Archive for December, 2020

Joceline Colvert was diagnosed with relapsing remitting Multiple Sclerosis in her early 20s and says she spent the first eight years researching and managing her condition while trying to mention it as little as possible to others and completing her Sound Production degree.

I spent most of my late 20s and early 30s finding ways to manage relapses, the symptoms of which have included whole body numbness, loss of the use of both hands, right eye blindness, vertigo and double vision, she said. Thankfully these symptoms did resolve however left scarring on my nerves. This results in reduced vision in my formerly blind eye and hands that dont function very well with repetitive tasks.

This semi-denial worked for me until about 2010 when I started to become a bit limpy which I did my best to hide. After a couple of memorable falls and fractures I decided to face up to being slightly rickety and got a hiking pole that I used occasionally in public. Since then Ive needed to get used to being visibly disabled, and switch between two hiking poles for very short distances and a wheelchair everywhere else.

Joceline, who lives with her husband and her five beloved cats and dogs, says she is not eligible for Haematopoietic Stem Cell Transplantation (HSCT), on the NHS which is the first treatment I have ever got excited about and believe could work. It could be truly life-changing.

As a result she is trying to raise money to fund the treatment herself.

HSCT is a procedure that aims to reset the faulty immune system which, in my case, is attacking my nervous system from within, Joceline said. Stem cells will be taken from my bone marrow or blood before my immune system is wiped out with chemotherapy. My cells are then reintroduced into my blood, where they grow a new immune system which will hopefully no longer attack my nerves or have any memory of MS.

The aim of HSCT is to completely halt progression, putting MS into remission with no requirement for immunosuppressant drug therapy. The success rate for relapsing remitting MS is 80% - 90% which is absolutely phenomenal compared to the limited available drug treatments, which only aim to slow down disability.

HSCT is available on the NHS, however there is a very strict criteria for which I do not qualify. The expense of the treatment and the increased pressures on the public purse mean the NHS will only treat patients who have been diagnosed for fewer than 15 years.

I have been diagnosed for 18 years.

I had prepared myself for this possibility and, for the last year, have been researching treatment with The National Pirogov Medical Centre Russia (Moscow). Russia has been pioneering in their use of HSCT to treat MS and are world renowned for their expertise and care. Im excited to have a treatment date in March 2021 which fills me with hope for a future free from progression. I need your help to get there.

Joceline, who loves making stop-motion animation puppets and props and playing musical instruments, says the treatment will cost 40,800, and the flights 800.

She has launched a Go Fund Me page at https://gf.me/u/y538k2 which has already seen donations of more than 26,000.

I am incredibly grateful for any help you can give towards enabling me to access this life-changing treatment, she said.

After almost two decades of managing MS flare-ups and their consequences, its hard to put into words just what a future without them would mean to me.

Thank you for reading this and for any help you can put towards this goal.

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Haywards Heath woman's bid to fund stem cell treatment to combat MS - Mid Sussex Times

Dublin, Dec. 09, 2020 (GLOBE NEWSWIRE) -- The "Alopecia Market Share, Growth & Analysis, By Disease, By Application, By Sales Distribution, By Gender And Segment Forecasts, 2016-2026" report has been added to ResearchAndMarkets.com's offering.

The growing prevalence of diseases that trigger alopecia, such as hyperthyroidism, hypopituitarism, lupus, acute stress disorder, and diabetes, are stimulating market growth.

Market Size - USD 9.08 billion in 2019, Market Growth - CAGR of 5.1%, Market Trends - Growing focus on aesthetic appearance and rise in disposable incomes.

The Global Alopecia Market size is expected to reach 13.65 Billion from USD 9.08 Billion in 2019, delivering a CAGR of 5.1% through 2027. The market growth is driven by the growing prevalence of chronic disorders, such as celiac disease, hypothyroidism, hyperthyroidism, Hodgkin's disease, acute stress disorder, hypopituitarism, Hashimoto's disease, lupus, diabetes, Addison's disease, and others, which trigger alopecia in patients.

Changing lifestyle habits, such as overconsumption of alcohol and tobacco and growing stress levels, are resulting in an increased number of alopecia cases globally. Increasing focus on physical appearance and rising disposable incomes are favoring industry growth.

The increasing prevalence of oral treatments, licensed topical treatments, such as minoxidil, finasteride, and surgical procedures, such as hair transplantation or replacement, is expected to boost alopecia market growth over the forecast period.

Further key findings from the report suggest

Key report features:

Key Topics Covered:

Chapter 1. Market Synopsis

Chapter 2. Executive Summary

Chapter 3. Indicative Metrics

Chapter 4. Alopecia Market Segmentation & Impact Analysis

Chapter 5. Alopecia Market By Disease Insights & Trends

Chapter 6. Alopecia Market By Sales distribution Insights & Trends

Chapter 7. Alopecia Market By Gender Insights & Trends

Chapter 8. Alopecia Market By Application Insights & Trends

Chapter 9. Alopecia Market Regional Outlook

Chapter 10. Competitive Landscape

Chapter 11. Company Profiles

For more information about this report visit https://www.researchandmarkets.com/r/jrlv6p

About ResearchAndMarkets.comResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

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

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Global Alopecia Market is Forecast to Deliver a CAGR of 5.1% Between 2019 and 2027 - GlobeNewswire

To get sample Copy of the report, along with the TOC, Statistics, and Tables please visit @https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-induced-pluripotent-stem-cells-market

Some of the key players profiled in the report areFUJIFILM Holdings Corporation, Astellas Pharma Inc, Fate Therapeutics, Bristol-Myers Squibb Company, ViaCyte, Inc., CELGENE CORPORATION, Vericel Corporation, KCI Licensing, Inc, STEMCELL Technologies Inc., Japan Tissue Engineering Co., Ltd., Organogenesis Holdings Inc, Lonza, Takara Bio Inc., Horizon Discovery Group plc, Thermo Fisher Scientific.

Global Induced Pluripotent Stem Cells (iPSCs) Market Scope and Market Size

Induced pluripotent stem cells (iPSCs) market is segmented of the basis of derived cell type, application and end- user. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.

Key Developments in the Market:

In March 2018, Kaneka Corporation announced that they have acquired a patent in the Japan for the creation of the method to mass-culture pluripotent stem cells including iPS cells and ES cells. This will help the company to use the technology to produce high quality pluripotent stem cells which can be used in the drug and cell therapy.

In March 2015, Fujifilm announced that they have acquired Cellular Dynamics International. The main aim of the acquisition is to expand their business in the iPS cell-based drug discovery support service with the use of CDS technology. It will help them to product high- quality automatic human cells with the help of the induced pluripotent stem cells. This will help the company to be more competitive in the drug discovery and regenerative medicine.

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Global Induced Pluripotent Stem Cells (iPSCs) Market Drivers:

Increasing R&D investment activities is expected to create new opportunity for the market.

Increasing demand for personalized regenerative cell therapies among medical researchers & healthcare is expected to enhance the market growth. Some of the other factors such as increasing cases of chronic diseases, growing awareness among patient, rising funding by government & private sectors and rising number ofclinical trialsis expected to drive the induced pluripotent stem cells (iPSCs) market in the forecast period of 2020 to 2027.

High cost of the induced pluripotent stem cells (iPSCs) and increasing ethical issues & lengthy processes is expected to hamper the market growth in the mentioned forecast period.

Table of Contents:

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Global Induced Pluripotent Stem Cells (iPSCs) Market Expectable to Exceed Global Market Revenue, Size, Segments and Market Competition Trend to...

Commercial constellations set new standards for the industry in numbers but GEO comsat and government still drive the biggest demand in manufacturing and launch revenues

In its latest analysis of satellite manufacturing and launch services, Satellites to be Built and Launched by 2029, analyst Euroconsult anticipates almost a quintupling in satellite demand in the next decade with an average of 1,250 satellites to be launched on a yearly basis. In comparison to the 260 yearly satellites launched in the previous decade, this skyrocketing number cements the structural changes occurring in the market and the industry, not only in the number of satellites but also in terms of satellite missions and operators, both governmental and commercial.

The satellite industry will indeed experience a quick and radical transformation when it comes to satellite numbers. However, despite this spike in satellite demand, we are looking at half of the market concentrated around a handful of mega-constellations. In addition, some being vertically integrated means that their procurement will not be done on an open competition basis. Nevertheless, GEO comsat remains the leading segment pulling 1/3 of the market revenues, but here too we anticipate -20% drop in operational assets by 2029. argued Maxime Puteaux, Editor-in-Chief of this research product and Principal advisor at Euroconsult.

Several key market trends are catalysing the satellite industrys structural changes.

Firstly, for the first time in a single year more than 1000 satellites were launched, of which 70% from Starlink alone. This symbolic threshold will become a new standard for the next ten years with significant annual variations mainly driven by the replacement of the commercial constellations.

Secondly, the orders of GEO comsat have been exceptionally high in 2020 at 18 units, of which 13 are for the accelerated C-band clearance plan of the FCC in the US. In addition, proof of a structural slowdown of that market in satellite numbers, GEO comsat replacement is also challenged by fleet rationalisation approaches, in-orbit life extension and transitioning of some of the traffic to upcoming NGSO constellations. Manufacturers GEO comsat product portfolios are diversifying, ranging from a few hundred kilograms to 6 Tons VHTS. Digital payloads become the rule for a data-centric market (rather than a broadcasting market).

Thirdly, beyond the commercial space momentum, governments will remain the first customers with 80% satellite manufacturing and launch revenues for the period. Investments by defence operators is driven by security applications and a growing endorsement of smallsats, COTS and constellations while civil agencies focus on large Earth observation systems.

Lastly, access to the space industry is diversifying with a few smallsat-dedicated launchers now operational and more expected to perform maiden flights in 2021. A new generation of GTO-capable launchers is expected to enter the market within the next two years with a design-to-cost approach. Meanwhile, SpaceX masters reusability and executes Starlinks launches at marginal cost, with Falcon 9 recovery and reuse becoming a standard endorsed by customer.

Original post:
Commercial constellations don't live up to the hype: Euroconsult - SatelliteProME.com

The globalAntioxidant Supplement Marketresearch report enlists the vital and practical information with regards to market situation. The present scenario of Antioxidant Supplement market, along with its previous performance as well as future scope are covered in the report. This eases the users understanding of the market thoroughly, while also gaining knowledge about market opportunities and the dominant players Puritan, GNC, Jarrow Formulas, Bulkpowders, NOW, Biocare, Life Extension, Vibrant Health, Cytoplan, AST R-ALA in the Antioxidant Supplement market.

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The Global Antioxidant Supplement Market Research Report Details

The beginning of the report summarizes the market with the definition of the overall Antioxidant Supplement market.

The following section includes Antioxidant Supplement market segmentation Medical Grade, Food Grade. Segmentation is done on the basis of application, type, end-user industries, and several such factors among others.

We have strived to include sub-segments Medical, Food, Cosmetics, Others in segmentation section, wherever possible. Also included are details regarding the dominant segments in the worldwide Antioxidant Supplement market.

The global Antioxidant Supplement market has also been classified on the basis of regions. On the basis of the regional diversification, details regarding market share and size have also been obtained.

In the succeeding part, growth factors for the Antioxidant Supplement market have been elucidated. This section also explains the technological advancements made to improve market size and position. Also enlisted is the information pertaining to the end-use industries for the Antioxidant Supplement market.

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Antioxidant Supplement Market COVID-19 Impact Analysis

As the world is still dealing with COVID-19 situation, many of the countries have slowly started to revive its economic situation by starting its trade and businesses. There has been enormous loss in these few months both in terms of economy and human lives. As the WHO has already suggested that there are very less chances that the virus will completely go, hence we will have start living with it. Many of the drug companies are getting positive response of their COVID-19 vaccines, but there is still time for its availability in the global market.

There are 15 Sections to show the global Antioxidant Supplement market

Sections 1, Definition, Specifications and Classification of Antioxidant Supplement , Applications of Antioxidant Supplement , Market Segment by Regions;Section 2, Assembling Cost Structure, Crude Material and Providers, Assembling Procedure, Industry Chain Structure;Sections 3,Technical Data and Manufacturing Plants Analysis of Antioxidant Supplement , Capacity and Commercial Production Date, Manufacturing Plants Distribution, R&D Status and Technology Source, Raw Materials Sources Analysis;Sections 4, Generally Market Analysis, Limit Examination (Organization Fragment), Sales Examination (Organization Portion), sales Value Investigation (Organization Section);Sections 5 and Six, Regional Market Investigation that incorporates United States, China, Europe, Japan, Korea and Taiwan, Antioxidant Supplement segment Market Examination (by Sort);Sections 7 and Eight, The Antioxidant Supplement Segment Market Analysis (by Application) Major Manufacturers Analysis of Antioxidant Supplement ;Sections Nine, Market Trend Analysis, Regional Market Trend, Market Trend by Product Type Medical Grade, Food Grade Market Trend by Application Medical, Food, Cosmetics, Others;Sections 10, Regional Promoting Type Investigation, Worldwide Exchange Type Examination, Inventory network Investigation;Sections 11, The Customers Examination of global Antioxidant Supplement;Sections 12, Antioxidant Supplement Research Findings and Conclusion, Appendix, system and information source;Sections 13, 14 and 15, Antioxidant Supplement deals channel, wholesalers, merchants, traders, Exploration Discoveries and End, appendix and data source.

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Particulars Of The Global Antioxidant Supplement Market Research Report

Further part in the report enlists the restraining factors for the Antioxidant Supplement market growth. The restraints are explained comprehensively and with details in order that the client can comprehend how these factors are affecting the global Antioxidant Supplement market and how such factors can be tackled effectively using suitable measures.

Also, regional study and analysis of global Antioxidant Supplement market focused on in the report. Here, the major regions with Antioxidant Supplement market establishment have been explained thoroughly. Due to this, our clients will have clarity in understanding the booming markets as well as the potential Antioxidant Supplement markets in the near future.

The concluding section relates to the conclusions and observations regarding the global Antioxidant Supplement market.

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Global Antioxidant Supplement Market Proceeds To Witness Huge Upswing Over Assessment Period by 2025 - The Courier

Three years ago, pictures of bulk carriers queued off the coast of Mackay in central Queensland were framed as evidence of a renaissance in the coal industry.

There were more than 70 coal ships in the offshore gridlock in December 2017. This year there are just 12 waiting equalling a record low mark set at the height of the coronavirus pandemic.

At the worlds biggest coal export port in Newcastle, no China-bound ships are waiting or scheduled to load before Christmas. More than 50 ships carrying Australian coal are reportedly waiting off the Chinese coast.

In the face of falling coal prices and volumes, the industry and governments have remained bullish about coals long-term prospects. They say twin pressures of the pandemic slowdown and Chinas ban on Australian coal will ultimately pass.

In an apparent show of faith, the Queensland government took a 9.9% stake in the float of Dalrymple Bay Infrastructure (DBI) one of two large terminals in Mackay, the main hub for exports from the Bowen Basin. The investment was welcomed by the resources sector as a clear vote of confidence ... in the role of resources in Queenslands Covid-19 recovery and economic growth for decades to come.

