Focused On-demand Library for NPC intracellular cholesterol transporter 1

Focused On-demand Libraries - Reaxense 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.

Our selection of compounds is from a large virtual library of over 60 billion molecules. The production and distribution of these compounds are managed by our partner Reaxense.

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 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 stands out due to several important features:

The Receptor.AI platform compiles comprehensive data on the target protein, encompassing previous experiments, literature, known ligands, structural details, and more, leading to a higher chance of selecting the most relevant compounds.
Advanced molecular simulations on the platform help pinpoint potential binding sites, making the compounds in our focused library ideal for finding allosteric inhibitors and targeting cryptic pockets.
Receptor.AI boasts over 50 tailor-made AI models, rigorously tested and proven in various drug discovery projects and research initiatives. They are crafted for efficacy, dependability, and precision, all of which are key in creating our focused libraries.
Beyond creating focused libraries, Receptor.AI offers comprehensive services and complete solutions throughout the preclinical drug discovery phase. Our success-based pricing model minimises risk and maximises the mutual benefits of the project's success.

partner

Reaxense

upacc

O15118

UPID:

NPC1_HUMAN

Alternative names:

Niemann-Pick C1 protein

Alternative UPACC:

O15118; B4DET3; Q9P130

Background:

NPC intracellular cholesterol transporter 1, also known as Niemann-Pick C1 protein, plays a pivotal role in cholesterol homeostasis. It facilitates the egress of cholesterol from the endosomal/lysosomal compartment, crucial for cellular lipid balance. The protein interacts with NPC2 for cholesterol transfer and binds oxysterol with higher affinity, indicating a nuanced role in lipid signaling and metabolism.

Therapeutic significance:

Niemann-Pick disease C1, a lysosomal storage disorder, is directly linked to mutations in the gene encoding this protein. Understanding its function and the molecular mechanisms underlying its interaction with cholesterol and other molecules could pave the way for targeted therapies, potentially alleviating the severe neurological and visceral symptoms associated with this condition.