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Genetic Modification Therapies Clinical Applications: Gene Therapies, Genetically Modified Cell Therapies, RNA Therapies and Gene Editing

  • Published Date: 31 Aug 2018
  • Number of Pages: 211
  • Category: Technology
  • Country: Global

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Summary

Genetic modification therapies are changing the landscape of medicine due to their unprecedented ability to offer a one-time cure for severe, debilitating diseases that were previously untreatable. Genetic modification therapies are advanced therapies that use genetic material (i.e., DNA or RNA) to treat diseases.

The four main genetic modification therapy platform technologies covered in this report include gene therapies, genetically modified cell therapies, RNA therapies and gene editing.

In Dec. 2017, Luxturna became the first gene therapy approved for treating a genetic disorder. Luxturna is indicated for treating a severe inherited retinal disorder caused by a mutation in a single gene. Luxturna meets a clear and pressing medical need of restoring functional vision to a patient.

Besides Luxturna, several genetic modification therapies have been approved, including Kymarih and Yescarta (genetically modified cell therapy), Spinraza (RNA therapy) and others, setting the stage for strong market growth in the next five years.

Reasons for Doing the Study

Genetic modification therapies, particularly gene therapy and RNA therapy, have existed for many years, with little clinical success. However, recent improvements in these therapies, including better delivery systems, more efficient and durable gene expression constructs, precise DNA editing tools, have brought this industry to the forefront, and it is now poised for explosive growth in the coming years.

Because of the potentially curative nature of these medicines there is enormous potential in many applications, ranging from cancer to neurology to rare diseases. Genetic modification therapies represent the next wave of medicines with enormous potential for treating and curing debilitating and serious diseases. Because of its wide scope, genetic modification therapy will play an important role in the future global medical economy.

Continuing advances in key technologies such as DNA editing, viral design and production, and gene expression, as well as a pressing medical need in many serious and debilitating disorders, are driving the growth of the market for genetic modification therapies. Developments in these multidisciplinary fields promise to advance the genetic modification therapies industry and create unique market opportunities. This report analyzes these trends and their impact on the future markets for genetic modification therapy products.

