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Category Archives: Gene Medicine

Gene Editing Tools Market(COVID-19 Impact Analysis) 2020 Global Industry Key Strategies, Historical Analysis, Segmentation, Application, Technology,…

This Gene Editing Tools Market research document helps a lot to businesses by giving an insightful market data and information to businesses for making better decisions and defining business strategies. Additionally, this report gives Gene Editing Tools Market size, trends, share, growth, and cost structure and drivers analysis. The Gene Editing Tools Market 2020 Report is a perfect window to the Gene Editing Tools Industry which explains what market definition, classifications, applications, engagements and market trends are. Such report is a key to achieve the new horizon of success. The report comprises of CAGR value fluctuation during the forecast period of 2020-2027, historic data, current market trends, market environment, technological innovation, upcoming technologies and the technical progress in the related industry.

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The Major Top Key Players associated with the Gene Editing Tools Market areThermo Fisher Scientific Inc.; CRISPR Therapeutics; Editas Medicine; National Human Genome Research Institute; Intellia Therapeutics, Inc.; Merck KGaA; Horizon Discovery Ltd.; GeneCopoeia, Inc.; ERS Genomics; Takara Bio Inc.; New England Biolabs; GenScript among others.

Market Definition:GlobalGene Editing Tools Market

Gene editing also known as genome editing is the method of modifications of DNA focusing on replacement and deletion of these DNA from a specific location inside of a genome in an organism/cell. This process requires specialized tools to be carried out and is generally undertaken in different labs with the help of engineered nucleases.

Market Drivers

Market Restraint

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This Gene Editing Tools report works as a reputable source of information to offer a telescopic view of the existing market trends, situations, opportunities and status. In addition, this market report consists of vital aspects of the market that contain but are not limited to historic data, present market trends, environment, future trends, technological innovation, upcoming technologies and the technical progress in the industry.

The Gene Editing Tools market report is a window to the industry which gives details about what market definition, classifications, applications, engagements and market trends are. The complete knowledge and info covered in the report is based on latest industry news, opportunities, and trends.

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Development policies and plansare discussed as well as manufacturing processes and cost structures are also analyzed.

This report also states import/export consumption, supply and demand Figures, cost, price, revenue and gross margins. Competitive analysis includes competitive information of leading players in Gene Editing Tools market, their company profiles, product portfolio, capacity, production, and company financials.

In addition, report also provides upstream raw material analysis and downstream demand analysis along with the key development trends and sales channel analysis.

Segmentation:Global Gene Editing Tools Market

By Product (CRISPR/Cas9, ZFNs, TALENs, Viral Systems, Transposon Systems, Others)

By Application (Veterinary Medicine, Cell Line Engineering, Bioremediation, Food & Brewing Development, Food Waste Management, Bio Sensing Development, Others)

By Disease Application (Sickle Cell Disease, Heart Disease, Diabetes, Alzheimers Disease, Obesity, Others)

By End-User (Biotech & Pharma Companies, CROs, Academic & Research Institutes, Food Industry, Others)

Few of the major competitors currently working in the Gene Editing Tools market areThermo Fisher Scientific Inc.; CRISPR Therapeutics; Editas Medicine; National Human Genome Research Institute; Intellia Therapeutics, Inc.; Merck KGaA; Horizon Discovery Ltd.; GeneCopoeia, Inc.; ERS Genomics; Takara Bio Inc.; New England Biolabs; GenScript among others.

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Gene Editing Tools Market(COVID-19 Impact Analysis) 2020 Global Industry Key Strategies, Historical Analysis, Segmentation, Application, Technology,...

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OPINION: Support gene researchers – The Daily Evergreen

Just because you've watched Jurassic Park doesn't mean you know that gene editing is bad

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Gene editing is an important area for further research.

Gene editing is the future and we should embrace it. I dont mean a wholehearted approval of the technique but to recognize that its here. We must be thoughtful about its applications and aware of its potential.

Weve been gene editing all throughout history. Selectively breeding animals and crops to promote the traits that are desirable or helpful to us. But todays gene editing is much different.

We use CRISPR-Cas9, which can target specific gene sequences to edit, said Samantha Noll, a bioethicist with the Functional Genomics Initiative.

