Archive for the ‘Bone Marrow Stem Cells’ Category
Stem Cell Therapy Market Revenue, Demands and Gross Margin, Forecasts to 2026 (Based on 2020 COVID-19 Worldwide Spread) – Jewish Life News
GlobalStem Cell Therapy Market, delivering a must-read report for industry stakeholders wanting to understand the strategic landscape of this burgeoning sector. Readers will find an in-depth analysis of the market and how it will impact existing traditional markets, as well as insights into future development and opportunities across the globe.
MarketInsightsReports has announced the addition of the Global Stem Cell Therapy Market Research Report 2020 The report focuses on global major leading players with information such as company profiles, product picture and specification.
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Key Players:
Osiris Therapeutics, NuVasive, Chiesi Pharmaceuticals, JCR Pharmaceutical, Pharmicell, Medi-post, Anterogen, Molmed, Takeda (TiGenix), request free sample for complete list of companies.
The leading players of industry, their market share, product portfolio, company profiles are covered in this report. The competitive market scenario among players will help the industry aspirants in planning their strategies.
Summary
Stem-cell therapy is the use of stem cells to treat or prevent a disease or condition. Bone marrow transplant is the most widely used stem-cell therapy, but some therapies derived from umbilical cord blood are also in use.
In the last several years, global stem cell therapy market developed fast at a average growth rate of 46.81%.
Since the COVID-19 virus outbreak in December 2019, the disease has spread to almost 100 countries around the globe with the World Health Organization declaring it a public health emergency. The global impacts of the coronavirus disease 2019 (COVID-19) are already starting to be felt, and will significantly affect the Stem Cell Therapy market in 2020.
COVID-19 can affect the global economy in three main ways: by directly affecting production and demand, by creating supply chain and market disruption, and by its financial impact on firms and financial markets.
The outbreak of COVID-19 has brought effects on many aspects, like flight cancellations; travel bans and quarantines; restaurants closed; all indoor events restricted; over forty countries state of emergency declared; massive slowing of the supply chain; stock market volatility; falling business confidence, growing panic among the population, and uncertainty about future.This report also analyses the impact of Coronavirus COVID-19 on the Stem Cell Therapy industry.
Stem Cell Therapy Market Segmentation by types, Applications and regions:
Market Segment by Type covers:
Autologous
Allogeneic
Market Segmented by Applications:
Musculoskeletal Disorder
Wounds & Injuries
Cornea
Cardiovascular Diseases
Others
Market Segment by Regions:
North America (the United States, Canada, and Mexico)Europe (Germany, France, UK, Russia, and Italy)Asia-Pacific (China, Japan, Korea, India, and Southeast Asia)South America (Brazil, Argentina, Colombia, etc.)
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These segments are thoroughly evaluated on an individual basis and a team of analysts has ensured to give a crystal clear idea about various lucrative segments of the industry. This detailed analysis using segmentation by providing precise results on industry-related markets.
The report also analyzed the evolution of industry trends. Several macroeconomic factors such as Gross domestic product (GDP) and the increasing inflation rate is expected to affect directly or indirectly in the development of the industry.
Frequently Asked Questions about global market:
Customization of the Report:This report can be customized as per your needs for additional data up to 3 companies or 3 countries or nearly 40 analyst hours.
Note:
All the reports that we list have been tracking the impact of COVID-19 the market. Both upstream and downstream of the entire supplychain has been accounted for while doing this. Also, where possible, we will provide an additional COVID-19 update supplement/report to the report in Q3, please check for with the sales team.
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Stem Cell Therapy Market Revenue, Demands and Gross Margin, Forecasts to 2026 (Based on 2020 COVID-19 Worldwide Spread) - Jewish Life News
Molecules identified that reverse cellular aging process – New Atlas
Central to a lot of scientific research into aging are tiny caps on the ends of our chromosomes called telomeres. These protective sequences of DNA grow a little shorter each time a cell divides, but by intervening in this process, researchers hope to one day regulate the process of aging and the ill health effects it can bring. A Harvard team is now offering an exciting pathway forward, discovering a set of small molecules capable of restoring telomere length in mice.
Telomeres can be thought of like the plastic tips on the end of our shoelaces, preventing the fraying of the DNA code of the genome and playing an important part in a healthy aging process. But each time a cell divides, they grow a little shorter. This sequence repeats over and over until the cell can no longer divide and dies.
This process is linked to aging and disease, including a rare genetic disease called dyskeratosis congenita (DC). This is caused by the premature aging of cells and is where the Harvard University team focused its attention, hoping to offer alternatives to the current treatment that involves high-risk bone marrow transplants and which offers limited benefits.
One of the ways dyskeratosis congenita comes about is through genetic mutations that disrupt an enzyme called telomerase, which is key to maintaining the structural integrity of the telomere caps. For this reason, researchers have been working to target telomerase for decades, in hopes of finding ways to slow or even reverse the effects of aging and diseases like dyskeratosis congenita.
Once human telomerase was identified, there were lots of biotech startups, lots of investment, says Boston Childrens Hospital's Suneet Agarwal, senior investigator on the new study. But it didnt pan out. There are no drugs on the market, and companies have come and gone.
Agarwal has been studying the biology of telomerase for the past decade, and back in 2015 he and his team discovered a gene called PARN that plays a role in the action of the telomerase enzyme. This gene normally processes and stabilizes an important component of telomerase called TERC, but when it mutates, it results in less of the enzyme being produced and, in turn, the telomeres becoming shortened prematurely.
For the new study, Harvard researchers screened more than 100,000 known chemicals in search of compounds that could preserve healthy function of PARN. This led them to small handful that seemed capable of doing so by inhibiting an enzyme called PAPD5, which serves to unravel PARN and destabilize TERC.
We thought if we targeted PAPD5, we could protect TERC and restore the proper balance of telomerase, says Harvard Medical Schools Neha Nagpal, first author on the new paper.
These chemicals were tested on stem cells in the lab, made from the cells of patients with dyskeratosis congenita. These compounds boosted TERC levels in those stem cells and restored telomeres to their normal length. However, rather than a scattergun approach, the team really wanted to test for safety and see if the treatment could precisely target stem cells carrying the right ingredients for telomerase formation.
More specifically, the team wanted to see if this could be achieved by having the PAPD5-inhibiting drugs recognize and respond to another important component of telomerase, a molecule called TERT. To do so, in the next round of experiments the team used human blood stem cells and triggered mutations in the PARN gene that give rise to dyskeratosis congenita. These were then implanted into mice that were treated with the compounds, with the team finding the treatment boosted TERC, restored telomere length in the stem cells and had no ill effects on the rodents.
This provided the hope that this could become a clinical treatment, says Nagpal.
The team will now continue its work in an effort to prove these small molecules are a safe and effective way to apply the brakes to dyskeratosis congenita, other diseases, and possibly aging more broadly.
We envision these to be a new class of oral medicines that target stem cells throughout the body, Agarwal says. We expect restoring telomeres in stem cells will increase tissue regenerative capacity in the blood, lungs, and other organs affected in DC and other diseases.
The research was published in the journal Cell Stem Cell.
Source: Boston Childrens Hospital via Harvard University
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Molecules identified that reverse cellular aging process - New Atlas
Coronavirus Doctors experiment with stem cell therapy on COVID-19 patients CBS News 9:39 AM – KTVQ Billings News
Doctors are hoping stem cell therapy could be a weapon in the fight against coronavirus. On Friday, regenerative medicine company Mesoblast announced a 300-person trial to determine whether stem cell treatments will work in COVID-19 patients suffering from severe lung inflammation.
One hospital in New York tried it as an experiment with 12 patients, 10 of whom were able to come off of ventilators.
"What we saw in the very first patient was that within four hours of getting the cells, a lot of her parameters started to get better," Dr. Karen Osman, who led the team at Mount Sinai, told CBS News' Adriana Diaz.
The doctor said she was encouraged by the results, though she was hesitant to link the stem cell procedure to her patients' recovery.
"We don't know" if the 10 people removed from ventilators would not have gotten had they not gotten the stem cells, she said. "And we would never dare to claim that it was related to the cells."
She explained that only a "randomized controlled trial" would be the only way "to make a true comparison."
Luis Naranjo, a 60-year-old COVID-19 survivor, was one of Mount Sinai's stem cell trial success stories. He told Diaz in Spanish that he was feeling "much better."
Naranjo's daughter, Paola, brought him to the emergency room, fearful she would not see her father again. Like so many families struck by the coronavirus, she was not allowed inside with him.
"I forgot to tell him that I love him," she said. "All I said was go inside, I hope you feel better."
During his hospital stay, Naranjo was unconscious and on a ventilator for 14 days.
Doctors proposed giving him stem cells from bone marrow in hopes it would suppress the severe lung inflammation caused by the virus.
Now, Naranjo credits the doctors who treated him for his survival. Though income from his family's jewelry business has been cut off and they found themselves falling behind on rent, Naranjo said he is focused primarily on his recovery and regaining the 25 pounds he lost at the hospital.
Although stem cell treatment, usually reserved for other diseases like rheumatoid arthritis, might end up being another step toward helping coronavirus patients recover, Dr. Osman was quick to say it would not be a "miracle treatment."
"The miracle treatment will be a vaccine," she said.
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Coronavirus Doctors experiment with stem cell therapy on COVID-19 patients CBS News 9:39 AM - KTVQ Billings News
‘Stem cell therapy more effective on Covid-19’ – Korea Biomedical Review – Korea Biomedical Review
I dont know why people pay attention only to vaccines and treatments against the new coronavirus. Stem cell therapies are more useful to treat Covid-19.
So claimed Lee Hee-young, president of the Korean Association of Stemcell Therapy, at a news conference in Seoul, Monday. He called for active use of stem cell therapies to treat Covid-19 patients.
Several studies have proved the effects of autologous stem cells in treating acute respiratory distress syndrome (ARDS), which is the leading cause of death in Covid-19 patients, Lee said. The concept of stem cell therapy is the same as that of blood transfusion or bone marrow transplantation. Decades of cell therapies have proved that stem cell therapy is safe.
While the development of a treatment or a vaccine against Covid-19 takes a long time and it may not be able to treat patients immediately because of virus mutation possibilities, stem cell therapies can restore damaged lungs directly, Lee claimed.
It is more important to restore damaged lungs than to fight the virus. Stem cell therapy restores the lungs, giving patients time to beat the virus, he went on to say. However, people are paying attention to vaccine or treatment candidates only. This is why I am holding a news conference.
Lee pointed out that the local environment makes it difficult to use stem cell therapies. Thus, the government should ease regulations on the management and use of cell culture facilities so that doctors can perform stem cell therapies with simple cell culture, he said.
As long as physicians have a positive pressure facility and a culture kit, they can separate and culture cells with simple training, he said. If the authorities allow doctors to perform stem cell therapies with a disposable mobile culture autonomously, the cost of stem cell therapies will go down significantly.
Lee added that he asked related officials to include such rules in the Act on Safety and Support for Advanced Regenerative Medicine and Advanced Biopharmaceuticals, which is to take effect in the second half of the year.
< Korea Biomedical Review, All rights reserved.>
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'Stem cell therapy more effective on Covid-19' - Korea Biomedical Review - Korea Biomedical Review
BCU biology professor offers tips to prevent COVID-19 infection – Sioux City Journal
In some patients, COVID-19 has triggered a cytokine storm, an immune system response in which the body attacks its own cells. Jung painted a picture of a boxing match in which "fighter" immune cells are being called upon to battle the virus. This battle generates lots of fluid, waste and pus, making it difficult for the alveoli to pick up the oxygen a person breathes in, leading to multi-organ failure.
"These immune cells, neutrophils and other fighter immune cells, are like that. They don't care if it's a virus or our own cells. If you're infected, they're all enemies," Jung said. "So what they're going to do is they're going to start to kill everybody, basically."
Why exactly some people's immune systems go into overdrive, Jung said, is unknown, but he said it can happen to anybody.
"If we are up to the level where we can fight well without going into a coma or anything, then 14 days later, our body can provide an antibody," Jung said. "An antibody will neutralize this virus very quietly."
Strengthening the immune system
Eating certain foods can help keep your immune cells strong. Jung said vegetables, for example, stimulate the circulation of blood cells from bone marrow.
"Those bioactive reagents can support our immune systems by sending them the appropriate amount of stem cells, just in case some tissue cells are damaged and we need to replenish them. For example, if your lung cell has been damaged and they need to be replaced, that could be done by the stem cell that has been moved from the bone marrow and located around the lung area," said Jung, who encourages eating a variety of different colored vegetables.
