StemCell Therapy

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In a year marked by so much uncertaintyeven far beyond a certain 43-year-olds future as a professional quarterbackone constant has remained: Tom Brady is playing in the Super Bowl.

Over 21 years in the league, there isnt much he hasnt achieved. A 14-time Pro Bowler, three-time MVP, and six-time Super Bowl champion, Brady has at one time or another led the league in touchdowns, passing yards, expected points added, defense-adjusted yards above replacement, total quarterback rating, passer rating and any other statistical category you can conceive. Hes been recognized as the Offensive Player of the Year, Comeback Player of the Year, Super Bowl MVP and 1st Team All-Pro.

If you can win it, Tom Brady probably has.

And on Sunday, hell have his chance at illustrious ring number seven, but first, hell have to get through Patrick Mahomes, his presumptive heir apparent as league standard-bearer.

To fully grasp Bradys longevity and consistency (and to see whats changed since he flew south to Tampa), its useful to see his achievements alongside his contemporaries. While others may rival his individual, per-game statistics, its hard to believe that anyone will ever amass the sustained dominance and jaw-dropping rsum that Brady has accrued over the course of his career.

Regardless of outcome, Sundays appearance will put Brady alongside Kareem Abdul-Jabbar as the only American professional team athletes in the last half-century (1971-2020) to play in 10 league finals and win more than half of them. In fact, no other NFL player has been to more than six Super Bowls (Stephen Gostkowski, Mike Lodish).

In addition to his regular attendance to the biggest game of the season, Bradys consistent yearly production over two decades has prompted some to wonder: If you chopped his time in the NFL into twoor even threechunks, would they each be individual Hall of Fame careers?

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Expected points added (EPA) measures how much better or worse a teams position on the field is from one play to the next; it provides meaningful units to measure a players total contribution to his team. As you can see in the chart below, Bradys seasonal EPA trajectory resembles that of Hall of Fame contemporaries Peyton Manning and Aaron Rodgers if they happened to share one career.

But whats even more stunning about this season is that after two decades with the same coach, owner and home field, Brady joined a new organization in an abbreviated offseason, learned and embraced a new passing philosophy and still managed to rise to the top.

From 2019 to 2020, Brady significantly reduced the frequency of his short throws (<11 air yards)the same ones completed at a league-leading 0.22 EPA/attempt during his time in New Englandand turned to Bruce Arians fabled vertical passing attack. Over the course of the regular season, Brady threw 21 more long passes (25+ yards from the line of scrimmage) than any other player in the league.

Not only is he throwing deep in a way we havent seen before, but hes doing it well; since Week 13, Bradys averaging 0.83 EPA/play on throws 15+ yards from the line of scrimmage, good for fifth in the NFL in that span and just ahead of likely MVP Aaron Rodgers (0.82).

In short, Tom Brady has taken a rsum the size of War and Peace and penned yet another chapter.

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Measure of The Man: Tom Bradys Career Numbers Show His Longevity, Versatility - Yahoo Sports

Lewis Hamiltons contract extension for 2021 was one of the least surprising developments of Formula 1s off-season but already some have suggested it may be his last dance. But is that really likely?

Lewis Hamiltons presence within Formula 1s record books is already cemented: most titles (currently held jointly with Michael Schumacher), most wins and most poles, with those figures likely to trickle into triple digits by the summer.

He is continuing to perform at an extraordinary high level as witnessed on a number of occasions in 2020: take your pick from the wet Styrian pole lap, triumphing on three wheels at Silverstone or the drive to seal the title at a grip-less Istanbul Park. There were other less memorable races, such as at Spa, Barcelona or Portimao, at which he merely pulverised the opposition.

Yet there are other statistics that are striking.

Hamilton is already Formula 1s seventh-most experienced driver, his Covid-enforced Sakhir absence concluded the longest streak of appearances at grands prix, while in claiming the title in 2020 he became the oldest champion since Damon Hill in 1996.

The focus, particularly in recent years, has been towards youth. Formula 1s youngest ever champions Fernando Alonso, then Hamilton, then Sebastian Vettel have been followed by its youngest entrant and race winner Max Verstappen whose very arrival prompted a re-writing of the regulations.

Verstappen, who debuted aged 17, is a special case but not quite an anomalous oddity. Contemporaries on the grid, Lance Stroll and Lando Norris, were the second- and fourth-youngest starters in history, while Esteban Ocon comes in at number 11, Charles Leclerc 18 and Carlos Sainz 20, out of the 767 to enter a race. Ferrari has placed its faith in Leclerc, the same age as Verstappen, while George Russell 23 next week has two years under his belt with Williams.

But all still need to displace the mercurial Hamilton, who has previously commented on how he relishes the challenge from the youngsters, from his plinth and there is no reason to expect the World Champion to walk away any time soon.

Every athlete is different but we are in the era of some of the greatest talents in any sport prolonging their astonishing careers beyond what has been widely anticipated some even into their fifth decade. They are special talents, incredible athletes, intensely focused, with an unrelenting rage to win, aided by advancements in science and technology such as a greater appreciation and understanding of elements such as nutrition, training and sleep management. No one path has been the same but the end result has been a train of success.

On Sunday Tom Brady claimed a record seventh Super Bowl title, having transferred from the New England Patriots to the Tampa Bay Buccaneers, aged 43. He has won in three different decades and now has more individual success than any franchise. Last October LeBron James, just nine days older than Hamilton, led the LA Lakers to their first NBA title in 10 years, as well as becoming the Finals MVP.

At our age, we can still dominate our sport, James is quoted by Lakers Daily last month, when referencing Brady. We have one common goal and thats to win and win at the highest level.

Tennis icons Serena Williams and Roger Federer, born seven weeks apart, will turn 40 later this year and both continue to strive to add to trophy cabinets that are overflowing with riches. Federer may currently be recuperating from surgery, and Williams has also struggled with injury, but neither is yet throwing in the towel.

Ive seen players in the locker room, the Legends tour, and at some points I was older than them and I was wondering if I should be there, joked Williams in 2017 on her longevity.

Said Federer recently, to Swiss broadcaster SRF, I like to play tennis for life. In the last few months I have given a lot in rehab. I had to go through it, but I always enjoyed it. I want to celebrate great victories again. And for that, I am ready to go the long, hard road.

Federers long-term rival, Rafael Nadal, is a 13-time French Open champion yet at 34 still hurtles around the court like a caged animal. World Number 1 Novak Djokovic plays with the elasticity of someone a decade younger than his 33 years.

Italys Serie A may not be the most fast-paced of Europes leading football leagues but at the top of the goalscoring charts are Cristiano Ronaldo, 36 exactly a month younger than Hamilton and the enigmatic Zlatan Ibrahimovic, who is 39. Across the Mediterranean the ferocious Luis Suarez, 34, is fronting La Ligas charts. In golf Tiger Woods has not had the sustained success of those just mentioned but his perseverance paid off in 2019, ending an 11-year wait for another major, by claiming the Masters aged 43.

None of this it to say that the athletes in their twenties or even teens and early thirties are lacking in ability, passion or technique. Bradys opposite number on Sunday, Patrick Mahomes, was 24 when he spearheaded Kansas City Chiefs Super Bowl triumph in 2020. NBAs MVP for the last two years, is the Milwaukee Bucks 26-year-old Giannis Antetokounmpo. Naomi Osaka, 23, is already a multi-Grand Slam champion of a talented generation seeking to emulate Williams. Collin Morikawa was 23 when he won last years PGA Championship. In Formula 1 Verstappen and Leclerc who was born on the same day as Osaka have already underlined their credentials. It is a special generation.

But sports GOATs are still going strong and showing little sign of slowing down those who adorned front covers and billboards in the 2000s (or even late 1990s) are still there in the 2020s, pushing away notions of retirement. Moving it closer to home Hamiltons two predecessors as World Champion are Fernando Alonso and Kimi Raikkonen. The returning Alonso, 39, has signed up to compete for Alpine for the next two years while Alfa Romeos Raikkonen, 41, continues to race on, 20 years after their respective Formula 1 bows. Scott Dixon, crowned IndyCar king for a sixth time, is 40, and among his team-mates this year will be NASCAR convert Jimmie Johnson, 45. Valentino Rossi, while no longer a potent front-running force, is still in MotoGP, revered worldwide, and next week turns 42. WRC champion Sebastien Ogier is 37 albeit insisting 2021 will be his swansong.

Hamiltons one-year extension was slightly surprising, given all his past deals have been multi-year arrangements, but there is reason for the relative brevity.

Because we left it very late we wanted to discuss the contract at the end of the season between the Bahrain races and then obviously Lewis didnt feel well, said Mercedes boss Toto Wolff on Monday.

At the end we started our conversations just before Christmas and it was important to get it done as soon as possible and in that respect we thought lets postpone the discussion about 2022 and onwards to a later stage in 2021.

As long as he enjoys racing, I think hes very capable of going longer. He develops as a driver, he looks after himself in terms of physical training and mental preparation side, so I dont think in terms of ability that ends in 2021, but at the end its [his career] his decision.

Hamilton has more to his life than merely Formula 1, most notably his interests in fashion, music, and more recently the push for diversity and equality the campaign for which is more effective the longer he is present. Inevitably, at some point, there will be a generational handover of the baton whether through choice or by circumstance. But his contemporaries have underlined that the older guys can still cut it.

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Hamilton, sports GOATs, and the era of longevity - Motorsport Week

When it comes to health, we are all constantly on the lookout for ways to boost our health and overall wellness. Often, we look to food as a means by which to achieve these goals. That seems to make logical sense. Were all constantly told you are what you eat. If that is indeed the case then it is vital that we take a long, hard look at what were putting into our bodies. One food which seems to have an outstanding track record when it comes to health benefits is garlic. Garlic has long been held in high esteem. In fact, the ancient Greek physician Hippocrates apparently used to prescribe garlic for all manner of health conditions. These benefits are not just based on ancient history. Modern science has since confirmed that garlic does in fact benefit the body.

Many of us enjoy the addition of garlic in meals like pizza, pasta and stir fry. But the benefits of adding garlic to your food might be more far-reaching than simply making your food taste good.

Garlic, relative to its small size and calorie content is incredibly nutritious. According to Healthline, just one clove of garlic contains:

And this same amount contains just 4.5 calories 0.2 grams of protein and 1 gram of carbs. It seems that garlic contains a little of almost every single nutrient which the body requires. This means that garlic is nutrient-dense without being calorie-dense. Making it one of the few options for adding flavour without excess calories.

Garlic, mostly in the form of supplements have long been used to boost the immune system. This is something we could all benefit from in 2021 whilst

Heart attacks and strokes are currently some of the worlds leading killers. These diseases are largely causedby high blood pressure (hypertension). Garlic, again in supplement form, seems to have a significant impact on reducing blood pressure. One study found that 6001,500 mg of aged garlic extract was as effective for reducing blood pressure as the drug Atenolol over a 24 week period. The dosage to achieve this effect is quite high and equates to about 4 cloves of garlic a day.

