Monthly Archives: November 2021

Braeden Lichti – Investing in Precision Medicine to Yield New Treatments for Neurodegenerative Diseases – PRNewswire

Posted: November 22, 2021 at 2:02 am

VANCOUVER, BC, Nov. 18, 2021 /PRNewswire/ --Advances in the collective genetic understanding of diseases, and the ability to identify disease biomarkers, is ushering in a new era of personalized medicine. Technologies such as CRISPR/Cas9 are also paving the way for improved, more tailored treatments targeted to a specific genetic marker of a disease. As our understanding of the molecular underpinnings of disease continue to improve, so, too, will the technologies at our disposal to treat them.

We've already seen the benefits of this type of personalized medicine in the cancer realm. Using a person's (or disease's) genes to drive cancer therapy is known as precision medicine. Precision medicine can help doctors identify high-risk cancer patients, choose treatment options, and evaluate treatment effectiveness. Precision medicine can also be used to prevent certain types of cancer, diagnose certain types of cancers early (leading to earlier treatment and better outcomes), and diagnose specific types of cancer more correctly.

As targeted therapies continue to advance, we will continue to see their impacts flow beyond that of the cancer realm. One area in which interest is ramping up is neurodegenerative diseases, which are chronic, progressive diseases affecting the brain and its constituent cells. Neurologic disease can be genetic, or caused by a stroke or brain tumor. Examples of neurodegenerative disease include Alzheimer's Disease, Parkinson's Disease, and Huntington's Disease. These diseases have a genetic component, with specific genes playing a role in the development and progression of disease, especially in rare forms. Neurodegenerative conditions, like cancer, are devastating and costly. Collectively, neurodegenerative conditions cost people in the United States $655 billion in 2020.

Can we apply concepts from targeted therapies developed for cancer to create better outcomes for patients suffering from neurodegenerative diseases? What's more, can precision medicine be used to treat other large unmet needs in the field of neurology, such as neuropsychiatry, pain, epilepsy, sleep disorders, and stroke?

Precision medicine in neuroscience and neurology is where many companies have dedicated their time and efforts. Three companies trading on the NASDAQ in this space that investors should research are Alnylam, Ionis Pharmaceuticals, and Regeneron.

Neuroscience research companies are clamoring to make use of the plethora of cellular and molecular biology data that is emerging about drugs and the patients who use them. There is much more information to be gleaned from diseases and patients than the genetics, which may not reveal information about the ways that genes are formally transcribed and expressed. Emerging technologies, therefore, also look at the RNA profiles of a drug response, patient, or disease state, called transcriptomics; and the set of proteins expressed by a cell, tissue, or organism, called proteomics. While a challenge with gene therapy is reimbursement by insurance providers, research is underway that can make gene therapies more common, and pave the way for more established insurance structures.

RNA targeting is an active area of research for neurodegenerative disease, with companies such as Skyhawk Therapeutics, Regeneron Pharmaceuticals, Alnylam Pharmaceuticals, and Takeda involved. By modifying genetic transcription via RNA technologies, these companies hope to develop novel treatments for disorders of the central nervous system. The study of RNA profiles in a given cell, tissue, or organism is known as transcriptomics, and this area will likely heat up as these researchers work to develop pioneering RNA technologies to target neurodegenerative disease.

Proteomics, or the study of the proteins expressed by a cell, tissue, or organism, will also play a role in precision medicine for neurological disorders. In June 2021, the United States Food and Drug Adminstration approved the first therapy addressing the underlying biology of Alzheimer's disease. The drug, Biogen's Aducanumab, is a monoclonal antibody therapy that works by clearing a substance known as beta-amyloid, a protein that scientists believe causes Alzheimer's, from the brain. The drug, which was found to exhibit a unique proteomic profile upon treatment in mice, was the first approved for Alzheimer's in 20 years, and while it is thought to be effective in a limited number of Alzheimer's disease cases (namely, people in the early stages of Alzheimer's), it represents a step forward in neurodegenerative disease research.

