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, and Others),   By Therapeutic Application (Oncology, Neurological Disorders, Ophthalmic Disorders, Hematological Disorders, Immunodeficiency Disorders, Metabolic Disorders, and Others), and Region (North America, Latin America, Europe, Asia Pacific, the Middle East, and Africa) - Size, Share, Outlook, and Opportunity Analysis, 2018 – 2026

  • Published On : Nov 2018 |
  • Pages : 240 Pages |
  • Format : CMI Insight PPT Format CMI Insight PDF Format

 Gene Therapy for Rare Disease Market - Insights

Gene therapy involves insertion of genetic material into cells of the patients to provide new function or to restore missing or inappropriate functioning of the cells due to cancer or gene mutations. According to National Center for Advancing Translational Sciences, although around 7,000 rare diseases are identified, only a few hundred have approved treatments. Moreover, developing gene therapy for rare diseases is gaining significant traction, as more than 80% of rare diseases have a known monogenic (single-gene) cause. Small molecule drugs often treat symptoms only rather than curing underlying cause of the disease. This requires frequent administration of the drugs to manage the condition. Gene therapy holds the potential to correct underlying genetic defects. Moreover, a clinically sound and successful gene therapy may only require a single dose to cure a disease rather than requiring a lifetime treatment. Commercially approved gene therapies include Kymriah (Novartis AG), Yescarta (Gilead Sciences, Inc.), Luxturna (Spark Therapeutics), and Strimvelis (Orchard Therapeutics).

Increasing drug approvals and launches of novel gene therapies for treatment of various rare diseases are expected to boost the gene therapy for rare disease market growth over the forecast period

In recent past, key players in the market have gained regulatory approvals and have launched their therapies in the market. Frequent approvals of novel gene therapies and their launches in the developed regions are expected to significantly drive gene therapy for rare disease market growth over the forecast period. For instance, in December 2017, Spark Therapeutics, Inc. received the U.S. Food and Drug Administration (FDA) approval for its LUXTURNA (voretigene neparvovec-rzyl), a one-time gene therapy product indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. Furthermore, in August 2018, Novartis International AG received European Commission’s (EC) approval for Kymriah (tisagenlecleucel, formerly CTL019) indicated for treatment of patients up to 25 years of age with relapse or refractory B-cell acute lymphoblastic leukemia (ALL) and for the treatment of adult patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy. Moreover, 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.

The global gene therapy for rare disease market size was valued at US$ 16.3 million in 2017, and is expected to witness a robust CAGR of 35.0% over the forecast period (2018–2026).

                            Figure No.1: Global Gene Therapy for Rare Disease Market Share, By Drug (%), 2018 -2026                                                                                                                                                                                                       

Source: Coherent Market Insights Analysis (2018)

Ongoing research and development in gene therapies for rare diseases is expected to boost growth of the global gene therapy for rare disease market

Presence of multiple novel gene therapies for various rare disease treatment in pipeline, which are expected to gain approval is expected to boost the market growth over the forecast period. For instance, Spark Therapeutics, Inc. is developing gene therapy namely Fidanacogene Elaparvovec, a novel, investigational gene therapy indicated for the treatment of patients suffering from hemophilia B. Currently, the therapy is under Phase 3 clinical trial and is expected to receive approval in near future. In August 2018, uniQure N.V. treated the first patient in its Phase IIb dose-confirmation study of AMT-061, an investigational AAV5-based gene therapy incorporating the FIX-Padua variant for the treatment of patients with severe and moderately severe hemophilia B. In May 2018, Orchard Therapeutics Plc. received Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for its gene therapy candidate OTL-200, indicated for treatment of patients with metachromatic leukodystrophy (MLD).  PTC Therapeutics, In. has GT-AADC in phase 3 clinical trials and is indicated for Aromatic L-Amino Acid Decarboxylase (AADC) deficiency.

Table No.1: Gene Therapy for Rare Disease Pipeline

Source: Company Websites, Clinicaltrials.gov

However, lack of awareness among population regarding rare diseases and their treatment is expected to negatively affect growth of the gene therapy for rare disease market. For instance, in 2017, uniQure N.V. dropped their plant to pursue the renewal of its gene therapy Glybera, which was approved in 2012, for treatment for a small subset of patients with familial lipoprotein lipase deficiency (LPLD). The company dropped their plan due to very low demand of therapy in the market

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.

