153 patents
Utility
Heterologous UTR Sequences for Enhanced mRNA Expression
16 Feb 23
mRNAs containing an exogenous open reading frame (ORF) flanked by a 5′ untranslated region (UTR) and a 3′ UTR is provided, wherein the 5′ and 3′ UTRs are derived from a naturally abundant mRNA in a tissue.
David H. Burkhardt, Romesh R. Subramanian, Christian Cobaugh
Filed: 21 Jun 22
Utility
Human Cytomegalovirus Vaccine
9 Feb 23
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: 13 Jun 22
Utility
Combinations of Mrnas Encoding Immune Modulating Polypeptides and Uses Thereof
9 Feb 23
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 FREDERICK, Ailin BAI, Vladimir PRESNYAK, Stephen HOGE, Kerry BENENATO, Iain MCFADYEN, Ellalahewage Sathyajith KUMARASINGHE, Susannah HEWITT
Filed: 27 May 22
Utility
Compositions and Methods for Delivery of Agents to Immune Cells
26 Jan 23
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.
Gilles BESIN, Luis BRITO, Stephen G. HOGE, Edward HENNESSY, Mark CORNEBISE, Kerry BENENATO, Staci SABNIS, Michael W. DANNEMAN
Filed: 18 May 22
Utility
Combinations of Mrnas Encoding Immune Modulating Polypeptides and Uses Thereof
26 Jan 23
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 FREDERICK, Ailin BAI, Vladimir PRESNYAK, Stephen HOGE, Kerry BENENATO, Iain MCFADYEN, Ellalahewage Sathyajith KUMARASINGHE, Susannah HEWITT
Filed: 7 Sep 22
Utility
Infectious Disease Vaccines
19 Jan 23
Aspects of the disclosure relate to nucleic acid vaccines.
Giuseppe Ciaramella, Eric Yi-Chun Huang, Kapil Bahl, Tal Zaks, Sunny Himansu, Sayda Mahgoub Elbashir
Filed: 5 May 22
Utility
Signal-sensor Polynucleotides for the Alteration of Cellular Phenotypes
19 Jan 23
The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of signal-sensor polynucleotides, primary transcripts and mmRNA molecules.
Stephen G. HOGE, Tirtha CHAKRABORTY, Joshua P. FREDERICK, Matthias JOHN, Antonin DE FOUGEROLLES
Filed: 29 Jan 21
Utility
Polynucleotides Encoding GLUCOSE-6-PHOSPHATASE for the Treatment of Glycogen Storage Disease
12 Jan 23
This disclosure relates to mRNA therapy for the treatment of glycogen storage disease type 1a, (GSD-Ia), and related symptoms such as hypoglycemia. mRNAs for use in the invention, when administered in vivo, encode human glucose-6-phosphatase (G6Pase or G6PC), and functional fragments and variants thereof. 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 G6PC expression and/or activity in subjects. mRNA therapies of the invention further increase the glucose production, and reduce the abnormal accumulation of glycogen and/or glucose-6-phosphate associated with GSD-Ia.
Paolo G. V. Martini, Athanasios Dousis, Vladimir Presnyak, Jingsong Cao
Filed: 12 Sep 19
Utility
Seasonal Rna Influenza Virus Vaccines
5 Jan 23
The disclosure provides RNA vaccines for seasonal influenza virus as well as methods of using the vaccines.
Raffael Nachbagauer, Carole Henry, Patricia Jorquera
Filed: 10 Jan 22
Utility
Novel Headgroup Lipid Compounds and Compositions for Intracellular Delivery of Therapeutic Agents
5 Jan 23
The disclosure features novel lipids and compositions involving the same.
Kerry E. BENENATO, Mark CORNEBISE, Edward HENNESSY, Andrew MCKENZIE
Filed: 18 Sep 20
Utility
Varicella Zoster Virus (VZV) Vaccine
29 Dec 22
Aspects of the disclosure relate to nucleic acid vaccines.
Giuseppe Ciaramella
Filed: 30 Apr 21
Utility
Compounds and Compositions for Intracellular Delivery of Therapeutic Agents
29 Dec 22
The disclosure features novel lipids and compositions involving the same.
Kerry E. BENENATO, Mark CORNEBISE, Edward HENNESSY
Filed: 19 Sep 19
Utility
Carbonate Containing Lipid Compounds and Compositions for Intracellular Delivery of Therapeutic Agents
29 Dec 22
The disclosure features novel lipids and compositions involving the same.
Kerry E. BENENATO, Souvik BISWAS, Mark CORNEBISE, Edward HENNESSY, Ellalahewage S. KUMARASINGHE
Filed: 18 Sep 20
Utility
Human Cytomegalovirus Vaccine
22 Dec 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: 14 Jun 22
Utility
Polynucleotides Encoding Uridine Diphosphate Glycosyltransferase 1 Family, Polypeptide A1 for the Treatment of Crigler-najjar Syndrome
22 Dec 22
This disclosure relates to mRNA therapy for the treatment of Crigler-Najjar Syndrome Type 1 (CN-1). mRNAs for use in the invention, when administered in vivo, encode uridine diphosphate glycosyltransferase 1 family, polypeptide A1 (UGT1A1). mRNA therapies of the disclosure increase and/or restore deficient levels of UGT1A1 expression and/or activity in subjects. mRNA therapies of the disclosure further decrease abnormal accumulation of bilirubin associated with deficient UGT1A1 activity in subjects.
Paolo G. V. Martini, Vladimir Presnyak
Filed: 13 Sep 19
Utility
Sterol Analogs and Uses Thereof
22 Dec 22
The invention relates to compositions and methods for the preparation, manufacture, and therapeutic use of compositions comprising mRNA and a lipid nanoparticle comprising a compound of the invention and an ionizable lipid.
Kerry BENENATO, Mark CORNEBISE, Edward J. HENNESSY
Filed: 19 Sep 19
Utility
Terminally Modified Rna
15 Dec 22
The invention relates to compositions and methods for the manufacture and optimization of modified mRNA molecules via optimization of their terminal architecture.
Tirtha CHAKRABORTY, Stephane BANCEL, Stephen G. HOGE, Atanu ROY, Antonin DE FOUGEROLLES, Noubar B. AFEYAN
Filed: 18 Dec 20
Utility
Messenger Rna Comprising Functional Rna Elements and Uses Thereof
8 Dec 22
The present disclosure provides messenger RNAs (mRNAs) having chemical and/or structural modifications, including RNA elements and/or modified nucleotides, which provide desirable regulation of mRNA localization, stability, and/or translation to yield increased mRNA expression and activity of an encoded polypeptide.
Ruchi JAIN, David REID, Vladimir PRESNYAK, Alicia BICKNELL, Caroline KÖHRER
Filed: 24 Jun 20
Utility
HMPV Mrna Vaccine Composition
1 Dec 22
Provided herein are vaccine composition comprising a chemically-modified messenger ribonucleic acid (mRNA) encoding a hMPV fusion (F) glycoprotein and a chemically-modified mRNA encoding a hPIV3 F glycoprotein formulated in a cationic lipid nanoparticle formulation, and related method for inducing an antigen-specific immune response.
Lori Panther, Christine Shaw, Igor Smolenov, Michael Watson, Tal Zaks
Filed: 5 May 22
Utility
Methods of Using OX40 Ligand Encoding Polynucleotides
1 Dec 22
The disclosure relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotide molecules comprising an mRNA encoding an OX40L polypeptide.
Joshua P. FREDERICK, Ailin BAI
Filed: 6 Jul 22