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Syncona Ltd - London-based investor in healthcare companies - Portfolio company Spur Therapeutics Ltd announces new data from its gene therapy programmes in Gaucher disease, adrenomyeloneuropathy (AMN) and GBA1 Parkinson’s disease, presented at the annual meeting of the American Society of Gene & Cell Therapy. Highlights include updated clinical data from the phase 1/2 Galileo-1 trial and a longer-term follow-up study of FLT201 in Gaucher disease, which shows continued durable enzyme expression and sustained clinical benefit from Spur’s adeno-associated virus (AAV) gene therapy candidate. Also says that updated pre-clinical data for FLT201 demonstrates robust and sustained enzyme expression out past 3.5 years, and that a safety update from the phase 1/2 Propel trial shows that AMN gene therapy candidate SBT101 remains well-tolerated. Finally, Syncona says pre-clinical data supports the best-in-class potential of SPR301 for a form of Parkinson’s characterised by mutations in GBA1, the same gene implicated in Gaucher, with the AAV gene therapy candidate enhancing enzyme expression in key brain regions. ‘The data presented by Spur at ASGCT is very encouraging, for their lead programme in Gaucher disease but also for the broader pipeline,’ comments Chris Hollowood, chair of Spur Therapeutics and chief executive of Syncona Investment Management Ltd. ‘We look forward to the company initiating their phase 3 trial of FLT201.’ Current stock price: 87.00 pence 12-month change: down 22% Copyright 2025 Alliance News Ltd. All Rights Reserved.
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