Cell Therapy News Volume 16.02 | Jan 19 2015

Enrichment of Human Hematopoietic Stem/Progenitor Cells Facilitates Transduction for Stem Cell Gene Therapy
CD34⁺/CD38^– cells were isolated from healthy cord blood CD34⁺ cells by fluorescence activated cell sorting and transduced with a lentiviral vector expressing an anti-sickling form of beta-globin. In vivo studies showed engraftment of transduced CD34⁺/CD38^– cells when transplanted in competition with 100-fold more CD34⁺/CD38⁺ cells. [Stem Cells] Abstract

Adeno-Associated Virus Serotype 9-Mediated Gene Therapy for X-Linked Adrenoleukodystrophy (X-ALD)
Scientists used recombinant adeno-associated virus serotype 9 (rAAV9) vector for delivery of the human ATP-binding cassette transporter gene (ABCD1) to mouse central nervous system (CNS). In vivo, AAV9-ABCD1 was delivered to Abcd1^-/- mouse CNS by either stereotactic intracerebroventricular or intravenous injections. [Mol Ther] Abstract

Triazine-Modified Dendrimer for Efficient TRAIL Gene Therapy in Osteosarcoma
A triazine-modified dendrimer G5-DAT₆₆ was synthesized and used as a vector for tumor necrosis factor related apoptosis inducing ligand (TRAIL) gene therapy in vitro and in vivo. In vivo studies prove that G5-DAT₆₆ efficiently transfects TRAIL plasmid in tumors and inhibits tumor growth in osteosarcoma-bearing mice. [Acta Biomater] Abstract

Evaluation of In Vivo Antitumor Effects of ANT2 shRNA Delivered Using PEI and Ultrasound with Microbubbles
To evaluate the antitumor effects of adenine nucleotide translocase-2 (ANT2) short hairpin RNA (shRNA) by intraperitoneal injection using the polyethylenimine (PEI) and an ultrasound gene delivery method, human breast carcinoma MDA-MB-231 cells were injected subcutaneously into NOD/Shi-scid/IL-2Rγ^null mice. [Gene Ther] Abstract

Sendai Virus-Mediated Gene Transfer of the c-Myc Suppressor Far-Upstream Element-Binding Protein-Interacting Repressor Suppresses Head and Neck Cancer
Using in vitro and in vivo xenograft mouse models, investigators observed efficient expression of green fluorescent protein (GFP) following transduction with the far-upstream element-binding protein-interacting repressor using fusion gene-deleted Sendai virus vector encoding GFP into head and neck squamous cell carcinoma cells. [Gene Ther] Abstract

Secretomes from Bone Marrow-Derived Mesenchymal Stromal Cells Enhance Periodontal Tissue Regeneration
Low survival rates and the potential tumorigenicity of implanted mesenchymal stromal cells (MSCs) could undermine the efficacy of cell-based therapy. The use of conditioned media from MSCs (MSC-CM) may be a feasible approach to overcome these limitations. Researchers aimed to confirm the effect of MSC-CM on periodontal regeneration. [Cytotherapy] Abstract

Stable Knockdown of LRG1 by RNA Interference Inhibits Growth and Promotes Apoptosis of Glioblastoma Cells In Vitro and In Vivo
Scientists further demonstrate that the silencing of leucine-rich α₂ glycoprotein 1 (LRG1) expression effectively reduced the tumorigenicity of U251 cells, delayed tumor formation, and promoted apoptosis in a xenograft tumor model in vivo. [Tumour Biol] Abstract

Prolonged Clinical Remissions in Patients with Relapsed or Refractory Follicular Lymphoma Treated with Autologous Stem Cell Transplantation Incorporating Rituximab
Three sequential Phase II trials were conducted with different immunotherapy approaches to enhance the outcome of autologous transplant (high-dose therapy and autologous stem cell transplantation) for recurrent follicular lymphoma. [Ann Hematol] Abstract

Tailoring Bioengineered Scaffolds for Stem Cell Applications in Tissue Engineering and Regenerative Medicine
New insights into the biology of stem cells are expected to eventuate from advances in material science, in particular, from synthetic hydrogels that display physicochemical properties reminiscent of the natural cell microenvironment and that can be engineered to display or encode essential biological cues. [Stem Cells Transl Med] Abstract

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OncoMed Presents Final Phase Ib Safety, Efficacy and Biomarker Data for Tarextumab in Pancreatic Cancer
OncoMed Pharmaceuticals, Inc. presented final safety, efficacy and biomarker data from the company’s Phase Ib ALPINE clinical trial of tarextumab in 40 patients with frontline metastatic pancreatic cancer. [Press release from OncoMed Pharmaceuticals, Inc. discussing research presented at American Society of Clinical Oncology (ASCO) Gastrointestinal Cancers Symposium, San Francisco] Press Release

