SciBX: Science-Business eXchange Contents: October 16 2014, Volume 7 / Issue 40

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Advertisement Biopharma Dealmakers A supplement to Nature Biotechnology and Nature Reviews Drug Discovery The September 2014 issue of Biopharma Dealmakers showcases companies with partnering opportunities. This week, find out about how you can collaborate with Covance   TABLE OF CONTENTS October 16 2014, Volume 7 / Issue 40 Analysis Cover Story Translational Notes Targets and Mechanisms The Distillery: Therapeutics Cancer Cardiovascular disease Endocrine/metabolic disease Infectious disease Musculoskeletal disease Neurology The Distillery: Techniques Assays and screens Disease models Drug platforms Imaging Markers Advertisement Biopharma Dealmakers A supplement to Nature Biotechnology and Nature Reviews Drug Discovery The September 2014 issue of Biopharma Dealmakers showcases companies with partnering opportunities. This week, find out about how you can collaborate with AnaptysBio Inc.    Analysis Cover Story Top DisABLing imatinib Kai-Jye Lou doi:10.1038/scibx.2014.1167 Upregulation of PKCη has been found to underlie some forms of imatinib resistance in CML, and the discovery could provide new targets for intervention. Full Text | PDF Translational Notes Top Translational tidbits Michael J. Haas, Stephen Parmley, Kai-Jye Lou and Lauren Martz doi:10.1038/scibx.2014.1168 The NIH pledges almost $100 million for big data programs; CRUK and MedImmune join forces in Cambridge, U.K.; UNC's research center gets a lifetime endowment of $15 million per year; MS benefits from a new PPP. Full Text | PDF New CAR shopping Steve Edelson doi:10.1038/scibx.2014.1169 Kite Pharma's Arie Belldegrun says that target selection is the most important and difficult aspect of early stage CAR T cell research. Full Text | PDF Targets and Mechanisms Top Alnylam interrupts preeclampsia Michael J. Haas doi:10.1038/scibx.2014.1170 Alnylam has generated an siRNA conjugate that blocks angiotensin-induced hypertension without causing fetal toxicity in a rat model of preeclampsia. Full Text | PDF Distillery: Therapeutics Cancer Top Breast cancer 1 early onset (BRCA1) doi:10.1038/scibx.2014.1171 In vitro and mouse studies suggest small molecules that promote DNA repair could help prevent progression of BRCA1+ cancers. Full Text | PDF HER2 (EGFR2; ErbB2; neu) doi:10.1038/scibx.2014.1172 In vitro and mouse studies suggest modified versions of the antibody-drug conjugate trastuzumab-DM1 could help treat breast cancer. Full Text | PDF G protein–coupled receptor 126 (GPR126); VEGF receptor 2 (KDR/Flk-1; VEGFR-2) doi:10.1038/scibx.2014.1173 In vitro, mouse and zebrafish studies suggest inhibiting GPR126 could help treat cancer and other disorders associated with increased angiogenesis. Full Text | PDF Tubulin doi:10.1038/scibx.2014.1174 In vitro and mouse studies identified derivatives of the plant-derived compound millepachine that could help treat cancer. Full Text | PDF γ-secretase doi:10.1038/scibx.2014.1175 Studies in human samples, cell culture and mice suggest inhibiting notch signaling could help treat esophageal cancer. Full Text | PDF Branched-chain amino acids (BCAAs) doi:10.1038/scibx.2014.1176 Studies in human samples and mice suggest monitoring circulating BCAA levels could enable early diagnosis of pancreatic ductal adenocarcinoma (PDAC). Full Text | PDF Cardiovascular disease Top Not applicable doi:10.1038/scibx.2014.1177 Mouse studies suggest compounds that scavenge γ-ketoaldehydes (isoketals) could help treat hypertension. Full Text | PDF Vanin 1 (VNN1); adrenergic receptor β1 (ADRB1) doi:10.1038/scibx.2014.1178 Genetic analysis and cell culture studies suggest inhibiting VNN1 could help prevent hypertension. Full Text | PDF Endocrine/metabolic disease Top Alanine-glyoxylate aminotransferase (AGXT) doi:10.1038/scibx.2014.1179 In vitro studies suggest dequalinium chloride (DECA) could help treat primary hyperoxaluria (PH1), a disease caused by AGXT mutations. Full Text | PDF α-L-iduronidase (IDUA) doi:10.1038/scibx.2014.1180 Feline studies