Abeona Congratulates Ultragenyx on FAYUVI FDA Approval
CLEVELAND, Ohio — September 18, 2026 Abeona Therapeutics congratulated Ultragenyx Pharmaceutical on the U.S. FDA approval of FAYUVI™ (rebisufligene...
lysosomal storage disorder
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PTC Therapeutics to Acquire ST-920 Fabry Gene Therapy Program
Warren, New Jersey, August 12, 2026 PTC Therapeutics announced that it was selected as the winning bidder to acquire...
Senhwa Wins TFDA Clearance for CX-5461 Immunotherapy Trial
Taiwan, August 6, 2026 Senhwa Biosciences, Inc. has received Taiwan Food and Drug Administration (TFDA) clearance for a multicenter...
Galibra Neuroscience Wins FDA Designations for SSADH Therapy
Boston, Massachusetts, August 5, 2026 Galibra Neuroscience has received both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation...
Phoenix Nest Secures $2.98M to Advance JLK-247 Gene Therapy
Brooklyn, New York, August 5, 2026 Phoenix Nest Inc. has been awarded a $2,984,658 Small Business Innovation Research (SBIR)...
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JCR Pharmaceuticals Wins UAE Approval for IZCARGO
HYOGO, Japan, July 21, 2026 JCR Pharmaceuticals announced that the United Arab Emirates (UAE) has granted marketing authorization for...
Apertura Gene Therapy Advances NPC1 Gene Therapy Research
NEW YORK | July 9, 2026 Apertura Gene Therapy, a biotechnology company developing next-generation adeno-associated virus (AAV) capsids for...
Spruce Biosciences Reports Six-Year TA-ERT Data in MPS IIIB
SOUTH SAN FRANCISCO, Calif., June 8, 2026 Spruce Biosciences announced new long-term clinical data demonstrating the potential of tralesinidase...
Sanofi’s Venglustat Gains FDA Priority Review for GD3
PARIS, France, May 28, 2026 Sanofi has achieved a major regulatory milestone with the U.S. Food and Drug Administration...
AskBio Expands Rare Disease Pipeline With AB-1009 Pompe Trial Milestone
DURHAM, N.C., May 11, 2026 AskBio Inc., a gene therapy company wholly owned and independently operated as a subsidiary...
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