One common theme was the importance of companies having their own launch protocols and fine-tuning them to suit individual therapy areas and markets. Although many companies have developed launch excellence codes, their diffusion and adoption across global organisations seem to be variable.

There are two key questions companies should ask themselves when theyre preparing to introduce a new product: do they have a launch framework, and is the company empowered to use it?, said Suzie Denton, Managing Director, Consulting at McCann Health.

The most successful organisations establish their own codes of practice to ensure theres a standardised approach to launching across the company. Then within that framework, theyll define clear launch archetypes; what type of launch is it? What level of investment does it require? What is the size and scale of expectation? Who needs to be involved? And what is the protocol for becoming launch ready from global to local? Secondly, empowering teams to use a framework is as important as establishing one.

"Everybody involved in the launch preparation phase must both understand and be able to implement the launch excellence approach the company believes in. It has to be cohesive, joined up and embedded across regions because, whatever the market nuances are, consistency in global and local delivery is essential.

Customer-focused

A second core component is the need to be customer-focused rather than brand-led. This philosophy underpins all successful launches throughout the brand life cycle. Launch excellence is about prioritising what matters for the launch brand. Every product is different, so you cant launch everything in the same way, said Denton.

The complexities of todays marketplace mean we really need to get beneath the surface of the brand and the opportunity, and take a bespoke approach to every launch. Running through all of this is the need to be customer-centric. Companies can sometimes become so focused on the data and the science that they forget to think about what matters to the doctor, the patient or the payer.

"What are the critical things that drive their behaviour now? What shift in behaviour is required to take them to where you want them to be? And how can you create meaningful engagement to help that transition? You wont change that belief state by focusing solely on the data you need to get closer to customers to help them along that journey. Customer-centricity is arguably the most important aspect of launch excellence. Without it, a brand cannot hope to succeed.

Sustainable healthcare: a key driver

Emma Gorton, Director, Hanover Communications, believes that advances in medicine are rewriting the rules of healthcare and changing the nature of pharmaceutical launches. The essence of what a launch is has changed, said Gorton.

Weve been through an era where we had incremental increases in innovation delivering blockbuster treatments for prevalent long-term conditions and headline diseases. However, were now moving into areas like immunotherapy and are on the edge of amazing innovations that will deliver great outcomes for patients.

"These advances are putting huge pressure on healthcare budgets. The debate has shifted; previously it was all around access to innovation but now theres a greater focus on the need for sustainability in healthcare systems.That has affected how launches work because people are looking for, and are now able to measure, different things from treatments cost- effectiveness, societal value, whole system costs.

There are huge implications for communications. Take gene editing, for example. Were going to get to the point where treatments like gene therapies will be ubiquitous so we need to set ourselves up for them now and embrace the ethical debate so that we bring the public along with us.

"We dont want to be in a situation where the public only understands the complex access challenges we will face for potentially curative medicines when people are being denied them due to cost. We need to get ahead of it. That means thinking about the things that might affect a launch as early as possible and shaping those communications proactively. As things get more complex, we need more time to explain them, to prepare the market.

Timing is everything

When it comes to launching a new medicine, timeliness is key. Essentially, a good launch is about getting breakthrough treatments to the people that need them as quickly as possible, said Gorton.

There are lots of stakeholders involved in that process right across the ecosystem and were all working towards that same goal. Progress is about developing relationships, creating collaborations and building long-term advocacy to ensure medicines get to the right people in the shortest possible time.

"Success requires being agile and responsive to fast-moving environments, and also being much more efficient. The rise of AI, along with better access to deep data, has the potential to reduce R&D time frames making it easier to develop launch plans closer to launch when the environment is much clearer.

Right customer, right time, right message

Stakeholder engagement is critical, but in an era characterised by significant pressure on costs both among customers and companies optimising resources is the name of the game. Companies are striving to be more innovative in how they go to market, said Sabine Dettwiler, Managing Director, Commercial Advisory Group, Syneos Health.

Its a huge ongoing challenge. How do you deploy your resources so you get to the right customer at the right time with the right messages but, at the same time, minimise costs? Theres a real focus on customer-centricity, yet we still see launches where companies go after the wrong stakeholder or dont tell the right story.

"Fundamentally, you need to develop a relevant, resonant story that differentiates your product and clearly conveys the value you bring to the patient and the healthcare system as a whole. Doing this means knowing your market and your stakeholders inside out, and understanding whether you can carve out a niche where youre likely to get more traction.

"The best launch plans define how theyre going to target that niche. Many companies have moved away from one-size-fits-all messaging.Theyre developing tight, customer-specific stories that align with an overarching message and theyre leveraging market insight to create sound strategies for how they target them. The key is to engage early.

Develop launch plans by purpose

One long-standing challenge in pharma launches is the engagement between R&D and commercial. In most pharma companies, the owner of an asset until phase 3 is typically development, said Dettwiler. However, development people rarely have commercial backgrounds or even if they have, they are incentivised by commercial metrics.Too often, companies are late in considering the market potential of a compound.

"Those that leave those commercial considerations until phase 3 are invariably getting there too late. Its improving, but its still an issue. The most progressive companies have shifted their approach; instead of developing launch plans by function, theyre designing them by purpose. Theyre looking at their strategic objectives and working backwards.

If pharma companies want to rewrite the headlines of launch excellence, they need to ensure that their planning, strategy and execution provide unambiguous answers and leave no lingering question marks at all

"What are we trying to achieve? What do we need to get there? Do we need a cross-functional approach that involves input from market access, HEOR, marketing etc and at what point do we get them on board? This approach is helping to tear down silos and develop a structured, cross- functional roadmap that helps get a product to market as efficiently as possible. Ultimately, all these functions have a shared purpose. Launch excellence is about understanding that purpose, as early as possible, and collaborating with the right people at the right time in order to deliver it.

Trial and error: the danger of isolating clinical and commercial

One of the most frequently voiced theories on launch excellence is the importance of starting with the end in mind. In pharmas case, this means configuring clinical development in line with identifiable customer needs. Its a common sense principle but its not yet become a default behaviour across the industry.

The traditional approaches to launching a product still tend to sit in the commercial function, said Chuck Stevens, Vice President and Global Head of Access, Commercialisation & Communications at ICON plc. However, those approaches need to migrate out and begin to align with HEOR, medical affairs and clinical teams. Companies need to look at how they design their clinical trials and what their target regulatory approaches are in different markets around the globe and start to overlay a commercial launch approach onto that regulatory sequencing.

Often, the way clinical and regulatory teams configure clinical trials to meet local regulatory requirements is misaligned with how a drug needs to be introduced commercially in those markets. Similarly, commercial teams need to drill down and understand HTA requirements within the disease category to ensure trials are designed to capture the necessary data for modelling.

"When drugs dont receive HTA regulatory support in major markets, its sometimes because companies havent modelled correctly so when HTA bodies reverse engineer those models, they find that either information is omitted or things are included that skew the way they would normally assess a drug. These costly errors underline why the early alignment of R&D and commercial is essential at launch.

Dont forget the patient

Another area where theres room for improvement is pharmas understanding of the end user the patient consumer. Although every company says and believes that theyre patient-centric, their understanding of what constitutes value to a patient is often suboptimal, said Stevens.

Definitions of value will obviously differ from customer to customer payers, patients, prescribers and providers will all have different views. In todays marketplace, with the level of innovation coming through in areas like rare disease and genetic disorders, its incredibly difficult to model cost-effectiveness to determine value for the patient or caregiver. Theres also lots to do before we can define societal value for expensive curative treatments.

So how does this translate into launch excellence? Very early in development as early as phase 1 and 2 patient insight is critical, said Stevens. Pharma must move beyond traditional market research and engage patients, one-to-one, to uncover their perspectives on everything from their disease experience and diagnostic pathways, to the things that prevent treatment and the real-world implications of taking a medicine.

"Understanding all these things is important it will shape trial design, identify unmet need and help articulate the patient value that will drive adoption at launch. True patient insight and engagement is critical.

The headline

Launching a new medicine is like crafting an article that captures attention: its how you start that matters. If pharma companies want to rewrite the headlines of launch excellence, they need to ensure that their planning, strategy and execution provide unambiguous answers and leave no lingering question marks at all.

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It started with a miss - PMLiVE

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(CNN) Inhis last interview with a Los Angeles Timessports columnist, Kobe Bryant, of course, talked about basketball and the LA Lakers.

But he also spoke about being a family man and how he wanted to elevate youth and womens sports, according to audio of the interview released by the LA Times Saturday.

When asked why he didnt attend many Lakers home games, Bryant said hed rather spend time at home.

I have gone through 20 years of the majority of my career with my kids Natalia and Gianna without being able to have that consistently, Bryant said.

So for me to make a trip up to the Staples Center, that means Im missing the opportunity to spend another night with my kids, and I know how fast it goes. Natalia is 16 and Gianna is 13. So that time came and went and so I want to make sure that the days Im away from them, are days that I absolutely have to, he said. Id rather just be hanging with them.

The interview is from October, just a few months before Bryant, his 13-year-old daughter Gianna and 7 others were killed in a helicopter crash. Columnist Arash Markazi decided to drive two hours to meet Bryant for a 17-minute interview.

It was one of the best decisions Ive ever made in my career, Markazi wrote.

Markazi asked Bryant abouthis relationship with Gianna, who went by Gigi, and coaching her youth basketball team.

Its a trip to see her move and the expressions that she makes, Bryant said. Its a trip, you know, the genetics. Genetics is a real thing, man.

He continued: What I love about Gigi is her curiosity about the game Even in a very heated situation in a game where its very competitive and back and forth, she can detach herself and come over and ask a very specific questions, which is not common.

All of our girls can do that, Bryant said, but the part that I think is most exciting is that its her curiosity and her ability to think critically in tight situations, (thats) pretty damn cool.

Bryant was asked about imagining his daughter moving away to play ball in college, and like many fathers he admitted, you never want to see your kids leave home, but eventually they have to.

Bryant also shared his thoughts about elevating female athletes, saying that hed always been abig supporter of the womens game. But having daughters who play sports his oldest, Natalia, plays volleyball was a big part of his desire to help push womens sports forward.

Just trying to enhance the womens game, not just in basketball but in volleyball and other sports, is extremely important. Anything I can do to help, Im gonna do.

He pointed to coaching his team at the Mamba Sports Academy, and telling the players and coaches that it was a group effort to win. Similarly, this is a joint effort to raise the womens game, he said.

Markazi also asked Bryant about his legacy, and whether he saw the Mamba Academy and youth sports playing a big role in how he was remembered.

Hopefully, he said, if we do it the right way, were known more for what we did after than what we did during.

I think you can have a lasting impact, he said. I mean winning championships, thats great. Building families, thats great. But when you can create stories and create moments and events and companies that can provide opportunities and inspire kids and create situations where people can be better, I think that has a lasting impact, more so than winning championships does.

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Kobe Bryant talked family and elevating female athletes in last interview with LA Times columnist - KFOR Oklahoma City

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NXIVMthe self-help organization lead by Keith Raniere that was actually a cult and possessed a secret wing that coerced women into sex through blackmailhas yet another damning accusation. Turns out, some of the sex abuse was also, allegedlyracist.

According to Times Union, a daily paper that services the NXIVM hub of Albany, Raniere created an empowerment group called One Asian to recruit East Asian women for sex. He also attempted to establish another group targeting women in sororities called Ten C, which stood for The Emperor Has No Clothes.

A newly filed federal lawsuit against Raniere alleges that he and his associates successfully lured over 100 women for One Asian, The Times Union reports. One Asian offered a special curriculum that Raniere tailored to what he characterized as women raised with more masculine values than Western women, the lawsuit reads. According to Raniere, this combination of female genetics and masculine attributes, such as discipline and self-denial, made the woman he recruited virtually perfect humans. Thus, he selected women who specifically met Ranieres criteria for potentially suitable sexual partners, the document states. One of the platniffs, a Canadian woman chosen to lead One Asian, said Ranieres right hand/Smallville actor Allison Mack told her sex was no big deal and just like playing tennis.

According to The New York Times, NXIVMs many programs became increasingly misogynistic over time, including indoctrination that instructed women that they did not deserve equal pay because they need to quit their jobs to have children, and that men are meant to be polyamorous while women are meant to be monogamous.

In June 2019, Keith Raniere was found guilty on seven counts, including racketeering, racketeering conspiracy, wire fraud conspiracy, forced labor conspiracy, sex trafficking, sex trafficking conspiracy and attempted sex trafficking. He is expected to be sentenced in Brooklyn on April 16.

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NXIVM's Keith Raniere Allegedly Created an 'Empowerment Group' to Recruit Asian Women for Sex - Jezebel

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A saleswoman takes meatloaf from a meat counter in a supermarket. Photo: Jan ... [+] Woitas/dpa-Zentralbild/ZB (Photo by Jan Woitas/picture alliance via Getty Images)

The notion that processed meat is not good for your health is not a new one. The high salt content, additives, and even just the red meat itself are all known to be linked to increased incidence of high blood pressure, heart disease, and obesity. But it is also known that studies assessing nutritional intake and health outcomes are notoriously difficult to carry out, and even more difficult to interpret. Even when such studies attempt to tease out other lifestyle variables such as exercise habits, tobacco use, alcohol intake, stress, and sleep quality, the data are always subject to debate, especially when a smattering of genetics and co-morbidities (other health issues) are thrown in.

A 2019 editorial in the Annals of Internal Medicine pointed out the reasons for these inconsistencies. In it, the authors describe several studies with vastly differing conclusions, ranging from processed meat is carcinogenic to processed meat is probably carcinogenic to the association between processed meat consumption and colon cancer and cardiovascular disease is weak. The editorial then reviews several meta-analyses which included hundreds of other studies, totaling over six million individuals. These studies found extremely small differences in overall health outcomes based on processed meat consumption, including all-cause mortality, cancer, and heart disease.

