GVR Report cover Gene Therapy Market Size, Share & Trends Report

Gene Therapy Market Size, Share & Trends Analysis Report By Indication (Acute Lymphoblastic Leukemia, Large B-cell Lymphoma), By Vector Type (Lentivirus), By Route Of Administration, By Region, And Segment Forecasts, 2024 - 2030

  • Report ID: GVR-2-68038-179-5
  • Number of Report Pages: 120
  • Format: PDF, Horizon Databook
  • Historical Range: 2018 - 2023
  • Forecast Period: 2024 - 2030 
  • Industry: Healthcare

Gene Therapy Market Size & Trends

The global gene therapy market size was estimated at USD 5.54 billion in 2023 and is projected to grow at a CAGR of 18.88% from 2024 to 2030. The market growth is attributed to many factors such as the expanding area of advanced therapies along with gene delivery technologies and progressive competition among key players focused on commercialization of their therapies. The biotechnology companies are investing in acquisitions, mergers/collaborations, and deals as a key strategy to increase in-house expertise and strengthen the product pipelines.

U.S. Gene Therapy Market size and growth rate, 2024 - 2030

The COVID-19 outbreak has negatively impacted the market growth. This sector has experienced severe disruption due to COVID-19, which has historically presented significant challenges in the supply of materials, manufacturing, and logistics operations. For instance, companies had lengthy delivery times for specific components. They later discovered that it was short on clinical trial supplies when a partner contract manufacturing company was compelled to shut down.

The robust pipeline is expected to boost the market growth over the forecast period. Researchers are working to make gene therapy available at clinics. Various universities and institutes exhibit a broad portfolio of products in the pipeline which is expected to boost revenue generation over the forecast period. Clinical trials for gene therapy increased significantly from 2017 to 2018, after the FDA approved the first gene therapy. According to the American Society of Gene & Cell Therapy (ASGCT), around 1,986 products, including CAR T-cell therapies and other genetically modified cell therapies, are currently under development.

Moreover, improving regulatory support creates growth opportunities for the market over the forecast period. Several positive changes have been made by many international regulatory organizations to promote therapies. Support for CAR-T technology from the FDA is one of the examples. In phase II and III studies, in particular, regulators allow flexibility in the usual hierarchy of how clinical trials are conducted. Moreover, the FDA expects 10 to 20 new therapies to be approved annually by 2025.

Furthermore, an increase in funding and investments in this sector is expected to provide lucrative growth opportunities to market players. Several biopharma companies are investing in this sector for novel product launches. For instance, in January 2022, Ori Biotech raised more than USD 100.0 million in Series B funding to introduce a novel cell & gene therapy developing platform. This funding allowed for a rapid transition from pre-commercialization to market launch.

Market Concentration & Characteristics

Market growth of gene therapy is exponential and market growth is accelerating. The increasing number of approvals of gene therapies within North America and Europe are the major reasons for market growth. Furthermore, a strong pipeline of numerous companies is anticipated to bring new therapies to the market.

Some of the major strategies opted by the companies include increased spending on R&D and new product launches.  For instance, in December 2023, the U.S. FDA approved a gene therapy for treating sickle cell disease. This is the first gene therapy that is based on CRISPR gene editing.  Such new launches are expected to fuel the market growth over the forecast period.

Gene Therapy Market Concentration & Characteristics

The gene therapy market is highly impacted by regulations. Regional regulatory bodies such as the U.S. Food and Drug Administration, European Medicines Agency, and more are responsible for all the approvals of therapies. Lengthy approval processes and clinical trials limit the entry of new players within the market.

The market’s low to moderate product expansion is driven by several factors: rigorous regulatory challenges, high development and manufacturing costs, technical complexities in delivery systems, niche markets targeting rare diseases, and competitive patent landscapes. Additionally, high treatment costs and reimbursement issues further slow market growth.

Currently, North America and Europe are the regions that have the most approved gene therapies as of January 2024. However, in countries such as India, South Korea, and Japan, a large number of clinical trials are being undertaken for various conditions. For instance, in July 2023, a team of researchers at the Narayana Nethralaya announced that they would be starting a clinical trial for a gene therapy for ocular diseases.

Vector Insights

The AAV segment shows a significant revenue contribution of 22% in 2023. Several biopharma companies are offering their viral vector platform for the development of AAV-based gene therapy products. For instance, in September 2016, Lonza signed an exclusive agreement with Massachusetts Eye and Ear to support its novel Anc-AAV gene therapy platform for the development and commercialization of next-generation gene therapies based on their AAV platform. Similarly, RegenxBio had made an agreement with companies AveXis & Biogen in March 2014 and May 2016, respectively, which would allow both companies to use RegenxBio’s AAV vector platform for the development of gene therapy molecules.

Furthermore, in May 2021, Biogen Inc. and Capsigen Inc. entered into a strategic research partnership to engineer novel AAV capsids that can deliver transformative gene therapies, which can address the fundamental genetic causes of numerous neuromuscular and CNS disorders. In July 2021, the U.S. Department of Commerce’s National Institute of Standards and Technology (NIST), National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL), and United States Pharmacopeia (USP) announced a collaboration to evaluate analytical methods and develop standards for AAV. As part of this partnership, NIST and USP will be conducting an interlaboratory study in which several laboratories will measure these serious quality attributes, and their results will be linked and examined. This collaboration will support the development of new promising gene therapies that will significantly advance people’s lives.

Indication Insights

The spinal muscular atrophy (SMA) segment dominated the market in 2023. Although SMA is a rare disorder, it is one of the most common fatal inherited diseases of infancy. The development of Zolgensma (AVXS-101), has proven its effectiveness in treating SMA and altering the phenotype of the illness. The FDA approved Novartis' Zolgensma approval in May 2019, which is aimed at treating the underlying cause of SMA. As of now, Zolgensma is the only gene treatment in this field to have been approved. The approval of this gene therapy is evidence of the growing use of therapies to treat serious hereditary illnesses like SMA.

