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

Focused On-demand Libraries - Reaxense Collaboration

Explore the Potential with AI-Driven Innovation

This extensive focused library is tailor-made using the latest virtual screening and parameter assessment technology, operated by the Receptor.AI drug discovery platform. This technique is more effective than traditional methods, offering compounds with improved activity, selectivity, and safety.

From a virtual chemical space containing more than 60 billion molecules, we precisely choose certain compounds. Our collaborator, Reaxense, aids in their synthesis and provision.

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

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 distinguishes itself through several key aspects:

The Receptor.AI platform integrates all available data about the target protein, including past experiments, literature data, known ligands, structural information and more. This consolidated approach maximises the probability of prioritising highly relevant compounds.
The platform uses sophisticated molecular simulations to identify possible binding sites so that the compounds in the focused library are suitable for discovering allosteric inhibitors and the binders for cryptic pockets.
The platform integrates over 50 highly customisable AI models, which are thoroughly tested and validated on a multitude of commercial drug discovery programs and research projects. It is designed to be efficient, reliable and accurate. All this power is utilised when producing the focused libraries.
In addition to producing the focused libraries, Receptor.AI provides services and end-to-end solutions at every stage of preclinical drug discovery. The pricing model is success-based, which reduces your risks and leverages 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.