Category Archives: Stem Cell Medicine

Global Induced Pluripotent Stem Cell (iPSC) Market Report 2021-2028 – Increasing Demand for Body Reconstruction Procedures and Tissue Engineering -…

DUBLIN--(BUSINESS WIRE)--The "Induced Pluripotent Stem Cell (iPSC) Market Share, Size, Trends, Industry Analysis Report By Application (Manufacturing, Academic Research, Drug Development & Discovery, Toxicity Screening, Regenerative Medicine); By Derived Cell; By Region, Segment & Forecast, 2021 - 2028" report has been added to ResearchAndMarkets.com's offering.

The global Induced Pluripotent Stem Cell (iPSC) market size is expected to reach $2,893.3 million by 2028

The ability to model human diseases in vitro as well as high-throughput screening has greatly propelled market growth. Companies have effectively overcome market hurdles faced in the recent past such as proper culturing and differentiation of derived cells at a commercial scale and have developed state-of-the-art manufacturing processes that can achieve scalability and can achieve stringent quality parameters. Such trends are propelling the overall industry growth.

Companies have also developed advanced platforms for Induced pluripotent stem cells that guarantee close connection with a host of in-house technologies that are useful in the proper definition of disease signatures as well as relationships between genetic mutations as well as that properly describe perturbation of specific molecular pathways. This has resulted in the creation of human translational models that are aiding better target identification of diseases that have high unmet medical needs.

Many companies have developed transfection kits, reprogramming vectors, differentiation media, live staining kits, immunocytochemistry, among others to aid the smooth workflow of iPSC production.

However, it has been observed in the recent past that the demand for cells for screening and other purposes is significant and that there are significant challenges that pose a significant hurdle in large-scale iPSC production and differentiation.

Heavy investment in R&D activities pertaining to the development and optimization of iPSC reprogramming process in order to achieve sufficient production is a key industry trend. In the recent past, companies focused more on hepatic, cardiac, pancreatic cells, among others.

However, with the advent of a number of new participants as well as advancements and breakthroughs achieved, it is anticipated that the application portfolio will further increase in the near future.

Industry participants operating in the industry are:

Key Topics Covered:

1. Introduction

2. Executive Summary

3. Research Methodology

4. iPSC Market Insights

4.1. iPSC - Industry Snapshot

4.2. iPSC Market Dynamics

4.2.1. Drivers and Opportunities

4.2.1.1. Increasing demand for body reconstruction procedures and tissue engineering

4.2.1.2. Rising Investments across the globe

4.2.2. Restraints and Challenges

4.2.2.1. Scalability Issues

4.3. Porter's Five Forces Analysis

4.4. PESTLE Analysis

4.5. iPSC Market Industry trends

4.6. COVID-19 Impact Analysis

5. Global iPSC Market, by Derived Cell

5.1. Key Findings

5.2. Introduction

5.3. Hepatocytes

5.4. Fibroblasts

5.5. Amniotic Cells

5.6. Cardiomyocytes

6. Global iPSC Market, by Application

6.1. Key Findings

6.2. Introduction

6.2.1. Global iPSC Market, by Application, 2017 - 2028 (USD Million)

6.3. Manufacturing

6.4. Academic Research

6.5. Drug Development & Discovery

6.6. Toxicity Screening

6.7. Regenerative Medicine

7. Global iPSC Market, by Geography

7.1. Key findings

7.2. Introduction

7.2.1. iPSC Market Assessment, By Geography, 2017 - 2028 (USD Million)

8. Competitive Landscape

8.1. Expansion and Acquisition Analysis

8.1.1. Expansion

8.1.2. Acquisitions

8.2. Partnerships/Collaborations/Agreements/Exhibitions

9. Company Profiles

9.1. Company Overview

9.2. Financial Performance

9.3. Product Benchmarking

9.4. Recent Development

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

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Global Induced Pluripotent Stem Cell (iPSC) Market Report 2021-2028 - Increasing Demand for Body Reconstruction Procedures and Tissue Engineering -...

CRISPR Therapeutics and ViaCyte, Inc. to Start Clinical Trial of the First Gene-Edited Cell Replacement Therapy for Treatment of Type 1 Diabetes -…

-Initiation of patient enrollment expected by year-end-

ZUG, Switzerland and CAMBRIDGE, Mass. and SAN DIEGO, Nov. 16, 2021 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (NASDAQ: CRSP), a biopharmaceutical company focused on developing transformative gene-based medicines for serious diseases, and ViaCyte, Inc., a clinical-stage regenerative medicine company developing novel cell replacement therapies to address diseases with significant unmet needs, today announced that Health Canada has approved the companies Clinical Trial Application (CTA) for VCTX210, an allogeneic, gene-edited, immune-evasive, stem cell-derived therapy for the treatment of type 1 diabetes (T1D). Initiation of patient enrollment is expected by year-end.

With the approval of our CTA, we are excited to bring a first-in-class CRISPR-edited cell therapy for the treatment of type 1 diabetes to the clinic, an important milestone in enabling a whole new class of gene-edited stem cell-derived medicines, said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics. The combination of ViaCytes leading stem cell capabilities and CRISPR Therapeutics pre-eminent gene-editing platform has the potential to meaningfully impact the lives of patients living with type 1 diabetes.

Being first into the clinic with a gene-edited, immune-evasive cell therapy to treat patients with type 1 diabetes is breaking new ground as it sets a path to potentially broadening the treatable population by eliminating the need for immunosuppression with implanted cell therapies, said Michael Yang, President and Chief Executive Officer of ViaCyte. This approach builds on previous accomplishments by both companies and represents a major step forward for the field as we strive to provide a functional cure for this devastating disease.

The Phase 1 clinical trial of VCTX210 is designed to assess its safety, tolerability, and immune evasion in patients with T1D. This program is being advanced by CRISPR Therapeutics and ViaCyte as part of a strategic collaboration for the discovery, development, and commercialization of gene-edited stem cell therapies for the treatment of diabetes. VCTX210 is an allogeneic, gene-edited, stem cell-derived product developed by applying CRISPR Therapeutics gene-editing technology to ViaCytes proprietary stem cell capabilities and has the potential to enable a beta-cell replacement product that may deliver durable benefit to patients without requiring concurrent immune suppression.

About CRISPR Therapeutics CRISPR Therapeutics is a leading gene editing company focused on developing transformative gene-based medicines for serious diseases using its proprietary CRISPR/Cas9 platform. CRISPR/Cas9 is a revolutionary gene editing technology that allows for precise, directed changes to genomic DNA. CRISPR Therapeutics has established a portfolio of therapeutic programs across a broad range of disease areas including hemoglobinopathies, oncology, regenerative medicine and rare diseases. To accelerate and expand its efforts, CRISPR Therapeutics has established strategic collaborations with leading companies including Bayer, Vertex Pharmaceuticals and ViaCyte, Inc. CRISPR Therapeutics AG is headquartered in Zug, Switzerland, with its wholly-owned U.S. subsidiary, CRISPR Therapeutics, Inc., and R&D operations based in Cambridge, Massachusetts, and business offices in San Francisco, California and London, United Kingdom. For more information, please visit http://www.crisprtx.com.

