Broad Institute and Partners Announce Initiatives to Develop Rare Disease Gene Therapy

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

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

The newly formed nonprofit Center for Therapeutic Genetics will develop precision medicines and share their methods, data and training with other clinicians so they can do the same. The goal is to bring these personalized treatments closer to clinical procedures, such as organ transplants, so that doctors don’t have to seek separate regulatory approval for each use, said Winston Yang, founding director of the new center.

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