time-course effects, and efficacy-related biomarkers. Additionally, within the realm of quantitative pharmacology, PK/PD models, dose-effect-time models, and PK/PB models play pivotal roles in the development of siRNA drugs, enabling the simulation, extrapolation, and prediction of data...
The landscape of biomedical uses of mRNA continues to rapidly evolve. Below, we divide our discussion into three areas: direct in vivo administration of mRNA for preventative vaccines against infectious disease (Table1), therapeutic mRNA vaccines against cancer (Table2) and mRNA-encoded immune therap...
More than two decades after the natural gene-silencing mechanism of RNA interference was elucidated, small interfering RNA (siRNA)-based therapeutics have finally broken into the pharmaceutical market. With three agents already approved and many others in advanced stages of the drug development pipeline...
This work provides a quantitative mechanistic framework and insights on modality‐specific factors that drive clinical translation and patient/disease‐related factors that impact specific dosing considerations and clinical outcomes to help accelerate the optimal development of siRNA therapeutics.Lumen, An...
90 The use of GN3 as an siRNA conjugate targeting ALAS1 (givosiran) has been approved by the FDA to treat acute hepatic porphyria.88 Several GN3-conjugated gapmers are currently being evaluated in clinical trials to target AGT, ANGPTL3, CFB, GHR, KLKB1, LPA, TMPRSS6, and TTR ...
RNA interference technology, using synthetic structurally well-defined short double-stranded RNA (small interfering RNA [siRNA]), has advanced rapidly in recent years. This introductory review describes the utility of siRNA, by exploring the underpinning biology, pharmacology, recent advances and clinical...
Small interfering RNA (siRNA) is one of several nucleic acid molecules representing a third class of therapeutic modalities, in addition to small molecules and biologics. Like small molecules, siRNAs have a defined chemical structure, are inexpensive to manufacture, and are stable at room ...
Clinical Development Based on product lifecycle management, we design the clinical trials of our drug candidates, select medical strategy, conduct our clinical trials to ensure speed of execution and data quality, manage risk, maintain constructive dialogues with the regulatory authorities to achieve opti...
We combine clinical pharmacology with model-informed drug development to help you make decisions and achieve program success. The involvement of clinical pharmacology with the support of MIDD approaches will help you to achieve this objective.
Clinical pharmacology of alendronate sodium. Osteopor Int. 1993; 3(suppl.3):513–16. Roschger P, Lombardi A, Misof BM, et al. Mineralization density distribution of postmenopausal osteoporotic bone is restored to normal after long-term alendronate treatment: qBEI and sSAXS data from the ...