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New funding to test personalised treatment for aplastic anaemia – Pharmafield

By daniellenierenberg

Patients with ultra-rare bone marrow disease are set to benefit from 1.15m grant from LifeArc and The Aplastic Anaemia Trust. The grant will support researchers from Kings College London and Kings College Hospital to test a personalised treatment for aplastic anaemia patients who have not responded to available therapies

The grant has been awarded to investigate the potential of a novel type of personalised cellular therapy to reverse the ultra-rare condition aplastic anaemia (AA). The results of this research could give new hope to people living with a severe, life-limiting form of this condition.

The grant will fund a clinical trial to investigate the safety and efficacy of using a patients own T-reg cells to restore the blood-making function of the bone marrow. This follows laboratory-based research from the team of scientists where T-reg cells from a patients own blood were collected, selected for activity and multiplied. In a test tube, these cells prevented the immune system from attacking the patients bone marrow stem cells.

AA is an ultra-rare life-threatening illness caused by the bone marrow failing to make enough of all three types of blood cells red blood cells, white blood cells and platelets. Only around 100-150 people in UK are diagnosed per year, affecting all ages but most commonly people between the ages of 10 to 20 years old and those over the age of 60 years.

People with the illness are at greater risk of infections, bleeding, and can experience extreme fatigue, which leaves them unable to carry out simple daily tasks that most people take for granted. Around one in three patients with severe AA fail to respond to existing drug treatments and the other option a bone marrow transplant is reliant on finding a suitable donor, requires life-long treatment with immunosuppression therapy and is unsuccessful in one in three people.

The trial at Kings College London and Kings College Hospital will run for three years and aims to recruit nine patients. A blood sample of the patients T-reg cells will be extracted, purified and grown in the lab before being given back to them in a higher concentration. As patients with AA are more susceptible to infection, this personalised treatment approach is more likely to avoid the risk of severe infection and inflammation.

Professor Ghulam Mufti, Department of Haematological Medicine at Kings College London and Kings College Hospital, and lead study investigator said: For patients with this ultra-rare disease, were looking for the first time at a personalised medicine approach where their own immune cells could be used to alter their disease. In AA there is a reduction in the number of T-regs and most of the ones that the AA patients do have are non-functional. Weve seen success in the laboratory by selecting and bolstering the number of functional T-reg cells. Now, with funding from LifeArc and the AAT, we can investigate the potential of this approach in treating AA patients who currently have very limited treatment options.

Dr Catriona Crombie, LifeArcs Head of Philanthropic Fund explained why the charity had approved the funding: LifeArc set up the Philanthropic fund to support translational research into rare diseases, where there is less interest from commercial organisations. Patients with AA can have limited treatment options; this opportunity with Kings College London, Kings College Hospital and the AAT has the potential to transform the lives of patients living with a severe form of the disease.

Grazina Berry, Chief Executive of the AAT said: AA can severely impact a persons quality of life. Through AATs close work with Kings College London and Kings College Hospital as a specialist centre of clinical care and research in AA, we identified the project with the most potential to directly benefit patients who are currently at a loss for solutions. We are delighted to have partnered with LifeArc and Kings College London and Kings College Hospital to progress this ground-breaking work, which could potentially enable people living with severe AA to once again lead a normal life.

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Rocket’s gene therapy shows long-term efficacy in rare blood disorder – MedCity News

By daniellenierenberg

A gene therapy for a rare blood disorder has shown what the manufacturer calls the first evidence of long-term improvement associated with the disease.

New York-based Rocket Pharmaceuticals said Thursday that it had presented long-term follow-up data from the Phase I/II study of RP-L102, its gene therapy for Fanconi anemia, at the annual congress of the European Society of Cell and Gene Therapy in Barcelona, Spain. The company said it represented the first evidence of long-term improvement and stabilization in blood counts and durable mosaicism among patients who received the therapy without the use of the conditioning regimens normally used for allogeneic stem cell transplants, which the company calls Process A.

Shares of Rocket were up slightly on the Nasdaq following the news. RP-L102 is a lentiviral vector-based gene therapy. Most other gene therapies in development, and both of the currently marketed ones Spark Therapeutics Luxturna (voretigene neparvovec-rzyl) and Novartis Zolgensma (onasemnogene abeparvovec-xioi) are adeno-associated viral vector-based.

According to the data, representing four of nine patients, there were improved blood counts and long-term bone marrow mitomycin C (MMC) resistance, thereby indicating durable phenotypic correction. The data met or exceeded a 10 percent threshold that the company said the Food and Drug Administration and European Medicines Agency had agreed to for its upcoming Phase II registration study, for which it plans to start enrolling patients by the end of the year.

FA is a rare, genetic bone marrow failure disorder, half of whose patients are diagnosed before the age of 10, while about 10 percent of patients are diagnosed as adults, according to the National Organization for Rare Disorders. It is often associated with progressive deficiency of production of red and white blood cells and platelets in the bone marrow and can eventually lead to certain solid and liquid tumor cancers. It occurs in 1-in-136,000 births and is more common among Ashkenazi Jews, Spanish Roma and black South Africans.

These results indicate the feasibility of engraftment in FA patients using autologous, gene corrected [hematopoietic stem cells] in the absence of any conditioning regimen, said Dr. Juan Bueren, scientific director of the FA gene therapy program at Spains Center for Energy, Environmental and Technological Research, in a statement. This indicates the potential of this therapeutic approach as a definitive hematologic treatment, while avoiding the burdensome side effects associated with allogeneic transplant, including the risk of post-transplant mortality and a substantially higher risk of head and neck cancer.

Photo: virusowy, Getty Images

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Worlds first cell atlas of developing liver created by Cambridge scientists – Cambridge Independent

By daniellenierenberg

The worlds first cell atlas of the human developmental liver has been created, giving fresh insight into how the blood and immune systems develop in the foetus.

A high-resolution resource, it will aid our understanding of normal development and efforts to tackle diseases that can form during development, such as leukaemia and immune disorders.

The cell atlas maps how the cellular landscape within the developing liver changes between the first and second trimesters of pregnancy, including how stem cell from the liver seed other tissues, supporting the high demand for oxygen required for growth.

Researchers from the Wellcome Sanger Institute in Hinxton, the Wellcome MRC Cambridge Stem Cell Institute, University of Cambridge, Newcastle University and their collaborators created the atlas by using single cell technology to analyse 140,000 liver cells and 74,000 skin, kidney and yolk sac cells.

In adults, it is bone marrow that is primarily responsible for the creation of blood and immune cells in a process called haematopoiesis.

In early embryonic life, the yolk sac and liver play a key role in creating these cells, which then seed peripheral tissues such as skin, kidney and ultimately bone marrow.

But until now, the precise process of how blood and immune systems develop in humans has been unknown.

Isolating cells from the developing liver, the researchers were able to identify them by what genes they were expressing and discover what the cells looked like.

They tagged haematopoietic cells in sections of developmental liver using heavy metal markers in order to map them to their location.

Prof Muzlifah Haniffa, a senior author of the study from Newcastle University and senior clinical fellow at the Wellcome Sanger Institute, said: Until now research in this area has been a little bit like blindfolded people studying an elephant, with each describing just a small part of it.

This is the first time that anyone has described the whole picture, how the blood and immune systems develop in such detail. Its been an extraordinary, multidisciplinary effort that is now available as a tool for the whole scientific community.

The scientists learned that during foetal development, mother haematopoietic stem cells stay in the liver. But the liver alone cannot supply enough red blood cells, so the next generation daughter cells called progenitor cells travel to other tissues, maturing in places such as the skin. Thee, they develop into red blood cells to help meet the high demand for oxygen in the developing foetus.

Dr Elisa Laurenti, a senior author from the Wellcome MRC Cambridge Stem Cell Institute and the Department of Haematology at the University of Cambridge, said: We knew that as adults age our immune system changes. This study shows how the livers ability to make blood and immune cells changes in a very short space of time, even between seven and 17 weeks post-conception.

If we can understand what makes the stem cells in the liver so good at making red blood cells, it will have important implications for regenerative medicine.

The study, published in Nature, also involved the mapping of genes involved in immune deficiencies to reveal which cells were expressing them.

It is known that gene mutations can lead to immune disorders such as leukaemia.

