Abstract: The present invention relates generally to medicine. More specifically the invention relates to the use of synergistic probiotic bacteria as intervention for health. In particular, the present invention provides a strain of Faecalibacterium prausnitzii and a bacterial strain which has one or more of the characteristics of: (i) being acetate producing, (ii) being lactate consuming and (iii) having the ability to be an electron acceptor, for use in the treatment or prevention of a disease associated with reduced butyrate levels or a disease associated with reduced or low numbers of Faecalibacterium prausnitzii bacteria.
Abstract: The present invention generally relates to therapeutic compositions and methods for treating cancer patients. The compositions and methods can increase efficacy of an anti-cancer therapy, or treat, prevent, or inhibit an oncology-treatment induced condition (OTIC) induced by an anti-cancer therapy. The therapeutic compositions comprise microbial compositions and, optionally, an anti-cancer therapeutic agent. The methods comprise administering a therapeutic composition comprising a microbial composition and administering an anti-cancer therapy.
Type:
Application
Filed:
February 22, 2019
Publication date:
December 31, 2020
Inventors:
Joseph LOBACKI, Ulrich THIENEL, Zain KASSAM, Marina SANTIAGO
Abstract: Compositions for the treatment of colorectal cancer target bacterial biofilms in the gastrointestinal tract. Methods of treatment include one or more agents which target bacteria and the bacterial biofilms.
Type:
Application
Filed:
February 28, 2019
Publication date:
December 31, 2020
Applicant:
The Johns Hopkins University
Inventors:
Cynthia L. Sears, Christine Craig, Drew M. Pardoll
Abstract: Novel nutritional compositions and methods for their manufacture, administration and use are disclosed. The nutritional compositions of the present invention are useful for the feeding of immature mammals, particularly those used for food, and/or performance animals, such as horses, and for companion animals, such as dogs and cats. The nutritional compositions and supplements of the present invention are effective in supporting the growth and health of the immature mammal, in supporting and stimulating its immune system, and in mitigating undesirable infections and other. Additionally, particular fractions of the unique peptides produced by the disclosed methods can be separated and utilized as therapeutic agents.
Abstract: A composition comprising at least two bacterial strains selected from the group consisting of Bacillus subtilis 281, Bacillus subtilis 3, Bacillus amyloliquefaciens 298 and a functional homolog of any of the preceding, wherein the composition does not comprise more than 5 different species of microbes, wherein: a sample of Bacillus subtilis 281, has been deposited as KCTC 13468BP at the Korean Collection for Type Cultures; a sample of Bacillus subtilis 3, has been deposited as KCTC 13467BP at the Korean Collection for Type Cultures; and a sample of Bacillus amyloliquefaciens 298, has been deposited as KCTC 13469BP at the Korean Collection for Type Cultures. Also provided are methods that utilize the bacterial strains or functional homologs of same.
Type:
Application
Filed:
September 14, 2020
Publication date:
December 31, 2020
Applicant:
Better Air International Limited
Inventors:
Wilhelm H. HOLZAPFEL, Yosep JI, Yuli HORESH, Michael HOFFMAN, Shimrit LAOR
Abstract: The inventive technology relates to novel paratransgenic strategies for the biocontrol of pathogens in animal systems using interfering RNA molecules expressed in genetically modified bacteria that may be configured to colonize a target host. In one preferred embodiment, the invention includes novel paratransgenic strategies for the biocontrol of pathogens in aquatic organisms raised in aquaculture environments.
Type:
Application
Filed:
September 14, 2020
Publication date:
December 31, 2020
Inventors:
Richard T. Sayre, Tatiana Vinogradova-Shah, Elena Sineva
Abstract: The use of orally administrable compositions comprising Streptococcus salivarius is described, for the prevention or the treatment of psoriasis in all its forms. In particular, the present invention relates to the use of compositions comprising Streptococcus salivarius, for the treatment of psoriasis in all its forms.
Type:
Application
Filed:
January 24, 2019
Publication date:
December 31, 2020
Inventors:
Stefano Di Girolamo, Laura Diluvio, Arianna Zangrilli
Abstract: Provided is a novel prophylactic and/or therapeutic agent for Pseudomonas aeruginosa infection. It was found that a bacterium belonging to the genus Enterococcus can prevent and/or treat Pseudomonas aeruginosa infection. The prophylactic and/or therapeutic agent for Pseudomonas aeruginosa infection, comprising a bacterium belonging to the genus Enterococcus. A medicine for prevention and/or treatment of Pseudomonas aeruginosa infection, comprising a bacterium belonging to the genus Enterococcus. A food for prevention and/or treatment of Pseudomonas aeruginosa infection, comprising a bacterium belonging to the genus Enterococcus.
