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These reports, excerpted and edited by Barrons, were issued recently by investment and research firms. The reports are a sampling of analysts thinking; they should not be considered the views or recommendations of Barrons. Some of the reports issuers have provided, or hope to provide, investment-banking or other services to the companies being analyzed.

American Water Works AWK-NYSE Buy Price $120 on Jan. 8 by Janney Montgomery Scott

American Water Works acquisition activity has been robust recently, with five announced deals in the past 30 days, totaling $108 million. Illinois and Pennsylvania accounted for the bulk of the deals, with New Jersey also generating a small transaction. This provides reinforcement to our positive investment thesis, and we maintain our Buy rating and $153 fair value on the shares, based on a price/earnings ratio of just over 35 times our 2021 earnings-per-share estimate of $4.32.

Newtek Business Services NEWT-Nasdaq Underperform Price $22.83 on Jan. 8 by Raymond James

We are downgrading our rating to Underperform from Market Perform.

We believe that there are a number of earnings headwinds ahead, with difficult growth targets in the Small Business Administration 7(a) lending business and a tougher comparison for Newteks merchant services segment. There is potential for upside versus our expectations from the conventional lending joint venture, but we do not believe that opportunity overwhelms the risk we see from other segments at this stage.

While there are always other levers to pull, such as one-time dividends from control subsidiaries, such earnings sources could result in a weaker quality of returns and [might] impact the sustainability of the current premium multiple valuation.

Royal Caribbean Cruises RCL-NYSE Overweight Price $130.44 on Jan. 7 by J.P. Morgan

We are raising our 2020 year-end price target to $154, from $135, on a slightly higher 2021 estimated P/E multiple of 13 times, from 11.4 times previously (no change to estimates).

A higher multiple is warranted, given that Royal proved in 2019 that it could comfortably grow core yields in the current elevated-capacity growth environment. Thirteen times is 1.0-1.5 times below the companys long-term average multiple and still well below the broader market multiple, and fair, in our view, given where we are in the economic cycle (late), and the inherently significant operating leverage in the cruise-line business model.

Cytokinetics CYTK-Nasdaq Buy Price: $10.83 on Jan. 6 by H.C. Wainwright & Co.

This morning, Cytokinetics announced the opening of enrollment in the Redwood-HCM study, a Phase 2 clinical trial testing CK-274 for the treatment of hypertrophic cardiomyopathy [HCM, a genetic disorder characterized by an abnormally thick heart muscle].

CK-274 has been optimized to ensure rapid symptom relief for HCM patients, which is particularly important in this patient population, and very valuable compared with gene-therapy approaches (which probably require longer periods of time, after administration, before becoming effective in relieving symptoms). We reiterate our Buy rating and price target of $30.

Lyft LYFT-Nasdaq Buy Price $43.58 on Jan. 2 by CFRA

Our Buy opinion reflects our outlook on Lyfts attractive end-markets/geographic exposure, scalable and recurring-based business model, as well as our expectation for considerable margin expansion through 2021. We see Lyft taking market share in the still nascent mobility-as-a-service space, with overseas expansion as a potential significant growth opportunity long term (the company is currently operating in the U.S. and Canada only). We like Lyfts initiatives and partnerships [for] autonomous capabilities.

Risks include slower-than-expected adoption of mobility-as-a-service, lower-than-expected revenue per active rider, greater-than-projected insurance costs, higher-than-expected expense growth, and greater-than-forecast intermediate-term cash burn.

Our 12-month $70 target price is based on our discounted-cash-flow analysis.

Bio-Techne TECH-Nasdaq Overweight (Volatile) Price $217.16 on Jan. 7 by Stephens

Since 2013, Bio-Techne has expanded its portfolio of assets from legacy reagents/diagnostics, moving into instruments and new clinical applications, which has resulted in growth accelerating from the low-single to the low-double digits. We see this continuing as Bio-Techne begins to benefit from cell- and gene-therapy capabilities, along with Exosome (liquid biopsy), in coming years. All of which should result in Bio-Techne achieving its targets of $1.2 billion in revenue and 40% margins by 2023.

Since CEO Chuck Kummeth joined the company in 2013, Bio-Techne has transformed into a more-diversified, higher-growth life-science company. Given the outlook for accelerating organic growth and expanding margins, we [give it] an Overweight (Volatile) rating, with a $250 price target.

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The Investment Allure of American Water Works - Barron's

Recommendation and review posted by Bethany Smith

ALBUQUERQUE, N.M., Jan. 6, 2020 /PRNewswire/ --Lovelace Biomedical, a non-profit contract research organization which has been specializing in various fields of biomedical research, including performing non-clinical studies in gene therapy, will host a webinar on their history and expertise in the field.

Experience the interactive Multimedia News Release here: https://www.multivu.com/players/English/8581851-lovelace-biomedical-gene-therapy-webinar/

For the past 12 years, Lovelace Biomedical has won competitive contracts for the Gene Therapy Resource Program (GTRP) at the National Heart Lung and Blood Institute (NHLBI), and has also received awards from the National Institute of Health (NIH), to perform studies to investigate and find new models for rare diseases with gene therapy studies. Now, reaching and focusing on commercial contracts, Lovelace continues to build on their history, and as a leader in pre-clinical studies with gene therapy as an important sector of their expertise.

Presenting the webinar is Lovelace's Chief Scientific Officer Jacob McDonald, Ph.D. and Dr. Janet Benson, who has led the Lovelace Gene Therapy Pharm/Tox program since 2007.

The webinar "Nonclinical Development in Gene Therapy, building on 12 years of the Lovelace Biomedical Center of Excellence in Gene Therapy" will be held on February 6, 2020 at 1 pm ET.

Join the Webinar

About Lovelace BiomedicalLovelace Biomedical is a contract research organization that helps pharmaceutical and biotechnology companies advance their complex drug development studies from the preclinical stage, and on to clinical trials. For over 70 years the organization has leveraged its multidisciplinary expertise in toxicology, gene therapy, medical countermeasures, and more, to provide excellence in preclinical research and fully understand the behavior of its clients' investigational products.

View original content:http://www.prnewswire.com/news-releases/lovelace-biomedical-to-host-webinar-on-their-history-and-expertise-in-the-rapidly-growing-field-of-gene-therapy-300981189.html

SOURCE Lovelace Biomedical

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Lovelace Biomedical to host webinar on their history and expertise in the rapidly growing field of Gene Therapy - BioSpace

Recommendation and review posted by Bethany Smith

The worldwide for Gene Therapy market is expected to grow at a CAGR of roughly +33% over the next five years.

Gene therapy is a test treatment that includes bringing genetic material into an individuals phones to battle or counteract ailment. Specialists are reading gene therapy for various maladies, for example, extreme joined immuno-insufficiencies, hemophilia, Parkinsons infection, disease and even HIV, through various methodologies. A gene can be conveyed to a cell utilizing a bearer known as a vector. The most widely recognized kinds of vectors utilized in gene therapy are infections. The infections utilized in gene therapy are modified to make them safe, albeit a few dangers still exist with gene therapy. The innovation is still in its infancy

Market Research Inc is one of the growing organizations whose proficiency is in making a far reaching analysis and reports an organization wishes to have. It offers the latest industry updates, industry patterns, and research implementations. At that point, it uses the reports they gather to design techniques and solutions for the organization. In addition to the fact that it is accessible in the realm of business, however, it likewise works over several industry divisions.

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Scope of the Report:

The Gene Therapy market is currently in a phase of transition as mobile operators seek to address increasing mobile traffic demands amidst economic uncertainties. This paradigm shift is bringing new challenges and opportunities to infrastructure vendors.

The key players covered in this study:

Market segment by Type, the product can be split into

Market segment by Application, split into

Different global regions such as North America, Latin America, Europe, Asia-Pacific, Africa, and India have been examined to get a better understanding of the competitive landscape.

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Key points of Gene Therapy Market Report

Key Questions Addressed by the Report

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Gene Therapy Market Aims to Expand at Double-Digit Growth Rate| Novartis AG, Gilead Sciences Inc., UniQure NV, Spark Therapeutics LLC, Bluebird Bio,...

Recommendation and review posted by Bethany Smith

NEW YORK--(BUSINESS WIRE)-- BTIG announced today that the BTIG Targeted Therapeutics Conference will take place on Monday, February 24, 2020 in New York. The conference will be hosted by BTIG Biotechnology Analysts Tim Chiang, Robert Bert Hazlett, Amanda Murphy, CFA, and Thomas Shrader, PhD, CFA, and BTIG Healthcare Strategy and Franchise Sales Specialists Mikhail Keyserman, CMT, CFTe, and Michael Karmiol. The firms private event will include one-on-one meetings and thematic panels featuring key opinion leaders and leadership team members from several embryonic and mature therapeutic companies in the space.

Some of the topics for discussion include:

Our moderators and panelists are each paving a unique path within the industry. Whether developing innovative therapeutic solutions, caring for patients or being a recognized thought leader, they are tasked with helping others better understand trends, products and whats next within biotechnology, commented David Lenchus, Director of Research at BTIG. We are excited about the lineup of speakers weve attracted for our 2020 event and look forward to helping our clients and partners make more informed trading and investment decisions.

BTIGs Corporate Access program hosts client events across the consumer, energy, financials, healthcare, real estate, shipping and technology, media and telecommunications sectors.

For more information about the conference, email info@btig.com. Please note that participants must be pre-registered to attend.

To access BTIG insights, contact a BTIG representative or visit http://www.btigresearch.com.

About BTIG

BTIG is a global financial services firm specializing in institutional trading, investment banking, research and related brokerage services. With an extensive global footprint and more than 600 employees, BTIG, LLC and its affiliates operate out of 18 cities throughout the U.S., and in Europe, Asia and Australia. BTIG offers execution, expertise and insights for equities, equity derivatives, ETFs and fixed income, currency and commodities (futures, commodities, foreign exchange, interest rates, credit, and convertible and preferred securities). The firms core capabilities include global execution, portfolio, electronic and outsource trading, transition management, investment banking, prime brokerage, capital introduction, corporate access, research and strategy, commission management and more. Disclaimer: https://www.btig.com/disclaimer.

To learn more about BTIG, visit http://www.btig.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200109005743/en/

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BTIG to Host Targeted Therapeutics Conference on February 24, 2020 in New York - BioSpace

Recommendation and review posted by Bethany Smith

The eighth edition of the annual Global Young Scientists Summit (GYSS) welcomes one of its largest groups of young scientists yet, numbering 320. The summit will give them a chance to interact with 17 prominent leaders in science and technology.

The 17 award-winning speakers from a wide variety of research fields will gather in Singapore from across the world. They include recipients of the Nobel Prize as well as other prestigious science and technology awards.

Organised by the National Research Foundation Singapore, GYSS 2020 will be held at the Matrix building at Biopolis from next Tuesday to Friday.

The theme is Advancing Science, Creating Technologies For A Better World.

The scientists will share their knowledge and experience through plenary lectures, panel discussions and interactive group sessions.

Topics to be discussed include how research can develop solutions to current global challenges, from growing antibiotic resistance to a looming shortage in data storage capacity.

The young scientists can also visit universities and research institutions in Singapore to better understand how science and technology solutions are being developed to meet national challenges.

Among the highlights of the summit is that it will have Professor Alain Fischer, chair of experimental medicine at the College de France in Paris, as its special guest speaker.

He was one of the first scientists to successfully use gene therapy to treat a rare form of severe combined immunodeficiency, also known as the "bubble boy disease", named after a patient who lived for years in a plastic bubble filled with filtered air.

Mr Ashish Rauniyar, a PhD research fellow at Oslo Metropolitan University, will be attending GYSS 2020. He said that he is looking forward to meeting talented young researchers from around the world and engaging with the speakers.

