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Available for licensing and commercial development is a three-dimensionally configured curved catheter for safe traversal of the right atrial appendage (RAA). The device is configured to optimize one-way access of the pericardial space through the right atrium and into the RAA reducing the risk of coronary lacerations. Specifically the curved catheter is best described in three segments: a proximal segment, a transitional segment and a distal segment; the transition segment having a clockwise spiral shaped curvature.

This invention is a platform technology that pertains to the advantages of conjugating therapeutics to Evans Blue thus providing long lasting pharmacokinetic profiles by complexing with albumin. Notably, albumin bound therapeutic- or prodrug-Evans Blue conjugates provide a complex with a total molecular size above 60 kDa thus eliminating the risk for renal clearance. Interestingly, since albumin also crosses the blood-brain barrier and since all circulating Evans Blue is bound to albumin, Evans Blue bound therapeutics or prodrugs can also cross the blood-brain barrier.

Left ventricular outflow tract obstruction is a life-threatening complication of transcatheter mitral valve replacement caused by septal displacement of the anterior mitral leaflet (AML). The AML is a mobile structure that physically separates inflow and outflow zones of the left ventricle. Preserving the AML during surgical mitral valve replacement can cause left ventricular outflow tract obstruction, either when the prosthesis struts protrude into the left ventricular outflow tract or when along redundant anterior leaflet prolapses into the left ventrical outflow tract.

This technology includes the identification and use of a combination therapy consisting of human recombinant N-acetylgalactosamine-6-sulfate sulfatase (hrGALNS) and the pharmacological chaperone compounds Ezetimibe and Pranlukast for the treatment of Mucopolysaccharidosis Type IVA (MPS IVA). MPS IVA is a rare disease caused by mutations in the gene encoding the lysosomal enzyme N-acetylgalactosamine-6-sulfate sulfatase (GALNS). Currently, hematopoietic stem cell transplantation (HSCT) and enzyme replacement therapy (ERT) are available for patients with MPS IVA.

This technology includes the use of the combination of the compounds Chroman-1 and Emricasan to achieve virtually 100% cell survival during human pluripotent stem cell passaging, cryopreservation/thawing, and differentiation in 2D and 3D cultures. Human pluripotent stem cells, including ESCs and iPSCs, are highly sensitive cells and undergo apoptosis during these routine procedures. A screening approach was used to identify the combination of the two compounds in this invention.

This invention relates to two devices that reliably, sensitively, and accurately measures force during handgrip contraction and subsequent relaxation. A delayed relaxation after a sustained and forceful handgrip is a cardinal symptom of myotonic dystrophies (DM). This delayed relaxation, handgrip myotonia, may be a therapeutic response biomarker in clinical trials.

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.

This technology includes methods for screening compounds and compositions useful for inhibiting or reducing platelet deposition, adhesion, and/or aggregation. The present invention further relates to methods of treatment or prophylaxis of thrombotic disorders, including stroke, myocardial infarction, unstable angina, abrupt closure following angioplasty or stent placement, thrombosis induced by peripheral vascular surgery, peripheral vascular disease or thrombotic disorders resulting from atrial fibrillation or inflammation.

This technology includes a series of diphenylpropanamides as potent and selective RORgt inhibitors for the treatment of Th17-related autoimmune diseases. The retinoic acid-related orphan receptor RORgt plays an important role in the differentiation of thymocytes, lymphoid tissue inducer cells, and inflammatory T helper-expressing interleukin 17a (Th17) cells. Small molecule RORgt inhibitors may provide means to regulate Th17 mediated immune response. The novel molecules have potential to treat Th17-related autoimmune diseases.

This technology includes novel systemic adeno-associated virus (AAV)-mediated CRISPR gene therapy technology. While some diseases (e.g., retinal diseases) can be treated through local gene transfer, many diseases such as Duchenne Muscular Dystrophy (DMD) require systemic therapy. The CRISPR technology has two components, the Cas9 endonuclease, and the gRNA. To explore systemic CRISPR therapy, we co-delivered the AAV.Cas9 and AAV.gRNA vector to mdx mice, a mouse DMD model. Direct delivery to muscle yielded efficient gene correction.