Dear CPVT Community,
We are pleased to introduce Solid Biosciences to the CPVT community—and to share important news about our investigational gene therapy, SGT-501.
Patient-focused and founded by individuals directly impacted by Duchenne muscular dystrophy, Solid’s mission is to improve the daily lives of people living with rare neuromuscular and cardiac conditions. What began as a deeply personal effort has grown into a broader commitment to develop meaningful therapies for other serious and underserved conditions—including CPVT.
We’re pleased to share that both the U.S. Food and Drug Administration (FDA) and Health Canada have cleared our applications to begin a clinical trial for SGT-501, a gene therapy being developed for individuals living with CPVT caused by RYR2 mutations. This marks an important step forward in our efforts to bring new treatment options to this community.
About SGT-501 and the Science Behind It
SGT-501 is a gene therapy candidate designed to address the underlying cause of CPVT—abnormal calcium signaling in the heart. It uses an AAV (adeno-associated virus) vector to deliver a functional copy of the CASQ2 gene directly to heart muscle cells. CASQ2 is a protein that helps regulate calcium levels inside cells. In individuals with RYR2 mutations, restoring CASQ2 levels may help stabilize heart rhythm and reduce the risk of life-threatening arrhythmias.
The science behind SGT-501 is built on the pioneering research of Dr. Silvia Priori and her team at ICS Maugeri in Pavia, Italy. Dr. Priori has spent decades advancing our understanding of inherited arrhythmias and has led groundbreaking work showing that increasing CASQ2 expression can restore calcium balance and reduce arrhythmias in models of CPVT. Solid in-licensed this therapeutic approach in 2023 and has since worked closely with Dr. Priori’s lab to translate this research into a clinical-stage therapeutic candidate.
Guided by the CPVT Community
The insights of individuals and families in the CPVT community have provided valuable guidance – ensuring that the patient perspective is integrated into every step of our clinical development. This collaboration has influenced how we design our study and helped us focus on what truly matters to individuals and caregivers living with CPVT. We are committed to continuing this engagement as the program advances.
What’s Next
We plan to initiate this Phase 1b clinical trial in the U.S. and Canada later this year (Q4 2025) to evaluate the safety and tolerability of SGT-501.
In the coming weeks, we will partner with the SADS Foundation to host a community webinar. This event will offer an opportunity to meet our team, ask questions, and learn more about our investigational gene therapy, SGT-501, and our plans for the upcoming clinical trial. We’ll share the date and details soon, and we hope you’ll join us for this conversation.
We are grateful for the opportunity to partner with the CPVT community and are committed to continuing this journey together—openly, respectfully, and always with patients at the center.
Sincerely,
Annie Ganot
Co-Founder & Head of Patient Advocacy
Solid Biosciences
www.solidbio.com