Category Archives: Stem Cells

Abstract: Background Brain strokes are the cause of death in many people, among survivors; it can cause problems such as motor and cognitive impairment. The role of the hippocampus and its damage in ischemia has been assessed by researchers. One of the treatments commonly used today by researchers in cell therapy. Therefore, this study aimed to evaluate the use of dental pulp stem cells and erythropoietin in mice hippocampus after ischemia-reperfusion. Methods In this study, NMRI male mice were divided into six groups. Except for the sham group, all groups group experienced ischemic hippocampus. A group received erythropoietin or dental pulp stem cells and the other group received a combination exposer of erythropoietin and DPSC, while the vehicle group received DPSC solvent and erythropoietin solvent. After eight weeks, they were subjected to a test of learning and memory by Morris water maze. Then, their brains were examined for histological assessment, and immunohistochemistry (DCX and NeuN for neurogenesis). Furthermore, VEGF was applied for angiogenesis and GFAP for gliosis examination. Results The behavioral function of the group receiving erythropoietin and the combined group (DPSC and erythropoietin) was better than other groups. The mean number of healthy cells in EPO, DPSC, and EPO + DPSC groups was significantly different from that of the vehicle group (P < 0.05). Besides, DPSC, EPO, and EPO + DPSC groups showed a significant increase in green density in comparison with the ischemia and vehicle groups (P < 0.05), but no difference was found between the ischemia and sham groups. Conclusion DPSC and erythropoietin were capable of increased neuronal function but behavioral studies revealed that outcomes of erythropoietin therapy are better than DPSC

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The neuroprotective effects of dental pulp stem cells and erythropoietin in mice hippocampus after ischemia-reperfusion - Newswise

This cross-section of a rat brain shows tissue from a human brain organoid fluorescing in light green. Scientists say these implanted clusters of human neurons could aid the study of brain disorders. Pasca lab / Stanford Medicine hide caption

This cross-section of a rat brain shows tissue from a human brain organoid fluorescing in light green. Scientists say these implanted clusters of human neurons could aid the study of brain disorders.

Scientists have demonstrated a new way to study conditions like autism spectrum disorder, ADHD, and schizophrenia.

The approach involves transplanting a cluster of living human brain cells from a dish in the lab to the brain of a newborn rat, a team from Stanford University reports in the journal Nature.

The cluster, known as a brain organoid, then continues to develop in ways that mimic a human brain and may allow scientists to see what goes wrong in a range of neuropsychiatric disorders.

"It's definitely a step forward," says Paola Arlotta, a prominent brain organoid researcher at Harvard University who was not involved in the study. "The ultimate goal of this work is to begin to understand features of complex diseases like schizophrenia, autism spectrum disorder, bipolar disorder."

But the advance is likely to make some people uneasy, says bioethicist Insoo Hyun, director of life sciences at the Museum of Science in Boston and an affiliate of the Harvard Medical School Center for Bioethics.

"There is a tendency for people to assume that when you transfer the biomaterials from one species into another, you transfer the essence of that animal into the other," Hyun says, adding that even the most advanced brain organoids are still very rudimentary versions of a human brain.

The success in transplanting human brain organoids into a living animal appears to remove a major barrier to using them as models of human disease. It also represents the culmination of seven years of work overseen by Dr. Sergiu Pasca, a professor of psychiatry and behavioral sciences at Stanford.

Human brain organoids are made from pluripotent stem cells, which can be coaxed into becoming various types of brain cells. These cells are grown in a rotating container known as a bioreactor, which allows the cells to spontaneously form brain-like spheres about the size of a small pea.

But after a few months, the lab-grown organoids stop developing, says Pasca, whose lab at Stanford devised the transplant technique. Individual neurons in the cluster remain relatively small, he says, and make relatively few connections.

"No matter how long we keep them in a dish, they still do not become as complex as human neurons would be in an actual human brain," Pasca says. That may be one reason organoids have yet to reveal much about the origins of complex neuropsychiatric disorders, he says.

So Pasca's team set out to find an environment for the organoids that would allow them to continue growing and maturing. They found one in the brains of newborn rats.

"We discovered that the [organoid] grows, over the span of a few months, about nine times in volume," Pasca says. "In the end it covers roughly about a third of a rat's hemisphere."

