Gene Therapy

Early safety results promising for Phase I/II trial of gene therapy treatment of hemophilia B

Published in Gene Therapy Weekly, December 23rd, 2010

2010 DEC 23 -- Investigators report no evidence of toxicity in the four hemophilia B patients enrolled to date in a gene therapy trial using a vector under development at St. Jude Children's Research Hospital and UCL (University College London) to correct the inherited bleeding disorder.

This trial was designed primarily as a safety test, with low and intermediate doses of the vector expected to produce little detectable Factor IX. The Factor IX protein helps the blood form clots. Individual with hemophilia B lack adequate levels of this clotting factor. The first participant in the open-label Phase I/II trial had an unexpectedly high level of Factor IX...

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Source: Gene Therapy Weekly (2010-12-23)

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