Category Archives: Stem Cell Medicine


Rethinking the Science of Skin – The New Yorker

When my sister and I were young, we liked to come home from school and turn on Guiding Light, a soap opera on CBS. We only ever caught the last fifteen minutes of the hour-long show, but, because it wasnt particularly subtle, this was plenty of time to follow even its most involved plotlinessuch as when Reva Shayne, a nine-times-married character who had arcs as a talk-show host, a psychic, the princess of a fictional island, and a time traveller to the Civil War and Nazi Germany, had to fight Dolly, a devious clone that her most recent husband had made of her in order to spare her children from grief during the most recent of her presumed deaths.

Guiding Light began, in 1937, as a radio show to promote a soap called Duz. (Duz does everything.) When it went off the air, in 2009, it was the longest-running show in broadcast history. It was owned, until the end, not by CBS but by Procter & Gamble, which began as a soap company and has been credited with inventing modern advertising in America. In addition to promoting its brands with paintings on trolley cars and billboards, the company developed more than twenty radio and television dramas. The first, Oxydols Own Ma Perkins, premired in 1933; the last, As The World Turns, left the airwaves in 2010, by which time the term soap opera had become freestanding. You could even watch them, as I did, without ever knowing they had anything to do with a soap company.

It was easy, until the COVID-19 outbreak, not to think very often or very deeply about soap. Early in the pandemic, this began to change. We learned which pop songs had choruses that we could sing to keep us scrubbing for a full twenty seconds; we learned that, at least during the pre-lockdown period, the lines outside mens rooms grew suddenly longerlikely because (according to one study) only thirty-one per cent of men had previously been in the habit of washing their hands after using the bathroom. As distilleries and breweries pivoted to producing hand sanitizer, the Times ran a piece explaining why old-fashioned soap was actually better at destroying the coronavirus: the hydrophobic tails of soap molecules bond with the lipid membrane that protects the virus, literally ripping it apart, while their hydrophilic heads bond with the water that washes the dead virus away. Like many people, I developed a new appreciation for soap, imagining with grim satisfaction a scene of microscopic destruction each time I scrubbed my hands.

So this has been a strange time to be reading a book by a medical doctor which takes a critical view of the soap industry and begins with the sentence Five years ago, I stopped showering.

Let me clarify at once that James Hamblin, a staff writer at The Atlantic and the author of Clean: The New Science of Skin (Riverhead), is still an advocate of regular handwashing, indisputably a world-changing innovation in public health, and of especially crucial importance at this moment in history. (Hamblin also writes that he would never wear a white coat two days in a row without cleaning it.) But hes doubtful about all the scrubbing and soapingnot to mention moisturizing and deodorizing and serum-and-acid applicationto which we subject the rest of our bodys largest organ, and about the companies that spend a lot of money to convince us that we must do so to be clean.

Soap is an ancient invention, so old that we can only assume it was the lucky result of animal fat spilling into fire ash and some people being alert enough to notice the cleaning power of the resulting lather. Still, early versions, made with lye, could burn skin, and were used more often for laundry than for people. Bathing more commonly involved water, sand, pumice, scrapers, and oils or perfumesthough in certain places the whole notion was seen as dangerous. Some historical records suggest that washing was comparatively rare in the Western world: Marco Polo wrote of his surprise at how frequently people in India and China bathed, and Ahmad ibn Fadlan, who travelled from the court of Baghdad to the Volga River in the early tenth century, wrote that the people he met on his journey did not wash after eating, shitting, peeing, or having sex, and were the filthiest of Allahs creatures. The French historian Jules Michelet described the European Middle Ages as a thousand years without a bath.

In America, soap made for skin became commonly sold only in the nineteenth century, largely as a way to make money from the leftovers of the meatpacking industry, which produced large quantities of unused animal fat. Entrepreneurs added potash and made soap, for which they then needed to create public demand. These early soapers included William Procter and James Gamble, who began working together after marrying a pair of sisters; another familial pair, whose company name eventually changed from Lever Brothers to Unilever; and a man named William Wrigley, Jr., who gave away chewinggum as a promotion for his soap, but found that the gum was in higher demand.

Last year, the beauty-and-personal-care market in the U.S. was valued at nearly a hundred billion dollars, which makes it hard to imagine a time when people had to be persuaded to use soap. But the soap industry, Hamblin argues, serves as an effective introduction to the history of American marketing. Early soap companies pioneered many techniques that we still see today: a single company owning lots of competing brands with nearly identical products, in order to foster feelings of consumer choice and loyalty; the use of sponsored content, such as the soap operas or Procter & Gambles How to Bring Up a Baby, which was part health pamphlet and part advertisement. The ad campaigns created a sense of lurking danger in the competition by claiming that their own products were safer and purer, or they promoted, as product virtues, obscure, jargony terms (triple milled) that consumers assumed to be important simply because they were touted on a package. The companies leaned, not at all subtly, on racism and classism to sell their products. They even used people who would now be called influencers, such as the film stars who appeared in 9 out of 10 screen stars use Lux Toilet Soap ads. Lever never even paid them, Hamblin writes, and the practice being so new, the stars apparently didnt think to ask.

The other innovation was to create, and then meet, needs that people didnt know they had. Hamblin notes that B.O. began as a marketing term, and that many soaps advertised as antimicrobial and antibacterial were less safethan standard soap, leaving behind dangerous compounds. (Many products that we now think of as soaps are actually detergents, made from synthetic compounds.)

Meanwhile, soap companies, in order to expand their product lines, had to sell the idea that soap was insufficient on its ownor that its effects had to be undone by yet more products, Hamblin writes. You needed separate soaps for your hair, your body, your face, and even for different members of a family. (Albert Einstein, asked why he didnt use shaving cream, then newly invented, is reported to have replied, Two soaps? That is too complicated!) To offset the drying effects of soap, you then needed other productsconditioners, moisturizers, toners. Hamblin identifies the 1957 introduction of Dove, whose cleaning power is reduced because its mixed with moisturizer, as the moment when the industry started moving toward selling a product that would do nothing at all.

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Rethinking the Science of Skin - The New Yorker

Boehringer Ingelheim acquires GST to strengthen its stem cell capabilities in Animal Health – Business Wire

INGELHEIM, Germany--(BUSINESS WIRE)--Boehringer Ingelheim, a market leader in animal health, has acquired Global Stem cell Technology (GST), a Belgian veterinary biotech company. GST is dedicated to the research, development and production of evidence-based, regenerative medicines (stem cell therapies) used to treat orthopedic and metabolic diseases in animals. Boehringer Ingelheim already entered into a partnership with GST in 2018; in 2019, the companies launched Arti-Cell Forte in Europe.

