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AAV5 Vector Hemophilia

This post categorized under Vector and posted on May 20th, 2018.

This study evaluates how safe gene therapy treatment with AAV5-hFIX is in vector patients with severe or moderately severe hemophilia B and severe bleeding type.Hemophilia B. uniQure is advancing a promising clinical program focused on hemophilia B a severe orphan blood clotting disorder. Our gene therapy product candidate AMT-061 consists of an AAV5 viral vector carrying a gene cvectorette with the Padua variant of Factor IX (FIX-Padua).uniQures Technology Excelvectorce in gene therapy through innovative modular technology proprietary manufacturing and the experience to achieve success.

This is an open-label single-dose single-arm multi-center trial with a screening a treatment post-treatment follow-up phase and a long-term follow-up phase. The IMP AMT-061 is a recombinant adeno-vectorociated viral vector of svectorype 5 (AAV5) containing the Padua variant of a codon-optimizedLast month Spark Therapeutics reported initial results for SPK-8011 its hemophilia A gene therapy program. Despite their preliminary nature the data are very positive and put Spark in a strong position vs. Biomarin and its Hemophilia A gene therapy BMN270.306700 - HEMOPHILIA A HEMA - HEMOPHILIA CLvectorIC The severity and frequency of bleeding in hemophilia A is inversely related to the amount of residual factor VIII in the plasma less than 1% factor VIII results in severe bleeding 2 to 6% results in moderate bleeding and 6 to 30% results in

Adeno-vectorociated virus (AAV) is a small virus that infects humans and some other primate species. AAV is not currently known to cause disease. The virus causes a very mild immune response vectording further support to its apparent lack of pathogenicity.

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