Pause, recalibrate, execute: Advancing ATMPs in an Era of Uncertainty
Tracy Humphries, Dir. Nucleic acids & Nanomedicine Marketing, Cytiva.
Over the last thirty years, we’ve seen incredible advances in genomics, which have enabled deeper insights into diseases. Genomic sequencing has revealed the blueprint of most pathogens, and this has enabled drug manufacturers to take a genome guided approach to developing novel therapeutics. Advances in molecular biology, genetics and genomics have enabled much of the human genome to be druggable. The therapeutic landscape has been changing and for the better, especially for patients!
Many drug developers and manufacturers are beginning to recognize the benefits of adopting a multimodal approach to next generation modalities or Advanced Therapy Medicinal Products (ATMPs). This multimodal toolbox can include cell and gene therapies (CGT), genomic medicines, biologics such as antibodies and other small molecules. By moving the emphasis to the identifying the right target, they are then able to select from a range of appropriate therapeutic approaches, alone or in combination, to provide better options for patients.
Whilst this rapidly evolving field offers many benefits for patients, it presents challenges for drug developers and manufacturers, as they navigate the risks of bringing scientific innovation through to clinical products. In addition to the need for significant innovations and investments to streamline development and scaling of such therapies, manufacturers are now facing a pressure cooker situation of unknowns due to policy shifts, leadership and company restructures, regulatory uncertainty and navigating the new AI landscape.
In 2025, there has been scrutiny and reductions into federal funding in the US and an increase in skepticism and misinformation for ATMPs. Collaboration within biotech is key for bringing new therapeutics to market. The cumulative effective of conferences, conversations, lectures and advice, could make a massive difference on a drug being commercialized successfully. A large majority of top-selling drugs were not discovered in-house, but originated elsewhere like academic licensing or acquisitions, therefore the impacts we are seeing on academic and scientific funding, are quite concerning.
Next generation modalities such as cell therapies and genomic medicines (often referred to as genetic therapies) are still increasing in presence though, in the early clinical stages of therapeutic development. As of June 2025, there are 45 cellular and gene therapy products approved by the FDA. Genetic therapies are transformative as they can be designed and formulated to silence, express, and edit specific genes, providing a flexible and powerful approach to preventing and treating diseases. More simply, where traditional therapies have failed, genetic therapies may be able to succeed. This approach is particularly suited to the treatment of cancer, as cancer cells continually change their genetic makeup to survive, and cancer differs from person to person.
It's crucial to try and avoid negative impact in early-stage drug discovery. There is still investment into next generation therapeutics, with many organizations using this a recalibration moment, looking at their pipeline strategies and announcing mergers and acquisitions where appropriate. To balance the reduced funding into academia, there may be other companies stepping up to fill the gap, such as venture capitalists, emerging biotech companies and funding from larger organizations into early-stage startups.
Now more than ever, manufacturers of next generation therapeutics are pulling back to the execution basics:
Collaboration and funding in early-stage development – most breakthroughs occur in academia; funding innovation is crucial to the field
Unification of large organizations and key voices – coordination to help provide better education, combat misinformation and to gain regulatory clarity
Accelerate manufacturing excellence – ATMPs need scalable, repeatable and cost-effective technologies
In my lifetime, I have seen some incredible cures come to market and I hope that I will see many more!
Tracy Humphries
Director, Nucleic acids and Nanomedicine Marketing,
Cytiva, now including Precision NanoSystems