Stem cell or bone marrow transplant is a way of giving very high dose chemotherapy. This treatment aimsto cure some types of cancer, including Hodgkin lymphoma.

Bone marrow is a spongy material that fills the bones.

It contains early blood cells, called stem cells. These develop into the 3 different types of blood cell.

You have a stem cell or bone marrow transplant after very high doses of chemotherapy. The chemotherapy has a good chance of killing the cancer cells but also kills the stem cells in your bone marrow.

Before your high dose chemotherapy, your team collects your stem cells or bone marrow. Or they collect adonor's stem cells or bone marrow. After the treatment you have the cells into a vein through a drip. The cells find their way back to your bone marrow. Then you can make the blood cells you need again.

You might have this intensive treatment if your Hodgkin lymphoma comes back after the first course of treatment. It can get rid of the lymphoma again for many people. Your doctor might also suggest this treatment if your Hodgkin lymphoma has not responded to the standard treatment.

You might have a course of high dose BEAM or LEAM chemotherapy. Then most people with Hodgkin lymphoma have their own stem cells or bone marrowback after the high dose treatment. This is called an autologous transplant.

You're now more likely to have a stem cell transplant (also called peripheral blood stem cell transplant) than a bone marrow transplant.

This is because:

You have injections of growth factors before, and sometimes after, the stem cell transplant. Growth factors are natural proteins that make the bone marrow produce blood cells.

You have daily injections of growth factor for between 5 and 10 days. Sometimes you might have low doses of chemotherapy with the growth factor injections.

After your growth factor injections, you have blood tests every day to see if there are enough stem cells in your bloodstream. When there are enough cells, you have them collected. This is called harvesting. Collecting the stem cells takes 3 or 4 hours. You are awake during this process. You lie down on a couch. Your nurse puts a drip into each of your arms and attaches it to a machine.

Your blood passes out of one drip. It goes through the machine and back into your body through the other drip. The machine filters the stem cells out of your blood. They are collected and frozen until after your high dose treatment.

You mayneed to go back the following day for a second harvest if they don'thaveenough cells from the first collection.

You might feel very tired after having your stem cell collection.

You might have:

This happens if your calcium level gets low during your collection. Your nurses will give you extra calcium through a drip if this happens.

You have your bone marrow taken (bone marrow harvest) under a general anaesthetic. This means you are asleep and can't feel anything during the procedure.

You lie on your side on a couch. Your doctor puts a needle through your skin into the hip bone (pelvis). The doctor gently draws out the bone marrow through the needle into a syringe. To get enough bone marrow the doctor needs to put the needle into several parts of the pelvis. You have about 2 pints (1 litre) of bone marrow taken out and then it's frozen until it's needed.

You might have a stem cell or bone marrow transplant using cells from a donor. This is called an allogeneic transplant. The cells need to be as similar as possible to yours.

So these can be from:

Youmight have bone marrow from a donor if:

Doctors are still learning how best to use allogeneic transplants for Hodgkin lymphoma.

The side effects of having a stem cell or bone marrow transplantare caused by high dose chemotherapy.

The main side effectsinclude:

You can call the Cancer Research UK nurses to talk about any worries you might have about having a transplant. The number is freephone 0808 800 4040, and the lines are open Monday to Friday, 9am to 5pm.

Visit link:
Stem cell and bone marrow transplants - Cancer Research UK

Bone Marrow Stem Cells Comments Off on Stem cell and bone marrow transplants – Cancer Research UK | November 22nd, 2022

Bone marrow donation is one of two methods of collecting blood forming cells for bone marrow transplants. Bone marrow donation is a surgical procedure that takes place in a hospital operating room. Doctors use needles to withdraw liquid marrow (where the bodys blood-forming cells are made) from both sides of the back of your pelvic bone. You will be given anesthesia and feel no pain during the donation. After donation, your liquid marrow is transported to the patients location for transplant.

Typically, the hospital stay for marrow donation is from early morning to late afternoon, or occasionally overnight for observation. The donation will take place in a hospital that is experienced and participates in marrow collections for Be The Match.

Common side effects of marrow donation reported 2 days after donation: Back or hip pain 84%, Fatigue 61%, Throat pain 32%, Muscle pain 24%, Insomnia 15%, Headache 14%, Dizziness 10%, Loss of appetite 10%, Nausea 9%.

The median time to full recovery for a marrow donation is 20 days. Recovery after marrow donation: 5% - 2 days, 18%-7 days, 71%-30 days, 97%-180 days, 99%-1 year

Learn more about what happensafter you donate.

Continue reading here:
Donating Bone Marrow Experience | Be The Match

Bone Marrow Stem Cells Comments Off on Donating Bone Marrow Experience | Be The Match | November 22nd, 2022

Join Be The Match Registry

The first step to being someone's cure is to join Be The Match Registry. If you are between the ages of 18-40, committed to donating to any patient in need, and meet the health guidelines, there are two ways to join.

Join in-person at a donor registry drive in your community.Be The One to Save a Life

Find a donor registry drive

Or join online today:

Join online

If you are between the ages of 18 and 35 patients especially need you. Research shows that cells from younger donors lead to more successful transplants. Doctors request donors in the 18-35 age group nearly 75% of the time.

Under 18 years old? Click here to sign up for the Under 18 Pre-Registry. You will receive information about ways to stay involved with our life-saving mission and a reminder to join when you're eligible.

There are many other ways you can be the cure for patients with blood cancers.

Check outFAQs about donationor call us at 1 (800) MARROW2 for more information about bone marrow donation.

Read more:
Learn How to Donate Bone Marrow | Be The Match

Bone Marrow Stem Cells Comments Off on Learn How to Donate Bone Marrow | Be The Match | October 29th, 2022

Stem cell/bone marrow transplant offers some patients with blood cancers and blood disorders the possibility of a cure, and others a longer period of disease-free survival. Founded in 1972, our Adult Stem Cell Transplant Program is one of the largest and most experienced in the world.

Our stem cell/bone marrow transplant program performs approximately 500 transplants each year and has performed more than 11,180 transplants in the programs history. This includes more than 5,500 allogeneic transplants and more than 5,100 autologous transplants. This experience makes a difference for our patients.

Our patients' outcomes regularly exceed expected outcomes as established by the Center for International Blood and Marrow Transplant Research, which reports and analyzes outcomes for recipients of allogeneic hematopoietic stem cell transplant. In the most recent report (2020), only 10% of centers achieved this outcome level. Dana-Farber Brigham Cancer Center was the largest center to achieve this outcome.

Stem cell/bone marrow transplant can be an effective treatment for a variety of hematologic malignancies, bone marrow failure syndromes, and rare and congenital blood disorders. We are experienced in stem cell transplant for a variety of hematologic malignancies, bone marrow failure syndromes, and rare and congenital blood disorders. This includes:

We perform both autologous and allogeneic stem cell/bone marrow transplants.

For allogeneic patients (i.e., those requiring donor stem cells), we offer:

Reduced-intensity transplants use lower doses of chemotherapy and have been a major factor in extending stem cell/bone marrow transplants for older adults up into their 70s. Our program has transplanted more than 5,000 patients over 55 years old. Our Older Adult Hematologic Malignancies Program provides dedicated support for older patients.

From exceptional medical care to support with housing and other logistics, we offer many services to international patients:

Learn more about international referrals and services.

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Stem Cell Transplantation Program - DanaFarber Cancer Institute

Bone Marrow Stem Cells Comments Off on Stem Cell Transplantation Program – DanaFarber Cancer Institute | October 29th, 2022

Fanconi anemia is a rare genetic disease in which essential DNA repair pathway genes are mutated, disrupting the DNA damage response. Patients with Fanconi anemia experience hematological complications, including bone marrow failure, and are predisposed to cancer. The only curative therapy for the hematological symptoms of Fanconi anemia is an allogeneic hematopoietic stem cell transplant, in which a patient receives healthy stem cells from a donor. While this may cure or prevent some of the diseases complications, stem cell transplantation can cause additional difficulties, including graft-versus-host disease (GvHD) and exacerbated cancer risk.1

There is growing interest in applying genome editing technologies like CRISPR-Cas9 to correct Fanconi anemia mutations in patient-derived cells for autologous transplants, in which corrected stem cells are given back to the patient. However, this disease poses a unique challenge: How do you apply a genome editing technique in cells that are particularly sensitive to DNA damage? Fanconi anemia cells cannot resolve the double-strand breaks that conventional CRISPR-Cas9 gene editing creates in the target DNA, which prevents researchers from effectively correcting disease-causing mutations with this method.

In a study published in International Journal of Molecular Science, a research team at the University of Minnesota led by Branden Moriarity and Beau Webber used Cas9-based tools called base editors (BEs) to edit genes in Fanconi anemia patient-derived cells without inducing double-strand DNA damage.2 BEs are fusion proteins made of a Cas9 enzyme that cleaves target DNA (nCas9) and a deaminase that converts cytidine to uridine (cytosine base editor, CBE) or adenosine to inosine (adenosine base editor, ABE). During DNA replication or repair, sites targeted by a BE are rewritten as thymine in the case of CBEs, or guanine with ABEs.

Although base editors do not induce double-strand breaks, they still nick the DNA and trigger a DNA repair response. Because of this, the researchers first examined if CBEs and ABEs would work on non-Fanconi anemia genes in patient-derived cells. There was that mystery, you know, because [Fanconi anemia patient cells are] DNA repair deficient. So we weren't surewe thought maybe it would work, but not as well as a normal cell. But indeed, it works on the same level, basically. So that was pretty exciting, Moriarity explained.

