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Patents Awarded for Genetic Editing of CAR T-Cells

U.S. Patent and Trademark Office

U.S. Patent and Trademark Office in Alexandria, Virginia (A. Kotok)

13 February 2018. U.S. patents were awarded to a biotechnology company developing therapies using the genomic editing process known as Crispr to create immune system cells with proteins for attacking cancer cells. Patents numbered 9,855,297 and 9,890,393 were awarded on 2 January and today respectively, by the U.S. Patent and Trademark Office to 3 inventors, and assigned to Cellectis, based in New York and Paris.

Cellectis develops cancer treatments that harness the immune system by breaking down defenses tumors create to prevent the body’s immune system from fighting the disease. The company’s platform builds on recent developments that take T-cells, white blood cells from the immune system, and reprogram the cells through genetic engineering to find and kill cancer cells. The engineered T-cells become hunter cells, containing proteins known as chimeric antigen receptors that act like antibodies. These modified chimeric antigen receptor or CAR T-cells are infused into the patient, seeking out and binding to proteins associated with the cancer.

Most current CAR T-cell methods genetically engineer a patient’s own T-cells, then re-infuse the altered T-cells back into the individual. Cellectis’s process is designed to produce off-the-shelf CAR T-cell treatments, it calls Universal CAR T-cells, or UCARTs. These treatments use T-cells from healthy donors, rather than a patient’s own T-cells, then are genetically engineered to match the attributes of specific cancer types.

Both patents list as inventors Cellectis executives Philippe Duchateau, the company’s chief scientist, with CEO André Choulika, and early discovery director Laurent Poirot. And both patents cover the use of the genomic editing technique Crispr,  for clustered regularly interspaced short palindromic repeats. The technique is based on bacterial defense mechanisms that use RNA to identify and monitor precise locations in DNA. The actual editing of genomes with Crispr employs enzymes that cleave DNA strands at the desired points, with Crispr-associated protein 9, or Cas9, the enzyme used most often.

The earlier patent, dated 2 January, covers Crispr-Cas9 editing of T-cells, where messenger RNA guides Cas9 enzymes to at least one target in the T-cell genome. At that point Cas9 edits the T-cell genome at the target site, allowing for production of modified T-cells with the desired properties for immunotherapies. The 13 February patent also covers Crispr editing of T-cells, but allows for variations from single edits, including targeting 2 places in the DNA simultaneously, and delivering an inactive enzyme to the target site, then activating the edit inside the T-cell.

Cellectis applies its UCART technology mainly to blood-related cancers. In December 2017, the company reported initial findings from early-stage clinical trials of UCART cells in patients with B-cell acute lymphoblastic leukemia, conducted with other pharmaceutical companies. In the study of adults, 5 of 7 participants with relapsed or stubborn disease achieved molecular remission of the leukemia, where few cancer cells remain, after 28 days. A similar test with children show all 5 participants achieved molecular remission of their disease.

The clinical studies reported few safety problems with the UCART treatments, but that was not always the case. As reported in Science & Enterprise in September 2017, both trials were stopped by FDA when a patient died from cytokine-release syndrome, a complex of immune-system reactions to immunotherapies that worsened with complications, and who later did not respond to treatment. FDA lifted the clinical hold in November 2017, allowing the trials to continue.

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Engineered Virus Shows Promise in Some Brain Tumors

Brain tumor graphic

(MD Anderson Cancer Center)

13 February 2018. A clinical trial testing a common virus modified to generate a specific immune response dramatically reduced deadly brain tumors in a small group of patients and extended their survival for years, but most other patients had only limited success. Results of the trial by researchers at MD Anderson Cancer Center in Houston, part of the University of Texas system, appear in yesterday’s issue of Journal of Clinical Oncology (paid subscription required).

