Updated FDA Policy Likely Enables Near-Term Availability of COVID-19 Test in the U.S.

ENGLEWOOD, CO / ACCESSWIRE / March 17, 2020 / Aytu BioScience, Inc. (AYTU), a specialty pharmaceutical company focused on commercializing novel products that address significant patient needs announced today that the company intends to expedite the U.S. launch of its recently licensed COVID-19 IgG/IgM Point-of-Care Rapid Test in light of yesterday's updated FDA policy on coronavirus diagnostic testing.

On March 16, 2020 the FDA announced, "as part of our ongoing and aggressive commitment to address the coronavirus outbreak, the FDA updated a policy originally issued on Feb. 29 on diagnostic testing for coronavirus (COVID-19) in order to achieve more rapid testing capacity in the U.S. We believe the unprecedented policy set forth in today's updated guidance, which addresses laboratories and commercial manufacturers, will help address these urgent public health concerns by helping to expand the number and variety of diagnostic tests..." Further, the agency made recommendations specifically for test developers stating that "the FDA does not intend to object to the distribution and use of serology tests to identify antibodies to SARS-CoV-2 where the test has been validated" and other labeling conditions are satisfied.

Josh Disbrow, Chief Executive Officer of Aytu BioScience, commented, "While we are still working with FDA on making the COVID-19 Rapid Test available, we believe that this updated policy may enable near-term availability of our recently-licensed point-of-care test. During this public health emergency, we are working tirelessly to satisfy all requirements in order to make the test available to the professional medical community as soon as possible." Disbrow also commented that, "We have already gotten confirmation from the manufacturer that our initial supply is being shipped and is expected to arrive in the U.S. very soon."

The company will continue to provide updates about the near-term availability of the COVID-19 test.

About Aytu BioScience, Inc.

Aytu BioScience is a commercial-stage specialty pharmaceutical company focused on commercializing novel products that address significant patient needs. The company currently markets a portfolio of prescription products addressing large primary care and pediatric markets. The primary care portfolio includes (i) Natesto, the only FDA-approved nasal formulation of testosterone for men with hypogonadism (low testosterone, or "Low T"), (ii) ZolpiMist, the only FDA-approved oral spray prescription sleep aid, and (iii) Tuzistra XR, the only FDA-approved 12-hour codeine-based antitussive syrup. The pediatric portfolio includes (i) AcipHex Sprinkle, a granule formulation of rabeprazole sodium, a commonly prescribed proton pump inhibitor; (ii) Cefaclor, a second-generation cephalosporin antibiotic suspension; (iii) Karbinal ER, an extended-release carbinoxamine (antihistamine) suspension indicated to treat numerous allergic conditions; and (iv) Poly-Vi-Flor and Tri-Vi-Flor, two complementary prescription fluoride-based supplement product lines containing combinations of fluoride and vitamins in various for infants and children with fluoride deficiency.

Aytu recently acquired Innovus Pharmaceuticals, a specialty pharmaceutical company commercializing, licensing and developing safe and effective consumer healthcare products designed to improve men's and women's health and vitality. Innovus commercializes over thirty-five consumer health products competing in large healthcare categories including diabetes, men's health, sexual wellness and respiratory health. The Innovus product portfolio is commercialized through direct-to-consumer marketing channels utilizing the company's proprietary Beyond Human marketing and sales platform.

Aytu's strategy is to continue building its portfolio of revenue-generating products, leveraging its focused commercial team and expertise to build leading brands within large therapeutic markets. For more information visit aytubio.com and visit innovuspharma.com to learn about the company's consumer healthcare products.

Forward-Looking Statement

This press release includes forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, or the Exchange Act. All statements other than statements of historical facts contained in this presentation, are forward-looking statements. Forward-looking statements are generally written in the future tense and/or are preceded by words such as ''may,'' ''will,'' ''should,'' ''forecast,'' ''could,'' ''expect,'' ''suggest,'' ''believe,'' ''estimate,'' ''continue,'' ''anticipate,'' ''intend,'' ''plan,'' or similar words, or the negatives of such terms or other variations on such terms or comparable terminology. These statements are just predictions and are subject to risks and uncertainties that could cause the actual events or results to differ materially. These risks and uncertainties include, among others: the regulatory and commercial risks associated with introducing the COVID-19 Rapid Test, effects of the business combination of Aytu and the Commercial Portfolio and the previously announced, but not yet consummated, merger ("Merger") with Innovus Pharmaceuticals, including the combined company's future financial condition, results of operations, strategy and plans, the ability of the combined company to realize anticipated synergies in the timeframe expected or at all, changes in capital markets and the ability of the combined company to finance operations in the manner expected, the diversion of management time on Merger-related issues and integration of the Commercial Portfolio, the ultimate timing, outcome and results of integrating the operations the Commercial Portfolio and Innovus with Aytu's existing operations, the failure to obtain the required votes of Innovus' shareholders or Aytu's shareholders to approve the Merger and related matters, the risk that a condition to closing of the Merger may not be satisfied, that either party may terminate the merger agreement or that the closing of the Merger might be delayed or not occur at all, the price per share utilized in the formula for the initial $8 million merger consideration in the Merger may not be reflective of the current market price of Aytu's common stock on the closing date, potential adverse reactions or changes to business or employee relationships, including those resulting from the announcement or completion of the Merger, risks relating to gaining market acceptance of our products, obtaining or maintaining reimbursement by third-party payors, the potential future commercialization of our product candidates, the anticipated start dates, durations and completion dates, as well as the potential future results, of our ongoing and future clinical trials, the anticipated designs of our future clinical trials, anticipated future regulatory submissions and events, our anticipated future cash position and future events under our current and potential future collaboration. We also refer you to the risks described in ''Risk Factors'' in Part I, Item 1A of the company's Annual Report on Form 10-K and in the other reports and documents we file with the Securities and Exchange Commission from time to time.

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Aytu BioScience Announces Acceleration of U.S. Availability of COVID-19 IgG/IgM Rapid Test Based on Updated FDA Policy - Yahoo Finance

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They're extremely now. Covetable, even the kind of thing some celebrity would Instagram from their "survival condo" in Kansas. In a series of large photographs, Elaine Whittaker sits for a variety of "face mask selfies" closeup portraits of the artist wearing hand-painted respirators. Cholera makes for a pleasingly haphazard pattern of kidney-shaped blobs. Ebola, that stringy so-and-so, could double for a handwritten logo a black-market YSL dupe. (An extremely busted one, maybe, but masks were the breakout dystopian trend at this year's Paris Fashion Week, after all.)

"A quick little story about the masks," says Whittaker, calling the same afternoon COVID-19 was declared a pandemic. "When I first displayed the photographs in a gallery, there were some people standing way at the back [of the room]. One was saying: 'Oh my god, she put ebola on your face! She has malaria on her face!'"

That was in 2015, when this project (Screened For) debuted at Toronto's Red Head Gallery. Like much of Whittaker's art, the series is meant to connect us with the unseen world of microorganisms in this case, infectious diseases. It wasn't her doomsday prediction of a global outbreak, and it's not a house of bio-horrors images designed to terrify the impressionable,like that panicked duo who thought a photo could give them SARS.

"They read them literally," says Whittaker. "It's interesting, that instinct we have to be fearful."

I wonder how those two are dealing with the news right now. In pictures alone, the story of coronavirus is empty shelves, empty airports,mobbed Costcos. It's people in surgical masks, just like the ones in Screened For. (And to repeat the official advice: save those items for healthcare workers and the ill, please. They help stop the spread of disease.)

It's a time for acting with caution, not panic. But if pictures of COVID-19 or any contagion prick at our reflex to freak out, how do we, as viewers, manage what we're seeing?

Earlier this month, Toronto's Photo Laureate Michle Pearson Clarke raised a few points on the subject of how we're illustrating COVID-19. "I've been thinking about the visual representation of this public health crisis, with the most commonly used images depicting people wearing masks," she wrote on Instagram. "We can't photograph this virus, which perhaps makes it more threatening for some folks."

It's Clarke's job to be thinking about issues of representation, she explains. And since the novel coronavirus was discovered in China, media images of COVID-19 have raised her concern. There's a lot of history to consider when reading images of COVID-19, Clarke says. When humans are facing some new invisible menace, they have a long track record of blaming an easier target, and Asian communities have borne the brunt. To pull one widely cited example, when New York's first confirmed case of COVID-19 was reported in early March, outlets including the New York Times and New York Post initially ran the piece with photos of Chinatown, though the patient caught the virus in Iran. Lazy editorial choices can wind up spreading a racist narrative, one that's textbook "fear of the other."

When Clarke was musing on the visual culture of COVID-19, she didn't Instagram a news photo. Instead, she posted a scientific illustration, a now-familiar 3-D rendering of the virus. "I just Googled COVID-19 that day, just to see what was coming up, and then saw that CDC image," she says. "And that particular illustration it is so stunning. It's gorgeous, you know? But also, right away seeing that image, seeing the kind of spikes on the virus you think about how a virus spreads."

That instant lesson is the power of medical illustration: communicating scientific data as quickly as your brain can process an image (which is as little 13 milliseconds.) And for scientists like Tahani Baakdhah, a PhD student at the University of Toronto, information is her Purell for panic.

"As a science communicator, I like to explain everything using a model," says Baakdhah, who teaches workshops to the general public. Past topics include neuroscience and retinal stem cells (her area of research). Her hand-made visual aids, however, are unique. Baakdhah crochets them.

Unlike the potential threat of contagion, there is absolutely nothing scary about a huggable pink virus with a super-kawaii grin. That's a strategic move on Baakdhah's part. Her Instagram photos are cute-overload clickbait, but the captions summarize the latest research. Says Baakdhah: "People all over the world are talking about this. Why not design a model to explain what COVID is?"

Baakdhah also sells her visual aids on Etsy, which has caused at least one goofy misunderstanding at the post office. (Question: "What are you shipping today?" Answer: "Coronavirus!") As of last week, she'd already made more than 40, and had 20 additional orders in progress.

Etsy, however, has banned all listings that mention COVID-19 or coronavirus, a policy they put in effect earlier this month. As of writing, if you try searching those terms, you'll be served a corporate statement explaining the action. The online marketplace says it's working to protect shoppers from snake-oil salesmen sellers out to hock phony remedies. But the ban's also stymied Baakdhah's educational outreach. Her coronavirus models and patterns are still available through the site (shop name: PurpleLilacAmigurumi) but can't be found if you search the disease.

"You cannot prevent scientists from explaining their science in whatever way they like," she says. "Some scientists like to talk, some scientists like to draw, some scientists like to crochet!"

Some, like California-based biologist David S. Goodsell, prefer painting. He debuted this illustration of a coronavirus on Twitter last month. "Art is the work of transforming fear and pain into beauty," one user replied. Tweeted another: "Would be a good puzzle to do while in quarantine." Luke Jerram, the same American artist behind the "Museum of the Moon" installation that appeared in Edmonton and Toronto (among other cities) in recent years, makes sculptures out of clear glass an instructional choice. Per his website: "Viruses have no colour as they are smaller than the wavelength of light."

"We need to do something to visualize the unknown, because it makes it less threatening and less frightening," says Tagny Duff. But as an artist, not a scientist, Duff approaches the challenge differently.

"It's the unknown that I find really exciting," says Duff. "It's the big question, right? How can we know something we don't see?"

Based in Montreal, Duff says she began researching viruses in 2007. At the time, she was teaching a course on the HIV/AIDS epidemic at Concordia University. "I realized that for all the knowledge I had about the social and cultural aspects of it, I didn't really understand the biological reality of HIV," she explains. And after learning some of the basic science, she was inspired to not just make work about viruses, but with them.

"I really felt this responsibility to not only teach myself but students the deeper levels of what a virus is, and how it can be used as a way to really teach us about different kinds of phenomena."

Living Viral Tattoos, an ongoing project she launched more than 10 years ago, expresses both the actual process of infection and the fear that surrounds it. Using a synthetic retrovirus (Lentivirus), Duff effectively inks lumps of actual skin. (A cosmetic surgery patient donated the samples.) As part of the project, Duff outlines her process in exhaustive detail, explaining the steps through video and written instruction. It's the same methodology used in gene therapy, a treatment for conditions including HIV/AIDS, she explains. But here, dye used to mark the site of a cellular reaction appears as bruised skin or, per the title, a tattoo.

"I wanted to be able to show how this was done, and demystify the fear around it," she says. Like a scientist, information is Duff's top strategy. But, she says: "I'm certainly not a science communicator. I mean, first and foremost, I'm interested in people having an experience, having conversations and having a kind of philosophical encounter with visual objects."

"These 3D graphics of a coronavirus: they're very colourful and easy to look at. And they kind of look not so intimidating," she says. "It's easier for people to look at [a scientific illustration] than, say, people with masks, running away from each other in the metro."

"But it's also very removed from the relationship to us as humans," she continues. And as an artist, that's where she comes in.

For Whittaker, her art invites people to think beyond a disaster scenarioand consider our larger relationship with the environment, including the organisms we can't see. "What I'm interested in is people seeing the natural world," she says. "And they can be enchanted by it, and they can be in awe of it and they can have respect for it."

"Looking at microscopic images, they're absolutely gorgeous. They're just really beautiful," she says. "I love that double-sidedness that right beside this beauty there is this terror and fear that we can't see them. They're invisible and we get sick from them. And that plays in all my work all my work about infectious diseases."

"[I'm] hoping to encourage people to think about what the critical role that microorganisms play in our lives, play on this earth, and how they've been here longer than we have and are due their respect. I want people to think about them culturally, historically, scientifically and I want to empower them to see the world in new and different ways by looking at them."

Beyond fear and crisis, viruses can symbolize change. They invade and transform host cells, sure, but there's a positive spin to consider, too. Facing a pandemic, how do we, as humanity, respond?

There's a strain of that in Duff's work, and her latest project, Wastelands, builds a speculative sci-fi world,suggesting a possible future where we've developed a new sustainable fuel. The stuff's generated using bacteriophages (viruses that attack bacteria). "It's really thinking about how these bacteriophages could be our friends," she says, chuckling. "Viruses could help us survive." Maybe they're not as friendly as one of those crocheted COVID-19s, but still.

As communities work to "flatten the curve," so to speak working from home, limiting travel, cancelling large gatherings, etc. these actions could ripple into our habits going forward. "I know it sounds really utopian," Duff says, "but right now, the coronavirus has been instrumental in reducing carbon emissions around the globe."

