Category Archives: Gene Medicine

Dr. Emeran Mayer is a gastroenterologist who specialises in the communication between the brain, the gut and our environment. He is widely recognised as a pioneer of medical research into the brain, gut and microbe interactions and author of The Mind-Gut Connection: How the Hidden Conversation Within Our Bodies Impacts our Mood, Our Choices and Our Overall Health. (Our conversation has been condensed and edited for clarity.)

You discuss in your book, The Mind-Gut Connection, the journey you took at medical school to study the link between the brain and body in disease. What was the prevailing thought at the time and how were you going against it?

When I got into medical school, I was interested in studying the biological underpinnings of psychological constructs. When looking for a thesis advisor, I went from one professor to the next and they all said that mind gut connections cant be studied even though they knew it was important. After doing a rotation in gastroenterology at the Mass General Hospital at Harvard I was convinced that I wanted to study how the brain interacts with the digestive system. It was surprising to me how big the disconnect was between psychological and holistic concepts and traditional medicine at the time. Coventional Medicine selected people who were interested in mechanical, linear concepts of disease rather than an interest in health as a complex whole.

What is this mechanical, linear view of traditional medicine and why is it not sufficient in treating disease?

The linear viewpoint of the world around us represents the whole paradigm of the Western World. We go from point A to point B and dont look at the holistic context in which this interaction is happening. This model has been very successful in surgery in treating infectious diseases, where you identify a pathogen and develop an antibiotic to kill it. In reality, chronic diseases are not linear phenomena. Chronic diseases are dysregulations of a whole network, in which every organ in the body is interconnected, including the brain. For instance, if you are suffering from obesity, you also have a high risk of metabolic syndrome, of cardiovascular, liver and brain disease and cancer. This is no longer a linear phenomenon. You are looking at a paradigm of interconnectedness of every organ in the body. Chronic disease is a rearrangement in this global network that links every cell in our bodies together. Western medicine has not recognised that and as a result, nearly half of the US population are on chronic medicines. We are clearly not healing the disease. We are treating the systems and suppressing the issue.

How would you describe your approach to disease?

My view is as a systems biology approach. I look at the connections between every part of the body, down to every cell. For instance, if you look at genes, initially we thought that a single gene determines how old you are going to get. Now we know that it is a whole network of genes. Its the same with microbes. We have a hundred trillion microbes in our gut. We have to apply a systems approach of interconnectedness to understand and model it. In chronic disease, the systems go way beyond our bodies. The microbes in your gut live off the food systems from which you get your food, for instance the plants in the soil. And if you pursue this consistently, you all of a sudden see that we are all part of this gigantic interconnected system. I think what is happening with these viral epidemics is in some ways a systems phenomenon. We are attacking the normal system by cutting down the forests, encroaching on ecological niches of wild animals, and overcrowding in cities. And the way these diseases spread is not linear either. The whole world and system is affected.

Why did you decide to focus your research specifically on the connection between the brain and the gut?

From an evolutionary standpoint, our nervous system and our gut were always very closely connected, more so than any other organ. The first primitive organisms were simply a floating digestive tube with a nerve net around them. This basic architecture persisted through millions of years, and we still have a similar design in our gut. I think if you had to choose two organs that are the core of our being I would say it is the gut and the brain. The gut itself is not just a digestive tube, it is also the immune system, the nervous system, and the endocrine system. Contrary to popular belief, 95% of our bodys serotonin is stored in our gut. We interact with the world more through our gut than we do with our skin.

Why are there so many hormones such as serotonin stored in our gut?

We still dont know the full answer to this question. On the one side, the serotonin that is released in the gut communicates with the brain by stimulating the vagus nerve. Serotonin is only one molecule; tryptophan is broken down by the microbes and cells in the gut into many molecules, one of which is serotonin. The ratio of serotonin to some of the other tryptophan metabolites is influenced by microbial activities. The microbes can talk to some of the cells lining our gut and tell them to make more serotonin and release it onto the vagus nerve, which carries the signal to the brain. It also is released back into the gut and influences the behaviour of the microbes, so its going full circle. The molecule that allows microbes to take up the serotonin is the same molecule that acts when you take an antidepressant. We are still at the beginning of understanding the mechanisms of this. What we do know for now is that there is a major link between what we eat, what the microbes do with our food and how it affects brain function.

