Cell and Gene Therapies in Rare Disorders Market Dynamics Indicate Upward Trajectory Through 2034, Reports DelveInsight

DelveInsight's "Cell and Gene Therapies in Rare Disorders Market Insights, Epidemiology, and Market Forecast-2034′′ report offers an in-depth understanding of the Cell and Gene Therapies in Rare Disorders, historical and forecasted epidemiology as well as the Cell and Gene Therapies in Rare Disorders market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.

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Some of the key facts of the Cell and Gene Therapies in Rare Disorders Market Report:
• The Cell and Gene Therapies in Rare Disorders market size was valued ~USD 2,000 million in 2023 and is anticipated to grow with a significant CAGR during the study period (2020-2034).
• In February 2025, Elixirgen Therapeutics' investigational therapy for genetic disorders linked to bone marrow failure has shown the ability to lengthen chromosome caps in two patients with telomere biology disorders (TBD). Early findings from the ongoing Phase I/II trial of EXG-34217 (NCT04211714) demonstrated successful extension of the protective chemical structures on chromosomes in these patients.
• In December 2024, EyeDNA Therapeutics, a subsidiary of Coave Therapeutics, has received Rare Pediatric Disease (RPD) Designation from the U.S. FDA for its investigational gene therapy, HORA-PDE6b, intended to treat retinal dystrophy caused by mutations in the PDE6b gene.
• In February 2024, REGENXBIO reported topline results from its pivotal phase trial of RGX-121 for Hunter syndrome, highlighting that the study successfully achieved its primary endpoints with statistical significance.
• In 2023, the market size for cell and gene therapies targeting rare disorders in the US was around USD 800 million and is expected to grow throughout the forecast period (2024-2034).
• By 2034, Hemophilia A is projected to generate the highest revenue among all indications in the US, followed by Retinitis Pigmentosa.
• In 2023, ZOLGENSMA (onasemnogene abeparvovec-xioi) held the largest market share among therapies in the US.
• In 2023, the total prevalent cases of selected indications for Cell and Gene Therapies in Rare Disorders across the 7MM were approximately 930,200, with projections indicating an increase during the forecast period.
• In 2023, Retinitis Pigmentosa accounted for the highest number of cases among the rare indications selected for Cell and Gene Therapies, followed by Limbal Stem Cell Deficiency.
• In 2023, the total treated cases for cell and gene therapies across selected indications in the 7MM were approximately 504,000, with numbers projected to rise throughout the forecast period (2024-2034).
• Key Cell and Gene Therapies in Rare Disorders Companies: REGENXBIO, Coave Therapeutics, GenSight Biologics, Ultragenyx, Pfizer, Sangamo Therapeutics, Roche, 4D Molecular Therapeutics, Astellas Gene Therapie, Actus Therapeutics, Nanoscope Therapeutics, Ocugen, jCyte, Amicus Therapeutics, Capricor Therapeutics, Nippon Shinyaku, Brainstorm Cell Therapeutics, Editas Medicine, Abeona Therapeutics, Ishin Pharma, and others
• Key Cell and Gene Therapies in Rare Disorders Therapies: RGX-121, Isaralgagene civaparvovec (ST-920), CTx-PDE6b (HORA-PDE6b), GS030, DTX301, Giroctocogene fitelparvovec, Dirloctocogene samoparvovec, 4D-310, AT845, ACTUS-101, MCO-010 (sonpiretigene isteparvovec), OCU400, jCell, AT-GTX-502 (scAAV9.P546.CLN3), CAP-1002, NurOwn (MSC-NTF cells), EDIT-301, EB-101, ISN001, UX111 (ABO-102), and others
• The Cell and Gene Therapies in Rare Disorders market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Cell and Gene Therapies in Rare Disorders pipeline products will significantly revolutionize the Cell and Gene Therapies in Rare Disorders market dynamics.

Cell and Gene Therapies in Rare Disorders Overview
Cell and gene therapies in rare disorders are advanced treatments designed to target the root genetic or cellular causes of disease. They work by repairing, replacing, or modifying defective genes or cells, offering long-term or potentially curative outcomes for patients with limited treatment options.

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Cell and Gene Therapies in Rare Disorders Epidemiology
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.

Cell and Gene Therapies in Rare Disorders Epidemiology Segmentation:
The Cell and Gene Therapies in Rare Disorders market report proffers epidemiological analysis for the study period 2020-2034 in the 7MM segmented into:
• Total Prevalence of Cell and Gene Therapies in Rare Disorders
• Prevalent Cases of Cell and Gene Therapies in Rare Disorders by severity
• Gender-specific Prevalence of Cell and Gene Therapies in Rare Disorders
• Diagnosed Cases of Episodic and Chronic Cell and Gene Therapies in Rare Disorders

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Cell and Gene Therapies in Rare Disorders Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Cell and Gene Therapies in Rare Disorders market or expected to get launched during the study period. The analysis covers Cell and Gene Therapies in Rare Disorders market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.
The report also covers the Cell and Gene Therapies in Rare Disorders Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Cell and Gene Therapies in Rare Disorders Therapies and Key Companies
• Fidanacogene elaparvovec: Pfizer
• GiroctocogenE fitelparvovec: Pfizer/ Sangamo Therapeutics
• OTL-103: Orchard Therapeutics/ SR-Tiget
• OAV101: Novartis
• TVI-Brain-1: TVAX Biomedical
• AV-GBM-1: Aivita Biomedical
• ABCB5+ MSCs: RHEACELL GmbH & Co
• CAP-1002: Capricor Therapeutics

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Scope of the Cell and Gene Therapies in Rare Disorders Market Report
• Study Period: 2020-2034
• Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
• Key Cell and Gene Therapies in Rare Disorders Companies: REGENXBIO, Coave Therapeutics, GenSight Biologics, Ultragenyx, Pfizer, Sangamo Therapeutics, Roche, 4D Molecular Therapeutics, Astellas Gene Therapie, Actus Therapeutics, Nanoscope Therapeutics, Ocugen, jCyte, Amicus Therapeutics, Capricor Therapeutics, Nippon Shinyaku, Brainstorm Cell Therapeutics, Editas Medicine, Abeona Therapeutics, Ishin Pharma, and others
• Key Cell and Gene Therapies in Rare Disorders Therapies: RGX-121, Isaralgagene civaparvovec (ST-920), CTx-PDE6b (HORA-PDE6b), GS030, DTX301, Giroctocogene fitelparvovec, Dirloctocogene samoparvovec, 4D-310, AT845, ACTUS-101, MCO-010 (sonpiretigene isteparvovec), OCU400, jCell, AT-GTX-502 (scAAV9.P546.CLN3), CAP-1002, NurOwn (MSC-NTF cells), EDIT-301, EB-101, ISN001, UX111 (ABO-102), and others
• Cell and Gene Therapies in Rare Disorders Therapeutic Assessment: Cell and Gene Therapies in Rare Disorders current marketed and Cell and Gene Therapies in Rare Disorders emerging therapies
• Cell and Gene Therapies in Rare Disorders Market Dynamics: Cell and Gene Therapies in Rare Disorders market drivers and Cell and Gene Therapies in Rare Disorders market barriers
• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
• Cell and Gene Therapies in Rare Disorders Unmet Needs, KOL's views, Analyst's views, Cell and Gene Therapies in Rare Disorders Market Access and Reimbursement

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About DelveInsight
DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.
It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.

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