Gene Therapy for Rare Disease Market Huge growth | 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., PT

Key players in the market are focused on research and development of gene therapies for rare diseases in order to introduce their pipeline products in the market. For instance, in October 2018, bluebird bio, Inc. received European Medicines Agency (EMA) marketing authorization application (MAA) for its investigational LentiGlobin gene therapy for the treatment of adolescents and adults with transfusion-dependent β-thalassemia (TDT) and a non-β0/β0 genotype.

In August 2018, Kite Pharma, Inc., a Gilead Company, received European Commission (EC) Marketing approval for its novel Yescarta (axicabtagene ciloleucel), as a treatment for adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL), after two or more lines of systemic therapy. Moreover, in September 2018, Spark Therapeutics Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending approval of LUXTURNA (voretigene neparvovec), a one-time gene therapy for the treatment of adult and pediatric patients with vision loss

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Some of the major players operating in the global gene therapy for rare disease market 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.

Increasing research and development by leading players in gene therapy for rare disease is expected to support growth of the gene therapy for rare disease market. Several companies have novel gene therapies in the clinical stages for treatment of rare diseases ranging from rare cancers to rare genetic diseases. For instance, Spark Therapeutics, Inc. has SPK-7001 (Choroideremia/ Phase ½), SPK-8011 (Hemophilia A/Phase ½), and SPK-3006 (Pompe Disease/preclinical) in the pipeline.

Moreover, Orchard Therapeutics has multiple therapies in the pipeline such as OTL-101, OTL-103, OTL-102, OTL-200, and OTL-201 for various indications. Some of these therapies are expected to receive the U.S. Food & Drug Administration approvals during the forecast period. These factors are expected to boost the global gene therapy for rare disease market growth over the forecast period.

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

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Detailed Segmentation:

Global Gene Therapy for Rare Disease Market, By Drug:
Approved Drugs
Tisagenlecleucel (Kymriah)
Axicabtagene ciloleucel (Yescarta)
Voretigene neparvovec (Luxturna)
Strimvelis
Pipeline Drugs
GT-AADC
Fidanacogene elaparvovec (SPK-9011)
OTL-200
bb2121
AMT-061
Others

Table of Contents

Research Objective and assumption
Research Objectives
Assumptions
Abbreviations
Market Purview
Report Description
Market Definition and Scope
Executive Summary
Market Snippet, By Drug
Market Snippet, By Therapeutic Application
Market Snippet, By Region
Coherent Opportunity Map (COM)
Market Dynamics, Regulations, and Trends Analysis
Market Dynamics
Drivers
Restraints
Market Opportunities
PEST Analysis
PORTER’s Five Forces Analysis
Regulatory Scenario
Reimbursement Scenario
Product Launches
Partnership & Collaboration Scenario
Pipeline Analysis
Investment Scenario
Epidemiology
Global Gene Therapy for Rare Disease Market, By Drug, 2018–2026 (US$ Mn)
Introduction
Market Share Analysis, 2017 and 2026 (%)
Y-o-Y Growth Analysis, 2018–2026
Segment Trends
Approved Drugs
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Mn)
Tisagenlecleucel (Kymriah)
Axicabtagene ciloleucel (Yescarta)
Voretigene neparvovec (Luxturna)
Strimvelis
Pipeline Drugs
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Mn)
GT-AADC
Fidanacogene elaparvovec (SPK-9011)
OTL-200
bb2121
AMT-061
Others
Global Gene Therapy for Rare Disease Market, By Therapeutic Application, 2018–2026 (US$ Mn)
Introduction
Market Share Analysis, 2017 and 2026 (%)
Y-o-Y Growth Analysis, 2018–2026
Segment Trends
Oncology
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Mn)
Neurological Disorders
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Mn)
Ophthalmic Disorders
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Mn)
Hematological Disorders
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Mn)
Immunodeficiency Disorders
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Mn)
Metabolic Disorders
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Mn)
Others
Introduction
Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Mn)
Global Gene Therapy for Rare Disease Market, By Regions, 2018–2026 (US$ Mn)
Introduction
Market Share Analysis, By Region, 2017 and 2026 (%)
Y-o-Y Growth Analysis, For Regions, 2018–2026
North America
Market Size and Forecast, By Drug, 2018–2026 (US$ Mn)
Market Size and Forecast, By Therapeutic Application, 2018–2026 (US$ Mn)
Market Size and Forecast, By Country, 2018–2026 (US$ Mn)

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