Abstract: In alternative embodiments, provided are probiotic formulations, including compositions, formulations, products of manufacture and kits comprising them, and methods of using them, for the treatment and alleviation of metabolic and oxidative stress, inflammation and neurodegeneration. In alternative embodiments, probiotic formulations as provided herein comprise or consist of any combination of the three probiotic strains: Lactobacillus fermentum NCIMB 5221, Lactobacillus plantarum NCIMB 8826 and Bifidobacteria longum spp. infantis NCIMB 702255. In alternative embodiments, probiotic formulations as provided herein are combined with a triphala. In alternative embodiments, probiotic formulations as provided herein are used for: the treatment of metabolic conditions such as diabetes and obesity, inflammatory conditions such as IBD, IBS, arthritis; and, neurodegenerative diseases such as Alzheimer's disease and Parkinson's disease.
Abstract: The present invention provides a pharmaceutical composition comprising dead cells of Lactobacillus strains useful for the protection of subjects against the development of conditions with behavioral, psychological and/or physical components caused or exacerbated by stress or anxiety, and/or useful in treating existing conditions with behavioral, psychological and/or physical components caused or exacerbated by stress or anxiety. Examples of specific conditions include stress, anxiety, depression, mood disturbances, sociability disorders, irritable bowel syndrome, autism, autism spectrum disorder, post-traumatic stress disorder, chronic stress and a range of other stress-related diseases.
Type:
Application
Filed:
March 22, 2019
Publication date:
December 31, 2020
Inventors:
Colin HILL, Ted DINAN, Alicja WARDA, Stephen PERRETT
Abstract: The present invention relates to a novel Lactobacillus sp. isolation strain having an activity to inhibit the growth of vaginitis pathogens, and a pharmaceutical composition, a health functional food, and a cleansing product, comprising the strain as an active ingredient. Therefore, the present invention exhibits an effect of inhibiting the growth of Sneathia spp. pathogens associated with the infection with human papillomavirus (HPV) and the incidence of bacterial vaginitis, Gardnerella vaginalis as a vaginitis pathogen, and Candida albicans as a causative bacteria of Candidal vaginitis, and an effect of recovering and maintaining the vaginal microflora, and thus can be used for the prevention and treatment of female vaginitis.
Abstract: The disclosure herein discloses L. plantarum CCFM1019, fermented foods thereof, and application thereof in preparation of a medicine for promoting excretion of plasticizers and metabolites thereof from the body. The L. plantarum CCFM1019 not only is significantly better than the intestinal resident bacteria E. coli and E. faecalis in terms of the effect of promoting the excretion of DEHP and MEHP, but also is better than the commercial strain L. rhamnosus LGG. Therefore, the L. plantarum CCFM1019 of the disclosure can be used as an effective means to prevent and alleviate body damage caused by DEHP and MEHP, and has no toxic side effects of drugs. L. plantarum CCFM1019 can be used to prepare pharmaceutical compositions and fermented foods for alleviating and preventing the toxicity of DEHP and metabolites thereof, and has a very broad application prospect.
Abstract: This document provides methods and materials related to using infectious nucleic acid encoding viruses to reduce the number of viable cancer cells within a mammal. For example, methods for using infectious nucleic acid to treat cancer, engineered viral nucleic acid, and methods for making engineered viral nucleic acid are provided.
Type:
Application
Filed:
March 12, 2019
Publication date:
December 31, 2020
Inventors:
Autumn J. SCHULZE, Stephen James RUSSELL, Noura B. ELSEDAWY
Abstract: An obligate oHSV vector comprising modified viral DNA genome is provided. A recombinant oHSV-1 construct comprising the obligate oHSV vector and a heterologous nucleic acid sequence encoding an immunostimulatory and/or immunotherapeutic agent is also provided. Compositions comprising the recombinant oHSV-1 construct can be used for treating cancers.
Type:
Application
Filed:
September 14, 2020
Publication date:
December 31, 2020
Inventors:
Grace Guoying Zhou, Xiaoqing Chen, Xianjie Liu
Abstract: Disclosed is the use of a mevalonate metabolic pathway inhibitor and an alphavirus in preparing an anti-tumor drug. The mevalonate metabolic pathway inhibitor can be used in preparing an alphavirus anti-tumor synergist. Disclosed are a pharmaceutical composition containing the mevalonate metabolic pathway inhibitor and the alphavirus, a drug kit containing the mevalonate metabolic pathway inhibitor and the alphavirus, and the use of the mevalonate metabolic pathway inhibitor and the alphavirus in treating tumors, especially those that are insensitive to the alphavirus.