Arti-Cell Forte is testimony to the innovation strength that lies within both companies. It is the first-ever stem cell product in the veterinary world granted marketing authorization by the European Commission. The acquisition and integration of GST will accelerate the development pipeline of Boehringer Ingelheim while maintaining its focus on setting new standards of care for animals.

Collaboration with external partners plays an essential role in helping us expand our portfolio. After two years of a very successful partnership, we have decided to acquire GST. We are convinced that its expertise in the field of state-of-the art stem cell products will help us bring even more innovative solutions to our customers, shares Jean-Luc Michel, Head of Global Strategic Marketing, Boehringer Ingelheim Animal Health.

Boehringer Ingelheim wants to lead a new wave of innovation in the veterinary field. This ambition is a natural fit with GSTs management, staff and vision. From the very beginning we aimed to change the veterinary field, a role we will continue to play as a new R&D division within Boehringer Ingelheim, says Jan Spaas, CEO of GST.

This decision is fully aligned with our recently refocused strategic direction. Stem cell research areas and regenerative medicine offer an exciting potential for the next wave of innovation we are actively pursuing. In addition, strengthening external partnerships to accelerate our innovative efforts and growth is one of the key elements of our strategy, adds Eric Haaksma, Head of Global Innovation at Boehringer Ingelheim Animal Health.

The companies did not disclose the financial terms of the deal.

For references and notes to editors, please visit:

http://www.boehringer-ingelheim.com/press-release/boehringer-ingelheim-acquires-global-stem-cell-technology

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This press release is issued from our Corporate Headquarters in Ingelheim, Germany and is intended to provide information about our global business. Please be aware that information relating to the approval status and labels of approved products may vary from country to country, and a country-specific press release on this topic may have been issued in the countries where we do business.

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Boehringer Ingelheim acquires GST to strengthen its stem cell capabilities in Animal Health - Business Wire

Why I Walk To End Alzheimer’s – TAPinto.net

The Alzheimer's Association Greater New Jersey Chapter is spotlighting people who make a difference in the fight to end Alzheimer's and all other dementia, by participating in the upcoming 2020 Walk To End Alzheimers (WTEA)the worlds largest event to raise awareness and funds for Alzheimers care, support and research. Today we say "thank you" toAnnie Butt.

Annie, a student at New Jersey Institute of Technology where she is studying biomedical engineering and stem cell research, is the Chair of the Essex-Hudson-Union Walk Committee. In addition to conducting outreach to local colleges and community-based organizations encouraging them to volunteer and participate in the Walk, she also creates promotional graphics used on the Chapters social media platforms designed to engage participants and show appreciation.

Joining the Walk to End Alzheimer's has been such an exciting opportunity for me! said Annie. As someone who has a passion for medicine, I want to participate in the Walk to End Alzheimer's, especially since there is no cure for the disease. I am constantly lookingforward to meeting new people through the Association and hearing the stories ofindividualswith loved ones affected by Alzheimer's and helping in any way that I can.

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More than 5 million Americans are living with Alzheimer's disease, the sixth-leading cause of death in the U.S. and the only disease among the top 10 causes that cannot be cured, prevented or even slowed. Additionally, more than 16 million family and friends provide care to people with Alzheimers and other dementias in the U.S. In New Jersey alone, there are more than 190,000 people living with the disease and 448,000 caregivers.

Register your team today. Sign up as a Team Captain or register to walk as an individual. Learn more at alz.org/njwalk. To donate, text 2ENDALZ to 51555, or contact us at gnjwalks@alz.org for more information.

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Why I Walk To End Alzheimer's - TAPinto.net

Stem Cell Market Status by Upcoming Trends 2020 Growth Drivers | Competitive Strategy, Regional Outlook with SWOT Analysis till 2024 – Owned

Stem cells banking is gaining importance with the support of government initiatives. The number of stem cell banks is increasing in developing countries, which is aiding the growth of the market. Also, increasing awareness about stem cell storage among the people has positively affected the market. Currently, the market is not well established in many therapeutic areas and has shown nascent success in history. However, it holds great potential in both the diagnosis and therapeutic fields.

Scope of the Report:

The scope of this market is limited to tracking the stem cell market. As per the scope of this report, stem cells are biological cells that can differentiate into other types of cells. Also, various types of stem cells are used for therapeutic purposes.

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Key Market Trends:

Oncology Disorders Segment is Expected to Exhibit Fastest Growth Rate Over the Forecast Period

Cancer has a major impact on society in the United States and across the world. As per the estimation of National Cancer Institute, in 2018, 1,735,350 new cases of cancer were anticipated to get diagnosed in the United States, and 609,640 deaths were expected from the disease. This increasing medical burden is due to population growth. Bone marrow transplant or stem cell transplant is a treatment for some types of cancers, like leukemia, multiple myeloma, multiple myeloma, neuroblastoma, or some types of lymphoma.

Embryonic stem cells (ESC) are the major source of stem cells for therapeutic purposes, due to their higher totipotency and indefinite lifespan, as compared to adult stem cells with lower totipotency and restricted lifespan. However, the use of ESCs for research and therapeutic purposes is restricted and prohibited in many countries throughout the world, due to some ethical constraints. Scientists from the University of California, Irvine, created the stem cell-based approach to kill cancerous tissue while preventing some toxic side effects of chemotherapy by treating the disease in a more localized way.

Although the market shows positive growth, due to the growing focus of stem cell-based research that can further strengthen the clinical application, its expensive nature for stem cell therapy may still hamper its growth.

North America Captured The Largest Market Share and is Expected to Retain its Dominance

North America dominated the overall stem cell market with the United States contributing to the largest share in the market. In 2014, the Sanford Stem Cell Clinical Center at the University of California, San Diego (UCSD) Health System, announced the launch of a clinical trial, in order to assess the safety of neural stem cell-based therapy in patients with chronic spinal cord injury. Researchers hoped that the transplanted stem cells may develop into new neurons that could replace severed or lost nerve connections, and restore at least some motor and sensory functions. Such numerous stem cell studies across the United States have helped in the growth of the stem cell market.

The report provides key statistics on the market status of the Stem Cell Market manufacturers and is a valuable source of guidance and direction for companies and individuals interested in the Stem Cell .

