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KMM001
- Development stage
- Preclinical
- Lead developer
- Kobe Gakuin University
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
KMM001 is a chimeric antisense oligonucleotide (ASO) designed to selectively inhibit the expression of myostatin (MSTN), a negative regulator of skeletal muscle growth. The molecule is composed of 2'-O-methyl RNA (2'-OMeRNA) and 2'-O,4'-C-ethylene-bridged nucleic acid (ENA) chemistry, which enhances its stability and binding affinity. KMM001 functions by targeting exonic splicing enhancer sequences within exon 1 of the MSTN gene, thereby blocking pre-mRNA maturation and reducing the production of mature myostatin protein. Preclinical studies have demonstrated that KMM001 is highly specific for myostatin and does not cross-react with the closely related GDF11. It is being investigated for the treatment of muscle-wasting conditions, including sarcopenia associated with chronic kidney disease (CKD) and spinal muscular atrophy (SMA).
- Other names
- myostatin-specific antisense oligonucleotideMSTN-specific ASOmyostatin-targeting ASO
02
Targets
GDF8 (Myostatin)
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