Focused On-demand Library for Ganglioside GM2 activator

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.

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

It includes comprehensive molecular simulations of the catalytic and allosteric binding pockets and the ensemble virtual screening accounting for their conformational mobility. In the case of designing modulators, the structural changes induced by reaction intermediates are taken into account to leverage 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

P17900

UPID:

SAP3_HUMAN

Alternative names:

Cerebroside sulfate activator protein; GM2-AP; Sphingolipid activator protein 3

Alternative UPACC:

P17900; B2R699; D3DQH6; Q14426; Q14428; Q6LBL5

Background:

The Ganglioside GM2 activator, known by its alternative names such as Cerebroside sulfate activator protein, GM2-AP, and Sphingolipid activator protein 3, plays a crucial role in lipid metabolism. It is instrumental in the degradation of gangliosides, particularly GM2, by facilitating its interaction with beta-hexosaminidase A. This interaction is vital for the conversion of GM2 to GM3, a process essential for normal brain function.

Therapeutic significance:

GM2-gangliosidosis AB, a lysosomal storage disease characterized by the accumulation of GM2 gangliosides, is directly associated with the Ganglioside GM2 activator protein. Understanding the protein's function and its interaction with hexosaminidase A offers a promising avenue for developing targeted therapies for this condition, highlighting the protein's significant therapeutic potential.