Category Archives: Stem Cell Medicine


SNACS: The FAANG Of The Roaring 20s – Yahoo Finance

The roaring 20s are upon us, and the investment opportunities set in front of us are exhilarating. This new decade has a lot in store for us with tech as the driving force behind it.

FAANG was the acronym that drove the stock market to continuously new highs over the past decade: Facebook (FB), Amazon (AMZN), Apple (AAPL), Netflix (NFLX), and Google aka Alphabet (GOOGL). This is an acronym that I am sure you are familiar with. These stocks exponential returns may be exhausted, and a new set of equities are ready to take their place. It is time to look for the new FAANG.

When assessing market-shifting companies, you need to look for firms with an exciting product offering characterized by longevity and a substantial total addressable market (TAM). Firms with savvy management teams that are able to navigate through both the best and worst times nimbly.

I have chosen a new acronym of stocks that I believe could change the world in the roaring 20s. The companies include Crispr (CRSP), Sea Limited (SE), Alibaba (BABA), Nvidia (NVDA), and Splunk (SPLK) or SCANS, as I like to call it.

Here I will give a brief introduction of each stock and explain why I believe these shares will drive the market in this new decade.

Sea Limited (SE)

Sea is the leading internet company in Southeast Asia and Taiwan. These economies are digitalizing at an exponential rate, and Sea is well-positioned to take on the quickly expanding addressable market. The company operates three market leader segments, including an ecommerce platform, a digital entertainment division, and a digital payment company (Shopee, Garena, and AirPay, respectively).

The internet economy in Southeast Asia has tripled in the past 5 years to $100 billion and is expected to triple again by 2025 to $300 billion. Sea is growing at an even faster rate, with year-over-year topline appreciation in the high triple-digit percentages as the company continues to take an increasing amount of market share.

Sea Limited is going to be the tech powerhouse that helps turn the third world economies of Southeast Asia and Taiwan into digitalized world markets.

CRISPR (CRSP)

CRISPR is a biomedical firm that is on the verge of changing the world. This company can edit an individuals DNA, an achievement that is going to change modern medicine. This technology could be used to cure almost any disease if it is successfully implemented. What CRISPRs gene therapy does is splice out the bad or disease driving DNA and add healthy strands. The company is also a leader in regenerative stem-cell medicine, which could save the lives of 100s of thousands.

CRISPR has an established portfolio of life-changing therapies in its pipeline at various stages of development. Hemoglobinopathy is the closest to commercially viable and is currently in clinical trials. If it passes clinical trials, I see this stock jumping substantially.

These shares are still a risky asset considering the possibility that none of its gene-therapies make it past the clinical stage. Based on early trials, it appears that the therapy does indeed work, and this potential has begun to be priced into CRSP. The stock has appreciated 350% since it went public in late 2016, and I believe that this is just the beginning of its growth. The ability to change an individuals DNA is going to change the world of medicine.

Alibaba (BABA)

The Amazonof the East has been driving substantial growth, but I dont believe that investors are correctly valuing Alibabas fundamentals. BABA is trading at roughly 1/3rd of Amazons forward P/E valuation (seen below), despite achieving wider margins, stronger profitable, and a greater growth outlook. Alibaba is operating in one of the worlds largest and fastest-growing consumer markets (China).

Story continues

Alibaba controls not only Chinas ecommerce market but also its cloud computing space with a 47% market share. Its cloud computing space has the most room to run as Chinas cloud infrastructure continues to expand at an exponential rate high double-digit to triple-digit percentages.

Alibaba still has some geopolitical risk due to the US-China trade war, but as far as this next decades biggest equity drivers, I would replace AZMN with BABA in my portfolio.

Nvidia (NVDA)

This is the most exciting chip maker in the world today. Nvidia is known for the invention of the GPU, which is a chip original purposed for image rendering, but Nvidia has taken its capabilities far beyond this. Nvidias chips are hyper-fast and slowly becoming smarter as the technology develops. Its chips are becoming a necessity in data centers and are an essential element of AI development. I believe that one of Nvidias integrated circuits will be apart of the first true AI, which is going to change the world.

Nvidia is also leveraging 5G with its anticipated cloud gaming platform. Like cloud computing is the future of business data and analytics, cloud gaming is the future of gaming. Nvidia is making a big bet in this field with its cloud platform, GeForce NOW. This platform allows gamers to use their Macs or PCs for gaming anywhere with the high-speed, low-latency technology of Nvidias GPUs without needing Nvidias hardware locally.

Nvidia is undoubtedly a company of the future, and despite its 4-digit gains over the past decade, I believe that this stock still has legs to run. I dont think that the company has scratched the surface of what its chips could do.

Splunk (SPLK)

Splunk is a platform that helps companies utilize real-time machine data for collection, indexing, and alerts, allowing companies to uncover actionable insight from this data no matter the source or format. The company is leveraging AI and machining learning for forecasting and anticipative decision making.

Real-time data management is becoming increasingly necessary in business across industries as this digital age makes speed a competitive advantage. Splunk is well-positioned to take on the massive addressable market that is yet to recruit Splunks services. This firm is well-suited to transform the way our economy utilizes real-time data.

Take Away

The market driving stocks will undoubtedly make excellent long-term investments for the roaring 20s. SCANS will be a force to be reckoned with in this next decade. Short term volatility in these stocks shouldnt cause you to shy away from their long-term potential. I believe we may be on the edge of a market correction, so if you are worried about short-term earnings, I may wait for a pullback. If you are a long-term investor that is willing to ride this decades waves, I wouldnt hesitate to pull the trigger on these stocks.

Breakout Biotech Stocks with Triple-Digit Profit Potential

The biotech sector is projected to surge beyond $775 billion by 2024 as scientists develop treatments for thousands of diseases. Theyre also finding ways to edit the human genome to literally erase our vulnerability to these diseases.

Zacks has just released Century of Biology: 7 Biotech Stocks to Buy Right Now to help investors profit from 7 stocks poised for outperformance. Our recent biotech recommendations have produced gains of +98%, +119% and +164% in as little as 1 month. The stocks in this report could perform even better.

See these 7 breakthrough stocks now>>

Click to get this free report Splunk Inc. (SPLK) : Free Stock Analysis Report Sea Limited Sponsored ADR (SE) : Free Stock Analysis Report NVIDIA Corporation (NVDA) : Free Stock Analysis Report Netflix, Inc. (NFLX) : Free Stock Analysis Report Alphabet Inc. (GOOGL) : Free Stock Analysis Report Facebook, Inc. (FB) : Free Stock Analysis Report CRISPR Therapeutics AG (CRSP) : Free Stock Analysis Report Alibaba Group Holding Limited (BABA) : Free Stock Analysis Report Amazon.com, Inc. (AMZN) : Free Stock Analysis Report Apple Inc. (AAPL) : Free Stock Analysis Report To read this article on Zacks.com click here. Zacks Investment Research

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SNACS: The FAANG Of The Roaring 20s - Yahoo Finance

20 Technology Metatrends That Will Define the Next Decade – Singularity Hub

In the decade ahead, waves of exponential technological advancements are stacking atop one another, eclipsing decades of breakthroughs in scale and impact.

Emerging from these waves are 20 metatrends likely to revolutionize entire industries (old and new), redefine tomorrows generation of businesses and contemporary challenges, and transform our livelihoods from the bottom up.

Among these metatrends are augmented human longevity, the surging smart economy, AI-human collaboration, urbanized cellular agriculture, and high-bandwidth brain-computer interfaces, just to name a few.

It is here that master entrepreneurs and their teams must see beyond the immediate implications of a given technology, capturing second-order, Google-sized business opportunities on the horizon.

Welcome to a new decade of runaway technological booms, historic watershed moments, and extraordinary abundance.

Lets dive in.

