Recent research on longevity is making the idea of an elixir of life sound increasingly plausible. But a startup thats started selling a $1 million anti-aging treatment is most likely jumping the gun.

Libella Gene Therapeutics says it will administer volunteers with a gene therapy that it claims can reverse aging by up to 20 years, according to OneZero. Despite the fact that this is the first human trial of the treatment, the company is charging volunteers $1m to take part. In an effort to side-step the FDA, the trial will take place in Colombia.

The therapy will attempt to repair peoples telomeres, the caps on the end of our chromosomes that shorten as people get older. Its long been thought that they play a role in aging, and efforts to extend telomeres in mice have shown that it can delay the signs of getting older and increase healthy lifespan, though its yet to be tested in humans.

Libellas therapy will use viruses to deliver a gene called TERT, which codes for an enzyme called telomerase that re-builds teleomeres, to the patients cells.

Experts told MIT Tech Review that the trial is unethical, poorly designed, and presents serious risks to participants, including the danger of activating dormant cancerous cells. But its also still unclear whether the trial will go ahead, because the company has made previous announcements before without following through.

Whether or not it does, though, medical treatments to head off the slow march towards death are likely to become increasingly common. A growing body of research suggests that aging is an entirely preventable condition and that there may be a variety of ways to treat it, from lifestyle changes to dramatic genetic interventions.

In 2017, scientists showed that using drugs to reprogram epigenetic markerschemical attachments responsible for regulating the genomein mice extended their lifespan by 30 percent. And in 2018, another team showed that using a combination of drugs to kill senescent cellszombie cells that leak harmful chemicals, damaging nearby tissuecould boost the longevity of mice by 36 percent.

Famous geneticist George Church has even launched a startup called Rejuvenate Bio that will use proprietary genetic treatments to prolong the lives of dogs, though he has admitted the ultimate goal is to extend its technology to humans. Last month Churchs group at Harvard also showed that using gene therapies to tackle three age-related diseases at once was effective in mice.

The first anti-aging treatments for people are already starting to appear as well. CEO of longevity company BioViva Elizabeth Parrish injected herself with a gene therapy similar to Libellas back in 2015, and the company has claimed it was successful in lengthening her telomeres, though results were never published.

Earlier this year a study on humans found that a cocktail of drugs could reset the epigenetic clock, epigenetic markers used to measure a persons biological age. The participants also showed signs of a rejuvenated immune system.

And more controversially, the FDA recently had to put out a public service announcement telling people to stop injecting blood plasma from younger people. The idea is built upon recent research that showed a rejuvenating effect in mice, but most experts say its far too early to apply it to humans.

Whether the FDA will be able to keep on top of this burgeoning and highly lucrative market remains to be seen, but given the potential side effects of many of these treatments, it should be a priority.

We also need to have a more in-depth conversation about what these longevity therapies mean for society. Assuming this new trial is effective, what does it mean if only those with $1m to spare get to extend their lives? If treating aging becomes trivial, how is that going to change the nature of our communities? These are questions that may become increasingly relevant in the coming decades.

Image Credit: Shutterstock.com

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A New Anti-Aging Therapy Is Starting Its First Human Trialand It Costs $1 Million - Singularity Hub

Recommendation and review posted by Bethany Smith

BRISBANE, Calif.--(BUSINESS WIRE)--Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, is hosting an R&D Day today beginning at 8am Eastern Time. During the event, Sangamo executives and scientists plan to provide updates across the Companys clinical and preclinical pipeline, as well as an overview of manufacturing capabilities to support clinical and commercial supply. A live webcast link will be available on the Events and Presentations page of the Sangamo website

The talent, R&D capabilities, manufacturing expertise, and operations infrastructure we have brought to Sangamo have enabled us to advance a genomic medicine pipeline that spans multiple therapeutic areas and now also extends into late-stage development, said Sandy Macrae, CEO of Sangamo. As we make progress in clinical development, we gain insights into the use of our technology and are applying those insights as we advance new programs, such as the gene therapy for PKU and the genome regulation candidates for CNS diseases we are announcing today.

Macrae continued: We will continue to pursue a dual approach of retaining certain programs for our proprietary pipeline while also establishing pharmaceutical partnerships to gain access to therapeutic area expertise and financial, operational, and commercial resources. Strategic collaborations will be a particularly important consideration as we advance programs for diseases affecting large patient populations.

R&D Day updates on clinical and preclinical pipeline programs:

Gene therapy product candidates for hemophilia A, Fabry disease, and PKU

SB-525 is a gene therapy product candidate for hemophilia A being developed by Sangamo and Pfizer under a global development and commercialization collaboration agreement. The transfer of the SB-525 IND to Pfizer is substantially completed. Pfizer is advancing SB-525 into a Phase 3 registrational study in 2020 and has recently begun enrolling patients into a Phase 3 lead-in study.

At R&D Day, Sangamo executives are presenting data from the SB-525 program which were recently announced at the American Society of Hematology (ASH) annual meeting.

The cassette engineering, AAV engineering and manufacturing expertise which Sangamo used in the development of SB-525 are also being applied to the ST-920 Fabry disease program, which is being evaluated in a Phase 1/2 clinical trial, as well as to the newly announced ST-101 gene therapy program for PKU, which is being evaluated in preclinical studies with a planned IND submission in 2021.

Engineered ex vivo cell therapy candidates for beta thalassemia, kidney transplantation, and preclinical research in multiple sclerosis (MS)

Sangamo is providing an overview of the Companys diversified cell therapy pipeline this morning. Cell therapy incorporates Sangamos experience and core strengths, including cell culture and engineering, gene editing, and AAV manufacturing. At R&D Day, Sangamo scientists today are reviewing the early data presented this month at ASH from the ST-400 beta thalassemia ex vivo gene-edited cell therapy program, which is being developed in partnership with Sanofi.

Sangamo is also providing updates on the companys CAR-TREG clinical and preclinical programs. CAR-TREGS are regulatory T cells equipped with a chimeric antigen receptor. Sangamo is the pioneer in CAR-TREGS, which may have the potential to treat inflammatory and autoimmune diseases. TX200 is being evaluated in the STEADFAST study, the first ever clinical trial evaluating a CAR-TREG cell therapy. Tx200 is being developed for the prevention of immune-mediated organ rejection in patients who have received a kidney transplant, a significant unmet medical need. Results from this trial will provide data on safety and proof of mechanism, building a critical understanding of CAR-TREGS in patients, and may provide a gateway to autoimmune indications such as Crohns disease and multiple sclerosis (MS). Sangamo is also presenting preclinical murine data demonstrating that CAR-TREGS accumulate and proliferate in the CNS and reduce a marker of MS.

In vivo genome editing optimization

Clinical data presented earlier this year provided evidence that Sangamo had successfully edited the genome of patients with mucopolysaccharidosis type II (MPS II) but also suggested that the zinc finger nuclease in vivo gene editing reagents were under-dosed using first-generation technology. Sangamo has identified potential improvements that may enhance the potency of in vivo genome editing, including increasing total AAV vector dose, co-packaging both ZFNs in one AAV vector, and engineering second-generation AAVs, ZFNs, and donor transgenes.

Genome regulation pipeline candidates targeting neurodegenerative diseases including Alzheimers and Parkinsons

Sangamo scientists today are presenting data demonstrating that the companys engineered zinc finger protein transcription factors (ZFP-TFs) specifically and powerfully repress key genes involved in brain diseases including Alzheimers, Parkinsons, Huntingtons, ALS, and Prion diseases. Sangamo is advancing its first two genome regulation programs toward clinical development:

Sangamo scientists are also presenting data demonstrating progress in the development of new AAV serotypes for use in CNS diseases.

Manufacturing capabilities and strategy

Sangamo is nearing completion of its buildout of a GMP manufacturing facility at the new Company headquarters in Brisbane, CA. This facility is expected to become operational in 2020 and to provide clinical and commercial scale manufacturing capacity for cell and gene therapy programs. The Company has also initiated the buildout of a cell therapy manufacturing facility in Valbonne, France. Sangamos manufacturing strategy includes in-house capabilities as well as the use of contract manufacturing organizations, including a long-established relationship with Thermo Fisher Scientific for clinical and large-scale commercial AAV manufacturing capacity.

R&D Day webcast

A live webcast of the R&D Day, including audio and slides, will be available on the Events and Presentations page of the Sangamo website today at 8am Eastern Time. A replay of the event will be archived on the website.

About Sangamo Therapeutics

Sangamo Therapeutics is committed to translating ground-breaking science into genomic medicines with the potential to transform patients lives using gene therapy, ex vivo gene-edited cell therapy, and in vivo genome editing and gene regulation. For more information about Sangamo, visit http://www.sangamo.com.

Sangamo Forward Looking Statements

This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of United States securities law. These forward-looking statements include, but are not limited to, the therapeutic potential of Sangamos product candidates; the design of clinical trials and expected timing for milestones, such as enrollment and presentation of data, the expected timing of release of additional data, plans to initiate additional studies for product candidates and timing and design of these studies; the expected benefits of Sangamos collaborations; the anticipated capabilities of Sangamos technologies; the research and development of novel gene-based therapies and the application of Sangamos ZFP technology platform to specific human diseases; successful manufacturing of Sangamos product candidates; the potential of Sangamos genome editing technology to safely treat genetic diseases; the potential for ZFNs to be effectively designed to treat diseases through genome editing; the potential for cell therapies to effectively treat diseases; and other statements that are not historical fact. These statements are based upon Sangamos current expectations and speak only as of the date hereof. Sangamos actual results may differ materially and adversely from those expressed in any forward-looking statements. Factors that could cause actual results to differ include, but are not limited to, risks and uncertainties related to dependence on the success of clinical trials; the uncertain regulatory approval process; the costly research and development process, including the uncertain timing of clinical trials; whether interim, preliminary or initial data from ongoing clinical trials will be representative of the final results from such clinical trials; whether the final results from ongoing clinical trials will validate and support the safety and efficacy of product candidates; the risk that clinical trial data are subject to differing interpretations by regulatory authorities; Sangamos limited experience in conducting later stage clinical trials and the potential inability of Sangamo and its partners to advance product candidates into registrational studies; Sangamos reliance on itself, partners and other third-parties to meet clinical and manufacturing obligations; Sangamos ability to maintain strategic partnerships; competing drugs and product candidates that may be superior to Sangamos product candidates; and the potential for technological developments by Sangamo's competitors that will obviate Sangamo's gene therapy technology. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamos operations. This presentation concerns investigational drugs that are under preclinical and/or clinical investigation and which have not yet been approved for marketing by any regulatory agency. They are currently limited to investigational use, and no representations are made as to their safety or effectiveness for the purposes for which they are being investigated. Any discussions of safety or efficacy are only in reference to the specific results presented here and may not be indicative of an ultimate finding of safety or efficacy by regulatory agencies. These risks and uncertainties are described more fully in Sangamo's Annual Report on Form 10-K for the year ended December 31, 2018 as filed with the Securities and Exchange Commission on March 1, 2019 and Sangamo's Quarterly Report on Form 10-Q for the quarter ended September 30, 2019 that it filed on or about November 6, 2019. Except as required by law, we assume no obligation, and we disclaim any intent, to update these statements to reflect actual results.

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Sangamo Highlights Advancements in Genomic Medicine Pipeline and Expanded R&D and Manufacturing Capabilities at R&D Day - Business Wire

Recommendation and review posted by Bethany Smith

CAMBRIDGE, Mass., Dec. 17, 2019 (GLOBE NEWSWIRE) -- LogicBio Therapeutics Inc. (Nasdaq:LOGC), a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients,today announced ithas entered into an exclusive license with Oregon Health & Science University (OHSU) to intellectual property rights owned by OHSU while also extending a sponsored research agreement (SRA) to explore methods for enhancing selective advantage of edited hepatocytes using pharmacological agents with the laboratory ofMarkusGrompe, M.D.,professor at OHSU. The initial phase of the research program provided proof-of-principle of enhanced selective advantage for cells edited by GeneRide in pilot murine experiments. This extension phase will focus on translating the enhancement strategy to non-human primates, a critical step before clinical translation to future GeneRide candidates and other technologies. GeneRide is LogicBios proprietarypromoterless, nuclease-free genome editing technology,whichis designed to provide a stable therapeutic effect by harnessing homologous recombination to precisely integrate corrective genes into a patients genome and leveraging endogenous promoters to drive gene expression.

LogicBiois currently working primarily in disorders where patients can benefit substantially even when only a modest percentage of their cells are modified and begin expressing the corrective transgene introduced by GeneRide. The Company has found, however, that in some genetic contexts, integrating the transgene gives hepatocytes a naturally-occurring selective advantage over cells that have not been modified. Over time, the percentage of modified cells expressing that transgene rises, potentially leading to more robust patient benefits. This wasobserved inan experimentin which a murine GeneRide construct was introduced into mice with and without a functioning copy of theMutgene (deficient in the pediatric disease methylmalonic acidemia) in the liver. The initial GeneRide integration frequency was less than 1% in both sets of mice. Over time, this percentage remained stable in heterozygous mice that naturally expressMutin the liver (Mut+/- in liver). However, the share of cells expressing Mut increased to approximately 25% over more than a year in the mice genetically deficient in liverMut(Mut-/- in liver). This selective advantage could be attributed to improvements in mitochondrial function as a result ofMutexpression and restoration of the deficient essential metabolic pathway.These data were presented at the 2019 American Society of Gene & Cell Therapy Annual Meeting and can be found on the LogicBio website at the following link: https://investor.logicbio.com/events-and-presentations/presentations.

The goal of the expanded SRA with OHSUis to refine the pharmacological approachto providing a selective advantage to gene modified cells even when the transgene does not naturally confer a selection advantage at the cellular level. One such method involves adding an element to a GeneRide construct that gives cells incorporating that element a selective advantage when patients are treated with an external approved pharmacological agent.This research could enable expansion of the GeneRide platform to address genetic disorders in which clinical benefit emerges only after a higher percentage of cells are modified and begin expressing the corrective transgene.

We are excited to explore novel methods for enriching the number of cells expressing the therapeutic gene, said Dr. Grompe. Such methods could improve the likelihood that patients derive long-term therapeutic benefit from a single treatment. They could also expand the range of serious genetic disorders we can address with GeneRide.

Dr. Grompes lab studies monogenic disorders, particularly metabolic liver diseases affecting children. He has focused extensively on the use of in vivo selection to enhance cell and gene therapies. Dr. Grompe received the E. Mead Johnson Award for research excellence from the Society for Pediatric Research in 2002. He retains an active clinical practice, focused on metabolic disease.

