Global Gene Therapy Market Analysis & Forecast to 2022
This report provides the reader with:
- Current Global Market Worth and Forecast with CAGR Through 2022
- Sub-Market Worth by Therapeutic Area (Cancer, Rare Diseases, Cardiovascular, Neurological, Ocular) and Forecast with CAGR Through 2022
- Sub-Market Worth by Geography (Americas, Europe, RoW) and Forecast with CAGR Through 2022
- Sub-Market Worth by Technology (Gene Product, Service, Viral Vectors) and Forecast with CAGR Through 2022
- Insight into gene therapy technologies, challenges associated with developing therapeutic genes and disadvantages of gene therapy.
- Full outline of the gene therapy industry from the formative years through to products discovered during 1990 and 2017.
- Detailed descriptions of commercialized products approved between 2003 and 2017 that include: Gendicine, Rexin-G, Oncorine, Neovasculgen, Glybera, Imlygic, Strimvelis, Zalmoxis, Kymriah, Yescarta and Luxturna.
- Description of seven of the Phase III product candidates that include: Generx, Collategene, LentiGlobin, Lenti-D, VM-202, Invosa and GS-010.
- Description of 21 Phase II product candidates that are set to have significant market share.
- Commercialization status of gene therapies in by geographic region
- Evaluation of gene therapy pricing
- Description of the firstever warranty offer by GSK for Strimvelis.
- A detailed analysis of various types of viruses used as vectors.
- Description of clinical applications of gene therapy and the various genetic and infectious diseases addressed by gene therapy.
- Description of 77 companies that are directly and indirectly associated with gene therapy industry.
- What is the size of gene therapy market?
- What is the CAGR and market size over the next five years?
- What are the different sub-markets and their worth/CAGR over the next five years?
- What is gene augmentation therapy?
- What is suicide gene therapy?
- How is ex vivo gene delivery different from in vivo gene delivery?
- What are the types of gene therapies classified on the basis of targeted cell types?
- What is the role of CRISPR technology in gene therapy?
- What are the approved gene therapy products?
- How many gene therapy product candidates have reached the Phase III stage?
- How many Phase II gene therapy product candidates are there?
- What is the commercialization status of gene therapies in E.U. member countries?
- What are the prices of gene therapy products?
- What are the reasons for this extortionate pricing of gene therapies?
- Which company is offering warranty for its gene- therapy product?
- What is the current strength of gene therapy industry?
- Is it true that the real strength of gene therapy industry is based on the number of clinical trials?
- What is the total number of ongoing clinical trials as of 2017?
- What is the distribution of clinical trials by geography?
- Which countries are associated with gene therapy clinical trials?
- What are the major indications addressed by the clinical trials?
- Which genes are transferred in these clinical trials?
- How many Big Pharma are associated with the gene therapy industry?
- What are non-viral and viral vectors?
- What are the various features of viral vectors?
- Which viral vectors are predominantly used in gene therapy clinical trials?
- What are the major diseases addressed by therapeutic genes?
- Where is the gene therapy market heading, and what opportunities and challenges will it face?
- 1.0 Introduction
- 1.1 Executive Summary
- 1.2 About this Report
- 1.3 Key Questions Answered in this Report
- 2.0 Gene Therapy: The Basics
- 2.1 Gene Therapy Techniques
- 2.1.1 Gene Augmentation Therapy
- 2.1.2 Gene Inhibition Therapy
- 2.1.3 Suicide Gene Therapy
- 2.2 Gene Therapy Routes
- 2.2.1 Ex Vivo Gene Delivery Route
- 2.2.2 In vivo Gene Delivery Route
- 2.3 CRISPR: Latest Technology in Gene Therapy
- 2.4 Challenges Associated with Developing Gene Therapies
- 2.5 Disadvantages of Gene Therapy
- 2.5.1 Short-Lived Nature of Gene Therapy
- 2.5.2 Immune Response
- 2.5.3 Problems with Viral Vectors
- 2.5.4 Multigenic Disorders
- 2.5.5 Insertional Mutagenesis
- 2.6 Concept to Product Candidates and Products
- 3.0 Historic Overview of Gene Therapy
- 3.1 The Formative Years of Gene Therapy
- 3.2 The Productive Years of Gene Therapy
- 3.2.1 Gendicine
- 3.2.2 Oncorine
- 3.2.3 Rexin-G
- 3.2.4 Neovasculgen
- 3.2.5 Glybera
- 3.2.6 Imlygic
- 3.2.7 Strimvelis
- 3.2.8 Zalmoxis
- 4.0 Gene Therapy Phase III Product Candidates
- 4.1 CardioNovo (Generx)
- 4.2 Collategene
- 4.3 LentiGlobin
- 4.4 Lenti-D
- 4.5 VM-202
- 4.6 Invossa
- 4.7 GS010
- 4.8 ADA-Lentiviral Gene Therapy
- 5.0 Gene Therapy Phase II Product Candidates
- 5.1 hF-IX gene/Hemophilia B
- 5.2 NaGlu gene/San Fillipo B Syndrome
- 5.3 StarGen/Stargardt disease
- 5.4 UshStat/Usher Syndrome Type 1B
- 5.5 Retinostat/Wet AMD
- 5.6 Lenti-D/Childhood Cerebral ALD
