Drug intelligence / Profile preview

biib080

Development stage
Phase 2
Lead developer
Biogen
Modality
Small Molecules, 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

BIIB080 is an investigational antisense oligonucleotide (ASO) therapy designed to target microtubule-associated protein tau (MAPT) messenger RNA (mRNA), thereby reducing the production of tau protein in the brain. Tau accumulation and aggregation are implicated in the pathogenesis of Alzheimer’s disease and related dementias. By silencing MAPT mRNA expression, BIIB080 aims to decrease both total and phosphorylated tau levels in cerebrospinal fluid and brain tissue. The drug is being developed primarily for early-stage Alzheimer’s disease—including mild cognitive impairment due to AD and mild AD dementia—and has shown robust dose-dependent reductions in tau biomarkers as well as favorable trends on exploratory clinical endpoints such as cognition and daily functioning. It is administered via intrathecal injection directly into cerebrospinal fluid[1][2][3][4][5][6].

Other names
MAPTRX ASO
02

Targets

MAPT (Microtubule-associated protein tau)

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