212 patents
Page 9 of 11
Utility
Ribonucleic Acids with 4'-THIO-MODIFIED Nucleotides and Related Methods
14 Jan 21
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
Lipid nanoparticle compositions and methods for mRNA delivery
12 Jan 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 Jul 20
Utility
Methods for Purification of Messenger Rna
6 Jan 21
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 Vargas, Cameron M. Smith
Filed: 14 May 20
Utility
Methods for purification of messenger RNA
28 Dec 20
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) 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: 25 Mar 18
Utility
Synergistic Enhancement of the Delivery of Nucleic Acids via Blended Formulations
16 Dec 20
Disclosed herein are pharmaceutical compositions that comprise “blends” of lipid nanoparticles and related methods of using such blended compositions to deliver polynucleotides to one or more target cells, tissues or organs.
Frank DeRosa, Lianne Smith, Michael Heartlein, Braydon Charles Guild
Filed: 30 Mar 20
Utility
Messenger RNA therapy for treatment of articular disease
14 Dec 20
The present invention provides, among other things, a method of intra-articular delivery of messenger RNA (mRNA), comprising administering into a joint of a subject in need of delivery a composition comprising an mRNA encoding a protein, such that the administering of the composition results in expression of the protein encoded by the mRNA in the joint.
Frank DeRosa, Michael Heartlein
Filed: 5 Mar 18
Utility
Multimeric oligonucleotide compounds having non-nucleotide based cleavable linkers
23 Nov 20
The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker.
Eugen Uhlmann, Romesh Subramanian, Arthur M. Krieg
Filed: 7 Sep 17
Utility
Messenger RNA therapy for the treatment of ornithine transcarbamylase deficiency
16 Nov 20
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: 12 Jun 17
Utility
Compositions and methods for modulating SMN gene family expression
16 Nov 20
Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of SMN1 or SMN2.
Arthur M. Krieg, Romesh Subramanian, James McSwiggen, Jeannie T. Lee
Filed: 15 Jan 18
Utility
Ribonucleic acids with 4′-thio-modified nucleotides and related methods
2 Nov 20
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: 20 Feb 19
Utility
Lipid Nanoparticle Compositions and Methods for Mrna Delivery
28 Oct 20
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: 15 Jul 20
Utility
Methods for purification of messenger RNA
19 Oct 20
The present invention relates, in part, to methods for large-scale purification of mRNA.
Jonathan Abysalh, Daniel Crawford, Frank DeRosa, Shrirang Karve, Anusha Dias, Michael Heartlein
Filed: 26 Feb 18
Utility
Quantitative Assessment for Cap Efficiency of Messenger Rna
7 Oct 20
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: 15 Jan 20
Utility
Lipid-derived neutral nanoparticles
28 Sep 20
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: 5 Nov 18
Utility
CNS delivery of MRNA and uses thereof
21 Sep 20
The present invention provides, among other things, methods and compositions for effective delivery of messenger RNA (mRNA) to the central nervous system (CNS).
Frank DeRosa, Michael Heartlein, Shrirang Karve
Filed: 13 Aug 17
Utility
Messenger RNA therapy for the treatment of Friedreich's ataxia
21 Sep 20
The present invention provides methods and compositions of treating Friedreich's ataxia (FRDA) based on administering an mRNA encoding a frataxin protein.
Michael Heartlein, Frank DeRosa, Jonathan Cherry, Paula Lewis, Shrirang Karve, Zarna Patel, Darshan Parekh, Susan Wood, Chi-Sung Chiu, Caroline J. Woo
Filed: 18 Jun 18
Utility
Ionizable cationic lipids
7 Sep 20
Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use.
Frank DeRosa, Braydon Charles Guild, Michael Heartlein
Filed: 9 Aug 18
Utility
Poly(phosphoesters) for Delivery of Nucleic Acids
2 Sep 20
Frank DeRosa, Michael Heartlein, Shrirang Karve, Yi Zhang
Filed: 10 Jun 18
Utility
Hybrid oligonucleotides and uses thereof
31 Aug 20
Provided herein are hybrid oligonucleotides comprising a region that promotes cleavage of a nucleic acid and a region that protects a nucleic acid from exonuclease activity.
Fatih Ozsolak
Filed: 11 Feb 16
Utility
Cleavable Lipids
29 Jul 20
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: 12 Feb 20