The gene therapy market is projected to grow at an annualized rate of 45%, till 2030 ..

Roots Analysis has done a detailed study on Gene Therapy Market (3rd Edition), 2019-2030, covering key aspects of the industry’s evolution and identifying potential future growth opportunities.
To order this 670+ page report, which features 190+ figures and 340+ tables, please visit this link 
Key Market Insights 
  • In the past four years, more than 31,000 patents related to gene therapies and gene editing have been filed / granted; this is indicative of the heightened pace of research in this domain
  • Presently, there are more than 10 approved gene therapies; over 465 product candidates are being evaluated for the treatment of a variety of disease indications
  • Most of the therapeutic leads are in the early stages of clinical development; a variety of viral and non-viral vectors are being used to introduce different types of gene modifications in such therapies
  • Although start-ups and mid-sized companies are spearheading the innovation, several big biopharmaceutical companies are also actively engaged
  • With multiple approved products and several under development, price is one of the major concerns in this market; the future is likely to witness the establishment of more affordable pricing and reimbursement strategies
  • As several candidates progress towards approval, developers are exploring diverse commercialization strategies to be implemented across different stages of a product’s launch cycle
  • CMOs offering vector manufacturing services have become an integral part of the gene therapy supply chain, owing to their ability to overcome the various associated challenges
  • Several investors, having realized the untapped opportunity within this emerging segment of genetic disorders, have invested over USD 16.5 billion across 280 instances, in the period between 2014 and 2019
  • Overall, prevalent trends indicate that the market for gene therapies is poised to grow significantly as multiple late stage molecules get commercialized in the near future for the treatment of different therapeutic areas
  • The projected future opportunity is expected to be distributed across different types of gene modifications, therapy delivery routes and key geographical regions
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Table of Contents
 

