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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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STAT+: Pharmalittle: Bluebird claims big insurer will cover its sickle cell therapy; House Republicans threaten to subpoena FDA commish

STAT

million price tag is nearly $1 million higher than its rivals’, even though its own treatment has a serious safety warning about a blood cancer risk. The treatment, Lyfgenia, was approved along with another sickle cell gene therapy, Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics. But Bluebird’s $3.1

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PharmaShots Weekly Snapshots (June 05 – 09, 2023)

PharmaShots

Quell Therapeutics Signed an Exclusive Option and License Agreement with AstraZeneca to Develop, Manufacture and Commercialize Engineered Treg Cell Therapies Date: June 09, 2023 | Tags: Quell Therapeutics, AstraZeneca, Engineered Treg Cell Therapies, Type 1 Diabetes, Inflammatory Bowel Disease, Biotech, Treg cell engineering modules Astellas and the (..)

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PharmaShots Weekly Snapshots (March 13 - 17, 2023)

PharmaShots

5-Adapted Bivalent Booster to Treat COVID-19 in Children ≤5 Years Date: Mar 15, 2023 | Tags: Pfizer, BioNTech, Omicron BA.4/BA.5-Adapted 5-Adapted Bivalent Booster to Treat COVID-19 in Children ≤5 Years Date: Mar 15, 2023 | Tags: Pfizer, BioNTech, Omicron BA.4/BA.5-Adapted

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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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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.

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Zynteglo halt re-ignites viral vector safety concerns; analysts

pharmaphorum

Bluebird Bio’s decision to hit pause on the launch of Zynteglo for beta thalassaemia after two cases of cancer were seen in a clinical trial could see fears over the safety of viral vectors used to deliver gene therapies resurface. . million in Europe.