Gene therapy is reshaping modern medicine, offering the potential for lasting, even curative treatments for a wide range of ...
Strategic acquisition will enable Genezen to deliver late-phase and commercial gene therapy development and manufacturing services to customers around the world. "uniQure has been a pioneer in gene ...
A study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers lessons learned to inform best practices as manufacturers and medical centers ...
SOMERVILLE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (NASDAQ: BLUE) (“bluebird bio” or the “bluebird”) today announced the details of its U.S. commercial infrastructure to support timely, equitable ...
Individually, rare diseases are rare, but there are so many rare diseases that over 400 million people are affected worldwide. Together, rare diseases aren’t so rare. Only 5% of rare diseases have an ...
Price of LYFGENIA reflects its value as a potentially curative gene therapy for sickle cell disease through durable production of anti-sickling adult hemoglobin (HbAT87Q) and resolution of ...