Most breast cancers utilize the female hormone estrogen to grow, so drug-induced estrogen deprivation is used as a treatment in many patients. However, cancer will recur in one-third of these patients. A research team at Dartmouths and Dartmouth-Hitchcocks Norris Cotton Cancer Center, led by Todd W. Miller, PhD, is trying to understand why dormant breast cancer cells survive despite being starved of estrogen. The team discovered that an anti-diabetes drug, metformin, which is being tested in many clinical trials as an anti-cancer agent, actually activated fat metabolism that protected dormant breast cancer cells during estrogen deprivation. The findings suggest that the drug has context-dependent effects on cancer cells. The results, entitled AMPK activation by metformin promotes survival of dormant ER+ breast cancer cells, are newly published online inClinical Cancer Research, a journal of the American Association for Cancer Research.

Knowledge that metformin has context-dependent effects on cancer cells will inform a better understanding of ongoing and prior clinical trials testing metformin, and help shape the design of trials moving forward. Our study indicates that the development of drugs targeting fat metabolism is warranted for breast cancer. Most excitingly, anti-angina drugs that block fat metabolism may be quickly repurposed as potential treatments for cancer and tested in clinical trials, says Miller.

Next steps include clinical trials testing drugs that block fat metabolism in breast cancer. Were also designing preclinical studies to further dissect the roles of fat metabolism in breast and other cancers, with the goal of identifying more refined therapeutic targets that will selectively kill cancer cells and not harm healthy cells, notes Miller.

Reference:Hampsch, et al. (2020) AMPK activation by metformin promotes survival of dormant ER+ breast cancer cells. Clinical Cancer Research DOI: 10.1158/1078-0432.CCR-20-0269.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

More here:
Team Reveal Key to the Survival of Dormant Breast Cancer Cells - Technology Networks

Recommendation and review posted by Bethany Smith

When our panelists were asked which innovation showed the best results, scalp injections of platelet-rich plasma (PRP) for hair growth was at the top of many lists. You get significant benefits with very little downside, Dr. Schultz says. First, your blood is drawn and spun in a centrifuge to separate the PRP, which is then injected all over your scalp. Using your bloods growth factors to stimulate collagen and follicles lets your own body work its magic, says dermatologist Mona Gohara, M.D.

Another new option is a laser called Lutronic KeraLase ($750 to $1,000 per treatment), which is paired with a synthetic growth factor serum. The device stamps across your scalp, stimulating the area and creating tiny channels in the follicles, where the serum is then applied. It delivers active ingredients where theyre needed, says dermatologist Jeanine Downie, M.D. The serum can contain even more growth factors than a persons own PRP, plus stem cells to help hair growth, she says. Nutrafol for Women (Buy It, $79 $88 for 1 month, nutrafol.com), a supplement brand for hair loss, also garnered multiple mentions from our panel. Ive seen promising results, especially in conjunction with the above treatments, Dr. Downie says.

Read more from the original source:
The Best Face and Body Skin-Care Products for 2020 - Shape Magazine

Recommendation and review posted by Bethany Smith

Global Cell-Based Immunotherapy Market report endows with the key measurements, the status of the manufacturers and is a significant source of direction for businesses and organizations. Competitive landscape is another major section of this Global Cell-Based Immunotherapy Market research report which presents with a clear insight into the market share analysis and actions of key industry players. This market research report highlights the most important market insights that take your business to the highest level of growth and success. Hence, this Global Cell-Based Immunotherapy Market report helps businesses to define their own strategies for the up gradation in the existing product, possible modifications required in the future product, sales, marketing promotion and distribution of the product in the existing and the new market.

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Cell-based immunotherapy is a potential future-oriented cancer treatment approach. It is evolving quickly as an alternative to traditional cancer treatment based on chemotherapy. Stem cells are used for the diagnosis of different types of cancer in cell-based immunotherapy. These cells have the ability to create extra placental or embryonic cells to cure cancer. This therapy is widely used in application such as breast cancer, skin cancer, ovarian cancer, lung cancer and prostate cancer.

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Global Cell-Based Immunotherapy Market is Thriving with Rising Latest Trends by 2027 | Top Players-AbbVie Inc., Genentech USA, Inc., Amgen Inc,...

Recommendation and review posted by Bethany Smith

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.

Read the rest here:
Planned Clinical Trial of Allogeneic Stem Cell Therapy Remestemcel-L in Patients with COVID-19 - Cancer Network

Recommendation and review posted by Bethany Smith

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

Recommendation and review posted by Bethany Smith

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.

Additional Resources:

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.

Here is the original post:
FDA Approves New Therapy for Triple Negative Breast Cancer That Has Spread, Not Responded to Other Treatments - Herald-Mail Media

Recommendation and review posted by Bethany Smith

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

See the original post here:
Pivotal REACH2 Study Data Published in NEJM Highlight Superior Efficacy of Ruxolitinib versus Best Available Therapy in Patients with Acute GVHD -...

Recommendation and review posted by Bethany Smith

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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

Recommendation and review posted by Bethany Smith

Testing is critical to controlling the coronavirus and eventually easing the restrictions that have halted daily life for most Americans. But theres been confusion about what kinds of tests are available and what they actually measure.

There are two main types in the United States. One is a diagnostic test to see if you have an active infection with the coronavirus, whether you have symptoms of COVID-19 or not. The other checks to see if you were previously exposed at some point and fought off an infection.

Currently, almost all testing in hospitals, clinics and drive-through sites uses the first testing method, to help doctors detect and treat people with active infections.

The second method looks for coronavirus antibodies in the blood because their presence is a sign that the immune system has fought off an infection. Experts predict this type of test will play a key role in allowing many Americans to safely return to work and school by identifying those who are likely immune from the virus.

Heres a closer look at both tests and how they work:

Genetic testing is the best method for detecting active COVID-19 infections and making a diagnosis. The process requires several steps and high-tech testing equipment to detect tiny traces of the virus that causes COVID-19.

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First, the doctor or nurse gathers samples from a patients nose or throat using swabs. The sample is developed through a process called polymerase chain reaction, or PCR, which is used to boost any traces of virus until they are detectable.

The same process has long been used to diagnose viruses such as HIV and hepatitis.

The U.S. Food and Drug Administration has now authorized dozens of these types of tests that can be run at hospitals, university laboratories and large testing chains such as Quest Diagnostics, along with one that patients can use at home. The tests typically take four to six hours to run and can take a day or more to turn around if a sample needs to be shipped to another site for processing.

Last month, several faster options that dont require laboratory processing came on the market. The fastest is a 15-minute test from Abbott Laboratories run on small, portable electronic machines found in thousands of hospitals, clinics and doctors offices. The test puts all the chemical ingredients into a small cartridge thats inserted into the Abbott machine along with the swabbed sample.

There are caveats to these types of tests: Someone can test negative one day and then positive the next. Much depends on the level of virus and whether the swab picked up enough of it to make a good sample.

The FDA recently authorized the first genetic test that uses saliva, rather than a nasal swab, but its availability is limited for now.

U.S. testing continues to be squeezed by huge demand, limited testing machines and shortages of key supplies such as swabs. While the U.S. is now conducting well over 1 million tests per week, most experts say that number will need to increase at least threefold before social distancing is dramatically eased.

The second type of test wont tell you whether youre currently infected with the virus, but whether you were infected sometime in the past.

Instead of searching for the virus itself, these tests search for blood proteins called antibodies, which the body produces days or weeks after fighting an infection. The same approach usually a finger-prick of blood on a test strip is used for HIV, hepatitis and many other infections.

Dr. Anthony Fauci, the nations top infectious disease official, says its a reasonable assumption, that if you have antibodies, you will be protected from another infection. But federal researchers still have to answer several key questions: How accurate are the tests? What antibody level is needed for immunity? How long does that immunity last?

By testing broad populations for antibodies, researchers hope to learn how widely the virus spread and how deadly it really is. Both questions remain unresolved since experts believe at least 25% of those infected never show symptoms. Some of those larger studies are getting started.

For now, FDA Commissioner Dr. Stephen Hahn suggests the blood tests could be used by frontline healthcare workers. For example, a doctor who tests positive for antibodies could be in a safer position to treat COVID-19 patients than a colleague who tests negative.

The FDA has authorized four antibody tests based on preliminary reviews. But more than 90 others have launched without FDA oversight under a federal emergency policy intended to quickly ramp up testing options. Those tests are supposed to bear disclaimers, including that they have not been FDA-approved, though many dont.

Several laboratory and physician groups have called for tighter FDA control.

They have a responsibility to go back, demand more rigorous clinical trials and probably put some guardrails around these tests, said Dr. Gary Procop of the Cleveland Clinic, president of the American Society for Clinical Pathology.

