A Phase I, Open-Label, Ascending Dose Study to Assess the Safety and Tolerability of AAV2/6 Factor IX Gene Therapy via Zinc Finger Nuclease (ZFN) mediated targeted integration of SB-FIX in Adult Subjects with Severe Hemophilia B

August 02, 2018
Hematology Non Malignant
Principal Investigator: Philip Kuriakose, MD
AAV2/6 Factor IX, Zinc Finger Nuclease, Hemophilia B
Accepting Participants