The Sickle Cell Disease Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.

“Sickle Cell Disease Pipeline Insight, 2026“ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Sickle Cell Disease Pipeline Landscape.

Some of the key takeaways from the Sickle Cell Disease Pipeline Report:

• Companies across the globe are diligently working toward developing novel Sickle Cell Disease treatment therapies with a considerable amount of success over the years.

• Sickle Cell Disease companies working in the treatment market are – 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, are developing therapies for the Sickle Cell Disease treatment

• Emerging Sickle Cell Disease therapies in the different phases of clinical trials are- 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 are expected to have a significant impact on the Sickle Cell Disease market in the coming years.

• In October 2026, vTv Therapeutics announced that the US FDA has granted Orphan Drug Designation to HPPD (HPP8668), an investigational oral Nrf2/Bach1 modulator being developed for sickle cell disease. Preclinical findings indicated that the candidate may increase fetal hemoglobin levels while reducing oxidative stress and red blood cell sickling. However, its safety and therapeutic efficacy in humans have not yet been established.

• In September 2026, A study published in Cell Stem Cell reported sustained clinical remission in patients with sickle cell disease and beta-thalassemia treated with tBE-mediated base-editing therapy across diverse genetic backgrounds. These findings highlight the potential of gene-editing approaches for treating inherited blood disorders; however, this remains a research advancement and does not represent a new FDA approval.

• In August 2026, Stanford Medicine Children’s Health announced plans for the Kamau Therapeutics-sponsored Restore clinical trial to extend eligibility to children aged 12 years and older, expanding beyond its existing adult population. The investigational approach employs CRISPR-based gene editing to correct the defective beta-globin gene directly, rather than stimulating fetal hemoglobin production as an indirect therapeutic strategy. However, the proposed trial expansion does not constitute FDA marketing approval.

• In August 2026, Genetix Biotherapeutics Inc. and the National Football League Alumni Association have announced a partnership to launch a nationwide awareness and education initiative focused on sickle cell disease (SCD). The collaboration will use community outreach and engagement to highlight the challenges faced by people living with SCD while improving understanding among patients, families, and healthcare professionals about available treatment options and support resources. The initiative will also raise awareness that FDA-approved genetic medicines, including one-time gene therapies, are now available and have the potential to significantly improve outcomes and transform the lives of individuals affected by sickle cell disease.

• In July 2026, Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) has announced that the U.S. Food and Drug Administration (FDA) has approved an expanded indication for CASGEVY® (exagamglogene autotemcel), allowing its use in patients 2 years of age and older with either sickle cell disease (SCD) experiencing recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta-thalassemia (TDT). With this expanded approval, CASGEVY becomes the first FDA-approved gene therapy indicated for children as young as 2 years for the treatment of both SCD and TDT, marking a significant advancement in genetic medicine for pediatric patients.

• In June 2026, Regulatory Challenges for Fulcrum’s Pociredir Program, Fulcrum Therapeutics announced the discontinuation of development of pociredir after FDA feedback raised concerns regarding potential malignancy risks associated with PRC2-targeting therapies. The company subsequently initiated strategic reviews and cost-reduction measures despite previously reported encouraging efficacy data.

• In May 2026, Agios Submits Supplemental NDA for Mitapivat, Agios announced the submission of a supplemental New Drug Application (sNDA) to the FDA seeking accelerated approval of mitapivat for patients with sickle cell disease requiring regular transfusions. The submission was supported by Phase III RISE UP data, with a confirmatory trial planned to further evaluate reduction in transfusion burden.

• In April 2026, Continued Advancement of Next-Generation Clinical Programs, Developers continued enrolling patients into studies evaluating emerging genomic medicines and targeted therapies for sickle cell disease. Research remained focused on expanding therapeutic options beyond currently approved treatments and improving long-term disease management.

• 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).

