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Cell and Gene Therapies for Rare Disorders Poised for Breakthrough Growth Through 2036, Fueled by Innovation, Expanding Pipelines and Unmet Patient Needs

globenewswire.com
CSL CSL Behring is recognized as a key company in the expanding cell and gene therapies market for rare disorders. Their collaboration with uniQure on HEMGENIX, a therapy for hemophilia B, positions them favorably. SANG Sangamo Therapeutics is listed as a key company in the rapidly growing cell and gene therapies market for rare disorders. The article points to continued investment and advancing candidates in this sector. CAPR Capricor Therapeutics is identified as a key company active in the cell and gene therapies for rare disorders market, which is expected to experience substantial growth. The article notes continued investment in this sector. SPRC Spark Therapeutics (a company of Roche) is mentioned for its therapy LUXTURNA, an approved product in the growing cell and gene therapy market for rare disorders. This indicates a positive market outlook for the company. NVS Novartis is mentioned in relation to LUXTURNA, which it commercializes. While LUXTURNA is a key therapy in the growing cell and gene therapy market, the article does not provide specific sentiment towards Novartis' overall performance or future prospects.

Cell and Gene Therapies for Rare Disorders Poised for Breakthrough Growth Through 2036, Fueled by Innovation, Expanding Pipelines and Unmet Patient Needs Dublin, Sept. 15, 2026 (GLOBE NEWSWIRE) -- "Cell and Gene Therapies in Rare Disorders - Market Insights, Epidemiology, and Market Forecast - 2036" has been added to ResearchAndMarkets.com's offering.

The global cell and gene therapies in rare disorders market is entering a period of substantial growth, supported by expanding clinical pipelines, rising regulatory approvals and continued advances in gene editing, vector delivery and cell engineering. More than 6,000 rare diseases have been identified, collectively affecting approximately 300 million people worldwide. Around 80% have a genetic origin, while nearly 70% begin during childhood, reinforcing the need for earlier diagnosis and targeted intervention.

Companies are developing cell and gene therapies for hemophilia A and B, lysosomal storage disorders, neurological diseases, musculoskeletal conditions and inherited retinal disorders. Key indications include Fabry disease, Pompe disease, Danon disease, mucopolysaccharidoses, Duchenne muscular dystrophy, limb-girdle muscular dystrophy, Batten disease, achromatopsia, choroideremia and retinitis pigmentosa.

These therapies offer the potential for durable disease modification, including one-time treatment in selected indications. Although high upfront costs, reimbursement uncertainty and complex manufacturing requirements remain significant barriers, successful therapies may reduce dependence on lifelong treatment and lower long-term healthcare utilization.

Cell and Gene Therapies in Rare Disorders Market Size and Forecast

The report, Cell and Gene Therapies in Rare Disorders - Market Insights, Epidemiology and Market Forecast - 2036, provides an assessment of historical and forecast epidemiology, treatment practices, market size, therapy uptake and competitive dynamics in the United States, Germany, France, Italy, Spain, the United Kingdom and Japan.

Its market analysis covers the 2022-2036 period and examines standards of care, treatment algorithms, diagnosed patient populations, revenue trends, therapy-level market share, peak patient share, pricing, access and unmet medical needs. It also evaluates clinical and commercial opportunities across marketed products and emerging cell and gene therapy candidates.

Key Cell and Gene Therapy Market Drivers

Growth is being supported by improved genetic screening, broader use of next-generation sequencing, increased rare disease awareness and stronger identification of patients with actionable molecular alterations. Advances in viral and non-viral vectors, CRISPR-based platforms, in vivo gene delivery and ex vivo cell modification are also broadening the range of addressable rare diseases.

Approved therapies such as HEMGENIX, LUXTURNA, ZOLGENSMA and ZYNTEGLO have established regulatory and commercial pathways for advanced therapies. Additional marketed products, including LIBMELDY and SKYSONA, further demonstrate the potential of gene replacement and ex vivo lentiviral approaches.

Despite this progress, gene and cell therapies carry clinical risks that vary by modality, vector and administration method. Long-term monitoring remains essential to assess durability, immune responses, vector-related safety and sustained gene expression or cellular function. Some scientifically viable programs also fail to advance because of limited commercial feasibility, manufacturing constraints or insufficient patient reach.

Diagnosis and Treatment Landscape

Rare disorder diagnosis frequently requires clinical assessment, laboratory testing, imaging and molecular confirmation. Next-generation sequencing, whole-exome sequencing and whole-genome sequencing increasingly support definitive diagnosis and patient stratification. Earlier identification is particularly important where treatment eligibility, disease stage or irreversible progression may influence therapeutic outcomes.

