A team of researchers at Osaka University in Japan successfully transplanted cardiac muscle cells created from iPS into a patient, who is now recovering in the general ward of the hospital.

The team, led by Yoshiki Sawa, a professor in the university's cardiovascular surgery unit, created the cardiac muscle cells from iPS cells in a clinical trial to verify the safety and efficacy of this type of procedure. The researches want to transplant heart muscle cells into ten patients who have serious heart malfunctions because of ischemic cardiomyopathy over a three year period.

RELATED: RESEARCHERS ORGANIZE STEM CELLS BASED ON A COMPUTATIONAL MODEL

Instead of replacing the heart of patients, the researchers developed degradable sheets of heart muscle cells that were placed on the damaged areas of the heart.

To grow the heart muscle cells in the lab, the researchers turned to induced pluripotent stem cells otherwise known as iPS. Researchers are able to take those iPS cells and make them into any cell they want. In this case, it was heart muscle cells.If the clinical trials prove successful it could remove someday the need for heart transplants.

I hope that (the transplant) will become a medical technology that will save as many people as possible, as Ive seen many lives that I couldnt save, Sawa was quoted at a news conference reported the Japan Times.

As for the patient, the team plans to monitor him during the next year to ascertain how the heart muscle cells perform. According to the Japan Times, the researchers opted to conduct a clinical trial instead of a clinical study because they want approval from Japan's health ministry for clinical application as soon as possible.

The report noted that during the trial the researchers will look at risks, probabilities of cancer and the efficacy of transplanting 100 million cells for each patient that could include tumor cells.

Continued here:
Heart Muscle Cells Made in the Lab Successfully Transplanted into Patient - Interesting Engineering

Recommendation and review posted by Bethany Smith

In what is a world-first and potentially the dawn of a new medical technology to treat damaged hearts, scientists in Japan have succeeded in transplanting lab-grown heart cells into a human patient for the first time ever. The procedure is part of a cutting-edge clinical trial hoped to open up new avenues in regenerative medicine, with the treatment to be given to a further nine patients over the coming years.

The clinical trial harnesses the incredible potential of induced pluripotent stem cells (IPSCs), a Nobel Prize-winning technology developed at Kyoto University in 2006. These are created by first harvesting cells from donor tissues and returning them to their immature state by exposing them to a virus. From there, they can develop into essentially any cell type in the body.

Professor Yoshiki Sawa is a cardiac surgeon at Osaka University in Japan, who has been developing a technique to turn IPSCs into sheets of 100 million heart muscle cells, which can be grafted onto the heart to promote regeneration of damaged muscles. This was first tested on pigs and was shown to improve organ function, which led Japans health ministry to conditionally approve a research plan involving human subjects.

The first transplantation of these cells is a huge milestone for the researchers, with the operation taking place earlier this month and the patient now recovering in the general ward of the hospital. The sheets are biodegradable, and once implanted on the surface of the heart are designed to release growth factors that encourage new formation of healthy vessels and boost cardiac function.

The team will continue to monitor the first patient over the coming year, and over the next three years aims to carry out the procedure on a total of 10 patients suffering from ischemic cardiomyopathy, a condition caused by a heart attack or coronary disease that has left the muscles severely weakened.

I hope that [the transplant] will become a medical technology that will save as many people as possible, as Ive seen many lives that I couldnt save, Sawa said at a news conference on Tuesday, according to The Japan Times.

Source: The Japan Times

Excerpt from:
Lab-grown heart cells implanted into human patient for the first time - New Atlas

Recommendation and review posted by Bethany Smith

Three-dimensional printers have now assembled candy, clothing, and even mouse ovaries. But in the next decade, specialized bioprinters could begin to build functioning human organs in space. It turns out, the minimal gravity conditions in space may provide a more ideal environment for building organs than gravity-heavy Earth.

If successful, space-printed organs could help to shorten transplant waitlists and even eliminate organ rejection. Though they still have a long way to go, researchers at the International Space Station (ISS) hope to eventually assemble organs from adult human cells, including stem cells.

The medical field has only recently embraced 3D printing in general, particularly in biomedical fields like regenerative medicine and prosthetics. So far, these printers have produced early versions of blood vessels, bones, and different types of living tissue by churning out repeated layers of bioinka substance comprised of living human cells and other tissue thats meant to mimic the natural environment that surrounds growing organs.

Recently, researchers are finding that Earth might not be the best environment for growing freestanding organs. Because gravity is constantly pushing down on these delicate structures as they grow, researchers must surround the tissues in scaffolding, which can often debilitate the delicate veins and blood vessels and prevent the soon-to-be organs from growing and functioning properly. Within microgravity, however, soft tissues hold their shape naturally, without the need for surrounding supportan observation thats driven researchers to space.

And one manufacturing lab based in Indiana thinks its tech could play a key role in space. The 3D BioFabrication Facility (BFF) is a specialized 3D printer that uses bioink to build layers several times thinner than human hair. It cost about $7 million to build and employs the smallest print tips in existence.

The brainchild of spaceflight equipment developer Techshot and 3D printer manufacturer nScrypt, the BFF headed to the ISS in July 2019 aboard the SpaceX CRS-18.

Currently, the project focuses on building increasingly thick artificial cardiac tissue and delivering it back to Earth. Once the printed cardiac tissue reaches a certain thickness, it gets harder for researchers to ensure that a printed structures layers effectively grow into one another. Ultimately, though, theyd like the organs to arrive here fully formed.

Printed organs would eventually require vasculature and nerve endings to work properly, though that technology doesnt yet exist.

The next stagetesting heart patches under microscopes and within animalscould span over the next four years. As for whole organs, Techshot claims it plans to begin production after 2025. For now, the project is still in its infancy.

If you were to look at what we printed, it looks very modest, says Techshot vice president of corporate advancement Rich Boling. Its just a cuboid-type shape, this rectangular box. Were just trying to get cells to grow one layer into the next.

Cooking organs like pancakes

Compare the manufacturing process to cooking pancakes, Boling says. The space crew first creates a custom bioink pancake mix with the cells sent from Earth, which they load with syringe-like tools into the BFF.

Researchers then insert a cassette into the BFF containing a bioreactora system that mimics the normal bodily functions essential for growing healthy tissue, like providing nutrients and flushing out waste.

Approximately 200 miles below in Greenville, Indiana, Techshot engineers connect with ISS astronauts on a NASA-enabled secure digital pathway. The linkup allows Techshot to remotely command BFF functions like pump pressure, internal temperature, lighting, and print speed.

Next, the actual printing process occurs within the bioreactor and can take anywhere from moments to hours, depending on the shapes complexity. In the final production step, the cell-culturing ADvanced Space Experiment Processor (ADSEP) cooks the theoretical pancake; essentially, the ADSEP toughens up the printed tissue for its journey back to earth. This step could take anywhere from 12 to 45 days for different tissue types. When completed and hardened, the structure heads home.

The researchers have gone through three testing processes so far, each one getting more exact. This March, theyll begin the third round of experiments.

The bioprinter space race

The BFF lab is the sole team developing this specific type of microgravity bioprinter, Boling says. Theyre not the only ones looking to print human organs in space, though.

A Russian project has also entered the bioprinting space race, however their technique highly differs. Unlike the BFFs bioink layering method, Russian biotechnology laboratory 3D Bioprinting Solutions uses magnetic nanoparticles to produce tissue. An electromagnet creates a magnetic field in which levitating tissue forms the desired structuretechnology that appears ripped from the pages of a sci-fi novel.

After their bioprinter fell victim to an October 2018 spacecraft crash, 3D Bioprinting Solutions rebounded; the team now collaborates with US and Israeli researchers at the ISS. Last month, their crew created the first space-bioprinted bone tissue. Similar to the US project, 3D Bioprinting Solutions aims to manufacture functioning human tissues and organs for transplantation and general repair.

Just because we have the technology to do it, should we do it?

If the 3D BioFabrication Facility prospers in printing working human organs, theyd be subject to thorough regulation here on Earth. The US approval process is stringent for any drug, Rich Boling says, posing a challenge for this unprecedented invention. Techshot predicts at least 10 years for space-printed organs to achieve legal approval, though its an inexact estimate.

Along with regulatory acceptance, human tissue printed in microgravity may encounter societal pushback.

Each country maintains varying laws related to medical transplants. Yet as bioengineering advances into the the final frontier, the international scientific research community may need to shape new guidelines for collaboration among the stars.

As the commercialization of low-Earth orbit continues to ramp up in the next few years, it is certainly true that were going to have to take a very close look at the regulations that apply to that, says International Space Station U.S. National Laboratory interim chief scientist Michael Roberts. And some of those regulations are going to stray into questions related to ethics: Just because we have the technology to do it, should we do it?

Niki Vermeulen, a University of Edinburgh science technology and innovation studies lecturer, has researched the social implications of 3D bioprinting experiments. Like any Earth-bound project, she urges scientists not to get peoples hopes up too early in the process; individuals seeking organ transplants could read about the BFF online and think it could soon be ready to meet their needs.

The most important thing now, I think, is expectation management, Vermeulen says. Because its really quite difficult to do this, and of course we really dont know if its going to work. If it did, it would be amazing.

Another main issue is cost. Like other cutting-edge biotechnology innovations, the organs could also pose a major affordability challenge, she says. Techshot claims that a single space-printed organ could actually cost less than one from a human donor, since some people must pay for a lifetime of anti-rejection meds and/or multiple transplants. Theres currently no telling how long the BFF process would actually take, however, compared to the conventional donor route.

Plus, theres potential health risks for recipients: Techshot chief scientist Eugene Boland says cell manipulation always presents a possibility of genetic mutation. Modified stem cells can potentially cause cancer in recipients, for example.

The team is now working to define and minimize any dangers, he says. The BFF experiment adheres to the FDAs specific regulations for human cells, tissues, and cellular and tissue-based products.

Researchers on the ground now hope to perfect human cell manipulation: Over 100 US clinical trials presently test cultured autologous human cells, and several hundred test cultured stem cells with multiple origins.

What comes next

After the next round of printing tests this March, Techshot will share the bioprinter with companies and research institutions looking to print materials like cartilage, bone, and liver tissue. Theyre currently preparing the bioprinter for these additional uses, Boling says, which could advance health care as a whole.

To speed things up for space crews, Techshot is now building a cell factory that produces multiple cell types in orbit. This technology could cut down the number of cell deliveries between Earth and space.

The ISS has taken in plenty of commercial ventures in recent years, Michael Roberts says, and its getting crowded up there. Space-based experiments ramped up between 40 and 50 years ago, though until recently they mostly prioritized satellite communications and remote observation technology. Since then, satellites have shrunk from bus-sized to smaller than a shoebox.

Roberts has witnessed the scientific areas of interest broaden over the past decade to include medicine. Organizations like the National Institutes of Health are now looking to space to improve treatments, and everything from large pharmaceutical companies to small-scale startups want in.

Theyve got something stuck on every surface up there, he says.

As the ISS runs out of space and exterior attachment points, Roberts predicts that commercial ventures will build new facilities built for specific activities like manufacturing and plant growth. He sees it as a good opportunity for further innovation, since the ISS was originally designed for far more general purposes.

Space, as a whole, may start to look quite different from the first exploration age.

Baby boomers may remember glimpsing at a grainy, black-and-white moon landing five decades ago. Within the same lifetime, they could potentially observe the introduction of space-printed organs.

See more here:
Space might be the perfect place to grow human organs - Popular Science

Recommendation and review posted by Bethany Smith

Biomedical researchers from Texas Tech University Health Sciences Center El Paso and the University of Texas at El Paso are working on a joint project to send miniature 3D bioprinted hearts to space. The research project, which has received backing from the National Science Foundation (NSF), seeks to understand how a microgravity environment affects the function of the human heart.

