Patents Assigned to Children's Hospital of Philadelphia
  • Methods for use in combination for the treatment and diagnosis of autoimmune diseases
    Patent number: 10385398
    Abstract: This disclosure provides new genetic targets, diagnostic methods, and therapeutic treatment regimens for multiple autoimmune disorders, including pediatric autoimmune disorders that are co-inherited and genetically shared. The disclosure, for example, provides methods of diagnosing or determining a susceptibility for one or more autoimmune diseases and methods of determining treatment protocols for patients with one or more autoimmune diseases based on determining if the patients have genetic alterations in particular genes.
    Type: Grant
    Filed: August 19, 2016
    Date of Patent: August 20, 2019
    Assignee: The Children's Hospital of Philadelphia
    Inventors: Hakon Hakonarson, Yun Rose Li, Brendan Keating
  • CXCR4 as a susceptibility locus in juvenile idiopathic arthritis (JIA) and methods of use thereof for the treatment and diagnosis of the same
    Patent number: 10344331
    Abstract: Compositions and methods useful for the diagnosis and treatment of juvenile idiopathic arthritis are disclosed.
    Type: Grant
    Filed: August 10, 2016
    Date of Patent: July 9, 2019
    Assignee: The Children's Hospital of Philadelphia
    Inventors: Terri H. Finkel, Haitao Zhang, Hakon Hakonarson
  • Magnetoencephalography biomarkers of GABA-B agonist drug activity in autism spectrum disorders
    Patent number: 10342480
    Abstract: Methods for screening for therapies against autism spectrum disorders and methods for determining whether a subject would be responsive to a therapy against an autism spectrum disorder are disclosed.
    Type: Grant
    Filed: March 17, 2014
    Date of Patent: July 9, 2019
    Assignee: The Children's Hospital of Philadelphia
    Inventors: Timothy P. L. Roberts, James Christopher Edgar
  • Azolylacryloyl derivatives as therapeutic agents for sickle cell disease
    Patent number: 10344001
    Abstract: Azolylacryloyl derivatives with hypoxic properties are provided. The compounds have a generic formula: and are suitable for treating sickle cell disease and hypoxia-underlying diseases, e.g. hemorrhagic and traumatic shock, cardiac arrest and cardiogenic shock, traumatic brain injury, cancer, stroke, myocardial infarction, myocardial ischemia, vaso-occlusive crisis, etc.
    Type: Grant
    Filed: September 30, 2016
    Date of Patent: July 9, 2019
    Assignees: VIRGINIA COMMONWEALTH UNIVERSITY, KING ABDULAZIZ UNIVERSITY, THE CHILDREN'S HOSPITAL OF PHILADELPHIA
    Inventors: Martin K. Safo, Yan Zhang, Mohini Ghatge, Osheiza Abdulmalik, Abdelsattar Mansour Ebid Omar, Moustafa El-Sayed El-Araby
  • METHODS AND VECTORS FOR TREATING CNS DISORDERS
    Publication number: 20190192693
    Abstract: Methods and uses of treating a disease in a mammal are provided by administering to a mammalian non-central nervous system (CNS) cell, organ or tissue, for delivery to mammalian CNS (e.g., brain). Methods and uses of treating a disease in a mammal include, inter alia, administering to a mammalian non-ocular cell, organ or tissue for delivery to a mammalian ocular cell, organ or tissue.
    Type: Application
    Filed: September 1, 2017
    Publication date: June 27, 2019
    Applicants: Spark Therapeutics, Inc., The Children's Hospital of Philadelphia
    Inventors: Katherine A. HIGH, Beverly L. DAVIDSON
  • Controlled gene expression methods
    Patent number: 10329333
    Abstract: Methods and compositions for controlling gene expression are disclosed.
    Type: Grant
    Filed: October 17, 2017
    Date of Patent: June 25, 2019
    Assignee: The Children's Hospital of Philadelphia
    Inventors: Gerd Blobel, Wulan Deng
  • Scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy
    Patent number: 10328145
    Abstract: Methods for preparing highly purified AAV vector formulations are provided. The highly pure AAV formulations described herein are superior for clinical use.