As well as the link between what we eat, our gut and brain function, you also discuss the effects of negative emotions such as stress on our body. What effect does this have in our gut?

Everybody now talks about the healthy diet and what it does to your gut and microbiome. Very few people talk about the fact that negative emotions in the brain can do almost the same damage as unhealthy food. Chronic stress decreases the diversity of your microbes, and changes the behaviour and leakiness of your gut. Your gut is a mirror image of your emotions. We dont listen and sense the effects of negative emotions or food on our gut on a daily basis. We tend to only notice the effects when we are in a lot of pain. People talk about the negative effects of the Western diet and obesity on cancer. You can imagine the combination of negative emotions and stress, plus the Western diet, will have twice the effect on increasing your risk of chronic disease. Typically in Western medicine, we dont pay too much attention to the mind but it is really key to realise this importance.

You also discuss how those with a positive attitude to life tend to heal faster from disease. What is the explanation behind this?

This comes back to the concept of our body as an interconnected network. How this network is constructed in our lives, determines how resilient it is to disease. This is shaped early in life, in the first two years of our lives for the microbiome and the first 18 years for the brain. The way this is programmed determines your resilience later in life. If framed in a positive way, such as with grit, enthusiasm, passion, compassion, and with the right diet, you are likely to be more resilient later in life. It offers an explanation for chronic diseases and longevity, determining how long we live and how healthy we are. As humans, we have this amazing ability to learn, our prefrontal cortex is incredibly plastic, providing our body with the opportunity to adapt and change to varying situations. I think our health ultimately all comes down to attitude and diet.

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What You Should Know About the Mind Gut Connection - Thrive Global

Scientists at the Icahn School of Medicine at Mount Sinai have discovered a new role for the brain chemical dopamine that is independent of classic neurotransmission. The new role appears to be critical to changes in gene expression related to chronic exposure to, or abuse of, cocaine, according to a study published Friday, April 10, in the journal Science.

Our study provides the first evidence of how dopamine can directly impact drug-induced gene expression abnormalities and subsequent relapse behavior, says Ian Maze, Ph.D., Associate Professor of Neuroscience, and Pharmacological Sciences, at the Icahn School of Medicine at Mount Sinai, and lead author of the study. Beyond transmission of signals between neurons in the brain, we have found that dopamine can be chemically attached to histone proteins, which causes cells to switch different genes on and off, affecting regions of the brain that are involved in motivation and reward behavior. This biochemical process significantly affects cocaine vulnerability and relapse when perturbed by drugs of abuse.

The study revolves around DNA and how it works to form each persons individual biological map. Each cell in the body contains two meters of DNA, the blueprint for all functions of all cells in the body. This DNA is wound around spools of histone proteins (proteins that package DNA in the nucleus of cells, and are heavily prone to chemical modifications that aid in the regulation of gene expression) into structures referred to as nucleosomes. When DNA encoding a specific gene is wound tightly within the spool, that gene is less likely to be expressed. When the gene is not wound as tightly, it is more likely to be expressed. This can affect many functions of a given cell.

Dopamine, known as the feel-good neurotransmitter, is a chemical that ferries information between neurons. The brain releases it when we eat food that we crave or while we have sex, contributing to feelings of pleasure and satisfaction as part of the natural reward system. This important neurochemical boosts mood, motivation, and attention, and helps regulate movement, learning, and emotional responses. Dopamine also enables us not only to see rewards but to take action to move toward them.

Vulnerability to relapse during periods of cocaine withdrawal is believed to result from functional rewiring of the brains reward circuitry, particularly within mid-brain regions, such as the ventral tegmental area (VTA). The research team discovered that a protein called transglutaminase 2 can directly attach dopamine molecules to histone proteins (a process called histone dopaminylation or H3Q5dop) which, in turn, affects the histone-DNA spool to enable environmentally regulated alterations in gene expression. They found that histone dopaminylation plays a critical role in fueling heightened vulnerability to relapse over a prolonged period of time. Specifically, accumulation of H3Q5dop in the VTA can, in effect, hijack the reward circuitry, making it difficult to distinguish between good and maladaptive behavior. The study found, however, that reducing H3Q5dop in rats programmed to undergo withdrawal from cocaine significantly reversed cocaine-mediated gene expression changes and reduced cocaine-seeking behavior.