About LogicBio TherapeuticsLogicBio Therapeutics is a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients with significant unmet medical needs using GeneRide, its proprietary technology platform. GeneRide enables the site-specific integration of a therapeutic transgene in a nuclease-free andpromoterlessapproach by relying on the native process of homologous recombination to drive potential lifelong expression. Headquartered in Cambridge, Mass., LogicBio is committed to developing medicines that will transform the lives of pediatric patients and their families.

For more information, please visitwww.logicbio.com.

Forward Looking Statements This press release contains forward-looking statements within the meaning of the federal securities laws. These are not statements of historical facts and are based on managements beliefs and assumptions and on information currently available. They are subject to risks and uncertainties that could cause the actual results and the implementation of the Companys plans to vary materially, including the risks associated with the initiation, cost, timing, progress and results of the Companys current and future research and development activities and preclinical studies and potential future clinical trials. These risks are discussed in the Companys filings with the U.S. Securities and Exchange Commission (SEC), including, without limitation, the Companys Annual Report on Form 10-K filed on April 1, 2019 with the SEC, and the Companys subsequent Quarterly Reports on Form 10-Q and other filings with the SEC. Except as required by law, the Company assumes no obligation to update these forward-looking statements publicly, even if new information becomes available in the future.

Contacts:

Brian LuqueAssociate Director, Investor Relationsbluque@logicbio.com951-206-1200

Stephanie SimonTen Bridge Communicationsstephanie@tenbridgecommunications.com617-581-9333

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LogicBio Therapeutics Extends Sponsored Research Agreement with Oregon Health & Science University to Explore Translation of...

Recommendation and review posted by Bethany Smith

One of the best things about being on the University of WisconsinMadison campus is the opportunity to learn from its passionate scientists and students. Covering the research stories of the institution keeps us busy, but it also provides the opportunity to have some fun (but dont tell the bosses!). While we believe that every story we do is important, here are some of the ones that taught us the most in 2019.

Chris Barncard, Eric Hamilton and Kelly April Tyrrell, Research Communications

Earths last magnetic field reversal took far longer than once thought

Some scientists think were living through the beginning of the next reversal of Earths magnetic field, which would affect our heavily electronic world in bizarre ways. Thankfully, new research from geoscientist Brad Singer found evidence that the last time Earths field reversed, it took about 22,000 years to complete. Thats several times longer than researchers previously thought and means that humanity would likely have generations to adapt to the next lengthy period of magnetic instability.

Genes behind lager yeasts cold- and sugar-loving success revealed

Lager beer is cold, crisp, dry and worth hundreds of billions of dollars. Genetics professor Chris Todd Hittinger and his lab recently discovered that lager yeast tolerates the cold because it inherited the power-generating portion of the cell from its cold-loving ancestor. And they figured out how this hybrid could have evolved the ability to digest all the sugars in wort to ferment a dry, crisp beer. Together, these two traits helped lager yeast and beer take over the world.

Memorial Union steam whistle sets rhythm of summer evenings and saves lives

To Terrace-goers, the steam whistle on Helen C. White Hall might just seem like a quaint way to herald another sunset. But to university and public boaters alike, its true mission is clear: keeping everyone on Lake Mendota safe enough to head out another day. UWPD Lake Rescue and Safety and the Hoofer Sailing Club collaborate to train sailors, monitor lake conditions and blow the whistle when approaching storms threaten boaters, ultimately saving lives. Thanks, whistle.

Reddit competes to visualize Madisons prized Lake Mendota ice data

For 166 years, university and state scientists have carefully tracked Lake Mendotas annual freeze and thaw. That trove of data was just what Reddit was looking for. The DataIsBeautiful subreddit competed to find the best way to visualize this long, icy record. As each graphic, animation and chart revealed, Lake Mendota has lost about a months worth of ice since record-keeping started as the result of a warming climate. That adds up to big changes for Madisons ice fishers and cross-country skiers and to the watery world beneath their feet.

Ancient poop helps show climate change contributed to fall of Cahokia

Nearly 1,000 years ago, the ancient city of Cahokia, near present-day St. Louis, was the most sophisticated prehistoric settlement north of Mexico. But then, the Mississippi River began to flood, and Cahokias population rapidly declined. Researchers at UWMadison and California State University, Long Beach found evidence using remnants of human poop and layers of sediment deposited in a nearby lake to suggest climate change contributed to Cahokias fall. Cultures can be very resilient in the face of climate change but resilience doesnt necessarily mean there is no change, says UWMadison anthropology professor Sissel Schroeder.

Fear of more dangerous second Zika, dengue infections unfounded in monkeys

Zika virus, already connected to heartbreaking consequences for newborns in recent epidemics in the Americas, was cause for additional concern among public health officials because it is so closely related to dengue virus which is dangerous enough in an initial infection, but can be even more life-threatening during a second infection. This year, UWMadisons Zika virus researchers allayed some fears that the viruses would make each other more dangerous by showing that, in monkeys, an earlier infection with one of the viral cousins does not make a later infection with the other more virulent.

Study confirms horseshoe crabs are really relatives of spiders, scorpions

When is a crab not actually crab? When its a horseshoe crab. These hard-shelled, blue-blooded creatures belong, it turns out, to the spiders and the scorpions. UWMadison evolutionary biologists Prashant Sharma and Jess Ballesteros subjected the genomes of horseshoes crabs, which have existed on Earth for 450 million years, to intense computational scrutiny and found they should live on the arachnid family tree. They are part of a lineage that makes them among the most successful animals on the planet.

Tiny capsules packed with gene-editing tools offer alternative to viral delivery of gene therapy

Gene therapy using chemical tools to edit a patients genetic code for inherited diseases, some cancers, and even stubborn viral infections is most often delivered using engineered viruses, but those viruses are hard to steer to specific cells within the body and can cause trouble by exciting the immune system. UWMadison biomedical engineers have created an alternative to viral delivery, loading tiny synthetic capsules with gene-editing tools and coating the shell with molecules that help them zero in on their therapeutic targets.

With fire, warming and drought, Yellowstone forests could be grassland by mid-century

Yellowstone National Park may be at a tipping point. Its forests are adapted for periodic fires, but large wildfires that once blazed the landscape every 100-to-300 years are now sweeping through much more frequently as the climate warms and drought conditions increase. UWMadison ecologist Monica Turner has studied the forests of Yellowstone for three decades and her work suggests forests may not be able to regenerate quickly enough. By the middle of this century, some of them may become grassland.

Lessons of conventional imaging let scientists see around corners

By playing the angles, UWMadison researchers are developing cameras that can see around corners. Led by Andreas Velten, a professor of biostatistics and medical informatics, the scientists can bounce thousands of pulses of laser light off a wall or other surface into an unseen space and collect the scattered photons that ricochet back to their sensors. Using math to reconstruct the path of the returning light, they can piece together a picture of the hidden space as seen from the perspective of their reflecting surface.

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2019's most memorable research stories - University of Wisconsin-Madison

Recommendation and review posted by Bethany Smith

IOWA CITY A University of Iowa clinician and neuroscientist has received an $18 million grant to continue a decade-long study on how a potential treatment for Huntingtons disease may affect childrens brain development.

Dr. Peg Nopoulos, chairwoman of the University of Iowa department of psychiatry, was awarded a five-year grant by the National Institution of Neurological Disorders and Strokes, part of the U.S. Institutes of Health.

These are really high-in-the-sky questions, she said. Were just boots on the ground, trying to understand how this affects children at risk for Huntingtons disease.

Huntingtons disease is a fatal genetic disorder that causes the progressive breakdown of nerve cells, diminishing thinking skills and emotions and disrupting fine motor function. If a parent has Huntingtons disease, there is a 50 percent chance her or his child will develop the same disease.

There is no cure for Huntingtons, but an emerging gene therapy in clinical trials has presented promising findings for slowing its progression, Nopoulos said. The gene therapy has been given only to individuals who have Huntingtons disease in an attempt to slow the progressive breakdown.

Nopoulos said the next step is to give the therapy to individuals before the genetic disorder takes effect, to test whether it could prevent it altogether.

However, she said this possibility presents a key question as the gene that causes the disease also is important for the growth and development of an individuals brain

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Our study is really examining how this gene affects brain development, Nopoulos said. And in that context, were examining how we would deliver a gene therapy and how that would impact brain development.

We certainly dont want to prevent the disease only to cause problems for brain development.

The gene that causes Huntingtons, called HTT, is key for an area of the brain called the striatum, which helps control voluntary movement, among other functions. Through their study, Nopoulos and other researchers on the project hope to understand what consequences could result from the therapy on development.

If we were to give the gene modifying therapy too early, it could prevent maximum brain function, she said.

Nopoulos has been studying this question for 10 years at the UI and will continue that work under the newly funded project, called Children to Adult Neurodevelopment in Gene-Expanding Huntingtons disease.

The $18 million grant from the national institute is a renewal of the same study from the UI, allowing Nopoulos and other researchers to expand the scope and size of the original study by including five times as many participants from five sites across the United States.

In addition to the UI, those sites will include the Childrens Hospital of Philadelphia, Columbia University in New York, the University of California, Davis and the University of Texas in Houston.

All subjects in the study, who are between the ages of 6 and 30, have a parent with Huntingtons, which means they are 50 percent at risk for developing the genetic disorder sometime in their lifetime.

Nopoulos said the test subjects dont know if they carry the genetic marker for the disease.

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Weve been doing this for 10 years, and Im always amazed at how strong these kids are, Nopoulos said. The study gives them meaning and an opportunity to help gain knowledge about the disease their parent is going through.

A lot of these children really take on the altruism of wanting to give back to the community, even when they know theyre at risk themselves, she said.

Comments: (319) 368-8536; michaela.ramm@thegazette.com

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University of Iowa researcher gets $18 million to study Huntington's disease - The Gazette

Recommendation and review posted by Bethany Smith

In an effort to understand aging, researchers from Stanford University and colleagues around the world analyzed almost 3,000 proteins in the blood of more than 4,000 people ages 18 to 94. They published their research in the journal Nature Medicine.

When we went into this, we assumed you aged gradually, so we would see these changes taking place relatively steadily as individuals get older, said Tony Wyss-Coray, professor of neurology at Stanford University, senior author of the study.

That isnt what they found, however. They found that the proteins appear to change in three distinct waves, with the first occurring during our 30s, peaking around age 34.

Then we found a second wave around 60, and then we found a third one, the most prominent one, really around 80 years of age, Wyss-Coray said.

The researchers are dubbing this a proteomic clock, that somewhat accurately predicts an individuals age.

One of the reasons for the research was the insight that by placing the blood of younger mice into older mice, the older mice appeared to grow youngerthe reverse happened, toothe blood from older mice aged younger mice. So, whats in the blood of different-aged animals (including people) that is making these changes for better or worse?

Another fact Wyss-Coray and his team knew was that in the blood proteins of people with Alzheimers disease, the biggest differences werent between Alzheimers patients and healthy people of the same age, but between people of different ages.

The researchers winnowed the thousands of proteins they were looking at down to 373 whose levels appeared to be predictive of age.

Wyss-Coray notes that most of the proteins found in the blood derive from other tissues. So we can start to ask where these proteins come from and if they change with age.

For example, proteins that trace back to the liver would suggest the liver is aging. Or if they derive from the kidney, the kidney is aging. Wyss-Coray hopes to eventually be able to analyze blood protein patterns to create a personalized aging clock where I can tell you, based on the composition of your blood, your kidney seems to be aging faster than it should.

And perhaps, if enough information is found, similar to the mouse aging experiments, it might be possible to isolate proteins that contribute to the effectpreferably the one that keeps you young, not makes you old.

Some companies went into the business of selling blood transfusion plasma from young people as an anti-aging potion. The U.S. Food and Drug Administration (FDA), in February 2019, issued warnings to consumers and health care providers against this, pointing out that the FDA did not test these in order to confirm therapeutic benefit or safety.

We strongly discourage consumers from pursuing this therapy outside of clinical trials under appropriate institutional review board and regulatory oversight, the agency said in a statement from then-Commissioner Scott Gottlieb.

Gottlieb added, Our concerns regarding treatments using plasma from young donors are heightened by the fact that there is no compelling clinical evidence on its efficacy, nor is there information on appropriate dosing for treatment of the conditions for which these products are being advertised. Plasma is not FDA-recognized or approved to treat conditions such as normal aging or memory loss, or other diseases like Alzheimers or Parkinsons disease.

The agency warned of possible risks of infectious, allergic, respiratory and cardiovascular problems from the treatments.

Meanwhile, groups like Wyss-Corays and others are attempting to identify which proteins are most effective and whether the proteins themselves are active in staying young or just markers of something else.

Irina Conboy, a researcher at the University of California, Berkeley, published research in the journal Aging in August 2019, that analyzed a protein known as TGF-beta, which is associated with aging. Her experiments in laboratory mice hint that by blocking TGF-beta, aging effects can be slowed.

Wyss-Coray has founded a biotech company, Alkahest, that is researching blood plasma infusions in Alzheimers disease, as well as other applications of blood plasma infusions. At the recent 12th Clinical Trials on Alzheimers Disease conference the company gave an oral presentation of Phase II trial of GRF6019 in mild-to-moderate Alzheimers disease. The Phase IIa trial was completed and patients were randomized and treated with 100mL or 250mL of GRF6019 for five days during Week 1 and again for five consecutive days until Week 13 with a treatment-free interval of 11 weeks after each dose.

GRF6019 and another of its products, GRF6021, are proprietary plasma fractions developed and provided by Grifols. In animal models the plasma fractions improved neurogenesis as well as age-related learning and memory deficits. They also decreased neuroinflammation.

Toshiko Tanaka, an investigator at the National Institute on Aging was the lead author of a study published in 2018 that leveraged similar techniques and also found proteins associated with aging. One of the great things about these advancements, she told NPR, is its becoming a lot cheaper to measure a lot of these molecules, so bigger studies and more studies can assess the same proteins.

At this time, the research is a way off from specifically identifying proteins associated with aging, but its a very good first step.

Paola Sebastiani, a biostatistician at Boston University who has also conducted research on aging and blood proteins, told NPR that, For a long time we have focused, in the field of healthy aging, on genetics.

However, we generally cant modify genesalthough gene therapies and genome editing techniques such as CRISPR are starting to do just that. Sebastiani points out that one of the interesting aspects of these blood proteins is they can be inhibited or blocked or modified using a variety of biochemical and small molecule process that theoretically be used to improve health and slow the aging process.

