153 patents
Page 6 of 8
Utility
Polynucleotides Encoding Phenylalanine Hydroxylase for the Treatment of Phenylketonuria
11 Feb 21
This disclosure relates to mRNA therapy for the treatment of hyperphenylalaninemias such as phenylketonuria (PKU). mRNAs for use in the invention, when administered in vivo, encode human phenylalanine hydroxylase (PAH), functional fragments thereof (e.g., those comprising the catalytic domain or the catalytic domain and the tetramerization domains), and fusion proteins comprising PAH. 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 PAH expression and/or activity in subjects. mRNA therapies of the invention further decrease abnormal accumulation of phenylalanine associated with deficient PAH activity in subjects.
Raj Rajendran, Patrick Finn, Paolo G.V. Martini, Ding An, Athanasios Dousis, Kanchana Ravichandran
Filed: 21 Nov 18
Utility
Combinations of Mrnas Encoding Immune Modulating Polypeptides and Uses Thereof
11 Feb 21
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 G. HOGE, Kerry BENENATO, Iain MCFADYEN, Ellalahewage Sathyajith KUMARASINGHE, Susannah HEWITT
Filed: 15 Oct 20
Utility
Combination HPIV3/HMPV Rna Vaccines
30 Dec 20
The disclosure relates to respiratory virus ribonucleic acid (RNA) vaccines and combination vaccines, as well as methods of using the vaccines and compositions comprising the vaccines.
Giuseppe Ciaramella, Sunny Himansu
Filed: 9 Jun 20
Utility
Modified Rna Encoding Vegf-a Polypeptides, Formulations, and Uses Relating Thereto
30 Dec 20
The disclosure relates to modified RNA molecules encoding VEGF-A polypeptides and formulations comprising the modified RNA.
Leif Karlsson PARINDER, Regina Desirée FRITSCHE DANIELSON, Kenny Mikael HANSSON, Li Ming GAN, Jonathan CLARKE, Ann-Charlotte Eva EGNELL, Kenneth Randall CHIEN
Filed: 5 Jun 17
Utility
Polynucleotides Encoding INTERLEUKIN-12 (IL12) and Uses Thereof
2 Dec 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: 6 Apr 20
Utility
Zika Virus Mrna Vaccines
25 Nov 20
Aspects of the disclosure relate to nucleic acid vaccines.
Giuseppe Ciaramella, Eric Yi-Chun Huang, Kapil Bahl, Tal Zaks, Sunny Himansu
Filed: 9 Jun 20
Utility
Nucleic Acid-Based Therapy of Muscular Dystrophies
25 Nov 20
The invention related to polynucleotides comprising an open reading frame of linked nucleosides encoding therapeutic proteins or variant therapeutic proteins, isoforms thereof, functional fragments thereof, and fusion proteins comprising therapeutic proteins.
Paolo Martini
Filed: 23 Feb 18
Utility
Chikv Rna Vaccines
25 Nov 20
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: 9 Jun 20
Utility
Polynucleotides Encoding Anti-chikungunya Virus Antibodies
25 Nov 20
This disclosure relates to compositions and methods for treating and preventing chikungunya virus infection by delivering polynucleotides encoding anti-chikungunya virus antibodies to a subject.
Sunny Himansu, James E. Crowe, Jr., Giuseppe Ciaramella
Filed: 3 Jan 19
Utility
USE OF MRNAS ENCODING OX40L, IL-23 AND IL-36gamma IN COMBINATION WITH IMMUNE CHECKPOINT BLOCKADE FOR TREATING CANCER
18 Nov 20
The disclosure features methods for treating solid tumor malignancies and lymphomas by administering LNP encapsulated mRNAs encoding human OX40L, IL-23 and IL-36y polypeptides alone or in combination with checkpoint blockade.
