SciBX: Science-Business eXchange Contents: October 24 2013, Volume 6 / Issue 41

TABLE OF CONTENTS October 24 2013, Volume 6 / Issue 41 Analysis Cover Story Translational Notes The Distillery: Therapeutics Autoimmune disease Cancer Dermatology Endocrine/metabolic disease Infectious disease Neurology The Distillery: Techniques Chemistry Computational models Disease models Drug platforms Imaging Markers Advertisement Biopharma Dealmakers A supplement to Nature Biotechnology and Nature Reviews Drug Discovery Check out the latest issue of Biopharma Dealmakers, which includes the following features: - Diseases related to the immune system touch off a spectrum of valuable business models - Oncology patent cliff makes way for younger biotechs  Click here to read the issue for FREE! SciBX: Science-Business eXchange Collection on Antibiotic Resistance This special Collection from SciBX: Science-Business eXchange provides an overview of the state of affairs on antibiotic resistance from the scientific, business and regulatory perspectives.  Click here to access the Collection for free! Produced with support from: Cempra Inc., Cubist Pharmaceuticals Inc., Durata Therapeutics Inc., Polyphor Ltd.   Analysis Cover Story Top A platform for RNA Amy Donner doi:10.1038/scibx.2013.1151 An information explosion on the numbers and mechanisms of functional RNAs has spurred oligonucleotide-based therapeutic development. Pharmas and big biotechs are carving up the space via partnerships, adding multiple nucleic acid–based technologies to their drug discovery toolboxes. The newest kids on the block are noncoding RNAs such as microRNAs and long noncoding RNAs, which can selectively turn on expression of a given gene and thus achieve therapeutic outcomes that no other drug platform can. Full Text | PDF Translational Notes Top Driving CAR-based cellular therapies Tracey Baas doi:10.1038/scibx.2013.1152 Adoptive T cell therapies recently entered the limelight owing to dramatic clinical results achieved with their latest incarnation, chimeric antigen receptor–based cell therapies. Novartis' 2012 CAR deal with the University of Pennsylvania signaled the start of a land grab. Full Text | PDF Synaptic synopsis C. Simone Fishburn and Lev Osherovich doi:10.1038/scibx.2013.1153 The early promise that neurotransmitter research would unlock the door for neurological diseases has devolved into sagging interest from several pharmas following repeated failures in clinical trials. Genetic findings that point to new targets in the synapse are reviving commercial interest in autism, schizophrenia and depression. Full Text | PDF Distillery: Therapeutics Autoimmune disease Top Porphyromonas gingivalis peptidyl arginine deiminase (PgPADI; PgPAD) doi:10.1038/scibx.2013.1154 Mouse studies suggest inhibiting PgPAD could help treat RA. Full Text | PDF Cancer Top MDS1 and EVI1 complex locus (MECOM) doi:10.1038/scibx.2013.1155 Cell-based and mouse studies suggest inhibiting MECOM could help treat AML. Full Text | PDF Amphiregulin (AREG); monocyte chemoattractant protein-1 (MCP-1; CCL2); chemokine CC motif ligand 7 (CCL7; MCP3; SCYA6); CC chemokine receptor 1 (CCR1; CD191) doi:10.1038/scibx.2013.1156 Mouse and comparative genomics studies suggest targeting combinations of fibroblast-secreted factors could help treat breast cancer. Full Text | PDF G protein–coupled receptor 116 (GPR116) doi:10.1038/scibx.2013.1157 Cell culture and mouse studies suggest antagonizing GPR116 could be useful for treating metastatic breast cancer. Full Text | PDF Oral-facial-digital syndrome 1 (OFD1) doi:10.1038/scibx.2013.1158 In vitro studies suggest inhibiting OFD1 could be useful for treating ciliopathies and cancers associated with aberrant ciliogenesis. Full Text | PDF PD-1 receptor (PDCD1; PD-1; CD279); programmed cell death 1 ligand 1 (CD274 molecule; PD-L1; B7-H1); epidermal growth factor receptor (EGFR) doi:10.1038/scibx.2013.1159 Primary tumor and mouse studies suggest inhibitors of PD-1 and PD-L1 could help treat lung cancer harboring EGFR mutations. Full Text | PDF Androgen receptor; protein kinase DNA-activated catalytic polypeptide (PRKDC; DNAPK) doi:10.1038/scibx.2013.1160 Cell culture and mouse studies suggest inhibiting components of the DNA damage response could help treat prostate cancer. Full Text | PDF Dermatology Top IL-17A doi:10.1038/scibx.2013.1161 Mouse studies suggest