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Contrasting of Brainstorm Cell Therapeutics Inc. (BCLI) and Magenta Therapeutics Inc. (NASDAQ:MGTA) – MS Wkly

As Biotechnology companies, Brainstorm Cell Therapeutics Inc. (NASDAQ:BCLI) and Magenta Therapeutics Inc. (NASDAQ:MGTA) are our subject to contrast. And more specifically their risk, analyst recommendations, profitability, dividends, institutional ownership, earnings and valuation.

Valuation and Earnings

Table 1 highlights Brainstorm Cell Therapeutics Inc. and Magenta Therapeutics Inc.s gross revenue, earnings per share (EPS) and valuation.

Profitability

Table 2 shows us Brainstorm Cell Therapeutics Inc. and Magenta Therapeutics Inc.s net margins, return on equity and return on assets.

Liquidity

The Current Ratio of Brainstorm Cell Therapeutics Inc. is 1 while its Quick Ratio stands at 1. The Current Ratio of rival Magenta Therapeutics Inc. is 17.1 and its Quick Ratio is has 17.1. Magenta Therapeutics Inc. is better equipped to clear short and long-term obligations than Brainstorm Cell Therapeutics Inc.

Analyst Recommendations

In next table is given Brainstorm Cell Therapeutics Inc. and Magenta Therapeutics Inc.s ratings and recommendations.

Brainstorm Cell Therapeutics Inc. has a 126.70% upside potential and an average price target of $9.

Institutional & Insider Ownership

Institutional investors owned 11.4% of Brainstorm Cell Therapeutics Inc. shares and 85.4% of Magenta Therapeutics Inc. shares. 0.6% are Brainstorm Cell Therapeutics Inc.s share owned by insiders. Comparatively, 2.2% are Magenta Therapeutics Inc.s share owned by insiders.

Performance

In this table we show the Weekly, Monthly, Quarterly, Half Yearly, Yearly and YTD Performance of both pretenders.

For the past year Brainstorm Cell Therapeutics Inc.s stock price has smaller growth than Magenta Therapeutics Inc.

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.

Magenta Therapeutics, Inc., a clinical-stage biopharmaceutical company, engages in developing medicines to bring the curative power of bone marrow transplant to patients. It is developing C100, C200, and C300 targeted antibody-drug conjugates for transplant conditioning; MGTA-145, a stem cell mobilization product candidate to control stem cell mobilization; MGTA-456, an allogeneic stem cell therapy to control stem cell growth; E478, a small molecule aryl hydrocarbon receptor antagonist for the expansion of gene-modified stem cells; and G100, an ADC program to prevent acute graft and host diseases. The company was formerly known as HSCTCo Therapeutics, Inc. and changed its name to Magenta Therapeutics, Inc. in February 2016. Magenta Therapeutics, Inc. was incorporated in 2015 and is based in Cambridge, Massachusetts.

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Contrasting of Brainstorm Cell Therapeutics Inc. (BCLI) and Magenta Therapeutics Inc. (NASDAQ:MGTA) - MS Wkly

PRP and IRAP: Where nature meets science in horse injury treatment – Horsetalk.co.nz – Horsetalk

Platelet Rich Plasma is injected into an injured area to encourage a morerobust healing response.Palm Beach Equine Clinic

As sport horses become faster and stronger, veterinary medicine is often challenged to break barriers to provide the best in diagnostic and maintenance care.

Two resources that have become increasingly popular to treat equine injuries are Platelet Rich Plasma (PRP) and Interleukin-1 Receptor Antagonist Protein (IRAP), which encourage regeneration of injured or degenerative tissue.

Managing joint diseases and injuries using these methods is ground-breaking, but logical at its core. They essentially use naturally occurring proteins, cells, and other natural processes originated from within the body of the horse to put the horses own biological mechanisms to work stimulating healing without the use of steroids or other drugs.

Platelets are among the very first cells to accumulate at an injured site, making them very important when simulating the repair process. Platelets contain granules filled with growth factors (the elements that aid in healing) and stimulate specified tissue to heal at an increased rate. To treat a horse with PRP, the veterinarians at PBEC are able to take a sample of the horses blood and concentrate the platelets in a high-speed centrifuge on-site. The harvest and processing procedures take approximately 30 minutes before the concentrated platelet-rich sample is injected back into the horse at the specific area of injury using sterile techniques and guided by ultrasound.

Explaining the process, Dr Weston Davis, Board-Certified Staff Surgeon at Floridas Palm Beach Equine Clinic, said that first, a large quantity of blood is harvested, anywhere from 60 to180ml.

