Archive for the ‘Gene Therapy Research’ Category

BOSTON and LONDON, March 18, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that company founder and gene therapy pioneer Bobby Gaspar, M.D., Ph.D., has been named chief executive officer, effective immediately. Dr. Gaspar, previously president of research, chief scientific officer, and a member of the Orchard board of directors, succeeds Mark Rothera, who has served as the companys chief executive officer since 2017. As part of this transition process, Frank Thomas, Orchards chief operating officer and chief financial officer, will take on the role of president.

As a world-renowned scientist and physician, and accomplished strategic and organizational leader with more than 25 years of experience in medicine and biotechnology, Bobby Gaspar is uniquely qualified to lead Orchard into the future, said Jim Geraghty, chairman of the Orchard board of directors. In addition, Frank Thomas proven track record of success in leading operations, corporate finance and commercialization at a number of publicly traded life sciences companies will continue to be invaluable in his expanded role. On behalf of the entire Board of Directors, Id like to personally thank Mark for his many contributions to building Orchard into a leading gene therapy company over the last three years and wish him all the best in his future endeavors.

One of the companys principal scientific founders, Dr. Gaspar has served on Orchards board of directors and has driven its research, development and regulatory strategy since its inception. Over the course of his long career he has been a leading force in the development of hematopoietic stem cell (HSC) gene therapy bringing it from some of the first studies in patients to potential regulatory approvals. Dr. Gaspars unparalleled expertise, in addition to his deep relationships with key physicians and treatment centers around the world, will continue to be integral to efforts to identify and treat patients with metachromatic leukodystrophy (MLD) and other diseases through targeted disease education, early diagnosis and comprehensive newborn screening.

Dr. Gaspar commented: I am honored to become Orchards next CEO at a time of such opportunity for the company and for patients with severe genetic disorders. Through the consistent execution of our strategy, our talented team has advanced a leading portfolio of gene therapy candidates, expanding our R&D, manufacturing and commercial capabilities. We will now focus on driving continued innovation and growth, as well as strong commercial preparation and execution. I look forward to providing greater detail around our commercialization plan, pipeline prioritization and how we can realize the full potential of our HSC gene therapy platform, in the coming quarter.

Mr. Thomas commented: Im excited to be part of this next phase of Orchards evolution as a gene therapy leader as we look to refine our strategic priorities, ensure financial strength through improved operating efficiencies and prepare for a new cycle of growth, which includes our anticipated upcoming launch of OTL-200 in Europe. Im confident we will achieve long-term growth and value for our shareholders while turning groundbreaking innovation into potentially transformative therapies for patients suffering from devastating, often-fatal inherited diseases.

Mr. Rothera commented: It has been a great privilege to lead Orchard and this outstanding management team for the past three years. Orchard is poised to make a huge difference to the lives of patients worldwide living with devastating rare genetic conditions. Having worked closely with Bobby for the last several years, I know that he is tremendously talented, extremely passionate about the patient-centric mission, and fully prepared to lead Orchard as it enters its next phase as a company.

About OrchardOrchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically-modified blood stem cells and seeks to permanently correct the underlying cause of disease in a single administration. The company has one of the deepest gene therapy pipelines in the industry and is advancing seven clinical-stage programs across multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist, including inherited neurometabolic disorders, primary immune deficiencies and blood disorders.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (twitter.com/orchard_tx and http://www.linkedin.com/company/orchard-therapeutics), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include express or implied statements relating to, among other things, the companys business strategy and goals, and the therapeutic potential of Orchards product candidates, including the product candidate or candidates referred to in this release. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the impact of the COVID-19 virus on Orchards clinical and commercial programs, the risk that any one or more of Orchards product candidates, including the product candidate or candidates referred to in this release, will not be approved, successfully developed or commercialized, the risk of cessation or delay of any of Orchards ongoing or planned clinical trials, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates, the delay of any of Orchards regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates, the receipt of restricted marketing approvals, and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards annual report on Form 10-K for the year ended December 31, 2019, as filed with the U.S. Securities and Exchange Commission (SEC) on February 27, 2020, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

InvestorsRenee T. LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaChristine C. HarrisonVP, Public Affairs & Stakeholder Engagement+1 202-415-0137media@orchard-tx.com

See the original post here:
Orchard Therapeutics Appoints Company Founder and Gene Therapy Pioneer Bobby Gaspar, MD, Ph.D., as New Chief Executive Officer - BioSpace

NEW YORK, March 18, 2020 /PRNewswire/ --

The report on the global zinc finger nuclease technology market provides qualitative and quantitative analysis for the period from 2017 to 2025.

Read the full report: https://www.reportlinker.com/p05874235/?utm_source=PRN

The report predicts the global zinc finger nuclease technology market to grow with a healthy CAGR over the forecast period from 2019-2025. The study on zinc finger nuclease technology market covers the analysis of the leading geographies such as North America, Europe, Asia-Pacific, and RoW for the period of 2017 to 2025.

The report on zinc finger nuclease technology market is a comprehensive study and presentation of drivers, restraints, opportunities, demand factors, market size, forecasts, and trends in the global zinc finger nuclease technology market over the period of 2017 to 2025. Moreover, the report is a collective presentation of primary and secondary research findings.

Porter's five forces model in the report provides insights into the competitive rivalry, supplier and buyer positions in the market and opportunities for the new entrants in the global zinc finger nuclease technology market over the period of 2017 to 2025. Further, IGR- Growth Matrix gave in the report brings an insight into the investment areas that existing or new market players can consider.

Report Findings1) Drivers Rising use of gene therapy and genome therapy Benefits offered by zinc finger nuclease technology such as permanent and heritable mutation and efficient creation of animal models2) Restraints Complexities associated with zinc finger nuclease technology3) Opportunities Application of zinc finger nuclease technology in drug discovery

Research Methodology

A) Primary ResearchOur primary research involves extensive interviews and analysis of the opinions provided by the primary respondents. The primary research starts with identifying and approaching the primary respondents, the primary respondents are approached include1. Key Opinion Leaders associated with Infinium Global Research2. Internal and External subject matter experts3. Professionals and participants from the industry

Our primary research respondents typically include1. Executives working with leading companies in the market under review2. Product/brand/marketing managers3. CXO level executives4. Regional/zonal/ country managers5. Vice President level executives.

B) Secondary ResearchSecondary research involves extensive exploring through the secondary sources of information available in both the public domain and paid sources. At Infinium Global Research, each research study is based on over 500 hours of secondary research accompanied by primary research. The information obtained through the secondary sources is validated through the crosscheck on various data sources.

The secondary sources of the data typically include1. Company reports and publications2. Government/institutional publications3. Trade and associations journals4. Databases such as WTO, OECD, World Bank, and among others.5. Websites and publications by research agencies

Segment CoveredThe global zinc finger nuclease technology market is segmented on the basis of type, and application.

The Global Zinc Finger Nuclease Technology Market by Type Cell Line Engineering Animal Genetic Engineering Plant Genetic Engineering Other

The Global Zinc Finger Nuclease Technology Market by Application Biotechnology Companies Pharmaceutical Companies Hospital Laboratory and Diagnostic Laboratory Academic and Research Institutes

Company Profiles Sigma-Aldrich Corporation Thermo Fisher Scientific Sangamo Therapeutics inc. LabOmics S.A. Gilead Sciences, Inc. OriGene Technologies, Inc Others

What does this report deliver?1. Comprehensive analysis of the global as well as regional markets of the zinc finger nuclease technology market.2. Complete coverage of all the segments in the zinc finger nuclease technology market to analyze the trends, developments in the global market and forecast of market size up to 2025.3. Comprehensive analysis of the companies operating in the global zinc finger nuclease technology market. The company profile includes analysis of product portfolio, revenue, SWOT analysis and latest developments of the company.4. IGR- Growth Matrix presents an analysis of the product segments and geographies that market players should focus to invest, consolidate, expand and/or diversify.

Read the full report: https://www.reportlinker.com/p05874235/?utm_source=PRN

About Reportlinker ReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

__________________________ Contact Clare: [emailprotected] US: (339)-368-6001 Intl: +1 339-368-6001

SOURCE Reportlinker

http://www.reportlinker.com

Read this article:
Global zinc finger nuclease technology market is expected to grow with a healthy CAGR over the forecast period from 2019-2025 - PRNewswire

Barth syndrome is an X-linked metabolic disorder, affecting only males. It has widespread systemic effects presenting with cardiomyopathy, neutropenia, muscle weakness, stunted growth, exercise intolerance and abnormal skeletal structures. In many cases, it results in stillbirth. It is strongly related to mutations in the tafazzin gene, also known as TAZ. Currently only symptomatic treatment exists, and no definite cure has been developed for Barth syndrome.

Read Also: CRISPR Used for the First Time to Treat a Blind Patient

Researchers at Boston Childrens Hospital have proposed gene therapy as a potential treatment method to inhibit processes that lead to heart failure. The researchers conducted the study on mouse models with Barth syndrome.

A research to better understand Barth syndrome was conducted in 2014 by William Pu, MD and colleagues at Boston Childrens Hospital. Together, they created heart on chip models of Barth syndrome by using cardiac myocytes derived from patients with TAZ mutation. This led the researchers to discover the correlation between Barth syndrome and dysfunction. When the defective mutated TAZ myocytes was replaced by healthy TAZ gene myocytes, the cardiac dysfunction was spontaneously corrected.

Pu and colleagues realized that in order to fully understand the effects of Barth syndrome on the system, an animal body was crucial. Attempts at creating a whole body model had previously been done, but had not been successful.

The Beatson Institute for Cancer Research in the U.K has recently been successful in creating mouse models of Barth syndrome. Two categories of these mouse models were created, in the first category TAZ gene was deleted throughout the whole system whereas in the second category of mouse models ha TAZ gene deleted only from the cardiac myocytes.

The mouse models with whole body TAZ deletion died before birth mostly due to hypotonic weak musculature. However, some of the mice survived and developed cardiomyopathy, similar to the dilated cardiomyopathy in humans. The hearts left ventricle had thinner walls and dilated substantially which decreased the systolic pressure resulting in decreased cardiac output.

In those mice with deleted TAZ in heart muscle cells, all subjects survived but had cardiomyopathy issues and reduced cardiac output. Under electron microscope, the heart muscles were found to have abnormal structures and poor organization.

Read Also:UC Berkeley Researchers Restore Vision in Mice Through Gene Insertion

Using gene therapy, the researchers replaced the TAZ gene by administering a gentically engineered virus subcutaneously or intravenously. Whole body TAZ deletion mice survived to an average life span of healthy mice. It successfully prevented cardiac dysfunction in all mice models.

Only when more than 70 percent cardiac myocytes had taken up the modified TAZ gene, significant improvement was seen.

The problem is that neutralizing antibodies to the virus develop after the first dose, said Pu. Getting enough of the muscle cells corrected in humans may be a challenge.

Post introduction of TAZ gene corrected cells, the major problem was seen in sustaining the levels of modified gene cells. In comparison to cardiac myocytes, the number of corrected gene cells in skeletal muscles declined progressively.

https://www.sciencedaily.com/releases/2020/03/200309165231.htm

Doctors Investigate CRISPR Gene-Editing for Treatment of Sickle Cell Disease

Herpes Could Finally Be Cured With CRISPR According to Study

CRISPR Prevents Liver Disease In Mice Before Birth

Study Shows That CRISPR-Edited T Cells for Cancer Treatment Are Safe and Long-Lasting

See the original post:
Correcting Barth Syndrome With Gene Therapy - Gilmore Health News

BETHESDA, Md., March 18, 2020 /PRNewswire/ -- Internationally acclaimed clinical geneticist and pediatrician Harvey Levy, MD, FACMG, is the recipient of the 2020 ACMG Foundation for Genetic and Genomic Medicine's David L. Rimoin Lifetime Achievement Award in Medical Genetics.

