The STXBP1 Summit: Powered By Hope
With the close of the Capsida clinical trial, many families have asked me and other board members: What do we do now; how do we move forward? As parents, we know there was so much hope attached to that first gene therapy clinical trial for STXBP1.
I want to assure you that there is reason to still have HOPE.
The Capsida gene therapy was only one of the therapies in development for STXBP1. In fact there are over 18 therapies currently in development -- across different types of treatments: gene therapies, ASOs, and other RNA therapies and small molecules.
At this year's STXBP1 Summit kickoff in Philadelphia, I will give an update on the types of treatments which are in development for STXBP1. The therapeutic pipeline for STXBP1 consists of medicines being developed by biopharma partners and academic researchers. And, a number of these treatments in the pipeline have been funded by our Foundation. In fact, just last summer the Foundation funded two new programs: a gene therapy with SUNY Buffalo, and a CRISPR activation therapy at UC Davis.
Paula Vij from UC Davis will be presenting their work on CRISPR activation for STXBP1 at the Family meeting. And Beverly Davidson, who is Director of Translational Research at the ENDD Center at CHOP / UPenn, will talk about her team's genetic therapy development. Additionally, we'll hear updates from other researchers on therapies, clinical trial readiness and natural history studies.
It is in fact HOPE that powers us and the researchers each and every day.
Now, read on for a parent perspective from Bridget Finn on what Summit means to her!
I look forward to seeing you soon at the STXBP1 Summit!
-Charlene Son Rigby
President of the Board and Mom to Juno
“As a parent, caregiver, and founder of FLOURISH, the STXBP1 Summit is one of the most hopeful moments of the year for me. It is where our community comes together with researchers, clinicians, industry partners, and foundation leaders to advance science with our children at the center. I leave feeling informed, inspired, and deeply grateful—not only for the progress being made, but for the role our community plays in making that progress possible. The Summit is a powerful reminder that families are not standing on the sidelines of this work; we are partners in driving it forward. Just as importantly, it is a place where my daughter Flo and our entire family feel truly seen, understood, and embraced. In a world that can often feel isolating, that sense of belonging—and the shared determination to build a better future for our children—is incredibly powerful.”