Technology platforms

PB012 PureApta

The search for new drugs is a challenge for modern science and medicine. Developed in response to this need, the PureApta platform offers a streamlined and efficient method for discovering new biomolecules with diagnostic and therapeutic potential.

Aptamers, identified and selected via the PureApta platform, are single-stranded DNA molecules, which fold into unique three-dimensional structures, allowing them to bind virtually any target with high affinity and specificity. The PureApta platform is the first such modular platform in Poland, offering an enormous possibility of chemical modifications to further enhance aptamer stability, resistance to degradation and binding properties.

The PureApta platform is a complex and modular environment, facilitating the search for new aptamer-based drug candidates, as well as molecules with applications in medical diagnostics and in other biotech areas, where specific recognition and binding to a target is required.

OBJECTIVES

The primary goal of the PureApta project was development, optimization and implementation of a technological platform for selecting uniquely-modified, short DNA molecules (aptamers), which  interact specifically with various molecular targets.

DESCRIPTION

Aptamers are an oligonucleotide-based alternative to antibodies. Importantly, they differ from antibodies in that they are cheaper to manufacture, are smaller in size, are resistant to harsh conditions with an ability to self-renature, and exhibit low immunogenicity. The PureApta project served to develop an innovative technological platform that facilitates discovery programs seeking to identify aptamers as targeted drugs or as molecules with potential diagnostic use.

The PureApta platform is based on SELEX  (Systematic Evolution of Ligands by EXponential enrichment) technology. Molecular targets for aptamers include small molecules, proteins, antigens, and even whole cells. Selection experiments performed using the PureApta platform mimic evolution by choosing over successive rounds, increasingly better-binding aptamers, culminating in the identification of aptamers which specifically interact with their intended molecular targets.

To increase the probability of success in comparison to standard SELEX procedures, the PureApta platform introduces unique nucleotides into the aptamer via “click chemistry.” These unique modifications were engineered to enrich the repertoire of possible interactions between an aptamer and its molecular target. To ensure selection of target-specific aptamers, negative (counter) selection steps are extensively performed to eliminate non-binding and/or weakly-binding oligonucleotides. The entire selection procedure can be finely controlled, allowing adjustment of conditions tailored to the specific needs of a given discovery campaign. Selection procedures culminate with next-generation sequencing (NGS) and bioinformatic data analyses to identify the most-promising aptamer(s).

Thanks to the novel solutions and improvements of existing components, selection times using the PureApta platform have been shortened, while increasing the probability of identifying specific aptamers.

Effectiveness of the platform has been confirmed and validated from dozens of selections performed in numerous R&D projects.

PROJECT PROGRESS

The project has concluded. Synthesis and purification of unique, chemically-modified nucleotides, being the main advantage of the designed platform is now patented.

PROJECT DATA SHEET

Title: Innovative modular PureApta service platform for selection of modified aptamers with diagnostic and therapeutic potential
Program: Program Operacyjny Inteligentny Rozwój 2014-2020
Value: 2 327 629,50 PLN
Contribution of European Funds: 1 807 003,90 PLN
Start: 1st September 2016
End: 28th February 2019

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Pure Biologics - Harnessing the power of antibodies and aptamers