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Page 1
The Nonclinical Disposition and Pharmacokinetic/Pharmacodynamic Properties of N-Acetylgalactosamine-Conjugated Small Interfering RNA Are Highly Predictable and Build Confidence in Translation to Human.
McDougall R, Ramsden D, Agarwal S, Agarwal S, Aluri K, Arciprete M, Brown C, Castellanos-Rizaldos E, Charisse K, Chong S, Cichocki J, Fitzgerald K, Goel V, Gu Y, Guenther D, Habtemariam B, Jadhav V, Janas M, Jayaraman M, Kurz J, Li J, Liu J, Liu X, Liou S, Maclauchlin C, Maier M, Manoharan M, Nair JK, Robbie G, Schmidt K, Smith P, Theile C, Vaishnaw A, Waldron S, Xu Y, Zhang X, Zlatev I, Wu JT. McDougall R, et al. Among authors: vaishnaw a. Drug Metab Dispos. 2022 Jun;50(6):781-797. doi: 10.1124/dmd.121.000428. Epub 2021 Jun 21. Drug Metab Dispos. 2022. PMID: 34154993 Review.
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.
Liver as a target for oligonucleotide therapeutics.
Sehgal A, Vaishnaw A, Fitzgerald K. Sehgal A, et al. Among authors: vaishnaw a. J Hepatol. 2013 Dec;59(6):1354-9. doi: 10.1016/j.jhep.2013.05.045. Epub 2013 Jun 12. J Hepatol. 2013. PMID: 23770039 Free article.
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.
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.
Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria.
Sardh E, Harper P, Balwani M, Stein P, Rees D, Bissell DM, Desnick R, Parker C, Phillips J, Bonkovsky HL, Vassiliou D, Penz C, Chan-Daniels A, He Q, Querbes W, Fitzgerald K, Kim JB, Garg P, Vaishnaw A, Simon AR, Anderson KE. Sardh E, et al. Among authors: vaishnaw a. N Engl J Med. 2019 Feb 7;380(6):549-558. doi: 10.1056/NEJMoa1807838. N Engl J Med. 2019. PMID: 30726693 Clinical Trial.
Neurofilament Light Chain as a Biomarker of Hereditary Transthyretin-Mediated Amyloidosis.
Ticau S, Sridharan GV, Tsour S, Cantley WL, Chan A, Gilbert JA, Erbe D, Aldinc E, Reilly MM, Adams D, Polydefkis M, Fitzgerald K, Vaishnaw A, Nioi P. Ticau S, et al. Among authors: vaishnaw a. Neurology. 2021 Jan 19;96(3):e412-e422. doi: 10.1212/WNL.0000000000011090. Epub 2020 Oct 21. Neurology. 2021. PMID: 33087494 Free PMC article.
Phase 1/2 Study of Lumasiran for Treatment of Primary Hyperoxaluria Type 1: A Placebo-Controlled Randomized Clinical Trial.
Frishberg Y, Deschênes G, Groothoff JW, Hulton SA, Magen D, Harambat J, Van't Hoff WG, Lorch U, Milliner DS, Lieske JC, Haslett P, Garg PP, Vaishnaw AK, Talamudupula S, Lu J, Habtemariam BA, Erbe DV, McGregor TL, Cochat P; study collaborators. Frishberg Y, et al. Among authors: vaishnaw ak. Clin J Am Soc Nephrol. 2021 Jul;16(7):1025-1036. doi: 10.2215/CJN.14730920. Epub 2021 May 13. Clin J Am Soc Nephrol. 2021. PMID: 33985991 Free PMC article. Clinical Trial.
50 results