• Like Moderna or BioNTech, but focused on developing gene therapies to treat severe neurological diseases.
• Imagine a specialized version of Gilead Sciences, but applying gene therapy to neurological conditions.

• VY-GBA01 (Parkinson's Disease): A gene therapy candidate for Parkinson's disease, developed to address GBA mutations.
• VY-HTT01 (Huntington's Disease): A gene therapy candidate aimed at treating Huntington's disease by lowering huntingtin protein levels.
• VY-FXN01 (Friedreich's Ataxia): A gene therapy candidate designed to treat Friedreich's Ataxia.
• TRACER™ Capsid Discovery Platform: A proprietary technology platform used to develop enhanced adeno-associated virus (AAV) capsids for gene therapy delivery.

• Neurocrine Biosciences, Inc. (NASDAQ: NBIX)
• Novartis Pharma AG (NYSE: NVS)
• Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) - A recently announced significant collaboration partner.

• Thermo Fisher Scientific Inc. (TMO)
• Sartorius Stedim Biotech S.A. (DIM)

Alfred W. Sandrock, Jr., M.D., Ph.D. President and Chief Executive Officer

Dr. Sandrock joined Voyager Therapeutics as President and CEO in March 2022. He previously spent 23 years at Biogen, serving in various senior executive roles including Executive Vice President, Research and Development, and Chief Medical Officer. During his tenure at Biogen, he led the discovery, development, and regulatory approval of numerous medicines, including ADUHELM™, AVONEX®, PLEGRIDY®, SPINRAZA®, TECFIDERA®, and TYSABRI®. Dr. Sandrock also serves on the board of directors for Verge Genomics, Inc., Transition Bio, Inc., and Neurimmune, Inc.

Nathan Jorgensen, Ph.D., MBA Chief Financial Officer

Mr. Jorgensen was appointed Chief Financial Officer of Voyager Therapeutics in July 2024. He previously served as Chief Financial Officer for Vor Biopharma Inc., where he oversaw finance, communications, facilities, GMP, and IT, and raised over $400 million in public and private financings. Before joining Vor Biopharma, he managed global public and private healthcare investing for the Qatar Investment Authority, a sovereign wealth fund, and led pharma and biotech investing efforts at Calamos Investments LLC. Prior to his financial sector roles, Mr. Jorgensen conducted postdoctoral research on Parkinson's disease at the Columbia University Irving Medical Center.

Todd Carter, Ph.D. Chief Scientific Officer

Dr. Carter is a neuroscientist with over 20 years of experience in the life science industry. He previously held the position of Senior Vice President of Research at Voyager Therapeutics, and his work has been cited in more than 20 key research publications on neurological indications.

Toby Ferguson, M.D., Ph.D. Chief Medical Officer

Dr. Ferguson serves as the Chief Medical Officer at Voyager Therapeutics.

Robin Swartz Chief Business Officer and Chief Operating Officer

Ms. Swartz has served as Chief Operating Officer of Voyager Therapeutics since February 2022, and also holds the title of Chief Business Officer. Her previous roles at Voyager include Senior Vice President, Business Operations and Senior Vice President, Portfolio Management and Patient Engagement. Before joining Voyager, Ms. Swartz held increasingly responsible positions at Genzyme Corporation and Sanofi Genzyme, culminating in her service as Senior Vice President, Head of Patient Support Services for Rare Diseases and Vice President, Head of Global and US Business Operations. Her earlier experience at Sanofi Genzyme included roles as Chief of Staff to the Executive Vice President and Senior Director, Finance.

The key risks to Voyager Therapeutics (VYGR) are primarily centered around the challenging nature of drug development in its focus areas, its financial sustainability, and the competitive and evolving regulatory landscape.

