Archive for October, 2020

Its this time of the year when those of us in northern temperate zones are spectators of a fascinating natural phenomenon the appearance of autumn leaf colors.

The leaves start to change colors as they age, or senesce. During this period a cluster of enzymes in the leaves start chewing up their green pigment, called chlorophyll. As the leaves become less green, their red, yellow, and orange pigments (known as anthocyanins and carotenes) begin to shine. However, exactly how chlorophyll breakdown works is still only partially understood.

While autumn leaves are a great example of chlorophyll breakdown, evergreen plants (those that remain green all year long), vegetables, and fruits also experience chlorophyll decay. However, this happens only under special conditions such when fruit ripen or when evergreen plants are deprived of nutrients and water.

Banana skin is one of the few fruits where chlorophyll degradation can be mapped under ultraviolet (UV) light. As bananas ripen, their skins lose their green color a sign of chlorophyll breakdown and form new pigments that fluoresce blue under the UV light. Scientists know that this new pigment, known as hypermodified fluorescent chlorophyll catabolite (hmFCC), is produced very briefly in most plants. Banana skins and grapevine leaves are known to produce hmFCCs for a longer period.

Recently scientists at Instituto de la Grasa, Spain analyzed devils ivy, an evergreen plant, in their quest to find signs of this fluorescent compound. They activated the aging process by starving the plant, and voila! Exposure to UV light produced the blue, fluorescent pigment hmFCC. The team also observed two other newly identified compounds that will provide more insights into the evergreen chlorophyll breakdown puzzle.

Although the actual benefit of producing this blue, fluorescent compound is still a mystery, scientists think that it could provide protection against radiation or function in communication between animals and plants.

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Scientists use 16 genes to distinguish between two types of lethal pancreatic cancer - Massive Science

Its one of the most important genes in biology: Sry, the gene that makes males male. Development of the sexes is a crucial step in sexual reproduction and is essential for the survival of almost all animal species.

Today in the journal Science, my international collaborators and I report the surprise discovery of an entirely new part of the Sry gene in mice a part we had no idea existed.

I co-discovered Sry in 1990. It is the gene on the Y (male) chromosome that leads to the development of male characteristics in mice, humans and most other mammals. Since then, Sry has been the subject of intense study worldwide because of its fundamental role in mammalian biology.

We have come to understand, in some detail, how Sry acts to trigger a cascade of gene activity that results in the formation of testes, instead of ovaries, in the embryo. Testes then stimulate the formation of other male characteristics.

But its clear we dont have all the answers just yet. Our results published today take us one step further in the right direction.

For 30 years, we have understood the Sry gene is made up of one exon, a segment of a gene used to code for amino acids, the building blocks of proteins. This can be compared to a computer file consisting of one contiguous block of data, on a hard disk.

Our newest research reveals theres actually a second exon in mouse Sry. This is like finding a whole new separate block of previously hidden data.

The mouse genome, like the human genome, has been extensively characterised due to the availability of advanced DNA sequencing and related technologies. Researchers commonly assume all the genes and all the parts of the genes have already been discovered.

But earlier this year, scientists in Japan uncovered what looked like a new piece of the Sry gene in mice. New sequencing approaches revealed what appeared to be two versions of Sry: a short, single-exon form and a longer, two-exon form. They called this two-exon version Sry-T.

They collaborated with my group at the University of Queensland and removed the new exon using CRISPR, a gene editing tool that lets researchers alter DNA precisely. Together we discovered this prevented Sry from functioning: XY mice (which would normally develop as males) developed as females instead.

Conversely, adding Sry-T to fertilised XX mouse eggs (which would normally develop as females) resulted in males.

On the left, an XY mouse lacking Sry-T that developed as female. On the right, an XX mouse carrying the Sry-T gene that developed as male. Makoto Tachibana, Osaka University, Author provided

Importantly, although human Sry does not have the added exon, our discovery may reveal new functions that might be shared between mouse and human Sry.

The DNA sequence of the new exon in Sry-T may point us towards discovering some of the genes and proteins that interact with Sry, something that has been elusive up till now.

And interactions we find in mice may also occur in humans. Studying what human Sry interacts with may help explain some cases of differences in human sex development, otherwise known as intersex development. This is a common but poorly understood group of mostly genetic conditions that arise in humans.

Intersex refers to people who are born with genetic, hormonal or physical sex characteristics that are not typically male or female. Shutterstock

Currently, we dont know the genetics behind a large proportion of intersex conditions. This is partly because we dont yet know all the genes involved in the human sex development pathway.

Scientifically, this discovery is a bit like discovering a new cell type in the body, or a new asteroid in the Kuiper belt. As with many scientific discoveries, it challenges what we thought we knew and raises many questions.

What is the function of the new exon in Sry-T?

Currently, we only have part of the answer. It turns out the first exon of Sry, the one we already knew about, contains instability sequences at its end. These are sequences that cause proteins to fray and degrade.

An important function of the newly discovered second exon is to mask the instability sequences, seal the end of the Sry protein and prevent it from degrading. In other words, this second exon is crucial to the development of male babies.

Whats more, this protection mechanism represents an unusual and intriguing evolutionary mechanism that has acted to help stop vulnerable Y-chromosome genes from literally falling apart.

But its early days yet. The challenge now is to understand whether there are more functions hidden within the newly discovered exon.

If so, this information may provide some of the missing links that have stood in the way of our full understanding of how Sry works at a molecular level and of how males and females come to be.

Peter Koopman, Professorial Research Fellow, The University of Queensland

This article is republished from The Conversation under a Creative Commons license. Read the original article.

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We Discovered a Missing Gene Fragment Thats Shedding New Light on How Males Develop - Gizmodo Australia

You wouldnt eat a food that shrinks your brain. You wouldnt drive a car that makes you sweat. You wouldnt buy a purse that worsens PMS. So why are we all so cool with stress being just a part of life? It doesnt have to be. And if you reduce stress, you will be better for it. Because the impact stress has on your body is remarkableand hazardous. Read on to discover 30 more things stress is doing to your bodyand to ensure your health and the health of others, dont miss these Sure Signs Youve Already Had Coronavirus.

1

Your head is more likely to throb when youre stressed, according to the Mayo Clinic, with a tension-type headache or even a migraine. And feeling under pressure is also likely to make your headaches worse.

2

Stress kicks your body into its fight or flight mode, ramping up the release of certain hormones and preparing you to deal with danger. The result can sometimes be hands that shake like maracas.

3

Were not kidding. When were stressed, our body releases the hormone cortisol, and in limited doses, it can actually be beneficial. But studiesincluding one at the University of California at Berkeleyhave shown that chronic stress actually decreases the weight and volume of the brain.

4

Stress can increase the production of stomach acid, leading to that annoying reflux, as acid irritates the esophagus. And if you already suffer from chronic heartburn, stress can make it worse. A study of nearly 13,000 sufferers published in Internal Medicine discovered that nearly half reported stress as the biggest factor that worsened symptoms.

5

Stress gets us all riled up and causes hyper-arousal, making it difficult to fall asleep, stay asleep and making the quality of our sleep worse.

Recommendation: The National Sleep Foundation suggests a cooling off period before bed time, which allows the brain to wind down. Two hours should do it. Put your work away, turn off the TV and grab a book or listen to music.

Story continues

RELATED: Doing This Just Minutes a Day Helps You Sleep Better

6

When youre stressed, the muscles responsible for breathing tense up, making it more difficult to catch your breath.

Recommendation: If you begin to feel panicky and short of breath, start by exhaling deeply, emptying your lungs. You can also try breathing through your nose, which automatically slows your breathing.

7

If it seems like youre more likely to be sick when youre stressed, you may not be imagining it. Studies have shed light on the link between stress and sickness, finding that those living with chronic stress (such as unemployment or caregiving to a dementia patient) had a suppressed immune system that left them more vulnerable to the flu and a host of other illnesses.

RELATED: 20 Reasons Why You Keep Getting Sick

8

When were under stress, our hearts beat faster to help blood reach our vital organs. Often its harmless, but it may not be for those suffering chronic stress. One study from the European Society of Cardiology found that people with stressful jobsnurses or bus drivers, for examplehad a 48% higher risk of atrial fibrillation, a condition marked by an irregular, often rapid heart beat.

9

Stress has been shown to cause reproductive problems in both men and women. In one study published in Fertility and Sterility, researchers tested 274 women who were trying to get pregnant and found that those with higher levels of a particular enzyme in their saliva correlated to stress had a 12% more difficult time getting knocked up.

10

Erectile dysfunction is complicated and can have physical as well as psychological causes. Science, however, has shown over and over that stress tends to make the condition worse by releasing more adrenaline and causing exaggerated contractions of the muscles in the penis, keeping it from filling with blood.

11

Research has shown that various kinds of stress can wreak havoc on a womans period, making it irregular or disappear altogether. And to make matters worse, the Eunice Kennedy Shriver National Institute of Child Health and Human Development studied 259 women and found that stress can also make PMS pain worse.

12

And its true in both men and women. The causes, a study shows, can be both physical and psychological. Stress causes hormonal changes in the body, which arent particularly conducive for getting it on. It also makes someone distracted, and when their mind is on something else, sex can take a back seat.

Recommendation: One way to break the no-sex cycle is to get more physical with your partner, according to the Gottman Institute. It simply forces the body to go from stress to relaxation, if you allow this. Kiss your stressed out partner a little bit more and hug them for 20 seconds longer.

13

Stress gets your heart pumping faster and spikes your blood pressure. Not good. Usually, the response in temporary, a reaction to a particular stressful event. But chronic stress over long periods of time can cause inflammation in the arteries, which could lead to a heart attack down the road.

Recommendation: Try some good ol fashioned exercise. Working out three to five times a week can reduce your stress and will make a difference long-term in lowering your blood pressure, according to the Mayo Clinic.

14

When youre stressed, your body behaves as if its under attack, and your liver reacts by releasing more glucose into your bloodstream. Ongoing stress can lead to long-term sugar spikes, putting you at risk of type 2 diabetes, say experts.

15

Your gut has the most nerves in your body, this side of your brain, and stress can adversely affect your entire digestive system. The hormones released when youre stressed can interfere with digestion and harm the microorganisms living in your digestive tract. Cue indigestion, cramps, nausea and a whole host of other GI issues.

16

That tightening can cause back aches and other ailments.

Recommendation: Next time youre feeling stressed, reach for the walnuts. Researchers have shown that foods containing polyunsaturated fats, like the nuts, may help us deal better with stress.

RELATED: This Can Be the First Sign of COVID, Study Finds

17

Stress can lead to a dry mouth in several ways, say experts. Anxious people tend to breathe through their mouths, drying out the inside. The acid reflux associated with stress can also have an affect on the salivary glands and keep them from producing as much.

18

Chronic stress can leave your feeling zapped of energy. It could be the accompanying insomnia, or some theorize that it might have something to do with exhausting your adrenal glandthough a 2016 review of the research debunked that diagnosis.

19

We sweat more when were stressed and we sweat differently, studies show. Anxiety causes sweat to be produced from the apocrine glands, which secrete a thicker, milkier sweat than our eccrine glands. The downside: sweat from the apocrine glands tends to stink more.

20

Getting headaches is one thing, but actually altering your genetic code? Yep. Researchers at the University of Copenhagen have found that stress can switch on genes that werent supposed to be switched on. The consequence is that genes that should be turned off are now active and this may disturb cellular development, identity and growth, the researcher said.

RELATED: The Scariest New Symptoms of COVID-19

21

A study at Johns Hopkins concluded that long-term stress may affect the way that the genes controlling mood and behavior are expressed, leading a stressed-out person to be at a higher risk of depression. Tests on mice found that stress led to an increase in a protein produced by a gene called Fkbp5, which in humans has been linked to depression and bipolar disease.

22

Are the strains and demands of modern society commonly exceeding human ability? Thats the question asked by the authors of this study of more than 17,000 working adults in Stockholm, Sweden. The scientists found that even mild stress can lead to long-term disability or an inability to work. Those who experienced mild stress were 70% more likely to collect disability benefits.

23

Watch out. Men who are moderately or highly stressed for a number of years were found to have a 50 percent higher mortality rate, according to the Journal of Aging Research. The good news is that two very stressful situations a year might actually be beneficial, teaching us to cope with adversity. Anything beyond that, however, and it might be an early grave.

24

There may be something to that cliche about stress eating. Researchers at University College London found that feeling stressed changes what we eat. Those under pressure didnt necessarily eat more, but they did reach for more sweet, fatty foods than usual.

Recommendation: If youre feeling stressed, be more aware of what youre eating and try to curb your consumption of junk foodno matter how much your brain is screaming that you need it. For food solutions, visit eatthis.com.

25

One of those areas of the brain that gets shrunken by stress is the hippocampus, which plays a big part in learning and memory. A 2018 study found that those with higher levels of the stress hormone cortisol did worse on memory tests, especially women.

