Abstract: The present disclosure generally relates to compositions comprising fibrin and to methods of forming such compositions. In an embodiment, a method of forming fibrin particles is provided. The method includes introducing a buffer, a fibrinogen solution, and a thrombin solution to a first end of a microfluidic device to form a mixture, the buffer comprising one or more amino acids. The method further includes contacting the mixture with a fluorocarbon oil and a surfactant to form fibrinogen-containing particles, and applying positive pressure to the microfluidic device to cause the fibrinogen-containing particles to flow towards a second end of the microfluidic device. The method further includes collecting the fibrinogen-containing particles at the second end of the microfluidic device; and polymerizing the fibrinogen-containing particles to form fibrin particles.

Abstract: Provided are methods for lyophilization of an aqueous thrombin solution, thrombin solutions for use in such lyophilization methods, and solid thrombin compositions produced by such methods.

Abstract: Methods and systems for vibration induced membrane rupture include an epiretinal membrane (ERM) treatment method that includes providing a vibrational device that includes an interface configured for engaging a closed eyelid of an eye of a patient, where the eye of the patient includes an ERM disposed on an epiretinal surface of a retina of the eye. The interface of the vibrational device engages with the closed eyelid and an instance of vibrational force is applied, via the vibrational device while the interface is engaged with the closed eyelid, to the eyelid, where the vibrational force is capable of propagating through the closed eyelid to the retina of the eye. Further, the instance of vibrational force is applied at predetermined frequency (s) and predetermined intensity(s) for predetermined period(s) of time.

Abstract: The invention provides a combination treatment for ischemia conditions in or otherwise affecting the CNS, such as stroke. The treatment involves administration of a PSD-95 inhibitor and performing reperfusion therapy (e.g., by administration of tPA). Administering a PSD-95 inhibitor in combination with reperfusion therapy increases the efficacy of the reperfusion therapy and/or slows the decline in efficacy of reperfusion therapy with time after onset of ischemia thus extending the window in which reperfusion therapy can be administered.

Abstract: Provided is a method of forming a fibrin hydrogel composition, the method including providing a fibrin hydrogel or precursor thereof, comprising fibrin hydrogel forming salt. The fibrin hydrogel forming salt concentration is greater than or equal to the threshold concentration to form a fibrin hydrogel. The method further includes combining the fibrin hydrogel with a carrier material. The concentration of the carrier material typically ranges from 0.1 to about 50 wt.-%. The method further includes reducing the salt concentration below the threshold concentration to form a fibrin hydrogel.

Abstract: Provided herein are cyclic polypeptide compounds that can, e.g., bind specifically to human proprotein convertase subtilisin/kexin type 9 (PCSK9) and optionally also inhibit interaction between human PCSK9 and human low density lipoprotein receptor (LDLR), and pharmaceutical compositions comprising one or more of these compounds. Also provided are methods of reducing LDL cholesterol level in a subject in need thereof that include administering to the subject one or more of the cyclic polypeptide compounds or a pharmaceutical composition provided herein.

Abstract: The disclosure relates to nanoparticles comprising a lipid component and a modified RNA encoding a VEGF-A polypeptide. Aspects of the disclosure further relate to uses of nanoparticles comprising a lipid component and a modified RNA encoding a VEGF-A polypeptide, for improving wound healing in a subject.

Abstract: Disclosed herein are methods and compositions for the treatment and/or prevention of ischemia and ischemia-reperfusion injury comprising administration of at least one peptide disclosed herein, or a pharmaceutically acceptable salt thereof.

Abstract: The present invention provides a synthetic MMACHC polynucleotide comprising a polynucleotide encoding MMACHC that is codon-optimized for expression in a human. Also provided is a polypeptide encoded by a synthetic MMACHC polynucleotide, an expression vector comprising a MMACHC gene sequence under the control of a chicken beta actin (CBA) promoter, and an expression vector comprising a synthetic MMACHC polynucleotide. Methods of treating cobalamin C deficiency and for detecting or tracking exogenous MMACHC are also provided.

Abstract: A hyperglycosylated recombinant human coagulation factor IX (FIX) fusion protein, a preparation method therefor, and use thereof. The fusion protein sequentially comprises, from N- to C-terminus, a human FIX, a flexible peptide linker, at least one human chorionic gonadotropin ? subunit carboxy-terminal peptide rigid unit, and a half-life extending moiety. The fusion protein has a biological activity similar to that of the recombinant FIX, an extended in vivo activity half-life, and reduced immunogenicity, so as to improve pharmacokinetics and pharmacodynamics.