The team led by MD Anderson neurosurgeon Frederick Lang is seeking effective and reliable treatments for glioblastoma, an aggressive brain cancer that affects astrocyte or glial cells supporting neurons or nerve cells in the brain. Glioblastoma is often difficult to treat, where usually the best hope is to slow progression of the disease with radiation or chemotherapy. The cancer generally grows and spreads quickly, often resulting death within 15 months of diagnosis. American Association of Neurological Surgeons estimates glioblastoma occurs in 2 to 3 out of 100,000 adults per year, and accounts for 52 percent of all primary brain tumors.

Lang, along with neuro-oncology colleagues Juan Fueyo and Candelaria Gomez-Manzano, are developing a therapy that enlists the immune system to fight glioblastoma. Their treatment uses a genetically modified adenovirus, a type of virus benign to most people, but may cause the common cold and other viral diseases. In this case, the engineered adenovirus, code-named DNX-2401, is made to attack glioblastoma cells both directly and with the immune system.

“We designed DNX-2401 to specifically infect cancer cells,” says Fueyo in an MD Anderson statement, “replicate inside those cells to kill them, and spread from cell to cell in a destructive wave throughout the tumor.” The authors say preclinical studies show their approach could work with this type of brain cancer, with the clinical study its first test in humans.

The clinical trial recruited 37 patients with glioblastoma to test the safety and response of tumor cells to DNX-2401. Of the 37 participants, 25 received a single injection of DNX-2401 by catheter into their tumors at various dosage levels, while the other 12 patients received a DNX-2401 injection, followed by surgical removal of the tumor 2 weeks later to study the therapy’s mechanism of action in the brain. The primary measure of efficacy in the trial was reduction of tumor size.

The findings show dramatic outcomes for some patients, but only limited results for most. Of the 25 participants receiving DNX-2401 injections, 5 of the patients survived for more than 3 years. In addition, 3 of those patients showed 95 percent or more reduction in tumor size. “In the case of these long-term complete responders,” says Gomez-Manzano, “the virus breaks the tumor’s shield against immune response by killing cells, creating multiple antigen targets for the immune system. These tumors are then completely destroyed.”

After 3 years, however, beneficial effects of the treatments appear to wear off. The researchers say all 3 of the longer-term survivors experienced recurrences of cancer that proved fatal. Two of the survivors developed gliosarcoma, a different type of brain cancer. All 3 of the long-term survivors lived for nearly 5 years after their treatments.

Of the remaining patients, 18 experienced some reduction in their brain tumors, with a median overall survival time of 9.5 months. Among the 12 patients receiving DNX-2401 to study its activity in the brain, the authors say the virus replicates and spreads within the tumors as designed. In addition, the researchers report little toxicity and low-grade reactions to the treatments among 2 participants.

The trial’s results indicate the team has more work to expand the effectiveness of DNX-2401 to a larger percentage of glioblastoma patients. The researchers are studying the addition of new factors to the treatments to cover a broader range of people with the disease.

Fueyo and Gomez-Candelaria founded the company DNAtrix Inc. in Houston to take DNX-2401 to market. The company licenses the rights to the intellectual property owned by MD Anderson, with MD Anderson also holding an equity stake in the company. The company plans to further develop DNX-2401 as a treatment for solid tumor and blood-related cancers, both alone and with other therapies.

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Cardiac Augmented-Reality Hologram System in Development

Cardiac hologram

Cardiac hologram (SentiAR Inc.)

12 February 2018. A start-up company spun-off from Washington University in St. Louis was awarded a grant to advance an augmented reality system that displays an interactive hologram of a patient’s heart, to simplify cardiac procedures. SentiAR Inc. in St. Louis, formed just last year, is receiving the first installment of a planned $2.2 million in funding from National Heart, Lung, and Blood Institute, part of National Institutes of Health.

SentiAR’s software is designed to display a three-dimensional image of the patient’s heart portrayed on an augmented reality headset. Through the headset, the surgeon can view the heart, constructed with images from computed tomography or CT and MRI scans, as well as mapping data from catheters used in cardiac procedures. The first application of the system is arrhythmia, defined as any change from the normal sequence of electrical impulses regulating heart beats. Heart rhythm disorders prevent the heart from pumping adequate supplies of blood throughout the body, and can lead to blood clots, strokes, or damage to other organs.

