Regenerative Medicine Market Investments, Share and Revenue Analysis | Latest InsightAce Report – Yahoo Finance

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The global Regenerative Medicine market is estimated to reach over USD 183.08 billion by 2031, exhibiting a CAGR of 15.02% during the forecast period

Jersey City, NJ, April 12, 2023 (GLOBE NEWSWIRE) -- InsightAce Analytic Pvt. Ltd. announces the release of a market assessment report on the "GlobalRegenerative Medicine Market Size, Share & Trends Analysis Report By Product (Therapeutics, Primary cell-based therapeutics, Stem Cell & Progenitor Cell-based therapeutics), By Therapeutic Category (Dermatology, Musculoskeletal, Immunology & Inflammation, and Oncology)- Market Outlook And Industry Analysis 2031"

The global Regenerative Medicine market is estimated to reach over USD 183.08 billion by 2031, exhibiting a CAGR of 15.02% during the forecast period.

In recent year, it has been determined that regenerative therapies can uniquely change the underlying pathological processes. Trial-stage regenerative medicines offer promising treatments for particular chronic diseases with unmet medical needs. Novartis announced the release of T-ChargeTM in December 2021, a next-generation CAR-T platform that would be used for cutting-edge investigational CAR-T cell treatments.

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The development of gene-based treatment, which uses targeted DNA delivery as a drug to combat numerous illnesses, results from significant effects in molecular therapeutics. With the restoration of gene function, gene therapy holds great promise for treating cancer and type 1 and type 2 diabetes. Gene-based medicines treat patients with conditions such as cancer, oncology, infectious diseases, cardiovascular disorders, monogenic diseases, genetic disorders, ophthalmological indications, and central nervous system illnesses. These elements have helped the market for regenerative medicine expand.

Recent Developments:

In April 2022, Obecabatagene autoleucel, a CD19-directed autologous chimeric antigen receptor T therapy being investigated in the ongoing FELIX Phase 2 study of leukaemia, has been given the Regenerative Medicine Advanced Therapy designation by the U.S. Food and Drug Administration (FDA). This was announced by Autolus Therapeutics plc.

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List of Prominent Players in the Regenerative Medicine Market:

AstraZeneca plc;

F. Hoffmann-La Roche Ltd.;

Integra Lifesciences Corp.;

Astellas Pharma, Inc.;

Cook Biotech, Inc.;

Bayer AG;

Pfizer, Inc.;

Merck KGaA;

Abbott;

Vericel Corp.;

Novartis AG;

GlaxoSmithKline (GSK);

Baxter International, Inc.;

Boehringer Ingelheim;

Amgen, Inc.;

Cesca Therapeutics, Inc.;

U.S. Stem Cell, Inc.;

Bristol-Myers Squibb;

Eli Lilly and Company;

NuVasive, Inc.;

Organogenesis, Inc.;

MiMedx Group, Inc.;

Takara Bio, Inc.;

Osiris Therapeutics, Inc.;

Corline Biomedical AB

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Regenerative Medicine Market Report Scope:

Report Attribute

Specifications

Market size value in 2022

USD 52.66 Bn

Revenue forecast in 2031

USD 183.08 Bn

Growth rate CAGR

CAGR of 15.02 % from 2023 to 2031

Quantitative units

Representation of revenue in US$ Million, and CAGR from 2023 to 2031

Historic Year

2019 to 2022

Forecast Year

2023-2031

Report coverage

The forecast of revenue, the position of the company, the competitive market statistics, growth prospects, and trends

Segments covered

Product And Therapeutic Category

Regional scope

North America; Europe; Asia Pacific; Latin America; Middle East & Africa

Country scope

U.S.; Canada; U.K.; Germany; China; India; Japan; Brazil; Mexico; The UK; France; Italy; Spain; China; Japan; India; South Korea; Southeast Asia; South Korea; Southeast Asia

Market Dynamics:

Drivers- The ability of adult stem cells to proliferate or self-renew forever and to develop all the cell kinds of the organ from which they originate has propelled research into these cells, with the potential to regenerate the complete organ from a few cells. No embryo must be destroyed in order to produce adult stem cells. Furthermore, medical research on stem cells has been thoroughly examined and attracted much attention. ExCellThera Inc. and Ossium Health recently announced a partnership to explore and advance opportunities to use adult stem cells from deceased donors from Ossium Health's first-ever bone marrow bank in combination with ExCellThera's ECT-001 cell expansion and rejuvenation technology. This collaboration will take place in April 2021. These kinds of developments are anticipated to accelerate market expansion.

Challenges:The market for regenerative medicine is projected to be hampered by a lack of information and moral considerations surrounding the usage of embryonic stem cells for research and development. Since cell therapy is a crucial component of regenerative medicine, it has a significant impact on the market growth rate. One of the leading market inhibitors may be the high cost of investment, which might be followed by problems with assay sensitivity, robustness, and reproducibility; the challenge of culture/propagation; and finally, the challenge of handling.

Regional Trends:Due to the presence of big players, the rapid advancement of technology, significant investments in stem cell and oncology research, and the presence of major players, North America is predicted to have the largest revenue share. The largest market in North America is the United States. In the U.S., numerous stem cell therapies are increasingly being used to treat a growing number of ailments like cancer and diabetes. According to the Heart Disease & Stroke Statistics Fact Sheet 2020, congenital heart abnormalities are predicted to affect at least 40,000 infants annually in the United States.

