New York, Nov. 28, 2022 (GLOBE NEWSWIRE) -- Propulsion of Thalassemia Pipeline as Novel and Extensive 25+ Therapies Likely to Enter in the Treatment Domain | DelveInsight
DelveInsight’s thalassemia pipeline report depicts a robust space with 20+ active players working to develop 25+ pipeline therapies for thalassemia treatment.
DelveInsight’s 'Thalassemia Pipeline Insight – 2022' report provides comprehensive global coverage of available, marketed, and pipeline thalassemia therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the thalassemia pipeline domain.
Key Takeaways from the Thalassemia Pipeline Report
- DelveInsight’s thalassemia pipeline report depicts a robust space with 20+ active players working to develop 25+ pipeline therapies for thalassemia treatment.
- Key thalassemia companies such as Vifor Pharma, CRISPR Therapeutics, Bioray Laboratories, Agios Pharmaceuticals, Inc., EdiGene (GuangZhou) Inc., EmeraMed, Editas Medicine, Inc., Forma Therapeutics, Inc., Ionis Pharmaceuticals, Inc., Silence Therapeutics plc, Vertex Pharmaceuticals, Graphite Bio, Global Blood Therapeutics, Fulcrum Therapeutics, Shanghai BDgene Technology Co., Ltd, Orchard Therapeutics, Beam Therapeutics, and others are evaluating new drugs for thalassemia to improve the treatment landscape.
- Promising thalassemia pipeline therapies in various stages of development include Sotatercept, VIT-2763, CTX001, BRL-101, AG-348, ET-01, Emeramide, EDIT-301, Etavopivat, sapablursen, SLN124, GPH102, HbF inducers, FTX-6058, OTL-300, BEAM-101, and others.
- In September 2022, Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics announced that Vertex had concluded discussions with the U.S. Food and Drug Administration (FDA), and the FDA granted exagamglogene autotemcel (exa-cel) a rolling review for the potential treatment of sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). Vertex will submit its biologics licensing application (BLA) for exa-cel for rolling review beginning in November 2022 and expects to complete the submission by the end of Q1 2023.
- In September 2022, Novo Nordisk and Forma Therapeutics, Holdings Inc. announced that they had entered into a definitive agreement under which Novo Nordisk would acquire Forma Therapeutics for $20 per share in cash, which represents a total equity value of $1.1 billion. Forma Therapeutics is a clinical-stage biopharmaceutical company focused on transforming the lives of patients with sickle cell disease (SCD) and rare blood disorders.
- In September 2022, Silence Therapeutics plc announced preliminary results from the single dose component of the GEMINI II phase 1 study of SLN124 in 24 adults with non-transfusion-dependent thalassemia. The primary objective of the single dose arm was to evaluate the safety and tolerability of SLN124 subcutaneous dosing (1.0, 3.0, and 6.0 mg/kg) in alpha/beta-thalassemia patients.
- In August 2022, Agios Pharmaceuticals, Inc. announced that data from the core period of the open-label, Phase 2 study of PYRUKYND® (mitapivat) in adults with non-transfusion-dependent α- or β-thalassemia were published on August 11, 2022, in The Lancet. In Adults with Non-transfusion-dependent α- or β-Thalassemia, PYRUKYND® Induced ≥1.0 g/dL Hemoglobin Increase from Baseline in 16 of 20 (80%) Patients Between Weeks 4-12.
- In May 2022, Editas Medicine, Inc. announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to EDIT-301, an investigational, gene editing medicine, for the treatment of beta-thalassemia.
- In May 2022, Graphite Bio, Inc. presented preclinical data for GPH102, the company’s differentiated gene replacement program for beta-thalassemia, in an oral presentation at the American Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting.
Request a sample and discover the recent advances in thalassemia drug treatment @Thalassemia Pipeline Report
The thalassemia pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage thalassemia drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the thalassemia clinical trial landscape.
Thalassemia Overview
Thalassemia is an inherited blood disorder. This means that it is inherited from one or both parents via their genes. When a person has thalassemia, their body produces less hemoglobin than usual. Genetics is among the prominent thalassemia causes. Thalassemia is classified into two types: alpha and beta. For each type, different genes are impacted.
Thalassemia symptoms include slow child growth, wide or brittle bones, an enlarged spleen (an organ in your abdomen that filters blood and fights disease), fatigue, weakness, pale or yellow skin, dark urine, and a poor appetite.
