Archive for the ‘Gene Therapy Research’ Category

FNF Research (fnfresearch.com) offering a comprehensive analysis on the Updated Research on Gene Therapy Market Analysis 2020 Size & Share Report Including COVID-19 Impact and Forecast to 2026 where users can benefit from the complete market research report with all the required useful information about this market. This is the latest report, covering the current COVID-19 impact on the market. The rapidly changing market scenario and initial and future assessment of the impact are covered in the report. The report discusses all major market aspects with an expert opinion on current market status along with historic data. This market report is a detailed study on the growth, investment opportunities, market statistics, growing competition analysis, major key players, industry facts, important figures, sales, prices, revenues, gross margins, market shares, business strategies, top regions, demand, and developments.

TheGene Therapy Marketreport entails a comprehensive database on Upcoming market estimation based on historical SWOT data analysis. It enables clients with quantified data for current market perusal. It is a professional and 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 Gene Therapy Market size, share, growth, trends, segment, and forecasts from 2020-2026.

According to the research report, [185+ Pages PDF Report]Global gene therapy market was valued at approximately 1.4 billion in 2019 and is expected to reach a value of around USD 3.4 billion, at a CAGR of around 14.7% between 2019 and 2027. Gene therapy is the kind of experimental method that makes use of genes for treating or preventing disease by inserting foreign genetic material like DNA or RNA into the persons cells.

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The market research report also offers information on potential investment opportunities, strategic growth market analysis, and probable threats that will adhere to the client to systematically and creatively plan out the business models and strategies. The critical data analysis in the Gene Therapy market report is laid out in an upright way. This means that the information is represented in form of infographics, statistics, and uncomplicated graphs to make it an effortless and time-saving task for the client.

Growth of the overall global trust and corporate service market has also been forecasted for the period 2020-2026, taking into consideration the previous growth patterns, the growth drivers, and the current and future trends.

(**The consulting and implementation services segment to account for the highest market share during the forecast period.**)

The scope of the Gene Therapy Market report has a wide spectrum extending from market scenarios to comparative pricing between major players, cost, and profit of the specified market regions. The numerical data is supported by statistical tools such as SWOT analysis, BCG matrix, SCOT analysis, and PESTLE analysis. The statistics are depicted in a graphical format for a clear picture of facts and figures.

The generated report is strongly based on primary research, interviews with top executives, news sources, and information insiders. Secondary research techniques are utilized for better understanding and clarity for data analysis.

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The report provides a complete view of the Gene Therapy market and encompasses a detailed type of portfolio and strategic developments of key vendors. To know the competitive landscape of the Gene Therapy market, an analysis of Porters five forces model is done. The study cover market attractiveness analysis, in which type, source type, and application segments are specialized based on the market size, growth rate, and attractiveness.

The report study further includes an in-depth analysis of industry players market shares and provides an overview of leading players market position in the Gene Therapy sector. Key strategic developments in the Gene Therapy market competitive landscape such as acquisitions & mergers, inaugurations of different products and services, partnerships & joint ventures, MoU agreements, VC & funding activities, R&D activities, and geographic expansion among other noteworthy activities by key players of the Gene Therapy market are appropriately highlighted in the report.

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Gene Therapy Market Historic Data (2019-2026):

Gene Therapy Market Forecast (2020-2026):

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The Gene Therapy market has been segmented based on offering, technology, end-use application, and end-user. It also provides a detailed view of the market across four main regions: North America, Europe, APAC, and RoW.

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Gene Therapy Market 2020 Size, Share, Demand and Detailed Competitive Outlook by 2026 - Illadel Graff Supply

Precise treatment leads to resolution of patient's debilitating symptoms and complete remodeling of her lymphatic system

PHILADELPHIA, Nov. 20, 2020 /PRNewswire/ -- Researchers at Children's Hospital of Philadelphia (CHOP) have resolved a severe lymphatic disorder in a girl with Noonan Syndrome that had led to upper gastrointestinal bleeding, fluid collection around the lungs, and numerous surgeries that had been unable to resolve her symptoms. By identifying a genetic mutation along a pathway related to lymphatic vessel development and function, the research team was able to target the pathway using an existing drug they had used in a previous case to remodel a patient's lymphatic system.

The case study, which was published today in Pediatrics, describes a resolution of the patient's symptoms within three months while on the medication.

"This study is quite significant," said first author Yoav Dori, MD, PhD, Director of the Jill and Mark Fishman Center for Lymphatic Disorders at CHOP. "Inhibiting this pathway seems to have sweeping, widespread effects on the lymphatic system. How this process occurs is not fully understood, but is remarkable in its speed and breadth. This gives us a lot of hope for treating other patients with genetic mutations along this same pathway in the future."

The patient described in the paper, Maria, first came to CHOP when she was 14, after experiencing severe anemia due to upper gastrointestinal bleeding, as well as other symptoms including fluid build-up in the cavity around her lungs, chronic fatigue, delayed puberty, and difficulty gaining weight. Maria had been born with Noonan Syndrome, a genetic disorder that prevents normal development in various parts of the body and often results in short stature, heart defects and other physical problems, including an abnormal lymphatic system. Despite aggressive medical therapy elsewhere, Maria continued to bleed internally, and she underwent multiple blood transfusions to try to stabilize her health.

Within two days of transferring to CHOP, the lymphatics team, led by Dori, determined Maria had many lymphatic irregularities, which were leading to internal bleeding and lung problems, so they scheduled Maria's first intervention, a lymphatic embolization procedure that would seal the leaky vessels in her gut.

However, within two months of the procedure, Maria's gastrointestinal bleeding recurred. Over the following 8 months, she underwent two additional procedures, as well as a cauterization procedure to close off some of the blood vessels in her gut, but the benefits of each procedure lasted only about three months before the bleeding and her symptoms returned.

Based on whole exome sequencing done at CHOP's Center for Applied Genomics, the research team learned that Maria had a genetic mutation in the SOS1 gene, which operates along the RAS-MAPK pathway. This pathway involves mitogen-activated protein kinase (MEK), and Maria's mutation caused an overproduction of MEK, which resulted in the uncontrolled proliferation of her lymphatic vessels.

The research team had previously used a MEK inhibitor in another patient with a severe lymphatic disorder with great success. That patient had a mutation in the ARAF gene, which is also on the RAS-MAPK pathway. Within months of beginning treatment with trametinib, a MEK inhibitor, the patient saw a resolution of his symptoms and a complete remodeling of his lymphatic system.

Given that SOS1 operates on the same pathway as ARAF, Jean Belasco, MD, an oncologist in CHOP's Cancer Center who co-led the study, applied for compassionate use of the drug in Maria's case, given the lack of other treatment options.

"The success of trametinib in another patient with a mutation on the RAS-MAPK pathway encouraged us to try this approach, since other procedures and therapies continued to be unsuccessful," Belasco said. "Although we are in the early days of this type of personalized medicine, the hope is that by looking at patients' mutations, we can find more drugs and better care for patients with genetic diseases."

Within three months of starting the drug, Maria's vital signs stabilized. The bleeding stopped, her electrolyte, hemoglobin, and albumin levels returned to normal, and she began to gain weight. Maria's mother noticed that Maria wasn't going through periods of exhaustion anymore, and her pallor improved.

"She looks better than she's ever looked," her mother said. "She looks like a normal teenager. It's like night and day. She's also a lot happier. I think she knew deep down she was dying. The medicine gave her hope."

Hakon Hakonarson, Director of the Center for Applied Genomics and co-author of the paper, said that although Maria's SOS1 mutation is distinctly different than the ARAF mutation seen in the other patient, the drug was equally effective because it targets and blocks the function of MEK. He likened the scenario to a pathway where 15 events need to occur for a cell to function. Maria's SOS1 mutation might occur at step nine, whereas the ARAF mutation might occur at step three, but both genes are on a chain that ultimately passes through a tunnel that leads to phosphorylation and overactivity of MEK. Since both mutations were so-called gain of function mutations, MEK and thus lymphatic activity was overexpressed in both patients. The MEK inhibitor put the brakes on a system in overdrive.

"Remarkable advances in genetics have allowed us to uncover these mutations and cluster them into selective pathways and determine effective therapies based on genetic mutations with very high precision," said Hakonarson. "No one could have guessed that this drug would have worked for Maria without knowing the underlying genetics. This discovery is extremely important because Noonan Syndrome has the biggest patient population with alterations in MEK signaling. Not all Noonan patients will have mutations that respond to this therapy, but a very good number of them will."

He added that the treatment could also benefit patients with other genetic defects, though he noted the ongoing use of the drug treats the symptoms caused by these mutations, but does not fix the gene or cure the underlying condition.

"MEK inhibition has the potential to have significant effects on other organ systems affected by RAS-MAPK gene defects, such as the heart, eyes, skin and the coagulation system," Hakonarson said.

Hakonarson is also part of CHOP's Comprehensive Vascular Anomalies Program (CVAP), a CHOP Frontier Program that uses state-of-the-art genomics and personalized research strategies to determine the causes of complex vascular conditions and identify targeted therapies. The program works closely with the Lymphatic Imaging and Interventions Frontier Program, which is led by Dori. CHOP's Frontier Programs conduct cutting-edge research that translates into advanced clinical care. The CVAP, in particular, draws on the extensive clinical and genomic research capacity within the Cancer Center and Center for Applied Genomics.

Even with the success of the breakthrough treatment pioneered by these programs, it is not entirely clear why MEK inhibitors not only resolve patients' symptoms but also completely remodel their lymphatic systems. Hakonarson said one possibility is that when mutated genes cause uncontrolled growth of the lymphatic system, the body's vessels leak fluid everywhere in the body. When you shut down the unregulated growth, other homeostatic mechanisms that are balancing the system come into effect, so the overreactive cells that were growing out of control die and are replaced by normal cells that gradually build up the lymphatic system.

Whatever the mechanism, Maria's mother said her daughter had no hesitation at being the first patient with Noonan Syndrome to try this treatment to resolve a lymphatic issue.

"Maria saw the value from the beginning," she said. "She saw the value for herself, but she was also thinking of other Noonan kids, some of whom have passed away from lymphatic issues. She was willing and eager."

Dori et al. "Severe Lymphatic Disorder Resolved with MEK Inhibition in a Noonan Patient with SOS1 Mutation," Pediatrics, published online November 20, 2020, doi: 10.1542/2020-000123

About Children's Hospital of Philadelphia: Children's Hospital of Philadelphia was founded in 1855 as the nation's first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals, and pioneering major research initiatives, Children's Hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country. In addition, its unique family-centered care and public service programs have brought the 564-bed hospital recognition as a leading advocate for children and adolescents. For more information, visit http://www.chop.edu

Contact: Natalie SolimeoChildren's Hospital of Philadelphia267-426-6246solimeon@chop.edu

View original content:http://www.prnewswire.com/news-releases/chop-researchers-reverse-severe-lymphatic-disorder-in-patient-with-noonan-syndrome-by-targeting-genetic-pathway-301177697.html

SOURCE Children's Hospital of Philadelphia

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CHOP Researchers Reverse Severe Lymphatic Disorder in Patient with Noonan Syndrome by Targeting Genetic Pathway - BioSpace

Helixmith said Thursday the first enrolled patient has received its new gene therapy, Engensis (VM202), in phase 3-2 study of diabetic peripheral neuropathy (DPN).

