Scientific interest in rare disease drug discovery has been growing exponentially and the number of new therapies targeting rare and orphan diseases has doubled in the past 6 years. In this white paper, we have reviewed innovative approaches to develop successful therapies for rare diseases such as gene replacement therapies, antisense oligos and monoclonal antibodies. We also highlight some of the new classes of therapies including the epigenome and microbiome.

If you're working to develop new personalized treatments for rare diseases, tell us how we can support your program.