Biologically Active Substance
Editas Medicine Reboots With Cholesterol Gene Therapy (EDIT-401) as Lead Asset
Editas Medicine; EDIT-401; gene therapy; hyperlipidaemia; LDL cholesterol; LDLR gene; in vivo gene editing; strategic pivot; clinical development
Merck’s Oral PCSK9 Inhibitor Enlicitide Decanoate Achieves Third Consecutive Phase 3 Success
Merck; oral PCSK9 inhibitor; enlicitide decanoate; Phase 3 trials; CORALreef Lipids; hypercholesterolemia; lowering LDL cholesterol; statin therapy; first oral PCSK9; clinical trial results
Novartis Takes Another Shot at Alpha-Synuclein With $2.2B Arrowhead CNS Deal
Novartis; Arrowhead Pharmaceuticals; alpha-synuclein; Parkinson’s disease; RNA interference (RNAi); gene silencing; ARO-SNCA; TRiM platform; neurodegeneration; biotech partnerships
Sanofi’s Wayrilz Receives FDA Approval as First BTK Inhibitor for Immune Thrombocytopenia
Wayrilz; rilzabrutinib; FDA approval; immune thrombocytopenia; ITP; BTK inhibitor; Sanofi; LUNA 3 trial
Servier Pays $210M Upfront for Ex-US Rights to Ideaya’s PKC Inhibitor Darovasertib
Servier; Ideaya Biosciences; darovasertib; protein kinase C inhibitor; PKC inhibitor; uveal melanoma; oncology licensing deal; clinical trials; milestone payments; royalties
Ionis Posts Strong Results for Tryngolza in Severe Blood Fat Disease
Ionis Pharmaceuticals; Tryngolza; olezarsen; hypertriglyceridemia; triglycerides; Phase III Essence study; FDA approval; familial chylomicronemia syndrome; cardiovascular risk reduction; drug launch
Recent Advances for Confident Cell Line Transfer
cell line development; cell transfer; clone stability; electroporation; transfection; quality assurance; GMP compliance; cell therapy
AbbVie’s Rinvoq Achieves Strong Phase 3 Results in Alopecia Areata, Clearing Key Efficacy Milestone
AbbVie; Rinvoq; Phase 3 trial; alopecia areata; hair regrowth; upadacitinib; SALT score; JAK inhibitor
FDA Rejects PTC’s Vatiquinone Application for Friedreich’s Ataxia, Requests Further Studies
FDA rejection; PTC Therapeutics; vatiquinone; Friedreich’s ataxia; complete response letter; drug efficacy; additional clinical studies
FDA Rejection Derails PTC Therapeutics’ Rare Disease Hopeful
PTC Therapeutics; FDA rejection; vatiquinone; Friedreich’s ataxia; rare disease; efficacy concerns; complete response letter; clinical trial failure