Compositions and Methods for Treating Neurodegenerative Disorders

Categories:"Neurological Disorders

Reference #: 2016-050

OTC Contact: Ruchika Nijhara, Ph.D., MBA, CLP (Directory Information | Send a Message)

Description

Discoidin domain receptor (DDR) over-expression has recently been understood to play a role in Parkinson’s and Alzheimer’s diseases. Excess activation of DDRs may switch off autophagy, resulting in build-up of toxic proteins inside brain cells and possibly breakdown of the blood-brain barrier. This invention relates to development and use of LCB-03110 - a DDR inhibitor- to specifically knockdown DDR1 and 2 receptors. DDR knockdown in vitro and in vivo reduces the levels of alpha-Synuclein, beta-amyloid and Tau and prevents cell death caused by accumulation of misfolded proteins.

Applications

  • Development of drug candidate in the treatment of neurodegenerative and neuroinflammatory diseases, including Parkinson, lewy body dementia, Alzheimer, progressive supranuclear palsy, prions disease, multiple sclerosis and neuro-inflammatory diseases, traumatic brain injury and chronic traumatic encephalopathies (CTE) as well other diseases of synucleinopathies, tauopathies, amyloidosis and nerve system inflammation.

Advantages

  • The novel shRNA molecules are likely small enough cross the blood brain barrier and directly modulate the immune response to treat chronic neurodegenerative and neuroinflammatory diseases.

Stage of Development

Morris water maze tests showed in  mice treated with either LCB-03110 or DMSO (three times a day for three weeks), those treated with LCB-03110 were able to find the submerged platform 30% quicker. Determination of PK/PD of these drugs is underway to establish their patho-clinical impact.

INVENTORS

Charbel Moussa

Patent Status

Published PCT - WO 2018/081777 A1