Global Gene Therapy Market share and size, 2023

The Beta-Thalassemia Major/SCD segment is anticipated to register the fastest CAGR over the forecast period. Gene therapy for SCD and β-thalassemia is based on the transplantation of gene-modified hematopoietic stem cells. Clinical and preclinical studies have shown the efficacy and safety of this therapeutic modality. However, several other factors, such as suboptimal gene expression levels & gene transfer efficiency, limited stem-cell dose and quality, and toxicity of myeloablative regimens are still hampering its efficacy.

Despite these challenges, in June 2019, bluebird Bio’s Zynteglo (formerly LentiGlobin) received conditional approval in Europe for the treatment of β-thalassemia and is expected to receive U.S. FDA approval in August 2022. Moreover, the product has already received Orphan Drug status by the U.S. FDA for the treatment of patients with sickle cell disease (SCD). Furthermore, in April 2021, Vertex Pharmaceuticals and CRISPR Therapeutics amended the partnership for the development, production, and commercialization of CTX001 in sickle beta thalassemia and cell disease. These achievements in this segment are anticipated to significantly boost the adoption of the product in this segment.

Route of Administration Insights

The intravenous segment dominated the global gene therapy market in 2023, primarily due to its effectiveness in delivering treatments directly into the bloodstream, ensuring widespread distribution and rapid uptake by target cells. This method's ability to treat a wide range of conditions, coupled with advancements in vector design and delivery mechanisms, has made intravenous administration the preferred choice for many gene therapies, contributing significantly to its market leadership. The segment is also expected to emerge as the most lucrative over the forecast period.

Regional Insights

North America dominated the market in 2023 with the largest revenue share of 18.95% in 2023. This region is expected to become the largest routine manufacturer of gene therapy in terms of the number of approvals and revenue generated during the forecast period. Increasing investments in R&D from large and small companies in the development of ideal therapy drugs are anticipated to further boost the market.

Gene Therapy Market Trends, by Region, 2024 - 2030

U.S. Gene Therapy Market Trends

The U.S. is the largest market for clinical trials related to gene therapy, as around 50% of all clinical trials in the world are conducted in the country. As of March 2023, more than 2,500 clinical studies are under investigation in the U.S. alone.

Europe Gene Therapy Market Trends

Europe gene therapy market was identified as a lucrative region in this industry. This is attributed to its large population with unmet medical needs and increasing demand for novel technologies in the treatment of rare but increasingly prevalent diseases. Asia Pacific market for commercial application of genetic therapies is anticipated to witness significant growth in the forecast period, which can be attributed to the easy availability of resources, local presence of major companies, and increased investment, by the governments.

The UK gene therapy market is anticipated to witness accelerated growth over the forecast period, due to increased investments by various big companies and governments, including the NHS & research laboratories.

The gene therapy market in Germany is anticipated to grow significantly over the forecast period. In January 2020, a U.S.-based company-bluebird bio-launched its first gene therapy for transfusion-dependent beta-thalassemia (blood disorder) in Germany. apceth Biopharma- a German manufacturer-is working with bluebird for the production of Zynteglo, the gene therapy launched by bluebird.

Asia Pacific Gene Therapy Market Trends

The Asia Pacific gene therapy market held a significant CAGR of 18.40% over the forecast period. This upsurge is attributed to the high birth rate and high prevalence of genetic disorders because of various factors, such as consanguineous marriages and founder mutations.

The gene therapy market in China is expected to grow over the forecast period. In March 2019, Merck KgaA collaborated with GenScript- a Chinese biotech company-for the manufacturing of plasmids and viral vectors

Key Gene Therapy Company Insights

The market players operating in the gene therapy market are adopting product approval to increase the reach of their products in the market and improve the availability of their products in diverse geographical areas, along with expansion as a strategy to enhance production/research activities. In addition, several market players are acquiring smaller players to strengthen their market position. This strategy enables companies to increase their capabilities, expand their product portfolios, and improve their competencies.

Key Gene Therapy Companies:

The following are the leading companies in the gene therapy market. These companies collectively hold the largest market share and dictate industry trends.

  • Amgen Inc.
  • Novartis AG
  • F. Hoffmann-La Roche
  • Gilead Sciences, Inc.
  • bluebird bio, Inc.
  • Bristol-Myers Squibb Company
  • Legend Biotech.
  • BioMarin.
  • uniQure N.V.
  • Merck & Co.
  • Sarepta Therapeutics, Inc.
  • Krystal Biotech, Inc.
  • CRISPR Therapeutics.

Recent Developments

  • In January 2024, Biogen and Ginkgo Bioworks announced the completion of their gene therapy collaboration involving AAV based vectors. This is expected to fuel the demand for gene therapies in the coming years.

  • In December 2023, the Swiss Agency for Therapeutic Products granted approval to Libmeldy for the treatment of early-onset metachromatic leukodystrophy.

  • In May 2023, Krystal Biotech was granted approval for VYJUVEK gene therapy for Dystrophic Epidermolysis Bullosa treatment

  • In June 2023, the U.S. FDA granted approval to Sarepta for  ELEVIDYS gene therapy to treat DMD in children of age 4-5 years

  • In January 2023, Voyager Therapeutics and Neurocrine Biosciences entered into a strategic collaboration for the commercialization & development of Voyager’s GBA1 program and other next-generation gene therapies for neurological diseases

  • In January 2023, Spark Therapeutics and Neurochase established a strategic collaboration to develop Neurochase’s unique delivery technology for use with selected gene treatments for rare disorders in the CNS. In this agreement, Neurochase will contribute its extensive knowledge in direct drug delivery technology to Spark’s premier AAV platform.

  • In January 2022, 64x Bio, a U.S.-based biotech company, raised USD 55.0 million in funding to advance its gene therapy manufacturing platform. This initiative was expected to expand the company’s VectorSelect platform.

