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Stem Cells Shown Effective with Autism in Lab

Autism puzzle badge


26 January 2015. The biotechnology company BrainStorm Cell Therapeutics Inc. reports that tests in laboratory mice show its stem cell technology derived from bone marrow is effective in reducing behaviors associated with autism. The study was conducted by Daniel Offen, a neuroscientist at Tel Aviv University in Israel, but no peer-reviewed publication was cited by the company. Offen is a scientific advisor to BrainStorm and an inventor of the NurOwn technology licensed by BrainStorm.

BrainStorm Cell Therapeutics, in Hackensack, New Jersey and Petach Tikvah, Israel, develops therapies for neurodegenerative disorders — amyotrophic lateral sclerosis or ALS, also known as Lou Gehrig’s disease, Parkinson’s disease, and multiple sclerosis — derived from stem cells drawn out of a patient’s own bone marrow. The technology, brand-named NurOwn, transforms the extracted stem cells into cells supporting development of nerve cells.

These transformed stem cells, says the company, secrete proteins called neurotrophic factors that protect nerve cells, as well as encourage their growth and interactions with muscles. Because the original cells come from the patient, they have little risk of rejection by the immune system.

The company’s work with autism is a new initiative, begun just last year. Autism spectrum disorder is a collection of neurodevelopmental conditions, marked by communication difficulties and impaired social interaction, as well as repetitive and stereotyped patterns of behavior. At age 8, some 1 in 88 children have autism spectrum disorder, according to Centers for Disease Control and Prevention. Classic autism is considered the most severe form of the syndrome.

In this study, Offen and colleagues tested the ability of NurOwn stem cells to affect behaviors of a special type of lab mice bred to exhibit actions associated with autism, including lack of social interaction, cognitive rigidity, difficulty adapting to the environment, and high levels of repetitive self-grooming. A group of similar mice given the equivalent of a placebo served as comparisons.

The results, say the company, show a single treatment with the NurOwn cells enabled the treated mice to exhibit more social activity, better adaption to the environment, and less rigid or repetitive behaviors, compared to the mice receiving placebo treatments. In addition, mice treated with NurOwn cells exhibited behaviors more like normal mice strains than mice bred with induced autism-like behaviors.

One of the tests used a water maze to test cognitive rigidity, where the mice learned one maze pattern, then were confronted with a different maze. The mice treated with NurOwn cells, says BrainStorm, were able to learn and adapt to the new maze 60 percent faster than the mice receiving the placebo equivalent.

“We have seen in these mouse studies,” says BrainStorm’s CEO Tony Fiorino in a company statement, “an impressive consistency of response across many different behavioral measures, with a particularly strong result on cognitive rigidity, and impressively, the apparent normalization of some behaviors. With these results in hand, we are already putting next steps in place for this program.”

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XPrize Awards $5.25M for Lunar Technologies

Lunar surface


26 January 2015. A competition to develop new technologies for landing and robotic exploration of the moon awarded $5.25 million in 9 prizes to 5 private companies, as part of the Google Lunar XPrize challenge. The companies — from the U.S., Germany, Japan, and India — received the prizes for their design and development of landing, mobility, and imaging technologies.

Contestants submitted their solutions for equipment and software designed to overcome risks and obstacles faced by a spacecraft as it lands and explores the lunar surface.The Google Lunar Xprize challenge is a $30 million contest to develop lower-cost methods for robotic exploration of the moon, including deployment of robotic devices that can explore at least 500 meters and send back high-definition images and video. Last month, the deadline for completion of the mission was extended to the end of 2016.

The awards announced today, called Milestone Prizes, recognize specific parts of the competition posing more daunting obstacles to the overall goal. Among the prizes were those awarded for technologies designed to land a spacecraft on the moon, including attitude and orbiting control, guidance and navigation of the descent, avionics, propulsion, and devices for the actual touch-down on the lunar surface.

Another set of awards recognized systems for making the exploring robots mobile on the lunar surface, such as deployment mechanisms, actuators for moving and guiding the device, avionics for navigation, distance verification, and communications with other devices as well as the relay station back to Earth. A third set of prizes awarded achievements in optics and imaging, including detection electronics, image quality-control mechanisms, controls protecting cameras from extremes in temperature, and communications with other systems and devices.