(1) Continued increase in global abundance: The number of individuals in extreme poverty continues to drop, as the middle-income population continues to rise. This metatrend is driven by the convergence of high-bandwidth and low-cost communication, ubiquitous AI on the cloud, and growing access to AI-aided education and AI-driven healthcare. Everyday goods and services (finance, insurance, education, and entertainment) are being digitized and becoming fully demonetized, available to the rising billion on mobile devices.

(2) Global gigabit connectivity will connect everyone and everything, everywhere, at ultra-low cost: The deployment of both licensed and unlicensed 5G, plus the launch of a multitude of global satellite networks (OneWeb, Starlink, etc.), allow for ubiquitous, low-cost communications for everyone, everywhere, not to mention the connection of trillions of devices. And todays skyrocketing connectivity is bringing online an additional three billion individuals, driving tens of trillions of dollars into the global economy. This metatrend is driven by the convergence oflow-cost space launches, hardware advancements, 5G networks, artificial intelligence, materials science, and surging computing power.

(3) The average human healthspan will increase by 10+ years:A dozen game-changing biotech and pharmaceutical solutions (currently in Phase 1, 2, or 3 clinical trials) will reach consumers this decade, adding an additional decade to the human healthspan. Technologies include stem cell supply restoration,wnt pathway manipulation, senolytic medicines, a new generation of endo-vaccines, GDF-11, and supplementation of NMD/NAD+, among several others. And as machine learning continues to mature, AI is set to unleash countless new drug candidates, ready for clinical trials. This metatrend is driven by the convergence ofgenome sequencing, CRISPR technologies, AI, quantum computing, and cellular medicine.

(4) An age of capital abundance will see increasing access to capital everywhere: From 2016 2018 (and likely in 2019), humanity hit all-time highs in the global flow of seed capital, venture capital, and sovereign wealth fund investments. While this trend will witness some ups and downs in the wake of future recessions, it is expected to continue its overall upward trajectory. Capital abundance leads to the funding and testing of crazy entrepreneurial ideas, which in turn accelerate innovation. Already, $300 billion in crowdfunding is anticipated by 2025, democratizing capital access for entrepreneurs worldwide. This metatrend isdriven by the convergence of global connectivity, dematerialization, demonetization, and democratization.

(5) Augmented reality and the spatial webwill achieve ubiquitous deployment: The combination of augmented reality (yielding Web 3.0, or the spatial web) and 5G networks (offering 100Mb/s 10Gb/s connection speeds) will transform how we live our everyday lives, impacting every industry from retail and advertising to education and entertainment. Consumers will play, learn, and shop throughout the day in a newly intelligent, virtually overlaid world. This metatrend will be driven by the convergence ofhardware advancements, 5G networks, artificial intelligence, materials science, and surging computing power.

(6) Everything is smart, embedded with intelligence: The price of specialized machine learning chips is dropping rapidly with a rise in global demand. Combined with the explosion of low-cost microscopic sensors and the deployment of high-bandwidth networks, were heading into a decade wherein every device becomes intelligent. Your childs toy remembers her face and name. Your kids drone safely and diligently follows and videos all the children at the birthday party. Appliances respond to voice commands and anticipate your needs.

(7) AI will achieve human-level intelligence:As predicted by technologist and futurist Ray Kurzweil, artificial intelligence will reach human-level performance this decade (by 2030). Through the 2020s, AI algorithms and machine learning tools will be increasingly made open source, available on the cloud, allowing any individual with an internet connection to supplement their cognitive ability, augment their problem-solving capacity, and build new ventures at a fraction of the current cost. This metatrend will bedriven by the convergence ofglobal high-bandwidth connectivity, neural networks, and cloud computing. Every industry, spanning industrial design, healthcare, education, and entertainment, will be impacted.

(8) AI-human collaboration will skyrocket across all professions: The rise of AI as a Service (AIaaS) platforms will enable humans to partner with AI in every aspect of their work, at every level, in every industry. AIs will become entrenched in everyday business operations, serving as cognitive collaborators to employeessupporting creative tasks, generating new ideas, and tackling previously unattainable innovations. In some fields, partnership with AI will even become a requirement. For example: in the future, making certain diagnoses without the consultation of AI may be deemed malpractice.

(9) Most individuals adapt aJARVIS-like software shell to improve their quality of life: As services like Alexa, Google Home, and Apple Homepod expand in functionality, such services will eventually travel beyond the home and become your cognitive prosthetic 24/7. Imagine a secure JARVIS-like software shell that you give permission to listen to all your conversations, read your email, monitor your blood chemistry, etc. With access to such data, these AI-enabled software shells will learn your preferences, anticipate your needs and behavior, shop for you, monitor your health, and help you problem-solve in support of your mid- and long-term goals.

(10) Globally abundant, cheap renewable energy: Continued advancements in solar, wind, geothermal, hydroelectric, nuclear, and localized grids will drive humanity towards cheap, abundant, and ubiquitous renewable energy. The price per kilowatt-hour will drop below onecent per kilowatt-hour for renewables, just as storage drops below a mere three cents per kilowatt-hour, resulting in the majority displacement of fossil fuels globally. And as the worlds poorest countries are also the worlds sunniest, the democratization of both new and traditional storage technologies will grant energy abundance to those already bathed in sunlight.

(11) The insurance industry transforms from recovery after risk to prevention of risk:Today, fire insurance pays youafteryour house burns down; life insurance pays your next-of-kinafteryou die; and health insurance (which is really sick insurance) pays onlyafteryou get sick. This next decade, a new generation of insurance providers will leveragethe convergence of machine learning, ubiquitous sensors, low-cost genome sequencing, and robotics to detect risk,preventdisaster, and guarantee safety before any costs are incurred.

(12) Autonomous vehicles and flying cars will redefine human travel (soon to be far faster and cheaper):Fully autonomous vehicles, car-as-a-service fleets, and aerial ride-sharing (flying cars) will be fully operational in most major metropolitan cities in the coming decade. The cost of transportation will plummet 3-4X, transforming real estate, finance, insurance, the materials economy, and urban planning. Where you live and work, and how you spend your time, will all be fundamentally reshaped by this future of human travel. Your kids and elderly parents will never drive. This metatrend will bedriven by the convergence ofmachine learning, sensors, materials science, battery storage improvements, and ubiquitous gigabit connections.

(13) On-demand production and on-demand delivery will birth an instant economy of things:Urban dwellers will learn to expect instant fulfillment of their retail orders as drone and robotic last-mile delivery services carry products from local supply depots directly to your doorstep. Further riding the deployment of regional on-demand digital manufacturing (3D printing farms), individualized products can be obtained within hours, anywhere, anytime. This metatrend isdriven by the convergence of networks, 3D printing, robotics, and artificial intelligence.

(14) Ability to sense and know anything, anytime, anywhere:Were rapidly approaching the era wherein 100 billion sensors (the Internet of Everything) is monitoring and sensing (imaging, listening, measuring) every facet of our environments, all the time. Global imaging satellites, drones, autonomous car LIDARs, and forward-looking augmented reality (AR) headset cameras are all part of a global sensor matrix, together allowing us to know anything, anytime, anywhere. This metatrend isdriven by the convergence ofterrestrial, atmospheric and space-based sensors, vast data networks, and machine learning. In this future, its not what you know, but rather the quality of the questions you ask that will be most important.

(15) Disruption of advertising:As AI becomes increasingly embedded in everyday life, your custom AI will soon understand what you want better than you do. In turn, we will begin to both trust and rely upon our AIs to make most of our buying decisions, turning over shopping to AI-enabled personal assistants. Your AI might make purchases based upon your past desires, current shortages, conversations youve allowed your AI to listen to, or by tracking where your pupils focus on a virtual interface (i.e. what catches your attention). As a result, the advertising industrywhich normally competes foryourattention (whether at the Superbowl or through search engines)will have a hard time influencing your AI. This metatrend isdriven by the convergence ofmachine learning, sensors, augmented reality, and 5G/networks.