Cell and gene therapy selection depends on the molecular defect, target tissue, disease severity and delivery requirements. Treatment strategies include in vivo gene delivery, ex vivo cell modification, gene replacement, gene editing and cell-based regeneration. Post-treatment follow-up evaluates efficacy, immune response, adverse events and treatment durability.

Unmet Needs in Cell and Gene Therapies for Rare Disorders

Epidemiology Highlights

Approved and Emerging Rare Disease Therapies

HEMGENIX, developed by uniQure and commercialized by CSL Behring, is an AAV5-based gene therapy for eligible patients with hemophilia B. It delivers the Factor IX Padua variant to liver cells to support sustained production of functional factor IX and reduce abnormal bleeding.

The emerging competitive landscape includes isaralgagene civaparvovec, also known as ST-920, RGX-121 and Deramiocel, also known as CAP-1002. These programs reflect continued investment in lysosomal storage disorders, neuromuscular diseases and other genetically driven conditions.

RGX-121, or clemidsogene lanparvovec, is an investigational AAV9-based gene therapy developed by REGENXBIO for mucopolysaccharidosis type II. The program has received multiple FDA designations, including Orphan Drug, Rare Pediatric Disease, Fast Track and Regenerative Medicine Advanced Therapy designations, as well as Advanced Therapy Medicinal Product classification from the European Medicines Agency.

Recent Cell and Gene Therapy Developments

Competitive Landscape and Leading Companies

Key companies active in the cell and gene therapies in rare disorders market include Sarepta Therapeutics, uniQure, CSL Behring, Bluebird Bio, Sangamo Therapeutics, REGENXBIO, Capricor Therapeutics and Nippon Shinyaku. Competition is expected to intensify as mid- and late-stage candidates advance toward regulatory review and potential commercialization.

Market growth will depend on clinical differentiation, treatment durability, safety, manufacturing reliability, pricing and reimbursement. Regulatory setbacks, clinical holds, supply constraints and commercial underperformance demonstrate the challenges of translating scientific innovation into sustainable market adoption.

Therapy Uptake, Pricing and Market Access

Cell and gene therapy uptake is expected to range from moderate to rapid during the 2026-2036 forecast period. Adoption will be driven by clinical efficacy, durability, patient eligibility, treatment-center capacity and payer coverage. Recently marketed therapies have established demand in high-unmet-need indications, while emerging products may experience gradual initial uptake followed by broader adoption as clinical evidence and manufacturing capacity expand.

The report evaluates approved therapy pricing, analogue selection for emerging products and the effects of price on access and long-term uptake. It also incorporates views from key opinion leaders and subject matter experts regarding prescribing patterns, treatment switching, patient adherence, accessibility and epidemiology. More than 10 experts were contacted, with interviews conducted with over six experts across the seven major markets.

Report Coverage and Strategic Value

The analysis supports pharmaceutical companies, biotechnology developers, investors and healthcare stakeholders seeking to understand the evolving cell and gene therapies in rare disorders market. It provides actionable insights for clinical development, portfolio prioritization, market access, launch planning and competitive strategy across the seven major markets.

Market estimates, epidemiological figures and clinical or regulatory information are subject to revision as new data and report updates become available.

Key Topics Covered:

1. Key Insights

2. Report Introduction

3. Executive Summary

4. Key Events

4.1. Upcoming Key Catalysts

4.2. Key Transactions And Collaborations

4.3. Key Conference Highlights

4.4. News Flow

5. Epidemiology and Market Forecast Methodology of Cell and Gene Therapies in Rare Disorders

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

6.1. Clinical Landscape Analysis (By Phase, Molecule Type, and RoA)

6.2. Market Share (%) Distribution of Indication-wise Cell and Gene Therapies in Rare Disorders by Indication in the 7MM, in 2025

6.3. Market Share (%) Distribution of Indication-wise Cell and Gene Therapies in Rare Disorders Rare Disorders by Indication in the 7MM, in 2036

