VivoSeq
Hematology

Thalassemia

70

Pipeline Programs↗

16

Active Trials↗

31

Companies Involved↗

Thalassemia is a moderately active landscape with 70 programs across 31 companies. The most active players are Hangzhou Zede Pharma-Tech Co., Ltd., Sideris Pharmaceuticals, CorrectSequence Therapeutics Co., Ltd. 20 therapies are approved with 6 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

65/100

Pipeline Velocity

6/100

Mech. Concentration

10/100

Completion Rate

86/100

Attrition Rate

9/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

H

Hangzhou Zede Pharma-Tech Co., Ltd.

1 program

Phase 1S

Sideris Pharmaceuticals

1 program

Phase 1C

CorrectSequence Therapeutics Co., Ltd

1 program

Phase 1P

Prism Pharmaceuticals

1 program

Phase 1C

Cardurion Pharmaceuticals, Inc.

1 program

Phase 2K

Kind Pharmaceuticals LLC

1 program

Phase 2

Pipeline

Programs by Phase

Preclinical12

Luspatercept

Bristol-Myers Squibb

Cyclophosphamide

Baxter Healthcare Corporation

Busulfan

Akero Therapeutics

Campath 1h

Mario Negri Institute for Pharmacological Research

Interleukin 2

Essen Biotech

Phase 16

Abatacept

Bristol-Myers Squibb

Sirolimus

Prism Pharmaceuticals

SP-420

Sideris Pharmaceuticals

Colla Corii Asini

The First Affiliated Hospital, Guangzhou University of Traditional Chinese Medicine

CS-101

CorrectSequence Therapeutics Co., Ltd

Phase 226

Alemtuzumab

Genzyme, a Sanofi Company

Glutamine

Sanofi

Sotatercept

Merck & Co.

Lidocaine Hydrochloride

Citius Pharmaceuticals, Inc.

Decitabine

Sun Pharma

Phase 36

Amlodipine

Viatris Specialty LLC

Silymarin

Bukwang Pharmaceutical

CTX001

Vertex Pharmaceuticals

Thalidomide and Hydroxyurea

National Institute of Blood and Marrow Transplant (NIBMT), Pakistan

Hydroxyurea and Thalidomide

Children's Hospital Karachi

Approved20

Deferasirox

Novartis

Deferiprone

Chiesi Farmaceutici S.p.A.

Thalidomide

Bristol-Myers Squibb

Tacrolimus

Veloxis Pharmaceuticals

Melphalan

Apotex Inc.

Analysis

Modality Breakdown

87%

Small molecule

67 of 77 classified programs — the dominant modality here.

13%ALL OTHERSProtein / enzyme45%Monoclonal antibody23%Cell therapy23%Vaccine23%

Small molecule leads; the ring shows all 77 classified programs, each share of the total. Modality classified for 100% of 85 pipeline programs.

Analysis

Trial Status

88

clinical trials

Completed49 · 56%
Unknown status15 · 17%
Recruiting10 · 11%
Terminated6 · 7%
Active, not recruiting4 · 5%
Withdrawn2 · 2%
Other2 · 2%

Clinical Trials

Trial Activity

88 trials

Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients
Recruiting·—·Phase 1·NCT05745532·10 enrolled
the Safety and Efficacy Evaluation of HGI-002 Injection in Patients With Transfusion-Dependent α-Thalassemia
Recruiting·—·Phase 1·NCT05851105·3 enrolled
Clinical Study of Super Transplantation in the Treatment of Severe β-thalassemia
Recruiting·GGuangzhou Women and Children's Medical Center·N/A·NCT06734520·3 enrolled
the Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia
Recruiting·—·Phase 1·NCT05864170·3 enrolled
A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia
Recruiting·MMabwell (Shanghai) Bioscience Co., Ltd.·Phase 1·NCT06772766·40 enrolled
A Study of Safety and Efficiency of AND017 in Patients With β-thalassemia
Recruiting·KKind Pharmaceuticals LLC·Phase 2·NCT06302491·64 enrolled
Β-Thalassemia Treatment with KL003 Cell Injection
Recruiting·KKanglin Biotechnology (Hangzhou) Co., Ltd.·Phase 1/2·NCT06280378·41 enrolled
Thal-Fabs: Reduced Toxicity Conditioning for High Risk Thalassemia
Recruiting·TThe Hospital for Sick Children·Phase 1/2·NCT05426252·20 enrolled
Efficacy of Combination of Hdroxyurea and Thalidomide Over Either Hydroxyurea or Thalidomide Alone in the Treatment of Transfusion Dependent Thalassemia in Children: A Quasi-Randomised Clinical Trial
Recruiting·—·Phase 4·NCT06299670·90 enrolled
Evaluation of Recombinant Humanized Anti-CD25 Monoclonal Antibody for Preventing Graft-versus-host Disease After Haploidentical/matched Unrelated Donor Hematopoietic Stem Cell Transplantation in Patients with Transfusion-dependent Thalassemia
Recruiting·—·Phase 2·NCT06657391·396 enrolled
A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.
Active, not recruiting·Bristol-Myers Squibb logoBristol-Myers Squibb·Phase 2·NCT05567458·94 enrolled
A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia
Active, not recruiting·AAgios Pharmaceuticals, Inc.·Phase 2·NCT03692052·20 enrolled
Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major
Active, not recruiting·—·Phase 1·NCT06065189·5 enrolled
A Real-World Study of β-Thalassemia Major Treatment With Luspatercept in Taiwan
Active, not recruiting·Bristol-Myers Squibb logoBristol-Myers Squibb·N/A·NCT06596642·61 enrolled
Safety and Efficacy of Hemoglobin F Inducers in Patients With Beta Thalassemia: a Prospective 12 Months Study
Not yet recruiting·RRiphah International University·Preclinical·NCT07673302·240 enrolled
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)