Global Market for Gene Therapy 2019 - Product Pipeline, Market Size, & Forecasts

Table of Contents

1. REPORT OVERVIEW
1.1 Statement of the Report
1.2 Executive Summary

2. GENE THERAPY: AN INTRODUCTION
2.1 Gene Augmentation Therapy
2.2 Gene Inhibition Therapy
2.3 Suicide Gene Therapy
2.4 The Process of Gene Transfer in Gene Therapy
2.5 Ex Vivo vs. In Vivo Gene Therapies
2.5.1 Ex Vivo Gene Therapy
2.5.2 In Vivo Gene Therapy
2.6 Autologous vs. Allogeneic Gene Therapies

3. STATUS OF GENE THERAPY INDUSTRY
3.1 Number of Gene Therapy Companies in the World
3.2 Gene Therapy Pipeline Volumes, 1995-2018
3.3 Big Pharma’s Involvement in Gene Therapy Development
3.4 Number of Gene Therapies Anticipated to be Approved in the Next Three Years
3.5 Gene Therapy Product Candidates Granted RMAT Designation
3.5.1 EB-101
3.5.2 ABO-102
3.5.3 AT132
3.5.4 LentiGlobin
3.5.5 JCAR017
3.5.6 NSR-RE P1
3.5.7 P-BCMA-101
3.5.8 VY-AADC
3.6 Gene Therapy Companies Active in Neurodegenerative Disorders
3.6.1 Gene Therapy Developers for Neurodegenerative Disorders
3.6.2 Neurodegenerative Diseases Addressed by Gene Therapy Studies
3.6.3 Anticipated Events in Neurodegenerative Space of Gene Therapy
3.7 Important Events in Gene Therapy Space in 2018
3.8 Important Gene Therapy Startups
3.9 Gene Therapy Industry and Global Financings, 2018
3.10 Corporate Partnerships in 2018
3.11 Private Placements and Venture Financings in 2018
3.12 Public Offerings (IPOs & Follow-Ons) in 2018

4. GENE DELIVERY VEHICLES
4.1 Viral Vectors
4.1.1 Physical Properties of Commonly Used Viruses
4.2 Retrovirus Vectors
4.3 Adenovirus Vectors
4.4 Herpes Simplex Viral Vectors
4.5 Lentiviral Vectors
4.5.1 Utilization of Lentivirus Vectors by Disease Type in Clinical Trials
4.5.2 Companies Developing Lentivirus Vectors
4.6 Adeno-Associated Virus Vectors
4.6.1 Types of Diseases in Clinical Trials using AAV
4.6.2 Genes Transduced by AAV for Specific Diseases
4.6.3 Companies Developing AAV Vectors
4.7 The Predominantly Used Viral Vectors
4.8 Non-Viral Vectors
4.8.1 Microinjection
4.8.2 Particle Bombardment
4.8.3 Laser Irradiation
4.8.4 Electroporation
4.8.5 Magnetofection
4.8.6 Sonoporation

5. GENE THERAPY CLINICAL TRIALS
5.1 Gene Therapy Clinical Trials by Geography
5.2 Diseases Targeted by Gene Therapy Clinical Trials
5.3 Gene Types Transduced in Clinical Trials
5.4 Vectors used in Gene Therapy Clinical Trials
5.5 Gene Therapy Clinical Trials for Neurodegenerative Indications by Type
5.6 Domination of Oncology in Gene Therapy Development
5.8 Anticipated Clinical Events in Gene Therapy Space, 2019/2020

6. CHIMERIC ANTIGEN RECEPTOR T CELL (CAR-T CELL) THERAPY
6.1 Geographical Distribution of CAR-T Cell Therapy Companies
6.2 Select Events in CAR-T Space in 2018
6.3 Timeline of CAR-T Therapy Development
6.4 CAR-T Manufacturing Processes
6.4.1 Manufacturing of Autologous CAR-T Cells
6.4.1.1 Source of T Cells
6.4.1.2 T Cell Activation
6.4.1.3 Genetic Modification of T Cells
6.4.1.4 Expansion of CAR-T Cells
6.4.2 Manufacturing of Allogeneic CAR-T Cells
6.5 Structure of a CAR-T Cell
6.5.1 First Generation CAR-T Cells
6.5.2 Second Generation CAR-T Cells
6.5.3 Third Generation CAR-T Cells
6.5.4 Fourth Generation CAR-T Cells
6.6 CAR-T Cells: Mechanism of Action
6.7 Common Designs of CAR-T Constructs
6.8 Transfection by Vectors
6.9 CAR-T Cell Targets in Solid Malignancies
6.10 Target Diseases for CAR-T Cell Therapy

