“Sickle Cell Disease Pipeline Insight, 2023“ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Sickle Cell Disease Market.
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.
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 Afimmune, IHP Therapeutics, Hillhurst Biopharmaceuticals, Agios Pharmaceuticals, Novo Nordisk, Alexion Pharmaceuticals, Prolong Pharmaceuticals, Pfizer, CRISPR Therapeutics, Vertex Pharmaceuticals, Agios Pharmaceuticals, Novartis, Chugai Pharmaceutical/Roche, Editas Medicine, Sangamo Therapeutics, Beam Therapeutics, CSL Behring, and others, are developing therapies for the Sickle Cell Disease treatment
- Emerging Sickle Cell Disease therapies in the different phases of clinical trials are- Epeleuton, IHP-102, HBI-002, AG-946, EPI01, ALXN1820, Sanguinate, Inclacumab, Exagamglogene autotemcel, Mitapivat, Canakinumab, Crovalimab, EDIT 301, BIVV003, BEAM101, Hemopexin, and others are expected to have a significant impact on the Sickle Cell Disease market in the coming years.
- In May 2023, For the treatment of sickle cell disease (SCD), the US Food and medication Administration (FDA) designated EDIT-301, an investigational cell-based gene-editing therapy, as an orphan medication. It is administered as a single infusion.
- In April 2023, The US Food and Drug Administration (FDA) has received a biologics license application (BLA) from bluebird bio, Inc. for lovotibeglogene autotemcel, also known as lovo-cel, gene therapy for sickle cell disease (SCD) patients 12 years of age and older with a history of vaso-occlusive episodes (VOEs). A request for Priority Review is included in the BLA; if approved, this would reduce the FDA’s usual 10-month review period for the application to six months from the date of filing. Building on more than a decade of leadership in gene therapy, lovo-cel would be bluebird bio’s third ex-vivo gene therapy approved by the FDA for a rare genetic condition and its second FDA approval for a hereditary hemoglobin deficiency, if approved.
- In March 2023, In order to advance a Phase I clinical trial that will assess the safety and viability of the company’s lead clinical candidate, motixafortide, to mobilize CD34+ hematopoietic stem cells (HSCs) for gene therapies in sickle cell disease (SCD), BioLineRx Ltd. announced a partnership with Washington University School of Medicine in St. Louis.
- In November 2022, AstraZeneca and C4X Discovery Holdings plc inked an exclusive global licensing deal worth up to $402 million for C4X’s NRF2 Activator program.
- In October 2022, Pfizer Inc. declared the successful completion of its acquisition of GBT, a biopharmaceutical business devoted to the discovery, development, and distribution of transformative therapies, beginning with treatments for sickle cell disease, that offer hope to marginalized patient communities.
- In October 2022, Pfizer declared the successful completion of its acquisition of Global Blood Therapeutics, Inc. (GBT), a biopharmaceutical business devoted to the research, development, and distribution of transformative therapies, beginning with sickle cell disease (SCD), that offer hope to marginalized patient communities.
- In October 2022, The acquisition of Forma Therapeutics Holdings, Inc. by Novo Nordisk is now complete. A clinical-stage biopharmaceutical firm, Forma Therapeutics is committed to improving the lives of people with uncommon blood diseases and sickle cell disease (SCD).
- In June 2022, Precision BioSciences, Inc., a clinical-stage gene editing startup creating ex vivo allogeneic CAR T and in vivo gene editing therapies based on ARCUS, announced that it has partnered with Novartis Pharma AG on an exclusive global basis for in vivo gene editing research and development, as well as a license agreement.
- In June 2022, GBT, Inc. said that inclacumab, a therapy for sickle cell disease, has been designated by the US FDA as an orphan medication and as a rare pediatric disease.
- In March 2022, To assess the long-term safety of inclacumab given to patients with sickle cell disease who have taken part in an inclacumab clinical trial, GBT started an open-label extension study. This open-label trial assesses the safety of giving patients with sickle cell disease inclacumab over an extended period of time.
Sickle Cell Disease Overview
The genetic red blood cell diseases known as sickle cell disease (SCD) impact hemoglobin, the protein responsible for carrying oxygen throughout the body. Round, healthy red blood cells travel via tiny blood channels to supply oxygen to every area of the body. Hemoglobin abnormalities in people with sickle cell disease (SCD) result in hard, sticky red blood cells that resemble a C-shaped “sickle,” a farm implement.
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Emerging Sickle Cell Disease Drugs Under Different Phases of Clinical Development Include:
- Epeleuton: Afimmune
- IHP-102 IHP Therapeutics
- HBI-002: Hillhurst Biopharmaceuticals
- AG-946 Agios Pharmaceuticals
- EPI01: Novo Nordisk
- ALXN1820: Alexion Pharmaceuticals
- Sanguinate: Prolong Pharmaceuticals
- Inclacumab: Pfizer
- Exagamglogene autotemcel: CRISPR Therapeutics/Vertex Pharmaceuticals
- Mitapivat: Agios Pharmaceuticals
- Canakinumab: Novartis
- 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
Sickle Cell Disease Molecule Type
Sickle Cell Disease Products have been categorized under various Molecule types, such as
- Monoclonal Antibody
- Small molecule
- Gene therapy
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 5+ 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 – Roche, Beam Therapeutics, Editas Medicine, Sangamo Therapeutics, Bellicum Pharmaceuticals, Invenux, EpiDestiny, Hillhurst Biopharmaceuticals, CRISPR therapeutics, Bluebird Bio, Pfizer, Novo Nordisk, Agios Pharmaceuticals, Alexion Pharmaceuticals, Takeda, Prolong Pharmaceuticals, CSL Behring, Fulcrum Therapeutics, Sana Biotechnology, 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.
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Sickle Cell Disease Pipeline Market Drivers
- Increasing prevalence of Sickle Cell Disease, 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: Afimmune, IHP Therapeutics, Hillhurst Biopharmaceuticals, Agios Pharmaceuticals, Novo Nordisk, Alexion Pharmaceuticals, Prolong Pharmaceuticals, Pfizer, CRISPR Therapeutics, Vertex Pharmaceuticals, Agios Pharmaceuticals, Novartis, Chugai Pharmaceutical/Roche, Editas Medicine, Sangamo Therapeutics, Beam Therapeutics, CSL Behring, and others
- Key Sickle Cell Disease Therapies: Epeleuton, IHP-102, HBI-002, AG-946, EPI01, ALXN1820, Sanguinate, Inclacumab, Exagamglogene autotemcel, Mitapivat, Canakinumab, Crovalimab, EDIT 301, BIVV003, BEAM101, Hemopexin, 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
20. 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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