Dr. Levy, senior physician in medicine and genetics at Boston Children's Hospital and professor of pediatrics at Harvard Medical School, is being honored for his many years of groundbreaking work with patients who have genetic metabolic diseases including phenylketonuria (PKU), homocystinuria, cobalamin metabolic disorder, and others; as well as for his training and mentoring of the next generation of genetics service providers; and for his major contributions to the development of newborn screening in the United States and around the world.

Dr. Levy's medical career spans more than 60 years. He hasmentored over 60 medical genetics fellows; published more than 400 research articles, reviews, book chapters, and proceedings from research meetings; written 2 books and created 2 educational videos for patients and clinicians; served on editorial boards and as a reviewer for numerous prominent research journals; and founded and formerly directed both the Maternal PKU Program and the Inborn Errors of Metabolism/PKU Program at Boston Children's Hospital.

"Harvey Levy is a physician scientist who has been instrumental in the development of newborn screening programs for metabolic diseases," said former ACMG Executive Director Dr. Michael S. Watson, FACMG. "Of particular importance has been his melding of knowledge of clinical genetics, population genetics and metabolic diseases to identify critical issues in the transition from a disease-based understanding of particular metabolic diseases to a population-based prevention program that has had enormous impact on hundreds of newborns in the United States."

"We take newborn screening for granted now," said Gerard Berry, MD, director of the Metabolism Program and professor of pediatrics at Harvard Medical School. "There are laws in different states that babies need to be screened for certain diseases. But when Harvey began, this was uncharted territory. People didn't understand the power of newborn screening and how it could change lives by allowing someone to get on a diet or a medication that they need to take for life in order to be healthy. Harvey played a major role in allowing all of this to come to fruition. These same individuals, who might have been institutionalized years ago because of severe intellectual disability, are now students in elite colleges. Harvey possesses insight and super-ability to understand what is really important for healthcare. Newborn screening is one of the major healthcare successes of the previous centurymaybe the most important healthcare success. And Harvey was part of a group of unique individuals who helped to see that through."

The news that he had received the David L. Rimoin Lifetime Achievement Award came to Dr. Levy as a delightful surprise. "This means a great deal to me because of the ACMG, where I've been an active member for a number of years," Levy shared. "It's a wonderful organization full of outstanding individuals, so to be in that company is particularly gratifying. And it's very, very nice to be appreciated."

"The Rimoin family is proud to recognize Dr. Harvey L. Levy, whose outstanding work includes studies that formed the basis for newborn metabolic screening, the discovery of the first human vitamin B12defect and the establishment of cobalamin defects, and the development of Maternal PKU programs," said Dr. Ann Garber, David Rimoin's surviving spouse."Based on his scientific accomplishments, along with his remarkable integrity, empathy and collaboration, our family is pleased to honor Dr. Levy with the David L. Rimoin Lifetime Achievement Award."

Beyond his list of academic achievements and leadership positions, the nominations for Dr. Levy to receive this award stressed his abounding generosity of time, knowledge and skill while working with patients, families and the broad range of clinical providers and researchers who have collaborated with him.

"He's dedicated himself to the study of PKU and metabolic disorders with an energy and intellect and soul that is extraordinary," said neuropsychologist Susan Waisbren, PhD, a professor of psychology at Harvard Medical School and Dr. Levy's long-time collaborator at Boston Children's Hospital Metabolism Clinic. "One of the qualities I've always found striking is the respect he has for professions outside of medicine. In his mind, every member of the clinical team is important. He truly feels this and it shows in his academic as well as clinical work. He has included as co-authors psychologists, dieticians, social workers, genetic counselors, nurses, administrators, secretaries, and parents.

"The patients adore him, always," she added, "and they recognize a certain compassion and ability to see the whole person, not just the metabolic disorder."

"Harvey is one of those special individuals who one may encounteronce in a lifetime," said Dr. Levy's collaborator at Boston Children's Hospital, Dr. Berry. "He's much more than an accomplished geneticistand investigator.First and foremost, he's a very endearing individual with a wonderful bedside manner, and he's beloved by patients and families whom he's cared for over the years. Harvey goes out of his way to make things better for patients and their families."

As an example, Dr. Berry, who has knownDr. Levy for several decades, recalled a case around 15 years ago, when a baby had been born with PKU in a suburban hospital outside of Boston. "Without telling anyone, Harvey drove to the hospital just to say hello to the new parents and to see the baby," Dr. Berry recounted. "He didn't need to do that. Everything was already in place, people were already taking care of what needed to be done, but he felt compelled to drive out there on a Friday evening to say hello."

Harvey L. Levy was born in Augusta, Georgia in 1935, the eldest of three sons. His father owned a one-room mercantile that supplied clothing to families of the surrounding area, which comprised mostly farmland during that period. His mother, who was a homemaker, graduated from Hunter College and served as a technician in a research laboratory in New York before her marriage. He credits her with some of his initial interest in research.

"I was a guy who was looking for answers to things, so I was always interested in science. And I particularly liked chemistry," Dr. Levy recalled. "My mother was a very intelligent person and very interested in education and music and arts, and also interested in science. I talked with my mother quite a bit about science. So, I think she had a feeling that maybe it would be a good idea for me to be a doctor."

Dr. Levy began studying history as an undergraduate student at Emory University and then switched to an early admission program at the Medical College of Georgia. One of his medical school professors, the famous Dr. Victor Vaughan, headed the department of pediatrics and had a profound influence on the direction of Dr. Levy's career. "I was always interested in pediatrics because of its developmental aspects," explained Dr. Levy. "I felt that if I was going to do something in terms of disease, preventing or helping patients in a significant way, I had to start early, and the earlier the better."

After completing his medical degree in 1960, Dr. Levy served an internship in pediatrics at the Boston City Hospital under Dr.Sydney Gellis, a renowned teacher of pediatrics. Following the internship he moved to New York and the Columbia-Presbyterian Medical Center, where he spent a year under Dr. Dorothy Anderson, the discoverer of cystic fibrosis. Then, as world events escalated toward the start of the Vietnam War, he was drafted and served 2 years in the Unites States Navy as a medical officer stationed in the Philippines.

His introduction to genetics came when he returned to his medical training in 1964 as a second-year pediatrics resident at Johns Hopkins University, where he met the pioneering pediatric clinical geneticist Dr. Barton Childs. What he learned from Dr. Childs about DNA triggered memories of an earlier time, and brought forth questions that further defined Dr. Levy's future career.

"If I go back to my childhood, my upbringing, I had three cousins from one of my father's brothers, whose family we were very close to, and all of these cousins were developmentally disabled," Dr. Levy said. " No reason was given for their disability and I always thought if I got into genetics, then maybe I could discover the causes of brain disease, particularly intellectual disability, and maybe I could influence the prevention of it."

Dr. Levy returned to Boston, where he served as Chief Resident in Pediatrics back at the Boston City Hospital. During that year he heard a lecture by Dr. Mary Efron, director of the Amino Acid Laboratory at Massachusetts General Hospital, in which she described her studies on metabolic disorders and their enzymatic defects as well as how newborn screening was helping clinicians to identify infants with these disorders so they could receive immediate preventive treatment.

"I became so fascinated with that. It was just absolutely the thing that I really wanted to do," recalled Dr. Levy. "Here was chemistry, biochemistry, genetics, and the prevention of disease! So I asked Dr. Efron if I could do a fellowship with her, which resulted in an NIH-funded fellowship at Massachusetts General Hospital. And that began the journey that has continued to this day."

One cold, fateful Friday afternoon while he was working in Dr. Efron's lab, a telephone call came from Dr. Robert MacCready, director of the Massachusetts Newborn Screening Program. Dr. MacCready asked if someone could come to the screening lab to look at an unusual screening result. Dr. Efron was ill, so Dr. Levy rode his bicycle seven miles across town to the State Laboratory Institute, where he recognized the unusual spot on the paper chromatogram test as a high level of methionine, the hallmark of a genetic disorder he had recently learned about called homocystinuria.

"I called the baby's doctor and asked if I could see the baby at the Massachusetts General Hospital the following Monday," Dr. Levy recalled. "The family and baby came that Monday and I confirmed that the infant indeed had homocystinuria. I asked if they had other children, and was told, 'Yes, we have a daughter.' And I asked if she was ok, and they said she was fine. I asked to see her and she was brought to the next visit, where I immediately recognized that she was developmentally delayed and had other features of homocystinuria that had only recently been described. She was born before screening for homocystinuria had begun. So that launched me into the field of methionine metabolism and some very interesting new areas of research." Much of this research was in collaboration with the late Dr. Harvey Mudd of the NIH, who was the world's foremost authority on methionine and on sulfur amino acid metabolism in general.

Dr. Efron passed away and Dr. Levy assumed Dr. Efron's position as consultant to the Massachusetts Newborn Screening Program and, in 1972, was appointed Director of the program. Four years later, he became Chief of Biochemical Genetics for the New England Newborn Screening Program, a position he held until 1997. Throughout this period, Dr. Levy collaborated with the famed, late microbiologist Robert Guthrie, MD, PhD, of Buffalo, New York, who had established newborn screening with his invention of the PKU test. During this time, he also continued to conduct research and to diagnose and treat patients with metabolic disorders at the Massachusetts General Hospital. An extraordinary influence for Dr. Levy during this time, and continuing to the present, is the internationally famous Canadian biochemical geneticist Dr. Charles Scriver, with whom Dr. Levy has often collaborated.

Toward the end of the 1970's Dr. Levy moved to Boston Children's Hospital, where he transformed the PKU Clinic it into a larger, comprehensive clinicthe Inborn Errors of Metabolism clinicthat now sees patients and families from around the world who are affected by a range of diseases: PKU, galactosemia, histidinemia, methylmalonic acidemia, problems with vitamin B12 metabolism and many other disorders. The hospital recently named the metabolic program after Dr. Levy.

At Boston Children's Hospital Dr. Levy became concerned about infants born to mothers who have genetic metabolic disease. "Before we began newborn screening girls who had PKU became delayed in their mental development, so very few bore children," Dr. Levy explained. "But now that we were treating them from infancy, they were bearing children. Even though their babies were genetically normal, they would be born with multiple severe problems if the mothers were not strictly treated for PKU during the pregnancies. So, with an extraordinary group of very talented professionals, including psychologists, nutritionists, a nurse, and a social worker, as well as physicians, we organized the New England Maternal PKU Program and followed these women on very strict dietary treatment throughout their pregnancies. We found that this regimen prevented many of these problems that the babies would otherwise have."

Today Dr. Levy is considered one of the foremost proponents worldwide for newborn screening. He led a successful effort in Massachusetts to expand newborn metabolic screening with new technology so that 20 to 30 disorders of amino acid, organic acid and fatty acid metabolism could be included rather than only 5 or 6 disorders previously screened. Within the ACMG, Dr. Levy led the effort to develop "ACT Sheets," one-page synopses of the newborn screened metabolic disorders so that physicians caring for infants can easily read an explanation of the biochemical, clinical and treatment characteristics of the disorders when contacted by a newborn screening program about an abnormality. As part of a contract funded by the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), Dr. Levy began and led the Newborn Screening Translational Research Network of the ACMG.

