Research & Development

A multi-faceted approach to a complex problem

While the RNAi pathway is well understood, we believe its therapeutic application requires multiple broad, flexible and innovative approaches. We have built a portfolio of capabilities in molecular biology, cellular biology, microbiology, peptide and alkylated amino acid chemistry, pharmacology, bioinformatics and preclinical and clinical development.

These capabilities, the in-licensing and assignment of key RNAi-related intellectual property (IP), and the strategic acquisition of delivery capability have enabled us to swiftly become a leading RNAi-based therapeutics company.

Currently, we are developing RNAi-based therapies in Familial Adenomatous Polyposis (FAP) and oncology utilizing our tkRNAi and tauRNAi platforms, respectively. We are focused on UsiRNA and shRNA constructs for activating the RNAi pathway. We have also built in-depth expertise in liposome, peptide-based and bacterial delivery systems.

Fueling RNAi Discovery and Development

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

Research Team

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

Our cutting-edge science and therapeutic programs have allowed us to attract experts in molecular and cellular biology, microbiology, peptide and alkylated amino acid chemistry, pharmacology, bioinformatics, clinical development, regulatory affairs and quality control, as well as an experienced pharmaceutical management team. By bringing the industry’s best and brightest to our team, Marina Biotech is creating an environment that fosters creative development and innovative research in an atmosphere of teamwork and dedication.

Combining Speed and Efficiency

Marina Biotech's approach to the development of RNAi-based therapeutics is founded on the natural precision of RNAi-based mechanisms to target and control the expression of disease-causing proteins. Our scientific expertise and breadth of drug discovery platforms allows us to create optimal drug candidates that achieve high potency with lower risk of immunological response and off-target activity.

We have the ability to identify, synthesize and formulate potent siRNA constructs for in vitro and in vivo testing as well as complete preclinical evaluation of RNAi-based compounds for human therapeutics. In addition, the team is capable of bringing a novel RNAi-based therapeutic through to successful filing of a US FDA IND and into early clinical trials. The breadth of our capability allows us to move from target identification through to IND filing in 12 to 18 months depending on specific gene targets and mode of administration. Our multiple drug discovery engines enable us to explore treatment options and select RNAi-based drug candidates with the greatest potential for clinical success.

Marina Biotech's RNAi Drug Discovery Capability

  • In silico siRNA design
  • siRNA target identification and characterization
  • Delivery system optimization
  • Lead candidate selection
  • Comprehensive analytical and biological evaluations
  • In vivo studies (efficacy, disease models, PK/PD, toxicology and immune response)
  • Methods development and analytical support
  • IND-enabling pharmacology and toxicology studies
  • Chemistry, Manufacturing & Control management
  • IND submission
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