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Klotho (Minicircle)
- Development stage
- Preclinical
- Lead developer
- Minicircle
- Modality
- Gene Therapies, Plasmid DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
Minicircle's Klotho gene therapy (MINI-002) is an investigational non-viral, plasmid-based gene therapy designed to increase the systemic expression of the Klotho protein. Klotho is a longevity-associated protein that typically declines with age and is involved in regulating various physiological processes. The therapy utilizes Minicircle's proprietary DNA plasmid platform to deliver the Klotho gene via a single subcutaneous injection, which is intended to enable sustained protein expression for up to one year. The mechanism of action involves the upregulation of Klotho, which subsequently activates the FOXO3A pathway (a key regulator of cellular stress response and longevity) and modulates GRIN2B-related pathways to enhance synaptic plasticity and cognitive function. The therapy is being developed to address aging-related conditions, specifically targeting cognitive decline, cardiovascular health, and kidney function.
- Other names
- Klotho gene therapyMinicircle Klotho
02
Targets
FOXO3 (Forkhead box protein O3)GRIN2B (N-methyl-D-aspartate Receptor Subunit Combination: NR1a/NR2B)KL (Klotho Protein)
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