There are two types of cholesterol, one is good and one is bad. LDL is the bad one and HDL is the good type of cholesterol. In sufferers of high cholesterol, taking garlic supplements reduced the total and/or LDL cholesterol by up to 15%. Garlic appears to have a direct impact on LDL cholesterol and works to lower it but has no effect on the good HDL cholesterol.

Garlic is full of antioxidants that aid in supporting the body to protect against oxidative damage. Oxidative damage comes from free radicals and contributes to the ageing process. Garlic supplements have been shown to increase antioxidant enzymes. They also reduce significantly oxidative stress in people with high blood pressure. The combination of reducing cholesterol and blood pressure alongside the antioxidants is hugely important. Studies have shown that it may be beneficial in reducing the risk of brain diseases such as Alzheimers and dementia. It is worth mentioning however that again, these benefits only come from a high dosage of garlic supplements.

The addition of garlic or garlic supplements into the diet seems to have an overall beneficial impact on humans. However, its almost impossible to prove that garlic helps you live longer. What it does do is decrease risk factors such as high blood pressure and high LDL cholesterol. The fact that it aids the immune system also seems to indicate that overall, garlic can have a beneficial impact on the body which may help you live a longer, healthier life.

https://www.healthline.com/nutrition/11-proven-health-benefits-of-garlic#TOC_TITLE_HDR_4

https://www.medicalnewstoday.com/articles/265853

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Could garlic help to increase your longevity? - Longevity LIVE

Healthy lifestyle: Physical activities abound on college campuses like William & Mary. These healthy habits are among the benefits of college education that contribute to health and longevity, according to a study led by W&M Assistant Professor of Economics Peter Savelyev. Photo by Jim Agnew

by Nathan Warters | February 4, 2021

A study led by William & Mary Assistant Professor of Economics Peter Savelyev and funded by the National Science Foundation argues that college education leads to healthier and longer lives.

The study, Understanding the Mechanisms Linking College Education with Longevity, was published in September in Journal of Human Capital.

Researchers around the world still debate whether education affects health. Savelyev and his team support the claim that education improves heath by demonstrating the mechanisms behind this effect while using state-of-the art statistical analysis.

In our paper, we show that education increases health and longevity though healthier lifestyles, superior earnings and better work conditions, he said.

Savelyev and his co-authors examined data obtained from men and women who graduated from high school in Wisconsin in 1957. This specific timeframe was important to allow for data collection on at least a partially deceased U.S. cohort.

William & Mary News recently spoke to Savelyev about this study. The interview has been edited for length and clarity.

Variables related to healthy lifestyles that proved to be important are participating in physical exercise, maintaining a healthy body weight and abstaining from smoking tobacco. We also study the role of extremely dangerous work conditions, such as cutting trees or being exposed to infectious diseases. Educated people are less likely to face such jobs. The work conditions mechanism does not necessarily imply diminishing job-related risks for the general population, but it is a personal benefit for those who study hard and receive advanced degrees.

It is well known that many essential life determinants emerge early in life. Later in life, things are harder to change. College education is a powerful contributor to human development that increases health and longevity, among many other good effects, and it should be supported in situations when free market solutions do not work well enough.

Our simulations demonstrate that major health differences created by college education are hard to close later in life, even by strong hypothetical policy interventions, such as greatly increasing taxation of cigarettes.

Partly, this is because the effect of education is strong, and it works through many different mechanisms. It is not easy for an intervention that targets just one specific mechanism to overcome a strong effect created through dozens of mechanisms. Also, some policies, such as taxation of cigarettes, affect behavior of both the college-educated and those who did not go to college, so the effect on the difference between these two groups is small.

We find that obtaining a bachelors degree leads to about three additional years of life for men. However, we could not find any effect of college education for women, who live longer than men regardless of their education status. We identified from our data two mechanisms that partly explain the gender difference. One is that men tend to take more dangerous jobs than women. Another is that educated women of this historic cohort born in late 1930s in Wisconsin were less likely to be married, which created a negative contribution to their longevity that masked a positive contribution related to higher income. Marriage is good for your longevity. Since a negative effect of college education on marriage no longer takes place for more recent cohorts of women, we can expect a stronger effect of college on their longevity, as confirmed by our simulations.

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Study examines why college education leads to healthier and longer lives - William & Mary News

A total of 1trn of defined benefit (DB) pension longevity risk will be insured through de-risking transactions by the end of 2031, Hymans Robertson has predicted.

According it the firms 2021 Risk Transfer Report, buy-ins, buyouts and longevity swaps have insured 0.3trn of risk from DB pension schemes since 2007. This is expected to increase by 0.7trn between now and the end of 2031.

Around 180bn of the 0.3trn came through buy-ins and buyouts, while approximately 110bn was insured through longevity swaps.

Hymans Robertson estimated that around 450bn of the 0.7trn of growth up to 2031 will be driven by buy-ins and buyouts at an average of over 40bn per year.

The remainder of the increase is expected to come through longevity swaps.

Commenting on the findings, Hymans Robertson head of risk transfer, James Mullins, said: 1trn of insurance would be equivalent to around half of the value of all gilts currently issued by the UK government or around half the value of all of the companies in the FTSE 100.

Indeed, with the level of growth in pension scheme buy-ins and buyouts that we are projecting, we can expect to see several insurance companies become some of the largest companies in the FTSE 100 over the next 10 years.

Our analysis projects when each individual DB pension scheme in the UK is expected to be able to afford to insure its pension promises.These projections show that we expect demand from pension schemes for buy-ins and buyouts to average at over 40bn a year during the next decade.

This is due to a combination of factors such as funding requirements meaning that sponsoring employers will need to fund pension schemes to a higher level and the cost of insuring deferred member liabilities having reduced materially in recent years. These points mean that the additional money a pension scheme needs to get to buyout is less than it has been in the past.

This gap will reduce further as pension schemes mature, as more contributions are paid in and as investment risk is reduced further.

Mullins added that the longevity risk associated with around 17 per cent of all DB liabilities in the UK has now been insured, up from around 1 per cent 10 years ago.

During 2020, approximately 54bn of DB pension risk was transferred to insurers, with around 30bn coming form buy-ins and buyouts, and approximately 24bn from longevity swaps, making 2020 the second-highest total on record, after 2019.

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DB pension de-risking expected to total 1trn by 2031 - Pensions Age

There are three hormones in your body specifically important for healthy aging and longevity. Although for many it seems we shouldnt focus on healthy aging when we are in our 20s and 30s but before you know it, the daily decisions that you are making matter and have a long-term impact on your health.

Many age-related conditions appear out of nowhere. They silently grow inside you before they are apparent and hard to ignore. Thats why regular check-ups and looking underneath the hood once in a while is important.

Well, all hormones matter for healthy aging but here we are going to focus on the three most important hormones for longevity and a healthy lifestyle.

Three Hormones to Focus on for Longevity and Healthy Aging: Which Hormones Matter for Healthy Aging?

Many experts would agree that cortisol is one of the most important ones. Its crucial for your health to keep the level of cortisol low. When your cortisol level is constantly high, it can have many causes namely inflammation, chronic stress, poor sleep, weight gain, and many other reasons. A high level of cortisol is an indicator that you are in a vicious cycle of issues and problems that are accumulating daily.

Cortisol can affect our muscle mass. The important part of healthy aging is keeping your muscles and bones strong. When the level of cortisol is high, it can have an impact on your muscles and bones as well as your overall health.

There are many ways of lowering the level of cortisol. You need to get enough sleep, exercise, learn to relax through methods that suit you from meditation to walking, and learn how to deal with stress. The most important thing is to have more fun in life, less stress. You can also lower the level of cortisol by taking certain supplements such as fish oil but overall healthy lifestyle habits will take you to longevity, healthy aging, and low cortisol levels.

This hormone is crucial for building muscles. When the level of testosterone is low it can be linked to age-related diseases. Older men with a low level of testosterone can have a high risk of death from heart disease. Its important to stress there is a link between testosterone and age since every man loses anywhere between 1% and 2% of testosterone during his life span. If your testosterone level stays higher with age you are in theory younger compared to your peers.

Most of the research on testosterone has been done on men since women naturally have a lower level of testosterone. However, testosterone is important for both genders especially in link with cortisol.

We need to find a way to keep a level of testosterone high as much as possible while keeping the level of cortisol low. In that case, you have better chances to build your muscles and stay healthy.

It seems like a logical question to ask, especially if you are a woman. How to have a higher level of testosterone without growing a mustache? Well, you can start with exercise and lift weights, eat more proteins, carbs, and fats. If you minimize stress and have a low level of cortisol, you will have a higher level of testosterone.

Vitamin D is essential for almost any function from regulating the production of thyroid hormones to helping the immune system and managing inflammation. When we talk about the immune system, some research has been done indicating that vitamin D can help manage the risks of COVID 19. However, we need more research to find a clear connection.

Vitamin D is so easy to take. Sometimes all you need is one supplement a day. Other things that can help include spending more time in the sun or consuming more seafood and fatty fish such as tuna, oysters, sardines, and mackerel. Your diet matters for vitamin D, so eat more eggs, mushrooms, and seafood. The majority of vitamin D supplements come from animal sources so if you are vegan opt for D2 supplements to get enough vitamin D.

There is much that can be done to keep healthy and to optimize your health over the years and keeping track of these hormones is a crucial part of it.

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Three Hormones to Focus on for Longevity and Healthy Aging - Gildshire Magazines

LAUPHEIM, Germany and STEVENAGE, United Kingdom, Feb. 11, 2021 (GLOBE NEWSWIRE) -- Rentschler Biopharma SE, a leading global contract development and manufacturing organization (CDMO) for biopharmaceuticals, today announced that the Company is establishing a Center of Excellence for cell and gene therapy, which will be located in Stevenage, UK. With the newly formed UK subsidiary, Rentschler ATMP Ltd., the CDMO will establish development and manufacturing capability in Advanced Therapy Medicinal Products (ATMPs). The Companys UK site is being set up at an already existing facility run by the Cell and Gene Therapy Catapult (CGT Catapult) through a unique collaborative model. Over the next five years, Rentschler Biopharma plans to make a significant investment at the site to set up state-of-the-art manufacturing of viral vectors for clinical supply and expects to be ready for cGMP manufacturing by the first half of 2022.

Dr. Frank Mathias, CEO of Rentschler Biopharma, said: With more than 1000 cell and gene therapies currently in clinical development, the demand for manufacturing capabilities and support is ever increasing. Leveraging our specific expertise in process development and manufacturing for challenging molecules with highest quality and within demanding timelines, we are ideally suited to implement this knowledge in the field of viral vector production for new modalities. Our well-planned move into this growing area of biopharmaceuticals fits well with our strategic plans to expand internationally ensuring proximity to our clients and important biopharma hubs.

Rentschler Biopharma will continue its well-established practice of forming true partnerships with its clients. At this new Center of Excellence, the company plans to partner with entrepreneurial players to enable them to transform their ideas into real products with the potential to treat and even cure patients with serious and life-threatening diseases. Rentschler Biopharma utilizes a highly flexible business model that addresses the specific needs of each client.

