BIO-6553 |
BIO-6553 : Splicing modulator of HTT
Probe Summary
Selectivity
Potency
In Vivo
Control Compounds
Chemical Information
References
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Probe Summary
| Targets | Biochemical/Biophysical Potency | Cellular Potency |
|---|---|---|
| HTT |
|
Splicing modulator
up to 1 uM
Selectivity
In Cell Selectivity Assessment
Potency Assay Off-Target:
RNA-seq analysis in SH-SY5Y cells to identify both differentially expressed genes (DEGs) and differe ...
Selectivity Assessment Description:
At three times its IC50, BIO-6553 (23) significantly regulated 11 additional genes and induced 23 sp ...
In Vitro Selectivity Assessment
Potency Assay Off-Target:
Kinase off-targets of 23 were evaluated at 1 μM in a broad kinome panel (Eurofins: 468 kinases).
Selectivity Assessment Description:
Despite having multiple potential hinge binding motifs, no kinase showed greater than 80% inhibition ...
In Cell Selectivity Assessment
Potency Assay Off-Target:
RASL-seq assay (RNA-mediated oligonucleotide annealing, selection, and ligation sequencing) in SH-SY ...
Selectivity Assessment Description:
The RASL-seq panel was composed of off-target genes previously identified by genome-wide RNA-seq ana ...
Potency Cellular
In Vitro
HTT
Mode of Action: Splicing modulator
Structure-Activity-Relationship data available? Yes
DOI Reference: 10.1021/acs.jmedchem.6c00224
In Vivo Validations
Rat
Dose: 1 mg/Kg
Route of delivery:
Intravenous
Plasma half life:
1.7 h
Systemic clearance:
54 mL/min/kg, (u) 750 mL/min/kg
Area Under the Curve::
316 ng*h/mL
Volume of Distribution at Steady-State:
7.9 L/Kg
DOI Reference: 10.1021/acs.jmedchem.6c00224
Dose: 1 mg/Kg
Route of delivery:
Oral
Tmax:
1 h
Bioavailability:
65%
DOI Reference: 10.1021/acs.jmedchem.6c00224
Monkey (Cynomolgus)
Dose: 0.3 mg/Kg
Route of delivery:
Intravenous
Plasma half life:
4 h
Systemic clearance:
6.32 mL/min/kg, (u) 80 mL/min/kg
Area Under the Curve::
591 ng*h/mL
Volume of Distribution at Steady-State:
2.04 L/Kg
DOI Reference: 10.1021/acs.jmedchem.6c00224
Chemical Information
Coming Soon...