Chemical Probes logo
BIO-6553 | BIO-6553 : Splicing modulator of HTT
RATINGS:
Cellular Use: (3 reviews)

In Model Organisms: (3 reviews)
Probe Summary
Selectivity
Potency
In Vivo
Control Compounds
Chemical Information
References
Vendors
Download

Probe Summary

Targets Biochemical/Biophysical Potency Cellular Potency
HTT
    • IC50:54 nM
    • IC50:56 nM
    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...

    References

    Publications

    Cross References

    Expert Reviews

    by SERP
    (on 15 Apr 2026)
    Cellular Use Rating
    In Model Organisms
    (The reviewer did not leave any public comments)
    by SERP
    (on 18 Apr 2026)
    Cellular Use Rating
    In Model Organisms
    BIO-6553 is one of the best molecules to evaluate the effect of alternative splicing modulation to introduce a pseudoexon (49N) and suppress HTT expression levels, especially because of its selectivity...
    by SERP
    (on 2 May 2026)
    Cellular Use Rating
    In Model Organisms
    Overall, BIO-6553 is a good quality probe for cellular work that has demonstrated excellent activity against HTT splicing in iPSC (54 nM). It also shows good peripheral activity in vivo in mice when dosed...
    Note: The Chemical Probes Portal only endorses compounds as chemical probes for use as specific and selective modulators of the proposed target if they receive three or more (3-4) stars. Read more about our evaluation criteria