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
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.
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.