Gene Therapy Market Research Report 2022 - ResearchAndMarkets.com

DUBLIN--(BUSINESS WIRE)--The "Gene Therapy Market by Type of Therapy, Type of Gene Delivery Method Used, Type of Vector Used, Target Therapeutic Areas, Route of Administration, and Key Geographical Regions: Industry Trends and Global Forecasts, 2022-2035" report has been added to ResearchAndMarkets.com's offering.

Gene Therapy Market (5th Edition) report features an extensive study of the current market landscape and the likely future potential associated with the gene therapy market, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene editing therapies.

One of the key objectives of the report was to estimate the existing market size and the future opportunity associated with gene therapies, over the next decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the evolution of the market for the period 2022-2035.

Over the last two decades, there have been several breakthroughs related to the development of gene therapies. In 2020, LibmeldyT, an ex vivo gene therapy received approval for the treatment of metachromatic leukodystrophy. To provide more context, the treatment regimen of such therapies, encompassing gene replacement and gene-editing modalities, is aimed at correction of the mutated gene in patients using molecular carriers (viral and non-viral vectors).

Further, post the onset of the COVID-19 pandemic, there has been a steady increase in the investigational new drug (IND) applications filed for cell and gene therapies. In fact, in 2021, more than 200 gene therapies were being evaluated in phase II and III studies. Moreover, in 2022, six gene therapies are expected to receive the USFDA market approval. Promising results from ongoing clinical research initiatives have encouraged government and private firms to make investments to support therapy product development initiatives in this domain.

In 2021 alone, gene therapy developers raised around USD 9.5 billion in capital investments. Taking into consideration the continuous progress in this domain, gene therapies are anticipated to be used for the treatment of 1.1 million patients suffering from a myriad of disease indications, by 2035.

Presently, more than 250 companies are engaged in the development of various early and late-stage gene therapies, worldwide. In recent years, there has been a significant increase in the integration of novel technologies, such as gene modification, gene-editing, genome sequencing and manipulation technologies (molecular switches), in conjugation with gene delivery methods.

For instance, the CRISPR-Cas9 based gene-editing tool is one of the remarkable technological advancements, which enables the precise alteration of the transgene. It is worth mentioning that the new generation delivery platforms, including nanoparticles and hybrid vector systems, have been demonstrated to be capable of enabling effective and safe delivery of gene based therapeutics.

Further, a variety of consolidation efforts are currently ongoing in this industry. Such initiatives are primarily focused on expanding and strengthening the existing development efforts; this can be validated from the fact that 56% of the total acquisitions reported in the domain were focused on drug class consolidation.

Driven by the collective and consistent efforts of developers and the growing demand for a single dose of effective therapeutic, the gene therapy market is anticipated to witness significant growth in the foreseen future.

Key Questions Answered

• Who are the key industry players engaged in the development of gene therapies?
• How many gene therapy candidates are present in the current development pipeline? Which key disease indications are targeted by such products?
• Which types of vectors are most commonly used for effective delivery of gene therapies?
• What are the key regulatory requirements for gene therapy approval, across various geographies?
• Which commercialization strategies are most commonly adopted by gene therapy developers, across different stages of development?
• What are the different pricing models and reimbursement strategies currently being adopted for gene therapies?
• What are the various technology platforms that are either available in the market or are being designed for the development of gene therapies?
• Who are the key CMOs/CDMOs engaged in supplying viral/plasmid vectors for gene therapy development?
• What are the key value drivers of the merger and acquisition activity in the gene therapy industry?
• Who are the key stakeholders that have actively made investments in the gene therapy domain?
• Which are the most active trial sites (in terms of number of clinical studies being conducted) in this domain?
• How is the current and future market opportunity likely to be distributed across key market segments?

