Sickle cell disease
The company had said in October said that it was looking to license out its ex vivo editor renizgamglogene autogedtemcel to focus its resources on its in vivo platform.
Vertex unveiled long-term durability data for Casgevy, while Beam presented Phase I/II findings for its investigational base editor BEAM-101, building up to a BLA by late 2026.
The payment scheme will tie gene therapy payments to improvements in health outcomes—and could potentially boost the uptake of these sickle cell disease treatments.
Bluebird has just two quarters until it’s out of cash. Executives are looking for financing to extend that runway to a projected breakeven point before the end of 2025, with analysts worried they won’t make it.
BEAM-101 seems to be competitive with approved sickle cell treatments, William Blair analysts said in a note to investors, but a patient death underscores the need for less-toxic preconditioning treatments.
In the wake of Pfizer’s voluntary market withdrawal of the popular sickle cell disease therapy, BioSpace looks at five investigational drugs currently making their way through the pipeline.
While ex vivo genome editing results in highly effective cell therapies, it can lead to off-target effects. Caribou Biosciences has come up with a novel approach for potentially more precise gene editing compared to all-RNA guides.
Since its inception in 1992, the FDA’s accelerated approval pathway has helped shepherd nearly 300 new drugs to the market. However, recent years have seen a number of high-profile market withdrawals and failed confirmatory trials.
Bristol Myers Squibb wins approval for the first novel schizophrenia drug in decades; Pfizer pulls Oxbryta from the market; new IVF and abortion laws could derail women’s health research; Roche touts CDK inhibitor deal and obesity pipeline and BioSpace heads to Meeting on the Mesa.
Pfizer’s sudden market withdrawal of sickle cell therapy Oxbryta, which some analysts predicted would reach $750 million in sales by the end of the decade, has left patients and healthcare providers with few options, while investors question the pharma giant’s dealmaking prowess.
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