Technology Platforms

The small molecule regenerative therapy (SMRT) technology uncovers the therapeutic potential of dual-target approach leveraging the direct activation of the nuclear factor - κB p65 subunit (NF-κB) and the increase of manganese superoxide dismutase (MnSOD) expression.

Targeted RNAi Platform
Self-delivering RNAi (sd-rxRNA® ) technology has the ability to inhibit gene expression providing a potentially powerful tool to treat neurodegenerative disorders where the pathogenic process involves the overexpression of one (or more) protein.

The sd-rxRNA® platform is positioned to allow discovery and development of innovative therapeutics with potential advantages over traditional drugs.

Mechanism of Action

Our SMRT compounds can selectively activate NF-κB p65 exclusively in neurons or glial cells without triggering any inflammatory effects or other evident toxicity (i.e. nitric oxide).

Activation of NF-κB p65 promotes expression of MnSOD, neurotrophins, and intracellular chaperones.

Our sd-rxRNA® targeting SOD1 library includes hybrid oligonucleotides that combine the beneficial properties of both conventional RNAi and antisense technologies.

sd-rxRNA® SOD1-targeting oligonucleotides have high specificity, high potency, and the ability to interfere with the expression of SOD1 gene.

Regenerative Effects

The regenerative and potentially disease-modifying impact of our compounds are mediated by:
• the neurotrophic and transcriptional effects induced by the selective/direct activation of NF-κB p65
• improved mitochondrial-induced cell viability and protein functionality promoted by the increase of MnSOD2