Archive for the ‘Gene Therapy Research’ Category

COVID-19 came with plenty of mysteries, some of which have been resolved, and many that haven't. One mystery since the beginning of the pandemic was why children seemed much less susceptible to the virus than adults. For that and more research news, continue reading.

Why Kids May Have More Protection Against COVID-19

Generally, children are less at risk of COVID-19 infection than adults - not immune, but less likely to be infected and less likely to have severe disease. But why?

Researchers at theUniversity of Queensland, Australia,suggestthat children's nasal lining, or the nasal epithelium, inhibits infection and replication, particularly of the original Wuhan wild-type strain of SARS-CoV-2 and the Delta variant. However, the Omicron variant and its subvariants do not seem to have the same issue. They published their research inPLOS Biology.

"We have provided the first experimental evidence that the pediatric nasal epithelium may play an important role in reducing the susceptibility of children to SARS-CoV-2," the authors wrote. "The data strongly suggest that the nasal epithelium of children is distinct and that it may afford children some level of protection from ancestral SARS-CoV-2."

They found that the wild-type virus replicated less efficiently and was linked to an increased antiviral response in the nasal epithelial cells of children. The effect was also seen with the Delta variant, but not with the Omicron. They also found that Omicron replicated better in pediatric nasal epithelial cells than the wildtype or Delta, although they caution that this was a small study and may reflect how the virus is evolving.

New Molecules Discovered for Treating Age-Related Diseases

Researchers at theHebrew University of Jerusalemhaveidentifieda group of molecules that help cells repair damaged components. Impaired mitophagy is associated with various aging-associated diseases, such as Alzheimer's and Parkinson's, as well as sarcopenia.

Augmenting mitophagy, where defective mitochondria are removed and replaced by new ones, is an emerging strategy for treating the elderly. The researchers designed and tested a family of compounds related to spermidine, a known mitophagy-promoting agent. A prototype, 1,8-diaminooctane (VL-004), was better than spermidine in its ability to induce mitophagy and protect against oxidative stress.

The researchwas publishedin the journalAutophagy. The researchers founded a biotech company, Vitalunga, that is working to develop the drug.

Unique Subset of Immune Cells Protects Against Stroke

A study from theUniversity of Pittsburgfounda subset of white blood cells, a novel subset of CD8+ regulatory-like T cells (CD8+TRLs), that appear to confer fast-acting and lasting protection against ischemic stroke in mice.

These cells were attracted to the site of ischemic injury by a unique "homing" signal produced by the damaged brain cells. The CD8+TRLs reached the brain 24 hours after the stroke, then released molecules that provide direct neuroprotective effects and knock down inflammation and secondary brain damage.

In the mice studies, the animals who received a transfusion of purified CD8+TRLs did better and recovered faster than the untreated mice over five weeks. The CD8+TRLs appear to act as early responders to alert and recruit defenses after a stroke.

Gene Therapy Shows Promise in ALS

Researchers at theUniversity of California San DiegoSchool of Medicinereportthat gene therapy in animal models of ALS measurably delayed disease onset. Although most ALS cases are of unknown cause, the mouse and rat models were of an inherited form of ALS.

The therapy involved an AAV-vector carrying synapsin-Caveolin-1 cDNA into the spinal cords of the mice. The SynCav1 protected and preserved spinal cord motor neurons and added to the mice's longevity. They speculate that SynCav1 might serve as novel gene therapy for ALS and other forms of CNS disease.

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease marked by progressive loss of motor neurons in the brain and spinal cord. It is fatal, and there are no cures.

New Therapeutic Target for Colorectal Tumors

Researchers at theTokyo University of Scienceidentifieda potential new therapeutic target for colorectal tumors.

Inflammatory bowel disease (IBD), which includes Crohn's disease and ulcerative colitis, can often lead to colorectal tumors. Innate immune receptors, such as C-type lectin receptors (CLRs), are responsible for the development of IBD. But they also play a key role in regulating gut microbiota and defending against pathogens.

One of those CLRs is a dendritic cell immunoreceptor (DCIR), responsible for maintaining the homeostasis of the immune and skeletal systems. Working with mice, the researchers found that mice without DCIR had less colitis severity and AOM-DSS-induced colorectal tumor growth. They found that using an antibody called anti-NA2 against asialo-biantennary-N-glycans (NA2), which binds to DCIR, decreased colitis symptoms and prevented colorectal tumor growth.

Two Common Viruses Associated with Alzheimer's Disease

Astudyout ofTufts Universityand theUniversity of Oxfordstrengthened the association between specific viral infections and Alzheimer's disease. Two common viruses can lie dormant in brain cells herpes simplex virus (HSV) and varicella-zoster virus (VZV); the latter is associated with shingles and chickenpox.

Researchers leveraged 3D human tissue culture models to mimic the brain and demonstrated that VZV may activate HSV to lay the groundwork for the early stages of Alzheimer's disease. HSV-1, the primary variant of HSV, when activated, leads to the accumulation of tau and amyloid-beta proteins, which are implicated in Alzheimer's disease. They published their research in theJournal of Alzheimer's Disease.

"Our results suggest one pathway to Alzheimer's disease, caused by a VZV infection which creates inflammatory triggers that awaken HSV in the brain," Dana Cairns, Ph.D., GBS12, a research associate in the Biomedical Engineering Department at Tufts, said. "While we demonstrated a link between VZV and HSV-1 activation, it's possible that other inflammatory events in the brain could also awaken HSV-1 and lead to Alzheimer's Disease."

Read the original:
Research Roundup: Why Kids Have More Protection Against COVID-19 & More - BioSpace

According toGrand View Research, the global cell therapy market was valued at $7.8 billion in 2020 and is expected to expand at a compound annual growth rate (CAGR) of 14.5% between 2021 and 2028.

The rising number of clinical studies for cell-based therapies and investments in the industry may have a symbiotic relationship. The industry is seeing a snowballing number of ongoingclinical trialswith funding from governments and private agencies.

Theres an arguably thin line between cell and gene therapy. Cell therapy is the transfer of intact, live cells into a patient to help lessen or cure a disease, according to theAmerican Society of Gene and Cell Therapy (ASGCT). The cells may originate from the patient (autologous cells) or a donor (allogeneic cells).

Gene therapy involves the transfer of genetic material, usually in a carrier or vector, and the uptake of the gene into the appropriate cells of the body. Some protocols use both gene therapy and cell therapy.

Companies are using thebuilding blocks of lifeand advanced technologies to improve the treatment of human diseases and disorders such as cancer, providing an alternative to traditionally relied-on drugs and surgical treatments.

Cell therapy companies like Longeveron Inc. LGVN, Biogen Inc. BIIB, Alzamend Neuro Inc. ALZN and Solid Biosciences Inc. SLDB, as a result, have gained attention for their progress in using living cells to treat previously incurable diseases and disorders.

COVID-19 has reportedly causedsignificant disruptionto the cell and gene therapy industry. The pandemic has exacerbated the woes of an industry thats had its fair share of challenges with the supply of materials and the manufacturing and logistics processes.

General investments also slowed for the industry as governments shifted focus to saving lives and reviving economies. But things are starting to pick up now that the pandemic is on a downward trend.

Regulatory bodies like the Food and Drug Administration (FDA) have been urged to be more flexible in their approval timelines to make therapies affordable. Discussions continue around access and ensuring these therapies are affordable, reimbursable and profitable for the biopharmaceutical companies that develop them.

Academic and industry collaborations are expected to continue to expand and grow with noticeable impacts on the approval of products. Partnerships among academia, global pharmaceutical companies and small biotechs are expected to continue to shape the cell and gene therapy industry.

Longeveron, a clinical-stage biotechnology company, is one example of a company in the industry that has seemingly done well even during the pandemic. The company reports developing cellular therapies for investigation in chronic aging-related and certain life-threatening conditions.

The companys lead investigational product is Lomecel-B, a cell-based therapy product, derived from culture-expanded medicinal signaling cells sourced from the bone marrow of young, healthy adult donors.

Longeveron believes using the same cells that promote formation of new blood vessels, enhance cell survival and proliferation, inhibit cell death, and modulate immune system function may result in safe and effective therapies for some of the most difficult disorders associated with aging and some medical disorders.

Longeveron is sponsoring Phase 1 and 2 clinical trials in the following indications: Aging frailty, Alzheimers disease, metabolic syndrome, acute respiratory distress syndrome and hypoplastic left heart syndrome.

The companys mission is to advance Lomecel-B and other cell-based product candidates into pivotal Phase 3 trials to achieve regulatory approvals, subsequent commercialization and broad use by the healthcare community.

Photo by Edward Jenner from Pexels

This post contains sponsored advertising content. This content is for informational purposes only and is not intended to be investing advice.

Read the original:
Is This Company In A Special Position Even As The COVID-19 Pandemic Affects Cell-Based Therapy Industry? - Benzinga

OHSU researchers will receive a grant to helpadvance a first-of-its-kind method to turn an individuals skin cell into an egg, with the potential to produce viable embryos. (OHSU/Christine Torres Hicks)

Scientists at Oregon Health & Science University have received significant philanthropic support to advance a first-of-its-kind method to turn an individuals skin cell into an egg, with the potential to produce viable embryos.

The technique, initially demonstrated in mice, could eventually provide a new avenue for child-bearing among couples unable to produce viable eggs of their own.

Paula Amato, M.D., professor of obstetrics and gynecology in the OHSU School of Medicine, andShoukhrat Mitalipov, Ph.D., director of the OHSU Center for Embryonic Cell and Gene Therapy. (OHSU/Christine Torres Hicks)

Even though the proof of concept in mice shows promise, significant challenges remain to be resolved before the technique could be ready for clinical trials under strict ethical and scientific oversight. Even then, Congress currently precludes the Food and Drug Administration from providing oversight for clinical trials involving genetic modification of human embryos.

Shoukhrat Mitalipov, Ph.D., (OHSU)

It will take probably a decade before we can say were ready, said Shoukhrat Mitalipov, Ph.D., director of the OHSU Center for Embryonic Cell and Gene Therapy. The science behind it is complex, but we think were on the right path.

This type of research is not funded by the National Institutes of Health, so it depends on philanthropic support. For this project, Open Philanthropy awarded $4 million over three years through the OHSU Foundation.

Paula Amato, M.D. (OHSU)

Paula Amato, M.D., professor of obstetrics and gynecology in the OHSU School of Medicine, sees the potential for an enormous benefit to families struggling to have children if the technique proves successful.

Age-related decline in fertility remains an intractable problem in our field, especially as women are delaying childbearing, said Amato, who is the principal investigator for the grant award.

The technique holds promise for helping families to have genetically related children, a cohort that includes women unable to produce viable eggs because of age or other causes, including previous treatment for cancer. It also raises the possibility of men in same-sex relationships having children genetically related to both partners.

The skin cell can come from somebody who doesnt have any eggs themselves, Amato said. The biggest implication is for female, age-related infertility. It can also come from women with premature ovarian insufficiency due to cancer treatment or genetic conditions, or from men who would be able to produce a genetically related child with a male partner.

The award from Open Philanthropy will enable OHSU researchers to develop the technique in early human embryos using eggs and sperm from research donors. As with other groundbreaking research at OHSU including a gene-editing discovery that generated worldwide attention in 2017 none of the early embryos will be allowed to develop past the early blastocyst stage.

Researchers will build on a study in mice published this January in the journal Communications Biology.

The study demonstrated that it is possible to produce normal eggs by transplanting skin-cell nuclei into donor eggs from which the nuclei have been removed. Known as somatic cell nuclear transfer, the technique was famously used in 1997 to clone a sheep in Scotland named Dolly. In contrast to a direct clone of one parent, the mouse study published earlier this year required OHSU and collaborating scientists to cut the donor DNA in half and then fertilize the resulting egg with sperm to generate a viable embryo with chromosomes from both parents.

The process involves implanting the skin cell nuclei into a donor egg, and then allowing the egg to discard half its skin cell chromosomes a process similar to meiosis, when cells divide to produce sperm or egg cells. This results in a haploid egg with a single set of chromosomes with precisely half the chromosomes of the diploid skin cell with two sets of chromosomes. At just the right phase of the cell cycle, the new egg is combined with sperm chromosomes through in vitro fertilization.

An embryo then develops with the correct diploid number of chromosomes from each parent.

We had to show in the mouse that this hypothesis works, Mitalipov said. Open Philanthropy saw the implications for fertility with a new way of looking into this. The key is inducing haploidy.

Follow this link:
OHSU advancing first-of-its-kind strategy to overcome infertility - OHSU News

The new report titled Hemophilia Gene Therapy Market offer by Key Players, Types, Applications, Countries, Market Size, Forecast to 2030offered by Market Research, Inc. includes a comprehensive analysis of the market size, geographical landscape along with the revenue estimation of the industry. In addition, the report also highlights the challenges impeding market growth and expansion strategies employed by leading companies in the Hemophilia Gene Therapy Market.

Hemophilia is a genetic bleeding disorder in which an individual lacks or has low levels of proteins called clotting factors. There are around 13 types of clotting factors that work with blood platelets, which are necessary for clotting process to initiate. There are three forms of hemophilia A, B, and C. Hemophilia A is the most common form and is caused due to deficiency in clotting factor VIII. Hemophilia B occurs due to deficiency of clotting factor IX and Hemophilia C is caused due to clotting factor XI deficiency. Hemophilia is incurable with current therapeutic options, which only reduces symptoms such as spontaneous bleeding in muscles and joints as well as increased risk for intracranial hemorrhage.

Click the link to get a Sample Copy of the Report: https://www.marketresearchinc.com/request-sample.php?id=115759

This market study covers and analyzes the potential of the global Hemophilia Gene Therapy industry, providing geometric information about market dynamics, growth factors, major challenges, PEST analysis and market entry strategy analysis, opportunities and forecasts. One of the major highpoints of the report is to provide companies in the industry with a strategic analysis of the impact of COVID-19 on Hemophilia Gene Therapy market.

