The World's First Clinical Base Editing Therapy for Hemoglobinopathy
BioPharma
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Modifying a patient’s DNA is no longer just for science fiction novels. The CRISPR gene editing technique developed by Jennifer Doudna and Emmanuelle Charpentier only took 10 years to reach the market as Casgevy (exagamglogene autotemcel/exa-cel, Vertex Pharmaceuticals Inc.), treating congenital pathologies such as β-thalassemia and severe sickle cell disease (SCD). But science does not stop.
Sickle cell disease is among the Beam Therapeutics programs spared, and here the company needs to show differentiation from genetic medicines under FDA review from Vertex Pharmaceuticals and Bluebird Bio. Meanwhile, Beam aims to find partners for its research in allogeneic cancer cell therapies and hepatitis B.
This article seeks to illuminate these pioneering developments and their potential impact on future scientific endeavors of biotechnology and genetics
A MAN was given weeks to live after going to A&E with back pain thinking he had just pulled a muscle.Dylan Lamb, 20, first noticed the problems in