Gene Therapy R&D and Revenue Forecasts 2018-2028

1. Report Overview

1.1 Global Gene Therapy Market Overview

1.2 Benefits of This Report

1.3 How This Report Delivers

1.4 Key Questions Answered by This Analytical Report

1.5 Who is This Report For?

1.6 Methods of the Study

1.7 Frequently Asked Questions (FAQ)

1.8 Associated Visiongain Reports

1.9 About Visiongain

2. Introduction to Gene Therapy

2.1 What is Gene Therapy?

2.2 Types of Gene Therapy

2.3 Products Excluded from the Report

2.4 Gene Therapy Vectors

3. Gene Therapy World Market: Leading Treatments and Forecasts, 2017-2028

3.1 The World Market for Gene Therapy: Overview of Products

3.2 Gendicine (rAd-p53) – The First Gene Therapy to Market

3.3 Oncorine: Growth is Hindered by Certain Factors

3.4 Neovasculgen: Russia’s First Gene Therapy

3.4.1 Neovasculgen: Historical Performance and Revenue Forecast, 2017-2028

3.5 Glybera: A Drug with Promise

3.5.1 Glybera: Pricing Ends the Dream to Become a Blockbuster Product

3.6 Strimvelis: Second Gene Therapy for an Inherited Disease Ever to be approved for Sale and the First Corrective Gene Therapy for Children to be Approved Anywhere in the World

