The potential of gene therapies as cures for some hard-to-treat genetic diseases can be very attractive. But one such product is now suspected of causing a serious safety problem.

Friday, Orchard Therapeutics said its Strimvelis treatment, approved by European authorities in 2016 to treatthe rare inherited condition ADA-SCID, has been linked to a patients leukemia.

Preliminary findings suggest this diagnosis may be attributable to an insertional event related to treatment with Strimvelis, the company said. Its now investigating whether theres indeed a causal relationship.

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Since its 2016 EU approvalwhen it was owned by original developer GlaxoSmithKlineonly 16 patients have been treated with Stimvelis. The patient who developed leukemia had apparently been treated under a GSK compassionate use program in 2016.

No more patients will get the therapy before the investigation is complete, Orchard said. The drug was never approved in the U.S.

ADA-SCIDis a condition marked by a mutation in the gene making the adenosine deaminase (ADA) enzyme, which is essential for maintaining normal white blood cells. ADA-SCID patients, with a dysfunctional immune system, have less than two years to live without effective intervention.

RELATED:Orchard licenses gene therapy tech from GSK

Strimvelis, originally developed by GSK and bought by Orchard in 2018, offers an option for patients who cant find a matched stem cell donor. It works by editing the patients own hematopoietic stem cells with the functional ADA gene. The cells arethen transferred back into the patient's bone marrow to mature and produce the normal ADA protein.

The therapy uses a gammaretrovirus as the vector to carry the gene. Problem is, retrovirus can incorporate their own genetic information into the human genome, causing unintended changes that can give rise to cancer. It is a known risk factor of gammaretroviral vector-based gene therapy and has been described as one of the important potential risks for Strimvelis in its EU approval.

Besides Strimvelis, Orchard is also developing OTL-101, which uses a lentivirus to insert a functional copy of the ADA gene into a patients cells. The drug is currently undergoing a registrational trial and has won breakthrough and orphan drug designations from the FDA.

All the gene therapy candidates in Orchards pipeline use lentiviral vectors that have been specifically designed to avoid insertional oncogenesis after administration, Orchard said, adding that no dangerous gene insertion has been reported around lentiviral vector-based stem cell gene therapy in any indication.

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Orchard Therapeutics' gene therapy Strimvelis linked to a leukemia case - FiercePharma

Recommendation and review posted by Bethany Smith

U.S. biotechnology company races to get its COVID-19 therapeutic approved for access, which has previously shownpositive results inseverely ill patients

Vancouver, WACytoDyn Inc. (CytoDyn) a late-stage biotechnology companydeveloping leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announcedit is currently accepting only 155 more candidates across the country for its Phase 2b/3 registrational trial in patients with severe-to-critical COVID-19.

CytoDyn is currently enrolling COVID-19 patients in its Phase 2b/3 clinical trial in at least 13 hospitals in nine states (California, Georgia, Massachusetts, New Jersey, New York, North Carolina, Ohio, Oregon and Texas). Please visit the Companys website atwww.cytodyn.com. The sites currently enrolling patients are listed on the home page under Clinical Trial Enrollment, then click on COVID-19 Severe or Critical in the drop-down screen. Please visit the website frequently, as the list of hospitals is updated on a regular basis.

CytoDyn has already enrolled 235 patients in its trial, and the Data Safety Monitoring Committee (DSMC) recently recommended CytoDyn continue its study without modification to achieve its primary endpoint, based on the results the DSMC observed so far.

The DSMC also added that it will look at the unblinded data again, once an additional 58 patients are enrolled.

We are very encouraged the DSMC recommended we continue our trial without modification, said Nader Pourhassan, Ph.D., President and CEO of CytoDyn.We believe this result, combined with the promising data already demonstrated with emergency INDs in over 60 severe and critical COVID-19 patients, is an indicator of positive data.

Its our hope this means our study is not only proving to be safe, but effective. If it wasnt, the DSMC would have stopped our trial or requested modifications.But instead, the DSMC recommended we continue our study without modification, and indicated it would look at the unblinded data once we are 75% enrolled.This, to us, is a very strong signal of positive data, added Pourhassan.

The recovery of a young woman in California provides some hopeful evidence of the effectiveness of leronlimab, Pourhassan said. She had contracted COVID-19 and was in the hospital on a ventilator. Within 24 hours after receiving a single injection of leronlimab, the amount of oxygen she needed started to drop, and 2 days later, she was able to be removed from her ventilator and later, sent home. She believes our drug saved her life.

We are optimistic and look forward to the completion of our study, or possible early evaluation to warrant an Emergency Use Authorization (EUA) from the FDA, Pourhassan concluded.

About Coronavirus Disease 2019

CytoDyn completed its Phase 2 clinical trial (CD10) for COVID-19, a double-blinded, randomized clinical trial for mild-to-moderate patients in the U.S. which produced statistically significant results for NEWS2. Enrollment continues in its Phase 2b/3 randomized clinical trial for the severe-to-critically ill COVID-19 population in several hospitals throughout the U.S.; an interim analysis on the first 195 patients was conducted mid-October.

CytoDyn is currently enrolling patients in its Phase 2b/3 COVID-19 trial for patients with severe-to-critical indications in at least 13 hospitals and clinics across the U.S., which are identified in this press release and on the Companys website under the Clinical Trial Enrollment section of the homepage.

About Leronlimab (PRO 140)

The FDA has granted a Fast Track designation to CytoDyn for two potential indications of leronlimab for critical illnesses. The first indication is a combination therapy with HAART for HIV-infected patients and the second is for metastatic triple-negative breast cancer. Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases, including NASH.Leronlimab has completed nine clinical trials in over 800 people and met its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients).

In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab could significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.

In the setting of cancer, research has shown that CCR5 may play a role in tumor invasion, metastases, and tumor microenvironment control. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is, therefore, conducting a Phase 1b/2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019.

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

About CytoDyn

CytoDyn is a late-stage biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a critical role in the ability of HIV to enter and infect healthy T-cells. The CCR5 receptor also appears to be implicated in tumor metastasis and immune-mediated illnesses, such as GvHD and NASH.

CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients. The FDA met telephonically with Company key personnel and its clinical research organization and provided written responses to the Companys questions concerning its recent Biologics License Application (BLA) for this HIV combination therapy in order to expedite the resubmission of its BLA filing for this indication.

CytoDyn has completed a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients. CytoDyn plans to initiate a registration-directed study of leronlimab monotherapy indication. If successful, it could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV. No drug-related serious site injection reactions reported in about 800 patients treated with leronlimab and no drug-related SAEs reported in patients treated with 700 mg dose of leronlimab. Moreover, a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients; some patients on leronlimab monotherapy have remained virally suppressed for more than six years.

CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is atwww.cytodyn.com.

Forward-Looking Statements

This release contains certain forward-looking statements that involve risks, uncertainties and assumptions that are difficult to predict.Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as believes, hopes, intends, estimates, expects, projects, plans, anticipates and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. Forward-looking statements specifically include statements about leronlimab, its ability to have positive health outcomes, including for patients with COVID-19, the possible results of clinical trials, studies or other programs or ability to continue those programs, the ability to obtain emergency use authorization or regulatory approval for leronlimab for commercial sales, and the market for actual commercial sales. The Companys forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i) the results of the Companys clinical trials, including the possibility of unfavorable clinical trial results,(ii) the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Companys products, (iii) the market for, and marketability of, any product that is approved, (iv) the sufficiency of the Companys cash position, (v) the Companys ability to raise additional capital to fund its operations, (vi) the Companys ability to meet its debt obligations, if any, (vii) the Companys ability to enter into partnership or licensing arrangements with third parties, (viii) the Companys ability to identify patients to enroll in its clinical trials in a timely fashion, (ix) the Companys ability to achieve approval of a marketable product, (x) the design, implementation and conduct of the Companys clinical trials, ((xi) regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii) general economic and business conditions, (xiii) changes in foreign, political, and social conditions, and (xiv) various other matters, many of which are beyond the Companys control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form 10-K, and any risk factors or cautionary statements included in any subsequent Form 10-Q or Form 8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this release.

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Angelika Amon, professor of biology and a member of the Koch Institute for Integrative Cancer Research, died on Oct. 29 at age 53, following a two-and-a-half-year battle with ovarian cancer.

"Known for her piercing scientific insight and infectious enthusiasm for the deepest questions of science, Professor Amon built an extraordinary career and in the process, a devoted community of colleagues, students and friends," MIT President L. Rafael Reif wrote in a letter to the MIT community.

Angelika was a force of nature and a highly valued member of our community, reflects Tyler Jacks, the David H. Koch Professor of Biology at MIT and director of the Koch Institute. Her intellect and wit were equally sharp, and she brought unmatched passion to everything she did. Through her groundbreaking research, her mentorship of so many, her teaching, and a host of other contributions, Angelika has made an incredible impact on the world one that will last long into the future.

A pioneer in cell biology

From the earliest stages of her career, Amon made profound contributions to our understanding of the fundamental biology of the cell, deciphering the regulatory networks that govern cell division and proliferation in yeast, mice, and mammalian organoids, and shedding light on the causes of chromosome mis-segregation and its consequences for human diseases.

Human cells have 23 pairs of chromosomes, but as they divide they can make errors that lead to too many or too few chromosomes, resulting in aneuploidy. Amons meticulous and rigorous experiments, first in yeast and then in mammalian cells, helped to uncover the biological consequences of having too many chromosomes. Her studies determined that extra chromosomes significantly impact the composition of the cell, causing stress in important processes such as protein folding and metabolism, and leading to additional mistakes that could drive cancer. Although stress resulting from aneuploidy affects cells ability to survive and proliferate, cancer cells which are nearly universally aneuploid can grow uncontrollably. Amon showed that aneuploidy disrupts cells usual error-repair systems, allowing genetic mutations to quickly accumulate.

Aneuploidy is usually fatal, but in some instances extra copies of specific chromosomes can lead to conditions such as Down syndrome and developmental disorders including those known as Patau and Edwards syndromes. This led Amon to work to understand how these negative effects result in some of the health problems associated specifically with Down syndrome, such as acute lymphoblastic leukemia. Her expertise in this area led her to be named co-director of the recently established Alana Down Syndrome Center at MIT.

Angelikas intellect and research were as astonishing as her bravery and her spirit. Her labs fundamental work on aneuploidy was integral to our establishment of the center, say Li-Huei Tsai, the Picower Professor of Neuroscience and co-director of the Alana Down Syndrome Center. Her exploration of the myriad consequences of aneuploidy for human health was vitally important and will continue to guide scientific and medical research.

Another major focus of research in the Amon lab has been on the relationship between how cells grow, divide, and age. Among other insights, this work has revealed that once cells reach a certain large size, they lose the ability to proliferate and are unable to reenter the cell cycle. Further, this growth contributes to senescence, an irreversible cell cycle arrest, and tissue aging. In related work, Amon has investigated the relationships between stem cell size, stem cell function, and tissue age. Her labs studies have found that in hematopoetic stem cells, small size is important to cells ability to function and proliferate in fact, she posted recent findings on bioRxiv earlier this week and have been examining the same questions in epithelial cells as well.

Amon lab experiments delved deep into the mechanics of the biology, trying to understand the mechanisms behind their observations. To support this work, she established research collaborations to leverage approaches and technologies developed by her colleagues at the Koch Institute, including sophisticated intestinal organoid and mouse models developed by the Yilmaz Laboratory, and a microfluidic device developed by the Manalis Laboratory for measuring physical characteristics of single cells.

The thrill of discovery

Born in 1967, Amon grew up in Vienna, Austria, in a family of six. Playing outside all day with her three younger siblings, she developed an early love of biology and animals. She could not remember a time when she was not interested in biology, initially wanting to become a zoologist. But in high school, she saw an old black-and-white film from the 1950s about chromosome segregation, and found the moment that the sister chromatids split apart breathtaking. She knew then that she wanted to study the inner workings of the cell and decided to focus on genetics at the University of Vienna in Austria.

After receiving her BS, Amon continued her doctoral work there under Professor Kim Nasmyth at the Research Institute of Molecular Pathology, earning her PhD in 1993. From the outset, she made important contributions to the field of cell cycle dynamics. Her work on yeast genetics in the Nasmyth laboratory led to major discoveries about how one stage of the cell cycle sets up for the next, revealing that cyclins, proteins that accumulate within cells as they enter mitosis, must be broken down before cells pass from mitosis to G1, a period of cell growth.

Towards the end of her doctorate, Amon became interested in fruitfly genetics and read the work of Ruth Lehmann, then a faculty member at MIT and a member of the Whitehead Institute. Impressed by the elegance of Lehmanns genetic approach, she applied and was accepted to her lab. In 1994, Amon arrived in the United States, not knowing that it would become her permanent home or that she would eventually become a professor.

While Amons love affair with fruitfly genetics would prove short, her promise was immediately apparent to Lehmann, now director of the Whitehead Institute. I will never forget picking Angelika up from the airport when she was flying in from Vienna to join my lab. Despite the long trip, she was just so full of energy, ready to talk science, says Lehmann. She had read all the papers in the new field and cut through the results to hit equally on the main points.

But as Amon frequently was fond of saying, yeast will spoil you. Lehmann explains that because they grow so fast and there are so many tools, your brain is the only limitation. I tried to convince her of the beauty and advantages of my slower-growing favorite organism. But in the end, yeast won and Angelika went on to establish a remarkable body of work, starting with her many contributions to how cells divide and more recently to discover a cellular aneuploidy program.

In 1996, after Lehmann had left for New York Universitys Skirball Institute, Amon was invited to become a Whitehead Fellow, a prestigious program that offers recent PhDs resources and mentorship to undertake their own investigations. Her work on the question of how yeast cells progress through the cell cycle and partition their chromosomes would be instrumental in establishing her as one of the worlds leading geneticists. While at Whitehead, her lab made key findings centered around the role of an enzyme called Cdc14 in prompting cells to exit mitosis, including that the enzyme is sequestered in a cellular compartment called the nucleolus and must be released before the cell can exit.