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BCU biology professor offers tips to prevent COVID-19 infection - Sioux City Journal
Planned Clinical Trial of Allogeneic Stem Cell Therapy Remestemcel-L in Patients with COVID-19 – Cancer Network
Mount Sinai Health System announced that they will be using remestemcel-L (Ryoncil), an innovative allogeneic stem cell therapy, in patients with coronavirus disease 2019 (COVID-19).
Additionally, Mount Sinai indicated that they will play a central role in a clinical trial for patients with severe acute respiratory distress syndrome, which affects individuals with severe cases of COVID-19.
Remestemcel-L has previously been tested in patients who have had a bone marrow transplant, who can experience an overactive immune response similar to that observed in severe cases of COVID-19.
Mount Sinai began administering remestemcel-L to patients in late March under the FDAs compassionate use program. The therapy was given to 10 patients with moderate to severe cases of COVID-19-related acute respiratory distress syndrome (ARDS), most of whom were on ventilators, and the doctors saw encouraging results.
We are encouraged by what we have seen so far and look forward to participating in the randomized controlled trial starting soon that would better indicate whether this is an effective therapy for patients in severe respiratory distress from COVID-19, Keren Osman, MD, medical director of the Cellular Therapy Service in the Bone Marrow and Stem Cell Transplantation Program at The Tisch Cancer Institute at Mount Sinai and associate professor of Hematology and Medical Oncology at the Icahn School of Medicine at Mount Sinai, said in a press release.
The randomized clinical trial evaluating the therapeutic benefit and safety of remestemcel-L will be conducted at Mount Sinai, which will serve as the clinical and data coordinating center. The stem cell therapy will be evaluated in 240 patients with COVID-19-related ARDS in the US and Canada. Moreover, the trial will be conducted as a public-private partnership between the Cardiothoracic Surgical Trials Network.
The coronavirus pandemic has caused exponential increases of people suffering with acute respiratory distress syndrome, requiring intubation and mechanical ventilation with many dying, Annetine Gelijns, PhD, the Edmond A. Guggenheim Professor of Health Policy at the Icahn School of Medicine at Mount Sinai, said in a press release. We have designed a clinical trial that will expeditiously determine whether the stem cell therapy will offer a life-saving therapy for a group of patients with a dismal prognosis.
Remestemcel-L consists of mesenchymal stem cells. The therapy was previously assessed in a phase III trial in children who had graft-versus-host disease (GVHD), which can occur after bone marrow transplants. Further, the inflammation that occurs in GVHD is the result of a cytokine storm. A similar cytokine storm has been found to take place in patients with COVID-19 who develop acute respiratory distress syndrome.
These stem cells have shown excellent response rates in severe graft-versus-host disease in children, John Levine, MD, professor of Hematology, Medical Oncology, and Pediatrics at the Icahn School of Medicine at Mount Sinai, who is also the co-director of the Mount Sinai Acute GVHD International Consortium (MAGIC), said in a press release. Mesenchymal stem cells have a natural property that dampens excessive immune responses.
Some institutions have also begun testing anti-IL-6 agents, such as tocilizumab (Actemra), for the treatment of cytokine release syndrome in patients with COVID-19 who develop acute respiratory distress syndrome.
Reference:
Mount Sinai Leading the Way in Innovative Stem Cell Therapy for COVID-19 Patients [news release]. New York, NY. Published April 9, 2020. newswise.com/coronavirus/mount-sinai-leading-the-way-in-innovative-stem-cell-therapy-for-covid-19-patients/?article_id=729684&sc=dwhr&xy=10019792. Accessed April 15, 2020.
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Planned Clinical Trial of Allogeneic Stem Cell Therapy Remestemcel-L in Patients with COVID-19 - Cancer Network
Robust response by NIT-T alumni to tackle Covid-19 pandemic – The Hindu
Alumni members of National Institute of Technology - Tiruchi (NIT-T) holding key positions in tech companies across the globe have responded overwhelmingly through their knowledge support, innovative devices, fund mobilisation, and production of personal protection equipment for dealing with the COVID-19 pandemic.
An associate of Global Consortium of Cell Therapy Companies, Stempeutics, an Indian stem cell company of which B. N Manohar, an alumnus of ECE 1977 batch is the Chief Executive Officer, will shortly be supplying clinical-grade Mesenchymal Stem Cells (multi-potent stem cells found in the bone marrow used for making and repairing skeletal tissues) to those in need. Manufactured in the Manipal GMP facility under approval of Drug Controller General of India, the multi-potent stem cells have been found to reduce the symptoms of pneumonia induced by COVID-19 and halt its advancement to Acute Respiratory Distress Syndrome, NIT-T Director Mini Shaji Thomas said.
S. K. Ramesh, an alumnus of 1981 batch ECE holding a senior position in California State University, Northridge, is involved along with his colleagues in creation of life saving face shields and other personal protection equipment for donating the same to healthcare workers in hospitals throughout Southern California.
Blooom Energy, founded by K. R. Sridhar, who had completed his mechanical engineering degree from the then Regional Engineering College Tiruchi, and subsequently did his masters degree in Nuclear Engineering, and Ph.D. in Mechanical Engineering from the University of Illinois, Urbana-Champaign, has undertaken the task of repairing ventilators on a bulk scale in partnership with Stanford Health Care.
Chief Innovation Officer at Dulso, United Arab Emirates, Madhumohan Sreeram, an alumnus of NIT-T who had completed B.Tech in Chemical Engineering in 1982, has been in the forefront in carrying out sanitisation of the municipality of Dubai after identifying a suitable disinfectant QUATPLUS TB, which is a Quaternary Ammonium Compound product approved by United States Environmental Protection Agency and American chemistry councils Center Biocide Chemistry (CBC) and has been listed in their recommended EPA pre-approved products for COVID-9 disinfection application.
Richard Sekar, an alumnus who had completed Production Engineering in 1983 leads Warriors Against Virus a team of 371 volunteers in the Bay Area, USA, for stitching facial masks for hospital requirement. IcarusNova, of which Sapna Behar, an alumna (1990, EEE), is the Director and Founder, has partnered with LifeSignals to design a wireless biosensor-based patch, with ISO 13485 accreditation, for early detection and continuous monitoring of COVID-19 symptoms. The patches when affixed on the chest area can monitor the temperature, breathing rate, trace ECG and heart rate as a real time data. The data can be transferred to the users phone through an app. The system reduces the risk of contamination between patients and other individuals.
Admiral Superintendent of Naval Dockyard, Vishakapatnam, Sreekumar Nair (ECE, 1986), has led a team to design an innovative portable multi-feed oxygen manifold using a six-way radial header fitted to a single cylinder. This becomes essential when the existing hospital facilities for critical care management becomes limited and a need arises for catering to multiple patients. Rapid trials have been done at Naval Hospital INHS Kalyani. The entire assembly could be set up within 30 minutes.
Appreciating the efforts of the alumni members, Prof. Mini Shaji Thomas said the various contributions in diverse sectors was a matter of pride.
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Robust response by NIT-T alumni to tackle Covid-19 pandemic - The Hindu
FDA Approves New Therapy for Triple Negative Breast Cancer That Has Spread, Not Responded to Other Treatments – Herald-Mail Media
SILVER SPRING, Md., April 22, 2020 /PRNewswire/ --Today, the U.S. Food and Drug Administration granted accelerated approval to Trodelvy (sacituzumab govitecan-hziy) for the treatment of adult patients with triple-negative breast cancer that has spread to other parts of the body. Patients must have received at least two prior therapies before taking Trodelvy.
"Metastatic triple-negative breast cancer is an aggressive form of breast cancer with limited treatment options. Chemotherapy has been the mainstay of treatment for triple-negative breast cancer. The approval of Trodelvy today represents a new targeted therapy for patients living with this aggressive malignancy," said Richard Pazdur, M.D., director of the FDA's Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA's Center for Drug Evaluation and Research. "There is intense interest in finding new medications to help treat metastatic triple-negative breast cancer. Today's approval provides patients who've already tried two prior therapies with a new option."
Trodelvy is a Trop-2-directed antibody and topoisomerase inhibitor drug conjugate, meaning that the drug targets the Trop-2 receptor that helps the cancer grow, divide and spread, and is linked to topoisomerase inhibitor, which is a chemical compound that is toxic to cancer cells. Approximately two of every 10 breast cancer diagnoses worldwide are triple-negative. Triple-negative breast cancer is a type of breast cancer that tests negative for estrogen receptors, progesterone receptors and human epidermal growth factor receptor 2(HER2) protein. Therefore, triple-negative breast cancer does not respond to hormonal therapy medicines or medicines that target HER2.
"As part of FDA's ongoing and aggressive commitment to address the novel coronavirus pandemic, we continue to keep a strong focus on patients with cancer who constitute a vulnerable population at risk of contracting the disease," said Pazdur. "At this critical time, we continue to expedite oncology product development. This application was approved more than a month ahead of the FDA goal date an example of that commitment. Our staff is continuing to meet with drug developers, academic investigators, and patient advocates to push forward the coordinated review of treatments for cancer."
The FDA approved Trodelvy based on the results of a clinical trial of 108 patients with metastatic triple-negative breast cancer who had received at least two prior treatments for metastatic disease. The efficacy of Trodelvy was based on the overall response rate (ORR) which reflects the percentage of patients that had a certain amount of tumor shrinkage. The ORR was 33.3%, with a median duration of response of 7.7 months. Of the patients with a response to Trodelvy, 55.6% maintained their response for 6 or more months and 16.7% maintained their response for 12 or more months.
The prescribing information for Trodelvy includes a Boxed Warning to advise health care professionals and patients about the risk of severe neutropenia (abnormally low levels of white blood cells) and severe diarrhea. Health care professionals should monitor patient's blood cell counts periodically during treatment with Trodelvy and consider treatment with a type of therapy called granulocyte-colony stimulating factor (G-CSF), which stimulates the bone marrow to produce white blood cells called granulocytes and stem cells and releases them into the bloodstream, to help prevent infection, and should initiate anti-infective treatment in patients with febrile neutropenia (development of fever when white blood cell are abnormally low).
Additionally, health care professionals should monitor patients with diarrhea and give fluid, electrolytes, and supportive care medications, as needed. Trodelvy may need to be withheld, dose reduced or permanently discontinued for neutropenia or diarrhea. Trodelvy can cause hypersensitivy reactions including severe anaphylactic (allergic) reactions. Patients should be monitored for infusion-related reactions and health care professionals should discontinue Trodelvy if severe or life-threatening reactions occur. If patients experience nausea or vomiting while taking Trodelvy, health care professionals should use antiemetic preventive treatment, to prevent nausea and vomitting. Patients with reduced uridine diphosphate-glucuronosyl transferase 1A1 (UGT1A1) activity are at increased risk for neutropenia following initiation of Trodelvy treatment.
The most common side effects for patients taking Trodelvy were nausea, neutropenia, diarrhea, fatigue, anemia, vomiting, alopecia (hair loss), constipation, decreased appetite, rash and abdominal pain.
Women who are pregnant should not take Trodelvy because it may cause harm to a developing fetus or newborn baby. The FDA advises health care professionals to inform females of reproductive age to use effective contraception during treatment with Trodelvy and for 6 months after the last dose. Male patients with female partners of reproductive potential should also use effective contraception during treatment with Trodelvy and for three months after the last dose.
Trodelvy was granted accelerated approval, which enables the FDA to approve drugs for serious conditions to fill an unmet medical need based on a result that is reasonably likely to predict a clinical benefit to patients. Further clinical trials are required to verify and describe Trodelvy's clinical benefit.
The FDA granted this application Priority Review andBreakthrough Therapydesignation, which expedites the development and review of drugs that are intended to treat a serious condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies. Trodelvy was also granted Fast Trackdesignation, which expedites the review of drugs to treat serious conditions and fill an unmet medical need.
The FDA granted approval of Trodelvy to Immunomedics, Inc.
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Media Contact:Nathan Arnold, 301-796-6248Consumer Inquiries: Emailor 888-INFO-FDA
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation's food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.
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FDA Approves New Therapy for Triple Negative Breast Cancer That Has Spread, Not Responded to Other Treatments - Herald-Mail Media
Pivotal REACH2 Study Data Published in NEJM Highlight Superior Efficacy of Ruxolitinib versus Best Available Therapy in Patients with Acute GVHD -…
WILMINGTON, Del.(BUSINESS WIRE)Incyte (Nasdaq:INCY) today announced that data from the Phase 3 REACH2 study have been published in The New England Journal of Medicine demonstrating that ruxolitinib (Jakafi) improves outcomes across a range of efficacy measures in patients with steroid-refractory acute graft-versus-host disease (GVHD) compared to best available therapy (BAT). The results of REACH2, the first Phase 3 study of ruxolitinib in acute GVHD to have met its primary endpoint, reinforce findings from the previously-reported Phase 2 REACH1 study.
