Orchard Therapeutics talks the benefits of HSC therapy – BioProcess Insider

Having raised $150 million, Orchard hopes to expand its therapy pipeline into larger indications and says its HSC approach is not limited to the vector it uses.

The firm has said the funding will also aid the launch of gene therapy Libmeldy in Europe. Last year, the firm received approval for the one-time treatment which is used to treat children with metachromatic leukodystrophy (MLD).

Though often viewed as a single technology, the delivery mechanism, the vector, and the type of cell modified in gene therapy can conjure different approaches. Libmeldy is an ex vivo Hematopoietic Stem Cell (HSC) gene therapy. The company told us HSCs are central to Orchards other products in its pipeline.

Image/iStock: CIPhotos

It also asserted that despite the use of a lentiviral vector, HSC is the facilitating technology in its gene therapy as it is not limited to the vector used.

We insert a working copy of the gene into the genome of HSCs, and once engrafted, these genetically modified cells can lead to multiple corrected cell types in the blood stream including white blood cells, red blood cells, platelets, and tissue macrophages, SVP, Leslie Meltzer told BioProcess Insider. Importantly, the progeny of HSCs can migrate into multiple organ systems including the brain and GI tract.

The firm uses a lentiviral vector where genes can be inserted, deleted, or modified. Orchard told us lentiviral vectors are an appealing choice because of its ability to stably integrate the genome and be passed on to all the progeny.

HSCs are particularly appealing because of their intrinsic ability to self-renew which means that these cells serve as the repository of stem cells is expected for the lifetime of the individual, said Meltzer.

To expand into larger indications, Chemistry Manufacturing and Controls (CMC) will be used to improve efficiency and the firm tout manufacturing as a critical step to transforming the capabilities of HSC.

We are focused on improving the HSC gene therapy manufacturing process through important technology innovations, including a scalable stable vector producing cell line, transduction enhancing compounds and a fully closed, automated drug product process.

With growing clinical data available, the firm is confident HSC gene therapy has the potential to make a durable impact in devastating disorders of the central nervous system.

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Orchard Therapeutics talks the benefits of HSC therapy - BioProcess Insider

Canine Stem Cell Therapy Market Is Projected To Reach 240.7 Million US$ By 2026 | Key Players, Market Dynamics, Market Trends, And Forecast. KSU |…

The Canine Stem Cell Therapy Market Was Valued at 129.52 million US$ in 2019 and Is Projected to Reach 240.7 million US$ By 2026, At A CAGR of 9.3 percentage During the Forecast Period. In This Study, 2019 Has Been Considered as The Base and 2020 to 2026 as the Forecast Period to Estimate the Market Size for Canine Stem Cell Therapy Market

Deep analysis about market status (2016-2019), competition pattern, advantages and disadvantages of products, industry development trends (2019-2026), regional industrial layout characteristics and macroeconomic policies, industrial policy has also been included. From raw materials to downstream buyers of this industry will be analyzed scientifically, the product value chain and sales distribution channel will be presented as well. This report will help you to establish comprehensive overview of the Canine Stem Cell Therapy Market

To avail the sample report for free: https://www.bonafideresearch.com/samplereport/93000002

The Canine Stem Cell Therapy Market can be split based on product types, major applications, and important regions.

By Cell Type: 1. Allogeneic Stem Cells 2. Autologous Stem Cells

By Investment: 1. Treatment 2. Research

By End-use: 1. Veterinary Hospitals 2. Veterinary Clinics 3. Veterinary Research Institutes

By Region 1. North America 1.1. U.S. 1.2. Canada 2. Europe 2.1. Germany 2.2. France 2.3. UK 2.4. Italy 2.5. Rest of Europe 3. Asia Pacific 3.1. China 3.2. Japan 3.3. India 3.4. Rest of Asia Pacific 4. Latin America 4.1. Brazil 4.2. Mexico 4.3. Rest of Latin America 5. Middle East & Africa 5.1. GCC 5.2. Rest of Middle East & Africa

Major Companies Present in the market

VetStem Biopharma Inc, Cell Therapy Sciences, Aratana Therapeutics Inc, Regeneus Ltd, Medivet Biologics LLC, Medivet Biologics LLC, Vetbiologics, Stemcellvet, Magellan Stem Cells, Medrego.