Though many of his contemporaries have retired, Dr. Levy continues to lead research efforts that examine the long-term outcomes of expanded newborn screening using tandem mass spectrometryincluding the medical, biochemical and neuropsychological outcomes in relation to early treatment. He is also involved with clinical trials to develop new therapies for PKU and homocystinuria. Dr. Levy is driven to continue his work because there is still much work to do. "The fact that we've had to rely on complicated diets that alter the lives of patients so they cannot enjoy a normal meal with their family or their friends, they have to only be able to eat this very difficult diet, and also the fact that we still discover diseases for which we have no treatment, " he explained, "these are the issues that trouble me. There are still individuals we discover during newborn screening or we discover later on because we didn't screen for their disorder, and they have severe disorders for which we have no treatments. There are still metabolic diseases that are not being prevented."

Dr. Levy still spends time communicating face-to-face with patients. "If you have a new baby, in a room with the family, you have to present this very complicated story, and the family has no idea what this is about," he explained. "So, we spend a great deal of time explaining the biochemistry, the genetics, the problems that can occur and the treatments that can prevent these problems. Early on, we just thought about biochemistry. But today we become more involved in talking about the genes, because we think it's important for families to understand the origin of these disorders since at some point we are likely to talk to them about the possibility of gene therapy, actually introducing the normal gene into the child. So, they need to understand where the disorder comes from. It's a complicated and long process. The family will take in as much information as they can, but as you can imagine, a lot of what we tell them will be forgotten or not understood. So, we go over everything with them again, and for as many times as they need."

One of the most pleasing aspects of Dr. Levy's career, he recounted, has been working with wonderful and dedicated individualspsychologists, nutritionists, dieticians, nurses, social workers, coordinators, administratorsand within the community of clinicians and researchers who study metabolic genetic disorders, a "relatively small, cohesive group of delightful, brilliant people" as he describes them. "It's been an extraordinarily wonderful professional life, as gratifying as any professional life I could ever dream of," reflected Dr. Levy. "Little did I know when I started that I would have this kind of life and little did I know that I would be awarded with the awards and certainly nothing comparable to the David L. Rimoin Lifetime Achievement Award."

The David L. Rimoin Lifetime Achievement Award is the most prestigious award given by the ACMG Foundation. A committee of past presidents of the American College of Medical Genetics and Genomics selects the recipient following nominations, which come from the general membership.

About the ACMG Foundation for Genetic and Genomic Medicine

The ACMG Foundation for Genetic and Genomic Medicine, a 501(c)(3) nonprofit organization, is a community of supporters and contributors who understand the importance of medical genetics and genomics in healthcare. Established in 1992, the ACMG Foundation supports the American College of Medical Genetics and Genomics (ACMG) mission to "translate genes into health." Through its work, the ACMG Foundation fosters charitable giving, promotes training opportunities to attract future medical geneticists and genetic counselors to the field, shares information about medical genetics and genomics, and sponsors important research.To learn more and support the ACMG Foundation mission to create "Better Health through Genetics" visit http://www.acmgfoundation.org.

Note to editors: To arrange interviews with experts in medical genetics, contact ACMG Senior Director of Public Relations Kathy Moran, MBA at kmoran@acmg.net.

Kathy Moran, MBAkmoran@acmg.net

See the original post:
Pediatrician and Geneticist Dr. Harvey Levy Receives 2020 David L. Rimoin Lifetime Achievement Award in Medical Genetics from the ACMG Foundation for...

Global Gene Therapy and Antisense Drugs Market: Snapshot

Since an escalating number of patients are being diagnosed with cancer every day, the global demand for gene therapy and antisense drugs is bound to multiply. The growing incidence of health conditions such as Parkinsons disease and high cholesterol is also boosting the demand for gene therapy and antisense drugs. Several new drugs and therapies have been making their debut in the global gene therapy and antisense drugs market of recent.

For instance, in June 2017, it was announced that a group of scientists at the University of Queensland have developed a new technique that is capable of permanently silencing severe allergies triggered by shellfish, peanuts, and venom. The treatment was found to be successful in animal trials. The technique holds considerable potential for treating asthma completely. During the same month, results of another research study for treating multiple myeloma, a type of blood cancer, were announced at a conference held by the American Society of Clinical Oncology. The new CAR-T therapy consists of filtering the blood of the patients in order to eliminate T cells which are then genetically altered and given back to the patients.

Get Sample Copy of the Report @https://www.tmrresearch.com/sample/sample?flag=B&rep_id=500

However, the difficulty pertaining to the delivery of antisense technology to the brain can present key challenges to the expansion of the global market for gene therapy and antisense drugs. Moreover, the unavoidable toxic aftereffects associated with the technology can also inhibit the growth of the gene therapy and antisense drugs market worldwide. However, the present attempts at developing safe and efficient antisense drugs by several market participants including Ionis Pharmaceuticals, which undertook testing of the technology on transgenic mice, can promote the further expansion of the market.

Global Gene Therapy and Antisense Drugs Market: Overview

Antisense gene therapy is emerging as one of the most beneficial therapeutics for various diseases such as tumors, cancer etc. These newer therapies are based on increased knowledge of molecular events that lead to disordered cellular growth. The therapy involves using a gene silencing technique rather than a gene repairing technique for silencing the genes effect.

The research report is a valuable tool for comprehending the progression of the global gene therapy and antisense drugs market between 2017 and 2025.

Global Gene Therapy and Antisense Drugs Market: Treatment Insights

Antisense drugs attach to the mRNA of a target protein, which inhibits the protein production process. Antisense therapeutics can obstruct the expression of oncogenes and other cancer-related genes that express growth factors. Antisense gene therapy involves the utilization of various therapeutic strategies which requires a clear knowledge of the molecular anatomy of cancer related genes. Antisense gene therapy is used to treat various diseases such as hemorrhagic fever, cancer, cystic fibrosis, renal diseases, HIV/AIDS, spinal muscular atrophy, and cardiovascular diseases.

Global Gene Therapy and Antisense Drugs Market: Market Segmentation

On the basis of therapeutic area, the gene therapy and antisense drugs market is segmented into cancer, anemia, rheumatoid arthritis, cardiovascular diseases, HIV/AIDS, cystic fibrosis, diabetes mellitus and obesity, and renal diseases.

By gene transfer method, ex vivo gene transfer and in vivo gene transfer are the segments of the market. The former involves the transfer of cloned genes into cells, i.e., cells are altered outside the body before being implanted into the patient, whereas the latter involves the transfer of cloned genes directly into the patients tissues. The outcome of in vivo gene transfer technology mainly depends on the general efficacy of gene transfer and expression.

Global Gene Therapy and Antisense Drugs Market: Regional Outlook

The global gene therapy and antisense drugs market is segmented into North America, Asia Pacific, Europe, and Rest of the World. Amongst these, North America holds the leading position in the market followed by Europe. The increasing incidence of cancer and other fatal diseases, unhealthy lifestyle practices such as excessive smoking and excessive consumption of high fat content food, and increasing research efforts for treatment against cancer are the major factors driving the gene therapy and antisense drugs market in these regions.

Asia Pacific is expected to emerge as a significant market for gene therapy and antisense drugs. The high population density including a large geriatric population, expeditiously increasing demand for technologically advanced therapeutics, and increasing government support for improved healthcare infrastructure in the region is driving the growth of this regional market. Furthermore, favorable reimbursement policies and tax benefits on newer therapies will further fuel the growth of the Asia Pacific gene therapy and antisense drugs market.

Major Companies Mentioned in Report

Some of the leading companies operating in the global gene therapy and antisense drugs market are GenVec Inc., Avigen Inc., Genome Therapeutics Corp., Tekmira Pharmaceuticals Corporation, Isis Pharmaceuticals, Cell Genesys Inc., and others. These companies are profiled for their key business attributes in the report.

Read Comprehensive Overview of Report @https://www.tmrresearch.com/gene-therapy-antisense-drugs-market

About TMR Research:

TMR Research is a premier provider of customized market research and consulting services to business entities keen on succeeding in todays supercharged economic climate. Armed with an experienced, dedicated, and dynamic team of analysts, we are redefining the way our clients conduct business by providing them with authoritative and trusted research studies in tune with the latest methodologies and market trends.

Contact:

TMR Research,3739 Balboa St # 1097,San Francisco, CA 94121United StatesTel: +1-415-520-1050

Link:
Gene Therapy and Antisense Drugs Market Growth Trends, Key Players, Competitive Strategies and Forecasts to 2025 - 3rd Watch News

Report Description

A recent market intelligence report that is published by Data Insights Partner onGene Therapy marketmakes an offering of in-depth analysis of segments and sub-segments in the regional and international Gene Therapy market. The research also emphasizes on the impact of restraints, drivers, and macro indicators on the regional and world Gene Therapy market over the short as well as long period of time. A detailed presentation of forecast, trends, and dollar values of international Gene Therapy market is offered. In accordance with the report, the global Gene Therapy market is projected to expand at a CAGR of 30% over the period of forecast.

Market Insight, Drivers, Restraints& Opportunity of the Market:

Gene therapy is a medical procedure which replaces defective genes or introduces new genes n order to prevent or cure genetic disorders. This procedure has become a bench mark in medical industry as there is no requirement of surgery or drugs or other procedure which has side effects on the individuals. Gene therapy was first commercialized in China in 2004 by China based SiBono Gene Tech (product Gendicine).

The global gene therapy market has been expanding due to the rigorous research conducted in the field of genetics. The rising awareness about the capability of cure of several rare genetic diseases by gene therapy is another important driver which leads the global gene therapy market during the forecast period. Gene therapy has capability cure several life threatening diseases such as cancer, cardiac diseases, AIDS, cystic fibrosis, age-related disorders, sickle cell anemia etc. In March 2019, the director of the National Health Institute (NIH), the U.S. announced that the recent clinical trials on the gene therapy for the treatment of sickle cell anemia showed promising result- therefore, increasing prevalence of aforementioned lie threatening diseases would likely to drive the growth of the global gene therapy market during the forecast period.

Request for Report Sample:https://datainsightspartner.com/request-for-sample?ref=8

On the other hand, treatment cost and stringent regulatory conditions etc. may hamper the growth of the global gene therapy market in the upcoming future. The results of Gendicine clinical trials were published in 2003 and the medicine got approval by the China State Food and Drug Administration in the same year. Although approved in China, Gendicines use is not very promising outside China. There are several concerns among the researchers about the quality of the clinical trials performed and safety and efficacy of the treatment. However, Gendicines equivalent Advexin (company Introgen Therapueitcs) is still waiting for the FDA approval.

Increasing investment to the gene therapy related research (around 10 Bn was invested in 2015 by private and public organizations), new product developments such as (Zolgensma in 2019), strategic alliance among the key players (such as collaboration between Axovant and Yposkesi) would bring the global gene therapy market an opportunity to propel during the forecast period. In May 2019, Avexis (a Novartis company) has got the FDA approval for Zolgensma for treatment of spinal muscular atrophy for the pediatric patients (less than 2 years of age).

Segment Covered:

This market intelligence report on the global gene therapy market encompasses market segments based on product, application, target user and geography. On the basis of product, the sub-markets is segmented into Yescarta, Kymriah, Strimvelis, Gendicine, Zolgensma and others (Advexin). Based on application, the global gene therapy market has been segregated into large B-Cell lymphoma, Car T Cell therapy, ADA-SCID (adenosine deaminase deficiency), muscular atrophy, head and neck squamus cell carcinoma, others (Crigler-Najjar syndrome). By target user, the global gene therapy market is also classified into adult and pediatric. By Geography, the global gene therapy market has been divided into North America (the U.S., Canada), Latin America (Brazil, Mexico, Argentina and other countries), Europe (Germany, France, the U.K., Spain, Italy, Russia, and other countries), Asia Pacific (India, Japan, China, Australia and New Zealand and other countries), Middle East and Africa (GCC, South Africa, Israel and Other countries).

