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QR-441A
- Development stage
- Discontinued
- Lead developer
- ProQR Therapeutics
- 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
- Inhalation
01
Overview
QR-441A is an investigational antisense oligonucleotide (ASO) developed by ProQR Therapeutics for the treatment of cystic fibrosis (CF). The therapeutic is designed to bind to the mRNA of the cystic fibrosis transmembrane conductance regulator (CFTR) gene to correct splicing errors or otherwise restore the production of functional CFTR protein. While many CF therapies target specific common mutations like F508del, QR-441A was part of a broader 'breadth' program aimed at addressing a wider range of rare CF-causing mutations. Development of the program was halted when ProQR Therapeutics strategically pivoted to focus on its Axiomer RNA editing technology and discontinued its internal cystic fibrosis pipeline.
02
Targets
F508del-CFTR (CFTR F508del)
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