StemCell Therapy

Ever since becoming a part of the Marvel Cinematic Universe in 2o11, Thor has had a rough go of it. Over his MCU history, he's lost both of his parents before his very eyes, along with his adopted brother, his lifelong home andto add insult to injuryhis right eye. That's a lot to endure over the course of seven years for someone who lives for millennia. And while Thor may have gotten a replacement eye in Avengers: Infinity War, with his luck, he is liable to lose it again in Thor: Love and Thunder.

Here's a quick look how Thor lost and gained an eye over the course of two films, how he may lose it again and how it compares to recent developments in the comic books.

RELATED:Wonder Woman & Superman Just Appeared in the Marvel Universe... as Thors?

Thor: Ragnarok had the Asgardians lose heavily right from the outset, with tragedy befalling them steadily throughout the film. Odin's death frees his secret daughter Hela, the Norse Goddess of Death, who immediately lays waste to Asgard, completely decimating its armies and killing the Warriors Three. After a brief period of exile on the faraway world of Sakaar, Thor returns to reclaim his throne from his evil sister, with Loki, Bruce Banner and Valkyrie in tow.

As the others assisted in rescuing the refugees hiding on the outskirts of Asgard from Hela and her undead army, Thor battled his long-lost sister in the Asgardian throne room. Despite putting up a valiant effort, Hela slashed out Thor's right eye, fulfillinghis arc in the movie and completing his transformation into the more mature king his father once needed him to be. Help is shortlydefeated, along with Asgard's prophesied destruction at the hands of Surtur. As Thor led his people into an uncertain future, he was seen wearing an eyepatch similar to his father.

RELATED: Thor's Eyepatch is CGI in Avengers: Infinity War

The Asgardians were the first casualties seen in Avengers: Infinity War, their ship laid waste as Thanos and the Black Order recovered the Tesseract secretly kept by Loki who was killed personally by Thanos. Responding to the ship's distress call after its destruction, the spacefaring team found Thor within the ship's wreckage and miraculously still alive. To replace Mjolnir, his mythical hammer destroyed by Hela during Ragnarok, Thor left for the dwarven forge of Nidavellir with Rocket and Groot.

RELATED:Thor: The Worthy #1 Is an Electric Dose of Thunderous Nostalgia

As the trio approach Nidavellir, located within the heart of a dying star, Rocket consoled Thor as he reflected over his recent losses. To raise his spirits, Rocket gave Thor a functioning, artificial eye he had stole from a hapless mark sometime earlier, heavily implying that he had smuggled the prosthetic in his anus. While Rocket cautioned Thor should wash the eye first, the God of Thunder instead promptly inserted it. He immediately regained his full sight through his new eyevisibly brown instead of natural blue.

The loss of Thor's eye has been hinted at in the comics in recentyears. Toward the start of Jason Aaron's acclaimed run on the character, a vision of the future incarnation of Thor is seen lacking his left eye. However, it was the recent crossover event War of the Realms that revealed what had actually occurred. The final issue of the series had Thor nailed to the World Tree Yggdrasil so he could gain the greatness of the All-Father. The fiery process proved excruciatingly painful itburned out his eye, but gave Thor the resolve he needed to defeat the villainous Malekith and take the Asgardian throne.

Thor may have regained his sight, but with Taika Waititi returning to helm Thor: Love and Thunder and the acclaimed filmmaker's proclaimed appreciation for Aaron's comic book run on the character, this may be fleeting. After all, the MCU Thor has been a hard-luck case since his debut and, even with Jane Foster back in tow, the Asgardian Avenger still stands to lose much.

KEEP READING: Avengers Just Introduced the Most Dangerous Version of Thor

Godzilla vs. Kong Footage Reveals King Kongs New Titan-Beating Height

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Avengers: How Thor Got His Eye Back (and Why He Might Lose It Again) - CBR - Comic Book Resources

Why James Chen is a man on mission to restore eyesight for all by 2035 | The Independent Independent Premium

The Hong-Kong businessman is using the wealth generated by his familys business for greater good. He tells Zlata Rodionovathat philanthropy is not about writing big cheques

Chen wants to sort the worlds eyesight out before humans are sent to Mars ( James Chen )

Futurist and inventor Elon Musk wants to send humans to Mars by the 2030s but James Chen has a no less ambitious mission:making sure the whole world can see the landing clearly when it happens.

For the past 15 years the philanthropist has been trying to tackle poor vision. According to Chen, the pace of innovation can be overwhelming when compared with the lack of progress in technology designed to tackle the most basic challenges still facing the developing world,from access to food and clean water to a simple pair of corrective glasses.

We really want to solve the vision issue before Nasa or Elon Musk put a man or a woman on Mars because we want everyone on Earth to be able to see it, he says.

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Charitable campaigns include everything from rescuing homeless dogs and planting trees to funding eye surgeries and donating tech skills.

WELL employees picked mandarin oranges for a food bank in Santa Barbara. Workers at Aadya bought Healthy Roots dolls for kids in Detroit from a startup toy company. Omintracs employees will place wreaths on the graves of military veterans on Wreaths Across America Day on Dec. 14.

Starting in November and stretching through this month, tech companies are marking the holiday season with donation drives, financial contributions, and volunteer projects.Salesforce has some of the biggest global ambitions to make a difference. At Dreamforce, Salesforce kicked off a Year of Action for the Sustainable Development Goals from the United Nations 17 global goals that measure collective progress against the world's biggest challenges.

Over the next year, Salesforce will donate $17 million to advance the SDGs through grants to its nonprofit partners and company matches to employee donations.

According to Deloitte, millennials, baby boomers, retirees, and Gen Xers want to support social impact work in the corporate sector, which could offer a competitive edge in hiring.

Tech companies can strengthen local communities and build a more loyal workforce by establishing meaningful social impact programs. Here are a few of the ways employees and executives are giving back this season.

Many companies stick with go-to holiday projects such as food and toy drives. Others find ways to support people and animals in need.

The cybersecurity company ReliaQuest sponsors Bike Build, an event that assembles bikes for foster kids in Tampa. The nonprofit onbikes holds the annual event to provide kids with their very first bike. ReliaQuest employees will help out at the event as well.

Sigstr employees are supporting a dog rescue group with the "Dogs First, Sigstr Second" campaign. Justin Keller, vice president of marketing for the email signature marketing platform, said that team members meet clients on a target account list, and the company makes a donation to rescue a homeless dog on the employee's behalf.

The software development company Chetu supported a tree-planting initiative in India last month. Team members volunteered with Mission 100 Crore Tree Plantation to add trees in a region that has poor air quality.

Chetu said its employees donated over 30 hours to the initiative last month and would continue to invest in this initiative in the future.