A better understanding of the development of healthy liver functions could aid our understanding of how to treat such conditions.

The work is part of the ambitious effort to create the first complete Human Cell Atlas.

Dr Katrina Gold, genetics and molecular sciences portfolio manager at Wellcome, said: Our immune system is vital in helping to protect us from disease, yet we know very little about how immune cells develop and behave in the early embryo. This study is hugely important, laying a critical foundation for future research that could help improve our understanding of disorders linked to the early immune system, such as childhood leukaemias.

The Human Cell Atlas has the potential to transform our understanding of health and disease and were excited to see these first discoveries from our Wellcome-funded multidisciplinary team of scientists.

Dr Sarah Teichmann, a senior author from the Wellcome Sanger Institute, University of Cambridge and co-chair of the Human Cell Atlas organising committee, said: The first comprehensive cellular map of the developmental liver is another milestone for the Human

Cell Atlas initiative.

The data is now freely available for anyone to use and will be a great resource to better understand healthy cellular development and disease-causing genetic mutations.

Read more

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AstraZeneca and Cancer Research UK launch joint Functional Genomics Centre in Cambridge

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United Therapeutics Receives Permit For Cell Therapy Facility Build-Out At Mayo – Pharmaceutical Online

By daniellenierenberg

The build-out is estimated at $9.5M.

United Therapeutics received a building permit Tuesday for a $9.5M build-out of its cell therapy facility on the second floor of Mayo Clinics Discovery and Innovation Building.

The 21,843-square-foot space will house an automated stem cell manufacturing site, which is one of the first of its kind in the country. The Whiting-Turner Contracting Co. is the project contractor.

The technology, approved by the FDA in 2018, allows the Mayo Clinic Center for Regenerative Medicine to produce cells from the bone marrow of a stem cell donor in large enough quantities to be used as treatments in clinical trials. It allows for the treatment of multiple patients at the same time.

Construction began in 2017 on the $32.4M building at 14221 Kendall Hench Drive. It held a grand opening in August.

The first floor houses three ex-vivo lung perfusion surgical suites used for lung restoration, another form of regenerative medicine. It turns donor lungs, which previously would have previously been unusable, into viable transplant organs. United Therapeutics also collaborates with Mayo Clinic on lung restoration.

The third floor houses the Life Sciences Incubator for biotech entrepreneurs, which offers coworking space, wet labs, business resources, networking and entrepreneurial training.

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BrainStorm Cell Therapeutics to Present at the Dawson James Securities 5th Annual Small Cap Growth Conference – BioSpace

By daniellenierenberg

NEW YORK, Oct. 25, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the development of innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, today announced that it will be presenting at the Dawson James Securities 5th Annual Small Cap Growth Conference, being held on October 28-29, 2019 at the Wyndham Grand Hotel in Jupiter, Florida.

Preetam Shah, PhD, MBA, Chief Financial Officer is scheduled to present on Tuesday, October 29th at 3:40 p.m. Eastern Time, in Track 2 - Preserve Ballroom B, with one-on-one meetings to be held throughout the conference.

Chaim Lebovits, President and CEO of BrainStorm said, We are pleased to have the opportunity to have Dr. Shah present at the Dawson James Small Cap Growth Conference. Dr. Shah, joined BrainStorm in September 2019, and we look forward to having him present the Companys growth strategy and future to a wide audience of accreditied investors.

About NurOwnNurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

About BrainStorm Cell Therapeutics Inc.BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn Cellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm received U.S. FDA clearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) in December 2018 and has been enrolling clinical trial participants since March 2019. For more information, visit the company's website.

Safe-Harbor Statements Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Corporate:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone: 646-666-3188uri@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PRPhone: +1.646.677.1839sean.leous@icrinc.com

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Imago BioSciences Preliminary Data from Ongoing Phase 2 Study of IMG-7289 for the Treatment of Myelofibrosis to be Presented at the 61st American…

By daniellenierenberg

SAN FRANCISCO--(BUSINESS WIRE)--Imago BioSciences, Inc., a clinical-stage biotechnology company focused on the treatment of myeloproliferative neoplasms (MPN) and related bone marrow diseases, announced today that preliminary data from its ongoing Phase 2 study of IMG-7289 (bomedemstat) in patients with myelofibrosis (MF) has been selected for an oral presentation at the American Society of Hematology (ASH) Annual Meeting, on December 9 in Orlando, Florida. The abstract will be published November 6, and the presentation will include results updated from those in the abstract.

Kristen Pettit, M.D., assistant professor at the University of Michigan and investigator in the study at the Rogel Cancer Center in Ann Arbor, will present both preliminary results from Phase 2a, as well as initial data from patients from the Phase 2b expansion. The objectives of the study are to evaluate the safety and efficacy of IMG-7289 (bomedemstat) in up to 75 patients at sites in Australia, the US, UK and Europe. In this study, bomedemstat is administered orally once-daily as monotherapy in adult patients with intermediate-2 or high-risk MF resistant to or intolerant of ruxolitinib.

The FDA recently approved a second JAK2 inhibitor but the majority of patients with myelofibrosis will eventually lose the benefit of those treatments, said Dr. Pettit. Patients have an urgent need for new treatments that manage their symptoms. We continue to be encouraged by the bomedemstat data we see in this clinical investigation.

Imago Presentation

Title: A Phase 2 Study of the LSD1 Inhibitor IMG-7289 (bomedemstat) for the Treatment of Myelofibrosis. Session: 634. Myeloproliferative Syndromes: Clinical: Emerging and Novel Targeted TherapiesSession Date: Monday, December 9, 2019Session Time: 7:00 AM - 8:30 AM ESTPresentation Time: 7:45 AM ESTRoom: Orange County Convention Center, W304EFGH

About IMG-7289

IMG-7289 (bomedemstat) is a small molecule discovered by Imago BioSciences that inhibits lysine-specific demethylase 1 (LSD1 or KDM1A). LSD1 is an enzyme regulating both cytokine expression and myeloid differentiation and sustaining self-renewal in malignant hematopoietic stem/progenitor cells. In non-clinical studies, bomedemstat demonstrated robust in vivo efficacy as a single agent and in combination with other therapeutic agents across a range of myeloid malignancy models, including the myeloproliferative neoplasms encompassing myelofibrosis, essential thrombocythemia and polycythemia vera. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to bomedemstat for the treatment of myelofibrosis. An international Phase 2b study of bomedemstat for the treatment of myelofibrosis remains ongoing (Clinicaltrials.gov NCT03136185). Additional clinical studies in hematologic disorders will begin in 2020.

About Imago BioSciences

Imago BioSciences is a clinical-stage, venture-backed pharmaceutical company whose investors include a fund managed by Blackstone Life Sciences, Frazier Healthcare Partners, Omega Funds, Amgen Ventures, MRL Ventures Fund, HighLight Capital, Pharmaron, Greenspring Associates and Xeraya Capital as well as other corporate and venture investors. Imago is focused on improving the management of malignant and life-threatening diseases of the bone marrow with a focus on the myeloproliferative neoplastic disorders including myelofibrosis, essential thrombocythemia and polycythemia vera. The company is based in California.

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What is aplastic anemia? Symptoms, causes, and treatment – Medical News Today

By daniellenierenberg

Aplastic anemia is a medical condition that damages stem cells in a person's bone marrow. These cells are responsible for making red blood cells, white blood cells, and platelets, which are vital to human health.

Doctors believe various conditions can cause aplastic anemia, while the disease itself ranges in severity from mild to life threatening.

Medical advancements mean that aplastic anemia is more treatable than ever. In this article, learn more about this rare medical disorder.

When a person has aplastic anemia, their bone marrow does not create the blood cells it needs. This causes them to feel ill and increases their risk of getting infections.

Doctors also call aplastic anemia bone marrow failure.

Doctors do not know exactly how many people in the United States have aplastic anemia.

According to the National Organization for Rare Disorders (NORD), doctors diagnose approximately 500 to 1,000 cases every year. It is most common in older children, teenagers, and young adults.

Researchers believe that most cases of aplastic anemia are due to the immune system attacking healthy bone marrow cells, according to NORD.

Doctors have also identified some of the possible causes of this immune system response, including:

However, doctors usually cannot pinpoint the underlying cause in most aplastic anemia cases.

