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Category Archives: Cell Medicine

Cell-Based Regenerative Medicine Market Size to Witness Rapid Growth at a CAGR of 15% by 2032 | insightSLICE – EIN News

Posted: May 9, 2023 at 12:08 am

Cell-Based Regenerative Medicine Market Size to Witness Rapid Growth at a CAGR of 15% by 2032 | insightSLICE  EIN News

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Cell-Based Regenerative Medicine Market Size to Witness Rapid Growth at a CAGR of 15% by 2032 | insightSLICE - EIN News

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Indian Pharma Congress: Gene-cell therapy, preventive medicine future of health care, says expert – Economic Times

Posted: January 21, 2023 at 12:24 am

Indian Pharma Congress: Gene-cell therapy, preventive medicine future of health care, says expert  Economic Times

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Indian Pharma Congress: Gene-cell therapy, preventive medicine future of health care, says expert - Economic Times

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Cell culture – Wikipedia

Posted: December 18, 2022 at 12:56 am

Process by which cells are grown under controlled conditions

Cell culture or tissue culture is the process by which cells are grown under controlled conditions, generally outside of their natural environment. The term "tissue culture" was coined by American pathologist Montrose Thomas Burrows.[1] This technique is also called micropropagation. After the cells of interest have been isolated from living tissue, they can subsequently be maintained under carefully controlled conditions the need to be kept at body temperature (37C) in an incubator.[2] These conditions vary for each cell type, but generally consist of a suitable vessel with a substrate or rich medium that supplies the essential nutrients (amino acids, carbohydrates, vitamins, minerals), growth factors, hormones, and gases (CO2, O2), and regulates the physio-chemical environment (pH buffer, osmotic pressure, temperature). Most cells require a surface or an artificial substrate to form an adherent culture as a monolayer (one single-cell thick), whereas others can be grown free floating in a medium as a suspension culture.[3] This is typically facilitated via use of a liquid, semi-solid, or solid growth medium, such as broth or agar. Tissue culture commonly refers to the culture of animal cells and tissues, with the more specific term plant tissue culture being used for plants. The lifespan of most cells is genetically determined, but some cell culturing cells have been transformed into immortal cells which will reproduce indefinitely if the optimal conditions are provided.

In practice, the term "cell culture" now refers to the culturing of cells derived from multicellular eukaryotes, especially animal cells, in contrast with other types of culture that also grow cells, such as plant tissue culture, fungal culture, and microbiological culture (of microbes). The historical development and methods of cell culture are closely interrelated to those of tissue culture and organ culture. Viral culture is also related, with cells as hosts for the viruses.

The laboratory technique of maintaining live cell lines (a population of cells descended from a single cell and containing the same genetic makeup) separated from their original tissue source became more robust in the middle 20th century.[4][5]

The 19th-century English physiologist Sydney Ringer developed salt solutions containing the chlorides of sodium, potassium, calcium and magnesium suitable for maintaining the beating of an isolated animal heart outside the body.[6] In 1885 Wilhelm Roux removed a section of the medullary plate of an embryonic chicken and maintained it in a warm saline solution for several days, establishing the basic principle of tissue culture. In 1907 the zoologist Ross Granville Harrison demonstrated the growth of frog embryonic cells that would give rise to nerve cells in a medium of clotted lymph. In 1913, E. Steinhardt, C. Israeli, and R. A. Lambert grew vaccinia virus in fragments of guinea pig corneal tissue.[7] In 1996, the first use of regenerative tissue was used to replace a small length of urethra, which led to the understanding that the technique of obtaining samples of tissue, growing it outside the body without a scaffold, and reapplying it, can be used for only small distances of less than 1cm.[8][9][10] Ross Granville Harrison, working at Johns Hopkins Medical School and then at Yale University, published results of his experiments from 1907 to 1910, establishing the methodology of tissue culture.[11]

Gottlieb Haberlandt first pointed out the possibilities of the culture of isolated tissues, plant tissue culture.[12] He suggested that the potentialities of individual cells via tissue culture as well as that the reciprocal influences of tissues on one another could be determined by this method. Since Haberlandt's original assertions, methods for tissue and cell culture have been realized, leading to significant discoveries in biology and medicine. His original idea, presented in 1902, was called totipotentiality: Theoretically all plant cells are able to give rise to a complete plant.[13][14][15]

Cell culture techniques were advanced significantly in the 1940s and 1950s to support research in virology. Growing viruses in cell cultures allowed preparation of purified viruses for the manufacture of vaccines. The injectable polio vaccine developed by Jonas Salk was one of the first products mass-produced using cell culture techniques. This vaccine was made possible by the cell culture research of John Franklin Enders, Thomas Huckle Weller, and Frederick Chapman Robbins, who were awarded a Nobel Prize for their discovery of a method of growing the virus in monkey kidney cell cultures. Cell culture has contributed to the development of vaccines for many diseases.[2]

In modern usage, "tissue culture" generally refers to the growth of cells from a tissue from a multicellular organism in vitro. These cells may be cells isolated from a donor organism (primary cells) or an immortalised cell line. The cells are bathed in a culture medium, which contains essential nutrients and energy sources necessary for the cells' survival.[16] Thus, in its broader sense, "tissue culture" is often used interchangeably with "cell culture". On the other hand, the strict meaning of "tissue culture" refers to the culturing of tissue pieces, i.e. explant culture.

Tissue culture is an important tool for the study of the biology of cells from multicellular organisms. It provides an in vitro model of the tissue in a well defined environment which can be easily manipulated and analysed. In animal tissue culture, cells may be grown as two-dimensional monolayers (conventional culture) or within fibrous scaffolds or gels to attain more naturalistic three-dimensional tissue-like structures (3D culture). Eric Simon, in a 1988 NIH SBIR grant report, showed that electrospinning could be used to produced nano- and submicron-scale polymeric fibrous scaffolds specifically intended for use as in vitro cell and tissue substrates. This early use of electrospun fibrous lattices for cell culture and tissue engineering showed that various cell types would adhere to and proliferate upon polycarbonate fibers. It was noted that as opposed to the flattened morphology typically seen in 2D culture, cells grown on the electrospun fibers exhibited a more rounded 3-dimensional morphology generally observed of tissues in vivo.[17]

Plant tissue culture in particular is concerned with the growing of entire plants from small pieces of plant tissue, cultured in medium.[18]

Cells can be isolated from tissues for ex vivo culture in several ways. Cells can be easily purified from blood; however, only the white cells are capable of growth in culture. Cells can be isolated from solid tissues by digesting the extracellular matrix using enzymes such as collagenase, trypsin, or pronase, before agitating the tissue to release the cells into suspension.[19][20] Alternatively, pieces of tissue can be placed in growth media, and the cells that grow out are available for culture. This method is known as explant culture.

Cells that are cultured directly from a subject are known as primary cells. With the exception of some derived from tumors, most primary cell cultures have limited lifespan.

An established or immortalized cell line has acquired the ability to proliferate indefinitely either through random mutation or deliberate modification, such as artificial expression of the telomerase gene.Numerous cell lines are well established as representative of particular cell types.

For the majority of isolated primary cells, they undergo the process of senescence and stop dividing after a certain number of population doublings while generally retaining their viability (described as the Hayflick limit).

