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Taking a break

Airport schedule board


16 April 2015. We will be traveling for the next two weeks, to recharge our batteries and see some of the world beyond beyond science and business. Regular posting will resume on 30 April.

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Plant Science Biotech Gets Genome Editing Technology

Dan Voytas

Dan Voytas (Cellectis Plant Sciences)

16 April 2015. Cellectis Plant Sciences, a biotechnology company in Minnesota developing higher quality crops through genetic engineering, licensed CRISPR genome editing technology from University of Minnesota. Financial details of the agreement between Cellectis and the university were not disclosed.

The technology licensed by Cellectis covers techniques known as CRISPR, short for clustered, regularly interspaced short palindromic repeats, applied to genomic engineering of plants. CRISPR is adapted from a natural process used by bacteria to protect against attack by viruses, where a protein that deactivates or replaces genes binds to targeted RNA molecules generated by the genome. The RNA molecules then guide the editing protein to specific genes needing changes.

University of Minnesota applied for a patent on the technology, which lists among its inventors Dan Voytas, a plant biology professor as well as chief scientist at Cellectis, a 5 year-old enterprise based in nearby New Brighton. The company is developing new crop varieties that increase their health benefits to consumers, using genetic engineering techniques.

The company is already working with existing genome editing techniques, including zinc finger nucleases, proteins of short-chain amino acids that make it possible to modify DNA sequences through corrections or insertions into those sequences. Another genomic-editing tool used by Cellectis is transcription activator-like effector nucleases or TALENs, programmable proteins that bind to DNA sequences and like CRISPR can address specific targets in the genome.

Cellectis operates mainly by licensing its technologies and collaborating with partners to develop commercial products. The company last year entered into two agreements with Bayer CropScience for gene-editing technologies. Cellectis and Bayer were already collaborating on development of genetically engineered potatoes, soybean, and canola plants.

A collaboration with SESVanderHave, a Belgian company producing sugar beet seeds, is applying genomic engineering to speed development of new sugar beet varieties. Another collaboration, with the European oil company Total, is developing genomic engineering techniques to produce new types of algae for renewable biofuel sources.

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Aduro Biotech Raises $108 million in IPO

Listeria bacteria

Listeria bacteria (

15 April 2015. Aduro Biotech Inc., a developer of immunotherapies to treat cancer, issued its initial public stock offering today that expects to net the company some $108 million, after issuing 7 million shares priced at $17.00. The Berkeley, California enterprise trades on the Nasdaq exchange under symbol ADRO. As of the Nasdaq closing bell at 4:00 pm ET today, Aduro shares were priced at $41.06.

Aduro’s immunotherapy technology creates therapeutic vaccines from an engineered form of listeria bacteria targeting specific tumor cells. Listeria, in its natural form, is a bacterium associated with food poisoning, but in the lab can be weakened and engineered to safely deliver antigens stimulating an immune response. Aduro calls its listeria-based cancer antigens live, attenuated, double-deleted or LADD agents, which the company says can work alone or with other cancer treatments, including chemotherapy.

An emerging technology at Aduro harnesses cyclic dinucleotides, naturally occurring molecules, found in both bacteria and mammals, but in mammals activate a signaling mechanism in immune-system cells. When stimulated, this pathway, known as Stimulator of interferon genes or Sting, induces production of cells and proteins that support and amplify the immune system.

The company says its engineered cyclic dinucleotides are more potent in stimulating the Sting pathway than the naturally-produced variety, to encourage a response in T cells, key immune system cells. In tests with lab animals, the company reports injections of its cyclic dinucleotides directly into tumors, sharply inhibited growth of melanoma, colon, and breast tumors, and protected against regrowth of those tumors as well as spreading of cancer cells.

In March, the pharmaceutical company Novartis licensed Aduro’s cyclic dinucleotide technology for commercialization outside the U.S. in a deal with a potential total value to Aduro of $750 million. Part of the deal includes Novartis taking a 2.7 percent equity stake in Aduro, with the option for expanding that stake later on.

Aduro has immunotherapies in early or intermediate-stage clinical trials being tested as treatments for pancreatic cancer, mesothelioma, and glioblastoma multiforme, an aggressive brain cancer.

