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Discovery Sciences

RNA Therapeutics Drug Discovery Services

Transform mRNA and oligonucleotide-based innovations into development-ready candidates with integrated drug discovery expertise. 

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As RNA therapeutics continue to reshape the treatment landscape across rare diseases, oncology, neuroscience, cardiovascular and metabolic disorders, and immunology developers need a partner capable of navigating the scientific, technical, and translational complexities of RNA drug development. Our RNA therapeutics CRO services support you from target identification through preclinical candidate selection. Combining disease biology expertise, advanced screening capabilities, translational pharmacology, and modality-specific development strategies, we help accelerate the path from discovery to clinical readiness.

End-to-End RNA Therapeutics Drug Discovery Services

mRNA and oligonucleotide therapeutics offer unique opportunities to modulate gene expression, silence disease-causing targets, restore protein function, express proteins and antibodies, correct mutations, and address previously undruggable pathways. Success requires a comprehensive understanding of disease biology, combined with drug discovery services including target validation, sequence optimization, in vitro screening and functional assays, delivery strategies, in vivo efficacy and pharmacology assessment, safety evaluation, and regulatory strategy support. Our integrated RNA therapeutics drug discovery services are designed to help sponsors reduce development risk and make confident portfolio decisions at every stage.

RNA Therapeutic Modalities We Support 

Every RNA-based drug modality presents distinct development challenges and opportunities. Our scientific teams develop customized strategies aligned with the mechanism of action, therapeutic area, and target product profile of your program.
Antisense Oligonucleotide (ASO) Development
siRNA/RNA Interference Therapeutics
mRNA Therapeutics
Emerging RNA Platforms

Antisense Oligonucleotide (ASO) Development 

Advance ASO candidates through rational design, sequence optimization, formulation, efficacy testing, early safety and translational evaluation. We help identify the most promising candidates while generating the data required for downstream development decisions.

siRNA/RNA Interference Therapeutics 

Leverage RNAi (RNA interference) approaches to selectively silence disease-associated genes. Our discovery workflows support target validation, siRNA design and screening, efficacy studies, and preclinical proof-of-concept generation.

 mRNA Therapeutics

Develop novel mRNA therapeutics with support for construct evaluation, biological characterization, translational assessment, formulation and preclinical testing strategies.

Emerging RNA Platforms

We support next-generation RNA medicines, including mRNA-encoded antibodies, mRNA CAR-T cell therapies and combination approaches that require specialized discovery expertise.

Rare Disease Drug Discovery Services

RNA therapeutics, including ASOs and siRNA therapies, are driving significant clinical success in the treatment of rare, ultra-rare and N=1 disorders. Our rare disease drug discovery services are designed to help you move promising therapies toward patients faster, with a focus on scientific rigor, collaboration and translational insight.

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Translational Science for RNA Therapeutics

Successful RNA and oligonucleotide therapeutics programs depend on a strong translational foundation. Our scientists integrate disease biology, biomarker strategy, pharmacology, and translational endpoints to generate meaningful data that support development decisions and regulatory interactions.

By linking preclinical outcomes with clinical relevance, we help sponsors:

    • Improve candidate selection
    • Understand mechanism of action
    • Identify predictive biomarkers
    • Establish proof of mechanism
    • Predict dose levels
    • Reduce development uncertainty
    • Enable informed go/no-go decisions

Why Choose Our RNA Therapeutics CRO?

RNA drug development demands more than technical execution. It requires scientific partnership, strategic guidance, and integrated expertise across discovery, preclinical, and regulatory development. Whether you are advancing an early-stage discovery concept or preparing a lead candidate for IND-enabling studies, our integrated RNA therapeutics drug discovery services can help accelerate progress while reducing development risk.

  • Deep RNA Therapeutics Expertise - Experienced scientists supporting multiple RNA and oligonucleotide modalities and mechanisms of action.
  • Integrated Discovery Capabilities - A seamless approach spanning disease biology, screening, pharmacology, and translational research.
  • Data-Driven Decision Making - Robust scientific evidence generated to support candidate advancement and portfolio prioritization.
  • Focus on Development Success - Programs designed with future development milestones in mind, helping sponsors move efficiently toward clinical evaluation.
  • Flexible Partnership Models - Support for standalone studies, functional work packages, or fully integrated discovery programs.
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Frequently Asked Questions About RNA Therapeutics Drug Discovery Services
What is an RNA Therapeutics CRO?

An RNA therapeutics CRO is a contract research organization that provides scientific services supporting the discovery, optimization, and preclinical development of RNA-based medicines, including ASOs, siRNA, mRNA therapeutics, and RNA vaccines.
What RNA therapeutic modalities do you support?

We support antisense oligonucleotides (ASOs), siRNA, mRNA therapeutics (gene replacement), RNA vaccines, and emerging RNA-based platforms, such as mRNA-encoded antibodies or CAR-T, and gene editing therapies.
What services are included in RNA therapeutics drug discovery?

Services may include target identification and evaluation, disease biology, oligonucleotide design, candidate screening, translational research, biomarker development, formulation, pharmacology studies, efficacy testing, and candidate selection.
How do RNA therapeutics differ from traditional small molecule drugs?

RNA therapeutics modulate gene expression or protein production at the nucleic acid level, enabling approaches that may not be achievable through conventional small molecule or biologic therapies.
Can you support preclinical development after candidate selection?

Yes. Our teams provide integrated support through formulation, preclinical pharmacology, safety assessment, biomarker strategy, and planning for IND-enabling activities.
What disease areas are best suited for RNA therapeutics?

RNA-based therapies are increasingly being developed for rare genetic disorders, oncology, liver diseases, neurological conditions, cardiovascular and metabolic diseases, retinal diseases, immunological disorders, and infectious diseases.