I was one of those blessed to share with her a eureka moment, as she would call it, says Rosella Visintin, a postdoc in Amons lab at the time of the discovery and now an assistant professor at the European School of Molecular Medicine in Milan. She had so many. Most of us are lucky to get just one, and I was one of the lucky ones. Ill never forget her smile and scream neither will the entire Whitehead Institute when she saw for the first time Cdc14 localization: You did it, you did it, you figured it out! Passion, excitement, joy everything was in that scream.

In 1999, Amons work as a Whitehead Fellow earned her a faculty position in the MIT Department of Biology and the MIT Center for Cancer Research, the predecessor to the Koch Institute. A full professor since 2007, she also became the Kathleen and Curtis Marble Professor in Cancer Research, associate director of the Paul F. Glenn Center for Biology of Aging Research at MIT, a member of the Ludwig Center for Molecular Oncology at MIT, and an investigator of the Howard Hughes Medical Institute.

Her pathbreaking research was recognized by several awards and honors, including the 2003 National Science Foundation Alan T. Waterman Award, the 2007 Paul Marks Prize for Cancer Research, the 2008 National Academy of Sciences (NAS) Award in Molecular Biology, and the 2013 Ernst Jung Prize for Medicine. In 2019, she won the Breakthrough Prize in Life Sciences and the Vilcek Prize in Biomedical Science, and was named to the Carnegie Corporation of New Yorks annual list of Great Immigrants, Great Americans. This year, she was given the Human Frontier Science Program Nakasone Award. She was also a member of the NAS and the American Academy of Arts and Sciences.

Lighting the way forward

Amons perseverance, deep curiosity, and enthusiasm for discovery served her well in her roles as teacher, mentor, and colleague. She has worked with many labs across the world and developed a deep network of scientific collaboration and friendships. She was a sought-after speaker for seminars and the many conferences she attended. In over 20 years as a professor at MIT, she has mentored more than 80 postdocs, graduate students, and undergraduates, and received the School of Sciences undergraduate teaching prize.

Angelika was an amazing, energetic, passionate, and creative scientist, an outstanding mentor to many, and an excellent teacher, says Alan Grossman, the Praecis Professor of Biology and head of MITs Department of Biology. Her impact and legacy will live on and be perpetuated by all those she touched.

Angelika existed in a league of her own, explains Kristin Knouse, one of Amons former graduate students and a current Whitehead Fellow. She had the energy and excitement of someone who picked up a pipette for the first time, but the brilliance and wisdom of someone who had been doing it for decades. Her infectious energy and brilliant mind were matched by a boundless heart and tenacious grit. She could glance at any data and immediately deliver a sharp insight that would never have crossed any other mind. Her positive attributes were infectious, and any interaction with her, no matter how transient, assuredly left you feeling better about yourself and your science.

Taking great delight in helping young scientists find their own eureka moments, Amon was a fearless advocate for science and the rights of women and minorities and inspired others to fight as well. She was not afraid to speak out in support of the research and causes she believed strongly in. She was a role model for young female scientists and spent countless hours mentoring and guiding them in a male-dominated field. While she graciously accepted awards for women in science, including the Vanderbilt Prize and the Women in Cell Biology Senior Award, she questioned the value of prizes focused on women as women, rather than on their scientific contributions.

Angelika Amon was an inspiring leader, notes Lehmann, not only by her trailblazing science but also by her fearlessness to call out sexism and other -isms in our community. Her captivating laugh and unwavering mentorship and guidance will be missed by students and faculty alike. MIT and the science community have lost an exemplary leader, mentor, friend, and mensch.

Amons wide-ranging curiosity led her to consider new ideas beyond her own field. In recent years, she has developed a love for dinosaurs and fossils, and often mentioned that she would like to study terraforming, which she considered essential for a human success to life on other planets.

It was always amazing to talk with Angelika about science, because her interests were so deep and so broad, her intellect so sharp, and her enthusiasm so infectious, remembers Vivian Siegel, a lecturer in the Department of Biology and friend since Amons postdoctoral days. Beyond her own work in the lab, she was fascinated by so many things, including dinosaurs dreaming of taking her daughters on a dig lichen, and even life on Mars.

Angelika was brilliant; she illuminated science and scientists, says Frank Solomon, professor of biology and member of the Koch Institute. And she was intense; she warmed the people around her, and expanded what it means to be a friend.

Amon is survived by her husband Johannes Weis, and her daughters Theresa and Clara Weis, and her three siblings and their families.

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Angelika Amon, cell biologist who pioneered research on chromosome imbalance, dies at 53 - MIT News

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In episode #99 of Berkeley Talks, Chancellor Carol Christ joins Manual Cinemas co-artistic director Drew Dir to discuss the collectives presentation of Frankenstein, a Cal Performances co-commission, in a talk moderated by Cal Performances executive and artistic director Jeremy Geffen. Listen on Berkeley News. (Photo by Drew Dir)

Jeremy Geffen: Hello everyone. Im Jeremy Geffen, executive and artistic director for Cal Performances, and it is my great pleasure today to have with me two incredible people who both have intersections with the world of Frankenstein. Ill start out in alphabetical order with Carol Christ, who is the chancellor of UC Berkeley, as well as a scholar of 19th century, particularly Victorian literature, and in even further a great fan of Frankenstein, the book. And from Manual Cinema, one of their co-artistic directors, Drew Dir, whos joining us from Chicago. It is Drews concept for Frankenstein that we will see in the performance that will follow. Thank you both for being here.

Jeremy Geffen: A first question for you Drew, because this may lay some of the groundwork for what we talk about beyond this. Could you explain what is meant by the term cinematic shadow puppetry?

Drew Dir: Yeah, yeah, of course. So the name of our company is Manual Cinema and it also describes sort of the mission statement of the company, which is to create something that looks like cinema, that looks like a movie by hand in front of you using handmade materials. And weve created a technique called cinematic shadow puppetry, which uses old-school overhead projectors, the same kind that you mightve used in math class when you were a kid, and uses a whole bank of overhead projectors to use slides, shadow puppets, and create something that doesnt look like a traditional childrens shadow puppet play, but create something that resembles an animated film made live in front of you.

Jeremy Geffen: Well, Im very glad that you made the introductory couple of minutes of video that show how these techniques are put to use in a live performance, because otherwise it could be mistaken for just incredible digital work. And there theres so much artistry manual artistry that goes into your work.

You said something in an interview, which I thought would be something that through to you, both, which is that there are no good or evil characters in Frankenstein.

Drew Dir: Weve always thought of the story as the story of two characters, Victor and the creature that he creates. And its also for us, a dual twin story about creation and about abandonment. And, we always wanted to be able to allow the audience to sympathize with Victor and to understand why he creates and even beyond the why of why he creates, but to feel the feeling of excitement, ambition, energy, that he feels when he brings something new into the world. And to also allow the audience to understand the immediate remorse and regret that he feels at the moment that the creature, you know, opens his eyes and looks back at his creator.

At the same time, we always wanted the audience to understand the violence of Victors creature as the product of a series of circumstances that brought him to violent acts. So, to us, its Greek tragedy: There arent good and evil characters. There are only characters who make errors based on choices that they made up to that point.

Carol Christ: Yeah, I think thats a wonderful way of talking about the book. I think the book is profoundly about good and evil. And I often think of the creature and Victor as almost a doppelgnger, its almost like theyre a single person and they keep switching roles, which one is Paradise Lost is a really important frame of reference for this book. And Mary Shelley is always asking you, which one is Adam? Which one is Satan? What responsibility does a God have for his creature? And if a creature is rejected, is he justified in the evil that he does afterwards? So, its profoundly about a theory of good and evil.

Jeremy Geffen: I was thinking, Carol, in your position as the chancellor of an enormous research university, about the prescience of the question that Mary Shelley presents us, is there something Frankenstein is trying to do good or trying to do something that has benefit to to society? But along with benefit comes the possibility of either manipulation or use for dangerous or reckless uses. Im thinking about the questions before us today regarding genetics, artificial intelligence and even in vitro fertilization.

Carol Christ: No, I think youre absolutely right. Frankenstein has proven to be an enormously resonant text today. There are a number of contemporary writers that have been rewriting Frankenstein, Ian McEwan in Machines Like Me or Jeanette Winterson has a wonderful book called Frankissstein that is a double narrative, one of Mary Shelley writing Frankenstein, the other of a kind of parallel story of artificial intelligence. There was even a Frankenstein in Baghdad, which was a really chilling book. So, I think its just its a narrative for our time with the incredible capability through genetic technology, through artificial intelligence that mankind has obtained.

Drew Dir: We spoke a lot about social media companies and the internet when we were developing the show and about Silicon Valley and about how, you know, there are no ill intentions. People in Silicon Valley there are few ill intentioned people in Silicon Valley who one means to do well. And yet the internet companies like Facebook grow so large and so complex that even their creators are just beginning to understand what it is theyve created and the consequences their creations have on the world.

Jeremy Geffen: I was wondering as I watched this performance, which is so wonderfully humane, even with regard to the monster, but especially with regard to the creature, what it means to be envisioning a dramatic work about this novel in the shadow of so many popular culture, not necessarily interpretations, but weights of images from Frankenstein.

Drew Dir: Yeah. We, I mean, one of the reasons we were attracted to Frankenstein in the first place was because there are so many iterations of it, it felt like such a rich text. Theres a whole Frankenstein text that lives outside of Mary Shelleys novel because, you know, the Frankenstein, the character has taken on a life of its own in the popular culture beyond like what she created. But when we first began looking at the title, we landed on Frankenstein because you know, most of our work is non-verbal, its told without language. And of course, one of the most famous nonverbal characters in popular culture is Frankensteins monster. Even though ironically, as, you know, Carol, Frankensteins monster is quite loquacious in the novel. He speaks very well for himself, but we were looking for a good marriage between our form and some sort of content. And so, doing our own interpretation on this famous non-verbal character of the creature was really exciting to us.

Another aspect that felt really exciting was when we landed on the title and went back to the novel, we were reminded about what an interesting structure the original novel has, the Gothic structure of the nested framing narratives. And we thought, Oh, this is fascinating. This is a book thats told with these Russian nesting dolls in which each part of the book is narrated by a different character. One part is narrated by Victor and another is narrated by the creature itself, another by this ships captain who finds Victor in the Arctic. And we thought what an interesting challenge to find a, to adapt a narrative voice for a visual idiom. So we cant have someone narrating our story, or we cant have different characters narrating our story, but what if we could create these different frames and create a different visual style for each one, it would be like personal narrative voice.

And so having all the different versions of Frankenstein the story was really helpful in that regard because across the history of American cinema, like every era of American cinema has produced its own Frankenstein. It was one of the very first novels to be adapted by Thomas Edisons cinema company, like a very simple 10-minute silent film of Frankenstein. And of course the 1930s James Whale version and the lots of different versions up through the 60s, 70s into today. And so, it was really rewarding to draw on each of these arrows, especially silent film and kind of stitch together our own Frankensteins monster from all these different cultural tropes that have been produced over the past 200 years of Frankenstein.

Carol Christ: Yeah, I think thats so interesting as one of the things that I loved about, about your version of Frankenstein is theres almost a meta-theatrical element to it in which the crafting of these puppets and the silhouettes, the shadow puppets is almost an analogy to what Victor is doing when creating the creature itself. Theres a really interesting point about the history of Frankenstein by the time Mary Shelley returns to London, which is five years after Frankenstein is published. There were five versions of Frankenstein on the stage. So, it almost immediately becomes this really copular theatrical property. And the arguments been made, I think really interestingly, that one of the things that draws filmmakers to Frankenstein is its a kind of analogy that Victors creation of the creature to the art of cinema itself.

Drew Dir: Oh, I love that. I love that.

Jeremy Geffen: That ties us into something that Manual Cinemas Frankenstein also creates another, a bigger Russian nesting doll.

Carol Christ: Yeah, thats absolutely right. I mean, I love, I always call them not Russian nesting dolls, but Chinese boxes. And youve created a Chinese box around the Chinese boxes of Mary Shelleys novel with the story of Mary Shelley herself and her creation of Frankenstein. I thought that was absolutely wonderful, and wonderful in your connection of Mary Shellys trauma about giving birth and about babies dying as analogous indeed, one of the motivations to her imagination to create the story.

Drew Dir: Yeah, from the very beginning we knew we wanted to include Mary Shelleys biography in the show somehow. And actually, the very first draft of the show had so much about Mary Shelley that we had to cut so much because her life is really fascinating. And, you know, just like briefly a couple interesting things about her was that her mother is Mary Wollstonecraft, the 18th century feminist and thinker, and Wollstonecraft actually died shortly after childbirth giving birth to Mary Shelley.

And she also had this half sister, Fanny, who committed suicide during the writing of Frankenstein. Of course, her husband, Percy Shelley, who she was running around Europe with at the time that she wrote Frankenstein along with Lord Byron. But the detail from the biography that stuck out to us was the story of the birth of first daughter, Clara, who died shortly after Mary gave birth to her.

And she wrote a journal entry shortly after the death, in which Mary had a nightmare in which she laid hands on the dead child and rubbed it, and basically rubbed it back to life. And when we read that, we thought, Well, this is fascinating because I dont think wed ever thought of Frankenstein Frankenstein is a story of a lot of things, but it didnt strike us as a story about grief and loss and, and putting that in the mix with everything else thats going on in the original novel felt really interesting to us thinking of it as a novel of grief.

And also, of course, introducing the idea of motherhood and childbirth back into the book, which it feels like the story really opens up when you read the novel in that way. And it felt really interesting to us that Mary wrote this novel, you know, basically like throughout a postpartum period. So, those were the, those were the elements that struck us as really interesting and why we want it to include Marys story alongside or around the original Frankenstein story.

Carol Christ: I think thats really profound about Frankenstein. One of the things that I emphasize when I teach Frankenstein is how young Mary Shelley was. I mean, she was 18 and 19 when she writes this and shes living in Europe in this kind of house, they all go to Percy Shelly, Gordon, Lord Byron, Byrons physician and Claire Clairmont who Byron is having an affair with and theyre young and theyre reckless. And yet, Mary Shelleys life is one of having lost this first child, had a second shot. Shes nursing when shes at this home in Geneva. And so, you know, reinforcing what you just said, when Victor Frankenstein, in the novel, falls asleep right after he finishes creating the creature, he has a dream about his dead mother walking, or Elizabeth, walking through the streets, whos his fianc. He grasps Elizabeth and she turns into the skeleton of his dead mother in his arms. So, its a book thats just haunted by death. And I think haunted by grief, I think youre right.

