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Science & Enterprise Featured in ACI Blog


3 July 2015. Because of the Independence Day holiday in the U.S., we’re taking today off. Blog posts will resume on Monday, 6 July.

We were looking for a suitable way to mark the 5th anniversary of Science & Enterprise — our first post while in stealth mode was 6 July 2010 — but the ACI Scholarly Blog Index took care of that. ACI Scholarly Blog Index compiles posts from 15,000 academic blogs, and since we’ve got “Science” in our name, they got us as well. Traci Hector of ACI talked to us this week and gave Science & Enterprise a nice write-up. Check it out.

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Engineered Mosquitoes Sharply Reduce Dengue Carriers

Mosquito biting a human

Aedes aegypti mosquito biting a human (U.S. Department of Agriculture)

2 July 2015. A variety of mosquito, engineered to produce offspring that die before maturity, was found to reduce the dengue mosquito population in a city in Brazil by 95 percent, well below the level needed to spread the disease. The team from the biotechnology company Oxitec Ltd. in Abingdon, U.K., with academic and business colleagues in the U.K. and Brazil, published its findings today in the journal PLoS Neglected Tropical Diseases.

Dengue is viral disease transmitted by infected mosquitoes (not person-to-person) that occurs most frequently in tropical urban regions of the world. World Health Organization estimates from 50 to 100 million dengue infections occur each year, with half of the world’s population at risk. WHO says the disease is now endemic in more than 100 countries, especially in Asia, Africa, the Americas, eastern Mediterranean, and Pacific islands.

The disease is a collection of four major types, but all types can cause high fever, severe headache, muscle and bone pain, and bleeding. Dengue hemorrhagic fever is a severe form of the disease that can cause respiratory problems, severe bleeding, and organ failure, and become fatal. There are no treatments for dengue other than caring for symptoms, and up to now, no vaccines to prevent its occurrence.

Dengue is spread by the Aedes aegypti mosquito. Oxitec uses genetic engineering techniques to insert a gene in males of the species that when released into the wild mate with females and produce offspring that die before becoming adults, and thus are prevented from having offspring. As a result the Aedes aegypti mosquito population in the area of the release is markedly reduced or eliminated.

Oxitec, a spin-off company from Oxford University, is currently testing its engineered mosquito, code-named OX513A, in field trials. The journal paper reported on one of those trials, in the Itaberaba suburb of Juazeiro city in Bahia State, Brazil, a region known to have a high incidence of dengue disease. The research team tested the engineered OX513A mosquito in an area of Itaberaba with 424 houses where 1,810 people reside. An adjacent area with 5,716 people living in 1,341 houses and a similar population density, but not exposed to the engineered mosquitoes, served as a comparison.

The team used ovitraps, devices that capture eggs from mosquitoes used for surveillance of the species, to track the number of mosquitoes in the test and comparison regions for a 1 year period, February 2011 to January 2012. The OX513A mosquitoes also had a fluorescent marker that appears in the eggs, which made it possible to follow the numbers engineered versus wild-type mosquitoes.

The results show the density of Aedes aegypti mosquitoes declined from 418 per hectare to 20 per hectare (1 hectare = 2.47 acres), a reduction of 95 percent, while densities in the adjacent region not receiving the engineered mosquitoes remained about the same over the period. A similar Oxitec study in Grand Cayman Island in 2010 found a similar, but not quite as dramatic reduction of 82 percent. The results of the later trial in Brazil, say the authors, suggest that the lower numbers of dengue-carrying mosquitoes could be below levels needed to spread the disease among human populations, according to accepted mathematical models of dengue transmission.

Other cities in Brazil are already beginning to use Oxitec’s engineered mosquitoes to control dengue. In May 2015, the Brazilian city of Piracicaba received approval from the nation’s biosafety authorities to begin a municipal dengue mosquito control program using Oxitec’s varieties.

