DelveInsight’s comprehensive market research provides critical insights into such market trends, enabling stakeholders to understand growth drivers, emerging opportunities, and potential challenges within the Sickle Cell Disease landscape.

DelveInsight’s report “Sickle Cell Disease Market Insights, Epidemiology, and Market Forecast-2034,” provides a comprehensive analysis of the Sickle Cell Disease landscape. The report delivers detailed insights into the disease, including historical and projected epidemiology, helping stakeholders understand the prevalence, incidence, and patient demographics across key regions.

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Some of the key facts of the Sickle Cell Disease Market Report:

• Across the 6MM countries, the Sickle Cell Disease market was valued at approximately USD 650 million in 2023 and is projected to grow steadily throughout the forecast period (2024–2034).

• The US accounts for the largest market size of Sickle Cell Disease in 2024, i.e., about 87% in comparison to EU4 (Germany, Italy, France, and Spain) and the UK.

• According to DelveInsight’s estimates, between EU4 and the UK, France accounted for the largest market of Sickle Cell Disease, while Spain accounted for the least market size.

• In August 2025, Pfizer is reevaluating its strategy after its investigational sickle cell disease therapy did not meet the primary endpoint in the Phase III THRIVE-131 trial (NCT04935879). The study, which assessed the P-selectin inhibitor inclacumab in patients aged 16 and older with sickle cell disease, failed to show a significant reduction in vaso-occlusive crises (VOCs) over 48 weeks, raising uncertainty about the drug’s future development.

• In June 2025, Ellarity has begun dosing the first participant in its Phase I clinical trial of CLY-124, an oral drug candidate for treating sickle cell disease (SCD). This milestone follows the US FDA’s approval of the company’s investigational new drug (IND) application. The global study aims to evaluate the safety, tolerability, and pharmacokinetics of the therapy, initially in healthy volunteers and later in patients with SCD.

• In June 2025, The US FDA has granted a special designation to Vascarta’s lead candidate, VAS-101 (Vasceptor), for treating sickle cell disease (SCD). This designation provides benefits such as seven years of market exclusivity post-approval and exemptions from certain FDA fees. VAS-101 is a patented topical curcumin formulation developed using Vascarta’s transdermal delivery technology to overcome the poor bioavailability and limited efficacy of oral curcumin. Preclinical animal studies suggest that VAS-101 may help reduce chronic pain, enhance red blood cell stability, and lower inflammation in patients with SCD.

• In May 2025, Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company focused on developing precision genetic medicines through base editing, announced that it will present new data from its BEACON Phase 1/2 clinical trial of BEAM-101 at the upcoming European Hematology Association (EHA) 2025 Congress, taking place from June 12–15, 2025, in Milan, Italy. BEAM-101 is an investigational ex vivo genetically modified cell therapy designed to treat sickle cell disease (SCD), particularly in patients suffering from severe vaso-occlusive crises (VOCs).

• In December 2024, Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company focused on precision genetic medicines through base editing, reported new safety and efficacy results from its BEACON Phase 1/2 trial evaluating BEAM-101 in patients with sickle cell disease (SCD) suffering from severe vaso-occlusive crises (VOCs). The findings were featured in the press program at the 66th American Society of Hematology (ASH) Annual Meeting.

• In November 2024, BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a commercial-stage biopharmaceutical company specializing in oncology and rare diseases, announced that an abstract presenting initial data from its Phase 1 trial of motixafortide evaluated alone and in combination with natalizumab for CD34+ hematopoietic stem cell (HSC) mobilization in gene therapies for sickle cell disease (SCD) has been selected for oral presentation at the 66th American Society of Hematology (ASH) Annual Meeting & Exposition, scheduled for December 7–10, 2024, in San Diego, California. Conducted in partnership with Washington University School of Medicine in St. Louis, the study aims to identify alternative HSC mobilization strategies to improve the gene therapy experience for SCD patients

• All batches of OXBRYTA, used for treating Sickle Cell Disease (SCD), were withdrawn from all markets in September 2024. Among the available therapies, CASGEVY and LYFGENIA are anticipated to emerge as market leaders, significantly shaping the Sickle Cell Disease landscape across the 6MM by 2034.

• According to estimates, the United States represented about 73% of the total diagnosed prevalent cases of Sickle Cell Disease (SCD) across the 6MM.

• DelveInsight’s 2024 analysis indicated that the total prevalent SCD cases in the 6MM were approximately 178,500, a figure projected to rise by 2034. Within the EU4 and the UK, France recorded the highest prevalence, with around 26,700 cases in 2024.

• According to the analysis, in the United States, individuals aged 18–44 years represented the largest proportion of Sickle Cell Disease (SCD) cases, followed by those aged 45–64 years.

• In France, sickle cell anemia (hemoglobin S/S) was the most prevalent SCD subtype in 2024, accounting for approximately 17,000 cases.

• Currently, NSAIDs, blood transfusions, chelating agents, nutritional supplements, and broad-spectrum antibiotics are being used for the treatment of Sickle Cell Disease .

• Established therapies in the Sickle Cell Disease market include DROXIA (hydroxyurea), ENDARI (L-glutamine oral powder), ADAKVEO (crizanlizumab-tmca), and others.

