Despite the attention being paid to how genetics affect our risk for certain diseases, inherited gene mutations are not the contributing cause to most breast cancer diagnoses. About 85% of breast cancers occur in women who have no family history. These cases occur due to genetic mutations that happen as a result of the aging process and lifestyles in general. What are the contributing lifestyle factors?

The Physicians Committee for Responsible Medicine (PCRM) and Dr. Kristi Funk, breast cancer surgeon and author of Breasts: The Owners Manual have teamed up in a campaign to educate women (and men!) about the ways that a healthy diet and lifestyle can help reduce our risk for breast cancer. Here are their recommendations:

Choose mostly plant-based foods Plant foodssuch as vegetables, fruits, whole grains, and beans lower breast cancer risk in several ways. They help with weight loss, because they are typically low in calories and high in appetite-taming fiber. In addition, high-fiber, low-fat diets can help you gently reduce estrogen levels. In turn, lower estrogen levels can lower your risk of cancer.

Exercise regularly Physical activity lowers the risk of breast cancer. Evidence suggests that exercise helps with weight loss, and it also strengthens immune defenses, which may help the body kill cancer cells that arise. If you are sedentary now, it helps to start exercising slowly and build up gradually. Briskly walking for 10 or 15 minutes three times per week is a good beginning. You can then add 5 minutes to each walk until you are walking for 30 or 40 minutes at a time. When you feel ready to take it up a notch, you can add running, swimming, cycling, or other activities you find enjoyable.

Limit alcohol Alcohol increases breast cancer risk. This is true for all kinds of alcohol, including beer, wine, and liquor. Even one drink a day increases risk. The less you drink, the lower your risk. Alcohol can increase estrogen levels, and it can cause DNA damage the first step in cancer.

Maintain a healthy weight Excess body weight increases the odds of getting breast cancer after menopause. Extra weight can also make cancer more likely to advance when it arises. Fat cells produce estrogens female hormones that can help cancer cells to form and spread.

As we learn more about the things that contribute to the formation of cancer, we can begin to reduce our risk for breast cancer to occur or to come back if youve already been diagnosed. You can learn more about the PCRM Lets Beat Breast Cancer campaign at pcrm.com/letsbeatbreastcencer.

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BEYOND TRIM: Don't give breast cancer a helping hand - SaukValley.com

Recommendation and review posted by Bethany Smith

Healthy women who have known risk factors for developing breast cancer should talk to their doctor about taking medication to prevent the disease, says a new recommendation from a national panel of health experts.

The new guidelines from the U.S. Preventive Services Task Force (USPSTF) recommend that clinicians consider prescribing tamoxifen (Soltamox), raloxifene(Evista), or aromatase inhibitors to women age 35 and up who are at higher than average risk for breast cancer.

What degree of risk warrants preventive medication must be determined by a woman and her doctor. Among the factors that will be considered include family history, genetics, and whether you havedense breast tissue.

The new recommendations which were published last month in the Journal of the American Medical Association and on the USPSTF website update a similar version published in 2013.

They differ in that they now include an additional class of medication, aromatase inhibitors, on the list of drugs believed to help reduce womens risk. (Aromatase inhibitors have not yet been approved by the Food and Drug Administration specifically for the purpose of preventing breast cancer.)

Although their particular mechanisms vary, the breast cancer medicines recommended by the task force all work by lowering or blocking estrogen a female hormone to decrease breast cancer risk. The medication must be taken daily for a period of five years.

RELATED: Speaking Genetics: A Glossary of Cancer Risk Gene Mutations

In 10 trials reviewed by the task force, all three types of drugs reduced invasive and ER-positive breast cancer but not ER-negative breast cancer. If 1,000 women at higher risk took these medications for five years, youd expect somewhere between 7 and 16 fewer cases of breast cancer, says task force member Michael Barry, MD, medical director of the John D. Stoeckle Center for Primary Care Innovation at Massachusetts General Hospital in Boston.

Again, thats out of 1,000, so it may seem like a small number, he says. But of course if youre one of those women, it might be quite important.

The USPSTF stresses that the medication should be considered only for women whose odds of developing breast cancer are higher than average but determining those odds is not always easy.

Widely available risk-assessment models such as the Gail model, the first used clinically, weigh factors including (but not limited to) a womans age, whether she has first-degree relatives with breast cancer, and how old she was when she first got her period.

But such tools are imperfect, and there is no single definition for what constitutes being at higher risk, according to the American Cancer Society.

Although there are a lot of models, they are very rough guidelines, says Leif W. Ellisen, MD, PhD, program director of Breast Medical Oncology at Massachusetts General Hospital Cancer Center in Boston, who was not a member of the task force. Thats challenge number one.

According to Dr. Ellisen, the second challenge for primary care providers is analyzing potential risk versus benefits of these medicines. If we say, Well, this could cut your risk for breast cancer in half if you just take the medicine for five years, that sounds pretty good. But if the womans risk is already quite low, it may not be very meaningful.

Whats more, the medication is not without side effects. Mild side effects include hot flashes, fatigue, and mood swings. More serious ones may include blood clots and other forms of cancer.

RELATED: The FDA Tightens Regulations on Mammograms, Especially for Women With Dense Breasts

Many women who are at increased risk may never learn about the option of taking preventive medication. The task force notes that only 10 to 30 percent of primary care doctors say they have prescribed breast cancer prevention medication. And most of those say they have done so only a handful of times.

I do think most women have never heard of these, says Mary B. Daly, MD, PhD, a professor in the department of clinical genetics at the Fox Chase Cancer Center in Philadelphia, who was not a member of the task force. And I think prescribing them has never really been in the realm of primary care.

Dr. Daly advises patients with a family history of breast cancer to mention this to their doctors during the context of family history. Similarly, if a woman is concerned about other risk factors, such as dense breasts, she should mention it.

If their primary care doctor doesnt feel comfortable discussing these [medications], a referral to a breast cancer risk specialist is indicated, says Ellisen.

See the original post here:
New Guidelines: Breast Cancer Prevention Drugs - Everyday Health

Recommendation and review posted by Bethany Smith

Exclusive Interview with New West Genetics President and CEO Wendy Mosher

New West Genetics is focused on bringing stable genetics to the hemp business. President and CEO Wendy Mosher spoke with New Cannabis Ventures about the companys experienced team, its research and development efforts and how she sees the competitive landscape for hemp genetics. The audio of the entire conversation is available at the end of this written summary.

100+ Years of Agriculture Experience

The New West Genetics team has extensive experience in agriculture. Moshers background is in education, but she spent many summers working in labs. Dr. John McKay, a fellow co-founder of the company and Moshers husband, is a plant geneticist. Over the 25 years they have been together, Mosher learned about genetics and found herself attracted to its practical applications.

When Dr. McKay and Dr. Richard Fletcher decided to start a business, Mosher was the perfect fit to lead it. Neither Dr. McKay nor Dr. Fletcher had any business experience, but Moshers time as a teacher equipped her with the skills to facilitate teamwork while pursuing goals.

Drs. McKay and Fletcher bring experience in plant genomics and breeding. The team also includes Daphne Preuss, Chief Business Officer; Frank Curtis, Director of Operations; John Berlejung, R&D Production Supervisor; and Sarah Hirstwood, Executive Assistant to the CEO.

Preuss comes from a genetics background. She was a professor at the University of Chicago, and she started a successful sorghum breeding company. Curtis, experienced in adapting crops, led a wheat program for 12 years before joining New West Genetics. Berlejung has an MSc. in soil and crop science. Hirstwood comes from a marketing background and leads the companys social media and marketing initiatives. The company also just added agronomist Daniel Willis to the team.

Stable Seeds and Extraction

Genomic knowledge in the hemp industry is 100 years behind a crop like corn, and the industry desperately needs stable genetics, according to Mosher. New West Genetics is focused on creating and licensing stable, proprietary seeds. Creating those stable seeds takes years. The company began in 2014, and its first stable seed was certified in 2017. The companys seeds are certified by the Association of Official Seed Certifying Agencies (AOSCA).

The company has received patent claims for some of its novel varieties, and it is submitting more claims to protect its work. New West Genetics has a pipeline of new varieties rolling out every year.

In addition to its seed licensing model, the company partners with extractors. Mosher is not concerned that uncertainty in the regulatory environment for CBD will affect New West Genetics. She sees continued demand for hemp-based extract and looks forward to clarified regulations.

Research and Development

Hemp genomics R&D is a part of the New West Genetics strategy. Dr. McKay publishes research through his work at Colorado State University. Recently, he has explored the combined impact of genetics and environment. He found that cannabinoids are mostly (95 percent) controlled by genetics, rather than the environment. As in the world of science, the study will need to be replicated.

The company has also worked on developing enhanced cannabinoids to make extraction more efficient. For example, New West Genetics has a zero THC product. The breeding process means concentrated THC does not need to be removed at the end of the extraction process, saving more than $1,000 per kilo, according to Mosher.

New West Genetics has also developed a genetically skewed gender variety of hemp. Hemp is naturally dioecious, meaning male and female flowers are found on separate plants. The company has skewed the ratio to 90 percent female because having seed is valuable and scalable. In contrast, the common method of chemical feminization means the producer gets no seeds and must redo the costly process each season, according to Mosher. New West Genetics is aiming to make access to cannabinoids more efficient and high-yielding.

Partnerships

Forming partnerships is one of the challenges New West Genetics has faced as it continues to grow. Finding commercial partners with the capital and expertise to deliver on promises did not become truly viable until after the 2018 Farm Bill, according to Mosher. The company has also found it beneficial to work with university partners.

The Competitive Landscape

On the seed side, New West Genetics serves companies creating wholesale seed for grain. On the extraction side, it serves operators in the hemp and food industries. For the first four years of operation, the company was protected by regulations and the inability of many states to participate in the industry.

The company now has a significant head start as more competitors enter the space. It is too early to determine market share, but Mosher believes the company has the most scalable seed-based hemp genetics. She welcomes the competition as a necessity for building a robust industry but also recognizes the importance of capital when it comes to staying ahead of that competition.

Funding and Future Growth

At the end of 2018, New West Genetics raised $3 million. That capital has been used to invest in equipment, particularly important as the company ramps up to production mode, and high-quality team members. As the company focuses on sales, Mosher is planning to hire someone with business development, sales, and seed licensing experience.

Mosher does not see an IPO in the companys future, but it has always considered strategic partnerships.

This year is the companys first step into large-scale production and its first year for revenue after years of developing its stable genetics. Mosher expects revenue to grow 5x annually over the next four years.

As the company grows, it will be focused on the continued development of genomic knowledge. Mosher is looking forward to the industrys work on USDA compliance and the opportunity of hemp in animal feed.

To learn more, visit the New West Genetics website. Listen to the entire interview:

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New West Genetics is Bringing Stable Seeds to the Hemp Industry - New Cannabis Ventures

Recommendation and review posted by Bethany Smith

Its an age-old question that many social scientists would love to have a definitive answer for are entrepreneurs born or made?

While there have been many studies of the genetics of entrepreneurs, on the face of it, there are several shared mental attributes among those who flourish. We dont need to delve too deeply into the psychological side to recognise the more obvious qualities: being highly motivated, confident, persistent, intuitive and impulsive all behaviours which can be learned by people who dont possess a special gift.