Three year old child eating salami

Because even the largest nutritional studies are observational, with so much of the data subject to confounding variables of other lifestyle habits and genetics, even those with high statistical significance for any link between sausage links and heart disease are not necessarily translatable in the real world of objective data. Better studies look at randomized controlled trials, which, even if performed over shorter duration, as short as six months as opposed to many years, may offer more accurate results than purely observational data. But even these studies have not determined with any certainty the health benefits or harms of meat consumption.

A more recent study, published in the February 3, 2020 issue of JAMA Internal Medicine, included not only processed meat consumption as a potential association with cardiovascular disease and death, but also unprocessed red meat, poultry, and fish consumption. But again, even this study looks at association, which is quite different from causation. As an analogy of association, a sunny day is associated with crowds at the beach, but the sunny skies did not cause the beach to be crowded. Association has been muddied into causation in countless debates on health data, including data on meat consumption, red wine consumption, vaccination schedules, and screen time, to name just a few. The authors of this months JAMA study sought to find an association (again, not causation) between various types of meats and fish with all-cause mortality and cardiovascular disease.

Young Woman Eating Chicken

The study collected data between 1985 and 2002, and participants were followed until 2016. The subjects included over 29,000 adults from six individual cohort studies. The average age was 53 years, and included roughly equal numbers of individuals who identified as male or female. Seventy percent of the subjects were white. Of the four types of consumed products, those with any of the three types of meat consumption, including poultry, had increased incidence of cardiovascular disease compared to those reporting only fish consumption. Both processed and unprocessed meat consumption were associated with increased all-cause mortality compared to those consuming just fish or poultry. All good news for you fish and fowl consumers? Perhaps. But even this study is one of association, despite the fact that they followed subjects for many years, in whats known as a prospective as opposed to a retrospective study.

In a prior Forbes article, I reported on clinical guidelines published in the October 2019 issues of the Annals of Internal Medicine, which found that there were no health benefits to reducing processed or unprocessed meat consumption. Digging a little into that study authors relationships quickly found that they had financial ties to the meat industry. Clearly an association that will bias any data, even when the data showed association, not causation.

Coming into play now is the health of our planet, not just our coronary arteries. Many environmental studies are pointing at meat (and dairy) consumption as major sources of greenhouse gases, and that if we humans all became vegetarians (or better, vegans), the impact on reducing climate change and improving air quality would save us all. Other data have found this not to be the case, showing that animal agriculture accounts for less than 4% of U.S. greenhouse gas emissions. Another way of looking at meat consumption data is by the energy needed to produce the energy meat gives. In other words, one study reported that livestock use 80% of farmland energy but produce only 18% of consumed calories. In addition, beef produce over 100 kilograms of greenhouse gases per 100 grams of protein they provide, whereas tofu, a plant-based soy protein, produces only 3.5 kilograms of greenhouse gas for that same 100 grams of protein.

Beef cattle standing in a field

We still do not have answers when it comes to meat consumption- neither for our own health nor for the health of our planet. While many human studies are leaning towards data showing poorer health outcomes with meat consumption, and environmental studies are also showing negative impacts on the climate due to beef agriculture, definitive data is still up for grabs. No matter how you slice it.

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The Heartbreak Of The Deli Counter: More Thinly Sliced Data - Forbes

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A reporter examines a 23andMe DNA genetic testing kit in Oakland, California.

Cayce Clifford | Bloomberg | Getty Images

It has not been a good year for consumer DNA testing companies.

In January, Silicon Valley-based 23andMe laid off 100 employees, about 14% of its workforce. A month later, Ancestry, which has offices in Utah and San Francisco, also cut 100 jobs, representing about 6% of its staff.

The major reason for the downsizing? Simply put, consumers aren't buying as many at-home DNA tests as they used to.

The first sign came in the summer, when Illumina, maker of the DNA sequencing machines that are used by Ancestry and 23andMe, acknowledged in an earnings call to investors that the category had hit a lull. CEO Francis DeSouza didn't share an explanation for that, but noted that Illumina was taking a "cautious view" of the opportunity in the near term. Orasure, maker of the spit tubes used by consumer DNA testing companies, has also seen its stock take a hit.

At that time, some smaller companies were already feeling the impact. Helix, a start-up that spun out of Illumina to build an "app store" model for DNA tests, cut staff in May. The company revealed to Bloomberg that it was shifting its focus away from consumers to population health, meaning it would work with health industry partners. A few months later, Veritas Genetics another company focused on consumers that sold more expensive but more detailed whole genome sequencing tests shuttered its U.S. operations.

So what happened? There hasn't yet been a detailed study to understand the shift in consumer thinking around these tests. But CNBC spoke with some of the leading genetics experts and doctors, who shared a few theories.

Dawn Barry, a former Illumina executive with a start-up in the space called LunaDNA, blames a few factors, especially privacy concerns.

Consumers have seen a slew of reports in the past few years about how companies are using their personal data for targeted advertising, without their knowledge, and might be feeling particularly sensitive about their health information.

Anne Wojcicki, CEO of 23andMe, has previously referred to these concerns as the "Facebook effect." In her view, consumers are increasingly freaked out about stories they're reading in the media about privacy, mostly about Facebook and other technology companies, and are reacting by feeling anxious about getting DNA tests.

Companies like 23andMe do make money off this information. Her company does ask for consent from users and it has publicly explained its revenue model, but a big part of its business involves its relationships with pharmaceutical companies like GlaxoSmithKline. 23andMe also has a therapeutics arm, where it is hoping to leverage its database of millions of people's DNA to develop new drugs.

Making matters worse for these companies, suggests Barry, is the Golden State Killer case. Law enforcement honed in on a suspect after running DNA from a decades-old crime scene through a free online database, where anyone can upload their genetic information.

A suspect was found through a distant relative who might have paid for a test via Ancestry or 23andMe, and then uploaded it into the database.

The case raised all sorts of complicated questions about whether genetic information is fundamentally different than other types of data because it implicates family members and not just individuals.

Other experts suspect that consumer DNA testing companies might have run out of early adopters. The theory goes that there's about 20 million or 30 million consumers who are naturally interested in learning more about their family background, and it's not that challenging or expensive to sell tests to them. At this point, many of these people have already been sold to, and there's no reason for them to buy a second test. Ancestry has sold about 14 million tests, and 23andMe has sold some 9 million.

But many people are wary about learning information they might not want to know like the father who raised them isn't their biological father or that they have a risk for a genetic disease that they can't take a pill to prevent.

There's likely a larger consumer segment that's interested, but still wary about these tests. They might not believe that the information is valuable enough to warrant the price tag. The cheapest tests sell for $99, and they'll cover ancestry and some health risks but lack truly actionable health information, like whether an individual might respond poorly to a drug based on their genetic makeup.

"The ancestry market is a finite market," said David Mittelman, CEO of Othram, a genomics start-up and a molecular physicist. A decade or so in, "these companies are beginning to tap out the market."

Mittelman notes that customer acquisition costs, including ad dollars these companies need to spend on sites like Facebook, will increase over time.

"I think the companies know this," he said. "The investment in health shows that they are working to appeal to a broader market."

What's noteworthy about the recent round of layoffs is that Ancestry kept all of its employees at its Ancestry Health business. And 23andMe is still highly focused on its drug development business. That suggests that both companies are indeed hinging their future on developing powerful health applications.

In light of that, some geneticists are optimistic about their future.

"First of all, a slowdown isn't a stoppage," said Dr. Robert Green, a professor of genetics at Harvard Medical School. "Our research is finding that genetics is about to take its rightful place in medical care for the world."

As Green explains, it's been a challenge for doctors to understand how genetics can inform their patient care. Many haven't had the education about genetics to understand how to talk about it with their patients or recommend tests that might be beneficial. But that's starting to change.

For instance, 23andMe is starting to roll out new tests that can identify people's risk for chronic diseases like diabetes, called polygenic risk scores. These results could be used by doctors to help steer their patients toward making healthier lifestyle choices to help them avoid getting the disease.

And for these companies, which already have genetic databases of millions of people, they might not need to keep spending ample marketing dollars to acquire new customers. Instead, they could focus on developing new insights from their existing databases. if they succeed at that, they can forge partnerships to the medical industry.

As Mittelman puts it, there's no need to "force people down an ancestry funnel."

Green agrees, saying companies like 23andMe and Ancestry might double down on more expensive but more detailed sequencing tests that provide a lot more relevant health information. 23andMe has dabbled with those kinds of tests but has been reluctant to roll out higher-priced tests while its main focus has been growth.

"The direct-to-consumer phenomenon will give way to a more of a proper integration of genomics into the day-to-day care of patients," said Green. "What we're seeing is a course correction, and consumers are waking up to the potential limitations of a $99 test."

CNBC Evolve will return, this time to Los Angeles, on June 8. Visit cnbcevents.com/evolve to apply to attend.

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Consumer DNA testing is a bust: Here's how companies like Ancestry and 23andMe can survive - CNBC

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Clinical vs. DTC Genetic Testing

When 23andMe laid off roughly 100 employees last month, it was another admission that direct-to-consumer, or at-home, genetic testing sales are down at some leading companies. The reasons, however, while varied, have nothing to do with the interest in genetic testing. People want the information but it seems many prefer to get it from their doctors.

Theres reason for the interest: The field of genetics is booming.

Since the 1990s when we started testing for BRCA1, BRCA2 [which most notably increase the risk of breastandovarian cancers]and TP53 [which regulates cell division and keeps tumors from forming], the number of indications, or signs for cancer and the number of genes we can identify has expanded. And it will only continue to grow, Dr. Banu Arun, co-medical director of the Clinical Cancer Genetics Program at MD Anderson Cancer Center, tells SurvivorNet.

She notes that clinical genetic tests can aid in making recommendations for surveillance, determine prognosis and assist in treatment decision-making for cancer patients. Direct-to-consumer genetic tests dont offer that proverbial microscope.

When trying to understand your hereditary cancer risk, saysDr. Arun, clinical testing is the way to go.

Comparing DTC to clinical testing for cancer is actually a bit like apples to oranges.

Direct-to-consumer tests, which are relatively inexpensive ($99 and up), can make predictions about peoples health and ancestry. But theyre limited when it comes to offering tests for cancer risk. Currently, theFood and Drug Administration (FDA) has allowed at least one direct-to-consumer genetic testing company, 23andMe, to offer a test for cancer risk; it looks for three specific variations in BRCA1andBRCA2.

With direct-to-consumer testing, its only a very small piece of the puzzle, Megan Frone, board certified genetic counselor in the Clinical Genetics Branch at the National Cancer Institute, tells SurvivorNet. I think about it like a typo somewhere in a novel, she says. Theyre only looking at three pages, she says. You could have a typo anywhere else in the novel thats breaking that gene and giving you a higher risk for cancer, and theyre just not looking.

Clinical-grade testing can analyze the risk of 50 different types of cancer, according to The National Cancer Institute.

A study last year from the National Center for Biotechnology Informationnotes some other drawbacks, including how DTC tests frequently do notprovide conclusive results.

Most genetic tests performed by DTC companies are limited to few major genetic variants related to the phenotypes of interest, which leads to poor discriminatory power, it notes.

This means DTC genetic testing does not guarantee that a consumer with a high genetic risk score will suffer from a certain disease, it only indicates a genetic propensity.

Plus, test results could reveal other, unexpected, health risks.

You have to ask, Are they going to tell me about specific results I might not want to know about?' Frone says. Some at-home tests will tell you your risk for carrying certain Alzheimer gene variants. We dont have any particular treatment for Alzheimers. Some people dont want to know about that stuff because theres nothing they can do about it. They dont necessarily realize that theyre going to get that back on a test report.

All of which points to that important ingredient: an expert who can interpret and make an action plan.

DTC tests are often conducted without the involvement of a healthcare provider and without an understanding of clinical validity and utility, notes theCenters for Disease Control.

A recent study based on anonline survey of 1,001 adultsrepresentative of the population, found that public awareness of genomics and personalized medicine was not increasing in line with advancements in the industry. Seventy-three percent of the survey respondents had not heard of genetic counseling which is conducted by certified health professionals to advise consumers/patients on how to interpret genetic test results.

The first step for someone interested in learning about their risk for developing cancer, according to Frone, is to speak with a health care provider or genetic counselor to learn about options. Then, a risk assessment can be conducted by a certified genetic counselor.

In this type of consult, theyll look at personal medical history and family medical history. And, if youre female, theyll look at your hormonal risk factors, Frone explains. There are computer models to identify risks and patterns within someones personal and family history.

The next step, she says, is to discuss what needs to be done to test for the potential hereditary cancer syndrome.

People need to go into it understanding that genetic testing is really complicated, Frone says. To achieve the full value of it, weve got to apply the genetic test results in the context of their family health history and their other risk factors to understand final risk. Direct-to-consumer testing is very different from that. Its more recreational.

For those identified during the consult as being higher risk, insurance typically covers clinical testing. For people at a lower risk, they may be advised to skip testing altogether, or, they can pay out-of-pocket (costs can vary from $250 or more depending on whether a single gene or the entire genome is being tested).

For example, the BRCA1 and BRCA2 test 23andMe runs has been given the green light from the FDA, meaning the agency has determined that the benefits of the product outweigh the known risks for the intended use.

This specific test looks at three variants to determine if a woman is at an increased risk for developing breast and ovarian cancer, or if a man is at a higher risk for developing breast and prostate cancer.

It really is most relevant for individuals with Ashkenazi Jewish background because it can only look at three variants that can occur in these genes, when there are thousands possible, Frone says.

The actual test has been put in the Medical Devices class II risk category by the FDA. Class I devices, such as dental floss, are lowest risk. Class II, which includes condoms and powered wheelchairs, are moderate risk. Class III devices, such as pacemakers, require FDA approval in order to be marketed, while class I and II do not.

These mutations are most common in people of Ashkenazi Jewish descent and do not represent the majority of the BRCA1/BRCA2 variants in the general population, the FDA cautions.

