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British Royals Survive 30 Years Longer Than Their Subjects – Yahoo News

April 17th, 2021 1:54 am

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In the U.K. it is customary to receive a personalized message from the queen on your 100th birthdaysuch is the relative rarity of reaching the milestone.

Prince Philip was just a couple months off, dying at the age of 99 years and 10 months on April 9, 2021. The last notable royal death before his was that of the queen mother in 2002. She was 101 years old.

Reaching such a ripe old age isnt uncommon among the British ruling familyin fact, my analysis shows that on average they live an additional 30 years compared with their subjects.

I looked at the duration of life of the last six British monarchs, along with the longevity of their spouses and childrenin total 27 royals. What it reveals is a fascinating and familiar story for those of us who study aging and longevity for a living. As a professor of epidemiology and biostatistics, I had previously observed the exact same phenomenon among U.S. presidentsthey also tend to live decades longer than the general population they serve.

If you love The Daily Beasts royal coverage, then we hope youll enjoy The Royalist, a members-only series for Beast Inside. Become a member to get it in your inbox on Sunday.

The ruling U.K. monarchs from Queen Victoria onward lived an average of 75 years. And this longevity will continue to rise with each day that Queen Elizabeth IIcurrently age 95lives. Their spouses survived even longer, reaching an average age of 83.5 years. If Victorias husband Prince Albert, who died of suspected typhoid fever at age 42 in 1861, is removed from the equation, the average duration of the life of the spouses of the monarchs was an astonishing 91.7 years.

By contrast, the average life duration of the wider U.K. population for the years the monarchs were born throughout this period was only 46 years, according to figures from the Human Mortality Database. For example, the typical life expectancy at birth for a female in the U.K. in 1819 was just under 41 years. Queen Victoria, also born in 1819, was 81 when she died. By the time Elizabeth II was born in 1926, life expectancy at birth for females in the U.K. had risen to 62the queen has already surpassed that by some 33 years.

Story continues

Such differences in lifespanwith some members of the royal family living to an age double that expected of the general populationare considered in aging circles to be extremely large, but not uncommon.

Lifespan differences of this magnitude are the result of a combination of genetic as well as social and behavioral influences.

No one can live long without first having won the genetic lottery at birth. To maximize the chances of achieving exceptional longevityupward of 85 years oldyou must begin by being lucky enough to have long-lived parents. But even for those blessed with the gift at birth of the potential for a long life, this is no guarantee youll outlive your contemporaries.

The next challenge is to avoid behaviors that shorten life. That list is longit is a lot easier to shorten life than extend itbut among the most well known are smoking, eating in excess and lack of exercise.

And then there is the influence of poverty and privilege. Being born into or living in poverty has been shown to be one of the most important factors that shortens lifespanand it is here that perhaps the royals have the greatest advantage.

Further evidence of privilege being a crucial ingredient in the recipe for exceptional longevity can be seen in the fact that the children of the last six U.K. monarchs that died from natural causes lived an average of 69.7 years. This is some 23 years more than the average age of British subjects over that period.

Put simply, British monarchs and their families live so much longer than their subjects for the same reason other subgroups of the population across the globe live longer than contemporaries born in the same year: privilege over poverty. A famous study conducted in Manchester, England, in 2017 demonstrated vast differences in life expectancy depending on the conditions of where people lived. Access to higher education and economic status was directly correlated with longer life, while lower education, income and poverty were linked to shorter lives.

In the U.S., similar studies of life expectancy by county, census tract and zip code demonstrated the same phenomenon. In fact, there are multiple instances of dramatic differences in longevity among people living as close as across the street from each othercaused by differences in poverty and privilege.

Differences in duration of life are first defined by genetics, but it is then heavily mediated by education, income, health care, clean water, food, indoor living and working environments, and the overall effects of high or low socioeconomic status.

The long life of Prince Philip is a cause for celebrating the progress of medical science in being able to keep people alive for longer. But it is in part the result of a privilege denied to many and a reminder that humanity has a long way to go to equalize the chances of living a long life.

S. Jay Olshansky is a professor of epidemiology and biostatistics at the University of Illinois at Chicago

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Dietary Spermidine Boosts Cognition in Insects and Rodents – The Scientist

April 17th, 2021 1:54 am

Feeding the organic compound spermidine to mice and fruit flies boosted mitochondrial activity in the animals brains and, with it, their cognitive ability, according to a paper in Cell Reportspublished April 13. The study also intimates a similar spermidine-linked cognition boost applies to humans.

Overall, I think that spermidine is probably one of the most promising molecules for aging research in general . . . and here they focus on cognition, says gerontologist Valter Longo of the University of Southern California who was not involved in the work. The most convincing data are from flies and mice, he says, because the human data are only correlative. But, he adds, the good news is that if you have a spermidine-rich diet, it seems to be a good one. Its certainly not bad for you in terms of cognition.

Spermidine is a natural compound present in a wide range of foods, such as wheat germ and aged cheese, though it was originally identified in semen. The compound promotes autophagya sort of cellular self-cleaning processand levels of both spermidine and autophagy appear to diminish in the bodys cells as animals, including humans, age.

Prior studies on lab animals have shown all manner of benefits from spermidine, from an increase in longevity to fortification of heart and immune cells to preservation of memory in old age.

Other groups have investigated the effects of spermidine injections . . . on behavioural and cognitive phenotypes, but we wanted to increase translatability for future applications and therefore concentrated on a dietary approach, study coauthors Frank Madeo and Stephan Sigrist write in an email to The Scientist. Both Madeo, who studies aging at the University of Graz in Austria, and Sigrist, a neuroscientist at Freie University in Germany, have equity stakes in The Longevity Labs, a company that sells spermidine supplements.

Madeo and Sigrist have already been involved in a pilot clinical study in which dietary spermidine was given daily to 14 older individuals for three months. The results of that placebo-controlled trial indicated a slight improvement in memory in the group taking spermidine. The researchers new work lends support to those preliminary trial results and dives into the physiological as well as cognitive effects of the compound.

For example, in addition to showing that old mice given spermidine in their drinking water for six months performed better than control mice did in learning and memory tests (associating a light stimulus with access to a reward, and remembering the location of a platform submerged in water), the team observed that the compound crosses the blood-brain barrier and steadily accumulates in the brain over a period of weeks. In the animals hippocampia brain region associated with learning and memorythe team found that the compound boosted cellular respiration, essentially increasing the brain cells energy levels, and promoted the modification of a protein called eIF5A, a translation factor that drives the production of a master autophagy regulator.

Compared with controls, spermidine-fed flies similarly displayed improved memory and higher brain cell respiration, both of which were dependent on autophagy, the team showed. And the treated insects exhibited the eIF5A modification, which Madeo and Sigrists team describe in detail in a second Cell Reports paper, also published this week. The modification, known as hypusination, involves the addition of hypusine, an uncommon amino acidso uncommon, in fact, that eIF5A is the only known protein to contain it.

Its quite convincing that they can show the same thing in mice and flies, says immunologist Katja Simon of the University of Oxford who was not involved in the study. As for the hypusination of eIF5A, which Simons team had previously reported drives immune cell rejuvenation in spermidine-fed mice, she adds, It was pleasing to see that they could see that in the brain [too]. Simon is a paid consultant for Oxford Healthspan, a company that sells a spermidine dietary supplement, but has no financial stake in the company.

In addition to the mouse and fly studies, the team also examined human epidemiological data. They examined cognitive test scores and dietary records of more than 800 older adults, calculating the participants spermidine intake from diet questionnaires. After five years from the studys baseline (1990), estimated spermidine intake positively correlated with cognitive test scores, with higher intakes also reducing the chance of developing cognitive impairment.

Altogether, the work indicates that dietary spermidine may support improved cognition later in life and, says Longo, points to a need for larger-scale clinical studies to investigate spermidines effects on cognitive decline.

Although decisive clinical proof for spermidines cognitive benefits are lacking, Oxford Healthspan and The Longevity Labs sell spermidine supplements for around $100 for a months supply. High levels of this molecule can also be acquired rather more cheaply by eating wheat germ, mature cheddar, soybeans, mushrooms, and other foods in which it is abundant.

S. Schroeder et al., Dietary spermidine improves cognitive function,Cell Rep, doi:10.1016/j.celrep.2021.108985, 2021.

YT. Liang et al., eIF5A hypusination, boosted by dietary spermidine, protects from premature brain aging and mitochondrial dysfunction,Cell Rep, doi:10.1016/j.celrep.2021.108941, 2021.

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Ce’Cile happy with rise of female talent – Says once they produce timeless music, longevity is a must – Jamaica Star Online

April 17th, 2021 1:54 am

CeCile happy with rise of female talent - Says once they produce timeless music, longevity is a must | Entertainment | Jamaica Star

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April 16, 2021

Entertainer Ce'Cile, who is celebrating two decades in the entertainment business, says that not only is she satisfied with the quantity of women rearing their heads in today's musical landscape, but also the quality these 'alpha females' possess.

"I am just so happy with what our women have been bringing to the table. There are so many of them that are doing well that it makes me proud to watch. The girls have been showing up and showing out and because of that the industry is a lot more balanced today than it was several years ago," she told THE WEEKEND STAR. "You have so many of these young ladies who are doing their thing not just in reggae but dancehall. And not only are they showing up in numbers but their work is impressive."

Ce'Cile, who just dropped her latest EP Sophisticated, said the only advice she would give to the current crop of young female artistes is to do music that will last through generations.

"I have tried from the beginning of my career to make sure that every song I put out there is one that will outlive me. I would be washed up already if I wasn't doing music that stood the test of time but I always thought about longevity," she said. "It's not by accident that I have lasted so long and why I seem fresh to a lot of people. I wrote some classic tracks. I can go anywhere and people a bawl fi hear I'm Waiting. That is what I want for these women in the business."