7. Disease Background and Overview on Cell and Gene Therapies in Rare Disorders

7.1. Introduction

7.2. Types

7.3. Symptoms

7.4. Causes

7.5. Pathophysiology

7.6. Diagnosis

7.7. Treatment

8. Treatment Guidelines of Cell and Gene Therapies in Rare Disorders

9. Epidemiology and Patient Population of Cell and Gene Therapies in Rare Disorders

9.1. Key Findings

9.2. Assumptions and Rationale

9.3. Total Prevalent Cases of selected indications for Cell and Gene Therapies Targeting Rare Disorders

in the 7MM

9.4. The United States

9.4.1. Total Prevalent Cases of Selected Indications for Cell and Gene Therapies in Rare Disorders in the US

9.4.2. Total Indication-wise Eligible Cases for Cell and Gene Therapies in Rare Disorders in the US

9.4.3. Total Indication-wise Treated Cases of Cell and Gene Therapies in Rare Disorders in the US

9.5. EU4 and the UK

9.5.1. Total Prevalent Cases of Selected Indications for Cell and Gene Therapies in Rare Disorders in EU4 and the UK

9.5.2. Total Indication-wise Eligible Cases for Cell and Gene Therapies in Rare Disorders in EU4 and the UK

9.5.3. Total Indication-wise Treated Cases of Cell and Gene Therapies in Rare Disorders in EU4 and the UK

9.6. Japan

9.6.1. Total Prevalent Cases of Selected Indications for Cell and Gene Therapies in Rare Disorders in Japan

9.6.2. Total Indication-wise Eligible Cases for Cell and Gene Therapies in Rare Disorders in Japan

9.6.3. Total Indication-wise Treated Cases of Cell and Gene Therapies in Rare Disorders in Japan

10. Patient Journey of Cell and Gene Therapies in Rare Disorders

11. Marketed Therapies

11.1. Marketed Competitive Landscape of Cell and Gene Therapies in Rare Disorders

11.2. Etranacogene dezaparvovec (HEMGENIX): CSL Behring/uniQure

11.2.1. Drug Description

11.2.2. Regulatory Milestones

11.2.3. Other Developmental Activities

11.2.4. Summary of Pivotal Trials

11.2.5. Clinical Development

11.2.5.1. Clinical Trial Information

11.2.6. Analyst Views

11.3. Voretigene neparvovec-rzyl (LUXTURNA): Sparks Therapeutics (a company of Roche)/Novartis

11.3.1. Drug Description

11.3.2. Regulatory Milestones

11.3.3. Other Developmental Activities

11.3.4. Summary of Pivotal Trials

11.3.5. Clinical Development

11.3.5.1. Clinical Trial Information

11.3.6. Analyst Views

List to be continued...

12. Emerging Therapies

12.1. Emerging Competitive Landscape of Cell and Gene Therapies in Rare Disorders

12.2. RGX-121: REGENXBIO

12.2.1. Drug Description

12.2.2. Other Developmental Activities

12.2.3. Clinical Development

12.2.3.1. Clinical Trials Information

12.2.4. Analyst Views

12.3. Isaralgagene civaparvovec (ST-920): Sangamo Therapeutics

12.3.1. Drug Description

12.3.2. Other Developmental Activity

12.3.3. Clinical Development

12.3.3.1. Clinical Trials Information

12.3.4. Analyst Views

List to be continued...

