A young Phumelela in Grade 1

Supplied: Phumelela Nkomozake

"My journey started, as [far back] as I could remember, being a young child growing up and always noticing that there is something particular about you or that you really do not find yourself comfortable in your own body.It is feeling in a particular way that there is a disconnect between your mind and your body."

These are the words of 24-year-old Phumelela Nkomazake, a transgender woman from the Eastern Cape whose personal journey towards affirming her gender led her to discover that the journey was more than just about her, and that it was one that the transgender community experiences as a whole.

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There is a disconnect between the mind and body - A transgender woman's journey exposes her to the difficulties experienced by her community - News24

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IVF clinics vied for patients in this highly competitive environment knowing women considering freezing their eggs scoured clinic websites and social media for guidance, Dr Beilby said.

"There's a lot of money involved in fertility preserving technology, and it's great to know this technology exists to meet the demand," she said.

But clinics had an obligation to provide clear, accurate information alongside their marketing material about what egg freezing entails, the chance of success, and the cost. "That's what we're not seeing," Dr Beilby said.

None of Australia's 21 most prominent fertility clinic websites specified whether the egg-freezing success rates they quoted were based on patients using their own eggs or donated eggs from cohorts of 20-something women.

Only half provided success rates based on a woman's age and fewer than one-third included the cost of the procedure, according to the analysis published in the Australian and New Zealand Journal of Obstetrics and Gynaecology.

One in three clinic websites published a percentage figure for the chance of getting pregnant after egg-freezing, but only one in 10 published the chance of having a baby.

Overall, the researchers rated 43 per cent of the clinics as "poor" and 57 per cent as "moderate" on an American matrix designed specifically to assess the quality of information on egg freezing.

Co-author Dr Karin Hammarberg said women need to be properly informed of these high costs, potential health risks and chances of having a baby before they decide to undergo egg freezing.

The chance of having a baby from frozen eggs depends on two numbers: the age of a woman when her eggs are frozen, and the number of eggs she freezes, Dr Hammarberg said.

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The quality of a woman's eggs declines the older she gets. The older the woman, the more eggs she'll need to freeze to have a reasonable chance of having a baby.

For a women under 30, the chance of taking home a baby per initiated IVF cycle if she uses her own eggs is 19.7 per cent, the latest official IVF data from the Australian and New Zealand Assisted Reproduction Database shows.

FFor women aged 30 to 34, it is 17.5per cent, dropping to 12.1 per cent for 35- to 39-year-olds, 4.6 per cent for 40- to 44-year-olds and 0.4 per cent for women 45 and over.

Freezing 10 eggs at 35 or younger gives a woman a 69 per cent chance of having a live birth, dropping to 50 per cent for 37-year-olds and 30 per cent for 40-year-olds.

The number of stimulation cycles needed to retrieve enough eggs increases with age. Most women over 35 will need more than one egg retrieval to collect enough eggs to have a reasonable chance of success.

More than half of the women who freeze their eggs when they are in their mid to late 30s will need at least two hormone stimulations to get the number of eggs that gives them an 80 percent chance of having a baby when they use their eggs. Almost all women aged 40 or more will need at least four hormone stimulations for the same chance.

There is also a third number to consider: cost. The egg-freezing process can cost $7000 to $10,000 per egg retrieval plus the additional cost of storing the eggs (approximately $200 to $500 a year).

"Something that may not be obvious is that in order to have a good reasonable chance of having a baby you might need to spend $30,000," Dr Beilby said.

The egg retrieval process also involves health risks, such as ovarian hyper-stimulation syndrome (OHSS), which is associated with the hormone injections required as part of the procedure.

Dr Beilby hoped clinics would use the audit to develop a checklist of information to include on their websites, and where possible publish clinic-specific success rates.

"There is a lot of hope tied up in this, and we need to err on the side of caution to align women's expectations with reality," Dr Beilby said.

Professor Luk Rombauts, President of the Fertility Society of Australia, said a consultation with a fertility specialist - not the internet - was the appropriate setting for a woman to be given the information she needed to make an informed decision about egg freezing.

"Just because it's not on the website doesn't mean patients won't get that information," Professor Rombauts said. "They will get it from their doctor, which is exactly where the information should be coming from. Most importantly, we will take into account a patient's personal circumstances."

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Professor Rombauts said the fertility industry had done exceptionally well when it came to transparency in advertising compared to other medical specialities.

It was impossible - particularly for smaller clinics - to provide meaningful success rates when only a very small proportion of women have returned to use their frozen eggs.

"A lot of women who freeze their eggs fortunately never have to use them because they do find a partner," he said. "That doesnt mean we couldn't improve the reporting on our websites."

In Australia, the FSA's Reproductive Technology Accreditation Committee sets standards for ART clinics through an audited Code of Practice, which states their websites "must include but not be limited to: processes, costs, risks and outcomes" but does not specify a minimum level of detail.

"This paper could give [clinics] another useful tool when they're considering what information they could include on their websites," Professor Rombauts said.

Kate Aubusson is Health Editor of The Sydney Morning Herald.

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Should you freeze your eggs? IVF websites don't crack the surface - Sydney Morning Herald

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At menopause as hormone levels decline, women start noticing signs of dryness or tightness and sex may become uncomfortable

There are several reasons for this, and a number of conditions that can arise at this time may need to be investigated.

Dealing with dryness

This is most common at menopause and related to the declining oestrogen levels that occur at this time.

This is not serious physically, though can certainly be distressing, and there are a number of lubricants available.

If you have mild to moderate vaginitis, using a lubricant can help relieve pain and discomfort while having sex but they provide short-term symptom relief and can improve vaginal dryness during sex, but theres no evidence to show theyre an effective long-term treatment.

Several types of lubricant are available some are water-based and some aresilicone-based. You may need to try a few before finding one thats suitable or your GP may wish to prescribe a localoestrogen cream, gel or pessary.

If you prefer a natural alternative then 20-1, which is a combination cream with the majority ingredient of progesterone with two natural oestrogens added, can help particularly if internal vaginal application is used.

Vaginitis

This is inflammation of the vagina that can cause itching, discomfort and discharge. Itcan be caused by any of the following infections or irritants:

thrush a common yeast infection that affects most women at some point

bacterial vaginosis a bacterial infection where the balance of bacteria inside the vagina is disrupted

trichomoniasis an STI (Sexually Transmitted Infection) caused by a tiny parasite

chemical irritation for example, from perfumed soap, bubble bath, or fabric conditioner, or from spermicide and somesanitary products

washing inside your vagina

chlamydia an STI caused by bacteria

gonorrhoea an STI caused by bacteria

genital herpes an STI caused by the herpes simplex virus.and symptoms include:

Symptoms can include the following

an abnormal vaginal discharge

vaginal irritation or itching

pain when peeing or having sex

light bleeding or spotting

a strong, unpleasant smell, particularly after sex, can be a sign of bacterial vaginosisor trichomoniasis, which can sometimes cause vaginitis.

Time to see your GP orgo to a sexual health clinicif you have any unusual vaginal symptoms, particularly if you have :

vaginal itching or an unpleasant smelling vaginal discharge

havent had a vaginal infection before

had vaginal infections before but now your symptoms are different

had a number of sexual partners, or you have a new sexual partner so you may have a sexually transmitted infection (STI)

finished a course of medication for vaginal thrush, but your symptoms are persisting

Theres no need to see your GP if youve been diagnosed with thrush in the past and your symptoms arethe same.If youre sure you have thrush and youve treated it successfully in the past with over-the-counter medication, you can treat it yourself again.

Treating vaginitis

Treatment for vaginitis depends on whats causing it.Yeast infections, such as vaginal thrush, are usually treated with antifungal medicines, and bacterial infections are usually treated with antibiotics.

Vaginal atrophy

This refers to thethinning of the lining of the vagina after the menopause and the resultingdryness, itching or discomfort (particularly during sex). It can also sometimes be caused by a decrease in oestrogen levels after the menopause.

Your GP may recommend using a local oestrogen and that is usually necessary with atrophy until the condition is more stable.

Unlike combined or other forms of HRT, thisonly restores oestrogen to your vagina rather than to your whole body, reducing the risk of side effects.

You can balance this additional oestrogen with bioidentical progesterone, and once the atrophy is under control you can use a combined bioidentical progesterone and oestrogen cream.

How to help yourself

There are number of self-help measures which can reduce the risk of these problems and help once they are diagnosed.

keep your genital area clean and drytake a warm bath rather than a hot one and useunperfumed soap to clean your genital area anddry yourself thoroughly

avoid douching (spraying water inside your vagina) it may make your vaginitis symptoms worse by removing the healthy bacteria that line the vagina and help keep it free from infection

do not use feminine hygiene products such as sprays, deodorants or powders

use pads rather than tampons if youre using intravaginal creams or pessaries to treat an infectiontampons may soak up the treatment meaning theres less available in the vagina

wear loose-fitting cotton underwearthis may be beneficial if you have external soreness, but it wont prevent you getting vaginitis in the future

Helpfulinformation:

Because women are often reluctant to talk about conditions like this, they do often suffer in silence but there really is no need as help is readily available.

Generally, maintaining good hormone balance with healthy levels of progesterone and oestrogen is a helpful measure too.

For any of these condition it is best topractice safe sex by usingcondoms to avoid getting or spreading sexually transmitted infections.

What Are the Symptoms of UTIs?

The rest is here:
Do You Have Dryness, Vaginal Atrophy or Vaginitis? - Bioidentical Hormone Health

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Oriaku Njoku was outside the U.S. Supreme Court alongside a crowd of activists and advocates for abortion rights as the nine jurists inside heard oral arguments in a case that, depending on its outcome, could destroy access to abortion in Louisiana.

It was a crisp morning in early March, mere days before the coronavirus pandemic would see the country all but completely locked down. Njoku, one of the founders of Access Reproductive Care-Southeast, a nonprofit that provides assistance to individuals seeking abortion care across six states in the Deep South, was rallying outside the court with her sister. There was so much energy, she recalled.

It was nearly four years to the day since the last time Njoku had been in front of the high court. That morning, in early 2016, the court was considering the constitutionality of a set of abortion restrictions in Texas that had shuttered half the states clinics. At the time, there were just eight justices on the bench; Antonin Scalia had died several weeks earlier. In the end, Anthony Kennedy joined the four more liberal justices, including Ruth Bader Ginsburg, to strike down the restrictions, which included a requirement that abortion providers have admitting privileges at a local hospital. The court found no evidence that this was necessary to ensure patient safety.

Now, Njoku was back in the same space rallying for the same cause: The restriction at issue in the Louisiana case was identical to the admitting privileges requirement the court had invalidated in Texas. It was a full-circle moment, where it was almost four years to the day; Im back here again, literally fighting for the same thing, she said. I was like, They have to uphold this precedent.

By then, Kennedy had left the bench and President Donald Trump had installed two new conservative justices, Neil Gorsuch and Brett Kavanaugh, who were appointed precisely because of their hostility to abortion. In the end, the court did strike down the Louisiana law, with Chief Justice John Roberts casting the deciding vote albeit with an opinion suggesting the court would be open to upholding additional restrictions in the future.

When the decision was announced, Njoku realized that it wasnt exactly a game-changing victory. Anti-abortion lawmakers have passed more than 450 abortion restrictions over the last decade, many of which still stand, making access to abortion difficult, if not nearly impossible, for millions of people. This is especially true for people of color, LGBTQ people, poorand low-income people, and people in rural areas, who are routinely hit hardest by restrictions on reproductive care, as well as broader inequalities within the health care system. These inequalities have been widely exposed not only by the pandemic, but also through a summer of civil rights protests that have thrown new light on the countrys continuing legacy of racial oppression.

Judge Amy Coney Barrett, President Donald Trumps nominee to the Supreme Court, at the U.S. Capitol on Oct. 1, 2020, in Washington, D.C.

Photo: Caroline Brehman- Pool/Getty Images

And with Ginsburgs death in September, the fight for reproductive justice faces new threats as Republicans scramble to fill her seat with appeals court jurist Amy Coney Barrett, who in 2006 signed on to a call for Roe v. Wade to be overturned, which described its legacy as barbaric. According to the Center for Reproductive Rights, if that were to happen, 24 states would prohibit abortion altogether.

If Barrett is seated, not only is the future of legal abortion in jeopardy, but also access to the larger health care system; the Supreme Court is slated to hear yet another challenge to the Affordable Care Act on November 10. Yes, this is a huge blow, but it doesnt stop the fact that we still have to do work on the ground, as grassroots organizations, as people who are directly impacted by whatever this administration has done or that prior administrations have done, Njoku said. We have to continue fighting until we get to that future of reproductive justice where were able to access whatever health care that we need without any bias or barrier.

WhileGinsburgs seat is still vacant, the Trump administration has already offered the eight-member court an opportunity to restrict abortion access amid the pandemic.

At issue is how medication abortion is provided to people seeking to terminate a pregnancy in its earliest stages. Available through 10 weeks gestation, medication abortion is a two-drug regimen that has been available in the U.S. for 20 years and used by more than 4 million people. Medication abortion accounts for 60 percent of all early terminations and nearly 40 percent of all abortions, according to the Guttmacher Institute.

It works like this: Patients first take mifepristone, a drug that blocks progesterone, a hormone needed to maintain pregnancy; 24 to 48 hours later, the patient takes a second drug, misoprostol, which creates contractions in the uterus that expel its contents. According to the U.S. Food and Drug Administration, medication abortion is very safe and serious complications are extremely rare. Importantly, medication abortion allows patients the ability to terminate a pregnancy in the privacy of their own home or wherever they choose to be.

Nonetheless, access to medication abortion has been restricted in ways that are medically unnecessary. The needless barriers amid a pandemic place individuals at greater risk of contracting Covid-19, according to a federal lawsuit the American Civil Liberties Union filed against the Trump administration in May on behalf of several doctors organizations and a leading reproductive justice group.

Even as state and federal health officials have implored people to avail themselves of telemedicine, abortion patients have been singled out for disparate treatment.

Medication abortion is constrained by what the FDA calls a Risk Evaluation and Mitigation Strategy, a drug safety program designed to control the administration of certain medications with serious safety concerns to help ensure the benefits of the medication outweigh its risks. Where medication abortion is concerned, the REMS has several elements that are onerous in normal times and two that are particularly problematic amid a pandemic: that patients must be handed mifepristone by a medical provider in a clinical setting and that the clinician must collect a physical signature from the patient at the time the drug is dispensed.

While in-person dispensing is required, patients are still in charge of administering the protocol themselves at a time and place of their choosing. And any complications that may arise such as excessive bleeding wouldnt occur until roughly two days later, after the patient ingests the second drug, misoprostol, which is not subject to the REMS. Of the 20,000 drugs regulated by the FDA, mifepristone is the only one that patients must receive in person at a hospital, clinic, or medical office, yet may self-administer unsupervised, reads the lawsuit.

So, even as state and federal health officials have implored people to avail themselves of telemedicine as much as possible, abortion patients have been singled out for disparate treatment, said Julia Kaye, a staff attorney with the ACLUs Reproductive Freedom Project. When youre forcing patients to make unnecessary, in-person trips to a health center during a pandemic, that imposes Covid-19 risks, and theres simply no justification for it, she said, particularly where the federal government has taken extraordinary action to suspend other kinds of in-person requirements. The government has even suspended in-person requirements for controlled substances, including opioids. And I think its important to note that the Covid-19 risks here are greatest for communities of color who make up a majority of impacted abortion patients and who are suffering severe illness and death from Covid-19 at vastly disproportionate rates, Kaye added.

Indeed, back in April, as ARC-Southeast was working to help patients secure abortion access during the worsening pandemic, Njoku recalls reading about the staggering impact the virus was having on Black people; in Georgia alone, they accounted for 80 percent of those hospitalized with Covid-19. I was like, yall, this is the great unraveling thats happening in our society, she said.

In July, a district judge in Maryland agreed with the medical experts and issued a preliminary nationwide injunction blocking enforcement of the in-person requirements during the pendency of the case. The Trump administration balked and asked the 4th U.S. Circuit Court of Appeals to lift the stay; that request was denied.