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Segmentation of Regenerative Medicine Market-

By Product-

Therapeutics

Primary cell-based therapeutics

Dermatology

Musculoskeletal

Surgical

Dental

Others

Stem Cell & Progenitor Cell-based therapeutics

Autologous

Allogenic

Others

Cell-based Immunotherapies

Gene Therapies

Tools

Banks

Services

By Therapeutic Category-

By Region-

North America-

Europe-

Germany

The UK

France

Italy

Spain

Rest of Europe

Asia-Pacific-

China

Japan

India

South Korea

South East Asia

Rest of Asia Pacific

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Regenerative Medicine Market Investments, Share and Revenue Analysis | Latest InsightAce Report - Yahoo Finance

FDA Grants FTD to Off-the-Shelf CAR T in R/R Multiple Myeloma – Cancer Network

The FDA has granted fast track designation to the investigational allogenic CAR T-cell therapy product CB-011 for the treatment of patients with relapsed or refractory multiple myeloma, according to a press release from Caribou Biosciences, Inc.1

"Fast track designation for CB-011 allows us instrumental interactions with the FDA as we progress our clinical development and regulatory plans for CB-011, according to the manufacturers of CB-011.

The FDA originally cleared an investigational new drug application for CB-011 in this patient population in November 2022, permitting investigators to advance with their evaluation of the agent in the phase 1 CaMMouflage trial (NCT05722418).2

According to findings from a poster session presented at the 2023 Tandem Meeting, CB-011 demonstrated potent anti-tumor activity in vitro and enhanced survival in multiple myeloma xenograft models.3 Additionally, investigators observed no adverse safety signals associated with treatment in vitro.

Fast track designation for CB-011 allows us instrumental interactions with the FDA as we progress our clinical development and regulatory plans for CB-011, Syed Rizvi, MD, chief medical officer of Caribou, said in the press release. Our goal is to develop CB-011 as a readily available off-the-shelf treatment option for patients with relapsed or refractory multiple myeloma to overcome the need for apheresis or bridging therapy, variable quality and long manufacturing timelines, manufacturing failures, or the inability to bear the burden of treatments that require frequent dosing over several months.

CB-011 is an allogeneic CAR T-cell therapy targeting BCMA that was engineered with Cas12a chRDNA technology. Investigators believe its design allows it to enable anti-tumor activity through an immune cloaking strategy that removes the B2M protein and inserts a B2MHLA-E fusion protein.

Investigators of the open-label, multi-center phase 1 CaMMouflage trial are assessing CB-011 as treatment for adult patients with relapsed or refractory multiple myeloma. Part A will include a dose escalation of CB-011 in ascending doses based on a traditional 3+3 design. In part B, up to 30 patients will receive CB-011 at the maximum tolerated dose or recommended phase 2 dose determined in part A.

The primary end point of part A is the number of patients who experience dose limiting toxicities. In part B, the primary end point is the overall response rate based on International Myeloma Working Group (IMWG) criteria.

Patients 18 years and older with a documented diagnosis of relapsed or refractory multiple myeloma and measurable disease per IMWG criteria are eligible to enroll on the trial. Additional inclusion criteria include receipt of at least 3 prior lines of therapy including a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 monoclonal antibody; having an ECOG performance status of 0 or 1; and adequate hematologic, hepatic, renal, pulmonary, and cardiac function.

Patients who received prior treatment with a CAR T-cell therapy or autologous stem cell transplant within 6 weeks prior to undergoing lymphodepletion are not eligible for enrollment. Patients are also unsuitable for enrollment if they have received allogeneic stem cell transplant within 6 months prior to lymphodepletion, known active or prior central nervous system involvement, stroke or seizure within 6 months of study entry, or are seropositive or have a history of human immunodeficiency virus.

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FDA Grants FTD to Off-the-Shelf CAR T in R/R Multiple Myeloma - Cancer Network

NSF CAREER Award: Rowan researcher takes aim at critical barrier … – Rowan Today

In humans, mesenchymal stem cells (MSCs) live in bone marrow and body fat. Their identity can be defined by their ability to divide into more stem cells, adapt to new environments and differentiate into specialized cells that produce tissues like bone, ligament, cartilage and fat.

When studied in the lab, these unique properties decline over time. The inability to produce large quantities of MSCs with their identity intact is a critical barrier that limits the use of these cells for reproducible research and stem cell therapies.

Sebastin L. Vega, Ph.D., an assistant professor of biomedical engineering at Rowan University, has received a five-year, $512,890 CAREER Award from the National Science Foundation (NSF) to investigate why stem cells lose their identity outside the body. The Faculty Early Career Development (CAREER) Program offers the independent federal agencys most prestigious awards to early-career faculty. Vega has dual appointments in the Henry M. Rowan College of Engineering and the Rowan-Virtua School of Translational Biomedical Engineering & Sciences, where he is also a founding faculty member of the Rowan-Virtua Institute of Regenerative Medicine and Transplantation.

Using stem cells, scaffolds and environments to grow functional tissuea replacement of a persons own tissueis the future, with treatment strategies beginning to be available to patients now, said Mark Byrne, Ph.D., founding dean of the Rowan-Virtua School of Translational Biomedical Engineering & Sciences. Dr. Vegas research is critical to improving and scaling stem cell therapies, which lead to much better outcomes for patients. At Rowan, we actively recruit top researchers like Dr. Vega, who have high potential to lead next-generation treatment strategies.

