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MADRID Because of the hormone changes that occur throughout their lives, women experience sleep problems that differ significantly from those experienced by men. Indeed, 75% to 84% of pregnant women don't sleep well during the third trimester, and up to 80% of women in menopause have symptoms that prevent them from getting a good night's rest. For those seeking to a precision medicine approach, the challenge is to identify the relationship between the different sex-related phenotypes and the sleep conditions.

Irene Cano, MD, PhD, is the coordinator of the sleep department at the Spanish Society of Pulmonology and Thoracic Surgery (SEPAR). She spoke with Medscape Spanish Edition about the significant impact of hormones on sleep disorders in women.

"Reproductive hormones like estrogen and progesterone play a meaningful role in brain functions not only those linked to the regulation of reproduction but also other physiological processes related to the regulation of circadian rhythms, cognitive performance, mood, and sleep. In addition, other hormones for example, prolactin, growth hormone, cortisol, and melatonin have sex-dependent effects on sleep," Cano said.

Girls start puberty at a younger age than boys. As girls enter adolescence, they go to bed later and waking up earlier. So, girls are getting less than the 10 hours of sleep that they should be getting at this stage of life. The result is sleep debt, which gives rise to various problems: poor academic performance, attention-deficit/hyperactivity disorder, obesity, and metabolic problems, to name a few. As Ariadna Farr, RN, a sleep unit nurse, noted at SEPAR's Joint Winter Meeting, "Schools would have to start morning classes later to get adolescents to perform well academically. As the situation is now, half of the kids are falling asleep at their desks."

Cano explained the issue as follows: "In adolescence, along with changes in young women's hormone levels, we begin to see differences between the sexes. The changes in levels of estrogens and progesterone are what's responsible for the changes that, to some extent, cause those disturbances in the quality of our sleep and in the stages of our sleep."

Thus, sleep can be affected by the changes in hormone level that occur during a menstrual cycle. Estrogens, which increase during the follicular phase, are associated with rapid eye movement (REM) sleep, while progesterone, which increases during the luteal phase, increases non-REM sleep. "In the 3 to 6 days prior to menstruation, it's quite common for a woman to report difficulties falling asleep and staying asleep, in connection with a decline in the percentage of time she spends in REM sleep, in the context of premenstrual syndrome. In addition," Cano pointed out, "menstrual bleeding, that loss of blood, is associated with a drop in iron levels, making it more likely that the woman will experience restless legs syndrome."

Medscape Spanish Edition also spoke with Milagros Merino, MD, PhD, president of the Spanish Sleep Society. "The consequences that lack of sleep have on the cardiovascular system we're essentially talking about certain arrhythmias, high blood pressure, thrombosis in some cases, stroke, and heart attack. Lack of sleep also gives rise to endocrine and metabolic issues, like overweight and being at a greater risk of developing diabetes. And as for mental health, we see, among other things, attention and memory problems, emotional lability, and irascibility. Numerous studies have confirmed all of this."

Sleep apnea also deserves mention, Merino added. "Although this disorder is more common in men, we're seeing it more and more now in women, along with the cardiovascular issues that it brings about."

Another cardiovascular risk factor is insomnia, said Merino. "This sleep disorder is more prevalent in women. As hormones constantly change, the ways women sleep constantly change, from one stage of life to the next. They sleep one way in childhood, another way in adolescence, and yet another way in menopause."

During pregnancy, hormone changes are much more pronounced. During the first trimester, progesterone levels increase, making the woman drowsy. On top of that, her sleep is interrupted by more frequent visits to the bathroom as well as greater general discomfort.

In the second trimester, sleep interruptions persist but are not as bad as they were during the first 3 months. In the third trimester, 75% to 84% of pregnant women find it difficult to sleep because of aches and pains, the need to urinate during the night, cramps, and heartburn.

"Major physical changes are happening. When the bladder gets compressed, the woman has to get up and go to the bathroom. There's an interruption in her sleep," Farr explained. In addition, as the pregnancy progresses, the woman gains weight and her body mass index (BMI) increases, which can bring on obstructive sleep apnea, high blood pressure, preeclampsia, and diabetes, if not closely monitored.

Other factors include concomitant treatments, such as contraceptives, and the stages of life, such as pregnancy and lactation. "When a woman of childbearing age has restless legs syndrome, more often than not, this means that she has an iron deficiency that needs to be treated with oral iron supplements," said Merino. "However, there are few medications that can be given to a pregnant woman and RLS is relatively common during pregnancy. So, we have to turn to oral or intravenous iron supplements. Yet another matter is narcolepsy. In these cases, all medications have to be stopped during pregnancy and lactation, as they can be harmful to the baby."

While 1 in 5 menopausal women are asymptomatic, the others experience mild to severe symptoms of apnea that frequently interrupt their sleep. In this stage of life, which begins around age 50 years, the hormones that had provided protection against sleep disruptions start to decrease. As a result, there is a rise in sleep problems, especially insomnia, breathing-related sleep disorders (eg, apnea), and restless legs syndrome.

The prevalence of breathing-related sleep disorders during menopause is attributable to weight gain, the drop in levels of estrogens, and the redistribution of adipose tissue in the body. Other factors also increase a woman's risk of experiencing apnea. They range from stress, depression, and other psychological and psychiatric conditions to health status, medication use, and simply the fact of getting older. "Sleep apnea is more common in men than in premenopausal women. The numbers even out, though, when we compare men against menopausal women," Cano noted.

In women, symptoms of sleep apnea are frequently attributed to menopause. There is some overlap: insomnia, headache, irritability, low mood, decreased libido, fatigue during the day, and feeling sleepy. Only much later is the woman's condition correctly diagnosed as sleep apnea. So, even though presenting with the same complaints, a man will be diagnosed with sleep apnea sooner than a woman will in some cases, around 10 years sooner.

"On the other hand, we'd always thought that, in menopause, insomnia was characterized by awakenings occurring throughout the second half of the night. But perhaps what happens more often is that women are regularly waking up repeatedly over the course of the entire night, as opposed to experiencing a wakefulness that starts early and lasts throughout the night or having a problem falling asleep to begin with," said Merino. "The good news is that hormone replacement therapy can get things back to the way they were. And getting better sleep will help to overcome insomnia."

Insomnia is the most common sleep disorder. It affects 10% to 20% of people, mostly women. "The fact that sleep problems are more prevalent in women can be explained by the fact that among women, there is a higher incidence of conditions that disrupt sleep, such as depression," said Cano.

"Insomnia is much more common in adult women than adult men. And at menopause, women find that the insomnia only gets worse," Merino added. "But around that same age, 50 years old, what we start to see more frequently in men is REM sleep behavior disorder, a type of parasomnia that's a risk marker of degenerative nerve diseases."

Cano emphasized one finding that, though basic, is not well known. "After adjusting for socioeconomic characteristics, the difference between the sexes in reporting sleep problems is cut in half. This suggests that an important factor that explains why there are differences in sleep problems between the sexes is that women's socioeconomic status is generally lower than men's.

"As for sleep apnea in particular," Cano continued, "the kinds of symptoms that women have can be different from the classic ones seen in men snoring, pauses in breathing, and daytime sleepiness; women are being underdiagnosed, and when they are diagnosed, that's happening at a later age and at a higher BMI."

So, it's alarming that, as reported by SEPAR, 90% of women with obstructive sleep apnea are not being diagnosed.

"The majority of research studies on sleep apnea have focused on men given the prevalence of cases and the results have been extrapolated to women. This is why there's still a lot of work to be done in terms of better defining the characteristics specific to each sleep disorder and how they relate to each sex," said Cano. "Being able to identify the relationship between the different sex-related phenotypes and each condition will allow us to take a precision medicine approach tailored to a patient's particular characteristics."

As Merino put it, "The approach to sleep disorders is always personalized. The patient's sex, in and of itself, doesn't have that great of an impact on this approach. What does have a great impact are women's life stages. There are some subtle differences here and there, such as types of continuous positive airway pressure machines. The ones that are designed for women have masks that are better suited to their facial features, which differ from men's."

A precision medicine approach can be taken to treat any sleep disorder. For insomnia, the approach allows healthcare professionals to employ an appropriate cognitive-behavioral therapy plan or to determine which drugs would be more effective all on the basis of symptoms and the characteristics of the particular case. Regarding sleep apnea, Cano explained, "Taking into account the different anatomical characteristics or the higher prevalence of positional apnea will also allow us to offer different therapeutic alternatives to continuous positive airway pressure, such as mandibular advancement devices or positional therapy devices."

Women should be encouraged to develop good sleep habits. These include taking circadian rhythms into account and aligning lifestyles accordingly. It also means going to bed earlier than the men in the household. For menopausal women, recommended sleep habits range from keeping their bedroom at an ideal temperature, following a diet rich in vegetables to avoid becoming overweight, and exercising daily. While this advice may be more applicable to teenagers, adults can benefit from it as well: electronic devices should be turned off well before bedtime. Whether from a phone screen, a tablet screen, or a TV screen, the light emitted can keep one awake, which can be harmful to one's health.

Cano and Merino have disclosed no relevant financial relationships.

This article was translated from the Medscape Spanish edition.

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Hormone Changes: The Star of Every Stage in Women's Sleep - Medscape

Youre one of a kind. Its not just your eyes, smile, and personality. Your health, risk for disease, and the ways you respond to medicines are also unique. Medicines that work well for some people may not help you at all. They might even cause problems. Wouldnt it be nice if treatments and preventive care could be designed just for you?

The careful matching of your biology to your medical care is known as personalized medicine. Its already being used by healthcare providers nationwide.

The story of personalized medicine begins with the unique set of genes you inherited from your parents. Genes are stretches of DNA that serve as a sort of instruction manual telling your body how to make the proteins and perform the other tasks that your body needs. These genetic instructions are written in varying patterns of only 4 different chemical letters, or bases.

The same genes often differ slightly between people. Bases may be switched, missing, or added here and there. Most of these variations have no effect on your health. But some can create unusual proteins that might boost your risk for certain diseases. Some variants can affect how well a medicine works in your body. Or they might cause the medicine to have different side effects in you than in someone else.

The study of how genes affect the way medicines work in your body is called pharmacogenomics.

If doctors know your genes, they can predict drug response and incorporate this information into the medical decisions they make, says Dr. Rochelle Long, a pharmacogenomics expert at NIH.

Its becoming more common for doctors to test for gene variants before prescribing certain drugs. For example, children with leukemia might get theTPMTgene test to help doctors choose the right dosage of medicine to prevent toxic side effects. Some HIV-infected patients are severely allergic to treatment drugs, and genetic tests can help identify who can safely take the medicines.

By screening to know who shouldnt get certain drugs, we can prevent life-threatening side effects, Long says.

Pharmacogenomics is also being used for cancer treatment. Some breast cancer drugs only work in women with particular genetic variations. If testing shows patients with advanced melanoma (skin cancer) have certain variants, two new approved drugs can treat them.

Even one of the oldest and most common drugs, aspirin, can have varying effects based on your genes. Millions of people take a daily aspirin to lower their risk for heart attack and stroke. Aspirin helps by preventing blood clots that could clog arteries. But aspirin doesnt reduce heart disease risk in everyone.

NIH-funded researchers recently identified a set of genes with unique activity patterns that can help assess whether someone will benefit from taking aspirin for heart health. Scientists are now working to develop a standardized test for use in daily practice. If doctors can tell that aspirin wont work in certain patients, they can try different treatments.

One NIH-funded research team studied a different clot-fighting drug known as clopidogrel (Plavix). Its often prescribed for people at risk of heart attack or stroke. Led by Dr. Alan Shuldiner at the University of Maryland School of Medicine, the team examined people in an Amish community. Isolated communities like this have less genetic diversity than the general population, which can make it easier to study the effects of genes. But as in the general population, some Amish people have risk factors, such as eating a high-fat diet, that raise their risk for heart disease.

Many of the Amish people studied had a particular gene variant that made them less responsive to clopidogrel, the scientists found. Further research revealed that up to one-third of the general population may have similar variations in this gene, meaning they too probably need a different medicine to reduce heart disease risks.

The findings prompted the U.S. Food and Drug Administration (FDA) to change the label for this common drug to alert doctors that it may not be appropriate for patients who have certain gene variations. Two alternative drugs have since been developed. If people have these gene variants, they know they have options, says Shuldiner. This is a great example of how study results made it onto a drug label and are beginning to be implemented into patient care.

Getting a genetic test usually isnt difficult. Doctors generally take a sample of body fluid or tissue, such as blood, saliva, or skin, and send it to a lab. Most genetic tests used today analyze just one or a few genes, often to help diagnose disease. Newborns, for example, are routinely screened for several genetic disorders by taking a few drops of blood from their heels. When life-threatening conditions are caught early, infants can be treated right away to prevent problems.

The decision about whether to get a particular genetic test can be complicated. Genetic tests are now available for about 2,500 diseases, and that number keeps growing. Your doctor might advise you to get tested for specific genetic diseases if they tend to run in your family or if you have certain symptoms.

While there are many genetic tests, they vary as to how well they predict risk, says Dr. Lawrence Brody, a genetic testing expert at NIH.

