Drug intelligence / Profile preview

ugt1a1 antisense morpholino oligonucleotide

Development stage
Preclinical
Lead developer
GeneTools
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
Intravenous
01

Overview

UGT1A1 antisense morpholino oligonucleotide is a synthetic antisense agent belonging to the phosphorodiamidate morpholino oligonucleotide (PMO) class. It is designed to specifically target and inhibit the translation of the UDP-glucuronosyltransferase 1-1 (UGT1A1) enzyme, which is responsible for the glucuronidation and subsequent excretion of bilirubin. By reducing UGT1A1 activity, the drug induces a controlled state of physiological unconjugated hyperbilirubinemia. This elevation in bilirubin levels provides therapeutic benefits due to bilirubin's role as a potent endogenous antioxidant and anti-inflammatory signaling molecule. In preclinical models of cyclosporine A (CsA)-induced chronic kidney disease, the administration of this morpholino has been shown to protect renal function, activate the nuclear receptor PPAR-α, and downregulate pro-inflammatory and injury markers such as NF-κB, Kim-1, and NGAL.

Other names
UGT1A1 antisense oligonucleotideUGT-1A1 antisense oligonucleotideUGT 1A1 antisense oligonucleotideUGT1A1 PMOUGT-1A1 PMOUGT 1A1 PMOUGT1A1 morpholinoUGT-1A1 morpholinoUGT 1A1 morpholino
02

Targets

UGT1A1 (UDP-glucuronosyltransferase 1A1)

Beyond the preview

Go deeper on ugt1a1 antisense morpholino oligonucleotide.

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Clinical trials

Full profile access

Follow clinical development from study design and recruitment through results.

  • Trial phase
  • Status
  • Readouts

Indications & development

Full profile access

Explore development by indication, patient population, and geography.

  • Indications
  • Development status
  • Countries

Licensing & deals

Full profile access

Trace asset ownership, licensing agreements, and commercial partnerships.

  • Partners
  • Deal terms
  • Milestones

Patents & exclusivity

Full profile access

Explore the patent landscape and regulatory exclusivity around an asset.

  • Patents
  • Expiration dates
  • Exclusivity

Competitive landscape

Full profile access

Compare development programs by target, modality, and indication.

  • Competing assets
  • Targets
  • Development stage

Research & analysis

Full profile access

Connect source evidence and development news to your research questions.

  • Publications
  • News
  • Analysis

Bring the full picture into focus.

See how Gosset can support your research on ugt1a1 antisense morpholino oligonucleotide.

Explore the full profile

Gosset Free

Get started with Gosset.

Enter your work email and we’ll be in touch with next steps.

Work email preferred.

Book a call