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Commercial Space Launch Site Slated for New Zealand

Peter Beck with Electron rocket

Rocket Lab CEO Peter Beck with Electron rocket (Rocket Lab Ltd)

2 July 2015. Rocket Lab Ltd, a company aiming to make orbital space launches frequent and less costly, says it plans to build a launch site in New Zealand, with completion scheduled by the end of 2015. The company says test flights will begin soon after completing construction of the site, with commercial operations planned for later in 2016.

Rocket Lab, headquartered in Los Angeles but operating largely in New Zealand, plans to offer commercial launch services for small satellites in low earth orbit at less than $5 million a launch. Customers for its launch services are expected to be companies and organizations using satellites for weather reporting, crop monitoring, environmental tracking, maritime commerce, Internet transmissions, and GPS services.

The launch site will be located on the Kaitorete Spit, a strip of beach separating Lake Ellesmere (Te Waihora in Maori) from the Pacific Ocean on New Zealand’s south island. Rocket Lab says the site has minimal air and sea traffic nearby, which will help make possible more frequent launches, as many as 100 per year, one of the company’s key business objectives.

Rocket Lab is building two-stage launch rockets it calls Electron fueled by liquid oxygen and kerosene, with battery-powered electric motors for its pumping mechanisms. The engine, called Rutherford for the physicist Ernest Rutherford born in New Zealand, uses a propellent that is solid when stored, but becomes viscous under shear force. This capability, says the company, simplifies the design of the engine, offering the performance of solid fuels with the control of liquid fuels. In addition, all primary components in the Rutherford engine are produced with 3-D printing.

Electron is made with carbon composite materials that the company says provides for extra strength while reducing weight, compared to all-metal construction of conventional rockets. Fuel tanks on Electron are also made with carbon composites, which are compatible with the liquid oxygen. Rocket Lab claims the lower weight will make it possible to launch Electron with less fuel than used by a 737 flying between Los Angeles and San Francisco.

Rocket Lab was founded in 2007 by New Zealander Peter Beck. The company conducted some of its early research and development under contract to Defense Advanced Research Projects Agency (Darpa), and in partnership with NASA and DoD contractor L2 Aerospace. The company completed two venture funding rounds, securing early financing from Khosla Ventures, and joined in a second round in March 2015 by Bessemer Venture Partners and K1W1 investment fund. Aircraft manufacturer Lockheed Martin is also investing in the company.

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Drug Abusers, Smokers More Likely to Use Opioids Long-Term

Pills in blister packs

(PublicDomainPictures, Pixabay)

1 July 2015. A study by the Mayo Clinic finds people with a history of substance abuse and using tobacco are those most at risk for using opioid pain killers on a continuous basis. The team led by Michael Hooten, an anesthesiologist at Mayo Clinic in Rochester, Minnesota, published its findings in the July issue of the Mayo Clinic Proceedings.

Opioids work by reducing the intensity of pain signals to the brain, particularly regions of the brain controlling emotion, which reduces effects of the pain stimulus. Examples of leading opioid prescription pain medications are hydrocodone, oxycodon, morphine, and codeine.

Abuse of opioid pain killers is described by Centers for Disease Control and Prevention as a growing epidemic, fueled in part by growing numbers of prescriptions written for pain killing drugs. CDC reports that in 2012, physicians in the U.S. wrote 259 million prescriptions for pain killers, enough for one bottle of pills for every adult in the country. As of July 2014, according to the CDC, 46 people die each day in the U.S. from an overdose of prescription pain killers. The 10 states with the highest rates of prescriptions for pain killers, says CDC, are in the South.

Hooten and colleagues looked into the types of people receiving prescriptions for pain killers to identify patterns and characteristics of those most likely to end up using the drugs on a long-term basis. The Mayo Clinic team took advantage of a database of common electronic health records in its own region, the Rochester Epidemiology Project that compiles for researchers decades of medical records from tens of thousands of individuals in southeastern Minnesota.

The Mayo Clinic team drew a sample of 293 individuals who received a new prescription for opioid pain killers in 2009. The researchers then reviewed further use of opioids by people in the sample for 1 year, classifying their use of the drugs as short-term, episodic, or long-term.