26

Research has shown that periods of mild to moderate stress may actually help the brain to better encode memories and improve learning. For example, a college student freaked out about an upcoming midterm may actually retain the material better and ace the test.

RELATED: 38 Ways Youre Treating Your Heart Wrong

27

In addition to generally suppressing the immune system, stress can lead to the growth of malignant cells, making a cancerous tumor bigger, says one study.

28

Children of parents with drinking problems face a greater risk of turning to booze after experiencing stressful situations, a University of Gothenburg study found.

Recommendation: If alcohol relaxes you when youre stressed, then you should try to find other ways of calming yourself downrelaxation exercises, for example, the researcher suggests.

29

Tinnitus, that same kind of annoying ringing you get after, say, sitting through a Metallica concert, might just be induced by stress. One study by the Egypts Minia University found that those suffering from chronic ringing tended to be more stressed. In short, There is a direct correlation between duration of tinnitus and severity of stress.

30

It doesnt get more serious than that. Severe mental stress can bring on sudden cardiac death, as medical professionals colorfully call it. In particular, suffering through a traumatic event, such as an earthquake or a war strike, can be so stressful that people literally keel over.

Recommendation: Dont worry. No ones ever died of a panic attack. If youre experiencing anxiety, dont miss these tips.

As for yourself: To get through this pandemic at your healthiest, dont miss these 35 Places Youre Most Likely to Catch COVID.

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30 Things Stress Is Doing to Your Body - KYR News

President Donald Trump, who tested positive for COVID-19 on October 1, is now taking a steroid previously used in a clinical trial for hospitalized patients with the virus in the United Kingdom.

White House physician Sean Conley on October 4 updated reporters at the Walter Reed medical Center, where Trump is seeking medical care, on the presidents current condition. Following several conflicting and concerning reports from aides and doctors over the weekend, Conley offered a more optimistic approach on the 74-year-olds progress including a new prescription for Dexamethasone.

Since we last spoke, the president has continued to improve, Conley said.

Trump was taken to the Maryland military hospital on Friday after experiencing a high fever and receiving supplemental oxygen, the doctor explained. The presidents oxygen levels fell again on Saturday morning, requiring oxygen and a lung scan.

Conley said the scans showed some indications of damage, but assured it was nothing of a major clinical concern.

You can watch the full briefing here.

Trump may be discharged as early as tomorrow, another medical expert added during the briefing.

Heres what you need to know about Dexamethasone and its relation to COVID-19:

According to MayoClinic, Dexamethasone is prescribed to relieve inflamed areas of the body, including symptoms stemming from severe allergies, arthritis, asthma, adrenal problems, kidney problems, immune system disorders and skin conditions, among others.

It works on the immune system to help relieve swelling, redness, itching, and allergic reactions, the Clinic states.

The drug works by lowering the bodys natural defense response, WebMD added.

It is only available via a doctors prescription and comes in tablets, elixirs and solutions, the medical website continued.

GettyA healthcare worker tends to a patient in the Covid-19 Unit at United Memorial Medical Center in Houston, Texas.

The drug was used to treat hospitalized patients with the coronavirus in the United Kingdom during a national clinical trial called, RECOVERY, according to the World Health Organization.

The organization said the trial found the drug to be beneficial for critically ill patients, writing:

According to preliminary findings shared with WHO (and now available as a preprint), for patients on ventilators, the treatment was shown to reduce mortality by about one third, and for patients requiring only oxygen, mortality was cut by about one fifth.

The World Health Organization states on its website that Dexamethasone is fairly low-risk, presenting a favourable benefit-risk profile, particularly in patients with severe forms of pneumonia. Patients with non-severe pneumonia see lower benefits, on the other hand, it continued.

Non-threatening side effects include upset stomach, headache, dizziness, menstrual changes, trouble sleeping, increased appetite, or weight gain, WebMD states. If side effects persist or worse, a doctor should be notified promptly.

Long-term use, such as more than two weeks, could potentially cause more adverse reactions, WHO added, including glaucoma, cataract, fluid retention, hypertension, psychological effects, weight gain, or increased risk of infections and osteoporosis.

To reiterate: All these adverse events are not associated with short term use (with the exception of hyperglycaemia that can worsen diabetes), the organization says on its website.

GettyU.S. President Donald Trump wears a mask as he visits Walter Reed National Military Medical Center in Bethesda, Maryland.

Dean of the Brown University School of Public Health Ashish Jha said earlier this weekend that the drug could be a very clear signal that he has a more severe disease, according to The Los Angeles Times.

The Dean emphasized similar sentiments on October 4, expressing that Trumps long-term lung damage is still up in the air, the newspaper reported.

Trump was also half-way through his five-day course of the anti-viral drug Remdesivir, the Los Angeles Times reported.

Dexamethasone is generally available in most countries through several product manufacturers, WHO says.

One manufacturer has already been prequalified by WHO (Kern Pharma in Spain) while another is under assessment, the organization states on its website.

WHO claims the medication is also generally affordable,with a median price of $0.33 per 4mg/ml injection ampoules (range: US$0.13-$3.5), citing 2016 and 2019 surveys of health facilities in low- and middle-income countries.

READ NEXT: WATCH: Trump Mocks Biden During Debate, I Dont Wear Masks Like Him

Excerpt from:
Dexamethasone: 5 Fast Facts You Need to Know - Heavy.com

The first of six replacement steam engines arrives at Koeberg. Photo: EE Publishers

On the basis of a three-month shorter delivery time, the R5-billion steam generator replacement contract was finally awarded to French state-owned nuclear contractor, Areva, which, after running into financial difficulties, is now controlled by French national electricity utility, Electricite d France (EDF).

This followed an extended High Court, Appeal Court and Constitutional Court battle in South Africa, in which Westinghouse (ultimately unsuccessfully) tried to challenge the irregular contract award to Areva on the grounds that the claimed urgency of the life extension was deliberately contrived by Eskom in order to wrongfully award the contract to Areva.

At the time when Koeberg was built between 1976 and 1985, Framatome (the predecessor to Areva) manufactured the two 900 MW nuclear reactors and the six associated steam generators at Koeberg, using designs licensed from Westinghouse.

Both Eskom and Areva claimed that it was absolutely critical for the safe operation of the power plant, and for the security of electricity supply in South Africa, that the steam generators should be finally replaced during the so-called X23 outage at Koeberg in 2018.

This was cited by Eskom as the reason for awarding the contract to Areva, where an allegedly contrived three-month float in Arevas project plan was indicated as the deciding factor. This, despite Westinghouse having met all the technical and commercial requirements, with a lower contract price, and a guarantee to meet the 2018 completion date on pain of substantial penalties.

However, all has not gone well for Areva in executing the project.

Areva is said to have experienced serious manufacturing quality problems with the steam generator forgings in France. In view of the claimed time criticality of the project, to resolve these issues Eskom and Areva agreed that the uncompleted forgings would be air-freighted to China where an Areva partner would complete the manufacture.

Apparently, each of the six steam generator forgings required the hiring of a Russian Antonov freighter aircraft (six flights in total) to fly the forgings from France to China. However, according to an impeccable source, after taking a closer look at the forgings, Arevas Chinese partner decided to scrap them and start again from scratch.

One can only speculate as to why it was not considered more economical for Areva to fly engineers from the Chinese manufacturer to France to examine the steam generator forgings, as opposed to flying all six steam generators to China for the inspection, prior to them being scrapped in China.

A former senior nuclear executive at Eskom commented to this writer at the time that: This was the most expensive transport of scrap metal in the history of humankind.

Eskom confirmed to EE Business Intelligence last week that: The forgings did not meet the expected quality standards and could not be accepted, and therefore they were scrapped by the contractor at their own cost.

According to Eskom, the contractual delivery date of all six steam generators was February 2018, with the contractual completion date for the replacement of all six steam generators being the end of 2019.

However, with all the delays in the project, including restarting manufacture of the steam generators from scratch in China, delivery of the first three steam generators for Unit 1 at Koeberg is only starting now in September 2020, with those for Unit 2 only scheduled for delivery in the third quarter of 2021.

Regarding the actual replacement of the steam generators, Eskom now says that: Installation is aligned with the outages scheduled for 2021 and 2022 respectively for the two Koeberg reactor units (Unit 1 and Unit 2). No mention is now made by Eskom of the criticality for completion in 2018 cited by Eskom at the time as the reason for placing a more expensive contract with Areva.

While not answering specific questions on the cost overruns arising for the project, Eskom insists that: The estimated cost-to-completion for the project is within the original budget approved in 2014. Of course, this is not very helpful because the original budget approved in 2014 is not disclosed, and may have been significantly higher than the contract price.

However, it seems clear that Eskom has in fact paid a premium for the replacement by Areva of the steam generators at Koeberg on the basis of a claimed three-month shorter completion time, when in fact completion will be some three years longer.

Areva has previously not responded to queries regarding the scrapping of the generators.

DM/EE Publishers

Further reading:

[1] A very tender process: Eskoms Koeberg contract ruled unlawful, by Aimee Clarke and Chris Yelland, EE Publishers, 13 January 2016

[2] Westinghouse opposes Eskom, Areva appeal to Concourt, and lodges counter appeal, by Aimee Clarke and Chris Yelland, EE Publishers, 4 February 2020

[3] Eskom changing the goalposts and rules of the game at half-time?, by Aimee Clarke, EE Publishers, 26 May 2016

[4] Eskom and Westinghouse respond to article in Energize magazine and other publications, by Chris Yelland, EE Publishers, EE Publishers, 7 June 2016

[5] Eskom wins Concourt judgement, EE Publishers, 21 December 2016

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Steam generators for Koeberg: the most expensive transport of scrap metal in the history of humankind - Daily Maverick

The federalgovernment is in the market for another used icebreaker that could be converted for use by the Canadian Coast Guard on the Great Lakes much to the dismay of shipbuilders across the country.

A request for proposals to acquire an existing light icebreaker was posted on the government's procurement website in mid-September.

The timing is interesting.Federal decision-makers have known for five years that the coast guard needssuch a vessel for the region.

The request for proposals which closes at the end of October was posted as U.S. lawmakers began to push bipartisan legislation through Congress to strengthen the U.S. Coast Guard's capacity to break ice and keep commerce flowing on the Great Lakes.

The plan for Canada to buy a used icebreaker follows a separate decision by Transport Canada to purchase a used ferry from Spain on an emergency basis.

The Canadian Marine Industry and Shipbuilding Association (CMISA), which represents most of the marine suppliers and shipyards across the country, said both decisions represent a loss of domestic jobs and at least $250 million in federal spending that could have gone into a Canadian economy hard hit by the coronavirus.

"We're of the strong belief that vessels such as light icebreakers can and should be built in Canada," said Colin Cooke, president and chief executive officer of the shipbuilding association.

"We have the capacity. We have the skilled trades. We have the expertise, the technical expertise. We have the shipyards. And that was what the point of the National Shipbuilding Strategy was all about."

That shipbuilding strategy is supposed to direct governmentwork to Canadian shipyards.Cooke said the plan to purchasean existing icebreaker and the deal to acquire a former Spanish ferry would both be unacceptable in normal times but they're even lessacceptablenow.

"We are in a COVID time when we're looking for all sorts of ways to make sure that people are employed, that businesses are able to survive I won't say thrive, I will say survive through the lockdowns caused by this pandemic," he said.

Public Services and Procurement Canada was asked for comment last Thursdaybut did notrespond.

The tender for the light icebreaker, posted online Sept. 18, describes the purchase as a necessaryinterim step for the coast guard to "bridge the gap while awaiting the delivery of dedicated new vessels."

Significantly, the request for proposals noted that the need for such a ship was identifiedfive years ago around the same time a comprehensive analysis warned that the coast guard icebreakingfleet was in dire straits and in need of immediate replacement.

"In 2015-16 the CCG identified a requirement for interim icebreaking capabilities to fill gaps in capacity resulting from ships being temporarily withdrawn from service" for refit and life extension, said the tender.

Two years ago, the Liberal government concluded a deal worth $827 millionwith Chantier Davie of Levis, Que., which operates the Davie shipyard, to refit three medium-sized commercial icebreakers for the coast guard.

Tim Choi, a University of Calgary shipbuilding expert, saidthis recent tendersuggeststhe federal government isoperating on the flawed assumption that there is an abundance of used icebreakers on the market.

The deal with the Davie shipyard was an anomaly and federal officials "got lucky" last time because there happened to be three vessels available, he said.

Choisaid he believes the federal government isn'tlikely tobe sofortunate this time:his research suggests there may beonly one light icebreaker out therethat would fit in the bill in Finland and it's notclear the Finns are ready to part with it.

"There are very few requirements for a vessel like that outside of Canada and the United States in the Great Lakes, St. Lawrence region," said Choi. "It's not like there's a used car lot where you can just go out and buy these things."