That consequence comes with complications, but it's a thought to chew before reacting to the latest COVID-19 update. And reflection is needed if we are going to prevent panic. Maybethis moment is a prompt to seek out art like Whittaker's, for instance: images that might scare people, but ask them to consider where the fear is coming from, too.

"I think there is an important role for art at this time," says Whittaker. "I think it's important to empower people, so that they can see art as a way of aiding them in living through these difficult times."

CBC News is keeping track ofdevelopments in the COVID-19 pandemic. This guide toCOVID-19 and its impact on life in Canada is regularly updated with the latest information.

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What is the role of art in helping us better understand coronavirus? - CBC.ca

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a Team of researchers from the University of Minnesota tested a new approach to growing human blood vessels. It will allow you to get unlimited number of vessels for transplantation, the risk of rejection is minimized.

Scientists have long known that human physiology has a lot to do with the physiology of pigs, so the latter often act as models for the design and testing of various drugs and treatment strategies.

In the new work, the team tried to grow human blood vessels in the body of a pig.

Specialists have got the Mature skin cells of a patient and reprogrammed them into induced pluripotent stem cells (IPSC), giving rise to endothelial cells. The latter, as we lined the inner surface of blood vessels.

the IPSC were injected into the fetal pig, and he was hoisted to a surrogate mother.

In the first phase of the test, the embryo has developed over 27 days. No non-target effects were found, all endothelial cells were of human origin.

the Team believes that proved the viability of its concept, and is now awaiting approval for further study of the embryo in the later stages of pregnancy.

as planned By the scientists, their method permits to obtain a viable piglets with blood vessels that will precisely fit the vessels of each patient awaiting transplantation. Thus, after the operation the man will not need to take immunosuppressants drugs for artificial immunosuppression.

the Authors hope that their approach will help patients with many chronic and incurable diseases requiring organ transplantation, and patients with damage to the peripheral arteries, for example, due to Smoking or diabetes (in many cases this disease leads to amputation of limbs).

Scientific article on the results of this work are presented in the journal Nature Biotechnology.

by the Way, earlier News.Science (nauka.vesti.ru) wrote about the universal acellular blood vessels and the artificial blood vessels from cells of patients and drug administration.

Text: To.Science

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Human blood vessels began to grow in the body of pigs - The KXAN 36 News

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A 40-year-old HIV patient has been declared cured after a promising treatment has left him with no active virus. The man, Adam Castillejo, was the subject of extensive research in early 2019 after doctors failed to find HIV in his body over an 18-month period after previously being diagnosed in 2003.

Castillejo, known by the nickname London Patient lived with the disease for many years, taking medicine to manage it since 2012. That same year he was diagnosed with Hodgkins Lymphoma and later endured a bone marrow transplant. That operation may have ultimately cured him of HIV and appears to have made him only the second person to ever be cured of the disease that causes AIDS.

As ScienceAlert reports, the bone marrow transplant that doctors performed on Castillejo used cells from a donor with a very special genetic quirk. The cells are thought to work against HIV in the body, but there was no guarantee that the transplant would provide any concrete benefits beyond treating the cancer.

However, it appears as though the decision to treat Castillejo with the unique stem cells worked in more ways than one and last year doctors announced they couldnt find the virus in his body after 18 months. At the time, they were hesitant to declare the London Patient cured, but after a new round of testing returned the same results, they are more confident that the active form of the virus has indeed been defeated.

This is a unique position to be in, a unique and very humbling position, Castillejo told the New York Times. I want to be an ambassador of hope.

While this sounds like incredible news and for Castillejo, it certainly is the treatment is not an option for everyone. With cancer limiting their options, doctors used the stem cell transplant as a last resort to keep him alive. Its a serious operation and one that was only performed because Castillejos condition was so dire.

Castillejo and the other HIV patient who had similar results, known as the Berlin Patient, may be uniquely fortunate. The doctors note that there are others who have had the same transplant performed but did not improve as rapidly as the others. There are obviously many factors at work here and as exciting as it is to see a second person cured of this terrible disease, theres a lot more work to be done before we can say HIV has been truly beaten.

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HIV patient appears to be cured after stem cell treatment - New York Post

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A second patient has been cured of HIV after undergoing stem cell transplant treatment, doctors said Tuesday, after finding no trace of infection 30 months after he stopped traditional treatment.

The so-called "London Patient", a cancer sufferer originally from Venezuela, made headlines last year when researchers at the University of Cambridge reported they had found no trace of the AIDS-causing virus in his blood for 18 months.

Ravindra Gupta, lead author of the study published in The Lancet HIV, said the new test results were "even more remarkable" and likely demonstrated the patient was cured.

"We've tested a sizeable set of sites that HIV likes to hide in and they are all pretty much negative for an active virus," Gupta told AFP.

The patient, who revealed his identity this week as Adam Castillejo, 40, was diagnosed with HIV in 2003 and had been on medication to keep the disease in check since 2012.

Later that year, he was diagnosed with advanced Hodgkin's Lymphoma, a deadly cancer.

In 2016 he underwent a bone marrow transplant to treat blood cancer, receiving stem cells from donors with a genetic mutation present in less than one percent of Europeans that prevents HIV from taking hold.

He becomes only the second person to be cured of HIV after American Timothy Brown, known as the "Berlin Patient", recovered from HIV in 2011 following similar treatment.

Viral tests of Castillejo's cerebral fluid, intestinal tissue and lymphoid tissue more than two years after stopping antiretroviral treatment showed no active infection.

Gupta said the tests uncovered HIV "fossils" - fragments of the virus that were now incapable of reproducing, and were therefore safe.

"We'd expect that," he said.

"It's quite hard to imagine that all trace of a virus that infects billions of cells was eliminated from the body."

Researchers cautioned that the breakthrough did not constitute a generalised cure for HIV, which leads to nearly one million deaths every year.

Castillejo's treatment was a "last resort" as his blood cancer would likely have killed him without intervention, according to Gupta.

The Cambridge doctor said that there were "several other" patients who had undergone similar treatment but who were less far along in their remission.

"There will probably be more but they will take time," he said.

Researchers are currently weighing up whether or not patients suffering from drug-resistant forms of HIV might be eligible for stem cell transplants in future, something Gupta said would require careful ethical consideration.

"You'd have to weigh up the fact that there's a 10-percent mortality rate from doing a stem-cell transplant against what the risk of death would be if we did nothing," he said.

Castillejo himself said that the experience had prompted him to come forward and identify himself in order to help spread awareness of HIV.

This is a unique position to be in, a unique and very humbling position," he told The New York Times.

Sharon Lewin, an infectious disease expert at the University of Melbourne and member of the International AIDS Society, said Castillejo's case was "exciting".

"But we need to also place it in context - curing people of HIV via a bone marrow transplant is just not a viable option on any kind of scale," she said.

"We need to constantly reiterate the importance of, prevention, early testing and treatment adherence as the pillars of the current global response to HIV/AIDS."

Agence France-Presse

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Second Person Declared 'Cured' of HIV, With No Trace of Infection After Nearly 3 Years - ScienceAlert

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UPDATE:

SPOKANE, Wash. -- Life-saving cells for alocal father of six are on their way to him from Poland. His family has been panicked after a travel ban was put in place by the Polish Government. They say they were told the status of the transport was stalled, and with time slipping away, they needed immediate action.

Jared Weeks was diagnosed withAcute Myeloid Leukemia back in October. His wife Janet contacted 'Help Me Hayley' on Saturday. On Sunday morning, Janet got word that the cells were on their way. She reached out to many government officials and is still trying to sort how and who helped make this happen for her husband.

"I heard that relief in (my husband's) voice and that's all I needed," she said. "I'm so thankful to everyone who shared the story, sent us prayers. I felt it. I really did. People are so overwhelmingly beautiful."

Janet says her husband will have the stem-cell transplant on Tuesday.

"I will be traveling over to Seattle on Monday evening to be there for his 're-birthday,'" she said of the procedure. "I'm so grateful."

PREVIOUS COVERAGE:

SPOKANE, Wash. -- A local father of six desperately needs help receiving life-saving cells provided by an overseas donor. His family says his life depends on it.

His wife Janet sent our Hayley Guenthner this 'Help Me Hayley' request:

"Dear Help Me Hayley,

My children and I are desperate to save my husband. He was diagnosed with Acute Myeloid Leukemia on 10/15/2019 (on his 42nd birthday of all days) since then he has been in the hospital. At the beginning of February we started our journey to the west side of the state to be under the care of Seattle Cancer Care Alliance and to make a long story short, we are now in the transplant stage of his disease.

My husband, Jared Weeks, went inpatient to the University of Washington Medical Center (UWMC) on behalf of the Seattle Cancer Care Alliance. He started his myeloablative chemo regimen on March 10th with the expectation of receiving an Unrelated Allogeneic Peripheral Blood Stem Cell transplant. He had the highest dose of chemotherapy to eliminate his disease and replace his immune system with a 38-year-old female peripheral blood stem cell donation from Poland. Because of the travel ban put in place by the Polish Government in response to the outbreak of the Novel COVID-19 virus, it is becoming impossible to transport these LIFE-SAVING cells that have been extracted from my husband's donor and brought back to the United States. I have left messages for Senator Cathy McMorris-Rodgers, Governor Jay Inslee, Mayor Woodward and Senator Maria Cantwell. I was able to speak personally with State Senator Shelly Short who is passing on this to some of her contacts in the cabinet. I reached out to the Polish Government agency handling the travel ban restrictions and have spoken with an Overseas Citizen Services Safety Officer out of Krakow Poland at the US Embassy-State Department. The travel ban has been put in place but I have been told that roads are still open as well as trains and planes, but as of midnight tonight (not sure if our time or their time) the borders will be closed until March 25th, and maybe extended depending on the COVID-19 outbreak. The cells have been collected from the donor and we are desperate to get them here. Please help us!! God help us.

My husband, Jared Weeks, was diagnosed with Acute Myeloid Leukemia on October 15, 2019 and is in DIRE need of these stem cells to survive.

We need some assistance from the "powers that be" to get these life-saving stem cells to my husband in Washington State ASAP.

His life depnds on it."

There have many people offering to test to see if they are a local match for Jared. Unfortunately, the family doesn't have the kind of time required to find a new donor.

"They would need to go to bethematch.org , however, it is too late in the game to be a donor for Jared but there are hundreds of others that need this life-saving donation as well," Janet said. "The HLA TYPING that is done can take weeks to complete and for Jared, we don't have that kind of time."

Janet is currently in Spokane with their children. She said she is doing everything she can to stay strong for her husband.

"(Jared) is one heck of a dad," Janet said. "He is hardworking, loves the outdoors, fishing, boating and taking his kids on adventures. He is amazing to us and is the center of gravity for our rather large family. He has been through hell and back with this cancer, and is still trusting God completely."

Seattle Cancer Cancer Care Alliance sent KHQ a statement on Jared and other cancer patients relying on life-saving bone marrow transplants during the COVID-19 outbreak.

"The COVID-19 outbreak is an evolving and fluid situation, and the global medical community is collaborating to address the needs of people who are relying on bone marrow transplants for their treatment and survival.

"Seattle Cancer Care Alliance is evaluating every patient who is currently connected with an international or USA-based donor to ensure we have an alternative solution for their treatment should the need arise.

"We are committed to continuing to coordinate with the National Marrow Donor Program and the World Marrow Donor Association, along with donor representatives in various countries, to prevent potential disruptions of critical medical transport so that every cancer patient has access to the life-saving treatment they need.

"SCCA is dedicated to providing the highest-quality cancer care, and we take that responsibility very seriously. We continue to work very closely with our alliance partners -Fred Hutch, UW Medicine and Seattle Childrens- and sharing our approach and best practices with other transplant centers around the country who may face similar unprecedented challenges."

Read more from the original source:
HELP ME HAYLEY: Spokane father of six receiving life-saving cells from Poland donor - KHQ Right Now

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Revolutionary Gene-Editing Tool

Cardiovascular or heart disease (CVDs) is the leading cause of death across the world. Heart attacks resulting due to CVDs can cause death, and severe damage to cardiac muscle a muscle that forms the wall of the heart in survivors. However, researchers claim that they have discovered stem-cell activated mechanisms that promote healing after a heart attack.

According to the study by researchers from Mayo Clinic, stem cells were found to reverse the damage and restore cardiac muscle back to its condition before a heart attack. Human cardiopoietic cells obtained from stem cells within the bone marrow were found to hone in on damaged proteins and reverse intricate changes that a heart attack caused.

"The response of the diseased heart to cardiopoietic stem cell treatment revealed development and growth of new blood vessels, along with new heart tissue," said Dr. Kent Arrell, first author of the study, in a statement.

For the study, the researchers examined the diseased hearts of mice. The hearts of mice that received human cardiopoietic stem cell therapy were compared with those of that did not. Nearly 4,000 cardiac proteins were identified using a data science technique to map proteins found in the cardiac muscle. Over 10 per cent of the discovered proteins were found to suffer damage as a result of a heart attack.

"While we anticipated that the stem cell treatment would produce a beneficial outcome, we were surprised how far it shifted the state of diseased hearts away from disease and back toward a healthy, pre-disease state," said Dr. Arrell.

While the organs in the human body have the ability to repair their damaged cells, they may be unable to restore the loss entirely, and this holds good for cardiac cells as well. Dr. Andre Terzic, senior author of the study, said: "The extent of change caused by a heart attack is too great for the heart to repair itself or to prevent further damage from occurring."

He explained that upon the administration of cardiopoietic stem cell therapy to mice, a partial or complete reversal of nearly two-thirds of the damage caused by a heart attack was noted. Around 85 per cent of all cellular functional categories struck by the disease responded favorably to the treatment.

According to the World Health Organisation (WHO), CVDs claim nearly 18 million lives every year, which translates to 31 per cent of all deaths. The findings of the study provide an improved understanding of the restoration of heart health using stem cells and provide a framework for wider utilization of stem cell therapy for the treatment of various conditions.

Stressing that the actual mechanism behind the repair of diseased organs by stem cells is poorly understood, Dr. Terzic added: "This study sheds light on the most intimate, yet comprehensive, regenerative mechanisms paving a road map for responsible and increasingly informed stem cell application."

