Syncona has committed to investing $80m in gene therapy-focused Freeline’s Series C financing round.

This builds upon Syncona’s status as the biotech’s founding investor and its previous total £123.2m ($161.5m) investment in Freeline’s Series A and B funding rounds.

Freeline is planning to use the $40m already received from Syncona to further strengthen its manufacturing process for its systemic disease gene therapies, as well as to progress its clinical programmes in haemophilia B and Fabry disease and to support the continued development of its pipeline in other diseases.

Freeline chief financial officer Brian Silver said: “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.”

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Freeline’s lead programme is adeno-associated virus (AAV) capsid gene therapy FLT-180a in Haemophilia B. In September this year, the company announced data for the first two patients in its Phase I/II dose escalation study of FLT-180a.

According to the results, the drug was well tolerated by both patients and their Factor IX activity was stable and constituent at 40%+-5.5% over 66 weeks and 74 weeks following the administration; normal levels of Factor IX should be between 50% and 150%. In addition, both patients remained free of spontaneous bleeding episodes and did not require any Factor IX supplements.

Syncona chief investment officer and Freeline executive chairman Chris Hollowood added: “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.”

Freeline’s second clinical programme is FLT-190 in Fabry Disease; the company has dosed its first patient in the Phase I/II study and claims this is the first global AAV gene therapy study for this condition. Other pre-clinical programmes Freeline is working on are in Haemophilia A and Gaucher Disease.