153 patents
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Expanded T Cell Assay
28 Jul 22
Assays for assessing the therapeutic efficacy of vaccines, including personalized cancer vaccines are provided.
Kristen Hopson
Filed: 29 May 20
Utility
Respiratory Virus Nucleic Acid Vaccines
30 Jun 22
Provided herein, in some embodiments, are vaccines (and vaccination methods) that include a ribonucleic acid (RNA) polynucleotide encoding a human metapneumovirus (hMPV) F protein and a RNA polynucleotide encoding a human parainfluenza virus 3 (hPIV3) F protein.
Giuseppe Ciaramella, Sunny Himansu
Filed: 26 Jul 21
Utility
Nucleic Acid Vaccines
23 Jun 22
The invention relates to compositions and methods for the preparation, manufacture and therapeutic use ribonucleic acid vaccines (NAVs) comprising polynucleotide molecules encoding one or more antigens.
Giuseppe Ciaramella, Axel Bouchon, Eric Yi-Chun Huang
Filed: 28 Feb 22
Utility
Mrna Based Enzyme Replacement Therapy Combined with a Pharmacological Chaperone for the Treatment of Lysosomal Storage Disorders
16 Jun 22
This disclosure relates to treatment of lysosomal storage disorders, such as Fabry disease or Gaucher disease, with a combination treatment containing (i) an mRNA encoding a lysosomal enzyme deficient in the lysosomal storage disorder, and (ii) a compound that is a glucosylceramide synthase inhibitor or a pharmacological chaperone of the lysosomal enzyme. mRNAs for use in the invention, when administered in vivo, encode the enzyme that is deficient in the lysosomal storage disorder, functional fragments thereof (e.g., those comprising the catalytic domain), or fusion proteins containing the enzyme that is deficient in the lysosomal storage disorder. mRNA therapies can be used to increase and/or restore deficient levels of a lysosomal enzyme's expression and/or activity in subjects.
John Mendlein
Filed: 22 Jul 19
Utility
Zika Virus Rna Vaccines
9 Jun 22
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: 19 Nov 21
Utility
Fed-batch In Vitro Transcription Process
12 May 22
The present disclosure provides methods of in vitro transcribing a ribonucleic acid (RNA) of interest.
Joseph Elich, Amy E. Rabideau, Michael Shamashkin, Rosalie Philpot, Brian Fritz, Peter Wojciechowski
Filed: 11 Mar 20
Utility
Rna Cancer Vaccines
28 Apr 22
The present application is related to a method of treating a cancer by administering to a human subject multiple doses of a mRNA cancer vaccine formulated as a lipid nanoparticle wherein the cancer vaccine comprises one or more mRNAs each having one or more open reading frames encoding 3-50 peptide epitopes, and wherein each of the peptide epitopes are portions of personalized cancer antigens or portions of cancer hotspot antigens.
Ted Ashburn, Kristen Hopson, Karen Keating, Joseph Senn, Christine Swenson, Benjamin Breton, Shan Zhong, Maija Garnaas
Filed: 7 Nov 19
Utility
Vortex Mixers and Associated Methods, Systems, and Apparatuses Thereof
28 Apr 22
A vortex mixer may have a vortex mixing chamber having a first wall, a second wall, and a side wall connecting the first wall and the second wall.
Stephanie KIRBY, Benjamin GELDHOF, Kaelyn VIK, Brie SKINNER
Filed: 31 Jan 20
Utility
CorrectedZoonotic Disease Rna Vaccines
21 Apr 22
The disclosure relates to Lassa virus, Nipah virus, and betacoronavirus ribonucleic acid vaccines as well as methods of using the vaccines and compositions comprising the vaccines.
