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遺伝子治療薬の世界市場:市場機会・臨床試験の分析 (2026年)

Global Gene Therapy Market Opportunity & Clinical Trials Insight 2026

発行 PNS Pharma 商品コード 923286
出版日 ページ情報 英文 1500 Pages
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遺伝子治療薬の世界市場:市場機会・臨床試験の分析 (2026年) Global Gene Therapy Market Opportunity & Clinical Trials Insight 2026
出版日: 2020年01月29日 ページ情報: 英文 1500 Pages
概要

他の治療法で対応できない疾患への遺伝子治療アプローチの潜在性は急激に現実味を帯び、かつてないブレークスルー薬の商業使用に対する承認が待たれています。

当レポートでは、世界の遺伝子治療薬の市場を調査し、遺伝子治療薬の概要、開発の経緯、作用機序、上市済み製品の概要、主な製品と適応症、臨床試験の企業・適応症・フェーズ別の動向、主要地域市場における動向、市場影響因子の分析、主要企業のプロファイルなどをまとめています。

第1章 イントロダクション:遺伝子治療薬

  • 遺伝子治療薬:概要
  • 開発の経緯

第2章 遺伝子治療薬・遺伝子変異への影響

第3章 遺伝子治療薬の作用機序

第4章 遺伝子治療薬に利用可能な分子ツール

第5章 遺伝子治療薬戦略

第6章 遺伝子治療薬と臨床試験

第7章 癌への遺伝子治療アプローチ

  • 癌細胞への遺伝子治療薬の臨床的有効性
  • GendicineR
  • Rexin-GR
  • KymriahR
  • YescartaR
  • OncorineR
  • ImlygicR

第8章 神経筋疾患の遺伝子治療薬:脊髄性筋萎縮症 (SMA)・デュシェンヌ型筋ジストロフィー (DMD)

  • 遺伝子治療薬の臨床的アプローチ:デュシェンヌ型筋ジストロフィー (DMD)
  • 遺伝子治療薬の臨床的アプローチ:脊髄性筋萎縮症 (SMA)
  • SpinrazaR
  • ZolgensmaR
  • Eteplirsen

第9章 希少遺伝性網膜変性の第一標的治療

  • 臨床的有効性
  • LuxturnaR

第10章 末梢動脈疾患治療と遺伝子治療

  • 臨床的干渉
  • NeovasculgenR

第11章 重症複合免疫不全症治療:アデノシンデアミナーゼ (ADA) 欠損症

  • 遺伝子治療薬アプローチの臨床的潜在性
  • StrimvelisR

第12章 遺伝子治療薬:その他の疾患における成果

第13章 遺伝子治療薬のアベイラビリティ・回転

第14章 投与量・価格の分析

  • KymriahR
  • YescartaR
  • SpinrazaR
  • ZolgensmaR
  • Luxturna
  • Strimvelis
  • Eteplirsen
  • Imlygic

第15章 臨床試験の分析

  • フェーズ別
  • 国別
  • 配合別
  • 企業別
  • 標的別
  • 適応症別

第16章 臨床試験の動向:企業・適応症・フェーズ別

  • 研究
  • 前臨床
  • 臨床
  • 第0相
  • 第I相
  • 第I/II相
  • 第II相
  • 第II/III相
  • 第III相
  • 申請済み
  • 承認済み

第17章 上市済み薬の臨床的考察

  • YESCARTA
  • Kymriah
  • ZOLGENSMA
  • Gendicine
  • IMLYGIC
  • Glybera
  • INVOSSA
  • LUXTURNA
  • Neovasculgen
  • Zalmoxis
  • Rexin-G

