Tagged with Gene Therapy

Europe In an extensive recent PharmaBoardroom conversation, EMA Head of Advanced Therapies Ana-Hidalgo-Simon highlighted some of the most important trends around advanced therapy regulation in Europe. Hidalgo-Simon touched on why moderate growth in regulatory applications is a positive development for the EMA, how patient insights are being better incorporated into the…

Europe Speaking exclusively to PharmaBoardroom, the EMA’s Head of Advanced Therapies Ana Hidalgo-Simon discusses steadily growing numbers of regulatory applications in the advanced therapy field, the vital importance of patient group insights, RWE collection and curation, hospital exemptions, manufacturing challenges, and more.   [Advanced] therapies are complex, difficult, and have a…

Spain Dr Manel Juan is part of the team behind the development of the first European CAR-T-cell therapy to treat multiple myeloma, the second most common type of blood cancer. As head of immunology at the Hospital Clinic Barcelona, he walks us through his personal journey with the project, the challenges…

Italy Gilead Sciences’ vice president and general manager for Italy, Valentino Confalone, outlines the company’s tumultuous pandemic journey in one of the first global epicentres; the role that Italy played in the early studies of remdesivir as a treatment against severe COVID-19 disease; how Gilead is working to eliminate viral Hepatitis…

Global As complex cutting-edge therapies come online, industry sponsors are increasingly looking to engage with patients, caregivers, and advocacy groups earlier and more broadly in the drug development process to better understand their needs. However, as the below insights from four of Novartis Oncology’s patient engagement leads show, successfully integrating patient…

Global One of the enduring conundrums around ground-breaking and potentially paradigm-shifting treatments such as CAR-T is the ever-thorny question of how fast to move them to market when the promise seems so great, but the evidence remains so patchy.   So far, top tier regulators such as the US FDA and…

Global Chimeric antigen receptor (CAR-T) therapy was first approved by the US FDA back in 2017 amid a great deal of fanfare and high expectations among patients about its potential as a revolutionary, perhaps even decisive, new pillar in the war on cancer. Five years later, however, many are still left…

Global In recent years, the much-discussed concept of “patient centricity” has been top-of-mind across all corners of the pharma industry. This is especially pertinent now that many innovative drug makers are seeking to become much more than just purveyors of pills and expand along the life science continuum, providing positive healthcare…

Global David H. Crean, Managing General Partner for Coast BioVentures, updates and highlights the latest trends in the cell and gene therapy market for deal activities and financings over the past two years since his original article on the topic was published on PharmaBoardroom in June 2019.   Cell and gene…

Saudi Arabia Amr Seif introduces the evolution in Astellas’ MEA strategy; the adoption of innovative access models to ensure that the companies’ therapies are available for all patients who need them; and the challenges and opportunities in the Saudi pharma market.   We are opening a new chapter in medicine. The early…

Global Marc Boutin, Novartis’ global head of patient engagement, outlines the company’s “bold vision” of patient engagement and the actions that need to be taken for CAR-T patients in the immediate, short- medium-, and long-term. This piece forms part of our special section, InFocus: Patient Engagaement in the Era of CAR-T.…

Europe Building on points developed in his 2020 PharmaBoardroom interview, Professor Nicolaus Kröger of the European Society for Blood and Marrow Transplantation (EBMT) outlines the progress made by the multi-stakeholder GoCART Coalition towards greater harmonisation of cell and gene therapy research in Europe, the challenges of integrating patient-reported outcomes into its…