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Phase 2a Study of ISIS 702843 in Non-Transfusion-Dependent Thalassemia
2019-003505-96Phase 2Completed
- Sponsor
- Ionis Pharmaceuticals
- Start
- Jan 2020
- Modality
- Gene Therapies, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
01
Overview
Phase 2, randomised, open-label trial of sapablursen in Thalassaemia (3 cohorts), sponsored by Ionis Pharmaceuticals. Primary endpoint: Primary efficacy endpoint at Week 27. Started Jan 2020; status: completed.
02
Drugs
sapablursen
03
Conditions
Thalassaemia
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