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Stem cells can reverse damage caused by heart attack; repair mechanism discovered: Study - International Business Times, Singapore Edition

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PERTH, Australia Australian stem cell therapy company Mesoblast Ltd. plans to evaluate its allogeneic mesenchymal stem cell (MSC) candidate, remestemcel-L, in patients with acute respiratory distress syndrome (ARDS) caused by coronavirus (COVID-19) in the U.S., Australia, China and Europe.

The company is in active discussions with various governments, regulatory authorities, medical institutions and pharmaceutical companies to implement these activities.

What people are dying of is acute respiratory distress syndrome, which is the bodys immune response to the virus in the lungs, and the immune system goes haywire, and in its battle with the virus it overreacts and causes severe damage to the lungs, Mesoblast CEO Silviu Itescu told BioWorld.

Were going to be evaluating whether an injection of our cells intravenously can tone down the immune system just enough so it gets rid of the virus but doesnt destroy your lungs at the same time.

Recently published results from an investigator-initiated clinical study conducted in China reported that allogeneic MSCs cured or significantly improved functional outcomes in all seven treated patients with severe COVID-19 pneumonia.

We have now looked at our own data in lung disease in adults where half the patients had the same kind of inflammation in the lungs as you get with coronavirus, and our cells significantly reduced the inflammation and significantly improved lung function, Itescu said, noting that he is awaiting emergency use authorization to treat patients under a clinical trial protocol.

In a post-hoc analyses of a 60-patient randomized controlled study in chronic obstructive pulmonary disease (COPD), remestemcel-L infusions were well-tolerated, significantly reduced inflammatory biomarkers, and significantly improved pulmonary function in those patients with elevated inflammatory biomarkers.

Since the same inflammatory biomarkers are also elevated in COVID-19, those data suggest that remestemcel-L could be useful in the treatment of patients with ARDS due to COVID-19. The COPD study results have been submitted for presentation at an international conference, with full results to be submitted for publication shortly.

Mortality in COVID-19-infected patients with the inflammatory lung condition is reported to approach 50% and is associated with older age, co-morbidities such as diabetes, higher disease severity, and elevated markers of inflammation.

Current therapeutic interventions do not appear to be improving in-hospital survival, and remestemcel-L has potential for use in the treatment of ARDS, which is the principal cause of death in COVID-19 infection.

Itescu said he didnt know of any other stem cell companies that were doing this. He said that other companies could try the approach from a research perspective but that Mesoblast has all the patents locked down.

The companys intellectual property portfolio encompasses more than 1,000 patents or patent applications in all major markets and includes the use of MSCs obtained from any source for patients with ARDS, and for inflammatory lung disease due to coronavirus (COVID-19), influenza and other viruses.

Remestemcel-L is being studied in numerous clinical trials across several inflammatory conditions, including in elderly patients with lung disease and adults and children with steroid-refractory acute graft-vs.-host disease (aGVHD).

Mesoblasts stem cell therapy is currently being reviewed by the FDA for potential approval in the treatment of children with steroid-refractory aGVHD. The company submitted the final module of a rolling BLA in January.

Remestemcel-L is being developed for rare pediatric and adult inflammatory conditions. It is an investigational therapy comprising culture-expanded MSCs derived from the bone marrow of an unrelated donor and is administered in a series of intravenous infusions.

The stem cell therapy is believed to have immunomodulatory properties to counteract the inflammatory processes that are implicated in several diseases by down-regulating the production of pro-inflammatory cytokines, increasing production of anti-inflammatory cytokines, and enabling recruitment of naturally occurring anti-inflammatory cells to involved tissues, according to Mesoblast.

Read this article:
Australia's Mesoblast plans to evaluate its stem cell therapy in patients infected with COVID-19 - BioWorld Online

Recommendation and review posted by Bethany Smith

Although most of the news from the world of health and medicine has been quite bleak lately, there are still major strides being made in the sector in an effort to combat the worst illnesses that plague humankind. One such stride was just announced, and its certainly worth celebrating: A second person has been cured of HIV.

In a study published in the medical journal, The Lancet, which comes via Medical News Daily, researchers in London say theyve been able to cure a patient of HIV; meaning the patient tested negative for HIV for an extended period of time (30 months as of March, 2020) despite the lack of antiretroviral therapy.

The person whos been cured, Adam Castillejo, was formerly known only as the London patient in order to protect his identity. But Castillejo, who lives in London, came forward recently, and said that he aims to be an ambassador of hope.

The first person to be cured of HIV, Timothy Ray Brown, an American known originally as the Berlin patient, revealed his identity in 2010, saying that I wanted to do what I could to make [a cure] possible. My first step was releasing my name and image to the public. Brown lived and was treated in Berlin. Incidentally, he is technically the second Berlin patient because the results from treatment of the first one are debatable.

AIDS Policy Project with Timothy Ray Brown (third from left with sunglasses). Griffin Boyce.

Castillejo, as well as Brown, were cured of HIV not by antiretroviral medications, which are often able to drastically mitigate the effects, and transmission rate of, HIV, but rather by stem cell transplants from donor bone marrow. Both Castillejo and Brown hadand may still have, that is unclearcancer along with HIV, and were treated with the stem cell transplants primarily to tackle the former disease. (It seems in Castillejos case doctors and researchers were hoping to cure both simultaneously.)

Both Brown and Castillejo underwent a procedure known as a Hematopoietic stem cell transplantation (or HSCT), which involves injecting bone marrow stem cells from a donor, whos often times a parent or sibling, into the recipients bloodstream. Castillejos HSCT treatment was different from Browns, as well as many others, because it was performed with cells that expressed the CCR5 gene.

A video from the MD Anderson Cancer Center that gives a brief outline of how bone marrow stem cell transplants work.

In Castillejos case, stem cells with genomes that express the CCR5 gene were selected because of the fact that it allows for the production of the CCR5 protein: a protein that makes people far more resistant to HIV-1, which accounts for the vast majority of global HIV infections.

While Castillejo received stem cells that did express the CCR5 gene, Brown did notat least according to the study in The Lancet. In fact, according to a 2017 article in New Scientist (which says that Brown received cells with a mutated CCR5 gene, rather than an unexpressed CCR5 gene), some experts believe the curing of Browns HIV was actually due to a potential side effect of his procedure, known as graft-versus-host disease. According to New Scientist, these experts believe that the donor cells attacked Browns native, HIV-infected immune cells, subsequently killing off the virus.

In Castillejos case, on the other hand, it seems there was no graft-versus-host issue that could account for his diminishment of HIV infection levels beyond whats expected to be detectable. Instead, the authors of the study say that one of the implications here is that the Long-term remission of HIV-1 can be achieved utilizing these kinds of cells. The authors also say this method does not require total body irradiation, which would usually be required in cases like these to weaken a recipients immune system in order to allow them to accept donor cells.

An HIV-infected T cell. NIAID

Unfortunately, it seems the treatment that cured Castillejo of HIV is a nonstarter when it comes to mass deployment. There are fatal side effects associated with HSCT, with host-versus-graft chief among them, and doctors say that it should only be performed when there are no other options left.

Prof. Ravindra Kumar Gupta from the University of Cambridge in the U.K., the lead author of the study, told Medical News Daily that [Its] important to note that this curative treatment is high risk and only used as a last resort for patients with HIV who also have life threatening hematological [blood] malignancies.

But Gupta and the other authors of the study still appear to be optimistic that this stands as a proof-of-concept for the idea of using CCR5 gene editing to cure HIV on a larger scale. They warn in their study, however, that several barriers, including the need for increased gene editing efficiency and a lack of robust safety data, still stand in the way of something that could be used as a scalable strategy for tackling HIV.

What do you think about this method of treating HIV? Do you think gene editing will play a big role in curing HIV, or do you think there are other, more promising treatments worth pursuing instead? Let us know your thoughts in the comments.

Feature image: C. Goldsmith / Eliot Lash

More:
A Second Person Has Been Cured of HIV - Nerdist

Recommendation and review posted by Bethany Smith

By: Cobey Bartels

Date: 16.03.2020

Friend of Owner//Driver and owner of the iconic Filthy White 4000 we covered last year, Mick Moo Lake, was diagnosed with cancer on Christmas Eve.

Mick and Mel Lake, with 'Filthy'

Since the devastating news, Mick and his wife Mel have been struggling to balance treatment and operating their business Truckin Stainless setting up a GoFundMe page to help cover the costs of hospital care.

Mick has Double Myeloma, a form of cancer that develops from plasma cells in the bone marrow, and has been receiving ongoing treatment since New Years Eve.

In 10 weeks Mick is having a Stem Cell Blood Transfusion, which will put him out of work for at least a month and will put significant strain on his business and familys livelihood.

At the time of writing, the GoFundMe page has raised $3,195 and Mick and Mel say the money will go towards covering the cost of treatment and help keep their business afloat.

Mick has been working through the treatment, where possible, to keep Truckin Stainless kicking along with the help of his good mate Steve who travelled down from Mackay to help lighten the load.

"He heard about what was going on when he was down here at the time, and he decided to stay and help while I was getting treatment," Mick says.

"Ive been working through it, going to hospital for treatments then back to the workshop - i havent stopped.

"In respect of all the wonderful people that have had to endure this terrible disease, we understand that asking for donations seems a bit steep, but if you would like to donate please do so, or think to donate to any cancer foundation," he says.

You can donate or find out more HERE.

You can also follow our updates by liking us on Facebook.

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Moo trucks on through cancer battle - Owner//Driver

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VANCOUVER, Washington, March 16, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY), ("CytoDyn" or the "Company"), a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announced today that the Company filed a modified IND and protocol for its Phase 2 clinical trial with leronlimab (PRO 140) as a therapy for patients who experience respiratory complications as a result of contracting Coronavirus Disease 2019 (COVID-19). The modification came at the behest of the U.S. Food and Drug Administration (FDA) in response to the Company's filing of its IND and protocol for its Phase 2 clinical trial on March 9, 2020.

The modified protocol is a Phase 2, Randomized, Double Blind, Placebo Controlled Study to Evaluate the Efficacy and Safety of Leronlimab in Patients with Coronavirus Disease 2019 (COVID-19) and calls for 75 planned patients in up to 10 centers in the United States. Patients enrolled in the trial are expected to have a treatment window of approximately 6 weeks.

Jacob Lalezari, M.D., CytoDyn's Interim Chief Medical Officer commented: "We appreciate the FDA's timely input on protocol design and hope to start the treatment study in the very near future."

Nader Pourhassan, Ph.D., president and chief executive officer of CytoDyn said: "We are pleased with the new trial design and are rapidly sending supplies of leronlimab to the clinics to begin patient treatment."

With respect to the Company's press release issued on March 12, 2020, management announces the following correction: Patient #1 had missed one treatment of carboplatin and will continue to be treated with carboplatin and leronlimab. Additional updates about this patient will be included in the next Company update on all cancer patients.

About Leronlimab (PRO 140) The U.S. Food and Drug Administration (FDA) have granted a "Fast Track" designation to CytoDyn for two potential indications of leronlimab for deadly diseases. The first as a combination therapy with HAART for HIV-infected patients and the second is for metastatic triple-negative breast cancer. Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases including NASH. Leronlimab has successfully completed nine clinical trials in over 800 people, including meeting its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard anti-retroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab can significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use. In the setting of cancer, research has shown that CCR5 plays an important role in tumor invasion and metastasis. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is therefore conducting aPhase 1b/2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019. Additional research is being conducted with leronlimab in the setting of cancer and NASH with plans to conduct additionalclinical studies when appropriate.

The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation and may be important in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells. CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to further support the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD and that blocking this receptor from recognizing certain immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted "orphan drug" designation to leronlimab for the prevention of GvHD.

About CytoDynCytoDyn is a biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a key role in the ability of HIV to enter and infect healthy T-cells. The CCR5 receptor also appears to be implicated in tumor metastasis and in immune-mediated illnesses, such as GvHD and NASH. CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard anti-retroviral therapies in HIV-infected treatment-experienced patients. CytoDyn plans to seek FDA approval for leronlimab in combination therapy and plans to complete the filing of a Biologics License Application (BLA) in the first quarter of 2020 for that indication. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients and plans to initiate a registration-directed study of leronlimab monotherapy indication, which if successful, could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV with no reported drug-related serious adverse events (SAEs). Moreover, results from a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients, with some patients on leronlimab monotherapy remaining virally suppressed for more than five years. CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is atwww.cytodyn.com.

Forward-Looking StatementsThis press releasecontains certain forward-looking statements that involve risks, uncertainties and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as "believes," "hopes," "intends," "estimates," "expects," "projects," "plans," "anticipates" and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. The Company's forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i)the sufficiency of the Company's cash position, (ii)the Company's ability to raise additional capital to fund its operations, (iii) the Company's ability to meet its debt obligations, if any, (iv)the Company's ability to enter into partnership or licensing arrangements with third parties, (v)the Company's ability to identify patients to enroll in its clinical trials in a timely fashion, (vi)the Company's ability to achieve approval of a marketable product, (vii)the design, implementation and conduct of the Company's clinical trials, (viii)the results of the Company's clinical trials, including the possibility of unfavorable clinical trial results, (ix)the market for, and marketability of, any product that is approved, (x)the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Company's products, (xi)regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii)general economic and business conditions, (xiii)changes in foreign, political, and social conditions, and (xiv)various other matters, many of which are beyond the Company's control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form10-K, and any risk factors or cautionary statements included in any subsequent Form10-Q or Form8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.

CYTODYN CONTACTSInvestors: Dave Gentry, CEORedChip CompaniesOffice: 1.800.RED.CHIP (733.2447)Cell: 407.491.4498dave@redchip.com

Read more here:
CytoDyn Files Modified IND and Protocol for Phase 2 Clinical Trial for Treatment of Patients with Coronavirus with Leronlimab (PRO 140) and Advises...

Recommendation and review posted by Bethany Smith

Reuben Jacob and Fiona Kellas, Maucher Jenkins share their expertise on IP strategies and considerations for gene therapy inventions.

Gene therapy enables the treatment of a disorder or disease through the insertion of a gene into a patients cells instead of using drugs or surgery.This technique involves the introduction of genetic material into cells to compensate for abnormal genes in the patient or to make protein that will be beneficial to the patient.As an example, if a mutated gene causes a protein that is necessary for the correct functioning of cells to be faulty or missing, gene therapy may be able to introduce a normal copy of the gene to restore the function of the protein.Gene therapy is understood to be useful in the treatment of a range of conditions such as cancer, cystic fibrosis, muscular dystrophy and Alzheimers disease.

