From Lab to Patient: Turning Cell and Gene Therapy into Real Treatments
Knut Steffensen is taking on the ambitious task of steering cell- and gene-based therapies from the research bench into routine patient care at Karolinska. As head of the university’s ATMP-Centre, he faces the challenge of coordinating the many moving parts required to translate discovery into approved therapies. His message is clear: the center should act as a virtual hub where research, manufacturing, and clinical practice come together under one roof to speed up development while maintaining rigorous standards.
ATMP-Centre’s mission revolves around two central duties. First, to counteract “silo thinking” in academia, industry, and healthcare; second, to identify bottlenecks in the value chain that delay bringing promising therapies to patients. Translating research into clinical trials is extraordinarily complex and demands meticulous planning, cross-disciplinary collaboration, and well-structured processes. Steffensen’s aim is to open doors along the way—establishing frameworks that streamline collaboration and reduce friction between laboratories, trial sites, and manufacturing facilities.
A Virtual Hub for a Cohesive Industry
Karolinska already possesses substantial capabilities to support ATMP development, including a robust clinical trial unit and Vecura, the in-house manufacturing facility responsible for producing medicines under strict quality controls. By positioning the ATMP-Centre as a connective tissue between these functions, Steffensen envisions a streamlined path from discovery to dosing, with decisions made more quickly and more coherently across stakeholders. The centre’s design is intentionally cross-functional: researchers, clinicians, and industry partners should meet in a shared ecosystem where insights, resources, and risks are openly discussed and managed.
Yet funding remains a critical hurdle. Both manufacturing and clinical trials require substantial investment. The centre’s strategic aim is to become a primary contact point for the pharmaceutical industry, fostering co-financed projects and long-term partnerships. At the same time, the center is building internal structures that researchers can leverage when applying for research grants, ensuring that funding opportunities are operationally practical and aligned with clinical needs.
Beyond Borders: A Scientist-Industrialist Mindset
Steffensen brings a rare blend of experience from both academia and industry. He notes that researchers are adept at pursuing independent, curiosity-driven projects, while industry tends to focus on teamwork and rapid resolution of business-critical questions. By combining these perspectives, he believes the path from basic science to patient care can be accelerated without compromising scientific rigor or patient safety. His goal is to prepare both the hospital system and policymakers for upcoming therapies, including the need for more beds or facilities to accommodate expanding patient access as these treatments become available.
Looking back over a career that has tracked the evolution of cell- and gene therapies—once dismissed by some as science fiction—Steffensen sees a practical frontier: many projects at Karolinska could reach clinical trials within five years. If even a small percentage of the current research pipeline advances, the impact could be transformative for patients with previously intractable diseases. That potential underpins the centre’s ambition: to become a catalyst for real-world therapies that improve outcomes and quality of life.
Positioning Europe as a Leader in ATMP
Today, Karolinska ATMP-Centre stands as one of Europe’s leading research environments in the ATMP field. But Steffensen stresses that leadership is not a finish line. The global landscape is increasingly competitive, and sustained progress requires ongoing investments and strategic collaboration across academia, healthcare systems, and industry. He emphasizes a proactive approach: continue to build partnerships, create scalable processes, and continuously refine the interfaces that connect discovery, production, and clinical care.
Ultimately, the effort is about people—the patients who stand to gain from faster, safer access to innovative therapies. If the centre’s models prove successful, more Karolinska projects could move from bench to bedside, unlocking new treatments for a range of difficult diseases. As Steffensen puts it, the success of ATMP-therapy development hinges on turning scientific insight into practical, patient-ready solutions.
Roadmap Ahead: What to Expect in the Coming Years
In the near term, the centre will continue to refine its governance, funding structures, and collaboration models while advancing selected projects toward early clinical trials. The aim is not only to accelerate timelines but also to ensure robust safety, scalable production, and clear pathways for regulatory approval. For Karolinska, the promise is simple: by acting as a catalytic hub that unites researchers, clinicians, and industry, they can unlock the full potential of cell- and gene-based therapies for patients—today and tomorrow.
Text: Karin Tideström