About Rentschler Biopharma SE

Rentschler Biopharma is a leading contract development and manufacturing organization (CDMO), focused exclusively on client projects. The company offers process development and manufacturing of biopharmaceuticals as well as related consulting activities, including project management and regulatory support. Rentschler Biopharma's high quality is proven by its long-standing experience and excellence as a solution partner for its clients. A high-level quality management system, a well-established operational excellence philosophy and advanced technologies ensure product quality and productivity at each development and manufacturing step. In order to offer best-in-class formulation development along the biopharmaceutical value chain, the company has entered into a strategic alliance with Leukocare AG. Rentschler Biopharma is a family-owned company with about 1,000 employees, headquartered in Laupheim, Germany with a second site in Milford, MA, USA. In Stevenage, UK, Rentschler Biopharma launched a company dedicated to cell and gene therapies, Rentschler ATMP Ltd.

For further information, please visit http://www.rentschler-biopharma.com. Follow Rentschler Biopharma on LinkedIn and Facebook.

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Rentschler Biopharma establishes Center of Excellence for cell and gene therapy in the UK - GlobeNewswire

Feb. 10, 2021 08:00 UTC

AMSTERDAM--(BUSINESS WIRE)-- VectorY, a fully integrated gene therapy company focused on the development of innovative vectorized antibodies for muscular and neurodegenerative disorders, today announces its official launch. The Company has been operational since October 2020 and has established its laboratories and offices at the Amsterdam Science Park in The Netherlands.

VectorY develops proprietary and partnered programs based on a novel AAV platform and innovative antibody-based targeted degradation technologies. The Company is creating a pipeline of innovative vectorized antibodies targeting muscular and CNS diseases, aimed at overcoming limitations of current therapies, specifically by improving delivery, durability and accessibility of targeted tissues and cells. These novel viral vectors will have enhanced properties of non-immunogenicity, cell type specificity and expression in selected sub-cellular compartments, thus unlocking new treatment opportunities in these disease areas.

This year, the Company is setting up its own state-of-the-art GMP manufacturing facilities in Amsterdam. Its technology platform will be developed for scalability and yield, with the aim of significantly lowering the costs of advanced gene therapies.

VectorY has already negotiated its first corporate collaboration agreement, gaining access to a highly relevant neuro-inflammation target to develop a vectorized antibody therapy for treatment of neurological disorders. VectorY will continue to consider further collaborations in parallel to progressing its proprietary programs.

The highly experienced management team will be led by Marco Boorsma, General Partner at Forbion, who is joining the management team as Interim CEO. Co-founders Carlo Incerti, Pavlina Konstantinova, Anthony Newcombe, and Barbara Sanders all bring significant longstanding experience in gene therapy, vector development, and biopharmaceutical manufacturing expertise. Carlo Incerti, Operating Partner at Forbion and previously Chief Medical Officer of Sanofi Genzyme, will join VectorYs board as Chairman, together with Sander Slootweg, Managing Partner at Forbion, as board member.

VectorY was seeded by Forbion, following previous investments in successful gene vector companies including bluebird bio (then named Genetix), Biovex, Hookipa, EnGene, Amsterdam Molecular Therapeutics, uniQure and Replimune.

Carlo Incerti, Co-Founder and Chairman of the Board of VectorY, said: I have been working in the gene therapy field for over 30 years, and, from experience, know that accurately targeting the right cells with the transgene has always been of paramount importance. Using viral vectors developed at VectorY to deliver the genetic sequence for therapeutic antibody expression in target cells has the potential to address some of the key shortcomings of intravenously injected molecules. Our aim is to create novel treatments for patients.

Marco Boorsma, Interim CEO of VectorY, said: VectorY is developing innovative technologies to create novel, potentiated vectorized antibodies that will target muscle and CNS disorders, for which substantial unmet medical needs exist. Our aim is to bring new, powerful treatments to patients suffering from serious diseases. The Company is hitting the ground running, capitalizing on the extensive experience of the co-founders, and the support and expertise from Forbion as a longstanding gene therapy investor.

-Ends-

Notes to Editors

About VectorY VectorY combines the therapeutic potential of antibodies and gene therapy to develop long-lasting therapeutic solutions for muscular and neurodegenerative diseases with high unmet medical need.

Founded in August 2020, and based in the Amsterdam Science Park, VectorY is a fully integrated gene therapy company focused on the development of innovative therapeutics based on a novel AAV gene therapy platform, antibody-based targeted degradation technologies, and proprietary manufacturing technology.

VectorY develops proprietary & partnered programs and product candidates are based on new technologies that will enable the next generation of highly scalable manufacturing processes within VectorYs own manufacturing facilities. VectorYs manufacturing capabilities will include a state-of-the-art multi-product GMP facility in the Netherlands, with the capability to deliver suspension based AAV viral vector manufacturing of up to 2000L for both clinical and commercial supply.

About Forbion Forbion is a dedicated life sciences venture capital firm with offices in The Netherlands, Germany and Singapore. Forbion invests in life sciences companies that are active in the (bio-) pharmaceutical space.

Forbion manages well over EUR 1.7 billion across multiple fund strategies that cover all stages of (bio)pharmaceutical drug development. Forbions current team consists of 20 life sciences investment professionals that have built an impressive performance track record since the late nineties with successful investments in over 69 companies.

The firm is a signatory to the United Nations Principles for Responsible Investment. Besides financial objectives, Forbion selects investments that will positively affect the health and well-being of patients.

Its investors include the EIF, through its European Recovery Programme (ERP), LfA, Dutch Venture Initiative (DVI), AMUF and EFSI facilities and KfW Capital through the Programme, ERP Venture Capital Fonds investments. Forbion operates a joint venture with BGV, the manager of seed and early-stage funds, especially focused on Benelux and Germany.

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

View source version on businesswire.com: https://www.businesswire.com/news/home/20210210005065/en/

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Launch of Novel Gene Therapy Company, VectorY, to Develop Innovative Vectorized Antibodies - BioSpace

New York, Feb. 10, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Europe Cell and Gene Therapy Market - Industry Outlook and Forecast 2021-2026" - https://www.reportlinker.com/p06021776/?utm_source=GNW

The global cell and gene therapy market is observing significant mergers and acquisition activities, product sales, and new market authorizations. In 2026, the market is expected to grow almost four times more than the current value, with new product approvals expected annually. Although initial product approvals have been for relatively small patient groups, the significant pipeline of cell & gene therapy studies for diseases such as hemophilia and various forms of blindness will significantly expand. In addition, the Europe market is witnessing steady growth due to the increased availability of funds from several public and private institutes. There is increased support from regulatory bodies for product approvals and fast-track product designations, which encourage vendors to manufacture products at a fast rate. Moreover, with over 237 regenerative medicines companies headquartered in Europe, the region is seen as the favorite destination for cell and gene therapy manufacturing.

The following factors are likely to contribute to the growth of the Europe cell and gene therapy market during the forecast period: CMOs Offering Vector Manufacturing Services for Cell and Gene Therapy Companies Robust Cell & Gene Therapies in the Pipeline Increase in Strategic Acquisitions Regulatory Support for Cell and Gene Therapy Products

The study considers the present scenario of the Europe cell and gene therapy market and its market dynamics for the period 2020?2026. It covers a detailed overview of several market growth enablers, restraints, and trends. The report offers both the demand and supply aspects of the market. It profiles and examines leading companies and other prominent ones operating in the market.

Europe Cell and Gene Therapy Market Segmentation The Europe cell and gene therapy market research report includes a detailed segmentation by product, end-user, application, geography. A high potential to treat several chronic diseases, which cannot be effectively treated/cured through conventional methods otherwise, is propelling the growth of gene therapies. Gene therapies are regarded as a potential revolution in the health sciences and pharmaceutical fields. The number of clinical trials investigating gene therapies is increasing in Europe, despite the limited number of products that have successfully reached the market. However, gene therapies show slow progress and promising prospect in terms of treatments. High support from regulatory bodies to commercialize these products and make them affordable to patients is another important factor contributing the market growth.

Delivering cell and gene therapies requires specialized facilities, capabilities, and clinician skills. Therefore, manufacturers are working in tandem with chosen treatment centers (hospitals) to establish the protocols and procedures necessary to receive the product and therapies. While cell therapies represent a paradigm shift in the treatment of several incurable, chronic diseases, with durable responses and long-term disease control measures, hospitals appear an ideal location to carry out these procedures. Hospitals are growing at a significant rate due to the increasing target population in Europe. Tier-I hospitals are proving to be sought-after network partners for cell and gene therapy developers. They tend to be in major population centers, have adequate financial and personnel resources, and value the prestige that comes with being the first movers in an innovative treatment area.

Oncology accounted for a share of over 30% in 2020. While cancer treatments have evolved and undergone massive developments in recent years, it continues to be one of the deadliest diseases confronted by humans. Traditional cancer therapies have a curative effect in the short term; however, they have side effects, thereby decreasing the patients quality of life. Cell and gene therapies for certain types of cancers have been promising results. The chimeric antigen receptor- (CAR-) T cell therapy is one of the most recent innovative immunotherapies and is rapidly evolving. CAR-T cell therapies are developing rapidly, and many clinical trials have been established on a global scale, which has high commercial potential for the treatment of cancer. Immunotherapies based on CAR-T cells go one step further, engineering the T cells themselves to enhance the natural immune response against a specific tumor antigen. CAR-T clinical trials have shown high remission rates, up to 94%, in severe forms of blood cancer, thereby increasing the market growth.

Product Cell Therapies Gene Therapies End-user Hospitals Cancer Care Centers Wound Care Centers Others Application Oncology Dermatology Musculoskeletal Others

INSIGHTS BY GEOGRAPHY Germany, France, the UK, Italy, and Spain play a significant role in the Europe cell and gene therapy market. Clinical trials and the number of manufacturing facilities are increasing slowly in the European region. The region has become a major R&D destination for several vendors as the funding for cell & gene therapies is increasing. Europe has supported collaborative efforts in gene transfer and gene therapy research. In addition, the target patient population is increasing across Europe; there were an estimated 3.9 million new cases of cancer and 1.9 million cancer deaths in Europe in 2018. In addition, the prevalence surveys in the UK and Denmark indicate that there are 34 people with one or more wounds per 1,000 people. Favorable government support in terms of product approvals, reimbursement and coverage, and high R&D funding to academic institutes that are involved in the development of cell and gene therapies are expected to boosting the market in Europe.

Geography Europe o UK o Germany o France o Italy o Spain o Switzerland o Netherlands

INSIGHTS BY VENDORS Novartis, Spark Therapeutics, Amgen, Gilead Sciences, and Organogenesis are the leading players in the Europe cell and gene therapy market. The market offers tremendous growth opportunities for existing and future/emerging players on account of the presence of a large pool of target patient population with chronic diseases such as cancer, wound disorders, diabetic foot ulcer, CVDs, and other genetic disorders. Recent approvals have prompted an unprecedented expansion among vendors. While a few vendors are opting for in-house production of cell and gene therapies, a substantial number of vendors are preferring third-party service providers, including CMOs.