The SentiAR system displays the heart hologram at eye level, without impairing the surgeon’s normal field of view during the procedure. The system, using Microsoft’s HoloLens platform, allows the patient’s hologram image to be manipulated with hand gestures, as well as expanded, rotated, entered, and measured as needed. The holograms can also be shared with other clinicians wearing headsets in the operating room.

SentiAR bases its technology on research conducted at Washington University by pediatric cardiologist Jennifer Silva and biomedical engineering professor Jonathan Silva, whose lab studies computational models to represent the various interacting scales of the heart’s electrical control system. The lab’s research aims to connect these scales and better understand their interactions to provide new therapeutic targets for patients with arrhythmia and other cardiac disorders.

The NIH grant, provided under the agency’s Small Business Innovation Research, or SBIR, program, is expected to deliver an augmented-reality/hologram system that can simplify ablation procedures to correct arrhythmias. These procedures use a catheter to deliver heat to destroy a small number of heart cells suspected of causing the irregular heart beat. While these procedures are relatively common — the company says some 1 million ablations occur each year — they’re still complex and can pose risks for patients. The SentiAR system aims to provide better real-time data about the patient’s heart to simplify these procedures for the surgeon.

SBIR funding for SentiAR is expected to total $2.2 million. The first installment of $223,532 covers the first 6 months of the project, through July 2018. Further funding, according to statement by SentiAR and BioGenerator, a biomedical business incubator in St. Louis supporting SentiAR, is based on achieving designated milestones. SentiAR already received $1.1 million in seed funds, co-led by BioGenerator that provided $400,000 of that amount.

“By improving the visualization of this information and empowering the physician with direct control of the model,” says co-founder and chief medical officer Jennifer Silva in the SentiAR-BioGenerator statement, “we will make these procedures both simpler and safer. Knowing that our peers – cardiologists and engineers – see the value of our solution and the potential impact it will have for both patients and practitioners is tremendous validation for SentiAR’s model.”


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Report – Industry Pain, Addiction Drug Efforts Flagging

Investment in cancer and pain drugs

(Biotechnology Innovation Organization)

12 February 2018. A report by a biotechnology industry organization says development efforts and financing for new drugs to treat pain and addiction are falling behind the health and safety needs of society. The Biotechnology Innovation Organization or BIO released its report, “The State of Innovation in Highly Prevalent Chronic Diseases: Pain and Addiction Therapeutics,” today.

BIO’s report is motivated in part by the continuing crisis in the U.S. in opioid addiction. The problem is intertwined with relief and management of pain, for which opioid drugs are usually prescribed. 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. Heroin is also considered an opioid.

The scale of the opioid abuse problem is huge. A report by the National Academies released in July 2017 says as of 2015, some 2 million Americans age 12 and older are addicted prescription opioid drugs, while 600,000 are addicted to heroin. Drug overdose, mainly by opioids, is now the leading cause of death from unintentional injury in the U.S. About 90 Americans die each day from opioid overdoses, and since 2011, the number of overdose deaths tripled from illicit opioids, such as heroin and fentanyl, a synthetic opioid.

The new report, prepared by 2 analysts employed by BIO, finds pharmaceutical and biotechnology companies have 220 new drugs for pain in their pipelines, with 125 of those treatments in clinical trials. The vast majority of these new therapies (87%) target non-opioid receptors to relieve pain, indicating they use different mechanisms from opioid drugs. By comparison, pharma and biotech companies have more than 2,600 new cancer therapies in development, of which 1,700 are in clinical trials.

Over the past 10 years, says the report, the industry mounted 142 clinical trials for pain drugs formulated to deter abuse, with 12 of those compounds later approved by FDA. Yet, during this same period, FDA approved only 2 drugs that use new biochemical mechanisms for pain. One reason for this low number of FDA approvals is the low success rate for new pain drugs in clinical trials. The report says only 2 percent of new chemical mechanisms for pain make it past early-stage clinical trials to FDA approval, compared to about 10 percent for the industry overall.

