#Cell therapy

[tha-wrecka-stow]

[jcmarchi]

Unlocking mRNA’s cancer-fighting potential

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Unlocking mRNA’s cancer-fighting potential

What if training your immune system to attack cancer cells was as easy as training it to fight Covid-19? Many people believe the technology behind some Covid-19 vaccines, messenger RNA, holds great promise for stimulating immune responses to cancer.

But using messenger RNA, or mRNA, to get the immune system to mount a prolonged and aggressive attack on cancer cells — while leaving healthy cells alone — has been a major challenge.

The MIT spinout Strand Therapeutics is attempting to solve that problem with an advanced class of mRNA molecules that are designed to sense what type of cells they encounter in the body and to express therapeutic proteins only once they have entered diseased cells.

“It’s about finding ways to deal with the signal-to-noise ratio, the signal being expression in the target tissue and the noise being expression in the nontarget tissue,” Strand CEO Jacob Becraft PhD ’19 explains. “Our technology amplifies the signal to express more proteins for longer while at the same time effectively eliminating the mRNA’s off-target expression.”

Strand is set to begin its first clinical trial in April, which is testing a proprietary, self-replicating mRNA molecule’s ability to express immune signals directly from a tumor, eliciting the immune system to attack and kill the tumor cells directly. It’s also being tested as a possible improvement for existing treatments to a number of solid tumors.

As they work to commercialize its early innovations, Strand’s team is continuing to add capabilities to what it calls its “programmable medicines,” improving mRNA molecules’ ability to sense their environment and generate potent, targeted responses where they’re needed most.

“Self-replicating mRNA was the first thing that we pioneered when we were at MIT and in the first couple years at Strand,” Becraft says. “Now we’ve also moved into approaches like circular mRNAs, which allow each molecule of mRNA to express more of a protein for longer, potentially for weeks at a time. And the bigger our cell-type specific datasets become, the better we are at differentiating cell types, which makes these molecules so targeted we can have a higher level of safety at higher doses and create stronger treatments.”

Making mRNA smarter

Becraft got his first taste of MIT as an undergraduate at the University of Illinois when he secured a summer internship in the lab of MIT Institute Professor Bob Langer.

“That’s where I learned how lab research could be translated into spinout companies,” Becraft recalls.

The experience left enough of an impression on Becraft that he returned to MIT the next fall to earn his PhD, where he worked in the Synthetic Biology Center under professor of bioengineering and electrical engineering and computer science Ron Weiss. During that time, he collaborated with postdoc Tasuku Kitada to create genetic “switches” that could control protein expression in cells.

Becraft and Kitada realized their research could be the foundation of a company around 2017 and started spending time in the Martin Trust Center for MIT Entrepreneurship. They also received support from MIT Sandbox and eventually worked with the Technology Licensing Office to establish Strand’s early intellectual property.

“We started by asking, where is the highest unmet need that also allows us to prove out the thesis of this technology? And where will this approach have therapeutic relevance that is a quantum leap forward from what anyone else is doing?” Becraft says. “The first place we looked was oncology.”

People have been working on cancer immunotherapy, which turns a patient’s immune system against cancer cells, for decades. Scientists in the field have developed drugs that produce some remarkable results in patients with aggressive, late-stage cancers. But most next-generation cancer immunotherapies are based on recombinant (lab-made) proteins that are difficult to deliver to specific targets in the body and don’t remain active for long enough to consistently create a durable response.

More recently, companies like Moderna, whose founders also include MIT alumni, have pioneered the use of mRNAs to create proteins in cells. But to date, those mRNA molecules have not been able to change behavior based on the type of cells they enter, and don’t last for very long in the body.

“If you’re trying to engage the immune system with a tumor cell, the mRNA needs to be expressing from the tumor cell itself, and it needs to be expressing over a long period of time,” Becraft says. “Those challenges are hard to overcome with the first generation of mRNA technologies.”

Strand has developed what it calls the world’s first mRNA programming language that allows the company to specify the tissues its mRNAs express proteins in.

“We built a database that says, ‘Here are all of the different cells that the mRNA could be delivered to, and here are all of their microRNA signatures,’ and then we use computational tools and machine learning to differentiate the cells,” Becraft explains. “For instance, I need to make sure that the messenger RNA turns off when it’s in the liver cell, and I need to make sure that it turns on when it’s in a tumor cell or a T-cell.”