- 5.7 LentiGlobin/Beta Thalassemia
- 5.8 SB-728/HIV/AIDS
- 5.9 CERE-110/Alzheimer’s disease
- 5.10 AATD/Alpha-1 Antitrypsin deficiency
- 5.11 RS1 gene/X-linked juvenile Retinoschisis (XLRS)
- 5.12 SPK-CHM/Choroideremia
- 5.13 SPK-FIX/Hemophilia B
- 5.14 AMG0001/Primary Lymphedema
- 5.15 AMG0001/Ischemic heart disease
- 5.16 WASp /Wiskott-Aldrich syndrome
- 5.17 X-linked CGD
- 5.18 GSK2696273/ADA gene
- 5.19 VM106/Chronic Granulomatous Disease
- 5.20 VY-AADC01/Parkinson’s disease
- 6.0 Commercialization, Cost and Warranty of Gene Therapies
- 6.1 Reasons for the Extortionate Price
- 6.1.1 High Cost of Manufacturing
- 6.1.2 High Cost of Delivery
- 6.1.4 Lack of Competition
- 6.1.5 Curing the Impossible
- 6.2 Strategies to Make Gene Therapy Affordable
- 6.3 The Age of First-Ever Warranty for Gene Therapy
- 7.0 The Strength of Gene Therapy Industry
- 7.1 Development of Appropriate Gene Delivery Vehicles
- 7.2 CRISPR-Based Therapy
- 7.3 Early Clinical Gene Therapy Protocols
- 7.4 Worldwide Scenario of Gene Therapy Clinical Trials
- 7.4.1 Geographical Distribution of Gene Therapy Clinical Trials
- 7.4.2 Gene Therapy Clinical Trials by Country
- 7.4.3 Major Indications Addressed by Gene Therapy Clinical Trials
- 7.4.4 Gene Types Transferred
- 7.4.5 Status of Gene Therapy Clinical Trials
- 7.5 Big Pharma’s Bold Venturing into Gene Therapy
- 7.5.1 Novartis’ Deal with GenVec
- 7.5.2 Strategic Alliance between GlaxoSmithKline and Fondazione Telethon
- 7.5.3 Amgen’s Acquisition of BioVex
- 7.5.4 Chiesi’s Rights to uniQure’s Glybera
- 7.5.5 Bayer’s Collaboration with Dimension Therapeutics
- 7.5.6 Genzyme’s (Sanofi) Collaboration with University of Florida
- 7.5.7 Pfizer’s Collaboration with Spark Therapeutics
- 7.5.8 Biogen Idec’s Collaboration with AGCT
- 7.5.9 Sanofi’s Tie-Up with Voyager Therapeutics
- 7.5.10 Bristol Myers Squibb’s Investment in uniQure
- 7.5.11 Joint Venture between Bayer and CRISPR Therapeutics AG
- 7.5.12 Flow of Funds to Gene Therapy Companies
- 7.6 Venture Investments by Body Systems
- 8.0 Vectors for Gene Delivery
- 8.1 Non-Viral Vectors
- 8.1.1 Inorganic Particles
- 8.1.1.1 Poly(lactic-co-glycolic acid) (PLGA) and poly lactic acid (PLA)
- 8.1.1.2 Chitosan
- 8.1.1.3 Poly(ethylene imine) (PEI)
- 8.1.1.4 Dendrimers
- 8.1.1.5 Polymethacrylates
- 8.1.2 Synthetic/Natural Biodegradable Particles
- 8.1.2.1 Cationic Liposomes
- 8.1.2.2 Lipid Nanoemulsions
- 8.1.2.3 Solid Lipid Nanoparticles (SLN)
- 8.1.2.4 Poly-L-Lysine
- 8.1.3 Physical Methods
- 8.1.3.1 Needle Injection
- 8.1.3.2 Gene Gun
- 8.1.3.3 Electroporation
- 8.1.3.4 Sonoporation
- 8.1.3.5 Photoporation
- 8.1.3.6 Magnetofection
- 8.1.3.7 Hydroporation
- 8.2 Viral Vectors for Gene Therapy
- 8.2.1 Key Properties of Viral Vectors
- 8.2.2 Best-Suited Viral Vectors for Gene Therapy
- 8.2.3 Potential Barriers for the Use of Viral Vectors
- 8.2.4 Types of Viral Vectors
- 8.2.4.1 Adenovirus Vectors
- 8.2.4.2 Retrovirus Vectors
- 8.2.4.3 Adeno-Associated Virus (AAV)
- 8.2.4.4 Lentivirus Vectors
- 8.2.4.5 Vaccinia Virus
- 8.2.5 Clinical Translation of Viral Vectors
- 8.2.6 Viral Vectors used in Clinical Trials
- 8.2.7 Culture Systems for Viral Vector Production
- 9.0 Clinical Applications of Gene Therapy
- 9.1 Leber Congenital Amaurosis (LCA)
- 9.1.1 Frequency
- 9.1.2 Genetic Basis of Leber Congenital Amaurosis
- 9.1.3 Pattern of Inheritance
- 9.1.4 Gene Therapy for LCA
- 9.2 Stargardt Macular Degeneration (SMD)
- 9.2.1 Frequency of SMD
- 9.2.2 Genetic Basis of SMD
- 9.2.3 Pattern of Inheritance
- 9.2.4 Gene Therapy
- 9.3 Choroideremia
- 9.3.1 Frequency
- 9.3.2 Genetic Basis of Choroideremia
- 9.3.3 Pattern of Inheritance
- 9.3.4 Gene Therapy
- 9.4 Leber Hereditary Optic Neuropathy (LHON)
- 9.4.1 Frequency of LHON
- 9.4.2 Genetic Basis of LHON
- 9.4.3 Pattern of Inheritance
- 9.4.4 Gene Therapy
- 9.5 Parkinson Disease
- 9.5.1 Frequency of Parkinson Disease
- 9.5.2 Genetic Basis of Parkinson Disease
- 9.5.3 Inheritance of Parkinson Disease
- 9.5.4 Current Treatment for Parkinson Disease
- 9.5.5 Gene Therapy for Parkinson Disease
- 9.6 Spinal Muscle Atrophy
- 9.6.1 Genetic Basis
- 9.6.2 Pattern of Inheritance
- 9.6.3 Gene Therapy
- 9.7 Alzheimer Disease
- 9.7.1 Frequency
- 9.7.2 Genetic Basis
- 9.7.3 Pattern of Inheritance
- 9.7.4 Gene Therapy
- 9.8 Cystic Fibrosis
- 9.8.1 Genetic Basis
- 9.8.2 Inheritance of CF
- 9.8.3 Frequency of CF
- 9.8.4 Currently Available Treatments for CF
- 9.8.5 Gene Therapy for Cystic Fibrosis
- 9.9.1 Frequency
- 9.9.2 Genetic Basis
- 9.9.3 Pattern of Inheritance
- 9.9.4 Currently Available Treatments
- 9.9.5 Gene Therapy
- 9.10 X-Linked Adrenoleukodystrophy
- 9.10.1 Frequency
- 9.10.2 Genetic Basis
- 9.10.3 Pattern of Inheritance
- 9.10.4 Gene Therapy
- 9.11 Pompe Disease
- 9.11.1 Frequency
- 9.11.2 Genetic Basis
- 9.11.3 Pattern of Inheritance
- 9.11.4 Gene Therapy
- 9.12 Batten Disease (CLN3 Disease)
- 9.12.1 Frequency of Batten Disease
- 9.12.2 Genetic Basis of Batten Disease