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What are the 2 main types of coronavirus testing? - Los Angeles Times

Recommendation and review posted by Bethany Smith

Certain genetic differences might separate people who fall severely ill with COVID-19 from those who contract the infection but hardly develop a cough, a new preliminary study suggests.

The research is still in its early days, though, experts say.

The immune system can react to viruses thanks, in part, to specific genes that help cells spot unfamiliar bugs when they enter the body. The genes, known as human leukocyte antigen (HLA) genes, contain instructions to build proteins that bind to bits of a pathogen; those proteins serve as warning flags to alert immune cells. The immune cells, once trained to recognize these bits, jumpstart the process of building antibodies to target and destroy the invasive germ.

Within each individual, HLA genes code for three different classes of proteins; in other words, HLAs come in a variety of flavors, and depending on which HLAs you have, your body may be better or worse equipped to fight off certain germs including SARS-CoV-2, the virus that causes COVID-19.

In a new study, published April 17 in the Journal of Virology, researchers used computer models to predict which combination of HLAs might be best at binding SARS-CoV-2, and which might be worst.

If certain HLAs can bind well to a large proportion of the virus's proteins, "we expect there to be a more protective immune response," authors Abhinav Nellore and Dr. Reid Thompson, who lead a computational biology research group at the Oregon Health and Science University, told Live Science in an email. A better bind means that the viral proteins are more likely to be presented to immune cells and prompt the production of specific antibodies, the authors said.

"If the interaction is not stable, you will not have a proper [immune] response," said Dr. Shokrollah Elahi, an associate professor in the Department of Dentistry and adjunct associate professor in the Department of Medical Microbiology and Immunology at the University of Alberta, who was not involved in the study.

Related: 10 deadly diseases that hopped across species

But a stable bond, alone, does not guarantee the best immune response, Elahi added. If an HLA binds a viral protein that happens to be critical for the germ to replicate and survive, the subsequent antibody activity will likely target the virus more effectively than that prompted by a less important protein, Elahi said.

"This is an issue we did not address in our analysis," the authors noted. Instead, the team focused on predicting how well different HLA types could bind to bits of SARS-CoV-2. Their analysis identified six HLA types with a high capacity to bind different SARS-CoV-2 protein sequences, and three with a low capacity to do so. Specifically, a HLA type known as HLA-B*46:01 had the lowest predicted capacity to bind to bits of SARS-CoV-2.

The same HLA type cropped up in a 2003 study published in the journal BMC Medical Genetics, which assessed patients infected with SARS-CoV, a closely related coronavirus that caused an outbreak of severe acute respiratory syndrome in the early 2000s. The study found that, in a group of patients of Asian descent, the presence of HLA-B*46:01 was associated with severe cases of the infection. In their paper, the research group noted that more clinical data would be needed to confirm the connection and the same goes for the new study of SARS-CoV-2, Nellore and Thompson said.

"The most substantial limitation of our study is that this was conducted entirely on a computer and did not involve clinical data from COVID-19 patients," the authors said. "Unless and until the findings we present here are clinically validated, they should not be employed for any clinical purposes," they added.

"In the body, we have so many things interacting," Elahi said. HLAs represent just one piece of a large, intricate puzzle that comprises the human immune system, he said. To better understand the variety of immune responses to COVID-19, Elahi and his research group aim to assess markers of immune system activity in infected patients and also catalog the ratio of immune cell types present in their bodies. While taking age, sex and other demographic factors into account, these so-called immunological profiles could help pinpoint when and why the illness takes a turn in some patients.

The clinical data could be assessed in parallel with genetic data gathered from the same patients, Elahi added. Similarly, Nellore and Thompson said that "COVID-19 testing should be paired with HLA typing, wherever [and] whenever possible," to help determine how different HLA types relate to symptom severity, if at all. Partnerships with genetic testing companies, biobanks and organ transplant registries could also offer opportunities to study HLA types in larger populations of people, they said.

"We cannot in good conscience predict at this point who will be more or less susceptible to the virus because we have not analyzed any clinical outcomes data with respect to HLA type to know that any of our predictions are valid," the authors said. If future studies support the notion that some HLA genes protect people from the virus, while others place patients at greater risk, those in the latter group could be first in line for vaccination, they added.

"In addition to prioritizing vaccinating the elderly or those with preexisting conditions, one could prioritize vaccinating people with HLA genotypes that suggest the SARS-CoV-2 virus is more likely to give them worse symptoms."

The authors went on to analyze how well HLAs can bind SARS-CoV-2 as compared with other coronaviruses, such as those that cause the common cold and infect humans often. They identified several viral bits shared between SARS-CoV-2 and at least one of these common viruses, suggesting exposure to one germ could somewhat protect the body against the other.

"If someone was previously exposed to a more common coronavirus and had the right HLA types ... then it is theoretically possible that they could also generate an earlier immune response against the novel SARS-CoV-2," the authors said. On the other hand, exposure to a similar virus could leave the body ill-equipped to fight off the new one, if, for instance, "the body is using an old set of tools that aren't ideally suited to address the new problem," the authors said.

Originally published on Live Science.

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Could genetics explain why some COVID-19 patients fare worse than others? - Live Science

Recommendation and review posted by Bethany Smith

LONDON, April 23, 2020 /PRNewswire/ -- The advances in genomics and proteomics is a key trend driving the growth of the molecular diagnostics market. Polymerase chain reaction (PCR) is a recent development in genomics and helps in discovering new approaches to molecular diagnosis for disease diagnosis and pathogenesis of diseases. This enables better monitoring and a fast diagnosis, and major organizations are investing to have a competitive edge. Following the trend, Genome Canada, a source of funding for genomics and proteomics, announced plans to invest $16 million between the period April 2015 and March 2017. The growing research in proteomics and genomics will help develop more advanced molecular diagnostic techniques in the forecast period.

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Molecular Diagnostics Market Overview And Segments

Molecular diagnostics is a term used to describe a class of techniques that are used to examine biological markers in the genetic code (genome) of an organism and to determine how their cells express their genes as proteins. The molecular diagnostics market covered in this report is segmented by technology into polymerase chain reaction (PCR), DNA sequencing, and next-generation sequencing. It is also segmented by product into instruments, reagents and consumables, and software & services. By application it is segmented into oncology, pharmacogenomics, infectious diseases, genetic testing, neurological disease, cardiovascular disease, microbiology, and others, and by end-user the market is segmented into hospitals, laboratories, and others (blood banks, home health agencies, and nursing homes).

The Molecular Diagnostics Market Will Reach $12.09 Billion By 2023

The global molecular diagnostics market was worth $8.62 billion in 2019. It is expected to grow at a compound annual growth rate (CAGR) of 8.82% and reach $12.09 billion by 2023. The global prevalence of infectious diseases and cancers of different forms drives the molecular diagnostics market. The increasing prevalence of infectious diseases and various types of cancer creates a demand for new diagnostic procedures including fast and specific molecular diagnostic tests. According to the International Agency for Research on Cancer (IARC), in 2018, there were 17.0 million new cancer cases and 9.5 million cancer deaths worldwide, and by 2040, the number is expected to increase to 27.5 million new cancer cases and 16.3 million cancer deaths. The increasing prevalence of cancer globally will increase the demand for molecular diagnostic tests for effective diagnosis of cancer over the coming years, thereby contributing to the growth of the market.

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Bruker Corporation Acquires Hain Lifescience GmbH

Companies in the molecular diagnostics market invest in mergers and acquisitions to strengthen their businesses. In August 2018, Bruker Corporation, an American based manufacturer of scientific instruments for molecular and materials research announced plans to acquire majority stake in Hain Lifescience GmbH for an undisclosed amount. This acquisition will help Bruker to expand its presence in attractive microbiology and virology infectious disease MDx markets, add tuberculosis and mycobacteria testing, virology, and human genetics MDx, and add a pipeline for multiplex syndromic panel testing to its portfolio. Hain Lifescience GmbH is an infectious disease molecular diagnostics (MDx) specialist and provides solutions for detecting microbial and viral pathogens, antibiotic resistance testing, and human genetic diseases.

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Major players in the global molecular diagnostics market include Becton, BioMrieux SA., Danaher Corporation, Grifols, Hologic Inc., Novartis AG, QIAGEN, Siemens Healthcare GmbH, Abbott, Dickinson and Company, Hoffmann-La Roche Ltd., BD, Bio-Rad Laboratories, Johnson & Johnson, Cepheid, Roche Diagnostics, Alere, Bayer AG, Dako, Sysmex Corporation, Agilent Technologies, Diasorin, Illumina, Thermo Fisher Scientific, and Biocartis Group NV.

