Solid Biosciences Doses First Participant in First-in-Class Phase 1b FALCON Trial Evaluating SGT-212 Dual-Route Gene Therapy for the Treatment of Friedreich’s Ataxia

About the FALCON Trial FALCON is a first-in-human, open-label, multi-center Phase 1b clinical trial designed to evaluate the safety and tolerability of SGT-212 in participants aged 18-40 who have been ...
The American Journal of Managed Care

Managing Advanced Stages of Friedreich’s Ataxia: Challenges and Emerging Therapeutic Avenues

A health care provider outlines treatment objectives and supportive interventions for managing Friedreich ataxia, addressing associated comorbidities and symptoms in clinical settings. August 7th ...

Solid Biosciences Receives FDA Orphan Drug Designation for SGT-212 Dual-Route Gene Therapy for the Treatment of Friedreich’s Ataxia

SGT-212 has received FDA Fast Track, Rare Pediatric Disease and Orphan Drug designations -- Dosing of the first participant in the Phase 1b ...
The American Journal of Managed Care

Managing Adverse Events in Novel Friedreich’s Ataxia Therapy

A key opinion leader outlines common adverse events associated with omaveloxolone treatment in Friedreich's ataxia patients and describes strategies used to manage or minimize these adverse effects.
Nasdaq

Solid Biosciences' SGT-212 Gene Therapy for Friedreich's Ataxia Receives FDA IND Clearance, Phase 1b Trial Set for 2025

Solid Biosciences announces FDA clearance for SGT-212, targeting Friedreich’s ataxia with dual gene therapy administration routes, initiating Phase 1b trial in 2025. Solid Biosciences Inc. announced ...
News Medical on MSN

CIRM awards $7.4 million to advance stem cell-based gene therapy for Friedreich's ataxia

The California Institute for Regenerative Medicine (CIRM) has awarded $7.4 million to support a University of California San Diego team developing a first-of-its-kind stem cell-based gene therapy for ...
FierceBiotech

Lexeo links gene therapy to improvements in rare heart disease but disappoints investors

Lexeo Therapeutics has guided its Friedreich ataxia (FA) gene therapy through an early test, generating evidence of improvements in cardiac status in early-phase trials. But the biotech ran into ...
BioWorld

Lexeo, FDA position Friedreich ataxia therapy for accelerated path

Lexeo Therapeutics Inc. feels like it’s in a faster lane to a BLA for its Friedreich ataxia cardiomyopathy gene therapy after talking with the U.S. FDA. The agency told Lexeo that LX-2006 could be on ...
Healio

First patient dosed in phase 1/2a trial of Huntington’s, spinocerebellar ataxia therapy

We were unable to process your request. Please try again later. If you continue to have this issue please contact [email protected]. The trial will evaluate VO659, an antisense ...
Business Wire

Papillon Therapeutics Receives Orphan Drug Designation from the U.S. Food and Drug Administration for PPL-001 for the Treatment of Friedreich's Ataxia

SAN DIEGO--(BUSINESS WIRE)--Papillon Therapeutics Inc., a clinical-stage biotechnology company advancing a pipeline of multi-systemic genetic medicines directed at the underlying causes of inherited ...
Nasdaq

Solid Biosciences Receives FDA Fast Track Designation for SGT-212, a Dual Route Gene Therapy for Friedreich’s Ataxia

Solid Biosciences' SGT-212, dual-route gene therapy for Friedreich’s ataxia, receives FDA Fast Track designation for expedited development. Solid Biosciences Inc. has announced that its gene therapy ...