The Gene Therapies For Rare Diseases Market is expected to grow at an 20.0 % CAGR during the forecast period for 2023-2031.
Gene Therapy is playing a major role in rare disease management. Gene therapy in rare diseases is a specialized and targeted treatment that acts via the insertion of corrected genes into cells of an individual. Around 80 percent of rare diseases are accounted with genetic etiology. Gene therapy is considered as proven with imbibing potential to transform the treatment for rare diseases. With the advantage of single-dose treatment against rare diseases, gene therapy confers effective lifelong improvement as compared to conventional treatment options. Development of Gene therapy for rare diseases comprises a broad range of manufacturing technologies such as Plug-and-play manufacturing processes for AAV (adeno-associated virus) serotypes, Compendium of standard analytical and bioanalytical methods, and cell suspension, and Cell potentiation technology.
Gene Therapies for Rare Diseases is witnessing a swift adoption due to the growing number of pipelines for gene therapy and rising drug approvals and the introduction of novel gene therapies for the treatment of various rare diseases. Growing penetration of healthcare facilities across various geographic areas and augmented affordability for healthcare expenses have led to a rise in the volume of patients availing healthcare treatments. In addition to this, surging gene therapy innovations for rare diseases treatment is further expected to show significant opportunities during the forecast period.
The Global Gene Therapies for Rare Diseases Market is categorized on the basis of product, Disease Type and region. On the basis of product type, the market is segmented into Zolgensma, Invossa K, Strimvelis, Neovasculgen, Glybera, Luxturna, Zynteglo, and Others. On the basis of Disease Type, the market is segmented into Hemophilia, Duchenne Muscular Dystrophy, Achromatopsia, Cystic Fibrosis, Inherited Retinal Dystrophy, Fragile X Syndrome, Friedreich ataxia, Alpha-1 Antitrypsin Deficiency, Paroxysmal Nocturnal Hemoglobinuria, Retinitis Pigmentosa, and Others. Based on region, the market is studied across North America, Asia-Pacific, Europe, and LAMEA. Among all, North America is expected to dominate the market during the analysis of forecast period.
| Report Attribute | Specifications |
| Growth Rate CAGR | CAGR of 20.0 % from 2023 to 2031 |
| Quantitative Units | Representation of revenue in US$ Million and CAGR from 2023 to 2031 |
| Historic Year | 2019 to 2022 |
| Forecast Year | 2023-2031 |
| Report Coverage | The forecast of revenue, the position of the company, the competitive market structure, growth prospects, and trends |
| Segments Covered | By Product, By Disease Type |
| Regional Scope | North America; Europe; Asia Pacific; Latin America; Middle East & Africa |
| Country Scope | U.S.; Canada; U.K.; Germany; China; India; Japan; Brazil; Mexico; The UK; France; Italy; Spain; China; Japan; India; South Korea; South East Asia; South Korea; South East Asia |
