News & Blog

Press Release: Graviton BioScience Announces Clearance of Investigational New Drug (IND) for GV101 for the Treatment of Friedreich’s Ataxia

Design Therapeutics Announces Four-Week IV Data from the RESTORE-FA Trial of DT-216P2 Demonstrating Clinical Improvements and Comprehensive Biomarker Activity in FA

Community Statement: Update from Biogen

Press release and community statement: Lexeo Therapeutics Reports Fourth Quarter and Full Year 2025 Financial Results and Operational Highlights

Larimar Therapeutics Announces FDA Breakthrough Therapy Designation for Nomlabofusp in FA and Reiterates Planned BLA Submission in June 2026

PTC Therapeutics Provides Corporate Update and Reports Fourth Quarter and Full Year 2025 Financial Results

CIRM announces $7.4 million funding award for CRISPR/Cas9-mediated gene editing of hematopoietic stem and progenitor cells for Friedreich’s ataxia

Solid Biosciences Receives FDA Rare Pediatric Disease Designation for SGT-212 Dual Route of Administration Gene Therapy for Friedreich’s Ataxia

Lexeo Therapeutics Reports Third Quarter 2025 Financial Results and Operational Highlights

Larimar Therapeutics Announces Positive Data from Ongoing Long-term Open Label Study and Updates to Nomlabofusp Program for Friedreich’s Ataxia

Lexeo Therapeutics Reports Second Quarter 2025 Financial Results and Operational Highlights

Design Therapeutics Initiated Phase 1 Single Ascending Dose Trial of DT-216P2 in Healthy Volunteers