Various career paths are available in the field of Genetic Medicine and Stem Cell Research

Various career paths are available in the field of Genetic Medicine and Stem Cell Research

Among the various streams of science and medicine that have evolved with time, stem cell research and genetic medicine have risen as top contenders for various path-breaking discoveries. The treatment of more than 1,800 known monogenic hereditary disorders today, depends on the development of genetic medicines coupled with advanced stem cell research.

The field of genetic medicine comprises many areas, including the clinical practice of physicians, genetic counsellors and nutritionists, clinical diagnostic laboratory activities, and research into the causes and inheritance of genetic disorders. Simply put, it incorporates areas such as gene therapy, personalised medicine, and the rapidly emerging new medical speciality, including predictive medicine.

Stem cells are basically adaptable and versatile cells in ones body that are capable of complex actions, unlike conventional medication. These cells exist both in embryos and adult cells and can differentiate into any cell of an organism with the ability of self-renewal. Stem cell therapy uses these cells to treat or prevent a disease or condition. Also known as regenerative medicine, it promotes the repair response of diseased, dysfunctional or injured tissue using stem cells or their derivatives.

Amid a prevailing global healthcare crisis, stem cell research and genetic medicine have given us hope. The former was and continues to be an integral part of research conducted to treat COVID-19 symptoms, and genome sequencing has been extensively employed globally to analyse mutations and variations of the virus. In the coming years, the industry is only expected to grow.

Also, there is a general misconception that only people with medical education can take up jobs in these fields. This is not completely true because students who have studied Biology, B.Sc. graduates with at least one subject of the Biological Sciences, MBBS, B.Pharma, B.D.S., B.V.Sc. or B.E. Biotechnology students are eligible too.

Possible career options include Clinical geneticist, Genetic Counsellor, Clinical Researcher, Research Scientist, Biochemical Diagnostics Professional, Biomedical Research Assistant, Biomedical Technician, Cancer Research Scientist, Biomedical Engineer, Molecular Genetics Professional, Laboratory Technician, and Laboratory Director.

The writer is Senior Medical Director - LifeCell.

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Growing scope of Genetic Medicine and Stem Cell Research - The Hindu

DURHAM, N.C.--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company developing ARCUS-based ex vivo allogeneic CAR T and in vivo gene editing therapies, today announced it has entered into an exclusive worldwide in vivo gene editing research and development collaboration and license agreement with Novartis Pharma AG (the Agreement). As part of the Agreement, Precision will develop a custom ARCUS nuclease that will be designed to insert, in vivo, a therapeutic transgene at a safe harbor location in the genome as a potential one-time transformative treatment option for diseases including certain hemoglobinopathies such as sickle cell disease and beta thalassemia.

Under the terms of the Agreement, Precision will develop an ARCUS nuclease and conduct in vitro characterization, with Novartis then assuming responsibility for all subsequent research, development, manufacturing and commercialization activities. Novartis will receive an exclusive license to the custom ARCUS nuclease developed by Precision for Novartis to further develop as a potential in vivo treatment option for sickle cell disease and beta thalassemia. Precision will receive an upfront payment of $75 million and is eligible to receive up to an aggregate amount of approximately $1.4 billion in additional payments for future milestones. Precision is also eligible to receive certain research funding and, should Novartis successfully commercialize a therapy from the collaboration, tiered royalties ranging from the mid-single digits to low-double digits on product sales.

We are excited to collaborate with Novartis to bring together the precision and versatility of ARCUS genome editing with Novartis gene therapy expertise and commitment to developing one-time, potentially transformative treatment for hard-to-treat inherited blood disorders, said Michael Amoroso, Chief Executive Officer at Precision BioSciences. This collaboration will build on the unique gene insertion capabilities of ARCUS and illustrates its utility as a premium genome editing platform for potential in vivo drug development. With this Agreement, Precision, either alone or with world-class partners, will have active in vivo gene editing programs for targeted gene insertion and gene deletions in hematopoietic stem cells, liver, muscle and the central nervous system showcasing the distinctive versatility of ARCUS.

We identify here a collaborative opportunity to imagine a unique therapeutic option for patients with hemoglobinopathies, such as sickle cell disease and beta thalassemia a potential one-time treatment administered directly to the patient that would overcome many of the hurdles present today with other therapeutic technologies, said Jay Bradner, President of the Novartis Institutes for Biomedical Research (NIBR), the Novartis innovation engine. We look forward to working with Precision and leveraging the ARCUS technology platform, which could bring a differentiated approach to the treatment of patients with hemoglobinopathies."

The in vivo gene editing approach that we are pursuing for sickle cell disease could have a number of significant advantages over other ex vivo gene therapies currently in development, said Derek Jantz, Ph.D., Chief Scientific Officer and Co-Founder of Precision BioSciences. Perhaps most importantly, it could open the door to treating patients in geographies where stem cell transplant is not a realistic option. We believe that the unique characteristics of the ARCUS platform, particularly its ability to target gene insertion with high efficiency, make it the ideal choice for this project, and we look forward to working with our partners at Novartis to bring this novel therapy to patients.

