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.
Get a Free Sample PDF Report to know more about Sickle Cell Disease Pipeline Therapeutic Assessment-
https://www.delveinsight.com/report-store/sickle-cell-disease-pipeline-insight
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
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 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
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.
Media ContactCompany Name: DelveInsightContact Person: Gaurav BoraEmail: Send EmailPhone: +14699457679Address:304 S. Jones Blvd #2432City: Las VegasState: NVCountry: United StatesWebsite: https://www.delveinsight.com/