In REACH2, patients treated with ruxolitinib experienced a significantly greater overall response rate (ORR) vs. BAT (62% vs. 39%; p<0.001) at Day 28, the primary endpoint of the study. For the key secondary endpoints, patients treated with ruxolitinib maintained significantly higher durable ORR (40% vs. 22%; p<0.001) at Day 56. In addition, ruxolitinib was associated with longer median failure free survival (FFS) than BAT (5.0 months vs. 1.0 months; hazard ratio 0.46, 95% CI, 0.35 to 0.60) and showed a positive trend with other secondary endpoints, including duration of response1,2.
No new safety signals were observed, and the ruxolitinib safety profile in REACH2 was consistent with that seen in previously reported studies in steroid-refractory acute GVHD. The most frequently reported adverse events among study participants were thrombocytopenia and anemia. While 38% and 9% of patients required ruxolitinib and BAT dose modifications, the number of patients who discontinued treatment due to AEs was low (11% and 5%, respectively)1,2.
The results from the REACH2 study reinforce findings from the pivotal REACH1 trial and demonstrate the potential that ruxolitinib has to effectively and safely improve outcomes for patients with GVHD, said Peter Langmuir, M.D., Group Vice President, Oncology Targeted Therapies, Incyte. We are committed to continuing our research in GVHD with the goal of providing more effective treatment options for patients living with this disease, and look forward to the results of the REACH3 study in steroid-refractory chronic GVHD later this year.
The REACH2 data were also accepted as an oral presentation as part of the Presidential Symposium at the European Society for Blood and Marrow Transplantation (EBMT) Annual Meeting to be held 30 August to 2 September in Madrid, Spain.
Patients with acute graft-versus-host disease face life-threatening challenges with limited treatment options, particularly for the nearly half of individuals who do not respond to initial steroid therapy, said Robert Zeiser, University Hospital Freiburg, Department of Haematology, Oncology and Stem Cell Transplantation, Freiburg, Germany. These new data from REACH2 showing superiority of ruxolitinib over current standard-of-care therapies add to a growing body of evidence on how targeting the JAK pathway can be an effective strategy in this difficult-to-treat condition.
In 2019, Jakafi (ruxolitinib) was approved by the U.S. Food and Drug Administration for the treatment of steroid-refractory acute GVHD in adult and pediatric patients 12 years and older, based on the positive results of the Phase 2 REACH1 trial6. The Phase 3 REACH3 study in patients with steroid-refractory chronic GVHD is ongoing and results are expected in the second half of this year. Jakafi is marketed by Incyte in the U.S.; ruxolitinib (Jakavi) is licensed to Novartis ex-U.S.
The NEJM publication of the REACH2 results is available online.
About REACH2
REACH2 (NCT02913261), a randomized, open-label, multicenter Phase 3 study sponsored by Novartis and conducted in collaboration with and co-funded by Incyte , is evaluating the safety and efficacy of ruxolitinib compared with best available therapy in patients with steroid-refractory acute GVHD.
The primary endpoint was overall response rate (ORR) at Day 28, defined as the proportion of patients demonstrating a best overall response (complete response or partial response). Secondary endpoints include durable ORR at Day 56, ORR at Day 14, duration of response, overall survival and event-free survival, among others. For more information about the study, please visit https://clinicaltrials.gov/ct2/show/NCT02913261.
About REACH
The REACH clinical trial program evaluating ruxolitinib in patients with steroid-refractory GVHD, includes the randomized pivotal Phase 3 REACH2 and REACH3 trials, conducted in collaboration with Novartis. The ongoing REACH3 trial is evaluating patients with steroid-refractory chronic GVHD with results expected later this year. For more information about the REACH3 study, please visit https://clinicaltrials.gov/ct2/show/NCT03112603.
The REACH program was initiated with the Incyte-sponsored REACH1 trial, a prospective, open-label, single-cohort, multicenter, pivotal Phase 2 trial (NCT02953678) evaluating Jakafi in combination with corticosteroids in patients with steroid-refractory grade II-IV acute GVHD. For more information about the study, including trial results, please visit https://clinicaltrials.gov/show/NCT02953678.
About Jakafi (ruxolitinib)
Jakafi is a first-in-class JAK1/JAK2 inhibitor approved by the U.S. FDA for the treatment of polycythemia vera (PV) in adults who have had an inadequate response to or are intolerant of hydroxyurea, in adults with intermediate or high-risk myelofibrosis (MF), including primary MF, post-polycythemia vera MF and post-essential thrombocythemia MF and for the treatment of steroid-refractory acute GVHD in adult and pediatric patients 12 years and older.
Jakafi is marketed by Incyte in the United States and by Novartis as Jakavi (ruxolitinib) outside the United States. Jakafi is a registered trademark of Incyte Corporation. Jakavi is a registered trademark of Novartis AG in countries outside the United States.
Important Safety Information
Jakafi can cause serious side effects, including:
Low blood counts: Jakafi (ruxolitinib) may cause your platelet, red blood cell, or white blood cell counts to be lowered. If you develop bleeding, stop taking Jakafi and call your healthcare provider. Your healthcare provider will perform blood tests to check your blood counts before you start Jakafi and regularly during your treatment. Your healthcare provider may change your dose of Jakafi or stop your treatment based on the results of your blood tests. Tell your healthcare provider right away if you develop or have worsening symptoms such as unusual bleeding, bruising, tiredness, shortness of breath, or a fever.
Infection: You may be at risk for developing a serious infection during treatment with Jakafi. Tell your healthcare provider if you develop any of the following symptoms of infection: chills, nausea, vomiting, aches, weakness, fever, painful skin rash or blisters.
Skin cancers: Some people who take Jakafi have developed certain types of non-melanoma skin cancers. Tell your healthcare provider if you develop any new or changing skin lesions.
Increases in cholesterol: You may have changes in your blood cholesterol levels. Your healthcare provider will do blood tests to check your cholesterol levels during your treatment with Jakafi.
The most common side effects of Jakafi include: for certain types of MF and PV low platelet or low red blood cell counts, bruising, dizziness, headache, and diarrhea; and for acute GVHD low platelet, red or white blood cell counts, infections, and fluid retention.
These are not all the possible side effects of Jakafi. Ask your pharmacist or healthcare provider for more information. Tell your healthcare provider about any side effect that bothers you or that does not go away.
Before taking Jakafi, tell your healthcare provider about: all the medications, vitamins, and herbal supplements you are taking and all your medical conditions, including if you have an infection, have or had tuberculosis (TB), or have been in close contact with someone who has TB, have or had hepatitis B, have or had liver or kidney problems, are on dialysis, have a high level of fat in your blood (high blood cholesterol or triglycerides), had skin cancer or have any other medical condition. Take Jakafi exactly as your healthcare provider tells you. Do not change or stop taking Jakafi without first talking to your healthcare provider.
Women should not take Jakafi while pregnant or planning to become pregnant. Do not breast-feed during treatment with Jakafi and for 2 weeks after the final dose.
Full Prescribing Information, which includes a more complete discussion of the risks associated with Jakafi, is available at http://www.jakafi.com.
About Incyte
Incyte is a Wilmington, Delaware-based, global biopharmaceutical company focused on finding solutions for serious unmet medical needs through the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit Incyte.com and follow @Incyte.
Forward-Looking Statements
Except for the historical information set forth herein, the matters set forth in this press release, including statements about the REACH2 data, when results from the REACH3 study will be available, the effect of the REACH2 results on patients with GVHD, and the overall REACH program, contain predictions, estimates and other forward-looking statements.
These forward-looking statements are based on the Companys current expectations and subject to risks and uncertainties that may cause actual results to differ materially, including unanticipated developments in and risks related to: unanticipated delays; further research and development and the results of clinical trials possibly being unsuccessful or insufficient to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials; determinations made by the FDA; the Companys dependence on its relationships with its collaboration partners; the efficacy or safety of the Companys products and the products of the Companys collaboration partners; the acceptance of the Companys products and the products of the Companys collaboration partners in the marketplace; market competition; sales, marketing, manufacturing and distribution requirements; greater than expected expenses; expenses relating to litigation or strategic activities; and other risks detailed from time to time in the Companys reports filed with the Securities and Exchange Commission, including its Form 10-K for the year ended December 31, 2019. The Company disclaims any intent or obligation to update these forward-looking statements.
References
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Pivotal REACH2 Study Data Published in NEJM Highlight Superior Efficacy of Ruxolitinib versus Best Available Therapy in Patients with Acute GVHD -...
4 Benefits of Stem Cell Therapy that you will Love to Discuss in Future – The Crypto Coin Discovery
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The benefits of Stem cells therapy vary, some of the benefits of stem cells are because of their potential in the treatment of medical conditions, but it is not the only way they can be utilized. A few of the benefits are as follows: To know what is a Stem Cell Therapy.
Medically, stem cells are used in therapies to treat certain diseases and conditions, many treatments require a transplant of organs and tissues but the organs and tissues are donated and the waiting list for donation is quite long. People stay on the transplant lists for years and many dont live to see their name move up the list or are not viable to receive donor organs or tissues, and in such cases stem cells can provide an effective and faster and better alternative in some diseases. The most popular form of stem cell therapy is the bone marrow transplant, and many others are considered safe in treatments of conditions and diseases like:
There are many other stem cell therapies but not all of them have been approved as safe and effective. But like in the case of a burn victim, the replacement of the burned area with stem cells can be utilized to make new tissues and save the trouble of finding a tissue donor. It is a very painful experience and everything that can be done should be done to lessen the pain and start the healing process quickly for the sake of the patient.
Research done on animals like dogs and horses and cats showed us that this form of research not only advances the development of stem cell therapies and treatments; for the benefit of veterinary medicine, but also resulted to be very beneficial for human treatments as well.
Animals that have diseases that nearly mimic the ones humans have as well are used as ideal models to experiment the development of stem cell therapies in medical conditions that humans and animals both have, like ligament injuries or stroke etc.
Diseases like cancer and conditions like birth defects are quite common these days so, many clinical and experimental trials are popping up to better fight these diseases. Scientists are now looking for ways that they can use to come up with stem cell treatments to better develop the human body when it is suffering from conditions like birth defects.
They are trying to study how the stem cells transform or separate into a wide range of specialized cells so that they can be utilize them in the treatment of certain diseases and conditions.
Stem cells have a huge potential in the testing of drugs as they are relatively safe and dont put anyone in harms way, drugs are now first tested on stem cells and then on animals and humans if the test on stem cells goes well.
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4 Benefits of Stem Cell Therapy that you will Love to Discuss in Future - The Crypto Coin Discovery
With Over 280 Therapies Under Evaluation, the Stem Cell Therapy Market is Estimated to be Worth USD 8.5 Billion by 2030, Claims Roots Analysis – PR…
The success of approved stem cell therapies has caused a surge in interest of biopharma developers in this field; many innovator companies are currently progressing proprietary leads across different phases of clinical development, with cautious optimism
LONDON, March 4, 2020 /PRNewswire/ -- Roots Analysishas announced the addition of "Global Stem Cells Market: Focus on Clinical Therapies, 20202030 (Based on Source (Allogeneic, Autologous); Origin (Adult, Embryonic); Type (Hematopoietic, Mesenchymal, Progenitor); Lineage (Amniotic Fluid, Adipose Tissue, Bone Marrow, Cardiosphere, Chondrocytes, Corneal Tissue, Cord Blood, Dental Pulp, Neural Tissue Placenta, Peripheral Blood, Stromal Cells); and Potency (Multipotent, Pluripotent))" report to its list of offerings.
There is a growing body of evidence supporting the vast applicability and superiority of treatment outcomes of stem cell therapies, compared to conventional treatment options. In fact, the unmet needs within this domain have spurred the establishment of many start-ups in recent years.
To order this 500+ page report, which features 185+ figures and 220+ tables, please visit this link
Key Market Insights
Over 280 stem cell therapies are under development, most of which are allogeneic products
More than 50% of the pipeline candidates are in the mid to late phase trials (phase II and above), and allogenic therapies (majority of which are derived from the bone marrow) make up 65% of the pipeline.
70% of pipeline candidates are based on mesenchymal stem cells
It is worth highlighting that the abovementioned therapies are designed to treat musculoskeletal (22%), neurological (21%) and cardiovascular (15%) disorders. On the other hand, hematopoietic stem cell-based products are mostly being evaluated for the treatment of oncological disorders, primarily hematological malignancies.