Upon completion of the transaction, Precision expects that existing cash and cash equivalents, expected operational receipts, and available credit will be sufficient to fund its operating expenses and capital expenditure requirements into Q2 2024.

Precision BioSciences Conference Call and Webcast Information

Precision's management team will host a conference call and webcast tomorrow, June 22, 2022, at 8:00 AM ET to discuss the collaboration. The dial-in conference call numbers for domestic and international callers are (866)-996-7202 and (270)-215-9609, respectively. The conference ID number for the call is 6252688. Participants may access the live webcast on Precision's website https://investor.precisionbiosciences.com/events-and-presentations in the Investors page under Events and Presentations. An archived replay of the webcast will be available on Precision's website.

About ARCUS and Safe harbor ARCUS Nucleases

ARCUS is a proprietary genome editing technology discovered and developed by scientists at Precision BioSciences. It uses sequence-specific DNA-cutting enzymes, or nucleases, that are designed to either insert (knock-in), remove (knock-out), or repair DNA of living cells and organisms. ARCUS is based on a naturally occurring genome editing enzyme, I-CreI, that evolved in the algae Chlamydomonas reinhardtii to make highly specific cuts in cellular DNA. Precision's platform and products are protected by a comprehensive portfolio including nearly 100 patents to date.

Precision can use an ARCUS nuclease to add a healthy copy of a gene (or payload) to a persons genome. The healthy copy of the gene can be inserted at its usual site within the genome, replacing the mutated, disease-causing copy. Alternatively, an ARCUS nuclease can be used to insert a healthy copy of the gene at another site within the genome called a safe harbor that enables production of the healthy gene product without otherwise affecting the patients DNA of gene expression patterns.

About Sickle Cell Disease and Beta Thalassemia

Sickle cell disease (SCD) is a complex genetic disorder that affects the structure and function of hemoglobin, reduces the ability of red blood cells to transport oxygen efficiently and, early on, progresses to a chronic vascular disease.1-4 The disease can lead to acute episodes of pain known as sickle cell pain crises, or vaso-occlusive crises, as well as life-threatening complications.5-7 The condition affects 20 million people worldwide.8 Approximately 80% of individuals with SCD globally live in sub-Saharan Africa and it is estimated that approximately 1,000 children in Africa are born with SCD every day and more than half will die before they reach five.9,10 SCD is also a multisystem disorder and the most common genetic disease in the United States, affecting 1 in 500 African Americans. About 1 in 12 African Americans carry the autosomal recessive mutation, and approximately 300,000 infants are born with sickle cell anemia annually.11 Even with todays best available care, SCD continues to drive premature deaths and disability as this lifelong illness often takes an extreme emotional, physical, and financial toll on patients and their families.12,13

Beta thalassemia is also an inherited blood disorder characterized by reduced levels of functional hemoglobin.14 The condition has three main forms minor, intermedia and major, which indicate the severity of the disease.14 While the symptoms and severity of beta thalassemia varies greatly from one person to another, a beta thalassemia major diagnosis is usually made during the first two years of life and individuals require regular blood transfusions and lifelong medical care to survive.14 Though the disorder is relatively rare in the United States, it is one of the most common autosomal recessive disorders in the world.14 The incidence of symptomatic cases is estimated to be approximately 1 in 100,000 individuals in the general population.14, 15 The frequency of beta-thalassemia mutations varies by regions of the world with the highest prevalence in the Mediterranean, the Middle-East, and Southeast and Central Asia. Approximately 68,000 children are born with beta-thalassemia.16

About Precision BioSciences, Inc.

Precision BioSciences, Inc. is a clinical stage biotechnology company dedicated to improving life (DTIL) with its novel and proprietary ARCUS genome editing platform. ARCUS is a highly precise and versatile genome editing platform that was designed with therapeutic safety, delivery, and control in mind. Using ARCUS, the Companys pipeline consists of multiple ex vivo off-the-shelf CAR T immunotherapy clinical candidates and several in vivo gene editing candidates designed to cure genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit http://www.precisionbiosciences.com.