| Competitive Landscape | 4d Molecular Therapeutics, LLC, Abeona Therapeutics Inc., Advaxis, Adverum Biotech, Aevi Genomic, Applied Genetic Technologies Corporation (AGTC), Alcyone Lifesciences, Allife Medical Science and Technology, Amarna Therapeutics, American Gene Technologies, Amgen, Amicus Therapeutics, Anchiano Therapeutics, AnGes MG, Apic Bio, Armata Pharmaceuticals, Arrowhead, Arthrogen, Asklepios, Astellas, AVROBIO, BCM Families Foundation, Beijing Northland, Benitec, Biogen, BioMarin, Biosidus, bluebird bio, Boryung Group, Brain Neurotherapeuticsy Bio, Celsion Corporation, Chiesi, CRISPR Therapeutics, CSL Behring, Daewoong Pharma, Dicerna, Editas Medicine, Eiger BioPharmaceuticals, Enzo Therapeutics, Esteve, Evox Therapeutics, Expression Therapeutics, Fibrocell, Flexion Therapeutics,, Fortress Biotech, Freeline Therapeutics, Gene Biotherapeutics, Genenta Science, GeneQuine, Genethon, GenSight, Gilead, Gradalis, GSK, Hanugen, Helixmith, Herantis, ID Pharma, Immusoft, Inovio, Intellia Therapeutics, Intrexon, J&J, Juventas, Kolon Life Science, Krystal Biotech, Locana, LogicBio Therapeutics, Lysogene, Medigene, MeiraGTx, Miltenyi Biotec, Molecular Templates, Momotaro-Gene, Neuralgene, Novartis, OncoSec, Orchard Therapeutics, Oxford Biomedica, Pfizer, Prevail Therapeutics, PTC Therapeutics, REGENXBIO, Renova Therapeutics, Reyon Pharmaceutical, Roche, Rocket Pharma, Roivant, Sangamo Therapeutics, Sarepta Therapeutics, Seelos Therapeutics, Solid BioSciences, Sterna Biologicals, Sutura Therapeutics, SynerGene, Takara, Takeda, Talee Bio, Theragene Pharma, Transgene, Ultragenyx, uniQure, VBL Therapeutics, Vertex, Voyager Therapeutics, Xalud, Xenon, and Ziopharm. |
| Customization Scope | Free customization report with the procurement of the report, Modifications to the regional and segment scope. Particular Geographic competitive landscape. |
| Pricing and Available Payment Methods | Explore pricing alternatives that are customized to your particular study requirements. |
Chapter 1. Methodology and Scope
1.1. Research Methodology
1.2. Research Scope & Assumptions
Chapter 2. Executive Summary
Chapter 3. Global Gene Therapies for Rare Diseases Market Snapshot
Chapter 4. Global Gene Therapies for Rare Diseases Market Variables, Trends & Scope
4.1. Market Segmentation & Scope
4.2. Drivers
4.3. Challenges
4.4. Trends
4.5. Penetration & Growth Prospect Mapping
4.6. Clinical Trial/Pipeline Analysis
4.7. Industry Analysis – Porter’s Five Forces Analysis
4.8. Competitive Landscape & Market Share Analysis
4.9. Technology Advancement in Gene Therapies for Rare Diseases Market
Chapter 5. Market Segmentation 1: Product Estimates & Trend Analysis
5.1. Product Type & Market Share, 2020 & 2028
5.2. Market Size (Value US$ Mn) & Forecasts and Trend Analyses, 2020 to 2028 for the following Product:
5.2.1. Zolgensma
5.2.2. Invossa K
5.2.3. Strimvelis
5.2.4. Neovasculgen
5.2.5. Glybera
5.2.6. Luxturna
5.2.7. Zynteglo
5.2.8. Others
Chapter 6. Gene Therapies for Rare Diseases Market Segmentation 2: Application Estimates & Trend Analysis
6.1. Disease Type Analysis & Market Share, 2020 & 2028
6.2. Market Size (Value US$ Mn) & Forecasts and Trend Analyses, 2020 to 2028 for the following Disease Type:
6.2.1. Human Medicine
6.2.2. Human Medicine
6.2.3. Hemophilia
6.2.4. Duchenne Muscular Dystrophy
6.2.5. Achromatopsia
6.2.6. Cystic Fibrosis
6.2.7. Inherited Retinal Dystrophy
6.2.8. Fragile X Syndrome
6.2.9. Friedreich ataxia
6.2.10. Alpha-1 Antitrypsin Deficiency
6.2.11. Paroxysmal Nocturnal Hemoglobinuria
6.2.12. Retinitis Pigmentosa
6.2.13. Others
Chapter 7. Gene Therapies for Rare Diseases Market Segmentation 3: Regional Estimates & Trend Analysis
7.1. North America
7.1.1. North America Gene Therapies for Rare Diseases Market revenue (US$ Million) estimates and forecasts by product, 2020-2028
7.1.2. North America Gene Therapies for Rare Diseases Market revenue (US$ Million) estimates and forecasts by Disease Type, 2020-2028
7.1.3. North America Gene Therapies for Rare Diseases Market revenue (US$ Million) estimates and forecasts by country, 2020-2028