Aside from temperature and gas mixture, the most commonly varied factor in culture systems is the cell growth medium. Recipes for growth media can vary in pH, glucose concentration, growth factors, and the presence of other nutrients. The growth factors used to supplement media are often derived from the serum of animal blood, such as fetal bovine serum (FBS), bovine calf serum, equine serum, and porcine serum. One complication of these blood-derived ingredients is the potential for contamination of the culture with viruses or prions, particularly in medical biotechnology applications. Current practice is to minimize or eliminate the use of these ingredients wherever possible and use human platelet lysate (hPL).[21] This eliminates the worry of cross-species contamination when using FBS with human cells. hPL has emerged as a safe and reliable alternative as a direct replacement for FBS or other animal serum. In addition, chemically defined media can be used to eliminate any serum trace (human or animal), but this cannot always be accomplished with different cell types. Alternative strategies involve sourcing the animal blood from countries with minimum BSE/TSE risk, such as The United States, Australia and New Zealand,[22] and using purified nutrient concentrates derived from serum in place of whole animal serum for cell culture.[23]

Plating density (number of cells per volume of culture medium) plays a critical role for some cell types. For example, a lower plating density makes granulosa cells exhibit estrogen production, while a higher plating density makes them appear as progesterone-producing theca lutein cells.[24]

Cells can be grown either in suspension or adherent cultures.[25] Some cells naturally live in suspension, without being attached to a surface, such as cells that exist in the bloodstream. There are also cell lines that have been modified to be able to survive in suspension cultures so they can be grown to a higher density than adherent conditions would allow. Adherent cells require a surface, such as tissue culture plastic or microcarrier, which may be coated with extracellular matrix (such as collagen and laminin) components to increase adhesion properties and provide other signals needed for growth and differentiation. Most cells derived from solid tissues are adherent. Another type of adherent culture is organotypic culture, which involves growing cells in a three-dimensional (3-D) environment as opposed to two-dimensional culture dishes. This 3D culture system is biochemically and physiologically more similar to in vivo tissue, but is technically challenging to maintain because of many factors (e.g. diffusion).[26]

There are different kinds of cell culture media which being used routinely in life science including the following:

Cell line cross-contamination can be a problem for scientists working with cultured cells.[27] Studies suggest anywhere from 15 to 20% of the time, cells used in experiments have been misidentified or contaminated with another cell line.[28][29][30] Problems with cell line cross-contamination have even been detected in lines from the NCI-60 panel, which are used routinely for drug-screening studies.[31][32] Major cell line repositories, including the American Type Culture Collection (ATCC), the European Collection of Cell Cultures (ECACC) and the German Collection of Microorganisms and Cell Cultures (DSMZ), have received cell line submissions from researchers that were misidentified by them.[31][33] Such contamination poses a problem for the quality of research produced using cell culture lines, and the major repositories are now authenticating all cell line submissions.[34] ATCC uses short tandem repeat (STR) DNA fingerprinting to authenticate its cell lines.[35]

To address this problem of cell line cross-contamination, researchers are encouraged to authenticate their cell lines at an early passage to establish the identity of the cell line. Authentication should be repeated before freezing cell line stocks, every two months during active culturing and before any publication of research data generated using the cell lines. Many methods are used to identify cell lines, including isoenzyme analysis, human lymphocyte antigen (HLA) typing, chromosomal analysis, karyotyping, morphology and STR analysis.[35]

One significant cell-line cross contaminant is the immortal HeLa cell line. Hela contamination was first noted in the early 1960s in non-human culture in the USA. Intraspecies contamination was discovered in nineteen cell lines in the seventies. In 1974, five human cell lines from the Soviet Union were found to be Hela. A follow-up study analysing 50-odd cell lines indicated that half had Hela markers, but contaminant Hela had hybridised with the original cell lines. Hela cell contamination from air droplets has been reported. Hela was even unknowingly injected into human subjects by Jonas Salk in a 1978 vaccine trial.[36]

As cells generally continue to divide in culture, they generally grow to fill the available area or volume. This can generate several issues:

The choice of culture medium might affect the physiological relevance of findings from cell culture experiments due to the differences in the nutrient composition and concentrations.[38] A systematic bias in generated datasets was recently shown for CRISPR and RNAi gene silencing screens,[39] and for metabolic profiling of cancer cell lines.[38] Using a growth medium that better represents the physiological levels of nutrients can improve the physiological relevance of in vitro studies and recently such media types, as Plasmax[40] and Human Plasma Like Medium (HPLM),[41] were developed.

Among the common manipulations carried out on culture cells are media changes, passaging cells, and transfecting cells.These are generally performed using tissue culture methods that rely on aseptic technique. Aseptic technique aims to avoid contamination with bacteria, yeast, or other cell lines. Manipulations are typically carried out in a biosafety cabinet or laminar flow cabinet to exclude contaminating micro-organisms. Antibiotics (e.g. penicillin and streptomycin) and antifungals (e.g.amphotericin B and Antibiotic-Antimycotic solution) can also be added to the growth media.

As cells undergo metabolic processes, acid is produced and the pH decreases. Often, a pH indicator is added to the medium to measure nutrient depletion.

In the case of adherent cultures, the media can be removed directly by aspiration, and then is replaced. Media changes in non-adherent cultures involve centrifuging the culture and resuspending the cells in fresh media.

Passaging (also known as subculture or splitting cells) involves transferring a small number of cells into a new vessel. Cells can be cultured for a longer time if they are split regularly, as it avoids the senescence associated with prolonged high cell density. Suspension cultures are easily passaged with a small amount of culture containing a few cells diluted in a larger volume of fresh media. For adherent cultures, cells first need to be detached; this is commonly done with a mixture of trypsin-EDTA; however, other enzyme mixes are now available for this purpose. A small number of detached cells can then be used to seed a new culture. Some cell cultures, such as RAW cells are mechanically scraped from the surface of their vessel with rubber scrapers.

Another common method for manipulating cells involves the introduction of foreign DNA by transfection. This is often performed to cause cells to express a gene of interest. More recently, the transfection of RNAi constructs have been realized as a convenient mechanism for suppressing the expression of a particular gene/protein. DNA can also be inserted into cells using viruses, in methods referred to as transduction, infection or transformation. Viruses, as parasitic agents, are well suited to introducing DNA into cells, as this is a part of their normal course of reproduction.

Cell lines that originate with humans have been somewhat controversial in bioethics, as they may outlive their parent organism and later be used in the discovery of lucrative medical treatments. In the pioneering decision in this area, the Supreme Court of California held in Moore v. Regents of the University of California that human patients have no property rights in cell lines derived from organs removed with their consent.[42]

It is possible to fuse normal cells with an immortalised cell line. This method is used to produce monoclonal antibodies. In brief, lymphocytes isolated from the spleen (or possibly blood) of an immunised animal are combined with an immortal myeloma cell line (B cell lineage) to produce a hybridoma which has the antibody specificity of the primary lymphocyte and the immortality of the myeloma. Selective growth medium (HA or HAT) is used to select against unfused myeloma cells; primary lymphoctyes die quickly in culture and only the fused cells survive. These are screened for production of the required antibody, generally in pools to start with and then after single cloning.

