StemCell Therapy

Good news for people suffering from genetic diseases and for those who could be helped with stem cell therapies. This week, Stanford announced the creation of the Center for Definitive and Curative Medicine, a new center that aims to bring life-changing advances to millions of patients.

The Center for Definitive and Curative Medicine is going to be a major force in theprecision healthrevolution, saidLloyd Minor, MD, dean of the School of Medicine, in a press release. Our hope is that stem cell and gene-based therapeutics will enable Stanford Medicine to not just manage illness but cure it decisively and keep people healthy over a lifetime.

The center plans to tap the rich vein of stem cell and gene therapy research underway at Stanford. These techniques pinpoint problems causing disease and introduce functional copies of genes or cells to replace malfunctioning ones. Its exciting work with the potential to make real changes in patient lives and Stanford with its deep strengths in research and clinical care is poised to lead.

The release explains:

Housed within theDepartment of Pediatrics, the new center will be directed by renowned clinician and scientistMaria Grazia Roncarolo, MD, the George D. Smith Professor in Stem Cell and Regenerative Medicine, and professor of pediatrics and of medicine.

It is a privilege to lead the center and to leverage my previous experience to build Stanfords preeminence in stem cell and gene therapies, said Roncarolo, who is also chief of pediatric stem cell transplantation and regenerative medicine, co-director of theBass Center for Childhood Cancer and Blood Diseasesand co-director of theStanford Institute for Stem Cell Biology and Regenerative Medicine. Stanford Medicines unique environment brings together scientific discovery, translational medicine and clinical treatment. We will accelerate Stanfords fundamental discoveries toward novel stem cell and gene therapies to transform the field and to bring cures to hundreds of diseases affecting millions of children worldwide.

Previously: Stanford scientists describe stem-cell and gene-therapy advances in scientific symposiumPhoto of Maria Grazia Roncarolo by Norbert von der Groeben

The rest is here:
Stanford Center for Definitive and Curative Medicine to tackle genetic diseases - Scope (blog)

Xconomy Boston

One day after the release of a Nature Medicine paper warning of the potential hazards of testing CRISPR-Cas9 gene editing in humans, Homology Medicines, a startup advancing a different genetic surgery technique, has just grabbed a big round of funding to make its own clinical push.

Homology, of Bedford, MA, wrapped up an $83.5 million Series B round this morning. A wide group of investors led by Deerfield Management provided the funding, bringing the companys total amount raised to a whopping $127 million since it was formed last year.

Homology is making the bold claim that its underlying science, technology it calls AMEnDR, is a better version of existing gene editing methods, among them the CRISPR-Cas9 technology that has taken the scientific research world by storm and has led to the formation of three now publicly traded companies, Editas Medicine (NASDAQ: EDIT), Intellia Therapeutics (NASDAQ: NTLA), and CRISPR Therapeutics (NASDAQ: CRSP).

CRISPR gene editing is a two-part biological system that researchers can use to help irreversibly alter DNA. The three companies are involved in a high-stakes race to use the technology to develop human therapeutics, with the first clinical trials expected to begin next year. Yet one of the fears involved in bringing the technology to human trials is the possibility of off-target effectsa genetic surgery error that causes irreparable damage, like cancer. One of the fields pioneers, Feng Zhang of the Broad Institute of MIT and Harvard, just co-authored a paper in Nature Medicine urging caution about the rush to move forward. Zhang and colleague David Scott argued that researchers should analyze patients DNA before giving them CRISPR-based drugs, citing the myriad differences between each persons genetic makeup.

Homology isnt using CRISPR, like its publicly traded rivals. Instead, its recreating a natural biological process known as homologous recombination, which cells in humans and other species do to repair DNA damage or, in the case of bacteria, to improve their genetic diversity. In homologous recombination, one chromosome essentially swaps one short DNA sequence for another similar one. Homology aims to engineer a piece of healthy DNA, pack it into a type of adeno-associated virus, or AAVa delivery tool commonly used in gene therapy and gene editing technologiesand infuse it into the body. The virus carrying the DNA locks on to the cell that needs a genetic fix, enters it, and releases its DNA payload. The healthy DNA then swaps places with the faulty gene inside the patients cells. If and when the cells divide, the new cells would carry the fixed gene, not the faulty one. One potential benefit of this approach is there may be less likelihood of an off-target error, like mutations in the target DNA that cause cancer, than with CRISPR.

Thats the hope, but the technology hasnt been tested in humans as of yet. With the new cash, however, Homology is getting a shot to try. In a statement, Homology CEO Arthur Tzianabos said the funding will help Homology bring its first drug candidate toward the clinic, though he didnt specify how long that might take. The company is focusing on rare diseasesno surprise given Tzianabos, chief operating officer Sam Rasty, and chief scientific officer Albert Seymour all worked with one another at rare disease giant Shire (NASDAQ: SHPG). According to its website, the company will develop therapies for inborn errors of metabolism, and Duchenne muscular dystrophy and cystic fibrosis are among its potential targets as well. (Duchenne and cystic fibrosis are early targets of CRISPR-based medicines as well.)

Fidelity Management and Research, Novartis, Rock Springs Capital, HBM Healthcare Investments, Arch Venture Partners, Temasek, 5AM Ventures, Maverick Ventures, Vida Ventures, Vivo Capital, and Alexandria Venture Investments also took part in the funding. Heres more on Homology, and gene editing with CRISPR-Cas9.

Ben Fidler is Xconomy's Deputy Biotechnology Editor. You can e-mail him at bfidler@xconomy.com

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Homology Med Bags $83.5M More, Fueling Push For Gene Editing Twist - Xconomy

Short Name: Nanomed Meeting 2017

Theme: Challenges and Innovations in next generation medicine

Website: http://www.meetingsint.com/pharma-conferences/nanomedicine-nanotechnology

Registration Link: http://www.meetingsint.com/pharma-conferences/nanomedicine-nanotechnology/registration

Nanomed Meeting 2017 Organizing Committee invites you to attend the largest assemblage of Nanomedicine and Nanotechnology researchers from around the globe during November 23-24, 2017 at Dubai, UAE.

Nanomed Meeting 2017 is a global annual event. This International Conference and Exhibition on Nanomedicine and Nanotechnology brings together scientists, researchers, business development managers, CEOs, directors, IP Attorneys, Regulatory Officials and CROs from around the world. The passage of Nanomed Meeting 2017 through a decade at Asia finds much requirement for discussion also focusing the latest developments in the field of Nanomedicine and Nanotechnology.

Why attend?

Join your peers around the world focused on learning about Nanomedicine and Nanotechnology related advances, which is your single best opportunity to reach the largest assemblage of participants from the Nanomedicine and Nanotechnology community, conduct demonstrations, distribute information, meet with current and potential professionals, make a splash with a new research works, and receive name recognition at this 2-day event. World-renowned speakers, the most recent research, advances, and the newest updates in Nanomedicine and Nanotechnology are hallmarks of this conference.

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International Conference and Exhibition on Nanomedicine and Nanotechnology - Technology Networks

What Debra Travers really wanted to be was a marine biologist, until I found out Jacques Cousteau wasnt hiring.

How she wound up as chief executive of PolyAurum LLC, a Philadelphia start-up developing biodegradable gold nanoparticles for treating cancerous tumors, involved a professional journey of more than 30 years in pharmaceutical and diagnostics industries, and a personal battle with the disease shes now in business to defeat.

After determining that studying sea creatures was not a viable career choice, Travers a military kid from all over switched her major at Cedar Crest College in Allentown to medical technology. She graduated in 1979, then worked for three years in a hospital laboratory until she concluded she didnt like shift work and could do more.

What followed was an impressive career progression: Travers started as a chemistry technician at DuPont Biomedical Products Division, advancing to executive positions in marketing and product development at Centocor, GlaxoSmithKline, Endo Pharmaceuticals, and IMS Health.

Much of that work involved bringing new products through the long development and regulation-heavy process from concept to launch, with experience in therapeutic areas including oncology, urology, pain medicine, cardiology, and rheumatology. In an industry of specialty silos, Travers developed a uniquely blended expertise in marketing and R&D.

It was on March 23, 2006, that her health-care vocation turned personal: Travers, then a 50-year-old mother of two, was diagnosed with breast cancer.

