WebMar 9, 2024 · FLT180a was studied in B-AMAZE, a Phase 1/2 dose-finding trial in patients with severe and moderately severe hemophilia B with the goal of normalizing FIX activity in patients with moderate and severe hemophilia. Patients treated in B-AMAZE are being followed in a long-term follow-up study. WebDec 14, 2024 · FLT180a (verbrinacogene setparvovec) is an AAV gene therapy product candidate for patients with severe hemophilia B. The recently completed Phase 1/2 trial, B-AMAZE, for FLT180a has demonstrated the potential for full normalization of FIX activity and a favorable safety profile.

Gene Therapy - Hemophilia News Today

WebFeb 8, 2024 · Targeting initiation of dose confirmation trial by end of 2024 with data readout by end of 2024 Initiation of Phase 3 pivotal trial targeted by mid-2024, with data readout and BLA filing... April 14, 2024 WebJul 21, 2024 · This is thanks to FLT180a (verbrinacogene setparvovec), a gene therapy that functions to normalize levels of factor IX, a blood protein that is insufficient in patients with the condition ... slowest airplane can fly

In Consultation with FDA, Freeline Modifies Hemophilia B ... - BioSpace

WebNov 15, 2024 · As a result of a previously announced evaluation of strategic options for FLT180a, its investigational gene therapy for hemophilia B, Freeline has decided to focus its resources on FLT201 and FLT190, which have the potential to be first-in-class and/or best-in-class programs, and stop investment in further development of FLT180a without … WebAug 22, 2024 · Following administration, FLT180a results in production of FIX in the participants' liver cells which is then released into the blood stream. The aim is to have the participants' own body produce levels of FIX that allow for clotting to occur as normal as would be seen in a non-HB individual. WebJun 24, 2024 · FLT180a has been studied in B-AMAZE, a Phase 1/2 dose-finding trial in patients with severe and moderately severe hemophilia B with the goal of normalizing … slowflower