7. CAR-T PATENT LANDSCAPE
7.1 Number of CAR-T Patents, 2013-2018
7.2 CAR-T Patent Types
7.3 A Brief Snapshot of CAR-T Patent Landscape
7.3.1 Patents for Anti-CD19 CAR-T
7.3.2 Patents for Anti-BCMA CAR-T
7.3.3 Patents for Regulatable CAR-T
7.4 CAR-T Patents for Solid Tumors
7.5 Major CAR-T Patent Applicants

8. THE CAR-T CLINICAL TRIALS LANDSCAPE
8.1 The Surge in Number of CAR-T Clinical Trials
8.2 Percentage of CAR-T Clinical Trials by Target Antigen
8.3 Research Focus on CAR-T Trials by Indication
8.4 CAR-T Projects with Commercial Licensees
8.5 CAR-T Projects with Sole Involvement from Academia
8.6 Anti-CD19 Projects
8.7 CAR-T Studies in Multiple Myeloma (MM) and Acute Myeloid Leukemia (AML)
8.8 CAR-T Projects in Solid Tumors
8.9 CAR-T Projects using mRNA Electroporation for Gene Insertion
8.10 CAR-T Projects using Suicide Genes
8.11 Early-Stage CAR-T Assets
8.12 CAR-T Clinical Trials in China
8.12.1 CD19-Directed CAR-T Projects in China
8.12.2 Chinese CAR-T Projects Targeting Non-CD19 Antigens
8.12.3 Chinese CAR-T Projects in Solid Tumors
8.12.4 Chinese Projects using Fourth Generation CAR-T Constructs
8.12.5 Clinical-Stage CAR-T Projects in China
8.13 Anticipated Developments in CAR-T Space, 2019-2020

9. MEDICAL FACILITIES OFFERING CAR-T THERAPIES
9.1 U.S. Hospitals Offering CAR-T Therapies
9.2 Facilities Offering CAR-T Therapies in Germany
9.3 Hospitals Offering CAR-T Therapies in the U.K

10. GENE THERAPY PRODUCTS
10.1 Gendicine
10.2 Oncorine
10.3 Rexin-G
10.4 Neovasculgen (vascular endothelial growth factor gene)
10.5 Imlygic (talimogene laherparepvec)
10.6 Strimvelis (autologous CD34+ enriched cells)
10.7 Zalmoxis
10.8 Invossa (tonogenchoncel-L)
10.9 Kymriah (tisagenlecleucel-t)
10.9.1 Overall Remission Rates in Patients Treated with Kymriah
10.9.2 Overall Event-Free Survival Rate with Kymriah
10.10 Yescarta (axicabtagene ciloleucel)
10.10.1 Effectiveness of Yescarta
10.10.2 Clinical Benefits of Yescarta
10.10.3 Objective Response Rates (ORR) Reported for Yescarta
10.10.4 Complete Remission Rate (CRR) for Yescarta
10.11 Luxturna (voretigene neparvovec-rzyl)
10.12 Patient Impact of the Three Products Approved in the U.S. in 2017
10.13 Anticipated Number of Product Launches between 2018 and 2030
10.14 Number of Gene Therapies Available in Eight Major Markets as of January 2019
10.15 Five Forthcoming Gene Therapies

11. PRICING AND PAYMENT MODELS FOR GENE THERAPIES
11.1 CMS and Payments for Kymriah and Yescarta
11.2 Kymriah’s Outcome-Based Contracts (OBCs)
11.3 Luxturna’s Installment Payment Plan
11.4 Other Suggested Payment Options for Gene Therapies

12. MANUFACTURING OF CELLS AND VIRUS VECTORS
12.1 Cost of Manufacture of Cells and Virus Vectors
12.2 Cost of Manufacture in a Partially-Automated Facility
12.3 Cost of Manufacture in a Fully-Automated Facility
12.4 Net Present Cost (NPC) of the Three Methods
12.5 In-House Gene Therapy Manufacturing Capabilities
12.6 External Gene Therapy Manufacturing
12.7 Manufacturing Capacity in North America
12.8 Manufacturing Capacity in Europe

13. CMOs & CDMOs
13.1 Appropriate Phase for Outsourcing
13.2 Important Points Considered While Fixing a CDMO
13.3 In-House vs. Outsourcing
13.4 Number of CMPs and CDMOs in Europe
13.5 Projected Growth of Outsourcing Market
13.6 Projected Growth of Manufacturing Capacity

