Optimalisatie en onderzoekverbeteringen voor cel- en gentherapie ontwikkeling
CORE-ATMP: Enhancing Research and Development for Cell and Gene Therapy
The CORE-ATMP project unites key institutions—Leiden University Medical Center (LUMC), the Centre for Human Drug Research (CHDR), the Central Committee on Research Involving Human Subjects (CCMO), and the Dutch Medicines Evaluation Board (CBG)—in a public-private partnership dedicated to advancing the development of cell and gene therapies with the aim to streamline the transition from the laboratory to clinical trials. These innovative therapies promise effective treatments for severe genetic diseases and cancer by targeting the conditions at their source.
Cell and gene therapies represent transformative advancements in treating serious health conditions, yet their development faces challenges, such as complex safety and efficacy requirements. The societal and economic impacts of genetic diseases and cancer are profound, with millions affected worldwide. Innovation in therapy development is needed to provide developers with clear methodologies and patients with timely, effective treatments.
Addressing these challenges, CORE-ATMP is pioneering advanced methodologies to ensure a robust and predictable transition into clinical trials. CORE-ATMP deliverables include validated methodologies for potency assays and non-clinical data evaluation supporting a well-considered and reliable approach to first-in-human application. These resources are being made freely available to researchers, regulatory bodies, and developers, ensuring widespread access to the latest advancements in therapy development. This will foster broad adoption and implementation. These tools are expected to reduce development timelines through harmonization and improved treatment reliability, and ultimately, enhance patient access to safe and effective therapies. Through this collaborative effort, CORE-ATMP exemplifies how partnerships can bridge gaps between research and clinical application, leading to significant health advancements.