Bioprinting in space is a growing venture. The microgravity environment found aboard the International Space Station (ISS) provides a unique setting for bioprinted tissues and cellular structures to culture and grow. Bioprinting specialists like CELLINK and 3D Bioprinting Solutions are showcasing the potential of bioprinting in space, both for the advancement of bioprinting technologies and to understand the impact of zero-gravity on the human body.

The three-year research project conducted by the Texas-based research team falls into the latter category. The team, led by Munmun Chattopadhyay, Ph.D., TTUHSC El Paso faculty scientist, and Binata Joddar, Ph.D., UTEP biomedical engineer, wants to understand how the human heart is impacted by microgravity by testing bioprinted cardiac organoids aboard the ISS.

The cardiac organoids consist of heart-tissue structures measuring less than 1 mm in thickness which are bioprinted using human stem cells. The organoids will be sent to the ISS, where they will exposed to microgravity environments. This will provide vital insights into a condition commonly experienced by astronauts.

The condition in question is cardiac atrophy and it is caused by a weakening of heart tissue. The condition can lead to other problems, like fainting, irregular heartbeats and even heart failure. Because astronauts often suffer from cardiac atrophy after spending long stints in space, the researchers want to better understand the link.

Cardiac atrophy and a related condition, cardiac fibrosis, is a very big problem in our community, said Dr. Chattopadhyay. People suffering from diseases such as diabetes, muscular dystrophy and cancer, and conditions such as sepsis and congestive heart failure, often experience cardiac dysfunction and tissue damage.

The project, which officially started in September, is currently focused on research design. In this stage of the research, the team is developing bioprinted cardiac organoids and exploring different material compositions using cardiac cells to create heart-like tissue. The second stage of the research will be focused on preparing to launch to organoid to space. The final stage will consist of analyzing data collected during the organoids time in space, once they have returned to Earth.

Dr. Chattopadhyay expressed excitement about the ongoing research project, saying: Knowledge gathered from this study could be used to develop technologies and therapeutic strategies to better combat tissue atrophy experienced by astronauts, as well as open the doorforimproved treatmentsforpeople who suffer from serious heart issues due to illness.

The researchers also hope to engage the community with their research by offering a workshop for K-12 students about their experiments aboard the ISS. The team will also host a seminar for medical students, interns and residents about conducting research in space and on Earth.

Read more here:
El Paso researchers sending bioprinted mini hearts to ISS - 3DPMN

Recommendation and review posted by Bethany Smith

Vincent Smith

Without long-term outcome data, the risks of individual procedures cant be quantified, nor can the advantages of waiting versus intervening early. Its also unclear whether patients whove gone public reflect most intersex peoples experiences. Clinicians dont know how much of the reported distress arises from outdated surgical techniques, nor do they know yet whether current procedures will prove any better.

Researchers are attempting to better gauge outcomes and satisfaction rates. A forthcoming European report will describe the opinions of more than one thousand intersex patients and their doctors regarding satisfaction with anatomical and functional results of genital surgery, according to a 2019 review article in theJournal of Pediatric Urology. Institutions are conducting other retrospective and prospective studies, such as a U.S. endeavor at multiple sites, including HMS.

Clinicians also are turning to transgender patients for insight. Teens and adults can provide immediate feedback on medical and surgical procedures and describe broad ranges of desired outcomes, which can then inform intersex care, says surgeon Diamond. The relationship seems fitting, since certain surgical interventions for transgender affirmation were informed by procedures developed for infants with DSDs.

But for many intersex advocates, the wait is too long for the results of such endeavors. Over the past decade, advocacy groups have led a global movement calling for a moratorium on genital and gonadal surgeries without patient consent. International health and human rights organizations, including the United Nations and the World Health Organization, have condemned the procedures, and several countries have restricted them. In February, the European Parliament urged member states to prohibit nonconsensual sex-normalizing surgeries as soon as possible. Some medical societies, consortia, and prominent figures such as a trio of former U.S. surgeons general have echoed the call. Several states, such as California, have considered bans.

This sea change has evoked an array of reactions, even among patients. People with CAH in particular say that an outright ban will do more harm than good by depriving families of the option to choose surgery. Appending an objection to a 2019 consensus paper by German academics that supported a ban, one CAH group said the majority of those with CAH who identify as female are satisfied with the results of their feminizing surgery and glad to have completed it in infancy.

The idea that the bodily autonomy of intersex children supersedes parents traditional roles as health care proxies remains a point of contention. National medical ethics councils in Finland, Germany, Sweden, and Switzerland say parents cannot authorize medically unnecessary surgery on genitals or gonads; the 2016 Global DSD Update says they can. Though the United States has not ruled on DSDs, its law and culture generally side with parents right to choose, say Garland and Diamond, and many clinicians continue to defer to them on intersex care.

When we discuss the pros and cons of surgery with the family and they say, We understand the different ways to go and this is what we think is best for our child, I accept that that is a responsible way to manage the child, says Diamond.

Some clinicians fear losing the ability to use their medical expertise to guide families and make decisions based on individual cases. It is not logical to impose mandatory restrictions on surgery in an area as complicated as this, reads a 2017 joint statement from seven U.S. urology and endocrinology societies.

Rosario served as chair of the medical advisory board for the Intersex Society of North America from 2002 to 2006 before he joined the UCLA DSD clinic. Initially against infant genital-normalizing surgery, he found that my opinion softened with actual clinical experience, he says.

Arguments roil about where gender-normalizing surgery falls along the spectrum of acts performed on infant genitalia. All fifty U.S. states condemn female genital mutilation, some advocates point out, so why should intersex surgery be considered differently? Others make comparisons to male circumcision, yet that practice also has been questioned. Professional societies are increasingly supporting interventions for transgender patients, so why deny the choice to those with DSDs, people ask?

While individual clinicians may support restricting infant genital-normalizing surgery, Garland wonders whether the threat of malpractice litigation explains why the U.S. medical profession tends to emphasize following the standard of care rather than trying nonintervention. He adds that in countries where the law requires scientific evidence and careful testing to establish the safety and efficacy of medical interventions, its been determined that these surgeries clearly dont meet that standard.

Pressure to change may come from peers, such as the Massachusetts Medical Society, which is debating a recommendation to delay surgeries on infants with DSDs that are of a non-emergent status until the individual has the capacity to participate in the decision. Doctors listen to other doctors, points out Smith, who serves on the LGBTQ committee that submitted the proposal.

Lawsuits also could influence U.S. medical practice. In a case that settled out of court in 2017, parents sued two South Carolina hospitals and a social services department for having performed feminizing surgery on a child they later adopted who grew up to identify as male.

Should DSD care shift, we will need a new way of thinking about how to determine when a child is able to consent, says Garland.

Those who worry about the lack of comparative data between early, delayed, and no intervention may take note as more nations and institutions restrict surgery on minors.

We may have our control group developing in Europe, says Diamond.

As more practitioners view forgoing surgery as an option, they turn to more flexible alternatives meant to support patients gender expression, such as hormone treatments. Surgeons also consider middle-ground procedures that preserve gender options as children grow.

In a 2018 case review in theJournal of Pediatric Urology, Diamond and colleagues described three infants with genetic mosaicism and complex urogenital and gonadal features whose parents all opted, among other procedures, to create vaginas but preserve the phalluses while they waited for their children to develop a gender. Two families were tentatively raising their children female; the other, gender neutral.

I wouldnt have thought that way at all ten years ago, says Diamond, who estimates he sees one hundred DSD patients a year in the Behavioral Health, Endocrinology, Urology (BE-U) program at Boston Childrens. My frame of mind would have been that the surgical options were more of a binary choice.

To those who believe that refraining from intervention does the least harm, Diamond says, You do your best, and you do it with a lot of humility because you know that no matter what you do, as much data as you have, you may be wrong.

Clinicians continue to learn how to avoid inadvertently making things worse for people with DSDs. Research studies and patient advocacy reports have documented the long-term psychological harm stemming from health care experiences such as repeated genital examinations and photography, depersonalization, and demeaning language.

Thats part of why psychologists and social workers have become essential members of DSD care teams over the past 20 years, although experts agree that psychosocial care still isnt available to enough families.

Surgeons and other specialists focus on their areas, particularly on the genitals, and they dont pay as much attention to the rest of the person, says Rosario. My job is to ask, how are you doing in school, and how are you doing with friends?

Although there is variation across conditions, initial research suggests that people with DSDs are more prone than the general population to mental health problems, including depression, anxiety, suicidal ideation, post-traumatic stress disorder, and trouble with intimacy. Such disparities may arise from treatment, culture, or the biology of the DSDs themselves.

Other studies assess the frequency, severity, and nature of parental distress when children receive DSD diagnoses. Researchers at HMS and elsewhere have found that unexpected anatomical variations, the possibility of stigma, and lack of clarity about the childs cancer risk, fertility, and future gender identity can cause significant anguish. Yet they also have found that caregivers of intersex children are no more depressed and, in fact, are less anxious than the general population.

Still more questions center on what should be done if the bulk of distress over DSDs arises from societal rather than medical issues.

In an era of gender-reveal parties and bathroom access controversies, having a perfectly happy baby with DSD can be like a crisis for families, says Smith. If there are no accompanying medical issues, then it becomes an entirely social-driven crisis.

Clinicians and parents often cite the desire to protect children from social harm when they opt for gender-normalizing procedures. Why, critics ask, in a culture built around binary sex, is the standard solution to alter bodies that are nonbinary rather than broaden societal conceptions of sex and gender?

Its really fraught when a concerned parent or physician thinks that a child who is intersex, and maybe doesnt present in a typical manner, is therefore going to have a harder time in the world, says Potter. That might be true, it might not be true, but in any event, trying to fix it so that they look like people with binary bodies may make a big mess of things.

Maybe we should be trying to help parents, and by extension the people surrounding the parentsthe extended family, the school system, all of these placesbecome more knowledgeable that theres a spectrum of sex presentation, she adds. Instead of conforming a child to something, transform the world in which they live. Then life may not be so hard.

Thats where law can also play a significant role, stopping discrimination and encouraging increased support for parents and children, says Garland.

While Garland, Potter, and others envision a more DSD-friendly future, they acknowledge that the systemic changes required will take time and effort. Meanwhile, others point out, clinicians, patients, and families must live in todays cultural contexts.

Discomfort with atypical sex characteristics is very much a societal problem, but we are caring for human beings who are brought up in our society to think in certain ways, says Diamond. As physicians and as a society, weve evolved a great deal, but were not at the point, I think, where we can routinely be comfortable with ambiguity. Some families can take that leap, but they are so uncommon.

As our culture progresses, that balance may shift. The sharing of peoples preferred pronouns, encompassing a spectrum of identities beyond he/his and she/hers, is becoming more common. People with transgender, gender nonconforming, nonbinary, and intersex identities are increasingly out and proud.

Ive been very surprised and pleased to see how much has changed in the LGBT arena in the past twenty years, says Garland. Its dramatic worldwide. Acceptance has increased of people with different sexualities and genders.

If trends continue, then in another generation or two, the agitation around DSDs may calm. Doctors may deliver healthy intersex babies and simply say: Congratulations.

Stephanie Dutchen is a science writer in the HMS Office of Communications and External Relations.

Image: Cici Arness-Wamuzky (top); John Soares (Smith and Diamond); John Davis (Rosario)

(Reprinted with permission from the Harvard Gazette.)

