Boston Children’s and Broad Institute partner on new center for rare disease treatment

Key Points:

• The Broad Institute, Boston Children’s Hospital, and Maine’s Jackson Laboratory have launched the non-profit Center for Therapeutic Genetics to develop affordable and accessible gene therapies for rare diseases, aiming to standardize personalized treatments like clinical procedures.
• The center will focus initially on gene-editing treatments for rare genetic epilepsies in children, supported by a federal ARPA-H grant, and plans to create shared treatment platforms, including disease models and clinical programs, to facilitate broader replication.
• With over 10,000 rare diseases affecting 1 in 10 Americans, mostly children, and less than 5% having approved treatments, the center seeks to overcome challenges in investment and access by raising philanthropic funds and advancing scalable therapies.
• The initiative aims to shift gene therapy regulation towards a model similar to surgical procedures, enabling clinicians to use approved resources without separate regulatory approvals for each treatment application.
• Experts emphasize that while scientific advances are rapid, building systems for safe, efficient, and scalable patient access requires collaboration across institutions, highlighting the complexity of expanding treatment for rare diseases.