SGT-212 / Solid BioSci - LARVOL DELTA

FALCON: A Study of SGT-212 Gene Therapy in Friedreich's Ataxia (clinicaltrials.gov) - P1 | N=10 | Recruiting | Sponsor: Solid Biosciences Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • First-in-human • Ataxia • Friedreich ataxia • Gene Therapies • Movement Disorders

Solid Biosciences Receives FDA Rare Pediatric Disease Designation for SGT-212 Dual Route of Administration Gene Therapy for Friedreich’s Ataxia (GlobeNewswire) - "FALCON Phase 1b clinical trial participant screening underway."

Breakthrough therapy • Enrollment status • Friedreich ataxia

Efficacy and Safety of a Novel AAV FXN Gene Therapy (SGT-212) for the Treatment of Friedreich's Ataxia (AHA 2025) - "These nonclinical studies demonstrate that a one-time administration of SGT-212 can restore FXN expression in disease-relevant tissues, improve cardiac and neurologic phenotypes in mouse models, and is well tolerated in NHPs. These findings support advancement to a Phase 1b clinical trial using combined IV/IDN administration."

Clinical • Gene therapy • Ataxia • Cardiomyopathy • Cardiovascular • Friedreich ataxia • Gene Therapies • Metabolic Disorders • Movement Disorders • FXN

FALCON: A Study of SGT-212 Gene Therapy in Friedreich's Ataxia (clinicaltrials.gov) - P1 | N=10 | Not yet recruiting | Sponsor: Solid Biosciences Inc.

First-in-human • New P1 trial • Ataxia • Friedreich ataxia • Gene Therapies • Movement Disorders

Solid Biosciences Receives FDA Fast Track Designation for SGT-212 Dual Route of Administration Gene Therapy for Friedreich’s Ataxia (GlobeNewswire) - "Solid Biosciences Inc...today announced that it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for SGT-212, the Company’s, AAV-based gene therapy candidate for the treatment of Friedreich’s ataxia (FA). SGT-212 will deliver the full-length frataxin gene via dual routes of administration incorporating intradentate nucleus (IDN) and intravenous (IV) infusions, designed to promote restoration of therapeutic levels of the frataxin protein to address neurologic, cardiac and systemic clinical manifestations of FA."

Fast track • Friedreich ataxia

Solid Biosciences Announces FDA IND Clearance for First-In-Industry Dual Route of Administration Gene Therapy to Treat Both Neurologic and Cardiac Manifestations of Friedreich’s Ataxia (GlobeNewswire) - "Solid Biosciences...today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for SGT-212 for the treatment of Friedreich’s ataxia (FA)...The IND was supported by a robust preclinical package demonstrating safe transduction and frataxin expression in these target tissues...In the second half of 2025, the Company expects to initiate a first-in-human, open-label, dose-finding Phase 1b clinical trial of SGT-212."

IND • New P1 trial • Friedreich ataxia