UK role in gene therapy techR&D

Gene therapy is considered to be very important to the future of medicine and as such, many companies are focussing their research and development into gene therapy technologies.The UK is a growing industry for research into these areas and it is anticipated that by 2035 the UK industry around cell and gene therapy technologies will be worth in the region of 10 billion.Gene therapy research is still at an early stage.Due to this length of time and the associated costs involved in developing an effective gene therapy and taking it through to approval, it will be important for companies working in this area to put into place an effective IP strategy that will provide protection for their inventions and assist them in maintaining their market position.In addition, the competitive nature of the gene therapy industry means that will be important for a company to obtain patent protection for inventions being developed, as well as reviewing the patent landscape to check that the company is free to operate in their chosen area.

What makes something patentable?

In order for an invention to be patentable, it must be new, inventive and capable of industrial application.In addition to the requirement that an invention meets the above requirements of patentability, it is also important that the invention does not contain subject matter that is excluded from patentability.One of the challenges associated with obtaining patent protection for gene therapy inventions is that the European and US patent systems include a number of exceptions to patentability that are relevant to biological material and natural products.In Europe, it is not possible to obtain patent protection for a method of treatment or surgery of the human body.Thus, the removal of cells from a patient would not be considered to be patentable in Europe.In addition, inventions relating to stem cells that are derived from the destruction of human embryos are not patentable in Europe.In the US, recent case law (Molecular Pathology v Myriad Genetics, Inc, 2013) has meant that inventions relating to natural phenomena and natural products must show characteristics that are different to their natural counterpart(s).

However, despite the above challenges, there are a number of aspects of the gene therapy technology that may be eligible for patent protection.Typically, the gene therapy procedure can involve performing the required modification procedure on cells that have been removed from a patient before reintroducing the cells into the subject to produce their modified effect.The process of modifying the cells may be patentable if it fulfils the above requirements of patentability.In addition, it may be possible to obtain protection for the methods that are used to culture, manipulate or modify the cells that are used for gene therapy.

At Maucher Jenkins, we have a team of attorneys who can provide IP advice and assistance in the area of patenting inventions involving gene therapy, molecular biology and biochemistry.

by Fiona Kellas, Reuben Jacob

16 March 2020

14:20

Original post:
Thinking out loud: IP strategies for gene therapy inventions - Med-Tech Innovation

Recommendation and review posted by Bethany Smith

The Center for Gene Therapy at Nationwide Childrens Hospital is working to develop childrens gene therapy treatments. Officials say the gene therapy research and clinical trials there are starting to attract companies to central Ohio.

Nationwide Childrens Hospital is in the forefront of curing several genetic childhood diseases, transforming Columbus into a major medical hub, several gene therapy experts say.

The hospital's Center for Gene Therapy at the Abigail Wexner Research Institute is working to develop treatments for children, which is attracting patients and companies to Ohio, according to officials at Nationwide Childrens and JobsOhio, the state's economic development organization.

The illnesses that were making use of in gene therapy are devastating illnesses, said Dr. Kevin Flanigan, the director of Nationwide Childrens Center for Gene Therapy. These are ones we know that children would be significantly impaired for life or die because of the disease.

Gene therapy involves altering the genes inside the patient's cells in an effort to treat or stop disease. It gives doctors the chance to treat many previously untreatable rare and genetic diseases.

Gene therapy is currently available primarily in a research setting, with only four gene therapy products approved by the U.S. Food and Drug Administration for sale in the United States. One of the four, Zolgensma, started as a clinical trial for spinal muscular atrophy at Nationwide Childrens in 2014.

The hospital is working on a handful of gene therapy treatments for various childhood diseases that affect muscle, motor or mental functions, Flanigan said.

Gene therapy presents a tremendous opportunity for our medical system, and Columbus has been a huge part of that growth thanks to the work being done at Nationwide Childrens Hospital, Edith Pfister, chairwoman of the American Society of Gene & Cell Therapys communications committee, said in an email.

The FDA approved Zolgensma, a one-time treatment that intravenously delivers the gene that is missing in children with spinal muscular atrophy, on May 24.

SMA is a progressive childhood neuromuscular disease that is caused by a mutation in a single gene that attacks nerve cells. It causes major physical limitations including the inability to breathe, swallow, talk or sit up. Children born with SMA typically die or need permanent breathing assistance by the time they turn 2 years old.

Donovan Weisgarber was diagnosed with SMA type 1 at Nationwide Childrens in November 2015 when he was 5 weeks old. His parents, Matt and Laura Weisgarber, decided to participate in a clinical trial at the hospital and Donovan received Zolgensma.

Before the treatment, Donovan was unable to swallow and had difficulty breathing. Today, the 4-year-old has doubled his life expectancy and is able to talk, sit up, roll over and hold his head up on his own. He also attends the Early Childhood Education and Family Center on Johnstown Road on the East Side, which offers services from the Franklin County Board of Developmental Disabilities.

(Gene therapy) has given us an opportunity that we otherwise wouldnt have to love Donovan and experience him, said Matt Weisgarber, 33, of northeast Columbus.

A lot of people hear Ohio and think flyover state, but now Columbus is going to be a hub of the most groundbreaking science known to mankind and thats a really cool thing, he said.

Boston Childrens Hospital and Childrens Hospital of Philadelphia also have impressive gene therapy centers, but Columbus sets itself apart from those East Coast cities, said Severina Kraner, JobsOhios health care director.

The cost to operate, manufacture and live in Ohio is cheaper than Boston and Philadelphia, putting Ohio in a position to win cell and gene therapy companies, she said.

People are being priced out of these coastal cities, Kraner said.

One of the companies who has committed to building in Columbus is Sarepta Therapeutics, a Massachusetts-based biopharmaceutical company. Sarepta signed an agreement with Nationwide Childrens in May 2019, giving the company the licensing to a gene therapy treatment that came out of hospital research for limb-girdle muscular dystrophies, a group of diseases that cause weakness and wasting of the muscles in the arms and legs.

Sarepta is scheduled to open an 85,000-square-foot Gene Therapy Center of Excellence near Nationwide Childrens Hospital in the fall to do early research for all the companys gene therapy programs. A team of about 30 employees from Sarepta is currently working at a facility at Easton Town Center.

The region has every ingredient needed for a thriving gene therapy cluster: a strong academic foundation, world-renowned research hospitals, and, now, industry investment, Louise Rodino-Klapac, Sareptas senior vice president of gene therapy, said in an email. All of these contribute to creating a pipeline of talented people who will accelerate scientific advances that help patients.

Nationwide Childrens recently also announced it will be expanding its gene therapy research by creating Andelyn BioSciences, a new for-profit subsidiary that will manufacture gene therapy products for the biotechnology and pharmaceutical industries.

Were hoping, and we have a vision, that Andelyn can help capitalize a biotechnology hub in central Ohio focused on developing and advancing gene therapies, said Dr. Dennis Durbin, Nationwide Childrens chief science officer.

Andelyn BioSciences will launch this summer and operate out of the Abigail Wexner Research Institute, 575 Children's Crossroad. Nationwide Children's is trying to secure a permanent location for Andelyn and is looking at land on Ohio State Universitys West Campus.

Gene therapy treatment, however, comes at a high price.

The manufacturer set the price of Zolgensma at more than $2.1 million. Insurers can pay $425,000 a year for five years for one treatment.

Insurance companies are used to regular installment payments, but the single-dose nature of gene therapies are adding a level of uncertainty to health insurance structures, Pfister said in an email. A one-time administration gene therapy costs less overall, but it occurs in one upfront payment.

Pfister said she is hopeful the cost of gene therapy will go down.

Currently, most of the FDA-approved gene and cell therapies are tailored for the specific patient, but theres an incredible amount of research going into standardizing the components and delivery mechanisms behind gene therapy, Pfister said in an email.

Dr. Jerry Mendell helped usher in the era of gene therapy at Nationwide Childrens when he came to the hospital in 2004.

Nationwide Childrens first gene therapy trial was in 2006 for duchenne muscular dystrophy, a rare, inherited, degenerative muscle disorder that almost exclusively affects boys.

Things have really changed significantly in the gene therapy world because of the contributions weve made here, and its been a very gratifying experience, said Mendell, the principal investigator in Nationwide Childrens Center for Gene Therapy.

Read the rest here:
Nationwide Childrens among hospitals leading the way in gene therapy - The-review

Recommendation and review posted by Bethany Smith

Researchers have successfully treated cardiac dysfuntion in mice models of Barth syndrome by using a gene therapy to replace TAZ.

A new study has demonstrated the success of a gene therapy in preventing or reversing cardiac dysfunction in mice models of Barth syndrome.

The research, conducted at Boston Childrens Hospital, US, investigated the mutations in a gene called tafazzin (TAZ), which causes life-threatening heart failure, weakens the skeletal muscles, undercuts the immune response and impairs overall growth in boys.

In 2014, the scientists from the study found that TAZ is essential in causing cardiac dysfunction, using a heart-on-a-chip model. Adding the TAZ gene normalised the heart muscle cells and organised the mitochondria inside these cells.

A gene therapy delivery vector (adeno-associated virus) being taken up in the heart, as shown in green at increasing magnification (credit: adapted from Prendiville TW et al., PLoS One 2015 May 29, https://doi.org/10.1371/journal.pone.0128105).

For the current study, the researchers moved to an animal model. They used two kinds of knockout mice: one with no TAZ in any cells and the other with TAZ absent in just the heart.

Most mice with the whole-body TAZ deletion died before birth, mostly because of skeletal muscle weakness. However some survived and these mice developed progressive cardiomyopathy, in which the heart muscle enlarges and loses pumping capacity. Their hearts also showed scarring similar to human patients with dilated cardiomyopathy, the left ventricles were also dilated and thin-walled.

Mice lacking TAZ just in their cardiac tissue, which all survived to birth, showed the same features. Electron microscopy showed heart muscle tissue to be poorly organised, as were the mitochondria within the cells.

The team then used gene therapy to replace TAZ, injecting an engineered virus under the skin in newborn mice or intravenously in older mice. Treated mice with whole-body TAZ deletions were able to survive to adulthood. TAZ gene therapy also prevented cardiac dysfunction and scarring when given to newborn mice and reversed established cardiac dysfunction in older mice whether the mice had whole-body or heart-only TAZ deletions.

Further tests showed that TAZ gene therapy provided durable treatment of the animals cardiomyocytes and skeletal muscle cells, but only when at least 70 percent of heart muscle cells had taken up the gene.

The problem is that neutralising antibodies to the virus develop after the first dose, said Dr William Pu, director of Basic and Translational Cardiovascular Research at Boston Childrens. Getting enough of the muscle cells corrected in humans may be a challenge.

L-R: Mitochondria from a normal mouse, a Barth syndrome mouse treated with the viral vector only, with no gene and a Barth syndrome mouse treated with a TAZ-carrying gene therapy vector (credit: Wang S; et al., Circ Res 2020 Mar 9; https://doi.org/10.1161/CIRCRESAHA.119.315956).

Maintaining populations of gene-corrected cells is another challenge, say the researchers. While levels of the corrected TAZ gene remained fairly stable in the hearts of the treated mice, they gradually declined in skeletal muscles.

The biggest takeaway was that the gene therapy was highly effective, concluded Pu. We have some things to think about to maximise the percentage of muscle cell transduction and to make sure the gene therapy is durable, particularly in skeletal muscle.

The findings were published in Circulation Research.

See the rest here:
TAZ gene therapy reverses Barth syndrome in mice - Drug Target Review

Recommendation and review posted by Bethany Smith

He swipes once. Twice. Three times.

The lidocaine goes in, to freeze the skin. Then the needle.

Slowly, precisely, Rasquinha removes spinal fluid through the lumbar puncture, commonly known as a spinal tap. He then gives him the injection that Snow and his wife, Kelsie, believe is saving his life.

They believe because they want to, because they have to, and because -- against every single odd, against every single thing you've ever heard regarding amyotrophic lateral sclerosis, or ALS -- it just might be working.

Snow, a 38-year-old assistant general manager for the Calgary Flames, was diagnosed with ALS on June 17. It was not a surprise. His family has been ravaged by the familial form of the disease, with his father, two uncles and a cousin having died from ALS caused by the SOD1 genetic mutation.

ALS affects motor neurons, the cells that control muscle movement. As ALS progresses, the motor neurons die, the muscles become weaker, and eventually movement slows or becomes impossible.

It's what Snow saw happen in his right hand, how he began to suspect that the disease had come for him too. But almost immediately after his diagnosis, which usually carries a life expectancy of between six and 18 months, Snow enrolled in a phase 3 clinical trial at Sunnybrook Health Sciences Centre in Toronto for those with the SOD1 mutation, which affects 2 percent of ALS patients.

In this, he was lucky, both that it was available and that he qualified.

He has made the trip to Toronto with Kelsie every four weeks since then, though initially there was a two-in-three chance that every trip brought him an injection of tofersen, a drug that doctors and researchers hoped could slow the progression of the disease.

For the first six months of the trial, two-thirds of the study participants are given the actual drug. One-third are given a placebo.

He does not know for sure whether he was in the group given the medication or the placebo. The Snows believe he was one of the lucky ones, one of those given tofersen. They believe because they, remarkably, have not seen a progression of the disease since he entered the trial. He still does not have use of his right hand. He has use of everything else, all the things they feared might slip away from him in the weeks and months after they confirmed the diagnosis.

The injection takes two minutes, two minutes of silent meditation for Snow. He thinks about his mom, Linda, who committed suicide in 2012. "Because," as he says, "she'd be really happy and really sad if she were here. One of the things I got from her was a real joy for life. She always was happy that I was that way. So, I think about her and how I get to move on because of this." He thinks about his dad, Bob, who died of ALS in 2018, and his uncles and his cousin.

He wishes they had had the opportunity for these two minutes. For that two-in-three chance.

"I move through those thoughts fairly quickly," he says. "I usually say a prayer or two. For them. Probably in part for myself."

****

The Snows have already been at Sunnybrook for hours at this point, Feb. 20 marking their 11th visit to this hospital, his 10th lumbar puncture. At 9:15 a.m., the driver picks them up at their hotel for the 20-minute ride. When they walk in the doors at the hospital, a massive campus that looks like a small airport, Snow is on edge.

