StemCell Therapy

PTC Therapeutics announced a strategic partnership with Aldevronto ensure the production of high-quality plasmid DNA to be used with PTCs investigational gene therapies, including AGIL-AS for the treatment of Angelman syndrome (AS).

PTCs growing gene therapy pipeline for genetic disorders of the central nervous system (CNS) also includes an investigational gene therapy for AADC deficiencythats nearing submission to the U.S. Food and Drug Administration (FDA), as well as candidates for Friedreichs ataxiaand Angelman syndrome that are at earlier development stages. Other candidates for cognitive disorders and inherited retinal disorders are in preclinical research.

Our strategic collaboration with Aldevron represents our continued commitment to produce and provide the highest quality product to patients, Neil Almstead, PhD, PTCs chief technical operations officer, said in a press release.

Our gene therapy pipeline is addressing the unmet needs of multiple patient populations, and we feel an urgent need to develop safe products with the utmost speed. The development of relationships with top-tier companies like Aldevron aligns with our goal of partnering with the best collaborators as we drive meaningful improvements in the lives of patients, Almstead said.

PTCs gene therapy candidate for Angelmans syndrome is called AGIL-AS. It uses a modified virus that does not cause infection called an adeno-associated virus (AAV) to deliver a working copy of the UBE3Agene, the faulty gene in Angelman syndrome, to the brain and spinal cord of patients. The process is designed to restore production of the E6-AP protein produced by the UBE3A gene, this way improving cell function and rescuing neurological defects in Angelman syndrome.

Preclinical studieshave shown that AGIL-AS targets nerve cells in the brain, increases levels of E6-AP, and eases AS-like cognitive deficits in animal models of the disease.

AGIL-AS was granted orphan drug designationfrom the U.S. Food and Drug Administration in 2015, followed by a similar designation from theEuropean Commission in 2016.

Under the agreement, Aldevron will manufacture the plasmid DNA (circular molecules of DNA) where the functional version of UBE3A gene will be enclosed for delivery. The company ensures the materials are produced under Good Manufacturing Practice (GMP), a set of guidelines allowing products to be consistently made and controlled according to quality standards.

It is truly an honor to work with PTCs motivated team of experts. They are making enormous contributions to the future of genetic medicine, saidMichael Chambers, founder and CEO of Aldevron.

This is Aldevrons mission to serve scientists and researchers who are relentlessly pursuing cures for people who need them, he added.

Ana is a molecular biologist enthusiastic about innovation and communication. In her role as a science writer she wishes to bring the advances in medical science and technology closer to the public, particularly to those most in need of them. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she focused her research on molecular biology, epigenetics and infectious diseases.

Total Posts: 11

Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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PTC, Aldevron Partner to Advance Angelman and Other Gene Therapy Candidates - Angelman Syndrome News

WATERTOWN, Mass. and RESEARCH TRIANGLE PARK, N.C., Nov. 07, 2019 (GLOBE NEWSWIRE) -- SQZ Biotechnologies (SQZ), and Asklepios BioPharmaceutical, Inc. (AskBio), announced a research collaboration to create tolerizing antigen carriers (TACs) containing AAV (adeno-associated virus) components to solve one of gene therapys biggest challenges the barrier to treatment posed by patients immune systems generating neutralizing antibodies toward therapeutic AAVs. SQZ and AskBio will combine their proprietary cell and gene therapy platform technologies to open the door to new treatment paradigms with potential impact across many genetic diseases.

Gene therapies utilizing AAV vectors can be transformative for patients with genetic diseases, but neutralizing antibodies can prevent large populations of patients from benefitting from AAV gene therapies. Patients immune systems develop neutralizing antibodies after receiving their first dose of AAV, or they can be pre-existing. This collaboration will strive to give these patients access to novel therapeutics and enable them to take multiple or repetitive doses to gain the full, durable benefit these treatments can provide. Expanding patient eligibility and allowing repeat treatment could change the future of how products are developed and significantly impact the long-term health of millions in need.

This is a tremendous opportunity to bring together the power of both cell and gene therapy for patients. AskBio has been an innovative leader in gene therapy and shares our patient-centric philosophy. By working together and leveraging the potential of both our platforms, we hope to bring more effective, more durable treatments to patients suffering from devastating rare genetic disorders, said Armon Sharei, PhD, founder and chief executive officer of SQZ Biotech.

The collaboration between SQZ and AskBio will evaluate the administration of SQZ TACs and AskBios gene therapies to potentially address AAV immunogenicity. SQZ is a pioneer in cell therapy, and the companys knowledge and expertise, as well as their advance capabilities in manufacturing, are critical to this collaborations approach to synergizing cell and gene therapies. Preclinical data from SQZ has demonstrated that SQZ TACs specifically inhibit undesired immune responses in multiple contexts, including AAV models. As a leader in the AAV field, AskBio brings expertise in AAV technology, capsid design, clinical processes and manufacturing that would allow for application of these novel methods to overcome immunogenicity. The two companies have a shared goal to increase world-wide access of transformative therapeutics.

R. Jude Samulski, PhD, chief scientific officer and co-founder of AskBio, noted, AskBio is firmly committed to improving the lives of underserved patients, such as those suffering from Pompe, Huntingtons and various neuromuscular and central nervous system diseases. Addressing AAV immunogenicity is essential to the future of gene therapy as it is one of the most significant limiting factors plaguing the gene therapy space today. SQZs pioneering approach to tolerance could offer a solution to this problem. Our collaboration with SQZ is exemplary of our goal to broadly explore potential redosing of AAV gene therapies, added Sheila Mikhail, chief executive officer and co-founder of AskBio. We are thrilled to be working with SQZ and are hopeful that this initial research collaboration utilizing two of the most promising therapeutic modalities currently available, cell and gene therapy, will ultimately provide options to improve patients immune response to gene therapy.

About AskBioFounded in 2001, Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, clinical-stage gene therapy platform company dedicated to improving the lives of children and adults with genetic disorders. AskBios gene therapy platform includes an industry-leading proprietary cell line manufacturing process known as Pro10 and an extensive AAV capsid library. Based in Research Triangle Park, N.C., the company has generated hundreds of proprietary third-generation gene vectors, several of which have entered clinical testing. An early innovator in the space, the company holds more than 500 patents in areas such as AAV production, chimeric vectors, and self-complementary DNA. AskBio maintains a portfolio of clinical programs across a range neurodegenerative and neuromuscular indications with a current pipeline that includes therapeutics for Pompe disease, Limb Girdle Muscular Dystrophy and congestive heart failure as well as out-license clinical indications for Hemophilia (Chatham Therapeutics acquired by Takeda) and Duchenne Muscular Dystrophy (Bamboo Therapeutics acquired by Pfizer). For more information, visit http://www.askbio.com.

About SQZ BiotechSQZ Biotech is a privately held, clinical-stage company creating innovative treatments by transforming cells into sophisticated therapeutics. Using its proprietary platform, SQZ has the unique ability to precision engineer virtually any cell type and deliver multiple materials, potentially resulting in powerful, multifunctional cell therapies for a range of diseases with an initial focus on cancer and autoimmune disease. The companys initial applications leverage SQZs ability to generate target-specific immune responses, both in activation for the treatment of solid tumors, and immune suppression for the treatment of immune reactions and diseases. For more information please visit http://www.sqzbiotech.com.

About SQZ TACsSQZ tolerizing antigen carriers (TACs) are being developed to induce tolerance to aberrant or unwanted immune activity. TACs are developed from red blood cells (RBCs) SQZd with target-specific antigens and piggyback on the natural process of RBC destruction in the body, also known as eryptosis. A process moderated by our liver and spleen, eryptosis causes macrophages to take up aged or senescent RBCs. When our bodies process RBCs for destruction, their components are presented in a tolerogenic manner, reminding our immune systems not to attack our own red blood cells. SQZ TACs drive targeted antigensthrough this powerful natural mechanism, specifically tolerizing the immune system, potentially stopping undesired immune responses.

AskBio Contacts: Mark Rosenbergmark@trueparallel.com919-412-7378

Roger Friedensen, APRroger@trueparallel.com919-349-3206

SQZ Contacts:Rebecca CohenSenior Manager, Corporate Relationsrebecca.cohen@sqzbiotech.com617-758-8672 ext. 728

Cait Williamson, PhDLifeSci Public Relations cait@lifescipublicrelations.com646-751-4366

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SQZ Biotech and AskBio Announce Research Collaboration to Create Immune Tolerization Products for AAV Gene Therapies - GlobeNewswire

Cell therapy CDMO Cognate Bioservices will add plasmid DNA and viral vector capabilities through the acquisition of Swedish manufacturer Cobra Biologics.

Memphis, Tennessee-based contract development and manufacturing organization (CDMO) Cognate has entered into an agreement to acquire Cobra for an undisclosed fee, led by existing Cognate investor EW Healthcare Partners.

The deal adds to Cognates presence in the regenerative medicine space by bringing on board plasmid DNA and viral vector manufacturing capacity and expertise, complementing its own autologous and allogeneic cell-based and cell-mediated gene therapy capabilities.

Image: iStock/Good_Stock

According to Cognate, the deal will create a fully integrated cell and gene therapy CDMO providing more scalable solutions to its clients.

This acquisition is central to Cognates strategy to build on its existing offerings and create an enterprise platform for life cycle management of cell and gene therapy products, accelerating the availability of new technologies to patients that need them most, said Cognate CEO J. Kelly Ganjei.

The combined Cognate-Cobra expertise, infrastructure, and geographical footprint immediately positions both businesses to better respond to current and future market needs more quickly, effectively, and comprehensively.

