Hemophilia

FDA approves first gene therapy for hemophilia B

24-Nov-2022 By Rachel Arthur

CSL’s Hemgenix (etranacogene dezaparvovec-drlb) becomes the first treatment to offer patients freedom from regular ongoing infusions.

Novo Nordisk advancing hemophilia drug along regulatory pathway

12-Jul-2022 By Jane Byrne

Novo Nordisk has announced positive results from a Phase 3 study assessing the efficacy and safety of concizumab, its prophylactic treatment of hemophilia A or B with inhibitors.

Charles River and ASC Therapeutics expand gene therapy manufacturing agreement

13-Jun-2022 By Rachel Arthur

Charles River Laboratories International and ASC Therapeutics have announced plans to manufacture ASC618, a second-generation gene therapy for hemophilia A.

FDA rejection of BioMarin gene therapy raises durability questions

20-Aug-2020 By Nick Taylor

The US Food and Drug Administration (FDA) has rejected approval of BioMarin’s hemophilia A gene therapy amid durability doubts.

CSL Behring in $450m spend on hemophilia gene therapy

30-Jun-2020 By Ben Hargreaves

The company buys the global rights to a gene therapy currently going through Phase III trials.

Novo Nordisk looks to bluebird for gene therapy targets

10-Oct-2019 By Ben Hargreaves

Novo Nordisk partners with bluebird on a three-year research arrangement looking to discover gene therapy candidates for the treatment of genetic diseases, including hemophilia.