第18章 市場環境

  • 市場概要
  • 地域分析
    • 米国
    • 欧州
    • 日本
    • 中国
    • インド
    • 韓国
    • 台湾

第19章 合併・提携

第20章 市場力学

  • 成長推進因子
  • 課題
  • 将来の市場機会

第21章 競合環境

図表

List of Figures

  • Figure 1-1: Basic Gene Therapy Technique
  • Figure 1-2: Milestones in Gene-Based Therapies
  • Figure 2-1: Molecular Requirements for Conducting Gene Therapy
  • Figure 2-2: Number of Protocols Approved for Gene Therapy, 1999 & 2015
  • Figure 2-3: Global - Percentage of Gene Therapy Protocols at Different Phases
  • Figure 3-1: General Procedure for Performing Gene Therapy
  • Figure 3-2: Classification of Gene Therapy on the Basis of Working
  • Figure 3-3: Differentiation of Gene Therapy on the Basis of Cell Type
  • Figure 3-4: General Mechanism of Somatic Gene Therapy
  • Figure 3-5: Areas of Somatic Gene Therapy Utilization
  • Figure 3-6: Success Percentage of Somatic Gene Therapy in Different Diseases
  • Figure 3-7: General Mechanism of Germline Gene Therapy
  • Figure 3-8: Differentiation of Gene Therapy on the Basis of Gene Delivery Mechanisms
  • Figure 3-9: Ex-Vivo Delivery Mechanism for Gene Therapy
  • Figure 3-10: Methods for In-Vivo Delivery of the Gene Therapy
  • Figure 3-11: Differentiation of Gene Therapy on the Basis of Gene Delivery Methods
  • Figure 3-12: Viral Vectors for Gene Therapy
  • Figure 3-13: General Mechanism of Virus Vectors for Gene Therapy
  • Figure 3-14: Non-Viral Methods of Gene Delivery
  • Figure 3-15: Ultrasound Irradiation Leading to Membrane Pores
  • Figure 3-16: Types of Gene Therapy on the Basis of End-Results
  • Figure 3-17: Strategies Opted by Targeted Gene Therapy
  • Figure 4-1: Molecular Tools Available for Performing Gene Therapy
  • Figure 4-2: CompoZr ® - Price of a Single Kit (US$), January' 2019
  • Figure 4-3: Domains of TALE & their Respective Functions
  • Figure 4-4: Applications of CRISPR-Cas9 Gene Editing Technology
  • Figure 5-1: Overall Strategy for Gene Alteration Played by Gene Therapy
  • Figure 5-2: Gene Replacement Therapy
  • Figure 5-3: Gene Therapy Molecular Tool Participation in Fixing Defective Genes
  • Figure 5-4: Modification of Immune Cells with Gene Therapy
  • Figure 6-1: Global - Gene Therapy Clinical Trials by Diseases (%), 2019
  • Figure 6-2: Global - Number of Gene Therapy Clinical Trials by Diseases, 2020
  • Figure 6-3: Global - Clinical Trials on Monogenic Diseases (%), 2004, 2007, 2012 & 2017
  • Figure 6-4: Global - Vectors Used in Clinical Trials (%), 1989 - 2017
  • Figure 6-5: Global - Number of Gene Therapy Clinical Trials Approved, 2010 - 2018
  • Figure 6-6: Global - Types of Genes Transferred in Gene Therapy Clinical Trials (%), 2017
  • Figure 6-7: Global - Number of Different Types of Genes Transferred in Gene Therapy Clinical Trials, 2017
  • Figure 6-8: Global - Number of Gene Therapy Trials in Different Phases, January' 2020
  • Figure 6-9: Global - Number of the Gene Therapy Clinical Trials in Different Status, January'2020
  • Figure 6-10: Global - Status of Gene Therapy Clinical Trials (%), January'2020
  • Figure 6-11 : Global - Total Number of Active, Not Recruiting Gene Therapy Clinical Trials, January' 2020
  • Figure 6-12: Global - Active, Not Recruiting Gene Therapy Clinical Trials (%), January' 2020
  • Figure 6-13: Global - Total Number of Completed Gene Therapy Clinical Trials, January' 2020
  • Figure 6-14: Global - Completed Gene Therapy Clinical Trials (%), January' 2020
  • Figure 6-15: Global - Total Number of Terminated Gene Therapy Clinical Trials, January' 2020
  • Figure 6-16: Global - Terminated Gene Therapy Clinical Trials (%), January' 2020
  • Figure 6-17: Global - Total Number of Withdrawn Gene Therapy Clinical Trials, January' 2020
  • Figure 6-18: Global - Withdrawn Gene Therapy Clinical Trials (%), January' 2020
