Voyager Demonstrates ALPL Receptor-Mediated Blood-Brain Barrier Transport of Novel AAV Capsids in Molecular Therapy Publication

Core Insights - Voyager Therapeutics has published data demonstrating the ability of alkaline phosphatase (ALPL) to transport a novel AAV capsid across the blood-brain barrier (BBB) [1] - The research indicates that the novel AAV capsid VCAP-102 shows a 20- to 400-fold increase in gene transfer across multiple brain regions compared to AAV9 in both rodents and non-human primates [2] - The company is advancing two gene therapy programs towards IND filings this year with a partner, leveraging the findings from the ALPL research [2] Group 1: Research and Development - The publication in Molecular Therapy outlines the generation of VCAP-102 and identifies ALPL as the primary receptor for crossing the BBB [2] - Voyager's next-generation capsids have demonstrated significant transduction rates, with up to 98% of dopaminergic neurons in the substantia nigra being transduced [2] - The TRACER™ capsid discovery platform enables rapid discovery of novel AAV capsids, facilitating gene therapy for neurological diseases [3] Group 2: Strategic Partnerships and Pipeline - Voyager's pipeline includes programs targeting Alzheimer's disease, Friedreich's ataxia, Parkinson's disease, and ALS, among others [4] - The company collaborates with partners such as Alexion, AstraZeneca Rare Disease, Novartis Pharma AG, and Neurocrine Biosciences to advance its gene therapy programs [4] - The multi-modality approach of combining viral and non-viral delivery methods is seen as critical for addressing unmet needs in neurological diseases [3]