In a recent discussion, Peter Marks, the director of the FDA’s Center for Biologics Evaluation and Research, and Janet Woodcock, former FDA deputy commissioner, highlighted the critical need for regulatory flexibility in developing rare disease therapies. They...
Graphical abstract. Credit: Cell Reports Medicine (2024). DOI: 10.1016/j.xcrm.2024.101552 Behind the scenes, as tumors progress and gain resistance to treatment, a protein called YB-1 quietly gives directions. Now, researchers at Oregon Health & Science University...
–Supplemental New Drug Application (sNDA) submission based on Phase 3 study of vibegron 75mg (GEMTESA) demonstrating statistically significant reductions in daily micturition and urgency episodes– –If approved, vibegron will be the first and only beta-3 agonist for...
LEXEO Therapeutics, Inc., a clinical stage genetic medicine company, entered an in-license agreement with Cornell University to expedite development of the investigational gene therapy candidate LX2006 for the treatment of Friedreich ataxia (FA) cardiomyopathy. Under...
CureVac N.V., a global biopharmaceutical company developing a new class of medicines based on messenger ribonucleic acid (mRNA), and The University of Texas MD Anderson Cancer Center entered a co-development and licensing agreement to develop novel mRNA-based cancer...