A cell strain is derived either from a primary culture or a cell line by the selection or cloning of cells having specific properties or characteristics which must be defined. Cell strains are cells that have been adapted to culture but, unlike cell lines, have a finite division potential. Non-immortalized cells stop dividing after 40 to 60 population doublings[43] and, after this, they lose their ability to proliferate (a genetically determined event known as senescence).[44]

Mass culture of animal cell lines is fundamental to the manufacture of viral vaccines and other products of biotechnology. Culture of human stem cells is used to expand the number of cells and differentiate the cells into various somatic cell types for transplantation.[45] Stem cell culture is also used to harvest the molecules and exosomes that the stem cells release for the purposes of therapeutic development.[46]

Biological products produced by recombinant DNA (rDNA) technology in animal cell cultures include enzymes, synthetic hormones, immunobiologicals (monoclonal antibodies, interleukins, lymphokines), and anticancer agents. Although many simpler proteins can be produced using rDNA in bacterial cultures, more complex proteins that are glycosylated (carbohydrate-modified) currently must be made in animal cells. An important example of such a complex protein is the hormone erythropoietin. The cost of growing mammalian cell cultures is high, so research is underway to produce such complex proteins in insect cells or in higher plants, use of single embryonic cell and somatic embryos as a source for direct gene transfer via particle bombardment, transit gene expression and confocal microscopy observation is one of its applications. It also offers to confirm single cell origin of somatic embryos and the asymmetry of the first cell division, which starts the process.

Cell culture is also a key technique for cellular agriculture, which aims to provide both new products and new ways of producing existing agricultural products like milk, (cultured) meat, fragrances, and rhino horn from cells and microorganisms. It is therefore considered one means of achieving animal-free agriculture. It is also a central tool for teaching cell biology.[47]

Research in tissue engineering, stem cells and molecular biology primarily involves cultures of cells on flat plastic dishes. This technique is known as two-dimensional (2D) cell culture, and was first developed by Wilhelm Roux who, in 1885, removed a portion of the medullary plate of an embryonic chicken and maintained it in warm saline for several days on a flat glass plate. From the advance of polymer technology arose today's standard plastic dish for 2D cell culture, commonly known as the Petri dish. Julius Richard Petri, a German bacteriologist, is generally credited with this invention while working as an assistant to Robert Koch. Various researchers today also utilize culturing laboratory flasks, conicals, and even disposable bags like those used in single-use bioreactors.

Aside from Petri dishes, scientists have long been growing cells within biologically derived matrices such as collagen or fibrin, and more recently, on synthetic hydrogels such as polyacrylamide or PEG. They do this in order to elicit phenotypes that are not expressed on conventionally rigid substrates. There is growing interest in controlling matrix stiffness,[48] a concept that has led to discoveries in fields such as:

Cell culture in three dimensions has been touted as "Biology's New Dimension".[63] At present, the practice of cell culture remains based on varying combinations of single or multiple cell structures in 2D.[64] Currently, there is an increase in use of 3D cell cultures in research areas including drug discovery, cancer biology, regenerative medicine, nanomaterials assessment and basic life science research.[65][66][67] 3D cell cultures can be grown using a scaffold or matrix, or in a scaffold-free manner. Scaffold based cultures utilize an acellular 3D matrix or a liquid matrix. Scaffold-free methods are normally generated in suspensions.[68] There are a variety of platforms used to facilitate the growth of three-dimensional cellular structures including scaffold systems such as hydrogel matrices[69] and solid scaffolds, and scaffold-free systems such as low-adhesion plates, nanoparticle facilitated magnetic levitation,[70] and hanging drop plates.[71][72] Culturing cells in 3D leads to wide variation in gene expression signatures and partly mimics tissues in the physiological states.[73] A 3D cell culture model showed cell growth similar to that of in vivo than did a monolayer culture, and all three cultures were capable of sustaining cell growth.[74] As 3D culturing has been developed it turns out to have a great potential to design tumors models and investigate malignant transformation and metastasis, 3D cultures can provide aggerate tool for understanding changes, interactions, and cellular signaling.[75]

3D cell culture in scaffolds

Eric Simon, in a 1988 NIH SBIR grant report, showed that electrospinning could be used to produced nano- and submicron-scale polystyrene and polycarbonate fibrous scaffolds specifically intended for use as in vitro cell substrates. This early use of electrospun fibrous lattices for cell culture and tissue engineering showed that various cell types including Human Foreskin Fibroblasts (HFF), transformed Human Carcinoma (HEp-2), and Mink Lung Epithelium (MLE) would adhere to and proliferate upon polycarbonate fibers. It was noted that, as opposed to the flattened morphology typically seen in 2D culture, cells grown on the electrospun fibers exhibited a more histotypic rounded 3-dimensional morphology generally observed in vivo.[17]

As the natural extracellular matrix (ECM) is important in the survival, proliferation, differentiation and migration of cells, different hydrogel culture matrices mimicking natural ECM structure are seen as potential approaches to in vivo like cell culturing.[76] Hydrogels are composed of interconnected pores with high water retention, which enables efficient transport of substances such as nutrients and gases. Several different types of hydrogels from natural and synthetic materials are available for 3D cell culture, including animal ECM extract hydrogels, protein hydrogels, peptide hydrogels, polymer hydrogels, and wood-based nanocellulose hydrogel.

The 3D Cell Culturing by Magnetic Levitation method (MLM) is the application of growing 3D tissue by inducing cells treated with magnetic nanoparticle assemblies in spatially varying magnetic fields using neodymium magnetic drivers and promoting cell to cell interactions by levitating the cells up to the air/liquid interface of a standard petri dish. The magnetic nanoparticle assemblies consist of magnetic iron oxide nanoparticles, gold nanoparticles, and the polymer polylysine. 3D cell culturing is scalable, with the capability for culturing 500 cells to millions of cells or from single dish to high-throughput low volume systems.

Cell culture is a fundamental component of tissue culture and tissue engineering, as it establishes the basics of growing and maintaining cells in vitro.The major application of human cell culture is in stem cell industry, where mesenchymal stem cells can be cultured and cryopreserved for future use. Tissue engineering potentially offers dramatic improvements in low cost medical care for hundreds of thousands of patients annually.

Vaccines for polio, measles, mumps, rubella, and chickenpox are currently made in cell cultures. Due to the H5N1 pandemic threat, research into using cell culture for influenza vaccines is being funded by the United States government. Novel ideas in the field include recombinant DNA-based vaccines, such as one made using human adenovirus (a common cold virus) as a vector,[77][78]and novel adjuvants.[79]

The technique of co-culturing is used to study cell crosstalk between two or more types of cells on a plate or in a 3D matrix. The cultivation of different stem cells and the interaction of immune cells can be investigated in an in vitro model similar to biological tissue. Since most tissues contain more than one type of cell, it is important to evaluate their interaction in a 3D culture environment to gain a better understanding of their interaction and to introduce mimetic tissues. There are two types of co-culturing: direct and indirect. While direct interaction involves cells being in direct contact with each other in the same culture media or matrix, indirect interaction involves different environments, allowing signaling and soluble factors to participate.[1][80]

Cell differentiation in tissue models during interaction between cells can be studied using the Co-Cultured System to simulate cancer tumors, to assess the effect of drugs on therapeutic trials, and to study the effect of drugs on therapeutic trials. The co-culture system in 3D models can predict the response to chemotherapy and endocrine therapy if the microenvironment defines biological tissue for the cells.