Abstract: A pharmaceutical composition including an anticancer virus and hydroxyurea as effective components and its use for preventing or treating cancer are disclosed. A method for preventing or treating cancer includes administering an anticancer virus and hydroxyurea as effective components exhibits superior tumor suppression effect as compared to a conventional case where only an anticancer virus is administered; and can kill even the cancer cells that are resistant to anticancer viruses.
Abstract: Human clinical use of a chimeric poliovirus construct has demonstrated excellent anti-tumor effect. The mechanism of action is believed to involve both viral oncolysis as well as immune recruitment, both of which lead to necrosis in the area of the tumor. No adverse effects have been observed.
Type:
Application
Filed:
September 10, 2020
Publication date:
December 31, 2020
Applicant:
Duke University
Inventors:
Matthias Gromeier, John H. Sampson, Darell D. Bigner, Annick Desjardins, Henry S. Friedman
Abstract: The present invention is concerned with a photosynthetic scaffold that delivers oxygen and its uses for tissue engineering and the treatment of ischemia.
Abstract: A transmucosal delivery device comprising a mucosal permeation enhancing agent and a nanocarrier carrying a biologically active substance are embedded within a colloidal polymer thin film. Such a transmucosal delivery device is intended to deliver the biological active substance to a target location in a two-stage process. First, the colloidal polymer thin film is dissolved when adhered to a mucosal membrane. Once absorbed through the mucosal tissue and into system circulation, the nanocarrier is subject to a number of physiologic effects that will lead to the nanocarrier reaching a target location, such as a target tissue, wherein the biologically active substance dissolves and the biologically active substance payload is released.
Abstract: The present disclosure relates to a pharmaceutical composition for preventing or treating respiratory diseases and a food composition for preventing or relieving respiratory diseases comprising as an active ingredient two or more mixed extracts selected from the group consisting of a Brassicae Semen extract, an Adenophorae Radix extract, a Lonicerae Folium extract and a Perillae Semen extract. A composition comprising mixed extracts of Brassicae Semen, Adenophorae Radix, Lonicerae Folium and Perillae Semen of the present disclosure has an advantage of preventing respiratory diseases and relieving the symptoms of respiratory diseases, restores damages in the lung tissue, inhibits aging action and inflammation induced by inflammation-inducing substances such as fine dust, and has an excellent antioxidant effect. More particularly, the composition of the present disclosure increases the expression of a telomerase which extends the length of a telomere.
Type:
Application
Filed:
March 15, 2019
Publication date:
December 31, 2020
Inventors:
Mi Won SON, Min Jung BAE, Won Woo LEE, Doo Suk LEE
Abstract: The present invention relates to compositions containing plant materials or extracts with inhibitory effect on a 5-HT3a and/or NK-1 receptor for preventing or treating idiopathic vomiting.
Type:
Application
Filed:
April 20, 2018
Publication date:
December 31, 2020
Applicant:
Mars, Incorporated
Inventors:
Jean Soon Park, Ping Hu, Yakang Lin, Lori Ann Reinsalu
Abstract: This document provides methods and materials for treating a mammal having Huntington's disease. For example, methods and materials for forming GABAergic neurons that are functionally integrated into the brain of a living mammal (e.g., a human) and/or for modifying one or both huntingtin (Htt) genes (or HTT RNAs or HTT polypeptides) present in a mammal with Huntington's disease are provided.
Abstract: The present invention provides a novel multi-functional peptide that effectively regulates the activity of immune cells while also exhibiting excellent antibacterial activity against various bacteria such as Gram-negative bacteria and Gram-positive bacteria.
Type:
Application
Filed:
March 19, 2020
Publication date:
December 31, 2020
Applicant:
NOVACELL TECHNOLOGY INC.
Inventors:
Tae Hoon LEE, Jae Wang GHIM, Hyun Ju LEE
Abstract: This disclosure provides peptides which have a strong affinity for the checkpoint receptor “programmed death 1” (PD-1). These peptides block the interaction of PD-1 with its ligand PD-L1 and can therefore be used for various therapeutic purposes, such as inhibiting the progression of a hyperproliferative disorder, including cancer; treating infectious diseases; enhancing a response to vaccination; treating sepsis; and promoting hair re-pigmentation or lightening of pigmented skin lesions.