- 9.12.3 Pattern of Inheritance
- 9.12.4 Gene Therapy for Batten Disease
- 9.13 Metachromatic Leukodystrophy
- 9.13.1 Frequency
- 9.13.2 Genetic Basis
- 9.13.3 Pattern of Inheritance
- 9.13.4 Gene Therapy
- 9.14 Sanfilippo Syndrome
- 9.14.1 Frequency
- 9.14.2 Genetic Basis
- 9.14.3 Patern of Inheritance
- 9.14.4 Gene Therapy
- 9.15 Hunter Syndrome
- 9.15.1 Genetic Basis
- 9.15.2 Pattern of Inheritance
- 9.15.3 Gene Therapy
- 9.16 Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
- 9.16.1 Frequency
- 9.16.2 Genetic Basis
- 9.16.3 Pattern of Inheritance
- 9.16.4 Gene Therapy
- 9.17 X-Linked SCID
- 9.17.1 Frequency
- 9.17.2 Genetic Basis
- 9.17.3 Pattern of Inheritance
- 9.17.4 Gene Therapy
- 9.18 Chronic Granulomatous Disease (CGD)
- 9.18.1 Prevalence of CGD
- 9.18.3 Pattern of Inheritance
- 9.18.4 Current Treatments for CGD
- 9.18.5 Gene Therapy for CGD
- 9.19 Wischott Aldrich syndrome (WAS)
- 9.19.1 Frequency
- 9.19.2 Genetic Basis
- 9.19.3 Pattern of Inheritance
- 9.20 Hemophilia
- 9.20.1 Frequency of Hemophilia
- 9.20.2 Genetic Basis
- 9.20.3 Inheritance of Hemophilia
- 9.20.4 Currently Available Treatments for Hemophilia
- 9.20.5 Gene Therapy for Hemophilia
- 9.21 Sickle Cell Anemia
- 9.21.1 Frequency of Sickle Cell Anemia
- 9.21.2 Genetic Basis of Sickle Cell Anemia
- 9.21.3 Inheritance of Sickle Cell Anemia
- 9.21.4 Current Treatment for Sickle Cell Anemia
- 9.21.5 Gene Therapy for Sickle Cell Anemia
- 9.22 Beta Thalassemia
- 9.22.1 Frequency
- 9.22.2 Genetic Basis
- 9.22.3 Pattern of Inheritance
- 9.22.4 Gene Therapy
- 9.23.1 Frequency
- 9.23.2 Genetic Basis
- 9.23.4 Gene Therapy
- 9.25 Limb Girdle Muscle Dystrophy 2C/2D
- 9.25.1 Frequency
- 9.25.2 Genetic Basis
- 9.25.3 Pattern of Inheritance
- 9.25.4 Gene Therapy
- 9.26 Duchenne and Becker Muscular Dystrophy
- 9.26.1 Frequency
- 9.26.2 Genetic Basis
- 9.26.3 Pattern of Inheritance
- 9.27 Human Immunodeficiency Virus (HIV)
- 9.27.1 Currently Available Treatments
- 9.28 Epidermolysis Bullosa
- 9.28.1 Frequency
- 9.28.2 Genetic Basis
- 9.28.3 Pattern of Inheritance
- 9.28.4 Gene Therapy
- 9.29 Leukemia
- 9.29.1 Currently Available Treatments
- 9.29.2 Genetic Basis of Leukemia
- 9.29.3 Gene Therapy Strategies for Leukemia
- 9.30 Ovarian Cancer
- 9.30.1 Frequency of Ovarian Cancer
- 9.30.2 Genetic Basis of Ovarian Cancer
- 9.30.3 Currently Available Treatments for Ovarian Cancer
- 9.30.4 Gene Therapy for Ovarian Cancer
- 9.31 Pancreatic Cancer
- 9.31.1 Frequency of Pancreatic Cancer
- 9.31.2 Currently Available Treatments for Pancreatic Cancer
- 9.31.3 Genetic Basis
- 9.31.4 Gene Therapy for Pancreatic Cancer
- 9.32 Head and Neck Cancers
- 9.32.1 Frequency of Head and Neck Cancers
- 9.32.2 Currently Available Treatments for Head and Neck Cancers
- 9.32.3 Gene Therapy for Head and Neck Cancers
- 9.33 Melanoma
- 9.33.1 Frequency of Melanoma
- 9.33.2 Genetic Basis of Melanoma
- 9.33.3 Pattern of Inheritance
- 9.33.4 Available Treatments for Melanoma
- 9.33.5 Gene Therapy for Melanoma
- 9.34 Prostate Cancer
- 9.34.1 Frequency of Prostate Cancer
- 9.34.2 Genetic Basis of Prostate Cancer
- 9.34.3 Pattern of Inheritance
- 9.34.4 Currently Available Treatments for Prostate Cancer
- 9.34.5 Gene Therapy for Prostate Cancer
- 9.35 Breast Cancer
- 9.35.1 Frequency of Breast Cancer
- 9.35.2 Genetic Basis of Breast Cancer
- 9.35.3 Pattern of Inheritance
- 9.35.4 Currently Available Treatments for Breast Cancer
- 9.35.5 Gene Therapy
- 9.36 Fabry Disease
- 9.36.1 Frequency of Fabry Disease
- 9.36.2 Genetic Basis of Fabry Disease
- 9.36.3 Pattern of Inheritance
- 9.36.4 Currently Available Treatments for Fabry Disease
- 9.36.5 Gene Therapy for Fabry Disease
- 9.37 Hypercholesterolemia
- 9.37.1 Frequency of Hypercholesterolemia
- 9.37.2 Genetic Basis of Hypercholesterolemia
- 9.37.3 Current Medications for Hypercholesterolemia
- 9.37.4 Gene Therapy for Familial Hypercholesterolemia
- 9.38 Huntington Disease
- 9.38.1 Frequency of Huntington Disease
- 9.38.2 Genetic Basis of Huntington Disease
- 9.38.3 Inheritance of Huntington Disease
- 9.38.4 Current Treatment for Huntington Disease
- 9.38.5 Gene Therapy for Huntington Disease
- 9.39 Tay-Sachs Disease
- 9.39.1 Frequency of Tay-Sachs Disease
- 9.39.2 Genetic Basis of Tay-Sachs Disease
- 9.39.3 Pattern of Inheritance
- 9.39.4 Currently Available Treatments
- 9.39.5 Gene Therapy for Tay-Sachs Disease
- 10.0 Market Analysis
- 10.1 Global Market for Gene Therapy by Market Segment
- 10.2 Global Gene Therapy Market by Geography
- 10.3 Commercialization of Gene Therapy in China
- 10.4 Commercialization of Gene Therapy in the Philippines
- 10.5 Commercialization of Gene Therapy in Russia
- 10.6 Commercialization of Gene Therapy in Europe
- 10.6.1 Gene Therapy in France
- 10.6.3 Gene Therapy Efforts in Germany
- 10.6.4 Australia’s Participation in Gene Therapy
- 10.6.5 New Regulatory Framework in Japan
- 10.7 Gene Therapy in the U.S.