Prominent Vendors Novartis Spark Therapeutics Amgen Gilead Sciences Organogenesis

Other Prominent Vendors APAC Biotech AVITA Medical bluebird bio CHIESI Farmaceutici CollPlant CO.DON Human Stem Cells Institute PJSC (HSCI) Medipost NuVasive Nipro Orchard Therapeutics RMS Regenerative Medical System Orthocell Osiris Therapeutics Sibino GeneTech Shanghai Sunway Biotech Takeda Pharmaceutical Company Terumo Vericel

Emerging Investigational Vendors In Europe Adaptimmune Therapeutics AgenTus Therapeutics Autolus Cellecits Celyad CombiGene EUKARS Freeline Therapeutics Innoskel PsiOxus Therapeutics Ltd SparingVision uniQure

KEY QUESTIONS ANSWERED 1. What is the Europe cell and gene therapy market size and growth rate during the forecast period? 2. What are the factors driving the demand for CAR-T therapy in the European region? 3. How are strategic acquisitions aiding in market growth of cell and gene therapy products? 4. Which segments are expected to generate the highest revenues during the forecast period? 5. Who are the leading vendors in the European cell and gene therapy market?Read the full report: https://www.reportlinker.com/p06021776/?utm_source=GNW

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The Europe cell and gene therapy market by revenue is expected to grow at a CAGR of over 23% during the period 20212026 - GlobeNewswire

ALISO VIEJO, Calif.--(BUSINESS WIRE)--As gene therapies and editing technologies rapidly advance, it is more urgent than ever to provide updates and information to the rare disease community on how these technologies can be applied across multiple diseases. Global Genes, a leading rare disease patient advocacy organization, is pleased to announce they will be publishing a multimedia series, titled Platforms of Hope: Advances in Gene Therapy and Gene Editing, throughout 2021 regarding upcoming data announcements and information on gene therapy and editing technology advances with thought leaders from the National Center for Advancing Translational Sciences (NCATS), the NIH Common Funds Somatic Cell Genome Editing (SCGE) program and other leading voices in these fields.

In addition to ongoing coverage through Global Genes videos, online publication RARE Daily and RARECast podcast, the organization will be publishing a special report at the end of 2021 on gene therapy and gene editing innovation. This will address a wide range of topics, with a focus on efforts to accelerate the translation of discoveries into genetic medicines that benefit patients with rare diseases.

With more than 7,000 rare diseases, there is an urgent need to keep the rare disease community abreast of developments in the rapidly changing fields of gene therapy and genome editing, said P.J. Brooks, program director at the Office of Rare Diseases Research at the NCATS. In this collaboration, NIH will help Global Genes identify the ideas, technologies and advances that have broad implications for many patients and families affected by rare diseases and provide information that could positively impact their lives and care in the future.

This collaboration will bring visibility into cutting-edge science at the frontier of genetic medicines and provide the rare disease community with insights into emerging technologies and therapies in development for rare diseases, said Christian Rubio, vice president, strategic advancement at Global Genes. Its critically important to educate the rare disease community on these rapidly evolving events.

For more information, visit http://www.globalgenes.org/media-hub.

About Global Genes

Global Genes is a 501(c)(3) nonprofit organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. In pursuit of our mission, we connect, empower, and inspire the rare disease community to stand up, stand out, and become more effective on their own behalf -- helping to spur innovation, meet essential needs, build capacity and knowledge, and drive progress within and across rare diseases. We serve the more than 400 million people around the globe and nearly one in 10 Americans affected by rare diseases. If you or someone you love has a rare disease, or are searching for a diagnosis, contact Global Genes at 949-248-RARE, or visit our resource hub.

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Global Genes Announces New Multimedia Series Focused on Advances in Gene Therapy and Editing, in Collaboration with the National Institutes of Health...

Shares of AVROBIO wereup nearly 20% in premarket trading after the company posted positive clinical data from its gene therapy trials in three different rare lysosomal diseases, Fabry, Gaucher type 1 and cystinosis.

The data from the Phase II study assessing AVR-RD-01, an investigational ex vivo lentiviral gene therapy for Fabry disease, was particularly promising. This morning, Cambridge, Mass.-based Avrobio said a second kidney biopsy conducted on the first patient dosed with AVR-RD-01 showed 100% clearance of the toxic substrate Gb3.

Kidney substrate reduction is the primary endpoint of the Phase II study and has been a cornerstone for evaluating and approving treatments in Fabry disease, Avrobio noted. Avrobio said the patient came in with significant toxic buildup in his kidneys, which is quite common with Fabry disease. One year after the gene therapy treatment was administered in the trial, two independent pathologists foundzero markersof toxic substrate across all the 99 biopsy slides each evaluated.

The first biopsy conducted on the patient showed an 87% clearance of the substrate. In addition to the substrate clearance in the Fabry disease study, AvroBio reported continued strong and durable results in other key metrics across all nine Phase I and Phase II Fabry patients. All patients are now producing the functional enzyme they need to clear toxic substrate from their cells and seeing a concurrent drop in plasma substrate. The farthest patient is out 3.5 years, the company said.

Avrobio Chief Executive Officer Geoff MacKay hailed the data and said it was a thrilling way to begin 2021. The data announced this morning builds on the breadth of strong clinical data weve reported across our leading lysosomal disorder pipeline of single-dose gene therapies, he added.

The Fabry disease study wasnt the only positive news from Avrobio. The company also announced six-month data from the first patient dosed in the Phase I/II study of AVR-RD-02, an investigational ex vivo lentiviral gene therapy for Gaucher disease type 1. That data showed plasma chitotriosidase and the toxic metabolite lyso-Gb1, which are key biomarkers of Gaucher disease, had both dropped 49% below the patients baseline levels that had been achieved on enzyme replacement therapy (ERT) before gene therapy was administered. Also, Lyso-Gb1, the toxic metabolite that builds up in cells throughout the body in Gaucher, is down 44% below the patients ERT baseline. Avrobio said this is an early sign of efficacy.

Based on the data observed to date, we believe lentiviral gene therapy drives down toxic metabolites below levels of ERT, supporting our view that gene therapy has the potential to prevent, halt or even reverse progression of these devastating diseases with a single infusion, MacKay said.

For the cystinosis study, Avrobio said three patients who are taking part in the study are now off of standard-of-care treatment. The first study patient has had sharp reductions in crystal density in the eyes and skin and a marked improvement in photophobia, which is an extreme sensitivity to light that is associated with the disease.

With 13 patients dosed across three clinical programs, we have observed sustained and potentially transformative improvements in key biomarkers and functional metrics, with data from our Fabry disease program out 3 years after dosing. Additionally, enrollment activities for our Fabry disease trial are accelerating, giving us added confidence in our efforts to meet our goal of having dosed a cumulative 30 patients across all our clinical programs by the end of the year. With this strong momentum, we look forward to clarifying the regulatory pathway with regulatory agencies, MacKay said in a statement.

Full data from the studies will be presented later this week and WORLDSymposium, an annual meeting dedicated to lysosomal disorders.

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Avrobio Gene Therapy Shows Early Promise in Fabry, Other Rare Lysosomal Diseases - BioSpace

Amicus Optimized Transgene Show Greater Substrate Reduction than Wild Type Construct Across All Tissues and Doses

Further Validates Combining Amicus-Engineered Transgenes with Penns AAV Gene Therapy Technologies to Develop Next Generation Gene Therapies

PHILADELPHIA, Feb. 08, 2021 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced initial preclinical data from its investigational adeno-associated viral (AAV) gene therapy program for Fabry disease in mice. The results are featured in a virtual poster presentation at the 17th Annual WORLDSymposium 2021, being held February 8-12, 2021. The poster is also available in the Events and Presentations section of the Amicus Therapeutics corporate website.

Fabry disease is an inherited lysosomal disorder caused by deficiency of the enzyme alpha-galactosidase A (GLA). Reduced or absent levels of GLA lead to accumulation of disease substrate leading to cellular disfunction and organ damage, which results in the clinical manifestations of Fabry disease. Amicus, in collaboration with the Gene Therapy Program of the Perelman School of Medicine at the University of Pennsylvania (Penn), is developing a novel gene therapy for Fabry disease that combines the Amicus protein-engineering expertise and deep knowledge and experience in Fabry disease with Penns adeno associated virus (AAV) gene transfer technologies.

This initial preclinical study assessed a range of single doses of AAV in Gla knockout (KO) mice with either natural unmodified hGLA (wildtype hGLA) or Amicus/Penn engineered hGLA transgenes (engineered hGLA). The Amicus/Penn engineered hGLAs are designed for improved stability which is believed to provide a larger window for the enzyme to stay active while in circulation prior to being taken up into the target tissues and for additional stabilization after cell uptake. The lead Amicus/Penn engineered hGLA declared as an IND candidate is designated as AT-GTX-701.

Preclinical Poster Highlights for Amicus/Penn AAV Gene Therapy for Fabry Disease:

Hung Do, Ph.D., Chief Science Officer of Amicus Therapeutics, stated, These very important preclinical results validate our capabilities to develop engineered proteins via a gene therapy that can result in superior substrate reduction compared with a wildtype transgene. This is the second program in our collaboration with Penn that has demonstrated the potential advantages of optimizing the target protein in these disorders, and may be applicable to other lysosomal disorders as we continue to combine our understanding of the molecular basis of these diseases and expertise in protein engineering, together with Penns vector engineering expertise, to develop novel gene therapies.

Amicus is currently developing AAV gene therapies in collaboration with Penn for Pompe disease, Fabry disease, CDD, CLN1, MPS IIIB, a next generation program in MPS IIIA, as well as Angelman Syndrome. The agreement between Amicus and Penn is a Research, Collaboration and License Agreement, providing funding to Penn to advance the preclinical research programs in the Wilson Lab and to license certain technologies invented under the funded Research Collaboration.

About Fabry DiseaseFabry disease is an inherited lysosomal disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A), which is the result of mutations in the GLA gene. The primary biological function of alpha-Gal A is to degrade specific lipids in lysosomes, including globotriaosylceramide (referred to here as GL-3 and also known as Gb3). Lipids that can be degraded by the action of alpha-Gal A are called "substrates" of the enzyme. Reduced or absent levels of alpha-Gal A activity lead to the accumulation of GL-3 in the affected tissues, including the central nervous system, heart, kidneys, and skin. Progressive accumulation of GL-3 is believed to lead to the morbidity and mortality of Fabry disease, including pain, kidney failure, heart disease, and stroke. The symptoms can be severe, differ from patient to patient, and begin at an early age. All Fabry disease is progressive and may lead to irreversible organ damage regardless of the time of symptom onset.

About Amicus Therapeutics Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the companys website at http://www.amicusrx.com, and follow us on Twitter and LinkedIn.