Profiling of Market Players:

This business intelligence report offers profiling of reputed companies that are operating in the market. Companies such as Novartis, Gilead Sciences, Orchard Therapeutics Ltd, SiBiono GeneTech Co, Introgen Therapeutics and among others have been profiled into detail so as to offer a glimpse of the market leaders. Moreover, parameters such as gene therapy market related investment & spending and developments by major players of the market are tracked in this global report.

Report Highlights:

In-depth analysis of the micro and macro indicators, market trends, and forecasts of demand is offered by this business intelligence report. Furthermore, the report offers a vivid picture of the factors that are steering and restraining the growth of this market across all geographical segments. In addition to that, IGR-Growth Matrix analysis is also provided in the report so as to share insight of the investment areas that new or existing market players can take into consideration. Various analytical tools such as DRO analysis, Porters five forces analysis has been used in this report to present a clear picture of the market. The study focuses on the present market trends and provides market forecast from the year 2017-2027. Emerging trends that would shape the market demand in the years to come have been highlighted in this report. A competitive analysis in each of the geographical segments gives an insight into market share of the global players.

Request for Report Analysis:https://datainsightspartner.com/report/gene-therapy-market/8

Salient Features:

This study offers comprehensive yet detailed analysis of the Gene Therapy market, size of the market (US$ Mn), and Compound Annual Growth Rate (CAGR (%)) for the period of forecast: 2019 2027, taking into account 2017 as the base year

It explains upcoming revenue opportunities across various market segments and attractive matrix of investment proposition for the said market

This market intelligence report also offers pivotal insights about various market opportunities, restraints, drivers, launch of new products, competitive market strategies of leading market players, emerging market trends, and regional outlook

Profiling of key market players in the world Gene Therapy market is done by taking into account various parameters such as company strategies, distribution strategies, product portfolio, financial performance, key developments, geographical presence, and company overview

Leading market players covered this report comprise names such as. Novartis, Gilead Sciences, Orchard Therapeutics Ltd, SiBiono GeneTech Co, Introgen Therapeutics and among others

The data of this report would allow management authorities and marketers of companies alike to take informed decision when it comes to launch of products, government initiatives, marketing tactics and expansion, and technical up gradation

The world market for Gene Therapy market caters to the needs of various stakeholders pertaining to this industry, namely suppliers, manufacturers, investors, and distributors for Gene Therapy market. The research also caters to the rising needs of consulting and research firms, financial analysts, and new market entrants

Research methodologies that have been adopted for the purpose of this study have been clearly elaborated so as to facilitate better understanding of the reports

Reports have been made based on the guidelines as mandated by General Data Protection Regulation

Ample number of examples and case studies have been taken into consideration before coming to a conclusion

Reasons to buy:

vIdentify opportunities and plan strategies by having a strong understanding of the investment opportunities in the Gene Therapy market

vIdentification of key factors driving investment opportunities in the Gene Therapy market

vFacilitate decision-making based on strong historic and forecast data

vPosition yourself to gain the maximum advantage of the industrys growth potential

vDevelop strategies based on the latest regulatory events

vIdentify key partners and business development avenues

vRespond to your competitors business structure, strategy and prospects

Follow this link:
Gene Therapy Market size Witness Growth Acceleration during 2027 - Packaging News 24

Britain is really good at biology. In physics and chemistry, or painting and music, we have often failed to match the Germans, the French or the Italians. But in the bio-sciences, nobody can equal us. Heres an astonishing list of firsts that happened on this damp island: William Harvey and the circulation of the blood. Robert Hooke and the cell. Edward Jenner and vaccines. Charles Darwin and natural selection. Alexander Fleming and antibiotics. Francis Crick and James Watson (and Rosalind Franklin and Maurice Wilkins) and the structure of DNA. Fred Sanger and DNA sequencing. Patrick Steptoe and Robert Edwards and the first test-tube baby. Alec Jeffreys and DNA fingerprinting. Ian Wilmut and Dolly the Sheep. The biggest single contribution to the sequencing of the human genome (the Wellcome Trust).

Annoyingly, the exciting new tool of genome editing is the one that got away. The best of the new tools, known as CRISPR, emerged from the work of a Spaniard, Francisco Mojica, who first spotted some odd sequences in a microbes genome that seemed to be part of a toolkit for defeating viruses. Then a few years ago French, American, Finnish, Dutch and Chinese scientists turned this insight into a device for neatly snipping out specific sequences of DNA from a genome in any species, opening up the prospect of neatly rewriting DNA to prevent disease or alter crops. Two American universities are squabbling over the patents (and Nobel prize hopes). Further improvements are coming thick and fast.

But we are well placed to catch up with superb labs straining at the leash to apply these new tools. The biggest immediate opportunity is in agriculture, and here leaving the European Union is absolutely key. There is no clearer case of a technology in which we will be held back if we do not break free from the EU approach. It would not be a race to the bottom in terms of safety and environmental standards, but the very opposite: a race to the top.

For example, if we allowed the genetically modified blight-resistant potatoes that have been developed at the Sainsbury Laboratory in Norfolk to be grown in fields here in the UK, we would be able to greatly reduce the spraying of fungicides on potato fields, which at present happens up to 15 times a year, harming biodiversity and causing lots of emissions from tractors. That would be a big improvement, not a regression, in environmental terms. But at the moment commercializing the Sainsbury Lab potato is in practice impossible because of onerous EU rules.

Other countries are already dashing ahead with the new technology. Last year a review of the patenting of CRISPR products in agriculture found that, whereas America had taken out 872 patent families and China 858, the European Union had taken out only 194. The gap is growing.

The reason is nothing to do with the quality of research in Europe. It is all about regulation. When genome editing first came along, the European Commission decided to delay for several years making up its mind about how to regulate the release of genome-edited organisms while it waited for the European Court of Justice to decide whether to treat this new technology as if it were like genetic modification (the process invented a generation ago for transferring genes between species) or a form of mutation breeding (the process invented two generations ago for randomly scrambling the genes of plants under gamma rays in the hopes of generating better varieties).

If it was like genetic modification, then it would be subject to draconian rules that amount to a de-facto ban. Nobody even tries to commercialize a GMO crop in Europe any more because you enter a maze of delay, obfuscation, uncertainty, expense and red tape from which you never emerge.

The result is that European agriculture is more dependent on chemical sprays than it would have otherwise been, as shown by research at Gottingen University: on average, GMOs have reduced the application of pesticides to crops wherever they have been grown by 37 per cent. So we have missed out on biological solutions and had to stick with chemical ones instead.

If on the other hand genome editing is like mutation breeding, then you can go ahead and plant a crop straight away here with no restrictions. This is, of course, mad, since mutation breeding is more likely (though still very unlikely) to produce an accidentally harmful result even than GMOs, but its an older technique and has been used for much of the food you eat, including organic food, and for some reason nobody at Greenpeace objects.

Brexit is a fantastic opportunity to do something no European continental competitor is allowed to

Genome editing is an even more precise and predictable technique than GMOs. It involves no transfer of foreign DNA and the incision is made at a specific location in a genome, not at random. It is clearly the safest of all these three techniques, and so said the European Courts advocate general in his advice to the court. But in July 2018 the ECJ, being a political entity, decided otherwise and told the commission what it wanted to hear, that it should treat genome-edited plants and animals as if they were GMOs.

There was fury and dismay throughout the laboratories of Europe. There would have been more in Britain if academics had not feared playing into the hands of Brexiteers while remaining was still a possibility. A Canadian biotech professor tweeted that this was a good day for Canada since it removed a competitor continent from the scene. The absurdity is illustrated by the fact that in some cases it is impossible to distinguish a genome-edited variety from a variety bred by hybridisation or lucky selection with the same trait. Stefan Jansson from Ume University in Sweden put it like this: Common sense and scientific logic says that it is impossible to have two identical plants where growth of one is, in reality, forbidden while the other can be grown with no restrictions; how would a court be able to decide if the cultivation was a crime or not?

Brexit therefore offers a fantastic opportunity to do something no European continental competitor is effectively allowed to do, and that will benefit the environment. We have great laboratories here, in Norwich, Nottingham, Rothamsted and Edinburgh among other places. But the private sector of plant biotechnology is all but extinct in Britain and will take some jump-starting.

Twenty years ago there were 480 full-time equivalent, PhD-level, private sector jobs in agricultural biotechnology in this country. Today there are just ten. That is what has happened to that whole sector in this country as a result of the misinformed and misguided green campaign against GMOs. Until politicians signal a sea change, the private sector will shun the UKs wonderful labs and the breakthroughs will be applied overseas, if at all.

As a new online tool called the Global Gene Editing Regulation Tracker has shown, America, Canada, Argentina, Brazil, Japan and much of the rest of the world are moving towards a nimbler and more rational regulatory approach: namely judging a crop not by the method used to produce it, but by the traits it possesses. If you can make a potato resistant to blight, what matters is whether the potato is safe, not whether it was made by conventional breeding, gamma-ray mutagenesis or genome editing.

[Visit GLPs global gene-editing regulation tracker and index to learn more.]

In the EU, if you made this potato by gamma-ray mutation breeding, scrambling its DNA at random in a nuclear reactor, the regulations would say: No problem. Go ahead and plant it. If you made it by the far more precise method of genome editing, in which you know exactly what you have done and have confined your activities to one tiny bit of DNA, you are plunged into a Kafkaesque labyrinth of regulatory indecision and expense. The House of Lords Science and Technology Committee, on which I sit, recommended we switch to regulation by trait, a few years back but it was not possible before Brexit.

Genome editing can bring not just environmental benefits but animal welfare benefits too. In 2017, scientists at the Roslin Institute near Edinburgh announced that they had genome-edited pigs to protect them against a virus called porcine reproductive and respiratory syndrome, PRRS. They used CRISPR to cut out a short section from the pig gene that made the protein through which the virus gained access to cell. The change therefore denied the virus entry. They did this without altering the function of the protein made by the gene, so the animal grew up to be normal in every way except that it was immune to the disease.

This means less vaccination, less medication and less suffering. What is not to like? (Incredibly, when I mentioned this case in a speech in the House of Lords, a Green Party peer objected that eradicating a disease that causes suffering in pigs might be a bad thing in case it allows a change in pig husbandry techniques. Even Marie Antoinette was never quite that callous.) But commercialising that animal in the UK is currently all but impossible until we change the rules.

Genome-editing technology could revolutionize conservation as well as agriculture. Looking far ahead into much more speculative science, the same scientists at the Roslin who made the virus-resistant pigs are now looking into how to control grey squirrels not by killing them, as we do now, but by using genome editing to spread infertility infectiously through the population, so that the population slowly declines while squirrels live happily into old age.

This technique, called gene drive, could transform the practice of conservation all around the world, especially the control of invasive alien species the single greatest cause of extinction among birds and mammals today. We could eliminate the introduced mosquitos on Hawaii whose malaria is slowly exterminating the native honeycreeper birds. We could get rid of the non-native rats and goats on the Galapagos which are destroying the habitat of tortoises and birds.