DBI was launched on the Australian stock exchange this week with government backing and the broader market surging. It was the second biggest Australian IPO this year (the largest, tech company Nuix, gained 63% on debut last week). DBI also promised investors a handsome 7% dividend.

When the stocks hit the market, DBI tanked, down 16%. It gained no ground the following day. Investors were not buying the pitch that coal has a rosy future.

Its a pivotal moment, says Tim Buckley, an energy markets expert at the Institute of Energy Economics and Financial Analysis.

The financial markets do move so much faster than the real world, they are all about constantly reevaluating the risk-return and growth prospects.

Theres no long-term growth prospect at all for the [coal] industry. Its like trying to catch a falling knife.

Australias biggest super fund, AustralianSuper, has committed to hitting net zero emissions across its $200bn investment portfolio by 2050 but has not specifically ruled out investing in coal projects.

Nonetheless, its chief executive, Ian Silk, seems less than enthusiastic about the idea.

The economic outlook for coal stocks generally is incredibly bleak, for obvious reasons, he says.

He says Aussie would approach any particular coal project on its financial merits.

But its pretty plain by the way were so underweight coal that thats not an attractive sector, he says.

Silk is not alone. Institutional investors the big pension funds and other piles of money that provide much of the capital businesses need to operate have increasingly turned away from coal and other fossil fuels.

Norways Government Pension Fund Global, which at US$1.2tn is so big it holds about 1.5% of all the shares in listed companies in the world, has strict rules forbidding it from investing in companies that produce more than 20m tonnes of thermal coal a year or produce power of more than 10,000MW a year from burning coal.

As a result, in May it excluded from investment two big multinationals that mine coal in Australia, Glencore and Anglo American, as well as Australian power company AGL Energy.

It also put BHP on notice that it could dump its stake in the Big Australian if it didnt get out of thermal coal.

In Australia, the big banks have displayed an increasing unwillingness to lend to coal, with ANZ in October saying it would not write new loans to businesses with more than 10% exposure to thermal coal and existing customers with more than 50% exposure would need to show it specific, time bound and public diversification strategies to continue receiving the banks cash.

The harder line from banks followed warnings from their regulator, the Australian Prudential Regulation Authority, that they needed to consider climate risks when making decisions.

Apra member Geoff Summerhayes laid down the regulators position in a speech in 2017 that was met with howls of dismay and derision by some.

This week, he said the criticism was good impetus for me to actually go harder, because its very much a financial risk with real prudential implications.

It is easy to blame the failure of Australian coal prospects to re-emerge from the pandemic on the situation in China. However, there are growing signals the industry is heading into its final bust cycle.

A few days before the Dalrymple Bay terminal was floated, the largest coal producer in Australia and the western world, Glencore, released its annual investor update and critically announced new plans for a managed decline of its coal business and net-zero emissions by 2050.

While those plans are ultimately long-term (and also play to the companys strategic interest by seeking to keep prices at viable levels by constraining supply) they also show the company expects volumes to drop substantially up to 20% in the next few years, compared to previous projections.

The same investor update last year envisaged Glencore would produce 140m tonnes of coal in 2022. Now the company only expects to mine 115m tonnes that year. It might consider mine-life extension projects but has no plans to develop new coal mines.

Coal is also on the nose at Australias two big mining companies, BHP and Rio Tinto, which have turned away from the black rock and towards red ones as the iron ore price continues to soar.

Rio Tinto sold its last Australian coal mine in 2018 and, under pressure from investors, BHP has promised to get out of thermal coal burned in power plants within two years but so far has found no buyers.

Of particular concern to miners in Queensland is the way financial markets have treated metallurgical (or coking) coal, which is used in steelmaking. More than 80% of the exports from Dalrymple Bay are metallurgical coal.

In the days before the DBI float, company chief executive Anthony Timbrell told the Australian Financial Review it would seek to emphasise the difference between metallurgical and thermal (energy producing) coal.

I guess its our job to draw out that story and remind people of the complexity, Timbrell said.

Thermal coal is the primary target of environmental activists; while metallurgical coal is less susceptible in the immediate-term to a global energy pivot towards renewables.

BHP has also been keen to draw the distinction, which is in its financial interests as metallurgical coal attracts a higher price than thermal coal.

However, as excitement builds around the prospect of (as yet, not commercialised) steelmaking alternatives like green hydrogen, the financial markets increasingly appear to be making little differentiation between the classes of coal.

Buckley points to a graph comparing the US and Australian metallurgical coal producer Coronado with Australian company Whitehaven, which largely mines thermal coal.

Since Coronado was listed in 2018, both Coronado and Whitehavens shares have dived almost in harmony by about 65%. The All Ordinaries is up about 20% over the same period.

The financial markets are no longer really differentiating between coking coal and thermal coal, Buckley said.

Dalrymple Bay is a really interesting bellwether for Queensland. Having already been priced down, having failed to get institutional support, taxpayers effectively did a bailout.

The vendor (Brookfield Asset Management) is the most successful investor in energy infrastructure, and you dont buy from the most successful energy investor in the world and think youre getting a bargain.

This isnt a resources sector problem either, this is a coal problem. The Australian resource sector is having the best year in history, iron ore prices are at phenomenal highs. Its the fossil fuel sector thats on its knees.

The coal company run by John Canavan, the brother of Queensland senator Matt Canavan, went under earlier this month.

The company, ICRA Rolleston, is a junior joint venture partner with Glencore in the Rolleston thermal coal mine in Queensland. Glencore will continue to operate the mine but a court case finalised last month showed how the collapse in the coal price had turned the mine into a loss-making venture.

John Canavans share of the mines costs were about $14m more than sales revenue in August. Glencore expected another $4m shortfall by the end of the year.

The Queensland Exploration Council (an offshoot of the Resources Council) this week released a report card showing some growth in spending by coal speculators during 2019-20 and said there was definitely a feeling of growth and optimism in the sector. In the detail of its report, though, for the first time in four years the QEC downgraded its view on coal prices, saying these has become cause for concern.

In its prospectus for potential investors, DBI warned about a series of risks that included its customers collapsing due to low coal prices, or long-term decline in global coal demand.

Its most significant new investor, the Queensland government, released a study in September that stated there is a substantial degree of uncertainty about assumptions used to underpin long-term market projections, including about the price of coking coal.

Queensland Treasurys analysis highlights that Queenslands future coal demand will continue to be primarily linked to key economies in north-east and south-east Asia. In particular, the future demand for Queenslands metallurgical coal likely hinges on demand from the worlds two largest coal consumers, China and India.

DBIs warnings included that ongoing political tensions between Australia and China could ultimately result in a decline in coal exports from the port.

Demand for metallurgical coal or coal generally may reduce over a period of time due to a variety of reasons, including reduced demand from key coal export markets, such as China, Japan, Taiwan, South Korean and India.

In addition to Chinas import restrictions, China, Japan and South Korea Australias three largest coal customers each announced aggressive pivots towards net zero emissions this year.

Adding to DBIs troubles is the nature of its business where contracts with exporters are regulated by a competition authority. The ports capacity is fully allocated. Unable to raise prices or attract new customers, its pitch to investors has been about expanding its capacity to grow the business, even as shipments are being shunned by China, export volumes contract, coal companies collapse and other Queensland ports face severe debt problems.

Of those terminals, Wiggins Island at Gladstone and the Abbot Point terminal near Bowen, owned by Adani, have both been operating at well below design capacity for all of this decade, a point Buckley and others say shows any expansion of Dalrymple Bay is not viable.

Abbot Point where Adanis debts are estimated in excess of $1.5bn typically has a queue of about three coal ships. Earlier this week, there were no ships waiting to enter the port.

Link:
The end of coal? Why investors aren't buying the myth of the industry's 'renaissance' - The Guardian

Researchers in California aim to develop a new CRISPR-based test for COVID-19 that can be read anytime, anywhereby turning a basic smartphone camera into a microscope capable of detecting the coronavirus genetic material.

The team consists of scientists from the University of California, San Francisco, UC Berkeley, and the Gladstone Institutesincluding a collaboration with Jennifer Doudna, president of the Innovative Genomics Institute, and winner of the 2020 Nobel Prize in Chemistry for co-discovering CRISPR-Cas genome editing, the technology that underpins the test.

"It has been an urgent task for the scientific community to not only increase testingbut also to provide new testing options," said Melanie Ott, director of the Gladstone Institute of Virology and one of the leaders of a study evaluating the test, published in Cell. "The assay we developed could provide rapid, low-cost testing to help control the spread of COVID-19."

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The smartphone diagnostic aims to provide a positive or negative result in less than 30 minutes, as well as gauge the amounts of SARS-CoV-2 virus present in a nasal swab sample.

"When coupled with repeated testing, measuring viral load could help determine whether an infection is increasing or decreasing," said UC Berkeley bioengineer Daniel Fletcher, a Chan Zuckerberg Biohub investigator. "Monitoring the course of a patient's infection could help health care professionals estimate the stage of infection and predict, in real time, how long is likely needed for recovery."

RELATED: Jennifer Doudna's new CRISPR company will tackle disease detection

Most current molecular tests for COVID-19 are based on PCRa method that requires the virus RNA to be converted into DNA before the diagnostics can work. It also needs to amplify that DNA, by repeatedly making copies to capture a detectable signal, which calls for specialized chemical reagents and laboratory equipment.

Alternatively, the researchers approach uses CRISPR proteins designed to hunt directly for the virus RNA, skipping the conversion and amplification steps.

"One reason we're excited about CRISPR-based diagnostics is the potential for quick, accurate results at the point of need," said Doudna, whose CRISPR-focused company Mammoth Biosciences was tapped earlier this year by GlaxoSmithKline to develop an over-the-counter COVID-19 test.

"This is especially helpful in places with limited access to testing, or when frequent, rapid testing is needed. It could eliminate a lot of the bottlenecks we've seen with COVID-19," she said.

The new test uses a Cas13 protein tagged with a molecule that glows once its cut, as part of the genetic-snipping that occurs when it matches up with a specific piece of RNA. When more of the virus genome is present, more cuts occurcreating a brighter glow that can be picked up by a smartphone camera used with a darkened box.

RELATED: Stanford develops CRISPR 'lab on a chip' for detecting COVID-19

The team of researchers had originally been pursuing the quick testing method as a potential diagnostic for HIV, but pivoted to the coronavirus as the pandemic began to spread this year.

"We knew the assay we were developing would be a logical fit to help the crisis by allowing rapid testing with minimal resources," co-first author Parinaz Fozouni, a UCSF graduate student working in Ott's lab at Gladstone. "Instead of the well-known CRISPR protein called Cas9, which recognizes and cleaves DNA, we used Cas13, which cleaves RNA."

The study also found that samples with high concentrations of the virus produced a signal much faster, with positive results for someone who potentially more likely to be contagious delivered in under 5 minutes.

"Recent models of SARS-CoV-2 suggest that frequent testing with a fast turnaround time is what we need to overcome the current pandemic," said Ott. "We hope that with increased testing, we can avoid lockdowns and protect the most vulnerable populations."

In addition to being widely available and cheaper compared to lab equipment, smartphones could also make use of their GPS and digital connectivity to help track the spread of infections in various regions.

"We hope to develop our test into a device that could instantly upload results into cloud-based systems while maintaining patient privacy, which would be important for contact tracing and epidemiologic studies," Ott said. "This type of smartphone-based diagnostic test could play a crucial role in controlling the current and future pandemics."

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Pairing CRISPR with a smartphone camera, this COVID-19 test finds results in 30 minutes - FierceBiotech

Identifying and isolating individuals who may be contagious with the coronavirus is key to limiting the spread of the disease. But even months into the pandemic, many patients are still waiting days to receive COVID-19 test results.

Scientists at UC Berkeley and Gladstone Institutes have developeda new CRISPR-based COVID-19 diagnostic testthat, with the help of a smartphone camera, can provide a positive or negative result in 15 to 30 minutes. Unlike many other tests that are available, this test also gives an estimate of viral load, or the number of virus particles in a sample, which can help doctors monitor the progression of a COVID-19 infection and estimate how contagious a patient might be.

Monitoring the course of a patients infection could help health care professionals estimate the stage of infection and predict, in real time, how long is likely needed for recovery and how long the individual should quarantine, said Daniel Fletcher, a professor of bioengineering at Berkeley and one of the leaders of the study.

The technique was designed in collaboration with Dr. Melanie Ott, director of the Gladstone Institute of Virology, as well as Berkeley professor Jennifer Doudna, who is a senior investigator at Gladstone, president of the Innovative Genomics Institute and a Howard Hughes Medical Institute investigator. Doudna recently won the 2020 Nobel Prize in Chemistry for co-discovering CRISPR-Cas genome editing, the technology that underlies this work.

Most COVID-19 diagnostic tests rely on a method called PCR, short for polymerase chain reaction, which searches for pieces of the SARS-CoV-2 viral RNA in a sample. These PCR tests work by first isolating the viral RNA, then converting the RNA into DNA and then amplifying the DNA segments making many identical copies so that the segments can be more easily detected.

The new diagnostic test takes advantage of the CRISPR Cas13 protein, which directly binds and cleaves RNA segments. This eliminates the DNA conversion and amplification steps and greatly reduces the time needed to complete the analysis.

One reason were excited about CRISPR-based diagnostics is the potential for quick, accurate results at the point of need, Doudna said. This is especially helpful in places with limited access to testing or when frequent, rapid testing is needed. It could eliminate a lot of the bottlenecks weve seen with COVID-19.

In the test, CRISPR Cas13 proteins are programmed to recognize segments of SARS-CoV-2 viral RNA and then combined with a probe that becomes fluorescent when cleaved. When the Cas13 proteins are activated by the viral RNA, they start to cleave the fluorescent probe. With the help of a handheld device, the resulting fluorescence can be measured by the smartphone camera. The rate at which the fluorescence becomes brighter is related to the number of virus particles in the sample.

Recent models of SARS-CoV-2 suggest that frequent testing with a fast turnaround time is what we need to overcome the current pandemic, Ott said. We hope that with increased testing, we can begin to reopen economies and protect the most vulnerable populations.

Now that the CRISPR-based assay has been developed for SARS-CoV-2, it could be modified to detect RNA segments of other viral diseases, like the common cold, influenza or even human immunodeficiency virus. The team is currently working to package the test into a device that could be made available at clinics and other point-of-care settings and that one day could even be used in the home.

The eventual goal is to have a personal device, like a mobile phone, that is able to detect a range of different viral infections and quickly determine whether you have a common cold or SARS-Cov-2 or influenza, Fletcher said. That possibility now exists, and further collaboration between engineers, biologists and clinicians is needed to make that a reality.

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New CRISPR-based COVID-19 test uses smartphone cameras to spot virus RNA - University of California

A solid clinical trial update convinced several Wall Street analysts to raise their price targets for CRISPR Therapeutics (NASDAQ:CRSP) on Thursday. Those new expectations include a bold $170 target from investment bank Needham -- about 16% higher than the biotech's closing price Wednesday.