Key Topics Covered:

1. PREFACE

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 Therapies

3.3.2. Ex Vivo and In Vivo Gene Therapies

3.4. Routes of Administration

3.5. Mechanism of Action

3.6. Overview of Gene Editing

3.6.1. Evolution of Genome Editing

3.6.2. Applications of Genome Editing

3.6.3. Available Genome Editing Techniques

3.7. Advantages and Disadvantages of Gene Therapies

3.7.1 Ethical and Social Concerns Related to Gene Therapies

3.7.2. Constraints and Challenges Related to Gene Therapies

3.7.3. Therapy Development Concerns

3.7.4. Manufacturing Concerns

3.7.5. 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

4.5. Types of Non-Viral Vectors

5. REGULATORY LANDSCAPE AND REIMBURSEMENT SCENARIO

5.1. Chapter Overview

5.2. Regulatory Guidelines in North America

5.3. Regulatory Guidelines in Europe

5.4. Regulatory Guidelines in Asia-Pacific

5.5. Reimbursement Scenario

5.6. Commonly Offered Payment Models for Gene Therapies

6. MARKET OVERVIEW

6.1. Chapter Overview

6.2. Gene Therapy Market: Clinical and Commercial Pipeline

6.3. Gene Therapy Market: Early-Stage Pipeline

6.4. Gene Therapy Market: Special Drug Designations

6.5. Analysis by Phase of Development, Therapeutic Area and Type of Therapy (Grid Representation)

7. COMPETITIVE LANDSCAPE

7.1. Chapter Overview

7.2. Gene Therapy Market: List of Developers

7.3. Key Players: Analysis by Number of Pipeline Candidates

8. MARKETED GENE THERAPIES

8.1. Chapter Overview

8.2. Gendicine (Shenzhen Sibiono GeneTech)

8.3. Oncorine (Shanghai Sunway Biotech)

8.4. Rexin-G (Epeius Biotechnologies)

8.5. Neovasculgen (Human Stem Cells Institute)

8.6. Imlygic (Amgen)

8.7. Strimvelis (Orchard Therapeutics)

8.8. LuxturnaT (Spark Therapeutics)

8.9. ZolgensmaT (Novartis)

8.10. Collategene (AnGes)

8.11. ZyntelgoT (bluebird bio)

8.12. LibmeldyT (Orchard Therapeutics)

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 Gene Therapy Developers

9.6.1. Strategies Adopted Before Therapy Approval

9.6.1.1. Participation in Global Events

9.6.1.2. Collaboration with Stakeholders and Pharmaceutical Firms

9.6.1.3. Indication Expansion

9.6.2. Strategies Adopted During/Post Therapy Approval

9.6.2.1. Geographical Expansion

9.6.2.2. Participation in Global Events

9.6.2.3. Patience Assistance Programs

9.6.2.4. Awareness through Product Websites

9.6.2.5. Collaboration with Stakeholders and Pharmaceutical Firms

9.7. Concluding Remarks

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

10.1. Chapter Overview

10.2. Lumevoq (GS010): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results

10.3. OTL-103

10.4. PTC-AADC

10.5. BMN 270

10.6. rAd-IFN/Syn3

10.7. beti-cel

10.8. eli-cel

10.9. lovo-cel

10.10. SRP-9001

10.11. EB-101

10.12. ProstAtak

10.13. D-Fi

10.14. CG0070

10.15. VigilT-EWS

10.16. Engensis

10.17. VGX-3100

10.18. InvossaT (TG-C)

10.19. VYJUVEKTT

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. Generx

10.27. ADXS-HPV

10.28. AGTC 501

10.29. LYS-SAF302

10.30. NFS-01

10.31. AG0302-COVID19

10.32. RGX-314

10.33. Hologene 5

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 Diseases

12.4. Neurological Diseases

12.5. Ophthalmic Diseases

12.6. Metabolic Diseases

12.7. Genetic Diseases

13. PATENT ANALYSIS

13.1. Chapter Overview

13.2. Gene Therapy Market: Patent Analysis

13.3. Gene Editing Market: Patent 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

15. FUNDING AND INVESTMENT ANALYSIS

15.1. Chapter Overview

15.2. Types of Funding

15.3. Gene Therapy Market: Funding and Investment Analysis

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

16.4. Analysis by Type of Sponsor

16.5. Analysis by Prominent Treatment Sites