Giuseppe Ciaramella, Sunny Himansu, Vladimir Presnyak, Kerry Benenato, Ellalahewage Sathyajith Kumarasinghe
Filed: 22 Jan 21
Utility
Polynucleotides Encoding Very Long-chain Acyl-coa Dehydrogenase for the Treatment of Very Long-chain Acyl-coa Dehydrogenase Deficiency
14 Apr 22
This disclosure relates to mRNA therapy for the treatment of very long-chain specific acyl-CoA dehydrogenase deficiency (VLCADD). mRNAs for use in the invention, when administered in vivo, encode human very long-chain specific acyl-CoA dehydrogenase (VLCAD). mRNA therapies of the disclosure increase and/or restore deficient levels of VLCAD expression and/or activity in subjects. mRNA therapies of the disclosure further decrease abnormal accumulation of acylcarnitine associated with deficient VLCAD activity in subjects.
Vladimir Presnyak, Paolo Martini
Filed: 29 Aug 19
Utility
COMPOSITIONS COMPRISING SYNTHETIC POLYNUCLEOTIDES ENCODING CRISPR RELATED PROTEINS AND SYNTHETIC SGRNAs AND METHODS OF USE
14 Apr 22
The present invention relates to compositions comprising and methods of using synthetic polynucleotides, e.g., modified mRNA, encoding CRISPR related proteins including dCAS9 and synthetic sgRNAs targeting a gene of interest.
Stephen G. HOGE, Eric Yi-Chun HUANG, Tirtha CHAKRABORTY
Filed: 25 May 21
Utility
Human Cytomegalovirus Vaccine
31 Mar 22
The disclosure relates to HCMV ribonucleic acid (RNA) vaccines, as well as methods of using the vaccines and compositions comprising the vaccines.
Giuseppe Ciaramella, Shinu John
Filed: 10 Dec 21
Utility
High Purity Rna Compositions and Methods for Preparation Thereof
31 Mar 22
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: 10 Nov 21
Utility
High Purity Rna Compositions and Methods for Preparation Thereof
31 Mar 22
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: 10 Nov 21
Utility
Mrna Combination Therapy for the Treatment of Cancer
31 Mar 22
The present disclosure relates to the use of nucleic acid (e.g., mRNA) combination therapies for the treatment of cancer.
Joshua P. FREDERICK, Susannah HEWITT, Ailin BAI, Stephen G. HOGE, Vladimir PRESNYAK, Iain MCFADYEN, Kerry BENENATO, Ellalahewage Sathyajith KUMARASINGHE
Filed: 16 Mar 21
Utility
CorrectedBroad Spectrum Influenza Virus Vaccine
17 Mar 22
The disclosure relates to broad spectrum influenza virus ribonucleic acid (RNA) vaccines, as well as methods of using the vaccines and compositions comprising the vaccine.
Giuseppe Ciaramella, Eric Yi-Chun Huang, Kerim Babaoglu, Jessica Anne Flynn, Lan Zhang, David Nickle
Filed: 15 Mar 18
Utility
Polynucleotides Encoding Citrin for the Treatment of Citrullinemia Type 2
10 Mar 22
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.
Paolo Martini, Stephen Hoge, Kerry Benenato, Vladimir Presnyak, Iain McFadyen, Ellalahewage Sathyajith Kumarasinghe, Jingsong Cao, Lin Tung Guey, Staci Sabnis
Filed: 21 Jan 21
Utility
Crystal Forms of Amino Lipids
10 Mar 22
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: 12 Nov 21
Utility
Human Cytomegalovirus Vaccine
3 Mar 22
Aspects of the disclosure relate to methods for producing an antigen-specific immune response to human cytomegalovirus (hCMV) in a subject by administering mRNA vaccines.
Jack F. Kramarczyk, Kimberly Hassett, Shinu John, Phil White, Andrea Carfi
Filed: 25 Aug 21
Utility
Methods of Preparing Lipid Nanoparticles
3 Mar 22
The present disclosure provides methods of producing lipid nanoparticle (LNP) formulations and the produced LNP formulations thereof.
Mike SMITH, Allen HORHOTA, Jason AUER, Brie SKINNER
Filed: 31 Jan 20