Abstract: The present invention is directed to a flowable hemostatic paste comprising a crosslinked carboxymethyl cellulose and at least one non-toxic dispersant. More specifically the present invention relates to a hemostatic paste containing citric acid cross-linked CMC, which is suspended or dispersed as a powder in a mixture of a first non-toxic glycerol-containing hygroscopic dispersant and a second non-toxic alcohol functionalized dispersant comprising propylene glycol or 1,3-butanediol.

Abstract: The present invention relates generally to agents and devices for promoting hemostasis and tissue sealing and, more particularly, to hemostatic pads comprising bioabsorbable scaffolds that can deliver lyophilized hemostasis promoting proteins, such as fibrinogen and thrombin, to a wound site or injured organ or tissue.

Abstract: The present invention provides compounds, compositions thereof, and methods of using the same.

Abstract: A method of treating, preventing, or delaying the progression of Type 1 diabetes mellitus autoimmunity by administering an effective amount of a cytotoxic T-lymphocyte-associated antigen 4 (CTLA4) molecule is provided herewith. The CTLA4 molecule may be a fusion protein of a CTLA4 extracellular region and an immunoglobulin, such as abatacept.

Abstract: The present invention relates to an antimicrobial composition at the pH of the skin. Particularly, it relates to an antimicrobial composition comprising water soluble vinyl polymer, a carboxylic acid having pKa greater than 4.5; and an anionic surfactant, wherein the composition has pH in the range 4.5 to 6.5. The compositions provides synergistic antimicrobial effect in relatively shorter contact time. The invention further relates to a method of disinfecting a surface using the composition.

Abstract: Provided herein are methods for safe and effective thrombolysis in therapy for human subjects with symptoms of a potential stroke or acute myocardial infarction (“AMI”) using a sequential administration of a low dose bolus of human tissue plasminogen activator (“tPA”) followed by an infusion of a mutant form of human pro-urokinase (“proUK”).

Abstract: The present invention relates to variants of factor IX (F.IX) or activated factor IX (F.IXa), wherein the variant is characterized in that it has clotting activity in absence of its cofactor. The present invention furthermore relates to variants of factor IX (F.IX) or activated factor IX (F.IXa), wherein the variant is characterized in that it has increased F.IX clotting activity compared to wildtype. The present invention furthermore relates to the use of these variants for the treatment and/or prophylaxis of bleeding disorders, in particular hemophilia A and/or hemophilia B or hemophilia caused or complicated by inhibitory antibodies to F.VIII. The present invention also relates to further variants of factor IX (F.IX) which have desired properties and can, thus be tailored for respective specific therapeutic applications.

Abstract: The present invention relates to the novel trypsin ZT isoforms. In particular, the invention relates to the use of trypsin ZT isoforms in medical devices, pharmaceuticals and cosmetics.

Abstract: Provided herein are methods for use in safe and effective thrombolysis, e.g., in therapy for a potential stroke or acute myocardial infarction (“AMI”) at a maximum arterial patency rate with minimal associated hemorrhagic side effects.

Abstract: The present disclosure describes use of oxytocin, in combination with one or more other agents as described herein, for use for example in the treatment of certain conditions.

Abstract: The subject invention relates to diffusion enhancing compounds and their use alone or with thrombolytic agents for the treatment of disorders resulting from the formation of a thrombus such as a myocardial infarction or stroke.

Abstract: The present invention provides for methods and at least partially automated devices suitable for producing a transplantable cellular suspension of living tissue suitable for promoting tissue regeneration in an epithelium-related procedure, as well as compositions produced therefrom. Tissue regeneration in humans is extremely limited and constitutes a major challenge to the repair of damaged organ function. Wound treatment is a typical area where tissue regeneration is required. Wounds (lacerations or openings) in mammalian tissue can result in tissue disruption and coagulation of the microvasculature at the wound face.

Abstract: The present invention is directed to compositions comprising topiramate and a sulfoalkyl ether cyclodextrin, and methods of making and using the same.

Abstract: The present disclosure relates to a dry composition which reconstitutes without mechanical mixing to form a flowable paste having a soft and light consistency suitable for use in haemostasis and wound healing procedures upon addition of an aqueous medium. The disclosure further relates to methods of preparing the dry composition, methods for reconstituting the dry composition and medical use of the composition.