Forward-Looking Statements

This press release contains forward-looking statements, as may any related presentations, within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this herein and in any related presentation that do not relate to matters of historical fact should be considered forward-looking statements, including, without limitation, statements regarding the goal of providing a one time, potentially curative treatment for certain hemoglobinopathies, the success of the collaboration with Novartis, including the receipt of any milestone, royalty, or other payments pursuant to and the satisfaction of obligations under the Agreement, clinical and regulatory development and expected efficacy and benefit of our platform and product candidates, expectations about our operational initiatives and business strategy, expectations about achievement of key milestones, and expected cash runway. In some cases, you can identify forward-looking statements by terms such as aim, anticipate, approach, believe, contemplate, could, estimate, expect, goal, intend, look, may, mission, plan, potential, predict, project, should, target, will, would, or the negative thereof and similar words and expressions. Forward-looking statements are based on managements current expectations, beliefs and assumptions and on information currently available to us. Such statements are subject to a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability to become profitable; our ability to procure sufficient funding and requirements under our current debt instruments and effects of restrictions thereunder; risks associated with raising additional capital; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities, preclinical studies and clinical trials; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators ability to identify, develop and commercialize product candidates; pending and potential liability lawsuits and penalties against us or our collaborators related to our technology and our product candidates; the U.S. and foreign regulatory landscape applicable to our and our collaborators development of product candidates; our or our collaborators ability to obtain and maintain regulatory approval of our product candidates, and any related restrictions, limitations and/or warnings in the label of an approved product candidate; our or our collaborators ability to advance product candidates into, and successfully design, implement and complete, clinical or field trials; potential manufacturing problems associated with the development or commercialization of any of our product candidates; our ability to obtain an adequate supply of T cells from qualified donors; our ability to achieve our anticipated operating efficiencies at our manufacturing facility; delays or difficulties in our and our collaborators ability to enroll patients; changes in interim top-line and initial data that we announce or publish; if our product candidates do not work as intended or cause undesirable side effects; risks associated with applicable healthcare, data protection, privacy and security regulations and our compliance therewith; the rate and degree of market acceptance of any of our product candidates; the success of our existing collaboration agreements, and our ability to enter into new collaboration arrangements; our current and future relationships with and reliance on third parties including suppliers and manufacturers; our ability to obtain and maintain intellectual property protection for our technology and any of our product candidates; potential litigation relating to infringement or misappropriation of intellectual property rights; our ability to effectively manage the growth of our operations; our ability to attract, retain, and motivate key executives and personnel; market and economic conditions; effects of system failures and security breaches; effects of natural and manmade disasters, public health emergencies and other natural catastrophic events; effects of COVID-19 pandemic and variants thereof, or any pandemic, epidemic or outbreak of an infectious disease; insurance expenses and exposure to uninsured liabilities; effects of tax rules; risks related to ownership of our common stock and other important factors discussed under the caption Risk Factors in our Quarterly Report on Form 10-Q for the quarterly period ended March 31, 2022, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SECs website at http://www.sec.gov and the Investors page of our website under SEC Filings at investor.precisionbiosciences.com.

References

1 Saraf SL, et al. Paediatr Respir Rev. 2014;15(1):4-12.2 Stuart MJ, et al. Lancet. 2004;364(9442):1343-1360.3 National Institutes of Health (NIH). Sickle cell disease. Bethesda, MD. U.S. National Library of Medicine. 2018:1-7.4 Conran N, Franco-Penteado CF, Costa FF. Hemoglobin. 2009;33(1):1-16.5 Ballas SK, et al. Blood. 2012;120(18):3647-3656.6 Elmariah H, et al. Am J Hematol. 2014(5):530-535.7 Steinberg M. Management of sickle cell disease. N Engl J Med. 1999;340(13):1021-1030.8 National Heart Lung and Blood Institute: What Is Sickle Cell Disease? 9 Odame I. Perspective: We need a global solution. Nature. 2014 Nov;515(7526):S1010 Scott D. Grosse, Isaac Odame, Hani K. Atrash, et al. Sickle Cell Disease in Africa: A Neglected Cause of Early Childhood Mortality. American Journal of Preventive Medicine 41, no. S4 (December 2011): S398-40511 Sedrak A, Kondamudi NP. Sickle Cell Disease. [Updated 2021 Nov 7]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2022 Jan-.12 Sanger M, Jordan L, Pruthi S, et al. Cognitive deficits are associated with unemployment in adults with sickle cell anemia. Journal of Clinical and Experimental Neuropsychology. 2016;38(6):661-671.13 Anim M, Osafo J, Yirdong F. Prevalence of psychological symptoms among adults with sickle cell disease in Korie-Bu Teaching Hospital, Ghana. BMC Psychology. 2016;4(53):1-9.14 NORD Rare Disease Database: Beta Thalassemia 15 Galanello R, Origa R. Orphanet J Rare Dis. 2010;5:1116 Needs T, Gonzalez-Mosquera LF, Lynch DT. Beta Thalassemia. [Updated 2022 May 8]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2022 Jan-.

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Precision BioSciences Announces In Vivo Gene Editing Collaboration with Novartis to Develop Potentially Curative Treatment for Disorders Including...

It was 1984. The doctors figured out baby Gakpos red blood cells were changing from the typical doughnut shape into the shape of a half moon, and what he was experiencing was a sickle cell disease pain crisis.

I had some damage done to my feet and my legs, and could have lost my legs, Paul said. But luckily they were able to save my walking ability.

He was diagnosed with sickle cell disease and placed on a strict regimen of prophylactic penicillin, which is known to reduce the risk of infections thatcould be life-threateningto children with the genetic disorder. His doctors also had him undergo medical screenings known to improve outcomes for pediatric sickle cell patients.

Jacob Dean /Side Effects Public Media

Paul, now 37, said sickle cell disease was just part of his life growing up in Kentucky. When pain crises hit, hed end up hospitalized and have to miss school for days at a time.

