Some biotech investors had probably never heard of exosomes until Codiak’s chief executive, Doug Williams, made his pitch at the JP Morgan conference two weeks ago. But the area has been gaining traction, and Jazz’s $56m up-front payment to Codiak is its biggest endorsement to date. Roche and Boehringer Ingelheim have also bought into rival technologies, which broadly seek to use exosomes as delivery vehicles for proteins, small molecules, RNA and other therapeutics. Naturally occurring exosomes are vesicles that bud off from many mammalian cells and are subsequently internalised by other cells, along with their cargo, hence their role in cell-cell communication. This gives rise to their potential as a new class of delivery vehicles for therapeutics, and Codiak seems to be furthest advanced here, boasting of being able to internalise various molecules via protein tags, and of targeting exosomes to specific cells using external markers. As a nascent technology this carries numerous problems, including accurately coding for and delivering the target therapeutic, not to mention questions around intellectual property and manufacturing at scale. While there are three listed players – Capricor, Puretech and Reneuron – no company is close to entering the clinic yet.
|The key exosome players (all early preclinical)|
|Company||Type||Technology source||Selected deals||Lead focus in exosomes|
|Capricor Therapeutics||Nasdaq-listed*||Cedars-Sinai Medical Center||2016 funding: $4.2m from NIH, $2.4m from US DoD||CAP-2003, cardiosphere-derived cells to generate exosomes|
|Puretech||London-listed||James Graham Brown Cancer Center & University of Louisville||Jul 2018: Roche||Milk exosomes for oral bioavailability of macromolecules, incl antisense oligos, and complex small molecules|
|Reneuron||London-listed||Jan 2019: undisclosed US biopharma company||Permanent stem cell line to produce exosomes at scale|
|Codiak Biosciences||Private, US||University of Gothenburg||Jan 2019: Jazz, $56m up front; $92m raised in private cash||Exosomes with internal/external targeting, delivering Sting agonist or IL-12|
|Evox Therapeutics||Private, UK||Oxford University||Dec 2017: Boehringer Ingelheim for mRNA delivery||Protein-replacement exosomes for Niemann-Pick & DMD|
|Stem Cell Medicine||Private, Israeli||Tel Aviv University||Mesenchymal stem cell derived vesicles for autism|
|Aruna Biomedical||Private, US||Raised $5.3m last Sep||AB126, neural exosomes that cross BBB to treat stroke|
|Ciloa||Private, French||Delivering membrane proteins|
|Molecuvax**||Private, US||Subsidiary appears inactive||Cancer clinical trial planned in 2016|
|Tavec Pharmaceuticals||Private, US||Johns Hopkins University||Company appears inactive||miRNA delivery to cancer cells|
|Notes: *reversed into Nile Therapeutics in 2013; **subsidiary of Therapeutic Solutions International.
Source: Vantage analysis.