The company plans to continue administering the drug to other participants, as it is screening many additional patients at five clinical trial centers. It is conducting DPN phase 3-2 trials, REGAIN-1A, at 15 clinical centers with 152 enrolled patients in the U.S.

Helixmith aims to meet two endpoints in the study.

The trial's primary efficacy endpoint is to compare changes in average daily pain scores between VM202 and placebo. The secondary endpoint is to reduce pain by 50 percent or more.

DPN is one of the most common complications of diabetes as 30 million U.S. adults have the disorder. Around 28.5 percent of diabetic patients develop DPN, and up to half of them may advance to painful DPN (PDPN).

Dr. Miguel Trevino, head of clinical trials at Innovative Research in which the first patient received VM202, noted that the existing treatments for DPN only relieve pain and show various adverse events with limited efficacy, inevitably leaving half of the patients in painful life.

"The delay of this phase 3 study was due to the preparation of clinical operation system and examining numerous items during the patient's screening process," Helixmith CEO Kim Sun-young said. "Our latest study will add considerable knowledge and evidence to support the optimal use of VM202 in patients needing therapeutic options."

The U.S. Food and Drug Administration recognized the scientific and clinical results of VM202 and designated it as RMAT (regenerative medicine advanced therapy) in 2018.

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Helixmith injects DPN therapy into 1st patient of P3 study - Korea Biomedical Review

Gene Therapy Market Growth, Trends, and Forecasts (2020 2025)

The Gene Therapy Market research report 2020 provides a basic overview of the industry including definitions, classifications, applications, and industry chain structure. The Gene Therapy market report provides information regarding market size, share, trends, growth, cost structure, Gene Therapy market competition landscape, market drivers, challenges and opportunity, capacity, revenue, and forecast 2025.

The report delivers a comprehensive overview of the crucial elements of the market and elements such as drivers, current trends of the past and present times, supervisory scenario & technological growth. This report also includes the overall and comprehensive study of the Gene Therapy market with all its aspects influencing the growth of the market. This report is exhaustive quantitative analyses of the Gene Therapy industry and provides data for making strategies to increase market growth and effectiveness.

The global Gene Therapy Market is expected to witness a 28.32% of CAGR during the forecast period.

Scope of the report:

The report evaluates the growth rate and the Market value based on Market dynamics, growth inducing factors. The complete knowledge is based on the latest industry news, opportunities, and trends. The report contains a comprehensive Market analysis and vendor landscape in addition to a SWOT analysis of the key vendors.

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The report presents the market competitive landscape and a corresponding detailed analysis of the major vendor/key players in the market. Top Companies in the Global Gene Therapy Market: Amgen Inc., Bluebird Bio, Gilead Sciences, Inc., Novartis AG, Orchard Therapeutics, Sibiono GeneTech Co. Ltd., Spark Therapeutics LLC, UniQure N.V., and others.

Key Market Trends

Cancer is Expected to Hold Significant Market Share in the Indication Segment

The ever-rising prevalence of cancer all across the globe is majorly driving the growth of this segment. Getting genes into cancer cells has been one of the most difficult aspects of present-day gene therapy. Researchers have been working on finding new as well as better ways of doing this. Usually, the gene is taken into the cancer cell by a carrier known as a vector. The most common types of a carrier that are used in gene therapy are viruses owing to the fact that they can enter cells and eventually deliver genetic material. The viruses are generally changed so that they cannot result in any serious disease. However, they may still cause mild, flu-like symptoms. Some viruses have been modified in the laboratory so that they can target cancer cells only and not the healthy cells. Therefore, they only carry the gene into cancer cells.

North America Dominates the Market and Expected to do Same in the Forecasted Period

For gene therapy, in the United States, the DHHS (Department of Health and Human Services) has been appointed to oversee the clinical trials. Two other organizations within DHHS, the OHRP (Office for Human Research Protections) and the U.S. FDA (Food and Drug Administration), have a specific authority that is described in the CFR (Code of Federal Regulations). All investigators must obey these regulations while conducting clinical gene therapy trials. The increasing approval of various gene therapies by the afore-mentioned organizations is driving the market growth in the region. For instance, in May 2019, AveXis, a Novartis company, announced that the US FDA had approved Zolgensma (onasemnogene abeparvovec-xioi) for treating pediatric patients of less than 2 years of age having spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

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Influence of the Gene Therapy market report

Comprehensive assessment of all opportunities and risks in the Gene Therapy market.

Gene Therapy market recent innovations and major events.

A Detailed study of business strategies for the growth of the Gene Therapy market-leading players.

Conclusive study about the growth plot of the Gene Therapy market for forthcoming years.

In-depth understanding of Gene Therapy market-particular drivers, constraints, and major micro markets.

Favorable impression inside vital technological and market latest trends striking the market report.

What are the market factors that are explained in the report?

Key Strategic Developments: The study also includes the key strategic developments of the market, comprising R&D, new product launch, M&A, agreements, collaborations, partnerships, joint ventures, and regional growth of the leading competitors operating in the market on a global and regional scale.

Key Market Features: The report evaluated key market features, including revenue, price, capacity, capacity utilization rate, gross, production, production rate, consumption, import/export, supply/demand, cost, market share, CAGR, and gross margin. In addition, the study offers a comprehensive study of the key market dynamics and their latest trends, along with pertinent market segments and sub-segments.

Analytical Tools: The Gene Therapy Market report includes the accurately studied and assessed data of the key industry players and their scope in the market by means of a number of analytical tools. The analytical tools such as Porters five forces analysis, SWOT analysis, feasibility study, and investment return analysis have been used to analyze the growth of the key players operating in the market.

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Gene Therapy Market 2020 After COVID-19 Developments Analysis of Best Key Players Amgen Inc., Bluebird Bio, Gilead Sciences, Inc. - The Daily...

The Gene Therapy Market research report recently presentedby Prophecy Market Insights which provides reliable and sincere insights related to the various segments and sub-segments of the market. The market study throws light on the various factors that are projected to impact the overall dynamics of the Gene Therapy market over the forecast period (2019-2029).

Influences of the market report:

An executive summary provides the markets definition, application, overview, classifications, product specifications, manufacturing processes; raw materials, and cost structures.

Market Dynamics offers drivers, restraints, challenges, trends, and opportunities of the Gene Therapy market

Segment Level Analysis in terms of types, product, geography, demography, etc. along with market size forecast

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Regional and Country- level Analysis different geographical areas are studied deeply and an economical scenario has been offered to support new entrants, leading market players, and investors to regulate emerging economies. The top producers and consumers focus on production, product capacity, value, consumption, growth opportunity, and market share in these key regions, covering

Australia, New Zealand, Rest of Asia-Pacific

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Segmentation Overview:

The Gene Therapy research study comprises 100+ market data Tables, Graphs & Figures, Pie Chat to understand detailed analysis of the market. The predictions estimated in the market report have been resulted in using proven research techniques, methodologies, and assumptions. This Gene Therapy market report states the market overview, historical data along with size, growth, share, demand, and revenue of the global industry.

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Competitive landscape Analysis provides mergers and acquisitions, collaborations along with new product launches, heat map analysis, and market presence and specificity analysis.

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Complete access to COVID-19 Impact on the Gene Therapy market dynamics, key regions, market size, growth rate and forecast to 2029

The report on the Gene Therapy market includes an assessment of the market, trends, segments, and regional markets. Overview and dynamics have been included in the report.

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Gene TherapyMarket by Top Manufacturers:

GlaxoSmithKline plc, Bluebird Bio, Inc., Adaptimmune Therapeutics plc, Celgene Corporation, Shanghai Sunway Biotech Co. Ltd., Merck KGaA, Transgene SA, and OncoGenex Pharmaceuticals, Inc.

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Product Portfolio Analysis and Technological Development of Gene Therapy Market during the forecasted period - PRnews Leader

Every time a cell inside your body replicates, a slither of your youth crumbles to dust. This occurs via the shortening oftelomeres, structures that 'cap' the tips of our chromosomes.

Now, scientists in Israel say they've been able to reverse this process and extend the length of telomeres in a small study involving 26 patients.

The participants sat in a hyperbaric oxygen chamber for five 90 minutes sessions per week over three months, and as a result, some of their cell's telomeres were extended by up to 20 percent.

It's an impressive claim - and something many other researchers have attempted in the past without success. But of course it's worth flagging that this is a small sample size, and the results will need to be replicated before we can get too excited.

However, the fact that hyperbaric oxygen therapy appears to affect telomere length is a compelling link worth investigating further.

Lead researcher Shair Efrati, a physician from the Faculty of Medicine and Sagol School of Neuroscience at Tel Aviv University, explained to ScienceAlert how the inspiration behind their experiment was somewhat out of this world.

"After the twin experiment done by NASA, where one of the twins was sent out to the outer space and the other stayed on Earth, demonstrated a significant difference in their telomere length we have realised that changes in the outside environment may affect the core cellular changes that happens along ageing," said Efrati.

Telomeres are repeating chunks of code that act as the DNA equivalent of the plastic or metal aglet capping the end of a shoelace.

They copy themselves along with the rest of the chromosomes whenever a cell divides. Yet with every replication, tiny fragments of code from the very tip of the sequence fail to make it into the new copy, leaving the freshly minted chromosome a touch shorter than its predecessor.

As anybody who has lost the cap of their shoelace knows, it doesn't take long for the shoelace to lose its integrity. Similarly, shorter telomeres put sequences further down the chromosome at higher risk of hazardous mutations.

These mutations coincide with changes that predispose us to a bunch of age-related conditions, not least of all diseases such as cancer.

That's not necessarily to say that we age because our telomeres shrink, but there is a connection between telomere length and health that researchers are keen to investigate further.

"Longer telomeres correlates with better cellular performance," Efriti explained.

There are plenty of ways to accelerate the erosion of our telomeres. Failing to get adequate sleepcould do it, as might chowing down on too much processed food, and maybe even having kids.

Slowing down the loss takes a bit more effort, but engaging in regular exercise and eating well are sound bets if you want your chromosomes to remain as long as possible.

A real achievement would be to flip our chromosomal hourglass completely and return lost sections of telomere. The fact that high-turnover tissues lining our gut do this naturally using an enzyme called telomerase has fuelled research over the years.

There have been plenty of milestones in attempts to achieve this task. Gene therapy in mice has shown it could one day be feasible in humans. More recently, stem cells from a supercentenarian woman had their telomeres completely reset outside of her body.

Some studies have found potential for tiny increases of maybe a few percent with provision ofnutritional supplementssuch asvitamin D.

But while there are plenty of hyped promises of reversing aging in living humans already on the market, the reality of science-backed therapies we can use to give us the telomeres of a 20-year-old has been underwhelming.

