1. A **Vertex Pharmaceuticals** specializing in gene therapy for a range of neurological and rare diseases.

2. Like an early-stage **Moderna** or **BioNTech**, but applying gene therapy to severe neurological diseases instead of mRNA to vaccines.

• VY-AADC: A lead clinical gene therapy candidate for the treatment of Parkinson's disease.
• VY-SOD102: A preclinical gene therapy candidate for the treatment of amyotrophic lateral sclerosis (ALS).
• VY-HTT01: A preclinical gene therapy candidate for the treatment of Huntington's disease.
• VY-FXN01: A preclinical gene therapy candidate for the treatment of Friedreich's ataxia.
• Tau program: A preclinical gene therapy program targeting tauopathies, including Alzheimer's disease, and spinal muscular atrophy.

Major Customers of Voyager Therapeutics (VYGR)

Voyager Therapeutics primarily sells its technology, research, and development services through collaboration and license agreements to other pharmaceutical and biotechnology companies. Its major customers are:

• Neurocrine Biosciences, Inc. (Symbol: NBIX)
• Pfizer Inc. (Symbol: PFE)
• Novartis Pharma, A.G. (Parent company symbol: NVS)

• Lonza Group Ltd. (LONN)
• Catalent, Inc. (CTLT)

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

Dr. Sandrock was appointed President and Chief Executive Officer of Voyager Therapeutics in 2022. He joined Voyager after a 23-year career at Biogen, where he served in various leadership positions, including Executive Vice President, Research and Development (culminating role), and held an eight-year term as Chief Medical Officer from 2012 to 2020. During his tenure at Biogen, he led the discovery, development, and regulatory approval of numerous medicines for neurological diseases, such as Aduhelm, Avonex, Plegridy, Spinraza, Tecfidera, and Tysabri. Prior to joining Biogen, he was an Assistant Professor of Neurology at Harvard Medical School.

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

Dr. Jorgensen was appointed Chief Financial Officer of Voyager Therapeutics, effective July 8, 2024. Before joining Voyager, he served as the Chief Financial Officer for Vor Biopharma Inc. from May 2020 to June 2024, where his responsibilities included finance, communications, facilities, GMP, and IT, and he raised over $400 million in public and private financings. His experience also includes managing global public and private healthcare investing for the Qatar Investment Authority, a sovereign wealth fund, and leading pharma and biotech investing efforts at Calamos Investments LLC. Additionally, Dr. Jorgensen worked as an equity research analyst at Stifel, Nicolaus & Company, Incorporated, and conducted postdoctoral research on Parkinson's disease at the Columbia University Irving Medical Center.

Robin Swartz Chief Business Officer and Chief Operating Officer

Ms. Swartz serves as Voyager's Chief Business Officer and Chief Operating Officer, a role she assumed in February 2022 as Chief Operating Officer. She joined the company in 2021 as Senior Vice President, Portfolio Management and Patient Engagement. Prior to Voyager, Ms. Swartz held various leadership positions at Genzyme and Sanofi Genzyme, including Vice President, Patient and Product Services for Rare Diseases, Senior Vice President, Head of Global and US Business Operations, and Chief of Staff to the Executive Vice President. From April 2, 2024, she also assumed the role of Principal Accounting Officer.

Todd Carter, Ph.D. Chief Scientific Officer

Dr. Carter is the Chief Scientific Officer at Voyager Therapeutics.

Trista Morrison Chief Corporate Affairs Officer and Chief of Staff to the CEO

Ms. Morrison holds the position of Chief Corporate Affairs Officer and Chief of Staff to the CEO at Voyager Therapeutics.

The key risks to Voyager Therapeutics (VYGR) are primarily associated with the inherent challenges of drug development in the gene therapy space, compounded by financial considerations and intense industry competition.