Visit link:
Don't Judge, Wait, and Know the Science: InterSex,The Body and The Self - India New England

Recommendation and review posted by Bethany Smith

The 2019 novel coronavirus (2019-nCoV) outbreak has sparked a speedy response, with scientists, physicians, and front-line healthcare professionals analyzing data in real-time in order to share findings and call out misinformation. Today, The Lancet published two new peer-reviewed studies: one which found that the new coronavirus is genetically distinct from human SARS and MERS, related viruses which caused their own outbreaks, and a second which reports clinical observations of 99 individuals with 2019-nCoV.

The first cases of the coronavirus outbreak were reported in late December 2019. In this new study, Nanshan Chen and colleagues analyzed available clinical, demographic, and laboratory data for 99 confirmed coronavirus cases at the Wuhan Jinyintan Hospital between Jan 1 to Jan 20, 2020, with clinical outcomes followed until 25th January.

Chen and colleagues reported that the average age of the 99 individuals with 2019-nCoV is around 55.5 years, where 51 have additional chronic conditions, including cardiovascular and cerebrovascular (blood flow to the brain) diseases. Clinical features of the 2019-nCoV include a fever, cough, shortness of breath, headaches, and a sore throat. 17 individuals went on to develop acute respiratory distress syndrome, resulting in death by multiple organ failure in 11 individuals. However, it is important to note here that most of the 2019-nCoV cases were treated with antivirals (75 individuals), antibiotics (70) and oxygen therapy (75), with promising prognoses, where 31 individuals were discharged as of 25th January.

Based on this sample, the study suggests that the 2019 coronavirus is more likely to affect older men already living with chronic conditions but as this study only includes 99 individuals with confirmed cases, it may not present a complete picture of the outbreak. As of right now, there are over 6,000 confirmed coronavirus cases reported, where a total of 126 individuals have recovered, and 133 have died.

In a second Lancet study, Roujian Lu and their fellow colleagues carried out DNA sequencing on samples, obtained from either a throat swab or bronchoalveolar lavage fluids, from eight individuals who had visited the Huanan seafood market in Wuhan, China, and one individual who stayed in a hotel near the market. Upon sequencing the coronaviruss genome, the researchers carried out phylogenetic analysis to narrow down the viruss likely evolutionary origin, and homology modelling to explore the virus receptor-binding properties.

Lu and their fellow colleagues found that the 2019-nCoV genome sequences obtained from the nine patients were very similar (>99.98% similarity). Upon comparing the genome to other coronaviruses (like SARS), the researchers found that the 2019-nCoV is more closely related (~87% similarity) to two bat-derived SARS-like coronaviruses, but does not have as high genetic similarity to known human-infecting coronaviruses, including the SARS-CoV (~79%) orMiddle Eastern Respiratory Syndrome (MERS) CoV (~50%).

The study also found that the 2019-nCoV has a similar receptor-binding structure like that of SARS-CoV, though there are small differences in certain areas. This suggests that like the SARS-CoV, the 2019-nCoV may use the same receptor (called ACE2) to enter cells, though confirmation is still needed.

Finally, phylogenetic analysis found that the 2019-nCoV belongs to the Betacoronavirus family the same category that bat-derived coronaviruses fall into suggesting that bats may indeed be the 2019-nCoV reservoir. However, the researchers note that most bat species are hibernating in late December, and that no bats were being sold at the Huanan seafood market, suggesting that while bats may be the initial host, there may have been a secondary animal species which transmitted the 2019-nCoV between bats and humans.

Its clear that we can expect new findings from the research community in the coming days as scientists attempt to narrow down the source of the 2019-nCoV.

Read this article:
Swamp sparrows can guess each other's ages from the sounds of their song - Massive Science

Recommendation and review posted by Bethany Smith

The 2019 novel coronavirus (2019-nCoV) outbreak has sparked a speedy response, with scientists, physicians, and front-line healthcare professionals analyzing data in real-time in order to share findings and call out misinformation. Today, The Lancet published two new peer-reviewed studies: one which found that the new coronavirus is genetically distinct from human SARS and MERS, related viruses which caused their own outbreaks, and a second which reports clinical observations of 99 individuals with 2019-nCoV.

The first cases of the coronavirus outbreak were reported in late December 2019. In this new study, Nanshan Chen and colleagues analyzed available clinical, demographic, and laboratory data for 99 confirmed coronavirus cases at the Wuhan Jinyintan Hospital between Jan 1 to Jan 20, 2020, with clinical outcomes followed until 25th January.

Chen and colleagues reported that the average age of the 99 individuals with 2019-nCoV is around 55.5 years, where 51 have additional chronic conditions, including cardiovascular and cerebrovascular (blood flow to the brain) diseases. Clinical features of the 2019-nCoV include a fever, cough, shortness of breath, headaches, and a sore throat. 17 individuals went on to develop acute respiratory distress syndrome, resulting in death by multiple organ failure in 11 individuals. However, it is important to note here that most of the 2019-nCoV cases were treated with antivirals (75 individuals), antibiotics (70) and oxygen therapy (75), with promising prognoses, where 31 individuals were discharged as of 25th January.

Based on this sample, the study suggests that the 2019 coronavirus is more likely to affect older men already living with chronic conditions but as this study only includes 99 individuals with confirmed cases, it may not present a complete picture of the outbreak. As of right now, there are over 6,000 confirmed coronavirus cases reported, where a total of 126 individuals have recovered, and 133 have died.

In a second Lancet study, Roujian Lu and their fellow colleagues carried out DNA sequencing on samples, obtained from either a throat swab or bronchoalveolar lavage fluids, from eight individuals who had visited the Huanan seafood market in Wuhan, China, and one individual who stayed in a hotel near the market. Upon sequencing the coronaviruss genome, the researchers carried out phylogenetic analysis to narrow down the viruss likely evolutionary origin, and homology modelling to explore the virus receptor-binding properties.

Lu and their fellow colleagues found that the 2019-nCoV genome sequences obtained from the nine patients were very similar (>99.98% similarity). Upon comparing the genome to other coronaviruses (like SARS), the researchers found that the 2019-nCoV is more closely related (~87% similarity) to two bat-derived SARS-like coronaviruses, but does not have as high genetic similarity to known human-infecting coronaviruses, including the SARS-CoV (~79%) orMiddle Eastern Respiratory Syndrome (MERS) CoV (~50%).

The study also found that the 2019-nCoV has a similar receptor-binding structure like that of SARS-CoV, though there are small differences in certain areas. This suggests that like the SARS-CoV, the 2019-nCoV may use the same receptor (called ACE2) to enter cells, though confirmation is still needed.

Finally, phylogenetic analysis found that the 2019-nCoV belongs to the Betacoronavirus family the same category that bat-derived coronaviruses fall into suggesting that bats may indeed be the 2019-nCoV reservoir. However, the researchers note that most bat species are hibernating in late December, and that no bats were being sold at the Huanan seafood market, suggesting that while bats may be the initial host, there may have been a secondary animal species which transmitted the 2019-nCoV between bats and humans.

Its clear that we can expect new findings from the research community in the coming days as scientists attempt to narrow down the source of the 2019-nCoV.

Read more here:
On Holocaust Remembrance Day, the stories of two Jewish scientists - Massive Science

Recommendation and review posted by Bethany Smith

Cryonics Technology Market research report 2019 gives detailed information of major players like manufacturers, suppliers, distributors, traders, customers, investors and etc. Cryonics Technology market Report presents a professional and deep analysis on the present state of Cryonics Technology Market that Includes major types, major applications, Data type include capacity, production, market share, price, revenue, cost, gross, gross margin, growth rate, consumption, import, export and etc. Industry chain, manufacturing process, cost structure, marketing channel are also analyzed in this report.The growth trajectory of the Global Cryonics Technology Market over the assessment period is shaped by several prevalent and emerging regional and global trends, a granular assessment of which is offered in the report. The study on analyzing the global Cryonics Technology Market dynamics takes a critical look at the business regulatory framework, technological advances in associated industries, and the strategic avenues.

Get Exclusively Free Sample Of This Report in PDF @ https://www.upmarketresearch.com/home/requested_sample/25072

Prominent Manufacturers in Cryonics Technology Market includes PraxairCellulisCryologicsCryothermKrioRusVWRThermo Fisher ScientificCustom Biogenic SystemsOregon CryonicsAlcor Life Extension FoundationOsiris CryonicsSigma-AldrichSouthern Cryoni

Market Segment by Product Types Slow freezingVitrificationUltra-rapid

Market Segment by Applications/End Users Animal husbandryFishery scienceMedical sciencePreservation of microbiology cultureConserving plant biodiversity

In order to identify growth opportunities in the market, the report has been segmented into regions that are growing faster than the overall market. These regions have been potholed against the areas that have been showing a slower growth rate than the market over the global. Each geographic segment of the Cryonics Technology market has been independently surveyed along with pricing, distribution and demand data for geographic market notably: North America (United States, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), Asia-Pacific (China, Japan, Korea, India and Southeast Asia), South America (Brazil, Argentina, Colombia etc.), Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa).

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Additionally, the complete value chain and downstream and upstream essentials are scrutinized in this report. Essential trends like globalization, growth progress boost fragmentation regulation & ecological concerns. Factors in relation to products like the products prototype, manufacturing method, and R&D development stage are well-explained in the global Cryonics Technology market research report with point-to-point structure and with flowcharts. It offers a comparative study between conventional and emerging technologies and the importance of technical developments in this market. At last, the market landscape and its growth prospects over the coming years have been added in the research.

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The Questions Answered by Cryonics Technology Market Report: What are the Key Manufacturers, raw material suppliers, equipment suppliers, end users, traders and distributors in Cryonics Technology Market? What are Growth factors influencing Cryonics Technology Market Growth? What are production processes, major issues, and solutions to mitigate the development risk? What is the Contribution from Regional Manufacturers? What are the Cryonics Technology Market opportunities and threats faced by the vendors in the global Cryonics Technology Industry? What are the Key Market segments, market potential, influential trends, and the challenges that the market is facing?And Many More

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Cryonics Technology Market Growth Rate, Demands, Status And Application Forecast To 2025 - Expedition 99

Recommendation and review posted by Bethany Smith

NEW YORK, Jan. 30, 2020 /PRNewswire/ --

High incidence of cancer & other target diseases is a major factor driving the growth of the gene therapy market

Read the full report: https://www.reportlinker.com/p05843076/?utm_source=PRN

The global gene therapy market is projected to reach USD 13.0 billion by 2024 from USD 3.8 billion in 2019, at a CAGR of 27.8% during the forecast period. The high incidence of cancer and other target diseases, availability of reimbursement, and the launch of new products are the major factors driving the growth of this market. In addition, the strong product pipeline of market players is expected to offer significant growth opportunities in the coming years. However, the high cost of treatment is expected to hamper market growth to a certain extent in the coming years.

Neurological diseases segment accounted for the largest share of the gene therapy market, by indication, in 2018Based on indication, the market is segmented into neurological diseases, cancer, hepatological diseases, Duchenne muscular dystrophy, and other indications.The neurological diseases segment accounted for the largest share of the market in 2018.

This can be attributed to the increasing number of gene therapy products being approved for the treatment of neurological diseases and the high market penetration of oligonucleotide-based gene therapies.

Viral vectors segment to register the highest growth in the gene therapy market during the forecast periodThe gene therapy market, by vector, has been segmented into viral and non-viral vectors.In 2018, the non-viral vectors segment accounted for the largest share of this market.

However, the viral vectors segment is estimated to grow at the highest CAGR during the forecast period, primarily due to the increasing demand for CAR T-based gene therapies and the rising incidence of cancer.

North America will continue to dominate the gene therapy market during the forecast periodGeographically, the market is segmented into North America, Europe, the Asia Pacific, and the Rest of the World.In 2018, North America accounted for the largest share of the gene therapy market, followed by Europe.

