Cellectis

Seattle Children's Hospital

Seattle Children's Research Institute

Beth Israel Deaconess Medical Center, Inc.

Wisconsin Alumni Research Foundation

University of Washington

Fred Hutchinson Cancer Center

Umoja Biopharma, Inc.

Cellectis S.a.

The Children's Hospital and Regional Medical Center

Seattle Children's Hospital Dba Seattle Children's Research Institute

Andrew M Scharenberg

Julianne Smith

Roman Galetto

Kamila Sabina Gwiazda

Laurent Poirot

Michael T Certo

David J Rawlings

Agnes Gouble

Stephanie Grosse

Cecile Mannioui

Cécile Schiffer-Mannioui

Kyle Jacoby

Alexandra E Grier

Hans-Peter Kiem

Olivier M Humbert

Iram F Khan

Yuchi Chiang Honaker

Christopher Lux

Sowmya Pattabhi

Karen Sommer

Cecile Schiffer-Mannioui

Michael C Jensen

Ryo Takeuchi

David Sourdive

John M Denu

Cècile Schiffer-Mannioui

Justin Eyquem

Laurie Beitz

Samuel West

Thor Wagner

Richard James

Shaun W Jackson

Jaya Sahni

Carole Desseaux

Cécile Mannioui

King Hung

Blythe Sather

Stéphanie Grosse

Agnès Gouble

Troy Torgerson

Swati Singh

Cécile Schiffer-mannioui

The present disclosure provides improved compositions for the homology directed repair of the human globin locus for the prevention, treatment, or amelioration of at least one symptom of a hemoglobinopathy.

Homology directed repair compositions for the treatment of hemoglobinopathies

The present disclosure relates to the co-expression of an endonuclease with an end-processing enzyme for the purpose of enhanced processing of the polynucleotide ends generated by endonuclease cleavage.

Coupling endonucleases with end-processing enzymes drives high efficiency gene disruption

The present application relates to compositions comprising fusion proteins and cells expressing the proteins. The application further relates to methods of using the fusion proteins, cells, and compositions for modulating cell signaling and for selective expansion of cells.

Methods of exogenous drug activation of chemical-induced signaling complexes expressed in engineered cells in vitro and in vivo

The disclosure relates generally to nucleic acid vectors and packaging cell lines for in vivo expansion of T-cells. More particularly, the disclosure relates to direct intratumoral injection of a lentiviral vector adapted for transduction and drug-mediated expansion of tumor-infiltrating lymphocytes in vivo.

Viral vectors and packaging cell lines

The present application relates to, inter alia, compositions including proteins for expression in host cells to render them resistant to rapamycin. The application further relates to methods of using the proteins, cells, and compositions disclosed therein for modulating cell signaling and for selective expansion of cells.

Rapamycin resistant cells

The present application relates to plasma cells and plasma cell precursors that express a macromolecule, such as a protein, protein mimetic or a peptide and compositions comprising these plasma cells or plasma cell precursors. The application further relates to methods of using and making the plasma cells and plasma cell precursors that express the macromolecule. Methods of treatment comprising administering the plasma cells or plasma cell precursors are also contemplated.

Engraftable cell-based immunotherapy for long-term delivery of therapeutic proteins

Methods for developing engineered T-cells for immunotherapy that are both non-alloreactive and resistant to immunosuppressive drugs. The present invention relates to methods for modifying T-cells by inactivating both genes encoding target for an immunosuppressive agent and T-cell receptor, in particular genes encoding CD52 and TCR. This method involves the use of specific rare cutting endonucleases, in particular TALE-nucleases (TAL effector endonuclease) and polynucleotides encoding such polypeptides, to precisely target a selection of key genes in T-cells, which are available from donors or from culture of primary cells. The invention opens the way to standard and affordable adoptive immunotherapy strategies for treating cancer and viral infections.

Methods for engineering allogeneic and immunosuppressive resistant T cell for immunotherapy

Aspects of the invention described herein concern the incorporation of a FOXP3 cDNA (e.g., full-length human codon-optimized cDNA) into a FOXP3 gene or a non-FOXP3 locus so as to provide constitutive or regulated FOXP3 expression in a primary human CD34cells or cells derived from edited CD34cells. In some embodiments, guide RNA sequences that are directed to FOXP3, AAVS1, or other candidate loci are used for CRISPR/Cas9-mediated gene regulation, and gene delivery cassettes for HDR based gene-modification are provided.

Expression of FOXP3 in edited CD34+ cells

Disclosed are methods of making a genetically cell that expressed FOXP3 and methods of treatment. In some embodiments, the method can providing a first nucleotide sequence, wherein the first nucleotide sequence comprises a coding strand, the coding strand comprising one or more regulatory elements and a FOXP3 gene or portion thereof providing a nuclease and performing a gene editing process on the first nucleotide sequence, which edits said one or more regulatory elements, and optionally edits the FOXP3 gene or portion thereof. Methods of treating a subject suffering from an autoimmune disease and subjects suffering the effects of organ transplantation are also provided.

Method for treating autoimmune disease using CD4 T-cells with engineered stabilization of expression of endogenous FOXP3 gene

A method of expanding deficient T-cells by expressing pTalpha or functional variants thereof into said cells, thereby restoring a functional CD3 complex. This method is particularly useful to enhance the efficiency of immunotherapy using primary T-cells from donors. This method involves the use of pTalpha or functional variants thereof and polynucleotides encoding such polypeptides to expand TCRalpha deficient T-cells. Such engineered cells can be obtained by using specific rare-cutting endonuclease, preferably TALE-nucleases. The use of Chimeric Antigen Receptor (CAR), especially multi-chain CAR, in such engineered cells to target malignant or infected cells. The invention opens the way to standard and affordable adoptive immunotherapy strategies for treating cancer and viral infections.

Use of pre T alpha or functional variant thereof for expanding TCR alpha deficient T cells

Growing community of inventors

Seattle, WA, United States of America

Andrew M Scharenberg