The winning companies of milestone awards are:

Astrobotic Technology Inc. in Pittsburgh, Pennsylvania, a spin-off enterprise from Carnegie Mellon University

Hakuto, in Tokyo, led by Professor Kazuya Yoshida of the Department of Aerospace Engineering at Tohoku University

Moon Express, a private company based in Mountain View, California

Part Time Scientists, headquartered in Berlin, Germany, but with participants from several countries

Team Indus, from New Delhi, India

Astrobotic received $1.75 million for technologies in all three categories. Moon Express was awarded $1.25 million for its imaging and landing entries. Team Indus received $1 million for its landing contributions. Part Time Scientists was awarded $750,000 for its imaging and mobility technologies. Hakuto received $500,000 for its entries on mobility.

The Milestone Prizes are an optional part of the Google Lunar XPrize challenge that requires completion of all phases of the mission by 31 December 2016, with a launch no later than 31 December of this year. The competition is open to private teams, those with no more than 10 percent in government funding. The grand prize is $20 million, with a total purse of $30 million.

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Challenge Seeks Exosome Drug Delivery Technologies

Exosome illustration

Exosome illustration (

23 January 2015. A new challenge on InnoCentive is asking for systematic techniques for using exosomes, tiny components that cells secrete for specialized functions, as vehicles to deliver drugs. The challenge has a purse of $30,000 and a submission deadline of 17 February 2015.

InnoCentive in Waltham, Massachusetts conducts open-innovation, crowdsourcing competitions for corporate and organization sponsors. The sponsor, in this case, is not disclosed. Innocentive calls this type of competition a theoretical challenge that requires a written proposal. Free registration is required to see details of the competition.

Exosomes are vesicles, tiny — 40 to 150 nanometer — lipid-membrane containers in cells that gather up and secrete cytoplasm, the gel-like material outside the cell nucleus. While originally believed to carry out waste removal and other maintenance tasks, exosomes were shown in recent years to perform useful delivery functions carrying proteins and genetic material to other cells, and drawing increased attention from a range of biological disciplines.

As more of these delivery functions were revealed, researchers focused initially on opportunities provided by exosomes as biomarkers for diagnosing disease. More recently, researchers are investigating exosomes’ therapeutic potential, such as delivering antigens to trigger immune reactions from T-cells. A number of clinical trials are currently testing exosome therapies with humans, mainly for cancer.

The InnoCentive challenge is seeking a systematic approach for harnessing exosomes for therapeutic delivery. The challenge’s sponsor is requesting “a highly efficient and broadly applicable methodology for loading exosomes with the therapeutic agent,” which is not yet available. Through the challenge, the sponsor hopes to attract “one or more technologies that will facilitate exosome drug loading without significant disruption of the membrane components and properties of the exosome.”

Participants in the challenge should submit a written proposal, which will be reviewed by the sponsor, with specific prize amounts dependent on the sponsor’s evaluation. Winning entries will be required to transfer exclusive intellectual property rights to the sponsor in order to qualify for prizes.

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UC-Davis, National Labs to Train Science Entrepreneurs

Microfluidics device

Microfluidics devices — labs on chips or disks — are among the technologies with commercial use developed at national labs. (Jeff McMillan, Sandia National Lab.)

23 January 2015. Lawrence Livermore and Sandia National Laboratories are partnering with the Graduate School of Management at University of California in Davis to train scientists in entrepreneurial skills to take their discoveries to market. The pilot program, called Lab-Corps, is funded by a $350,000 grant from U.S. Department of Energy, parent agency of the national labs.

The university says more than 60 researchers attended a Lab-Corps kick-off meeting on Wednesday. Under the program, researcher teams at Livermore and Sandia will compete for two spots at training sessions in July and October 2015 at National Renewable Energy Laboratory in Golden, Colorado. Each team selected will receive $75,000 to design commercialization plans for their technologies. The competition is scheduled for late March.

UC-Davis plans to apply entrepreneur training experience developed in its Child Family Institute for Innovation and Entrepreneurship, part of the Graduate School of Management. The institute brings together UC-Davis students and faculty with entrepreneurs, business executives, and investors to build skills and networks needed to launch a new enterprise. The university says since 2006, the institute trained some 1,200 researchers and academics resulting in 50 new companies that attracted about $100 million in funding.

The partnership includes i-GATE, a technology business incubator in Livermore, California that helps early-stage technology entrepreneurs and works with the Livermore and Sandia labs, both located in Livermore. “What makes Lab-Corps unique,” says i-GATE executive director Brandon Cardwell in a university statement, “is the application of lean start-up principles, which are common in Silicon Valley, to lab technologies and the national lab environment.”

In August, UC-Davis reported the formation of 14 new technology companies based on research at the university in the previous fiscal year ending on 30 June, almost double the 8 start-ups established in the previous year. Most of the new companies, says the university, are located in northern California. Currently, 4 start-ups are operating at i-GATE, with another 4 companies spun-off and operating on their own.

