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TRCN-1023

Development stage: Phase 2
Lead developer: Trace Neuroscience
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
Administration: Intrathecal

Overview

TRCN-1023 is an antisense oligonucleotide (ASO) designed to restore the expression of the UNC13A protein, which is frequently depleted in patients with amyotrophic lateral sclerosis (ALS) due to TDP-43-mediated cryptic splicing. Developed by Trace Neuroscience, TRCN-1023 targets the mis-spliced mRNA to ensure the production of functional UNC13A, a protein essential for neurotransmitter release and synaptic integrity. The drug is currently being investigated in the Phase 1/2 FUNCtion ALS clinical trial to evaluate its safety, tolerability, and potential to slow disease progression in people living with ALS.

Other names: UNC13A-restoring ASOUNC-13A-restoring ASOUNC 13A-restoring ASO

Targets

UNC13A (Unc-13 homolog A)

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