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URMC Researchers Form Company to Develop New Class of HIV Drugs

Monday, January 12, 2004

Researchers at the University of Rochester Medical Center have begun work on a new class of HIV drugs that may enable the body’s own immune system to virtually halt an HIV infection.  The researchers have formed a company, called OyaGen, Inc., to conduct further laboratory tests needed before clinical trials can begin.   

            The company’s groundbreaking research centers on a crucial enzyme called CEM15, which is produced by human immune system cells and is able to kill a wide range of viruses.  Known as an “editing enzyme,” CEM15 slips inside a virus and later attacks or “edits” its DNA – that is, it makes chemical changes to the genetic instructions that allow the virus to replicate.  With its DNA garbled, the virus can no longer replicate and infect other cells.

            But unlike other viruses, HIV possesses the deadly ability to disable CEM15 by unleashing a protein called “vif” that latches onto it and prevents CEM15 from functioning.  With CEM15 neutralized, HIV is able to infect cells and replicate freely, eventually overwhelming the immune system and leading to death.  

            Researchers at OyaGen are testing a potential drug designed to prevent vif from attaching to CEM15.  In lab tests on HIV-infected cells, the drug shielded CEM15 and enabled it to function normally, nearly halting the spread of the infection to other cells.  Studies seeking a variation of this drug – delivered as a smaller molecule that is less likely to have side effects – will begin within the year.  The researchers will also attempt to deliver additional CEM15 directly to cells in hopes of providing enough of the enzyme to overwhelm HIV’s vif proteins and mount a successful attack on the virus’ DNA.

            The new drugs have been classified as HIV editing enzyme drugs, or HIVEE drugs, and many in the scientific community believe that an approach of targeting editing enzymes therapeutically may represent the most effective method yet for disabling HIV.  While current antiviral drugs can stop HIV from replicating for a time, they ultimately prove ineffective because the virus eventually mutates, or changes, in ways that make it resistant to the current arsenal of drugs.  In contrast, because OyaGen’s HIVEE therapeutics facilitate a direct and lethal attack on the virus’ DNA, they may solve the intractable problem of viral resistance. 

              To bring the first HIVEE drugs to market, OyaGen has combined the technology and knowledge base of two researchers.  Harold Smith, Ph.D., professor of Biochemistry and Biophysics at the University of Rochester School of Medicine and Dentistry and a leading authority in the emerging field of editing enzymes, is OyaGen’s founder and Chief Scientific Officer.  Hui Zhang, M.D., Ph.D., an HIV researcher and associate professor of Medicine at Jefferson Medical College in Philadelphia, will serve as a scientific advisor to OyaGen.   

              Along with Dr. Smith’s active participation, the early business development efforts were led by Trillium Venture Development, manager of the Seed Fund, in collaboration with the Technology Transfer Office at URMC and the University of Rochester’s William E. Simon Graduate School of Business.   Members of the Trillium Venture Development Team will serve as interim executive managers and Bill Carpenter, former Chairman & CEO of Bausch & Lomb, will serve on the Board of Directors.

             “The formation of OyaGen is precisely the type of business-science partnership, or technology transfer, that is necessary to move this science from the lab to the clinic,” said C. McCollister Evarts, M.D., senior vice president and vice provost for Health Affairs at the University of Rochester and CEO of the UR Medical Center.  “Dr. Smith’s work has important implications for the treatment of HIV, and we want to move it into the commercial sector so that clinical trials can begin as rapidly as possible.”

            Because of market need, HIV drugs typically experience rapid market adoption and peak market revenues between $250 million and $1 billion.  If successful through all phases of its clinical trials, OyaGen’s first anti-HIV drug should be market-ready by 2010.  “The HIVEE drugs that could be developed by OyaGen  represent a new frontier in HIV therapeutics,” said Jose’ Coronas, General Partner of Trillium Group, the fund manager of the University Technology Seed Fund.

              Once the HIVEE drugs are optimized, OyaGen will shift its focus to therapies for other disease states in which editing enzymes play a role such as atherosclerosis and cancer.   A series of at least 14 editing enzymes exist.  These have been identified within the last two decades and have created an opportunity to harness the editing process to combat disease states.   

Trillium Group, LLC, is a venture capital and commercial development company specializing in start-up and early stage businesses.  The Trillium Group team includes Judy Albers, José J. Coronas, Dennis M. DeLeo, Bud Frame, Chris O’Donnell, Kevin J. Phelps and Frank P. Strong, Jr.  For information, contact Trillium Group at 585-383-5680, or visit the firm’s web site at

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