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Aging in Waves: New Findings on Aging-Related Proteins in the Blood - BioSpace

Recommendation and review posted by Bethany Smith

DUBLIN, Dec. 17, 2019 /PRNewswire/ -- The "Red Biotechnology Market - Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 2019-2027" report has been added to ResearchAndMarkets.com's offering.

This study on the red biotechnology market provides readers with a holistic market overview, through an extensive analysis of the market.

The report analyzes the market with regards to the historical and current data to provide a forecast for the period of 2019-2027. Actionable insights and findings pertaining to the red biotechnology market help report readers take major business decisions that support their long-term business growth.

The study includes the compilation of the assessment of significant market dynamics such as key industry trends and major developments carried out by leading players, along with a detailed competitive assessment. The study is divided into key sections to provide readers with an individual understanding of the various aspects of the red biotechnology market.

Key Questions Answered

Key Topics Covered

1. Global Red Biotechnology Market - Preface1.1. Market Definition and Scope1.2. Market Segmentation1.3. Key Research Objectives1.4. Research Highlights

2. Assumptions and Research Methodology2.1. Red Biotechnology Market Definition2.2. Red Biotechnology Market Taxonomy

3. Executive Summary : Global Red Biotechnology Market3.1. Introduction3.1.1. Definition3.1.2. Industry Evolution/Developments3.2. Overview3.3. Market Dynamics3.3.1. Drivers3.3.2. Restrains3.3.3. Opportunities3.4. Global Red Biotechnology Market Analysis and Forecast, 2017-20273.5. Market Revenue Projections (US$ Bn)4. Market Outlook4.1. Pipeline Analysis4.2. Mergers & Acquisitions

5. Global Red Biotechnology Market Analysis and Forecast, by Application 5.1. Introduction5.2. Key Findings/Developments, by Type 5.3. Global Red Biotechnology Market Value (US$ Bn) Forecast, by Application, 2017-20275.3.1. Biopharmaceutical Production5.3.2. Gene Therapy5.3.3. Pharmacogenomics5.3.4. Genetic Testing5.4. Global Red Biotechnology Market Attractiveness Analysis, by Application

6. Global Red Biotechnology Market Analysis and Forecast, by End-user 6.1. Introduction6.2. Key Findings/Developments, by End-user 6.3. Global Red Biotechnology Market Value (US$ Bn) Forecast, by End-user, 2017-20276.3.1. Pharmaceutical Industry 6.3.2. CMO & CRO6.3.3. Research Institutes6.3.4. Others 6.4. Global Red Biotechnology Market Attractiveness Analysis, by End-user

7. Global Red Biotechnology Market Analysis and Forecast, by Region/sub-Region7.1. Key Findings/Developments 7.2. Global Red Biotechnology Market Value (US$ Bn) Forecast, by Region/Sub-Region, 2017-20277.2.1. North America7.2.2. Europe7.2.3. Asia-Pacific 7.2.4. Latin America7.2.5. Middle East & Africa (MEA)7.3. Global Red Biotechnology Market Attractiveness Analysis, by Region/Sub-Region

8. North America Red Biotechnology Market Analysis and Forecast

9. Europe Red Biotechnology Market Analysis and Forecast

10. Asia-Pacific Red Biotechnology Market Analysis and Forecast

11. Latin America Red Biotechnology Market Analysis and Forecast

12. Middle East & Africa Red Biotechnology Market Analysis and Forecast

13. Competition Landscape13.1. Market Player - Competition Matrix 13.2. Market Share Analysis, by Company (2018)13.3. Company Profiles13.3.1. Pfizer Inc.13.3.2. Biogen Inc.13.3.3. Amgen Inc.13.3.4. AstraZeneca PLC13.3.5. Gilead Sciences Inc.13.3.6. Celgene Corporation13.3.7. F. Hoffmann-La Roche Ltd.13.3.8. Merck KGaA13.3.9. Regeneron Pharmaceuticals Inc.13.3.10. Takeda Pharmaceutical Company Limited

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Insights Into the World's Red Biotechnology Market, 2017-2019 & 2027 - Emerging Opportunities, Revenue Projections, Leading Players - PRNewswire

Recommendation and review posted by Bethany Smith

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

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

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

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Beyond the introduction, this report has four major components:

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

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

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

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

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

To buy the full report, please click here.

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

Recommendation and review posted by Bethany Smith

As 2019 comes to a close, Charles River Laboratories (CRL) has been busy. Following a partnership with Bit Bio announced earlier this month, this morning, CRL announced the acquisition of HemaCare for approximately $380 million in cash.

California-based HemaCare is a provider of human-derived cellular products for the cell therapy market. The company provides biomaterials, including a wide range of human primary cell types. Also, HemaCare supplies cell processing services to support the discovery, development, and manufacture of cell therapies, including allogeneic and autologous programs. Combined with CRLs integrated, early-stage portfolio of discovery, safety assessment, and manufacturing support services, the acquisition of HemaCare will create a comprehensive solution for cell therapy developers and manufacturers worldwide to help accelerate their critical programs from basic research and proof-of-concept to regulatory approval and commercialization, the company said in its announcement. For its clients, CRL believes the addition of HemaCare will enhance client retention and accelerate biopharmaceutical clients speed-to-market.

As more and more cell and gene therapies are expected to be approved over the next several years, CRL believed now was the time to push into the space. James C. Foster, president and chief executive officer of CRL, said that in order to continue to enhance the companys abilities to support the research efforts of its clients, CRL needed to expand its scientific capabilities into this high-growth market.

HemaCare advances the development of life-saving cell therapies through the use of its high-quality cellular products that represent critical inputs to these therapeutics. The addition of HemaCares innovative cell therapy products and services to our integrated, early-stage solutions will create a unique, go-to partner for clients to work with Charles River across a comprehensive cell therapy portfolio from idea to novel therapeutic, Foster said in a statement.

Foster added that HemaCares expertise is well-recognized and pointed to the fact the company has worked on all of the cell therapy drugs approved by the U.S. Food and Drug Administration to date. He said the addition of HemaCare will enhance CRLs value proposition for its clients and will also drive profitable revenue and generate value for the companys shareholders.

The proposed purchase price of $380 million equates to $25.40 per share of HemaCare. That represents a 27% premium to HemaCares closing price on Dec. 13. The acquisition is expected to close early in the first quarter of 2020.

For CRL, the acquisition of HemaCare follows a strategic partnership forged with Bit Bio earlier this month. Bit Bio reprograms human cells for use in research, drug discovery, and cell therapies. Through its partnership with Bit Bio, CRL said it will offer clients access to an expanding suite of authentic human cells through their use in target discovery, validation and screening services.

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Charles River Laboratories Moves into Cell Therapies with $380 Million Acquisition of HemaCare - BioSpace

Recommendation and review posted by Bethany Smith

The research study on Global Gene Therapy market 2019 presents an extensive analysis of current market size, drivers, trends, opportunities, challenges, as well as key market segments. Further, it explains various definitions and classification of the Gene Therapy industry, applications, and chain structure.

In continuation of this data, the Gene Therapy report covers various marketing strategies followed by key players and distributors. Also explains marketing channels, potential buyers and development history. The intent of global Gene Therapy research report is to depict the information to the user regarding market forecast and dynamics for the upcoming years.

The study lists the essential elements which influence the growth of Gene Therapy industry. Long-term evaluation of the worldwide market share from diverse countries and regions is roofed within the report. Additionally, includes type wise and application wise consumption figures.

After the basic information, the global Gene Therapy Market study sheds light on the technological evolution, tie-ups, acquisition, innovative business approach, new launches and revenue. In addition, the Gene Therapy industry growth in distinct regions and R&D status are enclosed within the report.

The study also incorporates new investment feasibility analysis of Gene Therapy. Together with strategically analyzing the key micro markets, the report also focuses on industry-specific drivers, restraints, opportunities, and challenges in the Gene Therapy market.

Highlights of Global Gene Therapy Market Report:

Table of Content:01: Gene Therapy Market Overview02: Global Gene Therapy Sales, Revenue (value) and Market Share by Players03: Gene Therapy Market Sales, Revenue (Value) by Regions, Type and Application (2014-2018)04: Region wise Top Players Gene Therapy Sales, Revenue and Price05: worldwide Gene Therapy Industry Players Profiles/Analysis06: Gene Therapy Manufacturing Cost Analysis07: Industrial Chain, Gene Therapy Sourcing Strategy and Downstream Buyers08: Gene Therapy Marketing Strategy Analysis, Distributors/Traders09: Gene Therapy Industry Effect Factors Analysis10: Global Gene Therapy Market Forecast (2019-2029)11: Gene Therapy Research Findings and Conclusion12: Appendix

This post was originally published on Market Research Sheets

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Gene Therapy Market by Industry Size, Share, Competitors' Landscape, And Segmentation Including: Regional Segment, Type Segment, Industry Segment,...

Recommendation and review posted by Bethany Smith

Sarepta Therapeutics (SRPT) recently publicized that the FDA approved Vyondys 53. This approval came only a few months after the drug received a CRL due to concerns over the risk of infection at the infusion site and renal toxicity. The company filed an appeal and it looks like the matters raised in the CRL were quickly resolved. SRPT has shot up over 30% following the news, and but is still trading well under its 52-week high of $158.80. Not only is this approval important for the companys commercial outlook, but it also reinstalls some confidence in the companys gene therapy pipeline and technology. Despite the recent big move to the upside, I still see SRPT to be discounted at this current price and could be a lucrative long-term investment.

I intend to provide a brief background on Vyondys 53 and its journey through the FDA. In addition, I discuss why I still think SRPT is still worth a speculative investment. Finally, I reveal my plans for my SRPT position for 2020.

Vyondys 53 is similar to Sareptas Exondys 51, which allows the skipping over gene mutation that inhibits patients with DMD from producing dystrophin. Exondys 51 works in roughly 13% of the roughly 15K boys in the U.S. who have DMD. Although Vyondys 53 will only take aim at 8% of the DMD population, it is still the second most common DMD mutation. Vyondys 53 will be priced at $300K a year per 20kg, so some patients could be paying over $1M a year for the treatment.

Figure 1: DMD and Exon Skipping (Source: SRPT)

Vyondys 53 was an accelerated approval, so Sarepta will need to conduct post-marketing studies to confirm its safety and efficacy. Sarepta is already enrolling patients in their confirmatory trial and should have the results in 2024.

Back in August, Vyondys 53 received a CRL primarily due to renal toxicity concerns from pre-clinical data...at a dose 10 times greater than what was submitted in the NDA. I have seen some ridiculous reasons for the FDA sending a CRL, but citing preclinical data at 10x dosage was by far the most outrageous. I have to imagine almost every drug or therapy is toxic at a 10x dosage. To be honest, I was convinced the FDAs CRL was more than suspicious, but it appears they have backtracked and granted Sarpetas appeal.

Not only is this a win for Sarepta, but it is also a win for gene therapy and the companies who are attempting to develop gene therapeutics. Most of the leading gene therapy stocks took a hit following the CRL as the prospects for gene products making through the FDA's scrutiny seemed to be degrading. Now that the FDA has recognized their error, investors should have renewed confidence in the regulatory and commercial outlook for gene therapies in the US.

As I mentioned before, Vyondys 53 approval has reassured the market that Sarepta's platform and technology are capable of producing more marketable gene therapies. Looking at Sarepta's pipeline (Figure 2) the company can see a great amount of potential value.

Figure 2: SRPT Pipeline (Source: SRPT)

Sarepta is now pushing their next-generation PPMO RNA platform. The company will have safety and dosing insight in 2020. If Sareptas PPMO shows encouraging results they plan to conduct additional research on new therapeutic targets using the PPMO platform.

In terms of microdystrophin manufacturing, the company has completed building out of Sareptas single-use microdystrophin manufacturing facility in Lexington, Massachusetts. In addition, the company also has dedicated suites with Paragon in Maryland, which is expected to have a greater capacity than the Lexington facility. So, it appears the company will have the capacity to support more commercial products as they continue to cross the FDA finish line.

Not only is the company have a strong R&D department, but their commercial team has demonstrated some success with payers, physicians, and prospective patients. The company has recorded revenue growth since Q4 of 2016 and is expected to continue to record sequential revenue growth for the next 7 years (Figure 3).

Figure 3: SRPT Estimated Annual Revenue Growth (Source: Seeking Alpha)

Exondys 51 continues to perform well with Q3 sales above consensus at $99M, which was a 26% increase over Q3 of last year. This prompted the company to boost the 2019 revenue guidance range from $365M to $375M to a range of $370M to $380M for Exondys 51. In fact, Exondys 51 numbers have grown quarter-over-quarter for over the past 3 years.

Another reason to be bullish on SRPT is the companys current financial position, which over $1B. Their healthy cash position will allow the company to fast-track Sareptas growing pipeline and support company operations. Indeed, the company revealed a net loss of $126.3M in Q3, so the company is still incinerating cash. However, Sarepta recently announced an agreement with Pharmakon Advisors, LP, for $500M to be given two $250M tranches. The first will be obtainable after closing and the second will be available at Sareptas option until December 31st of next year. This recent funding not only allows the company to keep the pedal down on R&D, but it was a non-dilutive fundraising event.

What is more, one of Sarepta's potential competitors Wave Life Sciences (WVE) recently announced that it has decided to drop its DMD exon skipping programs. This decision comes after some disappointing data for their exon 51 and exon 53 skipping product candidates. Not only has Sarepta lost a potential competitor, but Wave's exit from DMD shows how advanced Sarepta's exon-skipping products really are.

As a result, I believe Sarepta has the products, pipeline, commercial ability, manufacturing capacity, and the financials to execute on their long-term growth strategy. Returning to figure 3, I expect Sarepta to follow this growth trajectory for the next several years, and will rapidly grow into its current valuation. Sareptas aspiring strategy has created one of the biggest multi-platform genetic medicine pipelines in biotech, which contains over 25 active programs across Sareptas RNA and gene therapy platforms that will be providing multiple catalysts in the coming years. Considering the points above, I still see SRPT to be worthy of speculative buy that could become a lucrative long-term investment.

What's My Plan?

I took a leap of faith following the CRL and added to my speculative SRPT position. Now that I am in the green, I plan on holding my shares for at least four more years and will consider holding if the company is able to get their current pipeline across the finish line. In the meantime, I am going to take advantage of SRPT's volatility and will wait for a pull-back to add to my position. However, I am not going to go all-in until the company has completed their confirmatory studies.

