THERAPEUTIC PLATFORM
Structural Modification
To further strengthen its position within the RNAi therapeutics space, Silence has engaged in pursuing valuable structural modifications for its proprietary siRNA sequences.
These modifications are designed to improve both the efficacy and safety of these sequences, while also offering an increased depth of patent protection. The company’s work in this area involves two distinct approaches to structural modification.
AtuRNAi
Silence has developed a proprietary siRNA containing only naturally-occurring RNA which has been chemically modified to result in a chemically stabilized molecule. This stabilized siRNA, known as AtuRNAi, offers a number of key advantages including:
- Stability against nuclease degradation, offering longer half-life, lower doses and less frequent administration
- Absence of toxic metabolites due to the inclusion of only naturally occurring building blocks
- Lower manufacturing costs
- Increased yield and more rapid synthesis due to improved process development
Zamore "Design Rules"
Silence has three exclusive licenses from the University of Massachusetts that provide access to issued and pending patents associated with the Zamore "Design Rules". Access to this technology offers Silence additional insight into methods for increasing siRNA potency and reducing off-target effects of its therapeutics.