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BIIB132
- Development stage
- Discontinued
- Lead developer
- Biogen
- 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
- Intrathecal
01
Overview
BIIB132 is an investigational antisense oligonucleotide (ASO) drug developed by Biogen for the treatment of spinocerebellar ataxia type 3 (SCA3), also known as Machado-Joseph disease. The drug is designed to target and reduce the expression of ataxin-3 (ATXN3) pre-messenger RNA, thereby lowering levels of the mutant ATXN3 protein implicated in SCA3 pathogenesis. By inhibiting ATXN3 production through RNA interference mechanisms, BIIB132 aims to slow or halt disease progression. Clinical development reached Phase 1 trials, focusing on safety, tolerability, and pharmacokinetics via intrathecal administration in adults with SCA3[1][2][3][8].
- Other names
- ION260ION-260ION 260
02
Targets
ATXN3 (Ataxin-3)
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