Drug intelligence / Profile preview

ISIS-GSK4Rx

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
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Overview

**ISIS-GSK4Rx** is an antisense oligonucleotide drug developed by Isis Pharmaceuticals (now Ionis Pharmaceuticals) in collaboration with GlaxoSmithKline (GSK) for an undisclosed ocular disease. As a Generation 2+ antisense drug, it binds to specific messenger RNA to inhibit the production of a disease-causing protein, offering a targeted approach to treat severe eye conditions where traditional therapies may fall short. In 2013, it was designated as a development candidate, earning Isis a $5 million milestone payment from GSK, with Isis responsible for advancing it to Phase 2 proof-of-concept, after which GSK holds an option to exclusively license it. Isis was also eligible for additional milestones and double-digit royalties on potential sales.[1][2][12]

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