About ViaCyte ViaCyte is a privately held clinical-stage regenerative medicine company developing novel cell replacement therapies based on two major technological advances: cell replacement therapies derived from pluripotent stem cells and medical device systems for cell encapsulation and implantation. ViaCyte has the opportunity to use these technologies to address critical human diseases and disorders that can potentially be treated by replacing lost or malfunctioning cells or proteins. ViaCytes first product candidates are being developed as potential long-term treatments for patients with type 1 diabetes to achieve glucose control targets and reduce the risk of hypoglycemia and diabetes-related complications. To accelerate and expand ViaCytes efforts, it has established collaborative partnerships with leading companies, including CRISPR Therapeutics and W.L. Gore & Associates. ViaCyte is headquartered in San Diego, California. For more information, please visitwww.viacyte.comand connect with ViaCyte onTwitter,Facebook, andLinkedIn.

CRISPR Therapeutics Forward-Looking Statement This press release may contain a number of forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including statements made by Dr. Kulkarni and Mr. Yang in this press release, as well as regarding CRISPR Therapeutics expectations about any or all of the following: (i) the safety, efficacy and clinical progress of our various clinical programs including our VCTX210 program; (ii) the status of clinical trials (including, without limitation, activities at clinical trial sites) and expectations regarding data from clinical trials; (iii) the data that will be generated by ongoing and planned clinical trials, and the ability to use that data for the design and initiation of further clinical trials; and (iv) the therapeutic value, development, and commercial potential of CRISPR/Cas9 gene editing technologies and therapies, including as compared to other therapies. Without limiting the foregoing, the words believes, anticipates, plans, expects and similar expressions are intended to identify forward-looking statements. You are cautioned that forward-looking statements are inherently uncertain. Although CRISPR Therapeutics believes that such statements are based on reasonable assumptions within the bounds of its knowledge of its business and operations, forward-looking statements are neither promises nor guarantees and they are necessarily subject to a high degree of uncertainty and risk. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties. These risks and uncertainties include, among others: the potential for initial and preliminary data from any clinical trial and initial data from a limited number of patients not to be indicative of final trial results; the potential that clinical trial results may not be favorable; potential impacts due to the coronavirus pandemic, such as the timing and progress of clinical trials; that future competitive or other market factors may adversely affect the commercial potential for CRISPR Therapeutics product candidates; uncertainties regarding the intellectual property protection for CRISPR Therapeutics technology and intellectual property belonging to third parties, and the outcome of proceedings (such as an interference, an opposition or a similar proceeding) involving all or any portion of such intellectual property; and those risks and uncertainties described under the heading "Risk Factors" in CRISPR Therapeutics most recent annual report on Form 10-K, quarterly report on Form 10-Q and in any other subsequent filings made by CRISPR Therapeutics with the U.S. Securities and Exchange Commission, which are available on the SEC's website at http://www.sec.gov. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date they are made. CRISPR Therapeutics disclaims any obligation or undertaking to update or revise any forward-looking statements contained in this press release, other than to the extent required by law.

CRISPR Therapeutics Investor Contact: Susan Kim +1-617-307-7503 susan.kim@crisprtx.com

CRISPR Therapeutics Media Contact: Rachel Eides +1-617-315-4493 rachel.eides@crisprtx.com

ViaCyte Investor Contact: David Carey, Lazar-FINN Partners +1-212-867-1768 david.carey@finnpartners.com

ViaCyte Media Contact: Glenn Silver, Lazar-FINN Partners +1-973-818-8198 glenn.silver@finnpartners.com

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CRISPR Therapeutics and ViaCyte, Inc. to Start Clinical Trial of the First Gene-Edited Cell Replacement Therapy for Treatment of Type 1 Diabetes -...

Researchers use model of hypothalamus to implicate genes associated with sleep, BMI, puberty, and more – EurekAlert

Stem cells help researchers create more complete genomic picture of difficult-to-study part of the brain involved in many key functions

Philadelphia, November 19, 2021 A study led by researchers at Childrens Hospital of Philadelphia (CHOP) has implicated several genes involved in a variety of bodily functions associated with the hypothalamus, a notoriously difficult-to-study region of the brain. The findings could help clinicians identify potential causes of dysfunction for many important traits regulated by the hypothalamus, such as sleep, stress, and reproduction.

The findings were published today in the journal Nature Communications.

The hypothalamus helps maintain health and stable metabolism by influencing a variety of vital functions, including appetite and thirst, puberty and reproductive timing, sleep cycles, and body temperature. However, the hypothalamus is located in the center of the brain, making it extremely difficult to study the gene regulation associated with these traits.

To overcome that hurdle, the researchers used an embryonic stem cell (ESC) model to study gene expression during development of the hypothalamus. This model allowed them to study the genetic architecture first in hypothalamic progenitor cells cells prior to their full development into a hypothalamus and then in arcuate nucleus-like hypothalamic neurons. The hypothalamus contains several different subtypes of neurons, and the researchers integrated results from various genome-wide association studies (GWAS) to implicate genes driving particular traits regulated by the hypothalamus.

By studying the three-dimensional genomic architecture of these cell models, we can see the dynamic process of how the hypothalamus is formed over different stages of development, said senior study author Struan F.A. Grant, PhD, Director of the Center for Spatial and Functional Genomics and the Daniel B. Burke Endowed Chair for Diabetes Research at CHOP. The information we yielded in this study provides us with more concrete information about diseases that are relevant to hypothalamic function.

Grant and his collaborators assessed variants associated with puberty, body mass index, height, bipolar disorder, sleep, and major depressive disorder, among others. They identified both known and novel genes associated with these traits. For example, their data confirmed the role of the BDNF of gene in influencing body mass index and obesity risk. Another gene of interest identified in the study was PER2, which was implicated in sleep regulation.

All the data ascertained from this study will be made publicly available. Many of the disorders studied can be caused by other factors, so the findings will help researchers distinguish which genes play a more central role in this tissue and in turn inform clinical practice. For example, body mass index can be affected by variants in genes conferring their effects in hypothalamus or fat tissue cells, so being able to distinguish the context in which genes and subsequent tissues or hormones operate can lead to more personalized treatment options.

The data set we derived from this study allows other researchers to determine which diseases or conditions are relevant when doing a genetic workup of the patient, Grant said. As more information about the hypothalamus is known, that information can be queried against this data set and potentially identify therapeutic targets for multiple disorders.