"That is why I love and idolise people like Beres Hammond. My mom listened to Beres Hammond, I grew up listening to Beres Hammond, and Nana [her daughter] is now listening to him and she loves his songs. His songs connect with every generation," she continued. "As an artiste, it is great when you can produce work that will outlive you and last for generations. I really believe the women I see doing their thing in music today can have that. They just have to be deliberate about the business and serious about their craft."

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Longevity and Anti-senescence Therapy Market Analysis, Status and Global Outloo – Business-newsupdate.com

April 17th, 2021 1:54 am

The Longevity and Anti-senescence Therapy market research report identifies all the factors positively and negatively impacting the growth of this vertical, to direct stakeholders in making right decisions. Besides, it includes statistics coverage of the past and current business scenario to validate the forecast figures presented in the document. Moreover, the report elaborates the various industry segments and unveils the lucrative prospects that will assure high profits for the forthcoming years.

As per industry experts, Longevity and Anti-senescence Therapy market is predicted to accumulate notable profits during 20XX-20XX, recording a CAGR of XX% throughout.

Apart from these factors, the document illustrates the impression of COVID-19 on this domain, emphasizing on the challenges like variations in supply-demand, cost management, and digitizing operations faced by businesses. In this context, it suggests solutions that will ensure an upward growth trajectory in the ensuing years.

Request Sample Copy of this Report @ https://www.business-newsupdate.com/request-sample/77865

Importantpointers from Longevity and Anti-senescence Therapy market report:

Longevity and Anti-senescence Therapy market segments covered in the report:

Regional bifurcation: North America, Europe, Asia-Pacific, South America, Middle East & Africa, South East Asia

Product types: Hemolytic Drug Therapy , Gene Therapy , Immunotherapy and Other Stem Cell Therapies

Application spectrum: Hospital , Medical Service Institution and Drug and Device Sales

Competitive dashboard: CohBar , AgeX Therapeutics?Inc , TA Sciences , Unity Biotechnology and PowerVision Inc

Highlights of the Report:

The scope of the Report:

The report offers a complete company profiling of leading players competing in the global Longevity and Anti-senescence Therapy marketwith a high focus on the share, gross margin, net profit, sales, product portfolio, new applications, recent developments, and several other factors. It also throws light on the vendor landscape to help players become aware of future competitive changes in the global Longevity and Anti-senescence Therapy market.

Reasons to Buy the Report:

Major Points Covered in Table of Contents:

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What to Consider When Choosing Construction and Building Materials – MSN Money

April 17th, 2021 1:54 am

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A 2020 study by the National Association of Home Builders (NAHB) examined who decides what building materials to use in new home construction. Not surprisingly, builders and contractors exert the greatest influence over primary construction materials, such as windows and doors, siding and house wrap, lumber and plywood.

But this applies more to home builders in planned developments. If you're lucky enough to be custom-building a new home or vacation getaway, or remodeling an existing one, you'll probably have a much larger role in deciding what materials go into the build. And if you're taking on a DIY building or renovation project, then it's all on you to make sure the materials meet the demands of your location, climate and budget.

Here are seven questions to ask when choosing building materials:

Unless you have a limitless building budget, choose materials priced within your means without compromising too much on quality. Natural stone siding is the most expensive residential exterior building material, but it's also one of the most durable. When properly installed, stone siding can last more than 100 years. Make sure the materials you choose strike the right balance between price and longevity.

You may love the look of a certain material, but that doesn't mean it's right for your climate or region. Wood siding looks authentic and natural, but if you put it on a house in South Florida, the long rainy seasons can do lots of damage. So can termites, who also like the warm climates of the Southeast. Before you commit to a material, make sure it's hardy enough for the climate challenges of your specific region.

You loved those hand-painted tiles you saw on your trip to Portugal, and you absolutely want them in your new kitchen. But how long are you willing to wait for them?

The more unique or rare the material, the harder it may be to find, and the longer it may take to be delivered. You probably have a timeline in mind for your project, so make sure to choose materials that fit the timeline. If those hand-painted tiles direct from Lisbon take three months to arrive, it may not be worth it.

You want to build green, and a modular home on your wooded lot seems like the right solution. But will you need to cut down trees to position that home where you want it? Consider whether your materials can be delivered with relative ease. Keep in mind things like driveway width, overhead utility lines and if you're building in a remote area the size, condition and grade of access roads.

If building with sustainable materials is a priority, make sure you choose the right materials for your climate and setting. Solar panels, for example, might not power you through a long Midwestern winter.

Also keep transport costs in mind. If those sustainable materials must be hauled halfway across the country to your job site, the carbon emissions of transport might negate any eco-benefits.

A traditional log cabin looks great in the woods of Vermont. On a beach in Florida, not so much.

When choosing materials, especially wood, stone, stucco or other kinds of exterior siding, consider regional aesthetics. If you're building in a developed area, especially in deed-restricted communities, pay attention to local building codes, zoning ordinances and Homeowners Association (HOA) rules. These may dictate everything from what materials you can use to what color your house can be.

Concrete, steel, wood and vinyl are among the most common exterior materials because they are readily available and long-lasting. If you're deciding between these or building materials that are more sustainable, more aesthetically pleasing or new to the market, do your homework and think long-term.

Talk with homeowners or designers who've worked with the materials you're considering. Think about how long you plan to own the home you're building or renovating, and how much maintenance you're willing to do over the next five, 10 or 20 years. Let that guide your decisions, especially if you're building a vacation getaway. You don't want to spend all your time there on upkeep!

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Longevity and Anti-senescence Therapy Market 2021: Current and Future Market Potential NeighborWebSJ – NeighborWebSJ

April 17th, 2021 1:54 am

Longevity and Anti-senescence Therapy Market 2021-2027:

The Global Longevity and Anti-senescence Therapy market exhibits comprehensive information that is a valuable source of insightful data for business strategists during the decade 2015-2027. On the basis of historical data, Longevity and Anti-senescence Therapy market report provides key segments and their sub-segments, revenue and demand & supply data. Considering technological breakthroughs of the market Longevity and Anti-senescence Therapy industry is likely to appear as a commendable platform for emerging Longevity and Anti-senescence Therapy market investors.

The complete value chain and downstream and upstream essentials are scrutinized in this report. Essential trends like globalization, growth progress boost fragmentation regulation & ecological concerns. This Market report covers technical data, manufacturing plants analysis, and raw material sources analysis of Longevity and Anti-senescence Therapy Industry as well as explains which product has the highest penetration, their profit margins, and R & D status. The report makes future projections based on the analysis of the subdivision of the market which includes the global market size by product category, end-user application, and various regions.

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This Longevity and Anti-senescence Therapy Market Report covers the manufacturers data, including shipment, price, revenue, gross profit, interview record, business distribution, etc., these data help the consumer know about the competitors better.

Topmost Leading Manufacturer Covered in this report:

CohBar, TA Sciences, Unity Biotechnology, AgeX Therapeutics?Inc, PowerVision Inc.

Product Segment Analysis:

Hemolytic Drug Therapy Gene Therapy Immunotherapy Other Stem Cell Therapies

Application Segment Analysis:

Hospital Medical Service Institution Drug and Device Sales

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Regional Analysis For Longevity and Anti-senescence TherapyMarket

North America(the United States, Canada, and Mexico)

Europe(Germany, France, UK, Russia, and Italy)

Asia-Pacific(China, Japan, Korea, India, and Southeast Asia)

South America(Brazil, Argentina, Colombia, etc.)

The Middle East and Africa(Saudi Arabia, UAE, Egypt, Nigeria, and South Africa)

The objectives of the report are:

To analyze and forecast the market size of Longevity and Anti-senescence TherapyIndustry in theglobal market.

To study the global key players, SWOT analysis, value and global market share for leading players.

To determine, explain and forecast the market by type, end use, and region.

To analyze the market potential and advantage, opportunity and challenge, restraints and risks of global key regions.

To find out significant trends and factors driving or restraining the market growth.

To analyze the opportunities in the market for stakeholders by identifying the high growth segments.

To critically analyze each submarket in terms of individual growth trend and their contribution to the market.

To understand competitive developments such as agreements, expansions, new product launches, and possessions in the market.

To strategically outline the key players and comprehensively analyze their growth strategies.

View Full Report @ https://www.marketresearchupdate.com/industry-growth/longevity-and-anti-senescence-therapy-market-size-analysis-by-application-165495

At last, the study gives out details about the major challenges that are going to impact market growth. They also report provides comprehensive details about the business opportunities to key stakeholders to grow their business and raise revenues in the precise verticals. The report will aid the companys existing or intend to join in this market to analyze the various aspects of this domain before investing or expanding their business in the Longevity and Anti-senescence Therapy markets.

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Can strawberries slow down the aging process? – The Packer

April 17th, 2021 1:54 am

The long sought-after fountain of youth may have been hidden in plain sight all along. Three new studies suggest strawberries may be associated with slowing down aging of the brain, cardiovascular system, and gut microbiome.Courtesy Riss Design on Unsplash

As a person ages, the brain can experience changes that result in impairments in learning, memory, gait, and balance. Sometimes these changes lead to early cognitive decline, disability, or falls among older adults.

In a recent study by Dr. Barbara Shukitt-Hale and her team at the USDA Human Nutrition Research Center on Aging at Tufts University, 37 healthy older adults participated in a two-arm trial in which they consumed either freeze-dried strawberry powder beverages (24g/day, equivalent to two cups of fresh strawberries) or a calorie-matched control powder for 90 days. Participants completed a battery of balance, gait, and cognitive tests at baseline, midpoint, and conclusion of the trial.