The shipbuilding association said it can make a strong case for a fast-track build in Canada.Choi said he believes procurement services may be forced in that direction anyway because of market conditions.

In mid-September, three U.S. senators Tammy Baldwin (D-WI), Todd Young (R-IN) and Gary Peters (D-MI) introduced the Great Lakes Winter Commerce Act.

The bipartisan legislation is expected to codify the U.S. Coast Guard's icebreaking operations on the Great Lakes and, more importantly, increase the size of its fleet.

"Inadequate icebreaking capacity in the Great Lakes is costing us thousands of American jobs and millions in business revenue," said Baldwin in a statement. "We must boost our icebreaking capacity in the Great Lakes to keep our maritime commerce moving."

Continued here:
Shipbuilding industry pushes back as federal government shops for used icebreaker - CBC.ca

NEW YORK, Oct. 05, 2020 (GLOBE NEWSWIRE) -- Daxor Corporation (NYSE MKT: DXR), an investment company with innovative medical instrumentation and biotechnology operations focused on blood volume measurement, today announces compelling new data demonstrating further beneficial use of Daxors blood volume analysis technology presented during the Heart Failure Society of Americas Virtual Annual Scientific Meeting 2020.

New data titled, Cost-effectiveness Analysis of Early Blood Volume-Guided Management in Hospitalized Heart Failure Patients studied the economic benefits of the use of blood volume analysis in the population of heart failure patients, one of the largest cost-drivers of the national healthcare system. The data showed an extremely cost-effective incremental cost-effectiveness ratio (ICER) of $10,200 which was 80% less costly than other therapies that are considered good value by common quality metrics. The data also revealed an average life-extension of 2.32 quality-adjusted life years (QALYs) in addition to the cost savings.

The use of Daxors BVA-100 blood test in heart failure outcomes in this compelling study showed exceptional value by not only improving patient life span, but also at a cost which is significantly less than other commonly used therapies. Daxors blood test for use in treating heart failure is demonstrably better for patient life extension outcomes compared to many other treatment regimes, while also driving excellent economic value for hospital systems, said Jonathan Feldschuh, Daxors Chief Scientific Officer.

Jean Oertel, VP of Commercialization and Customer Experience at Daxor stated, This important data shows the full power of our diagnostic technology with the ability to achieve better outcomes at reduced costs. The value of Daxors BVA-100 blood test for hospital systems and payers managing heart failure is covered by both public and private insurance with CPT/APC coding, something that many other approved tests never achieve even years after market clearance by the FDA.

About Daxor Corporation

Daxor Corporation (NYSE: DXR) is an innovative medical instrumentation and biotechnology company focused on blood volume measurement. We developed and market the BVA-100 (Blood Volume Analyzer), the first diagnostic blood test cleared by the FDA to provide safe, accurate, objective quantification of blood volume status and composition compared to patient-specific norms. The BVA technology has the potential to improve hospital performance metrics in a broad range of surgical and medical conditions, including heart failure and critical care, by informing treatment strategies, resulting in significantly improved patient outcomes. Our mission is to partner with clinicians to incorporate BVA technology into standard clinical practice and improve the quality of life for patients. For more information, please visit our website at Daxor.com.

Forward-Looking Statements

Certain statements in this release may include forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including without limitation, statements regarding the impact of hiring sales staff and expansion of our distribution channels. Forward-looking statements are predictions, projections and other statements about future events that are based on current expectations and assumptions and, as a result, are subject to risks and uncertainties. Many factors could cause actual future events to differ materially from the forward-looking statements in this release, including, without limitation, those risks associated with our post-market clinical data collection activities, benefits of our products to patients, our expectations with respect to product development and commercialization efforts, our ability to increase market and physician acceptance of our products, potentially competitive product offerings, intellectual property protection, FDA regulatory actions, our ability to integrate acquired businesses, our expectations regarding anticipated synergies with and benefits from acquired businesses, and additional other risks and uncertainties described in our filings with the SEC. Forward-looking statements speak only as of the date when made. Daxor does not assume any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

Investor Relations Contact:Bret ShapiroSr. Managing Partner, CORE IR516-222-2560brets@coreir.com

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New Research Demonstrates Daxor BVA-100 Blood Test Provides Extremely Cost-Effective Improvement in Patient Life Span at the Heart Failure Society of...

This market study on Water Heater Market covers the global and regional market with an in-depth breakdown of the inclusive growth prospects in the market. Also, it sheds light on the wide-ranging competitive landscape of the global Water Heater market. The report supplementary offers a dashboard overview of leading companies with their successful marketing strategies, market contribution, recent developments in both historic and present contexts. The Water Heater Market analysis includes the global market by value, by segments and by competitors overall advancement and strategies.

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CRISPR Industry Size 2019, Market Opportunities, Share Analysis up to 2026 - The Research Process

Several years ago, a brand new method of genetic engineering called CRISPR was invented, and it was based on discoveries made about the rudimentary "immune system" possessed by bacteria. Essentially, bacteria have a way of "remembering" which viruses had infected them previously, and they possess a molecular system that destroys viral DNA that matches that of a prior infection.

The molecular system consists of a DNA-cutting protein called Cas9. (See infographic from Business Insider below.) When equipped with a special guide RNA, Cas9 can be used to cut specific DNA sequences, for instance, a mutated gene that is causing a health problem. Because a broken DNA molecule is dangerous, the cell will attempt to repair it. If a DNA segment is snuck into the cell before the repair occurs, the cell can insert the new (and usually improved) DNA piece, providing a method to "edit" DNA.

The implications for such a technology are obvious. Such a method could be used, for example, to cure a person of a genetic disease or more easily produce genetically enhanced crops for farmers. But there are even cleverer uses. Because the CRISPR-Cas9 system can be designed to be self-propagating, it can be used to force a gene into a population of animals, such as mosquitoes. If this system targets genes that are important for survival or reproduction, then once released, this "gene drive" would rapidly spread through the population, killing off mosquitoes. (See infographic from The Economist.)

Now, a team of researchers writing in the journal Nature Communications has shown that a gene drive can be used to suppress infection with cytomegalovirus, a type of herpesvirus. The underlying molecular mechanism of the gene drive is similar to others before it: A self-propagating chunk of DNA inserts itself into a gene that is important to the virus. In this case, the gene is UL23, which is needed for cytomegalovirus to avoid the human immune response.

The researchers showed that when a cell is infected by both the normal virus (called "wildtype" or "WT") and the modified virus carrying a gene drive ("GD"), the gene drive was able to quickly and efficiently spread through the entire population, representing up to 95% of the final proportion of viruses. The end result is the suppression of viral infection (in cell culture, not in an animal model) because the gene drive virus lacks the important UL23 gene, which is needed for the virus to avoid a potent immune molecule known as interferon gamma(IFN-), which the authors added to the cell culture.

Could such a system work to treat viral infections in humans? Possibly. The authors note that a different gene (other than UL23) might need to be targeted, since lack of this gene is only fatal to the virus if IFN- is added to the cell culture. There are also concerns that a gene drive system could cause the viruses to mutate in various ways and may have unforeseen consequences.

Still, the technology is powerful and should be researched further. The coronavirus pandemic reminds us that we want to have multiple weapons in the public health arsenal should we be confronted with another life-threatening microbe.

Source: Walter, M., Verdin, E. Viral gene drive in herpesviruses. Nat Commun 11, 4884 (2020). Published: 28-Sept-2020. DOI: 10.1038/s41467-020-18678-0

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Gene Drives Could Kill Mosquitoes and Suppress Herpesvirus Infections - American Council on Science and Health

One summer day almost 20 years ago, a group of protestors arrived at a plot of genetically modified corn growing near the town of Montelimar in southern France.

They were led by Jos Bov, a left-wing activist famous for his skirmishes with the law and his tremendous moustache. Using machetes and shears, the protestors uprooted the crops and dumped the debris outside the offices of the regional government.

I thought about Bov this week as I read a new report on the next generation of genetic food technology. The techniques in the report make the processes that Bov opposed look clunky.

The GMOs he destroyed were created by inserting genes from other organisms say a stretch of DNA that confers resistance to a particular herbicide into a plants genome. This brute force approach is time-consuming and hard to control. Now scientists are using a new suite of gene-editing techniques, including a process known as CRISPR, to rapidly and precisely control the behavior of specific plant genes.

Gene-edited crops already exist. Scientists at the biotech firm Corteva, for example, have developed a high-yield strain of a variety of corn used in food additives and adhesives. Yet these initial advances belie the technologys potential.

Is there a way that civil society, government and businesses can come together to prioritize development of gene-edited crops that deliver social and environmental benefits as well as economic ones?

The power of gene editing can be wielded to modify plants and, among other things, achieve significant sustainability wins.

Here are a few potential outcomes explored in the new report, published by the Information Technology & Innovation Foundation, a pro-technology think tank:

This potential is thrilling, and there are signs that it will arrive soon. In China, where the government has made a big bet on gene-editing technology, numerous labs are working on crop strains that require less pesticides, herbicides and water. In the United States, a small but growing group of gene-editing startups is bringing new varieties to market, including an oilseed plant that can be used as a carbon-sequestering cover crop during the winter.

Yet when I read the ITIF report, I thought of Bov. Not because I agree with everything he said. Twenty years and many studies later, we know that the anti-GMO activists were wrong to say that modified crops posed a threat to human health. (The demonization of GMOs had profound consequences nonetheless: Fears about the risks posed by the crops are one reason why the crops are highly restricted in Europe and viewed warily by some consumers on both sides of the Atlantic.)

The reason I thought of Bov is that, at one level, he and other activists were pushing society to take a broader view of GMOs. They wanted people to ask who and what the crops were for, because they believed, rightly, that the crops were produced mainly with the profits of ag companies in mind.

Thats not to say its a bad thing for ag companies to be profitable. But our food systems affect so many aspects of our lives from the composition of the atmosphere to the prevalence of disease. When GMOs first began to be planted, there hadnt been enough debate about how the technology might affect these things. No wonder people were angry.

Thats a lesson I hope we can remember as gene editing shapes agriculture. Is there a way that civil society, government and businesses can come together to prioritize development of gene-edited crops that deliver social and environmental benefits as well as economic ones? If they can, we might end up with crops that everyone wants.

This article was adapted from the GreenBiz Food Weekly newsletter. Sign up here to receive your own free subscription.

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Get ready for the next wave of GMOs | Greenbiz - GreenBiz

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CRISPR in Agriculture Market Potential Growth, Size, Share, Demand and Analysis of Key Players Research Forecasts to 2027 - The Daily Chronicle

The mistake Nobel Prize prognosticators yours truly included make is to look through the greatest hits of biochemistry, biology, and medicine (the areas STAT covers) nuclear hormone receptors! microRNAs! and figure (as last years prediction story did) one of those is due and deserving. The trouble is, as MITs Phillip Sharp, who shared the 1993 medicine Nobel, told me, There is just a lot of good science that will never get recognized.

So focusing on the greatest hits to forecast the science winners who will be announced next week is too simplistic. Theyre all contenders, but the smart money looks for other criteria. Like toggling between discoveries of what cells and molecules do and inventions of techniques that reveal what they do, or between disciplines, or (for medicine) between something that directly cures patients and something about the wonders of living cells.

By that criteria, it might be a techniques turn, since the last such winner in medicine was for turning adult cells into stem cells, in 2012. Could this be the year for optogenetics, which allows brain scientists to control genetically modified neurons with light? I dont think optogenetics has made a big enough impact outside of neuroscience yet, said cancer biologist Jason Sheltzer of Cold Spring Harbor Laboratory, who dabbles in Nobel predictions, but who knows.

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The last Nobel for DNA sequencing was way back in 1980, he pointed out, and since then we have seen the complete sequencing of the human genome, one of humanitys towering achievements. (Sheltzer correctly predicted 2018s medicine Nobel for immuno-oncology pioneer James Allison. The Human Genome Project could win it for the officials who led it, like Francis Collins of the National Institutes of Health and Eric Lander of the Broad Institute. Would Craig Venter, who led a competing private effort, make it to Stockholm, too? Let the betting commence!

Just to be clear, science Nobels arent chosen all that, well, scientifically. For medicine, a five-member Nobel Committee for Physiology or Medicine at Swedens Karolinska Institute sifts nominations and selects candidates. The 50-member Nobel Assembly votes, this year on Oct. 5. So you can get head-scratchers from, say, 20-18-12 or similarly split votes if, say, genetics fanciers split their votes among two contenders. (If you want to know if that happened, hang on until 2070: Nobel records are secret and sealed for 50 years.) For chemistry, chosen on Oct. 7 this year, the five-member Nobel Committee of the Royal Swedish Academy of Sciences likewise sifts nominations and recommends finalists to the academy for a vote.