14. MARKET ANALYSIS
14.1 Global Market for Gene Therapy by Product Category
14.2 Competitive Landscape in Gene-Modified Cell Therapy Products
14.3 Estimated Sales of Top Ten Clinical-Stage Gene Therapy Projects
14.3.1 LentiGlobin to be on Top among the Top Ten
14.4 Global Market for Viral Vector and Plasmid DNA Manufacturing
14.5 Top Nine Assets of Gene Therapy between 2018 and 2024
14.5.1 Zolgensma (AVXS-101)
14.5.2 JCAR017, bb2121
14.5.3 Lentiglobin (BB305)
14.5.4 Valoctocogene roxaparvovec
14.5.5 ABO-102
14.5.6 Tabelecleucel
14.6 Net Present Value (NPV) of Select Gene Therapy Product Candidates

15. COMPANY PROFILES
15.1 4D Molecular Therapeutics, LLC
15.1.1 Key Capabilities
15.2 Abeona Therapeutics, Inc.
15.3 Adaptimmune Therapeutics, plc
15.4 Advanced Bioscience Laboratories, Inc
15.4.1 Biomanufacturing Services
15.4.2 Manufacturing of Virus-Based Products
15.4.3 Manufacturing of Protein-Based Products
15.4.4 Asceptic Filling Services
15.4.5 Immunology and Biomarker Testing
15.4.6 Contract Research Assays
15.4.7 Clinical Immunology Solutions
15.4.8 Preclinical Solutions
15.4.9 In vivo and ex vivo Preclinical Studies
15.4.10 Product Development Solutions
15.4.11 Government Partnering
15.4.12 Translational Product Development
15.5 Adverum Biotechnologies, Inc
15.6 Akouos, Inc
15.7 Aldevron, LLC
15.7.1 Services
15.8 Allogene Therapeutics, Inc
15.9 Ambys Medicines, Inc.
15.10 American Gene Technologies International, Inc
15.11 Amgen, Inc
15.11.1 Imlygic (talimogene laherparepvec)
15.12 Amicus Therapeutics, Inc.
15.13 AskBio
15.14 Audentes Therapeutics, Inc.
15.14.1 AT132
15.14.2 AT342
15.14.3 AT982
15.14.4 AT307
15.15 AveXis, Inc.
15.15.1 AVXS-101
15.16 AVROBIO, INC.
15.16.1 AVR-RD-01
15.16.2 AVR-RD-02
15.16.3 AVR-RD-03
15.16.4 AVR-RD-04
15.17 Benitec Biopharma Ltd
15.18 Bioverativ, Inc.
15.19 bluebird bio, Inc
15.19.1 Partnering
15.19.2 Current Partners
15.20 BlueRock Therapeutics
15.20.1 BlueRock’s Pipeline
15.21 Brammer Bio, LLC
15.21.1 Services
15.21.2 Product Development
15.21.3 Gene Therapy Manufacturing
15.21.4 Viral Vectors
15.22 Carina Biotech
15.22.1 CNA1003
15.23 CARsgen Therapeutics, Ltd
15.24 Casebia Therapeutics
15.25 Celyad SA
15.26 Cevec Pharmaceuticals GmbH
15.26.1 Services
15.27 CombiGene AB
15.27.1 Epilepsy Project
15.28 CRISPR Therapeutics AG
15.29 Delphi Genetics S.A
15.29.1 Services
15.30 DNAlite Therapeutics, Inc.
15.31 Editas Medicine, Inc
15.32 Elixirgen Therapeutics, LLC
15.33 Fibrocell Technologies, Inc.
15.33.1 Fibrocell’s Pipeline
15.34 FinVector Vision Therapies Oy
15.34.1 Services
15.35 Freeline Therapeutics, Ltd.
15.36 Gemini Therapeutics, Inc
15.37 GEG Tech
15.37.1 Lenti-ONE Vectors
15.38 Genethon
15.39 Generation Bio Co.