"The opportunity to talk to them about their scientific experiences is an amazing opportunity," he added.

Members of the public will not miss out either, as they can hear from two speakers at two free public lectures. One will be held at the Singapore University of Technology and Design (SUTD), and the other at the National University of Singapore (NUS).

At SUTD next Tuesday, the public can listen to a talk by the winner of the 1985 Nobel Prize in physics, Professor Klaus von Klitzing, about how physics has contributed to the realisation of a new International System of Units.

And at NUS next Thursday, Turing Award winner Leslie Lamport will give a talk entitled "If you're not writing a program, don't use programming language".

Dr Lamport won the Turing Award, regarded as the Nobel Prize of computing, in 2013.

To sign up or learn more about the two lectures, go to http://www.nrf.gov.sg/gyss/programme/ public-lectures

There are limited seats available, and Dr Lamport's lecture at NUS has already hit the maximum number of registrations.

Read the original here:
Over 300 scientists from around the world to gather in Singapore - The Straits Times

Recommendation and review posted by Bethany Smith

Whoever comes out on top of the current race to gain pioneering approvals for new AAV-delivered gene therapies will have to look over their shoulders to watch the next tech wave forming on the horizon for gene therapy 2.0.

One of those next-gen players, Generation Bio, just brought in $110 million of venture cash to cover the cost of the rest of their preclinical journey toward something completely new in the field. The latest round brings the biotech which now has about 80 staffers up to $235 million in total since its inception about 3 years ago. That will fuel the rest of its preclinical stage of development as it looks to break into human studies in the back half of 2021.

That kind of 4-plus year timeline before the first human dosing could test the endurance level of a venture player. But Generation CEO Geoff McDonough looks over the past 2 years advancing a new lipid nanoparticle delivery system for their closed-end DNA therapies working to the day when gene therapies can be produced and sold for far less than the $2 million-or-so price tag today and sees lots of fast-paced advances.

I think the reality is we didnt have an expectation at the outset (on timelines), McDonough tells me. Recognizing the novel work needed to build the platform, the investors knew it would take time and money to bring them up to a GMP level.

I would say for a 40-year problem, adds the CEO, 2 years seems pretty good.

The founding tech at Generation was designed to do what AAV treatments do in the nucleus, offering enduring expression, while allowing manufacturing at a biologic scale with a more economical, capsid-free production method. Taking a page from the tech handbooks at companies like Alnylam and Moderna, theyre building a gene therapy that they believe can do much better than the fragile, one-time-only pioneers. And without the $1 million production cost that keeps wholesale prices in the low 7-figure range.

Theyre looking for much greater economy, eventually taking these therapies to much broader ailments and out of the realm of rare diseases with a new approach that they believe can be infinitely redosable on an as-needed basis.

Thats the big picture.

Generations team is working on 2 lead programs for hemophilia A and phenylketonuria (PKU) to go into IND-enabling studies. Theyve now identified Wilson disease and Gaucher disease as likely starting points for the next steps as they move past the liver to skeletal muscle and the retina and then other tissues. And McDonough the former CEO at Sobi is looking down the road 12 to 18 months when hed like to turn to the public markets with an IPO to fund the first clinical-stage work.

In the meantime, hed like to concentrate on opening another new chapter of the company on the dealmaking side.

It felt very important not to partner initially, says McDonough. The investors wanted to retain ownership of platform. We just had tremendous good fortune we didnt need to do that for finance reasons. But now that they have a better grasp of the technology and what needs to be done, its time to partner probably later in the year.

T. Rowe Price funds and accounts led the round, with Farallon and Wellington Management Company jumping in alongside. Existing investors Atlas Venture, Fidelity, Invus, Casdin, Deerfield, Foresite Capital and an entity associated with SVB Leerink came back to stay in the syndicate. Cowen served as exclusive placement agent for the offering.

Originally posted here:
With $110M to add to the bankroll, Generation Bio sets its sights on engineering a revolution in the gene therapy field - Endpoints News

Recommendation and review posted by Bethany Smith

Advance Market Analytics recently introduced Global Gene Therapy Market Report tracks the major market procedures including Market Overview, Business Revenue, Introduction, Gross profit & business strategies opted by key market players. The report also focuses on market size, volume and value, shipment, price, interview record, business distribution etc. These data help the consumer know about the competitors better. It also covers different industries clients information, which is very important to understand the market

According to AMA, the Global Gene Therapy market is expected to see growth rate of 19.3%

Summary: Genes are composed of DNA (Deoxyribonucleic Acid) that contains important information for making proteins which are vital for optimal human body function. Certain gene alterations result in these proteins not being made properly & so can lead to genetic disorders. Gene therapy is a new technique which uses genes to prevent or treat disease. Over the coming years, this technique may permit doctors to treat a disorder by inserting a gene into a patients cells rather than using drugs or surgery. This new therapy works by repairing, repressing, or replacing dysfunctional genes which cause disease with aiming of reestablishing normal function. Gene therapy is a striking area for drug development. With the right target & approach, it can address the original cause of a severe diseases.

The major players in Global Gene Therapy Market: Novartis International AG (Switzerland), Kite Pharma, Inc. (United States), Spark Therapeutics Inc. (United States), GlaxoSmithKline PLC (United Kingdom), Bluebird bio Inc. (United States), Genethon (France), Transgene SA (France), Applied Genetic Technologies Corporation (United States), Oxford BioMedica (United Kingdom)

These major players have adopted various organic as well as inorganic growth strategies such as mergers & acquisitions, new product launches, expansions, agreements, joint ventures, partnerships, and others to strengthen their position in this market.

Free Sample Report + All Related Graphs & Charts of Global Gene Therapy Market: https://www.advancemarketanalytics.com/sample-report/16796-global-gene-therapy-market

Market Drivers

Restraints

Opportunities

This report describes the development of the industry by upstream & downstream, industry overall and development, key companies, as well as type segment & market application and so on, and makes a scientific prediction for the development industry prospects on the basis of analysis, finally, analyses opportunities for investment in the industry at the end of the report.

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The Global Gene Therapy Market segments and Market Data Break Down are illuminated below:

Type (Somatic Cell Gene Therapy, Germ Cell Gene Therapy), Application (Neurological Disorders, Rare Diseases, Oncological Disorders, Cardiovascular Diseases, Infectious disease, Other Diseases), Vector Type (Viral Vector, Non-viral Vector)The regional analysis of Global Gene Therapy Market is considered for the key regions such as Asia Pacific, North America, Europe, Latin America and Rest of the World. North America is the leading region across the world. Whereas, owing to rising no. of research activities in countries such as China, India, and Japan, Asia Pacific region is also expected to exhibit higher growth rate the forecast period 2019-2025.

What Global Gene Therapy Market Report Contributes?

In short, the report is a vital guide for understanding the Global Gene Therapy industry accomplishments to the extent each significant perspective like all around learning of the genuine players and benefactors influencing the Global Gene Therapy Market advertise. The examination moreover bases on current Global Gene Therapy point of view, bargains edge, inconspicuous components of the Global Gene Therapy showcase movement.

Key highlights of the Study:

Comprehensive information about factors that will challenge the growth

Get More Information about Global Gene Therapy Market: https://www.advancemarketanalytics.com/reports/16796-global-gene-therapy-market

There are 15 Chapters analyzing in detail the Global Gene Therapy market.

Chapter 1, to describe Global Gene Therapy Introduction, product scope, market overview, market opportunities, market risk, market driving force;

Chapter 2, to analyze the top manufacturers of Global Gene Therapy, with sales, revenue, and price of Global Gene Therapy, in 2012 to 2018;

Chapter 3, to display the competitive situation among the top manufacturers, with sales, revenue and market share in 2012 to 2018;

Chapter 4, to show the global market by regions, with sales, revenue and market share of Global Gene Therapy, for each region, from 2012 to 2023;

Chapter 5, 6, 7,8 and 9, to analyze the key regions, with sales, revenue and market share by key countries in these regions;

Chapter 10 and 11, to show the market by type and application, with sales market share and growth rate by type, application, from 2012 to 2023;

Chapter 12, Global Gene Therapy market forecast, by regions, type and application, with sales and revenue, from 2018 to 2023;

Chapter 13, 14 and 15, to describe Global Gene Therapy sales channel, distributors, traders, dealers, Research Findings and Conclusion, appendix and data source.

Finally, this report covers the market landscape and its growth prospects over the coming years, the Report also brief deals with the product life cycle, comparing it to the relevant products from across industries that had already been commercialized details the potential for various applications, discussing about recent product innovations and gives an overview on potential regional market shares.

Key questions answered

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Definitively, this report will give you an unmistakable perspective on every single reality of the market without a need to allude to some other research report or an information source. Our report will give all of you the realities about the past, present, and eventual fate of the concerned Market.

Thanks for reading this article, you can also get separate chapter wise or region wise report versions including North America, Europe or Asia.

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Excerpt from:
Gene Therapy Market is expected to see growth rate of 19.3% - BulletintheNews

Recommendation and review posted by Bethany Smith

LONDON--(BUSINESS WIRE)--Technavio has been monitoring the global RNAi technology market since 2019 and the market is poised to grow by USD 1.96 billion during 2020-2024, progressing at a CAGR of around 22% during the forecast period. Request free sample pages

Read the 168-page report with TOC on RNAi Technology Market Analysis Report by Application (Cancer, Infectious diseases, Ophthalmic diseases, Genetic diseases, and Other diseases), Geography (Asia, Europe, North America, and ROW), and the Segment Forecasts, 2020-2024.

https://www.technavio.com/report/RNAi-technology-market-industry-analysis

The market is driven by the increasing adoption of RNAi technology for undruggable targets. In addition, recent advances in whole-genome RNAi screening is anticipated to boost the growth of the RNAi technology market.

RNAi technology is emerging as a promising therapeutic platform for the treatment of various diseases. This is due to efficient delivery mechanisms, high specificity in the targeted section, and the knockdown approach and safety of RNAi-based therapies. Moreover, RNAi technology has made every gene in the human genome as a druggable target, which is creating new opportunities for vendors to develop a wide range of targeted therapies. This is attracting many drug manufacturers to focus on the development of RNAi-based targeted cancer therapies. Several early-phase trials have reported clinical responses in the case of RNAi therapies directed against targets such as Kinesin spindle protein (KSP) and vascular endothelial growth factor (VEGF). Therefore, the increasing adoption of RNAi technology for undruggable targets is expected to fuel market growth during the forecast period.

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Major Five RNAi Technology Market Companies:

Alnylam Pharmaceuticals Inc.

Alnylam Pharmaceuticals Inc. operates in a single reporting segment, the discovery, development, and commercialization of RNAi therapeutics. The company offers ONPATTRO (patisiran) and GIVLAARITM (givosiran), and other RNAi therapeutics such as Fitusiran, Inclisiran, Lumasiran, and Vutrisiran in late stage (Phase 2 to Phase 4) clinical development pipeline.

Arbutus Biopharma Corp.

Arbutus Biopharma Corp. offers RNAi based therapeutic solutions for drug development, discovery, and commercialization to patients suffering from hepatitis B infection. Some of the key offerings of the company include LNP Delivery Platform and RNA Trigger Technologies.

Arrowhead Pharmaceuticals Inc.

Arrowhead Pharmaceuticals Inc. uses RNAi-based technologies in the development of therapeutics for treating intractable diseases. The company also offers licenses for its patented RNAi technology through collaboration. Targeted RNAi Molecule (TRiM) Platform is the key offering of the company.

Bio-Rad Laboratories Inc.

Bio-Rad Laboratories Inc. operates the business across segments such as Life Science and Clinical Diagnostics. Some of the key offerings of the company include siLentFect Lipid Reagent for RNAi and siLentMer.

Dicerna Pharmaceuticals Inc.

