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- Introduced new vectorized anti-Aβ antibody gene therapy research initiative, expanding Alzheimer’s disease portfolio - - Executed license agreement with Sangamo for prion disease treatment - - Presented validating preclinical results including increased brain transduction of IV-administered TRACER™ capsids across multiple non-human primate species at ASGCT 2023 - - Conference call at 8:30 a.m. ET today - CAMBRIDGE, Mass., Aug. 03, 2023 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to breaking through barriers in gene therapy and neurology, today reported second quarter 2023 financial and operating results. “The introduction today of Voyager’s vectorized anti-Aβ gene therapy research initiative expands our Alzheimer's disease portfolio, complementing our anti-tau antibody and tau knock-down gene therapy programs,” said Alfred W. Sandrock, Jr., M.D., Ph.D., Chief Executive Officer of Voyager. “We are encouraged by recent approvals in Alzheimer’s disease, and we believe this establishes a path forward for new approaches to existing targets, as well as new targets, to help address the heterogeneity of the massive Alzheimer’s disease population. Our anti-tau antibody VY-TAU01 remains on track for IND in the first half of 2024, and we continue to advance our growing pipeline of wholly-owned and partnered CNS gene therapies leveraging our neurotropic TRACER capsids.” Key Milestones Achieved in Q2 2023 and Subsequent Period: Launch of new vectorized anti-Aβ antibody early research initiative for Alzheimer’s disease: Voyager today announced an early research initiative investigating a gene therapy comprising a vectorized anti-Aβ antibody delivered via a TRACER capsid for the potential one-time treatment of Alzheimer’s disease. The new initiative leverages Voyager’s vectorized antibody expertise to express an anti-Aβ antibody to target amyloid plaques. Preliminary data in mice have shown vectorized antibody target engagement. This initiative further expands Voyager’s portfolio of Alzheimer’s programs including its anti-tau antibody program and siRNA tau knockdown gene therapy research initiative.Licensing agreement with Sangamo: In June, Voyager and Sangamo Therapeutics, Inc. announced a definitive license agreement for a potential treatment of prion disease. Under the agreement, Voyager provided Sangamo with access to a TRACER capsid to be combined with Sangamo’s zinc finger transcriptional regulators (ZF-TRs) designed to treat prion disease.ASGCT 2023 Conference: In May, Voyager shared seven data presentations at the American Society of Gene and Cell Therapy’s (ASGCT) Annual Meeting, including data demonstrating greater than 50% cell transduction in multiple areas of the brain at a dose of 2E12 vg/kg following intravenous administration of VCAP-102 in marmosets, as well as data further confirming the binding of VCAP-102 to a novel receptor, and progress identifying receptors to additional TRACER-derived capsid families.Advancement of anti-tau antibody program: In April, Voyager received pre-IND written feedback from the FDA for VY-TAU01, its lead humanized anti-tau antibody candidate. Voyager continues to expect to initiate GLP toxicology studies this year to enable an IND filing in the first half of 2024.Presentation of data on Huntington’s disease (HD) early research initiative: In April, Voyager presented data on the HD research initiative at the 18th Annual Huntington's Disease Therapeutics Conference held in Dubrovnik, Croatia. Voyager’s early research initiative for HD combines an intravenous TRACER capsid with vectorized siRNAs to enable specific knockdown of mutant HTT and MSH3.Key leadership additions: In May, Voyager announced the appointment of George Scangos, Ph.D., to its Board of Directors. In July, Voyager announced the appointment of Jacquelyn Fahey Sandell as Chief Legal Officer. Key Upcoming Milestones: Anti-tau antibody program for Alzheimer’s disease: Voyager continues to expect to initiate GLP toxicology studies in 2023 to support an IND in the first half of 2024.SOD1 gene therapy program for amyotrophic lateral sclerosis (ALS): Voyager expects to identify a lead development candidate in the second half of 2023 to support an IND in mid-2025.Voyager and Neurocrine Biosciences collaboration: Voyager and Neurocrine Biosciences continue to collaboratively advance the GBA1 gene therapy program for Parkinson’s disease and other GBA1-mediated diseases, as well as the FXN gene therapy program for Friedreich’s Ataxia. Second Quarter 2023 Financial Results Collaboration Revenues: Voyager had collaboration revenue of $4.9 million for the second quarter of 2023, compared to $0.7 million for the same period in 2022. The increase was primarily due to revenue recognized during the second quarter of 2023 on the 2023 and 2019 strategic collaborations with Neurocrine Biosciences.Net Loss: Net loss was $22.2 million for the second quarter of 2023, compared to $19.1 million for the same period in 2022. The difference