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Adeno-Associated Viruses: Future Shapers of Cell and Gene Therapy

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Cell and Gene Therapy (CGT) offer endless hopes to a wide range of illnesses, from rare genetic diseases to oncology, to diabetes. Although tremendous progress has been achieved in the last decade and the first drugs have already reached the market, multiple constraints and limitations remain to be overcome in order to provide safe, cheap and efficient therapies.


Among the many vectors that are being investigated in order to deliver the right genetic material to the right cells, Adeno-Associated Viruses (AAV) are already considered as candidates with high potential. Indeed, they offer the advantages of a virus when it comes to infiltrating a cell and delivering DNA, without the risks linked to viral genes. This has resulted in a continuously growing interest in AAV, with a steadily increasing amount of research, patents and drugs in development across all medical conditions.


Trends in AAV over the last 5 years


Source: Signals Pharma Playbook Insights


Trends in number of Research Papers mentioning AAV over time (May 2014 – April 2019)


Source: Signals Pharma Playbook Insights


Trends in number of Patents mentioning AAV over time (May 2014 – April 2019)


Source: Signals Pharma Playbook Insights


New developments in the AAV space

Various signals indicate that both academic institutions and companies are searching for solutions to the multiple obstacles that need to be overcome, such as virus mass production, development of a virion that will not cause a host immune reaction, and more.

For example, Virovek, a private company based in California and founded by Dr. Haifeng Chen, patented last year a unique technology using insect cells to produce large amounts of AAV. A lot of players are also working on the design of virus envelopes so that they do not wear the classical markers, preventing them from being recognized by the immune system and destroyed before they can reach their destination, e.g. University of California’s patent (Oct 2018).


When looking at the therapeutic applications of innovation in the CGT space, promising results are becoming more and more apparent. Specifically, gene therapy appears to be a promising solution against hemophilia, a rare disorder in which the patient's blood does not coagulate properly, leading to longer bleedings and potential disastrous consequences.

UniQure, one of the leading companies specialized in CGT taking part in the race to find a cure for this deadly disease, currently has four clinical trials. They are exploring an AAV5 targeting the defecting protein factor IX, typically found at levels of 50% to 150%, in healthy subjects, but below 50% in hemophiliac patients.

UniQure announced few days ago that a single infusion of their drug, AMT-061 using this very AAV, was still effective six months after transfusion, with resulting factor IX levels at 50% on average, a level that is significantly above the risk threshold. As per the graph below, we can see there are currently a total of 26 clinical trials, when there was not a single one in 2014.


Top 10 companies conducting clinical trials using gene therapy to treat hemophilia


Source: Signals Pharma Playbook Insights


A promising future for AAV

As difficulties linked to the safety, scalability and efficacy of AAV in cell and gene therapy are being overcome, treatments and even cures for diseases such as haemophilia, are becoming a reality for patients. Growing scientific evidence supports the use of AAV as a key mechanism for delivering cell and gene therapies, an increasing number of players will enter the race, attracted by the potentially high returns on the investment in therapies in the space.



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Written by Naomi Karoubi, PhD

Life Science Business Consultant at Signals Analytics

Naomi has worked for Signals Analytics for two years, both as a consultant and a project manager. She completed her Ph.D. in neuro-economics at Ben Gurion University and also holds an MBA from ETH Zurich.


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