Dicerna Pharmaceuticals Inc. develops RNAi-based therapeutics using GalXC RNAi platform. The company also offers licensed use of their platforms through collaboration. GalXC Technology Platform is the key offering of the company.

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Related Reports on Healthcare include:

Global Cancer Biologics Market Global cancer biologics market by product (monoclonal antibodies, vaccines, cell and gene therapy, and others) and geography (Asia, Europe, North America, and ROW).

Global RNA and DNA Extraction Kit Market Global RNA and DNA extraction kit market by product (RNA extraction kit and DNA extraction kit) and geography (Asia, Europe, North America, and ROW).

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Global RNAi Technology Market 2020-2024 | Evolving Opportunities with Alnylam Pharmaceuticals Inc. and Arbutus Biopharma Corp. | Technavio - Business...

Recommendation and review posted by Bethany Smith

The comprehensive report published by Persistence Market Research offers an in-depth intelligence related to the various factors that are likely to impact the demand, revenue generation, and sales of the CNS Gene Therapy Market. In addition, the report singles out the different parameters that are expected to influence the overall dynamics of the CNS Gene Therapy Market during the forecast period 2018 2028.

As per the findings of the presented study, the CNS Gene Therapy Market is poised to surpass the value of ~US$ XX by the end of 2029 growing at a CAGR of ~XX% over the assessment period. The report includes a thorough analysis of the upstream raw materials, supply-demand ratio of the CNS Gene Therapy in different regions, import-export trends and more to provide readers a fair understanding of the global market scenario.

ThisPress Release will help you to understand the Volume, growth with Impacting Trends. Click HERE To get SAMPLE PDF (Including Full TOC, Table & Figures) athttps://www.persistencemarketresearch.co/samples/27514

The report segregates the CNS Gene Therapy Market into different segments to provide a detailed understanding of the various aspects of the market. The competitive analysis of the CNS Gene Therapy Market includes valuable insights based on which, market players can formulate impactful growth strategies to enhance their presence in the CNS Gene Therapy Market.

Key findings of the report:

The report aims to eliminate the following doubts related to the CNS Gene Therapy Market:

Get Access To TOC Covering 200+ Topics athttps://www.persistencemarketresearch.co/toc/27514

key players and product offerings

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Continued here:
CNS Gene Therapy Market: Facts, Figures and Analytical Insights, 2018 2028 - Expert Recorder

Recommendation and review posted by Bethany Smith

Searching for new methods of attacking serious central nervous system diseases, Biogen has signed a deal that could be run into the hundreds of millions of dollars to essentially license a transcriptional map of the brain and scan it for new drug targets.

The partnership, worth $15 million upfront and far more in milestones, is with a new bioinformatics startup called CAMP4 Therapeutics. Founded in 2018 year by the Whitehead Institutes Richard Young and Harvard Medical Schools Leonard Zon, CAMP4 takes genes considered affiliated with a disease and maps out the various ways cells express those genes and turn them on or off. They then take that map and in the biological equivalent of stretching a war map across a table in a bunker mark up all the different methods of attack.

The more we know about a gene, it doesnt mean we can drug that gene, CEO Josh Mandel-Brehm, who worked at Biogen before joining CAMP4, told Endpoints News. Many genes are not quote-un-quote druggable, so then we have an interesting problem: I think I understand the genetics of the disease, but how do I drug it? And thats where our map solves for this problem.

Alongside the $15 million upfront payment, CAMP4 will be eligible for up to $96 million in milestones for each of the initial targets it supplies, and up to $173 million for each subsequent one.

Biogen is not disclosing what genes or diseases they will target, but the collaboration will focus on microglial cells. These macrophages are found in the central nervous system and have long been considered a key factor in neurodegenerative diseases such as Alzheimers and Parkinsons.

Biogen will bring CAMP4 the genes it believes are central to diseases in the central nervous system, and CAMP4 will inform them all the ways the gene is being expressed and where Biogen might go about turning up or down the expression.

So you say hey I want to move this particular gene for this disease, how should I do that? I want to change the expression of it,' Mandel-Brehm said, describing how they work with partners. Well show you the different nodes, if you will: hey you can drug this, you can drug this, you can drug this. Its modality agnostic.

CAMP4, founded out of Polaris Partners, is one of a couple of new biotechs that build on a discovery Young had back in 2015. The longtime transcriptional biologist published work showing that cells modify gene expression using a series of insulated neighborhoods, 3-D loops of DNA that control gene expression within them. CAMP4 maps those (along with other aspects of the transcriptome). Flagship Pioneerings new Omega Therapeuticsalso tries to drug them.

Since launching in 2018, CAMP4 has done extensive work on mapping genes for liver diseases, including NASH, as part of an in-house development program. Theyve also partnered with synthetic RNAi company Alnylam and are working to expand their maps of cells in the brain, heart, muscles, immune system, blood and kidneys, which they could then license out or use to develop drugs in-house.

The way I think about the diseases were going after is BFP: Big Fucking Problems,Mandel-Brehm said, citing NASH and other diseases that lack effective treatments. And Its well-validated and understood: If youre drugging something thats tied to the gene, youre much more likely to be successful.

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Targeting the BFPs of the CNS, Biogen licenses map of genetic neighborhoods in the brain - Endpoints News

Recommendation and review posted by Bethany Smith

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Gene Therapy Market Growth to 2025 - Instanews247

Recommendation and review posted by Bethany Smith

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Hemophilia Gene Therapy Market- Growth Opportunities by Manufacturers, Regions, Type and Application; Trends Forecast to 2026 - Expert Recorder

Recommendation and review posted by Bethany Smith

Polaris Partners has snagged Eli Lilly vet Darren Carroll as a partner. Carroll spent 22 years at Eli Lilly before making the switch to the investment firms Boston office. Most recently, Carroll served as SVP of corporate business development at Lilly, where he oversaw the $8.1 billion acquisition of Loxo Oncology and the divestiture of animal health company Elanco. Carroll is credited with forming Lilly Asia Ventures and was the founding CEO of Lilly spinout InnoCentive.

Mirati Therapeutics a keenly watched rival to Amgen in the KRAS G12C field has made some changes in its executive team. The biotech welcomed former Spark Therapeutics exec Daniel Faga as COO and Bristol-Myers Squibb vet Benjamin Hickey as CCO. In addition, the company promoted Vickie Reed to SVP, finance and chief accounting officer and Jessica Corson as VP, business development both joined in 2013.

MBX Biosciences has tapped co-founder and board director Kent Hawryluk to run the company as president and CEO. Hawryluk hops over after a stint as CBO of Avidity Biosciences, which he also co-founded. As a partner of Twilight Venture Partners, his previous experience includes helping launch Marcadia Biotech (acquired by Roche) and MB2 (acquired by Novo Nordisk). In addition to Hawryluk, the company has brought on Greg Davis as VP, product development. Davis most recently had a stint as VP of CMC, regulatory quality at Calibrium (acquired by Novo Nordisk).

Months after stepping down as the North America president of Sobi, Rami Levinhas been appointed to be CEO at Saniona focused on the treatment of eating disorders and diseases of the central nervous system. Levin will succeed Jrgen Drejer, who will transition to the role of CSO. Prior to his 5-year tenure at Sobi, Levin headed marketing at Merck Serono.

J&J-partnered Arrowhead Pharmaceuticalshas tappedJames Hassard as the companys first CCO to begin mapping a commercial path for its RNAi therapies. Most recently, Hassard served as SVP of marketing and market access at Coherus BioSciences. Prior to that, he held stints at Medivation, Amgen and Schering Plough.

As its lead therapy clears a pivotal Phase II, bringing it closer to its first BLA,ADC Therapeutics has tapped Joseph Camardo as head of medical affairs. In his new role, Camardo will be responsible for ensuring optimal medical impact for loncastuximab tesirine (ADCT-402) among patients with B-cell non-Hodgkin lymphoma. Camardo joins the Swiss biotech after a stint as SVP of Celgenes global health, which followed positions at Forest Research Institute and Wyeth Research (now Pfizer).

After scrapping their Phase I program and having their CSO hit the exit last May, German biotech Affimedhas brought on Andreas Harstrick as CMO. Most recently, Harstrick served in the same role at Molecular Partners. Harstrick is an alumnus of ImClone, Lilly Oncology and Merck Serono.

Dyve Biosciences has named Pfizer vet Chuck Harbert as CSO following the recent clearance the company received to begin a Phase II trial for their drug, DYV-700. Harbert spent three decades at Pfizer before transitioning to his new role. During his time at the pharma giant, Harbert served as VP, US exploratory development and strategic planning and had roles at Pfizer Central Research. In addition, Harbert was the co-inventor of Zoloft.

Biocon Biologics, a subsidiary of Biocon, has appointed MB Chinappa as CFO. Chinappas appointment comes at the same time as private equity fund True Norths acquisition of 2.44% of the company in a deal worth $74 million. Chinappa makes the jump from one of Biocons other subsidiaries, Syngene, where he was president (finance) and CFO. Chinappa led the Syngenes IPO in 2015.

Momenta Pharmaceuticalshas added the title of CFO to the companys CBO Young Kwon. Kwon joined the company in 2011 and prior to that, he worked at Biogen and Advanced Technology Ventures.

AVEO which had their drug tivozanib spurned once again by the FDA two months ago has appointed Erick Lucera to the position of CFO. Prior to joining the company, Lucera served in the same role at Valeritas. Before that, he was the CFO, treasurer and secretary of Viventia Bio (acquired by Eleven Biotherapeutics and now Sesen Bio).

Candel Therapeutics focused on the development of viral immunotherapies for multiple tumor indications has brought on Genzyme vet Sandra Poole as COO. Poole was most recently COO of LogicBio Therapeutics, after servingas EVP of technical operations and commercial development at ImmunoGen. During her time at Genzyme, Poole oversaw biologics manufacturing.

Antengene, a US-China biotech backed by Celgene, has snagged one of its top execs in China. John Chin, Celgenes general manager in the country, has joined as chief business officer just a few days before former Celgene CEO Mark Alles was appointed to the board of directors. Chins resume spans Aventis, Bristol-Myers Squibb and Merck.

Rubius Therapeutics has appointed autoimmunity and translational immunology expert Laurence Turka as the biotechs first CSO, joining its mission to pioneer a new class of medicines called red cell therapeutics. Turka hops over to the company from Rheos Medicines, where he was a co-founder and CSO. Turka was an entrepreneur-in-residence at Third Rock Ventures, turning to the VC life after an academic career that put him at the head of the American Society of Transplantation and a professor at Harvard Medical School and Massachusetts General Hospital.

Abeona Therapeutics which had some good news last month when the FDA lifted its hold on the companys butterfly disease therapy is parting ways with its executive chairman Steven Rouhandeh, revealed in an SEC filing.

Eiger BioPharmaceuticals has appointed Eldon Mayer as EVP and CCO. Mayer joins just as the company has initiated an NDA for its drug ionafarnib to treat Hutchinson-Gilford progeria syndrome (HGPS or progeria) and progeroid laminopathies. Mayer had the same job at Rigel Pharmaceuticals. Previously, Mayer was the SVP of commercial operations at Questcor Pharmaceuticals (acquired by Mallinckrodt).

Cancer-focused Cellectar Biosciences has named Igor Grachev as CMO. Grachev previously served as global development leader and head of innovative clinical trials initiative, R&D for TEVA Pharmaceuticals. His prior employers include GE Healthcare, Novartis, GSK, Merck, Schering PLough, Sanofi-Aventis and BioClinica.

The immunology experts at IFM Therapeutics who brought in $55.5 million in new venture backing last month has wooedMichael Cooke from Magenta Therapeutics as CSO. BeforeMagenta, Cooke was a founding scientist at the Genomics Institute of the Novartis Research Foundation.

