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GalXC-Plus TUBB3
- Development stage
- Unknown
- Lead developer
- Novo Nordisk
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal
01
Overview
GalXC-Plus TUBB3 is a **preclinical RNA interference therapeutic candidate** built on Dicerna's GalXC-Plus platform and designed to silence **tubulin beta 3 class III mRNA** in neuronal cells within the central nervous system. The construct appears to be an siRNA-based GalXC-Plus molecule evaluated mainly as a **platform demonstration payload** rather than a named clinical development asset, with reported CNS-wide neuronal knockdown of **TUBB3** after intrathecal administration in rodent studies. Its intended mechanism is sequence-specific degradation of TUBB3 mRNA through the RNAi pathway, leading to reduced expression of class III beta-tubulin in neurons; available public information ties it to Dicerna's preclinical delivery and target-engagement work rather than to a disclosed disease-specific clinical program.
- Other names
- GalXC-Plus beta-tubulin IIIGalXC-Plus Tubb3
02
Targets
TUBB3 (Tubulin beta-3 chain)
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