PIPELINE

PIPELINE

Candidate Indications Delivery Platform Pre-clinical IND Enabling IND PHASE I PHASE II PHASE III
Amphisiran
(SRN-001)
Healthy Volunteers SAMiRNA
SRN-001 IPF SAMiRNA
SRN-001 DKD SAMiRNA
SRN-001 Cancer(HNSCC) SAMiRNA
SRN002 Osteoarthritis SAMiRNA

Clinical trials

SRN-001 Phase 1 Study

SRN-001 is a novel small interfering RNA (siRNA) drug being developed to treat fibrosis using Self Assembled Micelle inhibitory ribonucleic acid (SAMiRNA™) technology.
Amphiregulin (AREG) is a growth factor involved in fibroblast proliferation and myofibroblast transformation which is the hallmark of fibrosis in lung and kidney tissues. AREG is a downstream gene overexpressed by Transforming growth factor-β (TGF-β) during fibrosis, promoting fibroblast to myofibroblast transition (FMT). SRN-001 is designed to downregulate generating amphiregulin by RNA interference (RNAi). The goal of this clinical trial is to evaluate safety, tolerability, and pharmacokinetics in healthy participants. This trial is first-in-human clinical trial to develop SAMiRNA™ to utilize as therapeutic use.

Trial at a Glance
Trial ID SRN-001-C01
Condition Idiopathic Pulmonary Fibrosis
Drug/Treatment SRN-001
Does this trial use a placebo? Yes
Trial Type Interventional
Number of Participants 25 participants
Trial dates September 8, 2023 – September 25, 2024