Focused On-demand Library for H/ACA ribonucleoprotein complex subunit DKC1

Focused On-demand Libraries - Reaxense Collaboration

Explore the Potential with AI-Driven Innovation

Our detailed focused library is generated on demand with advanced virtual screening and parameter assessment technology powered by the Receptor.AI drug discovery platform. This method surpasses traditional approaches, delivering compounds of better quality with enhanced 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 for enzymes.

Fig. 1. The sreening workflow of Receptor.AI

The procedure entails thorough molecular simulations of the catalytic and allosteric binding pockets, accompanied by ensemble virtual screening that factors in their conformational flexibility. When developing modulators, the structural modifications brought about by reaction intermediates are factored in to optimize activity and selectivity.

Our library is unique due to several crucial aspects:

Receptor.AI compiles all relevant data on the target protein, such as past experimental results, literature findings, known ligands, and structural data, thereby enhancing the likelihood of focusing on the most significant compounds.
By utilizing advanced molecular simulations, the platform is adept at locating potential binding sites, rendering the compounds in the focused library well-suited for unearthing allosteric inhibitors and binders for hidden pockets.
The platform is supported by more than 50 highly specialized AI models, all of which have been rigorously tested and validated in diverse drug discovery and research programs. Its design emphasizes efficiency, reliability, and accuracy, crucial for producing focused libraries.
Receptor.AI extends beyond just creating focused libraries; it offers a complete spectrum of services and solutions during the preclinical drug discovery phase, with a success-dependent pricing strategy that reduces risk and fosters shared success in the project.

partner

Reaxense

upacc

O60832

UPID:

DKC1_HUMAN

Alternative names:

CBF5 homolog; Dyskerin; Nopp140-associated protein of 57 kDa; Nucleolar protein NAP57; Nucleolar protein family A member 4; snoRNP protein DKC1

Alternative UPACC:

O60832; F5BSB3; O43845; Q96G67; Q9Y505

Background:

H/ACA ribonucleoprotein complex subunit DKC1, also known as Dyskerin, plays a pivotal role in cellular processes, including ribosome biogenesis and telomere maintenance. It catalyzes the pseudouridylation of rRNA, a modification crucial for stabilizing rRNA structure and function. Dyskerin's involvement in cell adhesion and proliferation further underscores its multifaceted biological significance.

Therapeutic significance:

Dyskerin's mutation is directly linked to Dyskeratosis congenita and Hoyeraal-Hreidarsson syndrome, diseases characterized by bone marrow failure and developmental anomalies. Understanding Dyskerin's role could pave the way for innovative treatments targeting these genetic disorders, highlighting its therapeutic potential.