Focused On-demand Library for Ribitol 5-phosphate transferase FKRP

Focused On-demand Libraries - Reaxense Collaboration

Explore the Potential with AI-Driven Innovation

This comprehensive focused library is produced on demand with state-of-the-art virtual screening and parameter assessment technology driven by Receptor.AI drug discovery platform. This approach outperforms traditional methods and provides higher-quality compounds with superior activity, selectivity and safety.

We carefully select specific compounds from a vast collection of over 60 billion molecules in virtual chemical space. Our partner Reaxense helps in synthesizing and delivering these compounds.

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.

Our high-tech, dedicated method is applied to construct targeted libraries for enzymes.

Fig. 1. The sreening workflow of Receptor.AI

It includes in-depth molecular simulations of both the catalytic and allosteric binding pockets, with ensemble virtual screening focusing on their conformational flexibility. For modulators, the process includes considering the structural shifts due to reaction intermediates to boost activity and selectivity.

Our library stands out due to several important features:

The Receptor.AI platform compiles comprehensive data on the target protein, encompassing previous experiments, literature, known ligands, structural details, and more, leading to a higher chance of selecting the most relevant compounds.
Advanced molecular simulations on the platform help pinpoint potential binding sites, making the compounds in our focused library ideal for finding allosteric inhibitors and targeting cryptic pockets.
Receptor.AI boasts over 50 tailor-made AI models, rigorously tested and proven in various drug discovery projects and research initiatives. They are crafted for efficacy, dependability, and precision, all of which are key in creating our focused libraries.
Beyond creating focused libraries, Receptor.AI offers comprehensive services and complete solutions throughout the preclinical drug discovery phase. Our success-based pricing model minimises risk and maximises the mutual benefits of the project's success.

partner

Reaxense

upacc

Q9H9S5

UPID:

FKRP_HUMAN

Alternative names:

Fukutin-related protein; Ribitol-5-phosphate transferase

Alternative UPACC:

Q9H9S5; A8K5G7

Background:

Ribitol 5-phosphate transferase FKRP, also known as Fukutin-related protein, plays a crucial role in the post-translational modification of alpha-dystroglycan (DAG1). This enzyme catalyzes the transfer of ribitol 5-phosphate to the phosphorylated O-mannosyl trisaccharide, a key step in the biosynthesis of a ligand-binding moiety essential for muscle integrity.

Therapeutic significance:

Mutations in FKRP are linked to a spectrum of muscular dystrophies, including congenital muscular dystrophy-dystroglycanopathy with brain and eye anomalies (A5), congenital muscular dystrophy-dystroglycanopathy with or without intellectual development impairment (B5), and limb-girdle muscular dystrophy-dystroglycanopathy (C5). Understanding the role of FKRP could open doors to potential therapeutic strategies for these debilitating conditions.