1.         PREFACE
1.1. Scope of the Report
1.2. Research Methodology
1.3. Chapter Outlines

2.         EXECUTIVE SUMMARY


3.         INTRODUCTION

3.1.       Context and Background

3.2.       Evolution of Gene Therapies

3.3.       Classification of Gene Therapies

3.3.1.    Somatic and Germline Gene Therapy

3.3.2.    Ex Vivo and In Vivo Gene Therapy

3.4.       Routes of Administration

3.5.       Mechanism of Action of Gene Therapies

3.6.       Concept of Gene Editing

3.7.       Advantages and Disadvantages of Gene Therapies

3.8.       Ethical and Social Concerns Related to Gene Therapies


3.9.       Future Constraints and Challenges Related to Gene Therapies

3.9.1.    Concerns Related to Therapy Development

3.9.2.    Concerns Related to Manufacturing

3.9.3.    Concerns Related to Commercial Viability


4.         GENE DELIVERY VECTORS

4.1.       Chapter Overview

4.2.       Viral Vectors

4.2.1     Types of Viral Vectors

4.2.1.1. Adeno-associated Viral Vectors

4.2.1.1.1. Overview

4.2.1.1.2. Design

4.2.1.1.3. Advantages

4.2.1.1.4. Limitations


4.2.1.2. Adenoviral Vectors

4.2.1.2.1. Overview

4.2.1.2.2. Design

4.2.1.2.3. Advantages

4.2.1.2.4. Limitations


4.2.1.3. Lentiviral Vectors

4.2.1.3.1. Overview

4.2.1.3.2. Design

4.2.1.3.3. Advantages

4.2.1.3.4. Limitations


4.2.1.4. Retroviral Vectors

4.2.1.4.1. Overview

4.2.1.4.2. Design

4.2.1.4.3. Advantages

4.2.1.4.4. Limitations


4.2.1.5. Other Viral Vectors

4.2.1.5.1. Alphavirus

4.2.1.5.2. Herpes Simplex Virus

4.2.1.5.3. Simian Virus

4.2.1.5.4. Vaccinia Virus


4.3.       Non-Viral Vectors

4.3.1.    Types of Non-Viral Vectors

4.3.1.1. Plasmid DNA

4.3.1.2. Liposomes, Lipoplexes and Polyplexes

4.3.1.3. Oligonucleotides


4.4.       Methods of Transfection

4.4.1.    Biolistic Method

4.4.2.    Electroporation

4.4.3.    Receptor Mediated Gene Delivery

4.4.4.    Gene Activated Matrix (GAM)


5.         REGULATORY LANDSCAPE AND REIMBURSEMENT SCENARIO

5.1.       Chapter Overview

5.2.       Regulatory Guidelines in North America

5.2.1.    The US Scenario

5.2.2.    The Canadian Scenario

5.3.       Regulatory Guidelines in Europe

5.4.       Regulatory Guidelines in Asia Pacific

5.4.1.    Chinese Scenario

5.4.2.    Japanese Scenario

5.4.3.    South Korean Scenario

5.4.4.    Australian Scenario

5.5.       Reimbursement Scenario

5.5.1.    Challenges Related to Reimbursement

5.6.       Payment Models for Gene Therapies


6.         COMPETITIVE LANDSCAPE

6.1.       Chapter Overview

6.2.       Gene Therapy Market: Clinical and Commercial Pipeline

6.2.1.    Analysis by Phase of Development

6.2.2.    Analysis by Therapeutic Area

6.2.3.    Analysis by Type of Vector Used

6.2.4.    Analysis by Type of Gene

6.2.5.    Analysis by Type of Modification

6.2.6.    Analysis by Type of Gene Therapy

6.2.7.    Analysis by Route of Administration


6.3.       Gene Therapy Market: Early Stage Pipeline

6.3.1.    Analysis by Stage of Development

6.3.2.    Analysis by Therapeutic Area

6.3.3.    Analysis by Type of Vector Used

6.3.4.    Analysis by Type of Gene

6.3.5.    Analysis by Type of Modification

6.3.6.    Analysis by Type of Gene Therapy

 