Researchers estimate that roughly 5-10 percent of all cancers have a known genetic element, so while DNA tells a story, it doesnt write the entire script. External factors, like environmental nutrients or toxins, and lifestyle choices also influence risk for developing cancer.

Learn more about SurvivorNet's rigorous medical review process.

Kim Constantinesco is a freelance writer who specializes in health and founder ofPurpose2Play, which reports on positive and inspiring stories in sports. Read More

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Genetic Testing Is on the Rise Heres Why to Get it Done Through a Health Care Provider - SurvivorNet

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(Reuters) - Genetic testing specialist Qiagen (QIA.DE) should have a targeted test for the coronavirus ready this month and is extremely active in providing products to disease control institutions in China, its interim CEO said on Wednesday.

But while Qiagen could boost profits by focusing on products identifying the coronavirus it will continue to bet on growth in its molecular diagnostics business, rather than uncertain one-off gains, CEO Thierry Bernard told a results conference call.

The company headquartered in the Netherlands, which makes diagnostic kits for cancer and tuberculosis as well as products for identifying viruses, reported better than expected quarterly sales and profit growth late on Tuesday.

Qiagen shares were up 5.6% at 1555 GMT on Wednesday, set for their best day in three months.

Bernard said in a results statement on Tuesday that Qiagen had not included global demand for products that can be used to recognise the coronavirus in its 2020 outlook, given the uncertainties around broader business trends in China.

The company is looking into different variants of coronavirus testing solutions, including options using QIAsymphony, NeuMoDx or a syndromic solution, Bernard told the call on Wednesday.

Qiagens increased focus on its molecular diagnostics division is one of the reasons why its adjusted operating margin of 33.5% in the fourth-quarter came in well above its own forecast and the market consensus, analysts have said.

Bernard said the divisions QIAsymphony automation system could become a important source of new growth after its long-established major growth driver, the tuberculosis test QuantiFERON, saw a slowdown in the fourth quarter.

After Qiagen reached a new milestone with more than 2,500 cumulative placements of QIAsymphony in January, the product has become a steady and consistent growth engine and it is planning for more than 200 new placements in 2020, he said.

Thanks to its expanded partnership with DiaSorin, Qiagen should be able to grow QuantiFERON by double digits again in 2020, Berenberg analysts said.

Reporting by Zuzanna Szymanska; Editing by David Clarke

Read the rest here:
Qiagen expects coronavirus test to be ready this month: CEO - Reuters

Recommendation and review posted by Bethany Smith

By Roy Freiman, Andrew Zwicker and Annette Quijano

We would never expect a bank to release our financial information any more than we would want the government to distribute our social security number, or a doctor to share our medical records. So why are we so compromising when it comes to the privacy of our most sensitive personal information: our genetic data.

Containing information as basic as your ethnicity to information comparably as intimate and unique as your fingerprint, genetic data can reveal a lot about who you are both on the surface and beneath it.

With the rise of direct-to-consumer genetic testing, millions of Americans began opting-in to services offered by private companies like 23andMe and Ancestry.com with hopes of discovering more about their heritage, connecting with lost relatives or identifying increased risk for disease like Alzheimers and breast cancer. However, as they swabbed their mouth or spit in a tube and mailed in their DNA for analysis, few paused to consider the implications for their data privacy.

As details of how your data could be used and where it could end up remained deep in the fine print of company policies, many remained unaware of what and how much they had consented to give away. A Deloitte survey from 2017 puts the number of Americans willing to consent to legal terms and service agreements without reading them at 91%. That fact, paired with limited industry transparency, has put a huge burden on consumers and made informed consent relatively elusive.

Today, it is quite likely if you submitted your DNA sample for testing, your genetic data sit anonymized in a database owned by a pharmaceutical company, academic research group or some other third-party entity. In 2018, 23andMe struck a $300 million deal to share its genetic database with pharmaceutical giant GlaxoSmithKline, and the company maintains partnerships with Procter & Gamble Beauty, Pfizer and others. Ancestry.com similarly shared data, collaborating with Googles Calico to study aging.

And, while in the aggregate this data presents enormous opportunity for new drug development and biomedical advancement you can never really be sure that your data wont be leveraged by bad actors for all the wrong reasons.

As a number of studies have pointed out, anonymized data are not fool proof. With enough effort, a significantly large chunk of data can be traced to the originating individual and their relatives. Running crime scene evidence against DNA profiles publicly uploaded to GEDmatch, police solved the four decades old Golden State Killer cold case. Therefore demonstrating that, despite efforts by companies to detach identifying information, the promise of anonymity and privacy is difficult to guarantee.

We are lacking a regulatory framework to address the privacy of genetic information. As strong federal policy in this specific area has yet to emerge, what has resulted is an industry that remains largely under-regulated and a public that is alarmingly uninformed.

New Jersey cant wait. We need better safeguards for genetic privacy now. Thats why weve charted our own course, sponsoring legislation (A-1170) that not only requires consent to use DNA samples and any resulting genetic information, but also makes DNA samples the exclusive property of the individual.

Stipulating ownership of genetic data is about securing New Jersey consumers right to choose. Empowered and informed decision-making can only arise when we are the ones in control of who can and cannot access or use our data. Under the measure, we assure the power always remains in the hands of the consumer.

With this legislation, New Jersey would join 24 other states that require informed consent to disclose genetic information. The state also would become sixth after Alaska, Colorado, Florida, Georgia, and Louisiana in explicitly defining genetic information as personal property, and second only to Alaska in extending personal property rights to DNA samples.

Protecting people and ensuring all industry competitors play by the same rules are fundamental strategies to securing greater genetic privacy for New Jersey residents. Laws requiring fair and responsible industry behavior are how we keep genetic data in the right hands - the individual to whom it belongs.

Assemblyman Roy Freiman and Assemblyman Andrew Zwicker represent the 16th Legislative District, and Assemblywoman Annette Quijano represents the 20th Legislative District. Together they are sponsors of genetic data privacy legislation (A1170) introduced on January 14, 2020.

The Star-Ledger/NJ.com encourages submissions of opinion. Bookmark NJ.com/Opinion. Follow us on Twitter @NJ_Opinion and on Facebook at NJ.com Opinion. Get the latest news updates right in your inbox.Subscribe to NJ.coms newsletters.

Go here to read the rest:
Do you want anyone to have your most personal information? We dont think so, lawmakers say. - NJ.com

Recommendation and review posted by Bethany Smith

New Squamish resident Dianne Russell moved to town in November, eight months after meeting her dad, Tom James, for the first time.

Long-time local James didn't know he even had another daughter before last summer.

Though dramatic, such a reunion tale really couldn't have worked out better.

Russell was raised in Kamloops, knowing she was adopted.

She was living in Vancouver until last fall.

Bouts with breast cancer led the 48-year-old to seek genetic testing in 2017. Denied this testing through the provincial government, she used the home DNA kit, 23andMe.

"I got my results, but found no genetic risk for breast cancer, she said, adding that at the time, she ignored the relatives section of the site.

But a year ago on Valentine's Day, she got a message in her inbox that would change the course of her life. "Hi, I'm not sure where you fit on my family tree, but it looks like we're cousins or something," read the message.

Long story short, she found cousins who eventually connected her to her Squamish father, James, who up until that point had no idea a short high school relationship had created a child, never mind that the baby girl had been adopted.

Russell was conceived in Squamish and born in Nanaimo.

"The next day after he found out, I got a phone call at 9 a.m. and we talked for over an hour and we have talked almost every day since," Russell said.

They met in person in June.

"My dad is just the most amazing man," she said. "He is a wonderful, emotional, supportive, empathetic man. I won the family lottery."

After their first meeting, there were trips to Squamish for Russell and her husband to spend time with her dad, his girlfriend and the rest of the family.

"Not only do I have a wonderful, loving father with whom I get along like a house on fire, but I have an amazingly supportive half-sister and half-brother, a nephew, four uncles, two aunts, in-laws, and a slew of great aunts, uncles, cousins, and more cousins many of them are here in Squamish, which is why my husband and I decided to move here from Vancouver."

The whole family and community of Squamish have enveloped her with open arms, Russell said.

"This situation has the possibility for a lot of discomfort and judgment and it hasn't felt like that ever."

There have been two large family reunions since she was introduced, Russell said, and the acceptance has been overwhelming.

"My family is Mtis, actually, and my great aunt presented me with a Mtis sash," Russell said, adding she had thought she was of British origin growing up.

"I am not English," she said with a hearty laugh.

Russell said she and her husband had been thinking of moving before all of this happened and once she met her long-lost family in Squamish that sealed the deal to move here.

"We missed so much time together that we just wanted to be here," she said.

From getting the first message about her family to today, the journey has been full of a mixed soup of emotions, Russell acknowledges.

Grief is one of the emotions she has dealt with.

"Because I think we both didn't have each other," she said. "We didn't have each other for 48 years. He didn't know I existed. He didn't have the opportunity to know me."

Her father's parents, her grandparents, both died without knowing she existed, she added. "There is grief on both sides, I think."

Her advice for anyone starting out on the journey of DNA testing is to be open to unexpected things and unexpected feelings, and reactions.

"You have to process it as you go."

"It was just like I had known her my whole life," James said of his first phone call with Russell. The pair talked long enough to kill the cellphone battery, he said.

"It was so bizarre and so easy. It was natural amazing."

When they met in the flesh at Park Royal on June 13,

"It was oh my God tears and hugs and happiness. It was absolutely crazy," he said. "There's my baby and she is 48 years old."

In looking at Russell, James said he sees some physical features from his mom's side of the family.

"There is some James in there too, in the ears and around the mouth a bit. She has got the James' ears," he said, with a chuckle.

James has been proud to show off his newly-gained daughter, taking her downtown to introduce her to the many who know him.

He looks forward to making up for lost time, he said.

"Just to be here for her, whenever she needs me. Just to be her dad."

Russell is currently changing her last name to her father's. By spring she will officially be a James.

See the original post:
All in the family - Squamish Chief

Recommendation and review posted by Bethany Smith

The global gene therapy market is expected to reach $13 billion by 2024 as new treatment options target cancers and other diseases.Now, a team of scientists from Purdue University and other research institutions around the world have come together to better understand the growing number of worldwide patented innovations available for gene therapy treatment. They specifically focus on non-viral methods, which use synthetic or natural compounds or physical forces to deliver materials generally less toxic than their viral counterparts into the therapy treatments.

The possibility of using non-viral vectors for gene therapy represents one of the most interesting and intriguing fields of gene therapy research, said Marxa Figueiredo, an associate professor of basic medical sciences in Purdues College of Veterinary Medicine, who helped lead the research team and works with the Purdue Research Foundation Office of Technology Commercialization to patent her technologies related to health. This is an innovative method for identifying the technological routes used by universities and companies across the world and uncovering emerging trends for different gene therapy sectors.

The scientists used big data, patent and clinical data mining to identify technological trends for the gene therapy field. They envision that their analysis will help guide future developments for gene therapy.

This work brought together investigators from across the globe in a joint effort to use new databases and methods to better understand the trends of the gene therapy field in respect to non-viral vectors. Dimas Covas, coordinator of the Center for Cell-based Therapy, affiliated with the University of So Paulo in Brazil, lent his extensive experience in cell therapy. Aglaia Athanassiadou, Virginia Picano-Castro and Figueiredo contributed their extensive experience on non-viral vectors for gene therapy. Cristiano Pereira and Geciane Porto brought their expertise in economics and business administration to the analyses. Each contribution was fundamental to achieving a new way to identify technological trends in this field.

This work brought together investigators from very diverse disciplines to create a different perspective of the gene therapy field, Figueiredo said. Our groups continue to work individually or in collaboration to generate and patent new vectors to help fill the needs of this re-emerging field of non-viral gene therapy.ReferencePicano-Castro et al. (2020) Emerging patent landscape for non-viral vectors used for gene therapy. Nature Biotechnology. DOI: https://doi.org/10.1038/s41587-019-0402-x

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Technological Trends in Gene Therapy - Technology Networks

Recommendation and review posted by Bethany Smith

Have you ever slipped when trying to avoid sugar, quit smoking, or break another habit or addiction? Usually that one piece of cake or one cigarette wont ruin your whole plan, but for people struggling with cocaine addiction, one slip can undo months of hard work.

Cocaine consumption is increasing, with 2.2 million people in the U.S. admitting to recent cocaine use in 2017. In 2014, the National Survey on Drug Use and Health estimated that nearly 1 million Americans were addicted to cocaine. The effect of cocaine on the brain and body is so powerful that, even after state-of-the-art treatments, many people trying to quit cocaine relapse within a year.

What if cocaine could be made less euphoric, so that a single use by a recovering addict doesnt result in a full-blown relapse? Scientists at the Mayo Clinic recently published progress toward making this idea a reality a gene therapy that would treat cocaine addiction by making cocaine less rewarding.

We are a molecular biologist and a neurobiologist who are interested in understanding and treating human disease, including neurological disorders such as cocaine addiction. As University of Tennessee faculty members leading basic biomedical research, we have worked for years on how genes are turned on and off in people and the effects of cocaine on mice, respectively. So, we were excited to see a promising convergence of novel gene therapy and cocaine addiction therapy.

Beginning more than 20 years ago, scientists have worked to engineer a new version of a human protein that could break down cocaine so quickly that it doesnt produce an addictive high. We all have the normal human protein BChE that helps regulate neurotransmitters, and which can slowly break down cocaine. Targeted mutations in BChE can turn it into a super-CocH a protein that can quickly break down cocaine. When this CocH is injected into the bloodstream, it breaks down cocaine very fast before the user can experience the pleasurable effects so a dose of cocaine is less rewarding. Being less rewarding means it is easier to stop using cocaine.

Previous research has shown that injections of the super-CocH protein drastically decrease addictive behavior in cocaine-addicted rats. Thats great. But the problem is that daily CocH injections would be too expensive and difficult to maintain for the years needed to prevent cocaine relapse for human users. It would be much more practical to provide a single treatment that could provide enough CocH to last for years.

