Alnylam Receives New Issued U.S. Patent Broadly Covering Small Interfering RNAs and Strengthens Leadership in Intellectual Property for RNAi Therapeutics - siRNA

Alnylam Receives New Issued U.S. Patent Broadly Covering Small Interfering RNAs and Strengthens Leadership in Intellectual Property for RNAi Therapeutics
July 18, 2006
CAMBRIDGE, Mass.--(BUSINESS WIRE)--July 18, 2006--
New Tuschl II '196 Patent Covers Methods of Making Small
Interfering RNAs With and Without Chemical Modifications
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that a new key fundamental patent from its exclusively held "Tuschl II" patent series was issued by the United States Patent and Trademark Office (USPTO). The newly issued U.S. patent (U.S. Patent No. 7,078,196 or " '196 patent") broadly covers methods of making small interfering RNAs (siRNAs), the molecules that mediate RNAi, and includes 79 claims covering chemical modifications needed to introduce "drug-like" properties in RNAi therapeutics. The issued patent is exclusively licensed to Alnylam for RNAi therapeutics on a worldwide basis through an agreement with Garching Innovation GmbH, the licensing agent for the Max Planck Society. The '196 patent further strengthens Alnylam's intellectual property (IP) leadership that includes another Tuschl II patent (U.S. Patent No. 7,056,704 or " '704 patent"), covering methods of making siRNAs to target any messenger RNA in a mammalian cell, and the recent successful outcome of the European Opposition Proceedings for the Kreutzer-Limmer I patent (EP 1144623 or " '623 patent"), where amended patent claims covering siRNAs with 15-21 base pairs were upheld.
"We are gratified that the USPTO has acknowledged the key inventions of Professor Thomas Tuschl performed at the Max Planck Institute in Gottingen," said Professor Peter Gruss, President of the Max Planck Society. "This critical patent for RNAi therapeutics is owned by the Max Planck Society and has been licensed exclusively to Alnylam for commercialization. We are impressed with the progress Alnylam is making in its effort to develop innovative medicines using one of our Institute's most important scientific discoveries."
The new '196 patent is part of the groundbreaking research published in 2001 in the journal Nature by Thomas Tuschl, Ph.D., a founder of Alnylam. In this seminal work performed at Max Planck, Professor Tuschl and his colleagues provided the first peer-reviewed published evidence that RNAi is mediated by small double-stranded RNAs with defined structural features and that synthetic siRNAs, with or without chemical modification, can be used to achieve RNAi in mammalian cells.
"This new patent extends what we believe is an unparalleled IP estate for RNAi therapeutics that includes over 150 patents issued in the world's largest pharmaceutical markets," said John Maraganore, Ph.D., President and Chief Executive Officer of Alnylam Pharmaceuticals. "The '196 patent, together with other Alnylam-held IP assets, strengthens our continued efforts to create value through partnerships and license agreements with major pharmaceutical companies, biotechnology companies, and research product suppliers. Indeed, the unmatched strength of our patent estate supports a track record of business execution with over 20 current partners and licensees that have provided in excess of $100 million in funding to date, and a source of committed and sustainable funding for the future. Strategically, this important funding source for Alnylam supports our efforts to bring RNAi therapeutics to the market in a capital-efficient manner."
The 79 claims for the '196 patent cover the preparation of double-stranded RNAs having key structural elements that are widely recognized as critical requirements for the therapeutic activity of siRNAs, including:
-- A double stranded region formed from two RNA strands of a
length of 19-25 nucleotides; and,
-- One or more 3'-overhangs at the ends of the double-stranded
molecule.
The claims cover siRNAs with or without chemical modifications that are used to introduce certain drug-like properties into siRNAs such as stability and delivery. The claims cover broad forms of such chemical modifications, including those of the 2' hydroxy position of the ribose backbone used to generate so-called "no-ribose" or "siNA" forms of RNAi therapeutics. These chemical modifications include the use of phosphorothioate backbone linkages or 2'------methyl, and/or 2'-fluoro chemistries, irrespective of the percent of nucleotides modified and the extent of such modifications.
The new issued claims for U.S. Patent No. 7,078,196 will be made accessible on the Alnylam website at: www.alnylam.com/patent-estate.