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nL-SAA1-01

Development stage: Phase 1
Lead developer: Mayo Clinic
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

Overview

nL-SAA1-01 is an antisense oligonucleotide (ASO) drug that targets serum amyloid A1 (SAA1). It functions as a SAA1 modulator, aiming to reduce the formation of amyloid deposits that characterize AA amyloidosis. The drug is administered via subcutaneous injection and is currently being evaluated in Phase 1 clinical trials for the treatment of AA amyloidosis.

Targets

SAA1 (Serum amyloid A-2 protein)

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