Therapeutic Modalities
RNA-Targeted
Small Molecules
Axcelead Holistic Solution for
RNA-targeted Small Molecule
Axcelead has comprehensive capabilities in integrated RNA-targeted small molecule drug discovery at the preclinical stages. No matter what stage your project is in, Axcelead will offer a tailored solution for you, whether you need support for part or all of it.
RNA-targeted Small Molecules
RNA-targeted small molecules represent a novel approach in drug discovery that involves the design of compounds to interact specifically with RNA molecules within cells. Unlike traditional drug discovery methods that primarily focus on targeting proteins, RNA-targeted small molecules aim to modulate RNA-derived function, which plays crucial roles in various cellular processes and disease mechanisms.
The RNA-targeted small molecules platform offers many advantages, including:
- Broad Target Space: RNA molecules represent a vast and diverse set of potential drug targets, offering opportunities to address diseases with unmet medical needs.
- Drugging Undruggable Targets: RNA-targeted small molecules can target RNA structures or sequences that are traditionally considered challenging to drug with other modalities.
- Oral administration: Systemic exposure including central nervous system by oral administration
In contrast, it also faces challenges:
- Discovery of “real promising” hits: Insufficiency of RNA-focused compound library and gap between cell-free and cellular assay remain significant challenges.
- Specificity: Ensuring specificity and minimizing off-target effects is crucial to the development of safe and effective RNA-targeted small molecule therapeutics.
- Optimization: Structure-based drug design is generally difficult due to the flexibility of RNA structures.
Axcelead Platform Solves Your
RNA-targeted Small Molecule Challenges
Special RNA-focused Library and Assay Capabilities
- RNA splicing focused library: ca.2500 compounds
All compounds were newly designed and synthesized by Axcelead and potential IP. - General RNA focused library: ca.12,800 compounds
The library was selected from our high-quality 1.2+ M library based on originalconcepts for various RNA-targeted small molecule discovery - Well-experienced and various assay capabilities
mini-gene reporter AS-MSmRNA-templated in vitro translationqPCRHigh content screening - Hit validation capabilities
SPRX-ray crystallography
Multiple Technologies
- DNA binding assay
High-throughput counter assay using DNA from cells enable to discover promising chemotypes and decrease genotoxicity risk - Comprehensive selectivity evaluation
Transcriptomics (RNA-seq, ampli-seq) and bioinformatics analysis followed by toxicity assessment