Jeremy Geffen: Yeah, youre right. Youre totally right. In fact, the dream, Im going to mess up the phrase livid with the power of death. Your point Carol, about Mary Shelleys youth is extraordinary because she went on to write another six novels. She was incredibly accomplished, but when the book was published, I dont believe her name was attached to it.

Carol Christ: Yeah, thats right. Its by Anonymous. And theres a big argument in the scholarly literature about how important Percy Shelley was to the revisions. And in the novel. One of the things I loved about your version of Frankenstein is the sense of the marital dynamics, or I guess they werent married at the point, that the dynamics between Mary Shelley and Percy Shelley that shes, you know, trying to get his attention and hes just writing away. And I felt that was really well done.

Drew Dir: Its a fascinating relationship on its own and is enough material for an entire novel or story itself, for sure.

Jeremy Geffen: Obviously this is not an unfamiliar concept to offer lovers, but it took me a little while to realize that that all of the actors, whether they are male or female are played by women. And I wanted to ask Drew how you arrived at that decision.

Drew Dir: Well, its a little bit of a marriage between the material and the companys mission. Manual Cinema, all of our shows for the most part, either portray female characters as protagonists or draw on work by female authors. Its maybe not the most visible part of our mission, but it is a sort of a core part of our mission. And when we got ahold of Frankenstein and discovered all these beams and ideas and motifs of motherhood, we really thought about how do we put Mary Shelley back into the story, which by the way, when you read the novel, it really is full of male characters. I mean, almost exclusively, except for, you know, Elizabeth and his mother, most notably. And so we just wanted to find any kind of way where we could remind the audience as much as possible that this is written by a woman. And there are, there are certain themes and motifs and Frankenstein that you mightve missed if you hadnt been thinking about maternity or childbirth before.

Carol Christ: Yeah. I love the fact that you made that you had the same actor play, both Mary Shelley and Victor Frankenstein, because it really makes it clear that that Victor Frankenstein is expressing so much of her own, you know, desires, dreams, traumas, in the character.

Drew Dir: Yeah. A lot of people, you know, think that Victor Frankenstein is just a stand-in for her has been Percy, and I think there is a totally a valid critique there, but what that loses is that Mary is a creator as well. And, and she has felt these things, shes felt that thrill of creation, shes felt the regret of ambition and all those things that Victor feels as an author.

Jeremy Geffen: Well, before two such deeply knowledgeable individuals as yourself on the subject of Frankenstein, I feel embarrassed to admit that my first exposure beyond cartoons was Young Frankenstein, which Ive watched.

It was this production that was the impetus for me to finally read the novel and to recognize that so much of what we think we know about the novel is not, its not an accurate reflection of what was in the text itself. And that is actually one of the themes of Cal Performances current season, Fact or Fiction. So, I think we could probably talk for another hour or so about Frankenstein and about your fantastic work Drew, but were going to call it quits here. And I want it to say a great thank you to you Drew Dir from Manual Cinema and Carol Christ of UC Berkeley, for making the time for this conversation. And we look greatly forward to the performance.

Carol Christ: Thank you Jeremy.

Drew Dir: Thank you so much for presenting Frankenstein, Jeremy and Carol good luck with your class. I have to say that a big reason why I really love Frankenstein the novel is because of an introduction to romanticism class that I took as a freshman in college. And, and the way, that Frankenstein was taught to me stuck with me all through the years and was so important to me putting up this production. So, I hope your students, Im sure your students will feel as inspired as I was.

Carol Christ: Well, thats wonderful to hear and thank you so much for such a spectacular theatrical event about Frankenstein.

Read more:
Berkeley Talks transcript: How Mary Shelley's 'Frankenstein' took on a life of its own - UC Berkeley

Recommendation and review posted by Bethany Smith

Beathan Report has released the International report on The Autologous Stem Cell Based Therapies market, which is made up of advice about each of the essential parameters of this market like ingestion and the manufacturing patterns coupled with all the earnings patterns for the prediction period. Concerning creation aspect, the report provides complete detailed analysis about the manufacturing procedures combined with the gross financials accumulated by the very best most producers working within this business. The main facet of this Autologous Stem Cell Based Therapies market thats covered in the report helps the customers and the associations to better comprehend the company profile concerning drivers, restraints, challenges, and opportunities affecting and pertaining the market dynamics.

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Key market players

Major competitors identified in this market include Regeneus, Mesoblast, Pluristem Therapeutics Inc, US STEM CELL, INC., Brainstorm Cell Therapeutics, Tigenix, Med cell Europe, etc.

Based on the Region:

Asia-Pacific (China, Japan, South Korea, India and ASEAN)

North America (US and Canada)

Europe (Germany, France, UK and Italy)

Rest of World (Latin America, Middle East & Africa)

COVID-19 has affected the Overall worldwide companies and itll have a enormous time for the company recovery. Vast majority of the business sectors have realigned their company plans, priorities, and have amended their economic planning so as to stay in the company and keep their standing on the international platform. The thorough evaluation of this Autologous Stem Cell Based Therapies market will enable the brand new market entrants to acquire reliable market approaches and strategy powerful action plans for the prediction period.

Based on the Type:

Embryonic Stem Cell

Resident Cardiac Stem Cells

Umbilical Cord Blood Stem Cells

Based on the Application:

Neurodegenerative Disorders

Autoimmune Diseases

Cardiovascular Diseases

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At Beathan Report, we understand that the research we provide is only as good as the outcome it inspires. These reports are generated by well-renowned publishers on the basis of the data acquired from an extensive research and credible business statistics. Thats why we are proud to provide the widest range of research products, multilingual 24/7 customer support and dedicated custom research services to deliver the insights you need to achieve your goals. Take a look at few of our aspects that makes Beathan Report an asset to your business.

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Autologous Stem Cell Based Therapies Market to Witness Increase in Revenues by 2020-2026 - PRnews Leader

Recommendation and review posted by Bethany Smith

KING OF PRUSSIA, Pa., Nov. 2, 2020 /PRNewswire/ --Global biotherapeutics leader CSL Behring announced today that its investigational, plasma-derived hemopexin therapy (CSL889) received orphan drug designation from both the European Commission and the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development for the treatment of sickle cell disease (SCD). These designations grant special status to drugs and biological products intended to treat a rare disease, affecting less than 200,000 patients in the US or affecting not more than five in 10,000 people in the European Union.

CSL889 is a form of plasma-derived hemopexin, an important, naturally occurring protein produced in the body whose levels are decreased in patients with SCD. Low levels of hemopexin have been associated with increased symptoms in SCD, particularly acute vaso-occlusive crises (VOC). VOC, the most common manifestation in SCD, are severe, debilitating episodes characterized by severe pain. There is no approved treatment for acute VOC, so episodes can only be managed with supportive measures such as fluids and pain killers.

"Having treated hundreds of adults and children living with sickle cell disease over 30 years, I'm intensely aware of the need for novel and effective therapies, especially to relieve the tremendous pain from VOC," said Professor Greg Kato, who is leading the clinical development of CSL 889 at CSL Behring. "This newly granted orphan status recognizes the urgency for progressing new treatment options into the clinic."

CSL Behring has two Phase I SCD programs poised to evolve the treatment paradigm for patients: CSL889 hemopexin therapy for the treatment of VOC and CSL200 lentiviral stem cell gene therapy for long-term disease management.

About Sickle Cell Disease

Sickle Cell Disease is a hereditary blood disorder in which red blood cells contain an abnormal type of hemoglobin, causing some of the cells to become distorted into a crescent, or sickle-shape. These misshapen red blood cells have difficulty passing through small blood vessels, slowing and blocking blood flow to areas of the body, damaging tissue that isn't receiving a normal flow of blood. Sickle Cell Disease can lead to episodes of severe pain, strokes, kidney, lung and heart problems, slow growth, vision problems and infection vulnerability. While frequency of Sickle Cell Disease varies globally, it is estimated to impact 100,000 people in the US and 1 in 10,000 persons in the European Union.

About CSL Behring

CSL Behringis a global biotherapeutics leader driven by its promise to save lives. Focused on serving patients' needs by using the latest technologies, we develop and deliver innovative therapies that are used to treat coagulation disorders, primary immune deficiencies, hereditary angioedema, respiratory disease, and neurological disorders. The company's products are also used in cardiac surgery, burn treatment and to prevent hemolytic disease of the newborn.

CSL Behring operates one of the world's largest plasma collection networks, CSL Plasma. The parent company, CSL Limited (ASX:CSL;USOTC:CSLLY), headquartered in Melbourne, Australia, employs more than 27,000 people, anddelivers its life-saving therapies to people in more than 100 countries. For inspiring stories about the promise of biotechnology, visit Vita CSLBehring.com/vita and follow us on Twitter.com/CSLBehring.

SOURCE CSL Behring

Read more here:
Orphan Drug Designation Granted for CSL Behring's Investigational Plasma-Derived Hemopexin Therapy for Sickle Cell Disease - PRNewswire

Recommendation and review posted by Bethany Smith

The Global Gene Therapies for Cancer Treatment Market report is deep study of the present market dynamics. It consists of the detailed study of current market trends along with the past statistics. The past years are considered as reference to get the predicted data for the forecasted period. Various important factors such as market trends, revenue growth patterns market shares and demand and supply are included in almost all the market research report for every industry. A significant development has been recorded by the market of Gene Therapies for Cancer Treatment, in past few years. It is also for it to grow further. Various important factors such as market trends, revenue growth patterns market shares and demand and supply are included in almost all the market research report for every industry. A systematized methodology is used to make a Report on the Global Gene Therapies for Cancer Treatment market. For the analysis of market on the terms of research strategies, these techniques are helpful. All the information about the Products, manufacturers, vendors, customers and much more is covered in research reports.

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There are different marketing strategies that every marketer looks up to in order to ace the competition in the Global Gene Therapies for Cancer Treatment market. Some of the primary marketing strategies that is needed for every business to be successful are Passion, Focus, Watching the Data, Communicating the value To Your Customers, Your Understanding of Your Target Market. There is a target set in market that every marketing strategy has to reach. The study is done with the help of analysis such as SWOT analysis and PESTEL analysis. SWOT analysis includes the study of Threats, weaknesses, strengths and opportunities that the Gene Therapies for Cancer Treatment market. Whereas PESTEL analysis is the study concerning Economic, Technological, legal political, social, environmental matters. For the analysis of market on the terms of research strategies, these techniques are helpful.

Major companies of this report:

Takara BioTocagenVBL TherapeuticsCold GenesysGenprexMomotaro-GeneMultiVirSynerGene TherapeuticsZiopharm OncologyAnchiano TherapeuticsCelgeneCelsionBluebird Bio

Browse the complete report @ https://www.orbispharmareports.com/global-gene-therapies-for-cancer-treatment-market-2019-present-status-and-future-opportunities-by-major-companies-typs-and-applications-2024/?utm_source=Nilam

Potential consumers, market values, and the future scope for the Gene Therapies for Cancer Treatment market are explained thoroughly to the users in this report. The key players of Gene Therapies for Cancer Treatment industry, their product portfolio, market share, industry profiles is studied in this report. It is very important for the vendors to provide customers with new and improved product/ services in order to gain their loyalty.

Segmentation by Type:

Somatic Cell Gene Therapy (SCGT)Germline Gene Therapy (GGT)

Segmentation by Application:

Cancer Research CentersDiagnostic LaboratoriesCancer HospitalsOthers

The study of various segments of the global Gene Therapies for Cancer Treatment market are also covered in the research report. In addition to that, for the forecast periods determination of factors like market size and the competitive landscape of the market is analyzed in the report. Due to the increasing globalization and digitization, there are new trends coming to the market every day. The research report provides the in-depth analysis of all these trends.

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About Us :

At Orbispharma we curate the most relevant news stories, features, analysis and research reports on the important challenges undertaken by the pharmaceutical and related sectors. Our editorial philosophy is to bring you sharp, focused and informed perspective of industries, the end users and application of all upcoming trends into the pharma sector. Orbispharma believes in conversations that can bring a change in one of the most crucial economic sectors in the world. With these conversations we wish our customers to make sound business decisions with right business intelligence.

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Read the original here:
Gene Therapies for Cancer Treatment Market 2020 Trends Analysis, Top Manufacturers 2024 : Mylan Pharmaceuticals, Pfizer, Roche, Almirall, Bausch...

Recommendation and review posted by Bethany Smith

Cancer Gene Therapy Market Forecast 2020-2026

The Global Cancer Gene Therapy Market research report provides and in-depth analysis on industry- and economy-wide database for business management that could potentially offer development and profitability for players in this market. This is a latest report, covering the current COVID-19 impact on the market. The pandemic of Coronavirus (COVID-19) has affected every aspect of life globally. This has brought along several changes in market conditions. The rapidly changing market scenario and initial and future assessment of the impact is covered in the report. It offers critical information pertaining to the current and future growth of the market. It focuses on technologies, volume, and materials in, and in-depth analysis of the market. The study has a section dedicated for profiling key companies in the market along with the market shares they hold.

The report consists of trends that are anticipated to impact the growth of the Cancer Gene Therapy Market during the forecast period between 2020 and 2026. Evaluation of these trends is included in the report, along with their product innovations.

Get a PDF Copy of the Sample Report for free @ https://www.upmarketresearch.com/home/requested_sample/42057

The Report Covers the Following Companies:Cell GenesysAdvantageneGenVecBioCancellCelgene and Epeius BiotechnologiesIntrogen TherapeuticsZIOPHARM OncologyMultiVirShenzhen SiBiono GeneTech

By Types:Insertion of new genes into the bodyOther

By Applications:HospitalsDiagnostic CentresDrug ManufacturersResearch Institutes

Furthermore, the report includes growth rate of the global market, consumption tables, facts, figures, and statistics of key segments.

By Regions:

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Years Considered to Estimate the Market Size:History Year: 2015-2019Base Year: 2019Estimated Year: 2020Forecast Year: 2020-2026

Important Facts about Cancer Gene Therapy Market Report:

What Our Report Offers:

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About UpMarketResearch:Up Market Research (https://www.upmarketresearch.com) is a leading distributor of market research report with more than 800+ global clients. As a market research company, we take pride in equipping our clients with insights and data that holds the power to truly make a difference to their business. Our mission is singular and well-defined we want to help our clients envisage their business environment so that they are able to make informed, strategic and therefore successful decisions for themselves.