Many companies use their particular technical skillset to support causes.

DataRobot, an artificial intelligence company, has an AI for Good program that works with educational institutes, hospitals, and environmental nonprofits.

The company shares technology, time, and resources with the organizations to make sure their machine learning applications generate real, long-term value.

Legacybox will help families in Southern California preserve memories from the holidays and throughout the year by donating $100,000 in digitizing kits to residents in communities at risk of wildfires. Residents can get slides, films, and audio recordings converted to digital format as a download, on a zip drive, or a DVD.

Ada, a marketing automation firm, partnered with a tech-centric nonprofit to develop the Chalmers chatbot designed for Toronto's homeless community. This service provides 24/7 access to information about where to get free meals and clothing as well as open shelter space.

"At Ada, we're inspired by our namesake Ada Lovelace, whose legacy reminds us to challenge norms and bring new thinking to helping solving important social problems," said Ruth Zive, Ada's head of marketing.

The digital banking company Quontic is addressing homelessness in the Dominican Republic.

The company is sending a group of 35 employees to the island for four days to build two homes for two homeless families. Quontic CIO Patrick Sells said service project fits with the company's corporate mission to provide mortgages to low-income families and immigrants.

"This trip will also serve as a time to do team building and corporate strategy work but most importantly remind all of us the critical work we do everyday in our offices to help families like those we served on the trip," he said.

Volunteers from SE2 help with website maintenance for United Way of Greater Topeka Christmas Bureau, a nonprofit that connects donor families with people in need during the holiday season.

Back in 2011, SE2 built a responsive web application to tackle the administrative processes that the community adoptions required.

Today, the United Way leverages the web-based system to scan and index paper applications, capture adopter/adoptee data, match families and share information between all the involved parties.

SE2 employees make improvements to the system each year and donate volunteer time as well.

"This has been a great opportunity for us to support United Way Christmas Bureau over the last several years using our skills to simplify and automate the family adoption process," said Ambrish Patel, an enterprise architect at SE2.

Many companies match donations of time and money made by employees through the Pledge 1% initiative. Led by Salesforce, this initiative builds corporate philanthropy by asking companies to make annual donations of 1% of product, equity, profit, or time to charitable organizations.

The cloud communications platform Twilio is a Pledge 1% company. To date, 232 Twilio employees have donated to or volunteered for the Pledge 1% program, which has resulted in $56,333.00 in donations to 145 charities.

"Through our Pledge 1% initiative, we match employees' donations and volunteer time to nonprofits of their choice, and we add in an additional boost between November 18 to December 18," said Erin Reilly, the company's chief social impact officer.

Advertising tech company Media Math is also a member of the Pledge 1% movement. The company's philanthropic division focuses on the 13% of people living on less than $2 per day through a Campaigns Count project.

"For every 20 campaigns in our platform, MediaMath.org funds an eyesight-saving surgery for individuals who suffer from low vision or whose blindness is treatable," said Michael Quinn, founder and director of the company's philanthropic arm.

In September, the company reached a milestone: funding surgeries for 5,000 people in some of the poorest regions of the world.

Our editors highlight the TechRepublic articles, galleries, and videos that you absolutely cannot miss to stay current on the latest IT news, innovations, and tips. Fridays

Each December on National Wreaths Across America Day, Wreaths Across America coordinates wreath-laying ceremonies at Arlington National Cemetery, as well as at more than 1,600 additional locations in all 50 U.S. states, at sea, and abroad.

Image: Wreaths Across America

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Tech companies find creative ways to give back - TechRepublic

A method of machine learning has proven capable of turning 2D images into 3D models. Created by researchers at multi-million-dollar GPU manufacturer NVIDIA, the framework shows that it is possible to infer shape, texture, and light from a single image, in a similar way to the workings of the human eye.

Close your left eye as you look at this screen. Now close your right eye and open your left, writes NVIDIA PR specialistLauren Finkle on the company blog, youll notice that your field of vision shifts depending on which eye youre using. Thats because while we see in two dimensions, the images captured by your retinas are combined to provide depth and produce a sense of three-dimensionality.

Termeda differentiable interpolation-based renderer, or DIB-R, the NVIDIA rendering framework has the potential to aid, and accelerate various areas of 3D design and robotics, rendering 3D models in a matter of seconds.

Making 3D from 2D

As explained by Finkle, the 3D world we live in is actually seen through a 2D lens otherwise deemed stereoscopic vision. Depth is created in the brain by combining images seen through each eye, creating the sense of a 3D image.

Based on a similar principle, DIB-R is capable of transforming input from a 2D image into a map, predicting shape, color, texture and lighting of an image. This map is then used to shape a polygon sphere, creating a 3D model representing the object in the original 2D image.

To achieve high 3D modeling speeds, DIB-R must be trained first with a wide dataset of images. One of the tests the team has performed so far is with a series of bird photos. All of the photos areRGBA modeled. After two days of training the framework using a singleNVIDIA V100 GPU, it could produce a 3D object from a 2D image in less than 100 milliseconds. The results can be seen below.

Giving sight to autonomous robots

One of the potential applications of such a framework is in the development of autonomous robots, capable of understanding the environment around them and perceiving depth. Another application is in the creation of rapid 3D mockups based on 2D sketches, i.e. for architecture and product design.

For Jonathan Beck, founder of 3D art heritage projectScan the World, the process also has certain implications in photogrammetry a method commonly used for the rendering of real-world 3D objects into digital, 3D models. Ive seen something similar before made by independentdevelopers but this is the first time its been released by a big organization, Beck states. It would be interestingto see how this leads into photogrammetry, where an AI can assume parts of a missing sculpture from other images. Much like how the Artec 3D scanner can calculate the form of an object if there are gaps in the scan data.

AI can help create a bridge where 3D scanning cannot currently deliver.

More details of the NVIDIA project can be found in this dedicated microsite, and in the paper Learning to Predict 3D Objects with an Interpolation-based Differentiable Renderer. The research is conducted and authored byWenzheng Chen, Jun Gao, Huan Ling, Edward J. Smith, Jaakko Lehtinen, Alec Jacobson, and Sanja Fidler.

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Featured image shows3D models of cars created from 2D source images. Image via NVIDIA

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NVIDIA proposes way of teaching robots depth perception, and how to turn 2D images into 3D models - 3D Printing Industry

WASHINGTON, D.C., Dec. 11, 2019 (GLOBE NEWSWIRE) -- via NEWMEDIAWIRE -- The Alliance for Regenerative Medicine (ARM), the international advocacy organization representing the cell and gene therapy and broader regenerative medicine sector, announced its 2020 Cell & Gene Therapies State of the Industry briefing will take place January 13, 2020 in San Francisco, held in conjunction with Biotech Showcase 2020.

