United States Biochemical Corp. and a group of venture capitalfirms have started Ribozyme Pharmaceuticals Inc. to developpharmaceuticals based on the work of Nobel Prize winnerThomas Cech of the University of Colorado.

Cech (pronounced "check"), who will chair RPI's scientificadvisory board, shared the 1989 Nobel Prize in chemistry withSidney Altman of Yale University for the discovery ofribozymes. The enzymatic RNA molecules are able to act as"molecular scissors," clipping RNA at selected sites without thehelp of proteins.

Products in development by RPI include ribozymes targeted atherpes simplex virus 1 and 2, HIV, cytomegalovirus andvarious forms of cancer. The company said it will file aninvestigational new drug application for one of thoseindications within the next 18 months.

RPI's viral work is targeting proteins crucial to viral replication,said Sean Sullivan, director of ribozyme delivery. In cancers,the company aims to develop ribozymes that target specificsequences within an oncogene's messenger RNA so that it canno longer code for the cancer-causing protein.

Privately held USB, which sells research compounds andsequencing technologies, spun off RPI to obtain better financingfor the technology, accelerate the science and attractmanagement, said Dale Schwartz, RPI vice president of businessoperations.

Venture firms Morgenthaler Ventures, Venrock Associates, CWGroup and Advent International have invested $6 million.

RPI is the second recent collaboration between a parentcompany and a venture group. In March, Onyx Pharmaceuticalswas started by Chiron Corp. and Avalon Ventures, InstitutionalVenture Partners, J.H. Whitney & Co., and Kleiner, Perkins,Caufield and Byers. Onyx will develop small-molecule drugstargeted at genes or proteins inside the cell that regulate thegrowth of cancer cells.

"The more typical past approach was for spinoffs to leave theirparents," said Anthony Evnin of Venrock. Now that the parentcompanies have more resources and management is moremature, they are better able to retain ties with their offspring,he said.

For venture capitalists, he said, "the issue isn't so much riskavoidance as perhaps having a richness in the technology. Insome cases you will get human, technological and capitalresources from the parent company, and it's often the only wayto get access to that technology. You also avoid any battle overrights to the technology. I suspect you'll see more of it."

In this case, Morgenthaler's partnership with a corporation wasan accident of history, and the firm isn't specifically seekingsuch deals, said Paul Brentlinger, a partner at Morgenthalerand currently president of RPI.

However, he added, "the fact that United States Biochemical isbehind this and had spent a lot of money to get it to that pointwas clearly a plus. There's no question that while RPI is a newcompany, it's way beyond the start-up stage, and that wasdefinitely a plus for us."

One concern is that the deals be structured so that the newcompany is not inhibited from developing relationships withother large companies, said Evnin.

Cleveland-based USB has exclusively licensed to RPI thetechnology it has developed during the past five years inexchange for an unspecified equity stake.

That technology includes rights to the first U.S. patent to coverribozymes, No. 4,987,071, issued in January 1991, as well as toseveral other issued patents. The '071 Cech patent coverspurified and man-made ribozymes, production methods andtheir use in cleaving other RNA molecules.

RPI expects to announce shortly that it has hired a newpresident and CEO, said Schwartz. The company, currentlybased in Cleveland, plans to relocate by autumn to Boulder,Colo., where the University of Colorado is located.

-- Karen Bernstein BioWorld Staff

(c) 1997 American Health Consultants. All rights reserved.