Orgenesis and Theracell to launch point-of-care cell and gene therapy centers in HYGEIA Group"s hospitals – Proactive Investors USA & Canada

CEO Vered Caplan says the move would enable the development and delivery of cell and gene therapies onsite at HYGEIA's hospitals in Greece

Inc (), a developer of advanced cell therapies, revealed Friday that it struck a strategic partnership agreement between the Theracell joint venture and the large HYGEIA Group which runs three hospitals in Greece.

In a statement, the Germantown, Maryland-based company said that under the terms of the agreement, the joint venture will implement point-of-care cell therapy platform for clinical development and commercialization of cell and gene therapies within the HYGEIA Groups network of hospitals in Greece.

and TheraCell Advanced Biotechnology earlier formed a joint venture to advance point-of-care platform in Greece, the Balkan region and some Middle Eastern countries.

The point-of-care platform is designed to collect, process and supply cells within the patient care setting for various treatments.

The main goal is to reduce the cost and complexity of supplying cell and gene therapies, said , as well as boostquality by integrating automated processing units and proprietary technologies.

Significantly, HYGEIA is the first hospital network in the region to implement Orgenesis point-of-care cell therapy platform. The partnership aims to provide the HYGEIA Group with resources to advance clinical development and deliver personalized, advanced therapies across its network for a range of diseases in oncology, hematology, orthopedics, nephrology, dermatology and diabetes.

This partnership with the HYGEIA Group further validates the significant value proposition of our point-of-care platform, as it enables the development and delivery of cell and gene therapies onsite at hospitals, said Orgenesis CEO Vered Caplan.

We believe this platform has the potential to transform the cell and gene therapy market, by bringing life-saving therapies to market in a much more time and cost-effective manner, she added.

The Orgenesis boss said Theracell had proved to be an ideal partner with extensive experience and capabilities in autologous cell therapy and regenerative medicine, and strong operations in Greece and relationships in the region.

We are in active discussions to establish PoCare locations and partnerships with hospitals and healthcare networks in other countries and regions across the world, said Caplan.

Greeces HYGEIA Group operates three hospitals with a capacity of 1,261 beds, 52 operating rooms, 19 delivery rooms and 10 intensive care units.

HYGEIA Group CEO Andreas Kartapanis said thanks to the partnership with Theracell and Orgenesis it would be the first hospital network in Greece to provide advanced cell and gene therapies.

We believe this partnership will provide us a strong competitive advantage in this rapidly developing field. More importantly, this partnership will benefit patients that will now have greater access to these important therapies, said Kartapanis.

For the fiscal third quarter ended September 30, Orgenesis generated meaningful revenue, over $1 million, through its rapidly advancing point-of-care cellular therapy platform.

Meanwhile, TheraCell has experience in the isolation, processing and application of adipose-derived stem cells, as well as somatic cells. It has developed a patented platform for tissue engineering and cell therapies in the areas of dermatology, articular cartilage defects, and chronic kidney injury.

Contact Uttara Choudhury at[emailprotected]

Follow her onTwitter:@UttaraProactive

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Orgenesis and Theracell to launch point-of-care cell and gene therapy centers in HYGEIA Group"s hospitals - Proactive Investors USA & Canada

Stem Cell Therapy Industry 2019 Global Market Size, Trends, Revenue, Growth Prospects, Key Companies and Forecast by 2023 – Techi Labs

Bone marrow transplant is the most widely used stem-cell therapy, but some therapies derived from umbilical cord blood are also in use. Research is underway to develop various sources for stem cells, and to apply stem-cell treatments for neurodegenerative diseases and conditions, diabetes, heart disease, and other conditions.

With the ability of scientists to isolate and culture embryonic stem cells, and with scientists growing ability to create stem cells using somatic cell nuclear transfer and techniques to create induced pluripotent stem cells, controversy has crept in, both related to abortion politics and to human cloning. Additionally, efforts to market treatments based on transplant of stored umbilical cord blood have been controversial.

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Analysis ofStem Cell Therapy Industry Key Manufacturers:

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Stem Cell Therapy Industry 2019 Global Market Size, Trends, Revenue, Growth Prospects, Key Companies and Forecast by 2023 - Techi Labs

Rocket Pharmaceuticals Announces First Patient Treated in Global Registrational Phase 2 Study of RP-L102 Process B for Fanconi Anemia – BioSpace

NEW YORK--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces that the first patient in the global Phase 2 registration-enabling study of RP-L102 Process B received investigational therapy. RP-L102 is the Companys lentiviral vector (LVV)-based gene therapy for the treatment of Fanconi Anemia (FA).

The initiation of Rockets first Phase 2 trial is an important milestone for the company as well as patients throughout the world battling FA, said Kinnari Patel, Pharm.D., MBA, Chief Operating Officer and Head of Development of Rocket. With the recent feedback received from the FDA and EMA of MMC-resistance as the primary endpoint, we are optimistic about the prospect of benefiting patients and, if the data are positive, working towards BLA and MAA submissions.

