Beta Thalassemia Pipeline 2025: Latest FDA Approvals, Clinical Trials, and Emerging Therapies Assessment by DelveInsight | Ionis Pharmaceuticals, Inc., Agios Pharma, Silence Therapeutics, Phoenicia

(Las Vegas, Nevada, United States) As per DelveInsight's assessment, globally, Beta Thalassemia pipeline constitutes 22+ key companies continuously working towards developing 22+ Beta Thalassemia treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight.

"Beta Thalassemia Pipeline Insight, 2025 [https://www.delveinsight.com/sample-request/beta-thalassemia-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]" report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Beta Thalassemia Market.

The Beta Thalassemia 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 Beta Thalassemia Pipeline Report:

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Companies across the globe are diligently working toward developing novel Beta Thalassemia treatment therapies with a considerable amount of success over the years.

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Beta Thalassemia companies working in the treatment market are Agios Pharmaceuticals, Vertex Pharmaceuticals, Pharmacosmos, Editas Medicine, HemaQuest Pharmaceuticals, Acceleron Pharma, Bristol-Myers Squibb, Hoffmann-La Roche, Sanofi, Novartis, EmeraMed, Pharmacosmos A/S, Celgene, DisperSol Technologies, LLC, Agios Pharmaceuticals, Shire, and others, are developing therapies for the Beta Thalassemia treatment

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Emerging Beta Thalassemia therapies in the different phases of clinical trials are- PYRUKYND (mitapivat), CTX001, SP-420, EDIT-301, dimethylbutyrate, ACE-536, Luspatercept, Bitopertin, Deferitrin (GT56-252), ICL670, Emeramide, SP-420, Luspatercept, DST-0509, Mitapivat, SPD602, and others are expected to have a significant impact on the Beta Thalassemia market in the coming years.

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In July 2025, Vertex Pharmaceuticals' Casgevy (exagamglogene autotemcel) demonstrated sustained benefits in sickle cell disease (SCD) for 5.5 years and in transfusion-dependent beta-thalassemia (TDT) for six years. Data from three ongoing Phase III trials-CLIMB-111 (NCT03655678), CLIMB-121 (NCT03745287), and CLIMB-131 (NCT04208529)-show that all 45 SCD patients treated with Casgevy achieved at least 12 consecutive months without hospitalization, averaging 36.1 months. Additionally, 95.6% remained free from vaso-occlusive crises (VOCs) for at least 12 months, with an average duration of 35 months. Among TDT patients, 98.2% of 55 participants achieved transfusion independence for at least 12 consecutive months, maintaining a weighted average hemoglobin of greater than or equal to 9 g/dL, with a mean duration of 40.5 months.

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In June 2025, Vertex Pharmaceuticals' Casgevy (exagamglogene autotemcel) has shown long-term efficacy, with benefits lasting 5.5 years in sickle cell disease (SCD) and six years in transfusion-dependent beta-thalassemia (TDT). Results from three ongoing Phase III studies-CLIMB-111 (NCT03655678), CLIMB-121 (NCT03745287), and CLIMB-131 (NCT04208529)-revealed that all 45 SCD patients achieved at least 12 consecutive months free from hospitalization, with an average of 36.1 months. Moreover, 95.6% of these patients remained free from vaso-occlusive crises (VOCs) for at least a year, averaging 35 months. In TDT, 98.2% of 55 patients reached transfusion independence for at least 12 consecutive months, maintaining hemoglobin levels of greater than or equal to 9 g/dL, with a mean duration of 40.5 months.

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In January 2025, Agios Pharmaceuticals (AGIO) announced that the FDA has accepted its supplemental new drug application (sNDA) to expand the label of Pyrukynd, its sole marketed drug, to include the treatment of thalassemia.

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In January 2025, YolTech Therapeutics is preparing to launch a clinical trial for YOLT-204, its in vivo gene-editing therapy designed to treat transfusion-dependent beta-thalassemia (TDT). TDT is a genetic blood disorder caused by mutations in the beta-globin gene, leading to reduced or absent hemoglobin production. Patients with this condition require regular blood transfusions to manage anemia and prevent future complications.

Beta Thalassemia Overview

Beta thalassemia is a genetic blood disorder that reduces the production of hemoglobin, a protein in red blood cells that carries oxygen throughout the body. It results from mutations in the HBB gene, which provides instructions for making the beta-globin subunit of hemoglobin. This disorder leads to a deficiency in the beta-globin chains, causing the red blood cells to become malformed and break down prematurely, leading to anemia.