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Besides invention and discovery switching off in the medicine Nobel, there certainly seems to be periodicity in terms of disciplines taking turns, said David Pendlebury of data company Clarivate Analytics. He has made 54 correct Nobel predictions (usually in the wrong year, but in 29 cases within just two) since 2002 by analyzing how often a scientists key papers are cited by peers and awarded predictive prizes like the Lasker or Gairdner awards.

Neuroscience won the medicine Nobel in 2000, 2004, 2014, and 2017, immunology in 2008, 2011, and 2018, for instance. Infectious disease and cancer win every decade or two, and so are probably also-rans for 2020. Thats why STAT said last year that the 2018 medicine award for immuno-oncology made cancer an unlikely 2019 winner. Yet William Kaelin, Peter Ratcliffe, and Gregg Semenza won for discovering how cells sense and adapt to oxygen availability, through gene regulation, which is tangentially related to cancer. Go figure.

For the medicine prize, periodicity also applies to toggling between super-basic molecular biology and stuff that actually cures people (not year by year, but generally). Last years award for how cells sense changing oxygen levels was pretty abstruse and might shape this years choice.

Prizes with a more clinical focus have been 2003 (MRI), 2005 (H. pylori and ulcers), 2008 (HIV), 2015 (roundworm and malaria therapy), and 2018 (immuno-oncology), [so] maybe a clinical type of prize this year, [such as] hepatitis C treatment, brain stimulation for Parkinsons, cochlear implant, statins Pendlebury said. We wouldnt be surprised at a hep C win for Charles Rice of Rockefeller University and Ralf Bartenschlager of Heidelberg University (2016 Lasker winners) for the super-basic discoveries that led to drugs that cure the viral disease.

Like Pendlebury, Sheltzer believes in predictive prizes. I looked back at the last 20 years of Nobel Prizes in medicine/physiology, he said. Eighty-three percent of them had won at least one of three prizes before the Nobel: the Lasker, the Gairdner, or the Horwitz Prize. Of the five people who have recently won all three, only one works in a field so far ignored by the Nobel committees, he said: Yale School of Medicines Arthur Horwich, a pioneer of protein folding and chaperone proteins. In addition to the Gairdner in 2004, Horwitz in 2008, and Lasker in 2011, he received the $3 million Breakthrough Prize in 2019. So thats guess #1, Sheltzer said.

Unless Weve had a few [medicine] awards that you could classify as cell biology recently oxygen sensing in 2019, autophagy in 2016, even immune regulation is kinda cell biological, Sheltzer acknowledged. So I think a genetics award is more likely than one to Horwich, whose discoveries about how cells fold the proteins they synthesize are central to the understanding of life. STATs nickel says look no further than the 2015 Lasker Basic Medical Research Award: It honored Evelyn Witkin of Rutgers and Stephen Elledge of Harvard for discovering how DNA repairs itself after being damaged.

Might David Allis of Rockefeller and Michael Grunstein of UCLA finally get the call to Stockholm? They discovered one way genes are activated (through proteins called histones). Theyve shared a 2018 Lasker and a 2016 Gruber Prize in Genetics, and basically launched the hot field of epigenetics. I think a prize related to epigenetic control of transcription by DNA and histone modifications could be in order, Kaelin told STAT.

For physiology or medicine, Pendlebury likes Pamela Bjorkman of Caltech and Jack Strominger of Harvard for determining the structure and function of major histocompatibility complex (MHC) proteins, a landmark discovery that has contributed to drug and vaccine development, as well as Yusuke Nakamura of the University of Tokyo for genome-wide association studies that led to personalized approaches to cancer treatment (personally, we doubt this is cancers year again), and Huda Zoghbi of Baylor College of Medicine for work on the origin of neurological disorders.

In chemistry, Pendlebury likes Moungi Bawendi of MIT, Christopher Murray of the University of Pennsylvania, and Taeghwan Hyeon of Seoul National University for synthesizing nanocrystals, a cool new way to deliver drugs, and Makoto Fujita of the University of Tokyo for discovering supramolecular chemistry, in which lab-made molecules self-assemble by emulating how nature makes them. That has some overlap with Frances Arnolds 2018 Nobel for chemistry, so were skeptical, but who knows?

Lets address the elephant in the Nobel anteroom, and the chatter that the revolutionary genome editing technique CRISPR will win for chemistry. (Its value in medicine is still TBD, but its stellar biochemistry.)

The discovery of the CRISPR-Cas9 system is certainly worthy of a Nobel Prize, Kaelin said. I suspect the challenge here will be to get the attribution right. Perhaps there could be a chemistry prize for the basic mechanism and a medicine prize for application to somatic gene editing in human cells.

By attribution, he means, who gets CRISPR credit? Only three people can share a Nobel. But CRISPR has more mothers and fathers than that. Jennifer Doudna of the University of California, Berkeley, and her collaborator Emmanuelle Charpentier have won a slew of predictive prizes for their work turning a bacterial immune system into a DNA editor, but dark horse Virginijus iknys of Vilnius University shared the 2018 $1 million Kavli Prize in nanoscience for his CRISPR work. And Feng Zhang of the Broad Institute is more widely cited than the above three, Pendlebury said, a marker of what colleagues think.

CRISPR citations built up more to Feng Zheng et al. than to Doudna and Charpentier, but I dont think that matters as much as judgments about priority claim, Pendlebury said. There are more than three to credit and I do think that is problematic. Bad feelings are not something the Nobel Assembly wants to generate, I am sure.

CRISPR will win, said CSHLs Sheltzer. Its a question of when, not if. Zhang/Doudna/Charpentier/Horvath/Barrangou shared the Gairdner. Pick 2 or 3 of them?

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Dust off the crystal ball: It's time for STAT's 2020 Nobel Prize predictions - STAT

The FDA has lifted the clinical hold on a phase 1/2 clinical trial of Solid Biosciences gene therapy treatment for Duchenne muscular dystrophy (DMD). Solid Bio secured clearance to resume dosing in the trial after making manufacturing changes to cut the number of viral particles given to patients.

SGT-001, the adeno-associated viral (AAV) vector-mediated gene transfer therapy being tested in the phase 1/2 trial, has suffered a series of setbacks since entering the clinic, most recently when the FDA put the study on hold in response to a case of acute kidney injury. The FDA imposed the hold 11 months ago. In July, Solid Bio said the FDA wanted to see more data before lifting the hold.

The request led Solid Bio to share further information on its gene therapy manufacturing process and its latest safety and efficacy data. The additional information proved sufficient to persuade the FDA to lift the clinical hold.

From Concept to Market: Overcoming the Challenges of Manufacturing and Clinical Trials

Learn how CRO/CDMOs successfully address operational and regulatory challenges for pharmaceutical and biotechnology clients; and how this can make the difference between study success or failure.

Solid Bio will resume dosing using a gene therapy made under a revised manufacturing process. The new process is intended to remove most empty viral capsids, thereby enabling Solid Bio to cut total viral load without reducing the dose. The focus on viral load reflects concerns systemic delivery of AAV vectors can damage organs and cause inflammation.

To get the FDA to lift the hold, Solid Bio shared data from a quantitative, in vitro microdystrophin expression assay designed to show the comparability of SGT-001 manufactured under the old and new processes. Solid Bio shared those results in response to the FDAs request for information in July.

The biotech is taking other precautions to manage the potential risk posed by SGT-001. Solid Bio has capped the maximum weight of the first two patients to receive SGT-001 after the hold lifts at 18 kg. As the dose of SGT-001 is determined by weight, heavier patients receive more vector genomes. The adverse events seen in some gene therapy trials, such as the deaths in Audentes Therapeutics trial, have happened in patients who were heavier and therefore received a higher viral load.

Solid Bio is further mitigating the potential for SGT-001 to cause harm by amending the protocol to include the prophylactic use of eculizumab, the anti-complement inhibitor sold by Alexion as Soliris, and C1 esterase inhibitor, while also increasing the prednisone dose in the month after treatment.

The protocol changes position Solid Bio to resume its pursuit of DMD gene therapy leader Sarepta Therapeutics, which suffered a setback of its own last month when the FDA asked it to use an extra potency assay in a planned clinical trial. Pfizer is also in the race but, like Solid Bio, has run into safety issues that could give Sarepta an edge.

Shares in Solid Bio, which had slumped to $2 apiece, rose 70% in response to the end of the hold.

The rest is here:
FDA lifts clinical hold on Solid Bio gene therapy trial - FierceBiotech

SAN RAFAEL, Calif., Oct. 2, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN), a pioneer in developing treatments for phenylketonuria (PKU) and gene therapies, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation toBMN 307, an investigational gene therapy for the treatment of individuals with PKU.

Fast Track designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fulfill an unmet medical need, enabling drugs to reach patients earlier. Clinical programs with Fast Track designation may benefit from early and frequent communication with the FDA throughout the regulatory review process. These clinical programs may also be eligible to apply for Accelerated Approval and Priority Review if relevant criteria are met, as well as Rolling Review, which means that completed sections of the Biologic License Application can be submitted for review before the entire FDA application is complete. Both the FDA and European Medicines Agency have granted BMN 307 Orphan Drug Designation.

"Fast Track designation combined with our ability to conduct our clinical studies incorporating material manufactured using a commercial-ready process will further facilitate rapid clinical development of BMN 307 gene therapy," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin. "We are looking forward to working closely with the FDA, as well as other health agencies, to evaluate the safety and efficacy of this promising investigational gene therapy as we continue our unwavering 15-year commitment to advance the standard of care for people with PKU."

PKU is a rare genetic disease that manifests at birth and is marked by an inability to break down Phe, an amino acid that is commonly found in many foods. Left untreated, high levels of Phe become toxic to the brain and may lead to serious neurological and neuropsychological issues, affecting a person's ability to think and problem solve, and can lead to depression, anxiety, and behavior disturbance impacting quality of life. Due to the seriousness of these symptoms, in many countries, infants are screened at birth to ensure early diagnosis and treatment to avoid intellectual disability and other complications. According to treatment guidelines, PKU patients should maintain lifelong control of their Phe levels.

BMN 307 Clinical Program

Last week, BioMarin announced that it had dosed the first participant in the global Phearless Phase 1/2 study with BMN 307, an AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU by inserting a correct copy of the PAH gene into liver cells. BMN 307 will be evaluated to determine safety and whether a single dose of treatment can restore natural Phe metabolism, increase plasma Phe levels, and enable a normalization of diet in patients with PKU.BioMarin is conducting this study with material manufactured with a commercial-ready process to facilitate rapid clinical development and potentially support approval. BMN 307 represents a potential third PKU treatment option in BioMarin's PKU franchise and a second gene therapy development program.

BioMarin's clinical program is composed of two key studies. Phearless, a Phase 1/2 study, will evaluate the safety, efficacy, and tolerability of a single intravenous administration of BMN 307 in patients with PKU. The study consists of a dose-escalation phase, followed by a cohort expansion phase once an initially efficacious dose has been demonstrated. In addition, BioMarin is sponsoring an observational study, Phenom, which includes patients with PKU to measure both established and new markers of disease and clinical outcomes over time.

BioMarin's 15-Plus Year Commitment to PKU Research

For more than 15 years, BioMarin has been a pioneer in ongoing research to help improve the lives of PKU patients. BioMarin has developed therapies that have been used to treat approximately 7,000 PKU patients around the world. The company has two approved PKU therapies, and the investigational gene therapy BMN 307 is currently in development. BioMarin has conducted 41 clinical studies in PKU and has sponsored 44 external clinical studies. BioMarin researchers have authored 65 publications in medical and scientific journals on PKU and supported another 57 publications by external researchers.

About Gene Therapy

Gene therapy is a form of treatment designed to address a genetic problem by adding a normal copy of the defective gene. The functional gene is inserted into a vector containing a small DNA sequence that acts as a delivery mechanism, providing the ability to deliver the functional gene to targeted cells. The cells can then use the information from the normal gene to build the functional proteins that the body needs, potentially reducing or eliminating the cause of the disease.

Gene Therapy Manufacturing

BioMarin has leveraged its knowledge and experience in manufacturing complex biological products to design, construct and validate a state-of-the-art vector production facility in Novato, California. This facility is the site of production for both valoctocogene roxaparvovec and BMN 307, investigational gene therapies. Manufacturing capabilities are an essential driver for BioMarin's gene therapy programs and allows the Company to control quality, capacity, costs and scheduling enabling rapid development. Production of BMN 307 with a commercial ready process at scale reduces risk associated with making process changes later in development and may speed overall development timelines significantly.

Ongoing process development efforts and experience gained at commercial scale have led to improvements in productivity and operational efficiency. The ability to scale out the facility with additional equipment combined with the improvements in productivity result in a doubling of overall potential capacity to 10,000 doses per year, combined for both products, depending on final dose and product mix. This improvement in productivity is anticipated to meet potential commercial and clinical demand for both valoctocogene roxaparvovec and BMN 307 well into the future.

