B cells for in vivo delivery of therapeutic agents and dosages thereof
OwnerMatthew Rein Scholz, Eric J. HERBIG +3inventors
InventorsMatthew Rein Scholz, Eric J. HERBIG, R. Scott MCIVOR, Rian DE LAAT, Erik Olson
Life of this patent
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Abstract
The present invention relates to methods for administering autologous and/or allogeneic B cells genetically modified to produce a therapeutic agent, such as a therapeutic protein. Specifically disclosed are methods for administering a single, maximally effective dose of genetically modified B cells and for administering multiple doses of genetically modified B cells. The compositions and methods disclosed herein are useful for the long-term, in vivo delivery of a therapeutic agent.
Claims27
-21. (canceled)
The method of claim 70, wherein the genetically modified B cells comprise a polynucleotide having a sequence that is identical to SEQ ID NO:
- 1.
The method of claim 70, wherein the genetically modified B cells comprise a polynucleotide having a sequence that is at least about 85% identical to SEQ ID NO:
- 1, or at least about 90%, 95%, 96%, 97%, 98%, 99%, or greater than 99% identical to SEQ ID NO:
- 1.
-51. (canceled)
A method for treating MPS I in a subject comprising administering two or more sequential doses of B cells genetically modified to produce IDUA to the subject with MPS I, wherein administration of the genetically modified B cells results in the reduction of glycosaminoglycans (GAGs) in diverse tissues of the subject.
(canceled)
The method of claim 70, wherein the genetically modified B cells comprise a polynucleotide encoding a pKT2/EEK-IDUA-DHFR bifunctional transgene.
-69. (canceled)
A method of administering genetically modified B cells to a subject to enable synergistic in vivo production of a therapeutic agent comprising:
- determining an optimal single-dose concentration of the modified B cells for inducing the greatest in vivo production of the therapeutic agent;
- decreasing the optimal single-dose concentration of the modified B cells to obtain a sub-optimal single-dose concentration of the modified B cells;
- and
- administering two or more doses of the sub-optimal single-dose concentration of the modified B cells to the subject.
-91. (canceled)
A composition comprising a population of genetically modified B cells that have been engineered to produce a therapeutic agent, wherein the genetically modified B cells are at optimal migratory capacity, wherein at least one genetically modified B cell out of the population of genetically modified B cells comprised in the composition migrates to one or more tissue selected from the group consisting of bone marrow, intestine, muscle, spleen, kidney, heart, liver, lung and brain when the composition of genetically modified B cells is administered to a subject.
-113. (canceled)
-150. (canceled)
Dependencies are inferred from the claim text.