Researchers at the University of Copenhagen in a new study presented the largest and most complex CRISPR system. Scientists believe that this system can find applications in biomedicine and biotechnology. Their work is featured in the scientific journal Molecular Cell.

CRISPR technology can be used to edit genes and revolutionized the scientific world when it was first introduced. CRISPR-Cas9 is probably the best-known CRISPR system and is widely known as gene scissors.

This is just one of many CRISPR systems in existence.

Now researchers at the University of Copenhagen (UCPH) have mapped and analyzed the atomic structure of one of the most complex CRISPR systems identified to date.

Scientists have disassembled the largest and most complex CRISPR-Cas complex seen so far. They now understand how this system works at the molecular level, said co-author Guillermo Montoya, professor at the Novo Nordisk Foundations Center for Protein Research at the University of Copenhagen (NNF CPR). Scientists have studied a complex called Cmr-, which belongs to a subgroup of so-called type III-B CRISPR-Cas complexes. The new results have already been presented to the public.

In the new study, the researchers examined the role of Cmr in the immune system and the mechanisms underlying its immune response against various bacteriophages, as well as how it is regulated.

The Cmr system, mapped by scientists in the new study, may, among other things, remove single-stranded RNA and DNA, although this will be quite difficult. But in the future, the Cmr system may still be the key to understanding the immune response of bacteria, and it may find applications in the fight against antibiotic resistance.

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Scientists have mapped and analyzed the atomic structure of CRISPR-Cas - FREE NEWS

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The team at MBL, led by MBL Senior Scientist Joshua Rosenthal and Crawford, has achieved the first gene knockout in a cephalopod using the squid Doryteuthis pealeii, an exceptionally important research organism in biology for nearly a century.

The team used CRISPR-Cas9 genome editing to knock out a pigmentation gene in squid embryos, which eliminated pigmentation in the eye and in skin cells (chromatophores) with high efficiency.

This is a critical first step toward the ability to knock out and knock in genes in cephalopods to address a host of biological questions, Rosenthal says.

Cephalopods (squid, octopus and cuttlefish) have the largest brain of all invertebrates, a distributed nervous system capable of instantaneous camouflage and sophisticated behaviors, a unique body plan, and the ability to extensively recode their own genetic information within messenger RNA, along with other distinctive features. These open many avenues for study and have applications in a wide range of fields, from evolution and development, to medicine, robotics, materials science, and artificial intelligence.

The ability to knock out a gene to test its function is an important step in developing cephalopods as genetically tractable organisms for biological research, augmenting the handful of species that currently dominate genetic studies, such as fruit flies, zebrafish, and mice.

CRISPR-Cas9 worked really well in Doryteuthis; it was surprisingly efficient, Rosenthal says. Much more challenging was delivering the CRISPR-Cas system into the one-celled squid embryo, which is surrounded by an exceedingly tough outer layer, and then raising the embryo through hatching. The team developed micro-scissors to clip the eggs surface and a beveled quartz needle to deliver the CRISPR-Cas9 reagents through the clip.

Studies with Doryteuthis pealeii have led to foundational advances in neurobiology, beginning with description of the action potential (nerve impulse) in the 1950s, a discovery for which Alan Hodgkin and Andrew Huxley became Nobel Prize laureates in 1963. For decades D. pealeii has drawn neurobiologists from all over the world to the MBL, which collects the squid from local waters.

Recently, Rosenthal and colleagues discovered extensive recoding of mRNA in the nervous system of Doryteuthis and other cephalopods. This research is under development for potential biomedical applications, such as pain management therapy.

For these reasons, the MBL Cephalopod Programs next goal is to transfer the new knockout technology to a smaller cephalopod species, Euprymna berryi (the hummingbird bobtail squid), which is relatively easy to culture to make genetic strains.

The MBL Cephalopod Program is part of the MBLs New Research Organisms Initiative, which is widening the palette of genetically tractable organisms available for research and thus expanding the universe of biological questions that can be asked.

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First Gene Knockout in a Cephalopod is Achieved at Marine Biological Laboratory by Professor Karen Crawford and Team | Southern Maryland News Net -...

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BOSTON, July 28, 2020 /PRNewswire/ -- Breakthrough Properties announced that leading gene editing company, CRISPR Therapeutics, has signed a 263,500-square-foot lease agreement for the development referred to as "The 105," located at 105 West First Street and scheduled for completion in early 2022. The full-building lease transaction was executed just one year after acquisition of the site in July 2019 and four months after groundbreaking.

Breakthrough Properties was launched in 2019 by Tishman Speyer, one of the world's leading real estate developers and owners, and Bellco Capital, a prominent biotechnology investment firm. Breakthrough was formed to acquire, develop and operate the finest life science properties in leading technology centers around the world, supporting scientific innovation across biotechnology, agriculture and nutrition. On The 105 project, the company has been working closely with Tishman Speyer's Boston-based team every step of the way, from site selection to future project completion.

The 105 has been designed by the Payette architecture firm to be a best-in-class laboratory building to accommodate tenants at the forefront of life-changing science. Conveniently located near the Red Line's Broadway Station, it will feature a tailored array of amenities, including a fitness facility and outdoor terraces accessible from two floors. LEED Gold and Fitwel certifications will be sought.

Breakthrough Properties Chief Executive Officer Dan Belldegrun commented, "Our mission at Breakthrough is to deliver cutting edge facilities and environments that support companies at the forefront of life-changing science.We are thrilled to partner with CRISPR, one of the world's emerging leaders in the biotech industry, as it continues to develop therapies that change the way we fight disease. There has never been a more important time to focus on the scientific innovation and we're honored to play a small but supportive role in CRISPR's exciting future."

Tishman Speyer President & Chief Executive Officer Rob Speyer said, "With Breakthrough Properties, we are combining Tishman Speyer's global property development capabilities with Bellco Capital's recognized life sciences expertise and strong relationships. It's already proving to be a great collaboration. We believe Breakthrough will become a major global player in creating the next generation of research facilities, where important scientific advances will take place to benefit people around the world."

Since its founding in 2013, CRISPRhas quickly grown to be a leading gene-editing company focused on developing transformative gene-based medicines for serious diseases. CRISPR has established a portfolio of therapeutic programs across a broad range of disease areas, including hemoglobinopathies, oncology, regenerative medicine and rare diseases. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom.

Brokerage firm Cushman & Wakefield represented CRISPR, while Breakthrough was represented by Newmark Knight Frank.

About Breakthrough Properties (btprop.com)

Breakthrough Properties is a life science real estate development company that leverages cross-sector collaboration to deliver environments that foster innovation and scientific breakthroughs. Breakthrough combines Tishman Speyer's decades of global real estate development experience with Bellco Capital's industry-making biotechnology entrepreneurship to reimagine environments where companies can create life-changing therapies for patients. At Breakthrough, we seek to be a home for scientific discovery and innovation because we understand what you do, how you do it and why you do it.

Environments that foster innovation. Discoveries that transform lives.

For more information, please visit http://www.btprop.com.

SOURCE Breakthrough Properties

http://www.btprop.com

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Breakthrough Properties Executes Full-Building Lease With Leading Gene Editing Company for Next Generation Life Science Development in Boston's...

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Dublin, July 31, 2020 (GLOBE NEWSWIRE) -- The "Cell and Gene Therapy Tools, and Reagents: Global Markets" report has been added to ResearchAndMarkets.com's offering.

Gene and cell therapy are emerging as important tools to treat human health. Techniques such as CAR-T therapy have emerged as key ways of treating many different types of cancers. The promise of gene therapy using technologies such as CRISPR is starting to be realized in clinical trials, and markets are scaling up to treat other diseases as well, particularly rare gene-based diseases. As these therapies are coming to the fore, a new market for tools to develop these therapies using standard methodologies is emerging. This report will cover what those tools are, how they impact the larger life science tools market, and how they will evolve over the next five years.

The scope of this study encompasses an investigation of the market's cell and gene therapy tools such as GMP proteins, media, cell separation and activation reagents, viral and non-viral, cytokine release syndrome monitoring products, GMP antibodies, leukapheresis instrumentation, immunoassays (multiplex and singleplex) and bioreactors. This research analyzes each tool type, determines its current market status, examines its impact on future markets, and presents forecasts of growth over the next five years. Technological issues, including the latest trends, are discussed. The report analyzes the industry on a worldwide basis, from both application and demand perspectives, in the major regions of the world.

The Report Includes:

Key Topics Covered:

Chapter 1 Introduction

Chapter 2 Summary and Highlights

Chapter 3 Market and Technology Background

Chapter 4 Market Breakdown by Region

Chapter 5 Market Breakdown by End User

Chapter 6 Government Regulations

Chapter 7 Patent Review/New Developments

Chapter 8 Analysis of Market Opportunities

Chapter 9 Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/86txdi

About ResearchAndMarkets.comResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Global Market Trends in the Cell and Gene Therapy Tools and Reagents Market 2020-2024 - GlobeNewswire

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Be wary of stem cell therapy as a preventative treatment for COVID-19, warns Laertis Ikonomou, a UB expert on stem cell and gene therapies.

While stem cell therapy, such as bone marrow transplantation, may be used to treat a limited number of diseases and conditions, there are currently no clinically tested or government-approved cell therapies available for the treatment or prevention of COVID-19, says Ikonomou, associate professor of oral biology in the School of Dental Medicine.

He urges the public to exercise caution as the nation experiences a rise in businesses offering direct-to-consumer, unproven and unsafe stem cell therapies that promise to prevent COVID-19 by strengthening the immune system or improving overall health.

What these patients are actually sold is false hope, he says. These businesses are continuously transforming and reinventing themselves, but the common thread is that they offer potentially dangerous treatments based on unproven science.

Ikonomou is also the chair of the International Society for Cell and Gene Therapy (ISCT) Presidential Task Force on the Use of Unproven and/or Unethical Cell and Gene Therapy.

