Nature of business	9 Months Ended
Sep. 30, 2022
Nature of business
Nature of business	1. Nature of business Voyager Therapeutics, Inc. (the “Company”) is a biotechnology company dedicated to breaking through barriers in gene therapy and neurology. The Company focuses on leveraging its expertise in capsid discovery and neuropharmacology to address the delivery hurdles that have constrained the gene therapy and neurology disciplines, with the goal of either halting or slowing disease progression or reduce symptom severity, therefore providing clinically meaningful impact to patients. The Company’s gene therapy platforms enable it to engineer, optimize, manufacture and deliver its adeno-associated virus (“AAV”) based gene therapies that it believes have the potential to safely provide durable efficacy. The Company’s team of experts in the field of AAV gene therapy and neuroscience first identifies and selects diseases in which the Company believes an AAV gene therapy or other biological therapy will answer a high unmet medical need, be supported by target validation, offer an efficient path to human proof of biology, present robust preclinical pharmacology, and offer strong commercial potential. The Company then engineers and optimizes an AAV vector or other biological therapy for activity in, efficacy in, or delivery to, the targeted tissue or cells. The Company is identifying proprietary AAV capsids, the outer viral protein shells that enclose genetic material that makes up the vector payload. The Company’s team has developed a proprietary AAV capsid discovery platform called TRACER^TM (Tropism Redirection of AAV by Cell Type-Specific Expression of RNA) that employs directed evolution to facilitate the selection of AAV capsids with enhanced tissue delivery characteristics, such as more effective delivery across the blood brain barrier (“BBB”). The TRACER discovery platform is a broadly applicable, functional RNA-based AAV capsid discovery platform that allows for rapid in vivo evolution of AAV capsids with cell-specific transduction properties in multiple species, including non-human primates. The Company believes that the capsids it discovers through its TRACER discovery platform (“TRACER Capsids”) have the potential to significantly enhance the efficacy and safety of its single dose gene therapies, which it expects to be delivered with targeted surgical delivery or systemic infusions, as compared with conventional capsids. In addition to leveraging TRACER capsids in potential licensing arrangements, the Company is advancing its own proprietary pipeline of drug candidates for neurological diseases. The Company's three prioritized pipeline programs include: GBA1 gene therapy for Parkinson’s disease, SOD1 gene therapy for amyotrophic lateral sclerosis (ALS), and an anti-tau antibody for Alzheimer’s disease. The Company plans to identify lead development candidates for all three programs between the fourth quarter of 2022 and the first half of 2023, with investigational new drug (IND) filings expected in 2024 and 2025. In addition to these three lead programs, the Company has a collaboration ongoing with Neurocrine Biosciences, Inc. (“Neurocrine”) to develop a gene therapy for Friedreich's ataxia, which Neurocrine is funding through Phase 1. At that point, the Company has an option to either (1) co-develop and co-commercialize the asset with Neurocrine in the United States under a 60/40 cost and profit-sharing arrangement, 60% to Neurocrine and 40% to the Company, or (2) retain the right to receive milestone payments and royalties based on global sales pursuant to the full global commercial rights granted to Neurocrine. The Company’s business strategy focuses on discovering, developing, manufacturing and commercializing its gene therapy and other biological therapy programs. As part of this strategy, the Company has developed core competencies specific to AAV gene therapy and antibody development and manufacturing. This business strategy also includes business development activities that may include in-licensing activities or partnering certain programs in specific geographies with collaborators, as the Company has demonstrated through its ongoing collaboration with Neurocrine under a collaboration agreement that became effective in January 2019 (the “Neurocrine Collaboration Agreement”), or out-licensing activities including option and license agreements related to the TRACER capsids such as the Company’s October 2021 option and license agreement with Pfizer Inc. (“Pfizer”), pursuant to which Pfizer exercised an option effective as of September 30, 2022, and the Company’s March 2022 option and license agreement with Novartis Pharma AG (“Novartis”). The Company believes there is an ongoing opportunity for option and license transactions related to the TRACER capsids. To maximize the potential of TRACER capsids for the Company’s own programs and option and license transactions, the Company has retained to date, and expects to retain in the future, all rights associated with such TRACER capsids other than the rights specific to their use in combination with a particular optionee’s or licensee’s transgenes. The Company is devoting substantially all of its efforts to product research and development, market development, and raising capital. The Company is subject to risks common to companies in the biotechnology and gene therapy industries, including but not limited to, the need to obtain sufficient capital to continue to fund its operations, risks of failure of preclinical studies and clinical trials, the need to obtain marketing approval for its product candidates, the need to successfully commercialize and gain market acceptance of its product candidates, dependence on key personnel, protection of proprietary information and technology, protection against data breaches and other cybersecurity threats, compliance with government regulations, development by competitors of technological innovations, and ability to transition from pilot-scale manufacturing to large-scale production of products. The Company has a history of incurring annual net operating losses. As of September 30, 2022, the Company had an accumulated deficit of $369.9 million. The Company has not generated any product revenue and has financed its operations primarily through public offerings and private placements of its equity securities and funding from fees, milestone payments, and cost reimbursements associated with its prior collaborations with Sanofi Genzyme Corporation (“Sanofi Genzyme”) and AbbVie Biotechnology Ltd and AbbVie Ireland Unlimited Company (collectively, “AbbVie”), and its ongoing collaboration with Neurocrine, its option and license agreement with Pfizer, and its option and license agreement with Novartis. As of September 30, 2022, the Company had cash, cash equivalents, and marketable securities of $131.6 million. Based upon its current operating plan, the Company expects that its existing cash, cash equivalents, and marketable securities at September 30, 2022, together with amounts expected to be received as reimbursement for development costs under the Neurocrine Collaboration Agreement, and the Pfizer option exercise payment of $10.0 million received in the fourth quarter of 2022 will be sufficient to meet the Company’s planned operating expenses and capital expenditure requirements into 2024. There can be no assurance that the Company will be able to obtain additional debt or equity financing on terms acceptable to the Company or generate product revenue or revenue from collaboration partners, on a timely basis or at all. The failure of the Company to obtain sufficient funds on acceptable terms when needed could have a material adverse effect on the Company’s business, results of operations, and financial condition.