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Emerging Beta Thalassemia Drugs Under Different Phases of Clinical Development Include:

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PYRUKYND (mitapivat): Agios Pharmaceuticals

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CTX001: Vertex Pharmaceuticals

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SP-420: Pharmacosmos

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EDIT-301: Editas Medicine

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dimethylbutyrate: HemaQuest Pharmaceuticals

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ACE-536: Acceleron Pharma

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Luspatercept: Bristol-Myers Squibb

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Bitopertin: Hoffmann-La Roche

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Deferitrin (GT56-252): Sanofi

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ICL670: Novartis

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Emeramide: EmeraMed

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SP-420: Pharmacosmos A/S

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Luspatercept: Celgene

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DST-0509: DisperSol Technologies, LLC

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Mitapivat: Agios Pharmaceuticals

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SPD602: Shire

Beta Thalassemia Route of Administration

Beta Thalassemia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs, such as

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Oral

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Parenteral

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Intravenous

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Subcutaneous

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Topical

Beta Thalassemia Molecule Type

Beta Thalassemia Products have been categorized under various Molecule types, such as

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Monoclonal Antibody

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Peptides

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Polymer

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Small molecule

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Gene therapy

Beta Thalassemia Pipeline Therapeutics Assessment

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Beta Thalassemia Assessment by Product Type

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Beta Thalassemia By Stage and Product Type

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Beta Thalassemia Assessment by Route of Administration

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Beta Thalassemia By Stage and Route of Administration

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Beta Thalassemia Assessment by Molecule Type

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Beta Thalassemia by Stage and Molecule Type

DelveInsight's Beta Thalassemia Report covers around 22+ products under different phases of clinical development like

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Late-stage products (Phase III)

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Mid-stage products (Phase II)

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Early-stage product (Phase I)

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Pre-clinical and Discovery stage candidates

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Discontinued & Inactive candidates

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Route of Administration

Further Beta Thalassemia product details are provided in the report. Download the Beta Thalassemia pipeline report to learn more about the emerging Beta Thalassemia therapies [https://www.delveinsight.com/sample-request/beta-thalassemia-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]

Some of the key companies in the Beta Thalassemia Therapeutics Market include:

Key companies developing therapies for Beta Thalassemia are - Ionis Pharmaceuticals, Inc., Agios Pharmaceuticals, Inc., Silence Therapeutics plc, Phoenicia Biosciences, Shanghai BDgene, Beam Therapeutics, EmeraMed, CRISPR Therapeutics, CSL Vifor, EdiGene (GuangZhou) Inc., Regenacy Pharmaceuticals, Editas Medicine, Fulcrum Therapeutics, Allife Medical Science and Technology, Global Blood Therapeutics, Inc, Orchard Therapeutics, Acceleron Pharma, Disc Medicine, BRL Medicine, Celgene, and others.

Beta Thalassemia Pipeline Analysis:

The Beta Thalassemia pipeline report provides insights into

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The report provides detailed insights about companies that are developing therapies for the treatment of Beta Thalassemia with aggregate therapies developed by each company for the same.

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It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Beta Thalassemia Treatment.

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Beta Thalassemia key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

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Beta Thalassemia 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.

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Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Beta Thalassemia 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 Beta Thalassemia drugs and therapies [https://www.delveinsight.com/sample-request/beta-thalassemia-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]

Beta Thalassemia Pipeline Market Strengths

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Strong pipeline activity with potential Phase III and phase II emerging therapies.

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Increasing prevalence and awareness of beta thalassemia

Beta Thalassemia Pipeline Market Opportunities

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Lack of any curative treatment options despite the launch of gene therapies which are subject to patient outcome assessment

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Improved national blood policies

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Advancement in genetic engineering because gene therapy has been hypothesized to serve as an effective cure for monogenic blood disorders for several decades

Scope of Beta Thalassemia Pipeline Drug Insight

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Coverage: Global

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Key Beta Thalassemia Companies: Agios Pharmaceuticals, Vertex Pharmaceuticals, Pharmacosmos, Editas Medicine, HemaQuest Pharmaceuticals, Acceleron Pharma, Bristol-Myers Squibb, Hoffmann-La Roche, Sanofi, Novartis, EmeraMed, Pharmacosmos A/S, Celgene, DisperSol Technologies, LLC, Agios Pharmaceuticals, Shire, and others

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Key Beta Thalassemia Therapies: PYRUKYND (mitapivat), CTX001, SP-420, EDIT-301, dimethylbutyrate, ACE-536, Luspatercept, Bitopertin, Deferitrin (GT56-252), ICL670, Emeramide, SP-420, Luspatercept, DST-0509, Mitapivat, SPD602, and others

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Beta Thalassemia Therapeutic Assessment: Beta Thalassemia current marketed and Beta Thalassemia emerging therapies

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Beta Thalassemia Market Dynamics: Beta Thalassemia market drivers and Beta Thalassemia market barriers

Request for Sample PDF Report for Beta Thalassemia Pipeline Assessment and clinical trials [https://www.delveinsight.com/sample-request/beta-thalassemia-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]

Table of Contents

1. Beta Thalassemia Report Introduction

2. Beta Thalassemia Executive Summary

3. Beta Thalassemia Overview

4. Beta Thalassemia- Analytical Perspective In-depth Commercial Assessment

5. Beta Thalassemia Pipeline Therapeutics

6. Beta Thalassemia Late Stage Products (Phase II/III)

7. Beta Thalassemia Mid Stage Products (Phase II)

8. Beta Thalassemia Early Stage Products (Phase I)

9. Beta Thalassemia Preclinical Stage Products

10. Beta Thalassemia Therapeutics Assessment

11. Beta Thalassemia Inactive Products

12. Company-University Collaborations (Licensing/Partnering) Analysis

13. Beta Thalassemia Key Companies

14. Beta Thalassemia Key Products

15. Beta Thalassemia Unmet Needs

16 . Beta Thalassemia Market Drivers and Barriers

17. Beta Thalassemia Future Perspectives and Conclusion

18. Beta Thalassemia 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.

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