Stem cell therapy involves the conversion of stem cells into specific types of cells, such as heart or blood cells. These cells are then transplanted into a patient to promote healing.

While there are companies that carefully develop cell-based treatments following established regulatory and ethical standards, there has also been an explosion of businesses since the mid-2000s that advertise directly to consumers and evade regulations to provide unsafe and ineffective treatments, he says.

These businesses operate in gray regulatory areas, frequently branding stem cell therapies as medical interventions rather than therapeutic drugs to avoid the need for U.S. Food and Drug Administration (FDA) approval, Ikonomou says, adding that according to published research, there are more than 1,000 of these unsafe businesses in the U.S.

They offer purported stem cell therapies for nearly every condition imaginable, from diabetes and autism to Alzheimers disease. There are also reports of people suffering physical harm including blindness and death from unsafe stem cell interventions, such as drawing and reinjecting patients with their own fat cells, he says.

Im not surprised that a lot of these businesses went into COVID treatments, says Ikonomou. They went where the money is and took advantage of peoples fears.

The treatments range in price from a few thousand to tens of thousands of dollars, and often patients are encouraged to receive the expensive infusions every few months. Many people go into severe debt to acquire these ineffective treatments, he says.

This year, the FDA has issued several letters to offending businesses, including those advertising cell therapies for COVID-19, says Ikonomou. The Federal Trade Commission has also cracked down on misleading advertising from stem cell therapy clinics, he says.

However, many of these clinics are small and difficult to track. Patient prudence is key to avoiding harmful interventions, he says.

Ikonomou shares a list of steps the public can take to ensure a stem cell therapy is safe, proven and ethical.

Ikonomou also urges patients to share any questions they have with their physicians, who often are the gatekeepers for medical treatment. His best advice to patients: If something sounds too good to be true, it probably isnt true.

For information on safe and ethical cell therapies, visit the ISCT website.

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Unproven 'stem cell' therapies for COVID-19 pose harm to public, says UB expert - UB Now: News and views for UB faculty and staff - University at...

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The first patient to undergo a bone marrow transplant in the UAE has told of how the stem cell treatment saved his life.

Abdullah Muhammad had blood cancer diagnosed in 2018 after he began to vomit blood.

Doctors said the health of the 49-year-old electrician from Pakistan had begun to rapidly deteriorate and his only option was to undergo an expensive bone marrow transplant.

I didnt have money to get a transplant and the UAE sponsored my treatment.

"I am happy to be first one to undergo this transplant in the UAE. It wouldnt have been able to afford it in Pakistan, said Mr Muhammad, whose family live in Khanewal District. The father of four received his transplant on July 18 at Sheikh Khalifa Medical City, with the involvement of Abu Dhabi Stem Cell Centre, which is also developing a treatment for Covid-19 patients.

I didnt have money to get a transplant and the UAE sponsored my treatment. I am happy to be first one to undergo this transplant in the UAE

Abdullah Nazir Ahmad Muhammad

The transplant procedure will bring hope to cancer patients in the UAE, who can now seek treatment closer to home.

Known as regenerative medicine, stem cell therapy promotes the repair of abnormal or injured tissue.

Doctors can manipulate the cells into the type the patient needs and inject them where repair is necessary. The cells can be taken from a matching donor or harvested from the patient, treated, and then reintroduced to the body.

The UAE has begun to harness stem cell therapy in recent months to fight the coronavirus, but this was the first time it was used in a transplant in the Emirates.

In Mr Muhammads case, stem cells were harvested from his own blood and were injected back into him after he underwent a short course of chemotherapy. This is called an autologous bone marrow transplant.

Dr Fatima Al Kaabi, executive director of Abu Dhabi Bone Marrow Transplant Programme, said the treatment was a milestone for the UAE.

Most of these cases travel abroad so, in the near future, we will be self-sufficient and efficient to take care of our own with the highest calibre of medical care and international standards," she said.

Dr Al Kaabi said that to harvest the cells, Mr Muhammad was injected with a stimulant that prompted the stem cells to leave his bone marrow and enter his bloodstream.

His blood was drawn using a machine similar to one used in kidney dialysis to separate the plasma containing the stem cells from the blood.

The plasma was safely stored while Mr Muhammad underwent chemotherapy to wipe out the bone marrow and give way to the new cells, she said.

Mr Muhammad was kept in a sterile area for 10 to 15 days to prevent him from catching infections until the stem cells were returned to his body. The reintroduction of cells to his body took about 20 minutes.

After the successful autologous bone marrow transplant, Abu Dhabi Stem Cells Centre aims to begin carrying out transplants from related donors.

For now only we are doing autologous transplants, where the patient and donor are the same person, but in the near future we will not only do related transplantation but more complicated ones where donors are not related to the patient, said Dr Yendry Ventura, general manager of the stem cell centre and director of Abu Dhabi Bone Marrow Transplant Programme.

Sheikh Khalifa Medical City and the Department of Health Abu Dhabi are working on a stem cell donor registry.

The next transplant is scheduled for a few weeks' time, with the centre aiming to perform 10 transplants before the related transplant programme. It also plans to begin a transplant programme for children.

We want to provide the people of UAE a programme that is not only comparable with any other programme in the rest of the world but also offers customised and personalised treatment, Dr Ventura said.

Mr Muhammad, who has been in hospital since July 8, will be released in a few days and plans to return to Pakistan in October to see his family.

Updated: July 29, 2020 03:10 PM

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UAE's first bone marrow transplant patient tells of life-saving treatment - The National

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Be the Match: Every three minutes, someone is diagnosed with a life-threatening blood cancer or blood disease, such as leukemia, lymphoma or sickle cell disease, and you might be able to help.

For those thousands, a cure exists, though about 70% of patients dont find it within their own families.

Thats why the College of the Canyons Biology Club is partnering with Be The Match to host a drive-in community registry event, where the community is invited to register to be a potential match for someone in need of a life-saving stem cell transplant through a simple swab test.

The science of stem cells is incredible, and it can actually cure over 70 different life-threatening diseases, said Christine Mantilla, member engagement, enrollment and experience specialist at Be The Match. A small population of stem cells from a donor can regenerate an entire bodys worth of bone marrow.

Biology Club President Brian Estarella-Murphy has been on the registry for two years, eagerly awaiting the day hes a match.

For Estarella-Murphy, its personal, as he has had two close friends with blood disorders and has seen firsthand the struggle of some of these patients.

I interned and shadowed at a hospital and a clinic, he added. Ive seen many patients come in that are on their last chance of life, and Id love to be able to give someone that possibility of living their full life without having to worry.

Being involved with the organization has actually changed his career aspirations, motivating him to go into the research side of medicine.

Im a cellular biology major, and the type of research that they do on these swab tests are exactly what I want to do in the future, he added. I want to pick apart these cells to see how I can help advance medicine (to create) life-saving treatment.

So upon taking up presidency of the club, Estarella-Murphy knew the first thing he wanted to do was partner with Be The Match.

Not only has the current health crisis been an extremely difficult time for those in need of transplants, as they are immunocompromised, but without community registry events, less have been joining.

This is an action that people can take in 10 minutes for free that can directly save someones life, Mantilla said.

Those interested in being a possible match, can drive up to the event from the safety of their car, where volunteers assist them in registering and administering the 10-second swab test on the inner cheeks while following all health and safety measures.

Were just doing a quick, little swab, and then we send your data out, and we do some research to see if youre possibly even a match, Estarella-Murphy said. Our goal out of this event is to sign up as many individuals as possible, so that through further research, we can then match them to patients awaiting dire transfusions.

Only about 1-in-430 U.S. registry members go on to donate bone marrow or peripheral blood stem cells to a patient.

Thats 0.2% and just emphasizes the need for as many people as possible to be available on the registry because it is so challenging to find that match, Mantilla said, adding that not everyone has an equal chance at finding a match, as ethnic heritage plays a significant role. Right now, the registry is overwhelmingly white. Its a health disparity that is pretty serious as far as equity of access to treatment.

The most common way to harvest stem cells is through a peripheral blood stem cell donation, involving a specialized blood draw, where stem cells are processed from your blood before the blood is returned to the donor.

Your stem cells are a renewable resource, so the donors stem cells actually regenerate in about four to six weeks, Mantilla added.

The event is scheduled 2-6 p.m. Aug. 14, with the location to be determined. For more information, visit biologyclubcoc.org/bethematch. To join the registry, visit join.bethematch.org/COCBio or text COCBio to 61474.

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It's Time to 'Be The Match,' Help Save a Life - SCVNEWS.com

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DEAR DOCTOR:Whats the connection between the novel coronavirus and the thymus gland? A friend of ours who is a doctor says its probably what keeps young kids from getting so sick. Ive never even heard of the thymus. What does it have to do with coronavirus?

DEAR READER: From the earliest days of the novel coronavirus pandemic, the data revealed a puzzling disparity. Older adults were at increased risk of grave illness when infected with the virus, but children seemed to have a certain level of protection. And while it has since become clear that children can indeed become seriously ill if they become infected, they do so at far lower rates than adults. The reasons for this are still being investigated, but some researchers have recently suggested the role of the thymus gland as a possible factor.

If you place your finger at the notch at the top of your breast bone and draw a vertical line downward a few inches, youve traced the location of your thymus. Its made up of two roughly triangular lobes, which sit behind the breastbone and between the lungs. The thymus has several functions, but perhaps its most important role is to help produce the cells that will become T-lymphocytes, or T-cells. (The T stands for thymus-derived.) These are white blood cells that protect the body from bacteria, fungi, viruses and other pathogens.

T-cells, which are the ninjas of the immune system, start out in the bone marrow as stem cells. The immature stem cells exit the marrow, move through the blood and enter a specific region of the thymus. There, they undergo a complex process that teaches them how to recognize a wide range of potentially dangerous and deadly invaders. As T-cells, their job is to circulate throughout the body and, when they encounter the molecular signature of the pathogen theyve been trained to recognize, to attack. T-cells also activate other immune cells, produce proteins known as cytokines and have a role in regulating immune response.