So, could any business owner adopt a similar winning mindset and have a positive impact on their companys outcome?

37pc-48pc of the tendency to be an entrepreneur is genetic

One study carried out by Kings College London into the behavioural and molecular genetics of entrepreneurship found that 37pc-48pc of the tendency to be an entrepreneur is genetic. The study assessed a general pool of 3,000 people, testing the number of businesses a person had started, how long they were self-employed for, and factors such as the desire to run a business. Even if those figures do paint an accurate picture, that still leaves us with 52pc-63pc who doesnt fall into the genetically gifted category.

Whether entrepreneurship is in the blood or not, there are several well-known executives who clearly had a natural entrepreneurial gift from an early age. Take Sir Richard Branson who started selling records from a church, or Lord Sugar, who sold car radio aerials and other electrical goods out of a van when he was a teenager. Humble beginnings and now billionaire business owners.

You cant determine where you start in life, but you can determine where you end up

On the other hand, we have those who had careers in different fields but were driven by entrepreneurial foresight, ambition and a determination to succeed. For example, Karren Brady began her career in advertising sales straight after leaving school and, aged just 23, was appointed managing director of Birmingham City Football Club. Now shes one of the most high-profile, respected business leaders in the UK. Also, Lady Michelle Mone, who was made redundant from a sales and marketing role at a brewing company before having a lightbulb moment which took her from designing bras to founder of the nations leading lingerie brand.

Ask those women the secret to success and you can bet your bottom dollar they wouldnt say it was just down to genetics. As Karren Brady says on her website: You cant determine where you start in life, but you can determine where you end up.

In other words, if people want to start their own business and really make it work, taking certain behavioural steps could help them on their journey. By adopting a different mindset, it will allow people to think and act like the successful entrepreneur they aspire to be.

It would be interesting to see from genetic studies whether a persons gender impacts their probability of being entrepreneurs. As champions of Women in Business, we aim to address the different challenges and barriers facing women when they rise to the top of their profession at our forthcoming Women in Business event, to be held in Farnborough October 16-17. Only one third of UK entrepreneurs are women, so there is a pool of untapped female potential out there who just need the right support and guidance to embrace their entrepreneurial spirit.

Genetics and gender aside, there are skills and behaviours which could be learned to get people into the entrepreneurial mindset.

See also: Everyone knows JK Rowling, but what about other women in business?

Here, we explore some of the most common traits of todays successful business leaders:

An entrepreneurial mindset involves having a steadfast commitment to a defined vision. This unwavering focus remains throughout the up and downs and daily demands of running the business. The end goal should always be front of mind and that will drive you to carry out the necessary steps to accomplish your vision. Strategic planning and thinking are critical for every business owner. Keeping eyes fixed on the big picture means you can see what direction the industry is going in, identify challenges for the company and devise the right solutions to meet your overall initiatives.

A fundamental characteristic of all successful entrepreneurs is their level of confidence in both their ideas and their ability. Buyers and investors will only believe in ideas if the business owner truly believes in them themselves. Be open to constructive feedback or critique but stay completely convinced that your business idea will yield positive results and that you have the capacity to make it happen. Carrying out in-depth research into your business idea will help to dispel any doubts. Know your market, your USP and your key personal strengths to bolster your self-belief.

Being willing to take risks and move outside of your comfort zone is all part of the journey. Entrepreneurs have amazing resilience and thrive off turning around negativity. Risks are inherent in any new venture and we are frequently told by those who have made it that failure is an inevitable part of success. Its crucial that you frame failure as an opportunity to learn. The path to success will hopefully have an upwards trajectory but it is rarely a straight line. Any failure should drive you forward. Observe and absorb what you have learned and use it to progress.

Successful entrepreneurs leverage their strengths and understand that they cant do everything. All business people are naturally better at some things and where there are areas of weakness or lack of knowledge that is the time to outsource and seek external help. Knowing that you cant tackle every obstacle on your own is a strength. If bringing people in house is ruled out on financial grounds, hire consultants to look at the more complex, labour-intensive parts of the business. That will ensure you can focus on the overarching business strategy and not get bogged down by minutiae.

The ability to adapt to change quickly is a key entrepreneurial attribute. Whether theres a new competitor springing on to the scene or dip in demand in the target market. Being flexible means having the courage and conviction to rethink a situation, keeping track of feedback about pricing, products and services and making tweaks when necessary. The path of an entrepreneur will occasionally go off course and flexibility is an important skill to keep you on track.

Christie Day is event director for Women in Business Expo, which takes place October 16-17 at Farnborough International Conference & Exhibition Centre

Start young, sleep less: New study reveals leadership formula

Visit link:
5 habits of highly successful entrepreneurs revealed - GrowthBusiness.co.uk

Recommendation and review posted by Bethany Smith

Oct 04, 2019 10:49 AM EDT

The old saying that people marry their parents may be true for poison dart frogs, and it may even lead to the formation of new species, according to a new study inNaturebased on work at the Smithsonian Tropical Research Institute (STRI).

Strawberry poison dart frogs live on the mainland in Panama's Bocas del Toro province and have been isolated on islands in the archipelago that formed during the past 10 million years as sea level rose. Only a single color morph exists on some islands--orange or green, for example, but on oth-er islands several color morphs exist together, like blue and red frogs.

"In the past, people assumed that this group of brightly colored poison dart frogs were warning predators that their skin is toxic," said Corinne Richards-Zawacki, research associate at STRI and pro-fessor of biological sciences at the University of Pittsburgh. "But predators don't seem to care what color the frogs are, at least based on our earlier experiments. That's why we started asking whether the way they choose mates might lead to populations of different colors on different islands."

The team set up three different situations: baby frogs raised with two parents of the same color (red baby, red parents), baby frogs raised with each parent a different color (red baby, one red and one blue parent) and baby frogs raised by foster parents of a different color (red baby, blue parents). In each case they asked which color the female offspring would choose as mates and which color the male offspring would perceive as a rival.

"We discovered that female frogs with parents of the same color tended to choose mates of that same color, whereas frogs with foster parents of a different color would choose mates the color of the foster parents," said Yusan Yang, who is completing her doctoral thesis at the University of Pitts-burgh. "The same was true for male-male aggression. This tells us that imprinting was more important than genetics when it comes to shaping these behaviors that are based on color."

When baby frogs were raised with one parent of the same color and one parent of a different color, females chose mates the color of their mother, and males chose rivals the color of their mother, indicating that maternal imprinting was probably more important than paternal imprinting.

They also created a mathematical model showing that male aggression based on imprinting, in concert with female mate choice based on imprinting was enough to cause a scenario to evolve, where like mates with like, which could lead to two color morphs becoming separate species.

"We're fascinated by the idea that behavior can play such an important role in evolution," Richards-Zawacki said.

2018 NatureWorldNews.com All rights reserved. Do not reproduce without permission.

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Imprinting on Mothers May Drive New Species Formation in Poison Dart Frogs - Nature World News

Recommendation and review posted by Bethany Smith

FELTON, California, Oct. 7, 2019 /PRNewswire/ -- The global Stem Cell Market was valued at US$ 8.65 billion in 2018 and is estimated to grow at an 8.8% CAGR and will touch the value of US$ 15.63 Billion by the completion of the year 2025.

During the previous insufficient years, stem cells therapy has been attaining grip all over the world. A substantial growth in the number of clinical use of stem cells and the arrival of new-fangled treatments for long-lasting sicknesses are projected to boost the development of the global stem cells market during the following insufficient years. Moreover, the growing investment by community along with private groups for research actions are expected to increase the general development of the market during the nearby future.

Classification:

The global stem cell market can be classified by Therapy, Technology, Application, Product End User, and Region. By Therapy it can be classified as Allogeneic Stem Cell Therapy, Autologous Stem Cell Therapy. The subdivision of Autologous Stem Cell therapy was the leading sector by means of generation of income in 2018. It is credited to big scale ingestion of these products owing to associative high compatibility. Furthermore, growing methodical evaluations and meta-analysis revisions on reviewing the efficiency of autologous cell therapy on treatment of lower limb illnesses will more offer thrust to the market.

By Technology it can be classified as Expansion and Sub-Culture, Cryopreservation, Cell Production, Cell Acquisition. The subdivision of Cell Acquisition technology is the elementary main step that has caused supremacy of this section. It is likely to uphold this position during the course of the forecast period, due to increasing alertness about the importance of stem cells. Bone marrow is the maximum utilized technology for cell acquisition due to the comparatively quicker manufacture of new cells from bone marrow. Additionally, cells that initiate from bone marrow are extra concentrated as equated to additional origin places.

Get Sample PDFand read more details about the "Stem Cells Market" Report 2025.

By Application it can be classified as Drug Discovery and Development, Regenerative Medicine. The subdivision of Regenerative Medicine is witnessed to grasp the prospective for creating early-intervention treatments to treat degenerative illnesses and painful injury. Additionally, obtainability of regenerative medicine through a widespread variety of clinical areas is motivating the development of the section.

By Product it can be classified as Very Small Embryonic like Stem Cells, Human Embryonic, Induced Pluripotent Stem Cells, and Adult Stem Cells. The subdivision of Adult Stem Cell detained the biggest share of the market which was prized US$ 7.38 billion in 2018. It is projected to carry on leading for the duration of the forecast. This is credited to the low-slung hazards of pollution associated to sub-culturing, negligible necessity of labor force for production and compatibility with humanoid figure. By End User it can be classified as Service Companies, Cell and Tissues Banks, Tools and Reagent Companies, Therapeutic Companies.

Regional Lookout:

By Region, the global stem cells industry can be classified as North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. Asia Pacific is expected to record speedy CAGR for the duration of the forecast. The nations such as Singapore, Australia, and Japan are mainly capitalizing in the R&D projects. This is set to motivate the development of the region. Economies from Asia Pacific are likely to be at the front position of speedily developing cell industry. Issues similar to advantageous controlling strategies together with openings for commercialization increase the provincial growth. Controlling alterations relating to regenerative medication in Japan have fascinated worldwide companies to capitalize in the Japanese market.

North America is expected to carry on holding the foremost market share during the period of forecast, due to the hard work from government and private segments operational in the direction of formation of distinct business models in Canada and the U.S.A.

Companies:

The companies are opting for multidisciplinary commercial growth and multi-sector team work to safeguard incessant source of great quality pluripotent and distinguished cells. This is set to step up the rivalry, motivating the continuous necessity to present innovative products.

Some of the important companies for stem cell market are Promethera Biosciences, Human Longevity Inc., Cytori Therapeutics, BIOTIME, INC., STEM CELL Technologies Inc., Mesoblast, Cynata, Advanced Cell Technology Inc., Osiris Therapeutics Inc., and Celgene Corporation.

Browse 190 page research report with TOC on "Global Stem Cells Market" at: https://www.millioninsights.com/industry-reports/stem-cells-market

Market Segment:

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Stem Cells Market to Gain Huge Traction Owing to Arrival of New-Fangled Treatments for Long-Lasting Sicknesses Till 2025 | Million Insights - P&T...