15.40 Homology Medicines, Inc
15.40.1 Gene Editing Platform
15.41 Horama SAS
15.42 Intellia Therapeutics Inc
15.43 Kite Pharma, Inc.
15.43.1 Yescarta
15.44 Kolon Lifesciences, Inc.
15.44.1 Invossa-K inj
15.45 Krystal Biotech, Inc.
15.46 LakePharma
15.46.1 Viral Vector Services
15.47 Lonza Group, Ltd.
15.47.1 Custom Development and Manufacturing
15.48 Lysogene S.A
15.48.1 MPS IIIA
15.48.2 GM1 Gangliosidosis
15.49 MeiraGTx Holdings plc
15.49.1 Meira’s Pipeline
15.49.1.1 AAV-CNGB3
15.49.1.2 AAV-RPE65
15.49.1.3 AAV-RPGR
15.49.1.4 AAV-AQP1
15.49.1.5 AAV-GAD
15.49.1.6 AAV-UPF1
15.50 MolMed S.p.A
15.50.1 MolMed’s Clinical Trial Programs
15.50.2 MolMed’s GMP Solutions
15.51 Mustang Bio, Inc.
15.51.1 MB-101
15.51.2 MB-102
15.51.3 MB-103
15.51.4 MB-104
15.51.5 MB-105
15.51.6 MB-106
15.51.7 MB-107
15.52 Myonexus Therapeutics, Inc
15.53 Nightstar Therapeutics plc
15.54 Novartis AG
15.54.1 Kymriah
15.55 Orchard Therapeutics plc
15.55.1 Strimvelis
15.55.2 OTL-101
15.55.3 OTL-102
15.55.4 OTL-103
15.55.5 OTL-200
15.55.6 OTL-201
15.55.7 OTL-202
15.55.8 OTL-300
15.56 OxfordBioMedica plc
15.56.1 Oxford BioMedica’s Partnering Activities
15.57 Oxford Genetics, Ltd.
15.57.1 Scalable AAV Custom Solutions
15.58 Paragon Bioservices, Inc.
15.58.1 Services
15.59 Posedia Therapeutics, Inc.
15.60 Precision BioSciences, Inc.
15.60.1 ARCUS Genome Editing Platform
15.61 Prevail Therapeutics, Inc.
15.61.1 Prevail’s Focus
15.62 PTC Therapeutics, Inc.
15.63 Quethera, Ltd.
15.63.1 Technology
15.64 REGENXBIO, INC.
15.64.1 NAV Technology
15.64.2 Therapeutic Programs
15.64.3 Product Candidates
15.64.3.1 RGX-314
15.64.3.2 RGX-501
15.64.3.3 RGX-111
15.64.3.4 RGX-121
15.64.3.5 RGX-181
15.64.4 Partnerships and Licensing
15.65 Rocket Pharmaceuticals, Inc.
15.66 Sangamo Therapeutics, Inc
15.67 Sarepta Therapeutics, Inc
15.68 Sibiono GeneTech Co. Ltd.
15.68.1 Gendicine
15.69 Sirion Biotech, GmbH
15.69.1 Viral Vectors
15.69.2 Technologies
15.70 Solid Biosciences, Inc
15.70.1 SGT-001
15.71 Spark Therapeutics, Inc
15.71.1 Luxturna
15.71.2 Spark Therapeutics’ Pipeline
15.72 Synpromics, Ltd.
15.72.1 Technology
15.72.2 PromPT
15.73 Takara Bio, Inc
15.73.1 Services & Support
15.74 The Cell and Gene Therapy Catapult
15.74.1 Catapult’s Industrialization Team
15.74.2 Catapult’s Manufacturing Center at Stevenage
15.74.3 Catapult’s Regulatory Team
15.75 Translate Bio, Inc.
15.76 Ultragenyx Pharmaceutical, Inc.
15.76.1 DTX301
15.76.2 DTX401
15.77 uniQure N.V.
15.78 Vybion, Inc.
15.78.1 INT41
15.79 Vigene Biosciences
15.80 Virovek, Inc.
15.80.1 Custom-Made AAVs
15.81 Vivebiotech SL
15.81.1 Lentisoma Technology
15.81.2 Services
15.82 Vivet Therapeutics
15.82.1 Vivet’s Approach
15.83 Voyager Therapeutics, Inc