What is more, Sareptas approval has bolstered my confidence in my other gene therapy investments and will look to add to those positions in the near future.

Disclosure: I am/we are long SRPT. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Original post:
Sarepta Therapeutics: FDA Approval Reinstalls Confidence In The Pipeline - Seeking Alpha

Recommendation and review posted by Bethany Smith

The research report Cancer Gene Therapy Market Global Industry Analysis 2019 2025 offers precise analytical information about the Cancer Gene Therapy market. The report identifies top players in the global market and divides the market into several parameters such as major drivers market strategies and imposing growth of the key players. Worldwide Cancer Gene Therapy Industry also offers a granular study of the market dynamics, segmentation, revenue, share forecasts and allows you to make superior business decisions. The report serves imperative statistics on the market stature of the prominent manufacturers and is an important source of guidance and advice for companies and individuals involved in the Cancer Gene Therapy industry.

This Cancer Gene Therapy market report bestows with the plentiful insights and business solutions that will support our clients to stay ahead of the competition. This market report contains categorization by companies, region, type, and application/end-use industry. The competitive analysis covered here also puts light on the various strategies used by major players of the market which range from new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and many others that leads to increase their footprints in this market. The transparent research method carried out with the right tools and methods makes this Cancer Gene Therapy market research report top-notch.

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Competitive Landscape

Global Cancer Gene Therapy market is highly split and the major players have used numerous tactics such as new product launches, acquisitions, innovation in products, expansions, agreements, joint ventures, partnerships, and others to increase their footprints in this market.

Key players profiled in the report include: Adaptimmune, GlaxoSmithKline, Bluebirdbio, Merck, Celgene, ShanghaiSunwayBiotech, BioCancell, ShenzhenSiBionoGeneTech, SynerGeneTherapeutics, OncoGenexPharmaceuticals, GeneluxCorporation, CellGenesys, Advantagene, GenVec, BioCancell, Celgene, EpeiusBiotechnologies, IntrogenTherapeutics, ZiopharmOncology, ShenzhenSiBionoGeneTech, AltorBioscience

Market Segmentation

Cancer Gene Therapy Market report segmentation on Major Product Type:GeneInducedImmunotherapy, OncolyticVirotherapy, GeneTransfer

Market by Application: Here, various application segments of the global Cancer Gene Therapy market are taken into account for the research study.

Hospitals, DiagnosticsCenters, ResearchInstitutes

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Regional Analysis

The Cancer Gene Therapy market report keenly emphasizes on industrial affairs and developments, approaching policy alterations and opportunities within the market. The regional development methods and its predictions are explained in every key point that specifies the general performance and issues in key regions such as North America, Europe, Asia Pacific, Middle East, South America, and Middle East & Africa (MEA). Various aspects such as production capability, demand, product value, material parameters and specifications, distribution chain and provision, profit and loss, are explained comprehensively in the market report.

Key Questions Answered in Global Cancer Gene Therapy Market Report:-

What will the market growth rate, overview, and analysis by type of global Cancer Gene Therapy Market in 2026?

What are the key factors driving, analysis by applications and countries Global Cancer Gene Therapy Market?

What are dynamics, this summary includes analysis of the scope and price analysis of top players profiles of Global Cancer Gene Therapy Market?

Who are the opportunities, risk and driving forces of the global Cancer Gene Therapy Market?

Who are the opportunities and threats faced by the vendors in the Global Cancer Gene Therapy Market?

What are the Global Cancer Gene Therapy market opportunities, market risk and market overview of the Market?

Thanks for reading this article, you can also get individual chapter wise section or region wise report versions like North America, Europe or Asia. Also, If you have any special requirements, please let us know and we will offer you the report as you want.

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Cancer Gene Therapy Market Expected to Deliver Dynamic Progression until 2028| Adaptimmune, GlaxoSmithKline, Bluebirdbio, Merck - E-Industry News

Recommendation and review posted by Bethany Smith

The global Cancer Gene Therapy market report offers a significant assistant that helps the reader to get a thorough understanding of the value chain analysis. The latest trends, developments, promotion, strategies, and many more provide an uphold success. To reveal the general market trends coupled with conditions and variable tendencies the global Cancer Gene Therapy market report acts as a bible for the reader. The report offers reliable information in relation to the market with proper planning techniques. This report is presented in a precise fashion that records state-of-art information regarding preferences, consumers demands, attitudes, and variable tendencies about the specific product pipelines. The report also aims to offer an open discussion about the global Cancer Gene Therapy market.

About Cancer Gene Therapy Market

Gene therapy is the insertion of a functional gene into the cells of a patient to correct an inborn error of metabolism, in order to alter or repair an acquired genetic abnormality, and to provide a new function to a cell. Owing to high success rate throughout the preclinical and clinical trials, cancer gene therapy is gaining popularity. Improvised technological advancements, increased adoption of developing genomic technologies such as next generation sequencing (NGS) and high density micro array are some of the major factors that will drive the global cancer gene therapy market in next upcoming years. Cancer cells can be differentiated from their usual neighbors on the basis of specific phenotypic changes, such as rapid division rate, attack of new cellular areas, high metabolic rate, and altered shape.

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The insights of the report cover the wide spectrum of the global Cancer Gene Therapy market. With deep insights the reader gets the feasibility while reading that focuses upon the market dynamics governing the trajectory. The in-depth analytical study conducted by the researchers offers strengthens the decision making of the specific market and provoke the analysts to come up with the solution. The report further includes figures and stats coupled with significant compound growth. The compound growth rate directs the reader or analyst to envisage the market growth in base year and forecast time frame.

Competitive Landscapes:

The competitive landscapes are a must-include chapter involving the global players that withstand the competition for the global Cancer Gene Therapy market. This assures the market participants to develop effective strategies to set a benchmark to adopt a significant market position. Further, a competitive environment helps them to determine not only potential advantages but also varied obstacles for the global Cancer Gene Therapy market. In this chapter, the players can examine various strategies and analyze the competitiveness among the players.

List of the Key Players Cited in the Report:

OncoGenex PharmaceuticalsSynerGene TherapeuticsShenzhen SiBiono GeneTechBioCancellShanghai Sunway BiotechCelgeneMerckBluebird bio Inc.GlaxoSmithKlineAdaptimmune

Market Segmentation

On the basis of types, the global Cancer Gene Therapy market is fragmented into

Product 1Product 2

Based on applications, the global Cancer Gene Therapy market is split into:

HospitalsDiagnostics CentersResearch Institutes

With the successive chapters of the Cancer Gene Therapy market, the report further throws the spotlight upon the thorough assessment of the segments at the global outlook. This supports the reader to get a view about the products pipelines, technology, services, end-users, and regions in the overall market. The segment analysis chapter further involves the factors responsible for driving the market on one side while restraining the market on the other side.

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Regional Segmentation

With a wide trend and factors influencing the market that directs regional as well as the primary direction of growth are swung by local market players and unique market drivers. The market study is uplifted at regional as well as country level. This helps to determine the past record and future records through revenue coupled with volume price analysis to involve the region-wise leaders based on the market share and revenue.

Major geographies covered in the report includeNorth America, Europe, Asia-Pacific, South America, and the Middle East & Africa.

Sub-regions includes

The years that were considered for the study of this report are the following:

The study objectives are:

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Cancer Gene Therapy Market 2019| Key Players, Industry Share, Trends, Growth Analysis, Regional Demand and Future Insights by 2026 - Techi Labs

Recommendation and review posted by Bethany Smith

"Our impressive portfolio of life sciences companies underscores the tremendous opportunity for this industry in Central Ohio."

COLUMBUS, Ohio (PRWEB) December 17, 2019

Today, Rev1 Ventures, the investor startup studio that combines capital and strategic services to help startups scale and corporations innovate, is announcing the launch of its second fund focused on life sciences companies. The $15MM Rev1 Catalyst Fund II with investment from The Ohio State University, Nationwide Childrens Hospital and Rev1 is three times the size of the first Life Sciences fund and aims to deliver critical investment capital and support for research-based spinouts and healthcare innovators. As a result of the success of the Rev1 Life Sciences Fund I, which has helped industry trailblazers commercialize technologies and build successful companies bringing novel therapies and solutions to the market, this fund will continue to help Rev1 fulfill its mission to propel the creation of high growth companies in the region.

"Our impressive portfolio of life sciences companies underscores the tremendous opportunity for this industry in Central Ohio, said Tom Walker, president and CEO of Rev1 Ventures. We are proud to help innovators fulfill their potential and bring life-saving technologies and therapies to more people. By aligning champions of healthcare innovation, including prominent research institutions like The Ohio State University and Nationwide Childrens Hospital, we are helping to foster life sciences innovation and support entrepreneurs and scientists as they build and grow new companies. The Rev1 Catalyst Fund II is dedicated to expanding the infrastructure to support life sciences and attracting talent and like-minded investors to fuel commercialization.

The Rev1 Life Sciences Fund I supports an impressive portfolio of companies driving the creation of innovative therapies and solutions, and has generated more than $250MM in exits, including the recent acquisitions of Celenex and Myonexus.

Ohio State is committed to supporting our accomplished researchers at every point along the innovation pipeline, said Cheryl Turnbull, senior director for new ventures at Ohio State. Catalyst Fund II supports Ohio States land grant mission and will help advance the promising life sciences technologies being developed at Ohio State and in Columbus, accelerating medical breakthroughs to market where they can have a positive impact on people throughout the world.

We have many significant success stories about the power of this approach--from the worlds first digital pathology cloud-based platform, Deep Lens, to Milo Biotechnology, a startup working to reverse muscular dystrophy using gene therapy, said Matt McFarland, vice president of commercialization and industry relations at Nationwide Children's Hospital. Myonexus, a Nationwide Childrens spinout that is developing the first-ever corrective gene therapy for limb-girdle muscular dystrophies, was recently acquired by Sarepta Therapeutics. Columbus has the foundational elements in place, so we anticipate this hotbed of activity will only get more robust.

Rev1 and Nationwide Childrens Hospital have been paramount to our success as well as to our ability to take a development and build a business around it, said Dave Billiter, co-founder and CEO of Deep Lens. In this field, it is critical that your investors and partners understand the industry, your potential, your market. The participation of these institutions and the fact that they are in our own backyard, is truly impacting lives and changing healthcare.

For more information about the fund and to learn more about the life sciences startups working with Rev1 Ventures, visit: https://www.rev1ventures.com/our-companies/.

About Rev1 VenturesRev1 Ventures is the investor startup studio that combines capital and strategic services to help startups scale and corporates innovate. Based in the Midwest, and in the number one city for scaling startups, Rev1 aligns innovators and founders with corporate and research partners to access customers and markets, helping entrepreneurs build great companies. With a proven track record of identifying, guiding and investing in high potential startups, Rev1 helps companies solve real problems for markets in need of real solutions. Rev1 has $100MM in capital under management, providing a capital continuum from corporate and community partners, as well as the Ohio Third Frontier. Rev1 is the most active seed investor in Ohio five years running, according to Pitchbook. For more information, visit http://www.rev1ventures.com.

About The Ohio State UniversityThe Ohio State University was founded in 1870 following the Land-Grant College Act of 1862. Classes began in the fall of 1873 with 24 students. The first class of six men graduated in 1878, followed by the first woman graduate in 1879. Today, Ohio State is among the largest and most respected public research universities in the nation, with more than 66,000 students on six campuses. The spring 2017 graduating class of over 11,500 was the largest in school history. Ohio State is known best for its vibrant student experience, research excellence, athletic prowess and highly engaged Buckeye family. The true strength of Ohio State is its people. Buckeye Nation includes more than 45,000 faculty and staff as well as 550,000 alumni living and working in 170 countries across the globe.

About Nationwide Childrens HospitalNamed to the Top 10 Honor Roll on U.S. News & World Reports 2019-20 list of Best Childrens Hospitals, Nationwide Childrens Hospital is one of Americas largest not-for-profit freestanding pediatric health care systems providing wellness, preventive, diagnostic, treatment and rehabilitative care for infants, children and adolescents, as well as adult patients with congenital disease. Nationwide Childrens has a staff of more than 13,000 providing state-of-the-art pediatric care during more than 1.5 million patient visits annually. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Childrens physicians train the next generation of pediatricians and pediatric specialists. The Abigail Wexner Research Institute at Nationwide Childrens Hospital is one of the Top 10 National Institutes of Health-funded freestanding pediatric research facilities. More information is available at NationwideChildrens.org.

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Rev1 Ventures Launches $15MM Fund with The Ohio State University and Nationwide Children's Hospital to Support Research-based Healthcare Innovation -...

Recommendation and review posted by Bethany Smith

Coherent Market Insights released a new market study on 2018-2026 Cell and Gene Therapy Market with 100+ market data Tables, Pie Chat, Graphs & Figures spread through Pages and easy to understand detailed analysis. At present, the market is developing its presence. Report offering you more creative solutions that combine our deep geographic experience, intimate sector knowledge and clear insights into how to create value in your business. The research study provides estimates for 2018-2026 Cell and Gene Therapy Market Growth Forecast till 2026*.

The Cell and Gene Therapy Market research report covers the present scenario and the growth prospects of the global Cell and Gene Therapy industry. The report enlists several important factors, starting from the basics to advanced Market intelligence which plays a crucial part in strategizing.

Get Free Sample Copy of the Report @ https://www.coherentmarketinsights.com/insight/request-sample/2475

Key companies (manufacturing situations, size and production, product specifications etc.) Amgen, Biogen, BioMarin Pharmaceuticals, Bristol-Myers Squibb Company, GlaxoSmithKline, Novartis, Pfizer, Regeneron Pharmaceuticals and Sanofi, Spark Therapeutics, Agilis Biotherapeutics, Angionetics AVROBIO, Freeline Therapeutics, Horama, MeiraGTx, Myonexus Therapeutics, Nightstar Therapeutics, Kolon TissueGene, Inc., JCR Pharmaceuticals Co., Ltd., and MEDIPOST.

Cell and Gene Therapy Market Report provides key statistics on the market status of the Cell and Gene Therapy manufacturers and is a valuable source of guidance and direction for companies and individuals interested in the Cell and Gene Therapy industry. The Cell and Gene Therapy Market report also presents the vendor landscape and a corresponding detailed analysis of the major vendors operating in the market.

Cell and Gene Therapy Market report analyses the Market potential for each geographical region based on the growth rate, macroeconomic parameters, consumer buying patterns, and Market demand and supply scenarios.

