Archive for the ‘Gene Therapy Research’ Category

Cryopreservation has become an indispensable step in the daily routine of scientific research as well as in a number of medical applications, ranging from assisted reproduction and transplantations to cell-based therapies and biomarker identification. It is hardly possible to picture todays scientific and medical advancements without this technique.The successful development and implementation of all the therapeutic and scientific discoveries involving cryopreservation relies on the correct and safe translation of the method from the laboratory to the clinical and manufacturing scale.

With the need to correctly use this technique, more research is focusing on optimizing cryopreservation methods and investigating what the long-term effects and consequences are on the physiology of the cryopreserved material.

An important part of cell therapy research is focused on adult stem cells (ASCs). ASCs can be derived from different sources such as peripheral blood, bone marrow or adipose tissue and display strong promises because of their capacity to differentiate into any cell type of the human body.In recent work3, the team of Michael Pepper at the Institute for Cellular and Molecular Medicine in Pretoria, South Africa, explored the effects of cryopreservation on the differentiation ability of adipose tissue-derived stem cells (ADSCs). After analyzing gene expression of key adipogenic genes and the degree of differentiating cells, characterized with high levels of CD36 and intracellular lipid droplets, the scientists reported that slow freeze cryopreservation of cells shortly after their isolation causes no alterations on their ability to differentiate. Pepper is convinced of the necessity to perform such analysis when cryopreserving important cell pools: It is critical to do a post-thaw analysis of cell function to determine how the cryopreservation may have affected the cells.His team is analyzing the effects of cryopreservation on other cell types largely used in cell-based therapies such as hematological stem cells and peripheral blood mononuclear cells (PBMCs). Although they didnt observe major alterations in terms of immunophenotyping or the post-thaw proliferation of the cells, Pepper expresses his concern that more subtle characteristics might be affected.

Correct cryopreservation of cells intended for therapeutic use is crucial. This is very important particularly as cells may persist for a long time in the recipient. This area of cell therapy research definitely requires more attention, Pepper says. Moreover, his words reflect on the need to evaluate not only the direct post-thaw recovery, but to look deeper into the late-onset effects cryopreservation might have and ensure that transplanted cells have preserved their therapeutic properties.

In contrast to slow freezing, vitrification relies on the fast freezing of the material by putting it in high concentration of cryoprotectant and in contact with liquid nitrogen. This method allows the direct transition of water from liquid to solid state without crystal formation. The highly concentrated cryoprotectant prevents ice formation and therefore there is no need for slow cooling.

Although vitrification has a great potential, there are a couple of parameters that are a point of concern. The quick and drastic freeze is possible thanks to the high concentration of cryoprotectant, but the latter is also associated with higher toxicity. In some cases, an additional limitation is the direct contact of the sample with liquid nitrogen which is a predisposition for viral or bacterial contamination.The team of Christiani Amorim at the Institute for Experimental and Clinical Research in Louvain, Belgium, is approaching the challenges of vitrification in the context of ovarian auto-transplantation. Ovarian auto-transplantation consists of preserving a piece of ovarian tissue with active follicles from the pre-therapeutic ovary of a cancer patient, as chemotherapy often has damaging effects on the reproductive organs. This tissue sample will be conserved and auto-transplanted onto the patients ovary when she has recovered and wishes to become pregnant.In their recent research4, the authors used stepped vitrification, in which the concentration of the cryoprotectant is gradually increased while simultaneously temperature decreases. This avoids ice crystal formation and also prevents cryoprotectant toxicity.Although stepped vitrification has previously given good results in bovine ovarian tissue5, this was not the case for human ovarian tissue. The scientists didnt detect normal follicles following thawing and linked this to high cryoprotectant toxicity. Indeed, they observed all signs of dimethyl sulfoxide (DMSO)-related cell membrane damage: significant organelle damage, cell membrane disintegration and apoptosis. These observations imply on the variability of outcomes that the method could give when applied to the same type of tissue but from a different organism.Amorim is positive about the future of their method and recognizes the need for further research on the topic: I can see a great potential in the stepped vitrification approach, but I also believe that there is a lot we still need to learn before thinking about using it as method of choice for human ovarian tissue cryopreservation. The high cryoprotectant concentration that should be applied in this approach is my first concern. () Our study clearly showed that 50% DMSO is too high, so we need to try lower concentrations or combine it with other cryoprotectants.

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Freezing Life: The Current Trends in Cryopreservation - Technology Networks

COVIDS WAR ON WOMEN During this plague year, there is almost never good news, only degrees of bad news. Even so, the pandemic has been different (and worse) for girls and women.

Its true that more men are dying than women from Covid-19 around the world but thats not exactly cause for celebration.

Another ambivalent data point: More workplace risk is falling on women, who are more likely to be considered essential workers. The upside to that is still having a job, but at what price? Swedens Foreign Minister Ann Linde pointed out today in a POLITICO interview that 70 percent of those working in health care and elderly care are women.

More of the daily grind tends to fall, on average, on women: From the increased cleaning and chores that come with more time spent in the home, which falls disproportionately to so many female household members, to the extra education and childcare work created through closures of school and day care, where men have also been known, on average, to skimp.

The real-life examples are heartbreaking: Alice Jorge, a woman living with a disability in Belgium who needs support from her sister and a visiting nurse, was recently asked to choose between keeping her Covid-19 positive caregiver or going without professional care. Three women bound to suffer no matter what choices they took.

Domestic violence is up sharply: A new research report by a consortium that includes Johns Hopkins University confirms this: 31 million additional cases of gender-based violence can be expected globally if lockdowns last for an average of six months.

Travel to shelters may be restricted, and a simple phone call to a helpline can itself trigger new violence. Support services are overwhelmed with requests: from a 47 percent increase in calls to Spains national hotline to a 113 percent spike at U.N.-supported hotlines in Ukraine.

We can expect 7 million unplanned pregnancies in 144 low- and middle-income countries, thanks in part to restricted access to contraception, not to mention the 2 million female genital mutilations and countless child marriages projected to increase by the United Nations population agency. The pandemic is deepening inequality, UNFPA Executive Director Natalia Kanem said, slamming the Swiss cheese of a safety net she sees in most countries.

Kanem speaks of childbirth horror stories: pregnant women unable to access caesarian procedures (many of which are unplanned) or blood pressure medication because of redeployed health care resources, or the woman gets to the clinic (and) the midwife isnt there, because theyre also redeployed or sick. Up to two-thirds of maternal and neonatal deaths globally occur because of the absence of properly trained midwives in better times.

During World War II, women on the U.S. homefront think Rosie the Riveter entered the workforce out of a call to sacrifice for the common good. During this pandemic, women are being called back but this time to the frontlines.

Welcome to POLITICO Nightly: Coronavirus Special Edition. Of course Matthew McConaugheys mask says just keep livin. Reach out with tips: [emailprotected] or on Twitter at @renurayasam.

A message from PhRMA:

In these unprecedented times, Americas biopharmaceutical companies are coming together to achieve one shared goal: beating COVID-19. We are working with governments and insurers to ensure that when new treatments and vaccines are approved, they will be available and affordable for patients. Explore our efforts.

THE COVID DOCTRINE For much of the nations 100 days at war with the coronavirus, Donald Trump has been a commander in chief in search of an exit strategy, Adam Cancryn writes. The president has promised the virus will simply disappear, touted unproven treatments as miracle cures and fantasized about a near future of economic resurgence and rapid return to normalcy. Yet as the White House shifts its focus away from the public health response and toward rebuilding an economy ravaged by the pandemic, there remains little clear sense even within his own administration of how close the U.S. is to victory, and what winning the war even looks like.

PINS AND NEEDLES Our executive health editor Joanne Kenen emails: Theres some good news on the vaccine front including word that the country is getting a new vaccine leader. Peter Marks has emerged as the Trump administrations unofficial vaccine czar (minus the cross and pearls) at the FDA, filling in the gap created by the abrupt ouster of Rick Bright from the Biomedical Advanced Research and Development Authority. Marks will advise BARDA and other agencies on vaccine and gene therapy approval, health care reporter Sarah Owermohle reports.

News of Marks growing involvement comes amid a spate of heartening though still, we cant emphasize enough, very preliminary news about vaccine development, here and abroad. Oxford has a candidate vaccine thats safe in humans; its still testing whether it can create a strong enough immune response to combat the coronavirus. Three other companies have announced accelerations of clinical trials, though widespread availability in the best possible scenario is still months away.

Even if we get a vaccine, Joanne writes, big questions have to be answered.

Who gets it second? First responders will get it first. But how are we going to define a first responder? Anyone who works at a hospital? Only doctors and nurses? Doctors in the community? Police? Firefighters? The military?

But then who gets it second? The elderly and immune compromised because they are vulnerable? The young and healthy because they transmit it? Essential workers because theyre essential? The well-connected? And who decides?

Its possible several vaccines will come on the market at around the same time in different countries, so there could be multiple answers to this question. But this is going to be a huge bioethical knot, colliding with geopolitics. Theres no guarantee that a U.S. company will get to market first and if the World Health Organization has a role in vaccine allocation, we can anticipate some obvious conflicts.

How effective is it? If we get a good but not great vaccine like the seasonal flu shots it will still reduce transmission, but it wont wipe out the virus completely. Well still have to deal with Covid-19, though on a more manageable scale.

Who pays for it? Even if insurers, governments or, in countries other than our own, national health systems pay for immunization, the costs can be passed on indirectly through higher taxes or higher premiums.

How much does it cost? Some of the companies say they dont plan to make a profit, but vaccines are expensive. In the U.S., Trump has largely shunned national approaches, leaving states to fend for themselves as they try to acquire lab testing supplies, protective gear, ventilators and other essential pandemic-fighting goods. A similarly fragmented approach could make vaccine acquisition more expensive and complicated.

How do we make enough of it? One of BARDAs roles is to help ramp up production, and theyve started addressing this. But to make 7 billion vaccines, enough for everyone around the globe, will require commitment, creativity and cooperation that the world hasnt been very good at of late.

How fast will poor countries get access? Good question.

Will the anti-vaxxers take it? Well see. Best guess is that some will, and some wont, because not everybody who opposes vaccines does so for the same reasons or with the same intensity. Some people who dont want their child to get a measles shot may weigh the costs and the benefits differently for a coronavirus vaccine. Amid rising fears of bioterrorism after 9/11, a poll found deep but not overwhelming support for a smallpox vaccination campaign. But that was a hypothetical threat. This one is all too real.

A SMALL BREAKTHROUGH More than three decades ago, researchers made their first big breakthrough against HIV, when they showed that the drug AZT could slow the progression of the virus. Its a moment that Anthony Fauci compared to todays results about the drug remdesivir, which a clinical trial showed could help Covid patients recover more quickly.

Faucis reference to AZT was a bit like a secret code, Sarah Owermohle tells us. He was suggesting that the remdesivir results were a breakthrough, but a modest one. AZT is the shorthand for azidothymidine, a drug that won FDA approval in March 1987, when HIV patients were desperate for any treatment even one with rough side effects that was dogged by questions about whether it actually extended life. It took another decade before the development of drugs that turned HIV from a death sentence into a chronic condition.

HIV and Covid-19 are complex, but distinctly different, viruses, and drug development times are a lot faster now than they were in the 1980s and 90s. But Faucis implication was clear: Remdesivir could be a good first step in fighting Covid, but probably isnt a miracle drug.

A SICK ECONOMY The U.S. economy shrank at a 4.8 percent annual rate last quarter as the pandemic shut down much of the country. A huge percentage of the decline came from the health care industry, with a halt in elective procedures harming profits.

CLAIMS DENIED As businesses in Georgia, Texas and other states throw open their doors, many employees are scared that their employers arent taking proper health precautions. Yet if they refuse offers to return to their jobs theyll be ineligible for unemployment, reporter Megan Cassella tells us.

Trump has declared meatpacking plants essential businesses even as they spawn outbreaks across the country. Frontline health workers are having trouble getting masks, gloves, gowns and other protection equipment, so what hope do nail salons and restaurants have of getting the gear they need?

But for now, Covid fears arent a valid reason not to go back to work.

Some states are trying to take steps so that workers who feel unsafe arent forced to choose a paycheck over their health. Colorado and New York are looking at how to give workers more flexibility. In Georgia, the state labor agency is encouraging employers to negotiate back-to-work plans with employees so that if a business partially reopens, workers who feel unsafe can continue to collect unemployment. In Texas, advocates are asking the workforce commission to add voluntarily leaving work due to COVID-19 as a valid reason to claim assistance.

But other states, like South Carolina and Tennessee, are telling workers they will lose unemployment aid the same week they turn down an offer.

Even in boom times, states reject a high share of unemployment claims. Well probably learn Thursday that another 3.5 million people filed for unemployment assistance last week. Thats on top of the 26 million whove already lost their jobs in the past five weeks.

Our question for readers this week: Seeing any interesting, fun or meaningful signs related to the coronavirus? Snap a photo sometime this week and send it to Renu at [emailprotected], and well share the best ones on Friday.

GRAND OLD PACHYDERM Matt Wuerker dives into an old question on partisan symbols in the latest edition of Punchlines: Why is the elephant the symbol of the Republican Party?

MASS HYSTERIA Italian politicians clash with the Catholic clergy at their own peril, and Prime Minister Giuseppe Conte has risked doing just that by keeping churches closed because of coronavirus. The prime minister's decision to extend the ban on all religious ceremonies until further notice, except for funerals, has infuriated religious officials. When the lockdown started, most priests quietly accepted the need to suspend services and found alternative ways to connect with their flocks, such as holding ceremonies by video or taking confession by the roadside. But now that other places are gradually reopening, the clergy don't see why they should be last on the list.

82,000

The number of job losses forecast in the bus industry, according to a report released last week by the American Bus Association. The industry could see losses of up to $14 billion. Many of the 3,000 private bus companies in the U.S. are small, serving a range of uses from taking kids to school, sporting events and field trips, ferrying seniors on weekend getaways and connecting small towns with major destinations. (h/t transportation reporter Tanya Snyder)

Portuguese army chief of staff Gen. Jos Nunes da Fonseca attends a briefing of school workers on disinfection procedures. | Armando Franca/AP Photo

DEEP FRIED STATE Belgium, the North Sea homeland of moules frites and mayonnaise, is the world's biggest exporter of frozen fries, but it has been hammered by the pandemics trade slowdown. The Belgian potato industry has warned that more than 750,000 tons of potatoes could be thrown away more than 40 percent of the harvest. And though Belgium's potato industry has urged patriots to take a high-calorie hit for the team by heading down to their local friteries twice a week to help reduce the spud surplus, it's increasingly clear that 11 million Belgians won't be able to handle the deep-fried mission alone. With restaurants and bars closed, and large summer events canceled, fries wont be as ubiquitous as they often are this summer. "Our entire sector is facing a big crisis. We don't just invite all Belgians to eat more fries, but the entire world," said Ward Claerbout from Agristo, a potato processing company in the west of Belgium.

