Naphthalene-containing Selective Inhibitors of BMP type 1 Receptors for the Treatment of Fibrodysplasia Ossificans Progressiva

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.

Creation and Use of Kinetin Derivatives for Treating RNA Missplicing Diseases Such as Familial Dysautonomia

This technology includes the creation and use of compounds, including kinetin derivatives, that improve mRNA splicing in a cell for the treatment of disorders associated with misspliced mRNA, including familial dysautonomia (FD). FD, the best-known and most common member of a group of congenital sensory and autonomic neuropathies, affects neuronal development and is associated with progressive neuronal degeneration. This disease is caused by mutations in the splicing of intron 20 of the IKMKAP gene that results in a unique pattern of tissue-specific exon skipping.

Process for Practical, Scalable, Commercially-viable Method for the Synthesis of Enantio-enriched Aminoalcohols, Including the Novel Antifungal VT-1129 Used to Treat Cryptococcal Meningitis

This technology relates to the discovery and development of a practical, scalable, and commercially viable method for the synthesis of the novel antifungal VT-1129. Cryptococcal meningitis (CM) is a fungal infection that is particularly prevalent in immune-compromised patients and can be treated by VT-1129. CM has a current estimated patient population of 1-1.25 million, predominately in sub-Saharan Africa and the developing world.

A Group of Compounds that Activate AMP-activated protein kinase (AMPK) that may Treat Niemann-Pick Disease Type C (NPC)

This technology relates to the identification and use of a group of compounds that activate the AMP-activated protein kinase (AMPK) and also effectively reduce lysosomal cholesterol accumulation in patients with Niemann-Pick disease Type C (NPC). Clinical trials are currently underway to determine the efficacy of beta-cyclodextrin in treating patients with NPC. A potential mechanism has been proposed indicating that beta-cyclodextrin activated AMP-activated protein kinase, leading to restoration of autophagy in cells from NPC patients.

Repurposing CDK Inhibitors for the Treatment of Zika Virus Infection

This invention includes the discovery and use of a group of CDK inhibitors that were found during a drug repurposing screen designed to find compounds that inhibit Zika virus caused cell death. The identified CDK inhibitors have all previously been used in clinical trials for other diseases, potentially reducing the long time course needed for new drug discovery and development.

Identification and Use of a Novel Functionally Selective GHSR1a Ghrelin Receptor Inhibitor, including NCGC00538279, for the Treatment of Food and Chemical Addiction

This technology includes a chemical series, including the NCGC00538279 compound, that selectively activates the GHSR1a G-protein pathway for calcium mobilization while only partially activating the beta-arrestin-2 translocation pathway. The resulting chemical series may be therapeutically valuable for addictive disorders. Activation of the GHSR1a G-protein pathway promotes production and secretion of multiple hormones, including insulin, growth hormone, and IGF1. Activation of the beta-arrestin-2 pathway stimulates dopamine production and may mediate addictive behaviors.

Establishment of Induced Pluripotent Stem Cells (iPSC) from the Thirteen-lined Ground Squirrel

The limited choice in cell types available for in vitro studies has become an obstacle in hibernation research. 

Researchers at the National Eye Institute for the first time have successfully established iPSC line(s) from a mammalian hibernator, which can be potentially used to generate various cell types and tissue models for in-depth mechanistic studies of hibernation and coldness tolerance in vitro. 

Novel Dopamine D2 Receptor Antagonists and Methods of Their Use

Investigators at the NIH have identified a series of novel, small molecule antagonists of the dopamine D2 receptor. Among the dopamine receptor (DAR) subtypes, D2 DAR is arguably one of the most validated drug targets in neurology and psychiatry. For instance, all receptor-based anti-Parkinsonian drugs work via stimulating the D2 DAR, whereas all FDA approved antipsychotic agents are antagonists of this receptor. Unfortunately, most agents that act as antagonists of D2 DAR are problematic, either they are less efficacious than desired or cause multiple adverse effects.

A Neural Stem Line from a Niemann Pick C (NPC) Type 1 Patient for Therapy Development

This technology includes a neural stem cell (NSC) line derived from a Niemann Pick C (NPC) patient, aimed at advancing research and drug development for NPC, an inherited neurodegenerative disorder characterized by the accumulation of cholesterol in neurons. The NSCs, which serve as a crucial intermediate cell type, can be differentiated into any neuronal or glial cell of the brain or spinal cord under appropriate culture conditions. These cells originate from fibroblasts reprogrammed into induced pluripotent stem cells.

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