212 patents
Page 4 of 11
Utility
Di-thioester Cationic Lipids
18 Aug 22
The present invention provides, in part, di-thioester cationic lipid compounds or a pharmaceutically acceptable salt thereof.
Shrirang Karve, Frank DeRosa
Filed: 1 May 20
Utility
COMPOSITIONS AND METHODS FOR mRNA DELIVERY
11 Aug 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: 11 Sep 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
Thioester Cationic Lipids
28 Jul 22
Shrirang Karve, Yi Zhang, Frank DeRosa
Filed: 21 Apr 20
Utility
Pulmonary Delivery of MRNA to Non-lung Target Cells
21 Jul 22
Compositions comprising mRNA formulated for pulmonary administration and related methods 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: 29 Jul 21
Utility
Macrocyclic Lipids
21 Jul 22
Yi Zhang, Shrirang Karve, Frank DeRosa, Michael Heartlein
Filed: 29 May 20
Utility
Encapsulation of Messenger Rna
21 Jul 22
The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation.
Frank DeRosa, Shrirang Karve, Michael Heartlein
Filed: 1 Dec 21
Utility
Nucleic Acid Conjugates and Uses Thereof
14 Jul 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: 16 Dec 21
Utility
Improved Process of Preparing Mrna-loaded Lipid Nanoparticles
14 Jul 22
The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation.
Shrirang Karve, Frank DeRosa, Michael Hearlein, Ashish Sarode, Zarna Patel, Rebecca L. Ball, Natalia Vargas Montoya, Priyal Patel, Asad Khanmohammed
Filed: 14 May 20
Utility
Mrna Therapy for Argininosuccinate Synthetase Deficiency
14 Jul 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 Boeglin
Filed: 6 Dec 21
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
Cleavable Lipids
30 Jun 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: 20 Dec 21
Utility
Novel Codon-Optimized CFTR MRNA
23 Jun 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: 6 Jan 22
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
Cystine Cationic Lipids
9 Jun 22
Shrirang Karve, Saswata Karmakar, Frank DeRosa
Filed: 17 Apr 20
Utility
Methods for Purification of Messenger Rna
2 Jun 22
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: 11 Oct 21
Utility
Compositions for Delivery of Codon-optimized Mrna
26 May 22
The present invention provides, among other things, improved pharmaceutical compositions comprising codon-optimized mRNA encoding a peptide or polypeptide encapsulated in a lipid nanoparticle comprising one or more of the cationic lipids that are particularly effective for pulmonary delivery.
John Androsavich, Lianne Boeglin, Shraddha Sharma, Gang Sun, Neha Kaushal, Shrirang Karve
Filed: 9 Nov 21
Utility
TREATMENT OF CYSTIC FIBROSIS BY DELIVERY OF CODON-OPTIMIZED mRNA ENCODING CFTR
26 May 22
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: 1 Oct 21
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