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To get it out of the way immediately, we are NOT talking about fetal or embryonic stem cells today.

At birth, the umbilical cord and placenta are routinely discarded. These healing stem cells are harvested in Colorado by vitro Bio Pharma and distributed around the world to help people heal and improve their quality of life. They cannot be used in the USA as they are not FDA approved, and large corporations in the pharmaceutical industry have lost interest in funding clinical trials on something you cant patent. Big pharma saves many lives through new research and development but ultimately must answer to their shareholders and show a profit.

I became fascinated with these umbilical and mesenchymal stem cells after meeting people whose conditions improved after I.V. and local injection of stem cells. I went to the island of Antigua where Dr. Joseph John, a Columbia medical school graduate, worked tirelessly to change laws there so that stem cells cold be used in the island nation.

The medical director who is based in Chicago and does the procedure is Chadwick Prodromos, M.D. Dr. Prodromos went to Princeton undergrad and the Johns Hopkins medical school as well as residency and fellowship training at Yale and Harvard. People from the East Coast travel to the Antigua clinic, and west coast people travel to Monterrey, Mexico to the other clinic to see Dr. Prodromos.

Conditions they treat are heart failure, multiple sclerosis, the effects of aging, Bells palsy, chronic obstructive pulmonary disease diabetes, erectile dysfunction and hip, knee and shoulder problems. People have experienced other benefits such as more energy, less pain and more mobility. The people I contacted in follow-up reported improved heart ejection fraction (pumping ability), improved vision, more mobility and reduced pain.

Stem cells are not rejected because they havent become any specific type of cell. Stem cells are injected into the I.V. and almost magically find where the problem is. We will be hearing more about the healing power of stem cells in the coming years.

Peter Michalos, MD is associate professor of clinical ophthalmology, Columbia University VP&S; chairman, Hamptons Health Society; and a Southampton resident.

See the article here:
Health on the Frontlines: The Healing Power of Stem Cells - Dans Papers

This months top grants in regenerative medicine, sourced from Dimensions, includes projects on: a novel platform to enhance single cell interrogation of nervous system development, human endothelial cell regulation of ossification and the development of a dynamic double network hydrogel for generating pancreatic organoids from induced pluripotent stem cells.

This project aims to investigate a strategy, which utilizes novel spatial transcriptomics approaches, integrated multiplexed RNA/protein detection and visualization and computational algorithms to identify and map molecular markers of the preganglionic neurons in the ventral spinal cord and progenitor cell populations of the sympathetic ganglia. If successful, the approach could provide a foundation for basic research of peripheral nervous system birth defects and repair using stem cell-based therapies, as well as future studies of neuroblastoma initiation.

Funding amount:US$206,000

Funding period: 8 August 2022 31 July 2024

Funder:Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Research organization:Stowers Institute for Medical Research (MO, USA)

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Over one million patients undergo bone repair procedures in the USA annually, with autologous bone grafting remaining the preferred treatment for bone defects. The development of therapies that exploit the osteogenic potential of bone marrow-derived mesenchymal stem cells (bm-MSCs) has been limited due to limited understanding of the regulatory mechanisms of in vivo bm-MSC osteogenesis. Previous research from the group showed that the osteogenic potential of bm-MSCs is dependent on sustained proximity to endothelial cells. The goal of the present study is to elucidate the cellular and molecular mechanisms by which endothelial cells regulate the osteogenic differentiation of bm-MSCs and develop a foundation of knowledge upon which to build therapeutic strategies for bone regeneration utilizing autologous bm-MSCs.

Funding amount:US$442,000

Funding period: 10 August 2022 31 May 2027

Funder:National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)

Research organization:Boston Childrens Hospital (MA, USA)

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Human induced pluripotent stem cells provide a valuable source of cells for basic research and translational applications. While there have been advances in lineage-specific differentiation of human induced pluripotent stem cells, there remains limited understanding on the impact of matrix stiffness, viscoelasticity and integrin ligand presentation on the multi-stage development of exocrine pancreatic organoids. This research aims to define the influence of matrix properties on the generation of exocrine pancreatic organoids by developing a viscoelastic dynamic double network hydrogel platform with controllable matrix mechanical properties and biochemical motifs. This will advance the application of chemically defined matrices as xeno-free artificial stem cell niches for organoid growth and tissue regeneration applications.

Funding amount:US$468,000

Funding period: 1 August 2022 31 July 2026

Funder:National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

Research organization: Indiana University Purdue University Indianapolis (IA, USA)

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Top 3 grants in regenerative medicine: July 2022 - RegMedNet

image:Prof. Christina Peters, MD, points out: "We have published highly topical and clinically relevant manuscripts that are of utmost importance for the treatment of children with high-risk acute lymphoblastic leukemia. One of the most burning questions for me is whether we still need stem cell transplantation in the era of modern immunotherapies." view more

Credit: St. Anna Children's Cancer Research Institute

Acute lymphoblastic leukemia (ALL) is the most common cancer in children, generally curable with contemporary chemotherapy. However, if the disease is classified as high-risk ALL and a stem cell transplantation becomes inevitable, total body irradiation is still the treatment of choice prior to transplantation. This was the conclusion drawn from the FORUM study, including 35 countries on five continents (Peters et al., Journal of Clinical Oncology 2020).