The transaction is subject to receipt of approval for the US authorities under the Hart-Scott-Rodino Antitrust Improvements Act of 1976.

Peter Coleman, CEO of Cobra Biologics, told Bioprocess Insider the two companies will operate as different business units and he will continue to be involved with Cobra following the acquisition.

There will be elements of integration, in particular linking the technical capabilities together, but the intention is to operate as two separate business units each with its own specialization and track record.

Cognate operates an 80,000 square-foot site in Memphis, originally built in 2017 for autologous cell therapy manufacturing but now produces various cell types for customers clinical projects. Our largest phase of clinical grade production was an autologous product for phase III clinical trials, mostly manufactured in Memphis with products shipped to more than 80 clinical sites in four countries, the company states.

Cobra brings to the table two GMP approved facilities: an advanced therapy medicinal product (ATMP) production site in northwest UK offering DNA and viral vector services, and an ATMP production site in Matfors, Sweden offering DNA and microbiota services.

Last month, Cobra inked a deal to manufacture the adeno-associated viral vector (AAV) for Nordic gene therapy company Combigenes epilepsy candidate CG01 from its facility in the UK.

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Cognate buys Cobra to boost gene therapy CDMO - Bioprocess Insider - BioProcess Insider

A team of scientists from City University of Hong Kong (CityU) and the Karolinska Institute has created a novel protein that can increase the target accuracy in genome editing. Their findings are published in the journal Proceedings of the National Academy of Sciences (PNAS).Meet CRISPRThe gene editing technology Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Cas9 looks set to revolutionize modern medicine, agriculture, and synthetic biology.The ability to edit the genome in vivo offers the potential to develop novel gene therapies for diseases that currently lack viable treatment options. Several clinical trials are underway exploring the utility of CRISPR technology in treating specific cancers, blood disorders and eye diseases.CRISPR-Cas9 as a gene editing tool is superior over other techniques due to its ease of use. In traditional gene therapy, additional copies of the "normal" gene are introduced into cells. Using CRISPR technology, this isnt necessary; CRISPR-Cas9 enters the cell and "repairs" the problematic gene by removing it or correcting it to restore normal physiological function.

There are different components to the CRISPR mechanism. Cas9 is the enzyme that flags and locates the problematic DNA throughout the genome, acting in a "hunting" fashion. However, the precision of Cas9 cannot always be established, and occasionally modifications of DNA at unintended places can occur. If CRISPR is to be utilized to repair faulty genes in patients, potential off-target genome editing could have serious adverse effects.

There are currently two versions of the Cas9 enzyme commonly adopted in CRISPR research: SpCas9 (Cas9 nuclease from the bacteria Streptococcus pyogenes) and SaCas9 (Cas9 nuclease from Staphylococcus aureus). Both of these enzymes are limited in that they possess a certain level of imprecision.

Thus, scientists have endeavored to develop variants of both enzymes, with the aim being to increase their precision and reduce off-target effects. The issue with SpCas9 is that the modified variants are often too large to "fit" in the delivery system adopted for inserting gene therapies into patients, known as adeno-associated viral (AAV) vectors.SaCas9 is advantageous over SpCas9 in that it can be easily packaged into the AAV vectors for delivering gene-editing contents in vivo. However, at present, there is no SaCas9 variant that possesses high accuracy in genome-wide editing. Until now.Now meet SaCas9-HFIn the new study published in Proceedings of the National Academy of Sciences (PNAS), a research team led by Zheng Zongli, Assistant Professor of Department of Biomedical Sciences at CityU and the Ming Wai Lau Centre for Reparative Medicine of the Karolinska Institute in Hong Kong, and Shi Jiahai, Assistant Professor of Department of Biomedical Sciences at CityU, has successfully engineered SaCas9-HF, a CRISPR Cas9 variant which has demonstrated high accuracy in genome-wide targeting in human cells without compromising on-target efficiency.In the study, the scientists conducted an extensive evaluation of 24 targeted human genetic locations comparing the original (known as wild-type) SaCas9, and the new variant, SaCas9-HF. They discovered that for targets with highly similar sequences in the genome (and therefore often disposed to off-target editing by wild-type Cas9), SaCas9-HF decreased the off-target activity by ~90%. When assessing targets that had relatively less off-targeting editing by wild-type SaCas9, the SaCas9-HF enzyme produced little to no detectable off-target effects.

"Our development of this new SaCas9 provides an alternative to the wild-type Cas9 toolbox, where highly precise genome editing is needed. It will be particularly useful for future gene therapy using AAV vectors to deliver genome editing 'drug' in vivo and would be compatible with the latest 'prime editing' CRISPR platform, which can 'search-and-replace' the targeted genes," said Dr Zheng.Reference: Tan et al. 2019. Rationally engineered Staphylococcus aureus Cas9 nucleases with high genome-wide specificity. Proceedings of the National Aacademy of Sciences (PNAS). DOI: https://doi.org/10.1073/pnas.1906843116

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A Highly Precise Cas9 Enzyme, SaCas9-HF, Is Added to the CRISPR Toolbox - Technology Networks

7-Nov-2019

Design and Build

Canadian cleanroom builder to work in the US on a new 1,600-sq-ft site of ISO Class 7

The New Jersey Innovation Institute is located in Newark, in the United States

Mecart, the Canadian cleanroom specialist, has been selected by the US-based New Jersey Innovation Institute (NJII) for a new GMP cleanroom at its Cell and Gene Therapy Development Center. The NJII is based in Newark, US.

Commenting on the project with Mecart, Dr Haro Hartounian, NJII Senior Executive Director, Biotechnology and Pharmaceutical Innovation, said: We were excited to meet with the Mecart team, travel to Quebec City to visit with their leadership, and review the project in detail with them. It has been a great experience thus far and we know that we picked the best partner for this critical project.

The project represents a new GMP cleanroom of approximately 1,600 square feet of ISO 7 space developed specifically for advanced cell and gene therapy processing and manufacturing operations.

Its an honour to work with such an innovative company that focuses on local collaboration with its technological resources, like this new cleanroom, said Patrice Genois, General Manager of Mecart and Vice President of PolR.

The NJII is an NJIT corporation that applies the intellectual and technological resources of the states science and technology university to challenges identified by industry partners.

Upon completion, the new GMP site will also serve as a training facility and a venue for collaborating with local manufacturing.

Charles Lipeles, Vice President of US Operations, commented: When NJII approached Mecart, they were clear that lead time was crucial as was a very tight specification for their state-of-the-art GMP cleanroom. They had very aggressive goals, made more challenging when dealing with an institution with government ties, but we were ready for the challenge. We are excited to work with the NJII and NJIT teams and help them exceed their goals with this new suite of cleanrooms."

Construction is planned to begin later this year and will be completed in Q1 of 2020.

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NJ Innovation Institute chooses Mecart for cell and gene therapy centre - Cleanroom Technology

Registrational Trial for Wiskott-Aldrich Syndrome Met Key Primary and Secondary Endpoints at Three Years; Data from Integrated Analysis Reinforce Treatment Benefits of Gene Therapy and Durability of Effect in Additional Patients

Similar Profiles Reported Between Cryopreserved and Fresh Formulations of OTL-101, Further Supporting Upcoming Regulatory Filing and Broad Patient Availability

BOSTON and LONDON, Nov. 06, 2019 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced the upcoming presentation of registrational data from multiple programs at the 61st American Society of Hematology (ASH) Annual Meeting in Orlando, FL.

Investigators will describe ongoing clinical progress for two lead development programs in the companys primary immune deficiencies portfolio: OTL-103, an investigational gene therapy in development for the treatment of Wiskott-Aldrich syndrome (WAS) at theSan Raffaele-Telethon Institute for Gene Therapy(SR-Tiget) inMilan, Italy; and OTL-101, an investigational gene therapy in development for the treatment of adenosine deaminase severe combined immunodeficiency (ADA-SCID).

In addition, investigators will deliver an oral presentation featuring updated data from the ongoing proof-of-concept study of OTL-203, an investigational gene therapy in development for the treatment of mucopolysaccharidosis type I (MPS-I) atSR-Tiget.

This growing body of positive data, from dozens of patients across multiple diseases, provides a solid foundation as we advance each program toward its next phase of development, including upcoming regulatory submissions for ADA-SCID and WAS, saidMark Rothera, president and chief executive officer ofOrchard Therapeutics. We now have two supportive data sets one from our OTL-101 program in ADA-SCID and one from our OTL-200 program in metachromatic leukodystrophy that demonstrate cryopreserved formulations are engrafting as expected, similar to the fresh formulation. This supports our strategy for making these therapies, if approved, broadly available to patients in need throughout the world.

We are extremely pleased with our continued clinical progress, including the duration of benefits seen in our WAS trial, which is the longest published follow-up of hematopoietic stem cell gene therapy durability to date using lentiviral vector transduction, said Bobby Gaspar, M.D., Ph.D., chief scientific officer of OrchardTherapeutics. The totality of these data underscores the broad applicability of our gene therapy platform approach and the opportunity we have to deliver potentially curative treatments for a variety of devastating and rare genetic disorders.

Full presentation details are below:

Poster Presentation Details

Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010Publication Number: 3346Session: 801. Gene Therapy and Transfer: Poster IIDate and time:Sunday, December 8, 6:00-8:00pm ET

This presentation includes results from an integrated analysis of 17 patients treated with OTL-103 for the treatment of WAS, including the complete data set for the eight patients from the registrational study and nine who received OTL-103 as part of an expanded access program (EAP). Participants have been followed for a median of three years.

