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uniQure’s AMT-130 Receives FDA Breakthrough Therapy Designation for Huntington’s Disease
Apr 17 2025 -
Rare Diseases in Malaysia: An Overlooked Crisis Affecting One Million
Apr 17 2025 -
French Biotech Brink Therapeutics Secures €3.5M to Pioneer Next-Generation Gene Editing with Recombinases
Apr 17 2025 -
Glycomine Secures $115M Series C to Advance PMM2-CDG Therapy
Apr 16 2025 -
Lexeo Reports Promising Interim Phase 1/2 Results for LX2006 in FA Cardiomyopathy, Plans Registrational Study
Apr 15 2025 -
Precision BioSciences Receives FDA Fast Track Designation for PBGENE-HBV, an In Vivo Gene Editing Therapy for Chronic Hepatitis B
Apr 15 2025 -
Atsena Therapeutics’ ATSN-201 Gene Therapy Receives FDA RMAT Designation for X-Linked Retinoschisis
Apr 15 2025 -
Sarepta Therapeutics Accelerates LGMD Gene Therapy Pipeline Following FDA Green Light
Apr 15 2025 -
Fate Therapeutics’ FT819 CAR T-cell Therapy Receives FDA RMAT Designation for Lupus Treatment
Apr 15 2025