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XCUR-FXN
- Development stage
- Discontinued
- Lead developer
- Exicure
- 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
01
Overview
XCUR-FXN is an investigational antisense oligonucleotide (ASO) therapeutic developed with Spherical Nucleic Acid (SNA) technology for the treatment of Friedreich's ataxia. It comprises two distinct oligonucleotides targeting frataxin (FXN) mRNA. One oligonucleotide prevents mRNA degradation in the nucleus, while the other stabilizes mRNA in the cytoplasm, increasing the production of frataxin protein, which is deficient in Friedreich's ataxia. Delivery is via intrathecal injection to effectively target the central nervous system. XCUR-FXN remained in preclinical development and the program was discontinued following internal misreporting of efficacy data[1][3][5][7][9].
- Other names
- XCUR-FXN
02
Targets
FXN (Frataxin)
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