But instead of allowing the case to play out, the Trump administration asked the Supreme Court to intervene. Given that surgical methods of abortion remain widely available, the enforcement of longstanding safety requirements for a medication abortion does not constitute a substantial obstacle to abortion access, the administrations petition reads, even if the Covid-19 pandemic has made obtaining any method of abortion in person somewhat riskier.

Attempts to truncate the normal course of litigation by running directly to the Supreme Court and forcing cases onto the so-called shadow docket has become a hallmark of the Trump administration, even though it is a move that is supposed to be reserved for extraordinary circumstances. In deciding these cases, the court often issues an order without the full facts before it, without hearing arguments, and without issuing opinions. It typically offers no explanation for its reasoning, even when dissenting justices voice serious objections, and even when the court is effectively overturning the unanimous decisions of lower courts, David Cole, the ACLUs national legal director, wrote in the Washington Post. In the case of the medication abortion challenge, the court could effectively ensure a higher risk of viral transmission with the stroke of a pen.

After pending on the docket for about six weeks, the Supreme Court finally weighed in on October 8, allowing the stay to remain in effect at least for now. In a short order, the court sent the question of whether the stay should be lifted back to the Maryland court for further consideration, essentially postponing any action until after the November election. Justice Samuel Alito, joined by Clarence Thomas, penned a dissent saying that the court should have granted the administrations request.

Had the court granted that request, Kaye said, it would have sent a chilling signal about what lies ahead with a post-Ginsburg court. And though the courts decision to stay out of this particular abortion fight at this particular moment is certainly a win for patients in the short term, she added, its hardly an indication that the right to abortion is secure.

As the pandemic raged this spring, Dr. Honor MacNaughton worried about her patients. MacNaughton, a plaintiff in the ACLU case, is a family physician working in a safety-net hospital system in the Boston area. Her patients are mostly lower-income and people of color, groups disproportionately impacted by the virus. As our health care system was closing down to in-person visits, and we were trying to do everything we could to limit exposure to the virus, it seemed so wrong to require people to still come, in person, to pick up a medication, she said.

Shed long known that the REMS was problematic and that two decades of data demonstrated the safety of mifepristone for pregnancy termination and miscarriage management. I think from a medical standpoint, theres enough evidence for us to feel really confident that the in-person visit isnt required, she said. Since the Maryland court issued its injunction, providers across the country have, for the first time, been able to provide the medication without that constraint. Being able to provide this care since the injunction has been a ray of hope, MacNaughton said. Its given me a glimpse into what more equitable or stigma-free care could look like.

Kirsten Moore, head of Expanding Medication Abortion Access, which was created in part to push for the REMS to be rescinded, has long known that medication abortion is burdened by unnecessary restrictions. She was formerly head of the Reproductive Health Technologies Project, which was founded in 1988 to help bring medication abortion to the U.S. and later successfully pushed for emergency contraception, also known as the morning-after pill, to be made available over the counter.

When medication abortion was approved by the FDA in 2000, Moore said that advocates assumed the restrictions would be revisited as safety data developed. That has not happened. Were still jumping through the same hoops today that we had to when it was first approved, she said. That doesnt make sense, right?

Yes, Roe v. Wade is a great gift. It made abortion legal, but the reality is that it did not make it accessible.

For both Moore and MacNaughton, the disconnect between science and public policy demonstrates that the REMS is about something else altogether a solution in search of a problem.

Of course, the same could be said about most restrictions on abortion access. Lawmakers often peddle these restrictions, like the hospital admitting privileges requirement, as a way to ensure patient safety, even though the alleged benefits fail to materialize. Forty-four states have passed one or more restrictions on abortion access. Twenty-six states mandate delays for patients seeking care; in South Dakota, the 72-hour waiting period doesnt include weekends or state holidays, so a patient could end up having to wait a week before being seen. Thirty states mandate pre-abortion counseling and many incorporate junk science, including, for example, that abortion leads to a greater risk of breast cancer. Several states require providers to counsel patients that medication abortion can be reversed, even though there is no sound science to back up this claim. Taken together, these restrictions have made abortion all but inaccessible across a wide swath of the country, often forcing women to travel long distances across state lines to receive care.

For those who live in the six states served by ARC-Southeast Alabama, Florida, Georgia, Mississippi, South Carolina, and Tennessee these struggles are common. Every single one of those states experiences some level of reproductive oppression, whether it comes from the legislature or just ideas of shame, stigma, and fear around abortion, Njoku said. She notes that her clients face obstacles to accessing all kinds of basic health care services. None of the states has had any sort of Medicaid expansion; roughly half of Georgias 159 counties are without an OB-GYN. These are all the things that weve always had to navigate, she said. Yes, Roe v. Wade is a great gift. It made abortion legal, but the reality is that it did not make it accessible.

And since Trump took office, lawmakers in states hostile to abortion rights have taken more direct aim at the heart of Roe and its promise of the right to pre-viability abortion.

Women wearing costumes from The Handmaids Tale protest in front of the Alabama State House after the state Senate passed HB314, whichwould ban nearly all abortions, on May 14, 2019, in Montgomery, Ala.

Photo: Elijah Nouvelage for The Washington Post via Getty Images

In 2019 alone, Georgia, Kentucky, Louisiana, Mississippi, and Ohio all banned abortion beginning at six weeks the point at which fetal cardiac activity can be detected and long before many people even know theyre pregnant. Alabama went a step further, passing a law that would ban nearly all abortion. Missouri passed a law to ban abortion at eight weeks, while both Arkansas and Utah have banned abortion at 18 weeks.

Other states have passed bans that interrogate a persons reason for seeking an abortion and would bar access if its based on the sex or race of the fetus or on a diagnosis of Down syndrome or other fetal abnormality. A district court in Missouri has blocked that states anti-discrimination law banning abortion based on a Down syndrome diagnosis, but on September 24, the states solicitor general told a panel of the 8th U.S. Circuit Court of Appeals that the law should be allowed to go into effect in part because individuals with Down syndrome are on the brink of complete elimination.

Since 1973, in Roe v. Wade, the court has given the right to the individual to decide whether or not they want to choose to become a parent at that point in time, said Helene Krasnoff, vice president of public policy litigation and law at Planned Parenthood Federation of America. These laws literally fly in the face of that, she said. They represent another way in which the Supreme Court can really undermine the protections that we have enjoyed, and that Ruth Bader Ginsburg upheld for so many years, without actually outright overruling Roe.

Currently, there are 17 abortion-related challenges to various state restrictions pending in federal appeals courts or before the Supreme Court.

Nothing about the growing storm around reproductive rights is new to Njoku. A lot of folks have been talking about this post-Roe reality, she said. Thats an actual lived reality in states like Texas and Mississippi and really the states we work in in the Southeast. Weve been preparing for things like this because weve been living in this context.

ARC-Southeast has received nearly 16,500 requests for support since they began operations in the summer of 2016. More than 60 percent of clients are already parents. From July 2019 through June 30, ARC-Southeast provided nearly $450,000 in abortion funding and practical support. As the pandemic has continued, jobs have been lost, and politicians across the country have tried to use the public health crisis as a pretext to further block abortion access, Njoku saidthe organization has provided funding not only for abortion, transportation, and lodging, but also for car repairs, food, and even masks. Njoku has seen patients from Texas, Louisiana, Kentucky, and West Virginia come to her region for care, and ARC-Southeast has helped patients to travel as far away as New Mexico, Colorado, and Maryland to get the care they need.

Njoku has been encouraged not only by the donations coming in to support ARC-Southeasts work, but also with a seeming awakening among many that abortion rights have to be viewed within a larger framework that a right without access means nothing, and that people of color are disproportionately impacted by lack of access to basic health care and the racism built into many of the nations social systems. The future that were fighting for is more than just whether or not abortion is legal, she said.

Theres no doubt that the nomination of Barrett presents a threat to that future. According to an analysis by the Center for Reproductive Rights, Barrett has signaled that she believes the Supreme Court should weigh in on the legality of banning abortion based on a patients reason for seeking it, and has suggested that abortion restrictions should be allowed to take effect while questions about their legality are sorted out in court. In Texas, the restrictions ultimately struck down by the Supreme Court were initially allowed to take effect, leading to widespread clinic closures; many never reopened.

And while the Affordable Care Act for the first time created gender parity in health coverage and opened access to maternity care and no-cost birth control, Barrett has expressed skepticism about the legality of the law. In 2012, she signed a letter opposing the ACAs birth control mandate that called contraception and sterilization gravely immoral and unjust and incorrectly described the morning-after pill as an abortion-inducing drug.

Still, Njoku is steadfast in her determination to press forward. Even in the midst of so much pain and so much trauma, she said, in the midst of all of this, we still have people who are willing to continue doing this fight no matter what.

Read more from the original source:
The Fight for Reproductive Justice in a Post-Ginsburg World - The Intercept

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WILDLY fluctuating hormones mean hot flushes and zero desire for sex are often just the tip of the iceberg when it comes to The Change.

The menopause usually occurs between the ages of 45 and 55 but symptoms can start years before periods stop a time of transition known as perimenopause.

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Subtle changes in mood or menstrual cycle, trouble sleeping and pain during sex can all be early signs.

Yet for many women, symptoms are mistaken for common illnesses. Ahead of next Sundays World Menopause Awareness Day, we speak to three women who were driven to despair in their forties by their misdiagnosed menopause.

SITTING on her bedroom floor sobbing, Jo Morcom told her husband Paul she wanted it all to stop.

The mother of three had convinced herself her family would be better off without her.

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For more than a year Jo, 46, had been back and forth to see her GP, complaining of migraines and terrifying loss of memory.

Looking back, she says: As time went on, I slowly turned into someone I barely recognised.

Yet, despite repeatedly asking if her hormones were to blame, Jo says she was dismissed. She was referred for a brain scan and when that was normal she was told it was just her chronic fatigue syndrome.

Eventually Paul, 51, booked an appointment. This time he went with Jo and they refused to leave the clinic until doctors agreed to give a blood test.

The results confirmed that Jo, who also lives with chronic fatigue syndrome, was undergoing the early stages of menopause.

Experts warn that many women similarly get an inaccurate diagnosis failures that cost the NHS millions of pounds in repeat appointments, while also ruining patients lives.

Dr Louise Newson, a GP and menopause specialist, says a survey of 5,000 women showed one in three had to wait at least three years before getting a diagnosis.

She says: I constantly hear from thousands of menopausal women how they are misdiagnosed with conditions such as fibromyalgia, migraines, depression, cystitis, IBS and chronic fatigue, when no healthcare professional has considered their menopause or perimenopause as the underlying cause of their symptoms.

There is currently no formal menopause training for doctors and nurses, she says a situation that must change.

Diane Danzebrink, a psychotherapist and menopause expert, echoed the call.

She says many women are forced to pay for costly private care, resorting to credit cards and loans to pay for help they should be getting on the NHS.

Ms Danzebrink says: Women are regularly told they are too young to be menopausal and this has to stop.

Doctors need to listen to women, who know their own bodies better than anyone, and stop telling them their symptoms are all in their heads.

"This is costing women their health and wellbeing, sometimes their jobs and relationships. And it costs the NHS a fortune in repeat appointments and referrals.

For Jo mum to daughters Helana, 24, and Cate, 16, and 12-year-old son Stanley the alarm bells started to ring months earlier, while heading to her home in Broxbourne, Herts, following a night out.

She had driven the route dozens of times before but that night she got completely lost.

For Jo, a personal assistant, memory loss was the latest addition to a long list of symptoms that had started to creep in five years previously. Debilitating migraines came first, followed by brain fog, joint pain and chronic fatigue.

Over the course of 13 months and numerous visits to her GP, Jo tried to convince the doc her symptoms were hormone-related.

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She says: When I couldnt find my way home that night, I knew there was something terribly wrong. I could have driven that route with my eyes shut.

I knew it so well, which is why it was terrifying.

Jo was 41 when she started to suffer horrendous migraines that floored her for days.

She says: I would get halfway to work then have to turn around and head home because I felt so ill.

My periods were irregular too, so it was in the back of my mind that it could all be linked. Over the following months, Jo made several further appointments with her GP. She was prescribed medication for the migraines and referred for an MRI scan.

Jo says: At no point did anyone ask me about my menstrual cycle and any other symptoms.

The scan checked for brain and pituitary tumours but when it came back normal, I was diagnosed with chronic fatigue syndrome. Each time I tried to bring up the subject of hormones it was dismissed.

I couldnt sleep despite being exhausted, and my body ached all over. I looked and felt unwell.

I started looking at menopausal symptoms online and went back and forth to doctors trying to get them to listen. Time and again, each said I was too young.

Paul and the kids walked on eggshells around me. I knew I was being intolerant but I couldnt stop myself. I detached myself from family and friends, which was so unlike me.

I asked for blood tests to check my hormone levels but I was made to feel like I was time wasting and being dramatic.

Things came to a head the day Paul, a domestic appliance engineer, found Jo sobbing in the corner of their bedroom.

She says: I couldnt cope any longer and started thinking how much easier it would be for everyone if I wasnt around any more. I just wanted it all to stop.

After demanding her blood test, Jo finally had her answer.

My oestrogen and progesterone levels were completely out, she says. I felt angry that I had suffered for so long.

It should not have taken that long to get the right help. I had an idea all along.

I know my body better than anyone.

Since then Jo has switched GPs and she reckons her new doctor is fantastic.

She takes the gel form of HRT and an antidepressant, which has helped her get back on track.

She says: While I am relieved to finally get a diagnosis, things need to change.

Women shouldnt need to put up a fight to get help or be ashamed or embarrassed of the menopause.

Talking openly and honestly about it is so important.

WHEN Maria Rooneys doctor suggested she might be pregnant aged 46, Maria laughed.

The Nottingham social worker and mother of two had booked an appointment after her periods had become increasingly unpredictable.

5

Shocked at the doctors suggestion, Maria, now 49, agreed to take a pregnancy test.

She says: I knew I couldnt be pregnant because I had the contraceptive implant and had lots of other symptoms.

I was constantly exhausted and extremely short-tempered. Until then I had been a good sleeper, but I laid awake most nights. I went from being a confident and happy person to feeling low and anxious.

She finally saw a doctor in February 2017. Her own mum had gone through early menopause meaning Maria recognised the signs in herself.

Even so, she says: I didnt believe it. I thought menopause happened to women in their fifties and sixties.

When the pregnancy test came back negative, I asked if I might be going through early menopause.

The doctor looked at me like I was being ridiculous. She said I was far too young for that.

Over the following year, Marias symptoms went from bad to worse.

She says: Id forget peoples names and had to leave a job I loved. Still, nobody could give me any answers.

Nobody was listening to me, which was really frustrating. I felt like I was falling apart.

I did my own research and took what I found back to my GP.

After 18 months, I was referred to an NHS menopause clinic and given HRT tablets.

Although Im still adjusting to the medication and trying to get the dosage right, I feel relieved Im on the right path.

When I look back now, I feel angry. I suffered for so long but all I wanted was for someone to listen.

Women need to stick up for themselves and if theres something wrong, they must be listened to.

WEIGHT gain, hot flushes and low moods left Vicki Leaver a nightmare to be around, she says.

Now 41, Vicki ticked almost every box for symptoms of perimenopause yet her GP offered her antidepressants, blaming her unhappiness on lockdown.

5

The admin assistant, who lives in Perth with son Dylan, 18, and daughters Lily, six, and Jasmine, three, says: As soon as I turned 40, everything went wrong. It felt like an extreme case of PMT, but I was grumpy and snappy every day.

Feeling low and hopeless took over my life but I had no idea why I felt that way. Everyday tasks like dealing with a sink full of dirty dishes or an untidy house became too much.

Dylan took the brunt of my bad moods, which made me feel incredibly guilty. I once spent the entire school run crying.

Id wake in the night dripping in sweat and I struggled to hold a conversation because Id forget certain words.

Her periods changed too. Some months they were short and light. Other times, she would bleed so heavily she couldnt leave the house.

She continues: I made an appointment with my GP and broke down, explaining how I was feeling.