Based on these market and technology dynamics, it is especially timely to comprehensively examine the current and future markets for genetic modification therapies.
Publisher Name : BCC Research
Chapter 1 Introduction
Study Goals and Objectives
Reasons for Doing the Study
Contribution of the Study and for Whom
Scope and Format
Methodology
Information Sources
Geographical Breakdown
Analysts Credentials
BCC Custom Research
Related BCC Research Reports
Chapter 2 Summary and Highlights
Chapter 3 Overview
Scope of Report
Genetic Modification Therapies Covered in Report
Genetic Modification Therapy Platform Technologies
Forces Driving the Growth of the Market for Genetic Modification Therapies
Global Market for Genetic Modification Therapies
Life-Cycle Status of Products and Technologies
Genetic Modification Therapies Industry
Chapter 4 Technology
Introduction to Genetic Technology
Genetic Variation and Analysis
Genetic Modification Therapies
Gene Therapy Technologies
RNA Therapy Technologies
Gene Editing Technologies
Genetically Modified Cell Therapy Technologies
Delivery Technologies
Introduction
Viral Delivery Technologies
Nonviral Delivery Technologies
Design and Manufacture of a Gene Therapy Medicine
Chapter 5 Genetic Modification Therapy Applications
Chapter 6 Genetic Modification Therapies Industry
Summary
Gene Editing Industry
Gene Therapy Industry
Genetically Modified Cell Therapy Industry
RNA Therapy Industry
Lentivirus Production Industry
Chapter 7 Acquisitions and Strategic Alliances
Acquisitions
Strategic Alliances
Chapter 8 Markets for Genetic Modification Therapies
Growth Driving Forces
Payment and Business Models for Genetic Modification Therapeutics
Market for Genetic Modification Therapies
Genetic Modification Therapies by Platform Technology
Genetic Modification Therapies by Disease Category
Genetic Modification Therapies by Delivery Technology
Genetic Modification Therapies by Indication
Genetic Modification Therapies by Region
Chapter 9 Patents
Gene Editing-Related Patents
Gene Editing Patents-Sangamo Case Study
Gene Therapy Delivery Patents
RNA Therapy-Related Patents
CAR T-cell Patents
Chapter 10 Company Profiles
4D MOLECULAR THERAPEUTICS
ABCLON
ABEONA THERAPEUTICS LLC
ACER THERAPEUTICS INC.
ADAPTIMMUNE THERAPEUTICS
ADVANTAGENE INC.
ADVERUM BIOTECHNOLOGIES
AEVI GENOMIC MEDICINE INC.
AGILIS BIOTHERAPEUTICS
ALDEVRON
ALLERGAN PLC
ALLOGENE THERAPEUTICS INC.
AMERICAN GENE TECHNOLOGIES INTERNATIONAL INC.
AMGEN INC.
ANGES INC.
APEIRON BIOLOGICS AG
APPLIED GENETIC TECHNOLOGIES CORP.