The question that has always challenged neuroscientists is, what are the underlying molecular phenomena that drive increased vulnerability to drug relapse in people, says Ashley Lepack, Ph.D., a researcher in the Department of Neuroscience, The Friedman Brain Institute, in Dr. Mazes lab at Mount Sinai, and first author of the study. Our research is shedding valuable light on this area by identifying histone dopaminylation as a new, neurotransmission-independent role for dopamine that hasnt been implicated before in brain pathology.

We believe these findings represent a paradigm shift in how we think of dopamine, not just in the context of drug abuse, but also potentially in other reward-related behaviors and disorders, as well as in neurodegenerative diseases like Parkinsons, where dopamine neurons are dying, says Dr. Maze. In this case, the question becomes, could this neuronal death be due, in part, to aberrant dopaminylation of histone proteins?

In a study published last year, Dr. Maze and his team found that another neurotransmitter, serotonin, a chemical involved in the regulation of mood, acts in a similar way as dopamine on gene expression inside brain cells.

When we observed this unique signaling mechanism with serotonin, we decided to look at other neurotransmitters, particularly dopamine, and found that it could also undergo this type of chemical modification on the same histone protein, explains Dr. Maze.

Early-stage work with human post-mortem tissues has demonstrated to Dr. Maze that strong parallels may well exist, but that basic questions around biochemical function still remain before human trials can begin. From a therapeutic standpoint, weve started to identify from rodent models the mechanisms that can actually reverse aberrant and addictive behaviors, says Dr. Maze, and that knowledge could be vital to moving this novel research into the clinic.

Provided byThe Mount Sinai Hospital

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Researchers discover a novel role for dopamine that impacts gene expression related to cocaine abuse. - Brinkwire

Indians have some genetic advantage, but these are still early days to come to any conclusion

The fewer-than-expected cases positive to the novel coronavirus (SARS-CoV-2) in Indiahave spawned severaltheories, one of them being,Indians being immune to the virus.

It is theoretically possible as Indians are constantly exposed to microbes that keep the immune system primed, destroying pathogens attempting to attack. This is why children in very clean environments fall sick at the slightest exposure to a pathogen a concept known as the hygiene hypothesis.

Indians have some genetic advantage as well: Theyhave evolved to gain more genes that protect against viral infections, according to Rajalingam Raja, director of Immunogenetics and Transplantation Laboratory at the University of California in San Francisco, US. He said:

These genes enable natural killer (NK) cells, a type of white blood cells in our body that provide a first line of defense against viral infections

Two families of genes KIR genes and HLA genes playa part in this protective function. Indians have more KIR genes than the Chinese and caucasians. This could makeIndians more immune to the virus, according to Raja.

A similar mechanismprotectsbats from viruses like Ebola and SARS. Bats are immune since they have expanded gene families that enhance NK cell function, said Raja, who first wrote about NK cells in 2008 in journal Genes and Immunity.

This alone is, however, not enough to guide Indias strategy to fight the disease or even suggest that strict measures are not needed. A team of researchers from India and the US studied umbilical cord blood of children in the two countries and found differences. The findings were published in journal PLoS One in 2018.

We interpreted our study to suggest that Indian babies could be more susceptible to early-life infections if they had lower frequencies of certain immune cells, said Holden Maecker, director of the Human Immune Monitoring Center at Stanford University School of Medicine.

Persistent pathogen assault especially early in life is almost certainly detrimental. Thisisseen in the phenomenon of environmental enteropathy, where kids with poor sanitation and high enteric pathogen loadsdevelop malnutrition and stunting, Maecker said. But he agreed exposure to pathogens could equip the immune system better to fight new assaults like Zika or coronavirus, to an extent.