Hemophilia Gene Therapy Market: Competition Landscape

The Hemophilia Gene Therapy market report includes information on the product presentations, sustainability and prospects of leading player including: BioMarin Pharmaceuticals, Inc., Spark Therapeutics, Pfizer, Inc., UniQure NV, Ultragenyx Pharmaceutical, Shire PLC Sangamo Therapeutics, Inc., and Freeline Therapeutics

Hemophilia Gene Therapy Market: Segmentation

By Type:

By Application:

Hemophilia Gene Therapy Market: Regional Analysis

All the regional segmentation has been studied based on recent and future trends and the market is forecasted throughout the prediction period. The countries covered in the regional analysis of the Global Hemophilia Gene Therapy market report are North America, Europe, Asia-Pacific (APAC), Middle East and Africa (MEA) and Latin America.

Years Considered for the Hemophilia Gene Therapy Market Size:

Key Benefits of the report:

Ask for Discount: https://www.marketresearchinc.com/ask-for-discount.php?id=115759

Major Points Covered in TOC:

Market Summary: It incorporates six sections, research scope, major players covered, market segments by type, Hemophilia Gene Therapy market segments by application, study goals and years considered.

Market Landscape: Here, the global Hemophilia Gene Therapy Market is dissected, by value, income, volume, market rate, and most recent patterns. The development and consolidation of the overall industry and top organizations is provided through graphs and piece of the pie for organizations.

Profiles of Companies: Here, driving players of the worldwide Hemophilia Gene Therapy market are considered depending on sales across regions, key innovations, net income, cost, and other factors.

Market Status and Outlook by Region: In this segment, the report examines the net deals, income, creation and portion of the overall industry, CAGR and market size by locale. The global Hemophilia Gene Therapy Market is profoundly examined based on areas and nations like North America, Europe, Asia Pacific, Latin America and Middle East & Africa.

Segment Analysis: Accurate and reliable foretell about the market share of the essential sections of the Hemophilia Gene Therapy market is provided

Market Forecasts: In this section, accurate and validated values of the total market size in terms of value and volume are provided by the research analysts. Also, the report includes production, consumption, sales, and other forecasts for the global Hemophilia Gene Therapy Market.

Market Trends: Deep dive analysis of the markets recent and future trends are provided in this section.

Enquiry before buying this premium Report: https://www.marketresearchinc.com/enquiry-before-buying.php?id=115759

Contact Us

Market Research Inc

Author: Kevin

US Address: 51 Yerba Buena Lane, Ground Suite,

Inner Sunset San Francisco, CA 94103, USA

Call Us: +1 (628) 225-1818

Write Us: [emailprotected]

Read this article:
Hemophilia Gene Therapy Market by Growth Analysis and Precise Outlook 2030 | Key Players BioMarin Pharmaceuticals, Inc., Spark Therapeutics, Pfizer,...

Twenty-three years ago, the field of gene therapy was bursting with the promise of breakthrough treatments. Then it was almost instantly derailed by the death of an 18-year-old clinical trial volunteer named Jesse Gelsinger after he received a genetically engineered virus that had been developed to treat his rare liver condition.

An FDA investigation revealed that the principal investigator and/or the team running the gene therapy trial at the University of Pennsylvania failed to disclose that before Gelsinger was treated, other patients had experienced alarming side effects and that monkeys administered the same engineered virus had died. The incident, coupled with dangerous outcomes involving other gene therapy treatments, had a chilling effect on the field and investors backed away.

Today, new approaches to gene therapy that include advances driven by CRISPR gene editing tools are raising hopes of a gene therapy revival. There are potential breakthroughs in the pipeline, including treatments for different types of cancer and sickle cell disease.

advertisement

Im concerned that gene therapy 2.0 is at risk of making the same mistakes that plagued the 1.0 version. Most notably, exciting work to translate gene therapy advances into safe, effective, and commercially viable treatments are at risk of being undermined by a reluctance to share data.

Im not saying that the field is on the verge of something analogous to the tragedy experienced in 1999. But the road to such extremes can be paved with a series of lesser nondisclosures that inhibit the free flow of scientific data essential for assessing potential risks long before treatments are given to people.

advertisement

I saw an example in May at the annual meeting of the American Society of Gene and Cell Therapy. A presenter was discussing a new approach for using gene therapy to treat a rare genetic disorder called Leber congenital amaurosis that causes blindness in children. The presenter discussed experiments in mice that involved disabling or deleting certain segments of a gene linked to the disorder, and this treatment appeared to at least slow the process of vision loss.

Assessing the safety and effectiveness of this potentially exciting advance requires knowing which regions of the gene were being modified. But when someone in the audience asked for more details, the presenter indicated that the information was proprietary and he would not disclose that. The exasperated questioner pointed out that the presenters approach was all about deleting [a segment of a gene], but you dont say what you are deleting. The response? Silence.

Tinkering with a particular genes function, even when the goal is to stop it from doing harmful things, can be risky. Knowing which region of a gene is being altered is essential for determining if solving one problem might create an even bigger one. For example, I and other scientists are interested in the potential of using CRISPR gene editing tools to disable a gene called nuclear factor erythroid 2-related factor 2 (NRF2), which produces a protein that protects a certain type of lung cancer tumor from the effects of chemotherapy or radiation. But that same gene confers a range of health benefits, such as withstanding toxic insults like nicotine or radiation or heat stress, and a poorly targeted edit might do more harm than good.

I am also regularly seeing a failure to disclose important details, like studies that use several strains of mice in a gene therapy experiment without clarifying which strain was linked to a particular result. That can make it difficult, or even impossible, to conduct a fundamental exercise in science: reproduce the results of other investigators. Reproducibility is a problem across all areas of science. But the stakes are higher in the fragile world of gene therapy, where investigators need to be doing all they can to protect patient safety.

My initial thoughts about the presenter who was loquacious in describing his success with gene therapy for Leber congenital amaurosis but quiet about revealing his methods was simply, If you arent willing to share your full information, dont present at scientific conferences.

I am not against protecting intellectual property or patenting biotechnology advances. Properly used, these strategies help attract investment that accelerates the search for transformative treatments. But researchers shouldnt come to a place where they benefit from everyone else freely sharing their findings but then refuse to reciprocate.

My broader message is that the field of gene therapy was once severely damaged by not sharing data that may have been able to prevent a young man from losing his life. Transparency in gene therapy research which can be accomplished without compromising commercial prospects is vital to success. One high-profile failure would badly hurt the revival of gene therapy; two would send it back into hibernation.

When everyone embraces transparency, all of our projects are likely to advance faster toward safe and effective treatments and everyone wins: scientists, investors and, most importantly, the people who could benefit from gene therapy.

Eric B. Kmiec is the executive director and chief scientific officer of the ChristianaCare Gene Editing Institute in Newark, Del.

Here is the original post:
Secrecy: A demon of gene therapy's past bedevils its future - STAT

Recently, a patient centricity concept has spread across all corners of the healthcare community with a straightforward goal: to improve and save patients lives through innovative and effective drug therapies adapted to patients' individual needs. However, for this concept to truly come to life, it is necessary to count on temperature-controlled supply chain logisticslike CRYOPDP to turn what could be a complex operation into a highly efficient process. This is extremely vital, because a patients life could literally be on the line with every shipment.Patient centricity in the clinical trial community involves meeting the patients where they are.The clinical trial industry is globalising and demands international healthcare logistics partners that can serve its growing and continuously evolving needs. For decades, temperature-controlled logistics experts have operated behind the scenes with patients barely aware of the hard work that went into their medicines journey. Thanks to the Covid-19 vaccine distributions impressive performance and its universal effect on people's health across the globe, it has become apparent to the world just how critical and important a logistics providers mission is for both patients and the industry.The endless pandemic lockdowns meant that patients could not travel to hospital sites for clinical trials due to travel and access restrictions. This led to a seismic shift where sponsors quickly moved to a direct-to-patient model to try to continue trials and keep patient treatments on schedule. As part of our response to this unique situation, CRYOPDP as a specialist in temperature-controlled logistics, with the mission to improve people's healthcare options, had to go further and dive deeper to better understand customers perceptions of the impact of the pandemic and work with them to provide those options.With the patient always at the centre of its business and considering the huge development of decentralised clinical trials, the development and implementation of a new service such as Direct-to-Patientseems to be the most natural evolution for CRYOPDP. We have been offering this turnkey solution completely adapted to patients needs, with the same efficiency and total peace of mind, to the benefit of many customers.This model makes life a lot easier for patients and their families, as they dont have to make multiple trips to a clinic or hospital that is potentially a great distance from their home. It is also beneficial for patients who may be too ill to travel, as well as saves patients time and money.Because of these benefits, the direct-to-patient model has increased clinical trial recruitment by up to 60 percent and helped to maintain patient retention by over 95 percent. Drug developers can also gain access to a larger patient population by onboarding those who are not located near participating hospitals or clinics.Patient centricity becomes more evident with the new generation of cell and gene therapies.From day one, CRYOPDP temperature-controlled logistics solutions have always been essential to improve and save patients lives. But, when speaking about cell and gene therapies in particular, this gains an even more significant meaning.The increase in personalised medicine, advanced therapies, and improved access to healthcare in the developing world are influencing future supply chain solutions development. Cell and gene therapies are enabling the healthcare community to shift the arrow and think about patients in a whole new way.Cell and gene therapies demand rigorous and precise temperature control to ensure that the therapies maintain their viability. And maintaining temperature control calls for flawless implementation and execution.From designing the best transportation route to selecting the correct packaging, every detail is critical to keep product integrity high under all conditions, and for this to happen, the healthcare community can count on CRYOPDP specialists who can meticulously handle the entire supply chain process. We've been supporting the life sciences and healthcare communities and focused on improving patient centricity with innovative temperature-controlled logistics solutions because thinking about the patient and the outcome of our work is what moves and inspires us to be better every day.

In all the geographies of the world that we cover, around 150 countries, we produce an operational performance of 99.96%. And to deliver such operational performance, we count on our employees, the specialists around the globe, to follow our quality standards and protocols in detail, so we can deliver a quality service.

When dealing with patients lives, there is no room for errors. Its all about quality of service making sure that lifesaving samples are distributed on time, within the correct specifications and at the right temperature to ensure the patient's health is never compromised.This commitment has helped CRYOPDP to win numerous industry awards, including Best Clinical Trial Logistics Provider in APAC at the Bioprocessing Excellence Awards 2021 and Most Advanced Healthcare Solution Providers from Europe 2021 by Healthcare Insights Magazine.Our achievements are being recognised across the industry, and as we continue to improve our services for healthcare communities, it will be the end patient that benefits the most.

See the rest here:
The Importance of Patient Centricity in Clinical Research and Cell / Gene Therapy Development - Contract Pharma

New York, July 13, 2022 (GLOBE NEWSWIRE) -- According to the World Health Organization (WHO), around 10 million deaths, or nearly 1 in 6 deaths, were caused by cancer in 2020, making it the top cause of death globally. Breast, lung, colon, rectum, and prostate cancers are the most prevalent types of cancer. If found early and appropriately treated, many tumors (30% to 50%) are curable. According to the American Cancer Society (ACS), 1,918,030 new cancer cases and 609,360 cancer deaths are expected in 2022, with lung cancer as the primary cause of death accounting for about 350 of those fatalities daily in the United States.

In recent research titled Global Gene Therapy Market, Kenneth Research provided a brief overview of market elements including growth drivers, restraint factors, current market trends, and potential for future growth. The influence of COVID-19 and its effects on end-users are both thoroughly examined in the market research report, which covers the forecast period, i.e., 2022-2031. In addition, the research study examines the product portfolios and market expansion plans of the principal competitors.In 2020, according to the World Cancer Research Fund (WCRF), there were 18 million new cases of cancer worldwide. 9.3 million of these instances involved men, while 8.8 million involved women. The growth of the global gene therapy market can be attributed on account of the rising prevalence of cancer cases. Also, the adoption of gene therapies for the treatment of cancer is predicted to grow the market further. For instance, at the University of Pennsylvania, the first trial for testing a CRISPR-created cancer medicine was launched in the United States in 2019.CRISPR is a gene-editing-tool, that can modify any DNA segment within the 3 billion letters of the human genome. The global gene therapy market is expected to gather around USD 6 billion in revenue by 2031 and grow with a CAGR of ~34% over the forecast period. Get A Sample Copy of This Report @ https://www.kennethresearch.com/sample-request-10070542

The global gene therapy market is segmented on the basis of region into North America, Europe, Asia Pacific, Latin America, and the Middle East and Africa. On the back of rapid rising cancer incidence rates, and the availability of high disposable income, the market in North America is predicted to experience significant expansion over the course of the forecast period. For instance, the Cancer Facts & Figures 2021 by the American Cancer Society, the study estimates that 1.9 million new instances of cancer were diagnosed and 608,570 cancer deaths in the United States in 2021. Also, an increase in the demand for gene-therapy-related R&D activities further helps the growth of the market. According to the World Bank Data, the domestic general government healthcare expenditure in the U.S. was 5,552.60 IN 2019 whereas in Canada the domestic general health care expenditure was 3,873.70 in 2019. Thus, a rise in government health care support is expected to expand the gene therapy-related R&D activities and further aid on to improve the market in the region.On the other hand, the global gene therapy market in the Asia Pacific region is anticipated to experience the greatest CAGR throughout the forecast period owing to the growing population in the region and increased approval and availability of gene therapy products. According to the World Bank data, the total population of China was 1.41 billion in 2020 whereas, India had 1.38 billion people in 2020. As the population grows, the likelihood of contracting a disease increases. Additionally, it is anticipated that increased government efforts to upgrade the health care infrastructure and rising healthcare costs in that region are expected to expand the industry. Also, the health care expenditure in Japan in 2019 was 10.74% whereas, in China, the GDP was 5.35%. In addition to that, the domestic general government health expenditure per capita for Japan was 3,846.54 in 2019 and China was 492.72 in 2019. Thus, growing health care expenditure and government support in health care expansion are further expected for the growth of the market in the region.