Participants in the strawberry group performed better on spatial memory tasks and word recognition tests relative to those in the control group. These findings show that the addition of strawberry to the diets of healthy, older adults can improve some aspects of cognition, although more studies are needed to confirm this finding.

This research comes on the heels of recent publications that indicate diets higher in long-term flavonoid intake, from foods such as strawberries, could contribute to reducing the risk of Alzheimers Disease and related dementias.CourtesyAnastasia Zhenina on Unsplash

Endothelium is a thin layer of cells that lines every blood vessel in the body. Its responsible for the relaxation and constriction of veins and arteries, playing a major role in blood flow, blood pressure regulation, blood clotting, and wound healing. High total cholesterol and LDL (the bad cholesterol) can impair the function of the endothelium, clog arteries, and lead to heart disease, particularly in later years of life.

A new study from Dr. Britt Burton Freemans team of researchers at Illinois Institute of Technology asked middle-aged adults with moderately-high LDL cholesterol to drink beverages two times a day made from freeze-dried strawberry powder (25g in each) or a control powder for four weeks. After completing their first assigned beverage, they switched to the other one (strawberry/control) for an additional four weeks.

The health of the endothelium, measured by flow-mediated dilation, improved in the strawberry group one hour after drinking the beverage. Systolic blood pressure decreased two hours after drinking the strawberry beverage compared to the control, and this was more pronounced four weeks after the strawberry intervention.

The findings suggest that strawberries may improve endothelial function and be considered a specific food to include in a heart-healthy diet for aging adults with moderately-high cholesterol.CourtesyVanesa Conunaese on Unsplash

Strawberries act as prebiotics and may increase gut bacteria associated with lean body weight, health, and longevity, according to a new study out of University of California, Los Angeles (UCLA).

The pilot study led by Dr. Zhaoping Li and her team at the Center for Human Nutrition considered whether strawberries would alter the gut microbiota.

Fourteen adults were asked to follow a beige diet low in fiber and polyphenols for 8 weeks. Two weeks in, a 13g strawberry powder drink was introduced to the diet twice a day, for four weeks. Then the participants returned to a beige-only diet for the two final weeks without strawberries.

Drinking strawberry powder beverages twice a day for four weeks was associated with an increase of 24 operational taxonomic units (OTUs), or gut microorganisms. Once the participants returned to a beige-only diet, several of the OTUs reversed back to their pre-strawberry status, suggesting strawberrys role in influencing the gut bacteria.

Something as simple as making strawberries a regular part of the diet may be the key to delaying aging of both the mind and the body.

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Catalent introduces cryogenic capabilities to support growing demand for cell and gene therapy – gasworld

April 17th, 2021 1:52 am

Sharing the news on Thursday (15th April), the company said the investment has enabled part of the facility to be dedicated to the safe handling of cell and gene therapy samples, including the installation of cryogenic storage, allowing biological materials to be preserved in liquid nitrogen vapor at temperatures of around -180C.

The facility also has the ability to package, label and distribute cryogenic materials, ensuring the integrity of the therapies being prepared for clinical trials, and has been designed so that capacity can be rapidly expanded further to meet growing clinical supply needs, as well as future commercial demand.

Establishing robust clinical supply chain services for cell and gene therapies is complex and challenging, and Catalent has undertaken an in-depth strategic review to evaluate how it can establish a safe, efficient and flexible approach to support this fast-growing area of the industry, said Ricci Whitlow, President, Catalent Clinical Supply Services.

The solution we have implemented at Philadelphia not only meets current needs, but also provides a template for us to easily replicate at other facilities in our global network, allowing incremental capacity expansion within the new infrastructure as demand grows.

With sites in the US, UK, Germany, Singapore, Japan and China, and an extended network of over 50 depots, Catalents clinical supply services can handle a broad range of international compliance and distribution requirements to support global clinical trials.

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Bioprocessing and Bioproduction Trends in Cell and Gene Therapies – Technology Networks

April 17th, 2021 1:52 am

Cell and gene therapies hold great promise for treating human diseases, for which current therapeutics are lacking. However, the research and development process for delivering these therapies to the clinic is complex, comprising various bioprocessing and bioproduction steps that must be fulfilled and optimized to ensure the safety and efficacy of the final product. The process continues to evolve to meet increasing demand and to overcome unexpected obstacles, such as the impact of the COVID-19 global pandemic on supply chains and manufacturing efforts in the biotech and biopharma industries.In this interview, Technology Networks spoke with Neal Goodwin, PhD, Chief Scientific Officer at Teknova, to learn more about current bioprocessing and bioproduction trends in the cell and gene therapy space. Goodwin also discusses the impact of the COVID-19 pandemic on the biopharmaceutical sector and highlights how the industry has united to contribute to the development of vaccines at this critical time.

Molly Campbell (MC): You have 20 years of experience working in biotechnology and therapeutics. Can you talk about the evolution of the field through this time?Neal Goodwin (NG):It has been a great 20 years! The human genome release is one of the most dramatic events during those 20 years. Once we had the human genome at our disposal, the field burgeoned. We've seen targeted therapeutics make a significant impact in personalized healthcare imatinib, erlotinib, and the new KRAS inhibitors, among other targeted therapeutics for oncology, come to mind. Also, the first immuno-oncology therapeutics are now positively affecting large numbers of patients which is terrific.

The ability to move into gene therapy and address severe genetic disorders and conduct gene replacement therapy is fantastic. The progress in gene delivery viral vectors and non-viral nanoparticle delivery contributed to the rapid advancement of prominent COVID-19 vaccines. The mRNA-based vaccines and non-viral nanoparticle-based gene delivery technologies have validated a promising approach to gene therapy that is being expanded to rare genetic diseases. The ability to mass-produce mRNA vaccines demonstrated the feasibility of mRNA treatments and gene therapies for more widespread use for treating broader therapeutic targets and conditions.

MC: Can you talk to us about some of the current trends you are seeing in the bioprocessing and bioproduction space?NG: First of all, there is a considerable demand for bioprocessing and commercial production of viral and non-viral vectors. These efforts must support what is now well over 1,000 clinical development programs in gene therapy alone. There is a need to develop better individualized processes that are readily scalable for GMP production matched to specific therapeutics. This effort will require the implementation of new strategies, new technologies, and increased global manufacturing capacity. It is an exciting place to be, but there is a great deal of development left to achieve streamlined processes and optimized capacity.

MC: Cell and gene therapies are key emerging markets. What are some of the key challenges that exist when developing these products from the lab, through to clinical testing and eventually authorization?NG:There is a wide breadth of requirements I will try to narrow them down to a few of the essential points. Some autologous cellular therapies require cells to be genetically modified ex vivo. This genetic modification is usually done with viral vectors, and a crucial aspect is the delivery of the gene, i.e., the active pharmaceutical ingredient, to the cells. There is a need for more advanced lentiviral vector systems that are safer for use in systemic gene delivery.

Among the non-cellular gene delivery segment, adeno-associated virus (AAV) vector gene delivery is the most widely used viral gene delivery platform. AAV production relies on the efficient expression in human cell lines of plasmids that encode viral packaging, capsid and replication genes, and expression plasmids encoding the therapeutic gene. One of the drawbacks of AAV vectors employed for gene therapy is they generally cannot deliver larger gene fragments required for optimal treatments. So, better viruses or non-viral gene delivery systems are needed.

In turn, choosing the best cell manufacturing conditions that provide the most efficient viral vector production is essential. A key challenge is how to scale up with reproducibility and efficiency.

MC: How can we look to overcome the challenge of scale-up in cell and gene therapies?NG: Because therapeutic programs are each different, having adaptable processes to individual programs is vital. Some strategies focus on gene delivery into autologous cells or allogeneic cells that become expanded and transplanted into patients other strategies require direct delivery of viral or non-viral gene therapy products into patients.In viral gene delivery, matching the most suitable virus to achieve the best target tissue tropism is essential. To date, AAV has been successfully used in two FDA-approved gene therapies, with many in advanced clinical trials. Traditionally, there have been few available AAV serotypes available for gene delivery. However, now we can use a directed evolution approach to manipulate and generate AAV capsids that have better target tissue tropism with reduced host immunogenicity.

Concerning AAV production, the purification processes of viruses need optimized, which is a focus of Teknova. We are barcoding different AAV serotypes with specific processes to achieve higher purity and viral vector yield.

MC: How has the COVID-19 impacted the biotech sector?NG: COVID-19 motivated and united the biotech sector. It remains a worldwide effort where enormous challenges are being addressed rapidly and collaboratively. The field appears more collaborative, almost cheering once competitive firms and laboratories. Teknova experienced this by retooling and supplying GMP viral transport media for COVID-19 testing and providing the global research community with research reagents and custom GMP manufactured products. This transformation is something that instils pride.

MC: How do you envision the biotech sector will change over the next few years? Are there any areas, aside from cell and gene therapies, that excite you?NG: The biotech sector's attitude is positive and more confident than 20 years ago, and this spirit enables us to tackle problems once considered unsolvable.

One of the most exciting biotech advancement areas is precision therapeutics. For example, a difficult hurdle to overcome was treating cancers linked to specific mutant KRAS alleles in a high percentage of cancers. Multiple firms have recently advanced precision therapeutics to late-stage clinical trials to treat this historically untreatable cancer type precisely. I think this can-do attitude will continue.

Another area where I expect to see progress is gene therapy delivery that will increasingly evolve non-viral delivery technologies. This evolution will allow precise targeting of therapeutics delivery and undoubtedly improve human health through this process.

Neal Goodwin was speaking to Molly Campbell, Science Writer for Technology Networks.

