Broad, Boston Children's and Jackson Lab Launch $34.5 Million Rare Disease Gene Therapy Center
Updated
Updated · The New York Times · Jul 21
Broad, Boston Children's and Jackson Lab Launch $34.5 Million Rare Disease Gene Therapy Center
3 articles · Updated · The New York Times · Jul 21
Summary
$34.5 million from the federal ARPA-H agency is funding a new nonprofit, the Center for Therapeutic Genetics, launched by Broad Institute, Boston Children's Hospital and Jackson Laboratory.
The center aims to make rare-disease gene therapy reusable by keeping the editing platform the same and changing only the genetic instructions, instead of building a new treatment from scratch for each disorder.
Its first work will target neurological diseases that cause seizures, with researchers hoping to show one brain-cell editing system can be adapted across multiple conditions, including adult disorders such as Huntington's.
The effort targets a market failure in which ultrarare treatments can take years and hundreds of millions of dollars to develop, even though rare diseases affect about 400 million people worldwide and 25 million to 30 million in the United States.
Can one gene-editing platform truly cure thousands of unique diseases, or is this a scientific oversimplification?
With 400 million patients globally, how will the new center ethically decide who gets a cure first?
If a nonprofit can make gene therapy affordable, is the current for-profit model for rare diseases obsolete?
PERC Initiative: $34.5M Federal Investment to Fast-Track Gene Editing Therapies for Dravet Syndrome and AHC
Overview
The Pediatric Epilepsies and Rare CNS (PERC) Gene Editing Platform, launched in July 2026, represents a major step forward in treating severe childhood neurological diseases. Backed by a $34.5 million federal award from ARPA-H’s THRIVE program, PERC brings together leading institutions like The Broad Institute, The Jackson Laboratory, and Boston Children’s Hospital. The initiative’s main goal is to speed up the development of gene-editing therapies, starting with challenging conditions such as Alternating Hemiplegia of Childhood and Dravet syndrome. By focusing on collaboration and innovative technology, PERC aims to deliver new hope to children and families affected by these devastating disorders.