The transplanted cells don't seem to cause problems for the rats, who behave normally as they grow, Pasca says.

"The rat tissue is just pushed aside," he says. "But now you also have a group of human cells that are integrating into the circuitry."

The human cells begin to make connections with rat cells. Meanwhile, the rat's blood vessels begin to supply the human cells with oxygen and nutrients.

Pasca's team placed each organoid in an area of the rat brain that processes sensory information. After a few months, the team did an experiment that suggested the human cells were reacting to whatever the rat was sensing.

"When you stimulate the whiskers of the rat, the majority of human neurons are engaged in an electrical activity that follows that stimulation," Pasca says.

Another experiment suggests the human cells could even influence a rat's behavior.

The team trained rats to associate stimulation of their human cells with a reward a drink of water. Eventually, the rats began to seek water whenever the human cells were stimulated.

In a final experiment, Pasca's team set out to show how transplanted organoids could help identify the brain changes associated with a specific human disorder. They chose Timothy Syndrome, a very rare genetic disorder that affects brain development in ways that can cause symptoms of autism spectrum disorder.

The team compared organoids made from the stem cells of healthy people with organoids made from the stem cells of patients with the syndrome. In the lab, the cell clusters looked the same.

"But once we transplanted and we looked 250 days later, we discovered that while control cells grew dramatically, patient cells failed to do so," Pasca says.

A better model, with ethical concerns

The experiments show that Pasca's team has developed a better model for studying human brain disorders, Arlotta says.

The key seems to be providing the transplanted organoids with sensory information that they don't get growing in a dish, she says, noting that an infant's brain needs this sort of stimulation to develop normally.

"It's the stuff that we get after we are born," she says, "especially when we begin to experience the world and hear sound, see light, and so on."

But as brain organoids become more like actual human brains, scientists will have to consider the ethical and societal implications of this research, Arlotta says.

"We need to be able to watch it, consider it, discuss it and stop it if we think we think one day we are at the point where we shouldn't progress," she says. "I think we are far, far away from that point right now."

Even the most advanced brain organoids have nothing even remotely like the capabilities of a human brain, says Hyun, who posted a video conversation he had with Pasca to coincide with the publication of the new study.

Yet many ethical discussions have focused on the possibility that an organoid could attain human-like consciousness.

"I think that's a mistake," Hyun says. "We don't exactly know what we mean by 'human-like consciousness,' and the nearer issue, the more important issue, is the well-being of the animals used in the research."

He says that wasn't a problem in the Pasca lab's experiments because the organoids didn't seem to harm the animals or change their behavior.

If human brain organoids are grown in larger, more complex animal brains, Hyun says, the cell clusters might develop in ways that cause the animals to suffer.

"What I'm concerned about," he says, "is what's next."

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Human neuron clusters transplanted into rats offer new tool to study the brain : Shots - Health News - NPR

Swinburne University of Technology has received a share of a $7 million Medical Research Future Fund grant to develop first-of-its-kind Australian research to allow live stem cells to be 3D printed and used in treatments.

The cross-institutional research team will develop novel cartilage-based stem cell therapies that will change the way we care for people living with painful joint disease, such as osteoarthritis, and facial disfigurement.

More than two million Australians live with the painful and degenerative joint disease, osteoarthritis, and one in 2,000 newborns are born with microtia an absent or poorly formed ear which can lead to hearing loss, speech and literacy delays.

This research could actually restore damaged or absent cartilage, transforming how these conditions are treated and vastly improving quality of life for sufferers.

It will use technology to revolutionise the way we think about personalised care, patient involvement and scientific advancements.

Cartilage, like that pictured, is a flexible connective tissue that protects our joints and bones

This ambitious project builds on many years of previous research, including at Swinburne.

In the initial stages, the five-year project will focus on the technologies used to take live stem cell printing from research labs into clinical settings. The team will then proceed to clinical trials to prove the efficacy of the solution.

Led by University of Melbourne Professor Peter Choong, the researchers also hope to simplify processes to bring these treatments into hospitals so that clinicians can treat conditions more quickly, with fewer complications than before.

In addition to Swinburne and the University of Melbourne, the research team also includes experts from La Trobe University, St Vincents Hospital Melbourne, University of Wollongong, University of Sydney, Royal Prince Alfred Hospital, Monash University, RMIT and the University of Toronto.