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Polymerase Chain Reaction (PCR) And Real-Time Polymerase Chain Reaction (PCR) Testing Global Market Report 2020-30: Covid 19 Implications And Growth

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Advances in Genomics and Proteomics in the $8.62 Billion Molecular Diagnostics Market - Yahoo Finance

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Whether its the southern hospitality or the world-class cuisine, Houston has been attracting the tech worlds top talent for decades. As the home of NASA Mission Control, the city has found itself at the center of space exploration since the beginning, serving as the operational hub of every U.S. human space mission since the launch of Project Gemini. In light of this, its no surprise that aerospace and defense is one of Houstons top tech sectors, with industry giants like Lockheed Martin and Boeing operating throughoutthe city. But perhaps its Houstons energy industry that holds the most economic weight. According to the Greater Houston Partnership, the citys energy sector employs more than 200,000 people across the city, while clean energyin particular boasts more than $3 billion in VC funding.

With its abundance of leading research facilities and rich exploration history, Houston houses one of the nations largest tech communities, which is expected to growover the next several years. In fact, researchers expect to see nearly 9,000 new tech jobs across the city by 2026, according to a 2019 report from CompTIA. In addition to hosting Microsoft, Houstons existing tech ecosystem boasts representation from a wide range of sectors like biotech, IT, energy and aviation. From developing flight tracking systems to uncovering new disease treatments, many of Houstons tech companies are true leaders in their industries, solidifying the Bayou Citys status as a hotbed of innovation and discovery. Heres a look at 25 tech companies in Houston that have helped shape the citys tech landscape.

Founded: 2005

Focus: Aviation + Aerospace

What they do: Founded by Daniel Baker, FlightAware is a flight tracking data company that offers flight tracking services for both private and commercial air traffic. The companysHyperFeed engine integrates data from sources around the world to deliver comprehensive flight tracking information. Leveraging data from air traffic control systems in over 45 countries, FlightAware boasts a terrestrial network of over 20,000 ADS-B ground stations worldwide.

Founded: 2000

Focus: TV Search + Streaming

What they do: SnapStream develops TV software that enables users to record, clip, share and search broadcast TV and monitor their feeds for regulatory compliance. The companys TV search engine can simultaneously record up to 10 TV shows and can store more than 17,000 hours of recordings, giving users the ability to search within recordings and find certain TV content, which they can then download. SnapStream works with a wide range of clients including The Daily Show, Last Week Tonight and the U.S. Senate.

Founded: 1981

Focus: Asset Optimization + Performance Management

What they do: Aspen Technology develops software that helps oil and gas, chemical and engineering companies achieve their goals in safety, sustainability and operational performance. Using advances in AI and machine learning, the company helps fuel clients competitive drive by discovering new possibilities in process modeling, simulation and optimization. Since its inception, the company has raised $100 million in funding in addition to acquiring seven organizations.

Founded: 1985

Focus: Pricing Effectiveness + Revenue Management

What they do: Established by Mariette and Ron Woestemeyer, PROS is dedicated to helping people and companies outperform by enabling smarter selling in the digital economy. The company provides companies with predictive and prescriptive guidance, enabling them to dynamically price, configure and sell their products and services. With offices located worldwide, PROS helps enterprises achieve previously unattainable levels of speed and consistency.

Founded: 1999

Focus: Managed IT Services

What they do: A subsidiary of Ricoh, mindSHIFT Technologies is an IT outsourcing and cloud services provider. Serving businesses of all sizes, the company helps organizations migrate to the cloud while implementing mobility and security. Organizations are granted secure and backed-up data with mindSHIFT Technologies, allowing them to work safely and securely from anywhere.

Founded: 1975

Focus: Business Software + Developer Tools

What they do: Since its inception, Microsoft has evolved into a true tech titan, serving customers worldwide with its software products and services. From Xbox to Office 365, the corporations products are some of the most widely used in the tech world, serving both entertainment and business sectors. In 2016, Microsoft opened its first cloud-based Microsoft Technology Center in Houston, where customers can take part in immersive experiences, proof-of-concept workshops and strategy briefings.

Founded: 1998

Focus: Software Development + IT Services

What they do: Cradle Solution is a software development company that serves clients in various industries. The company offers software-as-a-service for the home healthcare industry in addition to providing technical and executive training and IT governance consulting services for the oil and gas industry. Cradle Solutions offers other customized software tools for companies worldwide.

Founded: 1980

Focus: IT Strategy + Solutions

What they do: Sirius Computer Solutions provides business solutions that span the data center and other lines of business. The company provides a wide range of solutions to help IT professionals cut costs, increase reliability, ease the burden of management, maximize flexibility, mitigate risk and improve service. Sirius Computer Solutions has acquired three organizations since its founding including Forsythe and thinkASG.

Founded: 2001

Focus: Small Business Development + IT Solutions

What they do: Enstep Technology Solutions is a business partner and IT services provider for small and medium-sized businesses. The company is dedicated to helping clients understand why their technology plan isnt working and then provide the means to help them achieve their goals. Enstep Technology Solutions also helps businesses alleviate potential risks like losing data through their backup management services, which includes threat assessment and disaster recovery testing.

Founded: 1902

Focus: Gases + Chemicals

What they do: Headquartered in Paris, France, Air Liquide provides industrial gases, technologies and services to various industries. The company specializes in on-site industrial gas production, advanced precursor materials, gas and chemical management, and analytical and laboratory services. Serving more than 3.6 million customers worldwide, Air Liquide has acquired 43 organizations since its inception including Medidis and DiaLibre.

Founded: 1980

Focus: Cloud Computing + Security Management

What they do: BMC Software is committed to helping clients reinvent their businesses with open, scalable and modular solutions. The companys solutions encompass multi-cloud cost and security management, cloud migration, application performance improvement, big data insights and mainframe cost optimization. BMC Software has several offices located worldwide.

Founded: 2006

Focus: Cell Therapy + Regenerative Medicine

What they do: InGeneron is a clinical-stage cell therapy company that enables novel, safe and evidence-based therapies. The company is dedicated to developing treatments that unlock the healing potential of patients own regenerative cells. InGeneron specializes in helping patients who suffer from chronic pain and other musculoskeletal conditions.

Founded: 1985

Focus: Oil + Energy

What they do: ENGlobal is a specialty engineering services firm that specializes in oil and gas automation solutions and subsea control systems. The company offers a variety of services including advanced automated data gathering systems, IT projects, feasibility studies, cost estimation and environmental compliance. ENGlobal also operates a government services group that offers engineering, design, installation, operations and maintenance support to government facilities.

Founded: 2000

Focus: Genetic Testing

What they do: Gene By Gene is a biotech company that offers affordable genetic testing services. The companys genetic testing services encompass relationship DNA testing, carrier screenings, research genetics, forensics and ancestry DNA testing. Gene By Genes aim is to empower people everywhere to make decisions on their genetic health through affordable, high-quality services.

Founded: 1995

Focus: IT Systems Management

What they do: NetIQ is a global enterprise software company that offers a wide range of solutions. The companys services include identity and access management, security management and data center management. NetIQ is committed to helping organizations tackle information protection challenges and manage highly-distributed application environments.

Founded: 1994

Focus: Biopharmaceuticals

What they do: Founded by Yoseph Shaaltiel, Protalix Biotherapeutics is a clinical-stage biopharmaceutical company that develops and manufactures recombinant therapeutic proteins. The company specializes in developing complex therapeutic proteins for the treatment of genetic disorders such as Gaucher disease and Fabry disease in addition to advancing additional recombinant biopharmaceutical drug development programs. Since its founding, Protalix Biotherapeutics has raised $62.5 million over four funding rounds.

Founded: 1998

Focus: IT Services

What they do: Cybersoft Technologies is an IT company that focuses on mobile application development and software services. The company offers a wide range of products including PrimeroEdge, which provides software solutions for child nutrition departments in school districts, as well as YottaTime and Attendance, which streamlines the process of employee work hours, overtime and leave management. Cybersoft Technologies works primarily with city agencies, Fortune 500 companies and K-12 school districts across the nation.

Founded: 1919

Focus: Aerospace + Defense

What they do: KBR, Inc. is an engineering, procurement and construction company that provides services and technologies to government and industry clients. Operating across 40 countries, the company offers government solutions that cover the full lifecycle of defense, space and aviation, while their tech solutions encompass equipment, catalysts and digital solutions. Since its inception, KBR, Inc. has acquired 11 organizations such as RRT Global and Honeywell Technology Solutions.

Founded: 1986

Focus: HR Services

What they do: Insperity specializes in delivering human resources solutions that help clients strengthen and streamline their businesses. The company helps organizations manage costs and minimize risks, offering a wide range of services such as HR consulting, accounting and bookkeeping, expense reporting, performance reviews and organizational charting. With more than $4.3 billion in annual revenue, Insperity has 70 offices located across the country.

Founded: 1919

Focus: Oil + Energy

What they do: Halliburton provides products and services to the energy industry. With a focus on sustainability, the company helps its customers maximize value throughout the lifecycle of the reservoir, which includes locating hydrocarbons, managing geological data, drilling and formation evaluation, and well construction and completion. Halliburton works with national and independent oil and natural gas companies across the globe.