List of Figures
Figure 2.1: Diagrammatic Representation of Gene Augmentation Therapy
Figure 2.2: Schematic Representation of Gene Inhibition Therapy
Figure 2.3: Diagrammatic Representation of Suicide Gene Therapy
Figure 2.4: Illustration Showing the Viral Vector Transferring Gene into the Patient’s Cell
Figure 2.5: Autologous and Allogeneic Gene Therapies
Figure 3.1: Number of Gene and Gene-Based Medicine Companies by Geography
Figure 3.2: Gene Therapy Pipeline Volumes, 1995-2018
Figure 3.3: Minor Role Played by Big Pharma in Gene Therapy
Figure 3.4: Number of Gene Therapies to Gain FDA Approval by 2022
Figure 3.5: Gene Therapy Companies in Neurodegenerative Disorders by Geography
Figure 3.6: Gene Therapy Industry and Global Financings, 2018
Figure 4.1: Utilization of Lentivirus Vectors by Disease Type in Clinical Trials
Figure 4.2: Companies Developing Lentivirus Vectors
Figure 4.3: Types of Diseases in Clinical Trials using AAV
Figure 4.4: Companies Developing AAV Vectors
Figure 5.1: Number of Gene Therapy Clinical Trials, 2017-2018
Figure 5.2: Gene Therapy Clinical Trials by Geography, 2018
Figure 5.3: Diseases Targeted by Gene Therapy, 2018
Figure 5.4: Gene Types Transduced in Clinical Trials
Figure 5.6: Vectors used in Gene Therapy Clinical Trials
Figure 5.7: Gene Therapy Clinical Trials for Neurodegenerative Indications by Type, 2018
Figure 5.8: Domination of Oncology in Gene Therapy Development, 2018
Figure 5.9: Dominance of Viral Vectors in Gene Therapy Drug Development
Figure 6.1: Geographical Distribution of CAR-T Cell Therapy Companies
Figure 6.3: The Process of CAR-T Cell Manufacturing
Figure 6.4: Building a CAR-T Cell and its Binding to Tumor Antigen
Figure 6.5: First Generation CAR-T Cells
Figure 6.6: Second Generation CAR-T Cells
Figure 6.7: Third Generation CAR-T Cells
Figure 6.8: Fourth Generation CAR-T Cells
Figure 6.9: Recognition and Killing of Cancer Cells by CAR-T Cells
Figure 6.10: Most Frequent CAR-T Targets in Solid Malignancies
Figure 7.1: Number of CAR-T Patents, 2013-2018
Figure 7.1: Major CAR-T Patent Applicants
Figure 8.1: The Surge in Number of CAR-T Clinical Trials, 2007-2018
Figure 8.2: Percentage of CAR-T Clinical Trials by Target Antigen
Figure 8.3: Research Focus on CAR-T Trials by Indication
Figure 8.4: CAR-T Cells Targeting Antigens on Solid Tumors
Figure 10.1: Anticipated Number of Product Launches between 2018 and 2030
Figure 12.1: Expenditure Categories in Cell Manufacturing
Figure 12.2: The Impact of Head Count on Labor Cost
Figure 12.3: Cost of Manufacture in a Partially-Automated Facility
Figure 12.4: Cost of Manufacture in a Fully-Automated Facility
Figure 12.5: Net Present Cost (NPC) of the Three Methods
Figure 13.1: Appropriate Phase for Outsourcing
Figure 13.2: Important Points Considered While Fixing a CDMO
Figure 13.3: Share of In-House and Outsourcing
Figure 13.4: Number of CMPs and CDMOs in Europe
Figure 13.5: Projected Growth of Outsourcing Market, 2018-2025
Figure 14.1: Global Market for Gene Therapy Products, 2018-2025
Figure 14.2: Global Market for Gene Therapy by Product Category, 2018-2025
Figure 14.3: Vector and Plasmid DNA Manufacturing Market by Geography, 2018-2025