We process that to concentrate the segment that is very rich in platelets. We get a high concentration of platelets we are hoping for five to eight times the concentration that you would get from normal blood. Then we take that platelet-rich extract and inject it back into an injured area to encourage a more robust healing response.

Whenever you have an injury, platelets are one of the first cells that get there. They will aggregate, clump, and de-granulate. They release granules that are very rich in growth factors and signal the body to start the healing process.

IRAP is used to treat equine athletes that are susceptible to musculoskeletal injuries and osteoarthritis or degenerative joint disease. Joint trauma results in the release of inflammatory mediators such as Interleukin-1 (IL-1). IRAP uses a horses own anti-inflammatory protein found within the blood to counteract the destructive effects of IL-1 to slow the process of osteoarthritis. The process works by binding to the IL-1 receptors in the joint and blocking the continuation of damage and inflammation.

Palm Beach Equine Clinic veterinarian Dr Bryan Dubynsky said veterinarians often see joint damage in sport horses because of the nature of their work. But we try to avoid over-use of steroids in joints because steroids can have long-term effects on cartilage.

This is a way we can manage joint disease and stop inflammation without having to consistently use steroids. Some of our clients will maintain their horses on IRAP alone for joint injections.

The goal to better serve sport horses that continue to improve athletically is the driving force behind the search for even more developed and precise techniques used in regenerative medicine.

I believe we are learning more about these technologies with more advanced science behind what they do and how they do it, Dubynsky said. These treatments are natural, drug-free, and competition-safe, and necessity drives the need for regenerative therapies in the sport horse world.

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PRP and IRAP: Where nature meets science in horse injury treatment - Horsetalk.co.nz - Horsetalk

Protective mediators help heal tendon cells by attacking inflammation – Health Europa

Tendon tears, both to the rotator cuff and Achilles heel, are common injuries, especially in aged individuals. Painful and disabling, they can adversely impact quality of life.

New approaches are required to help patients suffering from chronic tendon injuries. A novelstudyinThe American Journal of Pathology, identified mediators that promote resolution of inflammation as potential new therapeutics to push chronically injured tendons down an inflammation-resolving pathway.

Stephanie Dakin of Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences, Botnar Research Centre and University of Oxford, said: Our study informs new therapeutic approaches that target diseased cells and promote resolution of tendon inflammation, harnessing the bodys own natural responses for therapeutic gain.

The study demonstrates the anti-inflammatory effects of two specialised pro-resolving mediators (SPMs), lipoxin B4 (LXB4) and Resolvin E1 (RvE1), on cultured tendon cells in which induced shoulder tendon disease was present.

According to Dr Dakin and colleagues, resident (meaning part of the normal tissue) stromal cells, especially fibroblasts, play a pivotal role in inflammatory diseases of joints. After injury, fibroblasts become activated and show inflammation memory, an important event underlying the switch from acute to chronic inflammation.

These cells become unable to return to their normal state. The SPMs identified by the researchers interfere with this chronic inflammatory process and help fibroblasts resolve tendon inflammation; hence the name pro-resolving mediators.

Commenting on the study, Undurti N Das of UND Life Sciences, Battle Ground and BioScience Research Centre and Department of Medicine, GVP Medical College and Hospital, Visakhapatnam, India, emphasised that understanding the crosstalk among resident stromal cells including fibroblasts that not only participate in inflammatory diseases of the joint, but also in the switch from acute to chronic inflammation, tissue resident and infiltrating macrophages, infiltrating immune cells, and endothelial cells is important to the disease process and for the development of newer therapeutic interventions.

Dr Das said: In this context, the report by Dakin et al is of substantial interest to the field. It establishes that tendon stromal cells isolated from patients with tendon tears show pro-inflammatory phenotype and secrete significantly higher amounts of interleukin (IL)-6 with dysregulated production and action of lipoxin A4, resolvins, protectins, and maresins compared to normal cells.

Dr Dakin said: There is a clear unmet clinical need to develop effective new therapeutic approaches to treat tendon disease.

SPMs, including LXB4 and RvE1, may target diseased cells and potentiate resolution of chronic tendon inflammation.

Shoulder pain is the third most common cause of musculoskeletal pain, and tears affecting shoulder rotator cuff tendons comprise a large proportion of this disease burden. Current treatments for tendon injuries include physical therapy, non-steroidal anti-inflammatory drugs, platelet rich plasma, steroid injections, and surgery to repair torn tendons.