is primarily due to the increase in operating expenses discussed in the two bullets immediately below.R&D Expenses: Research and development expenses were $22.0 million for the second quarter of 2023, compared to $12.5 million for the same period in 2022. The increase in R&D expenses was primarily a result of increased program-related spending, and increased compensation costs driven by headcount increases during the second quarter of 2023.G&A Expenses: General and administrative expenses were $8.3 million for the second quarter of 2023, compared to $7.6 million for the same period in 2022. The increase in G&A expenses was primarily a result of increased compensation costs driven by headcount increases.Cash Position: Cash, cash equivalents and marketable securities as of June 30, 2023, were $272.7 million. Six Months Ended June 30, 2023 Financial Results Collaboration Revenues: Voyager had collaboration revenue of $155.3 million for the six months ended June 30, 2023 period, compared to $1.4 million for the same period in 2022. The increase was primarily due to revenue recognized during the first quarter of 2023 on the 2023 strategic collaboration with Neurocrine Biosciences, as well as revenue recognized on the Novartis license of novel TRACER capsids.Net Income (Loss): Net income was $101.8 million for the six months ended June 30, 2023 period, compared to net loss of $40.4 million for the same period in 2022. The difference was primarily due to the revenue increases noted above.R&D Expenses: Research and development expenses were $40.6 million for the six months ended June 30, 2023 period, compared to $26.9 million for the same period in 2022. The increase in R&D expenses was primarily a result of increased program-related spend, and increased compensation costs driven by headcount increases during the 2023 period.G&A Expenses: General and administrative expenses were $17.3 million for the six months ended June 30, 2023 period, compared to $15.2 million for the same period in 2022. The increase in G&A expenses was primarily a result of increased compensation costs driven by headcount increases. Financial Guidance Voyager is committed to maintaining a strong balance sheet that supports the advancement and growth of its platform and pipeline. Voyager continues to assess its planned cash needs both during and in future periods. As communicated previously, we expect our cash, cash equivalents, and marketable securities, along with amounts expected to be received as reimbursement for development costs under the Neurocrine collaborations and interest income, to be sufficient to meet Voyager’s planned operating expenses and capital expenditure requirements into 2025. Conference Call Voyager will host a conference call and webcast today at 8:30 a.m. ET to discuss the second quarter 2023 financial and operating results. To participate via telephone and join the call live, please register in advance here. Upon registration, telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number and a unique passcode. A live webcast of the call will also be available on the Investors section of the Voyager website at ir.voyagertherapeutics.com, and a replay of the call will be available at the same link approximately two hours after its completion. The replay will be available for at least 30 days following the conclusion of the call. About the TRACER™ 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 Pharma AG, Neurocrine Biosciences, Inc., and Sangamo Therapeutics, Inc., 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 other diseases of the central nervous system, 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,” “will,” “expect,” “plan,” “believe,” “target,” “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 advance its AAV-based gene therapy programs and tau antibody program; the preclinical development of its potential development candidates; Voyager’s entitlement to receive option exercise, milestone and royalty-based fees from Novartis and Pfizer under the respective license option agreements; Voyager’s ability to advance gene therapy product candidates under the Neurocrine collaborations; Voyager’s ability to identify receptors to additional TRACER-derived capsid families; Voyager’s anticipated financial results, including the receipt by Voyager of revenues or reimbursement payments from collaboration partners; and Voyager’s cash runway and Voyager’s ability to generate sufficient cash resources to enable it to continue its business and operations 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 continued development of Voyager’s technology platforms, including Voyager’s TRACER platform and its antibody screening technology; the ability to initiate and conduct preclinical studies in animal models; 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 platform, the capsids identified by the platform, and development candidates for Voyager’s pipeline programs; the initiation, timing, conduct and outcomes of Voyager’s preclinical studies; the possibility or the timing of the exercise of development, commercialization, license and other options under the Pfizer and Novartis license option agreements and Neurocrine collaborations; the ability of Voyager to negotiate and complete licensing or collaboration agreements with other parties on terms acceptable to Voyager and the third parties; the ability to attract and retain talented directors, employees, and contractors; and the sufficiency of cash resources to fund its operations and pursue its corporate objectives. 