Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis
Coelho, Teresa, Adams, David, Silva, Ana, Lozeron, Pierre, Hawkins, Philip N, Mant, Timothy, Perez, Javier, Chiesa, Joseph, Warrington, Steve, Tranter, Elizabeth, Munisamy, Malathy, Falzone, Rick, Harrop, Jamie, Cehelsky, Jeffrey, Bettencourt, Brian R, Geissler, Mary, Butler, James S, Sehgal, Alfica, Meyers, Rachel E, Chen, Qingmin, Borland, Todd, Hutabarat, Renta M, Clausen, Valerie A, Alvarez, Rene, Fitzgerald, Kevin, Gamba-Vitalo, Christina, Nochur, Saraswathy V, Vaishnaw, Akshay K, Sah, Dinah W.Y, Gollob, Jared A, Suhr, Ole B
Published in The New England journal of medicine (29.08.2013)
Published in The New England journal of medicine (29.08.2013)
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Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial
Fitzgerald, Kevin, Dr, Frank-Kamenetsky, Maria, PhD, Shulga-Morskaya, Svetlana, MSc, Liebow, Abigail, BS, Bettencourt, Brian R, PhD, Sutherland, Jessica E, PhD, Hutabarat, Renta M, PhD, Clausen, Valerie A, PhD, Karsten, Verena, PhD, Cehelsky, Jeffrey, MBA, Nochur, Saraswathy V, PhD, Kotelianski, Victor, MD, Horton, Jay, MD, Mant, Timothy, MD, Chiesa, Joseph, MD, Ritter, James, MD, Munisamy, Malathy, MD, Vaishnaw, Akshay K, MD, Gollob, Jared A, MD, Simon, Amy, MD
Published in The Lancet (British edition) (04.01.2014)
Published in The Lancet (British edition) (04.01.2014)
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Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate
Zimmermann, Tracy S., Karsten, Verena, Chan, Amy, Chiesa, Joseph, Boyce, Malcolm, Bettencourt, Brian R., Hutabarat, Renta, Nochur, Saraswathy, Vaishnaw, Akshay, Gollob, Jared
Published in Molecular therapy (04.01.2017)
Published in Molecular therapy (04.01.2017)
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Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc-siRNA conjugates
Nair, Jayaprakash K, Attarwala, Husain, Sehgal, Alfica, Wang, Qianfan, Aluri, Krishna, Zhang, Xuemei, Gao, Minggeng, Liu, Ju, Indrakanti, Ramesh, Schofield, Sally, Kretschmer, Philip, Brown, Christopher R, Gupta, Swati, Willoughby, Jennifer L S, Boshar, Julie A, Jadhav, Vasant, Charisse, Klaus, Zimmermann, Tracy, Fitzgerald, Kevin, Manoharan, Muthiah, Rajeev, Kallanthottathil G, Akinc, Akin, Hutabarat, Renta, Maier, Martin A
Published in Nucleic acids research (02.11.2017)
Published in Nucleic acids research (02.11.2017)
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An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia
Sehgal, Alfica, Barros, Scott, Ivanciu, Lacramioara, Cooley, Brian, Qin, June, Racie, Tim, Hettinger, Julia, Carioto, Mary, Jiang, Yongfeng, Brodsky, Josh, Prabhala, Harsha, Zhang, Xuemei, Attarwala, Husain, Hutabarat, Renta, Foster, Don, Milstein, Stuart, Charisse, Klaus, Kuchimanchi, Satya, Maier, Martin A, Nechev, Lubo, Kandasamy, Pachamuthu, Kel'in, Alexander V, Nair, Jayaprakash K, Rajeev, Kallanthottathil G, Manoharan, Muthiah, Meyers, Rachel, Sorensen, Benny, Simon, Amy R, Dargaud, Yesim, Negrier, Claude, Camire, Rodney M, Akinc, Akin
Published in Nature medicine (01.05.2015)
Published in Nature medicine (01.05.2015)
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First-in-humans trial of an RNA interference therapeutic targeting VEGF and KSP in cancer patients with liver involvement
Tabernero, Josep, Shapiro, Geoffrey I, LoRusso, Patricia M, Cervantes, Andres, Schwartz, Gary K, Weiss, Glen J, Paz-Ares, Luis, Cho, Daniel C, Infante, Jeffrey R, Alsina, Maria, Gounder, Mrinal M, Falzone, Rick, Harrop, Jamie, White, Amy C Seila, Toudjarska, Iva, Bumcrot, David, Meyers, Rachel E, Hinkle, Gregory, Svrzikapa, Nenad, Hutabarat, Renta M, Clausen, Valerie A, Cehelsky, Jeffrey, Nochur, Saraswathy V, Gamba-Vitalo, Christina, Vaishnaw, Akshay K, Sah, Dinah W Y, Gollob, Jared A, Burris, 3rd, Howard A