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Alliance for Cancer Gene Therapy Summit 2021 Features World Renowned Cancer Researchers Advancing Solid Tumor Breakthroughs – WFMZ Allentown

April 17th, 2021 1:52 am

STAMFORD, Conn., April 16, 2021 /PRNewswire-PRWeb/ --April 29 marks a pivotal moment in cancer research when the world's brightest minds come together to discuss how to translate the success of CAR T-cell therapies for blood cancers into successful cell and gene therapies for the most complex and deadly solid tumor cancers. The scientists and companies driving the latest advances in cancer cell and gene therapy will gather online for a virtual Summit with the Alliance for Cancer Gene Therapy, who envision a cancer free future and want to change the C-word from Cancer to Cure. The Summit is open to the public, to medical professionals, scientists and companies interested in cell and gene therapy to fight cancer. To register for the Summit, visit acgtfoundation.org.

Summit 2021 is being held online on Thursday, April 29, 2021, from 10:00 a.m. until 6:00 p.m (ET), and features eight (8) panel discussions with leading researchers developing the next generation cancer cell and gene therapies, biotech companies who are bringing new treatments through the clinic, and investors who are funding this burgeoning pipeline of solid tumor breakthroughs.

The panels include:

Keynote: Does Cancer Have a Future? What's Next? Where Will We Be in 2025?

A conversation with Nobel Laureate James P. Allison, PhD, executive director of the Immunotherapy Platform at MD Anderson Cancer Center, and Carl H. June, MD, director of the Center for Cellular Immunotherapies, Perelman School of Medicine, University of Pennsylvania. The conversation will be moderated by Siddhartha Mukherjee, MD, founder, Myeloid Therapeutics, Pulitzer-prize winning author of "The Emperor of All Maladies."

Evolving Cell and Gene Regulatory and Manufacturing Processes to Match the Speed of Innovation

Panel discussion with: Rafael Amado, MD, executive vice president, head of R&D and chief medical officer, Allogene Therapeutics; Andre Choulika, PhD, chairman, CEO and founder, Cellectis; Amy DuRoss, founder and CEO, Vineti; Bruce Levine, PhD, Barbara and Edward Netter professor in Cancer Gene Therapy, University of Pennsylvania; Ke Liu, MD, PhD, senior vice president and head of Regulatory Affairs & Strategy at Sana Biotechnology, former FDA Oncology Center of Excellence associate director for Cell and Gene Therapy; and moderated by Asthika Goonewardene, managing director, Equity Research - Biotech, Truist Securities.

Innovating Science, From the Lab to the Clinic

Panel discussion with: Brian Brown, PhD, professor and associate director of the Precision Immunology Institute, Icahn School of Medicine, Mount Sinai; Yvonne Chen, PhD, associate professor of Microbiology, Immunology & Molecular Genetics, UCLA, Founder Kalthera; Franco Marincola, PhD, senior vice president and global head of Cell Therapy Research at Kite, a Gilead Company; Clodagh O'Shea, PhD, professor, Molecular and Cell Biology Laboratory, Salk Institute, scientific founder IconOVir Bio; and moderated by Jenna Foger, senior vice president, Science & Technology, Alexandria Venture Investments/Alexandria Real Estate Equities.

Tackling the Toughest Challenges Pancreatic Cancer

Panel discussion with: Sidi Chen, PhD, associate professor, Yale Cancer Center; Joseph A. Fraietta, PhD, director, Solid Tumor Immunotherapy Laboratory, Center for Advanced Cellular Therapeutics, University of Pennsylvania; Michael T. Lotze, MD, professor of Surgery and Bioengineering, University of Pittsburgh, chief cellular officer, Nurix, chair of Alliance for Cancer Gene Therapy's Scientific Advisory Council; Mark H. O'Hara, PhD, assistant professor of Medicine at the Hospital of the University of Pennsylvania; and moderated by Andrew Rakeman, PhD, vice president of Research, Lustgarten Foundation.

Tackling the Toughest Challenges Glioblastoma

Panel discussion with: Nduka Amankulor, MD, department of Neurosurgery, University of Pittsburgh Medical Center; Samantha Bucktrout, PhD, senior director of Research, Parker Institute of Cancer Immunotherapy; Noriyuku Kasahara, MD, PhD, principal investigator, Brain Tumor Center, UCSF; Crystal Mackall, MD, founding director, Stanford Center for Cancer Cell Therapy; and moderated by Klaus Veitinger, MD, PhD, venture partner OrbiMed.

New Approaches to Solid Tumor Breakthroughs

Panel discussion with: Ken Drazan, MD, chief executive officer, ArsenalBio; Daniel Getts, PhD, chief executive officer & co-founder, Myeloid Therapeutics; Garry E. Menzel, PhD, president and chief executive officer at TCR Therapeutics; and moderated by Luke Timmerman, founder and editor, The Timmerman Report.

Patients, Caregivers, Doctors, Oncologists, Advocates, Researchers, You, Me We all Have Cancer in Common

Panel discussion with: Caroline Corner, PhD, managing director, Westwicke; Robert Levis, director CLL Society; Callum Miller, CAR-T recipient, patient advocate; Tom Whitehead, co-founder, The Emily Whitehead Foundation; and moderated by Gregory C. Simon, former president of the Biden Cancer Initiative.

Innovating Finance, Non-Traditional Funding Sources

Panel discussion with: Jay Campbell, managing director of Cancer Research Institute Clinical Accelerator and Venture Fund; Marc Hurlbert, PhD, chief science officer, Melanoma Research Alliance; Ken Schaner, partner Schaner & Lubitz; Anna Turetsky, PhD, principal, Venture Investments, The Mark Foundation for Cancer Research; moderated by Luke Evnin, PhD, co-founder and managing director MPM Capital.

"It's so rare to have the opportunity to hear from the world's top cancer researchers, biotechs, investors and patients all in the same room," noted Barbara Lavery, chief program officer for Alliance for Cancer Gene Therapy. "We're excited to have Alliance for Cancer Gene Therapy be a catalyst for these important conversations that will help not only other scientists working on new cell and gene therapy approaches, but companies seeking investment to advance their clinical pipelines, and patients and their families looking for potentially life changing therapeutic options in their fight against cancer."

To learn more, see the list of speakers and panels and register for the event, visit the website Alliance for Cancer Gene Therapy Summit 2021.

Alliance for Cancer Gene Therapy

For 20 years, Alliance for Cancer Gene Therapy has funded research that is bringing innovative treatment options to people living with deadly cancers treatments that save lives and offer new hope to all cancer patients. Alliance for Cancer Gene Therapy funds researchers who are pioneering the potential of cancer cell and gene therapy talented visionaries whose scientific advancements are driving the development of groundbreaking treatments for ovarian, prostate, sarcoma, glioblastoma, melanoma and pancreatic cancers. 100% of all public funds raised by Alliance for Cancer Gene Therapy directly support research and programs. For more information, visit acgtfoundation.org, call 203-358-5055, or join the Alliance for Cancer Gene Therapy community on Facebook, Twitter, LinkedIn, Instagram and YouTube @acgtfoundation.

Media Contact

Jenifer Howard, J. Howard Public Relations, +1 (203) 273-4246, jhoward@jhowardpr.com

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Gene therapy startup StrideBio signs collaboration deal with Duke University – WRAL Tech Wire

April 17th, 2021 1:52 am

RESEARCH TRIANGLE PARK StrideBio, an emerging startup focusing on gene therapies which closed on a big $81.5 million round of venture capital in March, is partnering with Duke University as it expands efforts to create next-generation therapies.

The agreement announced Wednesday is described as a multi-technology collaboration.

Specific financial terms were not disclosed but includeequity, upfront and milestone-driven payments, and sponsored research commitments from StrideBio to Duke University. Also included are royalties on future product sales.

We are very excited to partner with Duke University to advance these technologies that can improve and expand on the potential benefits of gene therapies for patients who desperately need them, stated Sapan Shah, StrideBios CEO. We look forward to working together with a fantastic group of Duke researchers and clinicians to bring next-generation AAV-based gene therapies to patients with rare CNS diseases and beyond, starting with Alternating Hemiplegia of Childhood.

StrideBio is focused on what it calls engineered viral vectors, or AAV, for gene therapy. The firm has already announced it has struck a deal with Crispr Therapeutics to develop in vivo gene delivery applications. As part of the deal, StrideBio will receive development funding, milestones and royalties on licensed vectors, and retain certain rights to use the novel AAV vectors for gene therapy applications.

Heres how StrideBio describes the agreement and the tecnologies involved:

The agreements announced today provide StrideBio an exclusive license to multiple technologies that will enable best-in-class next-generation gene therapies developed at Duke University. Included are novel engineered AAV vectors which complement StrideBios existing STRIVETMcapsid engineering platform, having been selected through a cross-species evolution that results in significantly enhanced tropism and potency versus AAV9 across a wide range of tissues such as CNS, skeletal and cardiac muscle. Data on these novel vectors were presented by Duke researcher and StrideBio co-founder, Aravind Asokan, Ph.D., at the American Society of Gene & Cell Therapy 23rdAnnual Meeting in an abstract titled Cross Species Evolution of Synthetic AAV Strains for Clinical Translation (Gonzalez et al., ASGCT 2020, Abstract 24). In addition, StrideBio has licensed exclusive rights covering a novel use of IgG-degrading enzyme IdeZ to clear neutralizing antibodies in conjunction with AAV gene therapy administration. This innovative approach was recently published by members of the Asokan Lab in a manuscript titled Rescuing AAV gene transfer from neutralizing antibodies with an IgG-degrading enzyme (Elmore et al., JCI Insight, 2020, 5(19): e139881). Finally, StrideBio obtained license rights to a novel gene therapy approach for the treatment of AHC recently published by Duke researcher Mohamad Mikati, M.D., in a manuscript titled AAV Mediated Gene Therapy in the Mashlool, Atp1a3Mashl/+, Mouse Model of Alternating Hemiplegia of Childhood (Hunanyan et al., Human Gene Therapy, February 12, 2021).

