Edge Security: How to Secure the Edge of the Network – eSecurity Planet

Posted December 12, 2019

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Edge computing is a term that is becoming increasingly popular not just as a buzzword but as a way of understanding and modeling IT infrastructure in an era of pervasive cloud computing. With the rise of edge computing has come a need for edge security.

Edge security isn't just about securing edge computing though; it's also potentially a new approach to defining user and enterprise security in the cloud-connected world.

While there are no shortage of meaningless buzzwords in IT, edge computing isn't an abstract concept and neither is edge security. For both edge computing and edge security, there is an emerging set of definitions and standards.

In this eSecurity Planet guide, we look at what edge security is all about and some of the top vendors in the space.

In many modern IT deployments, a data center stands at the core of a network architecture. That data center can be an on-premises, corporate-owned facility, or increasingly the data center can be a collection of public cloud resources.

Edge computing is often defined as computing that happens at the edge of a network. The broader corporate campus, branch offices and retail locations can be considered at the edge of the network, since the core of the network is the data center or cloud. With the emergence of 5G cellular, with base station deployments that integrate powerful compute capabilities, 5G is also considered part of edge computing.

Simply stating that compute that is at the edge of the network is edge computing is not the formal definition, however, at least according to the Open Glossary of Edge Computing, an open source effort led by the Linux Foundation's LF Edge group.

"By shortening the distance between devices and the cloud resources that serve them, and also reducing network hops, edge computing mitigates the latency and bandwidth constraints of today's Internet, ushering in new classes of applications," the glossary explains.

As edge computing is a growing area, so too is edge security. There are several aspects involved in edge security, including:

Perimeter security: Securing access to edge compute resources via encrypted tunnels, firewall and access control

Application security: Beyond the network layer, edge compute devices run applications that must be secured

Threat detection: As edge computing is by definition not centralized, it's critically important for providers to employ proactive threat detection technologies to identify potential issues early

Vulnerability management: There are both known and unknown vulnerabilities that need to be managed

Patching cycles: Automated patching to keep devices up to date is important for reducing the potential attack surface

In 2019, a new term was coined by Gartner to define a category of hardware and services that help enable edge security; that term is Secure Access Service Edge (SASE).

According to Garnter, SASE is an emerging offering combining comprehensive WAN capabilities with comprehensive network security functions, such as secure web gateways (SWG), CASB, firewalls as a service (FWaaS) and zero trust network access (ZTNA), to support the dynamic secure access needs of digital enterprises.

Even though the term SASE is new, in August 2019 Gartner forecast that by 2024, at least 40% of enterprises will have explicit strategies to adopt SASE, up from less than 1% at year-end 2018.

Though the term edge security is relatively new, there are multiple vendors in the space that have product offerings. Not all the vendors listed below fall into the SASE category, as some lack the WAN functionality and only provide a subset of edge security needs.

Akamai was once primarily known as a content delivery network (CDN), with its global distributed network. In recent years, Akamai has expanded significantly into security, with multiple capabilities to help organizations defend against both network and application layer attacks.

The Akamai Intelligent Edge Platform provides what the company refers to as a defensive shield that can surround and protect users from the edge all the way up to full data centers.

Cisco has long been a dominant vendor in the networking market, with an expansive product portfolio that includes both hardware and software.

When it comes to edge security, Cisco is positioning a number of its capabilities as part of a security stack that includes traditional perimeter security as well as cloud-based security controls.

Cloudflare is a global cloud platform that has multiple content and application delivery services as well as security capabilities.

One of the controls needed for edge security is protection against attacks, which is where Cloudflare's DDoS and DNS Protection services can be useful.

Fortinet is a network security vendor that has increasingly been active in the SD-WAN space.

The FortiGate Secure SD-WAN offering can potentially fit in the SASE category of edge security, providing organizations with secure access as well as threat protection for edge computing deployments.

Palo Alto Networks got its start as a network security vendor and has come to fully embrace the SASE model.

The Prisma Access platform integrates Firewall as a Service (FWaaS), threat prevention, DNS security and data loss prevention (DLP) capabilities for edge resource protection.

Cato Networks got started in 2016 with a Security-as-a-Service model for network security, headed by Shlomo Kramer, perhaps best known as the founder of Check Point Software.

Cato Networks has strongly embraced the SASE model and its Cato Cloud provides a global SD-WAN architecture that delivers security services, including FWaaS, SWG, anti-malware, IPS and threat detection capabilities to both the edge and data center use cases.

While VMware is well known for its virtualization technologies, it is now also a strong player in the emerging SASE market as well, thanks to its SD-WAN by VeloCloud platform.

VMware acquired VeloCloud in 2017 and has steadily improved the platform since with increased security capabilities that can help to protect edge computing.

Zscaler has a global clout network that provides security-as-a-service that can be used to create a SASE-style deployment.

The Zscaler Cloud Security platform can provide full content inspection of both inbound and outbound traffic, and also benefits from the integration of threat intelligence feeds to help correlate and block threats in real time.

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Edge Security: How to Secure the Edge of the Network - eSecurity Planet

Three Key Takeaways From AWS re:Invent 2019 – Forbes

AWS re:Invent 2019, the industrys largest cloud computing conference concluded last week. With over 65,000 attendees and 2500 sessions, the scale of the event only grows each year.

Andy Jassy, CEO

Its not just the scale, the pace of innovation at Amazon refuses to slow down. This years re:Invent witnessed more than 100 new features, products, and services added to AWS portfolio.

Here are three key themes that emerged from AWS re:Invent 2019:

1. Redefining Edge Computing

Traditionally, the cloud is considered to be a highly centralized resource, much like an IBM mainframe. But the advent of edge computing has made the cloud truly distributed. Developers dont need to work around the limitations of latency and the performance trade-off involved in deploying applications in remote data centers.

Edge computing is often used either in the context of delivering static content through CDN or moving the processing closer to IoT devices. But, AWS has redefined edge computing in more than one way. It has built a continuum of compute by delivering a new form of edge computing.

First, there are AWS Outposts - hardware appliances that customers can rent from Amazon to run within their data center. Customers can launch Amazon EC2 instances on AWS Outposts, and manage them from the same set of tools such as CloudFormation and AWS Console. Each Outpost deployment is associated with an AWS region that is capable of running managed services such as Amazon RDS and Amazon S3.

If AWS Outposts are confined to the data center, AWS Local Zones make cloud hyper-local by bringing compute, storage, and network services closer to users within a city or a metro. Each AWS Local Zone location is an extension of an AWS Region where customers can deploy latency-sensitive applications using AWS services in geographic proximity to end-users. Developers can now deploy applications across availability zones that span traditional and local zones. The new distributed architecture of cloud opens up additional avenues for developers and businesses building modern applications for AR/VR experiences, smart cities, connected cars and more. The Los Angeles AWS Local Zone is generally available by invitation.

Amazon is expanding the footprint of AWS to telecom providers that offer 5G networks. AWS Wavelength Zones are AWS infrastructure deployments that embed AWS compute and storage services within telecommunications providers data centers at the edge of the 5G network. Wavelength brings the power of the AWS cloud to the network edge to enable latency-sensitive use cases that require near-real-time responses.

From the data center to the metro to the telco infrastructure, AWS is bringing the cloud to new territories. Amazon is certainly redefining edge computing.

2. Investments in Next-Gen Hardware

Amazon is investing in a new breed of chips and hardware that makes AWS infrastructure efficient and cost-effective.

Since the acquisition of Annapurna Labs in 2016, Amazon has been moving software-based heavy lifting to purpose-built hardware. Nitro System, a collection of hardware accelerators, offloads hypervisor, storage, and network to custom chips freeing up resources on EC2 to deliver the best performance.

AWS Graviton2 processors use a combination of hardware from ARM and Nitro System. The new breed of EC2 instances based on Graviton2 uses the Nitro System that features the Nitro security chip with dedicated hardware and software for security functions, and support encrypted EBS storage volumes by default. When compared to the first generation of Graviton processor-based EC2 instances, the current generation of instances delivers 40% price performance.

Amazon has also announced the general availability of Amazon EC2 Inf1 instances, a family of instances exclusively built for machine learning inference applications. Combined with 2nd Gen Intel Xeon Scalable processors and 100Gbps networking, these instances deliver unmatched performance for running applications that depend on machine learning inference. Inf1 instances are powered by AWS Inferentia chips that are purpose-built for accelerating inference.

