Splice Switching Antisense Oligonucleotides
We target pre-messenger RNA (pre-mRNA) using state-of-the-art antisense oligonucleotide technologies to modulate protein expression as innovative therapies for various rare and acquired diseases.
We pioneer in next generation RNA therapeutics including splice switching antisense oligonucleotide drugs to treat inherited rare and acquired diseases.
We target pre-messenger RNA (pre-mRNA) using state-of-the-art antisense oligonucleotide technologies to modulate protein expression as innovative therapies for various rare and acquired diseases.
Antisense oligonucleotides (ASOs) are short synthetic single-stranded oligonucleotides that can specifically bind to RNA targets to either induce splice-switching or block RNA translation.
Thiomorpholino oligonucleotide (TMO) is a newly developed robust class of therapeutic RNA chemistry. In comparison to other currently available nucleic acid chemistries, TMO was found to be far superior to induce splice switching. TMOs can be efficiently synthesized using solid-phase oligonucleotide synthesis methodologies, and has shown to have excellent exon skipping activity at extremely low doses.
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