The announcement comes just a month after Casgey was approved by the FDA in December 2023 to treat patients with sickle cell disease, when it became the first CRISPR gene editing therapy to reach the U.S. market.
Tag Archive for: Beta-thalassemia
The Institute for Clinical and Economic Review (ICER) concluded Tuesday that gene therapies for hemophilia A and B are worth it even at a hefty lifetime cost of $2.5 million.
The U.S. Food and Drug Administration on Wednesday approved bluebird bio’s gene therapy for patients with a rare disorder requiring regular blood transfusions, and the drugmaker priced it at a record $2.8 million.
Precision BioSciences entered into an exclusive global in vivo gene editing research and development collaboration and license deal with Novartis Pharma. The two companies will work to develop a potential cure for hemoglobinopathies such as sickle cell disease and beta-thalassemia.
Advisers to the U.S. Food and Drug Administration on Thursday voted to recommend approval of bluebird bio’s treatment for a rare neurological disorder, bringing it closer to becoming the third gene therapy to be available in the United States.
It’s game day for bluebird bio. The company, battered by layoffs and cash concerns, faces a two-day crucible as the U.S. Food and Drug Administration’s Cell, Tissue and Gene Therapies Advisory Committee will give two lentiviral vector gene therapies a thumbs up or down.
Bluebird bio’s blood disorder treatment demonstrates “clinically meaningful” benefit in patients, staff reviewers at the U.S. Food and Drug Administration said in briefing documents published on Tuesday.