212 patents
Page 6 of 11
Utility
Lipid Nanoparticle Compositions and Methods for Mrna Delivery
10 Feb 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
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
Multi-peg Lipid Compounds
20 Jan 22
Shrirang Karve, Yi Zhang, Frank DeRosa, Michael Heartlein
Filed: 7 Nov 19
Utility
Messenger Rna Therapy for Treatment of Ocular Diseases
20 Jan 22
The present invention provides, among other things, a method of ocular delivery of messenger RNA (mRNA), comprising administering into an eye of a subject in need of delivery a composition comprising an mRNA encoding a protein, such that the administration of the composition results in expression of the protein encoded by the mRNA in the eye.
John R. Androsavich, Frank DeRosa, Shrirang Karve
Filed: 8 Nov 19
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
Thioester Cationic Lipids
13 Jan 22
Yi Zhang, Shrirang Karve, Frank DeRosa, Michael Heartlein
Filed: 23 May 19
Utility
Modification of Rna-related Enzymes for Enhanced Production
13 Jan 22
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: 30 Mar 21
Utility
Mrna Therapy for Pompe Disease
30 Dec 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: 7 Jul 21
Utility
Methods and Compositions for Messenger RNA Purification
16 Dec 21
The present invention provides, among other things, methods for purifying mRNA, which involves removing impurities from a messenger RNA preparation synthesized by large scale in vitro transcription process (IVT), by precipitating the IVT-synthesized mRNA in a buffer comprising a denaturing salt in combination with a reducing agent, followed by capturing the precipitated mRNA and dissolving the captured mRNA into a solution to obtain purified mRNA.
Jonathan Abysalh, Jorel E. Vargas, Frank DeRosa, Anusha Dias
Filed: 8 Nov 19
Utility
Pumpless Encapsulation of Messenger Rna
9 Dec 21
The present invention provides, among other things, a process of encapsulating messenger RNA (mRNA) in liposomes comprising a. providing a first stream comprising an mRNA solution at a first controlled flow rate, b. providing a second stream comprising a lipid solution at a second controlled flow rate, and c. mixing the first stream and the second stream to form mRNA-encapsulated liposomes, wherein the first controlled flow rate and the second controlled flow rate are achieved without use of a pump.
Shrirang Karve, Priyal Patel, Natalia Vargas Montoya, Frank DeRosa, Michael Heartlein
Filed: 18 Oct 19
Utility
Dry Powder Formulations for Messenger RNA
9 Dec 21
The present invention provides stable, dry powder messenger RNA formulations for therapeutic use, and methods of making and using the same.
Shrirang Karve, Frank DeRosa, Michael Heartlein, Zarna Patel, Ashish Sarode
Filed: 23 Jul 19
Utility
Biodegradable Lipids for Delivery of Nucleic Acids
2 Dec 21
Frank DeRosa, Michael Heartlein
Filed: 5 Jan 21
Utility
Quantitative Assessment for Cap Efficiency of Messenger Rna
2 Dec 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: 19 May 21
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
Lipid Nanoparticle Formulations for mRNA Delivery
18 Nov 21
The present invention provides, among other things, methods of encapsulating messenger RNA in lipid nanoparticles without the use of flammable solvents, and compositions produced by these methods, for mRNA delivery in therapeutic use.
Shrirang Karve, Frank DeRosa, Natalia Vargas Montoya, Asad Khanmohammed
Filed: 14 May 21
Utility
Ice-based Lipid Nanoparticle Formulation for Delivery of Mrna
18 Nov 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: 23 Apr 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
Methods for Purification of Messenger Rna
21 Oct 21
The present invention provides, among other things, methods of purifying messenger RNA (mRNA) including the steps of subjecting an impure preparation comprising in vitro synthesized rnRNA to a denaturing condition, and purifying the rnRNA from the impure preparation from step (a) by tangential flow filtration, wherein the mRNA purified from step (b) is substantially free of prematurely aborted RNA sequences and/or enzyme reagents used in in vitro synthesis.
Michael Heartlein, Frank DeRosa, Anusha Dias, Shrirang Karve
Filed: 24 Nov 20