Sunday, October 6, 2024

Gene Therapy Infusions for Hemophilia B Patients

Recently, Penn Medicine published an article discussing how in the third phase clinical trial of an international study, adults with hemophilia B who received a single infusion of gene therapy saw a 71% decrease in the number of bleeding episodes. 



Hemophilia is a type of genetic disorder that slows the blood's clotting capabilities, which in turn causes spontaneous bleeding into critical areas such as joints. More specifically, hemophilia B is a type of hemophilia where clotting factor IX is not produced enough, so gene therapy is used to start the creation of clotting factor IX in the liver.


What the study showed was days after a patient's first infusion their body started to create clotting factor IX and after a year patients had a 71% reduction in spontaneous bleeding. On top of this over 45% of patients reported not having any bleeding after gene therapy. However, the most common adversive side effect of this was an immune system attack on liver cells, which can potentially be stopped with steroid treatment. The study is expected to continue for a minimum of five total years to observe all side effects, both long and short-term.




While this breakthrough could be critical for treating patients diagnosed with hemophilia B, it is important that the study continues to monitor all side effects before making this form of treatment widespread. This study as a whole is not only impressive for genetic medicine, but could be a breakthrough in treating a plethora of other diseases that have no known treatment as of now.



https://www.pennmedicine.org/news/news-releases/2024/september/gene-therapy-is-potentially-life-changing-for-hemophilia-b

https://www.pennmedicine.org/for-patients-and-visitors/patient-information/conditions-treated-a-to-z/hemophilia

https://www.pennmedicine.org/research-at-penn/gene-therapy

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