Applying RNAi to fight disease
At Dicerna, we use RNA interference, or RNAi, to create medicines that silence or turn off the genes that cause or contribute to disease. Using our GalXC™ and GalXC-Plus™ technologies, we aim to develop safe, effective, specific and convenient RNAi therapies – in-house and with our partners – for both rare and common diseases.
Our GalXC™ pipeline and platform
Our proprietary GalXC platform has the potential to open a world of possibilities for creating targeted RNAi therapies. Our GalXC platform is comprised of our liver-targeted GalXC technology and our GalXC-Plus technology for tissues outside the liver. Each utilizes a set of proprietary double-stranded RNA structures capable of inducing RNAi and associated chemical modifications and additions to these structures to enhance their pharmaceutical properties. Our RNAi-inducing RNA structures consist of two strands of nucleic acid. One of these strands, called the guide, or antisense, strand, is complementary to the mRNA sequence of the gene we are seeking to inhibit. The other strand, called the passenger, or sense, strand, includes sequences complementary to the guide strand. Together, the guide and passenger strands form a short double-stranded helix that is readily recognized and incorporated into the natural cellular RNAi process. In the case of our GalXC and GalXC-Plus technologies, additional sequences may be added to the passenger strand, including a four-base sequence, known as a tetraloop, which is designed to enhance stability and reduce immunostimulatory activity, to serve as an ideal attachment point for various additional functionalities that can facilitate delivery to a diverse range of tissues, including liver and non-liver tissues.
Our commitment to patients
We believe all patients deserve safe and effective medicines that can improve the quality and duration of their lives. Our goal is to understand and ease the unique disease-related burdens of patients and their caregivers and create new medicines with the potential to restore health by silencing the genes that cause or contribute to disease.
