Infrequent
Glycomine raises $115M to see if it can treat rare glycosylation disorder
Infrequent, GLM101, Glycomine, Congenital disorder of glycosylation type 1A
Arbor sails into rocky gene editing waters with $74M for rare liver disease therapy
Gene editing, CRISPR, Rare liver disease, Primary hyperoxaluria type 1 (PH1), Funding
FDA Approves Ono Pharmaceutical’s Romvimza for Rare Joint Tumor
Romvimza, tenosynovial giant cell tumor (TGCT), FDA approval, Ono Pharmaceutical, Deciphera acquisition, kinase inhibitor, rare cancer treatment
Biogen and Stoke Therapeutics Partner on Promising Dravet Syndrome Treatment
Biogen, Stoke Therapeutics, zorevunersen, Dravet syndrome, antisense oligonucleotide, rare epilepsy, drug development, pharmaceutical partnership
SpringWorks Therapeutics Secures FDA Approval for Gomekli to Treat NF1-Related Tumors
SpringWorks Therapeutics, Gomekli, mirdametinib, FDA approval, neurofibromatosis type 1 (NF1), plexiform neurofibromas (PN), rare disease treatment, MEK inhibitor
Narsoplimab Demonstrates Significant Survival Benefit in Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy
Narsoplimab, Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy (HSCT-TMA), Lectin Pathway Inhibition, MASP-2 Inhibitor, Rare Disease Treatment, Overall Survival Improvement
Relay Therapeutics Partners with Elevar Therapeutics in $500M Deal for Lirafugratinib in Rare Cancer Treatment
Relay Therapeutics, Elevar Therapeutics, Lirafugratinib, FGFR2-Driven Cholangiocarcinoma, Rare Cancer Treatment, Biotech Partnership
FDA Rejects Govorestat for Classic Galactosemia, Citing Clinical Deficiencies
FDA rejection, govorestat, classic galactosemia, Applied Therapeutics, rare disease drug approval, clinical deficiencies
Sarepta Therapeutics and Arrowhead Pharmaceuticals Enter $825M Global Licensing and Collaboration Agreement for Multiple siRNA Programs
Sarepta Therapeutics, Arrowhead Pharmaceuticals, siRNA programs, Global Licensing and Collaboration Agreement, Rare genetic diseases, Gene therapy, RNA interference
Sarepta Therapeutics and Arrowhead Pharmaceuticals Enter $10 Billion Collaboration for Rare Disease Treatments
Sarepta Therapeutics, Arrowhead Pharmaceuticals, RNAi, Rare Diseases, Genetic Medicine, Collaboration Agreement, Biotech, Pharmaceutical Industry