Gene Therapy Market Report Scope

Report Attribute

Details

Market size value in 2024

USD 6.45 billion

Revenue forecast in 2030

USD 18.20 billion

Growth rate

CAGR of 18.88% from 2024 to 2030

Actual data

2018 - 2023

Forecast period

2024 - 2030

Quantitative units

Revenue in USD million/billion, CAGR from 2024 to 2030

Report coverage

Revenue forecast, company ranking, competitive landscape, growth factors, trends

Segments covered

Vector type, indication, route of administration, region

Regional scope

North America; Europe; Asia Pacific; Rest of World

Country scope

U.S.; Canada; Mexico; UK; Germany; Switzerland; Japan; China; South Korea; Australia

Key companies profiled

Amgen Inc.; Novartis AG; F. Hoffmann-La Roche; Gilead Sciences, Inc.; bluebird bio, Inc.; Bristol-Myers Squibb Company; Legend Biotech.; BioMarin.; uniQure N.V.; Merck & Co.; Sarepta Therapeutics, Inc.; Krystal Biotech, Inc.; CRISPR Therapeutics.

Customization scope

Free report customization (equivalent up to 8 analyst’s working days) with purchase. Addition or alteration to country, regional & segment scope.

Global Gene Therapy Market Report Segmentation

This report forecasts revenue growth at global, regional, and country levels in addition to provides an analysis of the latest industry trends in each of the subsegments from 2018 to 2030. For this study, Grand View Research has segmented the global gene therapy market report based on indication, vector type, route of administration, and region:

Gene Therapy Market Report Segmentation

  • Indication Outlook (Revenue, USD Million, 2018 - 2030)

    • Large B-Cell Lymphoma

    • Multiple Myeloma

    • Spinal Muscular Atrophy (SMA)

    • Acute Lymphoblastic Leukemia (ALL)

    • Melanoma (lesions)

    • Inherited Retinal Disease

    • Beta-Thalassemia Major/SCD

    • Others

  • Vector Type Outlook (Revenue, USD Million, 2018 - 2030)

    • Lentivirus

    • RetroVirus & gamma RetroVirus

    • AAV

    • Modified Herpes Simplex Virus

    • Adenovirus

    • Others

  • Route of Administration Outlook (Revenue, USD Million, 2018 - 2030)

    • Intravenous

    • Others

  • Regional Outlook (Revenue, USD Million, 2018 - 2030)

    • North America

      • U.S.

