Sickle Cell Disease Pipeline, Clinical Trials Studies, Emerging Drugs, Agreements & Collaborations, and Latest News 2023 (Updated)

DelveInsight's, "Sickle Cell Disease Pipeline Insights 2023" report provides comprehensive insights about 40+ companies and 50+ pipeline drugs in Sickle cell disease pipeline landscape. It covers the Sickle Cell Disease pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Sickle Cell Disease pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Key Takeaways from the Sickle Cell Disease Pipeline Report
• DelveInsight's Sickle Cell Disease pipeline report depicts a robust space with 40+ active players working to develop 50+ pipeline therapies for Sickle Cell Disease treatment.
• The leading companies working in the Sickle Cell Disease Market include Cellectis, Sana Biotechnology, Global Blood Therapeutics, Inc., CSL Behring, Pfizer, Aruvant Sciences, Graphite Bio, Novartis, Agios Pharmaceuticals, Forma therapeutics, Vertex Pharmaceuticals, Global Blood Therapeutics, Inc., Alfasigma, Novo Nordisk, bluebird bio, ExCellThera, Gamida Cell, KM Biologics/Takeda, Editas Medicine, and others.
• Promising Sickle Cell Disease Pipeline Therapies in the various stages of development include SHP655, Prasugrel, FT-4202, Crizanlizumab, Sevuparin, Voxelotor, VIT-2763 120 mg, NKTT120, and others.
• October 2023: Pfizer announced a study of Phase 3 clinical trials for Voxelotor. This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to < 15 years old, with Sickle Cell Disease. The primary objective is to evaluate the effect of voxelotor on the TCD (Transcranial Doppler Ultrasound) measurements in SCD participants in this age range. This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ≥ 2 to < 15 years old, with Sickle Cell Disease. The study will be conducted at approximately 50 international clinical sites, and will enroll approximately 224 participants. Participants will be randomized in a 1:1 ratio to receive voxelotor or placebo. All participants younger than 12 years of age and randomized to voxelotor will receive a dose based on their body weight, to provide exposure corresponding to the adult dose of 1500 mg/day.
• October 2023 Alexion announced a study of Phase 2 clinical trials for ALXN1820. The primary objective of this study is to assess the safety and tolerability of ALXN1820 SC (subcutaneous) in participants with SCD (Sickle Cell Disease). A Phase 2a, Randomized, Open-Label Study to Evaluate Multiple Dosing Regimens of Subcutaneous ALXN1820 in Adult Participants With Sickle Cell Disease.
• October 2023: Afimmune announced a study of Phase 2 clinical trials for Epeleuton. To assess the pharmacokinetics, pharmacodynamics and safety of Epeleuton capsules in adult SCD patients who are aged ≥18 years. The trial will consist of a 28-day screening period, 16 weeks of active treatment and a 30-day post-treatment follow-up period. An Open-label Mechanistic Study to Assess the Pharmacokinetics, Pharmacodynamics and Safety of Orally Administered Epeleuton in Patients With Sickle Cell Disease.
• October 2023: Novo Nordisk A/S announced a study of Phase 2 Clinical Trials for NDec - oral decitabine-tetrahydrouridine and HU - Hydroxyurea. This study examines how well a new, potential medicine called NDec works and is tolerated in people with sickle cell disease. NDec is a combination of two medicines (decitabine-tetrahydrouridine). Both medicines are new for the treatment of sickle cell disease. Participants who are not taking Hydroxyurea (HU) will get NDec, NDec and placebo, or placebo. Participants who are on HU treatment before joining the study will get NDec, NDec and placebo, or continue on HU. Which treatment participants get is decided by chance. Participants getting NDec and/or Placebo will get capsules to take twice weekly. The study will last for about a year.
• October 2023: Agios Pharmaceuticals Inc. announced a study of Phase 2 & 3 clinical trials for Mitapivat. This clinical trial is a Phase 2/3 study that will determine the recommended dose of mitapivat and evaluate the efficacy and safety of mitapivat in sickle cell disease by testing how well mitapivat works compared to placebo to increase the amount of hemoglobin in the blood and to reduce or prevent the occurrence of sickle cell pain crises. In addition, the long-term effect of mitapivat on efficacy and safety will be explored in an open-label extension portion. The study is a Phase 2/3 study in which the recommended dose of mitapivat will be selected and further evaluated. The Phase 2 portion includes a 12-week randomized, placebo-controlled period in which participants will be randomized in a 1:1:1 ratio to receive 2 dose levels of mitapivat or placebo. The Phase 3 portion includes a 52-week randomized, placebo-controlled period in which participants will be randomized in a 2:1 ratio to receive the recommended mitapivat dose level or placebo. Participants who complete either the Phase 2 or Phase 3 portion will have the option to move into a 216-week open label extension period to receive mitapivat.
• October 2023: Hoffmann-La Roche announced a study of Phase 2 clinical trials for Crovalimab. This study is designed to evaluate the efficacy, safety and pharmacokinetics of crovalimab compared with placebo as adjunct therapy in the prevention of VOEs in participants with Sickle Cell Disease. Crovalimab will be administered at a dose of 1000 mg IV (for participants with body weight between 40 kg and 100 kg) or 1500 mg IV (for participants with body weight >= 100 kg) on Week 1 Day 1. On Week 1 Day 2 and on Weeks 2, 3 and 4, crovalimab will be administered at a dose of 340 mg SC. For Week 5 and Q4W thereafter, crovalimab will be administered at a dose of 680 mg SC (for participants with body weight between 40 kg and 100 kg) or 1020 mg SC (for participants with body weight >= 100 kg). Dosing schedule will be as per Arm Description.

