TAG: "Tech transfer"

Can ‘intelligent design’ deliver?

UC Berkeley bioengineer David Schaffer harnesses evolution to improve drug delivery.

David Schaffer, UC Berkeley (Photo by Peg Skorpinski, UC Berkeley)

By Wallace Ravven

What do Washington lobbyists and gene therapy have in common? Success for both depends on access and influence.

By and large, most drugs are “small molecules,” able to find their way to targets between cells or inside specific cells. They can provide relief from acid reflux, infection or inflammation, or keep serious conditions like diabetes and cardiovascular disease under control. But they demand refill after refill. They aren’t cures.

Serious genetic diseases also require a lifetime of treatment. Bronchodilators and inhaled antibiotics treat severe congestion and lung infections that often shorten the lives of people with cystic fibrosis. Penicillin, blood transfusions and even bone marrow transplants treat sickle cell anemia. A single gene defect causes both conditions.

If the normal gene could somehow replace the functions of the abnormal one in affected cells and tissues, the diseases would be cured. This, of course, is the high ambition of gene therapy.  For more than 25 years, researchers have tried the savvy-sounding approach of delivering corrective genes to affected cells by ferrying them inside benign human viruses. Viruses know the territory intimately, so why not piggy back on a proven vehicle?

But, says UC Berkeley bioengineer David Schaffer,  “There’s an old joke about gene therapy. ‘The are only three problems in the field: delivery, delivery, delivery.’”

The strategy has run up against a powerful force: evolution. Viruses and the human immune system are locked in a timeless arm’s race. The invader hones its skills, and our immune systems develop antibodies and other defenses to neutralize any innovations. The more persistent the pathogen, the more likely our immune system has encountered it before and has developed antibodies to protect against it.

“We’re fighting biology and evolution,” Schaffer says. The viruses must also reach the right cell type among many within a tissue, and in sufficient numbers to deliver its gene cargo. The challenges have stalled gene therapy from its outset.

Schaffer, director of the Berkeley Stem Cell Center, is a professor of chemical and biomolecular engineering. He was named one of the “Top 100 Innovators under 35” by Technology Review magazine in 2002 for his early efforts to actually use  evolution to overcome the hurdles of viral gene delivery. Clean-cut in a checkered shirt, he still looks to be that same young bioengineer. And he has kept his focus on delivering gene therapy.

Rather than trying to quiet the body’s defenses against viruses, Schaffer has favored a kind of intelligent design approach to modify the virus. Known as directed evolution, the strategy uses genetic engineering to find variations in the virus that will allow it to effectively deliver drugs to target cells.

His lab works with a common, harmless human respiratory adeno-associated virus, or AAV. More than 90 percent of the human population has been infected by this virus, so it’s got a proven track record for invasion, which our immune systems remember. Schaffer spurs the virus’s evolution in search of a variant that can overcome the immune and tissue defenses.

Our bodies pose many barriers to viral infection, including immune system recognition of the virus’s outer shell, or capsid. In the 1980s researchers discovered that natural variations of a 740-amino-acid-long protein self-assemble to make the AAV capsid.  Schaffer’s lab used genetic engineering to shuffle and mutagenize the amino acid sequences and generate “libraries” of millions of variants of the capsid protein.

The team then screened the millions of variants, selecting for the few that could best elude human antibodies, home to the  right tissues, and make their way to chosen target cells. The result of this directed evolution: a vehicle that can deliver healthy genes to defective cells.

Last year, he and UC Berkeley colleague John Flannery showed that the virus could deliver curative genes to eye cells to cure several blinding diseases in mouse models. The results show promise for a new way to treat disorders from the inherited defects of retinitis pigmentosa to age-related macular degeneration.

“We’ve created a virus that can deliver genes to a very-difficult-to-reach population of delicate cells in a way that is surgically non-invasive and safe,” Schaffer said at the time. Their success was published in the journal Science Translational Medicine.

His lab also applies the directed evolution strategy to stem cell gene therapy. He and colleagues recently reported using directed evolution of the AAV virus to deliver potentially curative genes to human pluripotent stem cells. “There are many diseases that can be cured by treating stem cells” he says.

The stem cell research field is still young, and the Berkeley Stem Cell Center encourages collaborations between investigators who examine fundamental stem cell properties and bioengineers such as Schaffer who aim to harness the new knowledge.

His success with directed evolution led him and colleagues to launch a startup last year, called 4D Molecular Therapeutics. They are now working with uniQure, the only company that has a clinically approved gene therapy product. uniQure intends to put 4D’s next generation, evolved viruses  into their clinical pipeline.