Factors such as the rising prevalence of chronic diseases, high healthcare expenditure, presence of advanced healthcare infrastructure, favorable reimbursement scenario, and the presence of major market players in the region are driving market growth in North America.

The primary interviews conducted for this report can be categorized as follows: By Company Type: Tier 1 - 32%, Tier 2 - 44%, and Tier 3 - 24% By Designation: C-level - 30%, D-level - 34%, and Others - 36% By Region: North America - 50%, Europe - 32%, Asia Pacific - 10%, and Rest of the World - 8%

List of companies profiled in the report Amgen, Inc. (US) Biogen (US) Novartis AG (Switzerland) Gilead Sciences, Inc. (US) Spark Therapeutics, Inc. (US) MolMed S.p.A. (Italy) Orchard Therapeutics plc. (UK) SiBiono GeneTech Co., Ltd. (China) Alnylam Pharmaceuticals, Inc. (US) Human Stem Cells Institute (Russia) AnGes, Inc. (Japan) Dynavax Technologies (US) Jazz Pharmaceuticals, Inc. (Ireland) Akcea Therapeutics (US) bluebird bio, Inc. (US) uniQure NV (Netherlands) AGTC (US) Mustang Bio (US) Cellectis (France) Poseida Therapeutics, Inc. (US) Sangamo Therapeutics (US)

Research Coverage:This report provides a detailed picture of the global gene therapy market.It aims at estimating the size and future growth potential of the market across different segments (by vector, indication, delivery method, and region).

The report also includes an in-depth competitive analysis of the key market players, along with their company profiles, recent developments, and key market strategies.

Key Benefits of Buying the Report:This report will help market leaders/new entrants by providing them with the closest approximations of the revenue numbers for the overall gene therapy market and its subsegments.It will also help stakeholders better understand the competitive landscape and gain more insights to position their business better and make suitable go-to-market strategies.

Also, this report will enable stakeholders to understand the pulse of the market and provide them with information on the key market drivers, challenges, and opportunities.

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The global gene therapy market is projected to reach USD 13.0 billion by 2024 from USD 3.8 billion in 2019, at a CAGR of 27.8% - PRNewswire

Recommendation and review posted by Bethany Smith

Taken together, cell and gene therapy could be called Philadelphias signature industry, and soon it may have its signature project.

Courtesy of The Discovery Labs

A rendering of the Center for Breakthrough Medicines, a cell and gene therapy manufacturing facility at The Discovery Labs in King of Prussia, Pa.

On Jan. 23, The Discovery Labs announced it will soon build a $1.1B, 680K SF contract development and manufacturing facility within its 1.6M SF campus in King of Prussia, as part of a joint venture with New York-based Deerfield Management. This Center for Breakthrough Medicines would take up the majority of the 1M SF former GlaxoSmithKline lab and manufacturing complex at 411 Swedeland Road that is part of Discovery Labs.

It will employ 2,000 people and become easily the largest contract development and manufacturing organizationin the world.

Though industry consensus isthat cell and gene therapy needs more manufacturing capability to keep growing and to speed up crucial development timelines, multiple sources told Bisnow that the Discovery Labs plan for CBM might be too ambitious in scale, price and timeline.

An unprecedented price tag for investors

Currently, the title for the largest cell and gene therapy manufacturing facility is held by Lonza, whichopened a 300K SF building in the Houston suburb of Pearland, Texas, in 2018. Lonza says itemploys over 200 full-time staffers at the facility. In the Philadelphia region, the only CDMO of any size is Wuxi AppTecs 287K SFcomplexat the Philadelphia Navy Yard. The new facility in King of Prussia has much larger ambitions.

[CBM] would service the world, Discovery Labs Executive Managing Director Audrey Greenberg said. Whats great about the King of Prussia location and the region, not just from the perspective of talent, is were very close to Europe on the East Coast. So well be able to supply viral vectors and plasmids to much of the U.S., Europe and possibly even Asia Pacific, South America and the Middle East.

Discovery Labs co-founder Brian ONeill, who also owns development firm MLP Ventures, is putting up initial funding for CBM, with some help from Deerfield and additional investors to join in later, Greenberg said. If ONeill needs to raise even half of the $1.1B price tag from outside investors, he may have a hard time finding anybody willing to put up the requisite cash for an operation that wont be developing its own intellectual property, sources said.

Both Greenberg and Jim Daly, a project director for biotech architecture and design firm CRB specializing in cell and gene therapy, believe that the pool of potential investors for the fledgling industry is deep and actively searching for deals.

I am amazed at the amount of capital that is behind so many companies, some of which didnt exist a year ago, Daly said. [But] thats a pretty rich number to try to get folks to step up for.

Courtesy of DIGSAU

A rendering of Iovance Biotherapeutics' lab, office and cell therapy manufacturing facility at the Philadelphia Navy Yard.

Raising funds is the hardest part about bringing a manufacturing facility online, whether a CDMO or a companys own build-to-suit,Gattuso Development Partners President John Gattuso said. Gattuso is developing a136K SF build-to-suit lab and manufacturing facility in the Navy Yard for Iovance Biotherapeutics. Until that is ready, Iovance contracts its production to Wuxi.

Amicus Therapeutics, which recently took space at uCity Square in University City, contracts with Wuxi, Daly said. Spark Therapeutics, the darling of Philadelphias gene therapy scene for getting multiple therapies FDA-approved, contracts out its manufacturing as well.

The University of Pennsylvania and Childrens Hospital of Philadelphia, the academic birthplace of Phillys gene and cell therapy startups, both have their own production needs met on campus, Daly said.

Before a company's therapy gets Food and Drug Administration approval, it relies on venture capital investment and grants to fund its research and development. Having a facility that can service many of them fills a gap in the market, Gattuso said, but a 680K SF facility may be considered too large todepend on non-revenue producers.

If you just think about how to finance a large facility filled with nothing but early, noncredit companies, its hard to imagine," Gattuso said.

Meeting the specific needs of a new, niche industry

Gene and cell therapy have material needs that require specialized manufacturing at every stage, from early research to commercial production for patients. Currently, no single contract facility has the manufacturing capability to meet those needs from bench to bedside, Greenberg said.

To research and develop new gene therapies, labs need plasmids (simple, circular strands of DNA) and viral vectors (benign containers for introducing new genes in a body) for every stage. Once a therapy is developed and can be used in clinical trials and eventually distributed to patients, then a company needs to effectively reproduce the finished product. All of these processes need to be done in highly sterilized clean room environments that are more complex and expensive than even most lab environments.

At full build-out, CBM is set to contain 36 suites for cell therapy processing, 10 for the production of plasmids, 20 for the production of viral vectors, and 20 for testing, process development and cell banking (or the storage of live cells needed for future batches). The suites will have a modular design so they can adapt to a changing client base and future advancements in production.

Courtesy of The Discovery Labs

A partial rendering of The Discovery Labs, MLP Ventures' planned biotech and life sciences coworking community in the Philadelphia suburb of King of Prussia.

Cell and gene therapy are both at such an early stageuntil some of those advancements come to pass, it is prohibitively expensive for a company to scale up to the point where it can fill and afford its own dedicated facility. Gene therapy can be produced in batches that can be only so large before the cell lines break down, and cell therapy is still dependent on autologous treatment, which means that each iteration only can serve one patient.

Those challenges and costs associated with project size are exactly what CBM is hoping to address, but multiple sources agreed that if a company has the resources, it will virtually always prefer to have its own facility to better control both the manufacturing process and its intellectual property.

Once you get to a certain scale, we see a strong desire on the part of certain firms to really control their own environment and be in their own building, Gattuso said. Its not just having the facility available, its about who controls that facility and how the IP is handled inside the facility.

Greenberg estimates there is five times more demand than supply for manufacturing in the gene and cell therapy industries nationwide, and that in three years that could explode to 5,000 times more demand than supply. Multiple sources in the industry agree that there is a substantial bottleneck in procuring the plasmids and viral vectors needed for even the early phases of research, but beyond that it is an unsettled issue.

The uncertain next steps

Though more than 200 "investigational new drug" permits have been issued in the past couple of years by the FDA, those only fast-track the clinical testing of methods for treating diseases and conditions so rare that few, if any, alternatives exist for sufferers.

Since two gene therapies and one cell therapy were fully approved by the FDA in 2017, only three more have gained approval since, Dark Horse Consulting founder Anthony Davies said at the Phacilitate conference in Miami on Jan. 22,according to BioProcess International. The first approved cell therapy, made by Novartis, has been halted by the FDA for manufacturing issues for over a year, Davies said.

Courtesy of The Discovery Labs

An interior rendering of gene and cell therapy hub The Discovery Labs in King of Prussia, Pa.

That frustration doesn't dampen CBM's prospects, as even therapies that will eventually fail need manufacturing capability. But if startups stuck in limbo between their earliest phases and full commercialization are in CBM's sweet spot, then a breakthrough that opens the floodgates could lead to those same companies instead opting for their own space, Daly said.

Part of the reason were seeing the bottleneck is in the supply side, and that would suggest that this is only a temporary phenomenon," Daly said. "As the Iovances of the world start to build out and manufacture their own plasmids and vectors, that demand will wane. That sides not super complicated or difficult; its more about what theyre prioritizing now.

Discovery Labs is in the design phase for CBM as of the end of January, but expects to begin gutting and renovating the former GSK facility within the next three months and bringing its first suites online by the end of this year. Greenberg estimates the first phase to open will amount to 50K to 75K SF of clean room space and about 100K SF for testing and development, which will require about 175 to 200 employees to staff.

Even working with a pre-existing facility that has many of the specialized needs for gene and cell therapy manufacturing, CBM's timeline of less than a year stretches credibility, multiple sources toldBisnow.Meeting the high standards for sterilization and IP protection is an exacting process that requires construction labor more specialized than for the average office building, though Gattuso said Philadelphia is maybe the best market in the country for finding that specific labor.

Staffing the building once operational will be an even taller task; sources estimate that training and getting clean room certification for technicians could take months on its own. The Discovery Labs hopes to address that by opening up its own training school, the Science University of Experiential Learning, within its grounds, but that won't be ready for that first wave of employees CBM will need by year's end.

Because of how much success Penn and CHOP have had in producing talent for gene and cell therapy production, the Philadelphia region makes sense as a place to try building something unprecedented. But despite the area's pride and optimism in its burgeoning industry's growth potential, CBM may be too much, too soon.

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The Biggest Gene And Cell Therapy Manufacturer Is Getting Built In King Of Prussia. Is It Too Ambitious? - Bisnow

Recommendation and review posted by Bethany Smith

A novel base editing technology invented at Rutgers, The State University of New Jersey with the potential to be used for the creation of new cell and gene therapies will be made available to researchers worldwide through an exclusive partnership with the Horizon Discovery Group.

The technology invented by Shengkan Victor Jin, associate professor of pharmacology, and co-inventor Juan C. Collantes, post-doctoral research fellow, at Rutgers Robert Wood Johnson Medical School can be potentially used for developing cell therapies for sickle cell anemia and beta thalassemia, HIV resistant cells for AIDS, off-the-shelf CAR-T cells for cancer, and MHC-compatible allogenic stem cells for transplantation. It could also be used as gene therapies for inherited genetic diseases such as antitrypsin deficiency and Duchenne muscular dystrophy.

The gene editing technology developed by our researchers has the potential to revolutionize how scientists think about their search for better options and outcomes in the treatment of disease worldwide, said S. David Kimball, PhD, Senior Vice President for Research and Economic Development at Rutgers University. Just as important is our ability, through this significant partnership with Horizon Discovery Group, to share our discoveries and inventions with the scientific community around the world who are equally committed to improving human health.

