Methods of manufacturing therapeutic proteins

Inventors

Tijani, RasheedSAMPEY, Darryl BaconRobblee, JohnWei, GanHe, Chaomei

Assignees

Momenta Pharmaceuticals IncBiofactura Inc

Publication Number

US-10774353-B2

Publication Date

2020-09-15

Expiration Date

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Abstract

Disclosed herein are methods of manufacturing therapeutic proteins.

Core Innovation

The invention is based, in part, on the discovery that the combination of cholesterol-auxotrophic cells with one or more additional selection markers can be exploited in methods of manufacturing therapeutic proteins, to avoid unexpected manufacturing complications associated with cholesterol-auxotrophic cells (e.g., low productivity). The disclosure features methods of manufacturing a therapeutic protein using cholesterol-auxotrophic and glutamine-auxotrophic cells transfected with nucleic acids encoding a protein capable of restoring cholesterol biosynthesis, a protein capable of restoring glutamine biosynthesis, and a nucleic acid encoding the therapeutic protein.

Exemplary embodiments recite use of NS0 cells and describe that the therapeutic protein can be an antibody, a fusion protein, or an Fc-containing fusion protein. The disclosure specifies nucleic acids encoding 3-ketosteroid reductase (3-KSR) to restore cholesterol biosynthesis and glutamine synthetase (GS) to restore glutamine biosynthesis, and further contemplates inclusion of selectable markers such as DHFR or various antibiotic resistance genes.

The specification describes alternative vector arrangements and selection strategies, and includes embodiments combining 3-KSR selection with GS selection and an aminoglycoside antibiotic resistance gene (e.g., neomycin/neo) for single, double, or triple selection schemes. [procedural detail omitted for safety]

Claims Coverage

One independent claim is identified. The claim covers transfection-based methods using a cholesterol-auxotrophic and glutamine-auxotrophic mammalian cell with defined nucleic acid components.

Transfection of cholesterol-auxotrophic and glutamine-auxotrophic mammalian cell with specified nucleic acids

Transfecting a mammalian cell which is cholesterol-auxotrophic and glutamine-auxotrophic with (i) a nucleic acid encoding a 3-ketosteroid reductase; (ii) a nucleic acid encoding a glutamine synthetase; (iii) a nucleic acid comprising an aminoglycoside antibiotic resistance gene; and (iv) a nucleic acid encoding the therapeutic protein.

The claim coverage centers on a method of manufacturing a therapeutic protein by introducing four specified nucleic acid components into a cholesterol-auxotrophic and glutamine-auxotrophic mammalian cell, with dependent claims describing vector configurations, culture conditions, selection agents, and exemplary cell types (e.g., NS0).

Stated Advantages

Avoids unexpected manufacturing complications associated with cholesterol-auxotrophic cells, including low productivity.

Enables increased amplification and copy number of the gene of interest.

Permits production in the absence of exogenously added cholesterol, avoiding cholesterol-associated manufacturing problems related to cholesterol insolubility.

Triple-selection strategies (e.g., 3-KSR, GS, and aminoglycoside resistance) maintain cell viability post-recovery and improve productivity, with embodiments claiming extended cell viability post-recovery (up to 30 days).

Documented Applications

Manufacture of therapeutic proteins, including antibodies, fusion proteins, and Fc-containing fusion proteins.

Production of a broad range of therapeutic proteins including anticoagulants, blood factors, bone morphogenic proteins, engineered protein scaffolds, enzymes, growth factors, hormones, hormone releasing factors, interferons, interleukins, and thrombolytics.

Preparation of pharmaceutical compositions comprising one or more therapeutic proteins produced by the described methods.

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