Browse to access In-depth research report on Gene Therapy Market with detailed charts and figures: https://www.kennethresearch.com/report-details/gene-therapy-market/10070542

The study further incorporates Y-O-Y Growth, demand & supply and forecasts future opportunities in North America (U.S., Canada), Europe (U.K., Germany, France, Italy, Spain, Hungary, Belgium, Netherlands & Luxembourg, NORDIC[Finland, Sweden, Norway, Denmark], Poland, Turkey, Russia, Rest of Europe), Latin America (Brazil, Mexico, Argentina, Rest of Latin America), Asia Pacific(China, India, Japan, South Korea, Indonesia, Singapore, Malaysia, Australia, New Zealand, Rest of Asia Pacific), Middle East and Africa(Israel, GCC[Saudi Arabia, UAE, Bahrain, Kuwait, Qatar, Oman], North Africa, South Africa, Rest of the Middle East and Africa).The global gene therapy market is segmented by indication into cancer, metabolic disorders, eye disorders, cardiovascular diseases, and others. Among that the cancer segment is predicted to hold the largest share over the forecast period. On account of the growing widespread presence of cancer cases, the growth of the market can be accredited. The estimated number of new cases of cancer patients in India was around 11,57,294 cases which had risen to 13,24,413 total cases in 2020. In addition to that, the total number of cancer patients was 1,708,921 in 2018 in the U.S., according to the Centers for Disease Control and Prevention (CDC) which got increased to an estimated rate of 1.8 million new cases in 2020. The statistical studies exhibit an increasingly widespread of the disease worldwide which is expected to drive the growth of the segment. Gene therapies are used to treat a variety of malignancies, including those of the brain, lung, breast, pancreatic, liver, prostate, bladder, head & neck, skin, and ovary. For instance, according to the World Cancer Research Fund (WCRF), the most common cancers around the world were breast and lung cancers, accounting to 12.5% and 12.2% respectively of all new cases that were expected to be diagnosed in 2020. Also, there were 1.9 million new instances of colorectal cancer, accounting for 10.7% of all cancer cases in 2020.

Get a Sample PDF of Global Gene Therapy Market @ https://www.kennethresearch.com/sample-request-10070542

The global gene therapy market is segmented by end-user into pharma & biotech, and academia. Numerous ongoing researches and studies have been conducted in the pharma and biotech sector which is anticipated to account for the growth of the segment. For instance, based on a study by PhRMA, there were 289 gene therapies done in clinical development by biopharmaceutical companies in 2018 which had increased to 362 gene therapies in 2020. Also, 6 diseases were already being treated using gene therapy, whereas 362 cell and gene therapies were in the development stage in 2020. In addition to that, 9 cell or gene therapy products have been approved by U.S. Food and Drug Administration (FDA) as of February 2020; they are used to treat cancer, eye conditions, and uncommon inherited diseases.

The global gene therapy market is also segmented on the basis of technology and application.

Global Gene Therapy Market, Segmentation by Technology:

Global Gene Therapy Market, Segmentation by Application:

Enquiry before Buying This Report @ https://www.kennethresearch.com/sample-request-10070542

Some of the well-known leaders in the global gene therapy market that are included in our report are Kineta, Inc., Orchard Therapeutics plc, SIBIONO, Questex, CRISPR Therapeutics, Editas Medicine, and others.

Browse More Related Reports:

Disposable Syringes Market Segmentation by Type (General/Conventional, Safety, and Pre-Filled Syringes); by Syringe Tip (Luer-Lock, Slip, Eccentric, and Catheter Syringe Tips); by Application (Immunization, and Therapeutic Injections, and Others); by End-User (Hospitals, Diagnostic Laboratories, Blood Banks, Pharmaceutical Companies, and Others)-Global Demand Analysis & Opportunity Outlook 2021-2031

Immunoassays in R&D Market Segmentation by Application (Cancer Research, Infectious Diseases, Autoimmune Diseases, Endocrinology, and Others); by End-Use (Academic Laboratories & Institutions, Biotechnology & Pharmaceutical Companies, and Others); and by Product & Services (Analyzers, Software & Services, and Kits & Reagents)-Global Demand Analysis & Opportunity Outlook 2031

Lancet Market Segmentation by Type (Push Button Safety, Pressure Activated Safety, Personal, and Side Button Safety); by Application (Glucose Testing, Hemoglobin Testing, and Coagulation Testing); and by End Users (Homecare, Hospitals & Clinics, Blood Banks, and Others)-Global Demand Analysis & Opportunity Outlook 2031

Anticholinergic Drugs Market Segmentation by Product Type (Natural, Semi-Synthetic Compound, and Synthetic Compound); by Route of Administration (Parenteral, Topical, and Oral); by Application (Muscle Spasm, Parkinsons Disease, Irritable Bowel Syndrome, Chronic Obstructive Pulmonary Disease, and Overactive Bladder); and by Channel of Distribution (Hospitals, Retail Pharmacies, and Online Pharmacies)-Global Demand Analysis & Opportunity Outlook 2031

Global Vaginal Slings Market Segmentation by End-Use (Clinics, Hospitals, and Ambulatory Surgical Centers); and by Slings Type (Conventional, and Advanced Vaginal Slings)-Demand Analysis & Opportunity Outlook 2031

About Kenneth Research

Kenneth Research is a leading service provider for strategic market research and consulting. We aim to provide unbiased, unparalleled market insights and industry analysis to help industries, conglomerates and executives to take wise decisions for their future marketing strategy, expansion and investment, etc. We believe every business can expand to its new horizon, provided a right guidance at a right time is available through strategic minds. Our out of box thinking helps our clients to take wise decision so as to avoid future uncertainties.

Contact for more Info:

AJ Daniel

Email: info@kennethresearch.com

U.S. Phone: +1 313 462 0609

Web: https://www.kennethresearch.com/

View original post here:
Global Gene Therapy Market to Garner a Revenue of About USD 6 Billion by 2031 by Growing with a CAGR of ~34% During 2022-2031 - GlobeNewswire

Cergentis is a cornerstone acquisition that expands Solvias' solutions supporting complex and emerging therapies.

According to pharmaceutical market intelligence provider, Evaluate, global sales of cell and gene therapies are projected to accelerate at a 63% compound annual growth rate through 2026. As more researchers leverage emerging genetic engineering techniques to develop complex, novel medicines, they require sophisticated solutions to analyze their safety and effectiveness.

With the addition of Cergentis, Solvias supports the increasing number of global pharmaceutical, biotech, and contract development and manufacturing organizations developing genetically engineered therapies with an expanded platform of testing services highlighted by:

Archie Cullen, CEO, Solvias, stated:"We are relentlessly focused on ensuring the safety of new therapies in development. Cergentis is a cornerstone acquisition that expands our solutions supporting complex and emerging therapies. We will continue to pursue strategic acquisitions that add specialized capabilities to our offering and advance our goal of being a forerunner in our industry."

Joris Schuurmans, CEO, Cergentis, added:"We are excited to become part of a global leader that complements our scientific expertise, innovation and customer service. Solvias and Cergentis share a deep commitment to providing our customers with the highest quality solutions and support to safely get their products into the hands of patients who need them."

Effective immediately, Mr. Schuurmans will join Solvias' leadership team and continue to lead operations for Cergentis.

Cergentis marks Solvias' second acquisition since partnering with health care investors Water Street Healthcare Partners and JLL Partners in 2020. The company has recruited industry leaders to join its board and commenced a program to significantly upgrade and expand its information technology platform and infrastructure to support its plans for global expansion.

Financial terms of the acquisition are not being disclosed. Achelous Partners served as the advisor to Cergentis on the transaction.

About CergentisCergentis is a trusted genomics-focused biotechnology company providing services and in-house solutions based on its proprietary genomic analysis platform to all leading biopharmaceutical companies and renowned research institutes. With widely published and recognized genetic analyses, Cergentis supports a global customer base in the characterization and QC of genetically engineered models, biopharmaceutical cell line development, and cell- and gene therapy products. By helping to de-risk R&D program decisions, minimizing time-to-clinic, providing objective genomic evidence for regulators, and supporting patient safety, Cergentis aims to support biopharmaceutical medicine development programs worldwide. For more information, visit cergentis.com.

About Solvias AGSolvias is a worldwide leader in contract research, development and manufacturing for the pharmaceutical, biotech, material science and cosmetic industries. Drawing on 20 years of scientific excellence, the company provides flexible and scalable analytical and manufacturing solutions that ensure the integrity of pharmaceutical and medical device products across their life cycle. Headquartered in Kaiseraugst near Basel, Switzerland, Solvias and its laboratories operate to the highest standards and in accordance with ISO, GMP, GLP and FDA regulations. For more information, visit solvias.com.

SOURCE Solvias

Original post:
Solvias Acquires Cergentis to Bolster Biologics and Cell & Gene Therapy Capabilities - PR Newswire

");$(".close-btn").click(function() { $(this).parent().fadeOut(800);});setTimeout(showpanel1, 5000);function showpanel1() { $(".close-btn").css("bottom","79px");}'); } }); } var menu = new cbpTooltipMenu(document.getElementById('cbp-tm-menu')); $(document).ready(function () { if ($(".ad-box").length) { $(".pushhead").addClass("right-space"); } });");$(".close-btn").click(function() { $(this).parent().fadeOut(800);});setTimeout(showpanel1, 5000);function showpanel1() { $(".close-btn").css("bottom","79px");}

Breaking News

Sign MOU for the discovery and development of novel drugs.

07.14.22

Through this agreement, both companies will cooperate in the overall development process, such as discovery, manufacturing and clinical trials of antibody and cell treatments, and related research and service projects.

DAAN Bio Therapeutics has conducted single cell transcriptomic and proteomic biomarker analysis through its own analysis platform using tissues secured from solid cancer patients along with DAAN Cancer Research Institute and Yonsei New Il Han Institute for Integrative Cancer Research.

Accordingly, DAAN Bio Therapeutics signed a contract manufacturing organization (CMO) service through GenScript ProBio to develop antibodies that specifically bind to the discovered target material, and established its own bispecific antibody production pipeline using antibodies derived through GMP production.

I hope this strategic partnership with DAAN Bio Therapeutics will be of great support as a global partner in accelerating the development of DAAN's antibody and cell therapy through the GenScript ProBio's one-stop CDMO service platform, said Brian Min, CEO, GenScript ProBio.

Byoung Chul Cho, co-founder and CEO, DAAN Bio Therapeutics, said, This strategic cooperation will be an opportunity to revitalize the development of antibody and cell therapy that control unique cancer treatment targets secured by DAAN Bio Therapeutics using GenScript ProBio's qualified and competitive technologies.

Through this partnership, both GenScript ProBio and DAAN Bio Therapeutics will continue their cooperation more closely with each other to develop subsequent pipelines of DAAN Bio Therapeutics, such as antibody drug and cell therapy.

See the original post:
GenScript ProBio, DAAN Bio Therapeutics Enter Cell and Gene Therapy Partnership - Contract Pharma

BioMarin Presents Findings from Studies of Valoctocogene Roxaparvovec, Investigational Gene Therapy, at the International Society on Thrombosis and Haemostasis (ISTH) 2022 Congress July 9-12, Including 4 Oral and 2 Poster Presentations

Commitment to Advancing Care for People with Hemophilia A Demonstrated with Largest and Longest, OngoingClinical Development Program for any Gene Therapy in Hemophilia A

European Commission Approval for Valoctocogene Roxaparvovec Expected Q3 2022

SAN RAFAEL, Calif., July 11, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that it presented four oral presentations and two poster presentations on valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, at the International Society on Thrombosis and Haemostasis (ISTH) 2022 World Congress from July 9-12 in London.

"With each passing year, the breadth of data supporting valoctocogene roxaparvovec increasingly demonstrate the positive impact gene therapy treatment may provide people with severe hemophilia A. These presentations at ISTH offer supportive evidence of long-term hemostatic efficacy, consistent safety results in clinical studies, efficacy from our pivotal study that is consistent with propensity scoring, the clearance of the vector, and on health-related quality of life over six years," said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. "We look forward to an anticipated approval of valoctocogene roxaparvovec in Europe and providing a therapy that could represent an important and valuable treatment choice for patients with severe Hemophilia A by offering the potential to reduce both the burden of the disease as well as the burden of treatment."

"BioMarin continues to increase and share important data about investigational valoctocogene roxaparvovec that may be useful for patients and physicians to evaluate therapeutic options based on an individual's unique circumstances," said one of the presenters Professor Johnny Mahlangu, a study investigator and Professor in Haematology and Head of School of Pathology in the Faculty of Health Sciences of the University of the Witwatersrand in Johannesburg, South Africa.

Presentation of these data at ISTH follows the Committee for Medicinal Products for Human Use (CHMP) adopting a positive opinion recommending conditional marketing authorization (CMA) for valoctocogene roxaparvovec for adults with severe hemophilia A. Valoctocogene roxaparvovec is the first gene therapy to be recommended for approval in Europe for hemophilia A. A final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in Q3 2022.

BioMarin's presentations at ISTH include:

Oral Presentations

Comparative effectiveness of valoctocogene roxaparvovec and prophylactic factor VIII replacement estimated through propensity scoring

Anthony J. Hatswell, PhD, Director and Analyst, Delta Hat Limited, Nottingham, UK

Sunday July 10, 2022, 3:45 PM - 4:00 PM BST

Relationship between transgene-produced FVIII and bleeding rates 2 years after gene transfer with valoctocogene roxaparvovec: Results from GENEr8-1

Professor Johnny Mahlangu, Professor of Haematology and Head of School of Pathology in the Faculty of Health Sciences of the University of the Witwatersrand in Johannesburg, South Africa.