Swinburne will develop a bioreactor system using its patent-protected materials, which allow stem cells to be expanded to large numbers that can be used to repair and replace damaged or missing cartilage.

Expert in biomedical electromaterials science, Professor Simon Moulton, will lead the Swinburne team.

This grant allows us to continue the work we have already been doing with the other partners over many years in developing innovative cartilage repair strategies, says Professor Moulton.

As a materials engineering researcher in the medical area, we do not always have the opportunity to translate our efforts from fundamental research into a clinical human solution. The $7 million of total grant funding will allow us to continue to develop the stem cell technology towards clinical translation that will provide benefit to a wide range of patients.

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A $7 million grant to grow stem cell research - Swinburne University of Technology

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Global Stem Cell Manufacturing Market

Global Stem Cell Manufacturing Market

Dublin, Oct. 11, 2022 (GLOBE NEWSWIRE) -- The "Stem Cell Manufacturing Market: Global Industry Trends, Share, Size, Growth, Opportunity and Forecast 2022-2027" report has been added to ResearchAndMarkets.com's offering.

The global stem cell manufacturing market size reached US$ 11.2 Billion in 2021. Looking forward, the publisher expects the market to reach US$ 18.59 Billion by 2027, exhibiting a CAGR of 8.81% during 2021-2027.

Stem cells are undifferentiated or partially differentiated cells that make up the tissues and organs of animals and plants. They are commonly sourced from blood, bone marrow, umbilical cord, embryo, and placenta. Under the right body and laboratory conditions, stem cells can divide to form more cells, such as red blood cells (RBCs), platelets, and white blood cells, which generate specialized functions.

They are widely used for human disease modeling, drug discovery, development of cell therapies for untreatable diseases, gene therapy, and tissue engineering. Stem cells are cryopreserved to maintain their viability and minimize genetic change and are consequently used later to replace damaged organs and tissues and treat various diseases.

Stem Cell Manufacturing Market Trends:

The global market is primarily driven by the increasing venture capital (VC) investments in stem cell research due to the rising awareness about the therapeutic potency of stem cells. Apart from this, the widespread product utilization in effective disease management, personalized medicine, and genome testing applications are favoring the market growth. Additionally, the incorporation of three-dimensional (3D) printing and microfluidic technologies to reduce production time and lower cost by integrating multiple production steps into one device is providing an impetus to the market growth.

Furthermore, the increasing product utilization in the pharmaceutical industry for manufacturing hematopoietic stem cells (HSC)- and mesenchymal stem cells (MSC)-based drugs for treating tumors, leukemia, and lymphoma is acting as another growth-inducing factor.

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Moreover, the increasing product application in research applications to produce new drugs that assist in improving functions and altering the progress of diseases is providing a considerable boost to the market. Other factors, including the increasing usage of the technique in tissue and organ replacement therapies, significant improvements in medical infrastructure, and the implementation of various government initiatives promoting public health, are anticipated to drive the market.

Key Players

Anterogen Co. Ltd.

Becton Dickinson and Company

Bio-Rad Laboratories Inc.

Bio-Techne Corporation

Corning Incorporated

FUJIFILM Holdings Corporation

Lonza Group AG

Merck KGaA

Sartorius AG

Takara Bio Inc.

Thermo Fisher Scientific Inc.

Key Questions Answered in This Report:

How has the global stem cell manufacturing market performed so far and how will it perform in the coming years?

What has been the impact of COVID-19 on the global stem cell manufacturing market?

What are the key regional markets?

What is the breakup of the market based on the product?

What is the breakup of the market based on the application?

What is the breakup of the market based on the end user?

What are the various stages in the value chain of the industry?

What are the key driving factors and challenges in the industry?

What is the structure of the global stem cell manufacturing market and who are the key players?

What is the degree of competition in the industry?

