Anne Smith
Isis Pharmaceuticals
13 Papers
25 Citations
Anne Smith is an academic researcher from Isis Pharmaceuticals. The author has contributed to research in topics: Huntington's disease & Huntingtin. The author has an hindex of 7, co-authored 11 publications. Previous affiliations of Anne Smith include University College London.
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Papers
Targeting Huntingtin Expression in Patients with Huntington’s Disease
Sarah J. Tabrizi,Blair R. Leavitt,G. Bernhard Landwehrmeyer,Edward J. Wild,Carsten Saft,Roger A. Barker,Nick F. Blair,D Craufurd,Josef Priller,Hugh Rickards,Anne Elizabeth Rosser,Holly B. Kordasiewicz,Christian Czech,Eric E. Swayze,Daniel A. Norris,Tiffany L. Baumann,Irene Gerlach,Scott Schobel,Erika Paz,Anne Smith,C. Frank Bennett,Roger M. Lane +21 more
TL;DR: Intrathecal administration of HTTRx to patients with early Huntington's disease was not accompanied by serious adverse events and dose-dependent reductions in concentrations of mutant huntingtin were observed.
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Antisense Inhibition of Glucagon Receptor by IONIS-GCGRRx Improves Type 2 Diabetes Without Increase in Hepatic Glycogen Content in Patients With Type 2 Diabetes on Stable Metformin Therapy.
Erin S. Morgan,Li-Jung Tai,Nguyen C. Pham,Julia K. Overman,Lynnetta Watts,Anne Smith,Shiangtung W. Jung,Martin Gajdošík,Martin Krššák,Michael Krebs,Richard S. Geary,Brenda F. Baker,Sanjay Bhanot +12 more
TL;DR: IONis-GCGRRx is a potent inhibitor of hepatic glucagon receptor expression with a potential to improve glycemic control at low weekly doses in combination with metformin, and novel results suggest that despite inhibition of glycogenolysis after GCGR antagonism, IONIS-GC GRRx did not increase hepatic glycogen content.
Translating Antisense Technology into a Treatment for Huntington's Disease.
Roger Lane,Anne Smith,Tiffany L. Baumann,Marc Gleichmann,Daniel A. Norris,C. Frank Bennett,Holly B. Kordasiewicz +6 more
TL;DR: A deep and wide-ranging understanding of the basic, preclinical, clinical, and epidemiologic components of drug development will improve the likelihood of success.
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•Journal Article
Design of the First-in-Human Study of IONIS-MAPTRx, a Tau-lowering Antisense Oligonucleotide, in Patients With Alzheimer Disease (S2.006)
Laurence Mignon,Holly Kordasiewicz,Roger Lane,Anne Smith,Timothy M. Miller,Padma Kumar Narayanan,Eric E. Swayze,Daniel A. Norris,Bethany Fitzsimmons,Frank C. Bennett +9 more
TL;DR: IONIS-MAPT Rx is the result of a comprehensive drug discovery effort to design a well-tolerated, potent ASO with high specificity to human MAPT mRNA that recently initiated dosing in patients with mild AD.
28
•Journal Article
Effects of IONIS-HTTRx in Patients with Early Huntington’s Disease, Results of the First HTT-Lowering Drug Trial (CT.002)
Sarah J. Tabrizi,Blair R. Leavitt,Holly Kordasiewicz,Christian Czech,Eric E. Swayze,Daniel A. Norris,Tiffany L. Baumann,Irene Gerlach,Scott Schobel,Anne Smith,Roger Lane,C. Frank Bennett +11 more
TL;DR: Significant dose-dependent reductions in CSF mutant HTT (mHTT) were observed, suggesting that IONIS-HTT Rx is a promising therapeutic for the treatment of HD, and ASO technology has the potential to provide disease-modifying benefits to patients with neurodegenerative diseases.
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