They find their way down to Room UG21, where Snow will undergo a battery of tests, all of them designed to determine whether the progress of the disease has slowed, whether it has stopped, whether it has -- God forbid -- picked up again.

They start with a detailed neurological assessment.

"Have you experienced any changes to your health since you were last here?"

"No."

"Have you noticed any difference in your speech?"

"No."

They test knowledge next, the year, the season, the province, the city. A series of words that never changes from visit to visit: apple, penny, table. He counts backward from 100 by seven.

These are the hardest weeks, when the anxiety sweeps up and the visit looms closer and the fear that "no change" might have turned into "some change" pierces the bubble they have formed around themselves.

"My bad hand, I don't have any expectations for it being better," Snow says. "It's more the opposite, that I'm always kind of concerned about and testing the good parts. The other hand, in particular. Living without one hand is not difficult. Living without two hands is difficult.

"It's testing my good hand, and then I overtire it. And then I convince myself something is wrong."

This is when Kelsie can sense his perpetual optimism faltering.

"None of the physical aspects of any of that, the lidocaine, the needle itself, I couldn't care less about those things," Snow says. "It's just the emotional anguish that you can put yourself through, with wondering, am I a little bit different? And if I'm a little bit different, what does that mean?

"Because that's totally unknown. But then you worry, that could be a slippery slope."

He does a breathing test, to determine lung capacity, and one to test muscle strength. That is the one that gives him the most anxiety, because they are testing his hand and his strength, and that is where the disease started to eat at him.

"This is our 10th time doing most of these tests," Snow says.

"And there has not been any changes," research coordinator Jahan Mookshah says.

"Those are our favorite words," Kelsie says.

Kelsie bends down and ties her husband's shoes. The testing is over, for the moment.

****

There is no blood test for ALS. It's only diagnosed by process of elimination, a factor that often can be problematic, as the disease progresses while the patient is still waiting for answers. Snow was told June 10 by an EMG technician in Calgary that it was likely ALS, a diagnosis that was confirmed a week later by Dr. Michael Benatar at the University of Miami.

It was the last answer they wanted in the world. It was a death sentence.

They crumbled, seeing an unknown present, a future robbed.

"I don't know how we did it," Snow said. "We did our days, and then we cried."

Back in Calgary, fellow assistant general manager Craig Conroy got the news on the phone from Snow, while looking at Snow's kids, Cohen and Willa, now 8 and 5, who were staying at his house while their parents were in Miami. As Conroy said, "That just breaks your heart."

But there was some good news.

Snow had the best-case scenario: a family history and a mutation of the SOD1 gene. He had pressed the issue with doctors, over and over again, after starting to feel the weakness in his right hand while lifting weights during the Stanley Cup Playoffs in April, even as he wanted to believe it was a pinched ulnar nerve, desperate for any answer but this. For Snow, that meant it was only a matter of months between the onset of symptoms and the diagnosis, as opposed to 1 1/2 to 2 years, which is the average.

Which was why when the Snows talked to Benatar on June 17, it was like being flattened and lifted up in the same moment. They confirmed Snow had ALS. They also learned there was a trial, with openings for those with a fast-progressing form of the disease, like him. There was something they could do. There was a place they could go. There was a medicine he could take, produced by Biogen, a company whose corporate headquarters are on Binney Street in Cambridge, Massachusetts, the exact street where Snow lived when he moved there to work at The Boston Globe, another career and another lifetime ago.

There was -- maybe, possibly -- hope. An impossibility in this world.

"It's just like someone believably telling you you're going to die, then telling you you might not die," Kelsie said. "You can't describe that in a more dramatic way than that. You feel like you got your life back. You've got a chance. Just a chance, right?"

Most of the time ALS is sporadic -- that is, not inherited. But in 5 to 10 percent of cases, there is a familial link, an altered gene that has folded. When Kelsie researched trials in the immediate aftermath of the diagnosis, she realized that the studies that were furthest along happened to be for SOD1, Snow's mutation, the second-leading cause of familial ALS.

They would head to Toronto every month, their kids left with a rotating selection of friends, off to get the injection that might -- or might not -- include the liquid hope that his future rested on.

They had six months in which he could have been receiving either the drug or the placebo, until last month, when they were finally assured he would be getting the medication from this point on.

They told their kids that they would have to have a summer to last a lifetime.

"Because we felt like it was maybe going to have to," Kelsie said.

They jammed in everything, all the bike rides and ice cream and pictures, the trip to Merrymeeting Lake in New Hampshire, Snow's family cabin. They jammed happiness in every moment, the tears reserved for private times, for when they couldn't hold it in any longer, wiped away when the kids approached. She wondered if he would make it to February and, if he did, whether he would want to hang around once he got there. She told him to just stay the way he was. They put one foot in front of the other and, still, they don't know exactly how.

By October, they had started to feel differently. Hopeful.

Snow got back on the ice, courtesy of a Flames equipment manager, who sewed his glove into a fist, enabling him to grip a hockey stick. He shot a puck. It rang off the crossbar. Kelsie took a video. It was evident to her that the disease had not progressed, not robbed him of any more strength.

That moment, that month, changed them.

"I felt like that was probably the first moment where I could really breathe," she said.

They went public on Dec. 18, and it ricocheted around the hockey community. Kelsie wrote a letter detailing their diagnosis and their hope, something she has continued to chronicle on her blog, kelsiesnowwrites.com, and recently, in a story that ran in Sports Illustrated.

It has become a piece of her every day, every thought, something that still fells her at a moment's notice. When she is asked if there's been a day that she hasn't thought about ALS since the diagnosis, tears start to flow down her face.

"These three letters are a part of my life forever," Kelsie says. "I sure wish they weren't. But I know that a lot of good things will come to us as a result of this. And I've seen a lot of good in a lot of people because of this.

"There's so much beauty in grief, and there's so much beauty in sadness and tragedy. Because you get to see the best in people. And that's not a small thing. But I wish I didn't have a byline in Sports Illustrated. I don't want a byline in Sports Illustrated. I don't want to be telling this story. But if this is what I'm here for, then I'm OK with that. It's not small. I know that. I know that being there for somebody you love is significant. It's enough for me."

She swipes at the tears, running her fingers underneath her eyes. She sniffles.

"This is because I know you, too," Kelsie says, starting to laugh amidst the tears. "That's the problem."

****

It has been 15 years since I met Kelsie and Chris, back when she was still Kelsie Smith and he was still a baseball writer. The summer of 2005 was one of those summers that's only possible when you are young and there is no responsibility, no ties, no worry. When a White Russian just before the bar closes seems like a good idea, and a shut-off car in a parking lot is the right place to bare a soul, for a friend to reveal she just might be falling in love.

As Kelsie texted me recently, "Honestly, best summer of my life."

They were too young, realistically, for it to work. She was 21. He was 23. They met in a bar, the White Horse Tavern, down the street from the apartment that Snow would buy in Allston, Massachusetts, and that I would later buy from him and live in for the next decade.

He was the Boston Red Sox beat writer at the Globe, she was an intern in the Globe's sports department out of the University of Kansas, and I would be hired into the sports staff that summer as a general assignment reporter.

They got engaged the next summer, and married in December 2007, on a frigid, 9-degree day in St. Paul, Minnesota, where they moved after he was hired as the director of hockey operations for the Minnesota Wild, an unorthodox move that would launch him on a career in hockey and give me a chance to succeed him on the Red Sox beat at the Globe.

She covered the Minnesota Twins for the St. Paul Pioneer Press. We spent spring trainings together in Fort Myers, Florida.

It's hard to think about that day in 2007, so many years ago now, when they promised a lifetime to each other. Because, as Kelsie said, "That I have extra months is not lost on me. I know that I've been already given a gift. It's just that I want what we all thought we were getting when we got married. That we are going to grow old together."

Kelsie has always trusted in Snow, something she has never been shy about expressing. She's always believed he would do what he said, that he would succeed despite all the odds: That he would make the unheard-of jump from baseball reporter to NHL front office member; that he would figure it out after the Wild let him go; that they could find their way on one income (first hers, then his) in a new city, in a new country with a new baby.

And he did. They did.

"It was always like, Chris will figure this out," she said. "And that's probably an unfair amount of pressure that I put on him, but I just believed in him. I've always believed in him that much."

She still does, even in a battle that, up to this point, has been unwinnable.

****

At 1 p.m., after a pizza lunch, it's time to head back to UG21 for the lumbar puncture. Snow laughs about the diet he has been instructed to stick to -- high protein, high fat, high carb -- because losing weight is a marker of the disease. It's one thing he doesn't mind.

This session, too, starts with some tests. Of reflexes. Hands. Ankles. Feet. Jaw.

"This might hurt," Rasquinha says. "Sorry."

Rasquinha flips his hands over, examines them, tells Snow to relax. This is not Snow's forte.

Snow acknowledges yet again that he cannot do anything with his right hand. That went in June, and the atrophy has set in up to his elbow. Kelsie -- or, if he's at work with the Flames, one of his colleagues -- makes sure to cut his food for him, if needed; they tie his skates and his shoes. "Tight, but not too tight," as she puts it.

Testing done, it's time for the lumbar puncture. Snow is now on the open-label extension of the trial. After this visit, he is assured that the fluid sent into his spine will be tofersen. This is a comfort, even as they believe he has been receiving the medication all along.

"All right," Snow says, "let's rock and roll."

The risks are read out, the warnings given, as they always are. The Snows know this nearly by heart. Rasquinha snaps on sterile green gloves and a baby blue face mask. The lidocaine goes in. "Mosquito bite," Rasquinha calls it. "A little burn. Sorry."

Lorne Zinman enters the room. He is part of the reason for their hope, a man who oozes sunshine as he talks, despite having devoted his life to a disease that to this point has claimed every one of its victims.

"This is exciting," says Zinman, the director of the ALS/Neuromuscular Clinic at Sunnybrook and an associate professor of neurology at the University of Toronto. "This is gene therapy for ALS. I've been talking about this for, like, two decades. The fact that it's a reality and we could be helping people like Chris, it's just the happiest thing. I always say -- apology to my children -- the happiest day of my life will be when we finally have something for this disease, because I've seen too many people go through it."

It is easy for Zinman to be joyful these days. He, like all those who work with ALS, has spent years and years in the darkness, and he somehow just might see a little light emerging, a speck that grows bigger with every day that Snow, and potentially others, are stable.

"It's been decades and decades of, really, failure after failure in ALS," Zinman said. "When I give lectures, I put up a slide and I call it the graveyard of failed trials. And it's just failure after failure. The big turning point came when we found the first gene for ALS in the early '90s."

Researchers put the gene into mice, and the mice started to show signs of disease. They thought a cure would come soon after. They were wrong.

Things started to turn when advances in gene therapy led the focus to shift. They knew SOD1 made a protein, and that the protein misfolded, leading to the killing of motor neurons that led to ALS. That became their target.

On Feb. 3, Kelsie posted a video to Twitter showing Snow lifting his right hand at his wrist. It was something he hadn't been able to do since the disease took hold. They don't know what it means, and they try not to read too much into it, but the idea of stability, let alone improvement, sometimes seems like a dream they do not want to end.

And that is what makes Zinman so excited.

"We don't usually see that in ALS trials," he said. "The objective is to slow things down, not to make things improved. It's really exciting when you hear something like that, where I'm actually able to do something that I couldn't do before. You always have to take it with a grain of salt it's just really exciting when you hear something like that."

He envisions a day when a patient like Snow or one of his family members could find out he has the SOD1 mutation, that they could start him on the drugs in the pre-symptomatic phase, as a preventative, similar to the way that doctors have been able to use PrEP to keep HIV from taking hold, or the way that doctors use vaccines to prevent diseases.

"We're diagnosing ALS about five to six times a week, and three to four of our patients die every week," Zinman said. "I've been doing this for 15 years now. I see the faces of these patients, not just them, their family members and what they went through, so to be able to offer someone something like this -- research is hope."

When Zinman and I exited the room, I later learned, Snow grew emotional. He cried. It was joy and relief and release. He had finished another session at Sunnybrook. There had been no change. He soon would be assured of getting more of the medicine that has given him more, where before, he expected there would be less and less.

Because these two minutes, every four weeks, might just be saving him.

"The first few times he was dosed I had full-on panic attacks," Kelsie said. "It was all about the fact that I didn't know what [he was getting]. They'd come in and they'd put this little Ziploc baggie on the desk in there and it had this syringe in it. I remember noticing right away that it was 15 milliliters of this clear fluid. And that was every ounce of hope I had in my life."

****

The appointment over for the month, Snow walks down the corridor, a drab, shabby, beige hallway. It is hardly inspiring, with its fluorescent lights and well-trod flooring. But Snow is nearly bouncing. This is the best he feels all month, his hopes confirmed, the medication, he assumes, flowing through his body.

"Every time I walk this hallway, it's a good feeling," he says. "Because you picture yourself being wheeled."

But he can walk. He can breathe. He can do his job, almost the same way he always has, with a few simple modifications. He wears a suit without a tie these days, because he cannot tie one and, really, who needs it? He has not yet transitioned to slip-on shoes, perhaps out of stubbornness.

Continue reading here:
Gene therapy giving Flames executive Snow hope in ALS fight - NHL.com

Recommendation and review posted by Bethany Smith

The hunt for the elixir of life is such a universal mythological trope that to talk about it in the context of science seems almost ridiculous. But breakthroughs in the last decade have made the impossible seem possible, and researchers are quickly converging on the consensus that aging may well be a disease that we can treat just as easily as any other.

Impressive results in animals that have had their lifespans boosted by up to 40 percenthave started making their way into humans. Some trials are more questionable than others, but most promisingly there seem to be multiple potential avenues, from cocktails of common drugs to gene therapies and stem cell treatments.

Stem cells are particularly promising, because they can be coaxed into becoming any kind of cell before being transplanted to treat damaged tissue. These therapies often fail to work well in older tissue, though, limiting their future use in older patients who could need them most. This appears to be because these tissues have significantly higher levels of inflammation that prevent stem cells from properly integrating.

Now Portuguese researcher Joana Neves has won the 2019 Sartorius & Science Prize for Regenerating Medicine & Cell Therapy for her discovery of a way to sidestep this roadblock and significantly increase the success of stem cell treatments.

Because of the central importance of tissue repair to all organisms, Neves assumed that many of the mechanisms behind it would be shared among all animals. So she started looking at proteins produced by immune cells in the well-known animal model of the fruit fly.