    Type: Grant
    Filed: March 25, 2016
    Date of Patent: June 25, 2019
    Assignee: The Children's Hospital of Philadelphia
    Inventors: John Fraser Wright, Guang Qu, Bernd Hauck, Katherine A. High
  • SYSTEMS AND METHODS FOR INTRAVENOUS CATHETER STABILIZATION AND MONITORING
    Publication number: 20190175877
    Abstract: Devices and methods for securing a catheter and monitoring a catheter insertion site. Exemplary embodiments can allow monitoring of the site without removal of the device. Exemplary embodiments can also secure a catheter while maintaining patient comfort.
    Type: Application
    Filed: May 15, 2017
    Publication date: June 13, 2019
    Applicant: THE CHILDREN'S HOSPITAL OF PHILADELPHIA
    Inventors: Jacqueline ANZALONE, Michele RUSSEN DAVEY, Cheryl GEBELINE-MYERS
  • FACTOR VIII VARIANTS, NUCLEIC ACID SEQUENCES, AND METHODS AND USES FOR TREATMENT OF HEMOSTASIS DISORDERS
    Publication number: 20190144524
    Abstract: Factor VIII variants and methods of use thereof are disclosed. In particular embodiments, Factor VIII variants exhibit one or more improvements compared to wild-type Factor VIII proteins, including wild-type Factor VIII proteins with a B-domain deletion (FVIII-BDD). Examples may include enhanced activity or function, secretion at increased levels by cells or are packaged more efficiently into viral vectors.
    Type: Application
    Filed: January 13, 2017
    Publication date: May 16, 2019
    Applicant: The Children's Hospital of Philadelphia
    Inventors: Denise E. SABATINO, Giang NGUYEN
  • Genetic alterations on chromosome 16 and methods of use thereof for the diagnosis and treatment of type 1 diabetes
    Patent number: 10266896
    Abstract: Compositions and methods for the detection and treatment of T1D are provided.
    Type: Grant
    Filed: September 14, 2016
    Date of Patent: April 23, 2019
    Assignee: The Children's Hospital of Philadelphia
    Inventors: Hakon Hakonarson, Struan F. A. Grant, Jonathan P. Bradfield, Constantin Polychronakos
  • LARGE COMMERCIAL SCALE LENTIVIRAL VECTOR PRODUCTION SYSTEM AND VECTORS PRODUCED THEREBY
    Publication number: 20190093126
    Abstract: In accordance with the present invention, a method for increasing the yield of rLV vector particles comprising a trans gene encoding a therapeutic protein or fragment thereof is disclosed. In one approach, cells are transfected with plasmids encoding the necessary components for rLV production using a calcium chloride transfection mix at pH 7.1 wherein the calcium chloride and plasmids form a complex which is added to the cells at a constant speed. The cells are then incubated for a suitable time period wherein virus particle media is collected at least twice during the incubation period and stored in a cold storage unit, thereby reducing virus inactivation.
    Type: Application
    Filed: August 27, 2018
    Publication date: March 28, 2019
    Applicant: The Children's Hospital of Philadelphia
    Inventors: Katherine A. High, John Fraser Wright, Bernd Hauck, Guang Qu
  • Compositions and methods for modulating thrombin generation
    Patent number: 10239933
    Abstract: Factor V peptides and methods of use thereof are disclosed.
    Type: Grant
    Filed: April 18, 2016
    Date of Patent: March 26, 2019
    Assignee: The Children's Hospital of Philadelphia
    Inventors: Rodney M. Camire, Matthew W. Bunce, Mettine H. A. Bos
  • COMPOSITIONS AND METHODS FOR DETECTION AND MODULATION OF T CELL MEDIATED IMMUNE RESPONSES AGAINST VIRAL VECTORS UTILIZED FOR GENE THERAPY
    Publication number: 20190083658
    Abstract: Compositions and methods are provided for inhibiting T cell mediated destruction of virally transduced, trangene containing cells.
    Type: Application
    Filed: September 20, 2018
    Publication date: March 21, 2019
    Applicant: The Children's Hospital of Philadelphia
    Inventors: Katherine A. High, Marcela V. Maus, Federico Mingozzi, Daniel J. Hui
  • THERAPEUTIC FOR TREATMENT OF DISEASES INCLUDING THE CENTRAL NERVOUS SYSTEM
    Publication number: 20190071671
    Abstract: The present disclosure provides filler or stuffer sequences, compositions thereof including expression cassettes and vectors, such as viral (e.g., AAV) vectors and methods of delivering a therapeutic agent to a mammal and/or treating a disease.
