Europe approves first gene therapy for treatment of haemophilia A
European approval of BioMarin’s ROCTAVIAN (valoctocogene roxaparvovec) one-time gene therapy represents a breakthrough in the treatment of adults with severe haemophilia A.
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European approval of BioMarin’s ROCTAVIAN (valoctocogene roxaparvovec) one-time gene therapy represents a breakthrough in the treatment of adults with severe haemophilia A.
In this article, Recipharm’s Joe Neale explores how learnings from the COVID-19 pandemic can be applied in the future and asks how nasal administration could help our fight against tomorrow’s outbreaks.
A new approach leveraging high-yield CRISPR could make it easier to re-engineer massive quantities of cells for therapeutic applications.
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Antibody-drug conjugates (ADCs) are designed to be highly-targeted therapies with the potential to maximise the potency of a treatment while reducing unwanted side effects on healthy tissues. Some 20 years after the first ADC product approval, Dominik Schumacher and Jonas Helma-Smets of Tubulis GmbH discuss how improved and optimised technologies…
In EPR Issue 4, experts discuss implementing a global strategy to improve the sustainability of endotoxin testing, how to accelerate timelines for manufacture of multi-specific antibodies and the potential of oncolytic viruses as a therapy for glioblastoma. Also featured: progress towards smart vaccine manufacturing, an update on edible anticounterfeiting technology,…
Securing 18 million doses of GSK’s RTS,S malaria vaccine, UNICEF calls the contract a major breakthrough for child health.
bluebird bio’s Zynteglo (betibeglogene autotemcel), a one-time treatment for transfusion-dependent beta-thalassemia, will roll out with a $2.8mn price tag per dose.
SGS’s CDMO Quay Pharma awarded UK’s highest accolade for business success in overseas sales, Queen's Award for Enterprise: International Trade.
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Phase II results show a high intracranial response rate in breast cancer patients with active brain metastases treated with trastzumab deruxtecan (T-Dxd), an antibody-drug conjugate (ADC).