Monthly Archives: June 2022

Top 10 Hormone Replacement Therapy Industry to Look Out for in 2022 by Abbott Laboratories, Novartis, Pfizer, Inc., Mylan Laboratories – Digital…

Posted: June 22, 2022 at 2:12 am

Hormone Replacement Therapy Market to 2022 Updated with Impact of COVID-19 is latest research study released by Adroit Market Research evaluating the market, highlighting opportunities, risk side analysis, and leveraged with strategic and tactical decision-making support. The study provides information on market trends and development, drivers, capacities, technologies, and on the changing investment structure of the Hormone Replacement Therapy Market to 2030 Updated with Impact of COVID-19 Market.

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Hormone Replacement Therapy Market to 2030 Updated with Impact of COVID-19 Industry Overview:

The Brainy Insights Hormone Replacement Therapy Market to 2030 Updated with Impact of COVID-19 provides a comprehensive coverage on Hormone Replacement Therapy industry. It provides historical and forecast data on the countrys coal production, consumption, and imports. The production section provides an extensive analysis over the trend of production, impact of the COVID-19 and information on production by company, by type, by grade and by state. The report also provides detail for reserves by state and country. The trade section briefs about major partners involve in this market. An extensive demand drivers section provides information on factors that are affecting the countrys coal demand such as domestic demand from power and steel industry. It further includes profiles of producers, information on the major active, planned and exploration projects.

Important years considered in the study are: Historical year 2015-2022; Base year 2022; Forecast period** 2022 to 2030 [** unless otherwise stated]

Market segments and sub-segments:

Type such as

Application such as

Hormone Replacement Therapy Market Scope:

The report contains an overview of Hormone Replacement Therapy industry and the impact of COVID-19 on the countrys Hormone Replacement Therapy market. It also includes key driving factors that affects global demand such as demand from the application industry.

It provides detailed information on reserves by country, production, production by state, company, type and grade. Along with this, major operating, exploration and development projects, competitive landscape and major importers are also included in the report.

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Reasons to Buy:

Key Answers Captured in Study are

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University of Medicine and Health Sciences to Host "LGBTQ+ Medicine & Theory: Providing Compassionate Care" – Yahoo Finance

Posted: June 22, 2022 at 2:12 am

Top-rated Caribbean Med School offers the latest research, insights, and resources for med students, healthcare providers, and allies

NEW YORK, June 21, 2022 /PRNewswire-PRWeb/ -- The University of Medicine and Health Sciences, (UMHS), a small, mission-driven medical school with a commitment to student support and a legacy of successful residency placements in the United States and Canada, today announced that it will host a live stream event, "LGBTQ+ Medicine & Theory: Providing Compassionate Care," on Wednesday, June 22 at 7 pm EDT. The discussion will be led by UMHS alumnus Soren Estvold, MD, MPH, a family medicine physician who specializes in treating LGBTQ+ patients at Augusta University Medical Center in Georgia and a volunteer physician at the Equality Clinic in Augusta, Georgia, a primary care clinic serving mostly transgender patients needing Hormone Replacement Therapy. The event will address key considerations for working with LGBTQ+ patients, describe how to provide compassionate care, offer practical advice for medical professionals and allies, and share resources for patients and providers. Following the presentation, current UMHS student Nisha Shetty will moderate a live Q & A session from the campus on St. Kitts. The event will be live-streamed on the UMHS YouTube channel as well as on the UMHS Facebook and LinkedIn pages. The presentation will also be recorded for future viewing.

"We are excited to welcome Dr. Estvold back for a presentation focused on the unique healthcare needs of the LGBTQ+ population at a time when this community is facing renewed attacks and barriers to receiving medical care," said Warren Ross, president of UMHS. "We're proud of Dr. Estvold's accomplishments in LGBTQ+ medicine, and are honored that he has once again agreed to share his insights with our students and offer specific guidance to deliver better health outcomes for LGBTQ+ patients."

Story continues

During the "LGBTQ+ Medicine & Theory" event, Dr. Estvold will define "full-spectrum medicine" and highlight the unique healthcare considerations of each subgroup within the community. He will also address the gender minority stress framework and how that impacts LGBTQ+ patients. Additionally, Dr. Estvold will offer insights on how to create an LGBTQ+-friendly practice and share advice for students interested in pursuing a specialty in LGBTQ+ medicine.

The discussion is the latest in a series of live stream events featuring UMHS faculty and alumni sharing their expertise on topics targeted toward current and prospective medical students and healthcare professionals. Past events include:

"Pathways to Practicing Medicine in Canada: UMHS Alumni Share Their Experiences"

"UMHS Women in Medicine: A Conversation About the First Year of Residency,"

"Cardiology: A Discussion About Cardiac Care & Careers in Cardiology,"

"Non-Traditional Medical Students - Medical School Admissions and Residency Advisors Reveal All!",

"Black Women in Medicine: A Conversation About the Black Experience",

"Ask a Microbiologist,"

"Suicide Prevention and the State of Psychiatry." and

"LGBTQ+ Medicine and Theory."

Links to view all past discussions may be found by visiting this link.

To join "LGBTQ+ Medicine & Theory: Providing Compassionate Care," on Wednesday, June 22, at 7 pm EDT visit the UMHS live events and meetings page.

About UMHS The University of Medicine and Health Sciences (UMHS), is a small, mission-driven medical school with a commitment to student support and a legacy of successful residency placements in the United States and Canada. UMHS was founded in 2007 by medical education pioneers Warren and Robert Ross to deliver a highly personalized school experience. Graduates of UMHS earn a Doctor of Medicine degree (MD) and qualify to practice medicine throughout the United States and Canada. Students begin their Basic Science studies in St. Kitts, West Indies, and complete their clinical training in the United States. With an unprecedented 96% student retention rate, the vast majority of students that begin their medical studies at UMHS go on to obtain residencies. For more information, visit https://www.umhs-sk.org/.

Media Contact

Megan Leer, UMHS, 619-708-9500, meganleerpr@gmail.com

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SOURCE University of Medicine and Health Sciences

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University of Medicine and Health Sciences to Host "LGBTQ+ Medicine & Theory: Providing Compassionate Care" - Yahoo Finance

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International Yoga Day: Womens health & yoga – Times of India

Posted: June 22, 2022 at 2:12 am

Anybody can breathe therefore anyone can practice yoga.T.K.V. Desikachar, yoga guru

Yoga is thousands of year-old discipline to engender mind-body harmony. Most of its development was between 500 BCE and 800 CE. From the 1970s, yoga spread worldwide and has become part of urban culture. The three main practices of Yoga are asana (posture), pranayama (breath control) and dhyana (meditation). Modern Yoga, or yoga in the modern age, is a combination of asanas and gymnastics.

Yoga was a spiritual practice. But in recent times it has emerged as a way to attain health which is defined by the World Health Organisation as complete physical, mental, and social well-being and not merely the absence of disease or infirmity.

Health benefits of yoga

Scientific evidence shows that therapeutic yoga has many health benefits. Numerous studies and anecdotal evidence also confirm health benefits of yoga.

