Focused On-demand Library for Alpha-galactosidase A

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.

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.

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.

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

P06280

UPID:

AGAL_HUMAN

Alternative names:

Alpha-D-galactosidase A; Alpha-D-galactoside galactohydrolase; Galactosylgalactosylglucosylceramidase GLA; Melibiase

Alternative UPACC:

P06280; Q6LER7

Background:

Alpha-galactosidase A, also known as Alpha-D-galactosidase A, Alpha-D-galactoside galactohydrolase, Galactosylgalactosylglucosylceramidase GLA, and Melibiase, plays a crucial role in the lysosomal degradation of glycosphingolipids. This enzyme's activity is pivotal for the breakdown and removal of glycolipids from the body, ensuring cellular health and proper metabolic functions.

Therapeutic significance:

The deficiency of Alpha-galactosidase A leads to Fabry disease, a rare X-linked sphingolipidosis characterized by the systemic accumulation of globotriaosylceramide. This accumulation causes a range of symptoms, including skin lesions, ocular deposits, and renal failure. Understanding the enzyme's function and its genetic variants opens avenues for targeted gene therapy and enzyme replacement therapies, offering hope for patients with Fabry disease.