November 29, 2018
November 9, 2018
Dicerna is developing a pipeline of innovative RNAi therapies using our GalXCTM RNAi platform for the treatment of diseases involving the liver, including rare diseases, viral infectious diseases, chronic liver diseases and cardiovascular diseases.Learn More
Dr. Ralf Rosskamp, Dicerna’s chief medical officer, participated in podcast with RARECast and provided an overview of the challenges facing drug development in rare diseases, as well as an update on Dicerna’s development program in primary hyperoxaluria.
The GalXCTM RNAi Technology Platform
GalXCTM is a proprietary RNAi technology platform that advances the development of next-generation RNAi-based therapies that act by silencing disease-driving genes in the liver. GalXC molecules are designed for infrequent subcutaneous administration.
Unlocking the Potential of RNAi
RNA interference (RNAi) is a natural biological method for silencing or “turning off” specific genes known to cause or drive disease. In the RNAi process, small, carefully selected molecules of RNA inhibit the expression of these harmful genes by causing the targeted destruction of their messenger RNAs (mRNAs). RNAi has the potential to generate a revolution in medicine.
RNAi: Timeline of Technological and Therapeutic Advances
Andrew Fire and Craig Mello publish their discovery of RNAi in Nature.
Nature publishes the first study to show short-interfering RNA (siRNA) activity in vivo in animals.
Nature Medicine publishes the first therapeutic application of RNAi in animals.
Nature Biotechnology publishes the discovery of DsiRNAs, which are siRNAs that have enhanced potency and efficacy by acting as substrates for processing by the enzyme Dicer.
Nobel Prize in Physiology or Medicine is awarded to Fire and Mello for their discovery of RNAi.
Molecular Therapy publishes a novel approach to optimizing DsiRNAs.
Journal of Biomolecular Techniques publishes data showing that DsiRNA molecules produce more potent, longer-lasting gene silencing than shorter siRNA molecules.
Dicerna is issued a U.S. patent to broadly cover DsiRNA technology.
Dicerna is issued a U.S. patent to broadly cover Dicerna’s Extended Dicer Substrate™ (DsiRNA-EX) therapeutic RNAs.
Molecular Therapy publishes a paper demonstrating how the use of DsiRNA compounds inhibit tumor growth by silencing β-catenin, a key oncogene in hepatocellular carcinoma (HCC).
Dicerna initiates a Phase 1 study of DCR-MYC in patients with solid tumors and hematologic malignancies.
Dicerna presents preclinical data indicating the rapid and durable effect of DCR-PH1 in a mouse model of primary hyperoxaluria type 1 (PH1), at the 10th annual meeting of the Oligonucleotide Therapeutics Society in San Diego, Calif.
Dicerna announces successful delivery of DsiRNA-EX Conjugate molecules in animals using subcutaneous injection.
Dicerna introduces GalXC, a proprietary next-generation RNAi technology platform, to rapidly identify new therapeutic agents that silence disease-driving genes in the liver and to efficiently advance these therapies into the clinic.
The β-catenin/Wnt pathway is among the most functionally validated targets for hepatocellular carcinoma and colorectal cancers. Dicerna demonstrated that inhibition of activated Wnt signaling causing tumor regression and terminal differentiation in well-established colorectal tumors.
Dicerna presents new preclinical data for DCR-PHXC highlighting role of LDHA in treating primary hyperoxaluria.
Dicerna doses first human with lead GalXC investigational candidate DCR-PHXC in PHYOX Phase 1 clinical trial.
Molecular Therapy publishes a paper written by Dicerna researchers showing that inhibition of glycogen synthase II with RNAi prevents liver injury in mouse models of glycogen storage diseases.
Dicerna doses first primary hyperoxaluria patient with lead GalXC investigational candidate DCR-PHXC in PHYOX Phase 1 clinical trial.
Molecular Therapy publishes a research paper from Dicerna scientists showing that the specific inhibition of hepatic lactate dehydrogenase (LDH) reduces oxalate production in mouse models of primary hyperoxaluria.