8th Jul 2019 09:04
(Alliance News) - Healthcare-focused investment firm Syncona Ltd said Monday that portfolio company Freeline Therapeutics has published 12-month data from an ongoing trial in haemophilia B, and will open a new programme in haemophilia A.
Haemophilia is a genetic disorder that impairs the body's ability to make blood clots. Haemophilia A occurs due to not having enough clotting factor VIII, and haemophilia B occurs from lacking enough clotting factor IX.
Freeline, which develops curative gene therapies for chronic systemic diseases, said that the phase 1/2 trial known a B-AMAZE, has the goal of normalising the human factor IX activity in patients with moderate and severe haemophilia B.
Using Freeline's platform technology known as its adeno-associated viral capsid on two patients at the lowest study dose, both of their liver enzymes remained within the normal range at all time points, with the transgene expression also remaining stable.
A capsid is the protein shell of a virus, which functions to protect the nucleic acid from digestion by enzymes, and allows the virus to attach itself to a host cell.
Following treatment, both patients have been free on spontaneous bleeding episodes, and has not required any factor IX supplementation.
Freeline will continue to enrol patients in the trial, in order to optimise the dose to express factor IX in the range of 50% to 150%, which would free from the disease's symptoms.
In addition, Freeline has launched a new programme known as FLT210 to target haemophilia A, which will use its AAVS3 capsid, and is expected to soon enter investigational new drug enabling studies.
"We are pleased to report updated data on the first two patients in our haemophilia B trial. The results provide early validation for the potential durability and stability of our therapy at the lowest dose cohort. We continue to progress dose optimisation in additional patients and look forward to providing a further update as we seek to develop a functional cure for people with haemophilia B," said Freeline Executive Chair Chris Hollowood.
Shares in Syncona were down 1.1% at 22.08 pence on Monday.
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