Preclinical evaluation of stereopure antisense oligonucleotides for allele-selective lowering of mutant HTT .

Autor: Iwamoto N; Wave Life Sciences, Cambridge, MA 02138, USA., Liu Y; Wave Life Sciences, Cambridge, MA 02138, USA., Frank-Kamenetsky M; Wave Life Sciences, Cambridge, MA 02138, USA., Maguire A; Wave Life Sciences, Cambridge, MA 02138, USA., Tseng WC; Wave Life Sciences, Cambridge, MA 02138, USA., Taborn K; Wave Life Sciences, Cambridge, MA 02138, USA., Kothari N; Wave Life Sciences, Cambridge, MA 02138, USA., Akhtar A; Wave Life Sciences, Cambridge, MA 02138, USA., Bowman K; Wave Life Sciences, Cambridge, MA 02138, USA., Shelke JD; Wave Life Sciences, Cambridge, MA 02138, USA., Lamattina A; Wave Life Sciences, Cambridge, MA 02138, USA., Hu XS; Wave Life Sciences, Cambridge, MA 02138, USA., Jang HG; Wave Life Sciences, Cambridge, MA 02138, USA., Kandasamy P; Wave Life Sciences, Cambridge, MA 02138, USA., Liu F; Wave Life Sciences, Cambridge, MA 02138, USA., Longo K; Wave Life Sciences, Cambridge, MA 02138, USA., Looby R; Wave Life Sciences, Cambridge, MA 02138, USA., Meena; Wave Life Sciences, Cambridge, MA 02138, USA., Metterville J; Wave Life Sciences, Cambridge, MA 02138, USA., Pan Q; Wave Life Sciences, Cambridge, MA 02138, USA., Purcell-Estabrook E; Wave Life Sciences, Cambridge, MA 02138, USA., Shimizu M; Wave Life Sciences, Cambridge, MA 02138, USA., Prakasha PS; Wave Life Sciences, Cambridge, MA 02138, USA., Standley S; Wave Life Sciences, Cambridge, MA 02138, USA., Upadhyay H; Wave Life Sciences, Cambridge, MA 02138, USA., Yang H; Wave Life Sciences, Cambridge, MA 02138, USA., Yin Y; Wave Life Sciences, Cambridge, MA 02138, USA., Zhao A; Wave Life Sciences, Cambridge, MA 02138, USA., Francis C; Wave Life Sciences, Cambridge, MA 02138, USA., Byrne M; Wave Life Sciences, Cambridge, MA 02138, USA., Dale E; Wave Life Sciences, Cambridge, MA 02138, USA., Verdine GL; Department of Stem Cell and Regenerative Biology, Department of Chemistry and Chemical Biology, Harvard University, Cambridge, MA 02138, USA., Vargeese C; Wave Life Sciences, Cambridge, MA 02138, USA.

Publikováno v: Molecular therapy. Nucleic acids [Mol Ther Nucleic Acids] 2024 Jun 11; Vol. 35 (3), pp. 102246. Date of Electronic Publication: 2024 Jun 11 (Print Publication: 2024).

Modulation of Ligand-Gated Glycine Receptors Via Functional Monoclonal Antibodies.