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Trial Shows Multiple Sclerosis Drug Improves Optic Nerves

Nerve cell networks


15 April 2015. A clinical trial testing a new therapy for multiple sclerosis shows the drug improves the performance of optic nerves in patients with acute optic neuritis, a condition highly associated with multiple sclerosis. Researchers from the biotechnology company Biogen present their findings next week at the annual meeting of American Academy of Neurology in Washington, D.C.

Multiple sclerosis is an autoimmune condition where the immune system attacks the central nervous system and damages myelin, the fatty, protective substance around nerve fibers, as well as nerve cells themselves. Scar tissue from the damaged myelin, known as sclerosis, distorts the nerve signals sent to and from the brain and spinal cord, causing symptoms ranging from mild numbness to loss of vision or paralysis. Optic neuritis is inflammation of the optic nerve that transmits visual information from the eye to the brain, leading to pain and temporary vision loss, and is considered an indicator of multiple sclerosis.

Biogen, in Cambridge, Massachusetts, is developing an antibody treatment for multiple sclerosis and optic neuritis that aims to block a neurologic protein called Lingo-1 that normally supports myelin growth on nerve cells. In people with multiple sclerosis, however, Lingo-1 proteins appear to limit rather than encourage myelin growth when it binds to its receptors. Biogen’s antibody, code-named BIIB033, is designed to block the actions of Lingo-1, allowing for myelin to regrow.

The intermediate-stage clinical trial enrolled 82 adults with acute optic neuritis who were randomly assigned to receive 6 infusions of BIIB033 or a placebo every 4 weeks over a 20-week period. Participants were then assessed every four weeks up to 6 months, with a final evaluation 8 months after the last infusion. The study looked primarily at responsiveness of the optic nerves between participants’ damaged and normal eyes, as indicators of myelin restoration around nerve fibers, measured by ability to conduct electrical signals between the retina and the brain.

Results show participants receiving BIIB033 showed faster average optic nerve response of 7.6 milliseconds or 34 percent after 6 months compared to their counterparts receiving a placebo. After 8 months, average optic nerve response improved to 9.1 milliseconds, or 41 percent, compared to the placebo group. In addition, more than half (53%) of participants receiving BIIB033 showed optic nerve responses in their damaged eyes within 10 percent of their normal eyes, compared to about a quarter (26%) of those receiving the placebo.

The trial also measured changes in thickness of optic nerve cells and fibers using optical coherence tomography, similar to MRI and ultrasound imaging, but found little change in nerve cell or fiber thickness as a result of the treatments. The researchers point to the extensive damage to patients’ optic nerves as a probable cause. The study reported as well that treatments of BIIB033 were well tolerated with comparable rates and severity of adverse effects — generally fatigue, nausea, and sensations of burning or tingling — between patients receiving the test drug or the placebo.

Biogen has another intermediate-stage clinical trial underway testing BIIB033 among people with multiple sclerosis, and expects to report its first results next year.

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Smart Treadmill Adjusts to Running Speed Changes

Steven Devor and treadmill

Steven Devor, right, with automated treadmill during tests of the system (Ohio State University)

14 April 2015. Researchers at Ohio State University designed an automated treadmill that adjusts to changes in running speed by users, thus making the experience more like running outdoors. Steven Devor, professor of kinesiology, with former graduate student Cory Scheadler (now on the faculty at Northern Kentucky University), describe tests of a prototype model of the treadmill in an article published last week in the journal Medicine & Science in Sports & Exercise (paid subscription required).

Ohio State reports filing a patent application for the system, with the objective of its licensing and commercialization for eventual use in health clubs.

Devor says the objective of the system is to make the experience of using a treadmill more realistic and pleasant. “So many people call it the ‘dreadmill,’ notes Devor in a university statement. “It is boring and monotonous. An automated treadmill makes the experience much more natural and you can just run without thinking of what pace you want to set.”

The key to device is an inexpensive, off-the-shelf sonar range finder, positioned behind the runner and aimed between the shoulder blades. Sonar beams measure the distance from the range finder to the runner. A built-in microcontroller and processor read the measurements and adjust the speed of the treadmill’s belt.

When the runner is in the middle of the belt’s length, measured from front to back, the treadmill keeps the speed constant. But if the distance of the runner from the sonar device increases — moves closer to the front of the belt — the treadmill increases the speed of the belt accordingly. Likewise, if the device detects the runner moving toward the back of the belt, the belt speed slows until the runner reaches the mid-point.