"As the largest study on this topic to date, we published the results in the top-ranked Journal of Clinical Oncology. Soon after that, Frontiers invited us the international transplant consortium for ALL to publish a collection of reviews and scientific reports on ALL in children," recalls Christina Peters, Affiliated Clinician at St. Anna Children's Cancer Research Institute (St. Anna CCRI) and Senior Physician at St. Anna Children's Hospital. The editorial board of this Research Topic is formed by Christina Peters together with Assoc.-Prof. Adriana Balduzzi, MD (University of Milano Bicocca, Italy) and Prof. Peter Bader, MD (Goethe University Frankfurt am Main, Germany).

Life at the price of long-term side effects?Although total body irradiation and hematopoietic stem cell transplantation (HSCT) from healthy donors can be life-saving, long-term side effects sometimes have a massive impact on the quality of life of children and young adults. These include organ damage, growth retardation, and the development of secondary cancers. Hence, there was a dire need to clearly outline recent and previously published data, as well as to discuss potential new approaches, as did the aforementioned reviews.

"One of the most burning questions for me is whether we still need stem cell transplantation in the era of modern immunotherapies," Christina Peters points out. In the future, CAR-T cell or antibody therapies directly targeting leukemia cells could replace HSCT, which is addressed by three of the reviews mentioned. Jochen Bchner, MD, PhD, and colleagues are discussing the question of whether and when CAR-T cell therapy could be considered to bridge the time until transplantation and under what conditions it could replace transplantation. Another review, authored by Assoc.-Prof. Tony H. Truong, MD, and colleagues, is dedicated to the question which children should receive a stem cell transplantation at all. Of course, transplantation should only be considered for those patients who would not have a realistic chance of survival with 'milder' therapies. But it is exactly these boundaries that are currently shifting.

More than 59,000 views of our workSo far, the review of Bianca A. W. Hoeben, MD, PhD, and colleagues, which deals with new methods of total body irradiation, has had the most views in this online collection. "In total, we have more than 59,000 views of our Research Topic by now. Accordingly, it seems to be on many people's minds whether and how we can improve transplantation methods to reduce side effects," says Christina Peters. For example, different radiotherapy centers have developed new methods of total body irradiation to achieve a lower irradiation dose in certain organs. There are, however, limitations to shield organs at risk without compromising the anti-leukemic and immunosuppressive effects the latter to preserve a rejection of the transplanted cells.

In addition to the aforementioned long-term effects of irradiation and transplantation, acute side effects of transplantation also play a major role. These include infections during the period in which the immune system reconstitutes (Olga Zajac-Spychala et al.), or complications arising from a donor cells attack against the patient's healthy tissue. The prevention and treatment of the so-called Graft-versus-Host Disease are addressed by six of the articles in this Research Topic (Steven J. Keogh et al., Anita Lawitschka et al., Jacob Rozmus et al., Agnieszka Sobkowiak-Sobierajska et al., Matthias Wlfl et al., Natalia Zubarovskaya et al.).

"The publication of this Research Topic is a huge success for St. Anna CCRI. Together with the valuable contributions of a number of clinicians of St. Anna Children's Hospital, we have published highly topical and clinically relevant manuscripts that are of utmost importance for the treatment of children with high-risk ALL," highlights Christina Peters.

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About the FORUM StudyThe FORUM study demonstrated that patients over four years of age with high-risk ALL in need of a stem cell transplantation live longer and have a lower risk of relapse when they receive total body irradiation instead of chemotherapy in preparation for transplantation. After random assignment of 417 pediatric patients with high-risk ALL, a futility stopping rule was applied because it became apparent that patients receiving chemo-conditioning had a lower chance of cure and survival. FORUM is a randomized, international, multicenter phase III trial designed to investigate whether chemotherapy with fludarabine, thiotepa, and busulfan or treosulfan is non-inferior to total body irradiation plus etoposide as preparation prior to transplantation. The study, led by Christina Peters, is the result of a collaboration among international study groups (AIEOP-BFM-ALL-SG, IBFM-SG, INTREALL-SG, and EBMT-PD-WP).

Frontiers Research TopicThe Frontiers in Pediatrics "Research Topic" Allogeneic Hematopoietic Stem Cell Transplantation for Children with Acute Lymphoblastic Leukemia in the Era of Immunotherapy is available for download here: https://www.frontiersin.org/research-topics/19704/allogeneic-hematopoetic-stem-cell-transplantation-for-children-with-acute-lymphoblastic-leukemia-in#overviewThe specific thematic areas envisaged to be addressed in this article collection are the following:

Are HLA-identical siblings still the best available donor for ALL? The challenge of treating older children: what is the best transplant strategy for Adolescents and Young Adults (AYAs)? Tyrosine Kinase Inhibitors (TKIs) for Philadelphia Chromosome positive (Ph+) and Ph-like ALL: could we omit Hematopoietic Stem Cell Transplantation (HSCT)? Bispecific Antibodies before HSCT: less toxicity for better transplant outcome? CAR-T cell therapy: only bridge to transplant? T-cell depletion: Cyclophosphamide after transplantation versus in vitro T-cell depletion Why is Total Body Irradiation so effective in high-risk ALL? Total Body Irradiation forever? New chemotherapeutic options for irradiation-free conditioning Minimal Residual Disease (MRD): Which level of negativity is relevant? Current treatment options for acute Graft-versus-Host-Disease (GVHD) in children Current treatment options for chronic GVHD in children Immunoreconstitution and chimerism: a different story compared to adults? Non-relapse mortality after HSCT: where are we now? High-risk ALL: Transplant indications in 2021 COVID-Infection after allogeneic stem cell transplantation Transplantation for the youngest: better than chemotherapy?