The thymus is unique in that it reaches maturity in utero and is at its largest and most active in children. Starting at puberty, it gradually becomes less active, and the glandular tissue begins to shrink. This continues throughout a persons life. By the time someone has reached their mid-60s, the thymus is largely inactive. By their mid-70s, the gland has been mostly replaced with fat. This decrease in thymus function is believed to be one of the reasons that, in their later years, older adults become more susceptible to disease and infection.

Emerging research into COVID-19 has shown a marked decrease in the number of T-cells in some gravely ill patients. Scientists are now asking whether age-related thymus decline, which means T-cells arent quickly replaced, may play a role in the severity of illness seen in older adults. The flip side of this is whether, due to their robust production of T-cells, childrens immune systems are able to stay one step ahead of the novel coronavirus. Its only a working theory, but it shows promise, and research into how this may affect and inform treatment continues.

Eve Glazier, M.D., MBA, is an internist and associate professor of medicine at UCLA Health. Elizabeth Ko, M.D., is an internist and assistant professor of medicine at UCLA Health. Send your questions to askthedoctors@mednet.ucla.edu, or write: Ask the Doctors, c/o UCLA Health Sciences Media Relations, 10880 Wilshire Blvd., Suite 1450, Los Angeles, CA, 90024. Owing to the volume of mail, personal replies cannot be provided.

We are making critical coverage of the coronavirus available for free. Please consider subscribing so we can continue to bring you the latest news and information on this developing story.

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Thymus may play role in severity of coronavirus - Cumberland Times-News

Recommendation and review posted by Bethany Smith

Dear Doctor: Whats the connection between the novel coronavirus and the thymus gland? A friend of ours who is a doctor says its probably what keeps young kids from getting so sick. Ive never even heard of the thymus. What does it have to do with coronavirus?

Dear Reader: From the earliest days of the novel coronavirus pandemic, the data revealed a puzzling disparity. Older adults were at increased risk of grave illness when infected with the virus, but children seemed to have a certain level of protection. And while it has since become clear that children can indeed become seriously ill if they become infected, they do so at far lower rates than adults. The reasons for this are still being investigated, but some researchers have recently suggested the role of the thymus gland as a possible factor.

If you place your finger at the notch at the top of your breast bone and draw a vertical line downward a few inches, youve traced the location of your thymus. Its made up of two roughly triangular lobes, which sit behind the breastbone and between the lungs. The thymus has several functions, but perhaps its most important role is to help produce the cells that will become T-lymphocytes, or T-cells. (The T stands for thymus-derived.) These are white blood cells that protect the body from bacteria, fungi, viruses and other pathogens.

T-cells, which are the ninjas of the immune system, start out in the bone marrow as stem cells. The immature stem cells exit the marrow, move through the blood and enter a specific region of the thymus. There, they undergo a complex process that teaches them how to recognize a wide range of potentially dangerous and deadly invaders.

As T-cells, their job is to circulate throughout the body and, when they encounter the molecular signature of the pathogen theyve been trained to recognize, to attack. T-cells also activate other immune cells, produce proteins known as cytokines and have a role in regulating immune response.

The thymus is unique in that it reaches maturity in utero and is at its largest and most active in children. Starting at puberty, it gradually becomes less active, and the glandular tissue begins to shrink. This continues throughout a persons life. By the time someone has reached their mid-60s, the thymus is largely inactive. By their mid-70s, the gland has been mostly replaced with fat. This decrease in thymus function is believed to be one of the reasons that, in their later years, older adults become more susceptible to disease and infection.

Emerging research into COVID-19 has shown a marked decrease in the number of T-cells in some gravely ill patients. Scientists are now asking whether age-related thymus decline, which means T-cells arent quickly replaced, may play a role in the severity of illness seen in older adults. The flip side of this is whether, due to their robust production of T-cells, childrens immune systems are able to stay one step ahead of the novel coronavirus. Its only a working theory, but it shows promise, and research into how this may affect and inform treatment continues.

Dr. Eve Glazier, MBA, is an internist and assistant professor of medicine at UCLA Health. Dr. Elizabeth Ko is an internist and primary care physician at UCLA Health. Send your questions to askthedoctors@mednet.ucla.edu, or write: Ask the Doctors, c/o Media Relations, UCLA Health, 924 Westwood Blvd., Suite 350, Los Angeles, CA, 90095.

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ASK THE DOCTOR: Theory suggests thymus plays role in severity of COVID - Lufkin Daily News

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A researcher holding up a container of stem cells.Photo: Spencer Platt (Getty Images)

A team of neurologists are raising a red flag over risky and unproven stem cell therapies. Their new study suggests that their fellow doctors are frequently asked about these controversial procedures by desperate patients. Many doctors are also seeing patients with serious complications from stem cell therapy, including seizures, infections, and spinal tumors.

Stem cells are the raw material that our body uses to replace other damaged or aging cells. Scientists have been hopeful about the potential to use these cells as a way to surpass the bodys natural regenerative ability and treat currently incurable diseases, including degenerative neurological disorders like multiple sclerosis.

But so far, there are only a few proven uses in medicine for stem cells, such as bone marrow transplants to treat certain blood cancers. Most of the evidence for their claimed benefits elsewhere is either limited or entirely lacking. These caveats havent stopped the consumer stem cell industry from growing, though, with clinics offering treatments on an experimental basis, often without express approval from the Food and Drug Administration.

This new study, published Wednesday in the Annals of Neurology, was a survey of over 200 neurologists across the country, including a third who specialize in multiple sclerosis. Among other questions, the neurologists were asked if they had ever come across patients who had used or wanted to use stem cells to treat their health problems. For those with patients previously given stem cells, they were also asked about any health complications they had seen that were possibly linked to stem cell treatment.

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Almost 90% of the neurologists surveyed said they had been asked by a patient or a close family member about stem cells, a rate that was slightly higher for those with patients who had incurable conditions like multiple sclerosis. Many of those conversations involved patients asking for the neurologists blessing to go ahead with stem cell therapy. But only 28% of the doctors reported feeling completely prepared to talk to their patients about the topic.

One of the studys points is that these discussions are happening in more than 90% of every outpatient neurology clinic, but many of the doctors are not ready, said senior author Jamie Imitola, a neuroscientist at the University of Connecticut who specializes in multiple sclerosis and stem cell research. And if theyre not fully prepared, then thats shocking, because these are the doctors that should be up to date.

Two-thirds of neurologists also said they had treated someone who had already gotten a stem cell therapy, while 25% of respondents said they had come across a patient with complications thought to be related to a stem cell therapy. Some of the specific complications listed have been reported before, but Imitola said the percentage of doctors who reported any at all is much higher than you would expect from the small number of case reports available. It suggests that side-effects linked to these procedures may be more common than previously thought.

Ive had doctors call us and tell us that patients dont want to report problems with their stem cell treatment, he added. They feel embarrassed, number one, because they have a complication or two, because they feel they were ripped off.

The long list of complications reported by doctors in the survey included stroke, paralysis, bloodborne infections like hepatitis C, and several deaths. In two cases, doctors reported the emergence of a tumor along the spinal cord; in one case, the tumor had cells that didnt seem to belong to the patient, suggesting the donors cells had somehow turned cancerous. In other cases, the therapy seemed to worsen the progression of the disease it was meant to treat. Still other patients had spent tens of thousands of dollars on their treatment, with no lasting improvement.

The surveys findings are limited, especially when it comes to the reported complications, since theyre reliant on the recollection of doctors rather than detailed case reports sourced from the medical records of patients.. But the study points to all sorts of systemic problems, Imitola said. Many of these patients are understandably looking for a miracle, and theres a growing mistrust of science in general, he noted. That makes the wondrous claims peddled by many clinics selling stem cell therapy look all the more appealing. And it makes the relationship between a doctor and their patient all the more important to get right.

There is a sense around the country that scientists are hiding new therapies for this or that. That we dont want people to get stem cell therapy, because there is some ulterior motive, he said. The reality is that for us to translate stem cells into something real, its going to require a great deal of work. Because, ultimately, we want to provide people the best therapy, the best opportunities.

To help neurologists be more prepared to talk about stem cells, the researchers have created a patient-friendly handout based on their findings, translated into 17 languages. Imitola has also set up a patient registry where doctors and patients can anonymously report any possible complications linked to stem cell therapy.

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Shady Stem Cell Therapies Can Cause Tumors, Infections, and Death, Doctors Report - Gizmodo

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By Siddhi Jain

New Delhi, Jul 30 (IANSlife): Cancer takes innumerable lives each year, and so does the unavailability of cancer treatment due to lack of funds. 33-year-old Nushafreen Palsetia, a software engineer based in Mumbai, was recently diagnosed with a very aggressive form of cancer. Despite a relapse, what helped was life-saving support pouring from over 1,700 donors.

Nushafreen was first diagnosed with Non-Hodgkin Lymphoma in April 2019. After a year of enduring aggressive treatment, she tried to get back to her normal life and work. Unfortunately, Non-Hodgkin Lymphoma, Diffuse Large B Cell Lymphoma (DLBCL), cancer relapsed in her liver in May 2020 which was an unexpected major shock, leaving her and all of her family overwhelmed.

Doctors planned to perform an autologous (her own stem cells) bone marrow transplant in India after the chemotherapy but further tests showed involvement of the bone marrow as well. Hence, her treating doctor recommended the modern CAR-T Cell therapy treatment, available only in the USA, UK, Israel, and a few European countries.

Nushafreen's family found Israel as the most affordable option as compared to all other countries offering the treatment. They reached out to Sheba Medical Centre in Israel which estimated the medical expenses as 200,000 USD (approximately Rs 1.5 crore). The treatment will require Nushafreen to be hospitalized for a month or more for the response to treatment and immediate follow up.