About Phenylketonuria

PKU, or phenylalanine hydroxylase (PAH) deficiency, is a genetic disorder affecting approximately 70,000 diagnosed patients in the regions of the world where BioMarin operates and is caused by a deficiency of the enzyme PAH. This enzyme is required for the metabolism of Phe, an essential amino acid found in most protein-containing foods. If the active enzyme is not present in sufficient quantities, Phe accumulates to abnormally high levels in the blood and becomes toxic to the brain, resulting in a variety of complications including severe intellectual disability, seizures, tremors, behavioral problems and psychiatric symptoms. As a result of newborn screening efforts implemented in the 1960s and early 1970s, virtually all individuals with PKU under the age of 40 in countries with newborn screening programs are diagnosed at birth and treatment is implemented soon after. PKU can be managed with a severe Phe-restricted diet, which is supplemented by low-protein modified foods and Phe-free medical foods; however, it is difficult for most patients to adhere to the life-long strict diet to the extent needed to achieve adequate control of blood Phe levels. Dietary control of Phe in childhood can prevent major developmental neurological toxicities, but poor control of Phe in adolescence and adulthood is associated with a range of neurocognitive disabilities with significant functional impact.

To learn more about PKU and PAH deficiency, please visit http://www.PKU.com. Information on this website is not incorporated by reference into this press release.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases.The company's portfolio consists of six commercialized products and multiple clinical and pre-clinical product candidates.For additional information, please visitwww.biomarin.com. Information on such website is not incorporated by reference into this press release.

Forward-Looking Statement

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: the Company's BMN 307 program being eligible to apply for Accelerated Approval and Priority Review if relevant criteria are met, as well as Rolling Review, the development of BioMarin's BMN 307 program generally, including the impact on the timing and process for regulatory interactions and decisions, BioMarin's gene therapy manufacturing capabilities and the anticipation that the current manufacturing capabilities will meet potential commercial and clinical demand for both valoctocogene roxaparvovec and BMN 307 well into the future and the impact of using material manufactured at commercial scale in a clinical trial on reducing risk and speeding up overall development timelines. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others:the content and timing of decisions by the U.S. Food and Drug Administration, the European Commission and other regulatory authorities; uncertainties inherent in research and development, including unfavorable new clinical data and additional analyses of existing clinical data; the results and timing of current and future clinical trials related to BMN 307; our ability to reproducibly and consistently manufacture sufficient quantities of BMN 307, the possibility that changes may be required to the current manufacturing process; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended June 30, 2020 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.

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BioMarin, Pioneer in Phenylketonuria (PKU) and Gene Therapy, Receives FDA Fast Track Designation for PKU Investigational Gene Therapy, BMN 307 -...

With ongoing advances in science and technology, the cell and gene therapy pipeline has grown especially robust over the past few years. At present, ClinicalTrials.gov shows more than 4,500 active gene therapy trials globally. In the United States, McKinsey experts expect to see 10 to 20 cell and gene therapy approvals per year over the next five years.

This rise in supply has created a heightened demand for contract development and manufacturing organizations (CDMOs) with biotech expertise. CDMOs typically supply materials and handle production and manufacturing, allowing life sciences companies to focus on innovation and marketing.

The bottleneck stems from a shortage of CDMOs with gene therapy expertise and resources. Considering the critical need for safe, effective gene therapies and the rapid pace of development, it's important for pharma and biopharma to find a CDMO with both gene therapy capabilities and availability to take on new partners nownot 18 months from now.

"Full-service CDMOs that can assist with both development and manufacturing are in highest demand,"said Richard Welch, PhD, vice president, development services for Emergent BioSolutions, a global CDMO and specialty life sciences company headquartered in Gaithersburg, Maryland. "As pharma and biopharma companies move from early phase to late phase, CDMOs need experience with process characterization and process validation as well as commercial production and supply chain."

"The supply chain is much more complex,"added Tarek Abdel-Gawad, senior director of commercial strategy for Emergent BioSolutions. "You aren't just growing cells. You're ensuring viruses, helper viruses, and plasmid DNA work together to produce the molecule of choice. Few companies have the capabilities, equipment, and GMP expertise."

Much of the gene therapy development as of late has stemmed from smaller biotech companies or research universities according to a McKinsey report. Large pharmaceutical companies may partner with these organizations on rare disease or oncology treatments two therapeutic areas where much of the research lies.

Many small to midsize companies have the idea and investor support, but do not have the employees, infrastructure or manufacturing space. "A CDMO is a good partner in those cases,"said Mukesh Mayani, PhD, principal scientist, gene therapy at Sanofi. "You can test your hypotheses and work with a CDMO that has the platform, the people, and the preclinical models. This arrangement speeds up the timeline and allows these innovative companies to focus on other modalities and molecules."

Pharma and biopharma companies of all sizes can learn from this "single-source"approach. Partnering with a CDMO earlier in the processfrom preclinical development through packagingfrees up resources to focus on innovation and communication.

"It is neither simple nor cheap to develop and manufacture gene therapies,"said Dr. Welch. "A CDMO has the built-in skill set to grow viruses at the densities necessary to meet early-phase studies while hitting safety margins. With the clinical trial failure rate as high as it is, working with a CDMO that has experience in different technologies and products makes for a more efficient, cost-effective process."

Although there is a high demand now for CDMOs with gene therapy expertise, the market is quickly growing. According to Grand View Research, the CDMO market is expected to grow from $115.6 billion in 2020 to $157.7 billion in 2025, outpacing the pharmaceutical industry as a whole. New cell and gene therapy CDMOs are emerging and established CDMOs are expanding capabilities.

Before you start your CDMO search, consider the following two factors:

When vetting CDMOs for your gene therapy studies, consider the strengths and weaknesses of your company as well as your potential CDMO partner. A few points to consider include:

As gene therapy research continues to expand, innovators in this space will need CDMOs with highly specific expertise, facilities, and equipment. Choose a partner that can assist from the earliest phases of product development all the way to commercialization.

Capra, Emily, et al. "Gene therapy coming of age: Opportunities and challenges to getting ahead."McKinsey, October 2, 2019

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Gene therapy solution: The value of a CDMO as your end-to-end partner - BioPharma Dive

Cell and Gene Therapy Market research report is the new statistical data source added by A2Z Market Research.

Cell and Gene Therapy Market is growing at a High CAGR during the forecast period 2020-2026. The increasing interest of the individuals in this industry is that the major reason for the expansion of this market.

Cell and Gene Therapy Market research is an intelligence report with meticulous efforts undertaken to study the right and valuable information. The data which has been looked upon is done considering both, the existing top players and the upcoming competitors. Business strategies of the key players and the new entering market industries are studied in detail. Well explained SWOT analysis, revenue share and contact information are shared in this report analysis.

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Top Key Players Profiled in this report are:

JW CreaGene, Vericel, Tego Sciences, CHIESI Farmaceutici, Spark Therapeutics, GC Pharma, MolMed, AnGes, Takeda Pharmaceutical Company, APAC Biotech, Gilead Sciences, Corestem, AVITA Medical, Novartis AG, JCR Pharmaceuticals, Dendreon, CO.DON, Medipost, Osiris Therapeutics, Amgen, Biosolution, CollPlant, Japan Tissue Engineering, Organogenesis, Orchard Therapeutics, Stempeutics Research

The key questions answered in this report:

Various factors are responsible for the markets growth trajectory, which are studied at length in the report. In addition, the report lists down the restraints that are posing threat to the global Cell and Gene Therapy market. It also gauges the bargaining power of suppliers and buyers, threat from new entrants and product substitute, and the degree of competition prevailing in the market. The influence of the latest government guidelines is also analyzed in detail in the report. It studies the Cell and Gene Therapy markets trajectory between forecast periods.

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The cost analysis of the Global Cell and Gene Therapy Market has been performed while keeping in view manufacturing expenses, labor cost, and raw materials and their market concentration rate, suppliers, and price trend. Other factors such as Supply chain, downstream buyers, and sourcing strategy have been assessed to provide a complete and in-depth view of the market. Buyers of the report will also be exposed to a study on market positioning with factors such as target client, brand strategy, and price strategy taken into consideration.

Global Cell and Gene Therapy Market Segmentation:

Market Segmentation by Type:

Cell TherapyGene Therapy

Market Segmentation by Application:

HospitalsWound Care CentersCancer Care CentersAmbulatory Surgical CentersOthers

Regions Covered in the Global Cell and Gene Therapy Market Report 2020:The Middle East and Africa(GCC Countries and Egypt)North America(the United States, Mexico, and Canada)South America(Brazil etc.)Europe(Turkey, Germany, Russia UK, Italy, France, etc.)Asia-Pacific(Vietnam, China, Malaysia, Japan, Philippines, Korea, Thailand, India, Indonesia, and Australia)

The report provides insights on the following pointers:

Table of Contents

Global Cell and Gene Therapy Market Research Report 2020 2026

Chapter 1 Cell and Gene Therapy Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

Chapter 5 Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6 Global Production, Revenue (Value), Price Trend by Type

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10 Marketing Strategy Analysis, Distributors/Traders

Chapter 11 Market Effect Factors Analysis

Chapter 12 Global Cell and Gene Therapy Market Forecast

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Comprehensive Report on Cell and Gene Therapy Market 2020 | Trends, Growth Demand, Opportunities & Forecast To 2026 | JW CreaGene, Vericel, Tego...

NEW YORK--(BUSINESS WIRE)--Pfizer Inc. (NYSE: PFE) today announced that its investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne muscular dystrophy (DMD) received Fast Track designation from the U.S. Food and Drug Administration (FDA). PF-06939926 is currently being evaluated to determine the safety and efficacy of this gene therapy in boys with DMD.

Fast Track is a process designed to facilitate the development, and expedite the review, of new drugs that are intended to treat or prevent serious conditions that have the potential to address an unmet medical need. This designation was granted based on data from the Phase 1b study that indicated that the intravenous administration of PF-06939926 was well-tolerated during the infusion period and dystrophin expression levels were sustained over a 12-month period.

The FDAs decision to grant our investigational gene therapy PF-06939926 Fast Track designation underscores the urgency to address a significant unmet treatment need for Duchenne muscular dystrophy, said Brenda Cooperstone, MD, Chief Development Officer, Rare Disease, Pfizer Global Product Development. DMD is a devasting condition and patients, and their parents, are waiting desperately for treatment options. We are working to advance our planned Phase 3 program as quickly as possible.

DMD is a devastating and life-threatening X-linked disease that is caused by mutations in the gene encoding dystrophin, which is needed for proper muscle membrane stability and function. Patients present with muscle degeneration that progressively worsens with age to the extent that they require wheelchair assistance when they are in their early teens, and unfortunately, usually succumb to their disease by the time they are in their late twenties. It is estimated that there are ~10-12,000 individuals affected with DMD in the US.

About PF-06939926

PF-06939926 is an investigational, recombinant adeno-associated virus serotype 9 (rAAV9) capsid carrying a shortened version of the human dystrophin gene (mini-dystrophin) under the control of a human muscle-specific promotor. The rAAV9 capsid was chosen as the delivery vector because of its potential to target muscle tissue. Pfizer initiated the Phase 1b multi-center, open-label, non-randomized, ascending dose study of a single intravenous infusion of PF-06939926 in 2018. The goal of the study is to assess the safety and tolerability of this investigational gene therapy. Other objectives of the clinical study include measurement of dystrophin expression and distribution, as well as assessments of muscle strength, quality and function.

About Pfizer Rare Disease

Rare disease includes some of the most serious of all illnesses and impacts millions of patients worldwide, representing an opportunity to apply our knowledge and expertise to help make a significant impact on addressing unmet medical needs. The Pfizer focus on rare disease builds on more than two decades of experience, a dedicated research unit focusing on rare disease, and a global portfolio of multiple medicines within a number of disease areas of focus, including rare hematologic, neurologic, cardiac and inherited metabolic disorders.

Pfizer Rare Disease combines pioneering science and deep understanding of how diseases work with insights from innovative strategic collaborations with academic researchers, patients, and other companies to deliver transformative treatments and solutions. We innovate every day leveraging our global footprint to accelerate the development and delivery of groundbreaking medicines and the hope of cures.

Click here to learn more about our Rare Disease portfolio and how we empower patients, engage communities in our clinical development programs, and support programs that heighten disease awareness.

Pfizer Inc.: Breakthroughs that change patients lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at http://www.pfizer.com. In addition, to learn more, please visit us on http://www.pfizer.com and follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

DISCLOSURE NOTICE: The information contained in this release is as of October 1, 2020. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about PF-06939926, an investigational gene therapy to potentially treat Duchenne muscular dystrophy, including its potential benefits and a planned Phase 3 study for PF-06939926, that involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; the risks associated with initial and preliminary data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when regulatory authorities will approve the commencement of our planned Phase 3 study; whether and when drug applications may be filed in any jurisdictions for any potential indication for PF-06939926; whether and when any such applications may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether PF-06939926 will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of PF-06939926; uncertainties regarding the impact of COVID-19 on our business, operations and financial results; and competitive developments.

