Drug intelligence / Profile preview

CTRO-1013

Development stage
Preclinical
Lead developer
insitro
Modality
MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Subcutaneous
01

Overview

CTRO-1013 is a liver-targeted oligonucleotide therapeutic candidate being developed by insitro, in collaboration with Eli Lilly, for the treatment of metabolic dysfunction-associated steatotic liver disease (MASLD) and liver fibrosis. Identified using insitro's proprietary AI/ML-based drug discovery platform, the drug specifically targets insulin receptor substrate-1 (IRS1), a key protein involved in the regulation of hepatic de novo lipogenesis. By inhibiting the expression of IRS1, CTRO-1013 aims to reduce liver fat accumulation and prevent the progression of fibrosis. The therapeutic utilizes liver-targeting technology, likely incorporating GalNAc (N-Acetylgalactosamine) conjugation, to ensure selective delivery to hepatocytes. As of late 2025, the program is in the preclinical stage, advancing through IND-enabling studies in preparation for first-in-human clinical trials.

02

Targets

IRS1 (Insulin receptor substrate 1)

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