About Christina PetersChristina Peters, MD, is Professor of Pediatrics at the Department of Stem Cell Transplantation of St. Anna Children's Hospital and Affiliated Clinician at St. Anna Childrens Cancer Research Institute in Vienna. She is principal investigator of active studies within the European Society for Blood and Marrow Transplantation (EBMT) and the International Berlin Frankfurt Mnster Study Group (IBFM) for the treatment of pediatric leukemia. Her research interests include allogeneic hematopoietic transplantation in children and adolescents with malignant and non-malignant diseases from related and unrelated donors, infectious and toxic complications after stem cell transplantation, adoptive therapies for hematological malignancies and family oriented rehabilitation for children with life threatening diseases.

Christina Peters chaired the EBMT Pediatric Diseases Working Party between 2008 and 2014. She has authored and co-authored numerous papers in peer-review journals such as The Lancet, The New England Journal of Medicine, or The Journal of Clinical Oncology. Christina Peters acts as a regular reviewer of publications for hematology, pediatric and leukemia journals. She is a member of many professional societies including IBFM, the Center for International Blood and Marrow Transplant Research (CIBMTR), the German and Austrian Society of Pediatric Hematology and the Austrian Gene Therapy Commission. Furthermore, Christina Peters is a member of the Advisory Board of the Austrian Ministry of Health, the Bioethical committee of the Austrian Prime Minister and member of the European Network Pediatric Research at the European Medicines Agency EMA (ENPREMA).

About St. Anna Childrens Cancer Research Institute, St. Anna CCRISt. Anna CCRI is an internationally renowned multidisciplinary research institution with the aim to develop and optimize diagnostic, prognostic, and therapeutic strategies for the treatment of children and adolescents with cancer. To achieve this goal, it combines basic research with translational and clinical research and focus on the specific characteristics of childhood tumor diseases in order to provide young patients with the best possible and most innovative therapies. Dedicated research groups in the fields of tumor genomics and epigenomics, immunology, molecular biology, cell biology, bioinformatics and clinical research are working together to harmonize scientific findings with the clinical needs of physicians to ultimately improve the wellbeing of our patients.www.ccri.at http://www.kinderkrebsforschung.at

About St. Anna Children's HospitalEstablished in 1837 in the former suburb of Schottenfeld, St. Anna was the first children's hospital in Austria and the third independent hospital in Europe dedicated exclusively to the health of children. St. Anna Children's Hospital has evolved into an institution that provides state-of-the-art medical care. Thus, in addition to its performance as a general children's hospital, the Center for Pediatrics and Adolescent Medicine has also been able to establish an excellent reputation throughout Austria and internationally over the past 40 years as a center for the treatment of pediatric hematologic disorders and tumor diseases (cancer).www.stanna.at

Frontiers in Pediatrics

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Allogeneic Hematopoetic Stem Cell Transplantation for Children with Acute Lymphoblastic Leukemia in the Era of Immunotherapy

The authors declare that the editorial of this Research Topic was written in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

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Childhood leukemia treatment 2022: Where we are now and what it takes - EurekAlert

One of the most frequently altered genes in AML is nucleophosmin1 (NPM1), which is frequently linked to a good prognosis. Immunological responses increasingly influenced treatment choices for AML. However, it was unclear how immune checkpoint inhibition functions. For a study, researchers sought to determine the particular immune responses of AML patients to NPM1, PRAME, Wilms tumor 1, RHAMM, and 3 additional leukemia-associated antigens (LAA).

Using colony-forming immunoassays and flow cytometry, they examined T cell responses against leukemic progenitor/stem cells (LPC/LSC). In addition, comparing cells from NPM1 mutant and NPM1 wild-type AML patients, they investigated whether immune checkpoint suppression with the anti-programmed death 1 antibody improved the immune response to stem cell-like cells.

Nivolumab, an anti-PD-1 antibody, was reported to enhance the number of LAA-stimulated cytotoxic T cells and to have a cytotoxic impact on LPC/LSC. When the immunogenic epitope was obtained from the area of NPM1 that had been altered, the impact was best against NPM1mut cells and the effects were strengthened by the addition of anti-PD-1.

The results implied that the immune checkpoint inhibitor anti-PD-1 may be used to treat individuals with NPM1 mutant AML and that this treatment, in combination with NPM1-mutation specific directed immunotherapy, may be even more successful for this particular subset of patients.

Reference: onlinelibrary.wiley.com/doi/10.1111/bjh.18326

Read more here:
Treatment Choices for AML Were Increasingly Influenced by Immunological Responses - Physician's Weekly

By Maria Aspioti and Paolo Siciliano, PA Consulting

The world of advanced therapeutics medicinal products (ATMPs) and, in particular, the cell and gene therapies (C>) space has been experiencing outstanding growth over the last few years, with a number of therapies transitioning from clinical research into regular clinical practice.In recent years, new cell types and new technologies have been used to overcome challenges posed by current treatments and by the nature of the targeted diseases, thus enabling us to treat, and in some cases potentially cure, severe disorders. The scientific and R&D efforts led to the discovery of new ways to engineer cells, enabling some of the most outstanding hurdles in complex disease areas such as oncology, cardiovascular, neurologic, and metabolic disorders to be addressed.

While these technology and scientific advancements are all positive and are promising signs of a growing and thriving sector, the other side of the coin shows a highly fragmented market, with very high levels of uncertainty on what type(s) of approaches will be successful in providing patients with a true alternative and which approaches will quickly become obsolete due to technical or commercial limitations. So, how do companies and investors in the CGT space hedge their bets in a fast-evolving and highly uncertain market?