6.4.       Gene Therapy: Special Designation Awarded

6.4.1.    Analysis by Special Designation Awarded

6.5.       Key Players: Analysis by Number of Product Candidates

6.6.       Developer Landscape

6.6.1.    Distribution by Year of Establishment

6.6.2.    Distribution by Size of Developer

6.6.3.    Distribution by Geographical Location

6.7.       Regional Landscape


7.         MARKETED GENE THERAPIES

7.1.       Chapter Overview

7.2.       Gendicine® (Shenzhen Sibiono GeneTech)

7.2.1.    Company Overview

7.2.2.    Development Timeline

7.2.3.    Mechanism of Action and Vectors Used

7.2.4.    Target Indication(s)

7.2.5.    Current Status of Development

7.2.6.    Manufacturing, Dosage and Sales


7.3.       Oncorine® (Shanghai Sunway Biotech)

7.3.1.    Company Overview

7.3.2.    Development Timeline

7.3.3.    Mechanism of Action and Vectors Used

7.3.4.    Target Indication(s)

7.3.5.    Current Status of Development

7.3.6.    Manufacturing, Dosage and Sales


7.4.       Rexin-G® (Epeius Biotechnologies)

7.4.1.    Company Overview

7.4.2.    Development Timeline

7.4.3.    Mechanism of Action and Vector Used

7.4.4.    Target Indication(s)

7.4.5.    Current Status of Development

7.4.6.    Manufacturing, Dosage and Sales


7.5.       Neovasculgen® (Human Stem Cells Institute)

7.5.1.    Company Overview

7.5.2.    Development Timeline

7.5.3.    Mechanism of Action and Vectors Used

7.5.4.    Target Indication(s)

7.5.5.    Current Status of Development

7.5.6.    Manufacturing, Dosage and Sales


7.6.       Imlygic® (Amgen)

7.6.1.    Company Overview

7.6.2.    Development Timeline

7.6.3.    Mechanism of Action and Vectors Used

7.6.4.    Target Indication(s)

7.6.5.    Current Status of Development

7.6.6.    Manufacturing, Dosage and Sales


7.7.       Strimvelis® (Orchard Therapeutics)

7.7.1.    Company Overview

7.7.2.    Development Timeline

7.7.3.    Mechanism of Action and Vectors Used

7.7.4.    Target Indication(s)

7.7.5.    Current Status of Development

7.7.6.    Manufacturing, Dosage and Sales


7.8.       Invossa™ (Kolon TissueGene)

7.8.1.    Company Overview

7.8.2.    Development Timeline

7.8.3.    Mechanism of Action and Vectors Used

7.8.4.    Target Indication(s)

7.8.5.    Current Status of Development

7.8.6.    Manufacturing, Dosage and Sales


7.9.       Luxturna™ (Spark Therapeutics)

7.9.1.    Company Overview

7.9.2.    Development Timeline

7.9.3.    Mechanism of Action and Vector Used

7.9.4.    Target Indication(s)

7.9.5.    Current Status of Development

7.9.6.    Manufacturing, Dosage and Sales


7.10.     Zolgensma™ (AveXis / Novartis)

7.10.1.  Company Overview

7.10.2.  Development Timeline

7.10.3.  Mechanism of Action and Vector Used

7.10.4.  Target Indication(s)

7.10.5.  Current Status of Development

7.10.6.  Manufacturing, Dosage and Sales


7.11.     Collategene® / Beperminogene Perplasmid (AnGes)

7.11.1.  Company Overview

7.11.2.  Development Timeline

7.11.3.  Mechanism of Action and Vector Used

7.11.4.  Target Indication(s)

7.11.5.  Current Status of Development

7.11.6.  Manufacturing, Dosage and Sales


7.12.     Zyntelgo™ (bluebird bio)

7.12.1.  Company Overview

7.12.2.  Development Timeline

7.12.3.  Mechanism of Action and Vector Used

7.12.4.  Target Indication(s)

7.12.5.  Current Status of Development

7.12.6.  Manufacturing, Dosage and Sales


8.         KEY COMMERCIALIZATION STRATEGIES

8.1.       Chapter Overview

8.2.       Successful Drug Launch Strategy: ROOTS Framework

8.3.       Successful Drug Launch Strategy: Product Differentiation

8.4.       Commonly Adopted Commercialization Strategies based on Development Stage of the Product

8.5.       Approved Gene Therapies

8.6.       Key Commercialization Strategies Adopted by Companies Focused on Gene Therapy

8.6.1.    Strategies Adopted Before Therapy Approval

8.6.2.    Strategies Adopted During / Post Therapy Approval

8.7.       Concluding Remarks


9.         LATE STAGE (PHASE II/III AND ABOVE) GENE THERAPIES

9.1.       Chapter Overview

9.2.       AMT-061: Overview of Therapy, Current Development Status and Clinical Results

9.3.       BIIB111 (NSR-REP1): Overview of Therapy, Current Development Status and Clinical Results

9.4.       BIIB112 (NSR-RPGR): Overview of Therapy, Current Development Status and Clinical Results

9.5.       BMN 270 (valoctocogene roxaparvovec): Overview of Therapy, Current Development Status and Clinical Results

9.6.       E10A: Overview of Therapy, Current Development Status and Clinical Results

9.7.       FLT180a: Overview of Therapy, Current Development Status and Clinical Results

