LSP-GR1

LSP-GR1 is a splice modulating oligonucleotide (SMO) developed by LifeSplice Pharma to selectively reduce the expression of the "GluA1-flip" isoform of the AMPA-type glutamate receptor subunit 1 (GRIA1) in central nervous system cells. By targeting and decreasing GluA1-flip, which is associated with increased excitotoxicity and hyperexcitability in diseases such as amyotrophic lateral sclerosis (ALS) and epilepsy, LSP-GR1 aims to lower levels of certain AMPA receptors implicated in these pathologies. Preclinical studies have shown that LSP-GR1 delays motor deficits, slows disease progression, increases lifespan in ALS mouse models, and demonstrates anti-seizure properties with antiepileptogenic effects in epilepsy models. The drug has been awarded orphan drug status for ALS and is currently at the preclinical development stage[2][3][4][5].