“The campus really recognizes that translating our research to clinical use is central to its mission to have an impact on society. It’s also a potential source of funds for the campus,” he says.  Schaffer sees gene therapy starting out on a track to treat relatively rare diseases for which no other therapies exist. Ultimately though, with effective delivery systems, gene therapy could find its way to treat common diseases involving faulty proteins, such as macular degeneration, diabetes, and heart disease, he says.

That, of course, is well down the line, but Schaffer can see it: “Medicine in the future may involve intelligent viruses that with a single injection can cure a host of diseases.”

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Entrepreneurship program prepares inventors to launch startups

UCSF helping to support its innovators who seek to commercialize life-changing inventions.

By Bob Rose

As a young girl growing up in the outskirts of Bombay, India, Charvi Shetty has distinct memories of her mother and older brother struggling to breathe.

Beyond coping with the air pollution that sometimes blankets their homeland, both family members suffered from chronic asthma. Shetty’s Filipino mother and Indian father wanted opportunities that India could not provide, so they immigrated to Petaluma with their children when Shetty was 10 years old.

“My parents wanted my brother Rajiz and me to have a better life by receiving a college education in America,” she said.

Her asthmatic mother and brother sparked Shetty’s interest in biology during high school, which led to her majoring in bioengineering at UC Berkeley. Upon graduating in 2012, Shetty enrolled at UC San Francisco where she pursued a master’s degree in biomedical imaging.

During that time, she developed an affordable medical device that measures lung function and capacity, and detects early onset of asthma. An accompanying mobile app can assist hospitals, as well as parents and individuals, with tools that measure airways and response to medications.

Shetty’s invention, which could significantly improve the quality of life for asthmatics, can benefit millions but only if the device can successfully go to market. And that’s the rub for many aspiring UCSF students and recent graduates, whose expertise is in research or patient care, not the business world.

Entrepreneurial ecosystem

Shetty’s story is not unusual, but there is hope for innovators like her. UCSF restarted its Entrepreneurship Center in March 2012. The program helps companies start from UCSF inventions and has built an entrepreneurial ecosystem at the University. UCSF’s environment is unique, differing from other universities such as Berkeley, Harvard and Stanford because UCSF lacks a business school or a computer science program whose specialized expertise can help innovators build apps.

Stephanie Marrus, who holds an M.B.A. from the Wharton School at the University of Pennsylvania, directs the Entrepreneurship Center. She has worked with hundreds of companies in science- and technology-based industries.

She recently partnered with tech entrepreneur Steve Blank, who has changed the paradigm of entrepreneurship education in the U.S. and last year was named one of the 30 most influential people in Tech by Forbes magazine. Marrus and Blank adapted his nationally respected course, Lean LaunchPad, to the life science and health care audience at UCSF.

Over the past three years, Marrus has recruited a network of 200 mentors and advisers with backgrounds as life sciences CEOs, investors, attorneys and consultants from Silicon Valley’s entrepreneurial ecosystem to advise students in the courses and UCSF entrepreneurs outside the classroom.

Meaningful mentorship

The Entrepreneurship Center provides training on startup businesses, hosts top-tier speakers from the business world, organizes a support group for serious UCSF entrepreneurs, and holds events open to UCSF, Berkeley, Stanford, startup accelerators and the business community.

“What I really learned from the class was that the greatest innovation isn’t necessarily the product,” Shetty said. “It’s the novel and innovative business model.”

As part of her participation in the Lean LaunchPad program, Shetty spoke to numerous people with asthma. She was also paired with an experienced venture capitalist as her mentor — a physician who has founded several companies.

“We tested a whole bunch of hypotheses,” Shetty added. “Was there a real need for kids? Was there a real need by doctors? Will individuals pay for the product or will they need to be reimbursed?”

Her efforts have been supported by an international company-building program called Founder.org, which helped to advance her fledgling company, Knox Medical. She competed with teams from 24 schools in the U.S. and Europe and was selected from approximately 1,000 applicants.

Last fall the National Institutes of Health piloted the Lean LaunchPad class based on the UCSF model to help aspiring life science entrepreneurs commercialize their technology. UCSF is continuing to teach the course on its campus and is extending its reach to the other UC biomedical campuses this June, including UCLA, UC Irvine, UC San Diego and UC Davis.

“We are excited that Lean LaunchPad in the life science/health care domain is becoming recognized as the model for training scientific and clinical entrepreneurs,” Marrus said. “We believe that this market-based approach to vetting startup ideas can make the difference between success and failure.”

The program could not come at a better time for UCSF, which is increasing its dedication to entrepreneurship in line with the UC president’s priorities.

