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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, 2024“ 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 2023, BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a biopharmaceutical company focused on innovative treatments in oncology and rare diseases, has initiated the Phase 1 clinical trial for motixafortide. This trial will evaluate motixafortide as a standalone therapy and in combination with natalizumab for mobilizing CD34+ hematopoietic stem cells (HSC) for gene therapies in sickle cell disease (SCD). In partnership with Washington University School of Medicine in St. Louis, the proof-of-concept study aims to enroll five SCD patients and assess the safety and tolerability of both treatment regimens.

• In May 2023, The orphan drug designation for EDIT-301, a novel cell-based gene-editing therapy administered through a single infusion, was approved by the US Food and Drug Administration (FDA) for the treatment of sickle cell disease (SCD).

• In April 2023, bluebird bio, Inc. has reported the submission of its Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for the gene therapy lovotibeglogene autotemcel (lovo-cel) intended for individuals aged 12 and above with sickle cell disease (SCD) who have a history of vaso-occlusive events (VOEs). The BLA includes a request for Priority Review, aiming to expedite the FDA’s assessment to six months from the filing date, as opposed to the standard 10-month review period. If granted approval, lovo-cel would be the third ex-vivo gene therapy by bluebird bio approved by the FDA for a rare genetic disease and the second FDA approval for an inherited hemoglobin disorder, showcasing the company’s over a decade of leadership in gene therapy.

• In March 2023, BioLineRx Ltd. has disclosed a partnership with Washington University School of Medicine in St. Louis to progress a Phase I clinical study focusing on assessing the safety and viability of the company’s primary clinical candidate, motixafortide. The trial aims to mobilize CD34+ hematopoietic stem cells (HSCs) for gene therapies in sickle cell disease (SCD).

• In November 2022, C4X Discovery Holdings plc entered into an exclusive global licensing deal with AstraZeneca valued at up to $402 million for its NRF2 Activator program.

• In October 2022, Pfizer has finalized its purchase of Global Blood Therapeutics, Inc. (GBT), a biopharmaceutical company specializing in the exploration, advancement, and provision of transformative treatments, initially focusing on sickle cell disease (SCD). This acquisition strengthens Pfizer’s dedication to SCD, furthering its three-decade involvement in the rare hematology field.

Sickle Cell Disease Overview

Sickle cell disease (SCD), also known as sickle cell anemia, is a genetic disorder characterized by abnormal hemoglobin, the protein in red blood cells responsible for carrying oxygen throughout the body. This condition affects the shape of red blood cells, causing them to become rigid and crescent-shaped, resembling a sickle.

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

• RL 101: BRL Medicine

• ORY-300: 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 

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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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