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GYS1 ASO#3
- Development stage
- Preclinical
- Lead developer
- Ionis Pharmaceuticals
- 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
- Parenteral, Subcutaneous
01
Overview
GYS1 ASO#3 is an antisense oligonucleotide (ASO) designed to target and downregulate glycogen synthase 1 (GYS1) mRNA. Developed by Ionis Pharmaceuticals in collaboration with researchers at the University of California, Irvine, GYS1 ASO#3 acts as a substrate reduction therapy by inhibiting the synthesis of GYS1, the enzyme responsible for glycogen production in muscle cells. In preclinical models of Pompe disease (Gaa-/- mice), GYS1 ASO#3 has demonstrated robust knockdown of Gys1 mRNA and GYS1 protein, leading to a significant reduction in glycogen accumulation in skeletal muscle. When combined with recombinant human acid alpha-glucosidase (GAA) enzyme replacement therapy (ERT), GYS1 ASO#3 further alleviates lysosomal and autophagic pathologies, resulting in improved muscle function.
- Other names
- Gys1 ASO 3Gys-1 ASO 3Gys 1 ASO 3Gys1 ASO-3Gys-1 ASO-3Gys 1 ASO-3Gys1 ASO#3
02
Targets
GYS1 (Glycogen synthase 1)
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