Monthly Archives: December 2020

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The challenges of gene and cell therapists can be divided into three broad categories based on disease, development of therapy, and funding.

Challenges based on the disease characteristics: Disease symptoms of most genetic diseases, such as Fabrys, hemophilia, cystic fibrosis, muscular dystrophy, Huntingtons, and lysosomal storage diseases are caused by distinct mutations in single genes. Other diseases with a hereditary predisposition, such as Parkinsons disease, Alzheimers disease, cancer, and dystonia may be caused by variations/mutations in several different genes combined with environmental causes. Note that there are many susceptible genes and additional mutations yet to be discovered. Gene replacement therapy for single gene defects is the most conceptually straightforward. However, even then the gene therapy agent may not equally reduce symptoms in patients with the same disease caused by different mutations, and even the samemutationcan be associated with different degrees of disease severity. Gene therapists often screen their patients to determine the type of mutation causing the disease before enrollment into a clinical trial.

The mutated gene may cause symptoms in more than one cell type. Cystic fibrosis, for example, affects lung cells and the digestive tract, so the gene therapy agent may need to replace the defective gene or compensate for its consequences in more than one tissue for maximum benefit. Alternatively, cell therapy can utilizestem cellswith the potential to mature into the multiple cell types to replace defective cells in different tissues.

In diseases like muscular dystrophy, for example, the high number of cells in muscles throughout the body that need to be corrected in order to substantially improve the symptoms makes delivery of genes and cells a challenging problem.

Some diseases, like cancer, are caused by mutations in multiple genes. Although different types of cancers have some common mutations, every tumor from a single type of cancer does not contain the same mutations. This phenomenon complicates the choice of a single gene therapy tactic and has led to the use of combination therapies and cell elimination strategies. For more information on gene and cell therapy strategies to treat cancer, please refer to the Cancer and Immunotherapy summary in the Disease Treatment section.

Disease models in animals do not completely mimic the human diseases and viralvectorsmay infect various species differently. The testing of vectors in animal models often resemble the responses obtained in humans, but the larger size of humans in comparison to rodents presents additional challenges in the efficiency of delivery and penetration of tissue.Gene therapy, cell therapy, and oligonucleotide-based therapy agents are often tested in larger animal models, including rabbit, dog, pig and nonhuman primate models. Testing human cell therapy in animal models is complicated by immune rejections. Furthermore, humans are a very heterogeneous population. Their immune responses to the vectors, altered cells, or cell therapy products may differ or be similar to results obtained in animal models.

Challenges in the development of gene and cell therapy agents: Scientific challenges include the development of gene therapy agents that express the gene in the relevant tissue at the appropriate level for the desired duration of time. There are a lot of issues in that once sentence, and while these issues are easy to state, each one requires extensive research to identify the best means of delivery, how to control sufficient levels or numbers of cells, and factors that influence duration of gene expression or cell survival. After the delivery modalities are determined, identification and engineering of a promoter and control elements (on/off switch and dimmer switch) that will produce the appropriate amount of protein in the target cell can be combined with the relevant gene. This gene cassette is engineered into a vector or introduced into thegenomeof a cell and the properties of the delivery vehicle are tested in different types of cells in tissue culture. Sometimes things go as planned and then studies can be moved onto examination in animal models. In most cases, the gene/cell therapy agent may need to be improved further by adding new control elements to obtain the desired responses in cells and animal models.

Furthermore, the response of the immune system needs to be considered based on the type of gene or cell therapy being undertaken. For example, in gene or cell therapy for cancer, one aim is to selectively boost the existing immune response to cancer cells. In contrast, to treat genetic diseases like hemophilia and cystic fibrosis the goal is for the therapeutic protein to be accepted as an addition to the patients immune system.

If the new gene is inserted into the patients cellularDNA, the intrinsic sequences surrounding the new gene can affect its expression and vice versa. Scientists are now examining short DNA segments that may insulate the new gene from surrounding control elements. Theoretically, these insulator sequences would also reduce the effect of vector control signals in the gene cassette on adjacent cellular genes. Studies are also focusing on means to target insertion of the new gene into safe areas of the genome, to avoid influence on surrounding genes and to reduce the risk of insertional mutagenesis.

