Research & Development

A multi-faceted approach to a complex problem

While the RNAi pathway is well understood, we believe that its therapeutic application requires a broad, flexible and innovative approach. Over the last decade, we have built a portfolio of capabilities in molecular biology, cellular biology, peptide and lipid chemistry, pharmacology, and bioinformatics. These capabilities, plus the in-licensing of key RNAi-related intellectual property (IP), have enabled us to swiftly become a leading RNAi-based therapeutics company with a robust pre-clinical pipeline.

Currently, we are developing RNAi therapies for oncology, inflammation, metabolic disorders and viral infections. Our platform is built on a broad portfolio of RNAi technologies and delivery methods. We focus on siRNA constructs such as Dicer substrates and three-stranded meroduplexes. We are also building in-depth unique expertise in lipid- and peptide-based delivery systems.

Fueling RNAi discovery and development

Working together, our multi-disciplinary team has developed a "target to clinic" discovery engine that optimizes leading-edge RNAi technology and novel delivery platforms. This engine enables us to apply RNAi technology and delivery to the development of safe, effective RNAi therapeutics for a wide range of medical conditions both independently and in collaboration with our partners.

Research team

MDRNA's employees are dedicated to the development and commercialization of innovative RNAi technologies, including therapeutics and delivery systems. The MDRNA team is composed of talented, focused individuals who are committed to pursuing products that may offer significant contributions to the future of human health.

Our cutting-edge science and therapeutic programs have enabled us to attract experts in molecular biology, peptide and lipid chemistry, pharmacology, bioinformatics, and quality control as well as a very experienced pharmaceutical management team and a distinguished Scientific Advisory Board. By bringing on to the team the best and brightest in the industry, MDRNA is creating an environment that fosters creative development and innovative research in an atmosphere of teamwork and dedication.

Combining speed and efficiency

We have the ability to identify, synthesize and formulate a wide range of siRNA constructs and then conduct both in vitro and in vivo testing as well as complete pre-clinical evaluation of RNAi-based compounds for human therapeutics. We expect to be able to move from target identification through to IND filing in 12 to 18 months depending on specific gene targets. Our platform flexibility enables us to freely explore treatment options and select RNAi-based drug candidates with the greatest potential for clinical success.

siRNA Diagram

From target to clinic (in 18 months)

The MDRNA Drug Discovery Engine
In silico siRNA design
siRNA target identification and characterization
Delivery optimization
Lead candidate selection

Comprehensive analytical and biological testing
In vivo analysis (efficacy, disease models, PK/PD, toxicology and immune response)
Methods development and analytical support

IND submission support

Complementary strengths

MDRNA's approach to the development of RNAi-based therapeutics is founded on the natural precision of siRNA's to target and control disease-causing protein expression. Our scientific expertise allows us to create the optimal drug candidates that achieve high potency with lower risk of immunological response and less off-target activity.

Delivering the right therapy to the right place, at the right time The challenge of delivering effective RNAi-based therapeutics requires broad expertise and a deep understanding of biology (molecular and cellular); chemistry (lipid and peptide); routes of drug administration (systemic, local and topical); and the interdependency of these three areas for delivering siRNAs to organs, individual cells, and specific gene targets.

Challenges specific to designing siRNA structures include:

Nuclease degradation prior to cell entry
Cellular uptake of siRNA and intracellular degradation
The specificity of siRNA interaction with cellular machinery
Off-target effects
Cytokine induction

Challenges specific to siRNA delivery include:

Safe and effective systemic administration for transport via blood to target site
Safe and effective local administration via direct application to target site
Cellular uptake and intracellular distribution for effective gene silencing

To address these challenges, MDRNA is discovering and advancing to clinic drug candidates comprised of various siRNA constructs (Dicer substrates and meroduplexes) within novel liposome and peptide-based delivery systems to ensure optimal siRNA delivery, activation of the RNAi process, and down regulation of protein expression.


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