Type:
Application
Filed:
September 9, 2020
Publication date:
December 31, 2020
Inventors:
Gabriel M. Gutierrez, Vinayaka Kotraiah, James Pannucci, Ramses Ayala
Abstract: The present disclosure relates to a BIN1 protein or a BIN1 nucleic acid sequence producing or encoding the same, for a use in the treatment of X-linked centronuclear myopathy. The present invention provides compositions and methods for treatment of X-linked centronuclear myopathy. The present invention relates to a method of delivering the BIN1 polypeptide to subjects with X-linked centronuclear myopathy.
Abstract: Disclosed herein are fusion proteins for use in treating an inflammatory or immune disorder and methods of use. In some examples, the fusion proteins include an anchor domain and a therapeutic polypeptide. In some examples, the fusion proteins and methods herein can be used to treat inflammatory or immune disorders.
Type:
Application
Filed:
March 11, 2019
Publication date:
December 31, 2020
Applicant:
University of Pittsburgh - Of the Commonwealth System of Higher Education
Inventors:
Robert Michael Queen Shanks, Jes K. Klarlund
Abstract: Provided are methods for clinical treatment of cancers or tumors (e.g., advanced solid tumors) using (i) a combination of a tetanus toxoid, anti-OX40 antibody and anti-PD-1 antibody, (ii) a combination of anti-OX40 antibody and anti-PD-1 antibody, (iii) a combination of a tetanus toxoid and anti-PD-1 antibody, or (iv) an anti-PD-1 antibody.
Type:
Application
Filed:
February 7, 2019
Publication date:
December 31, 2020
Applicant:
Bristol-Myers Squibb Company
Inventors:
Michael QUIGLEY, Praveen AANUR, Zheng YANG
Abstract: Nutritive polypeptides are provided herein. Also provided are various other embodiments including nucleic acids encoding the polypeptides, recombinant microorganisms that make the polypeptides, vectors for expressing the polypeptides, methods of making the polypeptides using recombinant microorganisms, compositions and formulations that comprise the polypeptides, and methods of using the polypeptides, compositions and formulations.
Type:
Application
Filed:
July 1, 2020
Publication date:
December 31, 2020
Inventors:
Alison Williams, Nathaniel W. Silver, Andrew Downey, David V. Erbe, Michael J. Hamill, David Arthur Berry
Abstract: Disclosed herein are methods for the treatment of a patient having an ocular conditions such as age-related macular degeneration or diabetic macular edema through administration of a recombinant binding protein comprising an ankyrin repeat domain. In some embodiments, the composition may be administered every 8 weeks to every 16 weeks. In some embodiments, the patient being treated may be refractory to existing anti-VEGF therapies.
Abstract: This invention relates to combination therapies comprising a cyclin dependent kinase (CDK) inhibitor that inhibits CDK4 and/or CDK6, and a bromodomain and extra-terminal domain (BET) family inhibitor, and associated pharmaceutical compositions, methods of treatment, and pharmaceutical uses.
Abstract: A composition comprising a gene therapy product for use in the treatment of a dystrophic disease in a subject, advantageously in humans, wherein: the gene therapy product comprises a nucleic acid sequence encoding a functional microdystrophin; the composition is systemically administered.
Type:
Application
Filed:
August 24, 2020
Publication date:
December 31, 2020
Inventors:
George Dickson, Thomas Voit, Philippe Moullier, Caroline Le Guiner
Abstract: Embodiments of the disclosure include methods and compositions related to immunotherapy that targets CD5. In particular embodiments, immune cells engineered to comprise a chimeric antigen receptor (CAR) that targets CD5 are contemplated, and uses thereof. In particular embodiments, the immune cells expressing the CAR do not commit fratricide to any great extent against T cells that express CD5 and which are endogenous to an individual receiving the immune cells.
Abstract: The invention provides for methods of delivering heparin binding epidermal growth factor (HB-EGF) to sites of intestinal injury using stem cell derived exosomes. In particular, the invention provides for methods of delivering HB-EGF loaded stem cell-derived exosomes to sites of intestinal injury and methods of protecting a subject and method of treating intestinal injury, such as necrotizing enterocolitis (NEC).
