Gene Therapy for Rare Disease Market, By Drug (Approved Drugs (Tisagenlecleucel (Kymriah), Axicabtagene ciloleucel (Yescarta), Voretigene neparvovec (Luxturna), and 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 By Region (North America, Europe, Asia Pacific, Latin America, and Middle East & Africa) - Trends, Analysis and Forecast till 2034

Report Code: PMI39719 | Publish Date: March 2024 | No. of Pages: 187

Global Gene Therapy For Rare Overview

Introduction:

Gene Therapy for Rare Disease   Market was valued at US$ 144.5 million in 2024 and is projected to grow at a CAGR of 5.10% to reach US$ 3848.7 million by 2034.

Gene therapy for rare disease involves insertion of genetic material into cells of the patients in order to provide new function as well as to restore missing function cells due to cancer or gene alteration. Gene therapies have ability to cure the genetic defects. Gene therapies can be used to treat rare disorders like Hemophilia B, Hemophilia A, Fabry disease, and neuronopathic disorders.

Increasing approvals of drugs for treatment of rare diseases by the regulatory bodies across the globe is a key factor driving growth of the gene therapy for rare disease market. In addition, increasing launches of various gene therapies across the globe is a factor expected to propel growth of the global market in the near future. Furthermore, ongoing research and development activities in the gene therapies for treatment of rare diseases is another factor expected to boost growth of the Gene Therapy for Rare Disease market over the forecast period.

 

 

Global Gene Therapy For Rare Dynamics

Launch of novel therapies with growing drug approvals to fuel market growth

Launch of novel therapies with growing number of drug approvals has contributed in Gene Therapy for Rare Disease market growth. Presence of major key players and strategic partnerships has given rise in launch of new novel therapies which, in turn boost the market growth.

For instance, in October 2021, U.S. FDA (Food and Drug Administration), five profit organizations and ten pharmaceutical companies joined forces to develop gene therapies for rare diseases. By standardizing process and leveraging on experience with platform technology, gene therapy product development will be accelerated to allow more timely access to new therapies for needy patients.

Restrains:

However, high costs associated with treating rare diseases, coupled with lack of awareness regarding gene therapy among the individuals are major factors expected to restrain growth of the Gene Therapy for Rare Disease market over the forecast period.

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Global Gene Therapy For Rare Segmentation

Gene Therapy for Rare Disease Market is segmented based on Drug, Therapeutic Application and Region.

Drug Insight

On the basis of Drug, Gene Therapy for Rare Disease Market is segmented into Approved Drugs - Tisagenlecleucel (Kymriah), Axicabtagene ciloleucel (Yescarta), Voretigene neparvovec (Luxturna), and Strimvelis. On the basis of pipeline drugs market has been segmented as GT-AADC, Fidanacogene elaparvovec (SPK-9011), OTL-200, bb2121, AMT-061, and others.

Therapeutic Application Insights

On the basis of Therapeutic Application, Gene Therapy for Rare Disease Market is segmented into Oncology, Neurological Disorders, Ophthalmic Disorders, Hematological Disorders, Immunodeficiency Disorders, Metabolic Disorders, and Others. Oncology segment is expected to dominate the market growth due to growing prevalence of cancer diseases.

For instance, in 2019, according to CDC (Centers for Disease Control and Prevention), around 1,752,735 new cancer cases were reported and 599,589 people were died of cancer in United States.

Regional Insights:

On region the Gene Therapy for Rare Disease Market is segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. The North America market is estimated to witness a significantly high revenue share over the forecast period. Moreover, rising prevalence of rare disease, unhealthy diet, sedentary lifestyle, high disposable income, growing research and development activities, new product launch and approval of drugs is anticipated to increase demand for Gene Therapy for Rare Disease in the region.

For instance, according to CDC (Centers for Disease Control and Prevention), in United States rare disease affects fewer than 200,000 individual and as a whole affect about 25 million individual in United States and around 400 million individuals worldwide.

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Report Scope:

Attribute

Details

Base year for estimation

2023

Forecast period

2024 – 2034

Market representation

Revenue in USD Million & CAGR from 2024 to 2034

Market Segmentation

By Drug – Approved Drugs and Pipeline Drugs

By Therapeutic Application - Oncology, Neurological Disorders, Ophthalmic Disorders, Hematological Disorders, Immunodeficiency Disorders, Metabolic Disorders, and Others

Regional scope

North America - U.S., Canada

Europe - UK, Germany, Spain, France, Italy, Russia, Rest of Europe

Asia Pacific - Japan, India, China, South Korea, Australia, Rest of Asia-Pacific

Latin America - Brazil, Mexico, Argentina, Rest of Latin America

Middle East & Africa - South Africa, Saudi Arabia, UAE, Rest of Middle East & Africa

Report coverage

Revenue forecast, company share, competitive landscape, growth factors, and trends

Segments Covered in the Report:

This report forecasts revenue growth at global, regional, and country levels and provides an analysis of the latest industry trends and opportunities in each of the sub-segments from 2022 to 2032. For the purpose of this study, has segmented the Gene Therapy for Rare Disease Market report based on Drug, Therapeutic Application and Region.

