SALEM Technologies such as sexed semen and genetic testing were once an expensive niche for elite cattle, but they are quickly becoming a necessary tool for success, according to a new report by Rabobank.

Rapid advances in breeding technologies and genetics are changing the way dairy producers run their businesses and design their herds, Ben Laine, dairy analyst and author of the report, said.

Sexed semen has become commonplace in the dairy industry, but dairy producers are also using genetics to design their dairy herds, he said.

Genomic testing in dairy began in 2009 on elite animals, costing about $200 per test. But the cost has fallen to a range of $30 to $50. Blood or hair follicle samples are taken shortly after birth, and lab results are available in a matter of days.

Producers using genetic testing are able to determine a heifers genetic potential early in life, before it enters the milking herd, he said.

Replacement costs are the third-highest cost on a dairy, and genetic testing is shifting the mindset from a tendency to keep every heifer, he said.

Instead, with the additional data, producers can focus more on maintaining the optimal size of their milking herd and maintaining only the best animals in that herd, he said.

Dairy producers are also pairing genetic testing with sexed semen, which increases the likelihood of a cows offspring being female to over 90%.

That allows the genetics of the most favorable cows to be maintained in the milking herd, he said.

Heifers will generally receive sexed semen for their first pregnancy due to their naturally high conception rates. Beef semen is then bred into the portion of the herd with lower genetic potential.

Breeding all first-pregnancy animals with sexed semen could depend on the replacement/milk cow ratio on the farm and herd growth aspirations. But that could start to change among farms that have already made progress on their genetics and arent looking to expand, he said.

Embryo transplant is also used to maintain favorable genetics in the milking herd. Newborn heifer calves have about 150,000 ova, but a dairy cow normally only has two to four calves over the course of its life.

By harvesting and fertilizing the ova and transplanting the embryos, the genetics from a single cow can be passed to many more offspring. In addition, embryos can be transferred to either dairy or beef cows to facilitate herd expansions with specific traits, he said.

As the cost of these technologies decreases and word spreads about their effectiveness, more producers will begin using them and it will become critical to long-term success, he said.

Producers who are not adopting these technologies will increasingly fall behind and see their efficiency lag compared to their peers, he said.

While the early focus of genomics has been on traits to optimize milk and component production, the next frontier will likely focus on traits, such as feed conversion, heat tolerance, disease resistance or physical traits that would work better in the context of robotic milking, he said.

Here is the original post:
Dairy industry moving toward 'designer' cows | Business - East Oregonian

Recommendation and review posted by Bethany Smith

More and more, we see the advertisements on television and social media touting at-home tests from everything from fertility to menopause, food sensitivities to genetic tests that not only reveal ancestral mysteries but also uncover health concerns.

Patients are more likely to try these tests and then arrive in the office, concerned about the findings. As a clinician, how can you be ready, and what should you know about at-home hormone and blood tests?

RELATED: Estimate Diabetes Patients Risk of Complications Over the Next 10 Years with This New Tool

Florence Health spoke to several health professionals whove seen an increase in at-home testing among their patients, including allergist Tania Elliott, MD, spokesperson for American College of Allergy, Asthma and Immunology; reproductive endocrinologist Dale Stovall, OB-GYN with Methodist Health System; and Kathy Moran, spokesperson for the American College of Medical Genetics (ACMG).

The resounding response: These tests are not accurate enough for consumers to be using without medical oversight.

The growing number of over-the-counter tests available to consumers include DNA tests, such as the popular 23andMe. This test doesnt just promise to match your ancestry, but it also offers health predisposition for diseases and other traits that may run in a persons DNA. The FDA-authorized reports are all based on a saliva sample.

Food sensitivity tests, such as those by EverlyWell, measures IgG levels in nearly 100 foods with just the prick of blood from a consumers finger. At-home pregnancy, fertility and menopause predictors, as well as testing for common STIs, sold in big-box stores like Walmart, use urine or blood samples to help patients self-diagnose their symptoms, without the need of a medical practitioner.

The most common hormone test is a pregnancy test, which provides a positive or negative result, Dr. Stovall says. These have been used for a long time and are very good and have gotten even more sensitive over the course of 30 years to accurately predict a pregnancy.

Newer tests used to predict menopause, on the other hand, are not for primetime, he adds. These tests are using the Anti-Mllerian hormone that we use to determine how well ovaries are ready to be stimulated for fertility injections. They think we can use this to see what fertility chances are, but they are not accurate as different people have different levels and a low dose doesnt mean earlier menopause or lower fertility rates.

Food allergy tests are similarly misleading.

RELATED: The Pros and Cons of Oral Immunotherapy

What I am seeing are at-home IgG tests to check allergy antibodies in foods, says Dr. Elliott. Unfortunately, there is no evidence that if a patient has any sensitivity to a food that they have an allergy.

Genome tests can review genes and spot mutations that may indicate an increased risk for certain cancers or diseases like Alzheimers, Huntingtons or Parkinsons. The FDA permits breast and ovarian cancer screenings at home using DNA tests. But the ACMG stresses that results are increasingly complex and must be placed in context with medical and family history to provide meaningful information.

Just like when a patient shows you a diagnosis from Dr. Google, you should trust the anxiety and ask questions to find what led them to the tests in the first place.

If a patient who is 35 is taking a test to determine menopause, I want to know what is making her think that she may need to worry about early menopause, says Dr. Stovall. Is she experiencing any symptoms, such as hot flashes and a lack of a period? Does she have a family history with a mother or aunt who went through early menopause? Once I determine the issue, I can then work on additional tests and how to manage my patients care.

For food allergies, explaining the reliability of the tests may help, too.

Patients feel if they have an antibody for a food that it must mean they have an allergy to that food, which isnt necessarily true, says Dr. Elliott. The best thing a patient should do if theyve taken an IgG test and feel they have an allergy is to keep a food diary for two weeks. Patients should write down what they ate and if they experienced any bloating, headaches or troublesome bowel movements after eating, then sit down with an allergist or nutritionist who is trained to do the detective work.

RELATED: Americans Search for Info About CBD Online More Than Any Other Health Product

You should also highlight the issues with over-the-counter tests and how to use them properly. At-home tests are most helpful when confirming a suspicion. If I had eggs and ketchup for breakfast and broke out in hives, then I can take the IgG test and it may show me it was the eggs and not the ketchup, says Dr. Elliott.

But, remind them not to treat their own symptoms and to continue seeking the help of a medical professional to manage the findings and properly confirm a diagnosis.

First, you can inform patients that test manufacturers often claim to have evidence that theyre more helpful than harmful, but they usually lack scientific studies to back this up. It also helps to use examples.

Take genome testing, for instance. A negative result in a health trait could lead to a false reassurance while a positive trait discovered without the appropriate counseling could lead to wrong medical decisions.

Consumers who consider participating in direct to consumer (DTC) genetic testing must be aware of the limited results that they will receive and the types of questions that they will have to anticipate in the follow-up of the results, according to the ACMG.

RELATED: You Know About the Importance of the Social Determinants of Health Now What?

You can also refer them to the appropriate medical foundation for their concerns, as many are attempting to quell the home-testing fervor by offering deeper explanations online. The Parkinsons Foundation, for example, explains proper gene testing for the disease includes genes GBA, PARK7, SNCA, LRRK2, parkin and PINK1. At-home kits only review mutations in LRRK2 and GBA.

And last, hit home that at-home tests are meant to help patients, but for most, the technology isnt advanced enough to be a final diagnosis to any medical concern.

Of all the tests available to patients, the most reliable are hormonal tests for women. Pregnancy tests can be up to 99 percent accurate, if performed correctly, and fertility tests are also very reliable.

Luteinizing hormone(LH) is typically very low in the body but has found to spike up to three times right before the release of an egg, Dr. Stovall says. (Patients should use the test every day until they see the spike to know when they are ovulating.)

These same tests can predict menopause for older women. The average age of a woman hitting menopause is 51.2 years. The closer to that age when a woman takes this test, it can indicate she is perimenopausal or menopausal, he says.

Although the technology to provide accurate results with most at-home tests is not 100 percent, manufacturers are finding consumers wanting more of these tests to manage their own care. This is not a bad thing, says Dr. Stovall.

RELATED: 7 Cant-Miss Talking Points When Patients Ask You about Trying Cannabis

This industry is going to grow over time, he explains. There are at-home tests for so many things, such as EKG tests, blood sugar tests overall these tests are good things and help get patients on board with their health.

The problem is the discrepancy between the tests accuracy and how much a patient trusts its resolve, which can lead to unnecessary anxiety or postponing seeking necessary care.

As more research is done, we can use these tests for diagnosis and testing can become more helpful to a patient, Dr. Stovall adds. Its too early right now, but it doesnt mean they wont be perfected.

Read the rest here:
The Pros and Cons of At-Home Testing for Patients: What Clinicians Should Know - Florence Health

Recommendation and review posted by Bethany Smith

Alopecia is a medical term, which is generally used for hair loss. It is a common autoimmune disorder that often results in unpredictable hair loss, which can lead to complete loss of hair on the scalp or, in uttermost cases, the entire body. This extreme condition can affect anyone irrespective of gender and age. Medications such as Minoxidil or Rogaine are the topical agents that are used for the treatment of disease. Other treatments of alopecia include medications that are sometimes used for other autoimmune disorders.

The alopecia market is anticipated to grow in the forecast, owing to the increasing prevalence of alopecia areata and Rise in incidence of chronic disorders such as, cancer, polycystic ovary syndrome (PCOS), hyperthyroidism, hypothyroidism, acute stress disorder, hypopituitarism, lupus. However, the rising awareness among people about hair loss is likely to add novel opportunities in the forecast period.

Top Companies Covered in this Report:1. CAPILLUS, 2. Sun Pharmaceutical Industries Ltd., 3. Cipla Inc., 4. Merck and Co., Inc., 5. Transitions Hair, 6. Follica, Inc., 7. Johnson and Johnson Services, Inc., 8. Concert Pharmaceuticals, 9. HCell Inc., 10. GlaxoSmithKline plc.

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The Global Alopecia Market Analysis to 2027 is a specialized and in-depth study of the healthcare IT industry with a special focus on the global market trend analysis. The report aims to provide an overview of Alopecia market with detailed market segmentation by service type, age group, and geography. The global Alopecia market is expected to witness high growth during the forecast period. The report provides key statistics on the market status of the leading Alopecia market players and offers key trends and opportunities in the market.

The global alopecia market is segmented on the basis of disease type, drug type, route of administration and distribution channel. Based on disease type, the market is classified as, alopecia areata, androgenic alopecia, alopecia totalis, ciatricial alopecia and traction alopecia. Based on the drug type, the market is segmented into minoxidil, finasteride and other drug types. On the basis of route of administration, the market is categorized as oral, topical and injectable. Based on the distribution channel, the market is segmented as hospitals, retail pharmacies and online pharmacies.

The report analyzes factors affecting Alopecia market from both demand and supply side and further evaluates market dynamics effecting the market during the forecast period i.e., drivers, restraints, opportunities, and future trend. The report also provides exhaustive PEST analysis for all five regions namely; North America, Europe, APAC, MEA and South & Central America after evaluating political, economic, social and technological factors effecting the Alopecia market in these regions.

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Fundamentals of Table of Content:

1 Report Overview1.1 Study Scope1.2 Key Market Segments1.3 Players Covered1.4 Market Analysis by Type1.5 Market by Application1.6 Study Objectives1.7 Years Considered

2 Global Growth Trends2.1 Alopecia Market Size2.2 Alopecia Growth Trends by Regions2.3 Industry Trends

3 Market Share by Key Players3.1 Alopecia Market Size by Manufacturers3.2 Alopecia Key Players Head office and Area Served3.3 Key Players Alopecia Product/Solution/Service3.4 Date of Enter into Alopecia Market3.5 Mergers & Acquisitions, Expansion Plans

4 Breakdown Data by Product4.1 Global Alopecia Sales by Product4.2 Global Alopecia Revenue by Product4.3 Alopecia Price by Product

5 Breakdown Data by End User5.1 Overview5.2 Global Alopecia Breakdown Data by End User

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Originally posted here:
Alopecia Market Outlook and Opportunities in Grooming Regions with Forecast to 2027 | CAPILLUS, Sun Pharmaceutical Industries, Cipla, Merck and Co,...

Recommendation and review posted by Bethany Smith

Alex Nowbar reviews the latest research from the top medical journals

Annals of Internal Medicine

Coaching for weight maintenance

Conroy et al examine the role of coaching in weight management in a randomised controlled trial of 194 participants who had intentionally lost at least 5% of their body weight in the last two years. They also threw in a bit of electronic health records for good measure. The study arms were: tracking via electronic health records versus the same plus coaching. So really its a trial of a two year coaching programme. Good news, at two years weight regain in the coaching group was lower (2.1kg vs 4.9kg in the tracking only group). Those in the coaching group were more likely to maintain weight loss of at least 5%. The coaching was in the form of personalised health coaching through the electronic health record patient portal, with intensive initial contact (weekly for a month) that tapered off over the two years. The electronic health record served as an important part of the control for those in the tracking group because it was used to send the tracking participants questionnaires on general health. This was a well designed study of an interesting intervention format i.e. an electronic health record-based lifestyle intervention.

