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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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

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Supported by educational grants from Bayer, BioMarin, CSL Behring, Freeline Therapeutics Limited, Pfizer Inc., Spark Therapeutics, and uniQure, Inc.