The FDA's approval of Aduhelm, which was under an accelerated timeframe, has created more interest in the area of Alzheimer's and Parkinson's disease treatments. Scientists believe that a protein called tau is more closely associated with dementia than beta-amyloid, so they are also seeking to develop drugs targeting tau protein. In the realm of Parkinson's disease, research is underway to target a compound called alpha-synuclein, which, like amyloid beta and tau protein in Alzheimer's, is associated with cognitive decline in Parkinson's disease. There are a number of approaches in development to target tau. Investors can expect many more biotech companies and venture firms moving into this space to develop innovative and alternative treatments.

This work is not without significant challenges. One obstacle in neurodegenerative research is creating drugs that can bypass the brain's blood-brain-barrier, which keeps the brain safe from toxic substances or pathogens that would otherwise make their way into the brain. Another challenge is the fact that neurodegeneration affects a subset of neurons, which may have different levels of vulnerability to such disease. It is not yet fully clear which factors predispose certain neurons to develop pathology over others.

Yet as drug discovery continues to leverage the latest techniques in genomics, transcriptomics, and proteomics, and combinations of these technologies, this will unlock new potential for companies to create novel, increasingly personalized, therapies. For example, advances in genomics may provide insight into how neurodegeneration occurs in the brain.

Drug discovery in neurodegeneration also overlaps with that of other diseases, due to common disease pathways. For example, phosphatidylinositol 3-Kinase (PI3K) inhibitors are implicated not only in COVID-19 and breast cancer, but also Parkinson's Disease. Stem cell therapies, which could benefit patients suffering from many conditions, can also have significant applications in the neurodegenerative realm. Stem cells could potentially be used to restore lost brain tissue, or to release compounds such as anti-inflammatory factors and growth factors supporting repair of the nervous system. Stem cell therapies, which are already in use for conditions such as cancer, could thereby restore function to neurodegenerative patients. Therefore, advances made in the treatment of other disease states could potentially innovate the field of neurodegeneration as well.

PRLog ID: http://www.prlog.org/12894142

SOURCE Braeden Lichti

Continue reading here:
Braeden Lichti - Investing in Precision Medicine to Yield New Treatments for Neurodegenerative Diseases - PRNewswire

Posted in Molecular Genetics | Comments Off on Braeden Lichti – Investing in Precision Medicine to Yield New Treatments for Neurodegenerative Diseases – PRNewswire

Exploring Microbial Interactions with Glowing Squid | Newsroom – UC Merced University News

Posted: November 22, 2021 at 2:02 am

As a young child, Professor Michele Nishiguchi would dive off the couch and into the shag carpet reefs of her family home, mimicking the turns and leaps of intrepid explorer and environmentalist Jacques Cousteau. This love of the ocean would remain with Nishiguchi as she progressed through her academic career.

Now a professor in the Department of Molecular and Cell Biology in the Schoolof Natural Sciences, her lab studies the relationship between ocean microbes and Bobtail squids.

Looking around the lab, with its ocean microbes in frosty freezer vials and precious squid specimens preserved in jars, one would think Nish and her team are marine biologists. But Nish, as she likes to be known, describes herself as an evolutionary biologist who studies the impact of microbes on organisms and how microbes interact with the world around them.

So why ocean microbes and squid?

Their relationship is dynamic and fascinating. The two organisms are so evolutionarily far apart but still able to communicate and interact with each other for mutual benefit and survival, Nish said.

At UC Merced, Nish and her team research the dynamic relationships between the Bobtail squid and Vibrio bacteria. There are approximately 20 labs nationally that use this species/host model but only Nishs lab is investigating all the different species of Vibrio and their coevolution with their squid hosts across various locations.

Just like the beneficial microbes living in the human gut and affecting digestion and nutrient uptake, Bobtail squid harbor special ocean microbes, bacteria in the genus Vibrio. These bacteria are taken up by the squid into specialized light organs, where they have a safe niche to multiply and provide the squid with a luminescent glow. Its a delicate balance that requires communication between the bacteria and the squid: Too much growth could harm the host and too little means no benefit.

