More than 100 years ago, scientists started unlocking the mysteries of genes and inherited traits. Today, gene therapy holds promise for conditions like hemophilia B.
Gene therapy is the first medical treatment to address underlying issues within our DNA, the genetic material inside our cells – and it is rapidly transforming formerly incurable conditions into manageable ones. Building on more than a century of scientific discovery, gene therapy is now an established form of medication with more than 10,000 individuals treated by various gene therapy products.
In 2020, CSL Behring acquired global rights to commercialize an investigational AAV-based gene therapy, which is being evaluated in the ongoing HOPE-B clinical study now in Phase 3.
Would you like to learn more about the steps on the way to developing a gene therapy for hemophilia B patients?www.haemevolution.de