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ISIS PTP-1BRx
- Development stage
- Discontinued
- Lead developer
- Ionis Pharmaceuticals
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
ISIS PTP-1BRx (also known as IONIS-PTP1BRx or ISIS 404173) is a second-generation 2'-O-methoxyethyl (2'-MOE) modified antisense oligonucleotide designed to target and inhibit the expression of protein tyrosine phosphatase 1B (PTP-1B) mRNA. PTP-1B is a non-receptor tyrosine phosphatase that acts as a key negative regulator of both insulin and leptin receptor signaling pathways. By selectively downregulating PTP-1B expression via RNase H-mediated mRNA degradation, ISIS PTP-1BRx enhances insulin sensitivity and leptin signaling, promoting glycemic control and weight loss. Developed by Ionis Pharmaceuticals (formerly Isis Pharmaceuticals), the drug completed Phase 2 clinical trials for type 2 diabetes mellitus and obesity but development was subsequently discontinued.
- Other names
- Antisense PTP-1B gene therapy
02
Targets
PTPN1 (Protein tyrosine phosphatase 1B)
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