This research was supported by the National Center for Research Resources (UL1RR024134) and the National Center for Advancing Translational Sciences (UL1TR000003). This research was also supported in part by the Institute for Translational Medicine and Therapeutics (ITMAT) Transdisciplinary Program in Translational Medicine and Therapeutics, the Eunice Kennedy Shriver National Institute of Child Health (NIH1K99HD099330-01), and National Institutes of Health grants DK52431-23, P30DK026687-41, R01 HD056465, R01 HG010067, R01 HL143790, and the Daniel B. Burke Endowed Chair for Diabetes Research.

Pahl et al, Cis-regulatory architecture of human ESC-derived hypothalamic neuron differentiation aids in variant-to-gene mapping of relevant complex traits. Nat Comm. Online November 19, 2021. DOI: 10.1038/s41467-021-27001-4.

About Childrens Hospital of Philadelphia: Childrens Hospital of Philadelphia was founded in 1855 as the nations first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals, and pioneering major research initiatives, Childrens Hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country. In addition, its unique family-centered care and public service programs have brought the 564-bed hospital recognition as a leading advocate for children and adolescents. For more information, visit http://www.chop.edu

Nature Communications

Experimental study

Lab-produced tissue samples

Cis-regulatory architecture of human ESC-derived hypothalamic neuron differentiation aids in variant-to-gene mapping of relevant complex traits

19-Nov-2021

Disclaimer: AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert system.

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Researchers use model of hypothalamus to implicate genes associated with sleep, BMI, puberty, and more - EurekAlert

COVID-19: Researchers warn against overhyping early-stage therapies – Medical News Today

The COVID-19 pandemic has created a sense of urgency to generate new drugs and vaccines. In many cases, this urgency became a regulatory opportunity to bypass established regulatory pathways for new drugs.

While this has led to the fast emergence of many useful drugs and vaccines for COVID-19, it has also led to a general reduction in the quality of medical research from which to derive conclusions.

For example, according to Janet Woodcock, former director of the Food and Drug Administrations (FDA) Center for Drug Evaluation and Research, an FDA analysis found that 6% of clinical trials are yielding results the agency deems actionable.

The lack of regulation coupled with a sense of urgency has also led to overhype and rushed development of certain treatments, including cell-based therapies often sold as stem cell treatments.

While some of these products have undergone well-designed, adequately controlled trials, most are in the early stages. Some clinics are nevertheless offering these unproven and unlicensed treatments to people, promising to boost their immune system or overall health to protect against COVID-19.

Promoting and selling unproven and unlicensed treatments can harm public health and could lead many to undergo untested and potentially harmful treatments.

Recently, a group of researchers from the University of California, Irvine, the Georgia Institute of Technology, the University at Buffalo, NY, and the University of Melbourne in Australia, published a report outlining misinformation around cell-based treatments for COVID-19, calling for their stronger regulation.

Efforts to rapidly develop therapeutic interventions should never occur at the expense of the ethical and scientific standards that are at the heart of responsible clinical research and innovation, said Dr. Laertis Ikonomou, assistant professor of Oral Biology at the University at Buffalo, and co-author of the study.

Scientists, regulators, and policymakers must guard against the proliferation of poorly designed, underpowered, and duplicative studies that are launched with undue haste because of the pandemic, but are unlikely to provide convincing, clinically meaningful safety and efficacy data, said co-author Dr. Leigh Turner, professor of Health, Society and Behavior at the University of California, Irvine.

The researchers published their report in Stem Cell Reports.

Researchers conducted a study in August 2020 of 70 clinical trials involving cell-based treatments for COVID-19. They found that most were small, with an average of 51.8 participants, and only 22.8% were randomized, double-blinded, and controlled experiments.

The authors concluded that the cell-based interventions for COVID-19 were likely to have a relatively small collective clinical impact.

Cell-based treatments for COVID-19 are still at an experimental stage, Dr. Ikonomou told Medical News Today. There are tens of clinical trials, of varied complexity and rigor, that evaluate various cell types, such as mesenchymal stromal cells, for COVID-19 treatment.

Expanded or compassionate use of cell-based interventions has also been reported, but these individual cases are unlikely to tell us whether and how cell therapies could help with COVID-19 and do not substitute for the systematic clinical evaluation of cell-based products, he added.

A few completed phase 1/2 trials have shown a favorable safety profile, but larger size trials are required. Eventually, properly-powered, controlled, randomized, double-blinded clinical trials will help determine whether cell-based treatments are a viable therapeutic option for COVID-19 and its complications, he explained.

The urgency of the pandemic has made it easy to exaggerate early-stage research. The scientists highlight this is especially the case in press releases, where media professionals can over-hype findings and understate or omit limitations to gain more media coverage.

The researchers also say that even when online media include limitations and key aspects of studies, other communication channels can strip these away easily. What is left then gets amplified, as the public is desperate to see positive news.

To address this, the researchers say science communicators should ensure they have an accurate understanding of the information they report and highlight the required steps for the science to advance without exaggerating its speed.

The researchers also say that simply feeding the public more information in what is known as the information deficit model alone is insufficient. They also suggest science communicators should strive for an engaged or dialogue-based communication approach.

Over-hyping of promising treatments and in particular cell-based treatments has been a longstanding problem, and it did not first emerge with the COVID-19 pandemic, said Dr. Ikonomou. It has become a salient issue during these times due to the global nature of this health emergency and the resulting devastation and health toll.

Therefore, it is even more important to communicate promising developments in COVID-19-related science and clinical management [responsibly]. Key features of good communication are an accurate understanding of new findings, including study limitations and avoidance of sensationalist language, he explained.

Realistic timeframes for clinical translation are equally important as is the realization that promising interventions at preliminary stages may not always translate to proven treatments following rigorous testing, he added.

The researchers say that commercial investments by biotechnology companies to develop cell-based therapies for COVID-19 have led to well-designed and rigorous clinical trials.

However, some other businesses have overlooked the demanding process of pre-marketing authorization of their products. Instead, they made unsubstantiated and inaccurate claims about their stem cell products for COVID-19 based on hyperbolic reporting of cell-based therapies in early testing.

Some clinics advertise unproven and unlicensed mesenchymal stem cell treatments or exosome therapies as immune boosters that prevent COVID-19 and repair and regenerate lungs.

Often, these businesses make their treatments available via infusion or injection. However, one anti-aging clinic in California shipped its kits to clients, where they were to self-administer with a nebulizer and mask.

Such companies often market stem cell treatments via online and social media. In an initial review of many of these brands, the researchers could not find published findings from preclinical studies and clinical trials to support their commercial activities.

Instead, they found that these companies drew from uncritical news media reports, preliminary clinical studies, or case reports in which those diagnosed with COVID-19 received stem cell interventions.

Promoting such therapies that have not undergone proper tests for safety and efficacy have the potential for significant physical and financial harm.

Health experts have documented adverse events due to unlicensed stem cell products, including vision loss and autoimmune, infectious, neurological, and cardiovascular complications.

Early in the pandemic, scientific and professional societies, including the Alliance for Regenerative Medicine and the International Society for Stem Cell Research, have warned the public against businesses engaged in the marketing of cell-based treatments that have not undergone adequate testing.

