Entrepreneur Dave Aspreys end-of-life plans are quite simple, really, even if some of his ambitions sound laughably optimistic to most of us.I want to die at a time and by a method of my own choosing, and keep doing awesome things until that day, he tells me. I dont think its outrageous to believe Ill make it to 180 years old. And if I run out of energy, itll just be because I did too much cool shit for my own good.

Asprey is strolling across his lush property in British Columbia, holding up his phone and pointing out the specimens in this years garden as we chat over Zoom in the midst of the global pandemic. Hes protecting his skin from the sun with a goofy Outdoor Research hat and wearing a long string of beads that he says are each over a hundred years old, from cultures around the world.

Asprey, 48, is the founder of the Bulletproof wellness empire and a vocal champion of the movement to extend human life expectancy beyond 100 years. Hes made millions by experimenting on his own body and packaging his home-brewed discoveries into books, a podcast, consulting services and consumer products (you may have even tried his butter-laced coffee). Asprey, who was a web-security executive before he became the Bulletproof Executive, is just one of a cadre of tech elite who have begun directing their attentionand truckloads of moneytoward the problem of life extension. Jeff Bezos, Peter Thiel, Sergey Brin, Larry Ellisonname a Silicon Valley A-lister and he or she is likely funding longevity research, experimenting with anti-aging interventions or both. These are the masters of the universe who see no reason they cant take the tech industrys optimization obsession and apply it to the ultimate challenge: conquering death itself.

And their efforts appear to be paying off: Thanks to a recent explosion of advances in longevity medicine, Aspreys vision of living healthfully into his second century might not be so crazy. In fact, for people in middle age right now, a handful of therapies in clinical trials have the potential, for the first time in human history, to radically transform what old age looks like. If the life extensionists are right, a person whos 40 today might reasonably expect to still be downhill skiing, running a 10K or playing singles tennis at 100.

Dave AspreyDave Asprey

If you do anti-aging right, Asprey insists, youll have a level of resilience and energy to fight what comes your way. If you get Covid-19, youre less likely to become very sick. The idea is that at a cellular level, youre making yourself very hard to kill.

The most extreme of the controversial interventions Asprey has undergone involved having stem cells extracted from his own bone marrow and fat and then injected into hundreds of locations on his body. Into every joint, between every vertebra and into my cerebrospinal fluid, face and sex organs, he tells me cheerfully. For what I spent on that, I could have bought a really nicely appointed Tesla.

He trots up a flight of stairs to his home office, which sits above a million-dollar lab filled with health gadgets, such as a cryochamber, a hypoxic trainer and an AI-enabled stationary bike. For a wealthy person, investing in your body should be a major part of your Im rich strategy, he explains. Personally, I think you should be spending at least 2 to 3 percent of your net worth on health and longevity. Get a personal chef who can cook you the right food. Its not that hard.

It might be an exaggeration to say BioViva CEO Liz Parrish believes death is optional, but for her, Aspreys goal of living to 180 shows a distinct lack of ambition. If you can reach homeostasis in the body, Parrish says, where its regenerating itself just a little bit faster than its degrading, then what do you die of? An accident or natural disaster, probably. Theres no expiration date at 90 or 100 years old.

Tall, blond and fit, Parrish cuts a strikingly youthful figure at 49one that might convince you to order whatever shes having. But, like Asprey, she has received criticism from the longevity research community for becoming patient zero in her own experimental drug trial, aimed at halting aging at the cellular level. In 2015, Parrish underwent telomerase and follistatin gene therapies in Bogota, Colombia. The procedures involved receiving around a hundred injections of a cocktail of genes and a virus modified to deliver those new genes into her bodys cells. The objective was to prevent age-related muscle loss and lengthen her telomeres: the caps at the end of our chromosomes. Scientists have identified their unraveling as not only a marker of aging but also a potential cause of age-related decline.

Liz ParrishLiz Parrish

Parrish told the media about her clandestine experiment and has published periodic updates on her condition in the five years since, and she reports that she has indeed increased her muscle mass and lengthened her telomeres. Parrishs punk-rock approach stems from her conviction that the medical-research communityboth the Food and Drug Administration (FDA) and researchers who arent business-mindedis moving too slowly, with too much red tape, when it comes to advancing aging therapeutics. But gene therapy is a relatively new area of medicine that brings with it a host of new risks, including cancer, severe immune reactions and infections caused by the viral vector used to deliver the drug.

Parrish downplays such worries. There may be risks, she tells Robb Report. But the known risk is that youre 100 percent likely to die. So you have to decide for yourself if the potential benefit outweighs that.

Humans have always aspired to find the fountain of youth, so people might be skeptical about the fact that anti-aging technologies are working now, says British investor and businessman Jim Mellon. But the fact is that this is finally happening, and we need to seize the moment. Mellon cofounded Juvenescence, a three-year-old pharmaceutical company thats investing in multiple technologies simultaneously to increase the odds of bringing winning products to market.

Mellon, 63, has made his fortune betting on well-timed investment opportunities, and he predicts that a new stock-market mania for life extension is just around the corner. This is like the internet dial-up phase of longevity biotech, he enthuses. If youd invested in the internet in the very early days, youd be one of the richest people on the planet. Were at that stage now, so the opportunity for investors is huge. According to a report by Bank of America Merrill Lynch, hes not wrong: The market for technologies to increase human life span is projected to grow sixfold to $610 billion in just the next five years.

When I talk to Mellon in the late spring, hes sequestered on the rugged coast of the Isle of Man, a tiny spit of land in the Irish Sea. Despite being what he describes as imprisoned there for 15 weeksand countingduring the Covid-19 shutdown, hes jovial and chatty and wants to make it clear that his interest in life extension is much more than financial. Working to extend life is an ethical cause, he says. If we can help people to live healthfully until the end of life, well transform the world completely. Well reduce a huge amount of pressure on failing health-care systems, and well have to reimagine pension and life insurance. This should be the number-one tick in anyones investment portfolio.

If youd like to get on board with this social-impact view of longevity, it helps to understand the trajectory of aging today. In Americas most affluent neighborhoods, the average life span is about 88 years. (Meanwhile, in this countrys poorest, it hovers around a meager 66 because of a raft of inequalities, such as diet, stress, smoking, pollution and health care.) For most people, health starts gradually diminishing in the last 15 years of life with the onset of chronic conditions, including arthritis, neurodegeneration and diabetes. If we could eliminate such diseases of aging, experts say, the US could save an estimated $7.1 trillion in health-care costs over the next 50 years. (Quite where all these sprightly centenarians might live on this already densely populated planet remains to be seen.)

Jim MellonEric Verdin

One of Mellons bets is on a class of drugs called senolytics, which destroy senescent cells: the so-called zombie cells that, for complex reasons, stop dividing as we age. Senescent cells harm the body by secreting compounds that cause inflammation in surrounding tissues. Many age-related conditionsarthritis, diabetes, Alzheimers, cancerhave an inflammatory component, and studies suggest that a buildup of senescent cells is a large part of the problem.

A number of biotech start-ups are devel- oping drugs that target cell senescence, but the furthest along is Unity Biotechnology, a company in South San Francisco that has three drugs in clinical trials to address aging conditions, starting with osteoar- thritis of the knee. Unity raised more than $200 million from such big names as Thiel and Bezos, who chipped in through their investment firms, before going public in 2018. Since then, Mellon has also bought a small stake.

The holy grail of senolytics will be the development of a preventive therapy to wipe out senescent cells in the body before they cause conditions of aging, theoretically extending life span. In June, a team from Sloan Kettering published new breakthrough research showing that CAR T cellstypically used for precision cancer therapycan also be used to target and kill senescent cells. Prescription senolytics for anti-aging therapy are still years away, but unsurprisingly, theres an audience of longevity enthusiasts who want to access such anti-aging miracles yesterdayand no shortage of FDA-unapproved ways to chase after them. For instance, after a few studies examined the senolytic effects of a chemotherapy drug called dasatinib, the website FightAging.org published a step-by-step guide to senolytic self-experimentation using chemotherapeutics.

It doesnt take a Ph.D. in biochemistry to guess that taking off-label chemo drugs might come with harmful side effects, but that hasnt stopped a zealous group of body-hackers from trying it themselves and chronicling their efforts online. The internet is littered with novice longevity adviceand sketchy anti-aging companies eager to separate the hopeful and desperate from their money, like the company that charges $8,000 for transfusions of plasma from the blood of teenagers and early-twentysomethings (yes, just like Gavin Belson on HBOs Silicon Valley). Many of these are at best ineffective and at worst deadly, since the same cellular systems that fuel growth in young people might cause cancer when tipped into overdrive. Imagine the tragic irony of paying tens of thousands for a therapy that promises to help you live longer but actually causes the cancer that kills you.

Adobe

Beyond the obvious red flags of repurposed chemo drugs and the bloodletting of teens, it can be difficult for a layperson to separate the world-changing longevity breakthroughs from the terrible ideas. Enter one of the worlds leading experts on longevity to help make sense of things.

Eric Verdin, 63, is president and CEO of the Buck Institute, a globally renowned center for aging research just outside San Francisco in Marin County. Verdin is bullish on the promise of living healthfully to at least 100. Today. But 180? Dont count on it. My prediction, based on everything we know today, is that getting to 120 is about the best we can do for the foreseeable future. Ill bet my house were not going to see anyone live to 180 for another 200 years, if ever, he says. But making everyone a healthy centenarian, this is something we can do today. And thats something to be excited about.

Verdins own lab at the Buck Institute studies the aging immune system and how its affected by lifestyle factors, such as nutrition and exercise. Informed by this research, Verdin follows a time-restricted diet in which he eats all of his meals in an eight-to-nine-hour window (similar to the Buchinger Wilhelmi process) and gets plenty of exercise mountain biking in Marins steep hills. The good news is that over 90 percent of what causes diseases of aging is environmental, and that means its within your control, he says.

But he emphasizes that responsible management of your health comes with limits, like avoiding experimental therapies. A group of people have decided to try some expensive and dangerous interventions, but there is zero evidence that any of these are going to help them live longer, he says. The problem, according to Verdin, is that the results of aging interventions in mouse trials can look very promising but rarely translate to success in humans. Theres a huge delta between the health of a stressed lab mouse and an optimally healthy mouse, Verdin says. So when you treat lab mice with longevity therapeutics, you see an outsized result that doesnt at all guarantee the same result in humans.

On the other hand, Verdin tells Robb Report, there are definitely new protocols worth getting excited about. Take, for instance, rapalogs, a class of drugs that interact with a protein called mTOR, which serves as a linchpin for multiple critical biological processes, including cell growth and metabolism. Rapalog drugs tamp down mTOR, possibly preventing age-related diseases such as diabetes, stroke and some cancers. The drug rapamycin, the most heavily studied formula, was approved in the US in 1999 to help prevent organ-transplant rejection. Last year the medical journal Aging published a rapturous opinion piece by oncologist Mikhail Blagosklonny in which he made the case that rapamycinin small or intermittent dosesis effective as a preventive treatment to ward off diseases of aging, and that, in the elderly, not taking rapamycin may be even more dangerous than smoking.

Eric VerdinJim Hughes Photography

Later this year, a biotech firm called resTORbio, which was spun out of the Swiss-based Big Pharma company Novartis in 2017, is expected to seek FDA approval for its rapalog RTB101, which clinical trials have shown to slow age-related decline of the immune system and improve immune response in elderly people by more than 20 percent, a key factor in protecting vulnerable aging populations from disease. (It is currently in trials on elderly patients with Covid-19.) This is the furthest-along program of anything in the aging field, Joan Mannick, cofounder and chief medical officer of resTORbio, told MIT Technology Review last year. If health authorities approve this drug well have a product for people to prevent age-related diseases. Not just in our lifetime, but in, you know, a few years.

One of the many effects of rapamycin is that it mimics the mechanisms of calorie restriction. As Verdins lab and others have shown, fasting provides a number of anti-aging benefits, including insulin regulation, reduced inflammation and, to put it colloquially, clearing out the gunky by-products of metabolismpart of the reason Twitter CEO Jack Dorsey and other tech titans eat just a few meals per week. For lesser mortals, fasting is extremely hard to commit to and not much fun, hence the huge interest in calorie-restriction mimetics like rapamycin, which provide all the benefits without the downer not-eating part.

Of all the calorie-restriction mimetics, the one sparking the most excitement among longevity researchers is already on the market: metformin, a decades-old diabetes drug. Metformin became a part of the Silicon Valley health regimen several years ago after an epidemiological study showed that Type 2 diabetics who took the drug lived longer than non-diabetics who didnt. Just about everyone in the longevity industry takes metformin, Verdin tells me. He takes it himself, and nearly everybody I interviewed is taking or has taken it, too.

In April, Nir Barzilai, the renowned endocrinologist who spearheaded research on the anti-aging properties of metformin, announced in an opinion piece he co-authored in the journal Cell Metabolism that his lab is launching a large clinical trial to investigate the anti-aging effects of the drug on non-diabetic populations. Barzilais goal is to prove to the FDA that aging itselfrather than conditions associated with it, like Alzheimers and arthritiscan be targeted as a disease. If Barzilai is successful and the FDA approves aging as a treatment indication, the process of bringing longevity therapies to market would accelerate rapidly.

Just as the FDA was able to move faster to bring Covid-19 therapies to market this year, we will reach a tipping point when public opinion pushes the FDA to approve aging as an indication, and the longevity-research field will make leaps as a result, Mellon says. He has contributed funding to Barzilais metformin research, which he believes will be instrumental in proving that there are compounds that can extend human life across the board.

The fact of the matter is that the US has the best regulatory system for new drug development in the world, Mellon says. Were in the first era ever when humans can be bioengineered to live longer. And in 10 years, well have solutions that are even better than today. Just wait, its coming.

Liz Parrish

Jim Mellon

Diet:Vegetarian.Mindfulness practice:Nightly meditation.

Exercise regimen:30 minutes of cardio and 10 minutes of weights,five days a week.

Anti-aging Rx:Regenerative gene therapies. Im certain most peoplewill take them in the next couple decades.

180th-birthday wish:Solving another critical issue.

Sleep routine:7.5 hours plus a 30-minute nap; in bed by 9 p.m.

Vitamins/supplements/ prescription meds:Vitamins D and B12, metformin.