3. Big Bets on Machine Learning and Artificial Intelligence

Amazon continues to expand the AI and ML portfolio through new services and platform offerings. At re:Invent 2019, AWS announced multiple services that take advantage of ML and AI.

Amazon CodeGuru is a managed service that helps developers proactively improve code quality and application performance through AI-driven recommendations. The service comes with a reviewer and profiler that can detect and identify issues in code. Amazon CodeGuru can review and profile Java code targeting the Java Virtual Machine.

Venturing into enterprise search, AWS has launched Amazon Kendra, a managed service that brings intelligent, contextual search to applications. Amazon Kendra can discover and parse documents stored in a variety of mediums including file systems, web sites, Box, DropBox, Salesforce, SharePoint, RDBMS and Amazon S3.

Amazon Fraud Detector is a service that can identify potentially fraudulent online activities such as online payment fraud and the creation of fake accounts. Based on the experience of fraud detection from Amazon.com, AWS has built this service to help customers integrate sophisticated fraud detection techniques in their applications.

Amazon SageMaker, the platform as a service (PaaS) offering from AWS has got new capabilities that make developers and data scientists productive. Built on top of Jupyter Notebooks, Amazon SageMaker now has a full-blown development environment. Branded as Amazon SageMaker Studio, the new tooling experience includes debugging, pipeline management, integrated deployment, and model monitoring. The platform is tightly integrated with Git and Jupyter Notebooks to bring collaboration among developers.

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Three Key Takeaways From AWS re:Invent 2019 - Forbes

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Would you pay $1M to partake in an anti-aging gene therapy trial? – Fox Business

Fox News senior judicial analyst Judge Andrew Napolitano on the potential fallout from reports DNA-testing company Family TreeDNA will share data with the FBI in an effort to solve crimes.

Libella Gene Therapeutics is charging volunteers $1 million to undergo clinical trials of a treatment it is working on that is designed to prevent, delay or even reverse aging.

However, participants will be required to go to a small clinic in Cartagena, Colombia, to participate, which the Kansas-based company said was the easiest site among eight different countries it looked into, calling it the path of least resistance.

In a press release, a company executive said traditional clinical trials in the U.S. take years and millions or even billions of dollars.

The treatment would be delivered intravascularly and participants will be monitored over the course of a year, according to the company's website. Gene therapy treatments are intended to be one-off treatments, attacking the problem at its source.

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The technology focuses on lengthening telomeres, which are structures found at the end of chromosomes. Their main function is to protect DNA during cell division.

Every time a cell divides, a part of the telomere is lost until it becomes too short and the cell dies. Some believe that as cells age, so does the body.

Telomerase is an enzyme that lengthens telomeres and thus prevents the cell from dying.

Libellas technology rebuilds the ends of telomeres, andthereby affects the aging process.

I know what were trying to do sounds like science fiction, but I believe its a science reality, Jeff Mathis, CEO of Libella Gene Therapeutics, said in an interview with OneZero.

The treatment may potentially treat other diseases, like cancer and Alzheimers.

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Not everyone, however, agrees that lengthening telomeres will have any effect on the aging process. For example, researchers at the University of Utah were unable to conclude whether shorter telomeres were simply a sign of aging or actually a contributor to the process.

Dr. Andrew Stern, who is one of the founders of Libella Gene Therapeutics, was also one of the principal discoverers of portions of human telomerase.

In order to be eligible for the trial, individuals must be 45 years or older. So far the company has recruited two people, according to the OneZero interview published on Medium.

The study will look into the change in the length of telomeres, and into the incidence of serious adverse events.

The FDA declined to comment specifically on Libella Gene Therapeutics and its decision to hold its trial outside of the U.S. It does, however, accept foreign clinical data and results so long as certain conditions are met.

A spokesperson for Libella Gene Therapeutics did not return FOX Business request for comment.

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Would you pay $1M to partake in an anti-aging gene therapy trial? - Fox Business

Pfizer to bring gene therapy production in-house – BioPharma-Reporter.com

Pfizer moved into gene therapies earlier than some of its peers, partnering with Spark Therapeutics in 2014 and paying close to $200m (180m) upfront to acquire Bamboo Therapeutics two years later. The Bamboo takeover gave Pfizer ownership of a manufacturing facility in North Carolina, US.

Earlier this year, Pfizer doubled down on in-house production of gene therapies, committing $500m to expand its footprint in North Carolina.

Talking at a recent investor conference, Mikael Dolsten, chief scientific officer at Pfizer, said the spending commitment is, in part, a reflection of a belief that keeping production in-house will deliver better results than relying on third parties.

Dolsten said, When we compare that with what we get from other companies, we think we can really improve the yield, the purity and the characterization of the product.

Across the industry, poor yields have exacerbated capacity constraints created by the rapid expansion of the gene therapy pipeline, turning quality manufacturing capacity into a sought after resource.

A desire to possess in-house manufacturing capacity was a factor in many of the recent acquisitions of gene therapy companies, such as Astellas $3bn takeover of Audentes Therapeutics.

Gene therapy startups, such as Audentes and Bamboo, bypassed the limitations of contract capacity by establishing internal capabilities. Those capabilities enabled the companies to advance their gene therapies and, ultimately, to attract takeover offers, but their creation required the sort of upfront investments in infrastructure that many venture-backed startups typically try to avoid.

Through its $500m gene therapy investment, Pfizer thinks it can provide an alternative for startups that are struggling to access high-quality contract capacity but are unable or unwilling to build their own facilities.

Dolsten said, We think it's a competitive advantage, not just for our product, but for companies that want to partner with Pfizer that may allow them to have an easier and more high-end dialogue with regulators across the globe about this new field and a new type of product.

If Dolsten is right, the North Carolina manufacturing capacity could give Pfizer an edge when it tries to partner with gene therapy startups that have other options open to them, such as alliances with rival drugmakers and contract manufacturing organizations.

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Pfizer to bring gene therapy production in-house - BioPharma-Reporter.com

Buyer beware of this $1 million gene therapy for aging – MIT Technology Review

Its said that nothing is certain except death and taxes. But doubt has been cast over the former since the 1970s, when scientists picked at the seams of one of the fundamental mysteries of biology: the molecular reasons we get old and die.

The loose thread they pulled had to do with telomeresmolecular timepieces on the ends of chromosomes that shorten each time a cell divides, in effect giving it a fixed life span. Some tissues (such as the gut lining) renew almost constantly, and it was found that these have high levels of an enzyme called telomerase, which works to rebuild and extend the telomeres so cells can keep dividing.

That was enough to win Elizabeth Blackburn, Carol Greider, and Jack Szostak a Nobel Prize in 2009. The obvious question, then, was whether telomerase could protect any cell from agingand maybe extend the life of entire organisms, too.

While telomere-extending treatments in mice have yielded intriguing results, nobody has demonstrated that tweaking the molecular clocks has benefits for humans. That isnt stopping one US startup from advertising a telomere-boosting genetic therapyat a price.

Libella Gene Therapeutics, based in Manhattan, Kansas, claims it is now offering a gene therapy to repair telomeres at a clinic in Colombia for $1 million a dose. The company announced on November 21 that it was recruiting patients into what it termed a pay-to-play clinical trial.

Buyer beware, though: this trial is for an unproven, untested treatment that might even be harmful to your health.

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The company proposes to inject patients with viruses carrying the genetic instructions cells need to manufacture telomerase reverse transcriptase, a molecule involved in extending the length of telomeres.

The dangers are enormous, says Jerry Shay, a world expert on aging and cancer at the University of Texas Southwestern Medical Center. Theres a risk of activating a pre-cancerous cell thats got all the alterations except telomerase, especially in people 65 and over.

For years now, people involved in the company have made shifting claims about the study, raising uncertainty about who is involved, when it might start, and even where it would occur. Trial listings posted in October to clinicaltrials.gov currently show plans for three linked experiments, each with five patients, targeting critical limb ischemia, Alzheimers, and aging, respectively.

Jeff Mathis, president of Libella, told MIT Technology Review that two patients have already paid the enormous fee to take part in the study: a 90-year-old-woman and a 79-year-old man, both US citizens. He said they could receive the gene therapy by the second week of January 2020.

The decision to charge patients a fortune to participate in the study of an experimental treatment is a red flag, say ethics experts. Whats the moral justification for charging individuals with Alzheimers? asks Leigh Turner, at the University of Minnesotas Center for Bioethics. Why charge those bearing all the risk?

