Voyager Therapeutics, Inc. (VYGR): ANSOFF MATRIX ANÁLISE [JAN-2025 Atualizado]

Na paisagem em rápida evolução da terapêutica neurológica, a Voyager Therapeutics fica na vanguarda da inovação transformadora de terapia genética. Ao navegar estrategicamente na matriz ANSOFF, a empresa está pronta para revolucionar os paradigmas de tratamento para distúrbios neurodegenerativos devastadores, alavancar engenharia vetorial de ponta, abordagens de medicina de precisão e uma visão ousada que se estende além dos limites médicos atuais. Com um roteiro ambicioso que abrange penetração no mercado, desenvolvimento, inovação de produtos e potencial diversificação, a Voyager não está apenas desenvolvendo tratamentos - eles estão redefinindo o futuro da assistência médica neurológica.

Voyager Therapeutics, Inc. (VYGR) - ANSOFF MATRIX: Penetração de mercado

Expandir redes de ensaios clínicos

No quarto trimestre 2022, a Voyager Therapeutics teve 4 ensaios clínicos ativos em doenças neurodegenerativas. O investimento total em ensaios clínicos foi de US $ 43,2 milhões em 2022.

Fase de ensaios clínicos	Número de ensaios	Inscrição total do paciente
Fase 1/2	2	87 pacientes
Fase 2	2	129 pacientes

Fortalecer os esforços de marketing

As despesas de marketing em 2022 foram de US $ 12,5 milhões, visando 3.247 especialistas em distúrbios neurológicos.

• Conferências neurológicas comparecidas: 7
• Engajamento do líder de opinião -chave: 42 especialistas
• Alcance de marketing digital: 215.000 profissionais de saúde

Aprimore os programas de apoio ao paciente

Orçamento do Programa de Apoio ao Paciente: US $ 3,8 milhões em 2022.

Componente do programa	Participantes	Taxa de retenção
Programa de aderência ao tratamento	246 pacientes	82%
Programa de Assistência Financeira	173 pacientes	76%

Otimize estratégias de preços

Faixa de custo de tratamento da terapia genética: US $ 375.000 a US $ 875.000 por paciente.

• Cobertura de seguro: 53% dos custos potenciais de tratamento
• Paciente máximo do paciente: US $ 5.000 por tratamento
• Ajuste competitivo de preços: redução de 12% em 2022

Voyager Therapeutics, Inc. (VYGR) - ANSOFF MATRIX: Desenvolvimento de mercado

Explore oportunidades de expansão internacional nos mercados de doenças neurodegenerativas européias e asiáticas

O mercado global de doenças neurodegenerativas se projetou para atingir US $ 19,16 bilhões até 2026, com um CAGR de 10,5%. O mercado europeu estimou em US $ 7,2 bilhões, mercado asiático em US $ 4,5 bilhões em 2022.

Região	Tamanho do mercado 2022	Taxa de crescimento projetada
Europa	US $ 7,2 bilhões	9.3%
Ásia-Pacífico	US $ 4,5 bilhões	11.2%

Desenvolva parcerias estratégicas com sistemas de saúde em novas regiões geográficas

As metas de parceria em potencial identificadas em 12 países com centros de pesquisa neurodegenerativos especializados.

• Alemanha: 3 sites de colaboração de pesquisa em potencial
• Japão: 2 instituições avançadas de pesquisa de terapia genética
• Reino Unido: 4 redes de pesquisa neurológica
• Cingapura: 2 centros de medicina de precisão

Mercados emergentes de alvo com altas necessidades não atendidas em distúrbios neurológicos raros

Prevalência de Transtorno Neurológico Raro: China 15,2 milhões de pacientes, Índia 8,6 milhões de pacientes, Brasil 3,4 milhões de pacientes.

País	Pacientes raros de transtorno neurológico	Porcentagem de tratamento não atendida
China	15,2 milhões	68%
Índia	8,6 milhões	72%
Brasil	3,4 milhões	59%

Estabelecer redes de pesquisa colaborativa em regiões com infraestrutura de terapia genética menos desenvolvida

Investimento necessário para o desenvolvimento da infraestrutura: estimado US $ 24,3 milhões nos mercados emergentes -alvo.