Though humans dont gain luminescence, we do strengthen our gut health when we consume probiotic bacteria from foods such as yogurt or kombucha. The bacteria break down food molecules that we cant on our own. They colonize our gastrointestinal tracts and deter disease-causing, harmful microbes from establishing. As with the squid, even healthy bacteria can overgrow and cause harm, leading to gut irritation, inflammation and sometimes, chronic disease.

By studying the relationship between Bobtail squids and various Vibrio bacteria species, Nishs lab hopes to discover how communication between squid and bacteria take place, how the relationship between host and microbe evolves over time, and what genes might regulate these interactions.

Only certain species of Vibrio can thrive and colonize the Bobtail squids light organ, indicating an evolved preference for one species or strain over the other, Nish and her students discovered. Examining this relationship sheds light on how host organisms manage and select beneficial microbes and what communication mechanisms are involved.

For Nish, coming back to California meant coming full circle She is a product of the UC system and a proud recipient of three UC degrees: her bachelors degree at UC Davis, masters degree at UC San Diego, and PhD at UC Santa Cruz.

But she has been all over the world studying squid. She relocated her lab from New Mexico to UC Merced during the COVID-19 pandemic, setting up a new squid hatchery to raise the model organism, reestablishing protocols and restarting experiments from scratch. Some lab members drove the precious frozen bacteria cell strains, preserved on dry ice, from New Mexico to California.

Just as there is more than meets the eye with the Bobtail squid, Nish and her team are multifaceted, exhibiting expertise from a range of fields such as microbiology and evolutionary genetics. Nish hopes eventually, her labs findings could lay the foundation for answering questions about humans and microbes.

Perhaps, one day, the bobtail squid and Vibrio will help us answer complex questions regarding our own relationships with microbes, Nish said.

Excerpt from:
Exploring Microbial Interactions with Glowing Squid | Newsroom - UC Merced University News

Posted in Molecular Genetics | Comments Off on Exploring Microbial Interactions with Glowing Squid | Newsroom – UC Merced University News

Nancy Hopkins wins STAT Biomedical Innovation Award – STAT

Posted: November 22, 2021 at 2:02 am

Nancy Hopkins, an MIT professor who has made significant strides in molecular biology and a tireless advocate for gender equity in science, was named the recipient of STATs 2021 Biomedical Innovation Award on Tuesday.

Its very easy to forget how much progress there has been because we havent arrived where wed like to be. So we see the problems that still lie ahead. But you periodically have to pause and say, Oh, my gosh, look how far we came, said Hopkins at the 2021 STAT Summit, where she was honored for her work. The STAT award, now in its third year, honors biology and medicine researchers whose work has helped define their field. Hopkins was selected by STAT editors with input from outside experts, and received the award during the annual STAT Summit, a three-day event focused on health care. She is the second woman to receive the award, which was given last year to CRISPR researcher Jennifer Doudna.

Were in a period of dramatic advances and daunting problems, said Matthew Herper, STATs senior medicine writer and editorial director of events. Society needs every great scientist we can train. And that means that we need women every bit as much as men. Hopkins advocacy in this area has been instrumental in starting to create a more level playing field a project on which there is still much to do.

advertisement

By the mid-1990s, Hopkins had worked at MIT for 20 years, but still found herself one of a small proportion of women faculty in science at MIT. She had seen no woman professor become head of an MIT science department, center, or lab. There were no women administrators in science or engineering.

I thought it was a choice, once the door opened, Hopkins said. And I think we were all surprised to discover that behind the door that had opened were a whole series of obstacles that we really hadnt thought about.

advertisement

Over time, her illusions faded so all she could see was a perplexing problem. So she did what any good scientist does: She studied it. She surveyed female colleagues about their experiences at MIT and then went big, chairing a committee that produced landmark reports exposing gender-based discrimination, structural sexism, and pay disparities across MIT departments.

But unlike some work that emerges from higher ed committees only to languish in a file cabinet, the committees work forced MIT administrators to confront reality by making the gender inequity issue in each of its schools front and center. In response, deans and provosts aggressively recruited women for faculty positions, opened an on-campus day care and fixed pay gaps treating the problem as a structural issue instead of a case-by-case situation. The findings were highlighted in the media, prompted other institutions to interrogate themselves, and sparked conversations about pervasive discrimination against women in the world beyond academia.