Founded: 1852

Focus:Logistics + Cash Handling

What they do: Headquartered in Stockholm, Sweden, Loomis offers solutions for cash handling. The company works primarily with banks, multi-location retailers, stores and other commercial enterprises to offer them efficient management of the physical flow of cash. Loomis operates across 13 countries including the U.S., Norway, Denmark, Austria and Portugal.

Founded: 2007

Focus: Online Document Storage + Secure File Sharing

What they do: Founded by Eric Pulaski, SmartVault has developed a platform that allows accountants and businesses to store, manage and securely share files. The companys platform enables users to create efficient, standardized workflows, automate manual paper-based tasks and eliminate security vulnerabilities. SmartVault integrates with a variety of apps including QuickBooks, DocuSign and TaxCalc.

Founded: 1988

Focus: IT Services

What they do: DataVox is a technology partner that helps organizations design, implement and maintain all aspects of their audio visual, cloud and data center. The company also specializes in cybersecurity, IT support and managed services, network cabling, phone systems, smart building technologies and physical security solutions.

Founded: 2002

Focus: Enterprise Content Management

What they do: Launched by Mike Alsup, Gimmal develops information management software that makes it possible for enterprises to manage information regardless of where it exists. The companys software captures, manages, governs and archives information, enabling businesses to manage the lifecycle of content in SharePoint, Office 365, Box, OneDrive for Business and other platforms. Gimmal is dedicated to helping organizations realize their digital workplace strategies more quickly and connect content with business transactions to improve efficiency and lower costs.

Founded: 2000

Focus: IT Services + Microsoft Consulting

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25 Tech Companies In Houston To Know - Built In

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Using induced pluripotent stem cells produced from the skin of a patient with a rare, genetic form of insulin-dependent diabetes calledWolfram syndrome, researchers transformed the human stem cells into insulin-producing cells and used the gene-editing tool CRISPR-Cas9 to correct a genetic defect that had caused the syndrome. They then implanted the cells into lab mice and cured the unrelenting diabetes in those mice.

The findings, from researchers at Washington University School of Medicine in St. Louis, suggest the CRISPR-Cas9 technique may hold promise as a treatment for diabetes, particularly the forms caused by a single gene mutation, and it also may be useful one day in some patients with the more common forms of diabetes, such as type 1 and type 2.

The study is published online April 22 in the journal Science Translational Medicine.

Patients with Wolfram syndrome develop diabetes during childhood or adolescence and quickly require insulin-replacement therapy, requiring insulin injections multiple times each day. Most go on to develop problems with vision and balance, as well as other issues, and in many patients, the syndrome contributes to an early death.

This is the first time CRISPR has been used to fix a patients diabetes-causing genetic defect and successfully reverse diabetes, said co-senior investigatorJeffrey R. Millman, PhD, an assistant professor of medicine and of biomedical engineering at Washington University. For this study, we used cells from a patient with Wolfram syndrome because, conceptually, we knew it would be easier to correct a defect caused by a single gene. But we see this as a stepping stone toward applying gene therapy to a broader population of patients with diabetes.

Wolfram syndrome is caused by mutations to a single gene, providing the researchers an opportunity to determine whether combining stem cell technology with CRISPR to correct the genetic error also might correct the diabetes caused by the mutation.

A few years ago, Millman and his colleagues discovered how to convert human stem cells into pancreatic beta cells. When such cells encounter blood sugar, they secrete insulin. Recently, those same researchers developed a new technique to more efficiently convert human stem cells into beta cells that are considerably better at controlling blood sugar.

In this study, they took the additional steps of deriving these cells from patients and using the CRISPR-Cas9 gene-editing tool on those cells to correct a mutation to the gene that causes Wolfram syndrome (WFS1). Then, the researchers compared the gene-edited cells to insulin-secreting beta cells from the same batch of stem cells that had not undergone editing with CRISPR.

In the test tube and in mice with a severe form of diabetes, the newly grown beta cells that were edited with CRISPR more efficiently secreted insulin in response to glucose. Diabetes disappeared quickly in mice with the CRISPR-edited cells implanted beneath the skin, and the animals blood sugar levels remained in normal range for the entire six months they were monitored. Animals receiving unedited beta cells remained diabetic. Their newly implanted beta cells could produce insulin, just not enough to reverse their diabetes.

We basically were able to use these cells to cure the problem, making normal beta cells by correcting this mutation, said co-senior investigatorFumihiko Urano, MD, PhD, the Samuel E. Schechter Professor of Medicine and a professor of pathology and immunology. Its a proof of concept demonstrating that correcting gene defects that cause or contribute to diabetes in this case, in the Wolfram syndrome gene we can make beta cells that more effectively control blood sugar. Its also possible that by correcting the genetic defects in these cells, we may correct other problems Wolfram syndrome patients experience, such as visual impairment and neurodegeneration.

In the future, using CRISPR to correct certain mutations in beta cells may help patients whose diabetes is the result of multiple genetic and environmental factors, such as type 1, caused by an autoimmune process that destroys beta cells, and type 2, which is closely linked to obesity and a systemic process called insulin resistance.

Were excited about the fact that we were able to combine these two technologies growing beta cells from induced pluripotent stem cells and using CRISPR to correct genetic defects, Millman said. In fact, we found that corrected beta cells were indistinguishable from beta cells made from the stem cells of healthy people without diabetes.

Moving forward, the process of making beta cells from stem cells should get easier, the researchers said. For example, the scientists have developed less intrusive methods, making induced pluripotent stem cells from blood and they are working on developing stem cells from urine samples.

In the future, Urano said, we may be able to take a few milliliters of urine from a patient, make stem cells that we then can grow into beta cells, correct mutations in those cells with CRISPR, transplant them back into the patient, and cure their diabetes in our clinic. Genetic testing in patients with diabetes will guide us to identify genes that should be corrected, which will lead to a personalized regenerative gene therapy.

Reference:

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Diabetes Reversed in Mice With CRISPR-Edited Stem Cells From Patients - Technology Networks

Recommendation and review posted by Bethany Smith

Municipalities must reexamine existing pet ordinances as a new state law takes effect.

A recently enacted law in the state of Washington sets a bold new standard for any county or city that restricts specific dog breeds. The state now mandates that if any dogno matter the breedpasses a canine good behavior test, local jurisdictions must exempt that dog from any breed-based regulation or ban.

The state law took effect on January 1, 2020 and has served as a catalyst for municipalities to consider whether regulations targeting specific dog breeds are in the best interest of public safety and fairness. Local governments throughout Washington have responded to the new law either by amending existing breed-based restrictions to create a good behavior exemption, or by abolishing breed regulations altogether.

Breed-specific legislation refers to laws, ordinances, and regulations that impose restrictions or bans on dogs of specific breeds. In the United States and in other countries, some governments completely ban residents from owning specific breeds of dogs. Others allow ownership but impose conditions such as requiring dogs of certain breeds to wear a muzzle in public at all times.

These regulations often target breeds such as American Pit Bull Terriers, Doberman Pinschers, and German Shepherds, as well as any mixed breed dog whose lineage includes a banned breed. And since enforcing a breed-specific regulation often requires animal control officers to identify a dogs breed visuallyrather than through genetic testing or breeding documentsthese laws regularly ensnare dogs that simply resemble a banned breed.

In total, more than 75 breeds are restricted or banned somewhere in the United States. In fact, over 700 jurisdictions in the United States have some form of breed-specific legislation, and federal law also prohibits several dog breeds from living on military bases.

Proponents of breed-specific legislation argue that certain dog breeds are inherently dangerous and that public safety improves when such dogs are not allowed in a community. Dog breed regulations are often enacted in jurisdictions after a dog bite incident receives extensive media coverage, prompting calls for government action against the breed of dog involved.

But opponents of breed-specific legislation maintain that breed bans cause significant negative impacts on good dogs and good people, with no measurable benefit to public safety. The American Kennel Club argues that breed laws are like racial profiling and that all they do is punish responsible dog owners without holding owners of truly dangerous dogs accountable. Owners of banned breeds often must choose between relocating to a different town or getting rid of their dog, increasing the number of dogs surrendered to and eventually euthanized by overcrowded animal shelters.

Instead of focusing on a dogs breed, the American Kennel Club argues that addressing the issue of irresponsible ownership is a much more effective method of animal control. A recent study of fatal dog attacks in the United States found that breed was not a statistically significant factor in dog bite cases. The American Society for the Prevention of Cruelty to Animals (ASPCA) opposes breed bans and states that no convincing data indicates this strategy has succeeded anywhere to date.

