Drug intelligence / Profile preview

VY1706

Development stage
Phase 1
Lead developer
Voyager Therapeutics
Modality
Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

VY1706 is an investigational, intravenously administered gene therapy being developed for the treatment of Alzheimer's disease. It combines a potent small interfering RNA (siRNA) construct designed to decrease the expression of tau protein with a blood-brain barrier-penetrant TRACER capsid, which is an engineered adeno-associated virus (AAV) vector. Preclinical studies in non-human primates have demonstrated that a single IV dose of VY1706 results in significant, dose-dependent knockdown of tau mRNA and protein across multiple brain regions affected by Alzheimer's disease, with effects sustained for at least three months post-dosing. The therapy achieves broad central nervous system distribution while minimizing off-target delivery to the liver. VY1706 is currently in IND-enabling studies, with clinical trial applications anticipated in 2026[1][3][5][6].

02

Targets

MAPT (Microtubule-associated protein tau)

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