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. 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. Contacts InvestorsInvestors@vygr.com Andrew Funderburkafunderburk@kendallir.com MediaTrista Morrisontmorrison@vygr.com Peg Rusconiprusconi@vergescientific.com Selected Financial Information($ amounts in thousands, except per share data)(Unaudited) Three Months Ended Six Months Ended June 30, June 30,Statement of Operations Items: 2023 2022 2023 2022 Collaboration revenue $4,853 $712 $155,333 $1,371 Operating expenses: Research and development 21,985 12,527 40,553 26,876 General and administrative 8,294 7,552 17,322 15,211 Total operating expenses 30,279 20,079 57,875 42,087 Operating (loss) income (25,426) (19,367) 97,458 (40,716)Total other income 3,277 280 5,141 310 (Loss) income before income taxes (22,149) (19,087) 102,599 (40,406)Income tax provision 59 — 763 — Net (loss) income $(22,208) $(19,087) $101,836 $(40,406) Net (loss) income per share, basic $(0.51) $(0.50) $2.42 $(1.06)Net (loss) income per share, diluted $(0.51) $(0.50) $2.33 $(1.06) Weighted-average common shares outstanding, basic 43,520,137 38,298,426 42,102,101 38,183,192 Weighted-average common shares outstanding, diluted 43,520,137 38,298,426 43,770,999 38,183,192 June 30, December 31, Selected Balance Sheet Items 2023 2022 Cash, cash equivalents, and marketable debt securities $272,746 $118,848 Total assets $315,536 $159,356 Accounts payable and accrued expenses $10,719 $10,382 Deferred revenue $83,049 $65,827 Total stockholders’ equity $199,026 $59,020

- Neurocrine Biosciences strategic collaboration and Novartis license option exercise demonstrate Voyager pipeline and platform value and strengthen balance sheet - - Alzheimer’s disease franchise advances: selected lead candidate for anti-tau antibody program; IND submission on track for first half of 2024; launched tau knock-down gene therapy program - - Data accepted for presentation at ASGCT 2023 validate blood-brain barrier penetration and central nervous system transduction with Voyager’s novel, intravenously administered capsids - - Conference call at 8:30 a.m. ET today - CAMBRIDGE, Mass., May 09, 2023 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to breaking through barriers in gene therapy and neurology, today reported first quarter 2023 financial and operating results. “Voyager started 2023 by securing $175 million upfront in a strategic collaboration with Neurocrine Biosciences for our GBA1 gene therapy program and three new gene therapy programs, followed by Novartis’s decision to exercise options to our novel capsids for two neurologic disease targets, triggering another $25 million payment. These transactions have strengthened our balance sheet while maintaining long-term value and enabled us to further advance our platform and pipeline,” said Alfred W. Sandrock, Jr., M.D., Ph.D., Chief Executive Officer of Voyager. “We also made important progress with multiple approaches to treating Alzheimer’s disease during the quarter, selecting a lead development candidate for our anti-tau antibody program and launching a new tau knockdown gene therapy program.” Upcoming Milestones: Pipeline update: Anti-tau antibody program for Alzheimer’s disease: Voyager continues to expect to begin IND-enabling studies in 2023 to support an IND in the first half of 2024.SOD1 gene therapy program for amyotrophic lateral sclerosis (ALS): Voyager previously announced that it expected to identify a lead development candidate for this program in the first half of 2023. The Company continues to evaluate the data from preclinical studies for this program and now expects to identify a lead development candidate in the second half of 2023.Voyager and Neurocrine Biosciences continue to collaboratively advance the GBA1 gene therapy program for Parkinson’s disease and other GBA1-mediated diseases, as well as the FXN gene therapy program for Friedreich’s Ataxia. ASGCT 2023 Conference: Voyager has announced seven data presentations at the American Society of Gene and Cell Therapy’s (ASGCT) Annual Meeting, May 16-20, 2023, in Los Angeles, including an oral presentation titled, “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.” Key Milestones Achieved in Q1 2023 and Subsequent Period: Strategic collaboration with Neurocrine Biosciences: In January 2023, Neurocrine Biosciences, Inc. and Voyager announced the formation of a new strategic collaboration to advance Voyager’s preclinical, intravenously administered GBA1 gene therapy program for Parkinson’s