Exercise regimen:Walk or run minimum 10,000 steps a day;weights three times week.

Anti-aging Rx:Green tea.

100th-birthday wish:Another 25 years.

Dave Asprey

Jim Hughes Photography

180th-birthday wish:Either a cruise to Mars or a 1970 Mustang Fastback,which by then will be 210 years old!

Sleep Routine:Avoid: coffee after 2 p.m., heavy workouts after 6 p.m.,alcohol during the week and heavy eating in the evening.

Vitamins/supplements:Vitamin D, omega fatty acids, NMN, citrus bioflavonoidcomplex, fiber supplement, prebiotic supplement.

Diet:Fasting-mimicking diet once every four to six months;roughly 16:8 intermittent fasting at other times.

Mindfulness practice:Daily meditation.

Anti-aging Rx:I love cooking and eating, so I do not restrict foodon the weekend. Happiness with friends and family is thesurest path to longevity.

100th-birthday wish:A bike tour across the US, from coast to coast.

Link:
Why Silicon Valley Execs Are Investing Billions to Stay Young - Robb Report

Recommendation and review posted by Bethany Smith

Google had a grand vision for Chrome, one that included treating web apps as first-class citizens, a vision that almost saw its fulfillment in Chrome OS. Chrome Apps, however, didnt exactly fly on their own and was eventually made redundant by the more browser-agnostic and standardized Progressive Web Apps or PWAs. That said, Chrome Apps did gather a few true believers and users, especially among its enterprise customers, causing Google to now push back its earlier plans to sunset Chrome Apps until June next year.

The idea of Chrome Apps was novel and exciting back in a time when web apps were still in constant flux. These days, however, they have become a platform to reckon with and, perhaps to Googles secret disappointment, grown to eclipse Chrome. PWA is the name of the game these days and Google has decided to focus its resources on those instead at the expense of obsoleting its own Chrome Apps platform.

Easier said than done since Google pushed Chrome Apps hard on users, developers, and especially enterprise customers. It earlier decided to end support for Chrome Apps on Windows, Mac, and Linux by December this year for both regular users and enterprise customers alike. That may not have sat well with the latter and Google is now giving Chrome Apps a life extension.

It has already stopped accepting new Chrome Apps into its Chrome store last March but Chrome Apps for regular users on Windows, Mac, and Linux will no longer be supported on June 2021 instead of June 2020 as earlier planned. Enterprise customers as well as regular users on Chrome OS will still see support for Chrome Apps until June 2022.

What hasnt changed, is that latter date, when Chrome Apps will completely become unsupported on its last bastion, Chrome OS, thereby ending support on all platforms, consumer and enterprise. Chrome Apps may still work beyond that point but they will no longer receive any updates. Google also reassures Chrome users that extensions will continue to be a thing as a completely different and separate platform exclusive to the web browser.

Original post:
Chrome Apps will continue to live on until next year - SlashGear

Recommendation and review posted by Bethany Smith

By: Editorial | Updated: August 11, 2020 4:22:22 amIn the US postal system, there is no room for Putin or Guccifer 2.0, and no possibility of a man in the middle attack.

After having issued ordinances removing stockholding restrictions on major foodstuffs and dismantling the monopoly of regulated mandis in the trading of farm produce, the Narendra Modi government has launched a new Agriculture Infrastructure Fund. A financing facility for setting up warehousing, cold chain, processing and other post-harvest management infrastructure, it provides an interest subvention of 3 per cent on loans of up to Rs 2 crore for a maximum seven-year period. The borrowers are mainly to be farmer producer organisations and primary agricultural cooperative societies, with a targeted disbursement of Rs 1 lakh crore over the current and next three fiscals. In order to make it attractive for banks, the loans would also have government-backed credit coverage against defaults. All in all, a good scheme at least on paper. No one can doubt the need for investments in produce shelf life extension and value addition. Also, there can be nothing better than this infrastructure coming up closer to farms and established farmer-owned institutions, thereby complementing the recent reforms that essentially aim at improving producers realisations and their share in the consumers rupee.

But theres a need to temper expectations. To start with, organisations such as the National Horticultural Board are already providing credit-linked subsidy on capital investments in pre-cooling units, controlled/modified atmosphere cold stores, reefer vans, ripening/curing chambers and other such post-harvest infrastructure. There is no dearth today of cold stores in potatoes, just as a lot of storage capacity, including low-cost scientifically-built on-farm structures, has been created for onions under the Rashtriya Krishi Vikas Yojana. So why one more scheme, is a natural question to ask. If at all, it would make sense to merge all existing schemes with the new fund so as to better leverage government money.

Secondly, cold chains and agro-processing cannot be a panacea. More than three-fourths of Indias sugarcane crop is processed by mills. Organised dairies, likewise, handle nearly a quarter of the officially-estimated milk production. Many have even installed bulk coolers allowing milk to be chilled at source in the village collection centres itself. But all that hasnt solved the problem of cane payment arrears or stopped the current crash in milk procurement prices. The same goes for onions and potatoes. Being able to store certainly enables farmers to harvest their crop, say, in March and make staggered sales till November to take advantage of higher off-season rates. But again, it has not ended price volatility that ultimately benefits neither producers nor consumers. The focus of policymakers during the first 40 years after Independence was raising farm production. In the subsequent two decades, they started paying more attention to agro-processing. The next revolution, especially in todays age of surplus, should be in crop planning and information dissemination to help farmers better align their production decisions what to grow and how much to market demand.

The Indian Express is now on Telegram. Click here to join our channel (@indianexpress) and stay updated with the latest headlines

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After the harvest - The Indian Express

Recommendation and review posted by Bethany Smith

By Johannes Stern 10 August 2020

In response to the dangerous further spreading of the COVID-19 pandemic in Germany and Europe, the ruling class is not reacting with an offensive to protect public health and to eliminate the deficiencies in the health system but is mobilizing further billions for rearmament and war.

On Thursday, the Defence Ministry informed members of parliament about the so-called 25 million euro proposals, which are to be handed over to the Budget Committee by the end of the year. All armament projects with an estimated cost of more than 25 million must be discussed and implemented by this committee.

The military blog Augen geradeaus! (Eyes Front!), which has close links to the Defence Ministry, has published an initial list of the 29 (!) such planned proposals. Among them are:

* A successor to the G36 assault rifle, the Bundeswehrs (armed forces) previous standard weapon. The first proposal for a new system, consisting of a basic weapon and accessories, is to be presented to the Budget Committee at the end of October.

* In addition to the 138 new fighter jets already launched in April, 38 Eurofighters (the latest version, Tranche 4) are to be procured. The corresponding proposal is also to be adopted in the last week of October.

* Also, there are numerous naval upgrade projects, including 31 Sea Tiger naval helicopters and the development and procurement of a so-called naval drone. The purchases are part of a comprehensive upgrade of the German navy. Among other things, four multi-purpose combat ships MKS180 are to be built in the next few years, at a cost of around 6 billion. These will be joined by further F125 frigates and Class 212A submarines.

* The tank units are also to be further upgraded. The old Marder infantry fighting vehicle is to get a service life extension for its thermal imaging targeting system, and the Leopard 2 main battle tank will receive a distance-activated protection system, reports Augen geradeaus! Also, a successor model to the Badger armoured engineering vehicle is planned, and the Boxer armoured transport vehicle will be built as a new model for joint fire support teams.

* It is also planned to increase ammunition stocks. Several proposals will address this. In the first week of September, the Budget Committee will discuss the supplementary procurement of the RBS15 Mk3 sea/land target drone for the first and second batch of corvettes, in mid-September the procurement of new GBU-54 guided bombs for the Eurofighter, and in October and November new ammunition for the 125 frigates, torpedoes and new tank ammunition.

* Significantly, the Special Forces Command (KSK), which is riddled with right-wing extremist terrorist structures, is also to be upgraded and will receive, among other things, new reconnaissance and combat vehicles and medium-sized tactical support vehicles to replace the Serval. From the outset, the WSWS has made clear that the announced restructuring of the KSK was primarily intended to make the elite right-wing extremist force more effective.

The billion-euro armament projects are aimed at expanding Germanys ability to make war. At the beginning of the week, the frigate Hamburg set sail with 250 soldiers to intervene in the escalating proxy war of the regional and great powers in Libya. In spring, the grand coalition had expanded and extended numerous foreign deployments of the Bundeswehr.

A few days ago, in an interview with Die Zeit, Defence Minister Annegret Kramp-Karrenbauer (Christian Democratic Union, CDU) demanded that it was high time to aggressively discuss how Germany must position itself in the world in the future. She said that Germany was expected to show leadership, not only as an economic power. It is about collective defence, it is about international missions, it is about a strategic view of the world, and ultimately it is about the question of whether we want to actively shape the global order.

To enforce the geostrategic and economic goals of German imperialism internationally, the German bourgeoisie is not only rearming its own military but also its allies within the European Union. It is becoming increasingly clear what militarist and fascist traditions it is resuming 75 years after the defeat of Nazi Germany in World War II.

According to an official report from the Defence Ministry, the handover of the first of a total of 44 German Leopard 2 battle tanks to the Hungarian army began at the end of July. The handover ceremony took place in the garrison town of Tata in the presence of Parliamentary State Secretary Thomas Silberhorn (Christian Social Union, CSU), who praised the military cooperation between Germany and the EU with the extreme right-wing Orban regime.

Hungary is modernising its land forces and Germany is a strategic partner in this process, said Silberhorn. The use of the same weapon systems and close cooperation in the training of tank crews increased the interoperability of the armed forces and was an important component of the Common Security and Defence Policy, he said. The German government would continue to be committed to close military cooperation between the two countries and thus also to strengthen cohesion in Europe based on the values and interests of the two countries.

The Defence Ministrys report does not go into more detail regarding the values and interests underlying the military cooperation between Berlin and Budapest. But the fascist character of the handover ceremony in Tata was obvious. Official participants in the event included, among others, members of the notorious neo-Nazi rock band Krptia, who had even written their own song for the tank handovercommissioned by the Hungarian army.

An entry on Krptias Facebook page says that the band was asked to write a march by tank crews in Tata in March. The timing for the song could not have been better, as the first Leopard 2A4 tanks have now been delivered, followed by 40 more Leopard 2A7 tanks in the next few years. One was lucky enough to admire these big cats, listen to them rumble, to see them get down to it... It is no big secret that the band has always been pro-military and satisfied with the development of the armed forces.

On Facebook, the band has published numerous pictures showing members of Krptia in martial gear posing in front of German battle tanks. Their posts clearly show their ideological outlook. They glorify Miklos Horthy, the former Reich administrator, anti-Semite and Hitler ally, drum up support for a new and ethnically pure Greater Hungary. The lyrics of their songs drip with fascist and militarist ideology. According to media reports, the Hitler salute can be regularly seen at Krptia concerts, and singer Jnos Petras rants against Roma and Jews.

Following the ceremony, the Hungarian government, which awarded Petras the countrys Golden Cross of Merit as early as 2013 and itself rehabilitated Horthy and Hungarian fascism, has defended its cooperation with Krptia. In response to an inquiry by Der Spiegel, the defence ministry in Budapest declared that the tank march was about love of the homeland and respect for the soldiers. We are pleased that a work of art has been created that popularises military service and the military vocation as widely as possible.

This is also the attitude of the German government. According to Der Spiegel, the Defence Ministry has stated that it does not want to take a position on the internal affairs of the Hungarian armed forces. It is becoming increasingly clear that the extreme right-wing terrorist networks in the Bundeswehr, the police and the secret services exist and can operate largely unchecked, mainly because these fascist forces enjoy the official support of the capitalist state and its political representatives.

The author also recommends:

German defence minister propagates rearmament, militarism and war [23 July 2020]

Fascist death squads preparing to carry out mass murder in Germany [7 August 2020]

Read the rest here:
German government continues to boost military spending and cover for fascists - WSWS

Recommendation and review posted by Bethany Smith

WASHINGTON On June 29, a solicitation titled, NATO International Competitive Bidding (ICB): Alliance Future Surveillance and Control (AFSC) Project-Risk Reduction and Feasibility Study, popped up on Beta.Sam.Gov, a U.S. government contracting site.

The appearance of the notice represented an early, but important, step in a long process of finding a replacement for NATOs fleet of airborne early warning and control AWACS planes, which have seen increased usage over the past five years.

What youve spotted online is the U.S. government preparing U.S. companies for this upcoming call for bids, a NATO official, speaking on background, explained to Defense News. Allies will then need to decide what form [the new design] should take.

Currently, 18 nations participate in NATOs early-warning-and-control force, which operates 14 E-3As: Belgium, Canada, the Czech Republic, Denmark, Germany, Greece, Hungary, Italy, Luxembourg, the Netherlands, Norway, Poland, Portugal, Romania, Spain, Turkey, the United Kingdom and the United States. The planes are based at NATO Air Base Geilenkirchen, Germany.

NATO plans to spend $1 billion for a final service life extension of the aircraft, which would keep it flying until 2035. Any delays in the decision-making process will likely increase the cost for the fleet, meaning there is heavy pressure to hit key milestones for an alliance that rarely buys military gear as collective.

As of July, six consortia from across the alliance have delivered concept studies to NATO leadership; Brussels is currently assessing those concepts with the goal of defining a more narrow scope for requirements before the end of 2020, per the NATO official. That will be followed in 2021 by another round of responses from industry, and a 2023 deep dive by NATO which is likely to set up the final requirements. Overall, the development stage through 2023 has a budget of EUR 118.2 million ($139 million).

In the U.S., expect Boeing and Northrop Grumman to be in the running, while the likely European contenders would be Saab and Airbus, according to Doug Barrie, senior military air analyst at the International Institute for Strategic Studies think tank in London. With all the usual caveats, the most likely outcome is that it is U.S., perhaps with some European add-ons, Barrie predicts.

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Richard Aboulafia, an analyst with the Teal Group, agrees that a U.S. prime, and lots of European mandates for local sustainment, support, and upgrade work is a likely outcome.

The European industrial role is a bit complicated by the fact that Airbus has zero experience here, Aboulafia argued. Saab certainly can do the job, but GlobalEye simply doesnt have the capabilities of a higher-end system, which means Boeing, or, just conceivably, Northrop Grumman/Lockheed Martin.

