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The gene therapy market is projected to grow at an annualized rate of 30%, till 2030

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May 2, 2022

Roots Analysis has done a detailed study on Gene Therapy Market (4th Edition), 2020-2030, covering key aspects of the industry’s evolution and identifying potential future growth opportunities.

To order this 720+ page report, which features 220+ figures and 375+ tables, please visit this – https://www.rootsanalysis.com/reports/view_document/gene-therapies-market/268.html

Key Market Insights

• The gene therapy market is characterized by a healthy drug pipeline, with 10 approved molecules and over 820 product candidates being evaluated for the treatment of multiple disease indications, worldwide
• Most of the contemporary therapeutic leads are currently in the early stages of clinical development; a variety of viral and non-viral vectors are being used across different therapeutic approaches for gene therapies
• Around 400 gene therapy focused clinical trials have been registered across the globe; most of these trials are evaluating the therapies for the treatment of oncological indications
• In order to tap into the lucrative opportunity offered by gene therapies, big pharma players have adopted various approaches, from proprietary product development to strategic investments
• Several patents related to gene therapies and affiliated technologies have been filed / granted; the value of this intellectual capital is being realized through both licensing agreements and marketing exclusivity
• In the recent past, a considerable increase in the M&A activity has been observed among industry stakeholders; this trend can be attributed to the ongoing capability / portfolio consolidation efforts of involved players
• Foreseeing a lucrative return, many public and private investors have made investments worth over USD 25 billion, across more than 350 instances, in the period between 2015 and 2020
• Case Study: CMOs offering vector manufacturing services have become an integral part of the gene therapy supply chain, owing to their ability to overcome the associated challenges
• Treatment cost is one of the major concerns in this market; we anticipate more affordable pricing and reimbursement strategies to evolve in due course
• Prevalent trends indicate that the demand for gene therapies is poised to grow significantly as more late-stage therapies get commercialized in the short team using different vectors and type of therapies
• The projected future opportunity is expected to be distributed across different therapeutic areas, types of therapies, types of therapeutic approaches, routes of administrations and key geographical regions
• To monetize the full potential, stakeholders are already actively exploring diverse commercialization strategies, across different stages of a product’s launch cycle

For more information, please visit https://www.rootsanalysis.com/reports/view_document/gene-therapies-market/268.html