In this article, we review the main cell technologies currently being developed in clinical research for oncology and other therapeutic areas, including examples of studies being conducted, developers, modes of action, and benefits of the different types of cell therapies, and share insight on how to avoid pitfalls and prepare for rapid market and technological directional changes.

Chimeric antigen receptor (CAR) T cells have been dominating the C> field for years, resulting in the approval and commercialization of Kymriah, the first therapy of this kind, in 2017.

Since then, CAR-T therapies have been the most researched type of cell therapy globally and five more products based on this technology have been approved for the treatment of various types of blood cancers worldwide (Yescarta, Abecma, Tecartus, Breyanzi, and Carvykti).

The current commercially available CAR T cell therapies (which are all autologous) have shown efficacy in the treatment of hematologic cancers such as acute lymphoblastic leukemia (ALL), chronic lymphocytic leukemia (CLL), non-Hodgkin's lymphoma (NHL), diffuse large B cell lymphoma (DLBCL), and other B cell malignancies. Given the increasing number of clinical studies utilizing CAR T cell therapies and the already successful application in cancer patients, this class of biotherapeutics is likely to dominate the C> market and R&D space for the next few years. This is also shown by the predicted CAGR of 30.6% over the period 2021-2031, which should lead to a total CAR T cell therapy market size of $23.2 billion in the next decade.

At present, there are 750 active CAR-T therapies in development across the globe (375 in clinical phases and 378 in preclinical stage). This represents over a 50% increase from 2019, when approximately 245 CAR-T therapies were in clinical development. Currently, CAR-T therapies still represent 31% of the clinical pipeline in C> (375 out of 1,191 active trials) The vast majority of these are in the early clinical development stage (predominantly Phase 1), with oncology counting for over 95% of the active CAR-T trials.

While still the predominant realm in C>, CAR-T therapies present some limitations.

Numerous allogeneic alternatives are being investigated to overcome some of the challenges faced by CAR-T therapies in oncology. In the field of adoptive cell immunotherapy for oncology, we are seeing an increasing exploitation of alternative cell sources with a high therapeutic potential that aim to evolve toward universal allogeneic alternatives to classic CAR-T therapies. Some examples include (further investigative product examples are shown in Table 1 [below]):

In addition to CAR-based technologies, the market is also seeing an increasing number of preclinical and clinical studies focusing on CAR-free cell therapy alternatives to cure cancer. The list of cell types is continuously growing, but we see five main categories that are showing promising results (investigative product examples are shown in Table 1):

Induced Pluripotent Stem Cells (iPSCs): iPSCs are a type of pluripotent stem cells that are generated ex vivo by treating nearly any human fully differentiated (somatic) cell (e.g., keratinocytes, fibroblasts, etc.) with the cocktail of small molecules described by the 2012 Nobel Laureate Shynia Yamanaka. iPSCs display several advantages over primary cells, including their virtually infinite proliferation capacity and amenability for genetic manipulation. Several T and NK cells derived from iPSC lines (iT and iNK, respectively) are currently under investigation, primarily for B cell lymphoma and advanced solid tumors.An example is represented by Shoreline Biosciences, which is developing a pipeline of iPSC-derived natural killer cell (iNK) and macrophage (iMACs) cellular immunotherapy candidates for the treatment of different types of cancers.

Mesenchymal Stem Cells (MSCs): MSCs are multipotent stem cells capable of self-renewal that are commonly found in the bone marrow but also in the umbilical cord, adipose tissue, and peripheral blood. In clinical trials, MSCs are used in cancer treatment either via direct transplantation (often used to support chemotherapy or radiotherapy), as genetically modified cell therapy, or as a carrier of anti-tumor agents like interferon , interleukins, bone morphogenic protein 4, and many others. At the end of 2021, 31 clinical trials concerning MSC-based therapies for cancer were registered on ClinicalTrials.gov. The majority of these studies focus on the direct infusion or transplantation of MSCs to treat cancer, while the remaining trials use engineered MSCs as vehicles of therapeutic agents such as cytokines or oncolytic viruses.MSCs are also being investigated for a wide range of non-oncology applications, including cardiovascular conditions as well as neurodegenerative disorders such as Alzheimers, multiple sclerosis, and amyotrophic lateral sclerosis. Brainstorm Cell Therapeutics is currently in the process of finalizing the regulatory filing for NurOwn (autologous MSC-NTF cells produced from autologous, bone marrow-derived mesenchymal stem cells) for the treatment of ALS.

Dendritic Cells: Dendritic cells (DCs) are antigen-presenting cells (APCs) that represent another valuable alternative to CAR-T therapies. In clinical settings, DCs find applications as vaccines owing to their ability to prepare the adaptive and innate immune system against specific tumors via presenting cancer-specific antigens. To date, the only FDA-approved DC-based vaccine is Provenge (sipuleucel-T, Dendreon), which targets patients with metastatic castration-resistant prostate cancer. Another DC-based medicinal product named Apceden, developed by Apac Biotech, was approved in India by the Central Drugs Standard Control organization in 2017. Currently, there are five Phase 1, 10 Phase 2, and five Phase 3 clinical trials ongoing that demonstrate the excitement around this cell therapy type.

