This segment focused on the research and development of gene therapies for Parkinson's disease patients with GBA1 mutations (PD-GBA). The primary technology utilized was AAV9-based gene therapy, designed to deliver therapeutic genes to the central nervous system. Research and development activities included preclinical studies, clinical trials (such as the Phase 1/2 trial of PR001), and manufacturing process development. The goal was to provide a disease-modifying treatment to improve motor and non-motor symptoms, and slow or halt disease progression. Market positioning was as a pioneer in gene therapy for Parkinson's, with a competitive advantage in its proprietary AAV9 platform. Future opportunities included expanding the application of the technology to other synucleinopathies. Regulatory and clinical aspects involved FDA approval processes and rigorous clinical trial designs. This segment was a key focus area before the acquisition by Eli Lilly.

This segment concentrated on developing gene therapies for neuronopathic Gaucher disease (nGD), a rare lysosomal storage disorder. The company's approach involved using AAV9 vectors to deliver therapeutic genes to the central nervous system. Research and development activities included preclinical studies, clinical trials (such as the Phase 1/2 trial of PR001), and manufacturing process development. The aim was to provide a disease-modifying treatment to improve neurological symptoms and slow disease progression. Market positioning was as a leader in gene therapy for rare diseases, with a competitive advantage in its proprietary AAV9 platform. Future opportunities included expanding the application of the technology to other lysosomal storage disorders. Regulatory and clinical aspects involved FDA approval processes and rigorous clinical trial designs. This segment was a key focus area before the acquisition by Eli Lilly.

This segment was dedicated to the development of gene therapies for frontotemporal dementia patients with GRN mutations (FTD-GRN). The company's approach involved using AAV9 vectors to deliver therapeutic genes to the central nervous system. Research and development activities included preclinical studies, clinical trials (such as the Phase 1/2 PROCLAIM trial evaluating PR006), and manufacturing process development. The goal was to provide a disease-modifying treatment to improve cognitive function and slow disease progression. Market positioning was as a pioneer in gene therapy for FTD, with a competitive advantage in its proprietary AAV9 platform. Future opportunities included expanding the application of the technology to other forms of dementia. Regulatory and clinical aspects involved FDA approval processes and rigorous clinical trial designs. This segment was a key focus area before the acquisition by Eli Lilly.