Recommendation and review posted by Bethany Smith

NEW YORK, Oct. 07, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc.(NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced that the United States Patent and Trademark Office (USPTO) has issued a Notice of Allowance for BrainStorm's new US Patent Application, number: 15/113,105, titled: Method of Qualifying Cells'.

The allowed claims cover a pharmaceutical composition for MSC-NTF cells secreting neurotrophic factors (NurOwn) comprising a culture medium as a carrier and an isolated population of differentiated bone marrow-derived MSCs that secrete neurotrophic factors.

Patent families protecting NurOwn have previously issued in the United States, Japan, Europe, Hong-Kong and Israel.

"This allowance further expands the patent protection of the NurOwn Cellular Therapeutic Technology Platform and enables us to accelerate clinical development for new neurodegenerative indications, commented BrainStorm President and CEO,Chaim Lebovits.

About NurOwn

NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm is currently conducting a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently received U.S. FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began in March 2019.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm is currently enrolling a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently received U.S. FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive Multiple Sclerosis. The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment in March 2019. For more information, visit the company's website at http://www.brainstorm-cell.com

Safe-Harbor Statements

Statements in this announcement other than historical data and information constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, risks associated with BrainStorm's limited operating history, history of losses; minimal working capital, dependence on its license to Ramot's technology; ability to adequately protect the technology; dependence on key executives and on its scientific consultants; ability to obtain required regulatory approvals; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

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BrainStorm Announces Notice of US Patent Allowance for NurOwn Cellular Therapeutic Technology Platform - Yahoo Finance

Recommendation and review posted by Bethany Smith

Blood, of course, plays a crucial role in keeping our bodies alive and functioning.

Red blood cells carry oxygen from our lungs to our muscles. White blood cells are the first responders of our immune systems, detecting infections and foreign agents and triggering the immune response needed to deal with the problem. Plasma, the liquid part of blood, transports not only the cells but also proteins, such as antibodies, and hormones, such as insulin, to every part of the body. It is a beautifully complex system that is the key to our bodies functioning correctly.

Parasites, viruses and bacteria all use the circulatory system to spread around the body. When cancer metastasises and spreads to other parts of the body, it is through tumour cells circulating within the bloodstream. There are also myriad blood cancers, such as leukaemias and lymphomas, and blood disorders, such as sickle cell anaemia, not to mention autoimmune diseases like diabetes or lupus. Sepsis, also known as blood poisoning, is a deadly overreaction to an infection which also has its roots in blood.

Magnetic blood filtration is a tool which enables the physical removal of specific substances from the bloodstream

Most of these diseases are treated with drugs or chemotherapies, some with great success, others, much less. There are also various methods of physically extracting different components from the blood by circulating a patients blood outside of the body through whats known as an extracorporeal circuit. Dialysis, for example, removes excess toxins from the blood, acting as a substitute for kidneys when they fail. Plasmapheresis and leukapheresis are methods of removing harmful antibodies from the plasma or white blood cells from the blood. Similar techniques are also used to harvest stem cells from the blood, which can then be used in cancer therapies (known as stem cell transplantation), if the donor and patient are a match.?

?Magnetic blood filtration

Despite these methods, millions of people still die every year from blood-borne diseases. At MediSieve a small, London-based start-up company we are using nanotechnology to develop a new technology which we think can transform our ability to tackle these conditions. Magnetic blood filtration (MBF) is a tool which enables the physical removal of specific substances from the bloodstream. It is similar to other extracorporeal procedures like dialysis, but instead of removing every component of a particular size or weight, MBF removes highly specific targets to address the specific medical issue, thereby removing only the substance that doctors want to remove. Alongside this high specificity, various targets, both big and small, can be removed simultaneously, raising the prospect of removing for example specific cells and harmful antibodies in a single procedure.

To achieve this, we use magnetic nanoparticles coated with binding moieties, such as antibodies, which bind specifically to the desired targets in the blood. These particles are infused into the blood within the extracorporeal circuit, binding to their targets. The blood then flows through a magnetic filter which captures the magnetic particles and the targets bound to them, while the rest of the blood flows back into the patient. Several different particles can be used in the same procedure in order to capture different components. Using this method, anything for which there is a specific antibody or other binding moiety can theoretically be removed directly from the bloodstream. I think that in the long-term the technology could be used to remove specific cells, antibodies, bacteria, viruses, toxins, drug molecules and inflammatory cytokines, the drivers of immune conditions such as sepsis.

The MediSieve Filter is a disposable, single-use device in which the magnetic particles and their targets are captured. It is inserted into the MediSieve Magnet, a reusable medical device which activates the filter. (The technology ???) Both can be incorporated into a variety of existing extracorporeal systems and integrate with standard blood pumps and tubing sets.

We are currently developing treatments for malaria, sepsis and leukaemia. The Filter and Magnet have completed pre-clinical testing and are now ready for clinical trials, which we hope to start shortly. Magnetic particles for various clinical targets are currently being developed and validated in the laboratory, with promising results. Animal trials for these are expected to start in 2020.

Malaria

Our potential treatment for malaria is the closest to market because malaria infected cells, uniquely, have naturally occurring magnetic properties is it therefore possible to remove them from the blood using the MediSieve Filter without the infusion of any magnetic particles. The magnetic properties arise from a core aspect of the malaria parasites lifecycle. After infecting a red blood cell, the parasite consumes the protein part of haemoglobin, leaving behind an iron-based waste-product known as haemozoin, which is stored inside the cell. Haemozoin is paramagnetic, thereby giving infected cells their unique magnetic properties.

MBF could be used in highly severe malaria cases in which the patient is hospitalised and at high risk of death. Currently, these patients receive intravenous drugs such as artesunate which can achieve parasite clearance in 36-48 hours; parasite clearance rate is the key indicator of patient recovery, and it can take up to eight doses of IV drugs to achieve complete clearance. Mortality in these cases can be as high as 20%.?

Using MBF alongside the first dose of IV drugs could drastically accelerate parasite clearance rate. We claim that, depending on the patient size and initial level of infection, this approach can remove over 90% of red blood cells containing haemozoin in just two hours. Because they have higher quantities of haemozoin, MBF is better at removing later stage infected cells, whereas drugs are much more effective against earlier stage cells, so they should be complimentary.

MBF has the additional benefit of removing free circulating haemozoin, also known as the malaria toxin, which should also improve the treatment for the patient since drugs can cause the large-scale release of haemozoin as infected cells die.

According to the WHO, in 2017 there were 219 million cases of malaria and 435,000 deaths, mostly children. While overall malaria cases and deaths have been trending downwards in recent years, the number of hospitalised patients is increasing as healthcare infrastructure improves in malaria endemic countries and more patients gain access to hospitals. In the future, MBF could be adapted for use in mobile clinics to reach harder to access areas.

While ourinitial target is severe malaria patients, I also believe MBF could be a valuable tool in the fight against drug-resistant malaria strains, which have been emerging in SE Asia and are causing great concern if drug resistance spreads to Africa, the effect could be catastrophic. It can also be used to treat patients for whom drugs cannot be used, such as pregnant women.

Sepsis

Sepsis is one of the leading causes of death in the developed world with more than 1.9M cases in Europe and the US and published mortality rates of 29% - 50%. Sepsis is a complex syndrome in which bacteria or other pathogens create a dysregulated immune response which can escalate to organ failure and death. The immune response creates an overproduction of pro-inflammatory cytokines, while cell damage over time creates damage-associated molecular patterns (DAMPs) that sustain the syndrome. ?

Our approach to sepsis, which we call SepSieve, uses a cocktail of different particles to remove a number of targets from a patients bloodstream: specific pro-inflammatory cytokines (IL-1, IL-6 and IL-18), DAMPs (HMGB-1), endotoxins (LPS), and gram-negative bacteria. This multi-modal approach tackles the disease from two key angles: Removing the pathogens and endotoxins that trigger the immune response and reducing magnitude of the immune response and preventing the cascade towards septic shock.

Like in malaria, SepSieve would be used alongside existing frontline treatments, specifically antibiotics. While antibiotics are critical for treatment of sepsis, the bacterial cell death they cause releases LPS which accelerates the dysregulated immune response MBF could remove the LPS to prevent the condition from worsening. The main benefit of MBF in sepsis is therefore not so-much the removal of bacteria itself (which is tackled by antibiotics and in any case is not present exclusively in the bloodstream), but rather the removal of all the other components driving the disease.

Gram-negative bacteria such as E. coli account for approximately 50% of sepsis patients, but thanks to the removal of other substances, particularly HMGB-1 and the inflammatory cytokines, I think the combined approach could benefit all sepsis patients. Since magnetic filtration is a purely physical method, it can also target and remove pathogens which are resistant to antibiotics, which again are a huge concern with increasing occurrences of resistant infections in hospitals.

Like in malaria, wwe plan to apply sepsis treatment to hospitalised patients and specifically those in Intensive Care Units. These are the most severe cases and those who stand to benefit the most from the treatment. The idea is to intervene early to prevent the sepsis cascade, in which the disease escalates eventually causing organ failure and death.

In fact, we managed to secure grants worth a total of 1.56M from Innovate UK, the UKs government grant funding body, and the UK National Institute of Health Research to develop and validate our sepsis particles. Currently being tested in human blood models in the companys laboratories, we plan to start animal trials in 2020 which, if successful, will be followed by clinical trials in 2021.

Leukaemia

One of the advantages of the particles we develop to remove pro-inflammatory cytokines for sepsis is that they can also be used in other diseases. This includes auto-immune diseases and cytokine storms such as cytokine release syndrome (CRS), a common side-effect of newer leukaemia treatments known as CAR T-cell therapies.?

In CAR-T therapies, T-cells, a type of white blood cell, are modified to attack cancer cells in a patients bone marrow. Taken either directly from the patient or from a matching donor, the modified cells are infused into the patient in order to directly attack the cancer. Results of clinical trials have been mixed, but these cell therapies are seen as a huge leap forward for leukaemia treatment.?

The problem is that the infused T-cells trigger massive immune reactions within the patient. Indeed, that is the intention the immune reaction is intended to kill the cancer cells but it can easily escalate into the condition called CRS. The result is similar to sepsis an immune over-reaction which attacks the patient and can be fatal. Immune mediators can be used to calm this reaction, but they then prevent the infused CAR-T cells from having their effect, eliminating the therapeutic benefit of the treatment.

Our proposal is to use MBF in CRS patients to remove cytokines from the bloodstream. This should calm the immune reaction, alleviating patient suffering and eliminating the risk of death. But since MBF only removes cytokines from the bloodstream, it shouldnt affect the immune effect of the CAR-T cells in the bone marrow, so the therapeutic benefit should be maintained. In addition, MBF can be stopped at will, so it can be used to control the immune response by maintaining the correct balance of cytokines this is of course not possible with immune mediators which are infused into the patient.

A further benefit that MBF can provide in leukaemia patients is the removal of leukaemia cells from the bloodstream leukaemia patients commonly have very high white blood cell counts due to circulating leukaemia cells. These cause a number of issues such as a reduction in immune function, making patients more vulnerable to infection. They can also prevent certain chemotherapies from working effectively, since they block the drug from targeting cancer cells in the bone marrow. High white blood cell counts also increase the risk of side-effects during treatment, since the sudden death of such a large numbers of cells causes debris to circulate in the blood, putting strain on the body and causing immune reactions like CRS; this is known as Tumour Lysis Syndrome. ?