For some diseases, such as sickle cell anemia or cystic fibrosis, inheriting 2 copies of abnormal genes means a person will get that disease. But for other diseases and conditions, the picture is more complex. For Type 2 diabetes, testing positive for some specific gene variants may help predict risk, but no better than other factorssuch as obesity, high blood pressure, and having a close relative with the disease.

The latest approach to personalized medicine is to get your wholegenomesequenced. Thats still expensive, but the cost has dropped dramatically over the past decade and will likely continue to fall. Since your genome essentially stays the same over time, this information might one day become part of your medical record, so doctors could consult it as needed.

You can start to get a sense of your genetic risks by putting together your familys health history. A free online tool calledMy Family Health Portraitfrom the U.S. Surgeon General can help you and your doctor spot early warning signs of conditions that run in your family.

But personalized medicine isnt just about genes. You can learn a lot about your health risks by taking a close look at your current health and habits. Smoking, a poor diet, and lack of exercise can raise your risks for life-threatening health problems, such as heart disease and cancer. Talk to your healthcare provider about the steps you can take to understand and reduce your unique health risks.

Matching Treatments to Your Genes was originally published by the National Institutes of Health.

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Biologic medications that neutralize IgE antibodies have long been considered a promising food allergy treatment. Now, one such medication omalizumab (or Xolair) looks likely to become the first anti-IgE biologic drug contender to treat multiple food allergies.

Researchers from the Consortium of Food Allergy Research are studying omalizumab in two ways for child and adult patients. The first is as a standalone treatment to protect against reacting to accidental exposures in those with allergies to peanut plus two or more other foods: milk, egg, wheat, cashew, hazelnut or walnut.

The second method is as an add-on treatment for oral immunotherapy (OIT) to reduce adverse reactions and improve safety.

If this drug works, its very likely to work for exposures to at least small amounts of any foods, where people could eat out without having to worry, says Dr. Robert Wood. Hes the trials principal investigator and director of pediatric allergy and immunology at Johns Hopkins Medicine in Baltimore.

Its quite possible omalizumab could help people safely eat more than small amounts of their allergens perhaps even introducing these foods into their diets.

The first 60 participants have completed stage 1 of the double-blind, placebo-controlled study. At this stage, neither researchers nor patients know who is receiving the injectable medication and who is getting the sham shot.

The initial patients then moved to an open-label extension of the trial. Everyone, including those who had received the placebo injections, got a series of omalizumab injections. After six months of shots every two to four weeks, these patients underwent food challenges. Depending on how much they could consume of the allergens without reacting, patients could either immediately start introducing those foods into their diet, or start multi-allergen OIT.

What we can say, even though the results are not fully analyzed, is that most of the participants were able to successfully introduce the foods they were allergic to into their diet, Wood says.

Those who were able to eat a lot of the food at their food challenge without reacting are given a minimum and a maximum amount of the allergenic foods to eat daily. This is to maintain their desensitization. Those who reacted to smaller amounts of the food are offered OIT, since it starts at a lower dose of the food protein.

Stage 1 of the trial is still enrolling, with a goal of getting to 225 patients. If things look good, this treatment could move out to the public in 2024 or possibly 2025, Wood says.

Omalizumab is a monoclonal, or lab-made, antibody. Its currently approved in the U.S. to treat moderate to severe asthma, nasal polyps and chronic hives. The drug, developed by Genentech and Novartis Pharmaceuticals, works by binding to IgE antibodies circulating in the blood. This prevents the IgE from binding to receptors on the surface of mast cells and basophils. Those immune system cells are involved in setting off allergic reactions and anaphylaxis.

In 2018, the U.S. Food and Drug Administration granted omalizumab breakthrough therapy designation for its potential as a food allergy treatment. Wood and his colleagues launched the OUtMATCH study the following year.

Omalizumab, and anti-IgE medications like it, have long been eyed as a food allergy treatment. The first study to look at treating peanut allergy with monoclonal antibodies was published in 2003.

Back then, researchers gave 84 peanut-allergic patients either an injection of an anti-IgE drug similar to omalizumab, called TNX-901, or a placebo. They found that after a single dose of TNX-901, participants who had initially reacted to the equivalent of half to one peanut could consume almost nine peanuts before starting to react. TNX-901, however, was never developed for commercial use by the drug makers.

Even so, anti-IgE biologic drug research continued. As well as studying biologics as standalone treatments (called monotherapy) for food allergy, researchers also wanted to know if combining a biologic with OIT could cut down adverse side effects.

During OIT, allergic individuals eat gradually increasing amounts of their allergen over several months. The goal is to increase protection in case an allergen is accidentally consumed. While OIT has a strong success rate, many patients experience adverse effects. Common symptoms are mouth and throat itching, stomach pain or vomiting. A minority of patients experience anaphylaxis.

We and many others had been pushing to move this forward, says Wood. One idea was using Xolair as a drug to treat food allergy. The second idea that has been studied is to use Xolair along with OIT as a way to make OIT safer.

The two approaches are going to look quite different, with one being a longer-term use of Xolair to increase the threshold of reactivity. While the other is a relatively short-term use of Xolair to get the dosing escalation done.

Indeed, some allergists already prescribe omalizumab off-label in conjunction with OIT. Others will wait for FDA approval of such a protocol. Wood says FDA approval is also needed for insurance coverage.

The OUtMATCH trial was about 80 percent enrolled, as of October 2022. At the start of the study, all participants do oral challenges to confirm their food allergies.

From there, one group receives injections of omalizumab every two to four weeks, while the other group receives placebo shots. After four months, a second round of food challenges determines how much of each allergen patients can tolerate.

We do think people can become protected to large amounts of food pretty quickly 20-plus peanuts. That is what we are hoping to see, says Dr. Edwin Kim, a study investigator and an associate professor of allergy and immunology at the University of North Carolina at Chapel Hill.

Additional stages are underway. Stage 2 looks at using Xolair with OIT. All Stage 2 participants receive omalizumab injections for the first eight weeks of treatment, then start either multi-allergen OIT or placebo OIT.

Then as oral immunotherapy begins, they receive the shots for the first eight weeks of treatment. After that,half the participantscontinue with multi-allergen OIT while receiving placebo shots.Theother group continues with placebo OIT, but receives realXolairinjections.All participants then go onto maintenance therapy in their respective groups to complete one year of treatment.

Stage 3 will be a real-world follow up of patients to see how they fare over the long-term having introduced the foods into their diet.

We are all very excited about this. Scientifically, its supposed to work. Hopefully we are correct, and it does work, Kim says.

If Xolair is approved as a standalone treatment, the label would say the medication is for the prevention of reactions to small, accidental exposures to food, Wood says. Patients would still be advised to avoid their allergen. (Although they could potentially do a food challenge to see if they could tolerate more, he adds).

The dosing amounts and intervals in the trial are the same as the dosing of omalizumab when its used to treat other conditions. In patients 12 and older, omalizumab can be self-injected at home after several in-office injections. Researchers say they expect food allergy patients would be able to do the same.

If injections are stopped, Xolairs effects will gradually wear off over several months. While omalizumab is usually considered very safe, it can have a range of side effects. These include rare but severe allergic reactions. The medication carries a black box warning about potentially life-threatening anaphylaxis.

Even with those caveats, Kim predicts the medication as a monotherapy would be a welcome option for many food allergy families, including those who choose not to pursue OIT.

OIT can be challenging for some families to manage. Parents work schedules and intense after-school sports schedules may make daily dosing and rest periods difficult. Many allergists arent set up to offer OIT, so accessibility is an issue in some areas.

An Olympic-level swimmer who is training constantly maybe cant do an OIT, but they can do a once a month shot, Kim says. Omalizumab might provide added protection for teenager about to leave home for college. Or, an adult might choose the shots to feel safer when dining out and traveling.

To date, there is only one FDA-approved food allergy therapy Palforzia, the oral immunotherapy for peanut allergy. While some allergists offer OIT for other foods, patients with many food allergies find it time-consuming to become desensitized to all of them.

One of the most exciting things about a drug to treat food allergy as opposed to OIT is that a drug like Xolair would not be food-specific, Wood says. If someone had food allergies other than peanuts, or peanut plus eight other food allergies, instead of having to wait for an OIT product to be available, this would be a way to cover everyones food allergies equally.

Kim says not to overlook that omalizumab as an adjunct to OIT is also exciting. If the medication can prevent adverse reactions during OIT, it could improve dropout rates during the dose escalation period. Or, it could help those worried about reactions to feel safer in trying it.

Kim envisions that one day soon food allergy families will have choices about what treatment route is best for them. This is about creating options for people. Until we have that one-shot cure, its going to be about personalized medicine. Food allergies affect people in different ways, he says.

Related Reading: Allergists See Palforzia as Start of Era of Food Allergy TherapiesOIT Study See Maturing of Immune System Over TimeNew Peanut Allergy Test Reveals if Trace Amounts Risky or Not

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Could Xolair Be the First Biologic Treatment for Food Allergies? - Allergic Living

DUBLIN, Oct. 13, 2022 /PRNewswire/ -- The "Monoclonal Antibodies Market Size, Share & Trends Analysis Report by Source Type (Chimeric, Murine, Humanized, Human), by Production Type (In Vivo, In Vitro), by Application, by End-use, by Region, and Segment Forecasts, 2022-2030" report has been added to ResearchAndMarkets.com's offering.

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The global monoclonal antibodies market is projected to reach USD 494.53 billion by 2030, at a compound annual growth rate (CAGR) of 11.30%

Increasing research and development activities aimed at the development of novel therapeutic monoclonal antibodies (mAbs) and supportive government initiatives for biologics production are anticipated to drive market growth in the forecast period.

The rising demand for personalized medicine is likely to positively affect the development of therapeutic mAb targeted therapies that are tailored to individual requirements. Moreover, the therapeutic use of mAbs offers several advantages such as fewer adverse effects, specificity of treatment, and large-scale production capabilities, as compared to conventional treatment options, and can significantly drive market growth.

In addition, several government entities are fueling clinical research activities and expanding the scope of applications for mAb therapies. For instance, in June 2022, the National Institutes of Health launched clinical trials for the evaluation of dupilumab, for the reduction of asthma attacks and the improvement of lung functionality in children.

Similarly, in March 2021, the U.S. Department of Health and Human Services invested USD 150 million in increasing patient access to monoclonal antibody therapeutics for COVID-19. Such initiatives are expected to increase the adoption of monoclonal antibodies and strengthen growth prospects.

Furthermore, technological advancements in genetic engineering, DNA cloning, and various peptide and protein display technologies have led to the generation and optimization of recombinant mAbs. Demand for such recombinant antibodies is driven by increasing commercial prospects and quality requirements that may not be fulfilled by hybridoma technologies. Hence, with the increasing commercial viability of recombinant technologies, the market is expected to witness significant growth.

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The COVID-19 pandemic has created several market expansion opportunities by fueling the development of several mAbs directed against the SARS-CoV-2 virus.

As mAbs represent a promising alternative for mitigation of the disease due to their safety and effectiveness, several mAbs such as Eli Lilly's bebtelovimab, and GlaxoSmithKline & Vir Biotechnology's sotrovimab have attained emergency use authorizations from the U.S. FDA. Such authorizations are likely to broaden the horizons for the growth of mAbs applications and propel market growth.

Monoclonal Antibodies Market Report Highlights

By source type, the human mAbs segment held the largest share of 54.07% in 2021 due to the low immunogenicity offered and increasing cancer treatment applications of these antibodies

In vitro production type held a dominating share of 78.45% in 2021 due to the low contamination levels offered by the technique and the availability of advanced bio-manufacturing capabilities

The oncology segment dominated the market in 2021 due to the rising number of approvals for mAb cancer therapeutics and increasing scientific and patient awareness about such therapies

Hospitals accounted for the largest share of the end-use segment in 2021 due to the widespread use of mAbs for cancer treatment in hospitals and increasing healthcare expenditure favoring the adoption of such therapeutics

North America accounted for 46.2% of the market value in 2021 due to the availability of advanced healthcare infrastructure, high growth in cancer incidence, and local presence of key players such as Pfizer Inc., Amgen, Inc., and Merck & Co., among others

The Asia Pacific is projected to witness the highest growth rate in the forecast period due to the availability of a large patient pool for cancer treatment and expanding the scope of clinical research prospects for mAbs.