Key Market Segmentation

Breakup by Product:

Consumables

Culture Media

Others

Instruments

Bioreactors and Incubators

Cell Sorters

Others

Stem Cell Lines

Hematopoietic Stem Cells (HSC)

Mesenchymal Stem Cells (MSC)

Induced Pluripotent Stem Cells (iPSC)

Embryonic Stem Cells (ESC)

Neural Stem Cells (NSC)

Multipotent Adult Progenitor Stem Cells

Breakup by Application:

Research Applications

Life Science Research

Drug Discovery and Development

Clinical Application

Allogenic Stem Cell Therapy

Autologous Stem Cell Therapy

Cell and Tissue Banking Applications

Breakup by End User:

Pharmaceutical & Biotechnology Companies

Academic Institutes, Research Laboratories and Contract Research Organizations

Hospitals and Surgical Centers

Cell and Tissue banks

Others

Breakup by Region:

North America

United States

Canada

Asia-Pacific

China

Japan

India

South Korea

Australia

Indonesia

Others

Europe

Germany

France

United Kingdom

Italy

Spain

Russia

Others

Latin America

Brazil

Mexico

Others

Middle East and Africa

Key Topics Covered:

1 Preface

2 Scope and Methodology

3 Executive Summary

4 Introduction

5 Global Stem Cell Manufacturing Market

6 Market Breakup by Product

7 Market Breakup by Application

8 Market Breakup by End User

9 Market Breakup by Region

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Stem Cell Manufacturing Global Market Report 2022: Widespread Product Utilization in Effective Disease Management, Personalized Medicine, and Genome...

Kristen Brennand

Kristen Brennand, who in her research integrates expertise in genetics, neuroscience, and stem cells to identify the mechanisms that underlie brain disease, was recently appointed the Elizabeth Mears and House Jameson Professor of Psychiatry.

She is also co-director of the Yale Science Fellows Program, a Yale School of Medicine initiative aimed at recruiting, supporting, and promoting outstanding young scientists from groups traditionally underrepresented in science and medicine.

Brennand completed her Ph.D. at Harvard University in the laboratory of the noted stem cell biologist Dr. Douglas Melton. During her postdoctoral fellowship at the Salk Institute, she drew international notice for publishing the first cellular model for schizophrenia. She developed a new method for reprogramming skin samples from patients into human induced pluripotent stem cells and then she differentiated these stem cells into neurons. Her initial report demonstrated that neurons derived from schizophrenia patients had profound deficits in synaptic connectivity, i.e., were less well connected to each other.

While on the faculty at the Icahn School of Medicine at Mount Sinai, Brennand developed a highly productive laboratory and a network of collaborations. By combining stem cell biology, psychiatric genetics, and neurobiology, she pioneered a new approach to studying brain disease. She and her collaborators shed light on the genetics and biology of schizophrenia, bipolar disorder, and other conditions. She was interim director of the Pamela Sklar Division of Psychiatric Genomics and then director of the Alper Stem Cell Center.

Although Brennand arrived at Yale during the pandemic, she rapidly established a productive laboratory, created new interdepartmental collaborations, and distinguished herself as a valued teacher and mentor. Her laboratory also is quite well funded with competitive grants from the National Institutes of Health (NIH).

She also has received numerous honors. The Brain and Behavior Research Foundation awarded her the Maltz Prize for Schizophrenia Research and elected her to its Scientific Council. This year, she was elected to the Connecticut Academy of Science and Engineering and named as a finalist for the 2022 Blavatnik Awards for Young Scientists. She also has developed a reputation as a mentor to her trainees and other young scientists. In 2019, she received the Friedman Brain Institute Neuroscience Mentorship Distinction Award. She serves as a standing member of NIH study section and the editorial boards of seven journals in psychiatry, stem cell biology, and neuroscience.

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Brennand named Elizabeth Mears and House Jameson Professor of Psychiatry - Yale News

MIAMI, Oct. 11, 2022 (GLOBE NEWSWIRE) -- The American Academy of Stem Cell Physicians will be hosting its fall Scientific Congress in Chicago, IL, on Oct. 28-30, 2022. The conference will feature three days of educational and networking events with leading physicians from across the fields of stem cells, live cells, and regenerative medicine. A Board Examination process will be available, creating a pathway for participants to earn a Diplomat and Fellowship Certification in Regenerative Medicine.

The Board of American Academy of Stem Cell Physicians is the official board certifying body of the American Academy of Stem Cell Physicians (AASCP). As a nationally recognized academy with a mission to bring like-minded physicians together to increase awareness and education for the evolving field of regenerative medicine, the AASCP is proud to announce its Fellowship and Diplomat Certification.