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But I got nowhere. I had blood tests that all came back normal, so I was prescribed the progesterone-only Pill to help with my heavy periods. The doctor suggested I was probably depressed because of lockdown and offered antidepressants.

But it was much more than just low moods and I wanted to get to the root of the problem, so I refused. I looked up perimenopause online and except for UTIs, which are common when hormone levels drop, I ticked almost every box.

Since January, I have talked to four different doctors and in June, I started HRT. Four months on, I feel like a different person. I wouldnt wish the last couple of years on anyone.

On Call with Dr Zoe

WHEN a woman will go through the menopause is largely predetermined before she is even born.

Thats because we are born with all our eggs. When they drop below a certain number, you are deemed to be in menopause. But how can you tell?

The best and first thing to do is speak to your mum. It is the biggest clue you have. If she went through it early, you are more likely to follow suit.

Its not an exact science but a good indication. The earlier you know, the better prepared you can be.

A good friend of mine, Zoe Hardman, recently shared her experience of facing the menopause at just 37 with Fabulous. It was only when her older sister discovered that she was menopausal aged just 34, while trying to get pregnant that they spoke to their mum and discovered that she had been through early menopause.

After openly talking about it she said she received so many messages from other women in similar situations, women who shared the shame they had felt.

No woman should feel any shame about facing menopause, whatever her age.

Its so important we all speak about it, to break the taboo and destigmatise something that is completely natural and happens to every woman.

Shame and stigma can be a barrier to women getting the right support and the right treatment. If you are under the age of 45 and start to notice you are suffering with symptoms you think are linked to menopause, it is really important you speak to your doctor.

Appointments with your GP are a partnership. We might have the knowledge but YOU know how you feel. You know when something is not right.

The important thing is to be straight with us. Dont just come in, describe your symptoms and leave it hanging. If you think you are suffering menopausal symptoms, tell us.

Unfortunately, the signs and symptoms of perimenopause and menopause are very similar to lots of other common conditions that affect women in their late thirties and early forties.

Gut feeling

Read more from the original source:
Life was hell but our GPs didnt believe we had started menopause in our 40s - The Sun

Recommendation and review posted by Bethany Smith

Olivia Munn opened up about freezing her eggs during an interview for the Anna Faris is Unqualified podcast in 2016, saying that she did it because her friend found out she "had the egg count of a 50-something-year-old woman." Munn decided to visit a doctor herself, and while she was told she has "a lot of eggs," she was also told that she might as well freeze some of them so that she would always have them available.

The star also talked about turning 35, saying that's when "you're high risk." However, "many doctors are quick to discount the belief that age 35 always and instantly draws a line between a healthy pregnancy and a high-risk one," according to Self. Indeed, Sarah J. Kilpatrick, M.D., Ph.D., chair of the department of obstetrics and gynecology at Cedars-Sinai, told Self, "I would never tell someone that just because she's 35 she has to see a high-risk doctor only if there's something in her history or something that happened during her pregnancy that warrants it."

That may be true, but Munn clearly still feels confident in her decision. "I think that every girl should do it. ... [For] one, you don't have to race the clock anymore. You don't have to worry about it," she told the former Mom star and podcast host. "Doomsday is now like 'whatevers day,' because I am prepared."

Original post:
Stars who have frozen their eggs - Nicki Swift

Recommendation and review posted by Bethany Smith

Emmanuelle Charpentier and Jennifer Doudna have been given the 2020 Nobel Prize in Chemistry for their discovery and development of CRISPR-Cas9 genome editing.

The Royal Swedish Academy of Sciences has decided to award the Nobel Prize in Chemistry 2020 to Emmanuelle Charpentier of the Max Planck Unit for the Science of Pathogens, Germany, and Jennifer Doudna of the University of California, Berkeley, US. The Nobel Prize is for the development of CRISPR-Cas9, a method for genome editing.

According to the Royal Swedish Academy of Sciences, Emmanuelle Charpentier and Jennifer Doudna discovered the CRISPR-Cas9 genetic scissors. Using these, researchers can change the DNA of animals, plants and microorganisms with extremely high precision. This technology has had a revolutionary impact on the life sciences, is contributing to new cancer therapies and may aid in curing inherited diseases.

Also, using the CRISPR-Cas9 genetic scissors, it is now possible to change DNA over the course of a few weeks. This used to be time-consuming, difficult and sometimes impossible work.

There is enormous power in this genetic tool, which affects us all. It has not only revolutionised basic science, but also resulted in innovative crops and will lead to ground-breaking new medical treatments, said Claes Gustafsson, chair of the Nobel Committee for Chemistry.

The discovery of CRISPR-Cas9 was made during Emmanuelle Charpentiers studies of Streptococcus pyogenes, one of the bacteria that cause the most harm to humanity. She discovered a previously unknown molecule, tracrRNA. Her work showed that tracrRNA is part of bacterias ancient immune system, CRISPR-Cas, that disarms viruses by cleaving their DNA.Charpentier published her discovery in 2011. The same year, she initiated a collaboration with Jennifer Doudna, an experienced biochemist with vast knowledge of RNA. Together, they succeeded in recreating the bacterias genetic scissors in a test tube and simplifying the scissors molecular components so they were easier to use.

In another experiment, they then reprogrammed the genetic scissors. In their natural form, the scissors recognise DNA from viruses, but Charpentier and Doudna proved that they could be controlled so that they can cut any DNA molecule at a predetermined site.

The academy highlights that since Charpentier and Doudna discovered the CRISPR-Cas9 genetic scissors in 2012 their use has exploded. This tool has contributed to many important discoveries in basic research and clinical trials of new cancer therapies are underway.

Continued here:
Nobel Prize in Chemistry awarded to discoverers of CRISPR-Cas9 - Drug Target Review

Recommendation and review posted by Bethany Smith

Graphic Source: CRISPR Therapeutics, Inc.

CRISPR Therapeutics (NASDAQ:CRSP) is a gene-editing company focused on the development and versatile application of CRISPR/Cas9 therapeutics, a special brand of therapeutics used for precision genome editing by applying a viral defense mechanism from bacteria to regulate, disrupt, or correct genes related to key diseases. CRSP is currently targeting disease areas, including hemoglobinopathies, oncology, and regenerative medicines.

Founded in 2013 in Switzerland, CRSP has since grown to over 304 employees producing relatively inconsistent revenues ranging from $3M in 2018 to $290M in 2019 with expectations for 2020 at $6.7M. Their lead candidate is CTX001, an investigational autologous gene-edited hematopoietic stem cell therapy developed in partnership with Vertex Pharmaceuticals (NASDAQ:VRTX) for treating transfusion-dependent beta-thalassemia ("TDT") and severe sickle cell disease ("SCD").

Products: CRSP's pipeline consists of 9 therapeutics: 4 in the clinical phase and 5 in the research phase. Of the 4 clinical phase therapeutics, the first targets TDT and SCD (mentioned above: CTX001), while the 3 others fall into immuno-oncology covering: CD19+ malignancies (Product: CTX110), multiple myeloma (CTX120) and solid tumors and hematologic malignancies (CTX130). All immuno-oncology therapeutics are allogeneic CRISPR/Cas9 gene-edited CAR-T cell therapies wholly owned by CRISPR Therapeutics with data updates typically every 6 months.

Customers/market: For CRSP's clinical phase pipeline, the total estimated 2022 global market potential is $220B with an average market size for each disease of $36.7B growing at an average 15.2% CAGR (median market: $13.3B | CAGR 10.9%). The largest market is Solid Tumors, at a 2022 estimated size of $145B (8.1% CAGR), and the highest CAGR market CAR T/CD19+ market at a 34.5% CAGR. For CTX001, the lead candidate, the target market can be broken down into the TDT market at very roughly $1.8B with a 10.8% CAGR and the SCD market at $4.1B with an 11% CAGR by 2022.

Management: Many are now familiar with co-founder Dr. Emmanuelle Charpentier, who in 2020 under much-debated circumstances co-received the Nobel Prize in Chemistry for her work with developing the CRISPR/Cas9 genetic scissors, the foundation of CRSP's therapeutics today. In addition to her role now as Scientific Advisory Board Member, CRSP has a variety of other accomplished leaders.

CEO: Dr. Samarth Kulkarni has served as CEO (covering long-term strategy) since December of 2017 when he was promoted from President and Chief Business Officer. Before CRSP, he was a Partner at McKinsey & Company (MCK) co-leading the biotech practice. His specialties are in strategy and operations, and he has a Ph.D. in Bioengineering and Nanotechnology.

Share Price Change under his leadership (Dec. 2017 - Present): 499% | CAGR: ca 71%.

President/Chairman: Dr. Rodger Novak, a co-founder with Charpentier and Shaun Foy in 2013, has served as CEO until 2017 and since, as President (day-to-day operations) and Chairman. Rodger is an experienced biotech/pharma executive having served in leadership positions (primarily covering infectious diseases and related) at Sanofi and Novartis. He co-also founded Nabriva Therapeutics (NASDAQ:NBRV). His specialty is in translating scientific technologies into pharmaceutical products. Before all else, he was a professor of Microbiology at the Vienna BioCenter (Austria).

Other management updates:

Strategy: In terms of strategy, CRSP stated they intend to use their scientific expertise, together with their unique platform to bring about a new class of highly active and potentially curative therapies for specialty patients to which biopharmaceutical approaches have limited exposure. CRSP has been investing heavily in its long-term platform.

Additionally, CRSP seems to be taking the lead in most of its partnerships, particularly the ViaCyte partnership evidenced by the structure of their collaboration agreements and payments due to ViaCyte. Investors also saw last year CRSP buying Bayer's (OTCPK:BAYZF) control of Casebia and expanding their internal clinical pipeline with internal funds (hence the cash stockpile). CRSP is acting as the all-or-nothing winner. It is a unique approach that expresses internal confidence in their technology and financial capabilities.

Financial position: CRSP received new upfront payments from Vertex in 2019 boosting revenues to $290M, which are not expected to be repeat. The estimated decline in 2020 brings revenue to $6.7M (-98%). 2019 was the only year in the company's history to achieve net income ($67M), whereas the 3-year average net loss is -$56M. For 1H 2020, net losses are already -$149M. CRSP operates with a strong cash cushion of $945M at 1H 2020, enough to cover the -$42M 3-year average CFO+CAPEX expenditures for 22+ years. Total debt as of 1H 2020 is a manageable $50M ($40M in capital leases). Accounts payable are a small $13M.

Investment thesis: Although most of CRSP's products are years away from revenue-generating outside of milestone payments, the constant updates from clinical trials offer a compelling progress report for the long position. Investors must be willing to pay for the premium that exists currently, but with CRISPR being in the public spotlight, investor interest may increase. The real question remains if they are truly the most advanced CRISPR position, though clearly not discounted. Operational strategy is a key selling point with the new McKinsey-inherited leadership, but science is still the core of any biotech investment. Below will be an expounded analysis of what their therapeutics are, but it seems to be that CRSP has a compelling niche for the long-term investor, given their 7+ years of experience with this new biotechnology. Therefore, with the Vertex partnership, enough cash to keep steady progress, and stable operational-based leadership, CRSP is a "buy".

CRSP's pipeline consists of 9 therapeutics: 4 clinical phases and 5 in the research phase. CRSP is supported by 1 strong partnership and 1 weaker partnership including:

Vertex partnership (est. 2015) for clinical analysis of TDT and SCD with a research phase target of cystic fibrosis ("CF") is based around co-developing CTX001 (since Dec. 2017). The partnership did expand in June 2019 for Duchenne muscular dystrophy ("DMD") and myotonic dystrophy type 1, which adds further upside potential for CRSP.

In 2015, CRSP received a $75M upfront payment and in 2017 received $7M and, thereafter, a low-seven-digit milestone payment for second patient dosing related to TDT and SCD. Looking forward, CRSP is eligible for up to $420M for further milestones and product-sales royalties.

In 2019, after the new collaboration agreement was established for DMD and myotonic dystrophy type 1 ("DM1"), CRSP has received an upfront payment of $175M with eligible milestone payments up to $825M. Tiered royalties on product sales are also available. The DMD program makes Vertex responsible for R&D, manufacturing, and commercialization. For DM1, CRSP will cover RNA research with Vertex responsible for all other costs. After DM1 IND filing, CRSP retains the option to co-develop/co-commercialize all DM1 products but must forgo milestone payments/royalties and cover 50% of R&D costs incurred by Vertex. Similar amendments were made to the 2015 agreement. In Oct. 2019, Vertex accepted the right to exclusively license the three remaining options granted under their 2015 agreement, resulting in CRSP receiving a $30M 4Q 2019 payment. CRSP also received a milestone $25M payment in 2Q 2020. At 1H 2020, CRSP had $11.8M of non-current deferred revenue related to Vertex.

ViaCyte partnership (est. Sep 2018) for diabetes through gene-edited allogeneic stem cell therapies. A key aspect of this partnership is ViaCytes stem cell capabilities and CRSP's gene-editing capabilities to enable beta-cell replacement without the need for immune system suppression. After successful completion of the studies proving verification of developing the immune-evasive stem cell line, CRSP and ViaCyte will jointly be responsible for further development and commercialization, globally. The partnership entitled ViaCyte to $16.2M in payments for participating which CRSP recognized as $15M in R&D expense and $1.2M in other expenses. The expected partnership term is in force for 5.5+ years and obligates both parties to jointly develop the research plan with each party responsible for their research costs.

Bayer partnership (JV est. 2015) was terminated in 4Q 2019 which included Casebia Therapeutics and focused on treating genetic causes related to bleeding disorders and autoimmune diseases, amongst others. CRSP retained full-ownership of Casebia and Bayer retained the right to co-develop two therapeutics related to autoimmune disorders, eye disorders, and hemophilia A disorders with exclusive licenses; termed the "2019 Option Agreements".

The clinical phase therapeutics of CRSP will be outlined below.

Graphic Source: CRISPR Therapeutics Investor Presentation (Sep 2020)

Therapeutic 1: CTX001 is CRSP's lead candidate targeting TDT and severe SCD through an investigational autologous gene-edited hematopoietic stem cell therapy. It is being co-developed by Vertex Pharmaceuticals.

TDT: CTX001 is currently in a Phase 1/2 open-label clinical trial (CLIMB THAL-111) for transfusion-dependent -thalassemia. The study aims to assess safety and efficacy for single dosages of CTX001 in a 12-35-year-old population with TDT. In 4Q 2019, Vertex & CRSP expanded the TDT patient population to include beta 0/beta 0 subtypes with the first two severe patients indicating successful dosing and engraftment. The study is designed for up 45 patients and aims to follow them for a duration of two years post-infusion with 6-month investor updates. CTX001 has received the Regenerative Medicine Advanced Therapy ("RMAT"), fast-track, and orphan drug designations (+European Commission) by the FDA for treating TDT. Preliminary clinical data was released in 4Q 2019, and in June 2020, their 15 months of follow-up data were also released in the ongoing study.

SCD: CTX001 is also currently in a Phase 1/2 open-label clinical trial (CLIMB SCD-121) for severe sickle cell disease testing safety and efficacy for single dosages of CTX001 in an older patient population than TDT (18-35). Similar to CLIMB THAL-111, the first two patients were treated sequentially then expanded for up to 45 concurrent patients for a 2-year following. CTX001 for SCD has also received the same designations as for TDT with the same data publication dates but in June releasing only 9 months of follow-up data.

Competition Notes:

Therapeutic 2: CTX110 is CRSP's lead candidate amongst their wholly-owned CAR-T therapies, which is a gene-edited allogeneic CAR-T therapy targeting CD19 in CD19+ malignancies cases. It's currently in a Phase 1 study focused on safety and efficacy ("S&E") in the treatment of relapsed/refractory B-cell malignancies. The study is designed for up to 131 patients on a multi-dose level.

Therapeutic 3: CTX120 is another gene-edited allogeneic CAR-T therapy but targeting B-cell maturation antigen. CTX120 is in a Phase 1 S&E study for treating relapsed/refractory("R&R") multiple myeloma. It is also a multi-center open-label trial but designed for up to 88 patients also on a multi-dose level investigation.