JAMA

Bempedoic acid for reducing LDL Cholesterol

If you name a trial CLEAR Wisdom, you should be prepared to be accused of being smug. But perhaps Esperion Therapeutics should be. Bempedoic acid was tested in a double-blind randomised controlled trial of 779 people with high cardiovascular risk and raised LDL-C despite maximally tolerated lipid-lowering therapy. At 12 weeks, it reduced LDL-C levels dramatically compared to placebo and was reasonably well-tolerated and safe. Its now ready for the next stage of clinical trials to establish the impact on cardiovascular outcomes. There are a number of players on this stage though, PCSK-9 inhibitors in particular. In fact there are a spate of new cardiovascular drugs making an appearance and each ones glory is being somewhat diluted by the others. It is therefore unclear which will become commonly prescribed and which will fall by the wayside. Unfortunately I suspect it will be marketing and trendiness that determines how this plays out because the head-to-head data is unlikely to come soon.

Lancet

Hope for NASH

Thyroid hormone analogue, resmetirom, is an oral drug for treatment of NASH fibrosis. Harrison et al tested it in a double-blind Phase 2 randomised controlled trial of 125 patients in the US. Naturally, drug-makers, Madrigal, were closely involved in this studys design and analysis. They found it reduced hepatic fat at 12 weeks as measured by MRI proton-density fat fraction. As well as singing the praises of this new drug, this study highlights the utility of this non-invasive measure of hepatic fat as opposed to biopsy which is not practical for serial monitoring. There were also significant lipid profile improvements with resmetirom compared to placebo. However all the efficacy outcomes assessed in this study were biomarkers not actual outcomes so the clinical benefits are not yet known. But these data are promising and certainly confirm that the thyroid hormone pathway has an important role in NASH fibrosis.

Steroids for the DIPs and PIPs

I hate to stereotype, but Dutch trials are often particularly robustly designed, like this double-blind randomised controlled trial of 6 weeks of prednisolone 10mg once daily versus placebo. The inclusion criteria are important here. Participants had to have symptomatic hand osteoarthritis with signs of inflammation in their distal and proximal interphalangeal joints. Objective evidence was required, including osteoarthritis nodes, swelling, or erythema and synovial thickening on ultrasound. And the inclusion criteria even went one step further to require finger pain of at least 30 mm on a 100-mm visual analogue scale (VAS) that flared up during a 48-h non-steroidal anti-inflammatory drug washout (defined as worsening of finger pain by at least 20 mm on the VAS). The primary endpoint of finger pain improvement on the VAS at 6 weeks was positive with a large effect size with no adverse safety signal. These data are extremely useful for those with this condition, but it sounds like itll be in the rheumatologists domain for now. The inclusion assessment appears too strict to be feasible in primary care.

JAMA Internal Medicine

Testosterone and thromboembolic risk

It seems obvious that testosterone therapy should be used only to treat hypogonadism and even then it should not be taken lightly. It has previously been linked to higher risk of heart attack or stroke. This large US observational study of men with deep vein thrombosis or pulmonary embolism (but without a cancer diagnosis) found an association between short-term testosterone therapy and increased risk of a thromboembolic event. The study design was interesting as each individual acted as their own control by examining them 6-12 months before the thromboembolic event while the 6 month period before the event was considered the case period. Other key findings were that thromboembolic risk was highest in the first 3 months after starting testosterone therapy, that route of testosterone made no difference to the risk and that the risk was present in men with or without hypogonadism.

NEJM

Apple watch rhythm notifications

Can an Apple watch detect atrial fibrillation? Probably. 419,297 young people participated in this study. They were monitored for a median of 117 days. An irregular rhythm was flagged in 0.52% of them (3% in the over 65s). These people got sent an ECG patch to wear for 7 days. Of those, only 21% returned the patch to the researchers. Of those who returned the patch, a third had documented atrial fibrillation. There is no comparator group to know whether the incidence of detected atrial fibrillation would have been similar without using the watch. And there was huge potential for selection bias, for example people who felt symptomatic might have been more likely to participate (so these data represent a skewed population) and people with more irregular rhythm periods and/or more symptoms might have been more likely to return the patch (again skewing the data). These bias issues are the reason studies arent conducted in this manner. This study design neither answers the question it says it set out to answer, nor provides clinically relevant evidence.

The new and improved smallpox vaccine

Pittman et al compared two doses of a new smallpox vaccine called modified vaccinia Ankara (MVA) to an existing vaccine (only requiring one dose) in an open-label randomised controlled trial of 440 people. They were looking for non-inferiority in peak antibody titres and the so-called take reaction which were both assessed in a blinded fashion. It is prudent but alarming that we are preparing our defences against a disease that has supposedly been eradicated. But there could be another outbreak one day. The existing vaccine is effective but carries fairly significant risks and hence the need for something better. MVA won the day with respect to equivalent efficacy and fewer serious adverse events. But do these efficacy endpoints (antibodies and the take) really reflect the protection this vaccine provides? And how long would the protection last? Who knows.

Colchicine after myocardial infarction

The inflammatory hypothesis in atherosclerosis has been bubbling away for many years. COLCOT is the first study that practically applies this with a degree of success. COLCOT was a double-blind randomised controlled trial of colchicine 500mcg once daily versus placebo for cardiovascular risk reduction after an MI. Colchicine started within 30 days of the MI was found to reduce cardiovascular events at 2 years, and surprisingly without substantially increasing diarrhoea rates. The cardiovascular event reduction was mainly driven by fewer strokes and fewer revascularisations for angina though which is a bit disappointing since MI and death prevention is what we were really hoping for. Colchicine for prevention of need for revascularisation doesnt even really make sense because there are plenty of other anti-anginal therapies. Arguably this event shouldnt have been part of the primary endpoint. Overall, there are benefits of colchicine that can justify its use but will patients really find it worth the hassle given all the other medications they get given (and often dont adhere to) after an MI?

Alex Nowbar is a clinical research fellow at Imperial College London

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Alex Nowbar's weekly review22 November 2019 - The BMJ - The BMJ

Recommendation and review posted by Bethany Smith

Human Chorionic Gonadotropin (HCG) Marketreport provides comprehensive insights, revenue generation information, and other significant information related to the global Human Chorionic Gonadotropin (HCG) market, as well as the different trends, drivers and restraints, opportunities, and threats in the parent market till 2024.

Rise in the incidence of male hypogonadism among the geriatric population is the key factor driving the market growth. In addition, increase in traction gained by hCG among healthcare professionals and patients would supplement the market growth. However, cautions and warnings issued by the FDA pertaining to the side effects of hCG therapy is expected to hamper the market growth. Moreover, untapped economies are set to offer lucrative opportunities for the expansion of the hCG market.

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Key Players covered in this report:

Scrippslabs

Leebio

Kamiya Biomedical Company

Human chorionic gonadotropin (hCG) belongs to the family of gonadotropin hormones and is naturally produced by the placenta. hCG hormone plays a pivotal role after the formation of the embryo, and hence has gained significant traction over the years for the treatment of infertility in women and men. hCG triggers ovulation in women and assists in increasing the sperm count in men

Market by Type

-Subunit (-hCG)

-Subunit (-hCG)

Others

Market by Application

Research institutions

Pharmaceutical

Others

Reasons for Buying this Report

This report provides pin-point analysis for changing competitive dynamics

It provides a forward looking perspective on different factors driving or restraining market growth

It provides a 5-year forecast assessed on the basis of how the market is predicted to grow

It helps in understanding the key product segments and their future

It provides pin point analysis of changing competition dynamics and keeps you ahead of competitors

It helps in making informed business decisions by having complete insights of market and by making in-depth analysis of market segments

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Table of Contents

1 Executive Summary Market Attractiveness Analysis

1.1 Global Human Chorionic Gonadotropin (HCG) Market, by Product Type

1.2 Global Human Chorionic Gonadotropin (HCG) Market, by End User

1.3 Global Human Chorionic Gonadotropin (HCG) Market, by Region

2 Market Introduction

2.1 Definition

2.2 Scope of the Study

2.3 Market Structure

2.4 Key Buying Criteria

3 Research Methodology

3.1 Research Process

3.2 Primary Research

3.3 Secondary Research

3.4 Market Size Estimation

3.5 List of Assumptions

4 Market Dynamics

4.1 Introduction

4.1 Drivers

4.2. Integration of Advanced Technologies

4.3 Restraint

4.4 Opportunities

5 Market Factor Analysis

5.1 Supply Chain Analysis

5.2 Porters Five Forces Analysis

5.2.1 Threat of New Entrants

5.2.2 Bargaining Power of Suppliers

5.2.3 Threat of Substitutes

5.2.4 Bargaining Power of Buyers

5.2.5 Intensity of Rivalry

6 Market Channel

6.1 Overview

6.2 Store-Based

6.3 Non-Store-Based

7 Global Human Chorionic Gonadotropin (HCG) Market, by Product Type

8 Global Human Chorionic Gonadotropin (HCG) Market, by End User

9 Global Human Chorionic Gonadotropin (HCG) Market, by Region

Continued

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Human Chorionic Gonadotropin (HCG) Market 2019 By Share, Size, industry Trends, Outlook, Revenue, Manufacturers, Demand and Forecast to 2024 - Downey...

Recommendation and review posted by Bethany Smith

By Paul Biegler

US scientists have overcome a major stumbling block in the creation of mini-organs, programming cells to take on the desired shape rather than relying on 3D printing or external scaffolds.

This inside out approach, described in a paper in the journal Cell Systems, could signal a paradigm shift in how mini-hearts, kidneys and brains are grown on the lab bench a technique used to study disease that may one day lead to personalised organ transplants.

The team, led by bioengineer Todd McDevitt at Gladstone Institutes in the US, was driven by an enduring issue with state-of-the-art ways of producing mini-organs such as 3D printing. The cells just wont stay put.

Making a mini-organ or organoid starts when scientists take a persons skin cell and, using the right mix of agents, turn it into an induced pluripotent stem cell. This IPS cell is the blank cheque of biology, capable of becoming almost any cell type.

Grow it into a mini-kidney, say, and you can reproduce kidney diseases and test treatments in a dish sitting on your lab bench. But how faithful that model is depends on the physical organisation of the cells; to mimic a real deal kidney, 3D printing is often used.

But cells, much like unruly teenagers, have a mind of their own and will often wander away from their printed position.

McDevitts team wanted to own those cellular minds and so took control of two genes that together make up something of a joystick that directs how the cells organise.

CDH1 and ROCK1 figure heavily in the complex moves that lead to the final configuration of a group of cells. The pair influences stickiness and repulsion between cells, the surface tension that makes them spherical and their overall speed of migration.

The researchers used the editing tool CRISPR to knock out the two genes at various stages in the evolution of a clump of cells. Their aim was to make a bulls eye pattern, a shape thats common in human development, including in early embryo formation.

To detect that aspirational pattern, they engineered another tweak making the cells fluoresce when CDH1 and ROCK1 were neutralised.

But there was a problem.

Factor in all the potential time points where the genes could be knocked out, the proportion of cells to be targeted, and a host of other variables, and the researchers calculated theyd need to do nearly 9000 trial-and-error experiments.

So they called on AI. They trained a machine learning model to compute the precise pattern of gene knockouts needed to realise their dream shape.

Machine learning can predict what movie you might like based on your viewing history, but it can also generate new insights into biological systems by mimicking them, says co-author Demarcus Briers, from the Boston University Bioinformatics Program.

Our machine-learning model allows us to predict new ways that stem cells can organise themselves, and produces instructions for how to recreate these predictions in the lab.

That model hit a bulls eye, quite literally, allowing the team to produce the concentric pattern of cells they were aiming at.

"We've shown how we can leverage the intrinsic ability of stem cells to organise," says McDevitt. "This gives us a new way of engineering tissues, rather than a printing approach where you try to physically force cells into a specific configuration."

Ultimately, that concrete target shape will give way to a target in the abstract, one with potential to shift the life course.

"We're now on the path to truly engineering multicellular organization, which is the precursor to engineering organs," McDevitt says. "When we can create human organs in the lab, we can use them to study aspects of biology and disease that we wouldn't otherwise be able to."

Read more:
AI helps cells pull themselves together - Cosmos

Recommendation and review posted by Bethany Smith

The ability to turn human cells into anything we want sounds like the stuff of science fiction. But one Cambridge biotech says it's cracked the code

A sustainable source of human stem cells is one of the holy grails of modern medicine.

With applications as broad as re-growing failed organs, fighting cancer, and stopping animal testing, stem cell therapy is predicted to be worth US$35bn by 2023.

Now, Cambridge startup bit bio, has a new approach to re-coding skin cells from adult humans, and rewinding the clock to give them the power of stem cells, and then turn them into whatever we want them to be all without the controversial involvement of human embryos.

This, says neurosurgeon and founder Dr Mark Kotter, will democratise stem cells, so that anyone can use them, at any time.

The private sector is already placing big bets on the technology, with start-ups in the space raising as much as US$16mln in recent funding rounds.

Kotter says that our inability to produce enough human stem cells to match our need puts troubling limits on research and drug development.

In drug discovery, the biggest bottleneck is the mismatch between animal models and animal cell lines used for drug discovery, and then human setting used in the clinical trial, he explains.

Around 3% of new drugs make it all the way through trials and to market, he says, and the biggest reasons treatments tend to fail in clinical study is that they are either toxic to humans, or they dont work.

The only solution is to bring the human element back to the early stages, says Kotter.

If new therapies were tested on human tissue first, it would reduce or even bypass the need to test on animals, as well as speeding up development.

Kotter founded bit bio, formerly known as Elpis BioMed, in 2016, in addition to startup Meatable, which produces meat by growing cultures in the lab, rather than rearing animals for the table.

The time is now for bit bio, because what it is doing has only been possible since a Nobel Prize-winning discovery twelve years ago, which turned the world of stem cell research upside down.