ARBOR BIOTECHNOLOGIES INC.
ARCTURUS THERAPEUTICS INC.
ASKLEPIOS BIOPHARMACEUTICAL INC.
ASTELLAS PHARMA INC.
AUDENTES THERAPEUTICS INC.
AVACTA GROUP PLC
AVROBIO INC.
BEAM THERAPEUTICS INC.
BELLICUM PHARMACEUTICALS INC.
BINEX CO. LTD.
BIOCUREPHARM CORP.
BIOGEN INC.
BIOMARIN PHARMACEUTICAL INC.
BLUEBIRD BIO
B-MOGEN BIOTECHNOLOGIES INC.
BRAMMER BIO
CARIBOU BIOSCIENCES
CASEBIA THERAPEUTICS
CATHERICS PTY LTD.
CELGENE CORP.
CELLECTIS SA
CELL MEDICA LTD.
CELLULAR BIOMEDICINE GROUP
CELYAD
COGNATE BIOSERVICES
COMBIGENE AB
CRISPR THERAPEUTICS AG
CSL BEHRING LLC
CUROCELL CO. LTD.
EDITAS MEDICINE INC.
ENCODED GENOMICS INC.
ERRANT GENE THERAPEUTICS LLC
ESTEVE
ETAGEN PHARMA
EUTILEX CO. LTD.
FATE THERAPEUTICS INC.
FERRING PHARMACEUTICALS SA
FIBROCELL SCIENCE
FKD THERAPIES OY
FORTRESS BIOTECH INC.
FREELINE THERAPEUTICS
GE HEALTHCARE
GENE BIOTHERAPEUTICS INC.
GENENTA SCIENCE SRL
GENERATION BIO
GENETHON SA
GENSIGHT BIOLOGICS SA
GILEAD SCIENCES INC.
GLAXOSMITHKLINE PLC
GLOBAL BIOTHERAPEUTICS USA INC.
GREEN CROSS CELL CORP.
HEMERA BIOSCIENCES INC.
HITACHI CHEMICAL ADVANCED THERAPEUTICS LLC
HOMOLOGY MEDICINES INC.
HORAMA SA
IMMUNOCHINA PHARMACEUTICALS
IMMUNOCORE LTD.
INDEE LABS INC.
INTELLIA THERAPEUTICS INC.
INTREXON CORP.
JIVANA BIOTECHNOLOGY
JOHNSON & JOHNSON
JUVENTAS THERAPEUTICS INC.
KOLON LIFE SCIENCES INC.
KRYSTAL BIOTECH
KUBOTA PHARMACEUTICAL HOLDINGS CO. LTD.
LIMELIGHT BIO INC.
LOGICBIO THERAPEUTICS INC.
LONZA GROUP LTD.
LYSOGENE SA
MARCALA BIOTECH INC.
MAXCYTE INC.
MEDIGENE AG
MEIRA GTX
MERCK & CO.
MERCK KGAA
MESOBLAST LTD.
MILLIPORE SIGMA
MILO BIOTECHNOLOGY LLC
MOLMED SPA
MORPHOGENESIS INC.
MYONEXUS THERAPEUTICS INC.
NANTKWEST INC.
NEXIMMUNE INC.
NEWLINK GENETICS CORP.
NIGHTSTAR THERAPEUTICS PLC
NOVARTIS PHARMA AG
NOVASEP LLC
OCUNEXUS THERAPEUTICS INC.
ORCHARD THERAPEUTICS
OXFORD BIOMEDICA PLC
PEPROMENE BIO INC.
PERIPHAGEN INC.
PFIZER INC.
POSEIDA THERAPEUTICS INC.
PRECISION BIOSCIENCE
REGENXBIO INC.
RENOVA THERAPEUTICS
ROCKET PHARMA LTD.
RXI PHARMACEUTICALS CORP.
SANGAMO THERAPEUTICS INC.
SANOFI SA
SAREPTA THERAPEUTICS INC.
SELECTA BIOSCIENCES INC.
SENTI BIOSCIENCES
SHIRE PLC
SIBIONO GENETECH CO. LTD.
SILLAGEN BIOTHERAPEUTICS
SOLID BIOSCIENCES LLC
SPARK THERAPEUTICS INC.
SYNPROMICS LTD.
TAKARA BIO
THERAGENE PHARMACEUTICALS INC.
THERMO FISHER SCIENTIFIC
TOCAGEN
TOOLGEN
TRANSLATE BIO
TRANSGENE SA
TX CELL
ULTRAGENYX PHARMACEUTICAL INC.
UNIQURE NV
UNUM THERAPEUTICS INC.
VASCULAR BIOGENICS LTD.
VIRACTA THERAPEUTICS
VIROMED CO. LTD.
VIVET THERAPEUTICS
VOYAGER THERAPEUTICS INC.
WUXI APPTECH
ZIOPHARM ONCOLOGY