Tumor Infiltrating Lymphocytes (TILs): TILs are immune cells that can infiltrate tumor masses and currently present an alternative therapeutic solution that has mainly been researched for the treatment of advanced solid tumor indications. Currently, TIL cell therapies are being explored at the clinical setting, predominantly for the treatment of melanoma with a number of other cancer indications also being under investigation.Two key players in this space are Achilles Therapeutics and Iovance Biotherapeutics, both of which are developing TIL-based therapies (Phase 2 trials) for the treatment of different types of cancer.

Regulatory T Cells (Tregs): Similar to TILs, biotherapeutics based on Tregs are also being investigated as a form of cell therapy for multiple indications. Treg-cell therapies are currently in early infancy with multiple opportunities being explored across a spectrum of indications such as type 1 diabetes, rheumatoid arthritis, multiple sclerosis, and others.Quell Therapeutics, GentiBio, and Sonoma (among others) are developing Treg-based therapies to address a number of autoimmune and alloimmune conditions.

Oncology is the field where the majority of C>s are commercially available currently. As described above, a number of biotherapeutics have been approved by the FDA and other regulatory agencies, including CAR T cell therapies as well as other immunotherapies such as talimogene laherparepvec (Imlygicby Amgen Inc.) and sipuleucel-T (Provenge by Dendreon Corp.).

While oncology has been driving R&D in C> since the beginning of the new wave of therapeutic innovation, the interest of academic groups, biotechnology firms, and large pharmaceutical companies in different disease areas for ATMPs is rapidly expanding. This is particularly the case for in vivo gene therapies, where we have recently seen the approval of products for the treatment of spinal muscular atrophy (Zolgensma by Novartis) and mutation-specific retinal dystrophy (Luxturna by Spark Therapeutics), as well as an increasing number of trials across a large spectrum of non-oncology applications, including several rare genetic disorders.

For cell therapies, in the last few years, a number of therapies have been launched on the market for non-oncology applications. These include Rethymic by Enzyvant Therapeutics (congenital athymia), Stratagraft (deep partial-thickness burns), Gintuit (epithelial damage), and Maci (cartilage damage). Several non-oncological biotherapeutics received approval for use in unrelated donor hematopoietic progenitor cell transplantation (Allocord, Clevecord, Ducord, Hemacord).

As indicated in our Cell & Gene Therapy 2040 Report, which looks at the future of the C> industry, the clinical development of C>s is predominantly aimed at cardiovascular, metabolic, neurological, inflammatory/autoimmune, and musculoskeletal disorders, with a particular focus on rare genetic conditions. Currently, oncology-unrelated Phase 3 trials focus on more than 20 different indications with over 25 lead companies involved.

Among the indications with the highest number of clinical studies, it is worth noting hemophilia A, for which Pfizer, Roche, and BioMarin Pharmaceutical developed similar C>s targeting coagulation factor VIII. BioMarin Pharmaceutical retains a slight commercial advantage as their asset also targets a different coagulation factor and is expected to reach approval by the end of 2022 both in Europe and the U.S. (approvals are expected for Pfizer in 2023 and Roche in 2024, both in the U.S. only).

Other indications that are seeing a surge in late-phase clinical trials include Duchenne muscular dystrophy and Crohns disease. For the former indication, Pfizer and Sarepta Therapeutics are currently recruiting patients for the virus-mediated administration of the gene encoding for microdystrophin to help rescue the muscle architecture. In Crohns disease, Takeda Pharmaceutical and Mesoblast Ltd. are the two front-runners. Notably, both are developing MSC-based therapies for this indication, with Takeda having already shown positive results (ClinicalTrials.gov Identifier: NCT03706456) and currently recruiting for two additional Phase 3 studies.

The field of C> is fast-growing and booming with novel technologies, new companies, and growing investment, with more and more positive results in treating, and even curing, life-threatening diseases. But how can organizations hedge their bets in such a fast-evolving and highly uncertain market?

Here are some tips on how different players in the CGT space can avoid pitfalls and better position themselves to succeed in this space:

Being aware of the C> landscape and how it is changing becomes paramount for developers. A clear view of the market and its evolution will enable developers to:

In addition, understanding the nature of the new biotherapeutics developed and how they are delivered is vital for C> manufacturers, healthcare professionals, and patients to enable a facilitated clinical application while reducing the overall costs of these transformative therapies.

Time to market is key to avoid a technology becoming obsolete in a fast-evolving market. The complexity of developing and launching new products in the C> market (these being therapies or technologies involved in their manufacturing) requires a level of investment, competencies, and capabilities that rarely are available in a single organization. Hence, innovators in this space should invest time and resources in identifying the right partners to support their product development, access the right technologies to manufacture their therapies, embrace digital tools early on to support the launch of their products, as well as work with experts to speed up the transition from R&D to clinical and commercial scale.

Focus is usually key in bringing new products and services to market in highly innovative sectors. However, to mitigate risk in a highly uncertain market, established pharma and biotech companies developing biotherapeutics should look at diversifying their portfolio through the development and/or acquisition of multiple C> platforms across different therapeutic areas. Similarly, equipment manufacturers should also look at how their current products and innovation portfolios can support the needs of different product lines, as certain technologies might quickly become obsolete if a specific set of therapies becomes predominant in the market.

Overall, the advent of C> therapies has the potential to revolutionize healthcare by providing therapies for rare, complex, and life-threatening diseases. Successful positioning of players in this flourishing market will require careful consideration of the evolving market dynamics coupled with successful go-to-market and risk mitigation strategies.