Robert MEEHAN, Tal ZAKS
Filed: 7 Jan 20
Utility
Methods of Preparing Modified Rna
18 Nov 20
The present disclosure relates to methods for preparing a modified RNA comprising enzymatically ligating an acceptor moiety having a 3′-hydroxyl group to the 5′-end of a donor moiety (e.g., installation of 5′-triphosphate groups, mRNA caps, or cap-like structures on chemically synthesized RNA or installation of 5′-triphosphate groups, mRNA caps, cap-like structures, or non-cap like structures on enzymatically prepared RNA).
Kristen OTT, Aaron LARSEN, I-Ping CHAN, Jennifer NELSON, William ISSA, Melissa J. MOORE
Filed: 16 Aug 18
Utility
Polynucleotides Encoding Relaxin
11 Nov 20
The invention relates to mRNA therapy for the treatment of fibrosis and/or cardiovascular disease. mRNAs for use in the invention, when administered in vivo, encode human relaxin, isoforms thereof, functional fragments thereof, and fusion proteins comprising relaxin. 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 relaxin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient relaxin activity in subjects.
Barry Ticho, Nadege Briancon-Eris, Zhinan Xia, Athanasios Dousis, Seymour de Picciotto, Vladimir Presnyak, Stephen Hoge, Iain Mcfadyen, Kerry Benenato, Ellalahewage Sathyajith Kumarasinghe
Filed: 29 Jul 20
Utility
Modified Polynucleotides for the Production of Secreted Proteins
11 Nov 20
The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.
Antonin DE FOUGEROLLES, Justin GUILD
Filed: 1 Jul 20
Utility
Lipid Nanoparticles for Delivering Modified Rna Encoding a Vegf-a Polypeptide
28 Oct 20
The disclosure relates to nanoparticles comprising a lipid component and a modified RNA encoding a VEGF-A polypeptide.
Kenny Mikael Hansson, Kerry Benenato, Maria Wågberg, Annika Pålsson, Regina Fritsche-Danielson
Filed: 30 Oct 18
Utility
Human Cytomegalovirus Vaccine
28 Oct 20
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: 30 Apr 20
Utility
Lipid Nanoparticles
7 Oct 20
The invention features methods and apparatus for producing lipid nanoparticles.
Benjamin Frank GELDHOF
Filed: 20 Jun 17
Utility
Methods and Apparatus for Filtration
7 Oct 20
The invention features methods and apparatus for concentrating a nucleic acid.
Benjamin Frank GELDHOF
Filed: 20 Jun 17
Utility
Methods and Apparatus for Purifying Rna
30 Sep 20
The invention features methods and apparatus for purifying a sample including an RNA (e.g., an RNA having a poly(A) sequence, such as an mRNA).
Benjamin Frank GELDHOF
Filed: 20 Jun 17
Utility
Methods of Making Lipid Nanoparticles
30 Sep 20
The disclosure features novel methods of producing nucleic acid lipid nanoparticle (LNP) compositions employing a modifying agent after formation of a precursor nucleic acid lipid nanoparticle, the produced compositions thereof, and methods involving the nucleic acid lipid nanoparticles useful in the delivery of therapeutics and/or prophylactics, such as a nucleic acid, to mammalian cells or organs to, for example, to regulate polypeptide, protein, or gene expression.
Joseph SCHARITER, Kimberly HASSETT, Mike SMITH, Orn ALMARSSON, Luis BRITO
Filed: 30 Aug 18
Utility
Polynucleotides Encoding Ornithine Transcarbamylase for the Treatment of Urea Cycle Disorders
23 Sep 20
This disclosure relates mRNA therapy for the treatment of ornithine transcarbamylase deficiency (OTCD). mRNAs for use in the invention, when administered in vivo, encode human ornithine transcarbamylase (OTC), isoforms thereof, functional fragments thereof, and fusion proteins comprising OTC. 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 OTC expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic ammonia associated with deficient OTC activity in subjects.
Zhijian Zhuo, Andrea Lea Frassetto, Paolo G.V. Martini, Vladimir Presnyak, Patrick Finn
Filed: 20 Nov 18