72 patents
Page 2 of 4
Utility
Lipid nanoparticle compositions and methods for mRNA delivery
5 Apr 22
Disclosed herein are compositions and methods for modulating the production of a protein in a target cell.
Braydon Charles Guild, Frank DeRosa, Michael Heartlein
Filed: 25 Oct 21
Utility
Nucleic acid conjugates and uses thereof
22 Feb 22
Provided herein are conjugates comprising targeting moieties such as sugars, folates and cell-penetrating peptides, which can be used for the improved delivery of agents (e.g., nucleic acids, such as oligonucleotides or mRNAs, or other agents) to cells.
Balkrishen Bhat, Saswata Karmakar, Debatosh Majumdar, Jia Tay, Nelson Chau
Filed: 11 Jul 17
Utility
Codon-optimized CFTR MRNA
22 Feb 22
The present invention provides, among other things, improved methods and pharmaceutical compositions for treating cystic fibrosis based on codon optimized mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein.
Anusha Dias, Jonathan Abysalh, Brian Bettencourt, Frank DeRosa, Michael Heartlein
Filed: 27 Feb 18
Utility
Nuclease resistant polynucleotides and uses thereof
22 Feb 22
The invention provides, among other things, methods of stabilizing mRNA and nuclease resistant mRNA prepared in accordance with such methods.
Michael Heartlein, Braydon Charles Guild, Frank DeRosa
Filed: 18 May 18
Utility
Cleavable lipids
1 Feb 22
Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use.
Braydon Charles Guild, Michael Heartlein, Frank DeRosa, Jerry Chi Zhang
Filed: 13 Feb 20
Utility
MRNA therapy for argininosuccinate synthetase deficiency
18 Jan 22
The present invention provides, among other things, methods of treating Argininosuccinate Synthetase Deficiency (ASD), including administering to a subject in need of treatment a composition comprising an mRNA encoding argininosuccinate synthetase (ASS1) at an effective dose and an administration interval such that at least one symptom or feature of ASD is reduced in intensity, severity, or frequency or has delayed in onset.
Michael Heartlein, Frank DeRosa, Lianne Smith
Filed: 22 Oct 14
Utility
Lipid nanoparticle compositions and methods for mRNA delivery
30 Nov 21
Disclosed herein are compositions and methods for modulating the production of a protein in a target cell.
Braydon Charles Guild, Frank DeRosa, Michael Heartlein
Filed: 7 Jun 21
Utility
Treatment of cystic fibrosis by delivery of codon-optimized mRNA encoding CFTR
16 Nov 21
The present invention provides, among other things, methods of treating cystic fibrosis, comprising a step of administering to a subject in need of treatment a composition comprising an mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, wherein the mRNA encoding the CFTR protein comprises a polynucleotide sequence at least 80% identical to SEQ ID NO: 1, wherein the mRNA is at a concentration of at least 0.4 mg/mL, and wherein the step of administering comprises inhalation.
Michael Heartlein, Frank DeRosa, Alan Kimura, Jonathan Abysalh, Anusha Dias, Shrirang Karve, Zarna Patel
Filed: 16 May 18
Utility
Methods for purification of messenger RNA
16 Nov 21
The present invention provides, among other things, methods for purifying mRNA based on normal flow filtration for therapeutic use.
Joseph Parella, Kimberly Gillis, Jonathan Abysalh, Travis Jeannotte, Erik Held, Frank DeRosa, Michael Heartlein, Rebecca Powell
Filed: 26 Aug 19
Utility
Composition and methods for treatment of ornithine transcarbamylase deficiency
9 Nov 21
The present invention provides, among other things, methods of treating ornithine transcarbamylase deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase protein at a low dose and at an administration interval such that at least one symptom or feature of the OTC deficiency is reduced.
Kim Askew, Jou-Ku Chung, Frank DeRosa, Michael Heartlein, Shrirang Karve, Thomas McCauley, Lianne Smith, Ann J. Barbier
Filed: 20 Dec 18
Utility
MRNA for use in treatment of human genetic diseases
5 Oct 21
Compositions for modulating the expression of a protein in a target cell comprising at least one RNA molecule which comprises at least one modification 5 conferring stability to the RNA, as well as related methods, are disclosed.
Braydon Charles Guild, Michael Heartlein, Frank DeRosa
Filed: 27 Mar 18
Utility
Multimeric coding nucleic acid and uses thereof
21 Sep 21
The present invention provides, among other things, multimeric coding nucleic acids that exhibit superior stability for in vivo and in vitro use.
Frank DeRosa, Michael Heartlein, Daniel Crawford, Shrirang Karve
Filed: 14 Aug 19
Utility
Stereochemically enriched compositions for delivery of nucleic acids
31 Aug 21
Frank DeRosa, Shrirang Karve, Michael Heartlein
Filed: 1 Nov 18
Utility
Quantitative assessment for cap efficiency of messenger RNA
31 Aug 21
The present invention provides, among other things, methods of quantitating mRNA capping efficiency, particularly mRNA synthesized in vitro.
Michael Heartlein, Frank DeRosa, Anusha Dias
Filed: 16 Jan 20
Utility
Pulmonary delivery of mRNA to non-lung target cells
17 Aug 21
Compositions comprising mRNA formulated for pulmonary administration and related method for delivery of the mRNA and/or encoded protein to a non-lung cell or tissue.
Michael Heartlein, Braydon Charles Guild, Frank DeRosa, Carsten Rudolph, Christian Plank
Filed: 25 Jan 19
Utility
MRNA therapy for Pompe disease
17 Aug 21
The present invention provides, among other things, methods of treating Pompe disease, including administering to a subject in need of treatment a composition comprising an mRNA encoding acid alpha-glucosidase (GAA) at an effective dose and an administration interval such that at least one symptom or feature of Pompe disease is reduced in intensity, severity, or frequency or has delayed in onset.
Frank DeRosa, Michael Heartlein
Filed: 14 Nov 18
Utility
Methods for purification of messenger RNA
13 Jul 21
The present invention provides, among other things, methods of purifying messenger RNA (mRNA) including the steps of (a) precipitating mRNA from an impure preparation; (b) subjecting the impure preparation comprising precipitated mRNA to a purification process involving membrane filtration such that the precipitated mRNA is captured by a membrane; and (c) eluting the captured precipitated mRNA from the membrane by re-solubilizing the mRNA, thereby resulting in a purified mRNA solution.
Frank DeRosa, Anusha Dias, Shrirang Karve, Michael Heartlein
Filed: 20 Nov 18
Utility
Lipid nanoparticle compositions and methods for mRNA delivery
6 Jul 21
Disclosed herein are compositions and methods for modulating the production of a protein in a target cell.
Braydon Charles Guild, Frank DeRosa, Michael Heartlein
Filed: 16 Nov 20
Utility
Ice-based lipid nanoparticle formulation for delivery of mRNA
25 May 21
The present invention provides, among other things, compositions and methods of formulating nucleic acid-containing nanoparticles comprising no more than three distinct lipids components, one distinct lipid component being a sterol-based cationic lipid.
Frank DeRosa, Michael Heartlein, Shrirang Karve
Filed: 11 Oct 19
Utility
Modification of RNA-related enzymes for enhanced production
4 May 21
The present invention provides, among other things, methods and compositions for large-scale production of capped mRNA using SUMO-Guanylyl Transferase fusion protein.
Bettina Strack-Logue, Frank DeRosa, Michael Heartlein
Filed: 19 Oct 18