The telomere study is occurring outside the US because it has not been approved by the Food and Drug Administration. Details posted to clincaltrials.gov indicate that the injections would be carried out at the IPS Arcasalud SAS medical clinic in Zipaquir, Colombia, 40 kilometers (25 miles) north of Bogot.

It takes a lot longer, is a lot more expensive, to get anything done in the US in a timely fashion, Mathis says of Libellas choice to go offshore.

To some promoters of telomerase gene therapy, urgency is justified. Heres the ethical dilemma: Do you run fast and run the risk of low credibility, or move slowly and have more credibility and global acceptancebut meanwhile people have died? says Mike Fossel, the president of Telocyte, a company planning to run a study of telomerase gene therapy for Alzhheimer's in the US if it can win FDA signoff.

Our reporting revealed a number of unanswered questions about the trial. According to the listings, the principal investigatorwhich is to say the doctor in charge--is Jorge Ulloa, a vascular surgeon rather than an expert in gene transfer. I dont see someone with relevant scientific expertise, says Turner.

Furthermore, Bill Andrews, who is listed as Libellas chief scientific officer, says he does not know who Ulloa is, even though on Libellas website, the mens photos appear together on the list of team members. He said he believed that different doctors were leading the trial.

Turner also expressed concerns about the proposed 10-day observation period described in the posting for the overseas study: If someone pays, shows up, has treatment, and doesnt stick around very long, how are follow-up questions taking place? Where are they taking place?

Companies seeking to try the telomere approach often point to the work of Maria Blasco, a Spanish scientist who reported that telomere-lengthening gene therapy benefited mice and did not cause cancer. Blasco, director of the Spanish National Centre for Cancer Research, says she believes many more studies should be done before trying such a gene experiment on a person.

This isnt the first time Libella has announced that its trial would begin imminently. It claimed in late 2017 that human trials of the telomerase therapy would begin in the next few weeks. In 2016, Andrews (then partnered with biotech startup BioViva) claimed that construction of an age reversal clinic on the island nation of Fiji would be complete before the end of the year. Neither came to pass.

Similar questions surround Libellas most recent claims that it has two paying clients. Pedro Fabian Davalos Berdugo, manager of Arcasalud, said three patients were awaiting treatment in December. But Bioaccess, a Colombian contract research organization facilitating the Libella trial, said that no patients had yet been enrolled.

Also unclear is where Libella is obtaining the viruses needed for the treatment. Virovek, a California biotech company identified by several sources as Libellas manufacturer, did not answer questions about whether any treatment had been produced.

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Buyer beware of this $1 million gene therapy for aging - MIT Technology Review

Dr. James Wilson, a scientific pioneer, on the future of gene therapy – STAT

Dr. James Wilson is a pioneer in gene therapy. That does not mean he is necessarily impressed with the current state of affairs.

In five years, when we look back on the way were executing on gene therapy now, were going to realize that things are going to be very different, Wilson said at the STAT Summit in Cambridge, Mass., recently. The way in which were going to treat Duchenne muscular dystrophy, potentially cure it, is not the way in which its being evaluated in the clinic now.

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Dr. James Wilson, a scientific pioneer, on the future of gene therapy - STAT

The Rise Of Patent Wars In Europe’s Gene Therapy Space – Law360

Law360 (December 11, 2019, 1:28 PM EST) -- The gene therapy industry is in an exciting phase of growth, undergoing significant mergers and acquisitions activity, product sales and new marketing authorizations that are being issued with increasing regularity globally.

Recent reports have estimated that the market is likely to be almost four times its current value by 2025[1], with up to 20 new product approvals expected every year[2].

This rapid growth brings inevitable challenges. Significant issues relating to regulatory standards in manufacturing plants, establishing acceptable reimbursement policies and antitrust investigations are among a few.

The intellectual property landscape has been lower profile, with the exception of the ongoing CRISPR...

In the legal profession, information is the key to success. You have to know whats happening with clients, competitors, practice areas, and industries. Law360 provides the intelligence you need to remain an expert and beat the competition.

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Pfizer cites competitiveness of inhouse gene therapy – Bioprocess Insider – BioProcess Insider

With a surge of gene therapies coming through the clinic and a lack of CDMO capacity, having inhouse capabilities is driving investment and M&A activity.

As increasing numbers of gene therapies progress through the clinic and towards commercialization, it is no secret that demand for production capabilities is high.

The complexity and cost of making viral vector means is a problem, but a biopharma with its own capabilities holds a major advantage, it would appear, something Pfizer CSO Mikael Dolsten claimed at the Evercore ISI 2nd Annual HealthCONx Conference last week.

Image: iStock/syahrir maulana

The Big Biopharma firm has a site in Sanford, North Carolina, supporting its gene therapy pipeline. In August this year, the firm made a $500 million (450 million) investment at the site to construct a manufacturing plant based on its recombinant adeno-associated virus (rAAV) vector platform for gene therapies and viral vaccines.

We think thats a very versatile flexible manufacturing platform, Dolsten said, adding it will be used both for Pfizers own manufacturing needs and for its partners.

Its a competitive advantage, not just for our product, but for companies that wants to partner with Pfizer that may allow them to have an easier and more high-end dialogue with regulators across the globe about this new field and a new type of product.

His comments came the same week that Astellas entered an agreement to buy Audentes for $3 billion. The firm cited a viral vector plant in San Francisco, California as a major driver in the deal which, management said, allows Astellas to gain a competitive advantage in the gene therapy business.

The other option for gene therapy developers is to use contract development and manufacturing organizations (CDMOs). However, the relatively little capacity available means gene therapy developers are having to reserve space years in advance and be subject to high-demand, low-supply price pressures.

But it is helping to shape the sector as third-parties look to acquisitions and expansions to take advantage of the demand. Thermo Fisher and Catalent have both bought their way into the market in the past year, buying Brammer Bio and Paragon, respectively.

Meanwhile, CDMOs like Fujifilm, Aldevron, and Viralgen have all invested in their capacity to help feed demand within the past few months.

Some CDMOs, have done both: Lonza, for example, acquired Dutch firm PharmaCell in 2017 and has built out its own capabilities in Pearland, Texas.

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Pfizer cites competitiveness of inhouse gene therapy - Bioprocess Insider - BioProcess Insider

Investment in UK cell and gene therapy manufacturing set to continue – European Pharmaceutical Review

An additional 6,000m2 of cell and gene therapy manufacturing space is expected to become available within the next 12 months, according to new data.

New data shows that a 60 percent increase in cell and gene manufacturing space reported in 2018 has since become fully functional, reports Cell and Gene Therapy Catapult (CGT Catapult).

This growth is also reflected in a surge in jobs in the cell and gene therapy manufacturing industry; numbers have expanded from 150 in 2014, to 500 in 2018 and doubling to over 1,000 staff in 2019, found the researchers.

With significant additional capacity planned, it is essential that skilled personnel are available

The manufacturing capacity in the UK is due to increase even further in 2020, with around 6,000m2 becoming available. Oxford Biomedica and the CGT Catapult are the main contributors, with 4,200m2 opening at Oxford Biomedicas new Oxbox facility and a 1,200m2 expansion of the CGT Catapult. Additional space is set to come from expansions at Great Ormond Street, Cobra Biologics, The Rayne Cell Therapy facility at Kings College London, NHS Blood and Transplant (NHSBT) and Scottish National Blood Transfusion Service (SNBTS).

Further extensions are also expected in 2021 from RoslinCT and in 2022 from The University of Birmingham and the Rayne Cell Therapy Facility at Kings College London. With more plans likewise being announced by Karoo Therapeutics, Exmoor Pharma, University of Oxford CBF, MeiraGTx and Immetacyte, cell and gene therapy manufacturing growth is set to continue.

These facilities coming on stream now are evidence of the acceleration of cell and gene therapies towards commercialisation. With significant additional capacity planned, it is essential that skilled personnel are available. The Advanced Therapies Apprenticeship Community, designed specifically to train and upskill individuals for the sector, setup in partnership with the Medicines Manufacturing Industry Partnership (MMIP), will become increasingly important, claimed Keith Thompson, Chief Executive Officer CGT Catapult.

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Investment in UK cell and gene therapy manufacturing set to continue - European Pharmaceutical Review

Biomanufacturing and Supply Chain Standardization Key to Success in Cell and Gene Therapy Industry Boom – BioBuzz

The Wild West. Like changing the engine of acar while driving down the highway.