Gene therapy startup StrideBio lands $81.5M in capital, new investors

This License and Master Sponsored Research Agreement will ensure that these innovative technologies receive the resources and expertise necessary to develop treatments that can ultimately benefit patients. We are delighted to have StrideBio as a partner on this important effort in the gene therapy area, said Robin Rasor, Executive Director of the Office of Licensing and Ventures at Duke, in the announcement.

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Cell and gene therapy services market forecast to see significant growth over the next decade – BioPharma-Reporter.com

April 17th, 2021 1:52 am

Roots Analysis has released its latest overview on theCell and Gene Therapy CROs Market (2nd Edition), 2021-2030, covering key aspects of the industry and identifying areas for potential future growth.

The opportunity is likely to be well distributed across therapeutic areas, scales of operation, types of therapies and key geographical regions, said the analysts.

The advanced therapy medicinal products (ATMPs) sector, including cell and gene therapy (CGTs) developers. is a relatively niche area, with the presence of several start-ups/small-sized developers dominating the development landscape. About 52% of cell and gene therapy developers are small players.

And, over time, said the analysts, outsourcing has become a preferred operating model for product development activity in this domain, with CROs making significant investments towards acquiring advanced technologies and the capacity to accommodate their growing clientele.

The R&D of biologics is complex and time intensive process, requiring sophisticated techniques and dedicated infrastructure, which is readily available with contract service providers. Further, these service providers adhere to the rapidly evolving regulatory environment, acting as a key enabler to the broader industrys evolution in the coming years.

The study noted that, currently, over 100 industry players claim to have the necessary capabilities to offer contract research and clinical trial support for ATMPs.

Close to 38% of the CROs engaged in this domain are large companies, having required capabilities to provide services for both types of therapies. Examples of new entrants those established since 2018 - that claim to offer clinical services include Geistek Pharma, The Discovery Labs, KCT and Longmore 60 Biotech, reported the market specialists.

Around 45% of cell and gene therapy CROs offer research related services across both, preclinical and clinical scales of operations, while some 74% of players offer CRO players offer regulatory and data management services for cell and gene therapies, according to the analysis.

Both established players and new entrants have forged several partnerships in the recent past; most of the deals were focused on cell therapy, said the analysts, citing examples of the companies that have signed multiple international deals: Accelovance, Charles River, Laboratories, and CitoxLab.

In pursuit of a competitive edge and to eventually establish themselves as one-stop shops, stakeholders are also actively consolidating their capabilities related to cell and gene therapies through mergers and acquisitions, commented the authors.

Some are pursuing such activity for geographical consolidation reasons as shown by the acquisition of Synteract by Syneos Health in December 2020, some for portfolio addition, as seen in Comparative Biosciences being bought by Genesis Biotechnology in August 2020. Others have gone down the M&A route to expand their portfolios: the acquisition of Absorption Systems by Pharmaron in December last year is a case in point, while geographic expansion was the rationale behind the acquisition of Veristat by TCTC Group in January 2020, according to the market specialists.

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TeamedOn and AGTC Announce a Licensing Agreement Advancing X-Linked Retinoschisis Gene Therapy Program – BioSpace

April 17th, 2021 1:52 am

AGTC Transfers Clinical Trial Materials and Phase 1/2 Data To TeamedON

ROCKVILLE, Md., April 13, 2021 (GLOBE NEWSWIRE) -- TeamedOn International, Inc., a biotechnology company dedicated to advancing gene therapies for rare diseases, including ophthalmic indications, and Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced a licensing agreement to advance gene therapy to treat X-linked retinoschisis (XLRS), an inherited disease that causes loss of vision due to degeneration of the retina in males. Under the terms of the agreement, AGTC will provide TeamedOn with the clinical trial material, pre-clinical and clinical data generated for the development of AGTCs investigational intravitreal gene therapy candidate, rAAV2tYF-CB-hRS1.

The license between AGTC and TeamedOn re-opens the possibility of a much-needed treatment for individuals with XLRS, said Peter Mu, CEO of TeamedOn. TeamedOn is very pleased to be able to build on AGTCs efforts, carry on their investment in XLRS to bring potential benefit to patients.

Under the agreement, TeamedOn will conduct all activities required to reinitiate clinical development of the program. AGTC will be eligible to receive milestones and royalties based on clinical progress.

There is no cure for XLRS, and patients living with this disease have an urgent need for disease-modifying therapies that have the potential to stabilize and/or improve their long-term vision outcomes, said Dr. Paul Yang, Assistant Professor of Ophthalmology at the Oregon Health and Sciences University, who was a clinical investigator for AGTCs prior XLRS Phase 1/2 clinical trial and recently engaged by TeamedOn. Intravitreal injection of this gene therapy for XLRS previously demonstrated a reasonable safety profile. When administered through subretinal injection as TeamedOn is planning, this investigational gene therapy may have an increased likelihood of producing detectable biological activity.

AGTC discontinued its XLRS clinical program and development of rAAV2tYF-CB-hRS1 in 2018 because defined efficacy endpoints were not met using intravitreal injection.

We are excited at the prospect of TeamedOn taking a license for this program to explore the opportunity for subretinal injection of rAAV2tYF-CB-hRS1 to provide potential clinical benefit to mitigate the devastating impact on the vision of individuals with XLRS for whom there are no effective treatments, said Sue Washer, President and CEO of AGTC.

Forward-Looking StatementsThis release contains forward-looking statements that reflect AGTC's plans, estimates, assumptions and beliefs, including statements regarding the licensing agreement between AGTC and TeamedOn International, Inc. for AGTC to provide TeamedOn with the clinical trial material, pre-clinical and clinical data generated for the development of AGTCs investigational intravitreal gene therapy candidate, rAAV2tYF-CB-hRS1. Forward-looking statements include information concerning possible or assumed future results of operations, financial guidance, business strategies and operations, preclinical and clinical product development and regulatory progress, potential growth opportunities, potential market opportunities, the effects of competition and the impact of the COVID-19 pandemic, including the impact on its ability to enroll patients. Forward-looking statements include all statements that are not historical facts and can be identified by terms such as "anticipates," "believes," "could," "seeks," "estimates," "expects," "intends," "may," "plans," "potential," "predicts," "projects," "should," "will," "would" or similar expressions and the negatives of those terms. Actual results could differ materially from those discussed in the forward-looking statements, due to a number of important factors. Risks and uncertainties that may cause actual results to differ materially include, among others: gene therapy is still novel with only a few approved treatments so far; AGTC cannot predict when or if it will obtain regulatory approval to commercialize a product candidate or receive reasonable reimbursement; uncertainty inherent in clinical trials and the regulatory review process; risks and uncertainties associated with drug development and commercialization; the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, results of operations, and financial condition; factors that could cause actual results to differ materially from those described in the forward-looking statements are set forth under the heading "Risk Factors" in our most recent annual or quarterly report and in other reports we have filed with the SEC. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Also, forward-looking statements represent management's plans, estimates, assumptions, and beliefs only as of the date of this release. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the reasons actual results could differ materially from those anticipated in these forward-looking statements, even if new information becomes available in the future.

About TeamedOnTeamedOn International, Inc. (TeamedOn) (https://www.TeamedOn.com) is advancing gene therapies for rare diseases and currently focused on ophthalmic indications. We are developing innovative medicine to treat inherited retinal disorders (IRDs) and other eye diseases with significant unmet medical need. In addition to our internal R&D programs, we also seek partners for collaboration or co-development. Our goal is to restore sight and prevent vision loss for patients all around the world. TeamedOn is privately funded and located in Rockville, MD.

About AGTCAGTC is a clinical-stage biotechnology company developing genetic therapies for people with rare and debilitating ophthalmic, otologic and central nervous system (CNS) diseases. AGTC is a leader in designing and constructing all critical gene therapy elements and bringing them together to develop customized therapies that address real patient needs. AGTCs most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 and ACHM CNGA3). Its preclinical programs build on the Companys industry leading AAV manufacturing technology and scientific expertise. AGTC is advancing multiple important pipeline candidates to address substantial unmet clinical need in optogenetics, otology and CNS disorders. In recent years AGTC has entered into strategic partnerships with companies including Otonomy, a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, and Bionic Sight, an innovator in the emerging field of optogenetics and retinal coding.

TeamedOn PR CONTACTS:Peter Mu, PhD, MBACEOTeamedOn International, Inc.(301) 461-1617Peter_Mu@teamedon.com

AGTC IR/PR CONTACTS: David Carey (IR) or Glenn Silver (PR)Lazar FINN PartnersT: (212) 867-1768 or (646) 871-8485david.carey@finnpartners.com or glenn.silver@finnpartners.com

AGTC Corporate Contacts:Bill SullivanChief Financial OfficerApplied Genetic Technologies CorporationT: (617) 843-5728bsullivan@agtc.com

Stephen PotterChief Business OfficerApplied Genetic Technologies CorporationT: (617) 413-2754spotter@agtc.com

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Jaguar Gene Therapy gains $139 million from investors – Daily Herald

April 17th, 2021 1:52 am

LAKE FOREST -- Jaguar Gene Therapy said it has raised $139 million through its most recent round of investments, led by Eli Lilly and Co. and Deerfield Management.