When the cause is unknown, doctors refer to the condition as idiopathic aplastic anemia.

Symptoms of aplastic anemia include:

These symptoms may be severe. Some people may have heart-related symptoms, such as chest pain.

A doctor will start by asking about a person's symptoms and their medical history.

They will usually use a blood test known as a complete blood count (CBC) to evaluate a person's red blood cells, white blood cells, and platelets. If all three of these components are low, a person has pancytopenia.

A doctor may also recommend taking a sample of bone marrow, which comes from a person's pelvis or hip.

A laboratory technician will examine the bone marrow. If a person has aplastic anemia, the bone marrow will not have typical stem cells.

Aplastic anemia can also have similar symptoms as other medical conditions, such as myelodysplastic syndrome and paroxysmal nocturnal hemoglobinuria. A doctor will want to rule out these conditions.

Sometimes, a person with other medical conditions can develop aplastic anemia. These conditions include:

If a person has these conditions, a doctor will recognize that they are more likely to get aplastic anemia.

Doctors usually have two goals when treating aplastic anemia. The first is to reduce the person's symptoms, and the second is to stimulate the bone marrow to create new blood cells.

People with aplastic anemia can receive blood and platelet transfusions to correct low blood counts.

A doctor may also prescribe antibiotics as a person needs white blood cells to fight infections. Ideally, these drugs will prevent infections until a person can build more new white blood cells.

Doctors usually recommend a bone marrow transplant to stimulate new cell growth in the long term.

For this, a doctor may first prescribe chemotherapy medications to kill off abnormal bone marrow cells that are affecting a person's overall bone marrow function.

Next, a doctor performs a bone marrow transplant by injecting the bone marrow into a patient's body.

Ideally, the individual will receive bone marrow from a close family member. However, even a sibling donor is only a match in 2030% of cases.

People can also receive bone marrow from someone who is not related to them if doctors can find a compatible donor.

Some people cannot tolerate bone marrow transplants, especially older adults, and those having difficulty recovering from chemotherapy. Others may not be able to find a donor that matches their bone marrow. In these instances, a doctor can prescribe immunosuppressive therapy.

Immunosuppressive medicines suppress the immune system, which ideally stops it from attacking healthy bone marrow cells. Examples of these medications include antithymocyte globulin (ATG) and cyclosporine.

According to NORD, an estimated one-third of people with aplastic anemia do not respond to immunosuppressive drugs.

If this is the case, doctors may consider other treatments, such as hematopoietic stem cell transplantation and a medication called eltrombopag (Promacta).

Those with aplastic anemia may face complications due to their disease as well as their treatment.

Sometimes, a person's body rejects a bone marrow transplant. Doctors call this graft-versus-host disease or GVHD.

GVHD can make a person feel extremely ill and can cause symptoms that include:

According to 2015 research, about 15% of aplastic anemia patients who receive immunosuppressive therapy will develop myelodysplastic syndromes or acute myeloid leukemia.

These conditions can develop years after a person's initial diagnosis.

Some people do not respond to aplastic anemia treatments. When this is the case, they are more vulnerable to infections that can be life threatening.

The outlook for a person with aplastic anemia depends on many factors, including:

A doctor will discuss a person's treatment outlook when considering the various therapies.

Aplastic anemia damages stem cells in a person's bone marrow. The bone marrow makes red blood cells, white blood cells, and platelets, which are all essential for the body.

A person with aplastic anemia may experience severe anemia symptoms. Treatment may include chemotherapy, stem cell transplants, and immunotherapy.

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A Discussion With Jennifer Delgado on Life After Cancer and Weathering the Storm – Thrive Global

By daniellenierenberg

JenniferDelgado grew up in St. Louis, Missouri. She attended Webster University, whereshe received her Bachelor of Arts in Media Communications. She then went to MississippiState University, where she received a Bachelor of Science in Geosciences witha concentration in Broadcast Meteorology.

In 2006,Jennifer Delgado worked as a morning and noon meteorologist for WTVR-TV inRichmond, Virginia. Then in 2008, she began working at CNN International inAtlanta, Georgia, as their primary meteorologist, as well as a fill-inmeteorologist on all CNN networks. In 2010, she won a Peabody Award for CNNscoverage on the Deepwater Horizon oil spill in the Gulf of Mexico.

In 2013,Delgado was hired as a co-host of AMHQ (Americas Morning Headquarters) at TheWeather Channel. She anchored continuous coverage of breaking news and weatherevents, including live interviews with state and local officials, experts andresidents. She was also their fill-in co-host of Wake-Up with Al.

JenniferDelgado began freelancing as a meteorologist/anchor for WXIA-TV in 2017. Shepresented weathercasts every six minutes during a two-hour morning newscast andproduced weathercasts for radio, web, and the 24-hour weather channel.

Two yearsago, Jennifer Delgado was diagnosed with blood cancer. She underwent treatmentand received a bone marrow/stem cell transplant. Since the transplant, she hasbeen receiving treatment at the Emory Winship Cancer Institute and advocatingfor cancer awareness and more bone marrow donors.

No one is ever prepared tohear the words, you have cancer. It literally blew up my world. I had to stopworking because beating cancer became my full-time job. I knew something waswrong for months based on my symptoms. I was tiredall the time, my bones were aching, had migraines, vertigo andconfusion. Dealing with any illness is stressful, especially if you arent ableto work. Some people say cancer changed their life for the better; however, Idont want to credit cancer for anything positive. It was a wake-up call. Lifeis short, and you have to enjoy every moment.

I immediately went into adeep depression. I hid and only shared the news with my close friends andfamily. I was trying to hide the awful chemo port in my chest and made excuses for my appearanceand fatigue. It was very stressful. I think anyone dealing with a seriousmedical condition should reach out to people going through the same battle. I got some amazing tips from fellow blood cancersurvivors on Instagram and Facebook support groups. I have formed many closebonds and when I am feeling down they completely understand. Cancer patients caneasily go through their savings in a short amount of time. I was lucky to haveamazing health insurance but not everyone is that fortunate. There is a lot of grant money out there forpeople struggling financially. The Leukemia & Lymphoma Society is anamazing organization and helps patients with everything from financial help,information on clinical trials etc.

If you are strong enough, Isay its important to be your own health advocate. You know your body best. Ialso suggest if you have one, reaching out to a friend or family member whoworks in medicine (nurse, PA, doctor) to be your medical advocate. The advocatecan come to your appointments or even join a conference call during yourappointments when you need help understanding your treatment options. I waslucky to have both my mom and one of my best friends to help me interpreteverything. Never be afraid to ask your doctor questions, and dont forgetabout the physicians assistant, who often has more availability.

I was going back and forthto the doctor for nearly a year, and they keep dismissing my symptoms. At onepoint, one doctor told me to take probiotics. I finally decided it was time toget a second opinion when I was having trouble walking. Luckily, I found Dr.Drew Freilich, whom I credit with saving my life. He recognized that mysymptoms were severe and insisted that I needed an MRI. Thats how theydiscovered I had a blood cancer that was attacking my bones. I could havebecome disabled if I had waited longer to get help. If you know something iswrong, you have to be persistent about getting answers.

I know it sounds clich, butmy friends, family, and neighbors. They all took excellent care of me. Theydrove me to the hospital for chemotherapy or bone marrow biopsies. My friends were great and woulddrop by to bring me food or help clean up myhouse.

I know it may sound sillybut my dogs really helped keep my spirits up. Quite often, it was just me and the dogs and duringisolation. I truly believe that pets are healing, and studies show that havingone improves your mental health. There were several weeks when I had to be awayfrom my dogs because my immune system was too weak. I was lucky enough to havegreat friends watch my fur babies. I even tried to convince my friends to driveby Emory Hospital so that I could see them.

I would say you have to bepositive. It seems like its a long way away, and you wonder at times whetheror not everything you did is going to pay off when you finally get toremission. So, I think you have to be positive because you get very paranoid. Ibelieve positive thinking can be healing and improve your health. Keeping inmind that everyones journey is different, I think its also important to see apsychologist or therapist. Sometimes its easier to share your real concernswith a stranger. We always try and put on a brave face for family and friends.

