Collaborative drug discovery expedites time to market for rare disease therapies. Alexion Pharmaceuticals (CT, USA) partnered with Charles River (MA, USA), an early-stage CRO, to perform drug development and safety studies on their potential therapeutic for hypophosphatasia. The collaboration helped to move the drug Strensiq® into clinical trials.
Watch the full video here.
Stakeholders in academia, industry and CROs are committed to building a complete platform to develop therapies for rare diseases and they are using their combined expertise and resources to get there.
“There is a continual push amongst people who work in rare diseases to bring [treatments] forward quicker,” notes Martin Mackay, the former Head of Research and Development at Alexion Pharmaceuticals.
The unique capabilities of CROs, like Charles River, allow the design of customized rare disease studies using pre-existing methods, considerably increasing the efficiency and effectiveness of novel therapeutic development.
Thousands of rare diseases still lack novel therapies. But the creation of a robust collaborative ecosystem will ultimately facilitate efficient drug development for the patients who desperately need it.