This was similar to the protective effect provided by latent tuberculosis. It was certainly possible that there was increasing resistance if not specific immunity to COVID-19 in certain genetic groups. It is difficult, however, to extrapolate this to all Indians who are a diverse collection of ethnic groups.

It is a balance and my guess is that its too soon to say where Indians as a whole will fall on this balance in terms of their sensitivity to COVID-19, hesaid.

Arguments about the Indian immune systemsare mostly speculative, according to Satyajit Rath of the National Institute of Immunology. He co-authored the 2018 study with Maecker.

I am yet to see any indication that COVID-19 will, in fact, turn out to be less prevalent and/or milder in India, since the epidemic is still in its early stages in the subcontinent, he said.

There are no publications, as of now, on the differential prevalence or outcome of COVID-19 among Indians and those of other ancestries worldwide.

Good nutrition, exercise and sleep can improve the immune system.

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COVID-19: Do Indians have higher immunity to novel coronavirus - Down To Earth Magazine

Alain Mrieux, founder of BioMrieux.

Latest update: April 1, 2020, at 4:47 pm ET.

Businesses around the world are shifting into overdrive to help battle the coronavirus, providing everything from rubber gloves and ventilators to diagnostic tools and, hopefully soon, vaccines. While the pandemic continues to wreak havoc, large corporations and small businesses are developing creative solutions to halt the spread of the virus.

Just as automakers famously shifted to make tanks and planes during World War II, todays global giants LVMH, Ford and GE to name a few are retooling their production lines to help make everything from hand sanitizers to respirators. On the medical front, there are more than three dozen COVID-19 vaccines under development, a smart move considering that two out of every three vaccines for infectious diseases fail, according to a study by the Massachusetts Institute of Technology.

Forbes will continue to update this list of private companies and how they are stepping up to fight the COVID-19 pandemic:

Testing:

Abbott Laboratories: Abbott Park, Illinois healthcare firm obtained emergency FDA authorization for its 5-minute coronavirus testing kit on March 27, with plans to start manufacturing 50,000 kits a day.

Alphabet: Through its healthcare arm Verily, Googles parent company launched a website where users can find nearby testing sites in four California counties.

Jeff Bezos.

Amazon: Jeff Bezos retail behemoth invested $20 million in the Amazon Web Services Diagnostic Initiative, which aims to speed up delivery of COVID-19 tests to the market.

BioMrieux: French biotech company, founded by billionaire Alain Mrieux,received emergency FDA approval for its subsidiarys new testing kit, which cuts testing times for the virus down to 45 minutes.

Carbon: California-based 3D printing unicorn backed by Russian tech investor Yuri Milner will soon be distributing testing swabs and face shields to hospitals in the Bay Area.

Cepheid: Sunnyvale, California molecular diagnostics company gained emergency FDA authorization for its new 45-minute COVID-19 testing kit.

Copan Diagnostics: Family-owned company located at the heart of Italys hard-hit Lombardy region makes diagnostic swabs for testing, airlifting 500,000 swabs to the U.S.

DiaSorin: Italian biotech company owned by billionaire Gustavo Denegri obtained emergency authorization from the FDA for its new 60-minute testing kit for COVID-19.

Mammoth Biosciences: South San Francisco-based biotech startup, founded by three 30 Under 30 alums, prototyped a rapid test by using the gene-editing tool Crispr to detect the disease.

Mesa Biotech: San Diego biotech business obtained FDA approval for its new 30-minute testing kit for COVID-19.

Puritan Medical Products: Maine-based diagnostic maker, one of the worlds largest makers of diagnostic swabs along with Italys Copan Diagnostics, is reportedly increasing production to make one million COVID-19 testing swabs a week.

Treatments:

AbbVie: North Chicago-based, publicly traded pharma firm is collaborating with authorities in the EU, the U.S. and China on experimental use of its HIV drug lopinavir/ritonavir to treat COVID-19.

AIM Immunotech: Florida-based pharmaceutical company announced on March 9 it would begin experimental testing of its chronic fatigue syndrome drug rintatolimod as a treatment for COVID-19 in Japan, at the National Institute of Infectious Diseases and the University of Tokyo.

Algernon Pharmaceuticals: Vancouver-based pharmaceutical firm is requesting FDA approval to begin trials of its chronic cough medication ifenprodil as a treatment for COVID-19.