About The Authors:

Maria Aspioti is a healthcare and life sciences expert at PA Consulting. She has several years of professional experience in product innovation for medical devices and a diverse academic background in life sciences. She has worked extensively with early-stage R&D teams as a biology specialist on technology landscaping, technology evaluation, and scientific diligence. She is the co-inventor of several patents in the field of advanced wound therapies. In addition, she has helped establish and managed preclinical research programs for concept evaluation in various areas, including wound care and regenerative medicine while working with clinical groups and commercial teams to support clinical evidence and business case generation. Aspioti holds a BSc (Hons) in molecular & cellular biology from the University of Glasgow and a MSc in regenerative medicine from the University of Bath.

Paolo Siciliano is an associate partner and life sciences expert at PA Consulting, and he leads PAs work in C> globally. He has several years of experience in supporting major pharma, biotech, and medtech companies to identify, develop, and leverage new technologies to solve business needs, as well as improve their innovation and product development processes. His main areas of expertise range from technology and commercial strategy to technology development, across a number of therapeutic areas. He obtained a Ph.D. in molecular biology and worked as a senior research scientist in biotech companies in the U.K.

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CAR-T Beyond CGTs In Development In 2022 - BioProcess Online

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BioRestorative Therapies to highlight addition of Phase 2 clinical trial for chronic lumbar disc disease to its mid-stage pipeline.

Melville, NY., Sept. 07, 2022 (GLOBE NEWSWIRE) -- BioRestorative Therapies. ("BioRestorative" or the "Company") (Nasdaq:BRTX), a clinical stage company focused on the development of first-in-class cell-based therapies,today announced that it will present on September 14, 2022 at the H.C. Wainwright 24th Annual Global Investment Conference being held virtuallySeptember 12-14, 2022.

Lance Alstodt, President and CEO of the Company,will be speaking and providing an update onthe Company's expanded pipeline and near-term catalysts of itsongoing Phase 2 clinical trial targeting chronic lumbar disc disease.

Further information on this conference, including registration links, can be found below:

H.C. Wainwrights 24th Annual Global Investment ConferenceSeptember 12-14, 2022@ Lotte New York Palace Hotel - New York, NYPresentation Information: BioRestorative on-demand presentation will be livestarting on Wednesday, September 14, 2022 at 11:30am EDTPresenter: Lance Alstodt, CEO & President, BioRestorative Therapies.Registration Link:www.hcwevents.com/annualconferenceWebcasting Link:https://journey.ct.events/view/8518bd6c-814c-477d-a15f-973612fc9e56

About BioRestorative Therapies, Inc.

BioRestorative Therapies, Inc. (www.biorestorative.com) develops therapeutic products using cell and tissue protocols, primarily involving adult stem cells. Our two core programs, as described below, relate to the treatment of disc/spine disease and metabolic disorders:

Disc/Spine Program (brtxDISC): Our lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a persons own) cultured mesenchymal stem cells collected from the patients bone marrow. We intend that the product will be used for the non-surgical treatment of painful lumbosacral disc disorders or as a complementary therapeutic to a surgical procedure. The BRTX-100 production process utilizes proprietary technology and involves collecting a patients bone marrow, isolating and culturing stem cells from the bone marrow and cryopreserving the cells. In an outpatient procedure, BRTX-100 is to be injected by a physician into the patients damaged disc. The treatment is intended for patients whose pain has not been alleviated by non-invasive procedures and who potentially face the prospect of surgery. Pursuant to authorization received from the Food and Drug Administration, we have commenced a Phase 2 clinical trial using BRTX-100 to treat chronic lower back pain arising from degenerative disc disease.

Metabolic Program (ThermoStem): We are developing a cell-based therapy candidate to target obesity and metabolic disorders using brown adipose (fat) derived stem cells to generate brown adipose tissue (BAT). BAT is intended to mimic naturally occurring brown adipose depots that regulate metabolic homeostasis in humans. Initial preclinical research indicates that increased amounts of brown fat in animals may be responsible for additional caloric burning as well as reduced glucose and lipid levels. Researchers have found that people with higher levels of brown fat may have a reduced risk for obesity and diabetes.

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events or results to differ materially from those projected in the forward-looking statements as a result of various factors and other risks, including, without limitation, those set forth in the Company's latest Form 10-K filed with the Securities and Exchange Commission and other public filings. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and the Company undertakes no obligation to update such statements.

CONTACT:Email: [emailprotected]

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BioRestorative Therapies to Present at the H.C. Wainwright 24th Annual Global Investment Conference on September 14 - StreetInsider.com

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Global Recombinant Cell Culture Supplements Market

Global Recombinant Cell Culture Supplements Market

Dublin, Sept. 05, 2022 (GLOBE NEWSWIRE) -- The "Global Recombinant Cell Culture Supplements Market: Focus on Pricing Analysis, Product, Application, Expression System, and Region - Analysis and Forecast, 2022-2032" report has been added to ResearchAndMarkets.com's offering.

The global recombinant cell culture supplements market was estimated at $308.6 million in 2021 and is expected to reach $1,188.6 million by 2032, growing at a CAGR value of 12.24% during the forecast period 2022-2032. The growth in the global recombinant cell culture supplements market is expected to be driven by the rising demand for cell culture supplements, increasing investment in life sciences research and development, as well as growing advantages of recombinant supplements over traditional animal-derived supplements.

Market Lifecycle Stage

The global recombinant cell culture market is increasing at a rapid pace. The growing need for animal-free supplements in cell culture applications is aiding the growth of the recombinant cell culture supplements market. Recombinant cell culture supplements play a crucial role in enhancing cell viability, maintaining a healthy culture, and customizing the cell culture in accordance with the needs of the individual.