Key Topics Covered:

Chapter 1 Methodology and Scope

Chapter 2 Executive Summary2.1 Market Outlook2.2 Market Summary

Chapter 3 Monoclonal Antibodies Market Variables, Trends & Scope3.1 Market Trends & Outlook3.2 Market Segmentation & Scope3.3 Market Driver Analysis3.3.1 Increasing Incidences Of Chronic Diseases3.3.2 Technological Advancements3.3.3 Growing Awareness Levels3.4 Market Restraint Analysis3.4.1 High Cost Of Therapeutic Mabs3.5 SWOT Analysis, by Factor (Political & Legal, Economic, and Technological)3.6 Porter's Five Forces Analysis3.7 COVID-19 Impact Analysis3.8 Penetration & Growth Prospect Mapping

Chapter 4 Monoclonal Antibodies Market - Segment Analysis, by Source Type, 2018 - 2030 (USD Billion)4.1 Monoclonal Antibodies Market: Source Type Movement Analysis4.2 Murine4.2.1 Murine Market Estimates And Forecast, 2018 - 2030 (USD Billion)4.3 Chimeric4.4 Humanized4.5 Human

Chapter 5 Monoclonal Antibodies Market - Segment Analysis, By Production Type, 2018 - 2030 (USD Billion)5.1 Monoclonal Antibodies Market: Production Type Movement Analysis5.2 In Vivo5.2.1 In Vivo Market Estimates And Forecast, 2018 - 2030 (USD Billion)5.3 In Vitro

Chapter 6 Monoclonal Antibodies Market - Segment Analysis, By Application, 2018 - 2030 (USD Billion)6.1 Monoclonal Antibodies Market: Application Movement Analysis6.2 Oncology6.2.1 Oncology Market Estimates And Forecast, 2018 - 2030 (USD Billion)6.3 Autoimmune Diseases6.4 Infectious Diseases6.5 Neurological Diseases

Chapter 7 Monoclonal Antibodies Market - Segment Analysis, By End-Use, 2018 - 2030 (USD Billion)7.1 Monoclonal Antibodies Market: End-Use Movement Analysis7.2 Hospitals7.2.1 Hospitals Market Estimates And Forecast, 2018 - 2030 (USD Billion)7.3 Specialty Centers

Chapter 8 Monoclonal Antibodies Market - Segment Analysis, By Region, 2018 - 2030 (USD Billion)8.1 Monoclonal Antibodies Market: Regional Movement Analysis

Chapter 9 Competitive Analysis

Abbott Laboratories

Amgen Inc.

Astrazeneca plc

Bayer AG

Biogen Inc.

Bristol Myers Squibb

Daiichi Sankyo Company, Limited

Eli Lilly and Company

F. Hoffman-La Roche Ltd.

GlaxoSmithKline plc

Johnson & Johnson Services, Inc.

Merck & Co. Inc.

Merck Kgaa

Novartis AG

Novo Nordisk A/S

Pfizer Inc

Sanofi S.A.

Thermo Fisher Scientific, Inc.

Viatris Inc.

For more information about this report visit https://www.researchandmarkets.com/r/1y2bed

Media Contact:

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SAN FRANCISCO & DUBLIN--(BUSINESS WIRE)--Xcell Biosciences Inc. (Xcellbio), an instrumentation company focused on cell and gene therapy applications, and aCGT Vector, a point-of-care cell and gene therapy-as-a-service (TaaS) company, today announced a collaboration to improve manufacturing and analytic procedures used to develop personalized cell and gene therapies for cancer patients. Through this alliance, aCGT Vector will provide its point-of-care, GMP-licensed manufacturing platform to validate Xcellbios core next-generation manufacturing and analytical AVATAR AI technology for use in precision cancer treatment.

We are looking forward to working with the Xcellbio team and to utilizing the well-established AVATAR platform to further power our TaaS platform to deliver and deploy precision medicine proximal to patients, said Gary McAuslan, CEO and co-founder of aCGT Vector. We believe our joint efforts will further accelerate the creation of automated, end-to-end cell therapy manufacturing and QC release platforms to streamline processing and optimize patient outcomes.

aCGT Vectors enclosed, GMP-compliant manufacturing environment will demonstrate the use of Xcellbios AVATAR AI to support development and deployment of cell therapeutic procedures proximal to patients in the treatment of cancers. Through mimicking the tumor microenvironment (TME) ex vivo, AVATAR AI delivers unique capabilities to support the development of therapeutic products with improved potency and persistence as well as reduced cell exhaustion. Proprietary technology enables tight control and modulation of atmospheric pressures and oxygen concentrations in direct contact with the cell therapy product.

The AVATAR AI leverages the tight environmental control of the proven AVATAR product family, and pairs it with a specialized reader to enable real-time, label-free cell killing analysis of cell therapies targeting solid tumors. Focused on establishing next-gen immunotherapy testing workflows, the AVATAR AI system is currently in late-stage beta.

With the tremendous promise of cell therapies and the current challenges in treating patients with solid tumors, there is more interest in both designing manufacturing conditions to optimize potency, persistence, and quality and in characterizing cell potency as a critical attribute in cell therapy manufacturing, said Brian Feth, co-founder and CEO at Xcellbio. We are pleased to partner with aCGT Vector and to pair their manufacturing expertise with our unique approach to measuring and improving therapeutic potency. Together, our objective is to advance the development and deployment of cell therapies, such as CAR-T and tumor-infiltrating lymphocyte technologies, to treat solid tumors. We look forward to supporting aCGT and their initiatives, including the next-generation cell therapies for the cancer-focused HEALED Consortium.

For more information about Xcellbios AVATAR AI system or its Beta program, please visit http://www.xcellbio.com/avatar-ai.

About aCGT Vector

aCGT Vector have assembled a world class team of cell therapy and processing expertise within major cellular therapy centers of excellence. aCGT is establishing a state-of-the-art, closed-system cellular processing standardization technology and digitization loopback platform within a network of GMP ATMP PODS located proximal to rare disease patients. aCGT Vector will deliver cellular therapy processing and procedures through co-locating multidisciplinary expertise more efficiently, effectively, and safely within centers of excellence. The result will be to place less burden on patients and less stress on their cells. Digitization will permit enhanced control of cell processing and monitoring patient outcomes, thus providing value enhancement for key stakeholders, including care providers and payors. For more information, please visit https://www.acgtvector.com/.

About Xcell Biosciences

Xcellbio is driven by its mission to enhance the performance and safety of cell and gene-based therapies through the design and development of revolutionary technology platforms. The companys commercial instruments and AI-driven software allow researchers to discover novel insights into immune and tumor biology and enable the translation of these insights at patient scale through the development of its pioneering cell therapy manufacturing platform. Based in San Francisco, Xcellbio can also be found online at http://www.xcellbio.com.

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Xcell Biosciences and aCGT Vector Collaborate to Accelerate Development of Cell and Gene Therapies - Business Wire

INCHEON AIRPORT, SEOUL, SOUTH KOREA - 2018/06/12: A young girl communicating with a robot that is on ... [+] display at Incheon International Airport in Seoul / South Korea. The Guide Robot recognises languages; In addition to English, it can also answer Korean, Chinese and Japanese and recognise boarding cards that are scanned on the touch screen. In South Korea robots are playing an increasing role in society. AI boosts the economy in the countrys rapidly growing industry. Still, threats by terminators and lost employment threats linger. (Photo by Jonas Gratzer/LightRocket via Getty Images)

The world of social technology and public-private partnerships has been rapidly changing, and organizations must adapt their strategies to stay ahead of the curve. UN recently announced that the Republic of Korea is one of the top leaders in the 2022 digital government ranking of the 193 United Nations Member States, scoring the highest in the scope and quality of online services, the status of telecommunication infrastructure, and existing human capacity. LG NOVA is a perfect example of a private sector enterprise constantly innovating and looking for new ways to drive growth. LG NOVA is a subsidiary of LG Electronics, one of the world's largest electronics firms headquartered in Korea. It is on a critical mission to reinvent social impact for the future. The company has been at the forefront of innovation for over five decades, and its commitment to social responsibility and sustainability has earned it a reputation as a trusted partner for businesses and organizations around the globe. In fact, LG Energy Solutions is teaming up with Honda to build the $3.5 billion battery factory and create 2,200 jobs, with an overall investment projected to reach $4.4 billion as part of a joint venture in southern Ohio. President Joe Biden recently mentioned that the Honda-LG joint venture was "committing more than $5 billion toward electric vehicle battery manufacturing and factory retooling across the state.

To realize its vision of becoming a leading global provider of social technology solutions, LG NOVA has invested heavily in its Mission for the Future program. The program is designed to identify and invest in early-stage companies working on cutting-edge technologies with the potential to impact society positively.

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In Silicon Valley and globally, LG NOVA seeks to achieve a leadership position in socially impactful technology solutions. The company is focused on investing in early-stage companies that are working on cutting-edge technologies with the potential to impact society positively. Social innovation is reinventing how we live, work, and play, and LG NOVA is committed to supporting the companies leading this charge. The organization can drive growth and create value for shareholders, customers, and partners.

According to Dr. Sokwoo Rhee, Corporate SVP of Innovation at LG Electronics and Head of the North America Innovation Center at LG NOVA, The key industries that the LG NOVA program is seeking submissions from are digital health, electric mobility, and the metaverse. Digital Health is going to be huge. Healthcare is already huge, and you can see that many people are jumping into it right now. Electric mobility is also exploding with new solutions for electric vehicles and charging infrastructure. The government is putting money into the market, so it will only get bigger. While metaverse is still in its early stage of development, LG is continuing to explore how we can play a leading role in its development. Its a key part of our future, and we anticipate that at some point, we'll have an excellent idea or solution that we can make commercially viable."

Dr. Sokwoo Rhee, Corporate SVP of Innovation at LG Electronics and Head of the North America ... [+]ca Innovation Center at LG NOVA speaking at 2022 LG NOVA Innovation Festival

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Significant trends in healthcare and electric mobility include the convergence of physical and digital health, the rise of personalized and precision medicine, the growth of the wearables market, and the increasing demand for EV infrastructure as the market for EVs grows. In the metaverse, the initiative looks for applications that can create new value for users by providing an immersive and interactive experience. Dr.Rhee, previously an Associate Director for Cyber-Physical Systems (CPS) Innovation at the National Institute of Standards and Technology, U.S. Department of Commerce, continued to emphasize that "In my opinion, healthcare is a key part of smart communities. Now, if you have to go to a hospital, it's not exactly a smart city; it's just a hospital. But when you are doing healthcare at home, also known as digital healthcare, it broadens to become a community issue. So I believe digital health indirectly contributes to the concept of a smart city." The notion of the smart city is a significant trend to watch in the industry, as it represents the future of urban living and has the potential to create substantial value for companies that can provide solutions that make cities more efficient and livable.

Moreover, according to Joshua Di Frances, Head of Incubation and Senior Director, Healthcare Strategy & Enterprise Business Development, "I focus primarily on healthcare and digital health. Some companies are hospital- and consumer-facing, but they're all looking at the patient and trying to improve health. But I think some companies in that area, like Digbi Health, are exciting. They're looking at personalized health using DNA, genetics, and microbiome to understand better what each person needs. Personalizing medicine represents a huge area of growth. Medicine is going that way; we've seen it for several years. LG has a screen presence at hospitals and a hardware presence, but we also have a powerful consumer brand, and healthcare is moving to the home. And I think there's an opportunity when you think about connected devices, remote patient monitoring, and empowering patients to understand their health better where we could play a significant role. I think our leadership sees that, and thats why health is a big area of focus for us."

According to Maria Patterson, Director of New Ventures, LG Electronics, and LG NOVA Entrepreneur in Residence, Electric Vehicles are a priority in the near future due to their lack of emissions and ability to be powered by renewable energy sources. However, there are concerns about the potential for EVs to overload the electrical grid, as they require a significant amount of power to charge. One way to address this issue is by using AI to model people's behavior and better understand when and how they will use their EVs. This information can then be used to manage the charging of EVs in a way that does not overload the grid. Additionally, it is essential to ensure that these new technologies are accessible to everyone. Those who cannot afford EV chargers should not have to pay for infrastructure improvements that primarily benefit those who can. She states, "Using AI to model peoples behavior is very interesting because it makes us realize everything is very much interconnected, especially with electrification.

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Based on the above information, it is evident that LG Electronics is focusing on emerging industries to stay ahead of the curve and create value for their shareholders, such as healthcare and electric vehicles. This aligns with vital global megatrends. The need for better healthcare solutions will only increase as the world's population ages, and the move towards electric vehicles is gathering momentum to combat climate change. LG is well-positioned to capitalize on these trends and create significant value for its shareholders in the years to come.

2022 LG NOVA Innovation Festival

LG NOVA has spotlighted several attractive solutions focusing on health care and electric vehicles as part of their leading finalists for the Mission for the Future challenge.

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With digital health capabilities key to delivering healthcare to diverse and disparate communities, LG NOVA is working on expanding and scaling greater access to healthcare and personal health services.

Digbi Health is a digital care platform to prevent and reverse chronic metabolic, digestive, and mental health conditions using gut microbiome insight, genetics, AI, and food-as-medicine. Together LG and Digbi will work to make care globally accessible at home and the office and advance cure.

LifeNome is a B2B2C precision health platform powered by genomics and AI, offering personalized health and well-being solutions to the world's leading enterprises. LG and LifeNome plan to bring to market the world's first precision maternal and family health platform, supporting pregnant individuals 24/7 from conception to the early stages of a child's life.

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Mindset Medical is a sensor-based technology platform that can use a camera in patient-owned devices to capture health and biological readings to assist doctors in enhancing medical diagnosis and treatment effectiveness. Expanding upon new services available to doctors and providers, LG's work with Mindset Medical is expected to make it easier for medical providers to understand patient health over time or during live virtual doctor visits.

XRHealth develops virtual treatment rooms, integrating immersive VR/AR technology, licensed clinicians, and real-time data analytics on one platform, providing a comprehensive therapeutic care solution for patients through the continuum of care, from the hospital to the patient's home. LG and XRHealth will continue to grow the service offering to deliver more insights to clinicians and enable more precise personalized care.

