Drug intelligence / Profile preview

TTX-3360

Development stage
Unknown
Lead developer
Triplet Therapeutics
Modality
MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, 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, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intracerebroventricular
01

Overview

TTX-3360 is an investigational **antisense oligonucleotide** developed by **Triplet Therapeutics** for **Huntington's disease** and potentially other repeat expansion disorders. The drug is designed to reduce expression of **MSH3**, a DNA mismatch repair protein in the DNA damage response pathway that contributes to somatic expansion of pathogenic CAG repeats. By lowering MSH3 expression, TTX-3360 is intended to slow or halt repeat expansion biology believed to drive disease progression in Huntington's disease. Reported preclinical work showed durable knockdown in relevant brain regions after **intracerebroventricular administration**, supporting development in presymptomatic and early manifest Huntington's disease, with broader potential across other central nervous system repeat expansion disorders.

02

Targets

MSH3 (DNA mismatch repair protein Msh3)

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