  • Figure 6-19: Global - Number of Clinical Trials with Male Participants, January'2020
  • Figure 6-20: Global - Clinical Trials with Male Participants (%), January'2020
  • Figure 6-21: Global - Number of Clinical Trials with Female Participants, January'2020
  • Figure 6-22: Global - Clinical Trials with Female Participants (%), January'2020
  • Figure 6-23: Global - Gene Therapy Clinical Trials Funding Status, January'2020
  • Figure 6-24: Global - Gene Therapy Clinical Trials Funding Status (%), January'2020
  • Figure 7-1: Oncolytic Viruses Used in Gene Therapy Clinical Trials for Cancer
  • Figure 7-2: In-Vivo & Ex-Vivo Approved Drugs for Cancer Gene Therapy
  • Figure 7-3: Strategies Adapted by Gene Therapy Products against Cancer Cells
  • Figure 7-4: Global - Kymriah® - Clinical Trials Start & Estimated Completion Year, January' 2020
  • Figure 7-5: Global - Yescarta® - Clinical Trials Start & Estimated Completion Year, January' 2020
  • Figure 7-6: Global - Imlygic® - Active Clinical Trials Start & Estimated Completion Year, January' 2020
  • Figure 8-1: Global - Population of Children below 14 Years (%), 2018
  • Figure 8-2: Global - Cases of Spinal Muscular Atrophy Type I before Birth (%), 2017
  • Figure 8-3: Global - Cases of Spinal Muscular Atrophy Type III (%), January' 2020
  • Figure 8-4: Patients with Homozygous Deletion of SMN1 Gene (%), 2017
  • Figure 8-5: Global - Origin of Spinal Muscular Atrophy Patients (%), 2017
  • Figure 8-6: Global - Number of Patients of Spinal Muscular Atrophy from Different Origins, 2017
  • Figure 8-7: Europe - Distribution of Spinal Muscular Atrophy Patients (%), 2017
  • Figure 8-8: Europe - Distribution of Spinal Muscular Atrophy Patients, 2017
  • Figure 8-9: Europe - Spinal Muscular Atrophy Patient Population (%), 2017
  • Figure 8-10: Europe - Number of Spinal Muscular Atrophy Patients, 2017
  • Figure 8-11: Global - Distribution of Patients with Spinal Muscular Atrophy Subtypes (%), 2017
  • Figure 8-12: Global - Spinal Muscular Atrophy Patients by Gender (%), 2017
  • Figure 8-13: Global - Number of Spinal Muscular Atrophy Patients by Gender, 2017
  • Figure 8-14: Global - Distribution of Spinal Muscular Atrophy Patients by Age Group (%), 2017
  • Figure 8-15: Global - Number of Spinal Muscular Atrophy Patients by Age Group, 2017
  • Figure 8-16: Age of Development of Clinical Signs for Duchenne Muscular Dystrophy, 2020
  • Figure 8-17: Mutations Leading to the Development of Duchenne Muscular Dystrophy (%), 2019
  • Figure 8-18: Strategies by Gene Therapy for Duchenne Muscular Dystrophy
  • Figure 8-19: Global - Conducted Gene Therapy Clinical Trials for Duchenne Muscular Dystrophy, January' 2020
  • Figure 8-20: Gene Therapy Strategies for Spinal Muscular Atrophy
  • Figure 8-21: Global - Spinal Muscular Atrophy - Clinical Trials Start & Estimated Completion Year, January' 2020
  • Figure 8-22: Global - Spinraza® - Active Clinical Trials for Spinal Muscular Atrophy Start & Estimated Completion Year, January' 2020
  • Figure 8-23: Global - Spinraza® - Recruiting Clinical Trials for Spinal Muscular Atrophy Start & Estimated Completion Year, January' 2020
  • Figure 8-24: Global - Spinraza® - Recruiting Clinical Trials for Spinal Muscular Atrophy II Start & Estimated Completion Year, January' 2020
  • Figure 8-25: Global - Spinraza® - Recruiting Clinical Trial for Spinal Muscular Atrophy III Start & Estimated Completion Year, January' 2020
  • Figure 8-26: Global - Zolgensma® - Active Clinical Trials for Spinal Muscular Atrophy Start & Estimated Completion Year, January' 2020
  • Figure 8-27: Global - Zolgensma® - Recruiting Clinical Trial for Spinal Muscular Atrophy Start & Estimated Completion Year, January' 2020
  • Figure 8-28: Global - Zolgensma® - Recruiting Clinical Trial for Spinal Muscular Atrophy I Start & Estimated Completion Year, January' 2020