These therapies are frequently ineffective, steroids are potentially harmful, and tendon tear surgery is associated with high postoperative failure rates. Therefore, alternative therapies targeting the cells driving chronic inflammation are required to help patients, and ideally avoid some of the problems associated with surgery, steroids, NSAIDS, or other interventions.

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Protective mediators help heal tendon cells by attacking inflammation - Health Europa

WindMIL Therapeutics and University of California, Irvine Announce Collaboration to Collect Bone Marrow from Patients with Gliomas to Develop Marrow…

BALTIMOREand PHILADELPHIA and IRVINE, Calif., Oct. 15, 2019 (GLOBE NEWSWIRE) -- WindMIL Therapeutics and the University of California, Irvine (UCI) today announced that the first patients have been identified in an investigator-sponsored study for the collection of bone marrow from patients with gliomas. The study will evaluate generating marrow infiltrating lymphocytes (MILs) for these patients through WindMILs proprietary cellular activation and expansion process. The study is being conducted at UCI.

Patients suffering with glioblastoma are in great need of new, promising treatments that might advance the current standard of care, said Daniela A. Bota, MD, PhD, director of theUCI Health Comprehensive Brain Tumor Program,seniorassociate dean for clinical research, UCI School of Medicine and clinical director, UCI Sue & Bill Gross Stem Cell ResearchCenter. The University of California, Irvine is excited toplay a key role in research that may lead to a clinical trial that enlists the immune system in novel ways to fight this terrible disease.

Gliomas are the most common of the malignant brain tumors. Glioblastoma, the most common glioma, has a five-year survival of less than 5 percent. Additional treatment options are urgently needed for these patients. Adoptive immunotherapy is a possible approach for gliomas and the use of MILs, a cell therapy that is naturally tumor-specific, is one such treatment option.

The bone marrow is a unique niche in the immune system to which antigen-experienced memory T cells traffic and are then maintained. WindMIL has developed a proprietary process to select, activate and expand these memory T cells into MILs. Because memory T cells in bone marrow occur as a result of the immune systems recognition of tumor antigens, MILs are specifically suited for adoptive cellular immunotherapy and are able to directly eradicate or facilitate eradication of each patients unique cancer. WindMIL is currently studying MILs in multiple myeloma, non-small cell lung cancer and squamous cell carcinoma of the head and neck, and plans to expand into other solid tumors.

WindMIL is looking forward to working with the University of California, Irvine on this exciting project and is optimistic that MILs may offer the potential to help patients with these hard-to-treat diseases, said Monil Shah, PharmD, MBA, Chief Development Officer at WindMIL.

About WindMIL Therapeutics

WindMIL Therapeutics is a clinical-stage company developing a novel class of autologous cell therapies based on marrow infiltrating lymphocytes (MILs) for cancer immunotherapy. As the leader in cellular therapeutics emanating from bone marrow, WindMIL translates novel insights in bone marrow immunology into potentially life-saving cancer immunotherapeutics for patients. WindMIL believes that Cell Source Matters and the companys proprietary process to extract, activate and expand these cells offers unique immunotherapeutic advantages, including inherent poly-antigen specificity, high cytotoxic potential and long persistence. For more information, please visit: http://www.windmiltx.com.

About UCI Health

UCI Healthcomprises the clinical enterprise of the University of California, Irvine. Patients can access UCI Health at primary and specialty care offices across Orange County and at its main campus, UCI Medical Center in Orange, California. The 417-bed acute care hospital provides tertiary and quaternary care, ambulatory and specialty medical clinics and behavioral health and rehabilitation services. UCI Medical Center features Orange Countys only National Cancer Institute-designated comprehensive cancer center, high-risk perinatal/neonatal program and American College of Surgeons-verified Level I adult and Level II pediatric trauma center and regional burn center. UCI Health serves a region of nearly 4 million people in Orange County, western Riverside County and southeast Los Angeles County. Follow us onFacebookandTwitter.

About the University of California, Irvine

Founded in 1965, UCI is the youngest member of the prestigious Association of American Universities. The campus has produced three Nobel laureates and is known for its academic achievement, premier research, innovation and anteater mascot. Led by Chancellor Howard Gillman, UCI has more than 36,000 students and offers 222 degree programs. Its located in one of the worlds safest and most economically vibrant communities and is Orange Countys second-largest employer, contributing $5 billion annually to the local economy. For more on UCI, visitwww.uci.edu.

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WindMIL Therapeutics and University of California, Irvine Announce Collaboration to Collect Bone Marrow from Patients with Gliomas to Develop Marrow...

