Voyager Therapeutics, Inc. (NASDAQ: VYGR), a biotechnology company dedicated to breaking through barriers in gene therapy and neurology, today announced seven data presentations at the upcoming American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting, taking place May 16-20, 2023, in Los Angeles. Accepted abstracts and the full preliminary program are available on the ASGCT website.

Details for the presentations are as follows.

TRACER™ AAV Capsid Discovery Platform

  • Oral Presentation: Directed evolution of an AAV9 library identifies a capsid variant with enhanced brain tropism and liver de-targeting in non-human primates and mice following systemic administration. Tyler Moyer, Ph.D., Senior Scientist I, Novel Capsid Discovery. Thursday, May 18, 2023, 2:15 p.m. PT - 2:30 p.m. PT.
  • Scientific Symposia: Discovery and characterization of novel cross-species BBB-penetrant capsids. Brett Hoffman, Ph.D., Senior Scientist II, Novel Capsid Discovery. Wednesday, May 17, 2023, 8:00 a.m. PT – 8:25 a.m. PT.
  • Poster Presentation: Stepwise evolution of the AAV5-derived capsid VCAP-100 identifies novel variants with improved CNS transduction and liver de-targeting following systemic injection (#464). Damien Maura, Ph.D., Senior Scientist I, Novel Capsid Discovery. Wednesday, May 17, 2023, 12:00 p.m. PT - 2:00 p.m. PT.
  • Poster Presentation: An evolved AAV variant with enhanced brain and spinal cord tropism and translation across primate species (#1393). Jing Lin, Ph.D., Scientist II, Novel Capsid Discovery. Friday, May 19, 2023, 12:00 p.m. PT - 2:00 p.m. PT.

GBA-1 Gene Therapy Program

  • Poster Presentation: Development of AAV-GBA1 gene replacement therapy via single-IV-delivery with a blood brain barrier penetrant AAV capsid. (#695). Charlotte Chung, Ph.D., Associate Director, Research. Wednesday, May 17, 2023, 12:00 p.m. PT - 2:00 p.m. PT.

Technical Operations

  • Poster Presentation: Effects of transgene size on self-complementary rAAV vectors (#1626). Amanda Ibe-Enwo, Ph.D, Senior Associate Scientist I, Technical Operations. Friday, May 19, 2023, 12:00 p.m. PT - 2:00 p.m. PT.
  • Poster Presentation: rAAV produced by Sf9-baculovirus system shows different product quality profile throughout the production cycle (#394). Zeynep Guillemin. Senior Scientist I, Technical Operations. Wednesday, May 17, 2023, 12:00 p.m. PT - 2:00 p.m. PT.

About the TRACER™ AAV Capsid Discovery PlatformVoyager’s TRACER™ (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) capsid discovery platform is a broadly applicable, RNA-based screening platform that enables rapid discovery of AAV capsids with robust penetration of the blood-brain barrier and enhanced central nervous system (CNS) tropism in multiple species, including non-human primates (NHPs). TRACER generated capsids have demonstrated superior and widespread gene expression in the CNS compared to conventional AAV capsids as well as cell- and tissue-specific transduction, including to areas of the brain that have been traditionally difficult to reach. Separate results have demonstrated the enhanced ability of certain capsids to target cardiac muscle and to de-target the dorsal root ganglia. Voyager is expanding its library of AAV capsids optimized to deliver diverse therapeutic payloads to address a broad range of CNS and other diseases. As part of its external partnership strategy, Voyager has established multiple collaboration agreements providing access to its next-generation TRACER capsids to potentially enable its partners’ gene therapy programs to treat a variety of diseases.

About Voyager TherapeuticsVoyager Therapeutics (Nasdaq: VYGR) is a biotechnology company dedicated to breaking through barriers in gene therapy and neurology. The potential of both disciplines has been constrained by delivery challenges; Voyager is leveraging cutting-edge expertise in capsid discovery and deep neuropharmacology capabilities to address these constraints. Voyager’s TRACER AAV capsid discovery platform has generated novel capsids with high target delivery and blood-brain barrier penetration at low doses, potentially addressing the narrow therapeutic window associated with conventional gene therapy delivery vectors. This platform is fueling alliances with Pfizer Inc., Novartis and Neurocrine Biosciences as well as multiple programs in Voyager’s own pipeline. Voyager’s pipeline includes wholly-owned and collaborative preclinical programs in Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), Parkinson’s disease, and Friedreich’s Ataxia, with a focus on validated targets and biomarkers to enable a path to rapid potential proof-of-biology. For more information, visit www.voyagertherapeutics.com.

Voyager Therapeutics® is a registered trademark, and TRACER™ is a trademark, of Voyager Therapeutics, Inc. 

Forward-Looking StatementsThis press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as “may,” “might,” “will,” “would,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “undoubtedly,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify forward-looking statements.

For example, all statements Voyager makes regarding the participation of scientists associated with Voyager making presentations at ASGCT 2023, and the presentations of data at ASGCT 2023, the potential for capsids generated by Voyager’s TRACER capsid discovery platform to have a positive impact for gene therapy development and the treatment of patients with medical conditions; and the ability to broaden the application of Voyager’s TRACER capsid discovery platform and establish human proof-of-concept across a range of serious diseases, are forward looking statements.

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. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. Such risks and uncertainties include, among others, a decision by the organizers of ASGCT 2023 not to allow the submissions presenting Voyager research to be presented notwithstanding prior acceptance of the submissions; the ability of Voyager scientists to effectively deliver their presentations at ASGCT 2023; the continued development by Voyager of various technology platforms, including the TRACER capsid discovery platform; and Voyager’s scientific approach and general development progress.

These statements are also subject to a number of material risks and uncertainties that are described in Voyager’s most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission, as updated by its subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

ContactsInvestors:        investors@voyagertherapeutics.com 

Andrew Funderburkafunderburk@kendallir.com  

Media: Trista Morrisontmorrison@vygr.com

Peg Rusconiprusconi@vergescientific.com

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