Economists Offer New Arguments for U.S. Research Tax Break
The National Association of Manufacturers released a report from three academic economists that reviews the scholarly literature on the economic impact of the so-called R&D tax credit, worth some $7 billion annually in recent years. [ScienceInsider] Editorial | Report

A One-Grant Limit: NIH Institute Puts Squeeze on Flush Investigators
In the latest example of budget stretching at the National Institutes of Health (NIH), the agency’s basic science institute is imposing a strict one-grant limit on scientists who already have plentiful no-strings support. The move could free up at least $6 million, or 25 grants for other scientists. [ScienceInsider] Editorial

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Amgen and MD Anderson Announce Agreement to Develop BiTE^® Therapies for Myelodysplastic Syndrome
Amgen and The University of Texas MD Anderson Cancer Center announced a research collaborative agreement focusing on Amgen’s bispecific T cell engager (BiTE^®) antibody constructs, an immunotherapy that serves as a “bridge” between T cells and cancer cells. [Amgen] Press Release

Kymab Partners with Cancer Immunotherapy Expert on Anti-Cancer Therapeutics
Kymab announced a collaboration with Dr. Stephen Gillies. Dr. Gillies is a pioneer in the field of cancer immunotherapy and inventor of immunocytokine platforms from which several molecules are currently in clinical development. [Kymab Limited] Press Release

Treeway Announces License and Collaboration Agreement with uniQure to Develop a Gene Therapy for Amyotrophic Lateral Sclerosis (ALS)
Treeway B.V. have announced a collaboration with uniQure N.V. to develop a gene therapy treatment for ALS. Under the terms of the licensing and collaboration agreement, uniQure has granted Treeway an exclusive license in this field to uniQure’s relevant AAV5 viral vector and glial cell-derived neurotrophic factor intellectual property. [Treeway B.V.] Press Release

Boehringer Ingelheim and Yale University Collaborate to Investigate Novel Immunotherapy Targets Across Several Therapeutic Areas
Boehringer Ingelheim announced a collaboration with Yale University with the goal of researching novel therapeutic targets in the field of immune-modulation. Under the terms of the agreement, the research capabilities of Boehringer Ingelheim and Yale University will be brought together to identify new immune-modulatory agents for oncology, autoimmune and respiratory disorders. [Boehringer Ingelheim GmbH] Press Release

Intrexon, ZIOPHARM, and MD Anderson in Exclusive CAR T Pact
Intrexon Corporation and its oncology partner, ZIOPHARM Oncology announced a broad exclusive licensing agreement with The University of Texas MD Anderson Cancer Center, including an exclusive sublicensing agreement through MD Anderson for intellectual property developed at the University of Minnesota for the development of non-viral adoptive cellular cancer immunotherapies. [Intrexon Corporation] Press Release

Seattle Genetics and Bristol-Myers Squibb Announce Clinical Collaboration to Evaluate Combination of Adcetris^® (Brentuximab Vedotin) and Opdivo^® (Nivolumab) in Hematologic Malignancies
Seattle Genetics, Inc. and Bristol-Myers Squibb Company announced that the companies have entered into a clinical trial collaboration agreement to evaluate the investigational combination of Seattle Genetics’ antibody-drug conjugate Adcetris and Bristol-Myers Squibb’s immunotherapy Opdivo in two planned Phase I/II clinical trials. [Bristol-Myers Squibb Company] Press Release

Bristol-Myers Squibb and Lilly Enter Clinical Collaboration Agreement to Evaluate Opdivo (Nivolumab) in Combination with Galunisertib in Advanced Solid Tumors
Bristol-Myers Squibb Company and Eli Lilly and Company announced a clinical trial collaboration to evaluate the safety, tolerability and preliminary efficacy of Bristol-Myers Squibb’s immunotherapy Opdivo in combination with Lilly’s galunisertib. [Bristol-Myers Squibb Company] Press Release

Tekmira Pharmaceuticals and OnCore Biopharma Announce Merger Agreement to Create Leading Global Hepatitis B Virus Company
Tekmira Pharmaceuticals Corporation and OnCore Biopharma, Inc. announced that they have agreed to merge to create a new leading global hepatitis B virus company focused on developing a curative regimen for hepatitis B patients by combining multiple therapeutic approaches. [Tekmira Pharmaceuticals Corporation] Press Release

Bone Therapeutics Demonstrates Efficacy in First Patient Cohort in ALLOB^® Phase I/IIa Delayed-Union Trial
Bone Therapeutics announced positive efficacy results for the first cohort of four patients enrolled in the Phase I/IIa delayed-union trial with its allogeneic bone-forming cell product ALLOB^®. [Bone Therapeutics] Press Release

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