      • Canada

      • Mexico

    • Europe

      • UK

      • Germany

      • Switzerland

    • Asia Pacific

      • Japan

      • China

      • South Korea

      • Australia

    • Rest of the world 

Frequently Asked Questions About This Report

Table of Contents

Chapter 1. Methodology and Scope
                    1.1. Market Segmentation and Scope
                    1.2. Market Definitions
                        1.2.1. Indication
                        1.2.2. Route of Administration
                        1.2.3. Vector Type
                    1.3. Information analysis
                    1.4. Market formulation & data visualization
                    1.5. Data validation & publishing
                    1.6. Information Procurement
                    1.7. Primary Research
                    1.8. Information or Data Analysis
                    1.9. Market Formulation & Validation
                    1.10. Market Model
                    1.11. Objectives
Chapter 2. Executive Summary
                    2.1. Market Snapshot
                    2.2. Segment Snapshot
                    2.3. Competitive Landscape Snapshot
Chapter 3. Market Variables, Trends, & Scope
                    3.1. Market Lineage Outlook
                        3.1.1. Parent Market Outlook
                        3.1.2. Related/Ancillary Market Outlook
                    3.2. Market Dynamics
                        3.2.1. Market Driver Analysis
                            3.2.1.1. Robust Gene Therapy Pipeline
                            3.2.1.2. Introduction Of Technological Advancements
                            3.2.1.3. Increasing Investment From Companies And Partnerships
                            3.2.1.4. Growing Prevalence Of Target Diseases And Increased Demand For Innovative Medicine
                        3.2.2. Market Restraint Analysis
                            3.2.2.1. Absence Of Effective Diagnosis Framework
                            3.2.2.2. High Prices Of Gene Therapy
                        3.2.3. Market Opportunity Analysis
                            3.2.3.1. Rising Investment For Adoptive T-Cell Transfer Approaches Of Disease Treatment
                            3.2.3.2. Facility Expansion For Cell And Gene Therapies
                            3.2.3.3. Technological Advancements In Manufacturing Vectors
                    3.3. Industry Analysis Tools
                        3.3.1. Porter’s Five Forces Analysis
                        3.3.2. PESTEL Analysis
                        3.3.3. COVID-19 Impact Analysis
Chapter 4. Route of Administration Business Analysis
                    4.1. Gene Therapy Market: Route of Administration Movement Analysis
                    4.2. Intravenous
                        4.2.1. Intravenous Market, 2018 - 2030 (USD Million)
                    4.3. Others
                        4.3.1. Others Market, 2018 - 2030 (USD Million)
Chapter 5. Indication Business Analysis
                    5.1. Gene Therapy Market: Indication Movement Analysis
                    5.2. Acute Lymphoblastic Leukemia
                        5.2.1. Acute Lymphoblastic Leukemia Market, 2018 - 2030 (USD Million)
                    5.3. Inherited Retinal Disease
                        5.3.1. Inherited Retinal Disease Market, 2018 - 2030 (USD Million)
                    5.4. Large B-Cell Lymphoma
                        5.4.1. Large B-Cell Lymphoma Market, 2018 - 2030 (USD Million)
                    5.5. Melanoma (lesions)
                        5.5.1. Melanoma (lesions) Market, 2018 - 2030 (USD Million)
                    5.6. Beta-Thalassemia Major/Sickle Cell Disease (Scd)
                        5.6.1. Beta-Thalassemia Major/Sickle Cell Disease (Scd) Market, 2018 - 2030 (USD Million)
                    5.7. Spinal Muscular Atrophy (SMA)
                        5.7.1. Spinal Muscular Atrophy (SMA) Market, 2018 - 2030 (USD Million)
                    5.8. Others
                        5.8.1. Others Market, 2018 - 2030 (USD Million)
Chapter 6. Vector Type Business Analysis
                    6.1. Gene Therapy Market: Vector Type Movement Analysis
                    6.2. Lentiviral Vectors
                        6.2.1. Lentiviral Vectors market, 2018 - 2030 (USD Million)
                    6.3. Adeno-Associated Viral (Aav) Vectors
                        6.3.1. Adeno-Associated Viral (Aav) Vectors market, 2018 - 2030 (USD Million)
                    6.4. RetroVirus & gamma RetroVirus
                        6.4.1. RetroVirus & gamma RetroVirus market, 2018 - 2030 (USD Million)
                    6.5. Modified Herpes Simplex Virus
                        6.5.1. Modified Herpes Simplex Virus market, 2018 - 2030 (USD Million)
                    6.6. Adenovirus Vectors
                        6.6.1. Adenovirus Vectors market, 2018 - 2030 (USD Million)
                    6.7. Others
                        6.7.1. Others market, 2018 - 2030 (USD Million)
Chapter 7. Regional Business Analysis
                    7.1. Gene Therapy Market Share By Region, 2023 & 2030
                    7.2. North America
                        7.2.1. North America Gene Therapy Market, 2018 - 2030 (USD Million)
                        7.2.2. U.S.
                            7.2.2.1. Key Country Dynamics
                            7.2.2.2. Target disease prevalence
                            7.2.2.3. Competitive Scenario
                            7.2.2.4. Regulatory Framework
                            7.2.2.5. U.S. Gene Therapy Market, 2018 - 2030 (USD Million)
                        7.2.3. Canada
                            7.2.3.1. Key Country Dynamics
                            7.2.3.2. Target disease prevalence
                            7.2.3.3. Competitive Scenario
                            7.2.3.4. Regulatory Framework
                            7.2.3.5. Canada Gene Therapy Market, 2018 - 2030 (USD Million)
                        7.2.4. Mexico
                            7.2.4.1. Key Country Dynamics
                            7.2.4.2. Target disease prevalence
                            7.2.4.3. Competitive Scenario
                            7.2.4.4. Regulatory Framework
                            7.2.4.5. Mexico Gene Therapy Market, 2018 - 2030 (USD Million)
                    7.3. Europe
                        7.3.1. Europe Gene Therapy Market, 2018 - 2030 (USD Million)
                        7.3.2. UK
                            7.3.2.1. Key Country Dynamics
                            7.3.2.2. Target disease prevalence
                            7.3.2.3. Competitive Scenario
                            7.3.2.4. Regulatory Framework
                            7.3.2.5. UK Gene Therapy Market, 2018 - 2030 (USD Million)
                        7.3.3. Germany
                            7.3.3.1. Key Country Dynamics
                            7.3.3.2. Target disease prevalence
                            7.3.3.3. Competitive Scenario
                            7.3.3.4. Regulatory Framework
                            7.3.3.5. Germany Gene Therapy Market, 2018 - 2030 (USD Million)
                        7.3.4. Switzerland
                            7.3.4.1. Key Country Dynamics
                            7.3.4.2. Target disease prevalence
                            7.3.4.3. Competitive Scenario
                            7.3.4.4. Regulatory Framework
                            7.3.4.5. Switzerland Gene Therapy Market, 2018 - 2030 (USD Million)
                    7.4. Asia Pacific
                        7.4.1. Asia Pacific Gene Therapy Market, 2018 - 2030 (USD Million)
                        7.4.2. Japan
                            7.4.2.1. Key Country Dynamics
                            7.4.2.2. Target disease prevalence
                            7.4.2.3. Competitive Scenario
                            7.4.2.4. Regulatory Framework
                            7.4.2.5. Japan Gene Therapy Market, 2018 - 2030 (USD Million)
                        7.4.3. China
                            7.4.3.1. Key Country Dynamics
                            7.4.3.2. Target disease prevalence
                            7.4.3.3. Competitive Scenario
                            7.4.3.4. Regulatory Framework
                            7.4.3.5. China Gene Therapy Market, 2018 - 2030 (USD Million)
                        7.4.4. Australia
                            7.4.4.1. Key Country Dynamics
                            7.4.4.2. Target disease prevalence
                            7.4.4.3. Competitive Scenario
                            7.4.4.4. Regulatory Framework
                            7.4.4.5. Australia Gene Therapy Market, 2018 - 2030 (USD Million)
                    7.5. Rest of the world
                        7.5.1. Rest of the world Gene Therapy Market, 2018 - 2030 (USD Million)
Chapter 8. Competitive Landscape
                    8.1. Company Categorization
                    8.2. Strategy Mapping
                    8.3. Company Market Position Analysis, 2023
                    8.4. Company Profiles
                        8.4.1. Amgen Inc.
                            8.4.1.1. Overview
                            8.4.1.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
                            8.4.1.3. Product Benchmarking
                            8.4.1.4. Strategic Initiatives
                        8.4.2. Novartis AG
                            8.4.2.1. Overview
                            8.4.2.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
                            8.4.2.3. Product Benchmarking
                            8.4.2.4. Strategic Initiatives
                        8.4.3. F. Hoffmann-La Roche
                            8.4.3.1. Overview
                            8.4.3.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
                            8.4.3.3. Product Benchmarking
                            8.4.3.4. Strategic Initiatives
                        8.4.4. Gilead Sciences, Inc.
                            8.4.4.1. Overview
                            8.4.4.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
                            8.4.4.3. Product Benchmarking
                            8.4.4.4. Strategic Initiatives
                        8.4.5. bluebird bio, Inc.
                            8.4.5.1. Overview
                            8.4.5.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
                            8.4.5.3. Product Benchmarking
                            8.4.5.4. Strategic Initiatives
                        8.4.6. Bristol-Myers Squibb Company
                            8.4.6.1. Overview
                            8.4.6.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
                            8.4.6.3. Product Benchmarking
                            8.4.6.4. Strategic Initiatives
                        8.4.7. Legend Biotech.
                            8.4.7.1. Overview
                            8.4.7.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
                            8.4.7.3. Product Benchmarking
                            8.4.7.4. Strategic Initiatives
                        8.4.8. BioMarin.
                            8.4.8.1. Overview
                            8.4.8.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
                            8.4.8.3. Product Benchmarking
                            8.4.8.4. Strategic Initiatives
                        8.4.9. uniQure N.V.
                            8.4.9.1. Overview
                            8.4.9.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
                            8.4.9.3. Product Benchmarking
                            8.4.9.4. Strategic Initiatives
                        8.4.10. Merck & Co.
                            8.4.10.1. Overview
                            8.4.10.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
                            8.4.10.3. Product Benchmarking
                            8.4.10.4. Strategic Initiatives
                        8.4.11. Sarepta Therapeutics, Inc.
                            8.4.11.1. Overview
                            8.4.11.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
                            8.4.11.3. Product Benchmarking
                            8.4.11.4. Strategic Initiatives
                        8.4.12. Krystal Biotech, Inc
                            8.4.12.1. Overview
                            8.4.12.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
                            8.4.12.3. Product Benchmarking
                            8.4.12.4. Strategic Initiatives
                        8.4.13. CRISPR Therapeutics.
                            8.4.13.1. Overview
                            8.4.13.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
                            8.4.13.3. Product Benchmarking
                            8.4.13.4. Strategic Initiatives


