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Spur Therapeutics Unveils SPR301: A Novel Gene Therapy for GBA1-Linked Parkinson’s Disease

NextEdge Admin

04 Dec 2024

2 min 32 sec

Spur Therapeutics announced SPR301 as its lead gene therapy candidate for Parkinson's disease linked to GBA1 mutations. These mutations impair glucocerebrosidase (GCase) enzyme function, causing toxic α-synuclein buildup and neuronal death, hallmarks of Parkinson’s disease.

SPR301 employs an AAV9 capsid to deliver GCase85, a rationally engineered, more stable version of GCase. Preclinical studies demonstrated SPR301's ability to deliver GCase85 to critical brain regions, achieving higher GCase activity and greater α-synuclein reduction than AAV9 constructs delivering wildtype GCase. SPR301 showed broader enzyme distribution, efficient delivery to the substantia nigra, and dose-dependent substrate reduction. Its wider therapeutic window suggests greater efficacy with improved safety compared to existing approaches.

“GBA1 mutations significantly increase Parkinson’s risk and lead to severe symptoms and earlier onset,” said Henning Stennicke, PhD, Spur's CSO. “By delivering stable GCase85 to the brain, SPR301 could alter disease progression and improve outcomes for patients with GBA1 Parkinson’s.”

Michael Parini, Spur’s CEO, highlighted SPR301 as a pivotal step in their mission to advance transformative gene therapies. Based on promising preclinical data, Spur plans to initiate IND-enabling studies to support a Phase 1/2 trial in 2026.

SPR301 builds on Spur’s expertise in Gaucher disease, which also stems from GBA1 mutations. GCase85 was developed during their work on FLT201, a gene therapy now in clinical trials for Gaucher disease. Data from FLT201 trials show substantial substrate reductions, clinical benefit, and favorable safety.

By tailoring SPR301 for brain delivery, Spur aims to address the unmet needs of GBA1 Parkinson’s, offering a novel approach to managing and potentially curing the disease.