Drug Development
Transfer RNA’s unique biology could enable a mutation-specific, gene-agnostic—and more cost effective—approach to developing treatments for thousands of rare and ultra-rare diseases.
FEATURED STORIES
Through substantial leadership turnover and workforce cuts, the FDA has continued to support the advanced therapy sector, actively working to remove obstacles to innovation.
With immunology and inflammation blockbusters like AbbVie’s Skyrizi and Rinvoq reeling in nearly $7 billion combined in the third quarter, the pipeline-in-a-product strategy has never been more attractive.
The cholesterol-lowering drug is part of a suite of medicines that also includes MariTide and that Amgen Chief Medical Officer Paul Burton hopes will make the company the “undisputed leader in the management of cardiometabolic risk for patients” by 2030.
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Compass’ twin clinical updates regarding its lead candidate spotlight the promise of psychedelic medicine.
Novartis reported Thursday that a Phase III trial of iptacopan met its primary endpoint in paroxysmal nocturnal hemoglobinuria (PNH) - the company’s second win in this indication in as many months.
Vertex Pharmaceuticals agreed to a collaboration deal with Entrada Therapeutics. The two companies announced a collaboration to develop an intracellular Endosomal Escape Vehicle.
Relmada’s investigational NDMA receptor channel blocker, REL-1017, failed to meet its primary endpoint in major depressive disorder in the Phase III RELIANCE I trial.
AVROBIO is planning a Phase II/III registrational trial for its Gaucher Disease Type 3 (GD3) gene therapy candidate, AVR-RD-02, in 2023.
Neurocrine’s NBI-827104 failed to meet the primary endpoint in a Phase II study for epileptic encephalopathy with continuous spike-and-wave during sleep, the company announced Tuesday.
Eli Lilly and Boehringer Ingelheim declared another victory Wednesday for Jardiance – this one for children and adolescents with Type 2 Diabetes.
Findings from the Phase II ARTEMIS-UC and Phase IIa APOLLO-CD trials show that Prometheus’ PRA023 is potentially safe and effective for treating ulcerative colitis and Crohn’s disease.
Novartis announced that its Kisqali with endocrine therapy offered almost a year of progression-free survival (PFS) for an aggressive form of HER+/HER2- metastatic breast cancer.
Apogee Therapeutics sprang onto the immunology/inflammatory scene Wednesday heavy on capital but light on salient details of the pipeline.