Sickle Cell Disease Overview

Sickle Cell Disease (SCD) is a genetic blood disorder caused by a mutation in the hemoglobin gene. It results in the production of abnormal hemoglobin (hemoglobin S), which causes red blood cells to become rigid and crescent-shaped. These misshapen cells can block blood flow, leading to pain, organ damage, anemia, and other complications. SCD is inherited when a child receives the defective gene from both parents. Symptoms include fatigue, pain crises, swelling, and increased risk of infections. Treatment focuses on managing symptoms, preventing complications, and may include medications, blood transfusions, or bone marrow transplants

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Emerging Sickle Cell Disease Drugs Under Different Phases of Clinical Development Include:

• 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

Sickle Cell Disease Route of Administration

Sickle Cell Disease pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs, such as

• Intranasal

• Intrathecal

• Intravenous

• Oral

• Oral/Intravenous

• Parenteral

• Subcutaneous

• Subcutaneous/Intramuscular

• Transdermal

Sickle Cell Disease Molecule Type

Sickle Cell Disease Products have been categorized under various Molecule types, such as

• Antisense oligonucleotide

• Gene therapy

• Hormones

• Neuropeptides

• Oligonucleotides

• Small Molecule

• Triglyceride

Sickle Cell Disease Pipeline Therapeutics Assessment

• Sickle Cell Disease Assessment by Product Type

• Sickle Cell Disease By Stage and Product Type

• Sickle Cell Disease Assessment by Route of Administration

• Sickle Cell Disease By Stage and Route of Administration

• Sickle Cell Disease Assessment by Molecule Type

• Sickle Cell Disease by Stage and Molecule Type

DelveInsight’s Sickle Cell Disease Report covers around 50+ products under different phases of clinical development like

• Late-stage products (Phase III)

• Mid-stage products (Phase II)

• Early-stage product (Phase I)

• Pre-clinical and Discovery stage candidates

• Discontinued & Inactive candidates

• Route of Administration

Further Sickle Cell Disease product details are provided in the report. Download the Sickle Cell Disease pipeline report to learn more about the emerging Sickle Cell Disease therapies

Some of the key companies in the Sickle Cell Disease Therapeutics Market include:

Key companies developing therapies for Sickle Cell Disease are – Novartis AG, Global Blood Therapeutics Inc., Emmaus Medical Inc., Addmedica, Medunik USA, Bristol Myers Squibb Co., Sanofi SA, Bluebird Biotechnology, Pfizer Inc., Aruvant Sciences Inc., Glycomimetics Inc., Editas Medicine Inc., CRISPR Therapeutic, and others.

Sickle Cell Disease Pipeline Analysis:

The Sickle Cell Disease pipeline report provides insights into

• The report provides detailed insights about companies that are developing therapies for the treatment of Sickle Cell Disease with aggregate therapies developed by each company for the same.

• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Sickle Cell Disease Treatment.

• Sickle Cell Disease key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

• Sickle Cell Disease Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.

• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Sickle Cell Disease market.

The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.

Download Sample PDF Report to know more about Sickle Cell Disease drugs and therapies

Sickle Cell Disease Pipeline Market Drivers

• Increasing prevalence of SCD, rising initiatives by various governments and private organizations are some of the important factors that are fueling the Sickle Cell Disease Market.

Sickle Cell Disease Pipeline Market Barriers

• However, clinical heterogeneity of the disease, associated complications of the disease and other factors are creating obstacles in the Sickle Cell Disease Market growth.

Scope of Sickle Cell Disease Pipeline Drug Insight

• Coverage: Global

• 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

Request for Sample PDF Report for Sickle Cell Disease Pipeline Assessment and clinical trials

Table of Contents

1. Sickle Cell Disease Report Introduction

2. Sickle Cell Disease Executive Summary

3. Sickle Cell Disease Overview

4. Sickle Cell Disease- Analytical Perspective In-depth Commercial Assessment

5. Sickle Cell Disease Pipeline Therapeutics

6. Sickle Cell Disease Late Stage Products (Phase II/III)

7. Sickle Cell Disease Mid Stage Products (Phase II)

8. Sickle Cell Disease Early Stage Products (Phase I)

9. Sickle Cell Disease Preclinical Stage Products

10. Sickle Cell Disease Therapeutics Assessment

11. Sickle Cell Disease Inactive Products

12. Company-University Collaborations (Licensing/Partnering) Analysis

13. Sickle Cell Disease Key Companies

14. Sickle Cell Disease Key Products

15. Sickle Cell Disease Unmet Needs

16 . Sickle Cell Disease Market Drivers and Barriers

17. Sickle Cell Disease Future Perspectives and Conclusion

18. Sickle Cell Disease Analyst Views

19. Appendix

20. About DelveInsight

About DelveInsight

DelveInsight is a leading 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 business growth and overcome challenges with a practical approach.

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