The outlook for drugs to treat addiction is even less promising, according to the report. For all types of substance abuse and addiction — alcohol, tobacco, opioids, and other stimulants — only 15 treatments are in pharma and biotech company pipelines beyond preclinical stages, with 5 of those 15 new therapies for alcohol abuse or smoking cessation.

The authors point to a lack of meaningful investment in pain and addiction drugs, particularly from venture capital companies, as part of the problem. From 2007 to 2016, companies with lead drug development programs of any kind dealing with pain received $1.5 billion in venture financing, with $576 million going specifically for new mechanisms to treat pain. In comparison, companies making drugs for cancer with new treatment mechanisms received $10.3 billion over this 10-year period.

For new addiction drugs, the report says, “Venture investment into U.S. companies with lead products in addiction has been virtually nonexistent over the past 10 years.” The authors could find only $16 million over the past 10 years invested in 2 companies with lead products treating addiction, and their products were for alcohol, not drug abuse.

The report notes that new incentives for development of pain and addiction drugs may be needed. The authors cite data that show even with a large potential market, more than 90 percent of current drugs prescribed for pain have a generic option. These and other challenges create uncertainties for investors, even for financing highly innovative drugs.

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Infographic – Software Security Risks at Record Level

Infographic: Software Security Risks at All Time High | Statista You You will find more infographics at Statista

10 February 2018. Science & Enterprise reports frequently on innovations that join information technology to science, while also pointing out some of the risks, particularly with security and privacy. This weekend’s infographic is from our friends at Statista who published a recent chart showing the almost constant rise in security risks since 2010 due to software, recorded by Hasso-Plattner-Institute in Germany. Last year’s total reached more than 11,000, with more than 3,300 considered high risks.

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Securing Your Small Business’s Profit

– Contributed content –

Counting money


10 February 2018. If you’re in the process of setting up your own small business, you’ll be well aware of how much hard work, time, and money you have to pour into every facet of your company. Before you even get things off the ground, you have to invest a whole lot into brand basics such as your company name, logo, and color scheme. Then you have to focus on developing your product concept and placing it in the market with market research. Once things are ready to go, you have to work on product manufacturing, marketing campaigns, and advertising.

It’s not surprising that you’re going to want to protect your assets and reputation from others who aren’t quite so willing to put in the time and effort necessary to make a success of a product and a brand. Unfortunately, if you don’t take the right steps, others will be able to directly copy your work and profit from it and there won’t be a whole lot that you can do. So, if you want to avoid this, read on for a comprehensive guide to protecting your company and secure the profits that are rightfully yours.

Understanding intellectual property

When people talk about “property”, thoughts of tangible items spring to mind. But you can indeed own something that isn’t physical. Intellectual property can be any creation of the mind, invention, symbol, name, image, or literary or artistic work that is intended to be used in commerce. Intellectual property rights allow you as a business owner to protect any of these things and ensure that others cannot profit from what is yours to profit from. In short, if you take the right protective measures, others cannot use your design.


So, where to start? Well, one of the first thing that you can do to secure your future profits is to engage with trademarking. A trademark is a word, phrase, symbol, or design that identifies and differentiates your design from anyone else’s. Things to consider trademarking include your brand name, slogan, and logo. This is a brilliant investment, as it does not have an expiry date. As long as you keep your trademarked asset in use, it is covered. So trademarking could, potentially, last forever! Bear in mind that it’s best to get this process started sooner rather than later, as it will take a year to eighteen months to get a trademark. It is by no means an overnight process. So get organized!


If your products are innovative and haven’t been seen on the market before, you may want to consider a patent. A patent is essentially a property right that ensures only you can sell this product on the market. All patents are a limited duration, but this will give you the opportunity to establish yourself as the market leader in the field and the original creator of the product.

Taking these steps to secure what is rightfully yours can help to reduce the amount of loss that you may see as a result of others immorally taking your ideas, design, and product designs. So ensure to carry them out as soon as possible!

The opinions expressed in this post are the contributor’s and not those of Science & Enterprise.