  • Figure 8-29: Global - Eteplirsen - Active Clinical Trial Starting & Estimated Completion Year for Duchenne Muscular Dystrophy, January' 2020
  • Figure 8-30: Global - Eteplirsen - Completed Gene Therapy Clinical Trials Starting & Completion Year for Duchenne Muscular Dystrophy, January'2020
  • Figure 9-1: Gene Therapy for Retinal Dystrophy Disorder
  • Figure 9-2: Global - Estimated Cases of Retinal Dystrophy (Million), January'2020
  • Figure 9-3: Total Genes Responsible for Causing Retinal Disorders
  • Figure 9-4: Completed Gene Therapy Clinical Trials for Retinal Dystrophy - Starting & Completion Year, January'2020
  • Figure 10-1: UK - Total Population with Heart Disease (Million), 2019
  • Figure 10-2: Global - Status of Gene Therapy Clinical Trials Conducted for Cardiovascular Diseases, January'2020
  • Figure 10-3: Global - Status of Gene Therapy Clinical Trials Conducted for Cardiovascular Diseases (%), January'2020
  • Figure 10-4: Therapeutic Genes for Cardiovascular Disorders Gene Therapy Approach
  • Figure 10-5: Global - Clinical Trials for Cardiovascular Diseases - Start & Estimated Completion Year, January' 2020
  • Figure 10-6: Global - Neovasculgen® - Completed Clinical Trials, January' 2020
  • Figure 11-1: Global - Cases of Adenosine Deaminase Deficiency (%), January' 2020
  • Figure 11-2: Global - Early & Late Onset Adenosine Deaminase Deficiency Deficiency (%), January' 2020
  • Figure 11-3: Global - Adenosine Deaminase Deficiency Gene Therapy Clinical Trials Completion Years, 2020
  • Figure 11-4: Global - Starting & Completion Year of Completed Gene Therapy Clinical Trials for Adenosine Deaminase Deficiency, 2020
  • Figure 11-5: Global - Strimvelis® - Ongoing Clinical Trial Expected Starting & Completion Year for Adenosine Deaminase Deficiency, January' 2020
  • Figure 13-1: Timeline of the Approved Gene Therapy Products
  • Figure 14-1: Kymriah - Dose for Acute Lymphoblastic Leukemia for Body Weight Less than 50 kg (CAR-T Cells Million/kg), January' 2020
  • Figure 14-2: Kymriah® - Dose for Acute Lymphoblastic Leukemia for Body Weight above 50kg (CAR-T Cells Million/kg), January' 2020
  • Figure 14-3: Kymriah® - Dose for Refractory or Relapsed Lymphoblastic Leukemia (CAR-T Cells Million/kg), January' 2020
  • Figure 14-4: Kymriah® - Half Yearly Sales (US$ Million), 2018
  • Figure 14-5: Global - Kymriah® Quarterly Sales (US$ Million), Q1- Q3, 2019
  • Figure 14-6: Global - Kymriah® Sales (US$ Million), Q1 - Q3, 2018 & 2019
  • Figure 14-7: Kymriah® - Initial Approval Year by US FDA & EMA
  • Figure 14-8: Yescarta® - Dose Strength for Lymphoma (CAR-T Cells Million/kg), January' 2020
  • Figure 14-9: Global - Yescarta® Annual Sales (US$ Million), 2017 & 2018
  • Figure 14-10: Global - Yescarta® Quarterly Sales (US$ Million), Q1 - Q4, 2018
  • Figure 14-11: Regional - Yescarta® Quarterly Sales (US$ Million), Q4, 2018
  • Figure 14-12: Global - Yescarta® Quarterly Sales (US$ Million), Q1 - Q3, 2019
  • Figure 14-13: Yescarta® - Initial US FDA & EMA Approval Year
  • Figure 14-14: Spinraza® - Treatment Course with the Required Dosage (mg/day), January' 2020
  • Figure 14-15: Spinraza® - Price of 5 ml Vial & Price/ml Solution (US$), January' 2020
  • Figure 14-16: Spinraza® - Cost of Single Treatment Cycle & Annual Treatment Cost (US$), January' 2020
  • Figure 14-17: Global - Spinraza® Quarterly Sales (US$ Million), Q1 - Q4, 2018
  • Figure 14-18: Global - Spinraza® Quarterly Sales (US$ Million), Q1 - Q4, 2018 & 2019
  • Figure 14-19: Spinraza - Annual Sales - US v/s Row (US$ Million), 2018
  • Figure 14-20: Spinraza - Annual Sales - US v/s Row (%), 2018
  • Figure 14-21: Spinraza® - Initial US FDA & EMA Approval Year
  • Figure 14-22: Zolgensma® - Concentration of Vector Genome per ml Available in the Kit of 5.5 & 8.3 ml (Trillion), January' 2020
