| PUBLICATIONS (Ranked by impact factor of the journal) | An RNAi Therapeutic Targeting Antithrombin to Rebalance the Coagulation System and Promote Hemostasis in Hemophilia Guided by reports suggesting that co-inheritance of prothrombotic mutations may ameliorate the clinical phenotype in hemophilia, investigators developed an RNA interference (RNAi) therapeutic (ALN-AT3) targeting antithrombin (AT) as a means to promote hemostasis in hemophilia. When administered subcutaneously, ALN-AT3 showed potent, dose-dependent, and durable reduction of AT levels in wild-type mice, mice with hemophilia A, and nonhuman primates. [Nat Med] Abstract | Press Release Faster T-Cell Development following Gene Therapy Compared to Haplo-Identical Hematopoietic Stem Cell Transplantation in the Treatment of SCID-X1 Researchers retrospectively analyzed and compared clinical outcomes and immune reconstitution in 13 consecutive severe combined immunodeficiency caused by ILR2G mutation (SCID-X1) patients having undergone haploidentical hematopoietic stem cell transplantation and 14 SCID-X1 patients treated with gene therapy over the same period at a single center level, the Necker Children’s Hospital. [Blood] Abstract | Full Article | Press Release ADAP and SKAP55 Deficiency Suppresses PD-1 Expression in CD8+ Cytotoxic T Lymphocytes for Enhanced Anti-Tumor Immunotherapy Scientists identified that the ADAP-SKAP55 signaling module reduced CD8+ cytotoxic T lymphocytes cytotoxicity and enhanced PD-1 expression in a Fyn-, Ca2+-, and NFATc1-dependent manner. [EMBO Mol Med] Full Article Long-Term Controlled Delivery of rhBMP-2 from Collagen-Hydroxyapatite Scaffolds for Superior Bone Tissue Regeneration Investigators aimed to design and develop a delivery system to release low recombinant human bone morphogenetic protein 2 (rhBMP-2) doses from a collagen-hydroxyapatite scaffold which had been previously been optimized for bone regeneration and recently demonstrated significant healing in vivo. [J Control Release] Abstract Ex Vivo Induced Regulatory Human/Murine Mesenchymal Stem Cells as Immune Modulators The authors established an ex vivo immunomodulatory triple combination treatment (TCT) for mesenchymal stem cells (MSCs), using IFNγ, TGFβ and kynurenine. To test the immunomodulatory function of TCT MSCs in vivo, they used the cells as graft-versus-host disease (GvHD) prophylaxis in a GvHD mouse model. [Stem Cells] Abstract Gene Electrotransfer of Plasmid AMEP, an Integrin-Targeted Therapy, Has Antitumor and Antiangiogenic Action in Murine B16 Melanoma Researchers investigated the antiangiogenic and direct antitumor effectiveness of gene therapy with Plasmid AMEP (antiangiogenic metargidin peptide) on low and highly metastatic B16 melanoma variants. In vivo, antitumor effectiveness was mediated by the inhibition of proliferation, migration and invasion of melanoma cells and correlated with the expression of integrins on tumor cells after intratumor delivery. [Gene Ther] Abstract Long-Term Results of Adipose-Derived Stem Cell Therapy for the Treatment of Crohn’s Fistula A previous Phase II clinical trial of adipose-derived stem cell therapy for fistulae associated with Crohn’s disease, a devastating condition with a high recurrence rate, demonstrated safety and therapeutic potential with a one-year sustained response. In the present study, 41 of the 43 Phase II trial patients were followed for an additional year, regardless of response in the initial year. [Stem Cells Transl Med] Abstract Delayed Minimally Invasive Injection of Allogenic Bone Marrow Stromal Cell Sheets Regenerates Large Bone Defects in an Ovine Preclinical Animal Model Researchers developed a unique treatment concept using the delayed injection of allogenic bone marrow stromal cell sheets to regenerate a critical-sized tibial defect in sheep to study the effect of the cells’ regeneration potential when introduced at a postinflammatory stage. [Stem Cells Transl Med] Abstract The Critical Role of Induced CD4+ FoxP3+ Regulatory Cells in Suppression of Interleukin-17 Production and Attenuation of Mouse Orthotopic Lung Allograft Rejection Researchers established mouse orthotopic lung transplantation models to investigate the importance of interleukin (IL)-17 and IL-17-producing cell types in acute lung allograft rejection and the efficacy of the adoptive transfer of induced regulatory T cells in attenuating pathologic lesions of lung allografts. [Transplantation] Abstract |