She discovered that a poorly-understood protein known as MANF (mesencephalic astrocyte-derived neurotrophic factor) plays a crucial role in reducing inflammation in fruit flies. More importantly, she found that mice and humans also produce it, and its prevalence reduces in all the species as they age, suggesting it plays a key role in limiting age-related inflammation.

That prompted her to see if introducing MANF would boost the effectiveness of stem cell treatments in older animals. She used the protein in combination with a procedure that uses stem cells to replace degenerating photoreceptors in the retina of older mice and found it greatly improved the restoration of vision.

Going further, her research team then investigated whether MANFs anti-inflammatory effects could have more general age-defying benefits. Previous research had already demonstrated that infusing old mice with blood from younger ones could reduce various signs of aging, and by carrying out similar experiments the team showed that MANF is one of the factors responsible for that outcome. They even showed that directly injecting mice with MANF could have similar effects.

Translating these ideas to treat other diseases and for use in humans will take some time, but the research chimes with work on an emerging class of drugs known as senolytics. These are drugs that kill senescent cells, which are zombie cells that become more prevalent as we age and spew out harmful chemicals that result in chronic inflammation.

Senolytics are generally seen as a broad-spectrum treatment that could help stave off multiple conditions at once, but they could also be used to create a more hospitable environment for stem cell treatments just like MANF.

There are still plenty of barriers to bringing any of these treatments to the clinic, from the difficulty of producing stem cells to the challenges of regulating treatments for aging (a condition we still dont formally class as a disease), or fighting back against the huge number of bogus treatments that threaten to undermine trust in the field. But given the huge potential for near-term impact, theres growing momentum.

Weve moved from being able to extend health and lifespan of simple organisms like yeast and worms and flies to being able to do this quite easily in animals, in mice and monkeys, David Sinclair, director of the Center for the Biology of Aging at Harvard Medical School, told the Harvard Gazette.

He added that instead of trying to treat one disease at a time, he believes we can develop medicines that will treat aging at its source, therefore having a much greater impact on health and lifespan than drugs that target single diseases.

The wheels are in motion for us to find out.

Image Credit: Monika Robak from Pixabay

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To Turbocharge Anti-Aging Treatment, Just Add... a Protein Found in Fruit Flies? - Singularity Hub

Recommendation and review posted by Bethany Smith

MarketandResearch.biz broadcasted a new title Global Hemophilia Gene Therapy Market 2020 by Company, Regions, Type and Application, Forecast to 2025 which delivers in-depth assessment on key market trends, upcoming technologies, industry drivers, challenges, regulatory policies, with key company profiles and strategies of players functioning in the market. Vast coverage of industry players has been analyzed and further compared with the overall sector for each component such as profit, purchases, marketing, utilities, and depreciation. The report has a segmented market, by its types and applications. All segments have analyzed completely on the basis of its production, consumption as well as revenue. It provides forecasts for the global Hemophilia Gene Therapy market from 2020 to 2025.

The report focuses on a competitive analysis of key players by product, price, financial position, product portfolio, growth strategies, and regional presence. Additionally, their company profile, capacity, production value, contact information, and market shares for the company are also covered. Figures, graphs, and flowcharts are used to represent the analyzed data. The research report enlists the information regarding the global Hemophilia Gene Therapy market growth tactics attempted by the industry players, such as expansion strategies and mergers and acquisitions.

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Global Hemophilia Gene Therapy Market 2020 Trending Technologies, Developments, Key Players and Forecast to 2025 - Bandera County Courier

Recommendation and review posted by Bethany Smith

The global Cancer Gene Therapy market research report initiates a comprehensive analysis of the global Cancer Gene Therapy market. It offers an in-depth analysis of the rate of development of the market in the estimated time period. Offering a brief outline, the report explores the influencing factors and size of the global Cancer Gene Therapy market in the estimated period. It also covers the major leading factors restraining the expansion of the global Cancer Gene Therapy market. The global Cancer Gene Therapy market research report emphasizes commanding players in the market linked with their market shares.

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The report offers helpful data that discloses the foremost players in the Cancer Gene Therapy market it also discusses the revenue division, business general idea, and product contributions of the key players in the market. The research study also analyzes the growth of the well-known market performers with the help of SWOT analysis. In addition, while evaluating the growth of main market players, the research report covers their recent business developments. Furthermore, various products and segments

Table of Content

Chapter 1: IntroductionChapter 2: Executive SummaryChapter 3: Market OverviewChapter 4: Cancer Gene Therapy Market, By TypeChapter 5: Cancer Gene Therapy Market, By ApplicationChapter 6: Cancer Gene Therapy Market, By RegionChapter 7: Competition Landscape

The global Cancer Gene Therapy market is bifurcated on the basis various segments. The development of each segment is evaluated along with their predicted growth in the future. Reliable data and statistics are collected from the regulatory authorities to calculate the growth of various segments of the market. Additionally, the global Cancer Gene Therapy market is also divided on the basis of various regions such as the North America, and Europe, Asia Pacific, Latin America, and Middle East & Africa.

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The Global Cancer Gene Therapy Market Anticipated to exhibit a CAGR 32.4% during Forecast Period 2018-2025. - Galus Australis

Recommendation and review posted by Bethany Smith

The Global Gene Therapy Industry Market analysis report published on Upmarketresearch.com is a detailed study of market size, share and dynamics covered in XX pages and is an illustrative sample demonstrating market trends. It covers the entire market with an in-depth study on revenue growth and profitability. The report also delivers on key players along with strategic standpoint pertaining to price and promotion.

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The Global Gene Therapy Industry Market report entails a comprehensive database on future market estimation based on historical data analysis. It enables the clients with quantified data for current market perusal. It is a professional and a detailed report focusing on primary and secondary drivers, market share, leading segments and regional analysis. Listed out are key players, major collaborations, merger & acquisitions along with upcoming and trending innovation. Business policies are reviewed from the techno-commercial perspective demonstrating better results. The report contains granular information & analysis pertaining to the Global Gene Therapy Industry Market size, share, growth, trends, segment and forecasts from 2020-2026.

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About UpMarketResearch:UpMarketResearch (https://www.upmarketresearch.com) is a leading distributor of market research report with more than 800+ global clients. As a market research company, we take pride in equipping our clients with insights and data that holds the power to truly make a difference to their business. Our mission is singular and well-defined we want to help our clients envisage their business environment so that they are able to make informed, strategic and therefore successful decisions for themselves.Contact Info UpMarketResearchName Alex MathewsEmail [emailprotected]Website https://www.upmarketresearch.comAddress 500 East E Street, Ontario, CA 91764, United States.

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Global Gene Therapy Industry Industry 2020 Market Research With Size, Growth, Manufacturers, Segments And 2026 Forecasts Research - Packaging News 24

Recommendation and review posted by Bethany Smith

The one-of-a-kind resource at LivingWithLynch.org offers voices of hope in the face of a Lynch syndrome diagnosis on Lynch Syndrome Awareness Day, March 22, 2020.

In November 2019, 13 Lynch-positive patients, seven women and six men, eight cancer survivors and five previvors, met for the first time in Houston at a weekend sponsored byPromega Corporation. While together they had the opportunity to talk with experts in the field of genetic counseling, oncology, research, treatment, and immunotherapy. They were also given the opportunity to connect with each other and bond over shared experiences.

Lynch syndrome is a genetic condition that increases a person's risk for certain cancers, the most common being colorectal and endometrial, said Dave Dubin, founder of AliveAndKickn, Lynch positive, and colon and kidney cancer survivor. The Living with Lynch weekend was designed to empower patients living with Lynch syndrome to be advocates for themselves, their families, and others with a shared diagnosis. Our goal is to increase awareness of this genetic condition and provide support and hope for those with the Lynch syndrome gene in their family.

The experience is captured atLivingWithLynch.org. The microsite features videos and photos of patients sharing how their Lynch syndrome diagnosis impacts them, their family, and their outlook on the future. AliveAndKickn and the Colon Cancer Coalition hope to help patients Living with Lynch make informed decisions about health and provide a community to relate to as they navigate future health needs.

The Living with Lynch weekend was an amazing and inspiring experience, says Jean Edelstein, Living with Lynch participant, Lynch positive, and a cancer previvor. As a previvor, I know what a Lynch diagnosis means for me, I understand that I have an elevated risk for many cancers in my future. This weekend and meeting these amazing advocates gave me strength to face whatever comes next in my Lynch journey.

It was a privilege to be a part of the Living with Lynch weekend, adds Sarah DeBord, stage IV colon cancer patient (non-Lynch related), communications and program manager for the Colon Cancer Coalition. To hear the stories of individuals and families impacted by Lynch syndrome has opened my eyes even wider to the realities of a hereditary cancer diagnosis. Many individuals with a Lynch mutation develop related cancers much younger than the general population. It is important for individuals to know their family history and begin screening earlier for those with a known Lynch mutation.

About the Colon Cancer CoalitionThe Colon Cancer Coalition is based in Minnesota and dedicated to encouraging screening and raising awareness of colorectal cancer. The organizations signature Get Your Rear in Gear and Tour de Tush event series are volunteer-driven in communities throughout the United States. In 2019, the Colon Cancer Coalition granted over $1 million dollars to local communities to build and sustain programs promoting early prevention, screening, as well as patient support efforts for those living with this disease. By making the words colon, colorectal and colonoscopy a part of the everyday language, we believe we can overcome the fear and decrease deaths from this largely preventable cancer. For more information visitColonCancerCoalition.org.

About AliveAndKicknAliveAndKickns mission is to improve the lives of individuals and families affected by Lynch Syndrome and associated cancers through research, education, and screening. It is the only nationally recognized patient advocacy organization to provide resources, education and awareness for patients with Lynch syndrome hereditary cancer genetic mutations. AliveAndKickn is the go-to patient advocacy resource for patients, clinicians and researchers. In launching the HEROIC patient registry, AliveAndKickn has developed the first RWE patient driven database and worked with the top Lynch syndrome researchers around the country to support and advance research in the field. To read more about AliveAndKickn, please visitwww.AliveAndKickn.org.

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AliveAndKickn and the Colon Cancer Coalition look to empower patients LIVING WITH LYNCH - Curetoday.com

Recommendation and review posted by Bethany Smith

Although anti-CD19 chimeric antigen receptor T-cell (CAR-T)therapy has led to dramatic results in patients with hematological malignancieswho are seemingly out of treatment options, it is far from a panacea for allpatients, as a meaningful portion have disease that either never responds toCAR-T or eventually comes back after treatment with CAR-T.

To develop drugs for these patients who have exhausted yetanother treatment option, drug developers need to know how long these patientssurvive after CAR-T therapy so that they have historical controls in thepostCAR-T setting. These controls are thought to be the most useful asbenchmarks for future trials.

Not a lot is known as to what happens in the patients forwhom CAR-T therapy does not work, James Gerson, MD, assistant professor ofclinical medicine, Perelman School of Medicine, University of Pennsylvania,Philadelphia, told Cancer Therapy Advisor. Anecdotally, we say thatpatients often progress rapidly and arent able to get further therapies, butits not something theres a lot of data for.

With CAR-T therapy coming to the forefront as a treatmentoption, he said a lot of patients are going to get CAR-T therapy earlier onin their treatment course. As a result, drug discovery is most likely going tomove forward in the postCAR-T space because that will be the unmet need.

Researchers from Fred Hutchinson Cancer Research Center and Seattle Cancer Care Alliance, Washington, are helping address this unmet need, specifically for patients with chronic lymphocytic leukemia (CLL).

At the Transplantation & Cellular Therapy Meetings held in early 2020 in Orlando, Florida, these researchers reported the outcomes and characteristics of patients with CLL who received an investigational anti-CD19 CAR-T therapy during a clinical trial (ClinicalTrials.gov Identifier: NCT01865617), but either never had a response or eventually had disease relapse.1 The trial enrolled approximately 200 patients with CD19-expressing CLL, acute lymphoblastic leukemia, or non-Hodgkin lymphoma.

A total of 28 patients with CLL were included in the study,and among these, 16 (57%) had stable or progressive disease and 12 (43%)initially had a response after CAR-T therapy but then had disease relapse aftera median of 11 months.

Patients were retrospectively evaluated and found to havelived a median of 10.4 months after CAR-T therapy. Now we at least have anumber that tells you whats the expected survival if you were to have CAR T cells,said Premal Lulla, MBBS, assistant professor at the Center for Cell and GeneTherapy at Baylor College of Medicine and member of the Dan L DuncanComprehensive Cancer Center at Baylor College of Medicine, Houston, Texas,during an interview with Cancer Therapy Advisor.

Although he was not involved in the study, Dr Lulla saidlarge, multicenter analyses are needed to get a more comprehensive picture ofwhat happens to patients after CAR-T therapy fails.

The study also revealed 2 factors that were predictive ofoutcomes in this patient population. However, lead author Mazyar Shadman, MD,MPH, assistant member, clinical research division at Fred Hutchinson CancerResearch Center, Seattle, Washington, cautioned against overinterpretation ofthe data given that the study was retrospective and had a relatively smallsample size.

The first factor identified was the extent of treatment beforeCAR-T therapy. Specifically, patients whose disease progressed during treatmentwith ibrutinib and treatment with venetoclax before CAR-T therapy lived only amedian of 7 months after CAR-T therapy. In contrast, patients whose diseaseprogressed during treatment with only 1 of these drugs lived a median of 16.4months, an improvement that was determined to be statistically significant (P =.01).

See the original post here:
An Early Look at When CAR-T Therapy Fails Patients With CLL - Cancer Therapy Advisor

Recommendation and review posted by Bethany Smith

Recently, Sangamo Therapeutics (SGMO) announced an extensive collaboration with Biogen (BIIB) to develop gene therapies for neurological diseases, comprising of Alzheimer's disease and Parkinson's disease. Sangamo is to receive a $350M from an upfront payment and proceeds from a stock purchase. The Biogen collaboration will be added to an impressive list that comprises of other big-name players such as Pfizer (PFE) and Gilead (GILD). Sangamos partnerships and collaborations are one of my primary reasons for making an early investment in Sangamo, so, I am even more bullish on SGMO following a collaboration with one of the leading neurological companies in the world.

I intend to review the details of the Biogen deal and why I am even more bullish on SGMO. In addition, take a look at the companys current market valuation in order to defend a long-term investment. Finally, I take a look at the charts to lay out a plan for starting a position in SGMO.

