228 patents
Page 11 of 12
Utility
Modified mRNA Encoding a Glucose 6 Phosphatase and Uses Thereof
10 Jun 20
Described herein are methods and compositions for treating a glucose 6 phosphatase deficiency based on mRNA therapy.
Jeremiah Farelli, Daniel Roseman, Romesh R. Subramanian
Filed: 23 Jul 18
Utility
Chikungunya virus RNA vaccines
8 Jun 20
The disclosure relates to tropical diseases such as viral mosquito borne illnesses and the treatment thereof.
Giuseppe Ciaramella, Sunny Himansu, Eric Yi-Chun Huang, Tal Zaks, Sayda Mahgoub Elbashir
Filed: 19 Sep 18
Utility
Modified mRNA Encoding a Propionyl-CoA Carboxylase and Uses Thereof
27 May 20
Disclosed are methods and compositions for treating propionic academia based on mRNA therapy.
Susan Sobolov-Jaynes, Romesh R. Subramanian, Judith L. Campagnari, Haren Vasavada, Zhiliang Cheng
Filed: 19 Jul 18
Utility
Modified Nucleoside, Nucleotide, and Nucleic Acid Compositions
27 May 20
The present disclosure provides, inter alia, formulation compositions comprising modified nucleic acid molecules which may encode a protein, a protein precursor, or a partially or fully processed form of the protein or a protein precursor.
Antonin DE FOUGEROLLES, Kristy M. WOOD, Sayda M. ELBASHIR, Noubar B. AFEYAN, Pedro VALENCIA, Jason P. SCHRUM
Filed: 28 Jul 19
Utility
Modified Polynucleotides for the Production of Cytoplasmic and Cytoskeletal Proteins
20 May 20
The invention relates to compositions including polynucleotides encoding polypeptides which have been chemically modified by replacing the uridines with 1-methyl-pseudouridine to improve one or more of the stability and/or clearance in tissues, receptor uptake and/or kinetics, cellular access by the compositions, engagement with translational machinery, mRNA half-life, translation efficiency, immune evasion, protein production capacity, secretion efficiency, accessibility to circulation, protein half-life and/or modulation of a cell's status, function, and/or activity.
Antonin DE FOUGEROLLES, Justin GUILD
Filed: 19 Sep 19
Utility
High purity RNA compositions and methods for preparation thereof
18 May 20
The invention relates to improved RNA compositions for use in therapeutic applications.
Stephen Hoge, William Issa, Edward J. Miracco, Jennifer Nelson, Amy E. Rabideau, Gabor Butora
Filed: 5 Jun 18
Utility
Zika virus MRNA vaccines
18 May 20
Provided herein, in some embodiments, are Zika virus RNA vaccines and methods of producing an antigen-specific immune response in a subject.
Giuseppe Ciaramella, Sunny Himansu
Filed: 13 Sep 18
Utility
Polynucleotides Encoding Alpha-galactosidase a for the Treatment of Fabry Disease
13 May 20
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 α-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.
Paolo MARTINI, Stephen G. HOGE, Kerry BENENATO, Vladimir PRESNYAK, Iain MCFADYEN, Ellalahewage Sathyajith KUMARASINGHE, Xuling ZHU, Lin Tung GUEY, Staci SABNIS
Filed: 12 Sep 19
Utility
Polynucleotides encoding interleukin-12 (IL12) and uses thereof
11 May 20
The present disclosure relates to polynucleotides comprising an open reading frame of linked nucleosides encoding human interleukin-12 (IL12), functional fragments thereof, and fusion proteins comprising IL12.
Joshua Frederick, Susannah Hewitt, Ailin Bai, Stephen Hoge, Vladimir Presnyak, Iain Mcfadyen, Kerry Benenato, Ellalahewage Sathyajith Kumarasinghe
Filed: 14 Nov 18
Utility
Crystal Forms of Amino Lipids
29 Apr 20
Provided herein are novel solid forms of each of four compounds: (1) heptadecan-9-yl 8-((2-hydroxyethyl)amino)octanoate (“Compound 1”), (2) heptadecan-9-yl 8-((2-hydroxyethyl)(6-oxo-6-(undecyloxy)hexyl)amino)octanoate (“Compound 2”), (3) heptadecan-9-yl 8-((2-hydroxyethyl)(8-(nonyloxy)-8-oxooctyl)amino)octanoate (“Compound 3”), and (6Z,9Z,28Z,31Z)-heptatriaconta-6,9,28,31-tetraen-19-yl 4-(dimethylamino)butanoate (“MC3”), and related compositions and methods.
