SPR301 / Spur Therapeutics - LARVOL DELTA

SPR301: Dose Dependent Reduction of Inflammation and Substrates in a Surrogate Model for GBA1-linked Neuroinflammation (ASGCT 2025) - "SPR301 at low to mid dose, ≥ 25-fold lower dose than that required for AAV9-GBA1 WT, successfully reduced CBE induced inflammation to near naïve levels in the thalamus, hypothalamus and substantia nigra and did not stimulate inflammation at the site of injection. Disease Focus of Abstract:Parkinson's Disease"

CNS Disorders • Gaucher Disease • Gene Therapies • Genetic Disorders • Inflammation • Metabolic Disorders • Movement Disorders • Parkinson's Disease • GBA • GBA1

SPR301 for GBA1 Parkinson’s: Enhanced enzyme expression in key brain regions (GlobeNewswire) - "Spur also presented preclinical data on SPR301, an AAV gene therapy candidate for a form of Parkinson’s disease characterized by mutations in GBA1, the same gene implicated in Gaucher disease...Key findings include: Superior GCase exposure in Parkinson’s-affected regions of the brain, including the substantia nigra, in GCase-deficient mice compared to wildtype GCase gene therapy, while minimizing harmful microglia activation; Broader GCase distribution than wildtype gene therapy in GCase-deficient mice, with dose-dependent substrate reduction; Superior substrate reduction compared to wildtype gene therapy, providing a therapeutic window that potentially allows for greater efficacy at much lower doses with a favorable safety profile."

Preclinical • Parkinson's Disease

Spur Therapeutics Announces Oral and Poster Presentations Highlighting Data from Clinical and Preclinical Programs at Upcoming ASGCT Annual Meeting (GlobeNewswire) - "Spur Therapeutics...announced that it will share new data from its gene therapy programs in Gaucher disease, GBA1 Parkinson’s disease and adrenomyeloneuropathy in oral and poster presentations at the American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting....The oral presentation will showcase clinical and preclinical durability data on FLT201, Spur’s adeno-associated virus (AAV) gene therapy candidate for Gaucher disease, which is expected to begin a Phase 3 trial this year."

Clinical data • New P3 trial • Preclinical • Gaucher Disease • Parkinson's Disease

Spur Therapeutics Announces Development Candidate in Parkinson’s Disease Gene Therapy Program (GlobeNewswire) - "Spur Therapeutics today announced that it has selected a lead development candidate in its gene therapy program for a genetically defined subset of Parkinson’s disease characterized by mutations in the GBA1 gene...'The preclinical data we’ve seen so far highlight SPR301’s potential to change the way we approach Parkinson’s disease. Based on these data, we are initiating IND-enabling studies to support the advancement of SPR301 into a Phase 1/2 trial in 2026.'...Preclinical data supporting the selection of SPR301 include: i) An order of magnitude higher GCase activity in vitro and in vivo compared to an AAV9 construct delivering wildtype GCase (AAV9-wtGase)...ii) Wider therapeutic window, with 25-fold lower dose showing substrate reduction equivalent to high-dose AAV9-wtGCase and higher doses showing further reductions, potentially allowing for greater efficacy at doses with a favorable safety profile."

New P1/2 trial • Preclinical • CNS Disorders • Parkinson's Disease