What are the main bottlenecks for advanced therapies for reaching patients?
What are the main challenges to develop repurposed drugs?
Why is the Natural History so important in rare diseases?
How do I develop my biomarker strategy?
How do I choose the right disease model?
How do I create the formulation and scalability of a lead compound?
How do I optimize a lead compound?
How do I validate a small molecule lead compound?
What are the main mechanisms and tools to search a hit compound?
How do I identify and validate the right disease target?
The toolbox has been developed through funding from the EJP RD project.
The European Joint Programme on Rare Diseases is an initiative that has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement N°825575