New Center for Therapeutic Genetics to Develop Rare Disease Genetic Medicines

The new center will focus on developing precision genetic medicines through collaboration between the Broad Institute, Boston Children’s Hospital, and The Jackson Laboratory

It’s estimated that nearly 400 million people across the globe live with a rare disease. Though many of these diseases are progressive, debilitating, and even life-threatening, less than five percent of them have an effective and approved treatment.

Nearly half of the rare disease patients are children and it’s estimated that 30% of children with a rare disease will not survive past their fifth birthday. Though there have been some major successes in developing and providing treatment for some children with rare disease, including Baby KJ Muldoon —who was the first patient to receive a personalized CRISPR gene editing therapy—N = 1 therapies are challenging on many levels, and these stories cannot be the functional standard practice for treating patients with rare disease.