New center is established to take on rare diseases

Quite a setting for research! The Jackson Laboratory campus in Bar Harbor, Maine

— Jackson Laboratory photo

Edited from a report by The New England Council

“Augusta, Maine-based Jackson Laboratory and Boston Children’s Hospital (BCH) have joined Cambridge, Mass.-based Broad Institute in launching the Center for Therapeutic Genetics (CTG), a collaboration focused on developing precision genetic medicines for patients with rare and ultra-rare diseases. The center aims to make patient-tailored treatments more precise, reliable, and repeatable by sharing methods, data, and training.

“An estimated 350 million-400 million people worldwide live with one of about 8,000 rare diseases, yet fewer one than in 20 has an approved treatment. The center will apply tools, models, processes, data, and protocols developed for one treatment program to others. Jackson Laboratory contributes expertise in rare-disease models and translational science, while BCH contributes expertise in individualized genetic therapies and the clinical implementation of genomic medicine.

“‘We’re at a moment in genomic medicine where, for many rare diseases, the question is no longer whether we can treat them, but whether we will build the systems to do it,’ said Cat Lutz, vice president of the Rare Disease Translational Center at Jackson Laboratory. Timothy Yu, a physician in the Division of Genetics and Genomics at BCH, said, ‘We used to see precisely tailored therapies for children with genetic diseases as remarkable exceptions. CTG is built on the conviction that they don’t have to be.”’