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The FDA has approved the first hemophilia B gene therapy, a one-time treatment that addresses a root cause of this inherited disorder and potentially eliminates the need for regular infusions that ...
The Pennsylvania biotech company CSL Behring says it is on track to file for approval of its hemophilia B gene therapy by the end of June after releasing pivotal data showing statistical superiority ...
94 percent of patients eliminated factor IX prophylaxis and remained free of continuous prophylaxis through four years post-treatment Mean factor IX activity levels were sustained at near normal ...
The U.K.’s cost-effectiveness watchdog has tossed cold water on CSL Behring and uniQure’s pricey new hemophilia B gene therapy Hemgenix. In draft guidance, the National Institute for Health and Care ...
– Etranacogene dezaparvovec (AMT-061) is an investigational gene therapy that may potentially provide people with hemophilia B with years of functional levels of Factor IX, a blood-clotting protein ...
The FDA has accepted CSL Behring’s priority review application for a hemophilia B gene therapy, potentially fast-tracking the drug after a bumpy ride that included a clinical hold—and later, a lift.
KING OF PRUSSIA, Pa., May 6, 2021 /PRNewswire/ — Global biotherapeutics leader CSL Behring today announced the closing of its global Commercialization and License agreement with uniQure (NASDAQ: QURE) ...