling growth and contributing value to patients and society.

At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into life-changing medicines for people living with frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). With our deep understanding of the brain and our suite of proprietary gene therapy platforms and delivery technologies, we are working relentlessly to overcome the challenges of delivering the right drug to the right place. Our innovative, neuroanatomy-led approach is designed to maximize the therapeutic potential of gene therapy to halt or potentially reverse neurodegenerative diseases. The Company’s technology is based on pioneering research from King’s College London and the UK Dementia Research Institute.

Axovia Therapeutics is dedicated to the research and development of novel AAV gene therapies that target key aspects of ciliopathies, addressing the devastating impact of the disease head-on for patients, their families and caregivers. Axovia is currently developing therapies for patients with mutations in the BBS1 gene which manifests as Bardet Biedl Syndrome. Our initial goal is to halt the progression of BBS by targeting the most debilitating aspects of the neurological, metabolic and retinal dysfunctions.

Bloomsbury Genetic Therapies ("Bloomsbury") is a clinical-stage biotechnology company, developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies. The Company was spun out of University College London and launched in October 2022 with funding from UCL Technology Fund. Bloomsbury is building a pipeline of highly differentiated first- or best-in-class programs.

The Offshore Renewable Energy (ORE) Catapult forms part of an elite network of eight technology and innovation centres established and overseen by Innovate UK, which represents a £1bn public and private sector investment over the next five years. ORE Catapult is the UK's flagship technology innovation and research centre for offshore wind, wave and tidal energy. We deliver prioritised research underpinned by world-class test and demonstration facilities, collaborating with industry, academia and Government to reduce the cost of offshore renewable energy and create UK economic benefit.

Concinnity is developing an AI-driven RNA design platform to build gene control systems for gene and cell therapies. Our RNA-based control systems will form part of the gene therapy cassette and enable precise control of therapeutic activity. Such control systems have huge potential to enhance both patient safety and therapeutic efficacy, for example by allowing a gene therapy to dynamically adjust its activity in response to patient disease state, recognise tissue type, or by allowing clinical control of the therapy after administration.

Evox Therapeutics is harnessing and engineering the natural trafficking capabilities of extracellular vesicles to develop an entirely novel class of biotherapeutics. By combining groundbreaking exosome technology from well-renowned Oxford University and the Karolinska Institutet – the home of the Nobel Prize – we aim to revolutionise the treatment of a broad range of severe disease, with profound implications for human health. Based in Oxford, UK, Evox Therapeutics was founded in 2016 by Professor Matthew Wood of Oxford University, Assistant Professor Samir EL Andaloussi and Dr Per Lundin of the Karolinska Institutet, to capitalise on foundational intellectual property springing from the groundbreaking exosome research carried out in these two world-leading labs. Backed by Oxford Sciences Innovation and with a driven multidisciplinary team, Evox is building an exosome powerhouse focusing on the development of a modular platform for clinical translation of a broad range of exosome-based therapeutics. Evox Therapeutics has built a comprehensive intellectual property portfolio encompassing key aspects of EV-based nucleic acid and protein delivery technology. Coupled with targeting technology and proprietary manufacturing and purification methods, the company is set to develop transformational therapeutics across a wide range of disease areas, using an equally wide array of therapeutic modalities.

Genflow Biosciences plc (LON:GENF) (OTCQB:GENFF) is a UK-based biotech company with R&D facilities in Belgium and a U.S. office in Cambridge, MA, driven by one mission: to deliver therapeutics that potentially halt or slow the ageing process in humans and dogs. The company’s lead compound works through the delivery of a centenarian variant of the SIRT6 gene and has yielded promising preclinical results. Managed by an experienced team with decades of experience in the pharmaceutical and biotechnology sectors, the company is optimistic that development programs will continue at pace in the next 24 months.

Discovering and developing innovative medicines to preserve people’s sight and fight the devastating impact of blindness around the world.

Intima Bioscience - A clinical stage gene and cell therapy company focused on curative intent in solid tumor cancer

MediSieve is a London biotech start-up developing Magnetic Haemofiltration, a revolutionary platform therapy that enables the physical removal of specific substances from the bloodstream of patients. It can be used (1) as a direct treatment, (2) to increase the safety and efficacy of other therapies, or (3) to enable personalised medicine. A unique, lifesaving and often cost-saving solution, there are several multi-billion-dollar markets available for this product in oncology, infectious diseases, auto-immune diseases, poisoning, drug overdose and others. Magnetic Haemofiltration has already been shown to rapidly, specifically, and effectively remove IL-6 from human plasma. The technology has completed pre-clinical validation, including animal studies, and is expected to start clinical studies in Q2 2022. MediSieve has raised £4M in equity funding and over £4M in non-dilutive grants. Seeking a further £8M at Series A to support pivotal clinical trials and regulatory approvals. EIS eligible. The company has been featured in numerous online and print media articles, including the Sunday Telegraph, Daily Mail, and Wired Magazine, and been winner or finalist in several start-up competitions such as Pitch@Palace, MassChallenge, OBN Awards, TCT Magazine Awards and CMS Healthcare Startup of the Year. Recently, our founder George Frodsham was awarded BBSRC Innovator of the Year and MIT Innovators Under 35.

