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Alnylam Joins GSK In Donating Intellectual Property To Patent Pool For Neglected Tropical Diseases

September 12, 2017

GlaxoSmithKline (GSK) and Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) announced that Alnylam will contribute more than 1500 issued or pending patents on its RNA interference (RNAi) technology patent estate to the patent pool established by GSK earlier this year.

Alnylam is the first company to add its patents to the approximately 800 patent filings GSK provided to populate the pool in March. The company's RNAi platform provides an innovative approach to drug discovery and development through "gene silencing," a technology that targets the cause of diseases by potently silencing specific messenger RNAs (mRNAs), thereby preventing disease-causing proteins from being made.

"We are delighted that Alnylam will join GSK in this important programme by adding their unique RNAi technology to the patent pool," said Andrew Witty, Chief Executive Officer of GSK. "The key objective of the pool is to make it easier for researchers across the world to access intellectual property that may be useful in the search for new medicines to treat neglected tropical diseases. The more companies, academic institutions and foundations that join the pool, the more effective it will be. Alnylam's announcement today is therefore a welcome and significant step forward."

The patent pool was formed to aid in the discovery and development of new medicines for the treatment of 16 neglected tropical diseases (NTD), as defined by the U.S. Food and Drug Administration, in the world's Least Developed Countries. By adopting a more flexible approach to intellectual property, the patent pool will facilitate access to compounds and technologies for organisations that want to conduct research on treatments for these neglected diseases.

"We are committed to the innovation of medicines for patients, so we cannot ignore the potential of our technology to make a difference in the discovery of important new medicines for neglected diseases that afflict millions of people each year," said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. "We are very proud to be joining GSK in this unique and bold vision of social responsibility for some of the world's poorest nations."

Through today's contribution, Alnylam is providing RNAi intellectual property, technology and know-how on a royalty-free, non-profit basis in the Least Developed Countries via licensing agreements with qualified third parties. Such organisations will be engaged in research efforts focused on discovery of new medicines for NTD and their distribution to Least Developed Countries.

In the near term, Alnylam RNAi technology is expected to help validate novel drug targets for the discovery and development of treatments for the targeted NTD in least developed countries. For example, the technology has already helped to identify new targets for malaria treatments. , , In the future, RNAi therapeutics may themselves be developed and used directly in the treatment of more neglected tropical diseases.

About the patent pool

The diseases targeted by the pool are the 16 diseases identified by the FDA for its own Neglected Tropical Diseases initiative. These are tuberculosis, malaria, blinding trachoma, buruli ulcer, cholera, dengue/dengue haemorrhagic fever, racunculiasis, fascioliasis, human African trypanosomiasis, leishmaniasis, leprosy, lymphatic filariasis, onchocerciasis, schistosomiasis, soil transmitted helminthiasis and yaws. The geographic focus of the pool will be the world's Least Developed Countries as identified by the United Nations and includes much of western and central Africa as well as several countries in Southeast Asia.

About RNA Interference (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 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 cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. RNAi therapeutics target the cause of diseases by potently silencing specific messenger RNAs (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.

Alnylam Pharmaceuticals