Challenges of cell therapy include the harvesting of the appropriate cell populations and expansion or isolation of sufficient cells for one or multiple patients. Cell harvesting may require specific media to maintain the stem cells ability toself-renew and mature into the appropriate cells. Ideally extra cells are taken from the individual receiving therapy. Those additional cells can expand in culture and can be induced to becomepluripotent stem cells(iPS), thus allowing them to assume a wide variety of cell types and avoiding immune rejection by the patient. The long term benefit of stem cell administration requires that the cells be introduced into the correct target tissue and become established functioning cells within the tissue. Several approaches are being investigated to increase the number of stem cells that become established in the relevant tissue.

Another challenge is developing methods that allow manipulation of the stem cells outside the body while maintaining the ability of those cells to produce more cells that mature into the desired specialized cell type. They need to provide the correct number of specialized cells and maintain their normal control of growth and cell division, otherwise there is the risk that these new cells may grow into tumors.

Challenges in funding: In most fields, funding for basic or applied research for gene and cell therapy is available through the National Institutes of Health (NIH) and private foundations. These are usually sufficient to cover the preclinical studies that suggest a potential benefit from a particular gene and cell therapy. Moving into clinical trials remains a huge challenge as it requires additional funding for manufacturing of clinical grade reagents, formal toxicology studies in animals, preparation of extensive regulatory documents, and costs of clinical trials.Biotechnology companies and the NIH are trying to meet the demand for this large expenditure, but many promising therapies are slowed down by lack of funding for this critical next phase.

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Gene & Cell Therapy FAQs | ASGCT - American Society of ...

What are cells and genes?

Cells are the basic building blocks of all living things, and genes can be found deep within cells. Genes are small sections of DNA that carry genetic information and instructions for making proteins, which help build and maintain the body1.

Every person has around 20,000 genes and two copies of each of their genesone from each parent. Small variations in genes result in differences in peoples appearance and, potentially, health1.

Genetic diseases happen when a critical piece or whole section of DNA is substituted, deleted or duplicated2. These changes are called genetic mutations3. Some serious genetic diseases caused by genetic mutations can be passed to future generations4.

Cell therapy and gene therapy are overlapping fields of biomedical research and treatment6. Both therapies aim to treat, prevent, or potentially cure diseases, and both approaches have the potential to alleviate the underlying cause of genetic diseases and acquired diseases6. But, cell and gene therapies work differently.

Cell therapy aims to treat diseases by restoring or altering certain sets of cells orby using cells to carry a therapy through the body5. With cell therapy, cells are cultivated or modified outside the body before being injected into the patient. The cells may originate from the patient (autologous cells) or a donor (allogeneic cells)6.

Gene therapy aims to treat diseases by replacing, inactivating or introducing genes into cells either inside the body (in vivo) or outside of the body (ex vivo)6.

Some therapies are considered both cell and gene therapies. These therapies work by altering genes in specific types of cells and inserting them into the body.

Learn more about how we use cell and gene therapies and why they are important

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What is cell and gene therapy | Novartis

By Pat Anson, PNN Editor

Scientists have developed an experimental drug that can lure stem cells to damaged tissues and help them heal -- a discovery being touted as a major advancement in the field of regenerative medicine.

The findings, recently published in the Proceedings of the National Academy of Sciences (PNAS), could improve the effectiveness of stem cell therapy in treating spinal cord injuries, stroke, amyotrophic lateral sclerosis(ALS), Parkinsons disease and other neurodegenerative disorders. It could also expand the use of stem cells to treat conditions such as heart disease and arthritis.

The ability to instruct a stem cell where to go in the body or to a particular region of a given organ is the Holy Grail for regenerative medicine, said lead authorEvan Snyder, MD, director of theCenter for Stem Cells & Regenerative Medicineat Sanford Burnham Prebys Medical Discovery Institute in La Jolla, CA. Now, for the first time ever, we can direct a stem cell to a desired location and focus its therapeutic impact.