AlloVir: Houston-based cell and gene therapy company is collaborating with Baylor College of Medicine to discover and develop T-cell therapies to fight COVID-19.

Apeiron Biologics: Vienna-based biotech firm started small-scale trials of its immunotherapy treatment on COVID-19 in China in February.

Ascletis: Hangzhou, China pharmaceutical company announced results of clinical trials of its antiviral drug danoprevir on COVID-19 patients in China; the small-scale study found that danoprevir combined with ritonavir is safe and well tolerated in all patients.

Bioxytran: Boston-based biotech outfit is developing a viral inhibitor to treat COVID-19.

Celltrion: South Korean healthcare firm is developing an antiviral treatment for COVID-19 as well as rapid self-testing kits that would provide results within fifteen to twenty minutes.

Cocrystal Pharma: Bothell, Washington pharma outfit is developing antivirals to treat COVID-19 using patents it recently acquired from the Kansas State University Research Foundation.

CytoDyn: Vancouver, Washington biotech firm announced preliminary results from three days of testing its antiviral drug leronlimab on COVID-19 patients in New York; the company stated in a press release that test results from the first four patients suggests immunological benefit within three days following treatment with leronlimab.

Eli Lilly: Indianapolis pharma company is partnering with Vancouver-based biotech outfit AbCellera to develop antibody-based treatments for COVID-19.

Emergent BioSolutions: Maryland drugmaker is developing treatments derived from the antibodies found in the blood of people who tested positive for the disease.

EUSA Pharma: British pharmaceutical firm initiated trials of its siltuximab antibody treatment on COVID-19 patients at the Papa Giovanni XXIII hospital in Bergamo, Italy; the company released initial data on April 1 showing that one third of patients experienced clinical improvement with reduced need for oxygen support and a further 43% saw their disease stabilise.

Fujifilm Toyama Chemical: Tokyo-based conglomerates flu drug favipiravir has shown promising results in early clinical trials on COVID-19 patients in China, and the company is investing $83 million in its biological manufacturing capabilities.

Gilead: The Californian biotech giant initiated clinical trials in March for its antiviral drug remdesivir on patients in the U.S.

Harbour BioMed: Cambridge, Massachusetts biomedical firm announced a collaboration with New Yorks Mount Sinai Health System to develop new human antibodies to treat COVID-19.

I-Mab Biopharma: Shanghai-based biopharma outfit announced it would begin clinical trials of its TJM2 antibody treatment on COVID-19 patients in the United States, with plans to expand to other countries affected by the pandemic.

ImmunoPrecise: Canadian life sciences company is teaming up with New York-based AI startup EVQLV Inc on researching antibody-based therapies and a vaccine for COVID-19.

Innovation Pharmaceuticals: Wakefield, Massachusetts biopharma firm is researching the use of its drug brilacidin part of a category of investigational new drugs called defensin mimetics, which could have antimicrobial effects as both a treatment and a vaccine for COVID-19, in separate efforts with a major U.S. university and with the Department of Health and Human Services.

ISR Immune System Regulation: Swedish immunotherapy firms subsidiary, ISR HBV, is conducting toxicological studies to determine whether its Immunolid ISR50 treatment could be used against COVID-19.

Kamada: Israeli pharmaceutical company is working on an antibody-based treatment for COVID-19 using the blood plasma of patients who recovered from the disease.

Mateon Therapeutics: Californian biopharma firm is testing a number of antiviral drugs as potential treatments for COVID-19 and is preparing to submit an application to the FDA in order to begin clinical trials on patients.

Merck KGaA: Darmstadt, Germany-based pharma multinational donated a supply of its multiple sclerosis drug interferon beta-1a to the French National Institute of Health and Medical Research in Paris for clinical trials on COVID-19 patients. The companys North American life sciences arm, MilliporeSigma, is supplying several vaccine efforts with reagents and other essential raw products for vaccine development.

Mesoblast: Australian medical firm is working with authorities in the U.S., Australia, China and Europe to evaluate the use of its remestemcel-L drug to treat COVID-19.