Yoga can benefit health in thirty-eight ways. It can relieve back pain, improve heart health, improve strength, coordination, balance and flexibility, reduce inflammation, help in osteopenia, in oncology, and in recovery from surgery, help reduce anxiety and stress, ease arthritis symptoms, help sleep better, give more energy and brighter moods. Yoga may also boost immunity, improve bone health, improve brain functioning, and self-esteem.

Yoga and womens health

Yoga is especially beneficial in certain medical conditions typical to women. Six of these conditions are menopause, endometriosis, polycystic ovary syndrome (PCOS), uterine fibroids, premenstrual syndrome (PMS) and pregnancy.

Yoga and menopause

Women stop mensurating at a certain age. This condition is menopause. When a woman has not mensurated for twelve consecutive months, she has reached menopause. Natural menopause occurs in 40s or 50s. Premature menopause may occur before the age of 40 if ovaries are damaged, or are surgically removed, or because of medical treatment. After menopause, a woman cannot conceive.

At menopause, ovaries stop producing most of the female hormone estrogen. Low estrogen levels cause thirty-four symptoms. These can be severe (in 20% women), mild (60%) or no symptoms (20%). The symptoms may start during perimenopause, a period of 8-10 years before menopause. After menopause, during the post menopause period, symptoms ease for most women. But for some the symptoms may continue for ten years or longer.

The symptoms during perimenopause are: Heavier or lighter than usual periods Irregular or skipped periods Aggravated premenstrual syndrome (PMS) Breast tenderness.

Main symptoms of menopause are: Frequent urination Night sweats and/or cold flashes Hot flashes Insomnia and sleep disorders Dry vagina, discomfort during sex Dry mouth, eyes, and skin Mild depression, irritation, mood swings

A few women may also have: Joint and muscle aches and pains Hair loss or thinning Racing heart Headaches Weight gain Changes in libido (sex drive) Difficulty concentrating, memory lapses Higher risk of cardiovascular disease (CVD)

Therapy for menopause symptoms

Hormone Replacement Therapy (HRT), giving estrogen, is the most effective therapy for menopause symptoms. But HRT has many side effects. It also increases the risk of blood clots, certain types of cancer (breast cancer), cardiovascular disease, and strokes. HRT is therefore given only if essential, in smallest doses and for shortest time.

Yogic breathing techniques help women reduce hot flashes and night sweats. And yoga may alleviate insomnia and sleep disorders, depression, irritation, and mood swings, reduce joint and muscle aches and pains, reduce symptoms of severe PMS, reduce risk of CVD, and improve concentration and memory.

Today women in India live forty to fifty percent of their life in peri and post menopause phase. Yoga can help them cope with the symptoms of menopause.

Yoga and polycystic ovary syndrome (PCOS)

PCOS affects women of reproductive age. It increases the risk of infertility, endometrial and breast cancer, obesity, high blood pressure, heart problems, and diabetes. The exact cause of PCOS is unknown. But production of excess male hormone androgen and insulin, and certain genes are contributing factors. Treatment for PCOS is weight loss, healthy lifestyle, and some medications.

Yoga reduces testosterone levels, helps weight loss, stimulates reproductive organs, and improves emotional health, and thus limits the complications of PCOS.

Yoga and endometriosis

Endometriosis is caused by abnormal growth of the inner tissue of the uterus. It can cause infertility and chronic pelvic pain (CPP). It has no cure except removal of uterus. Surgical removal of tissues gives only temporary relief. Yoga reduces CPP but does not improve fertility.

Yoga and uterine fibroids and polyps

Uterine fibroids are benign tumors in the female reproductive tract. These are fed by hormones and blood, but the precise cause of their occurrence is unknown.

Yoga does not help shrink fibroids. But doing yoga during menstruation may increase blood flow into uterus and dilate uterine blood vessels. This may cause heavy bleeding and accelerate fibroid growth. Therefore, yoga should not be done during the first three days of period, or if the blood flow is heavy.

Yoga and premenstrual syndrome (PMS)

Premenstrual syndrome (PMS) occurs during late luteal phase of menstrual cycle and is relieved after the onset of menstruation. Main symptoms of PMS are mood swings, tender breasts, food cravings, fatigue, irritability, and depression. Three out of four women experience some symptom of PMS. Yoga, can reduce, or relieve, PMS distress

Yoga and pregnancy

Prenatal yoga reduces stress and anxiety, improves sleep, decrease lower back pain, nausea, headaches, and shortness of breath, and increases the strength, flexibility and endurance of muscles needed for childbirth. Yoga also causes increase in babys birth weight, decrease in preterm labor, and decrease in intrauterine growth restriction (IUGR). Hatha yoga and restorative yoga are also viable choice for pregnant women.

But talk to your doctor and to yoga instructor before starting yoga during pregnancy.

Conclusion

Yoga is cost-free and non-invasive. People of all ages benefit from yoga. It is good for womens health and well-being and is beneficial in certain medical conditions typical to women. For best results, integrate yoga with healthy lifestyle, healthy diet, exercise, therapeutic massage, and other stress-reducing measures.

Views expressed above are the author's own.

END OF ARTICLE

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Exelixis Announces Initiation of the STELLAR-303 Phase 3 Pivotal Trial Evaluating XL092 in Patients with Metastatic Colorectal Cancer – Yahoo Finance

Posted: June 22, 2022 at 2:12 am

STELLAR-303 is the first phase 3 pivotal trial evaluating XL092, a next-generation oral tyrosine kinase inhibitor

ALAMEDA, Calif., June 21, 2022--(BUSINESS WIRE)--Exelixis, Inc. (Nasdaq: EXEL) today announced the initiation of STELLAR-303, a phase 3 pivotal trial evaluating XL092 in combination with atezolizumab versus regorafenib in patients with metastatic colorectal cancer (CRC) that is not microsatellite instability-high or mismatch repair-deficient, who have progressed after or are intolerant to the standard of care therapy. XL092 is a next-generation tyrosine kinase inhibitor (TKI) in development for multiple advanced tumor types.

"There is a significant need for new treatment options for the majority of metastatic CRC patients, who do not have microsatellite instability-high or mismatch-repair deficient disease and whose tumors do not respond to immunotherapy alone," said Vicki L. Goodman, M.D., Executive Vice President, Product Development & Medical Affairs, and Chief Medical Officer, Exelixis. "Following recent promising data evaluating cabozantinib in combination with immunotherapies in colorectal cancer, we are thrilled to initiate our first phase 3 pivotal trial for XL092, our next-generation tyrosine kinase inhibitor. We look forward to learning more about how XL092 in combination with atezolizumab may benefit patients with metastatic colorectal cancer."

STELLAR-303 is a global, multicenter, randomized phase 3 open-label study that will enroll approximately 600 patients with documented RAS status. Patients will be randomized 1:1 to receive either XL092 in combination with atezolizumab or regorafenib. The primary objective of the study is to evaluate the efficacy of the combination in patients with RAS wild-type disease; exploratory endpoints include examining efficacy in those with RAS-mutated disease. The primary endpoint is overall survival. Secondary endpoints include progression-free survival, objective response rate and duration of response per Response Evaluation Criteria in Solid Tumors version 1.1 as assessed by the investigator.

Previously announced results from two studies of cabozantinib in combination with immunotherapies for the treatment of advanced CRC supported Exelixis decision to pursue clinical development of XL092 in this setting. The trial is sponsored by Exelixis, and Roche is supplying atezolizumab.