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Biotechs Collaborate on Immunotherapies in $1.2B Deal

Investment graphic

(Gerd Altmann, Pixabay)

9 February 2018. Two biotechnology companies developing cancer therapies harnessing the immune system are partnering on new treatments for solid tumor and blood-related cancers. The licensing and research deal with Seattle Genetics Inc. in Bothell, Washington could bring Pieris Pharmaceuticals Inc. in Boston as much as $1.2 billion if all aspects of the agreement go into effect.

Both Pieris Pharmaceuticals and Seattle Genetics develop therapies with engineered biologics. Pieris, founded in 2010, designs synthetic proteins with a platform the company calls anticalins, low molecular weight proteins similar to lipocalin proteins found in blood plasma. The company says its anticalins retain the basic natural structure of lipocalins, but add an addressable pocket that provides a tight binding site for therapeutic agents. In addition, says Pieris, anticalin therapies act against a wide variety of treatment targets, provide a durable delivery, and are shown to be safe for patients.

Pieris says it has a library of more than 100 million anticalin proteins, which can be further configured to meet specific therapeutic needs. In 2017, the company entered into licensing agreements with the pharmaceutical company Servier for cancer immunotherapies and AstraZeneca for biologics to treat asthma and other respiratory diseases.

Seattle Genetics creates mainly cancer therapies, with its main technology using antibody-drug conjugates. These therapies combine the precise targeting of engineered antibodies with cancer-killing agents like chemotherapies. Because of their more exact targeting, they concentrate the cell-killing effects on cancer cells, sparing healthy cells and tissue, unlike systemic chemotherapy drugs. Like Pieris, Seattle Genetics has a large library of antibody-drug conjugates with engineered antibodies addressing a wide range of cancer targets.

The collaboration plans to produce cancer treatments that combine features of the two respective technologies, resulting in antibody-anticalin fusion proteins, according to the companies. These bi-specific proteins, say Pieris and Seattle Genetics, will be engineered to bind highly-targeted antibodies to anticalin proteins, and in the process overcome some limitations of current cancer immunotherapies. Antibody-anticalin proteins, say the companies, will be designed to activate the patient’s immune system cells to attack tumors and the supporting tumor microenvironment.

Under the agreement, Seattle Genetics is paying Pieris an initial fee of $30 million, with the two companies developing an unspecified number of antibody-anticalin fusion protein candidates. Seattle Genetics will then have the option of choosing up to 3 of these candidates for further development. Seattle Genetics will fund and develop 2 of these candidates, with Pieris retaining the option to co-develop and commercialize the third therapy. Pieris will be eligible under the deal for milestone payments of up to $1.2 billion, as well as royalties on sales of products developed under the collaboration.

“This partnership,” says Dennis Benjamin, vice-president for research at Seattle Genetics in a joint statement, “leverages our cancer targets and tumor-specific antibodies to explore multiple novel bi-specific combinations, with the goal of developing targeted therapies that improve outcomes for people with cancer.”

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Business Investment – Wealth Rules Backed By Science

Jamie Cooper-Hohn

Jamie Cooper-Hohn at Children’s Investment Fund Foundation (By DFID – UK Department for International Development, CC BY 2.0 via Wikimedia Commons)

9 February 2018. Businesses want to make as much money as possible to keep the Grim Reaper at arm’s length. However, methods that exist today are dated. Take the ‘gut feel’ posse as an example. As alarming as it sounds, CEOs and CFOs make decisions not based on the numbers but how an investment makes them feel in their stomach. What if they miss lunch? Jokes aside, you don’t want to be the kind of person who doesn’t use the facts to their advantage as it’s a sure-fire way to fail. Here, then, are the wealth rules which are backed by the boffins.

Use modeling services

Businesses often take punts on products and services which are hard to imagine. From real estate to materials, you don’t get to see the goods before signing on the dotted line. If either doesn’t turn out to be the right standard, there could be damaging consequences for the whole company. As technology has improved, BIM services have taken the risk out of investing. Consider renting or even building a new office. With 3D imaging, it’s easy to see the potential pros and cons of the project. Any that adds undue risk can be dealt with, or you can pull out altogether. Your gut doesn’t give you this data.

