Patents by Inventor Staci Sabnis

Staci Sabnis 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: 20230323371
    Abstract: The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the ?-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.
    Type: Application
    Filed: April 6, 2023
    Publication date: October 12, 2023
    Inventors: Paolo MARTINI, Stephen G. HOGE, Kerry BENENATO, Vladimir PRESNYAK, Iain MCFADYEN, Ellalahewage Sathyajith KUMARASINGHE, Xuling ZHU, Lin Tung GUEY, Staci SABNIS
  • Publication number: 20230233475
    Abstract: This disclosure relates to ionizable lipid-based lipid nanoparticles for delivery of mRNA encoding glucose-6-phosphatase. Lipid nanoparticle/mRNA therapies of the invention increase and/or restore deficient levels of glucose-6-phosphatase expression and activity in subjects and are useful for the treatment of glycogen storage disease type 1a (GSD-Ia). Lipid nanoparticle/mRNA therapies of the invention increase glucose production and reduce the abnormal accumulation of glycogen and glucose-6-phosphate associated with GSD-Ia.
    Type: Application
    Filed: June 1, 2021
    Publication date: July 27, 2023
    Inventors: Kerry Benenato, Kristine Burke, Jingsong Cao, Paloma Hoban Giangrande, Edward J. Hennessy, Stephen Hoge, Jaclyn Milton, Staci Sabnis, Timothy Salerno, Matthew Theisen
  • Patent number: 11649461
    Abstract: The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the ?-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.
    Type: Grant
    Filed: September 13, 2019
    Date of Patent: May 16, 2023
    Assignee: ModernaTX, Inc.
    Inventors: Paolo Martini, Stephen G. Hoge, Kerry Benenato, Vladimir Presnyak, Iain Mcfadyen, Ellalahewage Sathyajith Kumarasinghe, Xuling Zhu, Lin Tung Guey, Staci Sabnis
  • Publication number: 20230112986
    Abstract: The invention relates to mRNA therapy for the treatment of Acute Intermittent Porphyria (AIP). mRNAs for use in the invention, when administered in vivo, encode human porphobilinogen deaminase (PBGD), isoforms thereof, functional fragments thereof, and fusion proteins comprising PBGD. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to affect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of PBGD expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient PBGD activity in subjects, namely porphobilinogen and aminolevulinate (PBG and ALA).
    Type: Application
    Filed: July 21, 2022
    Publication date: April 13, 2023
    Applicants: ModernaTX, Inc., Fundacion Para La Investigacion Medica Aplicada
    Inventors: Paolo MARTINI, Stephen HOGE, Kerry BENENATO, Vladimir PRESNVAK, Lei JIANG, Iain MCFADYEN, Ellalahewage Sathyajith KUMARASINGHE, Antonio FONTANELLAS ROMA, Pedro BERRAONDO LOPEZ, Matias Antonio AVILA ZARAGOZA, Lin Tung GUEY, Staci SABNIS
  • Publication number: 20230027864
    Abstract: The disclosure features immune cell delivery lipid nanoparticle (LNP) compositions that allow for enhanced delivery of agents, e.g., nucleic acids, such as therapeutic and/or prophylactic RNAs, to immune cells, in particular T cells, as well as B cells, dendritic cells and monocytes. The LNPs comprise an effective amount of an immune cell delivery potentiating lipid such that delivery of an agent by an immune cell delivery LNP is enhanced as compared to an LNP lacking the immune cell delivery potentiating agent. Methods of using the immune cell delivery LNPs for delivery of agents, e.g., nucleic acid delivery, for protein expression, for modulating immune cell activity and modulating immune responses are also disclosed.
    Type: Application
    Filed: May 18, 2022
    Publication date: January 26, 2023
    Inventors: Gilles BESIN, Luis BRITO, Stephen G. HOGE, Edward HENNESSY, Mark CORNEBISE, Kerry BENENATO, Staci SABNIS, Michael W. DANNEMAN
  • Publication number: 20220296517
    Abstract: The disclosure features target cell delivery lipid nanoparticle (LNP) compositions that allow for enhanced delivery of agents, e.g., nucleic acids, such as therapeutic and/or prophylactic RNAs, to target cells, in particular liver cells and/or splenic cells. The LNPs comprise an effective amount of a target cell delivery potentiating lipid such that delivery of an agent by a target cell target cell delivery LNP is enhanced as compared to an LNP lacking the target cell delivery potentiating agent. Methods of using the target cell target cell delivery LNPs for delivery of agents, e.g., nucleic acid delivery, for protein expression, and for modulating target cell activity are also disclosed.
