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SEQUOIA
2018-003385-14Phase 2Completed
- Sponsor
- Arrowhead Pharmaceuticals
- Start
- Mar 2019
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → 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 trial in alpha-1 antitrypsin deficiency (AATD) comparing ARO-AAT with Placebo (2 arms). Primary endpoint: Histologic response without fibrosis worsening. Started Mar 2019; status: completed.
02
Drugs
fazirsiran
03
Conditions
alpha-1 antitrypsin deficiency (AATD)
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