(16) Cellular agriculture moves from the lab into inner cities, providing high-quality protein that is cheaper and healthier: This next decade will witness the birth of the most ethical, nutritious, and environmentally sustainable protein production system devised by humankind. Stem cell-based cellular agriculture will allow the production of beef, chicken, and fishanywhere, on-demand, with far higher nutritional content, and a vastly lower environmental footprint than traditional livestock options. This metatrend isenabled by the convergence ofbiotechnology, materials science, machine learning, and AgTech.

(17) High-bandwidth brain-computer interfaces (BCIs) will come online for public use: Technologist and futurist Ray Kurzweil has predicted that in the mid-2030s, we will begin connecting the human neocortex to the cloud. This next decade will see tremendous progress in that direction, first serving those with spinal cord injuries, whereby patients will regain both sensory capacity and motor control. Yet beyond assisting those with motor function loss, several BCI pioneers are now attempting to supplement their baseline cognitive abilities, a pursuit with the potential to increase their sensorium, memory, and even intelligence. This metatrend is fueled by the convergence ofmaterials science, machine learning, and robotics.

(18) High-resolution VRwill transform both retail and real estate shopping:High-resolution, lightweight virtual reality headsets will allow individuals at home to shop for everything from clothing to real estate from the convenience of their living room. Need a new outfit? Your AI knows your detailed body measurements and can whip up a fashion show featuring your avatar wearing the latest 20 designs on a runway. Want to see how your furniture might look inside a house youre viewing online? No problem! Your AI can populate the property with your virtualized inventory and give you a guided tour. This metatrend isenabled by the convergence of: VR, machine learning, and high-bandwidth networks.

(19) Increased focus on sustainability and the environment:An increase in global environmental awareness and concern over global warming will drive companies to invest in sustainability, both from a necessity standpoint and for marketing purposes. Breakthroughs in materials science, enabled by AI, will allow companies to drive tremendous reductions in waste and environmental contamination. One companys waste will become another companys profit center. This metatrend isenabled by the convergence ofmaterials science, artificial intelligence, and broadband networks.

(20) CRISPR and gene therapies will minimize disease:A vast range of infectious diseases, ranging from AIDS to Ebola, are now curable. In addition, gene-editing technologies continue to advance in precision and ease of use, allowing families to treat and ultimatelycurehundreds of inheritable genetic diseases. This metatrend isdriven by the convergence of various biotechnologies (CRISPR, gene therapy), genome sequencing, and artificial intelligence.

(1) A360 Executive Mastermind:If youre an exponentially and abundance-minded entrepreneur who would like coaching directly from me, consider joining my Abundance 360 Mastermind, a highly selective community of 360 CEOs and entrepreneurs who I coach for 3 days every January in Beverly Hills, Ca. Through A360, I provide my members with context and clarity about how converging exponential technologies will transform every industry. Im committed to running A360 for the course of an ongoing 25-year journey as a countdown to the Singularity.

If youd like to learn more and consider joining our 2020 membership,apply here.

(2) Abundance-Digital Online Community:Ive also created a Digital/Online community of bold, abundance-minded entrepreneurs called Abundance-Digital. Abundance-Digital is Singularity Universitys onramp for exponential entrepreneurs those who want to get involved and play at a higher level.Click here to learn more.

(Both A360 and Abundance-Digital are part of Singularity Universityyour participation opens you to a global community.)

This article originally appeared ondiamandis.com. Read theoriginal article here.

Image Credit: Image by Free-Photos from Pixabay

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20 Technology Metatrends That Will Define the Next Decade - Singularity Hub

LANL Scientists And International Partners Create 3-D Image Of Heart RNA Structure For First Time – My Blog – Los Alamos Daily Post

The first 3-D images have been created of an RNA molecule known as Braveheart for its role in transforming stem cells into heart cells. Courtesy/LANL

LANL News:

Scientists at Los Alamos National Laboratory and international partners have created the first 3-D images of a special type of RNA molecule that is critical for stem cell programming and known as the dark matter of the genome.

As far as we know, said corresponding author Karissa Sanbonmatsu, this is the first full 3-D structural study of any long, non-coding RNA (lncRNA) other than a partial structure.

Sanbonmatsu is a structural biologist at LANL.

A better understanding of these RNAs could lead to new strategies in regenerative medicine for people with heart conditions due to cardiovascular disease or aging,Sanbonmatsu said.

The team used a technique called small angle X-ray scattering (SAXS) that reveals the 3-D envelope of the RNA molecule, according to Trushar Patel, a Canadian professor on the team. Next, with the help of machine learning and high-performance computing, they made atomistic models to fit inside the envelopes this included the creation of an atomistic model that is also the longest of an isolated RNA (636 nucleotides) to date, said Doo Nam Kim, lead author on the Nature Communications paper.

Our work represents the first step in showing that these difficult-to-image RNAs do possess 3-D structures, and that these molecular structures may very well determine how they operate, Sanbonmatsu said. The RNA studied is called Braveheart it triggers the transformation of stem cells into heart cells.

Before the human genome was sequenced in 2000, it was thought that it mostly contained instructions for proteins, the workhorse molecules of human cells. Scientists were shocked to discover that less than 10 percent of the genome encoded proteins. Ever since, the other 90 percent was deemed to be junk DNA or dark matter. Enter RNA, the molecular cousin of DNA. Scientists originally assumed the main purpose of RNA was simply to coordinate as a messenger for DNA in the synthesis of proteins. However, it has recently been shown that more than 90 percent of the genome encodes a new and mysterous class of RNAs, called long non-coding RNA molecules (lncRNA).

These RNA molecules help to control the turning on and off of genes; their malfunction causes birth defects, autism and even cancer in some cases. They also are key to reprogramming adult stem cells. Even though the molecules make up 90 percent of the genome, scientists have almost no idea how they work, or even what they look like. In this study, one of the largest RNA-only 3-D studies, the new 3-D images sets the stagefor future studies that will shed more light on how they control genes.

Publication:

Zinc-finger protein CNBP alters the 3-D structure of lncRNA Braveheart in solution Authors: Karissa Y. Sanbonmatsu, Doo Nam Kim and Scott P. Hennelly of Los Alamos National Laboratory and New Mexico Consortium; Bernhard C. Thiel and Ivo L. Hofacker of University of Vienna, Austria; Tyler Mrozowich and Trushar R. Patel of University of Lethbridge, Canada. Nature Communications, DOI 10.1038/s41467-019-13942-4

Funding:

National Institutes of Health, Los Alamos Institutional Computing and Los Alamos Laboratory Directed R& D, and Diamond Light Source (UK).

AboutLos Alamos National Laboratory:

Los Alamos National Laboratory, a multidisciplinary research institution engaged in strategic science on behalf of national security, is operated by Triad, a public service oriented, national security science organization equally owned by its three founding members: Battelle Memorial Institute (Battelle), the Texas A&M University System (TAMUS), and the Regents of the University of California (UC) for the Department of Energys National Nuclear Security Administration.

Los Alamos enhances national security by ensuring the safety and reliability of the U.S. nuclear stockpile, developing technologies to reduce threats from weapons of mass destruction, and solving problems related to energy, environment, infrastructure, health, and global security concerns.