Mylan: Pennsylvania-based pharmaceutical firm restarted production of hydroxychloroquine, a drug used to fight lupus, malaria and arthritis, at its West Virginia factory; the drug is being tested as a treatment for COVID-19 in human trials in New York.

Pluristem Therapeutics: Haifa, Israel-based medical company is developing a cell-based therapy to treat COVID-19, announcing on March 30 it had dosed three Israeli patients under a compassionate use program, with plans to enroll more.

Leonard Schleifer.

Regeneron Pharmaceuticals: Westchester, New York biotech outfit, run by billionaires Leonard Schleifer and George Yancopoulos, is conducting clinical trials of its rheumatoid arthritis drug sarilumab, developed with French firm Sanofi, on patients in New York.

Roche: Swiss pharma titan, part-owned by billionaire Maja Oeri, is testing its arthritis drug tocilizumab to treat patients in China and received FDA approval to begin U.S. trials.

Roivant Sciences: Swiss pharma company is working with U.S. authorities to begin trials of its antibody treatment, gimsilumab, on COVID-19 patients.

Takeda: Japanese medical firm is working on hyperimmune therapy using blood plasma from previously infected patients.

Vir Biotechnology: The San Francisco-based firm is collaborating with Biogen and Chinese medical firm WuXi Biologics to manufacture antibodies that could treat the virus.

Vaccines:

AJ Vaccines: Danish vaccine developer is working on a COVID-19 vaccine that could hit the market in 2021.

Altimmune: The company is developing a novel intranasal vaccine for the coronavirus, making it one of three firms based in Gaithersburg, Maryland along with Emergent Biosolutions and Novavax thats working on treatments and vaccines for COVID-19.

Arcturus Therapeutics: San Diego-based vaccine maker is developing a COVID-19 vaccine with researchers at the Duke-National University of Singapore medical school in Singapore.

Biocad: Russian drug developer is researching a COVID-19 vaccine, with animal trials scheduled for late April.

Thomas and Andreas Struengmann.

BioNTech: German biotech firm backed by billionaire twins Thomas and Andreas Struengmann is working to develop a coronavirus vaccine in partnership with Pfizer and Fosun Pharma, chaired by billionaire Guo Guangchang.

CanSino Biologics: Tianjin, China-based pharma company isstarting clinical trials for its COVID-19 vaccine, using the vaccine technology deployed to develop the Ebola vaccine.

Codagenix: Melville, New York biotech firm is teaming up with the Serum Institute of India to develop a live-attenuated COVID-19 vaccine, which uses a live but weakened form of the virus.

Dietmar Hopp.

CureVac: German firm, funded by billionaire Dietmar Hopp and the Bill and Melinda Gates Foundation, received $87 million from the European Commission to scale up development of its coronavirus vaccine.

Dyadic: Jupiter, Florida company is collaborating with the Israel Institute for Biological Research on both treatment and a vaccine against COVID-19, using the firms gene expression platform.

Dynavax: Emeryville, California vaccine maker is working with the Coalition for Epidemic Preparedness Innovations (CEPI) and the University of Queensland to develop a COVID-19 vaccine.

EpiVax: Providence-based immunology firm is working with the University of Georgia and Miramar, Florida biotech outfit Generex on separate COVID-19 vaccine efforts.

ExpreS2ion: Danish biotech company received a grant of nearly $1 million from the European Union to develop a vaccine for COVID-19.

GeoVax: Atlanta-based medical company is collaborating with Wuhan-based BioVax to jointly produce a COVID-19 vaccine.

GlaxoSmithKline: British pharma titan is partnering with CEPI and Chengdu, China-based Clover Pharmaceuticals to use its pandemic vaccine adjuvant platform which boosts the immune response in patients receiving a shot to speed up development of COVID-19 vaccines.

Greffex: Houston-based genetic engineering firm is preparing to begin animal trials for its COVID-19 vaccine.

Heat Biologics: North Carolina biopharma company is developing a COVID-19 vaccine with the University of Miami.

iBio: Newark, Delaware biotech upstart is collaborating with Beijing-based CC-Pharming on the rapid development of a COVID-19 vaccine.

