153 patents
Page 2 of 8
Utility
Compounds and Compositions for Intracellular Delivery of Therapeutic Agents
1 Dec 22
The disclosure features novel lipids and compositions involving the same.
Kerry E. BENENATO, Mark CORNEBISE
Filed: 30 Nov 21
Utility
Stabilized Formulations
24 Nov 22
Stabilized formulations of nucleic acids, including lipid nanoparticle formulations which encapsulate nucleic acids, are provided.
Edward J. Hennessy
Filed: 22 Apr 22
Utility
Polynucleotides Encoding Methylmalonyl-coa Mutase for the Treatment of Methylmalonic Acidemia
24 Nov 22
This disclosure relates to mRNA therapy for the treatment of methylmalonic acidemia (MMA). mRNAs for use in the invention, when administered in vivo, encode methylmalonyl-CoA mutase (MUT). mRNA therapies of the disclosure increase and/or restore deficient levels of MUT expression and/or activity in subjects.
Tal Zaks, Kelly Lindert, Lin Tung Guey
Filed: 8 May 20
Utility
Human Cytomegalovirus Vaccine
3 Nov 22
Aspects of the invention relate to methods for producing an antigen-specific immune response to human cytomegalovirus (hCMV) in a subject by administering mRNA vaccines comprising hCMV antigenic polypeptides gH, gL, UL128, UL130, UL131 A and gB formulated in lipid nanoparticles, wherein the antigen-specific immune response to hCMV results in neutralizing antibodies that have i) a geometric mean titer of at least 3-fold against epithelial cell infection or ii) a geometric mean ratio of 9-41 against epithelial cell infection or iii) a geometric mean ratio of 4-8-fold against fibroblast infection.
Lori Panther, Shinu John, Tal Zaks
Filed: 11 Sep 20
Utility
Processes for Purifying Downstream Products of In Vitro Transcription
3 Nov 22
Provided herein, in some embodiments, are methods of purifying low-salt RNA compositions using denaturing oligo-dT chromatography.
Michael Shamashkin, Matthew Scott, Peter Wojciechowski
Filed: 13 Aug 20
Utility
Lipid Nanoparticle Compositions and Methods of Formulating the Same
27 Oct 22
Provided herein are compositions and methods of reducing adduct formation.
Meredith Packer, Dipendra Gyawali, Serenus Hua, Gabor Butora, Gregory John Mercer
Filed: 27 Jun 22
Utility
Lnp-formulated Mrna Therapeutics and Use Thereof for Treating Human Subjects
13 Oct 22
The disclosure features methods of treatment comprising systemic administration of mRNA encoding a therapeutic protein and delivered by lipid nanoparticle to human subjects.
Allison AUGUST, Tal ZAKS, Stephen G. HOGE
Filed: 11 Sep 20
Utility
Coronavirus Rna Vaccines
13 Oct 22
The disclosure relates to coronavirus ribonucleic acid (RNA) vaccines as well as methods of using the vaccines and compositions comprising the vaccines.
Mihir Metkar, Valdimir Presnyak
Filed: 3 Nov 21
Utility
Compositions and Methods for Delivery of Nucleic Acids
22 Sep 22
The present disclosure relates to methods and compositions for modulating protein expression.
Aaron LARSEN, Jennifer NELSON, Melissa MOORE
Filed: 20 Sep 19
Utility
Rna Affinity Purification
15 Sep 22
Provided herein, in some embodiments, are methods of purifying a nucleic acid preparation.
Edward J. Miracco
Filed: 2 Jun 22
Utility
Compositions and Methods for Delivery of Rna Interference Agents to Immune Cells
8 Sep 22
The disclosure features lipid nanoparticles (LNPs) comprising RNA interference agents, such as siRNAs, and methods of delivery thereof to immune cells.
Eric Yi-Chun HUANG
Filed: 31 Jul 20
Utility
Modified Polynucleotides for the Production of Membrane Proteins
1 Sep 22
The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.
Stephane BANCEL, Tirtha CHAKRABORTY, Antonin DE FOUGEROLLES, Sayda M. ELBASHIR, Matthias JOHN, Atanu ROY, Susan WHORISKEY, Kristy M. WOOD, Paul HATALA, Jason P. SCHRUM, Kenechi EJEBE, Jeff Lynn ELLSWORTH, Justin GUILD
Filed: 28 Apr 20
Utility
Engineered Nucleic Acids and Methods of Use Thereof
1 Sep 22
Provided are compositions and methods for delivering biological moieties such as modified nucleic acids into cells to modulate protein expression.
Jason P. SCHRUM, Stephane BANCEL, Noubar B. AFEYAN, Kenechi EJEBE
Filed: 17 Nov 21
Utility
Lipid Nanoparticle Compositions and Methods of Formulating the Same
1 Sep 22
Provided herein are compositions and methods of reducing adduct formation.
Meredith Packer, Dipendra Gyawali, Serenus Hua, Gabor Butora, Gregory John Mercer
Filed: 22 Oct 21
Utility
Polynucleotides Encoding Propionyl-coa Carboxylase Alpha and Beta Subunits for the Treatment of Propionic Acidemia
25 Aug 22
This disclosure relates to mRNA therapy for the treatment of propionic acidemia (PA). mRNAs for use in the invention, when administered in vivo, encode human propionyl-CoA carboxylase alpha (PCCA) and/or human propionyl-CoA carboxylase beta (PCCB), and isoforms thereof, functional fragments thereof, and fusion proteins comprising PCCA and/or PCCB. 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 propionyl-CoA carboxylase (PCC) expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of disease-associated toxic metabolites associated with deficient PCCA or PCCB activity, in subjects.
Lei Jiang, Lin Tung Guey, Paolo G.V. Martini, Vladimir Presnyak
Filed: 21 Nov 18
Utility
Polynucleotides Encoding INTERLEUKIN-12 (IL12) and Uses Thereof
25 Aug 22
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 James MCFADYEN, Kerry BENENATO, Ellalahewage Sathyajith KUMARASINGHE
Filed: 15 Apr 22
Utility
Compositions and Methods for Delivery of Agents
25 Aug 22
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.
Gilles Besin, Stephen Hoge, Joseph Senn, Kerry Benenato, Staci Sabnis
Filed: 25 Jan 22
Utility
Tropical Disease Vaccines
18 Aug 22
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
Filed: 1 Feb 22
Utility
Endonuclease-resistant Messenger Rna and Uses Thereof
11 Aug 22
The present disclosure provides messenger RNAs (mRNAs) with alterations which provide increased endonuclease resistance to the mRNA and methods and uses thereof.
Alicia BICKNELL, Vladimir PRESNYAK, Caroline KÖHRER
Filed: 24 Jun 20
Utility
Polynucleotides Encoding Branched-chain Alpha-ketoacid Dehydrogenase Complex E1-ALPHA, E1-BETA, and E2 Subunits for the Treatment of Maple Syrup Urine Disease
4 Aug 22
This disclosure relates to mRNA therapy for the treatment of maple syrup urine disease (MSUD). mRNAs for use in the invention, when administered in vivo, encode branched chain α-ketoacid dehydrogenase complex (BCKDC) E1α, E1β, or E2mRNA therapies of the disclosure increase and/or restore deficient levels of E1α, E1β, or E2 expression and/or BCKDC activity in subjects. mRNA therapies of the invention further decrease abnormal accumulation of branched chain amino acids associated with deficient BCKDC activity in subjects.
Paolo G. V. Martini, Vladimir Presnyak, Paloma Hoban Giangrande, Kimberly Ann Coughlan
Filed: 12 Sep 19