Regulatory approval of a gene therapy treatment in Europe could spark broader patient access to the technology.

By Susan Young on December 7, 2012

Last month, Europe’s Committee for Medicinal Products for Human Use approved a gene therapy for a rare genetic disease, the first time a Western regulatory agency has okayed such a treatment, though gene therapies have been approved in China.

The virus-mediated treatment, called Glybera, which is being developed by Amsterdam-based UniQure, introduces a normal version of a gene needed to properly break down fats in the blood. Though the condition is rare, patients with dysfunctional copies of this gene have dramatically increased levels of fat in their blood, which can lead to fatal inflammation of the pancreas. The genetic repair lowers blood fat concentration and reduces the frequency of pancreatitis, according to clinical trial data.

 The approval is just another sign that the field of gene therapy is quietly enjoying a resurgence after disastrous results in the late 1990s derailed testing of the technology. A number of companies are now testing various gene therapy products. For example, in 2010, Cambridge, Massachusetts-based Bluebird Bio reported it had successfully treated an 18-year-old patient for a rare blood disease (see “Gene Therapy Combats Hereditary Blood Disease”). Hemophilia, immune deficiencies, and blindness have all been treated with at least some benefit to the patients willing to try the experimental therapy. And large pharmaceutical companies such as Novartis,GlaxoSmithKline, and Baxter have taken on gene therapy projects in recent years.

“The fact that there were no approved gene therapy drugs up until recently probably made a subliminal impression on people that it wasn’t ready for prime time,” says Jean Bennett, a physician-scientist at the University and Pennsylvania and Perelman School of Medicine in Philadelphia. Bennett is involved in a clinical trial of gene therapy for an inherited form of blindness. “Now that there is one approved gene therapy product in the Western world, there is hope that there will be many others.”

Indeed, recent positive results have helped push the field beyond its early setbacks, perhaps the most public of which was the death of 18-year-old Jesse Gelsinger, who died in 1999 after receiving an experimental gene therapy for a genetic condition (see “A Death in Philadelphia”).  Other early gene therapy patients had severe or even deadly immune reactions.  Some patients who received gene therapy injections got cancer as a result of the treatment (see “The Glimmering Promise of Gene Therapy”). These setbacks raised the question of whether the treatment could be done safely.

The basic concept of gene therapy—replacing a defective gene or adding in a functional copy— is straightforward, but the trick is in the delivery. Gene therapies are often carried into a patient’s cells by viruses. Some of the early problems with the therapies were due to strong immune reaction to the transport viruses or cancer sparked by genomic changes induced by the viruses. But in recent years, researchers have found safer viruses and techniques for getting the replacement genes into a patient’s body.

 “The promise of gene therapy is phenomenal—a one-time treatment that will restore the natural function of the body,” says Jörn Aldag, CEO of UniQure. “Many people have pushed back and said it would never be approved, but now we are over the hump,” says Aldag.

UniQure has already built a manufacturing facility capable of commercial-scale production. Aldag expects Glybera to go on sale in the third quarter of 2013. In the meantime, the company must figure out what to charge for the treatment. The company is mulling the fact that treatments for other metabolic diseases can cost $200,000 per year or more, and that Glybera may be needed only once in a patient’s lifetime, says Aldag.  “How do you price a one-off treatment?”

(Reuters) – When he started looking around for start-ups in which to invest, Dan Scheinman noticed something: twenty-something entrepreneurs building Internet companies usually had a much easier time lining up early financing from venture capitalists compared to their forty- and fifty- something counterparts.

Age bias, increasingly acknowledged as a widespread phenomenon in Silicon Valley [nL1E8MFJM3], has created opportunity too.

“I was so excited you would not believe when I saw the pattern,” Scheinman, the former head of mergers and acquisitions at Cisco Systems (CSCO.O), recalls.

Many start-up investors claim they look for offbeat ideas put forth by gifted entrepreneurs, but in reality they often gravitate toward businesses that resemble past successes. In an era dominated by erstwhile start-ups — including Apple (AAPL.O), Microsoft (MSFT.O), Google (GOOG.O), and Facebook (FB.O) — that were founded by twenty-somethings and teenagers, a veritable cult of youth has many investors looking for the same.