Aftereverything, I felt like I had to give back to the cancer community and EmoryWinship Cancer Center. I got my dogs certified to be Happy Tails therapydogs, and now we visit patients battling cancer while they are getting chemo.Its amazing and emotional all at the same time. Many times, patients will say,your puppy made my day.

Iam also trying to raise awareness for the need of more bone marrow donors.Right now, the majority of donors come from Europe. It would be awesome if morepeople would register to be a bone marrow donor. Its a simple swab test. Ithink its a small price to pay, considering more than 170,000 people arediagnosed with blood cancer every year. Check out Be The Match or The Leukemia& Lymphoma Society.

I am not going to sugarcoatit, staying motivated is extremely challenging and a daily battle. I thinkevery cancer survivor questions, why did this happen to me? Is it gone? How longwill I stay in remission? It can be quite depressing, but you have to live forthe day and stick to a routine. I try to remind myself that there is a reasonwhy I am still alive, and I want to give back to others who are struggling.

Everything. I had months ofchemo to get my cancer level down enough to collect my stem cells for thetransplant. I wondered constantly, will I be in remission? And then once Iwas in remission, how long will I stay in remission before I relapse? Whenyoure dealing with blood cancers, most have no cure. So, theres always thatchance of relapse, and youre always worrying about it.

I did six rounds of chemobefore I was even ready to get a transplant. The stem cell transplant wassomething I was dreading because of the high dose of chemotherapy and losing myhair. That can be a very difficult experience, especially for women. After thosesix rounds, they collected my stem cells, which is not a fun process. Then theyprepped me, and I had the transplant.

After, I was in isolation atthe hospital for three weeks. Then I went home, and I was still under isolationfor another 100+ days. I felt like I was ready to lose my mind. During thistime, your white blood cells are regenerating, which means you dont have animmune system, and you suffer from extreme fatigue and pain. Walking up a shortflight of stairs would wipe me out. I couldnt eat salads, fruits, basicallyanything raw. When I left the house, Id have to wear a mask to protect myimmune system. I really hated that because everyone would stare and pretty muchknew I had cancer.

However, to put a positivespin on it, because of my time in isolation at home, I really felt my creativejuices start to flow. I began brainstorming and thinking of a lot of differentthings because life is short, and the cancer was my wake-up call.

So, my best advice duringthat period is to make a reading list and binge-watch shows on Netflix. I readthe Game of Thrones series. Iliterally had a calendar counting down to 100 days. Thats also the time whenyour hair finally starts to grow back!

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A Discussion With Jennifer Delgado on Life After Cancer and Weathering the Storm - Thrive Global

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BrainStorm Cell Therapeutics’ President and CEO to be Featured as Keynote Speaker at Cell Series UK 2019 – GlobeNewswire

By daniellenierenberg

NEW YORK, Oct. 24, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the development of innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, today announced, Chaim Lebovits, President and CEO, will serve as a Keynote Speaker at Cell Series UK.Cell Series UK, will be held October 29-30, 2019, at London Novotel West, London, UK. The Conference, organized by Oxford Global, is one of the foremost events in Europe focused on regenerative medicine and cellular innovation.

Ralph Kern MD, MHSc, Chief Operating and Chief Medical Officer of Brainstorm, who will also participate at Cell Series UK stated, We are very pleased to have Chaim Lebovits presenting at this prestigious conference where global leaders in stem cell and regenerative medicine will have the opportunity to learn more about NurOwn and the critical research being conducted by the Company. Mr. Lebovits Keynote Address, Stem Cell Therapeutic Approaches For ALS, will be presented to leading members of the scientific and business community including potential partners and investors.

About NurOwnNurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

AboutBrainStorm Cell Therapeutics Inc. BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn Cellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm received U.S. FDA clearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) in December 2018 and has been enrolling clinical trial participants since March 2019. For more information, visit the company's website.

Safe-Harbor Statements Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Corporate:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone: 646-666-3188uri@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PR Phone: +1.646.677.1839sean.leous@icrinc.com

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Ewing sarcoma: Causes, symptoms, and treatment – Medical News Today

By daniellenierenberg

Ewing sarcoma is a form of bone cancer that usually affects children and adolescents.

Ewing sarcoma can be very aggressive, but the cells tend to respond well to radiation therapy. Ideally, doctors will diagnose the cancer before it has spread.

According to the National Library of Medicine, an estimated 250 children in the United States receive a diagnosis of Ewing sarcoma each year.

In this article, learn more about Ewing sarcoma, including the symptoms, causes, and treatment options.

Ewing sarcoma is a rare type of cancer that usually starts in the bone typically in the pelvis, chest wall, or legs and occurs mostly in children and teenagers.

Dr. James Ewing first described Ewing sarcoma in 1921. He identified cancer cells that looked different than the cells in osteosarcoma, another type of bone tumor.

Doctors may also refer to this cancer type as the Ewing family of tumors. These tumors have distinct cells that usually respond well to radiation treatments.

This rare cancer type accounts for just 1.5% of all childhood cancers and is the second most common bone cancer type in childhood, after osteosarcoma.

Although researchers are unsure why some people develop Ewing sarcoma, they have identified mutations in certain genes in the tumor cells that cause this cancer.

These include the EWSR1 gene on chromosome 22 and the FLI1 gene on chromosome 11.

These genetic mutations occur spontaneously during a person's lifetime. The individual does not inherit them from a family member.

There are no known risk factors for Ewing sarcoma that make one person more likely than another to develop this cancer.

Ewing sarcoma can cause the following symptoms:

An estimated 87% of Ewing sarcomas are sarcoma of the bone. The other types form in the soft tissues, such as cartilage, that surround the bones.

Ewing sarcoma can spread to other areas of the body. Doctors call this process metastasis.

Areas that the cancer can spread to include other bones, bone marrow, and the lungs.

Doctors categorize Ewing sarcoma as one of three types according to its extent:

Before diagnosing Ewing sarcoma, a doctor will take a person's full medical history and ask them what symptoms they are having, when they noticed them, and what makes them better or worse. They will also perform a thorough physical exam, focusing on the area of concern.

A doctor will usually recommend an imaging study to view the bone or bones. These tests include:

If it looks as though a tumor may be present, a doctor will perform a biopsy, which involves taking a sample of bone tissue. They will send this tissue to a laboratory, where a specialist called a pathologist will check it for the presence of cancerous cells.

A doctor may also order blood tests, a bone marrow biopsy, and other scans when necessary. These tests can help determine whether the cancer has spread to other locations.

A doctor will work with a team of cancer specialists and surgeons to recommend and implement particular treatments.

Possible treatments for Ewing sarcoma include:

Doctors may use a combination of treatments depending on how far the cancer has spread and a person's overall health.

Research into new treatments for Ewing sarcoma is ongoing. Some doctors may inform their patients about clinical trials, which help test new treatments.

Possible complications of Ewing sarcoma include:

If Ewing sarcoma has spread to other areas of the body, it can be life threatening. For this reason, it is vital for a doctor to evaluate any symptoms as quickly as possible.

According to the American Academy of Orthopaedic Surgeons, an estimated two-thirds of people in whom cancer has not spread to other areas of the body survive at least 5 years after their diagnosis.

People who are more likely to have positive outcomes include those who have:

The likelihood of successful treatment is different for every individual, so people should speak to a doctor about their or their child's expected outlook.

Ewing sarcoma is a rare type of cancer that mostly affects young people.

When doctors detect it early enough, the condition usually responds well to treatment.

Anyone who notices signs or symptoms of Ewing sarcoma, such as a bone that breaks for no apparent reason or a painful lump or swelling, should speak to a doctor.

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Dad who called on the public for stem cells for his son is up for an award – Chronicle Live

By daniellenierenberg

Doting dad Stephen Armstrong knows all too well what its like to be waiting for a transplant donor.

His son Jacob was diagnosed at two years old with a rare blood disorder and called on the public to donate stem cells to find him a match.

He then set out to raise as much money as he could for the blood cancer charity Anthony Nolan in a bid to save lives.

And now, after raising over 20,000, his efforts have been recognised by the charity as they honour him at an awards ceremony held at the Tower of London in November.

Stephen, 33, of Wallsend, North Tyneside, has been shortlisted for the Individual Fundraiser of the Year Award at the Anthony Nolan Supporter Awards 2019.

