Healthcare and Pharmaceuticals industry

Global Gene Therapy for Rare Disease Market Size Share Forecast 2025




 

Impact Analysis of Covid-19

The complete version of the REPORT will include the impact of the COVID-19, and anticipated change on the future outlook of the industry, by taking into the account the political, economic, social, and technological parameters.

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".

 



Frequently Asked Questions

The report efficiently evaluates the market from various dimensions to deliver an end-product that is informative, elaborate, and accurate and includes detailed market segmentation, regional analysis, and competitive landscape of the industry.
The report efficiently evaluates the current market size and provides industry forecast. The market was valued at xx Million US$ in 2019, and is expected to grow at a CAGR of xx% during the period 2020-2027.
The report efficiently evaluates the current market size and provides forecast for the industry in terms of Value (US$ Mn) and Volume (Mn Units).
Market is segmented by Product Types, Drug Class, Indication, Applications, Services Types, End-users, Distribution channel, and Regions.
The report presents the current market size, and market forecast, market opportunities, key drivers and restraints, pipeline Analysis, incidence rate, epidemiology analysis, prevalence rate, regulatory scenario, industry trend, PESTLE analysis, PORTER’s analysis, new product approvals/launch, promotion and marketing initiatives, pricing analysis, export-import analysis, trade analysis, competitive landscape which help businesses in decision making. The analysis data is based on current and historical market trends which help in investment related decisions.
Custom research is crucial components of the business strategy which helps any organization to gain insight into specific business sector, aligned with specific area of interest. Thus, SMI offers more accurate, pragmatic, and actionable information specifically tailored to suit your business needs.

Get In Touch

In search of customized market research solution? We are here to help you. Contact us.