Inovio: Plymouth Meeting, Pennsylvania biotech business received $11.9 million in funding from the Department of Defense to rapidly produce a DNA vaccine for COVID-19 with drugmaker Ology Bioservices.

Johnson & Johnson: The companys pharma unit, Janssen, will start manufacturing its vaccine developed with the Department of Health and Human Services this month, with human trials set to begin by September and a public rollout hoped for early 2021. The company and the federal government are investing more than $1 billion in the vaccine effort.

Medicago: Quebec City-based biotech company received more than $7 million from the Canadian and Quebec governments to fund development of its COVID-19 vaccine.

Moderna: Massachusetts biotech company was the first tobegin human trials of its vaccine on March 16 in Seattle and could deploy it to health workers for emergency use by the fall.

Novavax: Maryland-based vaccine maker received $4 million in funding from CEPI to accelerate development of its vaccine candidates, with clinical trials expected in the late spring.

Sanofi: French medical firm is working with the federal government and Massachusetts-based Translate Bio to expedite its coronavirus vaccine, using technology previously used to develop one for SARS.

Sorrento Therapeutics: San Diego-based biotech firm is teaming up with Cambridge, MA gene therapy company SmartPharm Therapeutics to develop a gene-encoded COVID-19 vaccine; its also working with Chinese drugmaker Mabpharm on a fusion protein treatment for the disease.

Takis Biotech: Italian startup with just 25 employees is developing a vaccine with Stony Brook-based Applied DNA Sciences, with plans to begin human trials before the end of the year.

Themis Bioscience: Austrian biotech firm is part of a group, with the Institut Pasteur and the University of Pittsburgh, which received $4.9 million in initial funding from CEPI to build a COVID-19 vaccine modeled on the vaccine for measles.

Tonix Pharmaceuticals: New York-based pharma outfit is researching a potential COVID-19 vaccine based on the virus that causes horsepox.

Vaxart: San Francisco vaccine manufacturer Vaxart is working with Emergent Biosolutions to develop and manufacture an oral vaccine that can be taken as a tablet.

Vaxil: Israeli biotech startup began preclinical trials for its COVID-19 vaccine candidate.

Zydus Cadila: Indian pharma company announced it would fast-track development of a COVID-19 vaccine in February.

Protective Equipment And Sanitizer:

Anheuser-Busch InBev: The worlds largest beer company is making more than one million bottles of hand sanitizer from surplus alcohol at its breweries around the world.

Giorgio Armani.

Armani: Billionaire Giorgio Armanis luxury fashion brand converted all production at its Italian factories to manufacture single-use medical overalls on March 26.

Bacardi: The Bermuda-based spirits giant converted production at nine production facilities in Mexico, France, England, Italy, Scotland, Puerto Rico and the continental U.S. to make hand sanitizer.

BrewDog: Independent beermaker is making hand sanitizer at its distillery in Scotland.

Bulgari: The Italian luxury jeweler is manufacturing hand sanitizer with its fragrances partner, ICR, with plans to make hundreds of thousands of bottles by May.

Sandro Veronesi.

Calzedonia Group: Italian retail clothing group, owned by billionaire Sandro Veronesi, converted production at several plants in Italy and Croatia to manufacture masks and medical gowns, with initial production of 10,000 masks a day.

Cantabria Labs: Spanish health products and cosmetics firm converted production at one of its factories to make hand sanitizer.

Consomed: Tunisian mask and medical equipment maker put all of its workers, more than 70% of which are reportedly women, on quarantine inside the companys Kairouan factory to maximize production of protective gear.

Decathlon: Sporting goods empire founded by French billionaire Michel Leclercq partnered with Isinnova, a small engineering and design firm based in Italy, to convert snorkeling masks into respirators.

Diageo: The maker of Johnnie Walker whisky and Smirnoff vodka donated two million liters of ethyl alcohol, a byproduct of the distillation process, to hand sanitizer manufacturers.

Fanatics: Billionaire Michael Rubins online sportswear retailer converted its baseball jersey factory in Pennsylvania to make masks and gowns for medical workers.

Fiat Chrysler Automobiles: The multinational automaker announced on March 23 it would begin installing capacity to produce masks, which will be initially distributed in the U.S., Canada and Mexico.