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LANL Scientists And International Partners Create 3-D Image Of Heart RNA Structure For First Time - My Blog - Los Alamos Daily Post

MicroCures Awarded $1.5M SBIR Grant To Support Development of Novel Therapeutic Platform for Accelerated Tissue Repair – BioSpace

Funding to Support Ongoing Advancement of siFi2, Lead Candidate from Companys First-of-its-Kind Platform for Precisely Controlling Core Cell Migration Mechanisms

New York, NY, January 7, 2020 MicroCures, a biopharmaceutical company developing novel therapeutics that harness the bodys innate regenerative mechanisms to accelerate tissue repair, today announced that it has been awarded a Phase 2 Small Business Innovation Research (SBIR) grant from the National Institutes of Health (NIH). The two-year, $1.5 million award will support ongoing development of the companys lead product candidate, siFi2. siFi2, a small interfering RNA (siRNA) therapeutic that can be applied topically, is designed to enhance recovery after trauma. This Phase 2 grant continues the companys successful Phase 1 SBIR contract which demonstrated significantly improved repair of burn wounds following treatment with siFi2 in animal models.

MicroCures technology is based on foundational scientific research at Albert Einstein College of Medicine regarding the fundamental role that cell movement plays as a driver of the bodys innate capacity to repair tissue, nerves, and organs. The company has shown that complex and dynamic networks of microtubules within cells crucially control cell migration, and that this cell movement can be reliably modulated to achieve a range of therapeutic benefits. Based on these findings, the company has established a first-of-its-kind proprietary platform to create siRNA-based therapeutics capable of precisely controlling the speed and direction of cell movement by selectively silencing microtubule regulatory proteins (MRPs).

The company has developed a broad pipeline of therapeutic programs with an initial focus in the area of tissue, nerve and organ repair. Unlike regenerative medicine approaches that rely upon engineered materials or systemic growth factor/stem cell therapeutics, MicroCures technology directs and enhances the bodys inherent healing processes through local, temporary modulation of cell motility. The companys lead drug candidate, siFi2, is a topical siRNA-based treatment designed to silence the activity of Fidgetin-Like 2 (FL2), a fundamental MRP, within an area of wounded tissue. In doing so, the therapy temporarily triggers accelerated movement of cells essential for repair into an injury area. Importantly, based on its topical administration, siFi2 can be applied early in the treatment process as a supplement to current standard of care.

We are grateful for NIHs continued support of our work through this multi-year Phase 2 SBIR grant. This non-dilutive financial support allows us to continue building a robust portfolio of preclinical data in animal models that demonstrate the therapeutic potential of siFi2 to significantly improve and accelerate healing of burn wounds, said David Sharp, Ph.D., co-founder and chief science officer of MicroCures. This funding will help advance our research as we work towards first-in-human clinical trial in 2020.

The initial Phase 1 SBIR grant from NIH funded preclinical research by MicroCures which demonstrated that treatment with siFi2 accelerated re-epithelization, improved collagen deposit and maturation, and improved quality of healing in a porcine full thickness burn model. Specific findings showed that following eight weeks of treatment, 39% of siFi2-treated wounds were closed as compared to only 11% for control subjects and 0% for placebo. Additionally, siFi2-treated subjects demonstrated a significantly improved rate of healing as measured by epithelial surface measurements as compared to placebo (p = 0.0106) and control (p = 0.0012).

About MicroCures

MicroCures develops biopharmaceuticals that harness innate cellular mechanisms within the body to accelerate and improve recovery after traumatic injury. MicroCures has developed a first-of-its-kind therapeutic platform that precisely controls the rate and direction of cell migration, offering the potential to deliver powerful therapeutic benefits for a variety of large and underserved medical applications.

MicroCures has developed a broad pipeline of novel therapeutic programs with an initial focus in the area of tissue, nerve and organ repair. The companys lead therapeutic candidate, siFi2, targets excisional wound healing, a multi-billion dollar market inadequately served by current treatments. Additional applications for the companys cell migration accelerator technology include dermal burn repair, corneal burn repair, cavernous nerve regeneration, spinal cord regeneration, and cardiac tissue repair. Cell migration decelerator applications include combatting cancer metastases and fibrosis. The company protects its unique platform and proprietary therapeutic programs with a robust intellectual property portfolio including eight issued or allowed patents, as well as eight pending patent applications.

For more information please visit: http://www.microcures.com

Disclaimer: The SBIR Grant (2R44AR070696-02A1) is supported by the NIHs National Institute of Arthritis and Musculoskeletal and Skin Diseases. The content of this press release is solely the responsibility of MicroCures and does not necessarily represent the official views of the NIH.

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MicroCures Awarded $1.5M SBIR Grant To Support Development of Novel Therapeutic Platform for Accelerated Tissue Repair - BioSpace

2020 Exosome Technologies Market Top Companies Analysis Including Capricor Therapeutics Inc, Evox Therapeutics Ltd, ReNeuron Group Plc, Stem Cell…

ReportsnReports Added latest report on Exosome Technologies Market which explores the application of exosome technologies within the pharmaceutical and healthcare industries. Exosomes are small cell-derived vesicles that are abundant in bodily fluids, including blood, urine and cerebrospinal fluid as well as in in vitro cell culture.

These vesicles are being used in a variety of therapeutic applications, including as therapeutic biomarkers, drug delivery systems and therapies in their own right. Research within this area remains in the nascent stages, although a number of clinical trials have been registered within the field. Exosomes have several diverse therapeutic applications, largely centering on stem cell and gene therapy. Exosomes have been identified as endogenous carriers of RNA within the body, allowing for the intercellular transportation of genetic material to target cells.

Download FREE PDF sample of this Report @ https://www.reportsnreports.com/contacts/requestsample.aspx?name=1781607

Top Company Analysis in Exosome Technologies Market Report:-Capricor Therapeutics Inc.Evox Therapeutics LtdReNeuron Group PlcStem Cell Medicine LtdTavec Inc.Codiak Biosciences Inc.Therapeutic Solutions International Inc.ArunA Biomedical Inc.Ciloa

and more..

As such, developers have worked to engineer exosomes for the delivery of therapeutic miRNA and siRNA-based gene therapies. As RNA is highly unstable within the body, a number of different biologic vector systems have been developed to enhance their transport within the circulation, including viruses and liposomes. Similarly, exosomes derived from stem cells have also been identified for their therapeutic applications, particularly in the treatment of cancer and cardiovascular disease. Exosome technologies offer several advantages over existing biologic-based drug delivery systems.

They have a long circulatory half-life as a result of their high stability and ability to avoid breakdown by the mononuclear phagocyte system and reticuloendothelial systems. Moreover, exosomes have several functional properties that favor their use in therapeutic delivery. Exosomes can be engineered to incorporate targeting ligands, allowing them to deliver cargo selectively to cells. Their small size allows them to penetrate the blood-brain barrier for the delivery of central nervous system therapies, whereas in cancer they can accumulate within the tumor via enhanced permeability and retention effects.

Finally, clinical trials have shown relatively large-scale production to be possible and indicate that exosome therapies can be safely administered to humans. Additionally, exosomes are being investigated for their potential as prognostic and diagnostic biomarkers for several different disease indications. Exosomes make good candidates for biomarker research because of two unique characteristics: their presence in various accessible bodily fluids, and their resemblance to their parent cells of origin. R&D in exosome technologies has increased markedly in recent years. This report provides detailed information on the various healthcare applications of exosomes, and assesses the pipeline, clinical trial and company landscapes.

