Abstract: The present disclosure provides stable lyophilized pharmaceutical compositions comprising recombinant adeno-associated virus (rAAV) particles. In certain embodiments, the compositions contain, in addition to the rAAV particles, a buffer, one or more salts, a surfactant, a bulking agent, and a sugar. The pharmaceutical compositions of the present disclosure exhibit a substantial degree of rAAV stability upon stress and storage.

Abstract: The present invention provides stable pharmaceutical formulations comprising a human antibody that specifically binds to human angiopoietin-like protein 3 (ANGPTL3). The formulations may contain, in addition to an anti-ANGPTL3 antibody, a buffer; an organic cosolvent; at least one viscosity modifier, and optionally at least one amino acid. The pharmaceutical formulations of the present invention can be administered via intravenous infusion or subcutaneously and exhibit a substantial degree of antibody stability after storage for several months.

Abstract: Methods for characterizing DNA impurities in adeno-associated virus (AAV) samples or biopharmaceuticals are provided including the use of size exclusion chromatography and spectrophotometry. Methods and compositions are also provided to minimize leakage of packed DNA from AAV vector including the use of excipients, such as a sugar, an amino acid, a surfactant, or polyols.

Abstract: Methods for determining the relative abundance of intact adeno-associated virus (AAV) capsid components in a sample of recombinant AAV particles are disclosed. In embodiments, the methods include a system regeneration process that minimizes or eliminates the presence of ghost peaks to maximize analytical accuracy and ensure product quality and consistency.

Abstract: The present invention provides stable pharmaceutical formulations comprising a human antibody that specifically binds to human angiopoietin-like protein 3 (ANGPTL3). The formulations may contain, in addition to an anti-ANGPTL3 antibody, a buffer; an organic cosolvent; at least one viscosity modifier, and optionally at least one amino acid. The pharmaceutical formulations of the present invention can be administered via intravenous infusion or subcutaneously and exhibit a substantial degree of antibody stability after storage for several months.

Abstract: Methods for characterizing DNA impurities in adeno-associated virus (AAV) samples or biopharmaceuticals are provided including the use of size exclusion chromatography and spectrophotometry. Methods and compositions are also provided to minimize leakage of packed DNA from AAV vector including the use of excipients, such as a sugar, an amino acid, a surfactant, or polyols.

Abstract: Methods and systems for identifying capsid viral proteins in a sample containing viral vectors are provided, including determining the ratio of the capsid viral proteins of adeno-associated virus. The methods and systems comprise denaturing the capsid viral proteins in the sample, labelling the denatured capsid viral proteins with a lysine-conjugation dye, generating a separation profile of the denatured/labelled capsid viral proteins using microchip capillary electrophoresis, quantifying levels of the capsid viral proteins based on the separation profile, determining a quantification ratio of the capsid viral proteins based on the separation profile, and normalizing the quantification ratio based on lysine contents of the capsid viral proteins.

Abstract: The present disclosure provides stable pharmaceutical formulations comprising a human antibody that specifically binds to Ebola Virus (EBOV). In certain embodiments, the formulations contain, in addition to an anti-EBOV antibody, a buffer, an amino acid, a non-ionic surfactant, and a stabilizer. The pharmaceutical formulations of the present disclosure exhibit a substantial degree of antibody stability upon stress, for example, agitation during transport, and storage, for example, storage at temperatures greater than 40° C.

Abstract: The present disclosure provides stable pharmaceutical formulations comprising a human antibody that specifically binds to Ebola Virus (EBOV). In certain embodiments, the formulations contain, in addition to an anti-EBOV antibody, a buffer, an amino acid, a non-ionic surfactant, and a stabilizer. The pharmaceutical formulations of the present disclosure exhibit a substantial degree of antibody stability upon stress, for example, agitation during transport, and storage, for example, storage at temperatures greater than 40° C.

Abstract: Methods and systems for identifying capsid viral proteins in a sample containing viral vectors are provided, including determining the ratio of the capsid viral proteins of adeno-associated virus. The methods and systems comprise denaturing the capsid viral proteins in the sample, labelling the denatured capsid viral proteins with a lysine-conjugation dye, generating a separation profile of the denatured/labelled capsid viral proteins using microchip capillary electrophoresis, quantifying levels of the capsid viral proteins based on the separation profile, determining a quantification ratio of the capsid viral proteins based on the separation profile, and normalizing the quantification ratio based on lysine contents of the capsid viral proteins.