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Key Insights of Stem Cell Market:

Stem Cell Market Report Covers Following Points in TOC:

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Stem Cell Market Status by Upcoming Trends 2020 Growth Drivers | Competitive Strategy, Regional Outlook with SWOT Analysis till 2024 - Owned

Sobi will file for a re-examination of emapalumab in Europe following negative opinion by CHMP | Antibodies | News Channels – PipelineReview.com

Details Category: Antibodies Published on Friday, 24 July 2020 14:39 Hits: 93

STOCKHOLM, Sweden I July 24, 2020 I Swedish Orphan Biovitrum AB (publ) (Sobi) (STO:SOBI) today announced that the Committee for Medicinal Products for Human use (CHMP) has adopted a negative opinion recommending a refusal of the marketing authorisation for emapalumab for the treatment of primary haemophagocytic lymphohistiocytosis (HLH) in children under 18 years of age in Europe. Given the significant unmet medical need that emapalumab addresses in patients with primary HLH with no approved treatments in Europe, Sobi will be requesting a re-examination by the CHMP with an expected opinion by end of year 2020.

Primary HLH is a rare syndrome that typically presents in infancy but can also be seen in adults and is associated with high morbidity and mortality. In spite of some treatment advances, there continues to be a very high unmet medical need in particular in patients that have failed conventional therapy as there are no approved treatment options outside the US. In the US, emapalumab is the first therapy approved by the US Food & Drug Administration (FDA) for primary HLH. Over 100 patients have been treated in the US and the benefit/risk profile continues to be favourable.

Emapalumab has demonstrated a positive benefit/risk profile in primary HLH in a post-approval real life setting in the US since the FDA approval in 2018. The product has been able to make a substantial difference for a very vulnerable group of patients in the US. We are proud of having made a significant contribution with our product in the primary HLH indication and we are gratified by the recent academic validation of our work via publication in the New England Journal of Medicine. During the last years our team has gained a lot of experience in this rather complex disease area. We will do our utmost to share these insights and address the open questions by CHMP during the re-examination with a view to secure access for primary HLH in children to this treatment in Europe, says Guido Oelkers, CEO and President of Sobi.

HLH is a rare disease but with a large unmet medical need globally. The most important markets based on number of patients for both primary and secondary HLH are China followed by the US, Europe and Japan. In addition to HLH, Sobi will initiate clinical studies with emapalumab for potential indications such as pre-emptive treatment of patients with risk factors of HSCT acute graft failure which will further expand the patient population and market potential for emapalumab. Sobis earlier communicated estimated peak sales target for emapalumab beyond USD 500 million remains unchanged regardless of an approval in Europe.

Professor Franco Locatelli, Principal Investigator in the EU says In my role as Principal Investigator of the NI-0501-04/05 studies in Europe I was significantly surprised about the EMA decision not to approve emapalumab for children with primary HLH who failed or are intolerant to front-line therapy. I had the privilege to observe that this monoclonal antibody, targeting the main cytokine involved in the disease pathophysiology, was well tolerated and effective in a large proportion of the patients, representing a model of precision medicine. While US children have since almost 2 years the possibility to be treated with this novel, safe, highly effective and targeted therapy, the EMA decision paves the way for migratory health flows towards non-European Centers that can grant this treatment.

Professor Michael Jordan, Principal Investigator in the US confirms The NI-0501-04/05 studies have demonstrated that emapalumab has clear therapeutic activity in primary HLH and have validated interferon gamma as a key target in these patients. These studies have also demonstrated that this unique and targeted approach to therapy has a very favorable safety profile. I am grateful for the opportunity to help lead these trials which were conducted with the greatest rigor and transparency, far exceeding that of any trial to date in this very challenging patient population. The worldwide team of collaborators, including physicians at many centers in the US and Europe, as well as individuals at Sobi, should be proud of this ground-breaking achievement. I believe that emapalumab will benefit patients around the world with HLH, especially as we continue to learn how to best apply this unique drug in patients with HLH.

Recently, the results from the pivotal study evaluating the efficacy and safety of emapalumab in patients with primary HLH were published in one of the highest-ranking medical journals, New England Journal of Medicine.

About emapalumab Emapalumab is a monoclonal antibody that binds to and neutralises interferon gamma (IFN). In the US, emapalumab is indicated for paediatric (newborn and older) and adult primary haemophagocytic lymphohistiocytosis (HLH) patients with refractory, recurrent or progressive disease, or intolerance to conventional HLH therapy. Emapalumab is the first and only medicine approved in the US for primary HLH, a rare syndrome of hyperinflammation that usually occurs within the first year of life and can rapidly become fatal unless diagnosed and treated. The FDA approval is based on data from the phase 2/3 studies (NCT01818492 and NCT02069899). Emapalumab is indicated for administration through intravenous infusion over one hour twice per week until haematopoietic stem cell transplantation (HSCT). For more information please see http://www.gamifant.com including the full US Prescribing Information.

About SobiTM Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Sobi is providing sustainable access to innovative therapies in the areas of haematology, immunology and specialty indications. Today, Sobi employs approximately 1,400 people across Europe, North America, the Middle East, Russia and North Africa. In 2019, Sobis revenues amounted to SEK 14.2 billion. Sobis share (STO:SOBI) is listed on Nasdaq Stockholm. You can find more information about Sobi at http://www.sobi.com.

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Sobi will file for a re-examination of emapalumab in Europe following negative opinion by CHMP | Antibodies | News Channels - PipelineReview.com

The Long Game of Coronavirus Research – The New Yorker

Last month, Anthony Fauci, the director of the National Institute of Allergy and Infectious Diseases, spoke at a biotech conference, where he emphasized how much is still unknown about the coronavirus. I thought H.I.V. was a complicated disease, he said. Its really simple compared to whats going on with COVID-19. To anyone who knows the history of AIDS researchFauci has spent much of his career studying the diseasethis was a dismaying thing to hear. In 1984, President Reagans Health and Human Services Secretary, Margaret Heckler, said, We hope to have a vaccine ready for testing in about two years. Almost four decades later, there is still no vaccine. If Hecklers words now seem like wishful thinking, the Trump Administration has worried scientists and physicians with what may prove to be a similar overpromise. In May, it unveiled a plan to deliver hundreds of millions of doses of a COVID-19 vaccine by the end of this year. The plans nameOperation Warp Speedis meant to spark hope. But, in science, true hope is clear-eyed and brings a tight focus on the barriers and potential setbacks that exist along the path to desired results.

Read The New Yorkers complete news coverage and analysis of the coronavirus pandemic.