Correction: Tuesdays edition of POLITICO Nightly incorrectly stated which tracks Iowa will open without spectators. The state will reopen certain race tracks without spectators but not horse and dog tracks. We regret the error.

A message from PhRMA:

In these unprecedented times, Americas biopharmaceutical companies are coming together to achieve one shared goal: beating COVID-19. The investments weve made have prepared us to act swiftly: Working with governments and insurers to ensure that when new treatments and vaccines are approved, they will be available and affordable for patients Coordinating with governments and diagnostic partners to increase COVID-19 testing capability and capacity Protecting the integrity of the pharmaceutical supply chain and keeping our plants open to maintain a steady supply of medicines for patientsExplore our efforts.

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Covid's war on women - Politico

Advanced Therapy Medicinal Products Market: Overview

Lately, there has been remarkable shift in the therapeutics method. People are inclining towards personalized medication rather than pharmaceutical treatment methods. This is result of advancements in biological therapies. As a result, advanced therapy medicinal products market (ATMP) is emerging. Such products provide solution for conditions with no therapeutic alternatives. This is a key factor driving growth of the advanced therapy medicinal products market across the globe.

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At present, regulations for ATMPs is at its budding stage. Extensive research activities has been going on, which is resulting in Investigational New Drug (IND) applications.

The upcoming advanced therapy medicinal products market analysis report provides insight about the upcoming trends and restraining factors likely to shape growth of the market during forecast period (2019-2029). The report also provides a comprehensive analysis of the key companies of the market and offers details about the capacities and competencies of these companies. The market report also focusses on the markets competitive landscape and provides detail of the product portfolio of various companies.

Advanced Therapy Medicinal Products Market: Competitive Analysis

At present, the advanced therapy medicinal products market is growing at a lucrative rate. This growth rate is attributed to recent approval of various advanced therapy medicinal products. Post success of approved products, stakeholders are investing at enormously in clinical trials of advanced therapy medicinal products.

On the other hand, companies operating in the market are adopting various strategies to accelerate the product manufacturing rate. While most of the companies are relying on in-house production of therapies, few players such as Contract Manufacturing Organizations (CMOs) are opting for third-party service providers.

Upsurge in demand for gene therapy has resulted in widening of drug development landscape and rise in the number of new entrants. However, there is lack of production capabilities.

Also, several companies are strengthening their foothold in the global market by strategic alliances and acquiring small CAR T-cell therapy developers.

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Some of the key players operating in the advanced therapy medicinal products market are-

Advanced Therapy Medicinal Products Market: Key Trends

Despite high cost of the products, the market is expanding at lucrative rate. The growth rate is attributed to various health benefits provided by new classes of therapies.

Registering clinical benefits and efficiency of the products, stakeholders in the market are developing new strategies to overcome the challenged and boost application of advanced therapy medicinal products

Advanced Therapy Medicinal Products Market: Regional Outlook

Availability of significant number of FDA approved advanced therapy medicinal products in the U.S. has accounted for the prominent share of North America region, in terms of revenue. Recently, approval of products such as Yescarta, Zolgensma, and Kymriah has led to prominent investment in the U.S. advanced therapy medicinal products market.

Europe hold second-largest pharmaceutical market space across the globe. In coming years, cell therapy developers are anticipated to hold prominent share in the Europes drug revenue. Moreover, several academic institutes in Europe are conducting extensive research in early-stage cell therapy. This factor is likely to fuel the regional revenue contribution.

Meanwhile, global manufacturing companies operating in the market are enhancing their reach across Europe. This, in turn, may drive growth in the regional market.

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Advanced Therapy Medicinal Products Market Statistics, Segment, Trends and Forecast 2029 - Cole of Duty

XconomyTexas

Some former executives and investors behind the second gene therapy to win FDA approval are teaming up again. Their new company, Taysha Gene Therapies, is launching with $30 million in the bank and a plan to bring its first central nervous system (CNS) disorder program into human testing later this year.

Taysha is focusing on monogenic CNS diseases, which are caused by a single mutation in a single gene, says president and CEO RA Session II. The Dallas-based startup is developing treatments for rare genetic diseases as well as conditions that affect larger segments of the population, such as epilepsy and neurodegenerative disorders.

As long as its a monogenic CNS disease, were going to go after it, Session says.

Taysha has a pipeline of 15 gene therapies, and an option to add four more. The experimental treatments come from the University of Texas Southwestern (UTSW) Medical Center, which has a gene therapy program led by Steven Gray and Berge Minassian. Session, a former pharmaceutical industry executive who is currently an entrepreneur in residence at UTSW, says university administrators asked him to come up with ideas for funding the gene therapy research. Some entities were interested in taking on individual assets, but Sessions concluded that the best way to fund the entire portfolio was to create one company.

Rare diseases represent an unmet medical need, but even when the biology is clear a therapys commercial prospects can be murky. Drugs for chronic conditions can take several years to reach peak sales, but that peak then continues for years as more patients use a product, Session says. In rare diseases, this time to peak is typically achieved in the first year to 18 months of sales. Afterward, revenue falls because a rare disease by definition does not affect a big pool of potential new patients. Session says that in order to be financially sustainable, a drug company needs to constantly launch new products, and the UTSW assets give Taysha the opportunity to do just that.

It helps that Taysha is not starting from scratch. Session is a former business development executive for AveXis, a gene therapy developer that got its start in Dallas. Novartis (NYSE: NVS) acquired AveXis in 2018, and made the company its gene therapy division. Last year, AveXiss Zolgensma won FDA approval for spinal muscular atrophythe second gene therapy to receive the regulators nod.

Gene therapy uses an engineered virus to deliver a functioning gene that replaces a defective one. Tayshas gene therapies will be delivered by adeno-associated virus 9 (AAV9), a virus that can cross the blood-brain barrier. Its the same virus used for Zolgensma and Session says AveXiss work shows that the virus can be given to patients safely and effectively. Also, experience with the viral technology gives the company a leg up on knowing how to manufacture any therapies it produces quickly and at scale, he adds.

The seed financing announced Wednesday will fund Tayshas early work. PBM Capital, the first investor in AveXis, and Nolan Capital, the investment fund of former AveXis CEO Sean Nolan, co-led the round.

Tayshas lead program is TGTX-101, an experimental gene replacement therapy for GM2-gangliosidosis, a rare, inherited enzyme disorder that leads to the destruction of neurons in the brain and spinal cord. Session says this program is expected to begin clinical testing later this year. The company aims to ask the FDA for permission to start clinical trials for three additional gene therapies by the end of next year. Those programs include the mitochondrial disorder SURF1 deficiency; a form of epilepsy caused by a mutation to the SLC6A1 gene; and Rett syndrome, a neurological disorder caused by a genetic mutation.

In addition to advancing new gene therapies, Taysha is also developing new gene therapy technologies. Session says some of the research focuses on new capsids, the protein shells that enclose a virus. These next-generation capsids could potentially get a gene therapy to different cells in the body more efficiently and effectively, which could lead to therapies that require a lower dose, ultimately improving safety, he says.

Taysha is also developing technology for redosing a gene therapy. While these therapies are meant to be long-lasting, their durability over the course of a patients lifetime is still unknown, Session says. Redosing with the same therapy isnt possible because patients develop antibodies to the virus. Taysha is developing a way to redose via a new route of administrationthe vagus nerve. Session declined to elaborate, saying only that its early but its interesting. Its an issue the sector has been grasping at and trying to solve.

AveXis is still developing new gene therapies. A company executive told Xconomy earlier this year that an application to start human tests of a Rett syndrome therapy is expected to be ready by mid-2020. A number of companies on are on AveXiss heels, some of them focused in particular on the central nervous system and the brain. The CNS gene therapies at Passage Bio (NASDAQ: PASG) are from the University of Pennsylvania. New York-based Neurogene is also focused on rare monogenic neurological diseases. Prevail Therapeutics of New York and Voyager Therapeutics (NASDAQ: VYGR) are developing gene therapies for Parkinsons disease.

Taysha will develop its programs in partnership with UTSW. The university is handling discovery and preclinical research, as well as the studies leading up to an application to start clinical trials. UTSW will also handle manufacturing for clinical trials. Taysha will take the lead on clinical testing and regulatory work. If any of the gene therapies win regulatory approval, the company will take over manufacturing and handle commercialization.

Photo by Flickr user Katie Haugland Bowen via a Creative Commons license

Frank Vinluan is an Xconomy editor based in Research Triangle Park. You can reach him at fvinluan@xconomy.com.

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Taysha Launches With $30M to Bring Gene Therapy to CNS Disorders - Xconomy

Biotech executive, Julia Berretta, Ph.D., is named Chief Executive Officer

Genespire, a biotechnology company focused on the development of transformative gene therapies for patients affected by genetic diseases, announced today the successful close of a 16M Series A financing from Sofinnova Partners, a leading European life sciences venture capital firm based in Paris, London and Milan. The company also announced the appointment of Julia Berretta, Ph.D., as Chief Executive Officer and member of the Board of Directors. Graziano Seghezzi, Managing Partner at Sofinnova Partners, and Lucia Faccio, Ph.D., Partner at Sofinnova Partners, will also join the Board.

Genespire was founded in March 2020 as a spin-off of the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget), one of the worlds leading cell and gene therapy research institutes spearheaded by gene therapy pioneer Prof. Luigi Naldini. The Company was co-founded by Fondazione Telethon and the San Raffaele Hospital, along with Prof. Naldini and Dr. Alessio Cantore.

The funds will be used to advance Genespires leading-edge platform technologies towards the development of novel gene therapies in two main areas: primary immunodeficiencies and metabolic genetic diseases.

"Our mission has always been to develop breakthrough solutions for genetic diseases," said Prof. Naldini, Genespires co-founder and Director of SR-Tiget. "This financing enables the company to translate our innovative science and early stage programs into clinical development. The appointment of Dr. Berretta as CEO is a major reinforcement of our team."

Sofinnova Partners Dr. Faccio added, "Genespire is an exciting investment with all the key ingredients for success: Outstanding scientists that developed the first ex-vivo gene therapy to market, experienced executives brought in through Sofinnova Partners network and game changing technologies that have the potential to impact the lives of patients with genetic diseases."

"I am thrilled to be joining Genespire and such exceptional scientific founders," said Dr. Berretta. "Genespire was born of decades of experience in the gene therapy field, and is optimally positioned to advance transformative therapies for patients affected by severe inherited diseases."

Dr. Berretta was part of the Executive Committee of Cellectis S.A., a Nasdaq-listed clinical stage gene editing company developing CAR-T cell therapies for cancer, where she led business development as well as strategic planning. She is also an independent Board member of Treefrog Therapeutics, an innovative stem cell company.

About Genespire

Genespire is a biotechnology company focused on the development of transformative gene therapies for patients affected by genetic diseases, particularly primary immunodeficiencies and inherited metabolic diseases. Based in Milan, Italy, Genespire was founded in March 2020 by the gene therapy pioneer Prof. Luigi Naldini, Dr. Alessio Cantore, Fondazione Telethon and Ospedale San Raffaele. It is a spin-off of SR-Tiget, a world leading cell and gene therapy research institute and is backed by Sofinnova Partners. http://www.genespire.com

About Sofinnova Partners

Sofinnova Partners is a leading European venture capital firm specialized in Life Sciences. Based in Paris, France, with offices in London and Milan, the firm brings together a team of 40 professionals from all over Europe, the U.S. and Asia. The firm focuses on paradigm-shifting technologies alongside visionary entrepreneurs. Sofinnova Partners invests across the Life Sciences value chain as a lead or cornerstone investor, from very early-stage opportunities to late-stage/public companies. It has backed nearly 500 companies over more than 48 years, creating market leaders around the globe. Today, Sofinnova Partners has over 2 billion under management.

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

About Fondazione Telethon

Fondazione Telethon is a non-profit organisation created in 1990 as a response to the appeals of a patient association group of stakeholders, who saw scientific research as the only real opportunity to effectively fight genetic diseases. Thanks to the funds raised through the television marathon, along with other initiatives and a network of partners and volunteers, Telethon finances the best scientific research on rare genetic diseases, evaluated and selected by independent internationally renowned experts, with the ultimate objective of making the treatments developed available to everyone who needs them. Throughout its 30 years of activity, Fondazione Telethon has invested more than 528 million in funding more than 2.630 projects to study more than 570 diseases, involving over 1.600 scientists. Fondazione Telethon has made a significant contribution to the worldwide advancement of knowledge regarding rare genetic diseases and of academic research and drug development with a view to developing treatments. For more information, please visit: http://www.telethon.it

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About Ospedale San Raffaele

Ospedale San Raffaele (OSR) is a clinical-research-university hospital established in 1971 to provide international-level specialised care for the most complex and difficult health conditions. OSR is part of Gruppo San Donato, the leading hospital group in Italy. The hospital is a multi-specialty center with over 60 clinical specialties; it is accredited by the Italian National Health System to provide care to both public and private, national and international patients. Research at OSR focuses on integrating basic, translational and clinical activities to provide the most advanced care to our patients. The institute is recognized as a global authority in molecular medicine and gene therapy, and is at the forefront of research in many other fields. Ospedale San Raffaele is a first-class institute which treats many diseases and stands out for the deep interaction between clinical and scientific area. This makes the transfer of scientific results from the laboratories to the patients bed easier. Its mission is to improve knowledge of diseases, identify new therapies and encourage young scientists and doctor to grow professionally. For more information, please visit: http://www.hsr.it

About the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget)

Based in Milan, Italy, the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) is a joint venture between the Ospedale San Raffaele and Fondazione Telethon. SR-Tiget was established in 1995 to perform research on gene transfer and cell transplantation and translate its results into clinical applications of gene and cell therapies for different genetic diseases.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200429005417/en/

Contacts

Julia BerrettaCEO, Genespire S.r.linfo@genespire.com +39 02 83991300

Bommy LeeHead of Communications, Sofinnova Partnersblee@sofinnovapartners.com +33 (0) 6 47 71 38 11

North AmericaRooneyPartners LLCKate Barrettekbarrette@rooneyco.com +1 212 223 0561

FranceStrategiesImage (S&I)Anne Reinanne.rein@strategiesimage.com +33 6 03 35 92 05

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Genespire Secures 16 Million Series A Financing from Sofinnova Partners to Advance Transformative Gene Therapies - Yahoo Finance

DURHAM, N.C., April 28, 2020 (GLOBE NEWSWIRE) -- Precision BioSciencesInc. (Nasdaq: DTIL), a life sciences company dedicated to improving life through the application of its pioneering, proprietary ARCUS gene editing platform, today announced that the Company and its collaborators will present at the upcoming American Society of Genetic & Cell Therapy (ASGCT) Annual Meeting held virtually May 12-15, 2020.

