Plasma Pharmacokinetics of N-Acetylgalactosamine-Conjugated Small-Interfering Ribonucleic Acids (GalNAc-Conjugated siRNAs)

Preclinical development and phase 1 trial of a novel siRNA targeting lipoprotein(a)

(2022) Michael J. Koren et al.   NATURE MEDICINE

Pre-clinical assessment of SLN360, a novel siRNA targeting LPA, developed to address elevated lipoprotein (a) in cardiovascular disease

(2022) David A. Rider et al.   ATHEROSCLEROSIS

Single Ascending Dose Study of a Short Interfering RNA Targeting Lipoprotein(a) Production in Individuals With Elevated Plasma Lipoprotein(a) Levels

(2022) Steven E. Nissen et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Delivery of RNA Therapeutics: The Great Endosomal Escape!

(2022) Steven F. Dowdy et al.   Nucleic Acid Therapeutics

Considerations and recommendations for assessment of plasma protein binding and drug–drug interactions for siRNA therapeutics

(2022) Sara C Humphreys et al.   NUCLEIC ACIDS RESEARCH

Pharmacokinetic and Pharmacodynamic Modeling of siRNA Therapeutics – a Minireview

(2022) Jae Yoon Jeon et al.   PHARMACEUTICAL RESEARCH

Cross-Species Translation of Biophase Half-Life and Potency of GalNAc-Conjugated siRNAs

(2022) Alessandro Boianelli et al.   Nucleic Acid Therapeutics

Therapeutic siRNA: State-of-the-Art and Future Perspectives

(2022) Maik Friedrich et al.   BIODRUGS

Small interfering RNA (siRNA): discovery, pharmacology and clinical development – an introductory review

(2022) Priyanga Ranasinghe et al.   BRITISH JOURNAL OF PHARMACOLOGY

Clinical Pharmacology of RNA Interference–Based Therapeutics: A Summary Based on Food and Drug Administration–Approved Small Interfering RNAs

(2022) Xing Jing et al.   DRUG METABOLISM AND DISPOSITION

The growth of siRNA-based therapeutics: updated clinical studies

(2021) M. May Zhang et al.   BIOCHEMICAL PHARMACOLOGY

Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran – results of the phase 1 inhibitor cohort

(2021) K. John Pasi et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Phase 1/2 Study of Lumasiran for Treatment of Primary Hyperoxaluria Type 1

(2021) Yaacov Frishberg et al.   Clinical Journal of the American Society of Nephrology

The Nonclinical Disposition and PK/PD Properties of GalNAc-conjugated siRNA Are Highly Predictable and Build Confidence in Translation to Man

(2021) Robin McDougall et al.   DRUG METABOLISM AND DISPOSITION

Minimal physiologically based pharmacokinetic-pharmacodynamic (mPBPK-PD) model of GalNAc-conjugated siRNA disposition and gene silencing in preclinical species and humans

(2021) Vivaswath S Ayyar et al.   JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS

Safety, pharmacodynamics, and exposure-response modeling results from a first in human phase 1 study of nedosiran in primary hyperoxaluria.

(2021) Bernd Hoppe et al.   KIDNEY INTERNATIONAL

Pharmacokinetics and Pharmacodynamics of the Small Interfering Ribonucleic Acid (siRNA), Givosiran, in Patients with Acute Hepatic Porphyria

(2020) Sagar Agarwal et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Humans are animals, but are animals human enough? A systematic review and meta-analysis on interspecies differences in renal drug clearance

(2020) Katja Jansen et al.   DRUG DISCOVERY TODAY

Single‐Dose Pharmacokinetics and Pharmacodynamics of Transthyretin Targeting N‐acetylgalactosamine–Small Interfering Ribonucleic Acid Conjugate, Vutrisiran, in Healthy Subjects

(2020) Bahru A. Habtemariam et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Pharmacokinetic and Pharmacodynamic Properties of Cemdisiran, an RNAi Therapeutic Targeting Complement Component 5, in Healthy Subjects and Patients with Paroxysmal Nocturnal Hemoglobinuria

(2020) Prajakta Badri et al.   CLINICAL PHARMACOKINETICS

Investigating the pharmacodynamic durability of GalNAc–siRNA conjugates

(2020) Christopher R Brown et al.   NUCLEIC ACIDS RESEARCH

Effects of Renal Impairment on the Pharmacokinetics, Efficacy, and Safety of Inclisiran: An Analysis of the ORION-7 and ORION-1 Studies

(2019) R. Scott Wright et al.   MAYO CLINIC PROCEEDINGS

Nonclinical Safety Profile of Revusiran, a 1st-Generation GalNAc-siRNA Conjugate for Treatment of Hereditary Transthyretin-Mediated Amyloidosis

(2019) Jessica E. Sutherland et al.   Nucleic Acid Therapeutics

Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate

(2017) Tracy S. Zimmermann et al.   MOLECULAR THERAPY

Prediction of Human Pharmacokinetics of Therapeutic Monoclonal Antibodies from Simple Allometry of Monkey Data

(2011) Masataka Oitate et al.   Drug Metabolism and Pharmacokinetics

Interspecies scaling and prediction of human clearance: comparison of small- and macro-molecule drugs

(2011) Yeamin Huh et al.   XENOBIOTICA