Autor: Simard JR; Departments of Neuroscience (J.R.S., C.Y., B.Y. B.G., K.T., D.J.G., J.G.), Molecular Engineering (K.M., A.M.L., P.L.S.), Protein Technologies (Y.W., B.E.H.), and Pharmacokinetics and Drug Metabolism (R.S.F.), Amgen Research, Cambridge, Massachusetts; and Department of Pharmacokinetics and Drug Metabolism, Amgen Research, 1120 Veterans Boulevard, South San Francisco, California (K.Co., K.Ch.) jgingras@homologymedicines.com jsimard@c4therapeutics.com., Michelsen K; Departments of Neuroscience (J.R.S., C.Y., B.Y. B.G., K.T., D.J.G., J.G.), Molecular Engineering (K.M., A.M.L., P.L.S.), Protein Technologies (Y.W., B.E.H.), and Pharmacokinetics and Drug Metabolism (R.S.F.), Amgen Research, Cambridge, Massachusetts; and Department of Pharmacokinetics and Drug Metabolism, Amgen Research, 1120 Veterans Boulevard, South San Francisco, California (K.Co., K.Ch.)., Wang Y; Departments of Neuroscience (J.R.S., C.Y., B.Y. B.G., K.T., D.J.G., J.G.), Molecular Engineering (K.M., A.M.L., P.L.S.), Protein Technologies (Y.W., B.E.H.), and Pharmacokinetics and Drug Metabolism (R.S.F.), Amgen Research, Cambridge, Massachusetts; and Department of Pharmacokinetics and Drug Metabolism, Amgen Research, 1120 Veterans Boulevard, South San Francisco, California (K.Co., K.Ch.)., Yang C; Departments of Neuroscience (J.R.S., C.Y., B.Y. B.G., K.T., D.J.G., J.G.), Molecular Engineering (K.M., A.M.L., P.L.S.), Protein Technologies (Y.W., B.E.H.), and Pharmacokinetics and Drug Metabolism (R.S.F.), Amgen Research, Cambridge, Massachusetts; and Department of Pharmacokinetics and Drug Metabolism, Amgen Research, 1120 Veterans Boulevard, South San Francisco, California (K.Co., K.Ch.)., Youngblood B; Departments of Neuroscience (J.R.S., C.Y., B.Y. B.G., K.T., D.J.G., J.G.), Molecular Engineering (K.M., A.M.L., P.L.S.), Protein Technologies (Y.W., B.E.H.), and Pharmacokinetics and Drug Metabolism (R.S.F.), Amgen Research, Cambridge, Massachusetts; and Department of Pharmacokinetics and Drug Metabolism, Amgen Research, 1120 Veterans Boulevard, South San Francisco, California (K.Co., K.Ch.)., Grubinska B; Departments of Neuroscience (J.R.S., C.Y., B.Y. B.G., K.T., D.J.G., J.G.), Molecular Engineering (K.M., A.M.L., P.L.S.), Protein Technologies (Y.W., B.E.H.), and Pharmacokinetics and Drug Metabolism (R.S.F.), Amgen Research, Cambridge, Massachusetts; and Department of Pharmacokinetics and Drug Metabolism, Amgen Research, 1120 Veterans Boulevard, South San Francisco, California (K.Co., K.Ch.)., Taborn K; Departments of Neuroscience (J.R.S., C.Y., B.Y. B.G., K.T., D.J.G., J.G.), Molecular Engineering (K.M., A.M.L., P.L.S.), Protein Technologies (Y.W., B.E.H.), and Pharmacokinetics and Drug Metabolism (R.S.F.), Amgen Research, Cambridge, Massachusetts; and Department of Pharmacokinetics and Drug Metabolism, Amgen Research, 1120 Veterans Boulevard, South San Francisco, California (K.Co., K.Ch.)