Cell Therapy News Volume 16.30 | Aug 10 2015

Hemostatic Efficacy, Safety and Pharmacokinetics of a Recombinant von Willebrand Factor in Severe von Willebrand Disease
This Phase III trial evaluated the safety and hemostatic efficacy of a recombinant von Willebrand factor (rVWF) for treatment of bleeds in severe von Willebrand disease. rVWF was initially administered together with rFVIII and subsequently alone, so long as hemostatic FVIII:C levels were maintained. [Blood] Abstract | Press Release

Generation of Knock-In Primary Human T Cells using Cas9 Ribonucleoproteins
Researchers report efficient genome engineering in human CD4⁺ T cells using Cas9:single-guide RNA ribonucleoproteins (Cas9 RNPs). Cas9 RNPs allowed ablation of CXCR4, a coreceptor for HIV entry. [Proc Natl Acad Sci USA] Abstract | Press Release

In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector
In-silico-derived sequences were synthesized de novo and characterized for biological properties relevant to clinical applications. This effort led to the generation of nine functional putative ancestral adeno-associated viruses (AAVs) and the identification of Anc80, the predicted ancestor of the widely studied AAV serotypes 1, 2, 8, and 9, as a highly potent in vivo gene therapy vector for targeting liver, muscle, and retina. [Cell Rep]
Full Article | Graphical Abstract | Press Release

Intracisternal Gtf2i Gene Therapy Ameliorates Deficits in Cognition and Synaptic Plasticity of a Mouse Model of Williams-Beuren Syndrome
The authors’ findings indicate that Gtf2i haploinsufficiency plays an important role in the neurodevelopmental and cognitive abnormalities of Williams-Beuren syndrome and that it is possible to rescue part of this neurocognitive phenotype by restoring Gtf2i expression levels in specific brain areas. [Mol Ther] Abstract

Network Analysis of Immunotherapy-Induced Regressing Tumors Identifies Novel Synergistic Drug Combinations
The authors hypothesized that the effector response in the tumor could be visualized as a complex network of interacting gene products and that by mapping this network they could predict effective pharmacological interventions. They provide proof of concept for the validity of this approach in a murine mesothelioma model, which displays a dichotomous response to anti-CTLA4 immune checkpoint blockade. [Sci Rep] Full Article

Exclusive Transduction of Human CD4⁺ T Cells upon Systemic Delivery of CD4-Targeted Lentiviral Vectors
Scientists describe novel lentiviral vectors (CD4-LV) that have been rendered selective for human or simian CD4⁺ cells by surface engineering. The potential for in vivo delivery of the FOXP3 gene was also demonstrated, making CD4-LV a powerful tool for inducible regulatory T cell generation. [J Immunol] Abstract

Adipose Tissue-Derived Mesenchymal Stromal Cells Protect Mice Infected with Trypanosoma cruzi from Cardiac Damage through Modulation of Anti-Parasite Immunity
Researchers investigated if adipose tissue-derived mesenchymal stromal cells can alter the course of Chagas disease and attenuate pathology in a mouse model of chagasic cardiomyopathy. [PLoS Negl Trop Dis] Full Article | Press Release

Transient Suppression of Hepatocellular Replication in the Mouse Liver following Transduction with Recombinant Adeno-Associated Virus
Scientists showed, for the first time in vivo, that in addition to the known effects of hepatocellular replication on adeno-associated virus-mediated gene transfer, the vector itself exerts a potent, albeit transient suppressive effect on cell cycle progression that is relieved on a time course that correlates with the known rate of clearance of input single-stranded vector DNA. [Gene Ther] Abstract

Multipotent Bone Marrow Stromal Cell Therapy Promotes Endogenous Cell Proliferation following Ischemic Stroke
Investigators extended their understanding of the effects of bone marrow stromal cell (BMSC) treatment following experimentally induced ischemic stroke in rats. Animals were injected intravenously with BMSCs at 24 hours after stroke and were sacrificed seven days post injury. [Clin Exp Pharmacol Physiol] Abstract

Cell Therapy for Parkinson’s Disease: Functional Role of the Host Immune Response on Survival and Differentiation of Dopaminergic Neuroblasts
The authors discussed efforts on cell transplantation in animal models and Parkinson’s disease patients, highlighting the immune response and the immunomodulatory treatment strategies performed. [Brain Res] Abstract

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Denver Back Pain Specialists Physicians Begin Enrollment in Stem Cell Therapy Study in Subjects with Chronic Back Pain
Denver Back Pain Specialists announced that it has enrolled its first patient in a nationwide FDA-cleared adult stem cell study (Phase III) testing an investigational treatment for chronic low back pain associated with degenerative disc disease. The study will test the use of mesenchymal precursor cells – adult stem cells derived from bone marrow that will be directly injected into the lumbar disc. [Denver Back Pain Specialists] Press Release

Caution Urged Over Editing DNA in Wildlife (Intentionally or Not)
The US National Academy of Sciences, Engineering, and Medicine held the first in a series of meetings meant to find ways to balance the promise and perils of the ‘gene drive’ technique. The method can rapidly modify not just a single organism but a whole population, by inserting a desired genetic modification into an organism along with DNA that increases the rate at which the change is passed to the next generation. [Nature News] Editorial

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Sanofi and Regeneron Launch Major New Immuno-Oncology Collaboration
Sanofi and Regeneron Pharmaceuticals, Inc. have entered into a new global collaboration to discover, develop and commercialize new antibody cancer treatments in the emerging field of immuno-oncology. [Sanofi] Press Release