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Table of contents

1. Introduction 1.1. Market Scope and Segmental Definition 1.2. Assumptions & Limitation 2. Research Methodology 2.1. Research Approach & Data Sources 2.2. Forecasting Model 3. Executive Summary 3.1. Top Line Market Estimation 3.2. Future Outlook 4. Market Forces 4.1. Key Industry Drivers, Restraints and Opportunities 4.2. Industry Trends 5. Market Outlook (Current size & future market estimates) By Cell Type: 1. Allogeneic Stem Cells 2. Autologous Stem Cells

By Investment: 1. Treatment 2. Research 6. Market Outlook by Application (Current size & future market estimates) By End-use: 1. Veterinary Hospitals 2. Veterinary Clinics 3. Veterinary Research Institutes 7. Market Outlook by Regions (Current size & future market estimates) 1. North America 1.1. U.S. 1.2. Canada 2. Europe 2.1. Germany 2.2. France 2.3. UK 2.4. Italy 2.5. Rest of Europe 3. Asia Pacific 3.1. China 3.2. Japan 3.3. India 3.4. Rest of Asia Pacific 4. Latin America 4.1. Brazil 4.2. Mexico 4.3. Rest of Latin America 5. Middle East & Africa 5.1. GCC 5.2. Rest of Middle East & Africa 8. Competitive Landscape 8.1. Market Share/Market Ranking Analysis 8.2. Competitive Market Scenario (New Product Innovations, Key Strategic Moves & Partnerships, Start-ups Ecosystem) 9. Company Profiles

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Outlook on the Cell Therapy Global Market to 2027 – Opportunity Analysis and Industry Forecasts – Yahoo Finance

Dublin, Feb. 09, 2021 (GLOBE NEWSWIRE) -- The "Cell Therapy Market by Cell Type, Therapy Type, Therapeutic Area, and End User: Global Opportunity Analysis and Industry Forecast, 2020-2027" report has been added to ResearchAndMarkets.com's offering.

The global cell therapy market accounted for $7,754. 89 million in 2019, and is expected to reach $48,115. 40 million by 2027, registering a CAGR of 25. 6% from 2020 to 2027.

Cell therapy involves administration of somatic cell preparations for treatment of diseases or traumatic damages. Cell therapy aims to introduce new, healthy cells into a patient's body to replace diseased or missing ones.

This is attributed to the fact that specialized cells, such as brain cells, are difficult to obtain from human body. In addition, specialized cells typically have a limited ability to multiply, making it difficult to produce sufficient number of cells required for certain cell therapies. Some of these issues can be overcome through the use of stem cells. In addition, cells such as blood and bone marrow cells, mature, immature & solid tissue cells, adult stem cells, and embryonic stem cells are widely used in cell therapy procedures.

Moreover, transplanted cells including induced pluripotent stem cells (iPSCs), embryonic stem cells (ESCs), neural stem cells (NSCs), and mesenchymal stem cells (MSCs) are divided broadly into two main groups including autologous cells and non-autologous cells. Development of precision medicine and advancements in Advanced Therapies Medicinal Products (ATMPS) in context to their efficiency and manufacturing are expected to be the major drivers for the market. Furthermore, automation in adult stem cells and cord blood processing and storage are the key technological advancements that fuel growth of the market for cell therapy.

In addition, growth in aging patient population, The rise in cell therapy transplantations globally, and surge in disease awareness drive growth of the global cell therapy market. Furthermore, The rise in adoption of human cells over animal cells for cell therapeutics research, technological advancements in field of cell therapy, and increase in incidences of diseases such as cancer, cardiac abnormalities, and organ failure are the key factors that drive growth of the global market.

Moreover, implementation of stringent government regulations regarding the use of cell therapy is anticipated to restrict growth of the market. On the contrary, surge in number of regulations to promote stem cell therapy and increase in funds for research in developing countries are expected to offer lucrative opportunities to the market in the future.

The global cell therapy market is categorized on the basis of therapy type, therapeutic area, cell type, end user, and region. On the basis of therapy type, the market is segregated into autologous and allogenic. By therapeutics, it is classified into malignancies, musculoskeletal disorders, autoimmune disorders, dermatology, and others.

The global cell therapy market is categorized on the basis of therapy type, therapeutic, cell type, end user and region. On the basis of therapy type, the market is segregated into autologous and allogenic. By therapeutic area, it is classified into malignancies, musculoskeletal disorders, autoimmune disorders, dermatology, and others. On the basis of cell type, it is segregated into stem cell therapy and non-stem cell type. On the basis of end user, it is segregated into hospital & clinics and academic & research institutes. On the basis of region, the market is studied across North America, Europe, Asia-Pacific, and LAMEA.

Key Benefits

Story continues

The study provides an in-depth analysis of the global cell therapy market along with the current trends and future estimations to elucidate the imminent investment pockets.

Comprehensive analysis of factors that drive and restrict the market growth is provided in the report.

Comprehensive quantitative analysis of the industry from 2019 to 2027 is provided to enable the stakeholders to capitalize on the prevailing market opportunities.

Extensive analysis of the key segments of the industry helps in understanding the forms and types of cell therapy used across the globe.

Key market players and their strategies have been analyzed to understand the competitive outlook of the market.