List of Tables
Table 3.1: Number of Gene Therapy Companies by Geography
Table 3.2: Gene Therapy Product Candidates Granted RMAT Designation
Table 3.3: Gene Therapy Companies in Neurodegenerative Disorders by Geography
Table 3.4: Gene Therapy Developers for Neurodegenerative Disorders
Table 3.5: Neurodegenerative Diseases Addressed by Gene Therapy Studies
Table 3.6: Select Anticipated Events in Neurodegenerative Space of Gene Therapy
Table 3.7: Select Events in Gene Therapy Space
Table 3.8: Important Gene Therapy Startups
Table 3.9: Gene Therapy Industry and Global Financings
Table 3.10: Corporate Partnerships in 2018
Table 3.11: Private Placements and Venture Financings
Table 3.12: Public Offerings (IPOs & Follow-Ons)
Table 4.2: Retroviral Vectors: A Snapshot
Table 4.3: Adenovirus Vectors: A Snapshot
Table 4.4: Herpes Simplex Viral Vectors: A Snapshot
Table 4.5: Lentivirus Vectors: A Snapshot
Table 4.6: Adeno-Associated Virus Vectors: A Snapshot
Table 4.7: Genes Transduced by AAV for Specific Diseases
Table 4.8: The Predominantly Used Viral Vectors
Table 4.9: Microinjection Method of Gene Delivery
Table 4.10: Particle Bombardment Method of Gene Delivery
Table 4.11: Laser Irradiation Method of Gene Delivery
Table 4.12: Electroporation Method of Gene Delivery
Table 4.13: Magnetofection Method of Gene Delivery
Table 4.14: Sonoporation Method of Gene Delivery
Table 5.1: Number of Gene Therapy Clinical Trials
Table 5.2: Gene Therapy Clinical Trials by Geography
Table 5.3: Diseases Targeted by Gene Therapy
Table 5.4: Gene Types Transduced in Clinical Trials
Table 5.6: Vectors used in Gene Therapy Clinical Trials
Table 5.7: Gene Therapy Clinical Trials for Neurodegenerative Indications by Type
Table 5.8: Domination of Oncology in Gene Therapy Development
Table 5.9: Select Anticipated Clinical Data and Events in 2019/2020
Table 6.1: Geographical Distribution of CAR-T Cell Therapy Companies
Table 6.2: Select Events in CAR-T Space
Table 6.3: Timeline of CAR-T Cell Therapy Development
Table 6.4: Different Types of CAR-T Constructs, Corresponding Antigens and Indications
Table 6.5: Most Frequent CAR-T Targets in Solid Malignancies
Table 6.6: Target Diseases and Target Antigens for CAR-T Cell Therapy
Table 7.1: Major CAR-T Patent Holders
Table 7.2: Select List of CAR-T Patents
Table 7.3: Major CAR-T Patent Applicants
Table 8.1: The Surge in Number of CAR-T Clinical Trials
Table 8.2: Percentage of CAR-T Clinical Trials by Target Antigen
Table 8.3: Research Focus on CAR-T Trials by Indication
Table 8.4: CAR-T Projects with Commercial Licensees
Table 8.5: CAR-T Projects with Sole Involvement from Academia
Table 8.6: Anti-CD19 Projects
Table 8.7: CAR-T Studies in Multiple Myeloma (MM) and Acute Myeloid Leukemia (AML)
Table 8.8: CAR-T Projects in Solid Tumors
Table 8.9: CAR-T Projects using mRNA Electroporation for Gene Insertion
Table 8.10: CAR-T Projects using Suicide Genes
Table 8.11: Early-Stage CAR-T Assets
Table 8.12: Distribution of CAR-T Clinical Trials in China
Table 8.13: CD19-Directed CAR-T Projects in China
Table 8.14: Chinese CAR-T Projects Targeting Non-CD19 Antigens
Table 8.15: Select Chinese CAR-T Projects in Solid Tumors
Table 8.16: Chinese Projects using Fourth Generation CAR-T Constructs
Table 8.17: Clinical-Stage CAR-T Projects in China
Table 8.18: Anticipated Developments in CAR-T Space
Table 9.1: U.S. Medical Facilities Offering CAR-T Therapy
Table 9.2: Facilities Offering CAR-T Therapies in the U.K.
Table 10.1: Approved Gene Therapy Products
Table 10.2: Overall Remission Rates in Patients Treated with Kymriah
Table 10.3: Event-Free Survival at Six and 12 Months in ALL Patients
Table 10.4: Yescarta’s Timeline of Development
Table 10.5: Objective Response Rates (ORR) Reported for Yescarta
Table 10.6: CRR for Yescarta Compared with CRR of SCHOLAR-1 Trial
Table 10.7: The Development Timeline of Luxturna