La Jolla Institute Announces Affiliation with UC San Diego Health
The La Jolla Institute for Allergy and Immunology announced an official affiliation agreement with the University of California, San Diego Health System (UCSD). The multi-year agreement with UCSD will facilitate joint faculty appointments, enhance the range and depth of collaboration between the two organizations, and promote closer integration between basic immunology research and clinical medicine to speed the development of treatments for diseases of the immune system. [La Jolla Institute for Allergy and Immunology] Press Release

Moffitt Cancer Center Teams Up with MD Anderson to Discover, Test Novel Agents to Prevent Cancer
Moffitt Cancer Center has joined MD Anderson’s National Cancer Institute (NCI) Phase 0/I/II Cancer Prevention Clinical Chemoprevention Trials Consortia. Sponsored by the NCI’s Division of Cancer Prevention, the consortia conducts early clinical development of promising new preventive agents through its Phase 0/I/II Cancer Prevention Clinical Trials Program. [Moffitt Cancer Center] Press Release

AstraZeneca and Heptares Therapeutics Enter Agreement to Develop Novel Immuno-Oncology Treatments
AstraZeneca and Heptares Therapeutics announced that they have entered into a licensing agreement under which AstraZeneca will acquire exclusive global rights to develop, manufacture and commercialize the adenosine A_2A receptor antagonist, HTL-1071, a small molecule immuno-oncology candidate, and potential additional A_2A receptor-blocking compounds. [AstraZeneca] Press Release

Sorrento In-Licenses Four Late-Stage Clinical Biobetter and Biosimilar Antibodies
Sorrento Therapeutics, Inc. announced that it has entered into an exclusive licensing agreement to develop and commercialize multiple prespecified and undisclosed biosimilar or biobetter antibodies from Mabtech Limited, a holding company for premier antibody development and manufacturing companies in China. [Sorrento Therapeutics, Inc.] Press Release

MabVax Therapeutics and The Rockefeller University Enter into a Collaborative Research Agreement
MabVax Therapeutics Holdings, Inc. and The Rockefeller University’s Laboratory of Molecular Genetics and Immunology have entered into a research collaboration agreement to explore the mechanism of action of constant region variants of the HuMab 5B1 in the role of tumor clearance. [MabVax Therapeutics Holdings, Inc.] Press Release

Bristol-Myers Squibb Establishes Collaborative Immuno-Oncology Rare Population Malignancy (I-O RPM) Program in the U.S.
Bristol-Myers Squibb Company announced the establishment of the Immuno-Oncology Rare Population Malignancy (I-O RPM) program in the U.S. As part of the I-O RPM program, Bristol-Myers Squibb, the Robert H. Lurie Comprehensive Cancer Center of Northwestern University and the Northwestern Medicine Developmental Therapeutics Institute announced that they have entered into a collaboration agreement. [Bristol-Myers Squibb Company] Press Release

University of Toronto to Transform Regenerative Medicine Thanks to Historic $114 Million Federal Grant
The University of Toronto is set to cement its position as one of the world’s leading centers for the design and manufacture of cells, tissues and organs that can be used to treat degenerative disease, thanks to a $114-million grant from the federal government. [University of Toronto] Press Release

Cell Therapy Catapult Awards Major Contract to Cellular Therapeutics Limited to Accelerate WT1 Clinical Program
The Cell Therapy Catapult has awarded a manufacturing contract to Cellular Therapeutics Limited, to accelerate its clinical program to develop an engineered T cell treatment for blood cancers such as acute myeloid leukaemia and myelodysplastic syndrome. [Cellular Therapeutics Limited] Press Release

Newcastle Gets £5 Million to Boost Drug Development
Cancer Research UK’s Drug Discovery Committee has awarded Newcastle University’s Northern Institute of Cancer Research £5 million to bring more cancer drugs to clinical trials. [Cancer Research UK] Press Release

Stem Cell Transplant Program Earns International Accreditation
The Stem Cell Transplant Program at the University of Virginia Cancer Center has received international accreditation for its use of stem cells and bone marrow to treat patients with blood cancers. [University of Virginia Cancer Center] Press Release

Brief Summary and Adequate Directions for Use: Disclosing Risk Information in Consumer-Directed Print Advertisements and Promotional Labeling for Prescription Drugs; Revised Draft Guidance for Industry (Revision 2); Availability (FR Doc. No: 2015-19244)

Evidentiary Considerations for Integration of Biomarkers in Drug Development; Notice of Public Meeting; Request for Comments (FR Doc. No: 2015-19037)

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Research Associate – Cell Biology (Editas Medicine)

Senior Research Associate (Editas Medicine)

Postdoctoral Fellow – Biotechnology (Qu Biologics Inc.)

Cell Biology Lab Manager (Harvard Apparatus Regenerative Technology)

Science Writer – Cardiovascular Regeneration (Houston Methodist Research Institute)

PhD Studentship – Veterinary and Animal Sciences (University of Milan)

Postdoctoral Research Associate – Breast Stem Cell Biology (University of Miami Miller School of Medicine)

Aseptic Manufacturing Associate (Lonza Biologics, Inc.)

Postdoctoral Position – Translational Immunology (University of Utah School of Medicine)

Postdoctoral Fellow – Stem Cell Biology (INSERM)

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