In Vivo Gene Therapy for Rare Diseases Market
完整報告名稱與涵蓋範圍
In Vivo Gene Therapy for Rare Diseases Market Size, Share & Trends Analysis Report, By Vector Platform (AAV Vectors, Non-Viral and LNP Delivery, In Vivo Gene Editing), By Indication (Neuromuscular Disorders, Hemophilia and Blood Disorders, Inherited Retinal and Ophthalmic Diseases, Metabolic and Liver Disorders, CNS and Other Rare Diseases), By Route of Administration (Systemic Intravenous, Local and Targeted Delivery (Intrathecal, Subretinal, Intramuscular)), By End User, By Region, and Segment Forecasts, 2026-2035
報告摘要
The in vivo gene therapy for rare diseases market covers genetic medicines administered directly to the patient to add, replace or edit genes for rare inherited disorders, including AAV-based products, non-viral and LNP-delivered constructs and in vivo gene editing, across neuromuscular, hematologic, ophthalmic, metabolic and central nervous system indications. Ex vivo cell-based gene therapies and gene therapies for common diseases are excluded.
Approved products for spinal muscular atrophy, Duchenne muscular dystrophy, hemophilia, inherited retinal dystrophy and dystrophic epidermolysis bullosa have established the commercial model, yet uptake has varied widely with durability data, safety events, treatment-center readiness and payer willingness to fund one-time therapies priced in the millions. The pipeline is expanding with capsid engineering for tissue targeting and lower doses, redosable non-viral platforms, in vivo editing approaches that reached pivotal trials, and outcomes-based and annuity payment models that address affordability. Manufacturing cost per dose and long-term safety monitoring remain central to commercial viability.
The United States accounts for most revenue given approval breadth and pricing, with Europe following through country-level reimbursement and Japan and China expanding access. Competition combines large pharmaceutical groups that acquired gene therapy platforms, rare-disease specialists and editing-technology companies.
The InsightAce study delivers revenue forecasts in US$ across all vector platform, indication, route of administration and end user and regional segments for 2026-2035, alongside segment-level trend analysis, competitive landscape assessment and detailed company profiling.
授權報價
| Single User | $4,500 USD |
| Enterprise / Global Site Licence | $9,500 USD |
目錄 Table of Contents
Chapter 1. Methodology and Scope
1.1. Research Methodology
1.2. Research Scope & Assumptions
Chapter 2. Executive Summary
Chapter 3. Global In Vivo Gene Therapy for Rare Diseases Market Snapshot
Chapter 4. Global In Vivo Gene Therapy for Rare Diseases Market Variables, Trends & Scope
4.1. Market Segmentation & Scope
4.2. Market Drivers
4.3. Market Challenges
4.4. Emerging Trends
4.5. Investment and Funding Analysis
4.6. Industry Analysis - Porter's Five Forces Analysis
4.7. Competitive Landscape and Market Positioning
4.8. Market Opportunity Analysis
Chapter 5. Market Segmentation 1: By Vector Platform Estimates & Trend Analysis
5.1. Vector Platform & Market Share, 2026 & 2035
5.2. Market Size (Value US$ Mn) & Forecasts and Trend Analysis, 2026 to 2035, by Vector Platform:
5.2.1. AAV Vectors
5.2.2. Non-Viral and LNP Delivery
5.2.3. In Vivo Gene Editing
Chapter 6. Market Segmentation 2: By Indication Estimates & Trend Analysis
6.1. Indication & Market Share, 2026 & 2035
6.2. Market Size (Value US$ Mn) & Forecasts and Trend Analysis, 2026 to 2035, by Indication:
6.2.1. Neuromuscular Disorders
6.2.2. Hemophilia and Blood Disorders
6.2.3. Inherited Retinal and Ophthalmic Diseases
6.2.4. Metabolic and Liver Disorders
6.2.5. CNS and Other Rare Diseases
