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LSP-GR3
- Development stage
- Preclinical
- Lead developer
- LifeSplice Pharma
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal
01
Overview
LSP-GR3 is an experimental antisense oligonucleotide (ASO) developed by LifeSplice Pharma. It is a chemically-modified RNA oligonucleotide, specifically a splice modulating oligomer (SMO) with a 2-O-Methyl Phosphorothioate backbone. The drug targets the glutamate receptor 3 (GluR3) gene (*GRIA3*), modulating its alternative splicing to favor the expression of the flip isoform over the flop isoform. This modulation is intended to enhance glutamate receptor function in the central nervous system. LSP-GR3 has received Orphan Drug designation from the FDA for the treatment of epilepsy.
- Other names
- LSP-GR3LSP-GR-3LSP-GR 3
02
Targets
GRIA3 (AMPA receptor subunit GluA3)
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