Instead, research has identified several owner-related risk factors that do contribute to dog aggression. These include dogs that are not neutered or spayed, have been isolated from positive human contact, or belong to owners with prior incidents of dog mismanagement, neglect, or abuse. When even one of these risk factors is present, fatal dog attacks are more likely to occur regardless of breed. When more than one risk factor is present, the likelihood of dog aggression further increases.

Organizations like the ASPCA and the American Kennel Club advocate public safety laws promoting responsible dog ownershipsuch as enhanced animal cruelty and fighting laws, licensing laws with a surcharge for unaltered pets, and low-cost spay and neuter programs. These groups favor regulations that clearly define dangerous behavior in all breeds and establish a fair process to determine whether a particular dog is dangerous.

In response to growing opposition to breed-specific legislation, the Washington state legislature passed House Bill 1026 last year, mandating that any local ordinance imposing breed-based requirements must include an exemption for dogs that pass the American Kennel Club canine good citizen test or an equivalent dog behavior test. The state law is similar to a previously existing regulation in the city of Omaha, Nebraska, which includes an exemption for well-behaved dogs in its breed-specific legislation. These types of regulations may not completely satisfy advocates on either side of the debate over dog-related risks, but they do reflect a balance between the authority of local governments and the individual rights of responsible dog owners.

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Rethinking the Regulation of Dog Breeds - The Regulatory Review

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COVID-19 is an infectious disease caused by SARS-CoV-2. The outbreak of COVID-19 started in December 2019 with the first case reported in China. The World Health Organization (WHO) recognized the outbreak of COVID-19 as a pandemic on 11 March 2020. By April 2020, there are over 2 million confirmed cases, and it has brought the economies of many countries to a halt.

Diagnostic testing is possibly the only efficient way to monitor the spread of the SARS-CoV-2 in time and space, enabling policymakers and healthcare workers to track and mitigate the outbreak of COVID-19. The WHO has appealed for global mass testing. The demand for COVID-19 testing is estimated to be over 600 million tests, including 120 million genetic tests and over 500 million rapid tests.

Molecules derived from the virusnucleic acids like RNA or DNA, or proteinsform the basis of diagnostics, as well as being essential for developing new therapies and vaccines. Depending on the target biomarkers, the diagnostic methods can be separated into two categories: genetic testing (detecting the viral genome) and serological & antigenic testing (detecting antibodies and viral antigens, respectively). From the technological perspective, molecular diagnostics (MDx) and lateral flow assays (LAFs) dominate COVID-19 diagnostics. The gold standard used across clinical laboratories is quantitative Reverse Transcription-Polymerase Chain Reaction (qPT-PCR, MDx), which requires a central lab setting. Such qRT-PCR testing lasts for more than 2 hours and the sample shipment cost up to several days. With the demand for quicker tests at community settings, the market is moving into point-of-care (POC) devices, including POC MDx and POC LFAs.

All molecular diagnostics tests detecting viral genomes share three common steps: sample collection from Nasopharyngeal swab and extraction of viral RNA, amplification of the analyte and read-out. The amplification step is performed reliably by RT-PCR. However, alternatives that do not require expensive and bulky equipment exist, i.e. isothermal amplification. This approach, although less sensitive than PCR, allows for a quicker amplification step at a constant temperature.

The read-out of the amplified signal is normally achieved through fluorescence probes in the sample and detectors in qRT-PCR devices. Many companies have resorted to lateral flow assays and alternative read-out methods that require proprietary detection equipment. These "hybrid systems" benefit from the high specificity and sensitivity of MDx and the speed and low cost of LFAs.

Apart from genetic testing, antigenic tests and serological tests, so-called rapid tests, are also becoming central tools in the fight against the pandemic. Both types of immunoassays rely on antibody-antigen recognition. Antigen tests are able to detect the presence of viral proteins in the blood sample. On the other hand, antibody tests detect the presence of antibodies against SARS-CoV-2, which are normally present in the blood sample after 7 days of infection and may remain for months or years. Antibody tests are an important tool to assess the extent of the pandemic and to identify the real number of cases and level of immunization in a population.

Rapid tests have been developed using lateral flow assay technology. LFAs tests are usually much faster and cheaper than qRT-PCR tests. It does not need expensive and large equipment. Therefore, it can be suitable for home testing and is the preferred choice for many governments to guide their response to the pandemic. However, their sensitivity and specificity can be far from ideal, as these tests lack a step of signal amplification, as opposed to qRT-PCR. Therefore, they measure directly the viral load or the antibody concentration. Such tests require extensive validation before widespread deployment.

Table 1: comparison of different commercial COVID-19 test devices, including quantitative Reverse Transcription-Polymerase Chain Reaction (qPT-PCR, genetic testing, using PCR amplification and fluorescent detection), point-of-care molecular diagnostics (POC MDx, genetic testing, using both PCR or isothermal amplification), lateral flow assays (LFAs, immunoassays, no amplification steps, using colorimetric or fluorescent method for detection), and Hybrid systems (genetic testing, using eight PCR amplification or isothermal amplification, and LFAs or other method for detection).

Apart from the effort from biotech, multiple software companies have developed algorithms to identify signs of COVID-19-related pneumonia in patient scans. CT imaging is an effective way of detecting abnormalities indicative of COVID-19, and image recognition AI algorithms have the potential to detect these abnormalities faster and more efficiently than radiologists.

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COVID-19 Diagnostics: Technologies, Players and Trends - IDTechEx.com

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Updated April 23, 2020 20:02:27

Australia's Chief Medical Officer says a coronavirus cluster in Tasmania's north-west was likely sparked by a passenger from the Ruby Princess cruise ship.

Tasmania has 205 confirmed coronavirus cases, 131 of which are in the north-west, and health authorities confirmed late on Thursday night that no new cases were recorded in the 24 hours since 6:00pm on Wednesday night.

Chief Medical Officer Brendan Murphy previously came under fire for his comments that north-west healthcare workers had attended an "illegal dinner party" together, a claim rebuked by Tasmania's Premier as a "rumour".

Speaking on Thursday to a Senate committee examining Australia's COVID-19 response, Professor Murphy said the Tasmanian cluster was "a very good example of just how infectious this virus is".

"Tasmanian Health have been investigating it, obviously, and I haven't seen the final report, but it seems likely that healthcare workers picked up the virus probably from a Ruby Princess passenger who was being cared for," he said.

"It just shows once you have someone who is infected who might not know they're infected, they can spread it to a lot of other people before anyone knows."

Tasmania's Director of Public Health Dr Mark Veitch has previously said two Ruby Princess passengers who died at the North West Regional Hospital were considered potential sources of the outbreak.

However, he said investigators would wait for the results of genetic testing of the virus strain involved in the outbreak before making their conclusions.

An interim report on the outbreak is expected to be handed to the Tasmanian Government within days.

More than 10 per cent of Australia's coronavirus cases have been linked to the Ruby Princess, which docked in Sydney on March 19.

Meanwhile, the Tasmanian Government has warned north-west residents the additional coronavirus restrictions placed on the area may not be lifted as soon as hoped.

The increased restrictions, which saw non-essential retailers such as Harvey Norman, Kmart and Target shut down, are due to expire on Sunday evening.

Testing criteria are different for north-west residents.

Need an interpreter?Phone the Translating and Interpreting Service on 131 450 and tell them your language.

For more information and factsheets:Visit the Tasmanian Government's coronavirus page here.

Premier Peter Gutwein said he wanted north-west residents to be ready for the possibility they may be extended.

"[Lifting restrictions] may not occur for another week," he said.

"No final decision has been made yet, but I think it's important that people start thinking about what their circumstances will be should those restrictions not be lifted.

"I specifically want north-west parents to begin thinking about how they might manage their children next week if they are at home because if we aren't able [to lift restrictions] obviously schools may not be able to open across the north-west as previously planned," he said.

Mr Gutwein said the "plan had worked so far, however four additional cases [confirmed last night] is four additional positive tests too many".

"We do need to ensure that we work through this sensibly and not lift those restrictions too early," he said.

"However later today I'll be taking advice from public health and we'll be working through the scenarios planning in terms of the way forward and a final view will be formed later this afternoon based on that advice."

An announcement on those restrictions is expected tomorrow.

The Premier also urged Tasmanians in the north and the south of the state to remain vigilant.

"The response to date in terms of our actions has meant that we haven't seen cases [in the north and south] for some time, but it's important that people continue to follow the rules," he said.

"This is not a game, this is serious, at the end of the day this virus kills people."

It has been a week since the last case in the south and the north has not seen a case since April 4.

Despite this, Health Minister Sarah Courtney said there were plans to expand testing criteria in the north and the south.

"We are looking to further expand our testing criteria for those regions in coming days to people in those areas that area also experiencing respiratory symptoms," she said.

"We'll be writing to GPs today around the expanded guidelines so if you are in the north or south, please be aware of your symptoms."

