Overview


According to FutureWise analysis, the market for Gene Therapy for Rare Disease is expected to grow with a CAGR of 35% over the forecast period 2024-2032, and reach a market value of US$ 1.08 Billion by 2031. 

Gene therapy for rare diseases is a cutting-edge medical approach designed to combat disorders stemming from specific genetic mutations. Unlike conventional treatments that manage symptoms, gene therapy aims to target the underlying genetic cause of a disease directly. This entails the introduction of genetic material into a patient's cells, either to rectify, replace, or supplement the defective genes responsible for the disorder.

The process begins with meticulously identifying the genetic mutation or abnormality responsible for the rare disease. This necessitates thorough genetic testing and analysis to pinpoint the precise genetic alteration. Subsequently, therapeutic genes are developed to address the identified mutation. These therapeutic genes are typically introduced into the patient's cells through various techniques, which may include engineered viruses as vectors or alternative delivery systems. Gene therapy for rare diseases offers a host of advantages. It can provide a lasting or permanent solution for specific rare genetic disorders, addressing the root cause at the genetic level. Furthermore, this approach can be personalized to cater to the unique genetic makeup of each patient, paving the way for individualized treatment plans. Notably, successful gene therapy may eliminate the need for lifelong drug regimens for certain rare diseases, thus improving overall quality of life.

FutureWise Market Research has published a report that provides an insightful analysis of Gene Therapy for Rare Disease Market trends that are affecting the overall market growth. This report will provide a detailed analysis of market share, regional insights, and competitor analysis that includes stature of key manufacturers operational in this industry. By the end of the forecast period FutureWise research analysts' projects that the Gene Therapy for Rare Disease Market will experience a significant growth. According to the analysis done, this report will help understand the information referring to the total valuation held by this industry. Additionally, this report will help in understanding the growth opportunities held by various segments of this market, further assist in making better strategic and expansion decisions by key stakeholders of an organization.

  • 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.
  • BioMarin Pharmaceutical Inc.

(Note: The list of the major players will be updated with the latest market scenario and trends)

Advances in genetic research have led to a deeper understanding of the genetic basis of rare diseases, driving the demand for targeted gene therapies. Regulatory agencies have established expedited pathways and incentives for developing and approving gene therapies for rare diseases, accelerating market entry. Growing investments from pharmaceutical companies, biotech firms, and government initiatives fuel research and development efforts in gene therapy for rare diseases. Ongoing progress in genetic engineering, gene editing techniques (like CRISPR-Cas9), and viral vectors have significantly improved the precision and efficiency of gene therapy delivery. Many gene therapies targeting rare diseases receive orphan drug designation, providing benefits such as extended market exclusivity, tax credits, and research grants, facilitating their development and commercialization,  hence driving the market.

However, gene therapies' research, development, and production are resource-intensive, necessitating significant financial investment and time. Rare diseases affect a few individuals, potentially limiting the market size and making it challenging to justify development costs. The production and purification of gene therapies can be technically complex, potentially leading to manufacturing challenges and regulatory compliance issues, hampering market growth.

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 Region

  • North America
  • Europe
  • Asia-Pacific
  • Latin America
  • Middle East and Africa

This market research report also emphasis on factors affecting the growth rate in various regions listed above. A deep-down analysis of the region will also be provided in the final version of this market, which is based on the conclusion of primary interviews and secondary data points gathered during the process.

By region, the market is segmented into North America, Latin America, Europe, Asia-Pacific, and Middle East & Africa. North America registered the highest share in the market in 2022 due to robust healthcare infrastructure, the prominent presence of key industry players, and a Supportive regulatory environment for gene therapy development. High prevalence of rare diseases and a strong focus on orphan drug development. Conversely, the Asia-Pacific region is poised for substantial expansion in the forecast period due to the rapid adoption of evolving lifestyles, an upswing in research initiatives, access to extensive untapped markets, a sizable population base, and an escalating demand for high-quality healthcare services.