The research team then demonstrated that CBEs and ABEs can correct Fanconi anemia-causing mutations in the FANCA gene in primary patient fibroblast and lymphoblastoid cell lines. Base editing restored FANCA protein expression and improved the ability of the patient-derived cells to grow in the presence of a DNA damaging chemical. Additionally, in culture, fibroblasts with corrected FANCA mutations outgrew cells in which the base editing failed. Finally, the researchers assessed if BEs could correct mutations in different Fanconi anemia genes. Using an algorithm, they predicted that most Fanconi anemia mutations were correctable either by BEs or by another nCas9-fusion technology called prime editing (PE), which is capable of large genetic insertions and deletions.

This work comes on the heels of a preprint from another research group at The Centre for Energy, Environmental and Technological Research and ETH Zurich, who investigated ABEs in patient blood cell lines. This group also effectively targeted Fanconi anemia genes with BE technology, and their investigation went one step further: they corrected mutations in patient-derived hematopoietic stem cells.3This was something that Moriarity and Webber were unable to dobecause the disease is a bone marrow failure syndrome, these cells are scarce. Basically, these patients do not have stem cells, explains Annarita Miccio, a senior researcher and lab director at Institute Imagine of Paris Cit University, who was not involved in either study. These are very challenging experiments, and more than the experiments, the challenge of [treating] Fanconi anemia is exactly thatthe number of cells.

Despite this challenge, the researchers have laid the groundwork for genome editing as a treatment approach in Fanconi anemia, without the need for double-strand DNA breaks. I think the study we did is a good, solid proof of concept, and sets the stage for the next steps, but certainly, it's not the end of the story, said Webber.

References

Follow this link:
A CRISPR Alternative for Correcting Mutations That Sensitize Cells to DNA Damage - The Scientist

Bone Marrow Stem Cells Comments Off on A CRISPR Alternative for Correcting Mutations That Sensitize Cells to DNA Damage – The Scientist | October 13th, 2022

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.

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

Key Questions Answered in This Report:

Key Market Segmentation

Breakup by Product:

Breakup by Application:

Breakup by End User:

Breakup by Region:

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

10 SWOT Analysis

11 Value Chain Analysis

12 Porters Five Forces Analysis

13 Price Analysis

14 Competitive Landscape

For more information about this report visit https://www.researchandmarkets.com/r/5iujo7

Original post:
Stem Cell Manufacturing Global Market Report 2022: Widespread Product Utilization in Effective Disease Ma - Benzinga

Bone Marrow Stem Cells Comments Off on Stem Cell Manufacturing Global Market Report 2022: Widespread Product Utilization in Effective Disease Ma – Benzinga | October 13th, 2022

Company Logo

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.

Story continues

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...

Bone Marrow Stem Cells Comments Off on Stem Cell Manufacturing Global Market Report 2022: Widespread Product Utilization in Effective Disease Management, Personalized Medicine, and Genome… | October 13th, 2022

The FDA recently approved two gene therapies with hefty price tags, the first for an inherited anemia and the second for a degenerative brain condition. The two new treatments, from bluebirdbio, double the number of gene therapies on the market.

Most biotechnologies evolve over three decades or so, but the idea of gene therapy has been around since the late 1950s, blooming soon after Watson and Crick solved the structure of DNA. When my book The Forever Fix: Gene Therapy and the Boy Who Saved Itwas published a decade ago, it would still be 5 years before the first approval. That treatment, the subject of my book, enabled the blind to see, sometimes in just days.

Why has the pace of gene therapy been so slow? Cost is one barrier. Other concerns are the degree to which a gene therapy actually helps, how long the effect lasts, and what proportion of patients respond.

FDAs gene therapy roster ishere, but a caveat is necessary.

The list lumps gene therapy in with cell therapy, inviting unintentional hype from media folks unfamiliar with the science. Most entries actually refer to using stem cells to treat blood cancers and related conditions. An example: cartilage cells are sampled from a person with abum knee, mass-produced in a dish, and then injected into the knee, where they fuel production of more cartilage.

My favorite example of not-really-gene-therapy on the FDAs list targetsfacial wrinkles, also using patients lab-expanded cells: 18 million fibroblasts injected three times churn out collagen, filling in the offending skin craters.

Buried in the FDAs list are the first twoactualgene therapy approvals.Luxturna(Spark Therapeutics) treats RPE65 mutation-associated retinal dystrophy and has restored vision in many patients since its approval at the end of 2017. The second approved gene therapy, in 2019, isZolgensma, to treat spinal muscular atrophy, from Novartis Gene Therapies.

FDA approvedZynteglo on August 17, aka betibeglogene autotemcel or eli-cel. It treats the blood disorder beta thalassemia, which causes weakness, dizziness, fatigue, and bone problems. People with severe cases need transfusions of red blood cells every two to five weeks, which can lead to dangerous buildup of iron.

Zynteglo is a one-time infusion of stem cells descended from a patients bone marrow in which functional beta globin genes have been introduced aboard lentiviruses disabled HIV. The $2.8 million treatment is approved for adults and children.

Two clinical trials enrolled 91 patients, 36 of whom improved enough to no longer need transfusions. Bluebird estimates that 1,300 to 1,500 people in the U.S. may be candidates for Zynteglo.

The second go-ahead is forSkysona, approved September 16 for early active cerebral adrenoleukodystropy (CALD). The condition destroys the protective myelin sheath around brain neurons.

A stem cell transplant can cure CALD. Skysona is for the 700 or so boys aged 4 to 17 who cant find matched donors. Nearly fifty percent of them die within five years of symptom onset.

But like many gene therapies, Skysona isnt a magic bullet. In the two ongoing clinical trials, the metric for assessing improvement is slowing neurologic decline, tracking major functional disabilities. These include loss of communication skills, vision, and of voluntary movement, which impairs mobility, eating, and urinary retention.

The 2-year study that led to the FDA approval followed boys with mild or no symptoms, diagnosis possible early due to newborn screening in many states. Those who received Skysona had a 72% likelihood of survival over the two years without developing new major functional disabilities, compared to 43% among untreated boys. The trial will follow participants for 15 years. Since many states are nowscreening newborns for ALD, perhaps boys destined to develop symptoms can receive Skysona before that if someone will pick up the $3 million tab per patient.

Gene therapy companies have long justified high costs with the expense of the bench-to-bedside trajectory. So I was surprised to see a new study published inJAMA Network Open, Association of Research and Development Investments With Treatment Costs for New Drugs Approved From 2009 to 2018, finding none. The authors admonish companies to make further data available to support their claims that high drug prices are needed to recover research and development investments, if they are to continue to use this argument to justify high prices.

Becausethe paperuses terms like first-in-class, accelerated approval, breakthrough therapy, orphan, and priority review language Ive often seen attached to descriptions of gene therapy I assumed it would include Luxturna, which costs $850,000 for both eyes. But the new report omits drug names, instead citing a2020 paperfrom the team that did.No Luxturna. Thats probably because the researchers evaluated R&D costs only for products with publicly available data thats 63 drugs, a mere fifth of new approvals. The new report, of course sent out in news release form to the media, provides more a glimpse than a revelation.

So perhaps gene therapy is an exception for which high prices are indeed required to recoup investment. A viral vector to deliver DNA can cost $500,000 or more to produce, let alone engineer and develop.

Companies also use the one-and-done strategy to justify high prices. The homepage of bluebird bios website, for example, proclaims were pursuing curative gene therapies, although the data on Skysona for CALD indicate incremental change.Axios reports on how Medicaid, private insurers, and companies will help address cost concerns.

While bluebird bio bats around the c word cure it also introduces a long-needed granularity to the terminology. The company has replaced gene therapy with the more accurate gene addition therapy. Thats what the four approved gene therapies actually do add working copies of genes, not fixing them in place. Gene therapy is a little like patching a flat tire, not replacing it.

But the next stage of the evolving technology will in fact befixing genes, courtesy of gene and genome editing. This more precise strategy circumvents the problem of a piece of DNA inserting willy-nilly into a chromosome, perhaps disrupting a cancer-causing gene.

Gene editing with CRISPR has now been around for a decade. The components of the toolkit have been refined to minimize so-called off-target effects that can harpoon unintended genes.

A team atSt. Jude Childrens Research Hospitalhas developed what hematologist Yong Cheng terms the Google Maps of editing the genome. We provide a new approach to identify places to safely integrate a gene cassette. We created step-by-step directions to find safe harbor sites in specific tissues. The recipe is published inGenome Biologyand the tool availablehere.

The approach is seemingly simple. Using data from the 1000 Genomes Project, the tool identifies parts of the genome that often bear inserted or deleted DNA sequences among healthy people (and therefore are harmless) and are highly variable. These are the places where unwound DNA loops about itself when replicating just before a cell divides, and could tolerate a healing gene harpoon going astray.

Safe gene therapy requires two things. Number one, maintaining high expression of the new gene. And number two, the integration needs to have minimal effects on the normal human genome, Cheng said.

Gene addition therapy and gene/genome editing are slowly taking their places among other weapons against genetic disease. These include antisense treatments that glom onto mutant genes, small molecule-based drugs, repurposing existing drugs, supplements, and perhaps most important, the therapies that impact life on a daily basis. And so the toolbox expands to tackle the errors in our genes.

Ricki Lewis has a PhD in genetics and is a science writer and author of several human genetics books.She is an adjunct professor for the Alden March Bioethics Institute at Albany Medical College.Follow her at herwebsiteor Twitter@rickilewis

A version of this article originally appeared at PLOS and is reposted here with permission. Find PLOS on Twitter @PLOS

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Gene therapy approvals now at four with treatments for inherited anemia and degenerative brain condition but costs are stratospheric. Why? - Genetic...