His parents have always been hands-on when it comes to his health. They immigrated to the U.S. before Paul was born his dad from Ghana and his mom from Nigeria and used their advanced degrees to land careers in academia. Throughout Pauls childhood, his mom worked nights as a clinical scientist, and his dad worked the day shift as a professor. This helped ensure someone was always home to take care of sickle cell crises that could strike at any time.

It was tough, but Pauls mother, Philomena Gakpo, remembers the system was built to help. And they needed a lot of help. Pauls identical twin brother, Louis, was also diagnosed with sickle cell disease and the two took turns landing in the hospital due to their illness.

One goes in, then he comes out [and] the other one follows. So we were in the hospital like all the time, Philomena said. We had very good, compassionate pediatric physicians We didnt really know what we were doing, but we had a lot of support.

For decades, sickle cell was considered a pediatric disease because it claimed the lives of so many young children. In 1970, more than one in four children born with sickle cell anemia in the U.S. could expect to die before turning five.This changed whenCongress took actionto invest millions of dollars to establish sickle cell centers for kids and do more research.

Death rates for childrendecreased by68%in two decades. For the first time, the vast majority of sickle cell patients were surviving well into adulthood.

That also meant more sickle cell patients outgrew pediatric care and found themselves faced with a system not designed to meet their needs. Federal funding infusions led to the creation of 168 pediatric sickle cell programs across the country, compared toonly 49 sickle cell centersfor adults.

So while the situation improved dramatically for children, the same cannot be said of adults with the illness. Onepeer-reviewed study examining death rates of sickle cell patients between 1979 and 2005 found that while child death rates plummeted during that period, adult death rates increased 1% every year.

Despite their best efforts, the Gakpos would find their family represented in these heartbreaking statistics.

An unpredictable illness with many uncertainties

When the Gakpo twins turned 18, they moved away from their parents and out of the pediatric system that had cared so well for them.

They got to the adult stage with no transition. And then they were out of the house too, Philomena said.

That came at a cost for Louis Gakpo. His health took one blow after another in the years that followed.

When Louis went to the dentist for a wisdom tooth extraction, he ended up in the ICU for an 11-day stay due to complications from the surgery. While tooth extraction is an outpatient procedure for most, people with sickle cell need inpatient aftercare. Philomena said that didnt happen for Louis, since the dentist did not coordinate with Louiss hematologist.

A few months later, Philomena said Louis wasnt feeling well, but his sickle cell specialist wasnt available. So he went to his primary care provider. Without specialized doctors coordinating his care, she said things went downhill, fast.

Louis Gakpo died in 2004. He was 20 years old.

I was the last person who saw him, Philomena said. He died from complications of sickle cell. He had pneumococcal sepsis, which means he [had] been sick for a few days.

Many of the challengesfaced by people with sickle cell disease can be traced back to systemic racism. The vast majority of sickle cell patients in the U.S. are Black. The disease receives a fraction of thefederal and philanthropic dollarsthat other less-common genetic disorders receive.

This contributes to lack of data and understanding of many sickle cell complications, and a shortage of sickle cell specialists in many parts of the country.

The adult sickle cell system is tough to navigate alone. And to make matters worse, some sickle cell patients have silent brain strokes over the years that can cause cognitive challenges.

These can all affect memory and planning and the ability for patients to organize their medications and take them on a regular basis, saidDr. Brandon Hardesty, a sickle cell disease hematologist at the Indiana Hemophilia and Thrombosis Center.

The ideal approach to caring for sickle cell patients, he said, is a holistic one, involving a social worker and a psychologist. Such care is often only possible in comprehensive sickle cell centers.

But most sickle cell patients dont have access to those centers. So when they transition from pediatric to adult care, they often do so without that added layer of support.

Jacob Dean

/

Side Effects Public Media

When I show up at the hospital, Im scared

For sickle cell patients, pain crises the hallmark of the disease can happen at any time and frequency. Some people experience few pain crises during childhood, and then find sickle cell complications ramp up as they age.

Even with robust pediatric sickle cell care, many families stop seeing specialists if they find the childs symptoms become manageable, saidDr. Julie Kanter, co-director of the Lifespan Comprehensive Sickle Cell Center at the University of Alabama.

Others drop off from pediatric care due to structural barriers.

So they havent seen their hematologist since they were 10, and they start having sickle-cell-related issues when theyre 18 or 19, Kanter said. At that point, they may struggle to find a provider willing to take them on.

Then when crises arise, she said, they may have no choice but to see a non-specialist, like a family medicine physician or an emergency department doctor, who may put them on high doses of opioids for long-term use without addressing the underlying complications of their disease.

The idea that anyone should be able to treat sickle cell disease is as ridiculous to me as anyone should be able to treat breast cancer, Kanter said.

By the time someone in this position connects with an adult sickle cell hematologist, theyve lost several years of care, Kanter said, and unfortunately, often [have] multiple complications that sometimes could have been prevented.

