Alnylam to Host “RNAi Roundtable” Webcast Series

  Alnylam to Host “RNAi Roundtable” Webcast Series

Business Wire

CAMBRIDGE, Mass. -- July 7, 2014

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics
company, today announced that it plans to host and webcast a series of online
“RNAi Roundtables” during July and August. Alnylam scientists and key clinical
collaborators will review recent progress from several of the “Alnylam 5x15”
programs and discuss the related disease areas. Each event will be webcast
live on the Investors section of the company’s website, An
audio replay of the roundtables will be posted on the Alnylam website
approximately three hours after each event.

The RNAi Roundtable topics scheduled to date include:

Patisiran and ALN-TTRsc for the treatment of Transthyretin-Mediated
Amyloidosis (ATTR)

Tuesday, July 15: 12:30 – 2:00 p.m. ET

  *Jared Gollob, M.D., Vice President, Clinical Research
  *Moderator: John Maraganore, Ph.D., Chief Executive Officer
  *Guest Speaker: Philip N. Hawkins, M.B., B.S., Ph.D., FRCP, Professor of
    Medicine, National Amyloidosis Centre, University College London Medical

Advances in Delivery of RNAi Therapeutics with Enhanced Stabilization
Chemistry (ESC)-GalNAc-siRNA Conjugates

Tuesday, July 22: 11:00 a.m. – 12:00 p.m. ET

  *Rachel Meyers, Ph.D., Vice President, Research and RNAi Lead Development
  *Moderator: Laurence Reid, Ph.D., Senior Vice President and Chief Business

ALN-HBV for the treatment of Hepatitis B Virus (HBV) Infection

Tuesday, July 29: 9:30 – 10:30 a.m. ET

  *Laura Sepp-Lorenzino, Ph.D., Vice President, Entrepreneur-in-Residence
  *Moderator: Laurence Reid, Ph.D., Senior Vice President and Chief Business
  *Guest Speaker: Graham Foster, Ph.D., FRCP, Professor of Hepatology at
    Queen Mary University of London

ALN-AT3 for the treatment of Hemophilia and Rare Bleeding Disorders

Thursday, August 7: 9:30 – 10:30 a.m. ET

  *Akin Akinc, Ph.D., Director, Research
  *Moderator: John Maraganore, Ph.D., Chief Executive Officer
  *Guest Speaker: Flora Peyvandi, M.D., Ph.D., Head of the Department of
    Internal Medicine and Angelo Bianchi Bonomi Hemophilia and Thrombosis
    Centre, IRCCS Maggiore Hospital, University of Milan.

ALN-CC5 for the treatment of Complement-Mediated Diseases

Wednesday, August 13: 9:30 – 10:30 a.m. ET

  *Benny Sorensen, M.D., Ph.D., Medical Director, Clinical Development
  *Moderator: Barry Greene, President and Chief Operating Officer
  *Guest Speaker: Anita Hill, MBChB (Hons), MRCP, FRCPath, Ph.D., Consultant
    Haematologist for Leeds Teaching Hospitals NHS Trust, UK, and Honorary
    Senior Lecturer at the University of Leeds

Alnylam plans to schedule additional RNAi Roundtables covering other pipeline
programs to take place in the coming weeks, including ALN-AS1 for the
treatment of hepatic porphyrias; ALN-PCSsc for the treatment of
hypercholesterolemia; and ALN-AAT for the treatment of AAT
deficiency-associated liver disease. The topics, dates, and times of these
events will be posted on the Capella section of the company’s website,

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 with a core focus on RNAi therapeutics as
genetic medicines, including programs as part of the company’s “Alnylam
5x15^TM” product strategy. Alnylam’s genetic medicine programs are RNAi
therapeutics directed toward genetically defined targets for the treatment of
serious, life-threatening diseases with limited treatment options for patients
and their caregivers. These include: patisiran (ALN-TTR02), an intravenously
delivered RNAi therapeutic targeting transthyretin (TTR) for the treatment of
TTR-mediated amyloidosis (ATTR) in patients with familial amyloidotic
polyneuropathy (FAP); ALN-TTRsc, a subcutaneously delivered RNAi therapeutic
targeting TTR for the treatment of ATTR in patients with TTR cardiac
amyloidosis, including familial amyloidotic cardiomyopathy (FAC) and senile
systemic amyloidosis (SSA); ALN-AT3, an RNAi therapeutic targeting
antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders
(RBD); ALN-CC5, an RNAi therapeutic targeting complement component C5 for the
treatment of complement-mediated diseases; ALN-AS1, an RNAi therapeutic
targeting aminolevulinic acid synthase-1 (ALAS-1) for the treatment of hepatic
porphyrias including acute intermittent porphyria (AIP); ALN-PCS, an RNAi
therapeutic targeting PCSK9 for the treatment of hypercholesterolemia;
ALN-AAT, an RNAi therapeutic targeting alpha-1 antitrypsin (AAT) for the
treatment of AAT deficiency-associated liver disease; ALN-TMP, an RNAi
therapeutic targeting TMPRSS6 for the treatment of beta-thalassemia and
iron-overload disorders; ALN-ANG, an RNAi therapeutic targeting
angiopoietin-like 3 (ANGPTL3) for the treatment of genetic forms of mixed
hyperlipidemia and severe hypertriglyceridemia; ALN-AC3, an RNAi therapeutic
targeting apolipoprotein C-III (apoCIII) for the treatment of
hypertriglyceridemia; and other programs yet to be disclosed. As part of its
“Alnylam 5x15” strategy, as updated in early 2014, the company expects to have
six to seven genetic medicine product candidates in clinical development -
including at least two programs in Phase 3 and five to six programs with human
proof of concept - by the end of 2015. Alnylam is also developing ALN-HBV, an
RNAi therapeutic targeting the hepatitis B virus (HBV) genome for the
treatment of HBV infection. The company’s demonstrated commitment to RNAi
therapeutics has enabled it to form major alliances with leading companies
including Merck, Medtronic, Novartis, Biogen Idec, Roche, Takeda, Kyowa Hakko
Kirin, Cubist, GlaxoSmithKline, Ascletis, Monsanto, The Medicines Company, and
Genzyme, a Sanofi company. In March 2014, Alnylam acquired Sirna Therapeutics,
a wholly owned subsidiary of Merck. 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, the New England Journal of Medicine, and The Lancet. Founded in 2002,
Alnylam maintains headquarters in Cambridge, Massachusetts. For more
information, please visit


Alnylam Pharmaceuticals, Inc.
Cynthia Clayton, 617-551-8207
Vice President, Investor Relations and
Corporate Communications
Liz Bryan (Media), 202-955-6222 x2526
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