The solutions in this category lead to social innovation by bringing technology-enabled solutions to healthcare's most challenging problems and making it easier for people to get the care they need. Specifically, these companies are focused on making it easier for people to access care, whether that's through at-home solutions or by providing more data to doctors to help them make better decisions.

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With LG's commitment to sustainability as an organization, LG NOVA is working to pursue new opportunities to expand access to electric vehicles (EV) and electric mobility solutions by enabling more businesses to leverage their infrastructure to provide EV services.

Driivz empowers major EV service providers with an end-to-end EV charging and intelligent energy management software platform. LG and Driivz will work together to enable the hospitality industry across the United States to offer EV charging-as-a-service while optimizing their EV charging operations and providing their customers with an exceptional EV charging experience.

I-EMS Solutions, Ltd. uses innovative AI and blockchain-based distributed energy resource management systems (DERMS) and transactive energy software platforms to enable power optimization across smart cities, smart homes, and e-mobility. By working with I-EMS, LG will help modernize the electricity grid and optimize energy management to deliver better power management solutions for the market.

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SparkCharge offers an affordable and convenient way for electric vehicle owners to charge their EVs without a direct-access charger at home or on the road. LG and SparkCharge plan to work together to deliver turnkey EV solutions for businesses to create a stream of revenue from their parking spaces.

The companies in this category focus on making it easier for people to switch to electric vehicles and expanding the infrastructure necessary to support widespread adoption. This is an integral part of LG's commitment to sustainability, as electric vehicles have a lower environmental impact than traditional gas-powered cars.

LG NOVA's Mission for the Future program is a testament to the pressing need for social innovation globally. The company is working with startups in various industries to create new solutions for some of the world's most pressing problems. LG NOVA is committed to positive change in the world, from healthcare to electric vehicles. Other organizations can begin to emulate this program by establishing social innovation initiatives and partnering with startups to create new technologies and services that address pressing global issues.

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Important Mission By LG To Reinvent Society With Future Growth - Forbes

TUCSON, Ariz., Oct. 13, 2022 /PRNewswire/ -- Learn Look Locate (LLL) announced a partnership with Myriad Genetics, a leader in genetic testing and precision medicine, to advance awareness about hereditary breast cancer and risk assessment. As part of the partnership, Myriad will share educational information for breast cancer patients and survivors on LLL's doctor monitored and curated platform.

"We are partnering with Learn Look Locate to help further educate patients and healthcare providers about hereditary cancer testing," said Thomas Slavin, M.D., chief medical officer, Myriad Genetics. "This testing provides vital data-driven genetic insights and personalized medical information to help people with a personal or family history of cancer understand their risk of being born with a predisposition to cancer. With this information, patients and their providers can take steps to either proactively prevent disease or, if diagnosed with cancer, personalize their treatment."

The American Society of Breast Surgeons (ASBrS) recommends testing of all patients diagnosed with breast cancer. Patients without a breast cancer diagnosis, but have a family history of the disease, should discuss testing with their physicians. Genetic testing can provide an assessment of a patient's risk of developing breast cancer and provide data-driven medical information for use in personalizing medical management plans.

Myriad's MyRiskHereditary Cancer Test offers multi-gene panel testing that helps determine a patient's hereditary cancer risk associated with 11 primary cancer types, including breast cancer. Patients without breast cancer may also be eligible for further personalized risk stratification with RiskScore, a risk assessment tool that delivers a personalized five-year and remaining lifetime risk of breast cancer

"Genetic testing is one of the most powerful pieces of knowledge about yourself," said Allison DiPasquale, M.D., Breast Surgical Oncologist at Texas Oncology. "It can help predict your risk of certain cancers, help guide systemic therapy and surgical recommendations, as well as provide potential lifesaving information for relatives. Learning how to tap into the insights of your genes empowers both patients and family members. I am so honored to be part of LLL and partner with Myriad on the LLL webpage."

To learn more about hereditary cancer testing, visit LLL online. And stay up to date on the latest therapies and breast cancer news by following LLL on social media @learnlooklocate Facebook, Instagram, Twitter, LinkedIn.

About Myriad Genetics

Myriad Genetics is a leading genetic testing and precision medicine company dedicated to advancing health and well-being for all. Myriad develops and offers genetic tests that help assess the risk of developing disease or disease progression and guide treatment decisions across medical specialties where genetic insights can significantly improve patient care and lower healthcare costs. Fast Company named Myriad among the World's Most Innovative Companies for 2022. For more information, visit http://www.myriad.com.

About Learn Look Locate (LLL)

Founded in 2019 by breast cancer survivor, Cynthia Jordan, LLL represents a global movement for breast cancer support and education. As part of her guiding mission to diagnose breast cancer early and educate people, LLL provides answers to the most important questions patients have: What do I need to know? Who are my resources? Where do I turn now?

LLL focuses on early detection and early diagnosis, aggregating the latest technology and research, the most cutting-edge breast cancer treatments, and information from globally recognized doctors/specialists. Understanding breast cancer at the earliest possible point of a patient's journey is critical. LLL empowers everyone touched by this diagnosis with a supportive community who share their breast cancer stories all ages, genders, and at all stages of the disease.

Join the global movement and become part of the conversation at: http://www.LearnLookLocate.com.

Media Contact:Cynthia Jordan1-407-592-4474[emailprotected]

SOURCE Learn Look Locate

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Learn Look Locate Partners with Genetic Testing Leader, Myriad Genetics, in Educational Campaign - PR Newswire

If youve ever lied about your height on a dating app, we now know to whom you can address your grievances. No, not any reinforced societal expectations about what heterosexual relationships look like or how traditional masculinity or femininity manifests, but a stretch of genetic variations.

Height is one of the easiest physical characteristics to measure, and we know that its a trait with high hereditability, meaning inherited genes almost entirely determine it. This trait seems like it should be a fairly straightforward one to parse genetically, but the actual places where gene variations occur have been hard to pin down.

Researchers in Australia, the U.K., and the U.S. published a study on Wednesday in the journal Nature that provides evidence for more than 12,000 spots in the human genome that are associated with height.

Whats new In the largest and most diverse genome-wide association study (GWAS) to date, the studys authors have identified 12,111 places in the human genome associated with height. The team analyzed existing genetic data from nearly 5.4 million people, more than a million of whom were not of European ancestry.

Throughout ones DNA are single nucleotide polymorphisms (SNP, pronounced snip), which are spots for genetic variation. SNPs are, in part, whats responsible for eye and hair color, height variation, and pretty much any other inherited trait. These arent mutations but spots where a particular nucleotide (adenine, cytosine, guanine, and thymine) exists. These nucleotides encode certain traits.

This study has pinpointed more than 12,000 locations in the human genome where those SNPs coding for height occur.

Why it matters As simple as height may seem, theres so much we still dont know about it.

Loic Yengo, lead author, and statistical geneticist at the University of Queensland, says this work sets the stage for future genetic investigation of other traits and diseases, demonstrating that genetic data alone can contribute a significant portion of trait variation and risk of disease.

It also offers a guide on sample sizes. Though this is the largest GWAS to date, it will likely impel broader and broader studies with even more people.

Widening the sample size is important for the future of personalized medicine, Yengo wrote in an email to Inverse. The more genetic material included, the better researchers will get at identifying at-risk patients before an inherited disease or trait progresses beyond prevention.

More directly, this study teaches something about the biology of bone growth and can eventually help us design treatments for certain skeletal growth disorders, Yengo wrote to Inverse.

Digging into the details Genetic information on European ancestry is now saturated. There arent many more unknown common variants associated with height in this population. According to Yengo, these more than 12,000 genetic variants account for about 40 percent of height variation in European ancestry.

Weve more or less finished mapping them, at least for European ancestry, Joel Hirschhorn, senior author, and a pediatric endocrinologist at Boston Childrens Hospital tells Inverse. Genetic rarities, however, still remain. Yengo now wants to focus on rare variants present in less than 1 percent of that population.

And, there are plenty of other ancestry groups that must be investigated for common variants. In this study, fewer than 78,000 people represented South Asian ancestry. But Hirschhorn and Yengo point to the importance of better understanding height in African ancestries.

Ancestries with an s because of the large diversity existing on the African continent, Yengo writes. Considering human life originated in Africa, information from these populations could provide novel insights.

Humans all started as one relatively small group in Africa, Hirschhorn says. Some of that genetic diversity left Africa, and some of it stayed behind. Since these genes come from much older populations, it has more genetic variability because its had so much more time to evolve.

Theres more to discover and better ability to pinpoint where the right variants are, but to do that, you need large studies of folks with African ancestry, he says.

Whats next These analyses will only get bigger and more diverse. Yengos vision for this researchs future is to home in on rarer genetic variants that alter height and to find even more height variants in other populations.

Hirschhorns interested in the immediate biological understanding we can glean. The more relevant genes we understand, the better we can get to know the biological mechanisms behind how height and skeletal growth work. He also says this study offers an opportunity for prevention and intervention when it comes to genetic disorders or diseases that affect height.

And as a pediatric endocrinologist, I see a lot of kids where the parents come in because theyre concerned about their child growing too slowly, he says. As sci-fi as it may seem, its not too hard to look at that kids genetics to see if perhaps theyre going to end up on the shorter side based on variants identified. But if that kids genetics suggest they should end up on the taller side, then there may be something else going on worth investigating.

Nobody needs genetic testing to at least partially, if not mostly, understand their own height. If youre curious, see if you can learn your parents heights, and that should offer some insight.

LEARN SOMETHING NEW EVERY DAY.

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Scientists identify more than 12,000 spots in the human genome associated with height - Inverse

Former chairman of CareDx, Inc. and seasoned global industry executive with track record of transforming organizations will lead Kyverna and drive its growth as it aims to bring a new class of targeted gene therapies to treat severe immune-related diseases

Company plans to submit Investigational New Drug application for its lead program KYV-101, a novel CD19 CAR T-cell therapy, for the treatment of lupus nephritis

Dominic Borie, M.D., Ph.D., named president, research and development to continue advancing company's core program KYV-101 and strategic partnerships with Gilead and Intellia

Kyverna's therapeutic platform combines advanced T-cell engineering and synthetic biology technologies to suppress and eliminate autoreactive immune cells at the root cause of inflammatory disease

EMERYVILLE, Calif., Oct. 13, 2022 /PRNewswire/ -- Kyverna Therapeutics ("Kyverna"), a cell therapy company with the mission of engineering a new class of therapies for serious autoimmune diseases, today announced the appointment of Peter K. Maag, Ph.D., as the company's chief executive officer (CEO). Dr. Maag succeeds Dominic Borie, M.D., Ph.D., who has been named Kyverna's president of research and development (R&D). Dr. Maag also joins Kyverna's board of directors.

"We are thrilled to welcome Peter as Kyverna's new CEO," said Ian Clark, chair of Kyverna's board of directors. "Peter is an accomplished executive with extensive industry experience at private and public companies spanning three continents.His consistent record of driving transformational growth in healthcare through product innovation, creative business development, sequential financings, and sound investment strategieswill be invaluable to progress Kyverna and deliver on its mission to help bring new and much-needed treatment options to patients with serious autoimmune and inflammatory diseases."

Dr. Maag has more than 20 years of executive management experience in the pharmaceutical and diagnostic industry. Most recently, he was executive chairman, president and CEO of CareDx where he had built the company from a small start-up into a public company and industry-leading powerhouse in transplantation through a series of BD&L and financing transactions. CareDx serves the transplant community with breakthrough technologies in more than 60 countries and has created a strong presence in this specialty market. Prior to joining CareDx in 2012, Peter held multiple positions in Novartis with increasing responsibilities. As President of Novartis Diagnostics, he drove growth and innovation in its blood-screening business. Previously, he led one of Novartis' key affiliates as country president, Germany, and lived in a dynamically growing market as country president, Korea. At headquarters in Switzerland, he served as the head of strategy for Novartis Pharmaceuticals and helped launch the Infectious Diseases franchise.

"I am passionate about patient care and am honored to be appointed to lead Kyverna and this extraordinarily talented team. This is such an exciting time to join the company with key milestones like our submission of an IND for our lead candidate, KYV-101 in lupus nephritis tomorrow. This important regulatory milestone will provide a runway to move KYV-101 into the clinical phase and brings us a step closer to developing a new treatment option for lupus nephritis," said Dr. Maag. "I believe that Kyverna is at the forefront of revolutionizing how we treat severe immune-related and inflammatory diseases with its new class of cell therapies. With multiple shots on goal, I believe that Kyverna is well positioned to transform how autoimmune diseases are treated."

Prior to joining Novartis, Peter worked at McKinsey & Company in New Jersey and Germany, focusing on pharmaceuticals and globalization strategies. Besides supporting various healthcare and tech companies in their growth efforts, he holds board and advisory positions at Phoenix Pharma SE, CareDx, MiroMatrix, and the Personalized Medicine Coalition. Peter studied pharmaceutical sciences in Heidelberg, London, and Berlin.

In addition to Dr. Maag's appointment, Kyverna announced that Dr. Borie will step away from his CEO role and has been named president of R&D.