The researchers found the 293 individuals received 515 prescription for pain killers in 2009, with about 1 in 5 (21%) showing a pattern of episodic use and 6 percent becoming long-term opioid users. The team also found people who use tobacco and those with a history of substance abuse were more highly correlated with episodic and long-term opioid prescribing pattern.

“From a patient perspective, it is important to recognize the potential risks associated with these medications,” says Hooten in a Mayo Clinic statement. “I encourage use of alternative methods to manage pain, including non-opioid analgesics or other non-medication approaches. That reduces or even eliminates the risk of these medications transitioning to another problem that was never intended.”

Hooten tells more about the study in the following video.

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Foundation Supporting ALS Pilot Clinical Trials

Brain illustration


1 July 2015. ALS Association is funding pilot studies with patients having amyotrophic lateral sclerosis to confirm biomarkers, or biochemical indicators, that improve responsiveness to therapies. The studies are being conducted by Neuraltus Pharmaceuticals Inc., a biopharmaceutical developer in Palo Alto, California, and University of Washington Medical Center in Seattle.

ALS, also known as Lou Gehrig’s disease, is a progressive neurological disease that attacks the nerve cells controlling voluntary muscles, such as those in the arms, legs, and face. In ALS, muscles gradually weaken and waste away, leading to individuals losing their strength and their ability to move their arms, legs, and body. When diaphragm and chest wall muscles fail, people lose the ability to breathe without ventilatory support, often leading to death from respiratory failure.

Neuraltus Pharmaceuticals develops treatments for neurodegenerative disorders, with its lead product code-named NP001 designed to regulate the inflammation of macrophages, white blood cells in the immune system that ingest dead or damaged cells. People with ALS are believed to have higher levels of inflamed macrophage activity that release factors in the central nervous system that damage motor neurons.

The new clinical trial aims to confirm the mechanism of NP001 in reducing biomarkers associated with inflammation: interleukin-18 and lipopolysaccharide. In an earlier trial, individuals with ALS taking higher doses of NP001 reported no progression of their symptoms during the 6 months they were taking the drug. People responding to NP001 had higher levels of interleukin-18 and lipopolysaccharide before the treatments that decreased after the treatments.

The new study is led by Robert Miller, director of Forbes Norris MDA/ALS Research Center at California Pacific Medical Center in San Francisco., who led the earlier trial. ALS Association is providing a grant of $1.5 million for the study, in addition to about $1.2 million from Neuraltus.

ALS Association with other funders are also supporting an 8-week study of the drug mexiletine, designed to treat irregular heart rhythms that acts by reducing hyperexcitability of motor neurons. The study, led by neuromuscular specialist Michael Weiss at University of Washington, will investigate the effect of mexiletine on hyperexcitability in the brain’s motor cortex, which controls movements. The trial is expected to measure markers of hyperexcitability with transcranial magnetic stimulation, a non-invasive way to determine hyperexcitability in the brain.

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Environmental Sensor Developer Reveals Collaborations

Industrial air pollution

(National Park Service, Wikimedia Commons)

30 June 2015. Aclima Inc., a designer of environmental monitoring sensors and networks, unveiled today collaborations with Google Inc., U.S. Environmental Protection Agency, and Lawrence Berkeley National Lab, among others. The San Francisco company, operating with little publicity for long as 5 years, did not disclose financial details from any of these partnerships.

Aclima Inc. develops sensor-based systems for measuring health and environmental air quality. The systems are designed to capture data from large numbers of air quality sensors inside buildings or for fine-grained local conditions in cities or regions. In addition, says Aclima, sensors can be deployed on vehicles running regular routes, such as buses or delivery vans, to capture air quality data in snapshots at various times of the day. The company then integrates the data collected in a cloud-based platform, with big-data analytical tools for users in business, public health, traffic management, and urban planning.

The collaboration with Google, operating for “several years,” according to an Aclima statement, monitors indoor air quality at 21 Google offices worldwide. Aclima says some 500 devices currently track temperature, humidity, noise, and light, as well as carbon dioxide and particulate matter in the office air, to help make decisions on workplace design for employee health and productivity.