The registrational package will include twelve patients from the U.S. and EU, two from the U.S. Phase 1 study and 10 additional patients from the global Phase 2 study (NCT04069533). Patients will receive a single intravenous infusion of RP-L102 that utilizes fresh cells and Process B which incorporates a modified stem cell enrichment process, transduction enhancers, as well as commercial-grade vector and final drug product. Improved mitomycin-C (MMC) resistance in bone marrow colony forming (progenitor) cells is the primary endpoint, and may also serve as a surrogate endpoint for accelerated approval. Additional outcome measures include stability or increase in blood counts with no significant worsening in anemia, neutropenia or thrombocytopenia and peripheral blood and bone marrow genetic correction, as demonstrated by progressive increases in vector copy number (VCN) over the months subsequent to infusion.

Lucile Packard Childrens Hospital Stanford and Hospital Infantil Universitario Nio Jess are serving as the lead clinical sites and University of Minnesota is conducting centralized evaluation of bone marrow MMC-resistance and engaging in advisory activities for the global trial of RP-L102. RP-L102 was in-licensed from the Centro de Investigaciones Energticas, Medioambientales y Tecnolgicas (CIEMAT), Centro de Investigacin Biomdica en Red de Enfermedades Raras (CIBERER), Instituto de Investigacin Sanitaria Fundacin Jimnez Daz (IIS-FJD) and Fundacion para la Investigacion Biomedica Hospital Infantil Universitario Nio Jesus (FIB-HIUNJ).

About Fanconi Anemia

Fanconi Anemia (FA) is a rare pediatric disease characterized by bone marrow failure, malformations and cancer predisposition. The primary cause of death among patients with FA is bone marrow failure, which typically occurs during the first decade of life. Allogeneic hematopoietic stem cell transplantation (HSCT), when available, corrects the hematologic component of FA, but requires myeloablative conditioning, which is highly toxic for the patient. HSCT is frequently complicated by graft versus host disease and also increases the risk of solid tumors, particularly upper aerodigestive tract squamous cell carcinomas. Approximately 60-70% of patients with FA have a FANCA gene mutation, which encodes for a protein essential for DNA repair. Mutations in the FANCA gene leads to chromosomal breakage and increased sensitivity to oxidative and environmental stress. Chromosome fragility induced by DNA-alkylating agents such as mitomycin-C (MMC) or diepoxybutane (DEB) is the gold standard test for FA diagnosis. These assays can further differentiate FA patients from mosaic patients. Somatic mosaicism occurs when there is a spontaneous reversion mutation that can lead to a mixed chimerism of corrected and uncorrected bone marrow cells leading to stabilization or correction of an FA patients blood counts in the absence of any administered therapy. Somatic mosaicism provides strong rationale for the development of FA gene therapy and demonstrates the selective advantage of gene-corrected hematopoietic cells in FA1.

1Soulier, J.,et al. (2005) Detection of somatic mosaicism and classification of Fanconi anemia patients by analysis of the FA/BRCA pathway. Blood 105: 1329-1336

About Rocket Pharmaceuticals, Inc.

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

Rocket Cautionary Statement Regarding Forward-Looking Statements

Various statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to successfully demonstrate the efficacy and safety of such products and pre-clinical studies and clinical trials, its gene therapy programs, the pre-clinical and clinical results for its product candidates, which may not support further development and marketing approval, the potential advantages of Rocket's product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's and its licensors ability to obtain, maintain and protect its and their respective intellectual property, the timing, cost or other aspects of a potential commercial launch of Rocket's product candidates, Rocket's ability to manage operating expenses, Rocket's ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Quarterly Report on Form 10-Q for the quarter ended September 30, 2019, filed November 8, 2019. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Announces First Patient Treated in Global Registrational Phase 2 Study of RP-L102 Process B for Fanconi Anemia - BioSpace

Brave new world? Why the public might be ready for gene-edited babies – Genetic Literacy Project

Should we be able to genetically design our children before they are born giving them the eye and hair color we prefer, deciding their sex and height, and even genetically manipulating their intelligence? Most, including top scientists and genetic counselors, say no. But what if the genetic manipulation was done in order to cure a debilitating or potentially fatal illness before the individual was even born? And what if this one genetic treatment could save the individuals descendants from inheriting the disorder, too? A 2018 meeting of non-scientists in Germany indicated that public opinion may be moving in the yes direction.

Though gene editing technology isnt yet at the point where we can genetically alter unborn individuals at the clinical level by applying gene editing tools like CRISPR/Cas9 to embryos, sperm or egg cells the technology is rapidly advancing.

