Targeted delivery of dystrophin has the potential to be a highly effective treatment for Duchenne muscular dystrophy
Targeted delivery of dystrophin has the potential to be a highly effective treatment for Duchenne muscular dystrophy
OXFORD, England, November 13, 2018 /PRNewswire/ -- Evox Therapeutics Ltd (‘Evox’ or the ‘Company’), a leading exosome therapeutics company, today announces that it has secured £655,000 in funding from Duchenne UK. The grant will be invested to support exploration of the Company’s exosome-based therapeutic platform to deliver either full-length dystrophin or its shorter variants in pre-clinical models of Duchenne muscular dystrophy (‘Duchenne’).
Duchenne is a highly debilitating, progressive, muscle-wasting disorder caused by the lack of functional dystrophin protein, for which there is currently no cure. Delivery of dystrophin or its shorter variants to these patients has the potential to be a highly effective treatment option in Duchenne.
Evox is engineering exosomes, the body’s natural vesicular delivery system, to enable a wide variety of drugs to reach previously inaccessible tissues and compartments, such as crossing the blood brain barrier to deliver drugs to the central nervous system, intracellular delivery of proteins, and extra-hepatic delivery of RNA therapeutics. Evox is developing its own proprietary pipeline of exosome-based therapeutics for the treatment of rare, life-threatening diseases with significant unmet need.
Dr Antonin de Fougerolles, Chief Executive Officer of Evox, commented: “We are excited to be working with Duchenne UK on exploring a potential transformative solution to treat Duchenne patients. We will conduct research to assess the potential of our exosome drug platform to deliver functional dystrophin which is missing or defective in these patients. This work will also allow us to explore targeted delivery of exosomes to muscle which may be beneficial not only for Duchenne patients, but also ultimately for patients with other musculoskeletal diseases.”
Emily Crossley & Alex Johnson, Co-CEOs of Duchenne UK said: “We are delighted to be working with Evox to advance this potentially exciting work to help in the field of Duchenne muscular dystrophy. One of the most challenging aspects of using viruses to deliver gene therapy is that many patients may already have what are known as pre-existing antibodies - they are ‘resistant’ to the virus - and so the replacement gene carried by the virus will never reach its target. Exosomes could provide a potential new method for effectively, safely, and repeatedly delivering genetic material encoding for dystrophin to muscles without the problem of pre-existing antibodies.”
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is the most common fatal genetic disease diagnosed in childhood. Children born with DMD cannot produce the protein dystrophin which is vital for muscle strength and function. Muscle weakness starts in early childhood. Many use a wheelchair by around the age of 12. As deterioration continues it leads to paralysis and early death, often in their 20s. It almost exclusively affects boys. There is no treatment or cure. In the UK there are around 2,500 boys affected and around 300,000 worldwide. It is classified as a rare disease.
About Evox Therapeutics
Evox Therapeutics is a privately held, Oxford-based biotechnology company focused on harnessing and engineering the natural delivery capabilities of extracellular vesicles, known as exosomes, to develop an entirely new class of therapeutics. Backed by leading life sciences venture capital groups and supported by a comprehensive intellectual property portfolio, Evox’s mission is to positively impact human health by creating novel exosome-based therapeutics for the treatment of various severe diseases with limited options for patients and their families. Evox has created substantial proprietary technology to modify exosomes using various molecular engineering, drug loading, and targeting strategies to facilitate targeted drug delivery to organs of interest, including the brain and the central nervous system. Exosome-based drugs have the potential to address some of the limitations of protein, antibody and nucleic acid-based therapies by enabling delivery to cells and tissues that are currently out of reach using other drug delivery technologies, and Evox is leading the development within this emerging therapeutic space.
For further information visit: http://www.evoxtherapeutics.com
About Duchenne UK
Duchenne UK is a lean, ambitious and highly focused charity with a clear vision: to fund and accelerate treatments and a cure for Duchenne Muscular Dystrophy. The charity has been formed by the coming together of Joining Jack and Duchenne Children’s Trust, the two biggest funders of research in the UK in the last three years. Its president is HRH The Duchess of Cornwall. Its patrons include the broadcasters Krishnan Guru-Murthy and Mary Nightingale, and the sports stars Owen Farrell, Kris Radlinski and Andy Farrell.
SOURCE Evox Therapeutics