CRISPR, a gene editing tool taken from bacterial defenses against viruses, allows molecular biologists here at WSU to alter specific genes in big animals. Compared to CRISPR, selective breeding is crude and inaccurate, only using phenotypic traits, such as eye or hair color, as the roadmap for which animal to breed or not. Selective breeding attempts to manipulate the genome by prioritizing expressed traits whereas CRISPR allows the manipulation of the genome by access to the entire gene pool.

Charlie Powell, the public information officer for WSUs school of Veterinary Medicine, mentioned multiple ways this gene editing technique can be applied positively.

At any given moment here in the US there are a million pigs in transit. A certain percentage of those animals will develop upper respiratory diseases as a result of the stress, Powell said. If we could make those pigs resistant instead of vaccinating them then we have the possibility of limiting those losses in the industry. This involves adding back the wild-type genes that they originated with.

This suggests ethical solutions by way of medical intervention. Lingering just on livestock application, how much animal suffering could be eliminated by well-applied selective gene editing? Instead of injecting tons of antibiotics, genetic immunity may be the way to go.

Fostering the path to healthier and happier livestock could be inroads to alleviating human challenges such as hunger and poverty. Abundance of sustainable and ethically produced meats could ease food demand, and resilient healthy livestock could be a valuable investment for underprivileged individuals.

Being a land-grant university, WSU research is primarily aimed at helping the local community, hence the focus on big animals and local agriculture. One of the research programs seeks to knock out the genes responsible for horns in cattle. This avoids the painful horn removal process for the animals and prevents accidental injury between cattle, which cost time and money.

Though these are promising initiatives, we cant be short-sighted either. William Kabasenche, a bioethicist focusing on the therapeutic applications of CRISPR, described what he called off-target effects.

Its called pleiotropy, when one gene influences multiple phenotypes, Kabasenche said.

Phenotypes are just the expressed traits. The information for those traits is stored in the gene. Off-target effects occur when a gene has unaccounted phenotypes, meaning that the manipulation of that gene produced an unforeseen or undesirable change in a phenotype.

This is why we have to be very careful when gene editing. Yes, the potential is huge both for scientific discovery as well as the well-being of conscious entities but we must guard against a utopic vision of the technology. There are trade-offs involved. Changing one gene may produce the desired effects, but drastically impact an unrelated but necessary function.

This stresses the need for research and the role of ethics in research. We should all want this work to be done, but we cannot simply focus on positive outcomes and draw the conclusion that it justifies its good. We must also consider how these outcomes are achieved.

We must also consider the harmful potential of gene editing. How we choose to engage our resources, the decisions and norms we set in research will in some part determine how well apply this technology. These norms are being born at research institutions like WSU.

Its a promising start that WSU includes ethicists, educators and biologists to tackle these difficult issues.

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OPINION: Support gene researchers - The Daily Evergreen

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Mitchell disease: solving the medical mystery – Health Europa

A team of researchers led by Dr Hugo Bellen at Baylor College of Medicine, investigator at the Jan and Dan Duncan Neurological Research Institute at Texas Childrens Hospital and also a Howard Hughes Medical Institute investigator, and lead author, Hyunglok Chung, postdoctoral fellow in the Bellen lab, have discovered that a mutation in a gene that causes Mitchell disease.

However, they also discovered that the gene was not inherited, but was in fact a new mutation.

A patient with neurological symptoms enrolled in the Undiagnosed Diseases Network (UDN), presenting with an unidentified late-onset neurodegenerative disorder.

Bellen said: On comparing the patients and his parents DNA, the team identified a mutation in the patient that resulted in a single amino acid substitution (N237S) in the ACOX1 protein. This change was seen only in the patient and was not present in either of his parents DNA, indicating that the patient had a de novo, or new, mutation on this gene

With the help of the online gene-matching tool GeneMatcher, we found two more patients who had the same new mutation in the ACOX1 gene.

All three patients, who ranged from 3 to 12 years old at the time of disease onset, had remarkably similar clinical features, including degeneration of peripheral nerves that caused a progressive loss of mobility and hearing. The three individuals had identical gene variants, a clear indication that ACOX1 dysfunction likely was the cause of the symptoms.