Anti-edema therapies-focused Aeromics has brought on Joseph Schindler as its first CMO. Schindler is an associate professor of neurology and of neurosurgery at Yale University School of Medicine, where he is the clinical chief of the division of vascular neurology. He is also the director of the comprehensive stroke program and telestroke services at Yale New Haven Hospital.

Indias Vyome Therapeutics which back in 2016 raised $14 million for its R&D work on skin diseases has named Craig Tooman COO and CFO. Prior to joining Vyome, Tooman served as the CEO of Aratana Therapeutics. In addition, Tooman has served as founder and CEO of Avanzar Medical and CFO at Ikaria and Enzon Pharmaceuticals.

Baltimore-based startup WindMIL Therapeutics spun out of John Hopkins has named Bristol-Myers vet Karen LaRochelle as SVP, corporate & business development and promoted Patrick Dougherty to SVP, strategy, planning & operations. LaRochelle previously served as CBO of PsiOxus Therapeutics and during her time at BMS served as global head of negotiations and head of business development in China. Prior to his time at WindMIL, Dougherty served as chief of staff to the SVP R&D pipeline for pharmaceuticals R&D at GSK.

Vor Biopharma the biotech co-founded by Siddhartha Mukherjee to pioneer a new type of cancer cell therapy has made two new additions to its team. Amy Mendel joins as chief legal officer and Tania Philipps joins as VP of people. Mendel was the VP & associate general counsel for Ziopharm Oncology while Philipps was the VP and head of human resources at Tango Therapeutics.

JSR life sciences company Selexis has promoted Yemi Onakunle to the position of CBO. Onakunle joined the company in 2012 from Diosynth RTP, where he was the director of commercial development. His other stints include roles at Lonza Custom Manufacturing and Bachem Americas.

Immuneering whose current pipeline focuses on diseases such as cancer cachexia and metastasis has brought on Howard Kaufman as head of research and development. Kaufman, the former president of the Society for Immunotherapy of Cancer (SITC), was most recently CMO at Replimune Group. Kaufmans hiring comes a few weeks after the appointment of Scott Barrett to CMO at Immuneering.

CAR-T-focused Innovative Cellular Therapeutics (ICT) has welcomed Christopher Ballas onboard as SVP of manufacturing. Ballas career has brought him to Rocket Pharmaceuticals, Cook Medical and WuXi AppTec.

Allurion Technologies has recruited Whitney Cypes, former senior director of marketing for Insulet Corporation, as VP of global marketing and Chris Aronson, formerly of Restoration Robotics, as VP of North American sales. In addition, Benoit Chardon was promoted to EVP of commercial.

Eliot Forster-led cancer biotech F-star Therapeutics has appointed Edward Benz, the president and CEO emeritus of the Dana-Farber Cancer Institute, and Minerva exec Geoff Race to the board of directors.

Elias Zerhouni, the former R&D head at Sanofi and ex-NIH director, has taken up a seat at Waypoint Capitals board. The Geneva firm is led by Swiss billionaire and former Serono chief Ernesto Bertarelli and devised an $832 million buyout deal to take allergy drugmaker Stallergenes Greer private.

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Eli Lilly vet jumps aboard Polaris Partners; Mirati shakes up its leadership team with new hires and promotions - Endpoints News

Recommendation and review posted by Bethany Smith

The prospect of using the popular genome-editing tool CRISPR to treat a host of diseases in people is moving closer to reality.

Medical applications of CRISPRCas9 had a banner year in 2019. The first results trickled in from trials testing the tool in people, and more trials launched. In the coming years, researchers are looking ahead to more sophisticated applications of CRISPR genome editing that could lay the foundation for treating an array of diseases, from blood disorders to hereditary blindness.

But although the results of clinical trials of CRISPR genome editing so far have been promising, researchers say that it is still too soon to know whether the technique will be safe or effective in the clinic.

Theres been a lot of appropriate caution in applying this to treating people, says Edward Stadtmauer, an oncologist at the University of Pennsylvania in Philadelphia. But I think were starting to see some of the results of that work.

It has only been seven years since researchers discovered thata molecular defence system called CRISPRCas9, which microbes use to fend off viruses and other invaders, could beharnessed to rewrite human genes.

Since then gene-editing has attracted attention for its potential to modify embryosan application that is ethically and legally fraught if those embryos are destined tobecome human beings. But in parallel, scientists have been testing CRISPR's much less controversial ability to disable or correct problematic genes in other cells in order to treat a host of diseases.

In 2016, Chinese researchers announced that they hadtreated the first person with a CRISPRCas9 therapydesigned to fight cancer. In cells extracted from a participant's blood, the researchers disabled the gene that codes for a protein called PD-1,which holds the immune system in checkbut can shield cancer cells in the process. The scientists then reinjected the cells.

By 2019, the US governments clinicaltrials.gov database listed more than a dozen active studies that are testing CRISPRCas9 as a treatment for a range of diseases from cancer to HIV and blood disorders.

So far, too few people have been treated in these trials to draw any firm conclusions about the safety of CRISPRCas9 therapies or how well they work. Preliminary results from two trialsone in which gene-edited blood cells were transplanted into a man to treat HIV infection, and the other in which they were transplanted into three people to treat some forms of cancershowed no signs of clinical improvement.

In both cases, the transplanted cells flourished in the bone marrow of recipients, without any serious safety concerns, but did not produce a clear medical benefit. In the man treated for HIV, the researchers attempted to use CRISPR to disable a protein that many strains of HIV use to enter cells. But only 5% of the transplanted cells were editednot enough to cure disease, the researchers reported in September. The study has been placed on hold while researchers explore ways to boost that percentage, says Hongkui Deng, a stem-cell researcher at Peking University in Beijing and a lead author of the work.

There are early hints that another trial might meet with more success. CRISPR Therapeutics in Cambridge, Massachusetts, and Vertex Pharmaceuticals in Boston, Massachusetts, have treated two people with the genetic disorders sickle-cell anaemia and -thalassaemia. Both deplete oxygen-carrying haemoglobin molecules in the blood: the idea is to use CRISPR to disable a gene that otherwise shuts off production of another form of haemoglobin. Early results suggest that the treatment might have eased some symptoms of the disorders, but the participants will need to be followed for a longer period to be sure.

Other researchers are already itching to move beyond editing cells in a dish. The challenge is in finding ways to transport the gene-editing machinery to where it is needed in the body, says John Leonard, chief executive of Intellia Therapeutics, a biotechnology company in Cambridge, Massachusetts, that is focused on CRISPRCas9 genome editing. The delivery approach is so important.

Last July, the pharmaceutical companies Editas Medicine in Cambridge, Massachusetts, and Allergan in Dublin launched a trial to treat the genetic disorder Leber congenital amaurosis 10, which can cause blindness, by editing eye cells. Researchers will inject into the eye a virus containing DNA that encodes the CRISPR genome-editing machinery, bypassing the need to guide those tools through the bloodstream to the specific tissues. The virus will be responsible for carrying the genome-editing tools into cells. It is the first trial to attempt CRISPRCas9 gene editing inside the body, and early results could be reported this year.

That would be a landmark moment for the field, and could pave the way for future trials targeting other organs, says Charles Gersbach, a bioengineer at Duke University in Durham, North Carolina. But he and others say that they hope researchers will eventually move away from using viruses to shuttle genome-editing machinery into cells. Deactivated viruses can still sometimes provoke immune responses, and can only carry a limited amount of DNA.

What's more, some gene-editing tools are currently too large to fit inside commonly used gene-therapy viruses, says chemical biologist Andrew Anzalone at the Broad Institute of MIT and Harvard in Cambridge, Massachusetts. These include the souped-up CRISPR systems calledprime editorsthat were first reported in late 2019and might prove to be more precise and controllable than CRISPRCas9.

Intellia is looking for a way around the viruses. The company has partnered with Swiss pharmaceutical giant Novartis to develop fatty nanoparticles that can protect genome-editing molecules as they travel through the bloodstream, but also pass through the membranes of target cells.

These particles tend to accumulate in the liver, and researchers are working to develop particles that infiltrate other tissues, such as muscle or the brain. But for now, Intellia will focus on liver diseases, says Leonard, and plans to launch its first trial of the technology this year. Its crawl before you walk, so to speak, he says.

None of the technologies currently being tested is what researchers envision for the long-term applications of genome-editing, says Gersbach. The approaches that people are taking are the things that we can do today, he says, but not what we would do if we could design the ideal drug.

Leonard says that when he meets with investors, they often demand to know what medical advances will be made in the next six months. We do our best to describe that, but I always end it by saying, Can you imagine a future without gene editing? he says. I have yet to meet the person who says, yes.

This article is reproduced with permission and wasfirst publishedon January 6, 2020.

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Quest to Use CRISPR Against Disease Gains Ground - Scientific American

Recommendation and review posted by Bethany Smith

Bioengineering, once viewed primarily as an academic discipline, is growing up.

Our ability to engineer biology is on the verge of changing the landscape of health and health care. Tools and treatments that are engineered, not discovered CAR-T therapies for cancer, CRISPR for gene editing, stem cell therapies, and more are now making their way not just into new startups but into established industry. Just look at the first-generation CAR-T companies that have been acquired by major biopharma companies, like Bristol-Myers Squibb/Celgene acquiring Juno or Gilead acquiring Kite.

The acquirers, massive organizations built on the foundations of discovery, are now ingesting companies built with engineering DNA. These are two extremely different mindsets. For decades, biopharma companies essentially used scientists to build products, because there was no means to engineer them. The intersection of these worlds is driving us into the future.

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Here come the culture clashes.

These emerge every time major new technologies disrupt an industry. Think of the early days of oil and the evolution as the industry matured from wildcatters one man, one rig, one highly risky and unreliable process to Rockefellers and Rothschilds and a highly sophisticated engineering and discovery process that used every advanced technology and enterprise tool available.

In biopharma and health care today, the old culture of discovery the idea that science is driven by discovering new knowledge (hypothesis > test > repeat) is clashing with the new culture of engineering (design > test > iterate). This clash encompasses how everything is handled, from identifying biological targets to designing clinical trials and even to how we access health care.

Knowing that these clashes are coming will help smooth the way as the biopharma industry integrates bioengineering deeper and more broadly. I see four key clashes worth noting.

We cant yet take for granted a common understanding that we can engineer biology. In spite of bioengineering departments flourishing at revered institutions like Harvard and MIT and Stanford and Berkeley, in spite of the success of new tools like CAR-T and CRISPR, some think that bioengineering is either hype or a passing fad. Thats OK; every new field struggles with the old guard.

Bioengineering still needs to come from a place of clearly and repeatedly explaining its worth with evidence. Lets just get used to this. That said, naysayers are predisposed to cling to old-school approaches. After all, thats the value they have to offer. Ultimately, they will need to adapt to an engineering approach or get engineered out of the process.

The culture of discovery and the culture of engineering value progress differently. Discovery, for example, prioritizes the Eureka! moment above all. One of the most challenging aspects of drug development is that you cant establish a key performance indicator for such moments. Pure discovery is a lottery ticket business that exists in the biopharma industry only because of its incredibly high value and the potential to save millions of lives.

Engineering, on the other hand, values a repeatable process, one that can be stacked or adjusted the way we build with Legos, aiming for compounding results a consistent fractional improvement year over year, leading to exponential improvement over time.

Health care needs both approaches. Todays great discovery will be engineerable tomorrow (OK, maybe 50 years from now). CRISPR, for example, began with discoveries in Haloferax mediterranei, a species of salt-tolerant bacteria. That was pure scientific discovery. But using CRISPR as a tool, as a therapeutic, or as a platform for future innovations is squarely in the world of bioengineering.

The choice of staying or leaving is now at the heart of the debate about what engineering can or cant handle, and what should remain pure, unfettered empirical discovery.

