Compositions and methods to modify cells for therapeutic objectives

Abstract

The present disclosure provides compositions and methods that rapidly and selectively modify cells of the immune system to achieve therapeutic objectives. The methods can be practiced in vivo and any cell type that expresses a known marker can be targeted for a therapeutic objective. The present disclosure provides compositions and methods that rapidly and selectively modify cells of the immune system to achieve therapeutic objectives. The methods can be practiced in vivo and any cell type that expresses or is associated with a known marker can be targeted for a therapeutic objective by the modified cell.

Description

CROSS REFERENCE TO RELATED APPLICATIONS[0001]This application claims priority to U.S. Provisional Patent Application No. 61 / 785,907, filed Mar. 14, 2013, which is incorporated herein by reference in its entirety.FIELD OF THE DISCLOSURE[0002]The present disclosure provides compositions and methods that rapidly and selectively modify cells of the immune system to achieve therapeutic objectives. The methods can be practiced in vivo and any cell type that expresses or is associated with a known marker can be targeted for a therapeutic objective by the modified cell.BACKGROUND OF THE DISCLOSURE[0003]One of the primary goals of clinical health research is to develop compositions and methods that rapidly and selectively direct cells of the immune system to achieve therapeutic objectives. For example, vaccines are used to prime the immune system to target antigens associated with unwanted cells. The biological processes underlying conventional vaccines, however, can render them ineffective ...

AI Technical Summary

Benefits of technology

[0006]The present disclosure provides compositions and methods that can rapidly and selectively direct cells of the immune system to achieve therapeutic objectives. In particular embodiments, the compositions and methods modify cells of the immune system, such as T cells or natural killer (NK) cells, to target and destroy unwanted cell types. In other embodiments, the compositions and methods modify cells of the immune system, such as monocytes / macrophages, to target and destroy viruses before they infect cells and / or to target bacteria or fungus. In further embodiments, the compositions and methods modify cells of the immune system, such as B cells, to produce and release antibodies, such as broadly-neutralizing antibodies. In additional embodiments, the compositions and methods modify cells of the immune system, such as immunosuppressive regulatory T cells (TREG) to target and protect, rather than destroy, cell types. Compositions and methods disclosed herein can also be used to modify stem cells to achieve therapeutic objectives.

Problems solved by technology

The biological processes underlying conventional vaccines, however, can render them ineffective against many unwanted cells based on, among other factors, the time it takes to prime the immune system, the amount or degree to which the natural immune system can be primed against certain unwanted cell types and over time, the depletion of immune system resources.

As examples, conventional vaccine approaches can be ineffective against cancer cells and cells affected by certain infectious diseases.

The immune response using this approach, however, requires months to mature and during this time, cancers can significantly progress and become fatal.

Thus, conventional vaccines do not provide an adequate method to target and destroy unwanted cancer cells.

However, T-cell transfer therapies are also time and labor-intense and must be personalized for each patient in cell production facilities, which are available only at a few highly specialized cancer centers worldwide.

Similar issues are encountered with a number of other unwanted cell types.

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