Focused On-demand Library for Poly(U)-binding-splicing factor PUF60

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.

In the library, a selection of top modulators is provided, each marked with 38 ADME-Tox and 32 parameters related to physicochemical properties and drug-likeness. Also, every compound comes with its best docking poses, affinity scores, and activity scores, providing a comprehensive overview.

We use our state-of-the-art dedicated workflow for designing focused libraries.

Fig. 1. The sreening workflow of Receptor.AI

Our methodology leverages molecular simulations to examine a vast array of proteins, capturing their dynamics in both isolated forms and in complexes with other proteins. Through ensemble virtual screening, we thoroughly account for the protein's conformational mobility, identifying critical binding sites within functional regions and distant allosteric locations. This detailed exploration ensures that we comprehensively assess every possible mechanism of action, with the objective of identifying novel therapeutic targets and lead compounds that span a wide 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

Q9UHX1

UPID:

PUF60_HUMAN

Alternative names:

60 kDa poly(U)-binding-splicing factor; FUSE-binding protein-interacting repressor; Ro-binding protein 1; Siah-binding protein 1

Alternative UPACC:

Q9UHX1; A8K8K8; Q969E7; Q96D94; Q96H63; Q99628; Q9NZA0; Q9UJY7

Background:

Poly(U)-binding-splicing factor PUF60, also known as 60 kDa poly(U)-binding-splicing factor, plays a pivotal role in nuclear processes including pre-mRNA splicing, apoptosis, and transcription regulation. It influences MYC transcription, acts as a transcriptional repressor, and is involved in DNA binding at active promoter regions. PUF60's interaction with FUBP1 and its involvement in alternative splicing of mRNAs highlight its multifaceted biological functions.

Therapeutic significance:

PUF60's association with Verheij syndrome, characterized by growth retardation and developmental delays, underscores its clinical relevance. Understanding the role of Poly(U)-binding-splicing factor PUF60 could open doors to potential therapeutic strategies for managing this syndrome and possibly other related genetic disorders.