Tag Archive for: sickle cell disease

Novartis’ sickle cell disease therapeutic Adakveo (crizanlizumab) is under review by the European Medicines Agency’s Committee for Medicinal Products for Human Use after failing to replicate previous efficacy results in a Phase III trial.

2022 featured pivotal data that could change the landscape in non-alcoholic steatohepatitis, Alzheimer’s disease, sickle cell disease and many more indications.

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.

CRISPR gene editing leader Editas Medicine often makes biotech headlines for its therapies for sickle cell and retinal diseases. Less often does it make the news for its preclinical cancer pipeline – which could be why the company is reportedly considering a sale of these assets.

Shares of Global Blood Therapeutics (GBT) continue to surge in premarket trading after Pfizer officially announced it was acquiring the hematology-focused company for $5.4 billion in order to bolster its presence in the multi-billion dollar rare disease market.

In a perplexing social paradox, diseases that disproportionately affect people of color are frequently also ones that receive less attention from the medical world. Compounding the problem, diseases that affect people across all races often have fewer people of color in clinical trials for new treatments. The reasons for the problem are widely speculated.

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.