In recent past, most of the gene therapies received orphan drug designations. Orphan drugs are generally defined as those medicines with one or more indications approved under the Orphan Drug Act of 1983. The Orphan Drug Act supports the development of innovative treatments for rare disease patients. The creation of the orphan drug designation with the passage of the Orphan Drug Act in 1983 has facilitated the development and approval of drugs for rare diseases and 2017 and 2018, were marked by the highest number of orphan drug and indication approvals to date. Production of gene therapies is associated with use of high-end technologies, high research and development costs, and skilled scientists and researchers, which reflects in high prices of these therapies.
Market Dynamics
Key players in the market are dedicated to the development of gene therapies for the novel indications such as Huntington's disease for which there are no specific approved medication. For instance, uniQure N.V. is developing AMT-130, a novel Gene therapy for Huntington's disease (HD), a rare, fatal, neurodegenerative genetic disorder. AMT-130 is currently in pre-clinical trial and is expected to receive approval in near future. Furthermore, companies are developing novel gene therapies for rare diseases such as Hemophilia B and Hemophilia A, which can offer long term treatment and can replace lifelong medication for this diseases. Launches of such novel therapies in near future is expected to significantly create lucrative opportunity for growth of the market. Moreover, increasing reimbursement support as well as innovative payment options are being adopted by manufacturers such as pay for performance. However, high prices of these therapies and potential challenges in commercialization of these therapies in emerging economies such as Asia Pacific and Latin America may restrain growth of the market
Key features of the study:
• This report provides in-depth analysis of gene therapy for rare disease market and provides market size (US$ Million) and Cumulative Annual Growth Rate (CAGR %) for the forecast period (2017-2025), considering 2016, as the base year
• It elucidates potential revenue opportunity across different segments and explains attractive investment proposition matrix for this market
• This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, regional outlook, and competitive strategy adopted by the leading players
• It profiles leading players in the global Gene Therapy for Rare Disease Market based on the following parameters - company overview, financial performance, product portfolio, geographical presence, distribution strategies, key developments and strategies
• Key companies covered as a part of this study include Kite Pharma, Inc. (Gilead Sciences, Inc.), Novartis International AG, Juno Therapeutics Inc. (Celgene Corporation), Bluebird Bio, Inc., Spark Therapeutics, Inc., uniQure N.V, Orchard Therapeutics Plc., PTC Therapeutics, Inc., and BioMarin Pharmaceutical Inc.
• Insights from this report would allow marketers and the management authorities of the companies to make informed decision regarding their future product launches, by Technology up-gradation, market expansion, and marketing tactics
• The global Gene Therapy for Rare Disease Market report caters to various stakeholders in this industry including investors, suppliers, product manufacturers, distributors, new entrants, and financial analysts
• Stakeholders would have ease in decision making through the various strategy matrices used in analyzing the Gene Therapy for Rare Disease Market
Detailed Segmentation:
• Global Gene Therapy for Rare Disease Market, By Drug:
o Approved Drugs
o Tisagenlecleucel (Kymriah)
o Axicabtagene ciloleucel (Yescarta)
o Voretigene neparvovec (Luxturna)
o Strimvelis
o Pipeline Drugs
o GT-AADC
o Fidanacogene elaparvovec (SPK-9011)
o OTL-200
o bb2121
o AMT-061
o Others
• Global Gene Therapy for Rare Disease Market, By Therapeutic Application:
o Oncology
o Neurological Disorders
o Ophthalmic Disorders
o Hematological Disorders
o Immunodeficiency Disorders
o Metabolic Disorders
o Others
• Global Gene Therapy for Rare Disease Market, By Region:
o North America
o By Drug
o Approved Drugs
o Tisagenlecleucel (Kymriah)
o Axicabtagene ciloleucel (Yescarta)
o Voretigene neparvovec (Luxturna)
o Strimvelis
o Pipeline Drugs
o GT-AADC
o Fidanacogene elaparvovec (SPK-9011)
o OTL-200
o bb2121
o AMT-061
o Others
o By Therapeutic Application:
o Oncology
o Neurological Disorders
o Ophthalmic Disorders
o Hematological Disorders
o Immunodeficiency Disorders
o Metabolic Disorders
o Others
o By Country:
o U.S.
o Canada
o Europe
o By Drug
o Approved Drugs
o Tisagenlecleucel (Kymriah)
o Axicabtagene ciloleucel (Yescarta)
o Voretigene neparvovec (Luxturna)
o Strimvelis
o Pipeline Drugs
o GT-AADC
o Fidanacogene elaparvovec (SPK-9011)
o OTL-200
o bb2121
o AMT-061
o Others
o By Therapeutic Application:
o Oncology
o Neurological Disorders
o Ophthalmic Disorders
o Hematological Disorders
o Immunodeficiency Disorders
o Metabolic Disorders
o Others
o By Country:
o U.K.
