Focused On-demand Library for Sphingomyelin phosphodiesterase

Focused On-demand Libraries - Reaxense Collaboration

Explore the Potential with AI-Driven Innovation

Our detailed focused library is generated on demand with advanced virtual screening and parameter assessment technology powered by the Receptor.AI drug discovery platform. This method surpasses traditional approaches, delivering compounds of better quality with enhanced 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.

We use our state-of-the-art dedicated workflow for designing focused 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

P17405

UPID:

ASM_HUMAN

Alternative names:

Acid sphingomyelinase

Alternative UPACC:

P17405; A8K8M3; E9PKS3; P17406; Q13811; Q16837; Q16841

Background:

Sphingomyelin phosphodiesterase, also known as Acid sphingomyelinase, plays a pivotal role in converting sphingomyelin to ceramide within lysosomes and has a significant function in cholesterol export from intraendolysosomal membranes. This enzyme exists in two forms: one targeted to the endolysosomal compartment and another secreted extracellularly, which is crucial for plasma membrane repair and host defense mechanisms.

Therapeutic significance:

The enzyme's dysfunction is linked to Niemann-Pick disease types A and B, lysosomal storage disorders characterized by sphingomyelin accumulation leading to severe neurodegenerative and visceral symptoms. Understanding the enzyme's role could pave the way for innovative treatments targeting these genetic disorders.