Avoiding pitfalls

Hobart Harris, M.D., who has served as chief of general surgery at UCSF for the past 13 years, believes the Lean LaunchPad program is indispensable. Harris, a Harvard graduate who founded Vitruvian Medical Devices in 2012, saw firsthand how novice entrepreneurs can fall victim to faulty logic.

“I had fallen prey to a very common assumption that I think naïve or inexperienced entrepreneurs suffer and that is thinking that if your idea works, then all the additional steps required to bring your idea to the marketplace will just fall into place, like a row of dominoes,” Harris said. “Nothing could be further from the truth. When I attended the Lean LaunchPad course, through the interview process we saw our ideas kind of turned on their head in respect to both pricing and what would be the required information that would compel users to buy our product.”

The Flint, Michigan, native soon discovered that obtaining the regulatory approval for his product would cost more than $75 million and take four to five years. That realization led him to change the nature of the device and the patient population he would first look to serve.

“It probably would have been years before we ultimately realized we were taking the inappropriate path or approach,” Harris added. “I can say this: There have been two or three courses in my entire education that have genuinely changed the way I think, and this course is one of those.”

Innovation through adversity

Ernesto Diaz Flores, Ph.D., an assistant adjunct professor at UCSF and founder of Challenging Solutions, is another passionate advocate of the Lean LaunchPad program.

Born in El Salvador, his family moved to Spain when he was 10 years old. Since his childhood, Diaz Flores gravitated to athletics and displayed remarkable footwork as a competitive tennis player and also as a flamenco dancer. He was also an outstanding scholar, eventually earning a B.S. degree in biochemistry at University Complutense of Madrid — and finishing in the top five in his class — and then obtaining a Ph.D. at the Universidad Autonoma de Madrid in molecular biology with honors.

Yet, it was when he was derailed as a tennis player and dancer that his professional career began to take shape.

“When I was doing my Ph.D. work, I broke my left foot,” Diaz Flores recalled. “I tried to go to work on a cast and crutches but after getting to work, I realized I couldn’t use my hands very much because of the crutches.”

His only option to stay productive was to use a wheelchair.

“I soon realized how people with low or no mobility from the waist down get disabled by the chair itself,” he said. ”You cannot stand or reach high or be at eye level with your friends, even when you have partial or full mobility in the upper body.”

He hoped someone would eventually address this limiting feature, but as the years went by nobody did.

“People were still locked in one position in wheelchairs, so I decided to work on a device and bring it to fruition,” Diaz Flores said. “That’s how the idea was born.”

He designed and developed an innovative mobility device that allowed wheelchair-bound people to both sit and stand, and stably ride in both positions at adjustable speeds. His invention is also a hybrid in that a client can operate it in manual mode, which helps with exercising, or in automatic, which facilitates transportation. The device, which has a cutting-edge appearance, also is compact and lighter than the conventional wheelchair, allowing entry to non-accessible places like some restrooms and through narrow doors.

“When I started this venture, I had no entrepreneurial background so I took courses from the Entrepreneurship Center at UCSF,” Diaz Flores said. “That was an eye-opening experience into a world that was completely foreign to me. It helped me get acquainted with the different financial process, lingo and things that I needed to put in place to have a successful venture.”

After much time and effort, including seeking customer feedback on design and physical needs, Diaz Flores has established his team members and now the company is incorporated. He reports that the venture is close to having its first functional prototype and will start to test it.

Meanwhile, he continues to take courses and attend lectures organized by the Entrepreneurship Center and meets regularly with Marrus to seek her input and advice.

These anecdotes reflect how vital this program has been to not only Diaz Flores but also hundreds of other inventors who are fast establishing UCSF as one of the leading entrepreneurial universities in life sciences and health care.

“The mere fact that such a center exists here at UCSF serves as demonstration of the University’s investment in the futures of its trainees,” said Nima Emami, a current Ph.D. student studying bioinformatics in the Graduate Division. “It was a major factor in my decision to pursue my Ph.D. at UCSF over several other top research universities in my field.”

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Calico licenses technology from UCSF laboratory

Project seeks to develop potential therapies for cognitive decline.

Calico, a company whose mission is to harness advanced technologies to increase understanding of the biology that controls human lifespan, and UC San Francisco have partnered on an innovative project to develop potential therapies for cognitive decline.

Under the agreement, Calico will receive an exclusive license to technology discovered in the laboratory of Peter Walter, professor of biochemistry and biophysics at UCSF. Walter is an investigator in the Howard Hughes Medical Institute, member of the U.S. National Academy of Sciences and recipient of the prestigious Albert Lasker Basic Medical Research Award in 2014.

