Focused On-demand Library for BLOC-3 complex member HPS4

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

Fig. 1. The sreening workflow of Receptor.AI

Our methodology leverages molecular simulations to examine a vast array of proteins, capturing their dynamics in both isolated forms and in complexes with other proteins. Through ensemble virtual screening, we thoroughly account for the protein's conformational mobility, identifying critical binding sites within functional regions and distant allosteric locations. This detailed exploration ensures that we comprehensively assess every possible mechanism of action, with the objective of identifying novel therapeutic targets and lead compounds that span a wide spectrum of biological functions.

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

Q9NQG7

UPID:

HPS4_HUMAN

Alternative names:

Hermansky-Pudlak syndrome 4 protein; Light-ear protein homolog

Alternative UPACC:

Q9NQG7; B1AHQ4; Q5H8V6; Q96LX6; Q9BY93; Q9UH37; Q9UH38

Background:

The BLOC-3 complex member HPS4, also known as Hermansky-Pudlak syndrome 4 protein or Light-ear protein homolog, plays a pivotal role in the BLOC-3 complex. This complex acts as a guanine exchange factor for RAB32 and RAB38, facilitating the conversion from an inactive GDP-bound form to an active GTP-bound form. It is crucial in melanin production and melanosome biogenesis, promoting membrane localization of RAB32 and RAB38.

Therapeutic significance:

HPS4 is directly implicated in Hermansky-Pudlak syndrome 4, a disorder characterized by oculocutaneous albinism, bleeding due to platelet storage pool deficiency, and lysosomal storage defects. Understanding the role of HPS4 could open doors to potential therapeutic strategies for this syndrome, particularly in addressing pulmonary fibrosis, a leading cause of premature death.