Gene therapy holds immense promise for treating a variety of conditions, but high costs and complex manufacturing processes have limited its accessibility. Lentitek, a pioneering biotech startup, sought to address these challenges by improving vector production, a critical component for gene therapy delivery. UCL, home to cutting-edge expertise in biomedical research and strong ties to the biotech industry, was uniquely positioned to support this endeavour.
By collaborating with UCL’s Professor John Counsell and his research team, Lentitek aimed to validate its innovative technology – a crucial step towards making gene therapy more efficient, cost-effective and accessible to patients.
A synergistic relationship
Among several researchers and groups at UCL focused on the next generation of gene therapies is Professor Counsell’s team in the Department of Targeted Intervention – part of the UCL Division of Surgery. His group has close ties with the biotech industry and hospital clinicians, particularly at UCL Great Ormond Street Institute of Child Health, where Professor Counsell undertook his postdoctoral training.
A few years ago, Professor Counsell met Dr Adam Inche, a biotech entrepreneur, who in 2021 formed a new venture in the gene therapy space called Lentitek. The startup focuses on a key part of the manufacturing process for gene therapies, namely the ‘vectors’ ‘that are used to deliver genetic cargo into human T-cells (as well as other cell types) to reprogram them for therapeutic effect. Vectors are essentially repurposed viruses that have been rendered safe and unable to replicate. Lentitek is developing technology to improve the production quality of these vectors, with several potential benefits.
Collaborating with Professor Counsell, the company leveraged the world-class expertise of his research group and the UCL ecosystem, to rigorously assess the benefits of its technology. Through the validation project, which was facilitated by UCL Consultants (UCLC), Professor Counsell’s team delivered several work packages for Lentitek, focusing on key metrics such as vector yield and control of transgene expression. The project conclusively demonstrated the enhanced ability of Lentitek’s vector technology to efficiently deliver complex genetic payloads, including CAR-T cancer immunotherapy (see box, ‘What is CAR-T’).
“The team at UCL have not only allowed Lentitek to build out our technology, they have enabled us to spin up our internal R&D and commercial activities in a very capital efficient manner,” said Dr Inche, Founder and CEO at Lentitek.
The team at UCL have not only allowed Lentitek to build out our technology, they have enabled us to spin up our internal R&D and commercial activities in a very capital efficient manner
A potential game-changer for CAR-T therapy
Coming at a crucial time in the genesis of Lentitek, the project helped to validate their core technology – an essential step for any future licensing or partnership deals, which are the bedrock of the industry. The work also helped the company to identify new applications that they could develop based on their technology platform, and also supported investment including a recent £1 million investment round.
Perhaps most promisingly, Lentitek’s technology enables precise customisation of the surface of the vector, by ensuring only specific targeting proteins ‘decorate’ the surface that can home in on targeted human cell types to deliver the genetic payload. That homing capability has great potential in CAR-T cancer immunotherapy. Indeed, the tantalising possibility now exists for CAR-T therapy to be delivered via a direct route of administration, termed as “in vivo CAR-T ” – a ‘game changer’ for both health systems and patients, reducing hospital visits and overall costs.
The relationship has continued over a number of work packages, with the Lentitek team growing to include three researchers working alongside Professor Counsell at his lab, with Lentitek continuing to engage with UCL, UCL Consultants and UCL’s commercialisation arm UCL Business.
Following on from the consultancy project, Professor Counsell’s UCL team and Lentitek have secured a grant from Innovate UK to further investigate the potential of this more direct – or in vivo – CAR-T therapy.
Reflections on the collaboration
Reflecting on the consultancy, Professor Counsell commented: “I think this project would have been almost impossible to do independently, through the university system, without UCLC being there. It’s really reduced the complexity in doing this sort of thing. With UCLC, they operate like an agency, handling contracts and just smoothing out the process. They’ve been brilliant.
“I think it also elevates the external standing of academics, showing they can be skilled partners in building tech companies and driving commercial success, as well as advancing science.”
What is CAR-T?
Over the past decade, UCL has become a world-class hub for the research and development of new gene and cell therapies. Some therapies originating from UCL research, for example an innovative type of CAR T-cell therapy, have been approved and are set to benefit patients in the clinic.
CAR T-cell therapy is a complex treatment that involves collecting immune T-cells from a patient’s blood, then genetically re-configuring them in the lab so that they are more effective at recognising and attacking cancer cells. These therapeutic CAR-T cells are then put back into the patient’s bloodstream via a drip, where they can take effect.
The treatment has been refined and fine-tuned over time, following numerous clinical trials, and has been shown to be safe and effective for patients. However, it is still a relatively new approach and there is potential for further improvements, particularly in terms of the cost and complexity of the procedure.