Firms that end up as second-tier suppliers may still end up with a strong work share, depending on how the project shapes up. The official NATO line on the program follows the system of systems approach currently popular inside the U.S. Air Force, with the idea that a single platform may not be the optimal solution.

The replacement for the AWACS aircraft could include different combinations of systems in the air, on land, at sea, in space and in cyberspace, the NATO official said. The aim is for the solution to be ready by 2035, when the AWACS aircraft reach the end of their service life.

Barrie sees costs and benefits to either approach, noting that a distributed system is less vulnerable overall to kinetic attack but is heavily reliant on connectivity, while a traditional setup is more vulnerable to physical attack, but if there is onboard command and control less reliant overall on wider connectivity and off-board analysis.

Adds Aboulafia, That system-of-systems approach is a good talking point, but creating the broader architecture is quite complicated. Also, creating a system is kind of a given for airborne early warning, but there needs to be a central platform doing the bulk of the heavy lifting. Thus, the teams will need to revolve around a platform prime.

While the overall price of the program will depend on the final design, Aboulafia predicts everything put together could cost in the $10 billion range to buy an equivalent of the original 17-aircraft NATO purchase. And that money may well be worth it for the alliance, according to Barrie.

Its been a practical and a symbolic asset, he said, and in the current European security environment air surveillance and C2 isnt becoming any less important.

Valerie Insinna in Washington contributed to this report.

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The race is on to replace NATO's early-warning aircraft fleet - DefenseNews.com

Recommendation and review posted by Bethany Smith

In Minnesota, reducing the effects of oxidation is a continuous challenge.

In a new study, funded by the Local Road Research Board, researchers will compare the effectiveness of two different methods: applying a surface treatment (typically a fog or chip seal) and increasing the asphalt film thickness during original construction.

The purpose of this study is to provide recommendations for keeping the pavement structure as moisture resistant as possible for the least amount of cost.

Required minimum asphalt film thickness (AFT) is an important parameter when assessing the long-term durability of an asphalt mixture. Research shows higher film thicknesses create more durable mixtures. Minnesota specifications require a minimum AFT for mixture design acceptance. If the ATF is below acceptable limits during production, large payment reductions or orders to remove and replace may result.

Pavement preservation treatments are gaining momentum as cost-effective ways of enhancing pavement life. To prevent deterioration of pavements, chip seals are a proven preservation method and have been widely used in Minnesota.

A Minnesota study estimated that a chip seal placed at the time of construction will be cost-effective if pavement life is extended by approximately 0.45 years (Wilde et al. 2014). Typical life extension for chip sealed roads range 5-7 years. The value of AFT specifications has been debated within the paving community.

This project offers an opportunity to validate current specifications, investigate the role of chip seals in pavement durability, and use lab and field data to perform a cost-benefit analysis of increased AFT and chip seals placed at 1 year.

Data for the analysis will be collected from both laboratory performance testing and field performance. Pavement projects especially of interest are projects that incurred pay deductions due to low AFT and if/when chip seals were placed to preserve those roadways.

Details of the research study work plan and timeline are subject to change.

To receive email updates about this project, visit MnDOTs Office of Research & Innovation to subscribe.

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New Project: Benefit/Cost of Applying a Higher Asphalt Film Thickness vs. Doing a Chip Seal at One Year - Crossroads - A Minnesota Transportation...

Recommendation and review posted by Bethany Smith

07 August 2020

The restarts of three advanced gas-cooled reactor (AGR) units at EDF's Dungeness B and Hunterston B nuclear power plants in the UK have been postponed.

Dungeness Reactor 22 and Reactor 21 were taken offline, respectively, on 27 August and 19 September, 2018 - for pre-planned outages. During that work, EDF uncovered issues to do with the main steam line and then corrosion. In recent months attention has been focused on issues to do with the site's boilers.

Restart dates were 11 Septemberfor Reactor 22 and 21 Septemberfor Reactor 21, but these were re-forecast yesterday as 10 December for Reactor 22 and 20 Decemberfor Reactor 21.

Located in Kent, England, Dungeness B's two 520 MWe AGR reactors started up in 1983 and 1985, respectively. In 2015, EDFannounced a ten-year life extension for Dungeness B to 2028 as part of the company'sstrategy to keep its UK nuclear fleet in operation until at least 2023 - the yearthat its Hinkley Point C nuclear power plant - under construction in Somerset, England - was due to be commissioned. That commissioning date has since been moved to 2025 for the first of the plants two UK European pressurised water reactors.

John Benn, station director at Dungeness, said: "The team at Dungeness continues to work tirelessly to prepare the site for a return to operations. The issues identified early in this outage have been addressed, as part of our significant investment programme over the last two years. As more work has been done, further issues have surfaced that require further detailed analysis and engineering work. To enable this work to be done, and to givethe Office for Nuclear Regulation [ONR] sufficient time to consider its response, the decision has been made to push back our restart dates until mid-December."

Hunterston Reactor 3 and Reactor 4 were taken offline on 9 March and 3 October, 2018 to work on issues related to their graphite cores. Following the approval of a safety case with the ONR, Reactor 4 was returned to service on 25 August, 2019 until 10 December, 2019.

Hunterston Reactor 3 had been due to restart on 20 August, but that has now changed to 30 August. The restart date for Reactor 4 remains 17 September.

An EDF spokesman said: "The ONR is continuing its assessment into the Hunterston B return to service case, using the information EDF has provided after extensive rounds of modelling and analysis. In the last few years, EDF has invested over GBP200 million in understanding the likely impacts on the graphite reactor under a range of worse case scenarios, including up to a 1 in 10,000 year seismic event, much larger than the UK has ever experienced. We remain confident that we would be able to shut down the reactor in all such scenarios."

Hunterston Reactor 3 (also known as B-7) and Hunterston Reactor 4 (B-8) were taken offline when cracks were found during routine inspections. EDF has said it still expects Hunterston, which is in North Ayrshire, Scotland, to close in 2023. The 475 MWe and 485 MWe reactors began operations in 1976 and 1977.

The reactor cores of all 14 AGRsin the UK are made up of graphite bricks. Channels run through these bricks for nuclear fuel, and also for control rods which can stop the nuclear reaction if needed. This graphite was always expected to change over time. How it ages is one factor which will determine how long Britain's AGRs will operate.

EDF Energy publishesupdates to its plant outage schedules here.

Researched and written by World Nuclear News

Follow this link:
EDF Energy prolongs outages at Dungeness and Hunterston units : Regulation & Safety - World Nuclear News

Recommendation and review posted by Bethany Smith

DUBLIN, Aug. 10, 2020 /PRNewswire/ -- The "Cell & Gene Therapy Market - Global Outlook and Forecast 2020-2025" report has been added to ResearchAndMarkets.com's offering.

In-depth Analysis and Data-driven Insights on the Impact of COVID-19 Included

The study considers the present scenario of the cell and gene therapy market and its market dynamics for the period 2019-2025. It covers a detailed overview of several market growth enablers, restraints, and trends. The report offers both the demand and supply aspects of the market. It profiles and examines leading companies and other prominent ones operating in the market.

Key Questions Answered

1. What is the cell and gene therapy market size and growth rate during the forecast period?2. What are the factors impacting the growth of the cell and gene therapy market share?3. How is the growth of the healthcare segment affecting the growth of the cell and gene therapy market?4. Who are the leading vendors in the cell and gene therapy market, and what are their market shares?5. Which product type/ end-user type/region is generating the largest revenue in the Asia-Pacific region?

The global cell and gene therapy market by revenue is expected to grow at a CAGR of over 30.9% during the period 2019-2025

The global cell and gene therapy market is one of the fastest-growing segments in the regenerative medicine market. The market is expected to grow at a faster pace during the forecast period. The demand can be attributed to the growing prevalence of several chronic diseases such as cancer, cartilage related problems, wounds, diabetic foot ulcer, genetic disorders, and other rare diseases across the globe.

The prevalence of cancer and diabetes is increasing in the global population, which is influencing the growth of the market. There is a large unmet need in the treatment available, which is filled by cell and gene therapies. The market is growing due to the increased availability of funding from various public and private institutions. Besides, there is increased support from regulatory bodies for product approval. Several governments are creating awareness of cell and gene therapies in the population.

Cell and Gene Therapy Market Segmentation

The global cell and gene therapy market research report includes a detailed segmentation by product, disease, end-user, and geography.

In 2019, the cell therapy segment accounted for a market share of over 53% in the global cell and gene therapy market. The segment is expected to grow at a steady rate during the forecast period due to the increase in the target population and the rise in the number of countries preferring cell therapies in their patients. Increased therapeutic benefits are attracting several countries to invest in this technology and conduct a high number of clinical trials. However, the lack of advanced infrastructure in developing countries is hindering the growth of the segment.

In 2019, the oncology segment accounted for a share of over 40% in the global cell and gene therapy market. Oncology has been one of the targets of intense research for the gene therapy procedures & approach. More than 60% of on-going gene therapy clinical trials are targeting cancer. The segment is expected to grow at a promising rate on account of the high prevalence of cancer diseases, especially in low and middle-come countries. The market is growing at a double-digit CAGR, which is expected to help the segment as many cell and gene therapy for cancer are commercially available.

The dermatology application segment in the cell and gene therapy includes wound care management among patients. Vendors are focusing on the development and commercialization of advanced wound care products for the treatment of chronic and acute wounds, thereby increasing the growth of the wound care market. The increased pervasiveness of diabetics is increasing acute and chronic wounds, including surgical wounds, pressure ulcers, diabetic foot ulcers, and other wounds.

In 2019, the oncology segment accounted for a share of over 40% in the global cell and gene therapy market. Oncology has been one of the targets of intense research for the gene therapy procedures & approach. More than 60% of on-going gene therapy clinical trials are targeting cancer. The segment is expected to grow at a promising rate on account of the high prevalence of cancer diseases, especially in low and middle-come countries. The market is growing at a double-digit CAGR, which is expected to help the segment as many cell and gene therapy for cancer are commercially available.

The dermatology application segment in the cell and gene therapy includes wound care management among patients. Vendors are focusing on the development and commercialization of advanced wound care products for the treatment of chronic and acute wounds, thereby increasing the growth of the wound care market. The increased pervasiveness of diabetics is increasing acute and chronic wounds, including surgical wounds, pressure ulcers, diabetic foot ulcers, and other wounds.

Segmentation by Product

Segmentation by Disease

Segmentation by End-user

Insights by Geography

In 2019, North America accounted for a share of over 60% of the global cell and gene therapy market. There are more than 530 regenerative medicine companies, including cell and gene therapy manufacturing developers. The number of products approved in North America grew significantly in 2019, with developers filed for marketing authorization for 10+ regenerative medicines, many of which we expect to be approved in 2020. Within the next 1-2 years, the number of approved gene therapies is expected to double.

The US and Canada are the major contributors to the cell and gene therapy market in North America. Regulatory bodies are supporting several investigational products, fast track approvals, RMAT designation for the faster approval of the product into the market. The alliance for regenerative medicine and Medicare and Medicaid is working together to bring the structured reimbursement channels for cell and gene therapies.

Segmentation by Geography

Insights by Vendors

The global cell and gene therapy market is highly dynamic and characterized by the presence of several global, regional, and local vendors offering a wide range of therapies. Dendreon, Gilead Sciences, Novartis, Organogenesis, Osiris Therapeutics, Vericel, Amgen, and Spark Therapeutics are the leading players in the market with significant shares.

Vendors such as NuVasive, APAC Biotech, Nipro, Orthocell, bluebird bio, J-TEC, and Terumo are the other prominent players in the market with a presence, especially in the cell therapy market. Most leading players are focusing on implementing strategies such as product launches and approvals, marketing and promotional activities, acquisitions, increased R&D investments, and strengthening their distribution networks to enhance their share and presence in the market.

Prominent Vendors

Other Prominent Vendors

Market Dynamics

Opportunities & Trends

Growth Enablers

Growth Restraints

For more information about this report visit https://www.researchandmarkets.com/r/is5t27

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

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900

U.S. Fax: 646-607-1904 Fax (outside U.S.): +353-1-481-1716

SOURCE Research and Markets

http://www.researchandmarkets.com

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Global Cell and Gene Therapy Market, Forecast to 2025 by Product, Disease, End-user and Region - COVID-19 Updated - PRNewswire

Recommendation and review posted by Bethany Smith

The leading UK institution University College London and the firm AlbionVC have made the first closing of a 112M (100M) fund with the aim of investing in UCL research and spinouts with a focus on gene and cell therapy.

This is the second so-called UCL Technology Fund and could double the size of the first, which raised around 56M (50M) in 2016. The fund is managed by AlbionVC in collaboration with UCLs commercialization company UCL Business (UCLB). Investors in the second fund include the firm British Patient Capital and UCL itself.

While the value of the first closing was not disclosed, Anne Lane, CEO of UCLB, told me that investments from this fund have already begun, and the second close is expected within the next 12 months. The larger size of the fund than the first could also give the team more flexibility on how many investments it makes and how big they are.

We have a focus in terms of UCLs cell & gene therapy research and that is reflected in the portfolio of the fund, but no specific disease areas as such, Lane said.

Examples of investments in the pipeline for this fund include a gene therapy for an undisclosed neurometabolic disorder, a gene therapy for epilepsy, and a cell therapy for glaucoma.

One of the biggest success stories from the first fund is the gene therapy company Orchard Therapeutics, whose Nasdaq IPO raised around 290M in 2018. Another UCL gene therapy spinout, Freeline Therapeutics, is also geared to launch a Nasdaq IPO in the coming weeks.

UCL has a strong entrepreneurial scene comparable to the biotech hubs in Oxford and Cambridge. For example, UCL spinouts reportedly raised 644M (579M) in external investment between 2018 and 2019, the largest amount of any university in the country.

The European startup scene has been shaken by the Covid-19 pandemic this year, with the eurozone economy going into a deep recession. Furthermore, gene therapy developers have experienced disruption with their programs due to clinical trial delays and changes in strategy.

Regarding the effect of Covid-19 on their fundraising, Lane said that the pandemic definitely made it more challenging and took more time than expected. But UCLs research continues and we look to its research base in overcoming the challenges posed by the global pandemic.