But Scheinman, 49, claims good outcomes are possible — with less competition — by doing the opposite.

Scheinman has invested directly in eight companies since 2010, all with chief executive officers age 35 or older. On several occasions, he says he has heard the comment, “He doesn’t look like the traditional Internet CEO.”

The companies range from Think Big Analytics to free mobile video-calling service Tango to cloud-computing services company Arista Networks. Scheinman generally invests $50,000-$250,000 as part of a $1-$2 million funding round. He takes an active role, helping to line up other investors, generally taking a board seat, and providing strategy advice.

Of course, the true merits of Scheinman’s strategy will be clear only after a few years, when the companies in which he has invested have either failed, been acquired or gone through initial public offerings.

Little data exists on the average age of entrepreneurs that succeed in raising money.

But in a 2008 paper, “Education and Tech Entrepreneurship,” academics from Stanford, the National Bureau of Economic Research, and the Massachusetts Institute of Technology showed that the mean and median age of successful entrepreneurs was 39 at the time of company founding. The authors looked at companies with more than $1 million in sales and at least 20 employees.

Scheinman says he is pro-entrepreneur, no matter the age, but finds it easier to invest off the beaten track.

Another bonus: he manages to avoid the brazen ageism that he has often encountered among younger entrepreneurs. “I’d hear ‘Cisco is irrelevant, you’re old, you’re stupid,'” he says. “The level of hubris among the 25-year-olds, the ones who are getting funding, is very high.”

(Reporting By Sarah McBride; Editing by Jonathan Weber and Leslie Gevirtz)

(Reuters) – U.S. biotechnology group Amgen Inc has agreed to buy unlisted Decode Genetics, a pioneer in hunting down genes linked to disease, for $415 million in cash to boost its drive to develop better targeted drugs.

Founded in 1996, Decode blazed a trail in personal genomics by trawling Iceland’s unique genetic heritage, which has changed little since the Vikings arrived more than 1,000 years ago, to work out the links between gene variants and common diseases.

But it failed to live up to early expectations after going public in 2000 and filed for bankruptcy protection in 2009, weighed down by debts after 13 years of failing to make a profit, before re-emerging as a privately owned company.

Amgen and Decode said on Monday that the transaction did not require regulatory approval and was expected to close before the end of 2012.

As part of Amgen, Decode’s scientists will help in the task of ensuring that experimental medicines hit the right spot. Their know-how should allow Amgen to identify promising new avenues earlier and close down dead-ends more quickly.

“This fits perfectly with our objective to pursue rapid development of relevant molecules that reach the right disease targets, while avoiding investments in programs based on less well-validated targets,” Amgen Chief Executive Robert Bradway said.

PERSONALISED MEDICINE

UBS analysts said that the purchase, which will be funded by cash held offshore, was not surprising given that Amgen has key experimental drugs in its pipeline that were identified by human genetics work, including AMG 145 for heart disease and the bone drug romosozumab.

Understanding the genetic basis of disease has become increasingly important in drug discovery as the pharmaceutical industry shifts to developing personalized medicine that is suited for a patient’s particular genetic profile.

It is an area where Decode has extensive experience and its scientists have published prolifically on genetic mutations linked to a range of diseases including cancer, heart conditions and schizophrenia.

Commercially, however, the Reykjavik-based company has been far less successful. Its drug development programs stalled and its DNA tests for diseases have not brought in much cash.

The acquisition leaves Decode’s diagnostics business facing an uncertain future, with Amgen likely to evaluate this and other parts of the business after the deal closes.

Decode is currently owned by Saga Investments, a consortium including Polaris Venture Partners and ARCH Venture Partners, which bought it out of bankruptcy in 2010.

Polaris general partner Terry McGuire said his group had made a “substantial” return on its investment through the sale to Amgen, but he declined to give details or say if other large drug companies had been invited to bid for business.

CEO STAYS

Decode went public on a wave of euphoria about genetics after U.S. President Bill Clinton announced the completion of a working-draft DNA sequence of the human genome in 2000. Turning that gene promise into new drugs has proved harder and more time-consuming than initially hoped.