We are currently focussing development on their sepsis particles, but plan to trial their cytokine particles in CRS at the same time as they are trialled in sepsis, since the pre-clinical validation for each disease is the same. The particles to remove white blood cells, however, are at an earlier stage and will be developed further down the line.

Our ambitions for MBF are certainly large. In the long-term we want to revolutionise the way in which blood-borne diseases are treated. Going far beyond malaria, sepsis and leukaemia, we want to develop treatments for all blood-borne diseases if its in the blood, and doctors want it out, we want to be able to take it out.

My vision is that hospitals all around the world will have Magnetic Blood Filtration Units which will address a huge variety of patients. Only time will tell if this can be achieved, or even if our technology will work at all after all, there have, as of yet, been no clinical trials.

However, the ability to remove specific substances from blood would clearly be of benefit to huge numbers of patients. It is something that we cannot do today, but we certainly should want to be able to do tomorrow. Whether it is MediSieve who gets us there or not remains to be seen.

Author:

Dr George Frodsham is CEO and founder of MediSieve

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It's in the blood - Lab News

Recommendation and review posted by Bethany Smith

Whats more savage than submitting an extremely rare, aggressive form of cancer? Beating it a second time, which is what one brown belt and his jiu-jitsu community are currently hellbent on doing. Steve Springer, a father, husband, and one of those relentlessly positive mat rats who lights up the gym, just made the 1100 mile trek from New Orleans to Sloan Kettering to face Alk-positive Anaplastic Large Cell Lymphoma (ALCL)again. But this time around his black belt coach, Charles Haymon of New State Fitness, needs help making sure Steve and his family can keep the mortgage paid and food on the table while Dad undergoes a radical treatment far from home.

Springer is currently prepping for an Autologous Stem Cell Transplant. The GoFundMe page for the grappler explains just how serious a match this one is going to be:

Unlike traditional donor transplants, this process will harvest Steves own stem cells from his bone marrow. He will then be given a high dose of chemotherapy that will kill his stem cells and almost every white blood cell in his body. The harvested stem cells can then be transplanted back into his body, allowing for new blood cell production.

This is a high risk, and incredibly tough procedure for Steve and his family. Killing off his immune system will leave him at risk of a life threatening infection. A very sick Steve will lose all of his hair (again), and need to be isolated. Linda will have to be with him 24 hours a day, seven days a week, supporting him and keeping records for the transplant team. The hardest part may be that Steve will have to be separated from his 3-year-old son Tommy for the 2-3 months hes going through all this.

This process will continue into 2020. Steve could be immunocompromised for at least a year after the transplant. He will still need medications and follow up visits with his doctors in New York. Even after all this, there is no guarantee that the cancer will not come back, but this is Steves best hope.

Springer first took on cancer in 2017, completing six rounds of chemo before successfully hitting remission. But this past June a new lump was found, setting off another cascade of nightmare medical bills. For the last four months, Springer has been undergoing treatment while continuing to train, tapping out some black belts who didnt even know their opponent was fighting two battles at once.

Anyone who knows Steve knows he is one of the best of us, says Professor Haymon. Always cheerful and positive, he loves building others up. Steve is usually the last person to leave the mat, whether hes helping out teammates, or getting tips from the upper belts who gravitate towards his positive energy.

Heres the link to donate again in case you missed it above.

Also? Steve would love the chance to train with Danaher and the Death Squad at Renzo Gracies while hes up in NYCbecause who doesnt think about training when theyre in the middle of a life or death battle with rare cancerso if youre someone with gym connections, do the right thing a get this guy a spot on those famous blue mats.

See more here:
Brown Belt and Dad Needs Help Tapping Rare Cancer in Rematch - Jiu-Jitsu Times

Recommendation and review posted by Bethany Smith

The global Bone Marrow Aspirate Concentrates Market was valued around US$ 130.0 Mn in 2016 is anticipated to register a stable CAGR of over 5.0% during forecast period of 2017 to 2025, according to a new report published by Transparency Market Research (TMR) titled Bone Marrow Aspirate Concentrates Market Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 20172025.

Growth of the global bone marrow aspirate concentrates market is driven by increased prevalence of and incidences of orthopedic diseases, and sports injuries, along with high growth of the cosmetic surgery industry and increasing applications of the BMAC products in the cosmetic and orthopedic surgeries. The bone marrow aspirate concentrates market in Asia Pacific is expanding with a high potential to grow registering a CAGR above 6.0% on the backdrop of unmet clinical needs, rising geriatric population, large patient pool, favorable government regulations, development in health care sector, and increased focus on research and developmental activities.

Increase in incidences of Osteoarthritis on the backdrop of rising geriatric population to drive market growth

According to a collaborative survey conducted by the United Nations and the World Health Organization, 1.2 billion people in China are suffering from OA, of which more than 55% are aged 60 years or above. On the backdrop of such a huge patient base, there has been several developments in the field orthopedic surgery.

Bone marrow-derived stem cell treatment is considered a promising and advanced therapy. It reduces the injury healing time in orthopedic diseases to five to six weeks from four to six months in case of surgery. Reduction in the healing time is a factor likely to propel the Bone Marrow Aspirate Concentrates market during the forecast period. However, pain associated with the treatment, lack of product approval, and preference for alternative treatments are negatively affecting the market growth. Moreover, high investments in R&D and clinical trials, slow approval processes entailing sunken costs, and marginal returns on investment (RoI) for stakeholders are primary concerns faced by manufacturer further hampering growth of the market.

To garner compelling insights on the forecast analysis of Global Bone Marrow Aspirate Concentrates Market, Request a PDF Sample Here

Rise in the Number of BMAC Assisted Procedures to Boost Growth of Bone Marrow Aspirate Concentrates Accessories Segment

The product type segment is fragmented into bone marrow aspirate concentrates systems and bone marrow aspirate concentrates accessories. The bone marrow aspirate concentrates accessories segment is anticipated to carry major share of the market on the backdrop of rise in number of BMAC assisted procedures.

Cell therapies have been used extensively over the past decade for a variety of medical applications to restore cellular function and enhance quality of life. Owing to the differentiation property, stem cells are being used for repair and regeneration of bone. Moreover, increase in awareness about hygiene and risk of cross-contamination in developing countries such as Brazil, China and India are expected to increase the use of single-use Jamshidi needles for bone marrow stem cell procedures. This is likely to fuel the growth of the accessories segment in the near future.

Semi-consolidated Market with 3-4 key Players Operating in the BMAC Systems Market Segment

Key players covered in this report are Terumo Corporation (Terumo BCT), Ranfac Corp., Arthrex, Inc., Globus Medical, Inc., Cesca Therapeutics Inc., MK Alliance Inc. (TotipotentSC), and Zimmer Biomet Holdings, Inc. Companies operating in the global market for bone marrow aspirate concentrates are focusing on in-licensing and collaboration agreements to put new products in the developing markets like Asia Pacific, and Latin America. For instance, in August 2017, Cesca Therapeutics Inc. announced a distribution agreement with Boyalife WSN Ltd., a China based company. Through this agreement, Boyalife WSN Ltd. will distribute Cescas innovative biobanking and point-of-care solutions in China, India, Singapore, and the Philippines. As India and China represent two of the fastest growing economies in the world, successful penetration of these regions can generate more market opportunity to the companies.

Excerpt from:
Bone Marrow Aspirate Concentrates Market is Anticipated to Register a Stable CAGR of over 5.0% from 2017 to 2025 - Space Market Research

Recommendation and review posted by Bethany Smith

AUSTIN, Texas & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Genprex, Inc. (NASDAQ: GNPX), a clinical-stage company and leader in gene therapy using non-viral vector transfection delivery, wishes to draw attention to additional research in the field validating non-viral vector delivery as the next evolution in gene therapy.

A recently published paper by researchers in Australia, Spain and Austria supports the belief that non-viral delivery could be safer for patients than viral vectors and could speed up the production time while reducing the costs of production. The paper titled, Encapsulation, Visualization and Expression of Genes with Biomimetically Mineralized Zeolitic Imidazolate Framework-8 (ZIF-8) published in the September 4, 2019 issue of the scientific journal Small, presents data from a biomolecule-metal-organic framework (nano MOF) in zeolitic imidazolate framework-8 (ZIF-8) and found it to be a viable vehicle for intracellular transfection and gene delivery. Genprex was not involved in the study, which used a different nanotechnology.

One of the biggest differentiators between Genprex and other gene therapy companies developing technologies to treat cancer and other serious diseases is our proprietary non-viral nanoparticle delivery system, which has already been used to safely treat more than 50 patients to date, said Genprexs Chairman and Chief Executive Officer, Rodney Varner. Most gene therapy research has been focused on using viral delivery systems to deliver genes to cancer cells, and today most approved gene therapies for non-blood cell therapies use a viral vector to deliver the gene to the patient. Our proprietary non-viral delivery system enables us to potentially treat patients with a system that may be safer, with lower production costs and better scalability.

Based on the shortcomings that viral vectors have historically had, including severe adverse reactions, high production costs, difficulty in scaling and high immunogenicity responses, Genprexs founders partnered with the National Institutes of Health (NIH) during the companys inception to develop its proprietary non-viral delivery system.

Specifically, Genprexs platform technologies are designed to administer cancer fighting genes by encapsulating them into nanoscale hollow spheres called nanovesicles, which are then administered intravenously and taken up by tumor cells where they express proteins that are missing or found in low quantities. The nanovesicles are non-immunogenic, allowing repetitive therapeutic dosing. Genprexs nanovesicles are also clinically proven to effectively deliver molecular kinase inhibitors effectively.

A Phase I human clinical trial showed that Genprexs lead drug candidate, Oncoprex immunogene therapy, which is delivered through its nanovesicle non-viral delivery system, selectively and preferentially targeted primary and metastatic tumor cells, resulting in clinically significant anticancer activity. Genprexs clinical trials have also demonstrated that its delivery system is well tolerated in humans and can safely deliver high therapeutic doses.

About Genprex, Inc.

Genprex, Inc. is a clinical stage gene therapy company developing potentially life-changing technologies for cancer patients, based upon a unique proprietary technology platform, including Genprexs initial product candidate, Oncoprex immunogene therapy for non-small cell lung cancer (NSCLC). Genprexs platform technologies are designed to administer cancer fighting genes by encapsulating them into nanoscale hollow spheres called nanovesicles, which are then administered intravenously and taken up by tumor cells where they express proteins that are missing or found in low quantities. Oncoprex has a multimodal mechanism of action whereby it interrupts cell signaling pathways that cause replication and proliferation of cancer cells, re-establishes pathways for apoptosis, or programmed cell death, in cancer cells, and modulates the immune response against cancer cells. Oncoprex has also been shown to block mechanisms that create drug resistance. For more information, please visit the companys web site at http://www.genprex.com or follow Genprex on Twitter, Facebook and LinkedIn.