Published in Cancer discovery (01.04.2013)
Published in Cancer discovery (01.04.2013)
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First-in-human evaluation of anti von Willebrand factor therapeutic aptamer ARC1779 in healthy volunteers
Gilbert, James C, DeFeo-Fraulini, Tia, Hutabarat, Renta M, Horvath, Christopher J, Merlino, Patricia G, Marsh, H Nicholas, Healy, Judith M, Boufakhreddine, Sleiman, Holohan, Thomas V, Schaub, Robert G
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Published in Circulation (New York, N.Y.) (04.12.2007)
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Cytokine traps: multi-component, high-affinity blockers of cytokine action
Stahl, Neil, Economides, Aris N, Carpenter, Laura Rocco, Rudge, John S, Wong, Vivien, Koehler-Stec, Ellen M, Hartnett, Christopher, Pyles, Erica A, Xu, Xiaobing, Daly, Thomas J, Young, Michael R, Fandl, James P, Lee, Frank, Carver, Scott, McNay, Jennifer, Bailey, Kevin, Ramakanth, Swayampakula, Hutabarat, Renta, Huang, Tammy T, Radziejewski, Czeslaw, Yancopoulos, George D
Published in Nature medicine (01.01.2003)
Published in Nature medicine (01.01.2003)
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In vivo quantification of formulated and chemically modified small interfering RNA by heating-in-Triton quantitative reverse transcription polymerase chain reaction (HIT qRT-PCR)
Landesman, Yosef, Svrzikapa, Nenad, Cognetta, 3rd, Armand, Zhang, Xuemei, Bettencourt, Brian R, Kuchimanchi, Satya, Dufault, Keri, Shaikh, Sarfraz, Gioia, Maple, Akinc, Akin, Hutabarat, Renta, Meyers, Rachel
Published in Silence (23.08.2010)
Published in Silence (23.08.2010)
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Multivalent N‑Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing
Nair, Jayaprakash K., Willoughby, Jennifer L. S., Chan, Amy, Charisse, Klaus, Alam, Md. Rowshon, Wang, Qianfan, Hoekstra, Menno, Kandasamy, Pachamuthu, Kel’in, Alexander V., Milstein, Stuart, Taneja, Nate, O’Shea, Jonathan, Shaikh, Sarfraz, Zhang, Ligang, van der Sluis, Ronald J., Jung, Michael E., Akinc, Akin, Hutabarat, Renta, Kuchimanchi, Satya, Fitzgerald, Kevin, Zimmermann, Tracy, van Berkel, Theo J. C., Maier, Martin A., Rajeev, Kallanthottathil G., Manoharan, Muthiah
Published in Journal of the American Chemical Society (10.12.2014)
Published in Journal of the American Chemical Society (10.12.2014)
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Biodegradable Lipids Enabling Rapidly Eliminated Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics
Maier, Martin A, Jayaraman, Muthusamy, Matsuda, Shigeo, Liu, Ju, Barros, Scott, Querbes, William, Tam, Ying K, Ansell, Steven M, Kumar, Varun, Qin, June, Zhang, Xuemei, Wang, Qianfan, Panesar, Sue, Hutabarat, Renta, Carioto, Mary, Hettinger, Julia, Kandasamy, Pachamuthu, Butler, David, Rajeev, Kallanthottathil G, Pang, Bo, Charisse, Klaus, Fitzgerald, Kevin, Mui, Barbara L, Du, Xinyao, Cullis, Pieter, Madden, Thomas D, Hope, Michael J, Manoharan, Muthiah, Akinc, Akin
Published in Molecular therapy (01.08.2013)
Published in Molecular therapy (01.08.2013)
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A status report on RNAi therapeutics
Vaishnaw, Akshay K, Gollob, Jared, Gamba-Vitalo, Christina, Hutabarat, Renta, Sah, Dinah, Meyers, Rachel, de Fougerolles, Tony, Maraganore, John
Published in Silence (08.07.2010)
Published in Silence (08.07.2010)
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Metabolically Stable Anomeric Linkages Containing GalNAc–siRNA Conjugates: An Interplay among ASGPR, Glycosidase, and RISC Pathways
