Gene Therapies Bring New Hope to Rare Disease Patients

One of the most promising areas for rare disease treatments is gene therapy, part of the emerging area of regenerative medicine. Though long considered a therapy of the future, a number of therapeutics with the potential to treat rare diseases are advancing through the clinic. We spoke to Karen Kozarsky, managing partner at Vector Partners, ahead of the the Alliance for Regenerative Medicine’s January 27 roundtable on rare diseases. Kozarsky discussed the promise gene therapy holds for rare diseases, the challenges in developing these therapies, and the progress being made.