Close to 85% stem cell therapy developers are based in North America and Asia-Pacific regions
Within these regions, the US, China, South Korea and Japan, have emerged as key R&D hubs for stem cell therapies. It is worth noting that majority of the initiatives in this domain are driven by small / mid-sized companies
Over 1,500 grants were awarded for stem cell research, since 2015
More than 45% of the total amount was awarded under the R01 mechanism (which supports research projects). The NCI, NHLBI, NICHD, NIDDK, NIGMS and OD emerged as key organizations that have offered financial support for time periods exceeding 25 years as well.
Outsourcing has become indispensable to R&D and manufacturing activity in this domain
Presently, more than 80 industry / non-industry players, based in different regions across the globe, claim to provide contract development and manufacturing services to cater to the unmet needs of therapy developers. Examples include (in alphabetical order) Bio Elpida, Cell and Gene Therapy Catapult, Cell Tech Pharmed, GenCure, KBI Biopharma, Lonza, MEDINET, Nikon CeLL innovation, Roslin Cell Therapies, WuXi Advanced Therapies and YposKesi.
North America and Asia-Pacific markets are anticipated to capture over 80% share by 2030
The stem cell therapies market is anticipated to witness an annualized growth rate of over 30% during the next decade. Interestingly, the market in China / broader Asia-Pacific region is anticipated to grow at a relatively faster rate.
To request a sample copy / brochure of this report, please visit this link
Key Questions Answered
The USD 8.5 billion (by 2030) financial opportunity within the stem cell therapies market has been analyzed across the following segments:
The report features inputs from eminent industry stakeholders, according to whom stem cell therapies are currently considered to be a promising alternatives for the treatment of a myriad of disease indications, with the potential to overcome challenges associated with conventional treatment options. The report includes detailed transcripts of discussions held with the following experts:
The research covers brief profiles of several companies (including those listed below); each profile features an overview of the company, financial information (if available), stem cell therapy portfolio and an informed future outlook.
For additional details, please visit
https://www.rootsanalysis.com/reports/view_document/stem-cells-market/296.html email sales@rootsanalysis.com
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Contact:Gaurav Chaudhary+1(415)800-3415+44(122)391-1091Gaurav.Chaudhary@rootsanalysis.com
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With Over 280 Therapies Under Evaluation, the Stem Cell Therapy Market is Estimated to be Worth USD 8.5 Billion by 2030, Claims Roots Analysis - PR...
Stem cell activity linked to lifestyle – Harvard Magazine
Sleep, diet, exercise, and stress: these are factors known to change a persons risk of developing numerous non-communicable diseases. Such lifestyle impacts on healthbeneficial or harmfulexert much of their influence via inflammation. About 10 years ago, Matthias Nahrendorf began wondering just how inflammation and lifestyle might be linked biologically, and started thinking about how to pinpoint the mechanism in the cardinal case of cardiovascular disease.
A persons level of inflammation can easily be measured with a simple white blood cell test. White blood cells fight off bacterial invasions and repair damaged tissues, but they can also damage healthy tissue when they become too abundant. You can find them in atherosclerotic plaques, and you can find them in acute infarcts, says Nahrendorf, a professor of radiology who conducts high-resolution imaging research at Massachusetts General Hospital. You can find them in failing hearts and the brain, where they increase the risk of stroke.
By linking exercise to reduced white blood cell production, Nahrendorf shows how a lifestyle factor can modulate cardiovascular risk.
When Nahrendorf learned that the most potent, toxic, and pro-inflammatory white blood cells live only a few hours, or at most a day, he immediately realized that the paramount questionsgiven that they die off quickly yet remain abundant in the bloodare, where and why are they produced? What is their source? Perhaps, he hypothesized, lifestyle factors regulate hematopoiesis (blood production).
To test this idea, he decided to study the effects of exercise on the production of these leukocytes in healthy mice. First, though, he consulted the scientific literature on exercise in mice. Previous researchers, he learned, had found that exercise increases production of inflammatory immune cellswhich I thought was counterintuitive, Nahrendorf recalls. When he looked more carefully, he discovered that the type of exercise used in the studies was forced and thus possibly stressful because it was induced by electric shocks. He therefore decided to test only voluntary exercise. He and his colleagues put a wheel in each mouses cage, so the animals could choose to run if they were interested.
The mice never ran during the day. That is when they rest, Nahrendorf explains. But in the dark, they ran a lot, averaging six to seven miles every night. After three weeks, the exercising mice had measurably lower levels of circulating white blood cells. Exercise, he found, had pushed their blood stem cells (cells that can produce all the different types of blood cells) into a state of quiescence: a kind of dormancy in which they generate fewer pro-inflammatory white blood cells and platelets, without decreasing the number of oxygen-carrying red blood cells. Soon the exercising mice had fewer circulating white blood cells than their sedentary counterparts, dampening inflammationan effect that persisted for weeks.
The local signals within bone marrow that induce quiescence in blood stem cells were already well known, but the fact that exercise could trigger them was not. Nahrendorf next wanted to learn the identity of the trigger linking exercise to blood stem cell quiescence. Further investigation revealed that the only receptors with enhanced activity in the bone marrow niche where most blood stem cells exist were binding to a well-known hormone called leptin; it is produced by fat cells and regulates hunger.
Leptin is like the fuel gauge in a car. When the tank is fullmeaning energy (and food) are abundantleptin levels run high. As exercise uses up the gas in the tank, this lowers leptin levels, which signal that reserves are running low, thereby inducing hunger and the urge to eat in order to replenish depleted energy stores. Nahrendorf and his co-authors speculate in their 2019 Nature Medicine paper that leptins role in regulating energetically costly hematopoiesis may have evolved to produce blood cells only when whole body energy was abundantnot when people are exerting themselves. Contemporary sedentary behavior, they continue, which increases leptin and consequently hematopoiesis, may have rendered this adaptation a risk factor for cardiovascular disease (CVD) and perhaps also for other diseases with inflammatory components.
But with fewer circulating immune cells, would exercising mice be more vulnerable to infection? Nahrendorf challenged them with a protocol designed to induce infection in the blood, and found just the opposite: exercising mice had a more robust immune response, as semi-dormant blood stem cells swiftly sprang into activity and produced infection-fighting leukocytes, improving survival of the active mice as compared to those with no running wheels in their cages. Next, they investigated whether exercise would help mice with established atherosclerosis, and found that exercise was not only protective, it also reduced the size of existing plaques in the aorta.
Whether these associations would hold up in humans remained an open question. For answers, Nahrendorf turned to a study known as CANTOS, which had measured levels of inflammation in 4,892 patients who suffered heart attacks (see Raw and Red Hot, May-June 2019, page 46). When he approached the studys co-authors, Mallinckrodt professor of medicine Peter Libby and Braunwald professor of medicine Paul Ridker, he learned, serendipitously, not only that they possessed self-reported exercise levels for the participants, but also that they had tested leptin levels as well. They analyzed their raw data and found the same relationship among exercise, leptin, and leukocytes as in the mice. Data from a second human study cemented the result.
By identifying a previously unknown molecular mechanism linking voluntary exercise to reduced white blood cell production, Nahrendorf and his colleagues have highlighted how a lifestyle factor can modulate cardiovascular risk. Their discovery, the researchers hope, will point the way to wider adoption of healthy exercise regimens, and health-enhancing anti-inflammatory drugs.
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Stem cell activity linked to lifestyle - Harvard Magazine
Coronavirus: We need bone marrow donors to save my baby girls life from leukaemia, Hong Kong mother of two-year-old pleads amid London lockdown -…
Two-year-old Livia, the child of a Hong Kong mother and German father, is among patients in a London hospital as Britain grapples with a lockdown wrought by the coronavirus. But she is fighting a disease much rarer and even deadlier.
Livia was diagnosed in early March with acute myeloid leukaemia (AML). Because of the coronavirus pandemic, visits to her bedside have been restricted to reduce infection risks.
Only her father can stay with her. Her aunt and grandparents in Hong Kong cannot travel there for fear of exposing the child to more health risks.
Mother Olive Yu, who has been trying every means possible to save her daughter with access to donor registrations delayed during the lockdown told the Post in a phone interview: I still think that I'm in a really bad dream. I still find it very hard to accept that this is happening. Livia is everything to us because shes the only grandchild in the family, and our only child.
We need to be thinking about how we can help give her [as much time] as she can get
Olive Yu, mother
The family has called on those aged between 18 and 60 in Hong Kong to register as a bone marrow donor with the Hong Kong Bone Marrow Donor Registry, led by the Hong Kong Red Cross. The information is shared with the World Marrow Donor Association (WMDA), a global database of volunteer donors.
There are three options to save Livia, and the most ideal one is for a matching donor to be found and a bone marrow transplant to be conducted by June, according to Livias family.
Livias parents are appealing for bone marrow donors. Photo: Handout
Yu said: Everything takes time, especially now with the coronavirus. But the fact is, we are talking about the life of a two-year-old.
Everything is against us, but it doesn't mean that we should just stop and not do anything about it. So were going to try our best and do whatever we can to give her the best chance at life.
As of Wednesday, Britain had recorded more than 93,000 Covid-19 cases, with a death toll of over 12,000. Police enforced a lockdown on March 23, allowing people only to leave their homes for very limited purposes, such as for food and health reasons, and public gatherings of more than two people have been banned, while places such as restaurants, schools, pubs and gyms are closed.
In general, for bone marrow transplants, the donors human leucocyte antigens (HLA), proteins found on the surface of the blood and in tissue cells, must be closely matched so that the recipients body can accept the new stem cells into their bone marrow.
The second option is that the hospital also, at the same time, reaches out to stem cells from mothers who give birth and donate their umbilical cord, Yu added.
The third option is what they call a non-matching donor, which is either from the mother or father.
The blood samples of Livias parents are being analysed for matches, with results pending.
AML is a form of cancer involving the rapid growth of abnormal cells in the bone marrow and blood. This cancer type accounted for less than 1 per cent of all new cancer cases in Britain in 2017, and often occurs in adults, according to Cancer Research UK.
Livia, two, is now warded in a London hospital. Photo: Handout
A haematologist in Hong Kong, who spoke on condition of anonymity, said the incidence of cancer among children aged under 15 is around 1.2 per million. Fewer than 200 children are diagnosed with cancer in Hong Kong each year and about five out of the 200 have AML, according to him.
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Coronavirus: We need bone marrow donors to save my baby girls life from leukaemia, Hong Kong mother of two-year-old pleads amid London lockdown -...
Orphan Drugs Market Insights, 2020: Approval of Biological Orphan Drugs for Multiple Indications is Driving Market Growth – ResearchAndMarkets.com -…
The "Orphan Drugs Market Global Report 2020-30" report has been added to ResearchAndMarkets.com's offering.
The global orphan drugs market was worth $132.61 billion in 2019. North America is expected to be the largest region during the period 2015-2023. Major players in the market are Bristol-Myers Squibb Company, Celgene Corporation, F. Hoffmann-La Roche, Amgen, Biogen, Bayer, Novartis, GlaxoSmithKline, Johnson & Johnson and AbbVie.
This report covers market characteristics, size and growth, segmentation, regional and country breakdowns, competitive landscape, market shares, trends and strategies for this market. It traces the market's historic and forecast market growth by geography. It places the market within the context of the wider orphan drugs market, and compares it with other markets.
The rising prevalence of rare diseases is a key factor driving the growth of the orphan drugs market.
Orphan diseases or rare diseases occur rarely among the people (i.e. 7 out of 10,000). However, globally, the prevalence of rare diseases is increasing in recent years. In 2017, there were 7,000 identified rare diseases, including hemophilia, Gaucher disease, Hunter syndrome and many types of rare cancer. Some cases of aplastic anemia, caused by damage to stem cells in the bone marrow that are diagnosed in around 500 to 1,000 individuals in the USA each year, are inherited. Thus, the rising prevalence of rare diseases is driving the growth of the orphan drugs market.
Lack of supportive government policies hinders the orphan drugs market.
Due to the lack of relevant policies for orphan drug, certain drugs do not receive any special recognition or priorities for approval by regulatory authority. Medgenome Labs Ltd., global research partner in accelerating insights into complex genetic diseases, pointed out that companies manufacturing orphan drugs frequently drop out in foreign markets due to a lack of government funding. For example, orphan medical products (OMPs) in India, due to lack of proper regulations and clear guidelines, do not obtain tax cuts or exemptions from customs duties. Therefore, lack of supportive government policies limits the growth of the orphan drugs market.