The Biogen collaboration will utilize Sangamo's ZFP technology to control the expression of tau and alpha-synuclein genes that are thought to be responsible for Alzheimer's, Parkinson's, and 10 other neurological targets. In return, Sangamo is expected to receive a $125M upfront payment and $225M in proceeds from a stock purchase at $9.21 per share. In addition, Sangamo is eligible for up to $2.37B in other milestone payments and could claim "high-single-digit to sub-teen double-digit royalties on net sales."

In terms of responsibilities, Sangamo will be take on the early research activities and the development of the AAV delivery vectors and ZFP-TFs. Once Sangoma has finished their preclinical work, Biogen will take over and will take the programs through the regulatory pathway and commercialization.

This collaboration is a significant event for Sangamo for a number of reasons. Personally, I find it extremely bullish that one of the worlds leading neurological companies has tapped Sangamos technology to take on some of the worlds most notorious neurological diseases. In addition, the details of the collaboration show that Biogen is dedicated to Sangamo with an impressive financial commitment and is now one a large shareholder of the company.

If these neurological programs are successful, they could be the first products that actually slow or stop the progression of these diseases with a one-time treatment. Obviously, this would be a medical breakthrough and could change the way we treat some of the worst diseases known to man.

The Sangamo-Biogen collaboration will use the company's ZFP technology to develop genomic therapeutics that switch genes on and/or off in the brain that are responsible for neurological diseases. Sangamo has announced that the ST-501 program will target tau and the ST-502 program will target alpha-synuclein. These programs will employ Sangamo's zinc finger protein transcription factors ZFP-TFs, which is a genome regulation technology that uses adeno-associated viruses AAVs.

ZFP-TF has already shown some success in neurological diseases such as Huntington's and ALS. Sangamo presented preclinical data at the companys R&D Day, which showed that more than half of the ZFP-TFs tested reduced the total alpha-synuclein levels by greater than 50% in ex-vivo cell culture system. So, it looks as if ZFP-TF could address the underlying source, by repressing the genes expression at the DNA level with a one-time administration. As a result, I am feeling optimistic that Sangamo's differentiated ZFP-TF technology will show several benefits over other therapeutic approaches.

In my previous article, I discussed how Sangamo has partnerships and collaborations with some of the most distinguished biotech and pharmaceutical companies in the world onboard (Figure 1). Perhaps the most impressive collaborations are Sangamos hemophilia A gene therapy candidate, SB-525, being partnered with Pfizer (PFE). Sangamo transferred their SB-525 IND to Pfizer, which activated a $25M milestone payment from Pfizer. Now, SB-525 will be entering its late-stage of development and could be the companys first licensed product.

Figure 1: SGMO Partnerships (Source: SGMO)

In addition, the company has a partnership with Sanofi (SNY) for sickle cell disease and beta-thalassemia. Sangamo also has collaborations with Takeda Pharmaceuticals (OTCPK:TKPHF) and Gileads (GILD) Kite Pharma. It is important to note that these partners entered agreements with Sangamo at an early stage of development and were willing to agree to big financial commitments to Sangamo in order to get a piece of the action.

Thus far, Sangamo has brought in around $700M in license fees, milestones, and equity from these partnerships or collaborations. Sangamo could pull in up to a cumulative $6.34B in potential milestone payments from these partnerships.

Looking at the names in figure 1, we can say the company has a knack for attracting and closing major deals. Not only am I bullish about the details of these partnerships, but I also bullish about managements ability and willingness to sign big-name partners. Most developmental biotech/pharma companies would kill to get one of those names associated with their company and technology. Meanwhile, Sangamo has five big names willing to make major commitments before the company has marketed therapies. These partnerships should supply significant milestone payments that can be used to fund the companys impressive pipeline and OpEx. Looking at figure 2, we can see that Street analysts expect Sangamo to start collecting on these milestone payments and eventually royalties in the coming years (Figure 2).

Figure 2: Estimated Annual Earnings (Source: Seeking Alpha)

Currently, the SGMO is fairly valued for its expected 2020 annual revenue, however, it is considerably undervalued when looking at the companys estimated ~$550M annual revenue in 2025, which is a forward price-to-sales of 1.50x. The biotech industrys average price-to-sales of 5x, so we should expect the market to start to recognize the opportunity and the share price will start to be priced in line with the rest of the industry.

Can the company hit $550M? I would point to the estimated $6.5B in potential milestone payments. The company has only collected about $700M, which leaves over $5B of potential milestones to get dispersed in the coming years, therefore, $550M in revenue isnt impossible.

What is more, the company expects their near-term cash from Biogen, combined with the company existing cash to be sufficient to potentially get through Pfizer's first BLA filing of SB-525 for Hemophilia A and beyond. So, it possible that we have little-to-no dilution in the coming quarters.

This historic market sell-off has hammered small-cap biotechs and SGMO has not been immune. Admittedly, the charts are far from looking bullish, so I am waiting for SGMO to show some signs of support before hitting the buy button.

Figure 3: SGMO Daily (Source: Trendspider)

I am looking for a double or triple bottom to ensure a level of support has been formed. Once the stock is able to clear the high of the formation before committing to a buy. Although SGMOs valuation points to the buy, I am not going to bind my cash to a ticker that is being dragged down with the rest of the market.

Disclosure: I/we have no positions in any stocks mentioned, but may initiate a long position in SGMO over the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Original post:
Sangamo: Adding Another Partnership To The Bull Thesis - Seeking Alpha

Recommendation and review posted by Bethany Smith

Newswise What gets the leader of the NIH jazzed?

Speaking to a packed West Pavilion auditorium March 6, Francis Collins, M.D., Ph.D., director of the National Institutes of Health, shared his picks of 10 areas of particular excitement and promise in biomedical research. (Watch the full talk here.)

In nearly every area, UAB scientists are helping to lead the way as Collins himself noted in several cases. At the conclusion of his talk, Collins addedhis advice for young scientists. Here is Collins top 10 list, annotated with some of the UAB work ongoing in each area and ways that faculty, staff and students can get involved.

1. Single-cell sequencing

[see this section of the talk here]

I am so jazzed with what has become possible with the ability to study single cells and see what they are doing, Collins said. They have been out of our reach now we have reached in. Whether you are studying rheumatoid arthritis, diabetes or the brain, you have the chance to ask each cell what it is doing.

Single-cell sequencing and UAB:Collins noted that Robert Carter, M.D., the acting director of the National Institute of Arthritis and Musculoskeletal and Skin Diseases, was a longtime faculty member at UAB (serving as director of the Division of Clinical Immunology and Rheumatology). For the past several years, UAB researchers have been studying gene expression in subpopulations of immune cells inpatients with rheumatoid arthritis.

Join in:Researchers can take advantage of the single-cell sequencing core facility in UABsComprehensive Flow Cytometry Core, directed by John Mountz, M.D., Ph.D., Goodwin-Blackburn Research Chair in Immunology and professor in the Department of Medicine Division of Clinical Immunology and Rheumatology.

Learn more:Mountz and other heavy users of single-cell sequencing explain how the techniqueslet them travel back in time and morein this UAB Reporter story.

2. New ways to see the brain

[See this section of the talk here]

The NIHsBRAIN Initiativeis making this the era where we are going to figure out how the brain works all 86 billion neurons between your ears, Collins said. The linchpin of this advance will be the development of tools to identify new brain cell types and circuits that will improve diagnosis, treatment and prevention of autism, schizophrenia, Parkinsons and other neurological conditions, he said.

Brain tech and UAB:Collins highlighted thework of BRAIN Initiative granteeHarrison Walker, M.D., an associate professor in the Department of Neurology, whose lab has been developing a more sophisticated way to understand the benefits of deep brain stimulation for people with Parkinsons and maybe other conditions, Collins said.

Join in:UABs planned new doctoral program in neuroengineering would be the first of its kind in the country.

Learn more:Find out why neuroengineering is asmart career choicein this UAB Reporter story.

3. Induced pluripotent stem (iPS) cells

[See this section of the talk here]

Researchers can now take a blood cell or skin cell and, by adding four magic genes, Collins explained, induce the cells to become stem cells. These induced pluripotent stem (iPS) cells can then in turn be differentiated into any number of different cell types, including nerve cells, heart muscle cells or pancreatic beta cells. The NIH has invested in technology to put iPS-derived cells on specialized tissue chips. Youve got you on a chip, Collins explained. Some of us dream of a day where this might be the best way to figure out whether a drug intervention is going to work for you or youre going to be one of those people that has a bad consequence.

iPS cells at UAB:Collins displayed images of thecutting-edge cardiac tissue chipdeveloped by a UAB team led by Palaniappan Sethu, Ph.D., an associate professor in the Department of Biomedical Engineering and the Division of Cardiovascular Disease. The work allows the development of cardiomyocytes that can be used to study heart failure and other conditions, Collins said.

Join in:UABs biomedical engineering department, one of the leading recipients of NIH funding nationally, is a joint department of the School of Engineering and School of Medicine. Learn more about UABsundergraduate and graduate programs in biomedical engineering, and potential careers, here.

Learn more:See howthis novel bioprinterdeveloped by UAB biomedical researchers is speeding up tissue engineering in this story from UAB News.

4. Microbiome advances

[See this section of the talk here]

We have kind of ignored the fact that we have all these microbes living on us and in us until fairly recently, Collins said. But now it is clear that we are not an organism we are a superorganism formed with the trillions of microbes present in and on our bodies, he said. This microbiome plays a significant role not just in skin and intestinal diseases but much more broadly.

Microbiome at UAB:Collins explained that work led by Casey Morrow, Ph.D., and Casey Weaver, M.D., co-directors of theMicrobiome/Gnotobiotics Shared Facility, has revealed intriguing information abouthow antibiotics affect the gut microbiome. Their approach has potential implications for understanding, preserving and improving health, Collins said.

Join in:Several ongoing clinical trials at UAB are studying the microbiome, including a studymodifying diet to improve gut microbiotaand an investigation of the microbiomes ofpostmenopausal women looking for outcomes and response to estrogen therapy.

Learn more:This UAB News storyexplains the UAB researchthat Collins highlighted.

5. Influenza vaccines

[See this section of the talk here]

Another deadly influenza outbreak is likely in the future, Collins said. What we need is not an influenza vaccine that you have to redesign every year, but something that would actually block influenza viruses, he said. Is that even possible? It just might be.

Influenza research at UAB:Were probably at least a decade away from a universal influenza vaccine. But work ongoing at UAB in the NIH-fundedAntiviral Drug Discovery and Development Center(AD3C), led by Distinguished Professor Richard Whitley, M.D., is focused on such an influenza breakthrough.

Join in:For now, the most important thing you can do to stop the flu is to get a flu vaccination. Employees can schedule afree flu vaccination here.

Learn more:Why get the flu shot? What is it like? How can you disinfect your home after the flu? Get all the information atthis comprehensive sitefrom UAB News.

6. Addiction prevention and treatment of pain

[See this section of the talk here]

The NIH has a role to play in tackling the crisis of opioid addiction and deaths, Collins said. The NIHs Helping to End Addiction Long-term (HEAL) initiative is an all-hands-on-deck effort, he said, involving almost every NIH institute and center, with the goal of uncovering new targets for preventing addiction and improving pain treatment by developing non-addictive pain medicines.

Addiction prevention at UAB:A big part of this initiative involves education to help professionals and the public understand what to do, Collins said. The NIH Centers of Excellence in Pain Education (CoEPE), including one at UAB, are hubs for the development, evaluation and distribution of pain-management curriculum resources to enhance pain education for health care professionals.

Join in:Find out how to tell if you or a loved one has a substance or alcohol use problem, connect with classes and resources or schedule an individualized assessment and treatment through theUAB Medicine Addiction Recovery Program.

Learn more:Discover some of the many ways that UAB faculty and staff aremaking an impact on the opioid crisisin this story from UAB News.

7. Cancer Immunotherapy

[See this section of the talk here]

We are all pretty darn jazzed about whats happened in the past few years in terms of developing a new modality for treating cancer we had surgery, we had radiation, we had chemotherapy, but now weve got immunotherapy, Collins said.

Educating immune system cells to go after cancer in therapies such as CAR-T cell therapy is the hottest science in cancer, he said. I would argue this is a really exciting moment where the oncologists and the immunologists together are doing amazing things.

Immunotherapy at UAB:I had to say something about immunology since Im at UAB given that Max Cooper, whojust got the Lasker Awardfor [his] B and T cell discoveries, was here, Collins said. This is a place I would hope where lots of interesting ideas are going to continue to emerge.

Join in:The ONeal Comprehensive Cancer Center at UAB is participating in a number of clinical trials of immunotherapies.Search the latest trials at the Cancer Centerhere.

Learn more:Luciano Costa, M.D., Ph.D., medical director of clinical trials at the ONeal Cancer Center, discusses the promise ofCAR-T cell therapy in this UAB MedCast podcast.

Assistant Professor Ben Larimer, Ph.D., is pursuing a new kind of PET imaging test that could give clinicians afast, accurate picture of whether immunotherapy is workingfor a patient in this UAB Reporter article.

8. Tapping the potential of precision medicine

[See this section of the talk here]

The All of Us Research Program from NIH aims to enroll a million Americans to move away from the one-size-fits-all approach to medicine and really understand individual differences, Collins said. The program, which launched in 2018 and is already one-third of the way to its enrollment goal, has a prevention rather than a disease treatment approach; it is collecting information on environmental exposures, health practices, diet, exercise and more, in addition to genetics, from those participants.

All of Us at UAB:UAB has been doing a fantastic job of enrolling participants, Collins noted. In fact, the Southern Network of the All of Us Research Program, led by UAB, has consistently been at the top in terms of nationwide enrollment, as School of Medicine Dean Selwyn Vickers, M.D., noted in introducing Collins.

Join in:Sign up forAll of Usat UAB today.

Learn more:UABs success in enrolling participants has led to anew pilot study aimed at increasing participant retention rates.

9. Rare diseases

[See this section of the talk here]

Rare Disease Day, on Feb. 29, brought together hundreds of rare disease research advocates at the NIH, Collins said. NIH needs to play a special role because many diseases are so rare that pharmaceutical companies will not focus on them, he said. We need to find answers that are scalable, so you dont have to come up with a strategy for all 6,500 rare diseases.

Rare diseases at UAB: The Undiagnosed Diseases Network, which includes aUAB siteled by Chief Genomics Officer Bruce Korf, M.D., Ph.D., is a national network that brings together experts in a wide range of conditions to help patients, Collins said.