IL-17A could help treat dermal wounds and that wound healing in patients receiving IL-17 inhibitors should be monitored. Full Text | PDF Endocrine/metabolic disease Top G protein–coupled receptor 171 (GPR171) doi:10.1038/scibx.2013.1162 Cell culture and mouse studies suggest modulating GPR171 activity could help treat metabolic disorders. Full Text | PDF Infectious disease Top Activation-induced cytidine deaminase (AICDA; AID); IL-1β; tumor necrosis factor-α (TNF-α) doi:10.1038/scibx.2013.1163 In vitro studies suggest activating AID could help prevent HBV infection. Full Text | PDF Mycobacterium tuberculosis L-asparagine permease AnsP1 (AnsP1) doi:10.1038/scibx.2013.1164 In vitro and mouse studies suggest inhibiting AnsP1 could help treat tuberculosis. Full Text | PDF Neurology Top Leukocyte immunoglobulin-like receptor subfamily B member 2 (LILRB2; LIR2) doi:10.1038/scibx.2013.1165 Cell culture and mouse studies suggest blocking LILRB2 could help treat AD. Full Text | PDF Nav1.9 (SCN11A) doi:10.1038/scibx.2013.1166 Gene sequencing, cell culture and mouse studies suggest antagonizing Nav1.9 could help treat congenital analgesia. Full Text | PDF Distillery: Techniques Chemistry Top Trifluoromethylation chemistry for PET radioligand synthesis doi:10.1038/scibx.2013.1167 A synthetic chemistry method could help generate PET imaging probes using common pharmacophores. Full Text | PDF Computational models Top Algorithm to predict risk of developing neutralizing antibodies to factor VIII doi:10.1038/scibx.2013.1168 A computational algorithm to predict the risk of developing factor VIII–neutralizing inhibitory antibodies could help guide the treatment of patients with hemophilia A. Full Text | PDF Disease models Top A mouse model of schizophrenia involving disruption of GABAA receptor clustering doi:10.1038/scibx.2013.1169 Mice with disrupted clustering of GABAA receptors could provide a model of schizophrenia. Full Text | PDF Chemokine CX3C motif receptor 1 (CX3CR1) promoter–dependent gene expression in microglia doi:10.1038/scibx.2013.1170 A microglia-specific expression system could help study the role of such cells in various CNS diseases. Full Text | PDF Full-length infectious cDNA clone of Middle East respiratory syndrome coronavirus (MERS-CoV) doi:10.1038/scibx.2013.1171 A red fluorescent protein (RFP)-expressing, recombinant, full-length, infectious cDNA clone of MERS-CoV could be used to characterize viral pathogenesis and screen for antivirals. Full Text | PDF Drug platforms Top Crystal structures of Staphylococcus aureus penicillin binding protein 2a (PBP2a) doi:10.1038/scibx.2013.1172 In vitro studies identified an allosteric site on PBP2a that could aid the development of new antibiotics to treat methicillin-resistant S. aureus (MRSA) infection. Full Text | PDF Donor-derived, CD19 +, virus-specific T cells to treat B cell cancers doi:10.1038/scibx.2013.1173 Donor-derived, CD19+, virus-specific T cells could be used to treat B cell cancers and associated viral infections. Full Text | PDF HIV-1 Env epitopes engineered to enhance the production of broadly neutralizing antibodies doi:10.1038/scibx.2013.1174 A recombinant protein containing a combination of epitopes derived from the HIV-1 viral envelope glycoprotein Env could aid the development of an HIV vaccine. Full Text | PDF In vivo reprogramming of astrocytes to neuroblasts doi:10.1038/scibx.2013.1175 Mouse studies suggest proliferating neuroblasts generated in vivo from reprogrammed astrocytes could help treat CNS injury or degeneration. Full Text | PDF Imaging Top Detection of melanoma tumors with PET imaging using an 18F-labeled, melanin-binding small molecule doi:10.1038/scibx.2013.1176 A PET agent derived from a melanin-binding small molecule could be useful for detecting metastatic melanoma tumors. Full Text | PDF Markers Top Major histocompatibility complex class I (MHC)-associated phosphopeptides to predict clinical outcome in leukemia doi:10.1038/scibx.2013.1177 Tumor-associated phosphopeptides recognized by MHC could help predict outcomes for patients with leukemia. Full Text | PDF Top Advertisement Nature Publishing Group and Relay Technology Management present: The Epigenetics Target Explorer  Click here to access this free online tool and the accompanying article in Nature Reviews Drug Discovery.

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