Mogrify® has developed a proprietary suite of platform technologies that utilize a systematic big-data approach to direct cellular reprogramming (Rackham et al., Nature Genetics, 2016) and the maintenance of cell identity (Kamaraj et al., Cell Systems, 2020). The platforms, MOGRIFY® and epiMOGRIFY®, developed over a 12-year period via a multi-national research collaboration, deploy next-generation sequencing, gene regulatory and epigenetic network data to enable the prediction of the transcription factors and growth factors required to produce any target human cell type from any source human cell type. Mogrify is applying its proprietary and award-winning platforms to generate the functional cell types required to transform the lives of patients, by delivering a novel class of in vivo reprogramming therapies across ophthalmology, otology, metabolic and other areas of degenerative disease. Uniquely positioned to address a regenerative medicine market estimated to be worth $150 billion USD by 2028, Mogrify is commercializing its technology via a combination of in vivo reprogramming therapy development, co-development partnerships, as well as the exploitation of the platform in other therapeutic and non-therapeutic applications. Based in Cambridge, UK, the Company has raised over $40 million USD funding from Parkwalk, Ahren Innovation Capital, 24Haymarket, Trend Investment Group, Dr. Darrin M. Disley, OBE, Dr. Jonathan Milner and the University of Bristol Enterprise Fund III, as well as strategic investors; Astellas Venture Management.

Low-cost, rapid whole genome sequencing is the future of infection control and clinical microbiology. Next Gen Diagnostics offers the low cost, high throughput, and automated sequencing and bioinformatics for a range of application, including tracking the transmission of disease in hospitals and pathogen surveillance for public health labs.

NunaBio is a privately-owned specialist DNA synthesis and development company. We are engaged in CRO (Contract Research Organizations) and CDMO (Contract Development and Manufacturing Organisation) activities. The company has built a solid reputation for its R&D flexibility and innovative approach to complex and challenging projects. NunaBio's core expertise is research and development into cell-free DNA synthesis, process optimisation, route design, scale-up and small-scale manufacture for clinical studies.

OXB (LSE: OXB) is a global quality and innovation-led contract development and manufacturing organisation (CDMO) in cell and gene therapy with a mission to enable its clients to deliver life changing therapies to patients around the world. One of the original pioneers in cell and gene therapy, OXB has 30 years of experience in viral vectors; the driving force behind the majority of cell and gene therapies. OXB collaborates with some of the world’s most innovative pharmaceutical and biotechnology companies, providing viral vector development and manufacturing expertise in lentivirus, adeno-associated virus (AAV), adenovirus and other viral vector types. OXB's world-class capabilities span from early-stage development to commercialisation. These capabilities are supported by robust quality-assurance systems, analytical methods and depth of regulatory expertise. OXB offers a vast number of unique technologies for viral vector manufacturing, including a 4th generation lentiviral vector system (the TetraVecta™ system), dual plasmid system for AAV production, suspension and perfusion process using process enhancers and stable producer and packaging cell lines.

Oxford Genetics is a synthetic biology company focused on developing novel technologies to overcome challenges associated with the discovery, development and production of biologics, gene therapies, cell therapies and vaccines.

Purespring, which launched in November 2020 with £45 million of funding from Syncona Ltd, is the first company to be directly targeting the podocyte, a specialized kidney cell implicated in many kidney diseases, through AAV gene therapy. This approach is based on the work of Professor Moin Saleem, Professor of Paediatric Renal Medicine at the University of Bristol, where he heads a world leading group researching glomerular diseases.

Tenpoint Therapeutics Limited is a global, clinical-stage biotech company developing groundbreaking treatments to rejuvenate vision in the aging eye. Its pipeline includes paradigm-shifting treatments for ophthalmic indications with the greatest need and global market potential, including presbyopia, cataracts and geographic atrophy. Its lead asset, BRIMOCHOL PF, is a novel, pupil-modulating therapeutic designed to correct the loss of near vision associated with presbyopia, a condition that afflicts approximately 2 billion people globally. BRIMOCHOL PF has completed its first Phase 3 pivotal trial (BRIO-I) and will complete the second pivotal study, BRIO-II, in 2025. This topical ophthalmic is poised for launch in 2026. Tenpoint’s leadership team includes ophthalmic industry luminaries with track records of successful approvals and commercialization of blockbuster drugs. A privately held company, Tenpoint Therapeutics is backed by AdBio Partners, British Patient Capital, Eight Roads, EQT Life Sciences, F-Prime Capital, Hillhouse Capital Management, Qiming Venture Partners USA, Sofinnova Partners, and UCL Technology Fund.

Manufacturing.

Trogenix unites cutting-edge technologies in genomics, oncology, immunotherapy, and gene therapy to create a revolutionary therapeutic approach through its Odysseus platform. Using proven AAV vectors, our proprietary Synthetic Super-Enhancers (SSEs) are delivered directly to tumour cells without detection. Our SSEs enable unprecedented precision in gene control, effectively revealing cancer to the body's immune system and killing tumour cells. For any cells escaping Trogenix's technology, our 'Trojan horse' reawakens the immune system, countering recurrence and offering potentially curative 'one-and-done' treatments for aggressive tumours. With our lead asset showing curative potential in preclinical glioblastoma studies, we're poised to transform treatment paradigms across multiple cancer types and explore applications beyond oncology, such as regenerative medicine.