The abstracts being presented by Precision and our collaborators this year at ASGCT underscore the differentiated capabilities of our proprietary ARCUS genome editing platform and the breadth of our emerging pipeline applying this technology in vivo, commented Derek Jantz, Chief Scientific Officer and co-founder of Precision BioSciences. These presentations demonstrate the specificity and versatility of ARCUS-driven genome editing in a variety of large animal models and provide further preclinical evidence of potentially meaningful and durable therapeutic impact on a range of genetic and infectious diseases. As we continue to validate ARCUS potential in vivo, we are focused on advancing our gene correction pipeline. We look forward to selecting a clinical candidate for our wholly owned PH1 program, expected in 2020, and, in partnership with Gilead, developing a potential cure for chronic hepatitis B infection, for which submission of an IND is currently targeted for 2021.

Precision BioSciences Presentations:

Title:Engineering a Self-Inactivating Adeno-Associated Virus (AAV) Vector for ARCUS Nuclease DeliveryPoster Session: Gene Targeting and Gene Correction, Abstract: 654Presenting Author: Hui Li, Ph.D., Precision BioSciences

Title:A Gene Editing Approach to Eliminate Hepatitis B Virus Using ARCUS MeganucleasesPoster Session: Gene Targeting and Gene Correction, Abstract 1057Presenting Author: Cassie Gorsuch, Ph.D., Precision BioSciences

Partnered Presentations:

Title: Therapeutic Efficacy of ARCUS Meganuclease Gene Editing - Arrest of Rod Degeneration and Restoration of Rod Function in a Transgenic Pig Model of Autosomal Dominant Retinitis PigmentosaOral Presentation: Gene Therapy for the Special Senses, Abstract 2Date/Time: Tuesday, May 12, 2020, 10:30 10:45 a.m. ESTPresenting Author: Maureen Ann McCall, Ph.D., Professor, Department of Ophthalmology and Visual Sciences, University of Louisville

Title: Evaluation of the Long-term Effects of AAV-Meganuclease Genome Editing of PCSK9 in Macaque LiverOral Presentation: Evaluating Genome Editing Activity and Precision, Abstract 518Date/Time: Wednesday, May 13, 2020, 4:00 4:15 p.m. ESTPresenting Author: Lili Wang, Ph.D., Research Director, Discovery Research and Gene Editing, Research Associate Professor, Department of Medicine, Perelman School of Medicine, University of Pennsylvania

All abstracts for the ASGCT 2020 Meeting are available online at ASGCT Annual Meeting Abstracts.

About Precision BioSciences, Inc.Precision BioSciences is dedicated to improving life (DTIL) through its proprietary genome editing platform, ARCUS. Precision leverages ARCUS in the development of its product candidates, which are designed to treat human diseases and create healthy and sustainable food and agriculture solutions. Precision is actively developing product candidates in three innovative areas: allogeneic CAR T immunotherapy, in vivo gene correction, and food. For more information regarding Precision, please visitwww.precisionbiosciences.com.

Forward-LookingStatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including the timing of trials and results from clinical and non-clinical studies of our in vivo gene correction program and the safety, efficacy and delivery of our ARCUS genome editing technology. In some cases, you can identify forward-looking statements by terms such as anticipate, believe, could, expect, should, plan, intend, estimate, target, mission, may, will, would, should, could, target, project, predict, contemplate, potential, or the negative thereof and similar words and expressions.

Forward-looking statements are based on managements current expectations, beliefs and assumptions and on information currently available to us. Such statements are subject to a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to, our ability to become profitable; our ability to procure sufficient funding and requirements under our current debt instruments; our limited operating history; the success of our programs and product candidates; our dependence on our ARCUS technology; the initiation, cost, timing, progress and results of research and development activities, preclinical or greenhouse studies and clinical or field trials; our or our collaborators ability to identify, develop and commercialize product candidates; our or our collaborators ability to advance product candidates into, and successfully complete, clinical or field trials; our or our collaborators ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; the laws and regulatory landscape that will apply to our and our collaborators development of product candidates; our ability to achieve our anticipated operating efficiencies as we commence manufacturing operations at our new facility; delays or difficulties in enrolling patients in clinical trials; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriate of intellectual property rights; if our product candidates do not work as intended or cause undesirable side effects the potential for off-target editing or other adverse events, undesirable side effects or unexpected characteristics associated with any of our product candidates; risks associated with applicable healthcare, data privacy and security regulations and our compliance therewith; the rate and degree of market acceptance of any of our product candidates; the success of our existing collaboration agreements; our ability to enter into new collaboration arrangements; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, biotechnology and agricultural biotechnology fields; potential manufacturing problems associated with any of our product candidates; pending and potential liability lawsuits and penalties related to our technology, our product candidates; the outbreak of the novel coronavirus disease (COVID-19); our current and future relationships with third parties; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate key scientific and management personnel; effects of natural or manmade disasters, public health emergencies and other natural catastrophic events; insurance expenses and exposure to uninsured liabilities; market and economic conditions; dilution and fluctuations in our stock price; and other important factors discussed under the caption Risk Factors in our Annual Report on Form 10-K for the fiscal year ended December 31, 2019, as supplemented by the risk factor contained in our Current Report on Form 8-K filed with the SEC on April 6, 2020, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SECs website atwww.sec.gov.

All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we do not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

Investor Contacts:Nick RiddlePrecision BioSciencesTel. (919) 314-5512IR@precisionbiosciences.com

Josh RappaportStern Investor RelationsTel. (212) 362-1200josh.rappaport@sternir.com

Media Contact:Maurissa MessierPrecision BioSciencesTel. (919) 314-5512media@precisionbiosciences.com

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Precision BioSciences Announces Presentations at the American Society of Gene & Cell Therapy 23rd Annual Meeting - BioSpace

~22 Presentations at ASGCT, Including New Preclinical Data on Gene Therapy Candidates, Highlight uniQures Industry-Leading Research and Technology Capabilities ~

LEXINGTON, Mass. and AMSTERDAM, April 28, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that 22 data presentations, of which five are oral presentations, will be delivered at the American Society of Gene and Cell Therapy (ASGCT) Virtual 2020 Annual Meeting being held May 12-15.

"uniQures significant scientific presence at this years ASGCT Annual Meeting demonstrates the robustness of our research capabilities and our excellence in developing novel technologies and commercial-scale gene therapy manufacturing, stated Sander van Deventer, executive vice president of research and product development at uniQure. We are very pleased to present new preclinical data on our gene therapy candidates for hemophilia A, spinocerebellar ataxia type 3, Fabry disease and data on our advances in technology and manufacturing.

Specific details on uniQures virtual oral presentations at ASGCT include:

Title: One-Time Intrathecal Administration of AAV5-miATXN3 in Non-Human PrimatesDate and Time: Wednesday May 13, 4:15 p.m. EDT/ 10:15 p.m. CET

Title: A Novel NAGA Variant Designed to be Non-immunogenic In Humans and Provide Broad Cross-Correction in Fabry DiseaseDate and Time: Thursday May 14, 4:15 p.m. EDT/ 10:15 p.m. CET

Title: A Single Administration of AAV5-hFIX in Newborn, Juvenile and Adult Mice Leads to Stable hFIX Expression up to 18 Months after DosingDate and Time: Thursday May 14, 4:15 p.m. EDT/ 10:15 p.m. CET

Title: Characterizing Next-Generation Baculovirus Transduction Processes - A Quality by Design-based Approach for AAV ManufacturingDate and Time: Friday May 15, 10:45 a.m. EDT/ 4:45 p.m. CET

Title: Clearance of Vector DNA From Bodily Fluids in Patients with Severe or Moderate-Severe Hemophilia B Following Systemic Administration of AAV5-hFIX and AAV5-hFIX PaduaDate and Time: Friday May 15, 11:15 a.m. EDT/ 5:15 p.m. CET

The following presentations were approved for poster presentation:

The abstracts were published today at the ASGCT Annual Meeting website.

About uniQure

uniQure is delivering on the promise of gene therapy single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, hemophilia A, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases.www.uniQure.com

Originally posted here:
uniQure Announces Significant Presence at Upcoming American Society of Gene and Cell Therapy (ASGCT) Virtual Annual Meeting - Yahoo Finance

SAN RAFAEL, Calif., April29, 2020 /PRNewswire/ --

Financial Highlights (in millions of U.S. dollars, except per share data, unaudited)

Three Months Ended March 31,

2020

2019

% Change

Total Revenues

$

502.1

$

400.7

25

%

Net Product Revenues Marketed by BioMarin (1)

433.3

349.2

24

%

Vimizim Net Product Revenues

137.2

125.8

9

%

Kuvan Net Product Revenues

122.0

106.9

14

%

Naglazyme Net Product Revenues

114.3

86.9

32

%

Palynziq Net Product Revenues

34.6

12.3

181

%

Brineura Net Product Revenues

24.0

12.2

97

%

Aldurazyme Net Product Revenues

55.7

45.3

23

%

GAAP Net Income (Loss)

$

81.4

$

(56.5)

GAAP Net Income (Loss) per Share Basic

$

0.45

$

(0.32)

GAAP Net Income (Loss) per Share Diluted

$

0.44

$

(0.32)

Non-GAAP Income(2)

$

116.5

$

24.8

March 31,

2020

December 31,

2019

Cash, cash equivalents and investments

$

1,149.2

$

1,165.8

(1)

Net Product Revenues Marketed by BioMarin is the sum of revenues from Vimizim, Kuvan, Naglazyme, Palynziq, Brineura and Firdapse, each calculated in accordance with Generally Accepted Accounting Principles in the United States (U.S. GAAP). Sanofi Genzyme (Genzyme) is BioMarin's sole customer for Aldurazyme and is responsible for marketing and selling Aldurazyme to third parties. Refer to page9 for a table showing Net Product Revenues by product, including Firdapse. In January 2020, BioMarin divested the Firdapse assets to a third party in a sale transaction. The sale will be reflected in the Company's consolidated financial statements for the three months ending March 31, 2020; as a result of the transaction BioMarin will not recognize Net Product Revenues from Firdapse in the future.

(2)

Non-GAAP Income is defined by the Company as reported GAAP Net Income, excluding net interest expense, provision for (benefit from) income taxes, depreciation expense, amortization expense, stock-based compensation expense, contingent consideration expense and, in certain periods, certain other specified items. Refer to Non-GAAP Information beginning on page10 of this press release for a complete discussion of the Company's Non-GAAP financial information and reconciliations to the comparable information reported under U.S. GAAP.

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) (BioMarin or the Company) today announced financial results for the first quarter ended March31, 2020.

Total Revenues increased 25% to $502.1 million. The increase in Total Revenues was primarily attributed to increased Net Product Revenues which were $489.0 million in the first quarter of 2020, compared to $394.5 million for the first quarter of 2019. The increase in Net Product Revenues was attributed to the following:

The increase in GAAP Net Income for the first quarter of 2020, compared to GAAP Net Loss for the same period in 2019 was primarily due to the following:

Non-GAAP Income for the first quarter of 2020 increased to $116.5 million, compared to Non-GAAP Income of $24.8 million for the same period in 2019. The increase in Non-GAAP Income for the quarter, compared to the same period in 2019, was attributed to higher gross profit and decreased R&D expense, partially offset by higher SG&A expense.

As of March31, 2020, BioMarin had cash, cash equivalents and investments totaling approximately $1.1 billion, as compared to $1.2 billion on December31, 2019.

Commenting on first quarter 2020 results, Jean-Jacques Bienaim, Chairman and Chief Executive Officer of BioMarin, said, "With the arrival of COVID-19 to the many regions where we do business, BioMarin employees performed in unprecedented ways to ensure the continued supply of our critically-important medicines to the people we serve. I am proud of the commitment and dedication demonstrated by our colleagues in these challenging times. Our strong financial results in the first quarter underscore both the essential-nature of our products to patients and the extraordinary efforts made to maintain supply around the world. In the face of the many challenges of COVID-19, our regulatory team further progressed our next two potential commercial products. The Biologics License Application (BLA) for valoctocogene roxaparvovec for severe hemophilia A was accepted for Priority Review from the FDA with an action date of August 21, 2020. This milestone represents a tremendous achievement for BioMarin, but the potential approval of the first gene therapy in any type of hemophilia is an even greater triumph for the hemophilia community. They have been waiting decades for this groundbreaking advancement and we are honored to be on this journey together. With an approval decision for valoctocogene roxaparvovec expected later this year, our commercial team prepares eagerly to launch what we believe is the most innovative product yet for people with bleeding disorders."

Mr. Bienaim continued, "Based on positive interactions with U.S. and European regulatory authorities in the quarter, we plan to submit marketing applications in both regions for vosoritide to treat children with achondroplasia in the third quarter of this year. Our multi-pronged dossier of data encompasses long-term clinical results in 5 to 18 year-olds, natural history data, the ongoing study of newborns through 5 years, and highly statistically significant placebo-controlled Phase 3 results. The positive and significant results from our vosoritide clinical programs have led us to believe that this potential drug could be the first pharmacological treatment for the underlying cause of achondroplasia. Interest in our clinical studies with vosoritide has been extremely robust, demonstrating that families are keen to seek early treatment for their children."

Mr. Bienaim concluded, "2020 is expected to be a transformational year for BioMarin, despite impact from COVID-19 in the near-term. The agility demonstrated by BioMarin employees in the face of this global pandemic has enabled the continued supply of our essential medicines to the patients who need them. And while we expect minor financial impact in the near-term, our business is well-positioned to weather such challenges. Our first quarter revenue growth and improvement in profitability support our belief that 2020 continues to look poised to be one of our most significant value-creating years to date."

2020 Full-Year Financial Guidance

Due to the uncertainty surrounding the COVID-19 pandemic and the potential impact on its business, BioMarin is reducing its guidance for Total Revenues and Net Product Revenues for Vimizim, Naglazyme and Palynziq for 2020.

Item

Provided February 26, 2020

Updated April 29, 2020

Total Revenues (1)

$1,950

to

$2,050

$1,850

to

Read more from the original source:
BioMarin Announces First Quarter 2020 Total Revenue Growth of 25% to $502 million - PRNewswire

Dive Brief:

Merck KGaA, like other contract manufacturers such as Lonza, is betting the next big wave of demand will be for complex production of gene therapies and other products such as viral vaccines and immunotherapies. The gene therapy market will grow to about $10 billion by 2026 from $1 billion in 2018, the company said, citing estimates from Biotech Forecasts.

"Viral vector manufacturing has transitioned from a niche industry to the cornerstone of the future of biopharmaceuticals," said Udit Batra, head of Merck KGaA'slife science business, in a statement.