Forward-Looking Statements

Statements contained in this press release regarding matters that are not historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Such statements include, but are not limited to, statements regarding the effects of Oncoprex on cancer and the safety, production cost and scalability of Oncoprex and its non-viral delivery system. Risks that contribute to the uncertain nature of the forward-looking statements include the presence and level of Oncoprexs effect on cancer, the safety, cost and scalability of Oncoprex and its delivery system, as well as the timing and success of our clinical trials and planned clinical trials, Oncoprex and our other potential product candidates. These and other risks and uncertainties associated with Genprex and its lead product candidate Oncoprex are described more fully under the caption Risk Factors and elsewhere in our filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. We undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

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Genprex's Pioneering Use of Non-Viral Delivery for Gene Therapy is Gaining Industry Support - Business Wire

Recommendation and review posted by Bethany Smith

Food and Drug Administration leaders are concerned the agency won't be able to recruit and retain sufficient numbers of experts in fields that have undergone rapid scientific advances, such as cell and gene therapy or oncology.

In cell and gene therapy, the relevant FDA review unit aims to double in size over the next three to five years, competing with biopharmaceutical companies and venture firms for the same talent.

"Sometimes they're competing to actually pick people from our agency that's always fun," Peter Marks, director of the FDA's Center for Biologics Evaluation and Review, said Monday at the Biopharma Congress in Washington. "We're dealing with the issue of a very competitive job market."

Former agency head Scott Gottlieb, speaking at the same event, called recruitment and retention "an unrecognized challenge for the FDA."

In some respects, the FDA finds itself disadvantaged in enticing candidates who are in demand to fill high-paying private sector jobs. Congress acknowledged as much in the 21st Century Cures Act, a law passed in December 2016 that, among many other things, granted the regulator authority to pay some positions annual salaries of up to $400,000.

Marks said that flexibility will partially help address hiring problems, but doesn't see the issue going away, particularly given the anticipated expansion of the FDA's cell and gene therapy unit.

"We'll use that to the maximum, but it's going to continue to be a bit of a challenge," he added.

Other FDA leaders at Monday's event brought up alternative ways they hope to keep talent at the agency.

Gideon Blumenthal, the deputy director of the FDA's Oncology Center of Excellence, said they've shifted to a more academic model for the unit's roughly 100 medical oncologists.

A scientific liaison program encourages those FDA employees to take on more beyond the day-to-day review work. With it, those reviewers can become the agency's point person for a specific topic, such as breast cancer, melanoma or a research topic like health disparities, Blumenthal said.

"This is something we are really trying to push," he added. "We think it'll benefit not only the external community in understanding the FDA and oncology, but it'll help retain and attract the base talent, because not only are they reviewers, but they are really thought leaders in their fields."

In fast-moving fields, the FDA has also had difficulty filling its advisory committees with relevant, non-conflicted experts.

Janet Woodcock, a longtime FDA official and director of the Center for Drug Evaluation and Research, pointed to conflicts of interest policies as a sticking point.

"The ACs just have so many rules and lawyers ... it's really difficult to get people with the greatest expertise," she said Monday.

CBER's Marks agreed, adding that growth in rare disease drug development has made finding the right people more onerous.

"The conflict rules become so difficult that what you end up putting together is something that gets watered down ... to the point that you really can't have a discussion that's actually meaningful."

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FDA leaders worry agency will lose out on talent in gene therapy, cancer fields - BioPharma Dive

Recommendation and review posted by Bethany Smith

Scientists have developed a fast, efficient and economical method to create viral delivery vectors used in gene therapy to deliver modified versions of genes to treat disorders caused by genetic defects, including spinal muscular atrophy (SMA).

The findings were reported in the study, Production of adeno-associated virus vectors for in vitro and in vivo applications, and published in Scientific Reports.

Gene therapy is a relatively new approach that has been gaining popularity in clinical practice as a way to treat diseases caused by genetic mutations. It involves delivering a functional version of a gene to correct or replace a faulty gene within specific cells in the body.

In order to deliver the corrected version of the gene to cells, researchers use special viral vectors that work as carriers. Adeno-associated viruses, or AAVs, are one of the most used viral vectors in gene therapy due to their ability to infect and deliver the corrected gene to both dividing and non-dividing cells, without causing any harm to patients.

Gene therapy already is being used as a form of SMA treatment. The recent approval of Zolgensma (AVXS-101), a gene therapy co-developed by AveXis and Novartis, for the treatment of all types of SMA in newborns and toddlers up to the age of 2, was a historical landmark that highlighted the potential of AAV-based gene therapies to treat rare genetic disorders.

Most protocols recommend AAV purification from producer cells, grown in large cell stacks or cell culture factories to obtain sufficient AAVs for animal experiments. However, producer cells also release large quantities of AAV into the culture medium, which often remains unused, the investigators said.

In this study, researchers from the Boston University School of Medicine (BUSM) described a new protocol that allows them to maximize the quantity of AAVs that can be purified from producer cells and their culture medium in a fast, efficient and economic way.

With the new protocol, which involved several separation and purification laboratory techniques, they managed to obtain up to one milliliter of AAVs at a concentration of 10101011 viral genome copies per microliter, using five times less the number of producer cells normally used in conventional protocols.

They also showed the AAVs produced with the new protocol were viable and retained their ability to efficiently infect and deliver modified genes to cells in vitro and in vivo (outside and inside an organism, respectively).

Our protocol helps to produce AAVs efficiently and economically in regular laboratories so that researchers can easily conduct pre-clinical trialsforgene therapy, Markus Bachschmid, PhD, assistant professor of medicine at BUSM and corresponding author of the study, said in a press release.

Several labs in the Boston area and Japan have already tested this new protocol and found it useful, said Reiko Matsui, MD, assistant professor of medicine at BUSM and co-corresponding author of the study. Our hope is that many laboratories can adapt these procedures to accelerate research and promote gene therapy.

Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells cells that make up the lining of blood vessels found in the umbilical cord of newborns.

Total Posts: 85

Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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New Method Produces High Quantities of Viral Vectors for Gene Therapy - SMA News Today

Recommendation and review posted by Bethany Smith

Clustered Regularly InterSpaced Palindromic Repeats (CRISPR) is a new technology for gene editing, with promising potential in medicine and basic research. CRISPR can be found in the DNA of bacteria. There are many different types of CRISPR DNA, but they all make proteins that protect bacteria against viruses, much like the human immune system functions to kill off viruses. The way CRISPR proteins do this is to cut the DNA of viruses think of them as molecular scissors preventing the virus from growing.

CRISPR has been in the news a lot recently. Some of the news coverage has been positive (promising new therapies) and some of it has been negative (CRISPR babies).

Scientists discovered that CRISPR proteins could be adapted to cut the DNA of other organisms, including humans. This led to demonstrations that CRISPR could be used for gene editing and gene therapy. The most commonly used CRISPR protein is called Cas9.

As with all game-changing technologies, attention has focused on ethically responsible uses of the technology, and rightly so. What is often missing from many CRISPR debates is an understanding of technical limitations that could impact how and when CRISPR is used in human gene-editing applications.

Read more: Opening Pandora's Box: Gene editing and its consequences

My laboratory became interested in CRISPR because we were working on technologies for gene editing. It quickly became evident that CRISPR and Cas9 were the future of gene editing because Cas9 was significantly easier to use than other technologies but, like every new technology, was ripe for improvement.

One of the biggest issues when trying to modify human DNA is the problem of so-called off-target effects. Off-target effects arise when Cas9 cuts a piece of DNA that it wasnt programmed to do.

In describing off-target effects, I like the analogy of a programming a car GPS unit with the address Tim Hortons. In any Canadian city or town, this search will result in multiple individual Tims locations. But what is the correct location?

In much the same way, Cas9 is guided to its DNA target by a small piece of ribonucleic acid (RNA), appropriately called a guideRNA. If the address specified by the guideRNA is not unique, Cas9 will be guided to multiple locations where it will cut the DNA. The address in this case is a continuous DNA sequence in the human genome, typically 20 base pairs in length.

Why are off-target effects an issue? Not all 20 base pair stretches are unique in the genome, and Cas9 has the added problem of being able to recognize sites that are not perfect matches to the 20 base pair address (these are called off-target sites). This means that there is always the potential for Cas9 to cut DNA at off-target sites that could lead to unwanted and serious side effects, including cancer. For any human therapeutic application using gene editing, minimizing the potential for off-target effects is paramount.

Off-target effects are not a new issue in the gene-editing field, and the potential for Cas9 to cause off-target cuts was recognized as a serious issue soon after Cas9 was identified.

Significant efforts by multiple research groups resulted in the development of engineered versions of Cas9 with greater specificity and fewer off-target effects. Scientists are finding new CRISPR-related proteins with novel applications in gene therapy.

A related problem is predicting off-target sites and identifying where Cas9 cuts in the human genome. In theory, finding potential Cas9 off-target sites should be straightforward. Simply search the three billion base pairs of the human genome for matches to the 20 base pair target (this is easier than it sounds and can be done on a laptop with basic programming experience).

Some studies have found that predicted off-target sites dont always match up with experimentally identified Cas9 off-target sites, whereas other studies have found low numbers of Cas9 off-target sites. These findings underscore the need to understand how Cas9 recognizes DNA so that improvements can be made to software that predicts Cas9 sites.

For gene editing and gene therapy applications done in somatic tissues, such as blood cells, DNA changes are not heritable. In these cases, a higher amount of off-target effects may be tolerated. Any off-target effects are unacceptable simply because they are difficult to detect and reverse. Until these issues are resolved, the question of off-target effects should not be ignored in debates about CRISPR gene editing.

[ Youre smart and curious about the world. So are The Conversations authors and editors. You can read us daily by subscribing to our newsletter. ]

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Gene editing needs to become more precise to live up to its promise - The Conversation CA

Recommendation and review posted by Bethany Smith

By Curtis Sprung, freelance reporter

This article was written for our sponsor, the Sanford Area Growth Alliance.

It's not every day a major corporation decides to invest half a billion dollars in your community, but that's what happened for the town of Sanford in August.

Pharmaceutical giant Pfizer announced plans for a state-of-the-art gene therapy manufacturing facility in Sanford as part of the company's ongoing investment in the area. Pfizer already has approximately 650 employees in Sanford, and the new facility is expected to add around 300 new jobs to the area.

"This investment will further strengthen Pfizer's leadership in gene therapy manufacturing technology," said Mike McDermott, president of Pfizer Global Supply, in a press release. "The expansion of the Sanford site is expected to create hundreds of highly skilled jobs which would increase Sanford's high-tech manufacturing environment and is part of our overall plan to invest approximately $5 billion in U.S.-based capital projects over the next several years."

The investment is just the next step in a long history of life science successes in the area. That history has primed Sanford to be a major player in the life sciences field for years to come.

"It speaks to the future of manufacturing realm here in Sanford," said Sanford Area Growth Alliance Existing Industry Development Manager Jimmy Randolph. "It's sure to attract other folks active in that space because of the significant life sciences activity in the Research Triangle area and Sanford is so proximate to that facility. It's a wonderful affirmation of the activity we've been engaged in."

Pfizer has locations across the globe, but ultimately, Sanford was able to provide a proven workforce, dedicated acreage, and a community college educating potential future employees in life sciences and manufacturing to close the deal.