Table of Contents

1. PREFACE
   1.1. Scope of the Report
   1.2.      Research Methodology
   1.3.      Key Questions Answered

1.4.      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 Therapies

3.3.2.   Ex Vivo and In Vivo Therapies

3.4.      Routes of Administration

3.5.      Mechanism of Action of Gene Therapies

3.6.      Overview of the Basic Concepts of Gene Editing

3.6.1.   Evolution of Genome Editing

3.6.2.   Applications of Genome Editing

3.6.3.   Genome Editing Techniques

3.7.      Advantages and Disadvantages of Gene Therapies

3.8.      Ethical and Social Concerns Related to Gene Therapies

3.9.      Constraints and Challenges Related to Gene Therapies

3.9.1.   Therapy Development Concerns

3.9.2.   Manufacturing Concerns

3.9.3.   Commercial Viability Concerns

4. GENE DELIVERY VECTORS
   4.1.      Chapter Overview

4.2.      Viral and Non-Viral Methods of Gene Transfer

4.3.      Viral Vectors for Genetically Modified Therapies

4.4.      Types of Viral Vectors

4.4.1.   Adeno-associated Viral Vectors

4.4.1.1.            Overview

4.4.1.2.            Design and Manufacturing

4.4.1.3.            Advantages

4.4.1.4.            Limitations

4.4.2.   Adenoviral Vectors

4.4.2.1.            Overview

4.4.2.2.            Design and Manufacturing

4.4.2.3.            Advantages

4.4.2.4.            Limitations

4.4.3.   Lentiviral Vectors

4.4.3.1.            Overview

4.4.3.2.            Design and Manufacturing

4.4.3.3.            Advantages

4.4.3.4.            Limitations

4.4.4.   Retroviral Vectors

4.4.4.1.            Overview

4.4.4.2.            Design and Manufacturing

4.4.4.3.            Advantages

4.4.4.4.            Limitations

4.4.5.   Other Viral Vectors

4.4.5.1.            Alphavirus

4.4.5.2.            Foamy Virus

4.4.5.3.            Herpes Simplex Virus

4.4.5.4.            Sendai Virus

4.4.5.5.            Simian Virus

4.4.5.6.            Vaccinia Virus

4.5.      Types of Non-Viral Vectors

4.5.1.   Plasmid DNA

4.5.2.   Liposomes, Lipoplexes and Polyplexes

4.5.3.   Oligonucleotides

4.5.4.   Other Non-Viral Vectors

4.5.5.   Gene Delivery Using Non-Viral Vectors

4.5.5.1.            Biolistic Methods

4.5.5.2.            Electroporation

4.5.5.3.            Receptor Mediated Gene Delivery

4.5.5.4.            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.   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

5.4.5.   Hong Kong Scenario

5.5.      Reimbursement Scenario

5.5.1.   Challenges Related to Reimbursement

5.6.      Commonly Offered Payment Models for Gene Therapies

6. MARKET OVERVIEW
   6.1. Chapter Overview

6.1.1.   Analysis by Phase of Development of Gene Therapy

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 Therapeutic Approach

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 Phase 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 Therapeutic Approach