This award recognizes Dr. Vegas exceptional research and the promise of future breakthroughs associated with stem cell manufacturing, said Giuseppe Palmese, Ph.D., dean of the Henry M. Rowan College of Engineering. We are delighted to have Dr. Vega represent our college in this way.

Funding from Vegas CAREER Award will be used to develop materials with independently controllable properties to understand the role of stiffness, adhesion and the interactions between cells on MSC identity.

MSCs are typically grown in plastic petri dishes, which are stiffer than any tissue in the human body, Vega said. MSCs remember their culture history and prolonged exposure to unnatural materials like plastic negatively impacts their ability to divide, adapt and differentiate.

Instead of relying on traditional petri dishes for culturing cells, Vega is designing a replacementsofter, bioactive materials meant to preserve the cells identities even after countless cell divisions in the lab. The goal is to create materials that can be used to culture large populations of high-quality stem cells for research and clinical use.

Another unwanted side effect of prolonged exposure of these cells to plastic is a loss of cell-material communication, which is necessary for taking on the properties of specialized cell types theyre meant to become. The team will develop new culture environments with prescribed properties to significantly improve MSC adaptation to biomaterials used in regenerative medicine.

Despite being discovered over half a century ago and having become one of the most-studied cell types in regenerative medicine, FDA-approved MSC therapies do not exist, Vega said. Our proposed research will help overcome cell manufacturing challenges seen in many MSC clinical trials and I am hopeful that our studies will enable the clinical use of MSCs in tissue engineering and to treat degenerative diseases.

This award will also integrate research with ongoing educational outreach activities. Vega is the founder of BEAM (BioEngineering and Me), a program that exposes high school students to biomedical engineering. Through this program, Glassboro High School students learn about biomedical engineering through research talks and interactive activities in research labs. To reach more students, Vega developed the annual BEAM Summer Program, a free, two-week summer program open to any high school student nationwide. To date, 74 high school students from nine states have participated.

Vega also runs the RISER (Research Immersion in biomedical Science & Engineering at Rowan) Summer Program, a six-week research experience for high school students to join biomedical engineering or biomedical science labs, which culminates with student presentations at the annual RISER Summer Symposium.

CAREER awards from the National Science Foundation support early career researcher educators in attaining their long-term goals, said NSF program director Wendy Crone. Dr. Vega's project on regulating stem cell proliferation, mechanical adaptation, and differentiation is poised to make advancements in the ability to preserve stem cell identity while also engaging high school and undergraduate students with biotechnology research.

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NSF CAREER Award: Rowan researcher takes aim at critical barrier ... - Rowan Today

Germany Cell and Gene Therapy Market Focused Insights Report 2023: A $3.44 Billion Market by 2028 – Competitive Landscape, Pipeline Analysis, Clinical…

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Dublin, April 12, 2023 (GLOBE NEWSWIRE) -- The "Germany Cell and Gene Therapy Market - Focused Insights 2023-2028" report has been added to ResearchAndMarkets.com's offering.

The Germany cell and gene therapy market was valued at $0.34 billion in 2022 and is projected to reach at $3.44 billion in 2028, with a compound annual growth rate (CAGR) of 40.15%, during the study period.

The study considers a detailed scenario of the present cell and gene therapy market and its market dynamics for 2023-2028 in Germany. It covers a detailed overview of several market growth enablers, restraints, and trends. The report offers both the demand and supply aspects of the market. It profiles and examines leading companies and other prominent players operating in the market.

This report offers market size & forecast data for cell & gene therapy market in Germany. The report covers commercial cell & gene therapy products, such as conventional cell therapies, CAR-T cell therapies, gene therapies, cell-based immunotherapies, and oncolytic virus therapies.

Cell therapies sourced from mesenchymal stem cells, cell-based immune modulation therapies, and cell/tissue-based products derived from patients' blood are also covered in the report. Tissue-engineered products and tissue grafts/scaffolds with synthetic mechanical structures are excluded from the scope of the report.

This report provides a comprehensive and current market scenario of the Germany cell & gene therapy market, including the Germany cell & gene therapy market size, anticipated market forecast, relevant market segmentations, and industry trends.

MARKET DEFINITION

Cell and gene therapies are part of advanced therapy medicinal products (ATMPs). ATMPs are medicines that are developed based on genes, tissues and cells.

The introduction of many novel ATMPs in recent years, especially cell and gene therapies changed the treatment dynamics of many complex and challenging diseases like cancer and genetic disorders.

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Government, as well as industrial and academic financing and collaboration among small and large biopharmaceutical companies, offer significant growth to the market in the coming years.

KEY HIGHLIGHTS

Among all commercial cell & gene therapies, Zolgensma is the first and only product to achieve the blockbuster drug tag. Zolgensma generated a revenue of $1.37 billion in 2022 and has the potential to reach around $5.00 billion by 2026.

Drug developers are prioritizing to develop and commercialize CAR-T cell-based gene therapies. Globally, more than 1,000 clinical trials are being conducted on CAR T-cell therapies, of which atleast 500 clinical trials are for cancer alone. Kymriah (Novartis), Yescarta (Gilead Sciences), Tecartus (Gilead Sciences), Breyanzi (Bristol Myers Squibb), Abecma (Bristol Myers Squibb) and Carvykti (Janssen Biotech / Legend Biotech) are the commercial CAR-T therapies available in the market.

