Abstract: Pharmaceutical compositions comprising an mRNA-loaded nanoparticle, wherein the mRNA is an in vitro transcribed mRNA and has a coding sequence at least 80% identical to SEQ ID NO: 3, and wherein the mRNA encodes a human cystic fibrosis transmembrane conductance regulator (CFTR) protein comprising the amino acid sequence of SEQ ID NO:1 are provided. The present invention is particularly useful for treating cystic fibrosis.
Type:
Grant
Filed:
October 6, 2015
Date of Patent:
July 25, 2017
Assignees:
RaNA Therapeutics, Inc., Ethris GmbH
Inventors:
Michael Heartlein, Braydon Charles Guild, Frank DeRosa, Carsten Rudolph, Christian Plank, Lianne Smith
Abstract: Described is a conjugate of agent complex and at least one target-finding ligand, where the agent complex comprises an agent encapsulated by an encapsulation material and where the target-finding ligand is a prostacyclin analog, and the use of the conjugate.
Abstract: Materials, formulations, production methods, and methods for delivery of CFTR mRNA for induction of CFTR expression, including in the mammalian lung are provided. The present invention is particularly useful for treating cystic fibrosis.
Type:
Grant
Filed:
June 17, 2014
Date of Patent:
November 10, 2015
Assignees:
Shire Human Genetic Therapies, Inc., Ethris GMBH
Inventors:
Michael Heartlein, Braydon Charles Guild, Frank DeRosa, Carsten Rudolph, Christian Plank, Lianne Smith
Abstract: The invention relates to a polyribonucleotide with a sequence that codes a protein or protein fragment, wherein the polyribonucleotide comprises a combination of unmodified and modified nucleotides, wherein 5 to 50% of the uridine nucleotides and 5 to 50% of the cytidine nucleotides are modified uridine nucleotides or modified cytidine nucleotides.
Abstract: The invention relates to a polyribonucleotide with a sequence that codes a protein or protein fragment, wherein the polyribonucleotide comprises a combination of unmodified and modified nucleotides, wherein 5 to 50% of the uridine nucleotides and 5 to 50% of the cytidin nucleotides are modified uridine nucleotides or modified cytidin nucleotides.
Abstract: The invention relates to a polyribonucleotide with a sequence that codes a protein or protein fragment, wherein the polyribonucleotide comprises a combination of unmodified and modified nucleotides, wherein 5 to 50% of the uridine nucleotides and 5 to 50% of the cytidin nucleotides are modified uridine nucleotides or modified cytidin nucleotides.
Abstract: Described is a conjugate of agent complex and at least one target-finding ligand, where the agent complex comprises an agent encapsulated by an encapsulation material and where the target-finding ligand is a prostacyclin analog, and the use of the conjugate.
Abstract: Described is a conjugate of agent complex and at least one target-finding ligand, where the agent complex comprises an agent encapsulated by an encapsulation material and where the target-finding ligand is a prostacyclin analog, and the use of the conjugate.
Abstract: A spray system for production of a matrix formed in situ is described, which comprises at least one lipophilic component comprising at least one polymer based on glycolic acid and lactic acid and at least one biocompatible solvent having an X log P3 value of less than 0.2, and at least one hydrophilic component, wherein the at least two components are present separately from each other prior to use and are not mixed until or in the course of spraying, with components forming a film when sprayed onto human tissue.
Abstract: The present invention relates to aerosols containing magnetic particles, wherein the aerosols comprise magnetic particles and a pharmaceutical active agent. The invention furthermore relates to the use of such aerosols containing magnetic particles for directed magnetic field-guided transfer of the active agents contained therein in aerosol therapy.
Abstract: Described is a conjugate of agent complex and at least one target-finding ligand, where the agent complex comprises an agent encapsulated by an encapsulation material and where the target-finding ligand is a prostacyclin analog, and the use of the conjugate.
Abstract: The invention relates to a polyribonucleotide with a sequence that codes a protein or protein fragment, wherein the polyribonucleotide comprises a combination of unmodified and modified nucleotides, wherein 5 to 50% of the uridine nucleotides and 5 to 50% of the cytidin nucleotides are modified uridine nucleotides or modified cytidin nucleotides.