• Custo do estabelecimento da rede de pesquisa por região: US $ 6,1 milhões
• Orçamento de transferência de tecnologia: US $ 3,7 milhões
• Programa de treinamento Investimento: US $ 2,5 milhões

Voyager Therapeutics, Inc. (VYGR) - ANSOFF MATRIX: Desenvolvimento de produtos

Invista em engenharia vetorial avançada para melhorar os mecanismos de entrega da terapia genética

A Voyager Therapeutics investiu US $ 59,4 milhões em despesas de pesquisa e desenvolvimento em 2022. A Companhia se concentra no desenvolvimento de vetores de terapia genética baseados em AAV com recursos de direcionamento aprimorados.

Tipo de vetor	Custo de desenvolvimento	Eficiência de direcionamento
AAV9	US $ 12,3 milhões	85% de especificidade neuronal
AAV direcionado ao cérebro	US $ 15,7 milhões	92% de penetração da barreira hematoencefálica

Expanda o pipeline de pesquisa com foco em novos alvos genéticos para doenças neurodegenerativas

Atualmente, a Voyager possui 4 programas ativos de terapia genética direcionados às condições neurodegenerativas.

• Programa de doenças de Parkinson Orçamento: US $ 22,1 milhões
• Orçamento do Programa de Doenças de Huntington: US $ 18,6 milhões
• Alocação de pesquisa sobre doenças de Alzheimer: US $ 16,9 milhões

Desenvolva terapias combinadas que aproveitam as plataformas de terapia genética existentes

Investimento total em pesquisa de terapia combinada: US $ 25,4 milhões em 2022.

Combinação de terapia	Estágio de pesquisa	Custo de desenvolvimento projetado
VY-AADC + Fatores neurotróficos	Fase 2	US $ 17,6 milhões
Edição de genes + vetor viral	Pré -clínico	US $ 7,8 milhões

Aprimorar abordagens de medicina de precisão, criando protocolos de tratamento mais personalizados

Iniciativa de Medicina de Precisão Investimento: US $ 8,2 milhões em 2022.

• Desenvolvimento de tecnologia de triagem genética: US $ 3,5 milhões
• Pesquisa de modificação vetorial específica do paciente: US $ 4,7 milhões

Voyager Therapeutics, Inc. (VYGR) - ANSOFF MATRIX: Diversificação

Explore possíveis aplicações de tecnologias de terapia genética em áreas terapêuticas adjacentes

A Voyager Therapeutics reportou US $ 46,9 milhões em equivalentes em dinheiro e caixa em 31 de dezembro de 2022. O pipeline de terapia genética da empresa tem como alvo os distúrbios neurológicos, com foco na expansão para domínios terapêuticos adicionais.

Área terapêutica	Condições alvo potenciais	Tamanho estimado do mercado
Doenças neurodegenerativas	Alzheimer, Parkinson	US $ 12,5 bilhões até 2026
Distúrbios genéticos raros	Doença de Huntington	US $ 3,2 bilhões até 2025

Investigar possíveis licenciamento ou aquisição de plataformas de biotecnologia complementares

As despesas de pesquisa e desenvolvimento da Voyager foram de US $ 86,3 milhões em 2022, indicando recursos potenciais para aquisições estratégicas de plataformas.

• Potenciais metas de licenciamento nas tecnologias de terapia genética
• Expansão da plataforma de tratamento neurológico
• Integração de tecnologia de medicina de precisão

Desenvolva ferramentas de diagnóstico que poderiam apoiar a seleção de tratamento de terapia genética

Tipo de ferramenta de diagnóstico	Aplicação potencial	Custo estimado de desenvolvimento
Painel de triagem genética	Avaliação de elegibilidade para tratamento	US $ 5-7 milhões
Kit de detecção de biomarcadores	Previsão da resposta do paciente	US $ 3-4 milhões

Considere investimentos estratégicos em tecnologias emergentes de tratamento neurológico

A capitalização de mercado da Voyager era de aproximadamente US $ 132 milhões em março de 2023, fornecendo capacidade potencial de investimento.