Bone Marrow Stem Cells Comments Off on Gene therapy approvals now at four with treatments for inherited anemia and degenerative brain condition but costs are stratospheric. Why? – Genetic… | October 13th, 2022

CRANBURY, N.J.--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces data presentations at the 29th Annual Congress of the European Society of Gene & Cell Therapy (ESGCT) in Edinburgh, United Kingdom, taking place October 11-14, 2022. Presentations will include clinical data from Rockets lentiviral vector (LV)-based gene therapy programs for Leukocyte Adhesion Deficiency-I (LAD-I), Fanconi Anemia (FA) and Pyruvate Kinase Deficiency (PKD). Donald B. Kohn, MD, Distinguished Professor of Microbiology, Immunology & Molecular Genetics, Pediatrics, and Molecular & Medical Pharmacology at University of California, Los Angeles (UCLA) and Director of the UCLA Human Gene and Cell Therapy Program, will also give an Invited Talk incorporating previously disclosed data from the RP-L201 trial for LAD-I.

Positive Updated Safety and Efficacy Data from Phase 2 Pivotal Trial for Fanconi Anemia (FA)

The poster and presentation include updated safety and efficacy data from the Phase 2 pivotal trial of RP-L102, Rockets ex-vivo lentiviral gene therapy candidate for the treatment of FA.

Positive Top-line Clinical Data from Phase 2 Pivotal Trial for Severe Leukocyte Adhesion Deficiency-I (LAD-I)

The oral presentation includes previously disclosed efficacy and safety data at three to 24 months of follow-up after RP-L201 infusion for all patients and overall survival data for seven patients at 12 months or longer after infusion. RP-L201 is Rockets ex-vivo lentiviral gene therapy candidate for the treatment of severe LAD-I.

Interim Data from Ongoing Phase 1 Trial for Pyruvate Kinase Deficiency (PKD)

The poster and presentation include previously disclosed safety and efficacy data from the Phase 1 trial of RP-L301, Rockets ex-vivo lentiviral gene therapy candidate for the treatment of PKD.

Details for Rockets Invited Talk and poster presentations are as follows:

Title: Interim Results from an ongoing Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)Session: Clinical Trials (Plenary 2)Presenter: Donald B. Kohn, MD - University of California, Los Angeles, Distinguished Professor of Microbiology, Immunology & Molecular Genetics (MIMG), Pediatrics, and Molecular & Medical Pharmacology; Director of the UCLA Human Gene and Cell Therapy ProgramSession date and time: Wednesday, 12 October at 11:10-13:15 BSTLocation: Edinburgh International Conference Centre (EICC)Presentation Number: INV20

Title: Lentiviral-Mediated Gene Therapy for Patients with Fanconi Anemia [Group A]: Results from Global RP-L102 Clinical TrialsSession: Poster Session 1Presenter: Julin Sevilla MD, PhD - Fundacin para la Investigacin Biomdica, Hospital Infantil Universitario Nio JessSession date and time: Wednesday, 12 October at 19:30-21:00 BSTLocation: Edinburgh International Conference Centre (EICC)Poster Number: P139

Title: Preliminary Conclusions of the Phase I/II Gene therapy Trial in Patients with Fanconi Anemia-ASession: Blood Diseases: Haematopoietic Cell DisordersPresenter: Juan Bueren, PhD - Unidad de Innovacin Biomdica, Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT)Session date and time: Thursday, 13 October at 15:30-17:30 BSTLocation: Edinburgh International Conference Centre (EICC)Presentation Number: INV41

Title: Interim Results from an Ongoing Global Phase 1 Study of Lentiviral-Mediated Gene Therapy for Pyruvate Kinase DeficiencySession: Poster Session 2Presenter: Jos Luis Lpez Lorenzo, MD, Hospital Universitario Fundacin Jimnez DazSession date and time: Thursday, 13 October at 17:30-19:15 BSTLocation: Edinburgh International Conference Centre (EICC)Poster Number: P128

Abstracts for the presentations can be found online at: https://www.esgct.eu/.

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning. Graft-versus-host disease, a known complication of allogeneic HSCT, is associated with an increased risk of solid tumors, mainly squamous cell carcinomas of the head and neck region. Approximately 60-70% of patients with FA have a Fanconi Anemia complementation group A (FANCA) gene mutation, which encodes for a protein essential for DNA repair. Mutations in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Increased sensitivity to DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is a gold standard test for FA diagnosis. Somatic mosaicism occurs when there is a spontaneous correction of the mutated gene that can lead to stabilization or correction of a FA patients blood counts in the absence of any administered therapy. Somatic mosaicism, often referred to as natural gene therapy provides a strong rationale for the development of FA gene therapy because of the selective growth advantage of gene-corrected hematopoietic stem cells over FA cells.

About Leukocyte Adhesion Deficiency-I

Severe Leukocyte Adhesion Deficiency-I (LAD-I) is a rare, autosomal recessive pediatric disease caused by mutations in the ITGB2 gene encoding for the beta-2 integrin component CD18. CD18 is a key protein that facilitates leukocyte adhesion and extravasation from blood vessels to combat infections. As a result, children with severe LAD-I are often affected immediately after birth. During infancy, they suffer from recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics and require frequent hospitalizations. Children who survive infancy experience recurrent severe infections including pneumonia, gingival ulcers, necrotic skin ulcers, and septicemia. Without a successful bone marrow transplant, mortality in patients with severe LAD-I is 60-75% prior to the age of 2 and survival beyond the age of 5 is uncommon. There is a high unmet medical need for patients with severe LAD-I.

Rockets LAD-I research is made possible by a grant from the California Institute for Regenerative Medicine (Grant Number CLIN2-11480). The contents of this press release are solely the responsibility of Rocket and do not necessarily represent the official views of CIRM or any other agency of the State of California.

About Pyruvate Kinase Deficiency

Pyruvate kinase deficiency (PKD) is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway. Mutations in the PKLR gene result in increased red cell destruction and the disorder ranges from mild to life-threatening anemia. PKD has an estimated prevalence of 4,000 to 8,000 patients in the United States and the European Union. Children are the most commonly and severely affected subgroup of patients. Currently available treatments include splenectomy and red blood cell transfusions, which are associated with immune defects and chronic iron overload.

RP-L301 was in-licensed from the Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT), Centro de Investigacin Biomdica en Red de Enfermedades Raras (CIBERER) and Instituto de Investigacin Sanitaria de la Fundacin Jimnez Daz (IIS-FJD).

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare childhood disorders. The Companys platform-agnostic approach enables it to design the best therapy for each indication, creating potentially transformative options for patients afflicted with rare genetic diseases. Rocket's clinical programs using lentiviral vector (LVV)-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. Rockets first clinical program using adeno-associated virus (AAV)-based gene therapy is for Danon Disease, a devastating, pediatric heart failure condition. For more information about Rocket, please visit http://www.rocketpharma.com

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rockets future expectations, plans and prospects, including without limitation, Rockets expectations regarding its guidance for 2022 in light of COVID-19, the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), and Danon Disease, the expected timing and data readouts of Rockets ongoing and planned clinical trials, the expected timing and outcome of Rockets regulatory interactions and planned submissions, Rockets plans for the advancement of its Danon Disease program and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rockets ability to monitor the impact of COVID-19 on its business operations and take steps to ensure the safety of patients, families and employees, the interest from patients and families for participation in each of Rockets ongoing trials, our expectations regarding the delays and impact of COVID-19 on clinical sites, patient enrollment, trial timelines and data readouts, our expectations regarding our drug supply for our ongoing and anticipated trials, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rockets dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rockets Annual Report on Form 10-K for the year ended December 31, 2021, filed February 28, 2022 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Announces Presentations Highlighting Lentiviral Gene Therapies at the 29th Annual Congress of the European Society of Gene...

Bone Marrow Stem Cells Comments Off on Rocket Pharmaceuticals Announces Presentations Highlighting Lentiviral Gene Therapies at the 29th Annual Congress of the European Society of Gene… | October 13th, 2022

Cellectis Inc.

NEW YORK, Oct. 11, 2022 (GLOBE NEWSWIRE) -- Cellectis (the Company) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, announced today that the Company will present both an oral and poster at the European Society of Gene and Cell Therapys (ESGCT) 29th Congress, to be held in Edinburgh from October 11-14, 2022.

Arianna Moiani, Ph.D., Senior Scientist & Team Leader Innovation Gene Therapy, will give an oral presentation on encouraging pre-clinical data that leverages TALEN gene editing technology to develop a hematopoietic stem and progenitor cell (HSPCs)-based gene therapy to treat sickle cell disease.

Eduardo Seclen, Ph.D., Senior Scientist & Team Leader, Gene Editing, will present a poster illustrating a TALEN-based gene editing approach that reprograms HSPCs to secrete alpha-L-iduronidase (IDUA), a therapeutic enzyme missing in Mucopolysaccharidosis type I (MPS-I).

The pre-clinical data presented at ESGCT further demonstrate our ability to leverage TALEN gene editing technology to potentially address genetic diseases, namely, sickle cell disease and lysosomal storage diseases. By correcting a faulty mutation or inserting a corrected gene at the HSPC level, we aim to provide a lifelong supply of healthy cells in a single intervention, said Philippe Duchateau, Ph.D., Chief Scientific Officer at Cellectis. These new milestones bring us one step closer to our goal: providing a cure to patients that have failed to respond to standard therapy.

Presentation details

Pre-clinical data presentation on a non-viral DNA delivery associated with TALEN gene editing that leads to highly efficient correction of sickle cell mutation in long-term repopulating hematopoietic stem cells

Sickle cell disease stems from a single point mutation in the HBB gene which results in sickle hemoglobin.