Contact Info UpMarketResearchName Alex MathewsEmail [emailprotected]Organization UpMarketResearchAddress 500 East E Street, Ontario, CA 91764, United States.

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Global Cancer Gene Therapy Market 2019 Trends, Market Share, Industry Size, Opportunities, Analysis and Forecast To 2025 - TechnoWeekly

Recommendation and review posted by Bethany Smith

Jay Bradner, president of the Novartis Institutes for BioMedical Research, pictured above. Photo courtesy of Novartis.

Novartis acquired Cambridge, Massachusetts-based Vedere Bio in a deal totaling up to $280 million. The takeout broadens Novartis footprint in gene and cell therapy, with Vedere Bios gene therapy platforms for ophthalmic disorders.

In April 2018, Novartis bought Illinois-based AveXis for $8.7 billion, largely to acquire the companys gene therapy candidate, AVXS-101, for spinal muscular atrophy, but also for the companys gene therapy platform. The drug was subsequently approved in May 2019 as Zolgensma.

The technology platforms Novartis picks up with Vedere Bio include light-sensing proteins that can be delivered to retinal cells as well as adeno-associated virus (AAV) delivery vectors for intravitreal injection. They believe the technologies can be used widely to treat vision loss caused by photoreceptor death, including a broad range of inherited retinal dystrophies.

Inherited retinal dystrophies (IRDs) are marked by loss of photoreceptor cells and progressive vision loss. More than 2 million people worldwide are affected, often with complete blindness. Existing therapies target only one of more than 250 genes associated with IRDs.

The next frontier in ophthalmology involves finding ways to bring potentially transformative gene therapies to a broader patient population, said Jay Bradner, president of the Novartis Institutes for BioMedical Research. The acquisition of Vedere Bio reflects our commitment to next-generation gene therapy and brings hope to patients with otherwise untreatable forms of vision loss.

Novartis reports it has three specific programs in gene therapyAAVs, chimeric antigen receptor T cells (CAR-Ts) and CRISPR. The Vedere Bio platform directly effects surviving cells in the retina, altering their behavior. It also is promising for other conditions associated with photoreceptor loss, including a dry form of age-related macular degeneration (AMD) known as geographic atrophy. More than 5 million people worldwide are affected by geographic atrophy.

Under the terms of the deal, Novartis is paying Vedere Bio $150 million upfront. Vedere will be eligible for up to $130 million in milestone payments for a total of $280 million.

The company was founded on technology from UC Berkeley researchers Ehud Isacoff and John G. Flanner. The technology arose jointly between UC Berkeley and the School of Veterinary Medicine at the University of Pennsylvania. It was formed in June 2019 in the Atlas Venture incubator, then launched with a Series A financing worth $21 million and began operations at LabCentral in Cambridge, Massachusetts.

Shortly before the acquisition, some of the companys earlier-stage restoration and vision preservation assets were spun out into a new entity, Vedere Bio II, Inc.

Vedere Bios photoreceptor-protein-based optogenetics program has important advantages over competing approaches and brings us one step closer to delivering functional vision to patients in need, said Cyrus Mozayeni, chief executive officer and president of Vedere Bio and Atlas Venture Entrepreneur in Residence. Our proprietary intravitreal capsids enable not only Vedere Bios optogenetics products but also other ocular gene therapies. Our sale to Novartis is an important milestone in advancing Vedere Bios most advance programs to patients around the world. At the same time, I look forward to working with our experienced team to advance our highly innovative, earlier stage assets as part of the newly established Vedere Bio II.

Vedere Bio II will run completely independently from Novartis and Vedere Bio. Its goal is to develop a pipeline of novel vision restoration and vision preservation drugs by focusing on underserved indications. It is supported by the full Vedere Bio investor syndicate, including Atlas Venture, Mission BioCapital and Foundation Fighting Blindness (RD Fund).

Kevin Bitterman, partner at Atlas Venture and chairman of the Vedere Bio board of directors, said, The acquisition of Vedere Bio by Novartis speaks to the strength of the underlying science from our founders and to the incredible job the team has done in advancing these programs over the past year.

Original post:
Novartis Expands Footprint in Gene and Cell Therapy with Vedere Bio Acquisition - BioSpace

Recommendation and review posted by Bethany Smith

According to Market Study Report, Cancer Gene Therapy Market provides a comprehensive analysis of the Cancer Gene Therapy Market segments, including their dynamics, size, growth, regulatory requirements, competitive landscape, and emerging opportunities of global industry.

The Cancer Gene Therapy Market is projected to reach USD XX Billion by 2023 from USD XX Billion in 2018, at a CAGR of XX%. This report spread across 122 Pages, Profiling 10 Companies and Supported with 114 Tables and 24 figures are now available in this research.

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The Cancer Gene Therapy Market report is a compilation of first-hand information, qualitative and competitive assessment industry analysts, inputs from industry experts, and industry participants across the value chain. The research report market provides an in-depth analysis of parent market trends, macro-economic indicators, and governing factors along with market attractiveness as per segments. The report also maps the qualitative impact of various market factors on market segments, trending Key Factors, and geographies.

The following players are covered in this report:

Adaptimmune

Bluebird bio

Celgene

Shanghai Sunway Biotech

Shenzhen SiBiono GeneTech

SynerGene Therapeutics

Altor BioScience

Amgen

Argenx

BioCancell

GlaxoSmithKline

Merck

OncoGenex Pharmaceuticals

Transgene

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Growing popularity of soft adventure sports and increasing focus towards fitness are significant factors driving the growth of the Cancer Gene Therapy market. However, high cost of Cancer Gene Therapy might hinder the growth of the Cancer Gene Therapy market. The demand for Cancer Gene Therapy is high in North America and Europe region and the APAC is expected to grow at high CAGR which is creating lucrative business opportunities for the companies operating in the market.

MARKET SEGMENTATION

Breakdown Data by Type

Oncolytic Virotherapy

Gene Transfer

Gene-Induced Immunotherapy

Cancer Gene Therapy Breakdown Data by Application

Hospitals

Diagnostics Centers

Research Institutes

Based on regional and country-level analysis, the Cancer Gene Therapy market has been segmented as follows:

North America

United States

Canada

Europe

Germany

France

U.K.

Italy

Russia

Nordic

Rest of Europe

Asia-Pacific

China

Japan

South Korea

Southeast Asia

India

Australia

Rest of Asia-Pacific

Latin America

Mexico

Brazil

Middle East & Africa

Turkey

Saudi Arabia

UAE

Rest of Middle East & Africa

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This report provides a thorough analysis of the Cancer Gene Therapy market through detailed research on a variety of topics to help players build strong growth strategies and strong presence in the industry. Readers will also be informed of the important sustainability strategies adopted by leading companies when operating in the Cancer Gene Therapy market. The analyst also thoroughly evaluated the impact of these strategies on market growth and competition.

Chapter Details of Cancer Gene Therapy Market:

Part 01: Executive Summary

Part 02: Scope of The Report

Part 03: Cancer Gene Therapy Market Landscape

Part 04: Cancer Gene Therapy Market Sizing

Part 05: Cancer Gene Therapy Market Segmentation by Type

Part 06: Five Forces Analysis

Part 07: Customer Landscape

Part 08: Geographic Landscape

Part 09: Decision Framework

Part 10: Drivers and Challenges

Part 11: Market Trends

Part 12: Vendor Landscape

Part 13: Vendor Analysis

Reason to access this report:

The report will help the market leaders/new entrants in this market with information on the closest approximations of the revenue numbers for the overall Cancer Gene Therapy market and its sub segments. This report will help the stakeholders to understand the competitive landscape, to gain more insights to better position their businesses, and to plan suitable go-to-market strategies.

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About Us

At Beathan Report, we understand that the research we provide is only as good as the outcome it inspires. These reports are generated by well-renowned publishers on the basis of the data acquired from an extensive research and credible business statistics. Thats why we are proud to provide the widest range of research products, multilingual 24/7 customer support and dedicated custom research services to deliver the insights you need to achieve your goals. Take a look at few of our aspects that makes Beathan Report an asset to your business.

Read more:
Cancer Gene Therapy Market Growth and Future Prospects Analyzed - Aerospace Journal

Recommendation and review posted by Bethany Smith

Oct. 27, 2020 06:30 UTC

MONTPELLIER, France & LYON, France--(BUSINESS WIRE)-- Regulatory News:

Sensorion (Paris:ALSEN) (FR0012596468 ALSEN) a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders, and Novasep, a leading supplier of services and technologies for the life sciences industry, announce the signature of an agreement for the manufacturing of adeno-associated virus (AAV) vectors. The agreement covers the gene therapy program targeting OTOF (OTOF-GT), the gene encoding the otoferlin protein. Mutations in OTOF lead to severe or profound congenital hearing loss.

Sensorion is currently advancing two preclinical gene therapy programs conducted under a broad multi-program research partnership with the Genetics and Physiology of Hearing Unit at Institut Pasteur (Paris). OTOF-GT, the most advanced program, aims to restore the expression of Otoferlin in the inner hair cells of the ear where it plays an essential role in hearing.

Under the terms of the agreement, Novasep will be in charge of developing and manufacturing (cell culture, AAV expression, purification, aseptic distribution and quality control) the two AAV vectors designed for the Sensorion OTOF-GT project and will supply Drug Product batches to support preclinical and clinical studies.

Securing state-of-the-art research and development capabilities and Good Manufacturing Practice (GMP) capacity, like those offered by Novasep, is a key step and underlines the progress of our Otoferlin gene therapy program, said Nawal Ouzren, CEO of Sensorion. Earlier this year, we announced the first positive preliminary preclinical OTOF-GT data in non-human primates.

We are delighted and proud to contribute to the progress of Sensorions innovative gene therapy programs targeting OTOF. Through this agreement, Novaseps expertise in the field of viral vector development and manufacturing is once again recognized and valued, said Cedric Volanti, Novaseps President of Biopharma Solutions.

About Sensorion

Sensorion is a pioneering clinical-stage biotech company, which specializes in the development of novel therapies to restore, treat and prevent within the field of hearing loss disorders. Its clinical-stage portfolio includes one Phase 2 product: SENS-401 (Arazasetron) for sudden sensorineural hearing loss (SSNHL). Sensorion has built a unique R&D technology platform to expand its understanding of the pathophysiology and etiology of inner ear related diseases enabling it to select the best targets and modalities for drug candidates. The Company is also working on the identification of biomarkers to improve diagnosis of these underserved illnesses. In the second half of 2019, Sensorion launched two preclinical gene therapy programs aimed at correcting hereditary monogenic forms of deafness including Usher Type 1 and deafness caused by a mutation of the gene encoding for Otoferlin. The Company is potentially uniquely placed, through its platforms and pipeline of potential therapeutics, to make a lasting positive impact on hundreds of thousands of people with inner ear related disorders, a significant global unmet medical need.www.sensorion-pharma.com

About Novasep

Novasep provides cost-effective and sustainable manufacturing solutions for the life sciences industries.

For 20 years, Novasep has acquired experience in developing and manufacturing a wide range of viral vectors through its sites in Belgium: AAV, Adenovirus, Lentivirus, HSV, VEEV, VSV, for cell & gene therapy, immunotherapy, vaccination and other therapies from process development to cGMP production.

Novasep also offers Fill & Finish services for viral vectors, attenuated and live viruses, mAbs, plasmids and other biologics, from formulation to packaging.

As part of its growth strategy Rise-2, Novasep recently unveiled a new facility, Senrise-IV, dedicated to the commercial production of viral vectors which has been completed last year by Senefill, a new Fill & Finish commercial facility for aseptic operations. Both facilities located in Seneffe, Belgium, will contribute to the success of biopharmaceuticals projects.www.novasep.com

Label: SENSORION ISIN: FR0012596468 Mnemonic: ALSEN

Disclaimer

This press release contains certain forward-looking statements concerning Sensorion and its business. Such forward looking statements are based on assumptions that Sensorion considers to be reasonable. However, there can be no assurance that such forward-looking statements will be verified, which statements are subject to numerous risks, including the risks set forth in the 2019 Annual Report published on 29 April 2020 and available on our website and to the development of economic conditions, financial markets and the markets in which Sensorion operates. The forward-looking statements contained in this press release are also subject to risks not yet known to Sensorion or not currently considered material by Sensorion. The occurrence of all or part of such risks could cause actual results, financial conditions, performance or achievements of Sensorion to be materially different from such forward-looking statements.

This press release and the information that it contains do not constitute an offer to sell or subscribe for, or a solicitation of an offer to purchase or subscribe for, Sensorion shares in any country. The communication of this press release in certain countries may constitute a violation of local laws and regulations. Any recipient of this press release must inform him/herself of any such local restrictions and comply therewith.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201026005828/en/

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Sensorion and Novasep Announce Signature of a Gene Therapy Product Development and Manufacturing Agreement - BioSpace

Recommendation and review posted by Bethany Smith

Beathan Report recently released a research report on the Gene Therapy for Age-related Macular Degeneration market analysis, which studies the Gene Therapy for Age-related Macular Degeneration industry coverage, current market competitive status, and market outlook and forecast by 2025.

Gene Therapy for Age-related Macular Degeneration Market 2020-2025 Research Report categorizes the Gene Therapy for Age-related Macular Degeneration market by key players, product type, applications and regions, etc. The report also covers the latest industry data, key players analysis, market share, growth rate, opportunities and trends, investment strategy for your reference in analyzing the Gene Therapy for Age-related Macular Degeneration market.

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According to this latest study, the 2020 growth of Gene Therapy for Age-related Macular Degeneration will have significant change from the previous year. By the most conservative estimates of Gene Therapy for Age-related Macular Degeneration market size (most likely outcome) will be a year-over-year revenue growth rate of XX% in 2020, from US$ xx million in 2019. We give this scenario a XX% probability, where under the scenario the supply chain will start to recover and quarantines and travel bans will ease, over the Q2. Longer-term, the effect of COVID-19 will be felt throughout the year with some degree of harm done by the virus. Over the next five years the Gene Therapy for Age-related Macular Degeneration market will register a XX% CAGR in terms of revenue, the global market size will reach US$ xx million by 2025.

