Treatment of primary hyperoxalurias with small molecule lactate dehydrogenase inhibitors such as WO2018005807A1

This technology includes the use of novel lactate dehydrogenase (LDH) inhibitors, including WO2018005807A1, for the treatment of primary hyperoxalurias (PHs). PHs are rare autosomal recessive disorders caused by overproduction of oxalate, leading to recurrent calcium oxalate kidney stone disease, and in some cases end-stage renal disease. One potential strategy to treat PHs is to reduce the production of oxalate by diminishing the activity of LDH, the proposed key enzyme responsible for converting glyoxylate to oxalate.

Novel Codon-Optimized MUT Gene Therapeutic for Methylmalonic Acidemia (MMA)

Methylmalonic Acidemia (MMA) is a metabolic disorder characterized by increased acidity in the blood and tissues due to toxic accumulation of protein and fat by-products resulting in seizures, strokes, and chronic kidney failure. A significant portion of MMA cases stem from a deficiency in a key mitochondrial enzyme, methylmalonyl-CoA mutase (MUT), required to break down amino acids and lipids. Currently, there are no treatments for MMA and the disease is managed primarily with dietary restriction of amino acid precursors and liver-kidney transplantation in severe cases.

Fibroblast Cell Lines (with L444P/RecNci1 Genotype) for the Screening of Small Molecules for Gaucher Disease Treatment

This technology includes two human fibroblast cell lines to be used to study the defects in GBA1 gene and protein and to screen small molecules for involvement in Gaucher disease. Glucocerebrosidase (GBA1 or GCase or beta-glucosidase) is a lysosomal enzyme, responsible for breakdown of a fatty material called glucocerebroside (or glucosyl ceramide). Deficiency or malfunction of GBA1 leads to the accumulation of insoluble glucocerebrosides in tissues, which is a major symptom of Gaucher disease.

In-vivo System to Interrogate the Functions of Mucous Membranes and Identify Mucin/Glycan Mimetics and JAK/STAT Inhibitors for the Treatment of Diseases of the Oral Cavity and Digestive Tract

This technology includes a Drosophila mutant strain that can be used as an in vivo model for diseases of the oral cavity and digestive tract (Sjogren's syndrome, colitis, colon cancer, inflammatory bowel disease), where the mucous membrane is disrupted or non-functional. This mutant lacks a mucous membrane and displays epithelial cell damage, uncontrolled cell proliferation and the up-regulation of conserved signaling pathways (JAK/STAT).

Trans-auricular Left Atrial Appendage Ligation to Prevent Thrombosis

This technology includes an interventional device to occlude the left atrial appendage to prevent thrombus formation. Atrial fibrillation is the most common cardiac arrhythmia and is associated with formation of thrombus in the left atrial appendage. Standard preventative treatment involves anticoagulation, which is not tolerated by all patients. Existing devices necessitate improvement because they need trans-septal puncture and anticoagulation to prevent thrombus or are prone to life-threatening complications.

Single Scan Bright-blood and Dark-blood Phase Sensitive Inversion Recovery (PSIR) Late Gadolinium Enhancement (LGE) for Cardiovascular Magnetic Resonance (CMR) Imaging

This technology includes a technique to improves detection of myocardial scar compared with conventional bright-blood late gadolinium enhancement (LGE) techniques. Dark-blood late gadolinium enhancement (DB-LGE) improves tissue delineation with signal suppression of the blood pool based on T2-preparation pulse that is relatively independent from the blood flow velocities and improves scar detection in patients with known or suspected coronary artery disease.

Vascular Anchoring Introducer Sheath for Interventional Cardiac Procedures

This technology includes a device and method for maintaining access to a location in the body while reducing or eliminating the potential for pulling an access device (i.e., catheter) back through an opening, such as a cardiac procedure. An introducer sheath includes a distal indented portion and a balloon, so that once placed in a desired location through tissue, the balloon can be inflated to anchor the sheath against retraction.

Compositions and Methods for Reducing Serum Triglycerides

This technology includes a vaccine for lowering plasma triglycerides by inducing the formation of autoantibodies against either ANGPTL3 or ANGPTL4, which are inhibitors of Lipoprotein Lipase. This was done by conjugating synthetic peptides based on ANGPTL3 or ANGPTL4 to virus- like particles (VLPS). Injection of the vaccine in animal models was shown to induce the autoantibody against the target and to lower plasma triglycerides.

Systems and Methods for Applying Pressure to the Heart for the Treatment of Tricuspid Valve Regurgitation

This technology includes structures and methods for cinching a band around the heart for treating conditions including tricuspid valve regurgitation (TR). When positioned appropriately along the atrioventricular groove, the band is tightened around the heart which narrows the tricuspid annulus and relieves TR.

Helical Guidewires and Related Systems for Transcatheter Heart Valve Procedures

This technology includes a guidewire purpose-built for delivery of bulky transcatheter heart valves (THV). Conventional THV guidewires are rigid and have a distal tip shaped like a pigtail to prevent apical ventricular perforation. This invention is a 3-dimensional helical or antihelical curve that can protect against apical perforation, possibly better, and that allows subtle 3-mensional deflection to aid the operator in achieving coaxiality or overcoming delivery obstacles such as calcific spicules.

Subscribe to Cardiology