ARMs State of the Industry briefing is the largest cell and gene therapy-focused annual event taking place during the week of the 2020 J.P. Morgan Healthcare Conference. The briefing is expected to attract more than 500 of the fields leading executives, investors, life science media, patient advocates, and academic leaders.

This briefing offers a comprehensive industry overview, including insights into key sector trends and metrics, the financial and partnering outlook, recent advances, the clinical pipeline and potential product approvals, commercialization challenges, and a preview of the coming year.

Preliminary Agenda:

8:00 8:20am | Introduction & Industry UpdateJanet Lambert,CEO, Alliance for Regenerative Medicine

8:20am 9:05am | Emerging Cell Therapies for CancerPascal Touchon,CEO, Atara BiotherapeuticsMatthew Kane,Co-Founder and CEO, Precision BioSciencesSamarth Kulkarni,CEO, CRISPR Therapeutics

9:05am 9:50am | Next Generation Gene and Cell TechnologiesShelia Mikhail,CEO, AskBioLaurence Cooper,CEO, ZiopharmTimothy Miller,Co-Founder, President, & Chief Scientific Officer, Abeona Therapeutics

Registration is complimentary and open to the public; however,RSVP is required. The event will be broadcast live via streaming webcast,available on ARMs website.

The briefing will take place at the Parc 55 Hilton, 55 Cyril Magnin Street in San Francisco, California from 8:00 9:50am and is held in conjunction with Biotech Showcase, organized by EBD Group and Demy Colton. Please note that attendance at this briefing is separate from registering to attend the Biotech Showcase conference, which requires paid registration.

About The Alliance for Regenerative MedicineThe Alliance for Regenerative Medicine (ARM) is an international multi-stakeholder advocacy organization that promotes legislative, regulatory and reimbursement initiatives necessary to facilitate access to life-giving advances in regenerative medicine worldwide. ARM also works to increase public understanding of the field and its potential to transform human healthcare, providing business development and investor outreach services to support the growth of its member companies and research organizations. Prior to the formation of ARM in 2009, there was no advocacy organization operating in Washington, D.C. to specifically represent the interests of the companies, research institutions, investors and patient groups that comprise the entire regenerative medicine community. Today, ARM has more than 350 members and is the leading global advocacy organization in this field. To learn more about ARM or to become a member, visithttp://www.alliancerm.org.

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The Alliance for Regenerative Medicine Announces 2020 Cell & Gene Therapies State of the Industry Briefing - GlobeNewswire

When embryonic Stem Cell therapy was first discovered in 1998, it changed the face of medicine. The idea of being able to regenerate and replace damaged cells seemed futuristic at the time, yet today such treatments are commonplace. Now, science has taken another quantum leap this time into the nano-sized world of exosomes, tiny bubbles that grow out of cell walls and contain much of the information contained within the cell including Growth factors, microRNA and messenger RNA. Mesenchymal stem cell (MSC) exosome therapy is currently one of the hottest trends in regenerative medicine, one that patients at AM Medical in Yelm can now experience.

Everyone has heard of stem cell therapy, but it turns out that its not the stem cells that are doing the work, says Dr. Ana Mihalcea, President of AM Medical. Its the exosomes that carry the information of regeneration. Infused stem cells, attach to blood vessel walls, and then give off exosomes.

Exosomes have several key differences from stem cells; they do not get removed from the circulation like stem cells, which are in the body for less than 72 hours before they get destroyed by the immune system; they do not produce a rejection reaction because they are not a cell and contain no DNA, and they pass the blood brain barrier, Mihalcea notes. In a study on stroke scientists fluorescently tagged exosomes, and the infused exosomes went exactly to the region where the stroke had occurred, she adds. The same was not true of stem cells as they do not cross the blood brain barrier.

As a result of their powerful cargo, exosomes can be used to address a multitude of conditions, including arthritis, autoimmune disorders, cardiovascular and neurogenerative diseases like Parkinsons and Alzheimers. Old cells can be reprogrammed by MSC exosomes as the target cells can transcribe the microRNA into functional proteins. Just like a virus, the exosome information of the young stem cells can infect the old cells with Youth, explains Mihalcea.

Spinal cord injuries are an area in which exosomes have produced dramatic results. Mihalcea cites the example of Dr. Douglas J. Spiels Interventional Pain Specialty Practice in NJ. Dr. Spiel has been able to rehabilitate spinal cord injuries with Exosome infusions into the spine and intravenously, she says. After several weeks, hes had patients regain muscle strength and sensation. These are prolonged, ongoing regenerative effects that continued to improve for months after the infusion.

When it comes to autoimmune diseases, inflammation plays a key role. Again, exosomes are able to reduce the problem by downregulating inflammation. TGF Beta 3 [Transforming growth factor beta-3] is the most important anti-inflammatory protein in the body and is abundant in MSC exosomes says Mihalcea. Many more Growth factors for blood vessel growth, neuronal and other tissue growth are present, allowing regenerative effects in all organ systems including skin wounds and burns.

The exosomes at AM Medical come from a laboratory in Florida that conducted pioneering research in the field. They come from perinatal mesenchymal stem cells and are scanned for any possible viruses to ensure their safety. Once harvested, the exosomes are concentrated so they can be infused in large doses.

For patients who qualify, the infusion process takes 10 to 15 minutes. Already, its been producing results for AM Medical patients. Weve had people with arthritis and chronic pain who had great responses, Mihalcea notes. There is an overall increase in wellbeing and sense of rejuvenation that is definitely noticeable.

Perhaps one of the largest sources of excitement over exosomes has to do with their anti-aging effects. Recently, ideas about the root causes of aging have been evolving, according to Mihalcea. Its been thought that aging occurs due to multiple different reasons like stem cell exhaustion, epigenetic changes, telomere shortening and others, she explains. It turns out that exosomes can modify almost all the hallmarks of aging. Theyre changing epigenetic expression to youthful function, and there are many potential applications. This is a new frontier in regenerative medicine that can help many people.

Learn more by watching Dr. Ana Mihalceas video on Exosomes The New Frontier Part 1: Longevity and Age reversal or reading further on the AM Medical website.

Sponsored

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Cutting Edge Exosome Regenerative Therapy Comes to Yelm's AM Medical - ThurstonTalk

WASHINGTON, Dec. 10, 2019 /PRNewswire/ -- A new report released by the independent, non-profit Alliance for Cell Therapy Now, highlights the need for a national effort to measure outcomes related to regenerative cell therapies, as well as increased federal funding for research at the National Institutes for Health (NIH) and expanded capacity at the Food and Drug Administration (FDA) to support this rapidly growing field. The report is based on insights shared by several leaders during a September 2019 event on Capitol Hill hosted by Alliance for Cell Therapy Now in collaboration with the Regenerative Medicine Foundation and the Cord Blood Association.