Benefit of CD19-Targeted CAR-T Therapy in Patients With Transformed Waldenstrm Macroglobulinemia – Cancer Therapy Advisor

A case study published in Leukemia and Lymphoma described a patient with a diagnosis of Waldenstrm Macroglobulinemia (WM) that had subsequently undergone histological transformation to refractory high grade B-cell lymphoma and was successfully treated with CD19-targeted chimeric antigen receptor (CAR)-T cell therapy.1

WMis a type of B-cell non-Hodgkin lymphoma(NHL), typically characterized by overproduction of monoclonal immunoglobulinM, as well as infiltration of malignant lymphoplasmacytic cells into the bonemarrow.

Although considered incurable, WM often follows an indolent course andsome patients can be asymptomatic for long periods. Rarely, the diseasetransforms into a more aggressive form of NHL that has been associated with apoor prognosis.

The patient described in the case studywas a 71-year-old man who was first diagnosed with WM in 1998. The patient wasmonitored without undergoing active treatment for a period of 12 years, atwhich time he developed anemia and splenomegaly. At that time, he underwenttreatment with 6 cycles of fludarabine and rituximab and achieved a partial response totreatment. Following a worsening of symptoms 4 years later, the patient wastreated with 6 cycles of bendamustine and rituximab.

Biopsyof an enlarged cervical lymph node performed at that time revealed high-gradeB-cell lymphoma that was clonally related to the previously seenlymphoplasmacytic infiltrate, consistent with transformation.

Thepatient subsequently achieved a complete response to 6 cycles of rituximab,cyclophosphamide, doxorubicin, vincristine, prednisone (R-CHOP) plus ibrutinibfollowed by 6 months of ibrutinib maintenance therapy that lasted for 18months.

Salvagetherapy included 2 cycles of rituximab, dexamethasone, cytarabine, cisplatin(R-DHAP) followed by 1 cycle of rituximab plus high-dose cytarabine, followedby autologous stem cell transplantation several months later.

Asthe patients disease was considered to be chemorefractory based on subsequent imagingand pathological analyses, he was treated with axicabtagene ciloleucel, aCD19-targeted CAR-T cell therapy currently approved for the treatment of adultpatients with relapsed or refractory large B-cell lymphoma, including high gradeB-cell lymphoma, after 2 or more lines of systemic therapy.2

Althoughthe patient experienced pancytopenia, grade 1 cytokine release syndrome, andgrade 1 neurotoxicity following CAR-T therapy, he achieved a complete response1 month following treatment.

Notably,there was no evidence of either underlying WM or transformed disease at 6 and 12months follow-up.

Thestudy authors noted that longer term follow up in this patient will beinformative, as late relapses have occurred even in patients who achieve a deepresponse after transplant. CAR-T cell therapy may be an effective treatment forrelapsed or refractory WM that has not yet undergone histologicaltransformation, as CD19 is almost universally expressed on lymphoplasmacyticlymphoma cells. The researchers concluded that further analysis of this iswarranted in the context of clinical trials.

References

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Benefit of CD19-Targeted CAR-T Therapy in Patients With Transformed Waldenstrm Macroglobulinemia - Cancer Therapy Advisor

Equine Medical Research Funding Receives USEA Board of Governors – United States Eventing Association

Oct 15, 2019

The Equine Medical Research Fund was created in 2014 to provide consistent funding for equine medical research, which has historically been underfunded. A $1.00 starter fee was added to each USEA recognized event entry beginning in 2014, and at years end a majority of those dollars were distributed through the Morris Animal Foundation, which assists the USEA with selecting studies to fund, monitoring the funds, and reporting on progress. Morris has decades of experience in reviewing grant applications from universities and research institutions and has a team of veterinarians who spend thousands of volunteer hours assessing which studies are most likely to impact horse welfare. Ultimately, a committee from the USEA comprised of riders and veterinarians will determine which studies the USEA dollars will target.

This years committee recommendation is a bit different as the committee is recommending spreading funds among three entities - the Stephen Teichman Farrier study, a Grayson Jockey Club Research Foundation study, and two Morris Animal Foundation Studies.

Through these partnerships, the USEA is helping to address the major problem of equine research underfunding while maintaining control over how member dollars are distributed. Although the committee is always interested in recommending studies that concern general horse welfare, this year we took a closer look at recommending studies that were geared to solving issues that concern performance horses. The goal of our selection philosophy is to provide USEA positive PR through funding a variety of institutions and studies that show our support for horse care, welfare, and rehabilitation.