Orn ALMARSSON, Eugene CHEUNG
Filed: 14 Mar 18
Utility
Rna Polymerase Variants
29 Apr 20
The present disclosure provides, in some aspects, variant RNA polymerases, the use of which increases transcription efficiency while reducing the number of double-stranded RNA contaminates and run-on transcripts produced during an in vitro transcription reaction.
Amy E. Rabideau, Athanasios Dousis, Kanchana Ravichandran, Elissa Hobert
Filed: 17 Oct 19
Utility
Polynucleotides Encoding Methylmalonyl-coa Mutase
29 Apr 20
The disclosure relates to polynucleotides comprising an open reading frame of linked nucleosides encoding human methylmalonyl-CoA mutase precursor, human methylmalonyl-CoA mutase (MCM) mature form, or functional fragments thereof.
Paolo G. V. Martini, Vladimir Presnyak
Filed: 12 Jun 18
Utility
Respiratory Syncytial Virus Vaccine
29 Apr 20
The disclosure relates to respiratory syncytial virus (RSV) ribonucleic acid (RNA) vaccines, as well as methods of using the vaccines and compositions comprising the vaccines.
Giuseppe Ciaramella, Kapil Bahl, Amy Espeseth, Dai Wang, Andrew J. Bett
Filed: 14 Mar 18
Utility
Lipid Nanoparticle Formulation
29 Apr 20
The disclosure features novel lipids and compositions involving the same.
Siddharth PATEL, Emily ROBINSON, Anna BROWN, Orn ALMARSSON, Kerry E. BENENATO, Staci SABNIS, Gaurav SAHAY, Ashwani Kumar NARAYANA
Filed: 14 Mar 18
Utility
Herpes Simplex Virus Vaccine
29 Apr 20
The disclosure relates to herpes simplex virus (HSV) ribonucleic acid (RNA) vaccines, as well as methods of using the vaccines and compositions comprising the vaccines.
Giuseppe Ciaramella, Shinu John, Andrew J. Bett, Danilo R. Casimiro, Dai Wang, Lan Zhang
Filed: 14 Mar 18
Utility
Compounds and Compositions for Intracellular Delivery of Therapeutic Agents
22 Apr 20
The disclosure features novel lipids and compositions involving the same.
Kerry E. BENENATO, Mark CORNEBISE
Filed: 28 Aug 19
Utility
Combinations of Mrnas Encoding Immune Modulating Polypeptides and Uses Thereof
15 Apr 20
The disclosure relates to compositions and methods for the preparation, manufacture and therapeutic use of combinations of immunomodulatory polynucleotides (e.g., mRNAs) encoding an immune response primer polypeptide (e.g., an interleukin 23 (IL-23) polypeptide or an interleukin 36γ (IL-36-gamma) polypeptide), and an immune response co-stimulatory signal polypeptide (e.g., an OX40L polypeptide).
Joshua P. FREDERICK, Ailin BAI, Vladimir PRESNYAK, Stephen G. HOGE, Kerry BENENATO, Iain MCFADYEN, Ellalahewage Sathyajith KUMARASINGHE, Susannah HEWITT
Filed: 15 Aug 19
Utility
Reduction or Elimination of Immune Responses to Non-intravenous, E.g., Subcutaneously Administered Therapeutic Proteins
8 Apr 20
This disclosure provides improved lipid-based compositions, including lipid nanoparticle compositions, and methods of use thereof for delivering nucleic acids in vivo.
Luis Brito, Gilles Besin
Filed: 4 Apr 18
Utility
Polynucleotides Encoding Tethered INTERLEUKIN-12 (IL12) Polypeptides and Uses Thereof
1 Apr 20
The present disclosure relates to polynucleotides encoding tethered interleukin-12 (IL-12) polypeptides comprising an IL-12 polypeptide and a membrane domain.
Ankita MISHRA, Joshua FREDERICK, Sushma GURUMURTHY
Filed: 17 May 18
Utility
Polynucleotides Encoding Porphobilinogen Deaminase for the Treatment of Acute Intermittent Porphyria
18 Mar 20
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).
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
Filed: 17 May 17