A co-culture method is used in tissue engineering to generate tissue formation with multiple cells interacting directly.[81]

Microfluidics technique is developed systems that can perform a process in a flow which are usually in a scale of micron. Microfluidics chip are also known as Lab-on-a-chip and they are able to have continuous procedure and reaction steps with spare amount of reactants and space. Such systems enable the identification and isolation of individual cells and molecules when combined with appropriate biological assays and high-sensitivity detection techniques.[82][83]

OoC systems mimic and control the microenvironment of the cells by growing tissues in microfluidics. Combining tissue engineering, biomaterials fabrication, and cell biology, it offers the possibility of establishing a biomimetic model for studying human diseases in the laboratory. In recent years, 3D cell culture science has made significant progress, leading to the development of OoC. OoC is considered as a preclinical step that benefits pharmaceutical studies, drug development and disease modeling.[84][85] OoC is an important technology that can bridge the gap between animal testing and clinical studies and also by the advances that the science has achieved could be a replace for in vivo studies for drug delivery and pathophysiological studies.[86]

Besides the culture of well-established immortalised cell lines, cells from primary explants of a plethora of organisms can be cultured for a limited period of time before senescence occurs (see Hayflick's limit). Cultured primary cells have been extensively used in research, as is the case of fish keratocytes in cell migration studies.[87][47][88]

Plant cell cultures are typically grown as cell suspension cultures in a liquid medium or as callus cultures on a solid medium. The culturing of undifferentiated plant cells and calli requires the proper balance of the plant growth hormones auxin and cytokinin.

Cells derived from Drosophila melanogaster (most prominently, Schneider 2 cells) can be used for experiments which may be hard to do on live flies or larvae, such as biochemical studies or studies using siRNA. Cell lines derived from the army worm Spodoptera frugiperda, including Sf9 and Sf21, and from the cabbage looper Trichoplusia ni, High Five cells, are commonly used for expression of recombinant proteins using baculovirus.[89]

For bacteria and yeasts, small quantities of cells are usually grown on a solid support that contains nutrients embedded in it, usually a gel such as agar, while large-scale cultures are grown with the cells suspended in a nutrient broth.

The culture of viruses requires the culture of cells of mammalian, plant, fungal or bacterial origin as hosts for the growth and replication of the virus. Whole wild type viruses, recombinant viruses or viral products may be generated in cell types other than their natural hosts under the right conditions. Depending on the species of the virus, infection and viral replication may result in host cell lysis and formation of a viral plaque.

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The Legacy of Henrietta Lacks – Hopkins Medicine

Posted: December 10, 2022 at 12:20 am

In 1951, a young mother of five named Henrietta Lacks visited The Johns Hopkins Hospital complaining of vaginal bleeding. Upon examination, renowned gynecologist Dr. Howard Jones discovered a large, malignant tumor on her cervix. At the time, The Johns Hopkins Hospital was one of only a few hospitals to treat poor African-Americans.

As medical records show, Mrs. Lacks began undergoing radium treatments for her cervical cancer. This was the best medical treatment available at the time for this terrible disease. A sample of her cancer cells retrieved during a biopsy were sent to Dr. George Gey's nearby tissue lab. For years, Dr. Gey, a prominent cancer and virus researcher, had been collecting cells from all patients - regardless of their race or socioeconomic status - who came to The Johns Hopkins Hospital with cervical cancer, but each sample quickly died in Dr. Geys lab. What Dr. Gey would soon discover was that Mrs. Lacks cells were unlike any of the others he had ever seen: where other cells would die, Mrs. Lacks' cells doubled every 20 to 24 hours.

Today, these incredible cells nicknamed "HeLa" cells, from the first two letters of her first and last names are used to study the effects of toxins, drugs, hormones and viruses on the growth of cancer cells without experimenting on humans. They have been used to test the effects of radiation and poisons, to study the human genome, to learn more about how viruses work, and played a crucial role in the development of the polio and COVID-19vaccines.

Although Mrs. Lacks ultimately passed away on October 4, 1951, at the age of 31, her cells continue to impact the world.

Although these were the first cells that could be easily shared and multiplied in a lab setting, Johns Hopkins has never sold or profited from the discovery or distribution of HeLa cells and does not own the rights to the HeLa cell line. Rather, Johns Hopkins offered HeLa cells freely and widely for scientific research.

Johns Hopkins applauds and regularly participates in efforts to raise awareness of the life and story of Henrietta Lacks. Having reviewed our interactions with Henrietta Lacks and with the Lacks family over more than 50 years, we found that Johns Hopkins could have and should have done more to inform and work with members of Henrietta Lacks family out of respect for them, their privacy and their personal interests. Though the collection and use of Henrietta Lacks cells in research was an acceptable and legal practice in the 1950s, such a practice would not happen today without the patients consent.

We are deeply committed to the ongoing efforts at our institutions and elsewhere to honor the contributions of Henrietta Lacks and to ensure the appropriate protection and care of the Lacks familys medical information.

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CAR T Cell Therapy Offers a New Hope in the Treatment of Severe and Refractory Systemic Lupus Erythematosus – Rheumatology Network

Posted: October 4, 2022 at 1:42 am

Chimeric antigen receptor (CAR) T cell therapy has revolutionized the world of oncology with the advent of new personalized treatment for a wide array of malignancies. However, a recent study by Mackensen et al has raised the possibility that CAR T cell therapy may also have a role outside of the treatment in cancer, through positive findings that have been published in the treatment of refractory systemic lupus erythematosus (SLE).1

CAR T cell therapy is already an established treatment used in a variety of haematological malignancies. This is relatively recently developed technology, which has rapidly expanded over the last decade with more than 350 studies currently ongoing investigating the use of this treatment in an array of settings (predominantly oncological). For therapeutic use, circulating peripheral T cells are harvested from the patient and isolated from whole blood. These ex-vivo cells are then genetically engineered to express a chimeric antigen receptor against the desired target (in the case of this study the target antigen being CD19, found on B lymphocytes). The basis of the therapy is that these CAR T cells will then target and destroy cells expressing this antigen. The newly engineered T cells are then grown in cell culture before being infused back into the patient. The process takes approximately 2 weeks from T cell isolation to subsequent CAR T cell infusion. Prior to receiving the newly generated T cells, a period of conditioning treatment, which effectively allows for space for CAR T cell expansion within the bone marrow allowing for these new cells to populate, is required. In this study, the investigated used a combination of fudarabine and cyclophosphamide for this. It is also important to consider that the isolation of circulating T cells requires a sufficient quantity of circulating lymphocytes. As a result, the authors decided to taper immunosuppressive therapy before collection of host T cells (in particular this focused on decreasing T cell cytotoxic medication such as cyclophosphamide and mycophenolate three weeks prior to cell collection).

In their recent Nature Medicine publication, the authors demonstrated the use of anti-CD19 CAR T cell therapy in the treatment of 5 patients with severe and refractory SLE. Patients were aged between 18-24 years old and included 4 females and 1 male. Patients had active disease with a SLE Disease Activity Index 2000 (SLEDAI-2K) of 8-16 at the time of treatment. All had cutaneous disease, in addition to proliferative lupus nephritis (Class III or IV) with 2 having overlapping membranous nephritis (Class V). Four of the 5 patients also had joint involvement. All had been treated with glucocorticoids, hydroxychloroquine, mycophenolate and belimumab with 3 receiving cyclophosphamide and 1 treated with rituximab previously. This represents a group of patients with significantly active disease that was refractory to multiple therapies.