Past performance = future results?

The answer to the equation is no. Just because an investment made money in the past doesn’t mean it will fair well forever. There are thousands of variables which impact whether an asset will plummet or skyrocket. Just keeping this probability equation in your mind should be enough to help you avoid risk. For those that want to go a step further, check out the fees and their rate of consistency. Mutual funds, for instance, are proven to be successful when the prices are low. It’s surprising but true.

Hug the status quo

Entrepreneurs have an innate desire for success where others have failed. After all, it’s the sign of a visionary and a captain of industry. At least, it’s the perception. In reality, world-class investors stick to their lane and don’t take unnecessary gambles. Warren Buffett heads Berkshire Hathaway, one the biggest hedge funds in the world. One of his rules is to settle for steady returns instead of taking big risks and returning huge losses. Remember that time is your biggest friend as, scientifically, investments mature with age.

Diversify your portfolio

A business should act in regards to the long-term, but putting your eggs in one basket is dangerous. Portfolios that don’t use asset allocation leave themselves open to mismanagement and bad luck, both of which are destructive. From a science point of view, mixing up investments means there are less risk and a higher chance of success. Then, should the real estate market collapse, your stocks and shares may come to the rescue. If both are impacted, a foray into gold and precious metals becomes more important than ever.

When you make business decisions, do you use science or your stomach?

Opinions expressed in this post are those of the contributor and not those of Science & Enterprise.

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Smart Thermometer Data Speed Flu Forecasting

Smart ear thermometer and app

Smart ear thermometer and app (Kinsa Inc.)

9 February 2018. Data collected from electronic thermometers are shown by researchers to track and report the spread of influenza cases in the U.S. faster than federal health authorities, as well as provide more up-to-date forecasts. A team from University of Iowa in Iowa City, working with the company Kinsa Inc. in San Francisco, report their findings in yesterday’s issue of the journal Clinical Infectious Diseases (paid subscription required).

The U.S. is currently in the midst of a widespread influenza outbreak that shows no immediate signs of diminishing. The flu is a contagious respiratory illness caused by infections from a family of viruses that result in symptoms ranging from mild to severe. Young children, older people, and pregnant women are often at greater risk of contracting the flu or suffering complications. According to the latest report at press time from Centers for Disease Control and Prevention, dated 27 January, nearly every state, U.S. territory, and District of Columbia, are reporting higher than normal (statistical baseline) numbers of flu cases.

The Iowa team of internist and epidemiologist Philip Polgreen and computer science postdoctoral researcher Aaron Miller are seeking faster and more detailed data on influenza outbreaks, to achieve as close to real-time reporting as possible. Faster status reports can aid public health authorities forecast trends in the spread of the disease, as well as alert hospitals, clinics and residents. Reports from the CDC, however, describe the status of outbreaks about 2 weeks after data collection, usually from medical records of patients at hospitals and clinics, well after symptoms become evident.

Polgreen and Miller enlisted the help of Kinsa Inc., a maker of electronic fever thermometers. Kinsa offers a line of thermometers for adults and children, cleared by FDA, that connect through hard-wire or Bluetooth to smartphones, with apps that record and track users’ temperatures. The company says more than 1 million people use Kinsa thermometers, recording more than 25,000 readings per day during the flu season.

Kinsa also enables its customers to share their readings for studies like those done by Polgreen and Miller, and for alerts to health authorities. The data are captured directly from the devices, says Kinsa, and thus they provide direct measures of current outbreaks, rather than relying on indirect indicators, such as Internet searches or social media posts. The data are encrypted when shared, with identifying information removed.

For the Iowa team, Kinsa provided data from more than 450,000 ear thermometers, collected from August 2105 to December 2017, totaling more than 8 million individual temperature readings. Polgreen and Miller constructed statistical models to track current suspected influenza outbreaks, and forecast trends in activity up to 3 weeks in advance. In addition, the researchers tracked the duration of fevers and their spread within households.