6.3.6.   Analysis by Type of Gene Therapy

6.2.7.   Analysis by Route of Administration

6.4.      Gene Therapy Candidates: Special Designations

6.4.1.   Analysis by Special Designation(s) Awarded

6.5.      Grid Representation: Analysis by Phase of Development, Therapeutic Area and Therapeutic Approach

7. COMPETITIVE LANDSCAPE
   7.1.      Chapter Overview

7.2.      Gene Therapy Market: List of Developers

7.2.1.   Analysis by Year of Establishment

7.2.2.   Analysis by Company Size

7.2.3.   Analysis by Geography

7.3.      Key Players: Analysis by Number of Product Candidates

8. MARKETED GENE THERAPIES
   8.1. Chapter Overview

8.2.      Gendicine® (Shenzhen Sibiono GeneTech)

8.2.1.   Company Overview

8.2.2.   Development Timeline

8.2.3.   Mechanism of Action and Vectors Used

8.2.4.   Target Indication(s)

8.2.5.   Current Status of Development

8.2.6.   Manufacturing, Dosage and Sales

8.3.      Oncorine® (Shanghai Sunway Biotech)

8.3.1.   Company Overview

8.3.2.   Development Timeline

8.3.3.   Mechanism of Action and Vectors Used

8.3.4.   Target Indication(s)

8.3.5.   Current Status of Development

8.3.6.   Manufacturing, Dosage and Sales

8.4.      Rexin-G® (Epeius Biotechnologies)

8.4.1.   Company Overview

8.4.2.   Development Timeline

8.4.3.   Mechanism of Action and Vector Used

8.4.4.   Target Indication(s)

8.4.5.   Current Status of Development

8.4.6.   Manufacturing, Dosage and Sales

8.5.      Neovasculgen® (Human Stem Cells Institute)

8.5.1.   Company Overview

8.5.2.   Development Timeline

8.5.3.   Mechanism of Action and Vectors Used

8.5.4.   Target Indication(s)

8.5.5.   Current Status of Development

8.5.6.   Manufacturing, Dosage and Sales

8.6.      Imlygic® (Amgen)

8.6.1.   Company Overview

8.6.2.   Development Timeline

8.6.3.   Mechanism of Action and Vectors Used

8.6.4.   Target Indication(s)

8.6.5.   Current Status of Development

8.6.6.   Manufacturing, Dosage and Sales

8.7.      Strimvelis® (Orchard Therapeutics)

8.7.1.   Company Overview

8.7.2.   Development Timeline

8.7.3.   Mechanism of Action and Vectors Used

8.7.4.   Target Indication(s)

8.7.5.   Current Status of Development

8.7.6.   Manufacturing, Dosage and Sales

8.8.      Luxturna™ (Spark Therapeutics)

8.8.1.   Company Overview

8.8.2.   Development Timeline

8.8.3.   Mechanism of Action and Vector Used

8.8.4.   Target Indication(s)

8.8.5.   Current Status of Development

8.8.6.   Manufacturing, Dosage and Sales

8.9.      Zolgensma™ (Novartis Gene Therapies)

8.9.1.   Company Overview

8.9.2.   Development Timeline

8.9.3.   Mechanism of Action and Vector Used

8.9.4.   Target Indication(s)

8.9.5.   Current Status of Development

8.9.6.   Manufacturing, Dosage and Sales

8.10.    Collategene® (AnGes)

8.10.1. Company Overview

8.10.2. Development Timeline

8.10.3. Mechanism of Action and Vector Used

8.10.4. Target Indication(s)

8.10.5. Current Status of Development

8.10.6. Manufacturing, Dosage and Sales

8.11.    Zyntelgo™ (bluebird bio)

8.11.1. Company Overview

8.11.2. Development Timeline

8.11.3. Mechanism of Action and Vector Used

8.11.4. Target Indication(s)

8.11.5. Current Status of Development

8.11.6. Manufacturing, Dosage and Sales

9. KEY COMMERCIALIZATION STRATEGIES
   9.1.      Chapter Overview

9.2.      Successful Drug Launch Strategy: ROOTS Framework

9.3.      Successful Drug Launch Strategy: Product Differentiation

9.4.      Commonly Adopted Commercialization Strategies based on Phase of Development of Product

9.5.      List of Currently Approved Gene Therapies

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

9.6.1.   Strategies Adopted Before Therapy Approval

9.6.2.   Strategies Adopted During / Post Therapy Approval

9.7.      Concluding Remarks

10. LATE STAGE (PHASE II/III AND ABOVE) GENE THERAPIES
    10.1.    Chapter Overview

10.2.    GS010