Abstract: Provided herein are compounds, trifunctional antibody products thereof, and methods and pharmaceutical compositions for use in treatment of inflammatory and/or proliferative diseases
Type:
Application
Filed:
June 10, 2020
Publication date:
December 31, 2020
Inventors:
Alice YAM, Andreas MADERNA, Cristina ABRAHAMS, Willy SOLIS, Xiaofan LI, Ryan STAFFORD, Gang YIN, Venita DE ALMEIDA, Krishna BAJJURI, Adam A. GALAN
Abstract: In some aspects, the disclosure relates to GDF/BMP antagonists and methods of using GDF/BMP antagonists to treat, prevent, or reduce the progression rate and/or severity of pulmonary hypertension (PH), particularly treating, preventing or reducing the progression rate and/or severity of one or more PH-associated complications. The disclosure also provides methods of using a GDF/BMP antagonist to treat, prevent, or reduce the progression rate and/or severity of a variety of conditions including, but not limited to, pulmonary vascular remodeling, pulmonary fibrosis, and right ventricular hypertrophy. The disclosure further provides methods of using a GDF/BMP antagonist to reduce right ventricular systolic pressure in a subject in need thereof.
Abstract: The disclosure provides one-component, injectable, sustained release formulations which comprise microspheres containing active pharmaceutical ingredients (e.g., exenatide), wherein the microspheres are suspended in a non-aqueous carrier. The non-aqueous carrier can be an oil, a fractionated oil, triglycerides, diglycerides, monoglycerides, propylene glycol fatty acid diesters, and the like. The formulations offer distinct advantages of long shelf life for the stability and potency of the formulation and sustained release of active pharmaceutical ingredients to reduce the frequency of medication dosing and to increase patient compliance.
Type:
Application
Filed:
May 13, 2020
Publication date:
December 31, 2020
Inventors:
Mary L. Houchin, Robin H. Lee, Hong Qi, Greg Oehrtman, Robert N. Jennings, Scott H. Coleman
Abstract: The present application relates to novel methods for preventing, slowing the progression of, or treating nonalcoholic fatty liver (NAFL), nonalcoholic steatohepatitis (NASH), and/or liver fibrosis, and/or reducing the risks of liver cancer in subjects, such as HIV-infected subjects, using a GHRH molecule, e.g., trans-3-hexenoyl-GHRH(1-44)-NH2, or a pharmaceutically acceptable salt thereof. The subjects may have particular pathological features such as liver fibrosis, a hepatic fat fraction (HFF) of at least about 10%, serum alanine aminotransferase (ALT) levels of at least about 30 U/L, and/or a NAFLD Activity Score (NAS) of at least 4 or 5.
Abstract: This invention is directed to a general method for the chronic treatment, potential cure, or prevention of various metabolic and related diseases in people, including diabetes, by modulating IRS2 activity in cells and tissues in the body. IRS1 and IRS2 are part of the insulin or insulin-like growth factor signaling pathway. By upregulating the levels or functional activity of IRS2, insuling is used more efficiently by the body to control nutrient levels. By upregulating IRS2 levels or functional activity in pancreatic ?-cells, glucose sensing and insulin secretion are enhanced.
Abstract: Intestinal absorption is enhanced in short bowel syndrome patients presenting with colon-in-continuity by treatment with a GLP-2 receptor agonist, such as teduglutide.
Type:
Application
Filed:
September 3, 2020
Publication date:
December 31, 2020
Applicant:
SHIRE-NPS PHARMACEUTICALS, INC
Inventors:
Elizabeth L. SANGUINETTI, Thomas B. MARRIOTT, Jennifer LOPANSRI, Consuelo M. BLOSCH
Abstract: A composition in the form of an injectable aqueous solution, whose pH consists from 6.0 to 8.0, including at least: a basal insulin whose isoelectric point includes from 5.8 to 8.
Abstract: Disclosed are peptide-fatty acid conjugates, pharmaceutical compositions containing them, and methods of their medical use in the treatment of, e.g., a disease or condition associated with the PTHR1 signaling overactivity (e.g., hypercalcemia, hypophosphatemia, hyperparathyroidism, or Jansen's chondrodysplasia) or deficiency (e.g., hypoparathyroidism, hyperphosphatemia, osteoporosis, fracture repair, osteomalacia, arthritis, or thrombocytopenia).
