Pharmacokinetic and Pharmacodynamic Modeling of siRNA Therapeutics – a Minireview

An evaluation of the pharmacokinetics of inclisiran in the treatment of atherosclerotic cardiovascular disease

(2022) Brian Tomlinson et al.   Expert Opinion on Drug Metabolism & Toxicology

The growth of siRNA-based therapeutics: updated clinical studies

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

Physiologically based pharmacokinetic (PBPK) modeling of RNAi therapeutics: Opportunities and challenges

(2021) Kiara Fairman et al.   BIOCHEMICAL PHARMACOLOGY

Impact of Serum Proteins on the Uptake and RNA Interference Activity of N-Acetylgalactosamine-Conjugated Small Interfering RNAs

(2021) Saket Agarwal et al.   Nucleic Acid Therapeutics

Simulation-Based Pharmacokinetics Sampling Design for Evaluating Correlates of Prevention Efficacy of Passive HIV Monoclonal Antibody Prophylaxis

(2021) Lily Zhang et al.   Statistics in Biopharmaceutical Research

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

Transitioning from Basic toward Systems Pharmacodynamic Models: Lessons from Corticosteroids

(2020) Vivaswath S. Ayyar et al.   PHARMACOLOGICAL REVIEWS

Emerging siRNA Design Principles and Consequences for Biotransformation and Disposition in Drug Development

(2020) Sara C. Humphreys et al.   JOURNAL OF MEDICINAL CHEMISTRY

Two Phase 3 Trials of Inclisiran in Patients with Elevated LDL Cholesterol

(2020) Kausik K. Ray et al.   NEW ENGLAND JOURNAL OF MEDICINE

Population Pharmacokinetic–Pharmacodynamic Model of Serum Transthyretin Following Patisiran Administration

(2020) Varun Goel et al.   Nucleic Acid Therapeutics

Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

(2020) Manisha Balwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Introducing an In Vitro Liver Stability Assay Capable of Predicting the In Vivo Pharmacodynamic Efficacy of siRNAs for IVIVC

(2020) Babak Basiri et al.   Molecular Therapy-Nucleic Acids

The Evidence REVEAL Study: Exploring the Use of Real‐World Evidence and Complex Clinical Trial Design by the European Pharmaceutical Industry

(2020) Peter Varnai et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Modeling the kinetics of lipid nanoparticle mediated delivery of multiple siRNAs to evaluate the effect on competition for Ago2

(2019) Radu Mihaila et al.   Molecular Therapy-Nucleic Acids

The current state and future directions of RNAi-based therapeutics

(2019) Ryan L. Setten et al.   NATURE REVIEWS DRUG DISCOVERY

Pharmacokinetics of Patisiran, the First Approved RNA Interference Therapy in Patients With Hereditary Transthyretin‐Mediated Amyloidosis

(2019) Xiaoping Zhang et al.   JOURNAL OF CLINICAL PHARMACOLOGY

Development of a quantitative relationship between CAR-affinity, antigen abundance, tumor cell depletion and CAR-T cell expansion using a multiscale systems PK-PD model

(2019) Aman P. Singh et al.   mAbs

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

(2018) David Adams et al.   NEW ENGLAND JOURNAL OF MEDICINE

GalNAc-siRNA Conjugates: Leading the Way for Delivery of RNAi Therapeutics

(2018) Aaron D. Springer et al.   Nucleic Acid Therapeutics

Dose-Response-Time Data Analysis: An Underexploited Trinity

(2018) Johan Gabrielsson et al.   PHARMACOLOGICAL REVIEWS

Inferring Half-Lives at the Effect Site of Oligonucleotide Drugs

(2018) Rasmus Jansson-Löfmark et al.   Nucleic Acid Therapeutics

Lipid Nanoparticle Systems for Enabling Gene Therapies

(2017) Pieter R. Cullis et al.   MOLECULAR THERAPY

The chemical evolution of oligonucleotide therapies of clinical utility

(2017) Anastasia Khvorova et al.   NATURE BIOTECHNOLOGY

Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc–siRNA conjugates

(2017) Jayaprakash K. Nair et al.   NUCLEIC ACIDS RESEARCH

Mathematical Modeling: A Tool for Optimization of Lipid Nanoparticle-Mediated Delivery of siRNA

(2017) Radu Mihaila et al.   Molecular Therapy-Nucleic Acids

Preclinical and clinical development of siRNA-based therapeutics

(2015) Gulnihal Ozcan et al.   ADVANCED DRUG DELIVERY REVIEWS

Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing

(2014) Jayaprakash K. Nair et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Action and Reaction: The Biological Response to siRNA and Its Delivery Vehicles

(2012) Rosemary L Kanasty et al.   MOLECULAR THERAPY

Towards a platform PBPK model to characterize the plasma and tissue disposition of monoclonal antibodies in preclinical species and human

(2011) Dhaval K. Shah et al.   JOURNAL OF PHARMACOKINETICS AND PHARMACODYNAMICS

SPANosomes as Delivery Vehicles for Small Interfering RNA (siRNA)

(2011) Chenguang Zhou et al.   MOLECULAR PHARMACEUTICS

RNA-Induced Silencing Complex-Bound Small Interfering RNA Is a Determinant of RNA Interference-Mediated Gene Silencing in Mice

(2011) J. Wei et al.   MOLECULAR PHARMACOLOGY

Clinical Trial Simulation: A Review

(2010) N Holford et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS

Development of Translational Pharmacokinetic–Pharmacodynamic Models

(2008) DE Mager et al.   CLINICAL PHARMACOLOGY & THERAPEUTICS