Accelerating Cell and Gene Therapy Development

Many cell and gene therapies fail to reach patients despite promising research. Now, researchers and clinicians in Sweden have developed a new cooperation model to shorten lead times and reduce costs, aiming to provide more patients access to advanced treatments.

Cell and gene therapies have shown promising results in areas from cancer to neurological diseases. However, the path from academic breakthrough to healthcare implementation remains long, with many projects lost in the translational gap. “As academic researchers it is totally reasonable to devote all your energy to getting through to the first clinical study. But this is not enough if the objective is for the therapy to actually reach patients. Somewhere along the line, the development must also be attractive for healthcare, investors and industry—and we must think about this much earlier than we do today,” says Anna Falk, senior author of the research article published in Molecular Therapy, Methods and Clinical Development.

In the paper, Falk and colleagues from Lund University and Skåne University Hospital present a working method to break down silos between academia, healthcare, and innovation systems. “At present, crucial technical, regulatory or commercial obstacles are often detected too late. And then it’s an expensive and slow process to make corrections—or the therapy never reaches the patients. We present a model and a tool that can provide support and reminders about which parts you need to have in place when you develop new advanced cell and gene therapies. It’s development support that can be used by all those working in the field,” adds Falk.

The model emphasizes parallel and integrated work among university, hospital, and innovation stakeholders from the start, rather than sequential steps. Hospitals participate early in development and production, recognizing their role in both using and producing cell and gene therapies. Conventional drug development structures do not suit these advanced therapies. “It’s a paradigm shift in which the hospital not only receives or tests a finished product, but also all those involved—academia, hospital, and innovation stakeholders—need in different ways to participate throughout the development process, manufacturing, and quality control. There is a need here for a new working methods and tools. Together they constitute a model that has been developed in Lund,” explains co-author Stefan Jovinge.

A key component is the Cell and Gene Therapy Navigator, a practical tool that tracks technical, clinical and commercial maturity simultaneously. This approach identifies imbalances and bottlenecks early. “It’s not enough to be the first to test the therapy on humans. If we want the therapies to reach many patients we must think about production, regulations, and reimbursement right from the start. The Navigator works as a joint checklist and mirror for the project,” according to first author Gisela Helenius.