The German company has been on a spending spree in recent years, announcing plans to invest 1 billion euros in its global headquarters in Darmstadt, more than $400 million in two sites in Switzerland,and $70 million in a research and development hub expansion in Billerica, Massachusetts.

Carlsbad is already home to a Merck KGaA facility that has been involved in gene therapy since 1997, about the time that researchers beginning studying the potential for such treatments in people. At present, the site has 16 modular viral bulk manufacturing clean room suites and two fill/finish suites, Merck KGaA said.

With the new facility, the Carlsbad location will have 27 suites used in different parts of the manufacturing process and will support production at the 1000-liter scale using single-use equipment, Merck KGaA said.

The company also has a manufacturing facility in Glasgow that produces intermediates and final products for gene therapy and viral vaccines.

Merck KGaA, established in 1688, is majority owned by descendants of the original founder and had sales of 16.2 billion euros last year. The U.S. pharmaceutical giant Merck was once a subsidiary but is no longer associated with its German namesake.

View post:
Merck KGaA to spend $110M on new gene therapy facility in California - BioPharma Dive

What are exosomes?

Exosomes are a class of cell-derived extracellular vesicles of endosomal origin, and are typically 30-150 nm in diameter the smallest type of extracellular vesicle.1 Enveloped by a lipid bilayer, exosomes are released into the extracellular environment containing a complex cargo of contents derived from the original cell, including proteins, lipids, mRNA, miRNA and DNA.2 Exosomes are defined by how they are formed through the fusion and exocytosis of multivesicular bodies into the extracellular space.

Multivesicular bodies* are unique organelles in the endocytic pathway that function as intermediates between early and late endosomes.3 The main function of multivesicular bodies is to separate components that will be recycled elsewhere from those that will be degraded by lysosomes.4 The vesicles that accumulate within multivesicular bodies are categorized as intraluminal vesicles while inside the cytoplasm and exosomes when released from the cell.

*Confusingly, there is inconsistency in the literature; while some sources differentiate multivesicular bodies from late endosomes, others use the two interchangeably.

Exosomes are of general interest for their role in cell biology, and for their potential therapeutic and diagnostic applications. It was originally thought that exosomes were simply cellular waste products, however their function is now known to extend beyond waste removal. Exosomes represent a novel mode of cell communication and contribute to a spectrum of biological processes in health and disease.2One of the main mechanisms by which exosomes are thought to exert their effects is via the transfer of exosome-associated RNA to recipient cells, where they influence protein machinery. There is growing evidence to support this, such as the identification of intact and functional exosomal RNA in recipient cells and certain RNA-binding proteins have been identified as likely players in the transfer of RNA to target cells.5,6 MicroRNAs and long noncoding RNAs are shuttled by exosomes and alter gene expression while proteins (e.g. heat shock proteins, cytoskeletal proteins, adhesion molecules, membrane transporter and fusion proteins) can directly affect target cells.7,8Exosomes have been described as messengers of both health and disease. While they are essential for normal physiological conditions, they also act to potentiate cellular stress and damage under disease states.2

Multivesicular bodies are a specialized subset of endosomes that contain membrane-bound intraluminal vesicles. Intraluminal vesicles are essentially the precursors of exosomes, and form by budding into the lumen of the multivesicular body. Most intraluminal vesicles fuse with lysosomes for subsequent degradation, while others are released into the extracellular space.9,10 The intraluminal vesicles that are secreted into the extracellular space become exosomes. This release occurs when the multivesicular body fuses with the plasma membrane.

The formation and degradation of exosomes.

This is an active area of research and it is not yet known how exosome release is regulated. However, recent advances in imaging protocols may allow exosome release events to be visualized at high spatiotemporal resolution.11

Exosomes have been implicated in a diverse range of conditions including neurodegenerative diseases, cancer, liver disease and heart failure. Like other microvesicles, the function of exosomes likely depends on the cargo they carry, which is dependent on the cell type in which they were produced.12 Researchers have studied exosomes in disease through a range of approaches, including:

In cancer, exosomes have multiple roles in metastatic spread, drug resistance and angiogenesis. Specifically, exosomes can alter the extracellular matrix to create space for migrating tumor cells.13,14 Several studies also indicate that exosomes can increase the migration, invasion and secretion of cancer cells by influencing genes involved with tumor suppression and extracellular matrix degradation.15,16Through general cell crosstalk, exosomal miRNA and lncRNA affect the progression of lung diseases including chronic obstructive pulmonary disease (COPD), asthma, tuberculosis and interstitial lung diseases. Stressors such as oxidant exposure can influence the secretion and cargo of exosomes, which in turn affect inflammatory reactions.17 Altered exosomal profiles in diseased states also imply a role for exosomes in many other conditions such as in neurodegenerative diseases and mental disorders.18,19Cells exposed to bacteria release exosomes which act like decoys to toxins, suggesting a protective effect during infection.20 In neuronal circuit development, and in many other systems, exosomal signaling is likely to be a sum of overlapping and sometimes opposing signaling networks.21

Exosomes can function as potential biomarkers, as their contents are molecular signatures of their originating cells. Due to the lipid bilayer, exosomal contents are relatively stable and protected against external proteases and other enzymes, making them attractive diagnostic tools. There are increasing reports that profiles of exosomal miRNA and lncRNA differ in patients with certain pathologies, compared with those of healthy people.17 Consequently, exosome-based diagnostic tests are being pursued for the early detection of cancer, diabetes and other diseases.22,23Many exosomal proteins, nucleic acids and lipids are being explored as potential clinically relevant biomarkers.24 Phosphorylation proteins are promising biomarkers that can be separated from exosomal samples even after five years in the freezer25, while exosomal microRNA also appears to be highly stable.26 Exosomes are also highly accessible as they are present in a wide array of biofluids (including blood, urine, saliva, tears, ascites, semen, colostrum, breast milk, amniotic fluid and cerebrospinal fluid), creating many opportunities for liquid biopsies.

Exosomes are being pursued for use in an array of potential therapeutic applications. While externally modified vesicles suffer from toxicity and rapid clearance, membranes of naturally occurring vesicles are better tolerated, offering low immunogenicity and a high resilience in extracellular fluid.27 These naturally-equipped nanovesicles could be therapeutically targeted or engineered as drug delivery systems.

Exosomes bear surface molecules that allow them to be targeted to recipient cells, where they deliver their payload. This could be used to target them to diseased tissues or organs.27 Exosomes may cross the blood-brain barrier, at least under certain conditions28 and could be used to deliver an array of therapies including small molecules, RNA therapies, proteins, viral gene therapy and CRISPR gene-editing.

Different approaches to creating drug-loaded exosomes include27:

Exosomes hold huge potential as a way to complement chimeric antigen receptor T (CAR-T) cells in attacking cancer cells. CAR exosomes, which are released from CAR-T cells, carry CAR on their surface and express a high level of cytotoxic molecules and inhibit tumor growth.29 Cancer cell-derived exosomes carrying associated antigens have also been shown to recruit an antitumor immune response.30

The purification of exosomes is a key challenge in the development of translational tools. Exosomes must be differentiated from other distinct populations of extracellular vesicles, such as microvesicles (which shed from the plasma membrane, also referred to as ectosomes or shedding vesicles) and apoptotic bodies.31 Although ultracentrifugation is regarded as the gold standard for exosome isolation, it has many disadvantages and alternative methods for exosome isolation are currently being sought. Exosome isolation is an active area of research (see Table 1) and many research groups are seeking ways to overcome the disadvantages listed below, while navigating the relevant regulatory hurdles along the way.

Produces a low yield and low purity of the isolated exosomes as other types of extracellular vesicles have similar sedimentation properties.

Low efficiency as it is labor-intensive, time-consuming and requires a large amount of sample. specialized equipment. High centrifugal force can damage exosome integrity

Originally posted here:
Exosomes: Definition, Function and Use in Therapy - Technology Networks

The Personalized Gene Therapy Treatment Market report market intelligence study intended to offer complete understanding of global market scenario with the Impact of COVID-19 (Corona Virus). It attempts to analyze the major components of the Market which have greater influence on it. This includes various elements of significant nature including market overview, segmentation, competition landscape, Market chain analysis, key players stratergyand more. Also, the report provides a 360-degree overview of global market on the basis of various analysis techniques including SWOT and Porters Five Forces. Approximations associated with the market values over the forecast period are based on empirical research and data collected through both primary and secondary sources. This might help readers to understand the strengths, opportunities, challenges and perceived threats of the market.

Based on Classification, each type is studied as Sales, Market Share (%), Revenue (Million USD), Price, Gross Margin and more similar information. The report can help to realize the market and strategize for business expansion accordingly. In the strategy analysis, it gives insights from marketing channel and market positioning to potential growth strategies, providing in-depth analysis for new entrants or exists competitors in the Personalized Gene Therapy Treatment industry.

The Personalized Gene Therapy Treatment Market report wraps:

There are 13 Chapters to thoroughly display the Personalized Gene Therapy Treatment market. This report included the analysis of market overview, market characteristics, industry chain, competition landscape, historical and future data by types, applications and regions.

In the end, The objective of the market research report is the current status of the market and in accordance classifies it into a few objects. The report takes into consideration the first market players in every area from over the globe.

Note In order to provide more accurate market forecast, all our reports will be updated before delivery by considering the impact of COVID-19.

Read the rest here:
Personalized Gene Therapy Treatment Market: What are the Future Growth Opportunities with Covid-19? - Cole of Duty

The Gene Therapy For Rare Disease Market report market intelligence study intended to offer complete understanding of global market scenario with the Impact of COVID-19 (Corona Virus). It attempts to analyze the major components of the Market which have greater influence on it. This includes various elements of significant nature including market overview, segmentation, competition landscape, Market chain analysis, key players stratergyand more. Also, the report provides a 360-degree overview of global market on the basis of various analysis techniques including SWOT and Porters Five Forces. Approximations associated with the market values over the forecast period are based on empirical research and data collected through both primary and secondary sources. This might help readers to understand the strengths, opportunities, challenges and perceived threats of the market.

Based on Classification, each type is studied as Sales, Market Share (%), Revenue (Million USD), Price, Gross Margin and more similar information. The report can help to realize the market and strategize for business expansion accordingly. In the strategy analysis, it gives insights from marketing channel and market positioning to potential growth strategies, providing in-depth analysis for new entrants or exists competitors in the Gene Therapy For Rare Disease industry.

The Gene Therapy For Rare Disease Market report wraps:

There are 13 Chapters to thoroughly display the Gene Therapy For Rare Disease market. This report included the analysis of market overview, market characteristics, industry chain, competition landscape, historical and future data by types, applications and regions.

In the end, The objective of the market research report is the current status of the market and in accordance classifies it into a few objects. The report takes into consideration the first market players in every area from over the globe.

Note In order to provide more accurate market forecast, all our reports will be updated before delivery by considering the impact of COVID-19.

Continue reading here:
Gene Therapy For Rare Disease Market | Identify Which Types of Companies Could Potentially Benefit or Loose out From the Impact of COVID-19 - Cole of...

Increasing number of new drug launches, rise in chronic disease burden, and increasing government support for the pharmaceutical and biotechnology industries will also aid the growth of this market.

Viral inactivation testing is necessary by regulatory authorities for investigational new drug (IND) submission and is mainly critical in process development for biologicals including tissue and tissue products, stem cell products, cellular and gene therapy products, blood and blood products, and vaccine and therapeutics. The major drivers for the market are rapid growth in pharmaceutical and biotechnology industries and strong trend of R&D investments in life sciences industry.

The global viral inactivation market poised to grow at a CAGR of 12.3%, to reach USD 573.0 Million.

Viral inactivation is important and mandatory step in the manufacturing process of biological products to remove or inactivate potential contaminant viruses. These biological products are used for the treatment and diagnostic purposes in humans.

As the disease burden in the world is increasing the demand for the medicines is increasing hence the production of biologicals, injectables., is on a rise. many of pharmaceutical and biotechnology firms are gearing to cater to the growing demands of medicines.

This aids to the growth of market.

Download PDF Brochure:www.marketsandmarkets.com/pdfdown=121427017

The globalviral inactivation market is segmented based on method, product, application, end user, and geography.

Market Segmentation in depth:

Based on end user this market is categorized into pharmaceutical and biotechnology companies, contract research organizations, academic research institutes, and other end users. Other end user segment primarily includes cell banks, small cell culture laboratories and consultants, microbiology laboratories, immunology laboratories, molecular laboratories, animal facilities, toxicology laboratories, and media/sera manufacturers.

The application segments included in the report are blood & blood products, cellular & gene therapy products, stem cell products, tissue & tissue products, and vaccines and therapeutics. Based on geography the market is segmented into North America, Europe, Asia and Rest of the World (RoW).

Report: http://www.marketsandmarkets.com/request=121427017

Based on geography the viral inactivation market is segmented into North America, Europe, Asia and Rest of the World (RoW).

The major players in theViral Inactivation Marketinclude Clean Cells (France), Charles River Laboratories International, Inc. (U.S.), Danaher Corporation (U.S.), Merck KGaA (Germany), Parker Hannifin (U.S.), Rad Source Technologies (U.S.), Sartorius AG (Germany), SGS S.A.

(Switzerland), Texcell, Inc. (France), Viral Inactivated Plasma Systems SA (Switzerland), and WuXi PharmaTech (Cayman) Inc. (China).

This email address is being protected from spambots. You need JavaScript enabled to view it.

Original post:
Viral Inactivation Market estimated to grow according to forecasts - WhaTech Technology and Markets News

The research study presented here is an intelligent take on the global Human Interleukin-2 (IL-2) Market that explains important aspects such as competition, segmentation, and regional growth in great detail. Accuracy and preciseness are two of the key features of the report that reflect its authenticity. The authors of the report have focused on SWOT analysis, Porters Five Forces analysis, and PESTLE analysis of the global Human Interleukin-2 (IL-2) market. In addition, they have concentrated on qualitative and quantitative analyses to help with a deep understanding of the global Human Interleukin-2 (IL-2) market. Furthermore, the report provides powerful suggestions and recommendations to help players create strong growth strategies and ensure impressive sales in the global Human Interleukin-2 (IL-2) market.

