CSL Archives

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 by the European Commission, has been momentous in rare disease therapeutic research, paving the way for various gene therapies that are currently being studied. As we observed World Hemophilia Day on April 17, let us […]

April 18, 2023 - 10 minutesmins - By Roohi Mariam Peter

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 clotting protein factor IX often due to a spontaneous mutation in the F9 gene, leading to excessive bleeding. Until recently, routine prophylactic infusions of factor IX replacement therapy to maintain enough clotting factor to prevent […]

March 3, 2023 - 8 minutesmins - By Roohi Mariam Peter

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 Behring. The one-time gene therapy etranacogene dezaparvovec sold under the brand name Hemgenix is manufactured by UniQure Inc. and distributed by CSL Behring. The gene therapy is used to treat severe and moderately severe hemophilia […]

February 28, 2023 - 3 minutesmins - By Roohi Mariam Peter

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 observed on February 28, here are five recent advancements that have transformed research and boosted the scope for the development of novel therapies. FDA approves tissue-agnostic therapies for rare cancers Tissue-agnostic drug development has been […]

February 28, 2023 - 6 minutesmins - By Roohi Mariam Peter

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 recommending conditional marketing authorization (CMA) of etranacogene dezaparvovec. Etranacogene dezaparvovec is a one-time gene therapy for the treatment of appropriate adults with hemophilia B that reduces the rate of annual bleeds with a single infusion. […]

December 16, 2022 - 2 minutesmins - By Jim Cornall

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 of other approvals like Rocktavian and Upstaza, 2022 could be a turning point for the broader gene therapy field. Earlier this week, etranacogene dezaparvovec (Hemgenix) made history as the first gene therapy approved by the […]

November 24, 2022 - 7 minutesmins - By Jonathan Smith

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 one-time gene therapy for appropriate adults with hemophilia B. HEMGENIX is approved for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage or […]

November 23, 2022 - 5 minutesmins - By Jim Cornall

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 a rare kidney disorder. The news was announced today (August 21) by CSL Vifor and Travere Therapeutics Inc, and, if approved, the companies say sparsentan will address a significant unmet need in treating IgA nephropathy […]

August 22, 2022 - 3 minutesmins - By Liza Laws

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 positive top line phase 3 results were reported. Garadacimab, biotech company CSL’s investigational monoclonal antibody inhibiting factor XIIa, is being developed as a long-term preventive treatment for patients with HAE. Efficacy objectives The study met […]

August 18, 2022 - 3 minutesmins - By Liza Laws

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