Broad Institute, Boston Children's Hospital, and Jackson Laboratory Launch $160 Million Center for Rare Disease Gene Therapies

The Broad Institute, Boston Children's Hospital, and The Jackson Laboratory have launched the Center for Therapeutic Genetics, a nonprofit collaboration aimed at transforming rare disease treatments into repeatable clinical procedures. Led by Winston Yan, the center is backed by a $34.5 million ARPA-H grant to develop custom gene-editing therapies for pediatric epilepsies. By openly sharing its tools and protocols, the center hopes to make personalized genetic medicine scalable and affordable for the millions of rare disease patients who currently lack approved treatments.

Center for Therapeutic Genetics launch

• ▪The Center for Therapeutic Genetics is in its founding phase, is not accepting patient referrals, and aims to treat its first patient within two to three years.
• ▪The Center for Therapeutic Genetics is led by founding director Winston Yan, alongside co-founders David Liu, Wendy Chung, Timothy Yu, and Cat Lutz.
• ▪The Broad Institute, Boston Children's Hospital, and The Jackson Laboratory launched the Center for Therapeutic Genetics, a nonprofit center to develop repeatable gene therapies for rare diseases.

Rare pediatric epilepsy treatments

• ▪The ARPA-H THRIVE initiative is a federal program that will provide up to $160 million in funding for teams developing custom gene-editing treatments for rare diseases.
• ▪The Center for Therapeutic Genetics' initial epilepsy research will focus on Dravet syndrome caused by SCN1A mutations and alternating hemiplegia of childhood caused by an ATP1A3 mutation.
• ▪The Center for Therapeutic Genetics received a $34.5 million federal award from the ARPA-H THRIVE program to develop gene-editing treatments for children with rare genetic epilepsies.

FDA rare disease pathways

• ▪The Food and Drug Administration established a plausible mechanism pathway allowing rare-disease drugmakers to leverage data on similar components from prior successful applications.
• ▪The Food and Drug Administration issued draft guidance on June 2, 2026, aimed at streamlining gene therapy development for rare diseases.

Open-access protocol sharing

• ▪The Center for Therapeutic Genetics aims to work with regulators to establish standards that allow individualized genetic treatments to function as repeatable clinical procedures rather than unique drugs.
• ▪The Center for Therapeutic Genetics plans to openly share its design tools, disease models, safety data, manufacturing processes, and clinical protocols to enable other institutions to replicate its treatments.

Patient advocacy in genomics

• ▪The National Organization for Rare Disorders operates a network of nearly 50 medical and research institutions in the United States dedicated to rare disease care and research.
• ▪An estimated one in 10 Americans lives with a rare disease, and fewer than 5 percent of these conditions have an approved treatment.