Fippi: Italian diapers producer worked with the Lombardy region and the Polytechnic University of Milan to convert its factory to make up to 900,000 masks a day, which will go to frontline health workers facing a devastating outbreak in the region.

Original post:
Coronavirus Business Tracker: How The Private Sector Is Fighting The COVID-19 Pandemic - Forbes

Media Advisory

Tuesday, March 31, 2020

Findings provide insight that may inform search for treatments.

Researchers at the National Institutes of Health have discovered a second gene that causes melorheostosis, a rare group of conditions involving an often painful and disfiguring overgrowth of bone tissue. The gene, SMAD3, is part of a pathway that regulates cell development and growth. The researchers are now working to develop an animal model with a mutant version of SMAD3 to test potential treatments for the condition. The study appears in the Journal of Experimental Medicine.

Melorheostosis affects about 1 in 1 million people. Its causes have long been unknown. DNA tests of blood and skin could not identify a mutation. The key to finding the gene was to biopsy the affected bone directly and compare it to unaffected bone. Earlier, the researchers used this method to discover the gene for dripping candle wax bone disease, a form of melorheostosis in which excess bone growth appears to drip from the bone surface like hot wax. In that study, mutations in the gene MAP2K1 accounted for eight cases of the disease among 15 patients.

In the current study, researchers scanned the exome the part of the genome that codes for proteins and found mutations in the affected bone. These mutations occurred during the patients lifetime rather than being inherited from parents and are not present in all the cells of the body.

The researchers found SMAD3 mutations in four of the patients who did not have mutations in MAP2K1. SMAD3 is involved in a pathway crucial for skeletal development both before and after birth. The SMAD3 mutations increase the maturation of bone-forming cells and are involved in a cellular pathway distinct from the MAPK2K1 pathway.

The study was conducted by researchers at NIHs Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institute of Arthritis and Musculoskeletal and Skin Diseases and the NIH Clinical Center, as well as the Ludwig-Boltzmann Institute of Osteology at the Hanusch Hospital in Vienna, Austria.

Senior author Joan Marini, M.D., chief of the NICHD Section on Heritable Disorders of Bone and Extracellular Matrix, is available for comment.

Kang, H. et al. Somatic SMAD3 activating mutations cause melorheostosis by upregulating the TGF-/SMAD pathway. Journal of Experimental Medicine. DOI:10.1084/jem.20191499

About the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD): NICHD leads research and training to understand human development, improve reproductive health, enhance the lives of children and adolescents, and optimize abilities for all. For more information, visit https://www.nichd.nih.gov.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

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NIH researchers discover gene for rare disease of excess bone tissue growth - National Institutes of Health

FILE PHOTO: The company's logo is seen at a building of Swiss drugmaker Novartis in Rotkreuz, Switzerland, January 29, 2020. REUTERS/Arnd Wiegmann

ZURICH (Reuters) - Swiss drugmaker Novartis on Friday won a key European recommendation for its gene therapy Zolgensma against spinal muscular atrophy (SMA), clearing a hurdle for $2.1 million per patient treatment for approval in Europe within months.

The European Medicines Agencys (EMA) Committee for Human Medicines (CHMP) recommended conditional approval for Zolgensma for certain patients: those with Type 1 SMA, the severest form of the disease, or for SMA patients with up to three copies of the so-called SMN2 gene, an indicator of the diseases severity.

The EMAs conditional approval is meant to speed up access to medicines for unmet needs, based on less-complete data than normally expected.

Typically the European Commission approves medicines for use shortly after a CHMP recommendation, and Novartis is expecting a decision by June. The medicine, the worlds costliest one-time treatment at its U.S. list price, has already been approved in the United States and Japan.

Novartis is counting on the gene therapy becoming a billion-dollar-per-year seller. Zolgensma is the second treatment for SMA to be approved after Biogens Spinraza three years ago. Roche is expecting its oral drug risdiplam to win U.S. regulators blessing in May.

Reporting by John Miller; Editing by Michael Shields

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Novartis wins key European recommendation for gene therapy Zolgensma - Reuters