If one were to ask what was the most important factor in change to date, it would have to be the Reports that documented the problems and led to the engagement of administrators in solving them, Hopkins wrote about MITs progress in a subsequent report.

Hopkins has continued to push for the inclusion and recognition of women in science, technology, engineering, and math fields. As a biology professor and researcher, she watched the biotech industry be born and boom and saw how women were systematically excluded from it, barred from contributing their expertise. So Hopkins worked with MITs Sangeeta Bhatia and Susan Hockfield, and co-founded the Boston Biotech Working Group to foster women faculty as founders and board members of biotechnology companies.

While working to make science a viable career for more women, Hopkins was also doing groundbreaking genetics research. As a young scientist, she focused on identifying RNA tumor virus genes and unraveling how they were correlated to the severity of cancers. She turned her attention a decade later to a different study of cancer, becoming one of the first scientists to use zebrafish as a model to understand how genetic mutations contribute to the development of diseases such as cancer. What started as an attempt by Hopkins to switch fields in search of a more equitable department led to major scientific breakthroughs, even though she was denied adequate space and resources for her zebrafish work.

Social change is a fascinating thing, she told STAT. Why does it take so long? Were changing peoples brains. I think these unconscious biases are so deeply embedded in our brains. Why did it take me 20 years to figure out what was going on? You come in with the belief that science is a meritocracy.

Hopkins is a member of the National Academy of Sciences and the National Academy of Medicine, and has also been awarded the Harvard Centennial Medal, UCSF Medal, and MD Andersons Margaret L. Kripke Legend Award, among other honors. She is a 2021 fellow with the American Association for Cancer Research Academy.

See the original post here:
Nancy Hopkins wins STAT Biomedical Innovation Award - STAT

Posted in Molecular Genetics | Comments Off on Nancy Hopkins wins STAT Biomedical Innovation Award – STAT

Stem Cell Therapy Boosts Outcomes for Some Heart Failure Patients – HealthDay

Posted: November 22, 2021 at 1:58 am

TUESDAY, Nov. 16, 2021 (HealthDay News) -- Heart failure patients who fit a specific profile can benefit from injection of stem cells delivered directly into their heart muscle, a new study finds.

Patients with mild or moderate heart failure who have high levels of inflammation responded well to the stem cell injections, and experienced a decline in their risk of heart attacks, strokes and heart-related death, clinical trial results show.

Stem cells injected into targeted areas of a failing heart become activated by inflammation and start pumping out beneficial biochemicals, explained lead researcher Dr. Emerson Perin, medical director of the Texas Heart Institute in Houston.

"These cells are little factories of different proteins, cytokines and other products that then have an effect locally on the heart muscle cells," Perin said, adding that the cells also help improve the health of blood vessels both large and small.

For this clinical trial, Perin and his colleagues recruited 537 people suffering from advanced heart failure with reduced ejection fraction, which is when the main pumping chamber in the left side of the heart is significantly weakened.

Half of the patients chosen at random received 150 million stem cells into targeted areas of still-working heart muscle, delivered though 15 to 20 injections in a single procedure, Perin said.

The areas were selected using a mapping system that found places in the heart where electrical activity still occurred but might be hampered by inflammation.

The therapy did not significantly reduce the number of hospitalizations caused by heart failure, but the researchers found that it did improve participants' heart health in other ways during an average 30 months of follow-up:

These results show that a personalized approach with stem cell therapy can help some with heart failure, Perin said.

Doctors have the ability to single out patients with high levels of inflammation and either mild or moderate heart failure, "and then we're using a very precise way of delivering these cells in each individual patient exactly where they need to be placed," Perin said.

The findings were presented Sunday at the American Heart Association's online annual meeting. Research presented at medical meetings is considered preliminary until published in a peer-reviewed journal.

This new clinical trial "is really a promising study to provide further insights into potential subgroups of patients who may hopefully benefit from stem cell therapy," said Dr. Biykem Bozkurt, director of the Winters Center for Heart Failure at the Baylor College of Medicine in Houston.