Such a heavy investment means positive changes for the community and other companies that call Sanford home.

"Beyond the obvious things, I think what this will do is be a catalyst for further growth," Randolph said. "If you look at Sanford on the map, I think you can expect to see the rate of population growth and additional investment from an industrial manufacturing standpoint. Folks are already contacting us about additional space near the campus."

The continued investment in Sanford has resulted in quality of life improvements for residents ranging from new charter schools to the recently opened splash pad to the renovated downtown. All of this stems from a plan put in place nearly a decade ago to revitalize Sanford and continue to attract modern businesses and create a kind of economic development snowball effect, with improvements across many community aspects.

The Pfizer investment marks a major milestone for Sanford, but local leadership and SAGA board members are confident the growth will continue at a manageable pace. With their strong history of manufacturing, Sanford and Lee County remains open for business with an engaged community that's excited to see what happens next.

"The reality is, [we] are fairly far sighted," Randolph said. "The community around Sanford has a very favorable attitude toward manufacturing. I can't imagine a better place to live or work. The businesses that make it that way it really is a testament to the character of the citizens of this community."

This article was written for our sponsor, the Sanford Area Growth Alliance.

Continued here:
Pfizer to build a $500M gene-therapy facility in Sanford - WRAL.com

Recommendation and review posted by Bethany Smith

ST. PAUL, Minn., Oct. 7, 2019 /PRNewswire/ -- Recombinetics today announced a collaborative research project with Mayo Clinic to advance Osteogenesis Imperfecta (OI) research by developing the first-ever swine models of OI. These models will help preclinical researchers better understand disease etiology and progression and provide a reliable preclinical model for establishing the safety and efficacy of new therapies that patients with OI desperately need.

Also known as "brittle bone disease", clinical manifestations of OI vary from a mild increase in fractures to severe bone deformities, hearing loss and death in the neonatal period. To date, no treatment corrects the underlying cause or alleviates the complications of OI. Currently, research is hampered by mouse models that do not fully recapitulate the disease seen in patients and have been poor predictors of clinical efficacy. Providing the research community with gene-edited swine models that more closely mimic the human disease, could lead to better treatments, including in vivo gene-editing and gene therapy, orthopedic devices for intramedullary rodding, noninvasive techniques for malocclusion, and understanding the natural progression of bone mineralization after currently available therapeutics.

Project efforts will be led by Adrienne Watson, Ph.D., Recombinetics' Vice President of Research & Development and Mayo Clinic's David Deyle, MD, partially funded by an SBIR grant from the National Institutes of Health.

About RecombineticsFounded in 2008, Recombinetics (RCI) is producing gene-edited animals for biomedical and food production purposes and is generating commercial and collaborative revenues. RCI's technology platform supports three business lines: Acceligen (precision breeding to enhance health, well-being and productivity in food animals and aquaculture); Surrogen (gene-edited swine models of human diseases for biomedical research and pre-clinical trials by pharmaceutical and medical device companies); and Regenevida (development of human regenerative products including cells, tissues and organ products in swine models for exotransplantation to humans). Learn more at Recombinetics.com.

Contact:Kris Huson(651) 757-0427224755@email4pr.com

View original content to download multimedia:http://www.prnewswire.com/news-releases/recombinetics-announces-collaboration-with-mayo-clinic-to-develop-the-first-ever-swine-models-of-osteogenesis-imperfecta-300931565.html

SOURCE Recombinetics

Excerpt from:
Recombinetics Announces Collaboration with Mayo Clinic to Develop the First-Ever Swine Models of Osteogenesis Imperfecta - BioSpace

Recommendation and review posted by Bethany Smith

-- Improvements on functional measures seen in all three participants --

-- Significant reduction in creatine kinase maintained over nine months --

-- Results follow positive and robust expression and biomarker data presented earlier in 2019 --

CAMBRIDGE, Mass., Oct. 04, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (SRPT), the leader in precision genetic medicine for rare diseases, today announced the nine-month functional results from three Limb-girdle muscular dystrophy Type 2E (LGMD2E) clinical trial participants who received SRP-9003. SRP-9003 is an investigational gene therapy intended to transduce skeletal and cardiac muscle with a gene that codes for the full-length, native beta-sarcoglycan protein, the lack of which is the sole cause of LGMD2E.

In Cohort 1 of the SRP-9003 study, three participants ages 4-13 were treated with an infusion of SRP-9003 at a dose of 5x1013vg/kg. Improvements in functional outcomes were observed at day 270 (nine months) for all three participants.

We have now observed consistent functional improvements, in addition to high levels of expression of the missing protein of interest and strong results in related biomarkers, in both of our first cohorts for Duchenne muscular dystrophy (SRP-9001) and LGMD2E (SRP-9003). We intend to test one higher dose of SRP-9003 in LGMD2E participants, select our clinical dose and then advance our SRP-9003 program, along with our other five LGMD programs, as rapidly as possible, said Doug Ingram, Sareptas president and chief executive officer. With the results of our first LGMD2E cohort, Sarepta continues to build its gene therapy engine, an enduring model created to design, develop and bring to the medical and patient community transformative therapies for those living with, and too often dying from, rare genetic disease.

At Day 270, mean creatine kinase (CK) was significantly reduced compared to baseline. CK is an enzyme biomarker strongly associated with muscle damage.

At Day 270, all three participants showed improvements from baseline across all functional measures, including the North Star Assessment for Dysferlinopathy (NSAD), time to rise, four-stair climb, 100-m walk test and 10-meter walk test. These results are distinctly different from what an age-matched, natural history group would predict.

No new safety signals were observed and the safety profile seen to date supports the ability to dose escalate in the next cohort of the study. As previously disclosed, two participants in the study had elevated liver enzymes, one of which was designated a serious adverse event (SAE), as the participant had associated transient increase in bilirubin. Both events occurred when the participants were tapered off oral steroids and, in both instances, elevated liver enzymes returned to baseline and symptoms resolved following supplemental steroid treatment.

LGMD2E is a devastating neuromuscular disease with no current treatment options so we are very pleased to observe a functional improvement in study participants who received SRP-9003, said Jerry Mendell, M.D., principal investigator at the Center for Gene Therapy in the Abigail Wexner Research Institute at Nationwide Childrens Hospital and lead investigator for the study.

Sarepta had previously shared expression results from the study, which found that in two-month post-treatment muscle biopsies, clinical trial participants showed a mean of 51% beta-sarcoglycan positive fibers, as measured by immunohistochemistry (IHC), substantially exceeding the pre-defined 20% measure for success. Mean fiber intensity, as measured by IHC, was 47% compared to normal control.

About SRP-9003 and the Phase I/IIa Gene Transfer Clinical Trial

SRP-9003 uses the AAVrh74 vector, which is designed to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle without promiscuously crossing the blood brain barrier, making it an ideal candidate to treat peripheral neuromuscular diseases. As a rhesus monkey-derived AAV vector, AAVrh74 has lower immunogenicity rates than reported with other common human AAV vectors. The MHCK7 promoter has been chosen for its ability to robustly express in the heart, which is critically important for patients with LGMD2E, many of whom die from pulmonary or cardiac complications.

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This first-in-human study is evaluating a single intravenous infusion of SRP-9003 among children with LGMD2E between the ages of four and 15 years with significant symptoms of disease.

About Limb-Girdle Muscular Dystrophy

Limb girdle muscular dystrophies are genetic diseases that cause progressive, debilitating weakness and wasting that begin in muscles around the hips and shoulders before progressing to muscles in the arms and legs.

Patients with LGMD2E begin showing neuromuscular symptoms such as difficulty running, jumping and climbing stairs before age 10. The disease, which is an autosomal recessive subtype of LGMD, progresses to loss of ambulation in the teen years and often leads to death before age 30. There is currently no treatment or cure for LGMD2E.

Sarepta has five LGMD gene therapy programs in development, including subtypes for LGMD2E, LGMD2D, LGMD2C, LGMD2B and LGMD2L, and holds an option for a sixth program for LGMD2A.

About Sarepta Therapeutics

Sarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for Limb-girdle muscular dystrophy diseases (LGMD), MPS IIIA, Pompe and other CNS-related disorders, totaling over 20 therapies in various stages of development. The Companys programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. Sarepta is fueled by an audacious but important mission: to profoundly improve and extend the lives of patients with rare genetic-based diseases. For more information, please visit http://www.sarepta.com.

Forward-Looking Statements

This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding our intention to test one higher dose of SRP-9003 in LGMD2E participants, select our clinical dose and then advance our SRP-9003 program, along with our other five LGMD programs, as rapidly as possible; Sarepta continuing to build an enduring gene therapy model created to design, develop and bring to the medical and patient community transformative therapies for those living with rare genetic disease; the safety profile of SRP-9003 seen to date supporting the ability to dose escalate in the next cohort of the study; SRP-9003 being an ideal candidate to treat peripheral neuromuscular diseases; the potential benefits of the AAVrh74 vector and the MHCK7 promoter; and our mission to profoundly improve and extend the lives of patients with rare genetic-based diseases.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sareptas control. Known risk factors include, among others: success in preclinical testing and early clinical trials, especially if based on a small patient sample, does not ensure that later clinical trials will be successful, and initial results from a clinical trial do not necessarily predict final results; the data presented in this release may not be consistent with the final data set and analysis thereof or result in a safe or effective treatment benefit; different methodologies, assumptions and applications Sarepta utilizes to assess particular safety or efficacy parameters may yield different statistical results, and even if Sarepta believes the data collected from clinical trials of its product candidates are positive, these data may not be sufficient to support approval by the FDA or foreign regulatory authorities; Sareptas ongoing research and development efforts may not result in any viable treatments suitable for clinical research or commercialization due to a variety of reasons, some of which may be outside of Sareptas control, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product candidates; and even if Sareptas programs result in new commercialized products, Sarepta may not achieve any significant revenues from the sale of such products; and those risks identified under the heading Risk Factors in Sareptas most recent Annual Report on Form 10-K for the year ended December 31, 2018, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

Any of the foregoing risks could materially and adversely affect the Companys business, results of operations and the trading price of Sareptas common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Internet Posting of Information

We routinely post information that may be important to investors in the 'For Investors' section of our website at http://www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Sarepta Therapeutics, Inc.

Investors:Ian Estepan, 617-274-4052iestepan@sarepta.com

Media:Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

Excerpt from:
Sarepta Therapeutics Announces Positive Functional Results from the SRP-9003 (MYO-101) Gene Therapy Trial to Treat Limb-Girdle Muscular Dystrophy Type...

Recommendation and review posted by Bethany Smith

Vancouver, British Columbia--(Newsfile Corp. - October 7, 2019) - PreveCeutical Medical Inc.(CSE: PREV) (OTCQB: PRVCF) (FSE: 18H)(the "Company" or "PreveCeutical"), is pleased to announce that it has received a cash refund of $616,802 AUD from the Australian Taxation Office under the Research and Development ("R&D") Tax Incentive Program. The cash refund is related to expenditures on eligible R&D activities conducted in Australia during the Company's 2018 financial year. The R&D activities included work done in areas across PreveCeutical's portfolio, including the Sol-gel nose-to-brain drug delivery system and the non-addictive analgesics programs, which are being conducted at the University of Queensland, Australia.