Kyoto University researcher Shinya Yamanaka proved that it was possible to take a mature human skin cell and reprogram it to be like the stem cell of an embryo.

Until this revelation, stem cell research had been dogged by controversy and expense, as scientists had to use human embryos and umbilical cords as a source of stem cells, and then simulate complex conditions inside the womb in order to make them develop into the cells they desired.

One big problem in early cell reprogramming was that stem cells are incredibly alert to invading DNA and silences any foreign material it detects.

This meant that past attempts run a different program inside a cell often failed, because the cell destroyed it.

What happened next was a moment of "serendipity" in the lab, says Kotter.

Through trial and error, bit bio found they could use certain safe harbours where information is protected within cells, to stop theinterference.

By taking the genetic switch for gene silencing and placing it inside a safe harbour, and then separately running the new cell program inside another safe harbour, scientists found they could override gene silencing in order to change the cell type.

This approach is what Kotter says makes bit bio unique.

The lab can produce up to a kilogram of human cells now, and its tech platform opti-oxhas also proved that it can generate two human cell types with 100% accuracy.

Kotter says that now the range of cells able to be produced matters more than the quantity.

The company is now focused on discovering what separates one type of cell from another, which Kotter says will allow the firm to decode the building blocks of life.

To this end, bit bio is using machine learning to analyse the differences between every type of human cell, from bone marrow cells to liver cells, and create a reference map for all the different types.

Once the research is complete, the company hopes it willbe able to generate any type of human cell, at scale, and with ultimate precision.

Preparations are underway for a Series A funding round, and Kotter says that he is determined not to sell the business, having already rejected offers from would-be buyers.

Bit bio though is in an area hot with competition, which moves quickly.

A US$16mln Series A mega funding round was recently announced in October by another Cambridge start-up, Mogrify, which is hoping to master direct cell reprogramming and turn blood cells straight into brain cells, or any other type.

Mogrify uses big data to identify the small molecules needed to convert, maintain and culture a target cell type.

While both companies were finalists in the 2018 Cambridge Startup of the Year award, bit bio was the one to scoop the prize.

One aspect that separates the two companies is that Mogrify uses its technology to turn cells directly into other cell types, rather than using it to rewindto the stem cell phase, which is when cells can reproduce very quickly,

Kotter says that this stem cell phase focusis whatallows bit bio to havea stable supply of human cells.

If bit bio completes a similar, or even bigger, fundraise, it could advance the fledgling firm from seed to stem, in its attempt to stabilise a production line for essential cell technology.

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Decoding the building blocks of life: bit bio races toward a sustainable source of human cells - Proactive Investors UK

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DetailsCategory: Small MoleculesPublished on Friday, 22 November 2019 14:04Hits: 302

Two Phase III Calquence trials demonstrated superior progression-free survival across multiple settings while maintaining favourable tolerability

Calquence combined with obinutuzumab and as monotherapy reducedthe risk of disease progression or death by 90% and 80%, respectively in ELEVATE-TN

LONDON, UK I November 21, 2019 I AstraZeneca today announced that the US Food and Drug Administration (FDA) has approved Calquence (acalabrutinib) for adult patients with chronic lymphocytic leukaemia (CLL) or small lymphocytic lymphoma (SLL).1 The US approval was granted under the FDAs Real-Time Oncology Review and newly established Project Orbis programmes.

The approval is based on positive results from the interim analyses of two Phase III clinical trials, ELEVATE-TN in patients with previously untreated CLL and ASCEND in patients with relapsed or refractory CLL. Together, the trials showed that Calquence in combination with obinutuzumab or as a monotherapy significantly reduced the relative risk of disease progression or death versus the comparator arms in both 1st-line and relapsed or refractory CLL. Across both trials, the safety and tolerability of Calquence were consistent with its established profile.1

Dave Fredrickson, Executive Vice President, Oncology Business Unit said: With over 20,000 new cases anticipated this year in the US alone, todays approval of Calquence provides new hope for patients with one of the most common types of adult leukaemia, offering outstanding efficacy and a favourable tolerability profile. The chronic lymphocytic leukaemia patient population is known to face multiple comorbidities, and tolerability is a critical factor in their treatment.

Dr Jeff Sharman, Director of Research at Willamette Valley Cancer Institute, Medical Director of Hematology Research for The US Oncology Network, and a lead author of the ELEVATE-TN trial, said: Tolerability remains an issue in the current treatment landscape of chronic lymphocytic leukaemia, which may require ongoing therapy for many years. In the ELEVATE-TN and ASCEND trials comparing Calquence to commonly used treatment regimens, Calquence demonstrated a clinically meaningful improvement in progression-free survival in patients across multiple settings, while maintaining its favourable tolerability and safety profile.

The results of the interim analysis of the ELEVATE-TN trial will be presented at the upcoming American Society of Hematology congress.2

The trial showed a statistically significant and clinically meaningful improvement in progression-free survival (PFS) for patients treated with either Calquence in combination with obinutuzumab or Calquence monotherapy versus chlorambucil chemotherapy plus obinutuzumab, a current standard-of-care combination used in the control arm.1

In the Calquence combination arm, risk of disease progression or death was reduced by 90% (HR 0.10; 95% CI, 0.06-0.17, p<0.0001) and in the monotherapy arm it was reduced by 80% (HR 0.20; 95% CI, 0.13-0.30, p<0.0001).1

The median time to disease progression for patients treated with Calquence in combination with obinutuzumab or as a monotherapy has not yet been reached versus 22.6 months (95% CI, 20-28) for chlorambucil plus obinutuzumab.1

ELEVATE-TN safety overview (most common ARs, 15%):1

Includes multiple ADR terms.

In patients treated with the combination of Calquence plus obinutuzumab, adverse reactions (ARs) led to treatment discontinuation in 11% of patients and a dose reduction of Calquence in 7% of patients. In the monotherapy arm, ARs led to discontinuation in 10% and dose reduction in 4% of patients.1 In the control arm, ARs led to regimen discontinuation in 14% of patients with a dose reduction of chlorambucil in 28% of patients.3 There were no dose reductions for obinutuzumab.1,3

In 1,029 patients with haematologic malignancies who were treated with Calquence 100mg approximately every 12 hours across multiple clinical trials, where 88% received treatment for at least six months and 79% received treatment for at least one year, serious or Grade 3 infections occurred in 19%, and Grade 3 atrial fibrillation and flutter occurred in 1.1% of patients.In the same patient population, major haemorrhage occurred in 3.0% (serious or Grade 3 bleeding or any central nervous system bleeding), with fatal haemorrhage occurring in 0.1% of patients. Second primary malignancies (all grades) including skin cancers occurred in 12% of patients.1

The US approval is among the first to be granted under Project Orbis, an initiative of the US FDA Oncology Center of Excellence, which provides a framework for concurrent submission and review of oncology medicines among international partners. The FDA, the Australian Therapeutic Goods Administration, and Health Canada collaborated on this review. 4

About Calquence

In the US, Calquence (acalabrutinib) is indicated for the treatment of adult patients with chronic lymphocytic leukaemia (CLL)/small lymphocytic lymphoma (SLL). In the US, Canada, Australia, Brazil, Qatar, the United Arab Emirates, Mexico, Argentina, Singapore, Chile, and recently India, Calquence is indicated for adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy. Approved under accelerated review in the US, continued approval for previously treated MCL is contingent upon verification and confirmation of clinical benefit in confirmatory trials.

Calquence is a next-generation selective inhibitor of Brutons tyrosine kinase (BTK). Calquence binds covalently to BTK, thereby inhibiting its activity.1,5,6,7 In B-cells, BTK signalling results in activation of pathways necessary for B-cell proliferation, trafficking, chemotaxis, and adhesion.1

As part of an extensive clinical development programme, AstraZeneca and Acerta Pharma are currently evaluating Calquence in 23 company-sponsored clinical trials. Calquence is being developed for the treatment of multiple B-cell blood cancers including CLL, MCL, diffuse large B-cell lymphoma, Waldenstrm macroglobulinaemia and follicular lymphoma and other haematologic malignancies. Several Phase III clinical trials in CLL are ongoing, including ASCEND, ELEVATE-TN, ELEVATE-RR (ACE-CL-006) evaluating Calquence versus ibrutinib in patients with previously treated high-risk CLL, and ACE-CL-311 evaluating Calquence in combination with venetoclax and with/without obinutuzumab versus chemoimmunotherapy in patients with previously untreated CLL without 17p deletion or TP53 mutation.

About ELEVATE-TN

ELEVATE-TN (ACE-CL-007) is a randomised, multicentre, open-label Phase III trial evaluating the safety and efficacy ofCalquence in combination with obinutuzumab, a CD20 monoclonal antibody, or Calquence alone versus chlorambucil, a chemotherapy, in combination with obinutuzumab in previously untreated patients with CLL. In the trial, 535 patients were randomised (1:1:1) into three arms. Patients in the first arm received chlorambucil in combination with obinutuzumab. Patients in the second arm received Calquence (100mg twice daily until disease progression or unacceptable toxicity) in combination with obinutuzumab. Patients in the third arm received Calquence monotherapy (100mg twice daily until disease progression or unacceptable toxicity).1,8

The primary endpoint is PFS in the Calquence and obinutuzumab arm compared to the chlorambucil and obinutuzumab arm, assessed by an independent review committee (IRC), and a key secondary endpoint is IRC-assessed PFS in the Calquence monotherapy arm compared to the chlorambucil and obinutuzumab arm. Other secondary endpoints include objective response rate, time to next treatment and overall survival.1,8

About ASCEND

ASCEND (ACE-CL-309) is a global, randomised, multicentre, open-label Phase III trial evaluating the efficacy of Calquence in previously treated patients with CLL. In the trial, 310 patients were randomised (1:1) into two arms. Patients in the first arm received Calquence monotherapy (100mg twice daily until disease progression or unacceptable toxicity). Patients in the second arm received investigators choice of either rituximab, a CD20 monoclonal antibody, in combination with idelalisib, a PI3K inhibitor, or rituximab in combination with bendamustine, a chemotherapy.1,9

The primary endpoint is PFS assessed by an IRC, and key secondary endpoints include physician-assessed PFS, IRC- and physician-assessed overall response rate and duration of response, as well as overall survival, patient-reported outcomes and time to next treatment.1,9

About CLL

Chronic lymphocytic leukaemia (CLL) is one of the most common types of leukaemia in adults, with an estimated 105,000 new cases globally each year and 20,720 new cases in the US in 2019, and the number of people living with CLL is expected to grow with improved treatment as patients live longer with the disease.10,11,12,13 In CLL, too many blood stem cells in the bone marrow become abnormal lymphocytes and these abnormal cells have difficulty fighting infections.10 As the number of abnormal cells grows there is less room for healthy white blood cells, red blood cells and platelets.10 This could result in anaemia, infection and bleeding.10 B-cell receptor signalling through BTK is one of the essential growth pathways for CLL.

About AstraZeneca in haematology

Leveraging its strength in oncology, AstraZeneca has established haematology as one of four key oncology disease areas of focus. The Companys haematology franchise includes two US FDA-approved medicines and a robust global development programme for a broad portfolio of potential blood cancer treatments. Acerta Pharma serves as AstraZenecas haematology research and development arm. AstraZeneca partners with like-minded science-led companies to advance the discovery and development of therapies to address unmet need.

About AstraZeneca in oncology

AstraZeneca has a deep-rooted heritage in oncology and offers a quickly-growing portfolio of new medicines that has the potential to transform patients lives and the Companys future. With at least six new medicines to be launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, the Company is committed to advance oncology as a key growth driver for AstraZeneca focused on lung, ovarian, breast and blood cancers. In addition to AstraZenecas main capabilities, the Company is actively pursuing innovative partnerships and investments that accelerate the delivery of our strategy, as illustrated by the investment in Acerta Pharma in haematology.

By harnessing the power of four scientific platforms Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and, one day, eliminate cancer as a cause of death.

About AstraZeneca

AstraZeneca is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in three therapy areas - Oncology, Cardiovascular, Renal and Metabolism, and Respiratory. AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.

References

1. CALQUENCE (acalabrutinib) [prescribing information]. Wilmington, DE; AstraZeneca Pharmaceuticals LP; 2019.

2. Sharman JP, et al. ELEVATE TN: Phase 3 Study of Acalabrutinib Combined with Obinutuzumab (O) or Alone Vs O Plus Chlorambucil (Clb) in Patients (Pts) with Treatment-Naive Chronic Lymphocytic Leukemia (CLL). Abstract 31 at: American Society of Hematology 2019 Annual Meeting and Exposition. Available online. Accessed November 2019.

3. Data on File. REF-64711. AstraZeneca Pharmaceuticals LP, Wilmington, DE.

4. US Food and Drug Administration. Project Orbis. Available online. Accessed November 2019.

5. Wu J, Zhang M & Liu D. Acalabrutinib (ACP-196): a selective second-generation BTK inhibitor. J Hematol Oncol. 2016;9(21).

6. Khan Y & OBrien S. Acalabrutinib and its use in treatment of chronic lymphocytic leukemia. Future Oncol. 2018;15(6).

7. Byrd JC, et al. Acalabrutinib (ACP-196) in Relapsed Chronic Lymphocytic Leukemia. N Engl J Med. 2016; 374:323-332.

8. ClinicalTrials.gov. Elevate CLL TN: Study of Obinutuzumab + Chlorambucil, Acalabrutinib (ACP-196) + Obinutuzumab, and Acalabrutinib in Subjects With Previously Untreated CLL. NCT02475681. Available online. Accessed November 2019.