disease and other GBA1-mediated diseases as well as three new gene therapy programs directed to rare CNS targets. Voyager received up-front consideration of $175 million and is eligible to receive up to $1.5 billion in potential development milestone payments, up to $2.7 billion in potential commercial milestone payments, tiered royalties on net sales, program funding, and an option to elect 50/50 cost- and profit-sharing in the U.S. for the GBA1 program following Phase 1 readout. The transaction closed in February 2023.Novartis license of novel TRACER capsids: In March 2023, Voyager announced that Novartis exercised its options to license novel capsids against two undisclosed neurologic disease targets. Voyager received a $25 million option exercise payment and is eligible to receive up to $600 million in associated potential development, regulatory, and commercial milestone payments, as well as mid- to high-single-digit tiered royalties based on net sales of Novartis products incorporating the licensed capsids.Advancement of anti-tau antibody program: In January 2023, Voyager selected a lead humanized anti-tau antibody candidate, VY-TAU01, to advance for the treatment of Alzheimer’s disease. In March 2023, Voyager presented new data at the AD/PD 2023 Conference highlighting the differentiating characteristics resulting in the selection of VY-TAU01. In April 2023, Voyager received pre-IND written feedback from the FDA for VY-TAU01. Voyager continues to expect to initiate GLP toxicology studies this year to enable an IND filing in the first half of 2024.Launch of new tau knockdown gene therapy early research initiative for Alzheimer’s disease: During the first quarter of 2023, Voyager announced an early research initiative investigating a gene therapy which reduces tau expression in the brain for the treatment of Alzheimer’s disease. The program combines an siRNA tau knockdown payload with an intravenously delivered TRACER capsid.Launch of new Huntington’s disease (HD) early research initiative: Voyager announced in January 2023 the launch of an early research initiative for HD, which combines an intravenous TRACER capsid with vectorized siRNAs to enable specific knockdown of mutant HTT and MSH3. Early data were presented at the 18th Annual Huntington's Disease Therapeutics Conference held in Zagreb, Croatia, April 24 – 27, 2023.Board of Directors appointments: In May 2023, George Scangos, Ph.D., former CEO of Exelixis, Inc.; Biogen, Inc.; and most recently Vir Biotechnology, Inc.; joined Voyager’s Board of Directors. In February 2023, following the close of the collaboration with Neurocrine Biosciences, Jude Onyia, Ph.D., Chief Scientific Officer at Neurocrine Biosciences, joined the Board of Directors. In January 2023, Grace E. Colón, Ph.D., also joined the Board of Directors. First Quarter 2023 Financial Results Collaboration Revenues: Voyager had collaboration revenue of $150.5 million for the first quarter of 2023, compared to $0.7 million for the same period in 2022. The increase was primarily due to revenue recognized during the first quarter of 2023 on the 2023 Strategic collaboration with Neurocrine Biosciences, as well as revenue recognized on the Novartis license of novel TRACER capsids.Net Income (Loss): Net income was $124.0 million for the first quarter of 2023, compared to net loss of $21.3 million for the same period in 2022. The difference was primarily due to the revenue increase noted above.R&D Expenses: Research and development expenses were $18.6 million for the first quarter of 2023, compared to $14.3 million for the same period in 2022. The increase in R&D expenses was primarily a result of increased compensation costs driven by headcount increases and program-related spend increases during the first quarter of 2023. G&A Expenses: General and administrative expenses were $9.0 million for the first quarter of 2023, compared to $7.7 million for the same period in 2022. The increase in G&A expenses was primarily a result of increased compensation costs driven by headcount increases.Cash Position: Cash, cash equivalents and marketable securities as of March 31, 2023, were $273.3 million. Cash position does not include $25.0 million in option exercise payment from Novartis which was received after March 31, 2023. Financial GuidanceVoyager is committed to maintaining a strong balance sheet that supports the advancement and growth of its platform and pipeline. Voyager continues to assess its planned cash needs both during and in future periods. As communicated previously, the Company expects its cash, cash equivalents, and marketable securities, together with the $25.0 million options exercise payment received in April 2023 from Novartis, along with amounts expected to be received as reimbursement for development costs under the Neurocrine collaborations, to be sufficient to meet Voyager’s planned operating expenses and capital expenditure requirements into 2025. Conference Call Voyager will host a conference call and webcast today at 8:30 a.m. ET to discuss the first quarter 2023 financial and operating results. To participate via telephone and join the call live, please register in advance here. Upon registration, telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number and a unique passcode. A live webcast of the call will also be available on the Investors section of the Voyager website at ir.voyagertherapeutics.com, and a replay of the call will be available at the same link approximately two hours after its completion. The replay will be available for at least 30 days following the conclusion of the call. 