Many companies are operating in the market and conduct their businesses through joint ventures, which benefit the overall market. The Key Players Analysis for the industry is presented in this report.

Key market players

Major competitors identified in this market include RetroSense Therapeutics, REGENXBIO, AGTC, etc.

Based on the Region:

Asia-Pacific (China, Japan, South Korea, India and ASEAN)

North America (US and Canada)

Europe (Germany, France, UK and Italy)

Rest of World (Latin America, Middle East & Africa)

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This study especially analyses the impact of Covid-19 outbreak on the Gene Therapy for Age-related Macular Degeneration , covering the supply chain analysis, impact assessment to the Gene Therapy for Age-related Macular Degeneration market size growth rate in several scenarios, and the measures to be undertaken by Gene Therapy for Age-related Macular Degeneration companies in response to the COVID-19 epidemic.

In addition, this report discusses the key drivers influencing market growth, opportunities, the challenges, and the risks faced by key manufacturers and the market as a whole. It also analyzes key emerging trends and their impact on present and future development.

Based on the Type:

Subretinal

Intravitreal

Based on the Application:

Monotherapy

Combination Therapy

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Beathan Report,

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About Us

At Beathan Report, we understand that the research we provide is only as good as the outcome it inspires. These reports are generated by well-renowned publishers on the basis of the data acquired from an extensive research and credible business statistics. Thats why we are proud to provide the widest range of research products, multilingual 24/7 customer support and dedicated custom research services to deliver the insights you need to achieve your goals. Take a look at few of our aspects that makes Beathan Report an asset to your business.

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Gene Therapy for Age-related Macular Degeneration Market Analysis 2020 Growth by Top Companies and Trends by Types, Treatment, Diagnosis and...

Recommendation and review posted by Bethany Smith

Oct. 28, 2020 05:00 UTC

LONDON--(BUSINESS WIRE)-- Gyroscope Therapeutics Limited, a clinical-stage retinal gene therapy company, today announced that the first surgery using its proprietary Orbit Subretinal Delivery System (Orbit SDS) to deliver the companys investigational gene therapy, GT005, to a patient with geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD) has been conducted in the ongoing Phase I/II FOCUS trial. The surgery was conducted in the United States by Jeffrey Heier, M.D., at Ophthalmic Consultants of Boston.

Dry AMD is one of the leading causes of vision loss globally and gene therapies hold tremendous promise for this disease. Delivering gene therapy directly to the site of disease through a subretinal injection is a proven approach; however, the current method requires a vitrectomy. The Orbit SDS is an innovative delivery system that could address some of the challenges surgeons and patients may experience with a vitrectomy. I have been involved with the development of the Orbit SDS for several years, and I am excited to see its introduction into subretinal delivery of what we hope to be an important therapeutic intervention, said Dr. Heier, Co-President, Medical Director, Director of the Vitreoretinal Service, and Director of Retina Research at Ophthalmic Consultants of Boston.

In 2019, Gyroscope merged with Orbit Biomedical to create the only retinal gene therapy company that combines all elements of drug development, a manufacturing platform, and surgical delivery capabilities. Our vision is to develop gene therapy beyond rare disease and we believe our proprietary Orbit SDS has the potential to help us achieve that. We look forward to further evaluating the Orbit SDS in our GT005 clinical programme, said Khurem Farooq, Chief Executive Officer, Gyroscope.

About FOCUS

FOCUS [NCT03846193] is a Phase I/II open-label clinical trial that was initiated in January 2019. The trial is evaluating the safety and dose response of GT005 in people with GA secondary to dry AMD. Three doses of GT005 are being evaluated in FOCUS. In the initial dose-escalation phase of FOCUS, which is now complete, patients were treated using a subretinal injection involving vitrectomy and retinotomy. The trial has been expanded to include additional cohorts, including a dose-expansion cohort and two cohorts in which the Orbit SDS will be used to deliver GT005.

Gyroscope plans to enrol approximately 45 people who have a clinical diagnosis of GA secondary to dry AMD in FOCUS at sites in the United Kingdom and United States.

About Orbit SDS

The Orbit SDS delivers a controlled volume beneath the retina, with the aim of providing precise and consistent dosing. The subretinal space is accessed by advancing a flexible cannula through the suprachoroidal space (the space between the sclera and the choroid). A microneedle inside the cannula is then advanced to deliver a targeted dose beneath the retina. The microinjection procedure is designed to avoid damaging the structure of the eye by eliminating the need for both a vitrectomy (a procedure that involves removing the vitreous the gel-like substance that fills the eye) and a retinotomy (a hole in the retina).

The Orbit SDS is 510(k) cleared for microinjection into the subretinal space at the back of the eye using saline solution (BSS or BSS PLUS*).

About Dry AMD

Dry AMD is a leading cause of permanent vision loss in people over the age of 50, and is a devastating diagnosis. This gradual and permanent loss of central vision can severely impact a persons daily life as they lose the ability to drive, read and even see the faces of loved ones. There are currently no approved treatments for dry AMD.

Gyroscope estimates that nearly 3.5 million people in the United States and EU5 European countries have GA, and that more than 100,000 people with GA have certain Complement Factor I (CFI) mutations that correlate with low CFI levels in the blood and a higher risk of developing AMD.

About Gyroscope: Vision for Life

Gyroscope Therapeutics is a clinical-stage retinal gene therapy company developing gene therapy beyond rare disease to treat a leading cause of blindness, dry AMD. Our lead investigational gene therapy, GT005, is a one-time therapy delivered under the retina. GT005 is designed to restore balance to an overactive complement system by increasing production of the Complement Factor I protein. GT005 is currently being evaluated in a Phase I/II clinical trial called FOCUS and a Phase II clinical trial called EXPLORE.

Syncona Ltd, our lead investor, helped us create the only retinal gene therapy company to combine discovery, research, drug development, a manufacturing platform and surgical delivery capabilities. Headquartered in London with locations in Philadelphia and San Francisco, our mission is to preserve sight and fight the devastating impact of blindness. For more information visit: http://www.gyroscopetx.com and follow us on Twitter (@GyroscopeTx) and on LinkedIn.

*Balanced Salt Solution; BSS PLUS is a registered trademark of Alcon Laboratories, Inc.

View source version on businesswire.com: https://www.businesswire.com/news/home/20201027006180/en/

The rest is here:
Gyroscope Therapeutics Announces First Patient Received Investigational Gene Therapy GT005 Via Orbit Subretinal Delivery System in Ongoing Phase I/II...

Recommendation and review posted by Bethany Smith

Global Cell Banking Outsourcing Market: Overview

The global cell banking outsourcing market is likely to be driven by the rising demand for biopharmaceutical production targeting novel active sites, stem cell therapy, and gene therapy. A cell bank is a facility storing cells extracted from various organ tissue and body fluids so as to cater to the needs of the future. The cell banks make storage of cells with an elaborate characterization of the entire cell line as it reduces the possibilities of cross contamination. These benefits are estimated to fuel expansion of the global cell banking outsourcing market over the timeframe of assessment, from 2020 to 2030.

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Cell banking outsourcing industries engage testing, characterization, storage, and collection of tissues, cell lines, and the cells. These activities are done to assist in the production of biopharmaceuticals and in the research and development activities so as to ensure minimum adverse effects and high effectiveness. The procedure of the cell storage involves first proliferation of cells, which then multiplies in a huge number of identical cells and is then put inside cryovials safety for use in future. Cells are primarily utilized in the production of regenerative medicine. A surge in the number of cell banks together with the high demand for stem cell therapies is likely to work in favor of the global cell banking outsourcing market over the tenure of analysis, from 2020 to2030.

The global cell banking outsourcing market has been segmented on the basis of four important parameters, which are bank type, phase, cell type, and region.

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Global Cell Banking Outsourcing Market: Notable Developments

The global cell banking outsourcing market is considered a fairly competitive market and is marked with the presence of many leading market players. The companies in this market are forging mergers, partnerships, and collaborations so as to gain larger revenue and market share. The following development is expected to play an important role in the market:

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Global Cell Banking Outsourcing Market: Key Trends

The global cell banking outsourcing market is characterized by the presence of the following restraints, drivers, and opportunities.

High Demand for Stem Cell Therapies to Trigger Growth of the Market

The rising number of stem cell therapies across the globe primarily influences the global cell banking outsourcing market. According to a survey conducted by World Network for Blood and Marrow Transplantation (WBMN), nearly 1 million hematopoietic stem cell transplantation processes were conducted in between 2006 to 2014. These figure comprised removal of stem cells procedures from peripheral blood or bone marrow, proliferating, and then finally storing them cell banks for future use by patients. Stem cell therapies are able to multiple disease, such as amyotrophic lateral sclerosis, type 1 diabetes, cancer, Alzheimer's disease, Parkinson's disease, and so on. Ability to cure such a wide variety of diseases is expected to propel growth of the global cell banking outsourcing market in the years to come.

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Global Cell Banking Outsourcing Market: Geographical Analysis

North America is expected to dominate the global cell banking outsourcing market throughout the timeframe of analysis, from 2020 to 2030. Such high growth of the North America market is ascribed to the increased production of antibiotics, therapeutics protein, and vaccines. In addition, presence of several biopharmaceutical companies in the region is anticipated to foster growth of the cell banking outsourcing market in North America in the near future.

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Cell Banking Outsourcing Market: High Demand for Stem Cell Therapies to Trigger Growth of the Market - BioSpace

Recommendation and review posted by Bethany Smith

NEW YORK--(BUSINESS WIRE)--Nov 2, 2020--

IVERIC bio, Inc. (Nasdaq: ISEE) today announced financial and operating results for the fiscal quarter ended September 30, 2020 and provided a general business update.

It has been a landmark year as we have achieved several major milestones for Zimura, stated Glenn P. Sblendorio, Chief Executive Officer and President of IVERIC bio. Over the past year, we announced positive 12-month and 18-month results from our GATHER1 Phase 3 clinical trial of Zimura for the treatment of geographic atrophy secondary to age-related macular degeneration. We initiated patient enrollment in GATHER2, our second Phase 3 clinical trial for the treatment of GA secondary to AMD. Additionally, we strengthened our balance sheet, advanced our two lead gene therapy product candidates to be on track to enter into the clinic next year and identified a lead compound for our HtrA1 inhibitor program.

Pravin U. Dugel, M.D., Chief Strategy and Business Officer of IVERIC bio added, We are excited about the momentum generated with Zimura as a potential treatment for GA and, based on scientific data, Zimuras potential impact in wet (neovascular) AMD and in earlier stages of dry AMD. We are also encouraged by our HtrA1 inhibitor, IC-500, which we believe could be another important target in the treatment of AMD. Looking forward, our goal is to expand our footprint in multiple stages and types of AMD, dry and wet. We intend to do this by potentially studying Zimura in additional indications and by advancing the development of IC-500. We expect to make great strides in 2021, as we continue to move our pipeline of therapeutics and gene therapy product candidates forward.

Therapeutics Programs Targeting Age-Related Macular Degeneration

The GATHER1 data were recently presented at the following virtual conferences:

IC-500: HtrA1 (high temperature requirement A serine peptidase 1 protein) Inhibitor Program

Gene Therapy Programs in Orphan Inherited Retinal Diseases (IRDs)

Corporate Update

In July 2020, Mark S. Blumenkranz, M.D., M.M.S., joined the Companys board of directors. Dr. Blumenkranz is a biotechnology industry leader and internationally known Vitreo-Retinal Specialist with notable expertise in pharmaceuticals for age-related macular degeneration and ocular gene therapy.

Third Quarter 2020 Operational Update and Cash Guidance

As of September 30, 2020, the Company had $231.1 million in cash, cash equivalents and available for sale securities. The Company has revised its estimated year-end 2020 cash, cash equivalents and available for sale securities to range between $210 million and $215 million, reflecting the impact of a fourth quarter $6.0 million milestone payment to Archemix Corp. The Company also estimates that its cash, cash equivalents and available for sale securities will be sufficient to fund its planned capital expenditure requirements and operating expenses, excluding any potential approval or sales milestones payable to Archemix Corp. or any commercialization expenses for Zimura, through at least mid-2024. These estimates are based on the Companys current business plan, including the continuation of its ongoing clinical development programs for Zimura, the progression of its IC-100 and IC-200 programs into the clinic, and the advancement of its IC-500 development program. These estimates also assume that the Company will enroll approximately 400 patients in the GATHER2 trial. These estimates do not reflect any additional expenditures related to potentially studying Zimura in other indications or resulting from the potential in-licensing or acquisition of additional product candidates or technologies or commencement of new sponsored research programs, and any associated development the Company may pursue.

2020 Q3 Financial Highlights

Conference Call/Web Cast Information

IVERIC bio will host a conference call/webcast to discuss the Companys financial and operating results and provide a business update. The call is scheduled for November 2, 2020 at 8:00 a.m. Eastern Time. To participate in this conference call, dial 866-575-6539 (USA) or 323-794-2575 (International), passcode 6339331. A live, listen-only audio webcast of the conference call can be accessed on the Investors section of the IVERIC bio website at http://www.ivericbio.com. A replay will be available approximately two hours following the live call for two weeks. The replay number is 888-203-1112 (USA), passcode 6339331.

About IVERIC bio

IVERIC bio is a science-driven biopharmaceutical company focused on the discovery and development of novel treatment options for retinal diseases with significant unmet medical needs. The Company is currently developing both therapeutic product candidates for age-related retinal diseases and gene therapy product candidates for orphan inherited retinal diseases. Vision is Our Mission. For more information on the Company, please visit http://www.ivericbio.com.

Website Disclaimer

We have included website addresses in this press release solely as inactive references. The information contained on, or that can be accessed through, such websites is not a part of this press release.