In 2022, Germany was the first-largest market in Europe, followed by France and the UK. The market is majorly driven by an increase in target patient population, regulatory body support, and higher access to healthcare due to well-established healthcare infrastructure.

The European Medicines Agency (EMA) has approved more than nineteen cell and gene therapy drugs, but the new product pipeline has approximately 193 investigational therapies, with more than half of these in Phase 2 clinical trials. Oncology and rare diseases continue to be the top areas targeted by gene therapies from preclinical through pre-registration.

Approximately 261 clinical trials are under investigation for various cell and gene therapies in Germany. This increase could be because of an improvement in funding for cell and gene therapy. The industry sponsored trials are continued to dominate with a share of 88.51%.

Novartis, Gilead Sciences, Spark Therapeutics, Amgen, Orchard Therapeutics, Bristol-Myers Squibb, BioMarin, PTC Therapeutics, and Organogenesis are the leading players in the Europe cell and gene therapy market. The market offers tremendous growth opportunities for existing and future/emerging players because of the presence of a large pool of target patient populations with chronic diseases such as cancer, genetic and rare diseases, and other complex disorders.

PRODUCT SEGMENTATION & FORECAST

Product type

Application type

Oncology

Genetic Disorders

Dermatology

Musculoskeletal

Others

End-user type

Hospitals

Cancer Care Centers

Others

MARKET STRUCTURE

PRICING & REIMBURSEMENT SCENARIO

Pricing, Reimbursement and Market Access

Contract Manufacturing Organizations

Regulatory Landscape and Policies

For more information about this report visit https://www.researchandmarkets.com/r/ppr7fp

About ResearchAndMarkets.comResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

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Germany Cell and Gene Therapy Market Focused Insights Report 2023: A $3.44 Billion Market by 2028 - Competitive Landscape, Pipeline Analysis, Clinical...

How can stem cells treat diabetes? The Upcoming – The Upcoming

How can stem cells treat diabetes?

Diabetes is a persistent medical condition that impairs the bodys capacity to regulate blood sugar levels. Although various treatments and medications are available to mitigate the symptoms and avoid complications, the disease has no known cure. It is caused by an autoimmune response that targets and destroys pancreatic (beta) cells, which are responsible for producing insulin.

Since the body fails to maintain blood sugar levels on its own, people with diabetes require insulin injections. This is currently the only option for patients suffering from type 1 diabetes.

According to research, stem cell therapy has been shown to restore the function of insulin-producing beta cells in the pancreas potentially, thus helping treat this problem. In this article, this will be discussed.

Stem cell therapy for diabetes

The cell-based approach is a promising treatment for diabetes as it can help in the regeneration of damaged pancreatic cells that produce insulin. This factor makes stem cell therapy particularly important for patients with type 1 diabetes, considering their permanent dependency on insulin injections to control blood glucose levels. Lets look at the types of the disorder and how stem cells are helpful in minimising their harmful effects.

What is type 1 diabetes?

This autoimmune disease is characterised by the immune system attacking and destroying insulin-producing beta cells in the pancreas. Insulin controls the blood glycaemic index by allowing glucose to enter the bodys cells and use it for energy and storage. Insufficient amounts of insulin result in a rise in glucose levels in the bloodstream, leading to high blood sugar levels, which can cause various symptoms and complications.

The symptoms of the disorder include:

Type 1 diabetes usually develops in childhood or young adulthood but can occur at any age. It is often diagnosed through a blood test that measures blood sugar levels and the presence of certain autoantibodies such as:

Treatment for type 1 diabetes typically involves lifelong insulin therapy, monitoring blood sugar levels, and lifestyle modifications with a proper workout plan and dietary alterations.

What is type 2 diabetes?

Non-insulin-dependent diabetes, also known as diabetes type 2, is a chronic condition that affects how the body processes blood sugar (glucose). In type 2 variation, the body either doesnt produce enough insulin or becomes resistant to insulin, a hormone that regulates the blood glycemic index. After food consumption, the body converts carbohydrates into glucose, which is then transported into cells by insulin. However, in people with type 2 variation, cells in fat, muscles, and liver cannot take in enough glucose, leading to a buildup of it in the bloodstream.

Over time, high blood sugar levels can cause serious health problems, including damage to the heart, blood vessels, nerves, eyes, and kidneys. Some risk factors that may lead to the disorder include:

Old age is also one of the factors which may lead to the onset of type 2 diabetes.

Is there a cure for diabetes?

There is currently no known cure for this disease. Diabetes type 2 occurs by a combination of lifestyle and genetic factors, such as being overweight or inactive. People with type 2 can often manage their condition through a healthy diet and exercising regularly but may also need medications, insulin, or other treatments to help manage blood sugar levels.

While there is no cure for this disorder, ongoing research is focused on developing new treatments and therapies that can improve management and prevent complications. People suffering from this condition are advised to work with their healthcare providers to develop a personalised treatment plan that fits their individual needs and goals.

The International Diabetes Federation predicts that by 2045, nearly 700 million adults will have diabetes. The cause, specifically of type 1 diabetes (T1D), is not fully understood, but research suggests a combination of genetic, environmental, and viral factors may contribute to its development. Currently, the most widely used treatment for T1D is administering insulin externally, but it does not provide a cure for the disease.

The Role of Mesenchymal Stem Cells in the Treatment of Type 1 Diabetes, doi: 10.7759/cureus.27337.

Can stem cells help with diabetes?