The prestigious awards are back for their seventh year and will recognise the outstanding achievements of the volunteers, fundraisers and campaigners who help the pioneering blood cancer charity save lives.

Stephens nomination is in recognition of his incredible fundraising efforts, leading a group of 19 friends and family in a series of physical challenges, all while his son was undergoing treatment.

After Jacob was diagnosed in 2017, Stephen set out to find a matching stem cell donor, as well as raise awareness of the need for more people on the register.

From here Jacobs Journey was born, and through a series of challenges including the Great North Run, the Great North Bike Ride and climbing Ben Nevis, Stephen has helped raise over 20,000 for the charity.

Jacob, who turns four in November, and his family have been told he does not need a transplant, but Stephen and his family want to continue raising awareness for others who arent so lucky.

When Jacob was diagnosed, we were stunned by how few people were on the stem cell donor register. I couldnt believe how a stranger in the street could potentially save our little boys life, said Stephen, an assistant manager for Dixons Carphone.

Anthony Nolan helped us massively while Jacob was ill and provided a great support network. I feel very proud to be nominated for an award, and I hope it can help build even more awareness for the cause.

Stephen and mum Kirsty, 28, received the news in December 2017 that Jacob was suffering from bone marrow failure, which affects between 30 and 40 children each year.

They first became concerned about his health when they went abroad to get married and noticed he was getting bruised easily. The marks would take weeks to disappear, so when the couple returned to the UK they decided to take Jacob to the doctor for a check up.

After tests he was then diagnosed and was treated at the Great North Childrens Hospital in Newcastle, where he received two blood transfusions.

Stephen added: When we were told Jacob did not need the transplant it was the best news in the world, a total relief. He still needs check ups every three months and his consultants is keeping an eye on him. There are so few people on the stem cell donor register so I just wanted to create a ripple effect with awareness and get more people on it.

Stephen, who has raised a further 8,000 for other smaller charities, has also been nominated for our Chronicle Champions Award in the Champion Fundraiser category.

Henny Braund, Chief Executive of Anthony Nolan, said: It is remarkable to see how many people support our work to find a match for those in need of a stem cell transplant. Without them, none of our lifesaving work would be possible.

Stephen has shown tremendous commitment to Anthony Nolan by continually going above and beyond in his fundraising efforts.

Henny added: We want to extend a huge congratulations to Stephen and look forward to celebrating with him at the awards.

The awards take place on Thursday 28 November at the Tower of London, and all winners will be revealed on the night.

Anthony Nolan is the charity that finds matching stem cell donors for people with blood cancer and blood disorders and gives them a second chance at life. It also carries out ground-breaking research to save more lives and provide information and support to patients after a stem cell transplant, through its clinical nurse specialists and psychologists, who help guide patients through their recovery.

To see the full shortlist, and find out more about the charity visit http://www.anthonynolan.org/awards

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Cannabis use and the immune system: white blood cell count – Health Europa

By daniellenierenberg

The study, published in the Journal of Cannabis Research, looked at a number of studies covering cannabis use and the immune system, noting that little is known on circulating white blood cell counts and cannabis use.

The researchers looked at the National Health and Nutrition Examination Survey (20052016), a survey designed to be nationally representative of United States non-institutionalised population, and found that there was a modest association between heavy cannabis use and higher white blood cell count but that neither former nor occasional cannabis use was associated with total or differential WBC counts.

White blood cells are the cells in our body that function mainly as immune cells originating in the bone marrow.

Today, it is known that cigarette smoking generates several chemicals that are implicated in oxidative stress pathways and systemic inflammation and elevated white blood cell count in tobacco cigarette smokers have been well documented, whereas tobacco abstinence is associated with sustained decrease in white blood cell count.

The study highlights how cannabis is able to mediate its effects through the cannabinoid-1 (CB1) and cannabinoid-2 (CB2) receptors.

CB2 receptors can be found in numerous parts of the body related to the immune system, including bone marrow, thymus, tonsils and spleen. CB1 receptors are present in the central nervous system, and at lower levels in the immune system.

The effects of cannabinoids on hematopoiesis, and immune cell proliferation using animal and cell based models has been widely demonstrated and a number of studies have examined the association of cannabis use and white blood cell counts in human immunodeficiency virus (HIV).

The studies have shown a higher white blood cell count in HIV positive men who used cannabis.

Last year a study discovered certain cannabinoids that enhance the immunogenicity of tumour cells, rendering them more susceptible to recognition by the immune system. This discovery is important because the leading class of new cancer fighting agents, termed checkpoint inhibitors, activates the immune system to destroy cancer cells.

Enhancing recognition of cancer cells with cannabinoids may greatly improve the efficacy of this drug class. The Pascal study was the first to identify a mechanism in which cannabinoids may provide a direct benefit in immunotherapy.

When looking at white blood cell counts the study noted that: Several of the important study limitations merit attention. The observational nature of the study constrained causal inferences. Even though NHANES collects blood and urine specimens, drug testing is not conducted, and cannabis use was self-reported which may lead to non-differential misclassification bias. There was no available information on the route of administration of cannabis (smoking, ingestion, etc.) or cannabis preparation/potency.

In addition, the study is based on fairly recent NHANES surveys (200516) which might be more representative of the increasing cannabis potency compared to NHANES III (19881994) surveys.

A number of laboratory studies have reported suppression of immune responses with cannabinoid administration, and some epidemiological studies found lower levels of inflammatory biomarkers such as fibrinogen, C-reactive protein and interleukin-6 in adult cannabis users.

The study also noted that the reported anti-inflammatory effects of cannabis were greatly attenuated when body weight is controlled for and suggests that the inverse cannabis-body weight association might explain the lower levels of circulating inflammatory biomarkers in adult cannabis users.

The study highlights that these alterations of immune responses by cannabis use might be associated with increased susceptibility to infections and hence the higher white blood cell count, however, it notes that it is possible that the elevated white blood cell count and suboptimal health status contributed to cannabis use rather than cannabis use caused suboptimal health.

The study states: This hypothesis, though, cannot be tested as NHANES does not collect information on cannabis use motives. Another potential mechanism can be through the effect of cannabinoids on stem cells. Pre-clinical studies suggest that cannabinoids stimulate hematopoiesis and hence this stimulation to bone marrow tissues can be associated with increased circulating white blood cell count in cannabis users.

Positive associations between heavy cannabis use, and total white blood cell and neutrophil counts were detected. Clinicians should consider heavy cannabis use in patients presenting with elevated white blood cell count.

Research on cannabis use and the immune system is lacking and the study suggests further research is needed to understand the immune related effects of different modes of cannabis use.

The study noted: Research on heavy cannabis use and cardiovascular health is needed as systemic inflammation, increased cardiovascular risk and increased mortality risk have been all associated with white blood cell elevation within the normal physiologic range.

Studies with repeated measures are needed to study immunomodulatory changes in cannabis users, and whether the mode of cannabis use can differentially affect immune responses.

Additional research is needed to understand the immune related effects of different modes of cannabis use and to elucidate the role of proinflammatory chemicals generated from smoking cannabis.

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BrainStorm Cell Therapeutics to Present at the Dawson James Securities 5th Annual Small Cap Growth Conference – GlobeNewswire

By daniellenierenberg

NEW YORK, Oct. 25, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the development of innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, today announced that it will be presenting at the Dawson James Securities 5th Annual Small Cap Growth Conference, being held on October 28-29, 2019 at the Wyndham Grand Hotel in Jupiter, Florida.

Preetam Shah, PhD, MBA, Chief Financial Officer is scheduled to present on Tuesday, October 29th at 3:40 p.m. Eastern Time, in Track 2 - Preserve Ballroom B, with one-on-one meetings to be held throughout the conference.

Chaim Lebovits, President and CEO of BrainStorm said, We are pleased to have the opportunity to have Dr. Shah present at the Dawson James Small Cap Growth Conference. Dr. Shah, joined BrainStorm in September 2019, and we look forward to having him present the Companys growth strategy and future to a wide audience of accreditied investors.

About NurOwn NurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

AboutBrainStorm Cell Therapeutics Inc.BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn Cellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm received U.S. FDA clearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) in December 2018 and has been enrolling clinical trial participants since March 2019. For more information, visit the company's website.

