140 patents
Utility
Large-scale Synthesis of Messenger Rna
18 Jan 24
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.
Jonathan Abysalh, Daniel Crawford, Frank DeRosa, Anusha Dias, Michael Heartlein
Filed: 14 Dec 22
Utility
Mrna Therapy for Pompe Disease
11 Jan 24
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: 8 Jun 23
Utility
Improved Processes for In Vitro Transcription of Messenger Rna
21 Dec 23
The present invention provides methods for preparing optimized DNA sequences as templates for in vitro transcription of mRNA.
Dustin Cooper, Frank DeRosa, Anusha Dias, Jeffrey S. Dubins, Xiaobo Gu, Khang Anh Tran
Filed: 18 Feb 21
Utility
Improved Process and Formulation of Lipid Nanoparticles
23 Nov 23
The present invention provides an improved process for preparing a stable lyophilized composition comprising lipid nanoparticles encapsulating mRNA, which results in high mRNA integrity suitable for both short- and long-term storage for therapeutic use.
Frank DEROSA, Hardip R. GOPANI, Shrirang KARVE
Filed: 6 Oct 21
Utility
Methods and Compositions for Delivering Mrna Coded Antibodies
9 Nov 23
The present invention provides, among other things, method of treating an immune disease in a subject, the method comprising: administering to a subject in need thereof one or more mRNAs encoding a heavy chain and a light chain of an antibody that binds to a protein target selected from IL-4, IL-5, IL-6, IL-9, IL-13, IL-25, or IL-33, IL-4R, IL-5R, IL-6R, IL-9R, IL-13R, IL-25R or IL-33R, and wherein the one or more mRNAs are encapsulated in a lipid nanoparticle (LNP).
Lianne BOEGLIN, Christopher GUENARD, Khang Anh TRAN, Anusha DIAS, Frank DEROSA
Filed: 14 Jul 23
Utility
Tes-based Cationic Lipids
9 Nov 23
Shrirang KARVE, Frank DEROSA
Filed: 22 Sep 21
Utility
Piperazine-based Cationic Lipids
9 Nov 23
Saswata KARMAKAR, Ramesh DASARI, Ryan LANDIS
Filed: 23 Sep 21
Utility
Methods for Purification of Messenger Rna
9 Nov 23
The present invention relates, in part, to methods, systems and processes for large-scale purification of mRNA using a filtering centrifuge operating at lower gravitational forces.
Jonathan ABYSALH, Frank DEROSA, Cameron SMITH, Jorel E. VARGAS
Filed: 1 Oct 21
Utility
Lipid Nanoparticle Compositions and Methods for Mrna Delivery
19 Oct 23
Disclosed herein are compositions and methods for modulating the production of a protein in a target cell.
Frank DeRosa, Braydon Charles Guild, Michael Heartlein
Filed: 3 Apr 23
Utility
CFTR Mrna Compositions and Related Methods and Uses
28 Sep 23
Materials, formulations, production methods, and methods for delivery of CFTR mRNA for induction of CFTR expression, including in the mammalian lung are provided.
Michael Heartlein, Braydon Charles Guild, Frank DeRosa, Carsten Rudolph, Christian Plank, Lianne Smith
Filed: 24 Oct 22
Utility
Lipid Nanoparticle Compositions and Methods for Mrna Delivery
21 Sep 23
Disclosed herein are compositions and methods for modulating the production of a protein in a target cell.
Frank DeRosa, Braydon Charles Guild, Michael Heartlein
Filed: 3 Apr 23
Utility
Lipid Nanoparticle Compositions and Methods for Mrna Delivery
14 Sep 23
Disclosed herein are compositions and methods for modulating the production of a protein in a target cell.
Frank DeRosa, Braydon Charles Guild, Michael Heartlein
Filed: 3 Apr 23
Utility
Lipid Nanoparticle Compositions and Methods for Mrna Delivery
14 Sep 23
Disclosed herein are compositions and methods for modulating the production of a protein in a target cell.
Frank DeRosa, Braydon Charles Guild, Michael Heartlein
Filed: 3 Apr 23
Utility
Generation of Optimized Nucleotide Sequences
3 Aug 23
A method for generated an optimized nucleotide sequence is provided.
Khang Anh TRAN, Anusha DIAS, Frank DEROSA
Filed: 7 May 21
Utility
Composition and Methods for Treatment of Primary Ciliary Dyskinesia
20 Jul 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: 6 Dec 22
Utility
Peg Lipidoid Compounds
20 Jul 23
The compounds disclosed herein (e.g., compounds having a structure according to Formula (I), (II), (III), (IV), and (V)) are polymers wherein an organic polymeric segment (e.g., a polyethylene glycol (PEG) group) comprises covalent attachments to two or more lipid substructures, and each lipid substructure independently comprises a hydrophobic moiety and a hydrophilic moiety.
Shrirang KARVE, Frank DEROSA, Ashish SARODE
Filed: 13 May 21
Utility
Phenolic Acid Lipid Based Cationic Lipids
18 May 23
Shrirang KARVE, Frank DEROSA, Apiwat WANGWEERAWONG, Saswata KARMAKAR
Filed: 31 Mar 21
Utility
Composition and Methods for Treatment of Methylmalonic Acidemia
11 May 23
The present invention provides, among other things, methods and compositions for treating methylmalonic academia (MMA) based on mRNA therapy.
Lianne Boeglin, Christian Cobaugh, Frank DeRosa, Michael Heartlein, Kim Askew
Filed: 13 Sep 19
Utility
Tricine and Citric Acid Lipids
23 Mar 23
Shrirang Karve, Frank DeRosa
Filed: 19 Jun 20
Utility
2,5-DIOXOPIPERAZINE Lipids with Intercalated Ester, Thioester, Disulfide and Anhydride Moieities
9 Mar 23
Yi Zhang, Frank DeRosa, Shrirang Karve, Michael Heartlein, Saswata Karmakar, Ryan Landis
Filed: 7 Nov 19