The truth is that engineering can handle empirical approaches. For example, is an A/B test discovery or is it engineering? Its actually both discovery done via an engineering process with iteration. Because biology is so incredibly sophisticated and complex, there will always be discovery risk the risk that some heretofore unknown aspect of biology will lead to failure. But part of engineering is, and should be, handling discovery and failure, and discovery can and should be engineered.

Tools like artificial intelligence and machine learning allow us to introduce to the world of discovery faster throughput, faster iteration, and greater reproducibility. We need to know how and where to apply engineering and discovery frameworks, and where the two worlds meet. Is your discovery risk one where you must wait for serendipity, or can you improve your odds by engineering some part of it?

The discovery and engineering cultures speak something that sounds like the same language, but really isnt. Words like discovery and platform mean very different things in science than they do in engineering. Even success doesnt directly translate: Does it work and we know how we got there, or did we get there in a repeatable process we can tweak?

Getting lucky with a serendipitous discovery is not success in an engineering discipline, nor is getting unlucky a failure in engineering, since you can learn something valuable from failure with which to tweak the process. Like any language issue, we need to recognize the different meanings in those core concepts and know when to use which depending on the world you are in.

Integrating these cultures requires each side to understand core tools, language, and mindsets in both worlds, and knowing where to leverage the differences. Where can discovery yield new empirical information for an engineered process? Where can an engineered process increase the odds of success over a more traditional discovery route?

In health care, as more and more products and tools become engineerable, the world of discovery will need to transition toward integrating engineering. This will be bumpy and uncomfortable and lets be real, there will be blood. But there will also be many bright spots. There will be people trained in science who, when introduced to engineering, feel they can tap into a new world of possibility. There will be engineers who maybe started their careers because as kids they dreamed of engineering trains who suddenly feel they have the potential to help cure cancer.

The audacious dream of engineering biology on a molecular scale is finally being realized not just in practice but commercially if we can surmount the culture clashes.

Vijay Pande, Ph.D., is a general partner at Andreessen Horowitz, a Silicon Valley venture capital firm, and an adjunct professor of bioengineering at Stanford University. He serves on the boards of Apeel Sciences, Asimov, BioAge, Ciitizen, Devoted Health, Freenome, Insitro, Omada, and PatientPing.

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Welcome to the bioengineering culture clash - STAT

Recommendation and review posted by Bethany Smith

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Table of Contents

Global Testosterone Therapy Market Research Report 2020-2025

Chapter 1 Testosterone Therapy Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

Chapter 5 Global Supply (Production), Consumption, Export, Import by Regions

Chapter 6 Global Production, Revenue (Value), Price Trend by Type

Chapter 7 Global Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10 Marketing Strategy Analysis, Distributors/Traders

Chapter 11 Market Effect Factors Analysis

Chapter 12 Global Testosterone Therapy Market Forecast

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Massive Growth of Testosterone Therapy Market by 2025 with Top Key Players like AbbVie Inc, Actavis Inc, Eli Lilly & Co, Auxilium Pharmaceuticals...

Recommendation and review posted by Bethany Smith

ValuEngine cut shares of Aytu Bioscience (NASDAQ:AYTU) from a buy rating to a hold rating in a report published on Tuesday, December 24th, ValuEngine reports.

Several other research firms have also recently commented on AYTU. Northland Securities started coverage on shares of Aytu Bioscience in a report on Friday, November 15th. They set a buy rating and a $5.00 price target on the stock. Zacks Investment Research lowered Aytu Bioscience from a hold rating to a sell rating in a report on Tuesday, December 17th. Finally, LADENBURG THALM/SH SH boosted their target price on Aytu Bioscience from $4.00 to $4.75 in a research note on Wednesday, September 18th.

NASDAQ AYTU traded down $0.05 during trading on Tuesday, reaching $0.85. 69,438 shares of the companys stock were exchanged, compared to its average volume of 99,631. The firm has a market capitalization of $18.59 million, a PE ratio of -0.24 and a beta of 4.61. Aytu Bioscience has a fifty-two week low of $0.65 and a fifty-two week high of $2.61. The companys fifty day simple moving average is $0.86 and its 200-day simple moving average is $1.25.

Aytu Bioscience (NASDAQ:AYTU) last posted its quarterly earnings results on Thursday, November 14th. The company reported ($0.32) EPS for the quarter, missing the consensus estimate of ($0.30) by ($0.02). Aytu Bioscience had a negative return on equity of 252.42% and a negative net margin of 390.43%. The company had revenue of $1.44 million for the quarter, compared to analyst estimates of $1.45 million. As a group, sell-side analysts expect that Aytu Bioscience will post -1.3 earnings per share for the current fiscal year.

In other Aytu Bioscience news, CEO Joshua R. Disbrow bought 55,000 shares of the stock in a transaction on Thursday, December 19th. The shares were purchased at an average cost of $0.83 per share, with a total value of $45,650.00. Also, major shareholder Armistice Capital Master Fund bought 78,788 shares of the businesss stock in a transaction on Friday, December 20th. The shares were bought at an average price of $0.90 per share, for a total transaction of $70,909.20. Insiders own 5.80% of the companys stock.

Several institutional investors and hedge funds have recently added to or reduced their stakes in the company. BlackRock Inc. acquired a new position in shares of Aytu Bioscience during the 2nd quarter valued at about $36,000. Virtu Financial LLC acquired a new stake in Aytu Bioscience in the 3rd quarter worth approximately $36,000. Bank of New York Mellon Corp acquired a new stake in Aytu Bioscience in the 2nd quarter worth approximately $75,000. Finally, Searle & CO. boosted its position in Aytu Bioscience by 24.1% during the third quarter. Searle & CO. now owns 249,848 shares of the companys stock worth $302,000 after purchasing an additional 48,601 shares during the period. Institutional investors own 27.76% of the companys stock.

Aytu Bioscience Company Profile

Aytu BioScience, Inc, a specialty healthcare company, focuses on developing and commercializing novel products in the field of hypogonadism (low testosterone), insomnia, and male infertility in the United States and internationally. The company markets Natesto, a nasal gel for the treatment of hypogonadism (low testosterone) in men; and ZolpiMist, an oral spray for the treatment of insomnia.

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Aytu Bioscience (NASDAQ:AYTU) Stock Rating Lowered by ValuEngine - Riverton Roll

Recommendation and review posted by Bethany Smith

SAN FRANCISCO, Jan. 8, 2020 /PRNewswire/ --Today BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) and Invitae Corporation (NYSE: NVTA) announced that Biogen (NASDAQ: BIIB), Encoded Therapeutics, Neurogene, Praxis Precision Medicines and PTC Therapeutics joined Behind the Seizure, an innovative, cross-company collaboration that aims to provide faster diagnosis for young children with epilepsy. The program will also be expanded to make no-charge testing available for healthcare providers to order for any child under the age of eight who has an unprovoked seizure.

"Behind the Seizure is one of the longest-running cross-company collaborations aimed at increasing access to genetic testing. It has been shown to decrease time to diagnosis for children experiencing unprovoked seizures by one to two years from reported averages, and as more companies have joined the program, more children have been helped," said Robert Nussbaum, chief medical officer of Invitae. "Earlier diagnosis enables clinicians to focus on providing disease-specific care sooner, which is particularly important in neurodegenerative diseases. We applaud these companies for their commitment to expanding this unique effort to help children."

Previously the program was available to children under age five. The newest companies to join Behind the Seizure include:

Behind the Seizure is supported by eight sponsors in all, including Stoke Therapeutics and Xenon Pharmaceuticals who joined in 2019.

Since the program began, thousands of children have received genetic testing through Behind the Seizure and research has shown that participants in the program were diagnosed one to two years sooner than historic averages.1 Companies that participate in the program sponsor the cost of testing using the Invitae Epilepsy Panel, which includes more than 180 genes associated with both syndromic and non-syndromic causes of epilepsy, including neurodegenerative conditions. With the expansion of the program, healthcare providers now can order the test for patients under the age of eight with unprovoked seizures. Test results are available quickly (14 days on average).

More than half of epilepsies are based in genetics. When a child presents with seizures, genetic testing can help identify more than 100 underlying, often rare conditions. Early genetic testing may be the most cost-effective, direct and accurate diagnostic tool for children, shortening years-long diagnostic odysseys. Delays in diagnosis can be devastating for children, as some genetic epilepsies are neurodegenerative and early symptoms may be subtle and easy to misdiagnose.

Participating companies provide financial support for this program, which includes testing and services performed by Invitae. Healthcare professionals must confirm that patients meet certain criteria to use the program. Third parties and commercial organizations may receive de-identified patient data and contact information for healthcare providers who use this program, but at no time do they receive patient identifiable information. Genetic testing and counseling are available in the US and Canada. Healthcare professionals and patients who participate in this program have no obligation to recommend, purchase, order, prescribe, promote, administer, use or support any other products or services from Invitae or from third parties or commercial organizations.

About Behind the SeizureBehind the Seizure is an innovative, cross-company collaboration designed to increase access to genetic testing for children who experience unprovoked seizures in childhood in the United States and Canada. More than half of epilepsies have some genetic basis, and are often associated with rare, neurodegenerative conditions with non-specific symptoms. Early genetic testing may be the most direct, cost-effective, and accurate diagnostic tool. Participants in the Behind the Seizure program are diagnosed one to two years sooner than reported averages. The program was established by BioMarin and Invitae and now includes: Biogen, Encoded Therapeutics, Neurogene Inc., Praxis Precision Medicines, PTC Therapeutics, Stoke Therapeutics and Xenon Pharmaceuticals. To learn more about the Behind the Seizure program please visit https://www.invitae.com/en/behindtheseizure/.

About BioMarinBioMarin is a global biotechnology company that develops and commercializes innovative therapies for patients with serious and life-threatening rare and ultra-rare genetic diseases. The company's portfolio consists of seven commercialized products and multiple clinical and pre-clinical product candidates. For additional information, please visit http://www.biomarin.com.

About InvitaeInvitae Corporation (NYSE: NVTA) is a leading genetics company whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. For more information, visit the company's website at invitae.com.

About Encoded TherapeuticsEncoded Therapeutics, Inc. is a biotechnology company developing precision gene therapies for a broad range of severe genetic disorders. Our mission is to realize the potential of genomics-driven precision medicine by overcoming key limitations of viral gene therapy. We focus on delivering life-changing advances that move away from disease management and towards lasting disease modification. For more information, please visit http://www.Encoded.com.

About Neurogene Inc.Neurogene was founded to bring life-changing medicines to patients and families affected by rare neurological disorders. We partner with leading academic researchers, patient advocacy organizations and caregivers to bring to patients therapies that address the underlying genetic cause of a broad spectrum of neurological diseases where no effective treatment options exist today. Our lead programs use AAV-based gene therapy technology to deliver a normal gene to patients with a dysfunctional gene. Neurogene is also investing in novel technology to develop treatments for diseases not well served by gene therapy. For more information, visit http://www.neurogene.com.

About Praxis Precision MedicinesPraxis Precision Medicines is a clinical-stage genetic neuroscience company developing high-impact therapies for patients and families affected by complex and debilitating brain disorders, including rare pediatric epilepsies and neuropsychiatric disorders. These two disease areas share overlapping disease biology and genetic targets, as well as a profound need for new therapeutic options that target the underlying cause of the disease. Praxis is advancing a pipeline of breakthrough medicines with the potential to more precisely treat complex brain disorders. For more information, please visit http://www.praxismedicines.com.

About PTC TherapeuticsPTC Therapeutics is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically-differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need.