Jacob Dean/Jacob Dean

Negative prior experiences with health care providers can also keep adults with sickle cell from seeking medical attention when they need it.

Its not uncommon to find in patients medical records notes from previous doctors that come across as disparaging and distrusting of peoples reported experiences, saidDr. Patrick McGann, the director of the combined pediatric and adult sickle cell and hemoglobinopathy program at Rhode Island Hospital and Hasbro Childrens Hospital.

Things like, patients report 10 out of 10 pain, but theyre watching TV theyre joking theyre sleeping, he said. This tells you enough of what theyre thinking about that pain and how quickly they are going to treat it. And how are they going to interact with that patient.

Paul Gakpo said there have been times he was suffering through a pain crisis and felt he wasnt taken seriously by doctors. The thought of going to the hospital causes so much anxiety that he often puts it off as long as possible, opting instead to tough it out or try home remedies.

So when I show up at the hospital, Im scared, he said. Im fearing for my life at this point, that the only option I have is to go to the emergency room and, you know, hope that I get what I need there.

Today, Paul lives in Kentucky with his wife and son. He said suddenly losing his twin brother and best friend to a disease they shared was a huge wake-up call.

Ever since Louiss death, Paul has made sure to always carry around a piece of paper with him. Its his sickle cell treatment protocol, signed off by his hematologist.

[When] I get to the ER after they triage me, I just bring out my protocol, give it to the doctors and, you know, ask them to follow this guideline, Paul said.

Hes got copies everywhere: in his car, backpack, jacket pockets. Some are worn and tattered from being carted around to so many places.

Its his way to make sure hes believed and taken care of, to minimize the chance things could go wrong.

This story comes from a reporting collaboration that includes the Indianapolis Recorder andSide Effects Public Media, a public health news initiative based at WFYI. Contact Farah at fyousry@wfyi.org. Follow on Twitter:@Farah_Yousrym.

Farahs reporting on sickle cell disease is supported by a grant from the USC Annenberg Center for Health Journalisms 2022 Impact Fund for Reporting on Health Equity and Health Systems.

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When children with sickle cell grow up, they face a system not designed for them - 89.3 WFPL News Louisville

CHICAGO, June 21, 2022 /PRNewswire/ --According to the new market research report "Stem Cell Assays Market by Type (Viability, Proliferation, Differentiation, Apoptosis), Cell Type (Mesenchymal, iPSCs, HSCs, hESCs), Product & Service (Instrument), Application (Regenerative Medicine, Clinical Research), End User - Global Forecast to 2027", published by MarketsandMarkets, the global market is projected to reach USD 4.5 billion by 2027 from USD 1.9 billion in 2022, at a CAGR of 17.7 % during the forecast period of 2022 to 2027.

Browse in-depth TOC on "Stem Cell Assays Market"393 Tables 47 Figures 331 Pages

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The stem cell assays market is expected to grow at a CAGR of 17.7% during the forecast period. The growth of the market is projected to be driven by increasing funding for stem cell research, rising demand for cell-based assays in drug discovery, and the rising incidence of cancer across the globe.

The viability/cytotoxicity assays accounted for the largest share of the type segment in the stem cell assays market in 2021.

Cell viability assays help to determine the number of live and dead cells in a culture medium. The viability/cytotoxicity assays includes various types such as tetrazolium reduction assays, resazurin cell viability assays, calcein-AM cell viability assays, and other viability/cytotoxicity assays. The segment accounted for the largest share on 2021. Increase in demand for stem cell assays in drug discovery and development is projected to drive the segment growth.

The adult stem cells segment accounted for the largest share of the cell type segment in the stem cell assays market in 2021.

The adult stem cells accounted for the largest share of the stem cell assay market. The stem cells include mesenchymal stem cells, induced pluripotent stem cells, hematopoietic stem cells, umbilical cord stem cells, and neural stem cells. Increasing demand for mesenchymal stem cells and induced pluripotent stem cells for development of stem cell based therapies and rising R&D spending are various factors projected to drive the segment growth.

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The Asia Pacific region is the fastest-growing region of the stem cell assays market in 2021.

The Asia Pacific is estimated to be the fastest-growing segment of the market. The growth of the stem cell assays markets of China and India is mostly driven by growing public-private funding to support stem cell product development and commercialization and rising prevalence of cancer & other diseases. Furthermore, growing emphasis on strategic initiatives (such as acquisitions, partnerships, and collaborations) by biopharma and biotech companies is expected to support the market growth in the region.

Key players in the stem cell assays market include Thermo Fisher Scientific Inc. (US), Merck KGaA (Germany), Danaher (US), Becton, Dickinson and Company (US), Bio-Rad Laboratories (US), PerkinElmer (US), Agilent Technologies (US), Promega Corporation (US), Cell Biolabs (US), Miltenyi Biotec (Germany), STEMCELL Technologies (Canada), Bio-Techne Corporation (US), FUJIFILM Holdings Corporation (Japan), Charles River Laboratories (US), HemoGenix Inc. (US), Lonza Group (Switzerland), Takara Bio Inc. (Japan), Creative Bioarray (US), AAT Bioquest, Inc. (US), BPS Bioscience, Inc. (US), Enzo Biochem (US), PromoCell GmbH (Germany), Biotium (US), Geno Technology (US), Abcam plc (UK), and ReachBio Research Labs (US).