Some of the influential Key Player operated in the report are: Roche BMS Schering-Plough AbbVie Inc Alkermes Plc APT Therapeutics Mabtech Limited Philogen Sinopharm Jiangsu Jinsili Pharmaceutical Shenyang Sansheng Pharmaceutical Beijing Shuanglu Pharmaceutical Shandong Quangang Pharmaceutical Shanghai Huaxin Biological high-tech Beijing Yuance Pharmaceutical Beijing Sihuan Biological Pharmaceutical Beijing Sanyuan Gene Pharmaceutical Shenzhen Kexing Biological Engineering Shanghai Sanwei Biotechnology Xiamen Tebao Biological Engineering Chengdu huashen Biotechnology Shanghai Pharma Group Guangdong Xinghao Pharmaceutical Market Segment by Type 50000 U 100000 U 200000 U 500000 U 1 Million U 2 Million U Market Segment by Application Recombinant Interferon Recombinant Interleukin Natural Biological Products Poison Immune Gene Therapy Monoclonal Antibody Research Methodology To compile the detailed study of the global Human Interleukin-2 (IL-2) market, a robust research methodology has been adopted that aids in determining the key insights and also evaluates the growth prospects of the Human Interleukin-2 (IL-2) market. QY Research analysts have conducted in-depth primary and secondary research to obtain crucial insights into the Human Interleukin-2 (IL-2) market. To carry out secondary research, the analysts have collected the information through company annual reports, journals, company press releases, and paid databases that were referred to gain and identify better opportunities in the global market.

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As part of competitive analysis, the research study includes exhaustive company profiling of leading players of the global Human Interleukin-2 (IL-2) market. All of the segments studied in the report are analyzed based on different factors such as market share, revenue, and CAGR. The analysts have also thoroughly analyzed different regions such as North America, Europe, and the Asia Pacific on the basis of production, revenue, and sales in the global Human Interleukin-2 (IL-2) market. The researchers used advanced primary and secondary research methodologies and tools for preparing this report on the global Human Interleukin-2 (IL-2) market.

Segmentation by Type:

50000 U, 100000 U, 200000 U, 500000 U, 1 Million U, 2 Million U Market

Segmentation by Application:

Recombinant Interferon, Recombinant Interleukin, Natural Biological Products, Poison Immune, Gene Therapy, Monoclonal Antibody Research Methodology To compile the detailed study of the

Table of Contents

Table of Contents1 Report Overview1.1 Research Scope1.2 Top Human Interleukin-2 (IL-2) Manufacturers Covered: Ranking by Revenue1.3 Market Segment by Type1.3.1 Global Human Interleukin-2 (IL-2) Market Size by Type: 2015 VS 2020 VS 2026 (US$ Million)1.3.2 50000 U1.3.3 100000 U1.3.4 200000 U1.3.5 500000 U1.3.6 1 Million U1.3.7 2 Million U1.4 Market Segment by Application1.4.1 Global Human Interleukin-2 (IL-2) Consumption by Application: 2015 VS 2020 VS 20261.4.2 Recombinant Interferon1.4.3 Recombinant Interleukin1.4.4 Natural Biological Products1.4.5 Poison Immune1.4.6 Gene Therapy1.4.7 Monoclonal Antibody1.5 Study Objectives1.6 Years Considered 2 Global Market Perspective2.1 Global Human Interleukin-2 (IL-2) Revenue (2015-2026)2.1.1 Global Human Interleukin-2 (IL-2) Revenue (2015-2026)2.1.2 Global Human Interleukin-2 (IL-2) Sales (2015-2026)2.2 Human Interleukin-2 (IL-2) Market Size across Key Geographies Worldwide: 2015 VS 2020 VS 20262.2.1 Global Human Interleukin-2 (IL-2) Sales by Regions (2015-2020)2.2.2 Global Human Interleukin-2 (IL-2) Revenue by Regions (2015-2020)2.3 Global Top Human Interleukin-2 (IL-2) Regions (Countries) Ranking by Market Size2.4 Human Interleukin-2 (IL-2) Industry Trends2.4.1 Human Interleukin-2 (IL-2) Market Top Trends2.4.2 Market Drivers2.4.3 Human Interleukin-2 (IL-2) Market Challenges2.4.4 Porters Five Forces Analysis2.4.5 Primary Interviews with Key Human Interleukin-2 (IL-2) Players: Views for Future3 Competitive Landscape by Manufacturers3.1 Global Top Human Interleukin-2 (IL-2) Manufacturers by Sales (2015-2020)3.1.1 Global Human Interleukin-2 (IL-2) Sales by Manufacturers (2015-2020)3.1.2 Global Human Interleukin-2 (IL-2) Sales Market Share by Manufacturers (2015-2020)3.1.3 Global 5 and 10 Largest Manufacturers by Human Interleukin-2 (IL-2) Sales in 20193.2 Global Top Manufacturers Human Interleukin-2 (IL-2) by Revenue3.2.1 Global Human Interleukin-2 (IL-2) Revenue by Manufacturers (2015-2020)3.2.2 Global Human Interleukin-2 (IL-2) Revenue Share by Manufacturers (2015-2020)3.2.3 Global Human Interleukin-2 (IL-2) Market Concentration Ratio (CR5 and HHI)3.3 Global Top Manufacturers by Company Type (Tier 1, Tier 2 and Tier 3) (based on the Revenue in Human Interleukin-2 (IL-2) as of 2019)3.4 Global Human Interleukin-2 (IL-2) Average Selling Price (ASP) by Manufacturers3.5 Key Manufacturers Human Interleukin-2 (IL-2) Plants/Factories Distribution and Area Served3.6 Date of Key Manufacturers Enter into Human Interleukin-2 (IL-2) Market3.7 Key Manufacturers Human Interleukin-2 (IL-2) Product Offered3.8 Mergers & Acquisitions, Expansion Plans4 Market Size by Type4.1 Global Human Interleukin-2 (IL-2) Historic Market Review by Type (2015-2020)4.1.2 Global Human Interleukin-2 (IL-2) Sales Market Share by Type (2015-2020)4.1.3 Global Human Interleukin-2 (IL-2) Revenue Market Share by Type (2015-2020)4.1.4 Human Interleukin-2 (IL-2) Price by Type (2015-2020)4.1 Global Human Interleukin-2 (IL-2) Market Estimates and Forecasts by Type (2021-2026)4.2.2 Global Human Interleukin-2 (IL-2) Sales Forecast by Type (2021-2026)4.2.3 Global Human Interleukin-2 (IL-2) Revenue Forecast by Type (2021-2026)4.2.4 Human Interleukin-2 (IL-2) Price Forecast by Type (2021-2026)5 Global Human Interleukin-2 (IL-2) Market Size by Application5.1 Global Human Interleukin-2 (IL-2) Historic Market Review by Application (2015-2020)5.1.2 Global Human Interleukin-2 (IL-2) Sales Market Share by Application (2015-2020)5.1.3 Global Human Interleukin-2 (IL-2) Revenue Market Share by Application (2015-2020)5.1.4 Human Interleukin-2 (IL-2) Price by Application (2015-2020)5.2 Global Human Interleukin-2 (IL-2) Market Estimates and Forecasts by Application (2021-2026)5.2.2 Global Human Interleukin-2 (IL-2) Sales Forecast by Application (2021-2026)5.2.3 Global Human Interleukin-2 (IL-2) Revenue Forecast by Application (2021-2026)5.2.4 Human Interleukin-2 (IL-2) Price Forecast by Application (2021-2026)6 North America6.1 North America Human Interleukin-2 (IL-2) Breakdown Data by Company6.2 North America Human Interleukin-2 (IL-2) Breakdown Data by Type6.3 North America Human Interleukin-2 (IL-2) Breakdown Data by Application6.4 North America Human Interleukin-2 (IL-2) Breakdown Data by Countries6.4.1 North America Human Interleukin-2 (IL-2) Sales by Countries6.4.2 North America Human Interleukin-2 (IL-2) Revenue by Countries6.4.3 U.S.6.4.4 Canada7 Europe7.1 Europe Human Interleukin-2 (IL-2) Breakdown Data by Company7.2 Europe Human Interleukin-2 (IL-2) Breakdown Data by Type7.3 Europe Human Interleukin-2 (IL-2) Breakdown Data by Application7.4 Europe Human Interleukin-2 (IL-2) Breakdown Data by Countries7.4.1 Europe Human Interleukin-2 (IL-2) Sales by Countries7.4.2 Europe Human Interleukin-2 (IL-2) Revenue by Countries7.4.3 Germany7.4.4 France7.4.5 U.K.7.4.6 Italy7.4.7 Russia8 Asia Pacific8.1 Asia Pacific Human Interleukin-2 (IL-2) Breakdown Data by Company8.2 Asia Pacific Human Interleukin-2 (IL-2) Breakdown Data by Type8.3 Asia Pacific Human Interleukin-2 (IL-2) Breakdown Data by Application8.4 Asia Pacific Human Interleukin-2 (IL-2) Breakdown Data by Regions8.4.1 Asia Pacific Human Interleukin-2 (IL-2) Sales by Regions8.4.2 Asia Pacific Human Interleukin-2 (IL-2) Revenue by Regions8.4.3 China8.4.4 Japan8.4.5 South Korea8.4.6 India8.4.7 Australia8.4.8 Taiwan8.4.9 Indonesia8.4.10 Thailand8.4.11 Malaysia8.4.12 Philippines8.4.13 Vietnam9 Latin America9.1 Latin America Human Interleukin-2 (IL-2) Breakdown Data by Company9.2 Latin America Human Interleukin-2 (IL-2) Breakdown Data by Type9.3 Latin America Human Interleukin-2 (IL-2) Breakdown Data by Application9.4 Latin America Human Interleukin-2 (IL-2) Breakdown Data by Countries9.4.1 Latin America Human Interleukin-2 (IL-2) Sales by Countries9.4.2 Latin America Human Interleukin-2 (IL-2) Revenue by Countries9.4.3 Mexico9.4.4 Brazil9.4.5 Argentina10 Middle East and Africa10.1 Middle East and Africa Human Interleukin-2 (IL-2) Breakdown Data by Type10.2 Middle East and Africa Human Interleukin-2 (IL-2) Breakdown Data by Application10.3 Middle East and Africa Human Interleukin-2 (IL-2) Breakdown Data by Countries10.3.1 Middle East and Africa Human Interleukin-2 (IL-2) Sales by Countries10.3.2 Middle East and Africa Human Interleukin-2 (IL-2) Revenue by Countries10.3.3 Turkey10.3.4 Saudi Arabia10.3.5 U.A.E11 Company Profiles11.1 Roche11.1.1 Roche Corporation Information11.1.2 Roche Business Overview and Total Revenue (2019 VS 2018)11.1.3 Roche Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.1.4 Roche Human Interleukin-2 (IL-2) Products and Services11.1.5 Roche SWOT Analysis11.1.6 Roche Recent Developments11.2 BMS11.2.1 BMS Corporation Information11.2.2 BMS Business Overview and Total Revenue (2019 VS 2018)11.2.3 BMS Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.2.4 BMS Human Interleukin-2 (IL-2) Products and Services11.2.5 BMS SWOT Analysis11.2.6 BMS Recent Developments11.3 Schering-Plough11.3.1 Schering-Plough Corporation Information11.3.2 Schering-Plough Business Overview and Total Revenue (2019 VS 2018)11.3.3 Schering-Plough Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.3.4 Schering-Plough Human Interleukin-2 (IL-2) Products and Services11.3.5 Schering-Plough SWOT Analysis11.3.6 Schering-Plough Recent Developments11.4 AbbVie Inc11.4.1 AbbVie Inc Corporation Information11.4.2 AbbVie Inc Business Overview and Total Revenue (2019 VS 2018)11.4.3 AbbVie Inc Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.4.4 AbbVie Inc Human Interleukin-2 (IL-2) Products and Services11.4.5 AbbVie Inc SWOT Analysis11.4.6 AbbVie Inc Recent Developments11.5 Alkermes Plc11.5.1 Alkermes Plc Corporation Information11.5.2 Alkermes Plc Business Overview and Total Revenue (2019 VS 2018)11.5.3 Alkermes Plc Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.5.4 Alkermes Plc Human Interleukin-2 (IL-2) Products and Services11.5.5 Alkermes Plc SWOT Analysis11.5.6 Alkermes Plc Recent Developments11.6 APT Therapeutics11.6.1 APT Therapeutics Corporation Information11.6.2 APT Therapeutics Business Overview and Total Revenue (2019 VS 2018)11.6.3 APT Therapeutics Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.6.4 APT Therapeutics Human Interleukin-2 (IL-2) Products and Services11.6.5 APT Therapeutics SWOT Analysis11.6.6 APT Therapeutics Recent Developments11.7 Mabtech Limited11.7.1 Mabtech Limited Corporation Information11.7.2 Mabtech Limited Business Overview and Total Revenue (2019 VS 2018)11.7.3 Mabtech Limited Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.7.4 Mabtech Limited Human Interleukin-2 (IL-2) Products and Services11.7.5 Mabtech Limited SWOT Analysis11.7.6 Mabtech Limited Recent Developments11.8 Philogen11.8.1 Philogen Corporation Information11.8.2 Philogen Business Overview and Total Revenue (2019 VS 2018)11.8.3 Philogen Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.8.4 Philogen Human Interleukin-2 (IL-2) Products and Services11.8.5 Philogen SWOT Analysis11.8.6 Philogen Recent Developments11.9 Sinopharm11.9.1 Sinopharm Corporation Information11.9.2 Sinopharm Business Overview and Total Revenue (2019 VS 2018)11.9.3 Sinopharm Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.9.4 Sinopharm Human Interleukin-2 (IL-2) Products and Services11.9.5 Sinopharm SWOT Analysis11.9.6 Sinopharm Recent Developments11.10 Jiangsu Jinsili Pharmaceutical11.10.1 Jiangsu Jinsili Pharmaceutical Corporation Information11.10.2 Jiangsu Jinsili Pharmaceutical Business Overview and Total Revenue (2019 VS 2018)11.10.3 Jiangsu Jinsili Pharmaceutical Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.10.4 Jiangsu Jinsili Pharmaceutical Human Interleukin-2 (IL-2) Products and Services11.10.5 Jiangsu Jinsili Pharmaceutical SWOT Analysis11.10.6 Jiangsu Jinsili Pharmaceutical Recent Developments11.11 Shenyang Sansheng Pharmaceutical11.11.1 Shenyang Sansheng Pharmaceutical Corporation Information11.11.2 Shenyang Sansheng Pharmaceutical Business Overview and Total Revenue (2019 VS 2018)11.11.3 Shenyang Sansheng Pharmaceutical Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.11.4 Shenyang Sansheng Pharmaceutical Human Interleukin-2 (IL-2) Products and Services11.11.5 Shenyang Sansheng Pharmaceutical SWOT Analysis11.11.6 Shenyang Sansheng Pharmaceutical Recent Developments11.12 Beijing Shuanglu Pharmaceutical11.12.1 Beijing Shuanglu Pharmaceutical Corporation Information11.12.2 Beijing Shuanglu Pharmaceutical Business Overview and Total Revenue (2019 VS 2018)11.12.3 Beijing Shuanglu Pharmaceutical Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.12.4 Beijing Shuanglu Pharmaceutical Human Interleukin-2 (IL-2) Products and Services11.12.5 Beijing Shuanglu Pharmaceutical SWOT Analysis11.12.6 Beijing Shuanglu Pharmaceutical Recent Developments11.13 Shandong Quangang Pharmaceutical11.13.1 Shandong Quangang Pharmaceutical Corporation Information11.13.2 Shandong Quangang Pharmaceutical Business Overview and Total Revenue (2019 VS 2018)11.13.3 Shandong Quangang Pharmaceutical Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.13.4 Shandong Quangang Pharmaceutical Human Interleukin-2 (IL-2) Products and Services11.13.5 Shandong Quangang Pharmaceutical SWOT Analysis11.13.6 Shandong Quangang Pharmaceutical Recent Developments11.14 Shanghai Huaxin Biological high-tech11.14.1 Shanghai Huaxin Biological high-tech Corporation Information11.14.2 Shanghai Huaxin Biological high-tech Business Overview and Total Revenue (2019 VS 2018)11.14.3 Shanghai Huaxin Biological high-tech Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.14.4 Shanghai Huaxin Biological high-tech Human Interleukin-2 (IL-2) Products and Services11.14.5 Shanghai Huaxin Biological high-tech SWOT Analysis11.14.6 Shanghai Huaxin Biological high-tech Recent Developments11.15 Beijing Yuance Pharmaceutical11.15.1 Beijing Yuance Pharmaceutical Corporation Information11.15.2 Beijing Yuance Pharmaceutical Business Overview and Total Revenue (2019 VS 2018)11.15.3 Beijing Yuance Pharmaceutical Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.15.4 Beijing Yuance Pharmaceutical Human Interleukin-2 (IL-2) Products and Services11.15.5 Beijing Yuance Pharmaceutical SWOT Analysis11.15.6 Beijing Yuance Pharmaceutical Recent Developments11.16 Beijing Sihuan Biological Pharmaceutical11.16.1 Beijing Sihuan Biological Pharmaceutical Corporation Information11.16.2 Beijing Sihuan Biological Pharmaceutical Business Overview and Total Revenue (2019 VS 2018)11.16.3 Beijing Sihuan Biological Pharmaceutical Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.16.4 Beijing Sihuan Biological Pharmaceutical Human Interleukin-2 (IL-2) Products and Services11.16.5 Beijing Sihuan Biological Pharmaceutical SWOT Analysis11.16.6 Beijing Sihuan Biological Pharmaceutical Recent Developments11.17 Beijing Sanyuan Gene Pharmaceutical11.17.1 Beijing Sanyuan Gene Pharmaceutical Corporation Information11.17.2 Beijing Sanyuan Gene Pharmaceutical Business Overview and Total Revenue (2019 VS 2018)11.17.3 Beijing Sanyuan Gene Pharmaceutical Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.17.4 Beijing Sanyuan Gene Pharmaceutical Human Interleukin-2 (IL-2) Products and Services11.17.5 Beijing Sanyuan Gene Pharmaceutical SWOT Analysis11.17.6 Beijing Sanyuan Gene Pharmaceutical Recent Developments11.18 Shenzhen Kexing Biological Engineering11.18.1 Shenzhen Kexing Biological Engineering Corporation Information11.18.2 Shenzhen Kexing Biological Engineering Business Overview and Total Revenue (2019 VS 2018)11.18.3 Shenzhen Kexing Biological Engineering Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.18.4 Shenzhen Kexing Biological Engineering Human Interleukin-2 (IL-2) Products and Services11.18.5 Shenzhen Kexing Biological Engineering SWOT Analysis11.18.6 Shenzhen Kexing Biological Engineering Recent Developments11.19 Shanghai Sanwei Biotechnology11.19.1 Shanghai Sanwei Biotechnology Corporation Information11.19.2 Shanghai Sanwei Biotechnology Business Overview and Total Revenue (2019 VS 2018)11.19.3 Shanghai Sanwei Biotechnology Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.19.4 Shanghai Sanwei Biotechnology Human Interleukin-2 (IL-2) Products and Services11.19.5 Shanghai Sanwei Biotechnology SWOT Analysis11.19.6 Shanghai Sanwei Biotechnology Recent Developments11.20 Xiamen Tebao Biological Engineering11.20.1 Xiamen Tebao Biological Engineering Corporation Information11.20.2 Xiamen Tebao Biological Engineering Business Overview and Total Revenue (2019 VS 2018)11.20.3 Xiamen Tebao Biological Engineering Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.20.4 Xiamen Tebao Biological Engineering Human Interleukin-2 (IL-2) Products and Services11.20.5 Xiamen Tebao Biological Engineering SWOT Analysis11.20.6 Xiamen Tebao Biological Engineering Recent Developments11.21 Chengdu huashen Biotechnology11.21.1 Chengdu huashen Biotechnology Corporation Information11.21.2 Chengdu huashen Biotechnology Business Overview and Total Revenue (2019 VS 2018)11.21.3 Chengdu huashen Biotechnology Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.21.4 Chengdu huashen Biotechnology Human Interleukin-2 (IL-2) Products and Services11.21.5 Chengdu huashen Biotechnology SWOT Analysis11.21.6 Chengdu huashen Biotechnology Recent Developments11.22 Shanghai Pharma Group11.22.1 Shanghai Pharma Group Corporation Information11.22.2 Shanghai Pharma Group Business Overview and Total Revenue (2019 VS 2018)11.22.3 Shanghai Pharma Group Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.22.4 Shanghai Pharma Group Human Interleukin-2 (IL-2) Products and Services11.22.5 Shanghai Pharma Group SWOT Analysis11.22.6 Shanghai Pharma Group Recent Developments11.23 Guangdong Xinghao Pharmaceutical11.23.1 Guangdong Xinghao Pharmaceutical Corporation Information11.23.2 Guangdong Xinghao Pharmaceutical Business Overview and Total Revenue (2019 VS 2018)11.23.3 Guangdong Xinghao Pharmaceutical Human Interleukin-2 (IL-2) Sales, Revenue, Average Selling Price (ASP) and Gross Margin (2015-2020)11.23.4 Guangdong Xinghao Pharmaceutical Human Interleukin-2 (IL-2) Products and Services11.23.5 Guangdong Xinghao Pharmaceutical SWOT Analysis11.23.6 Guangdong Xinghao Pharmaceutical Recent Developments12 Supply Chain and Sales Channels Analysis12.1 Supply Chain Analysis12.2 Sales Channels Analysis12.2.1 Human Interleukin-2 (IL-2) Sales Channels12.2.2 Human Interleukin-2 (IL-2) Distributors12.3 Human Interleukin-2 (IL-2) Customers13 Estimates and Projections by Regions (2021-2026)13.1 Global Human Interleukin-2 (IL-2) Sales Forecast (2021-2026)13.1.1 Global Human Interleukin-2 (IL-2) Sales Forecast by Regions (2021-2026)13.1.2 Global Human Interleukin-2 (IL-2) Revenue Forecast by Regions (2021-2026)13.2 North America Market Size Forecast (2021-2026)13.2.1 North America Human Interleukin-2 (IL-2) Sales Forecast (2021-2026)13.2.2 North America Human Interleukin-2 (IL-2) Revenue Forecast (2021-2026)13.2.3 North America Human Interleukin-2 (IL-2) Size Forecast by County (2021-2026)13.3 Europe Market Size Forecast (2021-2026)13.3.1 Europe Human Interleukin-2 (IL-2) Sales Forecast (2021-2026)13.3.2 Europe Human Interleukin-2 (IL-2) Revenue Forecast (2021-2026)13.3.3 Europe Human Interleukin-2 (IL-2) Size Forecast by County (2021-2026)13.4 Asia Pacific Market Size Forecast (2021-2026)13.4.1 Asia Pacific Human Interleukin-2 (IL-2) Sales Forecast (2021-2026)13.4.2 Asia Pacific Human Interleukin-2 (IL-2) Revenue Forecast (2021-2026)13.4.3 Asia Pacific Human Interleukin-2 (IL-2) Size Forecast by Region (2021-2026)13.5 Latin America Market Size Forecast (2021-2026)13.5.1 Latin America Human Interleukin-2 (IL-2) Sales Forecast (2021-2026)13.5.2 Latin America Human Interleukin-2 (IL-2) Revenue Forecast (2021-2026)13.5.3 Latin America Human Interleukin-2 (IL-2) Size Forecast by County (2021-2026)13.6 Middle East and Africa Market Forecast13.6.1 Middle East and Africa Human Interleukin-2 (IL-2) Sales Forecast (2021-2026)13.6.2 Middle East and Africa Human Interleukin-2 (IL-2) Revenue Forecast (2021-2026)13.6.3 Middle East and Africa Human Interleukin-2 (IL-2) Size Forecast by County (2021-2026)14 Research Findings and Conclusion15 Appendix15.1 Research Methodology15.1.1 Methodology/Research Approach15.1.2 Data Source15.2 Author Details15.3 Disclaimer