Regenerative cell therapies represent the next generation of treatments that are showing great promise in cardiology, neurology, oncology, orthopedics, osteoarthritis, and wound healing. Several well-designed clinical trials are now being conducted under FDA-approved investigational new drug protocols. At the same time, some clinics have caused patient harm or made questionable claims, taking advantage of vulnerable patients and casting a negative light on this promising field.

The 21st Century Cures Act contained several provisions to make safe and effective regenerative cellular therapies available to patients, and the FDA and the NIH have taken several steps to advance and support the field. However, additional actions are needed to help bring safe and effective therapies to patients.

Leaders representing FDA, NIH, the Duke University School of Medicine and Cord Blood Association, the Georgia Institute of Technology, the Marcus Foundation, the Regenerative Medicine Foundation, Sanford Health, the Wake Forest Institute of Regenerative Medicine, and Alliance for Cell Therapy Now, participated in the event.

To read the entire report, go to http://allianceforcelltherapynow.org/wp-content/uploads/2019/12/Regenerative-Cell-Therapies-Alliance-for-Cell-Therapy-Capitol-Hill-Briefing-Sept-2019-1.pdf

To view speaker slides and a webcast of the event, visit http://allianceforcelltherapynow.org/events/.

SOURCE Alliance for Cell Therapy Now

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New Report Calls for Measurement of Outcomes and Federal Funding for Research and Increased Capacity at FDA to Advance Safe and Effective Regenerative...

Based on promising results in an early clinical trial, Mayo Clinic has formed a new joint venture with materials engineering firm W.L. Gore & Associates to spearhead a new therapy using stem cells to repair a painful tissue problem stemming from Crohn's disease.

Mayo and Gore on Tuesday announced the formation of a for-profit company called Avobis Bio ("a vobis" is Latin for "by you"), based in Delaware, where Gore is also based. The privately held company will draw on the expertise of scientists at Mayo and Gore to launch a second-phase clinical trial in the hopes of eventually offering the treatment commercially.

A laboratory director at Mayo Clinic said Avobis Bio's therapy, if successful, may be a first-of-its-kind in health care, involving the delivery of a person's own mesenchymal stem cells on a synthetic "scaffold" that biodegrades over time, eventually leaving behind only native tissue sealing a wound. The first application of the technology is treatment of a health problem called perianal fistulae. But if successful, Avobis Bio may one day offer a variety of tissue and organ-repair therapies combining Mayo's stem cell expertise and Gore's medical materials.

"This is a completely new approach, where we are trying to leverage what the body can do for itself," said Allan Dietz, co-director of the Human Cell Therapy Lab in Mayo's Center for Regenerative Medicine.

Mesenchymal stem cells can naturally convert into other kinds of tissue, like muscle or bone. For the Avobis Bio therapy, the cells are harvested from a biopsy of a person's body fat and cultivated at a Mayo laboratory to high purity. No one knows whether the cells deposited into the wound directly convert into scar tissue, or if the stem cells trigger genetic signals that cause other cells in the surrounding tissue to begin the healing process.

"We provide stem cells in the right frame, at the right time, for the body to recognize the signals that it should begin the healing process," Dietz said. "I think in some ways, it was a required simple first step but it appears to be a major step."

Gore is perhaps best known to the public for its Gore-Tex outerwear, but the privately held $3.7 billion engineering and manufacturing firm sells products in an array of industries, including a line of medical devices designed to repair nonnatural holes in body organs. Mayo has used Gore-made devices for many years.

Several years ago, physician-researchers at the not-for-profit Mayo Clinic in Rochester grew keenly interested in a Gore device called the Bio-A Fistula Plug, a flexible bioabsorbable plug made from a material similar to dissolving stitches.

The plug can be used to repair unnatural canals that form between a person's anal canal and their outer skin, after Crohn's disease weakens surrounding tissues. These canals, also known as perianal fistulae, are painful, disruptive and difficult to treat, doctors said. For patients with Crohn's disease, lifetime incidence of perianal fistulae ranges between 23% and 38%, according to past studies.

In 2017, Mayo announced first-in-human results of their experimental therapy treating Crohn's patients' perianal fistulae using a Gore Bio-A Fistula Plug coated with the patient's own stem cells. The study, run in consultation with the Food and Drug Administration, provided the open-label treatment to a small group of patients whose fistulae had not responded to treatment for a median time of six years.

After initial results proved encouraging, the trial eventually enrolled 20 people. Of the 19 who remained in the trial for at least a year, 76% experienced healing of their fistulae, according to results announced by researchers but not yet published in a journal. If validated in a larger clinical trial, that rate of healing would be dramatically better than outcomes under existing treatments, the companies said.

"We have done work in the past looking at combining cells and materials. For us, the clinical trial results from Mayo were incredibly compelling," said Tiffany Brown, a Gore employee and general manager of Avobis Bio. "It is a challenge to translate how cells behave in the lab to how they will behave in patients. So having that proof in real patients really got the conversation going on how we could work together."

If the therapy is proved safe and effective in larger trials, Brown said about 50,000 Crohn's patients per year could be eligible to get it for perianal fistulae. Although Gore is phasing out general sales of its Bio-A Fistula Plug, the device will be supplied exclusively to Avobis Bio.

Mayo and Gore declined to reveal financial details for Avobis Bio, except to note that both parties are contributing to the limited-liability joint venture. The company has a five-member board of managers, with Mayo appointing two members and Gore appointing three.

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Mayo Clinic, maker of Gore-Tex outerwear are teaming up in Crohn's fight - Minneapolis Star Tribune

(Reuters) - Privately held drug developer Enzyvant said on Thursday the U.S. Food and Drug Administration declined to approve its regenerative tissue therapy for a rare immunodeficiency disorder and raised concerns about its manufacturing.

The company was hoping to win its first approval for the therapy, RVT-802, aimed at treating congenital athymia, a disorder affecting babies born without a small gland called thymus, which produces T-cells needed to regulate the immune system.

The health regulator in a letter to Enzyvant raised questions about the manufacturing process for the treatment as well other issues based on its inspection of the manufacturing site, the company said.

Many of these are topics that we are very aware of and we anticipated those to be post-marketing commitments, not approval requirements, Chief Executive Officer Rachelle Jacques told Reuters on a phone call.

The good news is theres no requirement for us to foresee any additional animal studies or any additional clinical trials.

The company said it had planned to manufacture its treatment through a third-party manufacturer.