A further description of risks and uncertainties can be found in Pfizers Annual Report on Form 10-K for the fiscal year ended December 31, 2019 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned Risk Factors and Forward-Looking Information and Factors That May Affect Future Results, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at http://www.sec.gov and http://www.pfizer.com.

Read more:
Pfizer Receives FDA Fast Track Designation for Duchenne Muscular Dystrophy Investigational Gene Therapy - Business Wire

Latest Research Report: Gene Therapy industry

Gene Therapy Market report is to provide accurate and strategic analysis of the Profile Projectors industry. The report closely examines each segment and its sub-segment futures before looking at the 360-degree view of the market mentioned above. Market forecasts will provide deep insight into industry parameters by accessing growth, consumption, upcoming market trends and various price fluctuations.

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

Gene Therapy Market competition by top manufacturers as follow:SangamoSpark TherapeuticsDimension TherapeuticsAvalanche BioCelladonVicalAdvantagene

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Global Gene Therapy Market research reports growth rates and market value based on market dynamics, growth factors. Complete knowledge is based on the latest innovations in the industry, opportunities and trends. In addition to SWOT analysis by key suppliers, the report contains a comprehensive market analysis and major players landscape.The Type Coverage in the Market are: Ex vivoIn vivo

Market Segment by Applications, covers:Cancer DiseasesMonogenic DiseasesInfectious DiseasesCardiovascular DiseasesOthers

Market segment by Regions/Countries, this report coversNorth AmericaEuropeChinaRest of Asia PacificCentral & South AmericaMiddle East & Africa

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Gene Therapy Market Incredible Possibilities, Growth Analysis and Forecast To 2025 - The Daily Chronicle

The pharmaceutical industry is one of the most scientifically innovative and competitive industries, particularly in oncology. As of 2018, there were over 1,100 cancer therapies in development, and as of 2020, 362 of them were cell and gene therapies. As a result, there is a need for continued innovation and increased efficiency in terms of drug development to manage cost, complexity and speed to provide potentially transformative therapies for cancer patients.

Within the last two decades, large pharmaceutical corporations have established themselves firmly in oncology by prioritising internal R&D efforts, as well as developing and accessing novel science and technology through collaborations and alliances with biotech companies and academic institutions.

Dramatic advances in the understanding of basic molecular mechanisms of underlying disease has continued to shift R&D focus toward precision medicine choosing the right therapy for a patient based on molecular understanding of their disease and less on traditional cancer therapies such as cytotoxic chemotherapies and broad-cell cycle inhibitors.

As a result of this shift in drug development, a highly concentrated overlay in product modalities and mechanisms of action has crowded the oncology pipeline across a very broad range of hematological and solid tumour indications.

The industry is asking itself how to stay innovative, how to develop and bring to market higher quality therapies to patients and how to do this faster and more efficiently.

A diversity of collaboration types

There is broad recognition that given the breadth and complexity of emerging science driving innovation in oncology, collaborations are essential in order that relevant expertise, know-how and capabilities can be combined in the right way to address patient needs.

Such collaborations take on many forms, ranging from early, multi-party alliances and consortia which are often pre-competitive in nature driving the development and shared learnings from technologies that may be enabling the field as a whole, through to more bespoke collaborations between entities.

Cell therapy research has been built on collaborations amongst scientists and entrepreneurs, providing early proof of concept for modalities thought to be too difficult to commercialise but with a strong potential for patient benefit

These may be more focused on collaborative research and development of novel products, to secure the necessary data for regulatory approvals to make such products available widely to the patients who can benefit from them.

Pre-competitive collaborations, often in basic and preclinical research, can reduce the barrier of competition and drive benefits for all stakeholders, most notably, the patient. As summarised by The National Institutes of Health, this includes reducing the number of redundant clinical trials, enhancing the statistical strength of studies, reducing overall costs and risks, and improving study participant recruitment, all while triggering creativity and innovation between collaborators.

These benefits strengthen capabilities and accelerate product development, ultimately producing higher quality and more effective therapies.

One powerful example is The National Institutes of Healths Partnership for Accelerating Cancer Therapies (PACT), which brought together 11 pharmaceutical companies to accelerate the development of new cancer immunotherapies.

Aligning with the focus of the Cancer Moonshot Research Initiative, PACT aimed to retrospectively analyse patient data from past clinical trials with the goal of predicting future patient outcomes.

This type of approach supports the ability to compare data across all trials and facilitates information sharing between partners, undoubtedly accelerating the pathway to effective therapies.

A second example is the establishment of The Parker Institute for Cancer Immunotherapy, to enable leading academic researchers and companies to come together in a pre-competitive setting, to enable rapid shared understanding and development of immunotherapeutic approaches, including the study of combination regimens.

Such combination trials, particularly those encompassing investigational products, have historically been challenging to undertake given the need for bespoke company-to-company and other 1:1 collaborative agreements. Bringing together multiple academic and industry participants under an open innovation model provides a basis to significantly accelerate the generation of scientific and clinical data that may substantially inform the field of cancer immunotherapy as a whole.

Oncology cell therapy research has been built on foundational academic collaborations amongst scientists and entrepreneurs, providing early proof of concept for modalities thought to be too difficult to commercialise but with a strong potential for patient benefit.

Examples include Kite Pharma, formed from the foundational work at the National Cancer Institute, Juno from the collaboration between the Fred Hutchinson Cancer Center and Memorial Sloan Kettering Cancer Center (all working on the first CAR T-cell candidates), or Adaptimmune working with University of Penn to first show efficacy of optimised TCR T-cells.

For collaborations that are more geared to development of novel therapies, aiming for regulatory approval and commercial availability, bespoke collaborations between biotech and pharma companies are commonplace, whereby the respective expertise and capabilities of each partner are combined in order to optimise and accelerate development, and to enable subsequent, larger scale manufacture and distribution. There are many examples of such collaborations, for which the structure can vary widely depending on the expertise of each partner, and the collaborative ways of working.

For example, under a traditional pharma/biotech collaboration and licensing model, a biotech partner may have primary responsibility for significant elements of research and early product development, and the pharma partner may lead the majority of later stage development, as well as post-approval commercial manufacture and supply. This logically aligns with organisational expertise and scale, and this type of collaboration structure has historically proven to work well. Many novel therapies have been successfully developed through such partnerships.

The rapid emergence of cell and gene therapy has required the industry to establish new and distinct capabilities, such as optimal process development and manufacture of autologous, patient specific cell therapies, whilst minimising the vein-to-vein time (the elapsed time between apheresis treatment for a patient, and reinfusing the final autologous manufactured product).

There are a growing number of biotech and pharma companies that have established or are establishing such end-to-end cell therapy capabilities, which can also play into how collaborations are structured in the field.

Case Study: From Technology Agreement to co-development and co-commercialisation partnership

In 2015, Adaptimmune and Universal Cells signed an agreement to drive the development of technologies leveraging gene-edited Induced Pluripotent Stem Cell (iPSC) lines, towards the development of allogeneic, or off-the-shelf, T-cell therapies. Universal Cells brought leading gene editing capability to make targeted gene edits to modify the characteristics of selected iPSC cell lines, and Adaptimmune the technology to differentiate iPSCs into T-cells.

Back then the science for this collaboration was early and under-developed with both parties embarking on a long-term effort and making significant at-risk investments to determine if edited, functional T-cells could be produced.

Today, Universal Cells (now an Astellas company) and Adaptimmune have established capabilities and expertise to progress novel cell therapies into clinical development, as well as with manufacturing and supply chain.

Based on this progress, in January 2020, Adaptimmune and Astellas signed a product-focused agreement to co-develop and co-commercialise up to three new stem-cell derived allogeneic T-cell therapies for people with cancer.

Given the scientific synergy between Universal Cells and Adaptimmune, and that each company is developing capabilities that may effectively address later stage product development and post-approval commercial supply, the 2020 partnership was structured as a co-development and co-commercialisation agreement. It enables the companies to work closely together, throughout the continuum of research, development and commercialisation.

Astellas and Adaptimmune will collaborate through to the end of phase 1, with Universal Cells leading gene editing activities and Adaptimmune leading iPSC to T-cell differentiation, early product characterisation and development. Beyond that, Astellas and Adaptimmune will decide whether to develop and commercialise a product candidate together under a co-development and co-commercialisation cost and profit-sharing arrangement, or for one company to take it forward alone.

This partnership is an example of how companies can harness their individual science and bring together highly complementary skills and expertise. It will enable the development of new, off-the-shelf T-cell therapies for people with cancer, which could potentially offer significant advantages such as broader access, reduced vein-to-vein time, and lower cost. The co-development and co-commercialisation nature of the agreement allows both companies to collaborate closely and on a long term basis, whilst leveraging end-to-end capabilities established by each company, maximising the velocity of product development, and ultimately delivering novel therapies to patients.

This type of agreement exemplifies how early speculative scientific collaboration can benefit all parties, most importantly the patient. It is one example from many in oncology, that underlines the value of long-term partnership within a field that is evolving rapidly across many scientific, operational and commercial frontiers.

Bringing together both teams of passionate and forward-thinking scientists may contribute to unlocking the current opportunities and challenges of off-the-shelf T-cell therapy development more effectively and efficiently for patients.

Similarly to what we are seeing as the world comes together to fight COVID-19, we as leaders in oncology owe it to patients to constantly look for ways to bring our innovative ideas as quickly as possible to the market. Working together might make that happen faster.

About the author

Helen Tayton-Martin is chief business officer at Adaptimmune.

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The new pharma collaborations driving transformative research in oncology - - pharmaphorum

As the COVID-19 pandemic has progressed, we have seen an alarming amount of disinformation spread online, including by our elected officials. In Colorado we have seen elected officials visiting restaurants in the middle of stay-at-home orders without wearing a mask.

Other elected officials have flouted local health department advice by gathering with thousands at Bandimere Speedway, where few wore masks.

We have heard them say that statewide mask mandates somehow infringe on their rights, ignoring that placing the health of others infringes on the rights of so many innocent Coloradans.They have even sued the state government for its attempts to protect public health.

Despite advice from our public health leaders to practice social distancing and wear masks to prevent the spread of the virus, President Donald Trump and Vice President Mike Pence continue to refuse to wear masks in public.

Pence leads the White Houses coronavirus task force, yet he toured the Mayo Clinic without a mask on April 28 (that same day, the U.S. reached one million COVID-19 cases).

The latest from the coronavirus outbreak in Colorado:

>> FULL COVERAGE

Recently, we have learned the alarming fact that President Trump knew back in February that this virus is spread through the air and five times deadlier than the flu.

Amid lies and conspiracy theories, our light at the end of the tunnel is scientific innovation and trust in experts. Thanks to round-the-clock collaboration between the public and private sectors, there are several COVID-19 vaccines and treatments in development.

One, for example, blocks the novel coronavirus from binding to human cells and reproducing; by stopping the virus from connecting with human cells, the drug prevents it from multiplying and attacking the body.

READ:Colorado Sun opinion columnists.

There are more than 100 different vaccines at various stages of development, and researchers are using different avenues such as gene therapy, DNA and antibodies from survivors to develop an effective vaccine.

U.S. health care innovation has saved millions of lives.HIV is now a manageable disease, no longer a death sentence.Thanks to developments in early-detection mammogram technology, female breast cancer cases dropped by 40% in 2016.

We now have a drug that can treat over 90% of Hepatitis C patients, whereas older drugs took nearly a year to become effective and even then only worked on 50% of patients.

And vaccines have saved the health of millions by preventing once-fatal illnesses. History teaches us that our best bet is to support the researchers working to develop treatments and vaccines for COVID-19.

We owe it to the frontline essential workers our grocery store workers, health care workers, sanitation services, public transit operators and so many more risking their lives every day to do better in this pandemic.

We need to ignore disinformation, whether it comes from the internet or the White House and follow the advice of our public health professionals.

We need more medical professionals and scientists to run for office to be that expert voice to help stop the spread of misinformation before it starts. Supporting and investing in their research and innovation will get us through this crisis.

Dr. Yadira Caraveo, D-Thornton, is a pediatrician and represents House District 31 in the Colorado House of Representatives.

The Colorado Sun is a nonpartisan news organization, and the opinions of columnists and editorial writers do not reflect the opinions of the newsroom. Read our ethics policy for more on The Suns opinion policy and submit columns, suggested writers and more to opinion@coloradosun.com.

Support local journalism around the state.Become a member of The Colorado Sun today!

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Opinion: Scientific innovation and trust in experts is our light at the end of the tunnel - The Colorado Sun

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces two presentations at the European Society for Immunodeficiencies (ESID) 2020 Meeting to be held virtually October 14-17, 2020. An oral presentation will provide an update on data from the Phase 1/2 clinical trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). An e-poster will highlight preclinical study data on RP-L401 for Infantile Malignant Osteopetrosis (IMO).