A potential treatment proposed to cure the disorder is stem cell therapy. However, its effectiveness and safety are still being researched and debated in the scientific community, i.e., can stem cells cure diabetes?

Much work is being done to use stem cell for diabetes type 2 and metabolically programming them to create glucose-sensing, insulin-producing beta cells in the body. Some researchers are exploring the possibility of using stem cells as replacements for these, which would allow the body to produce insulin again.

The bodys immune system resulting in the destruction of insulin-producing cells in the pancreas causes type 1 diabetes. Scientists working on stem cells observed that they could help people with T1D better control their blood sugar levels. Several clinical trials are ongoing to study the safety and effectiveness of stem cell therapy for diabetes, with varying degrees of success.

We now understand stem cell transplants can succeed in treating diabetes for some. We discovered the immune signature predicting these outcomes either favourable or not which is the first step toward personalised medicine in type 1 diabetes. Understanding why it sometimes fails will allow us to design new treatment strategies for those less fortunate patients. Also, it is the first definitive proof that T1D can be cured.

Bart Roep, PhD, Professor of Diabetology.

Stem cells can be derived from various sources, including induced pluripotent, embryonic, and adult versions. However, each source has its advantages and limitations regarding availability, differentiation potential, and ethical considerations.

Benefits and disadvantages of stem cell therapy

Like any medical treatment, cell therapy for diabetes has both pros and cons. The disorder can cause several complications, including cardiovascular disease, neuropathy, and retinopathy. Stem cell therapy may reduce the risk of these complications by repairing and regenerating damaged cells in the body. Transplanted stem cells cures diabetes to some extent by potentially recreating insulin-producing pancreatic islets. It has the potential to manage the disease by regenerating damaged cells of the pancreas and thus restoring the bodys ability to produce and regulate insulin. It is a natural way of healing from the inside of the body.

Disadvantages of stem cell therapy for the disorder include its safety. There is a risk of complications, such as infection, and immune system reactions, in case the cell-based product was not properly prepared. In addition, stem cell therapy is an expensive treatment, and it may not be covered by health insurance and thus can be a significant barrier for some patients. The use of embryonic stem cells for therapy raises ethical concerns for some people. In this regard, adult mesenchymal stem cells are a better option for therapy.

Final thoughts

In conclusion, cell therapy has the potential to revolutionise the treatment of diabetes. Still, more research is needed to fully understand its benefits and risks and develop safe and effective protocols for its clinical application. It is essential to weigh stem cell therapys potential benefits and disadvantages carefully and consult with a healthcare professional before making any decisions about treatment.

The editorial unit

The information contained in this article is for general informational purposes only and is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of a qualified healthcare provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read.

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How can stem cells treat diabetes? The Upcoming - The Upcoming

Brave girl, 4, rings cancer-free bell after ending three years of treatment – Yahoo News UK

Watch: Brave girl, four, rings bell after ending three years of cancer treatment

This is the moment a brave four-year-old girl rang the cancer-free bell after ending three years of treatment.

A video showed Phoebe Ashfield receiving a round of applause and cheers from nurses.

She was diagnosed with acute lymphoblastic leukaemia at seven months old in 2019 and relapsed three times during her treatment.

Her mother Emma Wyke, 30, from Dudley, West Midlands, recorded Phoebe ringing the bell following her ordeal.

Read more: Spot on womans finger turned out to be false widow spider bite

Mother Emma Wyke and Phoebe (centre) alongside family members. (Caters)

Wyke said: "At one stage of her treatment I was told that she isn't going to pull through and to prepare myself for the worst.

"It's devastating and heartbreaking to watch my baby girl go through all this.

Read more: Boy, 9, nearly dies after falling eight foot off scooter

"When Phoebe was first diagnosed, I thought they had the wrong child, and it simply cannot be my daughter, as all she had was a cold and chest infection and it can't be cancer.

"When she finally rang the bell it's a mixture of emotions you want to cry happy tears, but you still have that worry of wondering if it's going to come back. You don't truly know how to feel."

Phoebe as a baby during her treatment. (Caters)

Phoebe started her treatment with chemotherapy but it wasn't strong enough to keep the cancer away, Wyke said.

She relapsed in June 2019 and had to go for a Car-T therapy, which involved taking her stem cells and modifying them to fight the cancer.

She relapsed again in September of that year.

Her mother said further treatment was successful until January 2020 when she relapsed for the third time and needed a stem cell transplant, and at this moment "time was against us".

Wyke added: "If there is one thing to come of this is that to register to become a stem cell donor through (non-profit) DKMS and (charity) Anthony Nolan because without these selfless people, adults and children, my daughter wouldn't be here to tell this tale.

If you can please register you could save someone's life."

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Brave girl, 4, rings cancer-free bell after ending three years of treatment - Yahoo News UK

Bluebird responds to FDA on sickle cell gene therapy’s production… – Sickle Cell Disease News

Bluebird Bio has provided the U.S. Food and Drug Administration (FDA) with information regarding the commercial manufacturing of its experimental gene therapy for sickle cell disease (SCD) in preparation for requesting its approval.

The company had expected to submit a biologics license application seeking the approval of the therapy, lovotibeglogene autotemcel or lovo-cel, to the FDA earlier this year.

In February, however, the agency asked for more information to ensure production processes for the commercial use of lovo-cel are comparable to those used in clinical testing. Because the treatment uses the patients own stem cells, which are altered and then infused back to the patient, the complex manufacturing process is a key aspect to take into consideration.

