Quoin Gains FDA Fast Track for QRX003 in Peeling Skin Syndrome
ASHBURN, Va. — September 22, 2026 Quoin Pharmaceuticals announced that the U.S. Food and Drug Administration has granted Fast...
Rare Pediatric Disease
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Arcturus to Present ARCT-810 Phase 2 OTC Deficiency Data
SAN DIEGO — September 18, 2026 Arcturus Therapeutics announced plans to provide an update on its ARCT-810 Phase 2...
Abbisko Therapeutics Reports Positive Phase 2 Lavengratinib Data
Shanghai, China, September 21, 2026 Abbisko Therapeutics has announced positive preliminary results from a Phase 2 clinical study evaluating...
Revvity to Acquire Human Cell Design for Metabolic Drug Discovery
WALTHAM, Mass. — September 9, 2026 Revvity, Inc. announced that it has entered into a definitive agreement to acquire...
Quoin Pharmaceuticals Gains FDA Pediatric Designation for QRX003
ASHBURN, Va. — September 8, 2026 Quoin Pharmaceuticals Ltd. announced that the U.S. Food and Drug Administration (FDA) has...
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Scholar Rock Advances Apitegromab FDA Review with Second Fill-Finish Facility
CAMBRIDGE, Mass., August 7, 2026 Scholar Rock announced that the U.S. Food and Drug Administration (FDA) review of its...
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)...
Opus Genetics Completes Enrollment in OPGx-LCA5 Phase 3 Trial
RESEARCH TRIANGLE PARK, N.C., Aug. 3, 2026 Opus Genetics, Inc. has completed patient enrollment in its registrational Phase 3...
Saol Therapeutics Advances SL1009 in FDA Review
ROSWELL, Ga., DUBLIN, Ireland, and HAMILTON, Bermuda, July 28, 2026 Saol Therapeutics has announced that the U.S. Food and...
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