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(Sharecast News) - Life science investor Syncona reported positive findings from its portfolio company Freeline Therapeutics on Thursday, after a phase one and two study of its gene therapy candidate for Gaucher disease, 'FLT20', showed encouraging results.
The London-listed firm said Gaucher disease, a genetic disorder stemming from GCase enzyme deficiency, saw promising outcomes from four patients who discontinued prior therapies as of 19 February.
Key highlights from the presentation included FLT201's favourable safety profile, marked by the absence of infusion reactions and severe adverse events.
Notably, robust and sustained expression of the GCase enzyme in the plasma was observed, indicating potential therapeutic efficacy.
Furthermore, significant reductions in Lyso-Gb1, a recognised biomarker of clinical response, were noted in patients with persistently high levels despite prior enzyme replacement or substrate reduction therapies.
Additionally, maintenance or improvement of haemoglobin levels and platelet counts post withdrawal of previous treatments was observed.
Emerging late-breaking data as of 8 April revealed early signs of clinical benefit. Improvement in bone marrow burden and notable alleviation of fatigue, leading to enhanced daily functioning, were observed in patients post-dosing.
"We are very encouraged by the data generated from FLT201 which demonstrates a favourable safety and tolerability profile and significant enzyme activity," said Chris Hollowood, chief executive officer of Syncona Investment Management and chair of Freeline.
"We are confident this treatment has the potential to challenge the standard of care in Gaucher disease, where current treatments are life-long and do not fully alleviate the disease.
"After a decade on ERT, 60% of patients still experience symptoms, including bone pain, lung dysfunction, enlarged organs, fatigue and low platelet counts."
Hollowood said Freeline's treatment sustained GCase expression, with data presented at ASGCT also suggesting that FLT201 could potentially transform the quality of life for patients.
"The company expects to publish further data this year, which we view as a key value inflection point, and is advancing FLT201 towards a phase three trial next year."
At 1246 BST, shares in Syncona were down 0.16% at 121.8p.
Reporting by Josh White for Sharecast.com.