Farabursen sodium (RGLS8429 sodium; RG1015 sodium) is a miR-17 inhibitor. Farabursen sodium inhibits the function of the miR-17 family, relieves the inhibitory effect on miR-17 target genes including PKD1 and PKD2, and increases the level of PC1/2. Farabursen sodium slows the growth of renal cysts, reduces the ratio of kidney weight to body weight, and decreases the cyst index and proliferation index. Farabursen sodium is applicable to research related to autosomal dominant polycystic kidney disease [1] [2].
In Vivo, Farabursen sodium (20 mg/kg, s.c., administered on postnatal days 8, 10, 12, and 15) significantly inhibits renal cyst growth and cell proliferation, and improves renal function in Pkd1F/RC ADPKD mice[2]. Farabursen sodium (0-300 mg/kg; s.c.; single dose) achieves sustained target binding to miR-17 in the kidneys of C57BL/6 mice (inhibition rate ≥75%, duration ≥14 days), and also achieves this effect in the liver (inhibition rate ≥75%, duration ≥7 days), with an EC75 of 2.2 μg/g in renal tissues[2].
In Vitro, Farabursen (sodium) (0.1-100 nM; 24 h) potently de-represses miR-17 activity in HeLa cells, with an EC50 of 22.40 nM after 24 h of incubation[2]. Farabursen (sodium) (0.1-100 nM; 24 h) de-represses PKD1-3'UTR activity in HeLa cells, with an EC50 of 43.54 nM after 24 h of incubation[2]. Farabursen (sodium) (0.1-100 nM; 24 h) de-represses PKD2-3'UTR activity in HeLa cells, with an EC50 of 40.76 nM after 24 h of incubation[2]. Farabursen (sodium) (120 nM) reduces 3D cyst size in primary human ADPKD cyst cells when transfected at 120 nM[2].
References:
[1]. Yu, Alan S.L.1, et al. Farabursen Increases Urinary Polycystin-1 and Polycystin-2 and Reduces Height-Adjusted Total Kidney Volume Growth in Patients with ADPKD: SA-OR089. Journal of the American Society of Nephrology 36(10S):10.1681/ASN.202527ntvtc3IF: 9.4 Q1, October 2025.
[2]. Valencia T, et al. The nucleobase guanine at the 3'-terminus of oligonucleotide RGLS4326 drives off-target AMPAR inhibition and CNS toxicity. Nat Commun. 2025;16(1):10762. Published 2025 Nov 28.
In Vivo, Farabursen sodium (20 mg/kg, s.c., administered on postnatal days 8, 10, 12, and 15) significantly inhibits renal cyst growth and cell proliferation, and improves renal function in Pkd1F/RC ADPKD mice[2]. Farabursen sodium (0-300 mg/kg; s.c.; single dose) achieves sustained target binding to miR-17 in the kidneys of C57BL/6 mice (inhibition rate ≥75%, duration ≥14 days), and also achieves this effect in the liver (inhibition rate ≥75%, duration ≥7 days), with an EC75 of 2.2 μg/g in renal tissues[2].
In Vitro, Farabursen (sodium) (0.1-100 nM; 24 h) potently de-represses miR-17 activity in HeLa cells, with an EC50 of 22.40 nM after 24 h of incubation[2]. Farabursen (sodium) (0.1-100 nM; 24 h) de-represses PKD1-3'UTR activity in HeLa cells, with an EC50 of 43.54 nM after 24 h of incubation[2]. Farabursen (sodium) (0.1-100 nM; 24 h) de-represses PKD2-3'UTR activity in HeLa cells, with an EC50 of 40.76 nM after 24 h of incubation[2]. Farabursen (sodium) (120 nM) reduces 3D cyst size in primary human ADPKD cyst cells when transfected at 120 nM[2].
References:
[1]. Yu, Alan S.L.1, et al. Farabursen Increases Urinary Polycystin-1 and Polycystin-2 and Reduces Height-Adjusted Total Kidney Volume Growth in Patients with ADPKD: SA-OR089. Journal of the American Society of Nephrology 36(10S):10.1681/ASN.202527ntvtc3IF: 9.4 Q1, October 2025.
[2]. Valencia T, et al. The nucleobase guanine at the 3'-terminus of oligonucleotide RGLS4326 drives off-target AMPAR inhibition and CNS toxicity. Nat Commun. 2025;16(1):10762. Published 2025 Nov 28.