  • Figure 14-23: Zolgensma - Available Dosing Pattern (Volume) per Body Weight of the Patient, January' 2020
  • Figure 14-24: Annual Treatment Cost - Zolgensma v/s Other Therapy (US$ Million), 2019
  • Figure 14-25: Zolgensma - Initial US FDA & EMA Approval Year
  • Figure 14-26: Luxturna - Recommended Dose of the Drug (Vector Genomes Million), January' 2020
  • Figure 14-27: Luxturna - Dose Concentrations (Vector Genomes Million), January' 2020
  • Figure 14-28: Luxturna - Treatment Price for Single & Both Eyes (US$ Million), January' 2020
  • Figure 14-29: Global - Luxturna Quarterly Sales (US$ Million), Q1 - Q4, 2018
  • Figure 14-30: Global - Luxturna Quarterly Sales (US$ Million), Q1, 2018 & 2019
  • Figure 14-31: Global - Luxturna Quarterly Sales (US$ Million), Q2, 2018 & 2019
  • Figure 14-32: Global - Luxturna External Research & Development Expenses (US$ Million), Q1, 2018 & 2019
  • Figure 14-33: Global - Luxturna External Research & Development Expenses (US$ Million), Q2, 2018 & 2019
  • Figure 14-34: Luxturna - Initial US FDA & EMA Approval Year
  • Figure 14-35: Strimvelis - Minimum & Maximum Concentration (CD34+ Million Cells/ml), 2020
  • Figure 14-36: Strimvelis - Recommended Dose (CD34+ Million Cells/kg), 2020
  • Figure 14-37: Strimvelis - Research & Development Expenses (US$ Million), 2017 & 2018
  • Figure 14-38: Strimvelis - Selling, General & Administrative Expenses (US$ Million), 2017 & 2018
  • Figure 14-39: Strimvelis - Research & Development Expenses (US$ Million), Q3, 2018 - 2019
  • Figure 14-40: Strimvelis - Selling, General & Administrative Expenses (US$ Million), Q3, 2018 & 2019
  • Figure 14-41: Strimvelis - EMA Issue & Expiry Year
  • Figure 14-42: Eteplirsen - Average Recommended Dose of the Drug (mg/kg), January' 2020
  • Figure 14-43: Eteplirsen - Price of Intravenous Solution (US$), January' 2020
  • Figure 14-44: Eteplirsen - Cost of Treatment (US$), 2016
  • Figure 14-45: Global - Eteplirsen Quarterly Sale (US$ Million), Q1 - Q4, 2018
  • Figure 14-46: Global - Eteplirsen Quarterly Sales (US$ Million), Q2 - Q3, 2018 & 2019
  • Figure 14-47: Imlygic - Recommended Dose & Schedule for Treatment (pfu/ml), January' 2020
  • Figure 14-48: Imlygic - Price of Drug Injectable Suspension 1mpfu/ml (US$), January' 2020
  • Figure 14-49: Imlygic - Price of Drug Injectable Suspension 100 mpfu/ml (US$), January' 2020
  • Figure 14-50: Global - Imlygic Expected Total Sale (US$ Million), 2016 & 2022
  • Figure 15-1: Global - Gene Therapy Clinical Trials by Phase (Number),2020 till 2026
  • Figure 15-2: Global - Gene Therapy Clinical Trials by Country (Number),2020 till 2026
  • Figure 15-3: Global - Gene Therapy Clinical Trials by Formulation (Number),2020 till 2026
  • Figure 15-4: Global - Gene Therapy Clinical Trials by Company (Number),2020 till 2026
  • Figure 15-5: Global - Gene Therapy Clinical Trials by Target (Number),2020 till 2026
  • Figure 15-6: Global - Gene Therapy Clinical Trials by Indication (Number),2020 till 2026
  • Figure 18-1: Factors Leading to Significant Growth of Gene Therapy Market
  • Figure 18-2: Global - Expected Gene Therapy Market Size (US$ Million), 2018 - 2026
  • Figure 18-3: India - Patients with Diseases Treated by Gene Therapy, 2019
  • Figure 18-4: India - Patients with Diseases Treated by Gene Therapy (%), 2019
  • Figure 18-5: India - Minimum & Maximum Number of People with Rare Diseases, 2019
  • Figure 18-6: India - Minimum & Maximum Number of People with Rare Diseases (%), 2019
  • Figure 18-7: Korea - Total Number of New Cancer Cases & Cancer Deaths, 2019
  • Figure 18-8: Korea - Total Number of New Cancer Cases & Cancer Deaths, 2019
  • Figure 18-9: Korea - Number of Cancer Deaths v/s Total Population, 2019
  • Figure 20-1: Predicted Future of Gene Therapy
目次

