The present disclosure relates to a cell processing container for use in one or more unit operations in cell and/or gene therapy manufacture, the container having a base section, a top section arranged substantially in parallel with the base section and a wall element arranged between the top section and the base section and defining an internal lumen of the container, in which the wall element of the cell culture container preferably is compressible with respect to the top and base section and the wall element of the cell culture container is composed of a flexible material, wherein the cell processing container comprises at least one sterile connector end configured to operatively couple with a further sterile connector end to form a sterile connector between the cell processing container and a further container to which the cell processing container is to be fluidly connected.

The present disclosure relates method for forming polyplexes, which find use in gene therapy applications as safe and non-toxic nucleic acid transfection agents.

Methods for the treatment of disorders associated with mitochondrial dysfunction, including rare, inborn errors of metabolism caused by genetic mutations; neurodegenerative disease; diabetes; and aging and age-associated decline. Generally, the methods include administering a therapeutically effective amount of one or more treatments that (i) induce hemoglobin “left-shifting” and/or (ii) induce anemia, as described herein, to a subject who is in need of, or who has been determined to be in need of, such treatment.

The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid.

Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.

This application provides improved methods of genome editing. Cas9 molecules can be used to create a break in a genomic region of interest. To increase the likelihood that the break is repaired by HDR (homology-directed repair), the cell can be contacted with molecules that bring a template nucleic acid in close proximity to the break, under conditions that allow the cell to repair the break using the template nucleic acid.

A cell processing device for use in performing one or more unit processes in one or more of cell or gene therapy manufacturing, comprising a cell processing platform fluidly coupled to at least one auxiliary container and to at least one primary container, the cell processing platform comprising a body portion comprising at least one fluid inlet fluidly connected to a fluid outlet, and an auxiliary container port fluidly coupled to the at least one fluid inlet of the body portion, wherein the at least one auxiliary container is received in sealing engagement with the auxiliary container port such that the auxiliary container lumen is fluidly connected with the at least one fluid inlet of the body portion, and a primary container is received in sealingly engagement with the primary container port such that the primary container lumen is fluidly connected with the fluid outlet of the body portion.

The present disclosure relates to a cell processing platform for use in one or more unit operations in cell and/or gene therapy manufacture, the platform comprising a body portion comprising at least one fluid inlet fluidly connected to a fluid outlet, and an auxiliary container port fluidly coupled to the at least one fluid inlet of the body portion, wherein the auxiliary container port is configured and arranged to receive and sealingly engage with an auxiliary container and to fluidly connect the auxiliary container lumen with the at least one fluid inlet of the body portion, and a primary container port configured and arranged to sealingly engage with a primary container and to fluidly connect the primary container lumen with the fluid outlet of the body portion.

The invention describes a method for obtaining purified recombinant Adeno-Associated Virus particles (rAAV), comprising the steps of: a) performing a depth filtration of a starting material previously obtained from cells producing rAAV particles, the said starting material being selected in a group comprising a cell lysate and a culture supernatant, whereby a rAAV-containing clarified composition is provided; b) submitting the rAAV-containing clarified composition to an affinity purification step, whereby a first rAAV enriched composition is provided; c) submitting the first rAAV enriched composition at least once to: c1) a step of anion-exchange chromatography on a chromatographic support wherein elution is performed by using a salt gradient, preferably a linear salt gradient, and wherein the rAAV-containing fraction is collected, whereby a second rAAV enriched composition is provided; or c2) a step of density gradient centrifugation, wherein the rAAV-containing fraction is collected, whereby a second rAAV enriched composition is provided; d) submitting the second rAAV enriched composition to a step of tangential flow filtration, whereby purified recombinant Adeno-Associated Virus particles (rAAV) are provided.

Methods for preparing and purifying viral particles, and compositions and uses comprising the same, are provided.