Spur Therapeutics reports progress in gene therapy trials

LONDON - Spur Therapeutics, a portfolio company of Syncona Ltd, has announced encouraging new data from its gene therapy programs targeting Gaucher disease, GBA1 Parkinson’s disease, and adrenomyeloneuropathy (AMN). These findings were presented at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting in New Orleans, US.

The Phase I/II GALILEO-1 trial for Gaucher disease showcased that FLT201, an adeno-associated virus (AAV) gene therapy candidate, continues to provide durable enzyme expression and sustained clinical benefit. Patients treated with FLT201 maintained stable enzyme levels and showed improvements in disease biomarkers up to 21 months post-treatment. Additionally, preclinical studies have shown sustained enzyme activity in non-human primates for over 3.5 years.

In the realm of Parkinson’s disease research, Spur’s preclinical data for SPR301, another AAV gene therapy candidate, indicated enhanced enzyme expression in critical brain regions affected by the disease. This supports the potential for SPR301 to be a best-in-class treatment for Parkinson’s disease associated with GBA1 mutations.

The safety update from the Phase I/II PROPEL trial for AMN reported that SBT101, Spur’s gene therapy candidate for the condition, has been generally well-tolerated across patient cohorts. The trial’s findings contribute to the growing evidence of gene therapy’s safety profile in treating AMN.

Chris Hollowood, CEO of Syncona Investment Management Limited and Chair of Spur Therapeutics, expressed optimism about the new data and the forthcoming Phase III trial of FLT201 for Gaucher disease. He highlighted the broader pipeline’s potential, including SPR301 for GBA1 Parkinson’s disease.

Spur’s approach to gene therapy involves tailoring treatments to the unique needs of each targeted disease. Pamela Foulds, M.D., Chief Medical (TASE:BLWV) Officer at Spur, noted the strong safety and efficacy signals of FLT201 and the promising results from their Parkinson’s program. The company is preparing to initiate a Phase III trial for FLT201, bolstering confidence in its potential to significantly reduce the burden of Gaucher disease.

The reported progress by Spur Therapeutics is based on a press release statement and reflects the company’s commitment to developing transformative treatments for patients with serious diseases.

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