The UK-based biotechnology company Freeline announced updates for its investigative gene therapies for hemophilia A and B at the recent XXVII Congress of the International Society on Thrombosis and Haemostasis. The Congress took place July 6-10, 2019 in Melbourne, Australia.
Freeline has two gene therapy programs. The newest, known as FLT210, was created for hemophilia A and is in its preclinical stage. FLT210 utilizes the company’s proprietary adeno-associated viral vector and its protein shell capsid, known as AAVS3, to deliver the genetic material that generates factor VIII in hemophilia A patients. According to a Freeline press release, the program is designed to provide “high and consistent expression” via a relatively low dose of the therapy.
FLT180a, the company’s hemophilia B program, is farther along in development as its investigators have 12 months’ worth of clinical data to report. FLT180a is currently in a phase 1/2 trial called B-AMAZE in which moderate and severe hemophilia B patients receive a single low dose administration of the therapy – this product also employs AAVS3 vectors to deliver the genetic payload, in this case to illicit therapeutic levels of factor IX (FIX). The first two patients who received a low dose of the therapy have exhibited FIX levels of 40.5%, plus or minus 4.5%, at 12 months post-administration. There were also no reports of spontaneous bleeding events and no scenarios that required supplemental infusions of a FIX replacement product. Investigators will continue to enroll patients in the trial with the ultimate goal of reaching FIX levels in the normal range of 50%-150%.
“We are pleased to report updated data on the first two patients in our hemophilia B trial, said Chris Hollowood, Executive Chairman of Freeline. “The results provide early validation for the potential durability and stability of our therapy at the lowest dose cohort. We continue to progress dose optimization in additional patients and look forward to providing a further update as we seek to develop a functional cure for people with hemophilia B.”
“We believe there is a significant opportunity for Freeline’s highly efficient capsid and manufacturing platform to offer gene therapies that substantially improve patients’ lives in a wide range of chronic systemic diseases, and we are seeking to leverage this same platform across hemophilia A and B, Fabry and Gaucher Diseases, and ultimately into non-monogenic disorders,” added Hollowood.
Source: Freeline press release dated July 9, 2019