Cellectis leveraged its TALEN technology to develop a gene editing process that leads to highly efficient HBB gene correction via homology directed repair, while mitigating potential risks associated to HBB gene knock-out. Overall, these results show that non-viral DNA delivery associated with TALEN gene editing reduces the toxicity usually observed with viral DNA delivery and allows high levels of HBB gene correction in long-term repopulating hematopoietic stem cells.

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The oral presentation titled Non-viral DNA delivery associated to TALEN gene editing leads to highly efficient correction of sickle cell mutation in long-term repopulating hematopoietic stem cells, will be made on Thursday, October 13th, 8:30AM-10:45AM BST by Arianna Moiani, Ph.D., Senior Scientist & Team Leader Innovation Gene Therapy. The presentation can be found on the Cellectis website on the day of the presentation.

Presentation details

Pre-clinical data presentation on TALEN-mediated engineering of HSPC that enables systemic delivery of IDUA

Mucopolysaccharidosis type I (MPS-I) is caused by deficiencies in the alpha-L-iduronidase (IDUA) gene and it is associated with severe morbidity representing a significant unmet medical need.

Cellectis established a TALEN-basedex vivogene editing protocol to insert an IDUA-expression cassette into a specific locus of HSPC.

Editing rates in vivo were 6-9% sixteen weeks after injection, depending on the tissue analyzed (blood, spleen, bone marrow). Lastly, 8.3% of human cells were edited in the brain compartment.

Cellectis established a safe TALEN-based gene editing protocol procuring IDUA-edited HSPCs able to engraft, differentiate into multiple lineages and reach multiple tissues, including the brain.

The poster presentation titled TALEN-mediated engineering of HSPC enables systemic delivery of IDUA, will be made on Thursday, October 13th, 5:30PM - 7:15PM BST by Eduardo Seclen, Ph.D., Senior Scientist & Team Leader, Gene Editing, and can be found on Cellectis website.

About Cellectis

Cellectis is a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies. Cellectis utilizes an allogeneic approach for CAR-T immunotherapies in oncology, pioneering the concept of off-the-shelf and ready-to-use gene-edited CAR T-cells to treat cancer patients, and a platform to make therapeutic gene editing in hemopoietic stem cells for various diseases. As a clinical-stage biopharmaceutical company with over 22 years of experience and expertise in gene editing, Cellectis is developing life-changing product candidates utilizing TALEN, its gene editing technology, and PulseAgile, its pioneering electroporation system to harness the power of the immune system in order to treat diseases with unmet medical needs. Cellectis headquarters are in Paris, France, with locations in New York, New York and Raleigh, North Carolina. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euronext Growth (ticker: ALCLS).

For more information, visit http://www.cellectis.com. Follow Cellectis on social media: @cellectis, LinkedIn and YouTube.

For further information, please contact:

Media contacts:Pascalyne Wilson,Director,Communications,+33 (0)7 76 99 14 33, media@cellectis.comMargaret Gandolfo, Senior Manager, Communications, +1 (646) 628 0300

Investor Relation contact:Arthur Stril, Chief Business Officer, +1 (347) 809 5980, investors@cellectis.comAshley R. Robinson, LifeSci Advisors, +1 617430 7577

Forward-looking StatementsThis press release contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995. Forward-looking statements may be identified by words such as anticipate, believe, intend, expect, plan, scheduled, could, may and will, or the negative of these and similar expressions. These forward-looking statements, which are based on our managements current expectations and assumptions and on information currently available to management. Forward-looking statements include statements about the potential of our preclinical programs and product candidates. These forward-looking statements are made in light of information currently available to us and are subject to numerous risks and uncertainties, including with respect to the numerous risks associated with biopharmaceutical product candidate development. With respect to our cash runway, our operating plans, including product development plans, may change as a result of various factors, including factors currently unknown to us. Furthermore, many other important factors, including those described in our Annual Report on Form 20-F and the financial report (including the management report) for the year ended December 31, 2021 and subsequent filings Cellectis makes with the Securities Exchange Commission from time to time, as well as other known and unknown risks and uncertainties may adversely affect such forward-looking statements and cause our actual results, performance or achievements to be materially different from those expressed or implied by the forward-looking statements. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future.

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Cellectis Presents Data on Two TALEN-based Gene Therapy Preclinical Programs for Patients with Sickle Cell Disease and Mucopolysaccharidosis type I at...

Bone Marrow Stem Cells Comments Off on Cellectis Presents Data on Two TALEN-based Gene Therapy Preclinical Programs for Patients with Sickle Cell Disease and Mucopolysaccharidosis type I at… | October 13th, 2022

OverviewWhat is bone marrow?

Bone marrow is the soft, fatty tissue inside of bone cavities. Components of your blood including red and white blood cells and platelets form inside of your bone marrow.

Bone marrow makes nearly all the components of your blood. It's responsible for creating billions of red blood cells daily, along with white blood cells and platelets. Bone marrow also stores fat that turns into energy as needed.

Bone marrow makes the components of your blood that you need to survive. Bone marrow produces red blood cells that carry oxygen, white blood cells that prevent infection and platelets that control bleeding. The absence of bone marrow can be fatal since it's an essential part of your body.

Yes, bone marrow and the healthy cells it produces are necessary for humans to live. Often, cell mutations harm healthy bone marrow cells, and a bone marrow transplant would be a treatment option for people diagnosed with blood cancers like leukemia.

A bone marrow transplant takes healthy cells from a donor and puts them into your bloodstream. The donors cells help your body grow healthy red and white blood cells and platelets.

There are three parts to the anatomy of your bones: compact bone, spongy bone and bone marrow. Compact bone is the strong, outer layer of your bones. Spongy bone makes up the ends of your bones. Bone marrow is in the center of most bones and in the end of spongy bones in your body. Bone marrow and blood vessels fill cavities in your bones, where they store fat and stem cells and produce blood cells that make your whole blood.

Bone marrow is a spongy, soft tissue that resembles a jelly or jam that you would spread on toast. It comes in two colors, red and yellow. Bone marrow fills the cavities of your bones and holds cells that create red and white blood cells and platelets, which make whole blood. The color of red bone marrow is the result of red blood cell production.

There are two types of bone marrow in your body, which are characterized by their color. Your body holds just under 6 lbs. (about 2.5 kg.) of red and yellow bone marrow.

Red bone marrow makes up all of your bone marrow until about age seven. Yellow bone marrow gradually replaces red bone marrow as you age.

Bone marrow is made of stem cells. These stem cells make red bone marrow, which creates blood cells and platelets for your blood. Yellow bone marrow consists mostly of fat and stem cells that produce bone and cartilage in your body.

Directly targeting bone marrow is leukemia, which is a blood and bone marrow cancer. Leukemia forms when a cell mutation occurs in your bone marrow and mutated cells multiply out of control, reducing the production of healthy, normal cells.

Since bone marrow is the foundation for the creation of blood cells, blood-related conditions often are the result of abnormally functioning bone marrow. These conditions include:

Common symptoms of bone marrow conditions include:

There are two tests to check the health of your bone marrow and/or blood cells:

For a bone marrow test or donation, youll receive an anesthetic, so you won't feel any pain during the procedure. After the procedure, you may feel side effects, which include aches and pain at the site of the incision. Each individual experiences pain differently, so the severity could vary from person to person. The pain may last for a few days or up to several weeks.

Treatments for bone marrow conditions vary based on the severity and progress of the diagnosis. Treatment options include:

Bone marrow is the foundation of your bones, blood and muscles. Keeping your bone marrow healthy focuses on supporting components of your body that grow from bone marrow cells. You can keep your bone marrow healthy by:

A note from Cleveland Clinic

Bone marrow is the soft center of the bones in your body. Bone marrow is necessary to create components of your blood and store fat. The best way to keep your bone marrow healthy is to support the parts of your body that your bone marrow produces, like your blood, muscles and bones.

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Bone Marrow: What it is & Why it is Important - Cleveland Clinic

Bone Marrow Stem Cells Comments Off on Bone Marrow: What it is & Why it is Important – Cleveland Clinic | October 5th, 2022

We explain a protocol for straightforward isolation and culture of mesenchymal stem cells (MSCs) from mouse bone marrow (BM) to supply researchers with a method that can be applied in cell biology and tissue engineering with minimal requirements. Our protocol is mainly on the basis of the frequent medium change in primary culture and diminishing the trypsinization time. Mouse mesenchymal stem cells are generally isolated from an aspirate of BM harvested from the tibia and femoral marrow compartments, then cultured in a medium with Dulbecco's modified Eagle's medium (DMEM) and fetal bovine serum (FBS) for 3 h in a 37 degrees C-5% CO(2) incubator. Nonadherent cells are removed carefully after 3 h and fresh medium is replaced. When primary cultures become almost confluent, the culture is treated with 0.5 ml of 0.25% trypsin containing 0.02% ethylenediaminetetraacetic acid for 2 min at room temperature (25 degrees C). A purified population of MSCs can be obtained 3 weeks after the initiation of culture.

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A protocol for isolation and culture of mesenchymal stem cells from ...

Bone Marrow Stem Cells Comments Off on A protocol for isolation and culture of mesenchymal stem cells from … | October 5th, 2022

BOSTON--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapies for patients with hematologic and solid cancers and other serious diseases, today announced the presentation of new long term follow-up data and health-related quality of life scores of patients treated with omidubicel at the Tenth Annual Meeting of the Society of Hematologic Oncology (SOHO), being held in Houston, Texas.

