Focused On-demand Library for Ganglioside GM2 activator

Details

Focused On-demand Libraries - Receptor.AI 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.


The compounds are cherry-picked from the vast virtual chemical space of over 60B molecules. The synthesis and delivery of compounds is facilitated by Reaxense.


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.


We employ our advanced, specialised process to create targeted libraries for enzymes.


 

Fig. 1. The screening 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
Receptor.AI
 
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:
Ganglioside GM2 activator, also referred to as GM2-AP, plays a pivotal role in the cellular lipid metabolism by binding and presenting gangliosides to beta-hexosaminidase A for degradation. This process is crucial for the maintenance of neuronal cell health. The protein's ability to transfer cholesterol and its phospholipase activity further underscore its importance in cellular processes.

THERAPEUTIC SIGNIFICANCE:
The involvement of the Ganglioside GM2 activator in GM2-gangliosidosis AB, a disease marked by harmful accumulation of GM2 gangliosides, underscores the therapeutic significance of this protein. Targeting the protein's function could lead to innovative treatments for this and potentially other lysosomal storage diseases, making it a focal point of drug discovery efforts.

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