The agreement is focused on modulators of the integrated stress response (ISR), a set of processes activated in cells under conditions of stress. Under some circumstances, the ISR can be deleterious. For example, under certain circumstances the ISR may contribute to memory decline, a significant problem potentially addressed by the licensed technology.

“We are delighted to enter into this agreement with UCSF and we are excited to translate these research findings into potential treatments for age-related cognitive disorders,” commented Hal Barron, Calico’s president of research and development. “Peter is a world-class basic scientist whose insights have fundamentally changed our understanding of how cells function under stress.”

The work conducted in the Walter laboratory was led by Carmela Sidrauski, a former UCSF researcher and now a scientist at Calico.

“Calico will be a great partner to explore the promise of our research,” Walter said. “Their commitment includes conducting key additional research, hiring outstanding investigators like Carmela and providing critical development expertise.”

Under the terms of the agreement, UCSF will receive an undisclosed up-front fee, and potential milestone and royalty payments. Calico will take responsibility for further research, development and commercialization of resulting therapeutics.

This partnership was facilitated by the UCSF Office of Innovation, Technology & Alliances (ITA), which coordinates UCSF’s efforts in forging collaborations and licensing technologies that translate cutting-edge science on campus into therapies and products that directly benefit patients worldwide.

Calico (Calico Life Sciences LLC) is a Google-founded research and development company whose mission is to harness advanced technologies to increase our understanding of the biology that controls lifespan. Calico will use that knowledge to devise interventions that enable people to lead longer and healthier lives.

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UC inventions on display at Tech Commercialization Forum

Breakthrough technologies creating public benefit, improving health.

A system for removing arsenic from groundwater, developed at Berkeley Lab, is helping to provide safe drinking water for people in India and Bangladesh.

By Carolyn McMillan

Lawrence Berkeley Laboratory scientist Ashok Gadgil has developed an inexpensive and easily maintained system for removing arsenic from groundwater.

It’s a breakthrough technology that could protect millions of people around the world from arsenic poisoning, a silent killer that is especially prevalent in the rural villages of India and Bangladesh.

Across the bay, a UC San Francisco orthopedic surgeon has tackled a different sort of epidemic — the thousands of young athletes who tear knee ligaments every year while playing soccer.

Dr. Jeffrey Lotz has developed a faster, better and cheaper way to assess whether an athlete is at risk for lower extremity injuries. His 3-D movement analysis system lets physical therapists and trainers assess an athlete’s body mechanics in order to prescribe the right exercises to avoid injury.

The products of research

These two innovations are just a fraction of the commercially viable discoveries and inventions to emerge recently from the University of California — and part of what makes UC such a powerhouse of economic growth for the state.

UC researchers reported more than 1,700 new inventions last year, according to the recently released Technology Commercialization Report. UC inventions led to 71 new start-up companies and produced roughly $106 million royalty and fee income for the university.

UC President Janet Napolitano, speaking at the university’s annual Technology Commercialization Forum on May 8, said that UC is committed to doing even more to help faculty, researchers and students bring their discoveries to the marketplace.

“Steering UC’s cutting-edge discoveries through our labs and into the world economy is central to our mission as a public university,” Napolitano said. “We are committed to supporting our faculty and students with a strong, nimble infrastructure that will help them pursue patents and develop start-up companies, and we will continue to develop partnerships with industry and investors.”

Gadgil and Lotz were among 20 UC inventors to showcase their innovative research at the forum. The work spanned a huge array of fields and came from eight UC campuses and Lawrence Berkeley National Laboratory.

Improving life and environment

At UC San Diego, a clinical trial is under way to test an oxygen delivery system for people with chronic pulmonary obstructive disorder (COPD), a respiratory ailment. The system automatically responds in real time to a patient’s changing oxygen needs, delivering the right amount of oxygen with each breath.

“Our goal is to increase the autonomy and mobility for people with COPD,” said Dr. Xaxier Soler, associate director of UC San Diego’s pulmonary rehabilitation program. “We want to extend their quality of life.”

The technology was developed  by  co-inventors Stephen Roberts and David Lischer — the latter a COPD patient who was frustrated that current oxygen devices limited his activity levels. Lischer has tested a prototype of the new device since 2010, and it works well enough that he now goes skiing, Soler said.

At UC Davis, Basam Younis has developed the next generation of water disinfection systems using UV light, rather than chlorine, to eliminate pathogens. Younis’ system achieves a higher level of water purification than earlier UV systems — and does it at a lower cost.

Better yet: His system has proven to be particularly effective at eliminating what Younis referred to as “emerging contaminants” — things like pharmaceuticals, flame retardants, human hormones and care products — which increasingly are found in water supplies.

