Pharmaceuticals - , ,
Seed & clinical stage RNAi platform technology biotech with lead candidates in fibrosis (CKD, NASH, PF) and dermatology (androgenetic alopecia, keloid) and an active and diverse pipeline in CNS, fibrosis, inflammatory, oncology, COVID19, and other areas.About SAMiRNA platform technology:• Delivery beyond the liver to inflamed, adipogenic, neoplastic tissues (in organs such as kidney, lung, skin, visceral fat, brain) via various RoA• Safe and efficacious based on human and animal studies (first human studies completed 2021)• Room temperature stable up to 1-year (this is RNAi!)• Cost effective manufacturing & QC• Strong IP (190+ patents)Lead candidate #1: amphisiran is a first in class RNAi candidate targeting AREG (amphiregulin) associated with numerous fibrosis, inflammatory, oncology indications. Our amphisiran lead indications are CKD (preclinical completed), IPF (preclinical completed), NASH, and obesity with preclinical in progress for psoriasis and pancreatic, ovarian, colorectal, breast, NSCL cancersLead candidate #2: CosmeRNA targets androgen recenptor (AR) and has shown excellent safety (no drug related AE, n=95) and best-in-class efficacy (comparable to Propecia) in human cosmeceutical trial completed in Korea (2021).
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