This was how several cell and gene therapy industry leaders characterized the fields rapidly developing Biomanufacturing and supply chain environment at this years Maryland Tech Council (MTC) BIO Conference held in the BioHealth Capital Region (BHCR).

Cell and gene therapy is so new that itsmanufacturing and supply chain processes and best practices are stillcalcifying, leaving many organizations to learn on the fly as they attempt tobuild the efficiency and standardization necessary for the industry to trulytake off.

Put simply, cell and gene therapy companiesare doing something thats basically never been done before. Only a handful ofcompanies have successfully taken a cell and gene therapy product to market.Gene and cell therapy manufacturing and supply chain is truly a new frontierthat is just starting to be explored and mapped.

Earlier this year at the Bio Innovation Conference during a panel entitled, New Frontiers of Biomanufacturing, had a vigorous discussion about the evolving state of cell and gene therapy manufacturing and innovation. Panelists included Vigenes Chief Manufacturing Officer Jeffrey Hung, Ph.D.; Aaron Vernon, VP of Engineering and Supply Chain at Autolus; John Rowley, Ph.D., Founder, and Chief Product Officer at Rooster Bio; Chris McDonald, VP of Manufacturing at Kite Pharma and Robert Lindblad, Chief Medical Officer at Emmes Corporation, and was moderated by John Walker, Manufacturers Extension Liaison at NIIMBL (National Institute for Innovation in Manufacturing Biopharmaceuticals).

When asked, What keeps you up at night?,Jeffrey Hung of Vigene perfectly captured the conundrum facing those operatingin the cell and gene therapy field: What keeps me awake at night is thecontinued demand for clinical trial materials and commercial product while wekeep having to innovate at a fast pace. Its like trying to change a carsengine while driving it down the highway.

Other panelists cited what seemed likeinherent contradictions faced by a nascient cell and gene therapy industry. Inessence, these companies are learning on the fly without an established set ofrules to follow or even question. Production needs conflict with innovation;personalization is anathema to standardization; and being cutting edge oftenmeans they lack the tools, materials and the well-worn paths to regulatoryapproval already established in other biotech sectors.

As a supporter of manufacturerswhat we see is that everyone wants to innovate but at some point, you have to just bite the bullet and lock down your process to get reliable manufacturing techniques to move it along the regulatory pathway. Every tweak you do requires a lot of other work. You can work on innovation in the second stage of your product, stated Robert Lindblad of Emmes.

In the new frontier space, there are no reagents and no GMP reagents. You cant source GMP reagents so you have to qualify reagents just for your product and your indication, which is not adequate to get a certificate of analysis from the FDA. As you are on the cutting edge, you dont have the equipment to create a closed system, you dont have the reagents you need to have GMP manufacturing, so you have to be creative and work with the agencies to get through the regulatory pathways to commercialization, he added.

The personalized nature of cell and genetherapy also creates challenges for manufacturing standardization and supplychain. The one batch, one patient equation of autologous cell therapy makes ita unique and highly challenging manufacturing process.

When you think about designing and building aplant you cant build inventory. Biologic manufacturing allows for 2 or 3 yearsof inventory. For us, you can never take the plant down, stated Chris McDonaldof Kite Pharma. He added that in many ways building a cell therapymanufacturing plant is a lot easier than running one due to the challengespresented by personalization, constant production, lack of inventory and theoverall newness of the industry.

Rooster Bio has built its business model around solving some of the fields efficiency and standardization issues. Rooster is making great strides in its efforts to standardize parts of the manufacturing and supply chain processes by becoming the Intel of cell banking. By creating off-the-shelf, high-quality media and cells-similar in concept to what the Intel microchip did for the computer industry-Rooster hopes to help standardize an important segment of cell and gene therapy manufacturing process and supply chain, thereby increasing manufacturing efficiency while lowering the cost of cell and gene therapy costs to patients.

One bad reagent going into a cell bank thats supposed to last for a few years can be really debilitating. This is what makes the Rooster Bio business model possible. Innovation cant happen without quality. On the innovation side were in the middle of the process of living cells transitioning from being just a tool for research into technology itself, stated John Rowley of Rooster Bio.

He also cited Moores Law as an apt parallel for whats currently developing in the cell and gene therapy field right now. Moores Law states that the capacity of microchips would likely double every year while computers would decrease in cost. Rowley drew a link between Moores Law, the rapid increase in monoclonal antibody manufacturing capacity and cost reductions of the 1990s and what is going on now in cell and gene therapy manufacturing and supply chain.

While improving the manufacturing and supply chain is critical, Aaron Vernon of Autolus reminded the audience of the real-world impact of cell and gene therapy development failure or success. He emphasized the need for stronger cell and gene therapy manufacturing and supply standardization because of the direct link between personalized therapies and impacts on individual patients.

We have to have zero tolerance for manufacturing failures because of their direct impact on patients. There are a lot of moving parts and things get more complex over time. This doesnt scale easily, he stated. We want to innovate all the time but we dont decisions made early in the research process that hamper supply chain for a very long time.

Because personalized medicine is tailored for specific patients-i.e. one batch, one patient-the stake, while always high in biotech manufacturing, are higher in cell and gene therapy manufacturing and supply chain.

This makes solving the industrysmanufacturing and supply chain questions even more pressing. Having morecompanies successfully commercialize their cell and gene therapy products andincreased information sharing, even among competing companies, are critical tothe industrys future.

Theres a huge amount of knowledge out therebut theres a black box that only gives us information about whats workedand what hasnt really late in the game. We only learn from the FDA after thefact, stated McDonald.

Instead of relying on the FDA, Walker wonderedabout the possibility of sharing successes and failures among cell and genetherapies competing for market share.

Walker offered the following thoughts to thepanel, Different companies know whats working and whats not but because ofIP no one is sharing. As cell therapy is trying to move forward everyone istrying to protect their own space so they are not sharing failures. If youretrying to move the field forward scientifically thats one thing, but right noweveryone is thinking commercially and everyone is in their silo, which istotally understandable

Vernon noted that the Standards Coordinating Body and other organizations are working to develop manufacturing and supply chain standards for the industry and are actively seeking input from companies in the space.

What Ive learned more than anything recentlyis that these organizations need more industry engagement. There are certainthings-how we qualify shipping lanes, logistics, freezing, microbial testing,method validation-that are absolutely inefficient when we are reinventing thewheel all the time at different companies, stated Vernon.

Because this industry is so new-we only have4 or 5 approved cell and gene therapy approved commercial products-its reallyjust too early to be able to standardize, added Hung.

Because it is in fact too early tostandardize, cell and gene therapy organizations find themselves confronting aCatch-22. Manufacturing demand will compete with the need to innovate. Thepersonalized nature of cell and gene therapy will be at odds with the push tostandardize manufacturing and supply chain best practices. The push to beat thecompetition to market will inherently limit the data sharing necessary touplift the entire industry.

While these manufacturing and supplychallenges appear daunting, they always are when it comes to revolutionizingmedicine. Its the energy created by these contradictions that will driveprogress and foment innovation; its the immense challenges of frontierindustries like cell and gene therapy that will ensure the very best of thebest rise to success to pave the way forward for the organizations thatfollow. And its success that will breedmore success, as the conflict between these seemingly opposing forces will onlyresolve itself over time as more companies take therapies to market and thestories of their struggles and successes become public knowledge.

It seems like the Wild West now but conquering new territory is always complex and messy. The car will eventually have time to slow down and get in the shop to fine-tune its engine, offering a smoother, more efficient and faster ride to its destination.

The BHCR regions burgeoning cell and gene therapy cluster, as represented by those on the New Frontiers in Biomanfucturing panel, will clearly play a leading role in fulfilling the promise of cell and gene therapies to deliver high-quality therapies and cures to patients in need while driving down costs over time.

Learn more about working at Kite from Chris McDonald, VP of Manufacturing and Site Lead.

Steve has over 20 years experience in copywriting, developing brand messaging and creating marketing strategies across a wide range of industries, including the biopharmaceutical, senior living, commercial real estate, IT and renewable energy sectors, among others. He is currently the Principal/Owner of StoryCore, a Frederick, Maryland-based content creation and execution consultancy focused on telling the unique stories of Maryland organizations.

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Biomanufacturing and Supply Chain Standardization Key to Success in Cell and Gene Therapy Industry Boom - BioBuzz

Research targets gene therapy for exudative AMD patients – Modern Retina

Abstract / Synopsis:

Two anti-VEGF gene therapies are being investigated in clinical trials of patients with exudative age-related macular degeneration. Initial efficacy and safety results are encouraging.

