Focused On-demand Library for Lysosomal acid glucosylceramidase

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.

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.

The library includes a list of the most promising modulators annotated with 38 ADME-Tox and 32 physicochemical and drug-likeness parameters. Also, each compound is presented with its optimal 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 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

P04062

UPID:

GBA1_HUMAN

Alternative names:

Acid beta-glucosidase; Alglucerase; Beta-glucocerebrosidase; Beta-glucosylceramidase 1; Cholesterol glucosyltransferase; Cholesteryl-beta-glucosidase; D-glucosyl-N-acylsphingosine glucohydrolase; Glucosylceramidase beta 1; Imiglucerase; Lysosomal cholesterol glycosyltransferase; Lysosomal galactosylceramidase; Lysosomal glycosylceramidase

Alternative UPACC:

P04062; A8K796; B7Z5G2; B7Z6S1; J3KQG4; J3KQK9; Q16545; Q4VX22; Q6I9R6; Q9UMJ8

Background:

Lysosomal acid glucosylceramidase, known by names such as Acid beta-glucosidase and Imiglucerase, plays a pivotal role in the lysosomal degradation of glucosylceramide into ceramide and glucose. This enzyme is essential for the turnover of cellular membranes and the metabolism of complex lipids.

Therapeutic significance:

Mutations in this enzyme lead to Gaucher disease, a lysosomal storage disorder with varying forms and severities, including neuronopathic and non-neuronopathic types. Its involvement in Parkinson disease highlights its broader impact on neurodegenerative disorders, making it a target for therapeutic intervention.