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. In December 2017, we initiated human dosing in normal health volunteers (NHVs) in a Phase 1 clinical trial of DCR-PHXC, called PHYOX. We have completed dosing of NHVs in the first phase of the trial. We plan to dose the first primary hyperoxaluria patient with DCR-PHXC in the next phase of the PHYOX trial in second quarter of 2018 and expect to have clinical proof-of-concept (POC) data in the second half of 2018. In addition to the active investigational new drug (IND) application with the U.S. FDA, we have active clinical trial applications (CTAs) in the United Kingdom, France and Germany, having received the appropriate regulatory and ethical approvals for the trial in these countries. A CTA has been submitted and is pending approval in the Netherlands. Additionally, we expect to initiate a multi-dose Phase 2/3 study in the first quarter of 2019, pending positive POC data and regulatory approvals.
We are committed to delivering transformative therapies based on our GalXCTM RNAi platform to patients with rare inherited diseases involving the liver and for other therapeutic areas involving the liver such as viral infectious diseases, chronic liver diseases, and cardiovascular 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.
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:||58|