Drug intelligence / Profile preview

GalXC-Plus ALDH2

Development stage
Unknown
Lead developer
Novo Nordisk
Modality
MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Subcutaneous, Intrathecal, Intracisternal
01

Overview

GalXC-Plus ALDH2 is an investigational **RNA interference** construct from Dicerna designed as a **GalXC-Plus** RNAi molecule that silences **aldehyde dehydrogenase 2** messenger RNA. Publicly available information identifies it primarily as a **preclinical GalXC-Plus research construct** used to demonstrate that the GalXC-Plus platform can achieve deep and durable ALDH2 knockdown, including across the central nervous system in animal models. In reported mouse and non-human primate studies, a single dose of an unconjugated ALDH2-targeting GalXC-Plus molecule produced substantial target mRNA reduction for at least 28 days. The available evidence does **not** clearly show this exact construct advanced as a named clinical drug; rather, the better-defined ALDH2-directed clinical program from Dicerna was **DCR-AUD**, a distinct GalXC liver-targeted RNAi candidate for alcohol use disorder.

Other names
ALDH2-targeting GalXC-Plus moleculeALDH-2-targeting GalXC-Plus moleculeALDH 2-targeting GalXC-Plus moleculeGalXC-Plus molecule engineered to silence ALDH2
02

Targets

ALDH2 (Mitochondrial Aldehyde Dehydrogenase)

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