Our Progress
Updates on our endeavor to develop an NPHP1 gene therapy for the retina.
Huge News – New Research, World-Class Advisors, and Global Partnerships
Our work has officially begun at Harvard’s Boston Children’s Hospital, guided by a nine-member Scientific Advisory Board stacked with experts from around the world. From Parisian partnerships to advancing mouse model studies, this issue is filled with exciting milestones on the road to a cure for NPHP1 retinal dystrophy. We’re aiming for a victory party in 2027 — and you’re invited.
A Year in Review – Assembling the Pieces for a Cure
2024 was a landmark year for the NPHP1 Family Foundation. From recovering cryopreserved mice and building patient cell lines, to advocating for rare disease families and forging new research partnerships, we’ve made real progress toward our goal: a gene therapy that preserves sight. Caryn shares a powerful personal pitch from Rare Disease Bootcamp, underscoring the urgency and achievability of our mission.
Cures in Sight – Building Momentum for NPHP1 Gene Therapy
We’re closer than ever to stopping NPHP1-associated retinal dystrophy in its tracks. From assembling a dream team of scientists and clinicians to forming partnerships with Odylia Therapeutics, Boston Children’s Hospital, and our Scientific Advisory Board, this update shares why our vision for a cure is clearer than ever. Your support is powering groundbreaking research and laying the foundation for clinical trials.