Of Tasmania's 205 coronavirus cases, 91 have recovered.

Topics:health,diseases-and-disorders,covid-19,epidemics-and-pandemics,tas,launceston-7250,hobart-7000

First posted April 23, 2020 12:46:28

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No new cases of coronavirus for Tasmania, as north-west cluster blamed on Ruby Princess - ABC Local

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HAMBURG, GERMANY / ACCESSWIRE / April 22, 2020 / Evotec SE (Frankfurt Stock Exchange: EVT, MDAX/TecDAX, ISIN: DE0005664809) announced today that it will regain global development and commercialisation rights to the iPSC-based programme for the treatment of diabetes developed under collaboration agreement with Sanofi.

Evotec has built a unique platform for iPSC-based drug discovery and cell therapy covering the generation of iPS cell lines, up to cell manufacturing of various cell types for drug screening as well as GMP production of clinical material for cell therapies. Evotec produces human beta cells in islet-like clusters from a GMP-compliant iPS cell line in a scalable bioreactor format, with extensive quality control ("QC") procedures. The beta cell programme has already achieved pre-clinical data demonstrating that they are functionally equivalent to primary human islets in their ability to normalise blood glucose levels in in vivo models over several months.

Evotec will continue the development of the beta cell programme on its own within its EVT Innovate initiative "QRbeta Therapeutics". In parallel, Evotec will explore the best strategic options for further long-term development and commercialisation. An off-the-shelf beta cell therapy product has the potential to revolutionise the treatment of insulin-dependent diabetic patients and therefore could represent a major therapeutic opportunity.

Dr Cord Dohrmann, Chief Scientific Officer of Evotec, commented: "Evotec and Sanofi have developed the beta cell replacement therapy programme since 2015 in a highly productive partnership. During this time, we have made tremendous progress towards bringing a potentially game-changing treatment option to the clinic. We would like to thank Sanofi for the collaboration and its contributions. Regaining full control of this innovative and promising programme to treat diabetes is of great value for Evotec. While we are continuing to move this programme forward, we are exploring partnering options to bring this therapy to patients."

About DiabetesDiabetes mellitus ("diabetes") is a chronic incapacitating disease associated with severe lifelong conditions which require intensive monitoring and control, such as cardiovascular diseases, kidney diseases, nerve damage and eye diseases. At present, there is no cure for diabetes and only symptomatic treatment options are available. According to the International Diabetes Federation, it is estimated that 463 million people worldwide suffered from diabetes in 2019 (2017:425 million) and this number is projected to reach 578 million by 2030. The disease is a major burden to the global healthcare systems with about $ 760 bn being spent on the treatment of diabetes in 2019 and it is projected that expenditure will reach $ 825 bn by 2030.

About Beta CellsBeta cells play a key role in the pathogenesis of diabetes. Beta cells reside in clusters of hormone producing cells ("islets") within the pancreas. They respond to elevated blood glucose levels (e.g. after a meal) by secreting the glucose lowering hormone insulin. In the type 1 form of diabetes ("T1D"), beta cells are destroyed by the patient's own immune system. As a result, T1D patients must follow a life-long regimen of carefully dosed insulin injections. In patients with type 2 diabetes ("T2D"), beta cells are functionally impaired and yet have to work in the presence of metabolic stress and increased workload due to an impaired tissue insulin response. T2D is progressive, and current therapeutic options cannot prevent the deterioration of beta cell function, eventually also creating a need for insulin injections. Despite the fact that insulin treatments are important and widely used for people with diabetes, they cannot fully mimic the normal control of blood glucose levels by normal beta cells necessary to avoid acute and long-term complications of diabetes. There is a critical medical need for novel therapeutic options which can restore beta cell mass and, thereby, reduce or eliminate the need for insulin injections. Furthermore, beta cell replacement therapy also has the potential to prevent or reverse the decline in beta cell function in type 2 diabetes.

ABOUT EVOTEC SEEvotec is a drug discovery alliance and development partnership company focused on rapidly progressing innovative product approaches with leading pharmaceutical and biotechnology companies, academics, patient advocacy groups and venture capitalists. We operate worldwide and our more than 3,000 employees provide the highest quality stand-alone and integrated drug discovery and development solutions. We cover all activities from target-to-clinic to meet the industry's need for innovation and efficiency in drug discovery and development (EVT Execute). The Company has established a unique position by assembling top-class scientific experts and integrating state-of-the-art technologies as well as substantial experience and expertise in key therapeutic areas including neuronal diseases, diabetes and complications of diabetes, pain and inflammation, oncology, infectious diseases, respiratory diseases, fibrosis, rare diseases and women's health. On this basis, Evotec has built a broad and deep pipeline of approx. 100 co-owned product opportunities at clinical, pre-clinical and discovery stages (EVT Innovate). Evotec has established multiple long-term alliances with partners including Bayer, Boehringer Ingelheim, Bristol-Myers Squibb, CHDI, Novartis, Novo Nordisk, Pfizer, Sanofi, Takeda, UCB and others. For additional information please go to http://www.evotec.com and follow us on Twitter @Evotec.

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FORWARD LOOKING STATEMENTSInformation set forth in this press release contains forward-looking statements, which involve a number of risks and uncertainties. The forward-looking statements contained herein represent the judgement of Evotec as of the date of this press release. Such forward-looking statements are neither promises nor guarantees, but are subject to a variety of risks and uncertainties, many of which are beyond our control, and which could cause actual results to differ materially from those contemplated in these forward-looking statements. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any such statements to reflect any change in our expectations or any change in events, conditions or circumstances on which any such statement is based.

Contact Evotec SE:Gabriele Hansen, SVP Corporate Communications, Marketing & Investor Relations, Phone: +49.(0)40.56081-255, gabriele.hansen@evotec.com

SOURCE: Evotec AG via EQS Newswire

View source version on accesswire.com: https://www.accesswire.com/586314/Evotec-Regains-Global-Rights-to-Beta-Cell-Replacement-Therapy

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Evotec Regains Global Rights to Beta Cell Replacement Therapy - Yahoo Finance

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CRISPR corrects genetic defect so cells can normalize blood sugar

Researchers at Washington University School of Medicine in St. Louis have transformed stem cells into insulin-producing cells. They used the CRISPR gene-editing tool to correct a defect that caused a form of diabetes, and implanted the cells into mice to reverse diabetes in the animals. Shown is a microscopic image of insulin-secreting beta cells (insulin is green) that were made from stem cells produced from the skin of a patient with Wolfram syndrome.

Using induced pluripotent stem cells produced from the skin of a patient with a rare, genetic form of insulin-dependent diabetes called Wolfram syndrome, researchers transformed the human stem cells into insulin-producing cells and used the gene-editing tool CRISPR-Cas9 to correct a genetic defect that had caused the syndrome. They then implanted the cells into lab mice and cured the unrelenting diabetes in those mice.

The findings, from researchers at Washington University School of Medicine in St. Louis, suggest the CRISPR-Cas9 technique may hold promise as a treatment for diabetes, particularly the forms caused by a single gene mutation, and it also may be useful one day in some patients with the more common forms of diabetes, such as type 1 and type 2.

The study is published online April 22 in the journal Science Translational Medicine.

Patients with Wolfram syndrome develop diabetes during childhood or adolescence and quickly require insulin-replacement therapy, requiring insulin injections multiple times each day. Most go on to develop problems with vision and balance, as well as other issues, and in many patients, the syndrome contributes to an early death.

This is the first time CRISPR has been used to fix a patients diabetes-causing genetic defect and successfully reverse diabetes, said co-senior investigator Jeffrey R. Millman, PhD, an assistant professor of medicine and of biomedical engineering at Washington University. For this study, we used cells from a patient with Wolfram syndrome because, conceptually, we knew it would be easier to correct a defect caused by a single gene. But we see this as a stepping stone toward applying gene therapy to a broader population of patients with diabetes.

Wolfram syndrome is caused by mutations to a single gene, providing the researchers an opportunity to determine whether combining stem cell technology with CRISPR to correct the genetic error also might correct the diabetes caused by the mutation.

A few years ago, Millman and his colleagues discovered how to convert human stem cells into pancreatic beta cells. When such cells encounter blood sugar, they secrete insulin. Recently, those same researchers developed a new technique to more efficiently convert human stem cells into beta cells that are considerably better at controlling blood sugar.

In this study, they took the additional steps of deriving these cells from patients and using the CRISPR-Cas9 gene-editing tool on those cells to correct a mutation to the gene that causes Wolfram syndrome (WFS1). Then, the researchers compared the gene-edited cells to insulin-secreting beta cells from the same batch of stem cells that had not undergone editing with CRISPR.