In 2017, $46,900 dollars were distributed between five different studies on different topics pertaining to equine health, from improving regenerative therapies to studying gaps in the equine genome related to tendon health.

In 2018, $44,858 dollars were distributed between four different studies on a variety of topics including insulin's relation to laminitis, stem cell therapies, equine asthma, and joint infections.

At the USEA Board of Governors meeting in August, the Equine Medical Research Committee recommended the funding of four studies using the $39,581 collected in 2018 and the Board approved their recommendation. Below are additional details about the four selected studies.

Non-Invasive Evaluation of Host-Microbiota Interactions Canaan Whitfield-Cargile, Texas A&M

Summary: This study aims to develop a non-invasive platform to serve as a diagnostic test for gastrointestinal inflammation prior to severe disease and to reveal how bacteria in the gut influence horse health.

Description: One of the most important and common classes of diseases affecting horses are gastrointestinal (GI) diseases including colic and colitis. Despite tremendous research efforts to both diagnose and prevent these diseases, they remain very common and frequently are not diagnosed until severe disease is present. One area that is gaining recognition as an important player in equine GI health and disease is GI bacteria or the microbiota. The microbiota consists of all of the bacteria and other microorganisms in the GI tract. Alterations in the amounts and types organisms that make up the microbiota have been linked to both GI and non-GI diseases of horses including colic, colitis, laminitis, and obesity. These findings are part of the reason why there are a vast array of prebiotics, probiotics, and other products on the market that claim to influence the GI microbiota. Unfortunately, there is minimal evidence to support the efficacy of these products and a lack of understanding of how these products work. A major contributor to the lack of evidence and understanding of these products is related to an inability to monitor the health and function of the equine GI tract. It is clear when severe disease is present but currently impossible to monitor subtle changes. Similarly, while we can easily diagnose severe inflammation and microbiota changes during disease, we cannot capture the changes that occurred prior to clinical disease. These limitations result in capturing results of GI injury and disease but not causes. Consequently, there is great need for improved understanding of the causes of disease and early identification of horses at-risk of developing GI disease. In the absence of such knowledge, effective intervention strategies to reduce GI and GI-related health risks will remain elusive.

Horses and other animals naturally shed cells lining the GI tract on a daily basis. People, in fact, shed up to 1/3 of the cells lining their GI tract daily. We have developed a technique for examining the gene expression profile of these cells to monitor the health and function of the GI tract from fecal samples. We have shown that this approach offers a promising means to non-invasively examine the response of the GI intestinal tract to injury. Further, we have convincing preliminary data demonstrating that the gene expression profile arising from these cells mirrors the gene expression profile of the tissues within the GI tract of the horse and provides a global view of the health and function of the GI tract. This approach offers a highly attractive, non-invasive means of monitoring GI responsiveness to disease and interventions. Thus, our long-term goals are to 1) develop inexpensive, non-invasive diagnostic tests in order to document GI inflammation prior to the onset of severe disease in horses; and, 2) by examining host and microbiota data simultaneously we aim to elucidate the mechanisms of host-microbiota interactions in the context of health and disease. Ultimately, we aim to use this approach to identify specific pathway by which the host and microbiota interact so that this critically important interaction can be targeted therapeutically.

In order to achieve our goals, we will utilize a reversible model of GI inflammation with which we have extensive experience. We will induce GI inflammation with the non-steroidal anti-inflammatory drug (NSAID) phenylbutazone and noninvasively interrogate the response and response of the microbiota. Importantly, we will collect samples sequentially both before and after induction of inflammation in order to gain predictive and diagnostic information about GI inflammation in horses. This approach will have broad application to many equine intestinal disorders and will provide the much needed mechanistic insight into disease development and progression, thereby enabling us to develop effective prevention and treatment strategies for equine GI disease.

The total cost of this study is $46,756 in 2019 and $45,814 in 2020. The EMRC recommends $12,000 for funding this study.

Paraoxonase-1 Activity as a Marker for Diagnosis of Equine Metabolic Syndrome Gabriele Rossi, DVM, Ph.D., DECVCP Murdoch University

Summary: Researchers will investigate a new laboratory test to improve diagnosis of equine metabolic syndrome, a metabolic and hormonal disorder in horses.

Description: Equine metabolic syndrome (EMS) is characterized by abnormal insulin regulation, obesity, and susceptibility to laminitis. EMS is challenging to diagnose since laboratory tests fail to distinguish EMS from other common equine diseases. Human metabolic syndrome (HMS) shares similar laboratory features to EMS and a new serum biomarker has been proposed in human medicine to improve the diagnosis of HMS. Researchers will investigate if this new HMS test also can provide an accurate diagnosis for horses with EMS. A new efficient and cost-effective EMS test will help veterinarians better diagnose and manage this complex disease in horses.

