Dear Friedreich’s ataxia (FA) community,
As we begin the new year, we want to wish you and your families a happy and healthy 2026 and thank the FA community for your trust and partnership. We are writing to share an important update on FALCON, our first-in-human clinical study evaluating SGT-212, Solid’s investigational gene therapy for Friedreich’s ataxia.
Today, Solid Biosciences announced the first participant has been dosed in FALCON, a Phase 1b, open-label, dose-finding clinical trial designed to evaluate the safety and tolerability of SGT-212 in individuals aged 18-40 living with FA. This study evaluates a novel, first-in-class FA gene therapy approach that uses dual routes of administration comprised of an intradentate nucleus (IDN) infusion, targeting the cerebellar dentate nuclei, followed by an intravenous (IV) infusion. By leveraging this dual-route approach, we aim to address the neurologic, cardiac, and systemic manifestations of FA.
We want to recognize the extraordinary leadership and partnership of the Friedreich’s Ataxia Research Alliance (FARA), whose ongoing guidance and support continue to shape and strengthen our clinical program. We also wish to acknowledge the many scientists, clinicians, and partners whose collaboration made the development of SGT-212 and this dual-route gene therapy approach possible. This work reflects our shared determination to advance clinically meaningful therapies that address the urgent unmet needs of this community – to improve both survival and quality of life for people living with FA.
We are immensely grateful to the patients and families whose support, guidance and participation made this clinical study possible. Their trust, courage, time, and sacrifices help advance research for the entire FA community.
We truly appreciate the strong interest and patience of the FA community as this carefully designed, small study is now underway at its first site. While not all individuals who are interested will be able to participate in this initial trial, we hope to increase opportunities for participation over time through future study expansion and through continued community engagement that helps guide our progress.
As the FALCON study progresses, we are committed to keeping the FA community informed through regular and transparent communication, including both written updates and a dedicated community webinar planned for the coming months.
The most up-to-date information about the study can be found on ClinicalTrials.gov (NCT07180355). Additional educational resources provided by FARA, including educational information on SGT-212 and gene therapy can be found at https://www.curefa.org/clinical-studies/a-study-of-sgt-212-gene-therapy-in-friedreichs-ataxia-falcon/. These resources serve as central hubs for information about our ongoing research and broader community education.
We anticipate sharing an initial data update from the FALCON trial in the second half of 2026, subject to patient enrollment.
If you have questions or would like to connect with our patient advocacy team, please reach out to us at community@solidbio.com.
Thank you again for your continued engagement and partnership. We look forward to staying connected with the FA community.
Sincerely,
Annie Ganot
Co-Founder and Head of Patient Advocacy
Solid Biosciences