• Antisense oligonucleotide (ASO)-based therapeutics have evolved as a fully accepted therapeutic modality, next to small molecules and therapeutic antibodies
• During the drug candidate selection process, RNA-sequencing combined with bioinformatic screens could provide a valuable tool to assess ASO-mediated off-target effects
• Data from RNA-sequencing experiments will guide the selection process of ASO sequences to generate drug candidates with an improved target to off-target profile and therefore enable the generation of potent and safe drugs

Munich/Martinsried, Germany, Januar 20, 2021 – Secarna Pharmaceuticals GmbH & Co. KG (“Secarna”), a biopharmaceutical company focusing on the discovery and development of next generation antisense oligonucleotide (ASO) therapies to address challenging or previously undruggable targets via its LNAplus™ platform, today announced the publication of data on the role of bioinformatic screening in the process of generating and selecting ASOs. The data were published in Molecular Diagnostics & Therapy and the findings support the development of more efficient and safer ASO-based therapeutics. The article, “Using RNA-seq to assess off-target effects of antisense oligonucleotides in human cell lines” is available here: https://rdcu.be/cb7d4

In this study, Secarna selected two 17-mer ASOs to be analyzed for off-target effects in distinct human cell lines by a whole-transcriptome study using RNA sequencing. Experimentally determined analysis of gene expression combined with in silico prediction tools, allows Secarna to calibrate bioinformatics criteria for ASO sequence selection and to generate LNA-gapmer ASOs with improved target to off-target profiles, thereby reducing the risk for off-target-mediated toxicities to support the development of safer ASO-based therapeutics.

“The field of antisense oligonucleotides therapeutics is rapidly growing, and oligonucleotide-based gene expression modifiers have developed as fully accepted therapeutics next to small molecules and therapeutic antibodies,” said Jonas Renz, Managing Director and Co-founder of Secarna Pharmaceuticals. “Our recently published data offer compelling insights into the benefits of bioinformatic screening in the process of selecting ASOs. Thorough in silico screens using our proprietary Oligofyer™ platform, which is constantly improved using RNA sequencing data reduces the risk for ASO-mediated off-target RNA suppression, providing for safer ASO-based therapeutics and preventing candidates with a poor specificity profile from entering advanced testing.”

About Secarna’s proprietary drug discovery platform, LNAplus™

For discovering, testing and selecting antisense oligonucleotides (ASOs) for pre-clinical and clinical development, Secarna employs its proprietary, customized LNAplus™ drug discovery platform. LNAplus™ encompasses all aspects of drug discovery and pre-clinical development and has proven to be fast, reliable, scalable, efficient and to provide for a uniquely integrated workflow, enabling the discovery of novel antisense-based therapies for challenging or currently undruggable targets. The platform includes the powerful proprietary Oligofyer™ bioinformatics and LNA Vit(r)ox™ screening system for best-in-class high volume screening, and target-specific functional assays. Secarna’s platform and ASOs have been validated by numerous in-house projects as well as in several academic and industry collaborations.

About Secarna Pharmaceuticals GmbH & Co. KG

Secarna Pharmaceuticals is the next generation antisense oligonucleotide (ASO) company addressing high unmet medical needs in the areas of immuno-oncology, immunology, as well as viral, neurodegenerative and cardiometabolic diseases. Secarna’s mission is to maximize the performance and output of its proprietary LNAplus™ antisense oligonucleotide discovery platform, as well as to develop highly specific, safe, and efficacious best-in-class antisense therapies for challenging or currently undruggable targets. With over 15 development programs focusing on targets in indications where antisense-based approaches have clear benefits over other therapeutic modalities, Secarna is the leading European antisense drug discovery and development company.