The researchers highlight that it is unclear whether these warnings reached individuals and their loved ones or significantly affected public understanding of the risks of receiving unlicensed and unproven stem cell treatments for COVID-19.

They also indicate that it is unclear whether these societies and organizations have an important role in convincing regulatory bodies to increase enforcement in this space. Nevertheless, at the time of writing, the FDA and Federal Trade Commission have issued 22 letters to businesses selling unproven and unlicensed cell-based therapies.

And while many of these companies have ceased market activity, the presence of other companies continuing to pedal the same claims makes it clear that regulatory bodies must increase their enforcement.

Additionally, the researchers question whether warning letters are sufficient to disincentivize clinicians and others to sell unlicensed products. They write:

If companies and affiliated clinicians are not fined, forced to return to patients whatever profits they have made, confronted with criminal charges, subject to revocation of medical licensure, or otherwise subject to serious legal and financial consequences, it is possible that more businesses will be drawn to this space because of the profits that can be generated from selling unlicensed and unproven cell-based products in the midst of a pandemic.

The researchers conclude that regulators should increase enforcement against unproven and unlicensed therapies for COVID-19.

They also say that science communicators should report on scientific claims more realistically and include the public in more discourse.

In the U.S. and elsewhere, there are regulations and enforcement mechanisms that deal with harms caused by unproven and unlicensed cell-based interventions and false advertising claims, said Dr. Ikonomou. It may be preferable to implement existing regulations more vigorously than introduce new ones.

Stakeholders, such as scientific, professional, and medical associations, can contribute towards this goal with reporting and monitoring of cell therapy misinformation. There is a shared responsibility to combat cell-therapy related misinformation and disinformation that undercuts legitimate research and clinical efforts and portrays unproven interventions as silver bullets for COVID-19, he concluded.

For live updates on the latest developments regarding the novel coronavirus and COVID-19, click here.

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COVID-19: Researchers warn against overhyping early-stage therapies - Medical News Today

Albert Einstein Cancer Center researcher receives NCI Outstanding Investigator Award to study two deadly blood diseases – EurekAlert

image:Dr. Ulrich G. Steidl view more

Credit: Albert Einstein College of Medicine

October 27, 2021(BRONX, NY)Ulrich G. Steidl, M.D., Ph.D., co-director of the Blood Cancer Institute and associate director of basic science at the Albert Einstein Cancer Center (AECC), has received a prestigious Outstanding Investigator Award from the National Cancer Institute (NCI). This award is accompanied by a seven-year, $7 million grant to study the molecular and cellular mechanisms that lead to two related blood diseases, myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). Dr. Steidl is one of only 17 recipients of this award in 2021, which is given to accomplished leaders in cancer research who provide significant contributions in their field. The ultimate goal of this research is to develop new treatments and cures for these usually fatal disorders.

Clinical outcomes in MDS and AML have not significantly improved over the past half-century, and cure rates remain below 15% for most patients, said Dr. Steidl, who is also professor of cell biology and of medicine and the Diane and Arthur B. Belfer Faculty Scholar in Cancer Research at Einstein. There is an urgent need to improve our understanding of how these diseases develop and to devise more effective therapies.

MDS and AML Explained

MDS occurs when blood-forming (hematopoietic) stem cells in the bone marrow acquire genetic and non-genetic irregularities, leading to the production of abnormal, dysfunctional blood cells, which out-compete healthy cells. Common symptoms include anemia, infections, and bleeding.

The incidence of MDS in the United States is unclear, with estimates ranging from 10,000 to 40,000 new cases annually; about one-third of MDS patients will go on to develop AML. Treatment for MDS is generally limited to preventing or reducing complications, particularly severe anemia. The only cure is a bone-marrow transplanta therapy not easily tolerated and therefore often reserved for the youngest, most resilient patients. However, most people diagnosed with MDS are elderly.

AML, like MDS, begins with abnormal bone marrow stem cells. But in AML, those cells, after becoming cancerous, proliferate rapidly and quickly spread to the blood and other hematopoietic organs, such as the bone marrow and spleen, and sometimes to other tissues, causing many of the same symptoms seen in MDS, plus others. AML is often fatal within just a few months and afflicts about 21,000 Americans each year. It is usually treated with chemotherapy. Bone-marrow transplantation can cure AML in some patients.

From Stem Cells to Cancer

Recent studies led by Dr. Steidl and his research team have shown that both MDS and AML arise from pre-leukemic stems cells (pre-LSCs), a subpopulation of blood-forming stem cells that have genetic and non-genetic aberrations. Certain varieties (clones) of these pre-LSCs go on to develop into leukemic stem cells (LSCs)cancer cells that are capable of self-renewal. These LSCs lead to sustained leukemia growth and are particularly resistant to drugs. We now know that the considerable diversity of pre-LSC clones affects the development, progression, and treatment resistance of both MDS and AML, said Dr. Steidl, one of the nations leading authorities on both diseases.

What causes some pre-LSCs but not others to become leukemic is not clear, but transcription factors are thought to play a key role. Transcription factors are proteins that turn specific genes on or off, determining a cells function by regulating the activity of genes. In the case of stem cells, transcription factors guide their differentiation into mature cells. Our recent work has shown that the actions of key transcription factors are dysregulated in pre-LSCs and LSCs, meaning that the transcription factors and the molecular programs they govern behave abnormally, he added.

Thanks to his new NCI grant, Dr. Steidl hopes to:

To accomplish these goals, Dr. Steidls research team will employ novel tools for analyzing stem cell clones in patients, as well as newly developed mouse models of pre-LSC progression to MDS and AML.

Developing New Cancer Therapies

The knowledge we gain from this research should enable us to develop drugs that target pre-LSCs and their aberrant transcription factors, said Dr. Steidl. Such an approach holds the promise of achieving lasting remissions and, ultimately, even cures. Hopefully, our understanding of the early events in the progression of MDS and AML may even allow us in the future to prevent these diseases by interrupting the transformation of pre-LSCs to LSCs before overt leukemia can occur.

The grant (R35CA253127) is titled Molecular and Cellular Regulation of Pre-Leukemic Stem Cells and their Therapeutic Targeting.

***

About Albert Einstein College of Medicine

Albert Einstein College of Medicine is one of the nations premier centers for research, medical education and clinical investigation. During the 2020-21 academic year, Einstein is home to 721 M.D. students, 178 Ph.D. students, 109 students in the combined M.D./Ph.D. program, and 265 postdoctoral research fellows. The College of Medicine has more than 1,900 full-time faculty members located on the main campus and at its clinical affiliates. In 2020, Einstein received more than $197 million in awards from the National Institutes of Health (NIH). This includes the funding of major research centers at Einstein in aging, intellectual development disorders, diabetes, cancer, clinical and translational research, liver disease, and AIDS. Other areas where the College of Medicine is concentrating its efforts include developmental brain research, neuroscience, cardiac disease, and initiatives to reduce and eliminate ethnic and racial health disparities. Its partnership with Montefiore, the University Hospital and academic medical center for Einstein, advances clinical and translational research to accelerate the pace at which new discoveries become the treatments and therapies that benefit patients. Einstein runs one of the largest residency and fellowship training programs in the medical and dental professions in the United States through Montefiore and an affiliation network involving hospitals and medical centers in the Bronx, Brooklyn and on Long Island. For more information, please visit einsteinmed.org, read our blog, followus on Twitter, like us on Facebook, and view us on YouTube.