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Mobile Heart Monitor Algorithm Approved in Europe

AliveECG app

AliveECG app on an Android device (AliveCor Inc.)

22 January 2015. An algorithm analyzing signals to detect atrial fibrillation from a heart monitor built into mobile devices received regulatory approval in Europe. AliveCor, a developer of heart monitoring systems for mobile devices, says the company received the Conformité Européene or CE mark for the algorithm that analyzes heart monitoring signals in its AliveECG App.

The CE Mark signifies approval to market regulated products such as medical devices in the European Union and associated countries, but the company says the AliveECG app is now available only in the U.K. and Ireland. The AliveECG app analyzes signals from AliveCor’s heart monitor app to detect atrial fibrillation. If the heart rhythm pattern suggests atrial fibrillation, the AliveECG app notifies the user. The data, stored online, can also be shared with physicians.

The AliveCor heart monitor works with Apple/iOS and Android mobile devices, and is intended for use by physicians for patients with known or suspected heart conditions, as well as health-conscious patients. In August 2014, the Food and Drug Administration approved the AliveECG algorithm for use in AliveCor systems sold in the U.S.

Atrial fibrillation, or AFib, is an irregular heartbeat that can lead to stroke or heart failure, and affects some 2 million people in the U.K. and Ireland, as well as 2.7 million Americans. With AFib, heart muscle contractions in the upper chambers beat irregularly instead of in a regular rhythm, which can cause blood to pool and lead to blood clots, including those that move to the brain and cause a stroke. Some 15 to 20 percent of stroke victims have this kind of irregular heartbeat, and left untreated, people with AFib are 4 to 5 times more likely to suffer a stroke.

While some people with AFib report symptoms, such as a racing heartbeat or light-headedness, many people with the condition experience no symptoms, making it difficult to detect. AliveCor, in San Francisco, says the AliveECG app reports the condition in real time for quick action by patients and physicians, which can be particularly important for people who do not experience symptoms.

Beginning in June 2014, AliveCor established heart monitoring centers at pharmacies in the U.K. and Ireland , where store customers could test for atrial fibrillation with the company’s heart monitor app. Community screenings of this kind are important, says the company, since about one-third of cases with AFib have not yet been diagnosed.

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Cancer Therapy Spin-Off Formed with $45M Funding


T-cell (NIAID/NIH)

22 January 2015. Autolus Ltd., a new enterprise developing engineered immune-system cells for treating cancer, is being formed in London, with £30 million ($45.4 million) in early financing. The company is founded and commercializing research by Martin Pule, a hematologist at University College London.

Pule, who serves as Autolus’s chief scientist, studies T-cells, white blood cells in the body’s immune system that hunt invading pathogens or help other T-cells protect against invaders. His research centers on harnessing a cancer patient’s own T-cells to battle cancer viruses, by reprogramming the T-cells to recognize and kill cancer cells, which the patient’s T-cells cannot do in their original state.

That reprogramming adds a new element to the surface of T-cells, a chimeric antigen receptor that enables T-cells to find identifying proteins on tumor cells, marking them for destruction. Chimeric antigen receptor-enabled T-cells, known as CAR T-cells, are then cultured and produced in the lab in large quantities and infused back into the patient.

Recent clinical trials are showing success with engineered CAR T-cells in treating cancer. An early-stage trial at Children’s Hospital of Philadelphia reports 90 percent of children and adult patients achieving full remission of their acute lymphoblastic leukemia. The technology is also attracting significant private investment in the past two months, with Amgen paying Kite Pharma $525 million to license each of Kite’s CAR T-cell programs, and Juno Therapeutics — a developer of CAR T-cell cancer treatments — raising $265 million in its IPO.

Autolus says its technology, based on Pule’s research, overcomes some early drawbacks of CAR T-cell treatments: limited applicability to solid-tumor cancers, serious adverse side effects from the immune reaction created by CAR T-cells, and toxicity to healthy tissues. When returned to patients, CAR T-cells proliferate in the body to fight cancer cells, but they also release large numbers of signaling cells called cytokines that can lead to high fever and sharp decline in blood pressure, according to National Cancer Institute.

The company is being launched by UCL Business, the university’s technology transfer unit, and Syncona Partners, a life sciences investment company in the U.K. Syncona Partners, the financier for Autolus, is a subsidiary of the Wellcome Trust, a leading science funding organization in the U.K. Founded in 2012, Syncona is an evergreen investor — one that provides revolving credit over a period of time — and currently supports five enterprises including Autolus.