7.1.3.1. U.S.
7.1.3.2. Canada
7.2. Europe
7.2.1. Europe Gene Therapies for Rare Diseases Market revenue (US$ Million) by product, 2020-2028
7.2.2. Europe Gene Therapies for Rare Diseases Market revenue (US$ Million) by Disease Type, 2020-2028
7.2.3. Europe Gene Therapies for Rare Diseases Market revenue (US$ Million) by country, 2020-2028
7.2.3.1. Germany
7.2.3.2. Poland
7.2.3.3. France
7.2.3.4. Italy
7.2.3.5. Spain
7.2.3.6. UK
7.2.3.7. Rest of Europe
7.3. Asia Pacific
7.3.1. Asia Pacific Gene Therapies for Rare Diseases Market revenue (US$ Million) by product, 2020-2028
7.3.2. Asia Pacific Gene Therapies for Rare Diseases Market revenue (US$ Million) by Disease Type, 2020-2028
7.3.3. Asia Pacific Gene Therapies for Rare Diseases Market revenue (US$ Million) by country, 2020-2028
7.3.3.1. China
7.3.3.2. India
7.3.3.3. Japan
7.3.3.4. Australia
7.3.3.5. Rest of Asia Pacific
7.4. Latin America
7.4.1. Latin America Gene Therapies for Rare Diseases Market revenue (US$ Million) by product, (US$ Million)
7.4.2. Latin America Gene Therapies for Rare Diseases Market revenue (US$ Million) by Disease Type, (US$ Million)
7.4.3. Latin America Gene Therapies for Rare Diseases Market revenue (US$ Million) by country, (US$ Million) 2020-2028
7.4.3.1. Brazil
7.4.3.2. Rest of Latin America
7.5. MEA
7.5.1. MEA revenue Gene Therapies for Rare Diseases Market revenue (US$ Million) by product, (US$ Million) 2020-2028
7.5.2. MEA Gene Therapies for Rare Diseases Market revenue (US$ Million) by Disease Type, (US$ Million)
7.5.3. MEA Gene Therapies for Rare Diseases Market revenue (US$ Million) by country, (US$ Million) 2020-2028
Chapter 8. Competitive Landscape
8.1. Company Profiles
8.1.1. 4d Molecular Therapeutics, LLC
8.1.2. Abeona Therapeutics Inc.
8.1.3. Advaxis
8.1.4. Adverum Biotech
8.1.5. Aevi Genomic
8.1.6. Applied Genetic Technologies Corporation (AGTC)
8.1.7. Alcyone Lifesciences
8.1.8. Allife Medical Science and Technology
8.1.9. Amarna Therapeutics
8.1.10. American Gene Technologies
8.1.11. Amgen
8.1.12. Amicus Therapeutics
8.1.13. Anchiano Therapeutics
8.1.14. AnGes MG
8.1.15. Apic Bio
8.1.16. Armata Pharmaceuticals
8.1.17. Arrowhead
8.1.18. Arthrogen
8.1.19. Asklepios
8.1.20. Astellas
8.1.21. AVROBIO
8.1.22. BCM Families Foundation
8.1.23. Beijing Northland
8.1.24. Benitec
8.1.25. Biogen
8.1.26. BioMarin
8.1.27. Biosidus
8.1.28. bluebird bio
8.1.29. Boryung Group
8.1.30. Brain Neurotherapeuticsy Bio
8.1.31. Celsion Corporation
8.1.32. Chiesi
8.1.33. CRISPR Therapeutics
8.1.34. CSL Behring
8.1.35. Daewoong Pharma
8.1.36. Dicerna
8.1.37. Editas Medicine
8.1.38. Eiger BioPharmaceuticals
8.1.39. Enzo Therapeutics
8.1.40. Esteve
8.1.41. Evox Therapeutics
8.1.42. Expression Therapeutics
8.1.43. Fibrocell
8.1.44. Flexion Therapeutics,
8.1.45. Fortress Biotech
8.1.46. Freeline Therapeutics
8.1.47. Gene Biotherapeutics
8.1.48. Genenta Science
8.1.49. GeneQuine
8.1.50. Genethon
8.1.51. GenSight
8.1.52. Gilead
8.1.53. Gradalis
8.1.54. GSK
8.1.55. Hanugen
8.1.56. Helixmith
8.1.57. Herantis
8.1.58. ID Pharma
8.1.59. Immusoft
8.1.60. Inovio
8.1.61. Intellia Therapeutics
8.1.62. Intrexon
8.1.63. Juventas
8.1.64. Kolon Life Science
8.1.65. Krystal Biotech
8.1.66. Locana
8.1.67. LogicBio Therapeutics
8.1.68. Lysogene
8.1.69. Medigene
8.1.70. MeiraGTx
8.1.71. Miltenyi Biotec
8.1.72. Molecular Templates
8.1.73. Momotaro-Gene
8.1.74. Neuralgene
8.1.75. Novartis
8.1.76. OncoSec
8.1.77. Orchard Therapeutics
8.1.78. Oxford Biomedica
8.1.79. Pfizer
8.1.80. Prevail Therapeutics
8.1.81. PTC Therapeutics
8.1.82. REGENXBIO
8.1.83. Renova Therapeutics
8.1.84. Reyon Pharmaceutical
8.1.85. Roche
8.1.86. Rocket Pharma
8.1.87. Roivant
8.1.88. Sangamo Therapeutics
8.1.89. Sarepta Therapeutics
8.1.90. Seelos Therapeutics
8.1.91. Solid BioSciences
8.1.92. Sterna Biologicals
8.1.93. Sutura Therapeutics
8.1.94. SynerGene
8.1.95. Takara
8.1.96. Takeda
8.1.97. Talee Bio
8.1.98. Theragene Pharma
8.1.99. Transgene
8.1.100. Ultragenyx
8.1.101. uniQure
8.1.102. VBL Therapeutics
8.1.103. Vertex
8.1.104. Voyager Therapeutics
8.1.105. Xalud
8.1.106. Xenon
8.1.107. Ziopharm
Global Gene Therapies for Rare Diseases Market Based on Product
Global Gene Therapies for Rare Diseases Market Based on Disease Type
Global Gene Therapies for Rare Diseases Market Based on Region
Europe Gene Therapies for Rare Diseases Market by Country
North America Gene Therapies for Rare Diseases Market by Country
Asia Pacific Gene Therapies for Rare Diseases Market by Country
Latin America Gene Therapies for Rare Diseases Market by Country
Middle East & Africa Gene Therapies for Rare Diseases Market by Country