Aclima is also partnering with EPA and Lawrence Berkeley National Lab on design of a miniaturized sensor to measure particulate matter in the air, of special concern for people with asthma and other respiratory problems. The sensors produced by the project, which includes participants at University of California in Berkeley and University of Illinois in Chicago, are expected to be small and inexpensive so they can be deployed in large numbers to capture outdoor air quality data.

A preview of the device can be found in an October 2013 paper published in the journal Sensors and Actuators A: Physical that outlines design of a sensor to measure airborne particulate matter. The paper, which says representatives from Aclima Inc. made contributions, has a micro electro mechanical system (MEMS) circuit that combines air and microfluidic components, which the authors — including those from EPA and Berkeley Lab — demonstrated with diesel exhaust and tobacco smoke.

In addition, EPA and Aclima in April 2013 entered into a 5-year research and development agreement to develop and integrate low-cost sensors into systems and networks deployed indoors and outdoors at stationary sites, and on mobile platforms. The sensors aim to detect the chemical composition of pollutants, including black carbon and particulate matter.

“Aclima has spent years in stealth creating a complete system to map environmental quality in an entirely new way,” says CEO Davida Herzyl in a company statement, “enabling us to see how our buildings, communities, and cities live and breathe.” According to Herzyl’s LinkedIn page, she co-founded the company in 2010.

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Juno, Celgene Partner in $1 Billion Immunotherapy Deal

Human T-cell lymphocyte

Scanning electron micrograph of a human T-cell lymphocyte (National Institute of Allergy and Infectious Diseases, NIH)

30 June 2015. Biotechnology company Juno Therapeutics Inc. and biopharmaceutical developer Celgene Corp. are collaborating on therapies that harness the immune system to treat cancer and autoimmune diseases over the next 10 years. The agreement is expected to bring Juno nearly $1 billion from Celgene in upfront payments and stock purchases.

Juno Therapeutics, headquartered in Seattle, is a two year-old spin-off enterprise from Fred Hutchinson Cancer Research Center in Seattle, Memorial Sloan-Kettering Cancer Center in New York, and Seattle Children’s Research Institute, with founding scientists from all three institutions. The company employs two technologies to encourage white blood cells in the immune system, known as T-cells, to target invading cancer cells without damaging healthy tissue. One process genetically modifies T-cells with molecules called chimeric antigen receptors to better identify and attack cancer cells on their own, without invoking immune-system signals that could target non-cancerous cells.

The second technique, known as T-cell receptors, also reprograms T-cells by adding molecules known as human leukocyte antigens that target corresponding proteins in tumors. The company harvests blood cells from cancer patients and separates their T cells for enrichment with the required genetic sequences in the cells’ DNA, then grown in the lab into dosage quantities for infusion back into the patient. In the body, the engineered T-cells multiply in the presence of target proteins and attack their corresponding tumor cells.

Under the deal Celgene — based in Summit, New Jersey — can commercialize two of Juno’s therapeutics in development to treat blood-related cancers, code-named CD19 and CD22. Both candidates are in early-stage clinical trials as treatments for acute lymphocytic leukemia, non-Hodgkin lymphoma, and other cancers of blood and lymph nodes of B-cells, white blood cells in the immune system that function separately from T-cells. Both CD19 and CD22 are based on Juno’s chimeric antigen receptor technology.

Celgene will be responsible for development and commercialization of these therapies worldwide, except for North America, where Juno retains responsibility. Celgene can also identify two other therapy candidates in Juno’s pipeline to promote worldwide, except for China, where the companies will share expenses and profits. Celgene can add a third Juno candidate to the mix, under further restrictions.

The agreement likewise gives Juno access to Celgene’s programs targeting T-cells. Celgene has experience with therapies that regulate the immune system it calls Imids, with two products — lenalidomide and pomalidomide — already approved in some jurisdictions. The deal calls for the companies to share costs and profits for developing new T-cell products, with 70 percent to Celgene and 30 percent to Juno. Celgene will lead their worldwide development and commercialization, except for the U.S. and some parts of Europe, where Juno has responsibility.