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,” “target,” “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 anticipated participation in future conferences, Voyager’s ability to continue to identify and develop proprietary capsids from its TRACER capsid discovery platform with increased transgene expression, increased blood-brain barrier penetration and increased biodistribution compared to conventional AAV9 and AAV5 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 capsids; Voyager’s ability to advance its AAV-based gene therapy programs and tau antibody program; the preclinical development of its potential development candidates; Voyager’s ability to perform its obligations under its respective license option agreements with Pfizer and Novartis; Voyager’s entitlement to receive option exercise, milestone and royalty-based fees from Novartis and Pfizer under the respective license option agreements; Voyager’s ability to advance gene therapy product candidates under the Neurocrine collaborations into, and successfully initiate, enroll and complete, clinical trials; Voyager’s entitlement to receive milestone and royalty-based fees under the Neurocrine collaborations; Voyager’s ability to maintain its current partnerships and collaborations and to enter into new partnerships or collaborations; the continued service and suitability of recently appointed senior officers and members of the Board of Directors; Voyager’s anticipated financial results, including the receipt by Voyager of revenues or reimbursement payments from collaboration partners; and Voyager’s cash runway and Voyager’s ability to generate sufficient cash resources to enable it to continue its business and operations 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 continued development of Voyager’s technology platforms, including Voyager’s TRACER platform and its antibody screening technology; the ability to initiate and conduct preclinical studies in animal models; 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 platform, the capsids identified by the platform, and development candidates for Voyager’s pipeline programs; the initiation, timing, conduct and outcomes of Voyager’s preclinical studies; the possibility or the timing of the exercise of development, commercialization, license and other options under the Pfizer and Novartis license option agreements and Neurocrine collaborations; the ability of Voyager to negotiate and complete licensing or collaboration agreements with other parties on terms acceptable to Voyager and the third parties; the ability to attract and retain talented directors, employees, and contractors; and the sufficiency of cash resources to fund its operations and pursue its corporate objectives. 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. 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. ContactsInvestorsInvestors@vygr.com Andrew Funderburkafunderburk@kendallir.com MediaTrista Morrisontmorrison@vygr.comPeg Rusconiprusconi@vergescientific.com Selected Financial Information ($ amounts in thousands, except per share data) (Unaudited) Three Months Ended March 31,  Statement of Operations Items: 2023      2022 Collaboration revenue $ 150,480      $ 658 Operating expenses: Research and development 18,568 14,349 General and administrative 9,028 7,659 Total operating expenses 27,596 22,008 Operating income (loss) 122,884 (21,350 )Total other income, net 1,864 31 Income (loss) before income taxes 124,748 (21,319 )Income tax provision 704 — Net income (loss) $ 124,044 $ (21,319 )Net income (loss) per share, basic $ 3.05 $ (0.56 )Net income (loss) per share, diluted $ 2.94 $ (0.56 )Weighted-average common shares outstanding, basic 40,632,087 38,049,430 Weighted-average common shares outstanding, diluted 42,161,326 38,049,430 March 31,  December 31, Selected Balance Sheet Items 2023      2022Cash, cash equivalents, and marketable securities $ 273,314 $ 118,848 Total assets $ 336,294 $ 159,356 Accounts payable and accrued expenses $ 11,337 $ 10,382 Deferred revenue $ 84,552 $ 65,827 Total stockholders’ equity $ 216,961 $ 59,020