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

Gene Therapy for Rare Disease Market, By Therapeutic Application:

  • Oncology
  • Neurological Disorders
  • Ophthalmic Disorders
  • Hematological Disorders
  • Immunodeficiency Disorders
  • Metabolic Disorders
  • Others

Gene Therapy for Rare Disease Market, By Region:

  • North America
    • U.S.
    • Canada
  • Europe
    • Germany
    • UK
    • France
    • Russia
    • Italy
    • Rest of Europe
  • Asia Pacific
    • China
    • India
    • Japan
    • South Korea
    • Rest of Asia Pacific
  • Latin America
    • Brazil
    • Mexico
    • Rest of Latin America
  • Middle East & Africa
    • GCC
    • Israel
    • South Africa
    • Rest of Middle East & Africa

Global Gene Therapy For Rare Key Players

The key players operating the Gene Therapy for Rare Disease Market includes, 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.

Global Gene Therapy For Rare Key Issues Addressed

Recent Development:

  • In March 2023, England’s NICE (National Institute for Health and Care Excellence) signs off an PTC Therapeutics “Upstaza” a gene therapy for ultra-rare genetic disease at $3.7 million to patients in U.K.
  • In April 2022, Novartis received FDA (Food and Drug Administration) approval for its first “Vijoice” treatment for pediatric and adult patients with severe PIK3CA – PROS (Related Overgrowth Spectrum) a spectrum of rare condition due to growth and blood vessel anomalies.
  • In May 2019, AaveXis received FDA (Food and Drug Administration) approval for its first and only gene therapy “Zolgensma” for pediatric patients with SMA (Spinal Muscular Atrophy) a rare genetic disease which leads to paralysis and progressive muscle weakness.

Global Gene Therapy For Rare Company Profile

  • Kite Pharma, Inc. (Gilead Sciences, Inc.)*
    • Company Overview
    • Product Portfolio
    • Key Highlights
    • Financial Performance
    • Business 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

