Cell and Gene Therapies in Rare Disorders Market to Evolve Rapidly Over the Next Decade by 2034, DelveInsight Observes | REGENXBIO, Coave Therapeutics, GenSight Biologics, Ultragenyx, Pfizer, Sangamo

The Key Cell and Gene Therapies in Rare Disorders Companies in the market include - 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.

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.

To Know in detail about the Cell and Gene Therapies in Rare Disorders market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Cell and Gene Therapies in Rare Disorders Market Forecast [https://www.delveinsight.com/sample-request/gene-and-cell-therapies-in-rare-disorder-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]

Some of the key facts of the Cell and Gene Therapies in Rare Disorders Market Report:

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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).

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In August 2025, The U.S. Food and Drug Administration (FDA) authorized Rocket Pharmaceuticals to resume its pivotal Phase II trial of the gene therapy RP-A501 for Danon disease. This approval comes after a previous clinical hold was placed following a patient's death caused by an acute systemic infection resulting from treatment-induced capillary leak syndrome. Another patient experienced similar symptoms but subsequently recovered.

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In July 2025, Charles River Laboratories International, Inc. (NYSE: CRL) and Elly's Team, a parent-led foundation dedicated to finding a cure for Neurodevelopmental Disorder with Regression, Abnormal Movements, Loss of Speech, and Seizures (NEDAMSS), a rare genetic condition, have announced a plasmid DNA contract development and manufacturing organization (CDMO) partnership. Through Charles River's Cell and Gene Therapy (CGT) Accelerator Program (CAP), Elly's Team gained access to established gene therapy CDMO capabilities and expert advisory services to produce essential starting materials for a Phase I clinical trial.

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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.

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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.

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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.

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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).

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By 2034, Hemophilia A is projected to generate the highest revenue among all indications in the US, followed by Retinitis Pigmentosa.

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In 2023, ZOLGENSMA (onasemnogene abeparvovec-xioi) held the largest market share among therapies in the US.

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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.

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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.

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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).

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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

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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

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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:

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Total Prevalence of Cell and Gene Therapies in Rare Disorders

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Prevalent Cases of Cell and Gene Therapies in Rare Disorders by severity

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Gender-specific Prevalence of Cell and Gene Therapies in Rare Disorders

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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

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Fidanacogene elaparvovec: Pfizer

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GiroctocogenE fitelparvovec: Pfizer/ Sangamo Therapeutics

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OTL-103: Orchard Therapeutics/ SR-Tiget

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OAV101: Novartis

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TVI-Brain-1: TVAX Biomedical

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AV-GBM-1: Aivita Biomedical

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ABCB5+ MSCs: RHEACELL GmbH & Co

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CAP-1002: Capricor Therapeutics

Discover more about therapies set to grab major Cell and Gene Therapies in Rare Disorders market share @ Cell and Gene Therapies in Rare Disorders Treatment Market [https://www.delveinsight.com/sample-request/gene-and-cell-therapies-in-rare-disorder-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]

Scope of the Cell and Gene Therapies in Rare Disorders Market Report

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Study Period: 2020-2034

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Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

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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

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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

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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

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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

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Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies

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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

To know more about Cell and Gene Therapies in Rare Disorders companies working in the treatment market, visit @ Cell and Gene Therapies in Rare Disorders Clinical Trials and Therapeutic Assessment [https://www.delveinsight.com/sample-request/gene-and-cell-therapies-in-rare-disorder-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]

Table of Contents

1. Cell and Gene Therapies in Rare Disorders Market Report Introduction

2. Executive Summary for Cell and Gene Therapies in Rare Disorders

3. SWOT analysis of Cell and Gene Therapies in Rare Disorders

4. Cell and Gene Therapies in Rare Disorders Patient Share (%) Overview at a Glance

5. Cell and Gene Therapies in Rare Disorders Market Overview at a Glance

6. Cell and Gene Therapies in Rare Disorders Disease Background and Overview

7. Cell and Gene Therapies in Rare Disorders Epidemiology and Patient Population

8. Country-Specific Patient Population of Cell and Gene Therapies in Rare Disorders

9. Cell and Gene Therapies in Rare Disorders Current Treatment and Medical Practices

10. Cell and Gene Therapies in Rare Disorders Unmet Needs

11. Cell and Gene Therapies in Rare Disorders Emerging Therapies

12. Cell and Gene Therapies in Rare Disorders Market Outlook

13. Country-Wise Cell and Gene Therapies in Rare Disorders Market Analysis (2020-2034)

14. Cell and Gene Therapies in Rare Disorders Market Access and Reimbursement of Therapies

15. Cell and Gene Therapies in Rare Disorders Market Drivers

16. Cell and Gene Therapies in Rare Disorders Market Barriers

17. Cell and Gene Therapies in Rare Disorders Appendix

18. Cell and Gene Therapies in Rare Disorders Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

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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