About XL092

XL092 is a next-generation oral TKI that inhibits the activity of receptor tyrosine kinases implicated in cancer growth and spread, including VEGF receptors, MET, AXL and MER. These receptor tyrosine kinases are involved in both normal cellular function and in pathologic processes such as oncogenesis, metastasis, tumor angiogenesis and resistance to multiple therapies, including immune checkpoint inhibitors. In designing XL092, Exelixis sought to build upon its extensive experience with and the target profile of cabozantinib, the companys flagship medicine, while improving key characteristics, including pharmacokinetic half-life. XL092 is currently being developed for the treatment of advanced solid tumors, including genitourinary cancers, as a monotherapy and in combination with immune checkpoint inhibitors. XL092 is the first internally discovered Exelixis compound to enter the clinic following the companys reinitiation of drug-discovery activities.

About Colorectal Cancer

Colorectal cancer is the third most common cancer and the third-leading cause of cancer-related deaths in the U.S. According to the American Cancer Society, about 150,000 new cases will be diagnosed and 53,000 people will die from the disease in 2022.1 Colorectal cancer is most frequently diagnosed among people aged 65-74 and is more common in men and those of African American descent. Nearly a quarter of colorectal cancer cases are diagnosed at the metastatic stage, at which point the five-year survival rate is just 15%.2 It has been estimated that approximately 40% of metastatic colorectal cancer cases exhibit a RAS mutation.3

About CABOMETYX (cabozantinib)

In the U.S., CABOMETYX tablets are approved for the treatment of patients with advanced renal cell carcinoma (RCC); for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib; for patients with advanced RCC as a first-line treatment in combination with nivolumab; and for adult and pediatric patients 12 years of age and older with locally advanced or metastatic differentiated thyroid cancer (DTC) that has progressed following prior VEGFR-targeted therapy and who are radioactive iodine-refractory or ineligible. CABOMETYX tablets have also received regulatory approvals in the European Union and additional countries and regions worldwide. In 2016, Exelixis granted Ipsen exclusive rights for the commercialization and further clinical development of cabozantinib outside of the U.S. and Japan. In 2017, Exelixis granted exclusive rights to Takeda for the commercialization and further clinical development of cabozantinib for all future indications in Japan. Exelixis holds the exclusive rights to develop and commercialize cabozantinib in the U.S.

CABOMETYX is not indicated as a treatment for metastatic colorectal cancer CRC that is not microsatellite instability-high or mismatch repair-deficient.

IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Hemorrhage: Severe and fatal hemorrhages occurred with CABOMETYX. The incidence of Grade 3 to 5 hemorrhagic events was 5% in CABOMETYX patients in RCC, HCC, and DTC studies. Discontinue CABOMETYX for Grade 3 or 4 hemorrhage and prior to surgery as recommended. Do not administer CABOMETYX to patients who have a recent history of hemorrhage, including hemoptysis, hematemesis, or melena.

Perforations and Fistulas: Fistulas, including fatal cases, occurred in 1% of CABOMETYX patients. Gastrointestinal (GI) perforations, including fatal cases, occurred in 1% of CABOMETYX patients. Monitor patients for signs and symptoms of fistulas and perforations, including abscess and sepsis. Discontinue CABOMETYX in patients who experience a Grade 4 fistula or a GI perforation.

Thrombotic Events: CABOMETYX increased the risk of thrombotic events. Venous thromboembolism occurred in 7% (including 4% pulmonary embolism) and arterial thromboembolism in 2% of CABOMETYX patients. Fatal thrombotic events occurred in CABOMETYX patients. Discontinue CABOMETYX in patients who develop an acute myocardial infarction or serious arterial or venous thromboembolic events that require medical intervention.

Hypertension and Hypertensive Crisis: CABOMETYX can cause hypertension, including hypertensive crisis. Hypertension was reported in 37% (16% Grade 3 and <1% Grade 4) of CABOMETYX patients. Do not initiate CABOMETYX in patients with uncontrolled hypertension. Monitor blood pressure regularly during CABOMETYX treatment. Withhold CABOMETYX for hypertension that is not adequately controlled with medical management; when controlled, resume at a reduced dose. Permanently discontinue CABOMETYX for severe hypertension that cannot be controlled with anti-hypertensive therapy or for hypertensive crisis.

Diarrhea: Diarrhea occurred in 62% of CABOMETYX patients. Grade 3 diarrhea occurred in 10% of CABOMETYX patients. Monitor and manage patients using antidiarrheals as indicated. Withhold CABOMETYX until improvement to Grade 1, resume at a reduced dose.

Palmar-Plantar Erythrodysesthesia (PPE): PPE occurred in 45% of CABOMETYX patients. Grade 3 PPE occurred in 13% of CABOMETYX patients. Withhold CABOMETYX until improvement to Grade 1 and resume at a reduced dose for intolerable Grade 2 PPE or Grade 3 PPE.

Hepatotoxicity: CABOMETYX in combination with nivolumab can cause hepatic toxicity with higher frequencies of Grades 3 and 4 ALT and AST elevations compared to CABOMETYX alone. Monitor liver enzymes before initiation of and periodically throughout treatment. Consider more frequent monitoring of liver enzymes than when the drugs are administered as single agents. For elevated liver enzymes, interrupt CABOMETYX and nivolumab and consider administering corticosteroids.

With the combination of CABOMETYX and nivolumab, Grades 3 and 4 increased ALT or AST were seen in 11% of patients. ALT or AST >3 times ULN (Grade 2) was reported in 83 patients, of whom 23 (28%) received systemic corticosteroids; ALT or AST resolved to Grades 0-1 in 74 (89%). Among the 44 patients with Grade 2 increased ALT or AST who were rechallenged with either CABOMETYX (n=9) or nivolumab (n=11) as a single agent or with both (n=24), recurrence of Grade 2 increased ALT or AST was observed in 2 patients receiving CABOMETYX, 2 patients receiving nivolumab, and 7 patients receiving both CABOMETYX and nivolumab. Withhold and resume at a reduced dose based on severity.

Adrenal Insufficiency: CABOMETYX in combination with nivolumab can cause primary or secondary adrenal insufficiency. For Grade 2 or higher adrenal insufficiency, initiate symptomatic treatment, including hormone replacement as clinically indicated. Withhold CABOMETYX and/or nivolumab and resume CABOMETYX at a reduced dose depending on severity.

Adrenal insufficiency occurred in 4.7% (15/320) of patients with RCC who received CABOMETYX with nivolumab, including Grade 3 (2.2%), and Grade 2 (1.9%) adverse reactions. Adrenal insufficiency led to permanent discontinuation of CABOMETYX and nivolumab in 0.9% and withholding of CABOMETYX and nivolumab in 2.8% of patients with RCC.

Approximately 80% (12/15) of patients with adrenal insufficiency received hormone replacement therapy, including systemic corticosteroids. Adrenal insufficiency resolved in 27% (n=4) of the 15 patients. Of the 9 patients in whom CABOMETYX with nivolumab was withheld for adrenal insufficiency, 6 reinstated treatment after symptom improvement; of these, all (n=6) received hormone replacement therapy and 2 had recurrence of adrenal insufficiency.

