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MC-GYS1
- Development stage
- Preclinical
- Lead developer
- miRecule
- Modality
- RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Parenteral
01
Overview
MC-GYS1 is a preclinical-stage microRNA therapeutic being developed by miRecule for the treatment of Glycogen Storage Disease (GSD). The therapeutic is designed to target and downregulate glycogen synthase 1 (GYS1), an enzyme responsible for synthesizing glycogen in skeletal muscle and other tissues. By inhibiting GYS1 expression, MC-GYS1 aims to reduce the pathological accumulation of glycogen, which is a hallmark of several glycogen storage disorders, such as Pompe disease (GSD type II).
02
Targets
GYS1 (Glycogen synthase 1)
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