Safe-Harbor Statements Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could causeBrainStorm Cell Therapeutics Inc.'sactual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available athttp://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Corporate:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone: 646-666-3188uri@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PR Phone: +1.646.677.1839sean.leous@icrinc.com

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Baby dies from AML, the same cancer his identical twin has – TODAY

By daniellenierenberg

Nicole Duhaney couldnt believe her luck when she learned she was having identical twins.

I felt like had won the lottery, Duhaney, 21, told TODAY Parents. "It was the happiest surprise."

After being pregnant for what felt like an eternity, Duhaney and her boyfriend, Niles Liburd, finally welcomed sons Emre pronounced Em-ree" and Elijah on Dec. 23, 2018.

Our life seemed perfect, the mom from Huddersfield, England, said.

But just three weeks later, Elijah developed a lump on his cheek, and both babies developed colds they couldnt seem to kick. Suddenly, they were projectile vomiting.

Trending stories,celebrity news and all the best of TODAY.

At just 4 months old, Emre and Elijah were both diagnosed with childhood acute myeloid leukemia. The disease, also known as AML, is a type of cancer in which the bone marrow makes a large number of abnormal white blood cells, according to the National Cancer Institute.

Myeloid leukemia is the second most common pediatric blood cancer, but it's still relatively rare. In the United States there are roughly 500 children a year between the ages of 0 and 14 that are diagnosed with AML, according to Dr. Richard Aplenc, a physician-scientist within the Division of Oncology at Children's Hospital of Philadelphia.

Aplenc said it is not surprising that Emre and Elijah were diagnosed at the same time.

"If the twins are identical, then they share the same placenta and the same blood supply, so that leukemic cell goes to the other twin," Aplenc explained. "We know that if leukemia is diagnosed before a year or so, there is 100 percent chance that the other twin will develop it."

Tragically, 10-month-old Elijah passed away at home in Tuesday. Doctors allowed Emre, who is currently undergoing chemotherapy, to leave the hospital so he could say goodbye to his brother.

The love they had for each other was just unbreakable, Duhaney noted. "They didn't like to be separated."

She recalled how Elijah pulled his brother in for a kiss after a recent stem cell transplant.

Elijah was beautiful. Every person he met, he touched their heart," Duhaney said. There were times when I cried and he rubbed my tears away. I wish God took me instead of him.

As Duhaney and Liburd, 26, make funeral arrangements a GoFundMe has been set up to help the couple with expenses they are finding comfort in knowing Elijah took his final breaths at home.

He spent six months of his life in a hospital, Duhaney told TODAY Parents. His final night he was where he wanted to be, with the people who loved him him the most.

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Bloomington Vet Joins Study For Stem Cell Therapy To Treat Dogs With Arthritis – WGLT News

By daniellenierenberg

The Eastland Companion Animal Hospital in Bloomington is asking dog owners if they want to participate in research on using stem cells to treat dogs with arthritis.

Local dogs wouldjoin a double-blind, placebo-controlled studyto show the effectiveness of stem cells in treating large dogs(70 pounds or more) with arthritis in up to two joints of the knee, hip, elbow, or shoulder. The veterinary clinic has partnered with Animal Cell Therapies, who it's worked with before, to bring this study to Bloomington.

Dr. Kathy Petrucci, founder and CEO of Animal Cell Therapies, explained how dogs will receive the treatment.

The dogs that will receive the stem cells will be sedated, Petrucci said. Depending on what joints are affected, they will receive up to two injections in the joint and they will also receive an IV dose of stem cells.

The FDA oversees the cells that are received from donors for the study. Mothers donating these cells are screened for diseases, and cells are tested for any infections to ensure safety.

Stem cell therapy has been controversial, especially related to humans.

I think a lot of the controversy comes from the misunderstanding of the cell types, Petrucci said. The research in stem cells first started centered around embryonic or fetal tissue use. Its controversial to use embryos and fetal tissues for treatment for anything. The fact that we are using a disposable tissue as our cell sources makes it not controversial at all.

Why Umbilical-Derived Cells

Petrucci explained why umbilical-derived cells are more effective in treating arthritis versus other sources.

We looked at fat, bone marrow, embryonic cells, Petrucci said. The embryonic cells are a lot more unpredictable, and the bone marrow cells are more difficult to work with and less predictable. We didnt think the fat cells are as potent as umbilical-derived cells. Umbilical-derived cells are a lot younger and theyre a little bit more predictable. They are more easy to collect. We obtain cells from donors when the tissue would be normally thrown away. Theres no surgery required, no extra biopsies to obtain fat, no bone marrow from research animals. Its a good, ethical source of stem cells.

Umbilical-derived stem cells have proven successful in past studies on treatment for arthritis, according to Petrucci.

We did a study at the University of Florida on elbows only and we had success with that study, Petrucci said. We had good success with dogs under 70 pounds and (less) success with dogs over 70 pounds, so we changed our dose, which is why were testing dogs 70 pounds and over in this study.

Criteria for eligibility includes dogs weighing 70 pounds or more, being one year of age or older, in general good health, no neurologic issues, arthritis in up to two joints of the knee, hip, elbow, or shoulder, and have all four functioning limbs.

Owners must bring their dogs back to the clinic after 30 days to check for progress and complete a questionnaire. About 50 to 100 dogs are expected to participate in the study.

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United Therapeutics receives permit for cell therapy facility build-out at Mayo – Jacksonville Daily Record

By daniellenierenberg

United Therapeutics received a building permit Tuesday for a $9.5 million build-out of its cell therapy facility on the second floor of Mayo Clinics Discovery and Innovation Building.

The 21,843-square-foot space will house an automated stem cell manufacturing site, which is one of the first of its kind in the country. The Whiting-Turner Contracting Co. is the project contractor.

The technology, approved by the FDA in 2018, allows the Mayo Clinic Center for Regenerative Medicine to produce cells from the bone marrow of a stem cell donor in large enough quantities to be used as treatments in clinical trials. It allows for the treatment of multiple patients at the same time.

Construction began in 2017 on the $32.4 million building at 14221 Kendall Hench Drive. It held a grand opening in August.

The first floor houses three ex-vivo lung perfusion surgical suites used for lung restoration, another form of regenerative medicine. It turns donor lungs, which previously would have previously been unusable, into viable transplant organs. United Therapeutics also collaborates with Mayo Clinic on lung restoration.

The third floor houses the Life Sciences Incubator for biotech entrepreneurs, which offers coworking space, wet labs, business resources, networking and entrepreneurial training.

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‘I’ve potentially saved a stranger’s life by donating my blood stem cells and it was painless’ – inews

By daniellenierenberg

NewsReal LifeLydia Burgess-Gamble has helped a sick woman in her twenties after they were genetically matched

Tuesday, 22nd October 2019, 10:38 am

Lydia Burgess-Gamble felt a lump in her throat when she got the letter saying her stem cells were a match for someone sick.

The 42-year-old had signed up to the register to be a donor almost three years earlier and hadn't given it much thought since.

Ahead of her 40th birthday, she'd wanted to do something altrusistic. Now she had the chance to potentially save someone's life who was battling a blood cancer or blood disorder.

Donating stem cells today is almost as easy as giving blood. "It was a straightforward and painless process and being able to relax and read a book for a few hours was a luxury," she said.

Patients face difficult odds

Every 20 minutes someone in the UK is diagnosed with a blood cancer, such as leukaemia, myeloma or lymphoma. That's more than 30,000 people a year. Worldwide, it's one every 30 seconds.

Patients suffering with these types of cancers can have their bone marrow damaged by the cancer itself, or from chemotherapy and radiation treatments. A stem cell transplant lets the new stem cells take over from the damaged marrow so the body can produce healthy, cancer-free blood cells.

Even though there are over 27 million people on the worldwide register, this isnt enough, according to charity DKMS. At any one time there are around 2,000 people in the UK in need of a transplant.

Matching donors and patients isnt easy because it's determined by tissue type, not blood group. There are thousands of different human leukocyte antigen (HLA) characteristics, in millions of combinations. Doctors look to relatives for a match but two out of three of those in need are unable to find one, and so must rely on the generosity of strangers.