Debate over the subject was rekindled last year, when a Chinese researcher announced that he used CRISPR to create the worlds first gene-edited babies. According to the Associated Press, He Jiankui said he altered embryos for seven couples during fertility treatments. The treatment was designed to protect the children of an HIV-positive parent by disabling a gene that would have allowed the virus to enter their cells. The trial resulted in one pregnancy twins who were born in November. According to the Associated Press, the researcher said:

I feel a strong responsibility that its not just to make a first, but also make it an example. Society will decide what to do next.

The gene-editing community has grappled with the unexpected announcement in the following months. It was not well received by other researchers, with many of them condemning the action. Feng Zhang, one of the inventors of CRISPR, called for an moratorium on gene-edited babies. Zhang wrote:

Not only do I see this as risky, but I am also deeply concerned about the lack of transparency surrounding this trial. All medical advances, gene editing or otherwise and particularly those that impact vulnerable populations, should be cautiously and thoughtfully tested, discussed openly with patients, physicians, scientists, and other community members, and implemented in an equitable way.

Its not unusual to see articles and reports in the media focusing on the worst possible scenarios when it comes to genome editing, as is often the case with new or particularly disruptive technologies. The mere mention of the topic tends to incite fears of a New Eugenics master race or exacerbation of medical inequalities along class lines where the poor are barred from genetic interventions and the rich have easy access. Still others, such as some Catholic and Muslim groups, worry that genetic manipulation of any sort is interfering with divine plans.

These kinds of fears have been gradually loosening their hold on our imaginations, while the coolly-measured possibilities of gene editing nothing more than a new and innovative medical tool are winning over. And as with any new technology, it comes with a particular set of risks and benefits that must be taken into consideration. The slow change in public attitude toward gene editing is evident in groups such as Germanys Citizens Delphi Germline Therapy project at Karlsruhe Institute of Technology (KIT).

After debating detailed information on the risks and benefits over the course of a few months, and undergoing a rigorous participation process that combined aspects of Citizens Jury with the Delphi survey method, the group has called for the loosening of bans on germline gene editing research in Germany. Their final report was presented at Berlin Science Week in late 2018. And though their verdict only applies to the current ban in Germany, which includes a ban on basic research in germline cells, the groups decision could have an impact on regulations around the world. Indeed, the participants were keen on having Germany play a more active role in the development of international gene editing guidelines.

Regarding the Citizens Jury and Delphi survey methods used, Ralf Grtker, who developed and carried out the project in collaboration with the Department of Science Communication at the Institute for German Studies at KIT, stated, The process is geared towards working on a complex topic with a group of laypeople, empower them to make an informed judgment, and eventually reach recommendations for politicians. Though the group only consisted of 26 German citizens, the group was representative of the population, and the topic of gene editing was thoroughly investigated from a variety of angles.

The group acknowledged the risks of genome editing, such as unknown and off-target effects, but ultimately agreed that the way forward was to open the doors to germline gene editing research at the national and global levels. Their conclusions also echoed those made by genetics experts in a position statement published in the August 2017 issue of the American Journal of Human Genetics, which stated, At this time, given the nature and number of unanswered scientific, ethical, and policy questions, it is inappropriate to perform germline gene editing that culminates in human pregnancy.

But to answer those scientific, ethical, and policy questions, basic research is crucial. And lines of communication need to be open between countries, and between scientists and non-scientists. Without these measures, misunderstandings and missed clinical opportunities will proliferate, along with the dangers of underground gene editing and gene editing tourism. Several governments around the world are grappling with heated and unresolved debates regarding germline gene editing, which in several cases appear to be impeding research.

Though a great deal of cooperation and knowledge exchange is taking place between countries, other nations have yet to move away from all-out bans on germline gene editing. Canada, for example, has criminalized germline gene editing under the 2004 Assisted Human Reproduction Act. Penalties for the crime of germline gene tampering include a $500,000 CAD fine and up to 10 years of jail time.

Following a presentation at the annual Till & McCulloch Meetings of stem-cell and regenerative-medicine researchers, Bartha Knoppers, a health policy expert at McGill University in Montreal, said, Canadas policy has simply shut down discussion (about gene editing). We need to start to talk. Some Canadian scientists worry that Canada is lagging behind in gene editing research, unable to fully participate in the global conversation taking place on this important subject.

At the other end of the spectrum are countries like Japan, where a proposal allowing for the use of gene editing tools in human embryos has been drafted. The new guidelines would restrict manipulation of human embryos for reproduction, though this restriction would likely not be legally binding.

Though several countries have formal bans in place restricting germline gene editing, many of these same countries allow for somatic (or non-reproductive) gene editing applications, which is when gene editing tools are applied to adult cells. Somatic gene edits are not passed on to future generations, so there is less concern about the possible repercussions.

A version of this article previously ran on the GLP on Nov 27, 2018.

Kristen Hovet covers genetics, medical innovations, and the intersection of sociology and culture. Follow her on her website or Twitter @kristenhovet

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Brave new world? Why the public might be ready for gene-edited babies - Genetic Literacy Project