Bluebird now has responded to FDA feedback on manufacture comparability referred to as chemistry, manufacturing, and controls (CMC) comparability and is expecting a response within a matter of weeks, according to a company press release.

The company also plans to request priority review of lovo-cel for SCD patients, ages 12 and older, with a history of vaso-occlusive crisis (VOC), a painful SCD complication. Priority review works to quicken an FDA approval decision.

We remain laser focused on our lovo-cel BLA for sickle cell disease, said Andrew Obenshain, Bluebirds CEO. Following feedback from the FDA in February, bluebird submitted additional information related to CMC comparability analyses to the FDA in early March.

Most importantly, we know that patients and their families are waiting, and we will move quickly to expedite our BLA submission, Obenshain said.

SCD is caused by mutations in the HBB gene, leading to the production of a faulty version of hemoglobin the protein in red blood cells that transports oxygen. As a result, red blood cells acquire a sickle-like shape, becoming more fragile and prone to clumping. Misshapen red blood cells also can block blood vessels and trigger VOCs.

Lovo-cel, previously known as bb1111 or LentiGlobin, is a one-time therapy designed to insert functional copies of a modified HBB gene into blood stem cells isolated from a patient. When returned, the modified stem cells are expected to give rise to new red blood cells capable of producing a working version of hemoglobin.

The therapy is being evaluated in HGB-206 (NCT02140554), an ongoing Phase 1/2 study in up to 50 SCD patients, ages 12-50.

The FDA recently lifted a yearlong partial clinical hold on HGB-206 for patients younger than age 18 after an adolescent developed persistent anemia, a condition caused by a lack of healthy red blood cells, following treatment.

Investigation revealed the patient carried specific mutations in a gene that encodes a part of hemoglobin. As such, these mutations were added to the exclusion criteria for ongoing lovo-cel studies.

Trial data, released in December, showed the one-time gene therapy led to the sustained production of HbAT87Q hemoglobin and nearly eliminated severe VOCs in 29 patients at two years after treatment. HbAT87Q is the protein coded by the modified version of the HBB gene.

Bluebird also is recruiting for its open-label Phase 3 HGB-210 trial (NCT04293185) of lovo-cel in up to 35 adults and children, ages 2-50, with SCD. Eligible patients are being enrolled at sites across the U.S.

The Phase 3 studys main goal is to assess the proportion of participants with a complete resolution of vaso-occlusive events at six to 18 months after treatment.

Lovo-cel is the most deeply studied gene therapy in development for sickle cell disease, with more than 50 patients treated and multiple patients followed for more than six years, Obenshain said. We remain extremely confident in the quality of our BLA submission.

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Bluebird responds to FDA on sickle cell gene therapy's production... - Sickle Cell Disease News

Regenxbio earns key FDA designation for DMD gene therapy – Clinical Trials Arena

Regnexbios bid to develop a gene therapy for Duchenne muscular dystrophy (DMD) has received an important regulatory lift.

The US Food and Drug Administration (FDA) granted fast track designation for Regenxbios DMD candidate RGX-202, hastening the gene therapys development timeline. RGX-202 is currently recruiting for the Phase I/II AFFINITY DUCHENNE study (NCT05693142), which will have initial data available in the second half of 2023.

The regulatory designation gives Regnexbio a much-needed push, as RGX-202 lags behind key competitors in the race to bring a DMD gene therapy to market.

Sareptas gene therapy SRP-9001 is in a Phase III trial (NCT05096221) and could receive an FDA accelerated approval by May 29. Meanwhile, Pfizers gene therapy PF-06939926 is recruiting patients in a Phase III trial (NCT04281485) after rebounding from earlier safety concerns.

The crowded field of DMD drug development also features monoclonal antibodies, synthetic steroids, and even an allogenic stem cell therapy.

DMD is characterized by alterations in the protein dystrophin, causing progressive muscle loss primarily in young boys. RGX-202 is a one-time gene therapy using an adeno-associated vector (AAV) to deliver microdystrophin, a shortened form of the dystrophin protein intended to restore partial muscle function.

RGX-202 forms part of Regenxbios self-described 525 strategy to have five AAV therapies in the market or in late-stage development by 2025. Regenxbio also has gene therapies in development for Hunter syndrome, age related macular degeneration, and diabetic retinopathy, among others.

The Phase I/II AFFINITY DUCCHENE study has a target enrollment of 18 boys between the ages of 4 and 11. The primary endpoint focuses on safety, while secondary endpoints include microdystrophin expression and the North Star Ambulatory Assessment (NSAA) which measures DMD function.

Cell & Gene Therapy coverage on Clinical Trials Arena is supported by Cytiva.

Editorial content is independently produced and follows thehighest standardsof journalistic integrity. Topic sponsors are not involved in the creation of editorial content.

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Regenxbio earns key FDA designation for DMD gene therapy - Clinical Trials Arena

7 Unusual Symptoms of Parkinson’s: Expert Advice on Diagnosis and Advances in Treatment | TheHealthSi – TheHealthSite

7 Unusual Symptoms of Parkinson's: Expert Advice on Diagnosis and Advances in Treatment Dr (Lt Gen) CS Narayanan Vsm, HOD And Consultant, Department of Neurology, HCMCT Manipal Hospital, Dwarka talks about the unusual and often ignored symptoms of this condition and the various ways in which it can be treated.