In January 2019, Rutgers University formed an exclusive partnership with Horizon to further the development of the proprietary base editing technology invented by Jin and Collantes. Since the initial partnership, Horizon, a global leader in the application of gene editing and gene modulation technologies, has been funding research in base editing at Jins laboratory. The company has now exercised its option to exclusively license the technology for commercialization of all therapeutic applications. This partnership places Rutgers among the front runners in the field of gene editing.

The technology could have a significant impact in enabling cell therapies to be progressed through clinical trials and towards commercialization. Horizon is pleased to offer an effective and precise base editing technology and, alongside Rutgers, aims to make base editing available to all appropriate cell and gene therapy companies as well as research departments. Partnering with leading organizations will help us to drive innovation and deliver the best therapy for the patient, stated Dr. Jonathan Frampton, Corporate Development Partner, Horizon Discovery.

Horizon has a number of internal programs designed to accelerate the clinical uptake of this technology and is now seeking partners to assess and shape the development of its Pin-point base editing platform. The company will offer partners access to a novel system that could be used to advance more effective multi-gene knockout cell therapy programs, with an improved safety profile, through clinical development. Partners will also gain access to the companys expertise in genome engineering of different cell types, access to early technical data, and influence over the direction of future development.

We intend to take full advantage of the unique modular and versatile features of the Pin-point platform and develop efficient gene inactivation agents for potential treatment of many devastating diseases where the leading causal contributing factors are well-defined. At the top of this disease list are Alzheimers disease, amyotrophic lateral sclerosis, and familial hypercholesterinemia, said Jin.

Base editing is a novel technology for engineering DNA in cells, with the potential to correct certain errors or mutations in the DNA or inactivate disease-causing genes. Compared with currently available gene editing methodologies such as conventional CRISPR/Cas9, which creates cuts in the gene that can lead to adverse or negative effects, this new technology allows for accurate gene editing while reducing unintended genomic changes that could lead to deleterious effects in patients.

# # #

About Rutgers, The State University of New Jersey

Rutgers, The State University of New Jersey, is a leading national research university and the state of New Jerseys preeminent, comprehensive public institution of higher education. Established in 1766, the university is the eighth oldest higher education institution in the United States. More than 70,000 students and 23,400 full- and part-time faculty and staff learn, work, and serve the public at Rutgers locations across New Jersey and around the world.www.rutgers.edu

As the premier public research university in the state, Rutgers is dedicated to teaching that meets the highest standards of excellence, to conducting cutting-edge research that breaks new ground and aids the states economy, businesses, and industries, and to providing services, solutions, and clinical care that help individuals and the local, national, and global communities where they live.research.rutgers.edu

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Rutgers partners with Horizon Discovery Group | - University Business

Recommendation and review posted by Bethany Smith

Pfizer aims to be the third big pharma with a significant presence in gene therapy. Its plans to initiate this year three Phase 3 trials targeting mutation-driven blood and muscular diseases would make it a large player in this cutting-edge area of medicine.

The difference between Pfizer and its Swiss rivals Novartis and Roche is that its treatments for muscular dystrophy and hemophilia do not look like they will be the first to market. With hopes that gene therapy could be a one-and-done treatment, arriving second could put Pfizer at a disadvantage if eager patients rush for curative therapies.

Having spun of its off-patent drugs business, the pharma is now trying to talk up the "new Pfizer." Its gene therapies are among seven pipeline projects that it cited Tuesday during its year-end earnings call as critical to its strategy of becoming a more innovation-focused company.

Company executives weren't, however, asked to answer how Pfizer views the emerging gene therapy competition. BioMarin Pharmaceutical looks set to get to the market earlier in hemophilia A than Pfizer, while Uniqure in hemophilia B and Sarepta Therapeutics in Duchenne muscular dystrophy appear ahead.

Pfizer's hemophilia A project, the Sangamo Therapeutics-originated SB-525, is up against BioMarin's valrox, which has been submitted to the Food and Drug Administration for an approval decision later this year.

In hemophilia B, fidanacogene elaparvovec, licensed from Roche subsidiary Spark Therapeutics, is in a neck-and-neck race with UniQure's etranacogene dezaparvovec in Phase 3 testing. Duchenne research, meanwhile, is led by Sarepta, which is launching a Phase 3 trial of its drug this year, putting Pfizer's at a disadvantage.

Other than announcing its intent to launch Phase 3 trials in hemophilia A and Duchenne, Pfizer didn't provide much more detail about these clinical programs. Mikael Dolsten, Pfizer's chief scientific officer, said more could be revealed about the DMD program at an upcoming research & development day.

Progress on that project had been delayed after one patient was hospitalized with kidney complications, but Dolsten said trial investigators had dosed additional patients. The Phase 2 will wrap up this spring, and the new data and longer follow-up will help guide a Phase 3 trial design, the company said.

Dolsten also described the hemophilia A project as having a 'best-in-class profile," even though BioMarin's valrox has impressed hematologists with its ability to increase expression of a key blood-clotting protein.

In addition, he said the company hopes it can bring one new gene therapy into its pipeline per year.

Building its drug development portfolio is one reason why the company has chosen not to buy back shares, said CEO Albert Bourla.

He pointed to the company's need in the past to buy back shares to support their valuation because of revenue declines, but now he said the company is in a different strategic position.

"The company is going to have a best-in-class revenue growth story," he said. "We can use the capital to invest in good Phase 2, Phase 3 assets to grow our pipeline."

Original post:
Pfizer lays out gene therapy aspirations - BioPharma Dive

Recommendation and review posted by Bethany Smith

Company signs option agreement with The Rockefeller University to access intellectual property covering compounds targeting key regeneration pathway

Decibel Therapeutics, a development-stage biotechnology company developing novel therapeutics for hearing loss and balance disorders, today announced a new strategic research focus on regenerative medicine approaches for the inner ear. The company is also announcing a collaboration and option agreement that gives Decibel exclusive access to novel compounds targeting proteins in a critical regenerative pathway.

Decibels research focus on regeneration will be powered by the companys research and translation platform. The company has built one of the most sophisticated single cell genomics and bioinformatics platforms in the industry to identify and validate targets. Decibel has also developed unique insights into regulatory pathways and inner ear delivery mechanisms that together enable precise control over gene expression in the inner ear and differentiate its AAV-based gene therapy programs.

"Our deep understanding of the biology of the inner ear and our advanced technological capabilities come together to create a powerful platform for regenerative medicine therapies for hearing and balance disorders," said Laurence Reid, Ph.D., acting CEO of Decibel. "We see an exciting opportunity to leverage this platform to address a broad range of hearing and balance disorders that severely compromise quality of life for hundreds of millions of people around the world."

The first program in Decibels regeneration portfolio aims to restore balance function using an AAV-based gene therapy (DB-201), which utilizes a cell-specific promoter to selectively deliver a regeneration-promoting gene to target cells. In collaboration with Regeneron Pharmaceuticals, Decibel will initially evaluate DB-201 as a treatment for bilateral vestibulopathy, a debilitating condition that significantly impairs balance, mobility, and stability of vision. Ultimately, this program may have applicability in a broad range of age-related balance disorders. There are currently no approved medicines to restore balance. Decibel expects to initiate IND-enabling experiments for this program in the first half of 2020.

Decibel is also pursuing novel targets for the regeneration of critical cells in both the vestibule and cochlea of the inner ear; these targets may be addressable by gene therapy or other therapeutic modalities. As a key component of that program, Decibel today announced an exclusive worldwide option agreement with The Rockefeller University, which has discovered a novel series of small-molecule LATS inhibitors. LATS kinases are a core component of the Hippo signaling pathway, which plays a key role in regulating both tissue regeneration and the proliferation of cells in the inner ear that are crucial to hearing and balance. The agreement gives Decibel an exclusive option to license this series of compounds across all therapeutic areas.

The agreement also establishes a research collaboration between Decibel and A. James Hudspeth, M.D., Ph.D., the F.M. Kirby Professor at The Rockefeller University and the director of the F.M. Kirby Center for Sensory Neuroscience. Dr. Hudspeth is a world-renowned neuroscientist, a member of the National Academy of Sciences and the American Academy of Arts and Sciences, and a Howard Hughes Medical Institute investigator. Dr. Hudspeth has been the recipient of numerous prestigious awards, including the 2018 Kavli Prize in Neuroscience.

"Rockefeller scientists are at the leading edge of discovery, and we are excited to see the work of Dr. Hudspeth move forward in partnership with Decibel," said Jeanne Farrell, Ph.D., associate vice president for technology advancement at The Rockefeller University. "The ambitious pursuit of harnessing the power of regenerative medicine to create a new option for patients with hearing loss could transform how we address this unmet medical need in the future."

In parallel with its new research focus on regenerative strategies, Decibel will continue to advance key priority preclinical and clinical programs. DB-020, the companys clinical-stage candidate designed to prevent hearing damage in people receiving cisplatin chemotherapy, is in an ongoing Phase 1b trial. Decibel will also continue to progress DB-OTO, a gene therapy for the treatment of genetic congenital deafness, which is being developed in partnership with Regeneron Pharmaceuticals. The DB-OTO program aims to restore hearing to people born with profound hearing loss due to a mutation in the otoferlin gene and is expected to progress to clinical trials in 2021.

Story continues

To support the new research focus, Decibel is restructuring its employee base and discontinuing some early-stage discovery programs.

About Decibel Therapeutics, Inc.Decibel Therapeutics, a development-stage biotechnology company, has established the worlds first comprehensive drug discovery, development, and translational research platform for hearing loss and balance disorders. Decibel is advancing a portfolio of discovery-stage programs aimed at restoring hearing and balance function to further our vision of a world in which the benefits and joys of hearing are available to all. Decibels lead therapeutic candidate, DB-020, is being investigated for the prevention of ototoxicity associated with cisplatin chemotherapy. For more information about Decibel Therapeutics, please visit decibeltx.com or follow @DecibelTx.

View source version on businesswire.com: https://www.businesswire.com/news/home/20200129005162/en/

Contacts

Matthew Corcoran, Ten Bridge Communicationsmcorcoran@tenbridgecommunications.com (617) 866-7350

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Decibel Therapeutics Announces Strategic Research Focus on Regenerative Medicine for the Inner Ear - Yahoo Finance

Recommendation and review posted by Bethany Smith

Innovative treatments have turned the tables somewhat in the battle against cancer but one stumbling block appears to be pricing. CAR-T cell therapy can cost more than $1.5 million including the $475,000 cost of the drug (in this case, Kymriah from Novartis).

We are seeing that price is becoming a barrier because if you can imagine, hundreds of thousands of people all suddenly becoming eligible for CAR-T, there isnt enough money in the healthcare system to pay for that, said Paul Wotton, CEO of Obsidian Therapeutics, based in Cambridge, Massachusetts.

In a recent interview in San Francisco, Wotton and another biopharma executive pointed to a solution to the price conundrum: an off-the-shelf approach.

To clarify, to date the approved CAR-T therapies involve cells from the patients own body that are re-engineered and placed back into the body in order to overpower and kill the cancerous growth. While revolutionary, this is not only a cumbrous process but also expensive.

Now, some companies are going the route of using donor cells to develop their own treatment regimens. These are known as allogeneic therapies.

Wugen Therapeutics is one such company that is taking an allogeneic approach to CAR-T therapy. Based in St. Louis Missouri with technology developed out of Washington University, the company targets T-cell malignancies in leukemia and lymphoma, said Ayman Kabakibi, vice president of research and development. In a recent interview, he explained that so far CAR-T therapies have focused on B-cell malignancies because it is less difficult to target B-cell malignancies with a T-cell.

