From Research to Clinic: Training the Next Generation of Advanced Therapies Researchers
The Advanced Therapeutics Hub hosted a free 2 half-day training course for academic researchers in gene and cell therapy, enabled by funding from BBSRC. Designed to give researchers a clear, practical understanding of what it takes to move their work from the laboratory into clinical testing and validation, the course was developed in partnership with Cell and Gene Therapy Catapult and Edinburgh Clinical Research Facility.
The importance of translational training
Equipping researchers with the skills to translate their research to the clinic is central to the Hub's mission. Many researchers in this field, particularly research staff and PhD students, haven't yet had direct exposure to what happens once work leaves the lab. Understanding that pathway early can make a real difference to research design.
With funding from BBSRC, the Hub was able to make the course free for all attendees and open it up to the wider advanced therapies research community. Around 40 delegates attended, not only from the Hub's partner centres, but from external organisations including MRC/BHF CoRE in Advanced Cardiac Therapies and EuroGCT. Attendees represented every stage of the career pipeline, from MSc students to research group leaders.
The preclinical pathway
Before any new therapy can be tested in people, it must go through the preclinical stage: developing, testing and refining a treatment in the lab and in models such as cells or animals, to build the safety and manufacturing evidence needed before a clinical trial can even be considered.
The first half-day focused on early research positioning for successful outcomes, covering regulatory pathways and the key aspects of advanced therapy product development and manufacturing.
A recurring theme was the value of working backwards from the final therapy, since early decisions shape how smoothly a project progresses later. Building in quality control from the start proved similarly important, as did a practical reminder: GMP work always takes longer than expected, so timelines need generous margin. Attendees also learned that free early-stage regulatory advice is available and worth seeking, as ATMP classification is rarely straightforward. Criteria differ between countries and are applied case by case, so few therapies follow the same regulatory path.
Delegates also had the chance to join an optional GMP-like facility tour hosted by CGT Catapult, followed by a networking reception to close out the day.
The clinical landscape
The clinical landscape covers everything that happens once a therapy moves on to testing in people. The second half-day introduced health economics, the importance of early patient and public involvement, and the fundamentals of planning and running clinical trials, along with their regulatory context and key players.
Delegates were introduced to the QALY, or quality-adjusted life year, and to the case for weighing up a therapy's health economic value before it reaches trial. The session also highlighted that meaningful patient and public engagement does not have to wait until a project is clinically mature, but can begin at a much earlier academic stage. On the regulatory side, attendees gained a clearer sense of how tightly clinical trials are governed, underpinned by Good Clinical Practice, the code that defines the standards trials must meet.
Translating research into the clinic cannot be done alone; it requires sustained effort across a wide range of people and professions. Researchers taking their first steps down this path will find no shortage of expertise on hand to support them.
The Hub extends its thanks to our attendees for joining us, our partners at CGT Catapult and ECRF for delivery of the content on behalf of the Hub, all speakers for sharing their expertise, and BBSRC for making this course possible.
