In Depth 18 Apr 2023
Gene therapy: a powerful tool for hemophilia B?
The approval of HEMGENIX, the first-ever gene therapy for hemophilia B, by the U.S. Food and Drug Administration (FDA) followed…
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Interview 3 Mar 2023
Interview: how has the first gene therapy for hemophilia B transformed research?
Hemophilia B, a rare disease that affects more than 230,000 people worldwide is caused by the lack of or defective…
News and Trends 28 Feb 2023
European Commission approves first gene therapy for hemophilia B
The European Commission has approved the first gene therapy for the treatment of hemophilia B developed by biotech company CSL…
Best in Biotech 28 Feb 2023
5 advancements in rare disease research over the past year
There are more than 7,000 rare diseases that affect hundreds of millions of people worldwide. As Rare Disease Day is…
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News and Trends 16 Dec 2022
CSL closer to gene therapy treatments for hemophilia B patients in Europe
CSL says the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion…
News and Trends 24 Nov 2022
Gene therapy approval “game changer” in hemophilia B treatment
The approval of CSL's gene therapy Hemgenix has ushered in a new era for hemophilia B treatment. With the addition…
News and Trends 23 Nov 2022
FDA approves CSL gene therapy for hemophilia B
CSL has revealed that the U.S. Food and Drug Administration (FDA) has approved HEMGENIX (etranacogene dezaparvovec-drlb), the first and only…
News and Trends 22 Aug 2022
Drug to treat rare kidney disease to be reviewed by EMA
The European Medicines Agency (EMA) has agreed to review a conditional marketing authorization (CMA) application for a drug to treat…
News and Trends 18 Aug 2022
CSL gets positive results for preventive treatment in patients with hereditary angioedema
Hereditary angioedema (HAE), a condition that causes recurrent attacks of severe swelling, could be prevented by a monoclonal antibody after…