About Stoke TherapeuticsStoke Therapeutics, Inc. (Nasdaq: STOK), is a biotechnology company pioneering a new way to treat the underlying causes of severe genetic diseases by precisely upregulating protein expression to restore target proteins to near normal levels. Stoke aims to develop the first precision medicine platform to target the underlying cause of a broad spectrum of genetic diseases in which the patient has one healthy copy of a gene and one mutated copy that fails to produce a protein essential to health. These diseases, in which loss of approximately 50% of normal protein expression causes disease, are called autosomal dominant haploinsufficiencies. The company's lead investigational new medicine is STK-001, a proprietary antisense oligonucleotide (ASO) that has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome, a severe and progressive genetic epilepsy. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts. For more information, visithttps://www.stoketherapeutics.com/or follow the company on Twitter at @StokeTx.

About Xenon PharmaceuticalsXenon Pharmaceuticals is a clinical stage biopharmaceutical company committed to developing innovative therapeutics to improve the lives of patients with neurological disorders, including rare central nervous system (CNS) conditions. We are advancing a novel product pipeline of neurology therapies to address areas of high unmet medical need, with a focus on epilepsy. For more information, please visit http://www.xenon-pharma.com.

Safe Harbor StatementsThis press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to the benefits of genetic testing and the Behind the Seizure program, including shortening the time to diagnosis and improved treatment outcomes for patients. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially, and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to: the company's ability to use rapidly changing genetic data to interpret test results accurately and consistently; the ability of genetic testing to result in faster or more accurate diagnosis; laws and regulations applicable to the company's business; and the other risks set forth in Invitae's filings with the Securities and Exchange Commission, including the risks set forth in its Quarterly Report on Form 10-Q for the quarter ended September 30, 2019. These forward-looking statements speak only as of the date hereof, and Invitae Corporation disclaims any obligation to update these forward-looking statements.

Contact:Laura D'Angelopr@invitae.com(628) 213-3283

1 Miller, Nicole, et al, "Behind the Seizure: A No-Cost 125-gene Epilepsy Panel for Pediatric Seizure Onset Between 24 Years". Presented at the American Society of Human Genetics Meeting: October 1620, 2018, San Diego, CA.

View original content to download multimedia:http://www.prnewswire.com/news-releases/behind-the-seizure-program-further-expands-access-to-genetic-testing-for-children-to-speed-the-diagnosis-of-genetic-epilepsy-300983302.html

SOURCE Invitae Corporation

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Behind the Seizure Program Further Expands Access to Genetic Testing for Children to Speed the Diagnosis of Genetic Epilepsy - P&T Community

Recommendation and review posted by Bethany Smith

Pharmacogenetics presents clinicians an attractive option to optimize drug therapy, minimize harmful effects, and contain costs; but, payers may not be taking full advantage of cost-saving potential by choosing only to cover the costs associated with interrogating a single gene.

Reports vary, but the cost of testing a single gene appears to range from $100 to $500, depending on the source. The cost of running an entire panel is a similar price.

Despite the virtually negative cost differential, payers are reluctant to cover the cost of running a full panel of tests. This common practice raises the question of why many payer organizations do not pay for running an entire test panel. Experts differ in their speculations as to why this is the case.

I dont think theyre familiar with the total body of knowledge, says Ruben Bonilla-Guerrero, MD, FACMG, FAACC, MB(ASCP), CGMBS, medical director of medical affairs at Admera Health, a leader in personalized medicine and non-invasive cancer testing in South Plainfield, New Jersey. Insurance companies consider running a pharmacogenetic test panel as experimental even though the labels on more than 200 drugs mention pharmacogenetics testing.

Related:Genetic Testing in Treatment Decision Making Goes Mainstream

Pharmacogenetic testing offers important advantages by facilitating the prescribers ability to select, initiate, and adjust a pharmaceutical drug product with a much higher level of precision than previously available with conventional dose titration. Pre-emptive testing also helps prevent adverse drug events that occur as a result of overdosing medications.

However, like many areas of healthcare, weighing the cost against the purported benefits is also critical.

Payers want to reimburse for tests that are done for a specific indication and that have evidence-based, actionable treatments based on the results, says Erin Lopata, PharmD, MPH, senior director of the Access Experience Team at Precision for Value, part of Precision Value & Health, which performs value and access consulting services for entities that may be involved in value-based contracting.

Read more:
The Benefits of Pharmacogenetic Testing - Managed Healthcare Executive

Recommendation and review posted by Bethany Smith

Biofidelity assay has potential to make high precision, cost-effective and non-invasive diagnosis more widely available, improving treatment and patient outcomes

Cambridge, UK, 9th January 2020 Biofidelity Ltd, a company developing high performing novel molecular assays for the detection of targeted, low-frequency genetic mutations, today announced the successful completion of a study to detect key lung cancer mutations in collaboration with Agilent Technologies, a global leader in life sciences, diagnostics, and applied chemical markets.

The collaboration, using an assay developed by Biofidelity, demonstrated an improvement in sensitivity of 50 times that achieved with current FDA-approved PCR-based diagnostics, matching that of specialized NGS assays, which require error-correction technology, while providing a dramatic simplification of workflows from more than 100 steps, to just 4 (four). Assays were performed using standard laboratory instrumentation, demonstrating the potential for straightforward adoption of Biofidelitys panels in decentralised testing laboratories around the world.

As well as extremely high sensitivity, 100% specificity was achieved in the detection of multiplexed panels of mutations from both tissue and plasma, with no false positives observed across more than 750 assays. Analysis of results is also dramatically simpler than sequencing-based assays, providing physicians a clear, simple, actionable result, with a turnaround time of less than 3 hours, making the Biofidelity assay suitable for recurrent patient monitoring.

Genetic testing for lung cancer mutations is usually carried out through invasive tissue biopsy, an expensive procedure carrying significant risk for patients with advanced disease. Up to 10% of such tests fail due to the lack of sensitivity of current testing solutions and poor sample quality.

Liquid biopsy, or testing directly from the patients blood, offers a non-invasive alternative with significant potential benefits to patients. However, its use has been limited by the lack of cost-effective, robust and rapid tests which are sufficiently sensitive to enable detection of the very small fractions of tumor DNA present in such samples.

Of the nearly 2 million new cases of non-small-cell lung cancer (NSCLC) diagnosed each year worldwide, fewer than 5% of patients receive high-sensitivity, non-invasive genetic testing. The assay developed by Biofidelity could provide a simple solution, enabling access to high-precision genetic testing for more than 1.7m new NSCLC patients every year with a test that outperforms DNA sequencing in a fraction of the time.

Work was supported by InnovateUK grant number 105202 as part of the Investment Accelerator: Innovation in Precision Medicine program.

Dr Barnaby Balmforth, Chief Executive Officer of Biofidelity, commented: Our goal is to improve patient outcomes in oncology by enabling much greater access to the highest precision diagnostic tests. This collaboration with Agilent in lung cancer has again demonstrated that Biofidelitys molecular assays dramatically increase the effectiveness and speed of diagnosis, supporting early detection of disease, better targeting of therapies and improved patient monitoring. By combining diagnostic outperformance and rapid results in a simple, cost-efficient format using existing instrumentation, we believe we have the potential to bring high precision testing to many more NSCLC patients, substantially reducing the need for invasive biopsies.

Tad Weems, Managing Director, Agilent Early Stage Partnerships, commented: As both a scientific collaborator and an investor in the company, Agilent has been impressed by the data from Biofidelitys assays, which detected a selection of NSCLC DNA mutations at extremely low frequencies in both tissue and plasma samples without the need for DNA sequencing. Biofidelitys assays are specific and sensitive, with the potential to provide improved and rapid routine cancer diagnostics.

Notes To Editors

About Biofidelity

Biofidelity has developed a molecular assay with a simple workflow and fast time-to-result which can transform the detection of genetic abnormalities within a sample by reliably detecting large panels of DNA mutations at extremely low frequencies.

This assay has a simple workflow and is suitable for routine use in diagnostics labs around the world, without the need for investment in new instrumentation or infrastructure.

Biofidelity is developing genetic panels for use in precision medicine and patient monitoring across a range of diseases including NSCLC and colorectal cancer

Located in Cambridge, UK, Biofidelity is a private company founded in 2019.

For more information, visit http://www.biofidelity.com, or follow us on LinkedIn: Biofidelity.

Issued for and on behalf of Biofidelity by Instinctif Partners.For more information please contact:

BiofidelityDr Barnaby Balmforth, CEOT: +44 1223 358652E: info@biofidelity.com

Instinctif PartnersTim Watson / Genevieve WilsonT: +44 20 7457 2020E: Biofidelity@instinctif.com

Read more from the original source:
Biofidelity and Agilent complete successful molecular assay study for rapid and accurate detection of key lung cancer mutations - BioSpace

Recommendation and review posted by Bethany Smith

Im fascinated by genetics, but I also find it extremely complicated and confusing. In fact, I remember genetics being one of the most intimidating topics we learned in biology back in high school. If you had told me back then that Id be writing about a genetics company all these years later for The Jerusalem Post, I might have laughed.When it comes to genetics, variables are limitless, and its mind-blowing to see how each of us, in our own unique way, essentially won the genetic lottery through the course of time and evolution.This explains why recreational genetic testing providers, such as 23andMe, Ancestry.com, and MyHeritage gained significant popularity over the years.However, it is worth noting that these services can only read a portion of our DNA data. There is so much more depth to the full picture of our DNA and gaining visibility into that data can improve health and prevent premature death, as well as identify and eliminate the risk of passing genetic disorders.The first human genome was sequenced in 2013 as part of an international project with the goal of identifying and mapping all human genes. The project took 13 years to complete and the estimated cost was a whopping $3 billion.To this day, it remains the worlds largest collaborative biological project and it has opened new frontiers in science and medicine, but also brought challenges that have yet to be fully addressed.It presently costs less than $1,000 and takes a single day to sequence the human genome. Whole genome sequencing (WGS) provides a readout of the three billion letters of an individuals entire DNA sequence. This data provides the foundation for identifying all types of disease-causing variants, or changes in an individuals DNA that cause disease.However, the biggest challenge is how to accurately (and economically) make sense of the vast amounts of data that WGS generates. I mean, there is big data and then there is this, more like astronomical data!Variantyx is a Tel Aviv-based clinically accredited genetic testing lab. Its headquarters are in Boston and it uses technology based on WGS to perform one-stop comprehensive testing for patients. The company was founded in 2014, and has raised $18 million in capital from Pitango. The analyses performed by Variantyx are carried out using an in-house developed analytical platform built and optimized for WGS data. Their proprietary algorithms identify a wide range of difficult-to-detect sequence variants, and is then presented to Variantyxs clinical team for interpretation and reporting. The potential of this technology is quite literally limitless.Variantyxs Genomic Unity is the industrys first comprehensive, all-in-one test for diagnosis of rare inherited disorders. It eliminates the need to order multiple tests, resulting in more diagnoses in a shorter period of time, and with lower overall costs. This is a big deal because rare disorders are not actually rare. With more than 7,000 rare diseases, its estimated that over 30 million people in the US and over 350 million people globally are affected. Many of these diseases are genetic in origin, and many of the individuals affected are children.THROUGHOUT THE typical diagnostic journey, a rare-disease patient will be seen by eight different physicians in less than a decade, and will spend an average of $21,000 to reach a diagnosis. They are the lucky ones. Many patients remain undiagnosed, despite multiple rounds of genetic testing. Variantyxs WGS-based Genomic Unity test is changing this.Since its introduction to the market in late 2017, the test has solved hundreds of previously undiagnosed cases for patients at many childrens hospitals worldwide. The benefits of comprehensive genetic testing extend to healthy individuals. Variantyxs Genomic Inform test has been developed to provide important information about disease predisposition risk, and carrier status. The test has applications in disease predisposition screening, newlywed carrier screening, prenatal screening and pharmacogenomics a field of precision medicine that identifies variants which influence how an individual responds to a particular prescribed drug.In terms of market demand, the overall genomic diagnostic market is valued to be greater than $20 billion, most of which is health-insurance driven. It has the potential to grow substantially as the cost of sequencing continues to decrease. In 2019, Francis deSouza, the CEO of Illumina, committed to making the $100 genome a reality. At that price, in the not so distant future, it will be possible to sequence every newborn and make their genomic data available for consultation whenever a clinical decision is made during their lifetime.Variantyx was formed as a spin-out of Tel Aviv University in 2014, and is now 45 team members strong. The idea of Variantyx actually started 10 years ago at the functional genomics lab at Tel Aviv University, headed by Prof. Noam Shomron. He and his PhD students were frustrated by the major bottleneck affecting analysis of patients exome data, as so much of the work had to be done manually. They set about developing a pipeline to more effectively process the information. To tackle the challenge of applying his teams work at the scale needed to help a larger number of patients, Noam teamed up with Tomer Jackman and Haim Neerman. Jackman had played a pivotal role at EMC, helping to develop their big data platform, and naturally wasnt daunted by the vast amounts of data that needed to be processed.Neerman, a serial technology entrepreneur in the area of IT systems and business process automation brought valuable expertise in eliminating time-consuming and error-prone manual steps. They collectively had the foresight to anticipate the shift from exome data to genome data and began building the first version of the companys analytical platform. The same platform is used today to perform its WGS-based Genomic Unity and Genomic Inform testing. The company has been growing rapidly to support the increased demand for its testing.Today, Variantyx is providing answers to rare disease patients and healthy individuals alike, and I personally cant want to see what unfolds as they prepare for a more broad population-based deployment as genome sequencing becomes more affordable, accessible, and penetrates the mainstream.