The reason is that if you go back to the basics of CAR-T you are trying to put a receptor (CAR) on the normal T-cell to allow it to recognize another antigen or marker on the tumor cell and when that interaction happens, two things happen the T-cell will proliferate and make more daughter cells and thats why we call it a living drug, Kabakibi explained. And the other thing that happens is that it secretes toxins that kill the tumor cells. Well, you can do that more easily for B cells because the antigens are not shared between the two.

But at Wugen, the company is using re-engineeerd normal T-cells to target cancerous T-cells and certain adjustments need to be made so that the cells dont end up killing each other.

So what we do is that we take the T-cells and we remove the antigen that we are targeting on the cancer cell using CRISPR CAS technology, he said. We take T-cells from a normal person. We eliminate that marker and then we put another gene to make it stress the receptor that it will recognize.

Why not take T-cells from the patients own body?

One reason is that in many cases by the time patients land on CAR-T therapy, they may have already undergone several rounds of other treatments that may have compromised the health of a normal T-cell.

Because the patient has had so many chemotherapies the chances of getting a healthy T-cell are actually pretty low, he said. There are 20-30 percent of patients that go for CAR-T therapy and they get into the clinic and get their blood taken out and then it takes six weeks to generate autologous CAR-T therapy but they cant do it because theres not enough starting material, not enough healthy cells.

This approach of using donor T-cells gets around this problem but Kabakibi believes that it also solves the problem of the skyrocketing cost of CAR-T therapies.

You dont get the economies of scale [with CAR-T using autologous approach,] he said. Thats why you have to be able to generate hundreds of doses and not from the patient, but from a normal patient. You need an off-the-shelf, allogenic version. You take the cells from a normal person and generate a hundred vials that could be shipped overnight to a patient and then that person can be treated in a few days.

Wotton, the CEO of Obsidian Therapeutics, echoed Kabakibi.

You have to find a way to drive down that cost of goods. The way science is going to address that I think is to have off-the-shelf approaches where you can grow billions of cells from a single cell, engineer it to be able to get it to all of us to treat whatever it is we are suffering from, Wotton said. It is much better to build that scale of manufacturing rather than on a patient by patient basis.

Obsidian Therapeutics has developed what it calls destabilizing domain technology to be able to more precisely dose cell and gene therapies.

We are pioneering controllable cell and gene therapies using technology that came out of Stanford University, Wotton said. So in a nutshell, if you give somebody gene therapy today, to produce a particular protein in the body you actually have no idea how much of that protein is going to express. And the same in the case of a CAR-T cell to patients, you actually dont know how many of those cells are going to survive, how quickly they are going to proliferate so being able to dose cell and gene therapies in the same way you can dose a small molecule would be extremely useful.

Obsidianhas a partnership with Celgene through which Celgene can in-license global rights to cell therapy product candidates developed by Obsidian that incorporate destabilizing domain-regulated interleukin 12 or CD40L to fight cancer. Obdisial is using an allogeneic approach.

Our goal in life is to turn cell therapy into the first line of treatment instead of the last line of treatment, Wotton said. The only way you are going to be able to do that is to provide a cell therapeutic that is off-the-shelf, readily available and easy for patients to access, which is actually the approach that we are trying to take.

However, he added that there has to be a balance between cost and value when it comes to novel treatment.

Kabakibi of Wugen Therapeutics agreed.

If you are generating a 100 vials of CAR-T cells, is the price going to drop by 100 percent? Unlikely, he said. I think some therapies will never be as inexpensive as a pill.

Photo: champc, Getty Images

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Makers of novel cell/gene therapy say off-the-shelf approach only way to solve pricing woes - MedCity News

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In a second research report published this year so far, investigators found that the U.S. Food and Drug Administration (FDA) is approving drugs faster than ever. Unfortunately, it appears that the agency is also approving those drugs on less data and weaker evidence.

The first studypublishedin the journal SSRN was by researchers at Harvard University, the University of Texas at Dallas, and the Massachusetts Institute of Technology (MIT).It questioned if the FDA and other regulatory agencies worldwide dont rush certain approvals, particularly at the end of the year in a kind of desk-clearing activity.

The report notes, In the United States, the number of December drug approvals is roughly 80% larger than in any other month. Similar approval spikes occur at the end of each calendar month. Additionally, approvals spike before holidays, such as before Thanksgiving in the United States and the Chinese New Year in China (but not vice versa).

And more troubling is that there appears to be a correlation with more problems with these drugs. Lauren Cohen, a professor of finance and entrepreneurial management at Harvard Business School and one of the authors, told the Wall Street Journal, We see about twice as many adverse effects.

The second study appeared in the journal JAMA Network and was conducted by researchers with Harvard Medical School. The lead author, Jonathan Darrow, a lawyer with the medical schools Program on Regulation, Therapeutics and Law, told NPR, There has been a gradual erosion of the evidence thats required for FDA approval. He points out that patients and physicians should not expect that new drugs will be dramatically better than older ones.

The study notes that about half of recent drug approvals were built on a single pivotal clinical trial. Typically, two pivotal, Phase III trials were the norm. In addition, the study says that surrogate measures, which are utilized as stand-ins for presumed patient benefits, has grown. For example, in oncology drugs, what most patients would want are improvements in survival after receiving treatment. But some cancer trials use a surrogate measure of tumor shrinkage. Ideally, both would be taken into consideration.

Darrow and his research associates studied FDA approvals, changes in the law and regulations, and how the industry funds agency reviews from 1983 through 2018. They found that the average number of new drug approvals annually grew from 34 in the 1990s to 41 in the 2010s. In the 2000s, it dropped to 25 a year. But now they are increasing. For example, in 2019, the FDA approved 48 new molecular entities and new therapeutic biological products. That doesnt include vaccines, allergenic products, blood and blood products, plasma derivatives, cellular and gene therapy products, or the numerous new indications approved for existing therapies.

Darrow, with Jerry Avorn and Aaron S. Kesselheim, both with the Division of Pharmacoepidemiology & Pharmacoeconomics at Brigham & Womens Hospital, found that faster approvals were related to legislative and regulatory modifications that started in the 1980s. Although there are probably several reasons for those changes beginning in that period, much of it is likely related to the beginning of the HIV epidemic and demands from patient populations and advocates to fund more research and get therapiesany therapiesto market faster.

Just some of those regulations include: the 21st Century Cures Act (2016), which authorized funds for the Precision Medicine Initiative and Cancer Moonshot; the Biologics Price Competition and Innovation Act (BPCIA, 2010), creating an abbreviated pathway for follow-on biologic products; Breakthrough Therapy designation (2012), for drugs that showed substantial improvement over existing therapies; the Hatch-Waxman Act (1984), which created an Abbreviated New Drug Application pathway for drugs approved after 1962; and the Pediatric Research Equity Act (2003), which required results from pediatric assessments to be submitted as part of New Drug Applications (NDAs).

Congress also passed the Prescription Drug User Fee Act in 1993, and that first year, FDA collected $29 million in fees. In 2018, the agency brought in $908 million in PDUFA fees. Or, as the study notes, industry fees were responsible for about 80% of the money spent on FDA employee salaries for drug reviews.

There is some concern about the incentives that this created within the FDA, Darrow told NPR. And whether it has created a culture in the FDA where the primary client is no longer viewed as the patient, but as the industry.

Another factor that is related, is the concept of me-too drugs. These are basically drugs that are very structurally similar to approved drugs, with only minor differences. Thats not necessarily a bad thing, because they need to be at least as good as the drugs already on the market, and generally need to be betteralthough not necessarily by much. Which does mean a number of companies spend time on developing drugs that are only incrementally better than others on the market.

Joshua Sharfstein, former FDA Principal Deputy Commissioner, told NPR that there are more changes needed to ensure drugs are worthwhile for patients. Some of them are really great, and some of them [are] not so great. And a lot of them are very expensive.

Sharfstein is currently a professor at the Johns Hopkins Bloomberg School of Public Health. He wrote an editorial in JAMA that accompanied the newer study. In it, he suggests its time to reevaluate the FDAs expedited approval programs to determine which ones are working and which ones are increasing healthcare costs.

Weve kind of reached a point where it makes sense to pause and see whether we can do things better, Sharfstein said. And I think we can.

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The FDA Is Approving Drugs Faster, But That May Not Be A Good Thing - PharmaLive

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Stroke is the third leading cause of death and disability in the United States. More than 87 percent are ischemic strokes, caused by obstruction of one or more cerebral arteries. With limited progress in developing treatments, there is a critical need for neuroprotective agents to effectively treat stroke.A study from Florida Atlantic University's Schmidt College of Medicine holds promise for a new way to treat stroke using an already FDA-approved drug granulocyte colony-stimulating factor (GCSF). GCSF enhances blood cellular development and is currently used to treat neutropenia (low white blood cells) caused by chemotherapy and has successfully been used with very few side effects for patients who require bone marrow transplants to stimulate blood cell formation.

The study is the first to report on the neuroprotective effect of GCSF against autophagy and mitochondrial stress in vivo. The data support the hypothesis that GCSF is one of the few growth factors that can reduce infarction by decreasing endoplasmic reticulum (ER) and mitochondrial stress while improving behavioral performance.

Results showed that GCSF improved neurological deficits that occur in the first few days following cerebral ischemia and improved long-term behavioral outcomes while also stimulating a neural progenitor recovery response. Researchers tested behavioral performance on corner and locomotor tests, used as an indicator of brain injury.

Using a mouse model, researchers investigated the efficacy of GCSF beyond the typical four-hour thrombolytic therapy (tPA) clot-busting drug the gold standard to treat stroke for global ischemia. They examined the pro-survival mechanisms of GCSF against apoptosis resulting from autophagy, mitochondrial stress and ER stress.

"In recent years, many studies including ours have shown that as an endogenous growth factor and immune system modulator factor, GCSF is beneficial in models of neurological disorders such as stroke and traumatic brain injury," said Jang-Yen (John) Wu, Ph.D., corresponding author, distinguished professor of biomedical science in FAU's Schmidt College of Medicine, and a member of the FAU Brain Institute (I-BRAIN). "Although the anti-apoptotic activity of GCSF is reported in global cerebral ischemia, this mechanism has not been fully explored."

Researchers used a mechanism-based therapeutic approach for stroke first to examine the connection of mitochondrial, autophagy and ER stress inhibition in the protective action of GCSF and then to analyze relevant ER stress pathways in the bilateral common carotid artery occlusion (BCAO) model of stroke. They confirmed the neuroprotection of GCSF gene therapy in the BCAO mouse stroke model by a decrease of dynamin-related protein (DRP1), a marker of mitochondrial stress, in the frontal and middle brain of the GCSF treated group.

The initial dose of GCSF was administered 24 hours post-BCAO and then followed by a single application of the same dose for another three days for a total of four days of administration. Researchers examined behavior and used immunoblotting to analyze key proteins in ER stress, autophagy and mitochondrial stress-induced apoptosis. BCAO mice receiving GCSF protein showed significantly less asymmetric turning in the corner test than BCAO mice without GCSF. In the behavioral assays, GCSF elicited increased locomotor sensitization verified by greater activity in the locomotor activity test, demonstrating the neuroprotective properties of the drug.

"More than 15 million people worldwide suffer from stroke and our study provides new and important insights into GCSF induced protection as it relates to ER stress and mitochondrial stress activated apoptosis, " said Howard Prentice, Ph.D., corresponding author, a professor of biomedical sciences in FAU's Schmidt College of Medicine, and a member of FAU's I-BRAIN. "Future research will need to focus on uncovering the complete mechanisms by which GCSF retains the ER and mitochondrial homeostasis."