Image from Shutterstock

Read more:
112M Fund Launched to Commercialize UCLs Gene Therapy... - Labiotech.eu

Recommendation and review posted by Bethany Smith

PHILADELPHIA, Aug. 10, 2020 (GLOBE NEWSWIRE) -- Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced the appointment of Gallia Levy, M.D., Ph.D., as chief medical officer. Dr. Levy will be responsible for strategic and operational leadership across all functions in the product development lifecycle, including setting the global development strategy for current and future pipeline programs.

We are thrilled to welcome Dr. Gallia Levy to our growing gene therapy company striving to create a world where no life is limited by genetic disease, said Jeffrey D. Marrazzo, chief executive officer, Spark Therapeutics. Dr. Levys passion for hematology and gene therapy research is immediately evident and exactly the perspective needed to achieve our goal of unlocking the full potential of gene therapy. Especially during this pivotal time in hemophilia research, Dr. Levys deep understanding of rare blood disorders and the community will help accelerate our ability to deliver potentially transformative gene therapies for hemophilia, while progressing potential gene therapies for other genetic disease across our pipeline.

Dr. Levy joins Spark Therapeutics from Genentech, also a member of the Roche Group, where she served as the Vice President and Global Head of the Rare Blood Disorders franchise in Product Development. In this role, she was responsible for the clinical development of HEMLIBRA for hemophilia A as well as treatments for other rare blood disorders such as paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). She played a key role in the evolution of gene therapy as a new modality within the Roche Group.

Ive spent my career working to find new, innovative treatment approaches for patients affected by rare, life-altering disorders, and it is with great pride that I join the Spark team to help advance novel gene therapy programs and create next-generation solutions for patients, said Dr. Levy. Spark Therapeutics shares the same affinity for breaking barriers and putting the patient first, and I look forward to what we will achieve together.

Dr. Levy first joined Genentech in 2009, where she worked in both early and late-stage clinical development. She later moved to Portola Pharmaceuticals, where she led the clinical development program for hematology and oncology indications and returned to Genentech in 2014 to lead the hemophilia program.

Dr. Levy is board-certified in hematology and holds an M.D. and Ph.D. in Molecular and Cellular Biology from the University of Michigan. She completed her residency in internal medicine at Stanford University and a fellowship in hematology and oncology at the University of California, San Francisco. She also holds an M.S. in of Molecular and Cellular Biology from the University of Paris, VI and a B.A. from the University of California, Berkeley.

About Spark Therapeutics AtSpark Therapeutics, a fully integrated, commercial company committed to discovering, developing and delivering gene therapies, we challengethe inevitability of genetic diseases,includingblindness, hemophilia, lysosomal storage disorders and neurodegenerative diseases.We currently have four programs in clinical trials.At Spark, a member of the Roche Group, we see the path to a world where no life is limited by genetic disease. For more information, visit http://www.sparktx.com, and follow us on Twitter and LinkedIn.

Media Contact:Kevin Giordanokevin.giordano@sparktx.com(215) 294-9942

Read the rest here:
Spark Therapeutics Deepens Drug Development Expertise in Hematology and Rare Disease with Appointment of Gallia G. Levy, MD, Ph.D., as Chief Medical...

Recommendation and review posted by Bethany Smith

LEXINGTON, Mass., Aug. 10, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC) (LogicBio or the Company), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, today reported financial results for the quarter ended June 30, 2020, provided a business update and announced the U.S. Food and Drug Administration (FDA) has cleared the Companys Investigational New Drug (IND) application for LB-001 for the treatment of methylmalonic acidemia in pediatric patients. LogicBio released a separate press release this morning providing further details on the planned Phase 1/2 clinical design for LB-001.

We are thrilled to have received clearance to move forward with this first-in-human clinical trial with our lead product candidate, LB-001, for the treatment of methylmalonic acidemia, a life-threatening congenital genetic disease with no current therapeutic treatment options. This represents a significant milestone in our goal of bringing a treatment to MMA patients as well as for our GeneRide platform. We have maintained continuous dialogue with the centers of excellence that are planned to participate in the Phase 1/2 clinical trial, and we look forward to activating these sites as quickly as possible, said Fred Chereau, CEO of LogicBio. We have instituted systems attempting to mitigate COVID-19 dynamics on our study start-up process and, based on our best estimates, we plan to enroll our first patient in early 2021.

Commenting on the Next Generation Capsid Program, Mr. Chereau said, We are very excited about the recent advances in our novel capsid program, which has generated liver-tropic capsids intended for use in gene editing technologies such as GeneRide and other gene therapy approaches. We are focused on executing across all of our programs and look forward to sharing further details on our novel capsids in early 2021.

Appointment of Daniel Gruskin, M.D. to SVP, Head of Clinical Development

Daniel Gruskin, M.D. was appointed as SVP, head of clinical development in August 2020. Dr. Gruskin has served as interim head of clinical development of LogicBio since June 2020. In April 2020, Dr. Gruskin started consulting with the Company as a special advisor. Previously, Dr. Gruskin served in roles of increasing responsibility at Sanofi Genzyme, most recently as vice president, head of global medical affairs, rare disease, in which capacity he oversaw medical affairs, life cycle management, scientific affairs and other medical and development activities related to metabolic, rare and/or genetic diseases. Prior to his role at Sanofi Genzyme, Dr. Gruskin served as assistant professor, human genetics and pediatrics at Emory University School of Medicine, where he was also the chief of the genetics section at Childrens Healthcare of Atlanta.

Daniel has been instrumental in leading LB-001 clinical development efforts including getting the IND cleared. His deep experience in genetic medicines and metabolic diseases will serve LogicBio well as we look to execute on our goals for both the GeneRide and Next Generation Capsid platforms in search of transformative medicines, said Mr. Chereau.

Anticipated Milestones for 2020 and 2021:

Second Quarter 2020 Financial Results

Three Months Ended June 30, 2020 and 2019

About LogicBio Therapeutics

LogicBio Therapeuticsis dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms.

LogicBios proprietary genome editing technology platform, GeneRide, enables the site-specific integration of a therapeutic transgene without nucleases or exogenous promoters by harnessing the native process of homologous recombination. LogicBio has received FDA clearance for the first-in-human clinical trial of LB-001, a wholly owned genome editing program leveraging GeneRide for the treatment of methylmalonic acidemia. Patient enrollment is expected to begin in early 2021. In addition, LogicBio has a collaboration with Takeda to research and develop LB-301, an investigational therapy leveraging GeneRide for the treatment of the rare pediatric disease Crigler-Najjar syndrome.

LogicBio is also developing a Next Generation Capsid platform for use in gene editing and gene therapies. Data presented have shown that the capsids deliver highly efficient functional transduction of human hepatocytes with improved manufacturability with low levels of pre-existing neutralizing antibodies in human samples. Top-tier capsid candidates from this effort demonstrated significant improvements over benchmark AAVs currently in clinical development. LogicBio is developing these highly potent vectors for internal development candidates and potentially for business development collaborations.

LogicBio is headquartered inLexington, Mass. For more information, please visitwww.logicbio.com.

Forward Looking Statements

This press release contains forward-looking statements within the meaning of the federal securities laws, including those related to the Companys plans to initiate, advance and complete its planned SUNRISE Phase 1/2 clinical trial of LB-001 in MMA; the timing, progress and results of the Companys research and development activities, including those related to the GeneRide technology platform and Next Generation Capsid Program; its plans for LB-301 in Crigler-Najjar; and the sufficiency of its cash and cash equivalents to fund operating expenses and capital expenditure requirements. These are not statements of historical facts and are based on managements beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Companys plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Companys current and future research and development activities and preclinical studies and potential future clinical trials. In particular, the impact of the COVID-19 pandemic on the Companys ability to progress with its research, development, manufacturing and regulatory efforts, including the Companys plans to initiate, advance and complete its Phase 1/2 clinical trial for LB-001 in MMA, and the value of and market for the Companys common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration of the pandemic, travel restrictions, quarantines, social distancing and business closure requirements in the United States and in other countries, and the effectiveness of actions taken globally to contain and treat the disease. These risks are discussed in the Companys filings with the U.S. Securities and Exchange Commission (SEC), including, without limitation, the Companys Annual Report on Form 10-K filed on March 16, 2020 with the SEC, the Companys Quarterly Report on Form 10-Q filed on May 11, 2020, and the Companys subsequent Quarterly Reports on Form 10-Q and other filings with the SEC. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, even if new information becomes available in the future.

Contacts:

Investors:Brian LuqueAssociate Director, Investor Relationsbluque@logicbio.com951-206-1200

Media:Stephanie SimonTen Bridge CommunicationsStephanie@tenbridgecommunications.com617-581-9333

See the original post:
LogicBio Therapeutics Reports Second Quarter 2020 Financial Results and Provides Business UpdatesFDA Clears IND Application for LB-001 for the...

Recommendation and review posted by Bethany Smith

More than half the patients with high-grade bacillus Calmette-Gurin (BCG)-unresponsive nonmuscle invasive bladder cancer (NMIBC) treated with nadofaragene firadenovec, an investigational intravesical viral gene therapy, achieved a clinical response at 3 months in a phase3 trial.

The results "provide a significant efficacy benefit that, pending regulatory approval, might offer patients with a difficult-to-treat bladder cancer a bladder-sparing alternative," said Neal Shore, MD, medical director for the Carolina Urologic Research Center in Myrtle Beach, South Carolina.

There is clearly an unmet need for new bladder-sparing treatments in these patients, said Fred Witjes, MD, from the Radboud Institute for Molecular Life Science in Nijmegen, the Netherlands, who discussed trial findings during the virtual European Association of Urology 2020 Congress.

"The drugs that we have are old and there is a limited availability for both MMC [mitomycin-C] and BCG. We need some alternatives for initial adjuvant therapy," he explained. "The unmet need is, of course, especially there in BCG-unresponsive patients or BCG-unresponsive CIS [carcinoma insitu]."

"Clinically appropriate patients with BCG-unresponsive NMIBC are currently faced with radical cystectomy," Shore explained during his presentation at the congress.

Nadofaragene firadenovec is a viral-based gene therapy that consists of a replication-deficient adenovirus that delivers the gene for interferon alpha-2b (IFN2b). When administered with the polyamide compound Syn3, the viral vector can deliver the IFN2b gene to the epithelial lining of the bladder. The gene is subsequently incorporated into cellular DNA, meaning that large amounts of the IFN2b protein can be produced locally.

For their open-label, randomized trial, Shore and his colleagues looked at 157 patients with a mean age around70 years. All participants had carcinoma insitu or high-grade Ta (noninvasive) or T1 (invasive) papillary disease with or without carcinoma insitu, and all had been unresponsive to standard intravesical treatment with BCG in the previous 12 months.

Nadofaragene firadenovec was administered once every 3 months, for up to four doses in the first year. If patients showed no signs of high-grade disease recurrence at 12 months, they were offered continued treatment.

For patients with high-grade carcinoma insitu, the complete response rate was 53.4% at 3 months and 24.3% at 12 months. For patients with papillary tumors, the response rate was 73.0% at 3 months and 44% at 12 months.

The majority of study participants (72%) received two or three courses of BCG overall; that rate was 68.3% for those with carcinoma insitu and 80.0% for those with papillary disease. Just over half the patients with carcinoma insitu were refractory to BCG, as were 70% of those with papillary disease.

Almost one third of patients will not respond to BCG, and more than 50% will experience recurrence and progression during long-term follow-up, according to results from the phase2 study of nadofaragene firadenovec, which Shore was involved in.

In that trial of 40 patients, the response rate was 30% at 12 months for those with carcinoma insitu, and durable responses were seen out to 36 months. Investigators reported no dose-limiting toxicities or immune toxicity.

In the phase3 study, treatment-emergent adverse events were experienced by 93% of participants, but the vast majority were transient and grade1 or 2 events; approximately 17% were grade3. There was one grade4 event, but this was not related to the study treatment.

The most common treatment-emergent adverse events were instillation-site discharge, reported by 33.1% of the patients; fatigue, reported by 23.6%; bladder spasm, reported by 19.7%; micturition urgency, reported by 17.8%; and hematuria, reported by 16.6%.

"Follow-up and treatment of these patients is ongoing in an extension study," Shore said.

"We do really need something new in nonmuscle invasive bladder cancer," Witjes observed. "There has to be an alternative to cystectomy and, fortunately, news is coming."

"There is a highly unmet need, but we have to realize that there is a lot in the pipeline," he explained. "We have trials with immune checkpoint blockade, vaccines, genetic therapy, and drug-delivery systems."

Nadofaragene firadenovec creates "adaptive immunity that may be lifelong," Witjes reported. "Nadofaragene firadenovec has a good basis, has consistent, good results, and it has a good safety profile. In light of current developments, I think this certainly is an interesting option."

The study was sponsored by FKD Therapies Oy and conducted in collaboration with the Society of Urologic Oncology Clinical Trials Consortium. Shore reports receiving research and consulting fees from Amgen, Astellas, Bayer, BMS, Dendreon, Fergene, Ferring, Janssen, Merck, Myovant, Nymox, Pacific Edge, Nucleix, Pfizer, Sanofi-Genzyme, Sun Pharma, and Tolmar. Witjtes reports receiving advisory or lecturer fees from multiple companies, but none relevant to his comments.

European Association of Urology (EAU) 2020 Congress. Presented July17, 2020.

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Gene Therapy Promising in BCG-Unresponsive Bladder Cancer - Medscape

Recommendation and review posted by Bethany Smith

DUBLIN, Aug. 4, 2020 /PRNewswire/ -- The "Cell and Gene Therapy Tools, and Reagents: Global Markets" report has been added to ResearchAndMarkets.com's offering.

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

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

The Report Includes:

Key Topics Covered:

Chapter 1 Introduction

Chapter 2 Summary and Highlights

Chapter 3 Market and Technology Background

Chapter 4 Market Breakdown by Region

Chapter 5 Market Breakdown by End User

Chapter 6 Government Regulations

Chapter 7 Patent Review/New Developments

Chapter 8 Analysis of Market Opportunities

Chapter 9 Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/p5fqx6

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

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

Media Contact:

Research and Markets Laura Wood, Senior Manager [emailprotected]

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Evolution and Expansion of Therapies in the Global Cell and Gene Therapy Tools and Reagents Market 2020-2024 - PRNewswire

Recommendation and review posted by Bethany Smith

In the early days of COVID-19, the Alliance for Regenerative Medicine (ARM) was unsure how the pandemic and its accompanying economic downturn would affect the cell and gene therapy space.