The candidate generating all this enthusiasm is CTX001, an experimental therapy for two hemoglobin-based disorders: beta-thalassemia and sickle cell disease. CRISPR Therapeutics is developing it in partnership with Vertex Pharmaceuticals (NASDAQ:VRTX). This single-application treatment involves reengineering a patient's own stem cells to produce fetal hemoglobin once they've been reinfused, and it appears to work as intended.

Image source: Getty Images.

At the latest update, seven beta-thalassemia patients had been under post-therapy observation for least three months, and so far, none have required transfusions. Among three patients with sickle cell disease who also have been observed for at least three months, none have experienced a vaso-occlusive crisis since undergoing the therapy.

CRISPR Therapeutics has also administered CTX001 to 10 patients who hadn't had their three-month observations in time for their data to be included in the company's recent presentation. In the summer of 2021, though, we should know if CTX001 has a shot at competing against LentiGlobin, an experimental treatment from bluebird bio (NASDAQ:BLUE).

LentiGlobin aims to treat the same limited groups of patients as CTX001, and it's been in development longer. Recently, bluebird bio published results from BCL11A, a treatment that more closely resembles CTX001, and the early data looks competitive.

There is a good chance that CTX001 and experimental cancer treatments rolling through CRISPR Therapeutics' pipeline will drive the stock to $170 and beyond. However, based on the potential for competition from bluebird bio, and considering the usual issues that clinical-stage biotechs must navigate, the company faces significant risks. Investors who buy this stock should only do so within a well-diversified portfolio.

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Is CRISPR Therapeutics Heading for $170? - The Motley Fool

Demonstrates modularity of Intellias in vivo liver insertion technology to durably restore protein, compared to traditional gene therapy

Single-course administration of genome editing system provides potentially curative approach to AAT deficiency

CAMBRIDGE, Mass., Dec. 12, 2020 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), is presenting the first demonstration of physiological protein levels of human alpha-1 antitrypsin (AAT) in non-human primates (NHPs) following a single administration. Compared to traditional adeno-associated virus (AAV) gene therapy, Intellias targeted liver gene insertion technology has the ability to achieve therapeutic levels of protein expression, in a stable and durable manner, after a single course of treatment. The company is presenting these data today at the Alpha-1 Foundations 20th Gordon L. Snider Critical Issues Workshop: The Promise of Gene-Based Interventions of Alpha-1 Antitrypsin Deficiency.

Our new data reinforce the promise for Intellia to potentially cure a variety of rare genetic diseases requiring the restoration of a functional protein in the liver with a single-course therapy, said Intellia President and Chief Executive Officer John Leonard, M.D. Weve now demonstrated our platforms modularity and translatability to multiple targets of interest by inserting genes to durably produce unprecedented levels of protein in NHPs for hemophilia B and AAT deficiency. In parallel with advancing to the clinic treatments for other severe diseases, we will continue preclinical studies that further validate our wholly owned, CRISPR-based AAT deficiency treatment strategies for achieving normal AAT protein levels.

Presentation Details

Title: CRISPR/Cas9-Mediated Targeted Gene Insertion of SERPINA1 to Treat Alpha-1 Antitrypsin DeficiencySession: Gene EditingTime: 3:15 p.m. ETPresenting Author: Sean Burns, M.D., senior director of Intellias Disease Biology and Pharmacology group

Intellia is advancing multiple genome editing strategies that may treat both lung and liver manifestations of AAT deficiency (AATD), which occur due to mutations in the SERPINA1 gene. The normal human AAT protein levels Intellia achieved following targeted insertion of the human SERPINA1 gene remained stable through 11 weeks in an ongoing NHP study. The observed levels of human AAT protein produced from the liver may be therapeutically sufficient to restore protease inhibition to protect the lungs and liver from improperly regulated neutrophil elastase activity. The NHP data build on previous resultsshowing that consecutive in vivo genome editing (knockout plus insertion) achieved therapeutically relevant results in an AATD mouse model.

The findings being presented today reinforce recent data showing the use of the same proprietary insertion technology for targeted gene insertion ofFactor 9 resulted in circulating human Factor IX, a blood-clotting protein that is missing or defective in hemophilia B patients, that ranged from normal levels (50-150%)1 to supratherapeutic levels in a six-week NHP study. Intellia and Regeneron, the lead party, are co-developing potential hemophilia A and B CRISPR/Cas9-based treatments using their jointly developed targeted transgene insertion capabilities. Intellia is continuing to develop its proprietary platform to advance its wholly owned research programs, such as AATD. Click here to register for the Alpha-1 Foundations virtual workshop and here to view Intellias presentation on the companys website.

____________________________1 National Hemophilia Foundation

About Alpha-1 Antitrypsin Deficiency and Intellias Genome Editing Treatment Approach

The SERPINA1 gene normally encodes the alpha-1 antitrypsin (AAT) protein produced in the liver that is then secreted to protect the lungs. SERPINA1 mutations can cause AAT deficiency (AATD), a rare, genetic disease that commonly manifests in lung dysfunction, as well as in liver disease in some patients. Intellias targeted in vivo insertion platform uses a hybrid delivery system combining a non-viral lipid nanoparticle (LNP), which encapsulates CRISPR/Cas9 components, with an adeno-associated virus (AAV) carrying a donor DNA template to enable therapeutic protein production. One of the editing strategies Intellia is studying as a potential single-course AATD treatment is using the companys SERPINA1 gene insertion approach to restore normal human AAT protein levels. Intellia also is investigating a consecutive genome editing approach, in which the PiZ allele, the most prevalent disease-causing mutation of SERPINA1, is knocked out and the normal human SERPINA1 gene is inserted.

AboutIntellia TherapeuticsIntellia Therapeuticsis a leading genome editing company, focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by both producing therapeutics that permanently edit and/or correct disease-associated genes in the human body with a single treatment course, and creating enhanced engineered cells that can treat oncological and immunological diseases. Intellias combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts it in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create new classes of therapeutic products. Learn more aboutIntellia and CRISPR/Cas9 atintelliatx.com. Follow us on Twitter@intelliatweets.

Forward-Looking Statements This press release contains forward-looking statements of Intellia Therapeutics, Inc. (Intellia or the Company) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellias beliefs and expectations regarding its: plans to advance and complete preclinical studies, including any necessary non-human primate studies, for its hemophilia A, hemophilia B, and other in vivo and ex vivo research and development programs, such as its AATD research program; development of a proprietary LNP/AAV hybrid delivery system, as well as its modular platform to advance its complex genome editing capabilities, such as gene insertion, as well as knockout editing capabilities; advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as its ability to maintain and expand its related intellectual property portfolio; ability to demonstrate its platforms modularity and replicate or apply results achieved in preclinical studies, including those in its hemophilia A and hemophilia B programs and its AATD research program, in any future studies, including human clinical trials; ability to develop other in vivo or ex vivo cell therapeutics of all types using CRISPR/Cas9 technology; expectations of the potential impact of the coronavirus disease 2019 pandemic on strategy, future operations and timing of its clinical trials or IND submissions; ability to optimize the impact of its collaborations on its development programs, including but not limited to its collaborations with Regeneron, including its co-development programs for hemophilia A and hemophilia B; statements regarding the timing of regulatory filings regarding its development programs; use of capital, expenses, future accumulated deficit and other 2020 financial results or in the future; and ability to fund operations at least through the next 24 months.

Any forward-looking statements in this press release are based on managements current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellias ability to protect and maintain its intellectual property position; risks related to Intellias relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation and conduct of studies and other development requirements for its product candidates; the risk that any one or more of Intellias product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; and the risk that Intellias collaborations with Regeneron or its other collaborations will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellias actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Intellias most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellias other filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and Intellia undertakes no duty to update this information unless required by law.

Intellia Contacts:

Media:Lynnea OlivarezDirectorExternal Affairs & Communications+1 956-330-1917lynnea.olivarez@intelliatx.com

Investors:Lina LiAssociate DirectorInvestor Relations+1 857-706-1612lina.li@intelliatx.com

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Intellia Therapeutics Achieves Normal Human Alpha-1 Antitrypsin Protein Levels in Non-Human Primates Through Targeted Gene Insertion for the Treatment...

Called de-extinction, the resurrection of lost species is one of the many applications to be revolutionized by the new gene-editing technology CRISPR-Cas9.

[A]ll scientists need are organic remnantssuch as pieces of bonethat contain fragments of DNA. Those fragments allow geneticists to discover the complete genome of the extinct animal (a process scientists refer to as sequencing). Once they have this recipe for the extinct species, CRISPR enables scientists to edit the DNA of its closest living relative to create a genome that, as edited, approximates the genetic code of the extinct species. Think of the living animals DNA as version 2.0 of a piece of software: the goal is to get back to version 1.0. You compare all of the millions of lines of code to spot differences, and then painstakingly edit the lines with differences to restore the code to its original state.

Once the DNA has been edited to reintroduce the key traits of the extinct plant or animal, the edited DNA is inserted into the nucleus of a reproducing cell. The resulting individual may not be genetically identical to the extinct species, but the key traits that made the extinct species unique are reintroduced.

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De-extinction: Why CRISPR gene editing might be the most revolutionary development in science ever - Genetic Literacy Project

Bacteria readily acquires a sequence of other species DNA into their own, in specific areas that we now call CRISPR. In the lab, CRISPR was synthesized by linking together two guide RNA sequences into a format that would provide the target information and allow us to edit multiple genes simultaneously.

Cancer is a genetic disease, it works by creating certain changes to genes that control the way our cells function, especially how they grow and divide. Some rare cancers, sarcomas in particular, have been treated using CRISPR, which is why the gene-editing tool seems like a good diagnostic and therapeutic tool in the future of cancer treatments.

Obtaining rare cancerous tumors for research is difficult, but luckily organizations such as Pattern.org and the Rare Cancer Research Foundation (RCRF) come into play. These sister groups perform a matching program that enables patients to directly donate their tumor tissue and medical data to research. All the data generated by the project is freely available to the research community and is dedicated to open science.

Using Pattern.org, the Broad Institute of MIT and Harvard has created over 40 next-generation de-identified cancer models, Ms. Barbara Van Hare, Director of Foundation partnerships at RCRF said, These models and associated data will be shared within the worldwide research community.

After procuring these rare disease samples, Dr. Jesse Boehm from Eli and Edythe L. Broad Institute might have the answer to decipher the genetic landscape of cancer cells and use that to our advantage. Dr. Boehm is the scientific director of the Broad Institutes Cancer Dependency Map Initiative where he works on the cancer cell line factory project and the cancer dependency map.

Cancer samples are broken apart into cell models and are coaxed into growing in different conditions over a year-long time period. The data from these new cell models are then shared broadly with the world. This is a pipeline activity called the cell line factory. It is a part of an international effort to create a large reference data set, that is called the cancer dependency map.

The cancer dependency map has a two-pronged approach, first by testing cell lines against drugs and then pooled CRISPR screening. First, all cell lines are systematically tested against all drugs developed for any disease. Some known drugs have shown to be effective against certain cancers, clinical trials are swift as these are existing therapies.

There are 20,000 proteins in the human genome and only 6000 drug therapies. Only five percent of human genes can be targeted with drugs. The cancer dependency Map is completed with the help of CRISPR, Dr Boehm said.

Pooled CRISPR screening is used and 100,000 CRISPRs target every gene in the genome. Every cell is challenged with all these CRISPRs and at the end of the experiment, the abundance is compared to the beginning of the experiment.

CRISPR is used to snip genes,the DNA repairs creating a broken gene. Cells that are required for viability die and drop out of the population. CRISPRs are bar coded, so if by the end of the experiment the CRISPR is absent, it targets the gene that the cell needed to survive. The genes that drop out are good drug targets, most of these make way for drug discovery projects right away.

CRISPR is such a sharp tool, it inspires a lot more confidence than its predecessors, Dr Boehm said. He uses the analogy of Google Maps for this project: It needs to tell clinicians what to do and where to go, but for it to be relevant-the data needs to be dense enough in that area.

An additional therapy for cancers involves making four genetic modifications to T cells (immune cells that can kill cancer). It basically adds genes to T cells to fight cancer. One of these is a synthetic gene that gives the T cells a protein that can identify cancer cells better. CRISPR is also used to mute three genes that limit the cells cancer-killing abilities (Stadtmauer et al. 2020). With these limiting genes removed, the T cells are less inhibited to fight cancer.

These therapeutics and the Cancer dependency map will take a few decades to develop but will prove to be a very sharp tool in our arsenal against rare cancers when complete.

References:

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How CRISPR could help us discover and treat rare cancers - ZME Science

THURSDAY, Dec. 10, 2020 (HealthDay News) -- Use of the ex vivo CRISPR-Cas9-based gene-editing platform to edit the erythroid enhancer region of BCL11A in hematopoietic stem and progenitor cells, producing CTX001, results in increased hemoglobin (Hb) among patients with transfusion-dependent -thalassemia (TDT) and sickle cell disease (SCD), according to a study published online Dec. 5 in the New England Journal of Medicine to coincide with the annual meeting of the American Society of Hematology, held virtually from Dec. 5 to 8.

Haydar Frangoul, M.D., from the Sarah Cannon Center for Blood Cancer at the Children's Hospital at TriStar Centennial in Nashville, Tennessee, and colleagues presented safety and efficacy results from patients with at least three months of follow-up from two first-in-human studies of CTX001 for TDT and SCD. Data were included for seven patients with TDT and three with SCD.

The researchers found increases in total Hb and fetal Hb among all patients over time. Patients with TDT stopped receiving packed red blood cell transfusions soon after CTX001 infusion; the first patients with TDT who received CTX001 remained transfusion-free for more than 15 months. Since CTX001 infusion, the patients with SCD have had no vaso-occlusive crises (VOCs); the first patient to receive CTX001 remained VOC-free for more than one year. The safety profile after CTX001 infusion was generally consistent with busulfan myeloablation in all 10 patients. One patient with TDT had four serious adverse events related or possibly related to CTX001.

"By gene editing the patient's own stem cells we may have the potential to make this therapy an option for many patients facing these blood diseases," Frangoul said in a statement.

Several authors disclosed financial ties to pharmaceutical companies, including CRISPR Therapeutics and Vertex Pharmaceuticals, which sponsored the trial.

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ASH: CRISPR-Cas9 Gene Editing Promising in TDT, SCD - HealthDay News

We cannot rewind the tape of life to see how we might have been and whether humans are inevitable products of evolutionary processes, but as Kevin Davies states in his lively and enthralling Editing Humanity: [The CRISPR Revolution and the New Era of Genome Editing], our unprecedented ability to engineer genomes rapidly and efficiently offers humankind the possibility of contemplating what we might become.

Benefiting from his presence at some of the key moments in gene-editing history, and armed with humor and an enthusiastic writing style, Davies provides a compelling account of CRISPRs discovery and the shenanigans accompanying its meteoric ascendance. These include the formation of biotechs, patent disputes, fallouts and disagreements over the limits of responsible editing.

