Patents Assigned to Shire Human Genetic Therapies, Inc.
  • Publication number: 20210283016
    Abstract: The present disclosure is directed to a vial adapter for interconnecting a vial and a fluid delivery device, and, more particularly, to a vial adapter having a modular design consisting of separately constructed components cooperatively arranged and coupled to one another. The modular construction allows for rapid manufacturing reconfigurations of one or more components with minimal costs to create new vial adapter configurations that meet specific needs.
    Type: Application
    Filed: June 3, 2021
    Publication date: September 16, 2021
    Applicant: Shire Human Genetic Therapies, Inc.
    Inventors: Scott Richard ARIAGNO, Daniel Edward ROUSH
  • Publication number: 20210275751
    Abstract: Plunger rod stops attach to existing syringes to limit the range of motion of the plunger, thereby to precisely control the amount of liquid that can be expelled from the syringe when the plunger rod stop is in place. In some embodiments, plunger rod stop devices generally include a body configured to attach to the plunger, and a stand-off extending from the body, configured to contact the finger flange when the plunger is depressed. In other embodiments, the plunger rod stop attaches to the finger flange or other fixed component of the syringe and the stand-off is configured to contact the plunger or plunger rod. Methods of the invention involve using the devices to interfere with the range of motion of the plunger, then reconfiguring or removing the device to allow the plunger to be moved a predetermined length, which corresponds to a precise dosage amount.
    Type: Application
    Filed: May 13, 2021
    Publication date: September 9, 2021
    Applicant: Shire Human Genetic Therapies, Inc.
    Inventors: Steve BOWMAN, Chase FETZER, Richard BRAGA, Evi SHIAKOLAS
  • Patent number: 11065307
    Abstract: Among other things, the present invention provides methods and compositions of treating Sanfilippo syndrome type B (Sanfilippo B) by, e.g., intrathecal (IT) administration of a Naglu protein. A suitable Naglu protein can be a recombinant, gene-activated or natural protein. In some embodiments, a suitable Naglu protein is a recombinant Naglu protein. In some embodiments, a recombinant Naglu protein is a fusion protein containing a Naglu domain and a lysosomal targeting moiety. In some embodiments, the lysosomal targeting domain is an IGF-II moiety.
    Type: Grant
    Filed: September 26, 2017
    Date of Patent: July 20, 2021
    Assignee: Shire Human Genetic Therapies, Inc.
    Inventors: Michael F. Concino, Pericles Calias, Jing Pan, Kevin Holmes, Paolo Martini, Alla Romashko, Muthuraman Meiyappan, Bohong Zhang, Andrea Iskenderian, Dianna Lundberg, Angela Norton, Bettina Strack-Logue, Huang Yan, Mary Alessandrini, Richard Pfeifer
  • Patent number: 11065308
    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 a heparan N-sulfatase (HNS) protein, salt, and a polysorbate surfactant for the treatment of Sanfilippo Syndrome Type A.
    Type: Grant
    Filed: July 16, 2018
    Date of Patent: July 20, 2021
    Assignee: Shire Human Genetic Therapies, Inc.
    Inventors: Farah Natoli, Gaozhong Zhu, Jennifer Terew, Yuan Jiang, Jamie Tsung, Zahra Shahrokh, Brian Vernaglia, Jing Pan, Richard Pfeifer, Pericles Calias
  • Patent number: 11033459
    Abstract: The present disclosure is directed to a vial adapter for interconnecting a vial and a fluid delivery device, and, more particularly, to a vial adapter having a modular design consisting of separately constructed components cooperatively arranged and coupled to one another. The modular construction allows for rapid manufacturing reconfigurations of one or more components with minimal costs to create new vial adapter configurations that meet specific needs.
    Type: Grant
    Filed: December 13, 2017
    Date of Patent: June 15, 2021
    Assignee: Shire Human Genetic Therapies, Inc.