Strand also uses techniques like mRNA self-replication to create more durable protein expression and immune responses.

“The first versions of mRNA therapeutics, like the Covid-19 vaccines, just recapitulate how our body’s natural mRNAs work,” Becraft explains. “Natural mRNAs last for a few days, maybe less, and they express a single protein. They have no context-dependent actions. That means wherever the mRNA is delivered, it’s only going to express a molecule for a short period of time. That’s perfect for a vaccine, but it’s much more limiting when you want to create a protein that’s actually engaging in a biological process, like activating an immune response against a tumor that could take many days or weeks.”

Technology with broad potential

Strand’s first clinical trial is targeting solid tumors like melanoma and triple-negative breast cancer. The company is also actively developing mRNA therapies that could be used to treat blood cancers.

“We’ll be expanding into new areas as we continue to de-risk the translation of the science and create new technologies,” Becraft says.

Strand plans to partner with large pharmaceutical companies as well as investors to continue developing drugs. Further down the line, the founders believe future versions of its mRNA therapies could be used to treat a broad range of diseases.

“Our thesis is: amplified expression in specific, programmed target cells for long periods of time,” Becraft says. “That approach can be utilized for [immunotherapies like] CAR T-cell therapy, both in oncology and autoimmune conditions. There are also many diseases that require cell-type specific delivery and expression of proteins in treatment, everything from kidney disease to types of liver disease. We can envision our technology being used for all of that.”

[rodspurethoughts]

FDA Approves Omisirge, a Cell Therapy for Blood Cancer Patients Undergoing Stem Cell Transplantation

The FDA has recently approved a cell therapy called Omisirge (omidubicel-onlv) for patients with blood cancers who are undergoing stem cell transplantation. This allogeneic cord blood-based cell therapy can help speed up the recovery of neutrophils in the body, a type of white blood cell, and reduce the risk of infection. Omisirge is intended for use in adults and pediatric patients 12 years and…

[omshinde5145]

Primary Cells Market Trends, Review, and Forecast 2024–2030

The Primary Cells Market was valued at USD 1.6 billion in 2023 and will surpass USD 3.1 billion by 2030; growing at a CAGR of 10.0% during 2024 - 2030. Primary cells, derived directly from living tissues, maintain the physiological relevance of human biology, making them invaluable in scientific research. Unlike immortalized cell lines, primary cells retain their unique characteristics, providing more accurate models for in vitro studies. This blog explores the key trends, growth drivers, opportunities, and challenges within the primary cells market.

Key Market Trends Driving Growth

Increasing Adoption in Drug Discovery and Development Pharmaceutical companies and research institutions are leveraging primary cells for drug screening and toxicity testing. These cells offer a more accurate prediction of drug responses compared to traditional cell lines. As personalized medicine gains momentum, primary cells enable more individualized and predictive models, allowing researchers to identify specific responses to therapeutic agents.

Advancements in 3D Cell Culture and Organoid Models One of the major trends in the primary cells market is the increasing use of 3D cell culture and organoid models. These advanced culture systems more closely mimic the structure and function of human tissues, offering an enhanced platform for studying disease progression, drug efficacy, and patient-specific therapies. The integration of primary cells into these models is expected to further accelerate research in fields such as oncology, neurology, and regenerative medicine.

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Growing Demand in Cancer Research Primary cells, especially cancer-associated cells such as tumor cells or cancer-associated fibroblasts, are crucial in cancer research. With the rising incidence of cancer, there is a pressing need for more accurate in vitro models that replicate the complex tumor microenvironment. Primary cancer cells, derived directly from patient tissues, are providing researchers with the tools to develop more effective therapies and understand tumor behavior better.

Expansion of Biobanking and Cryopreservation The expansion of biobanks and cryopreservation services is another major factor contributing to the market’s growth. Primary cell biobanks offer vast repositories of cells from diverse human populations, allowing researchers to study genetic variations and disease-specific models. With the increasing emphasis on precision medicine, the demand for high-quality, well-characterized primary cells has surged, enhancing the role of biobanks in supplying these valuable resources.

Opportunities in the Primary Cells Market

Rising Interest in Regenerative Medicine Regenerative medicine is poised to transform the treatment of various degenerative diseases, and primary cells play a key role in this revolution. Stem cells, a type of primary cell, have shown tremendous potential in regenerative therapies for conditions such as heart disease, neurological disorders, and diabetes. The growing pipeline of regenerative therapies represents a lucrative opportunity for companies specializing in primary cell production and related services.

