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Roche Kulmbach - Center of Excellence for RNA Therapeutics

Roche Kulmbach site

Roche Kulmbach GmbH is Roche's Center of Excellence for the discovery of novel therapeutics that belong to the family of ribonucleic acids (RNA). As a part of Roche's Pharma Division and, as a Center of Excellence, the Kulmbach site is currently focused on therapeutics based on the principle of RNA interference (RNAi). RNAi is a natural process to selectively silence genes in the body and is mediated by small interfering RNAs (siRNAs). Roche Kulmbach uses synthetic siRNAs to specifically silence genes that are associated with disease. This universal approach is being applied at Roche in the discovery of differentiated medicines for the treatment of oncological, respiratory and metabolic diseases.

 

First RNAi Therapeutics Company World-Wide

Scientists at Roche Kulmbach have accumulated a unique expertise and knowledge regarding the discovery and development of RNAi therapeutics. As the first company in the world to focus on RNAi therapeutics, Roche Kulmbach GmbH was started in June 2000 by Stefan Limmer and Roland Kreutzer under the name of Ribopharma AG. The company was a spin-off of the University of Bayreuth, Germany, and the two founders filed fundamental patents in the field of RNAi. In July 2003, Ribopharma AG merged with Alnylam Pharmaceuticals Inc. to create the leading RNAi therapeutics company. In August 2007 Roche Kulmbach GmbH was spun-off from Alnylam Europe AG. The company currently has more than 50 employees and is located in state-of-the-art facilities in Kulmbach, Germany. The scientists at the Center of Excellence in Kulmbach closely interact with Roche colleagues at research sites in Penzberg, Germany; Basel, Switzerland; and Nutley and Madison in the U.S.