10.3.    PTC-AADC

10.4.    BMN 270

10.5.    Instiladrin®

10.6.    TAVO™

10.7.    OTL-200

10.8.    EB-101

10.9.    ProstAtak®

10.10.  BIIB111

10.11.  LentiGlobin BB305®

10.12.  Lenti-D™

10.13.  FCX-007

10.14.  CG0070

10.15.  Vigil®

10.16.  VM202

10.17.  VGX-3100

10.18.  Invossa™

10.19.  KB103

10.20.  PF-06939926

10.21.  PF-06838435

10.22.  PF-07055480

10.23.  SPK-8011

10.24.  AMT-061

10.25.  VB-111

10.26.  OTL-101

10.27.  BIIB112

10.28.  FLT180a

10.29.  LYS-SAF302

10.30.  NFS-01

10.31.  OTL-103

11. EMERGING TECHNOLOGIES
    11.1. Chapter Overview

11.2.    Gene Editing Technologies

11.2.1. Overview

11.2.2. Applications

11.3.    Emerging Gene Editing Platforms

11.3.1. CRISPR / Cas9 System

11.3.2. TALENs

11.3.3. megaTAL

11.3.4. Zinc Finger Nuclease

11.4.    Gene Expression Regulation Technologies

11.5.    Technology Platforms for Developing / Delivering Gene Therapies

12. KEY THERAPEUTICS AREAS
    12.1.    Chapter Overview

12.2.    Analysis by Therapeutic Area and Special Designation(s) Awarded

12.3.    Oncological Disorders

12.3.1. Analysis by Target Indication

12.3.2. Analysis by Type of Vector Used

12.4.    Nervous System Disorders

12.4.1. Analysis by Target Indication

12.4.2. Analysis by Type of Vector Used

12.5.    Metabolic Disorders

12.5.1. Analysis by Target Indication

12.5.2. Analysis by Type of Vector Used

12.6.    Ophthalmic Diseases

12.6.1. Analysis by Target Indication

12.6.2. Analysis by Type of Vector Used

12.7.    Genetic Disorders

12.7.1. Analysis by Target Indication

12.7.2. Analysis by Type of Vector Used

13. PATENT ANALYSIS
    13.1. Chapter Overview

13.2.    Gene Therapy Market: Patent Analysis

13.2.1. Scope and Methodology

13.2.1.1. Analysis by Publication Year

13.2.1.2. Analysis by Publication Year and Type of Patent

13.2.1.3. Analysis by Geography

13.2.1.4. Analysis by CPC Symbols

13.2.1.5. Analysis by Emerging Focus Areas

13.2.1.6. Leading Players: Analysis by Number of Patents

13.2.1.7. Patent Benchmark Analysis

13.2.1.8. Patent Valuation Analysis

13.3.    Gene Editing Market: Patent Analysis

13.3.1. Scope and Methodology

13.3.1.1. Analysis by Publication Year

13.3.1.2. Analysis by Publication Year and Type of Patent

13.3.1.3. Analysis by Geography

13.3.1.4. Analysis by CPC Symbols

13.3.1.5. Analysis by Emerging Focus Areas

13.3.1.6. Leading Players: Analysis by Number of Patents

13.3.1.7. Patent Benchmark Analysis

13.3.1.8. Patent Valuation Analysis

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

14. MERGERS AND ACQUISITIONS

14.1.    Chapter Overview

14.2.    Merger and Acquisition Models

14.3.    Gene Therapy Market: Mergers and Acquisitions

14.3.1. Analysis by Year of Merger / Acquisition

14.3.2. Analysis by Type of Agreement

14.3.3. Analysis by Geography

14.3.3.1. Continent-wise Distribution

14.3.3.2. Intercontinental and Intracontinental Deals

14.3.3.3. Country-wise Distribution

14.3.4. Analysis by Key Value Drivers

14.3.4.1. Analysis by Key Value Drivers and Year of Acquisition

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

14.3.6. Analysis by Therapeutic Area

15. FUNDING AND INVESTMENT ANALYSIS

15.1.    Chapter Overview

15.2.    Types of Funding

15.3.    Gene Therapy Market: Funding and Investment Analysis

15.3.1. Analysis by Number of Funding Instances

15.3.2. Analysis by Amount Invested

15.3.3. Analysis by Type of Funding

15.3.4. Analysis by Type of Funding and Year

15.3.5. Analysis by Type of Gene Therapy

15.3.6. Analysis of Amount Invested by Geography

15.3.7. Most Active Players: Analysis by Number of Funding Instances and Amount Invested

15.3.8. Key Investors: Analysis by Number of Funding Instances

15.3.9. Analysis by Phase of Development

15.3.10. Analysis by Therapeutic Area

15.4.    Concluding Remarks

16. CLINICAL TRIAL ANALYSIS