    Type: Application
    Filed: August 6, 2020
    Publication date: September 22, 2022
    Inventors: Kerry Benenato, Staci Sabnis, Edward Hennessy, Kristine Burke, Matthew Theisen, Jaclyn Milton, Timothy Salerno, Stephen Hoge
  • Publication number: 20220265857
    Abstract: This disclosure provides improved lipid-based compositions, including lipid nanoparticle compositions, and methods of use thereof for delivering agents in vivo including nucleic acids and proteins. These compositions are not subject to accelerated blood clearance and they have an improved toxicity profile in vivo.
    Type: Application
    Filed: January 25, 2022
    Publication date: August 25, 2022
    Applicant: ModernaTX, Inc.
    Inventors: Gilles Besin, Stephen Hoge, Joseph Senn, Kerry Benenato, Staci Sabnis
  • Patent number: 11285222
    Abstract: This disclosure provides improved lipid-based compositions, including lipid nanoparticle compositions, and methods of use thereof for delivering agents in vivo including nucleic acids and proteins. These compositions are not subject to accelerated blood clearance and they have an improved toxicity profile in vivo.
    Type: Grant
    Filed: October 11, 2019
    Date of Patent: March 29, 2022
    Assignee: ModernaTX, Inc.
    Inventors: Gilles Besin, Stephen Hoge, Joseph Senn, Kerry Benenato, Staci Sabnis
  • Publication number: 20220071915
    Abstract: The invention relates to mRNA therapy for the treatment of Citrullinemia Type 2 (“CTLN2”). mRNAs for use in the invention, when administered in vivo, encode human Citrin, isoforms thereof, functional fragments thereof, and fusion proteins comprising Citrin. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of Citrin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of biomarkers associated with deficient Citrin activity in subjects, namely ammonia and/or triglycerides.
    Type: Application
    Filed: January 21, 2021
    Publication date: March 10, 2022
    Applicant: ModernaTX, Inc.
    Inventors: Paolo Martini, Stephen Hoge, Kerry Benenato, Vladimir Presnyak, Iain McFadyen, Ellalahewage Sathyajith Kumarasinghe, Jingsong Cao, Lin Tung Guey, Staci Sabnis
  • Publication number: 20210269830
    Abstract: The invention relates to mRNA therapy for the treatment of galactosemia type 1 (Gal-1). mRNAs for use in the invention, when administered in vivo, encode human galactose-1-phosphate uridylyltransferase (GALT), isoforms thereof, functional fragments thereof, and fusion proteins comprising GALT. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GALT expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GALT activity in subjects, namely galactose-1-phosphate (Gal-1-P).
    Type: Application
    Filed: January 21, 2021
    Publication date: September 2, 2021
    Applicant: ModernaTX, Inc.
    Inventors: Paolo Martini, Stephen Hoge, Kerry Benenato, Vladimir Presnyak, Iain McFadyen, Ellalahewage Sathyajith Kumarasinghe, Ding An, Staci Sabnis
  • Patent number: 11001861
    Abstract: The invention relates to mRNA therapy for the treatment of galactosemia type 1 (Gal-1). mRNAs for use in the invention, when administered in vivo, encode human galactose-1-phosphate uridylyltransferase (GALT), isoforms thereof, functional fragments thereof, and fusion proteins comprising GALT. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GALT expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GALT activity in subjects, namely galactose-1-phosphate (Gal-1-P).
    Type: Grant
    Filed: May 18, 2017
    Date of Patent: May 11, 2021
    Assignee: ModernaTX, Inc.
    Inventors: Paolo Martini, Stephen Hoge, Kerry Benenato, Vladimir Presnyak, Iain McFadyen, Ellalahewage Sathyajith Kumarasinghe, Ding An, Staci Sabnis
  • Patent number: 10993918
    Abstract: The invention relates to mRNA therapy for the treatment of Citrullinemia Type 2 (“CTLN2”). mRNAs for use in the invention, when administered in vivo, encode human Citrin, isoforms thereof, functional fragments thereof, and fusion proteins comprising Citrin. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of Citrin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of biomarkers associated with deficient Citrin activity in subjects, namely ammonia and/or triglycerides.
    Type: Grant
    Filed: May 18, 2017
    Date of Patent: May 4, 2021
    Assignee: ModernaTX, Inc.
    Inventors: Paolo Martini, Stephen Hoge, Kerry Benenato, Vladimir Presnyak, Iain McFadyen, Ellalahewage Sathyajith Kumarasinghe, Jingsong Cao, Lin Tung Guey, Staci Sabnis
  • Publication number: 20200206362
    Abstract: This disclosure provides improved lipid-based compositions, including lipid nanoparticle compositions, and methods of use thereof for delivering agents in vivo including nucleic acids and proteins. These compositions are not subject to accelerated blood clearance and they have an improved toxicity profile in vivo.