Additional presentation details can be found below:

Oral Presentation

Title: A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1 Session Title: TreatmentPresenter: Donald B. Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at the University of California, Los AngelesSession Date: Friday, October 16, 2020Session Time: 10:45 a.m. 12:01 p.m. CESTLecture Time: 11:45 a.m. CESTLocation: Hall D

This session will be followed by a Q&A from 12:01 p.m. to 12:30 p.m. CEST

E-Poster

Title: Preclinical Efficacy and Safety of EFS.HTCIRG1-LV Supports IMO Gene Therapy Clinical Trial InitiationPresenter: Ilana Moscatelli, Ph.D., Associate Researcher, Division of Molecular Medicine and Gene Therapy, Lund University, Sweden

About Leukocyte Adhesion Deficiency-I

Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I (less than 2% normal expression) are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other Agency of the State of California.

About Infantile Malignant Osteopetrosis

Infantile Malignant Osteopetrosis (IMO) is a rare, severe autosomal recessive disorder caused by mutations in the TCIRG1 gene, which is critical for the process of bone resorption. Mutations in TCIRG1 interfere with the function of osteoclasts, cells which are essential for normal bone remodeling and growth, leading to skeletal malformations, including fractures and cranial deformities which cause neurologic abnormalities including vision and hearing loss. Patients often have endocrine abnormalities and progressive, frequently fatal bone marrow failure. As a result, death is common within the first decade of life. IMO has an estimated incidence of 1 in 200,000. The only treatment option currently available for IMO is an allogenic bone marrow transplant (HSCT), which allows for the restoration of bone resorption by donor-derived osteoclasts which originate from hematopoietic cells. Long-term survival rates are lower in IMO than those associated with HSCT for many other non-malignant hematologic disorders; severe HSCT-related complications are frequent. There is an urgent need for additional treatment options.

RP-L401 was in-licensed from Lund University and Medizinische Hochschule Hannover.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is advancing an integrated and sustainable pipeline of genetic therapies that correct the root cause of complex and rare childhood disorders. The companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, Pyruvate Kinase Deficiency (PKD) a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding its guidance for 2020 in light of COVID-19, the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding when clinical trial sites will resume normal business operations, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-Q for the quarter ended June 30, 2020, filed August 5, 2020 with the SEC. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Announces Two Presentations at the European Society for Immunodeficiencies 2020 Meeting - Business Wire

LONDONandNEW YORK, Oct. 03, 2020 (GLOBE NEWSWIRE) -- MeiraGTx Holdings plc(Nasdaq: MGTX), a vertically integrated, clinical stage gene therapy company, today announced nine-month data from the ongoing Phase 1/2 clinical trial (NCT03252847) of AAV-RPGR, an investigational gene therapy in development for the treatment of patients with X-linked retinitis pigmentosa (XLRP). Data presented today at the EURETINA 2020 Virtual Congress demonstrated significant improvement in vision-guided mobility and retinal sensitivity in treated eyes compared to untreated eyes nine months after treatment.

MeiraGTx and Janssen Pharmaceuticals, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of Johnson & Johnson, are jointly developing AAV-RPGR as part of a broader collaboration to develop and commercialize gene therapies for the treatment of inherited retinal diseases.

In July 2020, MeiraGTx and Janssen announced six-month data from the ongoing MGT009 clinical trial showing significant improvement in retinal sensitivity in the low (n=3) and intermediate (n=4) dose cohorts in the dose escalation phase of the trial.

Data at the nine-month time point continued to demonstrate significant improvement in retinal sensitivity in treated eyes in both the low and intermediate dose cohorts. In addition, data from the assessment of vision-guided mobility carried out at the nine-month timepoint demonstrated a significant improvement in walk time compared to baseline in treated eyes compared to untreated eyes in the low and intermediate dose cohorts (n=6).

There are currently no treatment options for XLRP, and vision in patients suffering from this disease inevitably declines over time, said Michel Michaelides1, BSc MB BS MD(Res) FRCOphth FACS, MGT009 trial investigator, Consultant Ophthalmologist, Moorfields Eye Hospital and Professor of Ophthalmology, University College London. Data from this clinical trial demonstrate that patients treated with AAV-RPGR had significant and sustained improvement in retinal sensitivity, as well as improved ability to navigate in low light conditions. These exciting results continue to suggest that AAV-RPGR has the potential to be a much-needed and important treatment option for those living with XLRP.

EURETINA Data Summary:

Retinal sensitivity XLRP is characterized by progressive deterioration of the visual field. Octopus 900 full-field static perimetry and MAIA microperimetry were employed to determine change in retinal sensitivity following intervention.

Perimetry is a sensitive standard-of-care measure of retinal function that reproducibly determines retinal sensitivity both cross-sectionally and longitudinally, thereby accurately defining disease progression over time.

At the nine-month analysis (Octopus 900 static perimetry), compared to baseline:

Vision-guided mobility Markedly impaired mobility in low illumination is a hallmark symptom of XLRP. As part of the study, patients completed a vision-guided mobility maze to assess their ability to navigate across a broad range of controlled light levels (1 lux = deep twilight, 4 lux = residential street lighting, 16 lux = twilight conditions, 64 lux = car park and 256 lux = office work).

At nine-month analysis, compared to baseline:

Safety and tolerabilitySafety data obtained to date continue to suggest AAV-RPGR is well-tolerated. No dose-limiting events occurred. As previously presented, signs of inflammation were observed in two out of three patients in the high dose cohort, which may have been associated with decreased activity of the AAV-RPGR treatment in these patients. Inflammation was effectively managed with an extended steroid protocol.

Based on the safety and efficacy profile demonstrated to date, the low and intermediate doses are being evaluated in the ongoing randomized, controlled expansion portion of the Phase 1/2 study, which completed enrollment in the first half of 2020. As previously disclosed, MeiraGTx and development partner Janssen plan to advance AAV-RPGR into a Phase 3 pivotal study, called the Lumeos clinical trial.

About AAV-RPGRAAV-RPGR is an investigational gene therapy for the treatment of patients with XLRP caused by disease-causing variants in the eye specific form of the RPGR gene (RPGR ORF15). AAV-RPGR is designed to deliver functional copies of the RPGR gene to the subretinal space in order to improve and preserve visual function. MeiraGTx and development partner Janssen are currently conducting a Phase 1/2 clinical trial of AAV-RPGR in patients with XLRP with disease-causing variants in RPGR ORF15. AAV-RPGR has been granted Fast Track and Orphan Drug designations by the U.S. Food and Drug Administration (FDA) and PRIME, ATMP and Orphan designations by the European Medicines Agency (EMA).

About the Phase 1/2 MGT009 Clinical TrialMGT009 is a multi-center, open-label Phase 1/2 trial (NCT03252847) of AAV-RPGR gene therapy for the treatment of patients with XLRP associated with disease-causing variants in the RPGR gene. MGT009 consists of three phases: dose-escalation, dose-confirmation, and dose-expansion. Each patient was treated with subretinal delivery of AAV-RPGR in the eye that was more affected at baseline. The patients other eye served as an untreated control. In dose-escalation (n=10), adults were administered low, intermediate, or high dose AAV-RPGR. The primary endpoint was safety. Visual function was assessed at baseline, three, six, nine and 12 months with Octopus 900 full-field static perimetry and mesopic fundus-guided microperimetry (MP); mean retinal sensitivity, visual field modeling and analysis (VFMA; Hill-of-vision volumetric measure), and pointwise comparisons were examined.

About X-Linked Retinitis Pigmentosa (XLRP)XLRP is the most severe form of retinitis pigmentosa (RP), a group of inherited retinal diseases characterized by progressive retinal degeneration and vision loss. In XLRP, both rods and cones function poorly, leading to degeneration of the retina and total blindness. The most frequent cause of XLRP is disease-causing variants in the RPGR gene, accounting for more than 70% of cases of XLRP, and up to 20% of all cases of RP. There are currently no approved treatments for XLRP.

AboutMeiraGTxMeiraGTx(Nasdaq: MGTX) is a vertically integrated, clinical stage gene therapy company with six programs in clinical development and a broad pipeline of preclinical and research programs.MeiraGTx has core capabilities in viral vector design and optimization and gene therapy manufacturing, as well as a potentially transformative gene regulation technology. Led by an experienced management team,MeiraGTxhas taken a portfolio approach by licensing, acquiring and developing technologies that give depth across both product candidates and indications. MeiraGTxs initial focus is on three distinct areas of unmet medical need: inherited retinal diseases, neurodegenerative diseases and severe forms of xerostomia. Though initially focusing on the eye, central nervous system and salivary gland,MeiraGTxintends to expand its focus in the future to develop additional gene therapy treatments for patients suffering from a range of serious diseases.

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

Forward Looking StatementThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the development and efficacy of AAV-RPGR, plans to advance AAV-RPGR into Phase 3 clinical trial and anticipated milestones regarding our clinical data and reporting of such data and the timing of results of data, including in light of the COVID-19 pandemic, as well as statements that include the words expect, intend, plan, believe, project, forecast, estimate, may, should, anticipate and similar statements of a future or forward-looking nature. These forward-looking statements are based on managements current expectations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, our incurrence of significant losses; any inability to achieve or maintain profitability, acquire additional capital, identify additional and develop existing product candidates, successfully execute strategic priorities, bring product candidates to market, expansion of our manufacturing facilities and processes, successfully enroll patients in and complete clinical trials, accurately predict growth assumptions, recognize benefits of any orphan drug designations, retain key personnel or attract qualified employees, or incur expected levels of operating expenses; the impact of the COVID-19 pandemic on the status, enrollment, timing and results of our clinical trials and on our business, results of operations and financial condition; failure of early data to predict eventual outcomes; failure to obtain FDA or other regulatory approval for product candidates within expected time frames or at all; the novel nature and impact of negative public opinion of gene therapy; failure to comply with ongoing regulatory obligations; contamination or shortage of raw materials or other manufacturing issues; changes in healthcare laws; risks associated with our international operations; significant competition in the pharmaceutical and biotechnology industries; dependence on third parties; risks related to intellectual property; changes in tax policy or treatment; our ability to utilize our loss and tax credit carryforwards; litigation risks; and the other important factors discussed under the caption Risk Factors in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2020, as such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SECs website at http://www.sec.gov. These and other important factors could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent managements estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, unless required by law, we disclaim any obligation to do so, even if subsequent events cause our views to change. Thus, one should not assume that our silence over time means that actual events are bearing out as expressed or implied in such forward-looking statements. These forward-looking statements should not be relied upon as representing our views as of any date subsequent to the date of this press release.

Contacts

Investors:MeiraGTxElizabeth (Broder) Anderson(646) 860-7983elizabeth@meiragtx.com

or

Media:W2O pureChristiana Pascale (212) 257-6722cpascale@purecommunications.com

_____________________________1 Professor Michaelides is a scientific founder of and consultant to MeiraGTx.

Originally posted here:
MeiraGTx Announces Nine-Month Data from Phase 1/2 Trial of AAV-RPGR Demonstrating Significant and Sustained Vision Improvement in X-Linked Retinitis...

Gene Therapy Market analysis report encompasses infinite knowledge and information on what the markets definition, classifications, applications, and engagements are and also explains the drivers & restraints of the market which is obtained from SWOT analysis. Gathered market data and information is denoted very neatly with the help of most appropriate graphs, charts or tables in the entire report. Utilization of well established tools and techniques in this Gene Therapy Market document helps to turn complex market insights into simpler version. Competitive analysis studies of this market report provides with the ideas about the strategies of key players in the market.

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Market Analysis: Global Gene Therapy Market

Global gene therapy market is rising gradually with a healthy CAGR of 36.1% in the forecast period of 2019-2026. Increasing incidence of cancer and rare life threatening diseases and strong clinical pipeline drugs for gene therapy are major drivers for market growth.

Key Market Players:

Few of the major competitors currently working in the globalgene therapy marketarePfizer Inc., Thermo Fisher Scientific Inc., F. Hoffmann-La Roche Ltd, Spark Therapeutics, Inc., bluebird bio, Inc., ALLERGAN, Krystal Biotech, Inc., Amicus Therapeutics, Inc., Sarepta Therapeutics, Novartis AG, MeiraGTx Limited, Rocket Pharmaceuticals, Lonza, Biogen, Gilead Sciences, Inc., REGENXBIO Inc., uniQure N.V., Solid Biosciences Inc., Audentes Therapeutics among others.

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Market Definition: Global Gene Therapy Market

Gene therapy is a technique of insertion of genes into cells and tissues for treatment of any disease. In this technique the defective gene is replaced with a functional gene. It is the strategy of manipulation of expression of specific genes responsible for the disease. This therapy is a promising treatment option for a number of diseases. The application of gene therapy is wide and it is mostly used for treatment of cancer, cystic fibrosis, heart disease, diabetes, AIDS among others.