16.6. Gene Therapy Market: Analysis of Enrolled Patient Population

16.7. Concluding Remarks

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

18. START-UP VALUATION

18.1. Chapter Overview

18.2. Valuation by Year of Experience

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

19.1. Chapter Overview

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

19.3. Benchmark Analysis of Key Parameters

19.4. Benchmark Analysis of Big Pharmaceutical Players

20. DEMAND ANALYSIS

20.1. Chapter Overview

20.2. Methodology

20.3. Global Demand for Gene Therapies, 2022-2035

21. MARKET FORECAST AND OPPORTUNITY ANALYSIS

21.1. Chapter Overview

21.2. Scope and Limitations

21.3. Key Assumptions and Forecast Methodology

21.4. Global Gene Therapy Market, 2022-2035

21.5. Gene Therapy Market: Value Creation Analysis

21.6. Gene Therapy Market: Product-wise Sales Forecasts

21.6.1. Gendicine

21.6.2. Oncorine

21.6.3. Rexin-G

21.6.4. Neovasculgen

21.6.5. Strimvelis

21.6.6. Imlygic

21.6.7. LuxturnaT

21.6.8. ZolgensmaT

21.6.9. Collategene

21.6.10. LibmeldyT

21.6.11. Lumevoq (GS010)

21.6.12. OTL-103

21.6.13. PTC-AADC

21.6.14. BMN 270

21.6.15. rAd-IFN/Syn3

21.6.16. beti-cel

21.6.17. eli-cel

21.6.18. lovo-cel

21.6.19. SRP-9001

21.6.20. EB-101

21.6.21. ProstAtak

21.6.22. D-Fi

21.6.23. CG0070

21.6.24. VigilT-EWS

21.6.25. Engensis

21.6.26. VGX-3100

21.6.27. InvossaT (TG-C)

21.6.28. VYJUVEKTT

21.6.29. PF-06939926

21.6.30. PF-06838435

21.6.31. PF-07055480

21.6.32. SPK-8011

21.6.33. AMT-061

21.6.34. VB-111

21.6.35. Generx

21.6.36. AMG001

21.6.37. OAV-101

21.6.38. ADXS-HPV

21.6.39. AGTC 501

21.6.40. LYS-SAF302

21.6.41. NFS-01

21.6.42. AG0302-COVID19

21.6.43. RGX-314

21.6.44. Hologene 5

22. VECTOR MANUFACTURING

22.1. Chapter Overview

22.2. Overview of Viral Vector Manufacturing

22.3. Viral Vector Manufacturing Process

22.4. Bioprocessing of Viral Vectors

22.5. Challenges Associated with Vector Manufacturing

22.6. Contract Service Providers for Viral and Plasmid Vectors

23. CASE STUDY: GENE THERAPY SUPPLY CHAIN

23.1. Chapter Overview

23.2. Overview of Gene Therapy Supply Chain

23.3. Implementation of Supply Chain Models

23.4. Logistics in Gene Therapies

23.5. Regulatory Supply Chain Across the Globe

23.6. Challenges Associated with Gene Therapy Supply Chain

23.7. Optimizing Cell and Advanced Therapies Supply Chain Management

23.8. Recent Developments and Upcoming Trends

24. CONCLUSION

24.1. Chapter Overview

25. INTERVIEW TRANSCRIPTS

25.1. Chapter Overview

25.2. Buel Dan Rodgers (Founder and CEO, AAVogen)

25.3. Sue Washer (President and CEO, AGTC)

25.4. Patricia Zilliox (President and CEO, Eyevensys)

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

25.6. Adam Rogers (CEO, Hemera Biosciences)

25.7. Ryo Kubota (CEO, Chairman and President, Kubota Pharmaceutical Holdings (Acucela))

25.8. Al Hawkins (CEO, Milo Biotechnology)

25.9. Jean-Phillipe Combal (CEO, Vivet Therapeutics)

25.10. Robert Jan Lamers (former CEO, Arthrogen)

25.11. Tom Wilton (former CBO, LogicBio Therapeutics)

25.12. Michael Tripletti (former CEO, Myonexus Therapeutics)

25.13. Molly Cameron (former Corporate Communications Manager, Orchard Therapeutics)

25.14. Cedric Szpirer (former Executive and Scientific Director, Delphi Genetics)

25.15. Marco Schmeer (Project Manager) and Tatjana Buchholz, (former Marketing Manager, PlasmidFactory)

25.16. Jeffrey Hung (CCO, Vigene Biosciences)

26. APPENDIX 1: TABULATED DATA

27. APPENDIX 2: LIST OF COMPANIES AND ORGANIZATIONS

For more information about this report visit https://www.researchandmarkets.com/r/j3pnph

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