Global Genetic Modification Therapies Clinical Market Report 2018-2023: Focus on Gene Therapies, Genetically Modified Cell Therapies, RNA Therapies and Gene Editing

DUBLIN, Sept. 27, 2018 /PRNewswire/ --

The "Genetic Modification Therapies Clinical Applications: Gene Therapies, Genetically Modified Cell Therapies, RNA Therapies and Gene Editing" report has been added to's offering.

The study scope includes clinical therapies using gene editing, gene therapy, genetically modified cell therapy (chimeric antigen receptor T-cells, or CAR T-cells) or RNA therapy platforms. Delivery technologies covered include AAV, adenovirus, lentivirus, retrovirus, other viral and nonviral. Disease areas covered include cardiology, oncology, ophthalmology, hematology, musculoskeletal, neurology, rare diseases and others. Therapies that are already commercialized or are forecast to be commercialized with the next five years are included.

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.

Key Topics Covered:

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
    --  Analyst's Credentials
    --  Custom Research
    --  Related 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
    --  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

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