In the face of a crisis as urgent as COVID-19, speed is desirable, and the worldwide mobilization to conquer the virus has been inspiring. But what Fauci said illustrates why its a grave mistake to favor speed at the cost of rigor. Quite simply, this is a disease that weare only beginning to understand: since the outbreak began, it has become evident that its effects are, like those of AIDS, astonishingly diverse and complex. Still largely thought of as a respiratory diseaseit can indeed inflict devastating damage to the lungsit is actually, as Fauci noted, capable of roving throughout the body. There are cases in which it causes kidney failure, stroke, or a so-called cytokine storm, an overreaction of the bodys immune system that can lead to multiple organ failure. In children, infection can lead to multisystem inflammatory syndrome, a condition that can damage the heart and other vital organs. COVID-19 has a startling spectrum of severityfrom no symptoms to deathdepending on a host of poorly understood factors. Fauci also pointed out another unknown: whether some survivors, especially those with the severest symptoms, would end up dealing with lifelong debilitating effects. Currently, we are not even sure how to assess protective immunity if we had a vaccine in handwhether protection would be broad among all age groups and encompass the healthy as well as those whose clinical conditions, such as diabetes, heart disease, and obesity, predispose them to COVID-19. Even more worrying, for those who imagine a vaccine might end the pandemic like turning off a light switch, a number of recent studies suggest that people whove had the disease may not emerge with robust, lasting immunity. If so, its possible that the initial protection offered by a successful vaccine would similarly wane, and people could be infected again.

Its understandable how desperate we all are for quick solutions, with the number of infections and deaths skyrocketing. Some twelve hundred clinical studies have been designed since January, but many are too small to have much chance of producing clear results. Researchers have been publishing their papers online before they have undergone peer review. In May, the biotech company Moderna published initial results of an early trial of their vaccine in a press release. Modernas vaccine dominated research news again last week, afterfuller results of that trial were published in The New England Journal of Medicine. That work is still a preliminary achievement, since there were only fifteen healthy volunteers in each of three vaccine dose groups, and, in the moderate- and high-dose groups, almost every volunteer had side effects. An accompanying editorial from Penny Heaton, of the Gates Foundation, cautioned that we wont really know about the safety of Modernas vaccine until many thousands receive it, nor whether the reported immune response in volunteers is actually protective against the virus. There are clear risks with proceeding to human trials in haste. As Kenneth Frazier, the C.E.O. of Merck, pointed out last week, there have been cases, in the past, in which trial vaccines not only didnt confer protection, but actually helped the virus invade the cell, because it was incomplete in terms of its immunogenic properties. Promises to have a vaccine ready by the end of the year, he said, did a grave disservice to the public.

Producing a vaccine that is able to confer immunity on disparate age groups with varying levels of vulnerability is an enormous task, especially because COVID-19 is a brand new disease in humans, and therefore one to which we have no natural immunity. (Even a vaccine as comparatively simple as the annual flu shot reduces the risk of flu sickness for only about forty to sixty per cent of recipients.) All this means that a first vaccine, while a welcome tool in fighting COVID-19, may well turn out to be of limited use. The lesson learned from AIDS is the value of building a protective scientific infrastructure beyond a vaccine, something that requires legions of scientists working carefully and in concert to understand the numerous ways that a virus causes disease.

In early June, I visited the National Emerging Infectious Diseases Laboratories (NEIDL), to interview researchers there who are working on COVID-19. Part of Boston University, NEIDL (pronounced like needle) is one of two academically-affiliated institutions in the U.S. with laboratories that are certified to handle the deadliest pathogens known to man, like the Ebola and Marburg viruses and yellow fever. NEIDLs origins date to the period after 9/11, when Fauci warned that the country needed a better system to defend against possible bioterrorism attacks, and the government earmarked billions of dollars to prepare for such an event. NEIDL received its funding in 2003, but getting a facility ready to handle such pathogens takes years: the building itself was completed in 2008; acquiring the necessary environmental approvals from local government and community representatives took almost a decade. It wasnt until 2017, that NEIDL was fully approved to undertake all the work for which it was created.

The facility, which is on B.U.s medical campus, in Bostons South End, stands seven stories high, a modern structure of glass and concrete. Barriers surround the site at a distance of a hundred and fifty feet from the building; they are fitted with motion-detection sensors and are strong enough to stop a fifteen-thousand-pound truck going fifty miles an hour. Inside, there are around a dozen containment laboratories for dangerous microbes, which hold specialized microscopic equipment and robotic devices, and secure environmental facilities designed for pathogen vectors, like mosquitos and ticks, and animals, which researchers use to model human diseases. NEIDLs Biosafety Level 4 facilitythe part of the building that is licensed to handle the most dangerous microbesis constructed as a building within the larger building. That nested structure has twelve-inch-thick concrete walls coated with multiple layers of epoxy resin. While NEIDL itself is built on piling that go into bedrock, the B.S.L.-4 floors are flexible, and can move at a different frequency from the main structure in case of an earthquake. Researchers work in sealed suits, resembling those of astronauts, with a large transparent bubble over the head, supplied with filtered air via a hose connected to the ceiling. If there is a spill, the space is typically decontaminated with chlorine dioxide, and low air pressure maintained in the lab insures that air rushes in rather than out, so that no airborne virus can escape.

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The Long Game of Coronavirus Research - The New Yorker

Current research: Exosome Technologies Market Report- Growth with Top Companies Capricor, Codiak and Evox Therapeutics – WhaTech Technology and…

Exosome Technologies Market report is an in-depth research articulated by analysts by analyzing all the key factors such as regional market conditions, market boomers and decliners, opportunities, and size & scope of the market.

The research reports on Exosome Technologies Market report gives detailed overview of factors that affect global business scope. Exosome Technologies Market report shows the latest market insights with upcoming trends and breakdowns of products and services.

This report provides statistics on the market situation, size, regions and growth factors. Exosome Technologies Market report contains emerging players analyze data including competitive situations, sales, revenue and market share of top manufacturers.

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Top Company Profile Analysis in this Report

Capricor, Codiak and Evox Therapeutics

Exosome Technologies Market Report explores the application of exosome technologies within the pharmaceutical and healthcare industries. Exosomes are small cell-derived vesicles that are abundant in bodily fluids, including blood, urine and cerebrospinal fluid as well as in in vitro cell culture.

These vesicles are being used in a variety of therapeutic applications, including as therapeutic biomarkers, drug delivery systems and therapies in their own right. Research within this area remains in the nascent stages, although a number of clinical trials have been registered within the field.

Exosomes have several diverse therapeutic applications, largely centering on stem cell and gene therapy. Exosomes have been identified as endogenous carriers of RNA within the body, allowing for the intercellular transportation of genetic material to target cells.

As such, developers have worked to engineer exosomes for the delivery of therapeutic miRNA and siRNA-based gene therapies. As RNA is highly unstable within the body, a number of different biologic vector systems have been developed to enhance their transport within the circulation, including viruses and liposomes.