9.8.       GS010: Overview of Therapy, Current Development Status and Clinical Results

9.9.       Instiladrin®: Overview of Therapy, Current Development Status and Clinical Results

9.10.     Lenti-D™: Overview of Therapy, Current Development Status and Clinical Results

9.11.     LYS-SAF302: Overview of Therapy, Current Development Status and Clinical Results

9.12.     OTL-101: Overview of Therapy, Current Development Status and Clinical Results

9.13.     OTL-103: Overview of Therapy, Current Development Status and Clinical Results

9.14.     OTL-200: Overview of Therapy, Current Development Status and Clinical Results

9.15.     Pexa-Vec (pexastimogene devacirepvec): Overview of Therapy, Current Development Status and Clinical Results

9.16.     PF-06838435 (fidanacogene elaparvovec): Overview of Therapy, Current Development Status and Clinical Results

9.17.     ProstAtak®: Overview of Therapy, Current Development Status and Clinical Results

9.18.     SPK-8011: Overview of Therapy, Current Development Status and Clinical Results

9.19.     Toca 511 (vocimagene amiretrorepvec): Overview of Therapy, Current Development Status and Clinical Results

9.20.     VB-111 (ofranergene obadenovec): Overview of Therapy, Current Development Status and Clinical Results

9.21.     VGX-3100: Overview of Therapy, Current Development Status and Clinical Results

9.22.     Vigil®: Overview of Therapy, Current Development Status and Clinical Results

9.23.     VM202 (donaperminogene seltoplasmid): Overview of Therapy, Current Development Status and Clinical Results


10.        EMERGING TECHNOLOGIES

10.1.     Chapter Overview

10.2.     Gene Editing Technologies

10.2.1.  Overview

10.2.2.  Applications


10.3.     Emerging Gene Editing Platforms

10.3.1.  CRISPR / Cas9 System

10.3.2.  TALENs

10.3.3.  megaTAL

10.3.4.  Zinc Finger Nuclease


10.4.     Gene Expression Regulation Technologies

10.5.     Technology Platforms for Developing / Delivering Gene Therapies


11.        PROMISING THERAPEUTICS AREAS

11.1.     Chapter Overview

11.2      Analysis by Special Designations Awarded


11.3.     Autoimmune Disorders

11.3.1.  Analysis by Target Indication

11.3.2.  Analysis by Type of Vector Used


11.4.     Cardiovascular Diseases

11.4.1.  Analysis by Target Indication

11.4.2.  Analysis by Type of Vector Used

11.5.     Dermatological Disorders

11.5.1.  Analysis by Target Indication

11.5.2.  Analysis by Type of Vector Used


11.6.     Genetic Disorders

11.6.1.  Analysis by Target Indication

11.6.2.  Analysis by Type of Vector Used


11.7.     Hematological Disorders

11.7.1.  Analysis by Target Indication

11.7.2.  Analysis by Type of Vector Used


11.8.     Infectious Diseases

11.8.1.  Analysis by Target Indication

11.8.2.  Analysis by Type of Vector Used


11.9.     Metabolic Disorders

11.9.1.  Analysis by Target Indication

11.9.2.  Analysis by Type of Vector Used


11.10.   Muscle-related Diseases

11.10.1. Analysis by Target Indication

11.10.2. Analysis by Type of Vector Used


11.11.   Nervous System Disorders

11.11.1. Analysis by Target Indication

11.11.2. Analysis by Type of Vector Used


11.12.   Oncological Disorders

11.12.1. Analysis by Target Indication

11.12.2. Analysis by Type of Vector Used


11.13.   Ophthalmic Diseases

11.13.1. Analysis by Target Indication

11.13.2. Analysis by Type of Vector Used


12.        PATENT ANALYSIS

12.1.     Chapter Overview

12.2.     Gene Therapy-related Patents

12.2.1.  Scope and Methodology

12.2.1.1. Analysis by Publication Year

12.2.1.2. Analysis by Geographical Location

12.2.1.3. Analysis by CPC Classification

12.2.1.4. Emerging Focus Areas

12.2.1.5. Leading Players: Analysis by Number of Patents

12.2.1.6. Patent Benchmark Analysis

12.2.1.7. Patent Valuation Analysis


12.3.     Gene Editing-related Patents

12.3.1.  Scope and Methodology

12.3.1.1. Analysis by Publication Year

12.3.1.2. Analysis by Geographical Location


12.3.1.3. Analysis by CPC Classification

12.3.1.4. Emerging Focus Areas

12.3.1.5. Leading Players: Analysis by Number of Patents

12.3.1.6. Patent Benchmark Analysis

12.3.1.7. Patent Valuation Analysis


12.4.     Overall Intellectual Property Portfolio: Analysis by Type of Organization


13.        MERGERS AND ACQUISITIONS

13.1.     Chapter Overview

13.2.     Merger and Acquisition Models

13.3.     Gene Therapy: Mergers and Acquisitions

13.3.1.  Analysis by Year of Mergers and Acquisitions

13.3.2.  Analysis by Type of Mergers and Acquisitions

13.3.3.  Regional Analysis

13.3.3.1. Continent-wise Distribution

13.3.3.2. Intercontinental and Intracontinental Deals