| POLICY | Research Advocates Watch, Warily, as Congress Tries to Finish Its Budget Outline Congress returned to work after a two-week recess, and a top priority for House of Representatives and Senate Republican leaders is reconciling their versions of a largely symbolic but politically sensitive budget plan. Science boosters loathe the spending blueprints approved by the House and Senate. That’s because they would, if implemented, squeeze federal funding for civilian research over the long term. [ScienceInsider] Editorial Synthetic Biologists Seek Standards for Nascent Field Representatives from industry, academic institutions and government met at Stanford University to launch the Synthetic Biology Standards Consortium, an initiative led by the US National Institute of Standards and Technology to address issues preventing the field from reaching its potential. [Nature News] Editorial |
| BUSINESS | Advaxis and Merck Announce Initiation of Enrollment in the Phase I/II Study of ADXS-PSA in Combination with Anti-PD-1 Therapy, KEYTRUDA® (Pembrolizumab), for the Treatment of Advanced Prostate Cancer Advaxis, Inc. and Merck announced that enrollment has initiated in the Phase I/II clinical trial evaluating the combination of ADXS-PSA (ADXS31-142), an investigational Lm-LLO immunotherapy, and KEYTRUDA®, the first anti-PD-1 (programmed death receptor-1) therapy approved in the United States, in patients with previously treated, metastatic castration-resistant prostate cancer. [Advaxis, Inc.] Press Release Taxus Cardium Announces Agreement with Dr. Reddy’s Laboratories to Co-Develop, Market & Sell Generx Angiogenic Microvascular Gene Therapy in Certain International Markets Taxus Cardium Pharmaceuticals Group Inc. announced that it has entered into a binding term sheet with Dr. Reddy’s Laboratories Ltd. for an exclusive territorial license covering the co-development, marketing and sales of the Generx® angiogenic microvascular gene therapy Phase III product candidate for patients with refractory angina, and myocardial ischemia due to cardiac microvascular insufficiency. [Taxus Cardium Pharmaceuticals Group Inc.] Press Release MD Anderson, Astellas Pharma Sign Option Agreement for Monoclonal Antibody Drug Targeting Acute Myeloid Leukemia Astellas Pharma Inc. and The University of Texas MD Anderson Cancer Center announced the signing of an option agreement to research and develop a new treatment for patients with acute myeloid leukemia. [The University of Texas MD Anderson Cancer Center] Press Release Mount Sinai Receives $8.8 Million Grant to Further Stem Cell Research An $8.8 million grant from the New York State Stem Cell Science Program will accelerate efforts by the Icahn School of Medicine at Mount Sinai to develop new stem-cell-based treatments for chemotherapy-resistant blood cancer and other genetic blood disorders. [Icahn School of Medicine at Mount Sinai] Press Release FDA Modifies Partial Clinical Hold on Tekmira’s TKM-Ebola IND to Allow Multiple Dosing of Healthy Volunteers Tekmira Pharmaceuticals Corporation announced that the U.S. Food and Drug Administration (FDA) has notified the company that the partial clinical hold on the company’s Investigational New Drug application (IND) for TKM-Ebola has been modified to permit repeat dosing of healthy volunteers at a dose of 0.24 mg/kg/day. [Tekmira Pharmaceuticals Corporation] Press Release Purdue Startup Is Developing Self-Assembling, Bioinstructive Collagen Materials for Research, Medical Applications A Purdue University researcher and entrepreneur is commercializing her laboratory’s innovative collagen formulations that self-assemble or polymerize to form fibrils that resemble those found in the body’s tissues. [Purdue University] Press Release Cytori and Lorem Vascular Receive Regulatory Clearance in China Cytori Therapeutics, Inc. announced that its exclusive licensee, Lorem Vascular, has been granted regulatory clearance for the Cytori Celution® System by the State Food and Drug Administration of the People’s Republic of China. [Cytori Therapeutics, Inc.] Press Release Editing HIV Out of Our Genome with CRISPR In an attempt to render latent HIV completely harmless, researchers at the University of Massachusetts Medical School are using Cas9/CRISPR, a powerful gene editing tool, to develop a novel technology that can potentially cut the DNA of the latent virus out of an infected cell. [University of Massachusetts Medical School] Press Release |
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