Following infusion of CAR T cells, patients were observed in hospital for a total of 10 days prior to discharged (predominantly to monitor for signs of toxicity). In the use of CAR T cells in the treatment of malignancy, an array of side effects can be observed with the therapy. This includes generalized symptoms (such as fevers and headache). More severe effects include cytokine release syndrome and neurotoxicity. Interestingly, in this study in SLE the treatment was very well tolerated without severe consequences noted.

In the subsequent days following infusion of CAR T cells, the authors identified that these cells initially represented only a small subpopulation with the patients circulating T cell repertoire within the first 24 hours, before significantly expanding by day 9 post-infusion (at which point between 11-59% of circulating T cells were CARs). With regards to clinical response, a remarkable improvement was seen within the first 3 months of therapy. Four of the 5 patients achieved a SLEDAI-2K of zero (whilst the remaining patient seeing an improvement from 16 to 2).

Most impressively, there appeared to be a sustained response to this therapy and the authors report not only long-term remission but that this was treatment-free remission! It is quite astonishing that patients with severe disease that was refractory to multiple other agents showed a drastic improvement and ultimately were able to discontinue all therapy including glucocorticoids! The follow-up period is currently only at a maximum of 17 months, however long-term steroid-free response offers hope for a decrease in damage secondary to prolonged exposure to glucocorticoids (including osteoporosis and increased risk of cardiovascular disease).

Aside from being both extremely effective in achieving remission and well tolerated, the authors also importantly showed no significant reduction in vaccine response in the 5 patients treated with CAR T cells therapy, which is of particular interest at present.

In summary, CAR T cell therapy may not be required in many cases of SLE; however, the findings of this study of anti-CD19 directed CAR T cell therapy offers new hope for the most severe and refractory cases. In addition, the sustained long-term effects of treatment plus the ability to withdraw all treatment also confers that the risk of damage and infection (leading causes of morbidity and mortality in SLE) may also be reduced. The future is very exciting!

Reference:

Mackensen A, Mller F, Mougiakakos D, Bltz S, Wilhelm A, Aigner M, et al. Anti-CD19 CAR T cell therapy for refractory systemic lupus erythematosus. Nature medicine. 2022.

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HOME | Stem cell & Cancer

Posted: October 4, 2022 at 1:42 am

We play with stem cells!We innovate cancer treatment!

Overview

The ultimate goal of our research is to help people live longer and healthier. Preventing death and repairing the aged/diseased organs are essential to achieve this goal. Cancer is the most common cause of death, and organ failure is the most common feature of aging-related diseases. Therefore, regenerative medicine and cancer precision medicine are key areas of convergence biomedical research to prolong human life in the era of 4th industrial revolution.

Our mission is to make innovative and ground-breaking, convergence stem cell and cancer research and translate our research discoveries for the improvement of health and the cure of diseases. The core values of our research group include highest level of professionalism, creativity, innovation, integrity, motivation, resilience, mutual care and team-work. With this mission and core values in mind, we study these three inter-connected and synergizing research areas of

(1) Stem cell biology & regenerative medicine,

(2) Cancer biology & precision medicine,

(3) Aging & anti-aging medicine.

Lab with K-BioX

Research Summary

Central questions: What are the role of recurrent mutations in stem cell self-renewal, cancer pathogenesis,

and cancer therapeutic resistance?

Stem cells and cancers are tightly inter-related. Stem cells are oftentimes are the cell of origin for cancers. Also cancers have a subpopulation of cells, so called cancer stem cells (CSCs, a.k.a. tumor-initiating cells), which have stem cell-like characteristics and are considered the source of cancer recurrence. On the other hand, cancer is one of major aging diseases, and stem cells and stem-cell derived organs are the potential sources for anti-aging medicine. Therefore, stem cell research and cancer research are cross-connected and mutually applicable.

Our detailed research focuses include, but are not limited to,

I. Identification of tissue stem cells and their self-renewal mechanisms Still many tissue stem cells have not been identified. Furthermore, stem cell self-renewal and expansion are invaluable for regenerative medicine. We have extensive experience and expertise in these areas and will continue to achieve original and more significant research findings to identify and expand stem cells for regenerative medicine.

- We have for the first time identified human and mouse esophageal stem cells (Jeong et al, Gut, 2016), and we are currently trying to identify other tissue stem cells.

II. Organogenesis The ultimate goal of stem cell biology and regenerative medicine is to generate micro-, mini-, and macro-organ to be used for organ transplantation. Although some researchers including our group succeeded in generating epithelial organoids and some of micro-organs derived from ESCs or iPSCs, the destination is still far to reach. We have built up strong experiences and expertise in epithelial stem cell biology and gear up toward organogenesis.

- As the first step toward organogenesis, we have built up our expertise in organoid culture. We have for the first timedeveloped human and mouse esophageal organoids (Jeong et al, Gut, 2016) and other organoids (to be reported). We are also using a lot of other organoids including tracheal and lung organoids (Jeong et al, Cancer Discovery, 2017), and also tumor organoids.

- We are currently trying to develop mini-organs.

III. Development of targeted therapies for cancers Individualized precision medicine will ultimately refine and maximize the cancer treatment effect and minimize the side effects. We have shown that mutations in Keap1-Nrf2 anti-oxidant pathway promote the pathogenesis of lung squamous cell carcinoma by deregulating airway stem cell self-renewal. We further demonstrated that KEAP1/NRF2 mutations confer lung cancers therapeutic resistance and that genetic pre-screening of the mutation status of lung cancers could help us predict cancer recurrence (Jeong et al, Cancer Discovery, 2017). Now we aim to develop novel therapies precisely targeting KEAP1/NRF2 mutant cancers and cancers with other mutations.

IV. Targeting cancer stem cells (CSCs)- You have to remove the root if you want to get rid of weeds. Likewise, CSC theory suggests that we need to eliminate CSCs to cure cancers. CSCs are a subpopulation of cancer cells with the stem cell-like characteristics and are more resistant to chemotherapy and radiation therapy. We are particularly interested in identifying and targeting CSCs in head and neck and lung cancers.

V. Tumor immunology

- Cytotoxic T lymphocytes (CTLs) and Natural Killer (NK) cells are two major players in tumor immunology. We are interested in regulatory pathways of CTLs and NK cells' activation. By modulating those pathways, we aim to develop novel drugs or therapies against cancers.

VI. Stem cell therapy in lung fibrosis- Idiopathic pulmonary fibrosis (IPF) is one of the representative aging diseases. IPF is a progressive, restrictive lung disease. In IPF, lung epithelium becomes thickened and scarred, impairing gas exchange. However, the role of lung stem cells and their niche in IPF pathogenesis has not been well understood. Thus, we aim to further elucidate the role of lung stem cells in IPF pathogenesis and treatment.

Youngtae Jeong (), M.D., Ph.D.

