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

05 December 2020

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

REVIEW ABSTRACT

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

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.

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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

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