The finding that an ACOX1 mutation was linked to Mitchell disease initially baffled the researchers. The only known ACOX1-related disorder described in the medical literature at that time presented earlier in infancy with seizures, severe cognitive decline, neuro-inflammation and accumulation of very-long-chain-fatty acids in plasma and, more importantly, was caused by the lack of the ACOX1 protein none of which was true for these three patients.

The brain has large amounts of lipids, which are critical for the proper functioning of the nervous system. Abnormal breakdown of lipids in the brain and peripheral nervous system is associated with several neurodegenerative diseases, Bellen said.

The gene ACOX1 is involved in lipid breakdown. It produces an enzyme called Acyl-CoA oxidase 1 that initiates a series of reactions that break down very-long-chain-fatty acids in small intracellular organelles called peroxisomes.

The team used fruit flies to understand the problem, with Chung discovering that the ACOX1 protein is abundant and critical for the maintenance of glia, cells that support neurons.

To gain a better understanding of how ACOX1 variants affect the function of glia, they generated two mutant fly lines, the first one lacked both the copies of ACOX1 gene and the second, carried the substitution mutation (N237S) found in one of the ACOX1 genes in the Mitchell disease patients.

Chung said: Flies lacking ACOX1 mimicked the symptoms of ACOX1 deficiency in humans, including elevated levels of very-long-chain-fatty acids along with dramatic loss of glia and neurons and progressively impaired neuronal function. When we reduced the synthesis of very-long-chain-fatty acids in these flies by administering the drug bezafibrate, we observed significant improvement in lifespan, vision, motor coordination and neuronal function, implicating elevated levels of these lipids and their excessive accumulation in glia as an important contributor.

The researchers suggest that bezafibrate could offer a new therapeutic avenue for patients.

In contrast to the loss of ACOX1, the introduction of the single amino acid substitution (N237S) in ACOX1 gene resulted in a hyperactive ACOX1 protein.

Typically, breakdown of very-long-chain-fatty acids by the enzymatic action of ACOX1 produces small amounts of highly reactive oxygen species, but glial cells quickly neutralise them. However, in Mitchells disease, hyperactive ACOX1 produces copious amounts of toxic reactive oxygen species, leading to the destruction of glia and their neighbouring neurons.

The harmful effects due to hyperactive ACOX1 were potently reversed with the antioxidant N-acetyl cysteine amide (NACA). However, NACA did not suppress the lethality or toxic effects in flies that lacked ACOX1, a clear indication that the two diseases act via entirely different pathways and would need to be treated with two distinct therapeutic strategies.

Bellen said: This study is a prime example of how combining UDNs unique team science approach with power of fruit fly genetics is facilitating rapid and phenomenal progress in rare diseases research. We take on cases of patients with conditions never described before, uncover new diseases, and find definitive molecular diagnosis for them. We make significant progress in unravelling the causes of these novel diseases and rapidly identify and test promising new treatment options.

We have successfully identified more than 25 disease-causing genes within the past three years a task that typically takes many years.

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Mitchell disease: solving the medical mystery - Health Europa

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My Gene Counsel Partners with UConn Health to Provide Expanded Genetic Counseling Services – Yahoo Finance

Easy-to-understand genetic counseling reports will lead to more appropriate follow-up and better health outcomes

NEW HAVEN, Conn., April 16, 2020 /PRNewswire-PRWeb/ -- My Gene Counsel, a digital health company that provides innovative genetic counseling solutions, today announced it is teaming up with UConn Health's Neag Comprehensive Cancer Center's Hereditary Cancer Program to ensure that UConn's patients who have undergone genetic testing for cancer predisposition have access to timely and accurate genetic counseling information through the online delivery of My Gene Counsel's Living Lab Reports. Together, My Gene Counsel and UConn Health will use state-of-the-art digital tools to efficiently communicate up-to-date genomic information that will drive an improved standard of care.