Similarly, exosomes derived from stem cells have also been identified for their therapeutic applications, particularly in the treatment of cancer and cardiovascular disease. Exosome technologies offer several advantages over existing biologic-based drug delivery systems.

They have a long circulatory half-life as a result of their high stability and ability to avoid breakdown by the mononuclear phagocyte system and reticuloendothelial systems. Moreover, exosomes have several functional properties that favor their use in therapeutic delivery.

Exosomes can be engineered to incorporate targeting ligands, allowing them to deliver cargo selectively to cells. Their small size allows them to penetrate the blood-brain barrier for the delivery of central nervous system therapies, whereas in cancer they can accumulate within the tumor via enhanced permeability and retention effects.

Finally, clinical trials have shown relatively large-scale production to be possible and indicate that exosome therapies can be safely administered to humans. Additionally, exosomes are being investigated for their potential as prognostic and diagnostic biomarkers for several different disease indications.

Exosomes make good candidates for biomarker research because of two unique characteristics: their presence in various accessible bodily fluids, and their resemblance to their parent cells of origin. R&D in exosome technologies has increased markedly in recent years.

This report provides detailed information on the various healthcare applications of exosomes, and assesses the pipeline, clinical trial and company landscapes.

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Table of Contents

1 Table of Contents 4 1.1 List of Tables 6 1.2 List of Figures 7

2 Exosomes in Healthcare 8 2.1 Overview of Exosomes 8 2.2 Drug Delivery Systems 9 2.2.1 Modified Release Drug Delivery Systems 9 2.2.2 Targeted Drug Delivery Systems 10 2.2.3 Liposomes 12 2.2.4 Viruses 14 2.2.5 Exosomes 17 2.3 The Exosome Lifecycle 18 2.4 Exosomes in Biology 18 2.5 Exosomes in Medicine 19 2.5.1 Biomarkers 19 2.5.2 Vaccines 20 2.6 Exosomes as a Therapeutic Target 20 2.7 Exosomes as Drug Delivery Vehicles 21 2.8 Therapeutic Preparation of Exosomes 21 2.8.1 Isolation and Purification 22 2.8.2 Drug Loading 22 2.8.3 Characterization 23 2.8.4 Bioengineering 23 2.8.5 Biodistribution and In Vivo Studies 23 2.8.6 Advantages of Exosome Therapies 24 2.8.7 Disadvantages of Exosome Therapies 24 2.9 Exosomes in Therapeutic Research 25 2.9.1 Exosome Gene Therapies 25 2.9.2 Exosome in Stem Cell Therapy 26 2.10 Exosomes in Oncology 27 2.10.1 Immunotherapy 27 2.10.2 Gene Therapy 28 2.10.3 Drug Delivery 29 2.10.4 Biomarkers 30 2.11 Exosomes in CNS Disease 30 2.11.1 Tackling the Blood-Brain Barrier 30 2.11.2 Exosomes in CNS Drug Delivery 31 2.11.3 Gene Therapy 32 2.12 Exosomes in Other Diseases 33 2.12.1 Cardiovascular Disease 33 2.12.2 Metabolic Disease 33

3 Assessment of Pipeline Product Innovation 36 3.1 Overview 36 3.2 Exosome Pipeline by Stage of Development and Molecule Type 36 3.3 Pipeline by Molecular Target 37 3.4 Pipeline by Therapy Area and Indication 38 3.5 Pipeline Product Profiles 38 3.5.1 AB-126 - ArunA Biomedical Inc. 38 3.5.2 ALX-029 and ALX-102 - Alxerion Biotech 39 3.5.3 Biologics for Autism - Stem Cell Medicine Ltd 39 3.5.4 Biologic for Breast Cancer - Exovita Biosciences Inc. 39 3.5.5 Biologics for Idiopathic Pulmonary Fibrosis and Non-alcoholic Steatohepatitis - Regenasome Pty 39 3.5.6 Biologic for Lysosomal Storage Disorder - Exerkine 39 3.5.7 Biologics for Prostate Cancer - Cells for Cells 40 3.5.8 CAP-2003 - Capricor Therapeutics Inc. 40 3.5.9 CAP-1002 - Capricor Therapeutics Inc. 41 3.5.10 CIL-15001 and CIL-15002 - Ciloa 42 3.5.11 ExoPr0 - ReNeuron Group Plc 42 3.5.12 MVAX-001 - MolecuVax Inc. 43 3.5.13 Oligonucleotides to Activate miR124 for Acute Ischemic Stroke - Isfahan University of Medical Sciences 44 3.5.14 Oligonucleotides to Inhibit KRAS for Pancreatic Cancer - Codiak BioSciences Inc. 44 3.5.15 Proteins for Neurology and Proteins for CNS Disorders and Oligonucleotides for Neurology - Evox Therapeutics Ltd 44 3.5.16 TVC-201 and TVC-300 - Tavec Inc. 45

4 Assessment of Clinical Trial Landscape 48 4.1 Interventional Clinical Trials 48 4.1.1 Clinical Trials by Therapy Type 48 4.1.2 Clinical Trials by Therapy Area 49 4.1.3 Clinical Trials by Stage of Development 50 4.1.4 Clinical Trials by Start Date and Status 50 4.2 Observational Clinical Trials 51 4.2.1 Clinical Trials by Therapy Type 51 4.2.2 Clinical Trials by Therapy Area 51 4.2.3 Clinical Trials by Stage of Development 52 4.2.4 Clinical Trials by Start Date and Status 53 4.2.5 List of All Clinical Trials 54

5 Company Analysis and Positioning 67 5.1 Company Profiles 67 5.1.1 Capricor Therapeutics Inc. 67 5.1.2 Evox Therapeutics Ltd 72 5.1.3 ReNeuron Group Plc 73 5.1.4 Stem Cell Medicine Ltd 77 5.1.5 Tavec Inc. 78 5.1.6 Codiak Biosciences Inc. 80 5.1.7 Therapeutic Solutions International Inc. 81 5.1.8 ArunA Biomedical Inc. 83 5.1.9 Ciloa 85

6 Appendix 86 6.1 References 86 6.2 Abbreviations 91 6.3 About GBI Research 93 6.4 Disclaimer 93

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Current research: Exosome Technologies Market Report- Growth with Top Companies Capricor, Codiak and Evox Therapeutics - WhaTech Technology and...

Calling Attention to Treatment Considerations in MCL – OncLive

Adapting treatment selection for patients with mantle cell lymphoma (MCL) in the frontline, maintenance, and relapsed/refractory settings is critical in such a heterogenous disease, explained Lori A. Leslie, MD, who added that novel modalities such as BTK inhibitors and CAR T-cell therapy offer a wealth of new opportunities for these patients.