The journal article reports on a test with 13 experienced endurance runners who ran three sets on the automated treadmill, and later on the same treadmill, but without the sonar and computerized controller. Devor and Sheadler measured peak work rates and maximum oxygen volume, and report that the automated treadmill allowed for more accurate measurements of maximum oxygen volume than the non-automated device.

More accuracy in measuring maximum oxygen volume, say the researchers, helps determine heart rate target zones that guide training regimens.

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Trial Testing App to Manage Diabetes, Foot Ulcers

Sugar app screen

Sugar app, main screen. Click on image for full-size view. (Worcester Polytechnic Institute)

14 April 2015. A clinical trial is getting under way testing a smartphone app to help people with diabetes manage their condition, and in particular assess chronic foot ulcers associated with diabetes. The application is the work of an information technology and biomedical engineering team at Worcester Polytechnic Institute in Massachusetts, that described the system in an article appearing earlier this year in IEEE Transactions Biomedical Engineering (paid subscription required).

Diabetes is a chronic disorder where the body cannot regulate the amount of glucose or sugar in the blood, affecting more than 29 million Americans, according to Centers for Disease Control and Prevention. Another 86 million people in the U.S., more than 1 in 3 adults, have prediabetes: high blood sugar levels, but not yet reaching full-fledged diabetes.

Among the effects of diabetes is reduced blood flow to the legs and feet, leading to nerve damage and reduced feeling in those regions, as well as slower healing of wounds. CDC says in 2008, some 70,000 Americans required amputation of a leg or foot because of complications from diabetes. In addition, says CDC, people with diabetes are 8 times more likely to lose a leg or foot than people without diabetes.

A team of computer and biomedical engineers at Worcester Tech, led by business IT professor Diane Strong, developed a system to help people with diabetes, especially those with foot ulcers, better manage their conditions. The core of the system is a smartphone app called Sugar designed to keep track of key factors related to diabetes that connects wirelessly to an individual’s blood glucose meter. The app also records the users weight and amount of exercise performed, and based on the data collected, returns messages encouraging different courses of action (e.g., get more exercise), or continued good practices.

Sugar — written so far only in an Android version — has a section devoted to recording the state of an individual’s foot ulcers. The app asks the user to take photos of foot sores with the smartphone’s camera that sends images to a program on a PC for analysis. The individual places his or her foot in a box that illuminates the foot and captures the image.

The analytical program converts the image to a bit-mapped format, then runs a series of algorithms to assess the size and healing status of the wound, which are tracked over time. Tests of the wound analysis module over 12 months, reported in the journal article, show the technique can accurately monitor the status of diabetic foot ulcers.

Peder Pedersen, professor of computer engineering at Worcester Tech who led development of the wound analysis section of the app says in a university statement, “For the first time, this system will give patients the ability to play an active role in their wound care.”

The clinical trial will be an early pilot test of Sugar. The trial at nearby University of Massachusetts Medical School plans to enroll 30 patients with foot ulcers at the medical center’s wound clinic. Participants will be randomly assigned to use Sugar for 6 weeks, covering a period of 3 visits to the clinic, or receive the usual standard of care. The trial will measure wound care progress and changes in healthy lifestyle over the test period.

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Gene Therapy Biotech Raises $60M in Venture Funds

Adeno-associated virus

Adeno-associated virus (

13 April 2015. Voyager Therapeutics, a biotechnology company developing gene therapies for central nervous system disorders, raised $60 million in its second venture financing round. Financing for the 1 year-old enterprise in Cambridge, Massachusetts was led by new investors Brookside Capital and Partner Fund Management, with participation by Wellington Management Company and Casdin Capital, also new investors, as well as other investors who were not disclosed.

Voyager is developing treatments for diseases of the central nervous system including Parkinson’s disease, amyotrophic lateral sclerosis (ALS), Friedreich’s ataxia, a rare inherited disease causing damage to the nervous system and movement problems, and Huntington’s disease, an inherited brain disorder that results in progressive loss of both mental faculties and physical control. The company is commercializing research by the company’s founders whose work covers technologies for harnessing viruses to deliver genetic therapeutics, and treatments using RNA interference to inhibit the expression of certain genes.

