Evox Therapeutics Secures £1.5M Funding to Advance Exosome-Based Therapy for Argininosuccinic Aciduria (ASA)

VCBeat (WeChat Official Account: vcbeat) has learned that Evox Therapeutics, a leading biotechnology company specializing in exosome-based therapies, recently announced the completion of a new £1.5 million financing round, with Innovate UK, a UK non-profit investment organization, as the lead investor. The funds will be used to support Evox Therapeutics’ collaboration with University College London to jointly develop an exosome-based clinical therapy for argininosuccinic aciduria (ASA).

Evox Therapeutics has completed four rounds of financing totaling £47.2 million, with existing investors including Oxford Sciences Innovation, a science and technology investment firm; Redmile Group, a healthcare-focused investment firm; and Duchenne UK, a medical charity.

Argininosuccinic aciduria (ASA) is a rare, inherited metabolic disorder that can be life-threatening in severe cases. The hallmark of ASA is a deficiency in argininosuccinate lyase (ASL). ASL plays a central role in normal human metabolism: it detoxifies ammonia within the liver-based urea cycle, and it synthesizes nitric oxide from L-arginine as part of the citrulline-nitric oxide cycle. Symptoms of ASA can manifest as early as the neonatal period, presenting as hyperammonemia and a multi-organ disease with neurological phenotypes. Although ASA is one of the more common urea cycle disorders, the medical needs of most patients with ASA remain unmet.

Evox Therapeutics is dedicated to harnessing the natural delivery capabilities of extracellular vesicles (commonly referred to as exosomes) to develop a novel therapeutic approach that enables a wide variety of drugs to reach previously inaccessible tissues. For instance, this technology can help drugs cross the blood-brain barrier to target the central nervous system, facilitate intracellular delivery of biologics, and enable the application of RNA therapies for specific liver diseases. University College London, which possesses world-leading expertise in urea cycle disorders, will evaluate Evox Therapeutics’ natural vesicle delivery system using exosome-based therapies in models of argininosuccinate lyase (ASL) deficiency.

Dr. Per Lundin, Chief Operating Officer and Co-Founder of Evox Therapeutics, commented: “We are delighted that Innovate UK has chosen to support our work in developing an exosome-based therapy for patients with argininosuccinic aciduria (ASA). We aim to leverage the tissue distribution profile of exosomes to deliver fully functional enzymes to the liver and other critical tissues, such as the central nervous system, for the treatment of various diseases. We also look forward to our academic collaboration with University College London, which is at the forefront globally in developing clinical models and patient treatments for ASA.”

Dr. Paul Gissen of the UCL Great Ormond Street Institute of Child Health said, “We are delighted to receive joint funding from Innovate UK and Evox Therapeutics. This funding will enable us to evaluate the potential efficacy of this novel exosome-based therapy in a model of argininosuccinic aciduria (ASA), a metabolic disorder. Patients with ASA often suffer from liver, brain, and other organ dysfunctions, representing a significant subset of individuals with genetic diseases for whom there remains an unmet need for disease-specific treatments. If our project proves successful, the same principles could potentially be applied to the treatment of other severe conditions.”

Evox Therapeutics, a private biotechnology company headquartered in Oxford, UK, was founded in 2016. Backed by leading life sciences venture capital firms, Evox Therapeutics is on a mission to treat a range of serious diseases by developing novel exosome-based therapies. The company has generated extensive proprietary technologies for modifying exosomes and is dedicated to employing various molecular engineering, drug loading, and targeting strategies to facilitate targeted drug delivery to diverse organs, including the brain and central nervous system. By targeting cells and tissues that are generally inaccessible to conventional drugs, Evox Therapeutics’ exosome-based therapeutics have the potential to overcome certain limitations of protein-, antibody-, and nucleic acid-based treatments, positioning the company as a leader in this emerging therapeutic field.

Innovate UK, established in 2007 and headquartered in Swindon, UK, is a non-profit organization based in the United Kingdom that primarily provides investment to science and technology innovation companies. It has received the Biocatalyst Award in the medical category. Innovate UK also organizes funding competitions for enterprises and research institutions across various industries. To date, Innovate UK has made 205 investments.

(Compiled by: Wang Shuijing)