The UK is establishing itself as a hub for the development of advanced therapeutics which refers to new medical products that use gene or cell therapy. Whereas gene therapy involves the transfer of genetic material, usually in a carrier or vector, into the appropriate cells for uptake of the genetic material, cell therapy is the transfer of cells with relevant function into a patient.
The cell and gene therapy industry in the UK is part of a thriving biotech community supported by a large contingent of specialist manufacturing companies. Recently these companies have come to light as being key to the UK’s outstanding COVID-19 vaccine manufacturing response; utilising analogous processes for virus production.
According to data released this year by the UK Cell and Gene Therapy Catapult¹, the UK accounts for over 12% of global gene and cell therapy (or advanced therapy medicinal product – ATMP) clinical trials. In 2020 over 3,000 jobs had been created in the sector with over 90 advanced therapy developers based in the UK. With a £300 million turnover in 2020, the UK cell and gene therapy sector is booming. By 2035 it is expected that the industry could be worth £10 billion and provide 18,000 jobs.
Each year, the Cell and Gene Therapy Catapult publishes data from its clinical trials database. This government-supported body aims to build a world-leading cell and gene therapy sector in the UK. It enables the translation of early stage research into commercially viable and investable therapies by bridging the gap between scientific research and full-scale marketing. Its vision is for the UK to be “a global leader in the development, delivery and commercialisation of cell and gene therapies, where businesses can start, grow and confidently develop advanced therapies, delivering them to patients rapidly, efficiently and effectively”.
At the time of the report, there were 154 ongoing trials in the UK, representing more than a 20% increase from last year, with a majority (70%) employing viral vector mediated gene transfer. For in vivo gene therapy clinical trials, 81% are dominated by adeno-associated viral (AAV) vectors. The number of Phase III trials has grown by 27% since last year with an increase reported across the board. In fact, the number of trials has been growing by an average of 25% per year since 2013.
The data also suggests that trials are progressing through set up and reaching active recruitment more quickly. In contrast to traditional small molecule therapies, the development of cell and gene therapies requires novel approaches to deliver these lifesaving solutions to patients. The administration of cell and gene therapies needs to be expertly done and carefully tracked to identify any potential adverse effects. The UK now has a network of Advanced Therapy Treatment Centres, the first of their kind in the world, which will develop these new systems and processes, and alongside the NHS, will provide the right environment for allowing innovative therapies to reach patients, establishing the UK as a global-leader.
The main therapy area for cell and gene therapies clinical trials remains oncology (35%) followed by ophthalmology (12%) and haematology (12%).
For cell-based therapies, T cells remain the dominant cell type under investigation accounting for 44% of clinical trials. This is perhaps to be expected since research into oncology, the largest therapeutic sector, is largely T cell focused.
The UK Cell and Gene Therapy Catapult data further showed that just under 75% of UK cell and gene therapy trials are now sponsored by commercial organisations; a huge increase when compared with only 25% in 2013. The majority of commercially sponsored trails are backed by non-UK based companies, demonstrating the appeal of the UK ecosystem in terms of both expertise and regulation. In 2020 there were more than 90 companies developing advanced therapies in the UK (with 24% of European developers headquartered in the UK), 25 manufacturing facilities, and three companies that have each reached over $1 billion in value. It is evident that international companies are recognising the appeal of the UK cell and gene therapy community for the development and manufacture of advanced therapies.
As a further example of the UK commitment to gene therapies, the UK has delivered the 100,000 Genomes Project via Genomics England. The project sequenced 100,000 genomes from NHS patients affected by a rare disease or cancer.To date, actionable findings have been found for approximately 1 in 4 rare disease patients and around 50% of cancer cases contain the potential for a therapy or a clinical trial .
Despite the incredibly exciting future ahead, there are currently very few advanced therapy products approved for use in the UK. Due to the length of time and costs associated in taking an advanced therapy to approval, it is important for companies to have an effective IP strategy to maintain their market advantage.
Gene and cell therapies require different IP considerations to traditional small molecule drugs. Furthermore, different jurisdictions have different requirements for the patenting of biological material, stem cells and methods of treatment and surgery and it is important that such considerations are taken into account during the drafting of any patent application.
It is further important to consider the different aspects of cell and gene therapies which can be afforded patent protection. For example: cell harvesting, the gene therapy vector or therapeutic nucleic acid sequence, methods of modifying cells, the modified cells per se, the use of the vector/cells in therapy or for a particular medical condition. There are multiple avenues of protection to consider.
Inventors are often under pressure to get their technology protected prior to academic publication and there is a need to find a balance between early filing and developing supporting data for the patent application. It is important to be strategic in using the time available to generate the best kind of data to support the invention. Broad supporting data is important for platform-type inventions, in order to demonstrate that the invention can be used across a wide range of applications. For an incremental invention, more focused data may be appropriate. Your patent attorney will be able to advise you as to the right balance between early filing and supporting data depending on your overall strategy and commercialisation goals.
As the gene and cell therapy space is becoming crowded, it is also important to not only consider what is new and inventive from a patentability point of view, but also to remember to review third party IP rights to avoid any potential infringement and consider obtaining suitable licenses.
At Venner Shipley we have several attorneys who are experts in the field to help companies navigate their commercial strategies, patentability requirements and operational freedom to practice their inventions. If you would like any assistance in this regard, please do not hesitate to get in touch.