BridgeBio BBP-418 Shows Strong Phase 3 Results in LGMD2I/R9
PALO ALTO, CA — March 11, 2026 BridgeBio Pharma announced that its investigational therapy BBP-418 demonstrated consistent efficacy and...
rare disease therapy
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Quoin Pharmaceuticals Advances QRX003 for Netherton Syndrome
Ashburn, Virginia — March 9, 2026 Clinical-stage specialty biopharmaceutical company Quoin Pharmaceuticals Ltd. continues to advance the development of...
FDA Priority Review Granted to Garetosmab for FOP
Rare Disease Biologic Advances Toward U.S. Approval – February 19, 2026 | Tarrytown, New York Regeneron Pharmaceuticals announced that...
GenEditBio Wins FDA IND Clearance for GEB-101 Therapy
HONG KONG | January 5, 2026 — GenEditBio has received U.S. FDA clearance of its Investigational New Drug (IND)...
Health Canada OKs Sephience™ for Phenylketonuria (PKU)
TORONTO, Dec. 10, 2025 — Health Canada has granted regulatory approval to Sephience™ (sepiapterin), an oral therapy developed by...
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Immedica Submits NDS for Loargys® (pegzilarginase) in Canada
Stockholm, Sweden – October 31, 2025 — Immedica Pharma has announced that its New Drug Submission (NDS) for Loargys®...
Halia Therapeutics’ Ofirnoflast Gains FDA Orphan Drug Status for MDS
LEHI, Utah, Oct. 23, 2025 — Halia Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on therapies targeting the root...
FDA Grants Fast Track to AJ201 for Kennedy’s Disease Therapy
TAIPEI, Taiwan – October 23, 2025 AnnJi Pharmaceutical Co., Ltd. (AJ Pharm) announced that the U.S. Food and Drug...
BioCryst Acquires Astria Therapeutics to Expand HAE Pipeline
BOSTON – October 14, 2025 – BioCryst Pharmaceuticals, Inc. (NASDAQ: BCRX) and Astria Therapeutics, Inc. (NASDAQ: ATXS) announced a...
Nexiguran Ziclumeran Shows Durable Phase 1 Benefits in ATTR
CAMBRIDGE, Mass., Sept. 25, 2025 — Intellia Therapeutics announced positive longer-term Phase 1 data for its investigational CRISPR-based therapy...
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