"Global Gene Therapy Market Opportunity & Clinical Trials Insight 2026" Report Highlight:

  • Global Gene Therapy Market Opportunity: > US$ 8 Billion
  • Global Gene Therapy Clinical Pipeline: > 1000 Therapies In Clinical Trials
  • Commercially Available Gene Therapies: 11
  • USA Dominates Global Gene Therapy Pipeline: > 500 Therapies In Clinical Trials
  • Cancer Accounts For 50% of Global Gene Therapy Trials

The potential of gene therapy approach to address the diseases that are unable to get treated with any other treatment is quickly becoming a reality and the approval of gene therapy products for the commercial use is seeking for a fascinating breakthrough over the other achievements that mankind has achieved over the decades. Gene therapy brings with it the promise of one-time treatment of the target that are underlying the genetic level. The therapy has brought several fascinating companies that are currently conducting research about the trending area of biotech. Gene therapy tends to bring an increase in the baby boomer generation compelled to grow and live longer.

The research report “Global Gene Therapy Market Opportunity & Clinical Trials Insight 2026” is poised towards the current market trends followed by the therapy since its advent at international level and delivering a summarized information about the whole concept of the gene therapy in order to restore the gene functions by manipulating the genetic code present within the genome. The report discusses the current available gene therapy agents with their complete data regarding the dosage, sales and pricing compiled from multiple sources and from publications by companies that are thriving the market of gene therapy. The entire clinical and non-clinical framework along with the countries encompassed with the presence of agents is also discussed. The report highlights the need of such advanced technique and the entire huge breakthrough the technique is going through.

The whole arena of gene therapy came into limelight when the therapy treated the first patient having ADA-SCID. Since then, the fundamental benefits received from the therapy never got limited. The therapy has blessed the patients many agents with unique strategies and nonetheless many efforts made by the researchers are underway to get utilized against the diseases that have no other viable treatment. The therapy can alter the target DNA sequence by silencing, replacing or by manipulating the level of the substitute protein in order to restore the patient's body against the target disease.

As there are various diseases that are associated with cancer, therefore the technology provides a potential treatment for the patient and it is expected that the therapy will provide deep insight about the genetic diseases and its treatment in future too. For the future, the gene therapy process is believed to be at a crucial position and will undoubtedly revolutionize the clinical, preclinical and technical techniques utilized in the laboratories. The editing of specific genes present in the pool of genome is considered as a groundbreaking discovery like the discovery of vaccines and antibiotics. It is just the matter of time that the technology will be categorized as a dominating one over the other available technologies.

Gene therapy has been designed such that it can provide relief to the patients with rare genetic disease with one approach i.e. manipulation at the genetic level. One approach of the technique gets investigated for a number of results which in-turn results in the approval of several gene therapy agents in a regulated way. In exchange, the therapy also entails for a cheap and one-time treatment- single injection or infusion when compared with the many years of expensive ongoing treatment.

The advent of the therapy for the well-being of the patients has definitely shifted the timings of the healthcare costs. The novel gene therapy agents anticipation in the market has successfully determined the value of the gene therapy and its urgency. The value based research of the therapy has gained the interest of the clinicians and researchers ensuring the best of the benefits from the therapy. The comparative effectiveness and cost-effectiveness have inclined the dependency of the patients towards gene therapy while gaining excess commercial success in the global market.