Sunday July 10, 2022, 3:15 PM - 3:30 PM BST

Hemostatic results for up to 6 years following treatment with valoctocogene roxaparvovec, an AAV5-hFVIII-SQ gene therapy for severe hemophilia A

Professor Michael Laffan, faculty of Medicine, Department of Immunology and Inflammation at Imperial College London, Director of the Hammersmith Hospital Haemophilia Centre

Sunday July 10, 2022, 3:00 PM - 3:15 PM BST

Innate and Adaptive Immune Responses to Adeno-associated viral Gene Therapy in the Severe Hemophilia A Dog Model

Paul Batty, MBBS, PhD, Associate Professor at University College London and an Honorary Consultant in Haemostasis and Thrombosis at the Katharine Dormandy Haemophilia and Thrombosis Centre (Royal Free Hospital, London, UK)

Saturday, July 9, 2022, 2:00 PM 2:15 PM BST

Posters

Comparability of bleeding outcomes by prophylactic FVIII replacement intensity: A post hoc analysis of a noninterventional study of men with severe hemophilia A

Charlotte Camp, MSc, Associate Director, Health Economics and Outcomes Research, BioMarin

Monday July 11, 2022, 6:30 PM - 7:30 PM BST

Blood Biodistribution and Vector Shedding of Valoctocogene Roxaparvovec in People with Severe Hemophilia A: Results from the Phase 3 GENEr8-1 Trial

Suresh Agarwal, PhD, MS, RPh, Director, Clinical Pharmacology, BioMarin

Sunday July 10, 2022, 6:30 PM - 7:30 PM

About Hemophilia A

People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A (FVIII levels <1%) often experience painful, spontaneous bleeds into their muscles or joints. Individuals with the most severe form of hemophilia A make up approximately 50 percent of the hemophilia A population. People with hemophilia A with moderate (FVIII 1-5%) or mild (FVIII 5-40%) disease show a much-reduced propensity to bleed. The standard of care for individuals with severe hemophilia A is a prophylactic regimen of replacement Factor VIII infusions administered intravenously up to two to three times per week or 100 to 150 infusions per year. Despite these regimens, many people continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life.

Hemophilia A, also called Factor VIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective Factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have Hemophilia A.

About BioMarin

BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening rare diseases and medical conditions. The Company selects product candidates for diseases and conditions that represent a significant unmet medical need, have well-understood biology and provide an opportunity to be first-to-market or offer a significant benefit over existing products. The Company's portfolio consists of seven commercial products and multiple clinical and preclinical product candidates for the treatment of various diseases. For additional information, please visitwww.biomarin.com.

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including without limitation, statements about: the data presented at ISTH, including the four oral and two poster presentations, the development of BioMarin's valoctocogene roxaparvovec program generally, the impact of valoctocogene roxaparvovec gene therapy for treating patients with severe hemophilia A, a final approval decision for valoctocogene roxaparvovec, typically consistent with the CHMP recommendation, expected from the European Commission in Q3 2022 and the potential to transform the lives of these patients and the ongoing clinical programs generally. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: results and timing of current and planned preclinical studies and clinical trials of valoctocogene roxaparvovec, including final analysis of the data from these trials and the entire development program, including further assessment of safety events, any potential adverse events observed in the continuing monitoring of the patients in the clinical trials; the content and timing of decisions by the FDA, the EMA and other regulatory authorities; the content and timing of decisions by local and central ethics committees regarding the clinical trials; our ability to successfully manufacture valoctocogene roxaparvovec; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2022 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

BioMarin is a registered trademark of BioMarin Pharmaceutical Inc.

Contacts:

Investors

Media

Traci McCarty

Debra Charlesworth

BioMarin Pharmaceutical Inc.

BioMarin Pharmaceutical Inc.

(415) 455-7558

(415) 455-7451

SOURCE BioMarin Pharmaceutical Inc.

Follow this link:
BioMarin Presents Findings from Studies of Valoctocogene Roxaparvovec, Investigational Gene Therapy, at the International Society on Thrombosis and...

Gene Editing Market Is Expected To Reach USD 13.77 Billion By 2027 At A CAGR Of 18 percent.

Maximize Market Research has published a report on theGene Editing Marketthat provides a detailed analysis for the forecast period of 2022 to 2027.

Gene Editing Market Scope:

The report provides comprehensive market insights for industry stakeholders, including an explanation of complicated market data in simple language, the industrys history and present situation, as well as expected market size and trends. The research investigates all industry categories, with an emphasis on key companies such as market leaders, followers, and new entrants. The paper includes a full PESTLE analysis for each country. A thorough picture of the competitive landscape of major competitors in the Gene Editing market by goods and services, revenue, financial situation, portfolio, growth plans, and geographical presence makes the study an investors guide.

Request Free Sample:@https://www.maximizemarketresearch.com/request-sample/2748

Gene Editing Market Overview:

The prospect of treating genetic abnormalities, infectious illnesses, and cancer via gene editing is promising. Gene editing is a form of genetic modification in which, with the use of a designed nuclease or molecular scissor, DNA is added to, removed from, or replaced in an organisms genome in order to treat a specific condition. These nucleases damage DNA at specified sites in the genome to produce double-strand breaks. Targeted mutations result from the repair of the generated double-stranded breaks by non-homologous end joining or homologous recombination (edits).

Gene Editing Market Dynamics:

Gene-editing technologies ongoing technological breakthroughs are a key element fueling the markets growth. Additionally, government financing is readily available, there is more genomics research underway, and cancer and other genetic illnesses are becoming more common, all of which contribute to the markets growth. The rise of the genome editing market is also aided by the creation of breakthrough diagnostic tools based on CRISPR that help to lessen the negative effects of the COVID-19 pandemic.

However, there are several constraints that limit the markets growth to some extent, including the high cost of genomic equipment and rising worries about the dangers and moral dilemmas related to genome editing. However, the presence of major players in developing nations and applications in various stages of the drug development process are a few variables that soon open up profitable potential for the genome editing industry.

The gene editing market appears to be fragmented because there are numerous major and minor significant players. These market participants look to gain a competitive edge through strategic actions like partnerships, acquisitions, growths, collaborations, and the introduction of new products and technologies. Growing investments in genomics R&D, the prevalence of hereditary diseases and chronic disorders, and rapid advancements in genetic engineering are key factors supporting the market share for gene editing.

The market for genome alteration is hampered by certain problems. It is expected that ethical issues with genetic editing, such as safety and the high cost of gene therapies, may somewhat restrain market growth over the predicted years. Market limitations also are brought about by rigorous regulatory rules and unfavourable public opinions of genetic research.

The leading companies in the gene editing industry may face difficulties due to a number of market-related worries. This includes the price of the available genetic editing tools. The markets potential growth may be constrained by expensive maintenance and treatment expenses for genome alteration therapies and associated treatments, as well as by a lack of infrastructure in medium- and low-income countries.

Gene Editing MarketRegional Insights:

During the forecast period, North America is expected to hold a sizable portion of the worldwide genome editing market. The market in the region is expected to expand in the coming years due to the development of gene therapy in the United States, an increase in the consumption of genetically modified crops, an increase in the prevalence of infectious diseases and cancer, and the accessibility of research grants and funding.

Gene Editing MarketSegmentation:

By Technology:

By Application:

By End User:

Gene Editing Market Key Competitors:

To Get A Copy Of The Sample of the Gene Editing Market, Click Here:@https://www.maximizemarketresearch.com/market-report/gene-editing-market/2748/

About Maximize Market Research:

Maximize Market Research is a multifaceted market research and consulting company with professionals from several industries. Some of the industries we cover include medical devices, pharmaceutical manufacturers, science and engineering, electronic components, industrial equipment, technology and communication, cars and automobiles, chemical products and substances, general merchandise, beverages, personal care, and automated systems. To mention a few, we provide market-verified industry estimations, technical trend analysis, crucial market research, strategic advice, competition analysis, production and demand analysis, and client impact studies.

Contact Maximize Market Research:

3rd Floor, Navale IT Park, Phase 2

Pune Banglore Highway, Narhe,

Pune, Maharashtra 411041, India

[emailprotected]

View original post here:
Gene Editing Market is Expected to Reach USD 13.77 Billion With CAGR of 18% By Forecast 2027 Says Maximize Market Research (MMR) - Digital Journal

Contributed Commentary by Per Hammer, Cytiva

July 15, 2022 | Historically, the heavily regulated biopharma industry has been slow to adopt new technologies. However, a shift toward automation is vital to ensure that next-generation solutionssuch as cell and gene therapiesare produced at scale.

Less than one in five senior pharma executives strongly believe that frontier technologies, such as artificial intelligence, are widely adopted to support automation and increase the speed of specific processes. With cell therapies approved by global regulatory bodies, it is time to accelerate smart technologies and cell and gene therapy manufacturing.

Todays cell therapy treatments are often made on a small-scale, include manual preparation steps, and are produced for a clinical trial setting. Researchers spend days processing cellular material, monitoring its growth during the expansion phase, and preparing for re-administration to the patient. This process is demonstrated in administering autologous treatments so that every patient receives a unique living drug.

Though the current process is complex, it offers inspiring outcomes. For example, on April 1, 2022, the Food and Drug Administration (FDA) approved Kite Pharmas Yescarta, a chimeric antigen receptor (CAR) T-cell therapy for adult patients with large B-cell lymphoma. This kind of cancer is usually resistant to initial treatment and relapses within one year. With FDA approval, Yescarta (axicabtagene ciloleucel) is now the second-line treatment, a first for an autologous CAR T-cell therapy.

Cell Therapy Enters Mainstream

The exceptional results emerging from cell therapy clinical trials suggest we are entering a new phase of medical treatmentone where we can expect far more from our healthcare interventions than we ever imagined. Following the regulatory approval of autologous CAR T-cell therapies, the global cancer treatment landscape is changing, and the future is bright.

The success of COVID-19 vaccines signaled the arrival of the genomic medicines ageone where we hope to see cell and gene therapies deliver long-term remission and even cures for patients with some of the most complex diseases. According to the Alliance for Regenerative Medicine 2021 Annual Report, nearly 60% of the ongoing regenerative medicine clinical trials studied prevalent diseases by the end of the calendar year. But to get these powerful treatments to those who need them, we must have an automated manufacturing infrastructure that can generate cell therapies to meet increased demand in the coming years.

Saving Time Through Automation

Time is of the utmost importance, as biopharma manufacturing involves patient cells that have limited viability. Manual approaches to cell therapy production are time-consuming, and tasks such as checking cells at regular intervals during expansion are laborious. Another time-draining factor is the workflow and cleaning routines involved in maintaining a safe lab environment.

Automated solutions reduce or remove many of these challenges. After setting up a process, an operator can focus on other things while critical parameters such as temperature, pH level, gas transfer, and flow rates are monitored and controlled without human intervention.

Reducing Risks for Better Results

Manual cell processing solutions are complex, with many checkpoints across isolation, expansion, harvesting, and preservation stages. Unfortunately, each of these steps increases the risk potential. Despite the research teams expertise, there is still a chance that materials could be inadvertently contaminated during numerous open stages.

Additionally, limited process control can lead to difficulties in achieving high reproducibility. An automated modular solution minimizes these risks by bringing multiple steps within a closed, highly regulated, and controlled system.

Improving Manufacturing Efficiency

Changing a manufacturing process requires multiple manual routines and adjustments that must be checked and documented. However, documentation and protocols are less helpful when a customized process is used because they only apply to that specific setup.

Standardization would effectively improve manufacturing efficiency. This approach would ensure that what is learned in one project can be referenced in future work, with data and documentation applicable across different technology applications. A modular chain of connected systems allows for process variation with instruments running in customized configurations. Additionally, having control of an individual instrument leads to the straightforward use of built-in software and sensors.

Automated Manufacturing: The Way Forward for Cell and Gene Therapy

By using automated manufacturing to minimize human interaction, time, and resource requirements, it is possible to increase production speed and lower some risks and costs associated with commercialization.

The industry is ever-changing and adjusting its complex, yet exciting challenges will take some time. However, automation can create a significant advantage over competitors, providing the tools needed to produce cell therapies with the highest levels of safety and efficacy for patients.

Per Hammer has two decades of experience in the biopharma industry, mainly supporting customers in academics through process development and manufacturing. Per joined Cytiva in 2001, taking on several distinct roles in the company. Most recently, he progressed from Product Manager Leader for the Bioprocess Automation and Digital Team to Senior Global Product Manager for the Cell & Gene Therapy Automation and Digital Solutions. He can be reached at per.hammer@cytiva.com.

Read the original:
Automating the Genomic Medicines of the Future - Bio-IT World

- Study participants had an 81%-98% reduction in annualized anti-VEGF injections and demonstrated continuous aflibercept expression levels through three years

- Mean best-corrected visual acuity and central subfield thickness were maintained or improved in subjects treated with ADVM-022

-The Phase 2 LUNA trial is expected to dose the first subject in the third quarter of 2022 and preliminary data anticipated throughout 2023

REDWOOD CITY, Calif., July 15, 2022 (GLOBE NEWSWIRE) -- Adverum Biotechnologies Inc. (Nasdaq: ADVM), a clinical-stage company that aims to establish gene therapy as a new standard of care for highly prevalent ocular diseases, today announced new data from the OPTIC study treating wet age-related macular degeneration (wet AMD) during the American Society of Retina Specialists (ASRS) 2022 Annual Meeting. New data presented are as of February 24, 2022 and include best-corrected visual acuity (BCVA) and central subfield thickness (CST) maintenance, as well as reduction in CST fluctuation after a single, in-office intravitreal (IVT) injection of ADVM-022, ixoberogene soroparvovec (Ixo-vec), in subjects requiring frequent anti-VEGF injections for their wet AMD.

We are pleased to present our findings on BCVA and CST from the OPTIC trial, establishing that maintenance in both BCVA and CST, as well as a reduction in CST fluctuations, were sustained through at least two years, suggesting the long-term durability of Ixo-vec after a single IVT injection, stated Richard Beckman, M.D., chief medical officer at Adverum Biotechnologies. As several publications have recently highlighted, retinal CST fluctuations over time are associated with poor long-term visual outcomes for patients. The combination of durable maintenance of BCVA and reduced CST fluctuations in subjects who previously required frequent IVT injections further support our belief that Ixo-vec can provide better long-term benefit for wet AMD patients. We are excited by the possibility of extending the treatment benefit for this lifelong disease from the order of months to the order of years.