These data reinforce our commitment to advance transformational cell therapy research and underscore the potential of our NAM technology platform. Our lead stem cell therapy candidate, omidubicel, addresses the unmet need for patients with hematologic malignancies, demonstrated by the robust and growing body of encouraging clinical evidence, including the long-term follow up data and quality of life improvement, said Ronit Simantov, M.D., Chief Medical Officer of Gamida Cell. As we approach the PDUFA date of January 30, 2023, and upon potential FDA approval, we are prepared to execute our plan that ensures access to those patients who can benefit from omidubicel as quickly as possible.

The long-term, durable clinical benefit of omidubicel was observed at three years across a patient population that typically has a poor prognosis. A study titled, Multicenter Long-Term Follow Up of Allogeneic Hematopoietic Stem Cell Transplantation with Omidubicel: A Pooled Analysis of Five Prospective Clinical Trials, highlighted long-term follow-up of 105 patients transplanted with omidubicel between 2006-2020 (median follow-up of 22 months). The data demonstrated an overall survival and disease-free survival of 63% (95% CI, 53%-73%) and 56% (95% CI, 47%-67%) at three years, respectively, as well as durable long-term hematopoiesis and immune competence. Learn More

Overall well-being health-related quality of life scores for patients treated with omidubicel demonstrated clinical benefit compared to standard of care. A study titled, Health-Related Quality of Life Following Allogeneic Hematopoietic Stem Cell Transplantation with Omidubicel Versus Standard Umbilical Cord Blood featured an analysis of 108 patients that completed validated health-related quality of life (HRQL) surveys on screening and days 42, 100, 180, and 365 post-transplant. Measures of physical and functional well-being and other HRQL scores were more favorable with omidubicel. These data suggest clinically meaningful and sustained improvements in physical, functional, and overall well-being compared to umbilical cord blood transplantation. Learn More

About NAM Technology

Our NAM-enabling technology is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (nicotinamide), we can expand and metabolically modulate multiple cell types including stem cells and natural killer cells with appropriate growth factors to maintain the cells active phenotype and enhance potency. Additionally, our NAM technology improves the metabolic fitness of cells, allowing for continued activity throughout the expansion process.

About Omidubicel

Omidubicel is an advanced cell therapy candidate developed as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant for patients with blood cancers. Omidubicel demonstrated a statistically significant reduction in time to neutrophil engraftment in comparison to standard umbilical cord blood in an international, multi-center, randomized Phase 3 study (NCT0273029) in patients with hematologic malignancies undergoing allogeneic bone marrow transplant. The Phase 3 study also showed reduced time to platelet engraftment, reduced infections and fewer days of hospitalization. One-year post-transplant data showed sustained clinical benefits with omidubicel as demonstrated by significant reduction in infectious complications as well as reduced non-relapse mortality and no significant increase in relapse rates nor increases in graft-versus-host-disease (GvHD) rates. Omidubicel is the first stem cell transplant donor source to receive Breakthrough Therapy Designation from the FDA and has also received Orphan Drug Designation in the US and EU.

Omidubicel is an investigational stem cell therapy candidate, and its safety and efficacy have not been established by the FDA or any other health authority. For more information about omidubicel, please visit https://www.gamida-cell.com.

About Gamida Cell

Gamida Cell is pioneering a diverse immunotherapy pipeline of potentially curative cell therapy candidates for patients with solid tumor and blood cancers and other serious blood diseases. We apply a proprietary expansion platform leveraging the properties of NAM to allogeneic cell sources including umbilical cord blood-derived cells and NK cells to create therapy candidates with potential to redefine standards of care. These include omidubicel, an investigational product with potential as a life-saving alternative for patients in need of bone marrow transplant, and a line of modified and unmodified NAM-enabled NK cells targeted at solid tumor and hematological malignancies. For additional information, please visit http://www.gamida-cell.com or follow Gamida Cell on LinkedIn, Twitter, Facebook or Instagram at @GamidaCellTx.

Cautionary Note Regarding Forward Looking Statements

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to timing of initiation and progress of, and data reported from, the clinical trials of Gamida Cells product candidates (including omidubicel), regulatory filings submitted to the FDA (including the potential timing of the FDAs review of the BLA for omidubicel), commercialization planning efforts, and the potentially life-saving or curative therapeutic and commercial potential of Gamida Cells product candidates (including omidubicel), and Gamida Cells expectations for the expected clinical development milestones set forth herein. Any statement describing Gamida Cells goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to a number of risks, uncertainties and assumptions, including those related to the impact that the COVID-19 pandemic could have on our business, and including the scope, progress and expansion of Gamida Cells clinical trials and ramifications for the cost thereof; clinical, scientific, regulatory and technical developments; and those inherent in the process of developing and commercializing product candidates that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such product candidates. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cells Quarterly Report on Form 10-Q, filed with the Securities and Exchange Commission (SEC) on May 12, 2022, as amended, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cells actual results could differ materially and adversely from those anticipated or implied thereby. Although Gamida Cells forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Gamida Cell. As a result, you are cautioned not to rely on these forward-looking statements.

1CIBMTR 2019 allogeneic transplants in patients 12+ years with hematological malignancies.2Gamida Cell market research

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Long-Term Data from Omidubicel Phase 3 Trial Demonstrates Overall Survival and Sustainable Durable Outcomes for Patients with Blood Cancers at the...

Bone Marrow Stem Cells Comments Off on Long-Term Data from Omidubicel Phase 3 Trial Demonstrates Overall Survival and Sustainable Durable Outcomes for Patients with Blood Cancers at the… | October 5th, 2022

As cancer treatments continue to advance and new therapies are introduced, it's easy to get lost in your search for information. To help you better understand the differences between specific cancer treatments and how they work, we spoke with medical oncologist Bhavina Sharma, MD, MPH.

"Chemotherapy are drugs designed to directly attack all rapidly dividing cells in the body, including cancer cells," explains Dr. Sharma. "It relies on the idea that cancer cells reproduce much faster than most healthy cells in our body."

Chemotherapy drugs can be given by infusion or in pill form. Unfortunately, these drugs can't tell the difference between cancerous cells and fast-growing healthy cells like the gastrointestinal tract and hair follicles, leading to side effects such as diarrhea and hair loss. Thankfully, recent advancements in chemotherapy have helped lessen side effects such as nausea, pain and lethargy.

Targeted therapy are special drugs designed to target differences within cancer cells that help them thrive. Unlike chemotherapy, targeted therapy drugs actually change the inner workings of the cancer cell. Because targeted therapy focuses on the part of the cancer cell that makes it different from the normal, healthy cell, it often has fewer side effects than standard chemotherapy treatments.

Immunotherapy is very different than chemotherapy in that it helps our immune system to find and kill cancer cells.

"Cancer cells are abnormal cells that have formed in our body because of cell damage or mutations," explains Dr. Sharma. "Cancer cells hide from your immune system by shutting down certain pathways of the immune response. Immunotherapy unlocks those pathways so your immune system can recognize and remove the cancer cells."

Cellular therapies are treatments that improve the body's ability to fight cancer. "Stem cell therapy falls under the umbrella of cellular therapy," explains Dr. Sharma. "It uses stem cells to mount an immune response to attack your cancer cells."

Stem cells from blood and bone marrow can be used in transplants. These stem cells can either come from a matched donor (allogeneic) or from the patient themselves (autologous).

Chimeric antigen receptor therapy or CAR T-cell, is a type of cellular therapy.

"T cells are white blood cells that help our bodies fight infection and cancer," explains Dr. Sharma. "With CAR T-cell therapy, your own T cells are collected from your blood. These T cells are modified to recognize cancer as a foreign cell and attack it."

CAR T-cell therapy has been approved by the Food and Drug Administration to treat lymphoma, leukemia and multiple myeloma.

Hormone therapy slows or stops the growth of cancer that uses hormones to grow. It is also called hormonal therapy, hormone treatment or endocrine therapy. Hormone therapy is recommended for cancers that are hormone-receptor positive, such as certain breast and prostate cancers. It can't be used in cancers that don't carry hormone receptors.

"Hormone therapy can be used for both early stage and metastatic hormone-receptor positive breast cancers," explains Dr. Sharma. "In patients with early-stage breast cancer, it is used after surgery to help reduce the risk of the cancer coming back."

Chemotherapy, immunotherapy, targeted therapy, and hormone therapy are just a few of the treatments we use to treat cancer. Many of these cancer treatments can be combined with others like cancer surgery and radiation therapy. Every person's journey through cancer is different. Your oncology team will help you sort through the best therapies available to create your treatment plan.

The information in this article is for information purposes only. For specific questions regarding your medical condition or treatment plan, please consult with your doctor directly. To schedule an appointment with a Nebraska Medicine cancer specialist, call 402.559.5600.

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Are immunotherapy and chemotherapy the same thing? How cancer treatments work - Nebraska Medicine

Bone Marrow Stem Cells Comments Off on Are immunotherapy and chemotherapy the same thing? How cancer treatments work – Nebraska Medicine | October 5th, 2022

Dr. Jeff Hersh| Daily News Correspondent

Q: What is CAR T-cell therapy?

A: Our immune system protects our bodies from "invasion"by harmful substances, infections and abnormal cells (for example cancer cells). T-cells (also called T-lymphocytes or thymocytes) develop from stem cells in the bone marrow and mature in the thymus (a body organ in the neck that is part of the lymphoid system, along with the spleen, lymph nodes and the red bone marrow).

Cytotoxic T-cells identify body cells that have certain antigens (proteins on the surface of certain cancer cells, cells that have become infected, other cells) and directly kill them.