Anti-VEGF gene therapy for exudative age-related macular degeneration (AMD) has transformative potential for reducing treatment burden and improving patient outcomes, according to Szilrd Kiss, MD.

Two investigational anti-VEGF gene therapies are currently being investigated in clinical trialsRGX-314 (Regenxbio) and ADVM-022 (Adverum). Dr. Kiss described the two technologies and reviewed some preliminary clinical trial results that support their promise for providing sustained benefit with a single injection.

Considering the treatment burden of anti-VEGF therapy for other ocular diseases, we can imagine that exudative AMD is just the first indication that will be targeted for anti-VEGF gene therapy, said Dr. Kiss, chief, Retina Service, associate professor of ophthalmology, and associate dean at Weill Cornell Medical College, New York, NY.

RGX-314 delivers a gene for an anti-VEGF fab protein that is similar to ranibizumab. It uses adeno-associated virus-8 (AAV8) as a vector and is administered in the operating room as a subretinal injection.

AAV is the most common viral vector carrier used for gene therapy. Different AAV serotypes have different tissue selectivity, Dr. Kiss explained. AAV8 is a wild type AAV that has the propensity for greater transfection of retinal cells compared with AAV2 following subretinal gene therapy delivery.

RELATED:AAO 2019: Encouraging results revealed from early trial of subretinal gene therapy for wet AMD

Disclosures:

Szilrd Kiss, MDe: [emailprotected]This article was adapted from Dr. Kiss presentation at the 2019 meeting of the American Academy of Ophthalmology. Dr. Kiss is a consultant to RegenxBio and Spark Therapeutics and is a consultant and equity owner in Adverum.

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Research targets gene therapy for exudative AMD patients - Modern Retina

Magenta Therapeutics Demonstrates First-ever Successful Gene Therapy Transplant Without Chemotherapy in Primates Using a Single Dose of Antibody-drug…

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of immune reset to more patients, today announced that new results from its CD117-ADC patient preparation program were presented at the 61st Annual Meeting of the American Society of Hematology (ASH). These results, which were highlighted in an oral presentation at ASH by John Tisdale, M.D., Director, Molecular and Clinical Hematology Section, National Institutes of Health, showed the first-ever successful transplant of gene-modified cells in non-human primates using a targeted, single-agent antibody-drug conjugate (ADC), without the use of chemotherapy or radiation.

Todays conditioning regimens involve high doses of chemotherapy, often paired with radiation, to remove the disease-causing cells. As a result, patients undergoing gene therapy or stem cell transplant are all faced with a difficult choice: whether to endure severe toxicity and risk infertility and cancer for the chance for a cure. Magentas portfolio of targeted ADCs represents an extremely promising new option to prepare patients for gene therapy or transplant with no need for toxic chemotherapy or radiation, said Dr. Tisdale. The results presented today show that a single dose of single agent CD117-ADC achieves the same level of depletion as four doses of busulfan chemotherapy to enable successful engraftment and persistence of stem cells modified with the -globin gene, the gene that causes sickle cell disease and -thalassemia when mutated. Importantly, the animals undergoing preparation with CD117-ADC showed none of the damaging toxicities associated with busulfan conditioning.

Magenta is the only company with the people, platforms and a product engine committed to comprehensively transforming immune and blood system reset, which includes revolutionizing the toxic methods that are used to prepare patients for gene therapy and transplant today. said Jason Gardner, D.Phil., Chief Executive Officer and President, Magenta Therapeutics. The gene therapy field has learned that higher levels of stem cell depletion, which meant higher doses of busulfan, were needed to ensure long-term engraftment of the gene-modified cells and persistence of gene therapy. Across all the modalities we have tested, we have seen that ADCs are most effective at achieving these high levels of stem cell depletion without chemotherapy to enable engraftment and long-term durability of the transplant. Todays impressive results provide important validation of the ADC approach as well as the CD117 target for patient preparation and underscore Magentas leadership in the field of conditioning.

Results from the CD117-ADC Patient Preparation Program

Title: A Single Dose of CD117 Antibody Drug Conjugate Enables Autologous Gene-Modified Hematopoietic Stem Cell Transplant (Gene Therapy) in Nonhuman Primates (Abstract #610)Presenter: John Tisdale, M.D., Director, Molecular and Clinical Hematology Section, National Institutes of Health, Bethesda, Md.

Magentas most advanced patient preparation program, CD117-ADC, targets CD117, a protein expressed on hematopoietic stem cells. CD117-ADC is designed to remove the genetically mutated cells in the bone marrow that cause certain genetic diseases, such as sickle cell disease, enabling curative stem cell transplant or gene therapy.

Results presented by Dr. Tisdale showed:

About Magenta Therapeutics

Magenta Therapeutics is a clinical-stage biotechnology company developing medicines to bring the curative power of immune system reset through stem cell transplant to more patients with autoimmune diseases, genetic diseases and blood cancers. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise, a unique business model and broad networks in the stem cell transplant world to revolutionize immune reset for more patients.

Magenta is based in Cambridge, Mass. For more information, please visit http://www.magentatx.com.

Follow Magenta on Twitter: @magentatx.

Forward-Looking Statement

This press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as may, will, could, should, expects, intends, plans, anticipates, believes, estimates, predicts, projects, seeks, endeavor, potential, continue or the negative of such words or other similar expressions can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation risks set forth under the caption Risk Factors in Magentas Registration Statement on Form S-1, as updated by Magentas most recent Quarterly Report on Form 10-Q and its other filings with the Securities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Magenta believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Magenta nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

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Magenta Therapeutics Demonstrates First-ever Successful Gene Therapy Transplant Without Chemotherapy in Primates Using a Single Dose of Antibody-drug...

GenEdit and Editas Medicine Enter into Exclusive License and Collaboration Agreement for Nanoparticle Gene Therapy Delivery – Business Wire

BERKELEY, Calif.--(BUSINESS WIRE)--GenEdit, Inc., a developer of a novel polymer nanoparticle technology platform for non-viral- and non-lipid-based delivery of gene therapies, today announced that it has entered into a worldwide, exclusive license and collaboration agreement with Editas Medicine, Inc., a leading genome editing company. GenEdit has developed a comprehensive delivery system for CRISPR-based therapeutics, including gene knockout and gene repair therapies, to enable safer delivery options with improved efficiency.

"This license and collaboration agreement further validates the strength of our intellectual property portfolio and the potential of GenEdits technology," said Kunwoo Lee, Ph.D., co-founder and chief executive officer of GenEdit. "We are pleased to establish our relationship with Editas Medicine as they leverage our technology to develop potential genomic medicines."

Under the terms of the agreement, GenEdit has granted Editas Medicine an exclusive worldwide license, with rights to sublicense, to GenEdits Cpf1-based technologies. In return for these rights, GenEdit will receive undisclosed upfront and development milestone payments, including royalties on net sales of products incorporating the licensed intellectual property. In addition, GenEdit and Editas Medicine will collaborate on evaluating delivery of Cpf1-based technologies with GenEdits nanoparticle platform. Editas Medicine will provide research funding and have an option to continue development after the initial collaboration period.

GenEdits nanoparticle platform consists of a proprietary non-viral, non-lipid library of polymers that efficiently encapsulate and deliver cargo [RNA, DNA, protein and/or ribonucleic acid-protein complexes (RNP)] to specific tissues. The company screens the library to identify initial hits and then uses computational analysis and medicinal chemistry for iterative lead optimization. The company has used this platform to identify multiple candidate polymers for efficient and specific delivery of gene editing to a range of tissues.

"Compared to viral vectors and lipid-based nanoparticles, our approach has the potential for better targeting, more cargo, and lower manufacturing cost," said Timothy Fong, Ph.D., chief scientific officer of GenEdit. "In particular, our approach has the potential to enable in vivo gene editing of multiple tissues with CRISPR and expand the potential of gene therapies to treat more diverse sets of diseases."

About GenEdit

GenEdit was founded to transform the delivery of gene and gene editing therapies. We have synthesized the NanoGalaxy library of polymers that can encapsulate RNA, DNA, protein and/or RNP. Through advanced screening methods, computational analysis and iterative medicinal chemistry, we have demonstrated efficient delivery of gene editing cargo to specific tissues. We seek development partnerships for specific tissues and/or gene targets while advancing our internal pipeline of gene editing therapies.