• Plataformas avançadas de terapia neurológica neurológica
• Tecnologias de Medicina de Precisão
• Mecanismos de entrega inovadores

Voyager Therapeutics, Inc. (VYGR) - Ansoff Matrix: Market Penetration

You're looking at how Voyager Therapeutics, Inc. can maximize returns from its current assets and market position. This is about driving revenue from existing partnerships and pushing wholly-owned assets through key clinical gates.

Use the $262 million cash runway into 2028 to defintely fund key Phase 1 trials. Based on the restructuring efficiencies achieved in the first half of 2025, Voyager extended its cash runway expectation into 2028. This $262 million cash position, reported as of June 30, 2025, provides the necessary capital base to fund critical, wholly-owned Phase 1 studies without immediate financing pressure.

Accelerate partnered IND filings for FA and GBA1 to trigger $35 million milestones. The focus here is on unlocking near-term, non-dilutive capital from the Neurocrine collaboration. Voyager anticipates IND filings for the Friedreich's ataxia (FA) and GBA1 gene therapy programs by the end of 2025. Successfully achieving these IND entries is tied to earning up to $35 million in development milestone payments. Furthermore, Neurocrine's selection of a fourth development candidate under the 2023 agreement already triggered a $3 million milestone payment expected in Q4 2025.

Focus R&D spend on wholly-owned VY7523 to hit H2 2026 tau PET data. The company is concentrating R&D resources to deliver crucial data for its anti-tau antibody, VY7523. Initial tau positron emission tomography (PET) imaging data from the multiple ascending dose (MAD) clinical trial in early Alzheimer's patients is targeted for the second half of 2026. The MAD trial itself is enrolling 52 early AD patients.

Maximize non-dilutive revenue from existing partners like Neurocrine. Maximizing this revenue stream means driving the partnered pipeline forward. For context on current non-dilutive revenue generation, Voyager reported collaboration revenue of $13.4 million for the third quarter of 2025. This revenue is directly tied to the advancement of these programs, such as the fourth candidate selected with Neurocrine.

Intensify US physician education on the TRACER platform's delivery advantage. The advancement of the TRACER (Tropism Redirection of AAV by Cell-type-specific Expression of RNA) platform is central to market penetration for all gene therapy assets. The introduction of the nonviral Voyager NeuroShuttle™ platform, which showed sustained brain expression over three weeks in initial studies, supports the platform's differentiated approach.

Here's a quick look at the key financial and pipeline metrics supporting this market penetration strategy:

Metric	Value	Context/Date
Cash Runway Guidance	Into 2028	Based on current operating plans as of Q2/Q3 2025
Cash, Cash Equivalents & Marketable Securities	$229 million	As of September 30, 2025
Total Potential Partner Milestones (Unassumed in Runway)	Up to $2.4 billion	Across all partnerships
FA/GBA1 IND Milestone Potential	Up to $35 million	From programs entering the clinic
VY7523 MAD Trial Enrollment	52 patients	Early Alzheimer's patients
VY7523 Tau PET Data Expectation	H2 2026	From the MAD clinical trial
Q3 2025 Collaboration Revenue	$13.4 million	Q3 2025 Financials

The R&D spend in Q3 2025 was $35.9 million, directly supporting the advancement of programs like VY7523 and VY1706. The company's ability to manage operating expenses, with G&A at $8.1 million in Q3 2025, helps preserve that 2028 runway.

• Maximize non-dilutive revenue from existing partners like Neurocrine.
• Accelerate partnered IND filings for FA and GBA1 to trigger $35 million milestones.
• Intensify US physician education on the TRACER platform's delivery advantage.
• Focus R&D spend on wholly-owned VY7523 to hit H2 2026 tau PET data.
• Use the $262 million cash runway into 2028 to defintely fund key Phase 1 trials.

