Investigational Gene Therapy for Parkinson’s Disease with GBA1 Mutations
Автор: CheckRare
Загружено: 2025-01-07
Просмотров: 367
Michael Parini, CEO of Spur Therapeutics, discusses SPR301, an investigational gene therapy being developed for patients with Parkinson’s disease with GBA1 gene mutations.
Parkinson’s disease is a neurological disorder causing unintended or uncontrollable movements, such as shaking, stiffness, and difficulty with balance and coordination. Symptoms usually begin gradually and worsen over time. As the disease progresses, people may have difficulty walking and talking. They may also have mental and behavioral changes, sleep problems, depression, memory difficulties, and fatigue. Between 5% and 15% of patients with Parkinson’s disease have GBA1 gene mutations. These mutations are often associated with more severe symptoms. Mutations in the GBA1 gene are also involved in Gaucher disease.
SPR301 is an investigational gene therapy designed to deliver GCase85, a more stable version of the GCase enzyme deficient in patients with Parkinson’s disease with mutations in the GBA1 gene. In preclinical studies, successful delivery of GCase85 to critical regions of the brain with SPR301 was observed. Higher GCase activity and greater α-synuclein reduction were also observed.
Based on these results, IND-enabling studies are being initiated in the hopes of developing a phase 1/2 trial for SPR301 in 2026. Spur Therapeutics’ Parkinson’s disease program is an expansion of their work in Gaucher disease, where the investigational drug was originally developed.
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