Patents by Inventor Emil D. Kakkis

Emil D. Kakkis has filed for patents to protect the following inventions. This listing includes patent applications that are pending as well as patents that have already been granted by the United States Patent and Trademark Office (USPTO).

  • Publication number: 20210228694
    Abstract: Methods and compositions for the treatment or prevention of amyloidosis are provided. In some embodiments, the methods comprise administering to the subject a therapeutically effective amount of at least one catabolic enzyme or a biologically active fragment thereof. Such methods and compositions may be employed to reduce, prevent, degrade and/or eliminate amyloid formation in the lysosome and/or extracellularly.
    Type: Application
    Filed: October 8, 2020
    Publication date: July 29, 2021
    Inventors: Emil D. Kakkis, Michel Claude Vellard, Andrzej Swistowski
  • Patent number: 11040088
    Abstract: The present invention relates to the intrathecal (IT) administration of recombinant enzyme to treat lysosomal storage disorders. In an exemplary embodiment, intrathecal administration of human ?-L-iduronidase (rhIDU) injections in MPS I affected animals resulted in significant enzyme uptake, significant rh-iduronidase activity in brain and meninges and a decrease of glycosaminoglycan (GAG) storage in cells of MPS I subjects to that of normal subjects. Intrathecal administration proved more effective than intravenous treatment at alleviating MPS I symptoms, indicating it is a useful method of treating lysosomal storage disorders.
    Type: Grant
    Filed: January 6, 2017
    Date of Patent: June 22, 2021
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventor: Emil D. Kakkis
  • Publication number: 20210002211
    Abstract: The present disclosure provides creatine prodrug analogs and their compositions useful for the treatment of creatine deficiencies.
    Type: Application
    Filed: December 3, 2018
    Publication date: January 7, 2021
    Inventors: Yiumo CHAN, Emil D. KAKKIS, Alexandra TROTIER-FAURION, William F. BRUBAKER, Arjun NATESAN, Paul LEE, Sharyl FYFFE-MARICICH, David LAPOINTE, Mike E. LIZARZABURU
  • Publication number: 20200330575
    Abstract: The present invention provides compositions and methods for treating a hypophosphatemic disorder, such as X-linked hypophosphatemia (XLH). The method entails administering to a subject a pharmaceutical composition containing an anti-FGF23 ligand, wherein the dosing regimen of the pharmaceutical is designed to reach effective and efficient control of FGF23 activity.
    Type: Application
    Filed: March 26, 2020
    Publication date: October 22, 2020
    Inventors: Emil D. KAKKIS, Javier San Martin, Tomohiro Sudo
  • Publication number: 20200282028
    Abstract: The present application relates to sialylated glycoprotein compositions and methods of their use in treating various conditions and disorders.
    Type: Application
    Filed: January 9, 2020
    Publication date: September 10, 2020
    Inventors: Steven JUNGLES, Gabrielle MORRIS, Jeff GRUBB, Michael VELLARD, Emil D. KAKKIS
  • Patent number: 10639360
    Abstract: The present invention provides compositions and methods for treating a hypophosphatemic disorder, such as X-linked hypophosphatemia (XLH). The method entails administering to a subject a pharmaceutical composition containing an anti-FGF23 ligand, wherein the dosing regimen of the pharmaceutical is designed to reach effective and efficient control of FGF23 activity.
    Type: Grant
    Filed: May 29, 2015
    Date of Patent: May 5, 2020
    Assignees: Ultragenyx Pharmaceutical, Inc., Kyowa Kirin Co., Ltd.
    Inventors: Emil D. Kakkis, Javier San Martin, Tomohiro Sudo
  • Publication number: 20190345475
    Abstract: Provided herein are phenylalanine ammonia-lyase (PAL) variants produced by prokaryotes, wherein such prokaryotic PAL variant has a greater phenylalanine-converting activity and/or a reduced immunogenicity as compared to a wild-type PAL. Further provided are compositions of prokaryotic PAL and biologically active fragments, mutants, variants or analogs thereof, as well as methods for the production, purification, formulation, and use of such compositions for industrial and therapeutic purposes, e.g., treating hyperphenylalaninemia, including phenylketonuria, and other disorders, including cancer.