Also participating in the round were Arch Venture Partners, co-founded by Robert Nelsen and one of the largest early-stage technology venture firms in the United States; Goldman Sachs; and Nolan Capital, the investment fund of former AveXis CEO and current Jaguar Executive Chairman Sean P. Nolan.

Jaguar Gene Therapy is a company accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases, including galactosemia, genetic causes of autism spectrum disorder, Type 1 diabetes and Bardet-Biedl syndrome.

"We are thrilled to have attracted this prestigious group of visionary investors, which affirms our strategic approach, promising pipeline programs, and unique collaboration with Deerfield Management," said Joe Nolan, chief executive officer of Jaguar Gene Therapy. "With this capital infusion, we are well positioned to execute on our mission to accelerate breakthroughs in gene therapy and bring them to children and adults with severe genetic diseases."

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Medically Necessary: Growth of biologics and gene therapies means more cold chain – FreightWaves

April 17th, 2021 1:52 am

This is an excerpt from the April 13, 2021 edition of Medically Necessary, a health care supply chain newsletter.Subscribe here.

The trend: The number of biologic drugs and gene therapies is growing, and logistics companies are increasing their capacity for temperature-controlled transportation to support them.

Biologics are drugs created from biological sources, such as blood or other cells. Theyre usually much more complex than drugs produced chemically.

Gene therapies, which can be biologics, are drugs that replace or modify a patients gene to treat a disease.

Drugs in both categories require temperature to be carefully controlled during transportation.

The future: About a quarter of all drugs currently require some kind of temperature control during transportation, according to RanjeetBanerjee, CEO of the pharmaceutical logistics company Cold Chain Technologies.

He expects that number to jump to about a third of all drugs within the next two years.

With the launch of biologics, with the launch of personalized medicine and certain gene therapy formulations, that are happening now, the need for temperature and other condition monitoring is becoming even more acute, he said.

Biologics: The market for temperature-sensitive biologics is growing faster than that of other types of drugs.

Spending on biologics through Medicare Part B, which covers drugs that patients recieve at a doctors office or hospital, jumped from about 56% of total drug spending in 2006 to 77% in 2017, according to a Health and Human Services Department report.

In large part, that growth is because biologic drugs tend to be very expensive. The report found that biologics accounted for almost all of the programs drug spending growth during those years.

The number of patients receiving biologics also appears to be increasing.

Data from Medicare Part D, which covers prescription drugs, shows that the number of beneficiaries receiving a group of seven common biologics increased by more than 75% from 2011 to 2018.

A 2017 report from The Biosimilars Council predicts that the growth of biosimilars cheaper alternatives that have the same clinical effects as biologics will further expand access to these types of treatments.

Like biologics, many biosimilars require temperature control during transportation and storage.

Gene therapies: The cold chain logistics company Cryoport expects the value of the gene therapy market to increase sevenfold between 2019 and 2024.

FDA approved the first gene therapy in the U.S. in 2017 and has approved six more treatments since then.

The agency approved two of those seven treatments in the first quarter of 2021 alone.

In 2019, former FDA Commissioner Scott Gottlieb said he expected the agency to be approving 10 to 20 cell and gene therapy products every year by 2025.

This trend has a couple of logistics implications, Cedric Picaud, CEO of the Cryoport company CRYOPDP told FreightWaves. The value of each shipment is very high. The cost of managing such medication is high. In terms of logistics, the lead time requirement is very challenging.

The response: As the number of biologic drugs and gene therapies continues to grow, logistics companies are increasing their capacity for temperature-controlled transportation.

In March, Cold Chain Technologies, which previously focused on packaging, expanded the scope of its business.

The company will now offer last-mile delivery services, warehousing and point-of-care storage for temperature-controlled drugs. The goal is to provide logistics from the manufacturer to the patient, Banerjee said.

Over the past two years, Cryoport has acquired several cold chain logistics companies to expand its reach.

Cryoport acquired an Australian logistics company in April. That follows the acquisition of two cold chain logistics companies in August 2020 and another acquisition in May 2019.

The company grew from 57 employees at the end of 2018 to more than 625 by the end of 2020.

Tribe Transportation, a temperature-controlled trucking company based in Georgia, recently added more than 100 trailers to its fleet to meet demand from the life sciences industry.

There are still life sciences customers out there looking for capacity to move shipments, Executive Vice President Matt Handte told FreightWaves. Post-vaccine, weve seen that continue to rise.

The past three years have been the companys best years for growth of both revenue and fleet size, Handte said.

The virus: The coronavirus vaccine rollout has put the cold chain in the spotlight, but companies dont expect this to be a flash in the pan.

Cold Chain Technologies provides packaging for COVID-19 vaccines made by Moderna and Johnson & Johnson. The company opened a new manufacturing plant in Tennessee last year to meet the needs of the vaccine rollout, but Banerjee doesnt expect that capacity to go to waste.

If we plan this carefully, we could use this capacity to support COVID vaccinations outside the U.S., which will have a longer ramp down as well as taking care of the new drugs and biologics, he said.

Whats next? Banerjee said he hopes the vaccine rollout will help logistics companies do a better job of transporting temperature-controlled drugs after the pandemic.

How do we make sure we transform the cold chain so that the next time this happens we are way ahead? he said. And use all that learning for the day-to-day shipments of drugs and pharmaceuticals.

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Taysha Gene Therapies Announces New Data on Multiple Preclinical Programs and Upcoming R&D Day – BioSpace

April 17th, 2021 1:52 am

DALLAS--(BUSINESS WIRE)-- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric, pivotal-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS) in both rare and large patient populations, today announced new data for multiple preclinical programs and a planned R&D Day, which will be held in June 2021.

Collectively, these new preclinical data highlight Tayshas next wave of novel gene therapies that have the potential to impact meaningful patient populations. The promising data underscore our ability to rapidly and reproducibly investigate disease biology, design innovative gene therapies and efficiently advance the development of these drug candidates, said RA Session II, President, Founder and Chief Executive Officer of Taysha. Among the compelling new data, for the first time, we have shown that TSHA-113, an AAV9 gene therapy that utilizes AAV-mediated gene silencing, reduced tau expression in mouse models of human tauopathies. The potential implications of these data are far reaching, and we intend to further evaluate TSHA-113 in additional preclinical studies. The totality of the preclinical data presented today support the fundamental elements of our scientific approach of coupling validated technology with novel targeted payload design while utilizing a proven HEK293 suspension manufacturing process. We believe our deep pipeline and innovative scientific engine hold tremendous potential, and we are poised to continue delivering meaningful value to patients with monogenic CNS diseases.

Todays data demonstrate the breadth, depth and velocity of our development engine as a sustainable pivotal-stage gene therapy company. There are no approved disease modifying therapies for any of the programs in our portfolio and we are encouraged by the results of our gene therapy approach of vectorized RNA and gene replacement therapies across our portfolio, said Suyash Prasad, MBBS, M.SC., MRCP, MRCPCH, FFPM, Chief Medical Officer and Head of Research and Development of Taysha. We are very excited to further develop TSHA-113 in tauopathies, including Alzheimers disease, MAPT-associated frontotemporal dementia and progressive supranuclear palsy, based on the significant reduction in tau expression demonstrated in transgenic mouse models of human tauopathies. In addition, to date, we have advanced five programs into IND/CTA-enabling studies, including TSHA-105 in SLC13A5 deficiency, TSHA-111-LAFORIN in Lafora disease, TSHA-111-MALIN in Lafora disease, TSHA-112 in APBD and TSHA-119 in GM2 AB variant. We intend to file an IND/CTA for one of these five named programs by the end of 2021. By mid-year, we intend to select a development candidate for Angelman syndrome and obtain interim expression and safety data from confirmatory non-human primate studies by year-end. We remain on track to report Phase 1/2 biomarker data for TSHA-101 in GM2 gangliosidosis in the second half of this year and to provide a clinical and regulatory update for TSHA-120 in giant axonal neuropathy by year-end. Finally, in the second half of the year, we continue to expect dosing of the first patient with CLN1 disease in a Phase 1/2 trial for TSHA-118 under an already open IND, filing an IND/CTA for TSHA-102 in Rett syndrome and TSHA-104 in SURF1-associated Leigh syndrome, and filing an IND for TSHA-101 in GM2 gangliosidosis in the U.S. These anticipated clinical and regulatory milestones are expected to be followed by the initiation of Phase 1/2 clinical trials for each of these indications. We look forward to providing additional updates at our R&D Day in June.

TSHA-113 for Tauopathies

Taysha is developing tau-specific microRNA (miRNA) shuttles designed to target tau mRNA for all six isoforms found in the human brain and/or mouse brain. TSHA-113 is an AAV9 capsid that packages these miRNA shuttles and is delivered in the CSF for the treatment of tauopathies.

TSHA-105 for SLC13A5 deficiency

TSHA-105 is a recombinant self-complementary AAV9 vector that expresses the human SLC13A5 protein under the control of a ubiquitous promoter. The drug candidate is being developed for the treatment of SLC13A5 deficiency.

TSHA-106 for Angelman syndrome

TSHA-106 is an intrathecally delivered AAV9 viral vector designed for shRNA-mediated knockdown of UBE3A-ATS, the antisense transcript governing the expression of UBE3A through the paternal allele.

TSHA-112 for Adult Polyglucosan Body Disease (APBD)

TSHA-112 is an intrathecally delivered AAV9 viral vector designed for miRNA-mediated knockdown of the GYS1 gene to treat APBD.

TSHA-111-LAFORIN for EPM2A and TSHA-111-MALIN for EPM2B for Lafora disease

TSHA-111-LAFORIN and TSHA-111-MALIN are intrathecally delivered AAV9 viral vectors designed for miRNA-mediated knockdown of the GYS1 gene to treat Lafora disease.

