Broad Institute, partners unveil initiative to develop rare disease gene therapies

Matthew Orr/STAT By Allyson Chiu — Boston Globe

The Broad Institute, Boston Children’s Hospital, and Maine’s Jackson Laboratory announced a collaboration Tuesday to develop gene therapies for people with rare diseases and to make the life-saving therapies cheaper and more accessible to patients.

With relatively few gene therapies on the market, families of children with rare genetic disorders have few options but to partner individually with researchers and raise millions of dollars to develop treatments for their conditions.

The newly formed non-profit Center for Therapeutic Genetics will create precision medicines and share those methods, data, and training with other clinicians so they can do the same. The goal is to make these personalized treatments more like clinical procedures, such as organ transplants, so doctors don’t have to seek separate regulatory approvals for each use, said Winston Yan, founding director of the new center.