We could get rid of the signal crayfish from America that have devastated many British rivers. For those who worry that gene drive might run riot, there is a simple answer: it can and will be designed in each case to last for a certain number of generations, not forever. And it will be wholly species-specific, so it cannot affect, say, the native red squirrel.

Genome editing may one day allow the de-extinction of the great auk

Still more futuristically, genome editing may one day allow the de-extinction of the great auk and the passenger pigeon. To achieve this, we need to take four steps: to sequence the DNA of an extinct species, which we have done in the case of the great auk; to edit the genome of a closely related species inthe lab, which is not yet possible but may not be far off as genome editing techniques improve by leaps and bounds; to turn a cell into an adult animal, which is difficult, but possible through primordial germ cell transfer, again pioneered at the Roslin Institute; and to train the adults for living in the wild, which is hard work but possible.

Genome editing is also going to have implications for human medicine. Here the European Union is less of a problem, and home-grown regulation is already in good shape: cautious and sensibly applied under the Human Fertilization and Embryology Authority. Britain has already licensed the first laboratory experiments, at the Crick Institute, on the use of genome editing in human embryos, but this is for research into infertility, not for making designer babies.

There is universal agreement that germ-line gene editing to produce human beings with new traits must remain off-limits and be considered in future only for the elimination of severe disease, not for the enhancement of normal talents. This view is shared around the world: the Chinese rogue scientist He Jiankui, who claims he used CRISPR to make two babies HIV-resistant from birth, was sentenced to three years in prison last December.

In practice, fears about designer babies are somewhat exaggerated. The same issue comes up about once a decade with every new breakthrough in biotechnology. It was raised about artificial insemination in the 1970s, about in-vitro fertilization in the 1980s, about cloning in the 1990s and about gene sequencing in the 2000s. Indeed, it has been possible to choose or selectively implant sperm, eggs and embryos with particular genes for a long time now and yet demand remains stubbornly low.

Most people do not want to use IVF or sperm donation to have the babies of clever or athletic people, as they easily could, but to have their own babies: the technology has been used almost exclusively as a cure for infertility. Indeed, the more we find out about genomes, the harder it becomes to imagine anybody wanting to, let alone being able to, enhance specific traits in future children by fiddling with genes: there are just too many genes, each with only very small effects, interacting with each other in the creation of any particular behaviour or ability.

Imagine walking into a doctors clinic and being presented with a catalogue of expensive genetic changes that could be made to your future babys genes, each of which might have a tiny and uncertain effect. The truth is most people do not want to have especially clever or sporty offspring: they want children like themselves.

However, in contrast to germ-line gene editing, somatic genome editing will play a large part in medicine. It is already happening, for example in a process known as CAR-T cell therapy, in which an immune cell is genome-edited so that it will attack a specific tumour, then multiplied and injected back into the body as a form of live drug. If we encourage genome editing in Britain we will be in a position to cure some cancers, enhance agricultural yield, improve the nutrient quality of food, protect crops from pests without using chemicals, eradicate animal diseases, enhance animal welfare, encourage biodiversity and maybe bring back the red squirrel. If we do not, then China, America, Japan and Argentina will still push ahead with this technology and will follow their own priorities, leaving us as supplicants to get the technology second-hand.

Matt Ridley is a British journalist and businessman. He is the author of several books, including The Red Queen (1994), Genome (1999), The Rational Optimist (2010) and The Evolution of Everything (2015). Follow him on Twitter @mattwridley

This article originally ran at The Critic and has been republished here with permission.

View original post here:
Why Brexit could jump start UK GMO, CRISPR researchonce stifled by 'dead hand' of EU regulation - Genetic Literacy Project

In this new business intelligence Cancer Gene Therapy market report, PMR serves a platter of market forecast, structure, potential, and socioeconomic impacts associated with the global Cancer Gene Therapy market. With Porters Five Forces and DROT analyses, the research study incorporates a comprehensive evaluation of the positive and negative factors, as well as the opportunities regarding the Cancer Gene Therapy market.

With having published myriads of Cancer Gene Therapy market reports, PMR imparts its stalwartness to clients existing all over the globe. Our dedicated team of experts deliver reports with accurate data extracted from trusted sources. We ride the wave of digitalization facilitate clients with the changing trends in various industries, regions and consumers. As customer satisfaction is our top priority, our analysts are available 24/7 to provide tailored business solutions to the clients.

Request Sample Report @ https://www.persistencemarketresearch.co/samples/2988

The Cancer Gene Therapy market report has been fragmented into important regions that showcase worthwhile growth to the vendors Region 1 (Country 1, Country 2), region 2 (Country 1, Country 2) and region 3 (Country 1, Country 2). Each geographic segment has been assessed based on supply-demand status, distribution, and pricing. Further, the study provides information about the local distributors with which the Cancer Gene Therapy market players could create collaborations in a bid to sustain production footprint.

Some of the major companies operating in the global cancer gene therapy market are Cell Genesys, Advantagene, GenVec, BioCancell, Celgene and Epeius Biotechnologies. Other leading players in cancer gene therapy market include Introgen Therapeutics, ZIOPHARM Oncology, MultiVir and Shenzhen SiBiono GeneTech

Key points covered in the report

Request Report Methodology @ https://www.persistencemarketresearch.co/methodology/2988

What does the Cancer Gene Therapy market report contain?

Readers can get the answers of the following questions while going through the Cancer Gene Therapy market report:

And many more

For any queries get in touch with Industry Expert @ https://www.persistencemarketresearch.co/ask-an-expert/2988

About us:

PMR is a third-platform research firm. Our research model is a unique collaboration of data analytics and market research methodology to help businesses achieve optimal performance.

To support companies in overcoming complex business challenges, we follow a multi-disciplinary approach. At PMR, we unite various data streams from multi-dimensional sources. By deploying real-time data collection, big data, and customer experience analytics, we deliver business intelligence for organizations of all sizes.

Contact us:

305 Broadway, 7th Floor

New York City, NY 10007

United States

Ph.no. +1-646-568-7751

Originally posted here:
Global Briefing 2019 Cancer Gene Therapy Industry Analyzer Technique, Advancements, Market Size, Share, Opportunity and Trend with Growing CAGR till...

The response to the coronavirus is both impressive and thought-provoking. Governments, organisations and people around the world are taking peacetime actions not known in living memory. And rightly so. The projections of human loss the virus could cause are chilling. Nevertheless, I have not been able to stop myself wondering: Why do other crises not generate the same level of action, even when we know they are associated with equally or even more devastating projections?

I have often compared the state of our economy to a body that is suffering an advanced stage of cancer. This is, I realise, a shocking analogy to use, but bear with me: it can provide a new lens through which to look at our economy.

Imagine the cells in your body represented organisations, hard at work producing all the services and goods you need to live a healthy life. Cancerous cells, unlike healthy ones, grow and replicate as fast as they can at the expense of the body that hosts them. In our economy, growth has become just as prevalent at all levels: nationally we grow GDP, organisations maximise profits and individuals accumulate wealth and possessions.

And this has real consequences for human health. Air pollution a direct consequence of how we choose to organise our economy has overtaken smoking as a cause of global deaths. Consumerism and the addictive algorithms behind social media are eroding our mental health; worklessness and low-paid, insecure jobs are driving people to hunger, as evidenced by the huge rise in foodbanks across many countries.

What would happen if we took the same approach to the economic crisis that we are taking to the coronavirus crisis? The anti-viral response has four phases: contain the infection at source, delay its spread, conduct research to understand its behaviour, find a vaccine and mitigate the effects of the virus spreading.

When it comes to our global economy we have, sadly, long passed the contain phase. Cancer-like behaviours have been spreading for decades. In the UK, for example, the 1990s saw a wave of mutual building societies converting to shareholder-owned banks. Banking regulations that split retail and investment banking (such as the Glass-Steagall Act in the US) were repealed in multiple countries around about the same time.

Immunising organisations that operate with a different set of values is an urgent priority

Containing the economic cancer at source is no longer possible. We can no longer surgically remove the tumour because it has already spread. However, we can still protect some areas from becoming infected, those that run with a different ethos: the UKs National Health Service and its public broadcaster are two examples, the Swiss railway (SBB) is another. The co-operative movement has long conformed to values that include co-operating rather than competing with other co-operatives, democratic control and economic participation (thats profit sharing) for all members although sadly it has not been immune either, as the part-takeover of the UKs Co-operative Bank shows. Immunising organisations that operate with a different set of values whose genes have not yet suffered cancerous mutations is an urgent priority.

Delaying the spread of cancerous behaviours is the next priority. There are multiple approaches that can be taken here.

Business schools and management consultancies that educate and influence decision-makers have become amplifying feedback loops for the unfettered growth ideology. Happily, some are now starting to engage seriously with the need for new approaches to management and leadership, which will slow the spread of this kind of thinking.

Other delay mechanisms include representing workers on company boards (as happens in Germany), and publishing CEO-to-worker pay ratios (now mandatory in the UK), which can curb the growth of top salaries.

Any serious attempt to combat our economic cancer must understand its root causes. Human cancer is caused by mutations in our genes the manual for our human bodies that is stored in every one of our cells. Similarly, our economic cancer is caused by how our economic manual the values that underpin our economic and management practices have evolved.

Labs are conducting thousands of experiments as they race towards a vaccine for the coronavirus. Similarly, in every part of our economy people are trying out doing business according to different values

At this very moment, labs all around the world are conducting thousands of experiments as they race towards a vaccine for the coronavirus. Similarly, in every part of our economy people are experimenting, trying out doing business according to different values, conducting gene therapy in which they replace harmful values with healthy ones.

In Germany, Purpose is trialling new models of ownership; In the UK, Operation Upgrade is tackling company law to weaken the power of shareholders; complementary currencies such as the Swiss WIR have operated in parallel to mainstream finance for decades; Common Future is creating more regional and community based economies in the US; B Corps are testing lots of ways of using business as a force for good; and even multi-national corporations are committing to a net positive impact on our climate, Microsoft and Interface being just two examples.

Mitigation is arguably where most effort has been going for a very long time. The raison-detre of many charities and public services has, for centuries, been to mitigate the worst excesses of our current system. Looking after those who have fallen through the cracks of our welfare system, cleaning up environmental pollution or protecting endangered species are all mitigation activities.

In recent times the Deep Adaptation movement is providing more sobering advice on the consequence of climate change that is already inevitable.

Mitigation is necessary and often the only compassionate and practical course of action. But we mustnt let fighting the symptoms distract us from tackling the root cause of the disease. Unlike the coronavirus vaccine, the values and behaviours we need for a healthy economy are already among us today; but we need to spread them quickly. Values even collective ones reside in people. This is why, at On Purpose, we work with people who want to learn how to apply healthy values in the real life of their everyday work.

We mustnt let fighting the symptoms distract us from tackling the root cause of the disease

We are learning a tough lesson in how quickly a virus like corona can spread around the world. Viruses, as it happens, are also the vehicle by which gene therapists smuggle healthy genes into cancerous cells one of the newest approaches to tackle the root causes of cancer. This gives me hope that fundamental change can happen more quickly than we think. We are already seeing the rapid spread of healthy viruses from school climate strikes and veganism to the anti-plastic movement. I believe these are the trailblazers for many more gene therapies to come.

Tom Rippin is thefounder and CEO of On Purpose.

Thanks for reading Pioneers Post. As somebody working in the impact economy, you'll know that producing quality work doesn't come free. We rely on paid subscriptions and partnerships to sustain our purpose-led journalism so if you think it's worth having an independent, specialist media platform to share your news, insight and debate across the globe, please consider subscribing. You'll also be buying social: Pioneers Post is a social enterprise itself, reinvesting all profits to help you do good business, better.