Regions of Cell and Gene Therapy Market:

North America: United States, Canada, Mexico

Europe: Germany, France, UK, Russia, Italy, Rest of Europe

Middle East Africa: Turkey, Egypt, South Africa, GCC Countries, Rest of Middle East & Africa

Asia-Pacific: China, India, Australia, Japan, South Korea, Indonesia, Malaysia, Philippines, Thailand, Vietnam

Download Free PDF Brochure of Research Report @ https://www.coherentmarketinsights.com/insight/request-pdf/2475

In the end, the report makes some important proposals for a new project of Cell and Gene Therapy Industry before evaluating its feasibility. Overall, the report provides an in-depth insight into the global Cell and Gene Therapy industry covering all important parameters.

The Report provides a detailed Cell and Gene Therapy Industry overview along with the analysis of industrys gross margin, cost structure, consumption value, and sale price, Processing Techniques, Network Management, Services Offered, Related Software Market, Social Media Marketing, Cost Structure, Supply Chain, Development Management Techniques, Retailers Analysis, Financial Support, business Strategies, Marketing Channels, Market Entry Strategies, Industry Development Challenges and Opportunities, Investment Plans, Economic Impact on Cell and Gene Therapy Market.

This report can be customized to meet the desired requirements. Please connect with our analyst, who will ensure you get a report that suits your needs.

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Our team is of the most vital cog in our robust machinery that gives us the ability to deliver independent insight relying on our cognitive defusion training module. This allows for an objective and unbiased assessment of the market. We pride ourselves in my constantly striving to update our extremely in-depth understanding of the market by closely monitoring and analyzing markets, trends, and emerging best practices, across allfathomable industries under the sun. This enables us to equip our valued clientele with key decisive inputs to capitalize on lucrative growth opportunities in the market and to follow firmly position themselves on a high growth path in the future.

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Rapid Expansion Projected for Cell and Gene Therapy Market by 2026 - Galus Australis

Recommendation and review posted by Bethany Smith

MELVILLE, N.Y., Dec. 16, 2019 (GLOBE NEWSWIRE) -- BioRestorative Therapies, Inc. (BioRestorative or the Company) (OTC: BRTX), a life sciences company focused on stem cell-based therapies, announced today feature coverage in the news outlet, IEEE Pulse, a magazine of the IEEE Engineering in Medicine and Biology Society. According to IEEE, it is the worlds largest technical professional organization for the advancement of technology.

To view the IEEE Pulse Magazines article featuring BioRestorative, click here.

The published cover-story article features commentary from Francisco Silva, Chief Scientist and Vice President of Research and Development for BioRestorative, regarding BRTX-100, the Companys lead therapeutic candidate for chronic lumbar disc disease. Once the U.S. Food and Drug Administration (FDA) authorizes the sale of BRTX-100, we would ship it to your doctor, and with a 30-minute procedure the material would be injected into your disc in a 1.5 ml solution, explains Silva. He elaborates on the product, discussing growing and expanding stem cells from the patients bone marrow under hypoxic conditions that mimic those in the normal intervertebral space. We are enriching the cells to be able to survive in this harsh environment, says Silva.

In addition to BRTX-100, the magazine article also highlights BioRestoratives other research pursuit, its ThermoStem program, utilizing brown adipose (fat) derived stem cells to target treatment of metabolic diseases and disorders, like diabetes, obesity and hypertension.

About BioRestorative Therapies, Inc.

BioRestorative Therapies, Inc. (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders:

Disc/Spine Program (brtxDISC): Our lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. We intend that the product will be used for the non-surgical treatment of painful lumbosacral disc disorders. The BRTX-100 production process utilizes proprietary technology and involves collecting a patients bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure, BRTX-100 is to be injected by a physician into the patients damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. We have received authorization from the Food and Drug Administration to commence a Phase 2 clinical trial using BRTX-100 to treat persistent lower back pain due to painful degenerative discs.

Metabolic Program (ThermoStem): We are developing a cell-based therapy to target obesity and metabolic disorders using brown adipose (fat) derived stem cells to generate brown adipose tissue (BAT). BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in the body may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes.

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including, without limitation, whether the Company will be able to consummate the private placement and the satisfaction of closing conditions related to the private placement and those set forth in the Company's Form 10-K filed with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.

CONTACT:Email: ir@biorestorative.com

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BioRestorative Therapies Featured in IEEE Pulse Magazine's Cover Story About Stem Cell Therapies for Low Back Pain - GlobeNewswire

Recommendation and review posted by Bethany Smith

GREEN BAY, Wi. (WBAY) - More than 10,000 people couldn't find a match for a bone marrow transplant last year. The family of one Wisconsin teenager in desperate need of a match decided to host their own donor drive during Sunday's Packers game.

Nick Parins, who lives in Land O Lakes, Wisconsin, has T-Cell Lymphoma and needs a bone marrow transplant, but currently there is no match for him.

Its very frustrating for us, his family and friends, that theres so many people out there and so many people in the bank right now but theres just not the match for him, said Erick St. Aubin, Nicks cousin.

On the same day Nick turned 18, Eric St. Aubin and other family and friends gathered just outside Lambeau Field to host a bone marrow donor drive in his honor.

Right after kickoff today we facetimed with him to sing him happy birthday and it was pretty emotional, said St. Aubin.

The family partnered with Be The Match to set up the registration booth. People interested in putting themselves on the global donor registry only needed to provide some basic information and take a cheek swab test.

Its not saying youre signing up today and youre going to donate tomorrow. Thats really far from the truth, said Be The Match Community Engagement Specialist. The reality is its pretty rare to become a match and if you are its a very special thing.

Klingberg says there arent enough people on the registry to help all the patients who need bone marrow transplants.

This is their last hope. So its really frustrating when you hear people Oh, its really painful or It requires a lot of time or whatever, said Klingberg.

Klingberg says if you are a match, the donation process isnt actually all that painful, and it saves a life.

When somebody comes through and honors that commitment to donate some stem cells, thats all it took, and then this person is coming out of the hospital and going to overcome their disease, said Klingberg. Its amazing and it gives me goosebumps every time.

St. Aubin hopes the event will spread awareness and possibly lead to a life saved.

You could be a match [with someone] across the world, or you might be a match for Nick.

To register as a bone marrow donor, or for more information CLICK HERE.

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Bone marrow donor drive held in Green Bay for teen who needs a match - WBAY

Recommendation and review posted by Bethany Smith

Bone marrow aspiration and trephine biopsy are usually performed on the back of the hipbone, or posterior iliac crest. An aspirate can also be obtained from the sternum (breastbone). For the sternal aspirate, the patient lies on their back, with a pillow under the shoulder to raise the chest. A trephine biopsy should never be performed on the sternum, due to the risk of injury to blood vessels, lungs or the heart.

The need to selectively isolate and concentrate selective cells, such as mononuclear cells, allogeneic cancer cells, T cells and others, is driving the market. Over 30,000 bone marrow transplants occur every year. The explosive growth of stem cells therapies represents the largest growth opportunity for bone marrow processing systems.Europe and North America spearheaded the market as of 2016, by contributing over 74.0% to the overall revenue. Majority of stem cell transplants are conducted in Europe, and it is one of the major factors contributing to the lucrative share in the cell harvesting system market.

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In 2016, North America dominated the research landscape as more than 54.0% of stem cell clinical trials were conducted in this region. The region also accounts for the second largest number of stem cell transplantation, which is further driving the demand for harvesting in the region.Asia Pacific is anticipated to witness lucrative growth over the forecast period, owing to rising incidence of chronic diseases and increasing demand for stem cell transplantation along with stem cell-based therapy.

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Japan and China are the biggest markets for harvesting systems in Asia Pacific. Emerging countries such as Mexico, South Korea, and South Africa are also expected to report lucrative growth over the forecast period. Growing investment by government bodies on stem cell-based research and increase in aging population can be attributed to the increasing demand for these therapies in these countries.

Major players operating in the global bone marrow processing systems market are ThermoGenesis (Cesca Therapeutics inc.), RegenMed Systems Inc., MK Alliance Inc., Fresenius Kabi AG, Harvest Technologies (Terumo BCT), Arthrex, Inc. and others

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Bone Marrow Processing System Market Expected to Witness an Imperishable Growth over 2025 - Guru Online News

Recommendation and review posted by Bethany Smith

Gene therapy is fighting to enter mainstream medicine. With sickle cell disease, the fight is heating up.

Roughly two years ago, the FDA made the historic decision to approve the first gene therapy in the US, finally realizing the therapeutic potential of hacking our biological base code after decades of cycles of hope and despair. Other approvals soon followed, including Luxturna to target inherited blindness and Zolgensma, a single injection that could save children with a degenerative disease from their muscles wasting away and dying before the age of two.

Yet despite their transformative potential, gene therapy has only targeted relatively rareand often fataldisorders. Thats about to change.

This year, a handful of companies deployed gene therapy against sickle-cell anemia, a condition that affects over 20 million people worldwide and 100,000 Americans. With over a dozen therapies in the run, sickle-cell disease could be the indication that allows gene therapy to enter the mainstream. Yet because of its unique nature, sickle-cell could also be the indication that shines an unflinching spotlight on challenges to the nascent breakthrough, both ethically and technologically.

You see, sickle-cell anemia, while being one of the worlds best-known genetic diseases, and one of the best understood, also predominantly affects third-world countries and marginalized people of color in the US. So far, gene therapy has come with a hefty bill exceeding millions; few people afflicted by the condition can carry that amount. The potential treatments are enormously complex, further upping costs to include lengthy hospital stays, and increasing potential side effects. To muddy the waters even more, the disorder, though causing tremendous pain and risk of stroke, already has approved pharmaceutical treatments and isnt necessarily considered life-threatening.

How we handle gene therapies for sickle-cell could inform many other similar therapies to come. With nearly 400 clinical trials in the making and two dozen nearing approval, theres no doubt that hacking our genes will become one of the most transformative medical wonders of the new decade. The question is: will it ever be available for everyone in need?

Even those uninterested in biology have likely heard of the disorder. Sickle-cell anemia holds the crown as the first genetic disorder to be traced to its molecular roots nearly a hundred years ago.

The root of the disorder is a single genetic mutation that drastically changes the structure of the oxygen-carrying protein, beta-globin, in red blood cells. The result is that the cells, rather than forming their usual slick disc-shape, turn into jagged, sickle-shaped daggers that damage blood vessels or block them altogether. The symptoms arent always uniform; rather, they come in crisis episodes during which the pain becomes nearly intolerable.

Kids with sickle-cell disorder usually die before the age of five; those who survive suffer a lifetime of debilitating pain and increased risk of stroke and infection. The symptoms can be managed to a degree with a cocktail of drugsantibiotics, painkillers, and a drug that reduces crisis episodes but ups infection risksand frequent blood transfusions or bone marrow transplants. More recently, the FDA approved a drug that helps prevent sickled-shaped cells from forming clumps in the vessels to further combat the disorder.

To Dr. David Williams at Boston Childrens Hospital in Massachusetts, the availability of these treatmentshowever inadequatesuggests that gene therapy remains too risky for sickle-cell disease. Its not an immediately lethal diseaseit wouldnt be ethical to treat those patients with a highly risky experimental approach, he said to Nature.

Others disagree. Freeing patients from a lifetime of risks and pain seems worthy, regardless of the price tag. Inspired by recent FDA approvals, companies have jumped onto three different treatments in a bitter fight to be the first to win approval.

The complexity of sickle-cell disease also opens the door to competing ideas about how to best treat it.

The most direct approach, backed by Bluebird Bio in Cambridge, Massachusetts, uses a virus to insert a functional copy of the broken beta-globin gene into blood cells. This approach seems to be on track for winning the first FDA approval for the disorder.

The second idea is to add a beneficial oxygen-carrying protein, rather than fixing the broken one. Here, viruses carry gamma-globin, which is a variant mostly present in fetal blood cells, but shuts off production soon after birth. Gamma-globin acts as a repellent that prevents clotting, a main trigger for strokes and other dangerous vascular diseases.

Yet another idea also focuses on gamma-globin, the good guy oxygen-carrier. Here, rather than inserting genes to produce the protein, the key is to remove the breaks that halt its production after birth. Both Bluebird Bio and Sangamo Therapeutics, based in Richmond, California, are pursing this approach. The rise of CRISPR-oriented companies is especially giving the idea new promise, in which CRISPR can theoretically shut off the break without too many side effects.

But there are complications. All three approaches also tap into cell therapy: blood-producing cells are removed from the body through chemotherapy, genetically edited, and re-infused into the bone marrow to reconstruct the entire blood system.

Its a risky, costly, and lengthy solution. Nevertheless, there have already been signs of success in the US. One person in a Bluebird Bio trial remained symptom-free for a year; another, using a CRISPR-based approach, hasnt experienced a crisis in four months since leaving the hospital. For about a year, Bluebird Bio has monitored a dozen treated patients. So far, according to the company, none has reported episodes of severe pain.

Despite these early successes, advocates worry about the actual impact of a genetic approach to sickle-cell disease.

Similar to other gene therapies, the treatment is considered a last-line, hail Mary solution for the most difficult cases of sickle cell disease because of its inherent risks and costly nature. Yet end-of-the-line patients often suffer from kidney, liver, and heart damages that make chemotherapy far too dangerous.

Then theres the problem of global access. Some developing countries, where sickle-cell disease is more prevalent, dont even have consistent access to safe blood transfusions, not to mention the laboratory equipment needed for altering blood-producing stem cells. Recent efforts in education, early screening, and prevention have also allowed people to live longer and reduce the stigma of the disorder.

Is a $1 million price tag ever attainable? To combat exhorbitant costs, Bluebird Bio is offering an installment payment plan for five years, which can be terminated anytime the treatment stops working. Yet for patients in South Africa, India, or Cambodia, the costs far exceed the $3 per month price tag for standard treatment. Even hydroxyurea, the newly-approved FDA drug to reduce crisis pain episodes, is just a fraction of the price tag that comes with gene therapy.

As gene therapy technologies are further refined and their base cost reduced, its possible that overall costs will drop. Yet whether these treatments will be affordable in the long run remains questionable. Even as scientists focus on efficacy rather than price tag, NIH director Dr. Francis Collins believes not thinking about global access is almost unethical. There are historical examples for optimism: vaccines, once rather fringe, now touch almost every corner of our world with the help of scientific knowledge, advocacy groups, andfundamentallyproven efficacy.

With the rise of gene therapy, were now in an age of personalized medicine beyond imagination. Its true that perhaps sickle-cell disease genetic therapies arent quite there yet in terms of safety and efficacy; but without tackling access issues, the therapy will be stymied in its impact for global good. As genetic editing tools become more powerful, gene therapy has the potential to save even more livesif its made accessible to those who need it most.

