Braxlosiran is a component of the AOC reagent Delpacibart braxlosiran, consisting of an siRNA targeting DUX4 (double homeobox protein) and SMCC linker. Delpacibart braxlosiran binds to TfR1. Aberrant expression of the DUX4 protein causes changes in gene expression in muscle cells, which are associated with muscle function loss in facioscapulohumeral muscular dystrophy (FSHD) [1] [2].
In Vitro, Delpacibart braxlosiran binds to TfR1[1].
References:
[1]. Malecova B, et al. Development of a DUX4-targeting antibody oligonucleotide conjugate as a therapy for FSHD. Nucleic acids research. 2026 Apr 13;54(7):gkag301.
[2]. Philippe MOCHIRIAN. Compounds, pharmaceutical compositions, and methods of preparing compounds and of their use. WO2025177244A1. WIPO (PCT). 2025-02-21.
In Vitro, Delpacibart braxlosiran binds to TfR1[1].
References:
[1]. Malecova B, et al. Development of a DUX4-targeting antibody oligonucleotide conjugate as a therapy for FSHD. Nucleic acids research. 2026 Apr 13;54(7):gkag301.
[2]. Philippe MOCHIRIAN. Compounds, pharmaceutical compositions, and methods of preparing compounds and of their use. WO2025177244A1. WIPO (PCT). 2025-02-21.