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Stem Cell Therapy Market by Type (Allogeneic, Autologous), Therapeutic Application (Musculoskeletal, Wound & Injury, CVD, Autoimmune & Inflammatory), Cell Source (Adipose tissue, Bone Marrow, Placenta/Umbilical Cord) (2022 - 2026)https://www.marketsandmarkets.com/Market-Reports/stem-cell-technologies-and-global-market-48.html

Regenerative Medicine Market by Product (Cell Therapies (Autologous, Allogenic), Stemcell Therapy, Tissue-engineering, Gene Therapy), Application (Wound Care, Musculoskeletal, Oncology, Dental, Ocular), Geography - Global Forecast to 2025https://www.marketsandmarkets.com/Market-Reports/regenerative-medicine-market-65442579.html

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Stem Cell Assays Market worth $4.5 billion by 2027 - Exclusive Report by MarketsandMarkets - PR Newswire

June 21, 2022 A hematology fellow from Australia, Eddie Cliff, MPH 22, cares just as deeply about the well-being of his patients as he does about improving health systems as a whole. Hes also a writer published in the New York Times and on NPR, a Fulbright Scholar, and an advocate of healthy food and sustainability.

I grew up in Australia, where my grandmother was a nurse educator and my mom is a pediatric neurologist. Neither of them put any pressure on me to be interested in medicine, but I had some work experience with a neurologist, gastroenterologist, and a primary care physician and I found the combination of solving interesting problems and working with people appealed to me.

I fell in love with hematology by accident. I had planned to be an endocrinologist and work clinically on diabetes and obesity, with a parallel public health career focused on noncommunicable diseases and nutrition policy. Then I got rostered onto a hematology rotation, treating patients with myeloma and lymphoma with CAR T-Cell therapiesin which their own immune cells are genetically engineered to fight their cancerand I became fascinated by the science. With blood cancers and other blood disorders such as sickle cell disease, you are dealing with complex diseases, with interesting treatments, that affect people from all walks of life. Hematology also has a powerful human side; when I give a patient a scan result with good or bad news, I think carefully about how to break the news, and for each patient, we design a personalized treatment plan incorporating the values and priorities most important to them.

Working in Australia during the COVID-19 pandemic, I faced difficult conversations both on the COVID-19 wards and, subsequently, with blood cancer patients about end-of-life decisions while family members were barred from the hospital under COVID policy. I wrote about these dilemmas in an essay in JAMA Oncology titled A Decision Shared is a Decision Halved. Australias strategy for COVID was great, among the best in the world, and it saved tens of thousands of lives. Thats not to say there werent unintended consequences we perhaps didnt pay enough attention toincluding patients and families being unable to be togetherand those stories are also worth telling.

What got me into public health was hearing from leaders in the field like Atul Gawande and Michael Marmot, and getting involved with a nonprofit focused on preventing noncommunicable diseases called NCDFree, which I volunteered for during medical school at Monash University. If you look at society, the biggest killers are cardiovascular disease and stroke, and the biggest risk factor for those diseases, particularly in countries where smoking rates are low, is our food system. Big Food companies are manipulating our evolutionary drive for high-calorie, high-sugar, low-nutrition foods such as sugary drinks. I helped run a food festival called festival21 with many of the NCDFree team, led by Sandro Demaio, for 3,000 people that highlighted the intersection of food, health, and sustainability. I also got involved with the Australian Medical Students Association to advocate against things the government was doing to dismantle universal health coverage and deregulate university fees; to advocate for a soft-drink tax; and to contribute to campaigns on student mental health and blood donation.

The highlight of my time at Harvard was a course called Reimagining Global Health, taught by [the late Partners In Health cofounder] Paul Farmer, and three other leading medical anthropologists, Arthur Kleinman, Salmaan Keshavjee, and Anne Becker. What was so life-changing was the way they took concepts from anthropology and social theory and applied them to their firsthand experiences in Peru, Haiti, Rwanda, Russia, China, Fiji, and elsewhere. For low- or middle-income countries, multilateral organizations often prioritize the most cost-effective things; for example, they may say that these countries dont need cancer drugs because we need to cure them of HIV and TB first. But global health is not just about addressing perceived low-hanging fruit such as distributing mosquito nets to prevent malaria, its also about building hospitals, training doctors and nurses, and developing a health care delivery system. The course made me want figure out how we can influence global systems to ensure better access to therapies around the worldas PEPFAR [The U.S. Presidents Emergency Plan For AIDS Relief]and similar program have achieved with HIV medicinesinstead of just kind of throwing up our hands and saying this is too hard.