Which is why the latest study is attracting so much attention. Far from a measly two or three percent, this latest study found telomeres in white blood cells taken from 26 subjects had regained around a fifth of their lost length.

The key, it seems, is hyperbaric oxygen therapy (HBOT) the absorbing of pure oxygen while sitting in a pressurised chamber for extensive periods; in this case, five 90 minute sessions per week over three months.

HBOT has attracted controversy in the past for claims it could treat a range of conditions.It's usually the kind of therapy you'd give a diver who came up too fast from the ocean depths, or to kill off oxygen-sensitive microbes in a wound that just won't heal any other way.

But oxygen-rich environments are also behind a weird paradox, one where the body desperately stirs up a host of genetic and molecular changes that typically occur in a low oxygen one.

In this study, the researchers were able to show that the genetic changes provoked by the HBOT has extended telomeres, and also had a potentially positive effect on the health of the tissues themselves.

A slightly smaller sample of volunteers also showed a significant decrease in the number of senescentT cells, tissues that form a vital part of our immune system's targeted response against invaders.

Whether you'd sit in a small tank every day for a quarter of a year is a matter of preference, but future research could help make the whole process a touch more efficient, at least for some.

"Once we have demonstrated the reverse ageing effect on the study cohort using predefined HBOT protocol, further studies are needed in order to optimise the specific protocol per individual," Efrati told ScienceAlert.

In a press release from The Sagol Center for Hyperbaric Medicine and Research, Efrati says understanding telomere shortening is "considered the 'Holy Grail' of the biology of aging".

As significant as telomere shrinking seems to be, the failure of our biology as we age is no doubt a complicated matter involving far more than lost pieces of chromosomes.

Reactivation of telomerase is also a trick used by cancers to remain ahead of the growth-curve, making this holy grail a potentially poisoned chalice we need to understand better before drinking too heavily from.

Excitingly, research like this will help us develop a better picture of the aging process.

This research was published in Aging.

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In a First, Scientists Say They've Partially Reversed a Cellular Aging Process in Humans - ScienceAlert

Embargoed by the Journal of Clinical Investigation until

Thursday, Nov. 19, 2020 at 12 p.m. EST

Newswise November 19, 2020 - Canadian scientists looking at a rare genetic mutation think they may have discovered the proverbial fountain of youth. But its not for everyone: so far, the mutation has only been found in a handful of French-Canadian families.

Called PCSK9Q152H, the mutation of the PCSK9 gene was initially thought to protect against cardiovascular diseases. Recent studies reveal that it may protect against other human illnesses, mainly liver diseases. It may allow the lucky PCSK9Q152H mutant subjects to stay in good health and live longer, the researchers say in a study published today in the prestigious Journal of Clinical Investigation.

The work was led by vascular biologists Richard Austin and Paul Lebeau of McMaster University and by Montreal Clinical Research Institute endocrinologist Michel Chrtien, an emeritus professor at the University of Montreal.

Gene therapy next?

These are exciting findings what weve found may represent a kind of fountain of youth, said Austin, a medical professor and one of the senior authors of the study. Now we want to see whether we can come up with a gene therapy approach to overexpress this specific mutant gene variant in the liver, and thereby offer an innovative treatment for a number of diseases that normally lead to early death.

News of the PCSK9Q152H mutation was first published in 2011, after Chrtien, the current studys lead clinical investigator, discovered it in a French-Canadian family. Mainly expressed in the liver, the mutated gene lowers a persons plasma LDL-cholesterol (bad) and prevents cardiovascular diseases. Chrtien and his IRCM colleague Hanny Wassef found it in two other large French-Canadian families, as well.

People carrying this gene mutation are surprisingly healthy well into their late 80s and mid-90s. In addition to their low plasma LDL-cholesterol and their low risk of cardiovascular diseases, their liver function is completely normal when measured through imaging and a complete medical evaluation, the Montreal scientists found.

Until now, however, the underlying mechanism by which the mutation represented a health benefit other than cardiovascular was a mystery.

A surprising protective effect

In the new study, the McMaster researchers show that overexpression of this gene variant in the livers of mice who dont carry PCSK9Q152H had a surprising protective effect against injury and dysfunction of their liver. As well, overexpression led to a large reduction in their circulating PCSK9 levels just as it does in people, decreasing individuals bad cholesterol and keeping them in good cardiovascular health.

When we initiated these studies, we had speculated that introducing the mutant PCSK9Q152H protein into the liver of mice would cause liver injury or dysfunction, Austin said. But to our amazement, added Lebeau, overexpression of the mutant gene variant in the livers of mice failed to cause stress in the cellular manufacturing and packaging system called the endoplasmic reticulum, or ER, and actually protected against ER stress-induced liver injury.

In their laboratory, the McMaster scientists went on to show that the mutant gene variant acts as a unique co-chaperone protein to stabilize several well-known ER chaperones, namely GRP78 and GRP94, and to increase their protective activity against liver damage.

Particularly gratifying results

These results from Dr. Austins group are particularly gratifying since they experimentally explain that this gene mutation, known to lower cardiovascular accidents, also protects against liver injury and dysfunction, even in individuals who are in their late 80s and mid-90s, said Chrtien, also an emeritus scientist at the Ottawa Hospital Research Institute.

Furthermore, these findings should allow us to determine whether this unique mutation provides additional protection against liver diseases such as cancer, over and above its protective effect against cardiovascular accidents.

About this study

The article The loss-of-function PCSK9Q152H variant increases ER chaperones GRP78 and GRP94 and protects against liver injury , by Paul Lebeau and his collaborators will be published in the Journal of Clinical Investigation, on January 4, 2021.

The study was funded in part by the Canadian Institutes of Health Research and the Heart and Stroke Foundation of Canada, the Fondation J-Louis Lvesque, the Richard and Edith Strauss Foundation, the Fondation Notre-Dame de Zeitoun, and the Aclon Foundation.

-30-

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A gene mutation that protects against disease - Newswise

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Personalized Gene Therapy Treatment Market Dynamicas and Opportunity Analysis till 2030 - TechnoWeekly

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Hemophilia Gene Therapy Market Industry Outlines, Future Trends, Insight And Quality Analysis And Sustainable Growth Strategy Over 2026 - PRnews...

Just a few months after withdrawing its IPO filing, 4D Molecular Therapeutics is seeking to go public once again.

The Emeryville, CA-based company submitted a new S-1 on Tuesday, detailing plans for a $75 million raise as it aims for the second time to hit the Nasdaq. 4DMT had previously sought a $100 million IPO back in September 2019, but withdrew the filing in July of this year after completing a $75 million Series C in June.

Should 4DMT complete the transition to a public company this time around, theyll join a crowded IPO party thats lasted nearly the entire year.

Nasdaq head of healthcare listings Jordan Saxe provided the most recent tally for biotech IPOs in late October, counting 72 companies going public at the time. Combined, those outlets have raised roughly $13.2 billion. The debuts have slowed since the summer, but Saxe pegged a fair estimate of 75 IPOs and just under $14 billion in proceeds to round out 2020.

Several factors have contributed to this years wave, Saxe previously told Endpoints News, as the Covid-19 pandemic has highlighted innovation and crossover investors have steadily increased biotech investments in the second half of the 2010s. The pandemic economy has also made biotech companies more appealing given that theyre less reliant on quarter-to-quarter sales numbers.

In the last four years, only 2018 comes close in terms of the sheer amount of biotechs shooting for Wall Street. That years tab totaled 56 IPOs, according to independent analyst Brad Loncar.

Within the new S-1, 4DMT didnt provide too much detail about how much money theyd spend on each of their programs. The company did list, however, that ongoing clinical trials for their leading programs 4D-310 and 4D-125 would be their top priority. Both of those candidates are currently in Phase I/II with data likely coming next year.

The main research driving the company has been building out a base of more than a billion vector capsid sequences, which CEO David Kirn said in June needed years to take place. 4DMT needed that time to run the sequences through non-human primates to see which shells were the least toxic and most likely to prevent antibody resistance.

By doing so, the biotech hopes this screening model can help find the capsids most suitable for the vector delivery of gene therapies.

4DMTs lead candidate, 4D-310, is intended to treat Fabry disease, with the goal of initially treating early onset versions before expanding into severe, late-onset patients. 4D-125, meanwhile, has the goal of treating an inherited vision loss disorder called XLRP. Roche has partnered with 4DMT to in-license the program before it begins a pivotal trial.

The biotech is also conducting a Phase I study in 4D-110, which is targeted at patients with choroideremia related to mutations in the CHM gene.

Read the original here:
4DMT shoots for a $75M IPO, its second attempt to go public with its gene therapy vector programs - Endpoints News

BASEL, Switzerland & MINNEAPOLIS--(BUSINESS WIRE)--Be The Match BioTherapies and Lonza:

Quote from Alberto Santagostino, SVP, Head of Cell and Gene Therapy, Lonza:

"Be The Match BioTherapies brings deep experience in overcoming supply chain and logistical challenges impacting developers of cell and gene therapies, including factors that delay patients' ability to access the treatments they need. We're pleased to add their team to our growing network of strategic partners and look forward to working together to cultivate a more seamless, streamlined ecosystem for cell and gene therapy development."

Quote from Chris McClain, MBA, SVP, Sales and New Business Development, Be The Match BioTherapies:

"Lonza plays a central role in Be The Match BioTherapies' pursuit of accelerating patient access to high-quality manufactured cell therapies. This collaboration will allow our teams to combine our substantial expertise and resources across the cell therapy supply chain to ease the logistical burden for cell and gene therapy developers globally. By offering the ability to leverage a fully integrated cell and gene therapy supply chain, we can ultimately provide a brighter future for patients."

Be The Match BioTherapies, an organization offering solutions for companies developing and commercializing cell and gene therapies, and Lonza today announced a strategic partnership to integrate industry-leading solutions in CGT. The goal of the collaboration is to improve efficiency across the CGT supply chain, inclusive of apheresis network management, healthy donor tissue supply, manufacturing and, where appropriate, supply chain management and logistics.

The partnership establishes Be The Match BioTherapies and Lonza as preferred partners and is aimed at supporting the companies' shared goal of providing end-to-end solutions that streamline the development of cell and gene therapies across the CGT supply chain. Lonza and Be The Match BioTherapies will integrate each other's services in their respective offerings to provide a seamless offering to customers.

This collaboration builds on current partnerships announced by Lonza and Be The Match BioTherapies to build a seamless, vein-to-vein network for customers and their patients. Partners of Lonza include Cryoport, a leading temperature-controlled supply chain company, and Vineti, a company developing the first commercial, configurable, cloud-based digital platform to orchestrate advanced therapy supply chains. Be The Match BioTherapies brings expertise in successfully delivering more than 100,000 cell therapies, expertise in logistics, managing apheresis networks, and the ability to provide high-quality cellular source material from the Be The Match Registry which offers the world's largest, most ethnically diverse listing of more than 22 million potential blood and marrow donors, with Lonza's industry-leading capabilities in cell and gene therapy manufacturing

About Lonza

At Lonza, we combine technological innovation with world class manufacturing and process excellence. Together, these enable our customers to deliver their discoveries in the healthcare, preservation, and protection sectors.