For more information, please visit http://www.genedit.com.

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GenEdit and Editas Medicine Enter into Exclusive License and Collaboration Agreement for Nanoparticle Gene Therapy Delivery - Business Wire

First 2 Adults with Severe Hemophilia A Respond Well to Gene Therapy BAY 2599023 in Clinical Trial – Hemophilia News Today

Bayersinvestigationalgene therapy BAY 2599023 safely and effectively increased the levels ofclotting factor VIII (FVIII) and prevented or lessened bleeding in the first two people with severe hemophilia A treated ina Phase 1/2 clinical trial, preliminary data show.

The ongoing trial (NCT03588299; 2017-000806-39) is enrolling up to 30 eligible adult patients. More information, including recruiting sites in the U.S. and Europe, is availablehere.

These early results will be presented in the poster, First-in-human Gene Therapy Study of AAVhu37 Capsid Vector Technology in Severe Hemophilia A, at the 61st American Society of Hematology (ASH) Annual Meeting & ExpositionrunningDec. 710 in Orlando, Florida.

BAY 2599023 initially by Dimension Therapeutics as DTX201 is being developed by Bayer in collaboration with Ultragenyx Pharmaceuticals. The potential gene therapy aims to promote a sustained production of FVIII and overcome its deficit in hemophilia A patients, reducing or eliminating the need for prophylatic, or preventive, FVIII replacement therapy and the occurrence of bleeding events.

Administered as a single infusion, the therapy uses a modified and harmless version of the adeno-associated virus (AAV), called AAVhu37, to deliver a shorter but functionalcopy of the FVIII gene to liver cells, where clotting factors are produced. This version of the FVIII gene is known as B-domain deleted FVIII gene.

Preclinical studies showed that AAVhu37 effectively delivered the FVIII gene to liver cells, had a favorable distribution, and induced a durable FVIII production.

In addition, preclinical data showed that BAY 2599023 had a good safety profile, and the potential to promote FVIII production to levels considered to be therapeutic over a long period of time.

The ongoing, dose-establishingPhase 1/2 trial (NCT03588299; 2017-000806-39) is evaluating the safety, tolerability and early effectiveness of three ascending doses of BAY 2599023 in adult men with severe hemophilia A who have been previously treated with FVIII products.

It is the first clinical trial to evaluate a gene therapy based on the AAVhu37.

Up to 30 enrolled patients will be given a single intravenous infusion of one of three doses of BAY 2599023. The studys primary goal is to measure safety through reports of adverse events. Secondary goals include measuring FVIII activity and assessing the number of patients who reach more than 5% of FVIII production at six and 12 months after treatment at the different doses.

Data on the first two men treated at BAY 2599023s starting dose (0.5 x 1013 gene copies/kg) will be presented at the meeting. These men had more than 150 days of treatment with FVIII products, no history of FVIII inhibitors, and no detectable immune response against AAVhu37.

No adverse events were reported after more than 15 weeks of safety evaluations (about four months). Blood levels of liver enzymes also remained within a normal range, and either of these patients needed to be treated with corticosteroids.

The first man reached a stable FVIII production of around 5%, and was free of bleeding events or a need for prophylactic treatment for six weeks. The second patient, who had 99 bleeds in the year before receiving the gene therapy, reached a stable FVIII production of around 17%, and has been bleed-free for more than 5.5 months (at the time of data collection).

These preliminary data suggest that BAY 2599023 is safe and effective in promoting the production of FVIII and in reducing or preventing the occurrence of bleeding events and the need for prophylactic treatment, the researchers wrote.

Overall, data generated from this first dose cohort demonstrate that successful translation from pre-clinical to clinical development and proof-of-mechanism for BAY 2599023 was achieved, they concluded.

Marta Figueiredo holds a BSc in Biology and a MSc in Evolutionary and Developmental Biology from the University of Lisbon, Portugal. She is currently finishing her PhD in Biomedical Sciences at the University of Lisbon, where she focused her research on the role of several signalling pathways in thymus and parathyroid glands embryonic development.

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Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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First 2 Adults with Severe Hemophilia A Respond Well to Gene Therapy BAY 2599023 in Clinical Trial - Hemophilia News Today

New Anti-Aging Clinical Trial Begins. For $1 Million, You Can Be a Participant. – Livescience.com

An American biotech company has launched clinical trials in Colombia to test a new therapy designed to reverse the aging process, and in turn, treat age-related diseases, according to news reports.

But to steal a sip from this purported fountain of youth, participants in the trial must first fork over $1 million a fee that seems even more astronomical when you consider that most clinical trials are either free or provide participants with financial compensation, according to a report by OneZero, a Medium publication about tech and science.

The pricey trial is being run by Libella Gene Therapeutics, a Kansas-based company whose website proclaims that "the future is here." The company announced its intention to test its anti-aging remedies in Cartagena, Colombia, in 2018, and began recruiting for the trials in October of this year. Using a single-gene therapy, Libella aims to "prevent, delay, or even reverse" the general effects of aging, as well as treat diseases that emerge in old age, such as Alzheimer's, according to ClinicalTrials.gov.

In fact, in its own press release, the company boasted, without evidence, that its gene therapy "may be the world's first cure for Alzheimer's disease." The bold claim raises an obvious question: Will the treatment actually work?

Short answer: No one really knows, but the fact that Libella shipped its operation beyond the reach of the U.S. Food and Drug Administration (FDA) doesn't inspire confidence, experts told OneZero.

Related: 5 Reasons Not to Fear Getting Older

Unlike anti-aging face creams that soften the superficial signs of aging, the Libella therapy aims to reverse aging from the ground up, so to speak, starting at the level of our genes. Specifically, the gene therapy is intended to lengthen patients' telomeres structures that cap the tips of chromosomes and prevent the genetic material inside from fraying. Telomeres grow shorter each time a cell divides, and when the structures reach a critical length, cells either stop dividing or perish, according to Stanford Medicine.

The theory goes, if you rebuild the body's shortened telomeres, the process of aging might be thrown in reverse. This is not a new idea. Several studies in mice suggest that using gene therapy to lengthen telomeres can reverse certain signs of aging in the animals. A 2015 study from Stanford prompted similar effects in isolated human cells; the treatment lengthened cells' telomeres by fiddling with a close cousin of DNA, called RNA, which helps cells build proteins.

The Libella therapy aims to help cells rebuild telomeres by activating a gene in their DNA that would normally be switched "off." The gene, called TERT, contains instructions to build a protein called "telomerase," an enzyme that adds molecules to the end of telomeres and prevents the structures from shortening during cell replication, according to a 2010 report in the journal Biochemistry.

Libella's lead scientific officer, molecular biologist William Andrews, originally helped identify the human telomerase enzyme at the biotech firm Geron. Later, he licensed a gene therapy based on the finding to Libella, according to OneZero. "I can't say [telomere shortening is] the only cause of aging, but it plays a role in humans," Andrews told the publication.

Related: 8 Tips for Healthy Aging

Andrews' therapies will soon be put to the test in Colombia, where one 79-year-old will receive the anti-aging treatment in next month, according to OneZero. The anti-aging trial will include four more participants over age 45 and focus on verifying that the treatment is "safe and tolerable," meaning it does not harm patients or cause unacceptable side effects.

Two more trials will use the same therapy but aim to "prevent, delay, or even reverse the development" of Alzheimer's disease and critical limb ischemia, an age-related condition in which a person's arteries become severely obstructed. Participants in these trials must already be diagnosed with the disorders.

After treatment, participants in all three trials will remain in the clinic for 10 days for further monitoring, and then return at regular intervals for checkups over the following year.

Libella's gene therapy involves a one-time injection delivered through an IV; the Alzheimer's therapy uses the same formula but doctors inject the product into the patient's spinal fluid. Within the product, a modified virus carries the TERT gene into cells and injects the genetic material into their DNA. The modified viruses cannot transmit diseases to people, but in high enough doses, the germs could provoke a harmful immune response in the patient, according to a 2018 animal study. Libella representatives declined to say how high a dose their clinical trial participants will receive.

"All I can say is, it's a lot," Andrews told OneZero.

Potential side effects aside, the fact that the Libella treatment will be administered beyond the purview of the FDA is telling, according to one expert. Leigh Turner, a bioethicist at the University of Minnesota, told OneZero that "even though the company is based in the United States, they've managed to find a way to evade U.S. federal law by going to a jurisdiction where it's easier to engage in this activity."