As Nushafreen's family couldn't afford the high medical expenses, an ImpactGuru crowdfunding campaign was initiated. In two weeks, Nushafreen's ImpactGuru.com Page has raised over Rs 1 crore from 1750 donors. The platform raises money online for medical expenses via crowdfunding such as cancer, transplants, and accidents.

According to the co-founder and CEO of the healthcare crowdfunding platform, Piyush Jain, "Crowdfunding is driven by a culture of generosity, it allows people to raise money quickly in a hassle free manner without any payback liability. This is a new record for our platform with a single patient's family being able to raise more than Rs 1 crore. We hope Nushafreen recovers soon and more patients come forward to utilize our platform in their time of need to get the best available treatment for critical illnesses."

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Crowdfunding to the rescue for this cancer patient - Daijiworld.com

Recommendation and review posted by Bethany Smith

Stem Cells Market Overview 2020 2025

This has brought along several changes in This report also covers the impact of COVID-19 on the global market.

The risingtechnology in Stem Cells Marketis also depicted in thisresearchreport. Factors that are boosting the growth of the market, and giving a positive push to thrive in the global market is explained in detail.

Stem cells are a class of undifferentiated cells that are able to differentiate into specialized cell types. Commonly, stem cells come from two main sources: Embryos formed during the blastocyst phase of embryological development (embryonic stem cells) and Adult tissue (adult stem cells). Both types are generally characterized by their potency, or potential to differentiate into different cell types (such as skin, muscle, bone, etc.). This report studies the Stem Cell Therapy. Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition. Bone marrow transplant is the most widely used stem-cell therapy, but some therapies derived from umbilical cord blood are also in use.

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Key Competitors of the Global Stem Cells Market are: , Osiris Therapeutics, NuVasive, Chiesi Pharmaceuticals, JCR Pharmaceutical, Pharmicell, Medi-post, Anterogen, MolMed, Takeda (TiGenix)

Historical data available in the report elaborates on the development of the Stem Cells on national, regional and international levels. Stem Cells Market Research Report presents a detailed analysis based on the thorough research of the overall market, particularly on questions that border on the market size, growth scenario, potential opportunities, operation landscape, trend analysis, and competitive analysis.

Major Product Types covered are:AllogeneicAutologous

Major Applications of Stem Cells covered are:Musculoskeletal DisorderWounds & InjuriesCorneaCardiovascular DiseasesOthers

This study report on global Stem Cells market throws light on the crucial trends and dynamics impacting the development of the market, including the restraints, drivers, and opportunities.

To get this report at a profitable rate.: https://reportsinsights.com/discount/13815

The fundamental purpose of Stem Cells Market report is to provide a correct and strategic analysis of the Stem Cells industry. The report scrutinizes each segment and sub-segments presents before you a 360-degree view of the said market.

Market Scenario:

The report further highlights the development trends in the global Stem Cells market. Factors that are driving the market growth and fueling its segments are also analyzed in the report. The report also highlights on its applications, types, deployments, components, developments of this market.

Highlights following key factors:

:-Business descriptionA detailed description of the companys operations and business divisions.:-Corporate strategyAnalysts summarization of the companys business strategy.:-SWOT AnalysisA detailed analysis of the companys strengths, weakness, opportunities and threats.:-Company historyProgression of key events associated with the company.:-Major products and servicesA list of major products, services and brands of the company.:-Key competitorsA list of key competitors to the company.:-Important locations and subsidiariesA list and contact details of key locations and subsidiaries of the company.:-Detailed financial ratios for the past five yearsThe latest financial ratios derived from the annual financial statements published by the company with 5 years history.

Our report offers:

Market share assessments for the regional and country level segments. Market share analysis of the top industry players. Strategic recommendations for the new entrants. Market forecasts for a minimum of 9 years of all the mentioned segments, sub segments and the regional markets. Market Trends (Drivers, Constraints, Opportunities, Threats, Challenges, Investment Opportunities, and recommendations). Strategic recommendations in key business segments based on the market estimations. Competitive landscaping mapping the key common trends. Company profiling with detailed strategies, financials, and recent developments. Supply chain trends mapping the latest technological advancements.

Access full Report Description, TOC, Table of Figure, Chart, etc. @ https://reportsinsights.com/industry-forecast/Stem-Cells-Market-13815

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Reports Insights is the leading research industry that offers contextual and data-centric research services to its customers across the globe. The firm assists its clients to strategize business policies and accomplish sustainable growth in their respective market domain. The industry provides consulting services, syndicated research reports, and customized research reports.

Contact US:

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Link:
Stem Cells Market is expected to pick up in healthy CAGR by 2020-2025 Top companies | Chiesi Pharmaceuticals, JCR Pharmaceutical, Pharmicell,...

Recommendation and review posted by Bethany Smith

A year ago, measles was in the news. We saw more cases in 2019 than we had in more than a decade.

Today, its a virus that has become a global pandemic.

We have a vaccine for measles and were working toward one for the coronavirus.

Having a vaccine will go a long way toward relieving the angst of society. You see, vaccines are one way to achieve herd immunity. (Natural infection is the other.) This is when a large portion of a community becomes immune to a disease. When that happens, the spread of the disease becomes less likely.

This vaccine will happen. In part, because we want it to and because of the amazing advances weve made in the world of medicine.

Were seeing amazing changes in precision medicine today. As the name implies, it has made the practice of medicine more precise. We are sequencing genes to better understand disease at the cells. This has allowed us to personalize medicine.

Per the U.S. Centers for Disease Control and Prevention, sickle cell disease (SCD) affects 100,000 people in the U.S. alone. A disproportionate percentage of them are African American.

Normally healthy round red blood cells become hard and sticky. They look like a C-shaped tool called a sickle.

The cells die early, causing a constant shortage of red blood cells. The shape of these cells also causes them to get stuck and clog blood flow. This results in pain, infection and even stroke.

Gene therapy modifies someones DNA to treat disease.

In 2017, doctors announced they had used the therapy to cure a teens SCD and the painful symptoms that went with it.

They took some of his bone marrow stem cells, gave the cells extra versions of a needed gene and put them back in his body.

The blood tests show hes cured.

Medical professionals are excited about the prospects of gene therapy for other ailments like hemophilia and cystic fibrosis as well.

Immunotherapy is a treatment that helps your immune system fight cancer. Its a biological therapy. This means it uses substances made from living organisms.

One reason cancer cells thrive is they know how to hide from your immune system. Some of the immunotherapies were working on can mark cancer cells so its easier for your system to find them. Others boost your immune system, so it just works better against the diseased cells.

One version of this is T-cell therapy.

Kids who get leukemia most often suffer from ALL, or acute lymphoblastic leukemia.

In 2017, scientists conducted a trial on patients who had a less than 20% chance of survival using current treatments. Of those patients, 93% using the therapy achieved complete initial remission. Half remained in complete remission a year later.

Testing continues, but this is a huge leap forward.

Human ingenuity will help us beat the coronavirus, too.

Weve been fighting viruses for a long time. We have hundreds of years of insight. And weve never wanted a vaccine more than we do today.

Remdesivir is one of our most promising drugs to fight the coronavirus. Gilead Sciences first created it to treat Ebola, another virus.

Precision medicine will help, too.

More than 1,800 COVID-19 trials are taking place around the world right now. The U.S. is doing the most, but this is truly a global effort.

We will achieve our goal.

Never bet against us.

Further, if there ever was one, now is the time to come together. To use more compassion than (hate-filled) passion. The results will surprise us if we embrace kindness today.

Good investing,

Brian ChristopherEditor, Profit Line

P.S. In his Automatic Fortunes investing service, Ian King follows the latest advancements in precision medicine. He has already invested in names that benefit from it. Precision medicine will be a key player in the rise of the Great American Reset. Click here to learn more. Youll be happy that you did.

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Precision Medicine Achieves Our Goals and Makes Money in Stocks - Banyan Hill Publishing

Recommendation and review posted by Bethany Smith

On July 24, the Food and Drug Administration (FDA) granted accelerated approval of a new CAR-T therapy, Tecartus (brexucabtagene autoleucel), for adults with mantle cell lymphoma who have not responded to or who have relapsed after other kinds of treatment.

Tecartus (formerly known as KTE-X19), manufactured by Kite, a Gilead Company, is a living drug that reprograms a patients own T cells to fight cancer. About two thirds of participants treated in the ZUMA-2 study experienced complete remission, which in a majority of cases lasted at least a year.

"This approval is yet another example of customized treatments that use a patients own immune system to help fight cancer, Peter Marks, MD, PhD, director of the FDAs Center for Biologics Evaluation and Research, said in a press release. Were seeing continued advances in the field of gene therapy and remain committed to supporting innovation in this promising new area of medicine.

Mantle cell lymphoma (MCL) is an uncommon form of non-Hodgkinlymphoma that involves overgrowth of abnormal B cells in lymph nodes. By the time it is diagnosed, MCL has often spread to the bone marrow and other organs, and it is frequently refractory (nonresponsive) to existing treatments or relapses following a period of remission.

Chimeric antigen receptor T-cell therapybetter known as CAR-Tinvolves removing a sample of a patients white blood cells, genetically reprogramming the T cells to recognize and attack their cancer, manufacturing a large number of the modified cells in a laboratory and infusing them back into the body. Like Kites previously approved CAR-T Yescarta (axicabtagene ciloleucel), Tecartus targets the CD19 protein on B cells, but the manufacturing process includes greater T-cell selection and lymphocyte enrichment.

The Phase II ZUMA-2 trial enrolled 74 adults with relapsed or refractory MCL in the United States and Europe who had previously tried up to five prior therapies. Six people ended up not receiving Tecartus because of manufacturing failures or death due to disease progression.

The primary analysis included 60 treated participants. Most were men, and the median age was 65. Most had Stage IV metastatic cancer, more than 80% had tried three or more prior treatments and 43% had received a bone marrow stem cell transplant.