An oncologist recommended a double mastectomy, removal of both ovaries, and chemotherapy. The tearful pleadings of her daughter, Kelly, then 18 I need you here when I graduate college, when I get married, when I have kids persuaded Travers to follow that recommendation.

She returned to work at Endo for seven more years, as a director in project management, before being laid off in June 2013, one month before her daughters wedding. The break gave Travers time to concentrate on the big event and to start to think what Id like to do when I grow up.

That process would lead her in late 2015 to PolyAurum, a start-up spun out of the University of Pennsylvania.

I became a CEO and a grandmother in the same year, said Travers, now 61, chuckling during a recent interview at the Pennovation Center incubator in West Philadelphia. From there, her home in Delaware, and the sites of pitch opportunities with investors, she is working to raise $1.3 million in seed funding by early in the fourth quarter, to help get PolyAurum closer to clinical trials on humans.

So far, research and testing funded through $4 million in grants to the university has been limited to mice with tumors. It has shown that gold nanocrystals greatly enhance the effectiveness of radiation on tumors without increasing harm to healthy surrounding tissue, said Jay Dorsey, an associate professor and radiation oncologist at Penn and one of four university faculty who developed the technology.

The effectiveness of metals in improving a tumors ability to absorb radiation has long been known, Dorsey said. But one of the stumbling blocks to incorporating gold nanoparticles in such therapeutics is that the metal is not eliminated from the body well, posing serious problems to vital organs such as the liver and spleen.

Penns David Cormode, a professor of radiology, and Andrew Tsourkas, a professor of bioengineering, have worked to make gold more biocompatible, resulting in PolyAurums current technology, Dorsey said. The gold nanocrystals are contained in a biodegradable polymer that allows enough metal to collect in a tumor. The polymer then breaks down, releasing the gold for excretion from the body so that it does not build up in key organs.

The companys name is a combination of those two essential ingredients: Poly, derived from polymer, and Aurum, the Latin word for gold.

Explaining all that, and the potential that PolyAurums founders see for extending and saving lives, is the message Travers now is in charge of disseminating the part of the critical path to commercialization that is not the strength of most researchers toiling in laboratories.

She knows what the founders dont know it just makes a perfect match, said Michael Dishowitz, portfolio manager at PCI Ventures, an arm of Penn that helps university start-ups find investors, recruit management, and get to market.

Since its formation about eight years ago, PCI has helped more than 150 companies secure more than $100 million in funding, said Dishowitz, who has a doctoratein bioengineering from Penn and spent several years studying the impact of cell-signaling pathways on orthopedic injury.

While calling PolyAurums technology cool and very transformative for treatment, Dishowitz also delivered a dose of reality about the rigors ahead, as health-care start-ups must navigate a course with no guarantees their products will lead to actual clinical implementation.

PolyAurum is one of 13 companies that entered Philadelphia Media Networks second annual Stellar StartUps competition in the health-care/life sciences category. A total of nine categories drew 88 applicants. The winners will be announced Sept. 12 at an event at the Franklin Institutes Fels Planetarium. (Details at http://www.philly.com/stellarstartups.)

A lot has to go right, all the planets and stars have to align for this to hit the market, Dishowitz said of PolyAurums commercial prospects.

Which is why the team behind any start-up is so essential to investors, he said, calling Travers interest in joining a company that has yet been unable to pay her (she has equity in PolyAurum) incredibly lucky.

Margo Reed

At the Nanomedicine and Molecular Imaging Lab at Penn Medicine are (front row, from left) Jay Dorsey, a radiation oncologist and a founder of PolyAurum; Debra Travers, CEO; and Andrew Tsourkas, another founder of PolyAurum; and (back row, from left) Michael Dishowitz, portfolio manager, PCI Ventures at Penn; and David Cormode, lab director and PolyAurum founder. (MARGO REED / Staff Photographer)

The only thing Travers corporate-heavy background lacked, he said, was raising money for a start-up. It doesnt worry him, Dishowitz said, citing Travers perseverance, no-quit attitude.

When youre out there raising money, youre going to hear no about 100, 150 times before you hear yes, Dishowitz said.

When it comes to pitching for PolyAurum, Travers has extra incentive.

I am working on a cancer therapeutic, which is very important to the 11-year cancer survivor in me, she said.

As for handling nos, shes had plenty of professional experience with that.

After spending 30-plus years in the drug and diagnostic industries, where it is hard to find women CEOs or board members, Travers said, Ive learned to ignore the negative voices.

When: 5:30-8:30 p.m. Tuesday, Sept. 12.

Where: Fels Planetarium, Franklin Institute, 222 N. 20th St., Philadelphia 19103

For more information: http://www.philly.com/stellarstartups

Published: July 28, 2017 3:01 AM EDT

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Cancer survivor becomes a cancer fighter at a Philly start-up - Philly.com

There has been a growing interest in the different applications of nanomaterials in the field of medicine. An article published in Nanomedicine: Nanotechnology, Biology, and Medicine showed the ways in which Laponite, a synthetic clay made of nanomaterials, can be of use in clinical practice.

Current advances in technology have provided many opportunities to develop new devices that improve the practice of medicine. There has been a growing interest in the different applications of nanomaterials in the field of medicine.

An article published in Nanomedicine: Nanotechnology, Biology, and Medicine reviewed Laponite, a non-toxic synthetic clay composed of nanomaterials which has different uses in the field of medicine. Laponite can be used in drug delivery systems, as the synthetic clay protects substances from degradation in physiologic environments. Different experiments show that Laponite is effective not only in protecting drugs from degradation, but also in transporting and releasing drugs into the body. The degradation of Laponite in the physiologic environment also releases products which have biological roles, especially in bone formation.

Laponite has been shown to induce osteogenic differentiation of cells in the absence of other factors which are known to promote differentiation and cell growth. The application of nanomaterials in bioimaging has also been studied. In one experiment, Laponite was incorporated with gadolinum, a dye used in magnetic resonance imaging (MRI), resulting in brighter images and prolonged contrast enhancement for 1 hour post-injection. Laponite has also proven to be of use in the field of regenerative medicine and tissue engineering. This synthetic clay can elicit specific biologic responses, act as a carrier for biochemical factors, and improve the mechanical properties of scaffolds used for tissue growth.

In summary, nanomaterials and synthetic clays such as Laponite have many applications in the field of medicine. Although current published literature state no toxic effects on the human body, further studies are needed to assess safety before it can be applied to clinical practice.

Written By:Karla Sevilla

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Application of Nanomaterials in the Field of Medicine - Medical News Bulletin

A medical technologist at CombiMatrix examines live tissue looking for fetal cells to analyze. (File Photo)

From left, medical technologists Lilybeth Wilkens, Meg Purayil, and Catherine Marte work on live samples of tissue at CombiMatrix, a genetic testing lab in Irvine. (File Photo)

Catherine Marte works on live samples of tissue at CombiMatrix, a genetic testing lab in Irvine. (File Photo)

Chief Executive Mark McDonough stands in the lobby of his company, CombiMatrix Corp., in Irvine. The company hopes its testing method will be used to analyze cases of women whove had multiple miscarriages. (File Photo)

Irvine-based CombiMatrix Corp. has agreed to be acquired in an all-stock merger by San Francisco-based Invitae Corp. for $33 million.

The deal between two genetic biotech firms is subject to approval by CombiMatrixs stockholders and regulatory agencies.

CombiMatrix is a molecular diagnostics company that studies theremains of lost pregnancies, extracting DNA that is analyzed to uncover genetic abnormalities that can help explain why the women who gave the samples have had multiple miscarriages. It also does pre-implantation genetic diagnostics and screening, prenatal diagnosis andDNA-based testing forgenetic abnormalities.

In 2014, the companys chief executive, a former Navy navigator, told the Register the companys methods would soon become the standards for analyzing pregnancy loss and prenatal testing.

Merging with Invitae, which also specializes in genetic material and hereditary disorders, should expand the scope of CombiMatrixs reach.

By coming together with Invitae, we believe we can synergistically combine their scale, technology and expertise with the CombiMatrix product offering, human capital and sales channels to achieve even greater success in the future for the company and our shareholders, Mark McDonough, president and chief executive officer of CombiMatrix, said in a statement.