Most donations are day cases at hospital

Lydia, an environmental research scientist from Brighton, became aware of the process involved through a Facebook post. "A friend shared an appeal for a loved one who needed a donor," she said. "I remember watching a documentary about donating bone marrow in the 90s and I hadn't realised it mainly doesn't involve an invasive procedure until I read this post."

The donation usually involves a nonsurgical procedure called peripheral blood stem cell (PBSC) donation for around 90 per cent of all cases, which is the method Lydia used.

With this method, blood is taken from one of the donors arms and a machine extracts the blood stem cells from it. The donors blood is then returned to their body through their other arm. It is an outpatient procedure that usually takes four to six hours.

'I had no side effects, other than I felt a little more tired than usual the next day'

Lydia Burgess-Gamble

This procedure doesn't "deplete" a donor's supply of stem cells, as a donor's stem cells will completely replenish themselves within two to four weeks afterwards.

"I had no side effects, other than I felt a little more tired than usual the next day but within 24 hours I was completely back to normal," said Lydia.

"All I know about my recipient is that it's a woman in her twenties who lives in Turkey. I'd love to make contact one day. I'm not expecting anything but I'm hoping she gets well and we may be able to meet."

The other 10 per cent of donations are made through bone marrow, where donors give cells from the bone marrow in their pelvis. This is under general anaesthetic so that no pain is experienced. The collection itself takes one to two hours and most donors return to their regular activities within a week. Two weeks after donation, your bone marrow will have recovered fully, and the hip bone will have fully healed within six weeks.

Donating: the process

To become a potential blood stem cell donor first check your eligibility on the DKMS website and request a swab kit for your cheek.

Complete the swabs posted to you at home and send them back. Then yourtissue type will be analysed and your details will be added to the UK stem cell registry. Your details can be searched for a genetic match for people all over the world who need a second chance at life.

The odds are you may never be called upon, but if you are, you will have a blood test at your local GP or hospital and will be asked to complete a medical questionnaire and consent form. If you're deemed fit and healthy enough, you'll have a further medical assessment and consultation at a specialist collection centre (where you will later donate your blood stem cells).

It's important to read about the methods used to collect blood stem cells PBSC and bone marrow donation because if youre on the register, you should be willing to donate in either way. The method will be determined by what the doctors believe will be best for the patient. However, you will of course always have the final decision on whether you are happy to proceed.

When a donor is matched with a patient, DKMS will cover the costs (including any travel, meals, or accommodation expenses that may be necessary and lost wages if you are not covered by your employer).

Your blood stem cells will never be stored, they last for around 72 hours and are delivered straight to the person in need by a special courier.

You will be allowed to meet the patient, if they consent, eventually UK guidelines state this can happen two years after the donation (and tules vary by country).Contact through anonymous letters can be established before this time via DKMS.

You will stay on the register until your 61st birthday.

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Lucknow: In a first, 26-yr-old DMD patient in UP survives with stem cell therapy – ETHealthworld.com

By daniellenierenberg

Lucknow: Duchenne Muscular Dystrophy (DMD) is a deadly genetic disorder, 99.9 per cent people suffering from which, die between the age of 13 to 23 years. However, in a first, a 26-year-old patient from Lucknow has survived DMD by regularly taking stem cells for the last five years.

Children, suffering from DMD, usually die of cardio-respiratory failure. But with the stem cell therapy, this patient has not lost muscle power in last five years and heart and lung muscles and the upper half of the body are working well.

Dr. B.S Rajput, the surgeon who is treating this patient, said, "DMD is a type of muscular dystrophy and being a genetic disorder, it is very difficult to treat. Autologous (from your own body) bone marrow cell transplant or stem cell therapy in such cases was started in Mumbai about 10 years back.

Dr Rajput, who was recently appointed as visiting professor at GSVM Medical College, Kanpur, said he has treated several hundred DMD patients and recently this combination protocol was published in the international Journal of Embryology and stem cell research.

According to Dr Rajput, this disease is endemic in eastern UP, especially Azamgarh, Jaunpur, Ballia and some of the adjoining districts of Bihar, and one out of every 3,500 male child, suffers from the disease.

Yet the disease is not given as much attention as it should be.

Dr Rajput, who is consultant bone cancer and stem cell transplant surgeon from Mumbai, said though patients in Uttar Pradesh and Bihar get financial support from the Chief Minister's Relief Funds, the treatment of autologous bone marrow cell transplant is not included in the package list of Ayushman Bharat scheme, which deprives many from getting the treatment.

The doctor further informed that efforts are being made to establish the department of regenerative medicine in the medical college, where bone marrow cell transplant and stem cell therapy would be done even for other intractable problems like spinal cord injury, arthritis knee and motor neurone disease.

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Making blood in the lab | Interviews – The Naked Scientists

By daniellenierenberg

The volume of blood needed every day by the NHS, and around the world, is staggering. But what about growing blood in a lab, could we do that? Chris Smith was joined by Cedric Ghevaert from the department of haematology at the University of Cambridge, to find out more about lab grown blood...

Cedric - So platelets are one of the three main blood components. Theyre actually the smallest cell in the body, if you look at a millimetre on a ruler you can line 250 platelets in that millimetre. The interesting thing is that some people argue they're not even a cell, because they don't contain a nucleus, they don't have DNA.

Chris - When I was learning biology at school they said platelets are bits of cells.

Cedric - That's absolutely true. They are fragments of a parent cell that lives in the bone marrow, and called a megakaryocyte, and one megakaryocyte will release about 1000 to 2000 of these little fragments called the platelets.

Chris - Every day?

Cedric - Every day we produce 10 to the 11 platelets

Chris - 100 billion Every day?

Cedric - Thats right.

Chris - And so these cells are just budding off little bits of themselves, which then go circulating around in the bloodstream.

Cedric - That's absolutely right.

Chris - And what are they doing there? What is their role?

Cedric - The main role of the platelets is to monitor your blood vessels, so they contain two things: Their outer layer, which is called the membrane. And on that membrane they have all sorts of little receptors, and these receptors will tell the platelets that the blood vessel has been damaged. When the platelet detects that it does two things, it will attach to the damaged blood vessel, and it will become activated and by that, it will then tell other platelets around, you need to come and help. These platelets will then stick to each other and form a plug to literally block the hole.

Chris - Do they change shape or anything? Do they become, sort of spiky or anything, to become more jagged so they jam in the hole.

Cedric - So they do become spiky, indeed you see that under the microscope when you activate the platelets. They go from a disc to this sort of spider, and that allows them to indeed interact with each other even better. The thing that they also do, is to then pull. They literally pull the wound together to try to stem the blood flow.

Chris - And they're presumably the first responders when you have a wound. They're there first because they're at the scene of the crime already because they're in the blood. And then as more come along, they're recruiting more of their mates from the numbers that come in the blood flow, and what do they provide the initial foundation of a blood thrombus or a clot.

Cedric - That's absolutely right. So once they become activated, inside the platelets there are granules, and those granules contain things that tell the blood, you need to clot. These things are released when the platelets become activated. And that leads to an amplification of the blood clotting that proteins are linked together, they form a polymer, and that polymer is a sort of a mesh that will capture more platelets and really plug the hole.

Chris - So the platelets are pulling more raw materials that are dissolved in the blood into that wound site, and then turning it into this dense mesh work that's gonna be a stable repair.

Cedric - That's absolutely right.

Chris - So they're really critical aren't they?

Cedric - Absolutely.

Chris - We can't do without them. And what's the problem with just growing them in a dish, because we can grow loads of things in dishes these days. We can you know, cells grow in dishes easily. So why can't you just churn out platelets in a dish?

Cedric - The main challenge with producing platelets in a dish is to do it so efficiently that actually we have a product that can be used, for example by the NHS and cost efficient. So if you look at a bag of platelets which we give for a transfusion, it contains three times ten to the eleven.

Chris - So 300 billion platelets.

Cedric - So where the platelets score as it were, is that we only need to produce one megakaryocyte, to produce a thousand platelets. And we can grow the megakaryocyte from stem cells. So the idea is that we can take stem cells, grow them into a megakaryocyte, and then right at the last stage of production, suddenly you have this massive amplification, a thousand times more platelets than you had megakaryocytes.

Chris - But if it's that easy to just grow these things in a dish, why are we not doing it? What is the what's the problem at the moment.