Parkinson's disease is a neurodegenerative disorder that progressively affects the ability to move. It manifests through symptoms such as tremors, muscle stiffness, slow movement, and impaired balance. The disease results from the gradual loss of dopamine-producing cells in the brain, leading to insufficient dopamine levels, a neurotransmitter critical for regulating movement. Today, on World Parkinson's Day, we have Dr (Lt Gen) CS Narayanan Vsm, HOD And Consultant, Department of Neurology, HCMCT Manipal Hospital, Dwarka to tell us more about the unusual and often ignored symptoms of this condition and the various ways in which it can be treated.

Symptoms of Parkinson's disease usually develop slowly over time and can be different for each person. Some common signs and symptoms of Parkinson's disease include:

This is the most common symptom of Parkinson's disease. It usually starts in the hands or fingers and can spread to the arms, legs, and face. The tremors are often more noticeable when the person is at rest.

Parkinson's disease can cause stiffness and rigidity in the muscles, making it difficult to move freely.

This is a slowness of movement that can affect daily activities such as walking, getting dressed, or eating.

Parkinson's disease can cause problems with balance and coordination, leading to falls and injuries.

Parkinson's disease can affect speech and cause a soft, mumbled, or monotone voice.

Many people with Parkinson's disease lose their sense of smell.

Parkinson's disease can cause sleep disturbances, including insomnia and excessive daytime sleepiness.

Depression and anxiety are common in people with Parkinson's disease.

It is important to visit a doctor if you experience any of these symptoms, especially if they are persistent or worsen over time. Early diagnosis and treatment of Parkinson's disease can improve the quality of life and slow the progression of the disease.

The exact cause of Parkinson's disease is not known, but researchers believe it is caused by a combination of genetic and environmental factors. One of the main factors in Parkinson's disease is the death of dopamine-producing cells in the brain. Dopamine is a neurotransmitter that helps regulate movement, and when these cells die, the brain doesn't produce enough dopamine, which leads to the symptoms of Parkinson's disease.

While there is no cure for Parkinson's disease, there are treatments available that can help manage the symptoms. One of the most effective treatments is medication, particularly levodopa, which helps replenish dopamine in the brain. Other medications, such as dopamine agonists, can mimic the effects of dopamine in the brain.

"In addition to medication, there are other therapies that can help manage the symptoms of Parkinson's disease. One of these is deep brain stimulation (DBS), which involves implanting electrodes in the brain to stimulate specific areas. DBS has been shown to improve tremors, stiffness, and other symptoms of Parkinson's disease," says Dr (Lt Gen) CS Narayanan Vsm.

Researchers are also exploring other therapies, such as gene therapy and stem cell therapy, as potential treatments for Parkinson's disease. Gene therapy involves introducing new genetic material into cells to treat or prevent disease. Researchers are investigating gene therapy as a potential treatment for Parkinson's disease by introducing genes that produce dopamine into the brain. Stem cell therapy involves using stem cells, which are cells that can develop into different types of cells in the body, to replace the dopamine-producing cells that have died in the brain.

Dr (Lt Gen) CS Narayanan Vsm also highlighted the importance of regular exercises in the management of this condition. He said, "Exercise is another important part of managing Parkinson's disease. Regular exercise can improve balance, flexibility, and strength, and may help slow the progression of Parkinson's disease. Exercise can also improve mood and overall quality of life".

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7 Unusual Symptoms of Parkinson's: Expert Advice on Diagnosis and Advances in Treatment | TheHealthSi - TheHealthSite

Therapeutic effects of polydeoxyribonucleotide in an in vitro … – Nature.com

Cell culture

The Neuro-2a (N2a) cells, derived from mouse neuroblastoma, were purchased from the American Type Culture Collection (Manassas, VA, USA). N2a cells exhibit properties of neuronal stem cells and can differentiate into neuronal cells when treated with 20M retinoic acid (RA)1,15. The cells were incubated in culture dishes into Dulbeccos modified Eagles medium (DMEM; Hyclone, Logan, UT, USA) with 10% fetal bovine serum (FBS; Serum Source International, Charlotte, NC, USA) and 1% penicillin/streptomycin (Gibco, Rockville, MD, USA) in a humidified 5% CO2 atmosphere at 37C. When the cells reached 7080% of confluency, the medium was replaced as a differentiation medium, which contained 2% FBS and 20M RA in DMEM for four days. Differentiated N2a cells were maintained in a humidified atmosphere of 5% CO2 at 37C, and the differentiation medium was changed every two days.

The following procedures were adapted to establish an in vitro I/R injury model from previous studies1,16,17,18. Differentiated N2a cells were washed three times with phosphate-buffered saline (PBS), and the medium was replaced with deoxygenated, glucose-free balanced salt solution (Gibco) in hypoxic condition (O2 tension 1%) for 3h. Following OGD condition, injured cells were replaced onto the growth medium. PBS and 50 or 100g/ml PDRN (Placentex Integro, Mastelli Srl, Italy) was added to the growth medium of injured cells for 24h according to the following experimental groups. Cells treated with OGD and PBS were classified as the OGD group, and cells treated with OGD and PDRN were classified as the OGD+PDRN group. Differentiated N2a cells without OGD were classified as the non-OGD group (Fig.1A).

Effect of PDRN on an in vitro I/R injury model. (A) Experimental design of PDRN treatment on an in vitro I/R injury model. (B) Effect of PDRN on the cell viability of in vitro I/R injury model. (C) Bar graphs show the number of differentially expressed genes in OGD group compared with Non-OGD group and in OGD+PDRN group compared with OGD group. Values are presented as meansstandard error of the mean (SEM). Statistically significant differences are shown as **p<0.01, ***p<0.001.