- 10.7.1 Likely FDA Approval for Leukemia Treatment
- 10.8 Commercialization of Gene Therapy in South Korea
- 10.9 Additional Addresseable Markets for Gene Therapy
- 10.10 Challenges for Existing and Prospective Players
- 10.10.1 Challenges in Valuation
- 10.10.2 Challenges in Reimbursement
- 10.10.3 Challenges in Commercialization
- 10.11 Future Outlook for Gene Therapy
- 10.12 Potential Market for Gene Therapy Product Candidates
- 10.12.1 CardioNovo (Generx)
- 10.12.2 Collategene
- 10.12.3 Lentiglobin BB305
- 10.12.4 VM-202
- 10.12.5 Invossa
- 10.12.5 GS010
- 10.12.7 ADA Lentivirus
- 10.12.8 Lenti-D
- 11.0 Manufacturing of Viral Vectors and Logistics
- 11.1 Major Manufacturing Companies of Viral Vectors
- 11.2 Major Diseases Targeted by AAV Vectors in Clinical Trials
- 11.3 Major Companies Developing Lentiviral Vectors
- 11.4 Manufacturing Process
- 11.5 Contract Manufacturing
- 11.6 Targeted Delivery of Therapeutic Genes
- 11.7 Logistics Strategies for Gene Therapies
- 11.7.1 Threat to Gene Therapeutics during Transit
- 11.7.2 Impact of Varying Environmental Events on Cell and Gene Therapy Products
- 11.7.3 Pharmaceutical Cold Chain Logistics
- 11.6.4 Clinical Logistics
- 11.7 Cost of Clinical Trials for Biopharmaceuticals including Gene Therapy
- 11.7.1 Clinical Trial Expenditure by Disease
- 12.0 Company Profiles
- 12.1 4d Molecular Therapeutics LLC
- 12.1.1 AAV Vectors
- 12.1.2 4D’s Partnership with Pfizer
- 12.1.3 4D’s Partnership with uniQure
- 12.1.4 4D’s Partnership with Roche
- 12.1.5 4D’s Partnership with AGTC
- 12.1.6 4D’s Partnership with Benitec
- 11.1.7 4D’s Product Pipeline
- 12.2 Abeona Therapeutics LLC
- 12.2.1 Abeona’s Clinical Trial Programs
- 12.2.1.1 ABO-102 Phase I/II Clinical Trial
- 12.2.1.2 ABO-101 Phase I/II Clinical Trial
- 12.3 Advanced Cell & Gene Therapy LLC
- 12.3.1 Consulting Services
- 12.4 Advantagene Inc
- 12.4.1 Gene Mediated Cytotoxic Immunotherapy (GMCI)
- 12.5 Adverum Biotechnologies Inc.
- 12.6 Addgene Inc.
- 12.6.1 Viral Service
- 12.7 Agilis Biotherapeutics LLC
- 12.7.1 Agilis Engineered DNA Therapeutics
- 12.7.2 DNA Therapeutics for AADC Deficiency
- 12.7.3 DNA Therapeutics for Friedreich’s ataxia (FA)
- 12.7.4 DNA Therapeutics for Angelman’s Syndrome
- 12.8 Angionetics Inc.
- 12.8.1 Technology
- 12.8.2 Generx Therapeutic Positioning
- 12.8.3 Product Pipeline
- 12.8.4 Addresseable Market
- 12.9 Applied Genetic Technologies Corporation (AGTC)
- 12.9.1 AGTC’s Technology
- 12.10 AnGes MG Inc.
- 12.10.1 HGF Plasmid
- 12.10.2 NF-kB Dekoy Oligonucleotide
- 12.10.3 DNA Therapeutic Vaccines
- 12.10.4 AnGes’ Alliance Partners
- 12.11 Asklepios BioPharmaceutical Inc.
- 12.11.1 Asklepios’ Collaborators
- 12.12 Audentes Therapeutics Inc.
- 12.12.1 Audentes’ Technology
- 12.13 AveXis Inc.
- 12.13.1 AVXS-101
- 12.14 AvroBio Inc
- 12.14.1 Lentiviral Vectors for Rare Diseases
- 12.14.2 Cytokine IL-12 for Cancer Immunotherapy
- 12.14.3 Gene Vector
- 12.14.4 AvroBio’s Programs
- 12.15 Benitec Biopharma
- 12.15.1 ddRNAi Technology
- 12.15.2 Benitec’s In-House Programs
- 12.15.3 Benitecs Licensed Programs
- 12.16 BioCancell Therapeutic Inc.
- 12.16.1 The H19 Gene
- 12.16.2 BC-819
- 12.16.3 BC-821
- 12.17 BioMarin Pharmaceutical Inc.
- 12.17.1 BMN 270
- 12.18 Bluebird bio Inc.
