This invention includes the generation and use of two polyclonal antibodies that specifically recognizes the phosphorylation site pThr707 of Neuroligin 4 and pThr739 of Neuroligin 1. A peptide of the site around the phosphorylation site was generated and injected into rabbits to create an immune response. Serum was collected from the rabbits that was then affinity purified. The specificity of the resulting polyclonal antibodies was then determined using biochemical techniques.

This technology includes a robust and highly efficient protocol that differentiates human pluripotent stem cells (hPSCs) exclusively into nociceptors (also called sensory neurons) under chemically defined conditions. The use of hPSCs, including hESCs and iPSCs, holds great promise for drug screening, disease modeling, toxicology, and regenerative medicine. However, efficient and highly reproducible protocols have not been developed for most cell types that are relevant and urgently needed for translational applications.

This technology includes a robust and highly efficient protocol that differentiates induced pluripotent stem cells (iPSCs) exclusively into nociceptors (also called sensory neurons) under chemically defined conditions. The use of hPSCs, including hESCs and iPSCs, holds great promise for disease modeling, drug discovery, and cell therapy. However, efficient and highly reproducible protocols have not been developed for most cell types that are relevant and urgently needed for translational applications.

This technology includes a method for creating functional, brain region-specific neural spheroids that can be used for disease modeling and therapeutic testing of compounds for neurological diseases. The developed protocol uses somatic cells, including iPSC-derived neurons, as well as astrocytes using means such as 96- or 384-well ultra-low attachment round-bottom plates. Spheroids have been generated using this method that model brain regions such as the ventral tegmental area and prefrontal cortex, which are implicated in Parkinson’s and Alzheimer’s disease.

This technology includes the identification and use of small molecules to rescue cells undergoing ferroptosis, a type of programmed cell death. These small molecules can be used as treatments in situations where epithelial cells are being damaged, including respiratory disorders, brain injury (including traumatic brain injury), renal injury, radiation-induced injury, and neurodegenerative disorders. Ferroptosis is a type of programmed cell death that is triggered by an increased presence of oxidants.

This technology includes the use of a new class of molecules (nanomolar ALK2 inhibitor) to impede bone morphogenetic proteins (BMP) signaling for the treatment of Fibrodysplasia ossificans progressiva (FOP). FOP is a rare disease, characterized by malformation of the great (big) toes during embryonic development. Individuals with FOP have identical heterozygous activating mutation (R206H) in the gene encoding ACRV1 (also known as ALK2), a BMP type 1 receptor.

This technology includes the compositions and methods for inhibiting PIN1 for the treatment of disorders characterized by elevated PIN1 levels (e.g., immune disorders, proliferative disorders, and neurodegenerative disorders) with small molecules. Pin1 dysregulation has been associated with a number of pathological conditions. In particular, PIN1 has been shown to promote oncogenesis by modulating several oncogenic signaling pathways and its overexpression has been shown to correlate with poor clinical outcome.

This technology includes a stable pharmaceutical formulation of propofol hemisuccinate for inhalation delivery to treat intractable epilepsy and migraine. The formulation can be used to treat a patient experiencing a seizure aura to prevent a motor seizure. Alternatively, the formulation can be used to treat an epileptic patient who is experiencing seizure clusters in an out-of-hospital or in-hospital setting. For migraines, the formulation can be used to treat a patient experiencing a migraine aura or early migraine to forestall the development of the full symptoms of a migraine headache.

This technology includes two new salt forms for (2R,6R)-hydroxynorketamine (2R,6R-HNK), which is the lead molecule being developed for treatment-resistant depression. Currently, 2R,6R-HNK is being developed as the HCl salt. The HCl salt is slightly hygroscopic at high RH. This is a potential liability, especially in an oral pill form. Recently the malonate and salicylate salt have been discovered and found to have excellent crystalline behavior while also not having the hygroscopic liability the HCl salt holds. This represents a clear advantage.

This technology includes the compounds, compositions, and methods for treating CNS disorders and cancer with an inhibitor of a p21-activated kinase (PAK). PAK activation is shown to play a key role in spine morphogenesis, and attenuation of PAK can reduce, prevent or reverse defects in spine morphogenesis leading to improvements in synaptic function, cognition, and/or behavior. This could be used to treat a wide variety of CNS disorders such as schizophrenia, Alzheimer’s, Parkinson’s Disease, depression, bipolar, and many others.