o Germany
o Italy
o France
o Spain
o Russia
o Rest of Europe
o Asia Pacific
o By Drug
o Approved Drugs
o Tisagenlecleucel (Kymriah)
o Axicabtagene ciloleucel (Yescarta)
o Voretigene neparvovec (Luxturna)
o Strimvelis
o Pipeline Drugs
o GT-AADC
o Fidanacogene elaparvovec (SPK-9011)
o OTL-200
o bb2121
o AMT-061
o Others
o By Therapeutic Application:
o Oncology
o Neurological Disorders
o Ophthalmic Disorders
o Hematological Disorders
o Immunodeficiency Disorders
o Metabolic Disorders
o Others
o By Country:
o China
o India
o Japan
o ASEAN
o Australia
o South Korea
o Rest of Asia Pacific
o Latin America
o By Drug
o Approved Drugs
o Tisagenlecleucel (Kymriah)
o Axicabtagene ciloleucel (Yescarta)
o Voretigene neparvovec (Luxturna)
o Strimvelis
o Pipeline Drugs
o GT-AADC
o Fidanacogene elaparvovec (SPK-9011)
o OTL-200
o bb2121
o AMT-061
o Others
o By Therapeutic Application:
o Oncology
o Neurological Disorders
o Ophthalmic Disorders
o Hematological Disorders
o Immunodeficiency Disorders
o Metabolic Disorders
o Others
o By Country:
o Brazil
o Mexico
o Argentina
o Rest of Latin America
o Middle East
o By Drug
o Approved Drugs
o Tisagenlecleucel (Kymriah)
o Axicabtagene ciloleucel (Yescarta)
o Voretigene neparvovec (Luxturna)
o Strimvelis
o Pipeline Drugs
o GT-AADC
o Fidanacogene elaparvovec (SPK-9011)
o OTL-200
o bb2121
o AMT-061
o Others
o By Therapeutic Application:
o Oncology
o Neurological Disorders
o Ophthalmic Disorders
o Hematological Disorders
o Immunodeficiency Disorders
o Metabolic Disorders
o Others
o By Country:
o GCC
o Israel
o Rest of Middle East
o Africa
o By Drug
o Approved Drugs
o Tisagenlecleucel (Kymriah)
o Axicabtagene ciloleucel (Yescarta)
o Voretigene neparvovec (Luxturna)
o Strimvelis
o Pipeline Drugs
o GT-AADC
o Fidanacogene elaparvovec (SPK-9011)
o OTL-200
o bb2121
o AMT-061
o Others
o By Therapeutic Application:
o Oncology
o Neurological Disorders
o Ophthalmic Disorders
o Hematological Disorders
o Immunodeficiency Disorders
o Metabolic Disorders
o Others
o By Country:
o Central Africa
o South Africa
o North Africa
• Company Profiles
o Kite Pharma, Inc. (Gilead Sciences, Inc.)*
o Company Overview
o Product Portfolio
o Financial Performance
o Key Developments
o Strategies
o Novartis International AG
o Juno Therapeutics Inc. (Celgene Corporation)
o Bluebird Bio, Inc.
o Spark Therapeutics, Inc.
o uniQure N.V
o Orchard Therapeutics Plc.
o PTC Therapeutics, Inc.
o BioMarin Pharmaceutical Inc.
"*" marked represents similar segmentation in other categories in the respective section.
Table of Contents
1. Research Objective and assumption
o Research Objectives
o Assumptions
o Abbreviations
2. Market Purview
o Report Description
o Market Definition and Scope
o Executive Summary
o Market Snippet, By Drug
o Market Snippet, By Therapeutic Application
o Market Snippet, By Region
o Coherent Opportunity Map (COM)
3. Market Dynamics, Regulations, and Trends Analysis
o Market Dynamics
o Drivers
o Restraints
o Market Opportunities
o PEST Analysis
o PORTER's Five Forces Analysis
o Regulatory Scenario
o Reimbursement Scenario
o Product Launches
o Partnership & Collaboration Scenario
o Pipeline Analysis
o Investment Scenario
o Epidemiology
4. Global Gene Therapy for Rare Disease Market, By Drug, 2018-2026 (US$ Mn)
o Introduction
o Market Share Analysis, 2017 and 2026 (%)
o Y-o-Y Growth Analysis, 2018-2026
o Segment Trends
o Approved Drugs
o Introduction
o Market Size and Forecast, and Y-o-Y Growth, 2016-2026, (US$ Mn)
o Tisagenlecleucel (Kymriah)
o Axicabtagene ciloleucel (Yescarta)
o Voretigene neparvovec (Luxturna)
o Strimvelis
o Pipeline Drugs
o Introduction
o Market Size and Forecast, and Y-o-Y Growth, 2016-2026, (US$ Mn)
o GT-AADC
o Fidanacogene elaparvovec (SPK-9011)
o OTL-200
o bb2121
o AMT-061
o Others
5. Global Gene Therapy for Rare Disease Market, By Therapeutic Application, 2018-2026 (US$ Mn)
o Introduction
o Market Share Analysis, 2017 and 2026 (%)