In the eight-patient registrational trial, investigators reported that the study achieved its key primary and secondary endpoints at three years, including the elimination of severe bleeding episodes and a significant reduction in the frequency of moderate bleeding episodes. Successful engraftment was observed within three months, leading to an increase in WAS protein expression and a vector copy number that has been maintained for up to eight years. Nine months post-administration, all patients stopped receiving platelet transfusions, and no severe bleeding events were reported. A significant reduction in the rate of severe infections was also observed and all patients were able to stop immunoglobin replacement therapy (IgRT), suggesting a complete reconstitution of immune function with durability of effect of up to eight years of follow-up post-gene therapy.

Similar clinical results were seen in the integrated analysis of 17 patients and overall survival was 94% (16/17). One death occurred among the EAP cohort that was considered by the investigator to be unrelated to OTL-103.

Across the original and integrated data sets, there were no adverse events considered to be related to OTL-103, including no evidence of oncogenesis, replication competent lentivirus or abnormal clonal proliferation. Clinical benefit was also attained in patients older than five years of age, a group considered at higher risk when treated with allogeneic hematopoietic stem cell transplantation (HSCT).

Lentiviral Gene Therapy with Autologous Hematopoietic Stem and Progenitor Cells (HSPCs) for the Treatment of Severe Combined Immune Deficiency Due to Adenosine Deaminase Deficiency (ADA-SCID): Results in an Expanded CohortPublication Number: 3345Session: 801. Gene Therapy and Transfer: Poster IIDate and time: Sunday, December 8, 6:00-8:00pm ET

This presentation details the safety and efficacy of OTL-101 in 30 individuals with ADA-SCID, treated with either fresh (n=20) or cryopreserved (n=10) formulations. Patients were followed for a median of 24 months (range 12-24 months overall and 12-18 months for patients treated with the cryopreserved formulation), and results were compared with a historical cohort of 26 ADA-SCID patients treated with allogeneic hematopoietic stem cell transplantation (HSCT), including HSCT both with, and without, a matched related donor.

Results showed engraftment of genetically modified hematopoietic stem cells in 29 of 30 OTL-101 patients by six to eight months, which persisted through follow-up in both studies. Analysis of both the vector copy number in granulocytes (a measure of engraftment) and T-cell reconstitution (a relevant measure of immune recovery) showed consistent performance across the fresh and cryopreserved-treated patients.

In the OTL-101 treated patients, overall survival was 30/30 (100%) and event-free survival was 29/30 (97%). One of the 30 patients restarted treatment with enzyme replacement therapy (ERT) and subsequently withdrew from the study and received a rescue HSCT. In the historical control population, 42% of HSCT patients required re-initiation of ERT, rescue HSCT or other intervention, or died. As expected, there was no incidence of graft versus host disease in the OTL-101 group, compared with eight patients who received HSCT.

Eighteen of 20 patients (90%) in the fresh formulation study stopped immunoglobin replacement therapy (IgRT) after two years, compared to 52% of HSCT patients. Of the seven patients treated with the cryopreserved formulation with 18 months of follow-up, five had discontinued IgRT (71%), which is comparable to the 18-month data for patients treated with the fresh formulation.

Oral Presentation Details

Extensive Metabolic Correction of Hurler Disease by Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II TrialPublication Number: 607Session: 801. Gene Therapy and Transfer: Gene Therapies for Non-Malignant DisordersDate and time:Monday, December 9, 7:00am ET

Investigators will present updated analyses from the ongoing proof-of-concept trial of OTL-203 for mucopolysaccharidosis type I (MPS-I).

About ADA-SCID and OTL-101Severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID) is a rare, life-threatening, inherited disease of the immune system caused by mutations in the ADA gene resulting in a lack of, or minimal, immune system development.1-4The first symptoms of ADA-SCID typically manifest during infancy with recurrent severe bacterial, viral and fungal infections and overall failure to thrive, and without treatment the condition can be fatal within the first two years of life. The incidence of ADA-SCID is currently estimated to be one in 500,000 live births inthe United Statesand between one in 200,000 and one in 1 million inEurope.3OTL-101 is an autologous,ex vivo,hematopoietic stem cell-based gene therapy for the treatment of patients diagnosed with ADA-SCID being investigated in multiple clinical trials inthe United StatesandEurope, including a registrational trial at theUniversity of California, Los Angeles(UCLA). OTL-101 has received orphan drug designation from theU.S. Food and Drug Administration(FDA) and the European Medicines Agency (EMA) for the treatment of ADA-SCID, and Breakthrough Therapy Designation from theFDA.

About WAS and OTL-103Wiskott-Aldrich Syndrome (WAS) is a life-threatening inherited immune disorder characterized by autoimmunity and abnormal platelet function and manifests with recurrent, severe infections and severe bleeding episodes, which are the leading causes of death in this disease. Without treatment, the median survival for WAS patients is 14 years of age. Treatment with stem cell transplant carries significant risk of mortality and morbidities. OTL-103 is anex vivo,autologous, hematopoietic stem cell-based gene therapy developed for the treatment of WAS that Orchard acquired from GSK in April 2018 and has been developed at theSan Raffaele-Telethon Institute for Gene Therapy(SR-Tiget) inMilan, Italy. The global incidence of WAS is estimated to be about 100-260 births per year, with a global prevalence of 2,900-4,700 patients.

About MPS-I and OTL-203Mucopolysaccharidosis type I (MPS-I) is a rare inherited neurometabolic disease caused by a deficiency of the IDUA (alpha-L-iduronidase) lysosomal enzyme required to break down glycosaminoglycans (also known as GAGs or mucopolysaccharides). The accumulation of GAGs across multiple organ systems results in the symptoms of MPS-I including neurocognitive impairment, skeletal deformity, loss of vision and hearing, hydrocephalus, and cardiovascular and pulmonary complications. MPS-I occurs at an overall estimated frequency of one in every 100,000 live births.5There are three subtypes of MPS-I; approximately 60 percent of MPS-I patients have the severe Hurler subtype and, when untreated, these patients rarely live past the age of 10.IdTreatment options for MPS-I include hematopoietic stem cell transplant and chronic enzyme replacement therapy, both of which have significant limitations. Though early intervention with enzyme replacement therapy has been shown to delay or prevent some clinical features of the condition, it has only limited efficacy on neurological symptoms. OTL-203 is anex vivo, autologous, hematopoietic stem cell-based gene therapy being studied for the treatment of MPS-I. Orchard was granted an exclusive worldwide license to intellectual property rights to research, develop, manufacture and commercialize the gene therapy program for the treatment of MPS-I developed by theSan Raffaele-Telethon Institute for Gene TherapyinMilan, Italy.

About Orchard Orchard Therapeuticsis a fully integrated commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies.

Orchards portfolio ofex vivo, autologous, hematopoietic stem cell (HSC) based gene therapies includes Strimvelis, a gammaretroviral vector-based gene therapy and the first such treatment approved by theEuropean Medicines Agencyfor severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID). Additional programs for neurometabolic disorders, primary immune deficiencies and hemoglobinopathies are all based on lentiviral vector-based gene modification of autologous HSCs and include three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD), transfusion-dependent beta-thalassemia (TDT) and mucopolysaccharidosis type I (MPS-I), as well as an extensive preclinical pipeline. Strimvelis, as well as the programs in MLD, WAS and TDT were acquired by Orchard from GSK inApril 2018and originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene TherapyinMilan, Italyinitiated in 2010.

Orchard currently has offices in the UK and the U.S., including London, San Francisco and Boston.

Forward-Looking StatementsThis press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, intends, projects, and future or similar expressions that are intended to identify forward-looking statements.Forward-looking statements include express or implied statements relating to, among other things, the therapeutic potential of Orchards product candidates, including the product candidate or candidates referred to in this release, Orchards expectations regarding the timing of regulatory submissions for approval of its product candidates, including the product candidate or candidates referred to in this release, the timing of announcement of clinical data for its product candidates and the likelihood that such data will be positive and support further clinical development and regulatory approval of these product candidates, including any cryopreserved formulations of such product candidates, and the likelihood of approval of such product candidates by the applicable regulatory authorities. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the risk that any one or more of Orchards product candidates, including the product candidate or candidates referred to in this release, will not be successfully developed or commercialized, the risk of cessation or delay of any of Orchards ongoing or planned clinical trials, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates, the delay of any of Orchards regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates, the receipt of restricted marketing approvals, and the risk of delays in Orchards ability to commercialize its product candidates, if approved.Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading "Risk Factors" in Orchards annual report on Form 20-F for the year endedDecember 31, 2018as filed with theU.S. Securities and Exchange Commission(SEC) onMarch 22, 2019, as well as subsequent filings and reports filed with theSEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

1Orphanet. SCID due to ADA deficiency.2Whitmore KV, Gaspar HB. Front Immunol. 2016;7:314.3Kwan A, et al. JAMA. 2014;312:729-738.4Sauer AV, et al. Front Immunol. 2012;3:265. 5Beck et al. The Natural History of MPS I: Global Perspectives from the MPS I Registry. Genetics in Medicine 2014, 16(10), 759.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

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Orchard Therapeutics to Present New Registrational Data of Investigational Gene Therapies at the 61st American Society of Hematology Annual Meeting -...

St. Jude Childrens Research Hospital and the National Institutes of Health to present updated MB-107 clinical data for the treatment of X-linked severe combined immunodeficiency

Fred Hutchinson Cancer Research Center to present overview of ongoing MB-106 Phase 1/2 clinical trial

NEW YORK, Nov. 06, 2019 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (Mustang) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, announced today that updated Phase 1/2 clinical data for MB-107 lentiviral gene therapy for X-linked severe combined immunodeficiency (XSCID) have been selected for oral and poster presentations at the 61st American Society of Hematology (ASH) Annual Meeting. ASH will be held December 7-10, 2019, at the Orange County Convention Center in Orlando, FL.