All this culminated in the untimely and unethical use of CRISPR by the scientist He Jiankui to edit the germline DNA of human embryos, an irresponsible and cavalier act that affected the heredity of two girls forever. Daviess account of this sobering episode in CRISPRs short and turbulent history reminds us of the inherent dangers of genome editing and of the ease with which technologies may be subverted for totalitarian ends. Fortunately, many essential human characteristics, including free will, do not reduce to individual genes.

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Kevin Davies' 'Editing Humanity' explores the CRISPR revolution and the ethical dilemmas that await us - Genetic Literacy Project

It was a bumper year at the annual American Society of Hematology (ASH) meeting, with a slew of new data presented across the board. Here is a round-up of some of the key data presented.

Gilead eyes new first-line indication for CAR T Yescarta

Gileads Kite division presented new phase 2 data for its CAR T therapy Yescarta at ASH 2020 in relapsed or refractory high-risk large B-cell lymphoma (LBCL), a potential new indication.

In the phase 2 ZUMA-12 study, after a single infusion of Yescarta (axicabtagene ciloleucel), 85% of LBCL patients achieved a response, while 74% of patients achieved a complete response.

After a median follow-up of 9.5 months, the median progression-free survival, median overall survival and median duration of response were not yet reached.

Yescarta has already presented four-year survival data in patients with third-line refractory LBCL and we are now excited for what these ZUMA-12 results signal for its potential in earlier lines of treatment, said Ken Takeshita, global head of clinical development, Kite.

Yescarta was first approved by the US Food and Drug Administration (FDA) for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy.

Eli Lilly and Loxos BTK inhibitor shows blood cancer promise

Eli Lillys Loxo Oncology revealed some promising early-stage results for its Brutons tyrosine kinase (BTK) inhibitor LOXO-305 in patients with chronic lymphocytic leukaemia (CLL) and small lymphocytic lymphoma (SLL).

LOXO-305, an oral BTK inhibitor, is designed to address acquired resistance to currently available BTK inhibitors, such as J&Js Imbruvica.

In the phase 1/2 study of the BTK inhibitor, out of 139 CLL/SLL patients who were efficacy-evaluable, 88 responded to treatment, with 19 partial responses with ongoing lymphocytosis, 45 with stable disease and one with progressive disease.

In a subgroup of patients previously treated with a BTK inhibitor, the overall response rate was 62%, which increased to 84% for those with ten months or more follow-up.

It is important to note that the patients included in this study were heavily pre-treated, with all CLL/SLL patients having received a median of three prior lines of therapy.

Vertex/CRISPR Therapeutics make headway in sickle cell and beta thalassaemia

Also presenting data at ASH were Vertex and CRISPR Therapeutics, whose gene-editing therapy CTX001 scored some promising results in transfusion-dependent beta thalassaemia (TDT) and sickle cell disease (SCD) patients.

The CRISPR/Cas9-based gene therapy was investigated in a total of ten patients, seven of which had TDT and three with SCD.

All TDT patients treated with CTX001 were transfusion independent at last follow-up and all SCD patients were free of vaso-occlusive crises (VOCs), the companies announced at the meeting.

These are the first published results from CRISPR/Cas9 therapy in people with a genetic disease and represent an important milestone in medicine and for our collaboration with CRISPR Therapeutics, said Reshma Kewalramani, chief executive officer and president, Vertex.

Most importantly, this data represents a critical step in our effort to bring transformative and potentially curative therapies to patients, he added.

bluebird bios beta thalassaemia gene therapy Zynteglo scores promising long-term data

bluebird bio also posted long-term data for its own beta thalassaemia gene therapy Zynteglo (betibeglogene autotemcel) at ASH 2020.

The new data reflects up to six years of safety and efficacy data for the gene therapy in patients with TDT.

Among 32 patients enrolled in the 13-year, long-term LTF-303 study, 64% of patients treated in the phase 1/2 portion and 90% in the phase 2 portion achieved transfusion independence (TI).

In the LTF-303 study, all patients who achieved TI remained free from transfusions, with the median duration of ongoing TI coming in at 39.4 months.

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ASH 2020: Gilead eyes new Yescarta indication, Vertex/CRISPR make headway in sickle cell and more - PMLiVE

CiBER-seq dissects genetic networks

Cells integrate environmental signals and internal states to dynamically control gene expression. Muller et al. developed a technique to dissect this cellular logic by linking targeted, genome-wide genetic perturbations with a deep-sequencing readout that quantitatively measured the expression phenotype induced by each perturbation. The method, dubbed CiBER-seq, was able to recapitulate known regulatory pathways linking protein synthesis with nutrient availability in budding yeast cells. Unexpectedly, the authors found that the cellular logic also appears to consider protein production machinery in this decision. By uncovering additional facets of this deeply conserved pathway, the findings demonstrate the utility of comprehensive and quantitative CiBER-seq profiling in mapping the gene networks underlying cellular decisions.

Science, this issue p. eabb9662

Systematically profiling the effects of genetic perturbations is a powerful approach that has revealed the molecular basis for a wide range of biological phenomena. The simple, programmable DNA recognition of CRISPR-Cas9 enables genome-wide genetic analysis in human cells and many other systems. Cas9 is guided by a short RNA to a complementary sequence in the genome, where it can introduce mutations or alter gene expression. Pooled libraries of guide RNAs (gRNAs) that individually target each gene in the genome allow us to introduce genetic perturbations systematically into a population of cells. A key challenge is measuring the phenotypic effects caused by individual guides in these pooled libraries and linking these phenotypes back to the associated gRNA, thereby finding the gene that is responsible.

Molecular phenotypes such as gene expression changes provide a clear and sensitive measure for many cellular processes. We sought a general approach to profile how the expression of a particular gene of interest changed when other genes were perturbed. We began with a library of gRNAs, each disrupting one gene, and linked these guides with an expression reporter containing a guide-specific nucleotide barcode. gRNAs that alter reporter expression will change the abundance of the expressed RNA barcode specifically associated with that guide. Deep sequencing of these expressed barcodes quantifies each of these guide-specific reporter expression effects individually within a pooled, complex population. We have implemented this strategy by combining CRISPR interference (CRISPRi) with barcoded expression reporter sequencing (CiBER-seq).

We used CiBER-seq to profile the responses of several yeast promoters tied to a range of biological functions. Each promoter yielded a distinct pattern of responses that could be understood in terms of its known function and regulation. For example, we rediscover the control of MET6 expression by regulatory ubiquitylation and connect the bud scar protein Cwp1 to other genes required for budding and cytokinesis. Our analysis of the HIS4 promoter, a well-characterized target of the integrated stress response, yielded a range of genetic perturbations that activate this pathway by causing the accumulation of uncharged transfer RNAs (tRNAs). We also uncovered a notable role for tRNA depletion in this response, as impaired tRNA biogenesis activated HIS4 expression through a distinct pathway. In order to understand this regulation, we carried out genetic interaction analysis and looked for quantitative deviations in CiBER-seq profiles caused by the introduction of a second genetic perturbation. We also developed an indirect CiBER-seq approach to measure translational and posttranslational regulation, which both play roles in the signaling pathways upstream of HIS4.

CiBER-seq produces comprehensive phenotypic profiles that offer insights into gene function and regulation. These high-throughput and quantitative phenotypic measurements are also well suited for the systematic measurement of genetic interactions, which contain rich information about the operation of biological processes. This approach can be applied to study a wide range of transcriptional, translational, and posttranslational regulatory responses, and it has the potential to shed light on many areas of biology.

CRISPR-Cas9 gRNA cassettes are linked with transcriptional reporters containing specific barcodes. The RNA-to-DNA ratio for each barcode, measured by deep sequencing, reveals the reporter expression phenotype induced by each gRNA.

To realize the promise of CRISPR-Cas9based genetics, approaches are needed to quantify a specific, molecular phenotype across genome-wide libraries of genetic perturbations. We addressed this challenge by profiling transcriptional, translational, and posttranslational reporters using CRISPR interference (CRISPRi) with barcoded expression reporter sequencing (CiBER-seq). Our barcoding approach allowed us to connect an entire library of guides to their individual phenotypic consequences using pooled sequencing. CiBER-seq profiling fully recapitulated the integrated stress response (ISR) pathway in yeast. Genetic perturbations causing uncharged transfer RNA (tRNA) accumulation activated ISR reporter transcription. Notably, tRNA insufficiency also activated the reporter, independent of the uncharged tRNA sensor. By uncovering alternate triggers for ISR activation, we illustrate how precise, comprehensive CiBER-seq profiling provides a powerful and broadly applicable tool for dissecting genetic networks.

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CiBER-seq dissects genetic networks by quantitative CRISPRi profiling of expression phenotypes - Science Magazine

While breast cancer is usually considered a disease that only affects women, anyone with breast cells and tissue can be diagnosed with breast cancer, males included. Even so, male breast cancer is rare.

"This isn't a disease that's 'just for women.' Patients often don't realize this which can, unfortunately, lead to delays in diagnosis," says Abisola Olulade, MD, a family medicine physician at Sharp Rees-Stealy Downtown in San Diego, California. "In the United States, male breast cancer represents between 0.5% and 1.0% of all breast cancers diagnosed each year. This means that about one out of every 100 breast cancers diagnosed in the United States is found in a man."

In this article we'll discuss the most common forms of male breast cancers, its symptoms, and potential risk factors.

Male breast cancer occurs when malignant cells form in the tissues of the breast. Common forms of male breast cancer include:

Unfortunately, doctors are still unsure of the exact causes of breast cancer, though genetics and increased levels of estrogen may be risk factors.

In particular, increased estrogen is associated with conditions like liver disease, obesity, and certain hormone therapies like anti-androgen therapy, says Monisha Bhanote, MD, a triple board-certified physician and cytopathology specialist in Jacksonville Beach, FL. where she runs her own private practice.

"Hereditary causes may include BRCA2 mutation carriers, PTEN mutation (Cowden syndrome), and CHEK2 mutation carriers," says Bhanote

BRCA1 and BRCA2 (BReast CAncer genes 1 and 2) are genes that are related to breast cancer risk. While everyone has both BRCA1 and BRCA2 genes, some people inherit a gene mutation in one or both of these genes. This mutation increases the risk of developing breast cancer.

"Men with BRCA mutations are recommended to have annual screening," says Bhanote. "Examination for any masses, nipple discharge, or skin dimpling/puckering on a daily basis will

be helpful."

Additional factors that may increase a person's risk are medications such as antidepressants, marijuana, and radiation from cancer treatment.

Male breast cancer spreads in the same way that female breast cancer spreads, through the lymphatics and blood vessels. An estimated 520 men will die from breast cancer this year.

Signs and symptoms of male breast cancer can include:

While finding a lump on one or both of your breasts can be alarming, finding a lump doesn't necessarily mean that you have breast cancer. It could be gynecomastia or enlarged breast tissue.

Though gynecomastia has many causes, liver disease and drugs or medications that contain high doses of testosterone account for about 25% of gynecomastia cases, according to Harvard Men's Health Watch.

Gynecomastia is often benign and resolves on its own. That being said, it's still a good idea to see your doctor if you notice any lumps or changes, especially if you're experiencing pain or discomfort.

Because men are less likely to receive a mammogram, it's important to learn how to perform a male self-breast examination. As a patient, it's imperative to become familiar with your body so you can advocate for yourself and notice when changes occur. Here's a quick step-by-step guide on how to self-check your breasts at home:

You can also check your breasts while lying down. To do this, set a pillow underneath your right shoulder and bend your right arm over your head. Use the fingertips of your left hand to check all areas of your breast and armpit. Once you're finished, switch the pillow to your left shoulder and repeat this process.

If you're uncomfortable doing a self-breast examination, or you fear that you're not doing it correctly, ask your physician to perform a clinical breast exam for you. Depending on how the examination goes, your physician may order a mammogram, an ultrasound, or an MRI.

If you're diagnosed with male breast cancer, your treatment plan will depend on how far the cancer has spread. Practicing monthly self-breast exams, in addition to receiving a breast examination by your physician, could improve your chances of detecting breast abnormalities early. Early detection is the key to successful treatment.

Possible treatments for male breast cancer include:

Although researchers can't pinpoint the causes of breast cancer in men, they have found a few risk factors.

Risk factors that may lead to developing male breast cancer include:

"Unfortunately, there isn't anything you can do to prevent male breast cancer," says Nicholas Jones, MD, FACS. "However, you can lower your risks by being active, and limiting your alcohol consumption."

In addition, avoiding hormonal supplements, such as sexual performance enhancement supplements, may help to prevent male breast cancer. According to a 2019 study, the use of hormonal male enhancement supplements can lead to the higher levels of androgens, which may cause the growth of tumors.

Though the likelihood of developing male breast cancer is low, it's important to pay attention to your body. If you notice any changes in your skin color, new rashes, lumps, or bumps, you should seek medical help right away.

"There isn't a great screening tool for men," Jones adds. "The best action is to know your body, live a healthy lifestyle and if you notice anything out of the norm, visit your doctor. Specifically for male breast cancer, any change involving the nipple may be a sign that there is an underlying cancer."

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Yes, men can get breast cancer here are the symptoms to watch for - Insider - INSIDER

The COVID-19 lockdown had brought some cheer for Andheri-based Kamat couple as they welcomed their first child - Teera - on August 14. But soon things went awry when their daughter was diagnosed with a rare genetic disorder - Spinal Muscular Atrophy (SMA). Three-month-old Teera's parents - Mihir, 35, and Priyanka Kamat, 33, have been doing everything possible for their daughter's treatment but the biggest challenge for them is to raise '16 crore for a single shot of gene therapy, the only way she can survive.

Teera with her parents Mihir and Priyanka Kamat

Teera has been diagnosed with SMA Type 1, which is said to be a more serious form of the disorder. Speaking to mid-day, Mihir, who works for a software company, said, "Teera was born a happy and cheerful baby. As weeks went by, we noticed she was having heavy breathing patterns and it would also take a long time to feed her. Very soon, she developed multiple issues like severe dehydration, heavy breathing, choking on feeds etc, and we finally found a doctor in our area who recommended we start bottle feeding."

"On the day of her vaccination (September 30), her doctor noticed that she was choking on her first drops of vaccine. She was also not being able to resist with her hands and the right leg wasn't moving," Mihir recalled.

"The doctor referred us to a paediatric neurologist who mentioned that Teera might have hypotonia, slow reflexes, paradoxical respiration and a weak cry. Following her EMG and genetic test, we got to know that she is suffering from SMA Type 1. The next shock was the cost of the drug, which needs to be imported from the United States. Teera needs it before she turns six months old, the golden period to save her. We started doing a bit of research on the disorder and found out about some foreign nationals who have written positively about the treatment and how they could 'crowd fund' such a large sum by registering on a similar platform," added Mihir.

"We registered our daughter's details with crowd funding platform Impact Guru and in less than a month 8,187 donors contributed nearly '2.36 crore, said the couple. Mihir further said, "Our consulting paediatric neurologist Dr Neelu Desai has already registered Teera for the Switzerland HQ Novartis pharma company global lottery, where selected registered patients get the drug free of cost." "Just three weeks ago Teera had to be rushed to Holy Spirit hospital in Andheri East, as she got choked during her feed and turned pale. She had to be kept at the hospital for the next couple of days," her father said.

