Csl Behring Gene Therapy, Inc.

Csl Gene Therapy Pty Ltd.

Mallinckrodt Hospital Products IP Limited

Johnson & Johnson Research Pty Limited

Newsouth Innovations Pty Limited

Gene Shears Pty. Limited

St. Vincent's Hospital Sydney Limited

Calimmune, Inc.

Calimmune Australia Pty Ltd

Therakos Development Limited

University of California

Geoffrey Phillip Symonds

Lun-Quan Sun

David Peritt

Wayne Lyle Gerlach

Irvin Chen

Ming Yan

Dong Sung An

Janet Lesley Macpherson

Jeffrey S Bartlett

Philip Wong

Kevin Henrichsen

Susan Pond

Rafael G Amado

Anthony Dominic Kelleher

Chi-Lin Lee

Kazuo Suzuki

Jeffrey Ahlers

Gregory C Fanning

Christopher Walter Alma

Gene-Errol Eugenio Ringpis

Janet L MacPherson

Louis Randall Breton

Greg Fanning

Chantelle Lisa Evelyn Ahlenstiel

Maureen Boyd

Michelle L Millington

Michelle Millington

Methods (), devices, and systems of processing blood are described. The method () comprises the steps of: obtaining () blood from a patient coupled to a single blood processing device to form a closed loop between the patient and the blood processing device; collecting () bulk mononuclear blood cells from the blood by leukapheresis implemented using the blood processing device in the closed loop; and enriching () concurrently target cells separated from non-target cells in the bulk mononuclear blood cells using the blood processing device in the closed loop.

Processing blood

The present disclosure provides expression vectors comprising at least two nucleic acid sequences, namely a nucleic acid sequence encoding an anti-HPRT RNAi, and a nucleic acid sequence encoding a gamma globin gene. In some embodiments, the viral vector is a self-inactivating lentiviral vector. In some embodiments, the gamma-globin gene is used to genetically correct sickle cell disease or &#x3b2;-thalassemia or to reduce symptoms thereof.

Compositions and methods for treating beta-hemoglobinopathies

The disclosed methods are generally directed to preventing, treating, suppressing, controlling or otherwise mitigating side effects of T-cell therapy, the T-cell therapy designed to accelerate immune reconstitution, induce a GVM effect, and/or target tumor cells.

Modulatable switch for selection of donor modified cells

The present disclosure provides compositions (i.e., amplification primers and probes), methods, and kits that are particularly useful for detecting and/or quantifying nucleic acids present in a sample, such as those derived from HIV or a lentiviral vector.

Methods for discriminating between HIV-1 and lentiviral vectors

Described are compositions and methods relating to gene therapy, particularly as applied to hematopoietic progenitor (HP) cells, to transduced cells and methods of obtaining them, and to methods of using them to provide prolonged engraftment of modified hematopoietic cells in human subjects. The invention particularly relates to ex vivo gene therapy of HP cells for treatment or prevention of HIV infection.

Process for the preparation of a composition of genetically modified hematopoietic progenitor cells

Methods for genetic modification of hematopoietic progenitor cells and uses of the modified cells

A cell comprising a synthetic non-naturally occurring oligonucleotide compound comprises nucleotides whose sequence defines a conserved catalytic region and nucleotides whose sequence hybridizes with a predetermined target sequence within a MoMLV Psi packaging sequence on the HIV tat sequence. The catalytic region may be derived from a hammerhead ribozyme, a hairpin ribozyme a hepatitis delta ribozyme, an PNAase P ribozyme, a group I intron or a group II intron.

Ribozymes targeting the MoMLV PSI packaging sequence and the HIV tat

This invention is directed to a synthetic non-naturally occurring oligonucleotide compound which comprises nucleotides whose sequence defines a conserved catalytic region and nucleotides whose sequence is capable of hybridizing with a predetermined target sequence within a packaging sequence of an RNA virus. Preferably, the viral packaging sequence is a retrovirus packaging sequence or the HIV-1 Psi packaging sequence. The RNA virus may be HIV-1, Feline Leukemia Virus, Feline Immunodeficiency Virus or one of the viruses listed in Table I. The conserved catalytic region may be derived from a hammerhead ribozyme, a hairpin ribozyme, a hepatitis delta ribozyme, an RNAase P ribozyme, a group I intron, a group II intron. The invention is also directed to multiple ribozymes, combinations of ribozymes, with or without antisense, and combinations of ribozymes, with antisense, and TAR decoys, poly TARs or RRE decoys targeted against the RNA virus and combinations of ribozymes and antisense targetted against the RNA virus. Vectors are also described. Further, methods of treatment and methods of use both in vivo and ex vivo are described.

Ribozymes targeting retroviral packaging sequence expression constructs

Growing community of inventors

Rose Bay, Australia

Geoffrey Phillip Symonds