Columbia Technology Ventures

Reprogramming metabolism for treating neurodegeneration

This technology describes a metabolism regulator gene, which can be manipulated to improve neuronal survival in retinal degeneration and other neurodegenerative diseases.

Unmet Need: Improving neuronal survival in retinal degeneration

Retinitis pigmentosa is a progressive genetic neurodegenerative disorder that causes impaired vision and blindness. Current treatment options are extremely limited and specific gene therapies would only be effective in a fraction of patients due to the large number of genes associated with retinitis pigmentosa. There is an urgent need to develop therapies that could halt the deterioration of neurons in this disease and related neurodegenerative conditions.

The Technology: Genetic targeting of metabolic regulation for treating retinal degeneration

This technology describes a gene target that can be inhibited to improve the rate of cellular metabolism for preventing neuronal death. Recent research has implicated metabolic dysregulation as a critical factor leading to neurodegeneration. By selectively manipulating the gene von Hippel-Lindau (VHL), glycolysis can be promoted, which subsequently improves neuronal survival in retinitis pigmentosa. The ability to correct metabolic function through selectively targeting this gene could potentially treat a wide range of neurogenerative conditions that involve metabolic dysfunction such as age-related macular degeneration (AMD), Alzheimer’s disease, and Parkinson’s disease.

This technology has been successfully used to preserve the visual function in a mouse model of retinitis pigmentosa.

Applications:

  • Treatment for eye diseases such as retinitis pigmentosa, AMD, and glaucoma
  • Treatment for neurodegenerative conditions including Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, amyotrophic lateral sclerosis (ALS), and Lewy body dementia
  • Treatment for metabolic disorders

Advantages:

  • Allows for disease-modifying therapy by targeting an underlying disease mechanism
  • Can treat several neurodegenerative diseases
  • Downregulates catabolism while upregulating anabolism
  • Describes multiple means of inhibiting VHL including small molecules and gene therapy

Lead Inventor:

Stephen H. Tsang, M.D., Ph.D.

Patent Information:

Patent Status

Tech Ventures Reference:

  • IR CU17321

  • Licensing Contact: Ron Katz