Emerging Markets and Technological Innovations Emerging markets, particularly in Asia-Pacific and Latin America, are becoming attractive for key players in the primary cells market. The increasing healthcare investments, supportive government policies, and growing focus on biotechnology research in these regions are expected to fuel demand for primary cells. Additionally, technological innovations in cell isolation, culture, and cryopreservation techniques are likely to open new avenues for growth in the market.

Partnerships and Collaborations As the complexity of cellular research increases, partnerships between academic institutions, biotech companies, and pharmaceutical firms are becoming more common. Collaborations in areas such as cell sourcing, assay development, and therapeutic applications are enhancing the capabilities of market players. These partnerships are expected to drive innovation and accelerate the adoption of primary cell-based models in various industries.

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Challenges Facing the Primary Cells Market

Limited Availability and High Costs One of the primary challenges in the market is the limited availability of certain types of primary cells, particularly those from rare or difficult-to-access tissues. The cost of isolating, culturing, and maintaining these cells can be prohibitively high, which can restrict their widespread adoption, especially in resource-constrained settings. Additionally, ethical concerns surrounding the sourcing of human tissues remain a challenge that needs to be carefully managed.

Variability and Short Lifespan Unlike immortalized cell lines, primary cells have a finite lifespan, and their characteristics can vary between donors. This variability can introduce challenges in reproducibility and consistency of experimental results, making it difficult to standardize protocols across different labs. While efforts to improve cell culture techniques and reduce variability are ongoing, this remains a significant obstacle for researchers.

Regulatory Hurdles As primary cells are increasingly used in drug development and regenerative therapies, navigating the complex regulatory landscape is becoming a key challenge. Regulatory bodies such as the FDA and EMA require stringent validation of cell-based models, which can delay the approval and commercialization of new therapies. Ensuring compliance with ethical standards for human tissue sourcing and use further complicates the regulatory process.

Conclusion

The primary cells market is poised for robust growth in the coming years, driven by advancements in personalized medicine, drug discovery, and regenerative therapies. The increasing adoption of 3D cell culture systems, expansion of biobanking, and the growing focus on cancer research are key trends shaping the market's future. However, challenges such as high costs, cell variability, and regulatory complexities must be addressed to unlock the full potential of primary cells. As technological innovations continue to emerge and collaborations expand, the primary cells market is set to play a pivotal role in the future of biomedical research and therapeutic development.

[airises]

What are induced pluripotent stem cells, and how are they different from embryonic stem cells?

What are induced pluripotent stem cells (iPSCs)? Reprogrammed adult cells: iPSCs are created in the lab by taking adult cells (often skin or blood cells) and genetically reprogramming them back to an immature, embryo-like state. Pluripotency: Like embryonic stem cells, iPSCs are pluripotent. This means they have the exceptional potential to develop into almost any type of cell in the body. Key…

[regenhealthsolutions]

A micro-fragmented collagen gel as a stem cell-assembling platform for critical limb ischemia repair

Critical limb ischemia is a condition in which the main blood vessels supplying blood to the legs are blocked, causing blood flow to gradually decrease as atherosclerosis progresses in the peripheral arteries. It is a severe form of peripheral artery disease that causes progressive closure of arteries in the lower extremity, leading to the necrosis of the leg tissue and eventual…

[mindfulcells]

Mira's Lymphoma Warrior Journey: Hope and CAR-T Therapy

Estimated reading time: 7 minutes The bold headlines proclaimed the promise of CAR T therapy and its efficacy in combating lymphoma’s relentless grip. Mira – a woman who had weathered the storm of cancer diagnosis, treatment, and remission. She paused to reflect on hope with her life-changing lymphoma warrior journey. As the city’s symphony of car horns echoed outside her window, she stepped…

[dontsweatthefresh]

Danny Brown - Jenn's Terrific Vacation (Official Video)

Goodie Mob - Cell Therapy (Official HD Video)

Kassa Overall: Tiny Desk Concert

[root-analysis]

TIL Cell Therapy Market CAGR | Analysis Size & Forecast (2035) 

The TIL therapy market, a part of the broader cell therapy market, is experiencing significant growth and is projected to grow at a compounded annual growth rate (CAGR) of 40% during the forecast period. The report also provides sales forecasts for TIL therapies market CAGR that are currently in the mid-to-late stages of development. Get a detailed insights report now!