Image Credit: Image by Narupon Promvichai from Pixabay

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Gene Therapy for Sickle-Cell Anemia Looks Promisingbut It's Riddled with Controversy - Singularity Hub

Recommendation and review posted by Bethany Smith

The "Hematopoietic Stem Cell Transplantation (HSCT) Market" report contains data that has been carefully analyzed in the various models and factors that influence the industrial expansion of the Hematopoietic Stem Cell Transplantation (HSCT) market. An assessment of the impact of current market trends and conditions is also included to provide information on the future market expansion. The report contains comprehensive information on the global dynamics of Hematopoietic Stem Cell Transplantation (HSCT), which provides a better prediction of the progress of the market and its main competitors [Regen Biopharma Inc, China Cord Blood Corp, CBR Systems Inc, Escape Therapeutics Inc, Cryo-Save AG, Lonza Group Ltd, Pluristem Therapeutics Inc, ViaCord Inc]. The report provides detailed information on the future impact of the various schemes adopted by governments in different sectors of the world market.

The Hematopoietic Stem Cell Transplantation (HSCT) market report is crafted with figures, charts, tables, and facts to clarify, revealing the position of the specific sector at the regional and global level. The report also provides a brief summary of all major segments, such as [Autologous], with more detailed market share data in terms of supply, demand, and revenue from trading processes and after-sales.

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The Hematopoietic Stem Cell Transplantation (HSCT) report rates the market according to different segments, including geographic areas [Peripheral Blood Stem Cells Transplant (PBSCT), Bone Marrow Transplant (BMT), Cord Blood Transplant (CBT)] and current market trends. The market report contains information about different companies, manufacturers and traders.

The market report comprises an analysis of the latest developments in the field of innovative technologies, detailed profiles of the industry's top competitors, and an excellent business model. The report also contains information on market expectations for the coming years. The Hematopoietic Stem Cell Transplantation (HSCT) report also provides a detailed summary of the macro and microelement estimations that are important to market participants and newly developed companies.

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The different characteristics and performance of Hematopoietic Stem Cell Transplantation (HSCT) are analyzed based on subjective and quantitative techniques to give a clear picture of current and future evaluation.

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Our board of exchange givers additionally as exchange experts over the value chain have taken immense endeavors in doing this gathering activity and hard work add request to deliver the key players with helpful essential and optional information concerning the world Hematopoietic Stem Cell Transplantation (HSCT) advertise. moreover, the report furthermore contains contributions from our exchange experts that may encourage the key players in sparing their time from the inside examination half. firms WHO get and utilize this report will be totally benefitted with the derivations conveyed in it. but this, the report furthermore gives top to bottom investigation on Hematopoietic Stem Cell Transplantation (HSCT) deal in addition on the grounds that the elements that impact the customers additionally as undertakings towards this technique.

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Hematopoietic Stem Cell Transplantation (HSCT) Market Expected to Deliver Dynamic Progression until 2028| Regen Biopharma Inc - The World Industry...

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Myelodysplastic syndrome (MDS) is a blood disorder caused due to the production of abnormal blood cells in bone marrow. Bone marrow failure leads to drop in the number of healthy blood cells in the body. In MDS, bone marrow does not produce healthy red blood cells, white blood cells, and/or platelets. Symptoms of MDS in the beginning are no specific than causes of pancytopenia i.e., deficiency of RBC, WBC and platelets. Therefore, the final diagnosis of MDS is done after examination of the cells of bone marrow.

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Bone marrow sample is taken from inside of a bone (usually the hip bone) and examined with a microscope using special stains to look for abnormal and immature cells. According to the American Cancer Society, over 13,000 new cases of MDS occur in the U.S. each year. According to the National Organization for Rare Disorders, 30% of patients with MDS can develop a form of blood cancer known as acute myeloid leukemia. Majority of patients diagnosed with MDS are aged between 65 and 70; however, MDS can affect people of any age and the risk of developing MDS increases with age.

The global myelodysplastic syndrome treatment market is driven by rise in the global geriatric population as MDS is most commonly found in this population and remains an incurable disease. Moreover, significant progress has been made in the diagnosis of MDS with the help of sequencing technologies. Chromosomal abnormalities can be identified in MDS patients with these technologies. These are useful for both diagnosis and prognosis in MDS patients. For instance, patients with chromosome 5q deletions are more likely to respond to lenalidomide. No new drugs have been approved for MDS by the U.S. Food and Drug Administration since 2006. Current available therapies can be efficacious, but are generally not curative. Some of the challenges in developing new treatments are the complexity and heterogeneity of MDS as a disease.

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The global myelodysplastic syndrome treatment market can be segmented based on type of treatment and region. In terms of type of treatment, the market can be classified into supportive therapy, growth factors, chemotherapy (including hypomethylating agents), and stem cell transplant. Cytarabine, azacitidine, decitabine, and lenalidomide are the major drugs used during chemotherapy.

Stem cell transplant is the only cure for MDS; however, majority of patients are not treated with stem cell transplant due to various factors such as high treatment cost, transplant-related deaths, and relapse rate at five years (as high as 40%). The chemotherapy segment is expected to hold major share of the global myelodysplastic syndrome treatment market due to larger application and less complications than other therapies such as stem cell transplant.

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Geographically, the global myelodysplastic syndrome treatment market can be segmented into North America, Europe, Asia Pacific, Latin America, Africa, and Middle East. North America is projected to dominate the global myelodysplastic syndrome market during the forecast period due to factors such as the rise in aging population and growing awareness about the disease among the population coupled with unmet medical needs in this region.

Key players operating in the global myelodysplastic syndrome treatment market are Celgene Corporation, Otsuka Holdings Co., Ltd., Sandoz, Inc., Dr. Reddys Laboratories, Inc., Accord Healthcare Ltd., Mylan N.V., and Pfizer, Inc.

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Myelodysplastic Syndrome Treatment Market Assessment Latest Insights on Trends and Challenges - Techi Labs

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SAN FRANCISCO, Dec. 17, 2019 /PRNewswire/ -- The genome of human cells looks a lot like a tangled ball of yarn, with tightly wound clumps from which myriad loose strands escape and loop out. But there is order to this tangleand growing evidence that the genome's 3D architecture influences the activity of its genes. Understanding the rules that control gene activity has been the object of a long collaboration between Gladstone investigators Deepak Srivastava, Benoit Bruneau, Katherine Pollard, Bruce Conklin, and Nevan Krogan, and their UC San Francisco (UCSF) partner Brian Black. Together, they have already found many key regulators of gene activity in the heart.

Now, their collaboration has received a strong shot in the arm from the National Institute of Health with the recent award of a Program Project Grant totaling $13 million between the labs for the next five years.

With this new support, the researchers will carry out a comprehensive probe into gene activity in heart cells and its intersection with the genome's 3D organization during heart formation.

"It is truly gratifying to see our long collaboration supported in this way by the National Institute of Health,"says Srivastava, president of Gladstone Institutes and project leader on this multi-investigator grant. "This funding will allow us to dig deep into processes that are fundamental to heart cell biology, but that will also directly inform our efforts to design therapies for congenital heart disease, heart failure, and other heart diseases."

Heart failure is the most common cause of death in adults, and congenital heart defects the most common form of birth defects. These defects have been traced to mutations in a number of proteins that regulate gene activity in heart cells, including the proteins at the core of the researchers' new proposal.

"However, the investigation of the 3D organization of the genome is a relatively new area, particularly in the heart," says Srivastava, who is also a pediatric cardiologist and has devoted much of his career to understanding heart formation and congenital heart defects.

The work outlined in this grant is therefore expected to yield novel insight into heart disease and spur the design of new therapies. It will also help the researchers improve their ability to coax human cells into becoming various types of heart cells. This technology could eventually be used to regenerate failing heart tissue.

Gladstone Senior InvestigatorBruce Conklinwill lend his expertise in cardiac stem cell biology and CRISPR gene-editing technology to the project.

The researchers' plan is to correlate gene activity and genome organization at the whole-genome scale and during multiple stages of heart formation. This will require enormous technological power. It will also require massive computing power and statistical analysis to store and sift through the large data sets the group will generate.

But the team is well-positioned to take on this challenge.

"Our studies are facilitated by extraordinary new technology,"says Bruneau, also a cardiovascular development specialist and the director of the Gladstone Institute of Cardiovascular Disease.

The $13 million proposal will leverage Srivastava, Bruneau, and Black's deep understanding of heart development and disease, and enlist the state-of-the-art technologies and analytic tools that Pollard and Krogan have developed to collect and analyze information about biological networks on a grand scale.

"Our team combines a remarkable array of expertise and technologies," says Srivastava, who is also director of the Roddenberry Stem Cell Center at Gladstone. "It would be impossible for any one or two labs in isolation to pursue the complex goals we set out to achieve with this project."

Dynamic Protein Networks

The project focuses on a small set of proteins the team has previously shown to be crucial for the formation of a functional heart. These proteins, known as transcription factors, activate or silence genes by binding to specific DNA sequences in the genes' vicinity.

The scientists have shown that cardiac transcription factors can associate with each other and with other proteins. "Depending on the associations they form, they turn genes on, off, or somewhere in between, and different types of heart cells may form," says Black.

But for a transcription factor to turn a gene on or off, it needs to access the gene's DNA sequence. That's not as easy as it sounds, as much of the genome is wound up in tight coils that give no foothold to transcription factors.

Bruneau's team studies proteins that modulate the accessibility of DNA sequences along the genome, a process known as chromatin remodeling. These proteins unspool segments of the genome from the tightly wound coils, opening up stretches of DNA that transcription factors can bind.

Like transcription factors, chromatin remodeling proteins associate with each other and with other proteins, forming associations that vary depending on the cell type or the stage of heart formation.

Interestingly, Srivastava's group recently discovered that cardiac transcription factors may have long-range effects on the 3D organization of the genome. The genome is housed in a separate compartment of the cell, a spherical structure called the nucleus. Srivastava's team found that cardiac transcription factors may pull genome loops all the way to proteins lining the edges of the nucleus.

The picture that emerges from these findings is that of a vast network of proteins that coordinate gene activity and genome architecture, and change as the heart forms.

Now the researchers want to know how these networks form, how many proteins they entail, and what genes they affect.

Dynamic Lab Partnerships

To answer these questions, the team will analyze the associations between cardiac transcription factors, chromatin remodeling proteins, and their various partners during heart development. They will pair this analysis with a genome-wide survey of the genes these proteins target and of these genes' activity.

"Our overarching goal is to understand all the levels of gene regulation in developing hearts, from genes and transcription factors to chromatin remodeling and to genome organization within the nucleus," says Bruneau, who is also a professor of pediatrics at UCSF.

The researchers will use a battery of sophisticated techniques to capture the complexes that proteins form with each other or with DNA sequences and to record which genes are active or inactive in different types of heart cells.

They will leverage various models of heart development, including human induced pluripotent stem cells (hiPS cells) that can give rise to heart tissue in the dish, or cells from the developing heart of mouse embryos. They will also use CRISPR technology and other genetic tools to insert mutations in heart cells and evaluate the impact of these mutations on the protein-genome networks.

Their success will depend on high-throughput data collection and analysis, and powerful statistics to guarantee the validity of the findings. That's where Krogan and Pollard come in.

Krogan's labwill contribute technology his lab developed to determine how proteins interact with one another in the celland how those interactions affect the interaction of proteins with DNA.

Pollard's groupwill devise statistical methods to rigorously analyze the protein networks and gene activity profiles the researchers uncover through the lens of genetic causes of heart disease.

"The biggest challenge will be to develop novel computational methods, including artificial intelligence tools," says Pollard, who directs the Gladstone Institute for Data Science and Biotechnology. "This is the first time that scientists will integrate such diverse kinds of data to understand a disease."

Together, these tools will allow the researchers to reliably identify connections between protein networks and gene activity at all stages of heart formation, in the context of disease or healthy heart formation.

"This project crystallizes a more than a decade-long collaboration across our labs, with a laser focus on fundamental concepts of gene regulation," says Bruneau.

"We will learn how these concepts apply to the heart and to heart diseases," he adds, "but we think they will also be relevant to other organs and sets of diseases."

Media Contact:Megan McDevittmegan.mcdevitt@Gladstone.ucsf.edu

Related Images

team-of-researchers-who-received.jpg Team of Researchers who Received the Grant New funding from the NIH fuels collaboration between UCSF's Brian Black and Gladstone's Deepak Srivastava, Benoit Bruneau (front row, left to right), Katie Pollard, Bruce Conklin (back row, left to right), and Nevan Krogan (not shown).

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$13 Million Grant to Probe the Genome of Heart Cells - PRNewswire

Recommendation and review posted by Bethany Smith

Company closes $5.6 million in funding and secures distinguished board of directors as it seeks to empower individual health ownership with personalized biological analysis and storage

GoodCell ("LifeVault Bio"), the personal biobanking company with the health indicators to inform actionable next steps in your health journey, today announced it has secured a $2.6 million price round under LifeVault Bio and a $3 million convertible note, and brought on renowned technology executive Anthony Bay as its newest board member. The capital will be used to expand GoodCell Diagnostics, the companys commercial application, as it pioneers a cell quality test to measure the DNA damage to somatic cells over time, as well as fuel the formation of strategic partnerships across the healthcare and life sciences sectors, and grow the team at its headquarters in Waltham, Mass.

GoodCell helps individuals take control of their health through personalized biobanking of cells, DNA and blood plasma, with the belief that medical science will continue to progress, bringing forth new ways of preventing, detecting and treating diseases. Research continues to prove that cells are an essential starting material for the treatments of tomorrow. DNA and plasma are widely validated as critical information sources for monitoring and tracking health risk and informing lifestyle decisions. GoodCell aims to empower individuals with personal health information and storage resources to take full advantage of breakthrough medical science as it emerges.

"Stem cells are among the most promising areas of medical research because they are the starting materials from which all other cells originate," said Brad Hamilton, co-founder and chief science officer at GoodCell. "Some of these cells, specifically induced pluripotent stem (iPS) cells which can be derived from a persons own skin or blood, can be programmed to produce virtually any type of cell in the human body. This versatility has made them an instrumental tool, helping scientists understand and fight some of the biggest health threats of our time, such as Parkinsons disease, Type 1 diabetes and heart disease. GoodCell exists to help people preserve their access to these potentially lifesaving cells."