Bozkurt said future studies should be able to further refine and identify exactly who would most benefit from stem cell therapy among heart failure patients.

"There's always definitely a need to do more research," Bozkurt said.

More information

The Cleveland Clinic has more about heart failure.

SOURCES: Emerson Perin, MD, PhD, medical director, Texas Heart Institute, Houston; Biykem Bozkurt, MD, PhD, director, Winters Center for Heart Failure, Baylor College of Medicine, Houston; American Heart Association annual meeting, Nov. 14, 2021, online presentation

Here is the original post:
Stem Cell Therapy Boosts Outcomes for Some Heart Failure Patients - HealthDay

Posted in Texas Stem Cells | Comments Off on Stem Cell Therapy Boosts Outcomes for Some Heart Failure Patients – HealthDay

BioBridge Global continues history of innovation with umbilical cord collection and processing under cGMP standards for further manufacture – Yahoo…

Posted: November 22, 2021 at 1:58 am

Stem cells from cord tissue to be used to develop new cellular therapies

San Antonio, Texas, Nov. 18, 2021 (GLOBE NEWSWIRE) -- BioBridge Global is committed to supporting advanced therapies through the development and delivery of services from source material collection, donor and product testing, development and manufacturing services and logistics management. This week, the organization fulfilled a promise to customers to collect, deliver and process an umbilical cord under current good manufacturing process (cGMP) standards for further manufacture into advanced therapies.

BioBridge Global has been collecting, testing, and delivering material under cGMP and/or cGTP for clinical use for more than 45 years. Birth tissues were added to that portfolio in 2016 for use in graft applications by numerous tissue processors. Therapeutic developers of advanced therapies have been requesting tissue under cGMP for further manufacture for several years, having found no consistent and reliable source in the United States.

The complexities associated with donor qualification, tissue collection and tissue processing for umbilical cord tissue are far greater under these further manufacturing standards. Donor mothers must be exceptionally healthy, already have been identified to undergo a planned c-section, and then consented under an IRB-approved consent. The cord tissue, collected during the birth process, is then tested independently to ensure sterility of the product going into therapeutic development. To deliver the best possible therapeutic product, the cord must undergo very specific preparation steps in a clean room environment before it is released for life-saving therapies. The processed cord, which is rich in stem cells, can eventually be manufactured into a combination of either intermediates such as a master cell bank or end-product advanced therapies to deliver directly to a patient.

South Texas Blood & Tissue collected the birth tissue from a donor who was determined eligible based on custom specifications as well as significant regulatory agency requirements for tissues used for further manufacture. QualTex Laboratories performed donor screening and sterility testing on the cell product, and GenCure conducted time-sensitive aseptic processing to ensure that the tissue-derived cell product could be preserved for maximum benefit for therapeutic development.

Story continues

South Texas Blood & Tissue, QualTex Laboratories and GenCure are subsidiaries of San Antonio-based nonprofit BioBridge Global.

This is a significant accomplishment for the entire organization and the teams involved, said Martin Landon, Chief Executive Officer of BioBridge Global. To the best of our knowledge, based on our review of the market and customer feedback, there is no source material provider in the United States delivering qualified birth tissue under cGMP for further manufacture into advanced therapies.

Other organizations have programs using cells derived from umbilical cord, and various sources are available for early-stage research. But there were no consistent sources of cGMP collections and processing for further manufacture until BioBridge Global made its delivery. The market demand came to BioBridge Globals attention when it developed its biomanufacturing service line under GenCure. A number of customers had approached GenCure, knowing about BioBridge Globals donor material collection services, requesting development of processes to support collection and production of a cGMP cord product.

Dr. Rachel Beddard, Chief Medical Officer at Biobridge Global, led the development of the process. This achievement is a perfect example of the cross-collaboration within our organization. South Texas Blood & Tissue, QualTex and GenCure joined efforts to set-up a program to provide umbilical cord tissue collected, tested, and aseptically processed, following GMP standards and compliant with U.S. and international regulatory requirements for further manufacture into advanced therapies.