[jcmarchi]

A new way to miniaturize cell production for cancer treatment

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A new way to miniaturize cell production for cancer treatment

Researchers from the Singapore-MIT Alliance for Research and Technology (SMART), MIT’s research enterprise in Singapore, have developed a novel way to produce clinical doses of viable autologous chimeric antigen receptor (CAR) T-cells in a ultra-small automated closed-system microfluidic chip, roughly the size of a pack of cards. 

This is the first time that a microbioreactor is used to produce autologous cell therapy products. Specifically, the new method was successfully used to manufacture and expand CAR-T cells that are as effective as cells produced using existing systems in a smaller footprint and less space, and using fewer seeding cell numbers and cell manufacturing reagents. This could lead to more efficient and affordable methods of scaling-out autologous cell therapy manufacturing, and could even potentially enable point-of-care manufacturing of CAR T-cells outside of a laboratory setting — such as in hospitals and wards.

CAR T-cell therapy manufacturing requires the isolation, activation, genetic modification, and expansion of a patient’s own T-cells to kill tumor cells upon reinfusion into the patient. Despite how cell therapies have revolutionized cancer immunotherapy, with some of the first patients who received autologous cell therapies in remission for more than 10 years, the manufacturing process for CAR-T cells has remained inconsistent, costly, and time-consuming. It can be prone to contamination, subject to human error, and requires seeding cell numbers that are impractical for smaller-scale CAR T-cell production. These challenges create bottlenecks that restrict both the availability and affordability of these therapies despite their effectiveness.

In a paper titled “A high-density microbioreactor process designed for automated point-of-care manufacturing of CAR T cells” published in the journal Nature Biomedical Engineering, SMART researchers detailed their breakthrough: Human primary T-cells can be activated, transduced, and expanded to high densities in a 2-mililiter automated closed-system microfluidic chip to produce over 60 million CAR T-cells from donors with lymphoma, and over 200 million CAR T-cells from healthy donors. The CAR T-cells produced using the microbioreactor are as effective as those produced using conventional methods, but in a smaller footprint and less space, and with fewer resources. This translates to lower cost of goods manufactured (COGM), and potentially to lower costs for patients.

The groundbreaking research was led by members of the Critical Analytics for Manufacturing Personalized-Medicine (CAMP) interdisciplinary research group at SMART. Collaborators include researchers from the Duke-NUS Medical School; the Institute of Molecular and Cell Biology at the Agency for Science, Technology and Research; KK Women’s and Children’s Hospital; and Singapore General Hospital.

“This advancement in cell therapy manufacturing could ultimately offer a point-of-care platform that could substantially increase the number of CAR T-cell production slots, reducing the wait times and cost of goods of these living medicines — making cell therapy more accessible to the masses. The use of scaled-down bioreactors could also aid process optimization studies, including for different cell therapy products,” says Michael Birnbaum, co-lead principal investigator at SMART CAMP, associate professor of biological engineering at MIT, and a co-senior author of the paper.

With high T-cell expansion rates, similar total T-cell numbers could be attained with a shorter culture period in the microbioreactor (seven to eight days) compared to gas-permeable culture plates (12 days), potentially shortening production times by 30-40 percent. The CAR T-cells from both the microfluidic bioreactor and gas-permeable culture plates only showed subtle differences in cell quality. The cells were equally functional in killing leukemia cells when tested in mice.

“This new method suggests that a dramatic miniaturization of current-generation autologous cell therapy production is feasible, with the potential of significantly alleviating manufacturing limitations of CAR T-cell therapy. Such a miniaturization would lay the foundation for point-of-care manufacturing of CAR T-cells and decrease the “good manufacturing practice” (GMP) footprint required for producing cell therapies — which is one of the primary drivers of COGM,” says Wei-Xiang Sin, research scientist at SMART CAMP and first author of the paper.

Notably, the microbioreactor used in the research is a perfusion-based, automated, closed system with the smallest footprint per dose, smallest culture volume and seeding cell number, as well as the highest cell density and level of process control attainable. These microbioreactors — previously only used for microbial and mammalian cell cultures — were originally developed at MIT and have been advanced to commercial production by Millipore Sigma.