Therapeutic 4: CTX130 is CRSP's other CAR-T therapy with a target of CD70, the antigen expressed on solid-tumors and hematologic malignancies (study's target). The treatment is developed around solid-tumors (e.g. renal cell carcinoma) and T/B-cell hematologic malignancies. CRSP is currently running two independent Phase 1 S&E studies for CTX130 treating R&R renal cell carcinoma and other types of lymphoma. The first Phase 1 study, focused on R&R renal cell carcinoma, is a multi-center open-label investigation with an enrollment of up to 95 patients on a multi-dose level, and the second study for various lymphomas is designed for up to 46 patients.

For further analysis of the science and clinical trial updates, see clinical.gov studies linked above, the September 2020 Investor presentation, or the Chardan Conference Call (Sep 2020).

Table Source: Self Created | Data Source: Seeking Alpha - CRSP

Revenue/cash flow: Financially, CRSP is far from stable on any operating metric (other than cash resources), but survived COVID with 80-90% productivity. Revenue is earned from collaboration agreements and their associated milestones. This swayed investors in 2019 when $289M (99.6% of 2019 Revenue) was recognized from the Vertex partnership expansion. This comprised revenue from the DMD and DM1 licenses worth $202M and from Vertex exercising their "Collaboration Target Options" worth $76.7M and a $6.7M payment for Vertex waving their fourth exclusive license. Neither CRSP nor the author expects this to continue in the next 1-2 years on any metric unless new partnerships are formed and upfront payments are made.

CRSP does have $12.6M in unearned revenue accounted for, but only 1M as current. Analysts do expect another $6.5M in revenue 2H 2020, but that only brings the tangible revenue benchmark to $6.7M for the year; however, the focus is on the viability of the therapeutics and not revenue benchmarks. It seems investors in CRSP think the same. New and old investors should focus on the results of clinical trials for the foundation of any investment thesis as accessing the Vertex milestone payments worth up $1.25B is directly the result of the Phase 1/2 CTX001 results and the Pre-IND research phase targeting DMD, DM1, and CF.

Balance sheet composition:

Table Source: Self Created | Data Source: Seeking Alpha - CRSP

Regarding the balance sheet, what stands out for investors is the high cash balance accumulated ($945M), particularly from Vertex, which is sufficient to finance the existing pipeline of 4-core products (3 internal, 1 external) and a few promising research phase therapeutics. The low-unearned revenue ($13M) enlightens investors towards the short term, but with little debt ($50M) and capital leases making up the majority portion ($44M), there isn't a heavy draw on cash that shouldn't produce an advancement towards reaching clinical phases. What is worrisome though is the desire for only 2 external partners (1-lead and 1-sub) which may be attributed to the McKinsey style leadership that in the author's opinion does not foster the right developmental environment and deviates from the biotech norm. This may be a benefit in some investors' eyes as the rewards will be substantial in 3-7 years, but it is a long-term position an investor must make.

Table Source: Self Created | Data Source: Seeking Alpha - CRSP

Firstly, any valuation on CRSP at this point is highly speculative, given the inconsistent milestone marks being met and their highly divergent payments with relatively new technology (though bluebird and Novartis are setting precedents). A revenue basis seems the most closely tied to reality and by using 11-14 analyst forecasts, an approximate valuation can be made as above with base-case (+2% upside) being far too conservative due to the reactions investors take upon clinical trial announcements and certainly not enough to lure any biotech investor.

An upside of 311% in an optimistic scenario seems more than compensatory to the risk, given the large cash cushion. However, a premium of 894x Sales is remarkable, but outlandishly inaccurate due to the fluctuations of the revenue position which does not compensate for the high potential of gaining market share in the $220B market their combined therapeutics target. In summary, the author will say that an upside potential exists but is uncertain beyond 30% in the short term (1-2 years).

Data by YCharts

Upcoming Catalysts (1-12 months):

In summary, CRSP is in a very unique position with such a large $945M cash cushion and a promising advancement on therapeutic development with cutting-edge technology and 7+years of experience in it, far greater than most enterprising biotechs looking into genome-editing. The partnership with Vertex, a leader in its sphere, does provide substantiation to the technology CRSP is applying, but with only two partnerships, it seems CRSP is either too early to the game or is not moving fast enough. CRSP does face competition, such as bluebird's TDT gene-therapy; however, what CRSP is building should surpass competition if it can swiftly make it to market compensating investors that must withstand early-clinical phase results risk. CRSP has stated their platforms are worthy of reaching a $100B company status, but the author adds that investors must be patient. There are few companies that the author truly feels uncertain regarding valuing, and CRSP is one of them. Any realistic valuation would greatly underestimate the potential of CRSP, but by averaging the downside and the upside potential, a reasonable price target can be surmised from analyst expectations. Therefore, the author projects CRISPR Therapeutics as a "buy" for the long-term investor with an uncertain 2-year stock price target at $204.63 (+113% upside).

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Read the original here:
Crispr Therapeutics: Waiting On Early Data In 2H 2020, But The Clinical Pipeline Shows Promise (113% Upside) - Seeking Alpha

Recommendation and review posted by Bethany Smith

Researchers in the US led by the 2020 Nobel laureate for chemistry Jennifer Doudna have used a CRISPR gene-editing technology to develop a rapid, portable, accurate, and low-cost mobile-based test that can detect the pandemic coronavirus (Sars-CoV2) in five minutes.

This new CRISPR diagnostic method doesnt amplify coronavirus RNA but uses multiple guide RNAs that work in tandem to increase the sensitivity of the test, said the research team in the yet to be peer-reviewed study published in the pre-print server medRxiv. The test does not require expensive lab equipment, and can be deployed for rapid point-of-care testing at doctors offices, schools, and office buildings.

The diagnostic gold standard for coronavirus disease diagnosis is the quantitative reverse transcription-polymerase chain reaction (RT-qPCR) test, which takes five to six hours to produce result. CRISPR-based diagnostics that utilises RNA and DNA-targeting enzymes can augment gold-standard PCR-based testing if they can be made rapid, portable and accurate, said Doudnas team.

The assay achieved ~100 copies/L sensitivity in under 30 minutes and accurately detected a set of positive clinical samples in under 5 minutes. We combined crRNAs targeting SARS-CoV-2 RNA to improve sensitivity and specificity, and we directly quantified viral load using enzyme kinetics. Combined with mobile phone-based quantification, this assay can provide rapid, low-cost, point-of-care screening to aid in the control of SARS-CoV-2, write researchers.

The test also quantifies the amount of virus in a sample, with the strength of the fluorescent signal proportional to the amount of virus in a sample. This cannot be done in standard Covid-19 tests that amplify the virus genetic material to detect it. Detecting a patients viral load can guide treatment decisions. The test needs validation before it is commercially available.

CRISPR diagnosis work by identifying a sequence of RNAabout 20 RNA bases longthat is unique to SARS-CoV-2. They do so by creating a guide RNA that is complementary to the target RNA sequence so it binds to it in solution. The binding turns on the CRISPR tools Cas13 scissors enzyme that cuts single-stranded RNA to release a separately introduced fluorescent particle in the test solution. These fluorescent particles light up to when hit with laser light, signaling the presence of the virus.

CRISPR diagnostics is already being used for Sars-CoV-2 detection, but the new test is the fastest CRISPR-based diagnostic yet.

I think this is an interesting new approach that is faster because it doesnt have an amplification step. But, because it doesnt have the amplification step, it cannot easily detect low viral loads unlike qRT-PCR or FELUDA. The five minutes result is only when starting from RNA with high amount of virus. For usual samples it will be more than 30 minutes. The device requirement is not zero but low - a constant temperature holder and a laser illumination optical box. I think CRISPR based tests will see a lot of innovation and it is a good sign for the fight against COVID-19, said Dr Anurag Agrawal, director, Council of Scientific & Industrial Research-Institute of Genomics and Integrative Biology (CSIR-IGIB), New Delhi.

Scientist from CSIR-IGIB have also developed a precise and cost-effective strip test named after a popular Satyajit Ray detective FELUDA to detect Covid-19 in one hour, starting from RNA to giving a visual readout on the strip.

FELUDA, which is an acronym for FNCAS9 Editor-Linked Uniform Detection Assay, uses CRISPR gene-editing technology to identify and target the genetic material of Sars-CoV2, the virus that causes Covid-19. It has been developed by CSIR-IGIB senior scientists Dr Debojyoti Chakraborty and Dr Souvik Maiti.

FELUDA tests work by combining CRISPR biology and paper strip chemistry. A Cas9 protein, a component of the CRISPR system, is barcoded to interact specifically with the Sars-CoV2 sequence in the patients genetic material.

The complex of Cas9 with Sars-CoV2 is then applied to a paper strip, where using two lines (one control, one test) make it possible to determine if the test sample was infected with Covid-19.

Going forward, CRISPR-based tests have the potential for modification to detect the next emerging virus and rapidly scale up testing, if needed.

The rest is here:
Covid detection with CRISPR, phones in offing - Hindustan Times

Recommendation and review posted by Bethany Smith

With the vision of linking computers up to the LIVE signals of biology, Cardea now adds an advisory team of Key Opinion Leaders covering all the cross disciplinary efforts being done.

SAN DIEGO (PRWEB) October 13, 2020

Cardea Bio Inc., who is using graphene-based Biology-gated Transistors (Cardean Transistors) to directly link the live signals that run biology up to electronics and computers, today announced the Cardea Innovation Council. The Council will serve as an advisory body to guide and help the Cardea team of talents to continue the breakthroughs being made via Cardea's core technology, Cardean Transistors. The council members will also participate in the Company's Innovation Partnership Program on relevant projects. The Council consists of a body of Key Opinion Leaders and experts from diverse science and technical fields and will bring a depth of knowledge to aid Cardea in building the most complete Tech+Bio Communication Chipsets and Infrastructure available for current and future generations.

Cardea is pleased to welcome the Council Members to its team:

Professor Susan Wessler

Distinguished Professor of Genetics and the Neil and Rochelle Campbell Chair for Innovation in Science Education at the University of California Riverside. In 2011 she was elected Home Secretary of the U.S. National Academy of Sciences (NAS), the first woman to hold this position in its 150-year history. She is a plant molecular geneticist known for her contributions to the field of transposon biology and plant genome evolution.

Professor Virginijus Siksnys

Among the very first to discover and characterize the CRISPR-Cas9 complex and recognize its editing potential for "DNA surgery" in many life science applications. His pioneer work was recognized with several international awards including the Kavli Prize. Since CRISPR-Chip is an important chipset type for Cardea, his expertise regarding CRISPR is important in improving fast and precise (amplification-free) DNA and RNA detection.

Dr. Kurt Petersen

Founder and CTO of numerous MEMS (Micro-ElectroMechanical System) companies, including NovaSensor, Verreon and molecular testing company Cepheid. Kurt was recently awarded the IEEE Medal of Honor for his contribution to the field of MEMS. Cardean Transistors are very similar to MEMS in many regards and Kurt's experience with MEMS as it applies to biotechnology will elevate Cardea's chip designs, scalability and capabilities.

Dr. Phil Cotter

Fellow of the American College of Medical Genetics and Genomics as well as founder and Principal of ResearchDx. Dr. Cotter has been a leader in developing sequencing as a clinical diagnostics tool as the Director of Illumina Clinical Services Laboratory, and through ResearchDx, a leader in development of companion diagnostics based on DNA, RNA and protein biomarkers.

Dr. Lauge Farnaes

As Head of Medical Affairs at IDbyDNA, and Medical Doctor & Researcher formally at Rady Children's Genomic Institute with expertise in genetic disorders, infectious disease detection, and nucleic acid-based diagnostics, Dr. Farnaes has among other things helped pioneer the use of Rapid Whole Genome Sequencing to diagnose rare genetic disorders in children.

Dr. Elia Stupka

A visionary Key Opinion Leader in bringing the most advanced forms of big data and analytics to healthcare. Dr. Stupka's work has contributed to the understanding of the human genome, transcriptome, and the development of gene-therapeutics. His understanding of data and its role in enhancing the value of new technology will help steer Cardea's data management capabilities and add value to every Innovation Partner application.

Dr. Paul Grint

Chairman of the Innovation Council and Chairman of the Board of Directors of Cardea. Dr. Grint has served on the Boards of Illumina, AmpliPhi Biosciences, and as the CEO of several companies. Dr. Grint's ability to see the early potential in technology served Illumina well when Next Generation Sequencing was in its infancy as it will Cardea with its Biology-gated Transistors.

"Cardea's core technology is really the convergence of many highly complex technical fields such as life science, data analytics, and semiconductor technology." says Dr. Grint. "In order for Cardea to be successful in its mission to elevate the world's ability to gain new insight into biology, we need the best from every field. The Innovation Council will elevate Cardea's capabilities across all of these fields."

The news of Cardea's Innovation Council comes only weeks after the company announced the first close of their A2 round and first commercial "Powered by Cardea" product launch. This is a major move for Cardea on its mission to continue developing even more products together with their Innovation Partners. To learn more about the Council, visit Cardea's website.

About Cardea Bio

Cardea is linking biology directly up to computers for the very first time by building a Tech+Bio Infrastructure and offering chipsets based on proprietary Biology-gated Transistors, or Cardean Transistors. These transistors leverage graphene, a nanomaterial that in contrast to the common semiconductor material silicon, is biocompatible and a near perfect conductor due to only being one atom thick. It that way replaces optical static observations with interactive live-streams of multi-omics signal analysis, representing a new life science observation paradigm where multi-omics data-streams will be the new norm instead of most of the current standard technologies that are single-omics frozen-in-time datasets. Together with their Innovation Partners, Cardea can link biology directly to compute power and convert real-time biological signals to digital information, allowing for immediate biological insight and a new generation of applications Linking up to Life.

Contacts

Partnership inquiries

Rob Lozuk

Chief Business Officer

PublicRelations@Cardeabio.com

Media inquiries

Amanda Zimmer

Marketing Manager

marketing@cardeabio.com

For the original version on PRWeb visit: https://www.prweb.com/releases/crispr_pioneer_home_secretary_of_the_u_s_national_academy_of_sciences_ieee_medal_of_honor_recipient_and_other_experts_joins_cardea_bios_innovation_council/prweb17465996.htm

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CRISPR Pioneer, Home Secretary of the U.S. National Academy of Sciences, IEEE Medal of Honor Recipient, and other experts joins Cardea Bio's...

Recommendation and review posted by Bethany Smith

Germline genetic testing is essential in order to identify optimal treatments for patients with cancer, as well as detecting inherited mutations via cascade testing that could affect family members, according to John M.Carethers, MD, MACP, who emphasized that improvements to genetic testing technology and testing costs has increased not only the accuracy of, but access to these assays.

The technology in sequencing has moved from the old gels to capillary to ChIP [chromatin immunoprecipitation]-based, and has revolutionized the way we approached it. The depth of [genetic testing] coverage [has evolved], said Carethers. Sequencing technologies totally revolutionized this [process].

He added, There are some unusual situations in which additional technologies have to be used to figure out some of the ones that typical ChIP technologies don't fully explain. That has markedly changed the way we approach [testing] these days.

In an interview withOncLiveduring the 2020 Institutional Perspectives in Cancer (IPC) webinar on Precision Medicine, Carethers, a professor of Internal Medicine and Human Genetics at the University of Michigan, discussed recent developments in multi-gene panel testing.

OncLive: How are predictive and somatic genetic tests being utilized in clinical practice?

Carethers: In terms of germline testing, the benefit is knowing which disease you carry, and that information can also spread to other family members to understand whether they [are at an increased risk of getting a cancer diagnosis]. Sometimes, at least in my experience, [germline testing] does alleviate some anxiety. Some people get more anxious once they know they have a germline mutation, but in general, it does at least explain the reason why they're seeing certain diseases in the family. Thats the general benefit for germline testing.

The benefit of somatic testing is knowing the type of mutations that occur in the tumor; there may be a therapeutic drug or compound that is in current use that could benefit the patient. For instance, I had a patient with unresectable esophageal cancer. She was dying and her esophagus was almost completely obstructed with the tumor. She had a feeding tube put into her stomach and lost a lot of weight; she was literally counting out the days until she died. With some thought, we decided to take a sample of the tumor and do somatic testing.