Forward-Looking StatementsThis press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to initial preclinical data from its investigational adeno-associated viral (AAV) gene therapy program for Fabry disease in mice and the potential implications of these data for the future advancement and development of a gene therapy for Fabry disease and other lysosomal disorders and development of potential platform technologies. Words such as, but not limited to, look forward to, believe, expect, anticipate, estimate, intend, "confidence," "encouraged," potential, plan, targets, likely, may, will, would, should and could, and similar expressions or words identify forward-looking statements. The forward looking statements included in this press release are based on management's current expectations and belief's which are subject to a number of risks, uncertainties and factors, including that the preliminary data reported before completion of the study will not be predictive of future results, that results of additional preliminary data or data from the completed study or any future study will not yield results that are consistent with the preliminary data presented, that later study results will not support further development, or even if such later results are favorable, that the Company will not be able to successfully complete the development of, obtain regulatory approval for, or successfully commercialize. In addition, all forward looking statements are subject to the other risks and uncertainties detailed in our Annual Report on Form 10-K for the year ended December 31, 2019 and the Quarterly Report filed on Form 10-Q for the quarter ended September 30, 2020. As a consequence, actual results may differ materially from those set forth in this press release. You are cautioned not to place undue reliance on these forward looking statements, which speak only of the date hereof. All forward looking statements are qualified in their entirety by this cautionary statement and we undertake no obligation to revise this press release to reflect events or circumstances after the date hereof.

CONTACTS:

Investors:Andrew FaughnanSr. Director, Investor Relationsafaughnan@amicusrx.com(609) 662-3809

Media:Diana MooreHead of Global Corporate Communicationsdmoore@amicusrx.com(609) 662-5079

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Amicus Therapeutics Presents Positive Preclinical Fabry Disease Gene Therapy Data at the 17th Annual WORLDSymposium 2021 - GlobeNewswire

The "Adeno-Associated Virus (AAV) Vectors in Gene Therapy - Market Insight, Epidemiology and Market Forecast - 2030" drug pipelines has been added to ResearchAndMarkets.com's offering.

This report delivers an in-depth understanding of the AAV Vector Based Gene Therapy, historical and forecasted epidemiology as well as the AAV Vector Based Gene Therapy market trends in the United States, EU5 (Germany, France, Italy, Spain, and United Kingdom), and Japan.

The AAV Vector Based Gene Therapy market report provides emerging drugs, AAV Vector Based Gene Therapy market share of the individual diseases, current and forecasted AAV Vector Based Gene Therapy market size from 2017 to 2030 segmented by seven major markets. The Report also covers current AAV Vector Based Gene Therapy market drivers, market barriers and unmet medical needs to curate best of the opportunities and assesses underlying potential of the market.

The AAV Vector Based Gene Therapy epidemiology division provides the insights about historical and current AAV Vector Based Gene Therapy patient pool and forecasted trend for each seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of The report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

Key Findings

The disease epidemiology covered in the report provides historical as well as forecasted AAV Gene Therapies epidemiology [segmented as Total cases of AAV Gene Therapy Targeted Indications, Total diagnosed indication-specific cases, Number of Patients Eligible for AAV Gene Therapy and Total treated cases] scenario of AAV Gene Therapy in the 7MM covering United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom), and Japan from 2017 to 2030.

Country Wise- AAV Vector Based Gene Therapy Epidemiology

Estimates show that the highest cases of AAV vector based gene therapy in the 7MM were in the United States, followed by Germany, Japan, France, the United kingdom, Italy, and Spain in 2017.

Story continues

AAV vector based gene therapy Drug Chapters

Drug chapter segment of the AAV vector based gene therapy report encloses the detailed analysis of AAV vector based gene therapy marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the AAV vector based gene therapy clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.

Scope of the Report

The report covers a detailed overview of the development of AAV vectors and gene therapies based on them across the various therapeutic areas and several selected indications, explaining its various serotypes, benefits, and challenges for future

Comprehensive insight has been provided into the AAV Vector Based Gene Therapy epidemiology and treatment in the 7MM.

Additionally, an all-inclusive account of both the current and emerging therapies for AAV Vector Based Gene Therapy is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.

A detailed review of AAV Vector Based Gene Therapy market; historical and forecasted is included in the report, covering drug outreach in the 7MM.

The report provides an edge while developing business strategies, by understanding trends shaping and driving the global AAV Vector Based Gene Therapy market.

AAV Vector Based Gene Therapy Report Insights

Patient Population

Therapeutic Approaches

AAV Vector Based Gene Therapy Pipeline Analysis

AAV Vector Based Gene Therapy Market Size and Trends

Market Opportunities

Impact of upcoming Therapies

AAV Vector Based Gene Therapy Report Key Strengths

AAV Vector Based Gene Therapy Report Assessment

SWOT Analysis

Current Treatment Practices

Unmet Needs

Pipeline Product Profiles

Conjoint Analysis

Market Attractiveness

Market Drivers and Barriers

Reasons to Buy

The report will help in developing business strategies by understanding trends shaping and driving the AAV Vector Based Gene Therapy market.

To understand the future market competition in the AAV Vector Based Gene Therapy market and Insightful review of the key market drivers and barriers.

Organize sales and marketing efforts by identifying the best opportunities for AAV Vector Based Gene Therapy in the US, Europe (Germany, France, Italy, Spain, and the United Kingdom) and Japan.

Identification of strong upcoming players in market will help in devising strategies that will help in getting ahead of competitors.

Organize sales and marketing efforts by identifying the best opportunities for AAV Vector Based Gene Therapy market.

To understand the future market competition in the AAV Vector Based Gene Therapy market.

Companies Mentioned

For more information about this drug pipelines report visit https://www.researchandmarkets.com/r/p3sdwo

View source version on businesswire.com: https://www.businesswire.com/news/home/20210209005920/en/

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Global Adeno-Associated Virus (AAV) Vectors in Gene Therapy Market to 2030 - Insight, Epidemiology and Forecasts - ResearchAndMarkets.com - Yahoo...

TipRanks

Investors have been fixated on growth companies over the past year, and one segment which has been on the rise is the fledgling cannabis industry. The sector offers a unique proposition and the prospect of further growth, as there is still a major catalyst on the horizon which will completely alter the industry. As expected, a Democrat led senate has been good news for those banking on marijuana reform at the federal level; And it looks like the anticipated changes could happen faster than initially expected. Backed by Senate majority leader Chuck Schumer, Democratic Senators have stated that they will push for federal-level legalization of marijuana, promising a unified discussion draft on comprehensive [cannabis] reform in the first half of this year. The statement feeds expectations that the Democratic Congressional majority will pass and that President Biden will sign a bill to legalize marijuana. Investors are also looking at further state-level legalization moves; one key state in this regard is New York. So, the cannabis industry is looking up. There is an expanding network of state legalization regimes, and expectations of a change in federal policy; both are putting upward pressure on cannabis shares. Against this backdrop, we used TipRanks database to find two cannabis stocks that have been earmarked as 'Strong Buys' by the analyst consensus. Both have posted impressive year-to-date performances, and stand to rise even more in the year ahead. Village Farms International (VFF) We will start with Village Farms International, a company that has long been involved in the niche agricultural business. The company started out as a farmer, producing high-quality greenhouse vegetables year-round for sale in the North American market. That background fit the company well for a transition to the cannabis industry Village Farms has experience in greenhouse production and industrial-scale growing. Village Farms shares are showing a tremendous growth profile, up 327% in the past 12 months with a strong spike in recent days. Two important pieces of news precipitated the surge since the end of January. First, the company has fully repaid ahead of schedule the $15 million debt it incurred during its November acquisition of the cannabis growing company Pure Sunfarms. And second, Village Farms increased its investment in the Asian cannabinoid company Altum by 50%, to hold a 10% stake in the company. The move increases the international reach of Village Farms, and its ability to increase Altum holdings in the future. The company was able to fund these moves because it had a successful equity sale in January, putting an additional 10.8 million shares on the market, and raising US$135 million in new capital. In addition to its strong capital and expansion positions, Village Farms has been reporting solid financial results. The company saw US$43 million in revenue for 3Q20, a gain of 12.5% year-over-year. EPS came in at 1 cent per share, a turnaround from the US$0.10 loss in the year-ago quarter. Covering Village Farms for Craig-Hallum, 5-star analyst Eric Des Lauriers writes: Village Farms has clearly established itself as the leading cannabis producer in Canada with #1 brand share and industry-leading profitability. Canadian cannabis sales in 2020 through October (latest available) were up 128% y/y, and dispensary counts are set to accelerate through 2021, providing a tailwind to VFF revenues. Turning to the US markets, and VFFs position in Canadas larger neighbor, the analyst goes on to add, With 5.7M SF of greenhouses in TX, the company also has real US optionality, which is finally being appreciated by investors after the GA election. VFF has historically been undervalued compared to less profitable peers, but we expect shares to continue working higher as the prospect for US reform increases throughout the year. To this end, Des Lauriers rates VFF a Buy, and his $25 price target suggests the stock has room for ~26% upside in the coming year. (To watch Des Lauriers track record, click here) Overall, there are 3 recent reviews on VFF shares, and all are Buys, giving the stock a Strong Buy analyst consensus rating and showing a general agreement on Wall Street about the companys strengths. Shares are priced at $19.90, and the $24.33 average price target implies an upside of ~23% for the year ahead. (See VFF stock analysis on TipRanks) TerrAscend Corporation (TRSSF) The next cannabis stock were looking at, TerrAscend, is another major cannabis producer in both the US, Canada, and Europe. The company is involved in both the medical and recreational sides of the market, and both grows and produces cannabis and markets a range of products through numerous brand names. TerrAscends US operations are located in California, Pennsylvania, New Jersey, and Utah, and the company looks to expand as more states legalize cannabis. In a strong sign of the cannabis industrys strength, TRSSF shares are up a sky-high 624% over the past 12 months. Growth has been fueled by expansion of the cultivation operations in California and Pennsylvania, and by the move into the adult-use recreational market in New Jersey. Last month, TerrAscend closed a non-brokered private placement stock sale, putting more than 18 million common shares on the market. The sale price was C$12.35 (US$9.72), and the offering grossed C$224 million (US$176.3 million). The bulk of the proceeds some 80% of the total was put up by four large US-based institutional investors. The funds raised will be used to continue expansion of the companys cultivation operations (TRSSF has plans to expand growing and manufacturing ops in New Jersey), as well as to pursue merger & acquisition activities. TerrAscends rapid growth and strong future prospects have attracted attention from top-rated analysts, including 5-star analyst Eric Des Lauriers of Craig-Hallum (stated above). "TerrAscend is a leading multi-state operator (MSO) in the US cannabis market with top-tier management, assets, and access to deal flow. We have been bullish on the company since initiating coverage last year and are happy to say the TRSSF team has exceeded our expectations, generating rapid increases in margins and operating leverage that have earned them a place solidly in the Top Tier of MSOs," Des Lauriers noted. The analyst summed up, "[With] US$280M+ raised since the elections and federal reform moving quicker than expected, we think TRSSF does deserve a premium to peers." In line with his bullish comments, Des Lauriers rates TRSSF shares a Buy, and has a $20 price target that implies a ~31% upside potential for the next 12 months. Once again, were looking at a stock with broad agreement from Wall Streets analysts the Strong Buy consensus rating is unanimous, based on 7 recent reviews. Shares are selling for $15.30, and their recent appreciation has pushed that price almost up to the $15.43 average price target. (See TRSSF stock analysis on TipRanks) To find good ideas for cannabis stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights. Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