Forward-looking Statements

Any statements in this press release about the Companys future expectations, plans and prospects constitute forward-looking statements for purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. Forward-looking statements include any statements about the Companys strategy, future operations and future expectations and plans and prospects for the Company, and any other statements containing the words anticipate, believe, estimate, expect, intend, goal, may, might, plan, predict, project, seek, target, potential, will, would, could, should, continue, and similar expressions. In this press release, the Companys forward looking statements include statements about its expectations regarding patient enrollment and patient retention in its second Phase 3 trial (GATHER2) of Zimura in geographic atrophy secondary to AMD and to use the results of its completed clinical trial of Zimura for the treatment of geographic atrophy secondary to AMD (GATHER1) as a Phase 3 trial, its development and regulatory strategy for Zimura and its other product candidates, including additional indications that the Company may pursue for the development of Zimura, the implementation of its business plan, its expectations regarding expected cash, cash equivalents and available for sale securities and the sufficiency of its cash resources, the timing, progress and results of clinical trials and other research and development activities and regulatory submissions, the potential utility of its product candidates, and the potential for its business development strategy. Such forward-looking statements involve substantial risks and uncertainties that could cause the Companys development programs, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, those related to the progression and duration of the COVID-19 pandemic and responsive measures thereto and related effects on the Companys research and development programs, operations and financial position, the initiation and the progress of research and development programs and clinical trials, availability of data from these programs, reliance on contract development and manufacturing organizations, university collaborators and other third parties, establishment of manufacturing capabilities, expectations for regulatory matters, need for additional financing and negotiation and consummation of business development transactions and other factors discussed in the Risk Factors section contained in the quarterly and annual reports that the Company files with the Securities and Exchange Commission. Any forward-looking statements represent the Companys views only as of the date of this press release. The Company anticipates that subsequent events and developments will cause its views to change. While the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so except as required by law.

Three Months Ended September 30,

Nine Months Ended September 30,

2020

2019

2020

2019

$

18,841

$

10,383

$

45,311

$

28,077

6,643

4,674

17,930

15,353

25,484

15,057

63,241

43,430

(25,484

)

(15,057

)

(63,241

)

(43,430

)

33

495

437

1,782

Here is the original post:
IVERIC bio Reports Third Quarter 2020 Operational Highlights and Financial Results - The Baytown Sun

Recommendation and review posted by Bethany Smith

Newswise Mass Eye and Ear is proud to announce the successful conclusion of its historic campaign, Bold Science. Life-Changing Cures. which raised $252M from philanthropy to advance research to treat and cure diseases of vision, hearing, and the head and neck.

The campaign was led by co-chair Wyc Grousbeck, Boston Celtics CEO and Lead Owner and former Chairman of Mass Eye and Ear. Grousbeck was elected Chairman of the Foundation and Board of Directors of the Massachusetts Eye and Ear in 2010, and stepped down from the position earlier this year. He will continue to serve as a trustee and contribute to advancing Mass Eye and Ears mission, with a lasting legacy that includes two endowed chairs and a gene therapy research center he and his family established.

The campaigns finale culminated in a virtual celebration as part of the hospitals annual Sense-ation! Gala on Wednesday, October 28, 2020 at 7:30 PM EST. This years gala was held online due to the COVID-19 pandemic, and was hosted by longtime Mass Eye and Ear friends, Billy Costa, (Co-Host of KISS 108s Matty in the Morning and NESNs Dining Playbook) and founding gala chair Joey McIntrye (New Kids on the Block).

[Watch the entire 11th Annual Sense-ation Gala!]

Bold Science. Life-Changing Cures. Ends with Enduring Legacy

The Bold Science. Life-Changing Cures. campaign was launched with the goal of expediting cures for blindness, deafness and diseases of the head and neck by investing in three key areas: people, programs and places (our facilities). Through this campaign, the generosity of donors and organizations infused significant financial resources into Mass Eye and Ears cutting-edge research programs, and supported the facilities needed by the leading physicians and scientists in Ophthalmology and Otolaryngology-Head and Neck Surgery to conduct their world-class work.

The campaign resulted in a powerful and far-reaching impact on research at Mass Eye and Ear by faculty, including:

Additionally, new Research Centers were launched in gene therapy, tinnitus, and head and neck cancer and two world-class surgical training laboratories were built.

The campaign also will leave a lasting legacy of fourteen endowed Chairs for leading physicians and scientists that will remain for generations. The endowment of a Chair provides financial support for researchers, allowing them to focus on their efforts to develop treatments and cures.

Wyc Grousbeck Honored for Dedication to Mass Eye and Ear During 10 years as Chairman

Grousbeck, who served as Board Chairman at Mass Eye and Ear for the past decade, was honored at this years Sense-ation! Gala for his many contributions. During his tenure, he led the organization through remarkable growth, both clinically and as a world-renowned research center. Mass Eye and Ear surgical volume doubled and the number of clinical locations grew from 9 to 21 throughout the region. Grousbecks leadership played critical roles in two highly successful mergers: Schepens Eye Research Institute joining Mass Eye and Ear in 2012 and Mass Eye and Ear joining Mass General Brigham (formerly Partners HealthCare) in 2018. He also initiated and served as co-chair of the Bold Science. Life-Changing Cures. campaign.

Wycs leadership during the campaign has been nothing short of magnificent; he has showed us anything is possible, said John Fernandez, President of Mass Eye and Ear. When we first reached our $100M goal in the campaign, Wyc called on us to double that amount by 2020. With Wycs guidance, we were able to exceed that ambitious goal by more than $50M by the campaigns end.

Grousbeck first became involved with Mass Eye and Ear because his family has been touched by blindness, and since then, they have been devoted to research and educational initiatives relating to blindness and other vision-related conditions for many years.

Grousbeck and his familys philanthropic support established the Grousbeck Center for Gene Therapy at Mass Eye and Ear as well as the Grousbeck Family Chair in Gene Therapy, held by Luk H. Vandenberghe, PhD. Additionally, Wyc and his wife, Emilia Fazzalari, funded the Fazzalari-Grousbeck Chair in Otolaryngology-Head and Neck Surgery held by Tessa Hadlock, MD. Most recently, they seeded Dr. Vandenberghes work to develop an experimental and novel genetic vaccine for COVID-19.

Grousbeck was a driving force around establishing the first Sense-ation Gala 11 years ago, which brought celebrities, musicians, philanthropists, and scientists together to help raise funds for Mass Eye and Ear research and patient care. The gala to date has raised more than $12M for the Mass Eye and Ear Curing Kids Fund.

About Mass Eye and Ear

Massachusetts Eye and Ear, founded in 1824, is an international center for treatment and research and a teaching hospital of Harvard Medical School. A member ofMass General Brigham, Mass Eye and Ear specializes in ophthalmology (eye care) and otolaryngologyhead and neck surgery (ear, nose and throat care). Mass Eye and Ear clinicians provide care ranging from the routine to the very complex. Also home to the world's largest community of hearing and vision researchers, Mass Eye and Ear scientists are driven by a mission to discover the basic biology underlying conditions affecting the eyes, ears, nose, throat, head and neck and to develop new treatments and cures. In the 20202021 Best Hospitals Survey,U.S. News & World Reportranked Mass Eye and Ear #4 in the nation for eye care and #6 for ear, nose and throat care.For more information about life-changing care and research at Mass Eye and Ear, visit our blog,Focus, and follow us onInstagram,TwitterandFacebook.

More:
Mass Eye and Ear Capital Campaign Concludes with $252 Million Raised to Advance Research and Patient Care - Newswise

Recommendation and review posted by Bethany Smith

Analysis and estimation of important industry trends, market size, and market share are covered in the report. Global Precision Cancer Therapies market research report helps clients understand various drivers and restraints impacting the market during the forecast period. The market report comprises of data that can be pretty indispensable when it is about dominating the market or making a mark in the market as a latest emergent. Thus, the study of Precision Cancer Therapies report helps businesses to define their own strategies about the development in the existing product, modifications to consider for the future product, sales, marketing, promotion and distribution of the product in the existing and the new market.

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The overviews, SWOT analysis and strategies of each vendor in the Precision Cancer Therapies market provide understanding about the market forces and how those can be exploited to create future opportunities.

The following players are covered in this report:

Abbott Laboratories

Bayer HealthCare

GlaxoSmithKline

OncoGenex Pharmaceuticals

Hospira

Boehringer Ingelheim

AstraZeneca

Aveo Pharmaceuticals

Precision Cancer Therapies

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Scope of the Precision Cancer Therapies Market Report

The research study analyses the global KEYWORD industry from 360-degree analysis of the market thoroughly delivering insights into the market for better business decisions, considering multiple aspects some of which are listed below as:

Breakdown Data by Type

Hormone Therapy

Immunotherapies

Targeted Therapy

Monoclonal Antibody Therapy

Gene Therapy

Precision Cancer Therapies Breakdown Data by Application

Hospitals

Diagnostic Centers

Oncology Clinics

Research Institutes

Recent Developments

o Market Overview and growth analysis

o Import and Export Overview

o Volume Analysis

o Current Market Trends and Future Outlook

o Market Opportunistic and Attractive Investment Segment

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Key Questions Answered by Precision Cancer Therapies Market Report1. What was the Precision Cancer Therapies Market size in 2019 and 2020; what are the estimated growth trends and market forecast (2020-2025).2. What will be the CAGR of Precision Cancer Therapies Market during the forecast period (2020-2025)?3. Which segments (product type/applications/end-user) were most attractive for investments in 2018? How these segments are expected to grow during the forecast period (2020-2025).4. Which manufacturer/vendor/players in the Precision Cancer Therapies Market was the market leader in 2018?5. Overview on the existing product portfolio, products in the pipeline, and strategic initiatives taken by key vendors in the market.

Contact Us

Beathan Report,

4004 W Lake Sammamish,

Pkway B9 Redmond,

WA 98052 United States.

Tel: +44 115 888 3028

Web: http://www.beathanreports.com

About Us

At Beathan Report, we understand that the research we provide is only as good as the outcome it inspires. These reports are generated by well-renowned publishers on the basis of the data acquired from an extensive research and credible business statistics. Thats why we are proud to provide the widest range of research products, multilingual 24/7 customer support and dedicated custom research services to deliver the insights you need to achieve your goals. Take a look at few of our aspects that makes Beathan Report an asset to your business.

Originally posted here:
Precision Cancer Therapies Market Applications Insights by 2029 - Eurowire

Recommendation and review posted by Bethany Smith

PARIS, Nov. 2, 2020 /PRNewswire/ -- Corlieve Therapeutics today announced the closing of its seed financing led by Kurma Partners and IDinvest Partners, together with Pureos Bioventures. Corlieve is focused on developing novel therapeutics for severe neurological disorders.

Corlieve's lead program employs a novel AAV gene therapy approach for the treatment of refractory temporal lobe epilepsy (TLE), the most commonly diagnosed focal epilepsy in humans. The program is based on pioneering science from INSERM, CNRS, Aix Marseille University and the University of Bordeaux, and is being developed in collaboration with REGENXBIO Inc. (Maryland, US), a leading gene therapy company. In addition, Corlieve has licensed REGENXBIO's NAV AAV9 technology for the TLE program. Corlieve is led by Chief Executive Officer Richard Porter, Ph.D., who brings to the Company over 25 years of neuroscience leadership experience in the pharmaceutical and biotech industries, including most recently as Chief Operating Officer of Therachon Holding AG until its acquisition by Pfizer in July 2019.

"I am excited to build Corlieve on a strong scientific foundation and with valuable support from our partners and investors," said Dr. Porter. "Our novel approach allows us to bring potentially game-changing treatments to patients with neurological diseases."

Corlieve's board will consist of Vanessa Malier, Managing Partner at Kurma, Anja Harmeier, Partner at Pureos Bioventures and Olivier Danos, Ph.D., Chief Scientific Officer of REGENXBIO.

"The formation of Corlieve with REGENXBIO is another validation of our approach to build companies on the basis of leading French science combined with a strong technology partner. We at Kurma are thrilled to support this emerging science to tackle TLE," said Ms. Malier.

"Our mission at REGENXBIO is to develop and enable the use of our proprietary NAV Technology Platform to advance new gene therapies for diseases with significant unmet needs," said Dr. Danos. "We are pleased to collaborate with Corlieve in this application of our NAV AAV9 technology to potentially treat patients suffering from TLE, for which better treatment modalities are needed."

About Corlieve TherapeuticsCorlieve Therapeutics is a biotechnology company focused on bringing novel therapeutic options to patients with severe neurological disorders. The lead project is targeting aberrantly expressed kainate receptors in the hippocampus of patients with TLE using a gene therapy approach.

About KurmaKurma Partners is a key European player in the financing of Innovation in Healthcare and Biotechnology, from pre-seed to growth capital, notably through Kurma Biofund I through III and Kurma Diagnostics, as well as via strategic partnerships with prestigious European research and medical institutions.

About IDinvestIdinvest Partners is a leading European mid-market private equity firm. With 8bn under management, the firm has developed several areas of expertise including innovative start-up venture capital transactions; mid-market private debt, i.e. single-tranche, senior and subordinated debt; primary and secondary investment and private equity advisory services.

About Pureos BioventuresPureos Bioventures is a newly formed venture capital fund, advised by Bellevue Asset Management. Pureos invests exclusively in private innovative drug development companies, with a special emphasis on the next generation of biological drugs and drug modalities. The fund's portfolio companies are built on scientific excellence to develop therapies across a broad indication spectrum including oncology, immunology, ophthalmology, rare diseases and neuroscience. Pureos has built a team with deep investment, operating and clinical expertise, that strives to impact patients' lives by advancing innovative treatments for devastating diseases.

About REGENXBIOREGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates in multiple therapeutic areas.

Contact: Amy Conrad, 858-366-3243, amy@juniper-point.com

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Corlieve Therapeutics SAS Closes Seed Financing to Develop Therapies for Severe Neurological Conditions - BioSpace

Recommendation and review posted by Bethany Smith

Los Angeles, United States- The global Cancer Gene Therapy market is carefully researched in the report while largely concentrating on top players and their business tactics, geographical expansion, market segments, competitive landscape, manufacturing, and pricing and cost structures. Each section of the research study is specially prepared to explore key aspects of the global Cancer Gene Therapy market. For instance, the market dynamics section digs deep into the drivers, restraints, trends, and opportunities of the global Cancer Gene Therapy Market.With qualitative and quantitative analysis, we help you with thorough and comprehensive research on the global Cancer Gene Therapy market. We have also focused on SWOT, PESTLE, and Porters Five Forces analyses of the global Cancer Gene Therapy market.