Proteinuria: Proteinuria was observed in 8% of CABOMETYX patients. Monitor urine protein regularly during CABOMETYX treatment. For Grade 2 or 3 proteinuria, withhold CABOMETYX until improvement to Grade 1 proteinuria, resume CABOMETYX at a reduced dose. Discontinue CABOMETYX in patients who develop nephrotic syndrome.

Osteonecrosis of the Jaw (ONJ): ONJ occurred in <1% of CABOMETYX patients. ONJ can manifest as jaw pain, osteomyelitis, osteitis, bone erosion, tooth or periodontal infection, toothache, gingival ulceration or erosion, persistent jaw pain, or slow healing of the mouth or jaw after dental surgery. Perform an oral examination prior to CABOMETYX initiation and periodically during treatment. Advise patients regarding good oral hygiene practices. Withhold CABOMETYX for at least 3 weeks prior to scheduled dental surgery or invasive dental procedures, if possible. Withhold CABOMETYX for development of ONJ until complete resolution, resume at a reduced dose.

Impaired Wound Healing: Wound complications occurred with CABOMETYX. Withhold CABOMETYX for at least 3 weeks prior to elective surgery. Do not administer CABOMETYX for at least 2 weeks after major surgery and until adequate wound healing. The safety of resumption of CABOMETYX after resolution of wound healing complications has not been established.

Reversible Posterior Leukoencephalopathy Syndrome (RPLS): RPLS, a syndrome of subcortical vasogenic edema diagnosed by characteristic findings on MRI, can occur with CABOMETYX. Evaluate for RPLS in patients presenting with seizures, headache, visual disturbances, confusion, or altered mental function. Discontinue CABOMETYX in patients who develop RPLS.

Thyroid Dysfunction: Thyroid dysfunction, primarily hypothyroidism, has been observed with CABOMETYX. Based on the safety population, thyroid dysfunction occurred in 19% of patients treated with CABOMETYX, including Grade 3 in 0.4% of patients.

Patients should be assessed for signs of thyroid dysfunction prior to the initiation of CABOMETYX and monitored for signs and symptoms of thyroid dysfunction during CABOMETYX treatment. Thyroid function testing and management of dysfunction should be performed as clinically indicated.

Hypocalcemia: CABOMETYX can cause hypocalcemia. Based on the safety population, hypocalcemia occurred in 13% of patients treated with CABOMETYX, including Grade 3 in 2% and Grade 4 in 1% of patients. Laboratory abnormality data were not collected in CABOSUN.

In COSMIC-311, hypocalcemia occurred in 36% of patients treated with CABOMETYX, including Grade 3 in 6% and Grade 4 in 3% of patients.

Monitor blood calcium levels and replace calcium as necessary during treatment. Withhold and resume at reduced dose upon recovery or permanently discontinue CABOMETYX depending on severity.

Embryo-Fetal Toxicity: CABOMETYX can cause fetal harm. Advise pregnant women and females of reproductive potential of the potential risk to a fetus. Verify the pregnancy status of females of reproductive potential prior to initiating CABOMETYX and advise them to use effective contraception during treatment and for 4 months after the last dose.

ADVERSE REACTIONS

The most common (20%) adverse reactions are:

CABOMETYX as a single agent: diarrhea, fatigue, PPE, decreased appetite, hypertension, nausea, vomiting, weight decreased, constipation.

CABOMETYX in combination with nivolumab: diarrhea, fatigue, hepatotoxicity, PPE, stomatitis, rash, hypertension, hypothyroidism, musculoskeletal pain, decreased appetite, nausea, dysgeusia, abdominal pain, cough, and upper respiratory tract infection.

DRUG INTERACTIONS

Strong CYP3A4 Inhibitors: If coadministration with strong CYP3A4 inhibitors cannot be avoided, reduce the CABOMETYX dosage. Avoid grapefruit or grapefruit juice.

Strong CYP3A4 Inducers: If coadministration with strong CYP3A4 inducers cannot be avoided, increase the CABOMETYX dosage. Avoid St. Johns wort.

USE IN SPECIFIC POPULATIONS

Lactation: Advise women not to breastfeed during CABOMETYX treatment and for 4 months after the final dose.

Hepatic Impairment: In patients with moderate hepatic impairment, reduce the CABOMETYX dosage. Avoid CABOMETYX in patients with severe hepatic impairment.

Please see accompanying full Prescribing Information https://www.cabometyx.com/downloads/CABOMETYXUSPI.pdf.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit http://www.FDA.gov/medwatch or call 1-800-FDA-1088.

About Exelixis

Founded in 1994, Exelixis, Inc. (Nasdaq: EXEL) is a commercially successful, oncology-focused biotechnology company that strives to accelerate the discovery, development and commercialization of new medicines for difficult-to-treat cancers. Following early work in model system genetics, we established a broad drug discovery and development platform that has served as the foundation for our continued efforts to bring new cancer therapies to patients in need. Our discovery efforts have resulted in four commercially available products, CABOMETYX (cabozantinib), COMETRIQ (cabozantinib), COTELLIC (cobimetinib) and MINNEBRO (esaxerenone), and we have entered into partnerships with leading pharmaceutical companies to bring these important medicines to patients worldwide. Supported by revenues from our marketed products and collaborations, we are committed to prudently reinvesting in our business to maximize the potential of our pipeline. We are supplementing our existing therapeutic assets with targeted business development activities and internal drug discovery all to deliver the next generation of Exelixis medicines and help patients recover stronger and live longer. Exelixis is a member of the Standard & Poors (S&P) MidCap 400 index, which measures the performance of profitable mid-sized companies. For more information about Exelixis, please visit http://www.exelixis.com, follow @ExelixisInc on Twitter or like Exelixis, Inc. on Facebook.

Forward-looking Statements

This press release contains forward-looking statements, including, without limitation, statements related to: the clinical and therapeutic potential of XL092 in combination with atezolizumab as a treatment for patients with metastatic colorectal cancer; and Exelixis plans to reinvest in its business to maximize the potential of the companys pipeline, including through targeted business development activities and internal drug discovery. Any statements that refer to expectations, projections or other characterizations of future events or circumstances are forward-looking statements and are based upon Exelixis current plans, assumptions, beliefs, expectations, estimates and projections. Forward-looking statements involve risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in the forward-looking statements as a result of these risks and uncertainties, which include, without limitation: the potential failure of the combination of XL092 and atezolizumab to demonstrate safety and/or efficacy in STELLAR-303; uncertainties inherent in the product development process; complexities and the unpredictability of the regulatory review and approval processes in the U.S. and elsewhere; Exelixis and Roches continuing compliance with applicable legal and regulatory requirements; the continuing COVID-19 pandemic and other global events and their impact on Exelixis research and development operations, including Exelixis ability to initiate new clinical trials and clinical trial sites, enroll clinical trial patients, conduct trials per protocol, and conduct drug research and discovery operations and related activities; the costs of conducting clinical trials; Exelixis dependence on third-party vendors for the development, manufacture and supply of XL092; Exelixis ability to protect its intellectual property rights; market competition; changes in economic and business conditions; and other factors affecting Exelixis and its development programs discussed under the caption "Risk Factors" in Exelixis Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 10, 2022, and in Exelixis future filings with the SEC. All forward-looking statements in this press release are based on information available to Exelixis as of the date of this press release, and Exelixis undertakes no obligation to update or revise any forward-looking statements contained herein, except as required by law.