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Albert Einstein Cancer Center researcher receives NCI Outstanding Investigator Award to study two deadly blood diseases - EurekAlert

Medical Innovation In Pet Healthcare Is Taking Things Up a Notch – Entrepreneur

Opinions expressed by Entrepreneur contributors are their own.

You're reading Entrepreneur India, an international franchise of Entrepreneur Media.

India is currently home to over 10-plus million pets. Over time, pet parents have realized the importance of medicine and innovation and the change it can bring about in a pets life. Pet innovation today is booming across treats, food, grooming, wearables, insurance, online veterinary services, genetic testing, stem cell therapy, diagnostics and much more. According to PETEX INDIA 2021, the pet food market alone is projected to cross $310 million by FY22. The pet care industry is globally expanding horizontally and vertically with innovative and challenging minds at work. Medical innovation has brought about effective growth thus making way for new products, services and medicines for pets. Medical procedures such as lasers for joint pains and joint care have helped pet owners make the lives of their furry babies a little less painful.

Unsplash

A few medical innovations in India have particularly taken the Indian pet care sector a notch higher:

CBD & Hemp Seed Oil For Overall Health & Wellness

CBD, or cannabidiol, is a chemical found in the cannabis sativa plant and is known to have wonderful pain-relieving properties. CBD is one of the most effective natural therapies for dogs suffering from arthritis and many other diseases. It is safe and has no side effects. CBD oil interacts with the cells in the muscle, skin and nerves of pets thereby reducing their pain. CBD and hemp seed oils are now available in the form of oil, shampoos and treats. It also benefits pets with anxiety issues triggered due to loud noises, people or travel and keeps them calm and relaxed. CBD oil is finding an innovative use in health and wellness based products for pets and its multiple visible benefits.

Omega 3 and Omega 6 fatty acids in hemp seed oil help control skin breakouts and protect the skin.

They are responsible for creating a healthy cell membrane and help to avoid excessive fur shedding. Hemp serves as the optimum plant-based nutrition that is easily digestible and includes antioxidants that aid elevated health in pets. Hemp seed oil has recently been added to pet food and treats and is also being used in healing balms for dry paws and skin.

Prebiotics & Probiotics For Gut Health

Probiotics are the billions of good bacteria that live in the gastrointestinal tract of animals. Pets respond to supplements in the form of immunity boosters and probiotic strips are easy dissolving strips unlike pills, powders or tablets which the pet might refuse to intake. Probiotics treat diarrhoea, stomach related issues, irritable bowel syndrome, improve digestive health, prevent anxiety, reduce stress and improve general health and well-being. Given its health benefits, prebiotics and probiotics have found their way in pet food as they help to maintain the pH balance in the gut and keep the digestive tract healthy. Probiotics have also recently been introduced in shampoos as they help in supporting a healthy microbiome and defend against common skin problems such as itching dryness, dandruff, hot spots, excessive shedding and yeast in pets.

Assistive Devices To Aid Mobility

Harnesses and slings for dogs suffering from hip dysplasia are gaining popularity among vets and pet parents. These act as a wheel-chair and support the pets rear body and enable them to move around. They also help pets who have lost their hind legs in accidents. Harnesses for pets who are blind have been created such that they form a halo around their head and this will alert the pet if they happen to bump against any object. These innovations may alter the pets appearance but what is important is that it helps pets to live a normal life. The same goes for pets afflicted with arthritis. Elevated feeding stations, ramps to reduce the stress on joints have also become common to households who have aging pets.

Veterinary Diagnostics

Innovation in the use of technology that equips veterinarians to view real-time information on internal bone and muscle structure is in the fray. This will help to speed up the prototype required to create an animal bone model that is generated using the tomography scan technology. This technology is building steam though still in its nascent stage. Two new blood types, Langereis and Junior, have been identified in pets apart from the 12 dog blood types. New diagnostic techniques through tests which can help to diagnose the possibility of diseases in pets, confirm or even classify disease status in pets, is the need of the hour.

According to a study by Grand View Research on veterinary medicine, the global veterinary medicine market size was estimated at $29.2 billion in 2020 and is expected to expand at a CAGR of 7.4 per cent from 2021 to 2028. Steady medical innovation is solving unsolved and unattended problems that pets have faced since decades. While the offline gap has been filled in by the online D2C revolution in pet care and wellness, advancements in medical innovation remain unhindered despite the pandemic.

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Medical Innovation In Pet Healthcare Is Taking Things Up a Notch - Entrepreneur

San Diego research centers receive $15 million to train next generation of scientists – Three San Diego research institutions have been awarded nearly…

San Diego research centers receive $15 million to train next generation of scientists

San Diego Community News Group

Three San Diego research institutions have been awarded nearly $15 million from the California Institute of Regenerative Medicine (CIRM) to train the next generation of scientists in regenerative medicine, a field of research that holds great promise for generating transformative medicines.

Scripps Research, University of California San Diego, and Sanford Burnham Prebys each received grants of around $5 million from CIRM to support the training of graduate students, postdoctoral trainees, and clinical trainees.

The CIRM awards will also foster interdisciplinary regenerative medicine collaborations among the three San Diego recipient institutions and support outreach science activities in the broader San Diego community. The funding also will support educational programs for K-12 students and undergraduates on topics related to regenerative medicine that are intended to reduce disparities and disproportionate access to science.

"CIRM has provided critical leadership spearheading regenerative medicine and stem cell research," says Peter Schultz, president and CEO of Scripps Research. "This forward-looking investment in training the next generation of scientists will help ensure continued progress toward realizing the tremendous promise of regenerative medicine."

Schultz will head the Scripps Research program which received $4,931,353 to train scientists in disciplines and techniques central to stem cell biology and regenerative medicine. These include cellular processes involved in human embryonic and adult stem cell self-renewal and differentiation and the development of novel drugs to target related pathways.

University of California San Diego received $4,992,446 to train the next generation of stem cell biologists, driving advances ranging from the unraveling of fundamental mechanisms of cell function to the development of new therapies. The UC San Diego training program will be led by Alysson R. Muotri, PhD, professor of pediatrics and cellular and molecular medicine at UC San Diego School of Medicine, and Sheldon Morris, MD, primary care physician at UC San Diego Health.