., Gillie DJ; Departments of Neuroscience (J.R.S., C.Y., B.Y. B.G., K.T., D.J.G., J.G.), Molecular Engineering (K.M., A.M.L., P.L.S.), Protein Technologies (Y.W., B.E.H.), and Pharmacokinetics and Drug Metabolism (R.S.F.), Amgen Research, Cambridge, Massachusetts; and Department of Pharmacokinetics and Drug Metabolism, Amgen Research, 1120 Veterans Boulevard, South San Francisco, California (K.Co., K.Ch.)., Cook K; Departments of Neuroscience (J.R.S., C.Y., B.Y. B.G., K.T., D.J.G., J.G.), Molecular Engineering (K.M., A.M.L., P.L.S.), Protein Technologies (Y.W., B.E.H.), and Pharmacokinetics and Drug Metabolism (R.S.F.), Amgen Research, Cambridge, Massachusetts; and Department of Pharmacokinetics and Drug Metabolism, Amgen Research, 1120 Veterans Boulevard, South San Francisco, California (K.Co., K.Ch.)., Chung K; Departments of Neuroscience (J.R.S., C.Y., B.Y. B.G., K.T., D.J.G., J.G.), Molecular Engineering (K.M., A.M.L., P.L.S.), Protein Technologies (Y.W., B.E.H.), and Pharmacokinetics and Drug Metabolism (R.S.F.), Amgen Research, Cambridge, Massachusetts; and Department of Pharmacokinetics and Drug Metabolism, Amgen Research, 1120 Veterans Boulevard, South San Francisco, California (K.Co., K.Ch.)., Long AM; Departments of Neuroscience (J.R.S., C.Y., B.Y. B.G., K.T., D.J.G., J.G.), Molecular Engineering (K.M., A.M.L., P.L.S.), Protein Technologies (Y.W., B.E.H.), and Pharmacokinetics and Drug Metabolism (R.S.F.), Amgen Research, Cambridge, Massachusetts; and Department of Pharmacokinetics and Drug Metabolism, Amgen Research, 1120 Veterans Boulevard, South San Francisco, California (K.Co., K.Ch.)., Hall BE; Departments of Neuroscience (J.R.S., C.Y., B.Y. B.G., K.T., D.J.G., J.G.), Molecular Engineering (K.M., A.M.L., P.L.S.), Protein Technologies (Y.W., B.E.H.), and Pharmacokinetics and Drug Metabolism (R.S.F.), Amgen Research, Cambridge, Massachusetts; and Department of Pharmacokinetics and Drug Metabolism, Amgen Research, 1120 Veterans Boulevard, South San Francisco, California (K.Co., K.Ch.)., Shaffer PL; Departments of Neuroscience (J.R.S., C.Y., B.Y. B.G., K.T., D.J.G., J.G.), Molecular Engineering (K.M., A.M.L., P.L.S.), Protein Technologies (Y.W., B.E.H.), and Pharmacokinetics and Drug Metabolism (R.S.F.), Amgen Research, Cambridge, Massachusetts; and Department of Pharmacokinetics and Drug Metabolism, Amgen Research, 1120 Veterans Boulevard, South San Francisco, California (K.Co., K.Ch.)., Foti RS; Departments of Neuroscience (J.R.S., C.Y., B.Y. B.G., K.T., D.J.G., J.G.), Molecular Engineering (K.M., A.M.L., P.L.S.), Protein Technologies (Y.W., B.E.H.), and Pharmacokinetics and Drug Metabolism (R.S.F.), Amgen Research, Cambridge, Massachusetts; and Department of Pharmacokinetics and Drug Metabolism, Amgen Research, 1120 Veterans Boulevard, South San Francisco, California (K.Co., K.Ch.)., Gingras J; Departments of Neuroscience (J.R.S., C.Y., B.Y. B.G., K.T., D.J.G., J.G.), Molecular Engineering (K.M., A.M.L., P.L.S.), Protein Technologies (Y.W., B.E.H.), and Pharmacokinetics and Drug Metabolism (R.S.F.), Amgen Research, Cambridge, Massachusetts; and Department of Pharmacokinetics and Drug Metabolism, Amgen Research, 1120 Veterans Boulevard, South San Francisco, California (K.Co., K.Ch.) jgingras@homologymedicines.com jsimard@c4therapeutics.com.

