Abstract: Provided are methods for delivering lipid nanoparticles (LNPs) to a lung cell of a subject suffering from or at risk for primary ciliary dyskinesia (PCD), wherein the method comprises nebulizing a liquid pharmaceutical composition to generate an aerosolized pharmaceutical composition, and administering the aerosolized pharmaceutical composition to the subject.

Abstract: Provided are methods for delivering lipid nanoparticles (LNPs) to a lung cell of a subject suffering from or at risk for primary ciliary dyskinesia (PCD), wherein the method comprises nebulizing a liquid pharmaceutical composition to generate an aerosolized pharmaceutical composition, and administering the aerosolized pharmaceutical composition to the subject.

Abstract: Provided are methods for delivering lipid nanoparticles (LNPs) to a lung cell of a subject suffering from or at risk for cystic fibrosis (CF), wherein the method comprises nebulizing a liquid pharmaceutical composition to generate an aerosolized pharmaceutical composition, and administering the aerosolized pharmaceutical composition to the subject, wherein the LNPs comprise mRNA encoding a Cystic Fibrosis Transmembrane conductance Regulator (CFTR) protein.

Abstract: Provided are methods for delivering lipid nanoparticles (LNPs) to a lung cell of a subject suffering from or at risk for primary ciliary dyskinesia (PCD), wherein the method comprises nebulizing a liquid pharmaceutical composition to generate an aerosolized pharmaceutical composition, and administering the aerosolized pharmaceutical composition to the subject.

Abstract: A pH gradient mobile phase preparation kit is described. The preparation kit includes a first component and a second component. The first component includes a first reagent separate from a second reagent. The first reagent, the second reagent, and a solvent together provide a volatile buffer solution having a low pH. The second component includes a base. The base mixed in the solvent provide a volatile base solution having a high pH. A method of separating and detecting one or more analytes comprised in a matrix of components in a sample, using the kit is also described.

Abstract: A method of separating a liquid sample containing viral vectors includes flowing the liquid sample into an anion exchange column. A first viral vector contains a targeted genetic material and a second viral vector contains essentially no genetic material or a non-targeted genetic material, wherein the targeted genetic material is different than the non-targeted genetic material. A mobile phase can be flowed into the anion exchange column, wherein the mobile phase includes a buffer solution A and a buffer solution B. The buffer solutions A and B both include volatile buffer salts. The first viral vector and the second viral vector can be separated so that the first viral vector and the second viral vector elute off the anion exchange column at different times and then be detected.

Abstract: A method of separating a liquid sample containing viral vectors includes flowing the liquid sample into an anion exchange column. A first viral vector contains a targeted genetic material and a second viral vector contains essentially no genetic material or a non-targeted genetic material, wherein the targeted genetic material is different than the non-targeted genetic material. A mobile phase can be flowed into the anion exchange column, wherein the mobile phase includes a buffer solution A and a buffer solution B. The buffer solutions A and B both include volatile buffer salts. The first viral vector and the second viral vector can be separated so that the first viral vector and the second viral vector elute off the anion exchange column at different times and then be detected.

Abstract: A plurality of chromatograms is run using a salt gradient with a range of constant pH values or using a pH gradient with a range of constant salt concentrations. A chromatography optimization algorithm can be used to identify at least one salt gradient chromatogram or at least one pH gradient chromatogram to establish optimized buffer conditions. The chromatography optimization algorithm can include a total number of peaks algorithm, a peak-to-valley algorithm, and/or a peak capacity algorithm.

Abstract: A plurality of chromatograms is run using a salt gradient with a range of constant pH values or using a pH gradient with a range of constant salt concentrations. A chromatography optimization algorithm can be used to identify at least one salt gradient chromatogram or at least one pH gradient chromatogram to establish optimized buffer conditions. The chromatography optimization algorithm can include a total number of peaks algorithm, a peak-to-valley algorithm, and/or a peak capacity algorithm.