One way to do that is gene therapy: Give patients the DNA sequence (the gene) that contains the instructions for making super-CocH so their bodies can keep making it for months or potentially years. Fortunately, over the past decade, this type of gene therapy has been moving from science fiction to hopeful reality. Clinical trials have demonstrated the potential of gene therapy to treat diseases from hemophilia to neurodegenerative disorders, and a handful of these are FDA-approved. The new Mayo Clinic study takes an important step toward making CocH gene therapy a reality.

How exactly does a scientist give a person a gene? You cant just swallow DNA the way you would a pill. The Mayo Clinic scientists had to find a way to deliver the gene to every cell in the liver. The way they did this was to insert the gene for super-CocH into a virus called adeno-associated virus (AAV). AAV has been modified so that when it infects cells it cannot reproduce in the body or make someone sick. It is just a delivery vehicle. The virus works by delivering the CocH gene to liver cells, where it remains for months or years. The cells read the super-CocH gene and use it to manufacture many copies of the CocH protein, which then breaks down cocaine.

In the new study, the team tested this approach in mice. The results are very promising and suggest that this gene therapy is safe and effective. Mice receiving the gene therapy alone were healthy. Mice given cocaine became hyperactive and showed signs of liver damage. When the mice were given cocaine plus gene therapy they behaved normally, as if they had not been given the drug. The cocaine was quickly broken down by their new super-CocH proteins, and their livers showed no signs of damage.

The results are promising enough that the FDA has approved plans to proceed with human clinical trials.

Keep in mind, this treatment wont hit the market anytime soon. It took six years from initial tests of AAV-CocH therapy in mice to reach the point where the technique is safe enough for human trials. There are many aspects of the treatment that need to be evaluated and modified to make sure it is both safe and effective in humans.

For example, AAV gene therapy can produce unwanted immune responses in people that will need to be carefully monitored. Issues such as discomfort caused by the therapy, different responses based on an individuals genetic makeup and interactions with other medications or medical conditions will also need to be addressed.

Because this study only monitored mice for two months, longer-term effects of the gene therapy will need to be investigated. Also, how well this therapy works to treat cocaine addiction in mice is not really known, and treating addiction in humans is certain to be even more complicated.

This gene therapy could someday make a dose of cocaine less rewarding, but a full recovery from addiction will likely require a combination of treatments administered over many years.

Like many, the two us have family members or friends who struggle with addictions that cannot be cured simply by trying harder. This recent work combines careful scientific progress with a creative new idea, giving hope to those trying to overcome cocaine addiction.

See original here:
Potential gene therapy to combat cocaine addiction - The Conversation US

Recommendation and review posted by Bethany Smith

The collaboration with Childrens Medical Research Institute will boost the efficiency of AAV purification, leading to increased access to the viral vectors needed to manufacture gene therapies

GE Healthcare Life SciencesandChildrens Medical Research Institutewill jointly drive the development of new affinity ligands for the purification of adeno-associated viral (AAV) vectors used in gene therapies. The focus of the collaboration is to bring to market-specific ligands for multiple AAV types, enhancing the chromatographic separation of AAV-based vectors. This will improve the manufacturing efficiency and scalability of gene therapies, enabling the availability of viral vectors on a global scale.

With more than 800 gene therapies currently in clinical trials, there is an increasing demand for the raw materials needed in the manufacturing process of viral vectors. AAVs are viral vectors used in more than 70% of the in vivo gene therapy clinical trials. According to GlobalData, the 2025 gene therapy in vivo therapeutic market is expected to reach USD 32 billion with an estimated CAGR of 105% between 2019-2025.

The collaboration combines the expertise from the latest available research on AAVs with application testing, advancing a comprehensive understanding of the clinical functionality and the commercial opportunities of AAV-based gene therapies. Childrens Medical Research Institute will share with GE Healthcare Life Sciences AAV capsid variants targeting different tissues. GE Healthcare Life Sciences will then design and test ligand prototypes, which Childrens Medical Research Institute will assess. Based on the performance results, GE Healthcare Life Sciences will manufacture and commercialize novel improved AAV affinity ligands.

Dr Leszek Lisowski, the lead gene therapy scientist at Childrens Medical Research Institute, says: Bringing the fruits of our work to the patients requires a joint effort between academia and the industry. The collaboration with GE Healthcare Life Sciences will allow us to expedite the development of novel clinical options at a lower cost.

Olivier Loeillot, General Manager, Bioprocess at GE Healthcare Life Sciences, says: The industry needs better and more personalized technologies to speed biopharmaceuticals through clinical trials and bring them to market. Our long biomanufacturing expertise combined with Childrens Medical Research Institutes pioneering research will lead to purification technologies that will streamline the production of gene therapies.

Catarina Flyborg, General Manager, Cell and Gene Therapy at GE Healthcare Life Sciences, says: Collaborations with organizations such as Childrens Medical Research Institute are critical to developing the technologies needed to move the industry forward. By working directly with world-class researchers, GE Healthcare Life Sciences can develop the purification technologies that will contribute to increasing the availability of viral vectors globally.

Childrens Medical Research Institute in Australia is globally recognized for its work on microsurgery, cancer research, neurobiology, embryology and gene therapy. The AAV affinity ligands resulting from this collaboration will be compatible with GE Healthcare Life Sciences resin-based chromatography portfolio used in the purification of most FDA-approved biopharmaceuticals.

More:
GE Healthcare joins CMRI to optimize gene therapy manufacturing - BSA bureau

Recommendation and review posted by Bethany Smith

Surge in investments in R&D activities, rise in prevalence of cancer, and increase in awareness about benefits drive the growth of the global gene therapy market. However, expensive costs related to gene therapies and undesirable responses from immune system restrain the market growth. On the other hand, unlocked potential from the emerging markets would offer new opportunities in the near future.

Based on vector type, the viral vector segment accounted for the highest market share in 2018, holding more than half of the global gene therapy market, owing to ease in modifications offered by many viruses including Lentivirus, RetroVirus & Gamma RetroVirus, and Adeno-Associated Virus (AAV) for developing gene therapy drugs. However, the non-viral vector segment is estimated to grow at the fastest CAGR of 38.8% from 2019 to 2026. This is due to technological advancements taking place in physicochemical approaches of non-viral vectors.

Based on gene type, the tumor suppressor segment is estimated to portray the highest CAGR of 52.9% during the forecast period, owing to surge in number of methodology and clinical trials adopted for the gene therapy treatment. However, the antigen segment accounted for the highest share in 2018, accounting for more than one-fifth of the market, owing to the presence of different genetic mutations and dysregulated gene expression presented by tumor cells.

Based on region, North America accounted for the highest share in 2018, contributing for nearly half of the global gene therapy market, owing to rise in prevalence of rate of cancer and increase in investments for R&D activities. However, Asia-Pacific region is expected to grow at the fastest CAGR of 45.4% from 2019 to 2026, owing to increase in number of people getting affected by chronic diseases and launches of new gene therapy products.

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See the article here:
Global Gene Therapy Market to Reach $6.21 Billion by 2026: AMR - PRNewswire

Recommendation and review posted by Bethany Smith

DUBLIN--(BUSINESS WIRE)--The "Gene Therapy Market by Vectors [Non-viral (Oligonucleotides), Viral (Retroviral (Gammaretroviral, Lentiviral)), Adeno-associated], Indication (Cancer, Neurological Diseases), Delivery Method (In Vivo, Ex Vivo), Region - Global Forecast to 2024" report has been added to ResearchAndMarkets.com's offering.

High incidence of cancer & other target diseases is a major factor driving the growth of the gene therapy market

The high incidence of cancer and other target diseases, availability of reimbursement, and the launch of new products are the major factors driving the growth of this market. In addition, the strong product pipeline of market players is expected to offer significant growth opportunities in the coming years. However, the high cost of treatment is expected to hamper the market growth to a certain extent in the coming years.

Neurological diseases segment accounted for the largest share of the gene therapy market, by indication, in 2018

Based on indication, the market is segmented into neurological diseases, cancer, hepatological diseases, Duchenne muscular dystrophy, and other indications. The neurological diseases segment accounted for the largest share of the market in 2018. This can be attributed to the increasing number of gene therapy products being approved for the treatment of neurological diseases and the high market penetration of oligonucleotide-based gene therapies.

Viral vectors segment to register the highest growth in the gene therapy market during the forecast period

The gene therapy market, by vector, has been segmented into viral and non-viral vectors. In 2018, the non-viral vectors segment accounted for the largest share of this market. However, the viral vectors segment is estimated to grow at the highest CAGR during the forecast period, primarily due to the increasing demand for CAR T-based gene therapies and the rising incidence of cancer.

North America will continue to dominate the gene therapy market during the forecast period

Geographically, the market is segmented into North America, Europe, the Asia Pacific, and the Rest of the World. In 2018, North America accounted for the largest share of the gene therapy market, followed by Europe. Factors such as the rising prevalence of chronic diseases, high healthcare expenditure, presence of advanced healthcare infrastructure, favorable reimbursement scenario, and the presence of major market players in the region are driving market growth in North America.

Key Benefits of Buying the Report:

This report will help market leaders/new entrants by providing them with the closest approximations of the revenue numbers for the overall gene therapy market and its subsegments. It will also help stakeholders better understand the competitive landscape and gain more insights to position their business better and make suitable go-to-market strategies. Also, this report will enable stakeholders to understand the pulse of the market and provide them with information on the key market drivers, challenges, and opportunities.

Key Topics Covered:

1 Introduction

1.1 Objectives of the Study

1.2 Market Definition

1.3 Market Scope

1.4 Currency

1.5 Limitation

1.6 Stakeholders

2 Research Methodology

2.1 Research Data

2.2 Secondary Data

2.3 Primary Data

2.4 Market Size Estimation

2.5 Market Breakdown and Data Triangulation

2.6 Assumptions for the Study

3 Executive Summary

4 Premium Insights

4.1 Gene Therapy Market Overview

4.2 North America: Market, By Vector (2018)

4.3 Geographical Snapshot of the Market

5 Market Overview

5.1 Introduction

5.2 Market Dynamics

5.2.1 Drivers

5.2.1.1 High Incidence of Cancer and Other Target Diseases

5.2.1.2 Product Approvals

5.2.1.3 Funding for Gene Therapy Research

5.2.2 Opportunities

5.2.2.1 Strong Product Pipeline

5.2.3 Challenges

5.2.3.1 High Cost of Treatments

6 Gene Therapy Market, By Vector

6.1 Introduction

6.2 Non-Viral Vectors

6.3 Viral Vectors

7 Gene Therapy Market, By Indication

7.1 Introduction

7.2 Neurological Diseases

7.3 Cancer

7.4 Hepatological Diseases

7.5 Duchenne Muscular Dystrophy

7.6 Other Indications

8 Gene Therapy Market, By Delivery Method

8.1 Introduction

8.2 In Vivo Gene Therapy

8.3 Ex Vivo Gene Therapy

9 Gene Therapy Market, By Region

9.1 Introduction

9.2 North America

9.3 Europe

9.4 Asia Pacific

9.5 Rest of the World

10 Competitive Landscape

10.1 Overview

10.2 Market Share Analysis, 2018

10.3 Key Strategies

10.4 Competitive Leadership Mapping (2018)

10.4.1 Visionary Leaders

10.4.2 Innovators

10.4.3 Dynamic Differentiators

10.4.4 Emerging Companies

11 Company Profiles

11.1 Biogen

11.2 Gilead Sciences, Inc.

11.3 Amgen, Inc.

11.4 Novartis AG

11.5 Orchard Therapeutics Plc

11.6 Spark Therapeutics, Inc. (A Part of Hoffmann-La Roche)

11.7 Molmed S.P.A.

11.8 Anges, Inc.

11.9 Bluebird Bio, Inc.

11.10 Human Stem Cells Institute (HSCI)

11.11 SIBIONO Genetech Co., Ltd.

11.12 Shanghai Sunway Biotech Co., Ltd

11.13 Uniqure N.V.

11.14 Gensight Biologics S.A.

11.15 Celgene Corporation (A Bristol-Myers Squibb Company)

11.16 Cellectis

11.17 Sangamo Therapeutics

11.18 Mustang Bio

11.19 AGTC (Applied Genetic Technologies Corporation)

11.20 Poseida Therapeutics, Inc.

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

Read more:
Global Gene Therapy Market is Projected to Reach USD 13.0 Billion by 2024 from USD 3.8 Billion in 2019, at a CAGR of 27.8% - ResearchAndMarkets.com -...

Recommendation and review posted by Bethany Smith

BOSTON and LONDON, Feb. 10, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (ORTX), a global gene therapy leader, today announced upcoming presentations from its neurometabolic franchise at the 16th Annual WORLD Symposium on February 10-13 in Orlando, FL. Accepted abstracts include encore clinical presentations for OTL-200 and emerging data quantifying metachromatic leukodystrophy (MLD) caregiver-reported quality of life experiences, as well as clinical data for investigational treatments in mucopolysaccharidosis type I (MPS-I) and mucopolysaccharidosis type IIIA (MPS-IIIA).

Neurometabolic disorders such as MLD can have a devastating, lifelong impact, not only on children but on their caregivers, support systems and the broader community, said Mark Rothera, president and chief executive officer of Orchard. We look forward to showcasing both real-world and clinical study data from our neurometabolic portfolio at the upcoming WORLD Symposium as we strive to bring about a brighter future for all those affected by rare disease.

The presentations are listed below and the full preliminary program is available online at the conference website.