Read the original:
The other public health crisis: What the global economy can learn from our response to coronavirus | The Social Enterprise Magazine - Pioneers Post

The latest research report provides a complete evaluation on GlobalLongevity and Anti-senescence TherapyMarket for the forecast year 2020-2024, which is advantageous for companies irrespective of their size and revenue. The Market analysis provides a comprehensive market study including Key development trends, competitive landscape analysis, market dynamics, and key regions development status forecast 2020-2024. It includes numerous important aspects counting leading competitors which include their business profiling, Longevity and Anti-senescence Therapy market share, gross margin, sales, revenue, growth rate as well as it offers value chain analysis, capacity utilization analysis, SWOT analysis to further investigate.

The report comprehensively investigates the Global Longevity and Anti-senescence Therapy market status, supply, sales, and production. The global market divisions of production and exchanges are estimated along with the review of the production, capacity, sales, and revenue. several aspects such as Longevity and Anti-senescence Therapy import/export, price, gross margin, consumption, and value are also examined.

Request for Sample Report @https://www.industryandresearch.com/report/Global-Longevity-and-Anti-senescence-Therapy-Market-Report-2020/180433#samplereport

The Longevity and Anti-senescence Therapy research report has incorporated the analysis of different factors that augment the markets growth. It constitutes trends, restraints, and drivers that transform the market in either a positive or negative manner. It also provides the scope of different Longevity and Anti-senescence Therapy segments and applications that can potentially influence the market in the future. The detailed information is based on current trends and historic milestones.

Objective of the Report:The main objective of this Longevity and Anti-senescence Therapy report is to help the user understand the market in terms of its definition, segmentation, market potential, influential trends, and the challenges that the market is facing. in-depth researches and analysis were done during the preparation of the report. The readers will find this report very beneficial in understanding the market in depth. The data and the information regarding the market are taken from trustworthy sources such as websites, annual reports of the companies, journals, and others and were checked and validated by the industry experts. The Longevity and Anti-senescence Therapy facts and data are represented in the report using diagrams, graphs, pie charts, and other pictorial representations. This enhances the visual representation and also helps in understanding the facts much better.

Longevity and Anti-senescence Therapy Market Competitor Analysis:Some of the key players operating in the Longevity and Anti-senescence Therapy market areCohBar, TA Sciences, Unity Biotechnology, AgeX Therapeutics?Inc, PowerVision Inc.

Regional Segmentation of Longevity and Anti-senescence Therapy Market:North America Country (United States, Canada)South AmericaAsia Country (China, Japan, India, Korea)Europe Country (Germany, UK, France, Italy)Other Country (Middle East, Africa, GCC)

Longevity and Anti-senescence Therapy Market report Analysed Based on Major Product Type And Application :Product Type Segmentation :Hemolytic Drug Therapy, Gene Therapy, Immunotherapy, Other Stem Cell TherapiesIndustry Segmentation :Hospital, Medical Service Institution, Drug and Device Sales

Table of Contents:Market Overview:The report starts off with an executive summary, including top highlights of the research study on the global Longevity and Anti-senescence Therapy Market.

Market Segmentation:This section provides in-depth analysis of type and application segments of the global Longevity and Anti-senescence Therapy Market and shows the progress of each segment with the help of comprehensible statistics and graphical presentations.

Regional Analysis:All major regions and countries are covered in the report on the global Longevity and Anti-senescence Therapy Market.

Market Dynamics:The report offers deep insights into the dynamics of the global Longevity and Anti-senescence Therapy Market, including challenges, restraints, trends, opportunities, and drivers.

Competitor Analysis:Here, the report provides company profiling of leading players competing in the global Longevity and Anti-senescence Therapy Market.

Research Methodology:The report provides clear information on the research approach, tools, and methodology and data sources used for the research study on the global Longevity and Anti-senescence Therapy Market.

Browse full report @https://www.industryandresearch.com/report/Global-Longevity-and-Anti-senescence-Therapy-Market-Report-2020/180433

Reasons to Purchase this Report:1) Efficiency and quantitative analysis of the market based on segmentation involving both economic as well as non-economic factors2) Preparation of market value (USD Billion) data for each segment and sub-segment3) Specifies the region and segment that is expected to observe the fastest growth as well as to dominate the market4) Analysis by geography emphasizing the consumption of the product/service in the region as well as indicating the factors that are affecting the market within each region5) Competitive landscape which in undertake the market ranking of the major players, along with new service/product launches, partnerships, business expansions and acquisitions in the past five years of companies profiled6) Extensive company profiles comprising of company overview, company insights, product comparative analysis and SWOT analysis for the major market players7) The current as well as the future market viewpoint of the industry with respect to recent developments (which involve growth opportunities and drivers as well as challenges and constraints of both appearing as well as developed regions

Finally, the Longevity and Anti-senescence Therapy market report gives you details about the market research finding and conclusion which helps you to develop profitable market strategies to gain competitive advantage. Supported by comprehensive primary as well as secondary research, the Longevity and Anti-senescence Therapy market report is then verified using expert advice, quality check and final review. The market data was analyzed and forecasted using market dynamics and consistent models.

Request customize:-If you wish to find more details of the report or want a Customization Please contacts us. You can get a detailed of the entire research here.

Contact Us @[emailprotected]

Follow this link:
Global Longevity and Anti-senescence Therapy Market Report Business Plans & Strategies With Forecast 2020-2024 | CohBar, TA Sciences - 3rd Watch...

Gene therapy could dramatically alter how dozens of inherited diseases are treated. It's also transforming how the academic institutions working in this growing field move research from the laboratory to the clinic.

Private sector skepticism a decade or more ago spurred institutions like the University of Pennsylvania and Nationwide Children's Hospital to advance experimental projects much further before selling their ideas to biopharma companies a departure from the previous model of identifying a molecular target and letting industry do the heavy lifting.

As a result, university technology transfer officers are much more involved in the technical and commercial details of preclinical drug development, from assembling financing and creating private companies to building manufacturing capacity. The product is a host of new startups, such as AveXis, Spark Therapeutics and Bamboo Therapeutics, that in recent years have been swallowed up by large pharmaceutical companies.

"The old way is, 'I have a patent, I'm going to throw it over the fence to you and you throw me a sack of money,'" said John Swartley, managing director of the University of Pennsylvania's Penn Center for Innovation, in an interview. "This is completely different. This is co-development."

John Swartley

Permission granted by University of Pennsylvania

"We're directly involved over multiple years in helping to move the technology forward. And our commercialization partner is going to take it hopefully all the way to the market."

A paper published earlier this month in JAMA quantifies the shift. Together, hospitals, universities and the National Institutes of Health sponsored 206 of the 341 identified gene therapy trials that were active in 2019. Biotech and pharma companies led the remaining 135.

Measured by funding, hospitals, universities and the NIH had a hand in more than 280 of those studies, as some trials had multiple funders. Fourteen trials were funded by other federal sources or non-profit charities.

Hospitals and universities were most active in early-stage studies, with industry sponsoring only 22% of Phase 1 trials. But, in gene therapy, those initial human tests can hold more weight, as the benefits of a genetic fix can be quickly apparent.

"This is a sign that the model of drug development that was prominent in the past academia does basic science and finds some targets and then pharma develops the actual drug product is pretty different with gene therapy," one of the paper's authors, Walid Gellad, director of the Center for Pharmaceutical Policy and Prescribing at the University of Pittsburgh, wrote in an email to BioPharma Dive.

The changing academic model also raises questions about the rich price tags being sought by drugmakers for gene therapies, given the greater role played by universities and other non-profit entities.

"The paper, I think, informs discussions about how high prices really need to be in order to encourage private risk taking for gene therapies it may be a different number than for other drugs that have less late stage involvement by academia and NIH," wrote Gellad.

University involvement in gene therapy development was driven in part by the private sector's reluctance to get involved in a therapeutic approach perceived, until several years ago, as risky. The death of Jesse Gelsinger in a Penn gene therapy trial in 1999 inflicted severe reputational damage on the field, driving away drugmaker interest.

Scientists kept the faith, and their institutions carried the field forward for years afterward. When Swartley began working at Penn in 2007, one of his first meetings was with the university's gene therapy director James Wilson, who was in charge of the tragic trial that led to Gelsinger's death.

James Wilson

Permission granted by the University of Pennsylvania

"From an external perspective, from an industrial perspective, there was almost nothing happening," he said. "But it was evident from the kind of research that Dr. Wilson and his colleagues were sharing with us, they made a very convincing case that this was going to rapidly shift into a more of a developmental paradigm."

"They were anticipating a tremendous amount of industry interest when that shift occurred," Swartley added. "It turned out to be very prophetic."

At the University of North Carolina, the situation was similar in the early part of the 2000s. The institution reached a slightly different solution, however, spinning out companies like Asklepios BioPharmaceutical to advance gene therapy beyond the walls of the university laboratories.

"We had a lot of vector technology, but the market was not receptive to gene therapy at the time," said Kelly Parsons, associate technology commercialization director at UNC, in an interview. "We had a startup company that had to work very diligently to try to establish the merits of gene therapy."

Asklepios is still an independent company today, some of its gene therapy work having been folded into a Pfizer-owned Duchenne muscular dystrophy project that was previously developed by Bamboo Therapeutics.

But the time spent building the knowledge and expertise at universities or closely affiliated startups has been one of the reasons why big pharmas have rushed into the space. By advancing the technology, the universities reduced the risk of failure, making pharmas more willing to buy in.

"We had a recognition that if we wanted the for-profit sector and the investment sector and the [venture capital] world to give gene therapy a chance, it was important as an institution we were able to start that process of de-risking the asset," said Matthew McFarland, vice president of commercialization and industry relations at Nationwide, in an interview.

Doing so was a greater commitment than they expected. "What we did is say: 'What stage would these assets need to get to before external dollars would be interested in investing?'" he said. "And the reality is, oh my gosh, you have to de-risk it all the way to the point it's ready to go into the patients."

That included the initial Phase 1 study of the spinal muscular atrophy gene therapy now known as Zolgensma, which was licensed to AveXis and later acquired by Novartis.

More broadly, development included building production capabilities compliant with Good Manufacturing Practices, which govern quality and consistency standards for finished drug products, and a regulatory team that was able to prepare Investigational New Drug applications within the hospital's technology transfer office.

Building up manufacturing expertise has resulted in a new business for Nationwide: the for-profit Andelyn Biosciences, which will run a commercial scale gene therapy production facility.

Solving the manufacturing question is something many academic gene therapy centers are still grappling with as they near the point of handing off to private-sector partners. Biopharma companies want to have confidence that the therapies manufactured by university scientists will work as well in clinical trials and in wider use as they did in earlier study.

"There's no university that has the ability to ramp their early production manufacturing production to a level to get enough doses that industry doesn't have to recapitulate it," said Jim O'Connell, director of technology transfer at the University of Florida's UF Innovate, in an interview. "It's notorious for university labs, small molecules and others, to not be able to have their work reproduced out in the real world."

This very question may have been behind data quality issues for Zolgensma. Last summer, Novartis was chastised by the Food and Drug Administration for having submitted manipulated preclinical data, a scandal that the Swiss pharma tied to AveXis co-founder and former Nationwide trial investigator Brian Kaspar. Through his lawyer, Kaspar has denied all wrongdoing.