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Report Objectives

To carefully analyze and estimate the size of the global Human Interleukin-2 (IL-2) market. To clearly segment the global Human Interleukin-2 (IL-2) market and estimate the market size of the segments. To provide details about key strategies adopted by leading players of the global Human Interleukin-2 (IL-2) market. To help readers understand the current and future market scenarios. To provide information about the latest trends of the global Human Interleukin-2 (IL-2) market and its key segments. To assess the contribution of each region or country to the global Human Interleukin-2 (IL-2) market. To provide information on important drivers, restraints, and opportunities of the global Human Interleukin-2 (IL-2) market. To accurately calculate the market shares of key segments, regions, and companies in the global Human Interleukin-2 (IL-2) market.

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Human Interleukin-2 (IL-2) Market Study for 2020 to 2026 Providing Information on Key Players, Growth Drivers and Industry Challenges - Cole of Duty

According to a report titledGlobalGene TherapyMarketpublished byFior Markets, the market explores new growth possibilities from 2020 to2025. The research report affords a number one review of the business enterprise inclusive of characterizations, companies, displays and organization chain shape. This recently published take a look at statistics including key segmentation of the globalGene Therapymarket on the basis of product type, application, and geography (country/region). The market evaluation is recommended with market trends research, evaluation additionally covers each the winning and earlier cutting-edge market developments, drivers and barriers faced through the market.

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Major market leaders included:Spark Therapeutics LLC, Bluebird Bio, UniQure N.V., Juno Therapeutics, GlaxoSmithKline, Chiesi Farmaceutici S.p.A., Bristol Myers Squibb, Celgene Corporation, Human Stem Cell Institute, Voyager Therapeutics, Shire Plc, Sangamo Biosciences, Dimension Therapeutics and others.

The market has been segmented into regions to identify growth opportunities market such as:North America, Europe, Asia Pacific, South America, and the Middle East and Africa.

The competitive landscape chapter of the globalGene Therapymarket report presents key information about market players such as company overview, total revenue (financials), market potential, global presence, sales and revenue, market share, prices, production sites and facilities, products offered, and strategies adopted. Furthermore, the statistics and data collected are graphically presented in the globalGene Therapymarket size and trends research report. It highlights the command and drivers influencing the market. This market research report estimates the size of the market, sales, price, revenue, gross margin and market share, cost structure and growth rate. It additionally throws light on their crucial business aspects such as production, areas of operation, and product portfolio.

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View More Information @https://www.marketwatch.com/press-release/global-gene-therapy-market-2020-overview-manufacturing-analysis-development-status-competitive-analysis-to-2025-2020-04-21

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Gene Therapy Market 2020 Rising Recognition with its Global Trends by 2025 - Research Columnist

DetailsCategory: DNA RNA and CellsPublished on Monday, 27 April 2020 17:10Hits: 524

BOSTON, MA, USAandLONDON, UK I April 27, 2020 IOrchard Therapeutics(Nasdaq: ORTX), a global gene therapy leader, today announced that the first patient has been dosed in an open-label, proof-of-concept investigational study of OTL-201, an ex vivo autologous hematopoietic stem cell (HSC) gene therapy for the treatment of mucopolysaccharidosis type IIIA (MPS-IIIA). The study is designed to evaluate safety, tolerability and clinical efficacy and is intended to enroll up to five patients between three months and 24 months of age who will be followed for three years. The study also contains a number of key secondary outcome measures such as overall survival, cognition and behavior to help inform future clinical development of HSC gene therapy in this indication.

MPS-IIIA, also known as Sanfilippo syndrome type A, is a rare, inherited neurometabolic disorder caused by genetic mutations that leads to the buildup of sugar molecules called mucopolysaccharides in the body, resulting in progressive intellectual disability and loss of motor function. Children born with MPS-IIIA rarely live past adolescence or early adulthood, and no approved therapies currently exist to treat the disease.

I am very encouraged that we, together with our research and clinical collaborators in Manchester, could achieve this important milestone in our efforts to develop a gene therapy for MPS-IIIA despite the current, challenging global health circumstances, saidBobby Gaspar, M.D., Ph.D., chief executive officer of Orchard. It is a testament to the dedication of our collective teams and underscores the truly dire, life-limiting nature of the disease for affected children and their families. This study adds to Orchards clinical pipeline of HSC gene therapies for the treatment of severe neurometabolic disorders and further demonstrates the potential of our platform approach.

About MPS-IIIAMucopolysaccharidosis type IIIA (MPS-IIIA, also known as Sanfilippo syndrome type A) is a rare and life-threatening metabolic disease. People with MPS-IIIA are born with a mutation in theN-sulphoglucosamine sulphohydrolase (SGSH)gene, which, when healthy, helps the body break down sugar molecules called mucopolysaccharides. The buildup of mucopolysaccharides in the brain and other tissues leads to intellectual disability and loss of motor function. MPS-IIIA occurs in approximately one in every 100,000 live births. Life expectancy of children born with MPS-IIIA is estimated to be between 10-25 years.

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

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

SOURCE: Orchard Therapeutics

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Orchard Therapeutics Announces First Patient Dosed with OTL-201 Gene Therapy in Proof-of-Concept Clinical Trial for Sanfilippo Syndrome (MPS-IIIA) |...

ELEVECTA(R) takes viral vector manufacturing to the next level to serve the rapidly growing market of gene therapy applications

Growing serum-free in suspension, ELEVECTA(R) producer cell lines enable fully scalable high-performance AAV vector production based on standardized processes widely used in industry for monoclonal antibody production

With all components stably integrated into one cell, the ELEVECTA(R) technology provides large scale AAV production without expensive cGMP-grade plasmids and complex transient transfection steps

COLOGNE, GERMANY / ACCESSWIRE / April 28, 2020 / CEVEC Pharmaceuticals GmbH (CEVEC), the leading provider of high-performance cell technology for the manufacturing of advanced biotherapeutics from research to production scale, today announced the launch of the ELEVECTA(R) platform, a unique technology to manufacture AAV (Adeno-associated viral) vectors at large scale. With the launch, CEVEC is the first and only provider on the market to offer AAV gene therapy vector manufacturing technology on the basis of fully stable producer cell lines.