Over a decade ago, Snyder and his colleagues discovered that stem cells are drawn to inflammation -- a biological fire alarm that signals tissue damage has occurred. However, using inflammation as a therapeutic lure for stem cells wasnt advisable because they could further inflame diseased or damaged organs, joints and other tissue.

To get around that problem, scientists modified CXCL12 -- an inflammatory molecule that Snyders team discovered could guide stem cells to sites in need of repair to create a drug called SDV1a. The new drug works by enhancing stem cell binding, while minimizing inflammatory signals.

Since inflammation can be dangerous, we modified CXCL12 by stripping away the risky bit and maximizing the good bit, Snyder explained. Now we have a drug that draws stem cells to a region of pathology, but without creating or worsening unwanted inflammation.

To demonstrate its effectiveness, Snyders team injected SDV1a and human neural stem cells into the brains of mice with a neurodegenerative disease called Sandhoff disease. The experiment showed that the drug helped stem cells migrate and perform healing functions, which included extending lifespan, delaying symptom onset, and preserving motor function for much longer than mice that didnt receive the drug. Importantly, the stem cells also did not worsen the inflammation.

Researchers are now testing SDV1as ability to improve stem cell therapy in a mouse model of ALS, also known as Lou Gehrigs disease, which is caused by a progressive loss of motor neurons in the brain. Previous studies conducted by Snyders team found that broadening the spread of neural stem cells helps more motor neurons survive so they are hopeful that SDV1a will improve the effectiveness of neuroprotective stem cells and help slow the onset and progression of ALS.

We are optimistic that this drugs mechanism of action may potentially benefit a variety of neurodegenerative disorders, as well as non-neurological conditions such as heart disease, arthritis and even brain cancer, says Snyder. Interestingly, because CXCL12 and its receptor are implicated in the cytokine storm that characterizes severe COVID-19, some of our insights into how to selectively inhibit inflammation without suppressing other normal processes may be useful in that arena as well.

Snyders research is supported by the National Institutes of Health, U.S. Department of Defense, National Tay-Sachs & Allied Disease Foundation, Childrens Neurobiological Solutions Foundation, and the California Institute for Regenerative Medicine (CIRM).

Thanks to decades of investment in stem cell science, we are making tremendous progress in our understanding of how these cells work and how they can be harnessed to help reverse injury or disease, says Maria Millan, MD, president and CEO of CIRM. This drug could help speed the development of stem cell treatments for spinal cord injury, Alzheimers, heart disease and many other conditions for which no effective treatment exists.

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New Drug Could Improve Effectiveness of Stem Cell Therapy - Pain News Network

A study published in OncoTargets and Therapy found that CD19 chimeric antigen receptor (CAR) T-cells derived from patients who relapsed after receiving allogeneic hematopoietic cell transplantation (alloHCT) with a low level of donor chimerism were effective for salvage therapy. These T-cells could be restored to complete donor chimerism after 12 days of in vitro culture.

The study included nine patients with B-cell acute lymphocytic leukemia (ALL) who had low donor chimerism levels and relapsed after alloHCT. The first three patients received CD19 CAR T-cell therapy using cells derived from autologous peripheral blood mononuclear cells (PBMCs), comprising a mixture of patient and original donor cells, as their donors could not provide PBMCs.

Samples from the subsequent six patients were investigated in vitro only. The changes in the degree of donor chimerism, function of the CD19 CAR T-cells, and T-cells in all nine patients were analyzed in vitro.

CAR T-cells and T-cells in all nine patients showed complete donor chimerism restoration after a 12-day culture period in vitro. These CD19 CAR T-cells demonstrated strong cytotoxicity toward Nalm 6 cells in vitro except in two patients. In the latter patients, the absolute numbers of all subsets especially the CD8+ T-cell absolute numbers in peripheral blood were very low.

Two patients showed relatively short durations from transplant to recurrence and received chemotherapy after relapse. Among patients receiving CD19 CAR T-cell therapy, the most commonly observed adverse event was grade 1/2 cytokine release syndrome.

No patients had acute graft-versus-host disease during treatment. Among the first three treated patients, the first two achieved complete response with complete restoration of donor chimerism. Patient three, who received the same CD19 CAR-T cell therapy as the first two, did not respond to this therapy.

