End-to-end vector manufacturing optimizes batch success and delivers more doses at lower cost.

As we move into 2023, BioSpace takes a pause to reflect on the year gone by and some of the most remarkable news bits, looking at the top five noteworthy FDA drug and biologic therapy approvals in 2022.  

The agency’s top five represent an eclectic mix of cancer, cardiovascular and rare disease drugs.

Bluebird Bio said that the agency had lifted a partial clinical hold on studies for its gene therapy to treat the blood disorder sickle cell disease.

Multimillion-dollar gene therapies being tested for sickle cell disease will be cost-effective in the United States if health disparities tied in part to decreased access to healthcare are taken into account, researchers said at a meeting of blood-disease specialists.

The FDA approved bluebird bio’s lentiviral vector (LVV) eli-cel Friday as the first therapy to slow the progression of neurologic dysfunction in juvenile boys with early, active cerebral adrenoleukodystrophy (CALD).

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.

Leading healthcare venture capital firm Third Rock Ventures announced a new fund Wednesday morning, Third Rock Ventures Fund VI, providing the company with $1.1 billion to invest in new life science companies. This brings Third Rock to a total of $3.8 billion across its venture funds.