She had some mutations that werent typically found in esophageal cancer, and we did have drugs [to treat her]. She was actually put on those drugs and the tumor shrunk dramatically to the point that she could eat again, she gained weight, and she lived another 5 years. Normally, she wouldn't have lasted more than a few months. The benefits of somatic testing is understanding the genetic makeup of the tumor in which you might be able to use some compounds that exist to benefit the patient. Thats the real goal of somatic testing.

There is an unusual situation for somatic testing, as well. For instance, in colon cancer, we know about Lynch syndrome, but there is also a Lynch-like syndrome. In Lynch-like syndrome, there is no germline [mutation], but the tumor has 2 somatic mutations of a mismatch repair deficient tumor. They can look like a Lynch syndrome tumor, and maybe even behave a little bit like a Lynch syndrome tumor, but they're really not caused by a germline mutation. Sometimes, somatic genetics can help us understand tumor genesis as well as ways to treat the tumor.

What changes have we seen recently in multigene panel testing? How are test results interpreted and how do they help guide treatment strategies?

There are patients who will walk in with the classic phenotype and then there are patients walking in who don't have the classic phenotype, yet they carry that mutation in the same gene. Multigene testing allows us to account for phenotypic variation.

Someone may walk in with colon cancer, the next person in the family might walk in with endometrial cancer, and the next person in the family may walk in with a skin tumor, but they all line up with the same mutation in Lynch syndrome. However, if you saw the skin tumor first, would you have thought of Lynch [syndrome]? [What about] if you saw the endometrium or the colon cancer? It depends on the specialty and the type of disease presentation they show up with. In many cases, though, the disease could be subtle.

For instance, there was a family I followed, which comprised the grandmother, mother, and daughter. The grandmother, who was well into her late 60s, had a Lynch syndrome mutation and got her colon removed appropriately. The mother was in her 40s with no cancers, but the daughter who was 21, developed colon cancer. It looked like it skipped a generation, yet, they all carry the same mutation. There's phenotypic variation, even with this exact same mutation in the family, because we're all genetically different to some, so there's probably modifiers and other things going on. However, if I can see that in this one family who I know [harbor that specific] mutation [then I know that] if multiple people walk into the clinic and have variations in their family histories and in their personal history of cancer, that we are seeing a wide phenotypic variation.

Now, instead of testing 1 gene at a time, we will test 30 or 50 genes at a time, and you can pick up some of these less penetrant genes that are causing the phenotypic variation. Sometimes there are major penetrant genes in these families.

What other barriers to germline testing need to be addressed?

We're always learning. Every year or so we add a few more genes to our repertoire and then, maybe they get on some of these panels. E3 ubiquitin ligase WWP1 is associated with PTEN hamartomatumorsyndrome, which is not on any panels, but the paper was published in the New England Journal of Medicine. We keep learning as we discover more and more of these genes. The more genes that we find tend to occur in less and less people, based on our current knowledge, but some of these patients present with these rare phenomena.

We're also finding out that some of these mutations arent specifically a change in the DNA sequencethere are methylation, or rearrangement, or even a deletion. You have to use other techniques in addition to sequencing to figure those families out or those families will be left in the lurch.

The downside of doing multigene panel testing is that now, if you push for more whole-exome and whole-genome sequencing, we have a lot more variants. One commercial lab got [results] back to me 2 months ago from a patient we had tested 4 years ago. They said, We finally have enough people [where we could determine that] his variant is not significant. It was good news. We are now more sure of variants because they now have more families in their database at the commercial lab. Sometimes it takes years to figure it out, unless we have functional analysis for all variants. Thats a big challenge right now.

Where do you hope to see the future of genetic testing head?

In a good way, genetic testing will probably [have a lower] cost and there [will be an] ease of doing it [with] whole-exome and whole-genome sequencing. It will even overtake panel testing over time because the machines are better and faster. The key, though, is having a database that you can go back and forth and analyze. Youre going to need the analytics and tools. What happens with the patient? Do I carry this [information] on a flash drive? Is it in a database I have to have access to?

It's not an easy answer and I'm not sure if the health system that a particular patient goes to is going to store all this information3 billion base pairs of informationand go back to it each time. Each place is going to have to have the right analytic tools to go back and [retrieve that information]. There are going to be some challenges with that, even though that's the way the technology is going.

The more challenging pieces [are related to] direct-to-consumer (DTC) testing. You don't always know what you're getting on those tests. We can test you for common diseases, such as diabetes and hypertension, but we also test you for BRCA1/2. In reality, very few of the DTC [tests] are doing sequencing or panel testing like we do clinically. Many of them are using single nucleotide polymorphisms (SNPs) that give you a suggestion. Many of these start from ancestry companies,and they recently moved into [testing for] these diseases because people are interested. I don't blame them for doing this, but the information they give might only [include] a fraction of the actual disease variants. If someone finds an SNP in BRCA1/2 or Lynch syndrome, you might need to see a doctor. [Based on your family history or phenotype,] we may have to send a ChIP test to verify [the results].

In some cases, people will test just to be curious, and they think they're going to have something, but there is zero evidenceno personal history and no family history. There are going to be some challenges with the DTC [testing] because we don't always know the type of test theyre getting and the information is not going to be as precise and could present challenges in the clinics. Some people are going to get upset because we're going to say, No, you don't need testing, or [patients will ask], Why does this test say I might have it but your test says I don't? We have to explain all this and those are going to be challenges.

What else would you like to add regarding the evolution of genetic testing?

There is phenotypic variability in the presentation of many of these syndromes. The standard now is multigenetic panel testing to try to assuage the phenotypic variation; we do pick up [genes in] people who we didn't necessarily think had that disease. I've been surprised too many times, so I'm not surprised anymore. A lot of these inherited conditions have phenotypic variability. If you have any suspicion or your primary care physician has any suspicion, feel free to send [a test] to our clinic because we can investigate that and do testing that's relatively cheap if there's a good cause to investigate that. It may save their life and the lives of their loved ones.

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Focusing on the Future of Genetic Testing in Oncology - OncLive

Recommendation and review posted by Bethany Smith

A new study by University of California researchers shows the promise of high-throughput DNA-sequencing technologies to improve prenatal diagnosis and pregnancy outcomes for women who have experienced an abnormal prenatal ultrasound.

In the UC San Francisco-led study, scientists used a technique called exome sequencing to identify genetic diseases as the underlying cause in 37 of 127 cases of nonimmune hydrops fetalis (NIHF), a life-threatening condition in which the fetus is overloaded with fluid. The study was published online Oct. 7 in The New England Journal of Medicine (NEJM).

Corresponding author Teresa Sparks, MD, MAS, a UCSF assistant professor in the Department of Obstetrics, Gynecology & Reproductive Sciences, led the study with senior study author Mary Norton, MD, a professor in the same department. The cause of most cases of NIHF is not identified with standard testing, but when we apply exome sequencing, we find a genetic diagnosis in nearly 30 percent of cases of previously unknown cause, Sparks said.

NIHF affects about one in every 1,700 to 3,000 pregnancies in the United States and is associated with high risks of stillbirth, preterm birth, neonatal death and other complications. Although NIHF often leads to death, identifying the precise genetic cause is critical, as associated outcomes vary widely in severity.

NIHF can be a manifestation of many genetic diseases, but evidence of abnormal fluid accumulation in the fetus detected through an ultrasound exam whether it occurs under the skin, in the abdomen, or around the heart or lungs does not pinpoint an underlying cause.

Participants in the study were referred from throughout the United States after NIHF was identified with prenatal ultrasound but no underlying genetic disease was found using long established methods for detecting genetic abnormalities. These traditional genetic tests karyotype and chromosomal microarray analysis detect large abnormalities in chromosomes, not disorders caused by a defect in a single gene as are identified with exome sequencing.

Exome sequencing is the complete spelling out of the genetic code for DNA segments within the genome that serves as the blueprints for proteins. This has become possible to perform quickly and accurately in recent years, thanks to the continual refinement of technology that can sequence DNA strands that are thousands of nucleotide building blocks long, often in a massively parallel manner that helps ensure accurate results. Exome sequencing can identify even the smallest mutations, such as a change in a single building-block nucleotide base pair.

Importantly, many of the disorders identified in the study have not previously been reported in association with NIHF, so the findings broaden knowledge of genetic diseases that can present with the condition. Among the most common of 37 genetic disorders identified in the NEJM study were 11 cases affecting a key intracellular signaling pathway called RAS-MAPK, four cases of inborn errors of metabolism, four cases of musculoskeletal disorders, and three cases each of lymphatic, neurodevelopmental, cardiovascular and blood disorders. Many of these diagnoses would also have been missed by commercial gene panels, Sparks said.

Most mutations identified in the study newly arose in the fetus, but several were inherited, with the potential to affect future pregnancies with the same biologic mother or father.

There is a very wide range in genetic diagnoses underlying NIHF, and identifying the diagnosis is essential for families and healthcare providers, Sparks said. With advanced genetic testing, there is much more we can discover for families to help them understand the situation, for obstetricians and neonatologists to better take care of the pregnancy and anticipate the needs of the newborn, and ultimately to guide the development of novel prenatal management strategies such as in-utero therapies to improve health outcomes over the long term.

For some of the genetic disorders identified in the study, prenatal interventions that can improve or save lives already have been identified. For example, genetic causes of anemia in the fetus may be closely monitored, and the fetus may receive a blood transfusion if needed.

Similarly, for some of the inborn errors of metabolism identified in the study, enzyme therapies already are available after birth. Early diagnosis and treatment of these metabolic disorders leads to better outcomes. A co-author of the NEJM study, Tippi MacKenzie, MD, a professor with the UCSF Department of Surgery, is investigating in utero treatments for specific genetic disorders underlying NIHF in a new clinical trial. Sparks, Norton, and co-authors are also pursuing further investigations to identify additional genomic abnormalities underlying NIHF for the cases that remain unsolved.

Co-Authors: All co-authors of the NEJM study are affiliated with the University of California FetalMaternal Consortium or the UCSF Center for Maternal-Fetal Precision Medicine. Additional UCSF co-authors of the study include Billie Lianoglou, Sarah Downum, Sachi Patel, Amanda Faubel, Anne Slavotinek, Patrick Devine, Ugur Hodoglugil, Jessica Van Ziffle, and Stephan Sanders.

Funding: The study was funded by the UCSF Center for Maternal-Fetal Precision Medicine, the Fetal Health Foundation, the Brianna Marie Foundation, Ultragenyx, and the National Institutes of Health.

The University of California, San Francisco (UCSF) is exclusively focused on the health sciences and is dedicated to promoting health worldwide through advanced biomedical research, graduate-level education in the life sciences and health professions, and excellence in patient care.UCSF Health, which serves as UCSFs primary academic medical center, includes top-ranked specialty hospitals and other clinical programs, and has affiliations throughout the Bay Area.

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DNA Test Identifies Genetic Causes of Severe Fetal and Newborn Illness - UCSF News Services

Recommendation and review posted by Bethany Smith

Name: Carol Keton Parsons

Age: 79

Hometown: Born in Manchester-by-the-Sea, grew up and currently lives in Gloucester

Background: Married with three daughters, graduated from Gloucester High School and took psychology courses at North Shore Community College in Danvers, grew up at Hillcrest Nursing Home as her parents owned the facility, is a Daughter of the American Revolution, loves the local museums and library.

Favorite childhood memory: My grandmother lived on Centennial Avenue, and she would make homemade old-fashioned doughnuts. I can still see her standing there making doughnuts.

Experience with breast cancer: When Parsons felt a lump in her breast while taking a shower at the age of 69, she knew something was not right. That same week, she went to a cancer clinic, where an ultrasound confirmed her fear. The doctors diagnosed her with invasive ductal triple-negative breast cancer.

All breast cancer is serious, but they said this was an aggressive kind of cancer where you dont live very long, Parsons said.

A doctor in Gloucester suggested that Parsons pursue chemotherapy, which she ended up doing every other day for three weeks.

After finishing up her chemo treatment, Parsons was given radiation treatment at Massachusetts General Hospital and had a lumpectomy to remove the cancer from her breasts.

The Gloucester resident is now 10 years cancer-free.

What she wants people to know: First, older women should get tested. A lot of women who are my age dont think they can get it, she said.

Secondly, go to the support meetings for those who have breast cancer. For Parsons, the bond with other women who have or had breast cancer is a strong one. When she would see other women in head scarves, she would go up and hug them.

When you have breast cancer, it is like a bond, she said. Everyone hugs everyone.

What Is triple-negative breast cancer?

Triple-negative breast cancer is a kind of breast cancer that does not have any of the receptors that are commonly found in breast cancer.

Think of cancer cells as a house. The front door may have three kinds of locks, called receptors.

One is for the female hormone estrogen.

One is for the female hormone progesterone.

One is a protein called human epidermal growth factor (HER2).

If an individuals cancer has any of these three locks, doctors have a few keys (like hormone therapy or other drugs) they can use to help destroy the cancer cells.

But for those people who have triple-negative breast cancer, it means those three locks arent there. So the keys doctors usually use wont work. But chemotherapy is still an effective option.

Often, patients first need to have the lump removed (a lumpectomy) or the entire breast removed (a mastectomy). Then, they have chemotherapy treatments to target any cancer cells that cant be seen cells remaining in the breast or that may have spread into other parts of the body. Sometimes doctors recommend chemotherapy before surgery to shrink the cancer.

Source: Centers for Disease Control and Prevention

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Breast Cancer Awareness 2020: Carol Keton Parsons: Testing and support are key - The Salem News

Recommendation and review posted by Bethany Smith

Girls Night Out is an annual breast cancer education event for women hosted by Fayette County Memorial Hospital (FCMH) and Tanger Outlets Jeffersonville, and recently a previous honoree and breast cancer thriver, Dianna Hawes, shared her story.

Although a local breast cancer fighter or survivor is honored each year, this year the event was unable to occur due to the pandemic. Dianna was honored in 2018. All attendees that year released pink balloons to lift Dianna up in her journey.

Diannas story was shared with the R-H from FCMH as a testimony to the impact Girls Night Out has on its honorees and the relationships and support groups that are built out of the event.

In addition to the impacts the event has on the honorees and relationships between participants, it includes breast cancer education. Participants in the Girls Night Out can visit displays from local businesses and get health education information from FCMH and FCMH partners.

Diannas story:

My name is Dianna Hawes, and I am a breast cancer thriver.

Theres a lot of discussion around being called survivors versus thrivers. I personally prefer the latter. Why? Because cancer has not defined me because cancer is lowercase in my life because cancer was a valley to be crossed and not a mountain range to be abandoned.

But how do I use this aggressive adjective so confidently? Because of the broad support of both friends, family and strangers.

You see, I had made a big decision the day before the 2018 Girls Night Out Breast Cancer event. A decision that was met with great opposition from my husband and close friends.

I had decided that after three chemo treatments, I was done.

It was too much, I said.

I knew God was present in the deepest part of the valleys, but I felt weak and defeated.

In May of 2018, I had my annual mammogram. Having hardly ever gone to a doctor for any reason, I was stopped in my tracks when I was told I needed to return for further testing. Next was the biopsy, and at last, the diagnosis. Triple positive breast cancer rare and aggressive.

A very small tumor probably not in lymph nodes, along my chest wall and couldnt be felt.

Choices had to be made. Lumpectomy or mastectomy? Keep surgeon or get another opinion? Genetic testing or take a big guess?

After genetic testing showed only one possible issue and three second opinions, a lumpectomy it was. I was told I probably would just need radiation afterwards.

But when my best friend greeted me as I awoke from surgery, I knew things had changed. There were cancer cells in my lymph nodes the game changer.

Four types of chemotherapy every three weeks, then six weeks of radiation. So again, another very important choice whether or not to put on my big girl panties and do what needed to be done, or to crawl into the nearest hole and deny the inevitable.

I guess, in hindsight, there wasnt much of a choice. My family and friends seemed to take that away from me, and the treatment began late summer.