- 12.18.1 Bluebird’s Program Pipeline
- 12.18.2 Lenti-D
- 12.18.3 LentiGlobin
- 12.18.4 Bluebird’s Partnership with Celgene
- 12.18.5 Bluebird’s Partnership with FivePrime
- 12.18.6 Bluebird’s Partnership with Kite Pharma
- 12.18.7 Bluebird’s Partnership with ViroMed
- 12.19 Brammer Bio LLC
- 12.19.1 Cell Therapy Services
- 12.19.2 Ex Vivo Gene Therapy
- 12.19.3 Viral Vector Manufacturing
- 12.20 Cellectis S.A.
- 12.20.1 UCART19
- 12.20.2 UCART123
- 12.20.3 UCART38 & UCARTCS1
- 12.20.4 UCART22
- 12.21 Clontech Laboratories Inc.
- 12.22 Cobra Biologics Ltd.
- 12.22.1 DNA Services
- 12.22.2 Virus Services
- 12.22.3 Protein Services
- 12.22.4 Microbiota Services
- 12.22.5 Fill and Finish Services
- 12.23 Copernicus Therapeutics Inc.
- 12.23.1 Technology
- 12.23.2 Cystic Fibrosis (CF) Program
- 12.23.3 Retinitis Pigmentosa (RP) Program
- 12.23.4 Parkinson’s Disease (PD) Program
- 12.24 Dimension Therapeutics Inc.
- 12.24.1 Dimension’s Gene Therapy Programs
- 12.25.1 SB Therapeutics
- 12.26 Editas Medicine Inc.
- 12.26.1 CRISPR/Cas9 & TALENs
- 12.27 Fibrocell Sciences Inc.
- 12.27.1 Fibroblast’s Pipeline
- 12.27.2 FCX-007
- 12.27.3 FCX-013
- 12.28 Florida Biologix
- 12.28.1 Services
- 12.29 Freeline Therapeutics Ltd.
- 12.29.1 Freeline’s Platform
- 12.30 Genable Technologies Ltd.
- 12.30.1 RhoNova
- 12.31 Genethon
- 12.31.1 Product Pipeline
- 12.32 Genlantis
- 12.32.1 DNA Transfection
- 12.32.2 siRNA Transfection
- 12.32.3 Neuronal Transfection
- 12.32.4 Customer Services
- 12.32.5 Protein Delivery/Transfection
- 12.32.6 siRNA Generation Kits
- 12.32.7 siRNA Transfection
- 12.32.8 Dicer Enzyme Kits
- 12.33 GenSight Biologics S.A.
- 12.33.1 GS010
- 12.33.2 GS030
- 12.34.1 AdenoVerse Technology
- 12.34.2 Antigen Discovery
- 12.34.3 Cell Lines
- 12.34.4 GenVec’s Product Pipeline
- 12.34.4.1 GGF166 for Hearing Loss
- 12.34.4.2 GV2311 – RSV Vaccine
- 12.34.4.3 GV2207 – HSV-2 Immunotherapeutic
- 12.35 Ichor Medical Systems Inc.
- 12.35.1 Technology
- 12.36 Immune Design Corp.
- 12.36.2 GLASS
- 12.37 Immusoft Corp.
- 12.37.1 Gene Delivery Technology
- 12.37.2 Key Indications being Addressed
- 12.38 Inovio Pharmaceuticals Inc.
- 12.38.1 Inovio’s Technology
- 12.39 Intellia Therapeutics Inc.
- 12.39.1 CRISPR/CAS9
- 12.39.2 Intellia’s Programs
- 12.40 Juventa Therapeutics Inc.
- 12.40.1 Non-Viral Gene Therapy
- 12.40.2 JVS-100
- 12.41 Kite Pharma Inc.
- 12.41.1 Chimeric Antigen Receptor (CAR)
- 12.41.2 T cell Receptor (TCR)
- 12.41.3 Cancer Programs
- 12.41.4 Kite Pharma’s Partnership with National Cancer Institute (NCI)
- 12.41.5 Kite Pharma’s Partnership with The Netherlands Cancer Institute (NKI)
- 12.41.6 Kite Pharma’s Partnership with Adimab
- 12.41.7 Kite Pharma’s Collaboration with Alpine Immune Sciences (AIS)
- 12.41.8 Kite Pharma’s Collaboration with Amgen
- 12.41.9 Kite Pharma’s Partnership with Bluebird Bio
- 12.41.10 Kite Pharma’s Partnership with Cell Design Labs
- 12.41.11 Kite Pharma’s Collaboration with Genentech
- 11.41.12 Kite Pharma’s Partnership with GE Global Research
- 12.41.13 Kite Pharma’s Partnership with Leiden University Medical Center
- 12.41.14 Kite Pharma’s Partnership with Leukemia & Lymphoma Society (LLS)
- 12.41.15 Kite Pharma’s Partnership with The Tel-Aviv Sourasky Medical Center
- 12.41.16 Kite Pharma’s Partnership with The UCLA David Geffen School of Medicine
- 12.42 Kolon Life Sciences Inc.
- 12.42.1 Invossa
- 12.42.2 KLS-1010
- 12.42.3 KLS-2020
- 12.42.4 KLS3020
- 12.43 Lentigen Technology Inc.
- 12.43.1 Lentiviral Vectors for Translational Research
- 12.43.2 Lenti-Viral Vector Technology
- 12.43.3 Clinical Trial Support
- 12.44 Lysogene S.A.S.
- 12.44.1 Lysogene’s rAAV Vectors
- 12.44.2 CNS Administration
- 12.44.3 MPS IIIA
- 12.44.4 GMI Gangliosidosis
- 12.44.5 Partnership with Alcyone Lifesciences Inc.
- 12.44.6 Partnership with University of Massachusetts
- 12.44.7 Partnership with Auburn University
- 12.44.8 Partnership with University of Manchester
- 12.45 Medgenics Inc.
- 12.45.1 Transduced Autologous Restorative Gene Therapy (TARGT)
- 12.46 Mirus Bio LLC
- 12.46.1 TransIT – Lenti Transfection Reagent
- 12.46.2 Ingenio Electroporation Kits
- 12.47 Mologen AG
- 12.47.1 Technologies
- 12.47.1.1 dSLIM
- 12.47.1.2 EnanDIM
- 12.47.1.3 MIDGE
- 12.47.2 Allogeneic Tumor Cell Bank
- 12.48 NanoCor Therapeutics Inc.