McGuire said that Decode’s founder and chief executive Kari Stefansson, a neurologist by training, would continue as president of the company after the Amgen takeover and would be a vice-president of research at the U.S. company.

Other genomics companies have fallen by the wayside over the years, though one Human Genome Sciences has managed to develop the first new drug for lupus in half a century with its partner GlaxoSmithKline (GSK).

GSK bought Human Genome Sciences for $3 billion this year, taking advantage of a dip in the U.S. biotech company’s shares. Two people familiar with the situation said in July that Amgen had also offered to buy the business for twice as much in 2010.

(Editing by David Goodman)

(Reuters) – Startup rocket company Space Exploration Technologies, which flies NASA cargo to the International Space Station, has landed its first launch contracts for the U.S. military, the company said on Wednesday.

The U.S. Air Force will pay $97 million for a Falcon 9 rocket to launch in 2014 the Deep Space Climate Observatory, a solar telescope that will be operated by NASA. It will also pay $165 million for a Falcon Heavy rocket for the military’s Space Test Program-2 satellite, which is expected to fly in 2015.

Both spacecraft will be launched from Space Exploration Technologies’ Cape Canaveral, Florida, site.

The company, also known as SpaceX, has been pursuing U.S. military launch business for years, hoping to break the monopoly held by United Launch Alliance, a partnership of Boeing and Lockheed Martin.

“SpaceX deeply appreciates and is honored by the vote of confidence shown by the Air Force in our Falcon launch vehicles,” SpaceX founder and chief executive Elon Musk said in a statement.

In addition to a 12-flight, $1.6 billion space station cargo delivery contract with NASA, SpaceX has a backlog of about 20 commercial and non-U.S. government satellites and payloads to fly on its Falcon family of rockets over the next five years.

The privately owned company plans to begin using a second launch site at Vandenberg Air Force Base in California in 2013.

SpaceX also is one of three companies hired by NASA to design a spaceship that can fly astronauts to the station, a $100 billion research laboratory that flies about 250 miles above Earth.

SpaceX’s Air Force contracts are part of a four-year, $900 million program that also includes Orbital Sciences Corp and Lockheed Martin, which is offering a new Athena rocket outside the United Launch Alliance partnership.

(Editing by Kevin Gray and Mohammad Zargham)

Ducted turbine promises significant advances but delivery remains to be seen.

When it comes to wind power, unconventional schemes to boost power and cut costs have never been wanting. Quiet Revolution offers a vertical axis turbine that looks more like a blender than a power generation device. WhalePower proposes mimicking on turbine blades the tubercles found on whale fins to increase power production.  Meanwhile Altaeros Energies is developing a flying donut to harness increased wind speeds found at higher elevations.

Earlier this month SheerWind, a wind power startup based in Chaska, Minnesota, added a new design, INVELOX, to the list. INVELOX, short for “increasing the velocity of wind” is a ducted turbine that looks a bit like a giant funnel sitting on top of an equally large periscope. The ductwork is designed to capture wind from any direction, increase its speed and concentrate the moving airflow before passing it through a relatively small turbine at ground level.

It’s an interesting concept that attempts to address a number of challenges facing conventional wind turbines.  The power produced by a wind turbine increases with the cube of the wind speed so any increase in speed could offer a significant power boost. Increasing wind speed also reduces the cut in speed, or the minimum wind speed required to begin generating power.  SheerWind officials say that by speeding up the wind they can boost power output by 280 percent and reduce the cut in speed by 80 percent to a wind speed of 2 miles per hour.

Another key advantage touted by SheerWind is smaller, ground-based turbines. A growing challenge for conventional wind turbine developers is the ability to build, transport, and mount giant turbine blades. By funneling the wind down to a smaller diameter duct, SheerWind is able to use a turbine with blades that are 80 percent smaller than those used in conventional turbines with similar output. Keeping the turbine at ground level will also significantly reduce installation and maintenance costs. All told, INVELOX should generate power for roughly one third less cost than conventional wind turbines, company officials say.