In order to be eligible for certification or recertification through the AASCP, licensed physicians in good standing must meet the stringent eligibility requirements that have been defined by the board. AASCP places an emphasis on not only psychometrically evaluated testing and advanced training, but also moral character and experience. Furthermore, AASCP has a clear path toward recertification for qualified physicians. Their standards for recertification include a commitment to continuing medical education, successful completion of a recertification examination, participation in a non-remedial medical ethics program, and additional requirements.

AASCP is known for working with physicians to provide unique opportunities for board certification in their specialty of regenerative medicine. Specifically, the AASCP offers ongoing workshop modules led by esteemed physicians in this field who certify and educate on different treatment approaches and techniques. Another defining characteristic of the AASCP is their commitment to ongoing education and awareness. To support this goal, the AASCP has developed innovative committees, including its Institutional Review Board and created opportunities for physicians and researchers to submit their work for peer review and exposure.

The AASCP was founded to recognize licensed physicians who have shown a specialty and interest in regenerative medicine. Increasingly, hospitals and medical staff placement agencies are prioritizing hiring Board-Certified Physicians. For this reason, the AASCP feels it is important to offer qualified professionals a choice when they're researching board certifying bodies.

The American Academy of Stem Cell Physicians (AASCP) is an organization created to advance research and the development of therapeutics in regenerative medicine, including diagnosis, treatment and prevention of disease related to or occurring within the human body. Secondarily, the AASCP aims to serve as an educational resource for physicians, scientists and the public in diseases that can be caused by physiological dysfunction that are ameliorable to medical treatment.

For further information, please contact Wilson Demenessez at 305-891-4686, and you can also visit us at http://www.aascp.net.

Contact Information:Wislon DemenessezzAASCP account Sales managerwilson@genorthix.com305-891-4686

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American Academy of Stem Cell Physicians to Offer Licensed Physicians Board Examination in Regenerative Medicine - BioSpace

Overview

Stem cells have the ability to divide for indefinite periods in culture and give rise to multiple specialized cell types. They can develop into blood, neurons, bone, muscle, skin and other cell types. They have emerged as a major tool for research into the causes of ALS, and in the search of new treatments.

Types of Stem Cells:

The field of stem cell research is progressing rapidly, and The ALS Association is spearheading work on several critical fronts. The research portfolio supports innovative projects using IPSCs for drug development and disease modeling. The Association is supporting an IPSC core at Cedars-Sinai Medical Center providing access to lines for researchers globally. Several of the big data initiatives are collecting skin cells or blood for IPSC generation, such as Genomic Translation for ALS Clinical Care (GTAC), Project MinE, NeuroLINCS and Answer ALS. The ALS Association also sponsors pre-clinical studies and pilot clinical trials using stem cell transplant approaches to develop the necessary tools for stem cell transplant studies and to improve methods for safety and efficiency. We also support studies that involve isolating IPSCs to develop biomarkers for clinical trials through ALS ACT. In addition, the retigabine clinical trial that we sponsor uses iPSCs derived from participants in parallel with clinical data to help test whether the drug has the desired effect.

Stem cells are being used in many laboratories today for research into the causes of and treatments for ALS. Most commonly, researchers use iPSCs to make a unique source of motor neurons from individual ALS patients to try to understand why and how motor neurons die in ALS. Two types of motor neurons are affected in ALS are upper coriticospinal motor neurons, that when damaged, cause muscle spasticity (uncontrolled movement), and lower motor neurons, that when damaged, cause muscle weakness. Both types can be made from iPSCs to cover the range of pathology and symptoms found in ALS. Astrocytes, a type of support cell, called glia, of the central nervous system (CNS), are also being generated from iPSCs. It is well established that glia play a role in disease process and contribute to motor neuron death.

Motor neurons created from iPSCs have many uses. The availability of large numbers of identical neurons, made possible by iPSCs, has dramatically expanded the ability to search for new treatments. For example, they can also be used to screen for drugs that can alter the disease process. Motor neurons derived from iPSCs can be genetically modified to produce colored fluorescent markers that allow clear visualization under a microscope. The health of individual motor neurons can be tracked over time to understand if a test compound has a positive or negative effect.

Because iPSCs can be made from skin samples or blood of any person, researchers have begun to make cell lines derived from dozens of individuals with ALS. One advantage of iPSCs are that they capture a persons exact genetic material and provide an unlimited supply of cells that can be studied in a dish, which is like persons own avatar. Comparing the motor neurons derived from these cells lines allows them to ask what is common, and what is unique, about each case of ALS, leading to further understanding of the disease process. They are also used to correlate patients clinical parameters, such as site of onset and severity with any changes in the same patients motor neurons.