9. ClinicalTrials.gov. A Study of Acalabrutinib vs Investigator's Choice of Idelalisib Plus Rituximab or Bendamustine Plus Rituximab in R/R CLL. NCT02970318. Available online. Accessed November 2019.

10. National Cancer Institute. Chronic Lymphocytic Leukemia Treatment (PDQ)Patient Version. Available online. Accessed November 2019.

11. Global Burden of Disease Cancer Collaboration. Global, Regional, and National Cancer Incidence, Mortality, Years of Life Lost, Years Lived With Disability, and Disability-Adjusted Life-Years for 29 Cancer Groups, 1990 to 2016. JAMA Oncol. 2018;4(11):1553-1568.

12. American Cancer Society. Key Statistics for Chronic Lymphocytic Leukemia. Available online. Accessed November 2019.

13. Jain N, et al. Prevalence and Economic Burden of Chronic Lymphocytic Leukemia (CLL) in the Era of Oral Targeted Therapies. Blood. 2015;126:871.

SOURCE: AstraZeneca

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Calquence approved in the US for adult patients with chronic lymphocytic leukaemia | Small Molecules | News Channels - PipelineReview.com

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By Ameera StewardFor the Birmingham Times

November is Diabetes Awareness Month, a time to raise understanding about diabetes, an illness that affects millions of Americans of all ages.

There are two types of diabetes: Type 1 and Type 2.

Type 1, once known as juvenile diabetes or insulin-dependent diabetes, is a chronic condition in which the pancreas, an organ located in the abdomen that plays an essential role in converting food into fuel for the body, produces little or no insulin, the hormone needed to allow sugar (glucose) to enter the bodys cells to produce energy.

Type 2 diabetes is a chronic condition that affects the way the body metabolizes glucose, an important source of fuel for the body. With Type 2, the body either resists the effects of insulin or doesnt produce enough of the hormone to maintain normal glucose levels.

The Birmingham Times recently sat down with two physicians from Brookwood Baptist Healthinternal medicine physician Erin Townsley, MD, and interventional cardiologist Hutton Brantley, MDto discuss and get advice about dealing with diabetes.

Family and Food

People with a family history of Type 2 diabetes are at an increased risk for developing the illness, Townsley said.

If you have a family history of diabetes and then have the environmental or lifestyle factors that also play a role, thats going to increase your risks, she said. Just because its in your genetics and just because you have a family history doesnt mean youre going to definitely get it; it just means youre at increased risk, compared with someone who doesnt have a family history and lives that same kind of lifestyle.

Townsley recommends that those who have diabetes avoid foods high in carbohydrates (sugars, starches, and fibers found in fruits, grains, vegetables, and milk products), such as bread and pasta, as well as limit daily intake of foods with added sugars and excess caloriesall of which increase the risks of obesity and diabetes. The other thing to watch is being sedentary or not exercising, which also can increase the risks for being obese and having diabetes.

There is no cure, per se, but I have seen diabetes essentially resolve with dramatic lifestyle changes [and] weight loss, Townsley explained. Now, that risk will always be there for [some] people, so that never goes away. But if you can change your lifestyle, lose weight, change your eating habits, and increase your physical activity, you can potentially come off diabetes medications [and] dramatically reduce your risk for developing complications [from the illness].

And there are several potential complications. According to the American Diabetes Association, diabetes can increase the risk for many serious health problems, including skin disorders and infections; eye and vision problems, such as glaucoma and cataracts; neuropathy, or nerve damage, particularly in the legs and feet; kidney disease and failure, which can result in the need for dialysis; hypertension, or high blood pressure; and stroke and heart disease.

Symptoms to Watch For

Symptoms associated with diabetes might include tiredness and increased hunger. If blood sugar is really high, it can cause a catabolic reaction (a process in which the body breaks down large molecules for energy) and lead to weight loss. Also, high blood sugar is almost like a diuretic (a substance that causes increased urination), so it can have a water-pill effect and make patients urinate frequently.

Townsley wants people to understand how important it is to recognize diabetes and get it treated early.

If you know you struggle with being overweight or obese and you have a strong family history of type 2 diabetes, even if you feel like youre young, you need to see a physician about it and get tested, she said. I think thats the biggest thingearly detection.

Sometimes patients with Type 2 diabetes dont necessarily feel bad or realize that they feel bad, Townsley added. Their blood sugar may be elevated, and they may be slowly developing problems from diabetes, but they dont feel it on a day-to-day basis.

Some patients have a hard time understanding the importance of treating [diabetes], so [some] constantly have elevated blood sugar and dont necessarily have a lot of symptoms, or at least symptoms they recognize. Diabetes is sort of a slow process, and patients may not really realize that theyre having issues from it, she said.

Complications may not happen tomorrow, next month, or even in three to five years, but they eventually will happen, and it can be 20 years from when [someone] is diagnosed. Its really sometimes hard for me to get patients to look at the long goal, so I spend a lot of time educating patients about the importance of [monitoring diabetes]. The bottom line is most people dont want to take a lot of medicines, especially when they get to the point of having to inject insulin, [which is] a really big hurdle for patients to get used to. Its a lifestyle change to have to do that every day.

Heart Disease

Brantley said diabetes is considered the highest risk factor development of one particular complication: heart disease higher than smoking or high blood pressure.

Diabetes with elevated blood glucose damages nerves and blood vessels, making people with diabetes vulnerable to the risk factors for cardiovascular disease: smoking, high blood pressure, abnormal cholesterol levels and triglycerides, he said. People with Type 1 and Type 2 [diabetes] are both at risk for early-onset heart disease.

The heart specialist added that maintaining a healthy weight is best for people with diabetes because being overweight tends to increase blood glucose, blood pressure, and blood fat levels.

Even a modest 10- to 20-pound weight loss can improve your levels, he said.

Additionally, regular physical activity is not only associated with better blood glucose levels but also can help reduce blood pressure.

Aim for at least 30 minutes of moderate-intensity aerobic exercise most days of the week and at least two strength-training sessions per week, he said.

Brantley also emphasized the importance of not smoking.

If you have diabetes, youre already at risk for heart and blood vessel problems. Nicotine only exacerbates the problem by damaging and tightening blood vessels, and raising blood pressure and blood glucose, he said.

I recommend that patients talk to their health-care provider and discuss specific risk factors for diabetic heart disease to come up with an individualized treatment plan, including further cardiac evaluation.

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Screening for thyroid dysfunction in patients without symptoms does not improve health and creates unnecessary expenses for the health-care system, according to a new guideline from the Canadian Task Force on Preventive Health Care.

Family doctors commonly order thyroid screening tests during routine patient visits. But the task force found no evidence of a benefit to patients in a systematic review of 22 studies on the effectiveness of treatment for abnormal thyroid stimulating hormone (TSH) levels in adults without symptoms. Moreover, harms of unnecessary testing include the need to take medication unnecessarily and have regular medical visits as well as follow-up blood tests to check TSH levels.

The message to family doctors: Stop routinely checking that box on blood test requisition forms for patients without symptoms or risk factors.

Family doctors thought they were helping the patient by hopefully identifying thyroid disorder early on and preventing negative health outcomes, saysDonna Reynolds, a member of the task force working group who is an assistant professor in the University of Torontos department offamily and community medicine and at theDalla Lana School of Public Health.

Now weve found that we werent necessarily helping the patient and were potentially causing harm not necessarily physical harms, but turning a person into a patient when they otherwise didnt have to be one.

The Canadian Medical Association Journal published the guideline this week.

Symptoms of thyroid disorder can be quite vague, so the relationship that doctors form with patients over time can give insight into whether certain symptoms are starting to look more like a pattern, says Reynolds, who is also a family physician at Scarborough Health Network.

The longer you practice, the more you start to see patients with similar complaints, and we put patients on a routine to make sure we dont miss anything, says Reynolds. I think thats good clinical care, but its also important to know if were over-treating and over-diagnosing people.

The Canadian Institute for Health Information and Choosing Wisely Canada have found that up to 30 per cent of medical tests, treatmentsand procedures in some areas of health care may be unnecessary.

Choosing Wisely Canada has introduced several measures to reduce over-testing, and a recent effort to limit thyroid tests proved successful in a U of Tstudy published last week. Associate ProfessorMichelle Greiver and other researchers in U of Ts department of family and community medicine led that work, which found a 13 per cent reduction in thyroid tests over two years among family health teams participating in the Choosing Wisely Canada effort. Thatscompared to a minor reduction among teams that did not participate.

The group estimated $1.5 million in yearly savings from the reduced number of thyroid tests.

Reynolds and other members of the task force emphasize that the new thyroid screening guideline is for adults with no symptoms or risk factors for thyroid dysfunction.

The guideline does not apply to pregnant women, those who have exhibited symptoms or have been diagnosed with thyroid dysfunction,those who have had thyroid surgery, those who are taking medications that can affect the thyroidor those undergoing radiotherapy that could affect the thyroid.

Its good to have a critical eye on what were doing and why were doing it, and to practise evidence-based medicine to ensure were doing the best for our patients, says Reynolds.

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New guideline, created with input from U of T researchers, calls for end to routine screening for thyroid disorders - News@UofT

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November 19, 2019 Cover

Pam Tillis keeps busy touring, making appearances and forever moving forward to her next musical project. Just last week she was a presenter for the Country Music Association awards with Women of Country Music as the theme.Country music is lucky to have her in their genre. But it wouldnt have mattered what Tillis chose to record and perform, she has one of those rare voices that lends itself to anything she wants to sing. She can easily move from classic country, to pop, to a bluesy torch singer wherever her heart, her soul and the lyrics lead.

Its rare when the puzzle pieces just seem to fit the first time a person opens the jigsaw box, but when Norm Stulz had the ability to make people laugh as early as the second grade in Detroit, there was no denying opening the comedy box was his lifes calling.Just a few years later in the seventh grade, he met the girl of his dreams and to this day, he and his wife Sharon, continue to build on a life together as two crazy kids in love. Laughter has been the glue for the relationship and the career path that has sustained Stulz for nearly 40 years.

Hosting a holiday dinner for your family is an undertaking in itself, but resorts are tasked with preparing the perfect menu for thousands of guests at multiple restaurants.Which items do guests want on the menu? How much food to order? When to start cooking? How many guests to prepare for? These are all questions the food and beverage departments must consider when planning for a holiday.

This time of year box stores are filled to the brim with every electronic device and latest phone known to man, but are there people on your list who already have all that stuff? Everyone has that one relative who is a challenge when comes to finding the perfect gift. Unique people require unique items and thinking outside the traditional box store offerings. Maybe that difficult-to-buy-for person is yourself because you never know what might strike your fancy.

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Intermittent fasting is a popular weight loss diet which is loaded with amazing health benefits other than weight loss. When you consume fewer calories you lose weight. Intermittent fasting also reduces your calorie consumption along with time management. It is a quite popular diet which involves a cycle of fasting and eating. Intermittent fasting makes you follow a protocol that helps you fast in the proper way. Intermittent fasting has been widely used for weight loss. Following this diet is associated with more health benefits. A recent study has highlighted that intermittent fasting may lead to a longer and healthier life.

Intermittent fasting may sound like another diet fad but researchers have conclusively found that the practice of routinely not eating and drinking for short periods of time resulted in longer life in heart patients.

"It's another example of how we're finding that regular fasting can lead to better health outcomes and longer lives," said Benjamin Horne, principal investigator of the study.

Fasting affects a person's levels of hemoglobin, red blood cell count, human growth hormone, and lowers sodium and bicarbonate levels, while also activating ketosis and autophagy - all factors that lead to better heart health and specifically reduce risk of heart failure and coronary heart disease.

Also read:Intermittent Fasting: Know How To Practice It And The Benefits And Drawbacks That Follow

Type-2 diabetes is the most common type of diabetes which is affecting a huge population. Intermittent fasting has shown improvement in insulin resistance which lowers the risk of type-2 diabetes.

Intermittent fasting can help you control the risk of type-2 diabetesPhoto Credit: iStock

Many studies have shown that intermittent fasting can help in naturally fighting inflammation. Inflammation is a part of many health conditions. Intermittent fasting also helps in reducing oxidative stress.

As per the study mentioned above, intermittent fasting can help in reducing the risk of heart diseases and it can help you promote a healthy heart. It controls various risk factors which can lead to heart diseases like high blood pressure, bad cholesterol levels, inflammation and many more.

Studies have shown that intermittent fasting can help in boosting brain health. It also reduces the risk of Alzheimer's.

Intermittent fasting also helps on controlling the risk of Alzheimer's and improve brain healthPhoto Credit: iStock

Also read:Intermittent Fasting: Is It A Healthy Way To Lose Weight? Find Out Here

Fasting is not for everyone. Researchers cautioned that pregnant and lactating women should not fast, as well as young children and frail older adults. People diagnosed with chronic diseases - especially those who take medications for diabetes, blood pressure, or heart disease - should not fast unless under the close care and supervision of a physician.

Also read:Not Just Weight Loss But Intermittent Fasting Can Also Reverse Type 2 Diabetes: Here's The Right Way To Follow It

(With inputs from IANS)

Disclaimer: This content including advice provides generic information only. It is in no way a substitute for qualified medical opinion. Always consult a specialist or your own doctor for more information. NDTV does not claim responsibility for this information.

Get Breaking news, live coverage, and Latest News from India and around the world on NDTV.com. Catch all the Live TV action on NDTV 24x7 and NDTV India. Like us on Facebook or follow us on Twitter and Instagram for latest news and live news updates.

