Investigational SPK-9001: AAV-Mediated Gene Transfer for Hemophilia B – Persistent, Stable Factor IX Activity at One Year Independent of Downstream Purification Method


Year: 2018

Language: English

Author(s): Spencer Sullivan

View Online
Additional Information

In the Tuesday morning session entitled Free Papers – Gene Therapy, Spencer Sullivan presents clinical results of FIX gene transfer using in vivo recombinant AAV delivery with the novel bioengineered vector SPA-9001, discussing vector design, FIX activity attained, and safety.