- 12.48.1 Biological Nanoparticle (BNP) Technology
- 12.48.2 Carfostin
- 12.49 Nature Technology Corp. (NTC)
- 12.49.1 Vector Cell Lines
- 12.49.2 DNA Manufacturing
- 12.49.3 Cloning & QC
- 12.49.4 Protein Products
- 12.49.5 Technology & Consulting
- 12.50 NightstaRx Ltd.
- 12.50.1 Project AAV2 REP1
- 12.51 Novasep Process SAS
- 12.51.1 Manufacturing Services for Customers
- 12.51.2 Contract Manufacturing Services
- 12.51.3 Purification Technologies
- 12.52 Omnia Biologics Inc.
- 12.52.1 Services
- 12.52.1.1 Preclinical and GMP Manufacturing
- 12.52.1.2 Process Development
- 12.52.1.3 Cell and Viral Banking
- 12.52.1.4 Aseptic Filling
- 12.53 ORCA Therapeutics B.V.
- 12.53.1 Technology
- 12.53.2 ORCA-010
- 12.53.3 Oncolytic Viruses Expressing p35
- 12.53.4 RNA Interference
- 12.54 OrphageniX
- 12.54.1 Technology
- 12.55 Oxford BioMedica plc
- 12.55.1 LentiVector Gene Delivery Technology
- 12.55.2 OXB-102
- 12.55.3 OXB-201 (RetinoStat)
- 12.55.4 CAR-T Cell Therapy
- 12.55.5 OXB-301 (TroVax)
- 12.55.6 SAR 422459
- 12.55.7 SAR421869
- 12.55.8 Partnership with Sanofi
- 12.55.9 Partnership with GlaxoSmithKline
- 12.55.10 Partnership with Novartis
- 12.55.11 Partnership with Immune Design Corp.
- 12.56 Oxford Genetics Ltd.
- 12.56.1 DNA Services
- 12.56.1.1 High Throughput Cloning Services
- 12.56.1.2 DNA Design and Protein Optimization Services
- 12.56.2 Cell Line Services
- 12.56.2.1 Cell Line Development
- 12.56.2.2 Custom Cell Engineering
- 12.56.3 Protein and Virus Services
- 12.56.3.1 Protein Expression and Antibody Engineering
- 12.56.3.2 Virus Construction and Production
- 12.56.4 Standard DNA Services
- 12.56.4.1 DNA Synthesis
- 12.56.4.2 Custom Cloning
- 12.56.4.3 Plasmid Preparation
- 12.57 REGENXBIO Inc.
- 12.57.1 NAV Technology
- 12.57.2 RGX-501
- 12.57.3 RGX-314
- 12.57.4 RGX-111
- 12.57.5 RGX-121
- 12.57.6 RGX-321
- 12.58 Renova Therapeutics Inc.
- 12.58.1 Renova’s Pipeline
- 12.59 RetroSense Therapeutics LLC
- 12.59.1 RST-001
- 12.60 Sangamo Biosciences Inc.
- 12.60.1 Sangamo’s Technology
- 12.60.2 Sangamo’s Product Pipeline
- 12.60.3 Sangamo’s Research Collaborations
- 12.61 Sarepta Therapeutics Inc
- 12.61.1 RNA Medicine
- 12.61.2 RNA Modulation by PMO
- 12.61.3 Sarepta’s Programs for Duchenne Muscular Dystrophy (DMD)
- 12.61.4 Sarepta’s Programs for Infectious Diseases
- 12.62 Shanghai Sunway Biotech Co. Ltd.
- 12.62.1 Oncorine
- 12.63 SiBiono GeneTech Co. Ltd.
- 12.63.1 Gendicine
- 12.64 Sirion Biotech GmbH
- 12.64.1 Adenovirus
- 12.64.2 Lentivirus
- 12.64.4 Adeno-Associated Virus
- 12.64.5 AdenoBOOST & LentiBOOST
- 12.64.6 AdenONE
- 12.65 Spark Therapeutics Inc.
- 12.65.1 Spark Therapeutics’ Pipeline
- 12.66 Takara Bio Inc.
- 12.66.1 Takara’s Gene Therapy
- 12.66.1.1 Oncolytic Virus HF10
- 12.66.1.2 Engineered T-Cell Therapy (siTCR Gene Therapy)
- 12.66.1.3 CAR Gene Therapy
- 12.66.1.4 MazF Gene Therapy
- 12.67 Taxus Cardium Pharmaceutical Group Inc.
- 12.67.1 Generx
- 12.68 Tocagen Inc.
- 12.68.1 Technology
- 12.68.2 Toca 511 & Toca FC
- 12.69 ToolGen Inc.
- 12.69.1 Gene Editing
- 12.69.2 RNA-Guided Endonucleases (RGEN)
- 12.70 Transgene SA
- 12.70.1 TG4010
- 12.70.2 Pexa-Vec
- 12.70.3 TG6002
- 12.70.4 TG1050
- 12.70.5 TG4001
- 12.70.6 Immunotherapy against Tuberculosis
- 12.71 uniQure N.V.
- 12.71.1 Glybera
- 12.72 Vascular Biologics Ltd.
- 12.72.1 VBL’s Cancer Platform
- 12.73 Vical Inc.
- 12.73.1 Poloxamer Delivery System
- 12.73.2 ASP0113
- 12.73.3 HSV-2 Therapeutic Vaccine
- 12.73.4 CyMVectin Prophylactic CMV Vaccine
- 12.73.5 VL-2397 Antifungal
- 12.74 ViroMed Co., Ltd.
- 12.74.1 VM202
- 12.74.2 Fast Track Designation for VM202
- 12.74.3 VM501
- 12.75 Vivebiotech SL
- 12.75.1 GMP Solutions
- 12.75.2 Viral Vector Services
- 12.75.3 ZELIGEN Technology
- 12.76 Voyager Therapeutics Inc.
