Cell Therapy News Volume 14.26 | Jul 8 2013

Whole Body Correction of Mucopolysaccharidosis IIIA by Intracerebrospinal Fluid Gene Therapy
The authors demonstrated that intracerebrospinal fluid administration of serotype 9 adenoassociated viral vectors encoding sulfamidase corrects both CNS and somatic pathology in Mucopolysaccharidosis type IIIA mice. Following vector administration, enzymatic activity increased throughout the brain and in serum, leading to whole body correction of glycosaminoglycan accumulation and lysosomal pathology, normalization of behavioral deficits, and prolonged survival. [J Clin Invest] Full Article | Press Release

The Transfer of Host MHC Class I Protein Protects Donor Cells from NK Cell and Macrophage Mediated Rejection during Hematopoietic Stem Cell Transplantation and Engraftment in Mice
Researchers used a standard mouse model of bone marrow transplantation to demonstrate that surface protein transfer between cells plays an important role in protecting donor hematopoietic cells from NK cell and macrophage-mediated rejection. The transfer of intact MHC class I antigens from host cells to transplanted donor cells confers a self identity on these otherwise foreign cells. This gives them the ability to evade detection by the host NK cells and macrophages. [Stem Cells] Abstract

Reversing Bone Loss by Directing Mesenchymal Stem Cells to the Bone
Researchers developed a method to direct mesenchymal stem cells (MSCs) to the bone surface by conjugating a synthetic peptidomimetic ligand (LLP2A) that has high affinity for activated α4β1 integrin on the MSC surface, with a bisphosphonates (alendronate) that has high affinity for bone (LLP2A-Ale), to direct the transplanted MSCs to bone. LLP2A-Ale increased the homing of the transplanted MSCs to bone as well as the osteoblast surface, significantly increased the rate of bone formation and restored both trabecular and cortical bone loss induced by estrogen deficiency or advanced age in mice. [Stem Cells] Abstract

Engineered Neural Tissue for Peripheral Nerve Repair
Self-alignment of Schwann cells within a tethered type-1 collagen matrix, followed by removal of interstitial fluid produces a stable tissue-like biomaterial that recreates the aligned cellular and extracellular matrix architecture associated with nerve grafts. Sheets of this engineered neural tissue supported and directed neuronal growth in a co-culture model, and initial in vivo tests showed that a device containing rods of rolled-up sheets could support neuronal growth during rat sciatic nerve repair. [Biomaterials] Abstract | Press Release

Autologous Myoblast Transplantation for Oculopharyngeal Muscular Dystrophy: A Phase I/IIa Clinical Study
Oculopharyngeal muscular dystrophy (OPMD) is a late onset autosomal dominant genetic disease mainly characterized by ptosis and dysphagia. Researchers conducted a Phase I/IIa clinical study using autologous myoblast transplantation following myotomy in adult OPMD patients. [Mol Ther] Abstract

Persistence and Efficacy of Second Generation CAR-T Cell against the LeY Antigen in Acute Myeloid Leukemia
In a phase I study of autologous chimeric antigen receptor (CAR) anti-LeY T cell therapy of acute myeloid leukemia, the authors examined the safety and post-infusion persistence of adoptively transferred T cells. [Mol Ther] Abstract

Lentivirus-Mediated CD/TK Fusion Gene Transfection Neural Stem Cell Therapy for C6 Glioblastoma
Investigators aimed to transfect lentivirus-mediated CD/TK fusion gene into Wistar rat’s neural stem cells (NSC) and then implant the NSC into a C6 glioma model to observe a C6 glioma growth inhibition effect. [Tumor Biol] Abstract

Immunotherapy with Gene-Modified T Cells: Limiting Side Effects Provides New Challenges
This review discusses factors that might contribute to toxic side effects of therapy with gene modified T cells, and outlines potential strategies to retain anticancer activity while reducing unwanted side effects. [Gene Ther] Abstract

Visit our reviews page to see a complete list of reviews in the cell, gene and immunotherapy research field.

Alnylam Presents New Pre-Clinical Data on ALN-AT3, an RNAi Therapeutic Targeting Antithrombin (AT) for the Treatment of Hemophilia and Rare Bleeding Disorders
The new pre-clinical data demonstrate that ALN-AT3, a subcutaneously administered RNAi therapeutic targeting AT, can normalize thrombin generation and improve hemostasis in hemophilia mice and fully correct thrombin generation in a non-human primate hemophilia “inhibitor” model. [Press release from Alnylam Pharmaceuticals, Inc. discussing research presented at the XXIV Congress of the International Society on Thrombosis and Haemostasis, Amsterdam] Press Release

Anger as Spanish Funder Claws Back Science Money
Researchers at institutes funded by Spain’s National Council for Scientific Research (CSIC) are outraged over a move to take back unspent money from their budgets. The council’s decision is the latest development in Spain’s burgeoning science crisis. Any institute that has been frugal with its funding and has saved money will now find some of those savings absorbed back into the CSIC’s centralized pot. [National Council for Scientific Research, Spain] Press Release

