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Alnylam to Host Second Annual Summer "RNAi Roundtable" Webcast Series
[July 06, 2015]

Alnylam to Host Second Annual Summer "RNAi Roundtable" Webcast Series


Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, today announced that it plans to host and webcast its annual summer series of online "RNAi Roundtables" this July, August, and September. The 2015 series of events will consist of presentations by a mix of Alnylam scientists, clinical collaborators, and patient advocates, who will review recent progress in many of the company's development-stage pipeline programs and discuss the related disease areas. Each event will be webcast live on the Investors section of the company's website, www.alnylam.com. An audio replay of the roundtables will be posted on the Alnylam website approximately three hours after each event.

The 2015 RNAi Roundtable topics scheduled to date include the following investigational RNAi therapeutics:

ALN-CC5 for the treatment of Complement-Mediated Diseases
Friday, July 17, 8:00 - 9:00 a.m. ET

  • Pushkal Garg, M.D., Senior Vice President, Clinical Development
  • Moderator: Akshay Vaishnaw, M.D., Ph.D., Executive Vice President of R&D, Chief Medical Officer
  • Guest Speaker: Regis Peffault de Latour, M.D., Ph.D., Professor, Hematology and Transplantation, Saint-Louis Hospital, Paris

ALN-AT3 for the treatment of Hemophilia and Rare Bleeding Disorders
Wednesday, July 22, 9:00 - 10:30 a.m. ET

  • Benny Sorensen, M.D., Ph.D., Senior Director, Clinical Development
  • Moderator: John Maraganore, Ph.D., Chief Executive Officer
  • Guest Speaker: Margaret Ragni, M.D., MPH, Professor of Medicine, Division Hematology/Oncology, University of Pittsburgh, and Director, Hemophilia Center of Western Pennsylvania
  • Guest Speaker: Mark W. Skinner, J.D., Past President, World Federation of Hemophilia/National Hemophilia Foundation

ALN-HBV for the treatment of Hepatitis B Virus (HBV) Infection
Tuesday, July 28, 4:00 - 5:00 p.m. ET

  • Laura Sepp-Lorenzino, Ph.D., Vice President, Entrepreneur-in-Residence
  • Moderator: John Maraganore, Ph.D., Chief Executive Officer
  • Guest Speaker: Edward Gane, MBChB, M.D., FRACP, MNZM, Professor of Medicine, University of Auckland (NZ), and Chief Hepatologist, Transplant Physician, Deputy Director of New Zealand Liver Transplant Unit, Auckland City Hospital

ALN-AAT for the treatment of AAT Deficiency-associated liver disease
Friday, August 14, 2:00 - 3:00 p.m. ET

  • Alfica Sehgal, Ph.D., Principal Scientist, Research
  • Moderator: Akshay Vaishnaw, M.D., Ph.D., Executive Vice President of R&D, Chief Medical Officer
  • Guest Speaker: Jeffrey Teckman, M.D., Professor, Department of Pediatrics, St. Louis University School of Medicine

Patisiran and Revusiran for the treatment of Transthyretin (TTR)-Mediated Amyloidosis
Thursday, August 20, 9:00 - 10:30 a.m. ET

  • Eric Green, Vice President, General Manager, TTR Program
  • ared Gollob, M.D., Vice President, Clinical Research
  • Moderator: Barry Greene, President and Chief Operating Officer
  • Guest Speaker: Philip Hawkins, Ph.D., FRCP, FRCPath, FMedSci, Head, National Amyloidosis Centre, and Head, Periodic Fever Syndrome Service/Honorary consultant physician



Alnylam plans to schedule additional RNAi Roundtables covering other pipeline programs to take place in the weeks following the initial webcasts, including the following investigational RNAi therapeutics: ALN-AS1 for the treatment of acute hepatic porphyrias, ALN-PCSsc for the treatment of hypercholesterolemia, and ALN-GO1 for the treatment of Primary Hyperoxaluria Type 1. Speakers, dates, and times for these events will be posted on the Capella section of the company's website, www.alnylam.com/capella.

About RNAi
RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today which was awarded the 2006 Nobel (News - Alert) Prize for Physiology or Medicine. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.


About Alnylam Pharmaceuticals
Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is leading the translation of RNAi as a new class of innovative medicines. Alnylam's pipeline of investigational RNAi therapeutics is focused in 3 Strategic Therapeutic Areas (STArs): Genetic Medicines, with a broad pipeline of RNAi therapeutics for the treatment of rare diseases; Cardio-Metabolic Disease, with a pipeline of RNAi therapeutics toward genetically validated, liver-expressed disease targets for unmet needs in cardiovascular and metabolic diseases; and Hepatic Infectious Disease, with a pipeline of RNAi therapeutics that address the major global health challenges of hepatic infectious diseases. In early 2015, Alnylam launched its "Alnylam 2020" guidance for the advancement and commercialization of RNAi therapeutics as a whole new class of innovative medicines. Specifically, by the end of 2020, Alnylam expects to achieve a company profile with 3 marketed products, 10 RNAi therapeutic clinical programs - including 4 in late stages of development - across its 3 STArs. The company's demonstrated commitment to RNAi therapeutics has enabled it to form major alliances with leading companies including Merck, Medtronic, Novartis, Biogen, Roche, Takeda, Kyowa Hakko Kirin, Cubist, GlaxoSmithKline, Ascletis, Monsanto, The Medicines Company, and Genzyme, a Sanofi company. In addition, Alnylam holds an equity position in Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 200 peer-reviewed papers, including many in the world's top scientific journals such as Nature, Nature Medicine, Nature Biotechnology, Cell, New England Journal of Medicine, and The Lancet. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information about Alnylam's pipeline of investigational RNAi therapeutics, please visit www.alnylam.com.

Alnylam Forward Looking Statements
Various statements in this release concerning Alnylam's future expectations, plans and prospects, including without limitation, Alnylam's views with respect to the potential for RNAi therapeutics, expectations regarding its STAr pipeline growth strategy, and its plans regarding commercialization of RNAi therapeutics, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Alnylam's ability to discover and develop novel drug candidates and delivery approaches, successfully demonstrate the efficacy and safety of its drug candidates, the pre-clinical and clinical results for its product candidates, which may not be replicated or continue to occur in other subjects or in additional studies or otherwise support further development of product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials, obtaining, maintaining and protecting intellectual property, Alnylam's ability to enforce its patents against infringers and defend its patent portfolio against challenges from third parties, obtaining regulatory approval for products, competition from others using technology similar to Alnylam's and others developing products for similar uses, Alnylam's ability to manage operating expenses, Alnylam's ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, Alnylam's dependence on third parties for development, manufacture, marketing, sales and distribution of products, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the "Risk Factors" filed with Alnylam's most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC (News - Alert)) and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation to update any forward-looking statements.


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