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Press Release

Globally Recognized Nephrologist Joins Dicerna™ to Support Continued Development of DCR-PHXC for the Treatment of Primary Hyperoxaluria

Jun 20, 2019

– Prof. Bernd Hoppe, M.D., Appointed Vice President, Global Medical Affairs at Dicerna –

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun. 20, 2019--

Dicerna™ Pharmaceuticals, Inc. (Nasdaq: DRNA) (the “Company” or “Dicerna”), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced the appointment of Prof. Bernd Hoppe, M.D., as vice president, global medical affairs, effective July 1, 2019. Dr. Hoppe is a professor of Pediatrics and was head of the Division of Pediatric Nephrology in the Department of Pediatrics at the University Hospital in Bonn, Germany.

“Dicerna is delighted to welcome one of the world’s leading pediatric nephrologists to our team,” said Douglas M. Fambrough, Ph.D., president and chief executive officer of Dicerna. “We are privileged that Dr. Hoppe has chosen to apply his deep clinical and scientific knowledge of primary hyperoxaluria to the development of DCR-PHXC, our lead product candidate and the only RNAi-based therapy designed to treat all genetic types of primary hyperoxaluria.”

Dr. Hoppe noted, “As a researcher and physician, I have focused for more than three decades on understanding all types of primary hyperoxaluria and providing the best care possible for people diagnosed with this rare genetic disease. This same goal motivates me to join Dicerna and contribute to the development of DCR-PHXC. I am encouraged by the science of targeting the LDHA gene and impressed by the initial clinical data generated for DCR-PHXC in patients with PH. I look forward to advancing DCR-PHXC in partnership with the Dicerna team.”

Prior to joining the University Hospital in Bonn, Dr. Hoppe held research and teaching positions in pediatric nephrology and pediatric surgery at University Children’s Hospital in Cologne; Hannover (Germany) Medical School; University Children’s Hospital in Zurich, Switzerland; Children’s Hospital St. Marien in Landshut-Munich, Germany; and Children’s Hospital St. Augustin at the University of Bonn Teaching Hospital. Dr. Hoppe conducted basic research on oxalate metabolism at Children’s Memorial Hospital Division of Nephrology at Northwestern University in Chicago, Ill.

A board member of the Oxalosis & Hyperoxaluria Foundation (OHF), Dr. Hoppe’s professional society memberships include the German Society of Pediatrics, the German and European Societies of Pediatric Nephrology, the International Pediatric Nephrology Association and the American Society of Nephrology. He also serves on the medical and scientific advisory board of the non-profit organization Primäre Hyperoxalurie Selbsthilfe (Primary Hyperoxaluria Self-Support Germany). Dr. Hoppe has served as a reviewer for the German Research Foundation as well as for several medical journals including Kidney International, Nephrology Dialysis and Transplantation,American Journal of Kidney Diseases and Clinical Nephrology. At the upcoming OHF International Hyperoxaluria Workshop in Boston, Mass., on June 22, Dr. Hoppe will present updated findings from the ongoing PHYOX™1 Phase 1 clinical trial, in which DCR-PHXC reduced 24-hour urinary oxalate levels in adult and adolescent study participants with PH type 1 (PH1) and PH type 2 (PH2).

About Dicerna™ Pharmaceuticals, Inc.

Dicerna™ Pharmaceuticals, Inc., is a biopharmaceutical company focused on the discovery and development of innovative, subcutaneously delivered RNAi-based therapeutics for the treatment of diseases involving the liver, including rare diseases, chronic liver diseases, cardiovascular diseases and viral infectious diseases. Dicerna is leveraging its proprietary GalXC™ RNAi technology platform to build a broad pipeline in these core therapeutic areas, focusing on target genes where connections between target gene and diseases are well understood and documented. Dicerna intends to discover, develop and commercialize novel therapeutics either on its own or in collaboration with pharmaceutical partners. Dicerna has strategic collaborations with Eli Lilly and Company, Alexion Pharmaceuticals, Inc. and Boehringer Ingelheim International GmbH. For more information, please visit www.dicerna.com.

Cautionary Note on Forward-Looking Statements

This press release includes forward-looking statements. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Applicable risks and uncertainties include risks relating to Dicerna’s clinical and preclinical research and other risks identified under the heading "Risk Factors" included in the Company’s most recent Form 10-Q filing and in other future filings with the SEC. The forward-looking statements contained in this press release reflect Dicerna's current views with respect to future events, and Dicerna does not undertake and specifically disclaims any obligation to update any forward-looking statements.

Dicerna™, GalXC™ and PHYOX™ are trademarks of Dicerna Pharmaceuticals, Inc.

Source: Dicerna™ Pharmaceuticals, Inc.

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