Japan’s Science Policy Council Expands Role
Japan’s highest science advisory panel plans to be more active in implementing the current administration’s policies and in directly funding research. The new activism is intended to help it foster innovation, a central pillar of the new economic growth strategy of Prime Minister Shinzo Abe. [Council for Science and Technology Policy, Japan] Press Release

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Fondation Leducq Awards $6 Million Grant for Global Research Network for Cardiac Regeneration
The Fondation Leducq in Paris, France awarded a $6 million grant award to a new global research network of cardiovascular scientists which includes three researchers from the Cardiovascular Research Center at Icahn School of Medicine at Mount Sinai. This new large grant was awarded as part of the Fondation Leducq’s Transatlantic Networks of Excellence in Cardiovascular Research Program. Network researchers will aim to identify cellular and molecular targets to help advance cardiac regeneration therapeutics. [The Mount Sinai Hospital] Press Release

Nanofiber Solutions Receives Award from Ohio Third Frontier to Further Develop Tissue Engineered Intestine Technology
Nanofiber Solutions received a $100,000 grant to further develop the tissue engineered intestine technology developed in the lab of Dr. Besner, who also is principal investigator in the Center for Perinatal Research in The Research Institute at Nationwide Children’s. Nanofiber Solutions and The Research Institute have executed an option agreement for the technology and are expected to finalize an exclusive license agreement in the upcoming months. [Nationwide Children’s Hospital] Press Release

Cellectis Group Announces Groundbreaking In Vivo Proof of Concept Testing of Their Flagship UCART19 Product for Curative Therapy of Leukemia
Cellectis announced the successful in vivo proof of concept testing of their flagship UCART19 product for curative therapy of leukemia. UCART19 is a revolutionary engineered T-cell product that leverages Cellectis’ core technologies in genome engineering. [Cellectis] Press Release

Gamida Cell Reports FDA Recommending Further Study of StemEx^®
Gamida Cell announced that while the current single arm Phase II/III study, incorporating a historical cohort as the study control, provides evidence of activity of StemEx^®, the FDA has advised Gamida Cell to conduct another trial – a Phase III, randomized, controlled clinical study. StemEx^® is in development as an alternative transplantation treatment for patients with high risk leukemia and lymphoma, who could not find a family related matched bone marrow donor. [Gamida Cell] Press Release

Activartis’ Cancer Immunotherapy Receives Orphan Drug Designation from FDA
The US FDA notified the Austrian biotech company Activartis that Orphan Drug Designation (ODD) has been granted for its innovative Cancer Immunotherapy AV0113. The ODD applies specifically to the use of AV0113 for the treatment of malignant glioma, a very aggressive type of brain cancer. The European drug agency EMA already granted ODD at the end of last year. [Activartis Biotech GmbH] Press Release

Transgene Grants Ascend Biopharmaceuticals a License to TG1042, an Immunotherapy to Treat Basal Cell Carcinoma, a Common Type of Skin Cancer
Transgene SA announced it is granting Ascend Biopharmaceuticals a license to develop and commercialize TG1042 in basal cell carcinoma, a highly prevalent form of skin cancer, as well as in two other cancer indications. Transgene retains rights to other potential indications. TG1042 (now ASN-002 at Ascend) is an adenovirus-based immunotherapy that expects to enter phase I/II clinical trial in nodular basal cell carcinoma in Australia later in 2013. [Transgene SA] Press Release

Dicerna Announces Additional Patent Claims Allowed, Broadening Coverage of Extended Dicer Substrate Therapeutic Structures
Dicerna Pharmaceuticals, Inc. announced that the United States Patent and Trademark Office (USPTO) has issued a Notice of Allowance for patent application number 12/642,371. This allowance adds a broad new class of oligonucleotide structures to the company’s proprietary Extended Dicer Substrate™ therapeutic platform, reaffirming Dicerna’s unparalleled intellectual property position in the field of Dicer substrate therapeutics and commitment to developing new classes of oncology treatments using its Dicer Substrate Technology™. [Dicerna Pharmaceuticals, Inc.] Press Release  

Center for Scientific Review; Notice of Closed Meetings (FR Doc No: 2013-16081)

National Institute of Biomedical Imaging and Bioengineering; Notice of Meeting (FR Doc No: 2013-15772)  

NEW The European Cancer Congress 2013
September 27-October 1, 2013
Amsterdam, Netherlands

Visit our events page to see a complete list of events in the cell, gene and immunotherapy community.

NEW Postdoctoral Fellowship – Immunology (Johns Hopkins University School of Medicine)

PhD Positions – Tissue Engineering (8 European Research Institutes)

Postdoctoral Fellowship – Organic Synthesis and Medicinal Chemistry (Masaryk University)

Research Scientist/Senior Research Officer – Monoclonal Antibodies (Bioprocessing Technology Institute/A*STAR)

Postdoctoral Fellow/Research Scientist – Tissue Engineering (Institute of Bioengineering and Nanotechnology)

Director of Cell Processing Facility (S L Collins Associates, Inc.)

Postdoctoral Position – Developmental Neurobiology (North Carolina College of Veterinary Medicine)

Senior Postdoctoral Research Position – Molecular Cardiovascular and Renal Research (University of Oklahoma Health Sciences Center)

Postdoctoral Researcher – Hematopoietic Stem Cells (Albert Einstein College of Medicine)

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