Aeglea BioTherapeutics announced on April 11 promising Phase III results for patients diagnosed with arginase 1 deficiency (ARG1-D), a rare hereditary metabolic disorder. Aeglea said on April 12 the company had submitted its first-ever Biologics License Application (BLA) to the U.S. Food and Drug Administration for the drug.
Following the ongoing trend in the pharmaceutical industry, bluebird bio orchestrated a restructuring that includes saving financially and cutting staff. The restructuring is designed to prolong the company’s budget as bluebird bio awaits decisions from the U.S. Food and Drug Administration (FDA).
bluebird bio Inc. announced updated results from the clinical development program for the company’s investigational elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy in patients with cerebral adrenoleukodystrophy (CALD), including long-term results from the Phase 2/3 Starbeam study and data from the Phase 3 ALD-104 study.
The software of life is how Moderna Inc. CEO Stephane Bancel describes messenger RNA (mRNA), which is at the core of the company’s drug development process.
The U.S. Food and Drug Administration granted Fast Track designation for mRNA-3927, Moderna Inc.’s investigational messenger RNA therapeutic for propionic acidemia (PA).