MB-107 is currently being assessed in two Phase 1/2 clinical trials for XSCID: the first in newly diagnosed infants under the age of two at St. Jude Childrens Research Hospital, UCSF Benioff Childrens Hospital and Seattle Childrens Hospital and the second in patients over the age of two who have received prior hematopoietic stem cell transplantation at the National Institutes of Health. Positive Phase 1/2 clinical data from the trial for infants under the age of two were published in the New England Journal of Medicine in April 2019 and positive Phase 1/2 clinical data from the trial in patients over the age of two were published in Science Translational Medicine in April 2016. The U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to MB-107 for the treatment of XSCID in August 2019.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, We are extremely pleased that additional clinical data on MB-107, a lentiviral gene therapy for the treatment of XSCID, will be presented in oral and poster sessions at the 2019 ASH Annual Meeting. The curative potential of MB-107 based on previously announced compelling Phase 1/2 data is impressive, and we look forward to working with St. Jude and NIH to advance the development of this important treatment option.

Details of the MB-107 presentations are as follows.

Oral Presentation:Title: Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined ImmunodeficiencySession: 801. Gene Therapy and Transfer: Gene Therapies for Non-Malignant DisordersAbstract Number: 608Date and Time: Monday, December 9, 2019, 7:15 a.m. ET Location: Orange County Convention Center, Valencia BC (W415BC)Presenter: Harry Malech, M.D., Laboratory of Clinical Immunology and Microbiology, NIAID, NIH, Bethesda, MD, USA

Poster Presentation:Title: Lentiviral Gene Therapy with Low Dose Busulfan for Infants with X-SCID Results in the Development of a Functional Normal Immune System: Interim Results of an Ongoing Phase I/II Clinical StudySession: 801. Gene Therapy and Transfer: Poster IAbstract Number: 2058Date and Time: Saturday, December 7, 2019, 5:30-7:30 p.m. ETLocation: Orange County Convention Center, Hall BPresenter: Ewelina Mamcarz, M.D., Department of Bone Marrow Transplantation and Cellular Therapy, St. Jude Childrens Research Hospital, Memphis, TN, USA

In addition, Mustangs collaborator Fred Hutchinson Cancer Research Center will present a poster about the ongoing Phase 1/2 clinical trial investigating the safety and efficacy of MB-106 CD20-targeted CAR T for high-risk B-cell non-Hodgkin lymphomas.

Details of the MB-106 presentation are as follows.

Poster Presentation:Title: CD20 Targeted CAR-T for High-Risk B-Cell Non-Hodgkin LymphomasSession: 704. Immunotherapies: Poster IIAbstract Number: 3235 Date and Time: Sunday, December 8, 2019, 6-8 p.m. ETLocation: Orange County Convention Center, Hall BPresenter: Mazyar Shadman, M.D., M.P.H., Fred Hutchinson Cancer Research Center, Seattle, WA, USA

Copies of the abstracts can be viewed online through the ASH website at http://www.hematology.org.

About Mustang BioMustang Bio, Inc. (Mustang) is a clinical-stage biopharmaceutical company focused on translating todays medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T and CRISPR/Cas9-enhanced CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for XSCID. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission. Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit http://www.mustangbio.com.

ForwardLooking Statements This press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements include, but are not limited to, any statements relating to our growth strategy and product development programs and any other statements that are not historical facts. Forward-looking statements are based on managements current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third-party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in our SEC filings. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law.

Company Contacts:Jaclyn Jaffe and William BegienMustang Bio, Inc.(781) 652-4500ir@mustangbio.com

Investor Relations Contact:Daniel FerryLifeSci Advisors, LLC(617) 430-7576daniel@lifesciadvisors.com

Media Relations Contact:Tony Plohoros6 Degrees(908) 940-0135tplohoros@6degreespr.com

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Mustang Bio Announces MB-107 Lentiviral Gene Therapy and MB-106 CD20-Targeted CAR T Data Selected for Presentations at 61st American Society of...

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Cancer Genome Sequencing Market Report | What will be the Market size and the growth rate by 2025? - Health News Office

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Global Medical Casting & Splinting Market: Sales, Consumption, Demand and Forecast 2019-2023: 3M, DJO Global, BSN Medical, ssur, Zimmer Biomet,...

A New business Strategy report released by HTF MI with title Global Live Cell Imaging Consumables Market Insights, Forecast to 2025 . This Global Live Cell Imaging Consumables market report brings data for the estimated year 2019 and forecasted till 2025 in terms of both, value (US$ MN) and volume (MT). The report also consists of forecast factors, macroeconomic factors, and a market outlook of the Live Cell Imaging Consumables market. The study is conducted using top-down and bottom-up approaches and further analyzed using analytical tools such as porters five force analysis and uncover Opportunities, Challenges, restraints, and trends of the Global Live Cell Imaging Consumables market. This report also states import/export consumption, supply and demand Figures, cost, price, revenue and gross margins. Some of the Major Companies Profiled in the reports are Carl Zeiss AG (Germany), Leica Microsystems (Germany), Nikon Corporation (Japan), Molecular Devices, LCC (U.S.), PerkinElmer, Inc. (U.S.), GE Healthcare (U.K.), Becton, Dickinson and Company (U.S.), Olympus Corporation (Japan), Sigma Aldrich Corporation (U.S.) & Thermo Fisher Scientific, Inc.(U.S.) etc.

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If you are involved in the Live Cell Imaging Consumables industry or intend to be, then this study will provide you complete viewpoint. Its vital you keep your market knowledge up to date segmented by Applications [Cell Biology, Stem Cells, Developmental Biology & Drug Discovery], Product Types such as [, Assay Kits, Reagents, Media & Others] and some major players in the industry.

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2 Exclusive Summary

3 Research Methodology3.1 Primary Interviews3.2 Data Mining3.3 Validation3.4 List of Statistics

4 Live Cell Imaging Consumables Market Segment & Geographic Analysis4.1 By Type [2014 -2025]4.2 By Application [2014-2025]4.3 By Region [2014-2025]

5 Live Cell Imaging Consumables Market Outlook5.1 Overview5.2 Market Dynamics5.2.1 Opportunities5.2.2 Restraints5.2.3 Drivers5.3 Porters Five Force Model5.4 Value Chain Analysis

6 Live Cell Imaging Consumables Market Competitive Landscape6.1 Overview6.2 Key Development Policies6.3 Company Market Standing

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Thanks for reading this article; you can also get individual chapter wise section or region wise report version like North America, Europe or Asia.

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Live Cell Imaging Consumables Market Evolving to a Next-Generation Strategy with New Partnerships, Technologies and Targets by 2023 key players LCC...

Shahid Akhter, editor, ETHealthworld spoke to Dr Na'eem Sadiq, Medical Director, PLEXUS NEURO and STEM CELL RESEARCH CENTER, Bengaluru to know more about stem cell therapy and the challenges associated with it.

STEM CELL : TRENDSStem cell is a word which evokes lot of responses both positive and negative. Few people know that the origin was in the 1800s. The very first bone marrow transplant happened in the year 1968 and then subsequently stem cells have been used for various diseases and much more in blood cancer. They have also been used in chronic neurological disorders, autoimmune disorders and sports injuries.Globally, its all over the world such as in the US, Canada, Germany, China, Ukraine and of course in India as well. In India there are lots of centers and states who have been practicing stem cell technology for quite some time.

STEM CELLS : MARKETThe market is growing since stems cells promise hope for those who have lost hope, where there is no viable treatment and proper cure available for lots of diseases. Stem cells is emerging as a champion for all these people. It was much more available internationally and in the last decade India has taken up.

PLEXUS NEURO AND STEM CELL RESEARCH CENTER- JOURNEYI have been practicing in the field of neuroscience for the last 30 years. Neurosciences is a field where you see patients suffering from chronic diseases. I have been in this field right from early 90s and have been seeing trends changing, but when it comes to neurodegenerative disorders such as Parkinsons, ALS, Multiple Sclerosis, billions of dollars have been spent, new treatment modalities have been found, but nothing has been found to be successful.

We have very strict and rigid eligibility criteria. Once the patient approaches us, we subject the patient to a thorough clinical examination, which lasts anywhere between 2- 3 hours. Once we find that the patient is clinically treatable, or that the patient can be helped, then we subject the patient to other investigations.

The other major difference we have at Plexus is that we do not do only stem cells. Stem cell therapy is a part of our complete regenerative rehabilitation. The program starts after we do the transplant. The patient undergoes rigorous rehabilitation, which includes the entire gamete of practices such as physical therapy, occupational therapy, hand splinting, cognitive rehabilitation therapy, cognitive behavior therapy, speech therapy etc.

We customize and provide a tailor made program as per the patient's needs, with a goal once the patient joins the program and almost all the patients who are in the program get more than what we had aimed at achieving. At the end of the program we evaluate the goals and find that every single patient achieves them. We train the patients as to what they need to do once they finish the program and insist on regular follow up.

We have a team of learned scientists who are all qualified from the UK and our research is ongoing. We are working exclusively in the field of neurosciences to get the best quality of cells and to make it very affordable. Research is on and our data is huge, we will be publishing the results very soon.

PLEXUS : FUTURE PLANSWe have a complete state of the art rehabilitation center where we have some of the best therapists in the world working with us. In fact a few months back we launched one of its kind, Sensory Gym at Plexus and now we have started virtual and augmented reality.

In the last 4-5 years we have received more than 75 national and international awards and we stand as one of the leading regenerative rehabilitation centers not only in India, but in Asia.Our endeavor here is to make the treatment the best possible, to make the cells much more advanced, affordable to also provide the treatment in the shortest possible time.