• Clinical Trial Success and Efficacy/Safety Data: Voyager Therapeutics' business model and future profitability are highly dependent on the successful development and commercialization of its product candidates, many of which are in early-stage clinical development or preclinical stages and lack conclusive efficacy and safety data in humans. The company's gene therapy programs, including those leveraging its TRACER capsids, face the inherent risk of failure in clinical trials, particularly for complex neurological disorders like Alzheimer's and Parkinson's disease, which have high failure rates. The efficacy and safety of its core TRACER capsid technology in humans, especially concerning its ability to cross the blood-brain barrier and deliver therapeutics without damage to other tissues, are still pending definitive human testing.

• Financial Viability and Path to Profitability: Voyager Therapeutics is currently unprofitable and anticipates significant increases in expenses due to ongoing clinical trials, research and development, and operational expansion. While the company projects its cash resources, along with anticipated collaboration reimbursements, to be sufficient into mid-2027 or even 2028, there is an increasing net loss and a reliance on collaboration revenues, which can be unpredictable. Any decline in these collaboration revenues or failure to meet milestones could heighten financial risk and impact the company's cash runway, as profitability is still years away and contingent on successful product commercialization.

• Competition and Regulatory Landscape: The company operates in a highly competitive environment, particularly within the Alzheimer's disease market, where it competes with much larger pharmaceutical companies. Furthermore, the regulatory landscape for gene therapies is dynamic and evolving, which can lead to extended development timelines, increased costs, and potential delays in obtaining necessary approvals. Voyager has experienced past regulatory hurdles, such as FDA holds on trials due to manufacturing process concerns. The emergence of competitive capsid identification platforms by third parties also poses a risk to Voyager's proprietary technology.

Voyager Therapeutics (VYGR) is developing therapies for several neurological diseases. The addressable markets for their main products or services are as follows:

• Alzheimer's Disease (Anti-tau Antibody, Tau Silencing Gene Therapy, APOE Gene Therapy, Vectorized Anti-amyloid Antibody): The global gene therapy market for Central Nervous System (CNS) disorders, which includes Alzheimer's disease, is projected to reach USD 13.86 billion by 2025 and USD 191.04 billion by 2035, growing at a CAGR of 30%. Alzheimer's disease was the dominant segment in this market, holding a 37.4% market share in 2023.
• Friedreich's Ataxia (FXN Gene Therapy): The global Friedreich's Ataxia market was valued at USD 775.3 million in 2023 and is projected to reach USD 2,347.6 million by 2032, with a Compound Annual Growth Rate (CAGR) of 13.1% from 2024 to 2032. Another estimate indicates the global market will grow from USD 2.63 billion in 2024 to USD 8.4 billion by 2035. The 7 major markets (United States, Germany, France, United Kingdom, Italy, Spain, and Japan) for Friedreich's Ataxia reached USD 660.4 million in 2024 and are expected to reach USD 1,882.2 million by 2035. The United States accounts for the largest market share within these regions.
• Parkinson's Disease (GBA1 Gene Therapy): The global Parkinson's disease therapeutics market was estimated at USD 6.59 billion in 2024 and is predicted to increase from USD 7.02 billion in 2025 to approximately USD 13.34 billion by 2034, expanding at a CAGR of 7.39% from 2025 to 2034. The U.S. Parkinson's disease therapeutics market alone was valued at USD 2.15 billion in 2024 and is projected to reach USD 4.25 billion by 2034. The cell and gene therapy market for Parkinson's disease in the 7MM (United States, EU-4, United Kingdom, and Japan) is also expected to grow significantly by 2034.
• Huntington's Disease (Research Program): The global Huntington's disease treatment market size was estimated at USD 500 million in 2024 and is projected to reach USD 1,871.2 million by 2030, growing at a CAGR of 23.8% from 2025 to 2030. Another forecast places the global market at USD 724.6 million in 2025, reaching USD 1,543.9 million by 2035. The United States Huntington's Disease Treatment Market is anticipated to grow at a CAGR of 15.22% from 2025 to 2033, reaching US$ 552.5 million by 2033, from US$ 154.38 million in 2024.
• Amyotrophic Lateral Sclerosis (ALS) (TDP-43 Small Molecule, SOD1 Silencing Gene Therapy): ALS is a key target within the broader global gene therapy market for CNS disorders. This market is projected to reach USD 13.86 billion by 2025 and USD 191.04 billion by 2035.