To analyze the proliferation of the in vitro I/R injury model, the number of cells in the non-OGD, OGD, and OGD+PDRN groups were calculated using an advanced detection and accurate measurement (ADAM) automatic cell counter (NanoEnTek Inc., Seoul, South Korea).

After the cellular experiments, total RNA was isolated from the cells of all the groups with TRIzol reagent (Thermo Fisher Scientific, Waltham, MA, USA) was used for RNA isolation19 according to the manufacturers instructions. A NanoDrop spectrophotometer (Thermo Fisher Scientific) was used to confirm RNA quantity and purity.

RNA-seq transcriptome array of the non-OGD, OGD, and OGD+PDRN groups was performed at Macrogen Inc. (Seoul, Korea) with the HiSequation 2000 platform (Illumina, San Diego, CA, USA) according to methods detailed in our previous study20.

In this study, fold change (FC) criteria (FC|1.7|) were used to identify the differentially expressed genes (DEGs)from the results of RNA-seq transcriptome array. To identify their roles, three different pairs of DEGs were submitted to the Database for Annotation, Visualization and Integrated Discovery (DAVID) v.6.8 annotation tool21.

RT-qPCR was performed to validate the transcriptome analysis results. ReverTra Ace qPCR RT Master Mix with gDNA Remover (Toyobo, Osaka, Japan) was used to prepare the cDNA from total RNA, according to the manufacturers instructions. RT-qPCR was performed to measure the mRNA levels of the genes of interest using qPCRBIO SyGreen Mix Hi-ROX (PCR BIOSYSTEMS, London, UK) on a StepOnePlus Real-Time PCR System (Applied Biosystems, Foster City, CA, USA). The 2CT method was used for data analysis22. Supplementary Table S1 lists the primers used for RT-qPCR.

The proteins were extracted from the cell pellets. Proteins were boiled for 10min and loaded onto 412% bis Tris gels (Invitrogen, Waltham, MA, USA) for separation. The separated proteins were then blotted onto polyvinylidene difluoride (PVDF) membranes (Invitrogen) with 20% (v/v) methanol in NuPage Transfer Buffer (Invitrogen) at 15V for 4h at 4C. Tris-buffered saline containing 0.01% Tween 20 (TBST) in 5% skim milk (Difco, BD Biosciences, Oxford, UK) was used to block the membranes for 1h. The blots were washed three times with TBST for 10min and then incubated overnight at 4C with primary antibodies specific to the following target proteins: phosphorylated JAK1, phosphorylated JAK2, phosphorylated STAT1 (1:1000; Cell Signaling Technology, Cambridge, UK), CSF1, IL-6, PTPN6, RAC2, TNF, IL-1, IL-1, phosphorylated STAT3, STAT3 ADORA2A, JAK1, JAK2, STAT1, SOCS3, Bax, Bcl-2, and -actin (1:1000; Santa Cruz Biotechnology, Santa Cruz, CA, USA). After incubation, the blots were washed thrice with TBST and incubated for 1h with a horse-radish peroxidaseconjugated secondary antibody (1:3000; Santa Cruz) at 25C. Finally, the blots were visualized using an enhanced chemiluminescence detection system (Amersham Pharmacia Biotech, Little Chalfont, UK).

To analyze neuronal cell death, N2a cells were seeded on a 96-well cell culture plate (SPL Life Sciences, Gyeonggi-do, Korea) and OGD and PDRN treatments were performed as previously described above. The death of the in vitro I/R injury model was evaluated using a cytotoxicity lactate dehydrogenase (LDH) assay kit (Dojindo, Kumamoto, Japan) according to the manufacturer's protocol. Briefly, 10l of lysis buffer was added to each well and the cells were cultured at 37C in CO2 for 30min. A total of 100 L of the working solution was then added to each well, and the samples were cultured at room temperature in the dark. Stop solution (50l) was then added to each well, and LDH levels in the culture supernatant were analyzed immediately by measuring the absorbance at 490nm using a microplate reader (VersaMax, Molecular Devices, San Jose, CA, USA).

To analyze apoptosis, N2a cells were seeded on a cell culture slide (SPL Life Sciences, Gyeonggi-do, Korea) and OGD and PDRN treatments were performed as previously described above. The DeadEnd Fluorometric TUNEL System (Promega Madison WI USA) was used to assess apoptosis according to the manufacturers protocol. Briefly, the samples were mounted on glass slides with a fluorescent mounting medium with DAPI for imaging using an LSM 700 fluorescence microscope (Carl Zeiss, Gottingen, Germany). The number of positively stained cells over the total number of cells per specimen field was measured, and the percentage of positive cells was calculated. Four individual specimens were analyzed per group.

All data are expressed as the meanstandard error of the mean (SEM), and all experiments were repeated at least four times with four technical replicates in each group. The Statistical Package for Social Sciences v.25.0 (IBM Corp. Released 2015. IBM SPSS Statistics for Windows, v.25.0. Armonk, NY, USA) was used for the statistical analyses. The significance of intergroup differences was estimated using Students paired t-test or one-way analysis of variance (ANOVA). Statistical significance was set at p<0.05.

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Therapeutic effects of polydeoxyribonucleotide in an in vitro ... - Nature.com