Abstract: The present inventions relate to improved and enhanced chromatographic systems for analyzing DNA, such as DNA released from AAV virions. The present inventions avoid past problems through the use of larger pore size media, different column flow rates and buffers comprising surfactants and increased concentrations of inorganic salts, and typically have column pressures of about 1450 to about 1550 psi.

Abstract: Disclosed herein are methods for quantification of viral genomes present inside viral capsids using a droplet digital PCR assay. In embodiments, the methods include preparing a sample of viral genome by sample dilution, treating the diluted sample of viral genome with DNase I/Proteinase K, analyzing the sample of viral genome by ddPCR assay, and determining a viral titer based on analysis of data obtained from the ddPCR assay. In examples, the ddPCR assay in accordance with the present disclosure produces a variability rate of no more than 5%.

Abstract: The present disclosure relates to, inter alia, a method of quantitating an amount of an antibody molecule from a mixture comprising two or more antibody molecules, comprising separating each of the two or more antibody molecules from the mixture by hydrophobic interaction chromatography high performance liquid chromatography (HIC-HPLC) and quantitating an amount of each antibody molecule, wherein the molecular weight of each antibody molecule is within 15 kDa of any other antibody molecule in the mixture and either each antibody molecule is different from another antibody molecule in the mixture by more than about 0.25 unit on the Kyte & Doolittle hydropathy scale or each of the antibody molecules when nm alone on HIC-HPLC elutes at distinct run time with little overlap from the other antibody molecules in the mixture, or both.

Abstract: The present disclosure relates to, inter alia, a method of quantitating an amount of an antibody molecule from a mixture comprising two or more antibody molecules, comprising separating each of the two or more antibody molecules from the mixture by hydrophobic interaction chromatography high performance liquid chromatography (HIC-HPLC) and quantitating an amount of each antibody molecule, wherein the molecular weight of each antibody molecule is within 15 kDa of any other antibody molecule in the mixture and either each antibody molecule is different from another antibody molecule in the mixture by more than about 0.25 unit on the Kyte & Doolittle hydropathy scale or each of the antibody molecules when nm alone on HIC-HPLC elutes at distinct run time with little overlap from the other antibody molecules in the mixture, or both.

Abstract: The present disclosure provides stable lyophilized pharmaceutical compositions comprising recombinant adeno-associated virus (rAAV) particles. In certain embodiments, the compositions contain, in addition to the rAAV particles, a buffer, one or more salts, a surfactant, a bulking agent, and a sugar. The pharmaceutical compositions of the present disclosure exhibit a substantial degree of rAAV stability upon stress and storage.

Abstract: The present disclosure relates to AM-14 pharmaceutical formulations and therapeutic dosing regimens for the treatment of disease.

Abstract: The present invention provides stable pharmaceutical formulations comprising a human antibody that specifically binds to human programmed death-1 protein (PD-1). In certain embodiments, the formulations contain, in addition to an anti-PD-1 antibody, a buffer, an amino acid, a non-ionic surfactant, and a sugar. The pharmaceutical formulations of the present invention exhibit a substantial degree of antibody stability upon stress and storage.

Abstract: The present disclosure relates to, inter alia, a method of quantitating an amount of an antibody molecule from a mixture comprising two or more antibody molecules, comprising separating each of the two or more antibody molecules from the mixture by hydrophobic interaction chromatography high performance liquid chromatography (HIC-HPLC) and quantitating an amount of each antibody molecule, wherein the molecular weight of each antibody molecule is within 15 kDa of any other antibody molecule in the mixture and either each antibody molecule is different from another antibody molecule in the mixture by more than about 0.25 unit on the Kyte & Doolittle hydropathy scale or each of the antibody molecules when run alone on HIC-HPLC elutes at distinct run time with little overlap from the other antibody molecules in the mixture, or both.

Abstract: The present invention provides stable pharmaceutical formulations comprising a human antibody that specifically binds to human programmed death-1 protein (PD-1). In certain embodiments, the formulations contain, in addition to an anti-PD-1 antibody, a buffer, an amino acid, a non-ionic surfactant, and a sugar. The pharmaceutical formulations of the present invention exhibit a substantial degree of antibody stability upon stress and storage.

Abstract: The present invention provides pharmaceutical formulations comprising an antibody that specifically binds to human interleukin-33 (hIL-33). The formulations may contain, in addition to an anti-IL-33 antibody, a buffer, at least one amino acid, at least one sugar, or at least one non-ionic surfactant. The pharmaceutical formulations of the present invention exhibit a substantial degree of antibody stability after storage for several months and after being subjected to thermal and other physical stresses.