A Historic Milestone for the AHC Community: ARPA-H Awards Contract to Advance Gene Editing Toward the Clinic
Five years ago, RARE Hope began an ambitious partnership with David Liu and his team at the Broad Institute with a single goal: develop a gene-editing treatment for Alternating Hemiplegia of Childhood (AHC).
Today, together with the Broad Institute, Boston Children's Hospital, The Jackson Laboratory, and an extraordinary team of collaborators, RARE Hope is proud to announce that the consortium has been awarded a highly competitive contract from the Advanced Research Projects Agency for Health (ARPA-H), under its THRIVE program. THRIVE is led by ARPA-H Program Manager Daria Fedyukina, Ph.D.
Five years ago, our goal of developing a gene-editing treatment for AHC was a moonshot. Today, thanks to this landmark ARPA-H contract, we have a funded roadmap to the clinic.
The contract, valued at up to $34.5 million, will support the Pediatric Epilepsies and Rare CNS (PERC) Gene Editing Platform, a collaboration of academic researchers, clinicians, patient advocates, biotechnology companies, manufacturing experts, and regulatory leaders working to accelerate gene-editing therapies for children with rare neurological diseases, beginning with AHC and Dravet syndrome.
For the AHC community, this is a defining milestone.
Building a Scalable Development Pipeline
For decades, rare disease drug development has followed the same model: build a new program for each disease. While that approach can succeed for individual disorders, it is simply too slow, too expensive, and too difficult to scale across thousands of rare diseases.
The PERC Gene Editing Platform is designed to change that. Rather than rebuilding the scientific, manufacturing, regulatory, clinical, and patient engagement infrastructure for every disease, it brings them together in a shared platform that can be reused across multiple programs. The goal is to reduce development timelines, lower costs, and make gene-editing therapies possible for diseases that might otherwise never reach patients.
The platform will initially support AHC and Dravet syndrome, but its impact is intended to extend far beyond these two disorders. The scientific knowledge, manufacturing capabilities, regulatory experience, and clinical infrastructure developed through PERC can help accelerate future gene-editing programs for many additional rare neurological diseases.
For RARE Hope, the promise of PERC extends beyond the first two diseases. If successful, we will demonstrate that rare disease therapies do not have to be developed one disease at a time—that shared platforms can make treatments feasible for many more patient communities.
Building on Years of Scientific Progress
Today’s milestone builds on years of work across the consortium.
In 2025, investigators from the Broad Institute, The Jackson Laboratory, and RARE Hope demonstrated that prime editing could rescue multiple animal models of AHC, providing the first in vivo evidence that gene editing could reverse key features of the disease. Those studies established the scientific foundation for moving toward clinical translation.
The PERC program will combine advanced gene editors, including base editors and prime editors, with a novel AAV delivery vector known as TfR1 CapXTM, developed by Ben Deverman’s lab at the Broad Institute. CapX is engineered to engage the human transferrin receptor to cross the blood-brain barrier following intravenous infusion, with the goal of delivering gene-editing therapies throughout the central nervous system without neurosurgery or direct injection into the spinal fluid.
The consortium's objective is ambitious: to advance these programs toward first-in-human clinical studies within the next three years.
Building an Ecosystem for Translation
Since 2021, RARE Hope has worked alongside David Liu's laboratory at the Broad Institute and Cat Lutz’s team at The Jackson Laboratory’s Rare Disease Translational Center (RDTC) to move gene editing for AHC from an ambitious scientific concept toward clinical reality.
From the beginning, RARE Hope believed that success would require much more than a promising gene editor. It would require building an entire translational ecosystem around AHC. That meant advancing gene editing and gene therapy studies in parallel; developing the disease models needed to evaluate new treatments; investing in biomarkers, natural history studies, and clinically meaningful endpoints; engaging patients and families as research partners; and bringing together scientists, clinicians, industry, regulators, and patient organizations around a common strategy.
This ARPA-H contract is a powerful validation of that approach. Transformative therapies are not only built by a single laboratory or institution, but by an ecosystem working together toward a shared goal.
Looking Ahead
There is still significant bench science ahead before a gene-editing treatment reaches children living with AHC. Manufacturing, regulatory studies, safety testing, and clinical development also remain essential steps.
But for the first time, a coordinated, funded national effort has been established to move a gene-editing therapy for AHC toward the clinic, bringing together the scientific, clinical, manufacturing, regulatory, and patient communities around a shared goal.
As the program moves toward first-in-human clinical studies, the clinical translation effort will be led by Tim Yu at Boston Children's Hospital. Our longstanding collaboration on individualized RNA therapies for AHC makes this a natural next chapter in our partnership as gene editing advances toward patients.
The successful proposal was led by Winston Yan at the Broad Institute, whose leadership was instrumental in bringing this consortium together and securing the ARPA-H award. Winston will also lead the Broad Institute's expanding efforts in therapeutic translation.
Today marks the beginning of the next chapter: transforming years of scientific discovery into potential treatments for children living with AHC.