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AT466
- Development stage
- Preclinical
- Lead developer
- Astellas Gene Therapies
- Modality
- AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, 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
- Parenteral
01
Overview
AT466 is an investigational gene therapy being developed for the treatment of myotonic dystrophy type 1 (DM1). It utilizes an adeno-associated virus (AAV) vector to deliver antisense oligonucleotides or RNA interference constructs targeting the DMPK gene, which is mutated in DM1. The therapy aims to reduce toxic RNA accumulation and restore normal protein function by either exon skipping or RNA knockdown mechanisms. Originally developed by Audentes Therapeutics and now under Astellas Gene Therapies, AT466 remains in preclinical/early research stages[2][3][4][7].
- Other names
- rAAV-MBNL1rAAV-MBNL-1rAAV-MBNL 1AAV8-MBNL1AAV-8-MBNL1AAV 8-MBNL1AAV9-MBNL1AAV-9-MBNL1AAV 9-MBNL1
02
Targets
CUG repeat RNA (Dystrophia myotonica protein kinase messenger RNA with expanded CUG repeats)
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