Cell and Gene Therapies in Rare Disorders Market Segmentation:
- By Type of Therapy
- Cell Therapy
- Gene Therapy
- Gene-Modified Cell Therapy
- By Disease Indication
- Neurological Disorders (e.g., Spinal Muscular Atrophy, Parkinson's Disease)
- Hematological Disorders (e.g., Sickle Cell Disease, Hemophilia)
- Metabolic Disorders (e.g., Lysosomal Storage Disorders, Fabry Disease)
- Ophthalmological Disorders (e.g., Inherited Retinal Disorders)
- Immunological Disorders (e.g., Primary Immunodeficiencies, Severe Combined Immunodeficiency)
- Musculoskeletal Disorders (e.g., Duchenne Muscular Dystrophy)
- Others (e.g., Cystic Fibrosis, Huntington's Disease)
- By Vector Type
- Viral Vectors (e.g., Lentivirus, Adeno-Associated Virus)
- Non-Viral Vectors (e.g., Plasmid DNA, Oligonucleotides)
- By Cell Type
- Hematopoietic Stem Cells
- Mesenchymal Stem Cells
- Induced Pluripotent Stem Cells
- Others (e.g., T-cells, NK cells)
- By Route of Administration
- Intravenous
- Intramuscular
- Subretinal
- Others (e.g., Intranasal, Intrathecal)
- By End-User
- Hospitals
- Specialty Clinics
- Academic and Research Institutes
- Others (e.g., Biotechnology Companies, Contract Research Organizations)
- By Region
- North America
- U.S.
- Canada
- Europe
- Germany
- U.K.
- Spain
- France
- Italy
- Russia
- Rest of Europe
- Asia Pacific
- China
- India
- Japan
- Australia
- South Korea
- ASEAN
- Rest of Asia Pacific
- Latin America
- Brazil
- Argentina
- Mexico
- Rest of Latin America
- Middle East & Africa
- GCC Countries
- Israel
- South Africa
- North Africa
- Central Africa
- Rest of the Middle East
- North America
Frequently Asked Questions
The current market size of the Cell and Gene Therapies in Rare Disorders industry is USD 2.8 billion as of 2023.
The increasing prevalence of rare disorders, lack of effective treatment options, technological advancements in cell and gene therapies, rising investments in R&D activities, personalized medicine approach, and gene-editing technologies like CRISPR are contributing significantly to the growth of the Cell and Gene Therapies in Rare Disorders market.
The high costs of cell and gene therapies, regulatory and ethical concerns, challenges in manufacturing and scaling up, and limited understanding of disease mechanisms are the key factors hampering the growth of the Cell and Gene Therapies in Rare Disorders Market.
The gene therapy segment is the leading component segment in the Cell and Gene Therapies in Rare Disorders Market, providing innovative treatments for various rare genetic disorders.
Novartis AG, Gilead Sciences, Inc., Bluebird Bio, Inc., Spark Therapeutics, Inc., Amgen Inc., Sanofi, UniQure N.V., Orchard Therapeutics plc, Biogen Inc., and REGENXBIO Inc. are among the major players operating in the Cell and Gene Therapies in Rare Disorders Market.
The North America region is expected to lead the Cell and Gene Therapies in Rare Disorders Market, with a market size of USD 17.3 billion by 2031, growing at a CAGR of 25.6% from 2023 to 2031.
The increasing prevalence of rare disorders, lack of effective treatment options, technological advancements in cell and gene therapies, rising investments in R&D activities, personalized medicine approach, gene-editing technologies like CRISPR, and growing awareness about rare diseases are the key drivers of the Cell and Gene Therapies in Rare Disorders Market.