Sickle Cell Disease Pipeline 2025: MOA, ROA, FDA-Approved Drugs, And Clinical Trial Progress Assessment By Delveinsight Sanofi, Novartis AG, Bluebird Biotech, Pfizer Inc., Aruvant Sciences Inc

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(Las Vegas, Nevada, United States) As per DelveInsight's assessment, globally, Sickle Cell Disease pipeline constitutes 55+ key companies continuously working towards developing 60+ Sickle Cell Disease treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight.

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, 2025 " report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Sickle Cell Disease Market.

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 August 2025, Pfizer is reassessing its path forward after its experimental sickle cell disease therapy failed to achieve the primary endpoint in a Phase III trial. The THRIVE-131 study (NCT04935879) was testing inclacumab, a P-selectin inhibitor, in patients aged 16 and older with sickle cell disease. However, the trial did not demonstrate a significant reduction in vaso-occlusive crises (VOCs) over 48 weeks, casting uncertainty on the drug's future prospects.

• In June 2025, Ellarity has initiated dosing of the first participant in a Phase I clinical trial of its oral drug candidate, CLY-124, for the treatment of sickle cell disease (SCD). This milestone comes after the US FDA cleared the company's investigational new drug (IND) application. The global study is designed to assess the safety, tolerability, and pharmacokinetics of the therapy, first in healthy volunteers and subsequently in patients with SCD.

• In June 2025, The US FDA granted a special designation to Vascarta's lead candidate, VAS-101 (Vasceptor), for the treatment of sickle cell disease (SCD). This status offers key advantages, including seven years of market exclusivity upon approval and certain FDA fee exemptions. VAS-101 is a patented topical curcumin formulation utilizing Vascarta's transdermal delivery technology, developed to address the poor bioavailability and limited effectiveness of oral curcumin. Preclinical studies in animals indicate that VAS-101 may help reduce chronic pain, stabilize red blood cells, and decrease inflammation in SCD patients.

• In December 2024, Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company specializing in precision genetic medicines using base editing, has unveiled new safety and efficacy findings from its BEACON Phase 1/2 trial of BEAM-101 in sickle cell disease (SCD) patients experiencing severe vaso-occlusive crises (VOCs). These results were highlighted 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 focused on developing transformative therapies in oncology and rare diseases, announced that an abstract featuring the initial results from a Phase 1 clinical trial of motixafortide, both as a monotherapy and in combination with natalizumab for CD34+ hematopoietic stem cell (HSC) mobilization for gene therapies in sickle cell disease (SCD), has been accepted for oral presentation at the 66th American Society of Hematology (ASH) Annual Meeting & Exposition, scheduled for December 7-10, 2024, in San Diego, California. This proof-of-concept study, conducted in partnership with Washington University School of Medicine in St. Louis, aims to explore alternative HSC mobilization strategies that could enhance the treatment of sickle cell disease patients pursuing gene therapy.

• In October 2024, Zydus Lifesciences has signed a Memorandum of Agreement (MoA) with the Indian Council of Medical Research (ICMR) to begin Phase 2 clinical trials of Desidustat in patients with Sickle Cell Disease. This Phase IIa, double-blind, randomized, placebo-controlled, parallel, multi-center proof-of-concept study will be co-funded and co-monitored by INTENT, the Indian National Clinical Trial and Education Network, Clinical Studies and Trial Unit, Division of Development Research, ICMR. The study aims to assess the efficacy and safety of Desidustat oral tablets for the treatment of sickle cell disease.

• In May 2024, BioLineRx Ltd has announced a multi-center Phase I clinical trial, sponsored by St. Jude Children's Research Hospital, Inc., to assess motixafortide for mobilizing CD34+ hematopoietic stem cells (HSCs) in the development of gene therapies for sickle cell disease (SCD).

• In April 2024, Health Canada granted priority review to the application of the gene-editing therapy exagamglogene autotemcel (exa-cel) for patients aged 12 and older with sickle cell disease (SCD) experiencing recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta-thalassemia (TDT).

• In January 2024, GlycoMimetics announced promising initial results from a Phase Ia clinical trial of its E-selectin antagonist, GMI-1687, as a potential treatment for sickle cell disease (SCD).

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 60+ 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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