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Israel's all-in-one test for diagnosis of rare inherited disorders - The Jerusalem Post

Recommendation and review posted by Bethany Smith

A doctor diagnosing a 50-year-old patient based on a blood test taken during the patients infancy would be unthinkable.

Anecdotally speaking, however, thats what Michigan State University scientists have done with corn. Using plant RNA data from 2-week-old corn seedlings, Shinhan Shiu, professor of plant biology and computational mathematics, science and engineering, has shown that farmers and scientists can improve adult crop trait predictions with accuracy that rivals current approaches using DNA, i.e. genetic data.

Traditional breeding methods take months to years, which can be saved if we can predict the desirable traits just from DNA and RNA without growing them, without having to measure the actual traits directly, said Shiu, senior author of the paper appearing in the current issue of The Plant Cell. To continue the human medicine anecdote, its like sequencing an infants RNA and analyzing what sort of traits the infant may develop later in life.

Shiu has long been fascinated with using computational approaches to resolve evolution and genome biology questions. A well-recognized grand challenge in biology is how to connect information in the DNA, or genotype, with traits, or phenotype. Solving this mystery is fundamental to understanding how genetic information is translated into outward traits in any species, Shiu said.

Since RNA is a product of DNA, one step closer to the traits DNA ultimately influences, the RNA blueprints can potentially offer better predictions. Using machine learning approaches, Shiu and his colleagues have taken a step closer to connecting DNA, RNA and the underlying traits.

This is helpful for new breeding programs and may have implications in new ways to do genetic testing, Shiu said. We found that RNA measurements provide additional information that we cannot get from DNA alone.

In terms of reproduction, for example, the team was able to make accurate flowering and yield predictions even before the plants had developed their seed or flower organisms.

Traditional methods using genetic marker-based models identified only one of 14 known genes linked to flowering time as important. However, the gene expression-based model created by Shiu and his colleagues identified five.

Even with this increased accuracy, though, Shius team isnt saying the new method should replace the old.

Our findings are complementary to genetic marker-based prediction and identifies gene expression-trait associations that are not explained by genetic markers, Shiu said. Not only does this help in selection of breeding lines with desirable traits, but also enhances our understanding of the mechanisms involved in these processes.

Future research will work to improve the models accuracy, efficiency and cost.

Additional MSU scientists contributing to the study include Christina Azodi, Jeremy Pardo, Robert VanBuren and Gustavo de los Campos. For more, seethe original paper at http://www.plantcell.org/content/early/2019/10/22/tpc.19.00332.

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Scientists improve yield predictions based on seedling data - Ohio's Country Journal and Ohio Ag Net

Recommendation and review posted by Bethany Smith

Coverage of our peer-reviewed research and news reporting in the healthcare and mainstream press.

Philly Voices piece, Men with testicular cancer may only need one round of chemo, study says, cited an article published onAJMC titled, When Less Is More: Halving Chemo Keeps Testicular Cancer at Bay, Study Finds. The article outlined findings from a study stating that young men who undergo chemotherapy after surgery for testicular cancer may live with side effects for decades.

Dark Daily featured an April 2019 article of AJMC titled, As DTC Genetic Testing Grows Among Consumers, Insurers Are Beginning to Get on Board. The article stated that by 2021, 100 million people will have used a direct-to-consumer genetic test, indicating its growing popularity among healthcare providers and consumers.

A September 2019 article onAJMC titled, Physician Shortage Likely to Impact OB/GYN Workforce in Coming Years, was spotlighted by MedCity News, whichreported that a womens health startup providing care by blending technology and physical clinics has pulled in $45 million to support growth.

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AJMC in the Press, January 10, 2020 - AJMC.com Managed Markets Network

Recommendation and review posted by Bethany Smith

As we enter the New Year, health, wellness, and resolutions to eat better and get more exercise are priorities for most of us who have not yet made them permanent fixtures in our way of life. Whether you launch into 2020 with a keto or Mediterranean diet and/or join Orangetheory, Equinox or that cult (I mean gym) CrossFitor just subscribe to the burn more calories than you take in tried and true methodwe all start off with the best of intentions. In the last couple of years, the newest health threat has been sitting too muchin our offices and homesand we have seen the emergence of stand-up desks and "how many steps have you taken today" has become a normal part of everyday conversation. But maybe the most well-accepted cause and least understood "silent killer" that we collectively do a terrible job of managing is stress.

The human body functions best when it is in a state of homeostasisin a condition of balance, harmony, and stability. Our bodies have evolved to have a number of compensatory mechanisms to maintain a balance for pH, O2/CO2, electrolytes, glucose/insulin, immune response activation/de-activation, hormone levels, and many other physiological systems. When the homeostatic state for these physiological balances is thrown off due to genetic or environmental changes over time, they may eventually experience a number of problems in their mental and physical functions. This may result in a variety of symptoms from headaches, nausea, and exhaustion to more pronounced situations, such as high blood pressure, diabetes, auto-immune diseases, and cancer.

The medical community continues to elucidate the cause and effect relationship between bacteria, viruses, toxins and our genetics and how those lead to illness. Researchers and companies continue to develop new and better drugs, vaccines, medical devices, and even gene therapy and gene-editing technology to "fix us" when we get sick. But what about our "mind-body" connection? And no, that is not just a term used by alternative medicine charlatans. The connections between our physical and mental well being are well established if still poorly understood mechanistically. Psychosomatic Disorders are a set of physical diseases that are either caused or made worse by mental factors such as anxiety or stress. According to the Cleveland Clinic, depression can be caused by chronic illness and further exacerbate the illness thereby "causing a vicious cycle to develop."

The rates for depression that occurs with other medical illnesses is quite high (national average for depression without co-morbidities is ~7-10%):

A recent article in Psychology Today explains that stress is a constant in our lives; it is always in flux and that stress and our bodies' response to it is not in and of itself a bad thing. Think "fight or flight" our evolutionary response to making sure we didn't get eaten by that annoying dinosaur. That dino-stressor kicks off a cascade of hormones that elevate our blood pressure, heart rate and increase our energy burn presumably so we can outrun that T-Rex. That's all well and good, but I don't think I will need to outrun a dinosaur or even a lion anytime soon. So what if our bodies are exposed to other non-carnivore caused periodic high-level stressors or chronic state of stress that trigger these cascades? What does that do to our bodies?

In a recent review of the physiological effects of stress, scientists and clinicians have demonstrated the negative effects on everything, including our central nervous system, cardiovascular, immune, gastrointestinal and endocrine systems. We are all familiar with stress-induced headaches, difficulty sleeping, and upset tummies, but unmanaged stress can also negatively impact a person's cardiovascular system by increasing blood pressure and heart rate which over time can lead to "stress" cardiomyopathy or increased risk of heart attack and stroke. Studies have shown impairments in memory recall, as well as cognitive processing, are also linked to chronic stress.

Let's face it, we all encounter some form of stress usually on a daily basis. We have demanding workdays, tiresome and/or tedious long commutes to our jobs, worrying about bills and the demands and responsibilities that come with raising our kids. Speaking from experience, couples will also experience stress from their relationships at times and business owners can also be stressed out about the success of their personal enterprises. While we can all expect to be stressed out over something in our lives at some point, allowing ourselves to continually remain in a state of stress could lead to serious health problems.

Healthline reports people who are stressed usually have a hard time controlling their emotions. Sound familiar to anyone? These emotional outbursts can lead to or exacerbate already existing problems with our spouse (or significant other), friends, family or co-workers. This can lead to even more stress and physical illness.

So, as you make those New Year's resolutions, let's not lose sight of how important removing or managing our stress is to our health, happiness, and well being. Resolve to try to be more self-aware of your own stress and how it is affecting you and others around you. Don't be afraid to talk to your doctor, spouse, family or friends about things that are stressing you. There is no shame in speaking up and you can take comfort in the knowledge that you are definitely not alone. Take steps to remove stressors where you can. When that is not possible, identify and try a variety of ways to cope with, manage and reduce that stress. Look, I am not an expert on what works or doesn'tdifferent things work for different people. Maybe it's a long walk, listening to music, meditating, hitting the gym, playing a video game with your kids or just giving and receiving a BIG hug from your spouse.

Life is life and we will never be stress-freebut let's resolve to reduce our stress levels in 2020 and to help those around us as well.

Originally published at erikhalvorsen.org on January 9, 2020.

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The Deadly Effects of Stress on Our Bodies - Patch.com

Recommendation and review posted by Bethany Smith

Note: This article was co-published with the Daily Bruinas a product of the Bridget O Brien travel grant.

Sarat Kumar Borah had been taking enzalutamide for six weeks when he told his son he wanted to give up. The medicine was helping a little with his prostate cancer symptomshis headaches were milder, some days he had more energybut he became livid whenever he thought about the price of the drug.Im drying up your funds, he told his son. I am old. What is the point of me living any longer if its going to finish everyones reserves.

Without insurance to cover the cost of the medication, Borah had relied on his son, Rupam, to help pay for the drugs. Even with the leftover box of the medicine Sarats oncologist gave him, Rupam estimates he spent around 25 thousand rupees on treatments, nearly a fourth of the average annual salary in India.

Rupam insists his father is not the type to give up.

Im going to fight this damn thing out, his father had joked just weeks earlier. Who do you think youre dealing with?

But things had changed.

Although he owns a successful design business, Rupam stopped eating out, going on vacations, and dipped far into his youngest daughters college fund, leaving him up most nights trying to piece together a plan to send her to university.

Im just trying to tell her, Finish your class 12 and hold on, lets see how we do, he says. I cant break her heart.

Even if the medicine was just a little less expensive it would help, he said. Instead, in the next couple months, the price of enzalutamide could dramatically increase.

In 2016, the patent for enzalutamide, a wonder drug for late-stage prostate cancer discovered by UCLA, was rejected by the Indian patent office, a decision many believed would make the drug less expensive.

Generic versions of the drug flooded the market, offering enzalutamide for a fraction of the cost of the name-brand drug, Xtandi. Although it still wasnt as cheap as chemotherapy, Knowledge Ecology International, a consumer advocacy group, estimated that as more generic companies joined the market, the cost of a pill could be driven down to 50 cents.