List of Tables

Table 1 List of Secondary Sources
Table 2 List of Tables
Table 3 List of Secondary Sources
Table 4 List of Abbreviations
Table 5 Global Gene Therapy Market by Route Of Administration, 2018 - 2030 (USD Million)
Table 6 Global Gene Therapy Market by Vector Type, 2018 - 2030 (USD Million)
Table 7 Global Gene Therapy Market by Indication, 2018 - 2030 (USD Million)
Table 8 Global Gene Therapy Market by Region, 2018 - 2030 (USD Million)
Table 9 North America Gene Therapy Market by Route Of Administration, 2018 - 2030 (USD Million)
Table 10 North America Gene Therapy Market by Country, 2018 - 2030 (USD Million)
Table 11 North America Gene Therapy Market by Vector Type, 2018 - 2030 (USD Million)
Table 12 North America Gene Therapy Market by Indication, 2018 - 2030 (USD Million)
Table 13 U.S. Gene Therapy Market by Vector Type, 2018 - 2030 (USD Million)
Table 14 U.S. Gene Therapy Market by Route Of Administration, 2018 - 2030 (USD Million)
Table 15 U.S. Gene Therapy Market by Indication, 2018 - 2030 (USD Million)
Table 16 Canada Gene Therapy Market by Vector Type, 2018 - 2030 (USD Million)
Table 17 Canada Gene Therapy Market by Route Of Administration, 2018 - 2030 (USD Million)
Table 18 Canada Gene Therapy Market by Indication, 2018 - 2030 (USD Million)
Table 19 Europe Gene Therapy Market by Country, 2018 - 2030 (USD Million)
Table 20 Europe Gene Therapy Market by Route Of Administration, 2018 - 2030 (USD Million)
Table 21 Europe Gene Therapy Market by Vector Type, 2018 - 2030 (USD Million)
Table 22 Europe Gene Therapy Market by Indication, 2018 - 2030 (USD Million)
Table 23 Germany Gene Therapy Market by Route Of Administration, 2018 - 2030 (USD Million)
Table 24 Germany Gene Therapy Market by Vector Type, 2018 - 2030 (USD Million)
Table 25 Germany Gene Therapy Market by Indication, 2018 - 2030 (USD Million)
Table 26 UK Gene Therapy Market by Route Of Administration, 2018 - 2030 (USD Million)
Table 27 UK Gene Therapy Market by Vector Type, 2018 - 2030 (USD Million)
Table 28 UK Gene Therapy Market by Indication, 2018 - 2030 (USD Million)
Table 29 Switzerland Gene Therapy Market by Route Of Administration, 2018 - 2030 (USD Million)
Table 30 Switzerland Gene Therapy Market by Vector Type, 2018 - 2030 (USD Million)
Table 31 Switzerland Gene Therapy Market by Indication, 2018 - 2030 (USD Million)
Table 32 Asia Pacific Gene Therapy Market by Route Of Administration, 2018 - 2030 (USD Million)
Table 33 Asia Pacific Gene Therapy Market by Country, 2018 - 2030 (USD Million)
Table 34 Asia Pacific Gene Therapy Market by Vector Type, 2018 - 2030 (USD Million)
Table 35 Asia Pacific Gene Therapy Market by Indication, 2018 - 2030 (USD Million)
Table 36 China Gene Therapy Market by Vector Type, 2018 - 2030 (USD Million)
Table 37 China Gene Therapy Market by Route Of Administration, 2018 - 2030 (USD Million)
Table 38 China Gene Therapy Market by Indication, 2018 - 2030 (USD Million)
Table 39 Japan Gene Therapy Market by Vector Type, 2018 - 2030 (USD Million)
Table 40 Japan Gene Therapy Market by Route Of Administration, 2018 - 2030 (USD Million)
Table 41 Japan Gene Therapy Market by Indication, 2018 - 2030 (USD Million)
Table 42 South Korea Gene Therapy Market by Vector Type, 2018 - 2030 (USD Million)
Table 43 South Korea Gene Therapy Market by Route Of Administration, 2018 - 2030 (USD Million)
Table 44 South Korea Gene Therapy Market by Indication, 2018 - 2030 (USD Million)
Table 45 Australia Gene Therapy Market by Vector Type, 2018 - 2030 (USD Million)
Table 46 Australia Gene Therapy Market by Route Of Administration, 2018 - 2030 (USD Million)
Table 47 Australia Gene Therapy Market by Indication, 2018 - 2030 (USD Million)
Table 48 RoW Gene Therapy Market by Vector Type, 2018 - 2030 (USD Million)
Table 49 RoW Gene Therapy Market by Route Of Administration, 2018 - 2030 (USD Million)
Table 50 RoW Gene Therapy Market by Indication, 2018 - 2030 (USD Million)