I continued to work off and on throughout it all, and I found this to be a source of strength of sorts. My co-workers were amazing in their support.

Sometimes it appeared those around me were fighting harder than I was for myself. Chemo places you in a very wonky place.

People would ask how I was feeling. The only word that made sense was wonky. During the lowest days and nights, I could do nothing but whisper the name of Jesus. But this was enough. His presence was overwhelming, and I pursued the goal.

But after the third treatment and stuff going on in the family, I lost my steam my will. But I had been given the incredible opportunity to be honored at the Girls Night Out event. So I put my big girl panties back on and went. Little did I know, it was a God appointment that He scheduled and planned.

During the event, one stranger after another approached me. Most were thrivers, some of 2 years and others as much as 32 years.

They were not only encouraging me to thrive, they were also offering to drive me to treatments, to provide my family with meals, to share hats with me and more. Strangers yesterday, sister thrivers that day.

You see, I had to keep going. I had to do it for me, for them, for those who would follow me. I had to do more than survive. I HAD TO THRIVE and live life abundantly.

That event was the real game changer, and I will always be grateful for those who chose me to be honored.

Dianna was not the first nor will she be the last to be diagnosed with breast cancer in Fayette County. Breast cancer is the second most prevalent type of cancer seen in Fayette County. Nationwide, one in eight women will be diagnosed with breast cancer in her lifetime.

Likewise, it is the second most prevalent type of cancer that is being treated at the FCMH Cancer Care Clinic even though the clinic has only been open about a year.

The clinic is in collaboration with Adena Health System and Dr. Shylaja Mani, and is now able to provide chemotherapy, immunotherapy and hormone therapy. Residents no longer have to leave the county to receive these vital treatments.

Early detection through regular mammograms is key to fighting breast cancer. The National Breast Cancer Foundation reports that 64 percent of cases are diagnosed at a localized stage meaning there is no sign that cancer has spread outside the breast. When this early diagnosis is made, the five-year survival rate is 99 percent.

In conjunction with the FCMH Womens Wellness Center providers Emily Stephens, CNP and Dr. Loliya Idoniboye and the FCMH radiologist Dr. Michael Barrows, the FCMH team is here to support patients from diagnosis through treatment.

Local breast cancer thriver and 2018 Girls Night Out honoree Dianna Hawes.

Dianna Hawes shares her experiences with breast cancer

Read the rest here:
Not just a survivor a 'thriver' - Record Herald

Recommendation and review posted by Bethany Smith

Puma Biotechnology reports positive Neratinib Phase III data

Puma Biotechnology Inc. (PBYI) reported efficacy data for neratinib from the Phase III ExteNET trial. The study involved patients with HER2-positive, hormone receptor-positive (HR+), early-stage breast cancer. The primary endpoint of the trial was invasive disease-free survival. The key secondary endpoint was overall survival. The drug candidate is already approved in the European Union for patients with HR+ breast cancer who initiated treatment within one year of completing an adjuvant trastuzumab-containing regimen.

ExteNET was a multicenter, randomized, double-blind study. It involved 2,840 patients with HER2-positive eBC who were given neratinib after neoadjuvant and/or adjuvant therapy with chemotherapy and trastuzumab. The patients were classified according to their hormone receptor status. The patients were then randomized to be administered either oral neratinib 240 mg/day or placebo for a year.

The data showed that for the HR+ /< 1-year patient population, the absolute 5-year invasive disease-free survival benefit versus placebo was 5.1 percent. The absolute 8-year overall survival benefit was reported to be 2.1 percent. The 5-year cumulative incidence of CNS metastases was at 0.7 percent in the neratinib arm and 2.1 percent in the placebo arm. For the HR+/ <1 year, no pCR subgroup of patients that were at a high risk of disease recurrence, the absolute 5-year iDFS benefit in the neratinib cohort versus placebo was found to be 7.4 percent.

Professor Arlene Chan, Vice Chair Breast Cancer Research Centre - WA, said, This newly published study provides consistent and durable benefits of neratinib in a subset of HER2-positive early stage breast cancer patients who are considered to be at greater risk of relapse: namely patients with HR+ tumors that did not achieve a pCR after neoadjuvant treatment (no pCR). The benefits demonstrated are meaningful in all endpoints evaluated, including iDFS, OS and CNS recurrence, and thus should help guide future clinical decisions.

The data showed that the most common grade 3 adverse events included diarrhea, vomiting and fatigue.

Puma Biotechnology is a biopharmaceutical company. It is mainly engaged in developing and commercializing products in the cancer segment. The company in-licenses the global development and commercialization rights to PB272, the oral neratinib, as well as to PB272 and PB357. PB272 also goes by the name of intravenous neratinib. The oral version of the drug was approved by the FDA for the extended adjuvant treatment of adult patients with early-stage HER2-overexpressed/amplified breast cancer, following adjuvant trastuzumab-based therapy in 2017. It is marketed in the United States market under the brand name of NERLYNX in the tablet form.

NERLYNX was given further approval for treating adult patients with advanced or metastatic HER2-positive breast cancer who have received two or more prior anti-HER2-based regimens in the metastatic setting in February 2020. For this indication, the drug had been approved to be used in combination with capecitabine. The drug has been given marketing authorization by the European Commission for specified conditions.

Investment Thesis: The stock has remained steady in the recent past, making it suitable for even conservative long-term portfolios. The latest news and the upcoming catalysts are expected to provide a positive fillip to the stock price.

Y-mAbs Therapeutics Inc. (YMAB) reported that the FDA has delivered a Refusal to File letter with regard to its Biologics License Application for omburtamab for the treatment of pediatric patients with CNS/leptomeningeal metastasis from neuroblastoma. The BLA was submitted in August, 2020.

The letter states that the FDA requires further details pertaining certain parts of the Chemistry, Manufacturing and Control module and the Clinical module. However, the regulator has not requested or required any additional non-clinical data. The company stated that it is confident about addressing all points raised by the FDA. It also believes that it can provide the requested additional CMC information.

Y-mAbs plans to submit supplementary data from Study 101. This data includes tumor response data from patients with evaluable disease among the first 24 patients included in the protocol. The company intends to request a Type A meeting with the FDA. It is also looking to work with the FDA to make appropriate amendments to the BLA. Y-mAbs intends to file the reworked BLA before the end of this year.

Y-mAbs is a late-stage clinical biopharmaceutical company. It is mainly focused on developing and commercializing novel, antibody-based therapeutic products for treating cancer. The company has robust development pipeline with two pivotal-stage drug candidates. Out of these, naxitamab is designed to target tumors which express GD2, while omburtamab targets tumors expressing B7-H3.

Despite this setback, the company also reported a positive news, as its leading bispecific antibody program nivatrotamab for the treatment of neuroblastoma was granted Rare Pediatric Disease Designation and Orphan Drug Designation by the FDA.

Investment Thesis: The stock is currently trading close to its highs. Coupled with the latest setback, it is advisable to watch this stock for further development and some meaningful pullback in the price.

Halozyme Therapeutics Inc. (HALO) announced that it has expanded its collaboration and licensing agreement with Argenx. The collaboration deals with the companys Enhanze technology, and the original agreement was signed in February 2019.

The new terms of the agreement provide for Argenx to have an exclusive access to Halozymes Enhanze drug delivery technology for three more targets. The company now can name up to six targets under this collaboration. Dr. Helen Torley, president and chief executive officer of Halozyme, Argenx has made rapid progress in the clinic with efgartigimod utilizing ENHANZE since signing the original agreement, moving to a Phase 2 study initiation for an indication being developed only as SC, within just fourteen months.

So far, Argenx has named two targets for this program. These are human neonatal Fc receptor FcRn and complement component C2. The company can access Enhanze for its FcRn antagonist efgartigimod, its late-stage development for several severe autoimmune diseases.

Halozyme is a biopharmaceutical company. Its Enhanze technology is designed to make various treatments more efficient and time-effective. The technology is based on its proprietary enzyme, rHuPH20. It is mainly used for effectual delivery of injected drugs and fluids with the aim of reducing overall treatment burden. The company has licensed this technology to a number of major biotech and pharma companies, including Alexion (ALXN), Lilly (LLY), Janssen (JNJ) and Pfizer (PFE), among others. Its revenue stream consists of royalties and milestone payments received from such collaborations.

Investment Thesis: The company has strong potential ahead with its robust collaboration framework. However, the stock is currently trading at highs and may see some correction in the near future, providing an opportunity to build a position.

Thanks for reading. At the Total Pharma Tracker, we do more than follow biotech news. Using our IOMachine, our team of analysts work to be ahead of the curve.

That means that when the catalyst comes that will make or break a stock, weve positioned ourselves for success. And we share that positioning and all the analysis behind it with our members.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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Puma's Positive Neratinib Data, And Other News: The Good, Bad Ugly Of Biopharma - Seeking Alpha

Recommendation and review posted by Bethany Smith

The eatery specializes in double-fried chicken wing dishes served with a variety of sauces, including buffalo, mango habanero and garlic Parmesan, among others. (Courtesy WingNuts Express)

Read the latest business and community news from the Houston area.

Tomball-Magnolia

WingNuts Express now open on Tamina Road in Magnolia

Magnolia's latest wing eatery, WingNuts Express, opened on Tamina Road on Oct. 3, according to a Facebook post from the restaurant.

Sugar Land-Missouri City

Child Advocates of Fort Bend, partners open clinic for child victims of sexual abuse

Child Advocates of Fort Bend, along with AccessHealth and the Harris Health System, opened a medical clinic dedicated to child victims of sexual abuse Oct. 1, according to a CAFB press release.

Spring-Klein

ROUNDUP: 8 businesses, restaurants that recently opened in Spring, Klein

Twisted Sisters Nutrition Shack, Fajita Pete's and Cavender's Boot City are just a few of the businesses that have recently opened in the Spring and Klein area.

Wingstop now open on Spring Cypress Road

Wingstop opened a new location at 8675 Spring Cypress Road, Ste. 37B, Spring, on Oct. 8. Known for its 11 different flavors of classic wings, boneless wings and crispy tenders, the new location offers carryout and delivery services from 10:30 a.m.-midnight daily.

Cy-Fair

A Thousand Oaks Events & Retreats venue opening this month in Cypress

A new wedding and event venue, A Thousand Oaks Events & Retreats, is holding an open house Oct. 22 from 4-8 p.m. at 17011 Steinhagen Road, Cypress, to celebrate its grand opening.

Trattoria Pizza & Pasta offers Italian classics, family recipes on Cypresswood Drive

Elderin Berisha said his primary goal at Trattoria Pizza & Pasta is making customers feel at home.

If you come here on a Friday or a Saturday night, youll see all the customers know us, he said. We know their names; we talk to them; we sit with them. Its like a family. I want to try to make you comfortable when you come here. We know what you order, and we pay attention to the details.

Millennium Physicians opens new medical office in Cypress

Officials with Millennium Physicians announced the opening of a new office offering medical oncology and hematology, rheumatology and infusion services.

Heights-River Oaks-Montrose

Houston leaders react to death of transgender advocate Monica Roberts

Local leaders have joined human rights and LGBTQ organizations across the nation reacting to the loss of one of Houston's leading transgender rights advocates, Monica Roberts, who died this week.

DACAMERA chamber and jazz presenters announce virtual performance schedule

Chamber and jazz performance organizer DACAMERA announced its fall schedule Oct. 6, including a new collaboration with The Menil Collection.

Robot Noodle takes over Blackbird Izakaya spot in the Heights

Robot Noodle, a new restaurant by Delicious Concepts Group, has opened at 1221 W. 11th St., Houston, taking over the former space of Blackbird Izakaya, the group announced Oct. 7.

Pearland-Friendswood

Pearland's StrongFound Personal Training stays successful pivoting during pandemic

StrongFound Personal Training sets itself apart with its target demographic: those who are 40 years old or older.

Lake Houston-Humble-Kingwood

Millennium Physicians Internal Medicine group opens new Kingwood office

Millennium Physicians Internal Medicine group opened a new office July 15 at 22698 Professional Drive, Ste. 100, Kingwood. Internal medicine physicians Dr. Salvador Recio and Dr. Carmen Perez will be offering a variety of services at this location, including weight-loss assistance, diabetes management, well women exams, allergy testing and hormone pellet replacement.

Kelly Schafler, Emma Whalen, Adriana Rezal, Claire Shoop, Hannah Zedaker, Danica Lloyd, Matt Dulin and Haley Morrison contributed to this report.

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WingNuts Express opens in Magnolia and more Houston-area news - Community Impact Newspaper

Recommendation and review posted by Bethany Smith

One upside of the COVID-19 disruption is that many people have a bit more time if only because theyre not commuting, looking for a parking space, going for after-work drinks, etc. Ive been encouraging my students to pick up a fun new activity to make good use of this time bonus. Ive chosen to try to learn to draw and am making a start on it. Well, at least Ive bought some instructional books and supplies. Wish me luck!

One activity that we could all benefit from is paying attention to our long-term health. After all, youre far more likely to die from cancer or heart disease than from COVID-19. There are some concrete steps you can take to improve your long term health prospects. If youve been putting off a visit to your doctor for a physical, this might be a good time, even if its a telehealth visit. Some procedures like that dreaded prostate exam do require the hands-on approach. But youd be amazed at how much you and your doctor can sort out over the phone and with lab tests.

To see whats included in a good physical, have a look at the Preventive Care Checklist on the website of the College of Family Physicians of Canada (cfp.ca). It was recently updated, and I learned a lot from it, including that at my age I should be getting the pneumococcal 23-valent polysaccharide vaccine.

Discoveries also highlight the importance of knowing more about our genetic heritage. In a first-of-its-kind genomic study, researchers at Vanderbilt University Medical Center led by William D. Dupont and Joan P. Breyer studied prostate cancer patients with family histories of the disease. They note that prostate cancer heritability is twice that of breast cancer, at roughly 58 per cent. Their latest research focused on the detailed genetic makeup of chromosome 8, which is known to be related to prostate cancer. The Vanderbilt scientists found three mutations that helped protect us from prostate cancer, and four that raised the danger, in one case by 22-fold.

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Keenan: Thinking well beyond COVID-19 - Edmonton Journal

Recommendation and review posted by Bethany Smith

Named Cardamine inseuta, was first spotted in 1972 in the Urnerboden region after the land in the area went from being a forest to a grassland. Now, researchers at the University of Zurich (UZH) have narrowed down the two species of the plants that merged to create the hybrid that is thriving.

One is Cardamine amara, which grows in and around streams and the other is Cardamine rivularis, which inhabits moist, not wet, areas.

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It is the combination of genetic traits from its parents that enabled the new species to grow in a district environmental niche, said Rei Shimizu-Inatsugi, co-author of the study published in Frontiers in Genetics.

The best of both worldsC. inseuta is a so-called triploid plant. It means that it has three sets of chromosomes. Two come from C. rivularis and one set from C. amara.

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From its other parent, C. amara, the new species inherited the trait of submergence tolerance. That means that even if surrounded by water, C. inseuta would survive rather than drown.

Depending on the environmental situation, the plant activates a different set of genes it inherited from its two parent species, said Shimizu-Inatsugi.

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Andrea Ghez is the fourth woman to win the Nobel Prize for Physics found the first signs of the supermassive black hole at the center of the Milky Way

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Scientists unravel the mystery behind new plant species found in the Swiss Alps, which only took 150 years to - Business Insider India

Recommendation and review posted by Bethany Smith

Scribe Therapeutics, a biotech firm focused on developing next-generation gene-editing technology, has raised $20 million in its first major round of financing, backed by Andreessen Horowitz. The firm separately unveiled a deal with Biogen to develop CRISPR-based treatments for amyotrophic lateral sclerosis (ALS).

Scribe was cofounded in 2018 by several University of California, Berkeley, scientists, including gene-editing pioneer Jennifer Doudna and protein engineer Benjamin Oakes, who at the time was an entrepreneurial fellow at the Innovative Genomics Institute, where Doudna is president. Their goal was to engineer a newly discovered class of Cas proteins to make them behave better as therapies than the original CRISPR-Cas9 gene-editing system.