The small starting cell numbers required, compared to existing larger automated manufacturing platforms, means that smaller amounts of isolation beads, activation reagents, and lentiviral vectors are required per production run. In addition, smaller volumes of medium are required (at least tenfold lower than larger automated culture systems) owing to the extremely small culture volume (2 milliliters; approximately 100-fold lower than larger automated culture systems) — which contributes to significant reductions in reagent cost. This could benefit patients, especially pediatric patients who have low or insufficient T-cell numbers to produce therapeutic doses of CAR T-cells.

Moving forward, SMART CAMP is working on further engineering sampling and/or analytical systems around the microbioreactor so that CAR-T production can be performed with reduced labor and out of a laboratory setting, potentially facilitating the decentralized bedside manufacturing of CAR T-cells. SMART CAMP is also looking to further optimize the process parameters and culture conditions to improve cell yield and quality for future clinical use.

The research was conducted by SMART and supported by the National Research Foundation Singapore under its Campus for Research Excellence and Technological Enterprise (CREATE) program.

[healthcaretechnologynews]

Cell & Gene Therapy Clinical Trials Market by Phase (Phase I, Phase II), Indication (Oncology, Cardiology) – Global Outlook & Forecast 2023-2031

According to the deep-dive market assessment study by Growth Plus Reports, the global cell & gene therapy clinical trials market was valued at US$ 9.59 billion in 2022 and is expected to register a revenue CAGR of 13.5% to reach US$ 29.69 billion by 2031. 

Cell & Gene Therapy Clinical Market Fundamentals

Cell and gene therapy clinical trials refer to research studies conducted to evaluate the safety, efficacy, and potential applications of cell and gene therapies in human patients. These trials aim to assess the therapeutic benefits and risks associated with these innovative treatment approaches. Clinical trials for cell and gene therapies are typically conducted in multiple phases. In early-phase trials (Phase I and Phase II), the primary focus is on evaluating the safety and tolerability of the therapy, determining the optimal dosage and administration route, and gathering preliminary efficacy data. These trials often involve a small number of participants and closely monitor their responses.

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Cell & Gene Therapy Clinical Market Dynamics

The rising incidence of genetic disorders, chronic diseases such as cancer and cardiovascular diseases, and other conditions with limited treatment options have created a strong demand for innovative therapies like cell & gene therapy. Clinical trials in this field aim to develop effective treatments for these diseases, driving cell & gene therapy clinical trialsmarket growth. According to the WHO, cardiovascular diseases are the main cause of mortality worldwide, accounting for an estimated 17.9 million lives per year, or 32% of all fatalities worldwide. More than 75% of deaths occur in low and middle-income nations. Rapid advancements in biotechnology, gene editing technologies (such as CRISPR-Cas9), and our understanding of genetics have significantly contributed to developing cell and gene therapies. These advancements have enabled scientists to identify target genes and develop precise therapies, leading to increased clinical trials. Several landmark clinical trials have demonstrated the safety and efficacy of cell and gene therapies in treating various diseases. Positive outcomes in trials for diseases like leukemia, lymphoma, and inherited retinal disorders have generated enthusiasm among researchers, clinicians, and patients, leading to increased participation in clinical trials and further driving the cell & gene therapy clinical trialsmarket demand. The field of cell & gene therapy has attracted significant investments and funding from both public and private sources. Pharmaceutical companies, venture capitalists, and government organizations recognize the potential of these therapies and are investing in research and development, infrastructure, and clinical trials, which are also expected to boost the growth of the cell & gene therapy clinical trialsmarket.

However, developing and conducting cell & gene therapy clinical trials can be extremely expensive due to the complexity of these therapies. Costs are associated with research and development, manufacturing, regulatory compliance, and clinical trial operations. These high costs pose a challenge for small biotech companies and academic institutions with limited resources, restricting the growth of gene therapy clinical trials. While regulatory agencies have made efforts to facilitate the development and approval of cell and gene therapies, navigating the regulatory landscape can still be challenging. Meeting regulatory requirements for safety, efficacy, and quality is essential but can involve complex processes and lengthy approval timelines, which is also hindering the growth of the gene therapy clinical trialsmarket. 

Cell & Gene Therapy Clinical Market Ecosystem

The global cell & gene therapy clinical trialsmarket is analyzed from three perspectives: phase, indication, and region. 

Cell & Gene Therapy Clinical Market by Phase

Based on the phases, the global cell & gene therapy clinical trialsmarket is segmented into phase I, phase II, phase III, and phase IV.