- Transformational progress in 2022 marked by reprioritization of high-value neurology pipeline and validation of TRACER™ novel capsid platform through transactions with Pfizer and Novartis - - Partnering momentum continues into 2023 with $175 million payment associated with Neurocrine Biosciences strategic collaboration followed by $25 million payment triggered by Novartis capsid license option exercise - - Development candidate selected in tau antibody program for Alzheimer’s disease; development candidate selection in gene therapy program for ALS expected in H1 2023; expected IND filings for both programs on track for 2024 - - Voyager initiates new early research initiative combining an siRNA tau knockdown payload with an intravenously delivered TRACER capsid for Alzheimer’s disease - - Conference call at 8:30 a.m. ET today - CAMBRIDGE, Mass., March 07, 2023 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to breaking through barriers in gene therapy and neurology, today reported fourth quarter and full year 2022 financial and operating results. “The past year has been transformational for Voyager,” said Alfred W. Sandrock, Jr., M.D., Ph.D., Chief Executive Officer of Voyager. “Since I became CEO of Voyager last March, we have re-prioritized our pipeline to focus on some of the most significant unmet needs in neurology, advanced our four lead programs towards the clinic, achieved breakthrough innovations in novel capsid discovery including the identification of a receptor, and entered multiple high-value partnerships. The strategic collaboration with Neurocrine that we announced in January illustrates the value of our next-generation gene therapies for CNS diseases, and this value is further evidenced by capsid license option exercises by Pfizer in October and recently by Novartis for multiple CNS targets. These partnerships strengthen our balance sheet and allow us to invest in our platform as well as existing and new neuro-genetic medicine programs.” Key Milestones Achieved in Q4 2022 and Subsequent Period: Novartis license of novel TRACER capsids: In March 2023, Voyager announced that Novartis exercised its options to license novel capsids against two undisclosed neurologic disease targets. Voyager will receive a $25 million option exercise payment and is eligible to receive up to $600 million in associated potential development, regulatory, and commercial milestone payments, as well as mid- to high-single-digit tiered royalties based on net sales of Novartis products incorporating the licensed capsids.Strategic collaboration with Neurocrine Biosciences: In January 2023, Neurocrine Biosciences, Inc. and Voyager announced the formation of a new strategic collaboration to advance Voyager’s preclinical, intravenously administered GBA1 gene therapy program as well as three new gene therapy programs directed to rare CNS targets. Voyager received up-front consideration of $175 million and is eligible for up to $1.5 billion in potential development milestone payments, up to $2.7 billion in potential commercial milestone payments, tiered royalties on net sales, program funding, and an option to elect 50/50 cost- and profit-sharing in the U.S. for the GBA1 program following Phase 1 readout.Pfizer license of novel TRACER capsid: Voyager announced in October 2022 that Pfizer, Inc. exercised its option to license a novel capsid against an undisclosed rare neurologic disease target. Voyager received a $10 million option exercise payment and is eligible for up to $290 million in milestone payments plus mid- to high-single-digit tiered royalties.Selection of anti-tau antibody lead candidate: In January 2023, Voyager selected a lead humanized anti-tau antibody candidate to advance against Alzheimer’s disease. The lead candidate, VY-TAU01, targets the C-terminal domain. VY-TAU01 was selected for its affinity, selectivity, and biophysical characteristics. Voyager expects to present additional data at the AD/PD 2023 Conference, March 28 – April 1, 2023. Process development and manufacturing at a CDMO have been initiated, and Voyager expects to initiate a GLP toxicology study later in 2023 to enable an IND filing in the first half of 2024.Launch of updated Huntington’s disease gene therapy early research initiative: In January 2023, Voyager announced the launch of an updated early research initiative for Huntington’s disease. The updated gene therapy program, which leverages the latest insights in disease biology, combines an intravenous TRACER capsid with vectorized siRNAs to enable specific knockdown of mutant HTT and MSH3.Launch of new tau knockdown gene therapy early research initiative for Alzheimer’s disease: During the first quarter of 2023, Voyager announced an early research initiative investigating a gene therapy targeting intracellular tau for the treatment of Alzheimer’s disease. The program combines an siRNA tau knockdown payload with an intravenously delivered TRACER capsid.ESGCT data characterize novel CNS receptor and demonstrate low-dose potential: At the European Society of Gene & Cell Therapy (ESGCT) 29th Annual Congress in October 2022, Voyager presented data characterizing a novel cell surface receptor identified as a binding receptor for a family of Voyager’s next-generation TRACER capsids. In a separate presentation, another TRACER capsid demonstrated high levels of central nervous system (CNS) target engagement