List Of Tables

Summary Table : Global Market for Genetic Modification Therapies, by Disease Category, Through 2023
Table 1 : Scope of Report
Table 2 : Genetic Modification Therapies Covered in Report
Table 3 : Genetic Modification Platforms
Table 4 : Key Market Growth Driving Forces of Genetic Modification Therapies
Table 5 : Global Market for Genetic Modification Therapies, by Delivery Technology, Through 2023
Table 6 : Genetic Modification Therapy Technology Life-Cycle Stage
Table 7 : Characteristics of the Genetic Modification Therapies Industry
Table 8 : Chemical Building Blocks of DNA and RNA
Table 9 : DNA Translation and Transcription
Table 10 : Long-range DNA Structure
Table 11 : Genetic Variant Types
Table 12 : Therapeutic Intervention Platforms
Table 13 : Genetic Modification Approaches
Table 14 : Distinct Features of the Genetic Modification Approaches
Table 15 : Main Components of Gene Therapy Technology
Table 16 : Gene Therapy Delivery Technology Parameters
Table 17 : Clinical Criteria for Gene Therapy
Table 18 : RNA Therapy Segments
Table 19 : Gene Editing Therapeutic Interventions
Table 20 : Gene Editing Technology Approaches
Table 21 : Comparison of the Major Gene Editing Technologies
Table 22 : Genetically Modified Cell Therapy Technologies
Table 23 : Delivery Vector Technical Hurdles
Table 24 : Comparison of Viral Vector Types
Table 25 : Nonviral Delivery Methods
Table 26 : Steps in Designing and Manufacturing a Gene Therapy Medicine
Table 27 : Near-Term Applications for Genetic Modification Therapies
Table 28 : Characteristics of Liver Organ
Table 29 : Sectors within the Genetic Modification Therapies Industry
Table 30 : Gene Editing Industry
Table 31 : Gene Therapy Companies
Table 32 : Genetically Modified Cell Therapy Clinical Industry
Table 33 : RNA Therapy Companies
Table 34 : GMP Clinical-Grade Lentiviral Manufacturing Companies
Table 35 : Genetic Modification Therapies Industry Acquisitions, Jan. 2016-June 2018
Table 36 : Genetic Modification Therapies Industry Strategic Alliances, Jan. 2016-June 2018
Table 37 : Genetic Modification Therapies Key Market Growth Driving Forces
Table 38 : Global Market for Genetic Modification Therapies, by Platform Technology, Through 2023
Table 39 : Global Market for Gene Editing Therapies, by Disease Category, Through 2023
Table 40 : Global Market for Gene Editing Therapies, by Technology, Through 2023
Table 41 : Global Market for Gene Editing Therapies, by Delivery Type, Through 2023
Table 42 : CRISPR Clinical Pipeline
Table 43 : Global Market for Gene Therapy, by Disease Category, Through 2023
Table 44 : Inherited Retinal Diseases
Table 45 : Global Market for Gene Therapy, by Platform Technology, Through 2023
Table 46 : Global Market for Gene Therapy, by Delivery Technology, Through 2023
Table 47 : Gene Therapy AAV Vector Clinical Landscape
Table 48 : Global Market for Genetically Modified Cell Therapy, by Disease Category, Through 2023
Table 49 : Global Market for Genetically Modified Cell Therapy, by Platform Technology, Through 2023
Table 50 : Global Genetically Modified Cell Therapy Market, by Delivery Technology, Through 2023
Table 51 : Allogeneic versus Autologous Approaches
Table 52 : Allogeneic Clinical and Preclinical Cell Therapy Programs
Table 53 : Global Market for RNA Therapy, by Disease Category, Through 2023
Table 54 : Global Market for RNA Therapy, by Platform Technology, Through 2023
Table 55 : GalNAc-conjugated RNA Therapy Pipeline
Table 56 : Global Market for RNA Therapy, by Delivery Technology, Through 2023
Table 57 : Global Market for Genetic Modification Therapies, by Disease Category, Through 2023
Table 58 : Indications Driving Market Growth
Table 59 : Global Market for Genetic Modification Therapies Market, by Delivery Technology, Through 2023
Table 60 : Global Market for Genetic Modification Medicines: Marketed and Late-Stage Pipeline
Table 61 : Global Market for Genetic Modification Therapies in Cardiology, by Indication, Through 2023
Table 62 : Global Market for Genetic Modification Therapies in Oncology, by Indication, Through 2023
Table 63 : Barriers to CAR T-Cell Solid Tumor Development
Table 64 : CAR T-Cell Solid Tumor Development Status
Table 65 : Global Market for Genetic Modification Therapies in Ophthalmology, by Indication, Through 2023
Table 66 : Global Market for Genetic Modification Therapies in Hematology, by Indication, Through 2023
Table 67 : Hemophilia A and B: Drugs in Development
Table 68 : Global Market for Genetic Modification Therapies in Musculoskeletal Applications, by Indication, Through 2023
Table 69 : Global Market for Genetic Modification Therapies, by Indication, Through 2023
Table 70 : Global Market for Genetic Modification Therapies in Rare Diseases, by Indication, Through 2023
Table 71 : Global Market for Genetic Modification Therapies in Other Applications, by Indication, Through 2023
Table 72 : Global Market for Genetic Modification Therapies, by Region, Through 2023
Table 73 : Global Market for Gene Editing Therapy, by Region, Through 2023
Table 74 : Global Market for Gene Therapy, by Region, Through 2023
Table 75 : Global Market for Genetically Modified Cell Therapy, by Region, Through 2023
Table 76 : Global Market for RNA Therapy, by Region, Through 2023
Table 77 : CRISPR-Cas9 Patent Groups
Table 78 : CRISPR-Cas9 Patent Licensing Fields
Table 79 : Sangamos Collaboration Agreements Relating to ZFN Technology
Table 80 : Sangamo Therapeutics Patent Position
Table 81 : Oxford BioMedicas Key Patents
Table 82 : RNA Therapy Patents and Patent Applications, by Leading Companies
Table 83 : CAR T-cell Patentable Technologies
Table 84 : Affimers versus Antibodies for Gene Therapy Applications
Table 85 : Gene Editing versus Gene Therapy Approach

List Of Figures

Summary Figure : Global Market for Genetic Modification Therapies, by Disease Category, 2017-2023
Figure 1 : Complexity of Therapeutic Approaches
Figure 2 : Main Principle of Molecular Biology

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