Continued here:
Cost is a major challenge in stem cells therapy: Dr Na'eem Sadiq - ETHealthworld.com

Nov. 5, 2019 12:00 UTC

HOUSTON & OSAKA, Japan--(BUSINESS WIRE)-- The University of Texas MD Anderson Cancer Centerand Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (Takeda) today announced an exclusive license agreement and research agreement to develop cord blood-derived chimeric antigen receptor-directed natural killer (CAR NK)-cell therapies, armored with IL-15, for the treatment of B-cell malignancies and other cancers.

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20191105005250/en/

Under the agreement, Takeda will receive access to MD Andersons CAR NK platform and the exclusive rights to develop and commercialize up to four programs, including a CD19-targeted CAR NK-cell therapy and a B-cell maturation antigen (BCMA)-targeted CAR NK-cell therapy. Takeda and MD Anderson will also conduct a research collaboration to further develop these CAR NK programs.

Our vision is to improve upon existing treatments by developing armored CAR NKs that could be administered off-the-shelf in an outpatient settingenabling more patients to be treated effectively, quickly and with minimal toxicities, said Katy Rezvani, M.D., Ph.D., professor of Stem Cell Transplantation and Cellular Therapy at MD Anderson. With their expertise in hematologic malignancies and commitment to developing next-generation cell therapies, Takeda is the ideal collaborator to help our team advance CAR NK-cell therapies to patients in need of treatments.

A Novel Approach to Delivering Off-the-Shelf CARs in an Outpatient Setting MD Andersons allogeneic CAR NK platform isolates NK cells from umbilical cord blood and engineers them to express CARs against specified cancer targets. CAR NK cells are modified with a retroviral vector to deliver genes and enhance their effectiveness to attack specific tumors. A CD19 CAR increases the cells specificity for B-cell malignancies while the immunocytokine IL-15 enhances the proliferation and survival of the CAR NK cells in the body.

In contrast to current CAR T-cell therapies that utilize a patients own genetically modified T-cells and require a multi-week manufacturing process, CAR NK cells are intended to be manufactured from a non-related donor source and stored for off-the-shelf use, allowing treatment to be delivered more rapidly.

It is anticipated that the CD19 CAR NK-cell therapy could be administered in an outpatient setting. In an ongoing phase 1/2a clinical study treating patients with relapsed and refractory B-cell malignances, the CD19 CAR NK-cell therapy has not been associated with the severe cytokine release syndrome (CRS) or neurotoxicity observed with existing CAR-T therapies.

The development of MD Andersons CAR NK platform is led by Dr. Rezvani and is further supported by the adoptive cell therapy platform, Chronic Lymphocytic Leukemia Moon Shot and B-Cell Lymphoma Moon Shot, all part of the institutions Moon Shots Program, a collaborative effort to rapidly develop scientific discoveries into meaningful clinical advances that save patients lives.

Takeda: Accelerating the Development of Multiple Next-Generation CAR Platforms MD Andersons CAR NK platform represents the curative potential of cell therapies, which is why we are establishing the CD19 CAR NK as our lead cell therapy candidate in oncology, said Andy Plump, M.D., Ph.D., President of Research and Development at Takeda. We need to work swiftly and with purpose, and as such, we intend to initiate a pivotal study of the CD19 CAR NK in 2021.

In addition to CAR NK-cell therapies, Takeda and its partners are investigating multiple approaches to improving the safety, efficacy and accessibility of first-generation CAR T-cell therapies including gamma delta CAR Ts, induced pluripotent stem cell-derived CAR Ts, CAR Ts targeting solid tumors, and other next-generation approaches. Takeda plans to advance five oncology cell therapies to the clinic by the end of FY20.1 These platforms are being developed both with partners and by applying the expertise of Takedas translational cell therapy engine which provides bioengineering, chemistry, manufacturing and control (CMC), clinical and translational capabilities in a single footprint to overcome many of the manufacturing challenges experienced in cell therapy development.

Takeda is responsible for the development, manufacturing and commercialization of CAR NK products resulting under the agreement. MD Anderson will receive an upfront payment and is eligible to receive development and commercial milestones for each target as well as tiered royalties on net sales of any such CAR NK product.

MD Anderson and Takeda will continue research for the additional targets and CAR NK platform under the direction of a joint research committee. MD Anderson will implement an Institutional Conflict of Interest Management and Monitoring Plan for this research.

About MD Anderson The University of Texas MD Anderson Cancer Center in Houston ranks as one of the world's most respected centers focused on cancer patient care, research, education and prevention. The institutions sole mission is to end cancer for patients and their families around the world. MD Anderson is one of only 50 comprehensive cancer centers designated by the National Cancer Institute (NCI). MD Anderson is ranked No.1 for cancer care in U.S. News & World Reports Best Hospitals survey. It has ranked as one of the nations top two hospitals for cancer care since the survey began in 1990, and has ranked first 15 times in the last 18 years. MD Anderson receives a cancer center support grant from the NCI of the National Institutes of Health (P30 CA016672).

About Takeda Pharmaceutical Company Limited Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Gastroenterology (GI), Rare Diseases and Neuroscience. We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people's lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries and regions. For more information, visit https://www.takeda.com

Forward-Looking Statements This press release and any materials distributed in connection with this press release may contain forward-looking statements, beliefs or opinions regarding Takedas future business, future position and results of operations, including estimates, forecasts, targets and plans for Takeda. In particular, this press release contains forecasts and management estimates related to the financial and operational performance of Takeda, including statements regarding forecasts for Revenue, Operating profit, Adjusted EBITDA, Profit before income taxes, Net profit attributable to owners of Takeda, Basic earnings per share, Amortization and impairment and other income/expense, Underlying Revenue, Underlying Core Earnings margin, Underlying Core EPS and Net Debt. Without limitation, forward looking statements often include the words such as targets, plans, believes, hopes, continues, expects, aims, intends, will, may, should, would, could anticipates, estimates, projects or words or terms of similar substance or the negative thereof. Any forward-looking statements in this document are based on the current assumptions and beliefs of Takeda in light of the information currently available to it. Such forward-looking statements do not represent any guarantee by Takeda or its management of future performance and involve known and unknown risks, uncertainties and other factors, including but not limited to: the economic circumstances surrounding Takedas business, including general economic conditions in Japan, the United States and worldwide; competitive pressures and developments; applicable laws and regulations; the success of or failure of product development programs; decisions of regulatory authorities and the timing thereof; changes in exchange rates; claims or concerns regarding the safety or efficacy of marketed products or products candidates; and post-merger integration with acquired companies, any of which may cause Takedas actual results, performance, achievements or financial position to be materially different from any future results, performance, achievements or financial position expressed or implied by such forward-looking statements. For more information on these and other factors which may affect Takedas results, performance, achievements, or financial position, see Item 3. Key InformationD. Risk Factors in Takedas Registration Statement on Form 20-F filed with the U.S. Securities and Exchange Commission, available on Takedas website at: https://www.takeda.com/investors/reports/sec-filings/ or at http://www.sec.gov. Neither Takeda nor its management gives any assurances that the expectations expressed in these forward-looking statements will turn out to be correct, and actual results, performance or achievements could materially differ from expectations. Persons receiving this press release should not place undue reliance on forward looking statements. Takeda undertakes no obligation to update any of the forward-looking statements contained in this press release or any other forward-looking statements it may make. Past performance is not an indicator of future results and the results of Takeda in this press release may not be indicative of, and are not an estimate, forecast or projection of Takedas future results.

1 Takedas 2020 fiscal year begins April 1, 2020 and ends March 31, 2021.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191105005250/en/

Link:
Takeda and MD Anderson Announce Collaboration to Accelerate the Development of Clinical-Stage, Off-The-Shelf CAR NK-Cell Therapy Platform - BioSpace

First Patients Treated with FT516, an Off-the-Shelf NK Cell Cancer Immunotherapy for AML and for B-cell Lymphoma in Combination with Rituximab

Received FDA Clearance of IND Application for FT596, an Off-the-Shelf, Multi-Antigen Targeted CAR NK Cell Product Candidate

Opened State-of-the-art cGMP Facility Dedicated to Manufacturing iPSC-derived Cell Therapies

$303 Million in Cash & Short-term Investments as of September 30, 2019 following Completion of $173 Million Common Stock Offering

SAN DIEGO, Nov. 05, 2019 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, today reported business highlights and financial results for the third quarter ended September 30, 2019.

We achieved several significant clinical milestones over the past three months including treating the first patients with FT516, the first-ever engineered iPSC-derived cellular immunotherapy, and securing FDA clearance to initiate clinical investigation of FT596, the first-ever cellular immunotherapy engineered to express three active anti-tumor modalities. We also successfully opened our new cGMP facility specifically designed to enable consistent, large-scale, and cost-effective manufacture of allogeneic NK cell and CAR-T cell products using clonal master iPSC lines as a starting cell source, said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. We look forward to the ASH annual meeting in December, where we have had six abstracts accepted and will be sharing our first-in-human insights into the clinical safety and tolerability of FT500, the first-ever iPSC-derived cell therapy to be administered off-the-shelf in multiple doses over multiple cycles. With the completion of our recent common stock offering in September, we are well-positioned to generate clinical data across our iPSC-derived, cell-based cancer immunotherapy pipeline in 2020.

Clinical Programs

Corporate Highlights

Third Quarter 2019 Financial Results

Today's Conference Call and WebcastThe Company will conduct a conference call today, Tuesday, November 5, 2019 at 5:00 p.m. ET to review financial and operating results for the quarter ended September 30, 2019. In order to participate in the conference call, please dial 877-303-6235 (domestic) or 631-291-4837 (international) and refer to conference ID 4748666. The live webcast can be accessed under "Events & Presentations" in the Investors & Media section of the Company's website at http://www.fatetherapeutics.com. The archived webcast will be available on the Company's website beginning approximately two hours after the event.