Scope of Exosome Technologies Market Report:

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Reasons to buy Exosome Technologies Market Report:

Table of Contents in this Report:

1 Table of Contents 41.1 List of Tables 61.2 List of Figures 7

2 Exosomes in Healthcare 82.1 Overview of Exosomes 82.2 Drug Delivery Systems 92.2.1 Modified Release Drug Delivery Systems 92.2.2 Targeted Drug Delivery Systems 102.2.3 Liposomes 122.2.4 Viruses 142.2.5 Exosomes 172.3 The Exosome Lifecycle 182.4 Exosomes in Biology 182.5 Exosomes in Medicine 192.5.1 Biomarkers 192.5.2 Vaccines 202.6 Exosomes as a Therapeutic Target 202.7 Exosomes as Drug Delivery Vehicles 212.8 Therapeutic Preparation of Exosomes 212.8.1 Isolation and Purification 222.8.2 Drug Loading 222.8.3 Characterization 232.8.4 Bioengineering 232.8.5 Biodistribution and In Vivo Studies 232.8.6 Advantages of Exosome Therapies 242.8.7 Disadvantages of Exosome Therapies 242.9 Exosomes in Therapeutic Research 252.9.1 Exosome Gene Therapies 252.9.2 Exosome in Stem Cell Therapy 262.10 Exosomes in Oncology 272.10.1 Immunotherapy 272.10.2 Gene Therapy 282.10.3 Drug Delivery 292.10.4 Biomarkers 302.11 Exosomes in CNS Disease 302.11.1 Tackling the Blood-Brain Barrier 302.11.2 Exosomes in CNS Drug Delivery 312.11.3 Gene Therapy 322.12 Exosomes in Other Diseases 332.12.1 Cardiovascular Disease 332.12.2 Metabolic Disease 33

3 Assessment of Pipeline Product Innovation 363.1 Overview 363.2 Exosome Pipeline by Stage of Development and Molecule Type 363.3 Pipeline by Molecular Target 373.4 Pipeline by Therapy Area and Indication 383.5 Pipeline Product Profiles 383.5.1 AB-126 ArunA Biomedical Inc. 383.5.2 ALX-029 and ALX-102 Alxerion Biotech 393.5.3 Biologics for Autism Stem Cell Medicine Ltd 393.5.4 Biologic for Breast Cancer Exovita Biosciences Inc. 393.5.5 Biologics for Idiopathic Pulmonary Fibrosis and Non-alcoholic Steatohepatitis Regenasome Pty 393.5.6 Biologic for Lysosomal Storage Disorder Exerkine 393.5.7 Biologics for Prostate Cancer Cells for Cells 403.5.8 CAP-2003 Capricor Therapeutics Inc. 403.5.9 CAP-1002 Capricor Therapeutics Inc. 413.5.10 CIL-15001 and CIL-15002 Ciloa 423.5.11 ExoPr0 ReNeuron Group Plc 423.5.12 MVAX-001 MolecuVax Inc. 433.5.13 Oligonucleotides to Activate miR124 for Acute Ischemic Stroke Isfahan University of Medical Sciences 443.5.14 Oligonucleotides to Inhibit KRAS for Pancreatic Cancer Codiak BioSciences Inc. 443.5.15 Proteins for Neurology and Proteins for CNS Disorders and Oligonucleotides for Neurology Evox Therapeutics Ltd 443.5.16 TVC-201 and TVC-300 Tavec Inc. 45

4 Assessment of Clinical Trial Landscape 484.1 Interventional Clinical Trials 484.1.1 Clinical Trials by Therapy Type 484.1.2 Clinical Trials by Therapy Area 494.1.3 Clinical Trials by Stage of Development 504.1.4 Clinical Trials by Start Date and Status 504.2 Observational Clinical Trials 514.2.1 Clinical Trials by Therapy Type 514.2.2 Clinical Trials by Therapy Area 514.2.3 Clinical Trials by Stage of Development 524.2.4 Clinical Trials by Start Date and Status 534.2.5 List of All Clinical Trials 54

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2020 Exosome Technologies Market Top Companies Analysis Including Capricor Therapeutics Inc, Evox Therapeutics Ltd, ReNeuron Group Plc, Stem Cell...

Gene editing breakthroughs that cured genetic diseases in 2019 – The Star Online

IN the summer of 2019, a mother in Nashville, Tennessee in the United States, with a seemingly incurable genetic disorder finally found an end to her suffering by editing her genome.

Victoria Grays recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research gene therapy.

I have hoped for a cure since I was about 11, the 34-year-old said.

Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency.

Over several weeks, Grays blood was drawn so that doctors could get to the cause of her illness stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 pronounced Crisper a new tool informally known as a molecular scissors.

The genetically-edited cells were transfused back into Grays veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary, but theoretically, she has been cured.

This is one patient. This is early results. We need to see how it works out in other patients, said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

But these results are really exciting.

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease beta thalassemia.

She had previously needed 16 blood transfusions per year. Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified. But Crispr, invented in 2012, made gene editing more widely accessible.

It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

Its all developing very quickly, said French geneticist Emmanuelle Charpentier, one of Crisprs inventors and the co-founder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Gene cures

Crispr was the latest breakthrough in a year of great strides in gene therapy, a medical adventure that started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practising the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not, such as making normal red blood cells in Grays case or making tumour-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union.

They join several other gene therapies bringing the total to eight approved in recent years to treat certain cancers and an inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

Twenty-five, 30 years, thats the time it had to take, he said. It took a generation for gene therapy to become a reality. Now, its only going to go faster.

Just outside Washington, at the US National Institutes of Health (NIH), researchers are also celebrating a breakthrough period.

We have hit an inflection point, said US NIHs associate director for science policy Carrie Wolinetz.

These therapies are exorbitantly expensive, however, costing up to US$2 million (RM8.18 million) meaning patients face grueling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion and fighting a general infection.

You cannot do this in a community hospital close to home, said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to Massachusetts Institute of Technology (MIT) researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

In this Oct 10, 2018, photo, He speaks during an interview at his laboratory in Shenzhen, China. The scientist was recently sentenced to three years in prison for practicing medicine illegally and fined 3 million yuan (RM1.76 million). AP

Bioterrorism potential

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who dont necessarily share the medical ethics of Western medicine.

In 2018 in China, scientist He Jiankui triggered an international scandal and his excommunication from the scientific community when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA (deoxyribonucleic acid) of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV (human immunodeficiency virus), even though there was no specific reason to put them through the process.

That technology is not safe, said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr scissors often cut next to the targeted gene, causing unexpected mutations.

Its very easy to do if you dont care about the consequences, he added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species, e.g. malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesnt believe in the more dystopian scenarios predicted for gene therapy, including American biohackers injecting themselves with Crispr technology bought online.

Not everyone is a biologist or scientist, she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies crops?

Charpentier thinks that technology generally tends to be used for the better.

Im a bacteriologist -- weve been talking about bioterrorism for years, she said. Nothing has ever happened. AFP Relaxnews

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Gene editing breakthroughs that cured genetic diseases in 2019 - The Star Online

BrainStorm Cell Therapeutics Wins 2020 ‘Buzz of BIO’ Award for ALS Investigational Therapy – ALS News Today

For its promising investigational therapeutic approach to neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), BrainStorm Cell Therapeutics is theBuzz of BIO 2020 winnerin the Public Therapeutic Biotech category.

The Buzz of BIO contest identifies U.S. companies with groundbreaking, early-stage potential to improve lives. The event also is anopportunity to make investor connections that could take products to the next phase.

Ten biotechnology companies are nominated in each of the three categories ofBuzz of BIO: Public Therapeutic Biotech, Private Therapeutic Biotech, and Diagnostics and Beyond. In the Public Therapeutic Biotech category that BrainStorm won, nominated companies must be actively developing a publicly traded human treatment intended for review by theU.S. Food and Drug Administration (FDA).

As a developer of autologous cellular therapies treatments that use a patients own cells and tissues for debilitating neurodegenerative diseases, BrainStorm is now testing its NurOwn therapy for safety and effectiveness. The treatment involves extracting, from human bone, marrow-derived mesenchymal stem cells (MSCs), which are capable of differentiating into other cell types. The MSCs are then matured into a specific cell type that produces neurotrophic factors compounds that promote nervous tissue growth and survival. They are then reintroduced to the body via injection into muscles and/or the spinal canal.