"Academic institutions have got to ask themselves: How far into this do we want to go?," said O'Connell. "It's going to have a whole bunch of costs that universities aren't used to taking on. How do we share the expense? How do we share the risk appropriately?"

Thorny questions notwithstanding, the increased investment has led to better returns for universities. Technology transfer offices interviewed by BioPharma Dive report the licensing deals are much richer for gene therapies that have advanced to human testing or near it money which gets returned to scientists and their departments to fund new research.

Returns aren't equally shared, however. Schools blessed with research that is sought-after by private industry flourish, while others struggle, said Lee Vinsel, a Virginia Tech assistant professor who is writing a book called "The Innovator's Delusion."

Indeed, broadly speaking, universities reported a little more than $3 billion in licensing revenue in 2017, but spent $68 billion, according to the Association of University Technology Managers. Less than 1% of licenses yielded more than $1 million in revenue.

Moreover, Vinsel argues the potential for licensing revenue incentivizes universities to only conduct research the private sector wants to license.

"One reason why we need federal funding and university research is to do basic science that corporations aren't going to pay for and do," Vinsel said. "If we tack more university research towards the profitable, who is going to do this basic work, including research that could really help society but will enrich no one?"

McFarland of Nationwide, however, points to less lucrative licenses it has signed, such as a device to prevent pressure ulcers in patients with tracheostomies, along with a mental health research and treatment facility the institution has launched, as ventures that were enabled by bigger deals like in gene therapy.

"If we can take that return and continue to foster research not only in [gene therapy] but even further spread that out and have an impact across all of research," he said.

"There are a lot of times when we're not the office of tech commercialization, but instead we're the office of tech realization, because what we go into is just about getting it out there to the public, and we're not going to get a return on it."

See original here:
Big pharma shied away from gene therapy for years. Academia picked up the slack - BioPharma Dive

Revenue from marketed dermatology products increased 85% for fourth quarter 2019 and 49% for full-year 2019 compared to 2018

NDA for IV tramadol accepted for review by FDA; PDUFA date is set for October 10, 2020

Rolling NDA submission for CUTX-101 for the treatment of Menkes disease is on track to begin in the fourth quarter of 2020

NEW YORK, March 16, 2020 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (NASDAQ: FBIO) (Fortress), an innovative biopharmaceutical company, today announced financial results and recent corporate highlights for the fourth quarter and full year ended December 31, 2019.

Fortress achieved multiple key milestones in 2019 and early 2020, including:

Lindsay A. Rosenwald, M.D., Fortress Chairman, President and Chief Executive Officer, said, We have generated significant momentum throughout 2019 and into early 2020. In order to drive our next phase of growth, our world-class business development team continues to identify and acquire high-potential marketed and development-stage assets to further expand our portfolio of product opportunities. Additionally, Fortress and our development partners continue to advance our clinical-stage programs across multiple therapeutic categories. With five commercial products and over 25 programs in development, we aim to continue to meaningfully increase value and decrease overall risk for Fortress shareholders. Looking ahead, we expect 2020 to be a record revenue-generating year and a transformational one for many of the development-stage programs across Fortress and our partner companies. Finally, we look forward to continued acquisitions of marketable dermatology drugs and in-licenses of development-stage drug candidates.

2019 and Recent Corporate Highlights1:Marketed Dermatology Products

IV Tramadol

CUTX-101

CAEL-101

MB-107 (Lentiviral Gene Therapy for XSCID)

Cosibelimab (formerly CK-301, an anti-PD-L1 antibody)

CK-101 (third-generation EGFR inhibitor)

MB-102 (CD123-targeted CAR T cell therapy)

MB-101 (IL13R2-targeted CAR T cell therapy)

MB-108 (Oncolytic Virus C134)

MB-104 (CS1-targeted CAR T cell therapy)

MB-103 (HER2-targeted CAR T cell therapy)

MB-105 (Prostate Stem Cell Antigen (PSCA)-targeted CAR T cell therapy)

MB-106 (CD20-targeted CAR T cell therapy)

BAER-101 (novel 2/3subtype-selective GABA A positive allosteric modulator [PAM])

General Corporate

Financial Results:

About Fortress Biotech Fortress Biotech, Inc. (Fortress) is an innovative biopharmaceutical company that was recently ranked number 10 in Deloittes 2019 Technology Fast 500, an annual ranking of the fastest-growing North American companies in the technology, media, telecommunications, life sciences and energy tech sectors, based on percentage of fiscal year revenue growth over a three-year period. Fortress is focused on acquiring, developing and commercializing high-potential marketed and development-stage drugs and drug candidates. The company has five marketed prescription pharmaceutical products and over 25 programs in development at Fortress, at its majority-owned and majority-controlled partners and at partners it founded and in which it holds significant minority ownership positions. Such product candidates span six large-market therapeutic areas, including oncology, rare diseases and gene therapy, which allow it to create value while mitigating risk for shareholders. Fortress advances its diversified pipeline through a streamlined operating structure that fosters efficient drug development. The Fortress model is driven by a world-class business development team that is focused on leveraging its significant biopharmaceutical industry expertise to further expand the companys portfolio of product opportunities. Fortress has established partnerships with some of the worlds leading academic research institutions and biopharmaceutical companies to maximize each opportunity to its full potential, including Alexion Pharmaceuticals, Inc., City of Hope, Fred Hutchinson Cancer Research Center, InvaGen Pharmaceuticals Inc. (a subsidiary of Cipla Limited), St. Jude Childrens Research Hospital and Nationwide Childrens Hospital. For more information, visit http://www.fortressbiotech.com.

Forward-Looking StatementsThis press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, as amended. As used below and throughout this press release, the words we, us and our may refer to Fortress individually or together with one or more partner companies, as dictated by context. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; uncertainties relating to preclinical and clinical testing; risks relating to the timing of starting and completing clinical trials; our dependence on third-party suppliers; risks relating to the COVID-19 outbreak and its potential impact on our employees and consultants ability to complete work in a timely manner and on our ability to obtain additional financing on favorable terms or at all; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as may be required by law. The information contained herein is intended to be reviewed in its totality, and any stipulations, conditions or provisos that apply to a given piece of information in one part of this press release should be read as applying mutatis mutandis to every other instance of such information appearing herein.

Company Contacts:Jaclyn Jaffe and William BegienFortress Biotech, Inc.(781) 652-4500ir@fortressbiotech.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 591-2839tplohoros@6degreespr.com

____________________________________________1 Includes product candidates in development at Fortress, majority-owned and controlled partners and partners in which Fortress holds significant minority ownership positions. As used herein, the words we, us and our may refer to Fortress individually or together with our affiliates and partners, as dictated by context.

FORTRESS BIOTECH, INC. AND SUBSIDIARIESConsolidated Balance Sheets($ in thousands except for share and per share amounts)

FORTRESS BIOTECH, INC. AND SUBSIDIARIESConsolidated Statements of Operations($ in thousands except for share and per share amounts)

View original post here:
Fortress Biotech Reports Record Fourth Quarter and Full-Year 2019 Financial Results and Recent Corporate Highlights - BioSpace

Global Cancer Gene Therapy Report available at Digits n Markets contains an overview of the Global Cancer Gene Therapy which covers market size, opportunities, trends, growth rate, and competition landscape. The Global Cancer Gene Therapy is segmented Source, Product Type Applications and regions. With forecast to 2027.

Digits n Markets has recently published a comprehensive market research report on the Global Cancer Gene Therapy that includes evaluation of market size and various segments. The competitive environment is analyzed along with study of winning strategies adopted by key players.

The report is a detailed study on the accounting Global Cancer Gene Therapy with details regarding an in-depth evaluation of the industry vertical. The study is performed taking into consideration a twofold aspect of consumption and production. Speaking of the product category, the report provides detailed product remuneration, manufacturing of the product and the gross margins of the firms manufacturing the products. With regards to the consumption, the study reveals the product consumption value and the product consumption volume along with the status of import as well as the export of the products.

The Global Cancer Gene Therapy Market Anticipated to exhibit a CAGR 35.1% during Forecast Period. 2018-2025

Avail a free sample in PDF format along with a quick look at vital report briefs:https://digitsnmarkets.com/sample/6650-global-cancer-gene-therapy-market

Key Questions Answered by the Report:

Numerous micro and macro-economic factors impacting the growth of the market are analyzed and the data is represented in a way to aid the clients to enhance their strategic decision making. Key players operating in the Global Cancer Gene Therapy are:

Table of Content

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

Originally posted here:
Global Cancer Gene Therapy Market, Trends, Analysis, Opportunities, Share and Forecast 2018-2025 - Galus Australis

SAN DIEGO and PENNINGTON, N.J., March 17, 2020 /PRNewswire/ --OncoSec Medical Inc. Incorporated (NASDAQ:ONCS) (the "Company" or "OncoSec"), a company developing late-stage intratumoral cancer immunotherapies, today announced the publication of positive TAVO monotherapy data in patients with metastatic melanoma in the Annals of Oncology. The publication titled, "Intratumoral Delivery of Tavokinogene Telseplasmid Yields Systemic Immune Responses in Metastatic Melanoma Patients," features data previously highlighted at both American Association of Cancer Research (AACR) and the Melanoma Bridge annual meetings.Annals of Oncologyis the official publication of the European Society for Medical Oncology.

The complete publication in Annals of Oncology is linked here and available on OncoSec's website at https://oncosec.com/publications/.

The publication describes OncoSec's study of patients with Stage III/IV melanoma who were treated intratumorally with plasmid encoding IL-12 (tavokinogene telseplasmid or TAVO), followed by electroporation on days 1, 5, and 8 every 90 days in the main study with additional patients treated in two exploration cohorts with alternative schedules. Correlative analyses for programmed death-ligand 1 (PD-L1), flow cytometry to assess changes in immune cell subsets and analysis of intratumoral immune-related gene expression were carried out on pre-and post-treatment samples from study patients, as well as from additional patients treated during exploration of additional dosing schedules beyond the pre-specified protocol dosing schedule.Response was measured by study-specific criteria to maximize detection of latent and potentially transient immune responses in patients with multiple skin lesions.Toxicities were graded by the Common Terminology Criteria for Adverse Events version 4.0 (CTCAE v4.0).

The objective overall response rate (ORR) was 35.7% in the main study, with a complete response (CR) rate of 17.9%.The median progression-free survival in the main study was 3.7 months while the median overall survival was not reached at a median follow up of 29.7 months. A total of 46% of patients in all cohorts having both injected and uninjected lesions experienced regression of at least one of these uninjected lesions and 25% had a net regression of all untreated lesions. Transient procedural pain (n= 24, 80%) and injection site reactions were the most commonly experienced adverse events.

Transcriptomic and immunohistochemistry analysis showed that immune activation and co-stimulatory transcripts were up-regulated, with an increase of adaptive immune resistance.

The publication concluded that intratumoral TAVO was well-tolerated and led to systemic immune responses in advanced melanoma patients. While tumor regression and increased immune infiltration were observed in treated as well as untreated/distal lesions, adaptive immune resistance limited the response.

"TAVO treatment appears to drive a change in the immune microenvironment, which results in an immune response to melanoma with minimal systemic toxicity. These data demonstrate that thisin situtumor vaccination strategy may be a safe and effective approach to inducing multiple sustained, productive changes in the immune microenvironment that would be too toxic using similar systemic agents and drive significant clinical results," concluded study co-author Adil Daud, M.D., Department of Medicine, University of California, San Francisco."We look forward to continued evaluation of the TAVO approach as a monotherapy in future clinical trials."