The original system was found in bacteria, which use it to recognize and chop up DNA from invading pathogens. Scientists, including Doudna, quickly realized the system could be co-opted to make precise cuts to human DNA. The tool set off a race among companies trying to use it to address the genetic mutations underlying many diseases.

But even with its promise, the CRISPR-Cas9 system comes with evolutionary baggage, Oakes, who is now CEO of Scribe, says. Those systems arent designed to work within the context of the human cell or even the human genome, he says, complicating efforts to turn the technology into drugs.

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Scribe Therapeutics launches to explore next-generation CRISPR technology - Chemical & Engineering News

Recommendation and review posted by Bethany Smith

Recovery from experimental heart attacks can be improved with an injection of a mixture of heart muscle cells, endothelial cells and smooth muscle cells, but results are limited by poor engraftment and retention, plus there are concerns about potential tumorigenesis and heart arrhythmia.

Recent animal research in pigs has shown that using the exosomes naturally produced from a mixture of heart muscle cells, endothelial cells, and smooth muscle cells derived from human induced pluripotent stem cells yielded regenerative benefits that were the equivalent to the injected hiPSC-CCs.

Exosomes are membrane-bound extracellular vesicles that contain biologically active proteins, RNAs and microRNAs that are well known to participate in cell to cell communication, and are actively studied as potential clinical therapies for a wide range of conditions.

The hiPSC-CC exosomes are acellular and, consequently, may enable physicians to exploit the cardioprotective and reparative properties of hiPSC-derived cells while avoiding the complexities associated with tumorigenic risks, cell storage, transportation and immune rejection, said Ling Gao, Ph.D., and Jianyi Jay Zhang, M.D., Ph.D., University of Alabama at Birmingham corresponding authors of the study, published in Science Translational Medicine. Thus, exosomes secreted by hiPSC-derived cardiac cells improved myocardial recovery without increasing the frequency of arrhythmogenic complications and may provide an acellular therapeutic option for myocardial injury.

Studies involving large animals are required to identify, characterize and quantify all responses to potential treatments, prior to this study the feasibility of hiPSC-CC exosomes for cariad therapy had only been shown to be effective in mouse models and in vitro work.

The UAB studies involving juvenile pigs with experimental heart attacks had 1 of 3 treatments injected into the damaged myocardium: a mixture of cardiomyocytes, endothelial cells, and smooth muscle cells derived from human induced pluripotent stems cells, exosomes extracted from three cell types, and homogenized fragments from the cell types.

There were 2 primary findings from this study. Measurements of left ventricle function, infarct size, wall stress, cardiac hypertrophy apoptosis and angiogenesis in the animals treated with hiPSC-CCS, hiPSC-cc fragments or hiPSC-cc exosomes were found to be similar and significantly improved compared to those that recovered without any of the 3 treatments. Additionally, exosome therapy was found not to increase the frequency of arrhythmia.

During experiments with cells or aortic rings that were grown in culture, exosomes produced by hiPSC-CCs were found to promote blood vessel growth in cultured endothelial cells and isolated aortic rings. The exosomes also protected the cultured hiPSC-cardiomyocytes from the cytotoxic effect of serum-free lox oxygen media by reducing the programmed apoptosis cell death and by maintaining intracellular calcium homeostasis which had a direct beneficial effect on heart conductivity. Additionally, the exosomes also increased cellular ATP content which is beneficial as deficiencies in cellular ATP metabolism are believed to contribute to the progressive decline in heart function in those with left ventricle hypertrophy and heart failure.

Some of the in vitro beneficial effects were found to also be mediated by synthetic mimics of the 15 most abundant microRNAs that were found in the hiPSC-cc exosomes. It was noted that knowledge of the potential role of microRNAs in clinical application requires more research as it is far from complete.

The study: Exosomes secreted by hiPSC-derived cardiac cells improve recovery from myocardial infarction in swine, co-authors with Gao and Zhang are Lu Wang, Yuhua Wei, Prasanna Krishnamurthy, Gregory P. Walcott and Philippe Menasch, UAB Department of Biomedical Engineering. Menasch also has an appointment at the Universit de Paris, France. Gao is now at Tongji University School of Medicine, Shanghai, China.

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Preclinical Study Shows Improvement In Recovery From Heart Attack With Exosomes - Anti Aging News

Recommendation and review posted by Bethany Smith

Autologous Stem Cell Based Therapies Market Report Delivering Growth Analysis with Key Trends of Top Companies (2020-2026)

A comprehensive research study on the Autologous Stem Cell Based Therapies Marketwas recently published by Market Report Expert. This is an up-to-date report, covering the current COVID-19 impact on the market. The Coronavirus (COVID-19) has affected every aspect of life globally and thus altering the global market scenario. The changes in the market conditions are drastic. The swiftly changing market scenario and initial and future assessment of the impact on Autologous Stem Cell Based Therapies market is covered in the report.The Autologous Stem Cell Based Therapies Market report is a precise and deep-dive study on the current state that aims at the major drivers, market strategies, and imposing growth of the key players. Worldwide Autologous Stem Cell Based Therapies Industry also offers a granular study of the dynamics, segmentation, revenue, share forecasts, and allows you to make superior business decisions. The report serves imperative statistics on the market stature of the prominent manufacturers and is an important source of guidance and advice for companies and individuals involved in the Autologous Stem Cell Based Therapies industry.

The Global Autologous Stem Cell Based Therapies Market poised to grow from US$ XX million in 2020 to US$ XX million by 2026 at a compound annual growth rate (CAGR) of XX% during the projection period of 2020-2026.

An Outline of the Major Key Players covered in this Report:

Regeneus, Mesoblast, Pluristem Therapeutics Inc, U.S. STEM CELL, INC., Brainstorm Cell Therapeutics, Tigenix, Med cell Europe

Get Free LatestPDF Template of this Report(Including Covid-19 impact analysis on overall industry Forecast, Size, Share, CAGR and more.)@ https://www.marketreportexpert.com/report/Autologous_Stem_Cell_Based_Therapies_/13849/sample

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The report puts together a succinct analysis of the growth drivers influencing the current business scenario across various regions and countries. Substantial information pertaining to the industry analysis size, share, application, and statistics are summed in the report in order to present a collaborative prediction. Additionally, this report encompasses a precise competitive analysis of major market players, innovative companies, and their strategies during the projection timeline.

The latest report on the Autologous Stem Cell Based Therapies Market consists of an analysis of this industry and its type, application, and other segments. As per the report, the market is estimated to gain significant returns and register substantial y-o-y growth during the forecast period of 2020-2026.

Majortype, primarily split into

Embryonic Stem CellResident Cardiac Stem CellsUmbilical Cord Blood Stem Cells

Major applications/end users, including

Neurodegenerative DisordersAutoimmune DiseasesCardiovascular Diseases

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The research offers an analysis of the geographical landscape of the Autologous Stem Cell Based Therapies Market, which is divided into regions such as North America, Europe, Asia Pacific, South America, and the Middle East & Africa. The segment includes data about several parameters related to the regional contribution such as market share, application share, type share, key companies in respective regions, market share of key companies in regional market, growth rate and revenue of the regional market, sales, production, and consumption of the respective Autologous Stem Cell Based Therapies market.

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Information related to the growth rate, revenue, sales, production, consumption, during the forecast period is included in the report. The Autologous Stem Cell Based Therapies Market report claims that the industry is projected to generate significant revenue and sales during the forecast period. The report consists of information related to the market dynamics such as challenges involved in this vertical, growth opportunities, and driving factors affecting the market.

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Market Report Expert is a futuristic market intelligence company, helping customers flourish their business strategies and make better decisions using actionable intelligence. With transparent information pool, we meet clients objectives, commitments on high standard and targeting possible prospects for SWOT analysis and market research reports.

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Autologous Stem Cell Based Therapies Market Size, Business Revenue Forecast, Leading Competitors And Growth Trends 2026| Regeneus, Mesoblast,...

Recommendation and review posted by Bethany Smith

The University of Manchester, part of the prestigious Russell Group of universities, has announced today a groundbreaking gene therapy partnership to ease the lifelong suffering of people with Hunter syndrome.

The University has agreed to a worldwide license and collaborative research funding agreement with AVROBIO, Inc., a leading clinical-stage gene therapy company with a mission to free people from a lifetime of genetic disease, based in Cambridge, Massachusetts, USA.

The significant partnership agreement is for the clinical development of an investigational lentiviral gene therapy for mucopolysaccharidosis type II (MPS II), or Hunter syndrome, a rare and deadly lysosomal disorder that primarily affects young boys.

Hunter syndrome, which affects an estimated one in 100,000 males worldwide, causes devastating complications throughout the body and brain, including severe cardiac and respiratory dysfunction, skeletal malformations and hearing impairment. Children with severe cases of Hunter syndrome typically show early symptoms in their toddler years and begin to regress developmentally around age six, losing basic motor skills and cognitive function.

The current standard of care is weekly enzyme replacement therapy (ERT), which can delay some complications but does not halt overall progression of the disease and has not been demonstrated to address cognitive issues. Even with ERT, people with Hunter syndrome face life-limiting symptoms and a significantly reduced life span.

The University of Manchester will sponsor the investigator-led Phase 1-2 clinical trial for Hunter syndrome which is expected to begin in 2021. The Hunter syndrome program was developed by Brian Bigger, a professor of cell and gene therapy at The University of Manchester. Professor Bigger has published preclinical data demonstrating that the introduction of the transgene with an optimised, proprietary tag has the ability to correct peripheral disease and normalise brain pathology.

Primary investigators for the clinical trial will be; Professor Robert Wynn, Consultant Paediatric Hematologist at the Royal Manchester Childrens Hospital and Dr. Simon Jones, Consultant Paediatric Physician for inherited metabolic diseases at the Willink Unit, Saint Marys Hospital and the Manchester Centre for Genomic Medicine.

We feel an enormous urgency to bring forward a treatment that may halt this deadly disease in its tracks, before symptoms emerge and before children lose their physical and cognitive skills, said Professor Bigger. We are delighted to be working with AVROBIO on this program. Both of our teams have deep experience running international clinical trials in other lysosomal disorders. AVROBIO also has a leading gene therapy platform, plato, which is designed to optimise the consistency, predictability and efficacy of its gene therapies and to enable efficient scaling for worldwide commercialization. By working together, we believe we can greatly accelerate development of this important program.

The investigational gene therapy, which will be called AVR-RD-05, involves ex vivo transduction of the patients own hematopoietic stem cells with a therapeutic transgene designed to express functional enzyme the patient needs to maintain cellular health, coupled to a proprietary protein tag that is designed to improve stability of the enzyme in the bloodstream and facilitate uptake by tissues from head to toe. When reinfused into the patient, the gene-modified stem cells are expected to engraft in the bone marrow and produce generations of daughter cells, each carrying the transgene. Those daughter cells are then expected to differentiate into macrophages, microglia and other components of the immune system and circulate throughout the body and central nervous system, potentially enabling widespread distribution of functional enzyme.

Geoff MacKay, AVROBIOs president and CEO said: The lentiviral gene therapy approach is well suited to treat a progressive and pervasive disease such as Hunter syndrome, which affects organs throughout the body and severely impairs cognitive function. If we treat children early, before their symptoms arise, we hope to prevent the tragic complications that rob these young children of their futures.

We believe our deep experience with investigational gene therapies for lysosomal disorders will enable us to efficiently move the program through clinical development in collaboration with Professor Brian Bigger, who has done tremendous work to develop and optimize this investigational gene therapy. Were proud to add this program to our leading lysosomal disorder pipeline and excited about its potential to change the lives of patients and families living with Hunter syndrome.

The University of Manchesters technology transfer office, The University of Manchester Innovation Factory and AVROBIO have negotiated the exclusive, worldwide license to the technology. Under the terms of the license, AVROBIO will pay The University of Manchester an upfront cash payment and additional payments based on the achievement of development and regulatory milestones. The company will pay The University a mid-single digit percentage royalty on annual net sales of licensed products. Additionally, under the collaborative research funding agreement, AVROBIO will cover budgeted clinical trial costs.

Andrew Wilkinson, CEO of the Universitys technology transfer company, The University of Manchester Innovation Factory said: We are delighted that AVROBIO will be working with teams from The University of Manchester and The University of Manchester Foundation Trust to develop a therapy for this debilitating genetic disease. AVROBIOs strategic focus on bringing new personalised gene therapies to the world along with their technical and commercial expertise in this area make them an excellent partner for the investigational Hunter syndrome gene therapy programme.

About Hunter syndrome

Hunter syndrome, also known as mucopolysaccharidosis type II (MPS II), is a lysosomal disorder caused by a mutation in the IDS gene that leads to a deficiency of the lysosomal enzyme iduronate-2-sulfatase (IDS), which is essential for breaking down large sugar molecules called glycosaminoglycans (GAGs, also known as mucopolysaccharides). Without functional IDS, toxic levels of GAGs build up throughout the body and central nervous system, causing a wide range of symptoms including cognitive decline and cardiac and respiratory dysfunction. The current standard of care is weekly enzyme replacement therapy, which may delay some symptoms but does not halt the overall progression of disease and does not cross the blood-brain barrier, an intricate web of protective tissue that selectively prevents macromolecules from entering the brain. Even with treatment, people with Hunter syndrome face life-limiting symptoms and a significantly reduced life span. The disorder affects an estimated 1 in 100,000 males worldwide; about two-thirds of cases have an early, severe progressive form.

About lentiviral gene therapy

Lentiviral vectors are differentiated from other delivery mechanisms because of their large cargo capacity and their ability to integrate the therapeutic gene directly into the patients chromosomes. This integration is designed to maintain the therapeutic genes presence as the patients cells divide, which potentially enables dosing of pediatric patients, whose cells divide rapidly as they grow. Because the therapeutic gene is integrated using the vector into patients own stem cells, patients are not excluded from receiving the investigational therapy due to pre-existing antibodies to the viral vector.

About The University of Manchester

The University of Manchester is a member of the prestigious Russell Group and one of the UKs largest single-site universities.

We have over 40,000 students, 12,000 staff and, with almost 480,000 former students from more than 190 countries, are home to the largest alumni community of any campus-based university in the UK. No fewer than 25 Nobel laureates have either worked or studied here.

We are thetop UK University for graduate employabilityaccording to The Times and Sunday Times Good University Guide; ranked 27th in the world in the QS World University Rankings (2020) and 6th in the UK. Were also listed as 8th in Reuters Top 100: Europe's most innovative universities (2019).

Visit http://www.manchester.ac.uk for further information or https://www.manchester.ac.uk/discover/vision/ for our latest strategic vision.

Link:
University of Manchester announces partnership with AVROBIO for Hunter syndrome gene therapy - PharmiWeb.com

Recommendation and review posted by Bethany Smith

David Z Wang and colleagues look at the latest advances in brain research and how they might affect treatment of brain disorders

The world has come a long way in solving the mystery of the brain, understanding its fundamental role in human consciousness and discovering methods to treat its disorders. In The Sacred Disease in ~430 BC, Hippocrates wrote that the brain served to house the ventricles, whose main purpose was to be a container and transit point for the breath or air (pneuma) from outside the bodythe force that brought to life our joys, pleasures, laughter, and grief. Thus, the brain was a reservoir for an animated substance that produced the human experience of consciousness and personality rather than the source of that activity itself.1 Our knowledge of the brain and its functional complexity remained at the level of three ventricles where our soul lies (Nemesius, da Vinci) for hundreds of years until modern neuroscience began to uncover the fine network of neuronal circuits that made up the solid substance of the brain.

With the advent of modern neuroimaging, the complex structure of the brain has been brilliantly revealed, and this has helped greatly in the treatment of many brain related disorders. Other articles in this series have provided updates on a wide range of topics, including neurodegenerative diseases, mental disorders, cerebrovascular diseases, epilepsy, monogenic neurological diseases, and in vivo brain function testing.23456 With help from gross anatomy to electronic microscopy, tissue staining to profiling, cell physiology, and synaptic chemistry, neuroscientists have elucidated the mechanisms and pathophysiology of many common brain diseases. For example, trinucleotide repeat expansion is now known to be responsible for many genetically inherited degenerative diseases such as Huntingtons disease, and amyloid precursor gene or presenilin gene mutations can cause Alzheimers disease.