Wu and Prentice have been developing GCSF as a therapeutic method to replenish new brain cells because of its ability to preserve the central nervous system, suppress cell death and at the same time elicit neurogenesis as well as angiogenesis. GCSF works the same way for other neurological diseases such as Parkinson's disease due to its neuroprotective properties.ReferenceModi et al. (2020) Mode of action of granulocyte-colony stimulating factor (G-CSF) as a novel therapy for stroke in a mouse model. Journal of Biomedical Science. DOI: https://doi.org/10.1186/s12929-019-0597-7

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Repurposed Drug Proves Neuroprotective in Stroke Model - Technology Networks

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A new study by University of Massachusetts scientists has described the molecular process in which synaptic connections in the brain are compromised in multiple sclerosis.

The research, which was published in Immunity, investigates how gene therapy could be used to preserve neural circuits and prevent sight loss.

In experiments in mice, researchers inhibited a protein commonly found in the synapses of patients with multiple sclerosis.

Dr Sebastian Werneberg, from the University of Massachusetts Medical School, highlighted that the protein C3 prompts microglia to attack synapses.

A gene therapy approach was used to deliver a natural inhibitor of C3 to synapses in the visual system while leaving the rest of the brain untouched.

As a result of this inhibition, we saw improved visual function in mice, Dr Werneberg said.

Researchers will next explore how the C3 protein is being produced in multiple sclerosis and other neurodegenerative diseases.

Image credit: Pixabay/PublicDomainPictures

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Protecting against vision loss in multiple sclerosis OT - AOP

Recommendation and review posted by Bethany Smith

SAN DIEGO, Jan. 28, 2020 (GLOBE NEWSWIRE) -- Otonomy, Inc.(OTIC), a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, today announced preclinical results from the companys gene therapy collaboration with Applied Genetic Technologies Corporation (AGTC) focused initially on treating GJB2 deficiency for congenital hearing loss, and preclinical results demonstrating the therapeutic potential of a class of compounds being evaluated for otoprotection against cisplatin-induced hearing loss (CIHL). These results were presented during the ongoing Association for Research in Otolaryngology (ARO) 43rd Annual MidWinter Meeting being held in San Jose, California.

Together with our strategic partner, AGTC, we are encouraged by these initial preclinical results that demonstrate our ability to express a gene of interest in the target cells relevant to the treatment of congenital hearing loss due to GJB2 deficiency," said David A. Weber, Ph.D., president and chief executive officer of Otonomy. Also, the preclinical results presented for our OTO-510 program highlight the therapeutic potential of a novel class of cisplatin-binding molecules for protection against CIHL and the higher potency of these agents versus other molecules currently in clinical development.

Preclinical Results for GJB2 Gene Therapy Collaboration

In October 2019, Otonomy and AGTC announced a strategic collaboration to co-develop and co-commercialize an AAV-based gene therapy to restore hearing in patients with sensorineural hearing loss caused by a mutation in the gap junction protein beta 2 gene (GJB2) -- the most common cause of congenital hearing loss. The joint presentation by Otonomy and AGTC at ARO provided initial demonstration that a gene of interest can be expressed in support cells of the cochlea, which are the relevant target cells for treating GJB2 deficiency, using novel and proprietary AAV capsids. Furthermore, these studies identified several capsids with favorable tropism and gene expression level in support cells compared to previously reported capsids used in the field. Importantly, none of the novel AAV capsids evaluated for further development exhibited signs of cellular toxicity.

Preclinical Results for OTO-510 Otoprotection Program

Cisplatin is a potent chemotherapeutic agent that is widely used to treat a variety of cancers in adults and children. Unfortunately, the administration of cisplatin is commonly associated with severe adverse effects including CIHL that is progressive, bilateral and irreversible. At ARO, Otonomy presented preclinical results demonstrating varying degrees of otoprotection against CIHL for several different classes of therapeutic agents. In particular, a novel proprietary class of agents that potently bind to cisplatin demonstrated greater otoprotection than anti-oxidant and anti-apoptotic molecules, and increased potency relative to other cisplatin-binding molecules currently in clinical development. These results highlight the therapeutic potential of Otonomys novel otoprotectant agents as the basis for the OTO-510 program for CIHL.

About Otonomy

Otonomy is a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology. The company pioneered the application of drug delivery technology to the ear in order to develop products that achieve sustained drug exposure from a single local administration. This approach is covered by a broad patent estate and is being utilized to develop a pipeline of products addressing important unmet medical needs including Mnires disease, hearing loss, and tinnitus. For additional information please visit http://www.otonomy.com.

Cautionary Note Regarding Forward Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements generally relate to future events or the future financial or operating performance of Otonomy. Forward-looking statements in this press release include, but are not limited to expectations regarding the potential benefits, development activity and advancement of preclinical programs; the potential benefits of and activity under the collaboration agreement between AGTC and Otonomy, including but not limited to development activity; and statements by Otonomys president and CEO. Otonomys expectations regarding these matters may not materialize, and actual results in future periods are subject to risks and uncertainties. Actual results may differ materially from those indicated by these forward-looking statements as a result of these risks and uncertainties, including but not limited to: Otonomys limited operating history and its expectation that it will incur significant losses for the foreseeable future; Otonomys ability to accurately forecast financial results; Otonomys ability to obtain additional financing; Otonomys dependence on the regulatory success and advancement of its product candidates; the uncertainties inherent in the clinical drug development process, including, without limitation, Otonomys ability to adequately demonstrate the safety and efficacy of its product candidates, the nonclinical and clinical results for its product candidates, which may not support further development, and challenges related to patient enrollment in clinical trials; Otonomys ability to obtain regulatory approval for its product candidates; the risks of the occurrence of any event, change or other circumstance that could give rise to the termination of the collaboration agreement between AGTC and Otonomy; the risks of the occurrence of any event, change or other circumstance that could impact Otonomys ability to repay or comply with the terms of the loan provided by Oxford Finance LLC; side effects or adverse events associated with Otonomys product candidates; Otonomys ability to successfully commercialize its product candidates, if approved; competition in the biopharmaceutical industry; Otonomys dependence on third parties to conduct nonclinical studies and clinical trials; Otonomys dependence on third parties for the manufacture of its product candidates; Otonomys dependence on a small number of suppliers for raw materials; Otonomys ability to protect its intellectual property related to its product candidates in the United States and throughout the world; expectations regarding potential therapy benefits, market size, opportunity and growth; Otonomys ability to manage operating expenses; implementation of Otonomys business model and strategic plans for its business, products and technology; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled "Risk Factors" in Otonomys Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (the "SEC") on November 5, 2019, and Otonomys future reports to be filed with the SEC. The forward-looking statements in this press release are based on information available to Otonomy as of the date hereof. Otonomy disclaims any obligation to update any forward-looking statements, except as required by law.

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Otonomy Presents Preclinical Results for GJB2 Gene Therapy Collaboration and Cisplatin Otoprotection Program - Yahoo Finance

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BALTIMORE, Jan. 29, 2020 /PRNewswire/ --Green Park Collaborative (GPC) is partnering with the National Hemophilia Foundation (NHF) to develop a publicly available, patient-reported outcome measure that measures impact on mental health outlook associated with receiving gene therapy for hemophilia A and B.

This project follows work completed in coreHEM, where a multi-stakeholder group achieved consensus on a core outcome set for gene therapy in hemophilia. In coreHEM, a mental health outcome was included, and the proposed definition included elements of psychological and mental health status, and emotional functioning due to the transformational change of a potentially one-time treatment. Because gene therapy will serve as a durable treatment for hemophilia, changes in mental and emotional health are expected. No current measures exist to effectively evaluate this outcome.

"Developing a new instrument to measure the transformative potential of gene therapy on an individual's overall mental health, whether positive or negative, will further enable our understanding of the entire spectrum of functional and social impact of living with hemophilia," stated Mark Skinner, co-investigator of the coreHEM study.

"Mental health in the bleeding disorders community has always been vital to the individuals and families we serve," said Dawn Rotellini, Interim CEO at NHF. "As we move towards enhanced and innovative treatments in hemophilia it will become imperative to measure how the community responds and how NHF can support them. We are excited to continue our partnership with the Green Park Collaborative to better serve the bleeding disorders community."

About Green Park Collaborative (GPC)GPC is a major initiative of CMTP, an independent 501(c)(3) non-profit organization dedicated to improving the quality, relevance, and efficiency of clinical research. GPC is a multi-stakeholder forum for developing condition- and technology-specific study design recommendations to guide the creation of evidence needed to inform both clinical and payment decisions.

About the National Hemophilia Foundation (NHF)The National Hemophilia Foundation (NHF) is a 501(c)(3) non-profit organization dedicated to finding better treatments and cures for inheritable bleeding disorders and to preventing the complications of these disorders through education, advocacy and research. NHF's programs and initiatives are made possible through the generosity of individuals, corporations and foundations, as well as through a cooperative agreement with the Centers for Disease Control and Prevention (CDC).

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SOURCE Center for Medical Technology Policy

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Green Park Collaborative and the National Hemophilia Foundation launch project to develop a PROM to measure mental health outlook - P&T Community

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Passage Bio Jim Wilsons self-described legacy company has wooed a seasoned biotech executive to steer the clinical entry of its first three gene therapy programs.

Bruce Goldsmith jumps to the helm of Passage after a brief CEO stint at Civetta, a cancer-focused startup he helped launch while a venture partner at Deerfield. He takes over from OrbiMed partner and interim chief Stephen Squinto, who will now lead the R&D team.

He joins as the biotech preps IND filings for three lead programs in rare, monogenic diseases of the central nervous system in 2020 the lysosomal storage disorders GM1 gangliasidosis and Krabbe disease, as well as frontotemporal dementia.

Bruce is ideally suited to lead Passage Bio as chief executive officer given his strong neuroscience background coupled with his robust healthcare and biotechnology industry experience, board chairman Tachi Yamada said in a statement.

Passage launched last February with $115 million from a marquee group of Series A investors including Frazier (where Yamada is a partner), OrbiMed, Versant Ventures, New Leaf Venture Partners, Vivo Capital and Lilly Asia Ventures. With an office just a 10-minute walk away from Wilsons lab at the University of Pennsylvania, the company was designed to apply the gene therapy pioneers 35-year experience into cross-correctional therapies for CNS.

According to what he calls the Jim Wilson 90/10 rule, Squinto previously told Endpoints News, AAV vectors can cover and transduce 90% of motor neuron cells but only 10% to 15% of other brain cells making it difficult to go after indications where broad transduction is needed. But it can still prove useful in disorders that result from mutations in enzymes that can be taken up by neighboring cells once secreted normally.

A close pact with Penns Gene Therapy Program and Orphan Disease Center gave Passage rights to five programs right out of the gate, with options to license seven more.

Its a very aggressive clinical development strategy across a multitude of programs, Squinto said as he closed a $110 million Series B in September. Were not gonna rely on any one program to drive the value of Passage, were gonna rely on what is a very very full pipeline of opportunities.

Goldsmith will now lead a team of about 25 to build on preclinical and IND-enabling data from Wilsons lab a company growing exercise he honed as COO of Lycera. There, he was also credited for a number of business development initiatives.

The transition into the clinic would also mean moving production from early facilities at Penn to Paragons GMP sites, and eventually to a customized suite slated for completion in the third quarter of this year.

Squinto, a rare disease expert who devoted much of his career to Alexion, will continue to help oversee all of that as a board director.