• Gene therapy and gene editing techniques hold promise in correcting the underlying genetic mutation responsible for Sickle Cell Disease. Recent FDA approvals of two gene therapies, Vertex/CRISPR’s CASGEVY and Bluebird’s LYFGENIA offers the potential of a one-time transformative therapy for eligible patients with Sickle Cell Disease.

• Key Sickle Cell Disease Companies: BRL Medicine, Oryzon Genomics, GlaxoSmithKline, Agios Pharmaceuticals, Beam Therapeutics Inc., Graphite Bio, Roche, Quercis Pharma, Editas Medicin, Pfizer, Emmaus Medical, Inc, Vertex Pharmaceuticals, CRISPR therapeutics, Bluebird Bio, Pfizer, Novo Nordisk, Agios Pharmaceuticals, Alexion Pharmaceuticals, Takeda, Prolong Pharmaceuticals, Roche, Beam Therapeutics, Editas Medicine, Sangamo Therapeutics, Bellicum Pharmaceuticals, Invenux, EpiDestiny, Hillhurst Biopharmaceuticals, CSL Behring, Fulcrum Therapeutics, Sana Biotechnology, and others

• Key Sickle Cell Disease Therapies: RL 101, ORY-300, GSK 4172239D, AG-946, BEAM-101, Nula-cel, RG 6107, Isoquercetin, Renizgamglogene autogedtemcel, Inclacumab, L-glutamine, CASGEVY, CTX001, LentiGlobin BB305, Inclacumab, Etavopivat, Mitapivat, ALXN1820, TAK-755, Sanguinate, Crovalimab, BEAM101, EDIT 301, BIVV003, BPX-501, SCD-101, Nicotinamide, HBI-002, CSL889, FTX-6058, SG418n, and others

• The Sickle Cell Disease epidemiology based on gender analyzed that Sickle Cell Disease affects males and females equally

• The Sickle Cell Disease market is expected to surge due to the disease’s increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Sickle Cell Disease pipeline products will significantly revolutionize the Sickle Cell Disease market dynamics.

Sickle Cell Disease Overview

Sickle Cell Disease is a hereditary blood disorder caused by a mutation in the gene that produces hemoglobin, the protein in red blood cells responsible for carrying oxygen. This mutation leads to the formation of abnormally shaped, rigid “sickle” red blood cells that can block blood flow, causing pain episodes known as vaso-occlusive crises, anemia, organ damage, and increased risk of infections. SCD is typically inherited from both parents and affects millions worldwide, with symptoms ranging from mild to severe. Management focuses on preventing complications, reducing pain, and improving quality of life.

To Know in detail about the Sickle Cell Disease market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Sickle Cell Disease Market Forecast

Key Trends in Sickle Cell Disease Therapeutics Market:

• Emergence of Gene Therapy and Gene Editing: Growing focus on CRISPR-based and lentiviral therapies targeting the root genetic cause of SCD.

• Advancements in Disease-Modifying Drugs: Increased development of therapies like voxelotor, crizanlizumab, and hydroxyurea alternatives to improve hemoglobin function and reduce complications.

• Expansion of Personalized Medicine Approaches: Use of genetic profiling and biomarkers to tailor treatment plans for individual patients.

• Rising Clinical Trials and Research Initiatives: Surge in global clinical studies exploring novel therapies, combination treatments, and long-term safety.

• Integration of Supportive and Adjunct Therapies: Focus on improving patient quality of life with pain management, anti-inflammatory agents, and improved care protocols.

Sickle Cell Disease Epidemiology

The Sickle Cell Disease 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.

Sickle Cell Disease Epidemiology Segmentation:

The Sickle Cell Disease market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:

• Total Diagnosed Cases of Sickle Cell Disease in the US

• Age-specific Diagnosed Cases of Sickle Cell Disease in the US

• Type-specific Diagnosed Cases of Sickle Cell Disease in the US

Download the report to understand which factors are driving Sickle Cell Disease epidemiology trends @ Sickle Cell Disease Epidemiology Forecast

Sickle Cell Disease Drugs Uptake and Pipeline Development Activities

The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Sickle Cell Disease market or expected to get launched during the study period. The analysis covers Sickle Cell Disease 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 Sickle Cell Disease 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.

Sickle Cell Disease Therapies and Key Companies

• RL 101: BRL Medicine

• ORY-300:1 Oryzon Genomics

• GSK 4172239D: GlaxoSmithKline

• AG-946: Agios Pharmaceuticals

• BEAM-101: Beam Therapeutics Inc.