Key Topics Covered:

Chapter 1: Introduction 1.1. Report Description 1.2. Key Benefits for Stakeholders 1.3. Key Market Segments 1.4. Research Methodology 1.4.1. Secondary Research 1.4.2. Primary Research 1.4.3. Analyst Tools & Models

Chapter 2: Executive Summary 2.1. Key Findings of the Study 2.2. Cxo Perspective

Chapter 3: Market Overview 3.1. Market Definition and Scope 3.2. Key Findings 3.2.1. Top Player Positioning 3.2.2. Top Investment Pockets 3.2.3. Top Winning Strategies 3.3. Porter'S Five Forces Analysis 3.4. Impact Analysis 3.4.1. Drivers 3.4.1.1. Technological Advancements in the Field of Cell Therapy 3.4.1.2. The Rise in Number of Cell Therapy Clinical Studies 3.4.1.3. The Rise in Adoption of Regenerative Medicine 3.4.2. Restraint 3.4.2.1. Developing Stage and Pricing 3.4.3. Opportunity 3.4.3.1. High Growth Potential in Emerging Markets 3.5. Impact of Covid-19 on Cell Therapy Market

Chapter 4: Cell Therapy Market, by Cell Type 4.1. Overview 4.1.1. Market Size and Forecast 4.2. Stem Cell 4.2.1. Key Market Trends and Opportunities 4.2.2. Market Size and Forecast, by Region 4.2.3. Market Size and Forecast, by Type 4.2.3.1. Bone Marrow, Market Size and Forecast 4.2.3.2. Blood, Market Size and Forecast 4.2.3.3. Umbilical Cord-Derived, Market Size and Forecast 4.2.3.4. Adipose-Derived Stem Cell, Market Size and Forecast 4.2.3.5. Others (Placenta, and Nonspecific Cells), Market Size and Forecast 4.3. Non-Stem Cell 4.3.1. Key Market Trends and Opportunities 4.3.2. Market Size and Forecast, by Region

Chapter 5: Cell Therapy Market, by Therapy Type 5.1. Overview 5.1.1. Market Size and Forecast 5.2. Autologous 5.2.1. Key Market Trends and Opportunities 5.2.2. Market Size and Forecast, by Region 5.2.3. Market Analysis, by Country 5.3. Allogeneic 5.3.1. Key Market Trends and Opportunities 5.3.2. Market Size and Forecast, by Region 5.3.3. Market Analysis, by Country

Chapter 6: Cell Therapy Market, by Therapeutic Area 6.1. Overview 6.1.1. Market Size and Forecast 6.2. Malignancies 6.2.1. Market Size and Forecast, by Region 6.2.2. Market Analysis, by Country 6.3. Musculoskeletal Disorders 6.3.1. Market Size and Forecast, by Region 6.3.2. Market Analysis, by Country 6.4. Autoimmune Disorders 6.4.1. Market Size and Forecast, by Region 6.4.2. Market Analysis, by Country 6.5. Dermatology 6.5.1. Market Size and Forecast, by Region 6.5.2. Market Analysis, by Country 6.6. Others 6.6.1. Market Size and Forecast, by Region 6.6.2. Market Analysis, by Country

Chapter 7: Cell Therapy Market, by End-user 7.1. Overview 7.1.1. Market Size and Forecast 7.2. Hospitals & Clinics 7.2.1. Key Market Trends and Opportunities 7.2.2. Market Size and Forecast, by Region 7.2.3. Market Analysis, by Country 7.3. Academic & Research Institutes 7.3.1. Key Market Trends and Opportunities 7.3.2. Market Size and Forecast, by Region 7.3.3. Market Analysis, by Country

Chapter 8: Cell Therapy Market, by Region 8.1. Overview 8.2. North America 8.3. Europe 8.4. Asia-Pacific 8.5. LAMEA