    Type: Application
    Filed: October 11, 2019
    Publication date: July 2, 2020
    Applicant: Moderna TX, Inc.
    Inventors: Gilles Besin, Stephen Hoge, Joseph Senn, Kerry Benenato, Staci Sabnis
  • Publication number: 20200149052
    Abstract: The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the ?-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.
    Type: Application
    Filed: September 13, 2019
    Publication date: May 14, 2020
    Inventors: Paolo MARTINI, Stephen G. HOGE, Kerry BENENATO, Vladimir PRESNYAK, Iain MCFADYEN, Ellalahewage Sathyajith KUMARASINGHE, Xuling ZHU, Lin Tung GUEY, Staci SABNIS
  • Publication number: 20200129445
    Abstract: The disclosure features novel lipids and compositions involving the same. Nanoparticle compositions include an ionizable lipid, a phospholipid, a first sterol or a tocopherol, and optionally a second sterol different from the first sterol. Nanoparticle compositions further including therapeutic and/or prophylactics such as RNA are useful in the delivery of therapeutic and/or prophylactics to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.
    Type: Application
    Filed: March 15, 2018
    Publication date: April 30, 2020
    Inventors: Siddharth PATEL, Emily ROBINSON, Anna BROWN, Orn ALMARSSON, Kerry E. BENENATO, Staci SABNIS, Gaurav SAHAY, Ashwani Kumar NARAYANA
  • Publication number: 20200085916
    Abstract: The invention relates to mRNA therapy for the treatment of Acute Intermittent Porphyria (AIP). mRNAs for use in the invention, when administered in vivo, encode human porphobilinogen deaminase (PBGD), isoforms thereof, functional fragments thereof, and fusion proteins comprising PBGD. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to affect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of PBGD expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient PBGD activity in subjects, namely porphobilinogen and aminolevulinate (PBG and ALA).
    Type: Application
    Filed: May 18, 2017
    Publication date: March 19, 2020
    Inventors: Paolo Martini, Stephen Hoge, Kerry Benenato, Vladimir Presnyak, Lei Jiang, Iain McFadyen, Ellalahewage Sathyajith Kumarasinghe, Antonio Fontanellas Roma, Pedro Berraondo Lopez, Matias Antonio Avila Zaragoza, Lin Tung Guey, Staci Sabnis
  • Patent number: 10556018
    Abstract: This disclosure provides improved lipid-based compositions, including lipid nanoparticle compositions, and methods of use thereof for delivering agents in vivo including nucleic acids and proteins. These compositions are not subject to accelerated blood clearance and they have an improved toxicity profile in vivo.
    Type: Grant
    Filed: August 10, 2017
    Date of Patent: February 11, 2020
    Inventors: Gilles Besin, Stephen Hoge, Joseph Senn, Kerry Benenato, Staci Sabnis
  • Patent number: 10519455
    Abstract: The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the ?-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.
    Type: Grant
    Filed: August 23, 2018
    Date of Patent: December 31, 2019
    Assignee: ModernaTX, Inc.
    Inventors: Paolo Martini, Stephen G. Hoge, Kerry Benenato, Vladimir Presnyak, Iain McFadyen, Ellalahewage Sathyajith Kumarasinghe, Xuling Zhu, Lin Tung Guey, Staci Sabnis
  • Publication number: 20190382774
    Abstract: The invention related to polyribonucleotides comprising an open reading frame of linked nucleosides encoding a polypeptide of interest (e.g., a therapeutic polypeptide), isoforms thereof, functional fragments thereof, and fusion proteins comprising the polypeptide. In some embodiments, the open reading frame is sequence-optimized. In particular embodiments, the invention provides sequence-optimized polyribonucleotides comprising nucleotides encoding the sequence of the polypeptide of interest, or sequence having high sequence identity with those sequence optimized polyribonucleotides.
    Type: Application
    Filed: May 18, 2017
    Publication date: December 19, 2019
    Inventors: Stephen G. HOGE, Kerry BENENATO, Vladimir PRESNYAK, Iain MCFADYEN, Ellalahewage Sathyajith KUMARASINGHE, Staci SABNIS, William BUTCHER
  • Patent number: 10494636
    Abstract: The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the ?-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.
    Type: Grant
    Filed: August 23, 2018
    Date of Patent: December 3, 2019
    Assignee: ModernaTX, Inc.
    Inventors: Paolo Martini, Stephen G. Hoge, Kerry Benenato, Vladimir Presnyak, Iain McFadyen, Ellalahewage Sathyajith Kumarasinghe, Xuling Zhu, Lin Tung Guey, Staci Sabnis