Freeline, a biotechnology company focused on developing curative gene therapies for chronic systemic diseases, today announces Syncona Ltd (Syncona) has made an $80.0 million (£61.1 million) commitment in a Series C financing and has funded the first tranche of this commitment, amounting to $40.0 million (£30.6 million). This financing augments the previous £88.4 million Series B investment and the £34.8 million Series A investment previously made into Freeline by the founding investors, led by Syncona.
The first $40 million tranche will enable Freeline to expand its team, continue to develop its robust manufacturing platform, including commercial supply capability, generate further data in its clinical programmes for Haemophilia B and Fabry Disease, and continue to progress its pipeline.
Freeline is progressing FLT180a, its lead programme in Haemophilia B, through clinical development. The company has reported that the first two patients in its ongoing Phase 1/2 trial who were treated with the lowest dose have shown that Factor IX (FIX) activity remains stable and consistent at 40.5±4.5% through weeks 66 and 74, respectively, post administration. The normal range of FIX activity in the general population's blood is between 50 per cent and 150 per cent. Freeline uses a novel capsid which is capable of driving high protein expression. The company continues to enrol Haemophilia B patients as part of its dose-ranging trial. Freeline is currently seeking to identify the optimal dosing regimen to deliver FIX activity consistently in the normal range for all patients and expects to report further data on this trial in the first quarter of 2020.
Freeline has also recently dosed the first patient in its second programme in Fabry Disease, which it is estimated affects one in every 40,000 people . This programme is the first clinical stage AAV gene therapy study targeting Fabry Disease globally. The company also has disclosed pre-clinical programmes targeting Gaucher Disease and Haemophilia A, which are part of a broad pipeline of systemic disorders where high protein expression is crucial to providing functional cures for patients.
“The potential of gene therapy to change patients’ lives has never been greater, and we are delighted to have secured this Series C financing from our founding investor, Syncona. Freeline has made significant progress with two programmes in the clinic and a further pipeline of innovative products in late stage pre-clinical development. We are excited to use the proceeds of this financing to drive the progression of our pipeline, expand our manufacturing capabilities and build our commercial infrastructure.”
“Freeline has developed a high-quality gene therapy platform with a novel capsid capable of driving high expression levels to treat systemic diseases. We are encouraged by the potential product profile in our lead programme in Haemophilia B and believe Freeline has the opportunity to achieve functional cures for patients across a broad pipeline of systemic diseases. We are continuing with dose optimisation and remain on track to provide a clinical data update by financial year end.”
As of exchange rate on 18 December 2019
See footnote 1
The first cohort were treated at 4.5x10vg/kg
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