Chapter 9: Company Profiles 9.1. Allosource 9.1.1. Company Overview 9.1.2. Company Snapshot 9.1.3. Operating Business Segments 9.1.4. Product Portfolio 9.1.5. Key Strategic Moves and Developments 9.2. Cells for Cells 9.2.1. Company Overview 9.2.2. Company Snapshot 9.2.3. Operating Business Segments 9.2.4. Product Portfolio 9.3. Holostem Terapie Avanzate Srl 9.3.1. Company Overview 9.3.2. Company Snapshot 9.3.3. Operating Business Segments 9.3.4. Product Portfolio 9.4. Jcr Pharmaceuticals Co. Ltd. 9.4.1. Company Overview 9.4.2. Company Snapshot 9.4.3. Operating Business Segments 9.4.4. Product Portfolio 9.4.5. Business Performance 9.4.6. Key Strategic Moves and Developments 9.5. Kolon Tissuegene, Inc. 9.5.1. Company Overview 9.5.2. Company Snapshot 9.5.3. Operating Business Segments 9.5.4. Product Portfolio 9.5.5. Key Strategic Moves and Developments 9.6. Medipost Co. Ltd. 9.6.1. Company Overview 9.6.2. Company Snapshot 9.6.3. Operating Business Segments 9.6.4. Product Portfolio 9.6.5. Business Performance 9.7. Mesoblast Ltd 9.7.1. Company Overview 9.7.2. Company Snapshot 9.7.3. Operating Business Segments 9.7.4. Product Portfolio 9.7.5. Business Performance 9.8. Nuvasive, Inc. 9.8.1. Company Overview 9.8.2. Company Snapshot 9.8.3. Operating Business Segments 9.8.4. Product Portfolio 9.8.5. Business Performance 9.9. Osiris Therapeutics, Inc. 9.9.1. Company Overview 9.9.2. Company Snapshot 9.9.3. Operating Business Segments 9.9.4. Product Portfolio 9.10. Stemedica Cell Technologies, Inc. 9.10.1. Company Overview 9.10.2. Company Snapshot 9.10.3. Operating Business Segments 9.10.4. Product Portfolio

For more information about this report visit https://www.researchandmarkets.com/r/bja7iz

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Outlook on the Cell Therapy Global Market to 2027 - Opportunity Analysis and Industry Forecasts - Yahoo Finance

Australia’s best performing IVF clinics revealed – Queensland Times

Exclusive: It is often the last resort for parents desperate for a baby - and one that is not only expensive but shrouded in secrecy.

But now for the first time, would-be parents will have access to the performance rates of Australia's individual IVF clinics - allowing greater transparency about success rates.

Couples will also be able to predict their chance of having a baby through IVF by entering their age and clinical details into a calculator on the YourIVFSuccess website.

The online aid means prospective parents will be able to get the same information presented the same way about every clinic, all in one place.

The statistics underpinning the tool - which will be live from Monday - come from the University of New South Wales's (UNSW) Australian and New Zealand Assisted Reproduction Data Base which records every IVF procedure ever undertaken in Australia.

Of the 76 clinics that had been operating long enough to have their clinic success rates published, 92 per cent consented to have their results on the Website.

Couples can compare IVF clinic outcomes for the first time. Picture: iStock

Four measures of a clinics performance will be reported including the overall chance of having a baby from any egg retrieval and the chance of having a baby from the first ever egg retrieval.

For each measure the website compares the individual clinics performance to the national average success rate and it also compares success by age group given the age is such an important factor.

One in six Australian couples of reproductive age experience difficulties conceiving a child and Centre Alliance Senator Stirling Griff who led the charge for the release of the performance information said until now the industry had operated "as a secret society".

Data held by the UNSW showed in 2017 the worst performing IVF clinic in Australia had a birthrate of just 9.3 per cent, while at the top performing clinic, more than a third of women took home a baby after treatment.

Until now the identity of those clinics had been kept secret.

Women younger than 30 have a 40 per cent chance of having a baby using IVF but for women over the age of 44, the live birthrate is just 0.8 per cent for a fresh cycle and 7.8 per cent for frozen embryos.

Senator Sterling Griff led the charge for the release of the performance information. Picture: AAP

Senator Griff forced action on the issue in 2019 by proposing a law that would force clinics to publish the data.

"The reason I did it was that there were many instances of people I knew who changed clinic after a period of time and achieved success with going to a new clinic and so you start to think, why, what's the issue here?" he said.

The $500 million industry is a huge cash cow and a News Corp investigation found some providers were charging parents desperate for a child three times the Medicare fee for IVF and nearly twice the AMA fee while keeping them in the dark on performance rates.

The IVF industry took part in developing the new tool which was worked up by the UNSW and funded by the federal government.

"The YourIVFSuccess website will help people estimate their chances of success through this treatment so that they can make the decisions which are right for them," he said.

UNSW fertility expert academic Professor Georgina Chambers who developed the website said the four measures give a good overview of a clinic's average success rates.

"But clinics treat different types of patients, and therefore patients should always consult with their doctor to discuss your own chances of treatment success and what treatment is right for them," she said.

The chosen measures where developed so that they did not incentivise poor clinical practice.

"Australia is one of the safest countries in the world and we would not want to see more embryos being replaced to improve success rates or clinics only treating very good prognosis patients," she said.

Maree Pickens, the CEO of patient support group Access Australia, welcomed the new website but said her organisation would be advising patients league tables were not a fair and accurate representation of the care and service provided by all clinics.

"It's more than just success rates. An important consideration for many people is their clinic has to be accessible and convenient and offer services that allow them to fit their treatments and all the tests that they need to have into their working life, or if they've got children," she said.

Dr Luk Rombauts the president of the Fertility Society of Australia and New Zealand said it was "unbelievably hard to compare units".