Public benefit

Steven Beckwith, UC’s vice president for research and graduate studies, said that the breadth and depth of innovation is a reflection that UC has created an “ecosystem of discovery” across its campuses and labs.

“This is the largest, and without a doubt, the most prestigious university in the world,” Beckwith said. “Our researchers tackle complex issues and look to answer some of the biggest questions that society has. And ultimately, we are looking to create a benefit for the public.”

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Corporate-funded academic inventions spur increased innovation, analysis says

Findings based upon a study of two decades of records from the UC system.

Academic research sponsored by industry has a strong track record of leading to innovative patents and licenses, challenging assumptions that corporate support skews science toward inventions that are less accessible and less useful to others than those funded by the government or nonprofit organizations, according to a new analysis.

The findings, published today (March 19) in a commentary in the journal Nature, are based upon a study of two decades of records from the University of California system.

The authors, led by Brian Wright, UC Berkeley professor of agricultural and resource economics, analyzed 12,516 inventions and related licenses at nine UC campuses and three associated national laboratories. The inventions were disclosed between 1990 and 2005, and licensing activity was analyzed through 2010. Of the inventions, nearly 1,500 were supported at least partly by private industry. (UC Merced, the 10th UC campus, was not included because it opened in 2005.)

The analysis found that industry-funded inventions yielded patents and licenses more frequently than federally sponsored ones, with results consistent across technical fields. The researchers also found that industry-sponsored inventions were more highly cited in subsequent patent applications — known as “forward citations” — the most widely used marker of a patent’s quality and importance. Each corporate-sponsored invention generated an average of 12.8 forward citations compared with 5.6 for federally sponsored inventions.

“This runs counter to the expectation that corporate-sponsored inventions have narrow applications, and so create … few benefits for others,” the authors wrote.

Because corporations usually get first crack at negotiating licenses to the inventions they sponsor, there is an assumption that corporations would tie up innovative discoveries in a way that restricts access to a broader audience.

To illustrate those concerns, the authors referenced reactions to a 1998 deal with Swiss pharmaceutical company Novartis to support biotechnology research at UC Berkeley, and to a research consortium, the Energy Biosciences Institute (EBI), funded by energy giant BP and led by UC Berkeley.

In both cases, critics expressed fears that corporate interests would stifle UC Berkeley’s public mission by locking up discoveries for industry profits. (The Novartis project yielded no patents, and EBI began too late to be included in the study.)

However, the intellectual property data analyzed by the authors indicate that industry has not been more likely than federally sponsored research to tie up research discoveries in exclusive licenses. Overall, corporate-funded inventions were licensed exclusively 74 percent of the time, while federally funded inventions were licensed exclusively 76 percent of the time. Notably, among the corporate-funded inventions with exclusive licenses, half seemed to go to third parties and not the sponsor.

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Research leads to affordable technology to fight mosquito-borne diseases

UC Riverside discoveries are foundation for Olfactor Labs to develop easy-to-wear patch.

The Kite Mosquito Patch disburses non-toxic compounds that provide individuals with up to 48 hours of protection from mosquitoes.

The Kite Mosquito Patch disburses non-toxic compounds that provide individuals with up to 48 hours of protection from mosquitoes.

Technology that hampers mosquitoes’ host-seeking behavior, identified at UC Riverside in 2011, has led to the development of the world’s first product that blocks mosquitoes’ ability to efficiently detect carbon dioxide, their primary method of tracking human blood meals.

The initial research was performed in the laboratory of Anandasankar Ray, an associate professor of entomology, and was featured on the cover of the journal Nature. Ray’s lab identified volatile odor molecules that can impair, if not completely disrupt, mosquitoes’ carbon dioxide detection machinery.

The intellectual property was licensed to Olfactor Laboratories Inc., a company that grew around the technology, expanded the research, filed additional patents and developed related technologies that led to the mosquito-warding product.

Called the Kite Mosquito Patch, the product marks a significant advancement in the global fight against mosquito-borne diseases such as malaria, West Nile virus and dengue fever. The patch delivers mosquito-repelling compounds in a simple, affordable and scalable sticker that can be used by individuals in regions impacted by malaria and other mosquito-borne diseases.

“UCR is committed to strengthening and expanding its ties with industry partners,” said Michael Pazzani, the vice chancellor for research and economic development. “Olfactor Laboratories Inc. is a great example of how UCR innovations result in new industries, which, in turn, lead to the development of products impacting the lives of people around the globe.”

Simple and affordable, Kite is a colorful sticker, small enough to be worn virtually without notice. It disburses the non-toxic compounds that provide individuals with up to 48 hours of protection from mosquitoes. Estimated to cost a fraction of existing repellents, Kite is applied to clothing and can be used by people of all ages, including infants and pregnant mothers.