CombiMatrix will be a wholly-owned subsidiary of Invitae, according to documents filed with the Securities and Exchange Commission.

The company declined to provide any information not found in SEC documents, such as potential job impacts or executive changes.

For many people, preparing to have a child is their introduction to the power of genetics to inform health decisions, Sean George, chief executive officer of Invitae, said in a statement. The combination of Invitae and CombiMatrix will expand our ability to provide actionable answers to the complex questions that can arise when starting a family.

Testing has become more common as women have babies later in life. In 2016, theaverage age when women have their first child was 28. In 1970, it was 24.6, according to the CDC.

McDonough, in 2014, said he saw a market of up to $200 million a year for so called microarray prenatal testing and a$330 million marketfor recurrent pregnancy loss.

CombiMatrixspun off of Newport Beach-basedAcacia Research Corp. in 2007. It got its start supplyinglaboratories conducting microarray tests with biotech tools before founding a genetic testing lab.

In 2010, it moved to Irvine from Seattle.

Invitaeon Monday said it is also acquiring Cambridge, Mass.-based Good Start Genetics, another pregnancy-related molecular diagnostics company that specializes in preimplantation and carrier screening.

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Irvine-based CombiMatrix in $33 million merger deal with Bay Area genetics firm - OCRegister

Seattle Genetics (NASDAQ:SGEN) reported second-quarter revenue and earnings numbers, but the conference call focused mostly on potential near-term revenue growth for the biotech's one approved drug, Adcetris, with one expanded indication under review at the FDA and another to be there shortly.

Metric

Q2 2017

Q2 2016

Year-Over-Year Change

Revenue

$108.2 million

$95.4 million

13.4%

Income from operations

($59.4 million)

($33.4 million)

N/A

Earnings per share

($0.39)

($0.23)

N/A

Data source: Seattle Genetics.

Image source: Getty Images.

"Based on our review of pooled, blinded PFS events in the E2 trial, we have observed a lower rate of progression events compared with our projections. We plan to interact with FDA about the potential to unblind the trial prior to reaching the prespecified number of events. Based on the length of patient follow-up, we believe the trial data will be mature in 2018 and continue to expect to report E2 data next year." -- Seattle Genetics CEO and chairman Clay Siegall

There are a lot of terms in that quote, so let's break them down:

Blinded: The company doesn't know which drug the patients in the trial were treated with.

PFS: Progression-free survival, a measure of efficacy based on how long it takes a patient to progress or die -- whichever comes first -- while being treated.

E2: ECHELON-2, a clinical trial testing Adcetris in patients with mature T-cell lymphoma who haven't been previously been treated.

So to translate: Patients aren't progressing or dying as fast as expected, which is good news for patients and hopefully good news for Seattle Genetics if it's the patients being treated with Adcetris who are the ones responding better than expected, but we don't know for sure since the company is blinded. Since the trial is set up to end when a certain number of progression/deaths occur in both treatment arms combined, the trial is taking longer to complete than expected, so management plans to ask the FDA for permission to look at the data earlier.

Management increased 2017 guidance for ADCETRIS sales in the U.S. and Canada to a range of $290million to$310million. Not bad, but far from a blockbuster, which will only come from successful approval based on the ECHELON-1 trial. Investors will get a full look at that data at the American Society of Hematology meeting in December.

Beyond the aforementioned plan to get ECHELON-2 data earlier, Seattle Genetics has other clinical trial data coming. It recently started a phase 3 trial with Bristol-Myers Squibb testing Adcetris plus Bristol's Opdivo in relapsed or refractory Hodgkin lymphoma patients. And enfortumab vedotin, which is partnered with Astellas, will enter a phase 2 trial in metastatic urothelial cancer that management thinks should be enough to gain accelerated approval from the FDA if the trial is successful.

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Seattle Genetics, Inc.: Expanded Sales on Their Way (FDA Willing ... - Motley Fool

Healthcares been in the news a lot lately thanks to the Republicans efforts at healthcare reform. There are several bottoming heathcare and biotech stocks that could benefit from these efforts. One of those companies with potential is Myriad Genetics (MYGN). Myriad creates genetic tests to screen for genetic cancer risk and other diseases. Its currently coming out of a bottom and has the potential to continue moving up if it can capitalize on this growing market.

Myriad manufactures molecular diagnostic tests to screen for genetic dispositions to cancer, autoimmune diseases, and other maladies. The company is probably best known for its BRACAnalysis test. This test determines womens risk for breast cancer by detecting mutations in the BRCA1 or BRCA2 genes. Genetic mutations greatly increase the risk of developing cancer. So these type of diagnostic tests are growing more and more important for detecting cancer risks early. Advanced diagnostics also allow doctors to tailor their preventative medicine programs to better serve the patients individual needs. In fact, personalized medicine is expected to be the wave of the future. Theres also an increasing need for better diagnostic tools as the population ages. So Myriads products can fill an important niche in a growing market.

Myriads stock was in some trouble until recently. A monthly stock chart shows that MYGN was in a bear market for pretty much all of 2016. It finally started to bottom late in 2016, and completed its bottom in May of 2017. Its currently moving up strongly out of its bottom, but has retraced a bit over the past month. MYGNs all time high is at about $47, so this stock some room to move up.

Figure 1: Monthly chart of MYGN. Chart provided by FreeStockCharts.com.

A weekly chart confirms that MYGN completed its bottom last May. The chart shows very large and strong white candles coming out of the bottom, lacking indecisive wicks and tails. Small black candles form for about a month after each strong move up, likely due to profit taking. The stock is also currently undergoing some profit taking after the last strong move up in June. There have been green volume surges on the moves up, with green volume bars outnumbering the red bars since coming out of the bottom.

Figure 2: Weekly chart of MYGN. Chart provided by FreeStockCharts.com.

We can see the current retracement in more detail in the daily chart. Black candles have been larger and more numerous over the past month. We can see the strength of the green volume in May and June. But volume has been low in July, with red volume becoming more and more dominant. The chart also shows that high frequency traders (HFT) tried to sell down this stock on 7/21, possibly in response to a news release that day. It looks like the HFTs moved in at the start of the day, but failed to move the stock down much.

Figure 3: Daily chart of MYGN. Chart provided by FreeStockCharts.com

The chart indicators also confirm this weakness in the current price action. RSI is moving down steadily to the bottom of the chart. Stochastics has also moved down well into oversold territory. Both indicators look a little overextended but dont show any signs of a reversal quite yet.

Figure 4: Indicator charts for MYGN. Chart provided by FreeStockCharts.com

MYGN really looks a little week right now. There arent enough buyers to stop the slip-slide due to profit taking. The pattern of this stock, however, has been to move up strongly and then slowly slide back for a month. So well see if the stock continues that trend in August.

The charts show that MYGN has recovered nicely in 2017. So lets check the fundamentals and see if they justify the stocks uptrend. Myriads income statement shows that revenues started to turn around in the first quarter of fiscal year 2017. Revenues, however, were flat last quarter. Net income, on the other hand, has been inconsistent. Income has been up and down for the past several quarters. Costs have also continued to rise, but flattened out last quarter.

Figure 5: Income statement for MYGN. Data provided by Google Finance.

Myriad also took on a lot of debt in the second quarter of fiscal year 2017. The company started to pay down that debt last quarter, so at least its controlling its debt load. The current ratio is about 1.6, which is acceptable.

Figure 6: Balance sheet for MYGN. Data provided by Google Finance

Data from Nasdaq.com shows that the institutional holdings are above 100%. This usually means that some convertible bonds were executed, or that there was a direct stock sale to an institution. This should pattern out in the next quarter after Nasdaq.com updates its information. MYGN has 261 holders, so its pretty widely held for a small cap. And more funds started new positions or increased their positions compared to funds that sold out or decreased their holdings.

Figure 7: Institutional Holdings for MYGN. Data provided by Nasdaq.com.

A few giant funds are among the top holders of this stock, including Vanguard and Dimensional Fund Advisors. There are few giant banks in the mix as well, such as Blackrock and State Street. Some large banks have reduced their holdings in this stock though, which is interesting. The buy side institutions are doing the opposite; theyre increasing their positions or holding steady.

Figure 8: Top holders for MYGN. Data provided by Nasdaq.com.

