Fred Hutchinson Cancer Center

Seattle Children's Hospital

Memorial Sloan Kettering Cancer Center

Universitéde Montreal

British Columbia Cancer Agency Branch

Fred Hutchinson Cancer Research Center

Hans-Peter Kiem

Olivier M Humbert

Andrew M Scharenberg

David J Rawlings

Marcel Van Den Brink

Jarrod Dudakov

Jennifer E Adair

Kyle Jacoby

Christopher Lux

Sowmya Pattabhi

Enrico Velardi

Stefan Radtke

Scott James

Guy Sauvageau

Iman Fares

Keith Richard Humphries

Jalila Chagraoui

Roland B Walter

Grant D Trobridge

Systems and methods to selectively protect therapeutic cells by reducing CD33 expression in the therapeutic cells and targeting non-therapeutic cells with an anti-CD33 therapy. The selective protection results in the enrichment of the therapeutic cells while simultaneously targeting any diseased, malignant and/or non-therapeutic CD33 expressing cells within a subject.

Reducing CD33 expression to selectively protect therapeutic cells

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 use of luteinizing hormone receptor (LHR) binding agents and luteinizing hormone (LH) agonists to enrich for primitive hematopoietic stem cell (pHSC) populations, to target pHSC for ablation, and/or to expand pHSC populations are described. The methods can be used to prepare therapeutic hematopoietic stem cell (HSC) populations, to prepare patients for therapeutic HSC transplants, and/or to treat malignancies, such as those associated with hyperproliferative HSC.

Luteinizing hormone receptor binding agents and luteinizing hormone agonists to identify, expand, ablate and modify stem cells

A platform for ex vivo isolation, production, and formulation of genetically-modified cells is described. The platform utilizes a software-enabled point-of-care and/or portable device making gene therapy more widely available.

Point-of-care and/or portable platform for gene therapy

Methods and compositions for enhancing viral gene transfer, such as lentiviral gene transfer, and improving the efficacy of gene delivery to cells such as primitive hematopoietic cells, are described. These methods and compositions are based on the use of pyrimido[4,5-b]indole derivatives. Cell-based compositions and methods useful for therapeutic indications amenable to treatment with gene therapies, including hematopoietic stem cell therapies, are also described.

Compounds and methods for enhancing viral gene transfer to human hematopoietic cells

Provided herein are Cocal vesiculovirus envelope pseudotyped retroviral vectors that exhibit high titers, broad species and cell-type tropism, and improved serum stability. Disclosed Cocal vesiculovirus envelope pseudotyped retroviral vectors may be suitably employed for gene therapy applications and, in particular, for the ex vivo and in vivo delivery of a gene of interest to a wide variety of target cells.

Cocal vesiculovirus envelope pseudotyped retroviral vectors

Growing community of inventors

Seattle, WA, United States of America

Hans-Peter Kiem