Voyager Therapeutics published data in Molecular Therapy demonstrating the ability of alkaline phosphatase (ALPL) to transport a novel AAV capsid, VCAP-102, across the blood-brain barrier. This research highlights the company’s progress in developing gene therapies for neurological diseases using a novel method of brain delivery. The study confirms ALPL as the primary receptor used by VCAP-102 and suggests potential clinical translatability due to positive results in an •in vitro• human ALPL cell barrier model.
This breakthrough is important because effective delivery of gene therapies to the brain has been a major obstacle. Current methods often struggle to achieve sufficient penetration and distribution within the brain, limiting therapeutic efficacy. Voyager’s ALPL-mediated transport system could significantly improve the delivery of gene therapies, potentially leading to more effective treatments for a range of neurological diseases. This opens doors for treating diseases previously considered untreatable with gene therapy.
VCAP-102 demonstrated a 20- to 400-fold increase in gene transfer across multiple brain regions compared to AAV9 in preclinical models. Furthermore, Voyager’s next-generation capsids, built upon VCAP-102, have shown even greater brain transduction and reduced liver targeting in non-human primate studies, achieving up to 98% transduction of dopaminergic neurons and high transduction rates in other critical brain regions. The company is also exploring the use of ALPL and other receptors for non-viral CNS delivery methods.
This research positions Voyager as a leader in CNS gene therapy delivery. The development of next-generation and “stealth” capsids with improved brain penetration and immune-evading capabilities suggests a strong pipeline of potential therapies. The multi-modality approach, combining viral and non-viral delivery mechanisms, further strengthens Voyager’s platform and its potential to address a wide spectrum of neurological diseases. The advancement of two partnered programs toward IND filings this year underscores the potential for near-term clinical translation of this technology.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