In the test tube and in mice with a severe form of diabetes, the newly grown beta cells that were edited with CRISPR more efficiently secreted insulin in response to glucose. Diabetes disappeared quickly in mice with the CRISPR-edited cells implanted beneath the skin, and the animals blood sugar levels remained in normal range for the entire six months they were monitored. Animals receiving unedited beta cells remained diabetic. Their newly implanted beta cells could produce insulin, just not enough to reverse their diabetes.

We basically were able to use these cells to cure the problem, making normal beta cells by correcting this mutation, said co-senior investigator Fumihiko Urano, MD, PhD, the Samuel E. Schechter Professor of Medicine and a professor of pathology and immunology. Its a proof of concept demonstrating that correcting gene defects that cause or contribute to diabetes in this case, in the Wolfram syndrome gene we can make beta cells that more effectively control blood sugar. Its also possible that by correcting the genetic defects in these cells, we may correct other problems Wolfram syndrome patients experience, such as visual impairment and neurodegeneration.

In the future, using CRISPR to correct certain mutations in beta cells may help patients whose diabetes is the result of multiple genetic and environmental factors, such as type 1, caused by an autoimmune process that destroys beta cells, and type 2, which is closely linked to obesity and a systemic process called insulin resistance.

Were excited about the fact that we were able to combine these two technologies growing beta cells from induced pluripotent stem cells and using CRISPR to correct genetic defects, Millman said. In fact, we found that corrected beta cells were indistinguishable from beta cells made from the stem cells of healthy people without diabetes.

Moving forward, the process of making beta cells from stem cells should get easier, the researchers said. For example, the scientists have developed less intrusive methods, making induced pluripotent stem cells from blood and they are working on developing stem cells from urine samples.

In the future, Urano said, we may be able to take a few milliliters of urine from a patient, make stem cells that we then can grow into beta cells, correct mutations in those cells with CRISPR, transplant them back into the patient, and cure their diabetes in our clinic. Genetic testing in patients with diabetes will guide us to identify genes that should be corrected, which will lead to a personalized regenerative gene therapy.

Maxwell KG, Augsornworawat P, Velazco-Cruz L, Kim MH, Asada R, Hogrebe NJ, Morikawa S, Urano F, Millman JR. Gene-edited human stem cell-derived cells from a patient with monogenic diabetes reverse pre-existing diabetes in mice. Science Translational Medicine, published online April 22, 2020.

This work was supported by the National Institute of Diabetes and Digestive and Kidney Diseases, the National Institute of General Medical Sciences, the National Cancer Institute and the National Center for Advancing Translational Sciences of the National Institutes of Health (NIH). Grant numbers R01 DK114233, DK112921, TR002065, TR002345, T32 DK108742, R25 GM103757, T32 DK007120, P30 DK020579, P30 CA91842, UL1 TR000448 and UL1 TR002345. Additional assistance was provided by the Washington University Genome Engineering and iPSC Center, the Washington University Diabetes Center, and the Washington University Institute of Clnical and Translational Science, with additional funding from the JDRF, the Washington University Center of Regenerative Medicine, startup funds from the Washington University School of Medicine Department of Medicine, the Unravel Wolfram Syndrome Fund, Silberman Fund, Stowe Fund, Ellie White Foundation for Rare Genetic Disorders, Eye Hope Foundation, Snow Foundation, Feiock Fund, Childrens Discovery Institute, Manpei Suzuki Diabetes Foundation, and a JSPS Overseas Research Fellowship.

Washington University School of Medicines 1,500 faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Childrens hospitals. The School of Medicine is a leader in medical research, teaching and patient care, ranking among the top 10 medical schools in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Childrens hospitals, the School of Medicine is linked to BJC HealthCare.

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Diabetes reversed in mice with genetically edited stem cells derived from patients - Washington University School of Medicine in St. Louis

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Whether you're still completing a lengthy beauty routine each morning or your beloved products are collecting dust, there's one base makeup item that both can simplify things and persuade the fresh-faced crew to change their tune: BB cream. First developed in South Korea, this all-in-one product can replace your foundation and also your serum, moisturizer, primer, and sometimes even sunblock saving you time, money, and effort.

"Beauty balms" (i.e., "BB") are typically more sheer than traditional foundations (and even most tinted moisturizers), and are easy to apply with a brush, sponge, or even your fingers. Nearly all formulas include some form of SPF to protect against the sun, as well as active ingredients to help treat common skin concerns. Now, this versatility could explain why they seem to be flying off the (virtual) shelves at Sephora, but we picked out 12 popular options that get an A+ from us and other shoppers, too.

Here are the top-rated BB creams you can still nab at Sephora ahead.

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12 Top-Rated BB Creams That Can Simplify Your Beauty Routine and Protect Skin, Too - POPSUGAR

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Reprinted with permission fromWorld At Large, a news website which covers politics, nature, science, health, and travel.

Age-related macular degeneration (MD) is a variety of ocular disease that affects so many millions of people, its downright characterized as nothing more than the process of aging.

In fact, the study of MD has proven itself to be so fruitful for understanding aging as a whole, MD has become one of the first places that scientists have looked to in order to attempt to repair the damage of aging and restoring youthful, or normal function.

Now in a new paper published in Nature, researchers demonstrate an alternative to stem cells by creating replacement photoreceptors from skin fibroblast cells via pharmacological-conversiona process that promises to be cheaper, faster, and unburdened by ethical and legal restrictions.

Containing the genetic code for every protein we need to build and repair our cells, DNA can become damaged over time through the stresses of life. Similarly to how scratches or cracks in a CD prevent lasers from reading the information on the disk, damaged DNA becomes difficult, and even impossible for our RNA (which you could imagine as the laser inside the CD player) to read the genetic information contained therein.

WATCH: Blind Man Develops Smart Cane That Uses Google Maps and Sensors to Identify Ones Surroundings

The photoreceptor is the neuron in the eye that turns on visual circuity in response to light which enables us to have vision, says Sai Chavala, Ph.D. and author of the new paper.

The loss of photoreceptors can result in MD and other retinal diseases that lead to irreversible blindness. In this new study, however, cells called fibroblasts can be chemically reprogrammed to produce photoreceptor-like cells that are now shown to restore vision in mice.

Fibroblasts are cells that help maintain the structural integrity of connective tissues, and a reduction in fibroblast cell count leads to wrinkled skin.

Sai Chavala and colleagues from the Center for Retina Innovation in Dallas, Texas, found a set of five compounds that can drive embryonic fibroblast cells to convert themselves into retinal, rod-like, photoreceptors in both mice and human cells.

Stem cell-based strategies are extremely exciting, Dr. Chavala tells World at Large, but adds that generating these cells can be cumbersome and time-consuming. Describing his strategy of chemically reprogramming existing cells as a breakthrough, Chavala added that the generation of the photoreceptors using his method requires a fraction of the time.

In the study, these converted photoreceptors were transplanted into the eyes of 14 blind mice to see if they would restore vision.

CHECK OUT: LEGO Unveils New Bricks That Will Help Teach Blind Children to Read Braille

Owing to their nature, mice with working vision prefer dark spaces, and so a test of whether or not the transplant was a success was fairly straight forward; create a bright space and a dark space and wait to see which one was preferred by the mice.

Six mice were found to not only have restored visual function and reflexesnotably in the pupilbut they also preferred to spend their time in the dark space. Chavala hypothesizes that it could have been down to how many of the chemically-reprogrammed cells survived the transplant into the retina that determined whether vision was restored in the mice or not.

What makes interventions like thesewhich involve creating brand new cells to replace the damaged onesso effective and exciting in the field of aging is that they present an alternative to natural replication by bypassing the difficulties that our own cells have with trying to repair themselves from DNA that features double-strand breaks or other blemishes.

Adding that these conversions of fibroblasts to photoreceptors has also been done in humans, Chavala detailed why he believes the treatments and future research to spring from this discovery are going to change the field of ophthalmology.

CHECK OUT: In World First Blind People Have Their Vision Restored Thanks to Stem Cells From Deceased Organ Donors

We [] believe this can be a game changer in the field of regenerative ophthalmology. We also believe this is a platform technology and have already started establishing protocols to generate retinal ganglion cells valuable for patients suffering from glaucoma, says Chavala.

In addition to bypassing the ethical and political restrictions and hiccups with using embryonic human stem cells, the process takes two weeks, costs less, and is more scalable than using stem cells.

WATCH: Hundreds of People Are Being Cured of Blindness Every Day With Cheap, Minutes-Long Surgery

It is intriguing to postulate that the addition of other molecules or modifications to the culture conditions can yield other types of neurons beneficial for a variety of neurological diseases, he adds, discussing possible alternatives to photoreceptor generation from fibroblasts.

Finally, Chavala is rapidly approaching human trials and hopes to have an FDA-approved treatment out the other end of the pipeline in 2-3 years, saying he is thrilled with the possibility of allowing millions of people to regain their lost vision.