The authors found the data from connected thermometers correlate directly to CDC’s flu surveillance. The findings show data from Kinsa devices have a very high correlation with CDC’s reports — greater than 0.95, with 1.00 indicating a perfect match. In addition, high correlations also occur across regions and age groups. Moreover, the smart thermometer data could accurately forecast CDC’s findings up to 3 weeks in advance, as well as providing real-time reports.

“Our findings suggest that data from smart thermometers are a new source of information for accurately tracking influenza in advance of standard approaches,” says Polgreen in a university statement. “More advanced information regarding influenza activity can help alert health care professionals that influenza is circulating, help coordinate response efforts, and help anticipate clinic and hospital staffing needs, and increases in visits associated with high levels of influenza activity.”

The data also offer guidance for individual households. According to the authors, flu episodes last 3 to 6 days in individuals, with cases of the flu returning to the same person occurring more often during the flu season, increasing the risk of complications. “Because the device gives users the option of voluntarily reporting anonymized information on sex and age,” adds Miller, “we were able to estimate age-group specific incidence. Our results also suggest that influenza-like-illness moves more frequently from children to adults rather than vice-versa.”

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Neuro Therapy Company Spins-Off, Gains $20.8M Funding



8 February 2018. A company developing treatments for cognitive impairment and Alzheimer’s disease is starting-up in Belgium, with a technology and financial backing from local and global businesses and investors. The new company, Syndesi Therapeutics in Louvain-la-Neuve, is a spin-off enterprise from the Brussels-based pharmaceutical company UCB, staked to €17 million ($US 20.8 million) from investors in Belgium and elsewhere in Europe and the U.S.

Syndesi Therapeutics is creating therapies for cognitive impairment and Alzheimer’s disease with, according to the company, a different mechanism than other drugs so far. Alzheimer’s disease is a progressive neurodegenerative condition affecting growing numbers of older people worldwide. People with Alzheimer’s disease often have deposits of abnormal substances in spaces between brain cells, known as amyloid-beta proteins, as well as misfolded tangles of proteins inside brain cells known as tau.

Syndesi’s approach focuses on the synaptic vesicle protein SV2A, the same target as drugs for epilepsy developed by UCB. Because Alzheimer’s disease and cognitive impairment are not disorders being addressed by UCB, the company is spinning-off Syndesi, and providing an exclusive license to early drug candidates targeting SV2A, but with no anti-epilepsy properties.

SV2A regulates release of neurotransmitters, chemicals in the brain affecting signals among nerve cells, particularly across synapses, the connections between nerve cells. Synaptic vesicles are tiny containers in the synapse holding and releasing neurotransmitters. SV2A provides a binding site for drugs treating epilepsy, like those offered by UCB, but UCB also discovered related small molecule, or low molecular weight, drug candidates targeting SV2A that address synaptic dysfunctions related to cognitive impairment and Alzheimer’s. In addition, Syndesi says density of SV2A proteins is an indicator Alzheimer’s progression.

“Development of these small molecules that modulate the SV2A target in a distinct manner,” says Syndesi CEO Jonathan Savidge in a company statement, “represents an intriguing new approach for the treatment of cognitive deficits since they specifically target synaptic dysfunction, a hallmark of Alzheimer’s disease and other indications characterized by cognitive impairment.”

Syndesi is raising €17 million in its first venture funding round, with the financing led by venture investors Novo Seeds in Copenhagen and Fountain Healthcare Partners in Dublin and New York, both companies specializing in life science start-ups. Taking part in the funding are Johnson & Johnson Innovation, V-BIO Ventures, Walloon Investment Fund, and VIVES Louvain Technology Fund, which invests in spin-offs from l’Université catholique de Louvain (UCL) and start-ups in Belgium and neighboring countries. Proceeds from the financing are expected to support Syndesi through early proof-of-concept clinical trials.

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Disclosure: The author owns shares in Johnson & Johnson.

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