Myriads financial performance improved last year but slowed down last quarter. Can it pick up again? A quick look at the last earnings report can show us what to expect. Myriad expects accelerating growth going forward, per CEO Mark Capone. Capone said, Coupled with meaningful sequential volume growth in all of our major pipeline tests including GeneSight, Vectra DA, Prolaris, and EndoPredict, we believe we are rapidly approaching an important inflection in our business where our new products will drive accelerated revenue growth and profitability.

That sounds promising, but we need more specifics. Luckily, the report also provides guidance for the fourth quarter of fiscal year 2017. The company expects revenues of $192-$194 million, which is flat to slightly down compared to last quarter. Earnings per share are expected in the range of $0.11 to $0.13, which is a large improvement over the $0.06 per share reported last quarter. Revenues for the full fiscal year are expected to be $763-$765, up just 1% over 2016. Earnings per share are expected to drop from 2016, down to $0.23-$0.25. This reflects the slowdown in earnings that was observed in the first three quarters of fiscal year 2017. The company needs to show that it can continue to grow revenues and earnings more consistently. Itll be interesting to see what the guidance is for 2018 in the next earnings release. Myriad is expected to report around August 8th.

MYGN has trended up fairly strongly out of its 2016 bottom. Its also in a market with a lot of potential, notwithstanding the healthcare shenanigans going on in Congress right now. The companys financials showed improvement earlier in the year, but have started to lag more recently. It needs to continue to grow revenues and earnings, otherwise the current profit taking could turn into broader selling. The 2016 high of $40 could also challenge the uptrend if the financials dont continue to improve. The stock shows some promise, but theres also some uncertainty about companys performance in the next fiscal year. The fact that large funds are interested in this stock bodes well for it though. And an earnings report that beats expectations could cause the stock to move up strongly again. Investors that are interested this companys technology should wait until the profit taking ends before entering this stock though. The $27 level looks like a good place to enter. For everyone else, its best to wait until the next earnings report to see the companys projections for fiscal year 2018.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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Myriad Genetics' Stock Is Starting To Make A Run - Seeking Alpha

The News Minute

Genetics may have a role in Uddanam kidney disease, claim Harvard experts The News Minute A two-member committee from Harvard University has found that genetic predisposition and a strong genetic founder effect could be the reason for nephropathy (kidney disease) in Uddanam, according to a Times of India report. The hypothesis is based on ... and more »

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Genetics may have a role in Uddanam kidney disease, claim Harvard experts - The News Minute

The Canaanites lived at the crossroads of the ancient world. They experienced wars, conquests and occupations for millennia, and as a result evolutionary geneticists expected that their DNA would become substantially mixed with incoming populations.

Astonishingly, new genetic analysis shows that scientists were wrong. According to a new study in the American Journal of Human Genetics, todays Lebanese share a whopping 93% of their DNA with the ancient Canaanites.

The study also found that the Bronze Age inhabitants of Sidon, a major Canaanite city-state in modern-day Lebanon, have the same genetic profile as people living 300 to 800 years earlier in present-day Jordan.

Later known as Phoenicians, the Canaanites have a murky past. Nearly all of their own records have been destroyed over the centuries, so their history has been mostly pieced together from archaeological records and the writings of other ancient peoples.

Archaeologists at the Sidon excavation site have been unearthing ancient Canaanite secrets for the last 19 years in the still-inhabited Lebanese port city. The team has uncovered 160 burials from the Canaanite period alone, said Claude Doumet-Serhal, director of the excavation. They have found people of all ages in these Canaanite burials, she said children were buried in jars and adults were placed in sand.

Claude Doumet-Serhal / The Sidon Excavation

An aerial view of the Sidon excavation site.

An aerial view of the Sidon excavation site. (Claude Doumet-Serhal / The Sidon Excavation)

Aided by new DNA sampling techniques, a team of evolutionary geneticists including Marc Haber and Chris Tyler-Smith from the Wellcome Trust Sanger Institute stepped in.

They sequenced the whole genomes of five individuals found in Sidon who lived about 3,700 years ago. The team then compared the genomes of these ancient Canaanites with those of 99 Lebanese people currently living in the country, along with the previously published genetic information from modern and ancient populations across Europe and Asia.

First, they investigated the genetic ancestry of the Canaanites themselves. They found that these Bronze Age inhabitants of Sidon shared about half their DNA with local Neolithic peoples and the other half with Chalcolithic Iranians. Interestingly, this genetic profile is nearly identical to the one evolutionary geneticist Iosif Lazaridis and his team found last year in Bronze Age villagers near Ain Ghazal in modern-day Jordan.

This suggests that Canaanite-related ancestry was spread across a wide region during the Bronze Age and was shared between urban societies on the coast and farming societies further inland. This evidence supports the idea that different Levantine cultural groups such as the Moabites, Israelites, and Phoenicians may have had a common genetic background, the authors said.

The researchers were also able to determine that the genetic mixing of the Levantine and Iranian peoples happened between 6,600 and 3,550 years ago, a range they would be able to narrow down with more ancient DNA samples from the region.

Claude Doumet-Serhal / The Sidon Excavation

The buried remains of a Canaanite adult whose DNA was sequenced in the study.

The buried remains of a Canaanite adult whose DNA was sequenced in the study. (Claude Doumet-Serhal / The Sidon Excavation)

Next, the team wanted to compare the Canaanite genome with the genetic makeup of the people who currently inhabit the ancient Canaanite cities. To do this, they collected DNA from 99 Lebanese people Druze, Muslim, and Christian alike.

As expected, they found some new additions to the modern Lebanese genome since the Bronze Age. About 7% of modern Lebanese DNA originates from eastern Steppe peoples found in what is now Russia, but wasnt represented in the Bronze Age Canaanites or their ancestors. What surprised the team was what was missing from their genetic data.

If you look at the history of Lebanon after the Bronze Age, especially it had a lot of conquests, Haber said. He and Tyler-Smith expected to see greater genetic contributions from multiple conquering peoples, and were surprised that as much as 93% of the Lebanese genome is shared with their Canaanite predecessors.

Though a 7% genetic influx from the Steppe seems very small, that number might be covering some hidden complexities, said Lazaridis, who worked on the Bronze Age Jordanian samples but was not involved in the new study.

Not much is known about the migrations of these eastern Steppe populations, he said. If the genomes of the incoming people were only half Steppe, for example, 14% of the Lebanese genome could have come from the new migrants.

Haber and Tyler-Smith said they want to explore this complexity further. Who were those eastern migrants? Where did they come from? And why did they migrate toward the Levant region? Haber asked. Analyzing more samples from different locations and periods could lead to an answer.

The team also wanted to know if the individuals from Sidon are more similar to modern-day Lebanese than to other modern Eurasian populations.

Despite small genetic variations between the three religious groups caused by preferential mating over time, the Lebanese genome is not widely varied. As a whole, the Lebanese people have more genetic overlap with the Canaanites from Sidon than do other modern Middle Eastern populations such as Jordanians, Syrians or Palestinians.

The difference is small, but its possible that the Lebanese population has remained more isolated over time from an influx of African DNA than other Levantine peoples, Lazaridis suggested.

Claude Doumet-Serhal - The Sidon Excavation

An archaeologist sorts pottery at the Sidon excavation site.

An archaeologist sorts pottery at the Sidon excavation site. (Claude Doumet-Serhal - The Sidon Excavation)

The findings have powerful cultural implications, Doumet-Serhal said. In a country struggling with the ramifications of war and a society fiercely divided along political and sectarian lines, religious groups have often looked to an uncertain history for their identities.

When Lebanon started in 1929, Doumet-Serhal said, the Christians said, We are Phoenician. The Muslims didnt accept that and they said, No, we are Arab.

But from this work comes a message of unity. We all belong to the same people, Doumet-Serhal said. We have always had a difficult past but we have a shared heritage we have to preserve.

mira.abed@latimes.com

Twitter: @mirakatherine

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Fake news about statins is discouraging the use of these life-saving drugs, expert warns

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The DNA of ancient Canaanites lives on in modern-day Lebanese, genetic analysis shows - Los Angeles Times

ELSI Research ProgramThe Ethical, Legal and Social Implications (ELSI) Research Program

ELSI Research Program Overview

The National Human Genome Research Institute's (NHGRI) Ethical, Legal and Social Implications (ELSI) Research Program was established in 1990 as an integral part of the Human Genome Project (HGP) to foster basic and applied research on the ethical, legal and social implications of genetic and genomic research for individuals, families and communities. The ELSI Research Program funds and manages studies, and supports workshops, research consortia and policy conferences related to these topics.

