Leading the Way With Gene Therapy in Rare Disease

Leading the Way With Gene Therapy in Rare Disease

We are at the cutting edge of the science that has the potential to treat the more than 80 percent of rare diseases that have a known genetic component. Through collaborations, partnerships and acquisitions, we have investigational gene therapy treatments in various stages of development for hemophilia A, hemophilia B, amyotrophic lateral sclerosis (ALS) and Duchenne muscular dystrophy (DMD).

Additionally, we continue to engage the community on gene therapy, sponsoring sessions at key patient, policy, medical and health care congresses. We also hosted a multi-stakeholder summit on the value of rare disease treatments and have engaged key opinion leaders on reimbursement and access.