Exelixis, the Exelixis logo, CABOMETYX and COMETRIQ are registered U.S. trademarks of Exelixis.COTELLIC is a registered trademark of Genentech, Inc.MINNEBRO is a registered trademark of Daiichi Sankyo Company, Limited.

_______________________1 Cancer Facts and Figures 2022. American Cancer Society website. Available at: https://www.cancer.org/content/dam/cancer-org/research/cancer-facts-and-statistics/annual-cancer-facts-and-figures/2022/2022-cancer-facts-and-figures.pdf. Accessed June 2022.2 Cancer Stat Facts: Colorectal Cancer. SEER website. Available at: https://seer.cancer.gov/statfacts/html/colorect.html. Accessed June 2022.3 RAS in Colorectal Cancer: ESMO Biomarker Factsheet. OncologyPRO website. Available at https://oncologypro.esmo.org/education-library/factsheets-on-biomarkers/ras-in-colorectal-cancer. Accessed June 2022.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220620005441/en/

Contacts

Investors: Susan Hubbard EVP, Public Affairs andInvestor Relations Exelixis, Inc. (650) 837-8194 shubbard@exelixis.com

Media: Lindsay Treadway Executive Director, Public Affairsand Advocacy Relations Exelixis, Inc. (650) 837-7522 ltreadway@exelixis.com

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Exelixis Announces Initiation of the STELLAR-303 Phase 3 Pivotal Trial Evaluating XL092 in Patients with Metastatic Colorectal Cancer - Yahoo Finance

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7 Strong Buy Dividend Aristocrats Are Safe-Haven Stocks to Own During a Recession – 24/7 Wall St.

Posted: June 22, 2022 at 2:12 am

The market received the proverbial haymaker from the Federal Reserve last week, when the central bank raised interest rates by 75 basis points, the largest increase since November of 1994. To add insult to injury, unless the Fed governors and Chair Jay Powell see at least some decline in the staggering inflation, you can count on another 75-basis-point increase in July.

With the market getting absolutely torched last week, the venerable Dow Jones industrials dipped below the 30,000 level, and both the S&P 500 and the Nasdaq are in bear market territory. Many investors are worried, and with good reason. After years of loose money policy, the party is over, and it is time to move assets to safe, dividend-paying companies to ride out the storm. With the potential for a recession increasing, the time to reallocate is now.Often when income investors look for companies paying big dividends, they are drawn to the Dividend Aristocrats, and with good reason. The 66 companies that made the cut for the 2022 S&P 500 Dividend Aristocrats list have increased dividends (not just remained the same) for 25 years straight. But the requirements go even further. The following attributes are also mandatory for membership on the vaunted list:

With the potential for massive downside still looming, and interest rates definitely still going higher, we thought it would be a good idea to look for companies on the Dividend Aristocrats list that are in defensive sectors and look poised to do well the rest of 2022.

Seven stocks hit our screens, all of which are Buy rated at top Wall Street firms. It is important to remember that no single analyst report should be used as a sole basis for any buying or selling decision.

This is a top pharmaceutical and med-tech stock with very solid growth potential. Abbott Laboratories (NYSE: ABT) manufactures and sells health care products worldwide.

Its Established Pharmaceutical Products segment offers branded generic pharmaceuticals to treat pancreatic exocrine insufficiency; irritable bowel syndrome or biliary spasm; intrahepatic cholestasis or depressive symptoms; gynecological disorders; hormone replacement therapy; dyslipidemia; hypertension; hypothyroidism; Mnires disease and vestibular vertigo; pain, fever and inflammation; migraines; anti-infective clarithromycin; cardiovascular and metabolic products; and influenza vaccines, as well as to regulate physiological rhythm of the colon.

The LabsDiagnostic Products segment provides immunoassay and clinical chemistry systems; assays used to screen and/or diagnose cancer, cardiac, drugs of abuse, fertility, infectious diseases and therapeutic drug monitoring; hematology systems and reagents; diagnostic systems and cartridges; instruments to automate the extraction, purification and preparation of DNA and RNA from patient samples, and detects and measures infectious agents; genomic-based tests; informatics and automation solutions; and a suite of informatics tools and professional services.

Abbott Laboratories stock investors receive a 1.83% dividend. Morgan Stanleys price target is $145, and the consensus target is $139.29. The shares closed most recently at $102.53.

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BrainStorm Strengthens Executive Team with Key Appointments in R&D and Legal – GuruFocus.com

Posted: June 22, 2022 at 2:11 am

Netta Blondheim-Shraga, PhD Appointed as VP R&DAntal Pearl-Lendner, Adv. Appointed as Chief Legal Counsel

NEW YORK, May 12, 2022 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, announced two senior management appointments. Netta Blondheim Shraga, PhD has been appointed as VP, Research & Development, and Antal Pearl-Lendner, Adv. has been appointed to the newly created position of Chief Legal Counsel. Both will report directly to Chaim Lebovits, CEO.

"We are thrilled to welcome Netta and Antal, each of whom brings valuable experience in their respective areas of R&D and Legal Affairs," said Chaim Lebovits, Chief Executive Officer of BrainStorm. "As we prepare the company for growth, it is important that we continue to build out our senior executive team and attract professionals with the appropriate skillsets. We look forward to leveraging their backgrounds as we execute on our mission to bring autologous cell therapies to patients with debilitating neurodegenerative diseases."

Dr. Blondheim-Shraga will be responsible for advancing the company's pipeline and steering the R&D team towards significant breakthroughs in the field of cell therapy and development of novel solutions to positively impact patients' health. Dr. Blondheim-Shraga joins BrainStorm with over 14 years of translational research experience in academic, biotech and pharma settings, having led teams in Israel, USA and China, combining scientific, entrepreneurial and management skills. Prior to joining BrainStorm, she was Project Leader on the Academic Affairs team at Teva Pharmaceuticals, Israel. In this role, she managed a portfolio of diverse and highly impactful strategic scientific collaborations with Teva's academic partners and managed Teva's involvement in several international consortia. Prior to Teva, she was Study Director and Senior Scientist at CrownBio, San Diego, CA. Earlier in her career, she was a Senior Scientist at Lifemap Sciences LTD in Tel-Aviv and served as Scientific Advisor to ImmunoHiTech LTD, Ramat Hasharon, Israel for several years. Dr. Blondheim-Shraga received a PhD from the Faculty of Medicine, Bar-Ilan University, Safed, Israel, an MSc Med from The Faculty of Medicine, Tel Aviv University, Israel and a BSc Med from The Faculty of Medicine, Hebrew University Jerusalem, Israel.

