IP Review Summer 2018

5 field, likely due to a significant drop in research and development. Encouragingly, things started to change for the better in 2012. The Dutch company uniQure obtained the first European marketing authorisation for their gene therapy product Glybera for the treatment of lipoprotein lipase deficiency, a rare inherited disorder which can cause severe pancreatitis. This helped to prove the concept that gene therapy works and can be taken to the market, although Glybera itself was never a commercial success. This is perhaps partly due to very low patient numbers (about 1-2 in 1,000,000 people are affected with the disease) and the large price tag of about €1 million. With Glybera demonstrating that it could be done, there was a big jump in clinical trial approvals from 2012 to 2015 to the highest ever level (as shown in Figure 1), showing there was renewed interest in gene therapies. Further, for the first time since the late 1990s, there was a significant increase in patent filings from 2014 to 2016, showing that innovation in this area is on the increase as biotech companies once again believe it has legitimate commercial potential. Since then, Strimvelis, GlaxoSmithKline’s gene therapy product for ADA-SCID (the same condition treated in the first ever gene therapy clinical trial in 1990) received a marketing authorisation in 2016 in Europe. Following this, Spark Therapeutics’ gene therapy product Luxturna was granted the first US marketing authorisation towards the end of 2017 for the treatment of an inherited form of vision loss that may result in blindness. There have also been authorisations to allow the commercial use of CAR-T cells in Europe and the US. CAR-T cells are a type of immune cell engineered to detect and destroy cancer cells. Although these are not true gene therapy products, the production of these cells uses a gene transfer technique similar to gene therapy. Although only three gene therapy products and two CAR-T cellular products have received marketing authorisations in the US or Europe to date, a whole raft of further products are being developed for a range of diseases, some of which are more commonplace, such as haemophilia and sickle-cell disease. They are expected to be granted marketing authorisations over the next few years with estimates of there being two in 2018, up to 10 in 2019 and possibly as many as 13 in 2020. This surge in commercial development and success of new gene and cell therapies is on the back of a large increase in investment. Figure 3 below illustrates the increasing annual global investment into gene and cell therapy companies since 2013, with a jump from $3.3 billion in 2016 to $4.2 billion in 2017. This increase in investment also seems to correlate with the increase in patent filings in this period. Patent filing data is not yet completely available for 2017 but it will be interesting to see if the upward trend continues in line with investment and as the field continues to grow. At the end of 2017, there were almost 2,000 gene therapy clinical trials ongoing, including about 100 trials in pivotal Phase 2/3 or Phase 3 studies. Remarkably, gene therapy is now the second largest class of drugs being developed, bigger than antibodies and only behind more traditional New Chemical Entities (NCEs). Therefore, it seems that gene therapy is finally fulfilling its early promise. To find out more contact Justin Wilson jwilson@withersrogers.com Gene Therapy Patent Applications Filed 200 400 800 1000 1200 600 Figure 2 Annual Sum of Amount raised by Financing Type 2015 2017 2016 2013 2014 All IPO financings All follow-on financings All venture financings Figure 3