First-in-human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A: Safety and FVIII Activity Results
Highlights from the ASGCT 23rd Annual Meeting

First-in-human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A: Safety and FVIII Activity Results

Steven Pipe1, Charles R. M. Hay2, John P. Sheehan3, Toshko Lissitchkov4, Elke Detering5, Silvia Ribeiro5, Konstantina Vanevski5

1University of Michigan, Ann Arbor, MI

2University Dept of Haematology, Manchester Royal Infirmary, Manchester, United Kingdom

3Department of Medicine, University of Wisconsin–Madison, Madison, WI

4National Specialized Hospital for Active Treatment of Haematologic Diseases, Sofia, Bulgaria

5Bayer, Basel, Switzerland

Key Data Points

Inclusion criteria and escalating dosing regimen for the phase 1/2 open-label safety and dosing-finding study for AAVhu37 capsid vector in severe hemophila A. Two patients have been enrolled in cohorts 1, 2, and 3. Results for the first 2 cohorts were available for this presentation. (PTP, previously treated patients; ED, exposure days; GC, gene copies)

FVIII activity results for the 2 patients in cohort 1 showed sustained expression of FVIII in both patients for > 12 months. At this lowest dose, 1 patient (right panel) achieved clinically meaningful FVIII levels resulting in a reduction in the number of bleeds over 12 months from 99 to 4.

FVIII activity results for the 2 patients in cohort 2 have showed sustained expression of FVIII in for > 6 months. At this next higher dose, both patients achieved clinically meaningful FVIII levels; 1 patient (right panel) has been bleed- and treatment-free for 7 months.

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