Antal Pearl-Lendner, Adv. is an experienced bilingual attorney with a proven track record in legal and business development capacities. Prior to joining Brainstorm, Ms. Pearl-Lendner spent 8 years at Mizrahi-Tefahot Bank in Israel where her responsibilities included spearheading bank-wide complex projects, negotiating large scale international contracts and providing ongoing advice regarding the international activities of the bank. Before her tenure at the bank, she worked at GE Capital in Chicago and Connecticut, USA, where she served in GE's premier commercial leadership program, working in business development, strategy & analytics. Earlier in her career, Ms. Pearl-Lendner was an Associate Attorney in the international department of Caspi & Co. Advocates & Notaries in Tel Aviv, Israel. In this role she represented clients in M&A transactions and led due diligence processes for investments ranging from $5M to $350M. Ms. Pearl-Lendner received an MBA from the MIT Sloan School of Management in Cambridge, Massachusetts and an LLB from Tel Aviv University.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug designation status from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has completed a Phase 3 pivotal trial in ALS (NCT03280056); this trial investigated the safety and efficacy of repeat-administration of autologous MSC-NTF cells and was supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). BrainStorm completed under an investigational new drug application a Phase 2 open-label multicenter trial (NCT03799718) of autologous MSC-NTF cells in progressive multiple sclerosis (MS) and was supported by a grant from the National MS Society (NMSS).

Safe-Harbor Statement

Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may," "should," "would," "could," "will," "expect," "likely," "believe," "plan," "estimate," "predict," "potential," and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, prospects for future regulatory approval of BrainStorm's NurOwn treatment candidate, the success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our products and services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation; the impacts of the COVID-19 pandemic on our clinical trials, supply chain, and operations; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations, and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance, or achievements.

CONTACTS

Investor Relations:John MullalyLifeSci Advisors, LLCPhone: +1 617-429-3548 [emailprotected]

Media:Uri Yablonka[emailprotected]

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SOURCE BrainStorm Cell Therapeutics Inc.

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NorthX Biologics expands to Cell Therapy: Partnership with Alder Therapeutics and new manufacturing site on Karolinska campus – GlobeNewswire

Posted: June 22, 2022 at 2:09 am

MATFORS, Sweden, June 21, 2022 (GLOBE NEWSWIRE) -- Sweden-based NorthX Biologics (NorthX) is expanding into cell therapy manufacturing at its existing GMP-facility, as well as in premises at the Karolinska University Hospital campus in Stockholm. This initiative is part of NorthXs Innovation Hub, an Innovation Track designed to provide development and GMP-manufacturing services to the next generation of drug development companies and innovative research groups in need of NorthXs Good Manufacturing Practice (GMP) expertise.

NorthX has one of Northern Europes largest clinical-grade manufacturing capacities for plasmid DNA, recombinant proteins, cell banking and associated gene therapy services and this expansion into cell therapy is a major step to complete our offering for innovative clients. We are especially excited to work with Dr. Kristian Tryggvason, a leader in cell therapy technologies, and his team, said Dr. Ted Fjllman, CEO of NorthX.

After having built up BioLamina and its cell culture reagents that are used worldwide both in academia and industry, Dr. Tryggvason recently launched his latest venture: Alder Therapeutics. In addition to its own product development, the company now entered into an agreement to help NorthX expand its cell culture services to many new different cell types, including pluripotent stem cells. The Alder team will also help to design and validate NorthXs new process development and GMP-manufacturing labs in Matfors, alongside those being established at the Karolinska University Hospital campus in Stockholm.

Our goal is to be able to offer synergies to both cell and gene therapy clients and to collaborate with them through our Innovation Track, in which we work hand in hand with our clients regarding process development, manufacturing, and analytics to progress clinical programs and bring life-saving treatments to patients, added Aaron Small, NorthX VP of Global Sales and Corporate Development

Universities and cell therapy companies worldwide need GMP-grade development and manufacturing capacity, as it is complex and outside the scope of most biotech companies to build themselves. NorthX Biologics is already helping to translate cutting-edge gene therapy research into clinical development and now we will together build upon the existing broad cell therapy know-how in Sweden to do the same for cell therapies, said Dr. Kristian Tryggvason, CEO Alder Therapeutics.

About NorthX Biologics:

NorthX Biologics provides process development and manufacturing services with expertise in plasmids, proteins and other advanced biologics. NorthX Biologics sits in the heart of Sweden, and the team has been manufacturing biologics to GMP since 1988. In 2021 NorthX was recognized as a national innovation hub for advanced therapeutics and vaccines. NorthX has the ambition to become a leading cell and gene therapy manufacturer and partner of choice for innovative drug development companies. For more information see http://www.nxbio.com

About Alder Therapeutics:

Headquartered inStockholm, Alder Therapeutics AB is a novel development stage, cell therapy platform company aiming to develop and manufacture the best functional cell therapy products based on the most simple and robust processes. Alder Therapeutics unique cell therapy platform will allow manufacturing of better cells at a lower cost, which will make pluripotent cell therapy treatment available for ever more patients. Alder Therapeutics will play an important role in opening the next era in medicinal treatment. For more information and important updates, please visit. http://www.aldertx.com

For further information please email NorthX at:contact@nxbio.com

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Japan’s five hottest biotech companies in healthcare – Labiotech.eu

Posted: June 22, 2022 at 2:09 am

While historically lacking in foreign investments, Japans biotech scene is thriving with global investors showing increasing interest. Here are five of the hottest Japanese private companies innovating in the healthcare space.

Japan boasts one of the highest life expectancies in the world, and, faced with a rapidly aging population, is witnessing a growing burden of chronic conditions including cardiovascular disease and type 2 diabetes. For this reason, the Japanese healthcare authorities are encouraging research into the treatment and prevention of these diseases, in addition to promoting the potential of regenerative medicine.

In addition to having a roster of healthcare giants including Takeda, Astellas Pharma and Eisai, Japan is also an Asian hotspot for biotech companies. Upcoming startups have historically been limited in foreign funding and reliant on local venture capital players such as Nippon Venture Capital, Shinsei Capital Partners, and the University of Tokyo Edge Capital Partners.

In 2021, however, the amount of foreign investment flowing into the Japanese biotech space rose to $98 million, almost triple the haul of previous years. The most prominent global backers included Newton Biocapital, F-Prime Capital, and SoftBank Group. This trend arose as the COVID-19 pandemic triggered a wave of investor enthusiasm in biotechnology around the world.

With the help of local experts, weve listed five of the hottest private biotech companies in Japan. These firms, shown in alphabetical order, have raised large funding rounds in the last two years and are developing innovative treatments for a range of conditions including cancer, cardiovascular disease and inflammatory disorders.

Source: Shutterstock

Founded: 2017

Headquarters: Fujisawa

Chordia Therapeutics derives its name from the English term chord referring to a collection of musical notes normally played in harmony. In a similar way, the company aims to work in harmony with stakeholders and collaborators to develop first-in-class small molecule treatments for cancer.

Chordias lead program is a drug that disrupts the processing of RNA in tumor cells. In a healthy cell, RNA molecules are typically transcribed from a DNA template and spliced together to guide the production of new proteins. Some cancer cells accumulate mutations in the RNA splicing machinery and become vulnerable to Chordias drugs that interfere with this process.

Chordia raised $31 million (4 billion yen) in a Series C round in May 2022. The aim of the round was to push the companys lead drug through phase I testing and fund the preclinical development of the rest of its pipeline.

This month, the company announced interim results from the phase I trial of its lead candidate, with four of the recruited patients so far showing signs of responding to the treatment.