After GoodCell sends members a sample collection kit to their doorstep, they are prompted to schedule a convenient blood-draw with a certified phlebotomist, who then safely packages and ships the sample for processing. Once received, GoodCell isolates and preserves three components of the blood sample: cells, DNA and blood plasma. The DNA sample is then tested to inform genetic predisposition to disease, such as metabolic, neurologic and cardiac disorders, as well as certain cancers. Armed with deep insight into a members biology, the GoodCell Dashboard displays their health information as a comprehensive overview, designed to inform the next best action in their health journey. Samples are stored in a state-of-the-art, FDA-registered CLIA/CAP certified lab and biorepository that is trusted by larger biotechnology companies and the National Institutes of Health. Since it is the change in health indicators that indicates risk, recurrent sampling is possible to enable measuring the trajectory of change in plasma components or DNA. Since the samples belong to GoodCell members, they can decide whether or not to share their information with their doctor or allow researchers to use it in clinical studies.

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"To me, GoodCell represents the ultimate in personalized medicine. Individuals can now have their own biobank and their own biodata. These wont be owned by a hospital or in the case of your cells, by no one at all. These will be stored for you, accessible only on your instruction. As new tests come online or as cells become a broader therapy source, you will be able to tap into your own earlier, preserved self in the form of your blood," said David Scadden, MD, co-founder and chair of the Scientific Advisory Board at GoodCell. "Imagine two scenarios. First, a new blood test becomes available for Alzheimers disease. You get the test, but just like current tests for things like prostate cancer, it is only meaningful in light of how it is changing. Your doctor will likely advise waiting months or a year to re-test. With a GoodCell sample, we envision the test can be done on your blood from a previous time. Then you can know how things are changing without the prolonged wait and the anxiety it engenders. Second, lets say the stem cell field delivers on the therapies it is currently testing for diabetes, heart failure, Parkinsons disease and macular degeneration. Those therapies will likely be as cells derived from you. Would you want those to be from you at a younger age since we know our cells accumulate genetic damage with age? I think most people would, and would want cells from their blood, which the bones have shielded from radiation, rather than their skin as is currently done. GoodCell will have those blood cells for you and has shown they can be made into stem cells (iPSC) with high efficiency."

GoodCell is focused on continuing to grow its customer base and building up its talent pool at its new headquarters in Waltham, Mass. The company, which is poised to expand its headcount in early 2020, will also be exploring strategic partnerships with cell and gene therapy companies and interest groups that could benefit from GoodCell members deciding whether to opt-in to allow access to stored cells, DNA and plasma. GoodCell will also continue to recruit pioneers in business, science and technology to its board positions. Most recently, it welcomed Anthony Bay, former Global Head of Digital Video for Amazon and a veteran senior executive at other technology powerhouses, including Apple and Microsoft.

"Ive devoted my career to creating scalable and differentiated technology platforms and unique digital experiences in many industries, and am excited to lend my expertise and perspectives to GoodCell," said Bay. "I am delighted to play a role in helping the GoodCell team scale and expand to match the size of our opportunity to change peoples lives."

Bay joins an already robust and diverse group of consumer technology and life science leaders, including John Goscha, Lucidity Lights founder and Chairman of the Board of Directors, Finally Light Bulb Company founder and entrepreneur; David Scadden, MD, professor of medicine at Harvard Universitys Department of Stem Cell and Regenerative Biology; Daniel Marshak, principal consultant in therapeutics, diagnostics and medical devices; Avi Ellman, managing partner of Delta Global Investment Services; and Trevor Perry, co-founder and chief executive officer at GoodCell.

"Up until now, existing genetics offerings can only go so far as to inform your genetic makeup. GoodCell is taking that a step further today by combining genetics, health indicator testing and personal biobanking into one solution, and then turning this information right back to the individual so they can understand the story of their health and leverage actionable data at any age," said Perry. "We are taking advantage of leading scientific innovation to help people take control of their health through personalized biobanking of cells, DNA, and blood plasma, and we believe the tremendous amount of support we received during this initial funding round will further allow us to be a true enabler of and partner in this process. Our goal is to set a new standard for personal biobanking as an individual health milestone, and our mission is to ensure our members feel confident and prepared to own their aging experience, and we look forward to accelerating our efforts in the months ahead."

For more information about GoodCell, visit https://www.goodcell.com. To order your starter kit, visit https://www.goodcell.com/shop/.

About GoodCell

GoodCell helps you take control of your health through personalized biobanking of cells, DNA and blood plasma. Leveraging the best science, the technology provides health indicators for a comprehensive and proactive approach to self-care. Through the GoodCell Dashboard, the company informs the next best action in your health journey, offering access for you and for your doctor to actionable data and insights that relate to all aspects of your health through genetic reporting and blood analysis. Driven by mounting evidence in support of cellular therapy and united in the belief that you should be empowered to take control of your health, GoodCell is led by a founding team of scientific advisors with a diverse set of medical research and clinical expertise. By backing up your starting materials, GoodCell is setting a new standard of personal biobanking today for a healthier future. Learn more at: https://www.goodcell.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191217005485/en/

Contacts

PAN CommunicationsStaci Didner407 734 7325Goodcell@pancomm.com

Read more:
GoodCell Oversubscribes Upon Debut, Fueling Expansion of Health Tracking and Personal Biobanking Services; Adds Former Amazon and Microsoft Executive...

Recommendation and review posted by Bethany Smith

The ODAC discussions were based on the supplemental Biologics License Application (sBLA), currently under priority review at the FDA, seeking approval of KEYTRUDA monotherapy for the treatment of patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, NMIBC with carcinoma in-situ (CIS) with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy (removal of bladder). This application is based on results from the Phase 2 KEYNOTE-057 trial.

The positive vote from todays ODAC meeting supports the potential for KEYTRUDA in certain patients with high-risk, non-muscle invasive bladder cancer, who currently have limited non-surgical treatment options approved by the FDA, said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories. We are encouraged by todays productive discussion and look forward to working with the FDA as they continue their review of our supplemental application for KEYTRUDA in this patient population.

The ODAC provides the FDA with independent, expert advice and recommendations on marketed and investigational medicines for use in the treatment of cancer. The FDA is not bound by the committees guidance but takes its advice into consideration. Merck anticipates a Prescription Drug User Fee Act (PDUFA), or target action date, in January 2020, based on priority review.

About Bladder Cancer

Bladder cancer begins when cells in the urinary bladder start to grow uncontrollably. As more cancer cells develop, they can form a tumor and spread to other areas of the body. Bladder cancers are described based on how far they have invaded into the wall of the bladder. NMIBC occurs when the cancer has not grown into the main muscle layer of the bladder. It is estimated that more than 80,000 new cases of bladder cancer will be diagnosed in 2019 in the United States. Approximately 75% of patients with bladder cancer are diagnosed with non-muscle invasive bladder cancer (NMIBC). For high-risk NMIBC patients who are BCG-unresponsive with persistent or recurrent disease, treatment guidelines recommend radical cystectomy, a surgery to remove the entire bladder that often requires removal of other surrounding organs and tissues. In men, removal of the prostate is common, and in women, surgeons may also remove the uterus, fallopian tubes, ovaries and cervix, and occasionally a portion of the vagina.

About KEYNOTE-057

The filing was based on data from KEYNOTE-057 (NCT02625961), a Phase 2, multicenter, open-label, single-arm trial in 102 patients with Bacillus Calmette-Guerin (BCG)-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in-situ (CIS) with or without papillary tumors who were ineligible for or had elected not to undergo cystectomy (Cohort A). In this study, BCG-unresponsive high-risk NMIBC is defined as persistent disease despite adequate BCG therapy, disease recurrence after an initial tumor-free state following adequate BCG therapy, or T1 disease following a single induction course of BCG. Patients received KEYTRUDA 200 mg every three weeks until unacceptable toxicity, persistent or recurrent high-risk NMIBC or progressive disease. Assessment of tumor status was performed every 12 weeks, and patients without disease progression could be treated for up to 24 months. The major efficacy outcome measures were complete response (as defined by negative results for cystoscopy [with transurethral resection of bladder tumor (TURBT)/biopsies as applicable], urine cytology, and computed tomography urography [CTU] imaging) and duration of response.

About KEYTRUDA (pembrolizumab) Injection, 100mg

KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the bodys immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck has the industrys largest immuno-oncology clinical research program. There are currently more than 1,000 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patients likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

Selected KEYTRUDA (pembrolizumab) Indications

Melanoma

KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.

KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.

Non-Small Cell Lung Cancer

KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.

KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) 1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is stage III where patients are not candidates for surgical resection or definitive chemoradiation, or metastatic.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS 1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.

Small Cell Lung Cancer

KEYTRUDA is indicated for the treatment of patients with metastatic small cell lung cancer (SCLC) with disease progression on or after platinum-based chemotherapy and at least one other prior line of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

Head and Neck Squamous Cell Cancer

KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).

KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) 1] as determined by an FDA-approved test.

KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) with disease progression on or after platinum-containing chemotherapy.

Classical Hodgkin Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory classical Hodgkin lymphoma (cHL), or who have relapsed after 3 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Primary Mediastinal Large B-Cell Lymphoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.

Urothelial Carcinoma

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 [combined positive score (CPS) 10] as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.

KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

Microsatellite Instability-High (MSI-H) Cancer

KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR)

This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.

Gastric Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test, with disease progression on or after two or more prior lines of therapy including fluoropyrimidine- and platinum-containing chemotherapy and if appropriate, HER2/neu-targeted therapy. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Esophageal Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent locally advanced or metastatic squamous cell carcinoma of the esophagus whose tumors express PD-L1 (CPS 10) as determined by an FDA-approved test, with disease progression after one or more prior lines of systemic therapy.

Cervical Cancer

KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS 1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Hepatocellular Carcinoma

KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Merkel Cell Carcinoma

KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

Renal Cell Carcinoma

KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).

Selected Important Safety Information for KEYTRUDA

Immune-Mediated Pneumonitis

KEYTRUDA can cause immune-mediated pneumonitis, including fatal cases. Pneumonitis occurred in 3.4% (94/2799) of patients with various cancers receiving KEYTRUDA, including Grade 1 (0.8%), 2 (1.3%), 3 (0.9%), 4 (0.3%), and 5 (0.1%). Pneumonitis occurred in 8.2% (65/790) of NSCLC patients receiving KEYTRUDA as a single agent, including Grades 3-4 in 3.2% of patients, and occurred more frequently in patients with a history of prior thoracic radiation (17%) compared to those without (7.7%). Pneumonitis occurred in 6% (18/300) of HNSCC patients receiving KEYTRUDA as a single agent, including Grades 3-5 in 1.6% of patients, and occurred in 5.4% (15/276) of patients receiving KEYTRUDA in combination with platinum and FU as first-line therapy for advanced disease, including Grades 3-5 in 1.5% of patients.

Monitor patients for signs and symptoms of pneumonitis. Evaluate suspected pneumonitis with radiographic imaging. Administer corticosteroids for Grade 2 or greater pneumonitis. Withhold KEYTRUDA for Grade 2; permanently discontinue KEYTRUDA for Grade 3 or 4 or recurrent Grade 2 pneumonitis.

Immune-Mediated Colitis

KEYTRUDA can cause immune-mediated colitis. Colitis occurred in 1.7% (48/2799) of patients receiving KEYTRUDA, including Grade 2 (0.4%), 3 (1.1%), and 4 (<0.1%). Monitor patients for signs and symptoms of colitis. Administer corticosteroids for Grade 2 or greater colitis. Withhold KEYTRUDA for Grade 2 or 3; permanently discontinue KEYTRUDA for Grade 4 colitis.

Immune-Mediated Hepatitis (KEYTRUDA) and Hepatotoxicity (KEYTRUDA in Combination With Axitinib)

Immune-Mediated Hepatitis

KEYTRUDA can cause immune-mediated hepatitis. Hepatitis occurred in 0.7% (19/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.4%), and 4 (<0.1%). Monitor patients for changes in liver function. Administer corticosteroids for Grade 2 or greater hepatitis and, based on severity of liver enzyme elevations, withhold or discontinue KEYTRUDA.

Hepatotoxicity in Combination With Axitinib

KEYTRUDA in combination with axitinib can cause hepatic toxicity with higher than expected frequencies of Grades 3 and 4 ALT and AST elevations compared to KEYTRUDA alone. With the combination of KEYTRUDA and axitinib, Grades 3 and 4 increased ALT (20%) and increased AST (13%) were seen. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider more frequent monitoring of liver enzymes as compared to when the drugs are administered as single agents. For elevated liver enzymes, interrupt KEYTRUDA and axitinib, and consider administering corticosteroids as needed.

Immune-Mediated Endocrinopathies

KEYTRUDA can cause hypophysitis, thyroid disorders, and type 1 diabetes mellitus. Hypophysitis occurred in 0.6% (17/2799) of patients, including Grade 2 (0.2%), 3 (0.3%), and 4 (<0.1%). Hypothyroidism occurred in 8.5% (237/2799) of patients, including Grade 2 (6.2%) and 3 (0.1%). The incidence of new or worsening hypothyroidism was higher in 1185 patients with HNSCC (16%) receiving KEYTRUDA, as a single agent or in combination with platinum and FU, including Grade 3 (0.3%) hypothyroidism. Hyperthyroidism occurred in 3.4% (96/2799) of patients, including Grade 2 (0.8%) and 3 (0.1%), and thyroiditis occurred in 0.6% (16/2799) of patients, including Grade 2 (0.3%). Type 1 diabetes mellitus, including diabetic ketoacidosis, occurred in 0.2% (6/2799) of patients.

Monitor patients for signs and symptoms of hypophysitis (including hypopituitarism and adrenal insufficiency), thyroid function (prior to and periodically during treatment), and hyperglycemia. For hypophysitis, administer corticosteroids and hormone replacement as clinically indicated. Withhold KEYTRUDA for Grade 2 and withhold or discontinue for Grade 3 or 4 hypophysitis. Administer hormone replacement for hypothyroidism and manage hyperthyroidism with thionamides and beta-blockers as appropriate. Withhold or discontinue KEYTRUDA for Grade 3 or 4 hyperthyroidism. Administer insulin for type 1 diabetes, and withhold KEYTRUDA and administer antihyperglycemics in patients with severe hyperglycemia.