TSHA-119 for GM2 AB variant

TSHA-119 is a self-complementary AAV9 vector designed to deliver a functional copy of the GM2A gene to treat GM2 AB variant.

About Taysha Gene Therapies

Taysha Gene Therapies (Nasdaq: TSHA) is on a mission to eradicate monogenic CNS disease. With a singular focus on developing curative medicines, we aim to rapidly translate our treatments from bench to bedside. We have combined our teams proven experience in gene therapy drug development and commercialization with the world-class UT Southwestern Gene Therapy Program to build an extensive, AAV gene therapy pipeline focused on both rare and large-market indications. Together, we leverage our fully integrated platforman engine for potential new cureswith a goal of dramatically improving patients lives. More information is available at http://www.tayshagtx.com.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as anticipates, believes, expects, intends, projects, and future or similar expressions are intended to identify forward-looking statements. Forward-looking statements include statements concerning the potential of our product candidates, including our preclinical product candidates, to positively impact quality of life and alter the course of disease in the patients we seek to treat, our research, development and regulatory plans for our product candidates, the potential for these product candidates to receive regulatory approval from the FDA or equivalent foreign regulatory agencies, and whether, if approved, these product candidates will be successfully distributed and marketed, and the potential market opportunity for these product candidates. Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business are described in detail in our Securities and Exchange Commission (SEC) filings, including in our Annual Report on Form 10-K for the full-year ended December 31, 2020, which is available on the SECs website at http://www.sec.gov. Additional information will be made available in other filings that we make from time to time with the SEC. Such risks may be amplified by the impacts of the COVID-19 pandemic. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.

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Taysha Gene Therapies Announces New Data on Multiple Preclinical Programs and Upcoming R&D Day - BioSpace

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From gene therapies to medical treatments, cutting-edge research finds a home in NC – WRAL.com

April 17th, 2021 1:52 am

By Abbey Slattery, WRAL Digital Solutions

This article was written for our sponsor, the North Carolina Biotechnology Center.

For decades, North Carolina has been known as a nerve center for the life sciences industry. In fact, after the creation of the Biotech Center in the 1980s, North Carolina became the first state in the country to have a state-supported entity dedicated to growing the industry.

N.C. Biotech runs a number of programs to support technology and company development, to recruit companies and to connect North Carolina residents to high-paying jobs.

In the years since then, numerous life sciences companies have established a base in North Carolina, including major names like Biogen, BASF, LabCorp and Merck, among others. By committing to the industry early and cultivating a strong university system, the state established itself as a hub and prepared a workforce that rivals that of anywhere else in the country. Now, it's the site of global scientific breakthroughs and game-changing medical advancements.

"North Carolina has really done a good job with creating a flow from the workforce to the industry, and we've continued to grow and expand because of that. We now have a lot of research and development tools, and we have a tremendous amount of manufacturing here," said Laura Gunter, executive vice president at NCBIO. "All of the amenities of North Carolina and climate and education attract people, but then we've done a good job of educating folks and giving North Carolinians an opportunity to work and thrive here."

With a highly trained workforce of over 67,000, 775 diverse bioscience companies and nearly 2,500 companies, the state has created a thriving ecosystem for the industry. In the Triangle region alone, agricultural and biopharma manufacturing companies are developing exciting new advancements with potential for global impacts.

"A company called Pairwise in Durham is focused on using the gene-editing technology called CRISPR, but with plants. They're working on developing more nutrients or better growing capabilities with plants using that CRISPR technology," said Gunter. "They've developed a new variety of leafy green called brassica that's related to Brussels sprouts, cauliflower and kale. It's nutrient-dense and naturally resistant to pests, but apparently, it has a very pungent flavor. What they've done is work on the flavor, while keeping the nutrients and resistance."

"Another major company is Novozymes, and they specialize in industrial enzymes and microorganisms. If you've ever toured any of these facilities where they're doing fermentation, like a beer brewing facility, Novozymes does that on a massive scale with industrial enzymes," continued Gunter. "Those enzymes can be used in everything from pharmaceuticals to food to detergent. They're also working on some things on the agriculture side to improve crop yields through microbes."

Biogen founded in 1978 and one of the world's first global biotechnology companies is another major player that finds a home in Durham. While Pairwise and Novozymes are more focused on the agricultural side of biotech, Biogen is making advancements in pharmaceutical manufacturing and medical research programs. Its active research includes treatments for conditions like multiple sclerosis, spinal muscular atrophy and other serious neurological conditions.

The company announced a $200 million expansion in March to produce gene therapies and other treatments in their project pipeline. It joins Novartis Gene Therapies, which is producing its Zolgensma therapy for spinal muscular atrophy in Durham, alongside Adverum, Audentes, Beam Therapeutics, bluebird bio, Cellectis, Pfizer, Taysha Gene Therapies and more.

The benefit of cell and gene therapies is the treatments are potentially curative. Early applications are for diseases caused by single-gene mutations, and those that are life-threatening. Thanks to these companies, North Carolina will be the place these treatments are produced. The treatments would likely have a global impact on improving the lives of those who suffer.

One specific example is spinal muscular atrophy, which is the loss of nerve cells and the weakening of muscles near the spine. This degenerative disease has previously been a fatal diagnosis for infants.

"The fascinating thing about spinal muscular atrophy treatments, in the case of Biogen and Novartis, they're both game-changing and potentially curative treatments versus just trying to alleviate a symptom," said Gunter. "With the gene replacement therapy that Novartis is using, if it works, then that child could potentially no longer have the disease. If it ends up being a long-term fix, it's pretty miraculous and it's made right here."

The list of companies that develop products and treatments in North Carolina goes on from endoscopy devices at Cook Medical to in vitro products at bioMerieux to injectable drugs at Hospira. Thanks to forward-thinking from the state and organizations like N.C. Biotech, North Carolina is well-equipped to continue being the site of life sciences products and advancements.

"North Carolina has paid attention to reinforcing the entire pipeline starting with idea and proof of concept by a university researcher. Small companies are created from the research, and we help them navigate a bumpy road to success. Then, we work with companies all around the world looking for the right place to manufacture their products," said Doug Edgeton, president and CEO of N.C. Biotech.

"We continue to invest in this innovation infrastructure, and train the talent to do these important jobs. We think it's why North Carolina is a global leader in this field."

This article was written for our sponsor, the North Carolina Biotechnology Center.

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From gene therapies to medical treatments, cutting-edge research finds a home in NC - WRAL.com

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University of Cambridge research suggests gene therapies offer hope in treating glaucoma and dementia – Cambridge Independent

April 17th, 2021 1:52 am

Promising research at the University of Cambridge suggests gene therapies could help repair some of the damage caused in chronic neurodegenerative conditions such as glaucoma and dementia, writes editor Paul Brackley.

Their studies in animals offer hope that such therapies could be effective in polygenic conditions, which are complex and do not have a single genetic cause.

Gene therapies involve replacing a missing or defective gene with a healthy version.

They have become increasingly common, but typically for rare and monogenic conditions those caused by a single defective gene, such as Lebers congenital amaurosis, spinal muscular atrophy and Lebers hereditary optic neuropathy.

There have been limited applications of such therapies to polygenic conditions, which include the majority of neurodegenerative conditions.

In their new research, Cambridge scientists delivered two candidate molecules simultaneously to nerve cells using a single virus to achieve a strong effect on axonal growth.

Dr Tasneem Khatib, from the John van Geest Centre for Brain Repair at the University of Cambridge, the studys first author, explained: The axons of nerve cells function a bit like a railway system, where the cargo is essential components required for the cells to survive and function.

In neurodegenerative diseases, this railway system can get damaged or blocked. We reckoned that replacing two molecules that we know work effectively together would help to repair this transport network more effectively than delivering either one alone, and that is exactly what we found.

This combined approach also leads to a much more sustained therapeutic effect, which is very important for a treatment aimed at a chronic degenerative disease.

Rather than using the standard gene therapy approach of replacing or repairing damaged genes, we used the technique to supplement these molecules in the brain.

Axons are long fibres that transmit electrical signals, allowing nerve cells to communicate with one another and with muscles.

Axonal transport is a cellular process that moves key molecules and cellular building blocks such as mitochondria, lipids and proteins to and from the body of a nerve cell.

It has been suggested that stimulating this process by enhancing intrinsic neuronal processes in the diseased central nervous system could be a way of repairing damaged nerve cells.

The Cambridge team explored this idea using two candidate molecules for improving axonal function brain-derived neurotrophic factor (BDNF) and its receptor, tropomyosin receptor kinase B (TrkB).

They tested the idea in two models of neurodegenerative disease known to be associated with reduced axonal transport glaucoma and tauopathy, a degenerative disease associated with dementia.

Glaucoma involves damage to the optic nerve and is typically associated with abnormally high pressure in the eye.

Using rats, researchers deployed a tracer dye to show axonal transport between the eye and brain was impaired in glaucoma, while a reduction in electrical activity in the retina in response to light suggested vision was also impaired.

Dr Khatib and colleagues then used viral vectors which are gene therapy delivery systems to deliver the two molecules to the retina of rats. The movement of the dye showed this restored axonal transport between the retina and the brain and the retinas also showed an improved electrical response to light, which is a prerequisite for visual restoration.

Next they used transgenic mice bred to model tauopathy, which is the build-up of tangles of tau protein in the brain.

Tauopathy is seen in neurodegenerative diseases, including Alzheimers disease and frontotemporal dementia.

Injecting the dye again enabled the team to show that axonal transport was impaired between the eye and the brain and that it was restored using the viral vectors.

They even found preliminary evidence of possible improvement in the mices short-term memory using an object recognition task.