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:
    • 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
  • Global Gene Therapy for Rare Disease Market, By Therapeutic Application:
    • Oncology
    • Neurological Disorders
    • Ophthalmic Disorders
    • Hematological Disorders
    • Immunodeficiency Disorders
    • Metabolic Disorders
    • Others
  • Global Gene Therapy for Rare Disease Market, By Region:
    • North America
      • 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
      • By Therapeutic Application:
        • Oncology
        • Neurological Disorders
        • Ophthalmic Disorders
        • Hematological Disorders
        • Immunodeficiency Disorders
        • Metabolic Disorders
        • Others
      • By Country:
        • U.S.
        • Canada
    • Europe
      • 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
      • By Therapeutic Application:
        • Oncology
        • Neurological Disorders
        • Ophthalmic Disorders
        • Hematological Disorders
        • Immunodeficiency Disorders
        • Metabolic Disorders
        • Others
      • By Country:
        • U.K.
        • Germany
        • Italy
        • France
        • Spain
        • Russia
        • Rest of Europe
    • Asia Pacific
      • 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
      • By Therapeutic Application:
        • Oncology
        • Neurological Disorders
        • Ophthalmic Disorders
        • Hematological Disorders
        • Immunodeficiency Disorders
        • Metabolic Disorders
        • Others
      • By Country:
        • China
        • India
        • Japan
        • ASEAN
        • Australia
        • South Korea
        • Rest of Asia Pacific
    • Latin America
      • 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
      • By Therapeutic Application:
        • Oncology
        • Neurological Disorders
        • Ophthalmic Disorders
        • Hematological Disorders
        • Immunodeficiency Disorders
        • Metabolic Disorders
        • Others
      • By Country:
        • Brazil
        • Mexico
        • Argentina
        • Rest of Latin America
    • Middle East
      • 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
      • By Therapeutic Application:
        • Oncology
        • Neurological Disorders
        • Ophthalmic Disorders
        • Hematological Disorders
        • Immunodeficiency Disorders
        • Metabolic Disorders
        • Others
      • By Country:
        • GCC
        • Israel
        • Rest of Middle East
    • Africa
      • 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
      • By Therapeutic Application:
        • Oncology
        • Neurological Disorders
        • Ophthalmic Disorders
        • Hematological Disorders
        • Immunodeficiency Disorders
        • Metabolic Disorders
        • Others
      • By Country:
        • Central Africa
        • South Africa
        • North Africa
  • Company Profiles
    • Kite Pharma, Inc. (Gilead Sciences, Inc.)*
      • Company Overview
      • Product Portfolio
      • Financial Performance
      • Key Developments
      • Strategies
    • Novartis International AG
    • Juno Therapeutics Inc. (Celgene Corporation)
    • Bluebird Bio, Inc.
    • Spark Therapeutics, Inc.
    • uniQure N.V
    • Orchard Therapeutics Plc.
    • PTC Therapeutics, Inc.
    • BioMarin Pharmaceutical Inc.

 “*” marked represents similar segmentation in other categories in the respective section.

Table of Contents

  1. Research Objective and assumption
    • Research Objectives
    • Assumptions
    • Abbreviations
  2. 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)
  3. 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
  4. 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
  5. 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)
  6. 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)
        • U.S.
        • Canada
    • Europe
      • 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)
        • Germany
        • Italy
        • France
        • Spain
        • Russia
        • Rest of Europe
    • Asia Pacific
      • 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)
        • China
        • India
        • Japan
        • ASEAN
        • Australia
        • South Korea
        • Rest of APAC
    • Latin 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)
        • Brazil
        • Mexico
        • Argentina
        • Rest of Latin America
    • Africa
      • 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)
        • Central Africa
        • South Africa
        • North Africa
    • Middle East
      • 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)
        • GCC
        • Israel
        • Rest of Middle East
  7. Competitive Landscape
    • Heat Map Analysis
    • Company Profiles
      • Kite Pharma, Inc. (Gilead Sciences, Inc.)
        • Company Overview
        • Product Portfolio
        • Key Developments
        • Financial Performance
        • Strategies
      • Novartis International AG
        • Company Overview
        • Product Portfolio
        • Key Developments
        • Financial Performance
        • Strategies
      • Juno Therapeutics Inc. (Celgene Corporation)
        • Company Overview
        • Product Portfolio
        • Key Developments
        • Financial Performance
        • Strategies
      • Bluebird Bio, Inc.
        • Company Overview
        • Product Portfolio
        • Key Developments
        • Financial Performance
        • Strategies
      • Spark Therapeutics, Inc.
        • Company Overview
        • Product Portfolio
        • Key Developments
        • Financial Performance
        • Strategies
      • uniQure N.V
        • Company Overview
        • Product Portfolio
        • Key Developments
        • Financial Performance
        • Strategies
      • Orchard Therapeutics Plc.
        • Company Overview
        • Product Portfolio
        • Key Developments
        • Financial Performance
        • Strategies
      • PTC Therapeutics Inc.
        • Company Overview
        • Product Portfolio
        • Key Developments
        • Financial Performance
        • Strategies
      • BioMarin Pharmaceutical Inc.
        • Company Overview
        • Product Portfolio
        • Key Developments
        • Financial Performance
        • Strategies  
  8. Section
    • References
    • Research Methodology
    • About us and Sales Contact

*Browse 26 market data tables and 37 figures on "Gene Therapy for Rare Diseases Market - Global forecast to 2026”.

Research Methodology

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  • Primary Research (Trade Surveys and Experts Interviews)
  • Desk Research
  • Proprietor Data Analytics Model

In addition to this, Coherent Market Insights has access to a wide range of the regional and global reputed paid data bases, which helps the company to figure out the regional and global market trends and dynamics. The company analyses the industry from the 360 Degree Perspective i.e. from the Supply Side and Demand Side which enables us to provide granular details of the entire ecosystem for each study. Finally, a Top-Down approach and Bottom-Up approach is followed to arrive at ultimate research findings.


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  • National level desk research: It Includes research analysis of regional players, regional regulatory bodies, regional trade associations, and regional organization.
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Coherent Market Insights has a large amount of in-house repository of industry database. This is leveraged as a burner for initiating a new research study. Key secondary sources include:

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The raw data is obtained through the secondary findings, in house repositories, and trade surveys. It is then filtered to ensure that the relevant information including industry dynamics, trends, and outlook is retained for further research process.

Data Standardization:

Holistic approach is used to ensure that the granular and uncommon parameters are taken into consideration to ensure accurate results. The information from the paid databases are further combined to the raw data in order to standardize it.

Coherent Statistical model

We arrive at our final research findings through simulation models. Coherent Data Analytics Model is a statistical tool that helps company to forecast market estimates. Few of the parameters considered as a part of the statistical model include:

  • Micro-economic indicators
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Data Validation

This is the most crucial stage of the research process. Primary Interviews are conducted to validate the data and analysis. This helps in achieving the following purposes:

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  • Primary research includes online surveys, face-to face interviews, and telephonic interviews.

The primary research is conducted with the ecosystem players including, but not limited to:

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