An article describing the ELSI Research Program in greater detail can be found here:The Ethical, Legal and Social Implications Program of the National Human Genome Research Institute: Reflections on an Ongoing Experiment

The NHGRI Division of Genomics and Society has identified the following three research domains to be considered for support by the Ethical, Legal and Social Implications (ELSI) Research Program. The domains are overlapping, and it is anticipated that many research projects will address issues that cut across domains. A more detailed discussion of these domains and a list of examples of possible research topics is available atELSI Research Domains.

Genetic and Genomic Research. These projects may investigate and address the ethical, legal, social, and policy issues that arise in connection with the design and conduct of genetic and genomic research.

Genetic and Genomic Health Care. These projects may investigate and address the ethical, legal, social, and policy issues that arise in connection with the translation of genetic and genomic research into clinical medicine and health care in a variety of healthcare settings.

Broader Legal, Policy and Societal Issues. These projects may investigate and address a range of broader ethical, legal, policy and societal issues raised by the use of genetic and genomic technologies and information in research, clinical or non-medical settings.

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The NHGRI ELSI Program accepts Conference Grant (R13) applications. For specific instructions for preparing a conference grant application, see:

The ELSI program participates in a number of training and career development funding opportunities.

The Fogarty International Center's international bioethics training programs[fic.nih.gov] support education and research training to develop ethics expertise in low- and middle-income countries (LMICs). The programs complement other global health research and research training programs in the mission areas of NIH Institutes and Centers.

PA-16-288[grants.nih.gov]:Research Supplements to Promote Diversity in Health-Related Research (Admin Supp)Expiration Date: September 30, 2019

PA-16-288[grants.nih.gov].Research Supplements to Promote Re-Entry into Biomedical and Behavioral Research Careers (Admin Supp)Expiration Date: September 30, 2019

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In the Fall of 2003, the NHGRI in collaboration with U.S. Department of Energy (DOE) and the National Institute of Child Health and Human Development (NICHD) launched a new initiative to create interdisciplinary Centers of Excellence in ELSI Research (CEER). The CEERs are designed to bring investigators from multiple disciplines together to work in innovative ways to address important new, or particularly persistent, ethical, legal, and social issues related to advances in genetics and genomics. In addition, the centers will support the growth of the next generation of researchers on the ethical, legal and social implications of genomic research. Special efforts will be made to recruit potential researchers from under-represented groups.

NIH funds new studies on ethical, legal and social impact of genomic information NewMay 17, 2016

For more information about the CEER's program, see: Centers of Excellence in ELSI Research (CEER).

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Joy Boyer, B.A.E-mail: boyerj@exhange.nih.gov

Dave Kaufman, Ph.D.E-mail: dave.kaufman@nih.gov

Nicole Lockhart, Ph.D.E-mail: lockhani@mail.nih.gov

Jean McEwen, J.D., Ph.D.E-mail: mcewenj@mail.nih.gov

Erin CurreyE-mail:Erin.currey@nih.gov

Margaret GinozaE-mail:margaret.ginoza@nih.gov

Tasha StewartE-mail: Tasha.stewart@nih.gov

AddressThe Ethical, Legal and Social Implications Research ProgramNational Human Genome Research InstituteNational Institutes of Health5635 Fishers LaneSuite 4076, MSC 9305Bethesda, MD 20892-9305

Phone: (301) 402-4997Fax: (301) 402-1950E-mail: elsi@nhgri.nih.gov

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Last Updated: July 10, 2017

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ELSI Program - National Human Genome Research Institute

Newswise "Good morning, doctor, I am here for my gene editing appointment. In the future, could this be a greeting heard in physician offices around the world? With the introduction of CRISPR technology, genetic material can now be more easily and precisely edited, even creating changes that can subsequently be inherited by offspring.

CRISPR stands for clustered regularly interspaced short palindromic repeats, and is often used as shorthand for the CRISPR-associated protein 9 (Cas9) technology that in the lab can remove and replace specific sequences of DNA. Scientists now expect that it will be harnessed to treat and or possibly cure debilitating diseases such as sickle cell anemia, Duchenne muscular dystrophy, Huntingtons disease, HIV, and cancers. Preclinical studies are underway to assess the safety of CRISPR-Cas9 and its ability to discriminate healthy from non-healthy cells.

Jennifer Doudna, PhD, a geneticist and a co-creator along with Emmanuelle Carpenter, PhD, of the CRISPR-Cas9 technology, delivered the Wallace H. Coulter Lectureship Award plenary on Sunday with her talk, CRISPR Biology, Technology & Ethics: The Future of Genome Engineering.

A key leader in the field, Doudna detailed her laboratorys innovative findings that she has collectively referred to as a molecular scalpel for genomes. She intrigued the audience by reviewing the intricate bacterial CRISPR adaptive immune system and how her lab harnessed its power to edit genes. She also delved into CRISPRs potential applications, current limitations, and ethical concerns.

Work is ongoing aimed at making CRISPR more amenable to fighting disease.

Doudna explained how her current research is refining CRISPRCas9 to improve its efficiency and prevent off-target mutations. Her laboratory has developed a CRISPRCas9 targeted gene knockdown method with enhanced efficiency. This method employs techniques such as single-particle electron microscopy to delineate the molecular mechanisms underlying the highly diverse CRISPR-Cas networks.

Several researchers already are using CRSPR to edit genes. Gene editing research in animal models involving mice and monkeys, in human embryos, and in HIV-infected human cells show promise. For example, in 2016 Lu You, MD, and colleagues at Sichuan University in Chengdu became the first group in the world to inject CRISPRCas9 modified T cells into patients with non-small cell lung cancer. The technology also is being used to speed pharmaceutical research and identify targeted therapies for somatic and heritable diseases.

A scan of ClinicalTrials.gov produces more than 10 clinical trials focused on examining the efficacy of CRISPR-Cas9 in diseases as varied as human papillomavirus-related malignant neoplasm (NCT03057912), HIV-1 (NCT03164135), sickle cell disease (NCT03167450), and prostate cancer (NCT02867345). The majority of these trials have not commenced patient recruitment.

What social and ethical frameworks should we use to consider research using CRISPR-Cas9? Be sure to attend the Wednesday afternoon symposium, Ethics in Laboratory Medicine, where Seema Mohapatra, JD, MPH will present Legal and Ethical Issues with Mitochondrial Replacement and CRISPR-Cas9. She will discuss the applications of technologies such as developing so-called designer babies, and the controversy surrounding what impact CRISPR gene editing will have on modern eugenics. Mohapatra has written in law journals about the need to examine such technology through the lens of reproductive justice and disability justice to ensure that all voices are heard and all populations can benefit from such powerful scientific discoveries.

CRISPR-Cas9 and mitochondrial replacement technologies are here, and the widespread opportunity for their clinical use in humans is imminent. Laboratorians, what roles will you play in ensuring the ethnical use of these powerful technologies?

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How Powerful is CRISPR? - Newswise (press release)

WASHINGTON, DC--(Marketwired - Jul 27, 2017) - The Alliance for Regenerative Medicine (ARM) has issued the following statement in response to the news that a research team from the Oregon Health and Science University has, for the first time in the United States, utilized gene editing technology to modify human embryos:

ARM continues to monitor the latest developments in gene-editing technologies, given their enormous medical potential. We reiterate our encouragement and support of the use of these technologies in somatic (non-reproductive) human cells to durably treat and potentially cure human diseases. Patients will benefit more immediately from resources being directed towards somatic applications of the technologies at this time, as most genetic diseases manifest in and can be treated in somatic, not germline, cells. In addition, many unresolved safety, ethical and legal issues remain with human germline engineering approaches.

ARM continues to support the National Academies of Sciences, Engineering and Medicine (NASEM), which noted in its February 2017 report, "heritable germline editing is not ready to be tried in humans."

