SciBX: Science-Business eXchange Contents: September 11 2014, Volume 7 / Issue 35

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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 Life Length. TABLE OF CONTENTS September 11 2014, Volume 7 / Issue 35 Analysis Cover Story Translational Notes Targets and Mechanisms The Distillery: Therapeutics Autoimmune disease Cancer Cardiovascular disease Endocrine/metabolic disease Hepatic disease Infectious disease Musculoskeletal disease Neurology The Distillery: Techniques Computational models Disease models Drug delivery Drug platforms 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 Clarity Pharmaceuticals.    Analysis Cover Story Top Kinase convergence on eIF4F Kai-Jye Lou doi:10.1038/scibx.2014.1030 Researchers in France have shown that the eIF4F complex is a hub in which resistance pathways for BRAF and MEK converge and suggest that combining eIF4F inhibitors with marketed melanoma drugs could circumvent resistance. Full Text | PDF Translational Notes Top A conversation with Miguel Barbosa Steve Edelson doi:10.1038/scibx.2014.1031 SciBX talked with Janssen's VP and head of immunology research and scientific partnership strategy about rethinking how to attack inflammatory bowel disease. Full Text | PDF Buckets of money for the brain Michael J. Haas and Kai-Jye Lou doi:10.1038/scibx.2014.1032 A few years ago, many companies threw in the towel on neurological diseases because poor understanding of their biology made R&D too risky. The field is finally receiving increased private and public funding, but some not-for-profit organizations contend the renewed interest still does not meet the diseases' societal burden. Full Text | PDF Targets and Mechanisms Top A chromatin target for ALL Chris Cain doi:10.1038/scibx.2014.1033 An NYU School of Medicine–GSK collaboration has shown that an inhibitor of the chromatin regulator JMJD3 could help treat T cell acute lymphoblastic leukemia and might avoid the safety pitfalls of other notch 1–targeted therapies. Full Text | PDF Distillery: Therapeutics Autoimmune disease Top IL-4 (BSF1); fibronectin extra domain A (FnEDA) doi:10.1038/scibx.2014.1034 Mouse studies suggest an antibody-IL-4 fusion protein could help treat RA. Full Text | PDF Cancer Top c-Myc (MYC); MYC associated factor X (MAX) doi:10.1038/scibx.2014.1035 In vitro and mouse studies have identified compounds that could inhibit the MYC-MAX interaction and help treat cancer. Full Text | PDF Epidermal growth factor receptor (EGFR) doi:10.1038/scibx.2014.1036 In vitro studies identified allosteric EGFR inhibitors that could help treat cancer. Full Text | PDF Not applicable doi:10.1038/scibx.2014.1037 Rodent, canine and human studies suggest intratumoral injection of spores from the attenuated strain of Clostridium novyi (C. novyi-NT) could help treat cancer. Full Text | PDF K-Ras (KRAS); microRNA-34a (miR-34a) doi:10.1038/scibx.2014.1038 Mouse studies suggest nanoparticle delivery of siRNAs targeting KRAS and miR-34a could help treat lung cancers. Full Text | PDF Cardiovascular disease Top Myosin heavy chain 7 cardiac muscle-β (MYH7) doi:10.1038/scibx.2014.1039 Mouse studies suggest a cluster of long noncoding RNAs (lncRNAs) encoded by Myh7 loci could help treat pathological cardiac hypertrophy. Full Text | PDF Ectonucleoside triphosphate diphosphohydrolase 3 (ENTPD3; CD39L3) doi:10.1038/scibx.2014.1040 In vitro and canine studies suggest engineered ENTPD3 can help prevent thrombosis. Full Text | PDF Endocrine/metabolic disease Top Forkhead box O1 (FOXO1) doi:10.1038/scibx.2014.1041 Mouse studies suggest FOXO1 inhibition could convert pancreatic δ cells into β cells to help treat diabetes. Full Text | PDF Defensin β1 (DEFB1) doi:10.1038/scibx.2014.1042 Patient sample studies suggest increasing DEFB1 levels could help treat male infertility. Full Text | PDF Hepatic disease Top Complement receptor 2 (CR2); CD59; complement C5b-9 membrane attack complex (MAC) doi:10.1038/scibx.2014.1043 Mouse studies suggest a CR2-CD59 fusion protein could help treat liver injury after transplantation surgery. Full Text | PDF Infectious disease