About Fate Therapeutics iPSC Product PlatformThe Companys proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with cycles of other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Companys first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Companys platform is uniquely capable of overcoming numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics iPSC product platform is supported by an intellectual property portfolio of over 250 issued patents and 150 pending patent applications.

About FT500FT500 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line. The product candidate is being investigated in an open-label, multi-dose Phase 1 clinical trial for the treatment of advanced solid tumors (clinicaltrials.gov ID number NCT03841110). The study is designed to assess the safety and activity of three once-weekly doses of FT500 as a monotherapy and in combination with one of three FDA-approved immune checkpoint inhibitor (ICI) therapies nivolumab, pembrolizumab or atezolizumab in patients that have failed prior ICI therapy. Despite the clinical benefit conferred by approved ICI therapy against a variety of tumor types, these therapies are not curative and, in most cases, patients either fail to respond or progress on these agents. One common mechanism of resistance to ICI therapy is associated with loss-of-function mutations in genes critical for antigen presentation. A potential strategy to overcome resistance is through the administration of allogeneic NK cells, which have the inherent capability to recognize and directly kill tumor cells with these mutations.

About FT516FT516 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered to express a novel high-affinity 158V, non-cleavable CD16 Fc receptor, which has been modified to prevent its down-regulation and enhance its binding to tumor-targeting antibodies. The product candidate is being investigated in an open-label, multi-dose Phase 1 clinical trial as a monotherapy for the treatment of acute myeloid leukemia and in combination with CD20-directed monoclonal antibodies for the treatment of advanced B-cell lymphoma (clinicaltrials.gov ID number NCT04023071). CD16 mediates antibody-dependent cellular cytotoxicity (ADCC), a potent anti-tumor mechanism by which NK cells recognize, bind and kill antibody-coated cancer cells. CD16 occurs in two variants, either with high (158V) or low (158F) affinity for the Fc domain of IgG1 antibodies. Numerous clinical studies with FDA-approved tumor-targeting antibodies, including rituximab, trastuzumab and cetuximab, have demonstrated that patients homozygous for the 158V variant, which is present in only about 15% of patients, have improved clinical outcomes. In addition, ADCC is dependent on NK cells maintaining active levels of CD16 expression, and the expression of CD16 on NK cells has been shown to undergo considerable down-regulation in cancer patients, which can significantly inhibit anti-tumor activity.

About FT596FT596 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered with three anti-tumor functional modalities: a proprietary chimeric antigen receptor (CAR) optimized for NK cell biology, which contains a NKG2D transmembrane domain, a 2B4 co-stimulatory domain and a CD3-zeta signaling domain, that targets B-cell antigen CD19; a novel high-affinity 158V, non-cleavable CD16 Fc receptor that has been modified to augment antibody-dependent cellular cytotoxicity by preventing CD16 down-regulation and enhancing CD16 binding to tumor-targeting antibodies; and an IL-15 receptor fusion (IL-15RF) that promotes enhanced NK cell activity. The FDA has allowed investigation of FT596 in an open-label Phase 1 clinical trial as a monotherapy, in combination with rituximab for the treatment of advanced B-cell lymphoma, and in combination with obinutuzumab for the treatment of chronic lymphocytic leukemia. In preclinical studies of FT596, the Company has demonstrated that dual activation of the CAR19 and CD16 receptors, in combination with IL-15RF signaling, convey synergistic anti-tumor activity. Increased degranulation and cytokine release were observed upon dual receptor activation in lymphoma cancer cells as compared to activation of each receptor alone, indicating that multi-antigen engagement may elicit a deeper and more durable response. Additionally, in a mixed cellular composition cytotoxicity assay comprised of CD19+ and CD19- tumor cells, FT596 combined with CD20-directed monoclonal antibody therapy effectively eliminated the heterogeneous population of tumor cells, a result that was not observed with single-antigen targeted CAR19 T cells.

About Fate Therapeutics, Inc.Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to the development of first-in-class cellular immunotherapies for cancer and immune disorders. The Company has established a leadership position in the clinical development and manufacture of universal, off-the-shelf cell products using its proprietary induced pluripotent stem cell (iPSC) product platform. The Companys immuno-oncology product candidates include natural killer (NK) cell and T-cell cancer immunotherapies, which are designed to synergize with well-established cancer therapies, including immune checkpoint inhibitors and monoclonal antibodies, and to target tumor-associated antigens with chimeric antigen receptors (CARs). The Companys immuno-regulatory product candidates include ProTmune, a pharmacologically modulated, donor cell graft that is currently being evaluated in a Phase 2 clinical trial for the prevention of graft-versus-host disease, and a myeloid-derived suppressor cell immunotherapy for promoting immune tolerance in patients with immune disorders. Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit http://www.fatetherapeutics.com.

Forward-Looking StatementsThis release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the Companys results of operations, financial condition and sufficiency of its cash and cash equivalents to fund its operations, as well as statements regarding the advancement of and plans related to its product candidates, clinical studies and preclinical research and development programs, the Companys progress, plans and timelines for the manufacture and clinical investigation of its product candidates, the timing for the Companys receipt of data from its clinical trials and preclinical studies, the Companys development and regulatory strategy, and the therapeutic and market potential of the Companys product candidates. These and any other forward-looking statements in this release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that results observed in prior studies of the Companys product candidates, including preclinical studies and clinical trials, will not be observed in ongoing or future studies involving these product candidates, the risk of a delay or difficulties in the manufacturing of the Companys product candidates or in the initiation of, or enrollment of subjects in, any clinical studies, the risk that the Company may cease or delay preclinical or clinical development of any of its product candidates for a variety of reasons (including requirements that may be imposed by regulatory authorities on the initiation or conduct of clinical trials or to support regulatory approval, difficulties or delays in subject enrollment in current and planned clinical trials, difficulties in manufacturing or supplying the Companys product candidates for clinical testing, and any adverse events or other negative results that may be observed during preclinical or clinical development), and the risk that the Companys expenditures may exceed current expectations for a variety of reasons. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Companys actual results to differ from those contained in the forward-looking statements, see the risks and uncertainties detailed in the Companys periodic filings with the Securities and Exchange Commission, including but not limited to the Companys most recently filed periodic report, and from time to time in the Companys press releases and other investor communications.Fate Therapeutics is providing the information in this release as of this date and does not undertake any obligation to update any forward-looking statements contained in this release as a result of new information, future events or otherwise.

Availability of Other Information about Fate Therapeutics, Inc.Investors and others should note that the Company routinely communicates with investors and the public using its website (www.fatetherapeutics.com) and its investor relations website (ir.fatetherapeutics.com) including, without limitation, through the posting of investor presentations, SEC filings, press releases, public conference calls and webcasts on these websites. The information posted on these websites could be deemed to be material information. As a result, investors, the media, and others interested in Fate Therapeutics are encouraged to review this information on a regular basis. The contents of the Companys website, or any other website that may be accessed from the Companys website, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended.

Condensed Consolidated Statements of Operations and Comprehensive Loss(in thousands, except share and per share data)(unaudited)

Condensed Consolidated Balance Sheets(in thousands)(unaudited)

Contact:Christina TartagliaStern Investor Relations, Inc.212.362.1200christina@sternir.com

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Fate Therapeutics Reports Third Quarter 2019 Financial Results and Highlights Operational Progress - GlobeNewswire

Spero Therapeutics Inc. (NASDAQ:SPRO) and Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI), both competing one another are Biotechnology companies. We will compare their profitability, analyst recommendations, risk, dividends, earnings and valuation, institutional ownership.

Valuation & Earnings

Table 1 showcases the gross revenue, earnings per share and valuation of Spero Therapeutics Inc. and Brainstorm Cell Therapeutics Inc.

Profitability

Table 2 has Spero Therapeutics Inc. and Brainstorm Cell Therapeutics Inc.s return on assets, return on equity and net margins.

Liquidity

The Current Ratio and Quick Ratio of Spero Therapeutics Inc. are 17.5 and 17.5 respectively. Its competitor Brainstorm Cell Therapeutics Inc.s Current Ratio is 1 and its Quick Ratio is 1. Spero Therapeutics Inc. can pay off short and long-term obligations better than Brainstorm Cell Therapeutics Inc.

Analyst Recommendations

Spero Therapeutics Inc. and Brainstorm Cell Therapeutics Inc. Ratings and Recommendations are available on the next table.

Spero Therapeutics Inc.s average target price is $28, while its potential upside is 166.67%. Brainstorm Cell Therapeutics Inc. on the other hand boasts of a $9 average target price and a 134.99% potential upside. The results from earlier shows that analysts opinion suggest that Spero Therapeutics Inc. seems more appealing than Brainstorm Cell Therapeutics Inc.

Insider & Institutional Ownership

Spero Therapeutics Inc. and Brainstorm Cell Therapeutics Inc. has shares owned by institutional investors as follows: 53.1% and 11.4%. Insiders owned roughly 23.71% of Spero Therapeutics Inc.s shares. Insiders Competitively, owned 0.6% of Brainstorm Cell Therapeutics Inc. shares.

Performance

Here are the Weekly, Monthly, Quarterly, Half Yearly, Yearly and YTD Performance of both pretenders.

For the past year Spero Therapeutics Inc. was more bullish than Brainstorm Cell Therapeutics Inc.

Summary

Spero Therapeutics Inc. beats Brainstorm Cell Therapeutics Inc. on 6 of the 11 factors.