Voyager’s technology harnesses adeno-associated viruses to deliver healthy genetic material for expressing proteins missing from the mutated or damaged genes causing the disorder. Adeno-associated viruses are benign, naturally occurring microbes that can infect cells, but do not integrate with the cell’s genome or cause disease, and generate a mild immune response. One of the company’s founders is Guangping Gao, professor of microbiology at University of Massachusetts Medical School, whose work  involves the discovery, development, and use of adeno-associated viruses for gene therapy of inherited diseases.

In February 2015, Voyager announced a licensing deal with Genzyme, a biotechnology subsidiary of the drug maker Sanofi, that could earn Voyager as much $845 million. The agreement covers several Voyager gene therapies for Parkinson’s disease, Friedreich’s ataxia and Huntington’s disease, as well as other unspecified disorders of the central nervous system. Voyager is leading research and development of the therapies, working with Genzyme, a developer of treatments for multiple sclerosis and several rare diseases.

Genzyme has an option to license further rights to the therapies following early clinical trials. Voyager will continue to hold the U.S. rights to its lead treatments being developed for Parkinson’s disease and Friedreich’s ataxia, and will split profits in the U.S. with Genzyme from its Huntington’s disease therapy. Voyager’s ALS treatment is not included in the collaboration.

In addition to Guangping Gao, Voyager was founded by three other medical, genetics, and pharmacology faculty from University of Massachusetts, University of California in San Francisco, and Stanford University. First round financing of $45 million and early management for Voyager was provided by Third Rock Ventures, a life sciences venture capital company.

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Hat tip: Fortune/Term Sheet

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Janssen Licensing DNA Technology for Hep B Vaccine

Hepatitis B virus particles

Transmission electron micrograph of hepatitis B virus particles (Public Health Image Library, CDC)

13 April 2015. Janssen Pharmaceuticals, a division of Johnson & Johnson, is licensing an electronic DNA drug delivery technology from Ichor Medical Systems for vaccines to treat hepatitis B. Ichor, based in San Diego, expects to gain as much as $85 million in the deal.

Hepatitis B is a liver infection caused by a virus that can become a chronic condition, increasing the risk for life-threatening cirrhosis and liver cancer. The disease is spread through contact with blood and other bodily fluids, often from sexual contact or sharing hypodermic needles. World Health Organization calls hepatitis B a major global health problem, affecting some 240 million people with chronic conditions (lasting 6 months or more) and causing 780,000 deaths, mainly from cirrhosis.

Ichor Medical Systems is developing a drug delivery system using electrical impulses to enhance the effectiveness of DNA vaccines that introduce DNA plasmid molecules into cells. Those molecules give instructions to deposit antigens on cell surfaces for stimulating an immune response. The process, called electroporation, sends electrical impulses to create temporary pores in cell membranes, allowing for faster uptake of the DNA payload.

The company says its TriGrid drug delivery system overcomes efficiency problems with DNA vaccines using conventional injections, with a hand-held device that requires minimal user training. The system is being tested in clinical trials with vaccines for several types of cancer, HIV/AIDS, and other infectious diseases. Ichor says early-stage trials show TriGrid is safe and delivers vaccines that generate immune responses.

Under their agreement, Janssen is licensing the TriGrid technology, with the two companies collaborating on a hepatitis B vaccine. Janssen is responsible for some development costs and all commercialization costs in the deal, including expenses for manufacturing and distribution of TriGrid devices. Ichor will receive upfront, R&D, and milestone payments up to $85 million, as well as royalties on future product sales.

In November 2014, Ichor received a 5-year contract from Defense Advanced Research Projects Agency (DARPA) to develop a platform for producing vaccines that can work immediately with troops in the field, including those already exposed to pathogens. The contract, with a total value of $20.2 million, is supporting further development and clinical trials of the TriGrid system.

Disclosure: The author owns shares in Johnson & Johnson, parent company of Janssen Pharmaceuticals.

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Simple Avian Flu Test Designed for Poultry

Turkey (Agricultural Research Service, USDA)

(Agricultural Research Service, USDA)

10 April 2015. A bioengineering lab at University of Guelph in Ontario, Canada developed a quick, simple test for avian flu virus that infects poultry, including the type of virus now infecting turkeys in the U.S. and Canada. Guelph’s Bionano Lab led by engineering professor Suresh Neethirajan says a description of its device will appear in an upcoming issue of the journal Sensors.