“I am very excited to see how Olfactor Labs has rapidly taken our initial discovery to a product that can have great value in the war against mosquitoes and disease,” Ray said. “I am most impressed that they have designed something affordable and convenient for use in Africa and around the world. I am rooting for this to become a game changer in lowering instance of malaria, dengue, filariasis and other dangerous diseases.”

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UCSF to host life science tech transfer summit

Event will be July 29-30.

Erik Lium, UC San Francisco

An international conference focusing on life science technology transfer will take place in late July at UC San Francisco’s Mission Bay campus. The 2013 Tech Transfer Summit (TTS), North America will run from July 29 to 30, focusing on early stage tech transfer, licensing, partnering and investment.

The conference is organized by TTS Ltd. and UCSF’s Office of Innovation, Technology & Alliances (ITA), which oversees technology transfers, entrepreneurship and innovative research alliances with bioscience commercial, nonprofit and government organizations. ITA is serving as the host partner, and BayBio and QB3 are also providing support for the event, whose speakers and attendees include a broad range of stakeholders in the life sciences and health care industries.

“UCSF is pleased to host the Tech Transfer Summit as part of our commitment to partnering with industry, government, private nonprofit and peer organizations to advance health worldwide, directly benefiting patients,” said ITA Assistant Vice Chancellor Erik Lium, Ph.D. Lium pointed to UCSF’s newly created Center for Digital Health Innovation (CDHI) as an example of the university’s focus on leading revolutions in health.

Early bird registration discounts are available through June 30. Registration and fee information is available at techtransfersummit.com/northamerica2013/registration. For more information, email terry.graham@ucsf.edu.



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UC Santa Cruz grad students launch cancer genomics company

Five3 Genomics offers genomics software and services for personalized cancer therapy.

The co-founders of Five3 Genomics are (from left) Charles Vaske, Steven Benz, and Zachary Sanborn, all former graduate students in the UC Santa Cruz Baskin School of Engineering.

The co-founders of Five3 Genomics, a new biotech company based in Santa Cruz, are former graduate students in the Baskin School of Engineering at UC Santa Cruz, where they helped develop innovative cancer genomics software.

Their company, which has signed a license agreement with UCSC, offers software and services for cancer researchers, pharmaceutical companies, and health care organizations. Its goal is to provide the data processing and analysis required for personalized cancer therapy, in which treatments are matched to the specific genetic aberrations found in an individual patient’s cancer cells.

“We’re working with academic collaborators to build out the platform and starting conversations with pharmaceutical companies and insurance companies,” said CEO Steve Benz, who completed his Ph.D. in bioinformatics this year. “It’s a great opportunity to be able to take this technology and commercialize it so that it can be used to help patients.”

In addition to Benz, the co-founders of Five3 Genomics include Chief Technical Officer Zachary Sanborn and Chief Scientific Officer Charles Vaske. All three of them worked as graduate students with UC Santa Cruz bioinformatics experts David Haussler and Joshua Stuart, who are doing pioneering work in the field of cancer genomics. Haussler, a professor of biomolecular engineering and Howard Hughes Medical Institute investigator, said that Benz, Sanborn, and Vaske were “brilliant grad students.”

“Working at UCSC they were exposed to the cutting edge in computational genomics,” Haussler said. “They played a key role in developing our cancer genomics program. They are pure self-starters, and developed the code to implement their ideas from the bottom up. The algorithms they developed represented new breakthroughs in our ability to interpret DNA sequence information obtained from cancer tumors. This area is poised to move from the academic realm into the clinical realm in the next few years. By spinning off a startup company, they have put themselves in an excellent position to play a key role in this transformation.”

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Targeting tooth decay

New mouthwash targeting harmful bacteria may render tooth decay a thing of the past.

Wenyuan Shi, UCLA

A new mouthwash developed by a microbiologist at the UCLA School of Dentistry is highly successful in targeting the harmful Streptococcus mutans bacteria that is the principal cause tooth decay and cavities.

In a recent clinical study, 12 subjects who rinsed just one time with the experimental mouthwash experienced a nearly complete elimination of the S. mutans bacteria over the entire four-day testing period. The findings from the small-scale study are published in the current edition of the international dental journal Caries Research.

Dental caries, commonly known as tooth decay or cavities, is one of the most common and costly infectious diseases in the United States, affecting more than 50 percent of children and the vast majority of adults aged 18 and older. Americans spend more than $70 billion each year on dental services, with the majority of that amount going toward the treatment of dental caries.