List of Figures

Fig. 1 Information Procurement
Fig. 2 Primary Research Pattern
Fig. 3 Market Research Approaches
Fig. 4 Market Formulation & Validation
Fig. 5 Gene Therapy Market Segmentation
Fig. 6 Porter’s Five Forces Analysis
Fig. 7 Regional Marketplace: Key Takeaways
Fig. 8 Global Gene Therapy Market for Lentivirus, 2018 - 2030 (USD Million)
Fig. 9 Global Gene Therapy Market for AAV, 2018 - 2030 (USD Million)
Fig. 10 Global Gene Therapy Market for RetroVirus & gamma RetroVirus, 2018 - 2030 (USD Million)
Fig. 11 Global Gene Therapy Market for Modified Herpes Simplex Virus, 2018 - 2030 (USD Million)
Fig. 12 Global Gene Therapy Market for Adenovirus, 2018 - 2030 (USD Million)
Fig. 13 Global Gene Therapy Market for Other Vector Type, 2018 - 2030 (USD Million)
Fig. 14 Global Gene Therapy Market for Acute Lymphoblastic Leukemia (ALL), 2018 - 2030 (USD Million)
Fig. 15 Global Gene Therapy Market for Inherited Retinal Disease, 2018 - 2030 (USD Million)
Fig. 16 Global Gene Therapy Market for Large B-cell Lymphoma, 2018 - 2030 (USD Million)
Fig. 17 Global Gene Therapy Market for Melanoma (lesions), 2018 - 2030 (USD Million)
Fig. 18 Global Gene Therapy Market for Beta-Thalassemia Major/SCD, 2018 - 2030 (USD Million)
Fig. 19 Global Gene Therapy Market for Spinal Muscular Atrophy (SMA), 2018 - 2030 (USD Million)
Fig. 20 Global Gene Therapy Market for Others, 2018 - 2030 (USD Million)
Fig. 21 Global Gene Therapy Market for Intravenous, 2018 - 2030 (USD Million)
Fig. 22 Global Gene Therapy Market for Others, 2018 - 2030 (USD Million)
Fig. 23 Regional Outlook, 2022 & 2030
Fig. 24 North America Gene Therapy Market 2018 - 2030 (USD Million)
Fig. 25 U.S. Gene Therapy Market 2018 - 2030 (USD Million)
Fig. 26 Canada Gene Therapy Market 2018 - 2030 (USD Million)
Fig. 27 Europe Gene Therapy Market 2018 - 2030 (USD Million)
Fig. 28 Germany Gene Therapy Market 2018 - 2030 (USD Million)
Fig. 29 UK Gene Therapy Market 2018 - 2030 (USD Million)
Fig. 30 Switzerland Gene Therapy Market 2018 - 2030 (USD Million)
Fig. 31 Asia Pacific Gene Therapy Market 2018 - 2030 (USD Million)
Fig. 32 Japan Gene Therapy Market 2018 - 2030 (USD Million)
Fig. 33 China Gene Therapy Market 2018 - 2030 (USD Million)
Fig. 34 Australia Gene Therapy Market 2018 - 2030 (USD Million)
Fig. 35 South Korea Gene Therapy Market 2018 - 2030 (USD Million)
Fig. 36 Rest of the world Gene Therapy Market 2018 - 2030 (USD Million)

