HEMGENIX, approved by the U.S. Food and Drug Administration (FDA) in 2022, is the first gene therapy for the treatment of adults with hemophilia B who use factor IX prophylaxis therapy or have ...

First up is Alnylam-partnered RNAi candidate fitusiran, an antithrombin-targeting agent in development as a treatment for haemophilia A and B with as few as six injections per year, making it a ...

Hemophilia B is a genetic condition that limits your blood’s ability to clot. Without prompt treatment, it can become a life threatening emergency. Creating a plan can help you better manage ...

Australian scientists created the first kangaroo embryo via IVF, a breakthrough in species conservation. Using eastern grey kangaroo specimens, the technique involved injecting a single sperm cell ...

Pfizer has said it will launch its just-approved haemophilia B gene therapy Beqvez ... after setting a price directly in line with a rival treatment. Beqvez (fidanacogene elaparvovec) was approved ...

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CRISPR-Cas systems are prokaryotic immune systems that confer resistance to foreign genetic elements such as plasmids and phages. CRISPR-Cas systems have been exploited for targeted genome editing ...