Early clinical data raise the bar for hemophilia gene therapies


999 the response to two months because most recipients had pre-existing neutralizing antibodies to the virus. To overcome the immunity hurdle, the St. Jude–UCL collaborators placed the AAV2 sequences in a capsid from the less prevalent AAV8 strain. A single intravenous infusion of this vector restored factor IX expression in patients to sustained levels in the range of 1–6% of normal values with no toxic effects after three years, on average (N. Engl. J. Med. 371,1994–2004, 2014). At the highest dose, four out of six patients initially experienced a transient increase in liver transaminase levels, a marker of liver damage, but this was managed successfully with corticosteroid treatment. Nathwani is continuing to follow the patients from this landmark academic trial (which was supported by grants from the US and UK governments along with various charitable organizations), while also preparing to launch another phase 1/2 trial for hemophilia B with a next-generation AAV vector through his London-based gene therapy startup, Freeline Therapeutics. Also in phase 1/2 testing with therapies for hemophilia B are Spark, uniQure and Dimension Therapeutics; Sangamo BioSciences expects to join them before the Early clinical data raise the bar for hemophilia gene therapies

DOI: 10.1038/nbt1016-999

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@article{Dolgin2016EarlyCD, title={Early clinical data raise the bar for hemophilia gene therapies}, author={Elie Dolgin}, journal={Nature Biotechnology}, year={2016}, volume={34}, pages={999-1001} }