Global Gene Therapies For Rare Diseases Market

Report ID : 1091 | Published : 2021-07-23 | Pages: 140 | Format:

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 size is valued at US$ XX Million, and it is expected to reach US$ 11,441.6 Million in 2030, recording a promising CAGR of 21.7% during the period of 2019-2030. By region, in 2019, North America dominates the market with share of 85.3 % of global Gene Therapies for Rare Diseases market. US is the key market holding majority of market for Gene Therapies for Rare Diseases in the region.

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.

The key players of this market include 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.

 

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 Segmentation:

 

Global Gene Therapies for Rare Diseases Market Based on Product

  • Zolgensma
  • Invossa K
  • Strimvelis
  • Neovasculgen
  • Glybera
  • Luxturna
  • Zynteglo
  • Others

Global Gene Therapies for Rare Diseases Market Based on Disease Type

  • Hemophilia
  • Duchenne Muscular Dystrophy
  • Achromatopsia
  • Cystic Fibrosis
  • Inherited Retinal Dystrophy
  • Fragile X Syndrome
  • Friedreich ataxia
  • Alpha-1 Antitrypsin Deficiency
  • Paroxysmal Nocturnal Hemoglobinuria
  • Retinitis Pigmentosa
  • Others

Global Gene Therapies for Rare Diseases Market Based on Region

Europe Gene Therapies for Rare Diseases Market by Country

  • U.K.
  • Germany
  • France
  • Italy
  • Spain
  • Russia
  • Rest of Europe

North America Gene Therapies for Rare Diseases Market by Country

  • U.S.
  • Canada

Asia Pacific Gene Therapies for Rare Diseases Market by Country

  • India
  • China
  • Japan
  • South Korea
  • Australia & New Zealand

Latin America Gene Therapies for Rare Diseases Market by Country

  • Brazil
  • Mexico
  • Rest of Latin America

Middle East & Africa Gene Therapies for Rare Diseases Market by Country

  • GCC Countries
  • South Africa
  • Rest of Middle East & Africa

InsightAce Analytic follows a standard and comprehensive market research methodology focused on offering the most accurate and precise market insights. The methods followed for all our market research studies include three significant steps – primary research, secondary research, and data modeling and analysis - to derive the current market size and forecast it over the forecast period. In this study, these three steps were used iteratively to generate valid data points (minimum deviation), which were cross-validated through multiple approaches mentioned below in the data modeling section.

Through secondary research methods, information on the market under study, its peer, and the parent market was collected. This information was then entered into data models. The resulted data points and insights were then validated by primary participants.

Based on additional insights from these primary participants, more directional efforts were put into doing secondary research and optimize data models. This process was repeated till all data models used in the study produced similar results (with minimum deviation). This way, this iterative process was able to generate the most accurate market numbers and qualitative insights.

Secondary research

The secondary research sources that are typically mentioned to include, but are not limited to:

  • Company websites, financial reports, annual reports, investor presentations, broker reports, and SEC filings.
  • External and internal proprietary databases, regulatory databases, and relevant patent analysis
  • Statistical databases, National government documents, and market reports
  • Press releases, news articles, and webcasts specific to the companies operating in the market

The paid sources for secondary research like Factiva, OneSource, Hoovers, and Statista

Primary Research:

Primary research involves telephonic interviews, e-mail interactions, as well as face-to-face interviews for each market, category, segment, and subsegment across geographies

The contributors who typically take part in such a course include, but are not limited to: 

  • Industry participants: CEOs, CBO, CMO, VPs, marketing/ type managers, corporate strategy managers, and national sales managers, technical personnel, purchasing managers, resellers, and distributors.
  • Outside experts: Valuation experts, Investment bankers, research analysts specializing in specific markets
  • Key opinion leaders (KOLs) specializing in unique areas corresponding to various industry verticals
  • End-users: Vary mainly depending upon the market

Data Modeling and Analysis:

In the iterative process (mentioned above), data models received inputs from primary as well as secondary sources. But analysts working on these models were the key. They used their extensive knowledge and experience about industry and topic to make changes and fine-tuning these models as per the product/service under study.

The standard data models used while studying this market were the top-down and bottom-up approaches and the company shares analysis model. However, other methods were also used along with these – which were specific to the industry and product/service under study.

To know more about the research methodology used for this study, kindly contact us/click here.

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