The total cost of this study is $9,355. The EMRC recommends $9,355 to fund this study.

Cell-mediated immunogenicity and MHC expression regulation of TGF-beta2-treated mesenchymal stem cells for improved clinical use in the horse. Lauren V Schnabel, DVM, Ph.D., DACVS, DACVSMR North Carolina State University

Summary: Researchers will manipulate the expression of immune markers on stem cells to develop safer and more effective therapies for horses with musculoskeletal injuries.

Description: Stem cell therapies have the potential to improve the outcome of potentially severe and life-ending musculoskeletal injuries in horses. However, sometimes the recipients immune system will destroy transplanted stem cells if the immune system perceives the introduced cells as foreign. To improve stem cell treatment success, researchers will examine if a novel cell culture treatment can be used to prevent the immune system from destroying donor stem cells. This study will help researchers better understand the cellular regulation of the molecules that initiate immune responses and advance the development of convenient and effective off-the-shelf stem cell therapy.

The total cost of this study is $131,479. The EMRC recommends $12,000 to fund this study. This is the second year that we are recommending funding this 3 year study.

Stephen Teichman Study

The goal is to determine the effects of front shoe surface modifications in the sport horse on the duration of breakover. We discussed and agreed to support this study during the May BOG conference call providing the publishing and professional fees were researched and found to be reasonable, which they were.

The total cost of this study is $9,640. The EMRC recommends USEA to provide $4,820 and an additional $4,820 is to be contributed by other donors.

This brings the total recommended research total to $38,175, leaving $1,406 of use at another time.

For an update on studies previously selected for funding by the USEA Equine Medical Research Committee, click here. To learn more about how the Equine Medical Research Fund got started, click here. For additional information about the Fund, click here.

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Equine Medical Research Funding Receives USEA Board of Governors - United States Eventing Association

Rocket Pharmaceuticals Announces Upcoming Presentations at the European Society of Gene and Cell Therapy Annual Congress – Business Wire

NEW YORK--(BUSINESS WIRE)--Rocket Pharmaceuticals, Inc. (Nasdaq: RCKT) (Rocket), a leading U.S.-based multi-platform gene therapy company, today announces data presentations at the upcoming European Society of Cell and Gene Therapy (ESGCT) 27th Annual Congress taking place October 2225, 2019, in Barcelona, Spain. Presentations at this years meeting include four oral presentations and one poster presentation related to Rockets lentiviral pipeline programs for Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD), Pyruvate Kinase Deficiency (PKD), and Infantile Malignant Osteopetrosis (IMO).

Details for Rockets oral and poster presentations are as follows:Title: Towards Haematopoietic Stem Cell-Targeted Gene Therapy of Infantile Malignant OsteopetrosisSession Title: Skeletal Muscle & Bone Gene TherapySession Date: Wednesday, October 23, 2019Session Time: 5:30 PM 7:30 PM CESTRoom: 116-117

Title: Gene Therapy for Patients with Fanconi AnaemiaSession Title: Gene Therapy Clinical Trials IISession Date: Thursday, October 24, 2019Session Time: 8:30 AM - 10:30 AM CESTRoom: 113-117

Title: First Steps of a Lentiviral Gene Therapy Clinical Trial for Pyruvate Kinase DeficiencySession Title: Blood DiseasesSession Date: Thursday, October 24, 2019Session Time: 2:45 PM - 4:45 PM CESTRoom: 211

Title: Broad Applicability of NHEJ-Mediated Gene Editing to Correct Mutations in a Variety of Fanconi Anaemia GenesSession Title: New Approaches in Gene EditingSession Date: Friday, October 25, 2019Session Time: 9:00 AM - 11:00 AM CESTRoom: 113-115

Title: Stable Transduction of Long-Term HSCs Under Optimized GMP-Conditions for the Gene Therapy of LAD-I PatientsSession Title: Poster Session IISession Date: Thursday, October 24, 2019Session Time: 1:15 PM - 2:45 PM CESTPoster Number: P228

Full results from the ESGCT presentations will be available online at the conclusion of the presentation: https://www.rocketpharma.com/esgct-presentations/

About Rocket Pharmaceuticals, Inc.Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (Rocket) is an emerging, clinical-stage biotechnology company focused on developing first-in-class gene therapy treatment options for rare, devastating diseases. Rockets multi-platform development approach applies the well-established lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. Rocket's first two clinical programs using LVV-based gene therapy are for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, and Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal. Rockets first clinical program using AAV-based gene therapy is for Danon disease, a devastating, pediatric heart failure condition. Rockets pre-clinical pipeline programs for bone marrow-derived disorders are for Pyruvate Kinase Deficiency (PKD) and Infantile Malignant Osteopetrosis (IMO). For more information about Rocket, please visit http://www.rocketpharma.com.