The following tests may be used for thalassemia diagnosis to determine whether a person is a carrier and can pass the disorder on to their children: Total blood count (CBC), Hemoglobin electrophoresis with A2 and F quantitation measures the size, number, and maturity of different blood cells in a given volume of blood. A blood test determines the type of hemoglobin, FEP (free-erythrocyte protoporphyrin), and ferritin.
Find out more about thalassemia guidelines @Thalassemia Treatment Guidelines
A snapshot of the Thalassemia Pipeline Drugs mentioned in the report:
| Drugs | Company | Phase | MoA | RoA |
| CTX-001 | CRISPR Therapeutics | Phase III | Fetal hemoglobin expression stimulant | Intravenous |
| Mitapivat | Agios Pharmaceuticals | Phase III | Pyruvate kinase stimulant | Oral |
| Sotatercept | Acceleron Pharma/Celgene Corporation | Phase II | Transforming growth factor beta inhibitor | Subcutaneous |
| Emeramide | EmeraMed | Phase II | Antioxidant | Oral |
| Sapablursen | Ionis Pharmaceuticals | Phase II | TMPRSS6 protein inhibitor | Subcutaneous |
Learn more about the emerging thalassemia pipeline therapies @Thalassemia Clinical Trials
Thalassemia Therapeutics Assessment
The thalassemia pipeline report proffers an integral view of thalassemia emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration.
Scope of the Thalassemia Pipeline Report
- Coverage: Global
- Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination
- Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
- Therapeutics Assessment By Route of Administration: Intra-articular, Intraocular, Intrathecal, Intravenous, Ophthalmic, Oral, Parenteral, Subcutaneous, Topical, Transdermal
- Therapeutics Assessment By Molecule Type: Oligonucleotide, Peptide, Small molecule
- Therapeutics Assessment By Mechanism of Action: Cell replacements, Fetal haemoglobin expression stimulants, Pyruvate kinase stimulants, Chelating agents, Cytochrome P 450 cyp2c8 inhibitors, Activin inhibitors, Bone resorption factor inhibitors, Haemoglobin stimulants, Osteoblast stimulants, Osteoclast inhibitors, Osteogenesis stimulants, Transforming growth factor beta inhibitors, Antioxidants, TMPRSS6 protein inhibitors
- Key Thalassemia Companies: Vifor Pharma, CRISPR Therapeutics, Bioray Laboratories, Agios Pharmaceuticals, Inc., EdiGene (GuangZhou) Inc., EmeraMed, Editas Medicine, Inc., Forma Therapeutics, Inc., Ionis Pharmaceuticals, Inc., Silence Therapeutics plc, Vertex Pharmaceuticals, Graphite Bio, Global Blood Therapeutics, Fulcrum Therapeutics, Shanghai BDgene Technology Co., Ltd, Orchard Therapeutics, Beam Therapeutics, and others.
- Key Thalassemia Pipeline Therapies: Sotatercept, VIT-2763, CTX001, BRL-101, AG-348, ET-01, Emeramide, EDIT-301, Etavopivat, sapablursen, SLN124, GPH102, HbF inducers, FTX-6058, ,OTL-300, BEAM-101, and others.
Dive deep into rich insights for new drug for thalassemia treatment; visit @Thalassemia Medications
Table of Contents
| 1. | Thalassemia Pipeline Report Introduction |
| 2. | Thalassemia Pipeline Report Executive Summary |
| 3. | Thalassemia Pipeline: Overview |
| 4. | Analytical Perspective In-depth Commercial Assessment |
| 5. | Thalassemia Clinical Trial Therapeutics |
| 6. | Thalassemia Pipeline: Late Stage Products (Pre-registration) |
| 7. | Thalassemia Pipeline: Late Stage Products (Phase III) |
| 8. | Thalassemia Pipeline: Mid Stage Products (Phase II) |
| 9. | Thalassemia Pipeline: Early Stage Products (Phase I) |
| 10. | Thalassemia Pipeline Therapeutics Assessment |
| 11. | Inactive Products in the Thalassemia Pipeline |
| 12. | Company-University Collaborations (Licensing/Partnering) Analysis |
| 13. | Key Companies |
| 14. | Key Products in the Thalassemia Pipeline |
| 15. | Unmet Needs |
| 16. | Market Drivers and Barriers |
| 17. | Future Perspectives and Conclusion |
| 18. | Analyst Views |
| 19. | Appendix |
For further information on the thalassemia pipeline therapeutics, reach out @Thalassemia Drug Treatment
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