DRNA Dicerna Pharmaceuticals

Dicerna Announces Appointment of Ralf Rosskamp, M.D., as Chief Medical Officer

Dicerna Pharmaceuticals, Inc. (Nasdaq: DRNA), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced the appointment of industry veteran Ralf Rosskamp, M.D., as chief medical officer, effective June 15, 2017. Dr. Rosskamp brings to Dicerna more than 20 years of research and development (R&D) experience spanning the entire drug development cycle, from pre-clinical through product commercialization. He will be responsible for leading the Company’s clinical development, medical affairs and regulatory functions.

“Dr. Rosskamp is an accomplished biopharmaceutical executive with extensive global drug development and product launch experience across numerous therapeutic areas, including large population diseases such as cardiovascular disease, and, more recently, rare and orphan diseases,” said Douglas M. Fambrough, Ph.D., president and chief executive officer of Dicerna. “His in-depth experience, combined with his proven success in working with global regulatory agencies to obtain multiple drug approvals, including most recently the FDA approval of Natpara® for the treatment of a rare endocrine disorder called hypoparathyroidism, will be a huge asset to Dicerna. Dr. Rosskamp will play an integral role as we seek to advance our lead product, DCR-PHXC, into the clinic in early 2018, and continue to advance our broad pipeline of other promising GalXC™-based drug candidates.”

Dr. Rosskamp joins Dicerna from Summit Therapeutics where he served as chief medical officer. In this capacity, he was responsible for directing and overseeing all clinical development and medical activities for Summit’s clinical candidates in development for the rare genetic disease Duchenne muscular dystrophy and the infectious disease caused by Clostridium difficile bacteria. Prior to Summit, Dr. Rosskamp held executive-level positions in clinical research and drug development at NPS Pharmaceuticals (acquired by Shire), Ikaria, Kos Pharmaceuticals (acquired by Abbott Laboratories), Aventis Pharmaceuticals (formerly Hoechst Marrion Roussel), and Hoechst AG. Dr. Rosskamp has been responsible for numerous investigational new drug applications, the design and execution of clinical development programs, and new drug applications across multiple therapeutic areas including diabetes, cardiovascular, respiratory, and orphan drugs. In addition to Natpara®, approved products for which Dr. Rosskamp was involved include Amaryl®, Lantus®, Apidra® and Simcor®.

Dr. Rosskamp is a member of the Board of Directors of Bioblast Pharma in Tel Aviv, Israel. He has authored more than 20 peer-reviewed publications and published more than 50 scientific abstracts. Dr. Rosskamp is a pediatric endocrinologist with more than 15 years of experience in clinical medicine. He received his M.D. from the University of Bonn, Germany.

“I am delighted to join Dicerna at this pivotal time, and to have the opportunity to lead the clinical development of the Company’s pipeline of novel RNAi compounds,” said Dr. Rosskamp. “Dicerna has developed a differentiated technology platform that has generated a strong and growing pipeline of pre-clinical candidates capable of silencing genes in the liver across a spectrum of diseases, opening up a range of therapeutic opportunities for the Company. I look forward to working closely with the Dicerna team to initiate clinical studies early next year for its lead program, DCR-PHXC, which is being developed for patients with primary hyperoxaluria type 1, a devastating and rare genetic disease with limited treatment options.”

About Dicerna Pharmaceuticals, Inc.

Dicerna Pharmaceuticals, Inc. is a biopharmaceutical company focused on the discovery and development of innovative ribonucleic acid interference (RNAi)-based therapeutics for diseases involving the liver, including rare diseases, chronic liver diseases, cardiovascular diseases, and viral infectious diseases. The Company 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. The Company intends to discover, develop, and commercialize novel therapeutics either on its own or in collaboration with pharmaceutical partners. For more information, please visit www.dicerna.com.

Cautionary Note on Forward-Looking Statements

This press release includes forward-looking statements, including, for example, our anticipated timeline and plans for development. 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 our preclinical and clinical research and other risks identified under the heading "Risk Factors" included in our most recent Form 10-K 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.

EN
08/06/2017

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