Spur Therapeutics Presents Positive New Preclinical Data on Its Gene Therapy Candidate for GBA1 Parkinson’s Disease at 2025 GBA1 Meeting

Data show SPR301 lowered α-synuclein, a hallmark of Parkinson’s disease, in cellular models, indicating potential for efficacy at doses with favorable safety

Demonstrated greater α-synuclein reductions compared to other gene therapy approaches

LONDON, June 06, 2025 (GLOBE NEWSWIRE) -- Spur Therapeutics today announced positive new preclinical data for SPR301, an AAV gene therapy candidate for a form of Parkinson’s disease characterized by mutations in the GBA1 gene. These data were presented at the second annual GBA1 Meeting hosted by McGill University and sponsored by The Michael J. Fox Foundation, The Silverstein Foundation, J. Sebastian van Berkom and Ghislaine Saucier, Global Parkinson’s Genetics Program and the Hilary & Galen Weston Foundation.

“GBA1 mutations are the most common genetic risk factor for Parkinson’s disease and are associated with earlier onset and more severe disease,” said Henning Stennicke, PhD, Spur’s Chief Scientific Officer. “Spur designed SPR301 to deliver a more stable version of glucocerebrosidase, or GCase, the enzyme that is deficient in individuals with GBA1 mutations. By delivering this enzyme using an AAV9 capsid, a proven gene-delivery vehicle known for its broad distribution across the brain, we believe we could provide a transformative therapy for people living with GBA1 Parkinson’s disease.”

In GBA1 Parkinson’s disease, GCase deficiency leads to the accumulation of α-synuclein and subsequent death of neuronal cells that are hallmarks of Parkinson’s. SPR301 is designed to deliver GCase85, a rationally engineered version of the GCase enzyme with enhanced stability compared to wildtype GCase. Spur originally developed GCase85 as part of its work on FLT201, the company’s Phase 3-ready gene therapy program in Gaucher disease. Data from the Phase 1/2 GALILEO-1 study of FLT201 have shown significant reductions in accumulated substrate and compelling signs of clinical benefit.

The preclinical data on SPR301 presented at the GBA1 meeting showed:

• Significant reductions of α-synuclein, a hallmark of Parkinson’s disease, in neuronal cells in vitro, demonstrating GCase85’s potential to reach cells beyond those directly treated with SPR301.
• Greater reduction of α-synuclein in cellular models of GBA1 Parkinson’s disease compared to wildtype gene therapy approaches.
• AAV9 capsid showed broad distribution across the brain when directly delivered to both hemispheres via the occipitoparietal route in non-human primates.

These data support SPR301’s best-in-class potential as a gene therapy for GBA1 Parkinson’s disease, which currently has no disease-modifying treatment available. SPR301 is currently being evaluated in additional preclinical studies to support its advancement into a Phase 1/2 clinical trial.

About Spur Therapeutics
Spur Therapeutics is a clinical-stage biotechnology company focused on developing life-changing gene therapies for debilitating chronic conditions. By optimizing every component of its product candidates, Spur aims to unlock the true potential of gene therapy to realize outsized clinical results. Spur is advancing a breakthrough gene therapy candidate for Gaucher disease, a potential first-in-class gene therapy candidate for adrenomyeloneuropathy and a preclinical gene therapy candidate for Parkinson’s disease, as well as a research strategy to move gene therapy into more prevalent diseases, including forms of dementia and cardiovascular disease. Expanding our impact, and advancing the practice of genetic medicine.

Toward life-changing therapies, and brighter futures. Toward More™

For more information, visit www.spurtherapeutics.com or connect with Spur on LinkedIn.

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