All of this sounds great but can INVELOX deliver? Researchers at The City College of New York have done fluid dynamics modeling of INVELOX and say the company’s claims stand up. (The researchers, mechanical engineers Yiannis Andreopoulos and Ali Sadegh and are listed as “technical advisors” by SheerWind but say they have not received compensation from the company for their analyses)

Martin Hansen, a wind energy expert at the Technical University of Denmark, disagrees. He says INVELOX will draw in and speed up the wind as claimed, but when the turbine is placed inside the ductwork it will create such high pressure that little additional air will be drawn into the device, making it a poor alternative to conventional turbine designs.

SheerWind completed it’s first large scale prototype earlier this month. CEO Daryoush Allaei says initial testing without the turbine resulted in a near doubling of wind speed passing through the device as predicted in prior modeling.  Allaei says they will now install the turbine and begin monitoring power output.

A microfluidic panel lets users push buttons on a flat interface.

By Jessica Leber on December 3, 2012

Touch screens are ubiquitous: in 2012, 1.2 billion were made for smartphones and 130 million for tablets, and they’re showing up in everything from game consoles to car navigation interfaces. But typing on them can be difficult. Tactus is trying to solve that problem. The company’s cofounder and chief technology officer, Micah Yairi, helped create a multi-layered panel that contains microchannels filled with a proprietary oil. When signaled by, say, a person launching a text-messaging app, an actuator pumps additional fluid into the channels, and buttons rise up from an elastomeric cover. The user then depresses the button slightly to trigger the touch screen and enter the letter or number. When typing is done, the panel reverts to a flat screen for finger-swiping within one second.

Tactus isn’t the only company recognizing a need for screens to offer tactile or so-called haptic feedback. Many phones already have rudimentary versions; tapping a certain button makes the whole phone buzz. Emerging designs include piezoelectric actuators that make the vibrations more localized. (Apple recently filed a patent on such technology.) And other companies, including Disney and a startup called Senseg, are using electrodes to issue minuscule shocks to your finger, simulating a rough texture.

Tactus’s approach, however, is the only one that allows users to orient their finger on the screen before actually depressing the key, or to rest their fingers on buttons without triggering them. Tactus is working to improve the panel’s appearance and create custom demonstrations for equipment manufacturers. One partner collaborating on prototypes is Touch Revolution, a division of the Taiwanese company TPK, one of the world’s largest touch-screen manufacturers.

Button geometries can be customized during the manufacturing process. In a  tablet or smartphone, software would probably change touch–sensitive areas of the display on the fly so that they’d align precisely with button shapes. This would prevent accidental keystrokes when fingers touch areas between raised buttons.

Tactus CEO and cofounder Craig Ciesla, who raised $6 million last year in venture investments, expects products to reach market in late 2013. “It’s really a design tool to give to manufacturers,” he says.

(Reuters) – Borrowing by small businesses rose in October, a report on Monday showed, as the central bank launched its latest round of monetary stimulus to encourage borrowing and spending.

The Thomson Reuters/PayNet Small Business Lending Index, which measures the overall volume of financing to small U.S. companies, rose to 107.5 from an upwardly revised 96.4 in September, PayNet said.

PayNet had initially reported the September figure as 94.1.

Borrowing was up 11 percent in October from a year earlier.

PayNet founder Bill Phelan said the rise was likely less a reaction to the Fed’s low-rate policy, which has been in place since December 2008, than a sense of growing optimism among smaller firms.

“They are seeing some profit-producing opportunities, and are wading in,” Phelan said in an interview. “The odds have shifted toward some optimism for next year.”

Small businesses are often responsible for the bulk of new job creation after recessions. The recent recession ended in 2009, but sluggish growth has meant weak job growth, and unemployment in October registered 7.9 percent, well above the 5.5 percent to 6 percent that many economists view as normal.

PayNet’s lending index typically correlates to economic growth one or two quarters in the future.

The Federal Reserve in mid-September unleashed a new round of bond buying to lower borrowing costs and spur businesses to spend and, eventually, to hire.

Separate PayNet data showed financial stress at near-record-low levels. Accounts overdue by 30 days fell to 1.2 percent of the total from 1.21 percent the previous month, and were near the 1.17 percent record reached earlier this year. Phelan said a “normal” rate of delinquency is 1.5 percent to 1.6 percent.

Longer-term delinquency rates also eased. Accounts behind 180 days or more, which are considered in default and unlikely to be paid, fell to 0.29 percent from 0.32 percent.