Approval of biological orphan drugs for multiple indication act as a key trend driving the growth of the orphan drugs market.
The biological drugs are used for treating rare diseases such as cancer with fewer side effects that have a high prevalence rate in the developed world. For Instance, in 2018, in order to launch the company's biological orphan drug development program Cardax, Inc. announced that it has been engaged with biological orphan drug expert Frederick D. Sancilio, Ph.D. For the development of commercial products, the companies are focused on obtaining biological orphan drugs to increase their revenue.
In November 2019, Bristol-Myers Squibb, a biopharmaceutical company whose mission is to discover, develop, and deliver innovative medicines, acquired Celgene for an undisclosed amount. Through this acquisition, Celgene shareholders received for each share, 1 share of Bristol-Myers Squibb common stock, $50.00 in cash without interest and one tradeable Contingent Value Right (CVR), which will entitle the holder to receive a payment of $9.00 in cash if certain future regulatory milestones are achieved. Celgene, a biopharmaceutical company positioned to address the needs of the patients with serious diseases.
Key Topics Covered
1. Executive Summary
2. Orphan Drugs Market Characteristics
3. Orphan Drugs Market Size and Growth
3.1. Global Orphan Drugs Historic Market, 2015-2019, $ Billion
3.1.1. Drivers Of The Market
3.1.2. Restraints On The Market
3.2. Global Orphan Drugs Forecast Market, 2019-2023F, 2025F, 2030F, $ Billion
3.2.1. Drivers Of The Market
3.2.2. Restraints On the Market
4. Orphan Drugs Market Segmentation
4.1. Global Orphan Drugs Market, Segmentation By Therapy Area, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion
4.2. Global Orphan Drugs Market, Segmentation By Distribution Channel, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion
5. Orphan Drugs Market Regional and Country Analysis
5.1. Global Orphan Drugs Market, Split By Region, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion
Story continues
5.2. Global Orphan Drugs Market, Split By Country, Historic and Forecast, 2015-2019, 2023F, 2025F, 2030F, $ Billion
Companies Mentioned
For more information about this report visit https://www.researchandmarkets.com/r/vm9iey
View source version on businesswire.com: https://www.businesswire.com/news/home/20200417005515/en/
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Orphan Drugs Market Insights, 2020: Approval of Biological Orphan Drugs for Multiple Indications is Driving Market Growth - ResearchAndMarkets.com -...
What exactly are underlying conditions? And why people with them may experience more serious illness from coronavirus – Boston News, Weather, Sports |…
(CNN) Weve heard that elderly people and those with underlying health conditions are most at risk if theyre infected with coronavirus, but those can seem like really general terms. Who does that include? And why can they face more serious illness?
According to the [Centers for Disease Control and Prevention], some of the underlying conditions that may put you at higher risk include: chronic lung disease and asthma, heart disease and undergoing cancer treatment, said CNN Chief Medical Correspondent Dr. Sanjay Gupta in anepisode of CNNs Coronavirus: Fact vs. Fiction podcast. Anyone with diabetes, kidney failure or liver failure may also be at higher risk.
The role of the immune system is to protect against disease or other potentially damaging pathogens. A strong one is needed to help stave off coronavirus infection.
Think of it like this, Dr. Gupta suggested. In your everyday life, youre always fighting off pathogens. Most of the time you dont even realize it. If you have an underlying condition, it makes it more challenging to fight off a virus like this. You may develop a fever, shortness of breath or a cough more easily than someone who doesnt have a preexisting illness.
Additionally, there are more specific reasons why each condition has its own vulnerabilities. Heres a guide to underlying conditions affected by coronavirus and why, and how you can protect yourself or an at-risk loved one.
Eight out of 10 deaths reported in the US have been in adults ages 65 and older, according to theCDC. Older adults have also been more likely to require hospitalization and admission to an intensive care unit.
Older adults are more likely to have long-term health problems that can increase their risk for infection and serious disease. And, our immune systems usually weaken with age, making it more difficult for people to fight off infections, according toJohns Hopkins Medicine.
The quality of our lung tissue also declines over time, becoming more elastic and making respiratory diseases such as Covid-19 of important concern because of the potential for lung damage.
Inflammation in older adults can be more intense, leading to organ damage.
People with chronic airway and lung diseases such as chronic obstructive pulmonary disease (COPD), asthma, pulmonary fibrosis and interstitial lung disease can lay the foundations for more severe infection with coronavirus because of the inflammation, scarring and lung damage those conditions cause,Johns Hopkins Medicine reported.
Covid-19 affects a persons airway and lungs, but those organs work together to provide the body with oxygen. When the lungs are overburdened with an infection, the heart has to work harder, which exacerbates the challenges of people already living with heart disease.
According to the CDC, many conditions can cause a person to be immunocompromised, including cancer treatment, smoking, bone marrow or organ transplantation and immune deficiencies. Poorly controlled HIV or AIDS and prolonged use of man-made steroid hormones or otherimmune-weakening medicationscan also hamper a persons immune function.
Cancer can weaken immunity by spreading into the bone marrow, which makes blood cells that help fight infection, according toCancer Research UK. Cancer prevents bone marrow from making enough blood cells.
Some cancer treatments can temporarily weaken the immune system, too. Because cancer treatments such as chemotherapy, cancer drugs, radiotherapy or steroids are targeted toward cancer cells, they can also diminish the number of white blood cells created in the bone marrow.
A2017 studyfound cigarette smoking can harm the immune system by either causing extreme immune responses to pathogens or rendering the body less effective at fighting disease. This may occur by smoking, negatively altering the cellular and molecular mechanisms responsible for keeping an immune system strong.
When a person undergoes a bone marrow transplant using stem cells from a donor, or they receive an organ, a doctor may prescribe medications to prevent graft-versus-host disease andmitigate the immune systems reactionby suppressing its function. After the operation, it takes time for your immune system to be up and running again.
HIV and AIDS attack the bodys immune system, specifically the bodys T cells, which help the immune system fight off infection. When the diseases are untreated, HIV reduces the number of those cells, making the person more likely to contract other infections or infection-related cancer, according to theCDC.
People with severe obesity, or a body mass index of 40 or higher, are athigher risk of serious disease.
Obesity shares with most chronic diseases the presence of an inflammatory component, a2012 studysaid. Inflammatory responses were linked between the immune system and body fat. Obesity is known to impair immune function by altering white blood cell count as well as the cells that control immune responses.
People with type 1 or type 2 diabetes face an increased risk of getting really sick with Covid-19, as both cause a blood sugar spike. If blood sugar is poorly managed, viral diseases can be more dangerous as high blood sugar may give viruses a place to thrive, according toDiabetes in Control, a news and information resource for medical professionals.
Higherlevels of inflammationhave been discovered in the bodies of people with diabetes, weakening the immune system and making it more difficult for those affected to stave off sickness in general.
The kidneysproduce several hormonesthat affect immune responses. Having kidney disease and failure can weaken your immune system, making it easier for infections to take hold. According to theNational Kidney Foundation, doctors and researchers have found that most infections are worse in people with kidney disease.
The liver is an integral member of the bodysline of defense, helping to regulate the number of white blood cells utilized in immune responses and defend against harmful pathogens. Someone with liver disease is experiencing abnormalities in the function of the immune system, giving rise to more serious illness.
Neurological and neurodevelopmental conditions may also increase the risk of serious Covid-19 for people of any age.
These include disorders of the brain, spinal cord, peripheral nerve and muscle such as cerebral palsy, epilepsy, stroke and intellectual disability, according to theCDC. Those with moderate to severe developmental delay, muscular dystrophy or spinal cord injury are also more at-risk.
People with neurological conditions may not be more at risk due to solely their condition, but because medications they might take to control their condition could hamper their immune system. However, some neurological conditions, such as Parkinsons, have been recognized to haveinflammatory components, which may harm the immune system.
Others including muscular dystrophy, multiple sclerosis or amyotrophic lateral sclerosis (ALS) could cause paralysis to the diaphragm, which leaves those affected very at risk for respiratory failure if they were to be sick with Covid-19.
If you see yourself on the list of those at higher risk for severe illness, there are several things you can do to protect yourself. First, make sure you are contact your doctor or doctors about your risk level. Second, be extra vigilant about the recommendations that most people are being asked to follow.
Stay home whenever possible and avoid close contact with people, theCDC suggests. Wash your hands often to prevent transferring the virus from a surface to your face, and try to clean and disinfect frequently touched surfaces as often as you can.
If you dont have an underlying condition, doing your part by practicing these cautionary measures can help protect not only you, but your loved ones with existing conditions.
Click here for more coronavirus coverage.
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A Realist Who Believes in Miracles – Thrive Global
In 1994, I thought the biggest challenge in life would be finding a balance between a career as a Senior Trial Partner at a big Dallas firm and raising three children. In 1999, it became clear that the real challenge in my life was much more ominous. I was diagnosed with Multiple Sclerosis. There was no explanation as to why I contracted this devastating disease, what symptoms I would develop, or how fast I would become disabled. Would I be confined to a wheelchair or a bed? Would I become blind or simply have double vision? Would I have pain or just tingling? Would I die? I already had bladder problems, but would I also face bowel dysfunction? Over 2.5 million people are afflicted with MS, so why hasnt anyone found a cure? How could drug companies justify charging over $60,000 a year for medicines that dont improve a patients condition?
For the next fifteen years, I managed the kids and the disease with relative success. I learned what a bladder spasm is and the true definition of the word urgency. I learned that my husband really meant it when he said in sickness and health. I learned that pain from MS included the pain associated with doing a face plant into a door, and spilling boiling water on my leg but being unable to remove my pants before suffering 2nd degree burns. I learned that there were a few advantages to having MS, including speeding through airport security lines because I was in a wheelchair, and always being able to find a parking space.
In 2014 I fell and broke my leg. I was in a wheelchair for 6 weeks. It became obvious that it was time to become more aggressive with treatments. After scouring the internet for every treatment for MS in the world, I identified Dr. Dimitrios Karussis at Hadassah Medical Organization in Israel as my best hope. His approach was still experimental. He used the patients own stem cells, obtained through bone marrow extraction, grew the cells, and then infused the cells through a spinal tap.
After eight infusions the benefits of the treatment are unmistakable. Because I still walk with a walker, people realize that Im not cured. What they dont know is that I have my life back. Ive written three books, volunteer regularly at a hospital, travel around the country to raise awareness and financial support for the incredible work of Hadassah, the Womens Zionist Organization of America, Inc. (HWZOA) who operate Hadassah Hospital in Israel. I cook every week for my daughter in medical school. I have attended the graduation ceremonies of each of my three children from college, and I attended the wedding of my oldest son recently. This past year, we celebrated Thanksgiving at my house with 37 relatives.
Having MS has allowed me to stop sweating the small stuff. I have come to realize that what makes me happiest is making others happy. At the Dallas Childrens Hospital where I volunteer, my disability gives me the advantage of having an immediate connection to the kids. Making people smile is the best job at the hospital.
David Ben Gurion said: In Israel, in order to be a realist, you must believe in miracles.
I am a realist. I didnt simply wish to be cured of MS. I researched the possible options for treatment and used my best judgment to select one. Dr. Karussis is also a realist. Hes devoted over 30 years researching stem cell treatment of neurological diseases. He has published more than 120 peer reviewed scientific papers, given more than 150 lectures, served on editorial boards of major medical journals, was elected as the President of the Israeli Neuroimmunological Society and hosted an International Neurological Meeting. He has published the amazing results of the stem cell therapy he formulated for the treatment of MS and ALS.
I also believe in miracles. The miracle is that the people of Hadassah Hospital in Israel have given of their time, talent, and money to make this treatment possible and available to me. The miracle is that studies that I volunteered for twenty years ago in Dallas made me an attractive candidate for Dr. Karussis research. The miracle is that the Israeli Ministry of Health approved me to be treated in their Compassionate Care program. The miracle is that all MS patients can now have hope that an effective treatment is here and Hadassah Hospital is sharing it with the world.
VIDEO LINKS:
Watch MS patient Malia Litman dance at her sons wedding
Learn how MS patient Malia Litman got her life back
Learn more about Hadassah Medical Organization
Learn more about Dr. Dimitrios Karussis and his revolutionary stem cell treatment
CBS/Dallas News coverage of Malia Litmans MS treatment
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A Realist Who Believes in Miracles - Thrive Global
You’re my type: The donor lottery – RT
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You're my type: The donor lottery - RT
Horizon Research Fellow job with UNIVERSITY OF LEEDS | 203439 – Times Higher Education (THE)
Would you like to be involved in cutting edge research to develop novel solutions for smart resorbable bone implants for controllable and fast bone restoration, and to testa new generation acellular scaffold with bone-like architecture? Are you interested in acquiring and/or developing your skills to develop new tissue engineering solutions for in vitro reprogramming of adult stem cells for embedding into the smart implant?
