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Advancing RNA Therapeutics

Nucleic acid-based therapies and vaccines comprise diverse modalities such as mRNA, siRNA, antisense oligos (ASOs), and CRISPR/Cas, and offer many outstanding therapeutic characteristics (high specificity, functional diversity, easy adjustment). However, their design and development are highly complex processes that necessitate careful optimization of DNA/RNA molecules for both effect and stability, and the development of efficient delivery systems that target specific tissues and protect their cargo from premature clearance.

This is where Genedata comes in.


RNA Discovery. Better Candidates.

  • Captures all data relating to design, analysis, and production of a variety of RNA/DNA-based therapeutics end-to-end, and supports transparent decision-making at each step
  • Offers tools for DNA and RNA sequence design and in silico engineering out-of-the-box
  • Registers modified nucleosides, terminal modifications (e.g., 5' cap structures), and automatically calculates associated physicochemical properties
  • Tracks diverse delivery systems and their components, e.g., exact lipid composition of associated lipid nanoparticles (LNPs)
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Assay Analysis. Speed Up. Scale.

  • Accelerates testing of nucleic acid-based therapeutic candidates, by automating analysis of biological assays ranging from multiplexed qPCR to cellular functional assays
  • Streamlines and harmonizes analysis across an organization, improving data quality and enabling cross-assay analysis during target identification and validation, oligo optimization, or formulation screening
  • Ensures traceability from raw data to results, with automated data loading and vendor-agnostic Ready-to-Run instrument integrations
  • Visualizes and analyzes assay data according to the highest industry standards with built-in, scientifically validated normalization methods, fitting algorithms, and data models
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Bioprocess Development. Structured. E2E.

  • Tracks and integrates data during process development for nucleic acid therapeutics (USP, DSP and formulation), end-to-end
  • Facilitates identification and registration of critical quality attributes (CQAs), critical process parameters (CPPs) and critical material attributes (CMAs) during all production steps (e.g., in vitro transcription)
  • Enforces data integrity in complex formulations using automatic uniqueness validation
  • Ensures seamless data and material transfer between departments using an integrated request management system
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Mass Spectrometry-Based. Quality Ensured.

  • Streamlines MS-based analytics of therapeutic oligonucleotides, e.g., intact mass analysis, oligo metabolite IDs, impurity testing, and MS/MS analysis for sequence confirmation
  • Automates time-consuming and laborious manual operations during MS-based analyses
  • Facilitates and accelerates scale-up of nucleic acid-based therapeutic analytics
  • Supports deployment in regulated GxP environments through highly flexible custom workflows and compliance features
  • Enables systematic optimization of synthetic process parameters by eliminating analytical bottlenecks
More About RNA Characterization

Next-Generation Sequencing. Optimized.

  • Automates NGS-based workflows used in design, development, and quality control of nucleic acid-based therapeutics
  • Enables automated CRISPR-based target engineering, transgene integration assessment through target locus analysis (TLA), and registration and annotation of proprietary genomes
  • Facilitates NGS-based CQA analysis and Multi-Attribute Methodology (MAM) assessment during bioprocess development
  • Support all NGS-based biosafety testing in GMP environment
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Clinical & Translational Research. Accelerated.

  • Seamlessly integrates, harmonizes, and analyzes ever-increasing multimodal data in a secure, validation-ready environment
  • Facilitates advanced multi-omics analytics to empower discovery of predictive biomarkers and design of the most targeted and safest nucleic acid-based therapeutics
  • Tracks and visualizes clinical trial results in real time to support early data-informed decisions and ensure clinical success
  • Democratizes access to R&D data and data-driven insights across the whole organization to streamline cross-team collaboration and enhance productivity
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