Abstract: The present invention is directed to devices having a substance receiving portion; a membrane; and at least one blocking member configured for taking one position and another position to allow fluid communication when a vacuum force is applied to the bottom of the substance receiving portion for the purpose of an in-situ preparation of a sample to detect the presence of, and/or quantify, clottable fibrinogen. The present invention is also directed to corresponding methods, processes and systems.

Abstract: The invention provides a combination treatment for ischemia conditions in or otherwise affecting the CNS, such as stroke. The treatment involves administration of a PSD-95 inhibitor and performing reperfusion therapy (e.g., by administration of tPA). Administering a PSD-95 inhibitor in combination with reperfusion therapy increases the efficacy of the reperfusion therapy and/or slows the decline in efficacy of reperfusion therapy with time after onset of ischemia thus extending the window in which reperfusion therapy can be administered.

Abstract: This disclosure relates to compositions for use in managing respiratory distress and related disorders. In certain embodiments, the disclosure relates to methods of treating or preventing respiratory distress comprising administering an effective amount of a pharmaceutical composition comprising peptides or agents disclosed herein to a subject in need thereof. In certain embodiments, the peptides or agents decrease the concentration of claudin-5 in cells and tissues of the lungs.

Abstract: Provided are spray-dried thrombin powders comprising microcapsules, methods of preparation and uses thereof.

Abstract: This disclosure relates to compositions for use in managing respiratory distress and related disorders. In certain embodiments, the disclosure relates to methods of treating or preventing respiratory distress comprising administering an effective amount of a pharmaceutical composition comprising peptides or agents disclosed herein to a subject in need thereof. In certain embodiments, the peptides or agents decrease the concentration of claudin-5 in cells and tissues of the lungs.

Abstract: The present invention relates antidotes to anticoagulants targeting factor Xa. The antidotes are factor Xa protein derivatives that bind to the factor Xa inhibitors thereby substantially neutralizing them but do not assemble into the prothrombinase complex. The derivatives describe herein lack or have reduced intrinsic coagulant activity. Disclosed herein are methods of stopping or preventing bleeding in a patient that is currently undergoing anticoagulant therapy with a factor Xa inhibitor.

Abstract: The present invention provides an agent for reducing a side effect of an anticancer drug, which comprises a thrombin-like enzyme.

Abstract: The present invention relates to variants of factor IX (F.IX) or activated factor IX (F.IXa), wherein the variant is characterized in that it has clotting activity in absence of its cofactor. The present invention furthermore relates to variants of factor IX (F.IX) or activated factor IX (F.IXa), wherein the variant is characterized in that it has increased F.IX clotting activity compared to wildtype. The present invention furthermore relates to the use of these variants for the treatment and/or prophylaxis of bleeding disorders, in particular hemophilia A and/or hemophilia B or hemophilia caused or complicated by inhibitory antibodies to F.VIII. The present invention also relates to further variants of factor IX (F.IX) which have desired properties and can, thus be tailored for respective specific therapeutic applications.

Abstract: The invention relates to short RVG derived peptides for use in delivering therapeutic agents across the blood brain barrier and to target cells, for example those cells in the central nervous system. The invention provides method and compositions to treat diseases, such as neurodegenerative diseases, with therapeutic agents associated with targeting peptides.

Abstract: The instant disclosure provides replacement gene tolerizing vectors and methods of using such vectors for treating individuals receiving gene therapy for muscular dystrophy, e.g., Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD), etc. Aspects of the methods include administering to the individual, in need thereof, an effective amount of a gene therapy tolerizing vaccine composition that includes a replacement gene tolerizing vector to reduce one or more symptoms of a subject's immune response to gene therapy treatment for muscular dystrophy and/or improve the efficacy of the gene therapy. Compositions and kits for practicing the methods of the disclosure are also provided.

Abstract: The present invention provides methods of producing thrombin using coordinated coexpression systems, and particularly inducible coexpression systems, capable of controlled induction of expression of each gene product required for the production of thrombin, and the thrombin produced by this method.

Abstract: The present invention provides methods of administering a clotting factor by a fixed dosing regimen; methods of reducing, ameliorating, or preventing one or more symptoms of a bleeding disease or disorder; and a kit comprising a dotting factor useful for a fixed dosing regimen. While plasma-derived and recombinant clotting factor products allow hemophilia patients to live longer and healthier, hemophilia still remains one of the most costly and complex conditions to manage.

Abstract: Provided herein is a method of treating a subject believed to have suffered a stroke. This method comprises administering to the subject a therapeutically effective amount of a composition comprising conivaptan or an active derivative, stereoisomer, pro-drug, or pharmaceutically acceptable salt thereof.