Global Gene Therapy For Rare Table of Contents

  1. Research Objective and Assumption
    • Preface
    • Research Objectives
    • Study Scope
    • Years Considered for the study
    • Assumptions
    • Abbreviations
  2. Research Methodology
    • Research data
    • Primary Data
      • Primary Interviews
      • Primary Breakdown
      • Key data from Primary Sources
      • Key Thickness Insights
    • Secondary Data
      • Major Secondary Sources
      • Secondary Sources
    • Market Estimation
    • Top-Down Approach
      • Approach for estimating Market Share by Top-Down Analysis (Supply Side)
    • Bottom-Up Approach
      • Approach for estimating market share by Bottom-up Analysis (Demand Side)
    • Market Breakdown and Data Triangulation
    • Research Assumptions
  3. Market Preview
    • Executive Summary
    • Key Findings—Global Outlook for medical carts Strategies
      • Key Questions this Study will Answer
      • Market Snippet, By Drug
      • Market Snippet, By Therapeutic Application
      • Market Snippet, By Region
    • Opportunity Map Analysis
    • Executive Summary—3 Big Predictions
  4. Market Dynamics, Regulations, and Trends Analysis
    • Market Dynamics
      • Drivers
      • Restrains
      • Market Opportunities
      • Market Trends
    • DR Impact Analysis
    • PEST Analysis
    • Porter’s Five Forces Analysis
    • Opportunity Orbit
    • Market Investment Feasibility Index
    • Macroeconomic Factor Analysis
  5. Gene Therapy for Rare Disease Market, By Drug, Forecast Period up to 10 Years, (US$ Bn)
  • Overview
    • Market Value and Forecast (US$ Bn), and Share Analysis (%), Forecast Period up to 10 Years
    • Y-o-Y Growth Analysis (%), Forecast Period up to 10 Years
    • Segment Trends
  • Approved Drugs (Tisagenlecleucel (Kymriah), Axicabtagene ciloleucel (Yescarta), Voretigene neparvovec (Luxturna), and Strimvelis)
    • Overview
    • Market Size and Forecast (US$ Bn), and Y-o-Y Growth (%), Forecast Period up to 10 Years
  • Pipeline Drugs (GT-AADC, Fidanacogene elaparvovec (SPK-9011), OTL-200, bb2121, AMT-061, and Others)
    • Overview
    • Market Size and Forecast (US$ Bn), and Y-o-Y Growth (%), Forecast Period up to 10 Years
  1. Gene Therapy for Rare Disease Market, By Therapeutic Application, Forecast Period up to 10 Years, (US$ Bn)
  • Overview
    • Market Value and Forecast (US$ Bn), and Share Analysis (%), Forecast Period up to 10 Years
    • Y-o-Y Growth Analysis (%), Forecast Period up to 10 Years
    • Segment Trends
  • Oncology
    • Overview
    • Market Size and Forecast (US$ Bn), and Y-o-Y Growth (%), Forecast Period up to 10 Years
  • Neurological Disorders
    • Overview
    • Market Size and Forecast (US$ Bn), and Y-o-Y Growth (%), Forecast Period up to 10 Years
  • Ophthalmic Disorders
    • Overview
    • Market Size and Forecast (US$ Bn), and Y-o-Y Growth (%), Forecast Period up to 10 Years
  • Hematological Disorders
    • Overview
    • Market Size and Forecast (US$ Bn), and Y-o-Y Growth (%), Forecast Period up to 10 Years
  • Immunodeficiency Disorders
    • Overview
    • Market Size and Forecast (US$ Bn), and Y-o-Y Growth (%), Forecast Period up to 10 Years
  • Metabolic Disorders
    • Overview
    • Market Size and Forecast (US$ Bn), and Y-o-Y Growth (%), Forecast Period up to 10 Years
  • Others
    • Overview
    • Market Size and Forecast (US$ Bn), and Y-o-Y Growth (%), Forecast Period up to 10 Years
  1. Gene Therapy for Rare Disease Market, By Region, Forecast Period up to 10 Years, (US$ Bn)
    • Overview
      • Market Value and Forecast (US$ Bn), and Share Analysis (%), Forecast Period up to 10 Years
      • Y-o-Y Growth Analysis (%), Forecast Period up to 10 Years
      • Regional Trends
    • North America
      • Market Size and Forecast (US$ Bn), By Drug, Forecast Period up to 10 Years
      • Market Size and Forecast (US$ Bn), By Therapeutic Application, Forecast Period up to 10 Years
      • Market Size and Forecast (US$ Bn), By Country, Forecast Period up to 10 Years
        • U.S.
        • Canada
    • Europe
      • Market Size and Forecast (US$ Bn), By Drug, Forecast Period up to 10 Years
      • Market Size and Forecast (US$ Bn), By Therapeutic Application, Forecast Period up to 10 Years
      • Market Size and Forecast (US$ Bn), By Country, Forecast Period up to 10 Years
        • UK
        • France
        • Germany
        • Russia
        • Italy
        • Rest of Europe
    • Asia Pacific
      • Market Size and Forecast (US$ Bn), By Drug, Forecast Period up to 10 Years
      • Market Size and Forecast (US$ Bn), By Therapeutic Application, Forecast Period up to 10 Years
      • Market Size and Forecast (US$ Bn), By Country, Forecast Period up to 10 Years
        • India
        • Japan
        • South Korea
        • China
        • Rest of Asia Pacific
  • Latin America
    • Market Size and Forecast (US$ Bn), By Drug, Forecast Period up to 10 Years
    • Market Size and Forecast (US$ Bn), By Therapeutic Application, Forecast Period up to 10 Years
    • Market Size and Forecast (US$ Bn), By Country, Forecast Period up to 10 Years
  • Brazil
  • Mexico
  • Rest of Latin America
  • Middle East & Africa
    • Market Size and Forecast (US$ Bn), By Drug, Forecast Period up to 10 Years
    • Market Size and Forecast (US$ Bn), By Therapeutic Application, Forecast Period up to 10 Years
    • Market Size and Forecast (US$ Bn), By Country, Forecast Period up to 10 Years
  • GCC
  • Israel
  • South Africa
  • Rest of Middle East
  1. Competitive Landscape
    • Heat Map Analysis
    • Market Presence and Specificity Analysis
  2. Company Profiles
  • Novartis International AG
    • Company Overview
    • Product Portfolio
    • Key Highlights
    • Financial Overview
    • Business Strategies
  • Juno Therapeutics Inc.  (Celgene Corporation)
    • Company Overview
    • Product Portfolio
    • Key Highlights
    • Financial Overview
    • Business Strategies
  • Bluebird Bio, Inc.
    • Company Overview
    • Product Portfolio
    • Key Highlights
    • Financial Overview
    • Business Strategies
  • Spark Therapeutics, Inc.
    • Company Overview
    • Product Portfolio
    • Key Highlights
    • Financial Overview
    • Business Strategies
  • UniQure N.V
    • Company Overview
    • Product Portfolio
    • Key Highlights
    • Financial Overview
    • Business Strategies
  • PTC Therapeutics, Inc.
    • Company Overview
    • Product Portfolio
    • Key Highlights
    • Financial Overview
    • Business Strategies
  • Biomarin Pharmaceutical Inc.
    • Company Overview
    • Product Portfolio
    • Key Highlights
    • Financial Overview
    • Business Strategies
  • Cryo StemcelPrivate Limited
  1. The Last Word
    • Future Impact
    • About Us
    • Contact

 

FAQs

The Gene Therapy for Rare Disease Market is segmented into drug, therapeutic application and region.

Gene Therapy for Rare Disease Market is driven by factors like launch of novel therapies with growing drug approvals to fuel market growth.

By region, the Gene Therapy for Rare Disease market is segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. The North America market is estimated to witness a significantly high revenue share over the forecast period.

The key players operating the Gene Therapy for Rare Disease Market includes, 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.

Gene Therapy for Rare Disease Market was valued at US$ 144.5 Billion in 2024 and is projected to grow at a CAGR of 5.10% to reach US$ 3848.7 Billion by 2034.