The $1 million entry fee is also alarming, Turner said, given that most clinical trials don't charge patients anything to enter. Andrews told OneZero that the fee is justified because it costs the company hundreds of thousands of dollars to make enough product to treat just one person.

The appearance of the trials on ClinicalTrials.gov, an official registry maintained by the National Institutes of Health, does not boost their credibility, she added. The automated database can be easily manipulated and "can basically be used as a marketing platform," she said.

Other stakeholders in the telomere-lengthening business are concerned, too. Michael Fossel, founder and president of the biotech startup Telocyte, told OneZero that his company's own therapy is similar to the Libella treatment the difference is that Telocyte is seeking approval through the FDA. "We're afraid that something will go wrong [with the Libella trials], whether it's from a safety or efficacy standpoint," he said.

Related: Extending Life: 7 Ways to Live Past 100

But even in a best case scenario, wherein no patients come to harm, the Libella therapy still might not deliver any notable health benefits. Some research suggests that no link exists between telomere length and aging.

For instance, a study published this year examined more than 261,000 people between age 60 and 70, and found no correlation between participants' telomere lengths and their age-related health outcomes, including their overall cognitive function, muscular integrity and the age of their parents. Long telomeres were associated with a lowered risk of coronary heart disease as compared with short telomeres, but longer telomere length was also linked to a heightened risk of cancer.

"Telomere lengthening may offer little gain in laterlife health status" and lead to an increased risk of cancer, the authors noted.

It remains to be seen whether Libella has truly tapped the fountain of youth, but given the dubious nature of their clinical trials, potential participants may want to exercise caution before relocating to Colombia and shelling out $1 million for a chance to live longer.

Originally published on Live Science.

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New Anti-Aging Clinical Trial Begins. For $1 Million, You Can Be a Participant. - Livescience.com

Watch out, Keytruda. Ferring’s bladder cancer gene therapy rival has new dataand they look competitive – FiercePharma

On the heels of an FDA speedy review for Keytrudas potential use in non-muscle invasive bladder cancer (NMIBC), its close rival, a gene therapy by Ferring Pharmaceuticals spinout FerGene, has posted late-stage data. By the looks of it, the two drugs are up for a fight.

Among patients with high-risk NMIBC superficial disease thats unresponsive to standard-of-care Bacillus Calmette-Gurin (BCG), nadofaragene firadenovec eliminated tumors in 53%, or 55 of 103 patients,at month three in a phase 3 study, FerGene unveiled Thursday at the Society of Urologic Oncology meeting.

By comparison, in Keytrudas own registrational trial on the same target patient population, the Merck & Co. PD-1 completely cleared tumors in 41.2%, or 42 of 102 patients, after three months, according to an update at the European Society for Medical Oncology annual meeting in September.

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The length of time responses lasted appeared similar between the two drugs in their separate studies. For Keytruda, 24 patients (23.5%) continued to show no signs of disease after a year. As for nadofaragene firadenovec, 24.3%, or 25 patients, were still tumor-free at month 12.

In terms of safety, Keytruda recorded Grade 3/4 side effects in12.7% of patients, while FerGene said there were no Grade 4/5 events in its study.

We are pleased with these Phase 3 data results, including the complete response rates and favorable safety profile seen with nadofaragene firadenovec, Nigel Parker, scientific founder of FKD Therapies, said in a statement. The data have also helped FKD'snew drug application earn an FDA priority review.

RELATED:Merck's Keytruda is bound for new bladder cancer territory. But can it hold up against gene therapy?

Ferring recently gained commercial rights to the gene therapy from FKD, and, with $400 million in help from Blackstone Life Sciences, spun it into FerGene. Interestingly, it was Merck that licensed the drugout to FKD in the first place in 2011 in return for an equity stake in the then-newly formed Finnish company.

Priority reviews in hand, the two companies could be looking at FDA approvals soon. The burning question is, how does FerGene plan to price a gene therapy, which belongs to a class of drug thats notoriously costly? In a statement sentto FiercePharma, Ferring said it's too early to discuss pricing, that its top priority is still to get nadofaragene firadenovec approved andinvest into R&Dto study the product in more indications.

Keytruda is meant to be given ata fixed dose every three weeks. Nadofaragene firadenovec, which uses an adenovirus vector to deliver the gene interferon alfa-2b to stimulate an innate immune response to fight cancer, is administered into the bladder every three months.

Merck does have an upper hand against FerGene. The Big Pharma has been the sole supplier of BCG in the U.S. and several other key markets globally for several years now. So, it could offer BCG and Keytruda as a one-two punch for NMIBC, similar to the wayBayer is billing Nexavar and Stivarga as a part of the same continuumin first- and second-line liver cancer.

RELATED:Merck limits orders for bladder cancer drug as demand outstrips supply

There are other players eyeing the same patient population. Sesen Bio has Vicinium, an antibody-drug conjugate that targets epithelial cell adhesion molecule antigens on the surface of tumor cells to deliver a toxin payload. In its own phase 3 trial dubbed Vista also in high-risk, BCG-unresponsive NMIBC, Vicinium eradicated tumors in 40% of 89 patients at month three, according to an update the company provided in August. However, its response seems to wane over time more quickly than its rivals', as only 17% of patients showed no signs of tumor activity after 12 months.

The Cambridge, Massachusetts-based biotech recently held two meetings with the FDA and confirmed a submission process, including the design for a post-marketing confirmatory trial. It would enroll BCG-refractory patients who, because of supply constraints, haven't received an optimal BCG dose, which the company said represents a broader patientpopulation in light of anongoing shortage.

Sesen now expects to submit a biologics license application under rolling review by year-end with potential approval in 2020.

As for its pricing, during a presentation at the H.C. Wainwright investor conference in September, Sesens president and CEO Thomas Cannell pointed out that PD-1/L1s would cost about $150,000 to $200,000 per patient per year in NMIBC.

Weve done two rounds of market research with payers, and they think thats reasonable, he said. They think at those levels, there will probably be minimal prior authorization or step edits in terms of restricting a treatments use.

Assuming an official launch in 2021, Jefferies analysts, in a Nov. 12 note to clients, pegged $167.5 million for Viciniums U.S. sales in 2024. Before the priority designation, SVB Leerinks Daina Graybosch predicted a Keytruda launch in NMIBC in 2022 and forecastU.S. sales of $250 million in the indication for the Merck PD-1 inhibitor in 2025.

Editor's Note: The story has been updated with a statement from Ferring Pharma.

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Watch out, Keytruda. Ferring's bladder cancer gene therapy rival has new dataand they look competitive - FiercePharma

Be polite and follow the rules of fishing from shore, boat – Buffalo News

The concept of the golden rule is simple and straightforward: Treat other people as you would wish to be treated. Of course, if you dont know what the proper treatment should be for a specific situation, you can create problems without even knowing how or why, especially in the world of fishing.

An excellent case in point is the lower Niagara River, where anglers fishing from shore and boat constantly jockey for position at perceived hot spots in the river.

Ill seek out my shore fishing spot while its still dark out, says Ricardo Davila of Wheatfield, an avid Niagara Gorge angler. Quite often, after Ive established my area, I will have fishermen move into my angling area that I am targeting because they just dont understand how I am fishing.

For example, if I am tossing spoons or spinners under murky conditions, I will try and swing my lure along the shoreline downriver from me, using the current to give my lure that necessary action, says Davila.

If someone moves in, he will explain that to them and see how they react. If they wont move, Davila will tell them that you can have this spot, but the river is big and there are many, many spots along the river from Artpark to the Whirlpool. Dont be afraid to explore and be courteous.

Many anglers dont know how close the fish like trout and salmon are to the shoreline in the gorge, says Davila. Centerpin guys will fish the water differently from the spinning guys and from the fly-fishing folks. Try and understand how each of the different disciplines will work a stretch of water. And if they are courteous back, it can lead to a better understanding and even friendships as you educate each other. Thats happened to me for sure.

Jordyn and Adeline Davila of Wheatfield show off some of the garbage they helped to pick up in the Niagara Gorge. Keep things clean. (Photo courtesy Ric Davila)

While you are showing respect for your fellow outdoorsperson, you should show some respect for the natural resources that you are enjoying, too. Dont litter while you are fishing and bring a bag to haul out any garbage you may find during your fishing adventure. Leave the trail better than you left it.