Principal Investigator,Assistant Professor

Department of New Biology atDGIST

Office: E5-311

Tel: +82-53-785-1620

Email: jyt@dgist.ac.kr

Education and Training

1995-2001 M.D., Seoul National University College of Medicine

2001-2002 Intern, Seoul National University Hospital

2005-2009 Ph.D., Johns Hopkins University School of Medicine

Professional Experiences

2009-2010 Postdoc, Whitehead Institute for Biomedical Research (MIT)

2010-2015 Podstoc, Stanford University Cancer Institute

2015-2018 Instructor, Stanford Univ. Department of Radiation Oncology

2018-Current, Assistant Professor, DGIST Department of New Biology

Honors and Awards (Selected)

2020 DGIST Outstanding Research Award

2019 Outstanding Abstract Award, Korean Cancer Association

2016 Abstract Award, Cleveland Cancer Stem Cell Conference

2016 Travel Award, FASEB Science Research Conference

2014 ECFMG Certificate (US Medical License)

2012 Travel Award, Freston Conference

2008 Korean Honor Scholarship, Embassy of Korea, Washington D.C.

2000 Outstanding Field Research Award, LG Global Challenger Program

International Fellowships and Grants

2012-2015 California Institute for Regenerative Medicine

2012 Stanford University School of Medicine

2008-2009 American Heart Association

Byungmoo Oh (), Ph.D.

Postdoctoral Fellow

Education and Training

B.A., Chungbuk National University, Korea

Ph.D., University of Science and Technology, Korea

bmoh@dgist.ac.kr

Baul Lee (), Ph.D.

Postdoctoral Fellow

Education and Training

B.A., Sahmyook University, Korea

Ph.D., SeoulNational University, Korea

Licensed Pharmacist in Korea (2012)

paul36@dgist.ac.kr

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Cell and Gene Therapy: Rewriting the Future of Medicine – Technology Networks

Posted: October 4, 2022 at 1:42 am

Cell and gene therapies seek to correct the root cause of an illness at the molecular level. These game-changing medicines are reshaping how we address previously untreatable illnesses transforming peoples lives.

Cell and gene therapy represent overlapping fields of research with similar therapeutic goals developing a treatment that can correct the underlying cause of a disease, often a rare inherited condition that can be life-threatening or debilitating and has limited treatment options.

While these technologies were initially developed in the context of treating rare diseases caused by a single faulty gene, they have since evolved towards tackling more common diseases, says Professor Rafael J. Yez-Muoz, director of the Centre of Gene and Cell Therapy (CGCT) at Royal Holloway University of London.

A powerful example is the chimeric antigen receptor (CAR) T-cell therapies, which have been approved for treating certain blood cancers. The approach involves genetically modifying a patients T cells in the laboratory before reintroducing them into the body to fight their disease.

For the first time, we had an example of gene therapy to treat a more common disease demonstrating that the technology has wide applicability, enthuses Yez-Muoz.

To date, 24 cellular and gene therapy products have received approval from the US Food and Drug Administration (FDA) including life-changing treatments for patients with rare diseases, such as inherited forms of blindness and neuromuscular conditions. A variety of gene and cell-based therapies for both rare and common diseases are also currently in development across many therapeutic areas, offering hope for many more families in coming years.

This webinar will provide an introduction to the regulatory framework for cell and gene therapies and highlight the importance of chemistry, manufacturing and controls. Watch to learn about regulatory concerns, safety and quality testing throughout the product lifecycle and key acronyms and terminology.

Gene therapies seek to introduce specific DNA sequences into a patients body to treat, prevent or potentially cure a disease. This may involve the delivery of a functional gene into cells to replace a gene that is missing or causing a problem or other strategies using nucleic acid sequences (such as antisense oligonucleotides or short interfering RNAs [siRNAs]) to reduce, restore or modify gene expression. More recently, scientists are also developing genome-editing technologies that aim to change the cells DNA at precise locations to treat a specific disease.

The key step in successful gene therapy relies on the safe and efficient delivery of genetic material into the target cells, which is carried out by packaging it into a suitable delivery vehicle (or vector). Many current gene therapies employ modified viruses based on adenoviruses, adeno-associated viruses (AAV), and lentiviruses as vectors due to their intrinsic ability to enter cells. But non-viral delivery systems such as lipid nanoparticles (LNPs) have also been successfully employed to deliver RNA-based therapeutics into cells.

A big advantage of using viral vectors for gene delivery is they are longer lasting than non-viral systems, states Dr. Rajvinder Karda, lecturer in gene therapy at University College London. Many of the rare diseases were aiming to tackle are severe and we need to achieve long-term gene expression for these treatments to be effective.

While improved technological prowess empowers the development of CRISPR-edited therapies, supply-chain and manufacturing hurdles still pose significant barriers to clinical and commercialization timelines. Watch this webinar to learn more about the state of CRISPR cell and gene therapies, challenges in CRISPR therapy manufacturing and a next-generation manufacturing facility.

Viral-vector gene therapies are either administered directly into the patients body (in vivo), or cells harvested from a patient are instead modified in the laboratory (ex vivo) and then reintroduced back into the body. Major challenges for in vivo gene delivery approaches are with the safe and efficient targeting of the therapeutic to the target cells and overcoming any potential immune responses to the vectors.

As well as getting the genetic material into the affected cells, we also need to try and limit it reaching other cells as expressing a gene in a cell where its not normally active could cause problems, explains Dr. Gerry McLachlan, group leader at the Roslin Institute in Edinburgh.

For example, the liver was identified as a major site of toxicity for an AAV-based gene therapy approved for treating spinal muscular atrophy (SMA), a type of motor neuron disease that affects people from a very young age.

Unfortunately, these viruses are leaky as theyre also going to organs that dont need therapy meaning you can get these off-target effects, says Karda. Theres still work to be done to develop and refine these technologies to make them more cell- and organ-specific.

It is also important to ensure the gene is expressed at the right level in the affected cells too high and it may cause side effects and too little may render the treatment ineffective. In a recent major advancement in the field, scientists developed a dimmer switch system Xon that enables gene expression to be precisely controlled through exposure to an orally delivered small molecule drug. This novel system offers an unprecedented opportunity to refine and tailor the application of gene therapies in humans.

Download this whitepaper to discover an electroporation system that resulted in CAR transfection efficiencies as high as 70% in primary human T cells, can avoid the potential risks associated with viral transduction and is able to produce CAR T cells at a sufficient scale for clinical and therapeutic applications.

In 1989, a team of researchers identified the gene that causes the chronic, life-limiting inherited disease cystic fibrosis (CF) the cystic fibrosis transmembrane conductance regulator (CFTR). This was the first ever disease-causing gene to be discovered marking a major milestone in the field of human genetics. In people with CF, mutations in the CFTR gene can result in no CTFR protein, or the protein being made incorrectly or at insufficient levels all of which lead to a cascade of problems that affect the lungs and other organs.

Our team focuses on developing gene therapies to treat respiratory diseases in particular, were aiming to deliver the CTFR gene into lung cells to treat CF patients, says McLachlan.

The results of the UK Respiratory Gene Therapy Consortiums most recent clinical trial showed that an inhaled non-viral CTFR gene therapy formulation led to improvements in patient lung function.

While this was encouraging, the effects were modest and we need to develop a more potent delivery vehicle, explains McLachlan. Weve also been working on a viral-based gene therapy using a lentiviral vector to introduce a healthy copy of the CTFR gene into cells of the lung.

Kardas team focuses on developing novel gene therapy and gene-editing treatments for incurable genetic diseases affecting the central and peripheral nervous system and Yez-Muoz is aiming to develop new treatments for rare neurodegenerative diseases that affect children, including SMA and ataxia telangiectasia (AT).

But a significant barrier for academic researchers around the world is accessing the dedicated resources, facilities and expertise required to scale up and work towards the clinical development and eventually the commercial production of gene and cell therapies. These challenges will need to be addressed and overcome if these important advancements are to successfully deliver their potentially life-changing benefits to patients.

