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.