For my current research, I work with Harvard Medical Schools Aaron Kesselheim and his group called PORTAL (Program on Regulation, Therapeutics and Law), to help advocate for lower prices for drugs and make them more accessible. A lot of the lessons I learned about Big Food also apply to Big Pharma. Just to give an example of how pharma companies work the system to their advantage, one company has 88 patents on a drug called ibrutinib, used to treat chronic lymphocytic leukemia (CLL). The patents prevent generic competitors from entering the market so that they can keep profiting from it for as long as possible. Sometimes in medicine, the basic science about curing cancer is really emphasized and celebrated, but if weve got these drugs and people cant access them because theyre too expensive, well, then havent we failed at our jobs anyway?

I am now doing a fellowship at PORTAL looking at the intersection of blood cancer medicine and public policy, doing research on pricing and regulation for drugs and cellular therapies such as CAR T-cells. My dream job would be to be an academic hematologist who could use my clinical work to feed into research, policy, and advocacy work to make society and the health system work better for all.

Im very passionate about classical music, and I play the oboe and percussion in orchestras. They are very different types of instruments, but I love them both. The oboe is a key player in the middle of the orchestra, where you feel like you are in the thick of things, whereas with percussion you are more exposed and cant hide. It turns out that my Harvard Chan adviser, Michael Barnett, is also an oboist! Music is a very mindful activity, where you learn very early on how to block out the crying baby in the second row of the audience. When they tried to teach us mindfulness in medical school, I realized I already learned it from playing music.

Michael Blanding

photo: Alex Lebrowski

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Bringing heart and humanity to hematology | News | Harvard TH Chan School of Public Health - HSPH News

Belzutifan (Welireg), a hypoxia-inducible factor-2 alpha (HIF-2) inhibitor, demonstrated positive results when given as treatment to patients with Von Hippel-Lindau (VHL) disease and renal cell carcinoma (RCC).1,2

Previously, in August 2021, belzutifan was approved by the FDA for adult patients with VHL disease who require therapy for RCC, central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors not requiring immediate surgery.3 Since its approval, multiple studies examining the HIF-2 inhibitor have begun and made strides in the VHL and RCC spaces, including LITESPARK-001 (NCT02974738), LITESPARK-004 (NCT03401788), and LITESPARK-022 (NCT05239728).

At the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting, Eric Jonasch, MD, presented the findings of both the phase 1 LITESPARK-001 and phase 2 LITESPARK-004 studies which examined belzutifan in different patient populations.1,2

In LITESPARK-001, the efficacy and safety of belzutifan in addition to pembrolizumab (Keytruda) vs the combination of placebo plus pembrolizumab was compared as treatment in patients with clear cell RCC (ccRCC) post nephrectomy. LITESPARK-004, an open-label, phase 2 study, evaluated the efficacy and safety of belzutifan in patients with VHL disease.

While these recent studies of belzutifan continue to show promise and investigators are able to prolong survivalsomething that was not demonstrated 10 years agounmet needs remain in this space. According to Jonasch, his hope is to be able to provide patients with a complete and durable response.

In an interview with Targeted OncologyTM, Jonasch, a professor in the Department of Genitourinary Medical Oncology, Division of Cancer Medicine at The University of Texas (UT) MD Anderson Cancer Center, further discussed the 2 posters he presented in regard to belzutifan at 2022 ASCO.

Can you describe the posters you presented at ASCO this year?

Jonasch: There were 2 posters on belzutifan, which is a HIF-2 inhibitor. This is a drug that has arisen out of several decades of work, starting with the discovery of the VHL gene in 1993, then with work by doctors Peter Ratcliffe, Gregg Semenza, and William Kaelin that the led to the Nobel Prize in 2019 of how VHL regulates HIF-1 and HIF-2. This then led to the efforts to target HIF as a treatment target, the challenge there being that small molecule inhibitors of transcription factors are historically hard to develop.

The team at UT Southwestern came up with an initial foray into that which then led the development of a company called Peloton that created the precursor drugs and the current drug that is now known as belzutifan and the trials that that we presented the updates on at ASCO. They're called the LITESPARK-001 spark, which is looking at the phase 1b/2 study in RCC, and LITESPARK-004, which is the registrational study and VHL disease, which led to the approval of belzutifan in VHL disease.

Could you discuss the background and the basis of LITESPARK-001?

LITESPARK-001 is a phase 1b/2 study, which did dose-escalation to find the optimal dose of belzutifan and focused on the RCC patient population. Fifty-five patients with RCC were enrolled in this trial.

The primary end point was safety with the phase 1 study and the secondary end points were progression-free survival [PFS], objective response rate [ORR], and duration of response per RESIST 1.1. The trial was a multicenter study, and we accrued these patients relatively rapidly. This presentation is now a median 41-month follow-up of data that we have previously published.

What safety and efficacy findings were discovered in LITESPARK-001 for patients with RCC?

What was exciting was that both from a safety perspective, as well as from an efficacy perspective, the data continue to hold up. Safety shows that anemia was the most common adverse event in the metastatic advanced RCC, at 24%, patients who did have grade 3 anemia. This is an on-target effect because HIF-2 is the transcription factor that regulates urethral potent. So if you knock down HIF-2, you would expect less production, and indeed, that's what happened. This is something that's manageable either with exogenous erythropoietin blood transfusions, or dose adjustments.