Top Fc fragment of IgG receptor transporter-α (FCGRT; FCRN); HIV env doi:10.1038/scibx.2014.1044 Nonhuman primate studies suggest modifying therapeutic, broadly neutralizing antibodies against HIV to have increased FCRN affinity could help prevent HIV infection. Full Text | PDF Histone deacetylase (HDAC); CTLA-4 (CD152); BET bromodomain proteins; HIV env doi:10.1038/scibx.2014.1045 Mouse studies suggest combining broadly neutralizing antibodies (bNAbs) and inducers of HIV transcription could help treat HIV infection. Full Text | PDF HIV gp120; HIV gp41 doi:10.1038/scibx.2014.1046 Studies in human serum samples suggest broadly neutralizing antibodies (bNAbs) could help prevent HIV rebound after antiretroviral therapy. Full Text | PDF Musculoskeletal disease Top Dysferlin (DYSF) doi:10.1038/scibx.2014.1047 Studies in patients suggest proteasome inhibitors could help treat muscular dystrophies caused by DYSF mutations. Full Text | PDF Wingless-type MMTV integration site family member 4 (WNT4) doi:10.1038/scibx.2014.1048 Mouse studies suggest promoting WNT4 signaling could help prevent bone loss. Full Text | PDF Neurology Top Protein tyrosine phosphatase non-receptor type 5 (PTPN5; STEP) doi:10.1038/scibx.2014.1049 In vitro and mouse studies suggest benzopentathiepin-containing compounds could help treat AD. Full Text | PDF Chromosome 9 open reading frame 72 (C9orf72) doi:10.1038/scibx.2014.1050 In vitro studies suggest small molecules that bind C9orf72 repeats could help treat ALS in patients who carry the repeat. Full Text | PDF Calcineurin; α-synuclein (SNCA) doi:10.1038/scibx.2014.1051 Cell culture and animal studies suggest calcineurin inhibitors could help treat PD and other α-synucleinopathies. Full Text | PDF DNA (cytosine-5-)-methyltransferase 1 (DNMT1); 3-phosphoinositide dependent protein kinase-1 (PDPK1); aurora kinase A (AURKA; aurora-A); X inactive specific transcript (XIST); methyl CpG binding protein 2 (MECP2; RTT) doi:10.1038/scibx.2014.1052 Cell culture studies suggest small molecules that reactivate inactive X chromosomes could help treat Rett syndrome, which is linked to deficiencies in MECP2. Full Text | PDF Distillery: Techniques Computational models Top Computational model of synthetic lethality for predicting treatment responses to cancer therapy and identifying drugs for repurposing to treat cancer doi:10.1038/scibx.2014.1053 A computational model that predicts pairs of synthetically lethal (SL) genes could help guide treatment decisions in patients with cancer and identify drugs that could be repurposed to treat cancer. Full Text | PDF Disease models Top Bioengineered, functional cortical tissue doi:10.1038/scibx.2014.1054 Bioengineered, functional cortical tissue could help detect neurotoxicity and aid the discovery of therapies for neurological disorders. Full Text | PDF Drug delivery Top Annexin A1 (ANXA1)-mediated drug transport into tumors doi:10.1038/scibx.2014.1055 In vitro and mouse studies suggest targeting cancer therapeutics to ANXA1 could enable their active transport into tumors. Full Text | PDF Drug platforms Top Amphiphilic drug conjugate nanoparticles for cancer doi:10.1038/scibx.2014.1056 In vitro and rodent studies have identified nanoparticle-encapsulated amphiphilic drug conjugates that could help treat cancer. Full Text | PDF Exon 2 skipping to induce expression of an internal ribosome entry site (IRES)-driven isoform of dystrophin doi:10.1038/scibx.2014.1057 Exon 2 skipping could be used to induce dystrophin expression to improve muscle function in up to 6% of patients with Duchenne muscular dystrophy (DMD) with exon 2 duplications or 5′ mutations in the dystrophin gene. Full Text | PDF Pulmonary transplantation of macrophage progenitors to treat hereditary pulmonary alveolar proteinosis (herPAP) doi:10.1038/scibx.2014.1058 Mouse studies suggest pulmonary transplantation of macrophage progenitors could be used to treat herPAP, a rare condition characterized by abnormal protein accumulation in the lungs leading to impaired lung function and decreased lifespan. Full Text | PDF Top

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