pharmaceutical industry and academic scientists working in neuroscience, cell and gene therapy, molecular biology and imaging; accelerating the effective translation of bench discoveries to clinical pipelines. Both start-up and more established com- panies are successfully focusing on engineering EVs, mainly using smaller so-called exosomes, to facilitate drug delivery to target organs, paving the way towards drug delivery to challenging tissues, such as the central nervous system. Development of next-generation therapeutic

platforms involving EVs spans across various tis- sues, including those hard to reach. CAP-2003 from Capricor Therapeutics (USA) is currently undergoing pre-clinical studies, highlighting the drug’s potential as a treatment for diseases caused by inflammation and fibrosis through the modula- tion of the immune system to restore cell damage. The therapeutic agent consists of exosomes isolat- ed from the company’s proprietary cardiosphere- derived cells (CDCs). These exosomes also have the capacity to reduce scarring. Another US-based start-up company, Kimera

Labs, specialises in the production of exosomes isolated from Mesenchymal Stem Cells (MSC). Its highest-purity MSC exosomes are optimised to stimulate healing processes and tissue regenera- tion. XoGlo® is a sterile, cell-free isolate of MSC exosomes containing growth factors involved in skin repair, beautification and regeneration. The proprietary pipeline of Evox Therapeutics,

based in Oxford (UK), encompasses seven exo- some-based therapeutic products, ranging from therapeutic

discovery stage to FDA

Investigational New Drug assessment stage. These drugs have the potential to address lysoso- mal storage disorders (LSDs), with a combined incidence of around one in every 5,000 births. Another area of focus for Evox are genetic dis- eases described as Inborn Errors of Metabolism (IEMs), where toxic substances accumulate impacting normal metabolism and resulting in impaired muscle functioning. Evox’s technology revolves around the loading of therapeutics and tissue targeting moieties on the surfaces of exo- somes, which facilitates highly-specific delivery of therapeutic proteins to the target organ. A broad spectrum of serious diseases is also

addressed by Codiak (USA), whose engEx™ Platform aims at engineering exosomes targeting immune-based diseases, metabolic and fibrotic dis- orders, neurodegenerative disorders, cancer and rare diseases. The current product pipelines encompass eight candidates in various therapeutic areas.


A new way to tackle cancer and control the

tumor microenvironment is through exosome depletion, potentially as an adjuvant therapy. EVs generated by cancer cells can lead to the inhibition of immune responses and stimulation of new blood vessels. Depletion of cancer cell-generated exo- somes may lead to inhibition of tumour growth and increased efficiency of anticancer agents. US- based Aethlon Medical has devised a therapeutic hemofiltration technology to capture circulating viruses and cancer-promoting exosomes through affinity attachment. At present, the Hemopurifier® is being advanced under an FDA-approved clinical study. Aethlon is also the majority owner of Exosome Sciences, Inc, a company focused on the discovery of exosomal biomarkers to diagnose and monitor life-threatening diseases.

The growth of exosomes diagnostics and therapeutics Altogether, the global market for exosome diag- nostics and therapeutic market is expected to grow from $34.7 million in 2018 to $186.2 million in 2023, with a substantial five-year compound growth of 39.9%5. Major factors that are con- tributing to the growth of the market include the increased demand for the use of exosomes as biomarkers in cancer diagnosis, new approaches to develop EVs in the liquid biopsy market (a grow- ing sector in cancer diagnostics), the potential of using EVs for targeted delivery vehicles of thera- peutic molecules to target cells, as well as the increased prevalence of cancer in a global ageing population. In the US and Europe, the commercialisation of

EV-mediated targeted therapeutics is financially more rewarding than companion diagnostics, which is boosting the commercialisation of EV pipelines with product launches in the UK, the US, Italy and Korea. North America accounted for more than half of the market share in 2016. Emerging therapeutics in countries with ‘fast- track’” EV regulatory authorities including Korea, Italy and China, will also contribute to the growth, even if the FDA takes a longer time for its decision to approve EV therapeutics. Furthermore, EVs have the great potential to

impact and accelerate diagnostics and discovery of therapeutics in a vast range of markets, such as the biomarker and diagnostic industry, the microRNA market, the liquid biopsy market, the stem cell technologies market, the prenatal diag- nostic market, the agriculture and food industry, microfluidics and nanotechnology and the skin- care industry5,6.

Drug Discovery World Winter 2019/20

Page 1  |  Page 2  |  Page 3  |  Page 4  |  Page 5  |  Page 6  |  Page 7  |  Page 8  |  Page 9  |  Page 10  |  Page 11  |  Page 12  |  Page 13  |  Page 14  |  Page 15  |  Page 16  |  Page 17  |  Page 18  |  Page 19  |  Page 20  |  Page 21  |  Page 22  |  Page 23  |  Page 24  |  Page 25  |  Page 26  |  Page 27  |  Page 28  |  Page 29  |  Page 30  |  Page 31  |  Page 32  |  Page 33  |  Page 34  |  Page 35  |  Page 36  |  Page 37  |  Page 38  |  Page 39  |  Page 40  |  Page 41  |  Page 42  |  Page 43  |  Page 44  |  Page 45  |  Page 46  |  Page 47  |  Page 48  |  Page 49  |  Page 50  |  Page 51  |  Page 52  |  Page 53  |  Page 54  |  Page 55  |  Page 56  |  Page 57  |  Page 58  |  Page 59  |  Page 60  |  Page 61  |  Page 62  |  Page 63  |  Page 64  |  Page 65  |  Page 66  |  Page 67  |  Page 68