3.6.1 Even Big Pharma Struggle in the Rare Disease Space

4. Gene Therapy World Market and Disease Submarkets, 2017-2028

4.1 The Global Market for Gene Therapy Drug Treatments in 2017

4.2 Gene Therapy Market Forecast, 2017-2028

4.3 Gene Therapy for Cancer

4.3.1 Clinical Trials for Cancer

4.3.2 The Cancer Gene Therapy Treatment Submarket Forecast 2017-2028

4.4 Gene Therapy for Rare Diseases

4.4.1 The Rare Disease Treatment Submarket Forecast 2017-2028

4.5 Gene Therapy for Cardiovascular Diseases

4.5.1 The Cardiovascular Disease Treatment Submarket Forecast 2017-2028

4.6 Gene Therapy for Ophthalmologic Diseases

4.6.1 Advanced Stage Gene Therapies for Ophthalmologic Diseases

4.6.2 The Ophthalmologic Disease Treatment Submarket Forecast 2017-2028

4.7 Gene Therapy for Other Diseases

4.7.1 The Other Disease Treatment Submarket Forecast 2017-2028

4.7.2 Changing Market Shares: Cancer Disease Gene Therapies Will Dominate and Become Market Leader

5. R&D Pipeline Analysis and Forecasts, 2017-2028

5.1 Pipeline

5.2 Pipeline Analysis

5.3 Collategene (AMG0001) - AnGes MG/Vical

5.4 BC-819 - BioCancell

5.5 BC-821 - BioCancell

5.6 Lenti-D - Bluebird Bio

5.7 LentiGlobin (LentiGlobin BB305) - Bluebird Bio

5.8 SPK-RPE65 - Spark Therapeutics

5.9 SPK-CHM - Spark Therapeutics

5.10 SPK-FIX - Spark Therapeutics/Pfizer

5.11 SPK-TPP1- Spark Therapeutics

5.12 Invossa (TissueGene-C) - TissueGene Inc/Kolon Life Science

5.13 VM202-DPN - ViroMed

5.14 Important Pipeline Technology Developments

5.15 Clustered regularly interspaced short palindromic repeats (CRISPR)

5.16 The Future of the Gene Therapy Pipeline

6. Companies of Interest in the Gene Therapy Market: Robust Pipelines Will Yield Revenue in the Future

6.1 UniQure- the First Commercial Stage Gene Therapy Company in the West

6.2 Biogen - Focusing on Ophthalmologic and Rare Disease Gene Therapies

6.3 Bluebird Bio: Gene Editing Technology

6.4 Spark Therapeutics- Strong Change to Become the First Company with Marketed Gene Therapy in the US

6.5 Applied Genetic Technologies Corporation: Specialisation in Ophthalmologic Diseases

6.6 Oxford Biomedica: Collaboration as a Strategy

6.7 GenSight Biologics - Novel Mitochondrial Technology Platform

6.8 Gene Therapy and Big Pharma: Collaborations

7. Qualitative Analysis of the Gene Therapy Market, 2018-2028

7.1 SWOT Analysis of the Gene Therapy Treatment Market, 2018

7.2 Strengths: A Vast Pipeline for a Wide Range of Indications

7.3 Weaknesses: Low Patient Populations Cannot Sustain Long-Term Growth

7.4 Opportunities and Threats for the Gene Therapy Drug Market, 2018-2028

7.5 Opportunities: Collaborations and Novel Technologies Will Pave the Way

7.6 Threats: Expensive Drugs with Strict Regulations

7.7 National Markets: Regulation of the Gene Therapy Market

7.7.1 Gene Therapy Regulations: Evolving with the Market

7.7.2 US Regulations: Strict Regulation of a Cautious Market

7.7.3 EU Regulations: One Approval So Far

7.7.4 Japanese Regulations: A Rigorous Process

7.7.5 China Has Highest Gene Sequencing Capacity

8. Conclusions

8.1 The World Gene Therapy Treatment Market: Current World Outlook

8.1.1 Leading Gene Therapy Treatment Submarkets and Products, 2017-2028

8.1.2 Driver for Growth to 2028: A Vast Pipeline of Gene Therapy Products

8.1.3 Leading Regional Markets and Companies: Partnerships Will Achieve Success

8.2 The Future of the Gene Therapy Treatment Market

Associated Visiongain Reports

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Table of Tables

Table 1.1 Foreign Currency Exchange Rates Utilised: Exchange Rate to $US, 2015, 2016, and 2017

Table 3.1 Approved Gene Therapies, 2017

Table 3.2 Gene Therapies in Pipeline, 2017

Table 3.3 Neovasculgen Historical Revenue Comparisons, Revenue ($m), AGR (%), 2013-2017

Table 3.4 Neovasculgen Forecast, Revenue ($m), AGR (%), CAGR (%), 2017-2028

Table 4.1 Leading Gene Therapy Drugs by Revenue ($m), 2017

Table 4.2 World Market Forecasts for Gene Therapy Drugs by Indication, Market Sizes ($m), Annual Growth Rates (%), CAGR (%), 2017-2028

Table 4.3 Selected Anti-Angiogenic Gene Therapy Using a Non-Viral Vector, 2017

Table 4.4 Selected Anti-Angiogenic Gene Therapy Using a Retroviral Vector, 2017

Table 4.5 Selected Anti-Angiogenic Gene Therapy Using an Adenoviral Vector, 2017

Table 4.6 Selected Anti-Angiogenic Gene Therapy Using an Adeno-Associated Virus Vector, 2017

Table 4.7 Selected Anti-Angiogenic Gene Therapy Using Lentiviral Vector, 2017

Table 4.8 Global Gene Therapy Clinical Trials by Indication, 2017

Table 4.9 Selected Cancer Gene Therapies in Phase 2 and Phase 3 Clinical Trial Development, 2017

Table 4.10 Selected Cancer Gene Therapies in Phase 1 and Phase 1/2 Clinical Trial Development, 2017

Table 4.11 Global Gene Therapy Market: Revenue ($m) and Market Shares (%) by Indication, 2017

Table 4.12 Global Market Forecast for Cancer Gene Therapies: Revenue ($m), Annual Growth Rate (%), CAGR (%), 2017-2028

Table 4.13 Rare Diseases Gene Therapy Pipeline, 2017

Table 4.14 Global Market Forecast for Rare Disease Gene Therapies: Revenue ($m), Annual Growth Rate (%), CAGR (%), 2017-2028

Table 4.15 Cardiovascular Gene Therapy Pipeline, 2017

Table 4.16 Global Market Forecast for Cardiovascular Disease Gene Therapies: Revenue ($m), Annual Growth Rate (%), CAGR (%), 2017-2028

Table 4.17 Preclinical Ophthalmologic Pipeline, 2017

Table 4.18 Clinical Ophthalmologic Pipeline, 2017

Table 4.19 Additional Products in the Clinical Ophthalmologic Pipeline, 2017

Table 4.20 Global Market Forecast for Ophthalmologic Disease Gene Therapies: Revenue ($m), Annual Growth Rate (%), CAGR (%), 2017-2028

Table 4.21 Global Market Forecast for Other Disease Gene Therapies: Revenue ($m), Annual Growth Rate (%), CAGR (%), 2017-2028

Table 4.22 Global Market Forecast for Gene Therapies by Indication: Market Shares (%), 2017-2022