Helper T-cells detect various "invasions"and release cytokines to activate other immune system cells (including cytotoxic T-cells) to combat them.

Regulatory T-cells help moderate the immune response to maintain balance and the bodys ability to tolerate (rather than attack) itself (for example helping minimize inappropriate inflammatory responses).

This description of T-cells shows why it would be helpful to "manipulate"them in a specific manner to leverage the immune system to help fight certain diseases/conditions. This is where chimeric antigen receptor (CAR) T-cells come into play.

White blood cells (including T-cells) are collected from the patient by taking some of their blood via an intravenous (IV) catheter and filtering out the white cells using a leukapheresis machine, and then putting the filtered blood (minus the extracted white blood cells) back into the patient via a second IV catheter.

The T-cells are then separated from the other white blood cells, and a gene for the "targeted" antigen is added to the cells (you can think of this as a "lock and key"mechanism, with the antigen being the "lock"and the protein added to the T-cell being the "key"used to identify the "invading" cell with that particular antigen "lock."

These modified cells (the CAR T-cells) are then "multiplied"in the lab to create a large number of them. The CAR T-cells are then infused into the patient (again via an IV). These CAR T-cells can now specifically "hunt"the specific "invading"cell(s) they have been created to target.

There are many steps needed to create this personalized CAR T-cell treatment for an individual patient, and therefore it can take weeks to produce these treatments. In the future it may be possible to pre-prepare treatments from donor T-cells (possibly modifying these cells to target specific antigens using techniques like CRISPR, mRNA techniques, etc.) and then transfuse the appropriate CAR T-cells in a manner similar to how other blood products (for example red cells, platelets, etc.) are transfused to help a patient.

Since 2017 CAR T-cells have been specifically designed and utilized to treat individual patients with several different types of "blood cancers"(lymphomas, leukemias and multiple myelomas) that did not respond to the standard treatments (for example chemotherapy for that type of cancer).In many patients with very difficult to treat blood cancers, these treatments have been very effective.

Solid tumors (as opposed to blood cancers), such as brain, breast, lung and pancreatic cancers, are a bit more challenging to address with the CAR T-cell approach.This is because having the CAR T-cells gain "access"to the solid tumor cancer cells is more difficult.

From the description of T-cells above, it seems that this same conceptual approach might be utilized to treat certain autoimmune conditions (conditions where a patients own immune system "overreacts"and attacks the patients own body cells), and this has recently been studied. In this study, five patients with severe lupus who had not responded to standard treatments were treated with specifically designed CAR T-cells to "wipe out"the aberrant B cells causing their autoimmune complications, and all five showed very significant improvement. Future clinical studies will no doubt be designed to see what other conditions might benefit from this treatment approach!

However, treatment with CAR T-cells is not without risk, as these treatments can sometimes cause serious and even life-threatening complications. For example, some patients have had:

Cytokine release syndrome (CRS), where the patient reacts to the CAR T-cell infusion with an aggressive release of cytokines that causes an inflammatory reaction (for example causing symptoms like fever, breathing issues, gastrointestinal issues, other symptoms); nervous system issues (for example headaches, seizures, alterations in consciousness, others), and there may bemany other possible complications.

Bottom line: CAR T-cell therapy has become a more and more accepted therapeutic approach, and in the future it may be utilized earlier in a patients disease (rather than only for refractory cases), and for a broader array of disease states (not just blood cancers, but potentially autoimmune conditions, maybe certain solid tumors, and potentially other diseases).

Jeff Hersh, Ph.D., M.D., can be reached at DrHersh@juno.com.

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Diagnosis, Treatment, and Prevention of Blood Cancer By Dr Gaurav Kharya

Written by Tavishi Dogra | Updated : October 4, 2022 9:56 PM IST

In India, the increase in cancer cases over the past ten years has become a significant public health problem for the country. These cases have a long latent period, are primarily lifestyle-related and require specialised infrastructure and human resources to be treated. Cancer's physical, psychological and financial toll on people, families, communities and health systems keeps rising. The prevalence of cancer varies across India's regions, making prevention and management extremely difficult. Due to cancer not being a notifiable disease, the national burden assessment is still a task for which many developing nations, including India, rely on statistical models. The estimated number of cancer-related Disability-adjusted life years (DALYs) (AMI) in India in 2021 was 26.7 million, and that number was predicted to rise to 29.8 million in 2025.

Each year, 1.24 million new instances of blood cancer are reported worldwide, making up about 6% of all cancer cases. Blood cancer develops in the bone marrow, tissues that create blood and compromise the immune system. According to incidence rates, there are primarily three different forms of blood cancers: lymphoma/leukaemia, multiple myeloma, myelodysplastic syndromes (MDS)/myeloproliferative neoplasms (MPN). In addition, blood cancer may arise when the body produces abnormal White Blood Cells (WBCs). It typically starts in the bone marrow, which produces blood in our body. This malignancy impairs the normal development, growth and functioning of blood cells that fight infection and produce healthy blood cells.

White blood cells produced by the body during leukaemia are incapable of battling infections. Depending on the type of blood cell involved and whether it is fast-growing or slow-growing (acute or chronic), leukaemia is divided into distinct forms (myeloid or lymphoid). Consequently, it can be broadly divided into four subtypes: acute lymphocytic leukaemia (ALL), acute myeloid leukaemia (AML), chronic lymphocytic leukaemia (CLL) and chronic myeloid leukaemia (CML). Apart from these are some rare blood cancers such as Juvenile myelomonocytic leukaemia (JMML).

Diagnosis, Treatment, and Prevention of Blood Cancer By Dr Gaurav Kharya, Clinical Lead Apollo Center & Indraprastha Apollo Hospital

Various diagnostic techniques are used to identify blood cancer, including clinical examination, blood testing, bone marrow tests, cytogenetic/karyotyping, molecular analyses, and flow cytometry. Most pediatric patients diagnosed with ALL or AML can be treated by chemotherapy. However, a smaller percentage of patients who don't respond well to chemotherapy are candidates for Bone marrow transplant to offer a long-term cure to these patients. In contrast, almost half of adult patients need BMT as consolidation to provide long-term treatment. If required, BMT can safely be done now using half HLA identical donors in case HLA matching donors are unavailable in experienced centres.

In most cases, the doctor will make a treatment recommendation based on research on the most effective treatments and national recommendations developed by experts. They will assess the type of blood cancer, the outcomes of any tests the patient has had, the state of the overall health, the available therapies, their effectiveness, and any potential risks or side effects.

There is a range of different treatments for blood cancer. But the most common ones include:

The cost of blood cancer therapy in India has several significant advantages. First, the most outstanding hospitals in India, equipped with the most cutting-edge equipment and a staff of oncologists and doctors with years of experience, are accessible to offer blood cancer patients comprehensive care.

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NEW YORK, Oct. 4, 2022 /PRNewswire/ --

Growing Use of IVF and Stem Cell Therapies to Create US$ 323 Million Market Opportunity for Carbon Dioxide Incubator Manufacturers

The carbon dioxide incubators market is well covered by Fact.MR for the upcoming decade. The study looks closely at key growth factors such trends, future projections, and business strategies. The research also provides a thorough analysis of the top segments including product, application, capacity, and region, in order to provide well-rounded perspective.

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Fact.MR A Market Research and Competitive Intelligence Provider: The global carbon dioxide incubators market is likely to reach US$ 483.5 Million by 2027, growing at 8.4% CAGR between 2022 and 2027. Growing investment in research and clinical trial activities is likely to fuel the sales of carbon dioxide incubators during the assessment period. Further, use of carbon dioxide incubators in IVF and stem cell treatments is also likely to drive growth.

The popularity and acceptance of in-vitro fertilizations has grown significantly. According toNational Library of Medicine, around 10% to 15% couples in the U.S. have trouble in having a baby. These challenges have been well-addressed by in vitro fertilization (IVF), owing to which it has become a popular healthcare solution.

Use of in-vitro fertilization (IVF) to help couples in becoming parents is likely to grow in the future, which is likely to drive demand for accessories and equipment used in this process. Owing to this, demand for carbon dioxide incubators is likely to witness an upward trend over the upcoming decade.

Further, sales of carbon dioxide incubators are also likely to increase on account of growth in overall stem cell procedures. For instance, as perHealth Resources and Services Administration, 4,864 unrelated and 4,160 related bone marrow and cold transplants were conducted in the U.S. in 2020. Growing use of stem cell treatment is likely to be a key factor driving the sales of carbon dioxide incubators during the assessment period.

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Globally, North America and Europe are likely to remain at the pinnacle of growth in the carbon dioxide incubators market. The U.S., U.K., France, and Germany are at the forefront of new innovation in R&D, and sales of medical accessories and equipment will also remain high, as per Fact.MR. Owing to these factors, carbon dioxide incubator manufacturers are likely to witness incremental growth opportunities across these regions.

Key Takeaways:

By product, water-jacketed carbon dioxide incubators are likely to reman preferred among end-users.

By capacity, below 100-liter carbon dioxide incubators are expected to witness high demand during the assessment period.

By application, use of carbon dioxide incubators in laboratory research and clinical applications is likely to remain high during the assessment period.

By region, North America and Europe are likely to hold sway over the forecast period, with the U.S. and the U.K. leading the growth.

China and India are expected to create sizeable opportunities for market players on the back of improved healthcare infrastructure.

Growth Drivers:

Increasing applications of carbon dioxide incubators in in-vitro fertilization (IVF) and stem cell treatment is likely to drive the market.

Use of carbon dioxide incubators in cell culture development and tissue engineering is expected to create growth avenues for market players.

Efficiency of incubators in maintaining consistent temperature during genetically modified organism (GMO) cultivation is expected to drive growth.

