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A faster pipeline speeds new treatments from lab to patient

Medicine@Yale, 2006 - July Aug


Small biotechs with big ambitions hasten drug testing

The flow of discoveries from the medical school’s labs to patients in need is proceeding at a blistering pace—in the last four years, no fewer than five new therapies invented by School of Medicine researchers have advanced into human clinical trials. Yale’s Office of Cooperative Research (OCR), which takes the lead in licensing the fruits of faculty labors to biotechnology and pharmaceutical companies, currently lists a dozen additional potential therapies under license and poised to start early stage clinical trials (see graphic).

This unprecedented success in pushing investigators’ finds out of the lab comes from OCR’s strategy of partnering with up-and-coming biotech companies, and in some cases catalyzing the creation of brand-new companies tailored to the needs of particular therapies.

The result has been a significant contraction of the time it takes to move new treatments from licensing to first trials in humans. For example, Proteolix, founded in 2003 to commercialize compounds discovered by Craig M. Crews, Ph.D., associate professor of molecular, cellular and developmental biology and pharmacology, took just 18 months from its founding to obtain Food and Drug Administration (FDA) approval to test one of Crews’s cancer drugs in people.

Likewise, Achillion Pharmaceuticals, a start-up company whose first compound was discovered by Yung-Chi “Tommy” Cheng, Ph.D., the Henry Bronson Professor of Pharmacology, began a Phase I trial of the drug to treat HIV/AIDS only a year and half after the company’s inception.

In addition, Applied Spine Technologies, another young company, is poised to begin human trials of the M-Brace, an implantable device invented by Manohar M. Panjabi, Ph.D., Dr.Tech., professor of orthopaedics and rehabilitation. The M-Brace stabilizes the spine to relieve chronic low-back pain.

Because start-ups can garner venture capital based on a few promising clinical candidates or an exciting technology, small biotechs are driving the most focused and aggressive development of new treatments, says Jon Soderstrom, Ph.D., managing director of OCR. “We know that the most important thing we can be doing in medicine right now is developing new therapies,” Soderstrom says. “What we’re seeing is that the time frame that a biotech takes to do that is running about half of what we see in the traditional pharmaceutical industry.”

The intense focus of small biotechs on just a few compounds benefits academic researchers looking to quickly translate their work to new medicines, says John W. Puziss, Ph.D., OCR’s director of technology licensing. “When we license a compound or technology to a start-up, that company raises tens of millions of dollars committed to taking that compound to market,” says Puziss. “Where big pharma might take 5 years to get an invention into the clinic, investors in small companies want to see results in 2 years.”

If a biotech’s drug is promising in early-stage clinical trials, rights to the drug—and sometimes the entire biotech company—may be acquired by a traditional “big pharma” firm. Such acquisitions fill out big companies’ research and development pipelines, but the bigger firms’ greater expertise in manufacturing, marketing and distribution also ensure that useful drugs will reach the doctors and patients who need them.

Yale picked up on this drug-development trend early on, says Paul R. Pescatello, J.D., Ph.D., president and CEO of New Haven-based bioscience advocacy group CURE (Connecticut United for Research Excellence). “Historically, Yale worked well with big pharma, and OCR understood pretty quickly that the more little companies they could seed, the more they’d increase the odds of the research being picked up by big pharma to be developed and ultimately marketed.”

A bonus for New Haven is the fact that most of the new companies licensing Yale discoveries are local, a big plus for the area’s economy. By bringing together scientists with entrepreneurs and venture capitalists, OCR has helped launch 30 or so companies that form the core of New Haven’s biotech cluster, and most of the 49 biotechnology companies now doing business in Connecticut have their roots in Yale research.

Yale’s success story starts with the impressive research of its faculty, OCR’s Puziss is quick to point out. And their efforts have been rewarded—royalties from the HIV/AIDS drug Zerit, the brainchild of William H. Prusoff, Ph.D., professor emeritus and senior research scientist in the Department of Pharmacology (see related story), provided the bulk of the funds to build the Anlyan Center, the medical school’s main research facility, completed in 2003.

But money isn’t the main point of OCR’s efforts, Puziss emphasizes. “There are a number of examples in our pipeline now entering the clinic that could very well reach the marketplace—and save lives,” he says. “That’s what we’re all about.”