    Inventors: Scott Richard Ariagno, Daniel Edward Roush
  • Patent number: 11013864
    Abstract: Plunger rod stops attach to existing syringes to limit the range of motion of the plunger, thereby to precisely control the amount of liquid that can be expelled from the syringe when the plunger rod stop is in place. In some embodiments, plunger rod stop devices generally include a body configured to attach to the plunger, and a stand-off extending from the body, configured to contact the finger flange when the plunger is depressed. In other embodiments, the plunger rod stop attaches to the finger flange or other fixed component of the syringe and the stand-off is configured to contact the plunger or plunger rod. Methods of the invention involve using the devices to interfere with the range of motion of the plunger, then reconfiguring or removing the device to allow the plunger to be moved a predetermined length, which corresponds to a precise dosage amount.
    Type: Grant
    Filed: November 6, 2017
    Date of Patent: May 25, 2021
    Assignee: Shire Human Genetic Therapies, Inc.
    Inventors: Steve Bowman, Chase Fetzer, Richard Braga, Evi Shiakolas
  • Patent number: 10967073
    Abstract: The present invention is directed, in part, to the treatment of a subject having a neurodegenerative disorder, such as Parkinson's disease (PD), by providing glucocerebrosidase enzyme. The enzyme may be provided, e.g., through gene therapy or by administration of a glucocerebrosidase protein. Accordingly, the present invention encompasses glucocerebrosidase nucleic acids or proteins for use in the treatment of PD or other neurodegenerative disorders.
    Type: Grant
    Filed: May 6, 2016
    Date of Patent: April 6, 2021
    Assignees: The McLean Hospital Corporation, Shire Human Genetic Therapies, Inc.
    Inventors: Yung Hee Park, Ole Isacson
  • Publication number: 20210098103
    Abstract: A drug monitoring tool comprises a data receiver and an interactive user interface. The data receiver is configured to receive a pharmacokinetic (PK) profile of a patient. The interactive user interface is configured to display, to the patient, a time-varying therapeutic plasma protein level of the patient and delineate a plurality of zones within the interactive user interface associated with the time-varying therapeutic plasma protein level. The plurality of zones includes a safe zone indicating to the patient that the time-varying therapeutic plasma protein level is within a first concentration range considered safe for physical activity and a danger zone indicating to the patient that the time-varying therapeutic plasma protein level is within a second concentration range and physical activity should be limited.
    Type: Application
    Filed: December 11, 2020
    Publication date: April 1, 2021
    Applicant: Shire Human Genetic Therapies, Inc.
    Inventors: Gerald SPOTTS, Roman PICHLER, Michael NELSON
  • Publication number: 20210040465
    Abstract: The invention relates to an affinity resin functionalized with small molecule inhibitors of glycoside-cleaving enzymes, e.g., ?-galactosidase A (?-Gal A), glucocerebrosidase (GCB), ?-galactosidase, and acid alpha-glucosidase (GAA), and a method for purifying glycoside-cleaving enzymes produced in a cell line using the small molecule inhibitor-functionalized affinity resin.
    Type: Application
    Filed: March 5, 2019
    Publication date: February 11, 2021
    Applicant: SHIRE HUMAN GENETIC THERAPIES, INC.
    Inventors: Brian Dwyer, Bohong Zhang, Jun Hu, Muthuraman Meiyappan, Thomas Miller, Paul McLean, Clark Pan
  • Patent number: 10896749
    Abstract: Embodiments of the present disclosure relate to a drug monitoring tool. The drug monitoring tool comprises a data receiver and an interactive user interface. The data receiver is configured to receive a pharmacokinetic (PK) profile of a patient. The interactive user interface is configured to display, to the patient, a time-varying therapeutic plasma protein level of the patient. The time-varying therapeutic plasma protein level is based on an administered dose of a clotting factor VIII and the PK profile of the patient.