Table 10.8: Patient Impact of the Three Products Approved in the U.S. in 2017
Table 10.9: Number of Gene Therapies in Eight Major Markets a
Table 10.10: Details of the Five Forthcoming Gene Therapies
Table 11.1: Cost and Market Size for Organ Transplants in the U.S
Table 11.2: Cost and Market Size for Three Gene Therapies in the U.S.
Table 11.3: Suggested Financing and Payment Options for Gene Therapies
Table 12.1: Expenditure Categories in Cell Manufacturing
Table 12.2: The Impact of Head Count on Labor Cost
Table 12.3: Cost of Manufacture in a Partially-Automated Facility
Table 12.4: Cost of Manufacture in a Fully-Automated Facility
Table 12.5: Companies with In-House Gene Therapy Manufacturing Capability
Table 12.6: External Gene Therapy Manufacturing
Table 12.7: Manufacturing Capacity in North America
Table 12.8: Manufacturing Capacity in Europe
Table 13.1: Select CDMOs
Table 13.2: Appropriate Phase for Outsourcing
Table 13.3: Important Points Considered While Fixing a CDMO
Table 13.4: Share of In-House and Outsourcing
Table 14.1: Global Market for Gene Therapy Products, 2018-2025
Table 14.2: Global Market for Gene Therapy by Product Category, 2018-2025
Table 14.3: Competitive Landscape in Gene-Modified Cell Therapy Products
Table 14.4: Estimated Sales of Top Ten Clinical-Stage Gene Therapy Projects
Table 14.5: Vector and Plasmid DNA Manufacturing Market by Geography, 2018-2025
Table 14.6: Top Nine Assets of Gene Therapies between 2018 and 2024
Table 14.7: Net Present Value (NPV) of Select Gene Therapy Product Candidates
Table 14.8: Net Present Value (NPV) of Yescarta and Kymriah
Table 15.1: 4D Molecular Therapeutics’ Product Pipeline
Table 15.2: Abeona’s Product Pipeline
Table 15.3: Adaptimmune’s Pipeline Programs
Table 15.4: Allogene Therapeutics’ Pipeline of Product Candidates
Table 15.5: Amicus’ Gene Therapy Pipeline
Table 15.6: AskBio’s Therapeutic Pipeline
Table 15.7: Audentes’ Product Candidates
Table 15.8: AVROBIO’s Gene Therapy Programs
Table 15.9: Benitec’s Product Pipeline using Gene Silencing Technologies
Table 15.10: Bioverativ’s Product Pipeline
Table 15.11: bluebird bio’s Product Pipeline
Table 15.12: CARsgen Therapeutics’ Product Candidates
Table 15.13: Casebia’s Pipeline at a Glance
Table 15.14: Celyad’s Program Status at a Glance
Table 15.15: CRISPR Therapeutics’ Clinical Programs
Table 15.16: Editas’ Diverse Product Pipeline
Table 15.17: Elixirgen’s Pipeline for Genetic and Age-Related Diseases
Table 15.18: Fibrocell’s Personalized Biologics Pipeline
Table 15.19: Freeline’s Pipeline of Gene Therapy Treatments in Development
Table 15.20: Gemini’s Pipeline
Table 15.21: Genethon’s Products in Development
Table 15.22: Horama’s Product Pipeline
Table 15.23: Intellia’s in vivo and ex vivo RD Pipeline
Table 15.24: Kite’s CAR-T Pipeline
Table 15.25: Kolon’s Gene Therapeutics in Development
Table 15.26: Krystal Biotech’s P[peline Programs for Orphan Skin Diseases
Table 15.27: Meira’s Pipeline
Table 15.28: MolMed’s Product Candidates
Table 15.29: Mustang Bio’s Product Pipeline
Table 15.30: Myonexus’ Clinical Stage LGMD Gene Therapy Pipeline
Table 15.31: Nightstar Therapeutics’ Pipeline Programs
Table 15.32: Orchard Therapeutics’ Pipeline
Table 15.33: Oxford BioMedica’s Proprietary Product Pipeline
Table 15.34: Oxford BioMedica’s Partnered Products
Table 15.35: Oxford BioMedica’s IP-Enabled Royalty-Bearing Products
Table 15.36: Posedia’s Product Pipeline
Table 15.37: PTC’s Gene Therapy Programs for CNS Disorders
Table 15.38: REGENXBIO’S Product Pipeline
Table 15.39: REGENXBIO’S & Licensees’ Pipeline
Table 15.40: Sangamo’s Proprietary Programs
Table 15.41: Sangamo’s Programs for Partners
Table 15.42: Sarepta’s Huge Product Pipeline
Table 15.43: Spark Therapeutics’ Pipeline
Table 15.44: Translate Bio’s Lead Product Candidates
Table 15.45: uniQure’s Product Pipeline
Table 15.46: Vybion’s Drug Pipeline

