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(Alliance News) - Syncona Ltd on Friday said its portfolio company Freeline has secured further data from the ongoing B-AMAZE phase 1/2 clinical trial, investigating a novel gene therapy, FLT180a, for haemophilia B.
Freeline is a gene therapy company, focused on liver expression of proteins for a range of chronic diseases.
The FTSE 250-listed healthcare company said the data is based on the eight patients treated across four dose cohorts with FLT180a.
London-headquartered Syncona said, in the low dose cohort, stable expression of factor IX activity has been seen for over 18 months. Six patients have completed follow-up for six months, and amongst those, three have factor IX activity levels over 50%.
Normal ranges for factor levels are 50% to 150%. If the factor activity level is less than 50%, the patient may have haemophilia, but how severe the risk of bleeding is depends on what percentage.
"We believe that Freeline has developed a product that could be best-in-class for patients living with haemophilia B," said Freeline Executive Chair Chris Hollowood.
Looking ahead, Syncona said Freeline continues to enrol haemophilia B patients as part of its phase 1/2 dose-ranging trial in order to identify the optimal dose to achieve normal FIX activity levels and will provide a further update in the second half of 2020.
Syncona shares were trading 0.8% higher in London on Friday at 231.77 pence each.
By Evelina Grecenko; evelinagrecenko@alliancenews.com
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