16.1.    Chapter Overview

16.2.    Scope and Methodology

16.3.    Gene Therapy Market: Clinical Trial Analysis

16.3.1. Analysis by Trial Registration Year

16.3.2. Analysis by Trial Status

16.3.3. Analysis by Trial Phase

16.3.4. Analysis by Therapeutic Area

16.3.5. Analysis by Geography

16.3.6. Geographical Analysis by Trial Registration Year

16.3.7. Geographical Analysis by Trial Status

16.3.8. Geographical Analysis by Trial Status and Trial Phase

16.3.9. Geographical Analysis by Therapeutic Area

16.4.    Analysis by Type of Sponsor

16.5.    Analysis by Prominent Treatment Sites

16.6.    Gene therapy Market: Analysis of Enrolled Patient Population

16.6.1. Analysis by Trial Registration Year

16.6.2. Analysis by Trial Status

16.6.3. Analysis by Trial Phase

16.6.4. Analysis by Therapeutic Area

16.6.5. Analysis by Location of Trial Site

16.6.6. Analysis by Trial Status and Location of Trial Site

16.6.7. Analysis by Trial Status, Trial Phase and Location of Trial Site

16.6.8. Analysis by Therapeutic Area and Location of Trial Site

16.7.    Concluding Remarks

16.7.1. Emerging Molecules to Watch

16.7.2. Most Important Trials to Watch

17. COST PRICE ANALYSIS

17.1.    Chapter Overview

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

17.3.    Gene Therapy Market: Pricing Models

17.3.1. Based on Associated Product / Component Costs

17.3.2. Based on Competition

17.3.3. Based on Patient Segment

17.3.4. Based on Opinions of Industry Experts

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

18.1.    Chapter Overview

18.2.    Gene Therapy Market: List of Most Prominent Big Pharmaceutical Players

18.2.1. Analysis by Therapeutic Area

18.2.2. Analysis by Type of Vector Used

18.2.3. Analysis by Therapeutic Approach

18.2.4. Analysis by Type of Gene Therapy

18.3.    Benchmark Analysis of Key Parameters

18.3.1. Spider Web Analysis: Pipeline Strength

18.3.2. Spider Web Analysis: Merger / Acquisitions

18.3.3. Spider Web Analysis: Funding and Investments

18.3.4. Spider Web Analysis: Clinical Trials

18.3.4. Spider Web Analysis: Technologies

18.3.4. Spider Web Analysis: Patents

18.4.    Benchmark Analysis of Big Pharmaceutical Players

19. DEMAND ANALYSIS

19.1.    Chapter Overview

19.2.    Methodology

19.3.    Global Demand for Gene Therapies, 2020-2030

19.3.1. Analysis by Therapeutic Approach

19.3.2. Analysis by Therapeutic Area

19.3.3. Analysis by Geography

20. MARKET FORECAST AND OPPORTUNITY ANALYSIS

20.1.    Chapter Overview

20.2.    Scope and Limitations

20.3.    Key Assumptions and Forecast Methodology

20.4.    Global Gene Therapy Market, 2020-2030

20.4.1. Gene Therapy Market: Analysis by Therapeutic Approach

20.4.2. Gene Therapy Market: Analysis by Type of Therapy

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

20.4.4. Gene Therapy Market: Analysis by Therapeutic Area

20.4.5. Gene Therapy Market: Analysis by Route of Administration

20.4.6. Gene Therapy Market: Analysis by Geography

20.5.    Gene Therapy Market: Value Creation Analysis

20.6.    Gene Therapy Market: Product-wise Sales Forecasts

20.6.1. Gendicine®

20.6.1.1. Target Patient Population

20.6.1.2. Sales Forecast

20.6.1.3. Net Present Value

20.6.1.4. Value Creation Analysis

20.6.2. Oncorine®

20.6.2.1. Target Patient Population

20.6.2.2. Sales Forecast

20.6.2.3. Net Present Value

20.6.2.4. Value Creation Analysis

20.6.3. Rexin-G®

20.6.3.1. Target Patient Population

20.6.3.2. Sales Forecast

20.6.3.3. Net Present Value

20.6.3.4. Value Creation Analysis

20.6.4. Neovasculgen®

20.6.4.1. Target Patient Population

20.6.4.2. Sales Forecast

20.6.4.3. Net Present Value

20.6.4.4. Value Creation Analysis

20.6.5. Strimvelis®

20.6.5.1. Target Patient Population

20.6.5.2. Sales Forecast

20.6.5.3. Net Present Value

20.6.5.4. Value Creation Analysis

20.6.6. Imlygic®