A sample of the patients T cells were collected and modified, and they received strong conditioning chemotherapy to kill off some of their existing immune cells to make room for the new ones. They then received a single infusion of the genetically engineered cells.

As described at last years American Society of Hematology Annual Meeting and in The New England Journal of Medicine, the overall response rate, meaning complete or partial remission, was 93%, including 67% with complete responses. After a year of follow-up, 57% were still in remission, as were 43% of those followed for two years. The estimated one-year progression-free survival rate was 61%, and the estimated overall survival rate was 83%.

In a press release announcing the approval, Gilead/Kite gave an updated overall response rate of 87% and a complete response rate of 62%.

Therapies that receive FDA accelerated approval based on overall response rates are expected to undergo further testing in larger trials to confirm clinical benefits, and the agencycan rescind approval if they do not measure up. Tecartus is also currently in Phase I/II trials for acute lymphoblastic leukemia and chronic lymphocytic leukemia.

Treatment with Tecartus was generally safe. The most common severe adverse events were low blood cell counts due to the conditioning chemotherapy and infections. Introducing engineered T cells can trigger a strong immune reaction known as cytokine release syndrome (CRS), which can lead to falling blood pressure and organ failure. As previously reported, 15% of study participants experienced Grade 3 or higher CRS, and 31% developed severe neurological side effects. Based on a larger group of patients, Gilead/Kite updated these figures to 18% and 37%, respectively. These side effects were managed with corticosteroids or Actemra (tocilizumab), and none were fatal.

A risk evaluation and mitigation strategy (REMS) for Tecartus has been approved by the FDA and combined with the YescartaREMS (www.YescartaTecartusREMS.com). This program informs health care professionals about the risks associated with CAR-T therapy and how to manage it.

Tecartus will be produced at Kites commercial manufacturing facility in El Segundo, California. The median turnaround time from T-cell collection to administration of the modified cells is around 15 days, according to the press release. The Kite Konnectprogram offers financial and logistical support for eligible patients receiving Tecartus or Yescarta.

Despite promising advances, there are still major gaps in treatment for patients with MCL who progress following initial therapy, ZUMA-2 lead investigator Michael Wang, MD, of University of Texas MD Anderson Cancer Center, said in the press release. Many patients have high-risk disease and are more likely to keep progressing, even after subsequent treatments. The availability of Tecartus as the first-ever cell therapy for patients with relapsed/refractory MCL provides an important option with a response rate of nearly 90% and early clinical evidence suggesting durable remissions in later lines of therapy.

Click here to learn more about lymphoma.

See original here:
FDA Approves New CAR-T Therapy for Mantle Cell Lymphoma - Cancer Health Treatment News

Recommendation and review posted by Bethany Smith

Companies, Organizations Needed to Host Events

MONTVALE, NJ Because of the current, nationwide surge in COVID-19 infections throughout the Sun Belt, along with the constant importance of bolstering the local blood supply, theres now significant need for residents to donate blood and, if possible, convalescent plasma.

The nonprofit, blood-collection organization Vitalantis offering two local, open-to-the-public donation events during August:

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Vitalant is also seeking companies and organizations throughoutMonmouth Countyto host blood donation events. Information on hosting a donation event is available by clicking here.

Individuals who have recovered from COVID-19 are urged to donate blood plasma. Known as convalescent plasma, this blood component contains antibodies that may provide seriously ill patients an extra boost in fighting the disease.

Vitalant is providing an antibody test which is authorized by the U.S. Food and Drug Administration to all donors; results will be available in private, online donor accounts, approximately two weeks after a donation.There is also a great need for blood platelets small cells in the blood that form clots to prevent bleeding, while also helping with anemia and low blood countsand type O-negative,the universal blood type.

To promote the increase of blood, convalescent plasma, and platelet donations,regular event host companies and organizations many of which put their events on pause due to the pandemic are asked toconsider returning to a consistent schedule of donation events.

FEMA has specifically identified blood donation as an essential and integral component of the emergency support function. Of note, coronavirus cannot be transferred through the blood. And, as always, the blood collection process is safe with noimpact on the donor's immune system. Vitalant staff follows rigorous safety and disinfection protocols at its blood drives and donation centers and have always required individuals to be in good health to donate blood.

Vitalant also maintains four New Jersey blood centers, with hours and street addresses as follows:

Healthy individuals age 16 or older, who weigh at least 110 pounds, may donate blood; 16- and 17-year-olds must have proof of birth date and signed consent forms, either in English or Spanish. Donors should eat a moderate meal prior to donating, and also bring identification featuring their signature.

On occasion, last-minute changes to scheduling for a donation event will occur. As a result, it is recommended that anyone planning to donate blood at a Vitalant donation event call 201-251-3703, toll free, to confirm timing and location details. Additional information about donating blood is also available by visitingwww.vitalant.org.

About Vitalant in New Jersey

A not-for-profit organization that supplies blood and blood products to hospitals in the New Jersey/New York region, Bergen County-based Vitalant (previously Community Blood Services) has been devoted to serving the communitys transfusion medicine needs since 1953. Donations of blood and blood products, umbilical cord blood, stem cells, and bone marrow help to join individuals, organizations, businesses, and entire communities together in partnership to help save lives.

About Vitalant

Arizona-based Vitalant is among the nations oldest and largest transfusion medical organizations in the U.S. Founded in 1943, its blood centers division serves some 700 hospitals across the United States. A founding member of Americas Blood Centers and the AABB (formerly the American Association of Blood Banks), Vitalant also operates biological products distribution services, a quality consulting group, and a world-renowned transfusion medicine research institute. It also is a partner in the operation of high-volume donor testing laboratories.

Visit link:
Vitalant to Hold Two Blood Donation Drives in Monmouth County to Assist Health Care Facilities Hardest Hit by Coronavirus - TAPinto.net

Recommendation and review posted by Bethany Smith

Global PNH and aHUS Market research report helps to explain the market definition, classifications, applications, engagements and business challenges of industry. This PNH and aHUS market report provides key information about the industry, including very helpful and important facts and figures, expert opinions, and the latest developments across the globe. It also provides an overview of PNH and aHUS market, containing global revenue, global production, sales, and CAGR.

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About PNH and aHUS Market:

Some Companies Are Operating in the Global PNH and aHUS Market:

In this study, The Years Considered to Estimate the Market Size of PNH and aHUS:

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PNH and aHUS Market Report Segment by Types:

PNH and aHUS Market Report Segmented by Application:

Geographical Segmentation:

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Key Reasons to Purchase:

PNH and aHUS Market Report Contains Following Points in TOC:

1 PNH and aHUS Market Overview

1.1 Product Overview and Scope of PNH and aHUS

1.2 PNH and aHUS Segment by Type

1.3 PNH and aHUS Segment by Application

1.4 Global PNH and aHUS Market by Region 2020 VS 2026

1.5 Global PNH and aHUS Growth Prospects (2015-2026)

2 Market Competition by Manufacturers

2.1 Global PNH and aHUS Production Capacity Market Share by Manufacturers (2015-2020)

2.2 Global PNH and aHUS Revenue Share by Manufacturers (2015-2020)

2.3 Market Share by Company Type (Tier 1, Tier 2 and Tier 3)

2.4 Global PNH and aHUS Average Price by Manufacturers (2015-2020)

2.5 Manufacturers PNH and aHUS Production Sites, Area Served, Product Types

2.6 PNH and aHUS Market Competitive Situation and Trends

3 Production Capacity by Region (2015-2020)

3.1 Global Production Capacity of PNH and aHUS Market Share by Regions

3.2 Global PNH and aHUS Revenue Market Share by Regions

3.3 Global PNH and aHUS Production Capacity, Revenue, Price and Gross Margin

3.4 North America PNH and aHUS Production

4 Global PNH and aHUS Consumption by Regions

4.1. Global PNH and aHUS Consumption Market Share by Region

4.2 North America

4.3 Europe

4.4 Asia Pacific

4.5 Latin America

5 Production, Revenue, Price Trend by Type (2015-2020)

5.1 Global PNH and aHUS Production Market Share by Type

5.2 Global PNH and aHUS Revenue Market Share by Type

5.3 Global PNH and aHUS Price by Type

5.4 Global PNH and aHUS Market Share by Price Tier (2015-2020): Low-End, Mid-Range and High-End

For Detailed TOC Click Here

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Recommendation and review posted by Bethany Smith

NEW YORK, July 27, 2020 /PRNewswire/ --BrainStorm-Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in developing innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, announced today that the Company will hold a conference call to update shareholders on financial results for the second quarter ended June 30, 2020, and provide a corporate update, at 8:00 a.m., Eastern Daylight Time (EDT), on Wednesday, August 5 2020.

On the call, BrainStorm CEO Chaim Lebovits will present a corporate update, including details on the timeline for the data readout of the Company's Phase 3 pivotal trial studying the safety and efficacy of NurOwn (MSC-NTF cell) in people with ALS. In addition, Dr. Revital Aricha. Brainstorm's Vice President of Research and Development, will provide an R&D update including recently announced data from the Company's groundbreaking preclinical study evaluating NurOwn-derived exosomes for the treatment of COVID-19 ARDS.

Thereafter, senior management officers will join the call for a Q&A session. Participants are encouraged to submit their questions prior to the call by sending them to:q@brainstorm-cell.com. Questions should be submitted by5:00 p.m. EDT,Monday, August 3, 2020.

Teleconference Details BRAINSTORM CELL THERAPEUTICS 2Q 2020

The investment community may participate in the conference call by dialing the following numbers:

Participant Numbers:

Toll Free: 877-407-9205International: 201-689-8054

Those interested in listening to the conference call live via the internet may do so by visiting the "Investors & Media" page of BrainStorm's website atwww.ir.brainstorm-cell.comand clicking on the conference call link.