Abstract: A method of treating a cardio-renal disease is described that includes administering to a subject in need thereof a therapeutically effective amount of proprotein convertase subtilisin/kexin-6 (PCSK6), or an effective fragment thereof, which functions as a corin activator.

Abstract: The present invention relates to methods for the manufacture, purification, formulation, and use of biologically active recombinant elastase proteins. Described are recombinant methods for producing therapeutically useful elastase proteins, as are pharmaceutical compositions comprising said elastase proteins. Novel recombinant elastase proteins and protein preparations are also disclosed. Methods are described for treating and preventing diseases of biological conduits using pharmaceutical compositions containing the elastase proteins of the invention.

Abstract: This disclosure relates to the use of EACA (6-aminohexanoic acid) as an antifibrinolytic for the prevention of thrombocytopenic bleeds. In certain embodiment, the disclosure relates to methods of preventing bleeding in patients with thrombocytopenia resulting from hematological malignancies by administering 6-aminohexanoic acid, salts, or prodrugs thereof prophylactically to a subject in need thereof. In certain embodiments, subjects with stable chronic thrombocytopenia previously managed with platelet transfusion represent a selected group of patients that benefit from prophylaxis.

Abstract: Provided herein are methods for using a thrombin peptide derivative to treat a medulloblastoma. In one embodiment, the method includes administering to a subject having a medulloblastoma an effective amount of a thrombin peptide derivative, and exposing cells of the medulloblastoma of the subject to a cancer treatment after administering the thrombin peptide derivative to the subject. An example of a cancer treatment is radiation therapy. In one embodiment, viability of cells of the medulloblastoma is decreased compared to viability of cells of the medulloblastoma before the administering and the exposing. The cells of the medulloblastoma having decreased viability can be cancer stem cells. In one embodiment, shrinkage of the medulloblastoma in the subject is increased compared to shrinkage of the medulloblastoma before the administering and the exposing.

Abstract: Pharmaceutical formulations in suppository form, containing thrombolytic agents, which are able to penetrate rectal mucous, for the treatment of acute hemorrhoid diseases and/or diseases of thrombotic origin.

Abstract: The application discloses methods and compositions for the inhibition of the alternative complement pathway. The methods and compositions involve the use of aptamers for inhibiting complement Factor D. The application further provides anti-Factor D aptamers for the treatment of dry age-related macular degeneration, geographic atrophy, wet age-related macular degeneration or Stargardt disease.

Abstract: A composition is disclosed that includes pure silk fibroin-based protein fragments that are substantially devoid of sericin, wherein the composition has an average weight average molecular weight ranging from about 17 kDa to about 38 kDa, wherein the composition has a polydispersity of between about 1.5 and about 3.0, wherein the composition is substantially homogeneous, wherein the composition between 0 ppm to about 500 ppm of inorganic residuals, and wherein the composition includes between 0 ppm to about 500 ppm of organic residuals.

Abstract: Provided are spray-dried thrombin powders comprising microcapsules, methods of preparation and uses thereof.

Abstract: Methods and compositions containing a phorbol ester or a derivative of a phorbol ester are provided for the treatment and prevention of stroke and the sequelae of stroke. Additional compositions and methods are provided which employ a phorbol ester or derivative compound in combination with at least one additional agent to yield more effective treatment tools to treat or prevent stroke and the long term effects of stroke in mammalian subjects.

Abstract: Modified canine leptin polypeptides and formulations and uses thereof, are provided including polyethylene glycol (PEG) modified canine leptin polypeptides, wherein the PEG moiety is covalently attached to a para-acetyl-phenylalanine (pAF) residue of the polypeptide, and related compositions and methods useful in treating companion animal obesity and other leptin-related disorders.

Abstract: The invention provides a combination treatment for ischemia conditions in or otherwise affecting the CNS, such as stroke. The treatment invoices administration of a PSD-95 inhibitor and performing reperfusion therapy (e.g. by administration of tPA). Administration a PSD-95 inhibitor in combination with reperfusion therapy increases the efficacy of the reperfusion therapy and/or slows the decline in efficacy of reperfusion therapy with time after onset of ischemia thus extending the window in which reperfusion therapy can be administered.

Abstract: The present invention is in the field of hemophilia therapy. It relates to a new variant of antihemophilic factor VIII having increased specific activity in comparison to known factor VIII products.

Abstract: Factor VII variants and methods of use thereof are disclosed.