Yale and Innovir Laboratories Granted Broader Patent Rights For Nobel-Prize-Winning Method to Treat Diseases Like Hepatitis

Yale University and Innovir Laboratories Inc. of New York City recently announced they have been granted additional broad patent rights for a promising technology for treating and preventing a number of diseases, including hepatitis. The technology, also useful in genetic research, is based on discoveries that earned Yale biologist Sidney Altman the 1989 Nobel Prize in Chemistry.

Professor Altman shared the prize with Thomas Cech of the University of Colorado for experiments showing that RNA is not just a passive carrier of genetic code, but that it can also be an enzyme that actively engages in chemical reactions. Their discovery of RNA enzymes, or “ribozymes,” in separate experiments performed in the late 1970’s and early 1980’s triggered a new branch of genetic engineering aimed at treating lethal viruses and repairing genetic defects.

Innovir Laboratories is conducting research on External Guide Sequence EGS oligozymes, which seek out and attach to the genetic blueprint of specific viruses so a ribozyme can cut the blueprint into shreds, making it unreadable. Innovir is developing EGS molecules that target viruses that cause hepatitis B, hepatitis C, psoriasis and other inflammatory diseases, as well as drug-resistant infections.

As a research aid, EGS oligozymes hold potential both to identify how different genes function and to identify molecular targets for new drug therapies, which could help pharmaceutical companies discover new drugs.

Professor Altman, the Sterling Professor of Biology, recently was issued a U.S. patent, and Innovir has been allowed two additional patentapplications, which provide comprehensive patent protection for the EGS oligozyme technology. Innovir has an exclusive worldwide license from Yale to commercialize the technology, which already is backed by 3 issued patents, 6 allowed patents and 20 pending patents.

Sole rights for treatment in all animal species and cell types

Under its license with Yale, Innovir has sole rights to EGS for use in targeting infectious agents in all animal species and cell types. The company’s patents cover both the EGS technology – including design, synthesis, chemical modification and delivery – and its application for specific diseases.

“These patents further establish our commanding and unique competitive position in EGS oligozyme-based research and development,” said Allan R. Goldberg, Ph.D., who is president and chief executive officer of Innovir. “No intellectual property possessed by any other competitor compromises Innovir’s dominant patent position in being able to fulfill this technology’s extraordinary promise in therapy and research.”

Innovir announced in December key advances in its tests of EGS technology against hepatitis B, a widespread and serious viral disease for which there is currently no broadly effective treatment. Dr. Goldberg said EGS reduced the amount of viral DNA in cell cultures to undetectable levels – a decrease of at least a hundred-fold in comparison to untreated control cultures. EGS was ten times more potent in inhibiting the virus than the leading drug being developed to treat hepatitis B.

Initial test results in a mouse model that produces the hepatitis B virus demonstrate that Innovir’s technology also is a potent inhibitor of hepatitis virus growth in animals, Dr. Goldberg said.

In January, Innovir and VIMRx Pharmaceuticals Inc. of Wilmington, Delaware, announced an agreement giving VIMRx a controlling interest in Innovir, while Innovir acquired VIMRx’s subsidiary, VIMRx Holdings Ltd. VIMRx is developing another class of oligozymes called RILON*, as well as treatments for the HIV virus and brain cancer. “There are true opportunities for synergy here, and we have already begun to take advantage of them,” Dr. Goldberg said. “This is clearly a case of the whole being more than the sum of the parts.”

How EGS Oligozymes Work

EGS molecules are small oligozymes chemically modified oligonucleotides that have been engineered to bind to targeted “messenger RNA” mRNA , a family of genetic compounds that play a key role in controlling body chemistry. Once the EGS molecules attach to their target, they cause a ribozyme called RNase P to destroy the mRNA to which they are bound. The EGS molecules are then freed to repeat the process. The technology’s therapeutic value lies in the fact that it can be used to seek out and destroy the mRNAs associated with particular diseases, such as hepatitis.

Researchers also can use EGSs to inactivate mRNA molecules in a highly selective way to gain a better understanding of how cellular chemistry functions. To compliment its research programs, Innovir has developed a unique drug delivery tool called InnoPhor* that makes it possible to target oligozymes to specific tissues.