13. Cell and Gene Therapies in Rare Disorders: 7MM Analysis

13.1. Key Findings

13.2. Market Outlook of Cell and Gene Therapies in Rare Disorders

13.3. Key Market Forecast Assumptions

13.3.1. Cost Assumptions

13.3.2. Pricing Trends

13.3.3. Analogue Assessment

13.3.4. Launch Year and Therapy Uptakes

13.4. Conjoint Analysis of Cell and Gene Therapies in Rare Disorders

13.5. Total Market Size of Cell and Gene Therapies in Rare Disorders by Indications in the 7MM

13.6. Total Market Size of Cell and Gene Therapies in Rare Disorders by Therapies in the 7MM

13.7. The United States

13.7.1. Total Market Size of Cell and Gene Therapies in Rare Disorders by Indications in the United States

13.7.2. Total Market Size of Cell and Gene Therapies in Rare Disorders by Therapies in the United States

13.8. EU4 and the UK

13.8.1. Total Market Size of Cell and Gene Therapies in Rare Disorders by Indications in EU4 and the UK

13.8.2. Total Market Size of Cell and Gene Therapies in Rare Disorders by Therapies in EU4 and the UK

13.9. Japan

13.9.1. Total Market Size of Cell and Gene Therapies in Rare Disorders by Indications in Japan

13.9.2. Total Market Size of Cell and Gene Therapies in Rare Disorders by Therapies in Japan

14. Unmet Needs of Cell and Gene Therapies in Rare Disorders

15. SWOT Analysis of Cell and Gene Therapies in Rare Disorders

16. KOL Views of Cell and Gene Therapies in Rare Disorders

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

17.1. The United States

17.2. EU4 and the UK

17.2.1. Germany

17.2.2. France

17.2.3. Italy

17.2.4. Spain

17.2.5. United Kingdom

17.3. Japan

17.4. Summary and comparison of Market Access and Pricing Policy Developments in 2025

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

18. Appendix

18.1. Bibliography

18.2. Report Methodology

19. Analyst's Capabilities

20. Disclaimer

21. About the Publisher

List of Tables

Table 1: Summary of Cell and Gene Therapies in Rare Disorders Market and Epidemiology (2022-2036)

Table 2: List of Sources Used for Epidemiology Evaluation for Shortlisted Indication

Table 3: Total Prevalent Cases of Selected Indications for Cell and Gene Therapies in Rare Disorders in the 7MM (2022-2036)

Table 4: Total Indication-wise Eligible Cases in the 7MM (2022-2036)

Table 5: Total Indication-wise Treated Cases of Cell and Gene Therapies in Rare Disorders in the 7MM (2022-2036)

Table 6: Comparison of Marketed Products

Table 7: ROCTAVIAN (valoctocogene roxaparvovec); Clinical Trial Description, 2023

Table 8: HEMGENIX (etranacogene dezaparvovec), Clinical Trial Description, 2023

Table 9: LUXTURNA, Clinical Trial Description, 2023

Table 10: ZOLGENSMA, Clinical Trial Description, 2023

Table 11: LIBMELDY (OTL-200), Clinical Trial Description, 2023

Table 12: SKYSONA (elivaldogene autotemcel), Clinical Trial Description, 2023

Table 13: ZYNTEGLO, Clinical Trial Description, 2023

Table 14: UPSTAZA, Clinical Trial Description, 2023

Table 15: Key Cross of Emerging Therapies

Table 16: Giroctocogene Fitelparvovec, Clinical Trial Description, 2023

Table 17: SPK-8011, Clinical Trial Description, 2023

Table 18: Fidanacogene elaparvovec, Clinical Trial Description, 2023

Table 19: Verbrinacogene setparvovec (FLT-180a), Clinical Trial Description, 2023

Table 20: FLT190, Clinical Trial Description, 2023

Table 21: ST-920, Clinical Trial Description, 2023

Table 22: 4D-310, Clinical Trial Description, 2023

Table 23: SPK-3006, Clinical Trial Description, 2023

Table 24: AT845, Clinical Trial Description, 2023

Table 25: ACTUS-101, Clinical Trial Description, 2023

Table 26: LUMEVOQ (GS010; lenadogene nolparvovec), Clinical Trial Description, 2023

Table 27: AAV2/5-hPDE6B, Clinical Trial Description, 2023

Table 28: Botaretigene sparoparvovec, Clinical Trial Description, 2023

Table 29: ATGC-501, Clinical Trial Description, 2023

Table 30: GS030, Clinical Trial Description, 2023

Table 31: MCO-010, Clinical Trial Description, 2023

Table 32: 4D-125, Clinical Trial Description, 2023

Table 33: OCU400, Clinical Trial Description, 2023

Table 34: jCell, Clinical Trial Description, 2023

Table 35: hRPC, Clinical Trial Description, 2023

Table 36: RGX-121, Clinical Trial Description, 2023

Table 37: AT-GTX-502, Clinical Trial Description, 2023

Table 38: PF-06939926, Clinical Trial Description, 2023

Table 39: SRP-9001, Clinical Trial Description, 2023

Table 40: CAP-1002, Clinical Trial Description, 2023

Table 41: NurOwn, Clinical Trial Description, 2023

Table 42: CTX001, Clinical Trial Description, 2023

Table 43: EDIT-301, Clinical Trial Description, 2023

Table 44: BIVV003, Clinical Trial Description, 2023

Table 45: VYJUVEK (beremagene geperpavec), Clinical Trial Description, 2023

Table 46: EB-101, Clinical Trial Description, 2023

Table 47: D-Fi (dabocemagene autoficel), Clinical Trial Description, 2023

Table 48: RV-LAMB3-transduced Epidermal Stem Cells, Clinical Trial Description, 2023

