The Challenge
Progressing promising academic research to clinical development presents significant challenges. These include difficulties securing funding and navigating complex, evolving regulatory requirements.
Professor Andy Baker, working with University of Glasgow, NHS Greater Glasgow & Clyde and University of Edinburgh research teams, had developed a promising gene therapy programme. To demonstrate its effectiveness in clinical studies, Professor Baker’s team required specialised expertise in non-clinical studies, regulations and health economics to overcome the barriers required to progress this research.
The Collaboration
In 2022, Professor Andy Baker and the Cell and Gene Therapy Catapult (CGT Catapult) began a partnership that would support Baker’s team to initiate a first-in-human clinical trial for a gene therapy aiming to improve the outcomes for specialist heart surgery. The group had realised that the vein grafts used often fail due to the higher pressures associated with blood flow from the heart. They developed a gene therapy that could prolong the long-term functionality by preventing thickening and blockages.
Initially, CGT Catapult provided support to Professor Baker’s team focused on addressing key translational barriers that are commonly encountered when moving academic research into first-in-human clinical development. This included the delivery of a health economic analysis to help optimise the potential target value of the therapy and identifying the key clinical outcomes to inform the clinical trial design.
In parallel, CGT Catapult led interactions with regulatory authorities to determine the product’s classification as a gene therapy, and associated data requirements. This helped Professor Baker’s team to gain clarity on planning for evidence generation required for the programme’s continued development.
These interactions continued through the following years, where in 2023, CGT Catapult worked with Professor Baker to support the authoring of the documentation needed for the first-in-human clinical trial application to the UK Medicines and Healthcare products Regulatory Agency (MHRA). The following year, CGT Catapult provided guidance in responding to MHRA questions on the clinical trial application, which helped secure authorisation to proceed with the trial.
In addition, Professor Baker and his team are pursuing progression of a wider portfolio of gene therapies for cardiovascular disease; for which CGT Catapult recently supported the development of bespoke Target and Quality-Target Product Profiles (TPP/QTPP), and associated action plans, in an opportunity funded by Scottish Enterprise, to help steer development towards desired outcomes.
Where is this now?
In 2026, Professor Baker’s lead study reached a major milestone with the dosing of the first patient in a first-in-human cardiac gene therapy trial in Scotland (PROTECT study). The trial is designed as a safety study, with the primary objective of assessing tolerability and the absence of adverse events following administration of the investigational gene therapy alongside standard coronary bypass graft surgery.
This work with Professor Baker’s group provides a clear example of how academic innovators can strategically leverage the UK’s supportive translational ecosystem to accelerate their research to the clinic and demonstrate the effectiveness of their therapies in a patient population.