Focused On-demand Library for Cystinosin

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.

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.

The library features a range of promising modulators, each detailed with 38 ADME-Tox and 32 physicochemical and drug-likeness parameters. Plus, each compound is presented with its ideal docking poses, affinity scores, and activity scores, ensuring a thorough insight.

Our high-tech, dedicated method is applied to construct targeted libraries.

Fig. 1. The sreening workflow of Receptor.AI

Our methodology employs molecular simulations to explore a wide array of proteins, capturing their dynamic states both individually and within complexes. Through ensemble virtual screening, we address conformational mobility, uncovering binding sites within functional regions and remote allosteric locations. This thorough exploration ensures no potential mechanism of action is overlooked, aiming to discover novel therapeutic targets and lead compounds across an extensive 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

O60931

UPID:

CTNS_HUMAN

Alternative names:

Alternative UPACC:

O60931; D3DTJ5; Q8IZ01; Q9UNK6

Background:

Cystinosin, encoded by the gene with accession number O60931, functions as a Cystine/H(+) symporter, crucial for exporting cystine from lysosomes. It plays a pivotal role in melanin synthesis by facilitating cystine export from melanosomes, thereby influencing pigment production. Beyond its transporter activity, Cystinosin positively regulates mTORC1 signaling in kidney proximal tubular cells and is involved in vesicle trafficking and lysosomal localization of LAMP2A.

Therapeutic significance:

Cystinosin's malfunction is linked to cystinosis, a lysosomal storage disease characterized by defective cystine transport, leading to tissue damage and renal failure. Understanding the role of Cystinosin could open doors to potential therapeutic strategies for treating various forms of cystinosis, including the nephropathic type, adult non-nephropathic type, and late-onset juvenile or adolescent nephropathic type.