Congratulations to the team that helped design and implement the collection and testing procedures, execute on those procedures to collect, test, and process the cord, Landon said. We are still steps away from therapies that will save and enhance lives, but we have built a foundation where none existed before.

BioBridge Global and its subsidiaries have been collecting birth tissue, including umbilical cords, since 2016 and umbilical cord blood for therapeutic use since 2001. All donors consent to IRB-approved collection protocols authorizing the use of their donation for research, clinical, or commercial applications.

About BioBridge Global:

BioBridge Global (BBG) is a San Antonio, Texas-based 501(c)(3) nonprofit regenerative medicine enterprise that offers diverse services through its subsidiaries South Texas Blood & Tissue, QualTex Laboratories, GenCure and The Blood & Tissue Center Foundation. BBG provides products and services in blood resource management, cellular therapy, donated umbilical cord blood and human tissue as well as testing of blood, plasma and tissue products for clients in the United States and worldwide. BBG is committed to saving and enhancing lives through the healing power of human cells and tissue. It enables advances in the field of regenerative medicine by providing access to human cells and tissue, testing services and biomanufacturing and clinical trials support. Learn more at BioBridgeGlobal.org.

See the article here:
BioBridge Global continues history of innovation with umbilical cord collection and processing under cGMP standards for further manufacture - Yahoo...

Posted in Texas Stem Cells | Comments Off on BioBridge Global continues history of innovation with umbilical cord collection and processing under cGMP standards for further manufacture – Yahoo…

Quizartinib Added to Chemotherapy Demonstrates Superior Overall Survival Compared to Chemotherapy Alone in Adult Patients with Newly Diagnosed…

Posted: November 22, 2021 at 1:58 am

TOKYO & MUNICH & BASKING RIDGE, N.J.--(BUSINESS WIRE)--Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) today announced positive topline results from the global pivotal QuANTUM-First phase 3 trial evaluating quizartinib, a highly potent and selective FLT3 inhibitor, in patients with newly diagnosed FLT3-ITD positive acute myeloid leukemia (AML).1

QuANTUM-First met its primary endpoint, demonstrating that patients who received quizartinib in combination with standard induction and consolidation chemotherapy and then continued with single agent quizartinib had a statistically significant and clinically meaningful improvement in overall survival (OS) compared to those who received standard treatment alone. The safety of quizartinib was shown to be manageable and consistent with the known safety profile.

AML is one of the most common forms of leukemia in adults, representing about one-third of all cases.2 The five-year survival rate of AML is about 29%, and patients with FLT3-ITD positive AML have a particularly unfavorable prognosis, including an increased risk of relapse and shorter overall survival.1,3 There remains a high unmet need to improve survival for the majority of patients with AML.4

The results of the phase 3 QuANTUM-First trial showed that adding quizartinib, a potent and selective FLT3 inhibitor, to chemotherapy significantly prolonged overall survival in patients with newly diagnosed FLT3-ITD positive AML, said Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo. We look forward to sharing the QuANTUM-First data with the hematology community and will initiate discussions with global regulatory authorities.

Data from QuANTUM-First will be presented at an upcoming medical meeting and shared with regulatory authorities globally.

About QuANTUM-First

QuANTUM-First is a randomized, double-blind, placebo-controlled, multi-center global phase 3 study evaluating quizartinib in combination with standard induction and consolidation chemotherapy and then as continued single agent therapy in adult patients (age 18 75) with newly diagnosed FLT3-ITD positive AML.

Patients were randomized 1:1 into two treatment groups to receive quizartinib or placebo in combination with standard anthracycline and cytarabine-based induction and consolidation regimens. Eligible patients, including those who underwent allogenic hematopoietic stem cell transplant (HSCT), continued with single agent quizartinib or placebo for up to 36 cycles.

The primary study endpoint is OS. Secondary endpoints include event-free survival (EFS), post-induction rates of complete remission (CR) and composite complete remission (CRc), and the percentage of patients who achieve CR or CRc with FLT3-ITD minimal residual disease negativity. Safety and pharmacokinetics, along with exploratory efficacy and biomarker endpoints, were also evaluated.