Gene Therapy Market Drivers

Gene Therapy Market Restraints

Segmentation:Global Gene Therapy Market

Gene Therapy Market : By Type

Gene Therapy Market : By Gene Type

Gene Therapy Market : By Viral Vector

Gene Therapy Market : By Non-Viral Vector

Gene Therapy Market : By Application

Gene Therapy Market : By End Users

Gene Therapy Market : By Distribution Channels

Gene Therapy Market : ByGeography

Key Developments in the Market:

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Originally posted here:
Gene Therapy Market Sees a Faster Rebound in the Era of COVID-19 || Leading Players ALLERGAN, Krystal Biotech, Inc., Amicus Therapeutics, Inc.,...

Denise Paglinawan , The Canadian Press Published Wednesday, September 30, 2020 10:22PM EDT

The father of a young Ontario boy with a rare genetic disease is asking the federal government to help fund research that could treat his child's condition, saying the support is particularly needed since the COVID-19 pandemic has made it tough to raise donations.

Terry Pirovolakis said his two-year-old son Michael was diagnosed last year with SPG50, an extremely rare disorder that causes a loss of mobility and a decline in brain functions over time.

His family has been raising money for research that could help develop treatment for Michael but the pandemic has meant many planned fundraising events had to be cancelled, he said.

It's been very difficult because we had to stop all of our fundraising campaigns, Pirovolakis said. We're all in the same situation where our lives are turned upside down but ours is a bit more complex, where we're trying to build a cure.

The family noticed Michael was not meeting his milestones as a baby, Pirovolakis said. After many tests and doctors appointments, the young boy was diagnosed with the disease that could lead to developmental delays and confine him to a wheelchair by the age of 10, the family said.

Determined to help their son, the Pirovolakis family has been trying to raise money to get the child into experimental treatment.

The family had put on events that included a golf tournament, a gala and a Christmas market to fundraise last year, but can now only rely largely on online campaigns such as their GoFundMe page while COVID-19 restrictions are still in place, Pirovolakis said.

In an effort to keep raising funds and awareness for their son's condition, Pirovolakis said he planned to bike from Pickering, Ont., to Ottawa - starting on Saturday - and has asked to meet with the Prime Minister Justin Trudeau next week.

We're seeing if there's any way that we can get some sort of funding from the federal government, he said.

Hopefully, we'll meet Mr. Trudeau and bring awareness and bring funding and support not just for my son, but for the other children affected by this disease.

The family has raised almost $1.6 million so far but is hoping to raise $3 million. Pirovolakis said research on SPG50 is being conducted in several hospitals outside Canada and involves gene therapy.

Alex Wellstead, a spokesman for the prime minister's office, said the government is in contact with the family.

It is truly inspiring to see the love and dedication that the Pirovolaskis' have for their son Michael and the hard work they've put towards this campaign, he said in an email. We will let you know when we have more to say.

This report by The Canadian Press was first published on Sept. 30, 2020.

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Family of boy with rare disease asking Ottawa to fund research into condition - CP24 Toronto's Breaking News

Basel, October 1, 2020 Novartis Gene Therapies today announced new interim data from the ongoing Phase 3 STR1VE-EU clinical trial for Zolgensma (onasemnogene abeparvovec) that demonstrated patients with spinal muscular atrophy (SMA) Type 1 continued to experience significant therapeutic benefit, including event-free survival, rapid and sustained improvement in motor function and motor milestone achievement, including for some patients with more aggressive disease at baseline compared to previous trials. SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene that results in the progressive and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing, and basic movement.1,2,3 These data as of December 31, 2019, and presented today during a virtual Clinical Trial Poster Session as part of the World Muscle Society (WMS) 2020 Virtual Congress, support the robust clinical evidence that has demonstrated a consistent, transformative benefit across Zolgensma clinical trials for the treatment of patients with SMA.

We are seeing further evidence of the potential of Zolgensma to effectively halt motor neuron loss following a one-time, intravenous infusion. In STR1VE-EU, patients achieved rapid improvements in motor function following treatment with Zolgensma, and most have already achieved motor milestones not observed in the natural history of SMA Type 1, said Professor Eugenio Mercuri, M.D., PhD., Department of Pediatric Neurology, Catholic University, Rome, Italy. These interim results are especially encouraging considering STR1VE-EU includes some patients with a more severe phenotype than in the START and STR1VE-US studies, further supporting the gene therapys positive benefit/risk profile, even in this more fragile population.

"These strong interim results from the STR1VE-EU clinical trial continue to demonstrate consistent and significant therapeutic benefit in patients with SMA Type 1, the most common form of the disease, adding to the robust body of clinical evidence for Zolgensma, said Shephard Mpofu, M.D., SVP, Chief Medical Officer, Novartis Gene Therapies. With more than 600 patients now treated, including some more than five years post-treatment and more than five years old, these data further reinforce the transformative benefit a one-time dose of Zolgensma has on SMA patients.

Phase 3 STR1VE-EU Data as of December 31, 2019STR1VE-EU is designed to evaluate the efficacy and safety of a single, one-time IV infusion of Zolgensma in patients with SMA Type 1 who are less than six months of age at the time of gene therapy, with one or two copies of theSMN2backup gene and who have bi-allelicSMN1gene deletion or point mutations. The mean age of dosing was 4.1 months and the mean age at the onset of symptoms was 1.6 months. The mean Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) score at baseline was 28. Thirty-one of 33 patients (93.9%) were able to swallow thin liquids, and 10 patients (30.3%) required feeding support at baseline. Nine of thirty patients (27.3%) required ventilatory support at baseline. STR1VE-EU is distinct in its inclusion and exclusion criteria and baseline clinical characteristics of enrolled patients compared with START or STR1VE-US. Specifically, some patients in STR1VE-EU had a more severe disease phenotype at baseline, including lower CHOP-INTEND scores and the need for nutritional and ventilatory support.

At last visit before the data cutoff, patients in STR1VE-EU were between 6.9 and 18.6 months of age, and mean duration in the study was 10.6 (1.815.4) months. Thirty-one out of 32 (97%) patients in the intent-to-treat (ITT) population survived event-free, including 30 (93.8%) who could have reached 10.5 months of age and 18 (56.3%) who could have reached 13.6 months of age. An event is defined as the need for tracheostomy or the requirement of 16 hours of respiratory assistance per day (via non-invasive ventilatory support) for 14 consecutive days in the absence of an acute reversible illness, excluding peri-operative ventilation. Untreated natural history indicates that only 50% and 25% of babies with SMA Type 1 will survive event-free by the time they reach 10.5 months of age and 13.6 months of age, respectively.3

Twenty-one patients (65.6%) achieved motor milestones not observed in the natural history of SMA Type 1. This includes six patients (18.8%) who could sit independently for 10 seconds (the primary efficacy endpoint), 20 patients (66.7%) who gained head control, eight patients (25%) who were able to roll from back to sides and one patient who could stand with assistance, crawl and walk with assistance. The mean increase in CHOP INTEND from baseline was 5.9 points (n=31) which was observed as early as at one month post-dosing, 10.1 points at 3 months (n=29) post-dosing, and 13.3 points at six months (n=27) post-dosing. Twenty-one children (65.6%) enrolled in STR1VE-EU achieved and maintained a CHOP INTEND score of 40 points and 12 children (37.5%) were able to achieve a score of 50. According to natural history, untreated patients with SMA Type 1 almost never achieve a CHOP INTEND score 40.3,4

The majority (91.7%) of patients who were free of ventilatory support at baseline remained either completely free of ventilatory support or received prophylactic BiPAP support during the study for acute reasons. Two-thirds (66.7%) of patients in the ITT population were able to feed orally without the need for feeding support, an important indicator of stabilization/halting of disease progression.

As previously reported, one patient discontinued the study because of a serious adverse event of hypoxic-ischemic brain damage and respiratory distress that resulted in death. Novartis and the investigator considered the events and death to be unrelated to treatment with Zolgensma based on autopsy findings. Thirty-two of 33 patients were reported to have at least one adverse event (AE), of which six patients experienced serious adverse events that were considered by the investigator to be related to Zolgensma. Liver transaminase elevations, some of which were reported as adverse events, were experienced by 29 of 33 patients (87.9%), but all resolved with the use of prednisolone. Four patients had reported decreases in platelet counts <75,000, three of which were isolated laboratory abnormalities without adverse events reported. Overall, no new safety signals have been identified and the reported adverse events are consistent with the cumulative safety profile with Zolgensma.

Novartis Gene Therapies is grateful to the courageous patients and families who participate in clinical trials, enabling the company to further its efforts to make a meaningful difference in the lives of patients with rare genetic diseases.

About Zolgensma (onasemnogene abeparvovec)Zolgensma is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time IV infusion. Zolgensma was approved by the U.S. Food and Drug Administration in May 2019 and represents the first approved therapeutic in Novartis Gene Therapies proprietary platform to treat rare, monogenic diseases using gene therapy.5 In addition to the United States, Zolgensma is approved in Japan, Europe and Brazil. More than 600 patients have been treated with Zolgensma, including clinical trials, commercially and through the managed access program. Novartis Gene Therapies is pursuing registration in close to three dozen countries with regulatory decisions anticipated in Switzerland, Canada, Israel, Australia, and South Korea in late-2020 or early 2021.5

Novartis Gene Therapies has an exclusive, worldwide license with Nationwide Children's Hospital to both the intravenous and intrathecal delivery of AAV9 gene therapy for the treatment of all types of SMA; has an exclusive, worldwide license from REGENXBIO for any recombinant AAV vector in its intellectual property portfolio for the in vivo gene therapy treatment of SMA in humans; an exclusive, worldwide licensing agreement with Gnthon for in vivo delivery of AAV9 vector into the central nervous system for the treatment of SMA; and a non-exclusive, worldwide license agreement with AskBio for the use of its self-complementary DNA technology for the treatment of SMA.

About Spinal Muscular AtrophySMA is the leading genetic cause of infant death.1,2 If left untreated, SMA Type 1 leads to death or the need for permanent ventilation by the age of two in more than 90% of cases.3,4SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement.1 It is imperative to diagnose SMA and begin treatment, including proactive supportive care, as early as possible to halt irreversible motor neuron loss and disease progression.5 This is especially critical in SMA Type 1, where motor neuron degeneration starts before birth and escalates quickly. Loss of motor neurons cannot be reversed, so SMA patients with symptoms at the time of treatment will likely require some supportive respiratory, nutritional and/or musculoskeletal care to maximize functional abilities.6 More than 30% of patients with SMA Type 2 will die by age 25.7

About Novartis Gene TherapiesNovartis Gene Therapies (formerly AveXis) is reimagining medicine to transform the lives of people living with rare genetic diseases. Utilizing cutting-edge technology, we are turning promising gene therapies into proven treatments, beginning with our transformative gene therapy for spinal muscular atrophy (SMA). This therapy is now approved in the U.S., Japan, Europe and Brazil, and additional registrations are being pursued in close to three dozen countries, with regulatory decisions anticipated in Switzerland, Canada, Israel, Australia, Argentina and South Korea in late 2020 or early 2021. Our robust AAV-based pipeline is advancing treatments for Rett syndrome; a genetic form of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene; and Friedreichs ataxia. We are powered by the worlds largest gene therapy manufacturing footprint of more than one million square feet, enabling us to bring these therapies to patients around the world at quality and scale.

DisclaimerThis press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as potential, can, will, plan, may, could, would, expect, anticipate, seek, look forward, believe, committed, investigational, pipeline, launch, or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases such as COVID-19; safety, quality, data integrity or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AGs current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About NovartisNovartis is reimagining medicine to improve and extend peoples lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the worlds top companies investing in research and development. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of more than 140 nationalities work at Novartis around the world. Find out more athttps://www.novartis.com.

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References1. Anderton RS and Mastaglia FL. Expert Rev Neurother. 2015;15:895908.2. National Organization for Rare Disorders (NORD). Spinal Muscular Atrophy. Available at: http://rarediseases.org/rarediseases/spinal-muscular-atrophy/. Accessed October 29, 2019.3. Finkel RS, McDermott MP, Kaufmann P. et al. Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurology. 2014;83:8107.4. Kolb SJ, et al. Ann Neurol. 2017;82:88391.5. SolerBotija C, et al. Brain. 2002;125:162434.6. Wang CH, et al. J Child Neurol. 2007;22:102749.7. Darras BT, Finkel RS. Natural history of spinal muscular atrophy. In: Sumner CJ, Paushkin S, Ko CP, eds. Spinal Muscular Atrophy: Disease Mechanisms and Therapy, 2nd ed. London, UK: Academic Press/Elsevier;2017:399421.

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Zolgensma data including patients with more severe SMA at baseline further demonstrate therapeutic benefit, including prolonged event-free survival,...