Positive initial clinical data from the B-LIEVE dose-confirmation trial for FLT180a in Haemophilia B presented at the International Society on Thrombosis and Haemostasis Congress

Freeline Therapeutics today announced the presentation of safety and initial efficacy data from the first cohort of the Phase 1/2 dose-confirmation B-LIEVE trial for FLT180a, the company’s AAVS3-based gene therapy candidate for people with haemophilia B, at the International Society on Thrombosis and Haemostasis (ISTH) Congress being held in London, July 9-13, 2022.

As of the data cut-off of May 23, 2022, a one-time FLT180a dose of 7.7e11 vg/kg generated a rapid increase of coagulation factor IX (FIX), reaching levels in the normal range (93, 92 and 80 IU/dL) for the three patients in cohort 1 through days 77, 56 and 36, respectively. Patients stopped FIX prophylaxis and did not require FIX replacement or experience bleeding following treatment with FLT180a.

Read the full story on Freeline’s website.