    Type: Grant
    Filed: January 22, 2018
    Date of Patent: January 19, 2021
    Assignee: SHIRE HUMAN GENETIC THERAPIES, INC.
    Inventors: Gerald Spotts, Roman Pichler, Michael Nelson
  • Publication number: 20200316178
    Abstract: The invention provides a composition of glucocerebrosidase, such as velaglucerase alfa, and isofagomine, in a molar ratio of at least about 1:2.5. Also provided is a use of the composition for treatment of a disorder related to a dysfunction in a GCase pathway. The disorder could be a lysosomal storage disease, such as Gaucher disease, Fabry disease, Pompe disease, a mucopolysaccharidoses, or multiple system atrophy. The disorder could also be a neurodegenerative disorder, such as Parkinson disease, Alzheimer's disease, or Lewy body dementia. The composition can have 0.5 to 5.0 mg/kg of glucocerebrosidase and isofagomine in at least about a 3-fold molar excess to the glucocerebrosidase. The composition can be administered intravenously or subcutaneously.
    Type: Application
    Filed: October 25, 2018
    Publication date: October 8, 2020
    Applicant: SHIRE HUMAN GENETIC THERAPIES, INC.
    Inventors: Yung Hee PARK, Nancy CHEN, Jun HU, Muthuraman MEIYAPPAN, Thomas Allen MILLER
  • Patent number: 10787515
    Abstract: The present invention provides, among other things, anti-Flt-1 antibodies and methods for treating muscular dystrophy, in particular, Duchenne muscular dystrophy (DMD). In some embodiments, a method according to the present invention includes administering to an individual who is suffering from or susceptible to DMD an effective amount of an anti-Flt-1 antibody or antigen-binding protein thereof such that at least one symptom or feature of DMD is reduced in intensity, severity, or frequency, or has delayed onset.
    Type: Grant
    Filed: April 7, 2016
    Date of Patent: September 29, 2020
    Assignee: Shire Human Genetic Therapies, Inc.
    Inventors: Dennis Keefe, Hans De Haard, Natalie De Jonge, Sofie Gabriels
  • Patent number: 10760112
    Abstract: The present invention provides, among other things, methods and compositions for determining enzyme kinetic parameters (e.g., Vmax, Km, and specific activity, etc.) indicative of clinically relevant properties of glucocerebrosidase using a physiologically relevant substrate, in particular, a substrate that is representative of substrates that typically accumulate in patients suffering from Gaucher disease such as glucosylceramide. Thus, the present invention is particularly useful to measure a kinetic parameter relating to the activity of glucocerebrosidase in a drug substance, drug product, and stability sample for enzyme replacement therapy.
    Type: Grant
    Filed: December 16, 2015
    Date of Patent: September 1, 2020
    Assignee: Shire Human Genetic Therapies, Inc.
    Inventors: Peter Bernhardt, Chen-Chung Willy Yen, Vijay Chhajlani
  • Patent number: 10738123
    Abstract: The present invention provides, among other things, methods and compositions for treating chronic lung disorders, in particular, bronchopulmonary dysplasia (BPD). In some embodiments, a method according to the present invention includes administering to an individual who is suffering from or susceptible to BPD an effective amount of an anti-Flt-1 antibody, or antigen binding fragment thereof, such that at least one symptom or feature of BPD is reduced in intensity, severity, or frequency, or has delayed onset.
    Type: Grant
    Filed: April 7, 2016
    Date of Patent: August 11, 2020
    Assignees: Shire Human Genetic Therapies, Inc., The Regents of the University of Colorado, A Body Corporate
    Inventors: Dennis Keefe, Steven Abman, Gregory Seedorf
  • Patent number: 10729746
    Abstract: The present invention provides, among other things, methods and compositions for treating muscular dystrophy, in particular, Duchenne muscular dystrophy (DMD). In some embodiments, a method according to the present invention includes administering to an individual who is suffering from or susceptible to DMD an effective amount of a recombinant PLGF protein such that at least one symptom or feature of DMD is reduced in intensity, severity, or frequency, or has delayed onset. The present invention also provides exemplary recombinant PLGF proteins including monomeric, dimeric and single-chain PLGF proteins.
