Topic: sickle cell anemia
CRISPR is a promising treatment for blood disorders, but it comes with challenges, such as off-target effects.
Vertex and CRISPR will co-develop a beta-thalassemia and sickle cell treatment, the first to come out of their $105 million partnership.
Cord blood, approved for the treatment of some blood disorders, could lead to a new treatment for cerebral palsy.
The tweaked gene therapy triggered a jump in the proportion of vector-positive cells in the two patients treated so far.
California biotech Global Blood Therapeutics is canning its work in idiopathic pulmonary fibrosis (IPF).
Biogen’s newly spun off hemophilia biotech Bioverativ has signed a new pact with U.K. biotech Bicycle Therapeutics.
Anti-CRISPR proteins may be the key to preventing harmful side effects from off-target editing.
An FDA greenlight for sickle cell therapy Endari has put developer Emmaus into the spotlight, but commercializing the drug could be challenging.
Bluebird Bio reported encouraging data on revamped gene therapy for sickle cell disease and beta thalassemia, but shares slipped 6% premarket.
With pricing pressure mounting in the diabetes space, insulin specialist Novo Nordisk may be looking to wean itself off its core focus with a rumored buyout for Global Blood Therapeutics, a buy that could help boost its own blood disorder business.