Rocket Cautionary Statement Regarding Forward-Looking StatementsVarious statements in this release concerning Rocket's future expectations, plans and prospects, including without limitation, Rocket's expectations regarding the safety, effectiveness and timing of product candidates that Rocket may develop, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Infantile Malignant Osteopetrosis (IMO) and Danon disease, and the safety, effectiveness and timing of related pre-clinical studies and clinical trials, may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995 and other federal securities laws and are subject to substantial risks, uncertainties and assumptions. You should not place reliance on these forward-looking statements, which often include words such as "believe," "expect," "anticipate," "intend," "plan," "will give," "estimate," "seek," "will," "may," "suggest" or similar terms, variations of such terms or the negative of those terms. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket's ability to successfully demonstrate the efficacy and safety of such products and pre-clinical studies and clinical trials, its gene therapy programs, the pre-clinical and clinical results for its product candidates, which may not support further development and marketing approval, the potential advantages of Rocket's product candidates, actions of regulatory agencies, which may affect the initiation, timing and progress of pre-clinical studies and clinical trials of its product candidates, Rocket's and its licensors ability to obtain, maintain and protect its and their respective intellectual property, the timing, cost or other aspects of a potential commercial launch of Rocket's product candidates, Rocket's ability to manage operating expenses, Rocket's ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, Rocket's dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, and unexpected expenditures, as well as those risks more fully discussed in the section entitled "Risk Factors" in Rocket's Annual Report on Form 10-K for the year ended December 31, 2018. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

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Rocket Pharmaceuticals Announces Upcoming Presentations at the European Society of Gene and Cell Therapy Annual Congress - Business Wire

ROOT OF SKIN Skincare Products Sell Out in Tenth Consecutive Appearance on QVC Japan – PRNewswire

IRVINE, Calif., Oct. 15, 2019 /PRNewswire/ -- AIVITA Biomedical, Inc., a biotech company specializing in innovative stem cell applications, announced today that the Company's ROOT of SKIN line of rejuvenating skincare products sold out in its tenth appearance on the Japanese home shopping channel QVC Japan.

ROOT of SKIN is AIVITA's proprietary skincare line for skin rejuvenation, made possible through the same proprietary knowledge and expertise used to develop its cutting-edge stem cell therapies. SourceCode Technology, the proprietary actives complex in ROOT of SKIN, contains the complete set of factors and supporting biological components present in young, healthy skin.

AIVITA Biomedical uses 100% of the proceeds from ROOT of SKIN sales to support the treatment of women with ovarian cancer.

"Outstanding market traction, a product that truly works, and a benevolent use of proceeds," said Hans Keirstead, AIVITA's Chief Executive Officer. "This sets the stage for global expansion of our ROOT of SKIN product line."

About ROOT OF SKIN

ROOT of SKIN is a rejuvenating line of skincare products fueled by an unrelenting pursuit for advancements in life-changing and life-saving treatments. Harnessing breakthroughs in stem cell therapy, AIVITA Biomedical developed a technology that does more than just boost regeneration. The patented actives complex SourceCode Technology renews, repairs and protects, just as your skin did at its youngest and healthiest stage. Rich with every biological component healthy skin needs for development, and free of any unnecessary ingredients. All proceeds support treatment of women with ovarian cancer.

About AIVITA Biomedical

AIVITA Biomedical is a privately held company engaged in the advancement of commercial and clinical-stage programs utilizing curative and regenerative medicines. Founded in 2016 by pioneers in the stem cell industry, AIVITA Biomedical utilizes its expertise in stem cell growth and directed, high-purity differentiation to enable safe, efficient and economical manufacturing systems which support its therapeutic pipeline and commercial line of skin care products.

SOURCE AIVITA Biomedical, Inc.

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ROOT OF SKIN Skincare Products Sell Out in Tenth Consecutive Appearance on QVC Japan - PRNewswire

Joy as funding rises for Moscow treatment trip – Northern Times

A trip to Russia is top of Tain woman Michele Murrays Christmas wish list for 2020.