- 12.76.1 Product Pipeline
- 12.77 Xenon Pharmaceuticals Inc.
- 12.77.1 Glybera
- INDEX OF FIGURES
- Figure 2.1: Shematic of Gene Therapy using a Virus Vector
- Figure 2.2: Schematic to Explain Gene Augmentation Therapy
- Figure 2.3: Schematic to Exlain Gene Inhibition Therapy
- Figure 2.4: Schematic to Explain Suicide Gene Therapy
- Figure 2.5: Ex vivo Route of Gene Delivery in Gene Therapy
- Figure 2.6: In Vivo Route of Gene Delivery in Gene Therapy
- Figure 7.1: Academic Mettle: Number of Research Papers Published, 1975-2016
- Figure 7.2: Number of Gene Therapy Clinical Trials Approved Worldwide, 1989-2016
- Figure 7.3: Geographical Distribution of Gene Therapy Clinical Trials, 2016
- Figure 7.4: Major Countries Participating in Gene Therapy Clinical Trials, 2016
- Figure 7.5: Major Indications Addressed by Gene Therapy Clinical Trials, 2016
- Figure 7.6: Top Gene Types Transferred
- Figure 7.7: Phases of Gene Therapy Clinical Trials
- Figure 7.8: Venture Investment in Gene Therapy by Body System as of 2014
- Figure 8.1: Top Five Vectors used in Gene Therapy
- Figure 9.1: Global Market for Cystic Fibrosis Therapeutics, 20162022
- Figure 9.2: Global Market for Hemophilia Factor Proteins, 2016-2022
- Figure 9.3: Global Market for Sickle Cell Anemia, 2016-2022
- Figure 9.4: Global Market for HIV Therapeutics, 2016-2022
- Figure 9.5: Global Market for Leukemia Drugs, 2016-2022
- Figure 9.6: Global Market for Ovarian Cancer Therapeutics, 2016-2022
- Figure 9.7: Global Market for Pancreatc Cancer Therapeutics, 2016-2022
- Figure 9.8: Global Market for Head & Neck Cancer Therapeutics, 2016-2022
- Figure 9.9: Global Market for Melanoma Therapeutics, 2016-2022
- Figure 9.10: Global Market for Prostate Therapeutics, 2016-2022
- Figure 9.11: Global Market for Breast Cancer Therapeutics, 2016-2022
- Figure 9.12: Global Market for Cholesterol Lowering Drugs, 2016-2022
- Figure 9.13: U.S. Market for HD Therapeutics, 2016-2022
- Figure 10.1: Global Market for Gene Therapy Technologies, Services and Products, 2016-2022
- Figure 10.2: Market for Technologies, Services and Products by indication, 2016-2022
- Figure 10.3: Percent Share of Gene Therapy Market by Indication, 2016
- Figure 10.5: Percent Share of Gene Therapy market by Geography, 2016
- Figure 11.1: Major Ten Companies Developing AAV Vector Candidates
- Figure 11.2: Diseases and Corresponding Number of Drugs Studied using AAV Vectors
- Figure 11.3: Major Ten Companies Developing Lentiviral Vector Candidates
- Figure 11.4: Overview of a Typical Viral Vector Manufacturing Process
- Figure 11.5: Global Cold Chain and Non-Cold Chain Logistics Spending, 2016-2022
- Figure 11.6: Global Spending on Logistics Related to Clinical Trials, 2016-2022
- Figure 11.7: Estimated Average Per-Patient Clinical Trial Costs by Phase of Study
- Figure 11.8: Estimated Per-Patient Clinical Trial Expenditure by Indication
- INDEX OF TABLES
- Table 2.1: Differences between Somatic Gene Therapy and Germline Gene Therapy
- Table 2.2: Few Examples of Suicide Gene Products
- Table 2.3: Genetic Diseases Treatable by Ex Vivo Gene Therapyusing Bone Marrow Cells
- Table 2.4: Examples of Diseases Treatable by In Vivo Route of Gene Delivery
- Table 2.5: Important Gene Therapy Players and Product Candidates
- Table 3.1: Approved Gene Therapy Products
- Table 4.1: Number of Non-Cancer Phase III Gene Therapy Candidates as of 2016
- Table 5.1: Key Players with Phase II Product Candidates
- Table 6.1: Commercialization Status of Gene Therapies in E.U. Member Countries
- Table 6.2: Prices for Gene Therapy
- Table 7.1: Approved and Published Clinical Gene Therapy Protocols
- Table 7.2: Number of Gene Therapy Clinical Trials Approved Worldwide, 1989-2016
- Table 7.3: Geographical Distribution of Gene Therapy Clinical Trials, 2016
- Table 7.4: Gene Therapy Clinical Trials by Country, 2016
- Table 7.5: Gene Therapy Clinical Trials by Indications, 2016
- Table 7.6: Gene Types Transferred in Gene Therapy Clinical Trials
- Table 7.7: Phases of Gene Therapy Clinical Trials
- Table 7.8: Timeline of Big Pharma Investment and Licensing in Gene Therapy, 2010-2016
- Table 7.9: Funds Raised by Gene Therapy Companies Since 2013
- Table 8.1: Most Utilized Non-Viral Vectors in Gene Therapy
- Table 8.2: Advantages and Disadvantages of Four Physical Methods in Gene Delivery
- Table 8.3: Major Types of Viral Vectors used in Gene Therapy, Advantages and Disadvantages
- Table 8.4: Key Properties of Viral Vectors
- Table 8.5: Advantages and Disadvantages of Viral Vectors by Type
- Table 8.6: Salient Features of Adenovirus Vectors
- Table 8.7: Advantages and Disadvantages of Adenovirus Vectors
- Table 8.8: Salient Features of Retrovirus
- Table 8.9: Advantages and Disadvantages of Retroviral Vectors
- Table 8.10: Comparison of Retrovirus and Adenovirus
- Table 8.11: Salient Features of Adeno-Associated Virus Vectors
- Table 8.12: Advantages and Disadvantages of Adeno-Associated Vectors
- Table 8.13: AAV Serotypes
- Table 8.14: Genes Transduced by AAV for Specific Diseases
