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baliforsen
- Development stage
- Phase 2
- Lead developer
- Ionis Pharmaceuticals
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
Baliforsen (also known as ISIS 598769 or IONIS-DMPKRx) is an antisense oligonucleotide (ASO) developed by Ionis Pharmaceuticals in collaboration with Biogen for the treatment of myotonic dystrophy type 1 (DM1). The drug is designed to target the DMPK (DM1 protein kinase) mRNA transcript, specifically binding within exon 9 to promote RNase H1-mediated degradation of the toxic expanded CUG repeat-containing RNA. By reducing the levels of this toxic RNA, baliforsen aims to alleviate the sequestration of muscleblind-like (MBNL) proteins and restore normal alternative splicing. Although evaluated in a Phase 1/2a trial (NCT02312011), the program was discontinued for this specific candidate because skeletal muscle drug concentrations were insufficient to achieve the necessary target reduction for clinical efficacy.
- Other names
- baliforsen
02
Targets
CUG repeat RNA (Dystrophia myotonica protein kinase messenger RNA with expanded CUG repeats)
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