Syncona Ltd on Wednesday noted positive trial results for a portfolio company’s gene therapy aimed at treating people with Gaucher’s disease.
The life science investor said investee Spur Therapeutics Ltd has presented positive new data from its phase 1/2 trial Galileo-1 study of FLT201, a novel gene therapy candidate aimed at treating Gaucher’s disease.
The trial demonstrated a favourable safety and tolerability profile in six patients treated with a single infusion of FLT201 at a certain dose, after being followed for between seven and 15 months after dosing. Durable reductions were achieved in lyso-Gb1 levels, a biomarker of clinical response in Gaucher disease. This was in patients with persistently high lyso-Gb1 levels, despite years on prior treatment with enzyme replacement therapy or substrate reduction therapy, Syncona said.
Gaucher’s disease results from the build-up of some fatty substances in organs, especially the spleen and liver. It can build up in bone tissue, weakening the bone and increasing the risk of fractures. It can affect the bone marrow and interfere with the blood’s ability to clot.
The trial results were presented at the European Society of Gene & Cell Therapy annual congress in Rome.
‘The data de-risks Spur’s technology and supports the advancement of the company’s pre-clinical pipeline into more prevalent disorders, including Parkinson’s disease. The delivery of this data at European Society of Gene & Cell Therapy is a key value inflection point for Spur, and we continue to support the company as it prepares to initiate a Phase III trial in calendar year 2025,’ said Chris Hollowood, chief executive officer of Syncona Investment Management Ltd & chair of Spur Therapeutics.
Syncona shares were 0.2% higher at 110.20 pence each on Wednesday morning in London.
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