Under the agreement, Celgene provides Juno an initial payment of $150 million. In addition, Celgene is purchasing 9,137,672 Juno common stock shares at $93.00. The combination of upfront payment and equity purchases brings the total amount for Juno to $999.8 million. The deal gives Celgene the option of purchasing up to 30 percent of Juno’s shares under certain conditions, during the 10-year period.

Juno was founded in December 2013 and went public in December 2014, issuing 11 million shares at $24.00. As of 10:30 am today, its stock is trading on the Nasdaq exchange at about $55.00, giving the company a market capitalization of some $5 billion.

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Patient Registry Started for Rare Genetic Disorder

DNA illustration

(National Heart, Lung, and Blood Institute, NIH)

29 June 2015. A registry of families with individuals having Sjögren-Larsson syndrome aims to systematically collect data about families’ experiences with the disease and provide a source of participants for clinical trials of therapies. The registry is being formed by the National Organization for Rare Disorders and Sjögren-Larsson Syndrome Network Community.

Sjögren-Larsson Syndrome, or SLS, is a genetic disorder with symptoms affecting a number of organs and functions in the body. Infants with SLS tend to be born prematurely, often with skin that becomes dry, scaly, itchy, and discolored. Children with SLS often have developmental and neurological difficulties, characterized by leukoencephalopathy, affecting the myelin that insulates nerve cells and tissue in the brain. Developmental and neurological issues include delayed and impaired speech, vision problems, delayed motor skills, muscle stiffness, and seizures.

SLS was first diagnosed in Sweden, where about 1 in 250,000 are believed to have the condition. Prevalence of SLS outside Sweden is not known. The disease is caused by mutations in the ALDH3A2 gene — short for aldehyde dehydrogenase 3 family, member A2 — that make an enzyme called fatty aldehyde dehydrogenase, which breaks down fatty aldehyde molecules to fatty acids.

The mutations disrupt this normal process, where the build up of fats interfere with formation of membranes protecting cells against water loss, which promotes the ichthyosis or dry and scaly skin encountered by people with SLS. In the brain, the fat accumulation appears to interfere with development of myelin, which affects nerve cells and their transmissions, and thus affecting neurological development.

A biotechnology company, Aldeyra Therapeutics in Lexington, Massachusetts, is developing a topical cream code-named NS2 to treat ichthyosis by trapping the excess fatty aldehydes that build up in skin cells. In March 2015, the company began an intermediate-stage clinical trial at University of Nebraska medical center in Lincoln that aims to recruit 12 participants to test NS2 against a placebo. The company is recruiting patients through the SLS Network Community Facebook page.

The Facebook page describes the patient registry as a “a questionnaire that gathers information on history of diagnosis and treatment, family history, patient experiences, etc.” In addition, says the Facebook post, the registry form includes an option to receive information or be contacted about taking part in clinical trials.

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Novartis Acquires Pain Medication Developer in $200M Deal

Woman in pain

(o5com, Flickr)

29 June 2015. Global pharmaceutical company Novartis is acquiring Spinifex Pharmaceuticals Inc., a developer of new types of therapies for neuropathic pain. Spinifex reports Novartis is paying $200 million in cash for the company — based in Stamford, Connecticut and Melbourne, Australia — as well as undisclosed future milestone payments.

Spinifex Pharmaceuticals develops therapies for neuropathic pain, a complex and chronic condition caused by dysfunction or disorder in peripheral nerves, those found in motor or sensory functions, that feed into the central nervous system. The condition results from various causes — e.g., diabetes, chemotherapy for cancer, and postherpetic neuralgia from shingles — that make it difficult finding clear therapy targets, as well as translating results from preclinical tests with lab animals into clear targets for human trials.

In addition, most current drugs for neuropathic pain do not work consistently for some patients or are not well tolerated. Novartis cites data indicating 40 percent of patients with neuropathic pain do not respond to current initial treatments, and another 25 percent do not respond to follow-up treatment options.