"Some clinics might specialise in seeing women that have already done IVF, they failed somewhere else maybe in a cheaper clinic and now they want to pursue further treatment and if that's the type of population you're treating you of course can't expect that that clinic will have the best success rates," he said.

Fertility doctor Dr Fleur Cattrall, from Virtus Health, who has also had an IVF baby welcomed the transparency.

"Anything that will inform our patients is helpful but it doesn't replace a personal conversation with a fertility specialist taking into account all your personal fertility factors," she said.

"The website doesn't take into account the male's fertility factors, the length of time couples have been trying for or how low ovarian reserves are or if you need IVF for genetic reasons," Dr Cattrall said.

Dr Cattrall said the publishing of a league table of success rates could discriminate against those clinics that take on higher risk patients.

The site includes success rates for 2017 and 2018 but later this year will be updated with 2019 data.

From 2022 it will be publishing the clinical pregnancy rates for 2021 and will be updated every six months.

It has been a long, heartbreaking and expensive 12-year journey for Heidi and Dean Stevens to realise the dream of becoming parents.

Mrs Stevens suffered endometriosis and polycystic ovarian syndrome as well as unexplained infertility.

Six miscarriages, three rounds of IVF and five implantations later baby Elsa was born in 2019 and her little sister Sianna was born last year.

"Elsa because she was obviously frozen and Sianna because Elsa had a sister Anna (in the movie Frozen)," Mrs Stevens, 42 said.

What the Werrington Down's couple in Sydney's west learned along the way is that not all IVF clinics are the same.

Mrs Stevens' embryos had to undergo genetic testing to ensure a disease she carries - a degenerative gene known as HSP (Hereditary Spastic Paraplegia) which has a 50/50 chance of being passed onto her offspring and which has paralysing side effects - wasn't passed on.

Dean and Heidi Stevens with their daughters Elsa-Jodi, aged two, and Sianna, aged five months. Picture: Richard Dobson

But different clinics have different technology and processes for the Pre-implantation Genetic Diagnosis (PGD) which she was unaware of.

"The technology is vastly different, the previous clinic did the PGD tested on day three of the six cell embryo by extracting two cells to do the genetic testing, but of those embryos I didn't' fall pregnant," Mrs Stevens said.

The couple switched to Genea, which had a different technology for testing embryos, and had their first successful pregnancy in the first round.

"Genea didn't test until day five and the embryo was not physically touched, and the test was done on stem cells spat out by the embryo, so they never touched my embryos," Mrs Stevens said.

"My A grade embryo is Elsa today, she was never interfered with, was implanted and was my first successful pregnancy," she said.

"My miracle is here," she said through tears.

Mrs Stevens welcomes the transparency that will come with the new website.

"I think it is a compelling fundamental to the process, had I been able to pull out blanket information that compared apples with apples, I could have shortened my process and I could have honed in on my options more closely.

"Having the transparent information upfront, we would have taken a different path. It's about informed decisions and being able to look at it side-by-side is really important."

Originally published as Australia's best performing IVF clinics revealed

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Australia's best performing IVF clinics revealed - Queensland Times

Neurons from patient blood cells enable researchers to test treatments for genetic brain disease – Brown University

PROVIDENCE, R.I.[Brown University] New research provides insights into the treatment of Christianson syndrome (CS), an X-linked genetic disease characterized by reduced brain growth after birth, intellectual disability, epilepsy and difficulties with balance and speech.

One of the major challenges in developing treatments for human brain disorders, like CS, is developing an experimental system for testing potential therapeutics on human neurons, said study senior author Dr. Eric Morrow, an associate professor of molecular biology, neuroscience and psychiatry at Brown University. In recent years, advanced stem cell therapies that use tissues from patients have provided powerful new approaches for engineering human neurons from the patients themselves, who may undergo the treatment in the future.

For the study, published in Science Translational Medicine on Feb. 10, 2021, Morrow and his colleagues obtained blood samples from five CS patients and the patients unaffected brothers. They then reprogrammed these blood cells into stem cells, and these stem cells were converted into neurons in a petri dish. As a result, they obtained neurons that were representative of those from CS patients, and they used these neurons to test treatments.

Morrow who directs the Center for Translational Neuroscience at the Carney Institute for Brain Science and the Brown Institute for Translational Science said the team also used a new gene-editing approach that employs CRISPR-Cas9 technologies to correct patient mutations back to a healthy gene sequence.

CS is caused by a mutation in a gene encoding for NHE6, a protein that helps regulate acid levels within cell structures called endosomes. Past research suggests that the loss of NHE6 causes endosomes to become overly acidic, which disrupts the abilities of developing neurons to branch out and form connections in the growing brain.