It was a really specific time when the world and the markets were clearly reeling from the first appreciation for the seriousness of COVID-19, Janet Lambert, the organizations CEO said.

Now, the numbers are inand theyre better than ever. In the first half of 2020, the regenerative medicine sector raised $10.7 billion, more than the total capital raised in 2019 and a 120% jump over the first half of 2019, ARM found in a new report titled, Innovation in the Time of COVID-19. The proceeds were shared pretty evenly between cell therapy companies ($7.5 billion) and gene and gene-modified cell therapy companies ($7.9 billion), with companies focused on tissue engineering reeling in $84 million.

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RELATED: Biotech IPO bonanza: Legend's $350M offering as Repare, Forma get in on the action

That $10.7 billion was driven by a couple of outsize deals and includes $1.4 billion raised in five IPOs, $1.6 billion in follow-on offerings and $3 billion in venture capital. Chinese CAR-T player Legend Biotech led the pack with its mammoth $487 million Wall Street debut in June, but its peers netted considerable sums too. That same month, gene therapy companies Generation Bio and Akouos raised $230 million and $244 million, respectively. In February, another gene therapy outfit, Passage Bio, raised $284 million and gene-editing biotech Beam Therapeutics bagged $207 million.

On the venture side, Sana Biotechnology scored $700 millionalmost as much as the five next largest private rounds raised by Orca Bio Elevate Bio, Legend, Freeline Therapeutics and Poseida, the report found. Like Legend, Generation Bio and Akouos also completed sizable private rounds the same year they went public.

RELATED: 'The silver lining': Biotech IPOs in the time of coronavirus

All this enthusiasm for this sector right now is evidenced by these really astonishing financing numbers I think the drivers of that enthusiasm remain in place and make me optimistic for the second half of 2020, Lambert said. We continue to see really promising clinical results. We continue to see products making it to market. We continue to see patient, regulator and payer enthusiasm for these products.

Part of that enthusiasm stems from an appreciation for the biotech sector generally, Lambert said.

Attention is being paid to what the biopharma sector can do for us all as we try to weather and get out of the pandemic, she said, echoing the sentiments of venture capitalists whove managed to raise life sciences funds in spite of the pandemic.

The other side of the equation is the nature of biotechbecause the drug development cycle is long, biotech investors arent looking for quarter-to-quarter returns, but at milestone readouts that can come more than a year after IPO, Jordan Saxe, head of healthcare listings at Nasdaq, said in a previous interview.

Biotech is actually fairly well positioned to weather these kinds of events because youre not relying on day-to-day consumer spending. Youre relying on meaningful clinical catalysts at the end of the day to really generate value, and thats still going to be there in this environment, said Jason Pitts, Ph.D., a principal at Sofinnova, in ARMs report.

RELATED: Flagship raises $1.1B to create biotechs for post-pandemic world

All this gas in the tank isnt just bankrolling existing cell and gene therapies, but also driving company formation, Lambert said. For the first time, ARM counts more than 1,000 companies working in the sector, with more than 1,000 clinical trials going on worldwide. More than half of those studies are in phase 2, with just over a third in phase 1 and the remainder in phase 3.

Of those studies, 11 are testing regenerative medicine approaches against COVID-19, with several academic research centers and biopharma companies working on new treatments to treat the disease in the short and long term.

Most of them are using cell therapies to address ARDS, or acute respiratory distress syndrome, which is a consequence of COVID-19, Lambert said. Unlike other prospects in the pipeline, such as antibodies, which could potentially be used to prevent infection as well as treat it, regenerative treatments focus on repairing damage to the lungs or other organs that patients can suffer as part of COVID-19.

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In the face of COVID-19, cell and gene therapy space shows 'remarkable resilience:' report - FierceBiotech

Recommendation and review posted by Bethany Smith

-- Continued robust treatment response from both high and low doses --

-- Long-term durability beyond 15 months from single IVT injection with zero rescue injections in Cohort 1 --

-- Well tolerated across all Cohorts; encouraging early safety data from Cohort 4 --

-- OPTIC enrollment complete; planning to start pivotal trial in wet AMD mid-2021 --

-- Company to host conference call and webcast with Key Opinion Leader Dr. Arshad Khanani today at 1:30 pm PT / 4:30 pm ET --

REDWOOD CITY, Calif., Aug. 10, 2020 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced positive new interim data from Cohorts 1-4 of the OPTIC Phase 1 clinical trial of ADVM-022 intravitreal (IVT) injection gene therapy in patients requiring frequent anti-VEGF injections for their wet age-related macular degeneration (AMD). The company also reported recent business progress and financial results for the second quarter ended June 30, 2020.

For the first time, interim data from all four cohorts of the OPTIC trial (July 23, 2020 cutoff date), including preliminary safety data from Cohort 4, are presented. These data further demonstrate the transformative potential of ADVM-022 to greatly reduce the treatment burden for patients with wet AMD:

OPTIC Phase 1 Clinical Trial Data:

Before: 9.6After: 0

Before: 10.0After: 1.3

N/A

1 Best corrected visual acuity (BCVA)2 Central retinal thickness (CRT)3 All patients from Cohort 1 (n=6)4 All patients from Cohort 2 (n=6) and Cohort 3 (n=9)5 Annualized rate (Before) = (number of IVTs in 12 months prior to ADVM-022) / (days from the first IVT in the past 12 months to ADVM-022 / 365.25)Annualized rate (After) = (number of aflibercept IVTs since ADVM-022) / (days from ADVM-022 to the last study follow-up / 365.25)6 Fluorescein angiography of posterior pole

Arshad M. Khanani, M.D., M.A., managing partner and director of clinical research, Sierra Eye Associates, clinical associate professor of ophthalmology, University of Nevada, and top enrolling investigator in the OPTIC trial said, In my experience the sustained anatomical treatment response following a single intravitreal injection of ADVM-022 is unprecedented, which is remarkable considering that the patients enrolled in OPTIC were difficult-to-treat and had previously required frequent injections to maintain their vision. The safety profile to date shows that ADVM-022 is well tolerated, and that the prophylactic steroid eye drop regimen has been effective at limiting early ocular inflammation. I believe that ADVM-022 has the potential to be a transformational gene therapy improving real-world outcomes for patients living with wet AMD.

Aaron Osborne, MBBS, chief medical officer of Adverum, added, We have completed dosing patients in OPTIC, with 30 patients being followed up across four cohorts, and we look forward to presenting additional data from these cohorts by the end of this year. With the exciting data presented to date from OPTIC highlighting the potential of ADVM-022 to dramatically reduce treatment burden for patients with wet AMD, we plan to seek U.S. and international regulatory authorities input as we progress towards initiating a pivotal clinical trial in mid-2021.

Laurent Fischer, M.D., chief executive officer of Adverum stated, With these impressive OPTIC data, we are now poised to move our potentially transformative therapy into pivotal trials. ADVM-022, a novel gene therapy providing continuous delivery of aflibercept, has the potential to be a one and done IVT injection that would dramatically reduce the treatment burden for the millions of patients with wet AMD and DME worldwide. As we continue to see positive data from ongoing trials, we are preparing to accelerate our development and future commercial launch plans for ADVM-022. We continue to add industry-leading talent to our team and are beginning to expand our business operations and capabilities, including clinical, regulatory, manufacturing, and pre-commercial functions. Its an exciting time at Adverum, and Im humbled by the dedication of the retina specialists, their patients and our employees for their tireless efforts to help patients with severe ocular diseases during a pandemic.

Recent ProgressClinical DevelopmentINFINITY Phase 2 Clinical Trial of ADVM-022 in Diabetic Macular Edema

Future PlansADVM-022 in wet AMD

ADVM-022 in DME

Manufacturing

COVID-19To date, Adverum has experienced limited impact from COVID-19 on its operations and ongoing clinical programs, including the OPTIC and INFINITY clinical trials. The company is continuing to monitor and attempt to address or limit the potential impacts of COVID-19 on its employees and operations, patient safety, patient enrollment, continued participation of patients already enrolled in the companys clinical studies, protocol compliance, data quality, and overall study integrity.

Financial Results for the Three Months Ended June 30, 2020

Conference Call and Webcast InformationAdverum will host a conference call and webcast today with Key Opinion Leader Dr. Arshad Khanani at 1:30 pm PT / 4:30 pm ET to present new data from the OPTIC Phase 1 clinical trial of ADVM-022 in wet AMD and provide an update on recent business progress. The live webcast will be accessible under Events and Presentations in the Investors section of the company's website. To participate in the conference call dial 1-855-327-6837 (domestic) or 1-631-891-4304 (international) and refer to the Adverum Biotechnologies Conference Call. It is recommended call participants dial in 15 minutes in advance. The archived webcast and slide presentation will be available on the Adverum website following the call and will be available for 30 days.

About the OPTIC Phase 1 Trial of ADVM-022 in Wet AMDThis multi-center, open-label, Phase 1, dose-ranging trial is designed to assess the safety and tolerability of a single intravitreal (IVT) administration of ADVM-022 in patients with wet AMD who are responsive to anti-vascular endothelial growth factor (VEGF) treatment. Patients received a high dose (6 x 10^11 vg/eye) of ADVM-022 in Cohort 1 (n=6) and Cohort 4 (n=9) and patients received a low dose (2 x 10^11 vg/eye) of ADVM-022 in Cohort 2 (n=6) and Cohort 3 (n=9). Patients in Cohorts 3 and 4 received six weeks of prophylactic steroid eye drops rather than 13 days of prophylactic oral steroids which were used in Cohorts 1 and 2. The primary endpoint of the trial is the safety and tolerability of ADVM-022 after a single IVT administration. Secondary endpoints include changes in best-corrected visual acuity (BCVA), measurement of central retinal thickness (CRT), as well as the need for anti-VEGF rescue injections. Each patient enrolled will be followed for a total of two years.

Eleven leading retinal centers across the United States are participating in the OPTIC Phase 1 trial for ADVM-022. For more information, please visit https://clinicaltrials.gov/ct2/show/NCT03748784.

About Adverum BiotechnologiesAdverum Biotechnologies (Nasdaq: ADVM) is a clinical-stage gene therapy company targeting unmet medical needs in serious ocular and rare diseases. Adverum is advancing the clinical development of its novel gene therapy candidate, ADVM-022, as a one-time, intravitreal injection for the treatment of patients with wet age-related macular degeneration and diabetic macular edema. For more information, please visit http://www.adverum.com.

Forward-looking Statements

Statements contained in this press release regarding events or results that may occur in the future are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to statements regarding: the potential for ADVM-022 in treating patients with wet AMD and DME; Adverums expectations that it will present additional data from all four cohorts of the OPTIC Phase 1 trial for ADVM-022 in wet AMD by the end of this year; Adverums expectations as to its plans to advance ADVM-022 in wet AMD by initiating a pivotal trial mid-2021 and in DME by continuing to enroll patients in the INFINITY trial, including without limitation its expected enrollment numbers for the INFINITY trial; Adverums expectations that it will present data from the INFINITY trial in the second half of 2021; Adverums expectations that it will accelerate its development, manufacturing, and commercial launch plans for ADVM-022; and Adverums expectations that its current cash position will fund its operations into 2022. All of these statements are based on certain assumptions made by Adverum on current conditions, expected future developments and other factors Adverum believes are appropriate in the circumstances. Adverum may not achieve any of these in a timely manner, or at all, or otherwise carry out the intentions or meet the expectations disclosed in its forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include risks inherent to, without limitation: Adverums novel technology, which makes it difficult to predict the time and cost of product candidate development and obtaining regulatory approval; the results of early clinical trials not always being predictive of future results; the potential for preliminary or interim results of clinical trials to change as the clinical trial continues or in connection with the preparation and analysis of final results; the potential for future complications or side effects in connection with use of ADVM-022; obtaining regulatory approval for gene therapy product candidates; enrolling patients in clinical trials; reliance on third parties for conducting the OPTIC and INFINITY trials and vector production; the effects of the COVID-19 pandemic on the companys operations and on the companys ongoing clinical trials; and ability to fund operations through completion of the OPTIC and INFINITY trials and thereafter. Risks and uncertainties facing Adverum are described more fully in Adverums Form 10-Q filed with the SEC on August 10, 2020 under the heading Risk Factors. All forward-looking statements contained in this press release speak only as of the date on which they were made. Adverum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Investor and Media Inquiries:Investors:Myesha LacyAdverum Biotechnologies, Inc.mlacy@adverum.com1-650-304-3892

Media:Cherilyn Cecchini, M.D.LifeSci Communicationsccecchini@lifescicomms.com1-646-876-5196

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Adverum Biotechnologies Announces Positive Interim Data from Cohorts 1-4 from OPTIC Phase 1 Trial of ADVM-022 Intravitreal Gene Therapy for wet AMD,...

Recommendation and review posted by Bethany Smith

MALVERN, Pa., Aug. 10, 2020 (GLOBE NEWSWIRE) -- Ocugen, Inc. (NASDAQ: OCGN), a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases, today announced the U.S. Food and Drug Administration (FDA) granted the fourth Orphan Drug Designation (ODD) for OCU400 in the treatment of PDE6B gene mutation-associated retinal diseases. Retinitis Pigmentosa (RP) caused by PDE6B mutation is an inherited retinal dystrophy that leads to blindness by midlife and is characterized by the progressive loss of photoreceptors, with or without the loss of retinal pigment epithelium cells. At least one mutation in the PDE6B gene has been found to cause autosomal dominant congenital stationary night blindness, which is characterized by the inability to see in low light.

Ocugens Modifier Gene Therapy Platform offers a unique approach in ophthalmology by addressing multiple diseases with a single product. A novel gene therapy product candidate, OCU400 has the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases. It consists of a functional copy of a nuclear hormone receptor (NHR) gene, NR2E3, delivered to target cells in the retina using an adeno-associated viral vector. As a potent modifier gene, expression of NR2E3 within the retina may help reset retinal homeostasis and potentially offer longer benefit, stabilizing cells and rescuing photoreceptor degeneration and vision loss. In pre-clinical studies, OCU400 has demonstrated improved vision signals in the retina where Electroretinogram response reveals rescue under both Scotopic (dim-lit) as well as Photopic (well-lit) conditions. The Company believes targeting multiple diseases with one product could also offer a smoother regulatory pathway and the ability to recover development costs over multiple therapeutic indications. Ocugen is planning to initiate two parallel Phase I/II clinical trials next year targeting two unique IRDs.

