Gene Therapy Tested for Hemophilia B

Amsterdam Molecular Therapeutics has begun a Phase I/II exploratory clinical trial with a gene therapy product for hemophilia B. The trial is an open-label dose-escalation study using a vectorgene combination developed at St. Jude Children’s Research Hospital. The hemophilia B gene therapy, administered once, will introduce the functional gene for the Factor IX protein into the patient’s liver cells with the goal to restore blood clotting functionality long-term. In pre-clinical studies, Factor IX gene therapy resulted in long-term production of Factor IX protein at a therapeutically significant level after a single administration.

If this approach is successful, the long-term efficacy of one-time administered hemophilia B gene therapy is expected to be perceived as a significant advance over the current regular dosing of recombinant Factor IX. In addition, the efficacy profile of this gene therapy is anticipated to exceed that of current therapy, as the gene therapy will lead to stable Factor IX levels, whereas recombinant protein treatment causes peaks and troughs.