Increasing demand for immunotherapy and stem cell and regenerative medicine research is one of the major opportunities in the recombinant cell culture supplements market. Several cell culture companies, and biopharmaceutical companies are working collaboratively on drug development and using recombinant cell culture supplements as a therapeutic means for applications in biological drugs. Furthermore, the market witnessed major mergers and acquisitions in the past four years. For instance, recently, in March 2022, Thermo Fisher Scientific Inc. acquired PeproTech, Inc., a company that specializes in the development and manufacturing of recombinant proteins, in a deal of $1.85 billion.

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Impact

Many biopharmaceutical products are being developed by utilizing the cell culture technique. The study of cell physiology and biochemistry is made possible through laboratory cell culture, which also opens up research avenues that are challenging to pursue in vivo. Controlling variables such as the culture media, culture conditions, population density, and growth rate makes it simple to assess the effects of medications or other substances on cultured cells.

Additionally, it allows analyzing the function of various genes and offers enormous potential in the field of genetics. It enables the assessment of harmful and carcinogenic chemicals on cells in the fields of oncology and virology and the comprehension of how different medications, viruses, and physical or chemical carcinogens interact.

Furthermore, the recombinant cell culture has various applications, such as research on vaccines, stem cells, gene therapy, and genetic engineering, as well as the creation of protein therapies manufacturing of genetically edited proteins such as monoclonal antibodies, insulin, and hormones.

Market Dynamics

Market Drivers

Increasing Demand for Recombinant Cell Culture Supplements Due to their Advantages

Increased Funding and Investment in Research and Development for Biopharmaceutical Products in Life Sciences Sector

Growing Number of Mergers and Acquisitions for Expanding Recombinant Cell Culture Supplements Portfolio

Push from Regulatory Bodies to Utilize Animal-Free Media Supplements in Cell Culture Process

Market Restraints

Market Opportunities

Market Segmentationby Product

Recombinant Albumin (rAlbumin)

Recombinant Insulin (rInsulin)

Recombinant Epidermal Growth Factor (rEGF)

Recombinant Interleukin Growth Factor (rILGF)

Recombinant Transferrin (rTransferrin)

Recombinant Trypsin (rTrypsin)

Recombinant Insulin-like Growth Factor (rIGF)

Recombinant Stem Cell Factor (rSCF)

Recombinant Aprotinin (rAprotinin)

Recombinant Lysozyme (rLysozyme)

Others

by Application

by Expression System

Mammalian

E. Coli

Yeast

Others

by Region

North America

Europe

Asia-Pacific

Latin America

Rest-of-the-World

Demand - Drivers and LimitationsFollowing are the demand drivers for the global recombinant cell culture supplements market:

Increasing Demand for Recombinant Cell Culture Supplements Due to their Advantages

Increased Funding and Investment in Research and Development for Biopharmaceutical Products in Life Sciences Sector

Growing Number of Mergers and Acquisitions for Expanding Recombinant Cell Culture Supplements Portfolio

Push from Regulatory Bodies to Utilize Animal-Free Media Supplements in Cell Culture Process

The market is expected to face some limitations too due to the following challenges:

Key Topics Covered:

1 Market Overview

2 Product, $ Mn, 2021-2032

3 Application, $Mn, 2021-2032

4 Expression System, $Mn, 2021-2032

5 Region, $Mn, 2021-2032

6 Competitive Landscape

7 Company Profiles

Companies Mentioned

Abcam plc.

BBI Solutions

Corning Incorporated

FUJIFILM Irvine Scientific, Inc.

Gemini Bioproducts, LLC

HiMedia Laboratories

InVitria

Kingfisher Biotech, Inc.

Lonza Group AG

Merck KGaA

Novus Biologicals, LLC

R&D Systems, Inc.

Sartorius AG

Sino Biological Inc.

STEMCELL Technologies Inc.

Thermo Fisher Scientific Inc.

For more information about this report visit https://www.researchandmarkets.com/r/2s1ob7

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Global Recombinant Cell Culture Supplements Market Report 2022: Increasing Need for Immunotherapies and Stem Cell and Regenerative Medicines Presents...

LYON, France--(BUSINESS WIRE)--Regulatory News:

Adocia (Euronext Paris: FR0011184241 ADOC), a clinical-stage biopharmaceutical company focused on the research and development of innovative therapeutic solutions for the treatment of diabetes and other metabolic diseases, announces the establishment of a first proof of concept for its AdoShell Islets implant by achieving glycemic control without insulin injections in immunocompetent diabetic rats during the 132-day study.

"Adocias results are remarkable, having successfully performed the first islet transplantation without the use of immunosuppressants in immunocompetent animals. We are delighted to be actively involved in these unprecedent results, said Dr. Karim Bouzakri, Director of CEED (European Center for the Study of Diabetes).

AdoShell Islets is an immuno-protective synthetic biomaterial containing islets of Langerhans. After implantation in diabetic animals, the islets encapsulated in AdoShell secrete insulin in response to blood glucose levels. The physical barrier formed by the AdoShell biomaterial allows the implanted cells to be invisible to the host's immune system while allowing the necessary physiological exchanges to occur for the survival and function of the islets.

This study consisted of implanting islets from allogeneic rats (Wistar) - encapsulated in AdoShell into immunocompetent diabetic rats (Lewis). The insulin secreted by the transplanted islets was measured for 132 days and no slowing of secretion was observed during the duration of the study.