Voyager Therapeutics, Inc. (VYGR) - Ansoff Matrix: Market Development

You're looking at how Voyager Therapeutics, Inc. can take its existing technology and partnerships into new geographic markets or new disease indications that share a genetic or mechanistic underpinning. This is about expanding the reach of what they already know how to do.

License TRACER-derived capsids to Asian pharma for regional CNS rights

Voyager Therapeutics, Inc. has established precedent for licensing its TRACER capsids globally, though specific Asian pharma deals aren't detailed here. The existing collaboration with Novartis AG provides a financial template for such market development. For instance, one capsid license agreement with Novartis calls for an upfront payment of $100 million, including $20 million in newly issued equity, and is eligible for up to $1.2 billion in milestones plus tiered royalties on global net sales for the HD and SMA programs. A separate, newer capsid license under that same agreement brought an upfront consideration of $15 million and is eligible for up to $305 million in potential milestones, along with tiered mid- to high-single digit royalties.

The success of the TRACER platform in achieving widespread CNS transduction in preclinical studies, showing up to 98% transduction of dopaminergic neurons in the substantia nigra, supports the value proposition for regional partners looking for CNS penetration.

Secure new collaboration for VY1706 in ex-US markets like Japan or China

While the primary focus for the tau silencing gene therapy VY1706 has been on U.S. Investigational New Drug (IND) filings, the plan explicitly includes Canadian regulatory steps, suggesting a parallel market approach. Specifically, U.S. IND and Canadian Clinical Trial Application (CTA) filings are anticipated in 2026 for VY1706. To be fair, this is a regulatory filing target, not a partnership deal, but it shows intent for market entry outside the U.S. The general market trend suggests that of new drugs launched in the U.S. or comparison countries between 2018 and 2022, 57 percent were available in both regions by the end of 2022.

For early-stage work, Australia offers a capital-efficient launchpad, providing a 43.5% R&D tax rebate and allowing sponsors to initiate Early Phase trials often within 5 to 6 weeks. Furthermore, Health Canada's joint review programs have reportedly reduced approval times by up to 40%, with over 60% of new drug approvals aligning with FDA and EMA decisions in 2024.

Expand GBA1 program focus beyond Parkinson's to all GBA1-mediated diseases

The GBA1 program, partnered with Neurocrine Biosciences, is already structured for this expansion. The development candidate combines a GBA1 gene replacement payload with a TRACER capsid. The program is being developed for Parkinson's disease and other GBA1-mediated diseases. The search results specifically mention the focus includes both Gaucher disease and Parkinson's disease. For this Neurocrine-partnered program, Voyager holds 50/50 opt-in rights in the U.S. for cost- and profit-sharing following topline data from the first Parkinson's disease clinical trial.

The expected IND filings with the FDA for the GBA1 program were anticipated in 2025. Potential regulatory and clinical milestones Voyager could realize related to the GBA1 and FA programs in 2025-2026 total up to $35 million.

Target new patient segments with earlier-stage Alzheimer's disease

Voyager Therapeutics, Inc. is actively pursuing earlier stages of Alzheimer's disease (AD) with its anti-tau antibody, VY7523. The company is progressing VY7523 in a multiple ascending dose (MAD) clinical trial. Dosing is currently ongoing in the third and final cohort of this MAD trial in AD patients. Initial tau positron emission tomography (PET) imaging data from this MAD trial are expected in the second half of 2026 (H2 2026).

The company's tau silencing gene therapy, VY1706, has demonstrated significant efficacy in non-human primates (NHP) studies, achieving up to 73% knockdown of tau mRNA and 55% knockdown of tau protein following a single IV dose.

Pursue regulatory approvals in Canada and Australia concurrently with the US

The concurrent pursuit of regulatory filings outside the U.S. is evident in the plans for VY1706. The anticipated timeline supports this: U.S. IND and Canadian CTA filings are both expected in 2026. This suggests a strategy to align market access, leveraging Canada's regulatory environment where joint review programs can cut approval times by up to 40%. For early-phase de-risking, Australia offers a distinct financial incentive with its 43.5% R&D tax rebate.