Those that wish to listen to the replay of the conference call can do so by dialing the numbers below. The replay will be available for 14 days.

Replay Number:

Toll Free: 877-481-4010International: 919-882-2331Replay Passcode: 36017

Teleconference Replay Expiration:

Wednesday, August 19, 2020

About NurOwn

NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently received acceptance from theU.S. Food and Drug Administration(FDA) to initiate a Phase 2 open-label multicenter trial in progressive multiple sclerosis (MS) and initiated enrollment inMarch 2019.

AboutBrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc.is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from theU.S. Food and Drug Administration(FDA) and theEuropean Medicines Agency(EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at sixU.S.sites supported by a grant from theCalifornia Institute for Regenerative Medicine(CIRM CLIN2-0989). The pivotal study is intended to support a filing forU.S.FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently receivedU.S.FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive multiple sclerosis (MS). The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment inMarch 2019. For more information, visit the company's website atwww.brainstorm-cell.com.

Story continues

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, regulatory approval of BrainStorm's NurOwn treatment candidate, the success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Investor Relations:Preetam Shah, MBA, PhDChief Financial OfficerBrainStorm Cell Therapeutics Inc.Phone: +1-862-397-1860pshah@brainstorm-cell.com

Media:Paul TyahlaSmithSolvePhone: +1-973-713-3768Paul.tyahla@smithsolve.com

View original content:http://www.prnewswire.com/news-releases/brainstorm-cell-therapeutics-to-announce-second-quarter-financial-results-and-provide-a-corporate-and-rd-update-301100141.html

SOURCE Brainstorm Cell Therapeutics Inc

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BrainStorm Cell Therapeutics to Announce Second Quarter Financial Results and Provide a Corporate and R&D Update - Yahoo Finance

Recommendation and review posted by Bethany Smith

Cell Transplant Market Overview 2020 2025

This has brought along several changes in This report also covers the impact of COVID-19 on the global market.

The risingtechnology in Cell Transplant Marketis also depicted in thisresearchreport. Factors that are boosting the growth of the market, and giving a positive push to thrive in the global market is explained in detail.

Cell Transplant is a procedure in which cells, often stem cells or cells that can be induced to become pluripotent stem cells, are transferred to a site where the tissue is damaged or diseased. The transfer can occur within an individual (autologous transplantation), between individuals, or between species.

Get a Sample PDF copy of the report @ https://reportsinsights.com/sample/13951

Key Competitors of the Global Cell Transplant Market are: , Regen Biopharma, Global Cord Blood Corporation, CBR Systems, Escape Therapeutics, Cryo-Save, Lonza Group, Pluristem Therapeutics, Stemedica Cell Technology

Historical data available in the report elaborates on the development of the Cell Transplant on national, regional and international levels. Cell Transplant Market Research Report presents a detailed analysis based on the thorough research of the overall market, particularly on questions that border on the market size, growth scenario, potential opportunities, operation landscape, trend analysis, and competitive analysis.

Major Product Types covered are:Peripheral Blood Stem Cells Transplant (PBSCT)Bone Marrow Transplant (BMT)

Major Applications of Cell Transplant covered are:HospitalsClinicsOthers

This study report on global Cell Transplant market throws light on the crucial trends and dynamics impacting the development of the market, including the restraints, drivers, and opportunities.

To get this report at a profitable rate.: https://reportsinsights.com/discount/13951

The fundamental purpose of Cell Transplant Market report is to provide a correct and strategic analysis of the Cell Transplant industry. The report scrutinizes each segment and sub-segments presents before you a 360-degree view of the said market.

Market Scenario:

The report further highlights the development trends in the global Cell Transplant market. Factors that are driving the market growth and fueling its segments are also analyzed in the report. The report also highlights on its applications, types, deployments, components, developments of this market.

Highlights following key factors:

:-Business descriptionA detailed description of the companys operations and business divisions.:-Corporate strategyAnalysts summarization of the companys business strategy.:-SWOT AnalysisA detailed analysis of the companys strengths, weakness, opportunities and threats.:-Company historyProgression of key events associated with the company.:-Major products and servicesA list of major products, services and brands of the company.:-Key competitorsA list of key competitors to the company.:-Important locations and subsidiariesA list and contact details of key locations and subsidiaries of the company.:-Detailed financial ratios for the past five yearsThe latest financial ratios derived from the annual financial statements published by the company with 5 years history.

Our report offers:

Market share assessments for the regional and country level segments. Market share analysis of the top industry players. Strategic recommendations for the new entrants. Market forecasts for a minimum of 9 years of all the mentioned segments, sub segments and the regional markets. Market Trends (Drivers, Constraints, Opportunities, Threats, Challenges, Investment Opportunities, and recommendations). Strategic recommendations in key business segments based on the market estimations. Competitive landscaping mapping the key common trends. Company profiling with detailed strategies, financials, and recent developments. Supply chain trends mapping the latest technological advancements.

Access full Report Description, TOC, Table of Figure, Chart, etc. @ https://reportsinsights.com/industry-forecast/Cell-Transplant-Market-13951

About US:

Reports Insights is the leading research industry that offers contextual and data-centric research services to its customers across the globe. The firm assists its clients to strategize business policies and accomplish sustainable growth in their respective market domain. The industry provides consulting services, syndicated research reports, and customized research reports.

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COVID-19 Impact: Cell Transplant Market | Strategic Industry Evolutionary Analysis Focus on Leading Key Players and Revenue Growth Analysis by...

Recommendation and review posted by Bethany Smith

The Hackett Group at EMBL Rome explores epigenetics, genome regulation and cell identity. Recently, the scientists developed a novel CRISPR molecular tool for editing the epigenome, enabling transient modifications that can switch certain genes "on" and "off" temporarily.The SARS-CoV-2 virus that has caused the COVID-19 global pandemic makes its way into a host cell via a protein known as ACE2, which is involved in a range of physiological functions in the body.

What happens when you transiently turn "off" the gene that encodes this protein? Can SARS-Cov-2 still enter the cell and cause infection? This is what Hackett and colleagues are currently exploring in animal models, to determine whether epigenetic silencing could be a treatment approach for COVID-19 in humans.Technology Networks spoke with Dr James (Jamie) Hackett, group leader at EMBL, to learn more about the CRISPR tool, how it can be used to silence ACE2 in the context of SARS-CoV-2 infection and whether there could be any adverse implications from doing so.Molly Campbell (MC): For our readers that may be unfamiliar, can you please describe what epigenetic modifications are?Jamie Hackett (JH): Epigenetic modifications are small chemical tags that are physically grafted onto DNA (or the histones that DNA wraps around) to help control how and when the DNA is used. These epigenetic modifications act as signposts that encourage a specific part of DNA, such as a gene, to be switched on or off. In other words, they help control which genes are "expressed", and which are ignored in each cell. This is important to ensure that genes that are required specifically in liver, for example, are only switched on in the liver, and not say, in the brain.MC: You are developing a CRISPR-based molecular tool to conduct epigenetic editing. Can you please tell us about this approach? How have you developed the tool and how does it work?JH: CRISPR systems normally locate a specific section of DNA in the genome and alter its genetic sequence, known as genetic "editing". Epigenetic editing uses the same principle but instead alters the epigenetic modifications at a specific region rather than the genetic sequence. This turns genes on or off in a "programmable" manner. Importantly, unlike genetic editing, epigenetic editing is largely reversible, enabling transient changes in how genes operate without changing the DNA sequence itself.MC: You plan to test the tool in mice to target airway cells that express the ACE2 protein. Can you talk to us about the rationale behind this?JH: ACE2 is a protein that sits on the outside of many cells and is normally involved in controlling blood pressure. However, the COVID-19 virus hijacks ACE2 by using it as a docking site that enables entry of the virus into a cell. If the gene ACE2 is switched OFF, this should remove the access point for COVID-19 and restrict infection. To test this possibility, we will use mouse models where we attempt to epigenetically switch off ACE2, which will help inform us whether this could be a viable strategy in humans in the future.

MC: Could there be adverse effects from targeting the ACE2 protein, as it is involved in several physiological processes in humans, for example? How will you explore and monitor this?JH: Impaired levels of ACE2 over long periods are linked with elevated blood pressure. However, over short-term periods loss of ACE2 appears to be relatively tolerable. This is one reason why a reversible "epigenetic" approach could be appealing since it would only temporality deplete ACE2 from cells, potentially to provide protection during high risk periods, before allowing it to return to its original status at the appropriate time.MC: What broader applications might this tool have, beyond SARS-CoV-2?JH: The same technology can, in principle, be applied to change the expression of genes other than ACE2, that are linked with disease. We are at the very beginning of exploring the potential of this, so it is not clear what realistic expectations are, but there is nonetheless great excitement about such precision strategies. For example, diseases where one of the two gene copies is a "mutant", such as Huntingtons disease, could be targets. Here it is hoped to be possible to epigenetically switch off only the mutant version of the gene, leaving the normal copy on. This scenario is predicted to help mitigate symptoms in a very precise and specific way. Conversely, in the neurological disorder Fragile X syndrome, the FMR1 gene has become inappropriately silenced (switched off). Epigenetic editing can be applied to selectively reactivate this gene to switch it on, with initial indications being that this helps restore neuronal functions. Jamie Hackett was speaking to Molly Campbell, Science Writer for Technology Networks.

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Silencing the SARS-CoV-2 Receptor With CRISPR and Epigenetic Modifications - Technology Networks

Recommendation and review posted by Bethany Smith

There are good investments and there are great investments. CRISPR Therapeutics (NASDAQ:CRSP) stock certainly qualifies as the latter.

This is a company that, four years after it went public, has only been profitable for one year, yet its future is so promising that its stock is trading for 540% more than it did in 2016.