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Market segmentation

  • Gene Therapy Indication Outlook (Revenue, USD Million, 2018 - 2030)
    • Acute Lymphoblastic Leukemia (ALL)
    • Inherited Retinal Disease
    • Large B-Cell Lymphoma
    • Melanoma (lesions)
    • Beta-Thalassemia Major/SCD
    • Spinal Muscular Atrophy (SMA)
    • Others
  • Gene Therapy Route of Administration Outlook (Revenue, USD Million; 2018 - 2030)
    • Intravenous
    • Others
  • Gene Therapy Vector Type Outlook (Revenue, USD Million; 2018 - 2030)
    • Lentivirus
    • AAV
    • RetroVirus & gamma RetroVirus
    • Modified Herpes Simplex Virus
    • Adenovirus
    • Others
  • Gene Therapy Regional Outlook (Revenue, USD Million, 2018 - 2030)
    • North America
      • North America Gene Therapy Market, by Indication
        • Acute Lymphoblastic Leukemia (ALL)
        • Inherited Retinal Disease
        • Large B-Cell Lymphoma
        • Melanoma (lesions)
        • Beta-Thalassemia Major/SCD
        • Spinal Muscular Atrophy (SMA)
        • Others
      • North America Gene Therapy Market, by Vector Type
        • Lentivirus
        • AAV
        • RetroVirus & gamma RetroVirus
        • Modified Herpes Simplex Virus
        • Adenovirus
        • Others
      • North America Gene Therapy Market, by Route of Administration
        • Intravenous
        • Others
      • U.S.
        • U.S. Gene Therapy Market, by Indication
          • Acute Lymphoblastic Leukemia (ALL)
          • Inherited Retinal Disease
          • Large B-Cell Lymphoma
          • Melanoma (lesions)
          • Beta-Thalassemia Major/SCD
          • Spinal Muscular Atrophy (SMA)
          • Others
        • U.S. Gene Therapy Market, by Vector Type
          • Lentivirus
          • AAV
          • RetroVirus & gamma RetroVirus
          • Modified Herpes Simplex Virus
          • Adenovirus
          • Others
        • U.S. Gene Therapy Market, by Route of Administration
          • Intravenous
          • Others
      • Canada
        • Canada Gene Therapy Market, by Indication
          • Acute Lymphoblastic Leukemia (ALL)
          • Inherited Retinal Disease
          • Large B-Cell Lymphoma
          • Melanoma (lesions)
          • Beta-Thalassemia Major/SCD
          • Spinal Muscular Atrophy (SMA)
          • Others
        • Canada Gene Therapy Market, by Vector Type
          • Lentivirus
          • AAV
          • RetroVirus & gamma RetroVirus
          • Modified Herpes Simplex Virus
          • Adenovirus
          • Others
        • Canada Gene Therapy Market, by Route of Administration
          • Intravenous
          • Others
    • Europe
      • Europe Gene Therapy Market, by Indication
        • Acute Lymphoblastic Leukemia (ALL)
        • Inherited Retinal Disease
        • Large B-Cell Lymphoma
        • Melanoma (lesions)
        • Beta-Thalassemia Major/SCD
        • Spinal Muscular Atrophy (SMA)
        • Others
      • Europe Gene Therapy Market, by Vector Type
        • Lentivirus
        • AAV
        • RetroVirus & gamma RetroVirus
        • Modified Herpes Simplex Virus
        • Adenovirus
        • Others
      • Europe Gene Therapy Market, by Route of Administration
        • Intravenous
        • Others
      • Germany
        • Germany Gene Therapy Market, by Indication
          • Acute Lymphoblastic Leukemia (ALL)
          • Inherited Retinal Disease
          • Large B-Cell Lymphoma
          • Melanoma (lesions)
          • Beta-Thalassemia Major/SCD
          • Spinal Muscular Atrophy (SMA)
          • Others
        • Germany Gene Therapy Market, by Vector Type
          • Lentivirus
          • AAV
          • RetroVirus & gamma RetroVirus
          • Modified Herpes Simplex Virus
          • Adenovirus
          • Others
        • Germany Gene Therapy Market, by Route of Administration
          • Intravenous
          • Others
      • UK
        • UK Gene Therapy Market, by Indication
          • Acute Lymphoblastic Leukemia (ALL)
          • Inherited Retinal Disease
          • Large B-Cell Lymphoma
          • ADA-SCID
          • Melanoma (lesions)
          • Beta-Thalassemia Major/SCD
          • Spinal Muscular Atrophy (SMA)
          • Others
        • UK Gene Therapy Market, by Vector Type
          • Lentivirus
          • AAV
          • RetroVirus & gamma RetroVirus
          • Modified Herpes Simplex Virus
          • Adenovirus
          • Others
        • UK Gene Therapy Market, by Route of Administration
          • Intravenous
          • Others
      • Switzerland
        • Switzerland Gene Therapy Market, by Indication
          • Acute Lymphoblastic Leukemia (ALL)
          • Inherited Retinal Disease
          • Large B-Cell Lymphoma
          • Melanoma (lesions)
          • Beta-Thalassemia Major/SCD
          • Spinal Muscular Atrophy (SMA)
          • Others
        • Switzerland Gene Therapy Market, by Vector Type
          • Lentivirus
          • AAV
          • RetroVirus & gamma RetroVirus
          • Modified Herpes Simplex Virus
          • Adenovirus
          • Others
        • Switzerland Gene Therapy Market, by Route of Administration
          • Intravenous
          • Others
    • Asia Pacific
      • Asia Pacific Gene Therapy Market, by Indication
        • Acute Lymphoblastic Leukemia (ALL)
        • Inherited Retinal Disease
        • Large B-Cell Lymphoma
        • Melanoma (lesions)
        • Beta-Thalassemia Major/SCD
        • Spinal Muscular Atrophy (SMA)
        • Others
      • Asia Pacific Gene Therapy Market, by Vector Type
        • Lentivirus
        • AAV
        • RetroVirus & gamma RetroVirus
        • Modified Herpes Simplex Virus
        • Adenovirus
        • Others
      • Asia Pacific Gene Therapy Market, by Route of Administration
        • Intravenous
        • Others
      • China
        • China Gene Therapy Market, by Indication
          • Acute Lymphoblastic Leukemia (ALL)
          • Inherited Retinal Disease
          • Large B-Cell Lymphoma
          • Melanoma (lesions)
          • Beta-Thalassemia Major/SCD
          • Spinal Muscular Atrophy (SMA)
          • Others
        • China Gene Therapy Market, by Vector Type
          • Lentivirus
          • AAV
          • RetroVirus & gamma RetroVirus
          • Modified Herpes Simplex Virus
          • Adenovirus
          • Others
        • China Gene Therapy Market, by Route of Administration
          • Intravenous
          • Others
      • Japan
        • Japan Gene Therapy Market, by Indication
          • Acute Lymphoblastic Leukemia (ALL)
          • Inherited Retinal Disease
          • Large B-Cell Lymphoma
          • Melanoma (lesions)
          • Beta-Thalassemia Major/SCD
          • Spinal Muscular Atrophy (SMA)
          • Others
        • Japan Gene Therapy Market, by Vector Type
          • Lentivirus
          • AAV
          • RetroVirus & gamma RetroVirus
          • Modified Herpes Simplex Virus
          • Adenovirus
          • Others
        • Japan Gene Therapy Market, by Route of Administration
          • Intravenous
          • Others
      • Australia
        • Australia Gene Therapy Market, by Indication
          • Acute Lymphoblastic Leukemia (ALL)
          • Inherited Retinal Disease
          • Large B-Cell Lymphoma
          • Melanoma (lesions)
          • Beta-Thalassemia Major/SCD
          • Spinal Muscular Atrophy (SMA)
          • Others
        • Australia Gene Therapy Market, by Vector Type
          • Lentivirus
          • AAV
          • RetroVirus & gamma RetroVirus
          • Modified Herpes Simplex Virus
          • Adenovirus
          • Others
        • Australia Gene Therapy Market, by Route of Administration
          • Intravenous
          • Others
      • South Korea
        • South Korea Gene Therapy Market, by Indication
          • Acute Lymphoblastic Leukemia (ALL)
          • Inherited Retinal Disease
          • Large B-Cell Lymphoma
          • Melanoma (lesions)
          • Beta-Thalassemia Major/SCD
          • Spinal Muscular Atrophy (SMA)
          • Others
        • South Korea Gene Therapy Market, by Vector Type
          • Lentivirus
          • AAV
          • RetroVirus & gamma RetroVirus
          • Modified Herpes Simplex Virus
          • Adenovirus
          • Others
        • South Korea Gene Therapy Market, by Route of Administration
          • Intravenous
          • Others