TAVOis currently being evaluated as a combination therapy in multiple clinical trials, including KEYNOTE-695, a pivotal trial in late-stage anti-PD-1 checkpoint refractory metastatic melanoma, and two phase 2 trials, one for triple negative breast cancer (TNBC) and a second for head and neck cancer. TAVO enables the intratumoral delivery of DNA-based IL-12, a naturally occurring protein with immune-stimulating functions.OncoSec's technology, which employs electroporation, is designed to produce a controlled, localized expression of IL-12 in the tumor microenvironment, enabling the immune system to target and attack tumors throughout the body.Results from recently completed clinical studies of TAVOhave demonstrated a local immune response, and subsequently, a systemic effect as either a monotherapy or combination treatment approach.

"While our ongoing pivotal KEYNOTE-695 study is evaluating TAVO and KEYTRUDA combination therapy in late-stage checkpoint refractory metastatic melanoma patients and has begun to yield positive results, publication of monotherapy data with TAVO demonstrates its utility as a standalone treatment in this patient population," stated Christopher Twitty, Ph.D., OncoSec's Chief Science Officer and a co-author of the publication. "The increase in adaptive resistance observed in the tumor microenvironment, in particular PD-L1, makes TAVO a particularly well-suited partner with anti-PD-1 checkpoint therapies.We are encouraged to see such a high response rate and will continue to evaluate TAVO's utility as a monotherapy for metastatic melanoma."

Annals of Oncology is the latest among a presently growing volume of peer-reviewed journals to highlight the potential of TAVO as a novel immunotherapy. A recent publication in Cancer Immunology Research, linked here, also explored the mechanism of activation of systemic immunity in patients from the TAVO monotherapy study in metastatic melanoma patients. Additionally, Clinical Cancer Research featured TAVO monotherapy data in Merkel cell carcinoma on the cover of its February 2020 issue, linked here. You can find a list of all TAVO publications and scientific presentations at https://oncosec.com/publications/.

About OncoSec Medical IncorporatedOncoSec Medical Incorporated (the "Company," "OncoSec," "we" or "our") is a late-stage biotechnology company focused on developing cytokine-based intratumoral immunotherapies to stimulate the body's immune system to target and attack cancer. OncoSec's lead immunotherapy investigational product candidate TAVO (tavokinogene telseplasmid) enables the intratumoral delivery of DNA-based interleukin-12 (IL-12), a naturally occurring protein with immune-stimulating functions.The technology, which employs electroporation, is designed to produce a controlled, localized expression of IL-12 in the tumor microenvironment, enabling the immune system to target and attack tumors throughout the body. OncoSec has built a deep and diverse clinical pipeline utilizing TAVOas a potential treatment for multiple cancer indications either as a monotherapy or in combination with leading checkpoint inhibitors; with the latter potentially enabling OncoSec to address a great unmet medical need in oncology: anti-PD-1 non-responders.Results from recently completed clinical studies of TAVOhave demonstrated a local immune response, and subsequently, a systemic effect as either a monotherapy or combination treatment approach. In addition to TAVO, OncoSec is identifying and developing new DNA-encoded therapeutic candidates and tumor indications for use with its new Visceral Lesion Applicator (VLA), to target deep visceral lesions, such as liver, lung or pancreatic lesions. For more information, please visit http://www.oncosec.com.

TAVOis a trademark of OncoSec Medical Incorporated.

KEYTRUDAis a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc.

Risk Factors and Forward-Looking Statements This release, as well as other information provided from time to time by the Company or its employees, may contain forward-looking statements that involve a number of risks and uncertainties that could cause actual results to differ materially from those anticipated in the forward-looking statements. Forward-looking statements provide the Company's current beliefs, expectations and intentions regarding future events and involve risks, uncertainties (some of which are beyond the Company's control) and assumptions. For those statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. You can identify forward-looking statements by the fact that they do not relate strictly to historical or current facts. These statements may include words such as "anticipate," "believe," "could," "estimate," "expect," "intend," "may," "plan," "potential," "should," "will" and "would" and similar expressions (including the negative of these terms). Although we believe that expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements. The Company intends these forward-looking statements to speak only at the time they are published on or as otherwise specified, and does not undertake to update or revise these statements as more information becomes available, except as required under federal securities laws and the rules and regulations of the Securities Exchange Commission ("SEC"). In particular, you should be aware that the success and timing of our clinical trials, including safety and efficacy of our product candidates, patient accrual, unexpected or expected safety events, and the usability of data generated from our trials may differ and may not meet our estimated timelines. Please refer to the risk factors and other cautionary statements provided in the Company's Annual Report on Form 10-K for the fiscal year ended July 31, 2019 and subsequent periodic and current reports filed with the SEC (each of which can be found at the SEC's websitewww.sec.gov), as well as other factors described from time to time in the Company's filings with the SEC.

Company Contact:Gem HopkinsHead of Corporate Communications858-210-7334ghopkins@oncosec.com

View original content to download multimedia:http://www.prnewswire.com/news-releases/oncosec-announces-publication-of-positive-tavo-monotherapy-results-in-metastatic-melanoma-patients-in-annals-of-oncology-301025487.html

SOURCE OncoSec Medical Incorporated

Read more from the original source:
OncoSec Announces Publication of Positive TAVO Monotherapy Results in Metastatic Melanoma Patients in Annals of Oncology - BioSpace

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

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

UK role in gene therapy techR&D

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

What makes something patentable?

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

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

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

by Fiona Kellas, Reuben Jacob

16 March 2020

14:20

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

The findings were published in Circulation Research.

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

The wheels are in motion for us to find out.

Image Credit: Monika Robak from Pixabay

See the original post:
To Turbocharge Anti-Aging Treatment, Just Add... a Protein Found in Fruit Flies? - Singularity Hub

He swipes once. Twice. Three times.

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

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

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

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

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

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

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

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

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

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

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

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

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

****

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

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

They start with a detailed neurological assessment.

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

"No."

"Have you noticed any difference in your speech?"

"No."

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

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

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

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

This is when Kelsie can sense his perpetual optimism faltering.

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

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

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

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

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

"Those are our favorite words," Kelsie says.

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

****

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

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

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

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

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

But there was some good news.

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

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

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

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

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

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

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

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

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

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

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

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

That moment, that month, changed them.

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

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

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

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

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

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

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

****

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

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

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

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

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

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

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

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

And he did. They did.

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

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

****

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

And that is what makes Zinman so excited.

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

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

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

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

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

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

****

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

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

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

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

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

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

DOWNLOAD FREE SAMPLE REPORT: https://marketandresearch.biz/sample-request/115893

Product And Application Segments:

This report provides a detailed study of given products. The report also provides a comprehensive analysis of key trends & advanced technologies. Then the market is segmented by application with historical and projected market share and compounded annual growth rate. It offers an advanced approach to the market growth with a detailed analysis of the overall competitive scenario of the global Hemophilia Gene Therapy market.

Market statistics by types: Hemophilia A, Hemophilia B, etc.,

Market outlook by applications: Hemophilia A Gene Therapy, Hemophilia B Gene Therapy, etc.,

The elite players described in this report are: Spark Therapeutics, Freeline Therapeutics, Sangamo Therapeutics, Ultragenyx, uniQure, Shire PLC, BioMarin, Bioverativ, etc.

For a complete understanding of the market dynamics, the global Hemophilia Gene Therapy market is analyzed through key geographic areas, namely: North America (United States, Canada and Mexico), Europe (Germany, France, UK, Russia and Italy), Asia-Pacific (China, Japan, Korea, India and Southeast Asia), South America (Brazil, Argentina, etc.), Middle East & Africa (Saudi Arabia, Egypt, Nigeria and South Africa).

This Market Research Report Encloses Importance On:

ACCESS FULL REPORT: https://marketandresearch.biz/report/115893/global-hemophilia-gene-therapy-market-2020-by-company-regions-type-and-application-forecast-to-2025

The global Hemophilia Gene Therapy market report gives importance to product sales, price models, gross margins, and revenue generations. Along with the dealers of these products, the study also presents a summary of the top customers for the same. The report speaks about product consumption growth rate across the applicable regions as well as consumption market share as well as the consumption rate of all regions, based on product types and applications.

Customization of the Report:This report can be customized to meet the clients requirements. Please connect with our sales team (sales@marketandresearch.biz), who will ensure that you get a report that suits your needs. You can also get in touch with our executives on +1-201-465-4211 to share your research requirements.

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

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

Get FREE Exclusive PDF Sample Copy of This Report: https://www.upmarketresearch.com/home/requested_sample/92436

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

With an all-round approach for data accumulation, the market scenarios comprise major players, cost and pricing operating in the specific geography/ies. Statistical surveying used are SWOT analysis, PESTLE analysis, predictive analysis, and real-time analytics. Graphs are clearly used to support the data format for clear understanding of facts and figures.

Customize Report and Inquiry for The Gene Therapy Industry Market Report: https://www.upmarketresearch.com/home/enquiry_before_buying/92436

Get in touch with our sales team, who will guarantee you to get a report that suits your necessities.

Primary research, interviews, news sources and information booths have made the report precise having valuable data. Secondary research techniques add more in clear and concise understanding with regards to placing of data in the report.

The report segments the Global Gene Therapy Industry Market as:Global Gene Therapy Industry Market Size & Share, by Regions

Global Gene Therapy Industry Market Size & Share, by ProductsType 1Type 2Type 3

Global Gene Therapy Industry Market Size & Share, ApplicationsApplication 1Application 2Application 3

Key Playerscompany 1company 2company 3company 4company 5

Avail the Discount on this Report @ https://www.upmarketresearch.com/home/request_for_discount/92436

UpMarketResearch offers attractive discounts on customization of reports as per your need. This report can be personalized to meet your requirements. Get in touch with our sales team, who will guarantee you to get a report that suits your necessities.

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

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

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

Get Access to the FREE sample report: https://digitsnmarkets.com/sample/4233-cancer-gene-therapy-market

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

Table of Content

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

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

How This Report On Cancer Gene Therapy Market Can Help You?

About Digits N Markets:

Digits N Markets has a vast repository of latest market research reports on trending topics, niche company profiles, market size and other relevant data released by renowned publishers. We have access to the database related to niche markets and trending topics in various industries. We also update the data regularly to provide recent statistics to the client. Recent data and reports will be featured on our websites and clients will be able to access the same. Our clients will be able to benefit from qualitative & quantitative insights in the report which will support them in taking concrete business decisions.

Contact Us:

Digits N Markets410 E Santa Clara Street, Unit #762San Jose, CA 95113Phone :+1 408-622-0123Email: [emailprotected]Website:- http://www.digitsnmarkets.com

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Newswise What gets the leader of the NIH jazzed?

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

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

1. Single-cell sequencing

[see this section of the talk here]

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

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

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

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

2. New ways to see the brain

[See this section of the talk here]

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

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

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

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

3. Induced pluripotent stem (iPS) cells

[See this section of the talk here]

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

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

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

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

4. Microbiome advances

[See this section of the talk here]

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

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

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

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

5. Influenza vaccines

[See this section of the talk here]

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

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

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

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

6. Addiction prevention and treatment of pain

[See this section of the talk here]

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

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

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

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

7. Cancer Immunotherapy

[See this section of the talk here]

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

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

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

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

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

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

8. Tapping the potential of precision medicine

[See this section of the talk here]

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

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

Join in:Sign up forAll of Usat UAB today.

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

9. Rare diseases

[See this section of the talk here]

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

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

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

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

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

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

10. Diversity in the scientific workforce

[See this section of the talk here]

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

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

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

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

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