Voyager Therapeutics Reports Third Quarter 2021 Financial and Operating Results License option agreement with Pfizer provides Voyager $30M up front and up to $600M in potential exercise fees and milestones payments plus

Voyager Therapeutics Reports Third Quarter 2021 Financial and Operating Results

License option agreement with Pfizer provides Voyager $30M up front and up to $600M in potential exercise fees and milestones payments plus royalties in exchange for access to TRACER novel capsids for use with two Pfizer transgenes in neurologic and cardiovascular disease

TRACER platform identifies AAV9 and AAV5 capsid variants with broad CNS tropism and neuronal and glial cell targeting in non-human primates

Preclinical data demonstrate GBA1 gene therapy achieved widespread CNS distribution and sustained correction of GCase activity in multiple brain regions in mice

CAMBRIDGE, Mass., Nov. 2, 2021 -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a gene therapy company developing life-changing treatments and next-generation adeno-associated virus (AAV) technologies, today reported third quarter 2021 financial and operating results.

"We've taken notable steps in recent months to execute on our new strategy, including our recent capsid licensing agreement with Pfizer, continued progress with our RNA-driven AAV TRACER screening platform, and initial data from our GBA1 gene replacement program," said Michael Higgins, interim CEO of Voyager. "The licensing transaction with Pfizer showcases TRACER's ability to produce not only enhanced blood-brain barrier penetrant capsids, but also novel capsids with enhanced tropism for cardiac muscle, offering promise to unlock the fullest potential of gene therapies for a wide array of diseases. We've retained all rights to our capsid library outside of their use with two particular Pfizer transgenes, and we believe there is significant opportunity for similar transactions, leveraging the entirety of our libraries for other targets inside and outside CNS."

"We're pleased to also announce that, in addition to our prior results with AAV9 variants, our TRACER screening campaigns have identified AAV5 capsid variants with enhanced brain and spinal cord tropism compared to conventional AAV5 and AAV9, as well as a new class of AAV9 variants selective for glial cells, which may enable more precise targeting of CNS diseases affecting non-neuronal cells," said Glenn Pierce, M.D., Ph.D., interim CSO of Voyager. "In parallel, we're advancing an innovative gene therapy pipeline, leveraging our novel TRACER capsids. Preclinical data from our GBA1 gene replacement program illustrates the potential of a single-dose, IV administered therapy to

significantly raise target protein levels in the CNS, potentially impacting Parkinson's disease, Lewy body dementia, and Gaucher disease."

Pfizer License Option Agreement Supports TRACER Platform Potential for Multiple Therapeutic Areas

TRACER Identifies AAV9 and AAV5 Capsid Variants with Broad CNS Tropism, Neuronal and Glial Cell Targeting; Screening Campaigns Advancing

Positive Preclinical Data from GBA1 Program

Upcoming Events and Presentations

Third Quarter 2021 Financial Results

About the TRACER AAV Capsid Discovery Platform

Voyager's TRACER system is a broadly applicable, RNA-based functional screening platform that allows for rapid in vivo evolution of AAV capsids with enhanced tropisms and cell- and tissue-specific transduction properties in multiple species, including non-human primates (NHPs). Initial data from the first of many libraries screened in NHPs demonstrated the proprietary capsid variants effectively penetrated the blood-brain barrier and achieved widespread biodistribution and transduction of multiple regions of the brain. Separate results have demonstrated the ability of certain capsids to transduce cardiac muscle and to de-target the dorsal root ganglia. Voyager is proceeding with additional capsid campaigns derived from unique capsid serotypes to identify novel AAV vectors optimized for specific therapeutic applications.

About Voyager Therapeutics

Voyager Therapeutics (Nasdaq: VYGR) is leading the next generation of AAV gene therapy to unlock the potential of the technology to treat devastating diseases. Proprietary capsids born from the Company's TRACER screening platform are powering a rich early-stage pipeline of new and second-generation programs and may elevate the field to overcome the limitations of conventional gene therapy vectors across neurologic disorders and other therapeutic areas.

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Forward-Looking Statements

This 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 Voyager's ability to continue to identify and develop proprietary capsids from its TRACER AAV screening platform; Voyager's ability to identify and develop proprietary capsids from its TRACER AAV screening platform with increased transgene expression, increased blood-brain barrier penetration and increased biodistribution compared to conventional AAV5 and AAV9 capsids; Voyager's ability to utilize its novel proprietary capsids in its own product development programs; Voyager's ability to attract parties to license its novel proprietary capsids or to participate with Voyager in research and development collaborations utilizing its novel proprietary capsid; Voyager's ability to advance its AAV-based gene therapy programs; Voyager's ability to perform its obligations under its license option agreement with Pfizer; Voyager's entitlement to receive upfront, milestone and royalty based fees from Pfizer under the license option agreement; Voyager's ability to maintain its current partnerships and collaborations and to enter into new partnerships or collaborations; and Voyager's ability to generate sufficient cash resources to enable it to continue to identify and develop proprietary capsids from its TRACER AAV screening platform 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. 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, the severity and length of the COVID-19 health crisis, the imposition of governmental controls and guidance addressing the COVID health crisis, and the financial and human resources available to Voyager to manage the COVID-19 health crisis; the continued development of various technology platforms, including Voyager's TRACER platform; Voyager's scientific approach and general development progress; the ability to attract and retain talented contractors and employees, including key scientists and business leaders; the ability to create and protect intellectual property; the sufficiency of cash resources; the possibility or the timing of the exercise of development, commercialization, license and other options under the Pfizer license option agreement and other collaborations; the ability of Voyager to negotiate and complete licensing or collaboration agreements on terms acceptable to Voyager and third parties; and the availability or commercial potential of Voyager's product candidates.

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 the 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.

Scott Santiamo ssantiamo@vygr.com

Selected Financial Information

($-amounts in thousands, except per share data)