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Page 1
Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate.
Zimmermann TS, Karsten V, Chan A, Chiesa J, Boyce M, Bettencourt BR, Hutabarat R, Nochur S, Vaishnaw A, Gollob J. Zimmermann TS, et al. Among authors: vaishnaw a. Mol Ther. 2017 Jan 4;25(1):71-78. doi: 10.1016/j.ymthe.2016.10.019. Epub 2017 Jan 4. Mol Ther. 2017. PMID: 28129130 Free PMC article. Clinical Trial.
A Highly Durable RNAi Therapeutic Inhibitor of PCSK9.
Fitzgerald K, White S, Borodovsky A, Bettencourt BR, Strahs A, Clausen V, Wijngaard P, Horton JD, Taubel J, Brooks A, Fernando C, Kauffman RS, Kallend D, Vaishnaw A, Simon A. Fitzgerald K, et al. Among authors: vaishnaw a. N Engl J Med. 2017 Jan 5;376(1):41-51. doi: 10.1056/NEJMoa1609243. Epub 2016 Nov 13. N Engl J Med. 2017. PMID: 27959715 Free PMC article. Clinical Trial.
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 K, Frank-Kamenetsky M, Shulga-Morskaya S, Liebow A, Bettencourt BR, Sutherland JE, Hutabarat RM, Clausen VA, Karsten V, Cehelsky J, Nochur SV, Kotelianski V, Horton J, Mant T, Chiesa J, Ritter J, Munisamy M, Vaishnaw AK, Gollob JA, Simon A. Fitzgerald K, et al. Among authors: vaishnaw ak. Lancet. 2014 Jan 4;383(9911):60-68. doi: 10.1016/S0140-6736(13)61914-5. Epub 2013 Oct 3. Lancet. 2014. PMID: 24094767 Free PMC article. Clinical Trial.
Safety evaluation of 2'-deoxy-2'-fluoro nucleotides in GalNAc-siRNA conjugates.
Janas MM, Zlatev I, Liu J, Jiang Y, Barros SA, Sutherland JE, Davis WP, Liu J, Brown CR, Liu X, Schlegel MK, Blair L, Zhang X, Das B, Tran C, Aluri K, Li J, Agarwal S, Indrakanti R, Charisse K, Nair J, Matsuda S, Rajeev KG, Zimmermann T, Sepp-Lorenzino L, Xu Y, Akinc A, Fitzgerald K, Vaishnaw AK, Smith PF, Manoharan M, Jadhav V, Wu JT, Maier MA. Janas MM, et al. Among authors: vaishnaw ak. Nucleic Acids Res. 2019 Apr 23;47(7):3306-3320. doi: 10.1093/nar/gkz140. Nucleic Acids Res. 2019. PMID: 30820542 Free PMC article.
Single-Dose Pharmacokinetics and Pharmacodynamics of Transthyretin Targeting N-acetylgalactosamine-Small Interfering Ribonucleic Acid Conjugate, Vutrisiran, in Healthy Subjects.
Habtemariam BA, Karsten V, Attarwala H, Goel V, Melch M, Clausen VA, Garg P, Vaishnaw AK, Sweetser MT, Robbie GJ, Vest J. Habtemariam BA, et al. Among authors: vaishnaw ak. Clin Pharmacol Ther. 2021 Feb;109(2):372-382. doi: 10.1002/cpt.1974. Epub 2020 Aug 13. Clin Pharmacol Ther. 2021. PMID: 32599652 Clinical Trial.
ALN-RSV01 for prevention of bronchiolitis obliterans syndrome after respiratory syncytial virus infection in lung transplant recipients.
Gottlieb J, Zamora MR, Hodges T, Musk AW, Sommerwerk U, Dilling D, Arcasoy S, DeVincenzo J, Karsten V, Shah S, Bettencourt BR, Cehelsky J, Nochur S, Gollob J, Vaishnaw A, Simon AR, Glanville AR. Gottlieb J, et al. Among authors: vaishnaw a. J Heart Lung Transplant. 2016 Feb;35(2):213-21. doi: 10.1016/j.healun.2015.08.012. Epub 2015 Sep 3. J Heart Lung Transplant. 2016. PMID: 26452996 Clinical Trial.
Safety and efficacy of RNAi therapy for transthyretin amyloidosis.
Coelho T, Adams D, Silva A, Lozeron P, Hawkins PN, Mant T, Perez J, Chiesa J, Warrington S, Tranter E, Munisamy M, Falzone R, Harrop J, Cehelsky J, Bettencourt BR, Geissler M, Butler JS, Sehgal A, Meyers RE, Chen Q, Borland T, Hutabarat RM, Clausen VA, Alvarez R, Fitzgerald K, Gamba-Vitalo C, Nochur SV, Vaishnaw AK, Sah DW, Gollob JA, Suhr OB. Coelho T, et al. Among authors: vaishnaw ak. N Engl J Med. 2013 Aug 29;369(9):819-29. doi: 10.1056/NEJMoa1208760. N Engl J Med. 2013. PMID: 23984729 Free article. Clinical Trial.
A status report on RNAi therapeutics.
Vaishnaw AK, Gollob J, Gamba-Vitalo C, Hutabarat R, Sah D, Meyers R, de Fougerolles T, Maraganore J. Vaishnaw AK, et al. Silence. 2010 Jul 8;1(1):14. doi: 10.1186/1758-907X-1-14. Silence. 2010. PMID: 20615220 Free PMC article.
Phase 3 Multicenter Study of Revusiran in Patients with Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Cardiomyopathy (ENDEAVOUR).
Judge DP, Kristen AV, Grogan M, Maurer MS, Falk RH, Hanna M, Gillmore J, Garg P, Vaishnaw AK, Harrop J, Powell C, Karsten V, Zhang X, Sweetser MT, Vest J, Hawkins PN. Judge DP, et al. Cardiovasc Drugs Ther. 2020 Jun;34(3):357-370. doi: 10.1007/s10557-019-06919-4. Cardiovasc Drugs Ther. 2020. PMID: 32062791 Free PMC article. Clinical Trial.
50 results