Backed by a California Institute for Regenerative Medicine grant, Brainstorm has fully enrolledits randomized, double-blind, placebo-controlled Phase 3 clinical trial (NCT03280056) at six U.S. sites in California, Massachusetts, and Minnesota. Some 200 ALS patients are participating. A secondary safety analysis by the trials independent Data Safety Monitoring Board (DSMB) revealed no new concerns. Every two months, study subjects will be given three injections into the spinal canal of either NurOwn or placebo.

The trial is expected to conclude late this year. Results will be announced shortly afterward.

In a Phase 2 study (NCT02017912), which included individuals with rapidly progressing ALS, NurOwn demonstrated a positive safety profile as well as prospective efficacy.

The use of autologous MSC cells to potentially treat ALS was given orphan drug status by both the FDA and the European Medicines Agency.

Thanks to everyone who voted for BrainStorm during the Buzz of BIO competition,Chaim Lebovits, BrainStorm president and CEO, said in a press release. The entire management team at BrainStorm was very pleased with the results of this competition, and we look forward to presenting to an audience of accredited investors who may benefit from the companys story. We thank the BIO[Biotechnology Innovation Organization] team for singling out BrainStorms NurOwn as a key technology with the potential to improve lives.

As a contest winner, BrainStorm is invited to givea presentation at theBio CEO & Investor Conference, to be held Feb. 1011 in New York City, along with exposure to multiple industry elites and potential investors.

NurOwn cells also are being tested in a Phase 2 clinical study (NCT03799718) in patients with progressive multiple sclerosis.

Mary M. Chapman began her professional career at United Press International, running both print and broadcast desks. She then became a Michigan correspondent for what is now Bloomberg BNA, where she mainly covered the automotive industry plus legal, tax and regulatory issues. A member of the Automotive Press Association and one of a relatively small number of women on the car beat, Chapman has discussed the automotive industry multiple times of National Public Radio, and in 2014 was selected as an honorary judge at the prestigious Cobble Beach Concours dElegance. She has written for numerous national outlets including Time, People, Al-Jazeera America, Fortune, Daily Beast, MSN.com, Newsweek, The Detroit News and Detroit Free Press. The winner of the Society of Professional Journalists award for outstanding reporting, Chapman has had dozens of articles in The New York Times, including two on the coveted front page. She has completed a manuscript about centenarian car enthusiast Margaret Dunning, titled Belle of the Concours.

Total Posts: 6

Ins holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in blood vessel biology, blood stem cells, and cancer. Before that, she studied Cell and Molecular Biology at Universidade Nova de Lisboa and worked as a research fellow at Faculdade de Cincias e Tecnologias and Instituto Gulbenkian de Cincia. Ins currently works as a Managing Science Editor, striving to deliver the latest scientific advances to patient communities in a clear and accurate manner.

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BrainStorm Cell Therapeutics Wins 2020 'Buzz of BIO' Award for ALS Investigational Therapy - ALS News Today

Cell and Advanced Therapies Supply Chain Management Industry Report, 2019-2030 – GlobeNewswire

Dublin, Jan. 03, 2020 (GLOBE NEWSWIRE) -- The "Cell and Advanced Therapies Supply Chain Management Market, 2019-2030: Focus on Technological Solutions" report has been added to ResearchAndMarkets.com's offering.

Cell and Advanced Therapies Supply Chain Management Market: Focus on Technological Solutions, 2019-2030 report features an extensive study of the growing supply chain management software solutions market.

The focus of this study is on software systems, including cell orchestration platforms (COP), enterprise manufacturing systems (EMS), inventory management systems (IMS), laboratory information management systems (LIMS), logistics management systems (LMS), patient management systems (PMS), quality management systems (QMS), tracking and tracing software (TTS), and other such platforms that are being used to improve / optimize various supply chain-related processes of cell and advanced therapies.

One of the key objectives of the report was to understand the primary growth drivers and estimate the future size of the supply chain management software solutions market. Based on multiple parameters, such as number of cell and advanced therapies under development, expected pricing, likely adoption rates, and potential cost saving opportunities from different software systems, we have developed informed estimates of the evolution of the market, over the period 2019-2030.

In addition, we have provided the likely distribution of the current and forecasted opportunity across:

Advanced therapy medicinal products, such as cell and gene therapies, have revolutionized healthcare practices. The introduction of such treatment options has led to a paradigm shift in drug development, production and consumption. Moreover, such therapies have actually enabled healthcare providers to treat several difficult-to-treat clinical conditions.

In the past two decades, more than 30 such therapy products have been approved; recent approvals include Zolgensma (2019), RECELL System (2018), AmnioFix (2018), EpiFix (2018), EpiBurn (2018), Alofisel (2018), LUXTURNA (2017), Yescarta (2017), and Kymriah (2017). Further, according to a report published by The Alliance for Regenerative Medicine in 2019, more than 1,000 clinical trials are being conducted across the globe by over 900 companies.

In 2018, around USD 13 billion was invested in this domain, representing a 73% increase in capital investments in this domain, compared to the previous year. It is worth highlighting that, based on an assessment of the current pipeline of cell therapies and the historical clinical success of such products, it is likely that around 10-20 advanced therapies are approved by the US FDA each year, till 2025.

The commercial success of cell and advanced therapies is not only tied to whether they are capable of offering the desired therapeutic benefits, but also on whether the developers are able to effectively address all supply chain requirements. The advanced therapy medicinal products supply chain is relatively more complex compared to the conventional pharmaceutical supply chain. As a result, there are a number of risks, such as possible operational inefficiencies, capacity scheduling concerns, process delays leading to capital losses, and deliverable tracking-related issues, which need to be taken into consideration by therapy developers.

This has generated a need for bespoke technological solutions, which can be integrated into existing processes to enable the engaged stakeholders to oversee and manage the various aspects of the cell and advanced therapies supply chain, in compliance to global regulatory standards. Over the years, several innovative, software-enabled systems, offering supply chain orchestration and needle-to-needle traceability, have been developed.

The market has also recently witnessed the establishment of numerous partnerships, most of which are agreements between therapy developers and software solutions providers. Further, given the growing demand for cost-effective personalized medicinal products, and a myriad of other benefits of implementing such software solutions, the niche market is poised to grow significantly in the foreseen future.

Amongst other elements, the report features:

In order to account for the uncertainties associated with some of the key parameters and to add robustness to our model, we have provided three market forecast scenarios portraying the conservative, base and optimistic tracks of the industry's evolution.

The opinions and insights presented in this study were influenced by discussions conducted with several stakeholders in this domain. The report features detailed transcripts of interviews held with the following individuals:

Key Topics Covered

1. PREFACE1.1. Scope of the Report1.2. Research Methodology1.3. Chapter Outlines

2. EXECUTIVE SUMMARY

3. INTRODUCTION3.1. Context and Background3.2. An Introduction to Cell and Advanced Therapies3.2.1. Classification of Advanced Therapy Medicinal Products3.2.2. Current Market Landscape3.3. Cell and Advanced Therapies Supply Chain3.3.1. Key Processes3.3.2. Challenges Associated with the Cell and Advanced Therapies Supply Chain3.4. Software Solutions for Cell and Advanced Therapies Supply Chain Management3.4.1. Cell Orchestration Platform3.4.2. Enterprise Manufacturing System3.4.3. Inventory Management System3.4.4. Laboratory Information Management System3.4.5. Logistics Management System3.4.6. Patient Management System3.4.7. Quality Management System3.4.8. Tracking and Tracing System3.5. Growth Drivers and Roadblocks3.6. Emergence of Digital Technologies in Supply Chain Management3.6.1. Blockchain Technology3.6.2. Internet of Things3.6.3. Augmented Reality3.6.4. Big Data Analytics3.6.5. Artificial Intelligence