- Table 8.15: Salent Features of Lentivirus Vectors
- Table 8.16: Advantages and Disadvantages of Lentiviral Vectors
- Table 8.18: Advantages and Disadvantages of Pox/Vaccinia Vectors
- Table 8.19: Major Features of Viral Vectors by Type
- Table 8.20: Examples of Clinical Trials Using Retroviruses & Lentiviruses
- Table 8.21: Examples of Clinical Trials Using Retroviruses & Lentiviruses
- Table 8.22: Vectors Used in Gene Therapy Clinical Trials, 2016
- Table 8.23: Examples of Virus Vector Manufacturing Systems
- Table 9.1: Clinical Applications of Gene Therapy
- Table 9.2: Identifying Number, Intervention and Status of Gene Therapy Trials for Blindness 105
- Table 9.3: Gene Therapy Clinical Trial for Eye Disorders
- Table 9.4: Genes and Loci Underlying Parkinson Disease
- Table 9.5: Gene Therapy Clinical Trials for Parkinson Disease
- Table 9.6: U.S. Data on Cystic Fibrosis, 2000-2015
- Table 9.7: Gene Therapy Clinical Trials for Cystic Fibrosis
- Table 9.8: Gene Therapy Clinical Trials for Pompe Disease
- Table 9.9: Gene Therapy Clinical Trials for Batten Disease
- Table 9.10: Gene Therapy Clinical Trials for Immune Disorders
- Table 9.12: Gene Therapy Clinical Trials for Wiskott-Aldrich Syndrome
- Table 9.13: Gene Therapy Clinical Trials for Hemophilia
- Table 9.14: Gene Therapy Product Candidates for Hemophilia A and B
- Table 9.15: Ongoing Gene Therapy Clinical Trials for Sickle Cell, Fanconi and Thalassemia
- Table 9.16: Gene Therapy Clinical Trials for Heart Diseases
- Table 9.17: Gene Therapy for Muscular Dystrophies
- Table 9.18: Gene Therapy Clinical Trials for HIV
- Table 9.19: Gene Therapy Clinical Trials for Blood Cancer
- Table 9.20: Gene Therapy Clinical Trials for Ovarian Cancer
- Table 9.21: Gene Therapy Clinical Trials for Pancreatic Cancer
- Table 9.22: Gene Therapy Clinical Trials for Head and Neck Cancers
- Table 9.23: Gene Therapy Clinical Therapy for Melanoma
- Table 9.24: Gene Therapy Clinical for Prostate Cancer
- Table 9.25: Gene Therapy Clinical Trials for Breast Cancer
- Table 9.26: Gene Therapy Clinical Trials for Fabry Disease
- Table 9.27: Gene Therapy for Familial Hypercholesterolemia
- Table 9.28: Gene Therapy Clinical Trials for Tay-Sachs disease
- Table 10.1: Estimated Market for Five Gene Therapy Products in the U.S. in the Next 20 Years
- Table 10.2: Market for Technologies, Services and Products by Indication, 2016-2022
- Table 10.3: Global Market for Gene Therapy by Market Segment, 2016-2022
- Table 10.4: Percent Share of Gene Therapy market by Geography, 2016
- Table 10.5: The Estimated Drug Market that is Wide Open for Gene Therapy Product Candidates Focusing on 13 Diseases
- Table 11.1: Major Ten Companies Developing AAV Vectors
- Table 11.2: Diseases and Corresponding Number of Drugs Studied using AAV Vectors
- Table 11.3: Major Ten Companies Developing Lentiviral Vector Candidates
- Table 11.4: Global Cold Chain and Non-Cold Chain Logistics Spending, 2016-2022
- Table 11.5: Estimated Average Per-Patient Clinical Trial Costs by Phase of Study
- Table 11.6: Estimated Per-Patient Clinical Trial Expenditure by Indication
- Table 11.7: Patient-Per Clinical Trial Expenditure by Disease Area and Phase
- Table 12.1: 4D’s Product Pipeline
- Table 12.2: Abeona’s Product Pipeline
- Table 12.3: Advantagene’s Program Overview
- Table 12.4: Adverum’s Pipeline of Gene Therapy Programs
- Table 12.5: Agilis DNA Therapeutic Programs
- Table 12.6: Generx Product Pipeline
- Table 12.7: Potential Economic Opportunity for Generx (Ad5FGF-4)
- Table 12.8: AGTC’s Ophthalmology Development Programs
- Table 12.9: AnGes’ Product Pipeline
- Table 12.10: Asklepios’ Product Pipeline
- Table 12.11: Audentes’ Pipeline
- Table 12.12: AvroBio’s Programs
- Table 12.13: Benitec’s In-House Programs
- Table 12.14: Benitecs Licensed Programs
- Table 12.15: Bluebird’s Program Pipeline
- Table 12.16: Product Pipeline from Cellectis
- Table 12.17: Dimension’s Product Pipeline
- Table 12.18: Fibroblast’s Product Pipeline
- Table 12.19: Genethon’s Pipeline
- Table 12.20: Summary of GenVec’s Product Pipeline
- Table 12.21: Immune Design’s Product Pipeline
- Table 12.22: Inovio’s Pipeline of Products
- Table 12.23: Types of Genome Editing
- Table 12.24: Intellia’s Product Pipeline
- Table 12.25: Kite’s Product Pipeline
- Table 12.26: Lysogene’s Product Pipeline
- Table 12.27: Medgenics’ Product Pipeline
- Table 12.28: Mologen’s Product Pipeline
- Table 12.29: Oxford BioMedica’s Product Pipeline
- Table 12.30: REGENXBIO’s Therapeutic Programs
- Table 12.31: Renova’s Pipeline
- Table 12.32: Sangamo’s Product Pipeline
- Table 12.33: Sarepta’s Product Pipeline for DMD
- Table 12.34: Sarepta’s Programs for Infectious Diseases
- Table 12.35: Spark Therapeutics’ Pipeline
- Table 12.36: Tocagen’s Product Pipeline
- Table 12.37: Transgene’s Product Pipeline
- Table 12.38: unQure’s Pipeline
- Table 12.39: VBL’s Cancer Pipeline
- Table 12.40: Vical’s Product Pipeline
- Table 12.41: Voyager Therapeutics, Product Pipeline
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