MCL is very heterogenous type of lymphoma, said Leslie. It has a lot of the features of an aggressive lymphoma such as diffuse large B-cell lymphoma (DLBCL), but it also has a lot of the difficult-to-treat features seen in indolent lymphomas.

In an interview with OncLive, Leslie, a lymphoma attending at John Theurer Cancer Center (JTCC), Hackensack Meridian Health, discussed the current treatment landscape in MCL, factors to consider when selecting between BTK inhibitors, and other treatment options on the horizon.

OncLive: What does the current frontline treatment landscape look like in MCL?

Leslie: There are many different up-front treatment options for patients with MCL. [Treatment selection largely] depends on the patients age and fitness, as well as characteristics of their disease. Two things we look for that are predictive of a more aggressive disease course are high Ki-67 proliferative rate of over 30% and an abnormality in the p53 tumor suppressor gene. In general, when abnormal, a TP53 mutation predicts for a more aggressive course of MCL.

For the standard[-risk] patients without a TP53 abnormality, we dont typically watch-and-wait outside of a relatively rare, indolent subgroup of patients where that method may be appropriate. Instead, we treat the patient.

The first question is, Is this patient fit to consider an autologous stem cell transplantation (ASCT) or dose-intensive chemotherapy? Typically, patients will receive dose-intensive chemotherapy. Depending on the regimen, like hyper-CVAD [hyperfractionated cyclophosphamide, vincristine, doxorubicin hydrochloride, and dexamethasone] for example, we dont necessarily have to do a transplant for consolidation. Other high-dose regimens includedihydroxyacetone phosphateor the Nordic regimen, typically followed by consolidative ASCT. After transplant or high-dose chemotherapy, we consider maintenance rituximab (Rituxan) in the frontline setting as it has been associated with a survival benefit.

For patients with high-risk MCL, such as those with a TP53 abnormality, there is no established standard of care. In those patients, we typically consider clinical trials. There are some approaches looking at combining targeted therapy such as BTK inhibitors with chemotherapy to try to eradicate those more resistant clones. However, clinical trials are key in that high-risk patient group.

For patients who are not candidates for intensive chemotherapy, there are some less intensive chemotherapy options we consider, including R-BAC [rituximab, cytarabine, and bendamustine], bendamustine/rituximab, and VR-CAP [bortezomib (Velcade), rituximab, cyclophosphamide, doxorubicin, and prednisone]. We also consider maintenance rituximab after those options.

What treatment options are available to patients with relapsed/refractory disease?

Relapsed/refractory MCL is a rapidly evolving field with many new targets and novel therapies, including cellular therapy which is entering our armamentarium. Typically, the most important choice when someone comes in with relapsed/refractory MCL is what they received as frontline therapy and how long they were in remission.

If I had a patient who received chemoimmunotherapy and ASCT and enjoyed a 10-year remission, that patient is different than someone who received chemoimmunotherapy and is relapsing while they are on maintenance rituximab.

For patients with a higher risk of relapse or really any [risk of] relapse, the standard of care is usually targeted therapies, including BTK inhibitors. There are emerging data looking at venetoclax (Venclexta) in this group of patients. Lenalidomide (Revlimid) with rituximab is another potential option. Bortezomib-based therapies are also used here but fall a little further down the preference list now that we have BTK inhibitors.

In patients with multiple relapses, anti-CD19 CAR T-cell therapy is most exciting. There have been recent data looking at CD19-directed CAR T-cell therapy in patients with relapsed MCL who are resistant or refractory to BTK inhibitors showing a very high overall response rate (ORR). About two-thirds of patients are achieving complete responses (CRs) that appear durable. As more data emerge, hopefully that will become another standard option that is available outside of clinical trials for our patients with relapsed disease.

What is the role of maintenance therapy in the frontline and relapsed/refractory settings?

In the frontline setting, if a patient has had chemotherapy and transplant, its standard to consider maintenance rituximab therapy. The most common schedule is to give it every 2 months for 3 years, or for 2 years in certain situations. Rituximab is continued until a patient progresses or [develops] some toxicity with the therapy. Though, it is typically well tolerated.

There is a suggestion of overall survival benefit in some studies, as well as progression-free survival benefit [with maintenance rituximab]. Typically, we recommend maintenance therapy unless there is a reason not to give it after frontline therapy.

In the relapsed/refractory setting, it really depends on what the patients treatment is. BTK inhibitors are generally indefinite therapies, so there is not a maintenance-type approach [in that situation]. Patients will stay on BTK inhibitors until they progress or develop an unacceptable toxicity. Looking toward time-limited therapy, maybe we could add a BCL-2 inhibitor with venetoclax in MCL like we are doing in chronic lymphocytic leukemia (CLL).

What additional considerations need to be taken into account when treating an older or unfit patient with MCL?

Patients with MCL who are older or unfit have a unique need. A lot of what I do first is talk to the patient about what their expectations are. Typically, significantly older patients are not candidates for intensive frontline therapy or ASCT. In those patients, less intensive chemotherapy with maintenance rituximab [is preferred].

There are some emerging data about using BTK inhibitors in the frontline setting as induction with abbreviated chemotherapy afterward. That regimen is being evaluated more so in young patients to minimize the amount of chemotherapy used. This could potentially be used as a frontline regimen in older or unfit patients who cant tolerate the previous standard of care.

BTK inhibitors have had a significant impact in the treatment of patients with MCL. How are you currently selecting between the available BTK inhibitors in the relapsed/refractory space?

BTK inhibitors have rapidly impacted the treatment of patients with relapsed/refractory MCL in the past 5 years or so. There are an increasing number of BTK inhibitors that are approved in the space with more on the way. Currently, FDA-approved options include ibrutinib (Imbruvica), acalabrutinib (Calquence), and zanubrutinib (Brukinsa). These are all drugs that have minimal comparative data with each other.

Without direct comparison, the toxicity profiles seem to differ slightly across B-cell malignancies, including CLL, Waldenstrm macroglobulinemia, and MCL. The newer BTK inhibitors have a potentially lower risk of cardiac or bleeding toxicities. Though, that has not been shown in a head-to-head comparison in MCL, it may be a reason to select acalabrutinib or zanubrutinib [over ibrutinib].

Dosing also differs. Ibrutinib is once daily, acalabrutinib is twice daily, and zanubrutinib can be dosed once or twice daily. Sometimes that is a factor when deciding between these agents.