4. CURRENT MARKET LANDSCAPE4.1. Chapter Overview4.2. Cell and Advanced Therapies Supply Chain Management: Overall Market Landscape4.2.1. Analysis by Type of Software Solution4.2.2. Analysis by Key Specification and Benefit4.3.3. Analysis by Application4.3.4. Analysis by End User4.3.5. Analysis by Mode of Deployment4.3.6. Analysis by Scale of Management4.3.7. Analysis by Regulatory Certifications / Accreditations4.3. Cell and Advanced Therapies Supply Chain Management: Developer Landscape4.2.1. Analysis by Year of Establishment4.2.2. Analysis by Location of Headquarters4.2.3. Analysis by Size of Company4.3.4. Analysis by Support Services Offered4.3.5. Leading Developers: Analysis by Number of Software Solutions

5. COMPANY COMPETITIVENESS ANALYSIS5.1. Chapter Overview5.2. Methodology5.3. Assumptions and Key Parameters5.4. Competitiveness Analysis: Overview of Supply Chain Management Software Solution Providers5.4.1. Small-sized Companies5.4.2. Mid-sized Companies5.4.3. Large Companies

6. CORE SUPPLY CHAIN MANAGEMENT SOFTWARE SOLUTIONS: COMPANY PROFILES6.1. Chapter Overview6.2. Brooks Life Sciences6.2.1. Company Overview6.2.2. Financial Information6.2.3. BiobankPro: Software Description6.2.4. Recent Developments and Future Outlook6.3. Cryoport6.3.1. Company Overview6.3.2. Financial Information6.3.3. Cryoportal: Software Description6.3.4. Recent Developments and Future Outlook6.4. MasterControl6.4.1. Company Overview6.4.2. MasterControl Platform: Software Description6.4.3. Recent Developments and Future Outlook6.5. SAP6.5.1. Company Overview6.5.2. Financial Information6.5.3. SAP S/4HANA: Software Description6.5.4. Recent Development and Future Outlook6.6. Savsu Technologies6.6.1. Company Overview6.6.2. Financial Information6.6.3. evo Cold Chain 2.0: Software Description6.6.4. Recent Development and Future Outlook6.7. TraceLink6.7.1. Company Overview6.7.2. Financial Information6.7.3. Digital Supply Chain Platform: Software Description6.7.4. Recent Developments and Future Outlook

7. CELL ORCHESTRATION PLATFORMS: EMERGING TRENDS AND PROFILES OF KEY PLAYERS7.1. Chapter Overview7.2. Supply Chain Orchestration Platforms7.2.1. Key Functions of Supply Chain Orchestration Platforms7.2.2. Advantages of Supply Chain Orchestration Platforms7.2.3. Supply Chain Orchestration Platform Implementation Strategies7.3. Supply Chain Orchestration Platform: Trends on Twitter7.3.1. Scope and Methodology7.3.2. Historical Trends in Volume of Tweets7.3.3. Popular Keywords7.4. Key Industry Players7.4.1. Be The Match BioTherapies7.4.2. Clarkston Consulting7.4.3. Haemonetics7.4.4. Hypertrust Patient Data Care7.4.5. Lykan Bioscience7.4.6. MAK-SYSTEM7.4.7. sedApta Group7.4.8. Stafa Cellular Therapy7.4.9. Title 21 Health Solutions7.4.10. TrakCel7.4.11. Vineti

8. FUNDING AND INVESTMENT ANALYSIS8.1. Chapter Overview8.2. Types of Funding8.3. Cell and Advanced Therapies Supply Chain Management: Recent Funding Instances8.3.1. Analysis by Number of Funding Instances8.3.2. Analysis by Amount Invested8.3.3. Analysis by Type of Funding8.3.4. Analysis by Number of Funding Instances and Amount Invested across Different Software Solutions8.3.5. Most Active Players: Analysis by Amount Invested8.3.6. Most Active Investors: Analysis by Participation8.3.7. Geographical Analysis by Amount Invested8.4. Concluding Remarks

9. PARTNERSHIPS AND COLLABORATIONS9.1. Chapter Overview9.2. Partnership Models9.3. Cell and Advanced Therapies Supply Chain Management: Recent Collaborations and Partnerships9.3.1. Analysis by Year of Partnership9.3.2. Analysis by Type of Partnership9.3.3. Analysis by Partner's Focus Area9.3.4. Analysis by Type of Software Solution9.3.5. Most Active Players: Analysis by Number of Partnerships9.3.6. Analysis by Regions

10. PLATFORM UTILIZATION USE CASES10.1. Chapter Overview10.2. Cell and Advanced Therapies Supply Chain Management: Recent Platform Utilization Use Cases10.2.1. Analysis by Year of Utilization10.2.2. Analysis by User's Focus Area10.2.3. Analysis by Type of Software Solution10.2.4. Most Active Players: Analysis by Number of Utilization Instances10.2.5. Most Active Players: Regional Analysis by Number of Utilization Instances

11. VALUE CHAIN ANALYSIS11.1. Chapter Overview11.2. Cell and Advanced Therapies Value Chain11.2. Cell and Advanced Therapies Value Chain: Cost Distribution11.3.1. Donor Eligibility Assessment11.3.2. Sample Collection11.3.3. Manufacturing11.3.4. Logistics11.3.5. Patient Verification and Treatment Follow-up

12. STAKEHOLDER NEEDS ANALYSIS12.1. Chapter Overview12.2. Cell and Advanced Therapies Supply Chain Management: Needs of Different Stakeholders12.2.1. Comparison of Stakeholder Needs

13. COST SAVINGS ANALYSIS13.1. Chapter Overview13.2. Key Assumptions and Methodology13.3. Overall Cost Saving Potential of Supply Chain Management Software Solutions, 2019-203013.3.1. Cost Saving Potential in Donor Eligibility Assessment, 2019-203013.3.2. Cost Saving Potential in Sample Collection, 2019-203013.3.3. Cost Saving Potential in Manufacturing, 2019-203013.3.4. Cost Saving Potential in Logistics, 2019-203013.3.5. Cost Saving Potential in Patient Verification and Treatment Follow-up, 2019-2030

14. MARKET FORECAST14.1. Chapter Overview14.2. Key Assumptions and Forecast Methodology14.3. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market, 2019-203014.3.1. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by Application14.3.2. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by End User14.3.3. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by Type of Software Solution14.3.4. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by Mode of Deployment14.3.5. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by Geography14.4. Overall Cell and Advanced Therapies Supply Chain Management Solutions Market: Distribution by Application, Type of Software Solution and Mode of Deployment14.4.1. Cell and Advanced Therapies Supply Chain Management Solutions Market for Donor Eligibility Assessment, 2019-203014.4.2. Cell and Advanced Therapies Supply Chain Management Solutions Market for Sample Collection, 2019-203014.4.3. Cell and Advanced Therapies Supply Chain Management Solutions Market for Manufacturing, 2019-203014.4.4. Cell and Advanced Therapies Supply Chain Management Solutions Market for Logistics, 2019-203014.4.5. Cell and Advanced Therapies Supply Chain Management Solutions Market for Patient Verification and Treatment Follow-up, 2019-2030

15. EXECUTIVE INSIGHTS15.1. Chapter Overview15.2. Thermo Fisher Scientific15.2.1. Company Snapshot15.2.2. Interview Transcript: Bryan Poltilove, Vice President and General Manager15.3. Cell and Gene Therapy Catapult15.3.1. Company Snapshot15.3.2. Interview Transcript: Jacqueline Barry, Chief Clinical Officer15.4. McKesson15.4.1. Company Snapshot15.4.2. Interview Transcript: Jill Maddux, Director, Cell and Gene Therapy Product Strategy, and Divya Iyer, Senior Director, Corporate Strategy and Business Development15.5. TrakCel15.5.1. Company Snapshot15.5.2. Interview Transcript: Martin Lamb, Chief Business Officer