Advancement in carbon dioxide incubator technology is likely to create new growth avenues for market players.

Restraints:

Carbon dioxide incubators are highly prone to errors due to which they require highly experienced technicians. Due to skill shortage, sales of these incubators can be limited.

Lack of standardization is a longstanding challenge and failure to address this issue might hamper growth.

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Competitive Landscape:

Carbon dioxide incubator manufacturers are focusing on launching innovative technologies to consolidate their position in the market. Further, leading players are concentrating on providing training and guidelines to end-users so their products can be used without any issue.

For instance,

In May 2021, Esco introduced an innovative incubator featuring High Heat Sterilization that is highly effective in eliminating bacteria and vegetative cells.

In January 2020, CO2Meter Inc., launched incubators that regulate and monitor bacterial development patterns.

Key Companies Profiled by Fact.MR

More Valuable Insights on Carbon Dioxide Incubators Market

In its latest study, Fact.MR offers a detailed analysis of the global carbon dioxide incubators market for the forecast period of 2022 to 2027. This study also divulges key drivers and trends promoting the sales of carbon dioxide incubators through detailed segmentation as follows:

By Product:

Water Jacketed

Air Jacketed

Direct Heat

By Capacity:

Below 100 Litres

100-200 Litres

Above 200 Litres

By Application:

By Region:

North America

Latin America

Europe

East Asia

South Asia & Oceania

MEA

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Key Questions Covered in the Carbon Dioxide Incubators Market Report

What is the estimated market size of carbon dioxide incubators in 2022?

At what pace will worldwide carbon dioxide incubators sales increase till 2027?

What factors are driving demand in the carbon dioxide incubators market?

Which region is predicted to lead the worldwide carbon dioxide incubators market between 2022 and 2027?

What are the elements driving carbon dioxide incubators market sales during the forecast period?

What is the expected market estimation of the carbon dioxide incubators market during the forecast period?

Explore Fact.MR's Coverage on the Healthcare Domain

Biological Indicator Incubator Market:The biological indicator incubators market is projected to benefit from rising biopharmaceutical production. The market for biological indicator incubators may continue to increase quickly as a result of the manufacturing of biopharmaceuticals that are grown via cell culture.

Tissue Culture Incubator Market:The introduction of CO2 incubators with infrared radiation control systems and other technological advancements in tissue culture incubators, along with increased funding for tissue-based research, are anticipated to be major factors driving the growth of the tissue culture incubator market over the forecast period.

Pneumatic Nebulizers Market:Pneumatic nebulizer sales are anticipated to grow steadily at a CAGR of 4% and reach a market value of US$ 850.4 million by 2027 from US$ 699 million in 2022. An increase in local healthcare spending and patient awareness has spurred the need for pneumatic nebulizers.

Implantable Medical Devices Market: The global implantable medical devices market is predicted to reach US$ 155 billion by 2027. Key factors driving market growth include rising geriatric population & burden of chronic diseases and increasing demand for cosmetic dentistry.

Disinfection Caps Market: Key factors driving market growth include stringent regulations for safe injection practices and rising prevalence of hospital-acquired infections across the world. The global disinfection caps market is estimated to reach US$ 420 million by 2027.

Check it Out More Reports by Fact.MR on Healthcare Domain

https://www.factmr.com/industry/healthcare

About Fact.MR

Fact.MR is a market research and consulting agency with deep expertise in emerging market intelligence. Spanning a wide range from automotive & industry 4.0 to healthcare, technology, chemical and materials, to even the most niche categories. We are committed to deliver insights that help businesses gain deeper understanding of their target markets. We understand that making sense of the vast labyrinth of data can be overwhelming for businesses. That's why focus on offering insights that can actually make a difference to bottom-lines.

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Carbon Dioxide Incubators Market to Reach US$ 483.5 Million by 2027 as Application in In Vitro Fertilization Rises - Yahoo Finance

Bone Marrow Stem Cells Comments Off on Carbon Dioxide Incubators Market to Reach US$ 483.5 Million by 2027 as Application in In Vitro Fertilization Rises – Yahoo Finance | October 5th, 2022

Pain.

It gnawsat you. It drains you. It becomes the focus of your life.

Experiencing a few pain-free moments can be euphoric; it makes you realize how long youve been living with aches and pain. You might wonder how you can find a solution to relieve the pain and regain your freedom from discomfort.

Regenerative Spine and Pain Institute on how to lesson your pain.

Dr. Ronak Patel at Regenerative Spine and Pain Institute wants you to know there are two new revolutionary answers to pain relief.

Both platelet-rich therapy otherwise known as PRP and stem cell therapy give patients new hope by using the bodys powerful healing power to accelerate the battle against pain. Dr. Patel has seen incredible success implementing these cutting-edge treatments on hundreds of patients suffering from pain-related issues.

So if you are suffering fromany of the ailments below, theres a lifeline.

Heres the best news: Neither PRP or stem cell therapy involves drug use with side effects or any surgical procedures.

Both PRP and stem cell treatments use the bodys own healing resources to repair diseased or damaged tissue and the results are quite remarkable.

PRP therapy involves injecting concentrated platelets and growth factors into damaged tissue to stimulate the faster growth of new healthy cells. Platelets are cells that prevent and stop bleeding. If a blood vessel is damaged, the body sends signals to our platelets to get on the job and start the healing. Some call platelets the bodys natural bandage.

So how does PRP therapy work? Its basically drawing a one small vial of blood from the patient and then using a centrifuge to turn it into a potent and concentrated form of platelets. It is then injected back into the patient. Think of it as a boost of your own blood only superpowered.

Recovery time for PRP therapy is far shorter than for surgery. Patients usually experience soreness for a week or so, but the gradual improvement soon begins. Unlike a steroid shot, which gives you immediate relief and quickly wears off, a PRP patient will see pain symptoms improve over a period of months, and up to 80 percent of patients will see relief for up to two years.

Stem cell therapy can be an even more powerful way to harness the bodys healing power. Stem cells are the building blocks for every cell in our body. These powerful cells can be harvested to produce powerful new cells to fight inflammation and disease.

For those suffering from osteoarthritis, stem cell therapy has proven very effective. Thats because the stem cells may help develop new cartilage cells and suppress inflammation. Stem cells can be harvested through a sample of body fat or bone marrow or be harvested from donated umbilical cord tissue.

And yes, you can even augment PRP therapy with stem cell therapy for an even bigger boost!

Stop wondering if youll have to live with your pain forever. Contact Regenerative Spine and Pain Institute today at 609-269-4451 or go to http://www.njpaindoc.com to book an appointment and learn more.

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The main patient characteristics are described in Table2. Type of tumor and mobilization were similar between the groups.

Based on AIC minimization, the combined variability structure was selected. The equation of the estimated base model was AP-CD34+=1.63+1.02 (PB-CD34+)+e where e had a zero-mean normal distribution N (0, s2 (k+prediction)2).

The parameter values for intercept, PB-CD34+, k, and were 1.63 (SE=0.72), 0.12 (SE=0.01), 1.76 and 0.49, respectively.

The base model showed a satisfactory goodness-of-fit plot. In the predicted vs the observed scatterplot, the dots were well scattered around the identity line indicating unbiased model predictions. Additionally, the plot of residual values confirmed non-homogeneous variance with greater residual variability for larger predicted values of the AP-CD34+ cell counts. In terms of predictive accuracy, the base model was able to properly predict the percentage of patients achieving both 2 106 and 5 106 AP-CD34+ cells/kg (Fig.2).

AP-CD34+ cluster of differentiation 34+ cells on the first day of apheresis, CI confidence interval.

The base model can be used to characterize the necessary counts of PB-CD34+ to achieve thresholds of 2 106 and 5 106 AP-CD34+ cells/kg (Fig.3).

AP-CD34+ cluster of differentiation 34+ cells on the first day of apheresis, PB-CD34+ peripheral blood-cluster of differentiation 34.

According to the base model, an estimated PB-CD34+ counts of 57.01 (90% CI: 21.76130.76) and 125.24 (90% CI: 72.09330.71) 106/L were necessary to reach thresholds of 2 106 and 5 106 AP-CD34+ cells/kg, respectively, with a probability of 0.90.

Based on AIC minimization, the best model includes the tumor type (neuroblastoma and other) as covariate. The equation of the estimated final model was as follows:

$${{{{{{{mathrm{Neuroblastoma}}}}}}}}:{{{{{{{mathrm{AP}}}}}}}} {mbox{-}} {{{{{{{mathrm{CD}}}}}}}}34^ + = 3.01 + 0.13 times left( {{{{{{{{mathrm{PB}}}}}}}} {mbox{-}} {{{{{{{mathrm{CD}}}}}}}}34^ + } right) + e$$

$${{{{{{{mathrm{Other}}}}}}}};{{{{{{{mathrm{tumor}}}}}}}};{{{{{{{mathrm{types}}}}}}}}:{{{{{{{mathrm{AP}}}}}}}} {mbox{-}} {{{{{{{mathrm{CD}}}}}}}}34^ + = 0.01 + 0.13 times left( {{{{{{{{mathrm{PB}}}}}}}} {mbox{-}} {{{{{{{mathrm{CD}}}}}}}}34^ + } right) + e$$

where e had a zero-mean normal distribution N (0, s2 (k+prediction)2).

The parameter values for intercept-neuroblastoma, intercept-other, PB-CD34+, and were 3.01 (SE=1.10), 0.01 (SE=0.006), 0.13 (SE=0.01), (simeq) 0.00 and 0.54, respectively.