  • Tier 1 players- established companies in the market with a major market share
  • Tier 2 players
  • Emerging players which are growing rapidly
  • New Entrants

  • Growth prospects
  • SWOT analysis
  • Key trends
  • Key data-points affecting market growth

  • To provide with an exhaustive analysis on the Gene Therapy for Rare Disease Market By Drug, By Therapeutic Application and By Region
  • To cater comprehensive information on factors impacting market growth (drivers, restraints, opportunities, and industry-specific restraints)
  • To evaluate and forecast micro-markets and the overall market
  • To predict the market size, in key regions— North America, Europe, Asia Pacific, Latin America and Middle East and Africa.
  • To record and evaluate competitive landscape, mapping- product launches, technological advancements, mergers and expansions

  • We have a flexible delivery model and you can suggest changes in the scope/table of content as per your requirement
  • The customization Mobility Care offered are free of charge with purchase of any license of the report
  • You can directly share your requirements/changes to the current table of content to: sales@futurewiseresearch.com

Table of Contents


  • 1.  Market Introduction
    •   1. Objectives of the Study 
        2. Market Definition 
        3. Market Scope 
           3.1. Years Considered for the Study
           3.2. Market Covered
        4. Currency 
        5. Limitations 
        6. Stakeholders 

  • 2.  Research Methodology
    •   1 Research Data 
           1.1. Secondary Data
              1.1.1. Key Data from Secondary Sources
           1.2 Primary Data
              1.2.1. Key Data from Primary Sources
        3.Market Size Estimation 
        4. Market Breakdown and Data Triangulation 
        5. Assumptions for the Study 

  • 3.  Executive Summary
    •   1. Market Outlook
        2. Segment Outlook
        3. Competitive Insights

  • 4.  Gene Therapy for Rare Disease Market Variables, Trends & Scope
    •   1. Market Lineage Outlook
        2. Penetration and Growth Prospect Mapping
        3. Industry Value Chain Analysis
        4. Cost Analysis Breakdown
        5. Application Overview
        6. Regulatory Framework
           6.1. Reimbursement Framework
           6.2. Standards and Compliances

  • 5.  Market Overview
    •   1. Market Dynamics
           1.1. Market Driver Analysis
              1.1.1. Increasing focus of Gene Therapy for Rare Disease Market Companies on Brand Protection
              1.1.2. Untapped Opportunities in Emerging Regions
           1.2. Market Restraint Analysis
              1.2.1. High Cost Associated with Implementation of Predictive Analysis
           1.3. Industry Challenges
              1.3.1. Presence of Ambiguous Regulatory Framework

  • 6.  Gene Therapy for Rare Disease Market Analysis Tools
    •   1. Industry Analysis - Porter’s
           1.1. Supplier Power
           1.2. Buyer Power
           1.3. Substitution Threat
           1.4. Threat from New Entrants
           1.5. Competitive Rivalry
        2. Pestel Analysis
           2.1. Political Landscape
           2.2. Environmental Landscape
           2.3. Social Landscape
           2.4. Application Landscape
           2.5. Legal Landscape
        3. Major Deals And Strategic Alliances Analysis
           3.1. Joint Ventures
           3.2. Mergers and Acquisitions
           3.3. Licensing and Partnership
           3.4. Application Collaborations
           3.5. Strategic Divestments
        4. Market Entry Strategies
        5. Case Studies

  • 7.  Gene Therapy for Rare Disease Market, By Drug Historical Analysis and Forecast 2024-2032 (USD Million)
      1. Approved Drugs (Tisagenlecleucel (Kymriah),
      2. Axicabtagene ciloleucel (Yescarta),
      3. Voretigene neparvovec (Luxturna), Strimvelis)
      4. Pipeline Drugs (GT-AADC, Fidanacogene elaparvovec (SPK-9011)
      5. OTL-200
      6. bb2121
      7. AMT-061
      8. Others 
  • 8.  Gene Therapy for Rare Disease Market, By Therapeutic Application Historical Analysis and Forecast 2024-2032 (USD Million)
      1. Oncology
      2. Neurological Disorders
      3. Ophthalmic Disorders
      4. Hematological Disorders
      5. Immunodeficiency Disorders
      6. Metabolic Disorders 
      7. Others 
  • 9.  North America Gene Therapy for Rare Disease Market Analysis 2018-2023 and Forecast 2024-2032 (USD Million)
    •   1. Introduction
        2. Historical Market Size (USD Million) Analysis By Country, 2018-2023
           2.1. U.S.A
           2.2. Canada
           2.3. Mexico
        3. Market Size (USD Million) Forecast for North America 2024-2032

  • 10.  Latin America Gene Therapy for Rare Disease Market Analysis 2018-2023 and Forecast 2024-2032 (USD Million)
    •   1. Introduction
        2. Historical Market Size (USD Million) Analysis By Country, 2018-2023
           2.1. Brazil
           2.2. Venezuela
           2.3. Argentina
           2.4. Rest of Latin America
        3. Market Size (USD Million) Forecast for Latin America 2024-2032