Spinifex’s technology targets certain receptors for angiotensin II, a protein produced in the liver, then metabolized into hormones with various effects on the body, including constricting of blood vessels leading to high blood pressure and stimulation of some nerve cells, including those associated with pain. While compounds blocking receptors for some types of angiotensin II are able to relieve high blood pressure,  finding angiotensin II to control pain is proving more difficult.

Research by Maree Smith at University of Queensland in Australia succeeded in showing that a compounds can be developed that in lab animals block receptors for the variation of angiotensin II stimulating nerve cells and relieve symptoms of neuropathic and inflammatory pain. Smith’s findings are the basis of Spinfex’s technology that led to development of the company’s main product, code-named EMA401.

Later studies by Praveen Anand of Imperial College London show EMA401’s potential for blocking specific angiotensin II receptors to treat neuropathic pain.  Since these angiotensin II receptors are in the peripheral nervous system, EMA401 is not required to cross the blood-brain barrier and thus less likely to cause adverse side effects affecting the central nervous system, such as confusion or dizziness.

In February 2014, Anand and colleagues published results of a clinical trial testing EMA401 with 183 individuals having postherpetic neuralgia, a complication of shingles causing pain severe enough to interrupt sleep and appetite.  Participants were randomly assigned to take either EMA401 in capsule form or a placebo twice a day for 28 days. The results, reported in the journal The Lancet, show participants taking EMA401 reporting lower scores on a standard pain scale than their counterparts receiving a placebo. Participants also reported no serious adverse effects from EMA401.

Spinfex is already planning further clinical trials with postherpetic neuralgia as well as diabetic nerve pain. Novartis is expected to continue these studies with the aim of developing a new product line targeting neuropathic pain.

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Rare Disease Therapy Company Raises $60 Million in IPO

NASDAQ display

(bfishadow, WikimediaCommons)

26 June 2015. Catabasis Pharmaceuticals Inc., a biotechnology company creating treatments for rare diseases and cholesterol-related disorders, is raising $60 million in its initial public stock offering, trading on the Nasdaq exchange under the symbol CATB. The Cambridge, Massachusetts enterprise yesterday priced its 5 million shares at $12.00, and as of 4:00 pm today is trading at $13.41.

The company’s technology is based on research by Steven Shoelson at Harvard Medical School and Joslin Diabetes Center. Shoelson, a co-founder of the company in 2008, conducts research on the key role of inflammation linking obesity to insulin resistance, type 2 diabetes, and cardiovascular disease. He serves as a scientific adviser to Catabasis.

The Catabasis platform is called Safely Metabolized And Rationally Targeted or SMART linker technology that targets multiple points along particular disease pathways. Linkers, says the company, are small sub-molecular components that connect therapeutic molecules, but remain inactive in the bloodstream until they reach the target cells at various points along the pathway.

Once at their targets, the drug molecules are broken off into their active components where they can have an immediate therapeutic impact.  The company says this technology makes it possible to produce drugs that address their targets more precisely, with greater safety and fewer adverse effects.

Catabasis’s pipeline includes therapies for rare diseases including amyotrophic lateral sclerosis or ALS, Friedreich’s ataxia, an inherited disease causing damage to the nervous system and movement problems, and Duchenne muscular dystrophy, a genetic disorder resulting in muscle degeneration and weakness, primarily in the shoulders, arms, hips, and thighs of boys. Earlier this month, the company began recruiting participants for an early- and intermediate-stage clinical trial of its treatment for Duchenne muscular dystrophy code-named CAT-1004. A single therapy for ALS and Friedreich’s ataxia, code-named CAT-4001, is still in preclinical development.

Therapies for lipid disorders, particularly those related to cholesterol, are further along in the Catabasis pipeline. Its first candidate, code-named CAT-2003, addresses a pathway synthesizing fatty acids and in an intermediate-stage trial as a treatment for hypertriglyceridemia, a condition where triglyceride levels become elevated as a result of diabetes or obesity.

A second-generation therapy, targeting the same pathway, is being developed to treat hypercholesterolemia, an inherited form of high cholesterol, in people who do not respond to other types of treatments. That candidate, code-named CAT-2054, is in an early-stage clinical trial.

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Hat tip: Fortune/Term Sheet

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