We are a preferred global partner to the pharmaceutical, biotech and specialty ingredients markets. We work to prevent illness and promote a healthier world by enabling our customers to deliver innovative medicines that help treat or even cure a wide range of diseases. We also offer a broad range of microbial control solutions, which help to create and maintain a healthy environment.

Founded in 1897 in the Swiss Alps, Lonza today operates in 120 sites and offices in more than 35 countries. With approximately 15,500 full-time employees, we are built from high-performing teams and of individual employees who make a meaningful difference to our own business, as well as the communities in which we operate. The company generated sales of CHF 5.9 billion in 2019 with a CORE EBITDA of CHF 1.6 billion. Find out more at http://www.lonza.com and follow us on Twitter @LonzaGroup or Facebook @LonzaGroupAG.

About Be The Match BioTherapies

Be The Match BioTherapies is the only cell and gene therapy solutions provider with customizable services to support the end-to-end cell therapy supply chain. Backed by the industry-leading experience of the National Marrow Donor Program (NMDP)/Be The Match, and a research partnership with the CIBMTR (Center for International Blood and Marrow Transplant Research), the organization designs solutions that advance the development of cell and gene therapies across the globe.

Be The Match BioTherapies is dedicated to accelerating patient access to life-saving cell and gene therapies by providing high-quality cellular source material from the Be The Match Registry, the world's largest and most diverse registry of more than 22 million potential blood stem cell donors and more than 300,000 umbilical cord blood units. Through established relationships with apheresis, marrow collection and transplant centers worldwide, the organization develops, onboards, trains and manages expansive collection networks to advance cell therapies. Be The Match BioTherapies uses a proven integrated model of both cell therapy supply chain and logistics managers, complimented by regulatory compliance experts to successfully transport and deliver life-saving therapies across the globe. Through the CIBMTR, Be The Match BioTherapies extends services beyond the cell therapy supply chain to include long-term follow-up tracking for the first two FDA-approved CAR-T therapies.

For more information, visit http://www.BeTheMatchBioTherapies.com or follow Be The Match BioTherapies on LinkedIn or Twitter at @BTMBioTherapies.

Additional Information and Disclaimer

Lonza Group Ltd has its headquarters in Basel, Switzerland, and is listed on the SIX Swiss Exchange. It has a secondary listing on the Singapore Exchange Securities Trading Limited ("SGX-ST"). Lonza Group Ltd is not subject to the SGX-ST's continuing listing requirements but remains subject to Rules 217 and 751 of the SGX-ST Listing Manual.

Certain matters discussed in this news release may constitute forward-looking statements. These statements are based on current expectations and estimates of Lonza Group Ltd, although Lonza Group Ltd can give no assurance that these expectations and estimates will be achieved. Investors are cautioned that all forward-looking statements involve risks and uncertainty and are qualified in their entirety. The actual results may differ materially in the future from the forward-looking statements included in this news release due to various factors. Furthermore, except as otherwise required by law, Lonza Group Ltd disclaims any intention or obligation to update the statements contained in this news release.

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Lonza and Be The Match BioTherapies Partner to Expand Vein-to-Vein Cell and Gene Therapy Supply Chain Network - Business Wire

CAMBRIDGE, Mass., Nov. 10, 2020 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq: GBIO) is an innovative genetic medicines company creating a new class of non-viral gene therapy. Today the company reported recent business highlights and third quarter financial results.

2020 continues to be a year of progress and execution for Generation Bio as we advance our non-viral gene therapy approach, said Geoff McDonough, M.D., president and chief executive officer of Generation Bio.Despite the challenges of the COVID-19 pandemic, we remain on-track to advance our lead programs into IND-enabling preclinical development next year. We believe our strong cash balance positions us well to execute on our ambitions into 2023.

Recent Business Highlights

This period marks an expansion of our focus beyond our platform to include preclinical development and readiness for the clinic. To support this effort, I am pleased to announce the appointment of Tracy Zimmermann to chief development officer. Tracy will lead our pre-clinical development programs across the portfolio, building on the excellent foundation she has created since joining Generation Bio in 2018. Tracys new role allows for Doug Kerr to focus on building our clinical development capabilities as chief medical officer. Together with Matt Stanton, our chief scientific officer, Tracy and Doug make a terrific, complementary leadership team for our R&D work, Dr. McDonough said. A summary of the leadership appointments follows.

Dr. McDonough continued, Separately, Mark Angelino, our chief operating officer and co-founder, will undertake a planned transition from Generation Bio to return to early stage company formation work in early 2021. Although too soon for farewells, we are indebted to Mark for his vision and leadership in forming and building our community here.

Selected Anticipated Company Milestones

Upcoming Investor Conference Presentations

Management will present at two upcoming investor conferences:

Live webcasts of the presentation and the fireside chat will be available in the investor section of the company's website atwww.generationbio.com. The webcasts will be archived for 60 days following the presentations.

Financial Results

About Generation Bio

Generation Bio is an innovative genetic medicines company focused on creating a new class of non-viral gene therapy to provide durable, redosable treatments for people living with rare and prevalent diseases. The companys non-viral platform incorporates a proprietary, high-capacity DNA construct called closed-ended DNA, or ceDNA; a cell-targeted lipid nanoparticle delivery system, or ctLNP; and an established, scalable capsid-free manufacturing process. The platform is designed to enable multi-year durability from a single dose of ceDNA and to allow titration and redosing if needed. The ctLNP is designed to deliver large genetic payloads, including multiple genes, to specific tissues to address a wide range of indications. The companys efficient, scalable manufacturing process supports Generation Bios mission to extend the reach of gene therapy to more people, living with more diseases, in more places around the world.

For more information, please visit http://www.generationbio.com.

Forward-Looking Statements

Any statements in this press release about future expectations, plans and prospects for the Company, including statements about its strategic plans or objectives, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the identification and development of product candidates, including the conduct of research activities, the initiation and completion of preclinical studies and clinical trials and clinical development of the Companys product candidates; uncertainties as to the availability and timing of results from preclinical studies and clinical trials; whether results from preclinical studies will be predictive of the results of later preclinical studies and clinical trials; expectations for regulatory approvals to conduct trials or to market products; challenges in the manufacture of genetic medicine products; the Companys ability to obtain sufficient cash resources to fund the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements; the impact of the COVID-19 pandemic on the Companys business and operations; as well as the other risks and uncertainties set forth in the Risk Factors section of the Companys most recent quarterly report on Form 10-Q, and in subsequent filings the Company may make with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Companys views as of the date hereof. The Company anticipates that subsequent events and developments will cause the Companys views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Companys views as of any date subsequent to the date on which they were made.

Contacts:

InvestorsChelcie ListerTHRUST Strategic Communicationschelcie@thrustsc.com910-777-3049

MediaStephanie SimonTenBridge Communicationsstephanie@tenbridgecommunications.com617-581-9333

GENERATION BIO CO.CONSOLIDATED BALANCE SHEET DATA(unaudited)(in thousands)

GENERATION BIO CO.CONSOLIDATED STATEMENTS OF OPERATIONS(unaudited)(in thousands, except share and per share data)

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Generation Bio Reports Third Quarter 2020 Business Updates and Financial Results - GlobeNewswire

Final Report will add the analysis of the impact of COVID-19 on this industry

Global Gene Therapy Market forecast 2020-2024 market report includes types, applications, regions analysis and discussion of important industry trends, market share estimates and profiles of the leading industry players. Gene Therapy Market share report covers the manufacturers , price, revenue, gross profit, these data help the end user know about the competitors better.

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The content of the study subjects, includes a total of 15 chapters:

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Table of Contents of Gene Therapy Market:

1 Market Overview

1.1 Gene Therapy Introduction

1.2 Market Analysis by Type

1.2.1 Type 1

1.2.2 Type 2

1.3 Market Analysis by Applications

1.3.1 Application 1

1.3.2 Application 2

1.4 Market Analysis by Regions

1.4.1 North America (United States, Canada and Mexico)

1.4.2 Europe (Germany, France, UK, Russia and Italy)

1.4.3 Asia-Pacific (China, Japan, Korea, India and Southeast Asia)

1.4.4 South America, Middle East and Africa

1.4.4.5 Turkey Market States and Outlook (2014-2024)

1.5 Market Dynamics

1.5.1 Market Opportunities

1.5.2 Market Risk

1.5.3 Market Driving Force

2 Manufacturers Profiles

2.1 Manufacture

2.1.1 Business Overview

2.1.2 Gene Therapy Type and Applications

2.1.2.1 Product A

2.1.2.2 Product B

2.1.3 Manufacture Gene Therapy Sales, Price, Revenue, Gross Margin and Market Share (2019-2019)

3 Global Gene Therapy Sales, Revenue, Market Share and Competition by Manufacturer (2019-2019)

3.1 Global Gene Therapy Sales and Market Share by Manufacturer (2028-2019)

3.2 Global Gene Therapy Revenue and Market Share by Manufacturer (2018-2019)

3.3 Market Concentration Rate

3.3.1 Top 3 Gene Therapy Manufacturer Market Share in 2019

3.3.2 Top 6 Gene Therapy Manufacturer Market Share in 2019

3.4 Market Competition Trend

13 Sales Channel, Distributors, Traders and Dealers

13.1 Sales Channel

13.1.1 Direct Marketing

13.1.2 Indirect Marketing

13.1.3 Marketing Channel Future Trend

13.2 Distributors, Traders and Dealers

14 Research Findings and Conclusion

15 Appendix

15.1 Methodology

15.2 Data Source

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Gene Therapy Market 2020 Size, Share, Trends Analysis Report by Application, Region (North America, South America, Asia, and Europe) and Forecasts to...

Global Hemophilia Gene Therapy Market report explains the fundamental industry aspects and market statistics. The latest technological advancement, market plans, policies, growth opportunities and industry risks are elaborated. The two important segments of the report namely market revenue in (USD Million) and market size (k MT) is described. Hemophilia Gene Therapy Industry scope, market concentration and Hemophilia Gene Therapy presence across different topographies is presented in detail.

A visionary perspective about Hemophilia Gene Therapy Industry covers the geographical continents namely North America, European countries, Asia-Pacific, South America, Middle East & Africa. In the next segment, prominent Hemophilia Gene Therapy industry players, their company profiles, product details, and market size is described. Also, the SWOT analysis of these players, business plans & strategies are covered. The product definition, Hemophilia Gene Therapy classification, type and cost structures are covered.

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Hemophilia Gene Therapy Market Leading Players:

BioMarinFreeline TherapeuticsuniQureBioverativSpark TherapeuticsSangamo TherapeuticsUltragenyxShire PLC

Market Segment Analysis

By Types:

Hemophilia AHemophilia B

By Applications:

Hemophilia A Gene TherapyHemophilia B Gene Therapy

On a regional level, Hemophilia Gene Therapy production value and growth rate from 2015-2019 is presented. The detailed analysis of emerging industry segments and sub-segments are explained. Macro-economic plans & policies, economic status, cost structures and value chain analysis is covered. The Hemophilia Gene Therapy competitive landscape view, manufacturing base, production process analysis and upstream raw materials are evaluated.