Loss of this important protein can arise from a variety of gene mutations in patients. The majority of CS mutations are called nonsense mutations, which prevent NHE6 from being produced at all; four of the five CS patients involved in this study exhibited this class of mutation. However, some CS patients exhibit missense mutations. Individuals with missense mutations still have some NHE6, but it is produced in smaller amounts, and the protein fails to function as it should.

The research team tested two main forms of treatment on the stem-cell-derived neurons: first, gene transfer, which involves adding a healthy NHE6 gene into the cell; and second, administration of trophic factors, which are substances that promote neuron growth and encourage neurons to develop connections with other neurons. The researchers found that the neurons response to treatment depended on the class of mutation present.

The gene transfer studies, which may represent the first steps toward developing gene therapy, were successful in neurons with nonsense mutations. After the researchers inserted a functional NHE6 gene into nonsense-mutation CS neurons, the neurons branched out properly. In neurons with missense mutations, however, gene transfer failed completely. Further tests suggested that the abnormal NHE6 produced as a result of missense mutations may interfere with normal NHE6, thereby rendering gene transfer therapy ineffective in patient cells with these mutations.

In contrast, administration of trophic factors, such as brain-derived neurotrophic factor (BDNF) and insulin-like growth factor-1 (IGF-1), successfully promoted proper branching in all the CS neurons studied, regardless of mutation type.

While these initial results are encouraging, Morrow hopes that future studies will examine these treatments in animal models.

Our results provide an initial proof-of-concept for these treatment strategies, indicating that they should be studied further, he said. However, we may ultimately need to pay close attention to the class of mutation that a patient has when we choose a specific treatment.

In addition to Morrow, the research team included scientists from Brown University, the University of South Carolina and the Icahn School of Medicine at Mount Sinai. The study was supported by multiple grants from the National Institutes of Health as well as a number of awards from foundations and academic institutions.

This news story was authored by contributing science writerKerry Benson.

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Neurons from patient blood cells enable researchers to test treatments for genetic brain disease - Brown University

Zebrafish reveal regenerative protein that could inspire new treatments for muscle-wasting diseases and aging – FierceBiotech

Scientists in the field of regenerative medicine have long been interested in using muscle stem cells to repair injuries, but growing the cells in the lab has proven to be challenging. Now, a team of Australian researchers is suggesting an alternative: a naturally occurring protein that regenerates muscle.

A team from the Australian Regenerative Medicine Institute at Monash University discovered that a protein called NAMPT (nicotinamide phosphoribosyltransferase) stimulates the growth of muscle stem cells and healing in zebrafish and mice. They published their findings in the journal Nature.

The researchers started by studying the cells that migrated to injury sites in zebrafish. They discovered that a particular group of immune cells called macrophages stimulated the regeneration of muscle stem cells.

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Macrophages are known to migrate to injury sites, where some remove debris that appears immediately and others stay for long-term cleaning. But the Australian scientists discovered eight genetically distinct macrophagesonly one of which seemed to be involved in the regeneration of muscle stem cells.

They went on to discover that the macrophages with those regenerative abilities released NAMPT. So they tried removing the macrophages from the fish and then adding NAMPT to the aquarium water. It worked: Muscle stem cells started to grow and promote healing, showing that the protein took over for the missing macrophages, the researchers said.

RELATED: Stem cells don't repair injured hearts, but inflammation might, study finds

Several regenerative medicine research teams are focused on harnessing the healing power of macrophages. Researchers from the Cincinnati Children's Hospital Medical Center, for example, discovered that the inflammatory response to stem-cell injections into the heart activated macrophages, which in turn promoted healing.

The Monash-led research team did further studies with NAMPT, which included placing patches that contained the protein into mouse models of muscle-wasting disease. They observed significant muscle healing and are now in discussions with biotech companies about taking the technique into clinical trials, they said in a statement.

They believe NAMPT-based therapies could prove useful in treating a range of conditions including muscular dystrophy, limb injuries and muscle wasting due to aging.

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Zebrafish reveal regenerative protein that could inspire new treatments for muscle-wasting diseases and aging - FierceBiotech

Stem cell study illuminates the cause of an inherited heart disorder | Penn Today – Penn Today

Scientists in the Perelman School of Medicine have uncovered the molecular causes of a congenital form of dilated cardiomyopathy (DCM), an often-fatal heart disorder.

This inherited form of DCMwhich affects at least several thousand people in the United States at any one time and often causes sudden death or progressive heart failureis one of multiple congenital disorders known to be caused by inherited mutations in a gene called LMNA. The LMNA gene is active in most cell types, and researchers have not understood why LMNA mutations affect particular organs such as the heart while sparing most other organs and tissues.

In a study published in Cell Stem Cell, the Penn Medicine scientists used stem cell techniques to grow human heart muscle cells containing DCM-causing mutations in LMNA. They found that these mutations severely disrupt the structural organization of DNA in the nucleus of heart muscle cellsbut not two other cell types studiedleading to the abnormal activation of non-heart muscle genes.

