Focused On-demand Library for Alpha-L-iduronidase

Focused On-demand Libraries - Reaxense Collaboration

Explore the Potential with AI-Driven Innovation

The specialised, focused library is developed on demand with the most recent virtual screening and parameter assessment technology, guided by the Receptor.AI drug discovery platform. This approach exceeds the capabilities of traditional methods and offers compounds with higher 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.

Contained in the library are leading modulators, each labelled with 38 ADME-Tox and 32 physicochemical and drug-likeness qualities. In addition, each compound is illustrated with its optimal docking poses, affinity scores, and activity scores, giving a complete picture.

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

Fig. 1. The sreening workflow of Receptor.AI

The method includes detailed molecular simulations of the catalytic and allosteric binding pockets, along with ensemble virtual screening that considers their conformational flexibility. In the design of modulators, structural changes induced by reaction intermediates are taken into account to enhance 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

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.