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The gross margin, consumption pattern, growth rate of Hemophilia Gene Therapy is studied precisely. The top industry players are covered on a regional level and country level with the analysis of their revenue share from 2015-2019. Furthermore, forecast Hemophilia Gene Therapy industry status is determined by analysis of expected market share, volume, value and development rate. The forecast Hemophilia Gene Therapy industry view is presented from 2020-2027.

Abstract of implemented Research methodology and data sources used to derive Hemophilia Gene Therapy Market statistics:

The information presented in Hemophilia Gene Therapy Report includes qualitative and quantitative analysis. Under the qualitative analysis part, Hemophilia Gene Therapy status, trends, manufacturing base, distribution channels, market position, a competitive structure is covered. Also, complete details on product development, cost structures, growth opportunities, industry plans and policies are analysed. Under the qualitative analysis part, market size (from 2015-2019), sales, revenue, gross margin statistics, revenue is covered. Also, industry size by Hemophilia Gene Therapy type, application, demand and supply scenario, and economic status is explained.

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Our research methodology comprises of 80% primary and 20% secondary research. To derive the supply side Hemophilia Gene Therapy industry statistics, we conduct an interview with competitors, manufacturers, OEMs, raw material providers and others. To derive sales statistics, Hemophilia Gene Therapy industry information is gathered from distributors, traders and market dealers. Similarly, to analyse demand-side statistics we interview the end users, consumers and conduct custom surveys.

Under secondary research technique, the Hemophilia Gene Therapy production, sales and consumption statistics are gathered from company reports, annual publications, SEC filings, government data, case studies, custom groups and demographics.

Abstract of the report:

Remarkable Attributes Of Hemophilia Gene Therapy Report:

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Global Hemophilia Gene Therapy Market Grow with a CAGR 2020-2027 Focuses On Top Companies With Regional Analysis - The Think Curiouser

Final Report will add the analysis of the impact of COVID-19 on this industry

Global Cancer Gene Therapy Market report 2020 analysis in-depth development status, market size, growth, production and services, current and future trends. This report provides market dynamics, drivers, competitive landscape, opportunities and latest strategies to help the emerging players to make crucial decisions. The Cancer Gene Therapy research report covers niche markets, potential risks and comprehensive competitive strategy analysis in different fields.

The Cancer Gene Therapy market contains top manufactures with business overview, product sales, price and gross margin for the forecast period. This report analyzes in detail for the potential risks and opportunities which can be focused on.

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Top Key Manufactures of Cancer Gene Therapy Market:

The global impacts of the coronavirus disease 2019 (COVID-19) are already starting to be felt, and will significantly affect the Cancer Gene Therapy in 2020. This report helps users in comprehending the key product segments and their future developments. And also provide the perspectives for the impact of COVID-19 from the long and short term. The Cancer Gene Therapy market declared the influence of the crisis on the industry chain, especially for marketing channels. Timely update the industry economic revitalization plan of the country-wise government.

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The Cancer Gene Therapy market analysis and forecast by types, by applications, by regions, SWOT analysis which is included the industry growth rate, market constraints and challenges, characteristics and business expansion plans for the upcoming years.

on the basis of types, the Cancer Gene Therapy market from 2015 to 2025 is primarily split into:

on the basis of applications, the Cancer Gene Therapy market from 2015 to 2025 covers:

The report assesses the key opportunities in the market and the detailed analysis of consumption, revenue, market share and growth rate, historic and forecast (2015-2025).

Geographical Segmentation:

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Years considered for this report:

Key questions answered in this report:

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Detailed TOC of Cancer Gene Therapy Market Report Growth, Challenges and Forecast to 2020-2025:

1 COVID-19 Impact on Cancer Gene Therapy Market Overview

1.1 Product Definition and Market Characteristics

1.2 Global Cancer Gene Therapy Market Size

1.3 Cancer Gene Therapy market Segmentation

1.4 Global Macroeconomic Analysis

1.5 SWOT Analysis

2. COVID-19 Impact on Cancer Gene Therapy Market Dynamics

2.1 Cancer Gene Therapy Market Drivers

2.2 Cancer Gene Therapy Market Constraints and Challenges

2.3 Emerging Market Trends

2.4 Impact of COVID-19

2.4.1 Short-term Impact

2.4.2 Long-term Impact

3 Associated Industry Assessment

3.1 Supply Chain Analysis

3.2 Industry Active Participants

3.2.1 Suppliers of Raw Materials

3.2.2 Key Distributors/Retailers

3.3 Alternative Analysis

Continued

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Global Cancer Gene Therapy Market Analysis by Size: 2025, Impact of Covid-19 on Industry Chain, Market Growth, Drivers | Business Overview with Top...

The global cancer gene therapy market is estimated to surpass $4,188.5 million by 2027, exhibiting a CAGR of 23.0% from 2020 to 2027.

The report aims to offer a clear picture of the current scenario and future growth of the global Cancer Gene Therapy market. The report provides scrupulous analysis of global market by thoroughly reviewing several factors of the market such as vital segments, regional market condition, market dynamics, investment suitability, and key players operating in the market. Besides, the report delivers sharp insights into present and forthcoming trends & developments in the global market.

The report articulates the key opportunities and factors propelling the global Cancer Gene Therapy market growth. Also, threats and limitations that have the possibility to hamper the market growth are outlined in the report. Further, Porters five forces analysis that explains the bargaining power of suppliers and consumers, competitive landscape, and development of substitutes in the market is also sketched in the report.

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The report reveals various statistics such as predicted market size and forecast by analyzing the major factors and by assessing each segment of the global Cancer Gene Therapy market. Regional market analysis of these segments is also provided in the report. The report segments the global market into four main regions including Asia-Pacific, Europe, North America, and LAMEA. Moreover, these regions are sub-divided to offer an exhaustive landscape of the Cancer Gene Therapy market across key countries in respective regions. Furthermore, the report divulges some of the latest advances, trends, and upcoming opportunities in every region.

Furthermore, the report profiles top players active in the global Cancer Gene Therapy market. A comprehensive summary of 10 foremost players operating in the global market is delivered in the report to comprehend their position and footmark in the industry. The report highlights various data points such as short summary of the company, companys financial status and proceeds, chief company executives, key business strategies executed by company, initiatives undertaken & advanced developments by the company to thrust their position and grasp a significant position in the market.

RESEARCH METHODOLOGY

The research report is formed by collating different statistics and information concerning the Cancer Gene Therapy market. Long hours of deliberations and interviews have been performed with a group of investors and stakeholders, including upstream and downstream members. Primary research is the main part of the research efforts; however, it is reasonably supported by all-encompassing secondary research. Numerous product type literatures, company annual reports, market publications, and other such relevant documents of the leading market players have been studied, for better & broader understanding of market penetration. Furthermore, medical journals, trustworthy industry newsletters, government websites, and trade associations publications have also been evaluated for extracting vital industry insights.

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KEY MARKET BENEFITS

KEY MARKET SEGMENTS

The global Cancer Gene Therapy market is segmented on the basis of the following:

Global Cancer Gene Therapy Market By Product Type:

Global Cancer Gene Therapy Market By Applications:

Global Cancer Gene Therapy Market By Regions:

Top Leading key players stated in Global Cancer Gene Therapy Market report are:

Sirion Biotech, Vigene Biosciences, Bluebird bio, Ziopharm, Cellectis, Cobra, Finvector, Uniqure, Sarepta Therapeutics.

The report also summarizes other important aspects including financial performance, product portfolio, SWOT analysis, and recent strategic moves and developments of the leading players.

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Growth Outlook: Cancer Gene Therapy Market Is Projected to Grow at a Substantial Rate by 2027 Report by ResearchDive - The Think Curiouser

CAMBRIDGE, Mass., Nov. 09, 2020 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (NASDAQ: VYGR) today reported its third quarter 2020 financial results, program progress and corporate updates.

During the third quarter, we took important steps to advance our lead programs for Parkinsons disease and Huntingtons disease with the presentation of positive long-term clinical results and the filing of an IND, respectively. Together with our partner, Neurocrine, we are currently focused on resuming enrollment in the RESTORE-1 clinical trial for Parkinsons disease pending the requested review and assessment of patient imaging data by the DSMB. For our Huntingtons disease program, the FDA has provided clarity regarding the additional information it is requesting pursuant to our IND filing. We plan to work with the FDA to respond to these requests to allow for an IND clearance and the start of VY-HTT01s clinical evaluation, said Andre Turenne, President and CEO of Voyager. We look forward to continuing our progress on these two important programs, along with our broadening AAV gene therapy portfolio to treat severe neurological diseases.

Recent Program and Corporate Highlights

VY-AADC (NBIb-1817) for Parkinsons Disease

VY-HTT01 for Huntingtons Disease

Novel AAV Capsid Discovery Program

Recent Corporate Updates

Third Quarter 2020 Financial Results

Financial Guidance

About Voyager TherapeuticsVoyager Therapeutics is a clinical-stage gene therapy company focused on developing life-changing treatments for severe neurological diseases. Voyager is committed to advancing the field of AAV gene therapy through innovation and investment in vector engineering and optimization, manufacturing, and dosing and delivery techniques. Voyagers wholly owned and partnered pipeline focuses on severe neurological diseases for which effective new therapies are needed, including Parkinsons disease, Huntingtons disease, Friedreichs ataxia, and other severe neurological diseases. For more information on Voyager Therapeutics, please visit the companys website at http://www.voyagertherapeutics.com or follow @VoyagerTx on Twitter and LinkedIn.

Voyager Therapeutics is a registered trademark, and TRACER is a trademark, of Voyager Therapeutics, Inc.

Forward-Looking Statements This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as may, might, will, would, should, expect, plan, anticipate, believe, estimate, undoubtedly, project, intend, future, potential, or continue, and other similar expressions are intended to identify forward-looking statements. For example, all statements Voyager makes regarding Voyagers ability to deliver patient imaging data to the DSMB for the RESTORE-1 Phase 2 clinical trial, the review of such data by the DSMB prior to year-end, and pending the DSMBs evaluation of such data the resumption of enrollment in the RESTORE-1 Phase 2 clinical trial; the submission by Neurocrine Biosciences of an expedited safety report relating to the RESTORE-1 clinical trial in a timely manner; Voyagers efforts to work with the FDA to resolve additional information requests relating to the IND application for VY-HTT01, the clearance of the VY-HTT01 IND application by the FDA and the initiation of a Phase 1b clinical trial of VY-HTT01; Voyagers continuing efforts in the discovery and engineering of novel AAV capsids, including progressing non-human primate studies of selected novel capsids; the contributions that will be made to Voyager by key senior level officers and a new member to the Voyager Board of Directors; the timing, progress, activities, goals and reporting of results of Voyagers preclinical programs and clinical trials and its research and development programs; the potential clinical utility of its product candidates; Voyagers ability to add new programs to its pipeline; the regulatory pathway of, and the timing or likelihood of its regulatory filings and approvals for, any of its product candidates; Voyagers anticipated financial results, including Voyagers available cash, cash equivalents and marketable debt securities; Voyagers ability to fund its operating expenses with its current cash, cash equivalents and marketable debt securities through a stated time period; and the ability of Voyager to maintain a high level of business critical activity and maintain a level of scientific leadership during the COVID-19 health crisis are forward looking statements.