Then, just a year later, UCLA appealed the decision to the Delhi High Court and, last May, won. The court sent the patent back to the Indian patent office to evaluate anew. If the office approves the patent, the decision would outlaw the generic brands of enzalutamide, leaving only Xtandi, a medication with a daily dose that is 70 percent more expensive, costing about 2 lakhs for 112 capsules, or about $223,576 in U.S. dollars.

Liz Ketcham

In 2007, the UC system and 11 other universities signed on to a set of ethical licensing guidelines that emphasized the consideration of the needs of people in developing countries, according to the document. But to critics, the pledge was little more than a PR stunt. As it was signing its symbolic goodwill gesture, UCLA was also wrapping up the licensing agreement for Xtandi, with no provisions to ensure patients in developing countries would be able to afford the life-saving medication.

Phil Hampton, a UCLA spokesperson, says the university has since changed its licensing policies to include language that encourages licensees to consider the interests of underserved populations.

Still, Rupam Borah and countless others will not be able to afford the medication if the patent is approved. Spending a quarter of his salary on medicine is unaffordable spending 40 times his salary is impossible.

We will have to live by the reality that he will have to pass away sooner, he says. Thats it. What else can we do?

In a small lab on the southern end of UCLAs campus, Michael Jung spent most of his career as an organic chemist, developing new ways to synthesize chemicals. For 45 years he was content tinkering away at basic research questions and publishing results in journals like Tetrahedron Letters. Then on his 55th birthday, his wife pulled him aside.

What do you want to do for the rest of your life, she asked. More of the same?

Jung said he always dreamed of finding a cure for a human disease but never had the guts to try it. Some researchers worked years on developing a medication with nothing to show for it.

The day after his birthday, a colleague asked if he wanted to join a project dedicated to creating a prostate cancer drug. He took it as a sign, even though he knew almost nothing about prostate cancer. If nothing came of it, the worst people would think was that hed decided to retire early, he thought.

A couple years later, in 2009, he published a paper in Science, introducing enzalutamide, a molecule that could drastically alter the way prostate cancer was treated. While chemotherapy moves through the body quickly and indiscriminately, wiping out cancer cells and any other cells that divide quickly (like bone marrow), enzalutamide elegantly clicks into receptors on the surface of prostate cancer cells, blocking the hormones that cause them to grow.

Stephen Freedland, an oncologist at Cedars-Sinai, remembers the excitement around enzalutamide and other similar drugs.

There werent a lot of options for men. It was kind of like, Sorry, life sucks, Freedland says. Then all of a sudden we had a drug that could salvage a lot of these guys and just buy them time, a good quality of life.

Chemotherapy often leaves patients bed ridden with nausea. In contrast, patients on enzalutamide can usually maintain their normal lifestyles, only complaining occasionally about feeling a bit foggy. One of Freedlands patients said he couldnt lift as much at the gym, which is to say he, a late-stage cancer patient, was still working out.

In early clinical trials, late-stage prostate cancer patients who had already exhausted hormone therapies and chemotherapy, enzalutamide extended their lives by five months. In oncologist circles, the result was promisingusually treatments that work in late-stage cancer patients work even better for early stage patients.

Two years later, scientists stopped a clinical trial short after enzalutamide significantly reduced the risk of death in patients before they started chemotherapy, and in August, the FDA approved enzalutamide for patients before they even started hormone therapy.

Freedland thinks eventually enzalutamide could turn prostate cancer into a chronic illness something you can live with for years like diabetes or asthma.

We are in a renaissance period in the medical therapy of prostate cancer, an author of one of the clinical trials for enzalutamide said. Even at this early stage, enzalutamide is a game changer.

By weight, Xtandi is 65 times more expensive than gold. In the United States, that is 2.5 times the annual per capita income. In India, the name-brand drug costs more than 38 times the per capita income.

Rajeev Kumar, a urologist at a public hospital in Delhi, says if the patent were approved, almost none of his patients could afford the medication. Already, his patients struggle to afford the generic brands. Many of them run out of money a couple weeks into the treatment plan and stop taking the pills. Others, aware theyll inevitably exhaust their resources, refuse the prescription from the beginning.

With the patent, even his wealthiest patients would be unable to afford the medicine.

Liz Ketcham

In the United States, 80 percent of patients with insurance have a copay of less than $25 a month for Xtandi. In India, Kumar estimates less than 20 percent of his patients have insurance.If they do, their insurance policies usually cover a fixed amount of medical expenses for the entire family, which Xtandi would quickly deplete.

Rupam Borah has seen the worst of it in the hospital as he waits for his fathers appointmentschildren, mothers, and wives sobbing next to him, coming to terms with the fact that they cant afford medication.

Im still pushing for medicines and going desperate and having sleepless nights sometimes and all that but still look at people who are below us who cannot even imagine they will die, he says, shaking his head. Ive seen those kinds of people in hospitals. Ive seen them. I mean, they just cry. They are completely helpless.

Most of the men hes talked to in the hospital can scrape together enough for about a fourth of the cost of one box of enzalutamide. Sometimes he gives money to other patients at the hospital. Stretch it for as long as you can, he tells them.

If the pills became more expensive, Kumar isnt sure he would even prescribe enzalutamide to his patients anymore. A couple of months of life is only worth so much financial stress.

I know if I offered it to him he would take it and thatll end up destroying his family, he says. The next generation (will be) in debt for the next 10 years.

Even as an established doctor and professor at the most widely respected hospital in Delhi, one box of Xtandi would cost his entire monthly salary.

Would I give it all up to extend a couple of months? he says. Probably not.

Enzalutamide ultimately made the University of California more than a billion dollars. Even for the UC system, which owns the most patents of any American university, the drug generated more money than any other patent sale.

We are strategically supporting one of our essential missionsfunding and generating research with practical applications that serve the public good , Gene Block, the chancellor of UCLA, said in a university press release following the deal.

Except to many, it didnt seem as if the university was trying to serve the public good.

First, there was the billion dollars.

To R. Joseph Trojan, a pharmaceutical patent lawyer based in Los Angeles, it was simple economics: the more the UC charged for the patent, the higher pharmaceutical companies would have to price the medicine to recover what they had spent.

Where does it think the money was going to come from? he asks. It was coming out of the pockets of people who need the drug. Why are you driving up the cost of these drugs by demanding a billion dollar licensing fee upfront? That makes no sense at all.

Furthermore, although researchers used public funding from the National Institutes of Health and the U.S. Armys prostate cancer research program to discover Xtandi, UCLA was actively pursuing a patent that would make it inaccessible to the public. Meanwhile, money from the patent sale sits in a portfolio, generating $60 million dollars for UCLA every year.

Then there was the language in the licensing agreement. With no clause to protect developing countries, the agreement seemed to directly contradict the universitys own licensing guidelines, which say it should consider such public benefit and broad societal needs when developing licensing strategies.

John Mazziotta, the CEO of UCLA Health, says these ethical licensing guidelines are not always prescriptive, explaining there are a variety of complex and potentially contradictory issues that need to be taken into account when drafting such an agreement. Producing and testing medicine is expensive, Mazziotta argues. A pharmaceutical company might not agree to produce the medicine at all if there were a clause limiting the sales in developing countries.

Healthcare advocates have long been skeptical of that argument given that other companies, like the French NGO responsible for a successful gene therapy, included a reasonable pricing clause in their agreement and were still able to license the patent for millions.

Other institutions, like Harvard University, were able to include global access provisions in more than half of their pharmaceutical licensing agreements, clauses that allow generic companies in developing countries to produce their patented medications. They also allow Harvard to refuse to prosecute a patent in a developing country.

The UC created a committee in 2009 to oversee the inclusion of clauses that consider the needs of underserved people. These clauses have been included in 30 licensing agreements since 2018. Still, some argue this change was too little too late.

Without a global access provisions in the Xtandi agreement, if Medivation, a biopharmaceutical company that helped manufacture Xtandi, wanted UC to prosecute a patent that could take away medicine from thousands, the university has no legal avenue to refuse.

UC ultimately appealed the India patent decision on a technicality, claiming the patent office hadnt taken into account a piece of evidence. To argue its case, they hired Palaniappan Chidambaram, the former Union Minister of Finance in India. In October, Chidambaram was charged with corruption, forgery, and cheating by Indias Central Bureau of Investigations in October, and was granted bail in December. Chidambaram has denied wrongdoing.

Neda Ashtari, a second-year medical student at UCLA, stood on the stairs of Powell Library in October, dressed in all black.

In front of her were tens of white paper bags, each with a small plastic candle inside and a name scribbled on the front in colored marker. Students sprinkled plastic rose petals in between the bags, a sort of vigil for the lives lost as a result of UCLAs inaction to drop the patent on Xtandi, they wrote in a pamphlet.

Ashtari protested the patent appeal in India for years. In Regents meetings she stood in front of University of California leadership and demanded change.

Liz Ketcham

Can you imagine the guilt you would feel if all of your familys income went to your [medical] bills? Would you even want to live at that point? she asked. For one moment can you just step outside yourselves and imagine having to choose between the roof over your head and the (medicine) you needed to stay alive?

But on that day in October, standing in front of the bags of names, she seemed quieter more defeated than angry.

The pleading, public protests, and letters to the UC presidents officeone signed by 56 civil society organizations and doctors, then another with 3,500 signatures signed by students and advocacy groups had been mostly ignored.

To her, UCLAs choice to defend the Xtandi patent is personal. Her mother was diagnosed with breast cancer when she was four years old. For 12 years, as her mothers primary caregiver, she fought with insurance companies to get medication. She lost her house, her father left under the mounting stress over the cost of treatmentsand eventually her mother.

She says the worst part wasnt saying goodbye to her mom, it was knowing the same financial nightmare would happen over and over again.

They say time heals all wounds but it hasnt, she says, her voice breaking up. I still live with the consequences of these policies every day.

In a small coffee shop on the outskirts of New Delhi, Rupam Borah flips through pictures of his dogs on his phone.

Theres Bella, who he found badly beaten on the side of the road; Phi Phi, who he rescued on the way back from his vacation on Phi Phi Island, and Coco, whose paw was run over by a car.

He has a soft spot for stray dogs. He fosters stray puppies, pays to neuter dogs, and even puts out bowls of Purina dog food on the street in front of his house.

Feeding the dogs is getting harder. Paying for everything is getting harder.

Liz Ketcham

Rupams business has been slowly declining for months. When the economy is bad, the last thing people want to do is redecorate their houses, he says. Worried about the future of his company, he started teaching his employees the skills they would need to start their own businesses.

Regardless, he tries to stay positive, especially around his fatherhes seen too many children turn cold and resentful towards their parents under the financial strain of treatment.

He coos and zooms in on the well-groomed hounds as he goes through photos, occasionally stopping to take a sip from a clay cup of chai. In one, a dog with short white fur and pink ears is flashing a soft, squinty smile at the camera. In another, Rupam nuzzles into the dogs fur as the brown-and-white mutt playfully nibbles at the hand wrapped around him.

When he tells his story, he speaks calmly and matter-of-factly. If his mother were at the table, though, he wouldnt be able to keep it together, he said.

Hes wracked with guilt over the whole situationthat his oldest daughter walks dogs to help pay for her sisters tuition, that he had to take back money from his mother to cover the cost of medication.

Rupam doesnt blame his father for wanting the medicine, but he wouldnt choose enzalutamide for himself: I would opt for euthanasia any day.

RELATED: Developers Are Attempting to Evict an Elderly Cancer Patient from Her WeHo Apartment

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UCLA's Fight to Patent a Life-Saving Cancer Drug Could Make the Medicine Virtually Unobtainable in India - LA Magazine

Recommendation and review posted by Bethany Smith