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Taysha Gene Therapies Announces Participation in Upcoming Investor Healthcare Conferences - Yahoo Finance

Paragon Biosciences, a life science innovator that creates, invests in and builds life science companies in biopharmaceuticals, cell and gene therapy and synthetic biology utilizing artificial intelligence, has launched CiRC Biosciences, a cell therapy company developing treatments for serious diseases with high, unmet needs with an initial focus on the eye."The addition of CiRC Biosciences to our portfolio builds upon our cell and gene therapy platform, an area that has tremendous potential to address serious genetic diseases," said Jeff Aronin, founder, chairman and chief executive officer, Paragon Biosciences. "CiRC Biosciences gives us the science to target retinal diseases that could lead to vision restoration with numerous other applications in the years ahead."CiRC Biosciences is currently advancing pre-clinical development of chemically induced retinal cells for vision restoration in Geographic Atrophy Age-Related Macular Degeneration (Dry AMD), which is the most common cause of irreversible vision loss over the age of 65, and advanced Retinitis Pigmentosa (RP), a genetic disorder that causes tunnel vision and eventual blindness. There are no U.S. Food & Drug Administration (FDA) approved treatments to restore vision loss in Dry AMD or RP.The company's novel mechanism of action is designed for direct chemical conversion of fibroblasts into other cell types using a cocktail of small molecules in an 11-day chemical conversion process. Pre-clinical studies have shown efficacy in blind mice that demonstrated vision restoration. CiRC Biosciences has provisional patent applications to protect its platform."Our technology transforms ordinary skin cells into specialized retinal cells using a cocktail of small molecules," said Sai Chavala, M.D., co-founder and chief scientific officer, CiRC Biosciences. "This process is potentially safer, quicker, more cost effective and easier to manufacturer than using traditional stem cells. Working with Paragon Biosciences to build and advance CiRC Biosciences provides us the opportunity to efficiently progress this technology through research and development stages.CiRC Biosciences first reported its discovery in the highly respected scientific journal Nature (April 15, 2020). A recently published New England Journal of Medicine article (Nov. 5, 2020) discussed CiRC's technology of using chemically induced cells to restore retinal function. The article concluded, "The new and emerging strategies for the rescue, regeneration, and replacement of photoreceptors suggest a bright future in the fight to preserve and restore vision in blinding eye diseases."The abstract in Nature is available here.Access to the NEJM article is available here.

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Paragon Biosciences Expands Cell And Gene Therapy Platform - Contract Pharma

PROVIDENCE, R.I.[Brown University] New research provides insights into the treatment of Christianson syndrome (CS), an X-linked genetic disease characterized by reduced brain growth after birth, intellectual disability, epilepsy and difficulties with balance and speech.

One of the major challenges in developing treatments for human brain disorders, like CS, is developing an experimental system for testing potential therapeutics on human neurons, said study senior author Dr. Eric Morrow, an associate professor of molecular biology, neuroscience and psychiatry at Brown University. In recent years, advanced stem cell therapies that use tissues from patients have provided powerful new approaches for engineering human neurons from the patients themselves, who may undergo the treatment in the future.

For the study, published in Science Translational Medicine on Feb. 10, 2021, Morrow and his colleagues obtained blood samples from five CS patients and the patients unaffected brothers. They then reprogrammed these blood cells into stem cells, and these stem cells were converted into neurons in a petri dish. As a result, they obtained neurons that were representative of those from CS patients, and they used these neurons to test treatments.

Morrow who directs the Center for Translational Neuroscience at the Carney Institute for Brain Science and the Brown Institute for Translational Science said the team also used a new gene-editing approach that employs CRISPR-Cas9 technologies to correct patient mutations back to a healthy gene sequence.

CS is caused by a mutation in a gene encoding for NHE6, a protein that helps regulate acid levels within cell structures called endosomes. Past research suggests that the loss of NHE6 causes endosomes to become overly acidic, which disrupts the abilities of developing neurons to branch out and form connections in the growing brain.

Loss of this important protein can arise from a variety of gene mutations in patients. The majority of CS mutations are called nonsense mutations, which prevent NHE6 from being produced at all; four of the five CS patients involved in this study exhibited this class of mutation. However, some CS patients exhibit missense mutations. Individuals with missense mutations still have some NHE6, but it is produced in smaller amounts, and the protein fails to function as it should.

The research team tested two main forms of treatment on the stem-cell-derived neurons: first, gene transfer, which involves adding a healthy NHE6 gene into the cell; and second, administration of trophic factors, which are substances that promote neuron growth and encourage neurons to develop connections with other neurons. The researchers found that the neurons response to treatment depended on the class of mutation present.

The gene transfer studies, which may represent the first steps toward developing gene therapy, were successful in neurons with nonsense mutations. After the researchers inserted a functional NHE6 gene into nonsense-mutation CS neurons, the neurons branched out properly. In neurons with missense mutations, however, gene transfer failed completely. Further tests suggested that the abnormal NHE6 produced as a result of missense mutations may interfere with normal NHE6, thereby rendering gene transfer therapy ineffective in patient cells with these mutations.

In contrast, administration of trophic factors, such as brain-derived neurotrophic factor (BDNF) and insulin-like growth factor-1 (IGF-1), successfully promoted proper branching in all the CS neurons studied, regardless of mutation type.

While these initial results are encouraging, Morrow hopes that future studies will examine these treatments in animal models.

Our results provide an initial proof-of-concept for these treatment strategies, indicating that they should be studied further, he said. However, we may ultimately need to pay close attention to the class of mutation that a patient has when we choose a specific treatment.

In addition to Morrow, the research team included scientists from Brown University, the University of South Carolina and the Icahn School of Medicine at Mount Sinai. The study was supported by multiple grants from the National Institutes of Health as well as a number of awards from foundations and academic institutions.

This news story was authored by contributing science writerKerry Benson.

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Neurons from patient blood cells enable researchers to test treatments for genetic brain disease - Brown University

DetailsCategory: DNA RNA and CellsPublished on Monday, 08 February 2021 16:09Hits: 412

- Phase 1/2 trial expected to commence in first half of 2021

- Company has three INDs cleared for rare monogenic CNS disorders

PHILADELPHIA, PA, USA I February 08, 2021 I Passage Bio, Inc. (Nasdaq: PASG), a genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) disorders, today announced that the U.S. Food and Drug Administration (FDA) has cleared an investigational new drug (IND) application for PBKR03, an adeno-associated virus (AAV)-delivery gene therapy being studied for the treatment of early infantile Krabbe disease (Globoid Cell Leukodystrophy). Currently, there are no approved disease-modifying therapies available for Krabbe disease, a rare lysosomal storage disease that most often presents early in a childs life, resulting in rapid progressive damage to both the brain and peripheral nervous system and mortality by two years of age. Underscoring the urgent medical need in the patient population, the FDA has previously granted Passage Bio both Orphan Drug and Rare Pediatric Disease designations for PBKR03 for treatment in Krabbe disease.

As part of our commitment to deliver a transformative, one-time gene therapy to the children and their families who suffer from the devastating effects of Krabbe disease, we are excited to advance toward clinically evaluating the potential life-changing benefits of PBKR03, said Bruce Goldsmith, Ph.D., chief executive officer of Passage Bio. The FDA clearance of our IND for PBKR03 is an important milestone for Passage Bio, paving the way for the start of our third clinical program in rare monogenic CNS disorders in the first half of 2021. Having solidified our clinical trial preparedness and manufacturing readiness during the past year, we are well-positioned to move with urgency to advance PBKR03 into the clinic.

PBKR03 utilizes a next-generation proprietary AAV capsid to deliver, through intra-cisterna magna (ICM) administration, a functional GALC gene to Krabbe patients with mutations in the gene that codes for galactosylceramidase (GAL-C). Low GAL-C activity results in accumulation of psychosine which is toxic to the myelin-producing oligodendrocytes of the CNS and Schwann cells in the periphery, resulting in damage to both the central and peripheral nervous systems. PBKR03 has the potential to treat both the central nervous system and peripheral nerve manifestations observed in Krabbe disease patients.

Compelling preclinical data support advancement into clinical trials

PBKR03 is supported by extensive preclinical studies, conducted by our collaborator, the University of Pennsylvanias Gene Therapy Program, showing meaningful transduction of both the central and peripheral nervous system in animal models, with restoration of myelination in the brain and peripheral nerves. In a naturally occurring Krabbe animal model, a single ICM injection of an AAVhu68 capsid containing the normal canine GALC gene showed normalization of GALC activity, reduction of cerebral spinal fluid psychosine levels, normalization of peripheral nerve conduction velocity, improvement in brain myelination, reduction in brain inflammation and increased survival.

Phase 1/2 study anticipated for 1H21

Passage Bio expects to initiate a Phase1/2 clinical trial for PBKR03 in the first half of 2021. The trial is designed as a dose escalation study of a single ICM dose of PBKR03 in pediatric subjects with early infantile Krabbe disease. The primary endpoint of the Phase 1/2 study is safety and tolerability; secondary endpoints include CSF and serum GALC levels, disease biomarkers, and clinical outcome measures. Initial data from the trial is anticipated to potentially readout in late 2021 or early 2022, depending on the timing of when the first patient is treated in the study.

PENN Financial Disclosure

The University of Pennsylvania (Penn) and its Gene Therapy Program receives sponsored research funding from Passage Bio, and Penn has licensed intellectual property to Passage Bio that may result in future financial returns to Penn.

About Krabbe Disease

Krabbe disease is a rare and often life-threatening lysosomal storage disease caused by mutations in the GALC gene, which encodes galactosylceramidase, an enzyme that breaks down galactosylceramide and psychosine. Without adequate levels of galactosylceramidase, psychosine accumulates, causing widespread death of myelin-producing cells and progressive damage to nerves in both the brain and peripheral tissues. The early infantile form of the disease is the most severe and common, typically manifesting before six months of age and accounting for 60 percent to 70 percent of diagnoses. In these patients, the disease course is highly predictable and rapidly progresses to include loss of acquired milestones, staring episodes, apnea, peripheral neuropathy, severe weakness, unresponsiveness to stimuli, seizures, blindness, deafness and eventual death by two years of age. Late infantile patients, defined by onset between seven to twelve months of age, present similar symptoms and have a median survival of approximately five years from onset of symptoms. The estimated worldwide incidence of Krabbe disease is 2.6 in 100,000 births, which is higher than reported due to lack of adequate screening at birth.

About Passage Bio

At Passage Bio (Nasdaq: PASG), we are on a mission to provide life-transforming gene therapies for patients with rare, monogenic CNS diseases that replace their suffering with boundless possibility, all while building lasting relationships with the communities we serve. Based in Philadelphia, PA, our company has established a strategic collaboration and licensing agreement with the renowned University of Pennsylvanias Gene Therapy Program to conduct our discovery and IND-enabling preclinical work. This provides our team with enhanced access to a broad portfolio of gene therapy candidates and future gene therapy innovations that we then pair with our deep clinical, regulatory, manufacturing and commercial expertise to rapidly advance our robust pipeline of optimized gene therapies into clinical testing. As we work with speed and tenacity, we are always mindful of patients who may be able to benefit from our therapies. More information is available at http://www.passagebio.com.

