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
-
Jared 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 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) 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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