Use of Antihistamine Compounds for the Treatment of Hepatitis C Virus

The vast majority of people infected with Hepatitis C Virus (HCV) will have chronic infection. Over decades, this can lead to liver disease and liver cancer. In fact, HCV infection is the leading cause of liver transplants in the U.S. Several new drugs have recently come into the market that will likely change the HCV treatment paradigm. However, the effectiveness of these new drugs can vary depending on the HCV genotype. Thus, there is still the need for additional new therapeutics against HCV.

A Highly Efficient Differentiation Protocol for Placental Cells Derived from Human Pluripotent Stem Cells

This technology includes a robust and highly efficient protocol that differentiates human pluripotent stem cells (hPSCs) into the developmental precursor of placental cells, the trophectoderm (TE), under chemically defined conditions. The in vitro generation of TE cells holds great promise for modeling diseases of the placenta, drug screening, and cell-based therapies.

Glucocerebrosidase Activators as a Treatment for Gaucher Disease

This technology is a collection of small molecule activators of a genetically defective version of the enzyme called glucocerebrosidase (GCase), which causes Gaucher disease. Gaucher disease is a rare disease affecting 1 in 40,000 babies born. Ashkenazi Jews of eastern European descent (about 1 in 800 live births) are at particular risk of carrying this genetic defect. It is caused by inherited genetic mutations in the gene that encodes GCase, which result in reduced activity of the enzyme.

2-substituted Pyridines and Their Methods for Inhibiting BMP Signaling 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 an identical heterozygous activating mutation (R206H) in the gene encoding ACRV1 (also known as ALK2), a BMP type 1 receptor.

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.

Pyrazolo[1,5-a]pyrimidine Derivatives as Selective ALK Kinases Inhibitors for Inhibition of the Bone Morphogenetic Proteins Signaling Pathway for Treatment of Fibrodysplasia Ossificans Progressiva

This technology includes compounds which are selective inhibitors of anaplastic lymphoma kinases (ALK1, ALK2, ALK3 and ALK6), which inhibit these ALKs with low nanomolar potency. These compounds could be developed as a treatment of Fibrodysplasia ossificans progressiva (FOP) and other BMP-related diseases. FOP is a rare congenital disease with no current treatment options. Since the disease is driven by constitutively active ALK2, inhibition of ALK2 would be like hitting the Achilles’ heel of the disease and would potentially be an efficacious therapy for FOP patients.

CD206 Small Molecule Modulators, Their Use and Methods for Preparation

Pancreatic ductal adenocarcinoma (PDA) accounts for more than 90% of pancreatic cancer cases, and it is one of the most aggressive malignancies with a 5-year survival rate of 6%. The high mortality rate caused by PDA is primarily from the lack of early diagnosis – it is often asymptomatic in early stages – and a poor response to conventional chemotherapy and radiotherapy. One of the major immune cell types present in the PDA microenvironment is a subset of macrophages commonly termed tumor-associated macrophages (TAM).

Autophagy Modulators For Use in Treating Cancer

Cancer cells can upregulate autophagy – cell destruction – as a response to chemotherapy. Investigators in Dr. Melvin DePamphilis’ laboratory at the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) have shown that compounds identified by screening a library of compounds blocks autophagy in some cancer cells (e.g., melanoma) but are not toxic to normal cells. Cancer cells with mutations in the BRAF oncogene are especially dependent on autophagy. Treatment of cancer cells with the BRAF mutation can increase the efficacy of chemotherapy.

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. 

Topical Sodium Nitrate Ointment for Sickle Cell Disease

Chronic leg ulcers are a debilitating vasculopathic complication for some patients with sickle cell disease (SCD). Prevalence of leg ulcers varies based on age and geographic location; about 5-10% of all SCD patients may suffer leg ulcers. These leg ulcers are painful, result in infections, hospitalization, disability, and negatively impact the patient’s social and psychological wellbeing on an ongoing basis.

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