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Irreversible No Longer: Blind Mice See Again Thanks To New Method of Synthesizing Lost Cells - Good News Network

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New research results show promise in treating people who are blind.

The National Eye Institute funded the study, which is research considered to be ethical.

Dr. David Prentice of the Charlotte Lozier Institute says there have been discussions over using adult stem cells to restore sight, which he calls a different tack for advancing science and medicine.

It's still an ethical way to go about this, he observes. There's no embryonic stem cells, no fetal tissue, none of this unethical type of research direction.

What the scientists did was turn a skin cell directly into a photoreceptor for vision then transplanted it.

Prenticeadvises the testing is very preliminary after the experiment on mice.

But what they find was when they transplanted this newly formed type of vision cell into the eyes of these blind mice, he says, they restored their vision.

The researchers applied chemicals that transformed one cell type into another needed for vision, and there is now potential to help people with all forms of vision blindness or vision correction, which would include macular degeneration and other retinal disorders.

Editor's note: Original posting attribute comments to wrong person.

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Lozier praises promising, and ethical, blindness study - OneNewsNow

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- Phase 3 REACH2 data demonstrate 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)

- Results show a significantly greater overall response rate (ORR) in patients treated with ruxolitinib (62%) compared to BAT (39%) 1,2

- GVHD is a serious and common complication of allogeneic stem cell transplants with a one-year mortality rate as high as 80% in patients who develop acute GVHD3-5

- The results, published in The New England Journal of Medicine, were also selected for an oral presentation during 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

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.

Story continues

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

View source version on businesswire.com: https://www.businesswire.com/news/home/20200422005739/en/

Contacts

Incyte Contacts Media Jenifer Antonacci+1 302 498 7036jantonacci@incyte.com

Catalina Loveman+1 302 498 6171cloveman@incyte.com

Investors Michael Booth, DPhil+1 302 498 5914mbooth@incyte.com

Read more:
Incyte Announces Pivotal REACH2 Study Data Published in NEJM Highlight Superior Efficacy of Ruxolitinib (Jakafi) versus Best Available Therapy in...

Recommendation and review posted by Bethany Smith

No matter how many creams and other topical treatments we try, the cellulite on our bodies just doesnt seem to go away. Of course, its very difficult to get rid of cellulite completely but many strive to reduce its appearance as much as possible.

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This body cream uses a complex known as TFC8, which stands for Trigger Factor Complex. Its a patented formula that was developed by Professor Augustinus Bader, a top biomedical scientist, over the past three decades. This complex is said to help awaken stem cells that have been dormant, which ramps up the renewal process. This results in firmer skin, which can help quickly diminish the appearance of cellulite. This cream can also help even out tone, reduce sun spots and make the skin appear renewed and more youthful.

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This post is brought to you by Us Weeklys Shop With Us team. The Shop With Us team aims to highlight products and services our readers might find interesting and useful. Product and service selection, however, is in no way intended to constitute an endorsement by either Us Weekly or of any celebrity mentioned in the post.

The Shop With Us team may receive products free of charge from manufacturers to test. In addition, Us Weekly receives compensation from the manufacturer of the products we write about when you click on a link and then purchase the product featured in an article. This does not drive our decision as to whether or not a product or service is featured or recommended. Shop With Us operates independently from advertising sales team. We welcome your feedback at ShopWithUs@usmagazine.com. Happy shopping!

Continue reading here:
This Award-Winning Body Cream Can Reduce the Appearance of Cellulite by 75% - Us Weekly

Recommendation and review posted by Bethany Smith

Insulin injections cancontrol diabetes, but patients still experience serious complications such as kidney disease and skin infections. Transplanting pancreatic tissues containing functional insulin-producing beta cells is of limited use, because donors are scarce and patients must take immunosuppressant drugs afterward.

Now, scientists atWashington University in St. Louis havedeveloped a way to use gene editing system CRISPR-Cas9 to edit a mutation in human-induced pluripotent stem cells (iPSCs) and then turnthem into beta cells. When transplanted into mice, the cells reversed preexisting diabetes in a lasting way, according to results published in the journal Science Translational Medicine.

While the researchers used cells from patients with Wolfram syndromea rare childhood diabetes caused by mutations in the WFS1 genethey argue that the combination of a gene therapy with stem cells could potentially treat other forms of diabetes as well.

Virtual Clinical Trials Online

This virtual event will bring together industry experts to discuss the increasing pace of pharmaceutical innovation, the need to maintain data quality and integrity as new technologies are implemented and understand regulatory challenges to ensure compliance.

One of the biggest challenges we faced was differentiating our patient cells into beta cells. Previous approaches do not allow for this robust differentiation. We use our new differentiation protocol targeting different development and signaling pathways to generate our cells, the studys lead author, Kristina Maxwell, explained in a video statement.

Making pancreatic beta cells from patient-derived stem cells requires precise activation and repression of specific pathways, and atthe right times, to guide the development process. In a recent Nature Biotechnology study, the team described a successful method that leverages the link between a complex known as actin cytoskeleton and the expression of transcription factors that drive pancreatic cell differentiation.

This time, the researchers applied the technology to iPSCs from two patients with Wolfram syndrome. They used CRISPR to correct the mutated WFS1 gene in the cells and differentiated the edited iPSCs into fully functional beta cells.

After transplanting the corrected beta cells into diabetic mice, the animals saw their blood glucose drop quickly, suggesting the disease had been reversed. The effect lasted for the entire six-month observation period, the scientists reported. By comparison, those receiving unedited cells from patients were unable to achieve glycemic control.

RELATED:CRISPR Therapeutics, ViaCyte team up on gene-edited diabetes treatment

The idea of editing stem cells with CRISPR has already attracted interest in the biopharma industry. Back in 2018, CRISPR Therapeutics penned a deal with ViaCyte to develop off-the-shelf, gene-editing stem cell therapies for diabetes. Rather than editing iPSCs from particular patients themselves to correct a faulty gene, the pairs lead project used CRISPR to edit healthy cells so that they lackedthe B2M gene and expressed PD-L1 to protect against immune attack. The two companies unveiled positive preclinical data inSeptember.

Other research groups working on gene therapy or stem cells for diabetes include a Harvard University scientist and his startup Semma Therapeutics, whichdeveloped a method for selecting beta cells out of a mixture of cells developed from PSCs. Scientists at the University of Wisconsin-Madison recently proposed that removing the IRE1-alpha gene in beta cells could prevent immune T cells from attacking them in mice with Type 1 diabetes.

The Washington University team hopes its technology may help Type 1 diabetes patients whose disease is caused by multiple genetic and environmental factors as well as the Type 2 form linked to obesity and insulin resistance.

We can generate a virtually unlimited number of beta cells from patients with diabetes to test and discover new drugs to hopefully stop or even reverse this disease, Jeffrey Millman, the studys co-senior author, said in the video statement. Perhaps most importantly, this technology now allows for the potential use of gene therapy in combination with the patients own cells to treat their own diabetes by transplantation of lab-grown beta cells.

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Reversing diabetes with CRISPR and patient-derived stem cells - FierceBiotech

Recommendation and review posted by Bethany Smith

Glaucoma can be successfully treated with gene therapy

London, April 22 (IANS) A common eye condition, glaucoma, could be successfully treated with a single injection using gene therapy, which would improve treatment options, effectiveness and quality of life for many patients, say researchers.

Glaucoma affects over 64 million people worldwide and is a leading cause of irreversible blindness. It is usually caused by fluid building up in the front part of the eye, which increases pressure inside the eye and progressively damages the nerves responsible for sight.

Current treatments include either eye drops, laser or surgery, all of which have limitations and disadvantages.

"At present, there is no cure for glaucoma, which can lead to loss of vision if the disease is not diagnosed and treated early," said study researcher Dr Colin Chu from the University of Bristol in the UK.

For the findings, published in the journal Molecular Therapy, the research team tested a new approach that could provide additional treatment options and benefits.

The researchers designed a gene therapy and demonstrated proof of concept using experimental mouse models of glaucoma and human donor tissue.

The treatment targeted part of the eye called the ciliary body, which produces the fluid that maintains pressure within the eye.

Using the latest gene-editing technology called CRISPR, a gene called Aquaporin 1 in the ciliary body was inactivated leading to reduced eye pressure.

"We hope to advance towards clinical trials for this new treatment in the near future. If it''s successful it could allow a long-term treatment of glaucoma with a single eye injection, which would improve the quality of life for many patients whilst saving the NHS time and money," Chu said

The researchers are currently in discussion with industry partners to support further laboratory work and rapidly progress this new treatment option towards clinical trials.

--IANS

bu/na

Disclaimer :- This story has not been edited by Outlook staff and is auto-generated from news agency feeds. Source: IANS

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Glaucoma can be successfully treated with gene therapy - Outlook India

Recommendation and review posted by Bethany Smith