The patent-protected technology has been proven by CEVEC in pilot projects with several partners and is now commercially available for Pharma and Biotech companies running gene therapy programs from late research to all clinical phases.

ELEVECTA(R) takes AAV vector manufacturing to the next level

With the ELEVECTA(R) technology, CEVEC has taken a unique approach based on producer cell lines, which have all necessary elements for AAV production stably integrated in one cell. Up to now, manufacturing of AAV - the most widely used vector for in vivo gene therapy - has required expensive transfection reagents and cGMP-grade plasmids. ELEVECTA(R) now overcomes these limitations and enables efficient, high-performance AAV production from one cell line in consistent quality. The technology can easily be implemented in bioprocess development and large-scale GMP manufacturing facilities running standard suspension bioreactor equipment widely used in industry for monoclonal antibody and recombinant protein production.

Story continues

"The launch of ELEVECTA(R) forms a milestone on the way into a new era of viral vector manufacturing as it provides a solution for one of the major challenges in gene therapy development. By closing the production gap and providing the necessary quantities of viral vectors, it paves the way to address more common indications such as Alzheimer's, Parkinson's, or Rheumatoid Arthritis as well as therapies that require larger doses," said Nicole Faust, CEO of CEVEC Pharmaceuticals. "With this launch, we provide pharmaceutical and biotechnology companies with the opportunity to benefit right from the beginning from what we believe is the future for viral vector production. The superior features in terms of scalability, production efficiency and robustness of our stable producer cell lines position CEVEC at the leading edge in the rapidly growing field of gene therapy vector manufacturing."

ELEVECTA(R) closes the production gap in gene therapy viral vector production

Gene therapy is considered to be the most effective and often only treatment option for many severe and life-threatening diseases. Moreover, in a growing number of indications, gene therapies for the first time offer the opportunity to cure a disease. In addition, as gene therapy is moving from rare and ultra-rare to more common indications with larger patient numbers and systemic treatments, larger amounts of vector material are required. Consequently, production processes are needed that are fit for this purpose, enabling the manufacture of the necessary quantities of viral vectors with high yield and consistent quality.

ELEVECTA(R) - A concept to match the customers' needs

CEVEC's newly launched ELEVECTA(R) technology overcomes the limitations of existing manufacturing methods such as restricted scalability, time-consuming and cost-intensive material sourcing and complex production processes. ELEVECTA(R) technology is based on a patent-protected, fully documented human suspension cell line developed and optimized by CEVEC.

The ELEVECTA(R) technology works for any combination of serotype-specific capsid and therapeutic gene of interest. The stable integration of these components into the cell results in cell lines designed to produce highly functional AAV vectors. Custom-made ELEVECTA(R) Producer Cell Lines are available as research cell banks or as fully tested cGMP Master Cell Banks for manufacturing of clinical and commercial material.

CEVEC launches the technology in various formats matching the needs of customers in different development phases, with focus on gene therapy programs from late research to any clinical phases. CEVEC offers cell line development services to match the specific needs of gene therapy programs and various license packages from research and development to commercial manufacturing.

Further information:

To get a deeper insight into the ELEVECTA(R) technology register for a webinar on May 5th, at 5:00 p.m. CEST (11:00 a.m. EST) featuring CEVEC's CSO, Dr. Silke Wissing, talking about "Stable AAV producer cell lines: elevating vector manufacturing" and/or visit the ELEVECTA(R) web page.

In addition, you can meet CEVEC's management at the 23rd ASGCT virtual Annual Meeting taking place from May 12 - 15, 2020. During this event, interested parties can listen to the ELEVECTA(R) poster presentations and visit the CEVEC virtual booth. Please request a virtual meeting through the ASGCT webpage or directly contact CEVEC under bizdev@cevec.com.

About CEVEC:

CEVEC is a leading provider of high-performance cell technology for the manufacturing of advanced bio-therapeutics from R&D to manufacturing scale. The company's product portfolio comprises platform technologies for gene therapy viral vectors (AAV, Adenoviral vectors, Lentiviral vectors,), vaccines and complex recombinant proteins. With the ELEVECTA(R) Technology, CEVEC offers a unique solution for large-scale production of AAV vectors using producer cell lines with all necessary components stably integrated into the cell. The technology is based on suspension cells and does not require any expensive transfection reagents and cGMP plasmids. CEVEC's CAP(R) Technology based on human suspension cells is the ideal production platform for RCA-free Adenoviral vectors, Lentiviral vectors, Oncolytic viruses, Viral vaccines and exosomes. With the CAP-Go(R) Technology CEVEC provides a solution to the increasing need for recombinant production of complex and highly glycosylated protein molecules, including laminins, coagulation factors and plasma proteins.

For more information, please visit the Company's website.

Follow CEVEC on LinkedIn and Twitter.

Contact:

SOURCE: CEVEC Pharmaceuticals GmbH

View source version on accesswire.com: https://www.accesswire.com/587337/CEVEC-Announces-the-Launch-of-the-ELEVECTAR-Platform--the-Stable-Producer-Cell-Line-Technology-for-AAV-Gene-Therapy-Vectors

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CEVEC Announces the Launch of the ELEVECTA(R) Platform - the Stable Producer Cell Line Technology for AAV Gene Therapy Vectors - Yahoo Finance

Affinia Therapeutics proprietary AAV vector technology to be used in Vertexs genetic therapy efforts with focus on Duchenne muscular dystrophy, myotonic dystrophy type 1 and cystic fibrosis

BOSTON & WALTHAM, Mass.--(BUSINESS WIRE)-- Vertex PharmaceuticalsIncorporated (Nasdaq:VRTX) and Affinia Therapeutics announced today that the two companies have entered into a strategic research collaboration to engineer novel adeno-associated virus (AAV) capsids to deliver transformative genetic therapies to people with serious diseases. Affinia Therapeutics proprietary AAVSmartLibrary and associated technology provides capsids for improved tissue tropism, manufacturability and pre-existing immunity. The collaboration will leverage Affinia Therapeutics capsid engineering expertise and Vertexs scientific, clinical and regulatory capabilities to accelerate the development of genetic therapies for people affected by Duchenne muscular dystrophy (DMD), myotonic dystrophy type 1 (DM1) and cystic fibrosis (CF).

This collaboration with Affinia Therapeutics will enhance our existing capabilities in discovering and developing transformative therapies for people with serious diseases, said Bastiano Sanna, Executive Vice President and Chief of Cell and Genetic Therapies at Vertex. Affinia Therapeutics innovative approach to the discovery and design of AAV capsids brings yet another tool to our Vertex Cell and Genetic Therapies toolkit, and were excited to partner with them to bring together their technology platform with our research and development expertise.

At Affinia Therapeutics, were setting a new standard in genetic therapy by leveraging our platform to methodically engineer novel AAV vectors that have unique therapeutic properties, said Rick Modi, Chief Executive Officer. Vertex is an established leader in developing transformative medicines for genetic diseases and renowned for its scientific rigor. We are thankful for the scientific validation this partnership brings and look forward to working closely with them to advance life-changing, differentiated genetic therapies and make a meaningful difference to those affected by these diseases.

About the Collaboration

Under the terms of the agreement, Affinia Therapeutics will apply its vector design and engineering technologies to develop novel capsids with improved properties. The agreement provides Vertex an exclusive license under Affinia Therapeutics proprietary technology and intellectual property (IP) in DMD and DM1 with an exclusive option to license rights for CF and an additional undisclosed disease. The scope of the agreement covers all genetic therapy modalities in these diseases. Affinia Therapeutics will be eligible to receive over $1.6 billion in upfront and development, regulatory and commercial milestones, including $80 million in upfront payments and research milestones that will be paid during the research term, plus tiered royalties on future net global sales on any products that result from the collaboration. Affinia Therapeutics will be responsible for the discovery of capsids that meet certain pre-determined criteria. Vertex will be responsible for and will fund the design and manufacturing of genetic therapies incorporating the selected capsids, preclinical and clinical development efforts, and commercialization of any approved products in the licensed diseases.

About Affinia Therapeutics

At Affinia Therapeutics, our purpose is to develop gene therapies that can have a transformative impact on people affected by devastating genetic diseases. Our proprietary platform enables us to methodically engineer novel AAV vectors and gene therapies that have remarkable tissue targeting and other properties. We are building world-class capabilities to discover, develop, manufacture and commercialize gene therapy products with an initial focus on muscle and central nervous system (CNS) diseases with significant unmet need. http://www.affiniatx.com.

About Vertex Pharmaceuticals

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, Dr. Sannas statements in the second paragraph of the press release, Mr. Modis statements in the third paragraph of the press release, and statements regarding future activities of the parties pursuant to the collaboration. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent Vertex's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, Vertex may not realize the anticipated benefits of the collaboration, and the other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

View source version on businesswire.com: https://www.businesswire.com/news/home/20200427005151/en/

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Vertex Pharmaceuticals and Affinia Therapeutics Establish Multi-Year Collaboration to Discover and Develop Novel AAV Capsids for Genetic Therapies -...

A research team at the Seoul National University Bundang Hospital (SNUBH) has released the results of phase 1 clinical trial of a new treatment that kills cancer cells of pancreatic cancer patients. In the trial, the researchers tested a method that used genetically modified adenovirus, in which two different cancer treatment genes are inserted, on patients with irresectable pancreatic cancer.

Pancreatic cancer is deadly cancer that people fear most because of its poor prognosis, as shown by a five-year survival rate of only about 12.2 percent.

Patients with locally advanced pancreatic cancer, in which cancer cells spread to nearby lymph nodes and blood vessels, face difficulty in receiving surgery and growing resistance to anticancer treatment, resulting in the abandonment of therapy. With the recent rapid development of cancer treatments, however, anticancer therapies based on precision medicine get the spotlight, and new therapies are being studied for pancreatic cancer.

These new therapies are drawing attention as to whether they provide hope for patients with pancreatic cancer whose vein or artery.

The research team, led by Professors Hwang Jin-hyuk and Lee Jong-chan, studied to confirm the safety and possibility of a new way to treat non-resectable pancreatic cancer patients with adenovirus as a gene carrier in nine patients for two years from August 2018.

The team inserted an adenovirus that can create two enzymes -- cytosine deaminase and tyrosine kinase -- to pancreatic cancer patients through endoscopic ultrasonography. The injected adenovirus naturally disappears in healthy cells and multiplies only in pancreatic cancer cells due to the primary effect of genetic modification.

After the injection, if the patient takes an oral medication without an anticancer effect, the drug meets the enzyme of the adenovirus in the pancreatic cancer cell and turns into an anticancer reagent. The changed virus, which can survive in cancer cells, then kills only pancreatic cancer cells.

The study result showed that drug administration to the nine pancreatic cancer patients did not show significant side effects during 12 weeks of treatment, proving to be a relatively safe medicine. Pancreatic cancer did not progress further in all nine patients who have been taking 12 weeks of treatment. The median progression-free survival, which is used as an index for evaluating the efficacy, was 11.4 months.

The study was meaningful as we could confirm the safety and potential of new gene therapy via the phase 1 trial, Professor Hwang said. We not only confirmed that the patients could be treated by inserting the modified genes to pancreatic cancer but also saw the effect of slowing the progression of pancreatic cancer, providing a basis for follow-up studies.

The trial results were published in the latest issue of Gastrointestinal Endoscopy, a U.S. journal with top authority in the endoscopic field.

shim531@docdocdoc.co.kr

< Korea Biomedical Review, All rights reserved.>

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SNUBH team finds modified adenovirus halts pancreatic cancer progression - Korea Biomedical Review

The ongoing COVID-19 pandemic and the worldwide reaction to it has compelled companies to radically rethink their strategies and the way they operate. We salute the industry experts helping companies survive and sustain in this pandemic. At Healthcare Intelligence Market Analysts, are undertaking continuous efforts to provide analysis of the COVID-19 impact on the Gene Therapy Technologies Market. We are working diligently to help companies take rapid decisions by studying.

The global Gene Therapy Technologies Market is segmented on the basis of product & services, application, end user and geography. On the basis of product & services, the Gene Therapy Technologies Market is segmented into consumables, software and services. On the basis of application, the Gene Therapy Technologies Market is segmented into diagnostics, therapeutics, research & developmental activities and others. Based on end user, the Gene Therapy Technologies Market is classified as academic & research institutes, diagnostics laboratories, biotech & pharmaceutical companies and others.

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Key Market Players are:

Biogen (US), Novartis AG (Switzerland), Gilead Sciences, Inc. (US), Spark Therapeutics, Inc. (US), MolMed S.p.A. (Italy), Orchard Therapeutics plc. (UK), SIBIONO (China), Shanghai Sunway Biotech Co., Ltd. (China), bluebird bio, Inc. (US), Human Stem Cells Institute (Russia), AnGes, Inc. (Japan), Alnylam Pharmaceuticals, Inc. (US), Sarepta Therapeutics (US), Jazz Pharmaceuticals, Inc. (Ireland), Akcea Therapeutics (US), and Dynavax Technologies (US).

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Covid-19 Update Whats driving the Gene Therapy Technologies Market Growth? See with Prominent Players and High CAGR rate - Bandera County Courier

The Hemophilia Gene Therapy market demand is anticipated to flourish during the forecast period 2020-2027. The report offers information related to import and export, along with the current business chain in the market at the global level. This report provides an in-depth overview of the Hemophilia Gene Therapy market. This includes market characteristics, consisting of segmentation, market share, trends and strategies for this market. The Market Size section provides historical forecasts of market growth and future. An in-depth analysis of the major companies operating in the market is also mentioned in this research report.

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Hemophilia Gene Therapy Market- Growth Opportunities by Manufacturers, Regions, Type, Application and Trends Forecast - Cole of Duty

Swiss pharma Novartis AG NVS reported encouraging results for the first quarter of 2020, beating on both sales and earnings. Forward purchasing due to COVID-19 also boosted performance. Moreover, the company maintained its outlook for 2020 even amid uncertainties related to the coronavirus pandemic.

First-quarter 2020 core earnings (excluding one-time charges) of $1.55 per share easily beat the Zacks Consensus Estimate of $1.37 and increased from $1.21 reported in the year-ago quarter.

Revenues rose 11% year over year to $12.3 billion, driven by Entresto, Zolgensma, Cosentyx, Kisqali and Piqray. Revenues beat the Zacks Consensus Estimate of $12.15 billion. Excluding COVID-19-related forward purchases, sales grew approximately 9%.

The stock has lost 5.4% in the year so far against the industrys growth of 0.1%.

All growth rates mentioned below are on a year-over-year basis and at constant exchange rates.

Quarter in Detail

Novartis operates under two segments Innovative Medicines and Sandoz (generics).

