First-in-Human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A – BAY 2599023 has Broad Patient Eligibility and Stable and Sustained Long-Term Expression of FVIII
Highlights From the 62nd ASH Annual Meeting and Exposition

First-in-Human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A – BAY 2599023 has Broad Patient Eligibility and Stable and Sustained Long-Term Expression of FVIII

Steven W. Pipe, MD1, Francesca Ferrante, MD2, Muriel Reis3, Sara Wiegmann4, Claudia Lange5, Manuela Braun5, and Lisa A. Michaels6

1Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, MI

2Bayer, Basel, Switzerland

3Bayer, Sao Paulo, Brazil

4Bayer, Wuppertal, Germany

5Bayer, Berlin, Germany

6Bayer, Whippany, NJ

Key Data Points

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In an ongoing phase 1/2, open-label, dose-finding study, eligible patients have been enrolled sequentially into three dose cohorts (0.5, 1, and 2 x 1013 gc/kg) to receive a single intravenous infusion of BAY 2599023, with a minimum of two patients per dose. Patients will be followed for a total of 5 years to evaluate the safety and efficacy.

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This figure summarizes the FVIII response (using the chromogenic assay) for patients receiving a single infusion of BAY 2599023 up to the data cutoff of 26 October 2020. The 6 patient dosed in the 3 cohorts have shown dose-dependent, sustained FVIII expression over 40–80 weeks of follow-up.

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