Request a sample and discover the recent advances in Sickle Cell Disease Treatment Drugs @ Sickle Cell Disease Pipeline Report- https://www.delveinsight.com/report-store/sickle-cell-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

In the Sickle Cell Disease pipeline report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, Sickle Cell Disease NDA approvals (if any), and product development activities comprising the technology, Sickle Cell Disease (SCD) collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Sickle Cell Disease Overview
Sickle cell disease is the name given for a group of inherited conditions that affect the red blood cells and the most serious type is called Sickle cell anemia. Sickle cells are destroyed rapidly in the bodies of people with the disease, causing anemia which is why it is commonly known as sickle cell anemia.

Find out more about Sickle Cell Disease Therapeutics Assessment @ Sickle Cell Disease Preclinical and Discovery Stage Products- https://www.delveinsight.com/sample-request/sickle-cell-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Sickle Cell Disease Emerging Drugs Profile
• BPX-501 T cells: Bellicum Pharmaceuticals
• Canakinumab: Novartis
• EPI01: Novo Nordisk
• CTX001: Vertex Pharmaceuticals

Sickle Cell Disease Pipeline Therapeutics Assessment
There are approx. 40+ key companies which are developing the Sickle Cell Disease therapies. The Sickle Cell Disease companies which have their Sickle Cell Disease (SCD) drug candidates in the most advanced stage, i.e. Phase III include Vertex Pharmaceuticals.

DelveInsight's Sickle Cell Disease Pipeline 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) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

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

Learn more about the emerging Sickle Cell Disease Pipeline Therapies @ Sickle Cell Disease Clinical Trials Assessment- https://www.delveinsight.com/sample-request/sickle-cell-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Scope of the Sickle Cell Disease Report
• Coverage- Global
• Sickle Cell Disease Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Sickle Cell Disease Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
• Sickle Cell Disease Companies- Cellectis, Sana Biotechnology, Global Blood Therapeutics, Inc., CSL Behring, Pfizer, Aruvant Sciences, Graphite Bio, Novartis, Agios Pharmaceuticals, Forma therapeutics, Vertex Pharmaceuticals, Global Blood Therapeutics, Inc., Alfasigma, Novo Nordisk, bluebird bio, ExCellThera, Gamida Cell, KM Biologics/Takeda, Editas Medicine, and others.
• Sickle Cell Disease Pipeline Therapies- SHP655, Prasugrel, FT-4202, Crizanlizumab, Sevuparin, Voxelotor, VIT-2763 120 mg, NKTT120, and others.

Dive deep into rich insights for new drugs for Sickle Cell Disease Treatment, Visit @ Sickle Cell Disease Market Drivers and Barriers, and Future Perspective- https://www.delveinsight.com/sample-request/sickle-cell-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

Table of Content
1. Introduction
2. Executive Summary
3. Sickle Cell Disease (SCD): Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Sickle Cell Disease (SCD) - DelveInsight's Analytical Perspective
7. Mid Stage Products (Phase III)
8. CTX001: Vertex Pharmaceuticals
9. Drug profiles in the detailed report…..
10. Mid Stage Products (Phase II/III)
11. Mitapivat: Agios Pharmaceuticals
12. Drug profiles in the detailed report…..
13. Early Stage Products (Phase I/II)
14. ARU-1801: Aruvant Sciences
15. Drug profiles in the detailed report…..
16. Early Stage Products (Phase I)
17. GBT021601: Global Blood Therapeutics, Inc.
18. Drug profiles in the detailed report…..
19. Preclinical Stage Products
20. TALGlobin01: Cellectis
21. Drug profiles in the detailed report…..
22. Inactive Products
23. Sickle Cell Disease (SCD) Key Companies
24. Sickle Cell Disease (SCD) Key Products
25. Sickle Cell Disease (SCD)- Unmet Needs
26. Sickle Cell Disease (SCD)- Market Drivers and Barriers
27. Sickle Cell Disease (SCD)- Future Perspectives and Conclusion
28. Sickle Cell Disease (SCD) Analyst Views
29. Sickle Cell Disease (SCD) Key Companies
30. Appendix

For further information on the Sickle Cell Disease Pipeline therapeutics, reach out to Sickle Cell Disease Unmet Needs and Analyst Views- https://www.delveinsight.com/sample-request/sickle-cell-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=ypr

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DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve.

Company Name: DelveInsight Business Research LLP
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