1. Clinical Development and Regulatory Approval Risk: As a gene therapy company, Voyager Therapeutics faces substantial uncertainty regarding the success and timelines of its clinical trials. Biotechnology, especially in gene therapy, is characterized by high failure rates in clinical development. The company's lead candidates, including VY-AADC for Parkinson's disease and other preclinical programs for neurological disorders like Alzheimer's and Huntington's disease, are subject to extensive and often lengthy clinical testing, with no guarantee of favorable outcomes or regulatory approval. Delays or negative results in these trials could significantly impact the company's valuation and future prospects. The general challenges in gene therapy also include potential adverse effects like immune responses, toxicity, and off-target effects, which are under ongoing evaluation.
2. Financial Health and Need for Additional Capital: Voyager Therapeutics has a history of significant operating losses and anticipates continued losses as it advances its pipeline. Despite recent revenue beats, the company exhibits substantial operational inefficiencies, as reflected by negative operating and net margins. While the company reported a cash runway into 2028, high research and development expenses and declining collaboration revenue contribute to a need for substantial future funding. The necessity for additional capital may lead to further equity dilution for existing shareholders.
3. Competition and Industry-Specific Challenges in Gene Therapy (AAV): The gene therapy market, particularly for neurological diseases, is highly competitive with larger pharmaceutical companies vying for market share. Voyager's focus on adeno-associated virus (AAV) gene therapy products also exposes it to industry-wide setbacks and safety concerns that have impacted other companies in the AAV gene therapy space. These challenges include issues with AAV as a delivery mechanism, safety concerns, and the complexities of manufacturing and quality control for gene therapies.

Voyager Therapeutics (NASDAQ: VYGR) focuses on gene therapies for neurological disorders. The addressable markets for its main product candidates are substantial and span several debilitating diseases.

Parkinson's Disease (lead clinical candidate VY-AADC)

• The global Parkinson's disease diagnosis and treatment market is projected to increase from approximately USD 7.49 billion in 2025 to USD 17.57 billion by 2035.
• In the U.S., the Parkinson's disease treatment market was approximately USD 1,883 million in 2023 and is expected to reach USD 2,604.0 million by 2030.

Amyotrophic Lateral Sclerosis (ALS) (preclinical program VY-SOD102)

• The global amyotrophic lateral sclerosis treatment market size was estimated at USD 752.0 million in 2024 and is poised to grow to USD 1238.49 million by 2033.
• The market size for amyotrophic lateral sclerosis in the U.S. was USD 800 million in 2024.

Huntington's Disease (preclinical program VY-HTT01)

• The global Huntington's disease treatment market size was valued at USD 1.2 billion in 2024 and is expected to reach USD 3.7 billion by 2034.

Friedreich's Ataxia (preclinical program VY-FXN01)

• The global Friedreich's ataxia drug market is estimated to reach US$ 1 billion in 2024 and is projected to expand to US$ 3.49 billion by the end of 2034.
• The 7 major Friedreich's Ataxia markets (including the U.S. and key European countries) reached a value of USD 660.4 Million in 2024 and are expected to reach USD 1,882.2 Million by 2035.

Tauopathies (Tau program for Alzheimer's disease, progressive supranuclear palsy, and frontotemporal dementia)

• Alzheimer's Disease

  • The Alzheimer's disease market is expected to grow from USD 2.4 billion in 2023 to USD 17 billion by 2033 across the eight major markets (China, France, Germany, Japan, Italy, Spain, the UK, and the US).
  • The market across the top 7 regions (U.S., EU4, UK, and Japan) reached a value of USD 3.1 Billion in 2024 and is projected to reach USD 6.3 Billion by 2034.

• Progressive Supranuclear Palsy (PSP)

  • The global progressive supranuclear palsy market size was worth more than USD 136.05 million in 2025 and is projected to reach USD 1.82 billion by 2035. Another source indicates the global market will grow from USD 1.6 billion in 2024 to USD 2.8 billion by 2030.
  • The U.S. market for PSP was estimated at USD 411.2 million in 2024 and is projected to reach approximately USD 523.5 million by 2030.