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Eyeing a trio of trial initiations, Jim Wilson's gene therapy startup woos Bruce Goldsmith from Deerfield as CEO - Endpoints News

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The Cell and Gene Therapy Catapult and the University of Hertfordshire have launched a new course specifically addressing the foreseeable skills gap in the production of cell and gene therapies as they progress towards manufacturing at scale.

Developed in collaboration between the two organisations, this three-day course will provide theoretical and practical training on the aseptic manufacturing of cell and gene therapies in line with European regulatory guidance for good manufacturing practice.

The cell and gene therapy industry in the UK currently supports over 3,000 jobs, a six-fold increase since 2012, and employment in the sector is set to more than double by 2024 as more therapies progress towards commercialisation.

Manufacturing and bioprocessing roles in particular have tripled in the past two years alone, with scientists operating in the 26 cell and gene therapy manufacturing facilities throughout the UK.

Cell and gene therapies are transformative and potentially curative medicines and it is vital that manufacturing processes are safe and efficient whilst preserving the effectiveness of these living medicines.

The new training programme is designed for staff working in cell and gene therapy manufacturing. Delegates will benefit from experience in state-of-the-art facilities and will be awarded a University of Hertfordshire accredited certificate upon successful completion of the training, contingent on assessments of their knowledge, understanding and practical skills.

Dr Salman Rahman, commercial lead in the School of Life & Medical Sciences at the University of Hertfordshire, said: Students on the course will be taught theoretical knowledge including principles and operational aspects necessary for aseptic manufacturing of cell and gene therapy products in line with European regulatory guidance, complimented by practical learning experiences in state-of-the-art clean room facilities.

Keith Thompson, CEO of the Cell and Gene Therapy Catapult added: There is an accelerating demand for skills in the UK cell and gene therapy industry as employment, manufacturing space and the number of ongoing clinical trials increases year on year.

We are delighted to collaborate with the University of Hertfordshire in developing this new initiative which will support professionals andBy providing training on GMP quality standards which are vital to the manufacture of cell and gene therapies, we are furthering our mission to fuel the continuing growth of the industry.

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Boost for cell and gene therapy manufacture - Business Weekly

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Horizon Discovery is to provide access to a novel base editing technology licensed from Rutgers, The State University of New Jersey, for exclusive use in therapeutic, diagnostic and services applications.

The technology is incorporated into Horizons next-generation gene editing platform. It will enable the development of novel therapeutics that rely on engineering patients cells either directly in the body (gene therapy), or externally before transplanting back into the patient (cell therapy).

The platform will also expand the companys research tools and service provisions.

Horizon formed an exclusive partnership with Rutgers in January 2019 to further develop the novel base editing technology invented by Dr Shengkan Jin, associate professor of pharmacology, and co-inventor Dr Juan C. Collantes, post-doctoral research fellow at Rutgers Robert Wood Johnson Medical School, and has since been funding research in base editing at the university while undertaking its own evaluation and proof-of-concept studies.

Horizon has a number of internal programs designed to accelerate the clinical uptake of this technology and is now seeking 35 partners to assess and shape the development of its Pin-point base editing platform.

Horizon will offer partners access to a novel system that could be used to progress more effective multi-gene knockout cell therapy programs through clinical development with an improved safety profile.

Partners will also gain access to the companys expertise in genome engineering of different cell types, access to early technical data, and influence over the direction of future development.

Base editing is a novel technology for engineering DNA in cells, which the potential to correct certain errors or mutations in the DNA, or inactivate disease-causing genes.

Compared to currently available gene editing methodologies such as conventional CRISPR/Cas9, which creates cuts in the gene that can lead to adverse or negative effects, this new technology allows for accurate gene editing while reducing unintended genomic changes that could lead to deleterious effects in patients.

Dr Jonathan Frampton, corporate development partner at Horizon Discovery, said: The technology could have a significant impact in enabling cell therapies to be progressed through clinical trials and towards commercialisation.

Horizon is pleased to offer an effective and precise base editing technology and, alongside Rutgers, aims to make base editing available to all appropriate cell and gene therapy companies as well as research departments. Partnering with leading organisations will help us to drive innovation and deliver the best therapy for the patient.

Dr Shengkan Victor Jin of Rutgers University added: The cytidine deaminase version of the technology alone could potentially be used for developing cell therapies such as gene modified cells for sickle cell anaemia and beta thalassemia, HIV resistant cells for AIDS, over-the-shelf CAR-T cells for cancer, and MHC-compatible allogenic stem cells for transplantation.

Other applications could include use as gene therapies for inherited genetic diseases including antitrypsin deficiency and Duchenne muscular dystrophy. In addition, we intend to take full advantage of the unique modularity and versatility features of Pin-point platform and develop efficient gene inactivation agents for potential treatment of many devastating diseases where the leading causal contributing factors are well defined.

At the top of this disease list are Alzheimers disease, amyotrophic lateral sclerosis, and familial hypercholesterinemia.

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Horizon and New Jersey university power up gene therapy technology - Business Weekly

Recommendation and review posted by Bethany Smith

The global Gene Therapy market report includes a scrupulous analysis of the Gene Therapy market in the forecasted period. It also assesses the Gene Therapy market in terms of topography, technology, and consumers. The report also covers the volume of the market during the projected period. The uniqueness of the global Gene Therapy market research report is the representation of the Gene Therapy market at both the global and regional level.The key playersBluebird Bio, Sangamo, Spark Therapeutics, Dimension Therapeutics, Avalanche Bio, Celladon, Vical Inc., Advantagene play an important role in the global Gene Therapy market.

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The global Gene Therapy report offers the weaknesses as well as plus points of the established market players. It analyses numerous features of the global Gene Therapy market such as demand, drivers, challenges, and options. The report appraises the influence of these aspects on each market region during the estimated time. It presents the value chain analysis together with vendor list and highlights the present confronts between consumer and supplier.

There are 15 Segment to show the Global Gene Therapy market

Segment 1,Definition, Specifications and Classification of Gene Therapy, Applications of Gene Therapy, Market Segment by Regions;Segment 2,Aggregating Cost Structure, Rough Material and Suppliers, Social occasion System, Industry Chain Structure;Segment 3,Specialized Information and Assembling Plants Examination of Gene Therapy, Limit and Business Production Date, Assembling Plants Circulation, Research and development Status and Innovation Source, Raw Materials Sources Investigation;Segment 4,Generally Market Examination, Cutoff Examination (Affiliation Piece), Arrangements Examination (Affiliation Bit), bargains Regard Examination (Affiliation Portion);Segment 5 and 6,Regional Market Investigation that incorporates United States, China, Europe, Japan, Korea and Taiwan, Gene Therapy segment Market Examination (by Sort);Segment 7 and 8,The Gene Therapy Segment Market Analysis (by Application) Major Manufacturers Analysis of Gene Therapy;Segment 9,Market Trend Analysis, Regional Market Trend, Market Trend by Product Type Ex vivo, In Vivo Market Trend by Application Cancer, Monogenic, Infectious disease, Cardiovascular disease, Other;Segment 10,Common Propelling Sort Examination, By and large Exchange Type Examination, Stock framework Examination;Segment 11,The Clients Examination of worldwide Gene Therapy;Segment 12,Gene Therapy Research Findings and Conclusion, Appendix, system and information source;Segment 13, 14 and 15,Gene Therapy deals channel, wholesalers, merchants, traders, Exploration Discoveries and End, appendix and data source.

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Additionally, the global Gene Therapy market is segmented on the basis of the region as well. It employs some practical tools to assess the expansion of the global Gene Therapy market in the upcoming time. The global Gene Therapy market report also offers a synopsis of the market on a global level that helps users in the decision-making processes, which in turn helps to boost their businesses. This synopsis incorporates the index growth as well as the competitive framework of the global Gene Therapy market over the projected period.

The highlight of the global Gene Therapy market research report is the in-depth market segmentation {Ex vivo, In Vivo}; {Cancer, Monogenic, Infectious disease, Cardiovascular disease, Other}. The report uses primary and secondary sources for analysis. The global Gene Therapy market is assessed in terms of value (USD Million). The global Gene Therapy market research report offers the performance of all the related key players, vendors, and suppliers. Additionally, this report represents the majority of the data with the help of graphics and tables together with the projected statistics.

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Motivations to Purchase Gene Therapy Market Report Covered

1. The report studies how Gene Therapy market will perform in the future.2. Considering different perspectives on the Gene Therapy market with the assistance of Porters five powers examination.3. Separating the article type that is obviously to control the market and districts that are likely going to watch the quickest improvement between the assessed time period.4. Distinguish the new advancements, Gene Therapy market offers, and techniques utilized by the key market players.5. The focused scene including the market offer of huge players nearby the key frameworks recognized for advancement in the past five years.6. Complete organization profiles covering the item contributions, key monetary data, current improvements, SWOT examination and techniques utilized by the significant Gene Therapy market players.

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Global Gene Therapy Market 2019 by Manufacturers, Regions, Type and Application, Forecast to 2025 - Dagoretti News

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According to a recent report General market trends, the Gene Therapy economy is likely to witness a CAGR growth of XX% within the forecast period (2019-2029) and reach at a value of US$ at the ending of 2029. The micro and macroeconomic elements that are forecasted to influence the trajectory of this Gene Therapy market are examined in the market analysis that was presented.

The report throws light on the raw material Providers, vendors, manufacturers, and market participants at the value string of their market that is Gene Therapy . Whats more, the political and economic scenarios of various regions and its effect on the Gene Therapy market are discussed in the report.

Critical Details included from the record:

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Competitive Outlook

Light onto the throws Business prospects of players operating in the Gene Therapy industry. Preferred marketing channels the product pricing plans and product portfolio of prominent players, and market presence of each provider is included in the accounts. The dominant players covered in the report contain Business 2 Company, Company 3, and Business 4.

Regional Assessment

The market study that is introduced sheds light on the market Scenario in regional markets. Additionally, the governmental and regulatory policies on the prospects of this Gene Therapy market in every regions effect is examined in the report.

segmented as follows:

Global Gene Therapy Market, by Product

Global Gene Therapy Market, by Application

Global Gene Therapy Market, by Region

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Reasons Gene Therapy Market Report Stands Out

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Gene Therapy Market 1Q 2018: Current Trends, SWOT Analysis, Strategies, Industry Challenges, Business Overview and Forecast Research Study - Dagoretti...

Recommendation and review posted by Bethany Smith

What happens when a successful biotech venture capitalist is unexpectedly diagnosed with a chronic, life-disrupting vertigo disorder? Innovation in neurotology.

That venture capitalist was Jay Lichter, Ph.D., and after learning there was no FDA-approved drug treatment for his condition, Mnires disease, he decided to create a company to bring drug development to neurotology. Otonomy was founded in 2008 and is dedicated to finding new drug treatments for the hugely underserved community living with balance and hearing disorders. Helping patients like Jay has been the driving force behind Otonomy, a company heading into a transformative 2020 with three clinical trial readouts: Phase 3 in Mnires disease, Phase 2 in tinnitus, and Phase 1/2 in hearing loss. These catalysts, together with others in the field, highlight the emerging opportunity in neurotology.Otonomy is leading the way in neurotologyNeurotology, or the treatment of inner ear neurological disorders, is a large and untapped market for drug developers: one in eight individuals in the U.S. have moderate-to-severe hearing loss, tinnitus or vertigo disorders such as Mnires disease.1 With no FDA-approved drug treatments available for these conditions, the burden on patientsincluding social anxiety, lower quality of life, reduced work productivity, and higher rates of depressioncan be significant.2, 3, 4

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The FDA unveils a new regulatory framework to speed along gene therapies, rewarding the leading players - Endpoints News

Recommendation and review posted by Bethany Smith