SMA is a progressive, rare genetic disease that is caused by missing genes or those that don't work properly, which might be survival motor neuron 1 (SMN1). This gene is basically the blueprint responsible for making a protein required for the survival of the spinal nerve cells. Without this SMN gene the cells in the body start to die and motor neuron cells become weaker thus causing muscle weakness in every part of the body below the neck.

Piyush Jain, co-founder and CEO of ImpactGuru.com, said, "In one month, 8,187 donors have contributed '2.36 crore. This is Impact Guru's highest single fundraiser. Among the donors is a noted Bollywood actor, who donated '5 lakh We are currently working towards ensuring the target is met by all means."

Dr Neelu Desai

Dr Desai said, "Teera is one of the youngest patients I'm treating at the moment. If she doesn't get the special gene therapy, then she might survive for about two years. Until last five years, there was no treatment for SMA, but now we have gene therapy, for which Teera has been registered."

She further said, "This is a rarest of rare disorder (1 in 5,000 infants suffer from it world over). In case a baby does not develop proper motor skills or his/her body feels very soft then parents should consult paediatricians. Parents who have a child with SMA, have 25 per cent chances of the same disorder developing in the subsequent pregnancies, which can be confirmed in the first few months by doing a genetic test."

Link to the fundraiser - https://www.impactguru.com/fundraiser/donate-to-teera

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Mumbai: Baby with genetic disorder needs Rs 16 crore therapy to live - Mid-day

MedicalResearch.com Interview with:

Steven Pipe, MDProfessor of Pediatrics and PathologyLaurence A. Boxer Research Professor of Pediatrics and Communicable DiseasesPediatric Medical Director, Hemophilia and Coagulation Disorders ProgramDirector, Special Coagulation LaboratoryUniversity of Michigan

MedicalResearch.com: What is the background for this study?

Response: Hemophilia B is an inherited bleeding disorder where patients are missing clotting factor IX (9), a critical blood clotting protein.Patients with a severe deficiency are at risk for traumatic and spontaneous bleeds primarily into joints.Repeated bleeding into joints causes more than acute pain and swelling but also leads to inflammatory and degenerative changes in joints that eventually leads to severe debilitating arthritis that can be crippling.To try to prevent this, patients as young as infants are placed on regular infusions of clotting factor IX concentrates.The relatively short half-life of factor IX means patients must infuse on average once to twice a week.These can only be delivered intravenously parents and then patients themselves have to learn this.Prophylaxis must be continued lifelong to try to prevent bleeding events and protect joint health over the lifespan.This is a tremendous burden on the patient and their caregivers.

Even with regular prophylaxis, joint bleeds may still occur and arthropathy may still ensue.This is because the blood levels often reach critically low levels prior to the next infusion.Gene therapy aims to deliver a functional copy of the factor IX gene such that the patients own liver will make a continuous supply of factor IX that is delivered to the bloodstream.At steady state with levels close to or within the normal range, patients would no longer be subject to bleeding events and would not require prophylaxis any longer.We hope that such a one-time treatment would produced durable, functionally curative levels of factor IX.

MedicalResearch.com: What are the main findings?

Response: The 3 key takeaways are:

MedicalResearch.com: What should readers take away from your report?

Response: I think it is best to characterize this as a treatment that liberates patients with hemophilia from the burden of repeated prophylactic infusions of clotting factor to protect them from recurrent bleeding events.Gene therapy offers a chance to have steady state levels of factor in the blood that would eliminate risk for spontaneous and even traumatic bleeding events.

MedicalResearch.com: What recommendations do you have for future research as a result of this study?

Response: These are very encouraging results maximizes eligibility for patients by being able to treat in the presence of pre-existing neutralizing antibodies.Results are in a range that allow for cessation of prophylaxis and continued bleed protection.Results appear to be durable through the follow up period to date.The 26 week endpoint was a co-primary endpoint.The other endpoints are factor IX activity at 52 weeks and annualized bleeding rate over the 52 weeks post-dosing. Those endpoints should be analyzed in 2021 and decisions could then be made on submission for regulatory review.

MedicalResearch.com: Is there anything else you would like to add? Any disclosures?

Response: This could be potentially clinical practice changing, if approved. This looks to be a transformative therapy for patients and could be an option for patients across the adult lifespan we treated patients from age 19 through 75. Our patient population have been looking forward for a long time for a one-time therapy like this that would provide lasting protection from bleeds.

I have served as a paid consultant to uniQure and chair the Steering Committee for the global clinical trial program

Citation: ASH2020 Oral Abstract

First-in-Human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A BAY 2599023 has Broad Patient Eligibility and Stable and Sustained Long-Term Expression of FVIII

Steven W. Pipe, MD1, Francesca Ferrante, MD2*, Muriel Reis3*, Sara Wiegmann4*, Claudia Lange5*, Manuela Braun5*and Lisa A Michaels6*https://ash.confex.com/ash/2020/webprogram/Paper139803.html

The information on MedicalResearch.com is provided for educational purposes only, and is in no way intended to diagnose, cure, or treat any medical or other condition. Always seek the advice of your physician or other qualified health and ask your doctor any questions you may have regarding a medical condition. In addition to all other limitations and disclaimers in this agreement, service provider and its third party providers disclaim any liability or loss in connection with the content provided on this website.

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Gene Therapy Liberates Hemophilia B Patients from Requiring Regular Infusions of Clotting Factor - MedicalResearch.com

This article was originally published here

Lab Invest. 2020 Dec 9. doi: 10.1038/s41374-020-00520-2. Online ahead of print.

ABSTRACT

Disorders involving injury to tissue stem cells that ensure normal tissue homeostasis and repair have potential to show unusually devastating clinical consequences. Acute graft-versus-host disease (aGVHD) is one condition where relatively few cytotoxic immune cells target skin stem cells to produce significant morbidity and mortality. By analogy, SARS-CoV-2 is a vector that initially homes to pulmonary stem cells that preferentially express the ACE2 receptor, thus potentially incurring similarly robust pathological consequences. In older individuals, stem cell number and/or function become depleted due to pathways independent of disease-related injury to these subpopulations. Accordingly, pathologic targeting of stem cells in conditions like aGVHD and COVID-19 infection where these cells are already deficient due to the aging process may have dire consequences in elderly individuals. A hypothesis is herein advanced that, as with aGVHD, lung stem cell targeting is a potential co-factor in explaining age-related severity of COVID-19 infection.

PMID:33299126 | DOI:10.1038/s41374-020-00520-2

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COVID-19 and graft-versus-host disease: a tale of two diseases (and why age matters) - DocWire News

TipRanks

Investing is all about profits, and part of generating profits is knowing when to start the game. The old adage says to buy low and sell high, and while its tempting just to discount cliches like that, theyve passed into common currency because they embody a fundamental truth. Buying low is always a good start in building a portfolio.The trick, however, is recognizing the right stocks to buy low. Prices fall for a reason, and sometimes that reason is fundamental unsoundness. Fortunately, Wall Streets analysts are busy separating the wheat from the chaff among the markets low-priced stocks, and some top stock experts have tagged several equities for big gains. These stocks are trading low now but the reasons are not necessarily bad for investors.Weve used the TipRanks database to pull up the data and reviews on two stocks that are priced low now, but may be primed for gains. Theyve been getting positive reviews, and despite their share depreciation, they hold Buy ratings and show upwards of 60% upside potential.Digital Media Solutions (DMS)We will start with Digital Media Solutions, an adtech company which connects online advertisers with customers through performance-based branding and marketplace solutions. DMS boasts a powerful consumer intelligence database, which it uses to fine-tune customer acquisition campaigns while offering advertisers accountability for the project budget.DMS went public in July of this year, via a merger with a special purpose acquisition company, Leo Holdings. The combination took the DMS name for the ticker, and initiated trading at $10 per share. The stock has been volatile since, and is currently down 27% since it started trading.Digital advertising is a huge and growing sector, worth $100 billion in 2019 and expected to reach $130 billion by the end of next year. DMS has a solid piece of that cash cow, and the Q3 numbers demonstrate that. Quarterly revenue hit a company record, of $82.8 million, which was up 10% sequentially and 44% year-over-year. Of that total revenue, the company saw a gross profit of $25.1 million, for a 30% gross margin. All in all, DMSs first quarter as a publicly traded company showed strong results.Covering the stock for Canaccord is analyst Maria Ripps, who is rated 5 stars by TipRanks, and stands in the top 1% out of more than 7,100 stock analysts. The company saw meaningful volume growth from both new and existing clients, with particular strength from its auto insurance business along with the eCommerce, education, and non-profit verticals We continue to think investors will gradually come to appreciate DMS similarities with other leading digital marketing peers that trade at more premium valuations, and expect multiple expansion over time as the story becomes better understood, Ripps noted.To this end, Ripps rates DMS stock a Buy, and her $15 price target suggests an upside of 106% from the current share price of $7.20. (To watch Ripps track record, click here)Overall, DMS Moderate Buy consensus rating is based on 2 recent reviews, both positive. The stock has an average price target of $14, which indicates a 92% upside potential. (See DMS stock analysis on TipRanks)ViaSat, Inc. (VSAT)From digital advertising we move on to digital networking. ViaSat provides customers with high-speed broadband access through a secure satellite network system. The company serves both military and commercial markets, meeting the growing need for secure communications links.The anti-coronavirus shutdown policies have particularly hard on ViaSat. This may sound counterintuitive, as online networking has been busier than ever, but a large segment of ViaSats business comes from the airlines, and with air travel first grounded and still facing depressed travel volumes, ViaSats shares have yet to recover from their February/March swoon.On a positive note and one that is indicative of the essential nature of secure satellite communications in todays networked economy ViaSat reported $577 million in Q3 contract awards, representing a 29% yoy gain. For the year to date, the company has seen awards totaling $1.9 billion, which is up 5% from this time last year. The third quarter (the companys fiscal Q2) revenues and earnings were somewhat mixed, reflecting both the increase in contract awards and the decline in airline business. Revenues were $554 million, down 6% yoy, but up almost 4% sequentially. EPS was 3 cents per share, beating the predicted 5 cent loss by a wide margin.JPMorgan analyst Philip Cusick writes of ViaSat: [We] believe long-term growth levers remain intact highlighted by record segment backlog of $1.1b We view ViaSat as a satellite innovation leader and believe the companys future ViaSat-3 fleet will accelerate growth in satellite services over the coming years. At the same time, we see a long-term government systems tailwind driven by the companys radio portfolio, mobile broadband, and SATCOM.In line with his bullish comments, Cusick rates VSAT shares an Overweight (i.e. Buy), and his $60 price target implies ~72% upside on the one-year time horizon. (To watch Cusicks track record, click here)Overall, the stock has 5 recent reviews, including 3 Buys and 2 Holds. Shares are priced at $34.14, and the average price target of $55 suggests a 61% upside potential from that level. (See VSAT stock analysis on TipRanks)To find good ideas for stocks trading at attractive valuations, visit TipRanks Best Stocks to Buy, a newly launched tool that unites all of TipRanks equity insights.Disclaimer: The opinions expressed in this article are solely those of the featured analysts. The content is intended to be used for informational purposes only. It is very important to do your own analysis before making any investment.

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Avenoir Cosmetics Launches Cell Repair Serum - Clinical Strength to Enhance Skin Tone & Minimize Appearance of Sunspots, Blemishes, Discoloration,...

Even while legalization and regulations are in flux throughout the Americas region and around the globe, cannabis beauty is making tremendous progress.

The cannabis market is emerging very, very rapidly around the world, said Shane MacGuill, Senior Head of Nicotine and Cannabis at Euromonitor during a webinar last month reviewing the global cannabis industry across market sectors and showcasing the launch of the market research companys newest Passport Research System focus: cannabis. And he added,the market is currently heavily concentrated in US Canada.

This year2020was the year that both Amway and Avon launched CBD skin care. Avon made the announcement first. In April, as Cosmetics Design reported, the social-selling beauty maker announced plans for a new vegan skin care line that would include a CBD oil. And today, Avon has 3 CBD products in its portfolio: Green Goddess Facial Oil, Veilment CBD Soothing & Nourishing Body Cream, and Veilment CBD Nourishing Body Cream, all of which contain 100mg of CBD. Though as the online product descriptions note, our collection does NOT contain THC. The only high youll get is knowing your skin feels cool, calm and collected.

Amway, as Cosmetics Design reported, announced the launch of its new Artistry Skin Studio product collection in September 2020. And that product line now includes Artistry Studio Zen Daze Ahead Facial Oil +300 mg CBD.

Colgate also got involved in the CBD Beauty market this year. In January the company announced an acquisition deal to buy Hello Products and the following month, Hello launched its CBD product collection, as Cosmetics Design reported. Hello is best known for its oral care products but the brand also makes CBD lip balm, for instance.

Ayuna, a luxury skin care brand out of Spain, just launched the latest limited-edition product in its sought-after Terra collection at the start of November: Terra Bella.

There are no cannabinoids, no THC, no CBD in this new cream. But the Terra Bella face cream is formulated with a remarkable extract derived from cultured Cannabis Sativa stem cells, as Isabel Ramos, Chief Scientific Officer at Ayuna, tells Cosmetics Design.

And the ingredient does something truly extraordinary: it instigates communication between the skin microbiome and the brain, explains Ramos. This topical ingredient is, she says, the first known instance of a skin care input helping and demonstrating that microbiota are acting on [or affecting] how we feel.

As the Ayuna example illustrates, theres a lot more to cannabis beauty than the ever-enchanting CBD. In fact, the entire cannabis plant and a full range of cannabinoid molecules are shaping the future of this buzz-worthy cosmetics and personal care category.

In October, Jennifer Grant, a biomedical engineer turned beauty entrepreneur launched a clean skin care brand called empyri that relies on upcycled cannabis root at its hero ingredient. And not long before that news made headlines here on Cosmetics Design, the biotechnology company behind brands like Bissance and Pipette, announced having successfully scaled production of biotech CBG (one of many cannabinoids naturally occurring in cannabis) for use in skin care product formulations. So while CBD beauty is here to stay, theres much more to Cannabis Sativa and to cannabis beauty than this one molecule. Ready to learn more? Revisit all the top CBD beauty news from 2019 here on CosmeticsDesign.com.

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CBD: the beauty ingredient trend that can't be stopped - CosmeticsDesign.com USA

In the second year of Endpoints Newsbudding tradition of highlighting women blazing trails in biopharma R&D, weve seen a number of firsts.

For the first time, the biggest story in R&D is also top of mind for a world anxious to end the most devastating health crisis in decades. With its sweeping effects, the Covid-19 pandemic is turning the daily routines for many working women upside down, taking a toll on not just their physical and mental health, but also their career prospects. At the same time, the biotech industry is doing some serious soul searching with a new scorecard and plan for diversity and inclusion.

It is more important than ever to shine light on the growing number of women who have scaled the heights of drug discovery and development even though the odds are stacked against them, breaking open paths in labs and C-suites that can be followed by future generations, some of whom they are also actively nurturing and mentoring.