All forward-looking statements are based on estimates and assumptions by Voyagers management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected. Such risks and uncertainties include, among others, the ability to provide imaging data to the DSMB for the RESTORE-1 Phase 2 clinical trial, and the ability for the DSMB to complete its evaluation and to resolve questions that may exist regarding such patient data; the ability for Voyager to meet the information requests of, and to resolve questions raised by, the FDA relating to the IND application for VY-HTT01; the ability of Voyager to progress its research and engineering program for novel capsids and to conduct non-human primate studies; the initiation and conduct of preclinical studies and clinical trials; the availability of data from preclinical studies and clinical trials, and the ability to effectively present such data; Voyagers scientific approach and general development progress; the ability to attract and retain talented contractors and employees; the ability to create and protect intellectual property; the sufficiency of cash resources; the possibility or the timing of the exercise of development, commercialization, license and other options under collaborations; the commercial potential of Voyagers product candidates; the severity and length of the COVID-19 health crisis, the imposition of governmental controls and guidance addressing the COVID health crisis; and the financial and human resources available to Voyager to manage the COVID-19 health crisis. These statements are also subject to a number of material risks and uncertainties that are described in Voyagers Annual Report on Form 10-K filed with the Securities and Exchange Commission, as updated by its subsequent filings with the Securities and Exchange Commission. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

Investors:Paul CoxVP, Investor Relations857-201-3463pcox@vygr.com

Media:Sheryl Seapy W2Opure949-903-4750sseapy@purecommunications.com

Selected Financial Information($-amounts in thousands, except per share data)(Unaudited)

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Voyager Therapeutics Announces Third Quarter 2020 Financial Results and Corporate Updates - GlobeNewswire

The gene therapy market has undergone a series of transformation from past few years. Initially gene therapy of monogenetic diseases was popular; however now the gene therapy market is gaining popularity for treatment of cancer. Cancer gene therapy refers to a process of treatment of cancer by inserting therapeutic DNA into patient. Cancer gene therapy is gaining popularity as a result of its success rate in preclinical and clinical trial stages. The most common technique for cancer gene therapy involves replacing a mutated gene that is causing cancer with a healthy copy of gene. Other technique involves inactivation of a gene that is not working properly. A new technique that is being introduced to cancer gene therapy market involves insertion of new genes into the body that would help to fight against tumor cells.

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North America followed by Europe dominates the global market for cancer gene therapy market due to large number of aging population and technological advancement in the region. Asia is expected to witness high growth in cancer gene therapy market, due to increasing government initiatives, rising economy and improvement in healthcare infrastructure in the region. Some of the key driving forces for cancer gene therapy market in emerging countries are increasing R&D investment, large pool of patients and rising government funding.

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Several unmet medical needs for treatment of cancer have encouraged R&D of cancer gene therapy. Various factors, such as increasing prevalence of cancer, rising government initiatives, increasing funding from various government and non-government organizations, are driving the global cancer gene therapy market. In addition, ethical acceptance of gene therapy for treatment of diseases and growing popularity of DNA vaccines is driving the global cancer gene therapy market. However, less awareness and high cost involved in treatment are restraining the growth of global market for cancer gene therapy.

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Innovation of some innovative therapy with better success rate is expected to offer good opportunity for cancer gene therapy. In addition, despite of high cost involved in R&D, pharmaceutical companies are showing increased interest in this field. This is expected to offer good potential for cancer gene therapy market. Currently, most of the cancer gene therapy products are in clinical trial phases. The market is expected to rise after clearance or success of these products from clinical trials. 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

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The Cancer Gene Therapy market to personify growth in the next decade - Eurowire

The Global Cell and Gene Therapy Market Report, published by Emergen Research, is an all-encompassing study of the Cell and Gene Therapy market, which emphasizes the estimated market size, share, value, volume, and futuristic outlook. The report details the major products and application rangesavailable in this market, highlighting the revenue, price, sales, production, growth rate, and market share of each segment and sub-segment. The essential market presented in the report in a tabular format is aimed at helping readers interpret the global market dynamics. The market intelligence report delivers a profound study of the Cell and Gene Therapy business domain, discussing its principal aspects, such as the import & export dynamics, production, consumption, sales channels, and consumer bases in the major regional segments. The global Cell and Gene Therapy market, which held a significant value of USD 1,180.0 Million in 2019, is projected to reach a whopping market value of USD 6,570.0 Million by 2027 at a stunning CAGR of 19.8 %.

The latest research report expounds on the profound impact of the COVID-19 pandemic on the global Cell and Gene Therapy market and its crucial segments. The report thoroughly examines the vital market-influencing factors and considers the COVID-19 pandemic as a major contributing element to this business verticals potential downturn. As per industry analysts, the Cell and Gene Therapy industry is currently reeling from the pandemics gripping effects, and remarkable changes have been observed in the market dynamics and demand trends over recent months.The financial strains being suffered by each business organization in this industry have significantly slowed down their progress. Additionally, the report assesses the pandemics overall impact on the global market growth and involves a future COVID-19 impact assessment to help readers make prudent business decisions.

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Key Players Participating in the Industry:

Spark Therapeutics LLC, Novartis AG, Gilead Sciences Inc., Bluebird Bio, GlaxoSmithKline, Celgene Corporation, Shire PLC, Sangamo Biosciences, Voyager Therapeutics, and Dimension Therapeutics

Emergen Research has segmented the global Cell and Gene Therapy Market on the basis of indication, vector type, and region:

Regional Fragmentation:

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The report succinctly analyzes the potential revenue growth of the Cell and Gene Therapy market, its development patterns, and the future market trends. Moreover, the research report specializes in the meticulous analysis of the present market scenario. It forecasts the market value, volume, drivers, restraints, demand and supply ratio, production capacity, import/export status, growth rate, and other critical aspects over the projected period.

The report offers a panoramic view of the Cell and Gene Therapy market on both global and regional levels. The study is further supported by key statistical data and industry-verified facts.The study strives to carefully gauge the present and future market growth prospects, untapped avenues, demand and consumption patterns, and the crucial factors poised to impact each market regions revenue potential. Therefore, the report scrutinizes the numerous growth trends & prospects and the significant challenges and threats that the market players might face in the upcoming years.

Target Audience of the Global Cell and Gene Therapy Market report:

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At Emergen Research, we believe in advancing with technology. We are a growing market research and strategy consulting company with an exhaustive knowledge base of cutting-edge and potentially market-disrupting technologies that are predicted to become more prevalent in the coming decade.

Our expertise umbrellas the technological environment of all major industries, and our services help you map your actions to ensure optimal yield. Our analysts utilize their market proficiency to offer actionable insights that help our clients implement profitable strategies and optimize their return on investment. Our services are wide-ranging, right from technological environment analysis to technological profiling that highlights the existing opportunities in the market you can capitalize on to stay ahead of your competitors.

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Global Cell and Gene Therapy Market to Record Significant Revenue Growth during the Forecast Period 2020-2027 | Spark Therapeutics LLC, Novartis AG,...

A complete research offering of comprehensive analysis of the market share, size, recent developments, and trends can be availed in this latest report by Big Market Research.

As per the report, theGlobal Cell and Gene Therapy Marketis anticipated to witness significant growth during the forecast period from 2020to 2027.

The report provides brief summary and detailed insights of the market by collecting data from the industry experts and several prevalent in the market. Besides this, the report offers a detailed analysis of geographical areas and describes the competitive scenario to assist investor, prominent players, and new entrants to obtain a major share of the global Cell and Gene Therapy market.

Our analysis involves the study of the market taking into consideration the impact of the COVID-19 pandemic. Please get in touch with us to get your hands on an exhaustive coverage of the impact of the current situation on the market. Our expert team of analysts will provide as per report customized to your requirement. For more connect with us at help@bigmarketresearch.com or call toll free: +1-800-910-6452

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The report presents a summary of each market segment such as type, end-user, applications, and region. With the help of pie charts, graphs, comparison tables, and progress charts a complete overview of the market share, size, and revenue, and growth patterns areaccessible in the report.

Additionally, an outline of each market segments such as end user, product type, application, and region are offered in the report.The market across various regions is analyzed in the report which includes North America, Europe, Asia-Pacific, and LAMEA.The report explains future trends and growth opportunities in every region. These insights help in understanding the global trends in the market and form strategies to be implemented in the future. Moreover, the research report profiles some of the leading companies in the global Cell and Gene Therapy industry. It mentions their strategic initiatives and offers a brief about their business. Some of the players profiled in the global Cell and Gene Therapy market include:

Key players in the Cell and Gene Therapy covers :CHIESI FarmaceuticiTego SciencesMolMedCollPlantAmgenTakeda Pharmaceutical CompanyAVITA MedicalStempeutics ResearchOsiris TherapeuticsAPAC BiotechBiosolutionVericelGilead SciencesCorestemMedipostCO.DONNovartis AGOrganogenesisOrchard TherapeuticsDendreonSpark TherapeuticsJW CreaGeneAnGesJapan Tissue EngineeringGC PharmaJCR Pharmaceuticals

Analysts have also stated the research and development activities of these companies and provided complete information about their existing products and services. Additionally, the report offers a superior view over different factors driving or constraining the development of the market.

The Cell and Gene Therapy can be split based on product types, major applications, and important countries as follows:

The basis of applications, the Cell and Gene Therapy from 2015 to 2025 covers:HospitalsWound Care CentersCancer Care CentersAmbulatory Surgical CentersOthers

The basis of types, the Cell and Gene Therapy from 2015 to 2025 is primarily split into:Cell TherapyGene Therapy

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The report clearly shows that the Cell and Gene Therapy industry has achieved remarkable progress since 2025 with numerous significant developments boosting the growth of the market. This report is prepared based on a detailed assessment of the industry by experts. To conclude, stakeholders, investors, product managers, marketing executives, and other experts in search of factual data on supply, demand, and future predictions would find the report valuable.