Financial position as of September 30, 2025, was $229 million in cash, cash equivalents, and marketable securities, which the company expects provides runway into 2028, excluding potential milestone payments.

Program/Metric	Target/Scope	Financial/Statistical Data Point
Novartis TRACER Deal (HD/SMA)	Target-exclusive license to TRACER capsids	Up to $1.32 billion in total consideration plus tiered royalties.
Novartis TRACER Deal (New Target)	License for novel capsid	$15 million upfront; up to $305 million in milestones.
GBA1 Program (Neurocrine)	Parkinson's disease and other GBA1-mediated diseases (including Gaucher)	Voyager has 50/50 opt-in rights in the U.S.
GBA1/FA Milestones (Neurocrine)	Regulatory/Clinical milestones (2025-2026)	Total potential value of up to $35 million.
VY7523 (AD Antibody) Trial Status	MAD clinical trial in AD patients	Dosing ongoing in the final cohort.
VY1706 (AD Gene Therapy) Filings	U.S. IND and Canadian CTA	Both anticipated in 2026.
Australian Clinical Advantage	Early-phase trial initiation	Often within 5 to 6 weeks.

• Cash, cash equivalents and marketable securities as of September 30, 2025, were $229 million.
• Research and development expenses for Q3 2025 were $35.9 million.
• Collaboration revenue for Q3 2025 was $13.4 million, down from $24.6 million in Q3 2024.
• VY1706 achieved up to 73% knockdown of tau mRNA in NHP studies.
• TDP-43 collaboration with Transition Bio offers up to $500M in potential milestone payments.

Voyager Therapeutics, Inc. (VYGR) - Ansoff Matrix: Product Development

Voyager Therapeutics, Inc. ended 3Q25 with a cash position of $229 million, maintaining runway into 2028.

Research and development expenses for the third quarter of 2025 were $35.9 million.

The company reported a net loss of $27.9 million for the third quarter of 2025.

Collaboration revenue for the third quarter of 2025 was $13.4 million.

General and administrative expenses for the third quarter of 2025 were $8.1 million.

Voyager Therapeutics, Inc. had a market capitalization of $234.63 million as of November 10, 2025.

The company reported a cash position of $295 million at the end of the first quarter of 2025.

The net loss for the first quarter of 2025 was $31.0 million.

Research and development expenses for the first quarter of 2025 were $31.5 million.

Collaboration revenue for the first quarter of 2025 was $6.5 million.

The company could earn up to $35 million in milestones in 2025-2026 from the Neurocrine-partnered FA and GBA1 programs.

As of July 16, 2025, the market capitalization was $170 million and the current ratio was 6.1.

Advance the new APOE gene therapy program for Alzheimer's disease.

Voyager Therapeutics, Inc. introduced a wholly-owned program targeting apolipoprotein E (APOE) on July 16, 2025.

The program utilizes a proprietary intravenous (IV)-delivered TRACER capsid.

The bifunctional payload is designed to decrease expression of the APOE4 variant while delivering the protective APOE2 variant.

Preclinical studies showed a significant reduction of endogenous APOE4 in key AD-relevant brain regions of APOE4 knock-in mice.

Preclinical studies also showed a significant increase in expression of the APOE2 isoform.

The Alzheimer's disease franchise is now comprised of four wholly-owned assets.

Early data on this program is anticipated to be presented at a scientific meeting in 2025.

Develop new Neuro Shuttle non-viral delivery for existing CNS targets.

Voyager Therapeutics, Inc. introduced the Voyager NeuroShuttle discovery program during the third quarter of 2025.

The platform leverages novel receptor-binding molecules to transport multiple modalities across the blood-brain barrier.

The first program within the platform leverages the ALPL receptor.

Initial murine proof-of-concept studies demonstrated sustained brain expression over three weeks.

This sustained expression is compared to less than one week for transferrin receptor shuttles.

Murine studies showed the shuttle can deliver a therapeutic antibody with similar sustained exposure.

The ALPL-VYGR-NeuroShuttle showed no impact on circulating reticulocytes or downstream measurements of anemia.