Immune-Mediated Nephritis and Renal Dysfunction

KEYTRUDA can cause immune-mediated nephritis. Nephritis occurred in 0.3% (9/2799) of patients receiving KEYTRUDA, including Grade 2 (0.1%), 3 (0.1%), and 4 (<0.1%) nephritis. Nephritis occurred in 1.7% (7/405) of patients receiving KEYTRUDA in combination with pemetrexed and platinum chemotherapy. Monitor patients for changes in renal function. Administer corticosteroids for Grade 2 or greater nephritis. Withhold KEYTRUDA for Grade 2; permanently discontinue for Grade 3 or 4 nephritis.

Immune-Mediated Skin Reactions

Immune-mediated rashes, including Stevens-Johnson syndrome (SJS), toxic epidermal necrolysis (TEN) (some cases with fatal outcome), exfoliative dermatitis, and bullous pemphigoid, can occur. Monitor patients for suspected severe skin reactions and based on the severity of the adverse reaction, withhold or permanently discontinue KEYTRUDA and administer corticosteroids. For signs or symptoms of SJS or TEN, withhold KEYTRUDA and refer the patient for specialized care for assessment and treatment. If SJS or TEN is confirmed, permanently discontinue KEYTRUDA.

Other Immune-Mediated Adverse Reactions

Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue in patients receiving KEYTRUDA and may also occur after discontinuation of treatment. For suspected immune-mediated adverse reactions, ensure adequate evaluation to confirm etiology or exclude other causes. Based on the severity of the adverse reaction, withhold KEYTRUDA and administer corticosteroids. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Based on limited data from clinical studies in patients whose immune-related adverse reactions could not be controlled with corticosteroid use, administration of other systemic immunosuppressants can be considered. Resume KEYTRUDA when the adverse reaction remains at Grade 1 or less following corticosteroid taper. Permanently discontinue KEYTRUDA for any Grade 3 immune-mediated adverse reaction that recurs and for any life-threatening immune-mediated adverse reaction.

The following clinically significant immune-mediated adverse reactions occurred in less than 1% (unless otherwise indicated) of 2799 patients: arthritis (1.5%), uveitis, myositis, Guillain-Barr syndrome, myasthenia gravis, vasculitis, pancreatitis, hemolytic anemia, sarcoidosis, and encephalitis. In addition, myelitis and myocarditis were reported in other clinical trials, including classical Hodgkin lymphoma, and postmarketing use.

Treatment with KEYTRUDA may increase the risk of rejection in solid organ transplant recipients. Consider the benefit of treatment vs the risk of possible organ rejection in these patients.

Infusion-Related Reactions

KEYTRUDA can cause severe or life-threatening infusion-related reactions, including hypersensitivity and anaphylaxis, which have been reported in 0.2% (6/2799) of patients. Monitor patients for signs and symptoms of infusion-related reactions. For Grade 3 or 4 reactions, stop infusion and permanently discontinue KEYTRUDA.

Complications of Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)

Immune-mediated complications, including fatal events, occurred in patients who underwent allogeneic HSCT after treatment with KEYTRUDA. Of 23 patients with cHL who proceeded to allogeneic HSCT after KEYTRUDA, 6 (26%) developed graft-versus-host disease (GVHD) (1 fatal case) and 2 (9%) developed severe hepatic veno-occlusive disease (VOD) after reduced-intensity conditioning (1 fatal case). Cases of fatal hyperacute GVHD after allogeneic HSCT have also been reported in patients with lymphoma who received a PD-1 receptorblocking antibody before transplantation. Follow patients closely for early evidence of transplant-related complications such as hyperacute graft-versus-host disease (GVHD), Grade 3 to 4 acute GVHD, steroid-requiring febrile syndrome, hepatic veno-occlusive disease (VOD), and other immune-mediated adverse reactions.

In patients with a history of allogeneic HSCT, acute GVHD (including fatal GVHD) has been reported after treatment with KEYTRUDA. Patients who experienced GVHD after their transplant procedure may be at increased risk for GVHD after KEYTRUDA. Consider the benefit of KEYTRUDA vs the risk of GVHD in these patients.

Increased Mortality in Patients With Multiple Myeloma

In trials in patients with multiple myeloma, the addition of KEYTRUDA to a thalidomide analogue plus dexamethasone resulted in increased mortality. Treatment of these patients with a PD-1 or PD-L1 blocking antibody in this combination is not recommended outside of controlled trials.

Embryofetal Toxicity

Based on its mechanism of action, KEYTRUDA can cause fetal harm when administered to a pregnant woman. Advise women of this potential risk. In females of reproductive potential, verify pregnancy status prior to initiating KEYTRUDA and advise them to use effective contraception during treatment and for 4 months after the last dose.

Adverse Reactions

In KEYNOTE-006, KEYTRUDA was discontinued due to adverse reactions in 9% of 555 patients with advanced melanoma; adverse reactions leading to permanent discontinuation in more than one patient were colitis (1.4%), autoimmune hepatitis (0.7%), allergic reaction (0.4%), polyneuropathy (0.4%), and cardiac failure (0.4%). The most common adverse reactions (20%) with KEYTRUDA were fatigue (28%), diarrhea (26%), rash (24%), and nausea (21%).

In KEYNOTE-002, KEYTRUDA was permanently discontinued due to adverse reactions in 12% of 357 patients with advanced melanoma; the most common (1%) were general physical health deterioration (1%), asthenia (1%), dyspnea (1%), pneumonitis (1%), and generalized edema (1%). The most common adverse reactions were fatigue (43%), pruritus (28%), rash (24%), constipation (22%), nausea (22%), diarrhea (20%), and decreased appetite (20%).

In KEYNOTE-054, KEYTRUDA was permanently discontinued due to adverse reactions in 14% of 509 patients; the most common (1%) were pneumonitis (1.4%), colitis (1.2%), and diarrhea (1%). Serious adverse reactions occurred in 25% of patients receiving KEYTRUDA. The most common adverse reaction (20%) with KEYTRUDA was diarrhea (28%).

In KEYNOTE-189, when KEYTRUDA was administered with pemetrexed and platinum chemotherapy in metastatic nonsquamous NSCLC, KEYTRUDA was discontinued due to adverse reactions in 20% of 405 patients. The most common adverse reactions resulting in permanent discontinuation of KEYTRUDA were pneumonitis (3%) and acute kidney injury (2%). The most common adverse reactions (20%) with KEYTRUDA were nausea (56%), fatigue (56%), constipation (35%), diarrhea (31%), decreased appetite (28%), rash (25%), vomiting (24%), cough (21%), dyspnea (21%), and pyrexia (20%).

In KEYNOTE-407, when KEYTRUDA was administered with carboplatin and either paclitaxel or paclitaxel protein-bound in metastatic squamous NSCLC, KEYTRUDA was discontinued due to adverse reactions in 15% of 101 patients. The most frequent serious adverse reactions reported in at least 2% of patients were febrile neutropenia, pneumonia, and urinary tract infection. Adverse reactions observed in KEYNOTE-407 were similar to those observed in KEYNOTE-189 with the exception that increased incidences of alopecia (47% vs 36%) and peripheral neuropathy (31% vs 25%) were observed in the KEYTRUDA and chemotherapy arm compared to the placebo and chemotherapy arm in KEYNOTE-407.

In KEYNOTE-042, KEYTRUDA was discontinued due to adverse reactions in 19% of 636 patients; the most common were pneumonitis (3%), death due to unknown cause (1.6%), and pneumonia (1.4%). The most frequent serious adverse reactions reported in at least 2% of patients were pneumonia (7%), pneumonitis (3.9%), pulmonary embolism (2.4%), and pleural effusion (2.2%). The most common adverse reaction (20%) was fatigue (25%).

In KEYNOTE-010, KEYTRUDA monotherapy was discontinued due to adverse reactions in 8% of 682 patients with metastatic NSCLC; the most common was pneumonitis (1.8%). The most common adverse reactions (20%) were decreased appetite (25%), fatigue (25%), dyspnea (23%), and nausea (20%).

Adverse reactions occurring in patients with SCLC were similar to those occurring in patients with other solid tumors who received KEYTRUDA as a single agent.

In KEYNOTE-048, KEYTRUDA monotherapy was discontinued due to adverse events in 12% of 300 patients with HNSCC; the most common adverse reactions leading to permanent discontinuation were sepsis (1.7%) and pneumonia (1.3%). The most common adverse reactions (20%) were fatigue (33%), constipation (20%), and rash (20%).

In KEYNOTE-048, when KEYTRUDA was administered in combination with platinum (cisplatin or carboplatin) and FU chemotherapy, KEYTRUDA was discontinued due to adverse reactions in 16% of 276 patients with HNSCC. The most common adverse reactions resulting in permanent discontinuation of KEYTRUDA were pneumonia (2.5%), pneumonitis (1.8%), and septic shock (1.4%). The most common adverse reactions (20%) were nausea (51%), fatigue (49%), constipation (37%), vomiting (32%), mucosal inflammation (31%), diarrhea (29%), decreased appetite (29%), stomatitis (26%), and cough (22%).

In KEYNOTE-012, KEYTRUDA was discontinued due to adverse reactions in 17% of 192 patients with HNSCC. Serious adverse reactions occurred in 45% of patients. The most frequent serious adverse reactions reported in at least 2% of patients were pneumonia, dyspnea, confusional state, vomiting, pleural effusion, and respiratory failure. The most common adverse reactions (20%) were fatigue, decreased appetite, and dyspnea. Adverse reactions occurring in patients with HNSCC were generally similar to those occurring in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy, with the exception of increased incidences of facial edema and new or worsening hypothyroidism.

In KEYNOTE-087, KEYTRUDA was discontinued due to adverse reactions in 5% of 210 patients with cHL. Serious adverse reactions occurred in 16% of patients; those 1% included pneumonia, pneumonitis, pyrexia, dyspnea, GVHD, and herpes zoster. Two patients died from causes other than disease progression; 1 from GVHD after subsequent allogeneic HSCT and 1 from septic shock. The most common adverse reactions (20%) were fatigue (26%), pyrexia (24%), cough (24%), musculoskeletal pain (21%), diarrhea (20%), and rash (20%).

In KEYNOTE-170, KEYTRUDA was discontinued due to adverse reactions in 8% of 53 patients with PMBCL. Serious adverse reactions occurred in 26% of patients and included arrhythmia (4%), cardiac tamponade (2%), myocardial infarction (2%), pericardial effusion (2%), and pericarditis (2%). Six (11%) patients died within 30 days of start of treatment. The most common adverse reactions (20%) were musculoskeletal pain (30%), upper respiratory tract infection and pyrexia (28% each), cough (26%), fatigue (23%), and dyspnea (21%).

In KEYNOTE-052, KEYTRUDA was discontinued due to adverse reactions in 11% of 370 patients with locally advanced or metastatic urothelial carcinoma. Serious adverse reactions occurred in 42% of patients; those 2% were urinary tract infection, hematuria, acute kidney injury, pneumonia, and urosepsis. The most common adverse reactions (20%) were fatigue (38%), musculoskeletal pain (24%), decreased appetite (22%), constipation (21%), rash (21%), and diarrhea (20%).

In KEYNOTE-045, KEYTRUDA was discontinued due to adverse reactions in 8% of 266 patients with locally advanced or metastatic urothelial carcinoma. The most common adverse reaction resulting in permanent discontinuation of KEYTRUDA was pneumonitis (1.9%). Serious adverse reactions occurred in 39% of KEYTRUDA-treated patients; those 2% were urinary tract infection, pneumonia, anemia, and pneumonitis. The most common adverse reactions (20%) in patients who received KEYTRUDA were fatigue (38%), musculoskeletal pain (32%), pruritus (23%), decreased appetite (21%), nausea (21%), and rash (20%).

Adverse reactions occurring in patients with gastric cancer were similar to those occurring in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy.

Adverse reactions occurring in patients with esophageal cancer were similar to those occurring in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy.

In KEYNOTE-158, KEYTRUDA was discontinued due to adverse reactions in 8% of 98 patients with recurrent or metastatic cervical cancer. Serious adverse reactions occurred in 39% of patients receiving KEYTRUDA; the most frequent included anemia (7%), fistula, hemorrhage, and infections [except urinary tract infections] (4.1% each). The most common adverse reactions (20%) were fatigue (43%), musculoskeletal pain (27%), diarrhea (23%), pain and abdominal pain (22% each), and decreased appetite (21%).

Adverse reactions occurring in patients with hepatocellular carcinoma (HCC) were generally similar to those in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy, with the exception of increased incidences of ascites (8% Grades 34) and immune-mediated hepatitis (2.9%). Laboratory abnormalities (Grades 34) that occurred at a higher incidence were elevated AST (20%), ALT (9%), and hyperbilirubinemia (10%).

Among the 50 patients with MCC enrolled in study KEYNOTE-017, adverse reactions occurring in patients with MCC were generally similar to those occurring in patients with melanoma or NSCLC who received KEYTRUDA as a monotherapy. Laboratory abnormalities (Grades 34) that occurred at a higher incidence were elevated AST (11%) and hyperglycemia (19%).

In KEYNOTE-426, when KEYTRUDA was administered in combination with axitinib, fatal adverse reactions occurred in 3.3% of 429 patients. Serious adverse reactions occurred in 40% of patients, the most frequent (1%) were hepatotoxicity (7%), diarrhea (4.2%), acute kidney injury (2.3%), dehydration (1%), and pneumonitis (1%). Permanent discontinuation due to an adverse reaction occurred in 31% of patients; KEYTRUDA only (13%), axitinib only (13%), and the combination (8%); the most common were hepatotoxicity (13%), diarrhea/colitis (1.9%), acute kidney injury (1.6%), and cerebrovascular accident (1.2%). The most common adverse reactions (20%) were diarrhea (56%), fatigue/asthenia (52%), hypertension (48%), hepatotoxicity (39%), hypothyroidism (35%), decreased appetite (30%), palmar-plantar erythrodysesthesia (28%), nausea (28%), stomatitis/mucosal inflammation (27%), dysphonia (25%), rash (25%), cough (21%), and constipation (21%).

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FDA Oncologic Drugs Advisory Committee (ODAC) Recommends KEYTRUDA (pembrolizumab) for the Treatment of Certain Patients with High-Risk, Non-Muscle...

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The global cell separation technology market is highly competitive in terms of number of players. Key players operating in the global cell separation technology market includeAkadeum Life Sciences, STEMCELL Technologies, Inc., BD, Bio-Rad Laboratories, Inc., Miltenyi Biotech, 10X Genomics, Thermo Fisher Scientific, Inc., Zeiss, GE Healthcare Life Sciences, PerkinElmer, Inc., and QIAGEN.

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Cell Separation Technology Market is Estimated to Record Highest CAGR by 2027 - Techi Labs

Recommendation and review posted by Bethany Smith