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Study Observes Changes in Donor Chimerism in Patients with ALL Receiving CAR T-Cell Therapy - DocWire News

New York, Nov. 27, 2020 (GLOBE NEWSWIRE) -- Reportlinker.com announces the release of the report "Asia Pacific Cell Therapy Instruments Market Forecast to 2027 - Covid-19 Impact and Regional Analysis - By Product ; Cell Type ; Process ; End User, and Country" - https://www.reportlinker.com/p05989496/?utm_source=GNW However, the low success rate of cell therapies and the high cost of cell-based research is expected to restrain the market growth during the forecast period.

Cell therapy typically involves the administration of somatic cell preparations by injecting or grafting it into the patients body for the treatment of diseases or traumatic damages.The procedure is used to cure diabetes, neurological disorders, related injuries, several cancer types, bones and joints, and genetic disorders.

Continuous research and development activities have led to unique cell therapeutic instruments for the improvement of immune system and efficient treatment of genetic disorders. Various market players provide several consumables such as reagent kits and enzymes as well as devices, equipment, and software to perform various cell therapy processes.

The use of instruments is essential for handling cell therapies such as NSC, PSC, MSC, T cells, and HSC.These cell therapy products are derived from animals or human cells and thus need to be protected from contamination.

The instruments used in cell therapies help provide protection against contamination and allow scaling up of transplantation. Companies such as Hitachi Chemical Advanced Therapeutics Solutions Corning Incorporated; Thermo Fisher Scientific Inc., MiltenyiBiotec, LLC; Invetech; and Cytiva (General Electric Company) have introduced various equipment and consumables for the cell therapy procedures.

The global COVID-19 emergency has been particularly affecting the supply chain worldwide.The supply chain disruptions, along with the enormous demand for effective therapies for the treatment of COVID-19, have put the healthcare research industry in a crucial situation in the Asia Pacific region.

However, many medical companies have realized the importance of cell therapy in the treatment of COVID 19, which would raise its demand in the coming years.

The Asia Pacific cell therapy instruments market, by product, is segmented into consumables, software, equipment, and systems.The consumables segment held the largest share of the market in 2019 and is expected to register the highest CAGR during the forecast period.

On the basis of cell type, the cell therapy instruments market is segmented into animal cells and human cells. The human cells segment held a larger share of the market in 2019 and is estimated to register a higher CAGR during the forecast period.

On the basis of process, the Asia Pacific cell therapy instruments market is segmented into cell processing; cell preservation, distribution, and handling; and process monitoring and quality control.The cell processing segment held the largest share of the market in 2019 and is estimated to register the highest CAGR during the forecast period.

The Asia Pacific cell therapy instruments market, based on end user, is segmented into life science research companies, research institutes, and other end users. The life science research companies segment accounted for the largest share of the market in 2019 and is anticipated to register the highest CAGR during the forecast period.

A few of the major primary and secondary sources associated with the Asia Pacific cell therapy instruments market are National Center for Biotechnology Information (NCBI); World Health Organization (WHO); Medical Research Future Fund (MRFF); Asia-Pacific Economic Corporation (APEC); and Global Institute of Stem Cell Therapy and Research (GIOSTAR).Read the full report: https://www.reportlinker.com/p05989496/?utm_source=GNW

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Asia Pacific Cell Therapy Instruments Market Forecast to 2027 - Covid-19 Impact and Regional Analysis - By Product ; Cell Type ; Process ; End User,...

I would never have thought that a mutant flu virus could create that big panic in people at each and every corner of the world. In fact, the past few days in quarantine have really made me realize where exactly the humanity is heading.

For majority of us, our lives are on a temporary hold while the world is dealing with the global pandemic of COVID-19. In fact, past three months have served as something of an inflection for many countries and as the number of infected patients have surpassed one million, there is already a sense of worry looming across the industries.

Since Coronavirus is a newly identified pathogen, there is no known pre-existing immunity observed in humans and therefore everyone is assumed to be susceptible. While researchers all across the globe are putting efforts to develop immediate treatment, there are speculations, based on credible evidence, that infected patients treated with Stem Cell Therapy are more likely to survive the disease. Specifically, mesenchymal stem cells can be effectively used to improve patients resistance to the SARS-CoV-2 virus induced pneumonia as these cells have the potential to repair damaged tissues in the patients respiratory system leading to speedy recovery.