But the mother-of-two has a more serious reason to visit the country rather than just a holiday trip.

She was diagnosed with multiple sclerosis 15 years and is now desperate to undergo groundbreaking treatment available to her in Russia but which she cannot access in the UK.

Michele (46) launched a 42,000 fundraising drive at the start of September and it is already going well so her hopes of achieving her goal next year are high.

She said: My fundraiser currently stands at 15,000 and I wanted to say a massive thank you to everyone who has donated. The amount given is really staggering.

Michele had to retire from her job as a secretary eight years ago after her condition took a turn for the worse.

At present I use a walking stick, rollator and wheelchair, she said. I have fatigue, muscle spasms, balance problems and weakness in my legs and arms. My MS is getting worse all the time and I feel that if I dont do something now, it will be too late.

It is such an invisible disease. I look fine on the outside, however on the inside my immune system is attacking my central nervous system and causing scarring on my brain/spinal column.

She only recently heard about Hematopoietic Stem Cell Transplant (HSCT), a treatment designed to stop MS in its tracks.

A high dose of chemotherapy is used to kill off my existing failing immune system. she said. My stem cells will then be re-infused to build a brand new immune system free of MS.

The treatment is available in the UK but Michelle does not fit the criteria and to access it privately is costly. However the National Pirogov Medical Centre Russiain Moscow offers HSCT for 42,000.

I hope I will be able to get this life-changing treatment so I can have a better quality of life., said Michelle.

She has been organising various fundraisers and on Saturday, December 28 , RythmnReel will play at the Duthac Centre, Tain, with all proceeds going to help send her to Russia.

To make a donation, visit Go Fund Me and search for Michele Murray.

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Joy as funding rises for Moscow treatment trip - Northern Times

Sarah Ferguson reveals highlight of my life as she celebrates 60th birthday – Express

The Duchess of York, affectionately known by British royal fans as Fergie, shared the cover of The Perfect World Magazine, where she can be seen giving Sir David Attenborough an honorary award. Fergie, who turns 60 today, revealed in an Instagram post that moment is one of the best of her life.

She wrote on Instagram: "Thank you all for the birthday messages! This is one of the highlights of my life."

In the cover, Fergie can be seen smiling as she hands the award, shaped like a rhino, toSir Attenborough, the world-famousEnglish broadcaster and natural historian.

Fergie andSir Attenborough attended together last September thePerfect World Foundation gala dinner, where she will present him the award.

The same award has been previously given toMark Shand, the late brother of the Duchess of Cornwall, and primatologist Dr Jane Goodall.

In the early afternoon, the Duchess of York published on Instagram another picture, showing her wearing a colourful sari.

She added in the caption: "Colourful moments"

Fergie's Instagram messages come after her former husband Prince Andrew celebrated her birthday on social media.

Earlier this morning, the Duke of Yorkpublished two identical messages dedicated to Fergie on both Instagram and Twitter.

READ MORE:How Fergie was 'forced to cancel birthday party due to crisis'

The messages read: "Wishing Sarah Ferguson a very Happy 60th Birthday!"

On Instagram, the message was accompanied by three pictures, one depicting Sarah surrounded by members of the York family, including Princess Eugenie's husband Jack Brooksbank and Princess Beatrice's fiancee Edoardo Mapelli Mozzi.

The second picture shows Fergie surrounded by children supported by the charity she is a patron of, Street Child UK.

And the third snap depicts Eugenie, Beatrice and Fergie together while carrying out an engagement.

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Fergie has previously spoke with enthusiasm about her approaching 60th birthday.

Speaking at a charity event held onbehalf of Street Child and hosted by Hello, the Duchess said:"I think it's really important that my life is beginning at 60, I'm so excited by it."

Outspoken Fergie also revealed she has undertaken laser treatment to her skin to look her best for her milestone birthday.

Similarly, the Duchess had laser also before the wedding of her youngest daughter Princess Eugenie, which helped to make her skin glow.

But these haven't been the only beauty treatments Fergie has undertaken in the past years.

In March this year, Fergie travelled to the Bahamas toundergo stem cell therapy - an alternative to surgery which is yet to be proven fully safe and effective on humans and is therefore not widely available in the UK.

This therapy helped her solving a painful problem related to her feet, which eventually forced her to stop wearing high heels.

I went to the Bahamas in March to have the treatment.

I think my toes were ruined by all the riding I did when I was young.

Read more here:
Sarah Ferguson reveals highlight of my life as she celebrates 60th birthday - Express