Accounts behind 90 days or more, or in severe delinquency, were unchanged at 0.24 percent.

PayNet collects real-time loan information, such as originations and delinquencies, from more than 250 leading U.S. lenders.

(Reporting by Ann Saphir in Chicago; Editing by Chizu Nomiyama)

Sixty-six new energy research projects were announced on Wednesday. Here are some interesting ones.

(Reuters) – Secretary of State Hillary Clinton on Thursday unveiled a game plan for achieving a global “AIDS-free generation,” committing the United States to rapidly scaling up medical interventions that are beating back what once was seen as an unconquerable disease.

Clinton, announcing the next stage of the decade-long U.S. fight against AIDS around the world, said advances in drug treatment and prevention strategies had brought the end of the epidemic within reach.

“HIV may well be with us into the future but the diseases that it causes need not be,” Clinton declared, saying it was possible to foresee a time when the number of people receiving treatment worldwide outpaces the number of new infections.

“That will be the tipping point. We will then get ahead of the epidemic, and an AIDS-free generation will be in sight,” she said.

The U.S. PEPFAR program, launched by former President George W. Bush in 2003, has been a catalyst for advancing HIV treatment, particularly in Africa. It now supports some 5.1 million people worldwide who are receiving anti-retroviral drugs.

The U.N. AIDS program said this month that ending the pandemic was now “entirely feasible” as it released an annual report showing that both deaths from AIDS and new infections with the HIV virus that causes it were falling.

Worldwide some 34 million people were living with HIV at the end of 2011, the UNAIDS report said. Deaths from AIDS fell to 1.7 million in 2011, down from a peak of 2.3 million in 2005 and from 1.8 million in 2010.

The number of people newly infected with HIV, which can be transmitted via blood and by semen during sex, is also falling. At 2.5 million, the number of new infections in 2011 was 20 percent lower than in 2001.

Despite the good news, not everyone is optimistic that the end of the epidemic is around the corner.

The ONE foundation, a charity founded by Irish rock star Bono, said this week that budget cuts in major donor countries were slowing efforts to reduce new infections and that a goal set last year by global leaders of turning the corner on AIDS by 2015 was now unlikely to be reached until 2022.

NO CHILD BORN WITH AIDS VIRUS

The new PEPFAR blueprint aims to accelerate the fight by scaling up both drug treatment and new strategies for combating the spread of AIDS including voluntary male circumcision, microbicide gels and interventions to stop pregnant women from passing the virus on to their unborn children.

“We can reach a point where virtually no children are born with the virus, and as these children become teenagers and adults they are at far lower risk of acquiring HIV than they are today,” Clinton said.

Clinton has lobbied hard to save U.S. overseas development spending in an era of increasingly tight budgets, and has stressed that Washington will not step back from the AIDS fight despite potential cuts to other programs.

The Obama administration has asked Congress for $6.4 billion for PEPFAR and other AIDS programs in 2013, down from $7.2 billion in 2012. Officials say economies of scale and savings from the purchase of generic drugs are making programs more efficient.

The U.S. government spent roughly $46 billion on AIDS, tuberculosis and malaria programs from 2003-2010.

U.S. Global AIDS Coordinator Eric Goosby said the new PEPFAR plan could likely lead to increased spending on anti-retroviral drugs as more people start the treatment earlier.

But he emphasized that the United States would not be paying for this alone, and said that U.S. efforts now would be to rally support from other donors as well as the recipient countries themselves.

“This administration has put a huge amount of political capital on this issue from day one. We remain committed. And we realize that we’re the major motor on the planet,” he said in an interview.

The new PEPFAR plan includes a greater emphasis on marginalized populations most at risk for HIV, including injecting drug users, sex workers and men who have sex with men, as well as turning over more responsibility for management and oversight to recipient countries.

Reaction to the new PEPFAR plan was broadly positive, although some advocates said the onus was now on Congress to fully fund the campaign.

“What we’ve needed for a while is an action plan that had caught up to the science of today. The blueprint takes us a step toward that,” said Chris Collins, director of public policy at the Foundation for AIDS Research.

(Reporting By Andrew Quinn; Editing by Xavier Briand)