SUMMARY First-in-class small molecules to change how the PRNP gene is spliced, reducing harmful prion levels and offering a new treatment approach for prion diseases The Unmet Need: Safe, effective, and deliverable therapies to reduce prion protein levels in the central… Read More
SUMMARY Phosphorodiamidate morpholino oligomer (PMO)-based RNA-degrading chimeras reduce α-synuclein protein by degrading its messenger RNA preventing protein aggregation and propagation associated with neurodegenerative diseases. The Unmet Need: More potent and selective therapeutic strategies for neurodegenerative α-synucleinopathies Neurodegenerative diseases such as… Read More
SUMMARY This technology regulates the expression of the CACNA1A gene, which encodes a neuronal calcium channel subunit, by redirecting alternative splicing to treat related neurological diseases. This approach can potentially correct gene expression variations linked to disorders including episodic ataxia, migraines, epilepsy, autism, and… Read More
SUMMARY The Unmet Need: Precise evaluation and effective treatment planning for proprioception Proprioception is a critical aspect of human motor control and balance, traditionally evaluated during neurological and physical examinations. In these fields, there is a growing recognition that qualitative assessments alone… Read More
SUMMARY The field of metabolic disease treatment has long been challenged by the need for precise, long-term regulation of critical hormones and proteins that manage glucose homeostasis and energy balance. Current therapeutic strategies for conditions such as obesity and diabetes struggle to provide a sustained and adjustable intervention, leaving patients… Read More
SUMMARY Antisense oligonucleotide (ASO) therapeutic with a precise, targeted approach to the genetic underpinnings of C9ORF72-linked ALS and FTD The Unmet Need: Front-line disease modifying therapies for ALS and FTD The prevalence of ALS is increasing at a steady rate in line… Read More
SUMMARY This method enables high fidelity decoding of neural activity in an interpretable manner with a small training data set. The Unmet Need: Measuring neural activity in the brain with a strong signal to… Read More
SUMMARY A novel drug delivery system using exosomes derived from hippocampus neuron cells engineered to express the Fe65 protein targeting amyloid-β precursor protein to reduce amyloid plaques Alzheimer's disease. This system crosses the blood-brain barrier, enhances uptake by neuronal cells, and induces autophagy, improving cognition and reduce disease pathogenesis. Read More
SUMMARY Drug repurposing to use catechol-O-methyl-transferase inhibitors for treating impulse control disorders, obsessive-compulsive disorders, and substance addictions The Unmet Need: New options for treating impulsive behavior disorders Impulse control disorders, Obsessive-Compulsive Disorder (OCD), and substance addictions are conditions that significantly impair the… Read More
SUMMARY Synaptic transmission and plasticity are fundamental to neuronal functions, and alterations of synaptic protein expression are direct causes of autism, intellectual disability (ID), and epilepsy. De novo loss-of-function (LoF) mutations in SYNGAP1, encoding the synaptic Ras GTPase-activating protein, are among the most prevalent causes of ID and autism spectrum… Read More
SUMMARY Post-CPR cardiogenic shock is associated with hemodynamic instability and poor neurological outcomes. The pathophysiology is unknown and current treatment approaches merely restore basal cardiac function and do not protect against longer term damage. The inventors demonstrated that the main mechanism leading to post-CPR cardiogenic shock is myocardial stunning, which… Read More
SUMMARY Multiple Sclerosis (MS) is caused by degradation of the protective myelin sheath that surrounds nerve fibers in the brain and spinal cord. Current MS therapies only stop the progression of this demyelination and do not promote the repair of the myelin sheath, which significantly limits their efficacy. The invention… Read More