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 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-3001: 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
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
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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