Remove tag blood-cancer
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Disc wins orphan drug tag for rare blood cancer  

Pharmaceutical Technology

The humanised monoclonal antibody DISC-3405 is under investigation in a Phase I clinical trial, with data expected this year.

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Lyfgenia and Casgevy Become First FDA-Approved Gene Therapies for Sickle Cell Disease

XTalks

Sickle cell disease is a group of inherited red blood cell disorders that affect approximately 100,000 people in the US, most of whom are Black. The disease occurs due to a point mutation in the hemoglobin beta globin ( HBB ) gene that codes for one of the proteins that make up hemoglobin, the oxygen carrier in red blood cells.

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Bluebird’s $2.8M Gene Therapy Zynteglo Wins Landmark FDA Approval for Beta Thalassemia

XTalks

Bluebird bio’s gene therapy Zynteglo (betibeglogene autotemcel, beti-cel) has been awarded a much anticipated approval from the US Food and Drug Administration (FDA) for the treatment of adult and pediatric patients with beta thalassemia who need regular blood transfusions. Zynteglo’s hefty price tag of $2.8

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After a slow start for expensive CAR-T therapies, drug developers revisit oral therapies for blood cancer

pharmaphorum

There’s been huge progress in treatments for blood cancer in recent years – but drawbacks of expensive CAR-T cell and injected antibody therapies have led drug developers to look at novel oral therapies as patient-friendly alternatives. These are particularly important in the progression of certain blood cancers.

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Beam makes USD 120M bet; GRAIL and Quest announce the collaboration; Brain organoids mimic infant’s brains; Improvement in T cells to kill cancer

Delveinsight

Electroporation is used for ex vivo delivery of therapies to blood and immune cells. GRAIL and Quest collaborate for the cancer blood test. Through this pact with GRAIL, Quest’s phlebotomy network will benefit the Galleri test in blood specimen collection throughout the U.S.

DNA 52
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Perfusion MRI and Other Perfusion Imaging Techniques for Clinical Trials

XTalks

Perfusion is the passage of fluid through the lymphatic system or the circulatory system to an organ or tissue, and it typically refers to the delivery of blood to a capillary bed in tissue. Anti-angiogenic drugs can slow the growth of tumors and sometimes even shrink tumors by preventing them from growing their own blood vessels.

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Second unanimous FDA adcomm vote boosts bluebird bio

pharmaphorum

The panel voted 13 to 0 on the question of whether beti-cel’s benefits outweighed the risks of the gene therapy in patients with beta thalassaemia who are dependent on blood transfusions, in a dream result for the biotech which just a few weeks ago was expressing doubts about its ability to continue as an operating concern.