Oral presentation details:

Case report of the first patient treated with ex-vivo autologous haematopoietic stem cell gene therapy transplant in mucopolysaccharidosis type IIIA*Presenter: Jane Kinsella, Royal Manchester Childrens HospitalSession: Translational Research IIDate: Wednesday, February 12Time: 9:15-9:30 a.m. ET

Lentiviral hematopoietic stem and progenitor cell gene therapy (HSPC-GT) for metachromatic leukodystrophy (MLD): Clinical outcomes from 33 patientsPresenter: Francesca Fumagalli, San Raffaele Telethon Institute for Gene TherapySession: Clinical Trials II: Clinical OutcomesDate: Thursday, February 13Time: 8:15-8:30 a.m. ET

Poster presentation details:

Lentiviral hematopoietic stem and progenitor cell gene therapy (HSPC-GT) for metachromatic leukodystrophy (MLD): Clinical outcomes from 33 patientsPoster abstract #: P126Presenter: Francesca Fumagalli, San Raffaele Telethon Institute for Gene TherapySession: Poster Reception (Exhibit Hall)Date: Monday, February 10Time: 4:30-6:30 p.m. ET

Caregiver-reported impact on quality of life and disease burden in patients diagnosed with metachromatic leukodystrophy: Results of an online survey and a qualitative interviewPoster abstract #: P320Presenter: Francis Pang, Orchard TherapeuticsSession: Poster Reception (Exhibit Hall)Date: Tuesday, February 11Time: 4:30-6:30 p.m. ET

Extensive metabolic correction of mucopolysaccharidosis type I (MPS IH, Hurler syndrome) by hematopoietic stem and progenitor cell (HSPC) based gene therapy (GT): Preliminary results from a phase I/II trialPoster abstract #: LB-15Presenter: Francesca Tucci, San Raffaele Telethon Institute for Gene TherapySession: Poster Reception (Exhibit Hall)Date: Wednesday, February 12Time: 4:30-6:30 p.m. ET

About OrchardOrchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically-modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. The company has one of the deepest gene therapy product candidate pipelines in the industry and is advancing seven clinical-stage programs across multiple therapeutic areas, including inherited neurometabolic disorders, primary immune deficiencies and blood disorders, where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Forward-Looking StatementsThis press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, the therapeutic potential of Orchards product candidates, including the product candidate or candidates referred to in this release, Orchards expectations regarding the timing of regulatory submissions for approval of its product candidates, including the product candidate or candidates referred to in this release, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates, and the likelihood of approval of such product candidates by the applicable regulatory authorities. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the risk that any one or more of Orchards product candidates, including the product candidate or candidates referred to in this release, will not be approved, successfully developed or commercialized, the risk of cessation or delay of any of Orchards ongoing or planned clinical trials, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates, the delay of any of Orchards regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates, the receipt of restricted marketing approvals, and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

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Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards annual report on Form 20-F for the year ended December 31, 2018, as filed with the U.S. Securities and Exchange Commission (SEC) on March 22, 2019, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

Source: Orchard Therapeutics (Europe) Limited

*Patient was treated by the Royal Manchester Childrens Hospital (RMCH) under a Specials license, granted by the UK government for the use of an unlicensed pharmaceutical product in situations of high unmet need when there is no other treatment option available. Orchard holds the license to the MPS-IIIA investigational gene therapy product (OTL-201) and is funding the proof-of-concept clinical trial being conducted at RMCH, which utilizes the same technology and procedures that were used to treat this first MPS-IIIA patient.

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Orchard Therapeutics Announces Presentation of Clinical Data from Neurometabolic Franchise at 16th Annual WORLD Symposium - Yahoo Finance

Recommendation and review posted by Bethany Smith

NEW YORK, Feb. 4, 2020 /PRNewswire/ --Hoth Therapeutics, Inc. (NASDAQ:HOTH), a biopharmaceutical company focused on unique targeted therapeutics for patients suffering from dermatological indications ranging from atopic dermatitis, psoriasis and acne along with gene therapy treatment for asthmatics, is pleased to announce the initiation of a preclinical study for the treatment of asthma and allergic inflammation in collaboration withNorth Carolina State University(NC State).

The study has begun thedelivery and distribution of nebulized particleswhich willenable the therapeutic oligonucleotide (oligo), short DNA and RNA molecules that have a wide range of applications in gene testing.Hoth has appointed Dr. Glenn Cruse to its Scientific Advisory Board and will oversee the Company's gene therapy programs advancements.

Mr.Robb Knie, Chief Executive Officer of Hoth Therapeutics, Inc. commented,"We are extremely pleased that our gene therapy program with NC State has officially begun and that Dr. Cruse who is overseeing the advancement ofexperimentshas joined our Scientific Advisory Board. Commencement of this initiative is an important step in the development and growth of our company. Dr. Cruse's expertise asa leading mast cell biologist in allergic and inflammatory diseases will be invaluable for the preclinical development of Splice-switching oligonucleotides (SSOs) for asthma."

In November 2019 Hoth entered into a licensing agreement with North Carolina State University (NC State) to study NC State's Exon Skipping Approach for Treating Allergic Diseases. This Exon Skipping Approach was developed by Dr. Glenn Cruse, Principal Investigator and Assistant Professor in the Department of Molecular Biomedical Sciences at the NC State College of Veterinary Medicine. During Dr. Cruse's research, a new approach for the technique of antisense oligonucleotide-mediated exon skipping to specifically target and down-regulate IgE receptor expression in mast cells was identified. These findings set a breakthrough for allergic diseases as they are driven by the activation of mast cells and the release of mediators in response to IgE-directed antigens.

Glenn Cruse completed his Ph.D. at Glenfield Hospital, The University of Leicester, UK in 2009. He then moved to the National Institutes of Health in Bethesda, Maryland in January 2010 to start a visiting postdoctoral fellowship in the Laboratory of Allergic Diseases, NIAID, In January 2015, Dr. Cruse was appointed as a Research Fellow in the same laboratory. Dr. Cruse joined the Department of Molecular Biomedical Sciences at NC State in January 2016 as an Assistant Professor.

Dr. Cruse is a mast cell biologist that has authored and co-authored over 30 publications including articles in top journals such as the New England Journal of Medicine, Proceedings of the National Academy of Sciences USA and Immunity. The Cruse lab is interested in the role that mast cells play in allergic and inflammatory diseases and identifying novel therapeutics that target mast cells. Since mast cells act as sentinel cells that participate in both innate and acquired immunity, particularly at biological barriers, emphasis on diseases in tissues at the interface with the environment such as the lung, skin, gastrointestinal tract and even the neuro-immune axis are the main focus of the lab.

About Hoth Therapeutics, Inc.Hoth Therapeutics, Inc. isa clinical-stage biopharmaceutical company focused on developing new generation therapies for dermatological disorders. HOTH's pipeline has the potential to improve the quality of life for patients suffering from indications including atopic dermatitis, chronic wounds, psoriasis, asthma and acne. To learn more, please visitwww.hoththerapeutics.com.

Forward Looking StatementsThis press release includes "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements in this press release include, but are not limited to, statements that relate to the advancement and development of the BioLexa Platform, the commencement of clinical trials, the availability of data from clinical trials and other information that is not historical information. When used herein, words such as "anticipate", "being", "will", "plan", "may", "continue", and similar expressions are intended to identify forward-looking statements. In addition, any statements or information that refer to expectations, beliefs, plans, projections, objectives, performance or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking. All forward-looking statements are based upon Hoth's current expectations and various assumptions. Hoth believes there is a reasonable basis for its expectations and beliefs, but they are inherently uncertain. Hoth may not realize its expectations, and its beliefs may not prove correct. Actual results could differ materially from those described or implied by such forward-looking statements as a result of various important factors, including, without limitation, market conditions and the factors described under the caption "Risk Factors" in Hoth's Form 10K for the period endingDecember 31, 2018, and Hoth's other filings made with the Securities and Exchange Commission. Consequently, forward-looking statements should be regarded solely as Hoth's current plans, estimates and beliefs. Investors should not place undue reliance on forward-looking statements. Hoth cannot guarantee future results, events, levels of activity, performance or achievements. Hoth does not undertake and specifically declines any obligation to update, republish, or revise any forward-looking statements to reflect new information, future events or circumstances or to reflect the occurrences of unanticipated events, except as may be required by law.

ContactsInvestor Relations Contact:Phone: (646) 756-2997Email:investorrelations@hoththerapeutics.comwww.hoththerapeutics.com

KCSA Strategic CommunicationsValter Pinto (212) 896-1254Hoth@kcsa.com

View original content to download multimedia:http://www.prnewswire.com/news-releases/hoth-initiates-preclinical-gene-therapy-program-with-nc-state-for-the-treatment-of-asthma-and-allergic-inflammation-300998521.html

SOURCE Hoth Therapeutics, Inc.

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Hoth Initiates Preclinical Gene Therapy Program with NC State for the Treatment of Asthma and Allergic Inflammation - P&T Community

Recommendation and review posted by Bethany Smith

Biotech stocks recovered last week along with the broader markets, which bounced back from a China coronavirus-induced sell-off.

Big pharma earnings took the spotlight, with Merck & Co., Inc. (NYSE: MRK), Bristol-Myers Squibb Co (NYSE: BMY), Gilead Sciences, Inc. (NASDAQ: GILD) and AbbVie Inc (NYSE: ABBV) among the notable ones reporting in the week. Merck surprised the markets by announcing plans to spin-off its slow-growing Women's Health, Legacy Brands and Biosimilars business.

Here're the key biotech catalysts for the unfolding week.

Bio CEO & Investor Conference: Feb. 10-11 in New York16th Annual WORLDSymposium: Feb. 10-13 in Orlando, FloridaGuggenheim Healthcare Talks/Oncology Day: Feb. 13 in New YorkASCO 2020 Genitourinary Cancers Symposium: Feb. 13-15 in San Francisco, California

FDA's Tobacco Products Scientific Advisory Committee is scheduled to meet Friday to discuss the modified risk tobacco product applications submitted by 22nd Century Group Inc (NYSE: XXII) for VLN King and VLN Menthol King combusted, filtered cigarettes. The company projectsthese tobacco products to be "very low nicotine content" cigarettes.

Avrobio Inc (NASDAQ: AVRO) will present at the 2020 WORLDSymposium updates for its investigational programs in Fabry disease and cystinosis as well as data from the first clinical use of its plato gene therapy platform. At an analyst and investor event scheduled for Monday, the company will present updates on Phase 1/2 clinical trial of AVR-RD-04, an investigational gene therapy for cystinosis, and Phase 2 study of AVR-RD-01 in Fabry disease.

Regenxbio Inc (NASDAQ: RGNX) will make poster presentation of interim data from the Phase 1/2 trial of its investigational gene therapy RGX-121 for the treatment of Mucopolysaccharidosis Type II. The presentation is scheduled for Wednesday.

ASCO 2020 Genitourinary Cancers Symposium Presentations

Exelixis, Inc. (NASDAQ: EXEL): Phase 1b data for cabozantinib and Roche Holdings AG Basel ADR Common Stock's (OTC: RHHBY) Tecentriq in solid tumors (Thursday)

Advaxis, Inc. (NASDAQ: ADXS): Phase 1/2 data for ADXS-PSA and Merck's Keytruda from the KEYNOTE-046 study in castrate-resistant prostate cancer (Thursday)

TrovaGene Inc (NASDAQ: TROV): new Phase 2 data for onvansertib and zytiga in prostate cancer (Thursday)

Corvus Pharmaceuticals Inc (NASDAQ: CRVS): updated Phase 1b/2 data for CPI-444 in solid tumors

See Also: 13 Key NASH Drug Candidates To Watch For In A Potential $30-Billion Market

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Revolution Medicines, a biotech developing targeted cancer therapies using RAS pathway inhibitors, has filed to offer 10 million shares in an IPO, which is expected to be priced between $14 and $16. The company expects its shares to be listed on the Nasdaq under the ticker symbol "RVMD."

IPO Quiet Period Expiry

I-Mab (NASDAQ: IMAB)

2020 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

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The Week Ahead In Biotech: SMID-Cap Earnings In The Spotlight - Benzinga

Recommendation and review posted by Bethany Smith

PLYMOUTH MEETING, Pa., Feb. 10, 2020 /PRNewswire/ -- Inovio Pharmaceuticals, Inc. (NASDAQ: INO) today announced the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application to evaluate its DNA medicine INO-3107 in a Phase 1/2 trial for treatment of Recurrent Respiratory Papillomatosis or RRP. RRP is a rare disease caused by the human papillomavirus (HPV) types 6 and 11 infections, a condition that causes noncancerous tumor growths leading to life-threatening airway obstructions, and occasionally can progress to cancer. Currently, the disease is incurable and is mostly treated by surgery, which temporarily restores the airway. The tumor almost always recurs and the surgery must be repeated, often multiple times a year. RRP can severely impact the quality of life for those living with the disease.

The open-label, multicenter Phase 1/2 trial will enroll approximately 63 subjects in the U.S. and will evaluate the efficacy, safety, tolerability, and immunogenicity of INO-3107 in subjects with HPV 6 and/or 11-associated RRP who have required at least two surgical interventions per year for the past three years for the removal of associated papilloma(s). For this study, adult subjects will first undergo surgical removal of their papilloma(s) and then receive four doses of INO-3107, one every three weeks. The primary efficacy endpoint will be a doubling or more in the time between surgical interventions following the first dose of INO-3107 relative to the frequency prior to study therapy. Upon obtaining sufficient safety and potential efficacy data in adults, Inovio plans to expand the trial to include pediatric patients as well as a potential booster regimen.

"Inovio's investigational DNA medicine INO-3107 is designed to destroy and clear tumors caused by HPV 6 and 11 infections from the body exactly where they are hiding," said Jeffrey Skolnik, M.D., Inovio's Vice President of Clinical Development. "We believe this DNA medicine has the potential to provide people living with RRP a long-term, if not life-long, improvement in their disease, especially as an alternative to often successive and debilitating surgeries that may temporarily remove HPV growths from the airways but do not address the underlying recurring virus."

J. Joseph Kim, Ph.D., Inovio's President and Chief Executive Officer, said "Our mission at Inovio is to rapidly provide patients with urgent health needs access to our novel DNA medicines. We are pleased the FDA has authorized our INO-3107 clinical trial, and look forward to working closely with the RRP patient and medical community to drive recruitment as quickly as possible."