About The Alliance for Regenerative Medicine

The Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 265 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visit http://www.alliancerm.org.

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The Alliance for Regenerative Medicine Issues Statement in Response to Reports of the First Use of Gene Editing ... - Benzinga

Artwork via Angelica Alzona/Gizmodo

Today in America, if you are poor, you are also more likely to suffer from poor health. Low socioeconomic statusand the lack of access to healthcare that often accompanies ithas been tied to mental illness, obesity, heart disease and diabetes, to name just a few.

Imagine now, that in the future, being poor also meant you were more likely than others to suffer from major genetic disorders like cystic fibrosis, TaySachs disease, and muscular dystrophy. That is a future, some experts fear, that may not be all that far off.

Most genetic diseases are non-discriminating, blind to either race or class. But for some parents, prenatal genetic testing has turned what was once fate into choice. There are tests that can screen for hundreds of disorders, including rare ones like Huntingtons disease and 1p36 deletion syndrome. Should a prenatal diagnosis bring news of a genetic disease, parents can either arm themselves with information on how best to prepare, or make the difficult decision to terminate the pregnancy. That is, if they can pay for it. Without insurance, the costs of a single prenatal test can range from a few hundred dollars up to $2,000.

And genome editing, should laws ever be changed to allow for legally editing a human embryo in the United States, could also be a far-out future factor. Its difficult to imagine how much genetically engineering an embryo might cost, but its a safe bet that it wont be cheap.

Reproductive technology is technology that belongs to certain classes, Laura Hercher, a genetic counselor and professor at Sarah Lawrence College, told Gizmodo. Restricting access to prenatal testing threatens to turn existing inequalities in our society into something biological and permanent.

Hercher raised this point earlier this month in pages of Genome magazine, in a piece provocatively titled, The Ghettoization of Genetic Disease. Within the genetics community, it caused quite a stir. It wasnt that no one had ever considered the idea. But for a community of geneticists and genetic counsellors focused on how to help curb the impact of devastating diseases, it was a difficult thing to see articulated in writing.

Prenatal testing is a miraculous technology that has drastically altered the course of a womans pregnancy since it was first developed in the 1960s. The more recent advent of noninvasive prenatal tests made the procedure even less risky and more widely available. Today, most women are offered screenings for diseases like Down syndrome that result from an abnormal presence of chromosomes, and targeted testing of the parents can hunt for inherited disease traits like Huntingtons at risk of being passed on to a child, as well.

But there is a dark side to this miracle of modern medicine, which is that choice is exclusive to those who can afford and access it.

This is one of those aspects of prenatal testing that we dont want to talk about, Megan Allyse, who studies reproductive ethics at the Mayo Clinic, told Gizmodo. Theres a wide variety of reasons people might not get access to reproductive technologies. But what is unavoidable is that you are more likely to have access if you are socio-economically well-off.

The scenario Hercher imagines is this: Say you dont have insurance, or have insurance that does not cover the roster of prenatal tests that OB/GYNs commonly recommend. You also cannot afford the tests out-of-pocket, and your baby is born with a genetic disease. This scenario plays out over and over again among people who cannot afford testing, while at the same time many of those who can afford the test for that disease and test positive choose to terminate a pregnancy. Over time, Hercher predicts, that disease would become more prevalent in those communities that could not afford the tests.

Whether this hypothetical scenario will play out in the real world isnt totally clear, in part because there are many variables besides socioeconomic status at work. Maybe you live in a state where abortions are more difficult to access or against local norms, influencing your decision to undergo prenatal testing. Perhaps you oppose abortion for cultural or religious reasons. And there isnt data for on individuals who refuse prenatal testing altogether, even if they could afford it. Somewhere around 70 percent of women opt-in to some form of prenatal testing, but those numbers vary wildly by region, jumping up to about 90 percent on the coasts and dropping significantly in the midwest.

At this point, all researchers can really do is speculate about future disparities in genetic disease. For example, a 2012 meta-analysis published in Prenatal Diagnosis found that across the country, the mean termination rate for Down syndrome was 67 percent, meaning that a significant number of people who undergo prenatal testing and wind up testing positive for Down syndrome choose to end the pregnancy. Of course, not every parent who learns their future child will have Down syndrome wants to terminate the pregnancy. Its is a complex, personal choice. But access to prenatal testing also allows a parent to better plan for their childs future needs.

Some geneticists already see evidence of an accessibility gap in their own clinical practices.

Certainly we know that access to care varies, Massachusetts General medical geneticist Brian Skotko told Gizmodo. His own work has studied the demographic breakdown of Down syndrome, and has found a clear racial pattern in both Down syndrome births and pregnancy terminations.

In Massachusetts, were seeing more Hispanic and black mothers with [babies with Down syndrome], he said, and what weve learned from their stories is either they dont have access to testing or that if they did get tested, they had strong religious beliefs.

As access to prenatal testing increases, Skotko said, it is likely we will see a drastic reduction in genetic diseases. In the next five years, as tests get better and better, the global market for them is expected to balloon by 25 percent to over $10 billion. We can look to historical evidence, Skotko said. As more people get access to prenatal tests, there will be an increase in number of selective terminations.

Access to prenatal testing isnt the only thing that could lead to Herchers fear becoming a reality, either. Abortion access has become increasingly difficult in some parts of the country, with states like Texas stripping funding for clinics and placing more restrictions on the conditions under which they can take place. In vitro fertilization could one day also contribute, allowing those who can afford the tens of thousands of dollars to undergo IVF to select the most genetically-desirable eggs for implantation.

In her new book, Whittier Law School professor Judith Daar makes a terrifying prediction: that unequal access to IVF may wind up bringing about a new eugenics.

The growth and success of reproductive technologies, accounting for three out of every one hundred babies born in the United States today, have prompted lawmakers to introduce and occasionally pass legislation that expressly or indirectly limits access to [assisted reproductive technologies] by certain individuals, she writes. These formal legal barriers, combined with individual and practice-wide physician conduct, coalesce to suppress access to assisted conception for those who have historically experienced a devaluation of their reproductive worth.

Daar points out that while in the 1942 case Skinner v. Oklahoma, the Supreme Court affirmed that procreation is a right, striking down the states compulsory sterilization of certain criminals, the ruling only weighs in on procreating naturally. The court has yet to rule on anything that might also equalize access to technologies that could help with conception, or to ensure that a child conceived is healthy.

Whats missing in the conversation is how we adopt all of these technologies to a society that considers well-being for all, Eleonore Pauwels, a bioethicist at the Wilson Center, told Gizmodo. There is already an access problem. But what about when were editing out diseases? Who will pay for CRISPR? We are looking at much more disruption in the future.

The only real way to prevent genetic diseases from becoming diseases of poverty, said Josephine Johnston, a bioethicist at The Hastings Institute, is to make sure everyone has access to the same services. While the costs of todays tests may one day be affordable for more people, there will inevitably also be newer, more expensive technologies that create the same issues in the future. Thus is the cycle of healthcares disparity of accessthere are always people for whom treatment is not equal to the rest.

People have to have access to healthcare services, and [genetic testing] needs to be part of what those services include, she told Gizmodo. If you dont have access to testing and termination servicesor support if you continue the pregnancyyou dont really have a choice about what to do. Its not a choice if youre backed into a corner.

The inequality threat that prenatal testing, IVF and germline editing present, is of course a version of the same inequality that has always existed. If you are poor, there is a good chance your access to healthcare is not as good as someone who has more money.

But as these technologies grow in power and expense, the gulf of that inequality widens. Genetic disease has always been our shared vulnerability, Hercher wrote in Genome. When one part of society can opt out of risk, will they continue to feel the same obligation to provide support and resources to those who remain vulnerable, especially if at least some of them have deliberately chosen to accept the risk?

Hercher presents what is really a common vision of dystopia: a future of genetic haves and have-nots in which inequality becomes encoded in our basic biology. But arriving at that future does not require genetic engineering or some other as-yet-unknown technology. All it requires is that we keep doing what we are already doing, living in a world in which access to necessary healthcare is often a luxury off-limits to the poor.

Editors Note: A quotation from Brian Skotko has been changed to reflect the preferred language used to refer to people with Down syndrome.