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Hat tip: Fierce Biotech

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FDA Approves Novartis Psoriasis Drug

Interleukin-17 illustration

Interleukin-17 illustration (Boghog/Wikimedia Commons)

21 January 2015. U.S. Food and Drug Administration approved the biologic drug secukinumab for adults with moderate to severe plaque psoriasis, a common skin disorder. Secukinumab, an antibody designed specifically to address the condition, is marketed by the pharmaceutical company Novartis under the brand name Cosentyx.

Plaque psoriasis is the most common type of psoriasis, an autoimmune disorder, where the body’s immune system is tricked into attacking healthy cells, in this case resulting in inflammation and red, scaly patches of dead skin cells typically in the scalp, or near knees and elbows. According to National Psoriasis Foundation, some 125 million people worldwide have psoriasis, including  7.5 million Americans. Surveys cited by the foundation note that 6 in 10 people with psoriasis consider it to be a large problem in their lives, and people with moderate to severe forms of the disease — about a quarter of the total — experiencing a more negative impact on the quality of their lives.

FDA approved Cosentyx for psoriasis patients who qualify for systemic therapy — treatments that go through the blood stream — or treatments with ultraviolet light, or both. The drug is given as an injection under the skin.

Cosentyx works by binding to the interleukin-17A cytokine, a protein that activates inflammation. By binding to this protein, Cosentyx blocks interleukin-17A from binding to its receptor that triggers the inflammation, thus preventing the red, itchy scales on the skin.

FDA based its approval in part on results of 4 late-stage clinical trials with 2,403 patients having plaque psoriasis, and candidates for systemic or ultraviolet therapies, randomly chosen to receive either secukinumab or a placebo. The results show patients receiving the test drug experience clearer skin based on standard clinical assessments than those receiving the placebo. Novartis says the drug was tested in a total of 10 trials with nearly 4,000 patients.

Under the approval, Novartis will prepare a medications guide with warnings about potential effects of Cosentyx on the immune system, including reports of serious allergic reactions. The most common side-effects of Cosentyx are diarrhea and upper respiratory infections.

Novartis says Cosentyx is the first antibody-type drug approved by FDA to treat moderate to severe forms of psoriasis. On 19 January, the drug was approved by the European Commission for sale in Europe.

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Zymeworks, Celgene to Develop Double-Binding Antibodies

Currency dice (MD4 Group/Flickr)

(M4D Group/Flickr)

21 January 2015. Zymeworks Inc., a biotechnology company in Vancouver, Canada and pharmaceutical maker Celgene Inc. are developing biologic therapies using Zymeworks’ antibody technology. The deal makes Zymeworks eligible for up to $164 million from Celgene for each candidate developed under the partnership, as well as an unspecified initial payment and equity investment.

Under the agreement, Celgene, in Summit, New Jersey, and Zymeworks will design a series of therapies based on Zymeworks’ technology that creates engineered antibodies that bind to two targets or epitopes, the parts of antigens that generate an immune response. This design concept, says Zymeworks, enables its antibodies to address complex diseases that require more than one line of attack, as well as combining multiple treatments similar to a cocktail in one therapy. Despite the novel design, says the company, these bi-specific antibodies can be made with today’s monoclonal antibody processes.

Zymeworks has 9 candidate therapies in preclinical development for several types of solid tumor cancers, with 2 candidates for some types of breast and gastric cancer scheduled for new drug applications in 2016. The company also has a treatment for blood-related cancers scheduled for a 2016 new drug application. Celgene says it is taking part in some 300 clinical trials, including tests for a number of solid tumor and blood-related cancers.

The deal calls for Celgene and Zymeworks to jointly develop multiple bi-specific antibodies with the Zymeworks platform through preclinical stages. Celgene then will have the option of taking any of the candidates designed in the partnership through clinical trials and commercialization. Zymeworks will be eligible for up to $164 million in clinical, regulatory, and commercial milestone payments for each candidate advanced by Celgene. Zymeworks also  receives an unspecified initial payment as well as an equity stake from Celgene.

In December 2014, Zymeworks and Merck announced an extension of their 2011 research collaboration that gives Merck expanded access to the Zymeworks technology. As in the Celgene partnership, Zymeworks and Merck are collaborating on preclinical development, while Merck has responsibility for clinical trials and commercialization.

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University Spin-Off Develops Bone Repair Technology

Race horse Annagh Haven

Annagh Haven is a thoroughbred race horse who had her jaw repaired with a technology developed by a spin-off enterprise in Ireland. With Annagh Haven, l-r, is Ireland’s Minister for Jobs, Enterprise, and Innovation Damien English, Fergal O’Brien in whose lab the technology was developed, and Laurence Mulvany, owner of Annagh Haven.

