Focused On-demand Library for Alpha-L-iduronidase

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.

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.

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.

Our top-notch dedicated system is used to design specialised 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.

Several key aspects differentiate our library:

Receptor.AI compiles an all-encompassing dataset on the target protein, including historical experiments, literature data, known ligands, and structural insights, maximising the chances of prioritising the most pertinent compounds.
The platform employs state-of-the-art molecular simulations to identify potential binding sites, ensuring the focused library is primed for discovering allosteric inhibitors and binders of concealed pockets.
Over 50 customisable AI models, thoroughly evaluated in various drug discovery endeavours and research projects, make Receptor.AI both efficient and accurate. This technology is integral to the development of our focused libraries.
In addition to generating focused libraries, Receptor.AI offers a full range of services and solutions for every step of preclinical drug discovery, with a pricing model based on success, thereby reducing risk and promoting joint project success.

partner

Reaxense

upacc

P35475

UPID:

IDUA_HUMAN

Alternative names:

Alternative UPACC:

P35475; B3KWK6

Background:

Alpha-L-iduronidase, encoded by the gene with accession number P35475, plays a crucial role in the lysosomal degradation of glycosaminoglycans. This enzyme's deficiency leads to the accumulation of dermatan sulfate and heparan sulfate, manifesting in various forms of mucopolysaccharidosis type 1 (MPS1), including MPS1H, MPS1H/S, and MPS1S, each differing in severity and clinical presentation.

Therapeutic significance:

The therapeutic significance of Alpha-L-iduronidase is profound, particularly in the context of mucopolysaccharidosis type 1 (MPS1). Targeted enzyme replacement therapies have shown promise in managing symptoms and improving quality of life for patients with MPS1, highlighting the enzyme's critical role in disease pathology and treatment.