If approved, RVT-802 would have been the first to win an FDA nod under the agencys Regenerative Medicine Advanced Therapy (RMAT) designation, granted to drug developers making regenerative therapies for conditions lacking treatment options.

Babies born with congenital athymia - about 17 to 24 cases in the United States every year - usually do not survive beyond the age of two.

RVT-802 is a tissue-based therapy, manufactured by sourcing thymus tissue from infants undergoing heart surgeries unrelated to congenital athymia, and administered only once.

Ten clinical studies spanning over two decades and 85 patients were conducted at North Carolinas Duke University, where the treatment was developed.

Enzyvant is currently owned and entirely funded by Swiss drug developer Roivant, but will become here a subsidiary of Japan-based Sumitomo Dainippon in a deal expected to close next year.

We would anticipate that the deal would close before we could fully address the issues in the (FDA letter), Jacques said.

Reporting by Vishwadha Chander and Tamara Mathias; Editing by Maju Samuel and Shinjini Ganguli

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FDA declines to approve Enzyvant regenerative therapy on manufacturing concerns - Reuters

Circularity Healthcare, LLC Joins Forces with "Behind The Scenes" with Host Laurence Fishburne

MIAMI (PRWEB) December 11, 2019

Circularity is partnering with Telly Award-Winning "Behind the Scenes" to provide regular, ongoing, high-quality content from leading experts in a variety of fields with an initial focus on microcirculation science, regenerative medicine, and advanced wound healing and related symptoms.

"Behind The Scenes with Host Laurence Fishburne" is a public television series that has won numerous awards and delivers precision idea-telling at its best. Circularity is an innovative healthcare organization that is health-bent on their trademarked slogan: "Improving Lives by Improving Blood Flow." Their coming together to bring forward the ideas of modern health science on a stellar entertainment platform can only make for riveting content going forward. Viewers will find themselves in a win-win situation.

Watching informative content via this stylized venue will leave viewers feeling quite satisfied with their television watching experience. It is time well spent and information precisely delivered.

A Little More About Circularity

Circularity is concerned with bringing the very best in healthcare innovation to the public. In so doing, they have manufactured a product called D'OXYVA. This product has a two-pronged approach to health. First, it can be quite effective in reducing the debilitative effects of many of the diseases that are affecting the world today, such as COPD, diabetes, and cardiovascular illnesses. Secondly, Circularity's D'OXYVA can be used in a preventative capacity to improve microcirculation. The concept of microcirculation has far-reaching implications in neurology, oncology, endocrinology, cardiovascular health, respiratory health, dermatology, diabetic wound healing or diabetic wound care and other major fields.

Circularity Healthcare, LLC is the power behind D'OXYVA. This noninvasive trans-dermal and circulatory health technology is just the first product to be offered. Circularity is invested in revolutionizing the healthcare space. They plan to do this by creating cutting edge medical products and procedures that are both patient and physician friendly while being effective in minimizing and eradicating diseases.

What "Behind The Scenes with Host Laurence Fishburne" Will Bring to the Table

Behind The Scenes has been an innovator in bringing information to the forefront in the public television space. The award-winning series features segments on the newest technologies, as well as fresh takes on existing entities, phenomenon, and natural occurrences. The show's website boasts that the television series "highlights the evolution of education, medicine, science, technology and industry through inspiring stories."

Aside from the Emmy-winning and Academy Award nominated actor Lawrence Fishburne as host, the program has an award-winning creative development team. Viewers walk away with a rich knowledge of the subject. Viewers may have known about this subject their whole lives, or it may be about something completely new. Viewers learn an evolving aspect of the topic which keeps the perspective fresh.

The dawning of a new age has appeared with this collaboration. Individuals who want to know more about what the health science field is bringing into our hospitals and doctors' offices will not be disappointed. In today's world, it is imperative that we are advocates for our own health.

Coming Soon: Miami ReLife's Dr. Steven Gelbard

The first series is with Dr. Steven Gelbard, a nationally-famed authority with his ReLife Miami Institute on stem cells. Dr. Gelbard presents D'OXYVA's Nobel Prize-winning science as a regenerative medicine. Dr. Gelbard is involving his direct contacts with top NFL players and other top sports celebrities in the monthly series, along with 2540 top neurosurgeons and other experts working under ReLife.

Consumers might imagine having the ability to receive D'OXYVA and other innovative treatments and non-invasive procedures for chronic wound care amid the luxury of a five-star hotel. Behind The Scenes guest, Dr. Gelbard, a Tufts School of Medicine educated neurosurgeon, makes it happen right now. Medicine has left the hospital building and has become the proactive choice of the health conscious. Viewers can all look forward to learning more about how to live a healthier and more informed lifestyle from this awe-inspiring episode.

According to Norbert Kiss, President and CEO of Circularity Healthcare, this collaboration is door busting. Mr. Kiss tells us, "[We] can offer unprecedented access to this amazing Emmy-winning show called Behind the Scenes with very competitive terms due to our strategic involvement. We welcome any expert."

Don't miss the evolution. It's being televised. Circularity and Behind The Scenes viewers can stay tuned for a mind-fortifying experience!

Circularity Values:

Circularity believes in a long-sought-after goal in health care; people should have access to one health application that solves most of their short and long term health issues without compromising other aspects of their health while doing this quickly, affordably, and without pain.

Circularity develops, manufactures and markets advanced technologies that significantly improve quality of life by improving some of the most essential physiological functions in the body.

About Behind The Scenes With Laurence Fishburne

Behind The Scenes is an award-winning program that highlights new stories and innovative concepts through groundbreaking short-form and long-form documentary presentation. The program, which is anchored by a veteran production team with decades of industry experience, is able to effectively communicate the most critical stories to a wide and diverse audience.

Behind The Scenes with Laurence Fishburne, has established an impressive and heralded career, amassing over one-hundred credits across the varied platforms of stage, television and film. He's well known for major for roles in such films as; John Wick 2, Fantastic 4 Rise of the Silver Surfer, Mission Impossible III, Mystic River, Boyz n the Hood, What's Love Got to Do With It, and Apocalypse Now. On the small screen, the award-winning and versatile actor played compelling roles in shows such as CSI: Crime Scene Investigation, CSI: Miami, CSI: New York and Hannibal. The Behind The Scenes Actor currently stars as Pops on the hit TV comedy Black-ish.

About Circularity Healthcare, LLC

Circularity Healthcare, LLC, located in Los Angeles, CA is a private biotech and medtech products and services company that designs, makes, markets, sells, distributes and licenses its own patented and patent pending technologies, such as its flagship non-invasive deoxyhemoglobin vasodilator product line, D'OXYVA. One of the main mechanisms underlying D'OXYVA's science received the Nobel Prize for Medicine in 2019. Circularity enters into exclusive agreements with manufacturers to launch products and with large and small clinics and hospitals in order to help them enhance their profits and credit profiles with a wide variety of advanced products and services. In addition, Circularity Healthcare assists in the financing of equipment, working capital and also patient financing at industry-leading terms and speed.

