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IONIS-PTP-1BRx
- Development stage
- Phase 2
- Lead developer
- Ionis Pharmaceuticals
- Modality
- 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
- Administration
- Subcutaneous
01
Overview
IONIS-PTP-1BRx is a second-generation 2'-O-methoxyethyl antisense oligonucleotide designed to inhibit protein tyrosine phosphatase 1B (PTP1B), an enzyme that negatively regulates insulin and leptin signaling. By targeting the mRNA of PTP1B, this drug reduces its expression, thereby improving insulin sensitivity and glycemic control. In clinical studies, IONIS-PTP-1BRx demonstrated reductions in HbA1c, improved glycemic parameters, decreased leptin levels, increased adiponectin concentrations, and produced a distinct body weight–reducing effect in overweight or obese patients with type 2 diabetes inadequately controlled by metformin with or without sulfonylurea therapy. The drug was developed by Ionis Pharmaceuticals (formerly Isis Pharmaceuticals) as an antihyperglycaemic agent and insulin sensitizer[2][3][5][6].
- Other names
- Antisense PTP-1B gene therapy
02
Targets
PTPN1 (Protein tyrosine phosphatase 1B)
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