Following up on recent announcement of an ODD for RHO mutation-associated retinal degeneration and previous ODDs for both NR2E3 and CEP290 mutation-associated retinal degeneration, the ODD for PDE6B gene mutation-associated retinal degeneration continues to support Ocugens breakthrough modifier gene therapy platforms potential to treat multiple blindness diseases with a single product. RP is a group of heterogenic inherited retinal diseases associated with over 150 gene mutations, affecting over 1.5 million individuals worldwide. In addition, ~40% of RP patients cannot be genetically diagnosed, confounding the ability to develop personalized RP therapies. Traditional gene therapy or gene editing approaches may require more than 150 products to rescue these patients from vision loss. OCU400, a single product candidate, has potential to address broad-spectrum RP.

As principal investigator of numerous major clinical trials developing new medical and surgical treatments for retinal disorders, I have been on the cutting-edge of many new ophthalmology treatments. I am very encouraged by the potential for OCU400 given the uniqueness of Ocugens Modifier Gene Therapy Platform and the fact that FDA has issued four ODDs for this product. I look forward to Ocugen commencing clinical trials for OCU400 next year and the potential of helping patients by restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases including broad-spectrum RP, said Carl D. Regillo, M.D.,F.A.C.S., member of Ocugens Retina Scientific Advisory Board and Professor of Ophthalmology at the Sidney Kimmel Medical College at Thomas Jefferson University, Chief of the Retina Service at Wills Eye Hospital and founder and former director of the Wills Eye Clinical Retina Research Unit in Philadelphia.

I am thrilled to announce our fourth ODD for OCU400 from the FDA after announcing our third ODD for RHO mutation-associated retinal degeneration just a few days ago. With no approved treatments that slow or stop the progression of RP, we are dedicated to driving the development of our Modifier Gene Therapy Platform forward and potentially addressing the unmet need of multiple gene mutations, including mutations in the PDE6B gene with only one product, said Dr. Shankar Musunuri, Chairman, Chief Executive Officer and Co-Founder of Ocugen.

The FDA Office of Orphan Products Development grants orphan designation for novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. Orphan designation qualifies the sponsor of the drug for various development incentives of the Orphan Drug Act, including a seven-year period of U.S. marketing exclusivity, tax credits for clinical research costs, clinical research trial design assistance, the ability to apply for annual grant funding and waiver of Prescription Drug User Fee Act filing fees.

About OCU400OCU400 (AAV-hNR2E3) is a novel gene therapy product candidate with the potential to be broadly effective in restoring retinal integrity and function across a range of genetically diverse inherited retinal diseases. It consists of a functional copy of a nuclear hormone receptor gene, NR2E3, delivered to target cells in the retina using an adeno-associated viral vector. As a potent modifier gene, expression of NR2E3 within the retina may help reset retinal homeostasis, potentially stabilizing cells and rescuing photoreceptor degeneration and vision loss.

About Ocugen, Inc.Ocugen, Inc. is a biopharmaceutical company focused on discovering, developing, and commercializing transformative therapies to cure blindness diseases. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with one drug one to many and our novel biologic product candidate aims to offer better therapy to patients with underserved diseases such as wet age-related macular degeneration, diabetic macular edema and diabetic retinopathy. For more information, please visit https://ocugen.com/.

Cautionary Note on Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as predicts, believes, potential, proposed, continue, estimates, anticipates, expects, plans, intends, may, could, might, will, should or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (the SEC), including the risk factors described in the section entitled Risk Factors in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events or otherwise, after the date of this press release.

Corporate Contact:Ocugen, Inc.Sanjay SubramanianChief Financial OfficerIR@Ocugen.com

Media Contact:LaVoieHealthScienceEmmie Twomblyetwombly@lavoiehealthscience.com+1 857-389-6042

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Ocugen Receives Fourth FDA Orphan Drug Designation for the Same Product, OCU400 (AAV-NR2E3) Gene Therapy, for the Treatment of Another Key Inherited...

Recommendation and review posted by Bethany Smith

Through its commercialisation fund, Enterprise Ireland is investing in three research projects from Trinity College Dublin and RSCI.

Enterprise Ireland has awarded 1.3m to three third-level research projects through its commercialisation fund. Each of the projects will receive more than 400,000 in investment so researchers can transform their ideas into commercially viable and relevant businesses.

Enterprise Ireland has been working with third-level researchers for a decade through this initiative, with the aim of bringing commercially relevant technology out of the lab and into the marketplace.Previous awardees of the commercialisation fund include AudioSourceRE, Cala Medical and Senoptica Technologies.

The agencys senior commercialisation specialist, Eithne McShane, said: The commercialisation fund is an avenue for Irelands brightest scientists to commercialise their research and bring it to the market.

At Enterprise Ireland, we recognise that funding innovation is key to ensuring that the Irish economy remains competitive on the world stage through the creation of technology-based start-up companies and the transfer of innovations developed in higher education institutions and research-performing organisations to industry in Ireland.

In the latest iteration of the commercialisation fund, two research projects from Trinity College Dublin (TCD) and one from the Royal College of Surgeons in Ireland (RCSI) will receive investment. All three projects are led by women.

The medical research being undertaken by each of their teams will be looking at issues such as infectious diseases, adult blindness and drug delivery,McShane added.

We have seen in recent months the importance of medical research and look forward to assisting each on their journey to the marketplace with the ultimate goal of improving lives.

One of the two projects from TCD is a study focusing on the development of gene therapies for common retinal disorders, led by principal investigator Prof Jane Farrar.

Farrars team is researching age-related macular degeneration (AMD), which is the most common cause of blindness in the developed world. There are two distinct forms of AMD: dry and wet. While there are several treatments for wet AMD, there are no licensed medical therapies for dry AMD, which represents about 90pc of AMD cases and affects approximately 150m people globally.

The team has developed a novel gene therapy for dry AMD, based on a yeast-derived gene encoding mitochondrial complex in an adeno-associated virus vector. A commercial case feasibility study was conducted in 2019, with backing from Enterprise Ireland.

Among the outcomes targeted through the fresh funding from Enterprise Ireland are the development of in-vitro human retinal pigment epithelial cell models and the finalisation of in-vivo preclinical data packs for therapies with associated study reports, as well as the initiation of key commercialisation activities.

Led by TCDs Prof Aisling Dunne, Adjuvenate is developing a platform solution for improved subunit vaccines. The research is being conducted in response to the rise in infectious disease and the need to develop new vaccines that are capable of eliciting effective and sustained immune responses.

Adjuvenate focuses specifically on whooping cough, which is on the rise due to the inability of current vaccines to provide sustained immunity, according to the TCD researchers. They said that new, more effective adjuvants are needed, which would enable vaccine makers to produce a sustained, lasting immune response.

The team has discovered and patented a new whooping-cough vaccine component, which may have the potential to be a third-generation standalone booster vaccine for whooping cough.

The commercialisation fund will support the further development of this novel adjuvant to develop a new vaccine. In addition, the team will continue to develop the adjuvant molecule as a novel adjuvant for combination with other new vaccines in development.

The RCSI project receiving funding is StarMat Technologies, which focuses on star-shaped polypeptide materials for biomedical applications. Led by Prof Sally Ann Cryan with Prof Andreas Heise, the team is examining ways in which its patented StarMat technology can be tailored to deliver specific drug payloads.

The technology can be integrated with medical devices when required to target specific tissues and cells, and may be particularly well suited to applications in biotherapeutic delivery in respiratory and regenerative medicine.

The team conducted a commercial feasibility study, funded by Enterprise Ireland, through which the researchers worked with multiple industry partners. Their analysis highlighted the demand for direct-to-cell delivery technologies, particularly advanced therapeutics medicinal products. This includes nucleic acid-based therapies, such as RNA-based technologies, which are being used in some of the Covid-19 vaccine technologies currently in development.

The commercialisation funding will be used will support the development ofscalable processes for drug payload incorporation, product refinement and further toxicology studies.

Originally posted here:
Enterprise Ireland awards 1.3m to TCD and RCSI research projects - Siliconrepublic.com

Recommendation and review posted by Bethany Smith

Dallas-based Taysha Gene Therapies has raised $95 million in new funding for development of its treatments for rare childhood diseases.

The financial boost from investors such as Fidelity Management & Research, BlackRock and GV (formerly Google Ventures) marks the startups second big investment this year. In April, Taysha wrapped up a $30 million seed round that was the North Texas regions largest funding haul in the second quarter, despite funding for startups slowing amid the pandemic.

Tayshas work is focused on finding therapies for genetic diseases that can inhibit childrens ability to perform basic functions like walking, talking, eating and even breathing.

The biotech company will use the money to bolster its work on gene therapies for rare diseases affecting the central nervous system in partnership with UT Southwestern Medical Centers Gene Therapy Program. Itll also help build a scalable manufacturing facility.

This significant investment from premier, long-term investors will allow us to advance our mission of eradicating monogenic CNS disease for the thousands of patients who suffer from these devastating disorders, Taysha founder and CEO RA Session II said in a statement.

The company plans to file four investigational new drug applications by the end of 2021, according to Session. It will begin clinical studies later this year for treatment of GM2 Gangliosidosis, a group of rare diseases in children that progressively destroy nerve cells in the brain and spinal cord.

Taysha has a pipeline of 15 gene therapy drug candidates, with options on an additional four.

We believe this financing provides significant validation of our corporate strategy and will enable us to continue to rapidly translate programs from preclinical development into the clinic, Taysha board chairman Sean Nolan said in a statement.

Session formed Taysha with investors and executives from another well-known company, AveXis, which sold to Swiss pharmaceutical giant Novartis for $8.7 billion in 2018.

UT Southwestern has been on a roll in terms of commercializing its research. In May 2019, pharmaceutical giant Merck bought cancer drug spinoff Peloton Therapeutics in a $2.2 billion deal. Last June, a Boston-based pharmaceutical company acquired another spinoff, Exonics Therapeutics, in a deal that could be worth close to $1 billion.

More:
This North Texas biotech startup just raised a whopping $95 million amid the pandemic - The Dallas Morning News

Recommendation and review posted by Bethany Smith

Transparency Market Research (TMR) has published a new report titled, Advanced Wound Care Management Market - Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 20182026. According to the report, theglobal advanced wound care management marketwas valued at US$ 8.5 Bn in 2016 and is projected to expand at a CAGR of 5.8% from 2018 to 2026. Surge in incidence of chronic wounds and trauma cases is anticipated to boost the demand for advanced wound care management during the forecast period. North America and Europe are projected to dominate the global advanced wound care management market owing to higher rate of adoption and awareness regarding wound care management. Asia Pacific, Latin America, and Middle East & Africa are potential markets for advanced wound care management. The market in Asia Pacific is expected to expand at a CAGR of 6.3% from 2018 to 2026.

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Cost-effective advanced wound care management to treat complex wounds to drive global market

Increase in trauma and accident cases across the globe drives the advanced wound care management market. According to the World Health Organization (WHO), road accidents account for around 1.25 million deaths across the globe each year. Additionally, the number of patients with chronic wounds is rising rapidly across the world. This is likely to increase the number of surgical procedures; consequently, propelling the global advanced wound care management market.

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Chronic wounds to be highly lucrative segment

Chronic wounds is an emerging segment of the advanced wound care management market. In the chronic wounds segment, the ulcers sub-segment holds a prominent share. Venous leg ulcers account for 80% of all leg ulcers. Moreover, the chronic wounds segment is likely to hold major share due to high cost of therapy across the globe. As per a paper published by NCBI (National Center for Biotechnology Information), in May 2014, the annual burden of venous leg ulcers in the U.S. is nearly US$ 14.9 Bn, which includes indirect costs (productivity loss) as well. The diabetic foot ulcer sub-segment of the advanced wound care products market is expanding at a significant CAGR.

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Hospitals & clinics to be notably promising segment

In terms of end-user, the global advanced wound care management market has been segmented into hospitals & clinics, ambulatory surgical centers (ASCs), home health care, and others. The hospitals & clinics segment held major share of the global market in 2017. Expansion of the segment can be attributed to the availability of multiple service options and devices and tie-ups with health care companies in order to enhance health care product and service offerings. Moreover, hospitals are the preferred choice due to availability of advanced technology and better health care services. The home health care segment is expanding at a high growth rate, especially in developed economies, due to rise in geriatric population and increasing health care infrastructure and support. Moreover, rise in demand for advanced wound care management in home care settings for geriatric patients is projected to drive the segment.

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North America expected to dominate global market

In terms of region, the global advanced wound care management market has been segmented into five major regions: North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America dominated the global advanced wound care management market in 2017. The market in North America was valued at US$ 3.2 Bn in 2017 owing to a highly developed health care sector, increase in awareness among health care providers about advanced wound care management, and continuous evolution of wound care management. Advanced wound care management market offers significant growth potential in the region. The advanced wound care management market in Asia Pacific is anticipated to expand at a CAGR of 6.3% during the forecast period due to increase in awareness about advanced wound care products and expansion of the health care sector in countries such as China, Japan, and India. Moreover, technological advancements and increase in rate of adoption of advanced wound care management products are expected to propel the market in the region during the forecast period.

Smith & Nephew plc, Acelity L.P., Inc. and Mlnlycke Health Care AB anticipated to lead global market

The global advanced wound care management market is highly fragmented in terms of number of players providing different products. Key players in the global advanced wound care management market include Smith & Nephew plc, Acelity L.P., Inc., Mlnlycke Health Care AB, ConvaTec Inc., B. Braun Melsungen AG, Coloplast A/S, 3M Healthcare, Integra LifeSciences Corporation, Cardinal Health, PAUL HARTMANN AG, BSN Medical, Hollister Incorporated, Organogenesis Inc., and Medline Industries, Inc. Expansion of product portfolio through mergers and acquisitions is a key strategy followed by several global players. In December 2015, Acelity L.P., Inc. announced the acquisition of Spiracur, Inc., expanding its offering in disposable, portable, mechanical NPWT (Negative-pressure wound therapy) technology, and allowing sales and service channels to boost the expansion of the SNaP therapy system to patients and their care teams across the world who need access to NPWT devices.