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DUBLIN, Nov. 22, 2019 /PRNewswire/ -- The "Cell and Gene Therapy Market - Global Outlook and Forecast 2019-2024" report has been added to ResearchAndMarkets.com's offering.

The global cell and gene therapy market is growing at a CAGR of over 24% during the forecast period 2018-2024.

Key Market Insights

The major drivers contributing to the growth of the global cell and gene therapy market are the growing incidence of several chronic and terminal diseases, including cancer, the launch of new products, the increasing availability in clinical evidences of these products in terms of safety and efficacy, the rapid adoption of CAR T-cell therapy, favorable regulatory support in the development of these treatment, and improved manufacturing expertise in these products.

Market DynamicsMarket Growth Enablers

Market Growth Restraints

Market Opportunities and Trends

Cell and Gene Therapy Market: Segmentation

This research report includes detailed market segmentation by product, application, end-user, and geography.

The global cell therapy market is growing at a steady rate, and this trend is expected to continue during the forecast period due to the increased patient base with a wide range of diseases/ailments. The segment is likely to witness upward growth on account of expanded expertise in the manufacturing of stem cell-based products.

The gene therapy segment is expected to witness faster growth as the penetration of these products is increasing at a significant rate, especially in developed economies. The market is expected to grow during the forecast period due to the increased patient base for the existing gene remedy products, expected the launch of other gene therapy-based products for several indications, and expanded indication approvals for existing commercially available products.

The oncology segment accounts for the highest share of the global market. The growth of the oncology segment is increasing at a fast rate on account of the growing prevalence of several types of cancers. Currently, the available products not only modify the disease but also improve the quality of the patient's life, thereby decreasing the mortality rate. The market in the dermatology segment is increasing at a steady rate. This segment owns its growth to the increasing incidence and prevalence rate of several types of wounds, which are difficult to treat under normal conditions and the launch of innovative products. The dermatology segment is likely to showcase growth due to the high product availability of wound care products in the market.

Hospitals are the leading end-user segment. The segment is growing mainly due to the increasing incidence/prevalence of chronic diseases such as cancer, cardiovascular diseases, diabetes, and chronic wound on account of diabetes feet, pressure ulcers, and other injuries.

Market Segmentation by Products

Market Segmentation by Distribution Channel Type

Market Segmentation by End-users

Geographical Insights

The US market dominates the cell and gene therapy market in North America due to the high prevalence of chronic diseases and other conditions, which require these treatment methods. There is also comparably high utilization and wide accessibility of these therapies. The oncology segment is likely to witness significant growth in North America.

The market in Europe is expected to witness upward growth in the near future on account of the growing prevalence of chronic diseases and rising elderly population. In Europe, cell and gene therapy products are considered to be part of the Advanced Therapy Medicinal Products (ATMPs), which are commonly known as regenerative medicine globally.

Key Vendor Analysis

The global market is characterized by the presence of a few global, large-scale companies and several small to medium-scale companies offering one or two cell and gene therapy products. Global players are majorly offering innovative products with the potential of disease-modifying characteristics that are generating significant revenues, especially in Europe and US regions. Most innovative and breakthrough products are approved in the European countries and the US.

Vendors are targeting mostly developed economies such as the US, Germany, France, the UK, Spain, and Japan as the uptake of these products is higher in these countries than low and middle-income countries. However, the market in these regions is at the nascent stage.

Key Vendors

Other Prominent Vendors

For more information about this report visit https://www.researchandmarkets.com/r/oshmv7

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

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Worldwide Cell & Gene Therapy Market Insights Study, 2019-2024 - Market Set to Surpass a CAGR of 24%, Driven by Favorable Regulatory Support & Special...

Recommendation and review posted by Bethany Smith

Local and state officials gathered Friday in Alachua to commemorate a $6 million expansion at Progress Park.

Representatives of Thermo Fisher Scientific Inc., a global science company involved with research and biotechnology product development, presented the companys expanded gene therapy and viral vector services site. The day included tours of the 95,000-square-foot facility.

Chris Murphy, vice president and general manager of Thermo Fisher Viral Vector Services, said gene therapy provides genetic information to patients who lack it.

Ive been in this industry for over 20 years in gene therapy, and over the last three years, its completely transformed, he said. This site and this group and these folks here couldnt be better positioned to be part of that revolution.

Larry Pitcher, general manager and head of the Alachua site, said the company at its three-building location currently employs about 250 people who work with viral vectors to treat and potentially cure rare, complex diseases.

Key players in the more than decade-long development, who also spoke at the unveiling, included Greater Gainesville Chamber of Commerce Board Chair Bryan Harrington, the University of Floridas Assistance Vice President of Technology Commercialization Jim OConnell, state Sen. Keith Perry, and Alachua Mayor Gib Coerper.

OConnell noted that the research started 16 years ago at the University of Florida when Richard Snyder founded Florida Biologix, which changed to Brammer Bio through a merger with Brammer Biopharmaceuticals in 2016. Brammer Bio then merged with Thermo Fisher Scientific in May.

OConnell said a grant from state representatives originally kickstarted the initiative.

I think its critical to recognize these things dont happen overnight, he said.

Murphy said he first came to Progress Park 18 years ago. He highlighted the influence of the company merger and the scale of the industry.

(Its) been just a remarkable synergy and nothing but positiveness, and, frankly, the investment continues, he said. $270 million will be invested by pharma-services groups across the world.

Pitcher mentioned the continued growth of the Alachua site over the last two to three years and the increase of about 150 jobs.

Its really about the patient communities that we serve, Pitcher said, and getting these therapies to them as soon as possible.

The presentation concluded with Greater Gainesville President and CEO Eric Gobet gifting Thermo Fisher representatives with a key to the region from the Greater Gainesville Chamber.

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$6 Million Expansion For Gene Therapy Facility Completed At Alachua's Progress Park - WUFT

Recommendation and review posted by Bethany Smith

The central government has decided to support research on gene therapy in a big way to tackle Indias escalating burden of non-communicable diseases (NCDs) and inherited critical disorders such as spinal muscular atrophy (SMA), thalassemia and haemophilia. Scientific institutions and universities conducting research on genomics and its therapeutic applications will soon get a financial fillip from public coffers, under an initiative facilitated and funded by the Indian Council of Medical Research (ICMR).

Gene therapy is a technique that uses genetic modifications to treat or prevent diseases. The experimental technique allows clinicians to treat a disorder by inserting a gene into a patients cells instead of using drugs or surgery or replacing a mutated gene that causes diseases with a healthy copy of it.

According to the proposal note, a copy which CNBCTV18.com has reviewed, the funds will be disbursed for research projects across various therapeutic areas including gene editing, gene transfer techniques and immunotherapy based on genetic modifications. The apex body for biomedical research promotion has already started accepting proposals from scientists and clinicians.

Why gene therapy is crucial

In the current era of genomics and genetic testing, it is evident that a large swath of the Indian population harbours causative mutations leading to various disorders. Conversely, for many inherited and complex disorders, the underlying genetic causes are known.

And most of these illnesses are currently not treatable by molecule drugs or traditional therapies. According to patient advocacy groups, though there are major developments in gene therapy for cancer and other chronic diseases in the developed world, most of them are not accessible or affordable to a majority of the Indian population.

For instance, the US Food and Drug Administration (FDA) recently approved Zolgensma, the first gene therapy to treat children less than two years of age with SMA, a leading genetic cause of infant mortality. Though the drugs clinical trial in older SMA-affected children remains partially halted because of an inflammatory response observed in an animal study, there are very few options left for these patients.

Majority of these rare diseases, including SMA, are progressive and require life-long treatment and continuous support, says Archana Panda, co-founder of Cure SMA India, a non-profit group that works for making SMA treatment accessible to Indians.

In the case of SMA, a disease that leads to loss of motor neurons and early death, Nusinersen is the only drug approved by the US FDA. Developed by US-biotech major Biogen and marketed as Spinraza, the drug is administered directly to the central nervous system intravenously and the therapy could cost around $750,000 in the first year.

But in recent years, significant strides have been made in the field of gene therapy. This has culminated in the recent FDA approvals for Luxturna (gene therapy for retinitis pigmentosa) and Yescarta (CAR-T cell therapy for lymphoma). Many such approaches are currently under investigation or in early clinical trials. Now, the ICMR-backed initiative aims to fill the gaps in research thrust in India by providing emphasis on the focus areas which require more attention to address the needs for the large existing patient base, the note reads.

Hope on the horizon for patients

Apart from multi-factorial diseases such as cancer, diabetes and lung ailments, the research will also focus on treatment for rare diseases such as neuro-muscular diseases (including Duchenne muscular dystrophy, Becker muscular dystrophy, SMA and myopathies), retinal or corneal disorders (including Retinitis Pigmentosa, Stargardt disease and Fuchs dystrophy), inherited heart diseases and blood disorders including Thalassemia, Sickle Cell Disease and haemophilia.

The initiative assumes significance as it is estimated that one in 20 Indians is affected by one of the 7,000 diseases listed by the World Health Organisation as rare diseases. For many of these illnesses, gene therapy is the final hope. About 16 lakh Indians are diagnosed with cancer every year, and 8 lakh people lose their life to the disease.

While the fund support for research which can lead to human trials is only a baby step, experts such as Dr Mamta Muranjan, who heads the Genetic Clinic at KEM Hospital in Mumbai, emphasise the need for such focused long-term policies. Most patients with rare diseases remain undiagnosed for a long time. Even if the diagnosis is on time, the family of the patient is usually unable to afford the treatment cost. Insurance policies generally do not cover these life-long treatment expenses, she says.

Possibility of translation into human trials

According to official sources, the three-year state-funded research projects would be selected on the basis of their potential for development of functional treatment options. The strategies proposed should have the possibility of translation into future human trials.

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Govt to fund gene therapy research on NCDs, rare diseases - CNBCTV18

Recommendation and review posted by Bethany Smith

Timrepigene emparvovec is a potential first-in-class AAV2 gene therapy for the treatment of choroideremia, a rare, degenerative, X-linked retinal disorder that leads to blindness

CAMBRIDGE, Mass., Nov. 21, 2019 (GLOBE NEWSWIRE) -- Biogen Inc.(BIIB) announced today the enrollment of the last patient in the global Phase 3 STAR clinical study, which is evaluating the investigational gene therapy timrepigene emparvovec (BIIB111/AAV2-REP1) for the treatment of choroideremia (CHM). CHM is a rare, degenerative, X-linked inherited retinal disorder that leads to blindness.

We are excited to advance innovative investigational treatments for inherited retinal disorders that have significant unmet medical need due to the lack of treatment options, said Alfred Sandrock, Jr., M.D., Ph.D., Executive Vice President, Research and Development, and Chief Medical Officer at Biogen. Timrepigene emparvovec could be a transformative gene therapy for individuals living with choroideremia who would otherwise face inevitable blindness. Completing enrollment of our Phase 3 study represents a significant milestone in bringing this new potential therapy to patients.

STAR is a randomized, masked, prospective, parallel-controlled group Phase 3 study that enrolled 170 adult males with CHM. The study is evaluating the safety and efficacy of a single subretinal injection of timrepigene emparvovec. The primary endpoint is the proportion of patients with an improvement of at least 15 letters from baseline in best corrected visual acuity (BCVA) at 12 months post treatment as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity protocol. The STAR study was initiated based on proof-of-concept data from Phase 1/2 studies, which indicated that at month 24, over 90 percent of patients treated with timrepigene emparvovec via targeted subretinal injection maintained visual acuity instead of experiencing the natural decline in BCVA expected in this degenerative disease. In a subset of treated patients with moderate to severe visual acuity loss, 21 percent experienced a gain in visual acuity of at least 15 ETDRS letters from baseline as compared to one percent of untreated patients in a natural history study.

CHM primarily affects males and is caused by a loss of function in the CHM gene which encodes the Rab escort protein-1 (REP-1). The REP-1 protein plays a role in intracellular protein trafficking, and the loss of function in the CHM gene leads to abnormal intracellular protein trafficking and impaired elimination of waste products from the retinal pigment epithelium and photoreceptors. Initially, patients with CHM experience poor night vision and over time, progressive visual loss ultimately leads to blindness.

Biogen added timrepigene emparvovec to its portfolio in June 2019 as part of its acquisition of Nightstar Therapeutics.

For more information about the Phase 3 STAR study, visit http://www.clinicaltrials.gov (NCT03496012).

About timrepigene emparvovec (BIIB111/AAV2-REP1)Timrepigene emparvovec is an AAV2 vector administered by subretinal injection, which aims to provide a functioning CHM gene and expression of the REP-1 protein to restore membrane trafficking and thereby slow, stop or potentially reverse decline in vision. Data from the Phase 1/2 studies demonstrated a slower rate of decline in visual acuity in patients treated with timrepigene emparvovec compared to untreated patients in the natural history study. In addition, some patients treated with timrepigene emparvovec showed improvements in visual acuity. The studies also demonstrated that timrepigene emparvovec was generally well tolerated with an acceptable safety profile.

Timrepigene emparvovec has received regenerative medicine advanced therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA), which includes all of the benefits of the fast track and breakthrough therapy designation programs and orphan drug designations in the U.S., Europe and Japan. The safety and efficacy of a single subretinal injection of timrepigene emparvovec is currently being evaluated in the ongoing Phase 3 STAR study.

About Biogen At Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological and neurodegenerative diseases as well as related therapeutic adjacencies. One of the worlds first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Kenneth Murray, and Nobel Prize winners Walter Gilbert and Phillip Sharp. Today Biogen has the leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, commercializes biosimilars of advanced biologics, and is focused on advancing research programs in multiple sclerosis and neuroimmunology, neuromuscular disorders, movement disorders, Alzheimers disease and dementia, ophthalmology, immunology, neurocognitive disorders, acute neurology, and pain.