at low doses.Board of Directors appointments: In December 2022, Voyager announced the appointment of Grace E. Colón, Ph.D., to its Board of Directors, effective January 1, 2023. Dr. Colón most recently served as President, CEO and Director of the clinical-stage biotechnology company InCarda Therapeutics, Inc. In February 2023, following the close of the collaboration with Neurocrine Biosciences, Jude Onyia, Ph.D., Chief Scientific Officer at Neurocrine Biosciences, joined Voyager’s Board of Directors. Upcoming Milestones: AD/PD 2023 Conference: Voyager will present data on its GBA1 gene therapy and anti-tau antibody programs at the AD/PD 2023 Conference occurring March 28 – April 1, 2023, in Gothenburg, Sweden. Oral presentation: Development of AAV-GBA1 gene replacement therapy via single-IV-delivery with a blood brain barrier penetrant AAV capsid. Charlotte Chung. April 1, 2023, 11:55 CET.Poster presentation: Selection of an anti-tau antibody candidate targeting pathological tau for the treatment of Alzheimer’s disease. Wencheng Liu. P0643 / #1192. Pipeline progressing: Voyager expects to identify a lead development candidate in H1 2023 for its wholly-owned SOD1 gene therapy program for amyotrophic lateral sclerosis (ALS). Voyager expects to file INDs for the SOD1 program and its wholly-owned anti-tau antibody program for Alzheimer’s disease in 2024. In addition, Voyager and Neurocrine Biosciences continue to collaboratively advance the GBA1 gene therapy program for Parkinson’s disease and other GBA1-mediated diseases, as well as the FXN gene therapy program for Friedreich’s Ataxia. Fourth Quarter 2022 Financial Results Collaboration Revenues: Voyager had collaboration revenue of $(1.6) million for the fourth quarter of 2022, compared to $28.1 million for the same period in 2021. The negative collaboration revenue during the fourth quarter of 2022 was due to an increase in the estimate of the total expected cost of Voyager’s research services related to the 2019 collaboration with Neurocrine Biosciences. This increase in the estimate occurred during the fourth quarter of 2022.Net (Loss) Income: Net loss was $23.6 million for the fourth quarter of 2022, compared to net income of $5.7 million for the same period in 2021. The difference was primarily due to the collaboration revenue decrease.R&D Expenses: Research and development expenses were $14.6 million for the fourth quarter of 2022, compared to $14.0 million for the same period in 2021. The increase in R&D expenses was primarily a result of increased external research and development costs and employee-related costs in the fourth quarter of 2022 compared to the same period in 2021. G&A Expenses: General and administrative expenses were $8.5 million for the fourth quarter of 2022, compared to $8.4 million for the same period in 2021.Cash Position: Cash, cash equivalents and marketable securities as of December 31, 2022, were $118.8 million. Full Year 2022 Financial Results Collaboration Revenues: Voyager had collaboration revenue of $40.9 million for 2022, compared to $37.4 million for 2021. The increase in collaboration revenue was due to $40.0 million in collaboration revenue recognized during the third quarter of 2022 in connection with Pfizer exercising its option to license a novel TRACER capsid, offset by a decrease in collaboration revenue from the 2019 collaboration with Neurocrine Biosciences due to a reduction of the cost of Voyager’s research services.Net Loss: Net loss was $46.4 million for 2022, compared to $71.2 million for 2021. The difference is primarily due to the decrease in operating expenses discussed in the two bullets immediately below.R&D Expenses: Research and development expenses were $60.8 million for 2022, compared to $73.8 million for 2021. The decrease in R&D expenses was primarily a result of lower employee-related costs, external research and development costs, and facilities costs. G&A Expenses: General and administrative expenses were $31.0 million for 2022, compared to $37.2 million for 2021. The decrease in G&A expenses was primarily a result of lower employee-related costs, as well as lower facilities costs. Financial GuidanceVoyager is committed to maintaining a strong balance sheet that supports the advancement and growth of its platform and pipeline. Following the recent transactions announced with Neurocrine Biosciences and Novartis, Voyager expects that its cash, cash equivalents, and marketable securities, together with amounts expected to be received as reimbursement for development costs under the Neurocrine collaborations, will be sufficient to meet Voyager’s planned operating expenses and capital expenditure requirements into 2025. This could extend further based on potential future milestones from existing collaboration agreements. Conference CallVoyager will host a conference call and webcast today at 8:30 a.m. ET to discuss the fourth quarter 2022 financial and operating results. To participate via telephone and join the call live, please register in advance here. Upon registration, telephone participants will receive a confirmation email detailing how to join the conference call, including the dial-in number and a unique passcode. A live webcast of the call will also be available on the Investors section of the Voyager website at ir.voyagertherapeutics.com, and a replay of the call will be available at the same link approximately two hours after its completion. The replay will be available for at least 30 days following the conclusion of the call. 