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Recommendation and review posted by Bethany Smith

In the coming years, the cancer gene therapy market size is projected to witness considerable growth, particularly due to the surging prevalence of cancer, increasing investments in research & development (R&D) activities, improved regulatory framework, acceptance of gene therapy for the treatment of the disease, rising popularity of deoxyribo nucleic acid vaccines, and technological advancements. Clinical research laboratories, hospitals, biotechnology companies, and oncology institutes are the major end-users in the cancer gene therapy industry.

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The different therapies utilized for treatment are oncolytic virotherapy, gene transfer, and immunotherapy. Out of these, the gene transfer therapy is expected to lead the global cancer gene therapy market in the near future. There are several ways to apply gene therapy, for example, replacing absent or abnormal genes with healthy ones, changing the way genes are regulated, and introducing foreign genes in cells, which alter their function or/and survival. The gene transfer therapy further has several types, namely electroporation, magnetofection, naked/plasmid vectors, sonoportation, and gene gun.

Asia-Pacific (APAC) is projected to register the fastest growth in demand for cancer gene therapy in the coming years. The growth of the cancer gene therapy market in the region is ascribed to the improving healthcare infrastructure and increasing government initiatives. The healthcare infrastructure in the region is improving due to the rising disposable income of people in emerging economies, which, in turn, is resulting in the growing affordability of innovative treatment options. Moreover, countries including Korea and India have a large patient pool, which is further contributing to the growth of the industry.

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Some major players in the cancer gene therapy market are Cell Genesys Inc., GenVec Inc., Adaptimmune Therapeutics plc, Achieve Life Science Inc., Genelux Corporation, Advantagene Inc., and BioCanCell Ltd.

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Global Cancer Gene Therapy Industry Analysis, Share, Growth Drivers and Future Scope - Owned

Recommendation and review posted by Bethany Smith

How about a well-assessed report on the Gene Therapy market that provides insightful analysis of various trends/services/products which has the potential of bringing a paradigm shift in the growth rate? Fact.MR is the answer to all your questions based on the ongoing Developments in the Genetic Testing market! The report offers a comprehensive analysis of the most profitable opportunities across the various segments in the form of revenues and volumes during the forecast period. The report, with bulls eye analysis, has the potential of forming the crux of the success of your organization with a focus on various parameters such as drivers, restraints, challenges, opportunities, and competitive landscape assessment. Fact.MR projects the Gene Therapy market to expand at a CAGR of xx% during 2020-2026.

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Recommendation and review posted by Bethany Smith

Cranbury Aug 10, 2020 (Thomson StreetEvents) -- Edited Transcript of Amicus Therapeutics Inc earnings conference call or presentation Monday, August 10, 2020 at 12:30:00pm GMT

Amicus Therapeutics, Inc. - Director of IR

* Bradley L. Campbell

Amicus Therapeutics, Inc. - President, COO & Director

* Daphne E. Quimi

Amicus Therapeutics, Inc. - CFO

* Jeffrey P. Castelli

Amicus Therapeutics, Inc. - Chief Development Officer

* John F. Crowley

Amicus Therapeutics, Inc. - Chairman & CEO

* Debjit D. Chattopadhyay

H.C. Wainwright & Co, LLC, Research Division - MD of Equity Research & Senior Healthcare Analyst

Robert W. Baird & Co. Incorporated, Research Division - Senior Research Analyst

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics, Inc.'s Second Quarter 2020 Financial Results Conference Call and Webcast. (Operator Instructions) As a reminder, this conference call is being recorded.

I'd now like to turn the conference over to your host, Mr. Andrew Faughnan, Director of Investor Relations. You may begin.

Andrew Faughnan, Amicus Therapeutics, Inc. - Director of IR [2]

Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics' second quarter 2020 financial results and corporate highlights.

Speaking on today's call, we have John Crowley, Chairman and Chief Executive Officer; Bradley Campbell, President and Chief Operating Officer; Daphne Quimi, Chief Financial Officer; and Dr. Jeff Castelli, Chief Development Officer.

As referenced on Slide 2, we may make forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business as well as our plans and prospects. Our forward-looking statements should not be regarded as representation by us that any of our plans will be achieved. Any or all of the forward-looking statements made on this call may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. You are cautioned not to place undue reliance on any forward-looking statements, which speak only to the date hereof.

All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this presentation and conference call to reflect events or circumstances after the date hereof. For a full discussion of such forward-looking statements and the risks and uncertainties that may impact them, we refer you to the forward-looking statements and Risk Factors section of our annual report on Form 10-K for the year ended December 31, 2019, and the quarterly report on Form 10-Q for the quarter ended June 30, 2020, to be filed later today with the Securities and Exchange Commission.

At this time, it is my pleasure to turn the call over to John Crowley, Chairman and Chief Executive Officer. John?

John F. Crowley, Amicus Therapeutics, Inc. - Chairman & CEO [3]

--------------------------------------------------------------------------------

Great. Thank you, Andrew. Good morning, and welcome, everyone, to our second quarter 2020 results conference call.

As we did last quarter, let me start by stating that I hope everybody and their families are well and safe.

Our leadership team at Amicus continues to emphasize a range of programs and initiatives to protect and support our global workforce during this ongoing pandemic. While adapting to all of the changes brought about by the global pandemic, for Amicus, the first half of 2020 has been a period of excellent growth and execution across all aspects of our business, including science, clinical, regulatory, and as you see, our commercial effort, as we continue to build one of the next great global biotechnology companies poised to impact people around the world living with rare diseases.

As we did in this morning's press release, I'd like to highlight several key accomplishments. First, Galafold continues its strong launch performance and remains the cornerstone of our success. With $62.4 million in second quarter revenue, the Galafold launch continues to exceed expectations. Second quarter revenue represents the performance across the global business, including new patient starts from both switched and treatment-naive patients throughout the quarter in all major regions, including those hardest hit by COVID-19.

Second, our R&D time lines remain on track. We continue to expect the Phase III PROPEL study of AT-GAA in late-onset Pompe disease to read out in the first half of 2021. Additionally, the rolling BLA submission for AT-GAA remains on schedule, and we expect the first submission later this year. Within our gene therapy pipeline, we continue to move forward our lead Batten disease programs with CLN6 and CLN3 as well as our most advanced preclinical programs in gene therapy.

And third, following our strategic financing in July, Amicus' cash position is sufficient now to achieve profitability without the need for any future diluted financings.

Our continued revenue growth, prudent expense management and growth potential has allowed us to reach this important milestone of self-sustainability as we continue to realize our vision of delivering groundbreaking and potentially curative new medicines for people living with rare diseases around the world.

Turning now to Slide 4. We are well on track to achieve our 5 key strategic priorities for 2020, including, first, Galafold, our precision medicine for Fabry disease. We will continue to drive Galafold to more people living with Fabry disease with a minimal variance in existing and new markets around the world. We look to achieve global product revenue this year of $250 million to $260 million.

Second, we are increasing the clinical regulatory manufacturing and pre-commercial activities and planning surrounding our Pompe program for AT-GAA as we move this important therapy toward approval.

Third, again, we are advancing our industry-leading rare disease gene therapy portfolio. Standing from our new Global Research and Gene Therapy Center of Excellence in Philadelphia, we will be advancing the clinical development, manufacturing and regulatory discussions for both our CLN6 and CLN3 Batten programs.

Fourth, in addition, we are progressing our Pompe gene therapy towards IND, and we plan to disclose up to 2 additional IND candidates this year. A lot of work is underway with our manufacturing partners with the manufacture and scale-up of the Pompe gene therapy as well as our other potential IND candidates. So again, we look forward to sharing the additional IND candidates from our Penn collaboration later this year.

Just, again, we will continue to maintain a strong financial position as we carefully manage our expenses and our investments, and we remain fully funded now to profitability.

So with that, before I turn the call over to our team to provide more detail on the strong quarter, I just wanted to take a moment to acknowledge Dr. Ted Love's retirement from the Amicus Board of Directors. Ted, as many of you know, is a remarkable leader, a great friend of mine and a fierce advocate for innovation for patients. Now Ted and I will continue to work closely together as the Chair and Vice Chair of the Emerging Companies section, on the Bio Board, and I would very personally like to thank him for his nearly decade of service on our Board. He has helped to put Amicus in a much stronger position. So thank you, Ted.

So with that, let me turn the call now over to Bradley Campbell, our President and Chief Operating Officer, to further highlight the strong Galafold performance. Brad?

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Bradley L. Campbell, Amicus Therapeutics, Inc. - President, COO & Director [4]

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Great. Thanks, John. Good morning, everyone. As John mentioned, I'll now walk you through in more detail our Galafold performance for the quarter, and let me start on the continued growth of Galafold revenue in the second quarter of 2020 here on Slide 6. And this is where we give our global snapshot of the Galafold commercial progress.

For the second quarter, as John mentioned, total product revenue was $62.4 million, driven by strong patient demand, favorable reimbursement dynamics and business continuity even during the worst quarter of COVID so far. And importantly, our global compliance and adherence rates continue to exceed 90%.

The geographic breakdown of revenue during the quarter was $41.5 million or 67% of revenue generated outside the United States, and the remaining $20.8 million or 33% coming from within the United States. On a sequential basis, you'll note that ex-U. S., quarterly revenue did decline slightly from Q1 to Q2 due to the timing of orders. However, we continue to see strong growth in patients added during the second quarter, and that strength has continued now into the first month of Q3, so we expect to finish the year around 70-30 ex-U. S. to U.S. sales. And as a reminder, we have now over 4 countries around the world with regulatory approvals, and commercial sales now in over 30 of those countries. And this expanding global footprint is important not just to support the continued growth of patients with access to Galafold, but it really lays a strong foundation that is highly leverageable to support the potential launch of AT-GAA for Pompe and our future products as well.

One other interesting aspect of the expanding market access to Galafold, which is highlighted on this slide, and this is quite unique, is that we now have added over 1,000 additional amenable mutations to the European label. The previous 300 mutations were all associated with specific patients who have been identified with those mutations. These new mutations represent nearly all of the possible missense mutations in the GLA gene that are amenable to Galafold, as determined by our amenability assay. So if the physician now diagnoses a new patient with one of these mutations, they'll no longer have to wait for us to characterize the mutation and run it through the amenability assay, a process which previously could take several months. Now the physician can simply refer to the website and check the amenability of the mutation, and the patient can get immediate access to Galafold. Again, a really exciting application and a really unique application of the pharmacogenetic aspects of this medicine, and one that we're looking to apply to other geographies as well.

Turning now to Slide 7. We've had a very strong quarter even in the face of the COVID-19 pandemic. Our global supply chain remains fully intact. Our customers have great confidence that they can access Galafold, and our field team has been able to achieve a significant portion of their pre-COVID touch points through digital, telephonic and other means of interacting with their physicians. All of this has led to continued uptake with new patient starts all throughout the crisis in both switch and treatment-naive patients. Even in the hardest hit countries like the U.K., France, Italy, Spain, Japan, U.S. and many others, we are able to bring on patients to Galafold throughout the quarter.

And it's important to note that on a country-by-country basis, as local conditions allow, and in a very carefully prescribed manner, we're now allowing our field organization to go back out to visit their physicians and key accounts. We believe that these dynamics may continue to provide some tailwinds, even as the evolving global pandemic inevitably has caused some headwinds, which will continue to closely monitor as the full impact of the pandemic currently remains unknown.

From a numbers perspective, you can see that the second quarter sales increased 41% from second quarter 2019, which does include a 2% negative impact from foreign exchange. So from a true operational perspective, sales increased by 43% compared to last year.

On the left-hand side, we show our quarterly performance over the past several quarters. And as we've mentioned on previous calls, while we continue to expect strong growth quarter-to-quarter, due to a variety of factors, the rate of growth is typically nonlinear. Looking to the back half of the year, as we've seen in many years past, we expect a higher quarter-on-quarter growth in Q4 as compared to Q3, which means we expect a larger portion of the revenue in the second half of the year will fall into the fourth quarter. But overall, as we've just finished the 7th month of the year, here in July, we continue to be very confident in our guidance of $250 million to $260 million in full year global sales.

Now on Slide 8, with several years of performance behind us and now launched at most major markets around the world, we can confidently say we are on a path to that $500 million of sales opportunity in 2023.

As I've outlined previously, to get to that $500 million, we expect a 5-year average CAGR of about 40% from 2018 to 2023, and we expect to generate $1 billion in cumulative revenue between 2020 and 2022 alone, which goes a long way towards funding our R&D and OpEx over that period.

We also have even further confidence now in the $1 billion revenue opportunity at peak as we continue to see significant growth in the Fabry market globally, driven by continued diagnosis from high-risk screening, newborn screening and other diagnostic initiatives, in addition to penetration into the diagnosed untreated population.

As we have orphan exclusivity in the U.S. and Europe, in addition to our multiple orange book-listed patents that give us IP coverage into the late 2030s, so we have lots of opportunity to provide access to Galafold for a long period to come.

With that, let me hand the call now over to Dr. Jeff Castelli, our Chief Development Officer, who will further highlight our lead pipeline programs. Jeff?

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Jeffrey P. Castelli, Amicus Therapeutics, Inc. - Chief Development Officer [5]

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Yes. Thanks, Bradley, and good morning, everyone.

Moving on to our R&D updates on Slide 10. We wanted to remind everyone of our highly differentiated Pompe therapy, AT-GAA, and its mechanism of action. AT-GAA is our novel next-generation therapy consisting of ATB 200 or alglucosidase alfa, an investigational human recombinant GAA enzyme designed to target muscle cells throughout the body, administered via IV infusions, combined with AT2221, an orally administered enzyme stabilizer. AT2221 is administered shortly before the infusion of ATB200 begins and is intended to bind and stabilize ATB200 in circulation, allowing more active enzyme to be taken up into cells and deliver to lysosomes. The combination of ERT and enzyme stabilizer is one major distinction from the standard of care and other treatments in development for Pompe.