This grant comes at a time when stem cell research in San Diego has matured, thanks to strong support over the years from visionaries such as CIRM and philanthropist T. Denny Sanford, Muotri said. The only way to keep up this momentum is to have professionals that understand how to use and apply stem cell and gene therapies. We are building the next generation of researchers and clinicians to do this.

Sanford Burnham Prebys received $4,931,353 to launch a multidisciplinary stem cell training program. The grant will provide funds for competitive awards for PhD students, postdocs and clinical fellows in stem cell, gene therapy and regenerative medicine fields at the institute. The training program will be led by Evan Snyder, MD, PhD, professor and director of the Center for Stem Cells & Regenerative Medicine, as well as professors/directors Pier Lorenzo Puri, MD, and Alessandra Sacco, PhD, in the Development, Aging and Regeneration Program Center at Sanford Burnham Prebys.

Sacco says stem cell research holds tremendous promise for medical treatments, and that CIRMs support will allows scientists to learn more about the process through which science becomes medicine.

We are exceptionally grateful that CIRM is supporting this important program, says Sacco. This award helps the next generation of stem cell and regenerative medicine scientists who will work across boundaries and between disciplines to become capable of translating basic discovery science into clinical research for patient benefit.

This years awards build on an earlier Research Training program through which CIRM supported training in regenerative medicines from 2006-2016 and trained 940 CIRM Scholars including 321 doctoral students, 453 postdocs and 166 MDs.

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San Diego research centers receive $15 million to train next generation of scientists - Three San Diego research institutions have been awarded nearly...

UC Davis Medical Center faculty respond to the rise of treatments like Merck’s anti-COVID pill – The Aggie – The Aggie

Mercks pill, molnupiravir, shows reductions of hospitalizations and deaths by 50%

By BRANDON NGUYEN science@theaggie.org

Over a year and a half into the pandemic, with sweeping changes to the standard of living along with distressing upward trends of illness, hospitalizations and deaths, recent developments of treatments such as Mercks anti-COVID pill offer hope toward a return to the status quo.

According to an article in The Sacramento Bee, Pharmaceutical company Merck took the internet by storm when it announced Friday morning that findings from a recent study showed its experimental oral pill molnupiravir reduced COVID-19 related hospitalizations and deaths by 50%.

Dr. Christian Sandrock, a trained infectious disease pulmonary critical care doctor at the UC Davis Medical Center, discussed the current state of the pandemic as being dominated by downward trends in new cases by about 20% from where it was two weeks ago. However, he highlighted that hot spots of cases remain in certain states and areas within the U.S. where vaccination rates are not as high as in California.

With the advent of continuing trials for new treatments against COVID-19, a multi-faceted approach is necessary to reduce the impact of the pandemic, according to Dr. Rachael Callcut, the division chief of trauma and acute care surgery at the UC Davis Medical Center. Part of this approach, aside from initial prevention of the disease and treating the virus itself, includes risk mitigation and decreasing the chances of hospitalization by preventing the development of severe illness arising from the coronavirus.

This particular oral agent is targeted at people who are at risk of developing severe illness from the virus, and it is something to be given early in the course of illness to try to prevent the development of complications, Callcut said. I would say that this is sort of analogous to the approaches that you see with things like pills that we give to patients who have influenza to try to prevent them from developing complications of influenza.

Mercks study is important to altering the current landscape of the pandemic and its ferocious impact on the global population in the past year. In the same vein, Sandrock summarized the promising results of the pill and its significance proceeding toward a hopeful end to the pandemic.

The data looks promising: the trial had just about a little over 350 people in each arm so, not whopping numbers but still reasonably high, and the key is the major endpoints of hospitalization and death were certainly down, Sandrock said. So I think that the big thing is it can be taken orally, and this changes the landscape to something that is a single pill.

Other current treatments include the administration of monoclonal antibodies to the patient to combat the virus, and maintaining a steady supply of this product is relatively more costly in terms of price and time, according to Sandrock. For individuals who live further away and have little to no access to healthcare, reducing hospitalizations is key to facilitating a return to the status quo. With Mercks anti-COVID pill, it has the potential to drastically reduce the burdens of both the patient and the medical professional.

Fighting the COVID-19 pandemic has fostered collaboration among drug developers, academic medical and research centers as well as supportive funding agencies, Dr. Allison Brashear, the dean of the UC Davis School of Medicine, said. UC Davis School of Medicine, renowned for its research expertise, is a trusted partner on many COVID-19 clinical trials at the forefront of efforts to cure this terrible disease.

Callcuts lab is also involved in one of the many ongoing clinical trials, working with mesenchymal stromal cells, cells that are stem-cell like that aid in regeneration of damaged lung cells and reducing inflammation associated with acute respiratory disorders caused by viruses like COVID-19.

Hope is on the horizon for individuals actively fighting the pandemic with the race toward developing treatments attacking all realms of the virus. Callcut echoed this sentiment, encouraging the continual efforts in enforcing public health safety and support.

Theres got to be a multi-faceted approach that resides on the principles of our public health initiatives, and the first is prevention of illness, Callcut said. One of the most effective strategies for that is vaccinations, specifically investing in increasing vaccination education and administering vaccines. These types of measures will be our most important defenses in leading to the end of the pandemic. Written by: Brandon Nguyen science@theaggie.org

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UC Davis Medical Center faculty respond to the rise of treatments like Merck's anti-COVID pill - The Aggie - The Aggie

Managing superficial pyoderma with light therapy – DVM 360

Phovia is highly effective for treating superficial and deep skin infections.

This article is sponsored by Vetoquinol.

Superficial bacterial folliculitis, also called superficial pyoderma, is a commonly diagnosed dermatological condition in dogs.1,2 These infections are secondary to primary conditions affecting normal skin barrier function (eg, allergic skin disease, trauma, burns), keratinization (eg, nutritional deficiency, liver disease), and immune regulation (eg, neoplasia, autoimmunity, endocrinopathy).2 Cats less commonly develop superficial pyoderma perhaps because of decreased adhesion of staphylococci to feline corneocytes, but the primary issues causing infection are similar to those seen in dogs.3-8

The primary pathogen associated with superficial pyoderma in dogs and cats is a normal resident of the skin, Staphylococcus pseudintermedius, but other flora may be involved.2,8-12 As the normal homeostasis of this organism is disrupted from a primary disease, these gram-positive cocci invade deeper regions of the epidermis and hair follicle epithelium, increase in number, and enhance inflammation.

