Discovering Impactful Treatments with
Translatable Neuroscience
At NeuCyte we are working to make a difference in the lives of people suffering with neurological disorders. Our unique technology allows us to develop human neural in vitro disease models using patient derived cells to identify altered biological features. By focusing on specific phenotypes identified in disease relevant cells, our team can better understand the underlying disrupted biology leading to these neurological disorders. This knowledge gives our team unique and novel perspectives on how to treat these patients. We are actively pursuing drug discovery programs for several neurological diseases.
Alzheimer's Disease
Alzheimer’s Disease (AD) is the most prevalent cause of age-related dementia, impairing memory and causing cognitive and psychiatric deficits. AD remains a clinical, social, and economic liability for our society with no approved therapies to halt the disease process.
AD is characterized by β-amyloid accumulation, neurofibrillary tangles, neuroinflammation and frank neuronal and synaptic loss. Novel target development grounded in sound, fundamental scientific discovery will increase our understanding of AD etiology and the discovery of new therapeutic targets. Therefore, elucidating the molecular mechanisms of AD is of great importance.
Genetics play a critical role in the neuroinflammatory mechanisms of AD. Pathways perturbed by inherited genetics may serve as novel therapeutic targets for AD and AD-related Dementia. NeuCyte aims to develop novel therapies guided by altered pathways identified using human iPSC models.
Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis (ALS), also known as Lou Gehrig’s disease, is a motor neuron disease (MND) affecting over 200,000 people worldwide. The majority of cases occur without family history. ALS is an adult onset neurodegenerative disease characterized by damage and eventually death of upper (brain) and lower (brainstem and spinal cord) motor neurons. Progressive muscle weakness precedes generalized paralysis followed by death from respiratory failure, typically within three to five years of disease onset.
First described over 150 years ago, and despite extensive scientific efforts, precise pathological mechanisms remain inconclusive and complex. As such, an effective cure remains elusive. NeuCyte has adapted its pre-clinical “proprietary platform/technology” to the study of the pathophysiology of MNDs in an effort to answer this pressing need of therapeutic drugs.
Children with Rare Neurological Disorders
KIF1A Associated Neurological Disorder (KAND) is a rare and progressive neurodegenerative disorder caused by mutations in the KIF1A gene. Despite the exponentially growing patient population and well characterized disease etiology, there is currently no treatment or cure for KAND.
NeuCyte has partnered with KIF1A.ORG to establish the first set of iterative and cell-based screening platforms using induced pluripotent stem cell (iPSC)-derived neurons, generated from materials originated from the KIF1A Research Network for KAND therapeutic development and broader neurodegenerative disorders. Utilizing patient-derived iPSC lines ensures that the model system is representative of the clinical manifestations of KAND, which can be used to identify therapeutic candidates for clinical trials.
Epilepsy
Epilepsy is the fourth most common neurological disorder affecting over 50 million people of all ages worldwide. It is a chronic disorder characterized by epileptic seizures initiated by excessive and abnormal neuronal activity. Seizures are a symptom of the underlying disrupted biology.
The cause of most cases of epilepsy is unknown but several factors can increase the risk, including head injury (i.e. TBI), chronic alcohol and drug abuse, stroke or brain hemorrhage, brain infection including meningitis, encephalitis, brain malformations and genetic factors, brain tumors, and degenerative conditions affecting the brain such as Alzheimer’s.
Treatment is complicated by the multifactorial or complex nature of the disorders. Several epilepsy syndromes are thought to be driven by a combination of genetic and environmental factors. Furthermore, about 40% of newly diagnosed epilepsy patients are resistant to current therapies. This highlights the urgent need to develop novel drugs to help those patients suffering from this disorder.
Fragile X Syndrome (FXS) is the most common form of a heritable cognitive impairment and the most frequent monogenic cause of Autism Spectrum Disorders (ASD). The prevalence of the full mutation (most severe case) is about one in 4000 in males and one in 8000 in females. FXS patients can exhibit intellectual disabilities, behavioral and learning deficiencies, and social anxieties.
The disorder is caused by aberrations of the FMR1 gene that leads to epigenetic silencing and lack of expression of the encoded gene product, FMRP. FMRP plays an essential role for regulating localization, stability, and translation of mRNAs particularly in neural cells. The current understanding of the genetics and the neurobiology of FXS has led to many targeted treatments, but none have come close to curing the disorder.