Hypoxia is a relatively rare adverse event and about 13% of individuals developed that as well. Unclear mechanism, but something that's also quite manageable with dose adjustments. From an ORR perspective, we see that this has continued to hold up. The ORR in this follow-up of 41 months is 25%, as it was before, and there are some subset analyses that have been formed looking at whether or not this is different in favorable vs intermediate and poor risk. It really doesn't look like there's a major difference, it's 31% in the favorable vs 24% in the intermediate/poor risk.

The other thing we looked at was the 3 prior therapies received in this patient population, so heavily pretreated. Those who did not receive either prior IO [immunotherapy] or VEGF [inhibition], the ORR was 38%. For those who had received prior IO and VEGF, the ORR was 21%. Again, small numbers in this but still very interesting to see that this is quite effective, regardless of what prior therapies people have received.

The median PFS was 14.5 months, so from a metastatic RCC perspective, we're seeing that even as a single agent in this heavily pretreated patient population, the data continue to hold up that this drug those who defend it is active in this patient population. I'm looking forward to seeing this being an approved agent and advanced renal cell carcinoma.

What did you look at in LITESPARK-004?

LITESPARK-004 is in patients with VHL disease, and we did get approval for belzutifan in patients with VHL disease. The basis of this 61-patient study, which was published last year in the New England Journal of Medicinebut this is an immediate 29-month follow-up. With the metastatic RCC patient population, we're seeing that these data not only are holding up but they're improving with time.

The primary end point was ORR in RCC from these patients. We're seeing now that those numbers have gone up to an ORR of 59%. There is now a 3% CR [complete response] rate and a 56% partial response rate, so even better than we've seen before.

We see that of the number of patients that remain on study with nearly 30 months follow-up, 50 out of 61 patients remain on study and only 4 patients came off because of a progressive disease. Even more excitingly, we're seeing that RCCs are responding with pancreatic lesions. There's an 80% ORR. If you look at pancreatic neuroendocrine tumors, it's a 90% response rate. For CNS hemangioblastomas, there's now a 38% response rate and we're seeing CRs in all of these.

What do these findings mean for this patient population?

This is really amazing for this group of individuals who face a lifetime of surveillance, imaging, surgical intervention for these lesions as they reach various danger points. We are seeing, already in the study, that for those individuals who were on this trial across the population of study, there were about 20 procedures per year being performed on these patients. Since getting on the study in the entire population, there have only been 3 procedures. This is really game changing in terms of how many surgical procedures these patients have to be subjected to after starting those effects.

How do you think the findings of both LITESPARK-001 and LITESPARK-004 will impact the future of this space?

For patients with VHL, I think it's going to be a question of what is the ideal patient? When should we use this study? In the LITESPARK-004 study, there were no brakes built into the study, so should we treat it to maximal benefit and then take breaks? We don't know. Should we be starting this in patients as a prevention strategy? Obviously, a clinical study would have to be designed to do that. These are the sorts of questions that are being asked in the VHL world.

What are the determinants of resistance? Again, there's so few patients that have demonstrated resistance. It's hard at this point in time to answer that, but I think it's going to become easy. Unfortunately, it is easier as more people inevitably progress. I think for advanced RCC, the big questions are, how do we use this drug optimally? Do we use it as monotherapy, which I think is already likely to be effective, but can we improve this by adding a tyrosine kinase inhibitor or by adding IO? What should we be doing to really optimize the use of this drug?

Is belzutifan currently being examined in any other spaces?

It is being tested in other diseases. As of now, at least in the data that we have, there have not been extremely strong signals elsewhere. I think that is more due to a lack of data as opposed to a clear sign that it's not effective. I think the next steps are going to be finding the ideal combination [and] determining how to optimally use this drug in conjunction with other agents in the treatment landscape.

What unmet needs still exist in the advanced renal cell carcinoma space?

We need curative therapy. We are getting better at prolonging survival, which was something that 10 years ago wasn't even consistent, so we're getting there with regards to improving survival. But what we want is to be able to get people to have a CR that is durable, where we can even stop therapy for a while and allow them to enjoy a treatment-free interval. That is the new Holy Grail. Ten years ago, saying CRs would be our goal was perhaps fanciful, but now because we're seeing that in a small but significant group of patients, I think that is what we should be pushing forward to get. [And getting] that depth of response and to get that durability of response so we can have people living much longer.

What would you recommend for community oncologists working with patients in these patient populations?

If you have patients with VHL disease in your practice, my recommendation is that you work together with the clinical care centers that are around the country to optimize the management of these individuals.

For patients with advanced RCC in your practice, I would say stay tuned. This is not yet FDA approved for that indication, but I am really hopeful to see this as a choice for advanced RCC in the relatively near future.

Link:
Belzutifan Improves Survival in Patients With RCC and VHL - Targeted Oncology