Rare disease

Nearly one in 10 people globally are affected by a rare genetic disease. Patients with rare diseases are often severely ill, but they face limited treatment options.

The Broad is committed to leveraging the latest advances in biomedicine to rapidly accelerate the discovery and development of treatments and cures for rare diseases. To fulfill this mission, we are bringing together state-of-the-art technologies, scientific expertise, clinical experience, the biopharma and regulatory community, and patients and advocacy groups.

We are seeking philanthropic partners in the following projects:

• Defining the genomic basis of disease classes, understanding the functions of the key genes that are discovered, and developing novel approaches to therapy.

• Building a diverse community of collaborators empowered to apply scalable technologies to gain new mechanistic insights and propel treatment.

• Enabling clinicians to provide rare disease patients with definitive diagnoses.

• Partnering with patients living with rare diseases to capture essential information in secure data portals that can accelerate new discoveries.
  
  News coverage on our rare disease research