Brochure
Oligonucleotide Therapies-Focused Innovative Pharmacology Platform
This brochure introduces a specialized pharmacology platform for the preclinical evaluation of oligonucleotide therapeutics. It details in vivo humanized models and in vitro iPSC assays covering metabolic, neurological, and ophthalmic diseases. This resource provides essential validation strategies for researchers advancing RNA-based therapies toward IND filing.
Overview
This document provides an overview of preclinical evaluation services for oligonucleotide therapeutics, including pharmacology, pharmacokinetics, and toxicology. It catalogs diverse humanized mouse models and iPSC models across metabolic, neurological, and ophthalmic disease categories. Furthermore, it presents empirical pharmacodynamic validation data—such as target gene knockdown in B6-hPCSK9 and B6-hINHBE models—demonstrating the platform's utility in screening innovative RNA-based drugs.
Key Insights
This resource highlights a comprehensive suite of preclinical evaluation tools and empirical data to support oligonucleotide drug development:
- Extensive Portfolio: Catalogs in vivo humanized and in vitro iPSC models for diseases like MASH, Alzheimer's, ALS, and retinitis pigmentosa.
- End-to-End Evaluation: Details integrated services from in vitro knockdown screening to PK and toxicology studies.
- Clinical Translation: Features validated HUGO-GT™ humanized models designed to empower therapeutic candidates through successful IND submissions.
- Demonstrated Efficacy: Provides validation case studies, including LDL-C reduction in B6-hPCSK9 mice and dose-dependent knockdown in iPSC-dopaminergic neurons.
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