Before the treatment, a mouse was placed at the start of a Y-shaped maze and left to explore two identical objects at the end of the two arms.

Shortly after, the mouse was once again placed in the maze, but this time one arm contained a new object, while the other contained a copy of the repeated object.

The researchers measured the amount of the time the mouse spent exploring each object to see whether it had remembered the object from the previous task.

After the viral vector had been injected into the mouses brain, the test was repeated and suggested a small improvement in short-term memory. The result here was not deemed statistically significant, but the researchers were encouraged and plan a larger study to confirm it.

Professor Keith Martin, from the Centre for Eye Research Australia and the University of Melbourne, who led the study while at Cambridge, said: While this is currently early stage research, we believe it shows promise for helping to treat neurodegenerative diseases that have so far proved intractable.

Gene therapy has already proved effective for some rare monogenic conditions, and we hope it will be similarly useful for these more complex diseases which are much more common.

In the study, published in Science Advances, the researchers write: We feel that these findings do have implication for clinical practice.

The research was supported by Fight for Sight, Addenbrookes Charitable Trust, the Cambridge Eye Trust, the Jukes Glaucoma Research Fund, Quethera Ltd, Alzheimer's Research UK, Gates Cambridge Trust, Wellcome and the Medical Research Council.

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University of Cambridge research suggests gene therapies offer hope in treating glaucoma and dementia - Cambridge Independent

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What is AAV Gene Therapy and What Makes it Safe? – Freethink

April 17th, 2021 1:52 am

Genetic diseases were thought to be incurable letters carved in stone (or on a double helix). But gene therapy could change all that.

From treating Duchenne muscular dystrophy, autism, or glaucoma, there has been a surge in the news surrounding gene therapy (type "gene therapy" into the Freethink search tool, and you'll see endless stories about medical breakthroughs).

The total number of completed or ongoing gene therapy clinical trials reached 4000 in February 2020. This type of treatment is on its way toward addressing some of the most severe and complicated ailments known to man. Gene therapy even shows promise for treating diseases like cancer or Huntington's disease longstanding mysteries of medical science.

Instead of using medications or surgery, doctors can use gene therapy to fight diseases at their source. Gene therapy attempts to cure a disease, or boost the body's ability to combat a disease, by replacing a defective gene or adding a new gene.

The success of gene therapy all hinges on the ability to deliver that precious genetic cargo into the patient's cells safely. To do that, scientists use a "vector" as a delivery vessel for the genetic material. In most cases, that vector is a virus.

Scientists engineer an unwitting virus to act as a vector to deliver a therapeutic genetic payload.

We don't typically think fondly of viruses. Usually, they don't enter the human body to help. Instead, viruses like the flu or coronavirus make humans sick by inserting their genetic material into human cells and hijacking them. The cell reads the viral DNA (or RNA) and produces virus offspring, infecting other cells, repeating the process.

But, since viruses naturally deliver genetic material into a human cell, they are the most widely used vectors in gene therapy.

Scientists engineer an unwitting virus to act as a vector to deliver a therapeutic genetic payload. They strip the virus of its own genes and replace it with new genes. Once the virus transports its genetic cargo into tissues, the altered genes get to work producing further instructions to treat the disease.

But the human body can recognize the virus as a foreign invader and launch an immune response attempting to target and reject the virus.

Enter the adeno-associated viruses (AAV) a small virus that has emerged as the most promising platform for gene therapy.

These small, versatile viruses have several advantages over other gene therapy vectors.

First, despite their small size, they can carry a sizeable genetic payload. They are not known to cause many human diseases, and they can be designed to target particular types of cells or tissues. They cannot replicate on their own, so scientists can control how much gene therapy is delivered to the body.

The best part: they don't trigger a very large immune response like other viruses. A gene therapy trial in the 1990s that used an adenovirus (a kind of cold virus, different from AAVs) caused a deadly immune overreaction in one patient and effectively ended gene therapy research in humans for a decade.

After that, researchers went back to the drawing board, looking for safer vectors. AAVs, because they don't cause disease and can't replicate, generally fly under the immune system's radar. These characteristics make them perfect vessels for gene therapy. And clinical trials have since shown that AAV gene therapy is safe and, for some diseases, effective.

Now that AAVs are established as gene therapy vectors, scientists are working to improve their delivery and create the next generation of AAV gene therapy.

Researchers have been successful with AAV gene therapy in the clinical setting. Currently, two FDA-approved gene therapy treatments use AAVs: Luxturna, which was approved in 2017 for a rare vision disorder, and Zolgensma, which was approved last year for spinal muscular atrophy.

But there are challenges to overcome. Because AAVs are viruses that people are naturally exposed to, many people have already developed antibodies against them. When a patient who has been previously exposed to an AAV receives AAV gene therapy, these antibodies could block the therapy.

Recently, a team of researchers figured out how to cloak the AVV to sneak past the immune system and deliver its gene therapy payload without triggering any immune response.

With the recent surge in AAVs as viral vectors and more clinical studies in the pipeline, we can expect to see new gene therapies that are safe and effective.

We'd love to hear from you! If you have a comment about this article or if you have a tip for a future Freethink story, please email us at [emailprotected]

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What is AAV Gene Therapy and What Makes it Safe? - Freethink

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ATMPS and Ori Biotech Collaborate on an Integrated Digital Platform for Cell and Gene Therapy Developers – BioSpace

April 17th, 2021 1:52 am

The collaboration will improve visibility and reporting of cell and gene therapy manufacturing through a pre-integrated data solution

London, UK and Woodcliff Lake, NJ, US, April 14, 2021 ATMPS Ltd, a leader in blockchain-based vein to vein cell orchestration platforms for advanced therapies, and Ori Biotech, an innovator in cell and gene therapy (CGT) manufacturing platforms, today announce a global collaboration to create seamless integration between their respective data platforms to support the development of Advanced Therapies.

The non-exclusive partnership will result in the integration of Oris proprietary CGT manufacturing platforms data architecture with ATMPS HataaliTM, a secure data sharing platform, specially designed as a scalable system for advanced therapies. The combination of the two proprietary platforms will provide improved product tracking, scheduling as well as live monitoring of the manufacturing status across the entire CGT supply chain from vein to vein.

Ori selected ATMPS Hataali technology for integration with our platform to allow the secure sharing of relevant process data with therapy developers, contract development and manufacturing organizations (CDMOs) and even clinicians or patients in real-time. This collaboration will provide increased visibility into the treatment process for those involved at each stage of the treatment process, said Jason C. Foster, CEO, Ori Biotech. The addition of established technologies from companies like ATMPS allows Ori to further its mission of enabling partners across the CGT industry to bring therapies as safely, cost effectively and quickly as possible to large numbers of patients with few alternative therapeutic options.

Hataali encrypts and stores data using distributed ledger technology through a process known as hashing. This process ensures data security is protected through a system of private keys and cryptography. Hataali was the first cell orchestration platforminstalled in a hospital globally and ATMPS the first blockchain company to integrate with the UK National Health Service for the delivery of advanced therapies.

Ori is an ideal partner for ATMPS, as both companies share a common mission to lower the costs and improve processes for cell and gene therapies. Oris work in helping innovators scale from preclinical to commercial is a massive step towards reducing complexity and bringing these vital therapies to patients more quickly, said Raja Sharif, CEO, ATMPS. We all know how challenging and expensive the development and manufacturing of CGTs can be. With the help of Hataali, Ori can securely leverage vein-to-vein data to advance their manufacturing solutions much more efficiently. This will enable real-time therapy tracking, improved patient trust and expediated clinical development.

Ori continues to experience significant growth since closing a $30 million Series A round of funding as it prepares to bring its platform to market. The company is actively recruiting the best talent in cell and gene therapy technology through its networks and through http://www.oribiotech.com.

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About ATMPS

ATMPS Ltd provides patent pending advanced technical solutions for healthcare companies in the Advanced Therapy Medicines Products sector, including CAR-T treatments, stem cell and gene treatments. ATMPS have a unique distributed ledger solution which provides immutable and incorruptible data and automation capabilities. The solution is operational, configurable and can provide data analytics modules. It can integrate with all LIMS, MES and legacy solutions. It provides full chain of custody, identity and condition of samples, treatments and assets which it tracks. It also schedules and co-ordinates the supply chain participants, as well as giving them information at the same time, in real time, from a "single source of truth. For more information, please visit ATMPS website athttps://www.atmps.net/.

About Ori Biotech

Ori is a London and New Jersey based cell and gene therapy (CGT) manufacturing technology company. Ori has developed a proprietary, flexible manufacturing platform that closes, automates and standardizes CGT manufacturing allowing therapeutics developers to further develop and bring their products to market at commercial scale. The promise of the full stack Ori platform is to fully automate CGT manufacturing to increase throughput, improve quality and decrease costs by combining hardware, software and data services.

Ori was founded by Professor Chris Mason (AVRO) and Dr. Farlan Veraitch (UCL) and has brought together a seasoned Board and executive management team with over 100 years of pharmaceutical, CGT and venture building experience including CEO Jason C. Foster (Indivior), CBO Jason Jones (Miltenyi Biotec), VP Tom Heathman (Minaris), VP David Smith (Minaris) alongside expert advisers like Bruce Levine, Anthony Davies and Annalisa Jenkins. For more information, visit http://www.oribiotech.com.

For media enquiries, please contact:

Alex Heeley or Abdul Khalifeh

De Facto Communications

T: +44 (0) 203 735 8168

E:a.heeley@defacto.co.uk/a.khalifeh@defacto.co.uk

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ATMPS and Ori Biotech Collaborate on an Integrated Digital Platform for Cell and Gene Therapy Developers - BioSpace

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