Dicerna GalXC - Proprietary Technology
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How RNAi Therapies work and Their Advantages
The DNA within each of us is essentially an “instruction manual” for building proteins that greatly affect our health. Our messenger RNAs (mRNAs) carry these instructions from the DNA to sites in cells where proteins are made. During this biologic process, small interfering RNA (siRNA) molecules induce the destruction of the mRNAs of targeted genes.
Because scientists can easily create synthetic siRNAs that match almost any mRNA, this RNAi-based approach to drug development can silence virtually any gene and has distinct advantages over more traditional therapeutic approaches, including:
- RNAi has an impact earlier in the disease-causing process. RNAi can be used to target any gene because it targets the mRNA that encodes the disease-driving protein. While some conventional therapies block the activity of proteins responsible for disease, RNAi therapies prevent them from being created.
- RNAi is highly specific for the targeted gene because it uses a gene sequence match with the targeted gene and relies on naturally occurring cellular enzymes to mediate gene silencing. RNAi therapies are thought to be a safer option than permanently editing DNA, which could have unknown, unwanted and irreversible consequences. Conversely, RNAi therapies are reversible. RNAi therapies have the potential for fewer side effects compared to small molecules and antibodies.
- RNAi therapies also have the potential to reduce the treatment burden for patients. Their long duration of effect means they can be administered through infrequent injections under the skin.
Our scientists are exploring the use of GalXC technology beyond the liver in other organ systems, including neural tissues, to address a broad range of other diseases.
Data from clinical and preclinical studies suggest that GalXC may offer several benefits, including:
- Potentially stronger potency than comparable RNAi therapies
- Highly specific binding to disease-causing targets
- Long duration of action
- An infrequent, subcutaneous dosing regimen, which can minimize treatment burden
- A high therapeutic index, which indicates broad applicability
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