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Will Healthcare Inequality Cause Genetic Diseases to Disproportionately Impact the Poor? - Gizmodo

Jeff Boeke, director of the Institute for Systems Genetic s at New York University School of Medicine, has been working with his team on "rewriting" the yeast genome or, creating new yeast DNA codes to be transcripted into yeast cells.

The controversial project aims to supplement Genome Project-write or GP-write, which focuses on creating new genomes for humans and other animals that began in June 2016 by Boeke, and a group of other scientists, including the controversial geneticist from Harvard University, George Church.

The concept is that synthetic human genomes created, can be inserted into ordinary human cells whose natural DNA has been removed. This should, theoretically, then allow the scientists to match genetic sequences to their relevant traits, disease processes, and physiological functions.

A secret initial meeting to discuss GP-write was conducted in May this year and many experts are concerned about the ethical, legal and social issues, as well as skeptical as to whether the team will be able to fully synthesise human DNA.

The team hopes that this would be a stepping stone for GP-write and reveal basic, hidden rules that govern the structure and functioning of genomes and also possibly reveal new and useful characteristics that can yield new vaccines, biofuels or be part of the drug-manufacturing process in pharmaceutical factories.

"[Cell lines] have been cultured in dishes in labs for decades. But you can't engineer the genomes the tools for doing that are quite crude, relatively speaking," he says. A synthetic cell that lacked unnecessary genetic material could, consistently produce useful drugs to treat disease.

To design a stretch of DNA, the team begins with a stretch of normal, nature-made DNA and uploads that sequence onto a computer. Then, through an internally designed algorithm, specific changes are made to the sequence.

This altered sequence then becomes a blueprint and is sent to a company that manufactures chunks of the DNA containing the sequence. Finally, back in the laboratory, these short strands are joined together to make long sections of DNA.

Boeke's research has so far built one-third of the yeast genome and he hopes the rest will be constructed by the end of the year. But it will take longer to test the new DNA and fix problems, and subsequently combine the various chunks to complete the synthetic genome, he says.

At Harvard University, researchers Jeffery Way and Pamela Silver are developing harmless strains of salmonella DNA to be used as a vaccine against food poisoning that is caused by Salmonella, E. coli and Shigella.

To render the bacteria harmless when picking up DNA from other bacteria, requires altering the genome in 30,000 sites.

The only practical way to do that, Way says, is to synthesize it from scratch.

Whilst they said they were heartened to see that the leaders of GP-write have started discussions of ethical, legal and social issues, the idea of making a human genome is still a sensitive one.

Attorney Nancy J. Kelley, who organised the secret meeting in May and helped found the New York Genome Center, also mentioned that ethical concerns were only given a passing mention.

You can only introduce the concepts. You cant really discuss them or raise a debate about them in the paper," she added.

Zoloth and Endy were against the pursuit of the synthesis of human DNA, but the project has already begun taking shape with support from the Centre of Excellence for Engineering Biology, which will oversee the project and California-based software company Autodesk, which has committed USD250,000.

But more work still has to be done to convince the scientific community and the team recognises that.

The notion that we could actually write a human genome is simultaneously thrilling to some and not so thrilling to others, Boeke said. So we recognize this is going to take a lot of discussion. MIMS

Read more:Are we over-propagandising genetic research?FDA approves sale of genetic tests for disease riskHuman knockouts: A way to decipher why some drugs work, while others failSources:https://www.statnews.com/2016/06/02/project-human-genome-synthesis/https://www.statnews.com/2017/07/26/scientists-build-dna-scratch-alter-lifes-blueprint/http://www.cnbc.com/2017/05/02/synthetic-dna-scientist-says-it-could-be-inside-humans-within-5-years.html

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"Rewriting" yeast genomes: Paving the way to create human DNA from scratch - MIMS General News (Hong Kong) (registration) (blog)

Gaining market share in rheumatoid arthritis has been looking ever more difficult because of new entries and looming biosimilar competition, and now a spotlight on safety has only added to the uncertainty.

The US FDA has cited specific cardiovascular safety issues with two of the current wave of rheumatoid arthritis agents, Lilly's (NYSE:LLY) Olumiant and Johnson & Johnson's (NYSE:JNJ) Plivensia, the former of which might need to conduct a new clinical trial to rule out risk. The eligible population for these agents numbers in the millions and has several existing choices, so regulators seem to be taking a more assertive approach on the safety profile of follow-on products.

Briefing documents released yesterday before an FDA advisory committee meeting on Plivensia, whose active ingredient is sirukumab, revealed that the staff wants its expert consultants to consider an imbalance in deaths, major cardiovascular events like heart attacks and strokes, and cancer in its clinical program. This comes a week after Lilly revealed that Olumiant had been knocked back because the FDA wants to analyse an imbalance in thromboembolic events in its trials more closely.

In Plivensia's trials investigators said 34 of 35 deaths were in patients receiving the injection, and 13 of the 34 were from major adverse cardiovascular events. Like other IL-6 inhibitors, the project raises low-density lipoproteins. Overall, there was an imbalance in overall cardiovascular events in the Plivensia 50mg group, for which J&J is seeking approval, versus placebo, the FDA concluded.

Last week, Lilly reported that the FDA had cited five cases of deep-vein thrombosis or pulmonary embolism in its complete response letter denying approval of Olumiant.

In both cases, the regulator's caution might have been motivated by experiences with products in the same classes already on the market. The FDA's adverse events monitoring has identified 18 primary cases of pulmonary embolism with Olumiant's Jak inhibitor predecessor Xeljanz, according to FAERS data compiled by Advera Health. Actemra, which is an IL-6-inhibiting antibody like Plivensia, has reported 21 (Olumiant clot signal echoes Xeljanz experience, July 26. 2017).

As Lilly did last week with Olumiant, J&J says it is confident that the benefit of Plivensia outweighs the risk.

The end of unmet needs

The allure of the $25bn rheumatoid arthritis space is strong, but as biosimilars of established products prepare to enter it is becoming harder to argue that there is an unmet medical need in the disease and in associated autoimmune disorders.

The RA population numbers nearly 2 million in the US, and if all the indications for which the market leader, Humira, is prescribed excluding psoriasis are considered, the eligible population rises to more than 4 million. Thus the FDA might be growing more cautious about approving agents that could result in a significant number of complications and deaths.

Moreover, biosimilars are on their way - so far, the US FDA has approved copycats of Abbvie's (NYSE:ABBV) Humira and Roche's (OTCQX:RHHBY) Enbrel, and Pfizer (NYSE:PFE) has launched the Remicade biosimilar Inflectra. Their entry means that the US market for rheumatoid arthritis agents is not expected to expand measurably in sales terms from $14bn this year, and new entrants will not want to test a fiercely competitive market at a safety disadvantage.

Forecasts for Olumiant have shrunk to less than $1bn in 2022 since Lilly received the bad news from the FDA, and Plivensia US sales forecasts remain modest at $638m in 2022; Actemra will top out at about $1bn in 2021. Taken together the IL-6s are dwarfed in autoimmune disease by the anti-TNF class of Humira, Enbrel and Remicade.

In a saturated market it looks tough for Plivensia to meet these forecasts even with a clean label. A cardiovascular warning, should the FDA require one, will make it even more difficult.

Editor's Note: This article discusses one or more securities that do not trade on a major U.S. exchange. Please be aware of the risks associated with these stocks.

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Rheumatoid Arthritis Safety Spotlight Widens - Seeking Alpha

Axial spondyloarthritis is a form of arthritis, and it is estimated around 700,000 people in the UK live daily with inflammatory back pain.

A study funded by Arthritis Research UK, aims to see how big a role patients medication plays in slowing down diagnosis of axial spondyloarthritis.

Most manage the pain with anti-inflammatory drugs such as ibuprofen or other slightly stronger variants while they wait to have the condition formally diagnosed by a rheumatologist.

Axial spondyloarthritis is an inflammatory condition affecting the spine and joints in the pelvis and in extreme cases can lead to parts of the spine fusing together.

It is diagnosed using MRI scans after which it cannot be cured but can be managed with special anti-inflammatory medication.

Researchers from the University of Aberdeen suspect that some people who undergo an MRI scan receive a negative diagnosis for axial spondyloarthritis because the Ibuprofen-like drugs they are taking sufficiently reduce the inflammation and hide the symptoms.

