Drug intelligence / Profile preview

gataparsen

Development stage
Phase 2
Lead developer
Eli Lilly
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
Intravenous
01

Overview

**Gataparsen** (LY2181308) is a second-generation **antisense oligonucleotide (ASO)** that targets survivin mRNA, inhibiting its translation and downregulating survivin protein expression in tumor cells. Survivin, a member of the inhibitor of apoptosis (IAP) family (baculoviral IAP repeat-containing protein 5), is overexpressed in many cancers including lung, colon, breast, prostate, ovary, cervix, skin, and brain, but absent in most normal adult tissues, promoting cell survival and resistance to chemotherapy. Initially developed by **Ionis Pharmaceuticals** (formerly Isis Pharmaceuticals) in collaboration with **Eli Lilly**, it reached **Phase 2** trials for various solid tumors and hematologic malignancies but was discontinued overall due to lack of efficacy in key studies.

Other names
Gataparsen Sodium
02

Targets

BIRC5 (Survivin)

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