The UK preclinical database covers preclinical research activity concerning cell, gene and other advanced therapies that the Cell and Gene Therapy Catapult believes to be ongoing in the UK as of August 2017. It supersedes the database of July 2016, and both are available on our website.
As a centre of translational excellence in the UK, the CGT Catapult is progressing a portfolio of projects with the UK and international community. The UK preclinical research database gives us a valuable mechanism for tracking cell and gene therapy trends and enables us to plan activities appropriately and identify needs for the sector going forwards. It also complements our UK clinical trials database. The database is intended to be of use to academics, commercial organisations and investors operating in the cell and gene therapy space by providing insight and visibility into the diversity of preclinical research and demonstrating the rich science base in the UK. It is also used to provide an indication of the likely future directions of cell, gene and other advanced therapies.
In this year’s edition, project data were collected using a secondary research approach in which information was captured by surveying the literature and through inspecting webpages belonging to relevant companies and academic principal investigators undertaking preclinical research. For the avoidance of doubt, all data presented here are publicly available information.
Data concerning a large volume of projects were collected and these studies implement a diverse variety of therapeutic agents in a wide assortment of disease areas. The technology readiness level of the projects captured varies from early-stage research to projects in which clinical trials are expected to be undertaken in the coming years.
The aim of this study is to provide top level information about preclinical research within the UK, if more detail is required we can be contacted via the email address below. The input of the cell and gene therapy community is important to help us maintain its relevance, and we welcome your updates, additions and corrections, which you can submit to us at: firstname.lastname@example.org.
Overview of preclinical research being undertaken in the UK
The preclinical research that has been identified this year covers a broad range of disease areas with oncological studies (including both haematological malignancies and solid tumours) being the most frequent at over 100 projects. This sum is closely followed by neurological disorders, also having more than 100 projects. There is also significant activity in ophthalmological research programmes and in programmes studying cardiovascular disease and orthopaedics. Figure 1 provides details of number of projects specific to each disease area.
Therapeutic delivery method
In alignment with the range of disease areas represented by the identified preclinical research being undertaken in the UK, there are a wide assortment of therapeutic cell types being investigated. Figure 2 provides a broad overview of the types of therapeutic delivery methods being used and further breakdown of the individual cell and viral vector types. The most widely used delivery method is tissue specific/stem progenitor cells with 36%, which is due to the diversity of projects that utilise these types of cells. These include studies into ophthalmology, neurological disorders and gastroenterological disorders amongst others. The second most widespread delivery method is MSCs/Stromal cells, which are most commonly used to develop therapies targeting cardiovascular and orthopaedic conditions. Approximately 11% of the preclinical programmes identified for this study use in vivo gene therapies are their utility includes study into cardiovascular disease, cancer, haemophilia and conditions of the eye. Pluripotent stem cells were found to comprise around 13% of the therapies studied and are similarly diverse with respect to the disease area to which they are applied. However, their use is more frequently investigated in early-stage translational research.
The heatmap below (Figure 3) provides details of the various bodies funding preclinical advanced therapy research in UK and the disease areas that these organisations are targeting. The identified preclinical projects are supported by a wide range of funding organisations and from private investment. With regards to the number of projects funded, the Medical Research Council (MRC) is the most prolific funding body providing grants to nearly 140 projects including both preclinical studies and earlier translational research work (Figure 4).
The majority of late stage projects currently receive funding through EU grants. However, the following organisations also support late stage projects: Innovate UK, The MRC, The British Heart Foundation and the Wellcome Trust. Figure 4 gives a further breakdown of the various streams funding preclinical research in the UK’s advanced therapy industry by providing the number of studies funded by each organisation.
Figure 5 illustrates the usage of gene modification techniques for both ex-vivo and in vivo gene therapy applications. In recent years, the number of studies utilising gene modification techniques has increased owing to improved understanding of these methods, and this trend will likely continue into the future. Although the majority of projects detailed in this study did not use gene modification techniques, we identified 77 projects in which of gene modification was utilised. However, it is probable that the actual figure is considerably higher than reported here and this discrepancy is explained by the difficulty in capturing this type of project information through the methodology used. On the whole, lentiviral gene modification is the predominant technique with 26 studies reported. There were 13 reported instances of retroviral modification and, interestingly, 19 reported cases of CRISPR-Cas 9 usage. However, it should be noted that the bulk of cases where CRISPR-Cas 9 was being used were in projects in the early stages of development.
Generation of heat maps from collated data
In order to provide a broad description of the diversity of projects that are currently being carried out throughout the UK, a series of heatmaps were generated using the data collated. In each case the therapeutic delivery method was plotted against the disease area in question and each data point within each heat map displayed represented a project specific to that area of work. This method was then used to assess the frequency and types of projects being carried out at a number of commercial organisation and universities throughout the UK on a regional basis. If further information is required concerning specific studies, we can be contacted via our website at email@example.com.