Focused On-demand Library for Twinkle mtDNA helicase

Available from Reaxense
Predicted by Alphafold

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 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 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.







Alternative names:

Progressive external ophthalmoplegia 1 protein; T7 gp4-like protein with intramitochondrial nucleoid localization; T7-like mitochondrial DNA helicase; Twinkle protein, mitochondrial

Alternative UPACC:

Q96RR1; B2CQL2; Q6MZX2; Q6PJP5; Q96RR0


Twinkle mtDNA helicase, also known as Progressive external ophthalmoplegia 1 protein, plays a pivotal role in mitochondrial DNA replication and repair. It exhibits DNA strand separation activity essential for the formation of a processive replication fork, crucial for leading strand synthesis. This activity is facilitated by forming a replisome complex with POLG and mtSDB, highlighting its significance in mitochondrial function.

Therapeutic significance:

Given its involvement in diseases such as Progressive external ophthalmoplegia with mitochondrial DNA deletions, autosomal dominant, 3, Mitochondrial DNA depletion syndrome 7, and Perrault syndrome 5, Twinkle mtDNA helicase represents a promising target for therapeutic intervention. Understanding the role of Twinkle mtDNA helicase could open doors to potential therapeutic strategies, especially in conditions linked to mitochondrial dysfunction.

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