At the end of the study the graft was removed, which resulted in an observable drop of insulin secretion and rise in blood sugar levels, the animals rapidly returned to its diabetic state. At the same time, the animals in the control group (diabetic rats that did not receive AdoShell Islets) were unable to control their blood sugar levels.

Additional ongoing studies in diabetic rats, with the aim to optimize the AdoShell technology, confirm these initial results, producing insulin and normalizing the glycemia in 4 diabetic rats for 80 days (study still on-going). The weight gain of the studied rats - which is also an important clinical indicator of healthy test subjects - shows that the AdoShell Islets are performing as expected. In parallel the rats in the control group are not gaining weight as expected in diabetic rats.

These results will be presented at the upcoming cell therapy session of the PODD 2022 conference held in Boston in October.

"This first proof of concept in diabetic rats validates our AdoShell technology. Our purely physico-chemical approach is unique and being not biological it gives us confidence that these remarkable results can be translated from one species to another," said Olivier Soula, Deputy-CEO and Director of R&D at Adocia.

Our priority, treating life threatening cases with cells from donors

More than 40 million people worldwide suffer from type 1 diabetes1, also known as insulin-dependent diabetes: In these patients, the beta cells of the islets of Langerhans, cells that secrete insulin, are destroyed by an autoimmune mechanism. As a result, the patient survival depends on daily injections of insulin.

Despite the use of insulin, some patients have intensely unstable diabetes characterized by extreme glycemic variability, responsible for iterative and/or severe unfelt hypoglycemia, altering the quality of life and increasing morbidity and mortality. The prognosis of this so-called "brittle" diabetes is poor, with a mortality rate between 20 to 50% over 5 years, depending on the study2. Brittle diabetes affects about 3 out of 1000 people with insulin-dependent diabetes, which represents 1000 patients in France and nearly 75 000 worldwide.

Cell therapy techniques by replacing cells that have been destroyed exist and consist in injecting the patient with islets of Langerhans taken from pancreas of donors. These techniques are practiced in many countries and in 2020 the French High Authority for Health (the HAS) gave a favorable opinion on the registration of islets transplantation on the list of procedures that can be reimbursed by the public Health Insurance. However, this technique has a major pitfall because - like any allograft - islet transplantation as practiced to date requires the concomitant use of heavy immunosuppressive treatments to avoid rejection of the transplanted cells. These immunosuppressive protocols, whose undesirable effects are widely documented (hematological anomalies, infections, and neoplasia), limit the use of transplantation techniques to patients already under immunosuppressive treatment because they are also undergoing kidney transplantation.

The first application of AdoShell Islets concerns the improvement of these techniques performed with donor pancreases and is precisely aimed at these so-called "brittle" patients so that they can benefit from them.

"Our approach is first and foremost very pragmatic: to use donor cells already used in current therapeutics and to fit into existing protocols. In this way, we hope to make a first treatment available to the most severe patients as soon as possible, said Grard Soula, Chairman and CEO of Adocia.

A technology applicable to other cellular sources with the objective of treating the greatest number of people

In parallel with the development of AdoShell Islets from donor pancreases, Adocia also aims to develop its technology from stem cells, which would ultimately make this technology possible to free itself from the limit of the number of donors and thus treat a much larger number of patients.

"We are currently working on setting up collaborations with companies that develop stem cells with an ambitious vision: to offer the best curative treatment for diabetes without requiring immunosuppressants," concluded Grard Soula, Chairman and CEO of Adocia.

During the virtual conference held on September 20th to release the financial results of the first half of 2022, we will present the AdoShell Islets program in detail to investors and shareholders, along with the company's entire program portfolio.

About Adocia

Adocia is a biotechnology company specializing in the discovery and development of therapeutic solutions in the field of metabolic diseases, primarily diabetes and obesity. The company has a broad portfolio of drug candidates based on three proprietary technology platforms:

1) The BioChaperone technology for the development of new generation insulins and products combining insulins with other classes of hormones; 2) AdOral, an oral peptide delivery technology; 3) AdoShell, an immunoprotective biomaterial for cell transplantation with a first application in pancreatic cells transplantation for patients with "brittle" diabetes.

Adocia holds more than 25 patent families.

Based in Lyon, the company has approximately 115 employees. Adocia is listed on the Euronext Paris market (Euronext: ADOC; ISIN: FR0011184241).

Disclaimer

This press release contains certain forward-looking statements concerning Adocia and its business. Such forward-looking statements are based on assumptions that Adocia considers as being reasonable. However, there can be no guarantee that the estimates contained in such forward-looking statements will be achieved, as such estimates are subject to numerous risks including those which are set forth in the Risk Factors section of the universal registration document that was filed with the French Autorit des marchs financiers on April 21, 2022 (a copy of which is available at http://www.adocia.com, in particular uncertainties that are linked to research and development, future clinical data, analyses, and the evolution of the economic context, the financial markets and the markets in which Adocia operates.

The forward-looking statements contained in this press release are also subject to risks not yet known to Adocia or not considered as material by Adocia as of this day. The occurrence of all or part of such risks could cause that actual results, financial conditions, performances, or achievements of Adocia be materially different from those mentioned in the forward-looking statements.

__________________________1 International Diabetes Federation, around 10% of all people with diabetes2 HAS juillet 2020 https://www.has-sante.fr/jcms/p_3195137/en/transplantation-d-lots-pancreatiques-rapport-d-evaluation-technologique

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ADOCIA Announces First Cell Therapy Preclinical Proof of Concept of AdoShell Islets for the Treatment of Type 1 Diabetes - Business Wire