Emma CPM (affiliated with AGEM) is developing innovative therapies for children with rare genetic diseases and unmet medical needs. By integrating clinical expertise, innovative platform technology, and a pioneering valorization strategy, the team is tackling critical barriers to the availability, accessibility, and affordability of groundbreaking gene therapy treatments.
At the Emma Center for Personalized Medicine (Emma CPM), researchers are working to ensure that the promise of innovative therapies, such as using gene editing, reaches the patients who need them most. Spearheaded by Prof dr. Clara van Karnebeek (Department of Pediatrics) and Dr. Daniël Warmerdam (Department of Human Genetics), and colleagues across multiple departments, Emma CPM is developing a therapy platform targeting rare genetic conditions. This includes cutting-edge technologies like CRISPR, tailored to address specific unmet medical needs in children.
Innovative treatments such as gene therapies hold great promise for improving outcomes in patients with unmet medical needs. However, many of these therapies remain out of reach due to the high costs and risks of development, complex regulatory requirements, market failure linked to small patient populations, and unsustainable pricing models. Emma CPM is actively addressing these challenges. By advancing a scalable platform technology, engaging early with regulators and patients, academic-led development and initiating public-private partnerships, the team aims to ensure that scientific innovation results in innovative therapies that are available, accessible, and affordable for the patients who need them most.
These efforts were presented on June 13 to the Amsterdam Valorization Board (AVB), which expressed its support for Emma CPM’s approach. In close collaboration with the AVB and other institutional and external partners, Emma CPM is translating cutting-edge science into sustainable, real-world solutions that can directly benefit patients in need.