Stem cells may also have a role to play in treating the disease. The most likely application may be to use stem cells or cells derived from them to deliver growth factors or protective molecules to motor neurons in the spinal cord. Clinical trials of such stem cell transplants are in the early stages, but appear to be safe. In addition, transplantation of healthy astrocytes have the potential to be beneficial in supporting motor neurons in the brain and spinal cord.

While the idea of replacing dying motor neurons with new ones derived from stem cells is appealing, using stem cells as a delivery tool to provide trophic factors to motor neurons is a more realistic and feasible approach. The significant challenge to replacing dying motor neurons is making the appropriate connections between muscles and surrounding neurons.

Isolation of IPSCs from people with ALS in clinical trials is extremely valuable for the identification of unique signatures in the presence or absence of a specific treatment approach and as a read out to test whether a drug or test compound has an impact on the health of motor neurons and/or astrocytes. A positive result gives researchers confidence to move forward to more advanced clinical trials. For example, The ALS Association is currently funding a clinical trial to test the effects of retigabine on motor neurons, which use the enrolled patients individual iPSCs lines derived from collected skin samples and testing whether there is a change in the excitability of motor neurons in people with ALS. (see above).

See Stem Cells GrantsSee all Scientific Focus AreasView Glossary of Terms

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Stem Cells - alsa.org

1. Mouse embryo models built from stem cells take shape in a dish  Nature.com
2. Biologists Create a New Type of Human Cells  SciTechDaily
3. A new type of human cell created in the lab  Tech Explorist
4. Biotech firm wants to create human embryos from stem cells and raise them in a 'mechanical womb'  Daily Mail
5. Scientists have created a mechanical womb that can grow life in the lab  Inverse
6. View Full Coverage on Google News

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Mouse embryo models built from stem cells take shape in a dish - Nature.com

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.

Pharmaceutical TechnologyVolume 46, Number 10October 2022Pages: 54-55

When referring to this article, please cite it as C.B. Hartley, "Growth in Cell and Gene Therapy Market," Pharmaceutical Technology 46 (10) 5455 (2022).

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Growth in Cell and Gene Therapy Market - Pharmaceutical Technology Magazine

ELK CITY, Idaho--(BUSINESS WIRE)--Therapeutic Solutions International (TSOI) announced today new data suggesting that therapeutic effects of its universal donor stem cell product are mediated in part through CD103 expressing dendritic cells.

In a series of experiments, it was found that protection against both Chronic Obstructive Pulmonary Disease (COPD) and Acute Respiratory Distress Syndrome (ARDS) could be transferred to nave mice by dendritic cells expressing the molecule CD103. Furthermore, exosomes, which are nanoparticles produced by cells, were capable of transferring protection to nave mice.

I am pleased to have worked with a team of opinion leaders that are at the cutting edge to have discovered this quite unexpected finding, said Dr. James Veltmeyer, Chief Medical Officer of the Company. While the field of exosome therapeutics is growing exponentially, the use of dendritic cell exosomes for respiratory conditions is completely unheard of.

Therapeutic Solutions International is currently running a Phase III clinical trial using JadiCells in the treatment of COVID-19 associated ARDS. Additionally, the Company has an Investigational New Drug Application IND# 28508 for treatment of COPD, for which the Company is still in discussions with the FDA.

Dr. Veltmeyer has performed unparalleled work in advancing both clinical translation of the JadiCell, as well as leveraging scientific lessons learned from our cancer program to identify a new mechanism by which our cells exert this previously unknown therapeutic efficacy, said Timothy Dixon, President, and CEO of the Company. Having filed our patent today on this new finding, we anticipate potential development of adjuvant products around dendritic cell generated exosomes.

About Therapeutic Solutions International, Inc.

Therapeutic Solutions International is focused on immune modulation for the treatment of several specific diseases. The Company's corporate website is http://www.therapeuticsolutionsint.com.

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Therapeutic Solutions International Identifies CD103 Expressing Dendritic Cells and Exosomes Thereof as Novel Mechanism for JadiCell Mesenchymal Stem...