Thalassemia
Hematology
3
Pipeline Programs
3
Companies
2
Clinical Trials
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
0
0
0
0
2
Early DiscoveryClinical DevelopmentMarket
Drug Modality Breakdown
Small Molecule
2100%
+ 1 programs with unclassified modality
Competitive Landscape
2 companies ranked by most advanced pipeline stage
GS
Gilead SciencesFOSTER CITY, CA
2 programs2
sofosbuvir/velpatasvirPhase 4Small Molecule1 trial
sofosbuvir/velpatasvirPhase 4Small Molecule
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Gilead Sciencessofosbuvir/velpatasvir
Ionis PharmaceuticalsIONIS TMPRSS6-Lrx
Clinical Trials (2)
Total enrollment: 43 patients across 2 trials
Sofosbuvir/Ledipasvir for Hepatitis C Genotype 1-6 in Patients With Transfusion-Dependent Thalassemia: An Open Label Trial
Start: May 2017Est. completion: Aug 20187 patients
Phase 4Completed
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy Volunteers
Start: May 2017Est. completion: Sep 201836 patients
Phase 1Completed
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Phase Legend
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
Small Molecule is the dominant modality (100% of programs)
3 companies competing in this space