By supplementing its current genetic counseling services with My Gene Counsel's digital counseling tools, UConn Health will serve as a leader in this space with the ability to better support patients over time in the post-test period. Each incoming patient will receive an electronic Living Lab Report sponsored by UConn Health and the Linda Clemens Breast Cancer Foundation that is personalized to the individual's genetic test results and outlines the most frequently asked questions and answers on topics related to disease risk, medical management options, relevance to family, emotional support, and available resources. This information is supported by tools to enhance understanding, such as hover dictionary and graphics.

"Genetics can be overwhelming and confusing, so when a patient leaves my office, I worry about how much information they have retained," said Connor Linehan, MS, LGC, a board-certified genetic counselor for the Hereditary Cancer Program at UConn Health. "Our goal, in partnering with My Gene Counsel, is to increase patient understanding in the hopes that better comprehension equals appropriate medical follow-up and better health outcomes. The addition of a user-friendly genetic counseling report that patients can review before and after their appointment and over time will be invaluable to empower them to make informed decisions about their healthcare."

The Living Lab Reports are written and continuously updated by a network of top certified genetic counselors and medical experts and are vetted by patient advocates. In addition to delivering complex genomic information in a way that patients can understand, the reports will update and notify patients automatically by text and/or email as My Gene Counsel adds new information to reflect changes in disease risk, medical management options, variant reclassification, and relevant clinical trials.

"I encourage patients to contact us over the years as information about hereditary cancer changes over time," said Jennifer Stroop, MS, CGC, LGC, a board-certified genetic counselor for the Hereditary Cancer Program at UConn Health. "However, this is not always easy. We are very excited to now be able to offer our patients a reference tool with continuing updates and notifications. With these continued touchpoints for engagement and retention, we will be able to meet the expressed need to help our patients feel more connected and supported in the long term."

My Gene Counsel's wraparound solution, available at UConn in May 2020, will enable the responsible return of results, engage and update patients, and integrate data into UConn's health care system. Living Lab Reports will be personalized by gene and variant and provided for all results, whether testing is negative or identifies a variant of clinical or uncertain significance.

"We are excited to partner with UConn Health, a forward-thinking health system on the cutting-edge of hereditary cancer and precision medicine," said Ellen Matloff, MS, CGC, president and CEO of My Gene Counsel. "Their dedication to improving health through education, innovation, and patient-centered clinical care beyond the initial genetic test aligns seamlessly with our own ideals."

More than 600 people undergo genetic counseling and testing each year as part of the Neag Comprehensive Cancer Center's Hereditary Cancer Program, which is staffed by two genetic counselors. The expanding volume of patients and limited bandwidth led the team to proactively seek out a technical solution that could help solve the challenge of monitoring critical clinical updates and research and recontacting patients.

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"It is essential for UConn Health, as a major university center, to keep pace with the growing demand for up-to-date genomic information," said Susan Tanenbaum, MD, medical director of the Neag Comprehensive Cancer Center. "The integration with My Gene Counsel is a giant step towards UConn Health assuming a position of global leadership in genomics and personalized medicine."

About My Gene Counsel: My Gene Counsel bridges the gap between genetic testing and precision medicine by addressing one of the most critical pain points in the industry lack of accurate and timely genetic counseling information for patients and providers. Their Living Lab Reports deliver genetic counseling information that updates as new clinical information emerges, guidelines evolve, and genetic variants are reclassified. Founded by certified genetic counselors with 30+ years of clinical experience, My Gene Counsel empowers partners to efficiently deliver on the promise of precision medicine. For more information, visit http://www.mygenecounsel.com.

About UConn Health: UConn Health is Connecticut's only public academic medical center. Based on a 206-acre campus in Farmington, UConn Health has a three-part mission: research, teaching and patient care. Home to the UConn School of Medicine, School of Dental Medicine and UConn John Dempsey Hospital with nearly 5,000 employees supporting nearly 1,000 students, over 800,000 annual patient visits, and innovative scientific research contributing to the advancement of medicine. For more information, visit http://www.health.uconn.edu.

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My Gene Counsel Partners with UConn Health to Provide Expanded Genetic Counseling Services - Yahoo Finance

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Research Roundup: Another Promising COVID-19 Vaccine and More – BioSpace

Every week there are numerous scientific studies published. Heres a look at some of the more interesting ones.