• Frontotemporal Dementia (FTD)

  • The 7 major Frontotemporal Dementia markets (including the U.S. and key European countries) reached a value of US$ 142.8 Million in 2024 and are expected to reach US$ 688.2 Million by 2035.
  • The market size for frontotemporal dementia in the U.S. was USD 60 million in 2024.

Spinal Muscular Atrophy (SMA)

• The global spinal muscular atrophy treatment market size was estimated at USD 4.40 billion in 2024 and is projected to reach USD 13.09 billion by 2030.

Voyager Therapeutics (VYGR) is poised for potential revenue growth over the next 2-3 years, driven primarily by its strategic collaborations and the advancement of its gene therapy pipeline, particularly in neurological diseases like Alzheimer's. The company's future revenue drivers are expected to include:

1. Achievement of Milestones from Existing and New Collaborations: Voyager Therapeutics has established collaboration and license agreements with prominent pharmaceutical companies such as Neurocrine Biosciences, Inc., Pfizer Inc., and Novartis Pharma, A.G.. These partnerships are a significant source of non-dilutive revenue for the company through upfront payments, research funding, and various milestone payments as programs advance through development stages. Voyager actively seeks new collaboration opportunities, which could further contribute to its revenue stream through additional milestone payments.
2. Clinical Progression and Data Readouts for Tau-Targeting Assets in Alzheimer's Disease: Key clinical catalysts anticipated in the next 2-3 years include the advancement of VY1706 and VY7523, Voyager's tau-targeting assets for Alzheimer's disease. VY1706, a tau-silencing gene therapy, is expected to complete a GLP toxicology study in Q1 2026, submit an Investigational New Drug (IND) application in Q2 2026, and initiate first-in-human dosing in the second half of 2026. Additionally, initial tau PET imaging data from the multiple ascending dose (MAD) clinical trial for VY7523, an anti-tau antibody, are expected in the second half of 2026. Positive data from these clinical trials could trigger further milestone payments from partners and enhance the value of these pipeline assets.
3. Clinical Entry of Novel I.V.-Delivered Neuro Gene Therapies: Voyager expects two novel intravenously (I.V.)-delivered neuro gene therapies to enter clinical trials in the second half of 2026. The company emphasizes the importance of I.V. delivery for the commercial viability of gene therapies targeting most central nervous system (CNS) diseases. The successful clinical entry and validation of these brain-targeted capsids, leveraging Voyager's proprietary TRACER™ and NeuroShuttle™ platforms, could lead to new collaboration opportunities and potential future product revenue.

Share Issuance

• Voyager Therapeutics' forward-looking statements indicate plans to raise additional capital, potentially through equity offerings.
• The number of common shares outstanding increased from 55,600,084 as of November 3, 2025, to 59,599,375 as of March 2, 2026.

Inbound Investments

• Collaboration revenue for the year ended December 31, 2025, was $40.4 million, a decrease from $80.0 million in 2024.
• In 2025, collaboration revenue included $30.5 million from the 2023 Neurocrine Collaboration Agreement, $5.9 million from the 2023 Novartis Collaboration Agreement, and $3.5 million from the 2019 Neurocrine Collaboration Agreement.
• Voyager received a $3 million milestone payment from Neurocrine in Q4 2025 after Neurocrine initiated a preclinical toxicology study for a fourth development candidate under their collaboration agreement.

Capital Expenditures

• In the last 12 months, capital expenditures were -$2.60 million.
• Voyager anticipates that its cash, cash equivalents, and marketable securities, along with collaboration reimbursements and interest income, will be sufficient to cover planned operating expenses and capital expenditure requirements into 2028.
• Research and development (R&D) expenses, which often include investments in facilities or equipment that could be classified as capital expenditures, increased modestly to $134.67 million in 2025 from $127.37 million in 2024, reflecting focused investments on tau programs and the Voyager NeuroShuttle platform.