Denmark and the EU should strengthen efforts in AI and ATMPs
Despite the threat of tariffs, increased competitive pressure, a significant innovation gap, and uncertainty regarding future medicine prices, there are many positive prospects for Danish and European biotech and pharmaceutical companies.
There are several areas where the sector can achieve new and groundbreaking results that could lead to innovative medicines benefiting patients, society, and investors.
In particular, it is worth paying attention to how AI and ATMPs can contribute to growth and whether there is both the capability and willingness to make the necessary investments and regulatory adjustments to ride the wave.
How can AI be best utilized?
AI is not the solution to everything, but there are high expectations that AI will be a game-changer in optimizing and accelerating pharmaceutical research and development. The question is how to best harness the benefits of AI and who will gain the most from it.
At the beginning of the year, the FDA released a draft guidance discussing the use of AI models, focusing on generating information and data to support regulatory decision-making regarding drug safety, efficacy, and/or quality.
Examples of relevant AI applications mentioned in the draft guidance include reducing animal testing, predictive models for optimizing clinical trial design, and the ability to process and analyze various types of data to achieve a better understanding of disease presentation, heterogeneity, and prognosis.
However, challenges related to AI are also highlighted, including the importance of ensuring that the data used in AI model development is relevant, accurate, complete, and traceable.
Additionally, methodological transparency regarding AI methods (e.g., how they are developed and how they arrive at conclusions) is necessary, as is addressing the risk of data drift when new data inputs do not align with the data on which AI models were originally trained.
Limited discussion on AI’s potential for new medicines
The FDA’s guidance document does not discuss the use of AI models for discovering new drugs, such as identifying new therapeutic targets, mechanisms of action, or for screening, designing, and optimizing potential drug candidates. However, this is precisely one of the areas where revolutionary breakthroughs could occur.
Combining traditional R&D methods with AI can enhance the identification of new biological targets, while improving the understanding and prediction of molecular interactions and off-target effects. This could lead to new drug candidates with alternative or improved mechanisms of action or even make previously undruggable targets accessible for molecules with novel structures.
Furthermore, AI can contribute to improved operational development efficiency, including better resource utilization, optimization of internal workflows, and preparation of regulatory submission documents.
The EMA has so far released a reflection paper on the use of AI throughout the lifecycle of pharmaceutical products, and the Draghi report recommends implementing clear and timely guidance by 2027, including the analysis of clinical data.
Promising prospects in ATMPs
Another area of great expectations is Advanced Therapy Medicinal Products (ATMPs), which include gene therapies and biological medicines based on modified or manipulated cells and/or tissues.
A year ago, the first CRISPR-based drug (Casgevy) was approved in the EU, and many CRISPR-based drug candidates are either in or approaching clinical development, including for major indications such as cardiovascular/metabolic diseases and cancer.
A promising prospect is the potential to treat neurodegenerative diseases such as Parkinson’s with gene therapy. A prerequisite for targeting the central nervous system is the ability to cross the blood-brain barrier, which has been a limiting factor. However, with new delivery mechanisms—both viral and non-viral—it may soon be possible to reach previously inaccessible targets in the brain using ATMPs.
It would be fantastic if Denmark took the lead
One type of ATMP that has already yielded groundbreaking results is chimeric antigen receptor T-cells (CAR-T). CAR-T therapy, where a person’s T-cells are genetically reprogrammed to trigger a potent immune response against tumor cells, has primarily been used to treat hematologic cancers such as certain leukemias, lymphomas, and myeloma. However, progress has also been made in developing treatments for solid tumors using CRISPR technology. Additionally, CAR-T has the potential to treat autoimmune diseases by targeting autoreactive B-cells.
With the potential to treat solid tumors, as well as neurodegenerative, cardiovascular/metabolic, and autoimmune diseases, ATMPs present an obvious focus area for new investments.
ATMP medicines are also highlighted in the Draghi report, which suggests increasing focus on R&D investments to support the establishment of several “world-class innovation hubs” in this field.
How fantastic would it be if Denmark took the lead and worked more strategically toward becoming a leading ATMP innovation hub? And perhaps, by combining AI technology with ATMP development, we could reach new heights in a Danish life science success story.