The other and, we believe, potentially more impactful distinction is the unique carbohydrate profile of the enzyme itself. Dr. Hung Do, our Chief Science Officer, and his team have been working over the past decade to develop a Pompe ERT, which improved binding the target receptors for efficient uptake in the cells, while importantly retaining the ability of the ERT to be processed by those cells after it's taken up to the mature, more active form of GAA.

With that in mind, and now turning to Slide 11, we would like to highlight a recent publication in the Molecular Therapy - Methods & Clinical Development journal, where Dr. Nina Raben's team at the National Institute of Health published findings from an independent preclinical study, building upon previous work with AT-GAA that showed additional improvements with longer-term treatment to knock out mice. The studies show that AT-GAA was successful in completely reversing excess glycogen simulation, restoring muscle strength and significantly improving a cascade of secondary abnormality. Including the elimination or reduction of autophagic buildup in muscle fibers, improvements in markers of lysosomal damage and in cell signaling. These are particularly exciting findings as these impacts have not been observed in previously disclosed studies.

Finally, on Slide 12, we want to remind everyone of the integrity of our Pompe Phase III PROPEL clinical trial. We reiterate that steady time lines remain on track, with data expected in the first half of '20 -- I think first half of 2021. To date, more than 97% of the 2,800 plus planned infusions and off-study assessments for the ongoing PROPEL study have been completed on schedule. Additionally, we continue to enroll patients in our pediatric studies and advanced manufacturing to support a 2021 BLA and MAA.

We want to express thanks to all Pompe clinical study participants and their families, to all our investigators and their staff and to our cross-functional Amicus Pompe team for their collective unwavering commitment and supportive efforts during this unprecedented time.

Moving on now to Slides 14 and 15. I'll briefly highlight here our industry-leading portfolio of gene therapies for rare diseases. During this time of COVID, we've been able to maintain our critical science and lead programs across the gene therapy portfolio, including CLN6 and CLN3 Batten disease as well as the Pompe and Fabry gene therapy programs with University of Pennsylvania.

Starting with our Batten disease franchise in CLN6, we previously reported positive interim data in our clinical study that demonstrates meaningful impact of our AAV gene therapy in this extremely devastating form of Batten disease. In October, at the virtual Child Neurology Society Annual Meeting, we're expecting additional data from the CLN6 Phase I/II study. We continue to advance regulatory discussions to finalize the clinical and regulatory path, and expect to provide an update in early 2021.

We believe this initial CLN6 data provide an important readthrough for our clinical study in CLN3 Batten disease, which is the most common form of childhood neuro generation. We plan to report initial data from the ongoing Phase I/II CLN3 study early next year based on scheduling changes for some of the major gene therapy conferences. Concurrent with the data, early next year, we will provide the expected regulatory path for the CLN3 program.

We continue to make great progress in all of our commercial manufacturing process for both CLN6 and CLN3, and we remain on track to begin dosing additional patients in both of these programs next year.

Moving on to Slide 16. I would like to remind everyone of the research collaboration with the University of Pennsylvania, which will be an important driver of growth for Amicus in the future. This collaboration with the Wilson Lab at Penn combines Amicus' protein engineering and glycobiology expertise with Penn's gene transfer technologies and expertise to deliver novel gene therapies that are designed for optimal cellular uptake, targeting, dosing, safety and manufacturability. As part of this collaboration, Amicus has rights to over 50 different diseases, in addition to the active ongoing preclinical programs.

Several Amicus presentations were given at the American Society of Gene & Cell Therapy Annual Meeting back in the second quarter. This data was highlighted by our Pompe gene therapy results, which showed the Amicus engineered GAA protein had improved targeting and clearance of glycogen storage in Pompe diseased minds. Additionally, preliminary data in nonhuman primates suggested therapeutically relevant expression levels in target organ, which is a key factor as we move to larger species.

We remain very encouraged by this data and continue to progress our Pompe gene therapy towards an IND. We also continue to make progress across our preclinical gene therapy programs and look to disclose up to 2 additional IND candidates later this year.

With that, I would like now to turn the call over to Daphne to review our financial results, guidance and outlook. Daphne?

--------------------------------------------------------------------------------

Daphne E. Quimi, Amicus Therapeutics, Inc. - CFO [6]

--------------------------------------------------------------------------------

Thank you, Jeff, and good morning, everyone.

Our financial overview begins on Slide 18 with our income statement for the quarter ended June 30, 2020.

For the second quarter, we achieved Galafold revenue of $62.4 million, which is a 41% increase over the second quarter of 2019. This includes year-over-year operational revenue growth measured at constant currency exchange rates of 43%, offset by a negative currency impact of 2%. Cost of goods sold as a percentage of net sales was 10.8% in the second quarter as compared to 12.1% for the prior year period. Cost of goods sold as a percent of revenue was favorable as Galafold revenue continues to grow in the United States, where we do not owe royalties; as well as in other countries, where we are subject to lower royalties.

Total operating expenses were $107 million in the second quarter of 2020, which decreased as compared to $115.2 million in the second quarter of 2019. On a non-GAAP basis, total operating expenses were $95.9 million in the second quarter of 2020 as compared to $103.6 million in the second quarter of 2019. The decrease in research and development costs reflects decreased travel and third-party costs, offset by continued investments to support the PROPEL study and in our gene therapy pipeline.

Our investment in research and development includes the impact of the implementation of the cost reduction measures that were previously announced, as does the decrease in selling, general and administrative expenses.

Due to the timing of expenses, the non-GAAP operating expense is expected to increase in the third quarter of 2020 as compared to the second quarter of 2020. We define non-GAAP operating expense as research and development and SG&A expenses, excluding share-based compensation, changes in fair value of contingent consideration and depreciation.

Net loss for the second quarter was $52.5 million or $0.20 per share as compared to net loss of $84.6 million or $0.36 per share for the prior year period. As of June 30, 2020, we had approximately 257 million shares outstanding.

Turning now to Slide 19. As John mentioned, following our $400 million senior secured term loan facility, we are now on a clear path to profitability without the need for any future dilutive financing. As we initially laid out last year at our Analyst Day, we have achieved this milestone by our continued revenue growth with Galafold as well as driving efficiencies, cost savings and careful expense management. Securing this financing with market setting turns gives us a strong financial platform to advance both patient and Amicus shareholder interest. Going forward, again, to emphasize, we expect total non-GAAP operating expenses in 2020 to remain relatively flat from 2019 as we, first, leverage the commercial infrastructure that is already in place for the AT-GAA launch and other products in our pipeline; secondly, we transitioned the costs associated with the development of AT-GAA to multiple gene therapy programs in our pipeline; and third, maintain financial discipline while meeting our objective.

To reiterate, all high priority research programs in gene therapy are moving ahead on schedule, especially in CLN3, CLN6, Pompe and Fabry, and we continue to fully support the work with Jim Wilson and Penn.

A few comments about our cash position and 2020 financial guidance. Cash, cash equivalents and marketable securities were $309.6 million at June 30, 2020 compared to $452.7 million at December 31, 2019. Following the receipt of net proceeds from the July debt facility and retiring the prior term loan of $150 million, Amicus has a cash position of over $530 million as of July 31. We are reaffirming our full year Galafold revenue guidance of $250 million to $260 million in addition to our non-GAAP operating expense guidance of $410 million to $420 million.

And with that, let me turn the call back to John for closing remarks.

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John F. Crowley, Amicus Therapeutics, Inc. - Chairman & CEO [7]

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Great. Thank you, Daphne, Jeff and Bradley.

So as you can see, we have been relentlessly focused on performance across the business despite all of the unprecedented change and challenges that have been brought about by the global pandemic. We have a great global team of passionate entrepreneurs at Amicus, who have led and will continue to lead us through this. I am confident that as the world emerges from this crisis, Amicus will emerge even stronger.

So with that, operator, we're happy to take any questions.

================================================================================

Questions and Answers

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Operator [1]

--------------------------------------------------------------------------------

(Operator Instructions) And your first question comes from the line of Anupam Rama from JPMorgan.

--------------------------------------------------------------------------------

Anupam Rama, JPMorgan Chase & Co, Research Division - VP and Analyst [2]

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Just a quick question on the PROPEL study. For those small number of patients, who did not get an on-time infusion -- schedule an infusion, do these patients ultimately get an infusion outside of the window? Or was it just a skip infusion, and then they got the next one?

Link:
Edited Transcript of FOLD earnings conference call or presentation 10-Aug-20 12:30pm GMT - Yahoo Finance

Recommendation and review posted by Bethany Smith

Selecta Biosciences Inc (NASDAQ:SELB) posts a loss of $24.1 million in Q2 2020. Its general and administrative expenses in Q2 2020 are $5.6 million, whereas R & D spending surged to $10.7 million.

President of Selecta, Carsten Brunn, said it is a transformative period for the company. It is strengthening its leadership position in immune tolerance. To unlock its ImmTOR immune tolerance platforms full potential, the company entered a pact with Sobi to get funding. The funding will optimize the safety and efficacy of biologics and enable the redosing of gene therapies. It will use funds for developing innovative immunotherapies for the treatment of autoimmune diseases.

Selecta is committed to developing SEL-212. As per the terms of the pact, Sobi is responsible for commercial, regulatory, and development activities of SEL-212. In alliance with Sobi, the company will initiate Phase 3 clinical trials in Q3 2020 and report topline data of Phase 2 COMPARE trial. Sobi will bear all the expenses for the Phase 3 clinical trial. It will provide $100 million as an initial payment to Selecta.

Selecta will also advance its gene therapy in MMA (methylmalonic academia) in H1 2021 to the clinic. It also prepares for IND submission for autoimmune disease treatment in 2021.

Selecta inked an RLOA (Research License and Option Agreement) with Sarepta to provide its ImmTOR for curing certain neuromuscular diseases. Sarepta will use ImmTOR to treat LGMDs (limb-girdle muscular dystrophies) and DMD (Duchenne muscular dystrophy).

Sarepta will engage in the investigational study of gene therapies and ImmTOR to reduce the creation of neutralizing antibodies. Under the terms of the agreement, Selecta already received initial payment and expects to get fees for certain pre-clinical milestones.

Selecta confirmed Peter G. Traber as a full-time Chief Medical Officer with effect from August 1, 2020, from the current position of interim CMO. Peter brings significant experience in academia, biotech, and pharma companies. He is responsible for overseeing clinical strategy and development, program management, medical affairs, and clinical and scientific advisory services for business development.

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Selecta Biosciences Inc (NASDAQ:SELB) Will Advance Gene Therapy Program In H1 2021 In MMA: Closes SLA For SEL-212 With Sobi - BP Journal

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OrbiMed, Novartis Venture Fund and RA Capital Management have joined forces to fund and help launch GentiBio.

The Boston, Massachusetts-based biotech starts life with a $20 million seed funding with an aim of creating new engineered regulatory T cells to deliver immune tolerizing meds for autoimmune, allergic and inflammatory diseases.

GentiBio lands with tech out of the Seattle Childrens Research Institute, Benaroya Research Institute and MIGAL Galilee Research Institute.

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The biotech was in fact co-founded by a team of scientists from these three institutes to restore immune tolerance using Treg cell therapy engineered with novel technologies that direct potent EngTregs to specific tissues damaged by abnormal immune responses, said the biotech in a statement.

The hope is that this tech will overcome many of the current limitations of Tregs therapeutics, including rarity and plasticity of endogenous Tregs populations.

Regulatory T cells, or Tregs, play a key role in modulating other cells in the immune system so they don't attack the bodys own tissues. The failure of Tregs can lead to autoimmune diseases.

In a June paper published in the Science Translational Medicine journal, the team from the Seattle Childrens Research Institute and the Benaroya Research Institute made Treg-like cells by gene editing ordinary T cells to express Foxp3 and paired them with antigen-specific T cell receptors. The engineered cells prevented harmful autoimmune response in Type 1 diabetes in mice.

GentiBio is focused on addressing the technical bottlenecks that have throttled Treg therapeutics, and we are thrilled to exclusively in-license a portfolio of unprecedented breadth from Seattle Childrens and Benaroya Research Institute in the U.S. and MIGAL Galilee Research Institute in Israel, said Adel Nada, M.D., co-founder and CEO of GentiBio.

The technologies licensed from these premier research institutions are mature and well-differentiated, and will be further optimized in sponsored research collaborations with the scientific teams that discovered them to advance novel and potent therapeutics with the potential to treat and cure serious autoimmune and inflammatory diseases.

Tregs are rare cells within the immune system and current therapies that source Tregs cells from the blood stream can be costly and cumbersome. In contrast, Seattle Childrens unique technology enables the generation of engineered regulatory T cells, or EngTregs, from the more abundant CD4+ cell population, addressing a critical manufacturing shortcoming for this novel treatment, said David Rawlings, M.D., director, center for immunity and immunotherapies, Seattle Childrens Research Institute and scientific co-founder of GentiBio.

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Novartis and a string of high profile backers fund next-gen Treg cell therapy startup GentiBio - FierceBiotech

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Global Trade Impact on the Hemophilia Gene Therapy Market due to COVID-19; Key Statistics, Trends Followed, and Other Industry Analysis

Analysis of the Global Hemophilia Gene Therapy Market

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According to the research analysts based on their thorough research suggest that the global Hemophilia Gene Therapy market is predicted to register a CAGR of around XX% between 2020 and 2026. The global demand for the Hemophilia Gene Therapy market was USD XX Million in 2019 and anticipates to reach USD XX Million by the end of 2026. The report analyzes all the micro and macro-economic factors that are projected to influence the growth of the Hemophilia Gene Therapy market in the coming decade.

The global Hemophilia Gene Therapy market is segregated into{Hemophilia A, Hemophilia B};{Hemophilia A Gene Therapy, Hemophilia B Gene Therapy}. For further understanding of the market some of the major segments are categorized into sub-segments. In the regional analysis also the five major regions are sub-categorized into countries. Some of major market players that are listed in the report areSpark Therapeutics, Ultragenyx, Shire PLC, Sangamo Therapeutics, Bioverativ, BioMarin, uniQure, Freeline Therapeutics.

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Global Hemophilia Gene Therapy Market 2020 Trends Analysis and Coronavirus (COVID-19) Effect Analysis | Key Players Market With COVID-19 Impact...

Recommendation and review posted by Bethany Smith