In places like the lower Niagara River, there are etiquette situations for boaters, as well as boaters and shore fishermen combined. Capt. Jim Rores of Grand Island emphasizes the importance of paying respect to your fellow anglers.

As far as boat etiquette, there are a few things that are no nos in my book, says Rores. Short drifting another boat is a biggie. Im set up on the drift and coming down with the current. You are coming up the river. Instead of going in front of me (above me) you go behind me. By the time you set your boat and get your trolling motor down we are getting too close to each other. Your boat sitting in the current will make my boat catch you faster, thus screwing up my drift.

Another problem is when someone is running their boat right up the current seam where guys are fishing. Instead of going out and around on your way back upriver, they motor right up the seam. This does two things. It creates a wake that gets thrown at other boats and ruins their drift and it pushes the fish out of there because you just ran over them with your big motor.

A third problem is when other boats are egg fishing or bead fishing a drift. They are set up with their bow up, slow drifting eggs or beads. Suddenly a boat comes above you, turns their bow down and starts pulling Kwikfish through the drift right towards you at three times the speed you are going. Many times, the egg fishermen must move to avoid the slow troller.

Its not a problem when there is one boat on the drift fishing eggs but when you have 4 or 5 boats egg fishing a drift and someone decides to pull kwikies through there, you can have a problem, says Rores.

Always be cautious of your wake when running upriver and when setting up to drift. Some boaters might fly back upriver and then pull in to close to you and throw a big wake. Take your time when you get to the top of a drift. Slow down and pull in wide. Its common sense really. As far as dealing with shore guys, you kind of have your hands tied in the fall. They want your water and you want theirs. Just try not to wake them. Most of them are standing on slippery rocks and dont need a two-foot wake thrown at them. Just be courteous. Its tough especially with all the centerpin fishermen around now fishing long rods and letting their floats drift a long way.

Don't get too bunched up with other boats. (Bill Hilts Jr./Buffalo News)

In the streams, Danny Colville of Colville Outfitters points out that there is no book or exact rules. The etiquette differs from spot to spot. It's all about communication between anglers. Sometimes guys from out of town do not realize that they are not following local etiquette due to where they are from. For example, a guy from Canada or the Erie tributaries in Pennsylvania might think it's ok to step above or below you because there is room. However, it might not be okay with the fisherman in the space he is fishing.

I was always told by my mentor that you never fish for the same fish that someone else is fishing for, says Colville. The problem with that is if you go to somewhere like an easy access bridge or dam that is impossible. I know personally that it really makes me mad and messes my drift up when a person using a different tactic steps in below me. Now my drift is cut off. Again, they might not know so I just tell them that they are affecting my drift and need to position themselves above me or move on.

You don't want to impede someone's experience while they are fishing in an area. Just ask and most of the time people will be cool. On top of that, you might get into a conversation about the fishing and find that you share the same love and passion. I have made a lot of good friends like this over the years. We have tons of water, but the spots are tight.

Larger rivers can fit more people in spaces so consider that. Overall, the most important thing is to communicate and maybe change your tactic of fishing if you want to share a honey hole that badly with someone. Float fishing is the best because you can get in a synchronized drift and you know when the angler is done with their drift.

If fly fishing, you need to start upstream from the other angler far enough where you won't get in the way of casting and you are not jumping in front of the fly angler as they work down river. With fly fishing, the choice of space is usually not where the fish typically sit. Work your way down to them by drifting or swinging then stepping down after you retrieve. This way youre presenting the fly closer and closer to the fish hoping you get it in their zone. If you stand below that guy, you cut his drift off. Asking will avoid this.

"Float fishermen pick the spot that they can cast, drift and land fish best. They can cover the whole stretch of river from one location. Impeding their drift really messes them up unless you get in sync with their casting. You need to first ask and then let them cast. Start their drift and then cast behind their bobber and start your drift. This way no one get tangled. When he is done with the drift and starts to retrieve, you finish your drift and continue to repeat.

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Be polite and follow the rules of fishing from shore, boat - Buffalo News

30 Gifts Under $100 (Plus One That’s Too Good to Leave Out) – Mpls.St.Paul Magazine

Clockwise from top: Phone case ($36), by Moglea, from Anthropologie, multiple metro locations, anthropologie.com; recycled bag ($112), by People for Urban Progress, from Show & Tell, 5257 Chicago Ave., Mpls.; Ruby Minnescato wine ($19) from Carlos Creek Winery, Alexandria, 320-846-5443; vase ($88), by Cym Warkov Ceramics, from Combine, 1609 W. Lake St., Mpls., 612-353-5163; taper candle ($10, set of two), by The Floral Society, and candleholder and snuffer ($54), by Studio Macura, both from Russell+Hazel, 219 N. 2nd St., Mpls., 612-353-4149; French soap ($13 each), by Le Baigneur, from Flotsam+Fork, 3730 Chicago Ave., Mpls., 612-823-5799; First Avenue book ($35), from Patina, multiple metro locations, patinastores.com; Lagom book ($30), also from Show & Tell; wood bear ($18), by Plan Toys, from Kinoko Kids, 3803 Grand Ave. S., Mpls., 612-545-5741; earrings ($78), by There There Collective, from Golden Rule, 350 Water St., Excelsior, 612-598-2098; dog collar ($32), by Son of a Sailor, also from Show & Tell; olive oil ($37), by Brightland, also from Golden Rule; diffuser ($40), by Idlewilde, also from Russell+Hazel.

Chocolate ($12), by Mayana,from Golden Rule

Minnesota pencils ($7),from Patina

Frenchie canned cocktail ($15$18 for four cans), by Vikre Distillery, vikredistillery.com

Metal straw ($3), by Steel Straw, also from Golden Rule

Canned cocktails ($5$6), by Dashfire, dashfirebitters.com

Tea towels ($30), by The Rise and Fall, also from Show & Tell

Jewelry dish ($28), by Nightshift Ceramics, also from Golden Rule

MN mittens ($26), also from Patina

Arch stacker ($28), by Raduga Grz, also from Kinoko Kids

Mini kitchen utensils ($9), by Redecker, also from Flotsam+Fork

Leather wallet ($68), by Sandqvist, also from Russell+Hazel

Reusable bags ($36, set of three), by BAGGU, also from Flotsam+Fork

Knives ($62), by Laguiole, also from Flotsam+Fork

Paul Bunyan cards ($7), also from Patina

Socks ($12), by Le Bon Shoppe, also from Golden Rule

Radiant facial oil($49), by Aveda, aveda.com

Liquid color balm ($29), also by Aveda

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30 Gifts Under $100 (Plus One That's Too Good to Leave Out) - Mpls.St.Paul Magazine

Babies named Jolene are on the rise – Quartz

The Dolly Parton renaissance is upon us. In the last year, the American country music icons name has gilded everything from podcasts to college courses, a revived musical to a Netflix documentary. Her resurgence can even be seen in baby names.

Jolene, the titular character of Partons 1973 chart-topping hit, has not seen this much popularity as a baby name since, well, the release of the song in the 70s.

The heartbreak anthem, sung from the perspective of Parton, literally begs Jolene: Please, dont take my man. Partons pleas aside, each chorus of the song repeats Jolenes name four times in a row, twice. Each verse ends with the name almost as a punctuation. Finally, the song leaves us with two more ethereal utterances. All told, thats 31 times the name gets a chance to worm its way inside a listeners head.

Theres no question that the history of the name Jolene is tied up inextricably with the song, says Laura Wattenberg, author of The Baby Name Wizard. Before the hit, Wattenberg says, Jolene was just another compound mid-century name, where two names fuse to become one.

Now, though, the song itself is hard to escape, even if you dont fancy yourself a Dolly Parton fan. Its been covered well over a hundred times by artists from Miley Cyrus to Jack White of the White Stripes, according to Secondhandsongs.com.

With close to one million sales in the US, the songs popularity alone doesnt kick-off name adoption. I think it ultimately comes down to my Golden Rule of Celebrity Baby Names: Its not about the fame, its about the name, says Wattenberg. When baby names come into vogue, they require both the appeal of the name and the appeal of the sound. Jolene features two long vowel sounds, Wattenberg explains, which are highly prized in contemporary name style.

But what about Dolly? Sadly, the name has been a falling star since the start of the last century. Its not a name thats easily taken seriously, says Wattenberg. Dolly Parton has leaned into the name and made it work for her, with her larger-than-life exuberance. For now, at least, Jolene is just more captivating.

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Babies named Jolene are on the rise - Quartz