Couragene is pioneering advancements in the delivery of gene and biologic therapies through its proprietary STEP (Stimuli-responsive Traceless Engineering Platform) technology. This innovative platform utilizes chemical engineering techniques to facilitate the efficient intracellular delivery of various genetic medicine payloads, including proteins, gene editors, antibodies, mRNA, antisense oligonucleotides (ASOs), and DNA. A significant challenge in the field has been the delivery of these therapies to the brain, where existing methods, such as viral vectors and lipid nanoparticles, face safety concerns and limitations in payload size. Couragene's STEP technology addresses these issues by creating smaller, chemically engineered payloads that can effectively penetrate cells and release their biological functions once inside.

One of the notable applications of Couragene's STEP technology is the development of the STEP-CRISPR gene editor, which has an average diameter of 12.4 nm, making it particularly suitable for brain penetration. This technology has been validated in preclinical studies, demonstrating its ability to deliver CRISPR-Cas9 gene editors to embryonic fibroblast cells and achieve efficient brain-wide editing. Specifically, STEP-CRISPR has been shown to activate the expression of a yellow fluorescent chimeric protein (YFCP) in a transgenic mouse model, highlighting its potential for advancing genetic therapies and addressing the challenges associated with delivering treatments to the central nervous system.

United States

Couragene believes that delivery of gene and biologic therapies should be safer and more efficient.  Delivery of genome modification therapies to brain is further complicated by the physiological barriers.  Couragene's STEP (Stimuli-responsive Traceless Engineering Platform) technology has demonstrated great potential to overcome the delivery hurdles for treatment of various diseases, including neurogenetic disorders.

Couragene

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