US 12,685,751 B2 · Methods for gene modification of hematopoietic cells

Methods for gene modification of hematopoietic cells

AssigneesCentro de Investigaciones Energéticas, Medioambientales y Tecnológicas, O.A, M.P., Consorcio Centro de Investigación Biomédica en Red, M.P., Fundación Instituto de Investigación Sanitaria Fundación Jiménez Díaz, Spacecraft Seven, LLC

InventorsBrian Beard, Gaurav D. Shah, Juan Antonio Bueren Roncero, Jose Carlos Segovia Sanz, Paula Rio Galdo, Susana Navarro Ordonez, Elena Almarza Novoa, Oscar Quintana Bustamante, Cristina Mesa Nunez, Kenneth Law, Kinnari Patel

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Abstract

The present invention relates generally to methods for genetic modification of hematopoietic cells. In particular, the invention relates to use of Prostaglandin E2 (PGE2), poloxamer, and protamine sulfate to enhance transduction by a recombinant retroviral vector. The compositions and methods of the present disclosure are particularly suitable for gene therapy applications, including the treatment of monogenic genetic diseases and disorders.

Claims27

Dependent claims

A method of ex vivo genetic modification of hematopoietic cells comprising pre-stimulating the hematopoietic cells with a recombinant fibronectin fragment, and contacting the hematopoietic cells with a recombinant retroviral vector comprising a sequence encoding a therapeutic protein operably linked to a promoter sequence, Prostaglandin E2 (PGE2) or a derivative thereof, and a poloxamer.

Dependencies are inferred from the claim text.

Facts

Application

17/264,275Filed 30 Jul 2019 · us provisional application

Priority

30 Jul 2018

Published

US 2021/0290685 A123 Sep 2021

Granted

21 Jul 2026

Term ends

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Classification7 codes

A61K 35/28
Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically…:…
C12N 5/0647
Cells from the blood or the immune system: haematopoietic stem cells; Uncommitted or multipotent progenitors
C12N 15/86
For animal cells: viral vectors
C12N 2500/50
Specific components of cell culture medium: soluble polymers, e.g. polyethyleneglycol [PEG]