Founded: 2015

Headquarters: Tokyo

Heart failure occurs when the heart muscle is irreparably damaged and is unable to pump blood. While this deadly condition can be treated with a heart transplant, there is a general shortage of donors available, making a pressing need for alternatives.

In June 2021, the stem cell therapy developer Heartseed raised $36.5 million (4 billion yen) in a Series C round. The mission is to provide a regenerative route to saving the heart via stem cell therapy.

In the lab, Heartseed reprograms skin cells from the patient into a type of stem cell called induced pluripotent stem cells and grows these stem cells into heart muscle cells. The company then injects the muscle cells as a small cluster, or seed, into heart tissue to repair the muscle.

The proceedings from its Series C round will allow Heartseed to take its lead candidate into clinical development, including a phase I/II trial scheduled for later this year. Last year, Heartseed also licensed its treatment to Novo Nordisk in Denmark to co-develop the treatment outside of Japan.

Founded: 2018

Headquarters: Tokyo

LUCA Science hit the headlines in the last week for raising an impressive $30.3 million (3.86 billion yen) in a Series B round. The company is developing an unusual approach for treating a wide range of diseases: delivering a therapy based on mitochondria, the energy production plants in human cells.

One example where the technology could work well is in strokes and heart attacks, where blood flow is blocked to critical tissue in the brain and heart respectively. The reperfusion of blood to these tissues after the blockage can kill the tissue by damaging its mitochondria. Delivering healthy mitochondria could keep the tissue working properly and protect it from harm.

LUCA Science plans to use its recent Series B winnings to accelerate the preclinical development of its mitochondrial therapies and establish its manufacturing process. In May 2022, the firm also inked a collaboration deal with compatriot pharmaceutical company Kyowa Kirin Co., Ltd. to co-develop a mitochondrial therapy for rare genetic diseases.

Founded: 2016

Headquarters: Boston, U.S., and Tokyo

Modulus Discovery is a preclinical-stage drug discovery specialist. The company focuses on developing small molecule treatments for conditions such as cancer, inflammatory disorders and rare genetic conditions.

The firm uses a mixture of strategies to speed up the drug discovery process. These include simulating target proteins using a supercomputer; structural protein biology; forming collaborations such as with the peptide drug expert PeptiDream; and tapping into global networks for biological expertise. Modulus most advanced drug program is in late-stage preclinical testing for the treatment of chronic inflammatory diseases.

In March 2022, Modulus bagged $20.4 million (2.34 billion yen) in a Series C round. The cash is earmarked to advance the companys R&D programs by expanding its infrastructure, collaborations and headcount.

Founded: 2015

Headquarters: Tokyo

The name Noile-Immune is derived from blending together the phrases no illness and no immunity, no life. This company is developing CAR-T cell therapies for the treatment of cancer, which traditionally consist of extracting the patients immune T cells, engineering them in the lab to hunt down cancer cells, and reinfusing them into the patient.

Unlike approved CAR-T cell therapies, which are limited to treating forms of blood cancer, Noile-Immune aims its therapies at treating solid tumors. The company does this by engineering immune T cells to produce proteins that cause immune cells to migrate into the tumor site.

Noile-Immune is testing its lead candidate in a phase I in patients with solid tumors. The firm is also co-developing therapies with partners including Takeda and the European cell therapy specialists Adaptimmune and Autolus. Additionally, Noile-Immune has an allogeneic version of its cell therapy in the pipeline where immune T cells are sourced from healthy donors rather than the patient.

To finance the clinical development of its lead candidate, Noile-Immune raised $21.8 million (2.38 billion yen) in a Series C round in early 2021. The company hit a setback in January 2022 when a collaboration deal fell through with the U.S. player Legend Biotech. Nonetheless, other external companies remain interested in Noile-Immunes offering, including Japan-based Daiichi Sankyo Company Ltd., which opted to assess Noile-Immunes technology in late 2021.

Cover image via Elena Resko. Inline images via Shutterstock.

Thanks to feedback from Shiohara Azusa, VC investor at the University of Tokyo Edge Capital Partners, and Hironoshin Nomura, Chief Financial Officer at Sosei Group Corporation.

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Sickle cell beta thalassemia: Causes, symptoms, and treatments – Medical News Today

Posted: June 22, 2022 at 2:09 am

Sickle cell beta-thalassemia refers to an inherited condition that impacts hemoglobin. People with the condition have different changes in each copy of their hemoglobin gene. One causes red blood cells (RBCs) to form a sickle shape and another reduces the amount of hemoglobin.

Sickle cell beta-thalassemia is a type of RBC disorder known as a hemoglobinopathy. These are conditions that cause abnormal hemoglobin production or a change in its structure. Hemoglobin is the protein in RBCs responsible for carrying oxygen around the body.

Both sickle cell disease and beta-thalassemia are genetic conditions that affect hemoglobin. A person with sickle cell beta-thalassemia inherits a trait for both conditions, impacting the shape and number of hemoglobin.

In this article, we will discuss the causes and risk factors for sickle cell beta-thalassemia, as well as symptoms and treatment options for the condition.

Sickle cell beta-thalassemia is a genetic condition and a type of sickle cell disease that features symptoms of both sickle cell disease and beta-thalassemia. It causes RBCs to take on a sickle shape, making them unable to flow through the blood vessels as smoothly. It also affects the amount of normal hemoglobin a person has in their blood.

It is an inherited condition, meaning that parents pass it on to their children. Researchers estimate that 57% of the global population are carriers of a significant hemoglobin mutation. People with sickle cell beta-thalassemia inherit a sickle trait from one parent and a beta-thalassemia trait from the other.

There are two types of sickle cell beta-thalassemia: plus (HbS beta+) and zero (HbS beta0). The former is the milder variant. The plus indicates that the blood contains a lower-than-average amount of normal hemoglobin. This differs from the latter, in which a person has no normal hemoglobin.

Sickle cell beta-thalassemia results from a change in the beta-hemoglobin (HBB) gene. The beta-hemoglobin gene is responsible for forming the hemoglobin subunit beta component of the hemoglobin protein.

A person develops sickle cell beta-thalassemia when they inherit one sickle cell trait from one parent and one beta-thalassemia trait from the other. The beta-thalassemia gene can be either beta+, which results in a lower production of normal hemoglobin, or beta0, which leads to a complete absence of normal hemoglobin.

Risk factors for a person developing sickle cell beta-thalassemia include having parents that may be carriers of the sickle cell, HbS beta+, or HbS beta0 gene. The condition follows an autosomal recessive inheritance pattern. This means that if a person has a beta-thalassemia trait and their partner has a sickle cell trait, there is a 25% chance with each pregnancy that their child will have sickle cell beta-thalassemia.

The symptoms a person may experience will depend on whether they have HbS beta+ or HbS beta0. The amount of normal hemoglobin a person can produce largely determines the severity of the condition.

A person with HbS beta+ will likely have milder symptoms, as while they produce less functioning hemoglobin than is typical, they can still produce normal hemoglobin. Individuals with either type will produce sickle-shaped RBCs that can block circulation and cause cell damage and pain.

A person with HbS beta+ may experience:

The symptoms of HbS beta0 are typically similar but with a more severe case of anemia.