Classical clinical lesions of superficial pyoderma include papules and pustules that may eventually progress to alopecia, epidermal collarettes, scales, and crusts. Often the skin is erythematous and pruritic. Chronic cases may demonstrate lichenification, hyperpigmentation, and scarring alopecia from long-standing inflammation and infection.2 Cats may develop even more unique cutaneous reaction patterns and skin lesionsespecially when allergic skin disease is presentincluding miliary dermatitis, eosinophilic plaques, rodent ulcers, and eosinophilic granulomas.5

Identifying and addressing the primary disease is paramount in achieving complete, permanent resolution of the superficial pyoderma. Therefore, treatment is multifactorial and aimed at addressing the primary disease, reducing skin inflammation, and treating the infection directly. Current guidelines for the treatment of superficial pyoderma in dogs recommend the use of topical antimicrobials as sole therapy whenever possible; however, overuse of systemic antibiotics remains common.2,13-16

Topical therapy has many benefits including direct antimicrobial effects without use of an antibiotic, reduction in antibiotic-resistant bacterial populations, restoration of the normal skin barrier, enhancement of skin hydration, physical removal of keratinous debris, and removal of offending allergens from the haircoat.2,14 However, topical therapy is met with challenges that impede clinical application. Adherence is the biggest concern when recommending topical therapy to pet owners. Frequent bathing or application of medicated solutions to the skin can be difficult when busy owner lifestyles combine with a nonadherent patient. Skin inflammation can be painful and animals may be resistant to topical therapy. Cats are fastidious groomers and may lick away a medicated topical therapy before it can achieve appropriate contact time. Additionally, some topical agents can cause oral erosions and ulcerations or even gastrointestinal disturbance when groomed off. For these reasons, systemic antibiotics continue to be a common prescribing practice for superficial pyoderma.

All antibiotic use, despite duration or frequency, contributes to the development of antibiotic-resistant bacterial populations on the animal and in the environment.17-19 From that very first dose, bacteria are constantly evolving to implement inherent and acquired resistance mechanisms necessary for survival. One well-recognized mechanism is oxacillin resistance through the mecA gene, which produces a penicillin-binding protein receptor with poor affinity for -lactam antibiotics.2,14,15,20-23 Even more concerning than these oxacillin-resistant strains are those that develop multidrug resistance, which is defined as resistance to 3 or more antibiotic drug classes. This may happen over time with repeated antibiotic exposure or after a single dose of certain antibiotics such as fluorinated quinolones.2,20,23-25 The continued emergence of antibiotic-resistant bacteria inhibits the successful treatment of bacterial infections in pets and humans. As veterinarians consider how their antibiotic use contributes to this growing pandemic, they must look for alternative, safe, effective, affordable, and convenient antibacterial treatment modalities.

Phovia as a solution

Investigation into the photobiological effects of light therapy has been ongoing for the past 50 years. Photobiomodulation (PBM) therapy is a type of light treatment that uses visible or near infrared light to promote therapeutic benefits including induction of tissue healing and regeneration and inhibition of biological responses that induce pain or inflammation. The treatment distance, wavelength, fluence, pulse parameters, spot size, and irradiation time influence the effects of light energy on tissue. Visible light with wavelengths ranging from 400 to 700 nm can stimulate positive photobiomodulatory effects that promote wound healing, reduce inflammation and pain, modulate stem cell populations, and reduce bacterial contamination of wounds.26,27

Once visible light enters the skin, it is absorbed by the cells and initiates chemical changes dependent on the wavelength (or color) of light and the chromophore within the skin.27 Within each cell, membrane-bound organelles called mitochondria contain chromophores that absorb the light energy and begin making energy (adenosine triphosphate; ATP) via activation of cytochrome c oxidase. Outcomes of the mitochondrial respiratory pathway activation include stimulation of secondary messenger pathways, production of transcription factors and growth factors, and increased ATP production. However, excessive light energy exposure will overstimulate mitochondrial respiration and cause expenditure of all ATP reserves, which creates oxidative stress resulting in damaging elevations of nitric oxide, production of harmful free radicals, and activation of cytotoxic mitochondrial-signaling pathways leading to apoptosis.27,28 This is why creating PBM therapy protocols is important for targeting the beneficial effects while avoiding unintended harm.

Specific benefits of light energy within the visible light spectrum can be broken down into each color of light. Blue light (400-500 nm) has a lower penetration depth and primarily interacts with keratinocytes, reduces bacterial adhesion and growth, and increases intracellular calcium and osteoblast differentiation.29-31 Green light (495-570 nm) affects the superficial tissue and alters melanogenesis, reduces hyperpigmentation of the skin, and reduces tissue swelling.29,30 Red light (600-750 nm) penetrates deeper into the dermis and subcutis where it acts on cellular mitochondria to reduce inflammation and promote collagen synthesis through fibroblast proliferation and production of transforming growth factor-, fibroblast growth factor, platelet derived growth factor, and others.26-28,32,33 Red light has proliferative effects on mesenchymal stem cells and induces proliferation of epithelial colony forming units important for tissue repair and regeneration.34,35

Phovia, sold by Vetoquinol, is a form of fluorescent PBM therapy utilizing a blue light emitting diode (LED lamp, 400-460 nm) and topical photoconverter gel that emits low-energy fluorescent light (510-600 nm) when illuminated by the LED lamp.36,37 This interaction results in the formation of multiple wavelengths of visible light, each with a unique depth of penetration and effect on the tissue as described above. Application is fast and simple. The affected skin may be clipped free of hair and cellular debris removed with gentle cleaning. The skin is allowed to dry before application of the photoconverter gel. Just prior to application, 1 ampule of fluorescence chromophore gel is added to 1 container of photoconverter carrier gel and mixed thoroughly. The mixture is applied in a 2-mm layer to the affected skin, and the LED lamp is held 5 cm above the lesion and used to illuminate the area for 2 minutes. The gel is wiped away using saline-soaked gauze. The application can be repeated immediately after 5 to 10 minutes of rest or a second application can occur a few days later. Twice-weekly applications are continued until the wound is healed. Appropriate eyewear is required to protect the operator from the intensely bright light. Application is pain free and stress free for the patient, so sedation is not typically required.

Benefits of Phovia

Phovia shows great promise as a safe, effective therapy for treatment of numerous inflammatory dermatoses in dogs including superficial pyoderma,38 deep pyoderma,39 perianal fistula,40 interdigital dermatitis,41 calcinosis cutis,42 acute traumatic wounds,43 chronic wounds,37 surgical wounds,44 and otitis externa.45 Phovia as a sole therapy speeds time to healing by 36% in canine superficial pyoderma as compared with dogs receiving oral antibiotics alone.38 In one study, dogs with superficial pyoderma were treated with Phovia alone or with an oral antibiotic alone. Dogs treated twice weekly with Phovia demonstrated complete clinical healing in about 2.3 weeks (P < .05)whereas dogs receiving oral antibiotic healed in about 3.75 weeks.38 Additionally, Phovia speeds time to healing by nearly 50% in deep pyoderma when used with an oral antibiotic (5.7 weeks of treatment) compared with dogs receiving only oral antibiotic (11.7 weeks of treatment).39 The ability of this fluorescent PBM therapy to eliminate or significantly reduce duration of exposure to antibiotics will decrease the spread of antibiotic-resistant bacterial strains within pets and humans.