The refund received by the Company will support and reinforce the Company's continued investments in its R&D programs.

The R&D Tax Incentive Program encourages companies to engage in R&D programs, including ones that have the potential to improve global health outcomes, which boosts competitiveness and generates economic benefits locally.

PreveCeutical's President and Chief Science Officer, Dr. Mak Jawadekar stated, "We are highly supportive of the Australian Government's R&D Tax Credit incentive which recognises the critical role of R&D involved in potentially developing life-saving drugs, therapies and delivery devices. This refund would help enable PreveCeutical to further advance our proprietary therapeutic alternatives for preventive and curative therapies for some of the unmet medical needs."

About PreveCeutical

PreveCeutical is a health sciences company that develops innovative options for preventive and curative therapies utilizing organic and nature identical products.

PreveCeutical aims to be a leader in preventive health sciences and currently has five research and development programs, including: dual gene therapy for curative and prevention therapies for type 2 diabetes and obesity; a soluble gel drug delivery program; Nature Identical peptides for treatment of various ailments; non-addictive analgesic peptides as a replacement to the highly addictive analgesics such as morphine, fentanyl and oxycodone; and a therapeutic product for treating athletes who suffer from concussions (mild traumatic brain injury).

For more information about PreveCeutical, please visit http://www.PreveCeutical.com, follow us on Twitter: http://twitter.com/PreveCeuticals and Facebook: http://www.facebook.com/PreveCeutical.

On Behalf of the Board of Directors

"Dr. Makarand (Mak) Jawadekar"President & Chief Science Officer

For further information, please contact:

Deanna KressDirector of Corporate Communications & Investor Relations+1-778-999-6063deanna@PreveCeutical.com

Forward-Looking Statements:

This news release contains forward-looking statements and forward-looking information (collectively, "forward-looking statements") within the meaning of applicable Canadian and U.S. securities legislation, including the United States Private Securities Litigation Reform Act of 1995. All statements in this news release that are not purely historical are forward-looking statements and include any statements regarding beliefs, plans, expectations and orientations regarding the future including the Company's anticipated business plans, the intended use of the R&D Tax Incentive Program cash refund, and the prospect of its ability and success in executing its proposed plans. Often, but not always, forward-looking statements can be identified by words such as "pro forma", "plans", "expects", "may", "should", "budget", "schedules", "estimates", "forecasts", "intends", "anticipates", "believes", "potential", "will" or variations of such words including negative variations thereof and phrases that refer to certain actions, events or results that may, could, would, might or will occur or be taken or achieved. Forward looking statements are based on certain assumptions regarding the Company, including expected growth, results of operations, including the Company's research and development activities, performance, industry trends, growth opportunities, and that the Company will be able to obtain the financing required to carry out its planned future activities, retain and attract qualified research personnel and obtain and/or maintain the necessary intellectual property rights it needs to carry out its future business activities. Actual results could differ from those projected in any forward-looking statements due to numerous factors including risks and uncertainties relating to the inability of the Company, to, among other things, obtain any required governmental, regulatory or stock exchange approvals, permits, consents or authorizations required, including Canadian Securities Exchange acceptance of any planned future activities, commercialise therapeutic and diagnostic technologies, pursue business partnerships, complete its research programs as planned, and obtain the financing required to carry out its planned future activities. Other factors such as general economic, market or business conditions or changes in laws, regulations and policies affecting the biotechnology or pharmaceutical industry, may also adversely affect the future results or performance of the Company. These forward-looking statements are made as of the date of this news release and, unless required by applicable law, the Company assumes no obligation to update the forward-looking statements or to update the reasons why actual results could differ from those projected in these forward-looking statements. Although the Company believes that the statements, beliefs, plans, expectations, and intentions contained in this news release are reasonable, there can be no assurance that those statements, beliefs, plans, expectations, or intentions will prove to be accurate. Readers should consider all of the information set forth herein and should also refer to other periodic reports provided by the Company from time-to-time. These reports and the Company's filings are available at http://www.sedar.com.

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Readers are cautioned that forward-looking statements are not guarantees of future performance or events and, accordingly, are cautioned not to put undue reliance on forward-looking statements due to the inherent uncertainty of such statements.

To view the source version of this press release, please visit https://www.newsfilecorp.com/release/48515

See the article here:
PreveCeutical Receives $616,802 AUD Research and Development Tax Incentive Cash Refund - Yahoo Finance

Recommendation and review posted by Bethany Smith

For the past 15 years the University of Oxfords Simon Davis has been mapping the surface of T cells, exploring and examining the structures of surface proteins while determining what it takes to manage specific immune cell signaling. And now a group of UK investors says its ready to advance that research toward the clinic, inside a new biotech vehicle theyre setting up to take the tech into the commercial sphere.

Enter MiroBio, stage left. The newly crafted company has raised $34 million to launch their antibodies into the clinic. Oxford Sciences Innovation and Samsara Biocapital co-led the round, joined by Advent Life Sciences and SR One.

The plan is to use Davis insights and preclinical antibodies to hijack the natural mechanisms used to control immune cells for the purpose of targeting relevant diseases. And while the research has had obvious applicability in oncology where cell therapy evangelists are looking to develop the next wave of more sophisticated therapies MiroBio is starting out in autoimmune diseases, where errant attacks on healthy tissue trigger some major market ailments.

If you look at things from an investors perspective against a backdrop of intense research activity in oncology, says Executive Chairman Eliot Charles, the real opportunity near term was autoimmune disease.

Not that they arent interested in oncology.

For now, though, the emphasis is using the expertise at the venture groups to guide the company while they build out their team in the UK and follow up on the in vivo models that Davis worked with. And the first goal is resetting the immune system when it goes awry, as happens in autoimmune diseases.

Charles an Amgen vet whos now a venture partner at SR One, jumped on the phone with me early Monday to discuss the work with VP Operations Tim Funnell. Samsaras Bob Stein has stepped in as interim CSO as they follow up on 4 programs in-licensed from Davis lab, building out the initial team of 6 staffers to 15 or so.

The investor group has worked together on various projects through the years, says Charles, which is how the transatlantic syndicate came together to back Davis work in a startup.

On his website, Davis notes:

Our present goals are (1) to show that the kinetic segregation model does indeed explain T-cell receptor triggering, and (2) to use the idea to develop new types of therapeutic antibodies. The signaling concept is being tested using structural approaches and super-resolution imaging techniques, such as dSTORM. For this, the behavior of T-cell surface proteins is being studied at contacts with glass surfaces and supported lipid bilayers, in collaboration with Professor Klenerman. New, potentially therapeutic superagonistic antibodies are being developed and licensed to industry in collaboration with Professor Richard Cornall.

Its early days, of course, but that process has inspired a spinout with big plans.And they have enough cash to get to human studies on the lead, while building up a pipeline behind it.

See the article here:
Antibody research graduates from a top Oxford lab into the biotech world with $34M to fund R&D work - Endpoints News

Recommendation and review posted by Bethany Smith

Seven months after Oyster Point Pharma bridged its way into a Phase III study with $93 million in venture cash, the biotech is stepping back up to see if public investors are in a mood to back their play to jump into a big league market with a minor league team.

The Princeton, NJ-based biotech teed up an IPO on Friday, outlining their case on going after dry-eye disease a blockbuster market that accounts for tens of millions of patients. On the drug side, thats a market that has been dominated by Allergans cash cow Restasis now being carved up by generics as AbbVie buys out the company and Xiidra, a therapy which Takeda sold off to Novartis in a $5.3 billion deal in the wake of the Shire acquisition. Most patients get eye drops for the condition.

Tiny Oyster Points plan involves taking a mostly positive Phase IIb study where their drug, varenicline, a formulation of Chantix, managed to get the tear ducts to flow in a statistically significant manner and proving it works in a second registrational, confirmatory story. The FDA, according to the S-1, has signed off on using the Phase IIb as their first or 2 required pivotals. And that study, they maintain, is the first ever to show statistical significance against signs and symptoms of dry eye disease.

If they do get an approval, they believe that they can hire a sales force of 150 to 200 to get it out on the market. The biotech currently has a staff of 18, putting it in the leanest and meanest category of late-stage companies.

Whether they mean that or plan to use it as a classic bluff in preparation for a possible buyout deal is the big question. Startups traditionally have faced a mountainous challenge selling the drugs they manage to get approved, and thats even true in specialty cancer markets. For major markets like dry eye disease, the climb against Big Pharma and generics is distinctly more challenging.

The biotech has burned through $53 million and had $84 million banked at the end of H1.

New Enterprise Associates and Versant are the 2 big investors, with 32% of equity each. Jeffrey Nau, the CEO, has 2.1% of the shares going in.

Read the original:
Ophthalmology biotech upstart is asking investors to bet $85M-plus on their late-stage rival to Restasis and Xiidra - Endpoints News

Recommendation and review posted by Bethany Smith

Liver disease patients could one day benefit from a new cell therapy that has just completed its first clinical trial.

Researchers who tested the potential treatment in patients with liver cirrhosis where long term damage produces scarring found the therapy had no significant adverse effects.

Now the team, based at the Universitys MRC Centre for Regenerative Medicine, is to gauge the effectiveness of the treatment which is based on white blood cells called macrophages, that are key to normal liver repair.

The next stage of the trial will measure whether the therapy helps the liver to reduce scarring and stimulate regeneration. The results should be known within the next two years.

At present the only successful treatment for end-stage liver cirrhosis which claims around 14,000 lives in the UK each year (British Liver Trust) is an organ transplant. The safety trial is a vital step forward in finding an alternative therapy.

During the trial scientists took cells from the blood of nine patients with the disease and turned them into macrophages, in the Scottish National Blood Transfusion Services (SNBTS) cell therapy facility.

The new cells were then re-injected into the patient with the hope of repairing the damaged organ from within.

Causes of liver cirrhosis include infections such as hepatitis C, obesity, alcohol excess and some genetic and immune conditions.

Liver cirrhosis is a major healthcare issue in the UK and is one of the top five killers. The results from this first safety trial are encouraging and we can now progress to testing how effective it is in a larger group of people. If this was found to be effective it would offer a new way to tackle this important condition.

The research which was published in the journal Nature Medicine, received funding from the Medical Research Council and was conducted in partnership with the SNBTS and the Cell and Gene Therapy Catapult.

Excerpt from:
Cell therapy safe for liver patients, trial shows - Mirage News

Recommendation and review posted by Bethany Smith

Sareptas lousy summer has come to a close with some encouraging fresh functional data from its limb-girdle muscular dystrophy (LGMD) gene-therapy program.

In February, the drugmaker divulged data from three patients in the first cohort of an open-label Phase I/II study testing the use of an experimental gene therapy MYO-101 (now called SRP-9003) in LGMD patients aged four to 15 years.

Data showed the therapy rejuvenated the production, by an average of 51%, of beta-sarcoglycan the protein required for muscle function that is missing in this patient population. On Friday, Sarepta broke out data that showed that the induced uptick in beta-sarcoglycan did, in fact, translate to functional improvements, nine months post-infusion.

Based on the natural history of the disease, these changes were definitely not expected at these time points, Sarepta executives underscored on a conference call with analysts.

