Our partner University College London (UCL) have produced a great short brochure (link) showing the strength and depth of their pipeline of ATMPs. They use a managed portfolio approach in which they support ~100 projects concurrently. Most of the projects are in very early preclinical development, with a reduction seen in the number of projects the further along the clinical translational pathway they progress, with one project that has reached the market (to date). We need to state that UCL’s translational research office (TRO) headed by Jane Kinghorn also manages another large pipeline of classical medications. Here’s their diagram showing how many projects were in their ATMP pipeline as of December 2023:
Most early-stage projects are financed with public money, while later stage ones are commercially funded. The two diagrams below show the split between the public and the commercial parts of their portfolio:
On the publicly financed side, on average UCLs discovery stage project costs about €50k, preclinical project costs about €1M and phase I/II projects about €4,5M. Total investment in the public portfolio for UCL stands at almost €100M.
The investment data on the commercial side is not disclosed, but likely to be significantly higher with funding required for additional company infrastructure as well as that need to increase the scale of manufacture and the scale and number of trials.
There are several implications for CTGCT following this example.
One, we need to understand and market appropriately the strength of The National Institute of Chemistry and its sector D12. D12 Sector for synthetic biology and immunology has an extensive portfolio of existing projects which represents the strength of CTGCT. Head of D12, Prof Dr Roman Jerala has over 100 original scientific articles and over 500 contributions over the course of the last 5 years, here’s the bibliography (link).
Two, we need to build the capability to identify and support promising projects to enter the portfolio – we already do a lot of preclinical work, but don’t yet advance it towards clinical translation. We use our animal models for the discovery and basic scientific research. Our next step will be to advance appropriate translational preclinical work. This step is an imperative and where working in partnerships will be key.
Three, significant funding is required to advance ATMPs along the translational path – the financing of CTGCT via Horizon-Widera funding (Project 101059842) is for building and equiping our GMP facility and for salaries of about 25 employees, with the current infrastructure grant due to expire in 2029. CTGCT needs to raise both public and private financing to maintain sustainability of the centre as well as advance promising therapeutics for our patients.
There are many further implications which we will need to address. Progressing into clinical stages will require developing close collaborations with Slovenian hospitals and their clinical research arms along with fostering the ecosystem, especially in the light of the recent very high investments of pharmaceutical companies into new biosimilar and biotechnology facilities. We are also accelerating our hiring, with new colleagues expected to be on-boarded soon. With that we’re working on creating a dynamic system of measuring excellence that properly addresses both the research and development, as well as translational activities, alongside societal impact. All of these need to be accompanied with developing stronger technology transfer professionals/offices, research project management, and great communication and dissemination e.t.c.
However, the ambition is clear. CTGCT was created with the goal of putting new drugs – gene and cell therapies to treat cancer and rare diseases – on the market. That’s what we’re all in it for, to make a difference to patients.
Thanks to the example of UCL our path is becoming clearer. We have known from inception of the project that we are faced with a dauting and demanding task, but a hugely exciting one and we would love to hear from you if you are interested in partnering for the journey.