Remove tag gene-editing
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Upcoming sickle cell gene therapies cost effective at $2 million, says ICER

Pharmaceutical Technology

Two gene therapies up for approval this year for sickle cell disease could be cost effective in some cases at a $2 million price point, based on a draft evidence report published by the Institute for Clinical and Economic Review (ICER). Also known as lovo-cel, bluebird bio’s product is a lentiviral gene therapy.

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Q&A: A decade on, what’s next for CAR-T therapies?

Pharmaceutical Technology

But access to these treatments continues to remain limited due to high price tags and variable availability across regions. More broadly however, several advancements are on the horizon for cell and gene therapies in 2023. This interview has been edited for clarity and length. One challenge is in potency.

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Putting complex medicines under the microscope

European Pharmaceutical Review

By combining advanced light microscopy with gene editing technologies, it is now possible to fluorescently label subcellular compartments and gain insight into complex medicine delivery and mechanism of action. BT-474 cell line was gene-edited to incorporate a green fluorescent protein (GFP) tag into late endosomes/lysosomes (green).

Medicine 103