Drug intelligence / Profile preview

QRX-203

Development stage
Preclinical
Lead developer
ProQR Therapeutics
Modality
Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
01

Overview

QRX-203 is an RNA-based therapeutic developed for the treatment of Alzheimer's disease. It is part of ProQR’s research pipeline, which focuses on single-stranded RNA (ssRNA) antisense molecules that are chemically modified to enable targeted repair of genetic mutations. These oligonucleotide therapies are intended to restore or modify protein function, although the precise molecular target and mechanism for QRX-203 in Alzheimer's disease have not been publicly disclosed. All ProQR antisense molecules, including QRX-203, are designed to be vector-free and are manufactured via relatively simple, scalable processes[1][4].

02

Targets

Amyloid precursor protein mRNA

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