Another COVID-19 Vaccine Looks Promising

According to the World Health Organization, there are 70 vaccines being developed worldwide for COVID-19, with three already in human clinical trials. The furthest along is one by CanSino Biologics and the Beijing Institute of Biotechnology, which is in Phase II. The others are by U.S. companies, Inovio Pharmaceuticals and Moderna. Another institution, the University of Pittsburgh, also announced that in laboratory tests their COVID-19 vaccine, delivered via a fingertip-sized patch, showed positive results in laboratory mice, producing antibodies specific to SARS-CoV-2 at enough amounts to neutralize the virus. The research was published in EBioMedicine, published by The Lancet.

We had previous experience on SARS-CoV in 2003 and MERS-COV in 2014, said co-senior author Andrea Gambotta, associate professor of surgery at the Pitt School of Medicine. These two viruses, which are closely related to SARS-CoV-2, teach us that a particular protein, called a spike protein, is important for inducing immunity against the virus. We know exactly where to fight this new virus. Thats why its important to fund vaccine research. You never know where the next pandemic will come from.

It uses a more traditional process than the mRNA one being used by Moderna. The virus is being called PittCOVacc, and uses laboratory-manufactured pieces of viral protein to build immunity. Its a process similar to that used in seasonal flu shots. They also leveraged a new technique to deliver the drug, called a microneedle array, to increase the potency of the vaccine. The fingertip-sized patch has 400 tiny needles that deliver the spike protein pieces into the skin. It goes on like a Band-Aid. The needles are built from sugars and the antigens, and they just dissolve.

We developed this to build on the original scratch method used to deliver the smallpox vaccine to the skin, but as a high-tech version that is more efficient and reproducible patient to patient, said Louis Falo, co-senior author and professor and chair of dermatology at Pitts School of Medicine and UPMC. And its actually pretty painlessit feels kind of like Velcro.

They are currently submitting an Investigational New Drug (IND) with the U.S. Food and Drug Administration (FDA) and hope to begin Phase I human clinical trials in the next few months.

Genetic Mechanisms of Inflammatory Bowel Disease

Researchers at Children's Hospital of Philadelphia identified a genetic variant that causes the development of inflammatory bowel disease (IBD). The pathway is linked to other immune disorders. More than 240 genetic regions are already associated with IBD, but each region has multiple markers and not all are causative. The researchers focused on the single nucleotide polymorphism (SNP) rs1887428, located on the promoter region of the JAK2 gene. The protein coded by the gene controls the production of blood cells. The team found that two transcription factors, RBPJ and CUX1, recognize the DNA sequence altered by the rs1887428 SNP, and while it only has mild influence on JAK2 expression, it was amplified by other proteins in the JAK2 pathway.

Possible Mechanism of Link Between Obesity and Breast Cancer

Breast cancer (and other cancers) and obesity are associated, but the reason for that link isnt well understood. Researchers from the University of Louisville published research suggesting that the fatty acid binding protein family, especially FABP4, plays a critical role. Fat tissue produces FABP4 within fat cells, which processes and distributes water-insoluble long-chain fatty acids. Normally, some FABP4 enters the bloodstream, but the higher fat volume, the more FABP4 is secreted. They believe two mechanisms are in play. Within cells, FABP4 increases in certain tumor-associated macrophages, which accumulate in tumors and promote cancer growth. And second, when elevated levels of FABP4 circulate outside the fat cells, it promotes breast cancer by directly interacting with breast cancer cells.

Using Cellular Machinery Without the Cells to Develop Drugs

Northwestern University and ShanghaiTech University leveraged cell-free synthetic biology to produce a drug that kills SARS-CoV-2 in cell cultures. They indicate they could create the new drug by taking the molecular machinery out of cells and using that machinery to make a product in a safe, cheap and quick way. The molecule is called valinomycin. By using this method, they were able to increase production yields more than 5,000 times in only a few quick design cycles.

Glucose Metabolism Linked to Alzheimers Disease

Researchers with the National Institutes of Healths National Institute on Aging conducted the largest study so far on proteins related to Alzheimers and identified proteins and biological processes that regulate glucose metabolism that are associated with Alzheimers. The study was published in the journal Nature Medicine.

