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FBT102

Development stage: Preclinical
Lead developer: Facio Biotherapies
Modality: Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics

Overview

FBT102 is a preclinical-stage RNA inhibitor (such as an antisense oligonucleotide) being developed by Facio Biotherapies for the treatment of facioscapulohumeral muscular dystrophy (FSHD). The therapeutic candidate is designed to target and silence an undisclosed FSHD-related toxic gene, thereby preventing the expression of the toxic protein that drives muscle degeneration in patients with FSHD.

Targets

DUX4 (Double Homeobox 4)

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