Data Highlights as of February 24, 2022

We are excited to present these encouraging data, showing continuous and stable aflibercept expression for over three years in our OPTIC trial in subjects with wet AMD, commented Laurent Fischer, M.D., president and chief executive officer at Adverum Biotechnologies. As we recently announced, we completed our IND amendment with the U.S. Food and Drug Administration, received European Medicines Agency PRIME designation, and are preparing to initiate the Phase 2 LUNA trial of Ixo-vec in wet AMD. The LUNA trial was designed after a detailed data review from all 55 participants treated to date with a single ADVM-022 injection. Four new enhanced prophylactic steroid regimens will be evaluated in LUNA with the aim of providing steroid coverage during the period of peak immunogenicity. Our goal is to enhance the safety profile of ADVM-022 while building upon the impressive efficacy profile we continue to see in the OPTIC trial. We plan to dose the first subjects in the third quarter of 2022 and anticipate preliminary data from LUNA throughout 2023 and look forward to providing more detail on the expected timeline once we can assess the pace of enrollment.

The LUNA trial is a multicenter, double-masked, randomized, parallel-group Phase 2 trial evaluating two doses of Ixo-vec, including 2E11 and a new, lower 6x10^10 vg/eye (6E10) dose, in up to 72 patients with wet AMD. The LUNA trial will assess four new enhanced prophylactic steroid regimens, including local steroids and combinations of local and systemic steroids to test the relative contribution of local versus systemic AAV exposure on ocular inflammation. Specific regimens include topical difluprednate (Durezol), IVT Ozurdex, or a combination of either topical Durezol or IVT Ozurdex with oral prednisone.

The trial will randomize the participants equally between the 2E11 and 6E10 Ixo-vec doses across four prophylactic steroid regimens and will be conducted at approximately 40 sites in the U.S. and Europe. The primary endpoints will be similar to the OPTIC trial and focus on mean change in BCVA and CST from baseline to one year, and incidence and severity of adverse events. Other data points will include protein expression of aflibercept starting at 10 weeks and an interim analysis at 26 weeks. The study will also evaluate the effectiveness and tolerability of the prophylactic steroid regimens.

As an investigator in OPTIC, I have observed the potential of ADVM-022 to significantly reduce the treatment burden for my patients with neovascular age-relatedmacular degeneration. In the latest data from the OPTIC trial, we have seen a manageable safety profile, robust aflibercept expression, and sustained anatomical improvements after two years following a single 2E11 dose of ADVM-022, said Dante Pieramici, M.D., partner, California Retina Consultants, and presenter of the data at ASRS. I look forward to participating as a LUNA investigator and building on the efficacy and safety profile in OPTIC with a new, lower 6E10 dose and to determine an optimal prophylactic steroid regimen for patients.

About Wet Age-Related Macular Degeneration

Wet AMD, also known as neovascular AMD or nAMD, is an advanced form of AMD, affecting approximately 10% of patients living with AMD. Wet AMD is a leading cause of blindness in patients over 65 years of age, with a prevalence of approximately 20 million individuals worldwide living with this condition. The incidence of new cases of wet AMD is expected to grow significantly worldwide as populations age. AMD is expected to impact 288 million people worldwide by 2040, with wet AMD accounting for approximately 10% of those cases.

About OPTIC Trial of ADVM-022 in Wet AMD

ADVM-022 is Adverums clinical-stage gene therapy product candidate being developed for the treatment of wet AMD. ADVM-022 utilizes a propriety vector capsid, AAV.7m8, carrying an aflibercept coding sequence under the control of a proprietary expression cassette. Unlike other ophthalmic gene therapies that require a surgery to administer the gene therapy under the retina (sub-retinal approach), ADVM-022 has the advantage of being administered as a one-time IVT injection in the office and is designed to deliver long-term efficacy and reduce the burden of frequent anti-VEGF injections, optimize patient compliance, and improve vision outcomes for patients with wet AMD.

The OPTIC trial is designed as a multi-center, open-label, dose-ranging, safety and efficacy trial of ADVM-022 in patients with wet AMD who have demonstrated responsiveness to anti-VEGF treatment. Patients in OPTIC are treatment-experienced, and previously required frequent anti-VEGF injections to manage their wet AMD and to maintain functional vision.

About Adverum Biotechnologies

Adverum Biotechnologies (NASDAQ: ADVM) is a clinical-stage company that aims to establish gene therapy as a new standard of care for a number of highly prevalent ocular diseases with the aspiration of developing functional cures for these diseases to restore vision and prevent blindness. Leveraging the research capabilities of its proprietary, intravitreal (IVT) platform, Adverum is developing durable, single-administration therapies, designed to be delivered in physicians offices, to eliminate the need for frequent ocular injections to treat these diseases. Adverum is evaluating its novel gene therapy candidate, ADVM-022, ixoberogene soroparvovec (Ixo-vec), as a one-time, IVT injection for patients with neovascular or wet age-related macular degeneration. By overcoming the challenges associated with current treatment paradigms for these debilitating ocular diseases, Adverum aspires to transform the standard of care, preserve vision, and create a profound societal impact around the globe. For more information, please visit http://www.adverum.com.

Forward-looking Statements

Statements contained in this press release regarding events or results that may occur in the future are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include but are not limited to statements regarding Adverums plans to initiate a Phase 2 study in wet AMD to investigate the 2x10^11 vg/eye dose and a lower 6x10^10 vg/eye dose of ADVM-022, as well as new enhanced prophylactic steroid regimens, including local steroids and a combination of local and systemic steroids, planned for the third quarter of 2022, as well as the benefits Adverum expects from this trial, and the timing of preliminary data from the LUNA trial. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, including risks inherent to, without limitation: Adverums novel technology, which makes it difficult to predict the timing of commencement and completion of clinical trials; regulatory uncertainties; enrollment uncertainties the results of early clinical trials not always being predictive of future clinical trials and results; and the potential for future complications or side effects in connection with use of ADVM-022. Additional risks and uncertainties facing Adverum are set forth under the caption Risk Factors and elsewhere in Adverums Securities and Exchange Commission (SEC) filings and reports, including Adverums Quarterly Report on Form 10-Q for the quarter ended March 31, 2022 filed with the SEC on May 12, 2022. All forward-looking statements contained in this press release speak only as of the date on which they were made. Adverum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.

Corporate & Investor Inquiries

Anand ReddiVice President, Head of Corporate Strategy and External Affairs & EngagementAdverum Biotechnologies, Inc.T: 650-649-1358E: areddi@adverum.com

Media

Megan TalonAssociate Director, Corporate CommunicationsAdverum Biotechnologies, Inc.T: 650-649-1006E: mtalon@adverum.com

Read more:
Adverum Biotechnologies Presents Best-Corrected Visual Acuity and Central Subfield Thickness Analyses After a Single IVT Injection of ADVM-022...

According to the latest report by MarketandResearch.biz, titled Global Osteoarthritis Gene Therapy Market from 2022 to 2028, covers reliable and updated information, quantitative and qualitative assessments of industry analysts and key competitors across the industry value chain. The report contains a lot of features of the global Osteoarthritis Gene Therapy industry which includes general market conditions, trends, inclinations, key players, opportunities, and geographical analysis.

This research helps the clients with understanding the global Osteoarthritis Gene Therapy market in terms of its definition, division, market potential, compelling patterns, and the difficulties that the market is facing. The report deeply tracks factors such as growth, limitations, and the planned characteristics of each point related to this market.

DOWNLOAD FREE SAMPLE REPORT: https://www.marketandresearch.biz/sample-request/224119

The report presents decisive clarity towards highlighting the most effective segment that enables heavy revenue flow. Relevant details about other market segments are also discussed in the report to show logical conclusions about the most prominent segments in the global Osteoarthritis Gene Therapy market. The report is divided into various segments such as by-products, applications, and regions which will give a universal view of the market growth.

The well-established players in the global Osteoarthritis Gene Therapy market are:

In market segmentation by types, the report covers:

In market segmentation by applications, the report covers the following uses:

Major regions discussed in the research report of the market include:

ACCESS FULL REPORT: https://www.marketandresearch.biz/report/224119/global-osteoarthritis-gene-therapy-market-growth-status-and-outlook-2021-2027

The pointers covered in the market dynamics are the drivers, restraints, and opportunities which affect the growth of the global Osteoarthritis Gene Therapy market during 2022 to 2028 period. The global regional analysis of the market was conducted and is mentioned in the report. Moreover, all the key pointers are explained briefly on a regional basis. The report also includes Porters Five Forces model to give the competitive scenario of the global Osteoarthritis Gene Therapy market.

The report covers expected revenue growth, potential lucrative opportunities, product ranges, pricing factors, and parameters to confer the emerging and new entrants in the industry with structured market data. The report caters to know the dynamics of the global Osteoarthritis Gene Therapy market opportunities and anticipate change.

Customization of the Report:

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

Contact UsMark StoneHead of Business DevelopmentPhone: +1-201-465-4211Email: [emailprotected]

Other Related Reports:

https://www.marketwatch.com/press-release/global-smart-flat-tv-market-2022prime-factors-segments-insights-technology-advancement-outlook-and-growth-forecast-up-to-2028-2022-07-13

https://www.marketwatch.com/press-release/global-polyurethane-waterproof-paint-market-2022-regional-overview-product-analysis-growth-elements-and-recent-development-to-2028-2022-07-13

https://www.marketwatch.com/press-release/global-hereditary-angioedema-therapeutic-market-trend-analysis-and-revenue-growth-over-forecast-of-2022-2028-2022-07-13

https://www.marketwatch.com/press-release/global-yttrium-hydroxide-market-2022-growth-analysis-key-stakeholders-regional-outlook-end-user-applicants-by-2028-2022-07-13

https://www.marketwatch.com/press-release/global-xanthan-gum-for-pharmaceutical-market-2022future-trends-swot-analysis-remarkable-growth-and-competitive-landscape-by-2028-2022-07-13

https://www.marketwatch.com/press-release/global-polysomnography-systems-market-2022regional-scope-growth-statistics-demand-and-regional-outlook-2028-2022-07-13

https://www.marketwatch.com/press-release/global-sodium-chlorite-for-metal-surface-treatment-marketrising-demands-size-and-statistics-industry-share-and-precise-outlook-20222028-2022-07-13

https://www.marketwatch.com/press-release/global-language-education-software-market-2022regional-overview-substantial-demand-vendor-landscape-and-trends-by-2028-2022-07-13

https://www.marketwatch.com/press-release/global-wool-insole-market-2022-overview-by-rising-demands-and-precise-outlook-by-2028-2022-07-13

https://www.marketwatch.com/press-release/global-external-cables-and-connectors-market-2022-regional-overview-product-analysis-growth-elements-and-recent-development-to-2028-2022-07-13

Read the original here:
Global Osteoarthritis Gene Therapy Market 2022 Industry Growth and Product Scope 2028 Top Players as Medipost, Mundipharma Travel Adventure Cinema -...

Via this genetic engineering experiment, theyve literally injected seeds of demise into everyday people like a cockroach spray. Based on a 2011 estimate, he believes an extra 700 million will be killed from this bioweapon and theyve known about the risks since 2005.

In this revealing interview with Greg Hunter of USAWatchdog.com, David Martin, Ph.D., presents evidence that COVID-19 injections are not vaccines but bioweapons that are being used as a form of genocide across the global population.1

In March 2022, Martin filed a federal lawsuit against President Biden, the Department of Health and Human Services and the Centers for Medicare and Medicaid Services alleging that COVID-19 shots turn the body into a biological weapons factory, manufacturing spike protein. Not only is the term vaccination misleading when referring to COVID-19 shots, its inaccurate since they are actually a form of gene therapy.2

And we are not only not going to be sued for, you know, any libel or misinformation, we are actually holding people criminally accountable for their domestic terrorism, their crimes against humanity and the story of the coronavirus weaponization that goes back to 1998, Martin says.3

Martin has been in the business of tracking patent applications and approvals since 1998. His company, M-Cam International Innovation Risk Management, is the worlds largest underwriter of intangible assets used in finance in 168 countries. M-Cam has also monitored biological and chemical weapons treaty violations on behalf of the U.S. government, following the anthrax scare in September 2001.4

According to Martin, there are more than 4,000 patents relating to the SARS coronavirus. His company has also done a comprehensive review of the financing of research involving the manipulation of coronaviruses that gave rise to SARS as a subclade of the beta coronavirus family.

Much of the research was funded by the National Institutes of Allergy and Infectious Diseases (NIAID) under the direction of Dr. Anthony Fauci.5Martin explained:6

I think its important for your listeners and viewers to remember that it was 1999 when Anthony Fauci and Ralph Baric at the University of North Carolina Chapel Hill decided to start weaponizing coronavirus they patented in 2002 and you heard that date correctly, thats a year before the SARS outbreak in China.

You know, they knew it was a bioweapon since 2005. They knew it was effective at harming populations, intimidating and coercing populations

According to Martin, the spike protein that the COVID-19 shots manufacture is a computer simulation of a chimera of the spike protein of coronavirus. It is, in fact, not a coronavirus vaccine. It is a spike protein instruction to make the human body produce a toxin, and that toxin has been scheduled as a known biologic agent of concern with respect to biological weapons for the last now decade and a half, he said.7

Rather than being a public health measure as they were widely campaigned to be, COVID-19 shots are an act of bioweapons and bioterrorism. Martin shared that in 2015, Dr. Peter Daszak, head of the EcoHealth Alliance that funneled research dollars from the NIAID to the Wuhan Institute of Virology for coronavirus research, stated:8

We need to increase public understanding of the need for medical countermeasures such as a pan-coronavirus vaccine. A key driver is the media and the economics will follow the hype. We need to use that hype to our advantage, to get to the real issues. Investors will respond if they see profit at the end of the process.

Daszak, who Martin refers to as the money launderer in chief, actually stated that this entire exercise was a campaign of domestic terror to get the public to accept the universal vaccine platform using a known biological weapon. And that is their own words, not my interpretation, Martin said.9

Both Pfizer and Modernas COVID-19 shots contain nucleic acid sequences that are not part of nature and have not been previously introduced to the human body. This amounts to a genetic engineering experiment that did not go through animal studies or clinical trials.