Publikováno v: The Journal of pharmacology and experimental therapeutics [J Pharmacol Exp Ther] 2022 Oct; Vol. 383 (1), pp. 56-69. Date of Electronic Publication: 2022 Aug 04.

Preclinical evaluation of WVE-004, aninvestigational stereopure oligonucleotide forthe treatment of C9orf72 -associated ALS or FTD.

Autor: Liu Y; Wave Life Sciences, 733 Concord Avenue, Cambridge, MA 02138, USA., Andreucci A; Wave Life Sciences, 733 Concord Avenue, Cambridge, MA 02138, USA., Iwamoto N; Wave Life Sciences, 733 Concord Avenue, Cambridge, MA 02138, USA., Yin Y; Wave Life Sciences, 733 Concord Avenue, Cambridge, MA 02138, USA., Yang H; Wave Life Sciences, 733 Concord Avenue, Cambridge, MA 02138, USA., Liu F; Wave Life Sciences, 733 Concord Avenue, Cambridge, MA 02138, USA., Bulychev A; Wave Life Sciences, 733 Concord Avenue, Cambridge, MA 02138, USA., Hu XS; Wave Life Sciences, 733 Concord Avenue, Cambridge, MA 02138, USA., Lin X; Wave Life Sciences, 733 Concord Avenue, Cambridge, MA 02138, USA., Lamore S; Wave Life Sciences, 733 Concord Avenue, Cambridge, MA 02138, USA., Patil S; Wave Life Sciences, 733 Concord Avenue, Cambridge, MA 02138, USA., Mohapatra S; Wave Life Sciences, 733 Concord Avenue, Cambridge, MA 02138, USA., Purcell-Estabrook E; Wave Life Sciences, 733 Concord Avenue, Cambridge, MA 02138, USA., Taborn K; Wave Life Sciences, 733 Concord Avenue, Cambridge, MA 02138, USA., Dale E; Wave Life Sciences, 733 Concord Avenue, Cambridge, MA 02138, USA., Vargeese C; Wave Life Sciences, 733 Concord Avenue, Cambridge, MA 02138, USA.

Publikováno v: Molecular therapy. Nucleic acids [Mol Ther Nucleic Acids] 2022 Apr 20; Vol. 28, pp. 558-570. Date of Electronic Publication: 2022 Apr 20 (Print Publication: 2022).

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The discovery of benzoxazine sulfonamide inhibitors of Na V 1.7: Tools that bridge efficacy and target engagement.

Autor: La DS; Department of Medicinal Chemistry, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States. Electronic address: daniel.la@sagerx.com., Peterson EA; Department of Medicinal Chemistry, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., Bode C; Department of Medicinal Chemistry, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., Boezio AA; Department of Medicinal Chemistry, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., Bregman H; Department of Medicinal Chemistry, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., Chu-Moyer MY; Department of Medicinal Chemistry, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., Coats J; Department of Medicinal Chemistry, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., DiMauro EF; Department of Medicinal Chemistry, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., Dineen TA; Department of Medicinal Chemistry, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., Du B; Department of Medicinal Chemistry, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., Gao H; Department of Molecular Engineering, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., Graceffa R; Department of Medicinal Chemistry, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., Gunaydin H; Department of Molecular Engineering, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., Guzman-Perez A; Department of Medicinal Chemistry, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., Fremeau R Jr; Department of Neuroscience, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., Huang X; Department of Molecular Engineering, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., Ilch C; Department of Neuroscience, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., Kornecook TJ; Department of Neuroscience, Amgen Inc., One Amgen Center Drive, Thousand Oaks, CA 91320, United States., Kreiman C; Department of Medicinal Chemistry, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., Ligutti J; Amgen, Thousand Oaks, CA, United States. Electronic address: jligutti@amgen.com., Jasmine Lin MH; Department of Pharmacokinetics and Drug Metabolism, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., McDermott JS; Department of Neuroscience, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., Marx I; Department of Medicinal Chemistry, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., Matson DJ; Department of Neuroscience, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., McDonough SI; Department of Neuroscience, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., Moyer BD; Department of Neuroscience, Amgen Inc., One Amgen Center Drive, Thousand Oaks, CA 91320, United States., Nho Nguyen H; Department of Medicinal Chemistry, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., Taborn K; Department of Neuroscience, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., Yu V; Department of Neuroscience, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States., Weiss MM; Department of Medicinal Chemistry, Amgen Inc., 360 Binney Street, Cambridge, MA 02142, United States.

Publikováno v: Bioorganic & medicinal chemistry letters [Bioorg Med Chem Lett] 2017 Aug 01; Vol. 27 (15), pp. 3477-3485. Date of Electronic Publication: 2017 Jun 01.