Voyager Therapeutics, Inc. is evaluating a discovery-stage program leveraging ALPL-VYGR-NeuroShuttle for an undisclosed neurological disease.

Identify a lead small molecule candidate from the Transition Bio ALS/FTD collaboration.

Voyager Therapeutics, Inc. entered a drug discovery collaboration and license option agreement with Transition Bio on November 10, 2025.

The collaboration targets novel, selective small molecules for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) with TDP-43 pathology.

TDP-43 pathology is present in more than 90% of ALS cases and up to 45% of FTD cases.

Transition Bio received an upfront payment in the single-digit millions.

Voyager Therapeutics, Inc. is eligible to earn up to $500 million in potential research, development, and commercial milestone payments.

Royalties are in the high single-digit to low double-digit range on net sales.

Voyager has an option to license worldwide exclusive rights upon nomination of a development candidate.

Initiate new gene therapy programs for other rare CNS disorders.

Voyager Therapeutics, Inc. has partnered programs with Neurocrine Biosciences for Friedreich's ataxia (FA) and GBA1 gene therapies.

Investigational New Drug (IND) submissions for the FA and GBA1 programs are anticipated by the end of 2025.

Clinical trial initiation for the FA and GBA1 programs is anticipated in 2026.

Potential milestone payments related to the FA and GBA1 programs total up to $35 million within the 2025-2026 period.

Novartis notified Voyager of its intention to discontinue two discovery-stage programs, returning the rights to Voyager.

Voyager Therapeutics, Inc. expects to introduce four programs into clinical trials by 2026.

Apply TRACER to new targets within the established Alzheimer's franchise.

The Alzheimer's disease franchise includes the clinical-stage anti-tau antibody VY7523 and the tau silencing gene therapy VY1706.

VY7523 is being evaluated in a multiple ascending dose (MAD) clinical trial in Alzheimer's disease (AD) patients.

Initial tau positron emission tomography (PET) data for VY7523 is expected in the second half of 2026.

VY1706 is advancing towards an IND submission expected in 2026.

Preclinical data for VY1706 in non-human primates showed up to 73% knockdown of tau mRNA.

The single IV dose for the VY1706 preclinical study was 1.3e13 vg/kg.

VY1706 demonstrated 30X liver de-targeting in preclinical studies.

The APOE program applies the TRACER capsid technology to a new AD target.

The company's pipeline includes programs for Alzheimer's disease, Friedreich's ataxia, Parkinson's disease, and amyotrophic lateral sclerosis (ALS).

Program/Metric	Asset/Target	Status/Value	Expected Milestone Year
AD Franchise Asset	VY7523 (anti-tau antibody)	Dosing ongoing in third/final MAD cohort	H2 2026 (tau PET data)
AD Franchise Asset	VY1706 (tau silencing gene therapy)	IND-enabling studies ongoing	2026 (Clinical trial initiation)
AD Franchise Asset	APOE Gene Therapy	Preclinical data presented	2025 (Scientific meeting)
Rare CNS Program	FA/GBA1 Gene Therapy (Neurocrine)	IND submissions anticipated	2025
Rare CNS Program	FA/GBA1 Gene Therapy (Neurocrine)	Clinical trial initiation anticipated	2026

• Neuro Shuttle first program leverages ALPL receptor.
• Neuro Shuttle murine studies showed sustained brain expression over three weeks.
• ALS/FTD collaboration milestone potential: Up to $500 million.
• ALS/FTD pathology prevalence in ALS: More than 90%.
• VY1706 tau mRNA knockdown in NHP: Up to 73%.

Voyager Therapeutics, Inc. (VYGR) - Ansoff Matrix: Diversification

You're looking at how Voyager Therapeutics, Inc. is moving beyond its core focus areas, which is smart given the long development timelines in the CNS space. Diversification here isn't just about new diseases; it's about new technologies and new therapeutic modalities. The financial context for this expansion is set by their latest filings.