This new mouthwash is the product of nearly a decade of research conducted by Wenyuan Shi, chair of the oral biology section at the UCLA School of Dentistry. Shi developed a new antimicrobial technology called STAMP (specifically targeted anti-microbial peptides) with support from Colgate-Palmolive and from C3-Jian Inc., a company he founded around patent rights he developed at UCLA; the patents were exclusively licensed by UCLA to C3-Jian. The mouthwash uses a STAMP known as C16G2.

The human body is home to millions of different bacteria, some of which cause diseases such as dental caries but many of which are vital for optimum health. Most common broad-spectrum antibiotics, like conventional mouthwash, indiscriminately kill both benign and harmful pathogenic organisms and only do so for a 12-hour time period.

The overuse of broad-spectrum antibiotics can seriously disrupt the body’s normal ecological balance, rendering humans more susceptible to bacterial, yeast and parasitic infections.

Shi’s Sm STAMP C16G2 investigational drug, tested in the clinical study, acts as a sort of “smart bomb,” eliminating only the harmful bacteria and remaining effective for an extended period.

Based on the success of this limited clinical trial, C3-Jian Inc. has filed a New Investigational Drug application with the U.S. Food and Drug Administration, which is expected to begin more extensive clinical trials in March 2012. If the FDA ultimately approves Sm STAMP C16G2 for general use, it will be the first such anti–dental caries drug since fluoride was licensed nearly 60 years ago.

“With this new antimicrobial technology, we have the prospect of actually wiping out tooth decay in our lifetime,” said Shi, who noted that this work may lay the foundation for developing additional target-specific “smart bomb” antimicrobials to combat other diseases.

“The work conducted by Dr. Shi’s laboratory will help transform the concept of targeted antimicrobial therapy into a reality,” said Dr. No-Hee Park, dean of the UCLA School of Dentistry. “We are proud that UCLA will become known as the birthplace of this significant treatment innovation.”

The UCLA School of Dentistry is dedicated to improving the oral health of the people of California, the nation and the world through its teaching, research, patient care and public service initiatives. The school provides education and training programs that develop leaders in dental education, research, the profession and the community; conducts research programs that generate new knowledge, promote oral health and investigate the cause, prevention, diagnosis and treatment of oral disease in an individualized disease-prevention and management model; and delivers patient-centered oral health care to the community and the state.

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QB3 signs agreement to accelerate innovation

Partnership with Johnson & Johnson will fund proof-of-concept research that brings innovative science to market.

Regis Kelly

The California Institute for Quantitative Biosciences (QB3) has signed an agreement with the Johnson & Johnson Corporate Office of Science and Technology (COSAT) to fund University of California proof-of-concept research that brings innovative science to market.

This agreement, along with the Rogers Family Foundation of Oakland, helps fund QB3’s “Bridging-the-Gap” program, initiated in 2008 to support scientific projects that have high commercial potential led by faculty at UC San Francisco, UC Berkeley and UC Santa Cruz. These awards are intended to address the critical gap between federal funding for basic research and investments in product development. Each award typically provides $250,000 over two years for research.

Former award-winning projects include a prototype of an artificial kidney developed by Shuvo Roy, UCSF professor of bioengineering and therapeutic sciences, and an automated technique to improve prostate cancer screening, invented by Amy Herr, UC Berkeley associate professor of bioengineering.

The agreement establishes the JJSI-QB3 Awards as a component of the “Bridging-the-Gap” program and calls for annual awards over the next three years. QB3 will provide the awarded projects with services from the QB3 Startup-In-A-Box program, and potentially find space for them in the QB3 incubator.

“This agreement is an innovative academic-industry partnership model specifically focused on startup formation,” said Neena Kadaba, QB3’s director of industry alliances. “These awards are designed to provide a crucial amount of pre-commercial support to help would-be entrepreneurs address potential scientific risks in their technology before launching a company.”

The nine projects funded by QB3’s “Bridging-the-Gap” program to date already have resulted in five new companies and three licenses to existing firms.  The five companies formed in the past two years have raised over $9 million in private and public commercial funding.

”This agreement represents an important initiative for QB3 by providing additional opportunities for innovative researchers at our universities to create companies of value,” said Regis Kelly, director of QB3.

UCSF is a leading university dedicated to promoting health worldwide through advanced biomedical research, graduate-level education in the life sciences and health professions, and excellence in patient care.

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UCSF-Pfizer partnership yields projects aimed at clinical trials

Collaboration has resulted in five initial projects for therapies to treat cancer, other maladies.