However, already people are dying from the shots and, Martin states, many more will due to issues such as blood clots, damage to the cardiovascular system and problems with liver, kidney and pulmonary function.10

An onslaught of reproductive and cancer cases related to the shots are also anticipated. The fact of the matter is an enormous number of people who are injected are already carrying the seeds of their own demise, Martin said.11As for how many may die, Martin believes the numbers may have been revealed back in 2011, when the World Health Organization announced their decade of vaccination:12

Based on their own 2011 estimate, and this is a chilling estimate, but we just have to put it out there When the Bill and Melinda Gates Foundation, the Chinese CDC, the Jeremy Farrar Wellcome Trust and others published The Decade of Vaccination for the World Health Organization back in 2011 their stated objective was a population reduction of 15% of the worlds population.

Put that in perspective, thats about 700 million people dead and that would put the U.S. participation in that certainly as a pro rata of injected population somewhere between 75 and 100 million people.

When asked what timeframe these people may die in, Martin suggested theres a lot of economic reasons why people hope that its between now and 2028.13This is because of a tiny little glitch on the horizon the projected illiquidity of the Social Security, Medicare and Medicaid programs by 2028.

So the fewer people who are recipients of Social Security, Medicare and Medicaid, the better, Martin said. Not surprisingly, its probably one of the motivations that led to the recommendation that people over the age of 65 were the first ones getting injected.14Other populations at risk are caregivers, including health care providers, and others in the workforce who were forced to be injected, such as pilots.

Why is it that were suddenly having 700 flights a day being canceled because, allegedly, airlines dont have pilots? the dirty secret is there a lot of pilots who are having microvascular problems and clotting problems, and that keeps them out of the cockpit, which is a good place to not have them if theyre going to throw a clot for a stroke or a heart attack,Martin said.

But the problem is were going to start seeing that exact same phenomenon in the health care industry and at a much larger scale, which means we now have, in addition to the problem of the actual morbidity and mortality, meaning people getting sick and people dying.

We actually have that targeting the health care industry writ large, which means we are going to have doctors and nurses who are going to be among the sick and the dead. And that means that the sick and the dying also do not get care.15

Its been stressed by the media and public health officials that COVID-19 shots do not alter DNA. However, Martin brings attention to a little-known grant from the National Science Foundation, known as Darwinian chemical systems,16which involved research to incorporate mRNA into targeted genomes. According to Martin:17

Moderna was started on the back of a 10-year National Science Foundation grant. And that grant was called Darwinian chemical systems the project that gave rise to the Moderna company itself was a project where they were specifically figuring out how to get mRNA to write itself into the genome of whatever target they were going after.

That could be a single-celled organism, it could be a multi-celled organism or it could be a human. And the fact of the matter is Moderna was started on the back of having proven that mRNA can be transfected and write itself into the human genome.

It is completely unknown what the short- or long-term effects of the spike protein analog thats inside people who received COVID-19 injections will be. But with respect to alteration of the genome, Martin states that data show mRNA has the capacity to write into the DNA of humans, and as such, the long-term effects are not going to merely be symptomatic. The long-term effects are going to be the human genome of injected individuals is going to be altered.18

The 2001 anthrax attack, which came out of medical and defense research, led to the passage of the PREP Act, which removed liability for manufacturers of emergency medical countermeasures.

This means that as long as the U.S. is under a state of emergency, things like COVID-19 vaccines are allowed under emergency use authorization. And as long as the emergency use authorization is in effect, the makers of these experimental gene therapies are not financially liable for any harm that comes from their use.

That is, provided theyre vaccines. If these injections are NOT vaccines, then the liability shield falls away, because there is no liability shield for a medical emergency countermeasure that is gene therapy. Further, lawsuits that can prove the companies engaged in fraud will also negate the liability shield. Martin states:19

One of the convenient things about the PREP Act is the immunity shield from liability actually is only as good as the absence of fraud. Because if there was fraud in the promulgation of the events, leading to an emergency use authorization, then all of the immunity shield gets wiped out.

So the reason why it is so important for conversations like the one were having to actually be promoted and be advanced is because the pharmaceutical companies and this includes Pfizer and Moderna and J&J know they are perpetuating a fraud. The great thing about this is when that fraud is established, 100% of the liability flows back to them.

when a fraud was the basis for a fraud, then we actually have a number of other legal remedies that allow you to pierce that veil. So in the end, theres no question and its quite evident based on the current mortality and morbidity data that given the fact that when it comes to biological weapons and bioterror each count comes with $100 million penalty. Thats what the federal statute gives us.

The penalty for corporate domestic terrorism, when you have per count $100 million a pop liabilities that is an existential threat that takes a company like Pfizer or takes a company like Moderna out of existence. And that is what were working for every day.

If youd like to follow the progress of the ongoing legal cases seeking to expose the truth that a criminal organization is seeking to obtain control over the global population via the creation of patented bioweapons marketed as novel viruses and injections you can find all the details at ProsecuteNow.io, a website compiled by Martin and colleagues.20

Originally publishedJuly 16, 2022 on Mercola.com

Views expressed in this article are the opinions of the author and do not necessarily reflect the views of The Epoch Times. Epoch Health welcomes professional discussion and friendly debate. To submit an opinion piece, please follow these guidelines and submit through our form here.

See original here:
Will 100 Million Die From the COVID Vax by 2028? - The Epoch Times

Gene Synthesis Market report has a lot of features to offer for Gene Synthesis industry which includes general market conditions, trends, inclinations, key players, opportunities, and geographical analysis. This report emphasizes on changing dynamics, growth-driving factors, restraints, and limitations. It includes a detailed analysis of the Gene Synthesis market in customer requirements, customer preferences, and the competitive landscape of the overall market. This report employs SWOT analysis technique for the assessment of the development of the most remarkable market players. An influential Gene Synthesis market report offers broader perspective of the market place with its comprehensive market insights and analysis.

Gene Synthesis business report makes available an overview of the Gene Synthesis industry which is gaining momentum in the last few years. The market report uses a range of steps for collecting, recording, analysing and interpreting market data to make this report all-inclusive. It also offers an outline of the industry that might promote interest among prospective investors, large corporations and everyday users who could participate in the next big opportunity or make their lives just a little easier. The world class Gene Synthesis report comprises of the list of leading competitors, strategic industry analysis and the insights of key factors influencing the Gene Synthesis industry.

Download Sample [emailprotected] https://www.databridgemarketresearch.com/request-a-sample/?dbmr=global-gene-synthesis-market

Data Bridge Market Research analyses that the market is growing with a CAGR of 27.0% in the forecast period and is expected to reach USD 21,156.93 million by 2028. Rising prevalence of infectious diseases demands the novel and advanced gene therapy products, vaccines, personalized medicines among others which can be achieved by using gene sequences, thus acts as driver for the gene synthesis market growth.

Gene synthesisis technique of synthetic biology which is employed for the production or creation of artificial gene under laboratory conditions. Gene synthesis technique is used for creating fusion proteins, molecular cloning or in order to achieve high level of protein expression. Traditionally, molecular cloning techniques used which consumes a lot of time and requires a lot of money investment.

Rise in adoption of gene therapy has enhanced the requirement of rapid gene synthesis products which provides with error free gene sequences in less interval of time and hence acts as driver for the gene synthesis market. The ethical issues associated with genetic engineering lowers the demand of gene constructs having the potential to interfere with organisms genomic composition which is restraining the gene synthesis market growth. Increasing demand of personalized medicines has paved the way for genomic which demands the error free gene sequences in at low cost in less span of time, thus acts as an opportunity for the gene synthesis market. Long approval time for gene synthesizer and products synthesized through these products restrict the product manufacturers to earn potential benefit in the market and impacts the overall market size, hence acts as challenge for the gene synthesis market growth.

Market Players Covered:

Eurofins Genomics, ATG:biosynthetics GmbH, Merck KGaA, Aldevron, Synbio Technologies, Thermo Fisher Scientific Inc, Genscript, General Biosystems, Twist Bioscience, Evonetix, Bio-synthesis Inc, LGC Limited, Eurofins Genomics Blue Heron LLC, Macrogen,Inc, Science Exchange, Inc, Integrated DNA Technologies, Inc, Origene Technologies, INC., Proteo Genix, Biocat GMBH among other domestic players. DBMR analysts understand competitive strengths and provide competitive analysis for each competitor separately.

Browse Full Report Along With Facts and Figures @https://www.databridgemarketresearch.com/reports/global-gene-synthesis-market

The gene synthesis market is analysed and market size information is provided by the country, product & services, gene type, product type, application, method, end user and distribution channel as referenced above.

The countries covered in the gene synthesis market report are the U.S., Canada and Mexico, U.K., Germany, France, Spain, Italy, Netherlands, Switzerland, Russia, Turkey, Austria, Ireland, and Rest of Europe, China, South Korea, Japan, India, Australia, Singapore, Malaysia, Indonesia, Thailand, Philippines and rest of Asia-Pacific, South Africa, Saudi Arabia, U.A.E., Israel, Egypt and rest of Middle East and Africa, Brazil, Argentina, and rest of South America.

Software and services segment.in North American region is expected to grow with the highest growth rate in the forecast period of 2021 to 2028 because of increasing gene therapy based medications. The software and services segment in Germany is dominating the European market owing to increasing research and development for the gene synthesis used in vaccine preparation. China is leading the growth of the Asia-Pacific market and Software and services segment is dominating in this country due to presence of highest number of gene synthesis providers.

The country section of the report also provides individual market impacting factors and changes in regulation in the market domestically that impacts the current and future trends of the market. Data points such as new sales, replacement sales, country demographics, regulatory acts and import-export tariffs are some of the major pointers used to forecast the market scenario for individual countries. Also, presence and availability of global brands and their challenges faced due to large or scarce competition from local and domestic brands, impact of sales channels are considered while providing forecast analysis of the country data.

Highlights of TOC:

Chapter 1: Market overview

Chapter 2: Global Gene Synthesis Market

Chapter 3: Regional analysis of the Global Gene Synthesis Market industry

Chapter 4: Gene Synthesis Market segmentation based on types and applications

Chapter 5: Revenue analysis based on types and applications

Chapter 6: Market share

Chapter 7: Competitive Landscape

Chapter 8: Drivers, Restraints, Challenges, and Opportunities

Chapter 9: Gross Margin and Price Analysis

Check Complete Table of Contents with List of Table and Figures @ https://www.databridgemarketresearch.com/toc/?dbmr=global-gene-synthesis-market

Highlights of Following Key Factors of Global Gene Synthesis Market

A detailed description of the companys operations and business divisions

Analysts summarization of the companys business strategy

Progression of key events associated with the company

A list of major products, services and brands of the company

A list of key competitors to the company

A list and contact details of key locations and subsidiaries of the company

The latest financial ratios derived from the annual financial statements published by the company with 5 years history.

Browse Related Reports:

Psychedelic Drugs Market Global Trends, Share, Industry Size, Growth, Demand, Opportunities and Forecast By 2029

Proton Therapy Market Growing Impressive Business Opportunities, Industry Trends, Global Demand and Future Scope

Gene Synthesis Market Global Industry Analysis by Size, Demand, Future Trends, Challenges, Growth Opportunities and Key Players | Global Market Expected to Grow at 22.3% CAGR forecast by 2028

Healthcare 3D Printing Market Growth at a Rate of 22.8% & Industry Size, Trends, Growth, Insights and Forecast by 2028

Fitness App Market Global Industry Analysis by Size, Demand, Future Trends, Challenges, Growth Opportunities and Key Players | Global Market Expected to Grow at 25.6% CAGR forecast by 2028

Laboratory Information Management Systems (LIMS) Market Growth at a Rate of 9.3% & Industry Size, Trends, Growth, Insights and Forecast by 2029

About Data Bridge Market Research:

Data Bridge set forth itself as an unconventional and neoteric Market research and consulting firm with unparalleled level of resilience and integrated approaches. We are determined to unearth the best market opportunities and foster efficient information for your business to thrive in the market. Data Bridge endeavors to provide appropriate solutions to the complex business challenges and initiates an effortless decision-making process.

Contact us:

Data Bridge Market Research

US: +1 888 387 2818

UK: +44 208 089 1725

Hong Kong: +852 8192 7475

Email:[emailprotected]

Visit link:
Gene Synthesis Market Registered Substantial CAGR Growth Of 27.0% Forecast to 2029 and Market Is Analyzed By Size, Trends, Analysis, Future Scope,...

4D Molecular Therapeutics (NASDAQ:FDMT Get Rating) is one of 264 public companies in the Biological products, except diagnostic industry, but how does it weigh in compared to its peers? We will compare 4D Molecular Therapeutics to similar companies based on the strength of its institutional ownership, analyst recommendations, earnings, valuation, profitability, risk and dividends.

This table compares 4D Molecular Therapeutics and its peers net margins, return on equity and return on assets.

76.6% of 4D Molecular Therapeutics shares are owned by institutional investors. Comparatively, 53.5% of shares of all Biological products, except diagnostic companies are owned by institutional investors. 11.2% of 4D Molecular Therapeutics shares are owned by company insiders. Comparatively, 16.4% of shares of all Biological products, except diagnostic companies are owned by company insiders. Strong institutional ownership is an indication that large money managers, endowments and hedge funds believe a stock will outperform the market over the long term.

4D Molecular Therapeutics peers have higher revenue and earnings than 4D Molecular Therapeutics. 4D Molecular Therapeutics is trading at a lower price-to-earnings ratio than its peers, indicating that it is currently more affordable than other companies in its industry.

This is a summary of current ratings and price targets for 4D Molecular Therapeutics and its peers, as provided by MarketBeat.

4D Molecular Therapeutics presently has a consensus target price of $26.00, suggesting a potential upside of 157.94%. As a group, Biological products, except diagnostic companies have a potential upside of 92.79%. Given 4D Molecular Therapeutics stronger consensus rating and higher possible upside, analysts clearly believe 4D Molecular Therapeutics is more favorable than its peers.

4D Molecular Therapeutics has a beta of 1.26, meaning that its stock price is 26% more volatile than the S&P 500. Comparatively, 4D Molecular Therapeutics peers have a beta of 0.75, meaning that their average stock price is 25% less volatile than the S&P 500.

4D Molecular Therapeutics beats its peers on 8 of the 13 factors compared.

