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Sobetirome

From Wikipedia, the free encyclopedia
Chemical compound
Pharmaceutical compound
Sobetirome
Clinical data
Other namesGC-1
Routes of
administration		Oral
Legal status
Legal status
  • Investigational
Identifiers
  • 2-[4-[(4-Hydroxy-3-propan-2-ylphenyl)methyl]-3,5-dimethylphenoxy]acetic acid
CAS Number
PubChemCID
IUPHAR/BPS
DrugBank
ChemSpider
UNII
KEGG
ChEBI
ChEMBL
CompTox Dashboard(EPA)
Chemical and physical data
FormulaC20H24O4
Molar mass328.408 g·mol−1
3D model (JSmol)
  • CC1=CC(=CC(=C1CC2=CC(=C(C=C2)O)C(C)C)C)OCC(=O)O
  • InChI=1S/C20H24O4/c1-12(2)17-9-15(5-6-19(17)21)10-18-13(3)7-16(8-14(18)4)24-11-20(22)23/h5-9,12,21H,10-11H2,1-4H3,(H,22,23)
  • Key:QNAZTOHXCZPOSA-UHFFFAOYSA-N

Sobetirome (GC-1) is athyromimetic drug that binds to thethyroid hormone receptor TRβ1 preferentially compared to TRα1. It has been investigated for the treatment ofdyslipidemia, obesity,[1]Pitt–Hopkins syndrome,[2]cholestatic liver disease,[3]multiple sclerosis,[4]bleomycin-inducedlung fibrosis,[5] andCOVID-19 causedARDS.[6] It was designated as anorphan drug by the FDA for the treatment ofX-linked adrenoleukodystrophy.[7]

References

[edit]
  1. ^Lammel Lindemann, Jan; Webb, Paul (2016)."Sobetirome: the past, present and questions about the future".Expert Opinion on Therapeutic Targets.20 (2):145–149.doi:10.1517/14728222.2016.1090429.ISSN 1744-7631.PMID 26565124.
  2. ^Bohlen, Joseph F; Cleary, Colin M; Das, Debamitra; Sripathy, Srinidhi Rao; Sadowski, Norah; Shim, Gina; Kenney, Rakaia F; Buchler, Ingrid P; Banerji, Tapasree; Scanlan, Thomas S; Mulkey, Daniel K; Maher, Brady J (1 August 2023)."Promyelinating drugs promote functional recovery in an autism spectrum disorder mouse model of Pitt–Hopkins syndrome".Brain.146 (8):3331–3346.doi:10.1093/brain/awad057.PMC 10393406.PMID 37068912.
  3. ^Kosar, Karis; Cornuet, Pamela; Singh, Sucha; Liu, Silvia; Nejak-Bowen, Kari (May 2020)."The Thyromimetic Sobetirome (GC-1) Alters Bile Acid Metabolism in a Mouse Model of Hepatic Cholestasis".The American Journal of Pathology.190 (5):1006–1017.doi:10.1016/j.ajpath.2020.01.015.ISSN 0002-9440.PMC 7221298.PMID 32205094.
  4. ^Shah, Kamal; Chauhan, Durgesh Nandini; Chauhan, Nagendra Singh; Mishra, Pradeep (13 May 2020).Recent Advancement in Prodrugs. CRC Press.ISBN 978-1-000-05799-7.
  5. ^Matralis, Alexios; Karampitsakos, Theodoros; Papaioannou, Ourania; Katsaras, Matthaios; Sampsonas, Fotios; Bouros, Demosthenes; Aidinis, Vassilis; Tzouvelekis, Argyrios (5 September 2021)."Biosynthesis and implementation of Thyroid Receptor beta (TRß) agonists (thyromimetics) for the treatment of pulmonary fibrosis".European Respiratory Journal.58 (suppl 65) OA4334.doi:10.1183/13993003.congress-2021.OA4334.ISSN 0903-1936.S2CID 244680799.
  6. ^Hartley, Pamela."$100,000 Gift Speeds Exploration of Sobetirome as COVID-19 Treatment".medicine.yale.edu. Retrieved15 September 2023.
  7. ^"Search Orphan Drug Designations and Approvals".www.accessdata.fda.gov. Archived fromthe original on April 15, 2025. Retrieved15 September 2023.
Receptor
(ligands)
THRTooltip Thyroid hormone receptor
Agonists
Thyromimetics
(selective agonists)
Antagonists
Transporter
(blockers)
NISTooltip Sodium-iodide symporter
 
Enzyme
(inhibitors)
TPOTooltip Thyroid peroxidase
DIOTooltip Iodothyronine deiodinase
Others
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