20.6.6.1. Target Patient Population

20.6.6.2. Sales Forecast

20.6.6.3. Net Present Value

20.6.6.4. Value Creation Analysis

20.6.7. Luxturna™

20.6.7.1. Target Patient Population

20.6.7.2. Sales Forecast

20.6.7.3. Net Present Value

20.6.7.4. Value Creation Analysis

20.6.8. Zolgensma™

20.6.8.1. Target Patient Population

20.6.8.2. Sales Forecast

20.6.8.3. Net Present Value

20.6.8.4. Value Creation Analysis

20.6.9. Collategene®

20.6.9.1. Target Patient Population

20.6.9.2. Sales Forecast

20.6.9.3. Net Present Value

20.6.9.4. Value Creation Analysis

20.6.10. Zyntelgo™

20.6.10.1. Target Patient Population

20.6.10.2. Sales Forecast

20.6.10.3. Net Present Value

20.6.10.4. Value Creation Analysis

20.6.11. GS010

20.6.11.1. Target Patient Population

20.6.11.2. Sales Forecast

20.6.11.3. Net Present Value

20.6.11.4. Value Creation Analysis

20.6.12. PTC-AADC

20.6.12.1. Target Patient Population

20.6.12.2. Sales Forecast

20.6.12.3. Net Present Value

20.6.12.4. Value Creation Analysis

20.6.13. BMN 270

20.6.13.1. Target Patient Population

20.6.13.2. Sales Forecast

20.6.13.3. Net Present Value

20.6.13.4. Value Creation Analysis

20.6.14. rAd-IFN/Syn3

20.6.14.1. Target Patient Population

20.6.14.2. Sales Forecast

20.6.14.3. Net Present Value

20.6.14.4. Value Creation Analysis

20.6.15. TAVO™

20.6.15.1. Target Patient Population

20.6.15.2. Sales Forecast

20.6.15.3. Net Present Value

20.6.15.4. Value Creation Analysis

20.6.16. OTL-200

20.6.16.1. Target Patient Population

20.6.16.2. Sales Forecast

20.6.16.3. Net Present Value

20.6.16.4. Value Creation Analysis

20.6.17. EB-101

20.6.17.1. Target Patient Population

20.6.17.2. Sales Forecast

20.6.17.3. Net Present Value

20.6.17.4. Value Creation Analysis

20.6.18. ProstAtak®

20.6.18.1. Target Patient Population

20.6.18.2. Sales Forecast

20.6.18.3. Net Present Value

20.6.18.4. Value Creation Analysis

20.6.19. BIIB111

20.6.19.1. Target Patient Population

20.6.19.2. Sales Forecast

20.6.19.3. Net Present Value

20.6.19.4. Value Creation Analysis

20.6.20. LentiGlobin BB305®

20.6.20.1. Target Patient Population

20.6.20.2. Sales Forecast

20.6.20.3. Net Present Value

20.6.20.4. Value Creation Analysis

20.6.21. Lenti-D™

20.6.21.1. Target Patient Population

20.6.21.2. Sales Forecast

20.6.21.3. Net Present Value

20.6.21.4. Value Creation Analysis

20.6.22. FCX-007

20.6.22.1. Target Patient Population

20.6.22.2. Sales Forecast

20.6.22.3. Net Present Value

20.6.22.4. Value Creation Analysis

20.6.23. CG0070

20.6.23.1. Target Patient Population

20.6.23.2. Sales Forecast

20.6.23.3. Net Present Value

20.6.23.4. Value Creation Analysis

20.6.24. Vigil®

20.6.24.1. Target Patient Population

20.6.24.2. Sales Forecast

20.6.24.3. Net Present Value

20.6.24.4. Value Creation Analysis

20.6.25. VM202

20.6.25.1. Target Patient Population

20.6.25.2. Sales Forecast

20.6.25.3. Net Present Value

20.6.25.4. Value Creation Analysis

20.6.26. VGX-3100

20.6.26.1. Target Patient Population

20.6.26.2. Sales Forecast

20.6.26.3. Net Present Value

20.6.26.4. Value Creation Analysis

20.6.27. Invossa™

20.6.27.1. Target Patient Population

20.6.27.2. Sales Forecast

20.6.27.3. Net Present Value

20.6.27.4. Value Creation Analysis

20.6.28. KB103

20.6.28.1. Target Patient Population

20.6.28.2. Sales Forecast

20.6.28.3. Net Present Value

20.6.28.4. Value Creation Analysis

20.6.29. PF-06939926

20.6.29.1. Target Patient Population

20.6.29.2. Sales Forecast

20.6.29.3. Net Present Value

20.6.29.4. Value Creation Analysis

20.6.30. PF-06838435

20.6.30.1. Target Patient Population

20.6.30.2. Sales Forecast

20.6.30.3. Net Present Value

20.6.30.4. Value Creation Analysis

20.6.31. PF-07055480

20.6.31.1. Target Patient Population

20.6.31.2. Sales Forecast

20.6.31.3. Net Present Value

20.6.31.4. Value Creation Analysis

20.6.32. SPK-8011

