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.

Repurposed Use of the Alkaloids Emetine and Cephaeline to Treat Zika Virus Infection

This technology includes the use of two related compounds, Emetine and Cephaeline, as a potent inhibitor of the Zika virus (ZIKV). Emetine and it's analog Cephaeline were identified in a high-throughput assay aimed at identifying anti-ZIKV compounds. Both Emetine and Cephaeline are potent inhibitors of ZIKV infection in cell culture, and Emeline is a potent inhibitor of ZIKV infection in a live mouse model.

Novel ACRV1/ALK2 Inhibitors and Methods for Inhibiting BMP Signaling for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP)

This technology includes the identification and use of novel ACRV1/ALK2 inhibitors for the treatment of fibrodysplasia ossificans progressiva (FOP), an autosomal-dominant rare disease that affects one person in every 1-2 million. FOP is characterized by malformation of the great (big) toes during embryonic development and by progressive heterotopic endochondral ossification (HEO) postnatally, which leads to the formation of a second skeleton of heterotopic bone.

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.

3D Vascularized Human Ocular Tissue for Cell Therapy and Drug Discovery

Degeneration of retinal tissues occurs in many ocular disorders resulting in the loss of vision. Dysfunction and/or loss of Retinal Pigment Epithelium Cells (RPE) and disruption of the associated blood retinal barrier (BRB) tissue structures are linked with many ocular diseases and conditions including: age-related macular degeneration (AMD), Best disease, and retinitis pigmentosa. Engineered tissue structures that are able to replicate the function of lost BRB structures may restore lost vision and provide insight into new treatments and mechanisms of the underlying conditions. 

Novel Methods for Generating Retinal Pigment Epithelium Cells from Induced Pluripotent Stem Cells

The retinal pigment epithelial cells (RPE) make up a polarized monolayer in the vertebrate eye that separates the neural retina from the choroid, and performs a crucial role in retinal physiology by forming a blood-retinal barrier and closely interacting with photoreceptors to maintain visual function.  Many ophthalmic diseases, such as age-related macular degeneration, are associated with a degeneration or deterioration of the RPE. 

Using FDA-approved Small Molecule Drug Reserpine and related compounds (especially Halofantrine) To Protect Photoreceptors In Inherited Retinal Degenerations And Age-Related Macular Degeneration

Summary: 
The National Eye Institute seeks research co-development partners and/or licensees for a therapy using an FDA-approved small molecule drug reserpine (and related compounds especially halofantrine) that prevents photoreceptor cell death in retinal degenerations.

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.
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