This technology is an engineered protein construct capable of specifically targeting neurons and delivering a specific inhibitor for caspase-8 and caspase-1 to prevent apoptosis.
Current pharmaceutical strategies to prevent apoptosis via caspase inhibition generally exhibit undesirable side effects and toxicity. The toxicity and side-effects are caused by off-target cell apoptosis of normal, healthy, and functioning cells. Most caspase-targeting drugs available do not exhibit cell-type specificity which limits their use as neurodegenerative therapeutic agents. Therefore, there is a need for a neuron-targeting therapeutic to prevent the cell apoptosis that results in neurodegenerative diseases.
This technology is an engineered protein construct that prevents the uncontrolled apoptosis associated with neurodegenerative diseases. The protein consists of a neuron-targeted import peptide, allowing the protein to selectively enter neurons. Furthermore, it contains a specific inhibitor for caspase-8 and caspase-1 to block the activity of enzymes implicated in apoptosis – a strategy which may prevent the uncontrolled apoptosis seen in disease states. This protein, thus, represents a potential therapeutic approach to combat neurodegenerative diseases.
The efficacy of the drug was demonstrated in cultured neurons from a mouse model of spinal muscular atrophy and was observed to inhibit apoptosis.
Patent Pending
IR CU13324
Licensing Contact: Jerry Kokoshka