QuANTUM-First enrolled 539 patients at approximately 200 study sites worldwide including in Asia, Europe, North America, Oceania and South America. For more information, visit ClinicalTrials.gov.

About Acute Myeloid Leukemia (AML)

More than 474,500 new cases of leukemia were reported globally in 2020 with more than 311,500 deaths.5 AML is one of the most common types of leukemia in adults, representing about one-third of all cases.2 A heterogenous blood cancer, AML is characterized by a five-year survival rate of about 29%, the lowest by far among the major leukemia subtypes.6,7

Treatment guidelines for patients with newly diagnosed AML recommend a cytarabine-based chemotherapy regimen with or without a targeted therapy as determined by the presence of genetic mutations, age and other factors.8 Patients with newly diagnosed FLT3 mutated AML may receive a FLT3 inhibitor as part of their initial treatment regimen and/or subsequent regimens.8 While intensive chemotherapy and/or HSCT can improve chances for sustained remission in eligible patients, a substantial proportion of patients are not suitable for either intervention, and cure rates are particularly low for older patients.1,6 In recent years, new targeted treatments have increased options and improved outcomes for some patients with molecularly defined AML subtypes.6

About FLT3-ITD

FLT3 (FMS-like tyrosine kinase 3) is a transmembrane receptor tyrosine kinase protein normally expressed by hematopoietic stem cells; FLT3 plays an important role in cell development by promoting cell survival, growth and differentiation through various signaling pathways.1 Mutations of the FLT3 gene, which occur in approximately 30% of patients with AML, can drive oncogenic signaling.1 The most common type of FLT3 mutation is the FLT3-ITD (internal tandem duplication), which is present in about 25% of all AML patients and contributes to cancer cell proliferation.1 Patients with FLT3-ITD mutations have a particularly unfavorable prognosis, including an increased risk of relapse and shorter overall survival.1

About Quizartinib

Quizartinib, an oral, highly potent and selective type II FLT3 inhibitor, is in phase 1/2 clinical development in pediatric and young adult patients with relapsed/refractory FLT3-ITD AML in Europe and North America.1 Several phase 1/2 combination studies with quizartinib are also underway at The University of Texas MD Anderson Cancer Center as part of a strategic research collaboration focused on accelerating development of Daiichi Sankyo pipeline therapies for AML.

Quizartinib is currently approved for use in Japan under the brand name VANFLYTA for the treatment of adult patients with relapsed/refractory FLT3-ITD AML, as detected by an approved test. Quizartinib is an investigational medicine in all countries outside of Japan.

About Daiichi Sankyo Oncology

The oncology portfolio of Daiichi Sankyo is powered by our team of world-class scientists that push beyond traditional thinking to create transformative medicines for people with cancer. Anchored by our DXd antibody drug conjugate (ADC) technology, our research engines include biologics, medicinal chemistry, modality and other research laboratories in Japan, and Plexxikon Inc., our small molecule structure-guided R&D center in the U.S. We also work alongside leading academic and business collaborators to further advance the understanding of cancer as Daiichi Sankyo builds towards our ambitious goal of becoming a global leader in oncology by 2025.

About Daiichi Sankyo

Daiichi Sankyo is dedicated to creating new modalities and innovative medicines by leveraging our world-class science and technology for our purpose to contribute to the enrichment of quality of life around the world. In addition to our current portfolio of medicines for cancer and cardiovascular disease, Daiichi Sankyo is primarily focused on developing novel therapies for people with cancer as well as other diseases with high unmet medical needs. With more than 100 years of scientific expertise and a presence in more than 20 countries, Daiichi Sankyo and its 16,000 employees around the world draw upon a rich legacy of innovation to realize our 2030 Vision to become an Innovative Global Healthcare Company Contributing to the Sustainable Development of Society. For more information, please visit http://www.daiichisankyo.com.

