VivoSeq
Hematology

Beta-Thalassemia

42

Pipeline Programs↗

14

Active Trials↗

26

Companies Involved↗

Beta-Thalassemia is a moderately active landscape with 42 programs across 26 companies. The most active players are Prolong Pharmaceuticals, Sanofi, Protagonist Therapeutics. 10 therapies are approved with 6 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

59/100

Pipeline Velocity

11/100

Mech. Concentration

8/100

Completion Rate

83/100

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.

P

Prolong Pharmaceuticals

1 program

Phase 1Sanofi logo

Sanofi

2 programs

Phase 2Protagonist Therapeutics logo

Protagonist Therapeutics

1 program

Phase 2Regeneron logo

Regeneron

1 program

Phase 2C

Cardurion Pharmaceuticals, Inc.

1 program

Phase 2L

Lantu Biopharma

1 program

Phase 2

Pipeline

Programs by Phase

Preclinical5

Exa-Cel

Vertex Pharmaceuticals

Luspatercept

Bristol-Myers Squibb

Plerixafor

Genzyme, a Sanofi Company

Curcuminoids and Alpha-Tocopherol

Mahidol University

Spirulina

Tanta University

Phase 13

RG6026

Roche

Nigella Sativ

Tanta University

YOLT-204

First Affiliated Hospital of Guangxi Medical University

Phase 218

Epoetin Alfa

Johnson & Johnson Pharmaceutical Research & Development, L.L.C.

Sirolimus

Prism Pharmaceuticals

Bitopertin

Roche

SP-420

Sideris Pharmaceuticals

REGN7999

Regeneron

Phase 36

Alpha Lipoic Acid

National Center for Complementary and Integrative Health (NCCIH)

Mitapivat

Agios Pharmaceuticals, Inc.

CTX001

Vertex Pharmaceuticals

Iron Chelating Intervention

Tanta University

L_carnitine

Tanta University

Approved10

Deferoxamine

Alnylam Pharmaceuticals

Deferasirox

Novartis

Deferiprone

Chiesi Farmaceutici S.p.A.

Thalidomide

Bristol-Myers Squibb

Sildenafil

Viatris Specialty LLC

Analysis

Modality Breakdown

93%

Small molecule

38 of 41 classified programs — the dominant modality here.

7%ALL OTHERSCell therapy25%Protein / enzyme12%

Small molecule leads; the ring shows all 41 classified programs, each share of the total. Modality classified for 96% of 45 pipeline programs.

Analysis

Trial Status

44

clinical trials

Completed20 · 45%
Recruiting7 · 16%
Unknown status6 · 14%
Active, not recruiting4 · 9%
Withdrawn2 · 5%
Not yet recruiting2 · 5%
Other3 · 7%

Clinical Trials

Trial Activity

44 trials

A Real-World Study to Evaluate Luspatercept in Adults With Transfusion-Dependent Beta-Thalassemia in the Middle East
Recruiting·Bristol-Myers Squibb logoBristol-Myers Squibb·N/A·NCT07215975·200 enrolled
Effects of Hydroxyurea and Metformin in Transfusion Dependent Beta-Thalassemia
Recruiting·—·Phase 4·NCT06606886·96 enrolled
Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia
Recruiting·National Institute of Blood and Marrow Transplant (NIBMT), Pakistan·Phase 2·NCT06490627·54 enrolled
An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia
Recruiting·LLantu Biopharma·Phase 1/2·NCT06308159·6 enrolled
ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
Recruiting·—·Phase 1/2·NCT06364774·12 enrolled
A Phase 2a Study to Evaluate the Safety and Pharmacokinetics of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
Recruiting·Bristol-Myers Squibb logoBristol-Myers Squibb·Phase 2·NCT04143724·99 enrolled
Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants
Recruiting·National Institute of Allergy and Infectious Diseases (NIAID)·N/A·NCT03937817·300 enrolled
Long Term Beta Thalassemia Treatment: Findings From The Extension Period
Active, not recruiting·National Institute of Blood and Marrow Transplant (NIBMT), Pakistan·Phase 2·NCT06490601·30 enrolled
A PHASE 2, TWO-PART, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF SUBCUTANEOUSLY ADMINISTERED REGN7999 (AN INHIBITOR OF TMPRSS6) IN PARTICIPANTS WITH IRON OVERLOAD DUE TO NON-TRANSFUSION DEPENDENT β-THALASSEMIA
Active, not recruiting·Regeneron logoRegeneron·Phase 2·NCT06421636·19 enrolled
A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent β-Thalassemia
Active, not recruiting·Vertex Pharmaceuticals logoVertex Pharmaceuticals·Phase 3·NCT05356195·16 enrolled
CLIMB THAL-111: Exa-cel in Beta-Thalassemia
Active, not recruiting·CRISPR Therapeutics logoCRISPR Therapeutics·Phase 3·NCT04746027·42 enrolled
A Pilot Study on the Safety and Efficacy of YOLT-204 for Transfusion-Dependent Beta-Thalassemia
Not yet recruiting·FFirst Affiliated Hospital of Guangxi Medical University·Phase 1·NCT06678165·3 enrolled
Evaluating the Effect of N-Acetyl Cysteine and Alpha Lipoic Acid in Patients With Beta Thalassemia
Not yet recruiting·TTanta University·Phase 3·NCT07157722·66 enrolled
The BENeFiTS Trial in Beta Thalassemia Intermedia
Enrolling by invitation·PPhoenicia BioScience·Phase 1/2·NCT04432623·36 enrolled
Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia
Completed·Protagonist Therapeutics logoProtagonist Therapeutics·Phase 2·NCT03802201·63 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)