Focused On-demand Library for Ceroid-lipofuscinosis neuronal protein 5

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.

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

Fig. 1. The sreening workflow of Receptor.AI

By deploying molecular simulations, our approach comprehensively covers a broad array of proteins, tracking their flexibility and dynamics individually and within complexes. Ensemble virtual screening is utilised to take into account conformational dynamics, identifying pivotal binding sites located within functional regions and at allosteric locations. This thorough exploration ensures that every conceivable mechanism of action is considered, aiming to identify new therapeutic targets and advance lead compounds throughout a vast spectrum of biological functions.

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

Reaxense

upacc

O75503

UPID:

CLN5_HUMAN

Alternative names:

Alternative UPACC:

O75503; B3KQK7

Background:

Ceroid-lipofuscinosis neuronal protein 5, encoded by the gene with accession number O75503, is pivotal in neurodegenerative disease mechanisms. It influences the retrograde trafficking of lysosomal sorting receptors SORT1 and IGF2R, essential for cellular homeostasis. By regulating RAB7A localization and activation, it ensures the proper recruitment of the retromer complex to the endosomal membrane, a critical step in lysosomal function.

Therapeutic significance:

Given its role in neuronal ceroid lipofuscinosis type 5, a disease marked by seizures, dementia, and visual loss due to autofluorescent liposomal accumulation, understanding Ceroid-lipofuscinosis neuronal protein 5 could open doors to potential therapeutic strategies. Targeting its pathway may offer novel treatments for this progressive neurodegenerative disorder.