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, each with 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,” “target,” “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 anticipated participation in future conferences, Voyager’s ability to continue to identify and develop proprietary capsids from its TRACER capsid discovery platform with increased transgene expression, increased blood-brain barrier penetration and increased biodistribution compared to conventional AAV9 and AAV5 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 capsids; Voyager’s ability to advance its AAV-based gene therapy programs; the preclinical development of its potential development candidates; Voyager’s ability to develop its tau antibody program; Voyager’s ability to perform its obligations under its respective license option agreements with Pfizer and Novartis; Voyager’s entitlement to receive option exercise, milestone and royalty-based fees from Novartis and Pfizer under the respective license option agreements; Voyager’s ability to advance gene therapy product candidates under the Neurocrine collaborations into, and successfully initiate, enroll and complete, clinical trials; Voyager’s ability to maintain its current partnerships and collaborations and to enter into new partnerships or collaborations; the continued service and suitability of recently appointed senior officers and members of the Board of Directors; Voyager’s anticipated financial results, including the receipt by Voyager of revenues or reimbursement payments from collaboration partners; and Voyager’s cash runway and Voyager’s ability to generate sufficient cash resources to enable it to continue its business and operations 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 continued development of Voyager’s technology platforms, including Voyager’s TRACER platform and its antibody screening technology; the ability to initiate and conduct preclinical studies in pre-clinical animal models; 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 platform, the capsids identified by the platform and Voyager’s pipeline developmental programs; the initiation, timing, conduct and outcomes of Voyager’s preclinical studies; the possibility or the timing of the exercise of development, commercialization, license and other options under the Pfizer and Novartis license option agreements and other collaborations; the ability of Voyager to negotiate and complete licensing or collaboration agreements with other parties on terms acceptable to Voyager and the third parties; the ability to attract and retain talented contractors and employees; and the sufficiency of cash resources to fund its operations and pursue its corporate objectives. 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. 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. ContactsInvestorsInvestors@vygr.com Andrew Funderburkafunderburk@kendallir.com MediaTrista Morrisontmorrison@vygr.com Peg Rusconiprusconi@vergescientific.com Selected Financial Information($ amounts in thousands, except per share data)(Unaudited) Three Months Ended December 31, Statement of Operations Items: 2022 2021Collaboration revenue $(1,550) $28,073 Operating expenses: Research and development 14,551 14,020 General and administrative 8,462 8,351 Total operating expenses 23,013 22,371 Operating (loss) income (24,563) 5,702 Total other income, net 953 6 (Loss) income before income taxes (23,610) 5,708 Income tax provision 16 — Net (loss) income $(23,626) $5,708 Net (loss) income per share, basic $(0.61) $0.15 Net (loss) income per share, diluted $(0.61) $0.15 Weighted-average common shares outstanding, basic 38,547,652 37,804,372 Weighted-average common shares outstanding, diluted 38,547,652 37,821,320 Year Ended December 31,  2022 2021Collaboration revenue $40,907 $37,415 Operating expenses: Research and development 60,764 73,787 General and administrative 30,980 37,246 Total operating expenses 91,744 111,033 Operating loss (50,837) (73,618)Total other income, net 4,445 2,421 Loss before income taxes (46,392) (71,197)Income tax provision 16 — Net loss $(46,408) $(71,197)Net loss per share, basic and diluted $(1.21) $(1.89)Weighted-average common shares outstanding, basic and diluted 38,356,810 37,668,947 December 31,  December 31, Selected Balance Sheet Items 2022 2021Cash, cash equivalents, and marketable securities $118,848 $132,539 Total assets $159,356 $193,855 Accounts payable and accrued expenses $10,382 $11,524 Deferred revenue $65,827 $42,096 Total stockholders’ equity $59,020 $95,055