Table 4.23 Global Market Forecast for Gene Therapies by Indication: Market Shares (%), 2023-2028

Table 5.1 Promising Gene Therapies in Development, 2018

Table 5.2 Gene Therapies in Phase 3, 2017

Table 5.3 Collatagene Forecast, Revenue ($m), AGR (%), CAGR (%), 2017-2028

Table 5.4 BC-819 Forecast, Revenue ($m), AGR (%), CAGR (%), 2017-2028

Table5.5 Lenti-D Forecast, Revenue ($m), AGR (%), CAGR (%), 2017-2028

Table 5.6 SPK-RPE65 Forecast, Revenue ($m), AGR (%), CAGR (%), 2017-2028

Table 5.7 Invossa Forecast, Revenue ($m), AGR (%), CAGR (%), 2017-2028

Table 6.1 uniQure Pipeline, 2017

Table 6.2 Spark Therapeutics Collaborations, 2017

Table 6.3 Selected Big Pharma Collaborations in Gene Therapy, 2017

Table 7.1 Strengths and Weaknesses of the Gene Therapy Drug Treatment Market, 2017

Table 7.2 Gene Therapy Pipeline for Neurodegenerative Diseases, 2017

Table 7.3 Gene Therapy Pipeline for Metabolic Diseases, 2017

Table 7.4 Gene Therapy Pipeline for Musculoskeletal Diseases, 2017

Table 7.5 Gene Therapy Pipeline for Viral Diseases, 2017

Table 7.6 Opportunities and Threats of the Gene Therapy Drug Market, 2017

Table 7.7 Clinical Trials for Standard Drug Development Versus Orphan Drug Development, 2017

Table 8.1 Global Gene Therapy Drug Market Forecasts by Indication: Market Size ($m), Market Share (%), CAGR (%), 2017, 2022 and 2028

Table of Figures

Figure 1.1 Global Gene Therapy Market Segmentation Overview, 2017

Figure 2.1 Vectors Used in Gene Therapy Clinical Trials, 2017

Figure 3.1 Neovasculgen Forecast, Revenue ($m), 2017-2028

Figure 4.1 Gene Therapy World Market Forecast, Market Size ($m), AGR (%), 2017-2028

Figure 4.2 Global Gene Therapy Clinical Trials by Indication, 2017

Figure 4.3 Cancer Gene Therapy Pipeline by Phase of Development, 2017

Figure 4.4 Global Gene Therapy Market: Market Shares (%) by Indication, 2018

Figure 4.5 Global Market Forecast for Cancer Gene Therapies: Revenue ($m), 2017-2028

Figure 4.6 Rare Diseases Gene Therapy Pipeline by Phase of Development, 2017

Figure 4.7 Global Market Forecast for Rare Disease Gene Therapies: Revenue ($m), 2017-2028

Figure 4.8 Cardiovascular Gene Therapy Pipeline by Phase of Development, 2017

Figure 4.9 Global Market Forecast for Cardiovascular Disease Gene Therapies: Revenue ($m), 2017-2028

Figure 4.10 Global Market Forecast for Ophthalmologic Disease Gene Therapies: Revenue ($m), 2017-2028

Figure 4.11 Global Market Forecast for Other Disease Gene Therapies: Revenue ($m), 2017-2028

Figure 4.12 Global Market for Gene Therapy Drugs: Market Shares by Indication (%), 2022

Figure 4.13 Global Market for Gene Therapy Drugs: Market Shares by Indication (%), 2028

Figure 5.1 Gene Therapies in Clinical Development by Phase, 2017

Figure 5.2 Collategene Forecast, Revenue ($m), 2017-2028

Figure 5.3 BC-819 Forecast, Revenue ($m), 2017-2028

Figure 5.4 Lenti-D Forecast, Revenue ($m), 2017-2028

Figure 5.5 SPK-RPE65 Forecast, Revenue ($m), 2017-2028

Figure 5.6 Invossa Forecast, Revenue ($m), 2017-2028

Figure 5.7 Gene Therapy Market Strategy Analysis

Figure 5.8 Key Take Ways, Challenges and Application of CRISPR Market

Figure 5.9 Impact of CRISPR Technology on the Gene Therapy Market

Figure 8.1 Gene Therapy Drug Treatment Market Forecast by Indication: Market Sizes ($m), 2017, 2022, and 2028