In addition to initiating this efficacy trial, Inovio also plans to attain Orphan Disease designation with the FDA's Office of Orphan Products Development (OOPD). The FDA grants orphan status to drugs and biologic products that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States. OOPD provides a drug developer with certain benefits and incentives, including a period of marketing exclusivity if regulatory approval is ultimately received for the designated indication.

Inovio recently published data from its pilot clinical study of INO-3106 (DNA medicine candidate targeting HPV6 caused RRP) in the scientific journal Vaccines (MDPI). Study results demonstrated that INO-3106 generated immunogenicity and engagement and expansion of an HPV 6-specific cellular response, including cytotoxic T cells. The paper also showed that Inovio's immunotherapy allowed two out of two patients who previously required approximately two surgeries per year for several years to manage this disease to delay the need for surgery to a robust degree; with one patient able to delay surgery for over a year and a half (584 days surgery-free) and a second that remained surgery-free for over two and a half years (over 915 days surgery-free).

About Inovio's DNA Medicines

Inovio has 15 DNA medicine clinical programs currently in development focused on HPV-associated diseases, cancer, and infectious diseases, including the novel coronavirus (2019-nCoV) under a grant from the Coalition for Epidemic Preparedness Innovations (CEPI). DNA medicines are medicines composed of optimized DNA plasmids, which are small circles of double-stranded DNA that are synthesized or reorganized by a computer sequencing technology and designed to produce a specific immune response in the body.

Inovio's DNA medicines deliver optimized plasmids directly into cells intramuscularly or intradermally using Inovio's proprietary hand-held smart device called CELLECTRA. CELLECTRA uses a brief electrical pulse to open small pores in the cell reversibly to allow the plasmids to enter. Once inside the cell, the plasmids begin replicating, thereby strengthening the body's own natural response mechanisms. Administration with the CELLECTRA device ensures that the DNA medicine is delivered directly into the body's cells, where it can go to work immediately mounting an immune response. Inovio's DNA medicines are not interfering with or changing in any way an individual's own DNA, which is the case with gene therapy or gene editing.

With more than 2,000 patients receiving Inovio investigational DNA medicines in more than 6,000 applications across a range of clinical trials, Inovio's DNA medicines have consistently activated safe, robust, and fully functional T cell and antibody responses against targeted pathogens and cancers.

About RRP

Recurrent respiratory papillomatosis (RRP) is a rare disease (estimated at 15,000 active cases in the U.S.) that is characterized by the growth of tumors in the respiratory tract caused by the human papillomavirus. Although benign, papillomas can cause severe, even life-threatening airway obstruction and respiratory complications. A distinguishing aspect of this disease is the tendency for the papilloma to recur after surgical procedures to remove them. Left untreated, if RRP develops in the lungs, affected individuals can potentially experience recurrent pneumonia, chronic lung disease (bronchiectasis) and, ultimately, progressive pulmonary failure. In extremely rare cases (less than 1%), papillomas can become cancerous (malignant transformation) developing into squamous cell carcinoma. Additional symptoms of RRP can include hoarse voice, difficulty in sleeping and swallowing, and chronic coughing. RRP symptoms are usually more severe in children than in adults. In children, the disorder is most often diagnosed at or around the age of four years. In adults, the disorder occurs most often in the third or fourth decade.

About Inovio Pharmaceuticals, Inc.

Inovio is a biotechnology company focused on rapidly bringing to market precisely designed DNA medicines to treat, cure, and protect people from diseases associated with HPV, cancer, and infectious diseases. Inovio is the first and only company to have clinically demonstrated that a DNA medicine can be delivered directly into cells in the body via a proprietary smart device to safely produce a robust immune response to destroy and clear high-risk HPV 16 and 18, which are responsible for 70% of cervical cancer, 90% of anal cancer and 69% of vulvar cancer. In addition to HPV, Inovio's optimized plasmid design and delivery technology have been demonstrated to consistently activate robust and fully functional T cell and antibody responses against targeted cancers and pathogens. Inovio's most advanced clinical program, VGX-3100, is in Phase 3 development for the treatment of HPV-related cervical pre-cancer. Also in development are Phase 2 immuno-oncology programs targeting HPV-related cancers and GBM, as well as externally funded vaccine development programs in Zika, MERS, Lassa, HIV, and the novel coronavirus (2019-nCoV). Partners and collaborators include ApolloBio Corporation, AstraZeneca, The Bill & Melinda Gates Foundation, Coalition for Epidemic Preparedness Innovations (CEPI), Defense Advanced Research Projects Agency, GeneOne Life Science, HIV Vaccines Trial Network, Medical CBRN Defense Consortium (MCDC), National Cancer Institute, National Institutes of Health, National Institute of Allergy and Infectious Diseases, Regeneron, Roche/Genentech, University of Pennsylvania, Walter Reed Army Institute of Research, and The Wistar Institute. For more information, visit http://www.inovio.com.

This press release contains certain forward-looking statements relating to our business, including our plans to develop DNA medicines, our expectations regarding our research and development programs, as well as commercialization activities, including the planned initiation and conduct of clinical trials, the availability and timing of data from those trials and our commercialization strategy and tactics. Actual events or results may differ from the expectations set forth herein as a result of a number of factors, including uncertainties inherent in pre-clinical studies, clinical trials, product development programs and commercialization activities and outcomes, the availability of funding to support continuing research and studies in an effort to prove safety and efficacy of electroporation technology as a delivery mechanism or develop viable DNA vaccines, our ability to support our pipeline of SynCon active immunotherapy and vaccine products, the ability of our collaborators to attain development and commercial milestones for products we license and product sales that will enable us to receive future payments and royalties, the adequacy of our capital resources, the availability or potential availability of alternative therapies or treatments for the conditions targeted by us or our collaborators, including alternatives that may be more efficacious or cost effective than any therapy or treatment that we and our collaborators hope to develop, issues involving product liability, issues involving patents and whether they or licenses to them will provide us with meaningful protection from others using the covered technologies, whether such proprietary rights are enforceable or defensible or infringe or allegedly infringe on rights of others or can withstand claims of invalidity and whether we can finance or devote other significant resources that may be necessary to prosecute, protect or defend them, the level of corporate expenditures, assessments of our technology by potential corporate or other partners or collaborators, capital market conditions, the impact of government healthcare proposals and other factors set forth in our Annual Report on Form 10-K for the year ended December 31, 2018, our Quarterly Report on Form 10-Q for the quarter ended September 30, 2019, and other filings we make from time to time with the Securities and Exchange Commission. There can be no assurance that any product candidate in our pipeline will be successfully developed, manufactured or commercialized, that final results of clinical trials will be supportive of regulatory approvals required to market products, or that any of the forward-looking information provided herein will be proven accurate. Forward-looking statements speak only as of the date of this release, and we undertake no obligation to update or revise these statements, except as may be required by law.

View original content:http://www.prnewswire.com/news-releases/inovio-receives-authorization-from-the-us-fda-to-begin-phase-12-clinical-trial-for-ino-3107-a-dna-medicine-to-treat-a-rare-disease----recurrent-respiratory-papillomatosis-rrp-301001634.html

SOURCE Inovio Pharmaceuticals, Inc.

Company Codes: NASDAQ-NMS:INO

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Inovio Receives Authorization from the US FDA To Begin Phase 1/2 Clinical Trial for INO-3107, a DNA Medicine To Treat a Rare Disease -- Recurrent...

Recommendation and review posted by Bethany Smith

A new type of cancer cell therapy could avoid some of the serious side effects commonly associated with CAR-T treatments, and possibly offer an easier path to developing "off-the-shelf" treatments, suggest findings from a small study led by researchers at the MD Anderson Cancer Center in Houston, Texas.

The results, which were published Wednesday in the New England Journal of Medicine, are from just 11 patients. Other factors, such as the use of postremission therapy, limit what conclusions can be drawn about the researchers' approach, which relies on "natural killer" cells rather than the T cells used in cellular drugs like Novartis' Kymriah.

Still, the data offer a glimpse into why Japanese drugmaker Takedaagreed last November to license the CAR NK cell therapy from MD Anderson, part of the company's broader push into cell and gene treatments. Some of the data published Wednesday was previously disclosed by the pharma.

The success of cancer immunotherapy, of which CAR-T treatments are a major part, has put T cells at the center of a now decade-long research revival in oncology.

But T cells are only one component of the body's immune system, and scientists in academia and in biotech are exploring whether other cellular defenders could be similarly recruited.

Researchers at MD Anderson have turned to natural killer cells, which by design recognize and attack cancers or other invaders. Such cells have been tested as an anti-cancer treatment before,but using genetic engineering to improve their tumor-killing properties, which the MD Anderson team has done, is a newer innovation.

"To my knowledge, this is the largest body of evidence on the use of CAR NK cells in patients with cancer," said Katayoun Rezvani, the study's corresponding author and a professor of stem cell transplantation and cellular therapy at MD Anderson, in an interview.

Using NK cells derived from cord blood, Rezvani and her colleagues engineered the cells to express a receptor for a protein called CD19, commonly found on the surface of B-cell malignancies like leukemia and lymphoma. They also added a gene for interleukin-15 to boost the expansion and persistence of the infused NK cells, which without engineering would typically disappear after about two weeks.

While the CAR-T treatments Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel) also target CD19, they are made from a patient's own T cells, which are extracted and then engineered outside the body. The personalized process is time-consuming and laborious, hampering the commercial uptake of both Kymriahand Yescarta.

By using cord blood, Rezvani and her team are pursuing an allogeneic, or "off-the-shelf," approach to cell treatment something many consider to be the next step for the field.

Initial data look promising. Seven of the 11 treated patients, who had either chronic lymphocytic leukemia or non-Hodgkin lymphoma, responded to treatment, with the cancers of three going into remission.Most notably, none experienced cytokine release syndrome or neurotoxicity, two severe side effects that commonly occur in patients treated with CAR-T therapy.

"The lack of toxicity is very exciting here," wrote Stephan Grupp, an oncologist at Children's Hospital of Philadelphia and a leader in the CAR-T field, in comments emailed to BioPharma Dive. He was not involved with the MD Anderson study.

"We really think that this is something inherent to the biology of the natural killer cells, which means their profile of toxicity is different than that of T cells,"Rezvanisaid.

Study participants did have blood toxicities that researchers associated with the chemotherapy given prior to infusion of the CAR NK cells.

While positive, the results are limited by several factors which make drawing broader conclusions about the ultimate potential of the treatment difficult.

Five of the seven responding patients received postremission treatment, including stem cell transplants, Rituxan (rituximab) and Revlimid (lenalidomide), so researchers did not assess the duration of response to CAR NK therapy.

Additionally, a fresh CAR NK cell product was manufactured for each patient in this study, rather than using the cord blood to produce multiple therapies as would be envisioned for a true off-the-shelf product.

"I think the potential for this approach to be 'off-the-shelf' is also a little speculative at this time," wrote Grupp.

"We would need to see multiple patients treated from the same expanded product with no HLAmatching to know if 'off-the-shelf' is going to be part of the story here," he added, referring to the process by which patients are matched to donor cells.

If cord blood-derived CAR NK cells were able to be given without matching to a patient's HLA genotype, any resulting treatment could be used more widely. Nine patients were partially matched in the MD Anderson study, while the last two were treated without consideration of HLA type.

The MD Anderson researchers plan to continue enrolling patients in the study and are working with Takeda to design a larger, multi-center trial.

The drugmaker is planning to advance the treatment, which it licensed and now calls TAK-007, into pivotal studies in two types of lymphoma and CLL by 2021, with a potential filing for approval in 2023.

"Targeting CD19 was a proof of concept and now that we've demonstrated that this CAR NK approach can work and is safe we want to use this platform to target other types of cancers," said Rezvani, indicating interest in multiple myeloma and acute myeloid leukemia.

See more here:
In small study, hints of promise for 'natural killer' cell therapy - BioPharma Dive

Recommendation and review posted by Bethany Smith

Although vaping-related lung illnesses are declining, more than 2,700 have been reported across the U.S. and its territories since last March, according to the CDC. A total of 60 have died. Public health news is on a rare neuromuscular disease, cervical cancer, obesity, preparing for civilization's end, longevity, vocal disorders, postpartum depression, organ transplants, a sweet substitute for kids' cough medicines, Alzheimer's disease, mental health, and medical errors, as well.

