VivoSeq
Hematology

Sickle Cell Disease

238

Pipeline Programs↗

95

Active Trials↗

93

Companies Involved↗

Sickle Cell Disease is a highly competitive landscape with 238 programs across 93 companies. The most active players are ApoPharma, Takeda, Fulcrum Therapeutics. 31 therapies are approved with 40 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

85/100

Pipeline Velocity

50/100

Mech. Concentration

5/100

Completion Rate

67/100

Attrition Rate

20/100

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.

A

ApoPharma

1 program

Phase 1Takeda logo

Takeda

1 program

Phase 1F

Fulcrum Therapeutics

1 program

Phase 1N

NKT Therapeutics

1 program

Phase 1K

Karuna Therapeutics, Inc., a Bristol Myers Squibb company

1 program

Phase 1H

Hyundai Pharmaceutical Co., LTD.

1 program

Phase 1

Pipeline

Programs by Phase

Analysis

Modality Breakdown

91%

Small molecule

222 of 243 classified programs — the dominant modality here.

9%ALL OTHERSMonoclonal antibody115%Protein / enzyme31%Peptide31%Cell therapy31%Vaccine1<1%

Small molecule leads; the ring shows all 243 classified programs, each share of the total. Modality classified for 98% of 273 pipeline programs.

Analysis

Trial Status

303

clinical trials

Completed130 · 43%
Recruiting51 · 17%
Terminated50 · 17%
Active, not recruiting31 · 10%
Withdrawn12 · 4%
Not yet recruiting12 · 4%
Other17 · 6%

Clinical Trials

Trial Activity

303 trials

Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease
Recruiting·EEmory University·Phase 1/2·NCT02447874·21 enrolled
Cannabidiol in Sickle Cell Disease
Recruiting·IIcahn School of Medicine at Mount Sinai·Phase 1/2·NCT06930703·52 enrolled
A Safety, Efficacy, and Pharmacokinetic (PK) Study of HBI-002, an Oral Carbon Monoxide (CO) Therapeutic, in Subjects With Sickle Cell Disease (SCD)
Recruiting·HHillhurst Biopharmaceuticals, Inc.·Phase 1/2·NCT06144749·9 enrolled
Efficacy Safety Study of Gene Therapy for Sickle Cell DiseaseSCD Using Autologous CD34+ Cells Transduced ex Vivo, Carrying a Corrected Globin Gene and a Silencing RNA.
Recruiting·AAssistance Publique - Hôpitaux de Paris·Phase 1/2·NCT07432867·15 enrolled
Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients with Severe Sickle Cell Disease
Recruiting·MMark Walters, MD·Phase 1/2·NCT04774536·9 enrolled
A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)
Recruiting·MMemorial Sloan Kettering Cancer Center·Phase 2·NCT05736419·24 enrolled
A Phase 2 Open-label Study to Evaluate the Activity of Etavopivat on Transcranial Doppler Velocities in Pediatric Patients With Sickle Cell Disease Who Are at Increased Risk for Primary Stroke
Recruiting·FForma Therapeutics, Inc.·Phase 2·NCT05953584·27 enrolled
Safety of Anumigilimab (CSL324) in Adults With Sickle Cell Disease (SCD)
Recruiting·CSL Behring logoCSL Behring·Phase 2·NCT07224360·63 enrolled
Relationship Between Abnormal Myocardial Perfusion and Diastolic Dysfunction in Sickle Cell Disease Using PET
Recruiting·SSt. Jude Children's Research Hospital·Phase 2·NCT05583721·40 enrolled
Early Screening and Treatment of Heart Complication in Sickle Cell Disease
Recruiting·IInova Health Care Services·Phase 2·NCT07023666·100 enrolled
Gene Editing For Sickle Cell Disease
Recruiting·SSt. Jude Children's Research Hospital·Phase 1·NCT06506461·25 enrolled
Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease
Recruiting·—·Phase 1·NCT01245179·18 enrolled
131I-apamistamab-based Conditioning for Hematopoietic Stem Cell Transplant (HSCT) in Advanced Sickle Cell Disease (SCD)
Recruiting·—·Phase 1·NCT07015684·24 enrolled
Safety, Efficacy, and Pharmacokinetics of CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis
Recruiting·CSL Behring logoCSL Behring·Phase 2·NCT06699849·70 enrolled
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
Recruiting·Vertex Pharmaceuticals logoVertex Pharmaceuticals·Phase 3·NCT05477563·26 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)