The report constitutes:Chapter 1 provides an overview of Cell and Gene Therapy market, containing global revenue, global production, sales, and CAGR. The forecast and analysis of Cell and Gene Therapy market by type, application, and region are also presented in this chapter.Chapter 2 is about the market landscape and major players. It provides competitive situation and market concentration status along with the basic information of these players.Chapter 3 provides a full-scale analysis of major players in Cell and Gene Therapy industry. The basic information, as well as the profiles, applications and specifications of products market performance along with Business Overview are offered.Chapter 4 gives a worldwide view of Cell and Gene Therapy market. It includes production, market share revenue, price, and the growth rate by type.Chapter 5 focuses on the application of Cell and Gene Therapy, by analyzing the consumption and its growth rate of each application.Chapter 6 is about production, consumption, export, and import of Cell and Gene Therapy in each region.Chapter 7 pays attention to the production, revenue, price and gross margin of Cell and Gene Therapy in markets of different regions. The analysis on production, revenue, price and gross margin of the global market is covered in this part.Chapter 8 concentrates on manufacturing analysis, including key raw material analysis, cost structure analysis and process analysis, making up a comprehensive analysis of manufacturing cost.Chapter 9 introduces the industrial chain of Cell and Gene Therapy. Industrial chain analysis, raw material sources and downstream buyers are analyzed in this chapter.Chapter 10 provides clear insights into market dynamics.Chapter 11 prospects the whole Cell and Gene Therapy market, including the global production and revenue forecast, regional forecast. It also foresees the Cell and Gene Therapy market by type and application.Chapter 12 concludes the research findings and refines all the highlights of the study.Chapter 13 introduces the research methodology and sources of research data for your understanding.

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Cell and Gene Therapy Market Global Industry Analysis from 2020 to 2027 Explored in Latest Research - Illadel Graff Supply

ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Nov. 04, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced data in seven patients from two ongoing Phase 1/2 clinical trials of the investigational CRISPR/Cas9 gene-editing therapy CTX001 in severe hemoglobinopathies has been accepted for an oral presentation during the Plenary Scientific Session at the annual ASH Meeting and Exposition, which will take place virtually from December 5-8, 2020. Haydar Frangoul, M.D., Medical Director of Pediatric Hematology and Oncology at Sarah Cannon Research Institute, HCA Healthcares TriStar Centennial Medical Center, will deliver the presentation on behalf of all the authors on December 6, 2020.

An abstract posted online today includes data from five patients with three months to 15 months of follow-up after CTX001 infusion in the ongoing Phase 1/2 CLIMB-111 trial in transfusion-dependent beta thalassemia (TDT) and data from two patients with three months and 12 months of follow-up in the ongoing Phase 1/2 CLIMB-121 trial in severe sickle cell disease (SCD). Additional data will be presented at ASH, including longer-duration follow-up data for the patients included in the abstract and data for additional patients with greater than three months of follow-up.

CTX001 is being investigated in these two ongoing clinical trials as a potential one-time curative therapy for patients suffering from TDT and severe SCD.

The accepted abstract is now available on the ASH conference website.

About CTX001CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patients hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for TDT patients and reduce painful and debilitating sickle crises for SCD patients.

Based on progress in this program to date, CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA). CTX001 has also been granted Orphan Drug Designation from the European Commission for both TDT and SCD, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA) for SCD.

CTX001 is being developed under a co-development and co-commercialization agreement between CRISPR Therapeutics and Vertex. Among gene-editing approaches being investigated/evaluated for TDT and SCD, CTX001 is the furthest advanced in clinical development.

About CLIMB-111The ongoing Phase 1/2 open-label trial, CLIMB-Thal-111, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with TDT. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About CLIMB-121The ongoing Phase 1/2 open-label trial, CLIMB-SCD-121, is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 12 to 35 with severe SCD. The trial will enroll up to 45 patients and follow patients for approximately two years after infusion. Each patient will be asked to participate in a long-term follow-up trial.

About the Gene-Editing Process in These TrialsPatients who enroll in these trials will have their own hematopoietic stem and progenitor cells collected from peripheral blood. The patients cells will be edited using the CRISPR/Cas9 technology. The edited cells, CTX001, will then be infused back into the patient as part of a stem cell transplant, a process which involves, among other things, a patient being treated with myeloablative busulfan conditioning. Patients undergoing stem cell transplants may also encounter side effects (ranging from mild to severe) that are unrelated to the administration of CTX001. Patients will initially be monitored to determine when the edited cells begin to produce mature blood cells, a process known as engraftment. After engraftment, patients will continue to be monitored to track the impact of CTX001 on multiple measures of disease and for safety.

About the CRISPR-Vertex CollaborationCRISPR Therapeutics and Vertex entered into a strategic research collaboration in 2015 focused on the use of CRISPR/Cas9 to discover and develop potential new treatments aimed at the underlying genetic causes of human disease. CTX001 represents the first potential treatment to emerge from the joint research program. CRISPR Therapeutics and Vertex will jointly develop and commercialize CTX001 and equally share all research and development costs and profits worldwide.

About CRISPR TherapeuticsCRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

CRISPR Therapeutics Forward-Looking StatementThis press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, as well as statements regarding CRISPR Therapeutics expectations about any or all of the following: (i) the status of clinical trials (including, without limitation, the expected timing of data releases) related to product candidates under development by CRISPR Therapeutics and its collaborators, including expectations regarding the data and plans to present data at the annual ASH meeting and exposition; (ii) the expected benefits of CRISPR Therapeutics collaborations; and (iii) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients (as is the case with CTX001 at this time) not to be indicative of final trial results; the potential that CTX001 clinical trial results may not be favorable; the potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; that future competitive or other market factors may adversely affect the commercial potential for CTX001; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q, and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

CRISPR THERAPEUTICS word mark and design logo and CTX001 are trademarks and registered trademarks of CRISPR Therapeutics AG. All other trademarks and registered trademarks are the property of their respective owners.

About VertexVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Vertex Special Note Regarding Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding the expectations and plans to present data at the annual ASH meeting and exposition, the development, including expected timeline for development, and potential benefits of CTX001, our plans and expectations for our clinical trials and clinical trial sites, and the status of our clinical trials of our product candidates under development by us and our collaborators, including activities at the clinical trial sites and potential outcomes. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs, including its programs with its collaborators, may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at http://www.vrtx.com. You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

CRISPR Therapeutics Investor Contact:Susan Kim, +1 617-307-7503susan.kim@crisprtx.com

CRISPR Therapeutics Media Contact:Rachel EidesWCG on behalf of CRISPR+1 617-337-4167reides@wcgworld.com

Vertex Pharmaceuticals IncorporatedInvestors:Michael Partridge, +1 617-341-6108orZach Barber, +1 617-341-6470orBrenda Eustace, +1 617-341-6187

Media:mediainfo@vrtx.comorU.S.: +1 617-341-6992orHeather Nichols: +1 617-839-3607orInternational: +44 20 3204 5275

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CRISPR/Cas9 Gene-Editing Therapy CTX001 for Severe Hemoglobinopathies Accepted for Plenary Presentation at the 62nd American Society of Hematology...

Avantor executives joined other industry leaders at virtual Cell & Gene Therapy Bioprocessing & Commercialization Conference

Panels discussed critical topics including process efficiency and scaling solutions

RADNOR, Pa., Nov. 2, 2020 /PRNewswire/ --Executives from Avantor Inc. (NYSE: AVTR), a leading global provider of mission-critical products and services to customers in the life sciences, advanced technologies and applied materials industries, recently provided expert insight at the Cell & Gene Therapy Bioprocessing & Commercialization Virtual Conference. Cell and gene therapy (C>) are two of the most revolutionary applications driving the biopharmaceutical industry.

In separate panel discussions with other global industry leaders, Dr. Ger Brophy, EVP, Biopharma Production at Avantor and Claudia Berrn, SVP, Business Development and Commercial Operations at Avantor, each addressed novel considerations and challenges facing the biopharmaceutical industry, including the impact of COVID-19. They highlighted innovation to help drive the creation and commercialization of life-changing, personalized C> treatments.

In a panel on the evolution of C> and the path toward scalability and manufacturability of these treatments, Dr. Brophy gave his perspective on this growing need for the bioprocessing industry.

"Genuine progress is being made in the long-standing battle to effectively treat and control disease, and cell & gene therapies will only continue to unlock new frontiers in medicine," said Dr. Brophy. "We're beginning to see more clearly the issues that need to be addressed, and know that if products are to scale and become more accessible to patients worldwide, there needs to be efficiency in operations. There is an absolute requirement for automation, both to reduce variability and to generate process efficiencies. At Avantor, we are ready to make the essential materials and technologies available to companies leading the charge and treating patients. Innovation and agility are central to how we're partnering with the industry to resolve these issues."

On a separate panel, Ms. Berrn highlighted how the industry is navigating the challenges and impact of the global pandemic, from the fragmentation of supply chains to clinical trial disruption.

"In this critical moment for the industry and the world, it is more important than ever for leaders in the cell & gene therapy community to come together to strategize and share ideas," said Ms. Berrn. "Avantor is actively working with the world's leading pharmaceutical and biotechnology companies to accelerate the production of novel treatments. Collectively, our goal is to mitigate any challenges in the process of taking a groundbreaking treatment from its initial scientific discovery to delivery in treating patients."

Avantor provides products for biopharma production workflows, including cell and gene therapy offering. Search 'Avantor biopharma' in your browser.

About AvantorAvantor, a Fortune 500 company, is a leading global provider of mission-critical products and services to customers in the biopharma, healthcare, education & government, and advanced technologies & applied materials industries. Our portfolio is used in virtually every stage of the most important research, development and production activities in the industries we serve. One of our greatest strengths comes from having a global infrastructure that is strategically located to support the needs of our customers. Our global footprint enables us to serve more than 225,000 customer locations and gives us extensive access to research laboratories and scientists in more than 180 countries.We set science in motion to create a better world. For information, visit avantorsciences.com and find us on LinkedIn, Twitter and Facebook.

Robert DonohoeSenior Director, Corporate CommunicationsAvantorM: 484-688-4730[emailprotected]

Source: Avantor and Financial News

SOURCE Avantor and Financial News

http://avantorsciences.com

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Avantor Discusses Cell & Gene Therapy Production Trends Through the Lens of COVID-19 - PRNewswire

This report studies the Cell and Gene Therapy to get Covid-19 marketplace with Many details of the industry such as the market size, market standing, marketplace trends and prediction, the report also provides brief advice of their opponents as well as the particular growth opportunities with key market drivers. Locate the complete Cell and Gene Therapy to get Covid-19 market evaluation segmented by firms, area, type and software in the document.

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

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The report discusses the various Kinds of options for While the areas considered in the range of the report include North America, Europe, and assorted others. The analysis also highlights on how climbing digital security dangers is altering the industry situation.

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

This report concentrates on the international Cell and Gene Therapy for Covid-19 Status, future prediction, growth opportunity, key marketplace and players.

The following players are covered in this report:

Amgen Inc.

bluebird bio, Inc.

Dendreon Pharmaceuticals LLC.

Fibrocell Science, Inc.

Human Stem Cells Institute

Kite Pharma, Inc.

Kolon TissueGene, Inc.

Novartis AG

Orchard Therapeutics plc.

Organogenesis Holdings Inc.

Pfizer, Inc.

RENOVA THERAPEUTICS

Shanghai Sunway Biotech Co., Ltd.,

Sibiono GeneTech Co. Ltd.,

Spark Therapeutics, Inc.

Vericel Corporation

ViroMed Co., Ltd.