o Y-o-Y Growth Analysis, 2018-2026
o Segment Trends
o Oncology
o Introduction
o Market Size and Forecast, and Y-o-Y Growth, 2016-2026, (US$ Mn)
o Neurological Disorders
o Introduction
o Market Size and Forecast, and Y-o-Y Growth, 2016-2026, (US$ Mn)
o Ophthalmic Disorders
o Introduction
o Market Size and Forecast, and Y-o-Y Growth, 2016-2026, (US$ Mn)
o Hematological Disorders
o Introduction
o Market Size and Forecast, and Y-o-Y Growth, 2016-2026, (US$ Mn)
o Immunodeficiency Disorders
o Introduction
o Market Size and Forecast, and Y-o-Y Growth, 2016-2026, (US$ Mn)
o Metabolic Disorders
o Introduction
o Market Size and Forecast, and Y-o-Y Growth, 2016-2026, (US$ Mn)
o Others
o Introduction
o Market Size and Forecast, and Y-o-Y Growth, 2016-2026, (US$ Mn)
6. Global Gene Therapy for Rare Disease Market, By Regions, 2018-2026 (US$ Mn)
o Introduction
o Market Share Analysis, By Region, 2017 and 2026 (%)
o Y-o-Y Growth Analysis, For Regions, 2018-2026
o North America
o Market Size and Forecast, By Drug, 2018-2026 (US$ Mn)
o Market Size and Forecast, By Therapeutic Application, 2018-2026 (US$ Mn)
o Market Size and Forecast, By Country, 2018-2026 (US$ Mn)
o U.S.
o Canada
o Europe
o Market Size and Forecast, By Drug, 2018-2026 (US$ Mn)
o Market Size and Forecast, By Therapeutic Application, 2018-2026 (US$ Mn)
o Market Size and Forecast, By Country, 2018-2026 (US$ Mn)
o Germany
o Italy
o France
o Spain
o Russia
o Rest of Europe
o Asia Pacific
o Market Size and Forecast, By Drug, 2018-2026 (US$ Mn)
o Market Size and Forecast, By Therapeutic Application, 2018-2026 (US$ Mn)
o Market Size and Forecast, By Country, 2018-2026 (US$ Mn)
o China
o India
o Japan
o ASEAN
o Australia
o South Korea
o Rest of APAC
o Latin America
o Market Size and Forecast, By Drug, 2018-2026 (US$ Mn)
o Market Size and Forecast, By Therapeutic Application, 2018-2026 (US$ Mn)
o Market Size and Forecast, By Country, 2018-2026 (US$ Mn)
o Brazil
o Mexico
o Argentina
o Rest of Latin America
o Africa
o Market Size and Forecast, By Drug, 2018-2026 (US$ Mn)
o Market Size and Forecast, By Therapeutic Application, 2018-2026 (US$ Mn)
o Market Size and Forecast, By Country, 2018-2026 (US$ Mn)
o Central Africa
o South Africa
o North Africa
o Middle East
o Market Size and Forecast, By Drug, 2018-2026 (US$ Mn)
o Market Size and Forecast, By Therapeutic Application, 2018-2026 (US$ Mn)
o Market Size and Forecast, By Country, 2018-2026 (US$ Mn)
o GCC
o Israel
o Rest of Middle East
7. Competitive Landscape
o Heat Map Analysis
o Company Profiles
o Kite Pharma, Inc. (Gilead Sciences, Inc.)
o Company Overview
o Product Portfolio
o Key Developments
o Financial Performance
o Strategies
o Novartis International AG
o Company Overview
o Product Portfolio
o Key Developments
o Financial Performance
o Strategies
o Juno Therapeutics Inc. (Celgene Corporation)
o Company Overview
o Product Portfolio
o Key Developments
o Financial Performance
o Strategies
o Bluebird Bio, Inc.
o Company Overview
o Product Portfolio
o Key Developments
o Financial Performance
o Strategies
o Spark Therapeutics, Inc.
o Company Overview
o Product Portfolio
o Key Developments
o Financial Performance
o Strategies
o uniQure N.V
o Company Overview
o Product Portfolio
o Key Developments
o Financial Performance
o Strategies
o Orchard Therapeutics Plc.
o Company Overview
o Product Portfolio
o Key Developments
o Financial Performance
o Strategies
o PTC Therapeutics Inc.
o Company Overview
o Product Portfolio
o Key Developments
o Financial Performance
o Strategies
o BioMarin Pharmaceutical Inc.
o Company Overview
o Product Portfolio
o Key Developments
o Financial Performance
o Strategies
8. Section
o References
o Research Methodology
o About us and Sales Contact
*Browse 26 market data tables and 37 figures on "Gene Therapy for Rare Diseases Market - Global forecast to 2026".
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