    Type: Grant
    Filed: January 28, 2014
    Date of Patent: August 4, 2020
    Assignee: Shire Human Genetic Therapies, Inc.
    Inventors: Angela Norton, Michael F. Concino, Muthuraman Meiyappan, Andrea Iskenderian
  • Patent number: 10730874
    Abstract: The present invention provides compounds and compositions thereof which are useful as inhibitors of plasma kallikrein and which exhibit desirable characteristics for the same.
    Type: Grant
    Filed: March 12, 2019
    Date of Patent: August 4, 2020
    Assignee: Shire Human Genetic Therapies, Inc.
    Inventors: Nikolaos Papaioannou, Sarah Jocelyn Fink, Thomas Allen Miller, Gerald Wayne Shipps, Jr., Jeremy Mark Travins, David Edward Ehmann, Alastair Rae, John Mark Ellard
  • Patent number: 10723772
    Abstract: The present invention provides, among other things, methods and compositions for treating muscular dystrophy, in particular, Duchenne muscular dystrophy (DMD). In some embodiments, a method according to the present invention includes administering to an individual who is suffering from or susceptible to DMD an effective amount of a recombinant follistatin protein such that at least one symptom or feature of DMD is reduced in intensity, severity, or frequency, or has delayed onset.
    Type: Grant
    Filed: April 12, 2018
    Date of Patent: July 28, 2020
    Assignee: Shire Human Genetic Therapies, Inc.
    Inventor: Rochelle Mineau
  • Patent number: 10722559
    Abstract: A targeted therapeutic including a lysosomal enzyme and a lysosomal targeting moiety that is a peptide containing at least one N-linked glycosylation site. Methods of producing the targeted therapeutic may include nucleotide acids encoding the same and host cells co-expressing GNPT. Pharmaceutical compositions comprising the targeted therapeutic and methods of using the same to treat a lysosomal storage disease.
    Type: Grant
    Filed: August 11, 2015
    Date of Patent: July 28, 2020
    Assignee: Shire Human Genetic Therapies, Inc.
    Inventors: Michael F. Concino, Bettina Strack-Logue, Muthuraman Meiyappan, Angela W. Norton, Bohong Zhang, Andrea Iskenderian, Lieh Yoon Low, Dianna Lundberg, Alla Romashko, Hicham Naimy
  • Patent number: 10660944
    Abstract: The present invention provides, among oilier things, compositions and methods for CNS delivery of lysosomal enzymes (e.g., recombinant human arylsulfatase A (rhASA)) for effective treatment of lysosomal storage diseases (e.g. Metachromatic Leukodystrophy Disease), In some embodiments, the present invention includes a stable formulation for intrathecal administration comprising an ASA protein and a poloxamer, wherein less than 3% of the ASA protein exists in aggregated form.
    Type: Grant
    Filed: June 5, 2018
    Date of Patent: May 26, 2020
    Assignee: Shire Human Genetic Therapies, Inc.
    Inventors: Nazila Salamat-Miller, Katherine Taylor, Paul Campolieto, Zahra Shahrokh, Jing Pan, Lawrence Charnas, Teresa Leah Wright, Pericles Calias, Keethkumar Jain, Sujit Basu
  • Patent number: 10646554
    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: Grant
    Filed: August 23, 2017
    Date of Patent: May 12, 2020
    Assignee: Shire Human Genetic Therapies, Inc.
    Inventors: Nazila Salamat-Miller, Katherine Taylor, Paul Campolieto, Zahra Shahrokh, Jing Pan, Lawrence Charnas, Teresa Leah Wright, Pericles Calias