General
Preferred name
ATALUREN
Synonyms
PTC124 ()
PTC-124 ()
PTC124 (Ataluren) ()
Ataluren (PTC124) ()
Translarna ()
P&D ID
PD009339
CAS
775304-57-9
Tags
nuisance
drug
available
Approved by
EMA
First approval
2014
Drug Status
investigational
approved
Drug indication
Aniridia
Duchenne dystrophy
Muscular dystrophy
Dravet syndrome
Cystic fibrosis
Mucopolysaccharidosis
Max Phase
Phase 4
Probe control
Probe control not defined
Orthogonal probes
0
No orthogonal probes found
Similar probes
0
No structurally similar probes found
Structure
P&D probe-likeness score
Structure formats
[[ format ]]
[[ compound[format === 'MOL' ? 'molblock' : format.toLowerCase()] ]]
Description
(extracted from source data)
DESCRIPTION
Ataluren is a compound in an emerging class of chemicals which induce ribosomal readthrough. This developing pharmaceutical intervention has been termed 'readthrough strategy'. Clinically, this type of compound has potential benefit in the treatment of genetic disorders associated with loss of functional protein due to nonsense or PTC mutations, such as certain types of muscular dystrophy, cystic fibrosis and many cancers .
(GtoPdb)
MOA
Ataluren enables ribosomal readthrough of mRNA containing premature stop codons that otherwise would result in premature termination of protein chains. Use of ataluren allows cellular machinery to bypass nonsense mutations in genetic material, continue the translation process, and thereby restore the production of a full-length, functional protein.; ; The research on the effects of Ataluren on the translation and stability of nonsense-containing mRNA in vitor show that Ataluren promoted readthrough at each of the nonsense codons, showing maximal activity with UGA, while having no effect on mRNA levels. Unlike the stable cell line assays, Ataluren did not discriminate significantly between the UAG and UAA mRNAs. Ataluren was a more potent nonsense-suppressing agent than gentamicin, and exhibited 4- to 15-fold stimulation of in vitro readthrough relative to the controls at levels similar to those in the stable cell reporter assays. These results indicate that Ataluren modulates termination efficiency at premature nonsense codons.
[[ p.pathway_name ]]
[[ compound.targets[tid].gene_name ]]
Compound Sets
17
AdooQ Bioactive Compound Library
Cayman Chemical Bioactives
ChEMBL Approved Drugs
ChEMBL Drugs
Drug Repurposing Hub
DrugBank
DrugBank Approved Drugs
DrugCentral
DrugCentral Approved Drugs
DrugMAP
Guide to Pharmacology
MedChem Express Bioactive Compound Library
Novartis Chemogenetic Library (NIBR MoA Box)
Nuisance compounds in cellular assays
ReFrame library
Selleckchem Bioactive Compound Library
TargetMol Bioactive Compound Library
[[ a.name ]]
[[ ligand_id ]]
free of charge
External IDs
32
Molecular Weight
284.06
Hydrogen Bond Acceptors
4
Hydrogen Bond Donors
1
Rotatable Bonds
3
Ring Count
3
Aromatic Ring Count
3
cLogP
3.24
TPSA
76.22
Fraction CSP3
0.0
Chiral centers
0.0
Largest ring
6.0
QED
0.8
Structural alerts
1
Luciferase inhibiton
FLuc
Nuisance compounds in cellular assays
Related compounds
Custom attributes
(extracted from source data)
Pathway
Membrane Transporter/Ion Channel
Autophagy
Target
CFTR
DMD
Member status
member
MOA
Putative translational readthrough (TRT) molecule
Nonsense Mutation Suppressors
modulator of translational readthrough
CFTR channel agonist, dystrophin stimulant
Indication
duchenne muscular dystrophy (DMD), cystic fibrosis
Disease Area
genetics, pulmonary
Source data