    Type: Application
    Filed: January 11, 2019
    Publication date: November 14, 2019
    Applicant: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Augustus O. Okhamafe, Sean M. Bell, G. Nick Zecherle, Kris Antonsen, Yanhong Zhang, Kieu Ly Tran, Paul A. Fitzpatrick, Emil D. Kakkis, Michel Claude Vellard, Daniel J. Wendt, Mubarack Muthalif
  • Publication number: 20190183985
    Abstract: Methods and compositions for the treatment or prevention of amyloidosis are provided. In some embodiments, the methods comprise administering to the subject a therapeutically effective amount of at least one catabolic enzyme or a biologically active fragment thereof. Such methods and compositions may be employed to reduce, prevent, degrade and/or eliminate amyloid formation in the lysosome and/or extracellularly.
    Type: Application
    Filed: December 19, 2018
    Publication date: June 20, 2019
    Inventors: Emil D. KAKKIS, Michel Claude VELLARD, Andrzej SWISTOWSKI
  • Patent number: 10221408
    Abstract: Provided herein are phenylalanine ammonia-lyase (PAL) variants produced by prokaryotes, wherein such prokaryotic PAL variant has a greater phenylalanine-converting activity and/or a reduced immunogenicity as compared to a wild-type PAL. Further provided are compositions of prokaryotic PAL and biologically active fragments, mutants, variants or analogs thereof, as well as methods for the production, purification, formulation, and use of such compositions for industrial and therapeutic purposes, e.g., treating hyperphenylalaninemia, including phenylketonuria, and other disorders, including cancer.
    Type: Grant
    Filed: April 15, 2016
    Date of Patent: March 5, 2019
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Augustus O. Okhamafe, Sean M. Bell, G. Nick Zecherle, Kris Antonsen, Yanhong Zhang, Kieu Ly Tran, Paul A. Fitzpatrick, Emil D. Kakkis, Michel Claude Vellard, Daniel J. Wendt, Mubarack Muthalif
  • Publication number: 20190054153
    Abstract: The present application relates to sialylated glycoprotein compositions and methods of their use in treating various conditions and disorders.
    Type: Application
    Filed: April 2, 2018
    Publication date: February 21, 2019
    Inventors: Steven JUNGLES, Gabrielle MORRIS, Jeff GRUBB, Michael VELLARD, Emil D. KAKKIS
  • Publication number: 20180256582
    Abstract: The present invention is directed to a novel methods and compositions for the therapeutic intervention in hyperphenylalaninemia. More specifically, the specification describes methods and compositions for treating various types of phenylketonurias using compositions comprising BH4. Combination therapies of BH4 and other therapeutic regimens are contemplated.
    Type: Application
    Filed: May 8, 2018
    Publication date: September 13, 2018
    Inventors: Daniel I. Oppenheimer, Emil D. Kakkis, Fredric D. Price, Alejandro Dorenbaum, Rudolf Moser, Viola Groehn, Thomas Egger, Fritz Blatter
  • Patent number: 9993481
    Abstract: The present invention is directed to a novel methods and compositions for the therapeutic intervention in hyperphenylalaninemia. More specifically, the specification describes methods and compositions for treating various types of phenylketonurias using compositions comprising BH4. Combination therapies of BH4 and other therapeutic regimens are contemplated.
    Type: Grant
    Filed: September 2, 2016
    Date of Patent: June 12, 2018
    Assignee: BIOMARIN PHARMACEUTICAL INC.
    Inventors: Daniel I. Oppenheimer, Emil D. Kakkis, Fredric D. Price, Alejandro Dorenbaum, Rudolf Moser, Viola Groehn, Thomas Egger, Fritz Blatter
  • Patent number: 9937243
    Abstract: The present application relates to sialylated glycoprotein compositions and methods of their use in treating various conditions and disorders.
    Type: Grant
    Filed: May 26, 2017
    Date of Patent: April 10, 2018
    Assignee: Ultrageny x Pharmaceutical Inc.