The Innovative Medicines division recorded sales of $9.8 billion, up 13% year over year. Within this segment, the Pharmaceuticals business unit grew 14%, driven by continued momentum in Entresto and Cosentyx and the uptake of Zolgensma.

Psoriasis drug, Cosentyx, continues to gain traction. Cosentyx sales increased 19% to $930 million, driven by strong demand in the United States. Entresto sales grew 62% to $569 million, driven by demand growth across geographies. Increasing contribution from Zolgensma (gene therapy for pediatric patients with spinal muscular atrophy) also boosted this business unit.

The Oncology business unit grew 12%, driven by continued momentum in Promacta/Revolade, Tafinlar + Mekinist and Kisqali as well as the launch uptake of Piqray. Kisqali sales surged 82%, driven by strong double-digit growth owing to higher demand in all geographies. Kymriah grew strongly in Europe and the United States.

This growth was partially offset by generic competition for Afinitor, Exjade, Travatan and Exforge.

Sales at the Sandoz division were $2.5 billion, up 11%, driven by volume growth including COVID-19-related forward purchasing, partly offset by price erosion. Biopharmaceuticals sales grew 31%, driven by continued strong double-digit growth in Europe. Novartis has decided to retain Sandoz US generic oral solids and dermatology businesses, after terminating its mutual agreement with Aurobindo.

Guidance for 2020 Reiterated

The company expects net sales in 2020 to grow in mid to high-single digits. Innovative Medicines revenues are projected to grow in mid to high-single digits. Revenues from Sandoz are expected to grow in low-single digits.

COVID-19 Update

The pipeline progress during the quarter was encouraging. Novartis initiated a phase III clinical trial in collaboration with Incyte INCY to evaluate the use of Jakavi in combination with standard of care (SoC) compared to SoC alone for COVID-19 infection and a phase III study on Ilaris in patients with pneumonia as a result of SARS-CoV-2 infection.

Additionally, Novartis announced a phase III study on hydroxychloroquine, alone and in combination with azithromycin, for the treatment of hospitalized patients with COVID-19 disease. Under an expedited managed access program, Novartis has granted requests and provided Jakavi and Ilaris. Requests for investigator-initiated trials have also been granted for COVID-19-related clinical studies of Gleevec, Cosentyx, hydroxychloroquine and Diovan. The company has committed to donate up to 130 million doses of generic hydroxychloroquine to support the global COVID-19 pandemic response.

Story continues

Our Take

Novartis first-quarter results were strong, driven by the solid performance of key drugs like Cosentyx and Entresto, and contribution from gene therapy, Zolgensma. COVID-19 related forward purchasing also boosted performance.

New launches like Piqray and Beovu should further boost the companys performance in the upcoming quarters. In particular, strong uptake is expected from Beovu as it is the first FDA-approved anti-VEGF to offer greater fluid resolution as compared to Regeneron REGN and Bayers BAYRY market-leading drug, Eylea.

Novartis currently carries a Zacks Rank #3 (Hold). You can see the complete list of todays Zacks #1 Rank (Strong Buy) stocks here.

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Novartis (NVS) Q1 Earnings and Revenues Surpass Estimates - Yahoo Finance

Global Gene Therapy Market Research Report 2015-2027 of Major Types, Applications and Competitive Vendors in Top Regions and Countries Market has provided a comprehensive analysis of the industry. This included the existing market conditions, central or critical regions, the price of the product, capacity, production, demand and supply, profit, growth pace and the outlook. The study has presented recent project SWOT analysis apart from investment feasibility analysis. In turn, a review of the investment return has also been provided to help the stakeholders and any possible new entrants.

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Gene Therapy Market Latest Trends, New Innovation and Global Analysis 2020 to 2027 - Herald Writeup

Worldwide Gene Therapy Market has been thoroughly elaborated in a research report put on sale by Marketresearch.biz, bearing the title Gene Therapy MarketGlobal Industry Analysis, Size, Share, Growth, Trends, and Forecast 20202029. According to the report, the market is expected to be driven by a wide range of macroeconomic and industry-related factors. The key players in this market are focusing on product strategies and developments to increase their customer base and maintain their position.

The Gene Therapy Market is an intrinsic study of the current status of this business vertical and encompasses a brief synopsis about its segmentation. This industry report is inclusive of a nearly accurate prediction of the market scenario over the forecast period 20202029 market size with respect to valuation as sales volume. The study lends focus to the top magnates comprising the competitive landscape of Gene Therapy market, as well as the geographical areas where the industry extends its horizons, in magnanimous detail.

|| Access insightful study with over 100+ pages, list of tables & figures, profiling 10+ companies. Ask for Free Sample Copy(PDF) @https://marketresearch.biz/report/gene-therapy-market/request-sample

Operational and Emerging Players: Gene Therapy Market- Novartis, Kite Pharma Inc, GlaxoSmithKline PLC, Spark Therapeutics Inc, Bluebird bio Inc, Genethon, Transgene SA, Applied Genetic Technologies Corporation, Oxford BioMedica PLC, NewLink Genetics Corp., Amgen Inc

According to the current market situation, this report continuously observing promising growth of the global Gene Therapy market. The report further suggests market appears to progress at an accelerating rate over the forecast period. Also, the major players are elaborated on the basis of the proprietary technologies, distribution channels, industrial penetration, manufacturing processes, and revenue. In addition, report also examines R&D developments, legal policies, SWOT Analysis and strategies of Gene Therapy market players.

This Gene Therapy market research report surrounds importance on:

Manufacturing process and technology used in Gene Therapy market, key developments and trends changing in the development

Complete examination, including an evaluation of the parent market

Detailed account of market, volume and forecast, by leading key players, product type and end users applications

Environmental spread, development designs, pieces of the overall industry, key methodologies, and different financials systems of Gene Therapy market

Industrial analysis by upstream raw materials, downstream industry, current market dynamics and ensuing consumers analysis

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Gene Therapy Market Segmentation Outlook:

By Vector:Viral vectorRetrovirusesLentivirusesAdenovirusesAdeno Associated VirusHerpes Simplex VirusPoxvirusVaccinia VirusNon-viral vectorNaked/Plasmid VectorsGene GunElectroporationLipofection

By Gene Therapy:AntigenCytokineTumor SuppressorSuicideDeficiencyGrowth factorsReceptorsOther

By Application:Oncological DisordersRare DiseasesCardiovascular DiseasesNeurological DisordersInfectious diseaseOther Diseases

Gene Therapy Market Section by Region:

ASIA-PACIFIC MARKET: China, Southeast Asia, India, Japan, Korea, Western Asia

THE MIDDLE EAST & AFRICA MARKET: GCC, North Africa, South Africa

NORTH AMERICA MARKET: United States, Canada, Mexico

EUROPE MARKET: Germany, Netherlands, UK, France, Russia, Spain, Italy, Turkey, Switzerland

SOUTH AMERICA MARKET: Brazil, Argentina, Columbia, Chile, Peru

Our Research Methodology is based on the following main points:

i. Data Collections and Interpretation

ii. Analysis

iii. Data Validation

iv. Final Projections and Conclusion

The latest research on the Gene Therapy Market fundamentally delivers insights that can empower stakeholders, business owners, and field marketing executives to make effective investment decisions driven by facts and extremely thorough research. The study aims to provide an evaluation and deliver essential information on the competitive landscape to meet the unique requirements of the companies and individuals operating in the Gene Therapy Market for the forecast period, 20202029. To help firms comprehend the Gene Therapy industry in multiple ways, the report exhaustively assesses the share, size, and growth rate of the business worldwide.

There are 13 Sections to show the global Gene Therapy market:

Chapter 1: Market Overview, Drivers, Segmentation overview, Restraints and Opportunities

Chapter 2: Market competition by key Manufacturers

Chapter 3: Production by Regions

Chapter 4: Consumption by Regions

Chapter 5: Production By Types, Revenue and Market share by Types

Chapter 6: Market share (%) and Growth Rate by Applications, Consumption By Applications

Chapter 7: Complete profiling and analysis of leading Manufacturers

Chapter 8: Region-wise manufacturing expenses, Manufacturing cost analysis, Raw materials analysis

Chapter 9: Sourcing Strategy, Industrial Chain and Downstream Buyers

Chapter 10: Marketing Strategy Analysis, Distributors/Traders

Chapter 11: Market Effect Factors Analysis, Impact Analysis

Chapter 12: Market Forecast 2020-2029

Chapter 13: Gene Therapy Research Findings and Conclusion, methodology and data source, Appendix

CLICK HERE, To Get Complete TOC

Conclusively, this report will provide you a clean view of each and every truth of the market without a need to consult another research report or a statistics supply. Our report will offer you with all of the facts about the past, present, and future of the Market.

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Inclusive Comprehension 2020: Gene Therapy Market (Trending PDF) Addressing Structure, Scope, Potential, and Growth Prospects Till 2029 - Jewish Life...

In times of crisis, top quality management is more important than ever. There is no script to follow and few parallels to draw from history for these unprecedented times. So now more than ever it is important to have companies in the portfolio which not only have sound business models but also have exceptional management teams. CSL is one such company.

Despite the amazing run the share price has had, CSL remains a solid long term investment proposition. It operates in a global growing industry and occupies a strong position within it. It is well managed, earns a high return on capital due to its numerous competitive advantages and has plenty of opportunities to reinvest which should drive growth for many years to come.

Whilst not excessively cheap based on near term earnings, high-quality companies like CSL have the propensity to surprise on the upside - time and time again.

Two-year chart of the CSL share price

How is CSL navigating COVID-19?

CSL provides lifesaving and largely non-discretionary treatments to seriously ill patients as well as manufacturing the seasonal flu vaccine. The current pandemic has seen increased demand for CSLs core IVIG products and flu vaccine. It is a credit to CSLs execution it has been able to maintain consistent supply to all countries, despite country lockdowns and border closures.

However, CSL is not completely immune to some temporary disruption and is currently experiencing a reduction in collected plasma volumes as fewer people visit collection centres and stay home. The impact of this wont be seen for another 6-9 months due to CSLs long manufacturing cycle, which provides it with time to work on mitigation strategies to reduce the impact.

In the US donors are paid to give plasma so donation tends to increase in times of high unemployment. As a result, it is likely CSL will see a particularly strong rebound in collected plasma as social distancing measures are relaxed. This will help offset the reduction being experienced right now and may well see CSL gain further market share as it has invested more heavily in its collection network than other fractionators.

CSL is also responding to COVID-19 from 3 different angles:

These will be ongoing studies, none of which is expected to impact earnings in the short term, but may provide longer-term opportunities.

CSL is exceptionally well managed and has earned a reputation of focussing on patients, investing in its staff, being a reliable supplier of lifesaving products and a consistent executor of a sound business strategy.

It occupies a strong position within its industry and has built up numerous sustainable competitive advantages that have allowed it to consistently earn a high return on invested capital, which should drive shareholder value for many years to come.

The global plasma product market is growing at a healthy 8-10% pa and demand is outstripping supply. Demand for key products such as IG (Immunoglobulins) is being driven by increased diagnosis and awareness of conditions that benefit from IG treatment and supply is constrained due to the fact that CSL has been the only plasma fractionator that has continued to invest and open plasma collection centres over the past decade.

Opening a plasma collection centre requires both financial investment and time time to get the centre approved and time to draw in sufficient donors to collect enough plasma for the centre to reach scale. CSL had the foresight to continue investing in these centres while its competitors stopped, resulting in today having access to much more raw material (plasma) than its competitors. This has allowed it to gain market share and importantly, consistently supply its lifesaving treatments to patients.

CSL has also continuously invested in best in class manufacturing facilities. The amount of capital required to reach scale and meet the regulatory requirements of plasma fractionation is in itself a barrier to new entrants to this industry.

CSLs efficiency in manufacturing enables it to extract more product and value per litre of plasma than its competitors, making CSL the lowest cost producer. This results in a higher profit margin and boosts its ability to invest in R&D for new products and therapies.

Reinvest for long term growth

As mentioned, CSL reinvests a healthy chunk of cashflow back into R&D projects and history demonstrates a high hit rate in successfully commercialising this a return on investment of over 25%. This goes not only to the talent of the researchers working on the science of each project, but also to CSLs ability to select projects that have the most promise and the grit to shut a project down if its unlikely to stack up commercially.

New product launches to treat a variety of rare diseases as well as improvements on existing therapies have been an important driver of CSLs profitability. There is a strong pipeline of R&D projects addressing unmet clinical needs. Not all will be successful and these opportunities are impossible to accurately value, but using track record as a guide, there are enough long dated opportunities in the pipeline to underpin good long-term growth.

Good stewards of shareholder capital

As well as managing the operational aspects of the business exceptionally well, CSL management along with the board, also have an excellent track record of being good stewards of shareholder capital. CSLs business is by nature highly capital intensive, however it has still been able to achieve a return on invested capital of well in excess of 20% consistently for many years.

CSL is a good example of how linking management long term incentives to return on capital (which not enough companies do) can benefit shareholders over the long term. This has resulted in CSL investing back in the business where the return justifies the capital and at times over the years, returning excess capital to shareholders.

Rival therapies?

The nature of biotechnology is that there will always be competing new products and new breakthroughs that may threaten current products. This is why continuing to invest in R&D is so important. There are various therapies in early stages that may threaten or at least change the demand curve for CSLs products - two often mentioned are gene therapy and FcRn inhibitors.

Both are still in early trial phases and even if successful, the adoption profiles are unknown. It is worth noting that CSL is involved in its own research into these potentially competing therapies, for example acquiring gene therapy company Calimmune, demonstrating a willingness to disrupt itself if necessary.

CSL remains an attractive long-term investment opportunity

COVID-19 has not changed CSLs expected FY20 earnings. Guidance was recently affirmed by the company, however, FY21 presents both challenges and opportunities. Challenges regarding plasma collection volumes and opportunities regarding increased demand for the flu vaccine. The pandemic is likely to raise awareness of the flu vaccine and create a higher level of demand for it. Also, there is the potential to manufacture treatments and/or a vaccine for COVID-19 in the future.

Unlike many other companies through this crisis, CSL does not need to raise capital. Its balance sheet is solid and it has access to a further $1bn in liquidity if necessary.

CSLs share price has outperformed the market in the recent sell off but is off its highs in absolute terms. Like many healthcare companies at the moment its headline PE is higher than its historic average but using valuation methods which take into account the duration of CSLs growth prospects and current low interest rates as well as the current market and economic uncertainty, CSL remains an attractive long term investment opportunity.

We believe long term value creation is achieved by going deeper into company analysis, balanced with the rigour of an objective process. We provide investors with access to risk controlled, active equity returns through a unique investment process that combines quantitative analysis with deep fundamental research. To find out more visit our website, or click contact below.

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CSL still a solid long-term proposition - Kelli Meagher - Livewire Markets