Columbia Technology Ventures

Lecithin cholesterol acyltransferase (LCAT) lipid activators for prevention and treatment of neurodegeneration

This technology is a therapeutic targeting neurodegenerative disorders involving small molecules to enhance lipid and cholesterol metabolism in the brain via activation of lecithin cholesterol acyltransferase (LCAT).

Unmet Need: A therapeutic for neurodegenerative diseases by targeting lipid metabolism

Current methods to treat neurodegenerative diseases such as Alzheimer’s disease (AD) mainly involve the treatment of symptoms rather than addressing the underlying pathogenesis. The primary focus for developing AD therapeutics has currently been based on targeting biogenesis or clearance of amyloid plaques, but many of these clinical trials have resulted in failed efficacy tests. As a result, there is a need to develop therapeutics to treat and disrupt Alzheimer’s disease progression.

The Technology: A therapeutic intervention addressing the underlying pathogenesis of neurodegeneration

This technology describes a therapeutic to regulate lipid dyshomeostasis to treat Alzheimer’s disease and related disorders. This technology enhances lipid and cholesterol metabolism in the brain via activation of lecithin cholesterol acyltransferase (LCAT). Because dysregulation of lipid and cholesterol metabolism has been implicated in several neurodegenerative diseases, modification of lipid metabolism to enhance the function of lipid processing enzymes may be a potential therapeutic strategy for several neurodegenerative diseases.

This technology has been validated in vivo with an Alzheimer’s disease mouse model.

Applications:

  • Therapeutic strategy for Alzheimer’s Disease, Niemann-Pick type C (NPC), familial LCAT deficiency (FLD) and fish eye disease (FED), and cholesteryl ester storage disease (CESD)
  • Therapeutic strategy for other neurodegenerative diseases
  • Research tool for studying lecithin cholesterol acyltransferase (LCAT) activity

Advantages:

  • Treatment for neurodegenerative diseases that addresses disease progression
  • Preventative therapeutic targeting neurodegeneration
  • Broad application to diseases with lipid metabolism dysregulation as a pathologic feature

Lead Inventor:

Laura Beth McIntire, Ph.D.

Patent Information:

Patent Pending(WO/2023/122185)

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