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Read the original post:
We'll see this week if the public markets' love affair with biotech unicorns is still running hot or not - Endpoints News

Recommendation and review posted by Bethany Smith

In this report, the Global Gene Therapy for Inherited Genetic Disorders market is valued at USD XX million in 2017 and is expected to reach USD XX million by the end of 2025, growing at a CAGR of XX% between 2017 and 2025. Global Gene Therapy for Inherited Genetic Disorders market has been broken down by major regions, with complete market estimates on the basis of products/applications on a regional basis.

Browse full research report at https://www.crystalmarketreport.com/global-gene-therapy-for-inherited-genetic-disorders-market-report-history-and-forecast-2014-2025-breakdown-data-by-companies-key-regions-types-and-application

Summary

In the medicine field gene therapy (also called human gene transfer) is the therapeutic delivery of nucleic acid into a patients cells as a drug to treat disease.A genetic disorder is a genetic problem caused by one or more abnormalities formed in the genome.

Gene therapy has the potential to cure inherited diseases that cannot be cured using conventional drugs. It is being increasingly adopted in clinical trials because of its high efficacy and safety towards the treatment of inherited genetic disorders.

In 2018, the global Gene Therapy for Inherited Genetic Disorders market size was xx million US$ and it is expected to reach xx million US$ by the end of 2025, with a CAGR of xx% between 2019 and 2025.

This report studies the Gene Therapy for Inherited Genetic Disorders market size by players, regions, product types and end industries, history data 2014-2018 and forecast data 2019-2025; This report also studies the global market competition landscape, market drivers and trends, opportunities and challenges, risks and entry barriers, sales channels, distributors and Porters Five Forces Analysis.

This report focuses on the global top players, covered

BioMarin Pharmaceutical Inc.

bluebird bio Inc.

Novartis AG

Orchard Therapeutics Plc

Spark Therapeutics Inc.

Market segment by Regions/Countries, this report covers

North America

Europe

China

Rest of Asia Pacific

Central & South America

Middle East & Africa

Market segment by Type, the product can be split into

Eye Disorders

Hematological Disorders

Central Nervous System Disorders

Muscular Disorders

Others

Market segment by Application, the market can be split into

Hospital

Clinic

Research Institute

Others

The study objectives of this report are:

To study and forecast the market size of Gene Therapy for Inherited Genetic Disorders in global market.

To analyze the global key players, SWOT analysis, value and global market share for top players.

To define, describe and forecast the market by type, end use and region.

To analyze and compare the market status and forecast among global major regions.

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

To identify significant trends and factors driving or inhibiting the market growth.

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

To strategically analyze each submarket with respect to individual growth trend and their contribution to the market

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

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

In this study, the years considered to estimate the market size of Gene Therapy for Inherited Genetic Disorders are as follows:

History Year: 2014-2018

Base Year: 2018

Estimated Year: 2019

Forecast Year 2019 to 2025

For the data information by region, company, type and application, 2018 is considered as the base year. Whenever data information was unavailable for the base year, the prior year has been considered.

Key Stakeholders

Raw material suppliers

Distributors/traders/wholesalers/suppliers

Regulatory bodies, including government agencies and NGO

Commercial research & development (R&D) institutions

Importers and exporters

Government organizations, research organizations, and consulting firms

Trade associations and industry bodies

End-use industries

Available Customizations

With the given market data, QYResearch offers customizations according to the companys specific needs. The following customization options are available for the report:

Further breakdown of Gene Therapy for Inherited Genetic Disorders market on basis of the key contributing countries.

Detailed analysis and profiling of additional market players.

Browse full research report at https://www.crystalmarketreport.com/global-gene-therapy-for-inherited-genetic-disorders-market-report-history-and-forecast-2014-2025-breakdown-data-by-companies-key-regions-types-and-application

Reasons to Buy This Research Report

About Crystal Market Reports

Crystal Market Reports is a distributor of market research spanning 160 industries. Our extensive database consists of over 400,000 quality publications sourced from 400 plus publishers, this puts our research specialists in the unique position of been able to offer truly unbiased advice on what research provides the most valuable insights.

Contact Info.:-

Address: 90 State StreetSuite 700 AlbanyNew York 12207Email: [emailprotected]Web: https://www.crystalmarketreport.com

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Global Gene Therapy for Inherited Genetic Disorders Market Report, History and Forecast 2014-2025, Breakdown Data by Companies, Key Regions, Types and...

Recommendation and review posted by Bethany Smith

ALBANY An important question to consider during this special months is What is breast cancer? Breast cancer occurs when normal cells in the breast mutate and grow uncontrollably.

Breast cancer is the most ubiquitous form of cancer in women worldwide. In the United States, an estimated 240,000 women will be diagnosed with breast cancer annually. Breast cancer continues to rank second, after lung cancer, as a cause of cancer death in women in the United States, and it is a leading cause of premature mortality for women. In 2012, deaths from breast cancer accounted for 783 000 years of potential life lost and an average of 19 years of life lost per death.

Screening describes the process of looking for signs of a particular disease, such as breast cancer, before a person has noticeable symptoms. The goal of screening tests is to detect cancer at an early stage when it can be treated and may be cured. Researchers hope to better understand those who are predisposed to a particular type of cancer. For example, they look at the persons age, their family history, and certain exposures during their lifetime, which helps the physician recommend who should be screened for cancer, which screening tests should be used, and how often the tests should be done.

Early detection has been shown to be associated with reduced breast cancer morbidity and mortality. Breast cancer screening detects the breasts for early signs of cancer in patients without any symptoms.

Mammography is the most common screening test for breast cancer.

Magnetic resonance imaging (MRI) also may be used to screen women who have a high risk of breast cancer.

Other screening tests have been or are being studied in clinical trials, including:

Screening tests for breast cancer are being studied in clinical trials.

Who should be screened for breast cancer? Opinions regarding optimal breast cancer screening age can differ. Its important to talk to ones doctor or nurse about the benefits and drawbacks of screening.

The American Cancer society commissioned a systematic evidence review of the breast cancer screening literature:

They recommend that women with an average risk of breast cancer should undergo regular screening mammography starting at age 45 years.

Women aged 45 to 54 years should be screened annually.

Women 55 years and older should transition to biennial screening or have the opportunity to continue screening annually.

Women should have the opportunity to begin annual screening between the ages of 40 and 44 years. Women should continue screening mammography as long as their overall health is good and they have a life expectancy of 10 years or longer.

The primary benefits of screening are to find cancer early and lower the chances of dying of breast cancer. Drawbacks include:

False-positive test results can occur.

False-positive results can lead to extra testing and cause anxiety.

False-negative test results can delay diagnosis and treatment.

Finding breast cancer may lead to breast cancer treatment and side effects, but it may not improve a womans health or help her live longer.

Mammography exposes the breast to low doses of radiation.

Pain or discomfort during a mammogram.

If these tests show any suspicious findings, a more detailed test is ordered. A biopsy is undertaken and sent for tissue diagnosis.

Treatment options for breast cancer patients include:

Mastectomy is surgery to remove the whole breast.

Lumpectomy is surgery to remove the cancer and a section of healthy tissue around it.

Radiation therapy that kills cancer cells.

Chemotherapy The medical term for medicines that kill cancer cells or stop them from growing.

Hormone therapy Some forms of breast cancer grow in response to hormones.

Targeted therapy Some medicines work only on cancers that have certain characteristics.

There are medications to help prevent breast cancer for women who are at high risk. Women with a strong family history of breast cancer should ask their doctor what they can do to prevent cancer. Genetic testing also assists in identifying genes that can cause breast cancer. Once an abnormal gene is identified, surgical and hormonal interventions can reduce or prevent breast cancer.

Cancers that can be screened:

Breast cancer The main test used to screen for breast cancer is mammogram.

Colon cancer There are multiple screening tests for colon cancer.

Cervical cancer Tests used to screen for cervical cancer is the Pap smear.

Prostate cancer Test used to screen for prostate cancer is a PSA test.

Lung cancer Test used to screen for lung cancer is a low-dose CT scan.

Ovarian cancer To screen for ovarian cancer, a blood test called CA 125, and an ultrasound, or both, are used.

Yash Jani, a senior at Deerfield-Windsor School, plans to study medicine upon high school graduation.

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The facts about breast cancer awareness | News - The Albany Herald

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Newswise Washington, D.C., October 1, 2019 MedStar Heart & Vascular Institute has enrolled its first patient to a clinical trial to determine whether cardiac stem cells reduce inflammation enough to improve heart function in patients with heart failure severe enough to require a left ventricular assist device, or LVAD. STEMVAD is a randomized, double-blinded, placebo-controlled study that will assess the effects of multiple intravenous administration of CardioCells proprietary mesenchymal stem cells (MSCs). It is expected to enroll 30 patients.

The STEMVAD trial is the next step in MedStar Heart & Vascular Institutes earlier research that discovered one of the major problems in heart failure is persistent inflammation," said Stephen Epstein, MD, director of Translational and Vascular Biology Research at MedStar Heart & Vascular Institute. "And these mesenchymal stem cells control inflammation, leading to improved heart function.

Approximately six and a half million adult Americans have heart failure, of whom 200,000 to 250,000 are estimated to have end-stage heart failure and need a heart transplant. However, with the very low supply of donor hearts, LVADs are increasingly used. An LVAD is a small pump that helps circulate the patients blood when their heart becomes too weak to pump effectively on its own. Although highly effective in alleviating symptoms and improving longevity, patients with LVAD support have a high incidence of serious complications.

Innovative therapies to improve heart function and outcomes of patients with advanced heart failure are sorely needed, added Selma Mohammed, MD, PhD, research director of the Advanced Heart Failure Research Program at MedStar Heart & Vascular Institute.

If we are successful in showing intravenously delivered stem cells improve outcomes in patients, the results would likely extend to the general population of heart failure patients, and in the process, fundamentally transform current paradigms for treating heart failure, concluded Ron Waksman, MD, director of Cardiovascular Research and Advanced Education at MedStar Heart & Vascular Institute. For more information on whether patients may qualify for the trial, call Michelle Deville, research coordinator, at 202-877-2713 or email michelle.deville@medstar.net.

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Conflict of Interest Statement: Dr. Stephen Epstein is an equity holder in CardioCell, serves on its Board, and consults for the company.

About MedStar Heart & Vascular Institute:MedStar Heart & Vascular Institute is a national leader in the research, diagnosis and treatment of cardiovascular disease. A network of 10 hospitals and 170 cardiovascular physicians throughout Maryland, Northern Virginia and the Greater Washington, D.C., region, MedStar Heart & Vascular Institute also offers a clinical and research alliance with Cleveland Clinic Heart & Vascular Institute, the nations No. 1 heart program. Together, they have forged a relationship of shared expertise to enhance quality, improve safety and increase access to advanced services. MedStar Heart & Vascular Institute was founded at MedStar Washington Hospital Center, home to the Nancy and Harold Zirkin Heart & Vascular Hospital. Opened in July 2016, the hospital ushered in a new era of coordinated, centralized specialty care for patients with even the most complex heart and vascular diagnoses.

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First Patient Enrolled in Novel Stem Cell Trial for Heart Failure Treatment - Newswise

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