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We routinely post information that may be important to investors on our website atwww.biogen.com. To learn more, please visitwww.biogen.comand follow us on social media Twitter,LinkedIn,Facebook,YouTube.

Biogen Safe Harbor StatementThis news release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, relating to the potential, benefits, safety and efficacy of timrepigene emparvovec; the potential clinical effects of timrepigene emparvovec; results from the Phase 1/2 studies of timrepigene emparvovec; the clinical development program for timrepigene emparvovec; the treatment of CHM; the potential of our commercial business and pipeline programs, including timrepigene emparvovec; and risks and uncertainties associated with drug development and commercialization. These forward-looking statements may be accompanied by words such as aim, anticipate, believe, could, estimate, expect, forecast, intend, may, plan, potential, possible, will, would and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk and only a small number of research and development programs result in commercialization of a product. Results in early stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including without limitation, uncertainty of success in the development and potential commercialization of timrepigene emparvovec; unexpected concerns may arise from additional data, analysis or results obtained during the STAR study; regulatory authorities may require additional information or further studies, or may fail or refuse to approve or may delay approval of our drug candidates, including timrepigene emparvovec; the occurrence of adverse safety events; the risks of other unexpected hurdles, costs or delays; failure to protect and enforce our data, intellectual property and other proprietary rights and uncertainties relating to intellectual property claims and challenges; and product liability claims. The foregoing sets forth many, but not all, of the factors that could cause actual results to differ from our expectations in any forward-looking statement. Investors should consider this cautionary statement, as well as the risk factors identified in our most recent annual or quarterly report and in other reports we have filed with the U.S. Securities and Exchange Commission. These statements are based on our current beliefs and expectations and speak only as of the date of this news release. We do not undertake any obligation to publicly update any forward-looking statements, whether as a result of new information, future developments or otherwise.

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Biogen Announces Enrollment Completion of Global Phase 3 Gene Therapy Study for an Inherited Retinal Disorder - Yahoo Finance

Recommendation and review posted by Bethany Smith

MANHATTAN, Kan., Nov. 21, 2019 /PRNewswire/ -- Libella Gene Therapeutics, LLC("Libella") announces an institutional review board (IRB)-approved pay-to-play clinical trial in Colombia (South America) using gene therapy that aims to treat and ultimately cure aging. This could lead to Libella offering the world's only treatment to cure and reverse aging by 20 years.

Under Libella's pay-to-play model, trial participants will be enrolled in their country of origin after paying$1 million. Participants will travel to Colombia to sign their informed consent and to receive the Libella gene therapy under a strictly controlled hospital environment.

Traditionally, aging has been viewed as a natural process. This view has shifted, and now scientists believe that aging should be seen as a disease. The research in this field has led to the belief that the kingpin of aging in humans is the shortening of our telomeres.

Telomeres are the body's biological clock. Every time a cell divides, telomeres shorten, and our cells become less efficient at dividing again. This is why we age. A significant number of scientific peer-reviewed studies have confirmed this. Some of these studies have shown actual age reversal in every way imaginable simply by lengthening telomeres.

Bill Andrews, Ph.D., Libella's Chief Scientific Officer, has developed a gene therapy that aims to lengthen telomeres. Dr. Andrew's gene therapy delivery system has been demonstrated as safe with minimal adverse reactions in about 200 clinical trials. Dr. Andrews led the research at Geron Corporation over 20 years ago that initially discovered human telomerase and was part of the team that led the initial experiments related to telomerase induction and cancer.

Telomerase gene therapy in mice delays aging and increases longevity. Libella's clinical trial involves a new gene-therapy using a proprietary AAV Reverse (hTERT) Transcriptase enzyme and aims to lengthen telomeres. Libella believes that lengthening telomeres is the key to treating and possibly curing aging.

Libella's clinical trial has been posted at the United States National Library of Medicine (NLM)'s clinicaltrials.gov database. Libella is the world's first and only gene therapy company with a clinical trial posted at clinicaltrials.gov that aims to reverse the condition of aging.

On why they decided to conduct its project outside the United States, Libella's President, Dr. Jeff Mathis, said, "Traditional clinical trials in the U.S. can take years and millions, or even billions,of dollars. The research and techniques that have been proven to work are ready now. We believe we have the scientist, the technology, the physicians, and the lab partners that are necessary to get this trial done faster and at a lower cost in Colombia."

Media Contact:Osvaldo R. Martinez-ClarkPhone: +1 (786) 471-7814Email: ozclark@libellagt.com

Related Files

curing_aging_booklet.pdf

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william-bill-andrews-ph-d.jpg William (Bill) Andrews, Ph.D. Dr. Bill Andrews is a scientist who has spent his entire life trying to defeat the processes that cause us to age. He has been featured in Popular Science, The Today Show, and numerous documentaries on the topic of life extension including The Immortalists documentary.

Related Links

Dr. Bill Andrews speech at RAADfest 2018 (Sept 21, San Diego, CA)

bioaccess: Libella's CRO partner in Colombia.

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Breakthrough Gene Therapy Clinical Trial is the World's First That Aims to Reverse 20 Years of Aging in Humans - PRNewswire

Recommendation and review posted by Bethany Smith

Dive Brief:

Fujifilm is looking to take advantage of the gene therapy market which, for CDMOs, is expected to reach $1.7 billion by 2025, according to Fujifilm Diosynth Biotechnologies' market research.

"We are very much aware of the incredible growth in such an important therapeutic space," Martin Meeson, president and chief operating officer of FDB U.S., said in a Nov. 14 statement. "We know that we need to invest now, in technology, assets and people in order to achieve a market leadership position."

The new Gene Therapy Innovation Center on the existing FDB campus in Texas will be about 60,000 square feet and operational starting in fall 2021. FDB expects the center to triple its gene therapy development capabilities and add about 100 jobs.

Meanwhile, the first stage of the expansion for the existing manufacturing facility should be complete by the spring of 2021, Fujifilm said.

The Japan-based company's fresh investments in gene therapy come amid a wave of expansions, mergers and partnerships in the growing field.

This month, Swiss manufacturer Lonza announced a new partnership with cold chain specialist Cryoport as part of its goal to provide a seamless "vein-to-vein" network in cell and gene therapy. Lonza also opened a 300,000-square-foot plant last year in Texas dedicated to manufacturing the therapies.

Catalent earlier this year bought Paragon Bioservices for $1.2 billion to strengthen its position as a CDMO of gene therapies. And Thermo Fisher acquired viral vector manufacturer Brammer Bio for $1.7 billion.

Fujifilm forecasts sales of 100 billion yen for its CDMO business in the fiscal year ending March 2022.

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Fujifilm to invest $120M in gene therapy, build center in Texas - BioPharma Dive

Recommendation and review posted by Bethany Smith

Hertfordshire Local Enterprise Partnership is to invest 1.2m into Stevenage Bioscience Catalyst (SBC) to help grow a world-class cell and gene therapy cluster.

The 1.2m Local Growth Fund investment will provide much-needed temporary accommodation for growing cell and gene therapy companies, strengthening the UKs sectorial advantage and Hertfordshires position within the Oxford-London-Cambridge golden research triangle.

SBC is a world-leading science park of global significance and home to the largest cluster of cell and gene therapy companies in Europe. Since its opening in 2012, it has attracted over 40 high-profile occupier businesses and world-class research organisations to Stevenage.

This includes the Cell and Gene Therapy Catapult Manufacturing Centre which already supports five companies developing their manufacturing at scale and is currently undergoing expansion to help support up to 12 cell and gene therapy companies.

With both SBC and the Cell and Gene Therapy Catapults first large scale manufacturing centre on the campus, companies can benefit from close proximity to manufacturing, development and research. This unique co-location has been a key driver of growth in this exciting and emerging treatment area.

Occupiers of the SBC campus have raised almost 1bn of investment, of which around 60% has been invested into cell and gene therapy companies. Now several of these companies are wanting to expand their premises at SBC.

To help meet this demand, Hertfordshire LEP has granted 1.2m capital contribution to the development of the new Spark Building. This will provide interim lab and office accommodation whilst more permanent accommodation is developed nearby. The Spark Building, due for completion by the end of 2019, will be co-located with the SBC Incubator, Accelerator and the Cell and Gene Therapy Catapult Manufacturing Centre.

Dr Sally Ann Forsyth, Chief Executive Officer at Stevenage Bioscience Catalyst, commented: We are delighted to receive the investment and continued support from Hertfordshire LEP. This ensures that we support the growth of our occupiers and continue to build on our position as Europes hub for cell and gene therapy.

Paul Witcombe, Head of Enterprise and Innovation, Hertfordshire LEP, commented: Stevenage Bioscience Catalyst has made a huge contribution to the rapid clustering of world-class corporate and academic research excellence on the GSK campus at Stevenage. To date, we have already approved up to 12m from the Local Growth Fund to develop essential infrastructure to support scientific research and we anticipate further opportunities to support the cluster.

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1.2m investment in Stevenage Bioscience Catalyst for world-class cell and gene therapy - Pharmafield

Recommendation and review posted by Bethany Smith

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The worlds first breakthrough gene therapy clinical trial aims to reverse 20 years of aging in humans.

USA: Using genetherapy to express active telomerase (hTERT) in humans has the potential to treat many of the age-related diseases, including aging itself. Now, Libella Gene Therapeutics, has set up a pay-to-play clinical trial inColombia(South America) that aims to use the gene therapy to reverse aging by 20 years. If successful, the therapy will become the worlds only treatment to treat and cure aging.

The trial, approved by Libellas institutional review board (IRB), will be a pay-to-play model wherein volunteers have to pay $1m for enrollment in their country. Participants will then travel to Colombia to sign their consent. Gene therapy will be administered to participants under a controlled hospital setting.

................................ Advertisement ................................

Aging has been viewed as a natural aging process. But now, scientists believe aging to be a disease. Further research in this field has led to the belief that aging in humans is primarily caused by the shortening of telomeres caps at the end of each DNA strand that protects the end of the chromosome. Every time a cell divides telomeres shorten and the cells become less efficient at dividing again resulting in aging. This has been confirmed by a significant number of scientific peer-reviewed studies. Some of these studies have shown actual age reversal in every way imaginable simply by lengthening telomeres.

................................ Advertisement ................................

Bill Andrews, Libellas Chief Scientific Officer, has developed a gene therapy that aims to lengthen telomeres. Dr. Andrews gene therapy delivery system has been demonstrated as safe with minimal adverse reactions in about 200 clinical trials. Dr. Andrews led the research at Geron Corporation over 20 years ago that initially discovered human telomerase and was part of the team that led the initial experiments related to telomerase induction and cancer.

Telomerase gene therapy in mice delays aging and increases longevity. Libellas clinical trial involves a new gene-therapy using a proprietary AAV Reverse (hTERT) Transcriptase enzyme and aims to lengthen telomeres. Libella believes that lengthening telomeres is the key to treating and possibly curing aging.

In the Phase I trial, the gene therapy will be evaluated for its safety and tolerability in approximately five participants. It is expected to be completed in 2021.

The primary endpoint is the incidence of adverse events, while secondary outcomes are hTERT expression and telomerase activity.

................................ Advertisement ................................

For more information visit: Libella Gene Therapeutics

Medha Baranwal joined Medical Dialogues as an Editor in 2018 for Speciality Medical Dialogues. She covers several medical specialties including Cardiac Sciences, Dentistry, Diabetes and Endo, Diagnostics, ENT, Gastroenterology, Neurosciences, and Radiology. She has completed her Bachelors in Biomedical Sciences from DU and then pursued Masters in Biotechnology from Amity University. She has a working experience of 5 years in the field of medical research writing, scientific writing, content writing, and content management. She can be contacted atmedha@medicaldialogues.in. Contact no. 011-43720751

To know about our editorial teamclick here

Source: Libella Gene Therapeutics

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Gene therapy to reverse age by 20 years: World's first trial - Specialty Medical Dialogues

Recommendation and review posted by Bethany Smith

The report onGene Therapy Industryhas been published by Qurate Business Intelligence and it assesses the complex terrain of the Industry. Primary and secondary exploratory techniques have been used to analyze the data effectively. The competitive landscape of Gene TherapyIndustry companies has been studied to understand the rival products and services across the globe. For effective global regional outlook analysts of the report examine North America, Latin America, Japan, Asia-Pacific, and India on the basis of productivity, manufacturing base and raw material. Leading industry key players have been analyzed on the basis of revenue, productivity, applications and end users to give a complete view about Gene TherapyIndustry factors. SWOT and Porters Five Analysis have been used while curating this report. The entire demand and supply chain of Industry has been presented. A detailed description on the requirements of global as well as domestic clients has also been provided.

Get Free Sample PDF Copy of Gene Therapy Industry Research Report:https://www.qurateresearch.com/report/sample/HnM/global-gene-therapy-market/QBI-360ir-HnM-504284

Top Leading Manufactures Studied in Gene Therapy Industry: Audentes Therapeutics, Editas Medicine, Adaptimmune, Spark Therapeutics, Abeona Therapeutics, Inc.,, Bluebird bio, Inc., Merck & Co., Inc, Gilead, Achieve Life Sciences, Inc., Novartis, AGTC, Orchard Therapeutics, and Biogen.

GlobalGene Therapy Industrystatistics and figures are represented in a concise manner in the form of tables, pie charts, reference diagrams. The top industry players with their Industry share, development prospects, growth graph, and production rate in Gene Therapy are analyzed.