"We are grateful for the significant contributions Dominic has made in bringing Kyverna out of stealth in 2020 and growing the company and building out Kyverna's core programs," added Mr. Clark. "Dominic has performed admirably, and we could not be more pleased that he will serve as president of Kyverna's R&D operations. We look forward to working closely with him and his team to continue moving forward our autologous and allogeneic programs, as well as our collaborations with Gilead and Intellia so that we can continue to strive to bring new treatments to patients rapidly."

About KYV-101KYV-101 is an autologous version of a novel clinical-stage anti-CD19 chimeric antigen receptor T-cell (CAR T) construct with properties well suited for use in B cell-driven autoimmune diseases such as lupus nephritis, systemic sclerosis, and inflammatory myopathies. Kyverna has obtained exclusive, worldwide licenses from the National Institutes of Health (NIH) to use this CD19 construct in both autologous and allogeneic CAR T-cell therapies.

About Kyverna TherapeuticsKyverna Therapeutics is a cell therapy company with the mission of engineering a new class of therapies for autoimmune and inflammatory diseases. The Kyverna therapeutic platform combines advanced T-cell engineering and synthetic biology technologies to suppress and eliminate the autoreactive immune cells at the origin of autoimmune and inflammatory diseases. In addition to aiming to develop next-generationchimeric antigen receptor T-cell (CAR T) therapies in both autologous and allogeneic settings, Kyverna is creating synReg T cells, a synthetic version of Regulatory T cells (Tregs), powerful natural immune cells that control immune homeostasis through multiple immunosuppressive mechanisms. By offering more than one mechanism for taming autoimmunity, Kyverna is positioned to act on its mission of transforming how autoimmune diseases are treated. For more information, please visit https://kyvernatx.com.

SOURCE Kyverna Therapeutics

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Kyverna Therapeutics Names Peter Maag, Ph.D., as Chief Executive Officer - PR Newswire

Pokagon Flag

Harper Institute collecting Native American samples to address health disparities

Dr. Gerald Morris, the medical director for the Pokagon Band of Potawatomi Indians that live in northern Indiana and southwest Michigan, isnt certain that the Native American patients he sees show higher rates of cancer than the rest of the population though he suspects it.

The one thing that stands out to me is, oftentimes, theyre more advanced when we find them, said Morris, who is a Pokagon citizen. I think they have the same issues as other patients in terms of the types of cancers. Its just that they tend to be more advanced when I do see them.

Morris said the reason the cancers are more advanced could trace back to historical access to health care, which leads to lower rates of cancer screening. He hopes that a partnership being developed between the Pokagon Band and the University of Notre Dame'sHarper Cancer Research Institute (HCRI) will help raise awareness of the importance of screening and early detection to address measurable disparities in cancer treatment outcomes involving Native Americans.

The Pokagon Bands tribal leadership plans to invest $25,000 to help fund the project. Scott Brewer, a senior vice president at the tribes Four Winds Casino Resorts, oversees the companys philanthropic activities and proposed the HCRI project to his tribal council. He said the research disparities Harper presented speak for themselves of about 90,000 cancer tissue samples in the Cancer Genome Atlas at the National Cancer Institute, only 114 came from Native Americans.

Thats .001 percent of the total, even though people identifying as at least partially American Indian or Alaska Native constitute about 3 percent of U.S. population.

Weve partnered with the University on several things, but this is the first health initiative, Brewer said. It was the easiest sell for me because its research to try to get a cure, and the research is specific to Native Americans.

The project aims to double the number of cancer tissue samples that come from Native Americans through several outreach efforts. While Harper is in the early stages of relationship building with the Pokagon Band, it has been working for a few years with other minority groups: the Kalispel tribe in eastern Washington state, and medical groups in Puebla, Mexico.

Andy Bullock, associate director of HCRI, explained why its so important to diversify the cancer tissue samples available for research. His rationale starts with the direction the future of cancer research is taking.

Cancer is not one disease, its thousands, Bullock said. It used to be cancer of the breast, colon, kidney, etc. With more personalized medicine, its now more about the type of mutation.

Cancer comes from a mutation in a persons DNA, leading cells to grow uncontrollably in different ways. Targeted cancer drugs and chemotherapy attempt to block different pathways that are turned on by these mutations in the body. But people of different race or ethnicity with the same cancer might have different mutations that activate the cancer-driving pathway.

It might be different between a Caucasian person and Native American person and African American person, said M. Sharon Stack, the Anne F. Dunne and Elizabeth Riley Director of HCRI. We don't know for most cancers in minority populations because nobodys really looked.

Most cancer tissue is collected at elite cancer centers, where there is an over-representation of white patients that can afford treatment there. Minority cancer patients are more likely to seek treatment at local, often less-costly cancer centers. These trends drive the disparity that leads to such low numbers of Native Americans in the Cancer Genome Atlas.

The current model is we come up with a drug that helps white people, then hope it helps other people too, Bullock said. We are trying to invert that model. We want to research Native Americans and see how well those pathway blockers help them and other people.

Stack said the project fits the Universitys mission of serving populations often marginalized in the past, but its also a really compelling scientific question.

A 2022 American Cancer Society report on American Indian and Native Alaskan populations found that cancer incidence in these indigenous communities is higher nationally than among whites, especially for lung, colorectal and kidney cancers. The report notes that lower levels of health insurance and chronic underfunding of the U.S. Indian Health Services exacerbates the problem.

Within the national numbers are wide variations, partly because there are 574 federally recognized tribes and more than 200 that remain unrecognized. Even when factors like health care and poor housing are equalized, wide disparities in cancer cases remain. For instance, Northern Plains Indians are four times more likely to have kidney cancer than whites. Survivability charts also show disparities, likely due to later detection.

Bullock said HCRI has been working for several years with partners in eastern Washington because an alumnus there donated money for cancer research. Ryan Gee, who graduated in 1998, is CEO of Gee Automotive companies with 35 car dealerships across several western states.

Gee, whose wife survived cancer, said the fight against the disease is his side passion. He co-founded the Community Cancer Fund with a friend who survived cancer against great odds. The organization raises money for cancer research and to help families in the Northwest cope with the severe economic impact of cancer treatment.

The Kalispel Indian tribe is a large donor to the fund, so Gee asked Harper about cancer research among Native Americans and learned it was lacking. What if we set a goal to double the amount of Native American research samples in the database through Harper at Notre Dame, Gee said.

Gees nonprofit works with the MultiCare hospital system in Washington state, which has helped Harper collect about 20 cancer tissue samples from Native Americans so far.

Native American populations experience much higher cancer rates than non-Hispanic white people in the U.S and are historically under-represented and critically under researched, said Annie Reedy, MultiCares chief research and education officer. Through this collaboration with Notre Dame we are moving toward a greater understanding of cancers in Native Americans and building a foundation for future clinical trials and treatment advances for this population.

Fundraising is an important part of this research because the cancer tissues are expensive to analyze. When a lump or tumor is removed from a patient, the doctors will perform a biopsy on a small slice to determine if its cancerous. The rest is considered medical waste.

Stack said it can be difficult to convince people to share the leftover portions with tissue banks, especially if the target population is mistrustful of the medical community. Relationship building can take years. A tissue bank must strip out identifying information that could lead back to the donor, leaving only basic demographic information.

Harper contracts with a Chicago company that extracts DNA from samples and looks for about 600 mutations linked to cancer. The $2,500 process also analyzes the RNA sequence, which is a blueprint of the DNAs genetic code that gets made into the proteins that form hair and skin and bones the different parts of the body. Mens basketball coach Mike Brey raised money for the project through his Coaches vs. Cancer work.

Another branch of this ongoing project works with partners in Puebla, Mexico. There is a huge disparity in childhood leukemia survival between people in America and Mexico. In America, more than 90 percent of leukemia patients live to the 10-year marker that signals successful treatment. In Mexico, only about 60 percent live for four years.

HCRI has been collecting samples from pediatric patients in South Bend to compare them with samples from Mexican patients.

Ideally we would have Caucasian kids and also kids of Latin American origin that happened to be raised in South Bend to see if any differences are genetic, or if it could be an environmental effect, Stack said.

Maria Cristina Miranda Vergara earned a doctoral degree in biochemistry from Notre Dame in May. As a student, she studied biomarkers that could improve early cancer detection, working with partners at the Catholic Universidad Popular Autnoma del Estado de Puebla. Now working in biochemical research in Puebla, Miranda Vergara helps process Mexican samples using the same protocols so that the data can be compared to Notre Dames research.

My dream is to develop some sort of microfluidic device that would allow us to detect cancer biomarkers in the field, so we could do early diagnosis of children in their communities, she said. Its difficult to determine the symptoms of cancer, but we need to improve early detection to get the right treatment and improve outcomes.

The common thread between the Harper projects in Mexican and Native American communities is to improve cancer survival by analyzing distinct populations that have not been studied as thoroughly in the past.

Priscilla Gatties, director of Health Services for the Pokagon Band, said Harpers research can help address disadvantages that Native American communities have developed due to a lack of historical health data. She said her mother-in-law, a Pokagon citizen, is fighting against kidney cancer using medication that didnt exist a decade ago.

From my perspective, Im very excited about what Harper is doing, Gatties said. Theres a lot of new research and information, and we can be a part of that. I think thats going to help patients and save lives, even if its later on down the road.

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The future of cancer research | News - ND Newswire

Future Market Insights Global and Consulting Pvt. Ltd.

The laboratory information systems market in Germany is set to exhibit a growth at a 9.9% CAGR during the forecast period 2022 -2030. Sales of laboratory information systems in China are forecast to increase at a 9.4% CAGR over the forecast period

NEWARK, Del, Oct. 11, 2022 (GLOBE NEWSWIRE) -- The global laboratory information systems market is worth US$ 1.9 Bn as of now and expected to reach US$ 4.2 Bn by the year 2030 at a CAGR of 10.2% between 2022 and 2030.

Laboratory Information System (LIS) comes across as a collection of operating systems, software, and hardware used for processing, storing, and managing data in the clinical laboratories. As such, it has turned out to be one of the essential tools to support operations in the modern laboratories. They are inclusive of managing and processing data pertaining to several processes and testing.

The healthcare vertical has witnessed several technological advancements in the last few years, and laboratory information systems are no exception. The application areas include sample tracking and also as enterprise resource planning tools managing several facets of informatics. The systems actually let end-users accumulate patients data and also record it on database for managing the patients better.

Besides, quick adoption of explicit research on genomic studies and automation is catalysing utilization of laboratory information systems. With growing biobanks, notable investments in the novel technologies, easily available laboratory information systems products, services, and stern regulatory requirements across all the industries, the laboratory information systems market is all set to grow on an unstoppable note in the near future.

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Additionally, standardization of clinical trials coupled with modernization of regulatory pathways in the Peoples Republic of China is aiding the growth of laboratory information systems market. Cloud-based services are being rapidly adopted. High-speed internet is another factor to be attributed to.

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The ongoing trend implies tabling dedicated laboratory information systems with custom, commercial, and open source software interface with diverse budgetary specifications. Future Market Insights has etched these findings with future perspectives in its latest market study entitled Laboratory Information Systems Market.

Key Takeaways from Laboratory Information Systems Market

North America holds the largest market share due to the US being subject to quick adoption of well-structured healthcare IT sector and lab automation and the status quo is expected to remain unchanged even in the forecast period.

Europe stands second, with Germany leading from the front. It is expected to witness a CAGR of 9.9% in laboratory information systems market in the forecast period due to rising adoption of automation.

The Asia-Pacific is expected to grow at the fastest pace in the laboratory information systems market with China yielding personalized medicine with feasible support from the government.

Cloud-based laboratory information systems are dominating the market and would continue to do so even going forward.

By application, clinics hold the largest market share (over 48%).

Automation, in line with Industry 4.0, is expected to accelerate the laboratory information system (LIS) market in the forecast period, says an analyst from Future Market Insights.

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Competitive Arena

CTI, in August 2021, entered into partnership with LabWare Inc. with the objective of expanding the formers laboratory services for supporting research related to cell & gene therapy.

Oracle Corporation, in December 2021, partnered with Cerner Corporation for jointly announcing acquisition of the latter.

Orchard Software Corporation, in May 2021, completed acquisition of Corwen, LLC for expanding molecular testing capabilities and make way for integrated and comprehensive LIS solutions.

PathWest, in January 2021, integrated its SoftBank (new LIS transfusion medicine module) with NBAs (National Blood Authoritys) BloodNet LIS Interface.

CompuGroup, in December 2020, completed acquisition of Schuyler House for strengthening the footprint all across the US.

LabVantage Solutions, in July 2020, did launch its 100% integrated Scientific Data Management Systems (SDMS) via its LabVantage 8.5 edition.

Abbott, in July 2019, came up with STARLIMS Life Sciences Solution 11.1 for managing complex testing and sample workflows.

What does the Report Cover?

Future Market Insights offers an exclusive perspective and various real-time insights on the laboratory information systems market in its latest study, presenting historical demand assessment of 2016 2021 and projections for 2022 2030.

The research study is based on component (hardware, software, and services), by delivery mode (on-premise and cloud-based), and by end-user (hospitals, clinics, independent laboratories, and likewise).

