Braxlosiran
Braxlosiran is a small nucleic acid that targets DUX4 (double homeobox protein) and can be used to synthesize AOC drugs, such as Delpacibart braxlosiran. Abnormal expression of the DUX4 protein leads to changes in gene expression in muscle cells, and these changes are associated with the loss of muscle function in facioscapulohumeral muscular dystrophy (FSHD).
Product Specifications
UNSPSC
12352208
Target
Antibody-Oligonucleotide Conjugates (AOCs)
Related Pathways
Antibody-drug Conjugate/ADC Related
Field of Research
Inflammation/Immunology
Smiles
[Braxlosiran]
References & Citations
[1]Compounds, pharmaceutical compositions, and methods of preparing compounds and of their use. WO2025177244A1
Shipping Conditions
Room temperature
Scientific Category
Oligonucleotides
Clinical Information
No Development Reported
Frequently Asked Questions
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