The Wall Street Journal:Vaping-Related Deaths Fall, But Families Still Look For AnswersKimberly Boyd keeps a stack of her sons medical files on her dining-room table, in neatly organized folders. In a Ziploc bag, there are some of the nicotine vaping cartridges he used at their Orlando, Fla., home before the 28-year-old died in November. Across the country, in Seattle, Robin Hurt is waiting for a response to a public-record request she filed with the states medical examiner in Oregon, asking for the autopsy report on her 23-year-old grandson, who unexpectedly died in October after having recently taken up vaping. (Ansari, 2/9)

Stat:Lottery Like No Other Offers A Cutting-Edge Medicine With Lives On The LineThe treatment, a gene therapy called Zolgensma, is designed for children like Wynter who have a neuromuscular disease called spinal muscular atrophy, or SMA. Without it or other treatments, those with the most serious type are likely to die as babies. It was first approved by U.S. regulators only last year, and is not yet available in other countries. The lottery was devised by the drugs manufacturer, Novartis, to give families in those places a chance to get it through a novel form of compassionate use a way to get medications that have not been approved while they wait. Fifty doses are slotted to be given away for free in the first half of the year, with up to 100 total. The first drawing occurred Monday. (Joseph and Silverman, 2/7)

The Washington Post:WHOs Aggressive, Three-Part Strategy Aims To Make Cervical Cancer A Thing Of The PastIn just 35 years, the United States managed to reduce cervical cancer rates by 54 percent with the help of Pap smears. Now, human papillomavirus vaccination, double screening and more effective treatment might be able do away with the cancer. In two new studies in the Lancet, the World Health Organization lays out how. The studies model what might happen if the United Nations health agency commits to a three-part strategy to wipe out cervical cancer. (Blakemore, 2/8)

The New York Times:Half Of Us Face Obesity, Dire Projections ShowClimate change is not the only source of dire projections for the coming decade. Perhaps just as terrifying from both a health and an economic perspective is a predicted continued rise in obesity, including severe obesity, among American adults. A prestigious team of medical scientists has projected that by 2030, nearly one in two adults will be obese, and nearly one in four will be severely obese. (Brody, 2/10)

WBUR:Preparing For The End Of The World, On A BudgetA Harvard Ph.D. and former military intelligence officer with 30 years of experience, Miller would know a good defensible spot when he sees it. Miller is a self-described "prepper," someone who makes active preparations to survive the fall of human civilization. The nationwide prepper community is often painted as composed of conspiracy-crazed eccentrics, he said, thanks in large part to television shows such as the National Geographic Channel's Doomsday Preppers. (Boyce, 2/10)

The Washington Post:For Aging, Strength Training Is Vital In Avoiding Injuries And Staying IndependentWhen an intruder broke into the Rochester, N.Y., home of 82-year-old Willie Murphy a few months ago, he was met with a big surprise. Murphy, a diminutive but powerlifting woman, quickly jumped into action, using her strength to pummel the intruder with a broom and send him running for the door. Not surprisingly, the story went viral as people embraced the images of the elderly Murphy flexing her muscles for the cameras. (Loudin, 2/9)

Stat:The Vodka Trial: In Search Of A Treatment For Vocal Disorders, A Researcher Puts Patient Anecdotes To The TestPharmacists had prepared the therapy specifically for her, in little reddish bottles that reminded her of liquid penicillin. A research assistant gave her careful instructions. But Feeley already knew exactly what do to do. What do you do with a shot of vodka? Basically, you pour it down your neck. So I drank it and then banged the bottle on the table, Feeley said. Usually, shes more of a wine or daiquiri sort of person, but shed put aside her taste and a few days of her time to help answer a question that had been rattling around Simonyans mind. It had first appeared about 15 years ago, when Simonyan was doing neuroscience research in New York City, trying to understand exactly how the brain choreographs the intricate dance of muscle and air that gives rise to speech. (Boodman, 2/10)

CNN:Women Who Have General Anesthesia During C-Sections Are More Likely To Experience Postpartum Depression, Study FindsWomen who have general anesthesia during C-sections are significantly more likely to experience severe post-partum depression resulting in hospitalization, suicidal thoughts or self-harm, according to a study published last week. That might be because general anesthesia can delay breastfeeding and skin-to-skin interaction between the mother and infant, and often results in more acute and persistent pain after childbirth, researchers from Columbia University explained. "These situations are often coupled with a new mother's dissatisfaction with anesthesia in general, and can lead to negative mental health outcomes," said Jean Guglielminotti, lead author and an assistant professor of anesthesiology at Columbia, in a news release. (Kaur, 2/8)

Kaiser Health News:How Lifesaving Organs For Transplant Go Missing In TransitWhen a human heart was left behind by mistake on a Southwest Airlines plane in 2018, transplant officials downplayed the incident. They emphasized that the organ was used for valves and tissues, not to save the life of a waiting patient, so the delay was inconsequential. It got to us on time, so that was the most important thing, said Doug Wilson, an executive vice president for LifeNet Health, which runs the Seattle-area operation that processed the tissue. (Aleccia, 2/10)

NPR:A Cough Cure For Kids? Try HoneyIf you don't have little kids, or it's been a while, let me just break down for you why kids' coughs can be a truly miserable problem that can drive you to madness. Imagine this: Your kid's coughing it's almost always worse at night then they start crying because they're tired and can't sleep with all the coughing. The coughing and crying means that not only do they not sleep, but you also don't sleep no one in the house sleeps and this can go on for weeks. (Simmons-Duffin, 2/8)

Kaiser Health News:Finding Connections And Comfort At The Local CafeDoug and Connie Moore met at seminary. He was a student and pastor of an inner-city congregation, and she was a student and a public health nurse. Shes the one who drew me to the needs of the poor, Doug says. The pair wed in 1974, and Doug became a pastor at the First Evangelical Free Church of Los Angeles in 1983. They became deeply involved in their community and dedicated much of their free time to teaching English as a second language, creating tutoring programs and mentoring students in poor communities here and abroad. (De Marco, 2/10)

The Washington Post:How Anxiety And Midlife Crisis Are Playing Out Among Gen-X WomenThree years ago, Ada Calhoun couldnt sleep. The 41-year-old writer stared at her ceiling wondering why her hard-won accomplishments had left her feeling exhausted, anxious and wanting. She had written two well-regarded books and a long list of magazine articles. She was married and her son had just been accepted to a great public middle school. From the outside, she knew, her life looked good. (Schaaff, 2/8)

USA Today:Mom Of HBO's Bleed Out Filmmaker Steve Burrows Dies 11 Years After Hip Surgery, Brain DamageThe producer and director of an HBO documentary about his mother's medical errors at a Milwaukeehospital went to Capitol Hill lastweek to show clips of the film.Three nights later,Steve Burrows' mother, Judie, diedat 79 after an 11-year struggle to survive. Friday, the Milwaukee medical examiner's office saidher death was due to complications of repeat hip surgeries.(O'Donnell, 2/8)

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Investigation Into Vaping-Related Deaths Continues: With No Known Cause, Relatives Struggle To Find Out What Killed Family Members - Kaiser Health...

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With advances in modern medicine accelerating forward, demand for lab space and health-care facilities has also seen a rapid growth. In Cushman & Wakefields latest report on the life sciences industry, the sector has demonstrated steady and strong market fundamentals.

With an aging and growing population, the life sciences sector is continuously working on new technologies to improve quality of life. But to keep advancing, companies are seeking additional lab space and more employees. Combined with a steady flow of research capital, the life sciences sectors market fundamentals have made it more resilient to the cyclical nature of the real estate market.

READ ALSO: The Magnetism of Medical Office Buildings

Currently, there are 52.4 million people aged 65 and older in the U.S and by the next decade, that number is expected to exceed 73 million. Similarly, the report noted that the median age in the U.S. increased to 38.2 in 2018 and is expected to reach 40 by 2030. As the population grows, so does demand for new treatments, drugs and equipment.

With such inevitable demand, technological advances in life science categories like genomics, gene editing and cell and gene therapy have taken off. Specifically, the cell and gene therapy sector commanded a $1.1 billion market cap in 2018, but is expected to grow by 36.5 percent in the next five years and hit an $11 billion market cap. Similarly, both the genomics and gene editing sectors will also grow by more than 10 percent each in the next five years.

As these sectors grow, so does demand for employees. Starting from the end of 2013, the amount of jobs in the biotech research and development industry has increased by 70,000 each year, or roughly 7.5 percent. In the life sciences sector overall, there were 204,800 jobs in the industry in 2019, with 67 percent of them concentrated in 11 metropolitan life sciences hubs.

Considering the growing need for space and employees, rents have increased, while vacancy rates have declined throughout major markets. Throughout the U.S., vacancy rates dropped to 7.1 percent for lab space versus the 12.4 percent rate for office space. With lower vacancy rates, rent for lab space in the 12 most popular life sciences markets has increased to $43.10 per square foot, compared to office spaces average rent of $40.30 per square foot.

Certain markets have also developed into strong life sciences hubs, with areas like Boston/Cambridge having close to no vacancy. The markets of Boston/Cambridge and the San Francisco Peninsula offer the largest markets, with 21.8 million of life sciences space, but others like New Jersey, have also risen to the top due to its history of being home to major pharmaceutical manufacturers. Markets like Boston/Cambridge, the San Francisco Peninsula and Oakland/East Bay all experienced an explosive growth in their talent pools in the last decade.

Earlier this year, Boston Properties partnered with Alexandria Real Estate Equities to combine their nearby properties and develop their first life sciences real estate development in a San Francisco biotech campus.

Despite the last two economic downturns, the life sciences sectors employment has increased 87.9 percent since 2000. While the industry is still subject to the ups and downs of the market, the report noted that the life sciences market is more likely to weather the storm than other sectors.

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Behind the Projected Growth of Life Sciences in 2020 - Multi-Housing News

Recommendation and review posted by Bethany Smith

Facts and Factors Market Researchhas published a new report titled Gene Therapy Market By Type (Germ Line Gene Therapy and Somatic Gene Therapy), By Vector Type (Viral Vectors, Non-Viral Vectors, and Human Artificial Chromosome), and By Therapy Area (Cancer, Neurological Diseases, Infectious Diseases, Genetic Disorders, Rheumatoid Arthritis, and Others): Global Industry Perspective, Comprehensive Analysis, and Forecast, 2018 2027.

According to the report, the globalgene therapy market was valued at approximately USD 919 million in 2018 and is expected to reach a value of around USD 6,892 million by 2027, at a CAGR of around 25.1% between 2019 and 2027.

Gene therapy is the kind of experimental method that makes use of genes for treating or preventing disease by inserting foreign genetic material like DNA or RNA into the persons cells. Scientists are studying gene therapy for treating various kinds of immuno-deficiencies, Parkinsons disease, HIV, and cancer by using myriad approaches. Today, many of the approaches to gene therapy are undergoing most intensive & rigorously testing. This includes replacing the mutated gene causing disease with the healthy gene copy. Another approach includes knocking out or inactivating a mutated gene operating improperly. Yet another approach includes a new gene into the body to combat the disease.

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New product approval & commercialization to drive the market trends

Between the periods from 2012 to 2018, nearly five single-use gene treatments received approval from the U.S. FDA for treating a rare form of genetic disorders. Moreover, gene treatments that have received approval are being tested by pharmaceutical firms in the market. Apart from this, current approvals of gene therapy products across the U.S., as well as European countries for treating a plethora of life-threatening diseases, are anticipated to steer the growth of gene therapy industry over the forecast timeline. Moreover, gene therapy can also be used for treating neurodegenerative disorders like Alzheimer, amyotrophic lateral sclerosis, and spinal muscular atrophy.

Furthermore, many of the reputed pharma firms like Bristol-Myers Squibb, BioMarin, and Pfizer are investing massively into the research activities pertaining to gene therapy. Apart from this, a rise in the occurrence of cancer is prompting the demand to treat the disease. Gene therapy is one of the key treatment kinds that will propel the market growth over the forecast period. However, inadequate reimbursement policies pertaining to the one-time gene treatments will downgrade market expansion.

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In addition to this, conducting of randomized controlled trials can pose a threat to the expansion of the gene therapy industry as a result of the gene therapy features & projected patient population. Nevertheless, the ability of the gene therapy to eliminate the number of ailments with faulty or missing genes like hemophilia A will promote the market growth over the forecast period and thereby nullify the negative impact of hindrances on the business growth.

Somatic gene therapy to dominate the type segment

The growth of the segment over the forecast timeline is credited to the ability to treat the targeted cells in the patient population. The treatment is not passed to future generations and is restricted to only the patient who receives the somatic gene therapy. Moreover, it is used for treating a huge number of disorders like cystic fibrosis, cancer, and muscular dystrophy.

Cancer to lead the therapy area segment over the forecast period

The segmental expansion is attributed to a large number of pipeline drugs registered over the past few years along with increasing occurrence of cancer as a result of genetic changes.

Request Customized Copy of Report @ https://www.fnfresearch.com/customization/gene-therapy-market-by-type-germ-line-gene(We customize your report according to your research need. Ask our sales team for report customization).

North America to dominate the overall regional market share during the forecast timespan

North American market, which accrued revenue of USD 380 million in 2018, is set to contribute majorly towards the overall market revenue by 2027. The regional market surge is credited to robust healthcare amenities, high per capita healthcare spending, and improvement in the reimbursement policies.

The key players included in this market are Advanced Cell & Gene Therapy, Audentes Therapeutics, Benitec Biopharma, Biogen, Blubird Bio, Inc., Bristol-Myers Squibb Company, CHIESI Farmaceutici SPA, Eurofins Scientific, Geneta Science, Genzyme Corporation, Gilead, GlaxoSmithKline PLC, Human Stem Cells institute, Novartis AG, Orchard Therapeutics, Pfizer Inc., Sangamo therapeutics, Spark therapeutics, and Voyager Therapeutics.

Browse the fullGene Therapy Market By Type (Germ Line Gene Therapy and Somatic Gene Therapy), By Vector Type (Viral Vectors, Non-Viral Vectors, and Human Artificial Chromosome), and By Therapy Area (Cancer, Neurological Diseases, Infectious Diseases, Genetic Disorders, Rheumatoid Arthritis, and Others): Global Industry Perspective, Comprehensive Analysis, and Forecast, 2018 2027Report athttps://www.fnfresearch.com/gene-therapy-market-by-type-germ-line-gene

This report segments the gene therapy market as follows:

Global Gene Therapy Market: By Type Segment Analysis

Global Gene Therapy Market: By Vector Type Segment Analysis

Global Gene Therapy Market: By Therapy Area Segment Analysis

Global Gene Therapy Market: Regional Segment Analysis

About Us:

Facts & Factors is a leading market research organization offering industry expertise and scrupulous consulting services to clients for their business development. The reports and services offered by Facts and Factors are used by prestigious academic institutions, start-ups, and companies globally to measure and understand the changing international and regional business backgrounds. Our clients/customers conviction on our solutions and services has pushed us in delivering always the best. Our advanced research solutions have helped them in appropriate decision-making and guidance for strategies to expand their business.

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Global Gene Therapy Market Set to Grow USD 6892 Million By 2027 - TheInfobiz

Recommendation and review posted by Bethany Smith

In its recently added report by UpMarketResearch.com has provided unique insights about Gene Therapy Industry Market for the given period. One of the main objectives of this report is to categorize the various dynamics of the market and to offer latest updates such as mergers and acquisitions, various technological developments, new entrants in the market, which make an impact on different segments.

This Gene Therapy Industry Market report is based on synthesis, analysis, and interpretation of information gathered regarding the target market from various sources. Our analysts have analyzed the information and data and gained insights using a mix of primary and secondary research efforts with the primary objective to provide a holistic view of the market. In addition, an in-house study has been made of the global economic conditions and other economic indicators and factors to assess their respective impact on the market historically, as well as the current impact in order to make informed forecasts about the scenarios in future.