20.6.32.1. Target Patient Population

20.6.32.2. Sales Forecast

20.6.32.3. Net Present Value

20.6.32.4. Value Creation Analysis

20.6.33. AMT-061

20.6.33.1. Target Patient Population

20.6.33.2. Sales Forecast

20.6.33.3. Net Present Value

20.6.33.4. Value Creation Analysis

20.6.34. VB-111

20.6.34.1. Target Patient Population

20.6.34.2. Sales Forecast

20.6.34.3. Net Present Value

20.6.34.4. Value Creation Analysis

20.6.35. OTL-101

20.6.35.1. Target Patient Population

20.6.35.2. Sales Forecast

20.6.35.3. Net Present Value

20.6.35.4. Value Creation Analysis

20.6.36. BIIB112

20.6.36.1. Target Patient Population

20.6.36.2. Sales Forecast

20.6.36.3. Net Present Value

20.6.36.4. Value Creation Analysis

20.6.37. FLT180a

20.6.37.1. Target Patient Population

20.6.37.2. Sales Forecast

20.6.37.3. Net Present Value

20.6.37.4. Value Creation Analysis

20.6.38. LYS-SAF302

20.6.38.1. Target Patient Population

20.6.38.2. Sales Forecast

20.6.38.3. Net Present Value

20.6.38.4. Value Creation Analysis

20.6.39. NFS-01

20.6.39.1. Target Patient Population

20.6.39.2. Sales Forecast

20.6.39.3. Net Present Value

20.6.39.4. Value Creation Analysis

20.6.40. OTL-103

20.6.40.1. Target Patient Population

20.6.40.2. Sales Forecast

20.6.40.3. Net Present Value

20.6.40.4. Value Creation Analysis

21. VECTOR MANUFACTURING

21.1.    Chapter Overview

21.2.    Overview of Viral Vector Manufacturing

21.3.    Viral Vector Manufacturing Processes

21.3.1. Mode of Vector Production

21.3.2. Adherent and Suspension Cultures

21.3.3. Unit and Parallel Processes

21.3.4. Cell Culture Systems for Production of Viral Vectors

21.3.5. Culture Media Specifications

21.4.    Bioprocessing of Viral Vectors

21.4.1. Adeno-associated Viral Vector Production

21.4.2. Adenoviral Vector Production

21.4.3. Lentiviral Vector Production

21.4.4. γ -Retroviral Vector Production

21.5.    Challenges Associated with Vector Manufacturing

21.6.    Companies Offering Contract Services for Viral and Plasmid Vectors

22. CASE STUDY: GENE THERAPY SUPPLY CHAIN

22.1.    Chapter Overview

22.2.    Overview of the Gene Therapy Supply Chain

22.3.    Implementation of Supply Chain Models

22.4.    Logistics Associated with Gene Therapies

22.4.1. Autologous and Allogeneic Therapies

22.5.    Regulatory Supply Chain Across the Globe

22.6.    Challenges Associated with Gene Therapy Supply Chain

22.7.    Optimizing Cell and Advanced Therapies Supply Chain Management

22.7.1. Enterprise Manufacturing Systems

22.7.2. Laboratory Information Management Systems

22.7.3. Inventory Management Systems

22.7.4. Quality Management Systems

22.7.5. Logistics Management Systems

22.7.6. Patient Management Systems

22.7.7. Electronic Clinical Outcome Assessments Systems

22.7.8. Supply Chain Orchestration Platforms

22.8.    Recent Developments and Upcoming Trends

23. CONCLUSION

24. INTERVIEW TRANSCRIPTS

24.1.    Chapter Overview

24.2.    Adam Rogers, CEO, Hemera Biosciences

24.3.    Al Hawkins, CEO, Milo Biotechnology

24.4.    Buel Dan Rodgers, Founder and CEO, AAVogen

24.5.    Christopher Reinhard, CEO and Chairman, Gene Therapeutics (previously known as Cardium Therapeutics)

24.6.    Michael Tripletti, ex-CEO, Myonexus Therapeutics

24.7.    Robert Jan Lamers, ex-CEO, Arthrogen

24.8.    Ryo Kubota, CEO, Chairman and President, Acucela

24.9.    Tom Wilton, ex-CBO, LogicBio Therapeutics

24.10. Jeffrey Hung, CCO, Vigene Biosciences

24.11.  Cedric Szpirer, Executive & Scientific Director, Delphi Genetics

24.12.  Marco Schmeer, Project Manager and Tatjana Buchholz, ex-Marketing Manager, PlasmidFactory

24.13.  Molly Cameron, Corporate Communications Manager, Orchard Therapeutics

25. APPENDIX 1: TABULATED DATA

26. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS

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