Cedric - The main problem is that particular last step. When the megakaryocyte is in the bone marrow it gets its cue from its environment. And it will detect the blood flow. It will be talked to by the cells that are around it, and that, it's very difficult to reproduce in the dish. If we produce megakaryocytes in liquid, in a culture dish they can produce 1 to 10 platelets, so we are at least a hundred times below what a megakaryocyte can do.

Chris - So you've got to have some way of recreating that very specialised three dimensional relationship in the bone marrow, where all these cells are in contact in a particularly special arrangement which seems to be the cue to them, to churn out platelets with the efficiency that they do when they're inside the body.

Cedric - And that's exactly the challenge that my group and several other groups across the world are trying to answer. And there are two ways to do this. First we need to tell the megakaryocyte theres a flow. They sense the flow, and that makes them release the platelets. So we put them in a bioreactor where they're exposed to shear, which is basically fluid going along them.

Chris - That's kind of mimicking the blood flowing through the bone marrow. So that would normally be bending and distorting the cells a bit, I presume, and that's what makes them churn off or snap off bits.

Cedric - That's right. So they produce these long digits which we call proplatelets, and these long digits elongate in the bloodstream and then snap off these platelets.

Chris - And when you make them, having mimicked this as best you can, do the platelets that you produce in the dish look like, and critically work like, the ones that are made naturally in the bone marrow?

Cedric - That's the critical thing that we are trying to address at the moment. They are bigger when we produce them in the dish, and they don't seem to quite react like normal platelets. However that doesn't mean that it wont work really well. What we need to do is to test them through a range of assays to really make the statement; these platelets are good, they will monitor your blood vessel, they will last in circulation.

Chris - Is your aim to make platelets bespoke for a patient? Or would you make off the shelf platelets, a bit like we'd currently do with transfusion medicine, where we just make a big bag of platelets collected from a range of donors?

Cedric - So at the moment we can produce platelets from either four donations, from four different donors, or we take them off a special machine where we have one pool of platelets coming from one donor, but we've talked the blood group before, one of the challenges with platelets, is that some people are immunised and they need platelets of a very specific blood type.

Chris - When you say immunisation, you mean that they've made an immune reaction to certain types in the past, so you've got to basically restrict what types you give them?

Cedric - Exactly. The beauty of working with stem cells is that we can edit the DNA somewhat, and because we can edit the DNA we can actually make platelets that don't express blood group, that are universal platelets. The one we produce in the dish can go to anyone. And that's one of the beauties of this technology.

Chris - And are you far away?

Cedric - We are not that far away. We are looking at human clinical trials in the next two to three years.

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Orchard Therapeutics Presents Data from OTL-200 in Patients with Metachromatic Leukodystrophy Using Cryopreservation – BioSpace

By daniellenierenberg

BOSTON and LONDON, Oct. 22, 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 initial results from a clinical trial with a cryopreserved formulation of OTL-200, a gene therapy in development for the treatment of metachromatic leukodystrophy (MLD) at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. The initial data show that cellular engraftment with OTL-200 using a cryopreserved formulation is similar to that observed using a fresh formulation with the longest patient having 12 months of follow-up since treatment. The data are being featured this week in a poster session at the European Society of Gene & Cell Therapy (ESGCT) Annual Congress in Barcelona, Spain.

MLD is a devastating and rapidly progressing disease with no standard treatment options. In its most severe forms, patients will not survive beyond their first decade of life.

These data compare the initial results of OTL-200 in the first four MLD patients treated using a cryopreserved formulation to a previously presented integrated analysis of 29 patients treated with a fresh formulation that demonstrated meaningful clinical outcomes. Hematopoietic stem cells are collected, purified and transduced in the same way for both formulations. For the cryopreserved formulation, following transduction, the gene-corrected cells are placed in a specific medium that allows them to be stably frozen. After successful testing and release, the cryopreserved cells are shipped to the site of care where they are thawed and administered to patients who have received conditioning.

Presenting the first supportive data on OTL-200 using a cryopreserved formulation represents a cross-functional effort involving our clinical, CMC and regulatory teams as we prepare for the upcoming European regulatory submission for MLD followed by a BLA in the U.S., said Mark Rothera, president and chief executive officer of Orchard. If approved, a cryopreserved formulation of OTL-200 would more readily facilitate global commercialization and patient access efforts, which are key elements in our mission to deliver potentially curative therapies to patients suffering from often-deadly rare diseases.

Mr. Rothera continued, With over 40 patients now treated using a cryopreserved formulation across our pipeline of six clinical-stage programs, we are confident our approach is supported by a robust set of evidence.

Study Results At the time of the analysis, four early-onset MLD patients (two late infantile and two early juvenile) have been treated with the cryopreserved formulation of OTL-200. All patients are alive and were followed for a minimum of one month, with the longest follow-up out to 12 months in the first patient treated (median follow-up of 0.38 years). The age at the time of treatment ranged from seven months to 42 months.

The initial results in patients receiving the cryopreserved formulation (n=4) demonstrated the following:

Figure 1. Profiles of VCN in bone marrow CD34+ cells: OTL-200 cryopreserved vs. OTL-200 fresh

https://www.globenewswire.com/NewsRoom/AttachmentNg/83f41457-927b-4b1b-9ac2-9d48ac10353a

Figure 2. ARSA activity profile in peripheral blood: OTL-200 cryopreserved vs. OTL-200 fresh

https://www.globenewswire.com/NewsRoom/AttachmentNg/393ca5f0-98ad-47f8-b723-35c5c6c08d8f

c = cryopreserved; f = fresh; Sbj. = subject

We are pleased that these initial data suggest that using gene-corrected cells that have been cryopreserved has a similar impact on clinical biomarkers for early-onset MLD patients as the OTL-200 fresh formulation, said Dr. Valeria Calbi, a hematologist at San Raffaele Scientific Institute and SR-Tiget and an investigator of the study. The four treated patients showed good levels of engraftment of gene-corrected cells and reconstitution of ARSA activity at multiple time points, as well as encouraging early trends in GMFM scores that we look forward to evaluating with additional follow-up. We believe that these data further support the positive benefit / risk profile of OTL-200 as a therapy with potential lifelong benefit for patients with MLD.

Next Steps for OTL-200 Orchard remains on track to submit a marketing authorization application, or MAA, in Europe for MLD in the first half of 2020, as well as a biologics licensing application, or BLA, in the U.S. approximately one year later.

About MLD and OTL-200Metachromatic leukodystrophy (MLD) is a rare and life-threatening inherited disease of the bodys metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, the gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged and patients with MLD will experience neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures, finding it more and more difficult to move, talk, swallow, eat and see. Currently, there are no effective treatments for MLD. In its late infantile form, mortality at 5 years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1 OTL-200 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy being studied for the treatment of MLD. OTL-200 was acquired from GSK in April 2018 and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and Fondazione Telethon, acting through their joint San Raffaele-Telethon Institute for Gene Therapy in Milan, initiated in 2010.

About OrchardOrchard Therapeutics is 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 of ex vivo, autologous, hematopoietic stem cell (HSC) based gene therapies includes Strimvelis, a gammaretroviral vector-based gene therapy and the first such treatment approved by the European Medicines Agency for 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 in April 2018 and originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy initiated in 2010.

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

Forward-Looking StatementsThis press release contains certain forward-looking statements 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, Orchards expectations regarding the timing of regulatory submissions for approval of its product candidates, including OTL-200 for the treatment of metachromatic leukodystrophy, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates, including OTL-200, and the likelihood that such data will be positive and support further clinical development and regulatory approval of its 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 OTL-200, 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 ended December 31, 2018 as filed with the U.S. Securities and Exchange Commission (SEC) on March 22, 2019, as well as subsequent filings and reports filed with the SEC. 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.

1Mahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with the Late Infantile Variant and a Systematic Review of the Literature. Journal of Child Neurology 2010, DOI: http://doi.org/10.1177/0883073809341669

Contacts

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

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

Figure 1

Profiles of VCN in bone marrow CD34+ cells: OTL-200 cryopreserved vs. OTL-200 fresh

Figure 2

ARSA activity profile in peripheral blood: OTL-200 cryopreserved vs. OTL-200 fresh; c = cryopreserved; f = fresh; Sbj. = subject

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