Request Exclusive Free Sample PDF Of This Report At https://www.upmarketresearch.com/home/requested_sample/92436

The Gene Therapy Industry Market report is a trove of information pertaining to the various aspects of this industry space. Encompassing the ongoing as well as forecast trends likely to fuel the business graph of the Gene Therapy Industry Market across various geographies, the report also provides details about the driving factors that would help propel this industry to new heights during the projected period. Alongside a collection of the driving parameters, the Gene Therapy Industry Market reports also include a spate of other dynamics pertaining to the industry, such as the nominal risks prevailing in this marketplace as well as the growth prospects that this business sphere has in the future.

Some of key competitors or manufacturers included in this report are:company 1company 2company 3company 4company 5company 6company 7company 8company 9

Gene Therapy Industry Market Drivers & Challenges:The report covers the major driving factors influencing the revenue scale of the market and details about the surging demand for the product from the key geological regions.The latest trends and challenges that prominent industry contenders could face are highlighted in the report.

For More Information on this report, Request Inquiry At https://www.upmarketresearch.com/home/enquiry_before_buying/92436

The significant applications and potential business areas are also added to this report.The technological advancements, value and volume governing factors are explained in detail. The pricing structures, raw material analysis, market concentration scenario are analysed. In-depth information on upstream raw materials sourcing, downstream buyers, raw materials cost, labour cost and industry chain view is presented.The report uses tools such as comparison tables, graphs, pie charts, progress charts, etc. to give a clear picture of the market growth. Additionally, an overview of each market segments such as product type, application, end users, and region are offered in the report.

Market Segmentation By Type: Type 1Type 2Type 3

Market Segmentation By Applications: Application 1Application 2Application 3

The Regions covered are:Asia-PacificNorth AmericaEuropeSouth AmericaMiddle East & Africa

To provide the clarified representation of the current and upcoming growth trends of the market, the report provides the execution and attributes of the Gene Therapy Industry Market that are analyzed on the basis of the qualitative and quantitative process. Through the report, one can be able to take quick and precise business decisions by getting familiar with every aspect of the market. The Gene Therapy Industry Market report represents the analyzed data through graphs, charts, and figures for less complexity and better understandability about the Gene Therapy Industry Market.

To conclude, the Gene Therapy Industry Market report will provide the clients with a high-yielding market analysis assisting them to understand the market status and come up with new market avenues to capture hold of the market share.

If you have any special requirements, please let us know and we will offer you the report at customized price.

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Table Of Contents:Chapter 1 Market OverviewChapter 2 Industry ChainChapter 3 Environmental AnalysisChapter 4 Market Segmentation by TypeChapter 5 Market Segmentation by ApplicationChapter 6 Market Segmentation by RegionChapter 7 Market CompetitiveChapter 8 Major VendorsChapter 9 Conclusion

To purchase this report, Visit: https://www.upmarketresearch.com/buy/gene-therapy-industry-market-research-report-2019

About UpMarketResearch:Up Market Research (https://www.upmarketresearch.com) is a leading distributor of market research report with more than 800+ global clients. As a market research company, we take pride in equipping our clients with insights and data that holds the power to truly make a difference to their business. Our mission is singular and well-defined we want to help our clients envisage their business environment so that they are able to make informed, strategic and therefore successful decisions for themselves.

Contact Info UpMarketResearchName Alex MathewsEmail Alex@UpMarketResearch.comOrganization UpMarketResearchAddress 500 East E Street, Ontario, CA 91764, United States.

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Global Gene Therapy Industry Market 2019 Trends, Market Share, Industry Size, Opportunities, Analysis and Forecast To 2025 - Chronicle 99

Recommendation and review posted by Bethany Smith

Facts and Factors Market Research has published a new report titled Plasmid Market By General Type (Conjugative and Non-Conjugative), by Specific Plasmid Types (F-Plasmids, Col Plasmids, Resistance Plasmids, Cryptic Plasmids, Degradative Plasmids, and Virulence Plasmids), and by Application (Transfection, Recombinant DNA Technology, Gene Therapy, and Others): Global Industry Perspective, Comprehensive Analysis, and Forecast, 2019 2027.

According to the report, the global plasmid market is anticipated to be valued approximately USD 89 million in 2018 and is projected to hit the revenue of around USD 447 million by 2027, at a CAGR of around 19.5% between 2019 and 2027.

Plasmids are small circular double-stranded DNA molecules having the ability to duplicate independently. Moreover, the plasmid is different from chromosomal DNA and do not depend on chromosomal DNA for duplication. Plasmids are also referred to as replicons and they are the units of DNA that are capable of replicating independently within a suitable host.

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Plasmids are primarily found in bacteria. However, they can also be detected in archaea and multicellular organisms. Plasmids generally carry minimum one gene. Most of the genes that plasmids carry benefit to host organisms.

Growing occurrence of cancer, genetic disorders, & contagious ailments to steer the market growth

Vectors are utilized in developing cancer treatments and this is likely to steer the growth of the plasmid market over the forecast timeline. In addition to this, continuous ongoing research targeting cancer and approvals of many of the biopharmaceutical medicines for treating cancer will impel the growth of the plasmid market over the forecast timeframe. Apart from this, the growing occurrence of chronic as well as contagious diseases producing escalating demand for improved therapies will proliferate the market scope over the forecast timespan.

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Moreover, escalating awareness about the gene treatment along with the acceptance of gene therapy for treating ailments like Alzheimer and attention deficit hyperactivity will amplify the market growth over the forecast timeline. Apparently, gene therapy has a high potential for treating genetic disorders, as well as chronic ailments like cancer, will further enlarge the business growth over the forecast timespan. Nevertheless, mutagenesis coupled with huge costs related to gene therapy can act as a restraining factor for the plasmid industry over the forecast timeframe.

F-plasmids to dominate the specific plasmid types segment

F-plasmids segment is predicted to accrue a revenue of nearly USD 124 million by 2027. Some of the plasmids like F-plasmids possess the genes referred to as transfer genes enabling conjugation and this is likely to drive the expansion of the segment over the forecast timeline.

Gene therapy segment to contribute majorly towards the application landscape

The growth of the gene therapy segment is credited to a rise in the investment in research activities pertaining to gene treatment. Apart from this, increase in the awareness about the gene therapy, government support, and adoption of gene therapy for treating cancer will amplify the expansion of the gene therapy segment over the period from 2019 to 2027.

Request Customized Copy of Report @ https://www.fnfresearch.com/customization/plasmid-market-by-general-type-conjugative-and-non(We customize your report according to your research need. Ask our sales team for report customization).

North America to dominate the overall regional market revenue share

North American market is likely to accrue a revenue of approximately USD 160 million by 2027. The growth of the market in the region is attributed to rising in the healthcare spending, approval of gene treatments for various chronic ailments, and rise in the investments in research activities pertaining to gene therapy applications in various domains.

Key players involved in the plasmid industry include Aldevron, GenScript, PlasmidFactory GmbH & Co. KG, VGXI, Inc., Oxford Genetics Ltd., Applied StemCell, Altogen Biosystems, Cobra Biologics, Copernicus Therapeutics, Inc., Takara Bio Inc., InvivoGen, Miltenyi Biotec, Medigene Sdn Bhd, MaxCyte, Inc., Mirus Bio LLC, MolMed S.p.A., GenePharma, and Polyplus Transfection.

Browse the fullPlasmid Market By General Type (Conjugative and Non-Conjugative), by Specific Plasmid Types (F-Plasmids, Col Plasmids, Resistance Plasmids, Cryptic Plasmids, Degradative Plasmids, and Virulence Plasmids), and by Application (Transfection, Recombinant DNA Technology, Gene Therapy, and Others): Global Industry Perspective, Comprehensive Analysis, and Forecast, 2019 2027Report athttps://www.fnfresearch.com/plasmid-market-by-general-type-conjugative-and-non

This report segments the plasmid market as follows:

Global Plasmid Market: By General Type Segment Analysis

Global Plasmid Market: By Specific Plasmid Types Segment Analysis

Global Plasmid Market: By Application Segment Analysis

Global Plasmid Market: Regional Segment Analysis

About Us:

Facts & Factors is a leading market research organization offering industry expertise and scrupulous consulting services to clients for their business development. The reports and services offered by Facts and Factors are used by prestigious academic institutions, start-ups, and companies globally to measure and understand the changing international and regional business backgrounds. Our clients/customers conviction on our solutions and services has pushed us in delivering always the best. Our advanced research solutions have helped them in appropriate decision-making and guidance for strategies to expand their business.

Contact Us:

Facts & Factors

Global Headquarters

Level 8, International Finance Center, Tower 2,8 Century Avenue, Shanghai,Postal 200120, ChinaTel: +86 21 80360450

Email:sales@fnfresearch.com

Web:https://www.fnfresearch.com

Read more:
Global Plasmid Market Will Reach USD 447 Million By 2027 - TheInfobiz

Recommendation and review posted by Bethany Smith

The Global Gene Therapy Market Is Expected To Reach Around USD 2,269 Million By 2024 Research Report provides the newest industry data and industry future trends, allowing you to identify the products and end users driving Revenue growth and profitability. A leading market research firm,Zion Market Researchadded industry report onGene Therapy Marketconsisting of 110+ pages with TOC (Table of Contents) including a list of tables & figuresduring the forecast period and Gene Therapy Market report offers a comprehensive research updates and information related to market growth, demand, opportunities in the global Gene Therapy Market.

FREE | Request Sample Report of Gene Therapy Market Report @https://www.zionmarketresearch.com/sample/gene-therapy-market

Our Free Complimentary Sample Report Accommodate a Brief Introduction of the research report, TOC, List of Tables and Figures, Competitive Landscape and Geographic Segmentation, Innovation and Future Developments Based on Research Methodology

The global Gene Therapy Market report offers an extensive analysis of the realistic data collected from the global Gene Therapy Market. It demonstrates major drifts and key drivers playing an important role in the growth of the global Gene Therapy Market during the foretold time. The report focuses on the analysis of the key features such as drivers, new development opportunities, and restraints influencing the expansion of the Gene Therapy Market for the forecasted period.

The report covers a detailed analysis of the development of the Gene Therapy Market for the upcoming time. The global Gene Therapy Market is segmented based on the various product categories, delivery channels, and applications.

Major Market Players Included in This Report:

UniQure N.V, Spark Therapeutics LLC, Bluebird Bio, Juno Therapeutics, GlaxoSmithKline, Celgene Corporation, Shire Plc, Sangamo Biosciences, Dimension Therapeutics

A complete value chain of the global Gene Therapy Market is emphasized in the global Gene Therapy Market report along with the review of the downstream and upstream components influencing the global Gene Therapy Market. It analyzes the expansion of every segment of the Gene Therapy Market. The data presented in the research report is collected from various industry organizations to estimate the development of each segment of the global Gene Therapy Market in the coming period.

The global Gene Therapy Market research report presents market dynamics and inclinations influencing the growth of the global Gene Therapy Market. It uses SWOT analysis to review the competitive players of the Gene Therapy Market. Furthermore, the report also includes a synopsis of the various business strategies of the key players of the Gene Therapy Market.

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Promising Regions & Countries Mentioned In The Gene Therapy Market Report:

The report focuses on the latest market trends and major growth opportunities assisting in the expansion of the global Gene Therapy Market. On the basis of topography, the global Gene Therapy Market is classified into Europe, North America, Latin America, Middle East & Africa, and the Asia Pacific.

The Gene Therapy Market report provides company market size, share analysis in order to give a broader overview of the key players in the market. Additionally, the report also includes key strategic developments of the market including acquisitions & mergers, new product launch, agreements, partnerships, collaborations & joint ventures, research & development, product and regional expansion of major participants involved in the market on the global and regional basis.

Browse Press Release:https://www.zionmarketresearch.com/news/gene-therapy-market

Following 15 Chapters represents the Gene Therapy Market globally:

Chapter 1,enlist the goal of global Gene Therapy Market covering the market introduction, product image, market summary, development scope, Gene Therapy Market presence;

Chapter 2,studies the key global Gene Therapy Market competitors, their sales volume, market profits and price of Gene Therapy Market in 2018 and 2026;

Chapter 3,shows the competitive landscape view of global Gene Therapy Market on the basis of dominant market players and their share in the market growth in 2018 and 2026;

Chapter 4,conducts the region-wise study of the global Gene Therapy Market based on the sales ratio in each region, and market share from 2018 to 2026;

Chapter 5,6,7,8 and 9demonstrates the key countries present in these regions which have revenue share in Gene Therapy Market;

Chapter 10 and 11describes the market based on Gene Therapy Market product category, a wide range of applications, growth based on market trend, type and application from 2018 to 2026;

Chapter 12shows the global Gene Therapy Market plans during the forecast period from 2018 to 2026 separated by regions, type, and product application.

Chapter 13, 14, 15mentions the global Gene Therapy Market sales channels, market vendors, dealers, market information and study conclusions, appendix and data sources.

Inquire more about this report @https://www.zionmarketresearch.com/inquiry/gene-therapy-market

Available Array of Customizations:

Country-level bifurcation of data in terms of Product Type (Concentration, Temperature, Combustion, Conductivity, and Others) and Application (Petrochemical, Metallurgy, Electricity, and Others) for any specific country/countries.

Expansion of scope and data forecasts until 2030

Company Market Share for specific country/countries and regions

Customized Report Framework for Go-To Market Strategy

Customized Report Framework for Merger & Acquisitions and Partnerships/JVs Feasibility

Customized Report Framework for New Product/Service Launch and/or Expansion

Detailed Report and Deck for any specific Company operating in Gene Therapy Market

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Originally posted here:
Global Gene Therapy Market Hits Record Value of USD 2269 Million By 2024 - Global Newspaper 24

Recommendation and review posted by Bethany Smith