References

1 Daver N et al. Leukemia. (2019) 33:299312.2 American Cancer Society. Key Statistics for Acute Myeloid Leukemia. Updated January 2020.3 Leukemia and Lymphoma Society. Facts and Statistics. Leukemia: Survival (SEER Data for 2009-2015).4 Daver N et al. Blood Cancer Journal. (2020) 10:107.5 Global Cancer Observatory. Population Fact Sheet: World. Updated November 2020.6 Short et al. Cancer Discov. (2020);10:50625.7 Leukemia and Lymphoma Society. Facts and Statistics. Leukemia: Survival (SEER Data for 2009-2015).8 NCCN Practice Guidelines for Oncology. Acute Myeloid Leukemia. Version 3.2021 (March 2, 2021).

Go here to read the rest:
Quizartinib Added to Chemotherapy Demonstrates Superior Overall Survival Compared to Chemotherapy Alone in Adult Patients with Newly Diagnosed...

Posted in Texas Stem Cells | Comments Off on Quizartinib Added to Chemotherapy Demonstrates Superior Overall Survival Compared to Chemotherapy Alone in Adult Patients with Newly Diagnosed…

T2 Biosystems Announces Continued International Expansion into Taiwan with Exclusive Distribution Agreement

Posted: November 22, 2021 at 1:57 am

LEXINGTON, Mass., Nov. 19, 2021 (GLOBE NEWSWIRE) -- T2 Biosystems, Inc. (NASDAQ:TTOO), a leader in the rapid detection of sepsis-causing pathogens, today announced the execution of a territory exclusive distribution agreement in Taiwan.

More here:
T2 Biosystems Announces Continued International Expansion into Taiwan with Exclusive Distribution Agreement

Posted in Global News Feed | Comments Off on T2 Biosystems Announces Continued International Expansion into Taiwan with Exclusive Distribution Agreement

Context Therapeutics® to Present Clinical Data on ONA-XR in Breast Cancer at 2021 San Antonio Breast Cancer Symposium

Posted: November 22, 2021 at 1:57 am

Primary results from ONAWA (SOLTI-1802) trial of ONA-XR in early breast cancer to be presented in addition to updates from ongoing clinical trials of ONA-XR in metastatic breast cancer Primary results from ONAWA (SOLTI-1802) trial of ONA-XR in early breast cancer to be presented in addition to updates from ongoing clinical trials of ONA-XR in metastatic breast cancer

Read the rest here:
Context Therapeutics® to Present Clinical Data on ONA-XR in Breast Cancer at 2021 San Antonio Breast Cancer Symposium

Posted in Global News Feed | Comments Off on Context Therapeutics® to Present Clinical Data on ONA-XR in Breast Cancer at 2021 San Antonio Breast Cancer Symposium

European Commission approves Roche’s Gavreto (pralsetinib) for the treatment of adults with RET fusion-positive advanced non-small cell lung cancer

Posted: November 22, 2021 at 1:57 am

Basel, 19 November 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Commission (EC) has granted conditional marketing authorisation for Gavreto® (pralsetinib) as a monotherapy for the treatment of adults with rearranged during transfection (RET) fusion-positive advanced non-small cell lung cancer (NSCLC) not previously treated with a RET inhibitor. Gavreto is the first and only precision medicine approved in the European Union (EU) for the first-line treatment of people with RET fusion-positive advanced NSCLC.1

Go here to see the original:
European Commission approves Roche’s Gavreto (pralsetinib) for the treatment of adults with RET fusion-positive advanced non-small cell lung cancer

Posted in Global News Feed | Comments Off on European Commission approves Roche’s Gavreto (pralsetinib) for the treatment of adults with RET fusion-positive advanced non-small cell lung cancer

PAOG Announces Pending Acquisition To Expand CBD IP Portfolio Scheduled For Early December

Posted: November 22, 2021 at 1:57 am

Sandusky, OH, Nov. 19, 2021 (GLOBE NEWSWIRE) -- PAO Group, Inc. (OTC Pink: PAOG) today announced the company has finalized terms for the acquisition of a license to a patented cannabis extraction process that will expand the company’s existing CBD intellectual property.

See the rest here:
PAOG Announces Pending Acquisition To Expand CBD IP Portfolio Scheduled For Early December

Posted in Global News Feed | Comments Off on PAOG Announces Pending Acquisition To Expand CBD IP Portfolio Scheduled For Early December