Were now beginning to understand why patients with LMNA mutations have tissue-restricted disorders such as DCM even though the gene is expressed in most cell types, says study co-senior author Rajan Jain, an assistant professor of cardiovascular medicine and cell and developmental biology at the Perelman School of Medicine.

This story is by Sophie Kluthe. Read more at Penn Medicine News.

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Stem cell study illuminates the cause of an inherited heart disorder | Penn Today - Penn Today

Therapeutic Solutions International Acquires Stem Cell Therapy That Successfully Completed FDA Double Blind Placebo Controlled Efficacy Study for Lung…

ELK CITY, Idaho, Feb.10, 2021 /PRNewswire/ --Therapeutic Solutions International, Inc., (OTC Markets: TSOI), announced today acquisition of the JadiCell, cell therapy, for use in the treatment of acute respiratory distress syndrome and other lung pathologies.

"Having worked with the Team at Therapeutic Solutions International for over 4 years, I am glad to place our highly promising and clinically advanced stem cell therapy into this innovative and cutting-edge company," said Dr. Amit Patel, inventor of the JadiCell. "Therapeutic Solutions International is unique in that it is currently running clinical trials in the area of nutraceuticals, as well as developing preclinical and clinical stage immunotherapies. There are numerous synergies to be had with the existing work and expertise in the Company."

"While there is a lot of excitement about various approaches to lung inflammation, there are very few therapies that not only potently block pathological immunity while concurrently induce regeneration of pulmonary tissues," said Dr. James Veltmeyer, Chief Medical Officer of the Company. "To date, by far the most promising regenerative therapy our scientists have worked with for acute respiratory distress syndrome (ARDS) has been the JadiCell. I am honored to work with our team of experts such as Dr. Francesco Marincola and Dr. Santosh Kesari in leading the JadiCell through Phase III and into the hands of patients."

"It is a significant accomplishment to acquire rights to this extremely promising and cost-effective technology that is scalable and functions as a 'cellular drug,'" said Famela Ramos, Vice President of Business Development. "To our knowledge this is the only stem cell therapy for lung pathologies that does not require animal components and can be generated in sufficient quantities to address the multi-billion-dollar market of ARDS."

"Dr. Patel and his team have been strong collaborators with us since our first licensing deal using the JadiCell for Chronic Traumatic Encephalopathy," stated Timothy Dixon, President and CEO of the Company. "Having worked with these cells, we appreciate that to date they are by far the most effective at production of cytokines, stimulation of regeneration, and inhibition of pathological inflammation. We are extremely confident in our ability to take these cells to the finish line in treatment of end stage lung disease."

About Therapeutic Solutions International, Inc.Therapeutic Solutions International is focused on immune modulation for the treatment of several specific diseases. The Company's corporate website is http://www.therapeuticsolutionsint.com, and our public forum is https://board.therapeuticsolutionsint.com/

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Therapeutic Solutions International Acquires Stem Cell Therapy That Successfully Completed FDA Double Blind Placebo Controlled Efficacy Study for Lung...

The Role and Activation Mechanism of TAZ in Hierarchical Microgroove/N | IJN – Dove Medical Press

Penghui Hu,1,2,* Qian Gao,1,2,* Huimin Zheng,1,2 Yujuan Tian,1,2 Guoying Zheng,1 Xiaoyu Yao,1 Junjiang Zhang,1 Xudong Wu,2 Lei Sui1

1Department of Prosthodontics, Tianjin Medical University School and Hospital of Stomatology, Tianjin, Peoples Republic of China; 2Department of Cell Biology, 2011 Collaborative Innovation Center of Tianjin for Medical Epigenetics, Tianjin Key Laboratory of Medical Epigenetics, Tianjin Medical University, Tianjin, Peoples Republic of China

*These authors contributed equally to this work

Correspondence: Lei Sui Department of Prosthodontics, Tianjin Medical University School and Hospital of Stomatology, Tianjin, Peoples Republic of China Email suilei@tmu.edu.cn