Leading players of the global Cancer Gene Therapy market are analyzed taking into account their market share, recent developments, new product launches, partnerships, mergers or acquisitions, and markets served. We also provide an exhaustive analysis of their product portfolios to explore the products and applications they concentrate on when operating in the global Cancer Gene Therapy market. Furthermore, the report offers two separate market forecasts one for the production side and another for the consumption side of the global Cancer Gene Therapy market. It also provides useful recommendations for new as well as established players of the global Cancer Gene Therapy market.

Cancer Gene Therapy Market Leading Players: Adaptimmune, GlaxoSmithKline, Bluebird bio, Merck, Celgene, Shanghai Sunway Biotech, BioCancell, Shenzhen SiBiono GeneTech, SynerGene Therapeutics, OncoGenex Pharmaceuticals, Genelux Corporation, Cell Genesys, Advantagene, GenVec, BioCancell, Celgene, Epeius Biotechnologies, Introgen Therapeutics, Ziopharm Oncology, Shenzhen SiBiono GeneTech, Altor Bioscience

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Summary

As the world continues to deal with COVID-19, economies are moving into recession, under multiple adverse factors, the GDP of European and American countries in the second quarter suffered a historical contraction. At an annualized rate, the US GDP fell by 32.9% month on month, while the overall GDP of the euro zone fell by 12.1%.

Moreover, the economic prospects of Europe and the United States in the third quarter under the epidemic situation are hardly optimistic. The resumption of work and production not only brought economic data back, but also triggered a rebound in the epidemic situation. At present, the United States is still the epicenter of the global epidemic. The total number of confirmed cases has exceeded 4.8 million, and the epidemic situation in some European countries has also rebounded. Affected by this, more than 20 states in the United States have announced the suspension or withdrawal of part of the economic restart plan. Britain and Italy have also decided to extend the state of emergency. The rebound of the epidemic situation has posed considerable risks to the economic prospects of Europe and the United States.In the second quarter of this year, US GDP shrank by 9.5% on a month on month basis, or 32.9% at an annual rate, the largest decline since the 1940s. Data show that the sharp decline in personal consumption is the main drag on the U.S. GDP growth in the second quarter.Compared with the United States, Europes economic contraction in the second quarter was smaller, but it was also the lowest on record, with Germany and France contracting more than 10%. According to the data released by the Federal Bureau of statistics, Germanys GDP fell by 10.1% in the second quarter after adjusting for prices, seasons and working days, the largest decline since the quarterly economic data were available in 1970.Thanks to the effective control and policy support of the new epidemic, Chinas economy rebounded sharply in the second quarter. The growth rate of manufacturing industry, which accounted for about 28% of GDP, rebounded sharply to 4.4% from the negative value in the first quarter. Chinese original equipment manufacturers (OEMs) and suppliers are ramping up production. And there are increased investments in digital footprints in manufacturing. OEMs in other parts of the world are offering incentives to drive sales. ReportHive published a report for global Cancer Gene Therapy market in this environment.

In terms of revenue, this research report indicated that the global Cancer Gene Therapy market was valued at USD XXX million in 2019, and it is expected to reach a value of USD XXX million by 2026, at a CAGR of XX % over the forecast period 2021-2026. Correspondingly, the forecast analysis of Cancer Gene Therapy industry comprises of China, USA, Japan, India, Korea and South America, with the production and revenue data in each of the sub-segments.

The ????Adaptimmune aims at producing XX Cancer Gene Therapy in 2020, with XX % production to take place in global market, ????GlaxoSmithKline accounts for a volume share of XX %.

Regional Segmentation (Value; Revenue, USD Million, 2015 2026) of Cancer Gene Therapy Market by ReportHive IncludeChinaEUUSAJapanIndiaSoutheast AsiaSouth AmericaCompetitive Analysis; Who are the Major Players in Cancer Gene Therapy Market?AdaptimmuneGlaxoSmithKlineBluebird bioMerckCelgeneShanghai Sunway BiotechBioCancellShenzhen SiBiono GeneTechSynerGene TherapeuticsOncoGenex PharmaceuticalsGenelux CorporationCell GenesysAdvantageneGenVecBioCancellCelgeneEpeius BiotechnologiesIntrogen TherapeuticsZiopharm OncologyShenzhen SiBiono GeneTechAltor BioscienceMajor Type of Cancer Gene Therapy Covered in ReportHive report:Gene Induced ImmunotherapyOncolytic VirotherapyGene TransferApplication Segments Covered in ReportHive MarketHospitalsDiagnostics CentersResearch Institutes

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Highlights of TOC:

Market Overview: In this section, the authors of the report provide an overview of products offered in the global Global Cancer Gene Therapy market, market scope, consumption comparison by application, production growth rate comparison by type, highlights of geographical analysis in Global Cancer Gene Therapy market, and a glimpse of market sizing forecast.

Manufacturing Cost Analysis: It includes manufacturing cost structure analysis, key raw material analysis, Global Cancer Gene Therapy industrial chain analysis, and manufacturing process analysis.

Company Profiling: Here, the analysts have profiled leading players of the global Global Cancer Gene Therapy market on the basis of different factors such as markets served, market share, gross margin, price, production, and revenue.

Analysis by Application: The Global Cancer Gene Therapy report sheds light on the consumption growth rate and consumption market share of all of the applications studied.

Global Cancer Gene Therapy Consumption by Region: Consumption of all regional markets studied in the Global Cancer Gene Therapy report is analysed here. The review period considered is 2014-2019.

Global Cancer Gene Therapy Production by Region: It includes gross margin, production, price, production growth rate, and revenue of all regional markets between 2014 and 2019.

Competition by Manufacturer: It includes production share, revenue share, and average price by manufacturers. Global Cancer Gene Therapy market analysts have also discussed the products, areas served, and production sites of manufacturers and current as well as future competitive situations and trends.

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Adaptimmune, GlaxoSmithKline, Bluebird bio, Merck, Celgene, Shanghai Sunway Biotech, BioCancell, Shenzhen SiBiono GeneTech, SynerGene Therapeutics, OncoGenex Pharmaceuticals, Genelux Corporation, Cell Genesys, Advantagene, GenVec, BioCancell, Celgene, Epeius Biotechnologies, Introgen Therapeutics, Ziopharm Oncology, Shenzhen SiBiono GeneTech, Altor Bioscience, Cancer Gene Therapy, Cancer Gene Therapy Industry, Cancer Gene Therapy Market, Cancer Gene Therapy Market 2020, Cancer Gene Therapy Market analysis, Cancer Gene Therapy Market Analysis in Developed Countries, Cancer Gene Therapy Market by Application, Cancer Gene Therapy Market by Type, Cancer Gene Therapy Market comprehensive analysis, Cancer Gene Therapy Market comprehensive report, Cancer Gene Therapy Market Development, Cancer Gene Therapy Market Evolving Opportunities With Top Industry Experts, Cancer Gene Therapy Market forecast, Cancer Gene Therapy Market Forecast to 2025, Cancer Gene Therapy Market Forecast to 2026, Cancer Gene Therapy Market Forecast to 2027, Cancer Gene Therapy Market Future Innovation, Cancer Gene Therapy Market Future Trends, Cancer Gene Therapy Market Google News, Cancer Gene Therapy Market Growing Demand and Growth Opportunity, Cancer Gene Therapy market growth, Cancer Gene Therapy Market in Asia, Cancer Gene Therapy Market in Australia, Cancer Gene Therapy Market in Canada, Cancer Gene Therapy Market in Europe, Cancer Gene Therapy Market in France, Cancer Gene Therapy Market in Germany, Cancer Gene Therapy Market in Israel, Cancer Gene Therapy Market in Japan, Cancer Gene Therapy Market in Key Countries, Cancer Gene Therapy Market in Korea, Cancer Gene Therapy Market in United Kingdom, Cancer Gene Therapy Market in United States, Cancer Gene Therapy Market insights, Cancer Gene Therapy Market is Booming, Cancer Gene Therapy Market is Emerging Industry in Developing Countries, Cancer Gene Therapy Market Latest Report, Cancer Gene Therapy Market opportunities, Cancer Gene Therapy market report, Cancer Gene Therapy market research, Cancer Gene Therapy Market Research report, Cancer Gene Therapy Market research study, Cancer Gene Therapy Market Rising Trends, Cancer Gene Therapy Market Size in United States, Cancer Gene Therapy market strategy, Cancer Gene Therapy Market SWOT Analysis, Cancer Gene Therapy Market Updates

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Trending Now: Cancer Gene Therapy Market Segmentation, Analysis by Recent Trends, Development & Growth by Regions| Adaptimmune, GlaxoSmithKline,...

Recommendation and review posted by Bethany Smith

Overview

The following research report is a comprehensive study of the newest and most current trends established in the industry with reference to the Cancer Gene Therapy market. It encompasses a concise but enlightening description of the definition of the market, the various kinds of manufacturing methods put to use as well as fundamental applications. Other than this, the report also covers the price margins of the product alongside the risks faced by the manufacturers in the market. In order to understand and get a detailed analysis of the complexities of the global Cancer Gene Therapy market, data experts examine the existing competitive scene as well as the latest industry trends in the key regions. Besides this, it provides an all-inclusive understanding of various dynamics that affect the Cancer Gene Therapy market. Keeping 2020 as the base year, the research report gives meaningful insight into the current market situation during the forecast period that extends until 2027.

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Drivers and Risks

Other than playing a vital role in providing a general understanding of the vital dynamics that shape the Cancer Gene Therapy market, the market report offers valuable insight on the wide range of volume trends as well as the pricing history. Besides, it also investigates market value. In order to obtain a thorough knowledge of the market as a whole, there is a wide range of potential growth factors that are also analyzed. This includes risks, growth factors as well as opportunities.

Regional Description

The Cancer Gene Therapy market does not only take into consideration the analysis and forecast from a global perspective, but it also lays emphasis on a regional level. It makes it seamless to get a more up-close-and-personal perspective in the regions where the market is specifically concentrated. The market report lays emphasis on Europe, Middle East & Africa, Asia-Pacific, North America, and Latin America. These regions are examined by taking into consideration the existing trends as well as several other opportunities. It also takes an outlook that can benefit the market from a long-term point of view.

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Method of Research

While the main motive of the research report is to offer a general analysis of the Cancer Gene Therapy market during the forecast period, the market is studied depending upon a wide array of parameters that make up Porters Five Force Model. Other than this, the data experts make use of the SWOT Analysis. This makes it easy for the report to obtain explicit data points about the Cancer Gene Therapy market. This detailed and thorough analysis of the market makes it easy to recognize and highlight the risks involved, weaknesses that crop up, main strengths involved as well as opportunities that await.

Questions Answered by the Report:

Key Players

While analyzing the key players in this market, the report draws attention to the existing scenario of the competitive landscape of the market. It also takes into account the new and innovative trends that seep into the manufacturing space. Other than this, the report highlights the various key vendors that play a vital role when contributing to the market.

Key players in the Global Cancer Gene Therapy market are Bluebird bio, Inc., Merck, Adaptimmune, GlaxoSmithKline, Anchiano Therapeutics, Shenzhen SiBiono GeneTech, SynerGene Therapeutics, Celgene, Shanghai Sunway Biotech, OncoGenex Pharmaceuticals among other players.

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Data Library Research is a market research company that helps to find its passion for helping brands grow, discover, and transform. As a market research company, we take gratification by providing our clients with a detail insights report and data that will genuinely make a difference to the client business. Our mission is just one and very well defined that we want to help our clients to predict their business environment in the market so that they will able to make strategies and make their decision successful.

Alex Pandit,Senior Manager International Sales and MarketingData Library Research[emailprotected]Ph: +13523530818 (US)www.datalibraryresearch.com

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Cancer Gene Therapy Market: Overview, Opportunities, In-Depth Analysis and Forecasts, Outlook to 2027 Bluebird bio, Inc., Merck, Adaptimmune,...

Recommendation and review posted by Bethany Smith

This report studies the Cell and Gene Therapy for Covid-19 market with Many aspects of the industry such as the market size, market standing, marketplace trends and forecast, the report also provides brief advice of the opponents and the particular growth opportunities with key market drivers. Locate the complete Cell and Gene Therapy for Covid-19 market evaluation segmented by companies, region, type and applications in the document.

New sellers from the market are facing tough competition from Established international vendors as they fight with technological inventions, reliability and quality problems. The report will answer questions regarding the present market developments and the reach of competition, opportunity cost and more.

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The report discusses the various types of options for Cell and Gene Therapy for Covid-19 Market. While the regions considered in the scope of the report include North America, Europe, and assorted others. The analysis also emphasizes on how climbing digital security threats is altering the market situation.

Development policies and strategies are discussed along with Manufacturing processes and cost structures will also be examined. This report also states import/export consumption, supply and demand Statistics, cost, price, revenue and gross margins.

This report concentrates on the international Cell and Gene Therapy for Covid-19 Status, future forecast, growth opportunity, key marketplace and key players. The study objectives are to present the Cell and Gene Therapy to get Covid-19 growth in United States, Europe, China, Japan, Southeast Asia, India, and Central & South America.

key players in this market include:

Amgen Inc.

bluebird bio, Inc.

Dendreon Pharmaceuticals LLC.

Fibrocell Science, Inc.

Human Stem Cells Institute

Kite Pharma, Inc.

Kolon TissueGene, Inc.

Novartis AG

Orchard Therapeutics plc.

Organogenesis Holdings Inc.

Pfizer, Inc.

RENOVA THERAPEUTICS

Shanghai Sunway Biotech Co., Ltd.,

Sibiono GeneTech Co. Ltd.,

Spark Therapeutics, Inc.

Vericel Corporation

ViroMed Co., Ltd.

The Cell and Gene Therapy for Covid-19 market is a comprehensive record Which offers a meticulous summary of the market share, size, trends, demand, product analysis, application analysis, regional outlook, competitive strategies, forecasts, and strategies impacting the Cell and Gene Therapy for Covid-19 Industry. The report contains a thorough analysis of the market competitive landscape, with the help of detailed business profiles, SWOT analysis, project feasibility analysis, and several other details about the key companies working in the market.

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The study aims Of the report are:

Covid-19 in global sector.To examine the International Important players, SWOT analysis, worth and Global market share for best players.