Table 49: Allogenic ABCB5-positive Stem Cells, Clinical Trial Description, 2023

Table 50: ISN001, Clinical Trial Description, 2023

Table 51: ALLO-ASC-SHEET, Clinical Trial Description, 2023

Table 52: DTX301, Clinical Trial Description, 2023

Table 53: UX111(ABO-102), Clinical Trial Description, 2023

Table 54: DTX401 (AAV8G6PC); Clinical Trial Description, 2023

Table 55: Key Market Forecast Assumption of Cell and Gene Therapies in Rare Disorders in the US

Table 56: Key Market Forecast Assumption of Cell and Gene Therapies in Rare Disorders in EU4 and the UK

Table 57: Key Market Forecast Assumption of Cell and Gene Therapies in Rare Disorders in Japan

Table 58: Indication-wise Market Size of Cell and Gene Therapies in Rare Disorders in the 7MM, USD million (2022-2036)

Table 59: Market Size of Cell and Gene Therapies in Rare Disorders by Therapies in the 7MM, USD million (2022-2036)

Table 60: The United States Indication-wise Market Size of Cell and Gene Therapies in Rare Disorders, USD million (2022-2036)

Table 61: The United States Market Size of Cell and Gene Therapies in Rare Disorders by Therapies, USD million (2022-2036)

Table 62: Indication-wise Market Size of Cell and Gene Therapies in Rare Disorders in Germany, USD million (2022-2036)

Table 63: Indication-wise Market Size of Cell and Gene Therapies in Rare Disorders in France, USD million (2022-2036)

Table 64: Indication-wise Market Size of Cell and Gene Therapies in Rare Disorders in Italy, USD million (2022-2036)

Table 65: Indication-wise Market Size of Cell and Gene Therapies in Rare Disorders in Spain, USD million (2022-2036)

Table 66: Indication-wise Market Size of Cell and Gene Therapies in Rare Disorders in the UK, USD million (2022-2036)

Table 67: Indication-wise Market Size of Cell and Gene Therapies in Rare Disorders in EU4 and the UK, USD million (2022-2036)

Table 68: Market Size of Cell and Gene Therapies in Rare Disorders by Therapies in Germany, USD million (2022-2036)

Table 69: Market Size of Cell and Gene Therapies in Rare Disorders by Therapies in France, USD million (2022-2036)

Table 70: Market Size of Cell and Gene Therapies in Rare Disorders by Therapies in Italy, USD million (2022-2036)

Table 71: Market Size of Cell and Gene Therapies in Rare Disorders by Therapies in Spain, USD million (2022-2036)

Table 72: Market Size of Cell and Gene Therapies in Rare Disorders by Therapies in the UK, USD million (2022-2036)

Table 73: Market Size of Cell and Gene Therapies in Rare Disorders by Therapies in EU4 and the UK, USD million (2022-2036)

Table 74: Indication-wise Market Size of Cell and Gene Therapies in Rare Disorders in Japan, USD million (2022-2036)

Table 75: Market Size of Cell and Gene Therapies in Rare Disorders by Therapies in Japan, USD million (2022-2036)

List of Figures

Figure 1: Cell Therapy Process

Figure 2: Total Prevalent Cases of Selected Indications for Cell and Gene Therapies in Rare Disorders in the 7MM (2022-2036)

Figure 3: Total Indication-wise Eligible Cases in the 7MM (2022-2036)

Figure 4: Indication-wise Treated Cases of Cell and Gene Therapies in Rare Disorders in the 7MM (2022-2036)

Figure 5: Indication-wise Market size of Cell and Gene Therapies in Rare Disorders in the 7MM, USD million (2022-2036)

Figure 6: Market Size of Cell and Gene Therapies in Rare Disorders by Therapies in the 7MM, USD million (2022-2036)

Figure 7: Indication-wise Market Size of Cell and Gene Therapies in Rare Disorders in the United States, USD million (2022-2036)

Figure 8: Total Market Size of Cell and Gene Therapies in Rare Disorders in the United States by Therapy, USD million (2022-2036)

Figure 9: Indication-wise Market Size of Cell and Gene Therapies in Rare Disorders in EU4 and the UK, USD million (2022-2036)

Figure 10: Market Size of Cell and Gene Therapies in Rare Disorders by Therapies in EU4 and the UK, USD million (2022-2036)

Figure 11: Indication-wise Market Size of Cell and Gene Therapies in Rare Disorders in Japan, USD million (2022-2036)

Figure 12: Market Size of Cell and Gene Therapies in rare Disorders in Japan by Therapy, USD million (2022-2036)

A selection of companies mentioned in this report includes, but is not limited to:

For more information about this report visit https://www.researchandmarkets.com/r/9h8652

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