Companies Mentioned

• 4D Molecular Therapeutics, LLC
• Abeona Therapeutics, Inc.
• Adaptimmune Therapeutics, plc
• Advanced Bioscience Laboratories, Inc
• Adverum Biotechnologies, Inc
• Akouos, Inc
• Aldevron, LLC
• Allogene Therapeutics, Inc
• Ambys Medicines, Inc.
• American Gene Technologies International, Inc
• Amgen, Inc
• Amicus Therapeutics, Inc.
• AskBio
• Audentes Therapeutics, Inc.
• AveXis, Inc.
• AVROBIO, INC.
• Benitec Biopharma Ltd
• Bioverativ, Inc.
• bluebird bio, Inc
• BlueRock Therapeutics
• Brammer Bio, LLC
• Carina Biotech
• CARsgen Therapeutics, Ltd
• Casebia Therapeutics
• Celyad SA
• Cevec Pharmaceuticals GmbH
• CombiGene AB
• CRISPR Therapeutics AG
• Delphi Genetics S.A
• DNAlite Therapeutics, Inc.
• Editas Medicine, Inc
• Elixirgen Therapeutics, LLC
• Fibrocell Technologies, Inc.
• FinVector Vision Therapies Oy
• Freeline Therapeutics, Ltd.
• GEG Tech
• Gemini Therapeutics, Inc
• Generation Bio Co.
• Genethon
• Homology Medicines, Inc
• Horama SAS
• Intellia Therapeutics Inc
• Kite Pharma, Inc.
• Kolon Lifesciences, Inc.
• Krystal Biotech, Inc.
• LakePharma
• Lonza Group, Ltd.
• Lysogene S.A
• MeiraGTx Holdings plc
• MolMed S.p.A
• Mustang Bio, Inc.
• Myonexus Therapeutics, Inc
• Nightstar Therapeutics plc
• Novartis AG
• Orchard Therapeutics plc
• Oxford Genetics, Ltd.
• OxfordBioMedica plc
• Paragon Bioservices, Inc.
• Posedia Therapeutics, Inc.
• Precision BioSciences, Inc.
• Prevail Therapeutics, Inc.
• PTC Therapeutics, Inc.
• Quethera, Ltd.
• REGENXBIO, INC.
• Rocket Pharmaceuticals, Inc.
• Sangamo Therapeutics, Inc
• Sarepta Therapeutics, Inc
• Sibiono GeneTech Co. Ltd.
• Sirion Biotech, GmbH
• Solid Biosciences, Inc
• Spark Therapeutics, Inc
• Synpromics, Ltd.
• Takara Bio, Inc
• The Cell and Gene Therapy Catapult
• Translate Bio, Inc.
• Ultragenyx Pharmaceutical, Inc.
• uniQure N.V.
• Vigene Biosciences
• Virovek, Inc.
• Vivebiotech SL
• Vivet Therapeutics
• Voyager Therapeutics, Inc
• Vybion, Inc.

Methodology

The content and statistics contained within the publisher's reports are compiled using a broad range of sources, as described below.

Input Sources

• Clinical Trial Databases (ClinicalTrials.gov, International Clinical Trials Registry Platform, European Union Clinical Trials Register, Chinese Clinical Trial Registry, Others)
• Scientific Publication Databases (PubMed, Highwire Press, Google Scholar)
• Patent Databases (United States Patent and Trade Office, World Intellectual Property Organization, Google Patent Search)
• Grant Funding Databases (RePORT Database, CIRM, MRC, Wellcome Trust - UK, Others)
• Product Launch Announcements (Trade Journals, Google News)
• Industry Events (Google News, Google Alerts, Press Releases)
• Company News (SEC Filings, Investor Publications, Historical Performance)
• Social Analytics (Google Adwords, Google Trends, Twitter, Topsy.com, Hashtagify.me, BuzzSumo.com)
• Interviews with Stem Cell Industry Leaders

Research & Analysis Methodologies

The publisher employs the following techniques for deriving its market research:

• Historical Databases: As the first and only market research firm to specialize in the stem cell industry, the publisher has 13+ years of historical data on each segment of the stem cell the industry. This provides an extremely rare and robust database for establishing market size determinations, as well as making future market predictions.
• Prolific Interviews with Industry Leaders: As the global leader in stem cell industry data, the publisher has interviewed hundreds of leaders from across the stem cell industry, including the CEO of FUJIFILM CDI, FUJIFILM Irvine Scientific, Pluristem Therapies, Celularity, and many others.
• Industry Relationships: The research team and its President/Founder, Cade Hildreth, Chair and present at a wide range of stem cell industry events, including Phacilitate's Advanced Therapies Week, World Stem Cell Summit (WSCS), Perinatal Stem Cell Society Congress, AABB's International Cord Blood Symposium (ICBS), and other events hosted within the U.S. and worldwide.
• Global Integrated Feedback: Because the publisher maintains the world's largest stem cell industry news site that is read by nearly a million unique readers per year and the company has large social media audiences (25.7K+ followers on Linked, 21.2K+ followers on Twitter, and 4.3K+ followers on Facebook), the publisher is able to publish content relevant to the industry and receive immediate feedback/input from a global community of readers. In short, the publisher's data is crowd-sourced from market participants worldwide, including those in diverse geographic regions.
• Preliminary Research: In addition to the interviews described above, the publisher conducts market surveys, executes social media polls, and aggregates market data from stem cell industry announcements, press releases, and corporate filings/presentations.
• Secondary Research: The publisher summarizes, collects and synthesizes existing market research that is relevant to the market area of interest.
• Future Projections: Using the resources described above, the publisher is uniquely positioned to make future projections about market size, market growth by segment, market trends, technology evolution, funding activities (financing rounds, M&A, and IPOs), and importantly, market leadership (market share by company).