Browse Full Report @ https://www.futuremarketinsights.com/reports/laboratory-information-systems-market

Key Market Segments Covered in Laboratory Information Systems Industry Research

By Components:

By Delivery Mode:

By End User:

Hospitals

Clinics

Independent Laboratories

Others

By Region:

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Table of Content

1. Executive Summary

1.1. Global Market Outlook

1.2. Analysis and Recommendations

2. Global Laboratory Information Systems Market Overview

2.1. Introduction

2.1.1. Global Laboratory Information Systems Market Definition

2.1.2. Global Laboratory Information Systems Market Taxonomy

2.2. Market Dynamics

2.2.1. Drivers

2.2.2. Restraints

2.2.3. Opportunity

2.3. Global Laboratory Information Systems Market Trends

2.4. Global Laboratory Information Systems (LIS) Market Demand (in Value in US$ Mn) Analysis 2015-2021 and Forecast, 20222030

2.5. Global Laboratory Information Systems Market Size (Value) Forecast

2.5.1. Y-o-Y Growth Projections

2.5.2. Absolute $ Opportunity

Read More TOC.

Have a Look at Related Research Reports of Healthcare

Radiology Information System RIS Market Size: The Radiology Information System market is projected to register a CAGR of 7.6% during the forecast period, up from US$ 809.8 million in 2020 to reach a valuation of US$ 1,315.6 million by 2026

Clinical Information System Market Sales: The clinical information system market is expected to record a CAGR of 8% during the forecast period, reaching a valuation of US$ 1560 Mn by 2027

Laboratory Filtration Devices Market Trends: Laboratory Filtration Devices Market - Global industry segment analysis, regional outlook, share, growth; laboratory filtration devices market forecast 2017 to 2027 by future market insights

Oncology Information Systems Market Share: The oncology information systems market is projected to be worth US$ 2.59 Bn by 2022 and is expected to grow to US$5.4 Bn by 2032, at a CAGR of 7.8% from 2022 to 2032

Clinical Decision Support Market Growth: The global clinical decision support market is likely to reach a US$ 2.2 billion valuation in 2022

About Future Market Insights, Inc.

Future Market Insights, Inc. is an ESOMAR-certified business consulting & market research firm, a member of the Greater New York Chamber of Commerce and is headquartered in Delaware, USA. A recipient of Clutch Leaders Award 2022 on account of high client score (4.9/5), we have been collaborating with global enterprises in their business transformation journey and helping them deliver on their business ambitions. 80% of the largest Forbes 1000 enterprises are our clients. We serve global clients across all leading & niche market segments across all major industries.

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Call for Accurate Automation in Healthcare Practices to drive the Laboratory Information Systems (LIS) Market | Future Market Insights, Inc. - Yahoo...

Dublin, Oct. 11, 2022 (GLOBE NEWSWIRE) -- The "Stem Cell Manufacturing Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2022-2027" report has been added to ResearchAndMarkets.com's offering.

The global stem cell manufacturing market size reached US$ 11.2 Billion in 2021. Looking forward, the publisher expects the market to reach US$ 18.59 Billion by 2027, exhibiting a CAGR of 8.81% during 2021-2027.

Stem cells are undifferentiated or partially differentiated cells that make up the tissues and organs of animals and plants. They are commonly sourced from blood, bone marrow, umbilical cord, embryo, and placenta. Under the right body and laboratory conditions, stem cells can divide to form more cells, such as red blood cells (RBCs), platelets, and white blood cells, which generate specialized functions.

They are widely used for human disease modeling, drug discovery, development of cell therapies for untreatable diseases, gene therapy, and tissue engineering. Stem cells are cryopreserved to maintain their viability and minimize genetic change and are consequently used later to replace damaged organs and tissues and treat various diseases.

Stem Cell Manufacturing Market Trends:

The global market is primarily driven by the increasing venture capital (VC) investments in stem cell research due to the rising awareness about the therapeutic potency of stem cells. Apart from this, the widespread product utilization in effective disease management, personalized medicine, and genome testing applications are favoring the market growth. Additionally, the incorporation of three-dimensional (3D) printing and microfluidic technologies to reduce production time and lower cost by integrating multiple production steps into one device is providing an impetus to the market growth.

Furthermore, the increasing product utilization in the pharmaceutical industry for manufacturing hematopoietic stem cells (HSC)- and mesenchymal stem cells (MSC)-based drugs for treating tumors, leukemia, and lymphoma is acting as another growth-inducing factor.

Moreover, the increasing product application in research applications to produce new drugs that assist in improving functions and altering the progress of diseases is providing a considerable boost to the market. Other factors, including the increasing usage of the technique in tissue and organ replacement therapies, significant improvements in medical infrastructure, and the implementation of various government initiatives promoting public health, are anticipated to drive the market.

Key Players

Key Questions Answered in This Report:

Key Market Segmentation

Breakup by Product:

Breakup by Application:

Breakup by End User:

Breakup by Region:

Key Topics Covered:

1 Preface

2 Scope and Methodology

3 Executive Summary

4 Introduction

5 Global Stem Cell Manufacturing Market

6 Market Breakup by Product

7 Market Breakup by Application

8 Market Breakup by End User

9 Market Breakup by Region

10 SWOT Analysis

11 Value Chain Analysis

12 Porters Five Forces Analysis

13 Price Analysis

14 Competitive Landscape

For more information about this report visit https://www.researchandmarkets.com/r/5iujo7

Link:
Stem Cell Manufacturing Global Market Report 2022: Widespread Product Utilization in Effective Disease Ma - Benzinga

By David Willey, Benzinga

Melville, NY --News Direct-- BioRestorative Therapies, Inc.

Biotechnology companies have broken many medical barriers in the past 40 years, harnessing the technological revolution to bring innovative solutions to medical problems.

One flourishing field in biotech is regenerative medicine, a market worth $16.9 billion in 2021. Regenerative medicine harnesses the bodys amazing ability to heal itself, using cutting-edge technology to apply this regenerative power to prompt the body to recover from diseases previously uncured.a

An estimated one-third of Americans would benefit from regenerative therapeutic cures. There are wide applications for such regenerative therapy, with categories including stem cell research, gene therapy and tissue engineering. Some difficulties for regenerative medicine companies include arduous Food and Drug Administration (FDA) trial processes and the need for vertical integration of their product development to cut expenses.

Biotech companies involved in regenerative medicine include Mesoblast Ltd. (NASDAQ: MESO), Brainstorm Cell Therapeutics Inc. (NASDAQ: BCLI), Lineage Cell Therapeutics (NYSE: LCTX) and BioRestorative Therapies Inc. (NASDAQ: BRTX).

Here is a look at some companies looking to be leaders in the regenerative therapy field:

Mesoblast Ltd. develops novel treatments for back pain and various cardiovascular conditions. This Australia-based company focuses primarily on cell therapy solutions, with a mesenchymal lineage stem cell (MSC) technology platform. This develops MSCs, highly multipotent cells taken from healthy bone marrow, and develops treatments for tissue damage, heart disease and more.

Lineage Cell Therapeutics is a company pioneering cell-based therapies to treat serious diseases, including ocular disorders and cancer. It uses its proprietary cell-therapy platform to develop and manufacture self-renewing stem cells into differentiated cells, which can be transplanted to treat problems including cancer or degenerative diseases.

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Brainstorm Therapeutics focuses on cell therapies for neurodegenerative diseases. Its autologous cellular therapeutics platform NurOwn treats the disease by differentiating the patients healthy MSCs. Brainstorms work may eventually provide treatments for such neurodegenerative diseases as Alzheimers.

BioRestorative Therapies, which primarily develops products using highly therapeutic adult stem cells, focuses on disc/spine disease and metabolic disorders. BioRestoratives brtxDisc program is developing a treatment for the millions of Americans suffering from either chronic or acute back pain. Its product BRTX-100, which uses autologous stem cells to treat degenerative spinal discs, is in a Phase Two FDA trial.

BioRestorative is also tackling obesity, which currently affects over 40% of Americans. It is developing the product ThermoStem, which harnesses the bodys natural production of healthy brown fat cells to target patient obesity and other metabolic issues associated with obesity.

BioRestorative believes that its treatments will also help condition the body for better future regeneration and responses to medical treatment. A significant advantage for the company is the vertical integration of development and production it has through its clinical-grade cell therapy manufacturing facility. This facility, completed in April, gives BioRestorative control and oversight in the cell manufacturing process, apart from the flexibility to make its own decisions and to correct quality issues in real-time. Owning the facility mitigates the expense normally associated with these activities, which is a great benefit when conducting FDA trials.

Learn more about BioRestorative by visiting its website.

BioRestorative Therapies was founded by scientists and researchers committed to developing stem cell therapies to address unmet needs in patients with highly prevalent conditions.Our advances in stem cell biology and delivery protocols harbor great promise in conditioning our bodies own regenerative potential to treat major diseases more effectively than current interventions.Today, BioRestorative is actively developing programs that aim to dramatically increase quality of care for both (i) chronic back pain caused by disc degeneration, as well as (ii) metabolic disorders including obesity and diabetes.

This post contains sponsored advertising content. This content is for informational purposes only and is not intended to be investing advice.

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A Look At Some Of The Companies Innovating In the Cutting-Edge Regenerative Medicine Field - Yahoo Finance

As the 3rd presenter during the morning session of the American Society for Dermatologic Surgery Meeting, Emerging Concepts, Saranya Wyles, MD, PhD, assistant professor of dermatology, pharmacology, and regenerative medicine in the department of dermatology at the Mayo Clinic in Rochester, Minnesota, explored the hallmarks of skin aging, the root cause of aging and why it occurs, and regenerative medicine. Wyles first began with an explanation of how health care is evolving. In 21st-century health care, there has been a shift in how medical professionals think about medicine. Traditionally,the first approach was to fight diseases, such as cancer, inflammatory conditions, or autoimmune disorders. Now, the thought process is changing to a root cause approach with a curative option and how to rebuild health. Considering how to overcome the sequence of the different medications and treatments given to patients is rooted in regenerative medicine principles.

For skin aging, there is a molecular clock that bodies follow. Within the clock are periods of genomic instability, telomere attrition, and epigenetic alterations, and Wyles lab focuses on cellular senescence.

We've heard a lot atthis conference about bio stimulators, aesthetics, and how we can stimulate our internal mechanisms of regeneration. Now, the opposite force of regeneration isthe inhibitory aging hallmarks which include cellular senescence. So, what is cell senescence? This isa state that the cell goes into, similar to apoptosis or proliferation, where the cell goesinto a cell cycle arrest so instead of dividing apoptosis, leading to cell death,the cell stays in this zombie state, said Wyles.

Senescence occurs when bodies require a mutation for cancers. When the body recognizes there is something wrong, it launches itself into the senescent state, which can be beneficial. Alternatively, chronic senescence seen with inflammageing, like different intrinsic markers, extrinsic markers, and UV damage, is a sign of late senescence. Senescence cells can be melanocytes, fibroblasts, and cells that contribute to the regeneration of the skin.

I think were in a very exciting time ofinnovation and advancements in medicine, which is the meeting of longevity science of aging and regenerative medicine, said Wyles.

Regenerative medicine is a new field of medicine that uses native and bioengineered cells, devices, and engineering platforms with the goal of healing tissues and organs byrestoring form and function through innate mechanisms of healing.Stem cell therapy and stem cell application are commonly referenced with regenerative medicine. Typically, first-in-class treatments include cells, autologous or allogeneic, different types of cells that areassociated with high-cost due to the manufacturing.

With regenerative medicine, there's a new class of manufacturing. Regenerative medicine is not like traditional drugs where every product is consistent. These are cells, so the idea of manufacturing, and minimally manipulating, all comes into play. Now, there's a new shift towards next-generation care. This is cell-free technology. So, this is the idea of exosomes, because these are now products from cells that can be directly applied, they can be shelf-stable, accessible, and more cost-effective, said Wyles.

Exosomes are the ways that the cells communicate with each other. Cells have intercellularcommunications and depending on the source of the exosomes, there can be different signals. Wyles focused specifically on a platelet product, which is a pooled platelet product that can be purified and used for different mechanisms including wound healing, fat grafting, degenerative joint disease, and more.In a cosmetic studyconducted by Mayo Clinic, a topical platelet exosome product was applied to the face in the morning and the evening. Application included a 3-step regimen, a gentle cleanser, a platelet exosomeproduct, and then a sunscreen.

After 6 weeks, there was a significant improvement in redness and a 92% improvement in the hemoglobin process. Vasculature also improved across age groups. The study enrolled 56patients, and the average age was 54. Patients in their 40s, 50s, and 60s saw consistent improvement in redness and skin aging.

Lastly, Wyles stressed that as dermatologists think through the science-driven practices of these innovative strategies for skin aging, wound healing, and other regenerative approaches, they must think about responsible conducts of research. Currently, there are no FDA indications for exosomes being injected.

Reference:

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The Switch to Regenerative Medicine - Dermatology Times

Carlsbad, CA, Oct. 11, 2022 (GLOBE NEWSWIRE) -- The Alliance for Regenerative Medicine (ARM), the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies, today announced the election of its 2023 Officers, Executive Committee, and Board of Directors.