• Nula-cel: Graphite Bio

• RG 6107: Roche

• Isoquercetin: Quercis Pharma

• Renizgamglogene autogedtemcel: Editas Medicin

• Inclacumab: Pfizer

• L-glutamine: Emmaus Medical, Inc

• CASGEVY: Vertex Pharmaceuticals

• ESCAPE: Beam Therapeutic

• IHP-102: IHP Therapeutics

• HBI-002: Hillhurst Biopharmaceuticals

• BEAM101: Beam Therapeutics

• EPI01: Novo Nordisk

• VIT-2763: CSL Vifor

• Inclacumab: Pfizer

• L-glutamine: Emmaus Medical

• Oxbryta: Pfizer

• Exagamglogene autotemcel: CRISPR Therapeutics/Vertex Pharmaceuticals

• Mitapivat: Agios Pharmaceuticals

• Canakinumab: Novartis

• ALXN1820: Alexion Pharmaceuticals

• Crovalimab: Chugai Pharmaceutical/Roche

• EDIT 301: Editas Medicine

• BIVV003: Sangamo Therapeutics

• BEAM101: Beam Therapeutics

• Hemopexin: CSL Behring

Discover more about therapies set to grab major Sickle Cell Disease market share @ Sickle Cell Disease Treatment Landscape

Sickle Cell Disease Market Drivers

• Rising Global Prevalence: Increasing incidence of SCD, especially in regions like Africa, the Middle East, and the US, drives market demand.

• Advancements in Gene Therapy and Gene Editing: Emerging therapies such as CRISPR and lentiviral-based treatments offer potential curative options.

• Introduction of Disease-Modifying Drugs: Approval of therapies like voxelotor and crizanlizumab improves patient outcomes and quality of life.

• Growing Awareness and Screening Programs: Enhanced newborn screening and public health initiatives promote early diagnosis and treatment.

• Rising Investment in R&D: Pharmaceutical and biotech companies are actively developing novel therapies and clinical trials.

Sickle Cell Disease Market Barriers

• High Treatment Costs: Advanced therapies and gene-based treatments are expensive, limiting accessibility.

• Limited Access in Low-Income Regions: Many patients in developing countries lack access to diagnostics and advanced care.

• Complexity of Treatment: Multi-drug regimens, transfusions, and long-term monitoring pose adherence challenges.

• Regulatory and Reimbursement Challenges: Approval and insurance coverage for novel therapies can be time-consuming and restrictive.

• Risk of Side Effects and Safety Concerns: Long-term use of certain therapies, including gene-editing approaches, may present safety risks.

Scope of the Sickle Cell Disease Market Report

• Study Period: 2020–2034

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

• Key Sickle Cell Disease Companies: BRL Medicine, Oryzon Genomics, GlaxoSmithKline, Agios Pharmaceuticals, Beam Therapeutics Inc., Graphite Bio, Roche, Quercis Pharma, Editas Medicin, Pfizer, Emmaus Medical, Inc, Vertex Pharmaceuticals, CRISPR therapeutics, Bluebird Bio, Pfizer, Novo Nordisk, Agios Pharmaceuticals, Alexion Pharmaceuticals, Takeda, Prolong Pharmaceuticals, Roche, Beam Therapeutics, Editas Medicine, Sangamo Therapeutics, Bellicum Pharmaceuticals, Invenux, EpiDestiny, Hillhurst Biopharmaceuticals, CSL Behring, Fulcrum Therapeutics, Sana Biotechnology, and others

• Key Sickle Cell Disease Therapies: RL 101, ORY-300, GSK 4172239D, AG-946, BEAM-101, Nula-cel, RG 6107, Isoquercetin, Renizgamglogene autogedtemcel, Inclacumab, L-glutamine, CASGEVY, CTX001, LentiGlobin BB305, Inclacumab, Etavopivat, Mitapivat, ALXN1820, TAK-755, Sanguinate, Crovalimab, BEAM101, EDIT 301, BIVV003, BPX-501, SCD-101, Nicotinamide, HBI-002, CSL889, FTX-6058, SG418, and others

• Sickle Cell Disease Therapeutic Assessment: Sickle Cell Disease current marketed and Sickle Cell Disease emerging therapies

• Sickle Cell Disease Market Dynamics: Sickle Cell Disease market drivers and Sickle Cell Disease market barriers

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

• Sickle Cell Disease Unmet Needs, KOL’s views, Analyst’s views, Sickle Cell Disease Market Access and Reimbursement

To know more about Sickle Cell Disease companies working in the treatment market, visit @ Sickle Cell Disease Clinical Trials and Therapeutic Assessment

Table of Contents

1. Sickle Cell Disease Market Report Introduction

2. Executive Summary for Sickle Cell Disease

3. SWOT analysis of Sickle Cell Disease

4. Sickle Cell Disease Patient Share (%) Overview at a Glance

5. Sickle Cell Disease Market Overview at a Glance

6. Sickle Cell Disease Disease Background and Overview

7. Sickle Cell Disease Epidemiology and Patient Population

8. Country-Specific Patient Population of Sickle Cell Disease

9. Sickle Cell Disease Current Treatment and Medical Practices

10. Sickle Cell Disease Unmet Needs

11. Sickle Cell Disease Emerging Therapies

12. Sickle Cell Disease Market Outlook

13. Country-Wise Sickle Cell Disease Market Analysis (2020–2034)

14. Sickle Cell Disease Market Access and Reimbursement of Therapies

15. Sickle Cell Disease Market Drivers

16. Sickle Cell Disease Market Barriers

17. Sickle Cell Disease Appendix

18. Sickle Cell Disease 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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