This agreement secures dedicated production capacity and resources for Freeline from 2021 to 2027, inclusive. This will provide capacity for the planned Phase 2b/3 pivotal clinical trial and potential commercialisation of Freeline’s haemophilia B program, FLT180a, using the Company’s proprietary manufacturing platform and processes.
Freeline has established the manufacturing process for FLT180a at Thermo Fisher’s Cambridge facility over the past two years, most recently initiating clinical supply production there for its planned Phase 2b/3 pivotal clinical trial of FLT180a.
“I am delighted to announce this very significant extension of our partnership with Thermo Fisher,” said Jan Thirkettle, Chief Development Officer at Freeline. “The investment in, and commitment to, Thermo Fisher’s viral vector services operation gives us great confidence that they are the right partner to enable us to commercialise our haemophilia B therapy, if approved.”
“As demand for new gene therapies continues to grow, we have made strategic investments to expand our viral vector commercial manufacturing capabilities so we can provide our customers with an uninterrupted pathway from development to commercialisation. A great example of this is the work we are doing with Freeline to support its program to develop a novel treatment for haemophilia B and we are pleased to partner with them on this,” said Chris Murphy, vice president and general manager for Thermo Fisher’s viral vector services business.
Freeline is a clinical-stage biotechnology company focused on AAV-based gene therapy targeting the liver. Its vision is to create better lives for people suffering from chronic, systemic diseases using the potential of gene therapy as a one-time treatment to provide a potential functional cure. Freeline is headquartered in the U.K. and has operations in Germany and the U.S.
Haemophilia is a genetic bleeding disorder where a protein made by the body to help make blood clot is either partly or completely missing. This protein is called a clotting factor. In haemophilia A, there is a deficiency of the clotting factor VIII (eight) protein and in haemophilia B, there is a deficiency of the clotting factor IX (nine) protein. Haemophilia mainly affects boys and men; however, women can be ‘carriers’ of the affected gene and may experience symptoms. Haemophilia A is the most common type of haemophilia affecting about one in every 5,000 males, while haemophilia B affects about one in every 30,000 males. Haemophilia is classified as mild, moderate or severe, depending on the level of clotting factor VIII or IX in the blood and is diagnosed through blood tests.
The Freeline haemophilia B program, FLT180a, uses a synthetic AAVS3 capsid and a gain of function variant of human factor IX (FIX). The therapy is currently being studied in a Phase 1/2 trial with the goal of normalising FIX activity in patients with moderate and severe haemophilia.
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