(Get Rating)

4D Molecular Therapeutics, Inc., a clinical-stage gene therapy company, develops product candidates using its adeno-associated viruses vectors. It develops a portfolio of gene therapy product candidates focuses in three therapeutic areas: ophthalmology, cardiology, and pulmonology. The company has three product candidates that are in clinical trials: 4D-125 that is in a Phase 1/2 clinical trial for the treatment of X-linked retinitis pigmentosa; 4D-110 that is in a Phase 1/2 clinical trial for the treatment of choroideremia; and 4D-310, which is in a Phase 1/2 clinical trial for the treatment of Fabry disease. Its two IND candidates are 4D-150 for the treatment of wet age-related macular degeneration and 4D-710 for the treatment of cystic fibrosis lung disease. 4D Molecular Therapeutics, Inc. has research and collaboration arrangements with uniQure; CRF; Roche; and CFF. The company was founded in 2013 and is headquartered in Emeryville, California.4D Molecular Therapeutics, Inc., a clinical-stage gene therapy company, develops product candidates using its adeno-associated viruses vectors. It develops a portfolio of gene therapy product candidates focuses in three therapeutic areas: ophthalmology, cardiology, and pulmonology. The company has three product candidates that are in clinical trials: 4D-125 that is in a Phase 1/2 clinical trial for the treatment of X-linked retinitis pigmentosa; 4D-110 that is in a Phase 1/2 clinical trial for the treatment of choroideremia; and 4D-310, which is in a Phase 1/2 clinical trial for the treatment of Fabry disease. Its two IND candidates are 4D-150 for the treatment of wet age-related macular degeneration and 4D-710 for the treatment of cystic fibrosis lung disease. 4D Molecular Therapeutics, Inc. has research and collaboration arrangements with uniQure; CRF; Roche; and CFF. The company was founded in 2013 and is headquartered in Emeryville, California.

Receive News & Ratings for 4D Molecular Therapeutics Daily - Enter your email address below to receive a concise daily summary of the latest news and analysts' ratings for 4D Molecular Therapeutics and related companies with MarketBeat.com's FREE daily email newsletter.

Link:
Comparing 4D Molecular Therapeutics (FDMT) and Its Rivals - Defense World

BOYLE HEIGHTS, Calif., July 14, 2022 (GLOBE NEWSWIRE) -- Global biotherapeutics leader CSL Behring will host a Mark Cuban Foundation Artificial Intelligence (AI) Bootcamp for high school students in the Fall of 2022 in conjunction with the i.am/Angel Foundation. The No Cost AI Bootcamp in Boyle Heights will be targeted at high school students and will introduce underserved students in grades 9 through 12 to basic AI concepts and skills.

CSL Behring is one of 20+ host companies selected to host 30 camps across the US in Fall 2022.

The CSL Behring Bootcamp will be held over four consecutive Saturdays starting on October 22nd and ending on November 12th.

The student and parent application are now open at markcubanai.org/application. Students do not need any prior experience with computer science, programming, or robotics to apply and attend.

Throughout the AI Bootcamp, students will learn what artificial intelligence is and is not, where they already interact with AI in their own lives, and the ethical implications of AI systems including but not limited to TikTok recommendations, smart home assistants, facial recognition, and self-driving cars to name a few.

Students will benefit from volunteer mentor instructors who are knowledgeable about data science and able to help students quickly understand material normally taught at a collegiate level. As part of the 5-hour curriculum, students also get to work in Microsoft Azure, Microsoft's cloud computing platform, each day to build their own AI applications related to Chatbots, Computer Vision, Machine Learning, and Natural Language Processing.

The Mark Cuban Foundation provides the bootcamps curriculum materials, trains volunteer mentors, and recruits and selects local students to attend camp. In addition, the Mark Cuban Foundation and CSL Behring work together to provide food, transportation, and access to laptops for students at no cost throughout the duration of Bootcamp.

"This bootcamp was a great experience and opened my eyes to the multitude of ways that AI can be used. Would definitely recommend it!" - Nhaomi L., CSL Behring 2021

Founded by Mark Cuban in 2019, the AI Bootcamp initiative has hosted free AI bootcamps for students across several US cities, including Dallas, Pasadena, Chicago, Pittsburgh, Detroit, and Atlantic City to name a few. The Mark Cuban Foundation has impacted 450+ students to date and has a goal to have 1,000 students graduate from AI Bootcamps Program in 2023.

CSL Behring develops and delivers innovative therapies for people living with rare and serious conditions. The companys global cell & gene therapy research hub is located in nearby Pasadena.

Students interested in applying to the Mark Cuban Foundation AI Bootcamp should do so before Thursday, September 1st, 2022 at markcubanai.org/application. To see our 2022 camp locations and to learn more about the Mark Cuban Foundation AI Bootcamps, please visit markcubanai.org/faq.

Contact: Carli Lidiak, Mark Cuban FoundationPhone: 309-840-0348Email: carli.lidiak@markcubanai.org

See more here:
Boyle Heights Selected as Host Site for Artificial Intelligence Bootcamp APPLICATIONS NOW OPEN! - GlobeNewswire

A decade after CRISPR started to become a major tool in genetic research, a new generation of scientists is growing up with the technology. Even high school students are able to run CRISPR experiments. Some specialized public high schools teach CRISPR as a hands-on lesson in biotechnology. These classes cover everything from molecular biology to pipetting to biomedical ethics and career options.

Visualizing and comprehending whats happening on the molecular level is usually always the challenge, said Katy Gazda, a high school biotechnology teacher who taught CRISPR in her classroom last year. To help students better understand complex molecular movements, teachers use tools like paper models, 3-D printed models and online animations.

Note: Diagram is simplified.

1. Target the right gene

Scientists engineer a piece of RNA that is a match for the DNA they want to edit. This is called the guide RNA. Students can practice designing their own guide RNA sequences using the same free bioinformatics search tool that scientists use for research.

2. Bind the target

An enzyme called Cas9 binds to a piece of DNA and temporarily unwinds a section of the DNA. Students can model the process with paper cutouts, pushing a paper DNA sequence along a printed guide RNA until they match.

3. Cut the DNA

If the guide RNA matches a section of the DNA, the Cas9 enzyme cuts both strands of the DNA double helix. An interactive animation from the Howard Hughes Medical Institute shows students how the Cas9 enzyme changes its shape and snips through the DNA.

Repaired DNA with edited section

DNA fragments cut by Cas9

DNA fragments cut by Cas9

Repaired DNA with edited section

Note: Diagrams are simplified.

4. Repair and edit the DNA

Machinery inside the cell rushes to fix the broken DNA. One repair process uses a similar-looking, unbroken piece of DNA as a template to stitch the broken pieces back together.

Scientists can introduce tailor-made DNA into the cell tricking the repair machinery into using the engineered DNA as the template for stitching together the broken pieces.

Students also learn about real-life examples of DNA editing, such as therapies for genetic illnesses including sickle cell disease and cystic fibrosis. In some exercises, they can see examples of the actual DNA sequence associated with each illness and evaluate specific gene edits proposed to cure the illness.

Ms. Gazda believes that hands-on lab lessons help students open their minds to the idea that they can truly be a scientist. Several companies sell CRISPR curriculum kits to high schools and universities. One kit from Bio-Rad, a life science technology manufacturer, includes a prepackaged experiment using E. coli bacteria.

Bacteria Altered With CRISPR

E. coli

bacteria colonies

Petri

dishes full of

bacteria food

Petri

dish full of

bacteria food

Bacteria Altered With CRISPR

Petri

dish full of

bacteria food

A gene in the bacteria encodes an enzyme, called -gal, which can help break down certain molecules.

A bright blue color appears when X-gal is broken down by -gal.

A gene in the bacteria encodes an enzyme, called -gal, which can help break down certain molecules.

A bright blue color

appears when X-gal is

broken down by -gal.

Note: Diagrams and molecule names are simplified.

The E. coli bacteria used in the Bio-Rad kit are grown on a food mixture that includes a compound called X-gal. Normally, these bacteria are able to use an enzyme to break down the compound into two parts: a sugar molecule and an indicator molecule.

The indicator molecule turns deep blue, showing students that the bacterial enzyme is working. This colorful display is an important part of the experiment. It shows students what to expect in an unaltered, or control group a vital part of any scientific experiment.

The -gal enzyme is not produced.

The X-gal compound cannot be broken down without the -gal enzyme, so the bright blue indicator molecule is never produced.

After the students use CRISPR to transform a section of the gene, the gene is no longer functional.

After the students use CRISPR to transform a section of the gene, the gene is no longer functional.

The -gal enzyme is not produced. The X-gal compound cannot be broken down without the -gal enzyme, so the bright blue indicator molecule is never produced.

Note: Diagrams and molecule names are simplified.

The process of altering E. coli DNA with CRISPR involves lab techniques like pipetting liquids and carefully moving bacteria colonies. Teaching a new lab class like CRISPR can be intimidating, says Gregory Jubulis, a high school science teacher who uses the Bio-Rad kit in his biotechnology class. It takes you a few years before youre real comfortable with teaching something, he said.

But when classroom CRISPR lab kits first became available, he knew he wanted to teach it. I just want my kids to be ready for the future of science, he said.

Ms. Gazda uses lab classes as an opportunity to share career options with students. In a CRISPR lab, students may learn about careers in anything from molecular and cellular biology to entrepreneurship and science journalism.

Ethics always comes up, says Mr. Jubulis, explaining how he relates the lab experiment to real-life CRISPR applications like gene therapy. Many of his students have friends or family with genetic illnesses, so the topic can be deeply personal.

Read the rest here:
CRISPR in the Classroom - The New York Times

Chronic pain affects millions of people worldwide, yet popular treatments for pain including surgery and opioid medications can have disastrous side effects of their own. But with $1.8 million in funding from the National Institute of Neurological Disorders and Stroke (NINDS), a University of New England researcher will explore non-opioid treatments for chronic pain at the cellular level.

Benjamin Harrison, B.Sc., Ph.D., assistant professor of biochemistry and nutrition, will use the five-year R01 grant from the National Institutes of Health to study how to reduce the excitability of nociceptors, which are neurons that transmit pain signals in response to painful injuries.

Harrison and his team have discovered that nociceptors contain a protein called "CELF4, an RNA binding protein they theorize inhibits the production of pro-nociceptive, or pro-pain-sensing, cellular components. Harrisons research will focus on delivering CELF4 into pain neurons, where this protein will limit the synthesis of ion-channels, receptors, and other molecules that sensitize them.

Specifically, the researchers will study if a locally administered adeno-associated virus can stimulate production of CELF4 and reduce pain in those areas an approach known as gene vector therapy.

Harrison remarked that the innovative approach could prove beneficial for those living with chronic pain but who do not want to undergo surgeries which can be expensive and leave people with no sensation at all or use powerful pain-reducing medications like addictive opioids.

There are some chronic pain conditions that are simply intolerable, and people with those conditions are willing to do severe surgeries to reduce their pain, Harrison remarked. Using this novel gene therapy vector approach, we can develop pain therapies that are less invasive than surgery and carry fewer risks than conventional opioid medications.

Future directions for the research could include partnerships with clinicians for clinical trials, Harrison said.

Read the rest here:
UNE researcher awarded $1.8 million to study chronic pain relief through gene therapy - University of New England

PHILADELPHIA--(BUSINESS WIRE)-- SwanBio Therapeutics, a gene therapy company advancing AAV-based therapies for the treatment of devastating, inherited neurological conditions, today presented details about the companys ongoing natural history study of adrenomyeloneuropathy (AMN) at the 8th Congress of the European Academy of Neurology (EAN) in Vienna. The intention of the CYGNET study is to assess disease progression in patients with AMN to inform the research and development of potential treatments, including SwanBios lead candidate, SBT101, the first clinical-stage AAV-based gene therapy for AMN.

At SwanBio, our approach to drug development begins with deepening our understanding of the origin and trajectory of diseases like AMN, ideally enabling us to deliver gene therapies that are both highly effective and meaningful for the quality of life of patients. With this in mind, our proprietary CYGNET study was designed to better characterize AMN disease progression, which varies from person to person, said Steven Zelenkofske, D.O., chief medical officer, SwanBio Therapeutics. CYGNET is the first AMN clinical study to feature wearables, which may help us identify sensitive outcomes related to clinically relevant changes early in men with AMN.

SwanBios CYGNET natural history study will track a number of variables over a two-year period, including:

More details about the CYGNET study, including demographics and disease characteristics of the first 21 patients enrolled, were presented during a virtual poster session at EAN.

SwanBio anticipates recruiting approximately 80 patients for the CYGNET natural history study; as of early June, the study was over 40% enrolled across five different global sites. Clinicians or patients interested in learning more about this study can review SwanBios CYGNET flyer or contact SwanBio at clinicaltrials@swanbiotx.com.

Presentation Details ePoster EPO-599: CYGNET: A prospective multicenter observational study of disease progression in patients with adrenomyeloneuropathyDate & Time: June 27, 2022, 12:30 1:15 p.m. CEST

About SBT101 SBT101 is the first clinical-stage adeno-associated virus (AAV)-based gene therapy candidate for people with adrenomyeloneuropathy (AMN). SBT101 was designed to compensate for the disease-causing ABCD1 mutation. In preclinical studies, treatment with SBT101 demonstrated dose-dependent improvement of disease markers and functional improvement in AMN mouse models. SBT101 was also shown to be well-tolerated in non-human primates at six months post-treatment. The clinical program for SBT101 builds on this positive preclinical data, plus the companys already deep understanding of the underlying pathophysiology of the disease and the patient experience of AMN.

SwanBio expects to initiate a randomized, controlled Phase 1/2 clinical trial designed to assess the safety and efficacy of SBT101 in patients with AMN in the second half of 2022. In early 2022, the FDA cleared SwanBios Investigational New Drug application for SBT101 and granted SBT101 Fast Track and Orphan Drug Designation.

About Adrenomyeloneuropathy Adrenomyeloneuropathy (AMN) is a progressive and debilitating neurodegenerative disease caused by mutations in the ABCD1 gene that disrupt the function of spinal cord cells and other tissues. AMN is characterized by loss of mobility in adulthood, incontinence, pain, and sexual dysfunction, which all affect quality of life. Between 8,000-10,000 men in the United States and European Union are living with AMN. There are no approved therapies for the treatment of the disease; current standard of care is limited to symptom management.