Download this app note to discover how electron activated dissociation can obtain in-depth structural characterization of singly charged, ionizable lipids and related impurities, decrease risk of missing critical low abundance impurities and increase confidence in product quality assessment.

After many decades of effort, the future of gene and cell therapies is incredibly promising. A flurry of recent successes has led to the approval of several life-changing treatments for patients and many more products are in development.

Its no longer just about hope, but now its a reality with a growing number of rare diseases that can be effectively treated with these therapies, describes Yez-Muoz. We now need to think about how we can scale up these technologies to address the thousands of rare diseases that exist and even within these diseases, people will have different mutations, which will complicate matters even further.

But as more of these gene and cell-based therapies are approved, there is a growing urgency to address the challenge of equitable access to these innovative treatments around the world.

Gene therapies have the dubious honor of being the most expensive treatments ever and this isnt sustainable in the longer term, says Yez-Muoz. Just imagine being a parent and knowing there is an effective therapy but your child cant access it that would be absolutely devastating.

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Outlook on the Automated Cell Counters Global Market to 2028 – Use of Cell Counters in Personalized Medicine Presents Opportunities -…

Posted: October 4, 2022 at 1:42 am

DUBLIN--(BUSINESS WIRE)--The "Automated Cell Counters Market Forecast to 2028 - COVID-19 Impact and Global Analysis By Type and End User" report has been added to ResearchAndMarkets.com's offering.

The automated cell counters market is expected to grow from US$ 6,974.29 million in 2021 to US$ 10,365.95 million by 2028; it is estimated to grow at a CAGR of 5.9% from 2022 to 2028.

The report highlights trends prevailing in the market and factors driving the market growth. The market growth is attributed to the high prevalence of infectious and chronic diseases. Additionally, advancement in automated cell counters is likely to emerge as a significant trend in the market during the forecast period. However, the lack of a skilled workforce and the high instrument cost limit the market growth.

Chronic diseases are conditions that are present in an individual for one or more years, require ongoing medical attention, and can also result in limited daily activities. Chronic diseases are currently the major cause of death among adults in several countries. According to World Health Organization (WHO), 41 million people die yearly due to chronic diseases, equivalent to 71% of all deaths globally.

As per the Centers for Disease Control and Prevention (CDC), six in ten adults in the US have a chronic disease, and four in ten adults have two or more chronic diseases. According to Cancer Research UK, ~17 million new cases of cancer were detected worldwide in 2018. Further, in 2018, ~9.6 million deaths occurred due to cancer worldwide.

Infectious diseases are caused by infectious agents, such as viruses, bacteria, parasites, fungi, and toxic products. HIV is a major public health issue across the world. As per The Joint United Nations Programme on HIV/AIDS (UNAIDS), ~ 37.7 million people had HIV in 2020; out of these, 1.7 million were children aged 0-14 years, and 36 million were adults.

Further, over half of them (53%) were girls and women, and 1.5 million new HIV cases were globally reported in 2020. Similarly, hepatitis is inflammation of the liver caused by a viral infection. The five primary strains of hepatitis viruses are A, B, C, D, and E. According to WHO, ~58 million people have chronic hepatitis C, and ~1.5 million new infections occur every year.

According to WHO, tuberculosis (TB) is the thirteenth leading cause of death globally and the second leading infectious disease after COVID-19. Furthermore, 1.5 million deaths were caused by TB in 2020 (including 214,000 people affected by HIV). In 2020, the WHO estimated that 10 million people had TB, including 1.1 million children, 3.3 million women, and 5.6 million men. TB cases are present in all age groups and countries. Furthermore, 30 countries with high TB burdens accounted for 86% of new TB cases in 2020. Eight countries registered two-thirds of the total TB cases, with India at the forefront, followed by China, the Philippines, Indonesia, Nigeria, Pakistan, Bangladesh, and South Africa.

Diagnostics are essential in determining the direction of any medical treatment of infectious and chronic diseases. Cell counting is one of the methods that is used for the detection of such diseases. Therefore, the rising prevalence of infectious and chronic diseases across the globe is driving the growth of the automated cell counters market.

On the other hand, the lack of a skilled workforce and high instrument cost hinders the overall automated cell counters market growth. According to a WHO report, there is a drastic shortage of healthcare professionals or workers trained to use automated cell counter equipment. The ongoing research in pharmaceutical and biotechnology and the development of various drugs to treat diseases such as cancer, cardiovascular disorders, HIV/AIDS, etc.

With technological advancements in automated cell counters and a rise in application areas of the instrument, there has been a shift in usage of automated cell counters. The working of the automated cell counter is difficult, and knowledge of this instrument is highly important; hence, there is a demand for a skilled workforce. The preparation of a sample for such instruments is tedious work, and the consumables required during the procedure also need to be handled properly. Thus, a lack of a skilled workforce who can easily use these instruments is hampering the growth of the automated cell counters market.

Market Dynamics

Drivers

Restraints

Opportunities

Future Trends

Companies Mentioned

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

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Jcr Pharmaceuticals Co., Ltd. and Sysmex Establish A Joint Venture in the Field of Regenerative Medicine and Cell Therapy – Marketscreener.com

Posted: October 4, 2022 at 1:42 am

JCR Pharmaceuticals Co., Ltd. and Sysmex Corporation announced that they have established a joint venture(hereafter the "joint venture") for carrying out research and development, manufacture and sales of cell-based regenerative medicine products including hematopoietic stem cells and other stem cells. In recent years, the significant potential of regenerative medicine and cell therapy have been established in particular in areas that have traditionally been difficult to address with conventional chemically synthesized low molecular weight drugs1 or biopharmaceuticals2, such as the restoration of tissues and functions lost as a result of aging, illness, autoimmune diseases, or cancer. In particular, research and development on the therapeutic application of stem cells including hematopoietic stem cells, mesenchymal stem cells, and iPS cells have generated significant attention. Since its inception, JCR has been engaged in the research, development, manufacturing and sales of pharmaceutical products using regenerative medicine, genetic engineering, and gene therapy technologies to advance therapies in the rare disease field. This is exemplified in the field of regenerative medicine, by the approval of TEMCELL HS Inj.3, the first allogeneic regenerativemedicine in Japan (Non-proprietary name: Human (allogeneic) bone marrow-derived mesenchymal stem cells) in February 2016 for the treatment of acute graft-versus-host disease (acute GVHD)4, a serious complication that develops after hematopoietic stem cell transplantation. In recent years, JCR has further streamlined and integrated its expertise around the establishment of groundbreaking medicines for the advancement of highly innovative medicines that could not be developed without such groundbreaking technologies. In the joint venture, the two companies aim to realize the social implementation of regenerative medicine and cell therapy by integrating JCR's expertise in developing, manufacturing and marketing regenerative medicine products, with Sysmex's expertise in quality control testing technology and knowledge of workflows efficiency using robotics technology, including IoT. AlliedCel Corporation, which is the corporate name of the joint venture following prior discussions regarding the alliance both companies, was established on October 3, 2022. The joint venture will advance programs of the potential for technology development and commercialization, including the project currently being promoted by both companies using hematopoietic stem cell proliferation technology. The name AlliedCel stands for the joint venture's aspiration to integrate knowledge and expertise from a broad set of collaborators and stakeholders including business partners, patients and their families, with the united goal of unleashing the power of cells in supporting patients in their needfor life-changing therapies. Through the research and development of regenerative medicineproducts using diverse cells such as stem cells, AlliedCel aims to provide appropriate treatmentoptions to patients and improve their prognosis.

