Voyager Therapeutics, Inc. Demonstrates Alpl Receptor-Mediated Blood-Brain Barrier Transport of Novel Aav Capsids in Molecular Therapy Publication

Published: 2025-05-15 07:00:00 ET VYGR

Published on 05/15/2025 at 07:00, updated on 05/15/2025 at 07:50

Voyager Therapeutics, Inc. announced the first peer-reviewed publication of data demonstrating the ability of alkaline phosphatase (ALPL) to transport a novel AAV capsid across the blood-brain barrier (BBB). The Molecular Therapy paper outlines the generation of novel, cross- species AAV capsid VCAP-102, which demonstrates 20- to 400-fold increased gene transfer across multiple brain regions relative to AAV9 in bothrodents and non-human primates (NHP), and the identification of ALPL as the primary receptor used by VCAP-102 to cross the BBB. In addition, the confirmation that the ALPL capsid family binds and demonstrates transcytosis with human ALPL in a cell barrier in vitro model suggests clinical translatability.

As previously announced, Voyager presented next-generation and stealth-capsid data at the American Society of Gene & Cell Therapy's (ASGCT) 28th annual meeting. In multiple NHP studies utilizing a variety of payloads, a single intravenous 3e13 v vkg dose of Voyager's second-generation CNS capsids transduced up to 98% of dopaminergic neurons in substantia nigra, up to 94% of motor neurons in the spinal cord, up to 66% of neurons in the thalamus, up to 43% of neurons in the motor cortex, and 87-99% of astrocytes broadly across brain regions. The use of words such as "will," "anticipated," "expect," "believe," "anticipate," "pot potential," " may," or "continue," and other similar expressions are intended to identify forward-looking statements.

For example, all statements Voyager makes regarding Voyager's ability to advance its AAV-based gene therapy programs and non-viral CNS delivery programs, including the potential for Voyager's novel TRACER capsids to achieve desired results in humans, including neuronal and glial transduction across multiple brain regions, andALPL-mediated transcytosis similar to the results demonstrated in rodents and NHPs; potential clinical translatability in humans; increased patient eligibility to receive AAV gene therapies; and expectations for advancement of gene therapy product candidates under the collaboration programs, including anticipated submission of IND filings and initiation of clinical trials in two partnered programs are forward looking. All forward-looking statements are based on estimates and assumptions by Voyager's management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain and subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. Voyager's scientific approach and program development progress, and the restricted supply and increased costs of critical research components; the development by third parties of capsid identification platforms that may be competitive to Voyager's TRACER capsid discovery platform; Voyager's ability to create and protect intellectual property rights associated with the TRACER capsid discovery platforms, the capsids identified by the platform, and development candidates for Voyager's pipeline programs; the timing, initiation, conduct and outcomes of Voyager's preclinical and clinical studies; the availability of data from clinical trials; the expectations and decisions of regulatory authorities; the availability or commercial potential of product candidates under collaborations; the success of Voyager's product candidates; the willingness and ability of Voyager's collaboration partners to meet obligations under collaboration agreements with Voyager; the possibility or the timing of Voyager's receipt of program reimbursement, development or commercialization milestones, option exercise, and other payments under collaboration agreements; the ability of Voyager's existing licensing or collaboration agreements; the ability of Enterprise to negotiate and complete licensing or collaboration agreements with other parties on terms acceptable to Voyager and the third parties; the ability of Voyager and the third parties; The ability of Voyager to negotiate and complete licensing or partnership agreements with other parties on terms suitable to Voyager and the third parties, the third parties; the potential of Voyager and the third parties.