For more information, please visit http://www.circularityhealthcare.com, or doctors (Rx only) visit http://wound.doxyva.com and send your general inquiries via the Contact Us page. For specific inquiries contact Circularity Customer Care by phone toll free at 1-855-5DOXYVA or at 1-626-240-0956.

Forward-Looking Information

This press release may contain forward-looking information. This includes, or may be based upon, estimates, forecasts and statements as to management's expectations with respect to, among other things, the quality of the products of Circularity Healthcare, LLC, its resources, progress in development, demand, and market outlook for non-invasive transdermal delivery medical devices. Forward-looking information is based on the opinions and estimates of management at the date the information is given and is subject to a variety of risks and uncertainties that could cause actual events or results to differ materially from those initially projected. These factors include the inherent risks involved in the launch of a new medical device, innovation and market acceptance uncertainties, fluctuating components and other advanced material prices, new federal or state governmental regulations, the possibility of project cost overruns or unanticipated costs and expenses, uncertainties relating to the availability and costs of financing needed in the future and other factors. The forward-looking information contained herein is given as of the date hereof and Circularity Healthcare, LLC assumes no responsibility to update or revise such information to reflect new events or circumstances, except as required by law. Circularity Healthcare, LLC makes no representations or warranties as to the accuracy or completeness of this press release and shall have no liability for any representations (expressed or implied) for any statement made herein, or for any omission from this press release.

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Innovative Collaboration: The Cutting Edge of Medicine Goes - Benzinga

Policymakers around the world are determining how to apply existing regulations to 3D organ printing.

When Selena Gomez suffered from Lupus, her best friend Francia Raisa donated her kidney to her, saving Gomezs life. For decades, people just like Gomez have escaped death due to heroic organ donations, either from living or deceased donors.

Today, 3D printingalso referred to as 3DPmay revolutionize the practice of organ donation. Although 3D printing is currently used to make jewelry, food, and art, it may soon be used for medical solutions such as organ donations and bionic limbs.

As a result, policymakers around the world seek increased regulation of 3DP organs. Yet 3D bioprinting does not clearly fit into existing regulatory frameworks.

Since bioprinting generally falls within the regulatory domain of regenerative medicine, medical devices, and biologic drugs, regulators face the challenge of applying existing rules to this uncertain field. As of now, it is unclear whether policymakers can effectively regulate bioprinting under existing regulations, or if a new, specific regulatory process will be necessary.

In determining how to regulate 3D organ printing, policymakers must juggle many possible concerns. Since the technology is still developing, a lot of uncertainty remains about what the actual risks and ethical concerns are. For example, one ethical concern is that 3DP organs may be available to wealthy people only, while less affluent individuals will be blocked out from using these organs.

Another concern is safety. Since 3DP may require stem-cell technology, and the patients own cells may be used for replication, it is difficult to assess the safety risks. Stem-cell therapy cannot be tested on a large sample of healthy people, which limits effective clinical analysis. Also, 3DP biotechnology may open up new uses beyond 3DP organs, such as enhancement of human capacities for military use. Developers could use the technology to make military officers or even terrorists less vulnerable to injury in battle, but this would open up a whole new challenge for law enforcement and national security.

The U.S. Food and Drug Administration (FDA) focuses on the regulation of 3D printed organs. FDA so far has only released guidance on 3DP, and the recommendations do not cover bioprinting.

A significant concern in the United States is that 3D printed organs do not fit into any clear category of law. First, they are not organs because they are not born alive at any stage of development. Second, they are not drugs because drugs are used orally rather than through an invasive surgery, and drugs are primarily meant to relieve illness while donated organs may completely cure an illness.

Some policymakers in the United States propose regulating 3DP organs as a biological product, defined as a virus, therapeutic serum, toxinor analogous productapplicable to the prevention, treatment, or cure of a disease or condition of human beings.

Research company Biogelx suggests that biological products may be a promising category for printed organs. Within biological products, a 3DP organ is comparable to proteins because to print the organ, clinicians replicate healthy human cells, which include such proteins, says Biogelx. Although existing regulatory frameworks often compare 3DP organs to medical devices, Biogelx asserts that these organs should not be regulated as medical devices. Medical devices are not made of biological material and are often metal or plastic devices that help an individuals standard of life, but 3DP organs are different since they cause a chemical reaction in the body and have the purpose of wholly replacing an existing organ, says Biogelx.

International policymakers are also struggling to find a sufficient regulatory framework. In Canada, Health Canada released draft guidance last year to develop regulations for medical device manufacturers working towards bioprinting. Health Canada has several concerns about bioprinting, and it suggests that manufacturers looking for bioprinting licenses should be required to submit information regarding the use of additives in materials, the verification of the software for the bioprinting design, the method of sterilizing the machines, and the process of safe removal and reuse of bioprinting materials and residues.

Finally, Europes 3DP health technology is regulated by the European Medical Devices Directive, the Active Implantable Medical Devices Directive, and the Invitro Diagnostic Medical Devices Directive. The Medical Devices Directive categorizes bioprinting devices into several risk classes. Across the different classes, devices ranked as higher risk are subjected to third party assessment and more stringent requirements for clinical data. The highest risk class, implantable devices such as 3D organs, requires an independent design dossier review. A design dossier assesses risk, evaluates clinical data, and demonstrates the technologys compliance with regulations and requirements.

Although 3D printing of organs is right around the corner, policymakers around the world lack the information necessary to make regulatory decisions in this space. Different countries have different approaches, but many of the leading nations in 3DP share similar concerns. With more information, regulators will have to decide if existing regulatory frameworks can adequately address the safety concerns of 3D printed organs.

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The Uncertainty of Regulating 3D Organ Printing - The Regulatory Review

Following in the very successful footsteps of its sister companies GE Additive, GE Aerospace, and GE Oil and Gas, now GE Healthcare is starting to seriously look at 3D printing. After signing with Formlabs to streamline 3D printing of anatomical models, the giant is now partnering with Advanced Solutions Life Sciences (ASLS) toadvance the field of 3D biofabrication.

And if GE Healthcare is moving fast, bioprinting is not going any slower. It may be the positive momentum from the latest Termis (the leading regenerative medicine event) in Orlando, or maybe 3dpbm is just in the right place at the right time, but 3D Bioprinting Solutions has also been making some giant leaps recently. Now Advanced Solutions Life Sciences, the company founded by Michal Golway, one of the first pioneers in bioprinting, is going to benefit from GEs powerful distribution and R&D means.