Current testing techniques for avian flu viruses require taking blood samples from birds and sending them to remote labs for analysis. Just the analysis step takes 8 hours, says Neethirajan, and the entire process requires at least 2 days from sample to results.

When testing for avian flu outbreaks, particularly early on, getting results quickly is vital. “It’s critical to get out front of any outbreaks,” notes Neethirajanin in a university statement. “There are many strains, and we need to know the source of the flu. The identification of the strain determines what treatment options we should use.”

The Guelph test, returns results on the spot in about 2 to 3 minutes. The device designed by Neethirajanin and Longyan Chen, a postdoctoral researcher in the Bionano Lab, uses less blood from the birds than current techniques. In addition, the device tests the samples for characteristic surface proteins with a process using gold nanoparticles and quantum dots tuned to emit different colors. Quantum dots are pieces of semiconductor material that make it possible to measure and manipulate single electronic charges.

Not only can the Guelph test indicate the presence of avian flu virus, it can tell the strain of virus for determining the course of treatment. The researchers say their test can discriminate between H5N1 and H1N1 avian flu strains, and can be extended to indicate H5N2 strain causing the current outbreak. Of the 16 hemagglutinin subtypes of influenza — the “H” in virus codes — H5 is associated more with outbreaks in wild and domestic birds, while H1 can also affect humans.

The new avian flu test comes at a critical time for poultry producers in North America. The New York Times reports today that the deadly H5N2 virus, believed to originate in migrating wild birds, is causing turkey farmers Minnesota to euthanize some 525,000 birds, with quarantines occurring in the U.S. West and Midwest. That same virus hit British Columbia in December 2014 and January 2015, and this week was confirmed on a farm in southwestern Ontario by Guelph’s Animal Health Lab, which collaborated on the avian flu test.

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North Texas Campuses Researching Assistive Technologies

Ovidiu Daescu

Ovidiu Daescu (University of Texas, Dallas)

10 April 2015. Two University of Texas campuses, in Dallas and nearby Arlington, formed a research center to study software solutions for assistive technologies that benefit disabled and able-bodied people alike. The iPerform Center for Assistive Technologies to Enhance Human Performance, funded for five years by National Science Foundation, is also enlisting industry partners that would give them early access to the center’s work.

The joint project takes a broader view of assistive technologies, a term usually associated with solutions to help people with disabilities. The center plans to examine software for computer-assisted tools such as prosthetics for amputees, robotics to help people with failing eyesight, and smart wheelchairs. But the same techniques can be extended to solutions to help elderly people cope with day-to-day life and live securely in their own homes: e.g., better designed homes to prevent accidents, enhanced telecommunications, and application of games for rehabilitation.

Another extension of assistive technologies is for health care delivery, such as sensors to detect gestures or facial movements, along with speech recognition for personalized therapies. In addition, the same technologies can be applied to manufacturing to improve the efficiency of human-machine interactions and  increase worker safety.

The iPerform Center is led by computer science professors Ovidiu Daescu at UT-Dallas and Fillia Makedon at UT-Arlington. Daescu studies algorithms using computational geometry as well as biomedical computing applications. Makedon conducts research on human-centered computing, particularly in health care, manufacturing, and vocational safety. Several faculty from each campus plan to take part as co-investigators.

In a UT-Dallas statement Daescu notes “There currently is no research hub in the country for assistive technologies,” adding “The projects conducted within the center will help advance basic research in areas such as computer vision, machine learning, user interfaces, brain imaging, human robot interaction, human computer interaction, virtual reality and simulation.”

The joint project is funded under National Science Foundation’s Industry/University Cooperative Research Center program, where NSF makes a small initial seed grant, and the universities recruiting industry partners to fund the bulk of the center’s activity. UT-Dallas reports attracting three companies as industry partners — Bosch North America, Raytheon Co., and Texas Instruments — as well as National Institute of Standards and Technology, an agency of U.S. Department of Commerce.

An industry partnership, at an annual fee of $40,000, offers companies early first-hand access to the results of iPerform’s work and the opportunity to collaborate with faculty and graduate students at the two campuses. Industry partners also get to recommend research projects and vote on the center’s annual research agenda.

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