Gene Therapy Industry Research Goals:

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The research is based on varied secondary and primary data sources. The primary sources include access companys annual reports, product literature, government releases, industry magazines, paid sources and government magazines. Gathered data is verified by conducting paid primary interviews with industry experts.

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Gene Therapy Industry Analysis by Growth, Competitive Strategies and Forecast Research Report 2019-2025 - Markets Gazette 24

Recommendation and review posted by Bethany Smith

The legendary synthetic biologist Dr. George Church and team at the Wyss Institute at Harvard University took a first steptowards cracking the ultimate question of anti-aging research. They combined three gene therapies, each linked to a health problem associated with aging, into a single vaccine-like shot and gave it to ailing mice. The combination treatment reversed diabetes and obesity while improving heart and kidney functioneven when those organs had already begun failing.

If you hit enough specific diseases, youre getting at the core aging components that are common to all of them,saidChurch.

Rather than genetically modifying the mice, the team used a virus to encode genetic material that fine-tunes the activity of all three genes, but leaves the genome alone. In this way, the team explained, the combination therapy is far more easily applicable to humans in the long run.

Gene therapy gives you a testable therapy at scale in mice. And we can move from mice to dogs and then to humans. Were focusing on the reversal of age-related diseases so well be more healthy and youthful later in life, said Church.

Read full, original post: How Much Can We Delay Aging? A Gene Therapy Trial Is About to Find Out

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Trio of gene therapies seeks to reverse age-related diseases to make us 'healthy, youthful later in life' - Genetic Literacy Project

Recommendation and review posted by Bethany Smith

The University of Sheffield has been awarded the Queens Anniversary Prize for innovation in neuroscienceThe prize is the highest national honour that recognises outstanding work by UK universities and colleges that demonstrate quality and innovation in their researchSheffield has been recognised for improving patient outcomes for people living with neurodegenerative diseases such as Parkinsons Disease and Motor Neurone DiseaseNeuroscience research at the University of Sheffield has been recognised by Her Majesty The Queen for delivering real benefits in improving patient outcomes for people living with some of the most devastating neurodegenerative diseases.

The Sheffield Institute for Translational Neuroscience (SITraN) based at the University of Sheffield was awarded a Queens Anniversary Prize today (21 November 2019) at St Jamess Palace.

The prize awarded to SITraN is unique in the honours system and only bestowed upon a UK college or university which demonstrates new and innovative approaches to its research and development that have delivered benefits to the public at local, national and global levels.

SITraNs vision is to harness the rapidly emerging, exciting developments in neuroscience to translate into new treatments and improved quality of life for patients with neurodegenerative disorders such as Parkinsons Disease, Motor Neurone Disease (MND), Dementia and Alzheimers Disease and Multiple Sclerosis (MS).

Achievements highlighted by the award include:

a new orthotic device, HeadUp, for patients living with MND who suffer from muscle weakness in their neckground-breaking clinical stem cell clinical trials for MS patientsresearch which has improved the life-expectancy and quality of life for those living with MNDthe discovery of a biomarker linked to the development of Alzheimers Disease for the first time, which has the potential for earlier diagnosis and has sparked the development of new therapiesdrug discovery programmes to develop new treatments for Parkinsons Diseasenew gene therapy experimental medicine studies for MND which are showing promising early resultsProfessor Dame Pamela Shaw, Director of SITraN, said: Receiving this award is a great honour. It gives recognition to our research teams who have made enormous scientific progress in treating some of the most devastating neurological diseases, making a real difference to patients lives.

We hope that this award will inspire confidence for patients and their families, research partners and donors as we continue to make discoveries that deepen the understanding of neurological diseases and open up the potential for new treatments and therapies.

SITraN which will celebrate its 10th anniversary in 2020 is considered a world-leader in neuroscience research. Its work forms part of the University of Sheffields Neuroscience Institute, which aims to bring academics together from across varied specialties to translate scientific discoveries from the lab into pioneering treatments that will benefit people living with neurodegenerative diseases.

Professor Koen Lamberts, President and Vice-Chancellor of the University of Sheffield, said: Its wonderful to see the Queens Anniversary Award recognising the University of Sheffield as a centre for excellence in neuroscience research and teaching which has the power to transform peoples lives.

As well as making life-changing discoveries today, SITraN is nurturing the next generation of talented neuroscience students, whose research will lead to pioneering treatments for those living with neurological diseases in the future.

Chair of the Royal Anniversary Trust, Sir Damon Buffini, said: The prizes are granted every two years by the Queen and are the most prestigious national honour awarded to UK universities and colleges for their work.

Entries in the scheme are invited in any subject area and are subjected to rigorous independent assessment in a process managed by the Royal Anniversary Trust. Recommendations for the Queens approval are made on the Prime Ministers advice.

The criteria are demanding and look for outstanding excellence in the chosen field, for innovation and for evidence of real public benefit. Competition is strong and the award is a mark of high quality in education and training which is widely recognised internationally as well as in the UK.

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Royal recognition for neuroscience research at the University of Sheffield - India Education Diary

Recommendation and review posted by Bethany Smith

Global Sandhoff disease treatment market is growing at a steady CAGR in the forecast period of 2019-2026. The report contains data of the base year 2018 and historic year 2017. This rise in market value can be attributed to the orphan drug designation to novel drugs, along with the increasing investment of biotechnology and pharmaceutical industries in R&D.

This Sandhoff Disease Treatment report provides complete analysis of the market on global and regional level. It inspects the development rate and the market value dependent on the market elements and growth initiating factors. The market players are profiled and their improvement strategies are separated so as to manage new participants as well as set up players. It likewise features the top to bottom investigation of different unequivocal parameters. This Sandhoff Disease Treatment report also offers various methodologies for boosting the presentation of the organizations.

Overall overview of the market with estimated market size by production, application, market share, market growth and region is included in this Sandhoff Disease Treatment research report. It also provides market trends, market dynamics and grasp the recent technological advancements.

Sandhoff disease is also known as Beta-hexosaminidase-beta-subunit deficiency is a fatal pediatric lysosomal storage genetic disorder characterized by progressively destruction of neuron in the brain and spinal cord. It is caused by defects in HEXB gene which is responsible for regulation of vital enzyme called beta-hexosaminidase, as a result of accumulation of lipid called G2 gangliosides. This ongoing accumulation of lipid affects the function of the nerve cells and causes other neurological problem.

This Sandhoff Disease Treatment report provided segmentation of the market on the basis of the application, it focuses on the status and outlook for major applications, market share and growth rate of each player is included in this section.

By Types

ByTherapy

By Treatment

By Drugs

ByRoute of Administration

By Distribution Channel

By End-Users

This Sandhoff Disease Treatment report splits the market into different regions such as

The Market is analyzed using various different research methodologies like Porters Five Force Analysis, SWOT analysis, Pestle Analysis, Industry value chain analysis, Supply chain analysis through which the drivers, restraints and threats of the market is analyzed in this Sandhoff Disease Treatment research report.

This Sandhoff Disease Treatment research report provided analysis of the competitive landscape in the market and keeps focus on the key players, their economic situation and business strategies are examined to succeed in the market.

The key market players in the Sandhoff disease treatment market are Intrabio, Axovant Gene Therapies Ltd among others

Table of Content:

Part 01: Executive Summary

Part 02: Scope of the Report

Part 03: Research Methodology

Part 04: Sandhoff Disease Treatment Market Landscape

Part 05: Market Sizing

Part 06: Customer Landscape

Part 07: Sandhoff Disease Treatment Market Regional Landscape

Part 08: Decision Framework

Part 09: Drivers And Challenges

Part 10: Sandhoff Disease Treatment Market Trends

Part 11: Vendor Landscape

Queries Resolved In This Report

Download table of Contents with Figures & Tables @https://www.databridgemarketresearch.com/toc/?dbmr=global-sandhoff-disease-treatment-market

Contact:

Data Bridge Market Research

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UK: +44 208 089 1725

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Email:[emailprotected]

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Comprehensive Study Reveals How Sandhoff Disease Treatment Market is Trending | Intrabio, Axovant Gene Therapies Ltd - VaporBlash

Recommendation and review posted by Bethany Smith

ALPHARETTA Nov 22, 2019 (Thomson StreetEvents) -- Edited Transcript of Clearside Biomedical Inc earnings conference call or presentation Wednesday, November 6, 2019 at 9:30:00pm GMT

* Charles A. Deignan

Clearside Biomedical, Inc. - CFO

* George M. Lasezkay

Clearside Biomedical, Inc. - Interim CEO & Director

* Jenny R. Kobin

Clearside Biomedical, Inc. - Head of IR

* Thomas A. Ciulla

Clearside Biomedical, Inc. - Chief Medical Officer

Greetings, and welcome to the Clearside Biomedical Third Quarter 2019 Financial Results and Corporate Update Conference Call. As a reminder, this conference call is being recorded. I would now like to introduce your host, Jenny Kobin, Clearside Investor Relations. Please go ahead.

Jenny R. Kobin, Clearside Biomedical, Inc. - Head of IR [2]

Good afternoon, everyone, and thank you for joining us on the call today.

Before we begin, I would like to remind you that during today's call, we will be making certain forward-looking statements. Various remarks that we make during this call about the company's future expectations, plans and prospects constitute forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in the Risk Factors section of our annual report on Form 10-K for the year ended December 31, 2018, our quarterly report on Form 10-Q for the quarter ended June 30, 2019, and our other SEC filings available on our website.

In addition, any forward-looking statements represent our views as of today and should not be relied upon as representing our views as of any subsequent date. While we may elect to update these forward-looking statements in the future, we specifically disclaim any obligation to do so even if our views change.

On today's call, George Lasezkay, our Chief Executive Officer and member of the Board of Directors, will provide a strategic update; Dr. Thomas Ciulla, our Chief Medical Officer, will provide R&D highlights; and Charlie Deignan, our Chief Financial Officer, will provide a financial summary. We will then open the call for your questions. Brion Raymond, our Chief Commercial Officer, is also with us today and available for Q&A.

I would now like to turn the call over to George.

George M. Lasezkay, Clearside Biomedical, Inc. - Interim CEO & Director [3]

Thank you, Jenny. Good afternoon, everyone, and thank you for joining us on the call today. I'm pleased to report that we have made meaningful progress on our overall corporate strategy to broaden the reach of our suprachoroidal space injection platform. A component of this strategic plan is the establishment of key external collaborations.

Over the last 3 months, we have secured 3 significant partnerships that validate and will potentially expand the reach of our suprachoroidal injection platform. Two weeks ago, we announced that Bausch Health and its Ophthalmic division, Bausch + Lomb, acquired an exclusive license for the commercialization and development of XIPERE in the United States and Canada. As a reminder, XIPERE is our proprietary suspension of triamcinolone administered suprachoroidally with our SCS Microinjector for the treatment of macular edema associated with uveitis. We are thrilled to have licensed XIPERE to such a high-quality organization with a well-established and well-regarded presence in the ophthalmology community.

Bausch has the right to pursue development and commercialization of XIPERE for additional ophthalmic indications. And they also have the right to develop and commercialize our proprietary SCS Microinjector in combination with certain other specified corticosteroids and nonsteroidal anti-inflammatory drugs in the field of ophthalmology.

Licensing XIPERE to Bausch has achieved our primary corporate goal of finding a partner with an experienced ophthalmic sales force that can bring XIPERE to market more efficiently and cost effectively if approved. They also have the resources to potentially develop additional indications for XIPERE. We believe this is a win for patients and for Bausch and also a win for Clearside as validation of the potential benefits of our proprietary method of accessing the suprachoroidal space.

Also regarding XIPERE, as expected, last month, we received a complete response letter or CRL from the FDA. Consistent with the outcome of the meeting we had with the FDA in August, the agency requested additional stability data on our triamcinolone suspension. The CRL also included one new request for additional data on clinical use of the final to-be-marketed SCS Microinjector delivery system. Importantly, the FDA did not identify any efficacy issues. And there were no requests for further clinical efficacy studies. We currently believe we can readily address the issues raised by the FDA and resubmit our New Drug Application in the first quarter of next year. Tom will elaborate on the details of our planned NDA resubmission in his remarks.

In September, we announced an option and license agreement with REGENXBIO for exclusive worldwide rights to our proprietary in-office SCS Microinjector for the delivery of adeno-associated virus or AAV-based therapeutics to the suprachoroidal space to treat wet AMD, diabetic retinopathy and other conditions for which anti-VEGF treatment is currently the standard of care.

We are very pleased that REGENXBIO exercised their option last week. This is an exciting time for us to collaborate with REGENXBIO, one of the leaders in gene therapy field to evaluate the potential application of our injection platform for AAV ophthalmic gene therapy. We believe the delivery of gene therapy through the suprachoroidal space can potentially provide a targeted in-office nonsurgical approach to treat patients with challenging retinal conditions.

Finally, in July, we announced that Aura Biosciences licensed our SCS Microinjector to deliver their proprietary drug candidates into the suprachoroidal space for the potential treatment of ocular cancers. This is a therapeutic area where there is a significant unmet medical need. Based on their recent public comments, we expect Aura to submit an IND amendment and initiate a clinical trial using our SCS Microinjector in the first half of next year.

Our research and development team has also made progress on earlier-stage research projects. Our internal initiatives are focused on gene therapy delivery by nonviral DNA nanoparticles as well as small molecules that may show prolonged duration, utilizing the SCS injection platform to address unmet needs in the back-of-the-eye diseases. Based on a fresh analysis of our prior data and recently presented data in the scientific community, we have decided to advance as our next development asset, our proprietary suspension of axitinib for suprachoroidal injection, which we refer to as CLS-AX. We are targeting submission of an investigational New Drug Application in mid-2020. Tom will discuss this latest development in more detail in his remarks.

