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Thalassemia Pipeline and Clinical Trial Report | 30+ Companies and 30+ Drugs

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Thalassemia Pipeline and Clinical Trial Report | 30+ Companies and 30+ Drugs

January 12
20:04 2023
Thalassemia Pipeline and Clinical Trial Report | 30+ Companies and 30+ Drugs

DelveInsight’s, “Thalassemia Pipeline Insight, 2023” report provides comprehensive insights about 30+ Thalassemia companies and 30+ pipeline drugs in Thalassemia pipeline landscape. It covers the pipeline drug profiles, including Thalassemia clinical trial and nonclinical stage products. Thalassemia pipeline report also covers the 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 Thalassemia Pipeline Report

  • DelveInsight’s thalassemia pipeline report depicts a robust space with 30+ active players working to develop 30+ pipeline therapies for thalassemia treatment.
  • The leading Thalassemia Companies include 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.
  • 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.
  • The Thalassemia companies and academics are working to assess challenges and seek opportunities that could influence Thalassemia R&D. The Thalassemia pipeline therapies under development are focused on novel approaches to treat/improve Thalassemia.

 

Explore more about the latest breakthroughs of the Thalassemia Treatment Landscape report @ Thalassemia Pipeline Outlook

 

Thalassemia Overview

Thalassemia is a blood disorder that is inherited. This means it is passed down from one or both parents through their genes. When a person has thalassemia, the body makes less hemoglobin than normal. Hemoglobin is an iron-rich protein in red blood cells. It carries oxygen to all parts of the body. There are 2 main types of thalassemia: alpha and beta. Different genes are affected for each type. Thalassemia can cause mild or severe anemia. Anemia occurs when the body does not have enough red blood cells or hemoglobin. The severity and type of anemia depends on how many genes are affected. Alpha thalassemia occurs when some or all of the 4 genes that make hemoglobin (the alpha-globin genes) are missing or damaged. There are 4 types of alpha thalassemia: Alpha thalassemia silent carrier: One gene is missing or damaged, and the other 3 are normal. Alpha thalassemia carrier: Two genes are missing. Hemoglobin H disease: Three genes are missing. This leaves just 1 working gene. Alpha thalassemia major. All 4 genes are missing. This causes severe anemia. In most cases, a baby with this condition will die before birth. This is a genetic disease inherited from one or both parents.

 

Recent Developmental Activities in the Thalassemia Treatment Landscape

  • 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.

 

Learn more about the Thalassemia emerging therapies @ Thalassemia Ongoing Clinical Trials Analysis

 

Thalassemia Emerging Drugs Profile

 

CTX-001: CRISPR Therapeutics

CTX001 is an autologous CRISPR/Cas9 gene-edited hematopoietic stem cell therapy in development for patients suffering from β-thalassemia and sickle cell disease. Currently, the drug is in Phase III stage of Clinical trial evaluation for the treatment of Beta-Thalassemia. 

 

Mitapivat:  Agios Pharmaceuticals

Mitapivat is a first-in-class, investigational, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase R (PKR) enzymes. Currently, the drug is in Phase III stage of Clinical trial evaluation for the treatment of Transfusion-dependent Alpha or Beta Thalassemia. 

 

DST-0509: DisperSol Technologies

DST-0509, is an investigational drug being developed by DisperSol Technologies. It belongs, to the class of antianaemics.Currently, the drug is in Phase II stage of Clinical trial evaluation for the treatment of Thalassemia.

 

Thalassemia Therapeutic Assessment

There are approx. 30+ key Thalassemia companies which are developing the therapies for Thalassemia. The companies which have their Thalassemia drug candidates in the most advanced stage, i.e. phase III include, CRISPR Therapeutics.

 

Dive deep into rich insights for new drugs for thalassemia key players and drugs; visit @ Thalassemia Treatment Landscape

 

Scope of the Thalassemia Pipeline Report

  • Coverage- Global
  • 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.
  • 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.
  • Thalassemia Segmentation: Phases, Molecule Type, Route of Administration, Mechanism of Action, Product Type

 

Table of Content

  1. Introduction
  2. Thalassemia Executive Summary
  3. Thalassemia: Overview
  4. Thalassemia Pipeline Therapeutics
  5. Thalassemia Therapeutic Assessment
  6. Thalassemia– DelveInsight’s Analytical Perspective
  7. Late Stage Products (Phase III)
  8. CTX-001: CRISPR Therapeutics
  9. Drug profiles in the detailed report…..
  10. Mid-Stage Products (Phase II)
  11. DST-0509: DisperSol Technologies
  12. Drug profiles in the detailed report…..
  13. Early Stage Products (Phase I)
  14. Drug name: Company name
  15. Drug profiles in the detailed report…..
  16. Preclinical and Discovery Stage Products
  17. Drug name: Company name
  18. Drug profiles in the detailed report…..
  19. Inactive Products
  20. Thalassemia Key Companies
  21. Thalassemia Key Products
  22. Thalassemia- Unmet Needs
  23. Thalassemia- Market Drivers and Barriers
  24. Thalassemia- Future Perspectives and Conclusion
  25. Thalassemia Analyst Views
  26. Thalassemia Key Companies
  27. Appendix

 

For further information on the thalassemia pipeline therapeutics, reach out to Thalassemia Market Drivers and Barriers

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