SOURCE: Passage Bio

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FDA Clears IND Application for Passage Bio's Gene Therapy Candidate PBKR03 for Treatment of Patients with Early Infantile Krabbe Disease, A Rare...

New York, Feb. 05, 2021 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Gene Therapy Market by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of Administration, and Key Geographical Regions: Industry Trends and Global Forecasts, 2020-2030" - https://www.reportlinker.com/p06020737/?utm_source=GNW Considering the current pace of research and product development activity in this field, experts believe that the number of clinical research initiatives involving gene therapies are likely to grow by 17% annually. In this context, the USFDA released a notification, mentioning that it now expects to receive twice as many gene therapy applications each year, starting 2020. Despite the ongoing pandemic, it is worth highlighting that gene therapy companies raised approximately USD 5.5 billion in capital investments, in 2020 alone. This is indicative of the promising therapeutic potential of this emerging class of pharmacological interventions, which has led investors to bet heavily on the success of different gene therapy candidates in the long term.

Several technology platforms are currently available for discovery and development of various types of gene therapies. In fact, advances in bioanalytical methods (such as genome sequencing), and genome editing and manipulation technologies (such as molecular switches), have enabled the development of novel therapy development tools / platforms. In fact, technology licensing is a lucrative source of income for stakeholders in this industry, particularly for those with proprietary gene editing platforms. Given the growing demand for interventions that focus on the amelioration of the underlying (genetic) causes of diseases, it is expected that the gene therapy pipeline will continue to steadily expand. Moreover, promising results from ongoing clinical research initiatives are likely to bring in more investments to support therapy product development initiatives in this domain. Therefore, we are led to believe that the global gene therapy market is poised to witness significant growth in the foreseen future.

SCOPE OF THE REPORT The Gene Therapy Market (4th Edition) by Therapeutic Approach (Gene Augmentation, Oncolytic Viral Therapy, Immunotherapy and Others), Type of Gene Therapy (Ex vivo and In vivo), Type of Vectors used (Adeno Associated Virus, Adenovirus, Herpes Simplex Virus, Lentivirus, Plasmid DNA, Retrovirus and Others), Target Therapeutic Areas (Autoimmune Disorders, Cardiovascular Diseases, Dermatological Disorders, Genetic Disorders, Hematological Disorders, Metabolic Disorders, Muscle-related Diseases, Oncological Disorders, Ophthalmic Diseases and Others), Route of Administration (Intraarticular, Intracerebellar, Intradermal, Intramuscular, Intratumoral, Intravenous, Intravesical, Intravitreal, Subretinal and Others), and Key Geographical Regions (US, EU5 and rest of the world): Industry Trends and Global Forecasts, 2020-2030 report features an extensive study of the current market landscape of gene therapies, primarily focusing on gene augmentation-based therapies, oncolytic viral therapies, immunotherapies and gene editing therapies. The study also features an elaborate discussion on the future potential of this evolving market.

Amongst other elements, the report features: - A detailed review of the overall market landscape of gene therapies and gene editing therapies, including information on phase of development (marketed, clinical, preclinical and discovery) of pipeline candidates, key therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, liver diseases, metabolic disorders, muscle-related diseases, nervous system disorders, oncological disorders, ophthalmic diseases and others), target disease indication(s), type of vector used, type of gene, therapeutic approach (gene augmentation, oncolytic viral therapy and others), type of gene therapy (ex vivo and in vivo), route of administration and special drug designation(s) awarded (if any). - A detailed review of the players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain. - An elaborate discussion on the various types of viral and non-viral vectors, along with information on design, manufacturing requirements, advantages and limitations of currently available gene delivery vectors. - A discussion on the regulatory landscape related to gene therapies across various geographies, namely North America (the US and Canada), Europe and Asia-Pacific (Australia, China, Hong Kong, Japan and South Korea), providing details related to the various challenges associated with obtaining reimbursements for gene therapies. - Detailed profiles of marketed and late stage (phase II/III and above) gene therapies, including development timeline of the therapy, information on the current development status, mechanism of action, affiliated technology, strength of patent portfolio, dosage and manufacturing details, as well as details related to the developer company. - An elaborate discussion on the various commercialization strategies that can be adopted by drug developers across different stages of therapy development, including prior to drug launch, at / during drug launch and post-marketing. - A review of the various emerging technologies and therapy development platforms that are being used to design and manufacture gene therapies, featuring detailed profiles of technologies that were / are being used for the development of four or more products / product candidates. - An in-depth analysis of various patents that have been filed / granted related to gene therapies and gene editing therapies, since 2016. The analysis assesses several relevant parameters associated with the patents, including type of patent (granted patents, patent applications and others), publication year, regional applicability, CPC symbols, emerging focus areas, leading industry players (in terms of the number of patents filed / granted), and patent valuation. - A detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2020, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company product and target therapeutic area. - An analysis of the investments made at various stages of development in companies that are focused in this area, between 2015-2020, including seed financing, venture capital financing, IPOs, secondary offerings, debt financing, grants and other equity offerings. - A detailed geographical clinical trial analysis of completed, ongoing and planned studies of numerous gene therapies, based on various relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population. - An analysis of the various factors that are likely to influence the pricing of gene therapies, featuring different models / approaches that may be adopted by manufacturers to decide the prices of these therapies. - An analysis of the big biopharma players engaged in this domain, featuring a heat map based on parameters, such as number of gene therapies under development, funding information, partnership activity and strength of patent portfolio. - An informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength. - A case study on the prevalent and emerging trends related to vector manufacturing, along with information on companies offering contract services for manufacturing vectors. The study also includes a detailed discussion on the manufacturing processes associated with various types of vectors. - A discussion on the various operating models adopted by gene therapy developers for supply chain management, highlighting the stakeholders involved, factors affecting the supply of therapeutic products and challenges encountered by developers across the different stages of the gene therapy supply chain.

One of the key objectives of the report was to estimate the existing market size and the future opportunity associated with gene therapies, for the next decade. Based on multiple parameters, such as target patient population, likely adoption rates and expected pricing, we have provided informed estimates on the evolution of the market for the period 2020-2030. The report also features the likely distribution of the current and forecasted opportunity across [A] therapeutic approach (gene augmentation, oncolytic viral therapy, immunotherapy and others), [B] type of gene therapy (ex vivo and in vivo), [C] type of vectors used (adeno associated virus, adenovirus, herpes simplex virus, lentivirus, plasmid DNA, retrovirus and others), [D] target therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, metabolic disorders, muscle-related diseases, oncological disorders, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebellar, intradermal, intramuscular, intratumoral, intravenous, intravesical, intravitreal, subretinal and others), and [F] key geographical regions (US, EU5 and rest of the world). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industrys growth.

The opinions and insights presented in this study were influenced by discussions conducted with multiple stakeholders in this domain. The report features detailed transcripts of interviews held with the following individuals: - Adam Rogers (CEO, Hemera Biosciences) - Al Hawkins (CEO, Milo Biotechnology) - Buel Dan Rodgers (Founder & CEO, AAVogen) - Christopher Reinhard (CEO and Chairman, Gene Therapeutics (previously known as Cardium Therapeutics)) - Michael Triplett (CEO, Myonexus Therapeutics) - Robert Jan Lamers (CEO, Arthrogen) - Ryo Kubota (CEO, Chairman & President, Acucela) - Tom Wilton (CBO, LogicBio Therapeutics) - Jeffrey Hung (CCO, Vigene Biosciences) - Cedric Szpirer (Executive & Scientific Director, Delphi Genetics) - Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory) - Molly Cameron (Corporate Communications Manager, Orchard Therapeutics)

All actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this report are in USD, unless otherwise specified.

RESEARCH METHODOLOGY The data presented in this report has been gathered via secondary and primary research. For all our projects, we conduct interviews with experts in the area (academia, industry, medical practice and other associations) to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Where possible, the available data has been checked for accuracy from multiple sources of information.

The secondary sources of information include - Annual reports - Investor presentations - SEC filings - Industry databases - News releases from company websites - Government policy documents - Industry analysts views

While the focus has been on forecasting the market over the coming decade, the report also provides our independent view on various emerging trends in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market, gathered from various secondary and primary sources of information.

KEY QUESTIONS ANSWERED - Who are the leading industry players engaged in the development of gene therapies? - How many gene therapy candidates are present in the current development pipeline? Which key disease indications are targeted by such products? - Which types of vectors are most commonly used for effective delivery of gene therapies? - What are the key regulatory requirements for gene therapy approval, across various geographies? - Which commercialization strategies are most commonly adopted by gene therapy developers, across different stages of development? - What are the different pricing models and reimbursement strategies currently being adopted for gene therapies? - What are the various technology platforms that are either available in the market or are being designed for the development of gene therapies? - Who are the key CMOs / CDMOs engaged in supplying viral / plasmid vectors for gene therapy development? - What are the key value drivers of the merger and acquisition activity in the gene therapy industry? - Who are the key stakeholders that have actively made investments in the gene therapy domain? - Which are the most active trial sites (in terms of number of clinical studies being conducted) related to this domain? - How is the current and future market opportunity likely to be distributed across key market segments?

CHAPTER OUTLINES Chapter 2 provides an executive summary of the key insights captured in our research. It offers a high-level view on the current state of the market for gene therapies and its likely evolution in the short-mid term and long term.

Chapter 3 provides a general overview of gene therapies, including a discussion on their historical background. It further highlights the different types of gene therapies (namely somatic and germline therapies, and in vivo and ex vivo therapies), potential application areas of such products and route of administration of these therapeutic interventions. In addition, it provides information on the concept of gene editing, highlighting key historical milestones, applications and various techniques used for gene editing. The also chapter includes a discussion on the advantages and disadvantages associated with gene therapies. Further, it features a brief discussion on the ethical and social concerns related to gene therapies, while highlighting future constraints and challenges related to the manufacturing and commercial viability of such product candidates.

Chapter 4 provides a general introduction to the various types of viral and non-viral gene delivery vectors. It includes a detailed discussion on the design, manufacturing requirements, advantages and limitations of currently available vectors.

Chapter 5 features a detailed discussion on the regulatory landscape related to gene therapies across various geographies, such as the US, Canada, Europe, Australia, China, Hong Kong, Japan and South Korea. Further, it highlights an emerging concept of reimbursement which was recently adopted by multiple gene therapy developers, along with a discussion on several issues associated with reimbursement of gene therapies.

Chapter 6 includes information on over 800 gene therapies and gene editing therapies that are currently approved or are in different stages of development. It features a detailed analysis of pipeline molecules, based on several relevant parameters, such as key therapeutic areas (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, immunological disorders, infectious diseases, inflammatory disorders, liver diseases, metabolic disorders, muscle-related diseases, nervous system disorders, oncological disorders, ophthalmic diseases and others), target disease indication(s), phase of development (marketed, clinical, preclinical and discovery), type of vector used, type of gene, type of gene therapy (ex vivo and in vivo), therapeutic approach (gene augmentation, oncolytic viral therapy and others), route of administration and special drug designation (if any). Further, we have presented a grid analysis of gene therapies based on phase of development, therapeutic area and therapeutic approach.

