Drug intelligence / Profile preview

Klotho (Minicircle)

Development stage
Preclinical
Lead developer
Minicircle
Modality
Plasmid DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Gene Therapies
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)KL (Klotho Protein)GRIN2B (N-methyl-D-aspartate Receptor Subunit Combination: NR1a/NR2B)

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