Participants in theAlabama Genomic Health Initiative, also led by Korf, donate a small blood sample that is tested for the presence of specific genetic variants. Individuals with indications of genetic disease receive whole-genome sequencing. Collins noted that lessons from the AGHI helped guide development of the All of Us Research Program.

Collins also credited UABs Tim Townes, Ph.D., professor emeritus in the Department of Biochemistry and Molecular Genetics, for developing the most significantly accurate model of sickle cell disease in a mouse which has been a great service to the [research] community. UAB is now participating in anexciting clinical trial of a gene-editing technique to treat sickle cellalong with other new targeted therapies for the devastating blood disease.

Join in:In addition to UABs Undiagnosed Diseases Program (which requires a physician referral) and the AGHI, patients and providers can contact theUAB Precision Medicine Institute, led by Director Matt Might, Ph.D. The institute develops precisely targeted treatments based on a patients unique genetic makeup.

Learn more:Discover how UAB experts solved medical puzzles for patients by uncovering anever-before-described mutationandcracking a vomiting mysteryin these UAB News stories.

10. Diversity in the scientific workforce

[See this section of the talk here]

We know that science, like everything else, is more productive when teams are diverse than if they are all looking the same, Collins said. My number one priority as NIH director is to be sure we are doing everything we can to nurture and encourage the best and brightest to join this effort.

Research diversity at UAB:TheNeuroscience Roadmap Scholars Programat UAB, supported by an NIH R25 grant, is designed to enhance engagement and retention of under-represented graduate trainees in the neuroscience workforce. This is one of several UAB initiatives to increased under-represented groups and celebrate diversity. These include several programs from theMinority Health and Health Disparities Research Centerthat support minority students from the undergraduate level to postdocs; thePartnership Research Summer Training Program, which provides undergraduates and especially minority students with the opportunity to work in UAB cancer research labs; theDeans Excellence Award in Diversityin the School of Medicine; and the newly announcedUnderrepresented in Medicine Senior Scholarship Programfor fourth-year medical students.

Join in:The Roadmap program engages career coaches and peer-to-peer mentors to support scholars. To volunteer your expertise, contact Madison Bamman atmdbamman@uab.eduorvisit the program site.

Learn more:Farah Lubin, Ph.D., associate professor in the Department of Neurobiology and co-director of the Roadmap Scholars Program,shares the words and deeds that can save science careersin this Reporter story. In another story, Upender Manne, Ph.D., professor in the Department of Pathology and a senior scientist in the ONeal Comprehensive Cancer Center, explains how students in the Partnership Research Summer Training Program gethooked on cancer research.

Follow this link:
Looking to the future with Dr. Francis Collins - Newswise

Recommendation and review posted by Bethany Smith

SARASOTA, Fla. and NOVATO, Calif., March 16, 2020 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc.Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare diseases, today announced that it has dosed the first patient in its KIK-AS (Knockdown of UBE3A-antisense in Kids with Angelman Syndrome) study of GTX-102, an experimental antisense oligonucleotide being evaluated for the treatment of Angelman syndrome (AS).

The Phase 1/2 open-label, multiple-dose, dose-escalating study will enroll 20 patients to evaluate the safety, tolerability, and potential efficacy of GTX-102 in pediatric patients with Angelman syndrome. This is the first investigational study testing an antisense oligonucleotide as a potential therapy to treat AS. Further details can be referenced at: https://clinicaltrials.gov/ct2/show/NCT04259281.

Today is an important milestone with the dosing of the first patient in the KIK-AS study, stated Dr. Scott Stromatt, GeneTxs Chief Medical Officer GTX-102 has the potential to address the underlying deficiency that causes Angelman syndrome and we are excited, grateful and humbled to be leading this scientific quest. We look forward to the results of this study and sharing them with the Angelman community.

The GeneTx team has achieved a tremendous accomplishment rapidly advancing this program into the clinic, and GTX-102 may one day provide patients with Angelman syndrome with the first targeted therapy and a potentially transformative option for this devastating disease, said Camille L. Bedrosian, M.D., Chief Medical Officer of Ultragenyx.

Chicagos Rush University Medical Center is the first clinical site to begin enrolling patients in the KIK-AS study, with additional sites being planned in Boston, Cincinnati, Denver, Los Angeles, New York and Ottawa, Canada.

When I held the syringe with this investigational treatment in my hand to inject it for the first time, I thought about the scientific advances in genomic and molecular medicine to produce potential treatments that bring hope of changing the disease course and ameliorating severity of symptoms in those with Angelman syndrome, said Elizabeth Berry-Kravis, site principal investigator at Rush. This is an exciting time for the field of neurodevelopmental disorders as we embark on a path to understanding the outcomes of treatments directed at correcting the underlying molecular causes of disease.

Pending additional site activation, GeneTx Biotherapeutics expects to report preliminary data from the first cohorts in the study in early 2021.

About Angelman Syndrome

Angelman syndrome is a rare, neurogenetic disorder caused by loss-of-function of the maternally inherited allele of the UBE3A gene. The maternal-specific inheritance pattern of Angelman syndrome is due to genomic imprinting of UBE3A in neurons of the central nervous system, a naturally occurring phenomenon in which the maternal UBE3A allele is expressed and the paternal UBE3A is not. Silencing of the paternal UBE3A allele is regulated by the UBE3A antisense transcript (UBE3A-AS), the intended target of GTX-102. In almost all cases of Angelman syndrome, the maternal UBE3A allele is either missing or mutated, resulting in limited to no protein expression. This condition is typically not inherited but instead occurs spontaneously.

Individuals with Angelman syndrome have developmental delay, balance issues, motor impairment, and debilitating seizures. Some individuals with Angelman syndrome are unable to walk and most do not speak. Anxiety and disturbed sleep can be serious challenges in individuals with Angelman syndrome. While individuals with Angelman syndrome have a normal lifespan, they require continuous care and are unable to live independently. Angelman syndrome is not a degenerative disorder, but the loss of the UBE3A protein expression in neurons results in abnormal communications between neurons. Angelman syndrome is often misdiagnosed as autism or cerebral palsy. There are no currently approved therapies for Angelman syndrome; however, several symptoms of this disorder can be reversed in adult animal models of Angelman syndrome suggesting that improvement of symptoms can potentially be achieved at any age.

About GTX-102

GTX-102 is an investigational antisense oligonucleotide designed to target and inhibit expressionof UBE3A-AS. Nonclinical studies show that GTX-102 reduces the levels of UBE3A-AS and reactivates expression of the paternal UBE3A allele in neurons of the CNS. Reactivation of paternal UBE3A expression in animal models of Angelman syndrome has been associated with improvements in some of the neurological symptoms associated with the condition. GTX-102 has been granted Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA). InAugust 2019, GeneTx and Ultragenyx announced a partnership to develop GTX-102, with Ultragenyx receiving an exclusive option to acquire GeneTx.

About GeneTx Biotherapeutics

GeneTx Biotherapeutics LLC is a startup biotechnology company singularly focused on developing and commercializing a safe and effective antisense therapeutic for the treatment of Angelman syndrome. GeneTx was launched by FAST, a patient advocacy organization and the largest non-governmental funder of Angelman syndrome research. GeneTx licensed the rights to antisense technology intellectual property from The Texas A&M University System in December 2017.

AboutUltragenyx Pharmaceutical Inc.

Ultragenyx is a biopharmaceutical company committed to bringing patients novel products for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyxs strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the Company's website atwww.ultragenyx.com.

Ultragenyx Forward-Looking Statements

Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations regarding plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, including our partnership with GeneTx, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainties inherent in the clinical drug development process, such as the regulatory approval process, the timing of regulatory filings and approvals (including whether such approvals can be obtained), and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations and the availability or commercial potential of our products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Annual Report filed on Form 10-K with theSecurities and Exchange CommissiononFebruary 14, 2020, and its subsequent periodic reports filed with theSecurities and Exchange Commission.

Contacts:

GeneTxPaula Evans630-639-7271Paula.Evans@GeneTxBio.com

Ultragenyx Investors & MediaDanielle Keatley415-475-6876dkeatley@ultragenyx.com

See the original post here:
GeneTx and Ultragenyx Announce First Patient Dosed in Phase 1/2 Clinical Trial of GTX-102 in Patients with Angelman Syndrome - BioSpace

Recommendation and review posted by Bethany Smith

HOUSTON--(BUSINESS WIRE)--Kuur Therapeutics today announced the launch of its new business in partnership with Baylor College of Medicine and Baylors Center for Cell and Gene Therapy. Houston-based Kuur Therapeutics will advance the work of its predecessor, Cell Medica, to develop anti-cancer therapies using its innovative chimeric antigen receptor natural killer T cell (CAR-NKT) therapy platform.

IP Group, Baylor College of Medicine and Schroeder Adveq are investing to support two phase 1 studies: GINAKIT 2 (autologous CAR-NKT cells in neuroblastoma) and ANCHOR (allogeneic CAR-NKT cells in CD19 malignancy). The ongoing GINAKIT2 study is now enrolling patients at the third dose level and the ANCHOR study IND has recently been approved by the FDA, with first patient treatment expected in 1H 2020. The funding will also support the preclinical development of an allogeneic CAR-NKT product for treatment of hepatocellular carcinoma, ahead of an IND submission anticipated in 1H 2021.

In conjunction with the new investment, Kevin S Boyle, Sr, was named CEO of Kuur, succeeding Chris Nowers. Mr Boyle joined Cell Medica as CFO in February 2018. Kevin previously held senior finance roles at both NASDAQ-listed and private equity backed companies. He is an accomplished capital markets professional, having raised over $2.0 billion in equity and debt capital.

Kuurs novel CAR-NKT platform is a next-generation technology of engineered immune cells with enhanced functions for the treatment of hematological and solid tumors. It utilizes the unique properties of NKT cells, a specialized type of innate lymphocyte, which shares properties with both T and NK cells. This platform, developed in the laboratory of Baylor Principal Investigator Leonid Metelitsa, is exclusively licensed to Kuur by its partner and collaborator, Baylor College of Medicine.

Annalisa Jenkins, Chair of Kuurs Board, said: We are fortunate to have Kevin step up to the role of CEO. He will act as a change agent, leading the company during a crucial period for our clinical trials and working to secure the additional capital required to progress our two lead CAR-NKT products through the clinic.

The Board would also like to thank Chris for his exceptional leadership during a transition period that has resulted in a company on the right path forward for its investors, with a focus on its important collaborations.

Kevin S Boyle, Sr, Kuurs CEO, said: I am excited to lead Kuur Therapeutics at such a pivotal moment. We are making final preparations to take our off-the-shelf program into the clinic and believe the allogeneic approach holds huge promise for unlocking the potential of CAR therapies for large patient populations. Compared with patient-specific autologous CAR products, it is immediately available for treatment and less expensive to manufacture.

Leonid S. Metelitsa, BCM Principal Investigator, said: My goal is to make a difference in the lives of cancer patients, especially children, and Im excited to be working with the Kuur team to make this goal a reality. I believe that the NKT-cell platform technology, developed in my laboratory and progressed to first-in-human clinical testing in close collaboration with colleagues at BCM and Texas Childrens Hospital, offers a unique route to next-generation off-the-shelf CAR therapies for a broad range of malignancies.

- ENDS -

Notes to Editors

About Kuur Therapeutics

Kuur Therapeutics, headquartered in Houston, is a clinical-stage biotechnology company focused on transforming the treatment of solid and hematological cancers by developing next generation chimeric antigen receptor-natural killer T cell (CAR-NKT) therapies. Developing a portfolio of primarily allogeneic therapies, the companys revolutionary platform spanning both hematological and solid tumors is being created in partnership with Baylor College of Medicine and Baylors Center for Cell and Gene Therapy.

For further information, please visit http://www.kuurtx.com. Follow Kuur Therapeutics on LinkedIn

About Kuurs CAR-NKT cell technology

One of the challenges with allogeneic therapies is that infusing a patient with donor-derived lymphocytes can induce graft versus host disease (GvHD), a potentially life-threatening condition in which the infused cells recognize the patients tissues as foreign. The NKT cells used in Cell Medicas CAR-NKT platform have an invariant T cell receptor (iTCR) that does not distinguish between self- and non-self tissues, making them unlikely to induce GvHD when given to another person. Kuurs CAR-NKT cells are also engineered to secrete IL-15, to prolong persistence and enhance anti-tumor activity.

Baylor has previously reported evidence of clinical activity and a good safety profile in neuroblastoma patients treated at low dose levels of autologous CAR-NKT cells at the 2019 American Society for Gene and Cell Therapy conference.

About Baylor College of Medicine

Baylor College of Medicine (www.bcm.edu) in Houston is recognized as a premier academic health sciences center and is known for excellence in education, research and patient care. It is the only private medical school in the greater southwest and is ranked 16th among medical schools for research and 5th for primary care by U.S. News & World Report. Baylor is listed 21st among all U.S. medical schools for National Institutes of Health funding and number one in Texas. Located in the Texas Medical Center, Baylor has affiliations with seven teaching hospitals and jointly owns and operates Baylor St. Lukes Medical Center, part of CHI St. Lukes Health. Currently, Baylor trains more than 3,000 medical, graduate, nurse anesthesia, physician assistant and orthotics students, as well as residents and post-doctoral fellows.

Follow Baylor College of Medicine on Facebook (http://www.facebook.com/BaylorCollegeOfMedicine) and Twitter (http://twitter.com/BCMHouston).

About the Baylor College of Medicine Center for Cell and Gene Therapy

The Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children's Hospital, and Houston Methodist Hospital in Houston, Texas is led by Helen E Heslop, MD, DSc (Hon), Director and Malcolm K Brenner, MD, PhD, Founding Director. The Center for Cell and Gene Therapy provides an infrastructure to rapidly translate novel cell and gene therapy protocols from the laboratory to the clinic. The comprehensive approach of the center brings a wide variety of scientists and clinicians together to develop strategies for the treatment of cancer, HIV and cardiovascular disease. Patient facilities include the adult stem cell transplant unit at Houston Methodist Hospital and the pediatric stem cell transplant unit at Texas Children's Hospital.

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Kuur Therapeutics Launches to Develop and Commercialize Off-the-shelf CAR-NKT Cell Therapies Targeting Hematological and Solid Tumors - Business Wire

Recommendation and review posted by Bethany Smith