The Cell and Gene Therapy for Covid-19 marketplace is a comprehensive record Which supplies a meticulous summary of the market share, size, trends, demand, product evaluation, program analysis, regional perspective, competitive strategies, predictions, and strategies affecting the Cell and Gene Therapy for Covid-19 Industry. The report contains a thorough analysis of this marketplace competitive landscape, with the assistance of comprehensive business profiles, SWOT analysis, project feasibility analysis, and a lot of other specifics about the essential companies working on the marketplace.

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The analysis aims Covid-19 in global sector.

Breakdown Data by Type

Rare Diseases

Oncology

Hematology

Cardiovascular

Ophthalmology

Neurology

Other Therapeutic Classes

Cell and Gene Therapy Breakdown Data by Application

Pharmaceutical and Biotechnology Companies

Research and Academic Institutions

Contract Research Organizations (CROs)

Hospital

Others

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

North America

United States

Canada

Europe

Germany

France

U.K.

Italy

Russia

Nordic

Rest of Europe

Asia-Pacific

China

Japan

South Korea

Southeast Asia

India

Australia

Rest of Asia-Pacific

Latin America

Mexico

Brazil

Middle East & Africa

Turkey

Saudi Arabia

UAE

Rest of Middle East & Africa

In the competitive analysis section of the report, leading as well as prominent players of the global Cell and Gene Therapy market are broadly studied on the basis of key factors. The report offers comprehensive analysis and accurate statistics on revenue by the player for the period 2015-2020. It also offers detailed analysis supported by reliable statistics on price and revenue (global level) by player for the period 2015-2020.

The following players are covered in this report:

Amgen Inc.

bluebird bio, Inc.

Dendreon Pharmaceuticals LLC.

Fibrocell Science, Inc.

Human Stem Cells Institute

Kite Pharma, Inc.

Kolon TissueGene, Inc.

Novartis AG

Orchard Therapeutics plc.

Organogenesis Holdings Inc.

Pfizer, Inc.

RENOVA THERAPEUTICS

Shanghai Sunway Biotech Co., Ltd.,

Sibiono GeneTech Co. Ltd.,

Spark Therapeutics, Inc.

Vericel Corporation

ViroMed Co., Ltd.

The Cell and Gene Therapy for Covid-19 market study report entirely Covers the very important data of their capacity, manufacturing, value, cost/profit, supply/demand import/export, further separated by country and company, and from application/type for the best possible upgraded information representation from the statistics, tables, pie graph, and charts. These data representations give predictive information concerning the potential estimations for persuasive market development. The detailed and in depth understanding concerning our publishers makes us from the box in the event of market evaluation.

Key questions Answered within this report

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Table of Contents

Chapter 1: Global Cell and Gene Therapy for Covid-19 Market Overview

Chapter 2: Cell and Gene Therapy for Covid-19 Market Data Analysis

Chapter 3: Cell and Gene Therapy for Covid-19 Technical Data Analysis

Chapter 4: Cell and Gene Therapy for Covid-19 Government Policy and News

Chapter 5: Global Cell and Gene Therapy for Covid-19 Market Manufacturing Process and Cost Structure

Chapter 6: Cell and Gene Therapy for Covid-19 Productions Supply Sales Demand Market Status and Forecast

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Cell and Gene Therapy Market Reviewed in a New Study Eurowire - Eurowire

DUBLIN, Nov. 4, 2020 /PRNewswire/ -- The "Global Non-Oncology Precision Medicine Market: Focus on Application Area, Ecosystem Type, Country Data (15 Countries) - Analysis and Forecast, 2020-2030" report has been added to ResearchAndMarkets.com's offering.

The global non-oncology precision medicine market was valued at $41.1 billion in 2019, and it is expected to grow at an impressive double-digit rate of 11.03% and reach a value of $129.96 billion in 2030.

The precision medicine approach is used for several disease management, such as oncology, immunology, neurology, and infectious diseases. In the non-oncology precision medicine market, the application of molecular biology is for studying the cause of a patient's disease at the molecular level for diseases other than cancer, so that target-based therapies or individualized therapies can be applied to cure the patient's health-related problems.

The existing non-oncology precision medicine market is favored by multiple factors, which include rising government initiatives, coupled up with the general population's growing awareness pertaining to molecular level diagnosis and treatment. Additionally, the increasing number of advancements in molecular diagnostics, more key players interested in investing for the development of novel personalized therapies, and increasing FDA approvals for such drugs among others.

Competitive Landscape

The non-oncology precision medicine market witnessed 59 collaborations and partnerships, four fundings and investments, 44 product launches and enhancements,26 regulatory and legal activities,18 mergers and acquisitions, and six business expansions during the period January 2016-August 2020.

Key Topics Covered:

1 Product Definition

2 Scope of the Research Study

3 Research Methodology3.1 Primary Data Sources3.2 Secondary Data Sources3.3 Market Estimation Model3.4 Selection Criteria for Company Profiles

4 Market Overview4.1 Introduction4.2 Enabling Technologies of Precision Medicine Emphasizing Focus on Prevention4.3 Promising Potential and Role of Precision Medicine

5 Impact of COVID-19 Pandemic on Global Non-Oncology Precision Medicine Market5.1 Unprecedented Demand for Non-Oncology Precision Medicine Due to COVID-195.2 COVID-19 Affecting Supply Chain of Non-Oncology Precision Medicine-Based Therapies5.3 Measures Taken to Address Current Problem

6 Global Non-Oncology Precision Medicine Market: Industry Analysis6.1 Legal and Regulatory Framework and Requirements6.1.1 North America6.1.1.1 Regulatory Requirements in U.S.6.1.2 Europe6.1.3 Asia-Pacific6.2 Patent Landscape

7 Global Non-Oncology Precision Medicine Market: Market Dynamics7.1 Overview7.2 Market Driving Factors7.2.1 Improved Availability of Targeted Therapies and Decreasing Trial and Error-Based Prescription7.2.2 Reduced Chances of Adverse Drug Reactions7.2.3 Decreasing Overall Cost of Genome Sequencing7.2.4 Rising Prevalence of Chronic Diseases7.3 Market Restraining Factors7.3.1 Unclear Regulatory Framework and Reimbursement Scenario for Personalized Medicine7.3.2 Limited Knowledge About Molecular Mechanism/Interaction7.3.3 Integrating and Securing Electronic Health Records7.4 Market Opportunities7.4.1 Research Alliance Across Industry and Academia Accelerating Market Entry7.4.2 Strong Pipeline and Increasing FDA Approvals

8 Global Non-Oncology Precision Medicine Market: Competitive Landscape8.1 Key Strategies and Developments8.1.1 Product Offerings8.1.2 Partnerships and Alliances8.1.3 Regulatory and Legal8.1.4 Mergers and Acquisitions8.1.5 Funding and Investments8.2 Market Share Analysis8.3 Growth-Share Analysis (by Company), 2019

9 Global Non-Oncology Precision Medicine Market (by Application), $Billion, 2019-20309.1 Overview9.2 Infectious Diseases9.2.1 Respiratory Infections9.2.2 Gastrointestinal Infections9.2.3 Sexually Transmitted Infections9.2.4 Others9.3 Neurology/Physiatry9.3.1 Neurodegenerative Disorders9.3.2 Neuropsychiatric Disorders9.3.3 Others9.4 Lifestyle and Endocrinology9.5 Cardiovascular9.5.1 Cardiac Myopathies and Arrhythmia9.5.2 Others9.6 Gastroenterology9.7 Other Applications

10 Global Non-Oncology Precision Medicine Market (by Ecosystem), $Billion, 2019-203010.1 Overview10.2 Applied Sciences10.2.1 Genomics10.2.2 Global Non-Oncology Precision Medicine Genomics Market (by Technology)10.2.2.1 Polymerase Chain Reaction (PCR)10.2.2.2 Precision Medicine Next-Generation Sequencing (PM NGS)10.2.2.3 Genome Editing10.2.2.4 Other Technologies10.2.3 Pharmacogenomics10.2.4 Other Applied Sciences10.3 Precision Diagnostics10.3.1 Molecular Diagnostics (MDx)10.3.2 Medical Imaging10.4 Digital Health and Information Technology10.4.1 Clinical Decision Support Systems (CDSS)10.4.2 Big Data Analytics10.4.3 IT Infrastructure10.4.4 Genomics Informatics10.4.5 In-Silico Informatics10.4.6 Mobile Health10.5 Precision Therapeutics10.5.1 Clinical Trials10.5.2 Cell Therapy10.5.3 Drug Discovery and Research10.5.4 Gene Therapy

11 Global Non-Oncology Precision Medicine Market (by Region), $Billion, 2019-2030

12 Company Profiles12.1 Company Overview12.2 Role of Abbott Laboratories in Global Non-Oncology Precision Medicine Market12.3 Financials12.4 SWOT Analysis

For more information about this report visit https://www.researchandmarkets.com/r/j2b11m

Research and Markets also offers Custom Research services providing focused, comprehensive and tailored research.

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Research and Markets Laura Wood, Senior Manager [emailprotected]

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$129.96 Billion Non-Oncology Precision Medicine Market, 2019-2020 & 2030 - Research Alliance Across Industry and Academia Accelerating Market...

The gene therapy market has undergone a series of transformation from past few years. Initially gene therapy of monogenetic diseases was popular; however now the gene therapy market is gaining popularity for treatment of cancer. Cancer gene therapy refers to a process of treatment of cancer by inserting therapeutic DNA into patient. Cancer gene therapy is gaining popularity as a result of its success rate in preclinical and clinical trial stages. The most common technique for cancer gene therapy involves replacing a mutated gene that is causing cancer with a healthy copy of gene. Other technique involves inactivation of a gene that is not working properly. A new technique that is being introduced to cancer gene therapy market involves insertion of new genes into the body that would help to fight against tumor cells.

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North America followed by Europe dominates the global market for cancer gene therapy market due to large number of aging population and technological advancement in the region. Asia is expected to witness high growth in cancer gene therapy market, due to increasing government initiatives, rising economy and improvement in healthcare infrastructure in the region. Some of the key driving forces for cancer gene therapy market in emerging countries are increasing R&D investment, large pool of patients and rising government funding.

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Several unmet medical needs for treatment of cancer have encouraged R&D of cancer gene therapy. Various factors, such as increasing prevalence of cancer, rising government initiatives, increasing funding from various government and non-government organizations, are driving the global cancer gene therapy market. In addition, ethical acceptance of gene therapy for treatment of diseases and growing popularity of DNA vaccines is driving the global cancer gene therapy market. However, less awareness and high cost involved in treatment are restraining the growth of global market for cancer gene therapy.

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Innovation of some innovative therapy with better success rate is expected to offer good opportunity for cancer gene therapy. In addition, despite of high cost involved in R&D, pharmaceutical companies are showing increased interest in this field. This is expected to offer good potential for cancer gene therapy market. Currently, most of the cancer gene therapy products are in clinical trial phases. The market is expected to rise after clearance or success of these products from clinical trials. 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

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The Cancer Gene Therapy Market to get on to the elliptical growth mode in the next decade - Eurowire