suggest liver-directed IDUA gene therapy could help treat mucopolysaccharidosis I, which is caused by IDUA deficiency and characterized by pathological accumulation of glycosaminoglycans (GAGs) in various tissues. Full Text | PDF N-acetylglucosaminidase-α (NAGLU); insulin-like growth factor-2 (IGF-2); mannose-6-phosphate receptor (M6P receptor) doi:10.1038/scibx.2014.1181 Mouse studies suggest a NAGLU-IGF-2 fusion protein could help treat mucopolysaccharidosis IIIB and have improved properties over unfused recombinant NAGLU. Full Text | PDF Infectious disease Top HIV gp41 doi:10.1038/scibx.2014.1182 In vitro studies suggest conjugates of sapogenins, a class of antiviral natural products, and a peptide from Fuzeon enfuvirtide could help treat HIV/AIDS. Full Text | PDF VEEVgp1 non-structural polyprotein precursor P1234 (VEEVgp1) doi:10.1038/scibx.2014.1183 In vitro and mouse studies have identified quinazolinone derivatives that could help treat infections with Venezuelan equine encephalitis virus (VEEV) and related alphaviruses. Full Text | PDF Musculoskeletal disease Top Mucolipin 1 (MCOLN1; ML4) doi:10.1038/scibx.2014.1184 Mouse studies suggest enhancing ML4 activity could help treat muscular dystrophies. Full Text | PDF Fibroblast growth factor receptor 3 (FGFR3; CD333) doi:10.1038/scibx.2014.1185 In vitro and mouse studies suggest statins could help treat achondroplasia, thanatophoric dysplasia type 1 (TD1) and other skeletal dysplasias caused by FGFR3 mutations. Full Text | PDF Neurology Top Inhibitor of κ-light polypeptide gene enhancer in B cells kinase-β (IKBKB; IKK2); β-amyloid (Aβ) doi:10.1038/scibx.2014.1186 Mouse studies suggest inhibiting IKK2 in myeloid cells could help treat AD. Full Text | PDF Peroxisome proliferation–activated receptor-γ coactivator 1α (PPARGC1A; PGC-1α) doi:10.1038/scibx.2014.1187 Mouse studies suggest increasing skeletal muscle PGC-1α expression could help treat chronic stress–induced depression. Full Text | PDF Distillery: Techniques Assays and screens Top In vitro assay to screen for new inhibitors of leucine-rich repeat kinase 2 (LRRK2) doi:10.1038/scibx.2014.1188 An in vitro kinase assay could be useful in high throughput screens to identify new selective inhibitors of LRRK2 to treat Parkinson's disease (PD). Full Text | PDF Disease models Top Mouse models of non–small cell lung cancer (NSCLC) based on serial orthotopic transplantation of tumor tissue doi:10.1038/scibx.2014.1189 Mouse studies suggest serial passages of tumor tissues in nude mice could help model NSCLC. Full Text | PDF Patient-derived prostate organoids as prostate cancer models doi:10.1038/scibx.2014.1190 Prostate organoids derived from patient tumor cells could serve as models of prostate cancer. Full Text | PDF Drug platforms Top 3D matrix platform for generating mAbs to treat breast cancer doi:10.1038/scibx.2014.1191 A 3D matrix platform could be useful for generating mAbs to help treat breast cancer. Full Text | PDF Glycan recognition on HIV env by broadly neutralizing antibodies (bNAbs) doi:10.1038/scibx.2014.1192 Crystal structure analysis of bNAbs complexed with glycans of HIV env could guide the development of antibody vaccines against HIV infection. Full Text | PDF Small molecule–mediated ex vivo expansion of long-term repopulating progenitor cells in cord blood–derived hematopoietic stem cells (HSCs) doi:10.1038/scibx.2014.1193 A small molecule could enhance the number of HSCs with long-term repopulating capacity in cord blood–derived HSC transplants. Full Text | PDF Imaging Top Fluorescent PET imaging probe for hypochlorus acid (HClO) doi:10.1038/scibx.2014.1194 In vitro studies identified a fluorescent probe for HClO that could be useful as a PET imaging agent to detect cancer. Full Text | PDF Markers Top Gelsolin (GSN) as a marker for cisplatin sensitivity and patient survival in ovarian cancer doi:10.1038/scibx.2014.1195 Studies in patient samples and cell culture suggest GSN could be a marker for cisplatin sensitivity and poor survival in ovarian cancer. Full Text | PDF Top

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