To define, describe and predict the market by type, end use And region.

To analyze and compare the market standing and forecast among International significant regions.

To examine the global key regions market potential and Advantage, challenge and opportunity, restraints and dangers.

To identify Substantial trends and factors driving or Inhibiting the market development.

To analyze the opportunities in the marketplace for stakeholders

To analyze each submarket with respect to Individual growth trend and their participation to the market

To examine competitive developments such as expansions,

To profile the key players and Gradually Examine their growth strategies.

by Type, the market is primarily split into

Rare Diseases

Oncology

Hematology

Cardiovascular

Ophthalmology

Neurology

Other Therapeutic Classes

by Application, this report covers the following segments

Pharmaceutical and Biotechnology Companies

Research and Academic Institutions

Contract Research Organizations (CROs)

Hospital

Others

Global Cell and Gene Therapy market: regional analysis, the major regions covered in the report are:

North America

United States

Canada

Europe

Germany

France

U.K.

Italy

Russia

Nordic

Rest of Europe

Asia-Pacific

China

Japan

South Korea

Southeast Asia

India

Australia

Rest of Asia-Pacific

Latin America

Mexico

Brazil

Middle East & Africa

Turkey

Saudi Arabia

UAE

Rest of Middle East & Africa

The report lists the major players in the regions and their respective market share on the basis of global revenue. It also explains their strategic moves in the past few years, investments in product innovation, and changes in leadership to stay ahead in the competition. This will give the reader an edge over others as a well-informed decision can be made looking at the holistic picture of the market.

The Cell and Gene Therapy key players in this market include:

Amgen Inc.

bluebird bio, Inc.

Dendreon Pharmaceuticals LLC.

Fibrocell Science, Inc.

Human Stem Cells Institute

Kite Pharma, Inc.

Kolon TissueGene, Inc.

Novartis AG

Orchard Therapeutics plc.

Organogenesis Holdings Inc.

Pfizer, Inc.

RENOVA THERAPEUTICS

Shanghai Sunway Biotech Co., Ltd.,

Sibiono GeneTech Co. Ltd.,

Spark Therapeutics, Inc.

Vericel Corporation

ViroMed Co., Ltd.

The Cell and Gene Therapy for Covid-19 market study report entirely Covers the very important statistics of the capacity, production, value, cost/profit, supply/demand import/export, further separated by country and company, and by application/type for the best possible updated information representation in the figures, tables, pie chart, and graphs. These data representations provide predictive data regarding the potential estimations for persuasive market development. The detailed and in depth knowledge about our publishers makes us from the box in the event of market analysis.

Key questions Answered within this report

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Cell and Gene Therapy Market Size, Statistics, Growth, Revenue, Analysis & Trends Industry Forecast Report 2020-2028 - The Think Curiouser

Recommendation and review posted by Bethany Smith

Reportspedia presents most recent industry chain structure and complete analysis of Hemophilia Gene Therapy Market. The forecast Hemophilia Gene Therapy industry analysis is covered in this report. This research study offers critical information on Hemophilia Gene Therapy which will assist the industry players in making informed business move. The Hemophilia Gene Therapy Report is segmented by Application/ End User, Product Type and Geologies.

The market size of Global Hemophilia Gene Therapy Market, development rate and past industry information is provided in this study. The company profiles of top Hemophilia Gene Therapy manufacturers/ players, complete product portfolio is presented in this report. The cost structures, growth rate, and gross margin analysis is covered in this report. Global, regional and country level data is presented in this study. The import-export scenario, production rate, consumption and gross margin analysis from 2015-2019 is covered in this report. Market share by Hemophilia Gene Therapy region in 2018 for top players is analysed in this report.

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Hemophilia Gene Therapy Market analysis and forecast by type, applications and region is analysed in this report. The forecast period is 2020-2026 where market value, volume, consumption forecast is presented. Further, feasibility study, industry barriers, latest plans and policies are explained in this study. The production process analysis, manufacturing cost, Hemophilia Gene Therapy labor cost, marketing channels and manufacturing base is elaborated in this report.

Major players covered in this report:

UltragenyxBioMarinShire PLCuniQureSangamo TherapeuticsBioverativFreeline TherapeuticsSpark Therapeutics

Global Hemophilia Gene Therapy Market Segmentation:

By Type:

Hemophilia AHemophilia B

By Application:

Hemophilia A Gene TherapyHemophilia B Gene Therapy

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The below list highlights the important points considered in Hemophilia Gene Therapy report:

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Contact Us:

Name: Alex White

Phone: US: +1(806)4400782/ UK: +44 33 3303 4979

Email: [emailprotected]

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Global Hemophilia Gene Therapy Market in-Depth Analysis, Key Players, Challenges, Segmentation and Forecasts to 2026 - The Think Curiouser

Recommendation and review posted by Bethany Smith

Merger and acquisition activities have heated up in recent months and the niche spaces are in the limelight. After the telemedicine industry, gene therapy stocks jumped on the bandwagon. Gene therapy is a technique that uses genes to treat or prevent disease.

German drugmaker Bayer has made a big bet on gene therapy by announcing the acquisition of U.S. biotech firm Asklepios BioPharmaceutical for as much as $4 billion. The proposed acquisition will provide Bayer access to the adeno-associated virus (AAV) gene therapy platform and a pipeline led by clinical-phase treatments for Parkinsons, Pompe disease and congestive heart failure. Notably, AAV therapies offer improved efficacy, immune response, and tissue and organ specificity.

Additionally, the transaction will complements Bayers 2019 acquisition of BlueRock Therapeutics and advances its efforts to create platforms with the potential to have an impact on multiple therapeutic areas (read: Genomics ETFs Surge on Nobel Prize for Gene-Editing Pioneers).

Under the terms of the deal, Bayer will pay an upfront consideration of $2 billion and potential success-based milestone payments of up to $ billion. About 75 % of the potential milestone-based contingent payments are expected to be due during the course of the next five years and the remaining amount thereafter.

The deal, pending regulatory approvals, is expected to close during the fourth quarter of 2020. Once the deal closes, Bayer will allow Asklepios, known as AskBio, to operate autonomously as part of a new cell and gene therapy unit in a bid to preserve its entrepreneurial culture. The cell and gene therapy unit will bundle Bayer's activities in this area moving forward in order to establish an innovation ecosystem for the participating partners, the German company said (see: all the Healthcare ETFs here).

The proposed deal will provide a boost to the gene therapy industry. Below, we have highlighted four ETFs that are expected to benefit from Bayers entrance into the gene therapy space:

ARK Genomic Revolution Multi-Sector ETF (ARKG - Free Report)

This actively managed ETF is focused on companies that are likely to benefit from extending and enhancing the quality of human and other life by incorporating technological and scientific developments, and advancements in genomics into their business. With AUM of $2.9 billion, the fund holds 47 stocks in its basket and has 0.75% in expense ratio. It trades in an average daily volume of 978,000 shares (read: 4 Sector ETFs That Have Doubled This Year).

Invesco Dynamic Biotechnology & Genome ETF (PBE - Free Report)

This fund follows the Dynamic Biotech & Genome Intellidex Index and provides exposure to companies engaged in the research, development, manufacture and marketing and distribution of various biotechnological products, services and processes and companies that benefit significantly from scientific and technological advances in biotechnology and genetic engineering and research. It holds 31 stocks in its basket and charges 57 bps in annual fees. The ETF has managed $229.9 million in its asset base while trades in a light volume of 6,000 shares per day. Expense ratio comes in at 0.57%. The product has a Zacks ETF Rank #3 (Hold) with a High risk outlook.

Global X Genomics & Biotechnology ETF (GNOM - Free Report)

This product seeks to invest in companies that potentially stand to benefit from further advances in the field of genomic science, such as companies involved in gene editing, genomic sequencing, genetic medicine/therapy, computational genomics and biotechnology. It follows the Solactive Genomics Index, holding 40 stocks in its basket. This ETF has accumulated $68 million in its asset base and charges 50 bps in annual fees. It trades in average daily volume of 31,000 shares (read: Why You Should Invest in Genomics ETFs).

iShares Genomics Immunology and Healthcare ETF (IDNA - Free Report)

This ETF provides access to companies at the forefront of genomics and immunology innovation by tracking the NYSE FactSet Global Genomics and Immuno Biopharma Index. Holding 46 stocks in its basket, the fund has gathered $166.2 million in AUM and trades in moderate average daily volume of 58,000 shares. It charges 47 bps in annual fees.

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ETFs in Focus on Bayer's Bet on Gene Therapy - Zacks.com

Recommendation and review posted by Bethany Smith

Global Hemophilia Gene Therapy Market research report provides excellent vision to analysis Global as well as regional industry. This research report delivers a detailed analysis of distinguishable strategies for industrial growth that will help to determine commanding segments and know distinct factors. Scope of different segments and applications that can potentially influence the global Hemophilia Gene Therapy market in the future has been analyzed further in the report. The report acknowledges major industry vendors, key regions, demand & supply, applications, innovations, revenue cost, and challenges. The report covers trends, restraints, and drivers that transform the global Hemophilia Gene Therapy market in either a positive or negative manner.

The Hemophilia Gene Therapy Market research report presents a comprehensive analysis of the market and contains thoughtful insights, facts, historical data, and statistically supported and industry-validated market data. It additionally contains projections applying a suitable set of assumptions and methodologies. The research report provides analysis and information according to market segments such as geographies, application, and industry by considering major players.

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Key players in the global Hemophilia Gene Therapy market covered in Chapter 4:, Ultragenyx, BioMarin, Shire PLC, uniQure, Sangamo Therapeutics, Bioverativ, Freeline Therapeutics, Spark Therapeutics

In Chapter 11 and 13.3, on the basis of types, the Hemophilia Gene Therapy market from 2015 to 2026 is primarily split into:, Hemophilia A, Hemophilia B

In Chapter 12 and 13.4, on the basis of applications, the Hemophilia Gene Therapy market from 2015 to 2026 covers:, Hemophilia A Gene Therapy, Hemophilia B Gene Therapy

Geographically, the detailed analysis of consumption, revenue, market share and growth rate, historic and forecast (2015-2026) of the following regions are covered in Chapter 5, 6, 7, 8, 9, 10, 13:, North America (Covered in Chapter 6 and 13), United States, Canada, Mexico, Europe (Covered in Chapter 7 and 13), Germany, UK, France, Italy, Spain, Russia, Others, Asia-Pacific (Covered in Chapter 8 and 13), China, Japan, South Korea, Australia, India, Southeast Asia, Others, Middle East and Africa (Covered in Chapter 9 and 13), Saudi Arabia, UAE, Egypt, Nigeria, South Africa, Others, South America (Covered in Chapter 10 and 13), Brazil, Argentina, Columbia, Chile, Others

Browse the Full Global Hemophilia Gene Therapy Market research Report @https://hongchunresearch.com/report/hemophilia-gene-therapy-market-size-2020-77530

Some Point of Table of Content:

Chapter One: Report Overview

Chapter Two: Global Market Growth Trends

Chapter Three: Value Chain of Hemophilia Gene Therapy Market

Chapter Four: Players Profiles

Chapter Five: Global Hemophilia Gene Therapy Market Analysis by Regions

Chapter Six: North America Hemophilia Gene Therapy Market Analysis by Countries

Chapter Seven: Europe Hemophilia Gene Therapy Market Analysis by Countries

Chapter Eight: Asia-Pacific Hemophilia Gene Therapy Market Analysis by Countries

Chapter Nine: Middle East and Africa Hemophilia Gene Therapy Market Analysis by Countries

Chapter Ten: South America Hemophilia Gene Therapy Market Analysis by Countries

Chapter Eleven: Global Hemophilia Gene Therapy Market Segment by Types

Chapter Twelve: Global Hemophilia Gene Therapy Market Segment by Applications

Chapter Thirteen: Hemophilia Gene Therapy Market Forecast by Regions (2020-2026)

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Originally posted here:
Hemophilia Gene Therapy Market Size 2020, Share, Global Industry Analysis and Competitive Landscape (Effect of the COVID-19 Pandemic) - The Think...

Recommendation and review posted by Bethany Smith

CNS Gene Therapy Market Insights 2018, is a professional and in-depth study on the current state of the global CNS Gene Therapy industry with a focus on the Global market. The report provides key statistics on the market status of the CNS Gene Therapy manufacturers and is a valuable source of guidance and direction for companies and individuals interested in the industry. Overall, the report provides an in-depth insight of 2018-2025 global CNS Gene Therapy market covering all important parameters.

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The key points of the CNS Gene Therapy Market report:

The report provides a basic overview of the CNS Gene Therapy industry including its definition, applications and manufacturing technology.

The report explores the international and Chinese major industry players in detail. In this part, the report presents the company profile, product specifications, capacity, production value, and 2018-2025 market shares for each company.

Through the statistical analysis, the report depicts the global total market of CNS Gene Therapy industry including capacity, production, production value, cost/profit, supply/demand and Chinese import/export.

The total market is further divided by company, by country, and by application/type for the competitive landscape analysis.

The report then estimates 2018-2025 market development trends of CNS Gene Therapy industry. Analysis of upstream raw materials, downstream demand, and current market dynamics is also carried out.

The report makes some important proposals for a new project of CNS Gene Therapy Industry before evaluating its feasibility.

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There are 3 key segments covered in this report: competitor segment, product type segment, end use/application segment.

For competitor segment, the report includes global key players of CNS Gene Therapy are included:

key players and product offerings

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Reasons to Purchase this Report:

* Estimates 2018-2025 CNS Gene Therapy market development trends with the recent trends and SWOT analysis

* Market dynamics scenario, along with growth opportunities of the market in the years to come

* Market segmentation analysis including qualitative and quantitative research incorporating the impact of economic and policy aspects

* Regional and country level analysis integrating the demand and supply forces that are influencing the growth of the market.

* Competitive landscape involving the market share of major players, along with the new projects and strategies adopted by players in the past five years

* Comprehensive company profiles covering the product offerings, key financial information, recent developments, SWOT analysis, and strategies employed by the major market players

Read the original:
High Demand for CNS Gene Therapy from the Millennial Population to Foster the Growth of the CNS Gene Therapy Market between 2018 2028 - The Think...

Recommendation and review posted by Bethany Smith