13.3.3.3. Country-wise Distribution

13.3.4.  Analysis by Key Value Drivers

13.3.4.1. Analysis by Key Value Drivers and Year of Acquisition

13.3.5.  Analysis by Phase of Development of the Acquired Company’s Product

13.3.6.  Analysis by Therapeutic Area


14.        FUNDING AND INVESTMENT ANALYSIS

14.1.     Chapter Overview

14.2.     Types of Funding

14.3.     Funding and Investment Analysis

14.3.1.  Analysis by Number of Funding Instances

14.3.2.  Analysis by Amount Invested

14.3.3.  Analysis by Type of Funding

14.3.4.  Analysis by Amount Invested across Different Types of Therapies

14.3.5.  Regional Analysis by Amount Invested

14.3.6.  Most Active Players

14.3.7.  Key Investors

14.3.8.  Analysis by Stage of Development

14.4.     Concluding Remarks


15.        COST PRICE ANALYSIS

15.1.     Chapter Overview

15.2.     Gene Therapy Market: Factors Contributing to the Price of Gene Therapies

15.3.     Gene Therapy Market: Pricing Models

15.3.1.  On the Basis of Associated Product / Component Costs

15.3.2.  On the Basis of Competition

15.3.3.  On the Basis of Patient Segment

15.3.4.  On the Basis of Opinions of Industry Experts


16.        BIG PHARMA PLAYERS: ANALYSIS OF GENE THERAPY RELATED INITIATIVES

16.1.     Chapter Overview

16.2.     Top Pharmaceutical Companies

16.2.1.  Analysis by Therapeutic Area

16.2.2.  Analysis by Type of Vector Used

16.2.3.  Analysis by Type of Modification

16.2.4.  Analysis by Type of Gene Therapy

16.3.     Other Big Pharma Players


17.        MARKET FORECAST AND OPPORTUNITY ANALYSIS

17.1.     Chapter Overview

17.2.     Scope and Limitations

17.3.     Key Assumptions and Forecast Methodology

17.4.     Overall Gene Therapy Market, 2019-2030

17.4.1.  Gene Therapy Market: Analysis by Type of Gene Modification

17.4.2.  Gene Therapy Market: Analysis by Type of Therapy

17.4.3.  Gene Therapy Market: Analysis by Type of Vector Used

17.4.4.  Gene Therapy Market: Analysis by Therapeutic Area

17.4.5.  Gene Therapy Market: Analysis by Route of Administration

17.4.6.  Gene Therapy Market: Analysis by Geography


17.5.     Gene Therapy Market: Value Creation Analysis


17.6.     Gene Therapy Market: Product-wise Sales Forecasts

17.6.1.  Gendicine®

17.6.1.1. Target Patient Population

17.6.1.2. Sales Forecast

17.6.1.3. Net Present Value

17.6.1.4. Value Creation Analysis


17.6.2.  Oncorine®

17.6.2.1. Target Patient Population

17.6.2.2. Sales Forecast

17.6.2.3. Net Present Value

17.6.2.4. Value Creation Analysis


17.6.3.  Rexin-G®

17.6.3.1. Target Patient Population

17.6.3.2. Sales Forecast

17.6.3.3. Net Present Value

17.6.3.4. Value Creation Analysis


17.6.4.  Neovasculgen®

17.6.4.1. Target Patient Population

17.6.4.2. Sales Forecast

17.6.4.3. Net Present Value

17.6.4.4. Value Creation Analysis


17.6.5.  Strimvelis®

17.6.5.1. Target Patient Population

17.6.5.2. Sales Forecast

17.6.5.3. Net Present Value

17.6.5.4. Value Creation Analysis


17.6.6.  Imlygic®

17.6.6.1. Target Patient Population

17.6.6.2. Sales Forecast

17.6.6.3. Net Present Value

17.6.6.4. Value Creation Analysis


17.6.7.  Invossa™

17.6.7.1. Target Patient Population

17.6.7.2. Sales Forecast

17.6.7.3. Net Present Value

17.6.7.4. Value Creation Analysis


17.6.8.  Luxturna™

17.6.8.1. Target Patient Population

17.6.8.2. Sales Forecast

17.6.8.3. Net Present Value

17.6.8.4. Value Creation Analysis


17.6.9.  Zolgensma™

17.6.9.1. Target Patient Population

17.6.9.2. Sales Forecast

17.6.9.3. Net Present Value

17.6.9.4. Value Creation Analysis


17.6.10. Collategene® / Beperminogene Perplasmid

17.6.10.1. Target Patient Population

14.6.10.2. Sales Forecast

17.6.10.3. Net Present Value

17.6.10.4. Value Creation Analysis


17.6.11. Zyntelgo™

17.6.11.1. Target Patient Population

17.6.11.2. Sales Forecast

17.6.11.3. Net Present Value

17.6.11.4. Value Creation Analysis


17.6.12. AMT-061

17.6.12.1. Target Patient Population

17.6.12.2. Sales Forecast

17.6.12.3. Net Present Value

17.6.12.4. Value Creation Analysis


17.6.13. BIIB111

17.6.13.1. Target Patient Population

17.6.13.2. Sales Forecast

17.6.13.3. Net Present Value

17.6.13.4. Value Creation Analysis


17.6.14. BIIB112

17.6.14.1. Target Patient Population

17.6.14.2. Sales Forecast

17.6.14.3. Net Present Value

17.6.14.4. Value Creation Analysis


17.6.15. BMN 270

17.6.15.1. Target Patient Population

17.6.15.2. Sales Forecast

17.6.15.3. Net Present Value

17.6.15.4. Value Creation Analysis


17.6.16. E10A

17.6.16.1. Target Patient Population

17.6.16.2. Sal

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