Additionally, something about zanubrutinib that is different from acalabrutinib is the ability to take proton pump inhibitors. Sometimes those drug-drug interactions help select which BTK inhibitor to use.

Importantly, the cost can be quite different from agent to agent. Sometimes we will select 1 BTK inhibitor and the copay, despite whatever copay assistance is given, ends up being too high. In those cases, we may opt for another BTK inhibitor. Obviously, we dont want [cost] to drive our treatment choices. However, sometimes cost is prohibitive for the patient.

How might CAR T-cell therapy impact the treatment landscape? What does the future look like with that treatment modality?

CAR T-cell therapy has changed the landscape of lymphoma treatment over the past few years. Specifically, CD19-directed CAR T-cell therapy is in the late stages of investigation across B-cell malignancies. It is approved in DLBCL and has been extensively studied in other lymphomas, including MCL.

The ZUMA-2 study looking at anti-CD19 CAR T cells was presented at the 2019 ASH Annual Meeting and then published in theNew England Journal of Medicine. We participated in that study at JTCC and treated a number of patients. Of note, the patients on that study were very high risk. About one-third of patients had pleomorphic or blastoid-variant MCL, which is a challenging and aggressive type of MCL. All patients had to have been exposed to ibrutinib, but 88% of patients were primary refractory to BTK inhibitor therapy or initially responded and then relapsed while on ibrutinib. That is a really difficult-to-treat patient population.

Despite that, the ORR was high, at over 90%. Additionally, 67% of patients achieved a CR. According to the latest dataset, 80% of those patients who had a response were still in a response at around 1 year. It seems that those responses are durable.

Although there can be infusion-related toxicities that can be serious with cytokine release syndrome and neurotoxicity, it is a one-time cell infusion. Then patients are off therapy. Most of these patients have received several lines of treatment, and indefinite BTK inhibitor therapy. To go from that to having a one-time infusion and hopefully never needing treatment again is remarkable.

What are some remaining challenges or unanswered questions in MCL?

We need to continue working on how to identify those patients who are high risk from the beginning. Of course, we know that elevated Ki-67 and TP53 abnormalities are clinically prognostic, but outside of that, we need to know how to identify patients who would perhaps benefit from more targeted therapy plus chemotherapy early on.

Using minimal residual disease (MRD) is becoming increasingly important and increasingly available. A patient who has intensive therapy and is in CR but has detectable MRD may be someone who could benefit from consolidation, CAR T-cell therapy, or some other consolidative immunotherapy to try to eradicate the few cells that will eventually lead to relapse.

Are there any emerging agents on the horizon that look promising?

Another agent that is interesting and being developed for MCL is the ROR1 inhibitor cirmtuzumab. That is being studied in combination with ibrutinib in CLL and MCL. Some preliminary data were presented at the 2020 ASCO Virtual Scientific Program showing that the combination was well tolerated and led to a high ORR.

ROR1 is different than anything else were targeting in MCL. That is an appealing, well-tolerated agent that has a novel mechanism of action. Perhaps we can add cirmtuzumab to BTK inhibitors or other therapies to try to improve upon treatment for patients with relapsed/refractory MCL or potentially those with newly diagnosed disease.

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Calling Attention to Treatment Considerations in MCL - OncLive

Research Roundup: Global methane emissions at all-time high, neuronal pathway prevents relapse, possible drug target to help fight infectious diseases…

Our roundup this week highlights research that found global methane emissions have reached an all-time high, with the planet absorbing nearly 600 million tons of methane emissions in 2017. (Photo: Unsplash)

Each week, The Dailys Science & Tech section produces a roundup of the most exciting and influential research happening on campus or otherwise related to Stanford. Heres our digest for the week of July 12 July 18.

Global methane emissions reach an all-time high

The global methane emissions have reached an all-time high, despite carbon dioxide emissions temporarily dropping due to the coronavirus pandemic, a study published on July 14 in Earth System Science Data and Environmental Research Letters found.

We still havent turned the corner on methane, earth system science professor Rob Jackson told Stanford News. Emissions from cattle and other ruminants are almost as large as those from the fossil fuel industry for methane. People joke about burping cows without realizing how big the source really is.

The team analyzed emissions from 2017, the most recent year from which complete global methane data is available, and found that the atmosphere absorbed almost 600 million tons of methane. Methane traps heat more powerfully than carbon dioxide.

The findings suggest agriculture made up two-thirds of methane emissions between 2000 to 2017, with fossil fuel making up the final third.

Well need to eat less meat and reduce emissions associated with cattle and rice farming and replace oil and natural gas in our cars and homes, Jackson told Stanford News. Im optimistic that, in the next five years, well make real progress in that area.

Neuronal pathway prevents drug relapse in mice

Disrupting a neuronal pathway that causes opiate-associated memories in mice prevents drug relapse and has the potential to treat opioid addiction in patients, a study published on July 16 in Neuron found.

The most difficult part of treating addiction is to prevent relapse, especially for opioids, biology associate professor Xiaoke Chen told Stanford News. To prevent relapse, we really need to deal with the withdrawal.

The findings suggest when the researchers silenced the paraventricular thalamus in morphine-dependent mice, the mice no longer preferentially chose morphine over the drug-free saline solution. Through disrupting this neural pathway, researchers found that the animals had no memory of the effects of the drug. Paraventricular thalamus is a brain region that connects many different brain areas associated with drug addiction.

Drugs as a stimulus can drive a very robust behavior, Chen told Stanford News. I want to understand the mechanism underlying that behavior and hope that this knowledge can help address the devastating opioid epidemic in the U.S.

Molecular signal, a possible drug target, allows virus-infected cells to evade immune system

Blocking CD47, a molecular signal, in virus-infected cells allows the immune system to target infected cells, a study published on June 23 in mBio found. Decreasing CD47 may help the body fight infectious disease, serving as a potential drug target.

Cancer cells also increase their CD47 signaling to evade immune system attack.

We wondered whether the mechanism activated by all cancer cells to avoid being destroyed could also be used by persistent infections, so that the microbes can hide inside cells to evade immune cells, pathology stem cell and developmental biology professor Irving Weissman M.D. 65 told Stanford Medicine News. Amazingly, we found that to be true, and blocking the CD47 signal helped the body get rid of more infected cells.

The findings suggest mice and humans cells infected by pathogens show increased expression of the CD47 molecular signal. Additionally, the team found that mice without the gene for CD47 were more resistant to tuberculosis-causing bacteria than control mice.

Infected cells with SARS-CoV-2 the virus that causes COVID-19 also produce increased CD47 signaling, so treatments that block CD47 production could potentially be used in coronavirus patients.

