By Brady Huggett

The young company PTC Therapeutics Inc. said it now can build its value and pursue aggressive growth in the area of RNA technologies due to the $15 million it raised in the spring and disclosed last month.

"This should last us two to three years, depending on how aggressively we grow and what our board wants us to do," said Stuart Peltz, president and CEO of PTC. "But our view is really to be as aggressive as possible and be the leader in this field."

Vulcan Ventures Inc., of Bellevue, Wash; CSFB Private Equity, of New York; and New Medical Technologies, of Basel, Switzerland, led the round. Other shareholders in PTC are Tularik Inc., of San Francisco; the Swanson Family Trust Inc.; Broadview Ltd.; Fred Frank; and the University of Medicine and Dentistry of New Jersey.

Peltz founded the South Plainfield, N.J., company in 1998, along with Tariq Rana, Allan Jacobson and the financial help of Tularik. The company raised approximately $1.5 million in 1998 through a small seed round and a couple of milestone payments. The $15 million represents its first significant round, and a portion will fund PTC's imminent move to its new 22,000-square-foot facility.

"By next June, we should have spent $5 million," Peltz said. "The building will have about 12,000 square feet of laboratory space and we are going to equip it with chemistry and throughput screening equipment by that time. We also are in a hiring spree right now - we're at about 15 employees but are shooting for 30 to 40 by June. It has been so easy to recruit."

Peltz said the final inspection of the building is this week.

That will leave PTC with around $9 million in cash by June, and Peltz said although future financings or even going public is a possibility, $9 million gives PTC a chance to grow the way it wants.

"[An IPO] has entered out thoughts, and we may do another round of financing after a year or two," Peltz said. "But with the financing behind us, we can do what is good for us rather than what we need to do at the moment. Now is the time to build a strong company on RNA biology."

PTC has identified novel drug targets that arise from numerous intervention points in the mechanisms by which RNA is translated into proteins and subsequently broken down. Its proprietary technologies and chemical libraries have led to the identification of small molecules that affect these control processes. PTC's technologies also include chemical libraries and assays that screen for small molecules that modulate RNA function.

When synthesizing proteins, the presence of nonsense codons terminates the translation process. Peltz said that when looking at genetic disorders, 15 percent to 30 percent are caused by an early nonsense codon. Early nonsense codons cause many cystic fibrosis cases and are one of the primary causes of Duchenne's muscular dystrophy. PTC uses small molecules to induce translation machinery in the cell to bypass inappropriate nonsense codons and also to induce cells to produce complete, functional proteins.

"The beauty of this approach is that a single drug or a setup drug can treat a variety of these types of diseases," Peltz said. "You are focusing on gene expression, not the disease."

PTC is in collaboration with Tularik, which owns less than 5 percent of PTC, to discover therapeutics, but does not have any other partners or collaborations.

"We've been approached by groups, and if there is a good deal, we'll do it," Peltz said. "But we want to build value into what we have and partner later."

Based on the broad applications Peltz sees for RNA translation, he said the company is poised to make its move.

"The technology is so strong," he said. "This is not a one-trick pony - you can address so many diseases this way. I think there is so much opportunity in this field and our goal is to be as aggressive as possible."

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