16. CONCLUDING REMARKS16.1. Chapter Overview16.2. Key Takeaways

17. APPENDIX 1: LIST OF ADDITIONAL SUPPLY CHAIN MANAGEMENT SOFTWARE SOLUTIONS

18. APPENDIX 2: TABULATED DATA

19. APPENDIX 3: LIST OF COMPANIES AND ORGANIZATIONS

For more information about this report visit https://www.researchandmarkets.com/r/kw1hkc

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Cell and Advanced Therapies Supply Chain Management Industry Report, 2019-2030 - GlobeNewswire

miRNAs: A Promising Target in the Chemoresistance of Bladder Cancer | OTT – Dove Medical Press

Zhonglin Cai,1,* Fa Zhang,2,* Weijie Chen,3,* Jianzhong Zhang,1 Hongjun Li1

1Department of Urology, Peking Union Medical College Hospital, Peking Union Medical College, Chinese Academy of Medical Sciences, Beijing, Peoples Republic of China; 2Department of Urology, First Hospital of Lanzhou University, Lanzhou, Gansu, Peoples Republic of China; 3Department of Urology, Shanghai Municipal Hospital of Traditional Chinese Medicine, Shanghai Traditional Chinese Medicine University, Shanghai, Peoples Republic of China

*These authors contributed equally to this work

Correspondence: Hongjun LiDepartment of Urology, Peking Union Medical College Hospital, Peking Union Medical College, Chinese Academy of Medical Sciences, No. 1 Shuaifuyuan, Dongcheng District, Beijing 100730, Peoples Republic of ChinaTel +86 139 0117 1724Email lihongjun@pumch.cn

Abstract: Chemotherapy is an important cancer treatment method. Tumor chemotherapy resistance is one of the main factors leading to tumor progression. Like other malignancies, bladder cancer, especially muscle-invasive bladder cancer, is prone to chemotherapy resistance. Additionally, only approximately 50% of muscle-invasive bladder cancer responds to cisplatin-based chemotherapy. miRNAs are a class of small, endogenous, noncoding RNAs that regulate gene expression at the posttranscriptional level, which results in the inhibition of translation or the degradation of mRNA. In the study of miRNAs and cancer, including gastric cancer, prostate cancer, liver cancer, and colorectal cancer, it has been found that miRNAs can regulate the expression of genes related to tumor resistance, thereby promoting the progression of tumors. In bladder cancer, miRNAs are also closely related to chemotherapy resistance, suggesting that miRNAs can be a new therapeutic target for the chemotherapy resistance of bladder cancer. Therefore, understanding the mechanisms of miRNAs in the chemotherapy resistance of bladder cancer is an important foundation for restoring the chemotherapy sensitivity of bladder cancer and improving the efficacy of chemotherapy and patient survival. In this article, we review the role of miRNAs in the development of chemotherapy-resistant bladder cancer and the various resistance mechanisms that involve apoptosis, the cell cycle, epithelial-mesenchymal transition (EMT), and cancer stem cells (CSCs).

Keywords: miRNAs, chemoresistant, bladder cancer, biomarkers, targeted therapy

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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miRNAs: A Promising Target in the Chemoresistance of Bladder Cancer | OTT - Dove Medical Press

China confirms third birth of gene-edited baby; scientists involved get prison terms – WRAL Tech Wire

A Chinese scientist who set off an ethical debate with claims that he had made the worlds first genetically edited babies three, according to a Chinese court was sentenced to three years in prison because of his research, state media, the Associated Press and the New York Times have reported.

He Jiankui, who was convicted of practicing medicine without a license, was also fined 3 million yuan ($430,000) by a court in the southern city of Shenzhen, Chinas official Xinhua News Agency reported on Monday. Two other researchers involved in the project received lesser sentences and fines.

The verdict said the three defendants had not obtained qualification as doctors, pursued fame and profits, deliberately violated Chinese regulations on scientific research, and crossed an ethical line in both scientific research and medicine, according to Xinhua. It also said they had fabricated ethical review documents.

The court also confirmed a third birth, saying the researchers were involved in the births of three gene-edited babies to two women. It said all three scientists pleaded guilty during the trial, which Xinhua reported was closed to the public because of privacy concerns.

Hes declaration made him a pariah among scientists, cast a harsh light on Chinas scientific ambitions and embroiled other U.S. scientists who were connected to He. Although He offered no proof and did not share any evidence or data that definitively proved he had done it, his colleagues had said it was possible that he had succeeded, The New York Times reported.

U.S. scientists who knew of Hes plans came under scrutiny. Hes former academic adviser, Stephen Quake, a star Stanford University bioengineer and inventor, was cleared of any wrongdoing after an investigation into his interaction with his former student. Rice University has been investigating Michael Deem, Hes doctoral adviser, because of allegations that he was actively involved in the project.

Duke engineers improve CRISPR genome editing with biomedical tails

He, the lead researcher, shocked the scientific world when he announced in November 2018 that he had altered the embryos of twin girls who had been born the same month. He described his work in exclusive interviews with The Associated Press.

The announcement sparked a global debate over the ethics of gene editing. He said he had used a tool called CRISPR to try to disable a gene that allows the AIDS virus to enter a cell, in a bid to give the girls the ability to resist the infection. The identity of the children has not been released, and it isnt clear if the experiment succeeded.

The CRISPR tool has been tested elsewhere in adults to treat diseases, but many in the scientific community denounced Hes work as medically unnecessary and unethical, because any genetic changes could be passed down to future generations. The U.S. forbids editing embryos except for lab research.

He, who is known as JK, told the AP in 2018 that he felt a strong responsibility to make an example, and that society would decide whether to allow the practice to go forward. He disappeared from public view shortly after he announced his research at a conference in Hong Kong 13 months ago, apparently detained by authorities, initially in an apartment in Shenzhen, a city in Guangdong province that borders Hong Kong.

It wasnt clear if the three-year prison term includes any of the time he has already spent in Chinese custody.

A Chinese scientist said the sentence should have been harsher to deter others. Kehkooi Kee, a Tsinghua University researcher who conducts gene-editing research on stem cells, also said that He should be held responsible for any fallout from the experiment on the lives of the babies and their families.

Dr. William Hurlbut, a Stanford University bioethicist whose advice He sought for more than a year before his experiment, said he felt sorry for the scientist, his wife and two young daughters.

I warned him things could end this way, but it was just too late, Hurlbut wrote in an email addressed to the AP; the director of the U.S. National Institutes of Health, Dr. Francis Collins; and gene-editing pioneer Jennifer Doudna at the University of California, Berkeley.

Sad story everyone lost in this (JK, his family, his colleagues, and his country), but the one gain is that the world is awakened to the seriousness of our advancing genetic technologies, Hurlbut wrote.

Dr. Eric Topol, who heads the Scripps Research Translational Institute in California, noted its almost unheard of for a scientist to get imprisoned but in this case the sheer recklessness and unethical behavior warranted it. Topol praised China for standing up for proper medical research conduct.

Doudna told the AP she was concerned about the mysterious legal process in China, but she said the sentences are a step toward bringing this case to closure and send a strong message to discourage other such work. (Doudna is paid by the Howard Hughes Medical Institute, which also supports APs Health and Science Department.)

As a scientist, one does not like to see scientists going to jail, but this was an unusual case, Doudna said. Hes work was clearly wrong in many ways.

Before setting up a lab at the Southern University of Science and Technology of China in Shenzhen, He studied in the U.S. The verdict accused him of colluding with Zhang Renli and Qin Jinzhou, who worked at medical institutes in the same province.

Zhang was sentenced to two years in prison and fined 1 million yuan, Xinhua said. Qin received an 18-month prison sentence, but with a two-year reprieve, and a 500,000 yuan fine.

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China confirms third birth of gene-edited baby; scientists involved get prison terms - WRAL Tech Wire