Table of Contents

1. Introduction to Gene Therapy

  • 1.1. Gene Therapy: An Overview
  • 1.2. History of Development

2. Gene Therapy & its Impact on Genetic Alterations

  • 2.1. Gene Therapy as a Mastermind of Genetic Revolution
  • 2.2. Manipulations at Molecular Level by Gene Therapy

3. Working Mechanism of Gene Therapy

  • 3.1. Classification of Gene Therapy on the Basis of Cell Type
    • 3.1.1. Somatic Gene Therapy
    • 3.1.2. Germline Gene Therapy
  • 3.2. Classification on the Basis of Gene Delivery Mechanism
    • 3.2.1. Ex-Vivo Delivery of the Gene
    • 3.2.2. In-Vivo Delivery of the Gene
  • 3.3. Classification on the Basis of Gene Delivery Methods
    • 3.3.1. Viral Methods
    • 3.3.2. Non-Viral Methods
  • 3.4. Classification on the Basis of End-Results
    • 3.4.1. Augmentation Gene Therapy
    • 3.4.2. Targeted Gene Therapy

4. Molecular Tools Available for Gene Therapy

  • 4.1. Targeting Specific Loci with ZFN's
  • 4.2. Introduction of TALEN's as a Gene Therapy Tool
  • 4.3. Development of CRISPR to Mediate Precise Genome Editing

5. Strategies Opted by Gene Therapy

  • 5.1. Replacement of Defective Genes with Healthy Genes
  • 5.2. Fixing Mutated Genes by Gene Therapy Molecular Tools
  • 5.3. Gene Therapy Modifying the Diseased Cells & Making them Evident against Immune Cells

6. Gene Therapy & the Journey of Clinical Trials Associated

  • 6.1. Trends Associated with Clinical Research & Trials
    • 6.1.1. Clinical Trials Regarding Gene Therapy: Disease Analysis
    • 6.1.2. Clinical Trials Regarding Gene Therapy: Vector Analysis
    • 6.1.3. Clinical Studies Regarding Gene Therapy: Global Analysis
    • 6.1.4. Clinical Trials Regarding Gene Therapy: Gene Type Analysis
    • 6.1.5. Clinical Trials Regarding Gene Therapy: Current Status
    • 6.1.6. Clinical Trials Regarding Gene Therapy: Gender Analysis
    • 6.1.7. Clinical Trials Regarding Gene Therapy: Funding Analysis

7. Approach of Gene Therapy Against Cancer

  • 7.1. Clinical Efficacy of Gene Therapy for Cancer Cells
  • 7.2. Gendicine®: A Wide Spectrum Anti-Cancer Gene Therapy Agent
  • 7.3. Rexin-G® for Osteosarcoma & Soft Tissue Sarcoma
  • 7.4. Kymriah®: A Breakthrough Gene Therapy Product for B-Cell Acute Lymphoblastic Leukemia
  • 7.5. Yescarta®: Gene Therapeutic Approach for B-Cell Lymphoma
  • 7.6. Oncorine® Approval by Shanghai Sunway Biotech Co. Ltd.
  • 7.7. Gene Therapy Product Imlygic® against Melanoma

8. Gene Therapy for the Treatment of Neuromuscular Diseases - Spinal Muscular Atrophy & Duchenne Muscular Dystrophy

  • 8.1. Clinical Approach of Gene Therapy for Duchenne Muscular Dystrophy
  • 8.2. Clinical Approach of Gene Therapy for Spinal Muscular Atrophy
  • 8.3. Spinraza® - First Approved Gene Therapy Product for Spinal Muscular Atrophy
  • 8.4. Zolgensma® Treatment for Spinal Muscular Atrophy Patients
  • 8.5. Eteplirsen - A New Hope for the Patients with Duchenne Muscular Dystrophy

9. First Targeted Treatment for Rare Inherited Retinal Dystrophy

  • 9.1. Clinical Efficacy of the Gene Therapy Products
  • 9.2. Luxturna® for the Treatment of RPE65 Mutation Associated Retinal Dystrophy

10. Gene Therapy Mediated Treatment of Peripheral Artery Disease

  • 10.1. Clinical Interference for Cardiovascular Disorders Gene Therapy
  • 10.2. Neovasculgen® Gene Therapy in Cardiovascular Diseases

