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FMR1 antisense oligonucleotide
- Development stage
- Preclinical
- Lead developer
- QurAlis
- 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, Intravenous, Subcutaneous, Intranasal
01
Overview
Quiver Bioscience is developing an antisense oligonucleotide (ASO) therapeutic targeting the *FMR1* gene for the treatment of Fragile X Syndrome (FXS). Developed in collaboration with QurAlis and supported by funding from the FRAXA Research Foundation, the therapeutic approach aims to correct faulty RNA splicing of the *FMR1* gene. This correction is intended to restore the production of functional Fragile X messenger ribonucleoprotein 1 (FMRP), which is deficient in individuals with Fragile X Syndrome. The program utilizes Quiver's proprietary Genomic Positioning System (GPS) platform, which integrates human neuronal models with AI/ML analytics to develop CNS-targeted genetic medicines. The asset is currently in preclinical development.
- Other names
- FMR1 ASOFMR-1 ASOFMR 1 ASOQuiver Fragile X program
02
Targets
FMR1 (Fragile X messenger ribonucleoprotein 1)
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