Nature of business

9 Months Ended

Sep. 30, 2022

1. Nature of business

Voyager Therapeutics, Inc. (the “Company”) is a biotechnology company dedicated to breaking through barriers in gene therapy and neurology. The Company focuses on leveraging its expertise in capsid discovery and neuropharmacology to address the delivery hurdles that have constrained the gene therapy and neurology disciplines, with the goal of either halting or slowing disease progression or reduce symptom severity, therefore providing clinically meaningful impact to patients. The Company’s gene therapy platforms enable it to engineer, optimize, manufacture and deliver its adeno-associated virus (“AAV”) based gene therapies that it believes have the potential to safely provide durable efficacy. The Company’s team of experts in the field of AAV gene therapy and neuroscience first identifies and selects diseases in which the Company believes an AAV gene therapy or other biological therapy will answer a high unmet medical need, be supported by target validation, offer an efficient path to human proof of biology, present robust preclinical pharmacology, and offer strong commercial potential. The Company then engineers and optimizes an AAV vector or other biological therapy for activity in, efficacy in, or delivery to, the targeted tissue or cells.

The Company is identifying proprietary AAV capsids, the outer viral protein shells that enclose genetic material that makes up the vector payload. The Company’s team has developed a proprietary AAV capsid discovery platform called TRACER^TM (Tropism Redirection of AAV by Cell Type-Specific Expression of RNA) that employs directed evolution to facilitate the selection of AAV capsids with enhanced tissue delivery characteristics, such as more effective delivery across the blood brain barrier (“BBB”). The TRACER discovery platform is a broadly applicable, functional RNA-based AAV capsid discovery platform that allows for rapid in vivo evolution of AAV capsids with cell-specific transduction properties in multiple species, including non-human primates. The Company believes that the capsids it discovers through its TRACER discovery platform (“TRACER Capsids”) have the potential to significantly enhance the efficacy and safety of its single dose gene therapies, which it expects to be delivered with targeted surgical delivery or systemic infusions, as compared with conventional capsids.