Applications are invited for a Postdoctoral Fellow position working with Prof Peter Giannoudis and Dr Elena Jones on the Horizion 2020project SBR Smart Resorbable Bone and a small industry funded project Greenbone. Based on the St. James Campus, you will co-ordinate projects related to several SBR Work Packages (WPs).
More specifically you will be working on the WPs 6 with the aims to:
You will also be responsible for delivering Greenbone project to assess the biological support that GreenBone scaffold can provide for MSCs and endothelial cells.
Reporting to Dr Jones, your work will be carried out in collaboration with consortium members based in Patras, Greece and Munich, Germany.
Capitalising on the infrastructure and samples collected through the Leeds Institute of Rheumatic and Musculoskeletal medicine based across St Jamess University Hospital and LGI, you will generate primary MSC cells to be used by other consortium partners. You will develop and validate assays to assess the behaviour of MSCs on electrospun biocompatible scaffolds, as well as optimise the MSCs uptake of several bioactive DDRs. In collaboration with partners in Greece and Germany, you will also develop and validate assays for the assessment of MSC functionality on bioactive implants. In Greenbone project, you will be testing the attachment and growth of human bone marrow cells onGreenBone scaffold, as well as the endothelial cell-MSC interactions on the scaffold surface.
The post holder will also liaise with the research group, specifically dedicated to this project, and based in the School of Chemical and Process Engineering, as required.
To explore the post further or for any enquiries you may have, please contact:
Dr Elena Jones, Associate Professor (Non-Clinical)
Tel: +44 (0)113 2065647; email:e.jones@leeds.ac.uk
OR
Professor Peter Giannoudis, Professor of Orthopaedic Surgery
Tel + 44 (0) 113 0113-20-67068; email:P.Giannoudis@leeds.ac.uk.
Further Information
The University of Leeds is committed to providing equal opportunities for all and offers a range offamily friendly policies. The University is a charter member of Athena SWAN and holds the Bronzeaward. The School of Medicine holds theGoldaward. We are committed to being an inclusive medical school that values all staff, and we are happy to consider job share applications and requests for flexible working arrangements from our employees.
Location:Leeds - St James University HospitalFaculty/Service:Faculty of Medicine & HealthSchool/Institute:Leeds Institute of Rheumatic & Musculoskeletal Medicine (LIRMM)Section:Section of Experimental RheumatologyCategory:ResearchGrade:Grade 7Salary:33,797 to 40,322 p.a.Working Time:100% FTEPost Type:Full TimeContractType:Fixed Term (Available immediately on a fixed term basis for 27 months or until 31 December 2023 (wichever is sooner))ClosingDate:Tuesday 21 April 2020Reference:MHLRM1130Downloads:CandidateBrief
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Horizon Research Fellow job with UNIVERSITY OF LEEDS | 203439 - Times Higher Education (THE)
Gracell to Present the First-in-human, Universal TruUCAR GC027 Therapy for Relapsed or Refractory T-cell Acute Lymphoblastic Leukemia at the AACR…
SUZHOU, Chinaand SHANGHAI, April 13, 2020 /PRNewswire/ -- Gracell Biotechnologies Co., Ltd. ("Gracell"), a clinical-stage immune cell and gene therapy company, is pleased to announce that their first-in-human phase I data of Universal TruUCAR GC027 in relapsed or refractory (R/R) T-cell acute lymphoblastic leukemia (T-ALL) was accepted for plenary oral presentation at the America Association for Cancer Research (AACR) Annual Meeting.
Gracell Logo (PRNewsfoto/Gracell)
This year's AACR presentations are moved to be held virtually to allow sharing the data in a timely fashion. A series of online sessions featuring presentations will be provided. Gracell will report the clinical safety and efficacy of GC027, an off-the-shelf CAR-T product based on Gracell's TruUCAR technology, for treatment of adult T-ALL patients.
"We are very pleased that AACR has accepted the phase I results of GC027, a first-in-human off-the-shelf TruUCAR product for plenary oral presentation. Gracell's proprietary TruUCAR platform was protected with patents of novel designs and unique features. Remarkably, GC027 derived from HLA unmatched donor's cells, is a monotherapy without co-administration of other immunosuppressive drug." said Dr. William CAO, founder and CEO of Gracell. "We are pleased to share thefirst-in-human phase I data with the scientific community."
Presentation: Safety and efficacy clinical study of TruUCART GC027: the first-in-human, universal CAR-T therapy for relapsed/refractory T-cell acute lymphoblastic leukemiaAbstract #9564Online live section: Apr. 27-28, EDT
About GC027GC027 was manufactured fromT cells of human leukocyte antigen (HLA) unmatched healthy donors using TruUCAR technology. TruUCAR allows the allogeneic CAR-T cells to proliferate and persist in HLA-unmatched patients (recipients) with minimized risk of graft-versus-host-disease (GvHD). GC027 is currently being developed as an investigational, off-the-shelf CAR-T cell therapy for treatment of T cell malignancies. The use of HLA unmatched healthy donor's cells may improve efficacy and reduce production time, available for off-the-shelf use in a timely manner.
About TruUCARTruUCAR is Gracell's proprietary and patented platform technology, with selected genes being edited to avoid GvHD and immune rejection without using strong immunosuppressive drugs. In addition to T-ALL antigen, the platform technology can also be implemented for other targets of hematological malignancies.
About T-ALLT - Lymphoblastic Leukemia (T-ALL) is an aggressive form of acute lymphoblastic leukemia, with a diffuse invasion of bone marrow and peripheral blood. In 2015, T-ALL affected around 876,000 people globally and resulted in 110,000 deaths worldwide. T-ALL compromises about 15%-20% of all children and adult acute lymphoblastic leukemia[1].Current standard of care therapies for T-ALL are chemotherapy and stem cell transplantation. 40-50% of patients will experience relapse within two years following front line therapy with limited treatment options available[2] [3]. Treatment of relapsed and refractory T-ALL remains a high unmet medical need.
About GracellGracell Biotechnologies Co., Ltd. ("Gracell") is a clinical-stage biotech company, committed to developing highly reliable and affordable cell gene therapies for cancer. Gracell is dedicated to resolving the remaining challenges in CAR-T, such as high production costs, lengthy manufacturing process, lack of off-the-shelf products, and inefficacy against solid tumors. Led by a group of world-class scientists, Gracell is advancing FasTCAR, TruUCAR (off-the-shelf CAR), Dual CAR and Enhanced CAR-T cell therapies for leukemia, lymphoma, myeloma, and solid tumors.
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Gracell to Present the First-in-human, Universal TruUCAR GC027 Therapy for Relapsed or Refractory T-cell Acute Lymphoblastic Leukemia at the AACR...
Stem cell therapies slowly gain traction as viable treatments for brain disorders – Science Magazine
Jack PriceMIT Press2020288 pp.Purchase this item now
In the late 19th century, the Spanish neuroanatomist Santiago Ramn y Cajal documented, in exquisite detail, the fantastical, uncharted landscapes of the human brain. The ornate cellular structures he drew were, according to Cajal, fragile and irreplaceable. Brain cells, he stated, may die and cannot be regenerated. Cajal then threw down the gauntlet, asserting that it was the job of the science of the future to change, if possible, this harsh decree.
Jack Prices engaging bookThe Future of Brain Repairdetails past, present, and future attempts to address Cajals formidable challenge. In so doing, it provides a vibrant and compelling guide to the important and rapidly evolving fields of stem cellbased therapies and brain repair, which together, he believes, are poised to deliver unprecedented changes to the management of brain diseases.
Unlike the diverse blood cells generated throughout life by specialized stem cells in bone marrow, the two known brain stem cell typestucked into the underside of the dentate gyrus of the hippocampus and surrounding the ventricles of the forebrain in the subependymal zonedifferentiate into a much more restricted set of cells. This intrinsic lack of versatility, coupled with the fact that brain cells are postmitotic and consequently unable to divide, underlies the brains inability to efficiently repair itself.
Two broad strategies for repair are suggested. The first would be to bypass endogenous neural stem cells by introducing non-native brain cells. The second would be to coax native cells into a different set of behaviors.
Parkinsons disease, a progressive movement disorder caused by the incremental destruction of dopaminergic neurons, has provided a fertile testing ground for the first strategy. The first human neural transplantation experiments conducted during the 1980s, which used dopamine-producing cells from human fetal brain cells or from patients own adrenal medullas, failed to meet expectations. It appeared as if not any old cells would do. Although a proof of concept for the feasibility of such approaches was provided, a more precisely defined and renewable source of donor cells was lacking, as was an accompanying robust and affordable commercial-scale manufacturing process.
In the 1990s, researchers showed that neural progenitor cells could be cultured as neurospheresballs of cells that maintain self-replication and multipotency over protracted durations. This discovery led, in 2006, to the first human neural stem cell transplant in a group of children with Batten disease, a rare neurodegenerative disorder. The results, again, were disappointing: the overall survival of treated patients showed no discernible improvement over that of untreated patients. Similar studies in patients with ischemic stroke have proved more promising, but researchers suspect improvements in these cases resulted from the grafts secretion of neuroprotective proteins rather than from cell replacement.
Two other innovations, however, have the potential to address Cajals regenerative vision. The first is our ability to culture embryonic stem cells (ES cells) to produce billions of pluripotent stem cells, which are, in turn, capable of producing every cell type in the human body. Therapies derived from ES cells have been shown to be highly effective in animal models of Parkinsons disease, with evidence suggesting that the transplanted neurons exert their effect by synthesizing dopamine.
The second innovation issues from the work of Shinya Yamanaka, who demonstrated in 2006 that terminally differentiated cells of any type can be reprogrammed into induced pluripotent stem cells (iPSCs) through the administration of just four transcription factors. iPSCs may then be converted into select cell types using different transcription factor cocktails.
Although not without complicationsextended culture of iPSCs has been shown, for example, to result in mutations in P53 and other oncogenesES cells and iPSCs have the potential to transform the science of brain repair and regenerative medicine by enabling the generation and therapeutic deployment of relevant neuronal subtypes in a scalable and low-cost manner. These cells have the additional advantage of retaining the capacity to build new tissues from scratch.
Perhaps most interesting, however, is the recent convergence of pluripotent stem cells with gene editing. Together, these technologies offer the possibility of augmenting natural neural stem cell behavior.
ACKNOWLEDGMENTSThe reviewer is a shareholder of Sangamo Therapeutics.
The reviewer is the executive vice president of research and development at Sangamo Therapeutics, Brisbane, CA, USA.
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Stem cell therapies slowly gain traction as viable treatments for brain disorders - Science Magazine
NantKwest and ImmunityBio Announce Therapeutics and Vaccines for Combatting COVID-19; Clinical Trials Anticipated to Begin This Quarter – BioSpace
EL SEGUNDO, Calif.--(BUSINESS WIRE)-- NantKwest, Inc. (NASDAQ: NK) and ImmunityBio, Inc., clinical-stage immunotherapy companies within the NantWorks family of companies, today announced they are in active discussions with the U.S. Food and Drug Administration (FDA) for vaccines and therapeutics to combat COVID-19.
Leveraging ImmunityBios expertise in vaccine development and natural killer cell activation, with a broad platform of immunomodulators currently in clinical trials for cancer and infectious diseases, and NantKwests extensive experience in off-the-shelf, cell-based therapeutics, the companies are combining their resources to design and develop therapeutics and vaccines for COVID-19.
Were in a race against time, but I am confident that, as a result of the incredible hard work the NantKwest, ImmunityBio, and the global scientific communities are undertaking, we will find effective therapeutics and vaccines against this coronavirus, said Patrick Soon-Shiong, M.D., Chairman & CEO of NantKwest and ImmunityBio.
Therapeutics:
The biological, immunological, and physiological status of the patients medical state should inform the treatment strategy to reverse the infectivity and tissue damage caused by this virus. ImmunityBio and NantKwest have developed immunomodulator regimens for COVID-19 based on the biological stage of the patients infection - from the mild, moderate to the severe or critically ill state.
In the mild-to-moderate stage of infection, we believe that the patients infection and viral load could be mitigated with natural killer (NK) and T cell stimulation. Hence, in this early-moderate stage of the disease, we are proposing clinical trials of N-803 alone, and a second trial of haNK alone, or haNK combined with convalescent plasma, said Dr. Soon-Shiong.