Chapter 7. Market Segmentation 3: By Route of Administration Estimates & Trend Analysis
7.1. Route of Administration & Market Share, 2026 & 2035
7.2. Market Size (Value US$ Mn) & Forecasts and Trend Analysis, 2026 to 2035, by Route of Administration:
7.2.1. Systemic Intravenous
7.2.2. Local and Targeted Delivery (Intrathecal, Subretinal, Intramuscular)
Chapter 8. Market Segmentation 4: By End User Estimates & Trend Analysis
8.1. End User & Market Share, 2026 & 2035
8.2. Market Size (Value US$ Mn) & Forecasts and Trend Analysis, 2026 to 2035, by End User:
8.2.1. Specialized Treatment Centers
8.2.2. Academic Medical Centers
Chapter 9. In Vivo Gene Therapy for Rare Diseases Market: Regional Estimates & Trend Analysis
9.1. North America
9.1.1. United States
9.1.2. Canada
9.2. Europe
9.2.1. Germany
9.2.2. United Kingdom
9.2.3. France
9.2.4. Italy
9.2.5. Spain
9.2.6. Rest of Europe
9.3. Asia Pacific
9.3.1. China
9.3.2. Japan
9.3.3. India
9.3.4. South Korea
9.3.5. Australia
9.3.6. Southeast Asia
9.3.7. Rest of Asia Pacific
9.4. Latin America
9.4.1. Brazil
9.4.2. Mexico
9.4.3. Rest of Latin America
9.5. Middle East & Africa
9.5.1. GCC Countries
9.5.2. South Africa
9.5.3. Rest of Middle East & Africa
Chapter 10. Competitive Landscape
10.1. Company Market Positioning Analysis
10.2. Strategic Developments (Partnerships, Expansions, Product Launches, M&A)
Chapter 11. Company Profiles
11.1. Novartis AG
11.2. Sarepta Therapeutics, Inc.
11.3. Roche Holding AG (Spark Therapeutics)
11.4. BioMarin Pharmaceutical Inc.
11.5. Pfizer Inc.
11.6. CSL Limited (uniQure Partnership)
11.7. uniQure N.V.
11.8. Ultragenyx Pharmaceutical Inc.
11.9. PTC Therapeutics, Inc.
11.10. Regenxbio Inc.
11.11. Intellia Therapeutics, Inc.
11.12. 4D Molecular Therapeutics, Inc.
11.13. Astellas Pharma Inc.
11.14. Rocket Pharmaceuticals, Inc.
11.15. Krystal Biotech, Inc.
圖表清單 List of Tables & Figures
Table 1. Global In Vivo Gene Therapy for Rare Diseases Market Revenue Forecast by Vector Platform, 2026-2035 (US$ Mn)
Figure 1. Global In Vivo Gene Therapy for Rare Diseases Market Segmentation and Scope Overview
Table 2. Global In Vivo Gene Therapy for Rare Diseases Market Revenue Forecast by Indication, 2026-2035 (US$ Mn)
Figure 2. Global In Vivo Gene Therapy for Rare Diseases Market Share by Vector Platform, 2026 & 2035
Table 3. Global In Vivo Gene Therapy for Rare Diseases Market Revenue Forecast by Route of Administration, 2026-2035 (US$ Mn)
Figure 3. Approved and Late-Stage In Vivo Gene Therapies by Indication and Vector Platform
Table 4. Global In Vivo Gene Therapy for Rare Diseases Market Revenue Forecast by End User, 2026-2035 (US$ Mn)
Figure 4. Uptake Determinants: Durability Evidence, Safety Events and Treatment-Center Readiness
Table 5. Global In Vivo Gene Therapy for Rare Diseases Market Revenue Forecast by Region, 2026-2035 (US$ Mn)
Figure 5. Asia Pacific In Vivo Gene Therapy for Rare Diseases Growth Opportunity Analysis
Table 6. North America In Vivo Gene Therapy for Rare Diseases Market Revenue by Country, 2026-2035 (US$ Mn)
Figure 6. Competitive Positioning of Key In Vivo Gene Therapy for Rare Diseases Market Participants
Table 7. Europe In Vivo Gene Therapy for Rare Diseases Market Revenue by Country, 2026-2035 (US$ Mn)
Figure 7. Porter's Five Forces Analysis of the Global In Vivo Gene Therapy for Rare Diseases Market
Table 8. Asia Pacific In Vivo Gene Therapy for Rare Diseases Market Revenue by Country, 2026-2035 (US$ Mn)
Figure 8. Payment Model Innovation for One-Time Rare Disease Therapies by Region
Table 9. Strategic Developments of Leading In Vivo Gene Therapy for Rare Diseases Market Participants, 2023-2026
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