We set out this year with a dual goal: to celebrate women at the forefront of subduing Covid-19 either with diagnostics, vaccines or treatments and to honor those working day in and day out to address other equally pressing medical needs.

Profiles are, by definition, snapshots. Some of the women recognized in our 2019 special report have since become household names: Jennifer Doudna became a Nobel laureate for her pioneering work in CRISPR gene editing and zlem Treci helped create the worlds first vaccine proven effective against Covid-19. Even in the short period since we interviewed this years honorees, weve seen them mark new milestones, from taking a biotech public to scoring historic clinical data and regulatory authorizations.

We hope our profiles capture a unique moment in history as these highly accomplished figures take us with them down memory lane to illustrate what brought them to this moment and how they are helping other women do the same if not even more.

We hosted an online live event to introduce this years top women in biopharma R&D, followed by a panel discussion on what it will take to truly achieve gender diversity in the industry. You can watch the full event here.

Carolyn Bertozzi wasnt exactly looking to start a company when she tweeted about a preprint her group posted on ChemRxiv last November. The paper described LYTACs molecules that can tag extracellular proteins for degradation.

She was heading out to the American Chemical Society Conference in Orlando to talk about the project, led by a postdoc in her Stanford lab named Steve Banik.

(Steve) was about to go on the academic job market, so its a good time for me to go and sort of talk about his work publicly and drop his name and get him some name recognition, she recalled.

Within 24 hours, though, she got a dozen phone calls from venture capital groups who picked up on the preprint. There was a proliferation of startups utilizing and improving on PROTACs a targeted degradation technology born out of Craig Crews academic work at Yale but that magic was limited to intracellular proteins. Bertozzi was a self-described jealous fan of Crews work because glycoproteins, the subject of her own research, reside exclusively on cell surfaces; it turned out VCs had also been wishing there were ways to target secreted and membrane proteins.

In the end, she found Versant Ventures to be the best fit for the science. Now, Lycia Therapeutics, which unveiled a $50 million Series A just a few months ago, has been taking off like a rocket ship, she said.

The enthusiasm from investors might be expected for a scientist and entrepreneur whose last biotech creation, Palleon Pharmaceuticals, recently reeled in $100 million in fresh financing. Redwood, the one that came before, recently reported Phase I data as a subsidiary of the CRO giant Catalent. Then there are the handful of diagnostics players that she created with researchers under her wing.

But for Bertozzi, its all about timing.

Her first experience with entrepreneurship a biotech startup she co-founded in the late 1990s after completing a postdoc with Steve Rosen at UCSF actually ended in failure. Thios Pharmaceuticals, as it was known, had zeroed in on a target for sickle cell acute vaso-occlusive crisis and in-licensed a molecule that worked in a similar way as two therapies approved by the FDA late last year.

They raised $10 million for it, a respectable amount for a Series A at that time, but it wasnt enough to sustain the 30-person company when investors decided not to continue funding it. The drug was shelved as Thios closed up shop.

It was always very heartbreaking to us that we couldnt have brought that to those patients 15 years earlier, Bertozzi said. But thats how it goes in this business, and every company has a story like that.

In her day job as an academic, Bertozzi is known for spearheading a glycorevolution: developing chemical tools and technologies to elucidate the roles that complex sugar structures play in biological systems. Glycans, as she learned during her doctorate, are an order of magnitude more technically complex than polypeptides and oligonucleotides because of their structural diversity. But papers dating as far back as the 1950s have suggested they do play a role in diseases such as cancer, and spectacular breakthroughs in chemical synthesis the challenge that drew her to the space initially have helped scientists make inroads in their study.

Her biggest breakthrough which won her a MacArthur genius grant at age 33 came in what she coined bioorthogonal chemistry, a way for chemical reactions to take place within biological systems.

Both the tools and her deep knowledge of glycobiology provided the foundation for the subsequent technologies her group would later develop. Site-specific chemical modification of recombinant proteins promised to make better antibody-drug conjugates (ADC) than the very sloppy molecules that characterized the first generation of ADCs; digging deeper into new findings on how cancer cells sprouts sugars on their surfaces to evade the immune system led them to bispecific antibody-enzyme constructs that target the siglec-sialic acid pathway, billed as the next big I/O checkpoint; and utilization of lysosomal trafficking shuttles one of the first things you learn when you study glycobiology gave birth to chimeras that send unwanted extracellular proteins to the lysosome for targeted degradation.

More so than any particular technology, though, she considers the young scientists who trained with her and are now standing on their own feet her most important achievement.

Carolyns genius is not only in the research she leads, but also in those she recruits, Mireille Kamariza, a former PhD student, said. Now a junior fellow at Harvard University, she co-founded with Bertozzi a public benefit corporation to advance a point-of-care diagnostic device for tuberculosis.

She has an uncanny ability to bring out the best in people, Kamariza added. She leads by example and gives unyielding support to her students interests and pursuits.

Now 54, Bertozzi recently found herself reflecting on her career after the death of late Supreme Court Justice Ruth Bader Ginsburg. Harvard, after all, had only integrated with Radcliffe a few years before shed start undergraduate studies there.

I consider myself to be in sort of the first generation where you see a critical mass of women who made it through the academic pipeline and into industry, she said. There arent many of us, but you can count us. Were visible.

Having seen every shade of gender discrimination and bias (show me a woman who hasnt), Bertozzi is leveraging her influence as the co-director of Stanford ChEM-H to bring in more voices from outside the usual suspects reaching out and forging relationships with public universities and institutions serving underrepresented communities to find talent that is hiding in plain sight.

In previous interviews, shes noted that part of what got her hooked in those first organic chemistry classes was how molecules have personalities like people. So I couldnt help but ask: What molecule would she be?

The answer was surprisingly simple. She aspires to be a monoclonal antibody.

They multitask all over the place, she said. In fact, all of the next-generation biologics out of her lab have had antibodies as their backbones.

I have gotten so much mileage out of antibodies, she said. And so I would like to think maybe my students could get the mileage out of me that same way. That would be my aspiration.

Thats not all: Oh, and they are glycosylated. Amber Tong

Kathy Bowdish was sitting down at a San Diego restaurant after her first pitch meeting in 1997 when one of the potential investors turned and asked her a question: Does your husband know youre doing this?

Taken aback, Bowdish, 39 at the time, said yes, and that part of this was his idea. Of the 12 angel investors at the table that day, the concerned citizen was the only one who didnt decide to back her first startup. And he was the only one who didnt get a cut of the profits three years later, when Bowdish sold the startup to Alexion for 10 times their investment.

When the press release for the buyout hit the wire, her phone rang. It was the lone investor.

If you do this again, I want to be on speed dial, the investor told her. Bowdish looks back on that moment with victory-tinged humor: Clearly, his question had been troubling him.

The seed investor had made two mistakes. One shouldve been obvious at the time: plain misogyny. But the other would only become fully apparent over the ensuing 23 years as the young scientist-executive enmeshed herself in a series of top biotechs and eventually was handpicked by Elias Zerhouni to lead a unique and under her direction fruitful venture in Sanofis upper ranks.

He had underestimated Kathy Bowdish.

Bowdish would prove over the years a rare triple-threat in biotech: a brilliant scientist who could handle the business side but also had the poise and emotional IQ to massage needed personal relationships and wheedle folks who could get things done. After nearly two decades in biotech, she used those skills to build powerful new companies at Sanofi. And when the winds changed there, she pivoted mid-career to what she first learned to do in San Diego: build a company with cutting-edge science from the ground up.

Kathy impressed me because she had been CSO, she was a very good scientist, Zerhouni told me. But she had this very special natural calm A very thoughtful calm, a resilience. She had this analytical mind.

Bowdish is now working from her home in Cambridge, trying to get a three-person,RNA-based startup off the ground with $5 million in seed cash. Its a significant departure from her Sanofi perch, but its a position she knows and a place shes thrived before.

The daughter of an engineer and an artist, Bowdish was seduced by the cerebral yet creative challenge that scientific problems posed. After studying biology at William & Mary, she moved to the West Coast, where she was quickly recruited for a position at Scripps Research and worked with Richard Lerner on the then-pioneering field of catalytic antibodies.

She was clever and ambitious, devising new ways of sequencing antibodies in an era before you could simply plug them into RNA-Seq. She left to get a PhD at Columbia University, and picked a yeast lab because yeast multiplies like microbial rabbits, which meant you could do a lot of genetics work in a single program.

A postdoc followed, but she soon grew impatient. These were still early days for antibodies. She pitched Lerner on the idea of building a company around the work they did on combinatorial antibody libraries basically discovery engines in a test tube.

His immediate question to me was can you live without a salary for 6 months? Cause thats what its going to take in order to raise the money. And I said yes, she said. I was pretty naive maybe the naivet was a good thing.

The investors (but one) came on board, Bowdish finagled her way into some cheap equipment, hired another scientist a woman and for a year the pair worked at the bench to discover antibodies that would activate, rather than inactivate, receptors. She shifted over to a full CEO role after year one, and after year three Alexion bought the company for $41 million, betting that it could serve as their discovery engine.

She stayed at Alexion until 2007, by which time she was a sought-after name for biotechs with big ideas. Investors recruited her to relaunch Anaphore, a Danish biotech trying to develop antibody-like alternatives that were more selective or more potent. They struggled for years, which worried Bowdish until she realized every company in the space was struggling. It taught her a valuable lesson about what makes for good translational science.

You just cant beat evolution, she said.

Then ARCH and Flagship came calling. They had funded a bold Bob Langer idea for delivering proteins inside cells. Quickly, she realized they had to start testing this in animals to have any idea if it worked. And quickly they learned that it didnt everything went to the liver. So she leveraged their tech into antibody-drug conjugates.

It looked really good in vitro, she said. In vivo, not so much.

By then it was 2013. and Bowdish had been in the league for 16 years, helping run five biotechs on both coasts. She knew the game, but she was curious: about pharma, that behemoth in the background, and about investment, a general-esque perch from which she could ponder and evaluate. At the same time, Zerhouni was conceiving a new initiative at Sanofi: Sunrise, a way of giving biotechs the resources and expertise of pharma while allowing them to flourish on their own. It married the two.

Bowdish got the job, beating out such biotech titans as Michael Gilman. Inside, she learned how pharma worked and she built new companies. She was, Zerhouni said, singlehandedly responsible for MyoKardia, the cardiovascular upstart that Bristol Myers Squibb recently purchased for $13 billion. She found them, he said, designed a deal to get them the best resources and convinced Sanofi leadership to get on board.

Shes able to move the needle from a pretty established sort of approach to an innovative one, Zerhouni said. Shes a change agent.

Then Paul Hudson came in, reshuffled Sanofi, and Bowdish found herself on the outs (Sanofi also pulled out of her MyoKardia deal, a blunder that cost them a cool $1 billion). She went home, relaxed and did what she always does dove back into the literature: papers on immunology, diabetes, biomolecular condensates, whatever fascinated her.

Eventually, a recruiter called. She realized it was time to get moving. She mentioned to Gilman they had stayed in touch after a conference that she was on a hunt, and Gilman put her in touch with Genzyme Ventures vet Alan Waltz, who was looking for a CEO for a company that would develop small molecules that target structures on mRNA in an effort to block multiple oncogenes. She liked the science and the potential impact.

It was back to some of my earlier learnings, in terms of the right constellation of people around the table and the science that was ready for this very directed effort, she said.

So now she works from home, poring over the literature, directing CROs and her team of three, preparing for a fundraising round and building a whole team. Its a smaller company than she had led since she was just out of grad school, ignoring the pretensions of a sexist investor, and she couldnt be happier.

Its actually a lot of fun, she said. Jason Mast

Diana Brainard was studying abroad in France when she realized something just didnt quite feel right.

Intending to become a comparative literature professor while attending Brown, Brainard enrolled at a French university as part of her major and fully immersed herself for the first time in literature and lit theory classes. Brainard had mainly taken a balanced diet of courses stateside everything from math and science to English and the Study Abroad program was a necessary part of her program. She went into it head-on as her literature seminars were her favorite undergrad classes, with passionate professors seemingly putting on a production every class.

But after diving in, Brainard felt her studies had lost their attachments to the real world.

All of a sudden there was this realization of Wow, what Im really being assessed on in these classes, and how my career will be assessed in this field, is coming up with a theory and making that theory sexy, Brainard said. But that theory doesnt have to be true. It just has to be sexy and interesting and I can do all of that, but there is no truth here.

Since then, Brainard has moved past the days of Thomas Manns The Magic Mountain, though she hasnt lost her love of literature. Now, shes a senior executive at Gilead and has been a major part of the team that developed remdesivir, recently approved by the FDA as the first Covid-19 treatment for hospitalized patients.

The path from France to Gilead wasnt a straight line, however. Brainard notes that after Brown, she eventually went to med school expecting to become a physician. But that didnt end up being the right fit either, especially after Brainard fell in love with infectious diseases. She ended up as a researcher at Harvard focused on HIV, content to work off NIH-backed grants for the rest of her career.

Then biopharma came calling.

Leaving academics was really hard because I was happy doing what I was doing, and I hadnt envisioned in the future that I would be going to industry, Brainard said. There werent a lot of people at Harvard who had done that before, and so I felt like I was taking a big risk, I was leaving behind NIH funding, we were leaving Boston where wed been for a long time, and I didnt know if I would wind up regretting all of the things that I was letting go of.

Brainard jumped aboard first at Merck, then joined Gilead in 2010. Shes spent her entire career, in academia and otherwise, working in infectious diseases and immunology, and much of her time at Gilead has centered on hepatitis C treatments. When she was first alerted to remdesivirs potential benefits for a new coronavirus strain out of China almost a year ago, Brainard moved to get it into the lab as quickly and efficiently as possible.

Remdesivir was a team effort, Brainard said, and shes reluctant to take credit for spearheading the drugs pivot to Covid-19. Originally tested in hepatitis C and then Ebola, remdesivir showed early signs of in vitro activity in SARS and MERS. Brainard pushed for Gilead to begin a compassionate use program for the drug, and it received partial emergency authorization in May for severe Covid-19 cases.

That EUA was expanded in August to include moderate cases, and the FDA handed down a full approval in October for patients who have been hospitalized with the disease.

Its been exhilarating to do [this] for Covid because of the acute, unprecedented medical need, and because scientifically its fascinating to have a disease, as an infectious disease expert, to learn about a disease at the same time youre trying to figure out if a therapy for a disease works, Brainard said a few weeks before the approval. Its a really difficult problem, and one that doesnt come around very often.

The drug has received some pushback, as a study backed by the WHO found in mid-October that remdesivir had little to no effect in reducing mortality rates or the need for ventilators.

Though Brainard was originally hesitant to move from academia, shes happy to have found a home at Gilead and not have to deal with what she says are liabilities in that arena. She never really enjoyed the structure of how academic careers are traditionally judged, where the main goal is to work your way up the authorship ranks and get a couple of papers under your belt.

Brainard much prefers the collaborative environment in industry. And at Gilead, she thinks that collaboration helps her be a better role model for younger women rising through the field.

