Columbia Technology Ventures

Engineered neuron-specific protein inhibits uncontrolled cell death to combat neurodegeneration

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.

Unmet Need: Neuron-specific protein 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.

The Technology: Engineered protein for targeted delivery of apoptosis inhibitors to neurons

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.

Applications:

  • Broad-spectrum anti-neurodegenerative biopharmaceutical
  • Treatment to slow progression of neurodegeneration
  • Prophylactic measure for patients with high familial risk of developing neurodegenerative disease

Advantages:

  • Cell-type specific targeting for drug delivery
  • Reduced toxicity
  • Cost-effective
  • Simplified delivery method of therapeutic agents

Lead Inventor:

Carol Troy, M.D., Ph.D.

Patent Information:

Patent Pending

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