StemCell Therapy

DUBLIN, Aug. 8, 2022 /PRNewswire/ --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.

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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.

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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 ool 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. INTRODUCTION3.1. Context and Background3.2. Evolution of Gene Therapies3.3. Classification of Gene Therapies3.3.1. Somatic and Germline Gene Therapies3.3.2. Ex Vivo and In Vivo Gene Therapies3.4. Routes of Administration3.5. Mechanism of Action3.6. Overview of Gene Editing3.6.1. Evolution of Genome Editing3.6.2. Applications of Genome Editing3.6.3. Available Genome Editing Techniques3.7. Advantages and Disadvantages of Gene Therapies3.7.1 Ethical and Social Concerns Related to Gene Therapies3.7.2. Constraints and Challenges Related to Gene Therapies3.7.3. Therapy Development Concerns3.7.4. Manufacturing Concerns3.7.5. Commercial Viability Concerns

4. GENE DELIVERY VECTORS4.1. Chapter Overview4.2. Viral and Non-Viral Methods of Gene Transfer4.3. Viral Vectors for Genetically Modified Therapies4.4. Types of Viral Vectors4.5. Types of Non-Viral Vectors

5. REGULATORY LANDSCAPE AND REIMBURSEMENT SCENARIO5.1. Chapter Overview5.2. Regulatory Guidelines in North America5.3. Regulatory Guidelines in Europe5.4. Regulatory Guidelines in Asia-Pacific5.5. Reimbursement Scenario5.6. Commonly Offered Payment Models for Gene Therapies

6. MARKET OVERVIEW6.1. Chapter Overview6.2. Gene Therapy Market: Clinical and Commercial Pipeline6.3. Gene Therapy Market: Early-Stage Pipeline6.4. Gene Therapy Market: Special Drug Designations6.5. Analysis by Phase of Development, Therapeutic Area and Type of Therapy (Grid Representation)

7. COMPETITIVE LANDSCAPE7.1. Chapter Overview7.2. Gene Therapy Market: List of Developers7.3. Key Players: Analysis by Number of Pipeline Candidates

8. MARKETED GENE THERAPIES8.1. Chapter Overview8.2. 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 STRATEGIES9.1. Chapter Overview9.2. Successful Drug Launch Strategy: ROOTS Framework9.3. Successful Drug Launch Strategy: Product Differentiation9.4. Commonly Adopted Commercialization Strategies based on Phase of Development of Product9.5. List of Currently Approved Gene Therapies9.6. Key Commercialization Strategies Adopted by Gene Therapy Developers9.6.1. Strategies Adopted Before Therapy Approval9.6.1.1. Participation in Global Events9.6.1.2. Collaboration with Stakeholders and Pharmaceutical Firms9.6.1.3. Indication Expansion9.6.2. Strategies Adopted During/Post Therapy Approval9.6.2.1. Geographical Expansion9.6.2.2. Participation in Global Events9.6.2.3. Patience Assistance Programs9.6.2.4. Awareness through Product Websites9.6.2.5. Collaboration with Stakeholders and Pharmaceutical Firms9.7. Concluding Remarks

10. LATE STAGE (PHASE II/III AND ABOVE) GENE THERAPIES10.1. Chapter Overview10.2. Lumevoq (GS010): Information on Dosage, Mechanism of Action, Clinical Trials and Clinical Trial Results10.3. OTL-10310.4. PTC-AADC10.5. BMN 27010.6. rAd-IFN/Syn310.7. beti-cel10.8. eli-cel10.9. lovo-cel10.10. SRP-900110.11. EB-10110.12. ProstAtak10.13. D-Fi10.14. CG007010.15. VigilT-EWS10.16. Engensis10.17. VGX-310010.18. InvossaT (TG-C)10.19. VYJUVEKTT10.20. PF-0693992610.21. PF-0683843510.22. PF-0705548010.23. SPK-801110.24. AMT-06110.25. VB-11110.26. Generx10.27. ADXS-HPV10.28. AGTC 50110.29. LYS-SAF30210.30. NFS-0110.31. AG0302-COVID1910.32. RGX-31410.33. Hologene 5

11. EMERGING TECHNOLOGIES11.1. Chapter Overview11.2. Gene Editing Technologies11.2.1. Overview11.2.2. Applications11.3. Emerging Gene Editing Platforms11.3.1. CRISPR/Cas9 System11.3.2. TALENs11.3.3. megaTAL11.3.4. Zinc Finger Nuclease11.4. Gene Expression Regulation Technologies11.5. Technology Platforms for Developing/Delivering Gene Therapies