Its probably important for the innate immune system to have a balance of activating and suppressing forces, but we showed that if we play with that balance a little bit, we can clear some pathogens faster, Michal Tal, an instructor at the Institute for Stem Cell Biology and Regenerative Medicine, told Stanford Medicine News. In some cases, it might be better to ease up on this particular immunological brake by blocking CD47.

Contact Derek Chen at derekc8 at stanford.edu.

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Research Roundup: Global methane emissions at all-time high, neuronal pathway prevents relapse, possible drug target to help fight infectious diseases...

Citius Pharmaceuticals Forms Scientific Advisory Board for the Planned Development of its Proprietary Treatment for Acute Respiratory Disease…

CRANFORD, N.J., July 22, 2020 /PRNewswire/ --Citius Pharmaceuticals, Inc. ("CITIUS") ("Company") (NASDAQ:CTXR), a specialty pharmaceutical companyfocused on developing and commercializing critical care drug products, announced today the formation of the Citius ARDS (Acute Respiratory Distress Syndrome) Scientific Advisory Board to provide the company expert guidance on its planned development of induced mesenchymal stem cells (iMSCs) under option from Novellus, Inc. to treat and reduce the severity of acute respiratory distress syndrome (ARDS) associated with COVID -19.

The ARDS Advisory Board consultants are: Michael A. Matthay, MD, Professor of Medicine and Anesthesia at the University of California at San Francisco (UCSF), a Senior Associate at the Cardiovascular Research Institute, and Associate Director of the Critical Care Medicine at UCSF. Dr. Matthay's basic research has focused on the pathogenesis and resolution of the acute respiratory distress syndrome (ARDS), with an emphasis on translational work and patient-based research, including clinical trials. Dr. Matthay's recent research has focused on the biology and potential clinical use of allogeneic bone marrow derived mesenchymal stromal cells (MSCs) for ARDS. He is currently leading the "Mesenchymal Stromal Cells For Acute Respiratory Distress Syndrome (STAT)," a United States Department of Defense supported study of MSCs for ARDS.

Mitchell M. Levy, MD, Chief, Division of Pulmonary, Critical Care, and Sleep Medicine, Department of Medicine, The Warren Alpert Medical School of Brown University, where he is Professor of Medicine. Dr. Levy also serves as Medical Director of the Medical ICU at Rhode Island Hospital. He has been an investigator on numerous pharmacologic and biologic trials intended to treat sepsis, cardiovascular and pulmonary pathology. He has expertise in trial design, clinical trial execution and trial management and is one of the three founding members of the Surviving Sepsis Campaign (SSC). Dr. Levy is Past-President of the Society of Critical Care Medicine (2009).

Lorraine B. Ware, MD, Professor of Medicine and Ralph and Lulu Owen Endowed Chair,Professor of Pathology, Microbiology and Immunology, Vanderbilt University;Director, Vanderbilt Medical Scholars Program. Dr. Lorraine Ware's comprehensive bench-to-bedside research program centers on the pathogenesis and treatment of sepsis and acute lung injury with a current focus on mechanisms of lung epithelial and endothelial oxidative injury by cell-free hemoglobin. Dr. Ware is also a lead investigator for the "Mesenchymal Stromal Cells For Acute Respiratory Distress Syndrome (STAT)" study.

"We are extremely pleased to have been able to attract such a prestigious group of experts to advise and guide us in the Company's planned development of iMSC's for the treatment of ARDS" said Mr. Myron Holubiak, CEO of Citius. "These individuals are recognized opinion leaders and expert in the planning and execution of clinical trials in this therapeutic area. We will be seeking their advice in all phases of our clinical trial design."

About Citius Pharmaceuticals, Inc.Citius is a late-stage specialty pharmaceutical company dedicated to the development and commercialization of critical care products, with a focus on anti-infectives and cancer care. For more information, please visit http://www.citiuspharma.com.

About Citius iMSCCitius's planned induced mesenchymal stem cell (iMSC) product is derived from a human induced pluripotent stem cell (iPSC) line generated using a proprietary non-immunogenic and non-viral mRNA-based (non-viral) reprogramming process. Unlike the MSCs derived from bone marrow, placenta, umbilical cord, or adipose tissue these proprietary iMSCs are based on a clonal process and therefore are genetically homogeneous and exhibit superior potency and higher cell viability. The Citius iMSC is an allogeneic (unrelated donor) mesenchymal stem-cell product manufactured by expanding material from an iMSC master cell bank. The master cell bank produces "off-the-shelf" iMSCs that are uniform as compared to MSCs using donor-sourced cells, which is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. In vitro studies demonstrate that iMSCs are shown to secrete higher levels of immunomodulatory proteins than donor-derived cells, and may reduce or prevent pulmonary injury associated with acute respiratory distress syndrome (ARDS) in patients with COVID-19.

About Acute Respiratory Distress Syndrome (ARDS)ARDS is a type of respiratory failure characterized by rapid onset of widespread inflammation in the lungs. ARDS is a rapidly progressive disease that occurs in critically ill patients most notably now in those diagnosed with COVID-19. ARDS affects approximately 200,000 patients per year in the U.S., exclusive of the current COVID-19 pandemic, and has a 30% to 50% mortality rate. ARDS is sometimes initially diagnosed as pneumonia or pulmonary edema (fluid in the lungs from heart disease). Symptoms of ARDS include shortness of breath, rapid breathing and heart rate, chest pain (particularly while inhaling), and bluish skin coloration. Among those who survive ARDS, a decreased quality of life is relatively common.

Safe HarborThis press release may contain "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Such statements are made based on our expectations and beliefs concerning future events impacting Citius. You can identify these statements by the fact that they use words such as "will," "anticipate," "estimate," "expect," "should," and "may" and other words and terms of similar meaning or use of future dates. Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price.

Factors that could cause actual results to differ materially from those currently anticipated are: the risk of successfully negotiating within the option period a license agreement with Novellus, Inc. for our planned iMSCs therapy for ARDS; our need for substantial additional funds; risks associated with conducting clinical trials and drug development; the estimated markets for our product candidates and the acceptance thereof by any market; risks related to our growth strategy; risks relating to the results of research and development activities; uncertainties relating to preclinical and clinical testing; the early stage of products under development; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; our ability to identify, acquire, close and integrate product candidates and companies successfully and on a timely basis; our dependence on third-party suppliers; our ability to attract, integrate, and retain key personnel; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Contact:Andrew Scott Vice President, Corporate Development (O) 908-967-6677 x105 [emailprotected]

SOURCE Citius Pharmaceuticals, Inc.

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