As per the agreement, GE Healthcare will distribute the worlds first integrated 3D bioprinter + confocal scanner (BioAssemblyBot + GE IN Cell Analyzer 6500HS) as part of a strategic R&D and distribution partnership that sets out to personalize tissue regeneration. The integration of IN Cell Analyzer and BioAssemblyBot systems technologies will embed cellular-level assessments into the 3D bioprinting workflow used to create human tissue models.

Bioprinted tissues are small in size and die quickly, due to an inability to engineer small blood vessels the bodys supply network. ASLS patented Angiomics technology enables bioprinted microvessels to self-assemble into functional capillary beds, which deliver nutrients, oxygen, and hormones to the 3D tissue model and remove waste. This partnership would allow life scientists and tissue engineers to quickly design, build and image living, vascularized 3D tissues in a single, agile process.

Printing multi-material 3D objects inside of microwell plates allows scientists to efficiently move away from traditional 2D monocultures on plastic, to 3D discovery and cytotoxicity models that more accurately reflect native biology and disease, said Emmanuel Abate, General Manager of Genomics & Cellular Research, GE Healthcare Life Sciences. By combining this flexibility and precision of the BioAssemblyBot with the image quality and speed of the IN Cell Analyzer 6500 HS confocal screening platform, the prospect of automating high content screening in 3D models can become a reality.

Currently, biopharmaceutical companies test their drugs in 2D models and animal models. Precise 3D models provide a more physiologically relevant environment for drug testing because they mimic human reactions. The power of both of these platforms brings a new level of efficiency, speed and quality with assay designs and 3D biofabrication, said Michael Golway, President & CEO of ASLS.

Traditional 3D bioprinters are not designed for quality or interoperability with the high-throughput screening methods that pharmaceutical developers use to identify drug candidates. This alliance will result in a new product to address this challenge: an integration of GE Healthcare Life Sciences IN Cell Analyzer confocal imaging platform with IN Carta cell analysis software, and ASLS BioAssemblyBot 3D bioprinter with TSIM design software.

For pharmaceutical companies, where the average time to develop a new drug candidate may take over seven years, moving from traditional stage-gate testing processes to a lean, agile workcell for 3D tissue fabrication and assessments will shorten development timelines. The integration between IN Cell Analyzer and BioAssemblyBot enables the automated inclusion of cellular imaging information into the tissue modeling process so that new therapies can be scaled more quickly and effectively.

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GE Healthcare pairs up with Advanced Solutions on regenerative tissue manufacturing - 3DPMN

Gene therapy, once dismissed as too dangerous, has made a comeback, with two products approved in the U.S. since December 2017 and hundreds more in the pipeline. STATs latest report takes a deep dive into a crucial component of these new treatments: the viral vectors used to deliver gene therapies to cells and organs.

As dozens of new gene therapies near the market, we spoke with academic experts, pioneers in the field, and executives with 18 companies, large and small, to identify the most important challenges surrounding the engineering of better vectors, their safety, effectiveness, efficiency, production, and cost and how key players are thinking about overcoming those hurdles.

These engineered viruses are difficult to manufacture, particularly at the massive scale needed for some indications. Scientists are working hard to bring down the cost and speed up the process of making viral vectors, so that all the patients that could benefit from gene therapy will have access to it.

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Beyond the introduction, this report has four major components:

The basics of viral vectors and the history of their development;

Major challenges in the development, manufacturing, and testing of viral vectors, and possible solutions;

A close look at the status of gene therapies in 10 disease categories that are advancing through preclinical studies or are being tested in early-stage clinical trials;

And perspective on the U.S. Food and Drug Administrations approach to regulating viral vectors.

The report The STAT guide to viral vectors, the linchpin of gene therapy is intended for anyone with a strong interest in gene therapy, including biotech executives, investors, scientists, lawyers, policymakers, and patients and families interested in learning more. Our aim is to make the problems, stakes, and possibilities clear to everyone.

To buy the full report, please click here.

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New STAT report explores viral vectors, the linchpin of gene therapy - STAT - STAT

Earlier this year, the US Food and Drug Administration approved the most expensive drug ever to hit the market, a gene therapy for spinal muscular atrophy. SMA is a neuromuscular disorder that, in severe cases, can lead to infant death. The genetic correction is currently used to treat affected newborns, but as symptoms for some types of SMA may appear before birth, an earlier treatment would be potentially more effective.

In a study published December 4 in Molecular Therapy, researchers were able to fix a mutation in the survival motor neuron 1 (SMN1) genewhich causes SMA in humansin mice modelling the disease, while they were still inside their mothers uterus. The treated mice lived longer and had fewer symptoms than untreated animals.

Tippi MacKenzie, a fetal and pediatric surgeon at the University of California, San Francisco, who did not participate in this study, says it is an important paper because it is the first time fetal gene therapy has succeeded in SMA mice. Before you even think about doing something in patients, you have to first do it in the disease model of the mouse . . . so this group has supplied a very important piece to the literature, she adds.

SMN1encodes an essential protein for the maintenance of motor neurons, which are nerve cells in the brain and spinal cord responsible for controlling muscle movement. The result in children with mutations in the gene is the loss of motor neurons, leading to muscle weakness and associated complications. SMA affects one out of every 6,000 to 10,000 babies.

Correcting the SMN1 sequence is a potentially efficient treatment for those born with SMA. Zolgensma, the recently approved medication for this disorder, consists of an intravenous administration of an adeno-associated virus that ferries a functional copy of the SMN1 gene to the brain.

To see if the same fix could be accomplished before birth, the research team tested two different injection methods: one into the placenta (intraplacental or IP) and the other into one of the brain lateral ventricles (intracerebroventricular or ICV). The latter proved to be more effective. By injecting the viral vector into the fetuss brain, the virus will go directly into the cerebrospinal fluid, and it will transduce motor neurons in the spinal cord with a very high efficiency, compared to the IP [injection], says Afrooz Rashnonejad. who participated in this study while working at Ege University in Izmir, Turkey, but has recently moved to Nationwide Childrens Hospital in Columbus, Ohio.

Rashnonejad and her colleagues then monitored the injected mice that were carried to term. Those treated with the vector carrying a functional copy of SMN1 lived a median lifespan of 63 or 105 days (depending on the type of cassette carrying the gene), much longer than untreated SMA mice, which did not survive more than 14 days, but still less than wildtype pups, which had a median lifespan of 405 days. The treated mice were also heavier than untreated mice, but smaller than healthy mice.

The investigators also observed differences at the cellular and molecular levels. SMN protein levels were completely recovered in the brain and spinal cord, and the number of motor neurons was higher in treated animals.