According to the model, the predicted count of AP-CD34+ cells was slightly larger for neuroblastoma tumor types than for the other tumor types. It should be noted that the final model was selected considering the type of tumor as an additional covariate (in addition to PB-CD34+) based on statistical information criterion (AIC), and that the tumor type was correlated with the age of the patients - the patients with Neuroblastoma tumor type, with mean age of 3.7 years (standard deviation, SD=2.1 years), being younger than the others with mean age of 8.9 years (SD=4.8 years). However, the choice of considering tumor type in the final model instead of age was driven by the fact that the fit of the data was improved when tumor type was considered as predictor, as compared to age, which reflected in lower value of the statistical information criterion with tumor type (AIC=288.6) than with age (AIC=310.1).

The final model also showed a good predictive property in terms of goodness-of-fit plot and prediction of the percentages of patients achieving both 2 106 and 5 106 AP-CD34+ cells/kg (Fig.4). The model predicts that a smaller PB-CD34+ cell count was needed to reach 2 106 and 5 106 AP-CD34+ cells/kg with a probability of 0.90 in patients with neuroblastoma tumor type than in those with other tumor types (Fig.5). According to the final model, in patients with neuroblastoma tumor type, the estimated PB-CD34+ counts necessary to reach apheresis thresholds of 2 106 and 5 106 AP-CD34+ cells/kg with a probability of 0.90 were 27.32 (90% CI: 0.1650.51) and 103.20 (90% CI: 56.15165.18) 106/L, respectively. The estimated PB-CD34+ counts necessary to reach thresholds of 2 106 and 5 106 AP-CD34+ cells/kg with a probability of 0.90 in patients with other tumor type were 50.51 (90% CI: 29.3079.12) and 126.39 (90% CI: 77.25198.28) 106/L, respectively.

AP-CD34+ cluster of differentiation 34+ cells on the first day of apheresis, CI confidence interval.

AP-CD34+ cluster of differentiation 34+ cells on the first day of apheresis, PB-CD34+, peripheral blood-cluster of differentiation 34+.

The uncertainty related to these PB-CD34+ estimated values with the final model was slightly less in comparison to the base model probably due to a reduced residual variability.

The physiological process of stem cell mobilization via CXCR4 is comparably the same in subjects of all ages, and when adult data on CXCR4 is extrapolated into children it should closely mirror that seen in children [19]. We complemented our analyses with data from the adult NHL and MM patients who participated in the two plerixafor studies [15, 16], focusing on the first day of apheresis similar to the MOZAIC study. The details of the analyses can be found in theSupplementary section.

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Development and validation of a predictive model to guide the use of plerixafor in pediatric population | Bone Marrow Transplantation - Nature.com

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Visiongain has published a new report entitled Mesenchymal Stem Cells Market 2022-2032. It includes profiles of Mesenchymal Stem Cells Market and Forecasts Market Segment by Type {Product (Cell & Cell Lines, Kits Media & Reagents, Others), Services}, Market segment by Source (Bone Marrow, Adipose Tissue, Cord Blood, fallopian Tube, Fetal Liver, Lung, Peripheral Blood, Other Sources), Market Segment by Indication (Bone & Cartilage Repair, cardiovascular Disease, Cancer, GvHD, Inflammatory & Immunological Diseases, Liver Diseases, Other Diseases), Market Segment by Application (Disease Modelling, Drug Discovery & Development, Stem Cell Banking, Tissue Engineering, Toxicology Studies, Other Applications) plus COVID-19 Impact Analysis and Recovery Pattern Analysis (V-shaped, W-shaped, U-shaped, L-shaped), Profiles of Leading Companies, Region and Country.

The mesenchymal stem cells market was valued at US$2.44 billion in 2021 and is projected to grow at a CAGR of 13.82% during the forecast period 2022-2032.

Rising Awareness About Therapeutic Potential of Mesenchymal Stem CellsThe mesenchymal stem cell (MSC) market has a huge potential for expansion as it's the most prevalent stem cell type used in regenerative medicine. MSCs are now the most commonly used stem cell type in clinical trials and the most researched stem cell type in the scientific literature. MSC-based therapies are also gaining popularity due to the rapidly aging population and rising prevalence of chronic diseases. Mesenchymal Stem cells play a significant role in effective management of disease and research initiatives in specialized areas such as genomic testing and personalized medicine. As a result of rising awareness of the therapeutic potential of stem cells and the scarcity of effective therapeutic treatments for rare diseases there is rise in investment leading to the growth of the market, however significant operational cost associated with the mesenchymal stem cell expansion and banking is anticipated to hinder the market growth.

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Mesenchymal Stem Cells Market Report 2022-2032

How has COVID-19 had a Significant Negative Impact on the Mesenchymal Stem Cells Market?

The biotechnology industry has experienced evolutionary changes with regards to the operational management. Typical biopharmaceutical companies manufacturing products for mesenchymal stem cell development had a better response to staff disruptions and challenges evolving due to COVID-19.

There was an impact on the research & development activities and clinical trials as there were interruptions in the new patient enrolment for the active clinical trial. However, the business focused on inventing new therapies for the treatment of COVID-19 disease. In the past years, MSCs have established itself to be an effective technique to treat pulmonary disease, including COVID-19. MSC derived stem cell therapies have showed the potential for the treatment of the Covid 19 disease. Therefore, an increase in the number of clinical trials using MSCs has been observed. Countries such as the US, the UK, Belgium, France, Spain and Mexico are conducting clinical trials with mesenchymal stem cells to be used in the treatment of COVID-19.

How will this Report Benefit you?

Visiongains 281-page report provides 117 tables and 184 charts/graphs. Our new study is suitable for anyone requiring commercial, in-depth analyses for the mesenchymal stem cells market, along with detailed segment analysis in the market. Our new study will help you evaluate the overall global and regional market for Mesenchymal Stem Cells Market. Get financial analysis of the overall market and different segments including type, Source, Indication, Application, and company size and capture higher market share. We believe that there are strong opportunities in this fast-growing mesenchymal stem cells market. See how to use the existing and upcoming opportunities in this market to gain revenue benefits in the near future. Moreover, the report will help you to improve your strategic decision-making, allowing you to frame growth strategies, reinforce the analysis of other market players, and maximise the productivity of the company.

What are the Current Market Drivers?

MSCs in the Development of Engineered Tissues and OrganshMSCs are considered as one of the prominent bio fabrication materials for decades as they are proved safe and effective in treating various injuries and diseases such as bone or cartilage regeneration, stroke & cancer. Bioprinting is a rapidly expanding tissue engineering area with a lot of promise for product customization and addressing the global tissue and organ scarcity, with a global market of $1.82 billion USD predicted by 2022. hMSCs have also been found to be capable of being guided toward hepatocyte differentiation thus indicating huge demand for hMSCs as tissue engineering of organ develops. The requirement for hMSC in engineered tissue and organ applications is, of course, reliant on cell generation, differentiation, and maturation technologies for the parenchymal cells required for organ function and thus it is expected that the increased availability of hMSC sources as a result of manufacturing technology advancements will pave the way for quick improvement and growth of the mesenchymal stem cells market.

Rise in Focus Towards Regenerative Medicine TherapiesMSCs are a good cell source for tissue regeneration because of the following characteristics. MSCs can be sourced from various tissue, including umbilical cord, fetal liver, bone marrow, and synovium. MSCs have the ability to develop into practically any end-stage lineage cell, allowing them to seed specific scaffolds. MSCs are potential immune tolerant agents as they have characteristics such as anti-inflammatory, immunoregulatory & immunosuppressive. Several clinical papers back up MSC-based cell therapy's potential efficacy; while its efficacy is still restricted, the results are encouraging.

MSCs have been investigated and used extensively in regenerative medicine. MSCs have moved closer to therapeutic applications for disease therapy and tissue repair in recent years due to improvements in extraction, culture, and differentiation procedures , therefore future research into better biomaterials and effective inducing factors will help MSCs advance in their regenerative medicine applications.

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Mesenchymal Stem Cells Market Report 2022-2032

Where are the Market Opportunities?

MSC Therapy to Treat Multiple SclerosisThe therapeutic application of MSCs in treating Multiple Sclerosis has proved to provide huge potential by improving clinical symptoms, thereby stabilizing the disease progression. MSCs have properties such as immunomodulator, tissue-protector and repair promotion has proved MSCs to be an attractive therapy option in the treatment of Multiple Sclerosis as well as in other conditions such as inflammation and tissue injury.

MSCs when administered, combat the inflammation in body and regulate the immune system which will further prevent myelin degradation. Clinical trials demonstrating the application of MSCs in Multiple Sclerosis patients have shown increased energy levels, improved flexibility, strength, and mobility. It has also been observed that if MSCs are administered intravenously may have the ability to halt diseases progression for an extended time duration.

MSCs offer intrinsic benefits over hematopoietic stem cells, that MSCs can differentiate into a cell types, release immunoregulatory molecules and promote release of exosome and growth factors

Competitive LandscapeThe major players operating in the mesenchymal stem cells market are Thermo Fischer Scientific Inc., Merck KGaA (Millipore Sigma), STEMCELL Technologoes Inc., Cytori Therapeutics Inc. (Plus Therapeutics Inc.), Cyagen Biosciences, PromoCell GmbH, Celprogen Inc. Stemedica Cell Technologies Inc., Cell Application Inc., Lonza, Celltex Therapeutics Corporation. These major players operating in this market have adopted various strategies comprising M&A, investment in R&D, collaborations, partnerships, regional business expansion, and new product launches.

Recent Developments

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Mesenchymal stem cells market is projected to grow at a CAGR of 13.82% by 2032: Visiongain Research Inc - GlobeNewswire

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