As of September 30, 2025, Voyager Therapeutics, Inc. reported cash, cash equivalents, and marketable securities of $229 million. This cash position, following a restructuring in Q2 2025, is expected to provide a runway into 2028. This runway is crucial as they pursue these diversification strategies while managing elevated operating costs; for instance, Research and Development expenses were $35.9 million for the third quarter of 2025, up from $30.2 million in the same period of 2024. The net loss for the nine months ending September 30, 2025, was $92.3 million.

Here is a snapshot of the recent financial performance:

Metric (as of Q3 2025 or 9M 2025)	Amount	Context
Cash, Cash Equivalents, Marketable Securities (Sep 30, 2025)	$229 million	Provides runway into 2028
Collaboration Revenue (Q3 2025)	$13.4 million	Compared to $24.6 million in Q3 2024
R&D Expenses (Q3 2025)	$35.9 million	Increase due to VY7523 trial and VY1706 program spend
Net Loss (Nine Months Ended Sep 30, 2025)	$92.3 million	Increased from $65.0 million for FY 2024
Potential Non-Dilutive Milestones	Up to $2.4 billion	Includes up to $35 million from FA/GBA programs entering clinic

The company is actively diversifying its pipeline and technology application, which maps to several Ansoff Matrix diversification strategies.

Platform Expansion Beyond Core CNS Targets

Voyager Therapeutics, Inc. is using its established partnerships to push its technology into areas that are not strictly the company's primary Alzheimer's or Parkinson's disease focus. This is evident in the advancement of partnered programs:

• The Neurocrine-partnered Friedreich's ataxia (FA) gene therapy program is anticipated to enter clinical trials in 2026. There are approximately 5,000 patients living with FA in the US.
• The Neurocrine-partnered GBA1 gene therapy program, which targets both Gaucher disease (a non-CNS rare disease) and Parkinson's disease, is also expected to start clinical trials in 2026.
• These two programs alone have associated regulatory and clinical milestones totaling $35 million for Voyager, which is part of the larger potential non-dilutive capital of up to $2.4 billion.

While the TRACER platform is primarily known for crossing the blood-brain barrier (BBB) for CNS delivery, these FA and GBA1 programs represent a diversification into non-Alzheimer's/Parkinson's neurological rare diseases using the core gene therapy engine.

Diversification into Non-Viral Delivery Modalities

The introduction of the Voyager NeuroShuttle platform represents a significant diversification in delivery technology, moving beyond their established AAV capsid platform (TRACER) to a non-viral approach. This platform is designed to transport multiple modalities across the BBB, such as therapeutic antibodies, enzymes, ASOs, and siRNAs. Initial murine proof-of-concept studies for the ALPL-VYGR-NeuroShuttle demonstrated sustained brain expression over three weeks. This platform diversification helps de-risk the reliance on AAV vectors and expands the types of therapeutics Voyager can pursue within the CNS space.

Diversification into Small Molecule Development

Voyager Therapeutics, Inc. is diversifying its therapeutic modality by entering a collaboration with Transition Bio on November 10, 2025, to develop selective small molecules. This move is a clear diversification away from their genetics-focused gene therapy base. The initial focus is on treating Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD) by targeting TDP-43 pathology, which is present in over 90% of ALS cases. While the initial targets are neurological, this represents a diversification in the type of drug being developed.

Partnering and Asset Strategy

The existing partnerships already show a degree of diversification across therapeutic areas and partners. Voyager currently has programs advancing with Alexion, AstraZeneca Rare Disease; Novartis Pharma AG; and Neurocrine Biosciences, Inc.. The Novartis relationship saw a recent pruning, with Novartis discontinuing two discovery-stage programs, though rights returned to Voyager and this did not impact the cash runway guidance. The company is actively building its pipeline, having added a fourth wholly-owned Alzheimer's disease program, the APOE program, in June 2025. The strategy hinges on leveraging the TRACER engine, which has the potential to support non-CNS applications like ophthalmology or cardiovascular programs, though specific deals in those areas aren't detailed in the Q3 2025 results; the FA and GBA1 programs serve as the current concrete examples of non-Alzheimer's/Parkinson's expansion.

Finance: review the Q4 2025 projected milestone receipts against the $229 million cash balance by end of January 2026.