12. KEY THERAPEUTICS AREAS12.1. Chapter Overview12.2. Analysis by Therapeutic Area and Special Designation(s) Awarded12.3. Oncological Diseases12.4. Neurological Diseases12.5. Ophthalmic Diseases12.6. Metabolic Diseases12.7. Genetic Diseases

13. PATENT ANALYSIS13.1. Chapter Overview13.2. Gene Therapy Market: Patent Analysis13.3. Gene Editing Market: Patent Analysis13.4. Overall Intellectual Property Portfolio: Analysis by Type of Organization

14. MERGERS AND ACQUISITIONS14.1. Chapter Overview14.2. Merger and Acquisition Models14.3. Gene Therapy Market: Mergers and Acquisitions

15. FUNDING AND INVESTMENT ANALYSIS15.1. Chapter Overview15.2. Types of Funding15.3. Gene Therapy Market: Funding and Investment Analysis15.4. Concluding Remarks

16. CLINICAL TRIAL ANALYSIS16.1. Chapter Overview16.2. Scope and Methodology16.3. Gene Therapy Market: Clinical Trial Analysis16.4. Analysis by Type of Sponsor16.5. Analysis by Prominent Treatment Sites16.6. Gene Therapy Market: Analysis of Enrolled Patient Population16.7. Concluding Remarks

17. COST PRICE ANALYSIS17.1. Chapter Overview17.2. Gene Therapy Market: Factors Contributing to the Price of Gene Therapies17.3. Gene Therapy Market: Pricing Models

18. START-UP VALUATION18.1. Chapter Overview18.2. Valuation by Year of Experience

19. BIG PHARMA PLAYERS: ANALYSIS OF GENE THERAPY RELATED INITIATIVES19.1. Chapter Overview19.2. Gene Therapy Market: List of Most Prominent Big Pharmaceutical Players19.3. Benchmark Analysis of Key Parameters19.4. Benchmark Analysis of Big Pharmaceutical Players

20. DEMAND ANALYSIS20.1. Chapter Overview20.2. Methodology20.3. Global Demand for Gene Therapies, 2022-2035

21. MARKET FORECAST AND OPPORTUNITY ANALYSIS21.1. Chapter Overview21.2. Scope and Limitations21.3. Key Assumptions and Forecast Methodology21.4. Global Gene Therapy Market, 2022-203521.5. Gene Therapy Market: Value Creation Analysis21.6. Gene Therapy Market: Product-wise Sales Forecasts21.6.1. Gendicine21.6.2. Oncorine21.6.3. Rexin-G21.6.4. Neovasculgen21.6.5. Strimvelis21.6.6. Imlygic21.6.7. LuxturnaT21.6.8. ZolgensmaT21.6.9. Collategene21.6.10. LibmeldyT21.6.11. Lumevoq (GS010)21.6.12. OTL-10321.6.13. PTC-AADC21.6.14. BMN 27021.6.15. rAd-IFN/Syn321.6.16. beti-cel21.6.17. eli-cel21.6.18. lovo-cel21.6.19. SRP-900121.6.20. EB-10121.6.21. ProstAtak21.6.22. D-Fi21.6.23. CG007021.6.24. VigilT-EWS21.6.25. Engensis21.6.26. VGX-310021.6.27. InvossaT (TG-C)21.6.28. VYJUVEKTT21.6.29. PF-0693992621.6.30. PF-0683843521.6.31. PF-0705548021.6.32. SPK-801121.6.33. AMT-06121.6.34. VB-11121.6.35. Generx21.6.36. AMG00121.6.37. OAV-10121.6.38. ADXS-HPV21.6.39. AGTC 50121.6.40. LYS-SAF30221.6.41. NFS-0121.6.42. AG0302-COVID1921.6.43. RGX-31421.6.44. Hologene 5

22. VECTOR MANUFACTURING22.1. Chapter Overview22.2. Overview of Viral Vector Manufacturing22.3. Viral Vector Manufacturing Process22.4. Bioprocessing of Viral Vectors22.5. Challenges Associated with Vector Manufacturing22.6. Contract Service Providers for Viral and Plasmid Vectors

23. CASE STUDY: GENE THERAPY SUPPLY CHAIN23.1. Chapter Overview23.2. Overview of Gene Therapy Supply Chain23.3. Implementation of Supply Chain Models23.4. Logistics in Gene Therapies23.5. Regulatory Supply Chain Across the Globe23.6. Challenges Associated with Gene Therapy Supply Chain23.7. Optimizing Cell and Advanced Therapies Supply Chain Management23.8. Recent Developments and Upcoming Trends

24. CONCLUSION24.1. Chapter Overview

25. INTERVIEW TRANSCRIPTS25.1. Chapter Overview25.2. 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/kp21lo

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