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Recent Trends

Recent trends across several social medial platform highlight the immense efforts put by several stakeholders validating the therapeutic potential of stem cells. Specifically, Id like to appreciate the efforts of Azidus Brasil, CAR-T (Shanghai) Biotechnology, Janssen Research & Development, Mesoblast, Millennium Pharmaceutical, Pfizer, Stem Cell Arabia, Tianhe Stem Cell Biotechnologies and Tuohua Biological Technology that have progressed significantly in-terms of development of stem cell-based therapies to defeat this global pandemic of COVID-19 infection.

Looking at the efforts made by different pharmaceutical companies, I felt assured and hopeful as many patients have successfully recovered, while others are at the verge of getting discharged by using these novel classes of regenerative medicines.

What are the Key Hubs for Stem Cell Research?

With the virus strengthening its foothold in several countries across the globe, the threat of pandemic has become real and the question is are we ready?

As per the recent study published by Roots Analysis, the efforts for development of stem cell therapies have been undertaken by players all across the globe, majority of the developers (45%) are based in Asia-Pacific regions; China, South Korea and Japan; followed by developed countries, such as the US, Germany, Belgium, Spain and the UK.

Roots Analysis, in its recent report, has captured the clinical and research landscape of stem cell therapy-based treatment. To know further, check out the report here

Expert Opinion

In fact. several industry stakeholders are quite optimistic about the future market potential of stem cell-based therapies.

Bottom Line: Stay Cautious, Stay Hopeful!!

In the midst of the anxiety, worry, and uncertainty surrounding the COVID-19 pandemic, each day seems to bring news thats worse than the day before. However, remember this is not for the first time any pandemic outbreak has taken place in the history of mankind. About 200,000 (~20%) patients have already been recovered from this disease. The sky is not falling and for sure, life would return to normal. Stay cautious, stay hopeful.

For further information, check out the report here

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About Roots Analysis

Roots Analysis is one of the fastest growing market research companies, sharing fresh and independent perspectives in the bio-pharmaceutical industry. The in-depth research, analysis and insights are driven by an experienced leadership team which has gained many years of significant experience in this sector. If youd like help with your growing business needs, get in touch at [emailprotected]

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Stem Cell Therapy: A Ray of Hope to End Global Pandemic of COVID-19 | Roots Analysis - Khabar South Asia

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

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

For More Detail Insights, Download Sample Copy of the Report at: https://www.researchdive.com/download-sample/1819

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

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

RESEARCH METHODOLOGY

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

Connect with Our Analyst to Contextualize Our Insights for Your Business:https://www.researchdive.com/connect-to-analyst/1819

KEY MARKET BENEFITS

KEY MARKET SEGMENTS

The global Cell Therapy Technologies Market market is segmented on the basis of the following:

Global Cell Therapy Technologies Market Market By Product Type:

Global Cell Therapy Technologies Market Market By Applications:

Global Cell Therapy Technologies Market Market By Regions:

Top Leading key players stated in Global Cell Therapy Technologies Market Market report are:

Beckman Coulter, Becton, Dickinson and Company, GE Healthcare, Lonza, Merck KGaA, Miltenyi Biotec, STEMCELL Technologies, Inc., Terumo BCT, Thermo Fisher Scientific

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

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Mr. Abhishek PaliwalResearch Dive30 Wall St. 8th Floor, New YorkNY 10005 (P)+ 91 (788) 802-9103 (India)+1 (917) 444-1262 (US) TollFree : +1 -888-961-4454Email:support@researchdive.comLinkedIn:https://www.linkedin.com/company/research-diveTwitter:https://twitter.com/ResearchDiveFacebook:https://www.facebook.com/Research-DiveBlog:https://www.researchdive.com/blogFollow us on:https://covid-19-market-insights.blogspot.com

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Widening: Turn your losses to profit with Exclusive Insight on Cell Therapy Technologies Market - The Courier