Early diagnosis is key to preventing axial spondyloarthritis getting worse but typically it can take between eight to 11 years to be properly identified.

Dr Gareth Jones, a musculoskeletal pain and spondyloarthritis expert and lead author on the study said: The fact that it can take up to 11 years to get a formal diagnosis of axial spondyloarthritis is obviously a concern.

There are good drugs to help people manage the condition but they are only available to people who get a correct diagnosis.

We suspect some people who are getting scanned for the condition, who still have anti-inflammatory drugs like ibuprofen in their system, are scanning negative for axial spondyloarthritis because the drugs are masking the true extent of the inflammation.

This study aims to show whether or not these drugs do mask the condition, and what proportion of sufferers could potentially be affected.

If we see that the painkillers are indeed leading to negative diagnoses then it will lend support to the argument that anyone receiving an MRI scan for back pain should halt their regular anti-inflammatory medication for a week or so prior to the scan.

This study will give an indication as to what proportion of axial spondyloarthritis are misdiagnosed as a result of the anti-inflammatory drugs.

Two hundred and fifty patients with the condition will be recruited to the study from around 20 different centres.

They will stop taking their regular anti-inflammatory drugs (Ibuprofen, etc) for a week and then given an MRI scan. They will then start taking the drugs again for six weeks before receiving a second MRI scan.

The researchers will then compare the proportion of people who scanned positive for axial spondyloarthritis with those who scanned negative for the condition once they are back on them.

Stephen Simpson, director of research and programmes at Arthritis Research UK, added: Axial spondyloarthritis is an incredibly painful condition, which can have a devastating impact on a persons everyday life.

For example, those with ankylosing spondylitis are three times more likely to stop work than the general population.

This new research will prompt conversations around whether patients should be asked to refrain from taking non-steroidal anti-inflammatory drugs immediately prior to an MRI scan and to rely, instead, on other pain relief during this specific period.

This research could lead to earlier diagnosis for people living with the condition, leading to earlier commencement of appropriate treatment and improved outcome.

Read more here:
Arthritis symptoms: Painkillers could be hiding THIS serious condition from doctors - Express.co.uk

According to Arthritis Research UK, over eight million people in the UK suffer from osteoarthritis - a condition perceived as only a disorder that affects the elderly.

A new walking programme for adults with arthritis and musculoskeletal conditions, made popular in the US, is to be trialled in the UK for the first time.

Walk With Ease was developed by the Arthritis Foundation in the US and has been shown to reduce arthritis-related symptoms such as pain, stiffness and fatigue, as well as improve strength, balance and walking pace.

The study is being funded as a joint working programme between ARUK and Pfizer Ltd, in partnership with the Chief Scientists Office of the Scottish Government and led by Dr Kathryn R Martin.

Dr Martin said: Walk With Ease has proven extremely successful in the US at reducing pain, stiffness and fatigue while improving physical functioning for those who have taken part.

We want to see if the ethos and logistics of the programme can be implemented in the UK.

This study will examine whether or not individuals with arthritis and musculoskeletal conditions living in the UK are willing to take part in such a programme and whether or not they feel it benefits them.

We are starting with Aberdeen and hope that if successful it can be rolled out across the UK.

This comes after Ruby James, 55, told Express.co.uk that exercise was the best way to reduce the symptoms of osteoarthritis.

Ruby said: I make sure I keep moving and exercising, as I always have, as I really feel that this makes my osteoarthritis more manageable.

Letters are currently being sent out from GP practices to potential participants for the six-week community-based programme.

Following an initial assessment of their mobility, participants will either receive the walking programme or a booklet outlining physical activity programmes in Aberdeen.

Those who receive the walking programme will be given a guidebook and the option to either walk with a group or on their own.

All participants will be re-assessed after six weeks and be posted a questionnaire three months later to see what effects the programme has had on them.

Some participants will also be asked to take part in an interview to discuss their experiences of being in the study.

Dr Natalie Carter, head of research liaison and evaluation at Arthritis Research UK, said: At Arthritis Research UK, we know that exercise, such as walking, can help to both prevent and improve the symptoms of arthritis.

Much more needs to be done to help support people with joint pain to stay active.

That is why our charity funds a number of studies focusing on exercise and movement, and we are pleased to be supporting the Walk With Ease study.

View original post here:
Arthritis symptoms could be eased with THIS therapy: New hope for sufferers - Express.co.uk

23 percent of all adults are affected by arthritis in the United States, and the symptoms associated with this chronic disease cost our country over 81 million dollars in annual medical bills.

Posted on July 31, 2017 by Kris Swartzendruber, Michigan State University Extension

Arthritis is a disease that affects over 54 million people in the United States. According to the Centers for Disease Control and Prevention, arthritis is the leading cause of disability, with related medical expenses reaching $81 billion. It is estimated that by the year 2040 nearly 26 percent of all adults (over 78 million people) will have arthritis.

The CDC also reports that arthritis significantly limits the workforce in this country. Because 60 percent of adults with this disease are of working age (18-64 years), arthritis can limit the type of work they do, and in many cases, keep them from working at all.

What is Arthritis?

Arthritis is chronic pain condition that is most commonly caused by inflammation of the tissue lining the joints (joints are located where two or more bones meet, such as elbows and knees). Symptoms vary, but the most common are pain, aching, swelling and stiffness.

There are over 100 diseases and conditions that are referred to as arthritis, but the most prevalent is osteoarthritis. Osteoarthritis usually occurs in older adults, affecting their fingers, knees and hips and can be a result of a previous injury to a joint. Other well-known types of arthritis include gout, lupus and rheumatoid arthritis. Rheumatoid arthritis and lupus occur when the bodys defense system doesnt work properly, and overtime, can affect multiple organs and cause widespread symptoms.

Who is affected by arthritis?

Arthritis does not discriminateit affects people of every age, race and gender. However, statistics show that the risk of arthritis increases with age and is more common among women than men. The CDC reports that arthritis commonly occurs with other chronic diseases with half of adults with heart disease or diabetes, and one-third of people who are obese, having arthritis. When a person has to deal with arthritis, along with another chronic health condition, it can negatively affect their quality of life and make it more difficult for doctors to treat and manage their diseases.

What can I do if I have arthritis?

The good news is, there are several ways to treat and reduce the symptoms associated with arthritis. If you, or someone you care for, is affected by arthritis, Michigan State University Extension recommends that you first talk with a doctor. The National Institute of Arthritis and Musculoskeletal and Skin Diseases says that only a doctor can diagnose and provide you with the best form of treatment associated with diabetes.

In addition to working with a healthcare provider, there are several things a person can do on their own to help control their pain and maintain their health. My next three news articles will focus on these arthritis self-management tools:

MSU Extension also provides high-quality and affordable education and resources related to the prevention and management of chronic disease.

This article was published by Michigan State University Extension. For more information, visit http://www.msue.msu.edu. To have a digest of information delivered straight to your email inbox, visit http://www.msue.msu.edu/newsletters. To contact an expert in your area, visit http://expert.msue.msu.edu, or call 888-MSUE4MI (888-678-3464).

Original post:
Understanding arthritis - Michigan State University Extension

H

ello, everyone, and how are you today? We are doing just fine, thank you, especially now that the Pharmalot campus has quieted down. Both Mrs. Pharmalot and the shortest person have left for their respective destinations, and the official mascots are snoozing contentedly in their usual spots. This leaves us to forage for interesting items for you. On that note, here are some tidbits. Have a wonderful day and do let us know about juicy developments

An experimental rheumatoid arthritis drug from Johnson & Johnson was linked to more deaths than a placebo, Food and Drug Administration staff reviewers wrote in an advance of a Wednesday expert panel meeting, Reuters writes. Other possiblesafety issues were similar to other drugs in the class, but the trend of increased overall mortality seems unique for this medicine, they wrote.The most common causes of death were major heart problems, infection, and malignancies.

This is a STAT Plus article and is only available to STAT Plus subscribers.To read the full story, subscribe to STAT Plus or log in to your account.Good news: your first 30 days are on us.

Pharmalot Columnist, Senior Writer

Ed covers the pharmaceutical industry.

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Pharmalittle: FDA staff cautious on J&J arthritis drug; Kite seeks EU nod for CAR-T drug - STAT