On the other hand, despite centuries of discovery on mechanisms of brain disease, treatment options remain limited. Most treatments still provide only alleviation of symptoms, though recent breakthroughs in gene therapy such as onasemnogene abeparvovec-xioi to treat children with spinal muscular atrophy 7 and reperfusion therapy for acute ischaemic stroke hold the promise to truly revolutionise treatment for neurological disease. While options are available to modify disease expression with medicationssuch as in the treatment of Parkinsons disease, multiple sclerosis, and epilepsywe are far from curing them.

Entering the 21st century, perhaps we now have better ways to understand the mechanism of those brain disorders that are still a mystery and find the precise treatment. The key will likely be interdisciplinary research. Many ongoing brain health research programmes have already been multidimensional, combining neurobiology, physics, engineering, big data science, and artificial intelligence.

In the future, it is likely that humans will be able to live longer, and do so with augmented capabilities supported by machine-human interactions. One exciting advance is new ways of observing in vivo brain-wide activities at the cellular level. A real time, ultra-large scale, high resolution (RUSH) macroscope has recently been developed that can provide video-rate gigapixel imaging of biological dynamics at centimetre scale and micrometre resolution, with a data throughput of up to 5.1gigapixels a second.8 RUSH has enabled in vivo functional imaging of neural networks across the whole mouse brain at single dendrite resolution and brain-wide tracking of leucocytes during pathological processes, and the technology opens up a new horizon for large scale brain imaging to study various brain diseases at a systematic level.8

Another example is the better understanding of the precise number of brain cells needed to complete a particular task. By constructing an explicit model of face selective cells that could decode an arbitrary realistic face from face cell responses and predict the firing of cells in response to an arbitrary realistic face, Chao and colleagues identified that macaques require only 200 cells to remember a face.9 These findings have far reaching significance. For the first time, a specialised task of the brain can be attributed to a specific number and type of brain cells in a specific circuit. This may allow scientists to build artificial models of explicit brain functions and experiment with mechanisms of injury and repair at a cellular or molecular level. Such mapping may aid our understanding of brain function and recovery and guide the rebuilding of brain circuits or resection of dysfunctional brain cells rather than whole tissues. It may also help us pinpoint the cells and circuits that are responsible for addictive behaviours, from smoking to substance use disorders to gambling.

The common belief is that when a brain has been removed, brain death is imminent. However, such belief has recently been shattered. Sestan and colleagues collected brains of 6-8 month old pigs four hours after death and bathed them in specialised perfusate solutions. They found that brain cells and synapses of certain areas of brain began to recover and show signs of cellular activities.10 Their finding suggests that there may be a late window of treatment after onset of brain anoxia when brain tissue can recover, analogous to the benefit of late window thrombectomy. This discovery has taught us that brain cells can survive and recover after loss of circulation, and that favourable conditions may preserve a reservoir of resilient brain cells that are slow progressors to ischaemic necrosis.

Evidence is also emerging on how brain cells can adapt. A recent report of functional neuronal connectivity in adults without apparent loss of function after brain hemispherectomy sheds new light on brain plasticity. The study provides the first comprehensive analysis of whole brain functional connectivity across the full repertoire of resting state networks after hemispherectomy and shows preservation of resting state networks but an increase in internetwork connectivity with other functional brain networks. When hemispheric resection occurred in patients younger than 11, the retained hemisphere was able to protect the jeopardised functions by enhancing cellular interaction and synaptic activity.11

Artificial intelligence (AI) has been widely applied in clinical diagnosis and patient monitoring. Recent studies have attempted to classify or detect Alzheimers disease and other cognitive impairment,1213 acute neurological events,1415161718 focus of epilepsy, autism spectrum disorder, and attention deficit/hyperactivity disorder by using deep learning based algorithms. The data in these AI models include not only medical images but also clinical scores, in vitro diagnostic test results, and other functional and structure information.19202122232425 These studies showed high sensitivity and specificity from their test set, and work is ongoing on how to incorporate the routine use of these AI systems into a clinical setting.

The lack of a large dataset from multiple centres, the limited coverage of a disease spectrum, and unclear risk of using AI are major limitations of these blackbox systems. In contrast, Wang and colleagues have recently proposed a vascular aware unsupervised learning technique, VasNet,26 which provides the end users with explainable images, including both vascular structures and multidimensional features such as anatomical, physiological, biochemical, and cellular details. The enriched outputs could augment human decision making on treating vascular diseases and contribute to the emergence of the next generation of healthcare engineering.

The US Food and Drug Administration has already approved several automatic quantitative measurement software systems for disease classification (eg, NeuroQuant, Quantib, RAPID). Brain morphometry analysis software can automatically examine segments of brain tissue and detect minute changes. This technology can help early detection of degenerative brain diseases by comparing the results from individuals with a large dataset and images of healthy people. To take racial differences in the brain into account, some Asian companies have developed software based on datasets acquired from Asian populations (http://quant-health.com). Use of a deep learning based segmentation algorithm could improve the accuracy and test-retest stability in segmenting and measuring the volume of brain structure, abnormal lesions, perfusion deficit area, and other characteristics. The resulting quantified values could be used to assign a clinical score automatically, avoiding the variation arising from subjective measurement and interobserver inconsistency.

AI algorithms can also objectively analyse the data collected from a depth camera or wearable devices, assess behaviour, and evaluate facial expressions.272829 The quantified values produced would not be affected by the physicians experiences, and errors can be avoided since the spatial-temporal resolution of the hardware is much smaller than visual evaluation by humans. Such early detection may allow treatment of a disease before a person shows clinical signs of brain dysfunction. Quantified measurements can be used as biomarkers to monitor the progress of the disease and help evaluate the efficacy of precision therapy.

One of the potential ways of curing a brain disorder is to correct its diseased protein structure. Many neurological diseases are caused by misfolded proteins, including Huntingtons, Parkinsons, and Alzheimers disease. AlphaFold, a Google company, has successfully predicted a protein structure by using large genomic data. The 3D models of proteins that AlphaFold generates are far more accurate than any that have come beforemaking significant progress on one of the core challenges in biology. The ability to predict a proteins shape from its DNA sequence is useful to scientists because it is fundamental to understanding its role within the body, as well as diagnosing and treating diseases believed to be caused by protein misfolding.30

We have entered into an exciting new era of brain science research and discovery. With the advent of AI, advanced imaging, genomics, psychosocial analytics, and protein engineering we may be closer than ever to new precision medicine approaches to treat many brain disorders.

In the past decade, neuroscience and brain research have entered into a new era

It is now possible to understand brain physiology and pathophysiology better through direct and in vivo observation of live brain

In the coming years, artificial intelligence will likely be part of brain science and assist or replace certain brain function

Genetic or protein alterations may provide a cure for many brain disorders in the near future

Contributors and sources: DZW drafted the first manuscript. LHS, TYQ, and QHD critically reviewed and revised the manuscript. DZW is an expert in stroke clinical research. LHS is an expert in neuroscience research and stroke care quality improvement. TYQ is an expert in big data and artificial intelligence. QHD is an expert in brain research and artificial intelligence.

Competing interests: We have read and understood BMJ policy on declaration of interests and declare that we have no competing interest.

Provenance and peer review: Commissioned; externally peer reviewed.

This article is part of a series launched at the Chinese Stroke Association annual conferenceon 10 October 2020,Beijing, China.Open access fees were funded by the National Science and Technology Major Project. The BMJ peer reviewed, edited, and made the decision to publish these articles.

Gupta A, Ayhan M, Maida A. Natural image bases to represent neuroimaging data. Proceedings of 30th international conference on machine learning. Vol 28. Atlanta, GA. 2013:987-94.

Evans R, Jumper J, Kirkpatrick J, et al. De novo structure prediction with deep-learning based scoring. In: Thirteenth critical assessment of techniques for protein structure prediction. Abstracts, 1-4 December 2018.

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Decoding the brain through researchthe future of brain health - The BMJ

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Cancer Gene Therapy Market report offers in-depth analysis of the industry size, share, major segments, and different geographic regions, forecast for the next five years, key market players, and premium industry trends. It also focuses on the key drivers, restraints, opportunities and industry challenges.

Growing prevalence of cancer with rising mortality rates will augment cancer gene therapy industry forecast in the coming years. Cancer therapies incorporated with genetically modified genes ai in blocking the growth of the tumors.

Efficient PCR and isothermal amplification technologies for detecting mutations and CRISPR gene editing tools are some technical developments. These advancements have led to innovations and ensured availability of advanced cancer gene therapies driving the industry trends.

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Estimates have stated that the global cancer gene therapy market value is likely to cross an annual valuation of USD 2.5 billion by 2025.

Technological developments are majorly fueling cancer gene therapy growth. Genetically modified genes which block tumor growth have been incorporated into recent cancer therapies. Efficient isothermal amplification techniques and PCR technology are transforming the way gene mutations are detected. Recent launch of CRISPR gene editing tools is claimed to help enhance the process of gene therapy development. These innovations and advancements in technology are anticipated to propel cancer gene therapy industry size.

The in-vivo segment is estimated to witness about 22% growth over the forecast period owing to its multiple offered benefits. In-vivo gene therapy consists of direct delivery of therapeutic genes into the target cell, a process which has shown effective results in cancer treatment. Viral vectors are delivered using in-vivo gene therapy which help in stopping the activity of tumor inducing genes and has exhibited positive results in clinical trials.

Biopharmaceutical firms held approximately 48% of the industry revenue share in 2018. Major firms such as Roche and Novartis are working on cancer gene therapies that have high adoption rate of both non-viral and viral vectors. These firms are also carrying out clinical trials that are favoring the demand for such vectors, consequently driving market growth.

Broadening awareness regarding the availability of advanced cancer therapies have stimulated the market for cancer gene therapy in China. Government initiatives and funds have encouraged researchers to carrying out extensive R&D activities linked to cancer gene therapy.

Meanwhile, on a global landscape, companies like Vigene Biosciences, Cobra, Uniqure, Sirion Biotech, Bluebird Bio, Caribou, Ziopharm, Finvector, Cellectis and Sarepta Therapeutics are leading the cancer gene therapy market. These firms are focusing on enhancing their market position through business strategies such as product launches, mergers and acquisitions, among others.

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Partial Chapter of the Table of Content

Chapter 4. Cancer Gene Therapy Market, By Type

4.1. Key segment trends

4.2. Ex-vivo

4.2.1. Market size, by region, 2014 2025 (USD Million)

4.3. In-vivo

4.3.1. Market size, by region, 2014 2025 (USD Million)

Chapter 5. Cancer Gene Therapy Market, By Product

5.1. Key segment trends

5.2. Viral vectors

5.2.1. Market size, by region, 2014 2025 (USD Million)

5.2.2. Adenoviruses

5.2.2.1. Market size, by region, 2014 2025 (USD Million)

5.2.3. Lentiviruses

5.2.3.1. Market size, by region, 2014 2025 (USD Million)

5.2.4. Retrovirus

5.2.4.1. Market size, by region, 2014 2025 (USD Million)

5.2.5. Adeno associated virus

5.2.5.1. Market size, by region, 2014 2025 (USD Million)

5.2.6. Herpes simplex virus

5.2.6.1. Market size, by region, 2014 2025 (USD Million)

5.2.7. Vaccinia virus

5.2.7.1. Market size, by region, 2014 2025 (USD Million)

5.2.8. Others

5.2.8.1. Market size, by region, 2014 2025 (USD Million)

5.3. Non-viral vectors

5.3.1. Market size, by region, 2014 2025 (USD Million)

5.4. Others

5.4.1. Market size, by region, 2014 2025 (USD Million)

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Cancer Gene Therapy Market 2020 by industry trends, statistics, key companies growth and regional forecast - News by Decresearch

Recommendation and review posted by Bethany Smith

Strategic Acquisitions Lead to Innovations in Freezers and Bioproduction of Gene Therapies

Companies in the cryopreservation equipment market are focusing on strategic acquisitions to expand their product portfolio. For instance, BioLife - a manufacturer of cryopreservation freeze media, announced its acquisition of Custom Biogenic Solutions - a producer of cryopreservation equipment for the biotech industry, to expand its portfolio of liquid nitrogen laboratory freezers and other cryogenic equipment.

Strategic acquisitions have led to rise in investments in improving the technology of freezers. As such, freezers segment is expected to account for the highest revenue of the cryopreservation equipment market. The segment is projected to reach a value of ~US$ 3.5 Bn by the end of 2027. Companies are increasing efficacy of cloud-based monitoring systems that help in evaluating biologic sample storage conditions.

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Developments in cryogen techniques have led to innovations in liquid nitrogen laboratory freezers. As such, liquid nitrogen cryogen segment dominates the cryopreservation equipment market and is projected to reach a value of ~US$ 5.1 Bn by 2027. Companies are tapping into opportunities for the development of tools for bioproduction of cell and gene therapies.

Automated Systems in Sample-prep Support Cryo-em Analysis for Protein and Drug Discovery

The cryopreservation equipment market is witnessing innovations in advanced sample preparation systems. For instance, TTP Labtech - a manufacturer of products within sample management, announced the launch of its next-gen automated system for sample-prep. Companies are making efforts to innovate in cryogenic electron microscopy (cryo-EM) in advanced sample preparation systems. As such, sample preparation systems are projected for exponential growth in the cryopreservation equipment market.

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Innovations in sample-prep systems are paving the way for cryo-EM analysis, which is instrumental in protein research and drug discovery. High-quality foil grids in systems for cryo-EM analysis are increasingly replacing manual processes in the healthcare industry. Thus, stakeholders in biotechnology and research laboratories are benefitting from these advanced systems to assess structure of biomolecules and support protein research. Advanced sample-prep systems are pervasively replacing conventional methods of NMR (Nuclear Magnetic Resonance) and X ray crystallography. These systems offer areas for researchers to solve complex protein structures, which was not possible with conventional methods. Improved 3D imaging and high speed blot-free plunging are key attributes that are attracting research companies in the cryopreservation equipment market landscape.

Stem Cell Storage and New Cell Manufacturing Plants Create Income Sources for Companies

Emergence of new cell manufacturing plants is complementing the growth of the cryopreservation equipment market. For instance, Cellex Incorporated - a biotechnology company, announced the launch of its cell manufacturing plant in Cologne, Germany to produce advanced therapy medicinal products for cryopreservation and cell purification, among others.

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Companies in the cryopreservation equipment market are expanding their services in long-term cryo-storage of advanced therapy medicinal products. Thus, manufacturing innovative cell therapy products for cancer is beneficial for creating new income opportunities for manufacturers of cryopreservation equipment. However, growth of the stem cells industry is another driver of the cryopreservation equipment market growth. Growing awareness about stem cell storage at birth is gaining importance in the cryopreservation equipment market landscape.

Cutting-edge Sensor Technology Aids pH and Co2 Measurement in Benchtop Incubators

The cryopreservation equipment market is largely consolidated with three dominant players accounting for a combined share of ~66% of the cryopreservation equipment market. However, difficulty to establish the right culture environment and incubator conditions for laboratories and IVF clinics are some of the challenges faced by emerging players. Hence, manufacturers in the cryopreservation equipment market are increasing production capabilities to develop equipment that provide insights on pH measurement. For instance, Planer a supplier of controlled temperature products, innovated in PetriSenseST, a sensor that provides monitoring of pH and CO2 in benchtop incubators.

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Incubators type segment dominates the cryopreservation equipment market and is projected to reach a value of ~US$ 2.9 Bn by 2027. Hence, equipment companies are increasing technical expertise in sensor technology to support incubator applications in laboratories and IVF clinics. Portability and flexibility of petri dish-sized sensors is gaining application in laboratory equipment. Thus, manufacturers in the cryopreservation equipment market are expected to increase their scope for incremental opportunities by developing advanced sensor equipment to cater to the needs of end users in labs and clinics.

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Cryopreservation Equipment Market: Increase in Demand for Regenerative Medicines to Accelerate Market Growth - BioSpace

Recommendation and review posted by Bethany Smith