11. Correcting Severe Combined Immunodeficiency - Adenosine Deaminase Deficiency through Gene Therapy

  • 11.1. Clinical Potential of Gene Therapy Therapeutic Approach
  • 11.2. Strimvelis® Benefits for Rare Disease: Adenosine Deaminase Deficiency- Severe Combined Immunodeficiency

12. Success of Gene Therapy over Other Diseases

13. Availability & Revolution of Gene Therapy Drugs in the Market

14. Gene Therapy Products Dosage & Price Analysis

  • 14.1. Kymriah®
  • 14.2. Yescarta®
  • 14.3. Spinraza®
  • 14.4. Zolgensma®
  • 14.5. Luxturna
  • 14.6. Strimvelis
  • 14.7. Eteplirsen
  • 14.8. Imlygic

15. Global Gene Therapy Clinical Trials Insight

  • 15.1. By Phase
  • 15.2. By Country
  • 15.3. By Formulation
  • 15.4. By Company
  • 15.5. By Target
  • 15.6. By Indication

16. Global Gene Therapy Clinical Trials By Company, Indication & Phase

  • 16.1. Research
  • 16.2. Preclinical
  • 16.3. Clinical
  • 16.4. Phase-0
  • 16.5. Phase-I
  • 16.6. Phase-I/II
  • 16.7. Phase-II
  • 16.8. Phase-II/III
  • 16.9. Phase-III
  • 16.10. Preregistration
  • 16.11. Registered

17. Marketed Gene Therapy Clinical Insight

  • 17.1. YESCARTA
  • 17.2. Kymriah
  • 17.3. ZOLGENSMA
  • 17.4. Gendicine
  • 17.5. IMLYGIC
  • 17.6. Glybera
  • 17.7. INVOSSA
  • 17.8. LUXTURNA
  • 17.9. Neovasculgen
  • 17.10. Zalmoxis
  • 17.11. Rexin-G

18. Global Market Landscape of Gene Therapy

  • 18.1. Gene Therapy Market Overview
  • 18.2. Gene Therapy Market - Regional Analysis
    • 18.2.1. US
    • 18.2.2. Europe
    • 18.2.3. Japan
    • 18.2.4. China
    • 18.2.5. India
    • 18.2.6. South Korea
    • 18.2.7. Taiwan

19. Mergers & Collaborations between Major Key Players of the Market

  • 19.1. Axovant Gene Therapies & Yposkesi's Strategic Partnership
  • 19.2. Lonza and DiNAQOR AG's Strategic Collaboration
  • 19.3. US$ 1 Million to AavantiBio for Gene Therapy Development in Freidreich's Ataxia
  • 19.4. Actinium Pharmaceuticals Collaboration with UC Davis for HIV-Related Lymphoma for ACT Gene Therapy Program

20. Global Gene Therapy Market Dynamics

  • 20.1. Factors Driving the Growth of Gene Therapy
    • 20.1.1. Gene Therapy Dominant over Growing Prevalence of Cancer
    • 20.1.2. Increased R&D Activities with Rising Investments
    • 20.1.3. Increasing General Awareness Concerning Gene Therapy
  • 20.2. Challenges Overpowering the Technology
  • 20.3. Future Opportunities for Gene Therapy

21. Competitive Landscape

  • 21.1. Novartis
  • 21.2. Spark Therapeutics
  • 21.3. BioGen
  • 21.4. Sarepta Therapeutics
  • 21.5. Kite Pharma
  • 21.6. Amgen
  • 21.7. Solid Biosciences Inc.
  • 21.8. uniQure NV
  • 21.9. MeiraGTX Holdings
  • 21.10. Audentis Therapeutics
  • 21.11. Regenxbio Inc.
  • 21.12. Alnylam Pharmaceuticals
  • 21.13. Arrowhead
  • 21.14. SQZ Biotechnologies
  • 21.15. Bluerock Therapeutics
  • 21.16. Zydus Takeda
  • 21.17. Intrexon Corporation
  • 21.18. Celgene
  • 21.19. Roche
  • 21.20. Oxford Biomedica
  • 21.21. Genethon
  • 21.22. Sangamo Biosciences
  • 21.23. Juno Therapeutics
  • 21.24. Cellectis
  • 21.25. Autolus Therapeutics plc
  • 21.26. icell Gene Therapeutics
  • 21.27. Allogene Therapeutics