    Inventors: Steven Jungles, Gabrielle Morris, Jeff Grubb, Michael Vellard, Emil D. Kakkis
  • Publication number: 20170326210
    Abstract: The present application relates to sialylated glycoprotein compositions and methods of their use in treating various conditions and disorders.
    Type: Application
    Filed: May 26, 2017
    Publication date: November 16, 2017
    Inventors: Steven JUNGLES, Gabrielle MORRIS, Jeff GRUBB, Michael VELLARD, Emil D. KAKKIS
  • Publication number: 20170182134
    Abstract: The present invention relates to the intrathecal (IT) administration of recombinant enzyme to treat lysosomal storage disorders. In an exemplary embodiment, intrathecal administration of human ?-L-iduronidase (rhIDU) injections in MPS I affected animals resulted in significant enzyme uptake, significant rh-iduronidase activity in brain and meninges and a decrease of glycosaminoglycan (GAG) storage in cells of MPS I subjects to that of normal subjects. Intrathecal administration proved more effective than intravenous treatment at alleviating MPS I symptoms, indicating it is a useful method of treating lysosomal storage disorders.
    Type: Application
    Filed: January 6, 2017
    Publication date: June 29, 2017
    Inventor: Emil D. Kakkis
  • Patent number: 9687532
    Abstract: The present application relates to sialylated glycoprotein compositions and methods of their use in treating various conditions and disorders.
    Type: Grant
    Filed: August 30, 2016
    Date of Patent: June 27, 2017
    Assignee: Ultragenyx Pharmaceutical Inc.
    Inventors: Steven Jungles, Gabrielle Morris, Jeff Grubb, Michael Vellard, Emil D. Kakkis
  • Publication number: 20170119861
    Abstract: Methods and compositions for the treatment or prevention of amyloidosis are provided. In some embodiments, the methods comprise administering to the subject a therapeutically effective amount of at least one catabolic enzyme or a biologically active fragment thereof. Such methods and compositions may be employed to reduce, prevent, degrade and/or eliminate amyloid formation in the lysosome and/or extracellularly.
    Type: Application
    Filed: October 28, 2016
    Publication date: May 4, 2017
    Inventors: Emil D. KAKKIS, Michel Claude VELLARD, Andrzej SWISTOWSKI
  • Patent number: 9572870
    Abstract: The present invention relates to the intrathecal (IT) administration of recombinant enzyme to treat lysosomal storage disorders. In an exemplary embodiment, intrathecal administration of human ?-L-iduronidase (rhIDU) injections in MPS I affected animals resulted in significant enzyme uptake, significant rh-iduronidase activity in brain and meninges and a decrease of glycosaminoglycan (GAG) storage in cells of MPS I subjects to that of normal subjects. Intrathecal administration proved more effective than intravenous treatment at alleviating MPS I symptoms, indicating it is a useful method of treating lysosomal storage disorders.
    Type: Grant
    Filed: February 5, 2015
    Date of Patent: February 21, 2017
    Assignee: BioMarin Pharmaceutical Inc.
    Inventor: Emil D. Kakkis
  • Publication number: 20160367646
    Abstract: The present application relates to sialylated glycoprotein compositions and methods of their use in treating various conditions and disorders.
    Type: Application
    Filed: August 30, 2016
    Publication date: December 22, 2016
    Inventors: Steven JUNGLES, Gabrielle MORRIS, Jeff GRUBB, Michael VELLARD, Emil D. KAKKIS
  • Publication number: 20160367558
    Abstract: The present invention is directed to a novel methods and compositions for the therapeutic intervention in hyperphenylalaninemia. More specifically, the specification describes methods and compositions for treating various types of phenylketonurias using compositions comprising BH4. Combination therapies of BH4 and other therapeutic regimens are contemplated.
    Type: Application
    Filed: September 2, 2016
    Publication date: December 22, 2016
    Inventors: Daniel I. Oppenheimer, Emil D. Kakkis, Fredric D. Price, Alejandro Dorenbaum, Rudolf Moser, Viola Groehn, Thomas Egger, Fritz Blatter