  • 11.  Europe Gene Therapy for Rare Disease Market Analysis 2018-2023 and Forecast 2024-2032 (USD Million)
    •   1. Introduction
        2. Historical Market Size (USD Million) Analysis By Country, 2018-2023
           2.1. Germany
           2.2. U.K
           2.3. France
           2.4. Italy
           2.5. Spain
           2.6. Russia
           2.7. Poland
           2.8. Rest of Europe
        3. Market Size (USD Million) Forecast for Europe 2024-2032

  • 12.  Asia Pacific Gene Therapy for Rare Disease Market Analysis 2018-2023 and Forecast 2024-2032 (USD Million)
    •   1. Introduction
        2. Historical Market Size (USD Million) Analysis By Country, 2018-2023
           2.1. Japan
           2.2. China
           2.3. India
           2.4. Australia and New Zealand
           2.5. ASEAN
           2.6. Rest of Asia Pacific
        3. Market Size (USD Million) Forecast for Asia Pacific 2024-2032 

  • 13.  Middle East and Africa Market Analysis 2018-2023 and Forecast 2024-2032 (USD Million)
    •   1. Introduction
        2. Historical Market Size (USD Million) Analysis By Country, 2018-2023
           2.1. GCC
           2.2. Israel
           2.3. South Africa
           2.4. Rest of MEA
        3. Market Size (USD Million) Forecast for MEA 2024-2032

  • 14.  Market Share Analysis and Competitive Landscape
    •   1. Global Landscape - Key Players, Revenue and Presence
        2. Global Share Analysis - Key Players (Tier 1, Tier 2, Tier 3)
        3. Global Emerging Companies
        4. North America - Market Share Analysis and Key Regional Players
        5. Europe - Market Share Analysis and Key Regional Players
        6. Asia Pacific - Market Share Analysis and Key Regional Players
        7. Global Key Player - Growth Matrix

  • 15.  Company Profiles (Competition Dashboard, Competitors Deep Dive, Products Offered and Financial Layouts)
    •   1. Kite Pharma, Inc. (Gilead Sciences, Inc.)
           1.1. Company Overview
           1.2. Product Portfolio
           1.3. SWOT Analysis
           1.4. Financial Overview
           1.5. Strategic Overview
        2. Novartis International AG
           2.1. Company Overview
           2.2. Product Portfolio
           2.3. SWOT Analysis
           2.4. Financial Overview
           2.5. Strategic Overview
        3. CJuno Therapeutics Inc. (Celgene Corporation)
           3.1. Company Overview
           3.2. Product Portfolio
           3.3. SWOT Analysis
           3.4. Financial Overview
           3.5. Strategic Overview
        4. Bluebird Bio, Inc.
           4.1. Company Overview
           4.2. Product Portfolio
           4.3. SWOT Analysis
           4.4. Financial Overview
           4.5. Strategic Overview
        5. Spark Therapeutics, Inc.
           5.1. Company Overview
           5.2. Product Portfolio
           5.3. SWOT Analysis
           5.4. Financial Overview
           5.5. Strategic Overview
        6. uniQure N.V
           6.1. Company Overview
           6.2. Product Portfolio
           6.3. SWOT Analysis
           6.4. Financial Overview
           6.5. Strategic Overview
        7. Orchard Therapeutics Plc.
           7.1. Company Overview
           7.2. Product Portfolio
           7.3. SWOT Analysis
           7.4. Financial Overview
           7.5. Strategic Overview
        8. PTC Therapeutics, Inc.
           8.1. Company Overview
           8.2. Product Portfolio
           8.3. SWOT Analysis
           8.4. Financial Overview
           8.5. Strategic Overview
        9. BioMarin Pharmaceutical Inc.
           9.1. Company Overview
           9.2. Product Portfolio
           9.3. SWOT Analysis
           9.4. Financial Overview
           9.5. Strategic Overview

  • 16.  Pre and Post COVID-19 Impact
    •   1. Positive influence on the healthcare industry
        2. The financial disruption of the manufacturing sector
        3. Impact of COVID-19 on emerging companies
        4. Significant mandates in the healthcare regulations initiated by administrations
        5. The overall economic slowdown of the developing and developed nations

  • 17.  FutureWise SME Key Takeaway Points for Client
    •    

Partner

Our Clients