What's the big deal about CRISPR Therapeutics? The biopharmaceutical company says it is using breakthrough gene-altering therapies to treat diseases such as sickle cell disease (SCD) and beta-thalassemia, both of which are inherited blood disorders that don't have a cure and require frequent blood transfusions. The company is also working on gene therapies to treat type 1 diabetes, muscular dystrophy, cystic fibrosis, multiple myeloma, and solid tumors in pancreatic cancer and lung cancer.

Image source: Getty Images.

CRISPR Therapeutics is actually named for the technology it uses: CRISPR stands for "clusters of regularly interspaced short palindromic repeats." The gene-editing technology uses Cas9 proteins to locate a sequenceof DNA within a cell and alter it.

Though CRISPR Therapeutics has yet to bring a product to market, some of its clinical trials have had amazing results. In June, the Swiss company announced that in a joint trial with Vertex Pharmaceuticals (NASDAQ:VRTX), five patients with beta-thalassemia and two patients with sickle cell disease were treated with gene therapy CTX001.

Two of the early beta-thalassemia patients are now transfusion independent 15 months afterward. The first SCD patient in the trial is transfusion independent and free of vaso-occlusive crises (VOCs), a painful condition when blood vessels are blocked by sickled red blood cells, seven months after his dose of CTX001.

Beta-thalassemia, which reduces the body's production of hemoglobin, is extremely rare. Sickle cell anemia is common among African Americans but also can affect Latinos and people of Indian, Asian, Mediterranean backgrounds. The U.S. Department of Health and Human Services says 300,000 babies worldwide are born every year with the disease and 100,000 people in the United States are currently living with the disease.

While the results are quite promising, the study is still in its infancy. .

If you had invested $100 in CRISPR Therapeutics stock when it went public in 2016, how much would that be worth today?

By the end of the day on Oct. 19, 2016, the day CRISPR Therapeutics went public, the stock was trading at $14.09, you would have bought seven shares. Assuming you bought at that price, your original $98.63 investment would be worth $632.45 as of the close of trading Friday when the stock went for $90.35 per share. That's a gain of 541%.

CRISPR Therapeutics isn't the only biotech to use CRISPR-Cas9 technology. Others include Editas Medicine (NASDAQ:EDIT) and Intellia Therapeutics (NASDAQ:NTLA), both of which went public in 2016. Neither of those has done quite as well as CRISPR Therapeutics since its IPO.

Compared to a biotech ETF such as the iShares NASDAQ Biotechnology ETF (NASDAQ:IBB), CRISPR has shined. The ETF closed at $89.16 on the day of CRISPR's IPO. As of Friday's close, IBB was at $136.41. If you had invested $100 on Oct. 19, 2016, you would have a return of only 36%.

CRSP data by YCharts

Clinical-stage biotech stocks are inherently risky plays because there are so many hurdles these companies have to clear before they can make money. The therapies have to be approved by the Food and Drug Administration (FDA) and many therapies look promising only to fail in late-stage clinical trials.

CRISPR Therapeutics looks like a solid bet, though, both from a cash standpoint and in its strength of pipeline. The company has four candidates already in trials.It is already making money, though not a lot of it. Last year, CRISPR Therapeutics generated net income of $66.8 million, thanks to $289.5 million in collaboration revenue.

The other major question for clinical-stage biotechs is whether they have enough capital to pay for research and development until the therapies pay off. Last quarter, the company burned through $54 million in cash. However, CRISPR Therapeutics says it has more than $900 million left, so it can continue that burn rate for more than four years. The possibility that CRISPR Therapeutics' technology could actually cure diseases, not just treat them, easily makes this a risk worth taking.

Original post:
If You Invested $100 in CRISPR Therapeutics' IPO, This Is How Much Money You'd Have Now - The Motley Fool

Recommendation and review posted by Bethany Smith

New Jersey, United States,- The research report on Crispr And Crispr Associated Genes market comprises of insights in terms of pivotal parameters such as production as well as the consumption patterns alongside revenue estimations for the projected timeframe. Speaking of production aspects, the study offers an in-depth analysis regarding the manufacturing processes along with the gross revenue amassed by the leading producers operating in this business arena. The unit cost deployed by these producers in various regions during the estimated timeframe is also mentioned in the report.

Significant information pertaining to the product volume and consumption value is enlisted in the document. Additionally, the report contains details regarding the consumption graphs, Individual sale prices, and import & export activities. Additional information concerning the production and consumption patterns are presented in the report.

In market segmentation by manufacturers, the report covers the following companies-

Exploring the growth rate over a period

Business owners looking to scale up their business can refer this report that contains data regarding the rise in sales within a given consumer base for the forecast period, 2020 to 2027. Product owners can use this information along with the driving factors such as demographics and revenue generated from other products discussed in the report to get a better analysis of their products and services. Besides, the research analysts have compared the market growth rate with product sales to enable business owners to determine the success or failure of a specific product or service.

By Type

By Application

Regions Covered in the Global Crispr And Crispr Associated Genes Market:

The Middle East and Africa (GCC Countries and Egypt)

North America (the United States, Mexico, and Canada)

South America (Brazil etc.)

Europe (Turkey, Germany, Russia UK, Italy, France, etc.)

Asia-Pacific (Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)

Highlights of the Report:

Accurate market size and CAGR forecasts for the period 2020-2026

Identification and in-depth assessment of growth opportunities in key segments and regions

Detailed company profiling of top players of the global Crispr And Crispr Associated Genes market

Exhaustive research on innovation and other trends of the global Crispr And Crispr Associated Genes market

Reliable industry value chain and supply chain analysis

Comprehensive analysis of important growth drivers, restraints, challenges, and growth prospects

The scope of the Report:

The report offers a complete company profiling of leading players competing in the global Crispr And Crispr Associated Genes marketwith a high focus on the share, gross margin, net profit, sales, product portfolio, new applications, recent developments, and several other factors. It also throws light on the vendor landscape to help players become aware of future competitive changes in the global Crispr And Crispr Associated Genes market.

Reasons to Buy the Report:

About Us:

Market Research Intellect provides syndicated and customized research reports to clients from various industries and organizations with the aim of delivering functional expertise. We provide reports for all industries including Energy, Technology, Manufacturing and Construction, Chemicals and Materials, Food and Beverage, and more. These reports deliver an in-depth study of the market with industry analysis, the market value for regions and countries, and trends that are pertinent to the industry.

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Crispr And Crispr Associated Genes Market Growth Forecast Analysis Manufacturers, Regions, Type and Application to 2027 - Owned

Recommendation and review posted by Bethany Smith

CRISPR Therapeutics AG (CRSP) is expected to deliver a year-over-year increase in earnings on higher revenues when it reports results for the quarter ended June 2020. This widely-known consensus outlook gives a good sense of the company's earnings picture, but how the actual results compare to these estimates is a powerful factor that could impact its near-term stock price.

The earnings report might help the stock move higher if these key numbers are better than expectations. On the other hand, if they miss, the stock may move lower.

While the sustainability of the immediate price change and future earnings expectations will mostly depend on management's discussion of business conditions on the earnings call, it's worth handicapping the probability of a positive EPS surprise.

Zacks Consensus Estimate

This company is expected to post quarterly loss of $0.92 per share in its upcoming report, which represents a year-over-year change of +8.9%.

Revenues are expected to be $23.08 million, up 7112.5% from the year-ago quarter.

Estimate Revisions Trend

The consensus EPS estimate for the quarter has been revised 1.24% higher over the last 30 days to the current level. This is essentially a reflection of how the covering analysts have collectively reassessed their initial estimates over this period.

Investors should keep in mind that the direction of estimate revisions by each of the covering analysts may not always get reflected in the aggregate change.

Price, Consensus and EPS Surprise

Earnings Whisper

Estimate revisions ahead of a company's earnings release offer clues to the business conditions for the period whose results are coming out. Our proprietary surprise prediction model -- the Zacks Earnings ESP (Expected Surprise Prediction) -- has this insight at its core.

The Zacks Earnings ESP compares the Most Accurate Estimate to the Zacks Consensus Estimate for the quarter; the Most Accurate Estimate is a more recent version of the Zacks Consensus EPS estimate. The idea here is that analysts revising their estimates right before an earnings release have the latest information, which could potentially be more accurate than what they and others contributing to the consensus had predicted earlier.

Thus, a positive or negative Earnings ESP reading theoretically indicates the likely deviation of the actual earnings from the consensus estimate. However, the model's predictive power is significant for positive ESP readings only.

A positive Earnings ESP is a strong predictor of an earnings beat, particularly when combined with a Zacks Rank #1 (Strong Buy), 2 (Buy) or 3 (Hold). Our research shows that stocks with this combination produce a positive surprise nearly 70% of the time, and a solid Zacks Rank actually increases the predictive power of Earnings ESP.

Please note that a negative Earnings ESP reading is not indicative of an earnings miss. Our research shows that it is difficult to predict an earnings beat with any degree of confidence for stocks with negative Earnings ESP readings and/or Zacks Rank of 4 (Sell) or 5 (Strong Sell).

How Have the Numbers Shaped Up for CRISPR Therapeutics AG?

For CRISPR Therapeutics AG, the Most Accurate Estimate is higher than the Zacks Consensus Estimate, suggesting that analysts have recently become bullish on the company's earnings prospects. This has resulted in an Earnings ESP of +13.78%.

Story continues

On the other hand, the stock currently carries a Zacks Rank of #4.

So, this combination makes it difficult to conclusively predict that CRISPR Therapeutics AG will beat the consensus EPS estimate.

Does Earnings Surprise History Hold Any Clue?

Analysts often consider to what extent a company has been able to match consensus estimates in the past while calculating their estimates for its future earnings. So, it's worth taking a look at the surprise history for gauging its influence on the upcoming number.

For the last reported quarter, it was expected that CRISPR Therapeutics AG would post a loss of $1.09 per share when it actually produced a loss of $1.15, delivering a surprise of -5.50%.

Over the last four quarters, the company has beaten consensus EPS estimates two times.