Jeffrey Bluestone, UC San Francisco

An 11-month-old partnership between UC San Francisco and Pfizer Inc., aimed at rapidly moving new therapies into human clinical trials, has selected its first projects for funding and joint development. Teams from the university and Pfizer will work together on experimental therapies developed by UCSF scientists with a goal of testing them in people with five hard-to-treat, often deadly conditions, including lung and prostate cancer.

Three to five additional projects from university researchers will be selected after a second round of proposals, due Nov. 4, are evaluated. Details on the proposal process and how to submit the initial two- to three-page preproposal can be found at http://officeofresearch.ucsf.edu/ITA/CTI, or email ita@ucsf.edu with questions.

As part of the unique collaboration, Pfizer, the world’s largest drug company, will not only provide funding for the selected researchers, but has set up its own laboratory space next to UCSF’s Mission Bay campus. Scientists at the Pfizer lab, the Center for Therapeutic Innovation, will work directly with each of the UCSF teams.

“At UCSF, we are absolutely focused on finding new ways to turn the groundbreaking research of our scientists into therapies that benefit patients and the public,’’ said Jeffrey Bluestone, Ph.D., UCSF’s executive vice chancellor and provost. “Our work with Pfizer epitomizes our approach to building innovative, collaborative partnerships with industry.”

The Pfizer and UCSF researchers can visit each other’s labs, conduct experiments together and participate in joint team meetings, said Stephanie Robertson, Ph.D., who oversees the collaboration for the UCSF Office of Innovation, Technologies & Alliances with colleague Tuhin Sinha, Ph.D., alliance manager of the ITA.

“The proximity is key,” Robertson said. “People can literally walk across the street. That was a big reason for Pfizer locating right here.’’

As the cost of developing new drugs has skyrocketed — reaching $1.8 billion per approved drug, according to some recent research — drug companies have been searching for ways to lower the cost. Since they often spend years or months developing testing tools geared to the biology they’re interested in, the UCSF-Pfizer collaboration offers a way to jump-start that process by linking with academic researchers who know the biology and have already developed the tools.

“We are truly excited to work in this partnership with leading experts from UCSF to understand more about the mechanisms that drive diseases with high unmet medical need,” said Anthony Coyle, vice president and head of Pfizer’s Global Centers for Therapeutic Innovation. “By understanding the mechanisms underlying inflammatory diseases, cardiovascular disease and oncology, we can design better molecules to treat the right patients.”

Pfizer will have the right to commercialize the drugs and UCSF will earn milestone payments as the therapies advance through different stages of testing, as well as royalties from sales of approved therapies. This collaborative structure also provides the university the potential for a bigger return than it would normally receive when licensing out an early-stage technology.

“Best of all, it allows the scientists to be involved in turning research they’ve worked on for years into something that could actually be used to treat patients,” Robertson said.

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Lab spinoff receives grant for respiratory disease diagnostic device

Fast, inexpensive, disposable point-of-care technology also planned.

Bruce Cary

A spinoff of Los Alamos National Laboratory, Mesa Tech, has been awarded a $300,000 Phase I Small Business Innovation Research grant from the National Institutes of Health. The grant will allow Mesa Tech to develop an inexpensive, instrument-free, nucleic-acid testing device able to diagnose various respiratory diseases in record time.

The managers of Mesa Tech, who are currently developing a prototype, plan to initially target the global diseases surveillance market. They also envision applications in point-of-care diagnostics, particularly in poor areas of the world, said former LANL scientist Hong Cai, who cofounded Mesa Tech and is the principal investigator for the effort benefitting from the grant.

For point-of-care applications, Mesa Tech plans to develop an inexpensive handheld device about the size of a cell phone with a disposable cartridge, Cai said. In the case of a pandemic, such as SARS or avian influenza, the device also could be made disposable, she added.

Mesa Tech’s proposed instrument expands on technology developed by Cai and colleague Bruce Cary while they were researchers at LANL, which licensed the technology to the company. It also builds on previous work conducted by Mesa Tech under an $82,000 grant awarded under the American Recovery and Reinvestment Act. Using that grant, Mesa Tech began developing the consumable portion of the platform, an inexpensive nucleic-acid “dipstick” device capable of detecting and distinguishing multiple flu-like pathogens in under an hour, Cai said, explaining that current methods take anywhere from 60 to 90 minutes.

Mesa Tech’s research and development was also sped up by a 2009 Venture Acceleration Grant from Los Alamos National Security, LLC, the company that manages and operates LANL for the Department of Energy’s National Nuclear Security Administration. The VAF, which awards grants up to $100,000, was started in 2006 to fill a funding gap that slowed the commercialization of technologies by Northern New Mexico companies. Mesa Tech also participated successfully in LANL’s New Mexico Small Business Tech Assistance Program.

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