Cell and gene therapies open new horizons that can change the lives of people living with serious and rare diseases. That’s why CSL Behring created an audio series – The New Horizon Talks – featuring leading researchers and advocates for patients.
The first episode is launching alongside this week’s European Haemophilia Consortium Conference and as we count down to the 2021 Congress of the European Society of Gene and Cell Therapies, which begins October 19.
In our launch episode, we get a 360-degree introduction from Retina International CEO Avril Daly and Dr. Luigi Naldini, director of the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Science writer and broadcaster Vivienne Parry moderates the series, featuring leading experts, scientists, healthcare professionals, patients and patient representatives.
The conversation in the first episode touches upon the historical, scientific, clinical, policy and regulatory considerations and looks ahead to give perspective on the future of this new chapter in medicine. Cell and gene therapies offer the potential to durably change the course of many severe and debilitating diseases of genetic origin – transforming the lives of thousands of patients who may otherwise have little or no hope of treatment or improved quality of life.
However, various challenges need to be overcome to allow gene therapies and other transformative treatments to be integrated into health systems. CSL Behring sees a need for inclusive dialogue to support patient access to this disruptive innovation and the long-term sustainability of health care systems.
Naldini highlights the uniqueness of gene therapies compared with conventional treatments, and how they are changing the traditional approach to treating diseases.
“Decades ago, the first attempts failed because the idea was there but the tools were not ready, and some people lost confidence in the innovation. Now, we are in a different era,” he said.
Avril Daly, a well-known rare disease patient advocate with EURORDIS, discusses the importance of early diagnosis, which remains a major issue for many rare disease patients, impacting their potential access to innovative therapies.
“There is an exciting future ahead, if the right systems and policy frameworks are put in place to ensure uptake and patient access to these new treatments,” Daly said.
More episodes will debut later in October. Subscribe to the podcast series to get an alert when new episodes are added.
"We are proud to launch this series with distinguished experts and patient advocates and learn from their insights in the field of cell and gene therapies,” said Karen Pinachyan, CSL Behring’s Senior Director Medical Affairs EU. “At CSL Behring, we believe that these advanced therapies have the potential to offer significant benefits to patients suffering from genetic and rare diseases. We are committed to deliver on the promise of this innovation and the new era of medicine it signifies."