I was just very impressed by what theyve done, says Simon Waddington, a gene therapy researcher at University College London who did not participate in this work, but was one of the reviewers of the paper. He adds that he and other colleagues had previously tried fetal gene therapy on SMA mice, but had failed as it is a technically difficult experiment. So it was really nice to see this group actually did a really good job.

This is the first time viral vectors have been used to successfully boost gene expression in SMA mice before birth. Interventions to edit the genome in utero have been previously used in mice that model other severe genetic diseases. Last year, for instance, Waddington and colleagues used fetal gene therapy to treat mice affected by Gaucher disease, a neurodegenerative disorder that can be fatal for newborns. Other successful attempts include intrauterine gene editing for mice affected by -thalassemia, an inherited blood disorder, and mice suffering a monogenic lung disease that normally results in newborn death.

MacKenzie says that, in a recent national meeting on in utero gene therapy, it was discussed how to move forward with a clinical application to the FDA. We are definitively moving towards that direction, but we dont have a particular application yet, because its still not clear which disease should be the first.

SMA makes a lot of sense because its so severe, MacKenzie adds. But at the same time, the results that are coming out at conferences, she observes, suggest that newborn babies receiving Zolgensma are doing pretty well, better than anybody could have imagined. So its not clear that you have to go before birth. A good candidate, she explains, would be a very rare type of SMA, where the baby dies before birth.

Waddington says that researchers might have to wait for neonatal gene therapy to become standard for certain diseases before using fetal gene therapy in humans. Once we actually understand how efficient this is, and if we come to the point where we discover that the earlier that you go the more effective it is . . . in a human setting, then we may be able to do fetal gene therapy. I think that we are looking at more than five years away before thats even likely to happen, he hypothesizes.

A. Rashnonejad et al., Fetal gene therapy using a single injection of recombinant AAV9 rescued SMA phenotype in mice,Molecular Therapy, 27:212333, 2019.

Alejandra Manjarrez is a freelance science journalist. Email her atalejandra.manjarrezc@gmail.com.

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Fetal Gene Therapy Helps Mice with Spinal Muscular Atrophy - The Scientist

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...

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The Rise Of Patent Wars In Europe's Gene Therapy Space - Law360

An effective andinnovative way to treat people with sickle cell anemia using gene therapy may soon be available thanks to efforts by several pharmaceutical companies, a Bloomberg report says.

Sickle cell anemia, a genetic defect that causes red blood cells to form in theshape ofa sickle, hinders the bodys ability to adequately distribute oxygen. This is due to atypical hemoglobin molecules, which is the protein in blood that transports oxygen. Sickle cell disease can be extremely painful, causing blood cells to get trapped in blood vessels and lead to heart failure, debilitating fatigue, strokes and blood clots.About 100,000 people suffer from sickle cell anemia in the U.S,with African Americansbeing disproportionately affected by this condition.

New developments with gene therapy, however, could work to have a positive impact on these symptoms. One of the innovative manufacturers, Bluebird Bio, stole the show at the annual conference of the American Society of Hematology in Florida. Its product, LentiGlobin, debuted positive results; in 17 patients treated with LentiGlobin,more than 40 percent of the hemoglobin in patients' red blood cells appearedin a healthier form thanks to gene therapy, per the article.

Bluebird isnt the only biotechnology making strides in gene therapies. Another potential treatment being researched is based on the technology called CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats), a gene-editing tool that is being used for a wide range of biomedical applications.

Documented in an NPR report, sickle cell patient Victoria Gray recently became the first person in the U.S. to have billions of her own cells genetically edited with CRISPR and reintroduced into her body. These cells will hopefully produce fetal hemoglobin to compensate for the faulty hemoglobin in Grays red blood cells. The trial is being expanded to include more patients and is being conducted by Vertex Pharmaceuticals and CRISPR Therapeutics of the Boston area.

Current treatments for sickle cell include blood and bone marrow transfusions and medication. Studies on gene therapy treatments have been encouraging so far, but there is more testing to be done before either CRISPR or LentiGlobin hits the market.

Link:
Gene therapy could be a revolutionary new treatment for sickle cell disease - The Hill

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

One of the most popular methods for inserting therapeutic genes into cells to treat disease is to transport them using a virus that has been stripped of its infectious properties. But those noninfectious viruses can still sometimes touch off dangerous immune responses.

A team from Johns Hopkins Medicine is proposing an alternative method for transporting large therapies into cellsincluding genes and even the gene-editing system CRISPR. Its a nano-container made of a polymer that biodegrades once its inside the cell, unleashing the therapy. The researchers described the invention in the journal Science Advances.

The team, led by biomedical engineer Jordan Green, Ph.D., was inspired by viruses, which have many properties that make them ideal transport vehicles. They have both negative and positive charges, for example, which allows them to get close to cells. So Green and his colleagues developed a polymer containing four molecules with both positive and negative charges. They used it to make a container that interacts with the cell membrane and is eventually engulfed by it.

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RELATED: Could a grape-based compound improve gene therapy efficiency?

The Hopkins researchers performed four experiments to prove the nanocontainers would travel into cells and deliver complex therapies once inside. First, they packaged a small protein into the polymer material and mixed it with mouse kidney cells in a lab dish. Using fluorescent tags, they confirmed that the protein made it into the cells. Then they repeated the experiment with a much larger medicinehuman immunoglobulinand observed that 90% of the kidney cells received the treatment.

From there, they made the payload even bulkier, packaging the nanocontainers with the gene-editing system CRISPR. With the help of fluorescent signals, they were able to confirm that CRISPR went to work once inside the cells, disabling a gene 77% of the time.

"That's pretty effective considering, with other gene-editing systems, you might get the correct gene-cutting result less than 10 percent of the time," said graduate student Yuan Rui in a statement.

Finally, the Hopkins researchers injected CRISPR components into mouse models of brain cancer using the polymer nanocontainers. Again they saw evidence that successful gene editing had occurred.

Developing improved methods for gene therapy is a priority in the field. In October, for example, scientists at Scripps Research described a way to use a small molecule called caraphenol A to lower levels of interferon-induced transmembrane (IFITM) proteins, which could, in turn, allow viral vectors to pass more easily into cells. And earlier this year, an Italian team described a method for including the protein CD47 in lentiviral vectors to improve the transferring of therapeutic genes into liver cells.

The next step for Hopkins researchers Rui and Green is to improve the stability of the nanocontainers so they can be injected into the bloodstream. They hope to be able to target them to cells that have certain genetic markers, they reported.

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Hopkins team invents non-viral system for getting gene therapy into cells - FierceBiotech

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

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 - STAT