20 January 2015. A materials science research center and university spin-off company in Ireland are developing a technology using natural materials to repair bones in people and animals. The bone-repair technology is a product of Ireland’s Advanced Materials and BioEngineering Research (Amber) center at Trinity College in Dublin and SurgaColl Technologies in Cork, a spin-off enterprise from Royal College of Surgeons in Ireland.

The technology, marketed as HydroxyColl, is a bone graft substitute that combines two main ingredients in bone, collagen and hydroxyapatite, built on a bioactive collagen scaffold for repairing defects in bone tissue. Collagen is an abundant human protein that gives strength and elasticity to skin and connective tissue, as well as bones. Hydroxyapatite is a ceramic mineral that gives bone tissue its hardness, and because of its bioactivity, can support bone regeneration and growth.

The HydroxyColl technology was developed in the lab of Fergal O’Brien, a bioengineering professor at Royal College of Surgeons and deputy director of Amber. The company, founded by O’Brien, says HydroxyColl directs regeneration of bone tissue and blood vessels around the collagen scaffold. As the bone heals, new bone tissue develops and replaces HydroxyColl, which is resorbed into the body. While mechanically strong, HydroxyColl is also porous and permeable, enabling the flow of cells and fluid needed to grow new bone.

HydroxyColl was tested on Annagh Haven, a 2 year-old thoroughbred race horse suffering from an aneurysmal cyst that caused her jaw to swell with a growing risk of fracture. The swollen jaw made it difficult for the horse to chew, raising fears of permanent harm or euthanasia, according to Science Foundation Ireland that funds Amber.

Annagh Haven underwent surgery at University College Dublin’s Veterinary Hospital, where the cyst was removed and replaced with sheets of HydroxyColl. The implanted HydroxyColl made it possible for new bone to replace the removed cyst, with normal bone shape and function restored in the horse’s jaw. Science Foundation Ireland says Annagh Haven since returned to a successful racing career.

HydroxyColl is expected to undergo human clinical trials later this year, followed by submission for regulatory approvals.

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Trial Testing Gene Therapy for Rare Eye Disorder

Jean Bennett

Jean Bennett (University of Pennsylvania)

20 January 2015. An early-stage clinical trial is underway testing a gene-repair therapy for choroideremia, a rare progressive genetic eye disorder that leads to blindness. The trial, conducted at Children’s Hospital of Philadelphia began enrolling its first patients, according to Spark Therapeutics, also of Philadelphia, the biotechnology company leading the study.

Choroideremia is associated with mutations in the CHM gene that block production of a protein needed to transport other key proteins to various cells in the body, including the retina. The body finds ways to compensate for most of the missing transported proteins, but not those going to the retina. As a result, the lack of these critical proteins leads to damaged cells in the retina and progressive loss of vision.

The CHM gene mutations occur on the X-chromosome, and since males have one X-chromosome, choroideremia is much more likely to occur in males than females. The condition generally begins with night blindness, followed by loss of peripheral vision, leading to tunnel-vision and complete loss of sight. The condition affects about 1 in 50,000 people and no treatment is available for it.

Spark Therapeutics develops gene therapies for inherited diseases based on research conducted at Children’s Hospital of Philadelphia and University of Pennsylvania. The company’s technology uses engineered adeno-associated viruses, benign microbes designed to deliver genetic material into the cells of patients with defective genes. The company began in October 2013, founded by four scientists from Children’s Hospital, including Jean Bennett, a University of Pennsylvania professor who studies the molecular genetics of inherited retinal disorders.

The clinical trial is testing the safety of two dose levels for Spark’s gene therapy code-named SPK-CHM for choroideremia. SPK-CHM is based on research by Bennett at University of Pennsylvania that studies the use of viruses to deliver genetic material that corrects for mutations, in this case in the CHM gene, to specific cells in the retina.

The trial aims to enroll 10 patients with choroideremia, divided between high and low doses of the therapy delivered to the patients’ retinas. The research team will look primarily safety and tolerability of the therapy, but also for changes in visual function and development of an immune response to the treatments.

Spark’s lead product, code-named SPK-RPE65, is an experimental treatment for other inherited retinal disorders — Leber’s congenital amaurosis and retinitis pigmentosa — that use the same adeno-associated virus delivery methods from Bennett’s lab as SPK-CHM. SPK-RPE65 is currently in late-stage clinical trials.

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