In addition to leveraging TRACER capsids in potential licensing arrangements, the Company is advancing its own proprietary pipeline of drug candidates for neurological diseases. The Company's three prioritized pipeline programs include: GBA1 gene therapy for Parkinson’s disease, SOD1 gene therapy for amyotrophic lateral sclerosis (ALS), and an anti-tau antibody for Alzheimer’s disease. The Company plans to identify lead development candidates for all three programs between the fourth quarter of 2022 and the first half of 2023, with investigational new drug (IND) filings expected in 2024 and 2025.

In addition to these three lead programs, the Company has a collaboration ongoing with Neurocrine Biosciences, Inc. (“Neurocrine”) to develop a gene therapy for Friedreich's ataxia, which Neurocrine is funding through Phase 1. At that point, the Company has an option to either (1) co-develop and co-commercialize the asset with Neurocrine in the United States under a 60/40 cost and profit-sharing arrangement, 60% to Neurocrine and 40% to the Company, or (2) retain the right to receive milestone payments and royalties based on global sales pursuant to the full global commercial rights granted to Neurocrine.

The Company’s business strategy focuses on discovering, developing, manufacturing and commercializing its gene therapy and other biological therapy programs. As part of this strategy, the Company has developed core competencies specific to AAV gene therapy and antibody development and manufacturing. This business strategy also includes business development activities that may include in-licensing activities or partnering certain programs in specific geographies with collaborators, as the Company has demonstrated through its ongoing collaboration with Neurocrine under a collaboration agreement that became effective in January 2019 (the “Neurocrine Collaboration Agreement”), or out-licensing activities including option and license agreements related to the TRACER capsids such as the Company’s October 2021 option and license agreement with Pfizer Inc. (“Pfizer”), pursuant to which Pfizer exercised an option effective as of September 30, 2022, and the Company’s March 2022 option and license agreement with Novartis Pharma AG (“Novartis”). The Company believes there is an ongoing opportunity for option and license transactions related to the TRACER capsids. To maximize the potential of TRACER capsids for the Company’s own

programs and option and license transactions, the Company has retained to date, and expects to retain in the future, all rights associated with such TRACER capsids other than the rights specific to their use in combination with a particular optionee’s or licensee’s transgenes.

The Company is devoting substantially all of its efforts to product research and development, market development, and raising capital. The Company is subject to risks common to companies in the biotechnology and gene therapy industries, including but not limited to, the need to obtain sufficient capital to continue to fund its operations, risks of failure of preclinical studies and clinical trials, the need to obtain marketing approval for its product candidates, the need to successfully commercialize and gain market acceptance of its product candidates, dependence on key personnel, protection of proprietary information and technology, protection against data breaches and other cybersecurity threats, compliance with government regulations, development by competitors of technological innovations, and ability to transition from pilot-scale manufacturing to large-scale production of products.

The Company has a history of incurring annual net operating losses. As of September 30, 2022, the Company had an accumulated deficit of $369.9 million. The Company has not generated any product revenue and has financed its operations primarily through public offerings and private placements of its equity securities and funding from fees, milestone payments, and cost reimbursements associated with its prior collaborations with Sanofi Genzyme Corporation (“Sanofi Genzyme”) and AbbVie Biotechnology Ltd and AbbVie Ireland Unlimited Company (collectively, “AbbVie”), and its ongoing collaboration with Neurocrine, its option and license agreement with Pfizer, and its option and license agreement with Novartis.

As of September 30, 2022, the Company had cash, cash equivalents, and marketable securities of $131.6 million. Based upon its current operating plan, the Company expects that its existing cash, cash equivalents, and marketable securities at September 30, 2022, together with amounts expected to be received as reimbursement for development costs under the Neurocrine Collaboration Agreement, and the Pfizer option exercise payment of $10.0 million received in the fourth quarter of 2022 will be sufficient to meet the Company’s planned operating expenses and capital expenditure requirements into 2024.

There can be no assurance that the Company will be able to obtain additional debt or equity financing on terms acceptable to the Company or generate product revenue or revenue from collaboration partners, on a timely basis or at all. The failure of the Company to obtain sufficient funds on acceptable terms when needed could have a material adverse effect on the Company’s business, results of operations, and financial condition.