Our Disease Focus
Niemann-Pick Disease Type C (NPC) belongs to a group of diseases known as lysosomal storage disorders (LSDs). LSDs are characterized by dysfunctional lysosome homeostasis, in other words, a loss of function or expression of lysosomal enzymes and proteins.
While the pathological manifestations are observed throughout the body, neuronal dysfunction and degeneration pertaining to the loss of purkinje cells in the cerebellum involve the most deleterious and ultimately fatal features in patients.
One of the current leading therapeutic candidates for treating NPC is Hydroxypropyl beta cyclodextrin (HPbCD). The resulting effects of HPbCD treatment are cholesterol depletion and partial delay of disease progression, however, unmet needs, such as an inability to permeate the blood-brain barrier (BBB) and reduction of negative side-effects remain an issue.
Using BGN-112, which can pass through the blood-brain barrier (BBB), BioGraphene employs physically-induced cholesterol depletion therapy with the hope of extending the expected lifespan of patients.
Learn more about NPC and how you can help us work towards a cure with this amazing community at the National Niemann Pick Disease Foundation (NNPDF), the Ara Parseghian Medical Research Fund (APMRF), and the Niemann-Pick Type C Therapy Accelerator.
The pathogenesis of Parkinson’s disease (PD) is multifactorial. However, we understand a-synucleinopathy or the aggregation of the neuroprotein alpha-synuclein from its normally smaller monomeric size to a large fibril form as a major pathological hallmark of Parkinson’s disease. This process is directly correlated to the loss of dopaminergic neurons.
Through years of study, we substantiated that BGN-112 can inhibit the fibrillation process, and induce disaggregation of preexisting fibrils in a time-dependent manner. Induction of disaggregation is especially promising in the sense of developing BGN-112 as a translational nanomedicine because these existing fibrils can function as the nucleation seeds to recruit neighboring alpha-synuclein monomers to be misfolded, and thereby provoke further fibrillation in the brain.
To learn more about PD, current research efforts, or seek support, visit the Michael J. Fox Foundation (MJFF), Parkinson’s Foundation (PF), and the American Parkinson Disease Association (APDA).
Alzheimer's disease is a progressive neurodegenerative disease, which is characterized by the abnormal accumulating of toxic amyloid-β oligomers and plaques in the brain. Throughout the progression of Alzheimer’s disease, patients experience problems with memory impairment, speech disorder, cognitive impairment, and behavior issues. Although the cause of Alzheimer’s is still unknown, the misfolded amyloid-β fibrils is known to induce neuronal cell death in the brain, resulting in the worsening of these symptoms.
BGN-112 prevents the abnormal fibrillation of amyloid-β protein as well as directly dissolves the pre-existing amyloid-β fibrils into non-toxic monomers.
To learn more about current research trends and how you can be involved in the fight against Alzheimer’s visit the Alzheimer’s Foundation of American (AFA), the Alzheimer’s Research Foundation (ARF), or the Alzheimer’s Association (AA).
Amyotrophic lateral sclerosis (ALS), also called Lou Gehrig’s Disease, is a progressive, degenerative neurological disease that primarily affects the neurons in the brain and spinal cord responsible for controlling voluntary muscle movement. As the disease progresses, ALS patients gradually lose the abilities of muscle movement, speech, swallowing, and finally breathing. Although the cause of ALS is still unknown, abnormal accumulation of TDP-43 protein in cytoplasm of neuron is observed in more than 90% ALS patients, which is thought as a central factor of ALS.
BGN-112 prevents fibrillation of TDP-43 proteins as well as directly dissolves the protein aggregates into non-toxic monomers.
To learn more about ALS visit the ALS Association.
Renal fibrosis is a histological hallmark of chronic kidney disease (CKD), pathologically characterized by progressive kidney tissue scarring including glomerulosclerosis, tubulointerstitial fibrosis, and loss of renal parenchyma. It represents an excessive deposition of extracellular matrix components in the kidney, resulting in structural damage, impairment of renal function, and eventually the end-stage of renal disease. Oxidative stress is known to play a crucial role in provoking the expressions of proinflammatory cytokines and recruitment of other immunocytes in acute kidney injury (AKI)-to-CKD transition.
BGN-112 reduces oxidative stress due to their ability to scavenge reactive oxygen species (ROS), which in turn recovers mitochondrial dysfunction, and protects renal cells.