Some newborn screenings include testing for sickle cell disorders such as sickle cell beta-thalassemia. Screening involves taking a blood sample and looking at the types of hemoglobin present in the newborns blood. However, newborn screenings only suggest the possibility of sickle cell beta-thalassemia and require further confirmatory tests. This may include the parents and any siblings having tests.

These tests may include:

Treatment aims to prevent complications from occurring and treat the symptoms a person may experience. This generally involves continuous care to prevent and manage potential problems.

In addition to the treatments below, a person may receive pain relief medications and antibiotics to help reduce pain and infections. The spleen usually helps prevent infections, but many people with sickle cell beta-thalassemia may lose spleen function. As such, children may receive penicillin prophylaxis to prevent pneumococcal sepsis.

Treatment options for sickle cell beta-thalassemia may include:

People may require hydroxyurea if they experience frequent periods of pain. Hydroxyurea is a drug that makes RBCs bigger and changes their shape to the typical round and flexible composition. This can help slow or prevent complications.

Hydroxyurea increases the level of fetal hemoglobin (HbF) in the body. HbF is present in higher quantities in newborns and can help protect against sickle cell complications. With higher levels of HbF, RBCs are less likely to become sickle-shaped.

According to the American Society of Hematology, people with sickle cell beta-thalassemia who take hydroxyurea also have fewer:

Some people with sickle cell beta-thalassemia may require blood transfusions. This is when a healthcare professional infuses healthy donor blood into the body of a person with sickle cell beta-thalassemia via a tube. The donor blood will need to have matching antigens to the blood of the person receiving the transfusion. These antigens, known as human leukocyte antigens, are proteins present on the surface of RBCs.

If the antigens do not match, the immune system of the person receiving the blood donation is more likely to reject the transfusion, and it may lead to a reaction that can cause health problems.

The bone marrow in the body produces blood cells. A person with dysfunctional bone marrow, such as in sickle cell beta-thalassemia, may receive hematopoietic stem cells from a healthy donor. This may help improve bone marrow function and reduce the symptoms of sickle cell beta-thalassemia. A hematopoietic stem cell transplant is currently the only cure for this condition.

Sickle cell beta-thalassemia is a type of sickle cell disease. Some evidence suggests the life expectancy of a person living with sickle cell disease is reduced by 2030 years compared with a healthy individual. Similarly, a 2019 study suggests a person with sickle cell disease may live roughly 22 fewer years than a person without the condition.

Advances in therapy and treatment options are helping to improve the outlook of people with sickle cell disease. However, statistics like these highlight the necessity to further develop approaches to improve the underlying morbidity and mortality of individuals with the condition.

Most often, a person will receive a diagnosis of sickle cell beta-thalassemia shortly after birth. Later in life, if they believe their current treatment regime is not suitable or notice worsening symptoms, they should contact their doctor.

If a person is aware of a family history of sickle cell disease, they may wish to consider undergoing genetic screening before attempting to have children.

Sickle cell beta-thalassemia is a type of sickle cell disease. It occurs when a person inherits a sickle cell trait and a beta-thalassemia trait from their parents. It results in a person having sickle-shaped RBCs and either producing a low amount of hemoglobin or none at all.

Symptoms of the condition can include mild to severe anemia, tiredness, weakness, pain, and possible organ damage. Treatment may involve pain relief medications, antibiotics, hydroxyurea, and blood transfusions. Some people may also receive a bone marrow transplant to help them produce healthy hemoglobin.

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Umoja Biopharma Presents Data on its Engineered Induced Pluripotent Stem Cell Platform at the 2022 International Society for Stem Cell Research Annual…

Posted: June 22, 2022 at 2:09 am

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SEATTLE, June 16, 2022 (GLOBE NEWSWIRE) -- Umoja Biopharma, Inc., an immuno-oncology company pioneering off-the-shelf, integrated therapeutics that reprogram immune cells in vivo to treat patients with solid and hematologic malignancies, announced today that it will have a poster presentation at the 2022 International Society for Stem Cell Research (ISSCR) Annual Meeting, to be held June 15-18, 2022 in San Francisco, California.

On Wednesday, June 15th, Principal Scientist & iPSC Team Lead, Teisha Rowland, Ph.D., will give a poster presentation titled, A Synthetic Cytokine Receptor Platform for Producing Cytotoxic Innate Lymphocytes as Off-the-Shelf Cancer Therapeutics. The presentation will discuss Umojas engineered induced pluripotent stem cell (iPSC) platform, that incorporates the synthetic cytokine receptor system rapamycin-activated cytokine receptor (RACR) platform. Umojas engineered iPSCs that are modified to express RACR, called RACR-induced cytotoxic innate lymphoid (iCIL) cells, drive differentiation and expansion of the cells while eliminating the need for expensive cytokines and other raw materials. The RACR platform has the potential to enable cytokine-free manufacturing and engraftment of the engineered cells in the patient without the need for toxic lymphodepletion.

Despite the advances chimeric antigen receptor T cell therapies have provided to the oncology space, we continue to battle significant challenges that these therapies cannot address, like limited expansion capacity and scalability, manufacturing complexity, variability among patients, and the need for toxic chemotherapy administration to combat patients anti-allograft response, said Andy Scharenberg, M.D., co-founder and Chief Executive Officer of Umoja. We are developing an engineered iPSC platform, including the RACR platform, to address these challenges by enabling a scalable, virtually unlimited, and simplified manufacturing of engineered, cancer-fighting cytotoxic innate lymphocytes.

About Umoja Biopharma

Umoja Biopharma, Inc. is an early clinical-stage company advancing an entirely new approach to immunotherapy. Umoja Biopharma, Inc. is a transformative multi-platform immuno-oncology company founded with the goal of creating curative treatments for solid and hematological malignancies by reprogramming immune cells in vivo to target and fight cancer. Founded based on pioneering work performed at Seattle Childrens Research Institute and Purdue University, Umojas novel approach is powered by integrated cellular immunotherapy technologies including the VivoVec in vivo delivery platform, the RACR/CAR in vivo cell expansion/control platform, and the TumorTag targeting platform. Designed from the ground up to work together, these platforms are being developed to create and harness a powerful immune response in the body to directly, safely, and controllably attack cancer. Umoja believes that its approach can provide broader access to the most advanced immunotherapies and enable more patients to live better, fuller lives. To learn more, visit http://umoja-biopharma.com/.

About RACR

CAR T cells generated by the body with VivoVec can be expanded and sustained with the rapamycin activated cytokine receptor (RACR) system, an engineered signaling system designed to improve chimeric antigen receptor (CAR) T cell persistence and produce durable anti-tumor responses. The RACR/CAR payload is integrated into the genomic DNA of a patients T cells. Rapamycin activates the RACR system resulting in preferential expansion and survival of cancer-fighting T cells. The RACR technology enables a patients cells to expand in a manner that resembles a natural immune response that does not require lymphodepletion, promoting durable T cell engraftment. RACR/CAR technology can also be used to enhanceex vivomanufacturing in support of more traditional autologous or allogeneic cell therapy manufacturing processes. To learn more about Umojas RACR platform please visit https://www.umoja-biopharma.com/platforms/

Media Contact:Darren Opland, Ph.D.LifeSci Communications[emailprotected]

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