The impact that I have, a lot of it is the drugs, Brainard said, but a lot of it is the people. The people who I work with, a lot of the people who come to Gilead, for example, come right out of training and they make that transition in the same way that I did. Helping them learn how to think in this new environment and thrive, and flourish in a new environment, thats really meaningful to me. Max Gelman

Janice Chen is all about democratizing diagnostics.

The Mammoth Biosciences co-founder has made that her mission over the last three and a half years, working alongside newly-minted Nobel laureate Jennifer Doudna to develop diagnostic tools centered around CRISPRs gene editing techniques. But what might that look like in practice? Chen theorized some potential uses in a TEDx CERN Talk two years ago.

What if you could directly and accurately test for the flu at home? Chen said. What if you receive the prescription and treatment plan without having to step foot into a clinic? And what if the same principle could be applied to other dangerous diseases such as Ebola?

Chen kicked off her scientific career as an undergrad at Johns Hopkins, where she took a course in which students built the yeast genome from scratch. She credits that class with getting her the technical skills needed to apply to research and lab assistant positions, as well as explore how synthetic biology could lead to a swath of applications.

It also served as a jumping-off point for Chens ambitions, where she said she was able to get her hands wet in something with real-world applications rather than reading lines from a textbook. After a yearlong stint as a research technician at Harvard, where her work focused on Ralstonia eutropha bacteria, Chen moved on to her doctorate at UC Berkeley and joined Doudnas lab.

Its there where she first hooked on to CRISPR research.

Myself and colleagues in the lab, including co-founder Lucas Harrington, came across this unexpected finding that some of these Cas proteins were able to detect DNA, Chen told Endpoints News. A lot of it was serendipitous in terms discovering this activity, and being able to demonstrate it on real patient samples I think was really an exciting moment for me in thinking about OK, can we actually take this outside the academic lab and try to do something with it?

Though CRISPR has largely settled in the mainstream consciousness as a way to potentially cure a range of severe diseases along with the occasional headline that scientists are out to create gene-edited embryos in a lab Chen and the Mammoth team are looking at ways these tools can be used for disease detection. Instead of using the scissors normally associated with CRISPR to edit genes, Chen programs the tech to find a defined gene sequence and sends out a signal once its been located.

With the Covid-19 pandemic in full swing, Mammoth has also steered its research toward creating accessible and easy-to-use tests for the detection of SARS-CoV-2. In that instance, the platform is programmed with a guide RNA, and Chen hopes by working on tests that can function outside of the typical lab setting, this can be one of the ways to democratize the technology.

Its been successful because its programmable, Chen said. Theres a whole world of testing thats just starting to be closer to reality that has never quite been able to be done because of the limitations of infrastructure needs, the accuracy to actually be a viable solution. With CRISPR now on the scene, were really excited about the potential to address these point-of-need environments that dont rely on your traditional clinical laboratory.

Chen credits her doctoral advisors from Berkeley, Doudna included, with keeping her focused and transitioning from academia to becoming an entrepreneur at Mammoth. Ultimately, that led Chen to her north star of not just engineering new uses for CRISPR, but being able to create impactful technology in general.

(Doudna) herself is a serial entrepreneur, and so its been really important to have her mentorship, and seeing her as a role model being very successful in both the academic world as well as the startup world, Chen said, a few weeks before Doudna won the Nobel Prize.

As someone whos still in the early stages of her career, Chen recognizes the opportunity she has to make an impact on the women who follow in her footsteps. Shes already been named to Forbes 30 under 30 healthcare list from 2018 for being among the most influential millennials in the sector. By coincidence, all of her major research experiences from Johns Hopkins through Berkeley were led by women principal investigators.

And while Chen says theres not going to be one magic bullet that fixes everything in the industry, the best place to get started is by recruiting talent from all sorts of different backgrounds.

Once you have that talent and have them in the company its really important to ensure that they have the support systems and mentorship internally to help them grow, Chen said. Its important to recognize that there are really great leaders in companies that might not fit the traditional mold, and I think that there are a lot of companies that are starting to figure that out. Max Gelman

In a cramped, windowless lab basement beside Cambridges Blue Room bar, Ann Cheung stuck her head in the freezer and, with a vial of cells in one hand, used the other to phone a world-famous professor across town and a tech entrepreneur in San Francisco.

Cheung was 34. She had the academic pedigree: Brown, MIT, a postdoc at CalTech where she built nanoparticles and studied immunotherapy with a Nobel laureate. But it had been over four years since she worked with flasks and centrifuges. Her last job was at MIT but as an administrator and communicator. It involved a lot of tweeting.

Then a call came from Tyler Jacks, the renowned head of the MIT Center for Cancer Research (now called the Koch Institute) and her old doctoral advisor. Along with an old college friend, the Silicon Valley inventor Bill Haney, they wanted to bootstrap this new idea out of Jacks lab, a jackknife way of getting the immune system to turn on cancer. And Jacks knew Cheung was itching to get back to the bench.

Soon she found herself in the basement of a Kendall Square bar, in a lab no bigger than a university office, growing antibodies and natural killer cells with a single other employee and hearing the sounds of eating and loud talking whenever they went in the hall. She was the gritty and brilliant, if unlikely, CSO of Dragonfly Therapeutics, and her work there and in the shiny offices theyve since moved to would prove and develop ideas that eventually landed collaborations with Celgene, AbbVie and Bristol Myers Squibb. Last year, it entered patients for the first time.

Her growth has never stopped and this has been true for her entire career, Jacks told me. Theres no challenge that discourages her. Shes kind of fearless.

Cheung didnt arrive as a graduate student at Jacks MIT lab wanting to study immuno-oncology. This was 2002. Checkpoint inhibitors were still a fringe idea in the head of a wild-haired Berkeley professor. No one studied immuno-oncology in Jacks lab.

One evening, though, Bob Schreiber came in for a lecture. Schreibers mice work was just beginning to resurrect the idea of using T cells to attack tumors, and Cheung was captivated. On the walk back that night, she turned to Jacks with an epiphany. Of course the immune system can fight cancer, Cheung said. Cancer is something that is foreign to the body even though it comes from the body, so it absolutely makes sense.

Read more:
Special report: Twenty extraordinary women blazing trails in biopharma R&D Covid-19 and beyond - Endpoints News

Liz Tenety: I am Liz Tenety and I want to welcome you to The Motherly Podcast.

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Liz Tenety: So, as regular podcast listeners might know, I have four children and with three of those pregnancies, I experienced a condition called hyperemesis gravidarum, HG, which is horrible morning sickness. It's debilitating. It's just simply put like the hardest thing that I've ever gone through in my entire life.

And it's terrifying. I really never want to go through it again. For me, thankfully, it tends to let up around 20 weeks, but the first part of my pregnancy is unbelievably hard. I cannot get out of bed. I am vomiting constantly, even on medication. I can't do anything. I can't take care of my kids. I can barely work and I've had it with my first, my third and my fourth children.

Having HG is part of my story and it is one that has been incredibly humbling and scary, and almost every woman I know who has had a baby has some really hard thing that she's gone through, whether it's secondary infertility or HG or a genetic condition that gets diagnosed when she's pregnant. Finding out her baby is breech, having an unexpected C-section, postpartum depression We go through incredible hardships to bring babies into the world. And I don't think we talk about that enough. I don't think women get enough credit for being the heroes, the heroines that they are to endure this, to bring the next generation.

Liz Tenety: Hey mama. Welcome to the motherly podcast, honest conversations about modern motherhood. My name is Liz Tenety. I am the Co-founder of Motherly and I'm a mom of four myself. Today's interview is with Dr. Marlena Fejzo, a geneticist and the leading researcher on hyperemesis gravidarum. HG, as it's called for short, affects about 6 million women globally each year during pregnancy.

And it's characterized by severe nausea, vomiting, weight loss, dehydration, even sometimes organ failure. And in severe cases, it can lead to the death of mothers and babies. After experiencing HG herself and losing her baby to the condition Dr. Fejzo decided to devote her life to studying it. She's discovered two genes associated with hyperemesis gravidarum that has helped to shed a light on this little understood illness. In fact, her work has really changed the paradigm about what causes HG and this really debilitating condition for pregnant women. I actually experienced HG in three of my four pregnancies. So, I am so grateful for Dr. Fejzo's work.

Currently, Dr. Fejzo is a science advisor for the Hyperemesis Education and Fesearch Foundation called the HER Foundation for short. And that's the global voice for HG awareness research and support. Today it receives 200,000 visitors each year from around the world and they manage a global volunteer network with over 900 participants, all built to help women suffering from HG, find the resources and support they need.

I talked to Dr. Fejzo about her own experience with HG and how going through HG as a geneticist inspired her to do this kind of research and really lead the charge on understanding what the actual underlying causes of this debilitating condition. And we also talked about funding of women's health and how there is so little still understood about pregnancy and the changes that women go through biologically through their lives.

There are a lot of reasons why women's health has been historically underfunded and we get into it. Her work is incredible. I'm excited to share this conversation with you.

Dr. Marlena Fejzo, welcome to The Motherly Podcast.

Dr. Marlena Fejzo: Thank you for having me.

Liz Tenety: So, I know we just met, but you are one of my favorite people on the planet. I have been diving into your incredible research, your work on hyperemesis gravidarum known as HG and as a woman who has suffered from HG three times, like I cannot get enough of learning about what you are discovering.

And I can't wait to talk about all of that. So, can you actually define what hyperemesis gravidarum is and what are the symptoms that women should be looking out for?

Dr. Marlena Fejzo: So, hyperemesis gravidarum is nausea and vomiting. That's severe enough to affect your daily routine and to affect your intake so that you're not able to eat or drink properly. And that leads to weight loss. Usually it's diagnosed when you have more than 5% of body weight loss and electrolyte disturbances for not being able to drink or eat properly.

Liz Tenety: So, can you tell our listeners a bit about your own personal story and why you decided to devote your life to not only studying HG, but a lot of critical topics in women's health?

Dr. Marlena Fejzo: I did my PhD in genetics and women's health. My PhD was from Harvard on uterine fibroid tumors, which are also a big problem for women. And, you know, there's not as many female scientists as male scientists. And so, for me, it was very important to focus on women's problems. You know, there's plenty of scientists working on male problems and not very many working on female problems.

And then I went on then I had HG during my post-doc and then had it again. In my second pregnancy, I had a really, really, very severe HG pregnancy. My second pregnancy, the baby died in the second trimester because I was so ill. I didn't keep anything down for 10 weeks and I couldn't move without vomiting. I got so weak. I couldn't speak. I had to have a buzzer to signal when I needed a bed pan change or a medication change. I basically could not move for weeks and weeks. And finally, the baby died. And I decided to look into what was known about HG and there was so little known that I decided, I'm a scientist and I've got to devote my life to figuring this out.

Liz Tenety: There's so much you've already said that is so powerful. And I know our listeners can't see us, but I'm tearing up hearing it because there aren't words to describe what you go through with HG. That was part of this conversation as I was imagining it, I can't tell you what it's like to go through that, but you described in your particular case, you literally could not move your body. It was the most dark time of my life to go through those three pregnancies.

And yet, you have led a body of research into the evidence of what is actually happening inside women's bodies. When they experience HG, what was believed about the cause of HG before, you know, there was this new wave of research and understanding.

Dr. Marlena Fejzo: Yeah. So, that's the terrible thing, since it's a women's problem. And on top of that, a women's problem during pregnancy, and there's sort of always, you know, you see it still in the movies and on TV that there's this idea that pregnant women are hysterical or irrational. And so, that perpetuates this idea that it was all in a woman's head and over time, there were theories that really got picked up that it was a subconscious rejection of their pregnancy that they didn't want to be pregnant and all kinds of irrational theories that are just not scientific and not true.

Liz Tenety: So, what is the difference between morning sickness as we know it and what do we believe morning sickness like what is the purpose of morning sickness and what is the difference between that and HG?

Dr. Marlena Fejzo: No one really knows for sure, but we can guess that morning sickness has evolved as a way to avoid foodst hat would be dangerous. When the fetus is developing, when all the organs are developing and you don't want to ingest anything that could affect that.

And so, what comes with morning sickness is this increased sense of smell and taste. And so, those are generally thought as a way that has evolved to protect the fetus. With HG, it's just morning sickness out of control.

Liz Tenety: I kind of had heard from my doctor when I first had HG with my first child, she seemed to think it was in my stomach, that the problem was happening in my stomach. And it was about eating crackers and always having something in my belly. These were the ideas that I was being told to start to remedy it. But your research is showing that it's actually happening in the brain, or it's at least this cycle of feedback that is neurological.

Dr. Marlena Fejzo: Exactly. So, since I'm a geneticist, I decided to look first into seeing whether it was genetic. I don't have it in my family, but I decided to look into it. And what I found was that. There was a lot of evidence that it's genetic, there's a 17-fold, increased risk of having it if your sister has it. And twin studies where they compare the genes and the chance of getting hyperemesis in identical twins versus fraternal twins have shown that it is highly heritable.

So, there's a lot of evidence that it's genetic. There's 70% heritability. What I then did was to embark on a genetic study and I partnered with the personal genetics company, 23andme and we scanned over 15 million genetic variance in over 50,000 women that did not have HG compared to 1300 women that were hospitalized with HG.

And so, we compare their DNA at these variants and we found very significant differences in their DNA around this hormone called GDF 15. And that hormone is turned on in the placenta. It's expressed very highly in pregnancy. A lot of doctors thought that it was caused by a pregnancy hormone, if they didn't believe the psychological theory, but we found no evidence to support GDF 15 as being the causal hormone. We found very strong evidence for it being this hormone, GDF 15.

Then I went on to see, okay, so it's genetically associated, but what about the actual hormone itself and confirmed that there are significantly higher levels of this hormone in the blood in women with HG compared to women without HG. And then, recently the receptor for this hormone was found by other groups and the receptor, which is what the hormone binds in the brain that causes this nausea and vomiting and loss of appetite and change in taste, it goes up travels through the bloodstream. It goes into the brain, binds this receptor, and then causes this nausea and vomiting. And that can get out of control because if you already have this predisposition to have higher levels, you're going to vomit more than normal. You're going to have nutrient deficiencies, which lead to another further increase in the levels of GDF 15 and you get this downward spiral to hyperemesis gravidarum.

Liz Tenety: And those nutritional deficiencies that mothers get when they have HG, lead to, you know, negative impacts on the fetus, including much higher rates of autism.

Dr. Marlena Fejzo: Yeah. So, there is, I wouldn't say much higher I don't want to scare women, but there is an increased risk for neurodevelopmental delay.

There's an increased risk for autism, you know, speech, delay, language disorders, different kinds of neurodevelopmental disorders. And, you know, long-term outcomes in the mother, as well as the child. So, it used to always be thought that, you know, don't worry about what you get. The baby's getting everything it needs from mom, but in the case of HG, that is just not true.

Liz Tenety: That is literally what my doctor told me. And that child, that first pregnancy we have had diagnoses along those lines. So, you know, I can't say for sure, but I am living your research every day. I wonder when you're doing your work, you brought up how women's health and maternal health for a number of reasons has not been as much of a focus of the scientific community as the health and wellness of men.

