Driven to improve lives.
Dicerna is working to improve the lives of people suffering from diseases involving the liver, including rare diseases, viral infectious diseases, chronic liver diseases, and cardiovascular diseases. We discover and develop innovative therapies to stop or turn off destructive disease processes by silencing the genes underlying these processes.
A message from Douglas Fambrough, Ph.D., president and chief executive officer of Dicerna
Our proprietary, next-generation technology, known as RNA interference or RNAi, uses the body’s natural biological pathways to silence genes in the liver with a high degree of selectivity and specificity. By targeting genes that contribute to serious diseases, we seek to address the underlying cause of illness and restore health.
Dicerna is advancing a growing pipeline of product candidates, with our DCR-PHXC lead program in clinical development for the treatment of a progressive and debilitating rare disease called primary hyperoxaluria, or PH. DCR-PHXC utilizes our GalXCTM RNAi technology, a proprietary platform that advances the development of next-generation RNAi-based therapies designed to silence disease-driving genes in the liver. In December 2017, we initiated human dosing in normal healthy volunteers (NHVs) in a Phase 1 clinical trial of DCR-PHXC, called PHYOX, and dosed the first patient with PH in May 2018. The next step in the development process for DCR-PHXC is a multi-dose Phase 2/3 registration trial, which the Company is on track to initiate in the first quarter of 2019.
Our second program in development is DCR-HBVS, an investigational drug for the treatment of chronic hepatitis B virus (HBV) infection in adults. We dosed the first human volunteer in our Phase 1 clinical trial of DCR-HBVS, known as DCR-HBVS-101, in Q1 2019. We anticipate human proof-of-concept data from the Phase 1 trial in the second half of 2019.
We are committed to delivering transformative therapies based on our GalXC platform to patients with rare inherited diseases involving the liver and for other therapeutic areas involving the liver, including rare diseases, chronic liver diseases, cardiovascular diseases, and viral infectious diseases. We have qualified dozens of disease-associated genes in clinical indications where we believe an RNAi-based inhibitor may provide substantial benefit to patients, providing expansive therapeutic opportunities.
For more information about our other pipeline programs, please view our Pipeline chart.
Our people are our strength.
Dicerna brings together talented experts in biology, chemistry, clinical science and medicine. With decades of scientific and technical experience focused on RNAi technology, our team has the knowledge and experience needed to discover, develop and commercialize safe and effective therapies for patients with serious unmet medical needs.
Our purpose is clear: delivering life-changing therapies as efficiently as possible to meet the urgent needs of people living with debilitating genetic diseases.
|Listed on Nasdaq:||2014|
|Number of Employees:||90|