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UNC13A ASO

Development stage: Preclinical
Lead developer: AcuraStem
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: Intravenous, Intramuscular, Intraperitoneal, Intracerebrospinal, Subcutaneous

Overview

UNC13A ASO is an antisense oligonucleotide (ASO) therapeutic developed by AcuraStem using its iNeuroRx platform. It is designed to restore normal UNC13A protein function, which is compromised due to TDP-43 pathology in a majority of amyotrophic lateral sclerosis (ALS) and about half of frontotemporal dementia (FTD) cases. The development of this drug, also known as AS-241, is supported by a $4 million grant from the California Institute for Regenerative Medicine (CIRM) to advance its preclinical program towards clinical trials for ALS and FTD.

Targets

UNC13A (Unc-13 homolog A)

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