Kandasamy, Pachamuthu, Mori, Shohei, Matsuda, Shigeo, Erande, Namrata, Datta, Dhrubajyoti, Willoughby, Jennifer L. S., Taneja, Nate, O’Shea, Jonathan, Bisbe, Anna, Manoharan, Rajar M., Yucius, Kristina, Nguyen, Tuyen, Indrakanti, Ramesh, Gupta, Swati, Gilbert, Jason A., Racie, Tim, Chan, Amy, Liu, Ju, Hutabarat, Renta, Nair, Jayaprakash K., Charisse, Klaus, Maier, Martin A., Rajeev, Kallanthottathil G., Egli, Martin, Manoharan, Muthiah
Published in Journal of medicinal chemistry (23.02.2023)
Published in Journal of medicinal chemistry (23.02.2023)
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A randomised pilot trial of the anti-von Willebrand factor aptamer ARC1779 in patients with type 2b von Willebrand disease
Jilma, Bernd, Paulinska, Petra, Jilma-Stohlawetz, Petra, Gilbert, James C, Hutabarat, Renta, Knöbl, Paul
Published in Thrombosis and haemostasis (01.09.2010)
Published in Thrombosis and haemostasis (01.09.2010)
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Harnessing a physiologic mechanism for siRNA delivery with mimetic lipoprotein particles
Nakayama, Tomoko, Butler, James S, Sehgal, Alfica, Severgnini, Mariano, Racie, Tim, Sharman, Jennifer, Ding, Feng, Morskaya, Svetlana Shulga, Brodsky, Joshua, Tchangov, Lubomir, Kosovrasti, Verbena, Meys, Mike, Nechev, Lubomir, Wang, Gang, Peng, Chang Geng, Fang, Yupang, Maier, Martin, Rajeev, Kallanthottathil G, Li, Robert, Hettinger, Julia, Barros, Scott, Clausen, Valerie, Zhang, Xuemei, Wang, Qianfan, Hutabarat, Renta, Dokholyan, Nikolay V, Wolfrum, Christian, Manoharan, Muthiah, Kotelianski, Victor, Stoffel, Markus, Sah, Dinah Wy
Published in Molecular therapy (01.08.2012)
Published in Molecular therapy (01.08.2012)
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Disposition of drugs in cystic fibrosis. I. Sulfamethoxazole and trimethoprim
Hutabarat, R M, Unadkat, J D, Sahajwalla, C, McNamara, S, Ramsey, B, Smith, A L
Published in Clinical pharmacology and therapeutics (01.04.1991)
Published in Clinical pharmacology and therapeutics (01.04.1991)
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A Subcutaneously Administered RNAi Therapeutic (ALN-AT3) Targeting Antithrombin for Treatment of Hemophilia: Interim Phase 1 Study Results in Healthy Volunteers and Patients with Hemophilia a or B
Sorensen, Benny, Mant, Tim, Akinc, Akin, Simon, Amy, Melton, Lauren, Lynam, Christian, Strahs, Andrew, Sehgal, Alfica, Hutabarat, Renta, Chaturvedi, Prasoon, Barros, Scott, Vaishnaw, Akshay, Investigators, Aln-AT
Published in Blood (06.12.2014)
Published in Blood (06.12.2014)
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Abstract 11936: Development of Monthly to Quarterly Subcutaneous Administration of RNAi Therapeutics Targeting the Metabolic Diseases Genes PCSK9, ApoC3 and ANGPTL3
Borodovsky, Anna, Querbes, William, Sutherland, Jessica, Hutabarat, Renta, Milstein, Stuart, Kuchimanchi, Satya, Kuchimanchi, Rajeev, Charisse, Klaus, Yucius, Kristina, Liebow, Abigail, Sprague, Andrew, Maier, Martin, Kallend, David, Simon, Amy, Horton, Jay, Fitzgerald, Kevin
Published in Circulation (New York, N.Y.) (25.11.2014)
Published in Circulation (New York, N.Y.) (25.11.2014)
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Abstract 7: A Subcutaneous, Potent and Durable RNAi Platform Targeting Metabolic Diseases, Genes PCSK9, ApoC3 and ANGPLT3
Fitzgerald, Kevin, Borodovsky, Anna, Querbes, William, Sutherland, Jessica, Hutabarat, Renta, Milstein, Stuart, Kuchimanchi, Satya, Kallanthottathil, Rajeev, Charisse, Klaus, Yucias, Kristina, Liebow, Abigail, Sprague, Andrew, Maier, Martin, Kallend, David, Horton, Jay, Simon, Amy
Published in Arteriosclerosis, thrombosis, and vascular biology (01.05.2014)
Published in Arteriosclerosis, thrombosis, and vascular biology (01.05.2014)
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