Report content

Qualitative Analysis

  • Industry overview
  • Industry trends
  • Market drivers and restraints
  • Market size
  • Growth prospects
  • Porter’s analysis
  • PESTEL analysis
  • Key market opportunities prioritized
  • Competitive landscape
    • Company overview
    • Financial performance
    • Product benchmarking
    • Latest strategic developments

Quantitative Analysis

  • Market size, estimates, and forecast from 2018 to 2030
  • Market estimates and forecast for product segments up to 2030
  • Regional market size and forecast for product segments up to 2030
  • Market estimates and forecast for application segments up to 2030
  • Regional market size and forecast for application segments up to 2030
  • Company financial performance
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Research Methodology

Grand View Research employs a comprehensive and iterative research methodology focused on minimizing deviance in order to provide the most accurate estimates and forecast possible. The company utilizes a combination of bottom-up and top-down approaches for segmenting and estimating quantitative aspects of the market. In Addition, a recurring theme prevalent across all our research reports is data triangulation that looks market from three different perspectives. Critical elements of the methodology employed for all our studies include:

Preliminary data mining

Raw market data is obtained and collated on a broad front. Data is continuously filtered to ensure that only validated and authenticated sources are considered. In addition, data is also mined from a host of reports in our repository, as well as a number of reputed paid databases. For a comprehensive understanding of the market, it is essential to understand the complete value chain, and in order to facilitate this; we collect data from raw material suppliers, distributors as well as buyers.

Technical issues and trends are obtained from surveys, technical symposia, and trade journals. Technical data is also gathered from an intellectual property perspective, focusing on white space and freedom of movement. Industry dynamics with respect to drivers, restraints, pricing trends are also gathered. As a result, the material developed contains a wide range of original data that is then further cross-validated and authenticated with published sources.

Statistical model

Our market estimates and forecasts are derived through simulation models. A unique model is created customized for each study. Gathered information for market dynamics, technology landscape, application development, and pricing trends are fed into the model and analyzed simultaneously. These factors are studied on a comparative basis, and their impact over the forecast period is quantified with the help of correlation, regression, and time series analysis. Market forecasting is performed via a combination of economic tools, technological analysis, industry experience, and domain expertise.

Econometric models are generally used for short-term forecasting, while technological market models are used for long-term forecasting. These are based on an amalgamation of the technology landscape, regulatory frameworks, economic outlook, and business principles. A bottom-up approach to market estimation is preferred, with key regional markets analyzed as separate entities and integration of data to obtain global estimates. This is critical for a deep understanding of the industry as well as ensuring minimal errors. Some of the parameters considered for forecasting include:

    • Market drivers and restraints, along with their current and expected impact
    • Raw material scenario and supply v/s price trends
    • Regulatory scenario and expected developments
    • Current capacity and expected capacity additions up to 2030

We assign weights to these parameters and quantify their market impact using weighted average analysis, to derive an expected market growth rate.

Primary validation

This is the final step in estimating and forecasting for our reports. Exhaustive primary interviews are conducted, face to face as well as over the phone to validate our findings and assumptions used to obtain them. Interviewees are approached from leading companies across the value chain including suppliers, technology providers, domain experts, and buyers so as to ensure a holistic and unbiased picture of the market. These interviews are conducted across the globe, with language barriers overcome with the aid of local staff and interpreters. Primary interviews not only help in data validation but also provide critical insights into the market, current business scenario, and future expectations and enhance the quality of our reports. All our estimates and forecast are verified through exhaustive primary research with Key Industry Participants (KIPs) which typically include:

    • Market-leading companies
    • Raw material suppliers
    • Product distributors
    • Buyers

The key objectives of primary research are as follows:

    • To validate our data in terms of accuracy and acceptability
    • To gain an insight in to the current market and future expectations

Data Collection Matrix

Perspective

Primary research

Secondary research

Supply-side

  • Manufacturers
  • Technology distributors and wholesalers
  • Company reports and publications
  • Government publications
  • Independent investigations
  • Economic and demographic data

Demand-side

  • End-user surveys
  • Consumer surveys
  • Mystery shopping
  • Case studies
  • Reference customers


Industry Analysis Matrix

Qualitative analysis

Quantitative analysis

  • Industry landscape and trends
  • Market dynamics and key issues
  • Technology landscape
  • Market opportunities
  • Porter’s analysis and PESTEL analysis
  • Competitive landscape and component benchmarking
  • Policy and regulatory scenario
  • Market revenue estimates and forecast up to 2030
  • Market revenue estimates and forecasts up to 2030, by technology
  • Market revenue estimates and forecasts up to 2030, by application
  • Market revenue estimates and forecasts up to 2030, by type
  • Market revenue estimates and forecasts up to 2030, by component
  • Regional market revenue forecasts, by technology
  • Regional market revenue forecasts, by application
  • Regional market revenue forecasts, by type
  • Regional market revenue forecasts, by component

 

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