Investigational New Drug (IND) applications with the FDA for these trials are pending. ImmunityBios Il-15 superagonist N-803 is currently being used in clinical trials for other indications and has achieved Breakthrough Therapy Designation from the FDA[1] for the treatment of BCG-unresponsive non-muscle invasive bladder carcinoma in situ (NMIBC-CIS) patients. It has also demonstrated encouraging results in lowering the viral load in SHIV-infected monkeys[2], as announced last month at the Annual Conference on Retroviruses and Opportunistic Infections (CROI)[3].
In patients requiring ventilatory support in the severe state of COVID-19 disease, we are exploring the use of bone marrow-derived allogenic mesenchymal stem cells (BM-Allo-MSC) to mitigate the cytopathic storm, said Dr. Soon-Shiong.
NantKwest has proprietary isolation and expansion methods for growing MSCs and is using ImmunityBios automated, closed system (GMP-in-a-Box) to safely and rapidly grow these stem cells from a bone marrow cell bank in approximately 7-9 days. NantKwest has filed an IND with the FDA and anticipates beginning trials in Q2 2020.
Vaccines: Developing a platform for both initial immunizations and subsequent booster injections
First generation Adenovirus platforms (Ad5) currently in use are disadvantaged by inducing adenovirus neutralizing antibodies, thus limiting multiple doses and reducing the immune response to the antigen of interest. ImmunityBio has overcome this obstacle through the development of a second generation Ad5 platform. Through multiple deletions in the adenovirus genome, this next generation platform establishes a vector that is immunologically quiet as it relates to adenovirus protein production in the host dendritic cell and enables this same Ad5 vector to serve both as a prime and a boost treatment, even in patients with pre-existing adenovirus immunity. This second-generation Ad5 [E1-, E2b-, E3- deleted] platform has demonstrated safety in Phase I and Phase II studies in immunosuppressed cancer patients.
Furthermore ImmunityBio has extensive infectious disease experience with this second generation Ad5 platform and has published several peer-reviewed articles on studies demonstrating humoral and cell mediated immunity in H1N1 Influenza[4], HIV[5], SIV[6], Lassa Fever[7], Chikungunya, and Zika virus infections.
While development of therapies is urgently needed in this crisis, as urgent is the need to develop a vaccine with long-lasting cell-mediated immunity. Developing vaccines in the time of pandemics requires novel approaches and the use of modernized genomics, molecular dynamics, and vectors that are proven to induce cell-mediated immunity, with mass scale production capabilities. In 2009, with the H1N1 crisis, the scientific team developing this second generation Ad5 platform demonstrated that such a vaccine for the H1N1 pandemic could be developed in six weeks from identification of the H1N1 sequence. This experience in 2009 allows ImmunityBio to respond as rapidly as possible to the COVID-19 pandemic, continued Dr. Soon-Shiong. I view the spike (S) protein and the nucleocapsid (N) protein as the equivalent of a neoantigen in cancer. A recent study by the National Cancer Institute (NCI) in patients with advanced cancer, published in The Oncologist[8] reported positive evidence that this platform could induce antigen-specific T cell immunity, even in the face of previous adenoviral immunity, said Dr. Soon-Shiong. Together with our scientific collaborators at the NCI, we have recently published evidence[9] that the Ad5 platform can successfully induce cell-mediated immunity following the administration of Ad5-Neoantigens, with total remission of the tumor in pre-clinical models. Based on these findings, we are hopeful that the Ad platform could induce a similar immune response to this novel Coronavirus antigen.
About NantKwest
NantKwest (NASDAQ: NK) is an innovative, clinical-stage immunotherapy company focused on harnessing the power of the innate immune system to treat cancer and virally-induced infectious diseases. NantKwest is the leading producer of clinical dose forms of off-the-shelf natural killer (NK) cell therapies. The activated NK cell platform is designed to destroy cancer and virally-infected cells. The safety of these optimized, activated NK cellsas well as their activity against a broad range of cancershas been tested in phase I clinical trials in Canada and Europe, as well as in multiple phase I and II clinical trials in the United States. By leveraging an integrated and extensive genomics and transcriptomics discovery and development engine, together with a pipeline of multiple, clinical-stage, immuno-oncology programs, NantKwests goal is to transform medicine by delivering living drugs-in-a-bag and bringing novel NK cell-based therapies to routine clinical care. NantKwest is a member of the NantWorks ecosystem of companies. For more information, please visit http://www.nantkwest.com
haNK is a registered trademark of NantKwest, Inc.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements concerning or implying that NantKwest will be successful in improving the treatment of cancer. Risks and uncertainties related to this endeavor include, but are not limited to, obtaining FDA approval of NantKwests NK cells as well as other therapeutics as part of the NANT Cancer Vaccine platform as a cancer treatment.
Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements.
These and other risks regarding NantKwests business are described in detail in its Securities and Exchange Commission filings, including in NantKwests Annual Report on Form 10-K for the year ended December 31, 2019. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.
About ImmunityBio
ImmunityBio, Inc. is a privately-held immunotherapy company with a broad portfolio of biological molecules at clinical stages of development. The companys goals are to employ this portfolio to activate endogenous natural killer and CD8+ T cells in the fields of cancer and infectious disease. Specifically, ImmunityBios goal is to develop a memory T-cell cancer vaccine to combat multiple tumor typeswithout the use of high-dose chemotherapy. Regarding infectious disease, ImmunityBio is addressing HIV, influenza, and the coronavirus.
ImmunityBios first-in-human platform of technologies has enabled it to achieve one of the most comprehensive, late-stage clinical pipelines, activating both the innate (natural killer cell) and adaptive immune systems. The product pipeline includes an albumin-linked chemotherapeutic (Aldoxorubicin), a novel IL-15 cytokine superagonist (N-803), checkpoint inhibitors, macrophage polarizing peptides, bi-specific fusion proteins targeting TGFb and IL-12, adenovirus, and yeast vaccine therapies targeting tumor-associated antigens and neoepitopes.
In December 2019, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation to N-803 for BCG-unresponsive CIS non-muscle invasive bladder cancer (NMIBC). Other indications currently at registration-stage trials include BCG-unresponsive papillary bladder cancer, first- and second-line lung cancer, and metastatic pancreatic cancer.
ImmunityBios goal is to develop therapies, including vaccines, for the prevention and treatment of HIV, influenza, and the coronavirus SARS-CoV-2.
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements concerning or implying that ImmunityBio will be successful in improving the treatment of various diseases, including, but not limited to the novel coronavirus and cancer. Risks and uncertainties related to this endeavor include, but are not limited to, the companys beliefs regarding the success, cost and timing of its development activities and clinical trials.
Forward-looking statements are based on managements current expectations and are subject to various risks and uncertainties that could cause actual results to differ materially and adversely from those expressed or implied by such forward-looking statements. Accordingly, these forward-looking statements do not constitute guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. These forward-looking statements speak only as of the date hereof, and we disclaim any obligation to update these statements except as may be required by law.
[1]: ImmunityBio Granted FDA Breakthrough Therapy Designation for N-803 IL-15 Superagonist in NMIBC December 4, 2019 https://www.businesswire.com/news/home/20191204005300/en/ImmunityBio-Granted-FDA-Breakthrough-Therapy-Designation-N-803
[2]: ImmunityBio Announces Durable Virus Control of SHIV Without Anti-Retroviral Therapy (ART) by Activating NK and Memorty T Cells with N-803, an IL-15 Superagonist March 10, 2020 https://immunitybio.com/immunitybio-announces-durable-virus-control-of-shiv-without-anti-retroviral-therapy-by-activating-nk-and-memory-t-cells-with-n-803-an-il-15-superagonist/
[3]: Combination IL-15 Therapy in a SHIV NHP Model Presented at Conference on Retroviruses and Opportunistic Infections (CROI) March 8-11, 2020 Boston, Massachusetts http://www.croiconference.org/sessions/combination-il-15-therapy-shiv-nhp-model
[4]: Prevention of Influenza Virus Shedding and Protection from Lethal H1N1 Challenge Using a Consensus 2009 H1N1 HA and NA Adenovirus Vector Vaccine. Vaccine. 2011 Sep 16; 29(40): 70207026. Published 2011 Aug 5. doi: 10.1016/j.vaccine.2011.07.073
[5]: Induction and Comparison of SIV Immunity in Ad5 Nave and Ad5 Immne Non-Human Primates Using an Ad5 [E1-, E2b-] Based Vaccine. Vaccine. 2011 Oct 19;29(45):8101-7. doi: 10.1016/j.vaccine.2011.08.038. Epub 2011 Aug 22.
[6]: Control of SIV Infection and Subsequent Induction of Pandemic H1N1 Immunity in Rhesus Macaques Using an Ad5 [E1-, E2b-] Vector Platform.Vaccine. 2012 Nov 26; 30(50): 72657270. Published 2012 Oct 2. doi: 10.1016/j.vaccine.2012.09.058
[7]: Adenoviral Vector-Based Vaccine is Fully Protective Against Lethal Lassa Fever Vhallenge in Hartley Guinea Pigs. Vaccine..2019 Oct 23;37(45):6824-6831. doi: 10.1016/j.vaccine.2019.09.030. Epub 2019 Sep 24.
[8]: A Phase I Trial Using a Multitargeted Recombinant Adenovirus 5 (CEA/MUC1/Brachyury)Based Immunotherapy Vaccine Regimen in Patients with Advanced Cancer. The Oncol. doi:10.1634/theoncologist.2019-0608
[9]: Efficient Tumor Clearance and Diversified Immunity Through Neoepitope Vaccines and Combinatorial Immunotherapy. Cancer Immunology Research July 2019 DOI: 10.1158/2326-6066.CIR-18-0620
View source version on businesswire.com: https://www.businesswire.com/news/home/20200414005353/en/
Stem Cell Therapy Market Set to Witness an Uptick during 2017 to 2025 – Science In Me
Global Stem Cell Therapy Market: Overview
Also called regenerative medicine, stem cell therapy encourages the reparative response of damaged, diseased, or dysfunctional tissue via the use of stem cells and their derivatives. Replacing the practice of organ transplantations, stem cell therapies have eliminated the dependence on availability of donors. Bone marrow transplant is perhaps the most commonly employed stem cell therapy.
Osteoarthritis, cerebral palsy, heart failure, multiple sclerosis and even hearing loss could be treated using stem cell therapies. Doctors have successfully performed stem cell transplants that significantly aid patients fight cancers such as leukemia and other blood-related diseases.
Know the Growth Opportunities in Emerging Markets
Global Stem Cell Therapy Market: Key Trends
The key factors influencing the growth of the global stem cell therapy market are increasing funds in the development of new stem lines, the advent of advanced genomic procedures used in stem cell analysis, and greater emphasis on human embryonic stem cells. As the traditional organ transplantations are associated with limitations such as infection, rejection, and immunosuppression along with high reliance on organ donors, the demand for stem cell therapy is likely to soar. The growing deployment of stem cells in the treatment of wounds and damaged skin, scarring, and grafts is another prominent catalyst of the market.
On the contrary, inadequate infrastructural facilities coupled with ethical issues related to embryonic stem cells might impede the growth of the market. However, the ongoing research for the manipulation of stem cells from cord blood cells, bone marrow, and skin for the treatment of ailments including cardiovascular and diabetes will open up new doors for the advancement of the market.
Global Stem Cell Therapy Market: Market Potential
A number of new studies, research projects, and development of novel therapies have come forth in the global market for stem cell therapy. Several of these treatments are in the pipeline, while many others have received approvals by regulatory bodies.
In March 2017, Belgian biotech company TiGenix announced that its cardiac stem cell therapy, AlloCSC-01 has successfully reached its phase I/II with positive results. Subsequently, it has been approved by the U.S. FDA. If this therapy is well- received by the market, nearly 1.9 million AMI patients could be treated through this stem cell therapy.
Another significant development is the granting of a patent to Israel-based Kadimastem Ltd. for its novel stem-cell based technology to be used in the treatment of multiple sclerosis (MS) and other similar conditions of the nervous system. The companys technology used for producing supporting cells in the central nervous system, taken from human stem cells such as myelin-producing cells is also covered in the patent.
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Global Stem Cell Therapy Market: Regional Outlook
The global market for stem cell therapy can be segmented into Asia Pacific, North America, Latin America, Europe, and the Middle East and Africa. North America emerged as the leading regional market, triggered by the rising incidence of chronic health conditions and government support. Europe also displays significant growth potential, as the benefits of this therapy are increasingly acknowledged.
