Gene Therapies for Rare Diseases Market Gains Momentum with Increasing Prevalence of Genetic Disorders and Advances in Single-Dose Therapies

InsightAce Analytic Pvt. Ltd. announces the release of a market assessment report on the "Global Gene Therapies For Rare Diseases Market- by Product (Zolgensma, Invossa K, Strimvelis, Neovasculgen, Glybera, Luxturna, Zynteglo and Others), 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 and Others), Trends, Industry Competition Analysis, Revenue and Forecast To 2031."

The Gene Therapies For Rare Diseases Market is expected to grow at an 20.0 % CAGR during the forecast period for 2023-2031.

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Gene therapy plays a pivotal role in the treatment of rare diseases, offering targeted and specialized interventions by introducing functional genes into a patient's cells. Approximately 80% of rare diseases have a genetic origin, making gene therapy a promising approach for long-term and potentially curative outcomes. Unlike traditional therapies, gene therapy often provides significant and lasting benefits through single-dose administration. The development of gene therapies for rare conditions involves a variety of advanced manufacturing techniques, such as cell suspension systems, cell potentiation, and plug-and-play platforms for producing adeno-associated virus (AAV) serotypes.

The market for gene therapies targeting rare diseases is experiencing robust growth, driven by the increasing prevalence of these conditions and their associated burden on healthcare systems. Contributing factors include the expansion of gene therapy pipelines, a rise in regulatory approvals, and ongoing innovation in therapeutic development for a broad range of rare diseases. Enhanced affordability and improved access to healthcare services across diverse regions have also led to a greater number of individuals seeking medical treatment.

Furthermore, rapid advancements in gene therapy technologies are expected to unlock substantial opportunities over the forecast period. Continued emphasis on research and the introduction of novel therapeutics are further elevating the market's value. Additionally, supportive government initiatives aimed at increasing awareness and promoting the adoption of gene therapies are accelerating the integration of these treatments into mainstream healthcare for rare disease management.

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North America is anticipated to contribute to the Gene Therapies For Rare Diseases market. The market for gene therapies for rare diseases is expanding due to increased healthcare spending, which has fueled the growth of research organizations in the area. Growing numbers of regional government initiatives are also influencing industry growth tendencies. In addition, the Asia Pacific Gene Therapies For Rare Diseases market is expected to register significant growth due to the country's expanding biomedical and regenerative medicine research. The establishment has accelerated the discovery of novel medicines for the treatment of rare diseases by the Japanese government of support networks and special regulatory authorities.

Major market players operating in the Gene Therapies For Rare Diseases 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.

Recent collaborations and agreements in the market:
• In August 2019, Gaboxadol was developed, manufactured, and made widely available in the European market by the U.S. company Ovid Therapeutics in collaboration with the Italian company Angelini Pharma to treat Angelman condition, an uncommon hereditary illness.

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Market Segments
Global Gene Therapies For Rare Diseases Market, by Product, 2022-2030 (Value US$ Mn)
• Zolgensma
• Invossa K
• Strimvelis
• Neovasculgen
• Glybera
• Luxturna
• Zynteglo
• Others
Global Gene Therapies For Rare Diseases Market, by Disease Type, 2022-2030 (Value US$ Mn)
• 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, by Region, 2022-2030 (Value US$ Mn)
• North America
• Europe
• Asia Pacific
• Latin America
• Middle East & Africa
North America Gene Therapies For Rare Diseases Market, by country, 2022-2030 (Value US$ Mn)
• U.S.
• Canada
Europe Gene Therapies For Rare Diseases Market, by Country, 2022-2030 (Value US$ Mn)
• Germany
• France
• Italy
• Spain
• Russia
• Rest of Europe
Asia Pacific Gene Therapies For Rare Diseases Market, by country, 2022-2030 (Value US$ Mn)
• India
• China
• Japan
• South Korea
• Australia & New Zealand
Latin America Gene Therapies For Rare Diseases Market, by country, 2022-2030 (Value US$ Mn)
• Brazil
• Mexico
• Rest of Latin America
Middle East & Africa Gene Therapies For Rare Diseases Market, by country, 2022-2030 (Value US$ Mn)
• GCC Countries
• South Africa
• Rest of Middle East & Africa

Why should buy this report:
 To receive a comprehensive analysis of the prospects for the global Gene Therapies For Rare Diseases market
 To receive an industry overview and future trends of the Gene Therapies For Rare Diseases market
 To analyze the Gene Therapies For Rare Diseases market drivers and challenges
 To get information on the Gene Therapies For Rare Diseases market value (US$ Mn) forecast to 2030
 Significant investments, mergers & acquisitions in the Gene Therapies For Rare Diseases market industry

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