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Growth in Cell and Gene Therapy Market – BioPharm International

Posted: October 4, 2022 at 1:42 am

Biopharma focuses on streamlining biomanufacturing and supply chain issues to drive uptake of cell and gene therapies.

Cell and gene therapies (CGTs) offer significant advances in patient care by helping to treat or potentially cure a range of conditions that have been untouched by small molecule and biologic agents. Over the past two decades, more than 20 CGTs have been approved by FDA in the United States and many of these one-time treatments cost between US$375,00 and US$2 million a shot (1). Given the high financial outlay and patient expectations of these life-saving therapies, it is essential that manufacturers provide integrated services across the whole of the supply chain to ensure efficient biomanufacturing processes and seamless logistics to reduce barriers to uptake.

The following looks at the who, what, when, and why of biomanufacturing and logistics in CGTs in the bio/pharmaceutical industry in more detail.

According to market research, the global gene therapy market will reach US$9.0 billion by 2027 due to favorable reimbursement policies and guidelines, product approvals and fast-track designations, growing demand for chimeric antigen receptor (CAR) T cell-based gene therapies, and improvements in RNA, DNA, and oncolytic viral vectors (1).

In 2020, CGT manufacturers attracted approximately US$2.3 billion in investment funding (1). Key players in the CGT market include Amgen, Bristol-Myers Squibb Company, Dendreon, Gilead Sciences, Novartis, Organogenesis, Roche (Spark Therapeutics), Smith Nephew, and Vericel. In recent years, growth in the CGT market has fueled some high-profile mergers and acquisitions including bluebird bio/BioMarin, Celgene/Juno Therapeutics, Gilead Sciences/Kite, Novartis/AveXis and the CDMO CELLforCURE, Roche/Spark Therapeutics, and Smith & Nephew/Osiris Therapeutics.

Many bio/pharma companies are re-considering their commercialization strategies and have re-invested in R&D to standardize vector productions and purification, implement forward engineering techniques in cell therapies, and improve cryopreservation of cellular samples as well as exploring the development of off-the-shelf allogeneic cell solutions (2).

The successful development of CGTs has highlighted major bottlenecks in the manufacturing facilities, and at times, a shortage of raw materials (3). Pharma companies are now taking a close look at their internal capabilities and either investing in their own manufacturing facilities or outsourcing to contract development and manufacturing organizations (CDMOs) or contract manufacturing organizations (CMOs) to expand their manufacturing abilities (4). Recently, several CDMOsSamsung Biologics, Fujifilm Diosynth, Boehringer Ingelheim, and Lonzahave all expanded their biomanufacturing facilities to meet demand (5).

A major challenge for CGT manufacturers is the seamless delivery of advanced therapies. There is no room for error. If manufacturers cannot deliver the CGT therapy to the patient with ease, the efficacy of the product becomes obsolete. Many of these therapies are not off-the-shelf solutions and therefore require timely delivery and must be maintained at precise temperatures to remain viable. Thus, manufacturers must not only conform to regulations, but they must also put in place logistical processes and contingency plans to optimize tracking, packaging, cold storage, and transportation through the products journey. Time is of the essence, and several manufacturers have failed to meet patient demands, which have significant impacts on the applicability of these agents.

Several CAR T-cell therapies have now been approved; however, research indicates that a fifth of cancer patients who are eligible for CAR-T therapies pass away while waiting for a manufacturing slot (6). Initially, the manufacture of many of these autologous products took around a month, but certain agents can now be produced in fewer than two weeks (7). Companies are exploring new ways to reduce vein-to-vein time (collection and reinfusion) through the development of more advanced gene-transfer tools with CARs (such as transposon, CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) among others, and the use of centralized organization with standardized apheresis centers (5). Others are exploring the use of the of allogeneic stem cells including Regen Biopharma, Escape Therapeutics, Lonza, Pluristem Therapeutics, and ViaCord (7).

Several gene therapies have also been approved, mainly in the treatment of rare disease (8). Many companies are evaluating novel gene therapy vectors to increase levels of gene expression/protein productions, reduce immunogenicity and improve durability including Astellas Gene Therapies, Bayer, ArrowHead Pharmaceuticals, Bayer, Bluebird Bio, Intellia Therapeutics, Kystal Biotech, MeiraGTx, Regenxbio, Roche, Rocket Pharmaceuticals, Sangamo Therapeutics, Vertex Pharmaceuticals, Verve Therapeutics, and Voyager Therapeutics (8).

While many biopharma companies have established their own in-house CGT good manufacturing practice (GMP) operation capabilities, others are looking to decentralize manufacturing and improve distribution by relying on external contracts with CDMOs and CMOs such as CELLforCURE, CCRM, Cell Therapies Pty Ltd (CTPL), Cellular Therapeutics Ltd (CTL), Eufets GmbH, Gravitas Biomanufacturing, Hitachi Chemical Advances Therapeutic Solutions, Lonza, MasTHerCell, MEDINET Co., Takara Bio, and XuXi PharmaTech (6, 9, 10).

The top 50 gene therapy start-up companies have attracted more than $11.6 billion in funds in recent years, with the top 10 companies generating US$5.3 billion in series A to D funding rounds (10). US-based Sana Biotechnology leads the field garnering US$700 million to develop scalable manufacturing for genetically engineered cells and its pipeline program, which include CAR-T cell-based therapies in oncology and CNS (Central Nervous System) disorders (11). In second place, Editas Medicine attracted $656.6 million to develop CRISPR nuclease gene editing technologies to develop gene therapies for rare disorders (12).

Overall, CGTs have attracted the pharma industrys attention as they provide an alternative route to target diseases that are poorly served by pharmaceutical and/or medical interventions, such as rare and orphan diseases. Private investors continue to pour money into this sector because a single shot has the potential to bring long-lasting clinical benefits to patients (13). In addition, regulators have approved several products and put in place fast track designation to speed up patient access to these life-saving medicines. Furthermore, healthcare providers have established reimbursement policies and manufacturers have negotiated value- and outcome-based contracts to reduce barriers to access to these premium priced products

On the downside, the manufacture of CGTs is labor intensive and expensive with manufacturing accounting for approximately 25% of operating expenses, plus there is still significant variation in the amount of product produced. On the medical side, many patients may not be suitable candidates for CGTs or not produce durable response due to pre-exposure to the viral vector, poor gene expression, and/or the development of immunogenicity due to pre-exposure to viral vectors. Those that can receive these therapies may suffer infusion site reactions, and unique adverse events such as cytokine release syndrome and neurological problems both of which can be fatal if not treated promptly (14).

Despite the considerable advances that have been made in the CGT field to date, there is still much work needed to enhance the durability of responses, increase biomanufacturing efficiencies and consistency and to implement a seamless supply chain that can ensure these agents are accessible, cost-effective, and a sustainable option to those in need.

Cleo Bern Hartley is a pharma consultant, former pharma analyst, and research scientist.

BioPharm InternationalVol. 35, No. 10October 2022Pages: 4951

When referring to this article, please cite it as C.B. Hartley, "Growth in Cell and Gene Therapy Market," BioPharm International 35 (10) 4951 (2022).

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