140 patents
Page 5 of 7
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 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
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
Utility
Methods and Compositions for Messenger Rna Purification
21 Oct 21
The present invention provides, among other things, methods for purifying high quality messenger (mRNA) suitable for clinical use.
Jonathan Abysalh, Jorel Vargas, Cameron M. Smith, Joseph Parrella, Frank DeRosa
Filed: 10 Feb 21
Utility
Lipid Nanoparticle Compositions and Methods for Mrna Delivery
30 Sep 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
Processes of Preparing Mrna-loaded Lipid Nanoparticles
9 Sep 21
The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation.
Shrirang Karve, Ashish Sarode, Frank DeRosa
Filed: 25 Feb 21
Utility
PolyAnionic Delivery of Nucleic Acids
26 Aug 21
Michael Heartlein, Frank DeRosa, Shrirang Karve
Filed: 10 Feb 21
Utility
Cationic Lipids Comprising a Steroidal Moiety
22 Jul 21
Yi Zhang, Shrirang Karve, Frank DeRosa, Michael Heartlein
Filed: 30 May 19
Utility
Subcutaneous Delivery of Messenger RNA
22 Jul 21
The present invention provides, among other things, methods of formulating nucleic acid-containing nanoparticles with an mRNA encoding an enzyme to afford efficient delivery of payload to a cell or tissue of interest via subcutaneous administration.
Shrirang Karve, Frank DeRosa, Zarna Bhavsar, Michael Heartlein
Filed: 14 May 19
Utility
Phosphoester Cationic Lipids
15 Jul 21
Yi Zhang, Shrirang Karve, Frank DeRosa, Michael Heartlein
Filed: 29 May 19
Utility
Messenger Rna Therapy for the Treatment of Ornithine Transcarbamylase Deficiency
8 Jul 21
The present invention provides, among other things, methods of treating ornithine transcarbamylase (OTC) deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase (OTC) protein at an effective dose and an administration interval such that at least one symptom or feature of the OTC deficiency is reduced in intensity, severity, or frequency or has delayed onset.
Frank DeRosa, Michael Heartlein, Lianne Smith, Shrirang Karve
Filed: 10 Nov 20
Utility
Vitamin Cationic Lipids
8 Jul 21
Disclosed are cationic lipids comprising a vitamin substructure and an ionizable nitrogen-containing group.
Yi Zhang, Shrirang Karve, Saswata Karmakar, Frank DeRosa, Michael Heartlein
Filed: 29 May 19
Utility
Methods and Compositions for Delivering Mrna Coded Antibodies
8 Jul 21
The present invention provides, among other things, methods and compositions for delivering an antibody in vivo by administering to a subject in need thereof one or more mRNAs encoding a heavy chain and a light chain of an antibody, and wherein the antibody is expressed systemically in the subject.
Michael Heartlein, Frank DeRosa, Anusha Dias, Braydon Charles Guild
Filed: 18 Dec 20