The announcement comes as ARM kicks off its 2022 Cell & Gene Meeting on the Mesa, a gathering of 1,800 leaders in the cell and gene therapy sector.

The Executive Committee and Board of Directors oversee the formation and execution of ARMs strategic priorities and focus areas. These distinguished leaders are instrumental to ARMs leadership of the sector.

We are delighted to welcome our 2023 Officers, Executive Committee members and Board of Directors, said ARMs Chief Executive Officer Timothy D. Hunt. The pipeline of transformative cell and gene therapies will continue to accelerate in 2023, creating more urgency to ensure that patients have access to life-changing medicines. ARMs Board of Directors and our more than 450 member organizations globally are vital to this mission.

ARM 2023 Officers:

Devyn Smith, Ph.D. Chief Executive Officer, Arbor Biotechnologies (Chair)

Dave Lennon, Ph.D. Chief Executive Officer, Satellite Bio (Vice Chair)

Alison Moore, Ph.D. Chief Technology Officer, Allogene Therapeutics (Secretary)

Chris Vann Senior Vice President, Chief Operations Officer, Autolus (Treasurer)

ARM 2023 Executive Committee:

Devyn Smith, Ph.D. Chief Executive Officer, Arbor Biotechnologies (Chair)

Dave Lennon, Ph.D. Chief Executive Officer, Satellite Bio (Vice Chair)

Alison Moore, Ph.D. Chief Technology Officer, Allogene Therapeutics (Secretary)

Chris Vann Senior Vice President, Chief Operations Officer, Autolus (Treasurer)

Bob Smith, MBA Senior Vice President, Global Gene Therapy Business, Pfizer

Miguel Forte, M.D., Ph.D. Chief Executive Officer, Bone Therapeutics

Laura Sepp-Lorenzino, Ph.D. Executive Vice President and Chief Science Officer, Intellia Therapeutics

Arthur Tzianabos, Ph.D. Chair of the Board, Homology Medicines

ARM 2023 Board of Directors

* New to the Board for 2023

* Faraz Ali, MBA Chief Executive Officer, Tenaya Therapeutics

Robert Ang, MBBS, MBA Chief Executive Officer, Vor Biopharma

* Catherine Bollard, M.B.Ch.B., M.D. Director of the Center for Cancer and Immunology Research, Childrens National Hospital and The George Washington University

Amy Butler, Ph.D. President, Biosciences, Thermo Fisher

Bradley Campbell, MBA President and Chief Executive Officer, Amicus Tx

Miguel Forte, M.D., Ph.D. Chief Executive Officer, Bone Therapeutics

* Christine Fox President, Novartis Gene Therapies

Bobby Gaspar, M.D., PhD. Chief Executive Officer, Orchard Therapeutics

Jerry Keybl, Ph.D. Senior Director, Cell & Gene Therapy, MilliporeSigma

Brett Kopelan Executive Director, Debra of America

* Ann Lee, Ph.D. Chief Technical Officer, Prime Medicine

Dave Lennon, Ph.D. Chief Executive Officer, Satellite Bio

Tim Lu, M.D., Ph.D. Chief Executive Officer and Co-Founder, Senti Biosciences

John Maslowski, M.S. Chief Commercial Officer, Forge Biologics

Chris Mason, M.D., Ph.D. Founder & Director, Ori Biotech

Debra Miller Founder & Chief Executive Officer, CureDuchenne

Alison Moore, Ph.D. Chief Technology Officer, Allogene

Adora Ndu, PharmD, J.D. Chief Regulatory Officer, BridgeBio

Susan Nichols President & Chief Executive Officer, Propel BioSciences

Emile Nuwaysir, Ph.D. Chief Executive Officer, Ensoma

Karah Parschauer, J.D. Chief Legal Officer, Ultragenyx

* Jacob Petersen Corporate Vice President and Head of Stem Cell Research & Development, Novo Nordisk

Louise Rodino-Klapac, Ph.D. Executive Vice President, Head of Research & Development, Chief Scientific Officer, Sarepta Therapeutics

Jeff Ross, Ph.D. Chief Executive Officer, Miromatrix Medical

* Alberto Santagostino Senior Vice President, Head of Cell & Gene Technologies, Lonza

Laura Sepp-Lorenzino, Ph.D. Executive Vice President & Chief Scientific Officer, Intellia Therapeutics

R.A. Session, MBA, MSF President, Founder & Chief Executive Officer, Taysha Tx

Curran Simpson, M.S. Chief Operations and Chief Technical Officer, REGENXBIO

Bob Smith, MBA Senior Vice President, Global Gene Therapy, Pfizer

Devyn Smith, Ph.D. Chief Executive Officer, Arbor Biotechnologies

Arthur Tzianabos, Ph.D. Chair of the Board, Homology Medicines

Christopher Vann Senior Vice President & Chief Operating Officer, Autolus Therapeutics

Kristin Yarema, Ph.D. Chief Commercial Officer, Atara Bio

Continued here:
The Alliance for Regenerative Medicine Announces Election of 2023 Officers, Executive Committee, and Board of Directors - GlobeNewswire

Until recently, researchers could not see gene expression in an individual cell. Thanks to single cell sequencing techniques, they now can. But the timing of changes is still hard to visualize, as measuring the cell destroys it.

To address this, we developed an approach based on models in basic physics, explained Welch, treating the cells like they are masses moving through space and we are trying to estimate their velocity.

The model, dubbed MultiVelo, predicts the direction and speed of the molecular changes the cells are undergoing.

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Our model can tell us which things are changing firstepigenome or gene expression--and how long it takes for the first to ramp up the second, said Welch.

They were able to verify the method using four types of stem cells from the brain, blood and skin, and identified two ways in which the epigenome and transcriptome can be out of sync. The technique provides an additional, and critical, layer of insight to so called cellular atlases, which are being developed using single cell sequencing to visualize the various cell types and gene expression in different body systems.

By understanding the timing, Welch noted, researchers are closer to steering the development of stem cells for use as therapeutics.

One of the big problems in the field is the artificially differentiated cells created in the lab never quite make it to full replicas of their real-life counterparts, said Welch. I think the biggest potential for this model is better understanding what are the epigenetic barriers to fully converting the cells into whatever target you want them to be.

Additional authors on this paper include Chen Li, Maria C. Virgilio, and Kathleen L. Collins.

Paper cited: Single-cell multi-omic velocity infers dynamic and decoupled gene regulation, Nature Biotechnology. DOI: 10.1038/s41587-022-01476-y

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Mathematical model could bring us closer to effective stem cell therapies - Michigan Medicine

As a top regulator at the Food and Drug Administration, Peter Marks isnt responsible for weighing the cost of the treatments his teams review. But he is worried that some of the drug industrys most promising medicines may not reach patients with uncommon diseases if companies cant figure out how to sell them.

There are an estimated 7,000 rare diseases, many of which affect only small groups of people. Genetic medicines, including RNA-based drugs and gene replacement therapies, could offer a powerful way to treat, and potentially even cure, some of them. But for would-be developers, diseases affecting only a few dozen people might not represent a large enough market to justify the cost of developing and selling a new treatment.

We're not going to find enough philanthropic groups to foot the bill for gene therapies for the hundreds upon hundreds of different diseases that need to be addressed, said Marks, head of the FDAs Center for Biologics Evaluation and Research, at a conference hosted by the Alliance for Regenerative Medicine on Wednesday.

We're gonna have to find a way to make this commercially viable so that industry can find a way forward towards this."

According to Marks, commercial viability for a gene therapy means administering roughly 100 to 200 treatments a year, a threshold that could be difficult to clear in a single country for rare conditions like severe combined immunodeficiences or adrenoleukodystrophies.

It has not escaped our attention at FDA that there have been some clouds on the horizon in gene therapy, said Marks, noting instances when gene therapies were taken off the market or returned by their developers to the original academic researchers.

In Europe, for example, first GSK and then Orchard Therapeutics abandoned one of the first gene therapies approved there, a treatment called Strimvelis for a condition known as ADA-SCID. Only a few dozen patients were ever treated, and Orchard has also handed back rights to a successor treatment. More recently, Bluebird bio withdrew two gene therapiesfrom the EU market after running into difficulties securing reimbursement in several European countries.

Bluebird recently won FDA approval for both of those therapies in the U.S. One, to be sold as Skysona at a cost of $3 million, is for an inherited condition known as CALD that affects about 50 boys each year. Bluebird has said it expects to treat around 10 each year.

In his remarks to the conference, known as the Meeting on the Mesa and attended by many in the cell and gene therapy field, Marks highlighted a few areas where the FDA could help ease hurdles for ultra-rare disease treatments.

The agency is currently putting together a cookbook for developing and manufacturing of bespoke gene therapies, which could help academic groups more easily transfer treatments theyre working on to industry. Its also looking into how to use non-clinical and manufacturing data from one application to speed the review of others that share similar technology.

There are certain pieces of gene therapies that are not like your typical small molecule drug, because they're reused repeatedly, Marks said.

Automated manufacturing could be another solution to help lower the costs of production, which are significantly higher for cell and gene therapies than for other more established drug types.

The FDA is also hoping to get on the same page with other regulators so that developers could be more confident a product they gain approval for in one country would have a good chance of success in others.

Some of [these problems] may relate to how we can make gene therapies for small populations more widely available, Marks said. What may be a tiny population in the U.S. becomes a reasonable sized population when you go globally.

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'We have to find a way': FDA seeks solutions to aid bespoke gene therapy - BioPharma Dive

Free virtual conference for World Cord Blood Day 2022 (November 15th) to feature renowned transplant doctors and researchers presenting ground-breaking work in cellular therapy using cord blood to treat cancer, HIV, cerebral palsy and autism. In addition, bestselling author, Delia Ephron, to present her memoir about fighting leukemia, "Left on 10th: A Second Chance at Life."

TUCSON, Ariz., Oct. 13, 2022 /PRNewswire/ -- Innovations using cord blood in cellular therapy, including traditional stem cell transplants and regenerative medicine research, will be featured during World Cord Blood Day (WCBD) 2022. Recommended for healthcare professionals, expectant parents and STEM students, the official virtual conference, hosted by Save the Cord Foundation, will be held on November 15th (register free via Eventbrite).

We are proud to announce the following speakers:

View the full agenda here: https://www.worldcordbloodday.org/online-medical-conference-agenda-wcbd-2022.html

Hosted and organized by Save the Cord Foundation, a 501c3 non-profit, World Cord Blood Day 2022 brings together the cord blood community through numerous live events and activities held around the globe and online. QuickSTAT Global Life Science Logistics, recognized leader in medical shipping and healthcare logistics, is the Official Sponsor. Inspiring Partners include Be the Match (NMDP), World Marrow Donor Association (WMDA-Netcord), Association for the Advancement of Blood and Biotherapies (AABB), Cord Blood Association (CBA), and Foundation for the Accreditation of Cellular Therapy (FACT).

Visit http://www.WorldCordBloodDay.org to register free for the online conference and learn how you can participate on-line or at an event locally in your community (#WCBD22 #WorldCordBloodDay).

About Save the Cord Foundation (Organizer and host of WCBD 2022)

Save the Cord Foundation (a 501c3 non-profit) was established to advance cord blood education. The Foundation provides non-commercial information to parents, health professionals and the public regarding methods for saving cord blood, as well as current applications using cord blood and the latest research. Learn more at http://www.SaveTheCordFoundation.org.

About QuickSTAT Global Life Science Logistics (Official Sponsor of WCBD 2022)

Every day, QuickSTAT, a part of Kuehne+Nagel, safely and reliably moves thousands of critical shipments around the world. For over forty years, QuickSTAT has been entrusted with transporting human organs and tissue for transplant or research, blood, blood products, cord blood, bone marrow, medical devices, and personalized medicine, 24/7/365. QuickSTAT's specially trained experts work with hospitals, laboratories, blood banks and medical processing centers, and utilize the safest routes to ensure integrity, temperature control and chain of custody throughout the transportation process. Learn more at http://www.quickstat.aero.

Media Contact:

Charis Ober

(520) 419-0269

[emailprotected]

SOURCE Save the Cord Foundation

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Discover Medical Advances in Cellular Therapy Research Using Cord Blood for Cancer, HIV, Cerebral Palsy and Autism During World Cord Blood Day 2022 -...

MIAMI, Oct. 11, 2022 (GLOBE NEWSWIRE) -- The American Academy of Stem Cell Physicians will be hosting its fall Scientific Congress in Chicago, IL, on Oct. 28-30, 2022. The conference will feature three days of educational and networking events with leading physicians from across the fields of stem cells, live cells, and regenerative medicine. A Board Examination process will be available, creating a pathway for participants to earn a Diplomat and Fellowship Certification in Regenerative Medicine.

The Board of American Academy of Stem Cell Physicians is the official board certifying body of the American Academy of Stem Cell Physicians(AASCP). As a nationally recognized academy with a mission to bring like-minded physicians together to increase awareness and education for the evolving field of regenerative medicine, the AASCP is proud to announce its Fellowship and Diplomat Certification.

