long-term supply of KYNAMRO drug substance. Genzyme manufactures the finished drug product for
KYNAMRO and is offering KYNAMRO in the United States and other specific countries in pre-filled syringes.
Genzyme is producing the pre-filled syringes using one of its own manufacturing facilities.
Patents and Proprietary Rights
Our success depends, in part, on our ability to obtain patent protection for our products in the United States
and other countries. As of February 10, 2015, we owned or exclusively licensed more than 1,300 issued patents
worldwide. We focus our resources on patents and new patent applications that drive value for our company.
We own or control patents that provide exclusivity for products in our pipeline and patents that provide
exclusivity for our core technology in the field of antisense more generally. Our core technology patents include
claims to chemically-modified nucleosides and oligonucleotides as well as antisense drug designs utilizing these
chemically-modified nucleosides. These core claims are each independent of specific therapeutic target, nucleic
acid sequence, or clinical indication. We also own a large number of patents claiming specific antisense
compounds having nucleic acid sequences complementary to therapeutic target nucleic acids, independent of the
particular chemical modifications incorporated into the antisense compound. Most importantly, we seek and
obtain issued patent claims to specifically protect each of our drugs. For example, we file and seek to obtain
claims covering each drug’s nucleic acid sequence and precise drug design. In sum, we maintain our competitive
advantage in the field of antisense technology by protecting our core platform technology, which applies to most
of our drugs, and by creating multiple layers of patent protection for each of our specific drugs in development.
Type of Patent Claim
Breadth
Description
BroadlyApplicable
ChemicallyModifiedNucleosides andOligonucleotides
Antisense Drug DesignMotifs
TherapeuticMethods
Antisense Sequence Drug Composition
Target and sequence independent
Sequence independent
Chemistry independent
Specific claim to drug candidates
Specific
ChemicallyModified Nucleosides and Oligonucleotides
The most broadly-applicable of our patents are those that claimmodified nucleosides and oligonucleotides
comprising the modified nucleosides that we incorporate into our antisense drugs to increase their therapeutic
efficacy. Nucleosides and chemically-modified nucleosides are the basic building blocks of our antisense drugs,
therefore claims that cover any oligonucleotide incorporating one of our proprietary modified nucleosides can
apply to a wide array of antisense mechanisms of action as well as several therapeutic targets. Of particular note
are our patents covering our proprietary 2’-O-(2-methoxy) ethyl modified nucleosides, incorporated into nearly
all of our development compounds, as well as our generation 2.5 compounds, the constrained-ethyl nucleosides,
or cEt, nucleosides. In June 2011, Santaris PharmaA/S opposed our granted patent in Europe drawn to cEt
containing nucleotides and oligonucleotides and we intend to vigorously defend our patent in these proceedings.
Further information about litigation with Santaris can be found in Item 3,
Legal Proceedings.
The following are some of our patents in this category:
Jurisdiction
Patent No.
Title
Expiration
Description of Claims
United States
7,101,993
OLIGONUCLEOTIDES
CONTAINING 2’O-MODIFIED
PURINES
2023
Covers certainMOE nucleosides and
oligonucleotides containing said
nucleotides.
United States
7,399,845
6-MODIFIEDBICYCLIC
NUCLEICACIDANALOGS
2027
Covers our cEt nucleosides and
oligonucleotides containing these
nucleoside analogs.
United States
7,741,457
6-MODIFIEDBICYCLIC
NUCLEICACIDANALOGS
2027
Covers our cEt nucleosides and
oligonucleotides containing these
nucleoside analogs.
United States
8,022,193
6-MODIFIEDBICYCLIC
NUCLEICACIDANALOGS
2027
Covers our cEt nucleosides and
oligonucleotides containing these
nucleoside analogs.
United States
7,569,686
COMPOUNDSANDMETHODS
FOR SYNTHESIS OF BICYCLIC
NUCLEICACIDANALOGS
2027
Covers methods of synthesizing our cEt
nucleosides.
Europe
EP1984381
6-MODIFIEDBICYCLIC
NUCLEICACIDANALOGS
2027
Covers our cEt nucleosides and
oligonucleotides containing these
nucleoside analogs.
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