Chapter 7 provides a detailed review of the players engaged in the development of gene therapies, along with information on their year of establishment, company size, location of headquarters, regional landscape and key players engaged in this domain. Further, we have presented a logo landscape of product developers in North America, Europe and the Asia-Pacific region on the basis of company size.

Chapter 8 provides detailed profiles of marketed gene therapies. Each profile includes information about the innovator company, its product pipeline (focused on gene therapy only), development timeline of the therapy, its mechanism of action, target indication, current status of development, details related to manufacturing, dosage and sales, the companys patent portfolio and collaborations focused on its gene therapy product / technology.

Chapter 9 features an elaborate discussion on the various strategies that can be adopted by therapy developers across key commercialization stages, including prior to drug launch, during drug launch and post-launch. In addition, it presents an in-depth analysis of the key commercialization strategies that have been adopted by developers of gene therapies approved during the period 2015-2020.

Chapter 10 provides detailed profiles of drugs that are in advanced stages of clinical development (phase II/III and above). Each drug profile provides information on the current developmental status of the drug, its route of administration, developers, primary target indication, special drug designation received, target gene, dosage, mechanism of action, technology, patent portfolio, clinical trials and collaborations (if any).

Chapter 11 provides a list of technology platforms that are either available in the market or in the process of being designed for the development of gene therapies. In addition, it features brief profiles of some of the key technologies. Each profile features details on the various pipeline molecules that have been / are being developed using the technology, its advantages and the partnerships that have been established related to the technology platform. Further, the chapter includes detailed discussions on various novel and innovative technologies, along with brief information about key technology providers.

Chapter 12 highlights the potential target indications (segregated by therapeutic areas) that are currently the prime focus of companies developing gene therapies. These include genetic disorders, metabolic disorders, nervous system disorders, oncological disorders and ophthalmic diseases.

Chapter 13 provides an overview of the various patents that have been filed / granted in relation to gene therapy and gene editing technologies. It also features a detailed analysis, highlighting the prevalent trends related to type of patent, publication year, regional applicability, CPC symbols, emerging areas and leading industry players (in terms of number of patents filed). In addition, it features a competitive benchmarking analysis of the patent portfolios of leading industry players and patent valuation. For the purpose of this analysis, we have taken into consideration patents that have been filed / granted since 2016.

Chapter 14 features a detailed analysis of the various mergers and acquisitions that have taken place within this domain, during the period 2015-2020, based on several relevant parameters, such as year of agreement, type of deal, geographical location of the companies involved, key value drivers, highest phase of development of the acquired company product and target therapeutic area.

Chapter 15 presents details on various funding instances, investments and grants reported within the gene therapy domain. The chapter includes information on various types of investments (such as venture capital financing, debt financing, grants, capital raised from IPO and subsequent offerings) received by the companies between 2015 and 2020, highlighting the growing interest of the venture capital community and other strategic investors in this market.

Chapter 16 presents a detailed, geographical clinical trial analysis of completed, ongoing and planned studies focused on gene therapies, based on various relevant parameters, such as trial registration year, trial status, trial phase, target therapeutic area, geography, type of sponsor, prominent treatment sites and enrolled patient population.

Chapter 17 highlights our views on the various factors that may be taken into consideration while deciding the price of a gene therapy. It features discussions on different pricing models / approaches, based on the size of the target population, which a pharmaceutical company may choose to adopt in order to decide the price of its proprietary products.

Chapter 18 highlights top big biopharma players engaged in the field of gene therapy, featuring a heat map analysis based on several parameters, including therapeutic area, type of vector used, therapeutic approach and type of gene therapy.

Chapter 19 features an informed estimate of the annual demand for gene therapies, taking into account the marketed gene-based therapies and clinical studies evaluating gene therapies; the analysis also takes into consideration various relevant parameters, such as target patient population, dosing frequency and dose strength.

Chapter 20 presents an elaborate market forecast analysis, highlighting the future potential of the market till the year 2030. It also includes future sales projections of gene therapies that are either marketed or in advanced stages of clinical development (phase II/III and above). Sales potential and growth opportunity were estimated based on the target patient population, likely adoption rates, existing / future competition from other drug classes and the likely price of products. The chapter also presents a detailed market segmentation on the basis of [A] therapeutic approach (gene augmentation, oncolytic viral therapy, immunotherapy and others), [B] type of gene therapy (ex vivo and in vivo), [C] type of vector used (adeno associated virus, adenovirus, herpes simplex virus, lentivirus, plasmid DNA, retrovirus and others), [D] target therapeutic area (autoimmune disorders, cardiovascular diseases, dermatological disorders, genetic disorders, hematological disorders, metabolic disorders, muscle-related diseases, oncological disorders, ophthalmic diseases and others), [E] route of administration (intraarticular, intracerebellar, intradermal, intramuscular, intratumoral, intravenous, intravesical, intravitreal, subretinal and others), and [F] key geographical regions (US, EU5 and rest of the world).

Chapter 21 provides insights on viral vector manufacturing, highlighting the steps and processes related to manufacturing and bioprocessing of vectors. In addition, it features the challenges that exist in this domain. Further, the chapter provides details on various players that offer contract manufacturing services for viral and plasmid vectors.

Chapter 22 provides a glimpse of the gene therapy supply chain. It discusses the steps for implementing a robust model and provides information related to the global regulations for supply chain. Moreover, the chapter discusses the challenges associated with supply chain of gene therapies. In addition, it features the technological solutions that can be adopted for the management of gene therapy supply chain.

Chapter 23 summarizes the overall report, wherein we have mentioned all the key facts and figures described in the previous chapters. The chapter also highlights important evolutionary trends that were identified during the course of the study and are expected to influence the future of the gene therapy market.

Chapter 24 is a collection of interview transcripts of the discussions that were held with key stakeholders in this market. The chapter provides details of interviews held with Adam Rogers (CEO, Hemera Biosciences), Al Hawkins (CEO, Milo Biotechnology), Buel Dan Rodgers (Founder & CEO, AAVogen), Christopher Reinhard (CEO & Chairman, Gene Therapeutics (previously known as Cardium Therapeutics)), Michael Triplett (CEO, Myonexus Therapeutics), Robert Jan Lamers (CEO, Arthrogen), Ryo Kubota (CEO, Chairman & President, Acucela), Tom Wilton (CBO, LogicBio Therapeutics), Jeffrey Hung (CCO, Vigene Biosciences), Cedric Szpirer (Executive & Scientific Director, Delphi Genetics), Marco Schmeer (Project Manager) & Tatjana Buchholz (Marketing Manager, PlasmidFactory) and Molly Cameron (Corporate Communications Manager, Orchard Therapeutics). In addition, a brief profile of each company has been provided.

Chapter 25 is an appendix, which provides tabulated data and numbers for all the figures included in the report.

Chapter 26 is an appendix, which contains a list of companies and organizations mentioned in this report.Read the full report: https://www.reportlinker.com/p06020737/?utm_source=GNW

About ReportlinkerReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

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Gene Therapy Market by Therapeutic Approach, Type of Gene Therapy, Type of Vectors Used, Therapeutic Areas, Route of Administration, and Key...

Breyanzi (lisocabtagene maraleucel) was approved on the 54 percent complete remission rate achieved in diffuse large B-cell lymphoma trials.

Breyanzi (lisocabtagene maraleucel), a chimeric antigen receptor (CAR) T cell-based gene therapy to treat adult patients with certain types of large B-cell lymphoma who have not responded to, or relapsed, after at least two other types of systemic treatment has been approved by the US Food and Drug Administration (FDA).

According to the agency, Breyanzi is the third gene therapy approved in the US for certain types of non-Hodgkin lymphoma, including diffuse large B-cell lymphoma (DLBCL). Breyanzi is not indicated for the treatment of patients with primary central nervous system lymphoma.

Todays approval represents another milestone in the rapidly progressing field of gene therapy by providing an additional treatment option for adults with certain types of cancer affecting the blood, bone marrow, and lymph nodes, commented Dr Peter Marks, director of the FDAs Center for Biologics Evaluation and Research. Gene and cell therapies have evolved from promising concepts to practical cancer treatment regimens.

DLBCL is the most common type of non-Hodgkin lymphoma in adults. Approximately 77,000 new cases of non-Hodgkin lymphoma are diagnosed in the US each year, with DLBCL accounting for around a third of newly diagnosed cases.

Breyanzi is customised for each patient; their T cells, a type of white blood cell, are collected and genetically modified to include a new gene that facilitates targeting and killing of the lymphoma cells. Once the cells are modified, they are infused back into the patient.

The safety and efficacy of the treatment were established in a multi-centre clinical trial of more than 250 adults with refractory or relapsed large B-cell lymphoma. The complete remission rate after treatment with Breyanzi was 54 percent.

The treatment can cause severe side effects, including cytokine release syndrome (CRS), which is a systemic response to the activation and proliferation of CAR T cells, causing high fever and flu-like symptoms and neurologic toxicities. Both CRS and neurological events can be life-threatening, so the therapy is being approved with a risk evaluation and mitigation strategy (REMS) which includes elements to assure safe use (ETASU).

The requirements include, among other things, that healthcare facilities that dispense Breyanzi be specially certified, with staff involved in the prescribing, dispensing or administering of the treatment being trained to recognise and manage the risks of CRS and neurologic toxicities.

Other side effects include hypersensitivity reactions, serious infections, low blood cell counts and a weakened immune system. According to the FDA, side effects generally appear within the first one to two weeks following treatment, but some side effects may occur later.

To further evaluate the long-term safety, the FDA is also requiring the manufacturer to conduct a post-marketing observational study involving patients treated with Breyanzi.

The approval was granted to Juno Therapeutics Inc., a Bristol-Myers Squibb Company.

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FDA approves third gene therapy for large B-cell lymphoma - European Pharmaceutical Review

REDWOOD CITY, Calif., Feb. 10, 2021 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq: ADVM), a clinical-stage gene therapy company targeting unmet medical needs in ocular and rare diseases, today announced the presentation of data from Cohorts 1-4 of the OPTIC Phase 1 clinical trial of ADVM-022 intravitreal (IVT) injection gene therapy in patients requiring frequent anti-VEGF injections for their wet age-related macular degeneration (AMD). These data were previously presented as part of a corporate update on Saturday, November 14, 2020.

Oral Presentation Title: Intravitreal Gene Therapy for Exudative AMD and Diabetic RetinopathyDate and Time: Saturday, February 13, 2021 at 9:30 am ETSession V: Gene Therapy for Exudative AMD and Diabetic RetinopathyPresenter: Arshad M. Khanani, M.D., M.A., Director of Clinical Research, Sierra Eye Associates

A copy of this presentation will be available as the presentation begins in the publications and presentation section of Adverums website.

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

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Adverum to Present Data from the OPTIC Phase 1 Trial with ADVM-022 Intravitreal Gene Therapy in Wet AMD at the Angiogenesis, Exudation, and...