Spero Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on identifying, developing, and commercializing novel treatments for multi-drug resistant (MDR) bacterial infections in the United States. It is developing SPR994, an oral carbapenem-class antibiotic for use in adults to treat MDR gram-negative infections; SPR741 that has completed Phase I clinical trial to treat MDR gram-negative infections in the hospital setting; SPR206, an agent that is in preclinical development stage to disrupt the outer membrane of gram-negative bacteria; and SPR720, an oral antibiotic that is in preclinical development stage for the treatment of pulmonary non-tuberculous mycobacterial infections. The company was founded in 2013 and is headquartered in Cambridge, Massachusetts.

Brainstorm Cell Therapeutics Inc., a biotechnology company, develops adult stem cell therapies for neurodegenerative disorders that include amyotrophic lateral sclerosis, multiple sclerosis, Parkinsons disease, and others. The company holds rights to develop and commercialize its NurOwn technology through a licensing agreement with Ramot of Tel Aviv University Ltd. Its NurOwn technology is based on a novel differentiation protocol, which induces differentiation of the bone marrow-derived mesenchymal stem cells into neuron-supporting cells and secreting cells that release various neurotrophic factors, including glial-derived neurotrophic factor, brain-derived neurotrophic factor, vascular endothelial growth factor, and hepatocyte growth factor for the growth, survival, and differentiation of developing neurons. The company was formerly known as Golden Hand Resources Inc. and changed its name to Brainstorm Cell Therapeutics Inc. in November 2004 to reflect its new line of business in the development of novel cell therapies for neurodegenerative diseases. Brainstorm Cell Therapeutics Inc. was founded in 2000 and is headquartered in Hackensack, New Jersey.

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Contrasting of Spero Therapeutics Inc. (SPRO) and Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI) - FinanceMercury

After years of trying, researchers finally were able to transform human stem cells into mature cells that produce insulin. In the long term, this can cure people with diabetes. It can change the lives of millions of people worldwide with type 1 diabetes. For the first time, according to a study published in the journal Nature Cell Biology, American researchers have successfully transformed human stem cells into mature cells that produce insulin in the laboratory.

Diabetes Test

This success is the result of many years of work. The cells produced were trapped in an immature stage where they could not respond adequately to blood glucose and secrete insulin according to Matthias Hevrock of the San Francisco Diabetes Center, the author of the study. Then he and his team realized that the key to success is the neglected aspect of beta cell development, the physical process by which cells are separated from the rest of the pancreas and form the so-called Islets of Langerhans.

Then the researchers reproduced this process in the laboratory, artificially separating the stem cells from the pancreas and transforming them into groups of islets. As a result, beta cells and other cells delta and alpha cells began to react to glucose as mature insulin-producing cells.

The researchers then transplanted these islets into healthy mice and found that they worked for several days, producing insulin that reacts to blood sugar levels just like the natural islets in life animals.

According to Matthias Hevrock now they can produce insulin cells that look and act like the pancreatic cells that we have in our body. This is a big step towards our goal of creating cells that can be transplanted into diabetic patients.

Type 1 diabetes accounts for 10% of all diabetes cases worldwide. It is an autoimmune disease that occurs most often in childhood and destroys the insulin-producing cells of the pancreas. Without the insulin that regulates blood glucose levels, sugar peaks can cause severe organ damage and even death. Although the disease can be treated with regular insulin injections, some people still suffer from acute or chronic complications due to poor blood glucose control. These include retinopathy, neuropathy, heart disease, lower limb arteriopathy and renal failure. These complications can be fatal. Therefore, diabetics who are at risk of death may now receive pancreatic transplants which in most cases will be in addition to kidney transplants.

https://pubs.acs.org/doi/10.1021/acssynbio.9b00262

https://www.cdc.gov/diabetes/basics/type1.html

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Diabetes 1 Breakthrough: Researchers Are Now Able to Create Insulin-Producing Cells - Gilmore Health News

While the spotlight has lately been on dengue, polio and other virus-borne diseases, the most widespread illness in childrenfive times more prevalent than asthmacontinues to affect many kids. Im talking about early tooth decay.

Cavities? No big deal, kids get them, their baby teeth will fall out anyway, right?

No, its a disease. Cavities are triggered by bacteria that use sugar to make acids that damage teeth. It erodes the structure of teeth by draining off the calcium. Even without causing pain, decaying baby teeth could mean rotten permanent teeth.

When permanent teeth come into an environment where the baby teeth have already decayed, the same bacteria will attack the new teeth, said Burton Edelstein, DDS, MPH, professor of dentistry and health policy at Columbia University.

There are more cases of early tooth decay partly because kids are eating more processed food like pretzels, crackers, sweets, juice and soda than before. Gummy candy/vitamins are also to blame.

Kids need their parents to brush and floss their teeth for them until theyre 6 because they cant do it well yet. They tend to brush the same teeth in the front over and over again, but dont get to the back teeth or the inside surfaces, said Paul Casamassimo, DDS, chief of dentistry at Nationwide Childrens Hospital in Columbus, Ohio.

Mary Lynn R. Abellera, DMD, pediatric dentist at Dental Nook Santana Grove Sucat, is more conservative: Usually at 8-9 years old they have already developed the dexterity to brush their own teeth. Thats right along the time when theyre adept enough to tie their own shoes.

I let my feisty 4-year-old daughter brush her teeth by herself, but I follow up with an inspection, going over it again. We then add a tongue scraping to get rid of bad breath-causing gunk.

Its a great time to also introduce flossing, as its the only way to reach the cavity-causing plaque between teeth and below the gums.

Loose tooth

What to do when a child starts having a loose tooth? Parents should condition the child that they will lose a tooth because theres a new one coming out. Encourage the child to use the tooth in biting and brush it properly so it will wiggle more, said Abellera.

Tempting as it may be, dont yank it out. Said Grace Yum, DDS, a pediatric dentist at Childrens Memorial Hospital Chicago: Its better to let it come off on its own. Removing it early can be painful and may cause gum infection. Most kids are fascinated with wiggling a loose tooth and thats okay, but be certain her hands are clean.

What about when a child complains of pain during tooth development? Generally, an erupting tooth does not cause pain, but if it does, see a dentist. In the meantime, its safe to give paracetamol, advised Abellera.

With new permanent teeth in place, how else can we take better care of them? Ask your dentist to apply dental sealants, plastic coatings that can lower the chances of decay by about 89 percent.

Molar erosion

My 8-year-old son grinds his teeth in his sleep. This has led to the erosion of his molars, which makes biting painful. Since my son is too young to be fitted for a mouth guard, his bite is raised by applying a composite (pasta or filling) on the applicable surfaces every few months, depending on the severity of the erosion.

Night grinding or bruxism is an unconscious habit. It is a sleep-related movement disorder. There is still no concrete cause but its usually a combination of psychological, genetic and physical factors, explained Abellera. Sometimes thats how the child releases stress. Some grinders dont need treatment, but they can wear night guards to protect their teeth.

For parents who can afford it, cord blood banking is considered for its stem cell possibilities.

What about tooth banking?

Said Abellera: Tooth banking is saving or storing baby teeth, wisdom teeth or healthy extracted teeth for regenerative medicine. The dentist will extract the tooth at a certain time to preserve the vitality of the pulp, then it will be stored properly and be collected by the tooth bank. Like insurance, theres an annual cost for storage.

Teeth are an excellent source of stem cells, the dental pulp, especially if its a young permanent tooth. Since regenerative therapies are on the rise, tooth banking is gaining popularity. However, its still not prominent in the Philippines. Usually its used only for autogenous grafting for dental implants, not for regenerative therapies.

Looks like the tooth fairy is still in business. CONTRIBUTED

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Why you need to brush and floss your kid's teeth until he's 6 - INQUIRER.net

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Global Stem Cell Assay Market is anticipated to grow at a 19.9% CAGR from 2019 to 2030 - Health News Office

By: Jill Fox

After desk jobs continuously left him with back and neck issues, Dr. Jonathan Fields, an acupuncture physician and doctor of oriental medicine in Coral Springs, changed careers 15 years ago.

Fields spent too much time sitting at the computer, working on digital media, web design, and social media marketing, leading him to the field.

Now, in practice at Integrative Medicine US, his goal is to help others suffering from health issues like he experienced, achieve a new way of life through Chinese medicine.

I knew I needed to make a lifestyle change, said Fields, who has made it a family affair. His wife, Lauren, is an acupuncturist, and they are expecting their first child in December.

At Integrative Medicine US, the staff considers everything about an individuals physical, mental, and emotional health when making a holistic plan. Now, they are holding a free community-wide event to educate the public on alternative ways to get active and be healthy.

The Second Annual Integrative Health Expo, open to all ages, and residents in Tamarac. It will take place at the Coral Springs Gymnasium on Saturday, November 16, and Sunday, November 17, from 9 a.m. to 5 p.m. and it is FREE to the public.

The mission of the expo is to use an integrative health approach as a method of engaging the community and promoting a combination of preventative, holistic, and conventional medicine as a way to achieve optimal health.

People have their medications and their doctors, but when thats not working, we want to give them options to try different things, said Fields.

Once held in Coconut Creek, the event was changed to Coral Springs to make it entirely free for everyone.

At the expo, various fitness classes will be taking place for people to try and to get exposed to a variety of things that could be beneficial.

The Health Expo in Coral Springs will include free health screenings, blood pressure check-ups, and vendor booths with local health providers of all different specialties. Participants will be able to learn about and try acupuncture, massage, chiropractic reflexology, cupping, herbal teas, qi gong, tai chi, yoga classes, boot camp fitness, nutritional counseling, CBD, and healthy food and drinks.

