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ISIS 304309

Development stage: Preclinical
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

Overview

ISIS 304309 is an **antisense oligonucleotide** drug candidate designed to **modulate the expression of transthyretin (TTR)**, a liver-derived protein involved in the transport of thyroid hormone and retinol. Preclinical studies have shown that subcutaneous administration of ISIS 304309 can significantly reduce liver transthyretin mRNA levels, indicating potent downregulation of TTR synthesis[7]. This mechanism targets TTR-related diseases, such as familial amyloid polyneuropathy and related amyloidoses. ISIS 304309 was developed as part of a targeted RNA therapy approach.

Targets

TTR (Transthyretin)

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