About SwanBio Therapeutics SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, inherited neurological conditions. SwanBio is advancing a pipeline of gene therapies, designed to be delivered intrathecally, that can address targets within both the central and peripheral nervous systems. This approach has the potential to be applied broadly across three disease classifications spastic paraplegias, monogenic neuropathies, and polygenic neuropathies. SwanBios lead program is being advanced toward clinical development for the treatment of adrenomyeloneuropathy (AMN). SwanBio is supported by long-term, committed investment partners, including its primary investors Syncona, Ltd. (lead investor and majority shareholder) and Mass General Brigham Ventures. For more information, visit SwanBioTx.com.

References

View source version on businesswire.com: https://www.businesswire.com/news/home/20220627005114/en/

See more here:
SwanBio Presents Design of Innovative Natural History Study Aimed to Evolve Understanding of Adrenomyeloneuropathy and Inform Future Treatments -...

The Global Gene and Cell Therapy (GCT) Market is estimated at USD 2504.2 Million in the year 2020. Growth in the historic period in the cell and gene therapy market resulted from increase in investments in cell and gene therapies, growth in research and development, advances in cancer drug discovery, rise in public-private partnerships, strong economic growth in emerging markets, increased healthcare expenditure, and rising in pharmaceutical R&D expenditure.

Companies in the gene and cell therapy for oncology market are increasing their product innovation through strategic collaborations. To sustain in the increasingly competitive market, organizations are developing innovative products as well as sharing skills and expertise with other such enterprises. While oncology drug companies have long collaborated with each other as well as with academic and research institutions in this market by way of partnerships, in or out licensing deals, this trend has been increasing over the recent years.

Download Free Sample of This Strategic Report:https://reportocean.com/industry-verticals/sample-request?report_id=aa1096

Further, the market was restrained by inadequate reimbursements, challenges due to regulatory changes, low healthcare access, and limited number of treatment centers. Going forward, increasing prevalence of cancer and chronic diseases, rising geriatric population, rising focus on cell and gene therapy, and rise in healthcare expenditure will drive the growth in the gene and cell therapy market. Factors that could hinder the growth of the market in the future include high costs of therapy, stringent regulations, reimbursement challenges, and coronavirus pandemic.

Scope of the Report

The report presents the analysis of Gene and Cell Therapy market for the historical period 2016-2020 and forecast period of 2021-2026.The report analyses the Gene and Cell Therapy Market by value (USD Million).The report analyses the Gene and Cell Therapy Market by Vector (Lentivirus, AAV, Retrovirus & Gammaretrovirus and Others).The report analyses the Gene and Cell Therapy Market Application (Oncology, Neurological disorders, cardiovascular disorders, Others).The Global Gene and Cell Therapy Market has been analysed By Region (North America, Europe, Asia Pacific, LAMEA), By Country (United States, Canada, Germany, U.K, France, Italy, China, Japan, India and South Korea).The key insights of the report have been presented through the frameworks of the attractiveness of the market has been presented by region, by Vector, By Application.

SPECIAL OFFER (Avail an Up-to 30% discount on this report-https://reportocean.com/industry-verticals/sample-request?report_id=aa1096

Also, the major opportunities, trends, drivers and challenges of the industry has been analysed in the report.The report tracks competitive developments, strategies, mergers and acquisitions and new product development. The companies analysed in the report include F. Hoffman-La Roche Ltd., Novartis, Sanofi, Alnylam Pharmaceuticals Inc., Pfizer, BlueBird Inc., Sarepta Therapeutics, Voyager Therapeutics, Orchard Therapeutics Plc, AnGes Inc.

Key Target AudienceBiotechnology CompaniesPharmaceutical and Health CompaniesConsulting and Advisory FirmsGovernment and Policy MakersRegulatory Authorities

Table of Content:

Key Questions Answered in the Market Report

Request full Report-https://reportocean.com/industry-verticals/sample-request?report_id=aa1096

About Report Ocean:We are the best market research reports provider in the industry. Report Ocean believes in providing quality reports to clients to meet the top line and bottom line goals which will boost your market share in todays competitive environment. Report Ocean is a one-stop solution for individuals, organizations, and industries that are looking for innovative market research reports.

Get in Touch with Us:Report Ocean:Email:sales@reportocean.comAddress: 500 N Michigan Ave, Suite 600, Chicago, Illinois 60611 UNITED STATESTel:+1 888 212 3539 (US TOLL FREE)Website:https://www.reportocean.com

Read more here:
Gene and Cell Therapy (GCT) Market Report (COVID-19 Analysis) by Worldwide Market Trends & Opportunities and Forecast to 2030 Designer Women -...

that's the center of the macula right there. Okay, so if you had an area of degeneration over here or over here or over here, you would have no symptoms. But it's that center part that dr eric swan with the retina care center says causes vision loss once affected. And it can be *** little or *** lot depending on how much tissue has been lost due to age related dry macular degeneration. Typical patient comes to see me has the loss of central vision, they just can't read. They're having difficulties driving. And so this is *** huge impairment for patients because the tissue can't be regenerated. The goal is to slow progression. Dr swan is part of *** nationwide FDA approved investigative clinical trial. That involves *** one time surgery using *** patient's own cells to create more of *** certain protein. Thought to be low in patients with this eye disease. This is *** very novel technique. It's gene therapy. So we're actually injecting these genes underneath the retina and telling the cells to produce more of this protein. Dr swan is the first surgeon in Maryland to perform the surgery and says clinical trials are still in the early stages. Early detection is key and that's why exams he says are so important and why research is so crucial when it comes to quality of life issues like vision. It is I mean, you want your grandmother your love when your grandfather to actually be able to continue to drive and really to maintain their independence and that really is the most important for them, jennifer francie adi W. B. *** l tv 11 News.

Surgeon taking part in gene therapy trial to slow vision loss

Updated: 7:26 PM PDT Jun 25, 2022

A Baltimore doctor performed the first surgery in Maryland as part of a nationwide clinical trial to slow the progression of what's known as dry macular degeneration.Dr. Eric Suan, retinal surgeon at the Retina Care Center, said degeneration causes vision loss once affected. And it can be a little or a lot, depending on how much tissue has been lost due to age-related dry macular degeneration."The typical patient who comes to me has a loss of central vision," Suan said. "They just can't read, they're having difficulty driving, and so this is a huge impairment for patients."Because the tissue can't be regenerated, the goal is to slow progression.Suan is part of a nationwide Food and Drug Administration-approved investigative clinical trial that involves a one-time surgery using a patient's own cells to create more of a certain protein thought to be low in patients with this eye disease."This is a very novel technique. It's gene therapy. So, we are actually injecting these genes under the retina and telling the cells to produce more of this protein," Suan said.Suan said clinical trials are still in the early stages but that early detection is key, which makes eye exams important. Suan said research is crucial when it comes to a quality-of-life issue like vision."Do you want your grandmother and your loved one, grandfather, to actually be able to continue to drive to maintain independence? That really is the most important for them," Suan said.

A Baltimore doctor performed the first surgery in Maryland as part of a nationwide clinical trial to slow the progression of what's known as dry macular degeneration.

Dr. Eric Suan, retinal surgeon at the Retina Care Center, said degeneration causes vision loss once affected. And it can be a little or a lot, depending on how much tissue has been lost due to age-related dry macular degeneration.

"The typical patient who comes to me has a loss of central vision," Suan said. "They just can't read, they're having difficulty driving, and so this is a huge impairment for patients."

Because the tissue can't be regenerated, the goal is to slow progression.

Suan is part of a nationwide Food and Drug Administration-approved investigative clinical trial that involves a one-time surgery using a patient's own cells to create more of a certain protein thought to be low in patients with this eye disease.

"This is a very novel technique. It's gene therapy. So, we are actually injecting these genes under the retina and telling the cells to produce more of this protein," Suan said.

Suan said clinical trials are still in the early stages but that early detection is key, which makes eye exams important. Suan said research is crucial when it comes to a quality-of-life issue like vision.

"Do you want your grandmother and your loved one, grandfather, to actually be able to continue to drive to maintain independence? That really is the most important for them," Suan said.

Read the original post:
Surgeon taking part in gene therapy trial to slow vision loss - KCRA Sacramento

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Ambys Medicines, a company pioneering cell-replacement therapies for patients with liver disease, today announced the formation of its clinical and scientific advisory boards comprising leading clinical experts in liver disease and hepatocyte transplantation, and world-class scientists pioneering cell and gene technologies.

The clinical advisory board provides guidance on advancing Ambyss lead program, AMI-918, through the clinic and provides critical input on clinical trial design and patient selection. The scientific advisory board will provide a cross-discipline perspective on applying cutting-edge technology to Ambyss cell therapy platform to progress its discovery pipeline of next generation cell therapies.

Were honored to work with such an esteemed and diverse group of experts in liver disease, hepatocyte transplantation, and cell and gene therapies whose collective experience will be highly valuable as we finalize our clinical development strategy for AMI-918 and progress our genetically engineered hepatocyte follow-on programs, said Ronald Park, M.D., Chief Executive Officer of Ambys Medicines. Were grateful for the engagement and support from our advisors as we work to bring first-in-class hepatocyte replacement therapies to liver failure patients who currently lack treatment options.

Each of our advisors brings incredible knowledge and expertise in their respective fields that will be instrumental to Ambys as we continue to broaden our pipeline and move closer to becoming a clinical-stage company, said Markus Grompe, M.D., Founder and Chief Scientific Officer of Ambys Medicines. Were excited to partner together to realize the potential of our novel replacement cell therapy platform in restoring lost hepatic function to patients with acute or chronic liver failure and genetic liver diseases.

Clinical Advisory Board

Scientific Advisory Board

About Ambys MedicinesAmbys Medicines is focused on pioneering cell replacement therapies for patients with liver failure. Ambyss proprietary platform enables the company to be the first and only company able to develop and manufacture functional human hepatocytes at scale. Our scientific approach has the potential to fundamentally transform the treatment paradigm for patients with acute and chronic liver failure and genetic diseases of the liver. Our lead program, AMI-918, is a hepatocyte replacement cell therapy in development to restore lost hepatic function. Beyond AMI-918, we are building a pipeline of next-generation modified hepatocytes that will rapidly expand the range of treatable patient populations. Learn more at ambys.com and follow us on Twitter, LinkedIn and Instagram.

Read the rest here:
Ambys Medicines Announces Formation of Clinical and Scientific Advisory Boards with Leading Liver Disease and Cell and Gene Therapy Experts - Business...

New York, June 27, 2022 (GLOBE NEWSWIRE) -- The global animal stem cell therapy market recorded sales of around US$ 249.9 Mn in 2021 and the market is predicted to experience healthy growth over the years ahead at a CAGR of 5.2% (2022 to 2032).

Animal stem cell therapies are treatments for disorders such as arthritis, soft tissue injuries, traumatic fractures, tendonitis, inflammatory bowel disease, and others that are given to animals. Hematopoietic stem cells and mesenchymal stem cells are used in these treatment procedures.

As the focus on giving animals a better quality of life grows, so does the adoption of such therapies across the world. The market for animal stem cell therapy is predicted to continuously increase owing to the growing need for higher levels of veterinary care.

Request for sample copy of report: https://www.persistencemarketresearch.com/samples/14941

Regulatory bodies are also encouraging veterinary regenerative medicine research & development.

The European Commission has approved a few stem cell-based products within the region.

The animal stem cell therapy market is moderately fragmented with a few key market players and presents numerous opportunities for new market entrants to create a foothold in the industry. Increased pet adoption rate can be a beneficial factor for manufacturers of stem cells to expand into emerging markets.

Know the methodology of report: https://www.persistencemarketresearch.com/methodology/14941

Key Takeaways from Market Study

Get customized report as per requirement: https://www.persistencemarketresearch.com/request-customization/14941

With rising disposable incomes and higher adoption rate of pet animals, the global market for animal stem cell therapy is set to gain traction over the coming years, says an analyst of Persistence Market Research.

Market Competition

Key animal stem cell therapy providers are investing in innovating veterinary regenerative medicines and they are also coming up with new product launches for the well-being of animals.

Along with product innovation, market players are also aiming for various collaborations to strengthen their R&D in the field of animal stem cell therapy.

Get full access of report: https://www.persistencemarketresearch.com/checkout/14941

What Does the Report Cover?

Persistence Market Research offers a unique perspective and actionable insights on the animal stem cell therapy market in its latest study, presenting historical demand assessment of 2012 2021 and projections for 2022 2032.

The research study is based on product type (hemopoietic stem cells and mesenchymal stem cells), source (allogeneic and autologous), indication (osteoarthritis, soft tissue injuries, traumatic fractures, tendonitis, inflammatory bowel disease, and others), species (canine, feline, and equine), and end user (veterinary hospitals, veterinary clinics, and veterinary research institutes), across seven key regions of the world.

Other Trending Reports:

About Persistence Market Research:

Business intelligence is the foundation of every business model employed by Persistence Market Research. Multi-dimensional sources are being put to work, which include big data, customer experience analytics, and real-time data collection. Thus, working on micros by Persistence Market Research helps companies overcome their macro business challenges.

Persistence Market Research is always way ahead of its time. In other words, it tables market solutions by stepping into the companies/clients shoes much before they themselves have a sneak pick into the market. The pro-active approach followed by experts at Persistence Market Research helps companies/clients lay their hands on techno-commercial insights beforehand, so that the subsequent course of action could be simplified on their part.

Contact

Rajendra Singh

Persistence Market Research U.S. Sales Office:305 Broadway, 7th FloorNew York City, NY 10007+1-646-568-7751United StatesUSA - Canada Toll-Free: 800-961-0353Email: sales@persistencemarketresearch.com

Visit Our Website:https://www.persistencemarketresearch.com

Read the original post:
Animal Stem Cell Therapy Market projected CAGR of 5.2% for the next ten years (2022-2032) Persistence Market Research - GlobeNewswire