The phase II segment accounted for the largest revenue share, with a 51% cell & gene therapy clinical trials market share. Phase II trials aim to assess the efficacy of the therapy in a larger patient population. They provide more extensive data on the therapeutic benefits and effectiveness of the treatment. This phase often involves comparing the therapy to existing standard treatments or placebos, allowing for a more comprehensive evaluation of its efficacy. Phase II trials help refine the dosage and administration protocols of the therapy. The initial Phase I trials provide some insight into dosage levels, but Phase II allows for a more systematic exploration of different doses and administration schedules to identify the optimal therapeutic regimen. Phase II trials often involve a larger number of patients, allowing for better selection and inclusion of a diverse patient population. This enables researchers to assess the therapy's efficacy in different subgroups and evaluate its potential benefits across a broader range of patients. Regulatory agencies typically require data from well-designed Phase II trials to support the advancement of therapies to Phase III and subsequent stages. The data collected from Phase II trials are crucial for demonstrating the therapy's efficacy and safety profile, supporting regulatory submissions, and obtaining further approvals for larger-scale trials. Positive results from Phase II trials often generate significant interest from investors, as they indicate the therapy's potential for success. Promising efficacy and safety data from Phase II trials can attract funding and partnerships for further development and commercialization of the therapy, driving market dominance in this phase.

Cell & Gene Therapy Clinical Market by Indication 

Based on the indications, the global cell & gene therapy clinical trialsmarket is segmented into oncology, cardiology, CNS, musculoskeletal, infectious diseases, immunology & inflammation, ophthalmology, dermatology, endocrine, metabolic, genetic, hematology, gastroenterology, and others.

The oncology segment accounted for the prominent cell & gene therapy clinical trials market share in 2022, with a 45% market share. Oncology represents a significant unmet medical need, with a wide range of cancers having limited treatment options.  According to GLOBOCAN 2020 report, approximately 10 million deaths were cases by cancer in 2020.  Cell and gene therapies offer potential breakthroughs in cancer treatment by targeting specific genetic alterations or enhancing the immune system's ability to recognize and eliminate cancer cells. The urgent need for effective cancer treatments drives the focus on oncology in clinical trials. Oncology has been a well-established area of research and development for many years. This has led to a deeper understanding of cancer biology, genetic alterations, and immune responses in the context of cancer. The infrastructure and expertise required for conducting clinical trials in oncology are well-established. Oncology research centers, academic institutions, and specialized hospitals often have the necessary infrastructure, multidisciplinary teams, and patient populations available to conduct cell & gene therapy clinical trials. The market potential for oncology treatments is significant, given the high prevalence of cancer and the increasing demand for more effective therapies. Successful cell and gene therapies in oncology have the potential for commercial success, attracting investments from both pharmaceutical companies and venture capitalists. This market potential further drives the growth of the oncology segment in the cell & gene therapy clinical trials market.

Cell & Gene Therapy Clinical Market by Region

Geographically, the global cell & gene therapy clinical trials market has been segmented into North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa. 

The North America region has the largest cell & gene therapy clinical trials market size in terms of revenue generation accounting for around 46.2% share of the market. North America boasts a strong biotechnology and pharmaceutical industry, with numerous research institutions, academic centers, and biotech companies at the forefront of cell & gene therapy development. These entities contribute to the discovery, development, and commercialization of innovative therapies, driving the growth of clinical trials in the region. North America has a well-established, advanced healthcare infrastructure, including specialized treatment centers, research institutions, and clinical trial networks. This infrastructure supports the conduct of clinical trials by providing access to patient populations, expert clinicians, and specialized facilities required to deliver cell and gene therapies. North America is a hub for biotech and venture capital investments, attracting significant funding for cell & gene therapy research and development. Academic institutions, government agencies, and private investors provide financial support to advance clinical trials in the region. North America fosters a collaborative environment, with academic institutions, biotech companies, and research organizations working together on cell & gene therapy projects. Collaborations between academia and industry and partnerships between different stakeholders facilitate knowledge sharing, access to resources, and the progression of clinical trials.

Cell & Gene Therapy Clinical Market Competitive Landscape

The prominent players operating in the global cell & gene therapy clinical trials market are:

ICON Plc

IQVIA

Charles River Laboratories International, Inc.

Laboratory Corporation of America Holdings

Syneos Health

PAREXEL International Corp.

Medpace Holdings, Inc.