Asia Pacific is slated for maximum growth, thanks to the massive patient pool, bulk of investments in stem cell therapy projects, and the increasing recognition of growth opportunities in countries such as China, Japan, and India by the leading market players.
Global Stem Cell Therapy Market: Competitive Analysis
Several firms are adopting strategies such as mergers and acquisitions, collaborations, and partnerships, apart from product development with a view to attain a strong foothold in the global market for stem cell therapy.
Some of the major companies operating in the global market for stem cell therapy are RTI Surgical, Inc., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc., NuVasive, Inc., Pharmicell Co., Ltd., Anterogen Co., Ltd., JCR Pharmaceuticals Co., Ltd., and Holostem Terapie Avanzate S.r.l.
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Stem Cell Therapy Market Set to Witness an Uptick during 2017 to 2025 - Science In Me
Stem-Cell-Therapy Proves Effective in Treating COVID-19 – Iran Front Page – IFP News
The coronavirus pandemic has gripped all countries and infected more than half a million people around the world, so far. Some 100,000 people have also lost their lives to the virus. In Iran, too, all national plans have been drawn up while taking the epidemic into consideration.
The Royan Research Institute, which is a distinguished scientific centre in Iran, is naturally expected to adopt innovative measures in order to boost the societys health at a time when the country is gripped by the coronavirus outbreak. One of the measures for which the research centre is known is its cell-therapy capacity. Recently, Royan has taken action to tap into its cell-therapy potential to fight COVID-19.
Head of the research centre Abdolhossein Shahverdi has, in an interview, weighed in on the measures adopted by the institute to fight the coronavirus, namely the establishment of a molecular diagnostic laboratory.
The Royan Institute has good experience in the field of cell science, he said.
Given that the Royan Research Centre has the necessary infrastructure, we felt that we should tap into its potentialities to help tackle the ordeal which has gripped the country, he added.
Of course, treatments offered by the Royan Institute has, so far, been mostly related to infertility or cell-therapy for hardly curable diseases; however, there was good infrastructure at Roya, and with reliance on this very infrastructure, we began to put into service a molecular diagnosis lab in cooperation with the Ministry of Health and Medical Education to serve as a backup coronavirus diagnosis laboratory, he said.
One of the problems that develop in patients infected with the coronavirus is that their lungs are affected, and these problems may result in subsequent complications. So far, good treatment methods have been used for COVID-19 patients in Iran by drawing on Chinas experience in fighting the coronavirus as well as the findings of medical institutes inside Iran. In addition, a large percentage of patients have recovered using these very methods and returned to the bosom of their families.
he Royan Research Institute has experience in the field of cell therapy and using Mesenchymal stem cells (MSCs). The institute has a record of treating different illnesses using cell therapy. Mesenchymal stem cells are among good cells used in cell therapy and play a role in moderating reactions by the immune system and healing damaged tissue. The research institute has received initial licenses from the Ministry of Health and Medical Treatment to use MSCs. By drawing on its experience in cell therapy and its treatment record in that regard, the Royan Institute has taken the first step in developing a treatment for coronavirus patients through cell therapy and stem cells in cooperation with other hospitals and the Tehran University of Medical Sciences.
In this treatment method, MSCs are used. One of the sources of these cells are umbilical cord blood cells or marrow cells, he noted.
In the past, we had used these cells to treat some incurable illnesses such as cartilage and bone diseases, and we achieved good results, said the director of the institute.He expressed hope the treatment method will successfully pass the stage of clinical tests and prove useful in treating coronavirus patients.
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Stem-Cell-Therapy Proves Effective in Treating COVID-19 - Iran Front Page - IFP News
Patient-Doctor Duo: The Basics of Myeloproliferative Neoplasms – Curetoday.com
In this special edition of the CURE Talks Cancer podcast, we teamed up with our sister publication OncLive on Air to speak with a patient-doctor duo on myeloproliferative neoplasms.
BY Kristie L. Kahl and Gina Columbus
MPNS essential thrombocythemia, myelofibrosis and polycythemia vera begin with an abnormal change, or mutation, in a stem cell in the bone marrow, which leads to an overproduction of any combination of white cells, red cells and platelets.
In this special edition of the CURE Talks Cancer podcast, we teamed up with our sister publication OncLive on Air to speak with a patient-doctor duo on the disease.
Learn more from Dr. Ruben A. Mesa, director of the Mays Cancer Center at UT Health San Antonio MD Anderson, and Antje Hjerpe, a patient diagnosed with essential thrombocythemia in 1992. The pair discuss myeloproliferative neoplasms what they are, how theyre treated and how patients can talk to their doctors to be their own best advocates.
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Patient-Doctor Duo: The Basics of Myeloproliferative Neoplasms - Curetoday.com
Southern California Patients Treated with Leronlimab for COVID-19 under Emergency IND: 4 Patients with Moderate Indications Removed from Oxygen; 3…
More than 25 EINDs approved by FDA for leronlimab use in COVID-19 patients
Phase 2 trial - As of last week, 12 patients enrolled from 2 sites; 3 more sites to initiate enrollment this week, for a total of 5 sites
Phase 2b/3 trial - First hospital cleared to enroll patients beginning today
VANCOUVER, Washington, April 13, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY), (CytoDyn or the Company), a late-stage biotechnology company developing leronlimab (PRO 140), a CCR5 antagonist with the potential for multiple therapeutic indications, announced today a comprehensive update and overview of the therapeutic indications from over 30 COVID-19 patients recently treated with leronlimab in over 4 hospitals and clinics throughout the country. More than 25 hospitals, to date, have requested participation in the Companys trials.
Patient enrollment in the Companys two clinical trials and Emergency Investigational New Drug (EIND) is as follows:
-- More than 25 patients have been administered leronlimab under EINDs authorized by the U.S. Food and Drug Administration (FDA). -- Rate of response in mild-to-moderate patients under EIND has been very promising with the first five patients treated being removed from oxygen. -- As of last week, 12 patients have been treated in the Phase 2 trial for mild-to-moderate COVID-19 indications and, because it is a double-blinded, placebo-controlled trial, results are not yet available. -- First site cleared to enroll patients in Phase 2b/3 beginning today.
Nader Pourhassan, Ph.D., President and Chief Executive Officer of CytoDyn said, We continue to coordinate around the clock with healthcare professionals across the country to deliver leronlimab to patients and we are in regular contact with the FDA to ensure they receive current patient data. We are planning to rapidly enroll 75 patients and report the results to the FDA as quickly as possible.
About Coronavirus Disease 2019SARS-CoV-2 was identified as the cause of an outbreak of respiratory illness first detected in Wuhan, China. The origin of SARS-CoV-2 causing the COVID-19 disease is uncertain, and the virus is highly contagious. COVID-19 typically transmits person to person through respiratory droplets, commonly resulting from coughing, sneezing, and close personal contact. Coronaviruses are a large family of viruses, some causing illness in people and others that circulate among animals. For confirmed COVID-19 infections, symptoms have included fever, cough, and shortness of breath. The symptoms of COVID-19 may appear in as few as two days or as long as 14 days after exposure. Clinical manifestations in patients have ranged from non-existent to severe and fatal. At this time, there are minimal treatment options for COVID-19.
About Leronlimab (PRO 140) The FDA has granted a Fast Track designation to CytoDyn for two potential indications of leronlimab for deadly diseases. The first as a combination therapy with HAART for HIV-infected patients and the second is for metastatic triple-negative breast cancer. Leronlimab is an investigational humanized IgG4 mAb that blocks CCR5, a cellular receptor that is important in HIV infection, tumor metastases, and other diseases, including NASH. Leronlimab has completed nine clinical trials in over 800 people, including meeting its primary endpoints in a pivotal Phase 3 trial (leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients).
In the setting of HIV/AIDS, leronlimab is a viral-entry inhibitor; it masks CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab has been the subject of nine clinical trials, each of which demonstrated that leronlimab could significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent leading to potentially fewer side effects and less frequent dosing requirements compared with daily drug therapies currently in use.
In the setting of cancer, research has shown that CCR5 may play a role in tumor invasion, metastases, and tumor microenvironment control. Increased CCR5 expression is an indicator of disease status in several cancers. Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 98% in a murine xenograft model. CytoDyn is, therefore, conducting a Phase 1b/2 human clinical trial in metastatic triple-negative breast cancer and was granted Fast Track designation in May 2019.
The CCR5 receptor appears to play a central role in modulating immune cell trafficking to sites of inflammation. It may be crucial in the development of acute graft-versus-host disease (GvHD) and other inflammatory conditions. Clinical studies by others further support the concept that blocking CCR5 using a chemical inhibitor can reduce the clinical impact of acute GvHD without significantly affecting the engraftment of transplanted bone marrow stem cells. CytoDyn is currently conducting a Phase 2 clinical study with leronlimab to support further the concept that the CCR5 receptor on engrafted cells is critical for the development of acute GvHD, blocking the CCR5 receptor from recognizing specific immune signaling molecules is a viable approach to mitigating acute GvHD. The FDA has granted orphan drug designation to leronlimab for the prevention of GvHD.
About CytoDyn CytoDyn is a late-stage biotechnology company developing innovative treatments for multiple therapeutic indications based on leronlimab, a novel humanized monoclonal antibody targeting the CCR5 receptor. CCR5 appears to play a critical role in the ability of HIV to enter and infect healthy T-cells. The CCR5 receptor also appears to be implicated in tumor metastasis and immune-mediated illnesses, such as GvHD and NASH. CytoDyn has successfully completed a Phase 3 pivotal trial with leronlimab in combination with standard antiretroviral therapies in HIV-infected treatment-experienced patients. CytoDyn plans to seek FDA approval for leronlimab in combination therapy and plans to complete the filing of a Biologics License Application (BLA) in April of 2020 for that indication. CytoDyn is also conducting a Phase 3 investigative trial with leronlimab as a once-weekly monotherapy for HIV-infected patients. CytoDyn plans to initiate a registration-directed study of leronlimab monotherapy indication. If successful, it could support a label extension. Clinical results to date from multiple trials have shown that leronlimab can significantly reduce viral burden in people infected with HIV with no reported drug-related serious adverse events (SAEs). Moreover, a Phase 2b clinical trial demonstrated that leronlimab monotherapy can prevent viral escape in HIV-infected patients; some patients on leronlimab monotherapy have remained virally suppressed for more than five years. CytoDyn is also conducting a Phase 2 trial to evaluate leronlimab for the prevention of GvHD and a Phase 1b/2 clinical trial with leronlimab in metastatic triple-negative breast cancer. More information is at http://www.cytodyn.com.
Forward-Looking Statements This press release contains certain forward-looking statements that involve risks, uncertainties and assumptions that are difficult to predict. Words and expressions reflecting optimism, satisfaction or disappointment with current prospects, as well as words such as believes, hopes, intends, estimates, expects, projects, plans, anticipates and variations thereof, or the use of future tense, identify forward-looking statements, but their absence does not mean that a statement is not forward-looking. The Companys forward-looking statements are not guarantees of performance, and actual results could vary materially from those contained in or expressed by such statements due to risks and uncertainties including: (i) the sufficiency of the Companys cash position, (ii) the Companys ability to raise additional capital to fund its operations, (iii) the Companys ability to meet its debt obligations, if any, (iv) the Companys ability to enter into partnership or licensing arrangements with third parties, (v) the Companys ability to identify patients to enroll in its clinical trials in a timely fashion, (vi) the Companys ability to achieve approval of a marketable product, (vii) the design, implementation and conduct of the Companys clinical trials, (viii) the results of the Companys clinical trials, including the possibility of unfavorable clinical trial results, (ix) the market for, and marketability of, any product that is approved, (x) the existence or development of vaccines, drugs, or other treatments that are viewed by medical professionals or patients as superior to the Companys products, (xi) regulatory initiatives, compliance with governmental regulations and the regulatory approval process, (xii) general economic and business conditions, (xiii) changes in foreign, political, and social conditions, and (xiv) various other matters, many of which are beyond the Companys control. The Company urges investors to consider specifically the various risk factors identified in its most recent Form 10-K, and any risk factors or cautionary statements included in any subsequent Form 10-Q or Form 8-K, filed with the Securities and Exchange Commission. Except as required by law, the Company does not undertake any responsibility to update any forward-looking statements to take into account events or circumstances that occur after the date of this press release.
CYTODYN CONTACTSInvestors: Dave Gentry, CEO RedChip Companies Office: 1.800.RED.CHIP (733.2447) Cell: 407.491.4498 dave@redchip.com