Purpose: To investigate the role and activation mechanism of TAZ in periodontal ligament stem cells (PDLSCs) perceiving hierarchical microgroove/nanopore topography. Materials and Methods: Titanium surface with hierarchical microgroove/nanopore topography fabricated by selective laser melting combined with alkali heat treatment (SLM-AHT) was used as experimental group, smooth titanium surface (Ti) and sandblasted, large-grit, acid-etched (SLA) titanium surface were employed as control groups. Alkaline phosphatase (ALP) activity assays, qRT-PCR, Western blotting, and immunofluorescence were carried out to evaluate the effect of SLM-AHT surface on PDLSC differentiation. Moreover, TAZ activation was investigated from the perspective of nuclear localization to transcriptional activity. TAZ knockdown PDLSCs were seeded on three titanium surfaces to detect osteogenesis- and adipogenesis-related gene expression levels. Immunofluorescence and Western blotting were employed to investigate the effect of the SLM-AHT surface on actin cytoskeletal polymerization and MAPK signaling pathway. Cytochalasin D and MAPK signaling pathway inhibitors were used to determine whether actin cytoskeletal polymerization and the MAPK signaling pathway were indispensable for TAZ activation. Results: Our results showed that SLM-AHT surface had a greater potential to promote PDLSC osteogenic differentiation while inhibiting adipogenic differentiation than the other two groups. The nuclear localization and transcriptional activity of TAZ were strongly enhanced on the SLM-AHT surface. Moreover, after TAZ knockdown, the enhanced osteogenesis and decreased adipogenesis in SLM-AHT group could not be observed. In addition, SLM-AHT surface could promote actin cytoskeletal polymerization and upregulate p-ERK and p-p38 protein levels. After treatment with cytochalasin D and MAPK signaling pathway inhibitors, differences in the TAZ subcellular localization and transcriptional activity were no longer observed among the different titanium surfaces. Conclusion: Our results demonstrated that actin cytoskeletal polymerization and MAPK signaling pathway activation triggered by SLM-AHT surface were essential for TAZ activation, which played a dominant role in SLM-AHT surface-induced stem cell fate decision.

Keywords: TAZ, hierarchical micro/nanoscale topography, periodontal ligament stem cells, osteogenic differentiation, adipogenic differentiation

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The Role and Activation Mechanism of TAZ in Hierarchical Microgroove/N | IJN - Dove Medical Press

Translational Regenerative Medicine Market: Immunotherapy is projected to be the fastest growing segment during the forecast period – BioSpace

Regenerative medicine is a segment of translational research in molecular biology and tissue engineering. It involves the process of regeneration of human cells, tissues, or organs to re-establish their normal functions through stimulation of bodys repair system. They are widely used in the treatment of many degenerative disorders occurring in the areas of dermatology, orthopedic, cardiovascular and neurodegenerative diseases. Stem cell therapy is the available tool in the field of translational regenerative medicine. It has gained importance in the past few years as it is a bio-based alternative to synthetic options. Stem cells have high power of regeneration. Hence, these enable production of other cells in the body. This has increased demand for stem cell therapy in the treatment of degenerative diseases. Currently, stem cell therapy has applications in the treatment of diseases such as autism, cancer, retinal diseases, heart failure, diabetes, rheumatoid arthritis, Alzheimers. Extensive research is being carried out on stem cell therapy. The Centre for Commercialization of Regenerative Medicine (CCRM) has reported around 1900 active clinical trials undergoing currently. It also reported 574 active industry-sponsored cell therapy clinical studies, 50 of these are in phase 3 development. Hence, stem cell therapy is projected to contribute to the growth of the translational regenerative medicine market. However, ethical issues in the use of embryonic stem cells is likely to restrain the market.

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Rising prevalence of degenerative diseases, aging population, rapid growth of emerging countries, and technical advancements in developed countries are the major factors fueling the growth of the translational regenerative medicine market.

The global translational regenerative medicine market has been segmented based on product type, therapy, application, and region. In terms of product type, the market has been categorized into cellular and acellular. The cellular segment dominated the global market in 2016. Based on therapy, the global translational regenerative market has been segmented into cell therapy, gene therapy, immunotherapy, and tissue engineering. Immunotherapy is projected to be the fastest growing segment during the forecast period. In terms of application, the market has been segmented into orthopedic & musculoskeletal, cardiology, diabetes, central nervous system diseases, dermatology, and others. Cardiology and orthopedic & musculoskeletal are anticipated to be the fastest growing segments of the global translational regenerative medicine market. In terms of region, the global translational regenerative medicine market has been segmented into North America, Latin America, Europe, Asia Pacific, and Middle East & Africa. North America dominated the global regenerative medicine market owing to a large number of leading companies and expansion of research and development activities in the U.S. Increased medical reimbursement and advanced health care also drive the market in the region. Orthopedic is the leading application segment contributing to the growth of the market in the region. Asia Pacific is forecasted the huge growth because of large consumer pool, rising income, and health care expenditure. However, the market in Asia Pacific could face challenges such as high cost of bio-based medicines and stringent regulatory policies.

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The global translational regenerative medicine market is dominated by key players such as CONMED Corporation, Arthrex, Inc., Organogenesis, Inc., Nuvasive, Inc., Osiris Therapeutics, Inc., Celgene Corporation, Brainstorm Cell Therapeutics Inc. and Medtronic.

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The following regional segments are covered comprehensively:

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Translational Regenerative Medicine Market: Immunotherapy is projected to be the fastest growing segment during the forecast period - BioSpace