Do you feel excited about being a pioneer in this field, or do you feel frustrated that we've had women as long as we've had human beings and we still don't know some of the basic things of how our bodies work, because we haven't invested in this kind of research?

Dr. Marlena Fejzo: That's a good question. I would say I have days of both there's days where I'm really excited and so happy that we're making progress, but the progress is so slow. We need other people following my path. The fact that I am a world-renowned researcher in this field is not really because I'm so great. It's because there's barely anybody else. It does definitely bother me that there's so little research out there and that it's going so slowly and that, you know, I still hear stories of women being mistreated with this disease.

And I've been out there publishing for 20 years to try to change that. So, I definitely do feel frustration often, but yes, I'm very excited that we finally made some progress and hopefully we can change things. And a lot of people don't know about my research. And so, it's really important to get the word out and spread the word

Liz Tenety: As a result of your research, finding that there is DNA in certain women, that you will have particular genetics that lead to this cycle, and you have HGit will lead to a different kind of treatment. Is that right? Where are you in that process of now that we understand HG, what do we do about it?

Dr. Marlena Fejzo: Yeah. So, it's a pretty new finding and the fact that this is a hormone and the identification of the receptor for it are pretty new. So, we have a ways to go to understand how this all works, but we are hopeful that there will be medications that will be used to block this pathway so that we can actually have something that works to treat HG. The interesting thing about this hormone is that it also is involved in cancer.

Cachexia is basically when you can no longer eat, when you have cancer. And it kills about 20% of cancer patients. And just like GDF 15 is overexpressed by the placenta, it's also overexpressed in some cancers. And so, those patients that have high levels of GDF 15 turned on in their cancers also have this appetite loss muscle wasting and they ended up many of them dying from it.

When women say they feel like they're dying from HG, often people do not believe them. They say they're exaggerating. But the actual levels of GDF 15 in women that are pregnant and have HG is the same, or sometimes even higher than people that are dying from cachexia.

Liz Tenety: I'm getting choked up again because that's what it feels like. But I think people may not realize too, when you have HG, it doesn't just affect you when you're pregnant. I mean, it affects, of course, the experience. But many women decide to terminate these wanted pregnancies because they cannot endure what they're going through. Some women have PTSD. And even though it's a small segment of women, it is very traumatic and some very prominent women, Amy Schumer, Kate Middleton have gone through it. But like you said, we're just starting to wrap our heads around how overwhelming and how misunderstood this condition has been.

Dr. Marlena Fejzo: Yes, it is overwhelming, misunderstood, and it has long-term effects for the mom and it can have long-term effects for the child.

Liz Tenety: There are other considerations when it comes to reasons why this kind of research hasn't has not happened. One of them is that there was a medication in the 20th century that negatively affected a lot of women and children. There are other ethical considerations with pregnancy. So, how do you navigate that in your own research and understanding.

Dr. Marlena Fejzo: Yeah. So, there was a drug that was given in the fifties to women for HG in Europe. And it ended up causing birth defects, limb deformities. And so, unfortunately, that has caused pharmaceutical companies and doctors and patients themselves to be scared to develop, to treat patients and to take medications in pregnancy.

I think that the tide is starting to change. With that, as you know, we're starting to understand that some medications can be safe in pregnancy and, you know, we have to go slowly to make sure that I don't know if a GDF 15 inhibitor is going to be safe in pregnancy, but we're going to have to try.

And the fact that there can be long-term outcomes to the child if they don't take medication, has to be weighed against the possible risks.

Liz Tenety: Thrilled to hear that the tide is turning. If a woman is experiencing this and she's going to go to her doctor or her midwife, and just say, I am, I'm just so sick. How does she know how to flag that this is not normal morning sickness? That this is a severe condition that requires a different level of intervention.

Dr. Marlena Fejzo: Exactly. So that's why we developed our HG care app so that women could know, because especially in their first pregnancy, a lot of women don't know, and they haven't seen a doctor yet when the symptoms start.

And so, they can be missed before it's too late. There was actually a woman that died just in August. She was only nine weeks pregnant, so it's very important for women to know what the symptoms are and what is normal. And I feel like every woman should be screened immediately when they're first pregnant for this, because right now, a lot of women only go to the doctor and have had their first appointment pretty late after symptoms of HG can already get severe.

So, our app has alerts to tell you when you need to talk to your doctor, but generally if you're getting dehydrated, then it's time to see your doctor. If you're not able to eat, if you're not able to drink, you're not able to do your normal daily routine and you're losing weight, you're actively losing weight. Then you need to talk to your doctor about possibly having HG. There is a test where you can pull up your skin on the back of your hand, and normally it should bounce right back. If it goes back slowly, then that means you're dehydrated. And you need to get hydrated.

Liz Tenety: I want to talk a bit about the foundation. Can you tell me about it?

Dr. Marlena Fejzo: So, basically the way it happened was after my HG nightmare, I realized there was so little known and I put out a survey with my brother. He helped me. He's a statistician. And we put out a survey on the internet about HG and put in questions about it that I wanted to know the answers to. And one of the women that answered that survey faxed back her answers and that was Kimber McGiven and she was a nurse who was going through HG and she said, I'm so sick now, but I was so happy to find your survey. She wrote on her survey, as soon as I'm done with this pregnancy, I'm going to make a website. And so, she made a website on HG and she's amazing.

She's been working for years on this, only recently giving her itself a tiny salary, but she's just devoted all her time with no pay to do this. And she has an amazing website. And then she got joined by another couple that had gone through a horrible HG pregnancy and they then created the foundation: The HER Foundation. And so the webpage is hyperemesis.org and I'm a science advisor for them. And I'm on the board.

Liz Tenety: What is the current best evidence-based treatment for HG? And what can those around a woman going through HG do, if anything, to relieve any of her suffering?

Dr. Marlena Fejzo: So, we did research on what women found to be the most effective treatment. So, I'm basing my answer on that, which is that on Dantron or Zofran was the most effective treatment for women in our study for helping to lessen the, the vomiting. It doesn't necessarily help that much with the nausea in all women, but it does lessen the vomiting in more women than any other medication reported except for possibly steroids.

But doctors often do not give steroids to women because there are some possible safety concerns. So, that's from our study. But unfortunately, even on Zofran, it's reportedly only effective in maybe 50% of women. So, we definitely still need something better.

To answer your second question, what can we tell the families, women with HG need a lot of support? They need an advocate. When you're feeling nauseous, you cannot advocate for yourself. So, they need someone to go with them. To doctor's appointments and and to speak for how ill they are. When a woman is ill and feeling like they're about to throw up, they're not going to speak out to their doctor when their doctor says something that they don't agree with.

Like, oh, just take crackers. So, women need to have either a family member or friend go to the doctor and explain.

Liz Tenety: And yet, like you said, I mean, I literally heard from my doctors, the baby's a parasite and the baby will take everything that the baby needs, but your research has shown that that's just, that's just not true.

Why is it that there's so much conventional wisdom that isn't evidence-based that is still out there in modern medicine, especially as a relates to women?

Dr. Marlena Fejzo: I think it's really hard to get the word out and that these ideas are passed on then medical books and the medical schools. They're teaching the next generation of doctors, the same things that they learned and not updating it.

So, I've heard of this year, a doctor teaching her medical students. (One of them had HG. So, she's the one who told us about this.) She was teaching her medical students, that women, they're sometimes hospitalized, and they just don't want to get better. That's what she told this next generation of doctors in a class. Women just don't want to get better.

This was a female doctor teacher teaching her next generation of students. So, if they're learning that from someone that they trust to teach them the facts, then that's what's happening. And so somehow that has to change and we have to just get the word out the best we can.

Liz Tenety: What are the other big topics in women's health and maternal health that we don't know enough about?

Dr. Marlena Fejzo: There's menopause, lupus that occurs more in women, many, many women's issues. And just having your period and the medications to help with cramps, you know.

Liz Tenety: Well, it seems like being a woman is the qualifying factor, because if its periods to menopause, that covers a lot of our lives that we may not know as much about as we should.

Dr. Marlena Fejzo: One thing you asked me before about, you know, am I excited or am I I don't mean to be only negative because we are making progress and things are slowly changing. But one of the things that I was really upset about that really got me riled up was that the national Institute of health funded a genome-wide association study, which is the same type of study that I did with 23andMe, because I couldn't get funding from the NIH.

They funded a study on erectile dysfunction. Erectile dysfunction, first of all, is not nearly as heritable. There's not as much genetic evidence to support that it is genetic compared to hyperemesis and other women's issues.

And yet they funded that. In addition, with erectile dysfunction, there are millions of dollars, billions of dollars spent on it by drug companies by the government. That was really frustrating to me. Erectile dysfunction is bad too, but when you compare it to HG, that's affecting the mother and the child. I don't think you can compare it. And so, it's just an example of money being spent on male issues and not female issues.

Liz Tenety: After you lost your baby at 15 weeks through that experience you had with HG, you did go on to have other children. How did you navigate the decision to have more children? And I know you ended up working with a surrogate. So, what was that like? Tell us about that.

Dr. Marlena Fejzo: Yeah, so having a surrogate was never something that was on my radar, especially back then. There were very few people that did that.

Liz Tenety: What year was that?

Dr. Marlena Fejzo: That was back in 1999. It was something that my OB suggested to me that I was a good candidate for. And after I lost the baby, you know, I had one child and I come from a big family. I have two brothers and a sister, and I always wanted a big family too. So, it was going to be either surrogacy or adoption for me. I was not going to go through HG again.

Liz Tenety: Do you see those experiences as different motherhood's or is there a theme in how you've bonded with those children in the same way or in a different way? How do you see that?

Dr. Marlena Fejzo: I think it was the same as far as, you know, giving birth and having the surrogate. I was able to watch her give birth to my babies, and I really consider her my angel for doing that for me. And both ways are miracles, you know, giving birth to a child is a miracle, but having someone else do that for you is also such an amazing miracle.

Liz Tenety: Do you feel that the loss of your child that you did experience is a source of motivation or meaning in your ongoing research and advocacy for HG?

Dr. Marlena Fejzo: Definitely. I mean, that is what keeps me going. I don't want that to happen to anybody else. I know it's still happening to women and it's very sad for me when I, you know, women write me and tell me they've gone through that. It's very sad and frustrating to me because I've been working on this for 20 years and to see that women are still going through this and I hope that it's going to change. And I hope that we're going to find a medication that works and stops that. But yeah, definitely. And I don't want to see my daughters go through it either, of course. So, that will be devastating to me if they have to go through that.

Liz Tenety: Okay. So, at Motherly, we believe that motherhood brings out our superpowers and to me, a superpower is something unique, a strength perhaps within you that maybe you didn't even know was there before you became a mom. So, I wonder what you think is your superpower.

Dr. Marlena Fejzo: Patience. I would say patience is really important as a mother and as a scientist. Patience and trudging slowly on. There have been many, many setbacks in my research and just getting up and go forward again. Being patient. We're getting there. But yeah, with kids too. You need patience.

Liz Tenety: I love that. Well, Dr. Marlena Fejzo, thank you so much for sharing your story and your research with baths on The Motherly Podcast.

Dr. Marlena Fejzo: Thank you for having me.

Liz Tenety: Now for a quick word, from this episode's sponsor ThirdLove. Motherhood is one of the most amazing and transformational experiences a woman can have. It's about feeling the joy of bringing this new little person into the worlds and learning how to be comfortable in our own skin, finding a new routine and owning our new confidence.

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Liz Tenety: When I'm pregnant, do you remember what happens to me at the beginning of most of my pregnancies? What happens when I get really sick?

Henry: You start to throwing up like three times, each day, three to 10 times each day.

Liz Tenety: Yeah. It's like 10 to 15 times a day. Yes, you're totally right. If you remember, I just basically stayed in bed for three months. Do you remember that part?

Grant: How'd you eat?

Liz Tenety: I couldn't eat

Grant: But then you wouldn't be able to survive.

Liz Tenety: I just get the tiniest amounts of food and water. And sometimes they put the IVs in my arms to get me fluid. But you know, I was like that with you, Henry. But with Grant, I didn't get really sick, but so with three of the kids pregnancies, I got super, super sick, but one kid and I'm looking at him, he did not make me throw up 10 times a day. Good job, Grant!

Grant: Because I'm so disgusting.

Liz Tenety: Oh, is that it? Because you are so disgusting, that's why I didn't throw up?

Grant: Hmmhmmm.

Liz Tenety: Well, that's it for our show this week. I can't thank you enough, Dr. Fejzo for your time and for your work.

And I also want to thank our listeners for listening to our podcast. This season, we have an incredible lineup coming in 2021. I can't wait for you to listen. And as always, we would love it if you spread the word about The Motherly Podcast.

So, if you can leave us a review on Apple podcasts, it takes 30 seconds max, and it really helps other mamas discover our show.

The Motherly Podcast is produced by Jennifer Bassett with editing from Seaplane Armada. Our music is from the blue dot sessions and I am your host, Liz Tenety. Thank you so much for listening.

Read more from the original source:
Hyperemesis Gravidarum researcher Dr. Marlena Fejzo is on a mission to understand women's health - Motherly Inc.

The Telegraph

Australia has cancelled the production of a locally made Covid-19 vaccine after trial volunteers falsely tested positive for HIV, meaning the drug could interfere with diagnosis of that virus. Antibodies generated by the jabs developed by the University of Queensland (UQ) and biotech firm CSL led to trial subjects wrongly testing positive for the virus that causes AIDS. Further trials have been stopped. Scientists said the results were a blow to Australia's vaccine development and was likely to force the country to buy more doses of imported shots. "While this is a tough decision to take, the urgent need for a vaccine has to be everyone's priority," said UQ professor Paul Young. Australia has ordered a total of 140 million shots from different suppliers, to inoculate its 25 million people, making it one of the most highly stocked countries in the world. "We want to ensure that Australians ... have full confidence, absolute full confidence that when it gets the tick, they can get the jab, and they can make that decision for themselves and for their families, confidently, said Scott Morrison, prime minister. Prof Sarah Palmer, from the faculty of medicine at the University of Sydney, said: Sadly, this is a set-back for the development of Covid-19 vaccines. Generating a false positive for HIV is entirely unexpected for this vaccine, but underscores the critical necessity of testing the safety of newly-developed vaccines in large numbers of volunteers. She said the Australian government, which was a major backer of the UQ vaccine effort, would have to consider funding other alternatives, including imported vaccine from firms such as Pfizer and Moderna. Australia's strict quarantine regime has seen the country quash earlier outbreaks and its tally of 28,000 infections is far fewer than in many other developed countries Its success in keeping a lid on infections has meant the country is not racing to start vaccinations like countries in Europe and jabs are not scheduled to begin until March. CSL, had been under a contract to produce 51 million doses of the UQ vaccine, and will instead produce an extra 20 million doses of the Oxford vaccine being developed with Britain's AstraZeneca.

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Loeffler claims she is the candidate who will create jobs - Yahoo News