Type:
Application
Filed:
March 15, 2019
Publication date:
December 31, 2020
Inventors:
Thomas J. GARDELLA, Ashok KHATRI, Hiroshi NODA
Abstract: A polyacrylonitrile (PANi) based pharmaceutical composition providing a porous implant for use in treating spinal cord trauma and/or spinal cord injury. Particularly a pharmaceutical composition including polyacrylonitrile (PANi) and/or elastin (E) and/or collagen (C) to form a PANi-E and/or PANi-C and/or a PANi-EC polymer network. Particularly, a pharmaceutical composition including polyacrylonitrile (PANi), elastin (E), and collagen (C) together forming a polyacrylonitrile (PANi), elastin (E), collagen (C) polymer network (PANi-E-C), wherein the polyacrylonitrile (PANi) may be crosslinked to form a crosslinked interpenetrating polyacrylonitrile (PANi), elastin (E) and collagen (C) polymer network (xpi-PANi-E-C), and wherein secondary protein structures of elastin (E) and collagen (C) reorientate. The disclosure extends to use of the pharmaceutical composition in the treatment of spinal cord trauma and/or spinal cord injury.
Type:
Application
Filed:
December 19, 2018
Publication date:
December 31, 2020
Applicant:
UNIVERSITY OF THE WITWATERSRAND, JOHANNESBURG
Abstract: The present invention relates to a pharmaceutical composition/formulation for use in treatment of Iron Deficiency Anaemia (IDA), Anaemia of Inflammation (AOI) and neuro-degenerative disorders. More particularly, the invention relates to a pharmaceutical composition/formulation comprising a synergistic combination of Lactoferrin and Guanosine Nucleotides or a pharmaceutically acceptable salt thereof. The application also provides various formulations and methods of preparing the same.
Abstract: Alcohol intoxication causes serious diseases, whereas current treatments are mostly supportive and unable to remove alcohol efficiently. Upon alcohol consumption, alcohol is sequentially oxidized to acetaldehyde and acetate by the endogenous alcohol dehydrogenase and aldehyde dehydrogenase, respectively. We disclose a hepatocyte-mimicking antidote for alcohol intoxication through the co-delivery of the nanocapsules of alcohol oxidase (AOx), catalase (CAT), and aldehyde dehydrogenase (ALDH) to the liver, where AOx and CAT catalyze the oxidation of alcohol to acetaldehyde, while ALDH catalyzes the oxidation of acetaldehyde to acetate. Administered to alcohol-intoxicated mice, the antidote rapidly accumulates in the liver and enables a significant reduction of the blood alcohol concentration. Moreover, blood acetaldehyde concentration is maintained at an extremely low level, significantly contributing to liver protection.
Type:
Application
Filed:
March 29, 2019
Publication date:
December 31, 2020
Applicant:
The Regents of the University of California
Abstract: The present disclosure relates generally to methods of treating a subject having muscular dystrophy or DMD. The present disclosure also relates generally to methods of prophylactically treating a subject at risk of developing muscular dystrophy or DMD. In some embodiments, the methods may include administering a pharmaceutical composition including an RRM1 gene, an RRM2 gene, and a delivery vehicle to a subject. In another embodiment, the methods may include administering a pharmaceutical composition including an RRM1 gene and an RRM2 gene coupled to a regulatory cassette to a subject. In yet another embodiment, the methods may include administering a pharmaceutical composition including an RRM 1 gene, an RRM2 gene, a regulatory cassette, and a delivery vehicle to a subject.
Type:
Application
Filed:
June 26, 2020
Publication date:
December 31, 2020
Applicant:
UNIVERSITY OF WASHINGTON
Inventors:
Guy L. ODOM, Michael REGNIER, Jeffrey S. CHAMBERLAIN, Stephen D. HAUSCHKA
Abstract: The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising an arylsulfatase A (ASA) protein, salt, and a polysorbate surfactant for the treatment of Metachromatic Leukodystrophy Disease.
Type:
Application
Filed:
March 24, 2020
Publication date:
December 31, 2020
Inventors:
Nazila Salamat-Miller, Katherine Taylor, Paul Campolieto, Zahra Shahrokh, Jing Pan, Lawrence Charnas, Teresa Leah Wright, Pericles Calias
Abstract: The present invention provides engineered human alpha-galactosidase polypeptides and compositions thereof. The engineered human alpha-galactosidase polypeptides have been optimized to provide improved stability under both acidic (pH <4.5) and basic (pH >7) conditions. The invention also relates to the use of the compositions comprising the engineered human alpha-galactosidase polypeptides for therapeutic purposes.
Type:
Application
Filed:
August 5, 2020
Publication date:
December 31, 2020
Inventors:
Nicholas J. Agard, Mathew G. Miller, Xiyun Zhang, Gjalt W. Huisman