Results from B-AMAZE, A Phase 1/2 study by Freeline Therapeutics on FLT180a, was presented at the American Society of Hematology from December 11-14 at the Georgia World Congress Center in Atlanta.
Also Read - Two Years Without Bleeds? (Hemophilia A)
FLT180a is a gene therapy for hemophilia B which was studied in this "dose-finding trial." The 26-week B-AMAZE dose-finding trial consisted of cohorts where the patients held constant expression of factor IX for more than 3 years after treatment with FLT180.
Michael Parini, the CEO of Freeline Therapeutics, said, “The data presented today at ASH demonstrate that FLT180a gene therapy has the potential to deliver a durable, functional cure for hemophilia B... The data from our B-AMAZE study also informed our B-LIEVE dose-confirmation study, enabling us to identify a dose and prophylactic [preventive] immune management regimen that we believe can get and keep hemophilia B patients in the normal range of factor IX expression.”
Want to learn more about bleeding disorders? Sign up for our newsletter here! SIGN UP
Kommentare