PPD Inc.

Novotech

Veristat, LLC

Cell & Gene Therapy Clinical Market Strategic Developments

In June 2023, Arrowhead Pharmaceuticals submitted an application seeking approval to initiate a Phase I clinical trial of ARO-SOD1 to treat amyotrophic lateral sclerosis (ALS) harbouring superoxide dismutase 1 (SOD1) mutations. In compliance with the Australian Department of Health and Ageing's Therapeutic Goods Administration's clinical trial notification process, the company filed the application to an ethics committee. The RNAi-based experimental drug ARO-SOD1 is being tested in adults with ALS with SOD1 mutations in a dose-escalation, placebo-controlled, randomized research.

In June 2023, Beacon Therapeutics entered into the gene therapy field with a $120m Series A financing. The new ocular gene therapy firm was founded by integrating Applied Genetic Technologies Corporation's (AGTC) late-stage X-linked retinitis pigmentosa (XLRP) program with two unique preclinical studies. The finance includes AGTC's acquisition and funds to help speed Beacon Therapeutics' candidate development, with participation from Oxford Science Enterprises (OSE). The total amount of funding was £96 million ($120 million).

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[jupiterlaser]

Thing People Get Wrong About Benefits of Stem Cell Therapy

You may find yourself in an emotionally charged situation if you engage yourself in a discussion related to stem cell. While the controversy about stem cell therapy will cross the religious and political beliefs, it is controversial topic of discussion for everybody.

Due to personal biasness, much of the contention is the result of misleading and incorrect information. While the clinical research that was necessary to advance this field of medicine, personal biasness has slowed it down.

When researchers discovered that cells were building blocks of life, cell research started in the mid — 1880s. Doctors had performed 200 allogeneic stem cell transplants in human by 1950s and 60s without any success.

While treating immune deficiency through bone marrow transplant, stems cells in the late 1960s were also used for treatment of leukemia and aplastic anemia.

It is seen that there are some myths and facts related to stem cell, which are discussed in this article. Let us dig it in.

Stem Cells Are Taken from Embryos

True/False

It is said that practices, and clinics do use embryonic stem cells for their procedures. When it comes to medical injection therapy, it is not the case. It is seen that you can classify stem cell into two categorise viz: embryonic and adult cell.

It is seen that doctors can easily replicate the stem cells into other types of cells, which they use through the adult mesenchymal cells. These cells are very undifferentiated with the normal adult cells.

Before injecting to the affected site, these adults stem cells are taken from the patient’s stem cell or adipose tissue. Doctors can use platelet rich plasma injections for curing the condition.

Stem Cell Is Unsafe

False

If you go to an unreliable, and unauthorized clinic, you will know that doctors are providing unsafe stem cell therapy. It is seen that doctors will not provide such improper practices, as you do not have worry about it.

It is the trained team of doctors, who will perform such therapies. These doctors will take an autologous approach so that you do not get a transmitted infectious disease. While doctors try to make it risk free, you will know that the therapy is minimally invasive.

After analyzing the medical history of patients, doctors will go ahead with the injection therapy. Before administering the injection therapy, they will brief you about the process.

Therapy is Prone to Body Rejection

False

There is no chance of body rejecting the injected cells, as stem cells is autologous. While there are chances of elimination of chances of rejection which is a cause of concern in donor transplant, your immune system would not treat them as a foreign invasion.

While assisting the degenerative alignments, and condition, the stem cells will stimulate the body’s natural healing response. It will provide a long — lasting and quick solution.

Therapy Will Cure Several Aliments

True

Doctors can treat several illnesses, and medical conditions, as it has become possible with stem cell. While used for the healing sports related injuries like bruised tendons, torn ligaments and muscle pain, the therapy is very useful for the musculoskeletal injuries.

The therapy is also very effective for the osteoarthritis. It is seen stem cell therapy will promote cartilage regeneration.

Re Adult Stem Cells as Good — Or Better — Than Embryonic Stem Cells?

While helping for future therapies, adult stem cells have great potential and are extremely valuable. While adult stem cells can only follow certain paths, embryonic cells can grow virtually into any cell type in the body.

While the embryonic stem cells are not so flexible in treating all types of diseases, adult stem cells do not grow indefinitely in the labs.

Final Thoughts

There are lots of benefits for stem cell. You should not remain in notion that stem cell is a dangerous therapy for your disease.