Bottom Line

An earnings beat or miss may not be the sole basis for a stock moving higher or lower. Many stocks end up losing ground despite an earnings beat due to other factors that disappoint investors. Similarly, unforeseen catalysts help a number of stocks gain despite an earnings miss.

That said, betting on stocks that are expected to beat earnings expectations does increase the odds of success. This is why it's worth checking a company's Earnings ESP and Zacks Rank ahead of its quarterly release. Make sure to utilize our Earnings ESP Filter to uncover the best stocks to buy or sell before they've reported.

CRISPR Therapeutics AG doesn't appear a compelling earnings-beat candidate. However, investors should pay attention to other factors too for betting on this stock or staying away from it ahead of its earnings release.

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free reportCRISPR Therapeutics AG (CRSP) : Free Stock Analysis ReportTo read this article on Zacks.com click here.Zacks Investment Research

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Will CRISPR Therapeutics AG (CRSP) Report Negative Q2 Earnings? What You Should Know - Yahoo Finance

Recommendation and review posted by Bethany Smith

The 105. Image courtesy of Payette

One year after forming a firm to acquire, develop and operate life sciences assets, Breakthrough Properties has signed a 263,500-square-foot lease agreement for its first ground-up development, The 105 in Bostons Seaport District. Located at 105 W. First St., the building will be fully leased by gene editing company CRISPR Therapeutics once it is completed in 2022.

READ ALSO: Life Sciences Real Estate Thrives Amid Pandemic

Breakthrough Properties was launched in 2019 by real estate development firm Tishman Speyer and Bellco Capital, a biotech investment company. The firm is targeting life science developments in leading tech markets around the globe for companies involved in the biotech, agriculture and nutrition sectors. Breakthrough acquired the Seaport property in July 2019 and broke ground on The 105 in March 2020. The firm has been working closely with Tishman Speyers Boston-based team throughout the process, including site selection, and will continue the close working relationship through the projects completion.

CRISPR, which currently has its R&D operations in Cambridge, Mass., plans to consolidate various office and laboratory operations in the Greater Boston area into the new Seaport building. The company was founded in 2013 and has grown into a leading gene-editing company focused on developing transformative gene-based medicines for a broad range of diseases. The parent company, CRISPR Therapeutics AG, is headquartered in Zurich and the wholly owned U.S. subsidiary CRISPR Therapeutics also has business offices in San Francisco and London. Officials said this week that the single Boston location should support its anticipated growth for five to seven years from occupancy in 2022. CRISPR also announced Monday in its second-quarter 2020 earnings report that it is building a new cell therapy and manufacturing facility in Framingham, Mass.

Breakthrough Properties CEO Dan Belldegrun said Breakthroughs mission is to deliver cutting-edge facilities and environments that support companies like CRISPR. He noted in prepared remarks that the company was honored to play a small but supportive role in CRISPRs future as it develops therapies that change the way we fight disease.

Located on the A Street Corridor, The 105 is located near the Red Lines Broadway Station and a 10-minute ride from Kendall Square in Cambridge. Designed by the Payette architecture firm to be a best-in-class laboratory asset, Breakthrough will be seeking LEED Gold and Fitwell certifications for the building. Amenities will include a fitness center, indoor bicycle room, outdoor terraces accessible from the second and third floors, meeting space, and a locker room with showers.

Cushman & Wakefield represented CRISPR and Breakthrough was represented by Newmark Knight Frank in the lease transaction.

Last month, Hines also entered the life sciences market, unveiling a new partnership with 2ML Real Estate to develop a biotech and mixed-use hub in Houston. Levit Green will span more than 52 acres and be adjacent to the Texas Medical Center, the worlds largest cluster of medical facilities and businesses. 2ML is supplying the land for the project. Preliminary plans call for a mix of research, office, residential, retail and dining uses, along with outdoor amenities and green space.

Hines and Tishman Speyer are among a growing group of commercial real estate developers expanding recently into the burgeoning life sciences sector. Boston Properties teamed up with Alexandria Real Estate Equities in January to develop a 1.7 million-square-foot life science campus in South San Francisco. In October, LaSalle Investment Management took a stake in the San Diego headquarters of Illumina, a DNA sequencing and array-based technologies firm. Last summer, Thor Equities launched its Thor Sciences division to invest in biotech properties.

See the article here:
Breakthrough Properties Completes Full-Building Lease in Boston - Commercial Property Executive

Recommendation and review posted by Bethany Smith

Scientists at the University of WisconsinMadison have published a proof-of-concept method to correct an inherited form of macular degeneration that causes blindness, and that is currently untreatable.

Andrew Hellpap608-225-5024ahellpap@uwhealth.org

The researchers were able to correct the disease in stem cells from patients with BEST1 mutations by overwhelming broken copies of the gene with many functional copies of BEST1. This approach worked for most, but not all, of the BEST1 mutations that they tested. As an alternative approach for mutations that did not respond to this gene augmentation method, the team used CRISPR-Cas9 gene editing to target and correct the mutations.

A paper chronicling the research, co-led by David Gamm, MD, PhD, professor of ophthalmology and visual sciences in the School of Medicine and Public Health, was published online July 23 in the American Journal of Human Genetics. The study was also led by Kris Saha, PhD, associate professor of biomedical engineering and Wisconsin Institute for Discovery, and Bikash Pattnaik, PhD, assistant professor of pediatrics.

This BEST1 gene encodes a protein that regulates the movement of chloride across a layer of the retina called the retinal pigment epithelium (RPE). Best disease is dominant, meaning that people who inherit only one faulty copy of the BEST1 gene from either their mother or their father will develop the disorder. Mutations in BEST1 cause the retinal layer to break down, resulting in blurred central vision that progresses to irreversible vision loss.

People with Best disease have a wide range of mutations that can affect different parts of the protein, all of which were thought to require complex, individualized gene therapies to fix them, Gamm said. We found that many of these mutations were actually very sensitive to a broader gene therapy method that is already established for other retinal diseases.

Fixing a dominant genetic disease via gene therapy typically requires precise removal or repair of the nonfunctional gene without causing harm to the functional gene a difficult task that is frequently unsuccessful. In contrast, recessive genetic diseases that arise when a person inherits two nonfunctional genes one from each parent can be corrected by a technique called gene augmentation. This well-established process introduces a functional copy of the gene to fill the void.

To use another analogy, dominant mutations produce workers that actively look to sabotage the efforts of their capable coworkers, whereas recessive mutations produce proteins that never show up for work at all, Gamm said. As it turns out, the latter situation is usually simpler to treat than the former.

A team of researchers at the McPherson Eye Research Institute, which Gamm directs, hypothesized that it may be possible to adequately dilute the influence of the nonfunctional BEST1 protein by counter-balancing it with many functional copies of BEST1 protein through gene augmentation.

In the lab, the approach worked in RPE cells derived from induced pluripotent stem cells of patients with most, but not all, of the BEST1 gene mutations they tested. Where gene augmentation did not succeed, the team was able to correct the dysfunction using CRISPR-Cas9 gene editing.

The research was carried out in large part by Divya Sinha, PhD, an assistant scientist in Gamms lab, Ben Steyer, a former MD-PhD student in Sahas lab, and Pawan Shahi, PhD, postdoctoral research associate in Pattnaiks lab. The research team also included Sushmita Roy, PhD, associate professor of biostatics and medical informatics at the UW School of Medicine and Public Health and Wisconsin Institute for Discovery.

The scientists demonstrated that their two-pronged gene therapy strategy may hold potential to treat all Best disease mutations in a highly effective manner.

We were able to reverse the disease in all the cell lines using one method or the other, Gamm said. We were also able to determine which mutations were likely to respond to the first-line gene augmentation strategy, and which would be better served with the second-line gene editing approach.

An additional benefit came into focus as this research progressed, according to Gamm.

Our findings also could be applicable to some dominant genetic mutations that affect tissues elsewhere in the body, he said. Its very exciting.

More here:
UW researchers devise approach to treat rare, incurable form of blindness - University of Wisconsin-Madison

Recommendation and review posted by Bethany Smith

DUBLIN--(BUSINESS WIRE)--The "Cell and Gene Therapy Tools, and Reagents: Global Markets" report has been added to ResearchAndMarkets.com's offering.

Gene and cell therapy are emerging as important tools to treat human health. Techniques such as CAR-T therapy have emerged as key ways of treating many different types of cancers. The promise of gene therapy using technologies such as CRISPR is starting to be realized in clinical trials, and markets are scaling up to treat other diseases as well, particularly rare gene-based diseases. As these therapies are coming to the fore, a new market for tools to develop these therapies using standard methodologies is emerging. This report will cover what those tools are, how they impact the larger life science tools market, and how they will evolve over the next five years.

The scope of this study encompasses an investigation of the market's cell and gene therapy tools such as GMP proteins, media, cell separation and activation reagents, viral and non-viral, cytokine release syndrome monitoring products, GMP antibodies, leukapheresis instrumentation, immunoassays (multiplex and singleplex) and bioreactors. This research analyzes each tool type, determines its current market status, examines its impact on future markets, and presents forecasts of growth over the next five years. Technological issues, including the latest trends, are discussed. The report analyzes the industry on a worldwide basis, from both application and demand perspectives, in the major regions of the world.

The Report Includes:

Key Topics Covered:

Chapter 1 Introduction

Chapter 2 Summary and Highlights

Chapter 3 Market and Technology Background

Chapter 4 Market Breakdown by Region

Chapter 5 Market Breakdown by End User

Chapter 6 Government Regulations

Chapter 7 Patent Review/New Developments

Chapter 8 Analysis of Market Opportunities

Chapter 9 Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/nk3d0z

About ResearchAndMarkets.com

ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

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Global Cell and Gene Therapy Tools and Reagents Market 2020: Analysis of Market Opportunities - ResearchAndMarkets.com - Business Wire

Recommendation and review posted by Bethany Smith