Voyager Therapeutics (NASDAQ: VYGR) is strategically positioning itself for future revenue growth over the next two to three years primarily through the advancement of its neurotherapeutics pipeline and the expansion of its collaboration-driven business model.

Here are 3-5 expected drivers of future revenue growth:

1. Achievement of Milestone Payments from Existing Partnerships: Voyager's revenue is currently entirely derived from collaboration activities, and the company has the potential to earn significant non-dilutive capital through milestone payments from its existing partnerships. For instance, a $3 million milestone payment from the Neurocrine partnership was triggered in Q4 2025. Additionally, the Neurocrine-partnered Friedreich's ataxia (FA) and GBA1 gene therapy programs have the potential to generate up to $35 million in associated regulatory and clinical milestones if they enter clinical trials in 2026.
2. New Strategic Collaborations and Licensing Agreements: Voyager continues to pursue and establish new partnerships to leverage its proprietary platforms and pipeline assets. A recent collaboration with Transition Bio, announced in November 2025, focuses on developing small molecules targeting TDP-43 in ALS and frontotemporal dementia, with potential milestones of up to $500 million. This demonstrates an ongoing strategy to generate revenue through licensing opportunities and collaborative research and development efforts.
3. Advancement of Tau-Targeting Programs: Voyager is making significant progress with its wholly-owned tau-targeting programs for Alzheimer's disease. The anti-tau antibody program, VY7523, is currently in the third and final cohort of its multiple ascending dose (MAD) clinical trial. Furthermore, the tau silencing gene therapy program, VY1706, is undergoing Investigational New Drug (IND)-enabling studies, with clinical trial initiation anticipated in 2026. Positive clinical data and progression of these programs through development stages could trigger additional milestone payments from partners or attract new collaboration opportunities.
4. Utilization and Out-licensing of Proprietary Delivery Platforms: The company's innovative Voyager NeuroShuttle™ nonviral delivery platform and TRACER™ AAV capsid platform are designed to enhance the delivery of neurotherapeutics across the blood-brain barrier. As these platforms are further validated and developed, they present opportunities for new partnerships and licensing agreements, providing access to their advanced delivery technology for other neurological programs, thereby driving future collaboration revenue.

Share Issuance

• In January 2024, Voyager Therapeutics priced an underwritten public offering of common stock and pre-funded warrants, resulting in aggregate gross proceeds of approximately $100 million.
• Over the past few years, the company has issued shares, with reported amounts of $114.02 million in 2023, $33.65 million in 2022, $1.11 million in 2021, and $0.61 million in 2020 through "Issuance of Capital Stock."
• The 2015 Employee Stock Purchase Plan (ESPP) authorized the issuance of 262,362 shares initially, with an additional 1% of outstanding common stock added annually, totaling 3,132,873 shares through January 1, 2025.

Inbound Investments

• In November 2025, Voyager entered into a collaboration with Transition Bio, receiving a single-digit million-dollar upfront payment and potentially up to $500 million in milestone payments for developing small molecules targeting TDP-43 in ALS and FTD.
• In late 2023 and early 2024, Voyager formed a strategic collaboration and stock purchase agreement with Novartis, which included a $20.0 million payment for shares and an anticipated $80.0 million upfront payment.
• The company has the potential to receive up to $2.4 billion in additional non-dilutive funding from development milestone payments across its existing collaborations, including up to $35 million from GBA and FA programs as they enter clinical phases.

Capital Expenditures

• Voyager Therapeutics anticipates that its current cash, cash equivalents, and marketable securities will be sufficient to cover its operating expenses and capital expenditure requirements into 2028.
• As of September 30, 2025, the company reported $229 million in cash, cash equivalents, and marketable securities.
• Capital expenditures are primarily focused on advancing clinical trials, expanding research and development initiatives, and maintaining strategic collaborations.