The strategic shifts that we embarked on earlier this year has benefited Clearside in a number of ways. We have eliminated the inherent risks and financial investment related to building and maintaining a commercial infrastructure for XIPERE ourselves while retaining a significant financial upside in its potential commercial success. Our recent collaborations have validated our investment in suprachoroidal delivery using our SCS Microinjector. And as a result, we have expanded our overall internal and collaborative product development pipeline to include both gene therapy and small molecule opportunities, targeting a broad range of additional potential ophthalmic indications beyond uveitis to include choroidal melanoma, wet AMD and diabetic retinopathy.

We are entitled now to receive $7 million of nondilutive capital in upfront payments this year. And finally, we are eligible to receive over $200 million in potential future development and sales milestones and have the opportunity to receive additional sales royalties from all 3 partnering deals, which may be used to continue to fund our internal R&D pipeline projects.

I will now turn over the call to Tom Ciulla, our Chief Medical Officer.

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Thomas A. Ciulla, Clearside Biomedical, Inc. - Chief Medical Officer [4]

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Thank you, George. This afternoon, I'll provide a summary of clinical and scientific information related to our XIPERE NDA resubmission, our REGENXBIO partnership and our internal R&D progress.

As background on XIPERE, it's helpful to understand the delivery approach. Suprachoroidal injection is a novel drug-dispensing approach that employs proprietary piston syringe and a 30-gauge needle about 1 millimeter in length. The suprachoroidal injection procedure allows drugs to be administered into the transition region between the choroid and the sclera called the suprachoroidal space. Suprachoroidal injection provides almost direct drug access to the retina, the retinal pigment epithelial cells and the choroid. Over the course of the development life cycle for XIPERE, Clearside made quality enhancements to the drug product manufacturing process. While the formulation of the triamcinolone acetonide suspension has not changed, the FDA wants to verify the stability profile between the batches we submitted and the intended commercial product to ensure that the process enhancements have not affected the drug product.

During our meeting with the FDA in August, the agency provided clear guidance on the chemistry, manufacturing and controls or CMC data to be included in the NDA resubmission. We've been working closely with our contract manufacturer to produce the required material and to obtain the requested stability data.

The CRL also included a request for additional data on clinical use of the final to-be-marketed SCS Microinjector delivery system. We currently expect that this device use assessment will be conducted by at least 3 physicians in 30 patients per the FDA's recommendation. We believe that this type of assessment can be conducted in a quick and streamlined approach with clinicians who have worked with us before and who are experienced in using the device.

Currently, over 1,000 suprachoroidal injections of XIPERE have been performed across multiple clinical studies and multiple diseases without any significant adverse events related to the injector. And as George mentioned in the CRL, the FDA did not identify any efficacy issues, and there were no request for further clinical efficacy studies. We remain confident in the results of our clinical trials and the potential future approval of XIPERE.

Our CMC, clinical and regulatory teams are working diligently to satisfy these requests in the CRL, and we expect to have a formal meeting with the FDA before the end of the year to discuss our plans for this device assessment. Once we confirm our plans with the agency, we will prepare for and generate the requested data and expect to resubmit the NDA in the first quarter of next year. We believe the FDA will review the NDA resubmission within 6 months of the receipt date.

Next, our partnership with REGENXBIO further validates our suprachoroidal approach and the potential benefit in the growing gene therapy field. Dr. Peter Campochiaro's team at Johns Hopkins recently demonstrated in preclinical studies that AAV vector suprachoroidal gene transfer can produce widespread ocular transgene expression in several species.

Compared to subretinal injection in rats, suprachoroidal administration of REGENXBIO's asset, RGX-314, resulted in similar expression of anti-VEGF therapeutic protein and similar suppression of VEGF-induced vascular leakage. Likewise, our own preclinical studies of nonviral DNA nanoparticle gene therapy showed similar activities of a marker team when administered subretinally or suprachoroidally. We are excited to have the opportunity to collaborate with REGENXBIO to evaluate the application of our proprietary SCS microinjector for AAV gene therapy with the hope of offering patients nonsurgical, in-office access to onetime gene therapy treatment.

Internally, our discovery and R&D efforts have been primarily focused on performing nonclinical experiments around gene therapy and small molecules. We are leveraging our learnings from the XIPERE development program, which demonstrated that suprachoroidal delivery of small molecule drug suspensions can target effective choroidal retinal tissues with potential for enhanced clinical efficacy and prolonged durability.

As part of our strategic shift, we have reviewed our internal assets to determine how we should proceed now that XIPERE has been successfully partnered. After careful evaluation of our prior work and recently presented data in the scientific community, we have determined that our suspension of axitinib for suprachoroidal injection, CLS-AX, represents a very compelling opportunity.

With current wet AMD therapy, there is a ceiling of efficacy as increased dosage of more intense regimens yield no additional visual benefit. Axitinib is currently approved to treat renal cell cancer, and with its broad VEGF blockade, it may have efficacy advantages over existing retinal therapies, which predominantly focus on VEGF blockade and may up-regulate other forms of VEGF. Axitinib achieves pan-VEGF blockade by acting at a different level of the angiogenesis cascade, directly inhibiting VEGF receptors-1, -2 and -3 with high potency and high specificity. Axitinib has been shown to effectively inhibit corneal, retinal and choroidal angiogenesis in multiple animal models by independent investigators.

In one of these studies, axitinib was shown to be more effectively inhibit experimental corneal neovascularization than other tyrosine kinase receptor inhibitors. In another study, axitinib showed better biocompatibility with ocular cells than other tyrosine kinase inhibitors. In addition, current wet AMD therapy is associated with a very significant treatment burden. Real-world patients are undertreated, receiving only 6 to 7 injections in the first year and only improving by 1 to 3 letters.

Given the durability of small molecule suspensions in the suprachoroidal space, we have assessed the potential of a proprietary suspension of axitinib for suprachoroidal injection as a long-acting therapy for wet AMD in multiple species. These preclinical studies have demonstrated reduced growth of experimental neovascularization with decreased fluorescein leakage as well as durable drug levels via suprachoroidal administration, supporting axitinib's potential to address current treatment burden.

Based on preclinical data, we believe that suprachoroidal injection, our proprietary suspension of axitinib has meaningful potential. First, axitinib demonstrates intrinsic high potency and achieves pan-VEGF inhibition through receptor blockade. Second, preclinical results from Clearside and outside investigators show pharmacodynamic effect with reduced growth of experimental neovascularization and decreased fluorescein leakage. And third, suprachoroidal administration of axitinib can potentially achieve prolonged duration and targeted delivery to effected tissue layers.

Based on this data, we are working to submit an IND application for CLS-AX in mid-2020. Over the last several months, we have worked with prominent physicians to deliver over 10 data presentations at key medical congresses, including the American Academy of Ophthalmology or AAO Annual Meeting. These presentations, combined with our recent partnerships with Aura Biosciences and REGENXBIO, has significantly expanded our presence into additional therapeutic areas, including ocular oncology, wet AMD and diabetic retinopathy.

We remain very encouraged by the support from the medical community and their enhanced understanding of the value of our suprachoroidal treatment approach.

With that, I will now turn the call over to our CFO, Charlie Deignan, to review our financial results. Charlie?

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Charles A. Deignan, Clearside Biomedical, Inc. - CFO [5]

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Thank you. Thank you, Tom. I would like to provide a summary of key financial developments. General and administrative expenses were $3.8 million for the quarter ended September 30, 2019, compared to $3.9 million for the quarter ended September 30, 2018. The research and development expenses for the quarter ended September 30, 2019, were $2.7 million compared to $20.1 million for the quarter ended September 30, 2018.

We expect R&D expenses to increase over the next several quarters as we complete the work to resubmit our NDA for XIPERE and submitting an IND for CLS-AX. As of September 30, 2019, our cash and cash equivalents totaled $22.6 million.

As we disclosed in our 8-K filing last month, in conjunction with our XIPERE licensing deal, we amended our loan agreement with Silicon Valley Bank, repaid $5 million of the outstanding principal balance and extended the period of interest-only payments up to an additional year. Based on this debt repayment, upfront licensing payments and our planned increase in R&D expenses, we expect that our existing cash and cash equivalents will enable us to fund our operating expenses into the third quarter of 2020. This does not include any additional partnership-related payments that we may gain from the achievement of development milestones. We look forward to ongoing engagement with the investment community at upcoming events, including the Stifel Healthcare Conference.

Now I am pleased to turn the call back over to George for his closing remarks.

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George M. Lasezkay, Clearside Biomedical, Inc. - Interim CEO & Director [6]

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Thank you, Charlie. It was a productive quarter for Clearside, and we are proud to align ourselves with some of the leaders in the ophthalmology space. We expect to satisfy the request from the FDA and resubmit our XIPERE NDA in the first quarter of next year. We are also excited to submit a new IND for axitinib and continue to expand our internal development pipeline. We appreciate the support of our shareholders over the last year and look forward to making additional progress.

I would now like to ask the operator to open the call up for questions.

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Questions and Answers

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Operator [1]

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(Operator Instructions) We do have our first question from the line of Liana Moussatos from Wedbush.

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Vasiliana Vireen Moussatos, Wedbush Securities Inc., Research Division - MD of Equity Research [2]

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Congratulations on all your progress. So I remember from covering Clearside for a while that axitinib was part of a pipeline a few -- a couple of years ago. And in, I don't know, in 2017, it was discontinued because competition failed. So what has happened since then that makes you so confident about your current formulation?

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George M. Lasezkay, Clearside Biomedical, Inc. - Interim CEO & Director [3]

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Liana, this is George. And I'll start, and I'll also have Tom chime in. But in the past, you're correct that axitinib was part of the pipeline several years ago. And the work on it was put on hold. The company at that time had limited R&D resources, and it was singularly focused at that time on expanding the indications for XIPERE. So when -- this summer, we started a review internally, and it makes sense to review your internal assets or any assets periodically from time to time as the science progresses and new data comes out. And based on our recent assessment of both what we had done internally and what we've seen in the scientific community, we think that axitinib used in the suprachoroidal space really offers us an exciting development opportunity with a large market and a high unmet need, as Tom explained. And I'll have Tom chime in and contribute more detail on, again, the rationale for making this move back to axitinib and taking it forward and filing the IND. Tom?

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Thomas A. Ciulla, Clearside Biomedical, Inc. - Chief Medical Officer [4]

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Thanks, George. So I've worked with our team to take a fresh look at our prior data as well as preclinical data from scientific researchers in the ophthalmology community. We believe there's evidence of potential treatment advantages using this therapy. Axitinib demonstrates intrinsic high potency and achieves pan-VEGF inhibition to receptor blockade and therefore, may have efficacy advantages over existing therapies.

We also know that in preclinical work done by independent investigators that axitinib has shown promising results in numerous ocular models. We also believe that recent data on VEGF regulation indicates that there may be improved outcomes with broad VEGF blockade. And our own preclinical data has demonstrated durable drug levels as well as efficacy in preclinical models.

So we believe we have one of the most potent tyrosine kinase inhibitors. And when we combine it with suprachoroidal delivery, we can target the drug at the location of the disease while achieving durable drug levels. Ultimately, we believe that axitinib and suprachoroidal suspension can reduce treatment burden and might even improve visual outcomes over current therapies, which predominantly focus on VEGF blockade or VEGF-A blockade, not broad VEGF blockade through VEGF receptor inhibition.

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Vasiliana Vireen Moussatos, Wedbush Securities Inc., Research Division - MD of Equity Research [5]

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Okay. Any comments on the competition problems 2, 3 years ago?

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George M. Lasezkay, Clearside Biomedical, Inc. - Interim CEO & Director [6]

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In past years, is that your question?

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Vasiliana Vireen Moussatos, Wedbush Securities Inc., Research Division - MD of Equity Research [7]

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Right. The reason why was -- that it was discontinued and this is February 2017 press release was mentioned because competition had failed. And everything you said sounds good, but do you have any insight right now or maybe we can follow-up later on why you guys look like it could succeed where the competition failed?

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Thomas A. Ciulla, Clearside Biomedical, Inc. - Chief Medical Officer [8]

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I'm glad you asked that question. That's a good question. So the past trials you're referring to involves platelet-derived growth factor inhibitors added to VEGF-A therapy. And the company was looking at that as a combination therapy. But basically, I think it's not the perfect analogy. But we have -- with a fresh look, we can see that we can completely inhibit VEGF. We achieved broad VEGF inhibition.

Current therapies, as you know, focus on VEGF-A inhibition. And there's now some reports suggesting that when you inhibit VEGF-A, you have up-regulation of other members of the VEGF family. And that's been shown both in AMD patients and also in metastatic cancer patients. So when you have up-regulation of these other factors, that could potentially lead to treatment resistance and insufficient response. So while that analogy was made a couple of years ago, I don't think it's a perfect analogy. I think it's more about pan-VEGF inhibition or broad VEGF blockade.

And there's also some clinical -- early clinical data suggesting that, that may actually achieve better visual outcomes in AMD patients. So we're very excited about the prospect, not only because we can achieve broad VEGF blockade, but we can also target the suprachoroidal space, achieve high levels at the affected tissue level -- layers and also achieve durable drug levels.

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Original post:
Edited Transcript of CLSD earnings conference call or presentation 6-Nov-19 9:30pm GMT - Yahoo Finance

Recommendation and review posted by Bethany Smith