The Cell and Gene Therapy Industry: Looking at 2016 and Beyond

Politically 2016 was surprising and dramatic here in the United Kingdom and elsewhere. Although it was not surprising for cell and gene therapies per se, the growth that we have seen in this industry has been pretty dramatic around the world as well.

The industry has seen investors of all types maintaining their interest in cell and gene therapy, with a number of new deals made: e.g., Bluebird bio and Medigene signed a strategic R&D collaboration and licensing agreement, and Allergan acquired RetroSense Therapeutics. Therapies also started gaining approval, and I expect that trend will continue as more come through the pipeline — particularly in oncology — over the next 12 months. It’s no surprise that big pharmaceutical companies have openly described the cell and gene therapy industry as a strong area for growth and dramatic change to patient care as well as a source of future profitability. The Cell and Gene Therapy Catapult has continued to build on this growth toward its vision for the United Kingdom to be a global leader in development, delivery, and commercialization of cell and gene therapies.

One of the biggest booms of 2016 continued on the same theme from previous years in gene-modified cell therapies. We had identified this area early on and invested in two potential products with academic partners: a T-cell receptor (TCR) to the WT-1 cancer marker and a chimeric antigen receptor T (CAR-T) cell therapy to an angiogenesis marker. We were interested both in helping to develop those products and in using them as “pathfinders.” Their success could lower barriers to commercialization for others.

In the United States, 2016 also marked the start of a race for companies to submit such a product for treating cancer to the US Food and Drug Administration (FDA). I believe that we will start to see their oncology products on the market — possibly in 2017 — and that they will deliver great results to patients who have no other options. Over time, gene-modified cell products also could become first-line therapies.

Oncology isn’t the only area in which promising advanced therapies are seeing big-pharma investment. In autumn 2016, GlaxoSmithKline (GSK) launched Strimvelis gene therapy, which modifies a patient’s own bone marrow. The new product gained approval in Europe for treatment of adenosine-deaminase severe combined immunodeficiency (ADA-SCID), a rare immune disease in children.

Challenges Lie Ahead
Although growth has been dramatic, barriers remain for this industry to break down in 2017 and beyond. The cost of goods (CoG) and pricing of advanced-therapy products are key issues. We did see some interesting moves in 2016, however. For example, the approach GSK took to pricing Strimvelis — as a one-off treatment with a money-back guarantee if it didn’t work — was an innovative means of getting the product to patients who need it. I’m sure ideas will emerge that are different from traditional pharmaceutical strategies — and rightly so. Cell and gene therapies can be one-time cures rather than drugs that patients must take repeatedly for life, so the approaches to their pricing will have to be different as well.

The rewards will be substantial for whoever solves issues such as reimbursement models and CoG. I believe these problems need to be tackled at national and international levels rather than by individual companies. My organization has invested in a large-scale manufacturing center in Stevenage (Hertfordshire, UK) for cell and gene therapies that will open later in 2017. One goal for that center is to provide a site around which supply chain companies can locate, bringing additional long-term jobs to the area. Let’s hope that cell and gene therapies are high on the list when the UK government publishes its industrial strategy.

Several years ago, I couldn’t have imagined that regulatory systems would begin to evolve competitive approaches — yet that is what we have seen: in the United States with its breakthrough designation and in Japan with its twin-track system. This brings me back to 2016 politics and Brexit here in the United Kingdom. We are already starting to look at ways to make that advantageous to our industry.

I believe that the UK Medicines and Healthcare Products Regulatory Agency (MHRA) is one of the most proactive regulators in the world. It is creating the right sort of regulatory environment and with a “one-stop shop for regulatory advice” that has helped many companies make rapid progress into clinical trials. If the agency continues such work, then the United Kingdom can become increasingly attractive in Europe and globally as a test bed for cell and gene therapy industrialization.

Yes, 2016 was a great year of growth for the advanced-therapies industry. But there’s still much work to do toward ensuring that we get cell and gene therapy products to the patients who need them. This year will see our manufacturing center open, and more new products should gain approval and come to market. Those two things can help to continue the advancement of these therapies in people’s minds as a pillar of healthcare.

Keith Thompson is chief executive officer of Cell and Gene Therapy Catapult, 12th Floor Tower Wing, Guy’s Hospital, Great Maze Pond, London SE1 9RT, UK; 44-20-3728-9500; https://ct.catapult.org.uk.