The goal is to promote general health, wellness, and preventative medicine through lifestyle changes, including fitness, diet, nutrition, and social habits.

Not everybody wants to do yoga or CrossFit, but maybe taking a walk every day is good for them, said Fields.

Attendees can also observe the Legacy Kung Fu Tournament, an international kung fu exhibition featuring hundreds of competitors from all over the country.

Fields said a big part of Chinese medicine comes from kung fu. One of his kung fu teachers, also an acupuncturist, introduced him to Chinese medicine, which is thought to be holistic mind, body, and spirit, with the first part being diet and exercise.

There is no separation between the Chinese martial arts and Chinese medicine, said Fields. It was something he wanted to incorporate, and he wanted it to provide some entertainment for the people coming to the expo.

In addition to kung fu, attendees can learn about tai chi and qi gong, gentle movements that anyone at any age can do with benefits they wont get from other exercises. And, they can be done at home, with no need to join a gym or buy a special outfit.

Fields said this is what has kept some civilizations living long, healthy ages with a good quality of life, without medicines or being overweight.

By making the expo free and open to everyone, he hopes people will come out and look to see if there is something that possibly could benefit them.

There will also be onsite entertainment for children and families, like lion dance, drumming, calligraphy, art, games and stationary surfboards.

We see anxiety in younger kids, with some being put on medications. But if theyre taught how to breathe, meditate, and do some of these mindfulness practices from a young age, I think well have a little less of that, said Fields.

What hes most looking forward to is being able to educate people on what Integrative Medicine US does and why its is so beneficial.

The best part for me is to be able to help people who are having trouble and not getting the results they need with traditional, conventional medicine, he said.

The Coral Springs Gymnasium is located at 2501 Coral Springs Drive.

For more information and to view the schedule of events, go to integrativemedicine.us/health-expo. For more information about the kung fu tournament, visit lostlegacysystems.com/kung-fu-tournament.

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Free Integrative Health Expo Focuses on Holistic Wellness - Tamarac Talk

MILWAUKEE In 2019, alternative methods of self help and integrative medicine are becoming more and more popular. CBD products are found in large department stores. Yoga, while it has been prevalent in American culture for a long time, is still growing in popularity. Meditation is common, but there is one type of meditation that is gaining steam in Milwaukee and the United States. It's called Gong Bath Meditation.

"There's a lot of sound and it sounds really good," Andrew Royal, a psychotherapist and host of gong baths, said in simple terms.

It's a form of sound or music therapy.

The participant lays down on a yoga mat. They can use a pillow and/or blanket for added comfort. The host of the meditation will hit a gong, and the swelling sound and reverberations are meant to relax you.

"We dim the lights and then start to play and then let the sounds carry us all to whoever it needs to," Royal said.

These aren't small echoes either. The gong creates a thick and full sound that reverberates throughout the room and you can feel it in your chest.

Plus, there is no sheet music or specific song. It is all improvised, his partner and fellow practitioner Adriana Royal, said.

"It's usually just receiving the sound and responding to it," Adriana Royal, an acupuncturist, said.

Both can play at once or they can switch off playing the two gongs they own. They have different sized and textured mallets which they use to generate various sounds and frequencies. Some mallets are large, hollow, and soft. Others are small, dense, and hard.

"Size and also the density of it will create loud or softer sounds," Adriana said.

The setting of a gong bath is designed to create maximum comfort to allow the participant to loose themselves in the sound.

"We try to not describe at all what's going to happen, but we ask people to be receptive as they can be to whatever it is that's going to happen," Andrew Royal said.

Both Royals noted that gong baths can have various effects on a person. While the goal is to put you in an almost sedated-trance-like-state, that doesn't mean your mind will go blank.

"(Two people) laying right next to each other on the floor, and one person says something like it was so loud I could barely stand it and then the person next to them says I thought it was really quiet," Andrew Royal said.

The first descriptions of gongs dates back to 6th century China; however, it is believed that gongs were used for thousands of years before that. In the 1790's, gongs were used in western European orchestra performances. It's unclear when they became a specific meditative practice or brought to the U.S., but gongs have always been used for ceremonial, musical, and religious purposes.

"There (are) some people who know this is not like a secret anymore," Andrew Royal said.

Now, gong baths are gaining popularity in the U.S. and Milwaukee. There are some of the places that offer gong bath meditations in southeast Wisconsin including:

Gong baths can also be done during commemorative times. The Royals have hosted meditations at baptisms, birthdays, and memorial services.

"Literally we are holding space for people to meditate together," Adriana Royal said.

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Gong Bath Meditation is becoming more popular in Wisconsin - WTMJ-TV

One of the most popular supplements on the market is fish oil. Celebrities and wellness gurus alike take it to make their hair thicker, their skin more supple, and their nails stronger. Doctors recommend it for its anti-inflammatory benefits: It can actually help reduce the risk of heart disease, cancer, dementia, symptoms of rheumatoid arthritis, and age-related macular degeneration, among other diseases, says Sydney Axelrod, a dietitian at the Mount Sinai Health System. "For people who are at high risk of developing cardiovascular disease, taking fish oil supplements for at least six months has been shown to reduce risk of heart-related events, such as a heart attacks, and even death," she says.

Omega-3 fatty acids are the star nutrients in fish oil that give them these healthful, anti-inflammatory benefits. In a nutshell, omega-3s are a group of polyunsaturated fats that the body doesn't make on its own, so it's essential to get them from food. There are three well-known types of omega-3s: alpha-linolenic acid (ALA), eicosapentaenoic acid (EPA), and docosahexaenoic acid (DHA). ALA can be found in nuts and plants, but EPA and DHA can only be sourced from fish. All three, however, are needed for healthy mental and physical functionand fish oil supplements can help increase DHA and EPA levels. However, these supplementsas with everythingare neither a magic pill nor a quick fix. Here, everything you need to know about fish oil supplements.

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"As with many supplements, there is a difference between getting the nutrient from its source and getting it extracted into a supplement," says Dr. Richard Firshein, founder of the Firshein Center for Integrative Medicine. "My advice is always to get nutrients from foods first. With fish, twice per week would be ideal. Salmon and sardines are the best source of omega-3s, without the risk of excessive mercury exposure," he says.

Choose your supplement wisely, since fish oils can be contaminated with mercury or heavy metals. An easy way to protect yourself? Use one that's pharmaceutical grade. Look for a "third-party tested" stamp on them, too (a popular third-party tester is GOED, the Global Organization for EPA and DHA Omega-3); it's essentially a stamp of approval for purity. "It shows the supplements are probably safe and actually contain what they say they do," Alexrod says. Ensure that your supplement contains about 1,000 milligrams of fish oil, with 180 milligrams of EPA and 120 milligrams of DHA, she explains, adding that Nutrigold Triple Strength Fish Oil Omega-3 Gold ($22.38, nutrigold.com)covers all of those bases.

Finally, Axelrod says to consider fish oil that is certified by the Marine Stewardship Council, the Environmental Defense Fund, or a similar organization, as "small fish with short lifespans tend to be more sustainable," she says. And don't forget to check the expiration date. "Omega-3s are prone to going rancid," she says. "Once they go bad, they will have a foul smell, and become less potent or even harmful." Make sure the supplement contains an antioxidant, like vitamin E, which will keep it fresh and help prevent oxidation, she adds.

"Omega-3s are essential nutrients for both mom and baby," says Perri Halperin, clinical nutrition coordinator at Mount Sinai. "Research has shown that including EPA and DHA in the diet during pregnancy has a positive effect on the cognitive development of the baby and reduces the risk of pre-term labor."

The anti-inflammatory properties in fish oil can protect hair follicles and reduce dryness and irritation, Dr. Firshein says. "Some studies have shown a positive relationship between omega-3 fatty acids containing supplements and hair health," Halperin adds. "However, these studies are limited and more research is needed to examine the effect of fish oil on hair growth."

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While Axelrod says the anti-inflammatory factor in fish oil may reduce acne in some people, in others, they may actually cause blemishes, Dr. Firshein warns. "Fish oils may interact with hormones, and this may create an imbalance that promotes acne in people who are predisposed," he says. "Other less understood factors may include reactions to bacteria in the gut, and excess oil or sebum production, which stimulate bacterial growth on the skin."

"Taking too much fish oil can lead to vitamin toxicities and an overdose of omega-3 fatty acids," Halperin says. "The FDA recommends no more than three grams per day of EPA and DHA combined, including up to two grams per day from dietary supplements." Side effects from over-supplementation can include bleeding problems, compromised immune function, nausea, vomiting, and diarrhea. (Plus, fish oils may thin blood, Dr. Firshein says, so if you have a history of bleeding disorders or stroke, be sure to discuss taking supplements with your doctor; take the same approach if you have a diagnosed fish allergy.)

"This is a relatively new concern as we continue to pollute the earth with plastics and marine life consumes them," Dr. Firshein says. "At this point, I wouldn't recommend people just taking a fish oil pill without understanding why they are taking them. But with current purification processes, we can be relatively assured there is a low risk of plastic exposure. We currently lack tests to determine the smallest molecules, however fish liver would most likely be contaminated the most."

As for mercury? Halperin says that one independent study regarding fish oil supplements found that all of the products tested contained only very low levels of mercury, ranging from one six parts per billion per serving, which is well below 100 parts per billion, the upper safety limit set by the Global Organization for EPA and DHA Omega-3s. Even if fish oil supplements don't do everything doctors hoped, "they're still an important supplement to consider for anyone concerned about their health," Dr. Firshein says.

Excerpt from:
Here's Everything You Need to Know About Fish Oil Supplements - Yahoo Lifestyle