Antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs), as vital components of precision medicine, have demonstrated tremendous potential in the field of gene therapy.
In modern gene therapy, antisense oligonucleotides (ASOs) and small interfering RNAs (siRNAs) are key nucleic acid drugs that offer new treatment options for genetic and complex diseases.
Tofersen, developed by Biogen in collaboration with Ionis Pharmaceuticals, is an antisense oligonucleotide that targets the superoxide dismutase 1 (SOD1) mRNA.