Table of Content

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



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