Results from B-LIEVE, a Phase 1/2 Dose-Confirmation Study of FLT180a AAV Gene Therapy in Patients With Hemophilia B
Highlights From the 30th Congress of ISTH

Results from B-LIEVE, a Phase 1/2 Dose-Confirmation Study of FLT180a AAV Gene Therapy in Patients With Hemophilia B

Presented by: Guy Young, MD, Keck School of Medicine of USC, Los Angeles, California, United States

G. Young1, P. Chowdary2, S. Barton3, D. Yee3, F. Ferrante3

1Children’s Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, USA, Los Angeles, California, United States
2Royal Free Hospital, London, England, United Kingdom
3Freeline, Stevenage, England, United Kingdom

 

Key Data Points

FIX Levels

FIX levels for the first 3 patients with hemophilia B dosed with FLT180a AAV gene therapy in the phase 1/2 B-LIEVE study. FIX values are from a one-stage assay at a central laboratory; horizontal dashed line is the lower limit of normal FIX expression (50 IU/dL). As of the May 23, 2022, data cut, FIX levels were 93, 92, and 80 at study day 77, 56, and 36 for patients 1, 2, and 3, respectively.

FIX and Concomitant ALT Levels

FIX (red) and ALT (yellow) levels for the 3 patients dosed with FLT180a in the B-LIEVE study. Three weeks after FLT180a infusion, patients began a tapering course of oral corticosteroids and a short course of oral tacrolimus as prophylaxis to minimize vector-related immune responses. Vertical dotted lines are prophylactic exogenous FIX infusions received on days after FLT180a infusion.

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

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