The study was part of the Accelerating Medicines Partnership for Alzheimers Disease (AMP-AD). The investigators assayed the levels and analyzed the expression patterns of more than 3,000 proteins in brain and cerebrospinal fluid samples collected at centers across the U.S.

This is an example of how the collaborative, open science platform of AMP-AD is creating a pipeline of discovery for new approaches to diagnosis, treatment and prevention of Alzheimers disease, said Richard J. Hodes, NIA director. This study exemplifies how research can be accelerated when multiple research groups share their biological samples and data resources.

The study involved analyzing protein expression patterns in more than 2,000 human brain and almost 400 cerebrospinal fluid samples taken from both healthy individuals and Alzheimers patients. They analyzed how the protein modules relate to Alzheimers and other neurodegenerative diseases. They observed changes in proteins related to glucose metabolism and an anti-inflammatory response in glial cells in brain tissues from both Alzheimers patients and people with documented brain pathology who were cognitively normal. This also would seem to support increasing evidence that brain inflammation is involved in the disease as well.

In Alzheimers patients, they found that how cells extract energy from glucose is increased in both the brains and spinal fluid of Alzheimers patients. The proteins observed were also elevated in preclinical Alzheimers patients, which is to say, people with brain pathology of the disease who had not shown cognitive decline.

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Evotec extends Takeda pact to gene therapy – Bioprocess Insider – BioProcess Insider

Evotec is building a gene therapy R&D center manned by ex-Takeda scientists and has already landed a multi-year drug discovery pact for it with their former Japanese employer.

Evotecis expanding into the field of gene therapy by building an R&D center in Austria using scientists previously employed by Takeda, whose first project will be a long-term research and discovery project with their former Japanese employer, exploring oncology, rare diseases, neuroscience and gastroenterology.

The entry by the German discovery alliance and development partnership group into gene therapy further expands its capabilities beyond small molecules, cellular therapies and biologics while the research alliance withTakeda Pharmaceutical, announced on 6 April, builds upon an existing collaboration begun last September around drug discovery programs in which Evotec will deliver clinical candidates for Takeda to pursue into clinical development.

Image: iStock/sittithat tangwitthayaphum

This addition of gene therapy is the latest step in a very long strategy at Evotec to build a truly comprehensive organization which can deliver medicines discovery and development right across the different modalities, or scientific tools, that we see in modern medicine today, Evotec chief operating officer Craig Johnstone toldScrip.

He said the move was consistent with Evotecs two-pronged drug discovery strategy, which uses the Hamburg-based groups so-called Execute segment allying with external pharma partners which in turn supports reinvestment into Evotecs internal pipeline within its so-called Innovate segment.

This allows us to bring gene therapy project concepts into the Evotec Innovate pipeline at our choice and discretion. That was not possible before, so we can now use gene therapy to support our partners and also use it to support ourselves.

Johnstone said Evotec already makes broad use of CRISPR in its discovery and development activities. This will only amplify that aspect of Evotecs activities, he added.

Evotec Gene Therapy (Evotec GT) will start operations with a team of gene therapy experts at an R&D site in Orth an der Donau, Austria. Its scientists have deep expertise in vectorology and virology as well as disease insights, in particular in hemophilia, hematology, metabolic and muscle diseases.

We are recruiting the team, which will be composed of ex-Takeda employees. Its leadership has a long history in gene therapy stretching back years and to legacy organizationsBaxaltaandShire which were acquired by Takeda, but who were made redundant. Well be bringing them all on board over the next eight weeks, Johnstone said. Friedrich Scheiflinger, previously head of drug discovery for Takeda in Austria, will head up the new gene therapy unit.

No financial details were disclosed about Evotecs latest collaboration with Takeda.

Its a fairly straight-forwardcontractual framework that has been agreed between Evotec and Takeda, Johnstone said, without elaborating.

Sten Stovall is a London-based editor and writer with 40 years of experience in the field of journalism, including more than 20 years with Reuters and eight years with The Wall Street Journal/Dow Jones Newswires. He can be reached at sten.stovall@informa.com

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