Selected publications with contributions from Kulmbach scientists

  • siRNA Down-regulation of the PATZ1 Gene in Human Glioma Cells Increases Their Sensitivity to Apoptotic Stimuli. Tritz R, Mueller BM, Hickey MJ, Lin AH, Gomez GG, Hadwiger P, Sah DW, Muldoon L, Neuwelt EA, Kruse CA. Cancer Ther. 2008;6(B):865-876. View Abstract on PubMed
  • Host scavenger receptor SR-BI plays a dual role in the establishment of malaria parasite liver infection. Rodrigues CD, Hannus M, Prudêncio M, Martin C, Gonçalves LA, Portugal S, Epiphanio S, Akinc A, Hadwiger P, Jahn-Hofmann K, Röhl I, van Gemert GJ, Franetich JF, Luty AJ, Sauerwein R, Mazier D, Koteliansky V, Vornlocher HP, Echeverri CJ, Mota MM. Cell Host Microbe. 2008 Sep 11;4(3):271-82. View Abstract on PubMed
  • Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates. Frank-Kamenetsky M, Grefhorst A, Anderson NN, Racie TS, Bramlage B, Akinc A, Butler D, Charisse K, Dorkin R, Fan Y, Gamba-Vitalo C, Hadwiger P, Jayaraman M, John M, Jayaprakash KN, Maier M, Nechev L, Rajeev KG, Read T, Röhl I, Soutschek J, Tan P, Wong J, Wang G, Zimmermann T, de Fougerolles A, Vornlocher HP, Langer R, Anderson DG, Manoharan M, Koteliansky V, Horton JD, Fitzgerald K. Proc Natl Acad Sci U S A. 2008 Aug 19;105(33):11915-20. Epub 2008 Aug 11. View Abstract on PubMed
  • A combinatorial library of lipid-like materials for delivery of RNAi therapeutics. Akinc A, Zumbuehl A, Goldberg M, Leshchiner ES, Busini V, Hossain N, Bacallado SA, Nguyen DN, Fuller J, Alvarez R, Borodovsky A, Borland T, Constien R, de Fougerolles A, Dorkin JR, Narayanannair Jayaprakash K, Jayaraman M, John M, Koteliansky V, Manoharan M, Nechev L, Qin J, Racie T, Raitcheva D, Rajeev KG, Sah DW, Soutschek J, Toudjarska I, Vornlocher HP, Zimmermann TS, Langer R, Anderson DG. Nat Biotechnol. 2008 May;26(5):561-9. Epub 2008 Apr 27. View Abstract on PubMed
  • siRNA knock-down of mutant torsinA restores processing through secretory pathway in DYT1 dystonia cells. Hewett JW, Nery FC, Niland B, Ge P, Tan P, Hadwiger P, Tannous BA, Sah DW, Breakefield XO. Hum Mol Genet. 2008 May 15;17(10):1436-45. Epub 2008 Feb 7. View Abstract on PubMed
  • Effective RNAi-mediated gene silencing without interruption of the endogenous microRNA pathway. John M, Constien R, Akinc A, Goldberg M, Moon YA, Spranger M, Hadwiger P, Soutschek J, Vornlocher HP, Manoharan M, Stoffel M, Langer R, Anderson DG, Horton JD, Koteliansky V, Bumcrot D. Nature. 2007 Oct 11;449(7163):745-7. Epub 2007 Sep 26. View Abstract on PubMed
  • Bcl-2 downregulation sensitizes nonsmall cell lung cancer cells to cisplatin, but not to docetaxel. Losert D, Pratscher B, Soutschek J, Geick A, Vornlocher HP, Müller M, Wacheck V. Anticancer Drugs. 2007 Aug;18(7):755-61. View Abstract on PubMed
  • Interfering with disease: a progress report on siRNA-based therapeutics. de Fougerolles A, Vornlocher HP, Maraganore J, Lieberman J. Nat Rev Drug Discov. 2007 Jun;6(6):443-53. Review. View Abstract on PubMed
  • Hyperoxia causes angiopoietin 2-mediated acute lung injury and necrotic cell death. Bhandari V, Choo-Wing R, Lee CG, Zhu Z, Nedrelow JH, Chupp GL, Zhang X, Matthay MA, Ware LB, Homer RJ, Lee PJ, Geick A, de Fougerolles AR, Elias JA. Nat Med. 2006 Nov;12(11):1286-93. Epub 2006 Nov 5. View Abstract on PubMed
  • Effective silencing of EGFR with RNAi demonstrates non-EGFR dependent proliferation of glioma cells. Vollmann A, Vornlocher HP, Stempfl T, Brockhoff G, Apfel R, Bogdahn U. Int J Oncol. 2006 Jun;28(6):1531-42. View Abstract on PubMed
  • RNAi-mediated gene silencing in non-human primates.  Zimmermann TS, Lee AC, Akinc A, Bramlage B, Bumcrot D, Fedoruk MN, Harborth J, Heyes JA, Jeffs LB, John M, Judge AD, Lam K, McClintock K, Nechev LV, Palmer LR, Racie T, Röhl I, Seiffert S, Shanmugam S, Sood V, Soutschek J, Toudjarska I, Wheat AJ, Yaworski E, Zedalis W, Koteliansky V, Manoharan M, Vornlocher HP, MacLachlan I. Nature. 2006 May 4;441(7089):111-4. Epub 2006 Mar 26. View Abstract on PubMed
  • Antibody-directed cell-type-specific delivery of siRNA. Vornlocher HP. Trends Mol Med. 2006 Jan;12(1):1-3. Epub 2005 Nov 14. View Abstract on PubMed
  • Targeting MLL-AF4 with short interfering RNAs inhibits clonogenicity and engraftment of t(4;11)-positive human leukemic cells. Thomas M, Gessner A, Vornlocher HP, Hadwiger P, Greil J, Heidenreich O. Blood. 2005 Nov 15;106(10):3559-66. Epub 2005 Jul 26. View Abstract on PubMed
  • RNA interference in vivo: toward synthetic small inhibitory RNA-based therapeutics.de Fougerolles A, Manoharan M, Meyers R, Vornlocher HP. Methods Enzymol. 2005;392:278-96. View Abstract on PubMed
  • Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs. Soutschek J, Akinc A, Bramlage B, Charisse K, Constien R, Donoghue M, Elbashir S, Geick A, Hadwiger P, Harborth J, John M, Kesavan V, Lavine G, Pandey RK, Racie T, Rajeev KG, Röhl I, Toudjarska I, Wang G, Wuschko S, Bumcrot D, Koteliansky V, Limmer S, Manoharan M, Vornlocher HP. Nature. 2004 Nov 11;432(7014):173-8. View Abstract on PubMed
  • Steroid and lipid conjugates of siRNAs to enhance cellular uptake and gene silencing in liver cells. Lorenz C, Hadwiger P, John M, Vornlocher HP, Unverzagt C. Bioorg Med Chem Lett. 2004 Oct 4;14(19):4975-7. View Abstract on PubMed
  • Small interfering RNA targeting bcl-2 sensitizes malignant melanoma. Wacheck V, Losert D, Günsberg P, Vornlocher HP, Hadwiger P, Geick A, Pehamberger H, Müller M, Jansen B. Oligonucleotides. 2003;13(5):393-400. View Abstract on PubMed
  • The apparent uptake of fluorescently labeled siRNAs by electroporated cells depends on the fluorochrome. Dunne J, Drescher B, Riehle H, Hadwiger P, Young BD, Krauter J, Heidenreich O. Oligonucleotides. 2003;13(5):375-80.  View Abstract on PubMed
  • Inhibition of bcr-abl gene expression by small interfering RNA sensitizes for imatinib mesylate (STI571). Wohlbold L, van der Kuip H, Miething C, Vornlocher HP, Knabbe C, Duyster J, Aulitzky WE. Blood. 2003 Sep 15;102(6):2236-9. Epub 2003 May 15. View Abstract on PubMed
  • Selective inhibition of FLICE-like inhibitory protein expression with small interfering RNA oligonucleotides is sufficient to sensitize tumor cells for TRAIL-induced apoptosis. Siegmund D, Hadwiger P, Pfizenmaier K, Vornlocher HP, Wajant H. Mol Med. 2002 Nov;8(11):725-32.  View Abstract on PubMed
  • Gene silencing by RNAi in mammalian cells. John M, Geick A, Hadwiger P, Vornlocher HP, Heidenreich O. Curr Protoc Mol Biol. 2003 May;Chapter 26:Unit 26.2. View Abstract on PubMed
  • AML1/MTG8 oncogene suppression by small interfering RNAs supports myeloid differentiation of t(8;21)-positive leukemic cells. Heidenreich O, Krauter J, Riehle H, Hadwiger P, John M, Heil G, Vornlocher HP, Nordheim A. Blood. 2003 Apr 15;101(8):3157-63. Epub 2002 Dec 12.  View Abstract on PubMed