LNAplus™ Drug Discovery Platform
For discovering, testing and selecting ASOs for pre-clinical and clinical development, Secarna employs its proprietary, customized, in-house LNAplus™ drug discovery platform.
We start this process with bioinformatic analysis of the RNA coding for the target, providing us with sufficient information to select and synthesize relevant ASO drug candidates. These are then tested in our labs for target knock-down, dose dependency and toxicities in relevant cell lines or primary cells. ASOs testing successfully at this stage will be further investigated for their therapeutic effects and safety aspects. Molecules with the best overall therapeutic and safety profile are advanced to in vivo testing in relevant disease models.
Oligofyer™ - Secarna`s proprietary bioinformatics modelling system Maximizing safety and potency
Secarna has developed Oligofyer™, a proprietary bioinformatics screening system. With our system we can perform comprehensive compound screens, eliminate ASOs with theoretical sequence-specific off-target effects (match/mismatch analysis), avoid known potentially toxic sequence motifs and include cross reactivity with other species as required.
The power of our Oligofyer™ system allows us to rationally design ASO molecules which lead to a significantly higher hit rate in relevant assays.
- Thorough target evaluation
- Detailed compound screening
- Rationally designed ASO candidates
- High hit rate
Secarna`s state-of-the art integrated high throughput platformFocus on safety and efficacy
Our state-of-the-art high throughput platform has been validated by in-house projects and in partnerships with academic and industry collaborators. It has successfully proven to be fast, reliable, scalable, efficient and to provide for a uniquely integrated work flow.
We typically start the screening and selection process with up to 50.000 potential ASO candidates. Hundreds of ASOs are then synthesized and advanced to testing in cell lines or primary cells. At each stage of the process, candidates with highest potency and best safety profile are selected for further testing. At the end of this process, we select a hand full of candidates for in vivo testing. Information gained at different steps is then used to improve properties of short-selected compounds.
Secarna`s ASO discovery & developmentOptimization of safety & tolerability
Although later generation ASOs are generally safe and well tolerated, isolated safety and tolerability incidences were reported in selected clinical studies.
Secarna minimizes the potential of ASO therapy related complexities (e.g. unintended immune system responses, liver and kidney toxicities) by using the company`s own focused molecule design strategies and customized development solutions to address the afore mentioned matters and generate ASO candidates meeting the highest safety and efficacy requirements.
Secarna`s LNAplus™ based ASOs Efficacy enhancement with next generation chemical modification
Secarna`s ASOs are characterized by excellent stability and target affinity. Employing state-of-the-art chemistry, we achieve a circa 10-fold increased potency compared to preceding generations of ASOs.
Furthermore, our antisense oligonucleotides do not require conjugation, transfection or delivery reagents to exert their in vitro and in vivo activity in various cell types, tissues, and organs. IC50 levels of Secarna`s molecules are routinely in the low or sub nanomolar range without the support of delivery systems.
Consequently, our molecule`s therapeutic window can be expected to be greatly wider when compared to predating generations of ASOs and alternative modalities.