72 patents
Utility
Methods for purification of messenger RNA
21 Nov 23
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 mRNA to a denaturing condition, and purifying the mRNA 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.
Frank DeRosa, Anusha Dias, Michael Heartlein, Shrirang Karve
Filed: 8 Jun 22
Utility
Lipid nanoparticle compositions and methods for mRNA delivery
22 Aug 23
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: 21 Dec 21
Utility
MRNA therapy for pompe disease
1 Aug 23
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 for purification of messenger RNA
4 Jul 23
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 mRNA to a denaturing condition, and purifying the mRNA 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.
Frank DeRosa, Anusha Dias, Michael Heartlein, Shrirang Karve
Filed: 8 Jun 22
Utility
Composition and methods for treatment of primary ciliary dyskinesia
24 Jan 23
The present invention provides, among other things, methods and compositions for treating primary ciliary dyskinesia (PCD) based on mRNA therapy.
Anusha Dias, Darshan Parekh, Jeffrey S. Dubins, Christian Cobaugh, Shrirang Karve, Zarna Patel, Sara J. Dunaj, Frank DeRosa, Michael Heartlein
Filed: 7 Jan 20
Utility
Large-scale synthesis of messenger RNA
24 Jan 23
The present invention provides methods for large-scale production of a composition enriched for full-length mRNA molecules using an SP6 RNA polymerase and compositions produced using such methods and uses thereof.
Anusha Dias, Daniel Crawford, Frank DeRosa, Jonathan Abysalh, Michael Heartlein
Filed: 27 Feb 18
Utility
Cationic lipids comprising a steroidal moiety
10 Jan 23
Yi Zhang, Shrirang Karve, Frank DeRosa, Michael Heartlein
Filed: 30 May 19
Utility
Lipid nanoparticle compositions and methods for MRNA delivery
10 Jan 23
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: 20 Dec 21
Utility
Poly(phosphoesters) for delivery of nucleic acids
20 Dec 22
Frank DeRosa, Michael Heartlein, Shrirang Karve, Yi Zhang
Filed: 11 Jun 18
Utility
CFTR MRNA compositions and related methods and uses
29 Nov 22
The present disclosure relates to materials, formulations, production methods, and methods for delivery of CFTR mRNA, including but not limited to chemically modified mRNA for induction of CFTR expression, including in the mammalian lung.
Michael Heartlein, Braydon Charles Guild, Frank DeRosa, Carsten Rudolph, Christian Plank, Lianne Smith
Filed: 14 Aug 19
Utility
Lipid-derived neutral nanoparticles
15 Nov 22
Disclosed herein are novel lipids and liposomal compositions prepared using such compounds and related methods of neutralizing or otherwise modifying such liposomal compositions.
Frank DeRosa, Braydon Charles Guild, Michael Heartlein
Filed: 26 Aug 20
Utility
Methods for purification of messenger RNA
27 Sep 22
The present invention provides, among other things, methods for purifying high quality messenger RNA (mRNA) suitable for clinical use, without using any caustic or flammable solvents.
Jonathan Abysalh, Frank DeRosa, Jorel E. Vargas, Cameron M. Smith
Filed: 15 May 20
Utility
Ribonucleic acids with 4′-thio-modified nucleotides and related methods
20 Sep 22
Disclosed are messenger RNA molecules and related compositions incorporating a 4′-thio modification in the furanose ring of at least one nucleotide residue, and methods of using these mRNAs to produce an encoded therapeutic protein in vivo and to treat or prevent diseases or disorders.
Frank DeRosa, Michael Heartlein
Filed: 25 Sep 20
Utility
Biodegradable lipids for delivery of nucleic acids
6 Sep 22
Frank DeRosa, Michael Heartlein
Filed: 5 Jan 21
Utility
Polyanionic delivery of nucleic acids
30 Aug 22
Michael Heartlein, Frank DeRosa, Shrirang Karve
Filed: 10 Feb 21
Utility
Subcutaneous delivery of messenger RNA
2 Aug 22
The present invention provides, among other things, methods of formulating nucleic acid-containing nanoparticles with 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: 10 Nov 17
Utility
mRNA therapy for phenylketonuria
5 Jul 22
The present invention provides, among other things, methods of treating phenylketonuria (PKU), including administering to a subject in need of treatment a composition comprising an mRNA encoding phenylalanine hydroxylase (PAH) at an effective dose and an administration interval such that at least one symptom or feature of PKU is reduced in intensity, severity, or frequency or has delayed in onset.
Frank DeRosa, Michael Heartlein, Anusha Dias
Filed: 11 Jan 19
Utility
Quantitative assessment for cap efficiency of messenger RNA
21 Jun 22
The present invention provides, among other things, methods of quantitating mRNA capping efficiency, particularly for mRNA synthesized in vitro.
Michael Heartlein, Frank DeRosa, Anusha Dias
Filed: 26 Mar 18
Utility
Process of preparing mRNA-loaded lipid nanoparticles
14 Jun 22
The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation.
Shrirang Karve, Zarna Patel, Yi Zhang, Ashish Sarode, Rebecca L. Goldman, Frank DeRosa, Michael Heartlein
Filed: 28 Aug 19
Utility
Lipid nanoparticle compositions and methods for mRNA delivery
24 May 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: 20 Dec 21