In order to be eligible for certification or recertification through the AASCP, licensed physicians in good standing must meet the stringent eligibility requirements that have been defined by the board. AASCP places an emphasis on not only psychometrically evaluated testing and advanced training, but also moral character and experience. Furthermore, AASCP has a clear path toward recertification for qualified physicians. Their standards for recertification include a commitment to continuing medical education, successful completion of a recertification examination, participation in a non-remedial medical ethics program, and additional requirements.

AASCP is known for working with physicians to provide unique opportunities for board certification in their specialty of regenerative medicine. Specifically, the AASCP offers ongoing workshop modules led by esteemed physicians in this field who certify and educate on different treatment approaches and techniques. Another defining characteristic of the AASCP is theircommitment to ongoing education and awareness. To support this goal, the AASCP has developed innovative committees, including its Institutional Review Board and created opportunities for physicians and researchers to submit their work for peer review and exposure.

The AASCP was founded to recognize licensed physicians who have shown a specialty and interest in regenerative medicine. Increasingly, hospitals and medical staff placement agencies are prioritizing hiring Board-Certified Physicians. For this reason, the AASCPfeels it is important to offer qualified professionals a choice when they're researching board certifying bodies.

The American Academy of Stem Cell Physicians (AASCP) is an organization created to advance research and the development of therapeutics in regenerative medicine, including diagnosis, treatmentand prevention of disease related to or occurring within the human body. Secondarily, the AASCP aims to serve as an educational resource for physicians, scientistsand the public in diseases that can be caused by physiological dysfunction that areameliorableto medical treatment.

For further information, please contact WilsonDemenessez at 305-891-4686, and you can also visit us at http://www.aascp.net.

Contact Information: Wislon Demenessezz AASCP account Sales manager wilson@genorthix.com 305-891-4686

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American Academy of Stem Cell Physicians to Offer Licensed Physicians Board Examination in Regenerative Medicine - GlobeNewswire

As activity in this space grows, so do the hurdles in moving these products forward.

Cell and gene therapy (CGT)its risks and promisesare succinctly summarized in this description of clinical trial number NCT01129544, a Phase I/II study in children born with X-linked severe combined immunodeficiency (SCID-X1), an inherited, rare, and life-threatening disease. The eight-person trial, which began in May 2010, continues today. The following paragraph has been edited.1

Gene transfer is still research for two reasons. One, not enough children have been studied to tell if the procedure is consistently successful. [And] we are still learning about its side effects and doing gene transfer safely. In previous trials, five children developed gene transfer-related leukemia; four are in remission; one died.

If the above information has stifled the research communitys scientific curiosity about CGT, it is not evident. Evidence from numerous sourcesClinicalTrials.gov, the Alliance for Regenerative Medicine (ARM), FDAare chock-a-block with studies, trials, and figures showing these therapies popularity. In the second quarter of 2022, 3,633 such treatments were in development, up from 1,745 in May 2021. The vast majority are in the preclinical stage.2,3

Some sources are revealing more.

Most indicate that academics now have a remarkable presence in the CGT development space, including sponsorship. Last year, for the first time, ARM included sponsorship figures in its twice-annual industry report.4 Academic- and government-sponsored trials far exceeded industry for sponsored trials in CGT. Stephen Majors, senior director for public affairs, ARM, says the alliance knew of academias presence for the past few years, but only was able to get data this year from its partner, Global Data.

Less reliable, but still noteworthy, are data from ClinicalTrials.gov: for active Phase I trials, industry has 89; others, which covers academia and government, have 50. Industry enrollment for Phase I is 172; others, 116.Phase III is one for others, eight for industry.

A little disruption in pharmas corner of the world? It seems that way. While basic bench to preclinical to clinical trial has long been the traditional route to FDA approvaland no one interviewed for this article suggested a reroutewhat it does imply is that pharma members have some competition from the spin-offs and academic biotechs that historically they have absorbed.

There are suspected trends that we are watching, says Majors.As to whether academias presence in this spot can be called a trend depends on ones definition of what a trend is. The Centers for Disease Control and Prevention (CDC) considers changes over a number years to determine a trend; financial investment firms typically evaluate over a two-year period.Considering that CGT companies raised $23.1 billion in 2021, 16% more than 2020,3 the answer to the above question could be, maybe.

The CGT space is still immature, according to Mike Rea, founder of Protodigm, a self-described exploratory research organization that partners with biopharma clients on alternative development and commercial solutions. Physicians need time to be comfortable with these therapies, notes Rea, so they may not be used on a regular basis.

For example, physicians have to understand how to deliver the gene, agrees cardiologist Arthur M. Feldman, MD, PhD, whose lab worked on a heart failure-related mutation in BAG3 for decades.

Last month, the company he founded, Renovacor, agreed to be acquired by Rocket Pharmaceuticals.5 We are asking physicians to do something they never did before and to understand a very different set of information, including risk/benefit discussions that they didnt learn about in medical school, he says. Feldman is a Laura H. Carnell Professor of Medicine, Division of Cardiology, and a member of the Center for Neurovirology and Gene Editing at the Lewis Katz School of Medicine at Temple University.

Chris Learn, Parexels vice president of cell and gene therapy, is unequivocal regarding academias increased presence in the drug development space focused around these treatments. He cites MD Anderson and Moffitt Cancer Center as two institutions that are sponsoring their own trials. The lines are really blurring here, he tells Applied Clinical Trials. It is indisputable.

The following is a look at how academia is showing up in various reports.

In its 2022 report4, ARM separated sponsorship, type of therapygene, cell-based, and tissue engineeringand trial phase. What these data show are industry far exceeding academic and government sponsored trials for gene therapy, while for cell therapy alone, the reverse is true: 656 cell therapy trials for academic and government, and 424 for industry. For gene therapy, there are 84 for the academics and government, and 222 for industry. In a later report, ARM found non-industry trials dropped.

Pharma Intelligences Pharma R&D Annual Review does not break down trials by their sponsors. It does, however, break down whats in the pipeline in various categories, including by the number of therapies per company, and by disease type.6 In numbers captured prior to March 2020, the analysis reported 1,849 companies with asingle drug in its pipeline, up from 1,633 in 2019, comprising more than half of all drug companies. As for types of therapies, gene therapy was in third place, the same spot it occupied in 2019. (Cancer-related therapies occupy the top spots.) Overall, biotech therapies in the pipeline increased by 13.2% in 2020 over 20196,135 vs. 5,422. Cellular therapy, the field in which academia is dominating, rose to 14th place, up from 33.

In 1982, Feldman was a resident in the cardiac care unit at the Johns Hopkins Hospital in Baltimore when he took care of a 22-year-old woman, a native Pennsylvanian, who was dying of heart failure. Sadly, we didnt have drugs with which to treat her, he recalls. Feldmans involvement with the case and the womans family led to his career as a cardiologist, he says. Twenty years later in Philadelphia, he was asked to see a heart-failure patient in consult, who turned out to be the aunt of the younger woman. It would take almost another 10 years until the technology became available to identify the genomic anomaly in this family. Here, a genetic variant that is produced by one of two alleles causes the protein product to be unstable. The result: the cell removes it, so the person with the variant has just half the amount of required protein.

BAG3 is an interesting protein that is found in the heart, the skeletal muscles, and the nervous system, including the brain. Its function is to help remove degraded and misfolded proteins, stop apoptosis or programmed cell death, and maintain the structure of the skeletal muscles. A missing allele isnt the only genetic cause for heart failure, Feldman said. Other patients, while having the correct amount of DNA, have a point mutationa single amino acidin half of the produced DNA. That single letter is the wrong amino acid in the specific site in the protein.

Around this time, Kamel Khalili, PhD, Laura H. Carnell Professor, and chair of the department of microbiology, immunology, and inflammation; director of the Center for Neurovirology and Gene Editing; and director of the Comprehensive NeuroAIDS Center, Lewis Katz School of Medicine, Temple University, had created a method by which he could excise the HIV virus from patients using the new technique of CRISPR-Cas9.

Khalili believes that BAG3 may be involved in the pathogenesis of HIV-1 in brain diseases and protein quality control caused by viral infection as well as several other disorders, including Alzheimers disease and dementia. BAG3 changes the homeostasis of the cell, he says. The only solution is to fix the cell. Khalili has used CRISPR technology to excise the viral genome in both small and large investigational animals and has recently started a Phase I trial to test the safety of the new gene-editing treatment. Khalili, too, started a company, but Temple holds the license. In the case of Renovacor, it was granted the license by Temple.

As a scientist, when you are doing something in biomed research, [the] goal is to translate bench work to the clinic for [the] wellness of people. We are doing long hours and long days because we want to help. We are trying to see if discovery can help people, says Khalili. I know my limit, I stop at business aspects. My interest is to discover research which can help populations.

Was Feldman happy with his business experience? As a company gets bigger, others join the team who fulfill other roles, like acquiring funding or developing the actual product, he says. Releasing the control reins are difficult. But if it speeds up the timeline to get an approved product into the clinic, then its all worth it, he adds.

Researchers such as Feldman and Khalili, says Kaspar Mossman, PhD, director of communications and marketing at QB3, a University of California biotech accelerator, are normally not deeply interested in business. He notes the new flagship space in UC Berkeley called Bakar Lab. So far, it has 25 companies, one-third from university labs. They collaborate, they share equipment, [at times] they merge, Mossman tells Applied Clinical Trials.

And, he adds, Academics tend to be very smart individuals. The more time they spend in business, they learn stuff and become serial founders, says Mossman. They are honest about not wanting to be a CEO.

In terms of business, the academics employers are also pretty smart. The huge bugaboo with CGT commercialization is the manufacturing processthe need for an apheresis unit, ultra-cold storage, and regulated cell processing facilities.

Some institutions are building their own manufacturing facilities to more easily meet the increasingly complicated standards pertaining to regenerative medicine production. Harvard, MD Anderson, Moffitt, the University of Pennsylvania, and the University Hospital of Liege in Belgium8 all have or are planning to build their own facilities.

As for how academias presence impacts the traditional pharma space, those interviewed cited pros and cons. More research is better, more companies vying for venture capital funding is not. But more trials mean more competition among similar therapies, which, says Majors, is a good thing.

We need experimentation, adds Rea. If left to pharma, he says, the research wouldnt happen. Smaller biotechs are taking the risk. Over the last 10 years, Rea believes pharma has been slow in the risk-taking department. Once upon a time, pharma didnt have many competitors. Now, with many numerous smaller companies with viable assets, willing to accept a smaller net profit, the competition is creating some angst. Pharma cant project everyones movement, says Rea. The gene/cell therapy landscape [for products] is huge.

Likely adding to the angst: Those smaller biotechs are getting financial help. Between April 4, 2021, and June 24, 2021, of 23 start-up financing deals, 19 involved academics.2

Learns viewpoint is different. He says there are too many players out there, and while large pharma may be averse to risk, I really do believe what we are witnessing are simply market forces that have played into this. There is so much cash coming in, he continues, that people can be blinded by the pitfalls. The CGT area, he adds, is bloated and he says the industry needs an overall strategy.

Learn doesnt think that academias presence in the CGT space is a flash in the proverbial pan. The enthusiasm to find cures is real, and some research institutions have the endowments to see the trials through. I think it is just the beginning, says Learn. Academia will put their futures in front of them. Why put all your sweat equity into it and not have any fiduciary benefit of the approved product?

In Pharma Intelligences 2020 Pharma R&D Review, its author questioned the wisdom of so many drugs, overall, in the pipeline4,001 added in 2018 and 4,730 added in 2019, for a total of 17,737 drug candidates. [A]re the industrys eyes getting too big for its belly? Unless it can continue to provide [approved therapies] then a certain degree of control in the pipeline might be advisable, the report stated.6

And now to costs. While no one doubts these cures change lives, the question of access persists. FDAs approval of Bluebird Bios second therapy this year, branded as Skysona, for early but active cerebral adrenoleukodystrophy, is expected to cost $3 million. Learn doubts that payers are jumping up and down to get Skysona on their formularies.

Its still a fairly dicey business proposition for companies to invest in this field, Steven Pearson, MD, president of the Institute for Clinical and Economic Review (ICER), said recently.8Theres still a risk that next-generation therapies will not flourish even in developed countries health systems, he added.

One positive development in the US, however, occurred late last month when Congress reauthorized the Prescription Drug User Fee Act (PDUFA) for the next five years, 2023-2027. The action maintained FDAs authority to collect fees from manufacturers and keep and recruit agency staff to review the increased number of CGT applications. Majors says most of FDAs review of CGT products involves scalability and consistent reproducibility in the manufacturing process, which, of course, means traveling.

According to a Senate press release9, FDA is seeking to hire at least 320 new staff members. In a statement, Pharmaceutical Research and Manufacturers of America (PhRMA) said a modern regulatory framework supported by PDUFA helps ensure patients have timely access to lifesaving medicines.

PDUFA reauthorization aside, there is little argument that the field of CGT, from research and drug discovery through commercialization, is advancing rapidly. In turn, so are the unique operational and manufacturing challenges that these therapies present. This reality may thin the currently crowded playing field in CGT going forward, with those sponsors and partners best prepared to deliver on the numerous touchpoints required separating from the pack.

Christine Bahls, Freelance Writer for Medical, Clinical Trials, and Pharma Information

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The Risk-Reward Proposition for CGT Clinical Trials - Applied Clinical Trials Online