Focused On-demand Library for pre-mRNA splicing regulator USH1G

Focused On-demand Libraries - Reaxense Collaboration

Explore the Potential with AI-Driven Innovation

The focused library is created on demand with the latest virtual screening and parameter assessment technology, supported by the Receptor.AI drug discovery platform. This method is more effective than traditional methods and results in higher-quality compounds with better activity, selectivity, and safety.

The compounds are cherry-picked from the vast virtual chemical space of over 60B molecules. The synthesis and delivery of compounds is facilitated by our partner Reaxense.

The library includes a list of the most effective modulators, each annotated with 38 ADME-Tox and 32 physicochemical and drug-likeness parameters. Furthermore, each compound is shown with its optimal docking poses, affinity scores, and activity scores, offering a detailed summary.

We utilise our cutting-edge, exclusive workflow to develop focused libraries.

Fig. 1. The sreening workflow of Receptor.AI

By deploying molecular simulations, our approach comprehensively covers a broad array of proteins, tracking their flexibility and dynamics individually and within complexes. Ensemble virtual screening is utilised to take into account conformational dynamics, identifying pivotal binding sites located within functional regions and at allosteric locations. This thorough exploration ensures that every conceivable mechanism of action is considered, aiming to identify new therapeutic targets and advance lead compounds throughout a vast spectrum of biological functions.

Key features that set our library apart include:

The Receptor.AI platform integrates extensive information about the target protein, such as historical experiments, academic research, known ligands, and structural insights, thereby increasing the likelihood of identifying highly relevant compounds.
The platform’s sophisticated molecular simulations are designed to discover potential binding sites, ensuring that our focused library is optimal for the discovery of allosteric inhibitors and binders for cryptic pockets.
With over 50 customisable AI models, verified through extensive testing in commercial drug discovery and research, Receptor.AI is efficient, reliable, and precise. These models are essential in the production of our focused libraries.
Receptor.AI not only produces focused libraries but also provides full services and solutions at every stage of preclinical drug discovery, with a success-based pricing structure that aligns our interests with the success of your project.

partner

Reaxense

upacc

Q495M9

UPID:

USH1G_HUMAN

Alternative names:

Scaffold protein containing ankyrin repeats and SAM domain; Usher syndrome type-1G protein

Alternative UPACC:

Q495M9; Q8N251

Background:

The pre-mRNA splicing regulator USH1G, also known as Scaffold protein containing ankyrin repeats and SAM domain, plays a crucial role in pre-mRNA splicing. It regulates the release and transfer of U4/U6.U5 tri-snRNP complexes, contributing to the assembly of the pre-catalytic spliceosome on target pre-mRNAs. Additionally, it is involved in the recycling of snRNPs during splicing and plays a role in MAGI2-mediated endocytosis. It is also essential for the development and maintenance of cochlear hair cell bundles, required for normal hearing.

Therapeutic significance:

USH1G's involvement in Usher syndrome 1G, characterized by profound congenital sensorineural deafness, absent vestibular function, and progressive retinitis pigmentosa, highlights its therapeutic significance. Understanding the role of USH1G could open doors to potential therapeutic strategies for this condition.