    Type: Application
    Filed: March 17, 2017
    Publication date: March 7, 2019
    Applicant: The Children's Hospital of Philadelphia
    Inventors: Beverly L. DAVIDSON, Alejandro Mas MONTEYS, Megan S. KEISER
  • METHODS OF IMPROVING MYOCARDIAL PERFORMANCE IN FONTAN PATIENTS USING UDENAFIL COMPOSITIONS
    Publication number: 20190030038
    Abstract: The present invention relates generally to the field of using udenafil or a pharmaceutically acceptable salt thereof in patients who have undergone the Fontan operation.
    Type: Application
    Filed: September 28, 2018
    Publication date: January 31, 2019
    Applicants: Mezzion Pharma Co., Ltd., The Children's Hospital of Philadelphia, The Children's Hospital of Philadelphia
    Inventors: James L. Yeager, David J. Goldberg, Stephen M. Paridon
  • Methods and compositions for treating hypoglycemic disorders
    Patent number: 10188702
    Abstract: This invention relates to methods of treating and ameliorating congenital and neonatal hyperinsulinism and post-prandial hypoglycemia, comprising the step of administering an antagonist of the Glucagon-Like Peptide-1 (GLP-1) receptor, e.g. a GLP-1 fragment or analog thereof.
    Type: Grant
    Filed: March 8, 2017
    Date of Patent: January 29, 2019
    Assignees: THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA, CHILDREN'S HOSPITAL OF PHILADELPHIA
    Inventors: Doris Stoffers, Diva D. De Leon, Charles Stanley
  • Mutations in and as causes of autosomal dominant infantile myofibromatosis
    Patent number: 10155995
    Abstract: This invention relates to a method of diagnosing a subject as having and/or being a carrier for infantile myofibromatosis. This method involves providing an isolated biological sample from a subject; contacting the sample with one or more reagents suitable for detecting the presence or absence of one or more mutations in PDGFRB and/or NOTCH3; detecting, in the sample, the presence or absence of the one or more mutations in PDGFRB and/or NOTCH3 based on said contacting; and diagnosing the subject as having and/or being a carrier for infantile myofibromatosis based on said detecting, where the presence of the one or more mutations in PDGFRB and/or NOTCH3 indicates the subject has a mutation that causes infantile myofibromatosis. Also disclosed is a method of treating a subject having infantile myofibromatosis and a method of preventing or treating symptoms associated with infantile myofibromatosis.
    Type: Grant
    Filed: October 20, 2017
    Date of Patent: December 18, 2018
    Assignees: ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI, THE CHILDREN'S HOSPITAL OF PHILADELPHIA
    Inventors: John A. Martignetti, Hakon Hakonarson, Lifeng Tian
  • Methods of improving myocardial performance in fontan patients using udenafil compositions
    Patent number: 10137128
    Abstract: The present invention relates generally to the field of using udenafil or a pharmaceutically acceptable salt thereof in patients who have undergone the Fontan operation.
    Type: Grant
    Filed: June 30, 2015
    Date of Patent: November 27, 2018
    Assignees: Mezzion Pharma Co., Ltd., The Children's Hospital of Philadelphia
    Inventors: James L. Yeager, David J. Goldberg, Stephen M. Paridon
  • Compositions and methods for the treatment of fatty acid metabolism disorders
    Patent number: 10137103
    Abstract: Compositions and methods for inhibiting, treating, and/or preventing fatty acid metabolism disorders, particularly fatty acid oxidation disorders, in a subject are provided.
    Type: Grant
    Filed: May 9, 2017
    Date of Patent: November 27, 2018
    Assignee: The Children's Hospital of Philadelphia
    Inventors: Harry Ischiropoulos, Paschalis-Thomas Doulias
  • Compositions and methods for treating autoimmune and inflammatory diseases
    Patent number: 10130650
    Abstract: Compositions and methods are provided for the treatment and/or prevention of an inflammatory and/or autoimmune disease or disorder.
    Type: Grant
    Filed: January 27, 2015
    Date of Patent: November 20, 2018
    Assignees: The Children's Hospital of Philadelphia, The Trustees of The University of Pennsylvania
    Inventors: Yanping Huang, Janis K. Burkhardt, Taku Kambayashi