Phovias high safety profile makes it a beneficial tool to implement in everyday practice. Training the veterinary team to communicate therapy benefits with clients as well as to perform treatments is fast and easy. Training the veterinary technicians to perform treatments will give the veterinarian time to examine other patients. A single back-to-back application takes about 15 minutes, so pet owners can be in and out of the clinic quickly; however, the 2 weekly treatments can be separated by a few days if the veterinarian prefers to evaluate the patient more frequently. Additionally, when used as a sole therapy, clients are not required to administer oral or topical medications at home. This greatly improves treatment adherence and success. Instruct clients to use once-daily smartphone photos to document improvement at home. This can be useful when deciding how many treatments to perform. Most cases of superficial pyoderma will resolve completely by the third treatment.38 It is a good idea to communicate to clients that 3 to 4 weekly treatments may be required.

Conclusion

Phovia is a versatile, innovative therapeutic approach to numerous types of dermatitis.36 It is easy to implement in general practice, and is safe, pain free, and affordable. Phovia is highly effective for superficial and deep skin infections and eliminates the need for clients to administer numerous at-home treatments. This greatly improves the pet-owner bond and treatment outcomes by promoting adherence. Phovia accelerates time to wound healing, which decreases duration of antibiotic exposure and may reduce risk of antibiotic resistance development in these cases.2,13,36-39 Phovias efficacy against antibiotic-susceptible and antibiotic-resistant bacteria shows promise as an alternative therapeutic approach that promotes the principles of antimicrobial stewardship.36 If you are interested in purchasing this medical device for your practice, contact your Vetoquinol service representative.

Amelia G. White, DVM, MS, DACVD is an associate clinical professor of dermatology at Auburn University College of Veterinary Medicine.

REFERENCES

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Managing superficial pyoderma with light therapy - DVM 360

Completion of Enrollment in Phase III Comparative Study for Investigational Regenerative Cellular Medicine (gMSC1) for Knee Chondrogenesis Using…

Chugai Pharmaceutical Co., Ltd.

TWOCELLS Co., Ltd.

Completion of Enrollment in Phase III Comparative Study for

Investigational Regenerative Cellular Medicine (gMSC1) for Knee Chondrogenesis Using Allogeneic 3D Artificial Tissue of MSC

TOKYO, October 27, 2021 --Chugai Pharmaceutical Co., Ltd.(TOKYO: 4519) and TWOCELLS Co., Ltd.

(Head Office: Hiroshima City, Hiroshima; President and CEO: Toshiki Hiura; hereafter, TWOCELLS) announced completion of target enrollment into a Phase III comparative study for an investigational regenerative cellular medicine for chondrogenesis in the knee (development number "gMSC1"), with surgery on the 70th patient.

This study examines the safety and efficacy of transplanting gMSC1, a three-dimensional artificial tissue of allogeneic MSCs, in comparison with microfracture surgery for patients with symptomatic traumatic cartilage defects or osteochondritis dissecans in the knee. The enrollment had started on November 29, 2017 and completed with the 70th surgery, fully enrolling the target number of patients. Going forward, the primary endpoints of histological evaluation of the cartilage and subjective symptoms will be analyzed at week 52 after surgery.

"The articular cartilage in the knee plays an important role in assisting with smooth leg movement. It has a very limited regenerative capacity, and various methods of treatment are under investigation for damages on the cartilage. gMSC1 aims to repair the cartilage in the knee as a regenerative cell therapy which does not require the patient's own tissue taken from their joint, potentially paving the way for solving unmet medical needs in existing treatments. We hope that the assessment ahead will prove the value of gMSC1, Chugai's first regenerative medicine project, and its benefit for patients," said Dr. Osamu Okuda, President and CEO of Chugai.

Toshiki Hiura, President and CEO of TWOCELLS, said, "Despite significant impact from COVID-19 pandemic, we have successfully completed enrollment in the study. Supported by the partnership with Chugai, we, here in Hiroshima, are smoothly advancing the development of basic technologies required for allogenic tissues, the provision of tissues from donors and establishing a GCTP-compatible facility to manufacture final products in Japan for the first time. We will build commercial production capacity as early as possible and strive to make regenerative medicine a familiar treatment option."

Chugai and TWOCELLS concluded a licensing agreement for gMSC1 in 2016. Under the agreement, TWOCELLS is conducting the clinical trial, and responsible for manufacture and supply of gMSC1. Chugai has joint development and exclusive distribution rights for gMSC1 in Japan and is responsible for regulatory application.

To provide more patients with an innovative treatment option as soon as possible, Chugai and TWOCELLS will work on the practical application of the cartilage regenerative therapy using allogenic synovium-derived mesenchymal stem cell, which is the first of its kind in the world.

TWOCELLS and Chugai Announce Performing Surgery of the First Patient in Phase III Trial for "gMSC1," a Regenerative Cellular Medicine for Chondrogenesis in the Knee (press release on November 29, 2017) https://www.chugai-pharm.co.jp/english/news/detail/20171129170000_50.html

Chugai and TWO CELLS Announce a License Agreement for "gMSC1" a Regenerative Cellular Medicine for Chondrogenesis in the Knee (press release on April 25, 2016) https://www.chugai-pharm.co.jp/english/news/detail/20160425150000_144.html

Sources of reference for the study:

Japan Pharmaceutical Information Center (JAPIC) Drug Information Database

http://www.clinicaltrials.jp/user/cteSearch.jsp

About gMSC

gMSC1 is a tissue-engineered medical product currently developed by TWOCELLS and was prepared for the regenerative chondrogenesis using synovium-derived mesenchymal stem cell (MSC) in collaboration with Osaka University and Hiroshima University. This product is a scaffold-free allogeneic 3D artificial tissue of MSC provided by TWOCELLS with their own technologies and serum-free medium (STK1 and STK2), which is expected to provide an effective treatment for cartilage regeneration. Development of gMSC1 has been supported by JST (Japan Science and Technology Agency), NEDO (New Energy and Industrial Technology Development Organization), the Ministry of Economy, Trade and Industry, and AMED (Japan Agency for Medical Research and Development).

About Chugai

Chugai Pharmaceutical is one of Japan's leading research-based pharmaceutical companies with strengths in biotechnology products. Chugai, based in Tokyo, specializes in prescription pharmaceuticals and is listed on the 1st section of the Tokyo Stock Exchange. As an important member of the Roche Group, Chugai is actively involved in R&D activities in Japan and abroad. Specifically, Chugai is working to develop innovative products which may satisfy the unmet medical needs.

About TWOCELLS

TWOCELLS is a bio-venture company established in Hiroshima in 2003, aiming to promote regenerative medicine so that patients may have a new treatment option. By particularly targeting MSC (mesenchymal stem cell), it is engaging in the development of cellular medicine with MSC, peri-MSC culturing technique and a system for regenerative medicine.

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Completion of Enrollment in Phase III Comparative Study for Investigational Regenerative Cellular Medicine (gMSC1) for Knee Chondrogenesis Using...